S-1/A
As filed with the Securities and Exchange Commission on
June 19, 2006.
Registration
No. 333-134191
UNITED STATES SECURITIES AND EXCHANGE COMMISSION
Washington, D.C. 20549
Amendment No. 1
to
Form S-1
REGISTRATION STATEMENT
UNDER
THE SECURITIES ACT OF 1933
AMICUS THERAPEUTICS, INC.
(Exact Name of Registrant as Specified in its Charter)
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Delaware |
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2834 |
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20-0422823 |
(State or Other Jurisdiction of
Incorporation or Organization) |
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(Primary Standard Industrial
Classification Code Number) |
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(I.R.S. Employer
Identification Number) |
6 Cedar Brook Drive
Cranbury, New Jersey 08512
(609) 662-2000
(Address, including zip code, and telephone number,
including area code, of Registrants principal executive
offices)
John F. Crowley
Chief Executive Officer
Amicus Therapeutics, Inc.
6 Cedar Brook Drive
Cranbury, New Jersey 08512
(609) 662-2000
(Name, address, including zip code, and telephone number,
including area code, of agent for service)
Copies to:
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Julio E. Vega
Bingham McCutchen LLP
150 Federal Street
Boston, Massachusetts 02110-1726
(617) 951-8000 |
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Patrick OBrien
Ropes & Gray LLP
One International Place
Boston, Massachusetts 02110-1726
(617) 951-7000 |
Approximate date of commencement of proposed sale to the
public: As soon as practicable after this Registration
Statement is declared effective.
If any of the securities being registered on this Form are to be
offered on a delayed or continuous basis pursuant to
Rule 415 under the Securities Act of 1933, as amended (the
Securities Act), please check the following
box. o
If this Form is filed to register additional securities for an
offering pursuant to Rule 462(b) under the Securities Act,
please check the following box and list the Securities Act
registration statement number of the earlier effective
registration statement for the same offering.
o
If this Form is a post-effective amendment filed pursuant to
Rule 462(c) under the Securities Act, please check the
following box and list the Securities Act registration statement
number of the earlier effective registration statement for the
same
offering. o
If this form is a post-effective amendment filed pursuant to
Rule 462(d) under the Securities Act, please check the
following box and list the Securities Act registration statement
number of the earlier effective registration statement for the
same
offering. o
The Registrant hereby amends this Registration Statement on
such date or dates as may be necessary to delay its effective
date until the Registrant shall file a further amendment which
specifically states that this Registration Statement shall
thereafter become effective in accordance with Section 8(a)
of the Securities Act or until this Registration Statement shall
become effective on such date as the Commission, acting pursuant
to said Section 8(a), may determine.
The
information contained in this prospectus is not complete and may
be changed. We may not sell these securities until the
registration statement filed with the Securities and Exchange
Commission is effective. This prospectus is not an offer to sell
these securities and we are not soliciting offers to buy these
securities in any jurisdiction where the offer or sale is not
permitted.
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PROSPECTUS (Subject to Completion)
Issued June 19, 2006
Shares
COMMON STOCK
Amicus Therapeutics, Inc. is
offering shares
of its common stock. This is our initial public offering, and no
public market currently exists for our shares. We anticipate
that the initial public offering price will be between
$ and
$ per
share.
We have applied to have our common stock approved for
quotation on The Nasdaq National Market under the symbol
AMTX.
Investing in our common stock involves risks. See
Risk Factors beginning on page 8.
PRICE
$
A SHARE
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Underwriting |
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Price to |
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Discounts and |
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Proceeds to |
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Public |
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Commissions |
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Amicus |
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Per Share
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$ |
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$ |
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Total
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$ |
We have granted the underwriters the right to purchase up to an
additional shares
of common stock to cover over-allotments.
The Securities and Exchange Commission and state securities
regulators have not approved or disapproved these securities or
determined if this prospectus is truthful or complete. Any
representation to the contrary is a criminal offense.
Morgan Stanley & Co. Incorporated expects to deliver
the shares to purchasers
on ,
2006.
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MORGAN STANLEY |
GOLDMAN, SACHS & CO. |
PACIFIC GROWTH EQUITIES, LLC
,
2006
Table of Contents
You should rely only on the information contained in this
prospectus. We have not authorized anyone to provide you with
information different from that contained in this prospectus. We
are offering to sell, and seeking offers to buy, shares of our
common stock only in jurisdictions where offers and sales are
permitted. The information contained in this prospectus is
accurate only as of the date of this prospectus, regardless of
the time of delivery of this prospectus or of any sale of our
common stock. In this prospectus, unless otherwise stated or the
context otherwise requires, references to Amicus
Therapeutics, Amicus, we,
us, our and similar references refer to
Amicus Therapeutics, Inc.
Until ,
2006, 25 days after the commencement of this offering, all
dealers that buy, sell or trade shares of our common stock,
whether or not participating in this offering, may be required
to deliver a prospectus. This is in addition to the
dealers obligation to deliver a prospectus when acting as
underwriters and with respect to their unsold allotments or
subscriptions.
For investors outside the United States: Neither we nor any of
the underwriters have done anything that would permit this
offering or possession or distribution of this prospectus in any
jurisdiction where action for that purpose is required, other
than in the United States. You are required to inform yourselves
about and to observe any restrictions relating to this offering
and the distribution of this prospectus.
i
PROSPECTUS SUMMARY
This summary highlights selected information contained
elsewhere in this prospectus. This summary may not contain all
of the information that is important to you. Before investing in
our common stock, you should read this prospectus carefully in
its entirety, especially the risks of investing in shares of our
common stock that we discuss in the Risk Factors
section of this prospectus beginning on page 7 and our
financial statements and related notes beginning on
page F-1.
AMICUS THERAPEUTICS, INC.
Our Company
We are a clinical-stage biopharmaceutical company focused on the
discovery, development and commercialization of novel small
molecule, orally-administered drugs, known as pharmacological
chaperones, for the treatment of a range of human genetic
diseases. Since our founding in 2002, we have generated three
product development programs: Amigal for Fabry disease, AT2101
for Gaucher disease and AT2220 for Pompe disease. Fabry, Gaucher
and Pompe are relatively rare disorders but represent
substantial commercial markets due to the severity of the
symptoms and the chronic nature of the diseases. The worldwide
net product sales for the four approved therapeutics to treat
Fabry and Gaucher disease were more than $1.3 billion in
2005, as publicly reported by companies that market these
therapeutics.
We are currently conducting Phase II clinical trials of
Amigal and have observed results in the first four patients
after 12 weeks of treatment that suggest that treatment
with Amigal causes an increase in the activity of the enzyme
deficient in Fabry disease. These results do not necessarily
predict final results for our Phase II clinical trials, and
subsequent results from these clinical trials or future clinical
trials may not corroborate the results we have observed to date.
We expect to complete enrollment in our current Phase II
trials for Amigal by the end of 2006 and, assuming positive
results, we intend to initiate a Phase III trial in 2007.
We have initiated a Phase I trial for AT2101 and plan to
file an investigational new drug application, or IND, for AT2220
by the end of 2006.
Human genetic diseases result from mutations in specific genes
that, in many cases, lead to the production of proteins with
reduced stability. Proteins with these mutations may not achieve
their correct three-dimensional shape and are generally referred
to as misfolded proteins. The cell ensures that proteins are
folded into their correct shape before they can move from where
they are made, the endoplasmic reticulum, or ER, to the
appropriate destination in the cell, a process referred to as
protein trafficking. Proteins that do not achieve their correct
shape are often eliminated by the cell, resulting in reduced
biological activity that can lead to impaired cellular function
and ultimately to disease. In certain instances, misfolded
proteins can accumulate in the ER instead of being eliminated.
This accumulation of misfolded proteins may lead to various
types of stress on cells, which may also contribute
significantly to cellular dysfunction and disease.
Our novel approach to the treatment of human genetic diseases
consists of using a pharmacological chaperone that selectively
binds to the target protein, which increases the stability of
the protein and helps it fold into its correct three-dimensional
shape. This restores appropriate trafficking of the protein,
thereby increasing protein activity, improving cellular function
and reducing stress on cells.
The current standard of treatment for Fabry, Gaucher and Pompe
is enzyme replacement therapy. This therapy compensates for the
reduced level of activity of specialized proteins called enzymes
through regular infusions. Instead of adding enzyme from an
external source by intravenous infusion, our approach uses small
molecule, orally-administered pharmacological chaperones to
restore the function of the enzyme that is already made by the
patients own body. We believe our product candidates may
have advantages relative to enzyme replacement therapy relating
to biodistribution, treatment effect and ease of use,
1
potentially improving treatment of these diseases. In addition,
we believe our technology may be broadly applicable to other
diseases that have been linked to misfolded proteins, including
certain types of neurological disease, metabolic disease,
cardiovascular disease and cancer.
Our Lead Programs
Our three most advanced product development programs target
lysosomal storage disorders, which are chronic genetic diseases
that frequently result in severe symptoms. Each of these
disorders results from the deficiency of a single enzyme.
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Amigal for Fabry disease. We are developing Amigal for
the treatment of patients with Fabry disease, which commonly
causes kidney failure, cardiac abnormalities and progressive
neurological complications. We are currently conducting multiple
Phase II clinical trials of Amigal. We expect to complete
enrollment in our current Phase II trials by the end of
2006 and, assuming positive results, we intend to initiate a
Phase III trial in 2007. |
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AT2101 for Gaucher disease. We are developing AT2101 for
the treatment of Gaucher disease, which commonly causes an
enlarged liver and spleen, low levels of red blood cells and
platelets, bone pain and fractures. Some patients also present
with neurological complications. In preclinical studies,
administration of AT2101 resulted in a dose-related increase in
the activity of the enzyme known to be deficient in Gaucher
disease. We filed an IND for AT2101 in April 2006 and have
initiated a Phase I trial. If our Phase I trials are
successful, we plan to initiate a Phase II trial in the
first half of 2007. |
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AT2220 for Pompe disease. We are developing AT2220 for
the treatment of Pompe disease, which commonly causes
progressive muscle weakness, particularly affecting breathing,
mobility and heart function. In preclinical studies,
administration of AT2220 resulted in an increase in the activity
of the enzyme known to be deficient in Pompe disease. We plan to
file an IND for AT2220 in the second half of 2006. |
Initial Data from our Phase II Studies in Fabry
Disease
We are currently conducting multiple open-label Phase II
clinical trials in up to 48 adult male and female patients
with Fabry disease. The initial data from the first four Fabry
disease patients enrolled in one of our Phase II trials
showed that after six weeks of treatment the activity of
alpha-galactosidase A, or
a-GAL, the enzyme
deficient in Fabry disease, was on average more than five-fold
higher in white blood cells than before treatment. The four
patients had three different genetic mutations and we observed
an increase in the level of
a-GAL
enzyme activity in all of these patients.
After the initial six weeks of treatment, in accordance with the
protocol, the dose was decreased to the same dose used during
the first two weeks of the study. Patients received this lower
dose for an additional six weeks. The initial data obtained
after 12 weeks of treatment show
that
a-GAL enzyme activity in
white blood cells remained elevated at levels approximately
four-fold higher than before treatment. In two of the four
patients,
a-GAL enzyme activity, as
measured in biopsies of the skin, increased after 12 weeks
of treatment, compared to levels before treatment. Results
of
a-GAL enzyme activity
levels in skin biopsies of the other two patients were
inconclusive due to insufficient sample size.
GL-3 levels in patient
plasma, urine and skin, both before and after 12 weeks of
treatment, were in the normal range of healthy individuals.
GL-3, a complex lipid
called globotriaosylceramide, is the substrate broken down
by
a-GAL.
In all four patients, the measures of cardiac, renal and
central nervous system function before treatment were normal or
near normal, and no clinically meaningful changes were observed
after 12 weeks of treatment.
Amigal was well-tolerated with no reported serious adverse
events. Adverse events in the first four patients were mostly
mild and reported by the investigators as unlikely to be related
to Amigal. We note that a fifth patient with a history of
hypertension withdrew from the trial due to increased blood
pressure, which was reported by the investigator as possibly
related to the study drug.
2
We believe the results from the 12 weeks of treatment of
the first four Fabry patients support the continuation of our
current Phase II clinical trials. It is generally believed
that even small increases in lysosomal enzyme activity may have
clinical benefits. These results do not necessarily predict
final results for our Phase II clinical trials. The results
from additional patients in our current Phase II clinical
trials or additional data from these first four patients may
cause the final results of our Phase II clinical trials to
differ from or be less favorable than the results observed to
date in the first four patients.
Our Strategy
Our goal is to become a leading biopharmaceutical company
focused on the discovery, development and commercialization of
pharmacological chaperone therapies for the treatment of a wide
range of human genetic diseases. The introduction of
pharmacological chaperones as a treatment option has the
potential to address significant unmet medical needs and improve
the quality of life for patients.
To achieve this goal, we intend to:
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focus our initial efforts on developing pharmacological
chaperones for severe genetic diseases; |
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rapidly advance our lead programs; |
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leverage our proprietary approach to discover and develop
additional small molecules; and |
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build a targeted sales and marketing infrastructure. |
Our success in achieving our goal, however, depends in part on
the risks and uncertainties described in this prospectus in the
section entitled Risk Factors, including, without
limitation, those relating to our ability to conduct preclinical
and clinical trials that demonstrate safety and efficacy of our
product candidates, our ability to obtain regulatory approvals
and our ability to attract and retain effective sales and
marketing personnel.
Risks Associated with Our Business
Our business is subject to a number of risks of which you should
be aware before making an investment decision. We discuss these
risks more fully in the Risk Factors section of this
prospectus immediately following this prospectus summary. We
have a limited operating history and have not yet commercialized
any products. We have incurred substantial operating losses in
each year since inception. Our net loss attributable to common
stockholders was $19.8 million and $7.7 million for
the year ended December 31, 2005 and three month period
ended March 31, 2006, respectively. As of March 31,
2006, we had an accumulated deficit of $44.7 million. We
expect to incur significant and increasing net losses for at
least the next several years. It is uncertain whether any of our
product candidates under development will become effective
treatments. All of our product candidates are undergoing
clinical trials or are in earlier stages of development, and
failure in the development of new drugs is common and can occur
at any stage of development. None of our product candidates has
received regulatory approval for commercialization, and we do
not expect that any drugs resulting from our research and
development efforts will be commercially available for a number
of years, if at all. We may never generate any revenues or
achieve profitability.
Our Corporate Information
We were incorporated under the laws of the State of Delaware on
February 4, 2002. Our principal executive offices are
located at 6 Cedar Brook Drive, Cranbury, New Jersey 08512,
and our telephone number is
(609) 662-2000.
Our website address is www.amicustherapeutics.com. The
information on, or that can be accessed through, our website is
not part of this prospectus. We have included our website
address in this prospectus solely as an inactive textual
reference.
The names Amigal and Amicus and the
Amicus logo are our trademarks.
Fabrazyme®,
Cerezyme®,
Myozyme®,
Replagal and
Zavesca®
are the property of their respective owners.
3
THE OFFERING
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Common stock we are offering |
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shares |
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Common stock to be outstanding after this offering |
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shares |
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Over-allotment option |
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shares |
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Use of proceeds |
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We estimate that the net proceeds from this offering will be
approximately
$ million,
or approximately
$ million
if the underwriters exercise their over-allotment option in
full, assuming an initial public offering price of
$ per
share, after deducting estimated underwriting discounts and
commissions and offering expenses payable by us. We expect to
use most of the net proceeds from this offering to fund clinical
trial activities and preclinical research and development
activities, and the balance for other general corporate
purposes. See Use of Proceeds. |
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Risk factors |
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You should read the Risk Factors section of this
prospectus for a discussion of the factors to consider carefully
before deciding to purchase any shares of our common stock. |
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Proposed Nasdaq National Market symbol |
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AMTX |
The number of shares of common stock to be outstanding
immediately after the offering is based on 5,507,024 shares
of common stock outstanding as of May 3, 2006, and gives
effect as of May 3, 2006 to the automatic exercise upon the
closing of this offering of outstanding warrants to purchase
465,486 shares of series B redeemable convertible
preferred stock, and the issuance of 84,009,190 shares of
common stock issuable upon the automatic conversion of all
outstanding shares of our redeemable convertible preferred stock
upon the closing of this offering. The number of shares of
common stock to be outstanding after this offering excludes:
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13,552,120 shares of common stock issuable upon the
exercise of stock options outstanding as of May 3, 2006,
with a weighted average exercise price of $0.48 per share; |
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40,000 shares of common stock issuable upon exercise of a
warrant to purchase common stock at an exercise price of
$0.75 per share; |
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an aggregate
of shares
of common stock reserved for future issuance under our 2006
equity incentive plan as of the closing of this
offering; and |
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an aggregate
of shares
of common stock reserved for future issuance under our 2006
employee stock purchase plan as of the closing of this offering. |
Unless otherwise noted, all information in this prospectus
assumes:
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no exercise of the outstanding options or warrant described
above; and |
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no exercise by the underwriters of their option to purchase
shares of common stock to cover over-allotments. |
We expect to complete a
one-for- reverse
stock split of our common stock before completion of this
offering. All share numbers will be adjusted to give effect to
this reverse stock split.
4
SUMMARY FINANCIAL DATA
The following is a summary of our financial data. You should
read the summary financial data together with our financial
statements and the related notes appearing at the end of this
prospectus, and Managements Discussion and Analysis
of Financial Condition and Results of Operations and other
financial information appearing elsewhere in this prospectus.
The pro forma net loss per share data for the year ended
December 31, 2005, and three month period ended March 31, 2006,
give effect, as of the beginning of each such period, to the
issuance on April 17, 2006 of 21,825,131 shares of our
series C redeemable convertible preferred stock, the
automatic or voluntary exercise upon the closing of this
offering of all outstanding warrants to purchase
555,003 shares of our series B redeemable convertible
preferred stock, and the automatic conversion of all outstanding
shares of our redeemable convertible preferred stock into
84,009,190 shares of common stock upon the closing of this
offering. The pro forma balance sheet data set forth below also
give effect, as of March 31, 2006, to the foregoing events.
The pro forma as adjusted balance sheet data gives further
effect to our issuance and sale of shares of common stock in
this offering at an assumed initial public offering price of
$ per
share, which is the midpoint of the price range listed on the
cover page of this prospectus, after deducting estimated
underwriting discounts and commissions and offering expenses
payable by us.
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Three Months Ended | |
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Period from | |
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Year Ended December 31, | |
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March 31, | |
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February 4, 2002 | |
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(inception) to | |
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2003 | |
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2004 | |
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2005 | |
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2005 | |
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2006 | |
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March 31, 2006 | |
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(unaudited) | |
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(unaudited) | |
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(unaudited) | |
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(Restated) | |
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(Restated) | |
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(in thousands, except shares and per share data) | |
Statement of Operations Data:
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Revenue
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$ |
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$ |
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$ |
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$ |
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$ |
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$ |
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Operating expenses:
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Research and development
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4,433 |
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6,301 |
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13,652 |
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2,238 |
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5,546 |
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30,720 |
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General and administrative
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1,005 |
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2,081 |
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6,878 |
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1,178 |
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2,065 |
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12,582 |
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Impairment of leasehold improvements
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1,030 |
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1,030 |
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Depreciation and amortization
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132 |
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146 |
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303 |
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47 |
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199 |
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804 |
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In-process research and development
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418 |
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Total operating expenses
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6,600 |
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8,528 |
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20,833 |
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3,463 |
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7,810 |
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45,554 |
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Loss from operations
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(6,600 |
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(8,528 |
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(20,833 |
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(3,463 |
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(7,810 |
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(45,554 |
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Other income (expenses):
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Interest income
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5 |
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190 |
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610 |
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57 |
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238 |
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1,056 |
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Interest expense
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(172 |
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(550 |
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(82 |
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(4 |
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(59 |
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(869 |
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Loss before tax benefit
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(6,768 |
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(8,888 |
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(20,305 |
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(3,410 |
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(7,631 |
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(45,367 |
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Income tax benefit
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83 |
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612 |
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695 |
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Net loss
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(6,768 |
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(8,805 |
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(19,693 |
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(3,410 |
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(7,631 |
) |
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(44,672 |
) |
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Preferred stock accretion
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(17 |
) |
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(126 |
) |
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(139 |
) |
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(32 |
) |
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(41 |
) |
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(333 |
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Net loss attributable to common stockholders
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$ |
(6,785 |
) |
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$ |
(8,931 |
) |
|
$ |
(19,832 |
) |
|
$ |
(3,442 |
) |
|
$ |
(7,672 |
) |
|
$ |
(45,005 |
) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Net loss attributable to common stockholders per common
sharebasic and diluted
|
|
$ |
(2.94 |
) |
|
$ |
(3.87 |
) |
|
$ |
(6.45 |
) |
|
$ |
(1.49 |
) |
|
$ |
(1.81 |
) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Weighted-average common shares outstandingbasic and diluted
|
|
|
2,306,541 |
|
|
|
2,306,541 |
|
|
|
3,076,649 |
|
|
|
2,314,804 |
|
|
|
4,228,564 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Unaudited pro forma net loss
|
|
|
|
|
|
|
|
|
|
$ |
(19,692 |
) |
|
|
|
|
|
$ |
(7,631 |
) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Unaudited pro forma basic and diluted net loss per share
|
|
|
|
|
|
|
|
|
|
$ |
(0.23 |
) |
|
|
|
|
|
$ |
(0.09 |
) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Unaudited shares used to compute pro forma basic and diluted net
loss per share
|
|
|
|
|
|
|
|
|
|
|
87,085,839 |
|
|
|
|
|
|
|
88,237,754 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
5
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
As of March 31, 2006 | |
|
|
| |
|
|
|
|
Pro Forma as | |
|
|
Actual | |
|
Pro Forma | |
|
Adjusted | |
|
|
| |
|
| |
|
| |
|
|
|
|
(unaudited) | |
|
|
(in thousands) | |
Balance Sheet Data:
|
|
|
|
|
|
|
|
|
|
|
|
|
Cash and cash equivalents and marketable securities
|
|
$ |
19,552 |
|
|
$ |
47,524 |
|
|
|
|
|
Working capital
|
|
|
16,006 |
|
|
|
43,978 |
|
|
|
|
|
Total assets
|
|
|
23,995 |
|
|
|
51,967 |
|
|
|
|
|
Total liabilities
|
|
|
5,819 |
|
|
|
5,819 |
|
|
|
|
|
Redeemable convertible preferred
stock(1)
|
|
|
60,509 |
|
|
|
|
|
|
|
|
|
Deficit accumulated during the development stage
|
|
|
(44,671 |
) |
|
|
(44,671 |
) |
|
|
|
|
Total stockholders equity (deficiency)
|
|
|
(42,334 |
) |
|
|
46,148 |
|
|
|
|
|
|
|
(1) |
In April 2006, we completed the sale of an additional
21,825,131 shares of series C redeemable convertible
preferred stock for proceeds of $27.5 million. After
evaluating the fair value of the common stock issuable upon
conversion by our preferred stockholders, we determined that the
issuance of the series C redeemable convertible preferred
stock sold in April 2006 resulted in a beneficial conversion
feature of approximately $19.4 million which will be
recorded as a deemed dividend to preferred stockholders during
the second quarter of 2006. |
6
RISK FACTORS
Investing in our common stock involves a high degree of risk.
You should carefully consider the risks and uncertainties
described below together with all of the other information
included in this prospectus, including the financial statements
and related notes appearing at the end of this prospectus,
before deciding to invest in our common stock. If any of the
following risks actually occur, they would materially harm our
business, prospects, financial condition and results of
operations. In this event, the market price of our common stock
could decline and you could lose part or all of your
investment.
Risks Related to Our Financial Position and Need for
Additional Capital
We have incurred significant losses since our inception. We
currently do not, and since inception never have had, any
products available for commercial sale. We expect to incur
losses for the foreseeable future and may never achieve or
maintain profitability.
Since inception, we have incurred significant operating losses.
Our net loss attributable to common stockholders was
$19.8 million and $7.7 million for the year ended
December 31, 2005 and the three months ended March 31,
2006, respectively. As of March 31, 2006, we had an
accumulated deficit of $44.7 million. To date, we have
financed our operations primarily through private placements of
our redeemable convertible preferred stock. We have devoted
substantially all of our efforts to research and development,
including our preclinical development activities and clinical
trials. We have not completed development of any drugs. We
expect to continue to incur significant and increasing operating
losses for at least the next several years and we are unable to
predict the extent of any future losses. We anticipate that our
expenses will increase substantially as we:
|
|
|
|
|
continue our ongoing Phase II clinical trials of Amigal for
the treatment of Fabry disease and potentially conduct
later-stage clinical trials of Amigal; |
|
|
|
|
continue our ongoing Phase I clinical trial of AT2101 for
the treatment of Gaucher disease, initiate another Phase I
clinical trial and potentially conduct later-stage clinical
trials of AT2101; |
|
|
|
|
continue our ongoing preclinical development activities of
AT2220 for the treatment of Pompe disease and potentially
conduct clinical trials of AT2220; |
|
|
|
continue the research and development of additional product
candidates; |
|
|
|
seek regulatory approvals for our product candidates that
successfully complete clinical trials; |
|
|
|
establish a sales and marketing infrastructure to commercialize
products for which we may obtain regulatory approval; and |
|
|
|
add operational, financial and management information systems
and personnel, including personnel to support our product
development efforts and our obligations as a public company. |
To become and remain profitable, we must succeed in developing
and commercializing drugs with significant market potential.
This will require us to be successful in a range of challenging
activities, including the discovery of product candidates,
successful completion of preclinical testing and clinical trials
of our product candidates, obtaining regulatory approval for
these product candidates and manufacturing, marketing and
selling those products for which we may obtain regulatory
approval. We are only in the preliminary stages of these
activities. We may never succeed in these activities and may
never generate revenues that are large enough to achieve
profitability. Even if we do achieve profitability, we may not
be able to sustain or increase profitability on a quarterly or
annual basis. Our failure to become or remain profitable could
depress the market price of our common stock and could impair
our ability to raise capital, expand our business, diversify our
product offerings or continue our operations. A decline in the
market price of our common stock would also cause you to lose a
part or all of your investment.
8
We will need substantial additional funding and may be unable
to raise capital when needed, which would force us to delay,
reduce or eliminate our product development programs or
commercialization efforts.
We expect our research and development expenses to increase in
connection with our ongoing activities, particularly as we
continue our Phase II clinical trials of Amigal, our
Phase I clinical trials of AT2101 and our ongoing
preclinical studies of AT2220 and potentially file an IND for
AT2220. In addition, subject to obtaining regulatory approval of
any of our product candidates, we expect to incur significant
commercialization expenses for product sales and marketing,
securing commercial quantities of product from our manufacturers
and product distribution. We currently have no additional
commitments or arrangements for any additional financing to fund
the research and development and commercial launch of our
product candidates.
We believe that the net proceeds from this offering, together
with our existing cash and cash equivalents and marketable
securities, will be sufficient to enable us to fund our
operating expenses and capital expenditure requirements until at
least (see
Use of Proceeds). Additional funds may not be
available when we need them on terms that are acceptable to us,
or at all. If adequate funds are not available to us on a timely
basis, we may be required to reduce or eliminate research
development programs or commercial efforts.
Our future capital requirements will depend on many factors,
including:
|
|
|
|
|
the progress and results of our preclinical development
activities and clinical trials of Amigal, AT2101 and AT2220; |
|
|
|
the scope, progress, results and costs of preclinical
development, laboratory testing and clinical trials for our
other product candidates; |
|
|
|
the costs, timing and outcome of regulatory review of our
product candidates; |
|
|
|
the number and development requirements of other product
candidates that we pursue; |
|
|
|
the costs of commercialization activities, including product
marketing, sales and distribution; |
|
|
|
the emergence of competing technologies and other adverse market
developments; |
|
|
|
the costs of preparing, filing and prosecuting patent
applications and maintaining, enforcing and defending
intellectual property related claims; |
|
|
|
the extent to which we acquire or invest in businesses, products
and technologies; and |
|
|
|
our ability to establish collaborations and obtain milestone,
royalty or other payments from any such collaborators. |
Any additional funds that we obtain may not be on terms
favorable to us or our stockholders or may require us to
relinquish valuable rights.
Until such time, if ever, as we generate product revenue to
finance our operations, we expect to finance our cash needs
through public or private equity offerings and debt financings,
corporate collaboration and licensing arrangements and grants
from patient advocacy groups, foundations and government
agencies. If we raise additional funds by issuing equity
securities, our stockholders will experience dilution. Debt
financing, if available, may involve agreements that include
covenants limiting or restricting our ability to take specific
actions, such as incurring additional debt, making capital
expenditures or declaring dividends and may include rights that
are senior to the holders of our common stock. Any debt
financing or additional equity that we raise may contain terms,
such as liquidation and other preferences, which are not
favorable to us or our stockholders. If we raise additional
funds through collaboration and licensing arrangements with
third parties, it may be necessary to relinquish valuable rights
to our technologies, future revenue streams, research programs
or product candidates or to grant licenses on terms that may not
be favorable to us or our stockholders.
9
Our short operating history may make it difficult for you to
evaluate the success of our business to date and to assess our
future viability.
We are a development stage company. We commenced operations in
February 2002. Our operations to date have been limited to
organizing and staffing our company, acquiring and developing
our technology and undertaking preclinical studies and limited
clinical trials of our most advanced product candidates. We have
not yet demonstrated our ability to successfully complete
large-scale, clinical trials, obtain regulatory approvals,
manufacture a commercial scale product or arrange for a third
party to do so on our behalf, or conduct sales and marketing
activities necessary for successful product commercialization.
Consequently, any predictions you make about our future success
or viability may not be as accurate as they could be if we had a
longer operating history.
In addition, as a new business, we may encounter unforeseen
expenses, difficulties, complications, delays and other known
and unknown factors. If we are successful in obtaining marketing
approval for any of our lead product candidates, we will need to
transition from a company with a research focus to a company
capable of supporting commercial activities. We may not be
successful in such a transition.
Risks Related to the Development and Commercialization of Our
Product Candidates
We depend heavily on the success of our most advanced product
candidates, Amigal, AT2101 and AT2220. All of our product
candidates are still in either preclinical or clinical
development. Clinical trials of our product candidates may not
be successful. If we are unable to commercialize Amigal, AT2101
or AT2220, or experience significant delays in doing so, our
business will be materially harmed.
We have invested a significant portion of our efforts and
financial resources in the development of our most advanced
product candidates, Amigal, AT2101 and AT2220. Our ability to
generate product revenue, which we do not expect will occur for
at least the next several years, if ever, will depend heavily on
the successful development and commercialization of these
product candidates. The successful commercialization of our
product candidates will depend on several factors, including the
following:
|
|
|
|
|
obtaining supplies of Amigal, AT2101 and AT2220 for completion
of our preclinical activities and clinical studies on a timely
basis; |
|
|
|
successful completion of preclinical studies and clinical trials; |
|
|
|
obtaining marketing approvals from the United States Food and
Drug Administration, or FDA, and similar regulatory authorities
outside the United States; |
|
|
|
establishing commercial-scale manufacturing arrangements with
third party manufacturers whose manufacturing facilities are
operated in compliance with current good manufacturing practice,
or cGMP, regulations; |
|
|
|
launching commercial sales of the product, whether alone or in
collaboration with others; |
|
|
|
acceptance of the product by patients, the medical community and
third party payors; |
|
|
|
competition from other companies and their therapies; |
|
|
|
successful protection of our intellectual property rights from
competing products in the United States and abroad; and |
|
|
|
a continued acceptable safety and efficacy profile of our
product candidates following approval. |
If the market opportunities for our product candidates are
smaller than we believe they are, then our revenues may be
adversely affected and our business may suffer.
Each of the diseases that our product candidates are being
developed to address is relatively rare. Our projections of both
the number of people who have these diseases, as well as the
subset of people with these diseases who have the potential to
benefit from treatment with our product candidates, are based on
10
estimates. Currently, most reported estimates of the prevalence
of these diseases are based on studies of small subsets of the
population of specific geographic areas, which are then
extrapolated to estimate the prevalence of the diseases in the
broader world population. In addition, as new studies are
performed the estimated prevalence of these diseases may change.
In fact, as a result of some recent studies, we believe that
previously reported studies do not accurately account for the
prevalence of Fabry disease and that the prevalence of Fabry
disease could be many times higher than previously reported.
There can be no assurance that the prevalence of Fabry disease,
Gaucher disease or Pompe disease in the study populations,
particularly in these newer studies, accurately reflect the
prevalence of these diseases in the broader world population.
We estimate the number of potential patients in the broader
world population who may respond to treatment with our product
candidates by further extrapolating estimates of the prevalence
of specific types of genetic mutations giving rise to these
diseases. For example, we base our estimate of the percentage of
Fabry patients who may respond to treatment with Amigal on the
frequency of missense and other similar mutations that cause
Fabry disease reported in the Human Gene Mutation Database. As a
result of recent studies that report that the prevalence of
Fabry disease could be many times higher than previously
reported, we believe that the number of patients diagnosed with
Fabry disease will increase and estimate that the number of
Fabry patients who may benefit from the use of Amigal is
significantly higher than some previously reported estimates of
Fabry disease generally. If our estimates of the prevalence of
Fabry disease, Gaucher disease or Pompe disease or of the number
of patients who may benefit from treatment with our product
candidates prove to be incorrect, the market opportunities for
our product candidates may be smaller than we believe they are,
our prospects for generating revenue may be adversely affected
and our business may suffer.
Initial results from a clinical trial do not ensure that the
trial will be successful and success in early stage clinical
trials does not ensure success in
later-stage clinical
trials.
We will only obtain regulatory approval to commercialize a
product candidate if we can demonstrate to the satisfaction of
the FDA or the applicable
non-U.S. regulatory
authority, in well-designed and conducted clinical trials, that
the product candidate is safe and effective and otherwise meets
the appropriate standards required for approval for a particular
indication. Clinical trials are lengthy, complex and extremely
expensive processes with uncertain results. A failure of one or
more of our clinical trials may occur at any stage of testing.
We have limited experience in conducting and managing the
clinical trials necessary to obtain regulatory approvals,
including approval by the FDA.
Our efforts to develop all of our product candidates are at an
early stage. We are currently conducting Phase II clinical
trials of Amigal for Fabry disease. We expect to complete
enrollment in these Phase II clinical trials by the end of
2006. We are currently conducting a Phase I clinical trial
of AT2101 for Gaucher disease in healthy volunteers and expect
to commence another Phase I clinical trial in the second
half of 2006. We expect to file an IND for AT2220 for Pompe
disease by the end of 2006. Success in preclinical testing and
early clinical trials does not ensure that later clinical trials
will be successful, and initial results from a clinical trial do
not necessarily predict final results. For example, results to
date in our Phase II clinical trials of Amigal for the
treatment of Fabry disease caused by missense mutations are
based on data from only four patients. Data from additional
patients enrolled in these trials may be less favorable than the
results to date. We cannot assure you that these trials will
ultimately be successful.
Patients may not be compliant with their dosing regimen or trial
protocols or they may withdraw from the study at any time for
any reason. We note that a fifth patient in the ongoing
Phase II clinical trials for Amigal for the treatment of
Fabry disease elected to withdraw from the study. This patient
had a history of hypertension and discontinued study treatment
due to increased blood pressure, which was reported by the
investigator as possibly related to the study drug. In addition,
we have only initial data from these four continuing patients.
We will obtain additional data regarding the safety and efficacy
of Amigal from these four patients as well as additional
patients that enroll in our ongoing Phase II studies and
any additional data may be less favorable than the data observed
to date.
11
Even if our early stage clinical trials are successful, we will
need to conduct additional clinical trials with larger numbers
of patients receiving the drug for longer periods for all of our
product candidates before we are able to seek approvals to
market and sell these product candidates from the FDA and
regulatory authorities outside the United States. In addition,
each of our product candidates is based on our pharmacological
chaperone technology. To date, we are not aware that any product
based on chaperone technology has been approved by the FDA. As a
result, we cannot be sure what endpoints the FDA will require us
to measure in
later-stage clinical
trials of our product candidates and it may be difficult for us
to obtain FDA approval of our product candidates. If we are not
successful in commercializing any of our lead product
candidates, or are significantly delayed in doing so, our
business will be materially harmed.
We have limited experience in conducting and managing the
preclinical development activities and clinical trials necessary
to obtain regulatory approvals, including approval by the
FDA.
We have limited experience in conducting and managing the
preclinical development activities and clinical trials necessary
to obtain regulatory approvals, including approval by the FDA.
To date, we have only three lead product candidates: Amigal,
AT2101 and AT2220. We have not obtained regulatory approval nor
commercialized any of these or any other product candidates. We
are currently conducting Phase II clinical trials for
Amigal, Phase I clinical trials for AT2101 and preclinical
studies for AT2220 but have not yet initiated a Phase III
clinical trial, or even completed a Phase II clinical
trial, for any of our product candidates. Our limited experience
might prevent us from successfully designing or implementing a
clinical trial. We have limited experience in conducting and
managing the application process necessary to obtain regulatory
approvals and we might not be able to demonstrate that our
product candidates meet the appropriate standards for regulatory
approval. If we are not successful in conducting and managing
our preclinical development activities or clinical trials or
obtaining regulatory approvals, we might not be able to
commercialize our lead product candidates, or might be
significantly delayed in doing so, which will materially harm
our business.
We may find it difficult to enroll patients in our clinical
trials.
Each of the diseases that our lead product candidates are
intended to treat is relatively rare and we expect only a subset
of the patients with these diseases to be eligible for our
clinical trials. Given that each of our product candidates is in
the early stages of required testing, we may not be able to
initiate or continue clinical trials for each or all of our
product candidates if we are unable to locate a sufficient
number of eligible patients to participate in the clinical
trials required by the FDA or other non-U.S. regulatory
agencies. The requirements of our clinical testing mandates that
a patient cannot be involved in another clinical trial for the
same indication. We are aware that our competitors have ongoing
clinical trials for products that are competitive with our
product candidates and patients who would otherwise be eligible
for our clinical trials may be involved in such testing,
rendering them unavailable for testing of our product
candidates. Additionally, many patients with Fabry disease,
Gaucher disease and Pompe disease may already be receiving
existing therapies, such as enzyme replacement therapy, which
would render them ineligible for our current clinical trials if
they are not willing to stop receiving such therapies. Our
inability to enroll a sufficient number of patients for any of
our current or future clinical trials would result in
significant delays or may require us to abandon one or more
clinical trials altogether.
If our preclinical studies do not produce positive results,
if our clinical trials are delayed or if serious side effects
are identified during drug development, we may experience
delays, incur additional costs and ultimately be unable to
commercialize our product candidates.
Before obtaining regulatory approval for the sale of our product
candidates, we must conduct, at our own expense, extensive
preclinical tests to demonstrate the safety of our product
candidates in animals,
12
and clinical trials to demonstrate the safety and efficacy of
our product candidates in humans. Preclinical and clinical
testing is expensive, difficult to design and implement, and can
take many years to complete. A failure of one or more of our
preclinical studies or clinical trials can occur at any stage of
testing. We may experience numerous unforeseen events during, or
as a result of, preclinical testing and the clinical trial
process that could delay or prevent our ability to obtain
regulatory approval or commercialize our product candidates,
including:
|
|
|
|
|
our preclinical tests or clinical trials may produce negative or
inconclusive results, and we may decide, or regulators may
require us, to conduct additional preclinical testing or
clinical trials or we may abandon projects that we expect to be
promising; |
|
|
|
regulators or institutional review boards may not authorize us
to commence a clinical trial or conduct a clinical trial at a
prospective trial site; |
|
|
|
conditions imposed on us by the FDA or any
non-U.S. regulatory
authority regarding the scope or design of our clinical trials
or may require us to resubmit our clinical trial protocols to
institutional review boards for re-inspection due to changes in
the regulatory environment; |
|
|
|
the number of patients required for our clinical trials may be
larger than we anticipate or participants may drop out of our
clinical trials at a higher rate than we anticipate; |
|
|
|
our third party contractors or clinical investigators may fail
to comply with regulatory requirements or fail to meet their
contractual obligations to us in a timely manner; |
|
|
|
we might have to suspend or terminate one or more of our
clinical trials if we, the regulators or the institutional
review boards determine that the participants are being exposed
to unacceptable health risks; |
|
|
|
regulators or institutional review boards may require that we
hold, suspend or terminate clinical research for various
reasons, including noncompliance with regulatory requirements; |
|
|
|
the cost of our clinical trials may be greater than we
anticipate; |
|
|
|
the supply or quality of our product candidates or other
materials necessary to conduct our clinical trials may be
insufficient or inadequate or we may not be able to reach
agreements on acceptable terms with prospective clinical
research organizations; and |
|
|
|
the effects of our product candidates may not be the desired
effects or may include undesirable side effects or the product
candidates may have other unexpected characteristics. |
If we are required to conduct additional clinical trials or
other testing of our product candidates beyond those that we
currently contemplate, if we are unable to successfully complete
our clinical trials or other testing, if the results of these
trials or tests are not positive or are only modestly positive
or if there are safety concerns, we may:
|
|
|
|
|
be delayed in obtaining, or may not be able to obtain, marketing
approval for one or more of our product candidates; |
|
|
|
obtain approval for indications that are not as broad as
intended or entirely different than those indications for which
we sought approval; or |
|
|
|
have the product removed from the market after obtaining
marketing approval. |
Our product development costs will also increase if we
experience delays in testing or approvals. We do not know
whether any preclinical tests or clinical trials will be
initiated as planned, will need to be restructured or will be
completed on schedule, if at all. Significant preclinical or
clinical trial delays also could shorten the patent protection
period during which we may have the exclusive right to
commercialize our product candidates. Such delays could allow
our competitors to bring products to market before we do and
impair our ability to commercialize our products or product
candidates.
13
The commercial success of any product candidates that we may
develop, including Amigal, AT2101 and AT2220, will depend upon
the degree of market acceptance by physicians, patients, third
party payors and others in the medical community.
Any products that we bring to the market, including Amigal,
AT2101 and AT2220 if, they receive marketing approval, may not
gain market acceptance by physicians, patients, third party
payors and others in the medical community. If these products do
not achieve an adequate level of acceptance, we may not generate
significant product revenue and we may not become profitable.
The degree of market acceptance of our product candidates, if
approved for commercial sale, will depend on a number of
factors, including:
|
|
|
|
|
the prevalence and severity of any side effects, including any
limitations or warnings contained in a products approved
labeling; |
|
|
|
the efficacy and potential advantages over alternative
treatments; |
|
|
|
the ability to offer our product candidates for sale at
competitive prices; |
|
|
|
relative convenience and ease of administration; |
|
|
|
the willingness of the target patient population to try new
therapies and of physicians to prescribe these therapies; |
|
|
|
the strength of marketing and distribution support and timing of
market introduction of competitive products; |
|
|
|
publicity concerning our products or competing products and
treatments; and |
|
|
|
sufficient third party insurance coverage or reimbursement. |
Even if a potential product displays a favorable efficacy and
safety profile in preclinical and clinical studies, market
acceptance of the product will not be known until after it is
launched. Our efforts to educate the medical community and third
party payors on the benefits of our product candidates may
require significant resources and may never be successful. Such
efforts to educate the marketplace may require more resources
than are required by the conventional technologies marketed by
our competitors.
If we are unable to obtain adequate reimbursement from
governments or third party payors for any products that we may
develop or if we are unable to obtain acceptable prices for
those products, our prospects for generating revenue and
achieving profitability will suffer.
Our prospects for generating revenue and achieving profitability
will depend heavily upon the availability of adequate
reimbursement for the use of our approved product candidates
from governmental and other third party payors, both in the
United States and in other markets. Reimbursement by a third
party payor may depend upon a number of factors, including the
third party payors determination that use of a product is:
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a covered benefit under its health plan; |
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safe, effective and medically necessary; |
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appropriate for the specific patient; |
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cost-effective; and |
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neither experimental nor investigational. |
Obtaining reimbursement approval for a product from each
government or other third party payor is a time consuming and
costly process that could require us to provide supporting
scientific, clinical and cost effectiveness data for the use of
our products to each payor. We may not be able to provide data
sufficient to gain acceptance with respect to reimbursement or
we might need to conduct post-marketing studies in order to
demonstrate the cost-effectiveness of any future products to
such payors satisfaction. Such studies might require us to
commit a significant amount of management time and financial and
other resources.
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Even when a payor determines that a product is eligible for
reimbursement, the payor may impose coverage limitations that
preclude payment for some uses that are approved by the FDA or
non-U.S. regulatory
authorities. In addition, there is a risk that full
reimbursement may not be available for high priced products.
Moreover, eligibility for coverage does not imply that any
product will be reimbursed in all cases or at a rate that allows
us to make a profit or even cover our costs. Interim payments
for new products, if applicable, may also not be sufficient to
cover our costs and may not be made permanent. A primary trend
in the United States healthcare industry and elsewhere is toward
cost containment. We expect recent changes in the Medicare
program and increasing emphasis on managed care to continue to
put pressure on pharmaceutical product pricing. For example, the
Medicare Prescription Drug Improvement and Modernization Act of
2003 provides a new Medicare prescription drug benefit that
began in 2006 and mandates other reforms. While we cannot
predict the full outcome of the implementation of this
legislation, it is possible that the new Medicare prescription
drug benefit, which will be managed by private health insurers
and other managed care organizations, will result in additional
government reimbursement for prescription drugs, which may make
some prescription drugs more affordable but may further
exacerbate industry wide pressure to reduce prescription drug
prices. If one or more of our product candidates reaches
commercialization, such changes may have a significant impact on
our ability to set a price we believe is fair for our products
and may affect our ability to generate revenue and achieve or
maintain profitability.
Governments outside the United States tend to impose strict
price controls and reimbursement approval policies, which may
adversely affect our prospects for generating revenue.
In some countries, particularly European Union countries, the
pricing of prescription pharmaceuticals is subject to
governmental control. In these countries, pricing negotiations
with governmental authorities can take considerable time (6 to
12 months or longer) after the receipt of marketing
approval for a product. To obtain reimbursement or pricing
approval in some countries, we may be required to conduct a
clinical trial that compares the cost effectiveness of our
product candidate to other available therapies. If reimbursement
of our products is unavailable or limited in scope or amount, or
if pricing is set at unsatisfactory levels, our prospects for
generating revenue, if any, could be adversely affected and our
business may suffer.
If we are unable to establish sales and marketing
capabilities or enter into agreements with third parties to
market and sell our product candidates, we may be unable to
generate product revenue.
At present, we have no sales or marketing personnel. In order to
commercialize any of our product candidates, we must either
acquire or internally develop sales, marketing and distribution
capabilities, or enter into collaborations with partners to
perform these services for us. We may not be able to establish
sales and distribution partnerships on acceptable terms or at
all, and if we do enter into a distribution arrangement, our
success will be dependent upon the performance of our partner.
In the event that we attempt to acquire or develop our own
in-house sales, marketing and distribution capabilities, factors
that may inhibit our efforts to commercialize our products
without strategic partners or licensees include:
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our inability to recruit and retain adequate numbers of
effective sales and marketing personnel; |
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the inability of sales personnel to obtain access to or persuade
adequate numbers of physicians to prescribe our products; |
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the lack of complementary products to be offered by our sales
personnel, which may put us at a competitive disadvantage
against companies with broader product lines; |
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unforeseen costs associated with creating our own sales and
marketing team or with entering into a partnering agreement with
an independent sales and marketing organization; and |
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efforts by our competitors to commercialize products at or about
the time when our product candidates would be coming to market. |
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We may co-promote our product candidates in various markets with
pharmaceutical and biotechnology companies in instances where we
believe that a larger sales and marketing presence will expand
the market or accelerate penetration. If we do enter into
arrangements with third parties to perform sales and marketing
services, our product revenues will be lower than if we directly
sold and marketed our products and any revenues received under
such arrangements will depend on the skills and efforts of
others.
We may not be successful in entering into distribution
arrangements and marketing alliances with third parties. Our
failure to enter into these arrangements on favorable terms
could delay or impair our ability to commercialize our product
candidates and could increase our costs of commercialization.
Dependence on distribution arrangements and marketing alliances
to commercialize our product candidates will subject us to a
number of risks, including:
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we may not be able to control the amount and timing of resources
that our distributors may devote to the commercialization of our
product candidates; |
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our distributors may experience financial difficulties; |
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business combinations or significant changes in a
distributors business strategy may also adversely affect a
distributors willingness or ability to complete its
obligations under any arrangement; and |
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these arrangements are often terminated or allowed to expire,
which could interrupt the marketing and sales of a product and
decrease our revenue. |
If we are unable to establish adequate sales, marketing and
distribution capabilities, whether independently or with third
parties, we may not be able to generate product revenue and may
not become profitable.
Product liability lawsuits against us could cause us to incur
substantial liabilities and to limit commercialization of any
products that we may develop.
We face an inherent risk of product liability exposure related
to the testing of our product candidates in human clinical
trials and will face an even greater risk if we commercially
sell any products that we may develop and which are approved for
sale. We may be exposed to product liability claims and product
recalls, including those which may arise from misuse or
malfunction of, or design flaws in, such products, whether or
not such problems directly relate to the products and services
we have provided. If we cannot successfully defend ourselves
against claims that our product candidates or products caused
injuries, we will incur substantial liabilities. Regardless of
merit or eventual outcome, liability claims may result in:
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decreased demand for any product candidates or products that we
may develop; |
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damage to our reputation; |
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regulatory investigations that could require costly recalls or
product modifications; |
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withdrawal of clinical trial participants; |
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costs to defend the related litigation; |
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substantial monetary awards to trial participants or patients,
including awards that substantially exceed our product liability
insurance, which we would then be required to pay from other
sources, if available, and would damage our ability to obtain
liability insurance at reasonable costs, or at all, in the
future; |
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loss of revenue; |
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the diversion of managements attention from managing our
business; and |
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the inability to commercialize any products that we may develop. |
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We have liability insurance policies for our clinical trials in
the geographies in which we are conducting trials. The aggregate
annual limit of coverage amount under these policies expressed
in United States dollars is approximately $16.4 million,
and these policies are also subject to per claim deductibles.
The amount of insurance that we currently hold may not be
adequate to cover all liabilities that we may incur. Insurance
coverage is increasingly expensive. We may not be able to
maintain insurance coverage at a reasonable cost and we may not
be able to obtain insurance coverage that will be adequate to
satisfy any liability that may arise. On occasion, large
judgments have been awarded in class action lawsuits based on
drugs that had unanticipated side effects. A successful product
liability claim or a series of claims brought against us could
cause our stock price to fall and, if judgments exceed our
insurance coverage, could decrease our available cash and
adversely affect our business.
We face substantial competition which may result in others
discovering, developing or commercializing products before or
more successfully than we do.
The development and commercialization of new drugs is highly
competitive and competition is expected to increase. We face
competition with respect to our current product candidates and
any products we may seek to develop or commercialize in the
future from major pharmaceutical companies, specialty
pharmaceutical companies and biotechnology companies worldwide.
For example, several large pharmaceutical and biotechnology
companies currently market and sell products for the treatment
of Fabry disease. These products include Genzyme
Corporations Fabrazyme and Shire PLCs Replagal. In
addition, Genzyme Corporation and Actelion, Ltd. market and sell
Cerezyme and Zavesca, respectively, for the treatment of Gaucher
disease, and Genzyme Corporation markets and sells Myozyme for
the treatment of Pompe disease. We are also aware of other
enzyme replacement and substrate reduction therapies in
development by third parties.
Potential competitors also include academic institutions,
government agencies and other public and private research
organizations that conduct research, seek patent protection and
establish collaborative arrangements for research, development,
manufacturing and commercialization. Our competitors may develop
products that are more effective, safer, more convenient or less
costly than any that we are developing or that would render our
product candidates obsolete or noncompetitive. Our competitors
may also obtain FDA or other regulatory approval for their
products more rapidly than we may obtain approval for ours. We
may also face competition from off-label use of other approved
therapies. There can be no assurance that developments by others
that will not render our product candidates obsolete or
noncompetitive either during the research phase or once the
products reach commercialization.
We believe that many competitors, including academic
institutions, government agencies, public and private research
organizations, large pharmaceutical companies and smaller more
focused companies, are attempting to develop therapies for many
of our target indications.
Many of our competitors have significantly greater financial
resources and expertise in research and development,
manufacturing, preclinical testing, conducting clinical trials,
obtaining regulatory approvals, prosecuting intellectual
property rights and marketing approved products than we do.
Smaller and other early stage companies may also prove to be
significant competitors, particularly through collaborative
arrangements with large and established companies. These third
parties compete with us in recruiting and retaining qualified
scientific and management personnel, establishing clinical trial
sites and patient registration for clinical trials, as well as
in acquiring technologies complementary to or necessary for our
programs or advantageous to our business. In addition, if we
obtain regulatory approvals for our products, manufacturing
efficiency and marketing capabilities are likely to be
significant competitive factors. We currently have no commercial
manufacturing capability, sales force or marketing
infrastructure. Further, many of our competitors have
substantial resources and expertise in conducting collaborative
arrangements, sourcing in-licensing arrangements and acquiring
new business lines or businesses that are greater than our own.
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Our business activities involve the use of hazardous
materials, which require compliance with environmental and
occupational safety laws regulating the use of such materials.
If we violate these laws, we could be subject to significant
fines, liabilities or other adverse consequences.
Our research and development programs involve the controlled use
of hazardous materials, including microbial agents, corrosive,
explosive and flammable chemicals and other hazardous compounds
in addition to certain biological hazardous waste. Ultimately,
the activities of our third party product manufacturers when a
product candidate reaches commercialization will also require
the use of hazardous materials. Accordingly, we are subject to
federal, state and local laws governing the use, handling and
disposal of these materials. Although we believe that our safety
procedures for handling and disposing of these materials comply
in all material respects with the standards prescribed by local,
state and federal regulations, we cannot completely eliminate
the risk of accidental contamination or injury from these
materials. In addition, our collaborators may not comply with
these laws. In the event of an accident or failure to comply
with environmental laws, we could be held liable for damages
that result, and any such liability could exceed our assets and
resources or we could be subject to limitations or stoppages
related to our use of these materials which may lead to an
interruption of our business operations or those of our third
party contractors. While we believe that our existing insurance
coverage is generally adequate for our normal handling of these
hazardous materials, it may not be sufficient to cover pollution
conditions or other extraordinary or unanticipated events.
Furthermore, an accident could damage or force us to shut down
our operations. Changes in environmental laws may impose costly
compliance requirements on us or otherwise subject us to future
liabilities and additional laws relating to the management,
handling, generation, manufacture, transportation, storage, use
and disposal of materials used in or generated by the
manufacture of our products or related to our clinical trials.
In addition, we cannot predict the effect that these potential
requirements may have on us, our suppliers and contractors or
our customers.
Risks Related to Our Dependence on Third Parties
Use of third parties to manufacture our product candidates
may increase the risk that we will not have sufficient
quantities of our product candidates or such quantities at an
acceptable cost, and clinical development and commercialization
of our product candidates could be delayed, prevented or
impaired.
We do not own or operate manufacturing facilities for clinical
or commercial production of our product candidates. We have
limited personnel with experience in drug manufacturing and we
lack the resources and the capabilities to manufacture any of
our product candidates on a clinical or commercial scale. We
currently outsource all manufacturing and packaging of our
preclinical and clinical product candidates and products to
third parties. The manufacture of pharmaceutical products
requires significant expertise and capital investment, including
the development of advanced manufacturing techniques and process
controls. Manufacturers of pharmaceutical products often
encounter difficulties in production, particularly in scaling up
initial production. These problems include difficulties with
production costs and yields and quality control, including
stability of the product candidate.
We do not currently have any agreements with third party
manufacturers for the long-term commercial supply of any of our
product candidates. We may be unable to enter into agreements
for commercial supply with third party manufacturers, or may be
unable to do so on acceptable terms. Even if we enter into these
agreements, the manufacturers of each product candidate will be
single source suppliers to us for a significant period of time.
Reliance on third party manufacturers entails risks to which we
would not be subject if we manufactured product candidates or
products ourselves, including:
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reliance on the third party for regulatory compliance and
quality assurance; |
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limitations on supply availability resulting from capacity and
scheduling constraints of the third parties; |
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impact on our reputation in the marketplace if manufacturers of
our products, once commercialized, fail to meet the demands of
our customers; |
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the possible breach of the manufacturing agreement by the third
party because of factors beyond our control; and |
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the possible termination or nonrenewal of the agreement by the
third party, based on its own business priorities, at a time
that is costly or inconvenient for us. |
The failure of any of our contract manufacturers to maintain
high manufacturing standards could result in injury or death of
clinical trial participants or patients using products. Such
failure could also result in product liability claims, product
recalls, product seizures or withdrawals, delays or failures in
testing or delivery, cost overruns or other problems that could
seriously harm our business or profitability.
Our contract manufacturers will be required to adhere to FDA
regulations setting forth current good manufacturing processes,
or cGMP. These regulations cover all aspects of the
manufacturing, testing, quality control and recordkeeping
relating to our product candidates and any products that we may
commercialize. Our manufacturers may not be able to comply with
cGMP regulations or similar regulatory requirements outside the
United States. Our manufacturers are subject to unannounced
inspections by the FDA, state regulators and similar regulators
outside the United States. Our failure, or the failure of our
third party manufacturers, to comply with applicable regulations
could result in sanctions being imposed on us, including fines,
injunctions, civil penalties, failure of regulatory authorities
to grant marketing approval of our product candidates, delays,
suspension or withdrawal of approvals, license revocation,
seizures or recalls of product candidates or products, operating
restrictions and criminal prosecutions, any of which could
significantly and adversely affect regulatory approval and
supplies of our product candidates.
Our product candidates and any products that we may develop may
compete with other product candidates and products for access to
manufacturing facilities. There are a limited number of
manufacturers that operate under cGMP regulations and that are
both capable of manufacturing for us and willing to do so. If
the third parties that we engage to manufacture products for our
preclinical tests and clinical trials should cease to continue
to do so for any reason, we likely would experience delays in
advancing these trials while we identify and qualify replacement
suppliers and we may be unable to obtain replacement supplies on
terms that are favorable to us. Later relocation to another
manufacturer will also require notification, review and other
regulatory approvals from the FDA and other regulators and will
subject our production to further cost and instability in the
availability of our product candidates. In addition, if we are
not able to obtain adequate supplies of our product candidates
or the drug substances used to manufacture them, it will be more
difficult for us to develop our product candidates and compete
effectively.
Our current and anticipated future dependence upon others for
the manufacture of our product candidates may adversely affect
our future profit margins and our ability to develop product
candidates and commercialize any products that obtain regulatory
approval on a timely and competitive basis.
Materials necessary to manufacture our product candidates may
not be available on commercially reasonable terms, or at all,
which may delay the development and commercialization of our
product candidates.
We rely on the manufacturers of our product candidates to
purchase from third party suppliers the materials necessary to
produce the compounds for our preclinical and clinical studies
and will rely on these other manufacturers for commercial
distribution if we obtain marketing approval for any of our
product candidates. Suppliers may not sell these materials to
our manufacturers at the time we need them or on commercially
reasonable terms and all such prices are susceptible to
fluctuations in price and availability due to transportation
costs, government regulations, price controls, changes in
economic climate or other foreseen circumstances. We do not have
any control over the process or timing of the acquisition of
these materials by our manufacturers. Moreover, we currently do
not have any agreements for the commercial production of these
materials. If our manufacturers are unable to obtain these
materials for our preclinical
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and clinical studies, product testing and potential regulatory
approval of our product candidates would be delayed,
significantly impacting our ability to develop our product
candidates. If our manufacturers or we are unable to purchase
these materials after regulatory approval has been obtained for
our product candidates, the commercial launch of our product
candidates would be delayed or there would be a shortage in
supply, which would materially affect our ability to generate
revenues from the sale of our product candidates.
We rely on third parties to conduct our preclinical
development activities and our clinical trials and those third
parties may not perform satisfactorily, including failing to
meet established deadlines for the completion of such activities
and trials.
We do not independently conduct preclinical development
activities of our product candidates, such as long-term safety
studies in animals, or clinical trials for our product
candidates. We rely on, or work in conjunction with, third
parties, such as clinical data management organizations, medical
institutions and clinical investigators, to perform this
function. Our reliance on these third parties for preclinical
and clinical development activities reduces our control over
these activities. We are responsible for ensuring that each of
our preclinical development activities and our clinical trials
is conducted in accordance with the applicable general
investigational plan and protocols, however, we have no direct
control over these researchers or contractors (except by
contract), as they are not our employees. Moreover, the FDA
requires us to comply with standards, commonly referred to as
Good Clinical Practices, or GCP, for conducting, recording and
reporting the results of our preclinical development activities
and our clinical trials to assure that data and reported results
are credible and accurate and that the rights, safety and
confidentiality of trial participants are protected. Our
reliance on third parties that we do not control does not
relieve us of these responsibilities and requirements.
Furthermore, these third parties may also have relationships
with other entities, some of which may be our competitors. If
these third parties do not successfully carry out their
contractual duties, meet expected deadlines or conduct our
preclinical development activities or our clinical trials in
accordance with regulatory requirements or our stated protocols,
we will not be able to obtain, or may be delayed in obtaining,
regulatory approvals for our product candidates and will not be
able to, or may be delayed in our efforts to, successfully
commercialize our product candidates. Moreover, these third
parties may be bought by other entities or they may go out of
business, thereby preventing them from meeting their contractual
obligations.
We also rely on other third parties to store and distribute drug
supplies for our preclinical development activities and our
clinical trials. Any performance failure on the part of our
existing or future distributors could delay clinical development
or regulatory approval of our product candidates or
commercialization of our products, producing additional losses
and depriving us of potential product revenue.
Extensions, delays, suspensions or terminations of our
preclinical development activities and our clinical trials as a
result of the performance of our independent clinical
investigators and contract research organizations will delay,
and make more costly, regulatory approval for any product
candidates that we may develop. Any change in a contract
research organization during an ongoing preclinical development
activity or clinical trial could seriously delay that trial and
potentially compromise the results of the activity or trial.
We may not be successful in maintaining or establishing
collaborations, which could adversely affect our ability to
develop and, particularly in international markets,
commercialize products.
For each of our product candidates, we are collaborating with
physicians, patient advocacy groups, foundations and government
agencies in order to assist with the development of our
products. We plan to pursue similar activities in future
programs and plan to evaluate the merits of retaining
commercialization rights for ourselves or entering into
selective collaboration arrangements with leading pharmaceutical
or biotechnology companies. We also may seek to establish
collaborations for the sales, marketing and distribution of our
products outside the United States. If we elect to seek
collaborators in the future but are unable to reach agreements
with suitable collaborators, we may fail to meet our business
objectives for
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the affected product or program. We face, and will continue to
face, significant competition in seeking appropriate
collaborators. Moreover, collaboration arrangements are complex
and time consuming to negotiate, document and implement. We may
not be successful in our efforts, if any, to establish and
implement collaborations or other alternative arrangements. The
terms of any collaborations or other arrangements that we
establish, if any, may not be favorable to us.
Any collaboration that we enter into may not be successful. The
success of our collaboration arrangements, if any, will depend
heavily on the efforts and activities of our collaborators. It
is likely that any collaborators of ours will have significant
discretion in determining the efforts and resources that they
will apply to these collaborations. The risks that we may be
subject to in possible future collaborations include the
following:
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our collaboration agreements are likely to be for fixed terms
and subject to termination by our collaborators in the event of
a material breach or lack of scientific progress by us; |
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our collaborators are likely to have the first right to maintain
or defend our intellectual property rights and, although we
would likely have the right to assume the maintenance and
defense of our intellectual property rights if our collaborators
do not, our ability to do so may be compromised by our
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our collaborators may utilize our intellectual property rights
in such a way as to invite litigation that could jeopardize or
invalidate our intellectual property rights or expose us to
potential liability. |
Collaborations with pharmaceutical companies and other third
parties often are terminated or allowed to expire by the other
party. Such terminations or expirations may adversely affect us
financially and could harm our business reputation in the event
we elect to pursue collaborations that ultimately expire or are
terminated.
Risks Related to Our Intellectual Property
If we are unable to obtain and maintain protection for the
intellectual property relating to our technology and products,
the value of our technology and products will be adversely
affected.
Our success will depend in large part on our ability to obtain
and maintain protection in the United States and other countries
for the intellectual property covering or incorporated into our
technology and products. The patent situation in the field of
biotechnology and pharmaceuticals generally is highly uncertain
and involves complex legal, technical, scientific and factual
questions. We may not be able to obtain additional issued
patents relating to our technology or products. Even if issued,
patents issued to us or our licensors may be challenged,
narrowed, invalidated, held to be unenforceable or circumvented,
which could limit our ability to stop competitors from marketing
similar products or reduce the term of patent protection we may
have for our products. Changes in either patent laws or in
interpretations of patent laws in the United States and other
countries may diminish the value of our intellectual property or
narrow the scope of our patent protection.
The degree of future protection for our proprietary rights is
uncertain, and we cannot ensure that:
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we or our licensors were the first to make the inventions
covered by each of our pending patent applications; |
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we or our licensors were the first to file patent applications
for these inventions; |
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others will not independently develop similar or alternative
technologies or duplicate any of our technologies; |
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any patents issued to us or our licensors will provide a basis
for commercially viable products, will provide us with any
competitive advantages or will not be challenged by third
parties; |
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we will develop additional proprietary technologies that are
patentable; |
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we will file patent applications for new proprietary
technologies promptly or at all; |
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our patents will not expire prior to or shortly after commencing
commercialization of a product; or |
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the patents of others will not have a negative effect on our
ability to do business. |
In addition, we cannot assure you that any of our pending patent
applications will result in issued patents. In particular, we
have filed patent applications in the European Patent Office and
other countries outside the United States that have not issued
as patents. These pending applications include, among others,
the patent applications we license pursuant to a license
agreement with Mount Sinai School of Medicine of New York
University. If patents are not issued in respect of our pending
patent applications, we may not be able to stop competitors from
marketing similar products in Europe and other countries in
which we do not have issued patents.
The U.S. patents that we own or have licensed relating to
Amigal and AT2220 expire in 2018 and the U.S. patents that
we own or have licensed relating to AT2101 expire between 2015
and 2018. The patent rights that we own or have licensed
relating to our product candidates are limited in ways that may
affect our ability to exclude third parties from competing
against us if we obtain regulatory approval to market these
product candidates. In particular:
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We do not hold composition of matter patents covering Amigal and
AT2220, two of our three lead product candidates. Composition of
matter patents can provide protection for pharmaceutical
products to the extent that the specifically covered
compositions are important. For our product candidates for which
we do not hold composition of matter patents, competitors who
obtain the requisite regulatory approval can offer products with
the same composition as our products so long as the competitors
do not infringe any method of use patents that we may hold. |
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For some of our product candidates, the principal patent
protection that covers, or that we expect will cover, our
product candidate is a method of use patent. This type of patent
only protects the product when used or sold for the specified
method. However, this type of patent does not limit a competitor
from making and marketing a product that is identical to our
product that is labeled for an indication that is outside of the
patented method, or for which there is a substantial use in
commerce outside the patented method. |
Moreover, physicians may prescribe such a competitive identical
product for indications other than the one for which the product
has been approved, or off-label indications, that are covered by
the applicable patents. Although such off-label prescriptions
may infringe or induce infringement of method of use patents,
the practice is common and such infringement is difficult to
prevent or prosecute.
Our patents also may not afford us protection against
competitors with similar technology. Because patent applications
in the United States and many other jurisdictions are typically
not published until 18 months after filing, or in some
cases not at all, and because publications of discoveries in the
scientific literature often lag behind the actual discoveries,
neither we nor our licensors can be certain that we or they were
the first to make the inventions claimed in our or their issued
patents or pending patent applications, or that we or they were
the first to file for protection of the inventions set forth in
these patent applications. If a third party has also filed a
U.S. patent application covering our product candidates or
a similar invention, we may have to participate in an
adversarial proceeding, known as an interference, declared by
the U.S. Patent and Trademark Office to determine priority
of invention in the United States. The costs of these
proceedings could be substantial and it is possible that our
efforts could be unsuccessful, resulting in a loss of our
U.S. patent position.
If we fail to comply with our obligations in our intellectual
property licenses with third parties, we could lose license
rights that are important to our business.
We are a party to a number of license agreements including
agreements with the Mount Sinai School of Medicine of New York
University, the University of Maryland, Baltimore County and
Novo Nordisk A/S, pursuant to which we license key intellectual
property relating to our lead product candidates. We
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expect to enter into additional licenses in the future. Under
our existing licenses, we have the right to enforce the licensed
patent rights. Our existing licenses impose, and we expect that
future licenses will impose, various diligence, milestone
payment, royalty, insurance and other obligations on us. If we
fail to comply with these obligations, the licensor may have the
right to terminate the license, in which event we might not be
able to market any product that is covered by the licensed
patents.
If we are unable to protect the confidentiality of our
proprietary information and know-how, the value of our
technology and products could be adversely affected.
We seek to protect our know-how and confidential information, in
part, by confidentiality agreements with our employees,
corporate partners, outside scientific collaborators, sponsored
researchers, consultants and other advisors. We also have
confidentiality and invention or patent assignment agreements
with our employees and our consultants. If our employees or
consultants breach these agreements, we may not have adequate
remedies for any of these breaches. In addition, our trade
secrets may otherwise become known to or be independently
developed by others. Enforcing a claim that a party illegally
obtained and is using our trade secrets is difficult, expensive
and time consuming, and the outcome is unpredictable. In
addition, courts outside the United States may be less willing
to protect trade secrets. Costly and time consuming litigation
could be necessary to seek to enforce and determine the scope of
our proprietary rights, and failure to obtain or maintain trade
secret protection could adversely affect our competitive
business position.
If we infringe or are alleged to infringe the intellectual
property rights of third parties, it will adversely affect our
business.
Our research, development and commercialization activities, as
well as any product candidates or products resulting from these
activities, may infringe or be accused of infringing one or more
claims of an issued patent or may fall within the scope of one
or more claims in a published patent application that may
subsequently issue and to which we do not hold a license or
other rights. Third parties may own or control these patents or
patent applications in the United States and abroad. These third
parties could bring claims against us that would cause us to
incur substantial expenses and, if successful against us, could
cause us to pay substantial damages. Further, if a patent
infringement suit were brought against us, we or they could be
forced to stop or delay research, development, manufacturing or
sales of the product or product candidate that is the subject of
the suit.
No assurance can be given that patents do not exist, have not
been filed, or could not be filed or issued, which contain
claims covering our products, technology or methods. Because of
the number of patents issued and patent applications filed in
our field, we believe there is a risk that third parties may
allege they have patent rights encompassing our products,
technology or methods.
We are aware, for example, of U.S. patents, and corresponding
international counterparts, owned by third parties that contain
claims related to treating protein misfolding. If any of these
patents were to be asserted against us we do not believe that
our proposed products would be found to infringe any valid claim
of these patents. If we were to challenge the validity of any
issued U.S. patent in court, we would need to overcome a
presumption of validity that attaches to every patent. This
burden is high and would require us to present clear and
convincing evidence as to the invalidity of the patents
claims. There is no assurance that a court would find in our
favor on infringement or validity.
In order to avoid or settle potential claims with respect to any
of the patent rights described above or any other patent rights
of third parties, we may choose or be required to seek a license
from a third party and be required to pay license fees or
royalties or both. These licenses may not be available on
acceptable terms, or at all. Even if we or our future
collaborators were able to obtain a license, the rights may be
nonexclusive, which could result in our competitors gaining
access to the same intellectual property. Ultimately, we could
be prevented from commercializing a product, or be forced to
cease some aspect of our business operations, if, as a result of
actual or threatened patent infringement claims, we are unable
to enter into licenses on acceptable terms. This could harm our
business significantly.
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Others may sue us for infringing their patent rights or file
nullity, opposition or interference proceedings against our
patents, even if such claims are without merit, which would
similarly harm our business. Furthermore, during the course of
litigation, confidential information may be disclosed in the
form of documents or testimony in connection with discovery
requests, depositions or trial testimony. Disclosure of our
confidential information and our involvement in intellectual
property litigation could materially adversely affect our
business.
There has been substantial litigation and other proceedings
regarding patent and other intellectual property rights in the
pharmaceutical and biotechnology industries. In addition to
infringement claims against us, we may become a party to other
patent litigation and other proceedings, including interference
proceedings declared by the United States Patent and Trademark
Office and opposition proceedings in the European Patent Office,
regarding intellectual property rights with respect to our
products and technology. Even if we prevail, the cost to us of
any patent litigation or other proceeding could be substantial.
Some of our competitors may be able to sustain the costs of
complex patent litigation more effectively than we can because
they have substantially greater resources. In addition, any
uncertainties resulting from any litigation could significantly
limit our ability to continue our operations. Patent litigation
and other proceedings may also absorb significant management
time.
Many of our employees were previously employed at universities
or other biotechnology or pharmaceutical companies, including
our competitors or potential competitors. We try to ensure that
our employees do not use the proprietary information or know-how
of others in their work for us. However, we may be subject to
claims that we or these employees have inadvertently or
otherwise used or disclosed intellectual property, trade secrets
or other proprietary information of any such employees
former employer. Litigation may be necessary to defend against
these claims and, even if we are successful in defending
ourselves, could result in substantial costs to us or be
distracting to our management. If we fail to defend any such
claims, in addition to paying monetary damages, we may
jeopardize valuable intellectual property rights, disclose
confidential information or lose personnel.
Risks Related to Regulatory Approval of Our Product
Candidates
If we are not able to obtain and maintain required regulatory
approvals, we will not be able to commercialize our product
candidates, and our ability to generate revenue will be
materially impaired.
Our product candidates, including Amigal, AT2101 and AT2220, and
the activities associated with their development and
commercialization, including their testing, manufacture, safety,
efficacy, recordkeeping, labeling, storage, approval,
advertising, promotion, sale and distribution, are subject to
comprehensive regulation by the FDA and other regulatory
agencies in the United States and by comparable authorities in
other countries. Failure to obtain regulatory approval for a
product candidate will prevent us from commercializing the
product candidate in the jurisdiction of the regulatory
authority. We have not obtained regulatory approval to market
any of our product candidates in any jurisdiction. We have only
limited experience in filing and prosecuting the applications
necessary to obtain regulatory approvals and expect to rely on
third party contract research organizations to assist us in this
process.
Securing FDA approval requires the submission of extensive
preclinical and clinical data and supporting information to the
FDA for each therapeutic indication to establish the product
candidates safety and efficacy. Securing FDA approval also
requires the submission of information about the product
manufacturing process to, and inspection of manufacturing
facilities by, the FDA. Our future products may not be
effective, may be only moderately effective or may prove to have
undesirable or unintended side effects, toxicities or other
characteristics that may preclude our obtaining regulatory
approval or prevent or limit commercial use.
Our product candidates may fail to obtain regulatory approval
for many reasons, including:
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our failure to demonstrate to the satisfaction of the FDA or
comparable regulatory authorities that a product candidate is
safe and effective for a particular indication; |
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the results of clinical trials may not meet the level of
statistical significance required by the FDA or comparable
regulatory authorities for approval; |
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our inability to demonstrate that a product candidates
benefits outweigh its risks; |
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our inability to demonstrate that the product candidate presents
an advantage over existing therapies; |
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the FDAs or comparable regulatory authorities
disagreement with the manner in which we interpret the data from
preclinical studies or clinical trials; |
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the FDAs or comparable regulatory authorities
failure to approve the manufacturing processes, quality
procedures or manufacturing facilities of third party
manufacturers with which we contract for clinical or commercial
supplies; and |
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a change in the approval policies or regulations of the FDA or
comparable regulatory authorities or a change in the laws
governing the approval process. |
The process of obtaining regulatory approvals is expensive,
often takes many years, if approval is obtained at all, and can
vary substantially based upon a variety of factors, including
the type, complexity and novelty of the product candidates
involved. Changes in regulatory approval policies during the
development period, changes in or the enactment of additional
statutes or regulations, or changes in regulatory review for
each submitted product application may cause delays in the
approval or rejection of an application. The FDA and
non-U.S. regulatory
authorities have substantial discretion in the approval process
and may refuse to accept any application or may decide that our
data is insufficient for approval and require additional
preclinical, clinical or other studies. In addition, varying
interpretations of the data obtained from preclinical and
clinical testing could delay, limit or prevent regulatory
approval of a product candidate. Any regulatory approval we
ultimately obtain may be limited or subject to restrictions or
post approval commitments that render the approved product not
commercially viable. Any FDA or other regulatory approval of our
product candidates, once obtained, may be withdrawn, including
for failure to comply with regulatory requirements or if
clinical or manufacturing problems follow initial marketing.
Our product candidates may cause undesirable side effects or
have other properties that could delay or prevent their
regulatory approval or commercialization.
Undesirable side effects caused by our product candidates could
interrupt, delay or halt clinical trials and could result in the
denial of regulatory approval by the FDA or other regulatory
authorities for any or all targeted indications, and in turn
prevent us from commercializing our product candidates and
generating revenues from their sale. For example, in a clinical
trial of Amigal for Fabry disease, one patient with a history of
hypertension experienced increased blood pressure during the
course of the trial which was reported by the investigator as
possibly related to the drug. Further, Amigal has been shown to
cause reversible infertility effects in mice.
In addition, if any of our product candidates receive marketing
approval and we or others later identify undesirable side
effects caused by the product:
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regulatory authorities may require the addition of restrictive
labeling statements; |
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regulatory authorities may withdraw their approval of the
product; and |
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we may be required to change the way the product is administered
or conduct additional clinical trials. |
Any of these events could prevent us from achieving or
maintaining market acceptance of the affected product or could
substantially increase the costs and expenses of commercializing
the product candidate, which in turn could delay or prevent us
from generating significant revenues from its sale or adversely
affect our reputation.
25
We may not be able to obtain orphan drug exclusivity for our
product candidates. If our competitors are able to obtain orphan
drug exclusivity for their products that are the same drug as
our product candidates, we may not be able to have competing
products approved by the applicable regulatory authority for a
significant period of time.
Regulatory authorities in some jurisdictions, including the
United States and Europe, may designate drugs for relatively
small patient populations as orphan drugs. We obtained orphan
drug designations from the FDA for Amigal for the treatment of
Fabry disease on February 25, 2004 and for AT2101 for the
treatment of Gaucher disease on January 10, 2006. We also
obtained orphan drug designation from the European Medicines
Agency, or EMEA, for Amigal on May 22, 2006. We anticipate
filing for orphan drug designation from the EMEA for AT2101 for
the treatment of Gaucher disease and from the FDA and EMEA for
AT2220 for the treatment of Pompe disease. Generally, if a
product with an orphan drug designation subsequently receives
the first marketing approval for the indication for which it has
such designation, the product is entitled to a period of
marketing exclusivity, which precludes the applicable regulatory
authority from approving another marketing application for the
same drug for that time period. The applicable period is seven
years in the United States and ten years in Europe. For a drug
composed of small molecules, the FDA defines same
drug as a drug that contains the same active molecule and
is intended for the same use. Obtaining orphan drug exclusivity
for Amigal and AT2101 may be important to each of the product
candidates success. Even if we obtain orphan drug
exclusivity for Amigal or AT2101 for these indications, we may
not be able to maintain it. For example, if a competitive
product that is the same drug as our product candidate is shown
to be clinically superior to our product candidate, any orphan
drug exclusivity we have obtained will not block the approval of
such competitive product.
Any product for which we obtain marketing approval could be
subject to restrictions or withdrawal from the market and we may
be subject to penalties if we fail to comply with regulatory
requirements or if we experience unanticipated problems with our
products, when and if any of them are approved.
Any product for which we obtain marketing approval, along with
the manufacturing processes, post approval clinical data,
labeling, advertising and promotional activities for such
product, will be subject to continual requirements of and review
by the FDA and comparable regulatory authorities. These
requirements include submissions of safety and other post
marketing information and reports, registration requirements,
cGMP requirements relating to quality control, quality assurance
and corresponding maintenance of records and documents,
requirements regarding the distribution of samples to physicians
and recordkeeping. Even if we obtain regulatory approval of a
product, the approval may be subject to limitations on the
indicated uses for which the product may be marketed or to the
conditions of approval, or contain requirements for costly post
marketing testing and surveillance to monitor the safety or
efficacy of the product. We also may be subject to state laws
and registration requirements covering the distribution of our
products. Later discovery of previously unknown problems with
our products, manufacturers or manufacturing processes, or
failure to comply with regulatory requirements, may result in
actions such as:
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restrictions on such products, manufacturers or manufacturing
processes; |
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warning letters; |
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withdrawal of the products from the market; |
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refusal to approve pending applications or supplements to
approved applications that we submit; |
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voluntary or mandatory recall; |
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fines; |
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suspension or withdrawal of regulatory approvals or refusal to
approve pending applications or supplements to approved
applications that we submit; |
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refusal to permit the import or export of our products; |
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product seizure or detentions; |
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injunctions or the imposition of civil or criminal
penalties; and |
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adverse publicity. |
If we, or our suppliers, third party contractors, clinical
investigators or collaborators are slow to adapt, or are unable
to adapt, to changes in existing regulatory requirements or
adoption of new regulatory requirements or policies, we or our
collaborators may lose marketing approval for our products when
and if any of them are approved, resulting in decreased revenue
from milestones, product sales or royalties.
Failure to obtain regulatory approval in international
jurisdictions would prevent us from marketing our products
abroad.
We intend to have our products marketed outside the United
States. In order to market our products in the European Union
and many other jurisdictions, we must obtain separate regulatory
approvals and comply with numerous and varying regulatory
requirements. The approval procedures vary among countries and
can involve additional testing and clinical trials. The time
required to obtain approval may differ from that required to
obtain FDA approval. The regulatory approval process outside the
United States may include all of the risks associated with
obtaining FDA approval. In addition, in many countries outside
the United States, it is required that the product be approved
for reimbursement by government-backed healthcare regulators or
insurance providers before the product can be approved for sale
in that country. We may not obtain approvals from regulatory
authorities outside the United States on a timely basis, if at
all. Approval by the FDA does not ensure approval by regulatory
authorities in other countries or jurisdictions, and approval by
one regulatory authority outside the United States does not
ensure approval by regulatory authorities in other countries or
jurisdictions or by the FDA. We may not be able to file for
regulatory approvals and may not receive necessary approvals to
commercialize our products in any market.
Risks Related to Employee Matters and Managing Growth
Our future success depends on our ability to retain our chief
executive officer and other key executives and to attract,
retain and motivate qualified personnel.
We are highly dependent on principal members of our management
team and scientific staff. These executives each have
significant pharmaceutical industry experience, including our
President and Chief Executive Officer, John F. Crowley, with
whom we have entered into an employment agreement that runs for
successive one year terms until either we or Mr. Crowley elect
to terminate the agreement. Mr. Crowley is a commissioned
officer in the U.S. Navy (Reserve) and may be called to
active duty service at any time. The loss of Mr. Crowley
for protracted military duty would materially adversely affect
our business. We do not maintain key person
insurance on Mr. Crowley or on any of our other executive
officers.
Recruiting and retaining qualified scientific personnel,
clinical personnel and sales and marketing personnel will also
be critical to our success. Our industry has experienced a high
rate of turnover in recent years. We may not be able to attract
and retain these personnel on acceptable terms given the
competition among numerous pharmaceutical and biotechnology
companies for similar personnel, particularly in New Jersey and
surrounding areas. Although we believe we offer competitive
salaries and benefits, we may have to increase spending in order
to retain personnel.
We also experience competition for the hiring of scientific and
clinical personnel from universities and research institutions.
In addition, we rely on consultants and advisors, including
scientific and clinical advisors, to assist us in formulating
our research and development and commercialization strategy. Our
consultants and advisors may be employed by employers other than
us and may have commitments under consulting or advisory
contracts with other entities that may limit their availability
to us.
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We expect to expand our development, regulatory and sales and
marketing capabilities, and as a result, we may encounter
difficulties in managing our growth, which could disrupt our
operations.
We are a development stage company with 52 employees as of
April 30, 2006. Of these employees, 35 work in
research and development and 17 provide administrative
services. We expect to experience significant growth in the
number of our employees and the scope of our operations,
particularly in the areas of drug development, regulatory
affairs and sales and marketing. Assuming our plans and business
conditions progress consistent with our current projections, we
plan to have grown to a total of
90-100 employees
by the end of 2006 and to a total of
140-150 employees
by the end of 2007. To manage our anticipated future growth, we
must continue to implement and improve our managerial,
operational and financial systems, expand our facilities and
continue to recruit and train additional qualified personnel.
Due to our limited resources, we may not be able to effectively
manage the expansion of our operations or recruit and train
additional qualified personnel. The physical expansion of our
operations may lead to significant costs and may divert our
management and business development resources. Any inability on
the part of our management to manage growth could delay the
execution of our business plans or disrupt our operations.
Risks Related to Our Common Stock and This Offering
After this offering, our executive officers, directors and
principal stockholders will maintain the ability to control all
matters submitted to our stockholders for approval.
When this offering is completed, our executive officers,
directors and stockholders who owned more than 5% of our
outstanding common stock before this offering will, in the
aggregate, beneficially own shares representing
approximately % of our common
stock. As a result, if these stockholders were to choose to act
together, they would be able to control all matters submitted to
our stockholders for approval, as well as our management and
affairs. For example, these persons, if they choose to act
together, will control the election of directors and approval of
any merger, consolidation, sale of all or substantially all of
our assets or other business combination or reorganization. This
concentration of voting power could delay or prevent an
acquisition of us on terms that other stockholders may desire.
The interests of this group of stockholders may not always
coincide with your interests or the interests of other
stockholders, and they may act, whether by meeting or written
consent of stockholders, in a manner that advances their best
interests and not necessarily those of other stockholders,
including obtaining a premium value for their common stock, and
might affect the prevailing market price for our common stock.
Provisions in our corporate charter documents and under
Delaware law could make an acquisition of us, which may be
beneficial to our stockholders, more difficult and may prevent
attempts by our stockholders to replace or remove our current
management.
Provisions in our corporate charter and our bylaws that will
become effective upon the closing of this offering may
discourage, delay or prevent a merger, acquisition or other
change in control of us that stockholders may consider
favorable, including transactions in which you might otherwise
receive a premium for your shares. These provisions could also
limit the price that investors might be willing to pay in the
future for shares of our common stock, thereby depressing the
market price of our common stock. In addition, these provisions
may frustrate or prevent any attempts by our stockholders to
replace or remove our current management by making it more
difficult for stockholders to replace members of our board of
directors. Because our board of directors is responsible for
appointing the members of our management team, these provisions
could in turn affect any attempt by our stockholders to replace
current members of our management team. Among others, these
provisions:
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establish a classified board of directors, and, as a result, not
all directors are elected at one time; |
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allow the authorized number of our directors to be changed only
by resolution of our board of directors; |
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limit the manner in which stockholders can remove directors from
our board of directors; |
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establish advance notice requirements for stockholder proposals
that can be acted on at stockholder meetings and nominations to
our board of directors; |
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require that stockholder actions must be effected at a duly
called stockholder meeting and prohibit actions by our
stockholders by written consent; |
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limit who may call stockholder meetings; |
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authorize our board of directors to issue preferred stock,
without stockholder approval, which could be used to institute a
poison pill that would work to dilute the stock
ownership of a potential hostile acquirer, effectively
preventing acquisitions that have not been approved by our board
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require the approval of the holders of at least 67% of the votes
that all our stockholders would be entitled to cast to amend or
repeal certain provisions of our charter or bylaws. |
Moreover, because we are incorporated in Delaware, we are
governed by the provisions of Section 203 of the Delaware
General Corporation Law, which prohibits a person who owns in
excess of 15% of our outstanding voting stock from merging or
combining with us for a period of three years after the date of
the transaction in which the person acquired in excess of 15% of
our outstanding voting stock, unless the merger or combination
is approved in a prescribed manner.
If you purchase shares of common stock in this offering, you
will suffer immediate dilution of your investment.
We expect the initial public offering price of our common stock
to be substantially higher than the net tangible book value per
share of our common stock. Therefore, if you purchase shares of
our common stock in this offering, you will pay a price per
share that substantially exceeds our net tangible book value per
share after this offering. To the extent outstanding options or
warrants are exercised, you will incur further dilution.
Based on an assumed initial public offering price of
$ per
share, which is the midpoint of the price range listed on the
cover page of this prospectus, you will experience immediate
dilution of
$ per
share, representing the difference between our pro forma net
tangible book value per share after giving effect to this
offering and the assumed initial public offering price. In
addition, purchasers of common stock in this offering will have
contributed approximately % of the
aggregate price paid by all purchasers of our common stock but
will own only approximately % of
our common stock outstanding after this offering.
An active trading market for our common stock may not
develop.
This is our initial public offering of equity securities and
prior to this offering, there has been no public market for our
common stock. The initial public offering price for our common
stock will be determined through negotiations with the
underwriters. Although we have applied to have our common stock
approved for quotation on The Nasdaq National Market, an active
trading market for our common stock may never develop or be
sustained following this offering. If an active market for our
common stock does not develop, it may be difficult for you to
sell shares you purchase in this offering without depressing the
market price for our common stock.
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If the price of our common stock is volatile, purchasers of
our common stock could incur substantial losses.
The price of our common stock is likely to be volatile. The
stock market in general and the market for biotechnology
companies in particular have experienced extreme volatility that
has often been unrelated to the operating performance of
particular companies. As a result of this volatility, investors
may not be able to sell their shares of our common stock at or
above the initial public offering price. The market price for
our common stock may be influenced by many factors, including:
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results of clinical trials of our product candidates or those of
our competitors; |
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our entry into or the loss of a significant collaboration; |
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regulatory or legal developments in the United States and other
countries, including changes in the health care payment systems; |
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variations in our financial results or those of companies that
are perceived to be similar to us; |
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changes in the structure of healthcare payment systems; |
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market conditions in the pharmaceutical and biotechnology
sectors and issuance of new or changed securities analysts
reports or recommendations; |
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general economic, industry and market conditions; |
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results of clinical trials conducted by others on drugs that
would compete with our product candidates; |
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developments or disputes concerning patents or other proprietary
rights; |
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public concern over our product candidates or any products
approved in the future; |
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litigation; |
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future sales or anticipated sales of our common stock by us or
our stockholders; and |
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the other factors described in this Risk Factors
section. |
For these reasons and others you should consider an investment
in our common stock as risky and invest only if you can
withstand a significant loss and wide fluctuations in the marked
value of your investment.
We have broad discretion in the use of the net proceeds from
this offering and may not use them effectively.
Our management will have broad discretion in the application of
the net proceeds from this offering and could spend the proceeds
in ways that do not improve our results of operations or enhance
the value of our common stock. The failure by our management to
apply these funds effectively could result in financial losses
that could have a material adverse effect on our business, cause
the price of our common stock to decline and delay the
development of our product candidates. Pending the application
of these funds, we may invest the net proceeds from this
offering in a manner that does not produce income or that loses
value.
We intend to use the proceeds from this offering for clinical
activities, including clinical supplies, preclinical research
and development activities, general and administrative expenses,
working capital needs and other general corporate purposes,
including capital expenditures. Because of the number and
variability of factors that will determine our use of the
proceeds from this offering, their ultimate use may vary
substantially from their currently intended use. For a further
description of our intended use of the proceeds of this
offering, see the Use of Proceeds section of this
prospectus.
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We have never paid cash dividends on our capital stock and we
do not anticipate paying any cash dividends in the foreseeable
future. You should not invest in us if you require dividend
income. Any income from an investment in us would only come from
a rise in the market price of our common stock, which is
uncertain and unpredictable.
We have paid no cash dividends on our capital stock to date. We
currently intend to retain our future earnings, if any, to fund
the development and growth of our business and do not foresee
payment of a dividend in any upcoming fiscal period. In
addition, the terms of existing or any future debt agreements
may preclude us from paying dividends. As a result, capital
appreciation, if any, of our common stock will be your sole
source of gain for the foreseeable future.
A significant portion of our total outstanding shares of
common stock is restricted from immediate resale but may be sold
into the market in the near future. This could cause the market
price of our common stock to drop significantly, even if our
business is doing well.
Sales of a substantial number of shares of our common stock in
the public market could occur at any time. These sales, or the
perception in the market that the holders of a large number of
shares of common stock intend to sell shares, could reduce the
market price of our common stock. After this offering, we will
have
outstanding shares
of common stock based on the number of shares outstanding as
of ,
2006. Of these
shares, may
be resold in the public market immediately and the
remaining shares
are currently restricted under securities laws or as a result of
lock-up agreements but
will be able to be sold after the offering as described in the
Shares Eligible for Future Sale section of this
prospectus. Moreover, after this offering, holders of an
aggregate of 84,049,190 shares of our common stock will
have rights, subject to some conditions, to require us to file
registration statements covering their shares or to include
their shares in registration statements that we may file for
ourselves or other stockholders. We also intend to register
all shares
of common stock that we may issue under our equity compensation
plans. Once we register these shares, they can be freely sold in
the public market upon issuance, subject to the 180 day lock-up
periods under the
lock-up agreements
described in the Underwriters section of this
prospectus.
If securities or industry analysts do not publish research or
reports or publish unfavorable research about our business, the
price of our common stock and trading volume could decline.
The trading market for our common stock will depend in part on
the research and reports that securities or industry analysts
publish about us or our business. We do not currently have and
may never obtain research coverage by securities and industry
analysts. If no securities or industry analysts commence
coverage of us the trading price for our common stock would be
negatively affected. In the event we obtain securities or
industry analyst coverage, if one or more of the analysts who
covers us downgrades our common stock, the price of our common
stock would likely decline. If one or more of these analysts
ceases to cover us or fails to publish regular reports on us,
interest in the purchase of our common stock could decrease,
which could cause the price of our common stock or trading
volume to decline.
We will incur increased costs as a result of being a public
company.
As a public company, we will incur significant legal,
accounting, reporting and other expenses that we did not incur
as a private company, including costs related to compliance with
the regulations of the Sarbanes-Oxley Act of 2002. We expect
these rules and regulations to increase our legal and financial
compliance costs and to make some activities more time-consuming
and costly. We also expect these new rules and regulations may
make it more difficult and more expensive for us to obtain
director and officer liability insurance and we may be required
to accept reduced policy limits and coverage or incur
substantially higher costs to obtain the same or similar
coverage. As a result, we may experience more difficulty
attracting and retaining qualified individuals to serve on our
board of directors or as executive officers. We cannot predict
or estimate the amount of additional costs we may incur as a
result of these requirements or the timing of such costs.
31
SPECIAL NOTE REGARDING FORWARD-LOOKING STATEMENTS
This prospectus contains forward-looking statements that involve
substantial risks and uncertainties. All statements, other than
statements of historical facts, included in this prospectus
regarding our strategy, future operations, future financial
position, future revenues, projected costs, prospects, plans and
objectives of management are forward-looking statements. The
words anticipate, believe,
estimate, expect, intend,
may, plan, predict,
project, will, would and
similar expressions are intended to identify forward-looking
statements, although not all forward-looking statements contain
these identifying words.
The forward-looking statements in this prospectus include, among
other things, statements about:
|
|
|
|
|
our plans to develop and commercialize Amigal, AT2101 and AT2220; |
|
|
|
our ongoing and planned discovery programs, preclinical studies
and clinical trials; |
|
|
|
our ability to enter into selective collaboration arrangements; |
|
|
|
the timing of and our ability to obtain and maintain regulatory
approvals for our product candidates; |
|
|
|
the rate and degree of market acceptance and clinical utility of
our products; |
|
|
|
our ability to quickly and efficiently identify and develop
product candidates; |
|
|
|
the extent to which our scientific approach may potentially
address a broad range of diseases across multiple therapeutic
areas; |
|
|
|
our commercialization, marketing and manufacturing capabilities
and strategy; |
|
|
|
our intellectual property position; and |
|
|
|
our estimates regarding expenses, future revenues, capital
requirements and needs for additional financing. |
We may not actually achieve the plans, intentions or
expectations disclosed in our forward-looking statements, and
you should not place undue reliance on our forward-looking
statements. Actual results or events could differ materially
from the plans, intentions and expectations disclosed in the
forward-looking statements we make. We have included important
factors in the cautionary statements included in this
prospectus, particularly in the Risk Factors
section, that we believe could cause actual results or events to
differ materially from the forward-looking statements that we
make. Our forward-looking statements do not reflect the
potential impact of any future acquisitions, mergers,
dispositions, joint ventures, collaborations or investments we
may make.
You should read this prospectus and the documents that we
reference in this prospectus and have filed as exhibits to the
registration statement of which this prospectus is a part
completely and with the understanding that our actual future
results may be materially different from what we expect. We do
not assume any obligation to update any forward-looking
statements.
32
USE OF PROCEEDS
We estimate that the net proceeds from the sale
of shares
of common stock in this offering will be approximately
$ million,
or
$ million
if the underwriters exercise their over-allotment option in
full, based on an assumed initial public offering price of
$ per
share, which is the midpoint of the price range listed on the
cover page of this prospectus, and after deducting the estimated
underwriting discounts and commissions and estimated offering
expenses payable by us.
We intend to use the net proceeds from this offering to fund the
growth of our business, including:
|
|
|
|
|
$ to $ million
for clinical development of Amigal for the treatment of Fabry
disease; |
|
|
|
$ to $ million
for clinical development of AT2101 for the treatment of Gaucher
disease; |
|
|
|
$ to $ million
for development of AT2220 for the treatment of Pompe disease; |
|
|
|
$ to $ million
for research and development activities relating to additional
preclinical programs; and |
|
|
|
|
the balance, if any, to fund working capital and other general
corporate purposes, which may include the acquisition or
licensing of complementary technologies, products or businesses,
the expansion of our current corporate offices and laboratory
space in Cranbury, NJ, and the leasing of additional space at
one or more different facilities. |
|
The expected use of net proceeds of this offering represents our
intentions based on our current plans and business conditions.
The amount and timing of our actual expenditures will depend on
numerous factors, including the progress of our research and
development activities and clinical trials, the number and
breadth of our product development programs, whether or not we
establish corporate collaborations and other arrangements, and
the amount of cash, if any, generated by our operations and any
unforeseen cash needs. As a result, we will retain broad
discretion in the allocation and use of the remaining net
proceeds of this offering. We do not expect the net proceeds
from this offering and our other available funds to be
sufficient to fund the completion of the development of our lead
product candidates, and we expect that we will need to raise
additional funds prior to being able to market any products. We
have no current plans, agreements or commitments for any
material acquisitions or licenses of any technologies, products
or businesses.
Pending application of the net proceeds, as described above, we
intend to invest any remaining proceeds in a variety of
short-term, investment-grade, interest-bearing securities.
DIVIDEND POLICY
We have never declared or paid any dividends on our capital
stock. We currently intend to retain any future earnings to
finance our research and development efforts, the further
development of our pharmacological chaperone technology, and the
expansion of our business. We do not intend to declare or pay
cash dividends to our stockholders in the foreseeable future.
33
CAPITALIZATION
The following table sets forth our capitalization as of
March 31, 2006:
|
|
|
|
|
on an actual basis; |
|
|
|
on a pro forma basis to give effect, as of March 31, 2006,
to our issuance on April 17, 2006 of 21,825,131 shares
of series C redeemable convertible preferred stock, the
automatic or voluntary exercise upon the completion of this
offering of all outstanding warrants to purchase
555,003 shares of series B redeemable convertible
preferred stock, and the automatic conversion of all outstanding
shares of our redeemable convertible preferred stock into an
aggregate of 84,009,190 shares of common stock upon the
completion of this offering; and |
|
|
|
on a pro forma as adjusted basis to give further effect to our
issuance and sale of shares of common stock in this offering at
an assumed initial public offering price of
$ per
share, which is the midpoint of the price range listed on the
cover page of this prospectus, after deducting estimated
underwriting discounts and commissions and offering expenses
payable by us. |
The pro forma information below is illustrative only and our
capitalization following the closing of this offering will be
adjusted based on the actual initial public offering price and
other terms of this offering determined at pricing. You should
read this table together with Managements Discussion
and Analysis of Financial Condition and Results of
Operations and our financial statements and the related
notes appearing at the end of this prospectus.
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
As of March 31, 2006 | |
|
|
| |
|
|
|
|
Pro | |
|
Pro Forma | |
|
|
Actual | |
|
Forma | |
|
As Adjusted | |
|
|
| |
|
| |
|
| |
|
|
(unaudited) | |
|
|
(in thousands) | |
Capital lease obligations
|
|
$ |
2,846 |
|
|
$ |
2,846 |
|
|
|
|
|
|
Series A redeemable convertible preferred stock, par value
$0.01 per share; 3,333,334 shares authorized, issued
and outstanding, actual; no shares authorized, issued or
outstanding, pro forma and pro forma as adjusted
|
|
|
2,470 |
|
|
|
|
|
|
|
|
|
|
Series B redeemable convertible preferred stock, par value
$0.01 per share; 37,025,594 shares authorized, actual,
36,470,591 shares issued and outstanding, actual; no shares
authorized, issued or outstanding, pro forma and pro forma as
adjusted
|
|
|
30,696 |
|
|
|
|
|
|
|
|
|
|
Series C redeemable convertible preferred stock, par value
$0.01 per share; 43,650,262 shares authorized, issued
and outstanding, actual; no shares authorized, issued or
outstanding, pro forma and pro forma as adjusted
|
|
|
27,343 |
|
|
|
|
|
|
|
|
|
Stockholders equity:
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Common stock, par value $0.01 per share;
115,000,000 shares authorized, actual and pro forma;
4,635,231 shares issued and outstanding, actual;
88,644,421 shares issued and outstanding, pro
forma; shares
authorized
and shares
issued and outstanding, pro forma as adjusted
|
|
|
46 |
|
|
|
886 |
|
|
|
|
|
Additional paid-in
capital(1)
|
|
|
2,297 |
|
|
|
89,938 |
|
|
|
|
|
Accumulated other comprehensive loss
|
|
|
(5 |
) |
|
|
(5 |
) |
|
|
|
|
Deficit accumulated during the development stage
|
|
|
(44,671 |
) |
|
|
(44,671 |
) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Total stockholders equity
(deficiency)(1)
|
|
$ |
(42,334 |
) |
|
$ |
46,148 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Total
capitalization(1)
|
|
$ |
21,021 |
|
|
$ |
48,994 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
(1) |
A $1.00 increase (decrease) in the assumed initial public
offering price of
$ per share
would increase (decrease) each of cash, and cash equivalents and
short-term investments, additional paid-in capital, total
stockholders equity and total capitalization by
approximately
$ million,
assuming the number of shares offered by us, as set forth on the
cover page of |
34
|
|
|
this prospectus, remains the same
and after deducting the estimated underwriting discounts and
commissions and estimated offering expenses payable by us.
|
The table above does not include:
|
|
|
|
|
14,573,975 shares of common stock issuable upon exercise of
options outstanding as of March 31, 2006 at a weighted average
exercise price of $0.43 per share; |
|
|
|
40,000 shares of common stock issuable upon exercise of a
warrant to purchase common stock at an exercise price of
$0.75 per share; |
|
|
|
an aggregate
of shares
of common stock reserved for future issuance under our 2006
equity incentive plan as of the closing of this
offering; and |
|
|
|
an aggregate
of shares
of common stock reserved for future issuance under our 2006
employee stock purchase plan as of the closing of this offering. |
We expect to complete a
one-for- reverse
stock split of our common stock before the completion of this
offering. All share numbers have been retroactively adjusted to
give effect to this reverse stock split.
35
DILUTION
If you invest in our common stock in this offering, your
interest will be diluted to the extent of the difference between
the public offering price per share of our common stock and the
pro forma net tangible book value per share of our common stock
after this offering.
The historical net tangible book value of our common stock as of
March 31, 2006 was approximately $ million or
$ per
share, based
on shares
of common stock outstanding, as adjusted to reflect the
one-for- reverse
split of our common stock to be effected prior to the completion
of this offering. Historical net tangible book value per share
represents the amount of our total tangible assets less total
liabilities, divided by the number of shares of common stock
outstanding. Our pro forma net tangible book value as of
March 31, 2006 was approximately
$ million,
or
$ per
share of common stock. Pro forma net tangible book value per
share represents the amount of our total tangible assets reduced
by the amount of our total liabilities, divided by the pro forma
number of shares of common stock outstanding after giving
effect, as of March 31, 2006, to the issuance on April 17,
2006 of 21,825,131 shares of our series C redeemable convertible
preferred stock, the automatic or voluntary exercise upon
completion of this offering of all outstanding warrants to
purchase 555,003 shares of series B redeemable
convertible preferred stock, and the automatic conversion of all
outstanding shares of our redeemable convertible preferred stock
into an aggregate of 84,009,190 shares of common stock upon
completion of this offering.
After giving effect to our issuance and sale
of shares
of our common stock in this offering at an assumed initial
public offering price of
$ per
share (the midpoint of the price range set forth on the cover
page of this prospectus) less the estimated underwriting
discounts and commissions and estimated offering expenses
payable by us, our pro forma net tangible book value as
of ,
2006 would have been approximately
$ million,
or
$ per
share of our common stock. This represents an immediate increase
in pro forma net tangible book value of
$ per
share to our existing stockholders and an immediate dilution in
pro forma net tangible book value of
$ per
share to new investors purchasing shares in this offering at the
initial public offering price. Dilution per share to new
investors is determined by subtracting pro forma net tangible
book value per share after this offering from the initial public
offering price per share paid by a new investor.
The following table illustrates this per share dilution:
|
|
|
|
|
|
Assumed initial public offering price per share
|
|
$ |
|
|
|
Historical net tangible book value per shares as of
March 31, 2006
|
|
$ |
|
|
|
Increase attributable to the conversion of outstanding preferred
stock
|
|
|
|
|
|
Pro forma net tangible book value per share before this offering
|
|
|
|
|
|
Increase per share attributable to new investors
|
|
|
|
|
Pro forma net tangible book value per share after this offering
|
|
|
|
|
Dilution per share to new investors
|
|
$ |
|
|
A $1.00 increase (decrease) in the assumed initial public
offering price of
$ per
share would increase (decrease) our pro forma net tangible book
value after this offering by approximately $ million, our
pro forma net tangible book value per share after this offering
by approximately
$ by
$ per
share and dilution per share to new investors in this offering
by approximately
$ assuming
that the number of shares offered by us, as set forth on the
cover page of this prospectus, remains the same and after
deducting the estimated underwriting discounts and commissions.
If the underwriters exercise their over-allotment option or if
any shares are issued in connection with outstanding options or
warrants, you will experience further dilution.
The following table sets forth, as of March 31, 2006, on a
pro forma basis to give effect to our issuance on April 17,
2006 of 21,825,131 shares of series C redeemable
convertible preferred stock, the automatic or voluntary exercise
upon completion of this offering of all outstanding warrants to
purchase 555,003 shares of series B redeemable
convertible preferred stock, and the automatic conversion of all
36
outstanding shares of our redeemable convertible preferred stock
into an aggregate of 84,009,190 shares of common stock upon
the closing of this offering, the total consideration paid
investors in this offering and the average price per share paid,
or to be paid, to us by existing stockholders and by new
investors in this offering at an assumed initial public offering
price of
$ per
share, which is the midpoint of the price range listed on the
cover page of this prospectus, before deducting estimated
underwriting discounts and commissions.
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Total | |
|
|
|
|
Shares Purchased | |
|
Consideration | |
|
Average | |
|
|
| |
|
| |
|
Price Per | |
|
|
Number | |
|
Percent | |
|
Amount | |
|
Percent | |
|
Share | |
|
|
| |
|
| |
|
| |
|
| |
|
| |
Existing stockholders
|
|
|
|
|
|
|
|
% |
|
|
|
|
|
|
|
% |
|
$ |
|
|
New
investors(1)
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Total
|
|
|
|
|
|
|
100 |
% |
|
|
|
|
|
|
100 |
% |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
(1) |
A $1.00 increase (decrease) in the assumed initial public
offering price of
$ per share
would increase (decrease) the total consideration paid by new
investors by $ million and increase (decrease) the
percentage of total consideration paid by new investors by
approximately %, assuming that the
number of shares offered by us, as set forth on the cover page
of this prospectus, remains the same. |
The discussion and tables above exclude:
|
|
|
|
|
14,573,975 shares of common stock issuable upon exercise of
stock options outstanding as of March 31, 2006 at a
weighted average exercise price of $0.43 per share; |
|
|
|
40,000 shares of common stock issuable upon exercise of a
warrant to purchase common stock at an exercise price of
$0.75 per share; |
|
|
|
an aggregate
of shares
of common stock reserved for future issuance under our 2006
equity incentive plan as of the closing of this
offering; and |
|
|
|
an aggregate
of shares
of common stock reserved for future issuance under our 2006
employee stock purchase plan as of the closing of this offering. |
If the underwriters exercise their over-allotment option
in full, the following will occur:
|
|
|
|
|
the percentage of shares of common stock held by existing
stockholders will decrease to
approximately % of the total
number of shares of our common stock outstanding after this
offering; and |
|
|
|
the pro forma as adjusted number of shares held by new investors
will be increased
to ,
or approximately %, of the total
pro forma as adjusted number of shares of our common stock
outstanding after this offering. |
37
SELECTED FINANCIAL DATA
You should read the following selected financial data together
with our financial statements and the related notes appearing at
the end of this prospectus and the Managements
Discussion and Analysis of Financial Condition and Results of
Operations section of this prospectus. We have derived the
statements of operations data for the years ended
December 31, 2003, 2004 and 2005 and the balance sheet data
at December 31, 2004 and 2005 from our audited financial
statements, which are included in this prospectus. We have
derived the statement of operations for the period of
February 4, 2002 (inception) to December 31, 2002, and
the balance sheet data at December 31, 2002 and 2003, from our
audited financial statements, which are not included in this
prospectus. We have derived the statements of operations data
for the three months ended March 31, 2005 and 2006, the
period February 4, 2002 (inception) to March 31, 2006,
and the balance sheet data at March 31, 2006, from our
unaudited financial statements. The unaudited financial
statements include, in the opinion of management, all
adjustments, consisting of only recurring adjustments, that
management considers necessary for the fair presentation of the
financial information set forth in those statements. Our
historical results for any prior period are not necessarily
indicative of results to be expected for any future period.
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Period from | |
|
|
|
Three Months Ended | |
|
Period from | |
|
|
February 4, 2002 | |
|
Year Ended December 31, | |
|
March 31, | |
|
February 4, 2002 | |
|
|
(inception) to | |
|
| |
|
| |
|
(inception) to | |
|
|
December 31, 2002 | |
|
2003 | |
|
2004 | |
|
2005 | |
|
2005 | |
|
2006 | |
|
March 31, 2006 | |
|
|
| |
|
| |
|
| |
|
| |
|
| |
|
| |
|
| |
|
|
|
|
|
|
|
|
|
|
(unaudited) | |
|
(unaudited) | |
|
(unaudited) | |
|
|
|
|
|
|
|
|
|
|
|
|
(Restated) | |
|
(Restated) | |
|
|
(in thousands, except shares and per share data) | |
Statement of Operations Data:
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Revenue
|
|
$ |
|
|
|
$ |
|
|
|
$ |
|
|
|
$ |
|
|
|
$ |
|
|
|
$ |
|
|
|
$ |
|
|
Operating expenses:
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Research and development
|
|
|
1,147 |
|
|
|
4,433 |
|
|
|
6,301 |
|
|
|
13,652 |
|
|
|
2,238 |
|
|
|
5,546 |
|
|
|
30,720 |
|
|
General and administrative
|
|
|
197 |
|
|
|
1,005 |
|
|
|
2,081 |
|
|
|
6,878 |
|
|
|
1,178 |
|
|
|
2,065 |
|
|
|
12,582 |
|
|
Impairment of leasehold improvements
|
|
|
|
|
|
|
1,030 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
1,030 |
|
|
Depreciation and amortization
|
|
|
21 |
|
|
|
132 |
|
|
|
146 |
|
|
|
303 |
|
|
|
47 |
|
|
|
199 |
|
|
|
804 |
|
|
In-process research and development
|
|
|
418 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
418 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Total operating expenses
|
|
|
1,783 |
|
|
|
6,600 |
|
|
|
8,528 |
|
|
|
20,833 |
|
|
|
3,463 |
|
|
|
7,810 |
|
|
|
45,554 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Loss from operations
|
|
|
(1,783 |
) |
|
|
(6,600 |
) |
|
|
(8,528 |
) |
|
|
(20,833 |
) |
|
|
(3,463 |
) |
|
|
(7,810 |
) |
|
|
(45,554 |
) |
Other income (expenses):
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Interest income
|
|
|
13 |
|
|
|
5 |
|
|
|
190 |
|
|
|
610 |
|
|
|
57 |
|
|
|
238 |
|
|
|
1,056 |
|
|
Interest expense
|
|
|
(6 |
) |
|
|
(172 |
) |
|
|
(550 |
) |
|
|
(82 |
) |
|
|
(4 |
) |
|
|
(59 |
) |
|
|
(869 |
) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Loss before tax benefit
|
|
|
(1,776 |
) |
|
|
(6,768 |
) |
|
|
(8,888 |
) |
|
|
(20,305 |
) |
|
|
(3,410 |
) |
|
|
(7,631 |
) |
|
|
(45,367 |
) |
Income tax benefit
|
|
|
|
|
|
|
|
|
|
|
83 |
|
|
|
612 |
|
|
|
|
|
|
|
|
|
|
|
695 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Net loss
|
|
|
(1,776 |
) |
|
|
(6,768 |
) |
|
|
(8,805 |
) |
|
|
(19,693 |
) |
|
|
(3,410 |
) |
|
|
(7,631 |
) |
|
|
(44,672 |
) |
|
Preferred stock accretion
|
|
|
(10 |
) |
|
|
(17 |
) |
|
|
(126 |
) |
|
|
(139 |
) |
|
|
(32 |
) |
|
|
(41 |
) |
|
|
(333 |
) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Net loss attributable to common stockholders
|
|
$ |
(1,786 |
) |
|
$ |
(6,785 |
) |
|
$ |
(8,931 |
) |
|
$ |
(19,832 |
) |
|
$ |
(3,442 |
) |
|
$ |
(7,672 |
) |
|
$ |
(45,005 |
) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Net loss attributable to common stockholders per common
sharebasic and diluted
|
|
|
|
|
|
$ |
(2.94 |
) |
|
$ |
(3.87 |
) |
|
$ |
(6.45 |
) |
|
$ |
(1.49 |
) |
|
$ |
(1.81 |
) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Weighted-average common shares outstandingbasic and diluted
|
|
|
|
|
|
|
2,306,541 |
|
|
|
2,306,541 |
|
|
|
3,076,649 |
|
|
|
2,314,804 |
|
|
|
4,228,564 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Unaudited pro forma net loss
|
|
|
|
|
|
|
|
|
|
|
|
|
|
$ |
(19,692 |
) |
|
|
|
|
|
$ |
(7,631 |
) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Unaudited pro forma basic and diluted net loss per share
|
|
|
|
|
|
|
|
|
|
|
|
|
|
$ |
(0.23 |
) |
|
|
|
|
|
$ |
(0.09 |
) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Unaudited shares used to compute pro forma basic and diluted net
loss per share
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
87,085,839 |
|
|
|
|
|
|
|
88,237,754 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
38
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
As of December 31, | |
|
|
|
|
| |
|
As of March 31, | |
|
|
2002 | |
|
2003 | |
|
2004 | |
|
2005 | |
|
2006 | |
|
|
| |
|
| |
|
| |
|
| |
|
| |
|
|
|
|
|
|
|
|
|
|
(unaudited) | |
|
|
(in thousands) | |
Balance Sheet Data:
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Cash and cash equivalents and marketable securities
|
|
$ |
1,341 |
|
|
$ |
15 |
|
|
$ |
4,336 |
|
|
$ |
24,418 |
|
|
$ |
19,552 |
|
Working capital
|
|
|
947 |
|
|
|
(5,588 |
) |
|
|
3,569 |
|
|
|
22,267 |
|
|
|
16,006 |
|
Total assets
|
|
|
1,919 |
|
|
|
501 |
|
|
|
5,073 |
|
|
|
28,670 |
|
|
|
23,995 |
|
Total liabilities
|
|
|
752 |
|
|
|
5,776 |
|
|
|
922 |
|
|
|
3,327 |
|
|
|
5,819 |
|
Redeemable convertible preferred
stock(1)
|
|
|
2,416 |
|
|
|
2,432 |
|
|
|
20,014 |
|
|
|
60,469 |
|
|
|
60,509 |
|
Deficit accumulated during the development stage
|
|
|
(1,775 |
) |
|
|
(8,503 |
) |
|
|
(17,348 |
) |
|
|
(37,040 |
) |
|
|
(44,671 |
) |
Total stockholders (deficiency)
|
|
$ |
(1,249 |
) |
|
$ |
(7,708 |
) |
|
$ |
(15,863 |
) |
|
$ |
(35,126 |
) |
|
$ |
(42,334 |
) |
|
|
(1) |
In April 2006, we completed the sale of an additional
21,825,131 shares of series C redeemable convertible
preferred stock for proceeds of $27.5 million. After
evaluating the fair value of our common stock issuable upon
conversion by our preferred stockholders, we determined that the
issuance of the series C redeemable convertible preferred
stock sold in April 2006 will result in a beneficial conversion
feature of $19.4 million which will be recorded as a deemed
dividend to preferred stockholders during the second quarter of
2006. |
39
MANAGEMENTS DISCUSSION AND ANALYSIS OF FINANCIAL
CONDITION
AND RESULTS OF OPERATIONS
You should read the following discussion and analysis of our
financial condition and results of operations together with our
financial statements and the related notes appearing at the end
of this prospectus. Some of the information contained in this
discussion and analysis or set forth elsewhere in this
prospectus, including information with respect to our plans and
strategy for our business and related financing, includes
forward-looking statements that involve risks and uncertainties.
You should read the Risk Factors section of this
prospectus for a discussion of important factors that could
cause actual results to differ materially from the results
described in or implied by the forward-looking statements
contained in the following discussion and analysis. We expressly
disclaim any obligation or intention to provide updates to the
forward-looking statements contained herein and the estimates
and assumptions associated with them.
Overview
We are a clinical-stage biopharmaceutical company focused on the
discovery, development and commercialization of novel small
molecule, orally-administered drugs for the treatment of a range
of human genetic diseases. Human genetic diseases result from
mutations in specific genes that, in many cases, lead to the
production of proteins that fold improperly or with reduced
stability. Proteins with these mutations may not achieve their
correct three-dimensional shape and are generally referred to as
misfolded proteins. Misfolded proteins are often recognized by
cells as having folding defects and, as a result, are eliminated
prior to reaching their intended location in the cell. The
reduced or completely absent biological activity of these
proteins leads to impaired cellular function and ultimately to
disease. Our novel approach to the treatment of human genetic
diseases consists of using a new type of drug, which we refer to
as a pharmacological chaperone, that selectively binds to the
misfolded protein which increases the stability of the protein
and helps it fold into the correct three-dimensional shape. This
increased stability and corrected folding allow the protein to
move to its proper destination in the cell, where it performs
its intended biological activity. We believe this approach may
restore and improve the function of the affected cells and
ultimately have a beneficial therapeutic effect on the body. We
are currently conducting Phase II clinical studies of our
lead product candidate, Amigal, for Fabry disease. We filed an
IND in April 2006 for our product candidate, AT2101, for Gaucher
disease and have initiated a Phase I study. In addition, we
plan to file an IND by the end of 2006 to initiate clinical
testing of our product candidate, AT2220, for Pompe disease. We
are also leveraging our expertise in pharmacological chaperones
to build an extensive pipeline of product candidates for a range
of other human genetic diseases resulting from misfolded
proteins.
We have generated significant losses as we have progressed our
lead product candidates into clinical development and expect to
continue to generate losses as we continue the clinical
development of Amigal, AT2101, and AT2220. From our inception in
February 2002 through March 31, 2006, we have accumulated a
deficit of $44.7 million. Since we do not generate revenue
from any of our product candidates, our losses will continue as
we continue to conduct our research and development activities.
These activities are budgeted to expand over time and will
require further resources if we are to be successful. As a
result, our operating losses are likely to be substantial over
the next several years. We will need to obtain additional funds
to further develop our research and development programs.
Financial Operations Overview
We have not generated any revenue since our inception. To date,
we have funded our operations primarily through the sale of
equity securities, and capital lease and equipment financings.
If our development efforts result in clinical success,
regulatory approval and successful commercialization of our
products, we could generate revenue from sales of our products.
40
|
|
|
Research and Development Expense |
We expect our research and development expense to increase as we
continue to develop our product candidates. Research and
development expense consists of:
|
|
|
|
|
internal costs associated with our research activities; |
|
|
|
payments we make to third party contract research organizations,
contract manufacturers, investigative sites, and consultants; |
|
|
|
manufacturing development costs; |
|
|
|
personnel related expenses, including salaries, benefits,
travel, and related costs for the personnel involved in drug
discovery and development; |
|
|
|
activities relating to regulatory filings and the advancement of
our product candidates through preclinical studies and clinical
trials; and |
|
|
|
facilities and other allocated expenses, which include direct
and allocated expenses for rent and maintenance of our
facilities, as well as laboratory and other supplies. |
We have multiple research and development projects ongoing at
any one time. We utilize our internal resources, employees and
infrastructure across multiple projects and we do not track time
spent by employees on specific projects. As a result, we do not
record or maintain information regarding internal costs incurred
for our research and development programs on a program specific
basis. In addition, we do not believe that allocating costs on
the basis of estimates of time spent by our employees would
accurately reflect the actual costs of a project. We do,
however, record and maintain information regarding external,
out-of-pocket research
and development expenses on a project specific basis.
We expense research and development costs as incurred, including
payments made to date under our license agreements. We believe
that significant investment in product development is a
competitive necessity and plan to continue these investments in
order to realize the potential of our product candidates. From
our inception in February 2002 through March 31, 2006, we
have incurred research and development expense in the aggregate
of $30.7 million, including stock-based compensation
expense of $0.5 million.
The following table summarizes our principal product development
programs, including the related stages of development for each
product candidate in development, and the
out-of-pocket, third
party expenses incurred with respect to each product candidate
(in thousands).
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Three Months | |
|
Period from | |
|
|
|
|
Ended | |
|
February 4, 2002 | |
|
|
Year Ended December 31, | |
|
March 31, | |
|
(inception) to | |
|
|
| |
|
| |
|
March 31, | |
Product Candidate |
|
2003 | |
|
2004 | |
|
2005 | |
|
2005 | |
|
2006 | |
|
2006 | |
|
|
| |
|
| |
|
| |
|
| |
|
| |
|
| |
Third party direct project expenses
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Amigal (Fabry Disease Phase II)
|
|
$ |
3,041 |
|
|
$ |
4,547 |
|
|
$ |
5,579 |
|
|
$ |
1,452 |
|
|
$ |
800 |
|
|
$ |
13,967 |
|
|
AT2101 (Gaucher Disease Phase I)
|
|
|
|
|
|
|
26 |
|
|
|
2,164 |
|
|
|
|
|
|
|
2,017 |
|
|
|
4,207 |
|
|
AT2220 (Pompe Disease Preclinical)
|
|
|
|
|
|
|
|
|
|
|
374 |
|
|
|
|
|
|
|
436 |
|
|
|
810 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Total third party direct project expenses
|
|
|
3,041 |
|
|
|
4,573 |
|
|
|
8,117 |
|
|
|
1,452 |
|
|
|
3,253 |
|
|
|
18,984 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
41
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Three Months | |
|
Period from | |
|
|
|
|
Ended | |
|
February 4, 2002 | |
|
|
Year Ended December 31, | |
|
March 31, | |
|
(inception) to | |
|
|
| |
|
| |
|
March 31, | |
Product Candidate |
|
2003 | |
|
2004 | |
|
2005 | |
|
2005 | |
|
2006 | |
|
2006 | |
|
|
| |
|
| |
|
| |
|
| |
|
| |
|
| |
Internal project
costs(1)
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Personnel related costs
|
|
|
1,175 |
|
|
|
1,363 |
|
|
|
4,031 |
|
|
|
580 |
|
|
|
1,616 |
|
|
|
8,589 |
|
|
Other internal costs
|
|
|
217 |
|
|
|
365 |
|
|
|
1,504 |
|
|
|
206 |
|
|
|
677 |
|
|
|
3,146 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Total internal project costs
|
|
|
1,392 |
|
|
|
1,728 |
|
|
|
5,535 |
|
|
|
786 |
|
|
|
2,293 |
|
|
|
11,735 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Total research and development costs
|
|
$ |
4,433 |
|
|
$ |
6,301 |
|
|
$ |
13,652 |
|
|
$ |
2,238 |
|
|
$ |
5,546 |
|
|
$ |
30,719 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
(1) |
We utilize our internal resources across multiple projects and
do not allocate internal costs to specific projects. |
The successful development of our product candidates is highly
uncertain. At this time, we cannot reasonably estimate or know
the nature, timing and costs of the efforts that will be
necessary to complete the remainder of the development of, or
the period, if any, in which material net cash inflows may
commence from Amigal, AT2101, AT2220 or any of our other
preclinical product candidates. This is due to the numerous
risks and uncertainties associated with the duration and cost of
clinical trials which vary significantly over the life of a
project as a result of differences arising during clinical
development, including:
|
|
|
|
|
the number of clinical sites included in the trials; |
|
|
|
the length of time required to enroll suitable patients; |
|
|
|
the number of patients that ultimately participate in the
trials; and |
|
|
|
the results of our clinical trials. |
Our expenditures are subject to additional uncertainties,
including the terms and timing of regulatory approvals, and the
expense of filing, prosecuting, defending and enforcing any
patent claims or other intellectual property rights. We may
obtain unexpected results from our clinical trials. We may elect
to discontinue, delay or modify clinical trials of some product
candidates or focus on others. A change in the outcome of any of
the foregoing variables with respect to the development of a
product candidate could mean a significant change in the costs
and timing associated with the development of that product
candidate. For example, if the FDA or other regulatory
authorities were to require us to conduct clinical trials beyond
those which we currently anticipate will be required for the
completion of clinical development of a product candidate, or if
we experience significant delays in enrollment in any our
clinical trials, we could be required to expend significant
additional financial resources and time on the completion of
clinical development. Drug development may take several years
and millions of dollars in development costs.
|
|
|
General and Administrative Expense |
General and administrative expense consists primarily of
salaries and other related costs, including stock-based
compensation expense, for persons serving in our executive,
finance, accounting, information technology and human resource
functions. Other general and administrative expense includes
facility-related costs not otherwise included in research and
development expense, advertising expenses, promotional expenses,
costs associated with industry and trade shows, and professional
fees for legal services, including patent-related expense and
accounting services. We expect that our general and
administrative expenses will increase as we add personnel and
become subject to the reporting obligations applicable to public
companies. From our inception in February 2002 through
March 31, 2006, we spent $12.6 million, including
stock-based compensation expense of $0.7 million, on
general and administrative expense.
42
|
|
|
Beneficial Conversion Feature |
In March 2006, the investors in our series C redeemable
convertible preferred stock financing committed to the purchase
of an additional 21,825,131 shares of series C
redeemable convertible preferred stock. These shares were issued
for proceeds of $27.5 million in April 2006. After
evaluating the fair value of our common stock issuable upon
conversion by the holders of the shares, we determined that the
issuance of the additional shares of series C redeemable
convertible preferred stock resulted in a beneficial conversion
feature calculated in accordance with Emerging Issues Task Force
(EITF) Issue No. 98-5, Accounting for Convertible
Securities with Beneficial Conversion Features or Contingently
Adjustable Conversion Ratios, as interpreted by EITF Issue
No. 00-27,
Application of Issue No. 98-5 to Certain Convertible
Instruments, of $19.4 million which will be recorded as
a deemed dividend to preferred stockholders for the three month
period ended June 30, 2006.
The accompanying financial statements for the quarter ended
March 31, 2006 were restated as we determined that our
deemed dividend related to the Series C redeemable
convertible preferred stock should have been recorded as a
second quarter event instead of a first quarter event. The
deemed dividend was initially recorded in the financial
statements as of the measurement date instead of the date the
shares were issued, which occurred in April 2006. The change had
no impact on net loss for the three months ended March 31,
2006 and decreased net loss attributable to common shareholders
for the three months ended March 31, 2006 by
$19.4 million; resulting in an increase to basic and
diluted earnings per share attributable to common stockholders
of $4.60, from ($6.41) to ($1.81). This change was confined to
the first quarter of 2006 only and had no impact on the related
balance sheet accounts and statement of cash flows. The
statement of changes in stockholders deficiency were revised to
remove the beneficial conversion charges, however, the net
impact was zero.
|
|
|
Interest Income and Interest Expense |
Interest income consists of interest earned on our cash and cash
equivalents and marketable securities. Interest expense consists
of interest incurred on our capital lease facility.
Critical Accounting Policies and Significant Judgments and
Estimates
The discussion and analysis of our financial condition and
results of operations are based on our financial statements,
which we have prepared in accordance with U.S. generally
accepted accounting principles. The preparation of these
financial statements requires us to make estimates and
assumptions that affect the reported amounts of assets and
liabilities and the disclosure of contingent assets and
liabilities at the date of the financial statements, as well as
the reported revenues and expenses during the reporting periods.
On an ongoing basis, we evaluate our estimates and judgments,
including those described in greater detail below. We base our
estimates on historical experience and on various other factors
that we believe are reasonable under the circumstances, the
results of which form the basis for making judgments about the
carrying value of assets and liabilities that are not readily
apparent from other sources. Actual results may differ from
these estimates under different assumptions or conditions.
While our significant accounting policies are more fully
described in Note 2 to our financial statements appearing
at the end of this prospectus, we believe that the following
accounting policies are the most critical to aid you in fully
understanding and evaluating our financial condition and results
of operations.
43
As part of the process of preparing our financial statements, we
are required to estimate accrued expenses. This process involves
identifying services that have been performed on our behalf and
estimating the level of service performed and the associated
cost incurred for the service when we have not yet been invoiced
or otherwise notified of actual cost. The majority of our
service providers invoice us monthly in arrears for services
performed. We make estimates of our accrued expenses as of each
balance sheet date in our financial statements based on facts
and circumstances known to us. Examples of estimated accrued
expenses include:
|
|
|
|
|
fees owed to contract research organizations in connection with
preclinical and toxicology studies and clinical trials; |
|
|
|
fees paid to investigative sites in connection with clinical
trials; |
|
|
|
fees owed to contract manufacturers in connection with the
production of clinical trial materials; |
|
|
|
fees owed for professional services, and |
|
|
|
unpaid salaries, wages, and benefits. |
|
|
|
Adoption of SFAS No. 123(R) |
Effective January 1, 2006, we adopted Statement of
Financial Accounting Standards No. 123(R), Share-Based
Payment (SFAS 123(R)), which requires compensation
costs related to share-based transactions, including employee
stock options, to be recognized in the financial statements
based on fair value. SFAS 123(R) revises SFAS 123, as
amended, Accounting for Stock-Based Compensation
(SFAS 123), and supersedes Accounting Principles Board
(APB) Opinion No. 25, Accounting for Stock Issued
to Employees (APB 25). We adopted SFAS 123(R)
using the prospective method. Under this method, compensation
cost is recognized for all share-based payments granted
subsequent to December 31, 2005. Prior to January 1,
2006, we used the minimum value method, to determine values of
our pro forma stock-based compensation disclosures.
At March 31, 2006, we had one stock-based employee
compensation plan, which is described more fully in Note 7
to our financial statements appearing at the end of this
prospectus. Prior to January 1, 2006, we accounted for this
plan under the recognition and measurement provisions of
APB 25 and related interpretations, as permitted by
SFAS 123. Stock-based employee compensation cost was
recognized in the statement of operations for 2003, 2004 and
2005, to the extent options granted under the plan had an
exercise price that was less than the fair market value of the
underlying common stock on the date of grant. Under the
prospective transition method, compensation cost recognized for
all share-based payments granted subsequent to January 1,
2006 is based on the grant-date fair value estimated in
accordance with the provisions of SFAS 123(R). Results for
prior periods have not been restated. As a result of adopting
SFAS 123(R) on January 1, 2006, our net income for the
period ended March 31, 2006 was less than had we continued
to account for share-based compensation under APB 25.
Prior to the adoption of SFAS 123(R), we presented our
unamortized portion of deferred compensation cost for nonvested
stock options in the statement of changes in shareholders
deficiency with a corresponding credit to additional paid-in
capital. Upon the adoption of SFAS 123(R), these amounts
were offset against each other as SFAS 123(R) prohibits the
gross-up of stockholders equity. Under
SFAS 123(R), an equity instrument is not considered to be
issued until the instrument vests. As a result, compensation
cost is recognized over the requisite service period with an
offsetting credit to additional paid-in capital.
44
The following table illustrates the effect on our net loss and
earnings per share if we had applied the provisions of
SFAS 123 to options granted under our stock option plan for
all periods presented prior to the adoption of SFAS 123(R).
For purposes of this pro forma disclosure, the value of the
options is estimated using a minimum value option-pricing
formula and amortized to expense over the options vesting
periods of the options.
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Three Months | |
|
|
Years Ended December 31, | |
|
Ended | |
|
|
| |
|
March 31, | |
|
|
2003 | |
|
2004 | |
|
2005 | |
|
2005 | |
|
|
| |
|
| |
|
| |
|
| |
Net loss attributable to common stockholders, as reported
|
|
$ |
(6,784,589 |
) |
|
$ |
(8,930,924 |
) |
|
$ |
(19,830,557 |
) |
|
$ |
(3,441,673 |
) |
Add: Non-cash employee compensation
|
|
|
70,340 |
|
|
|
59,842 |
|
|
|
364,551 |
|
|
|
91,138 |
|
Less: Total stock-based employee compensation expense determined
under the minimum value method for all awards
|
|
|
(76,207 |
) |
|
|
(74,499 |
) |
|
|
(437,296 |
) |
|
|
(109,324 |
) |
|
|
|
|
|
|
|
|
|
|
|
|
|
Pro forma net loss attributable to common stockholders
|
|
$ |
(6,790,456 |
) |
|
$ |
(8,945,581 |
) |
|
$ |
(19,903,302 |
) |
|
$ |
(3,459,859 |
) |
|
|
|
|
|
|
|
|
|
|
|
|
|
Net loss attributable to common stockholders per common share:
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Basic and fully diluted:
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
As reported
|
|
$ |
(2.94 |
) |
|
$ |
(3.87 |
) |
|
$ |
(6.45 |
) |
|
$ |
(1.49 |
) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Pro forma
|
|
$ |
(2.94 |
) |
|
$ |
(3.88 |
) |
|
$ |
(6.47 |
) |
|
$ |
(1.49 |
) |
|
|
|
|
|
|
|
|
|
|
|
|
|
We recognized employee compensation expense of $70,340, $59,842,
$364,551, and $91,138 and $315,671 for the years ended 2003,
2004 and 2005 and for the three months ended March 31, 2005
and 2006, respectively.
During the three months ended March 31, 2006, we recorded
incremental compensation expense of approximately $152,000
($0.04 per basic and diluted share) related to the
expensing of our options under SFAS 123(R) during the
quarter. The compensation expense had no impact on our cash
flows from operations and financing activities. The total
compensation cost related to non-vested stock option awards
issued during 2006 but not yet recognized as of March 31,
2006 was approximately $7.1 million. This expense will be
recorded on a
straight-line basis
over approximately four years.
Upon adoption of SFAS 123(R), we selected the Black-Scholes
option pricing model as the most appropriate model for
determining the estimated fair value for stock-based awards. The
fair value of stock option awards subsequent to
December 31, 2005 is amortized on a straight-line basis
over the requisite service periods of the awards, which is
generally the vesting period. Use of a valuation model requires
management to make certain assumptions with respect to selected
model inputs. Expected volatility was calculated based on a
blended weighted average of historical information of our stock
and the weighted average of historical information of similar
public entities for which historical information was available.
We will continue to use a blended weighted average approach
using our own historical volatility and other similar public
entity volatility information until our historical volatility is
relevant to measure expected volatility for future option
grants. The average expected life was determined according to
the SEC shortcut approach as described in Staff Accounting
Bulletin, or SAB, 107, Disclosure about Fair Value of
Financial Instruments, which is the mid-point between the
vesting date and the end of the contractual term. The risk-free
interest rate is based on U.S. Treasury zero-coupon issues
with a remaining term equal to the expected life assumed at the
date of grant. Forfeitures are estimated based on voluntary
termination
45
behavior, as well as a historical analysis of actual option
forfeitures. The assumptions used in the Black-Scholes option
pricing model are as follows:
|
|
|
|
|
|
|
Three Months | |
|
|
Ended | |
|
|
March 31, 2006 | |
|
|
| |
Expected stock price volatility
|
|
|
72.70 |
% |
Risk free interest rate
|
|
|
4.59 |
% |
Expected life of options (years)
|
|
|
6.25 |
|
Expected annual dividend per share
|
|
$ |
0.00 |
|
The weighted-average fair value (as of the date of grant) of the
options granted during the three months ended March 31,
2006 is $1.52.
During the three months ended March 31, 2006 we granted
stock options with exercise prices as follows:
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Retrospective | |
|
|
|
|
|
|
|
|
Fair Value | |
|
|
|
|
Number of | |
|
|
|
Estimate per | |
|
Intrinsic Value | |
Grant Date |
|
Options Granted | |
|
Exercise Price | |
|
Common Share | |
|
per Share | |
|
|
| |
|
| |
|
| |
|
| |
January 2, 2006
|
|
|
17,000 |
|
|
$ |
0.71 |
|
|
$ |
1.44 |
|
|
$ |
0.73 |
|
January 12, 2006
|
|
|
5,000 |
|
|
|
0.71 |
|
|
|
1.44 |
|
|
|
0.73 |
|
February 6, 2006
|
|
|
5,000 |
|
|
|
0.71 |
|
|
|
1.44 |
|
|
|
0.73 |
|
February 9, 2006
|
|
|
23,000 |
|
|
|
0.71 |
|
|
|
1.44 |
|
|
|
0.73 |
|
February 13, 2006
|
|
|
7,500 |
|
|
|
0.71 |
|
|
|
1.44 |
|
|
|
0.73 |
|
February 22, 2006
|
|
|
35,000 |
|
|
|
0.71 |
|
|
|
1.44 |
|
|
|
0.73 |
|
February 28, 2006
|
|
|
5,752,500 |
|
|
|
0.71 |
|
|
|
1.84 |
|
|
|
1.13 |
|
March 27, 2006
|
|
|
50,000 |
|
|
|
0.71 |
|
|
|
1.84 |
|
|
|
1.13 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
5,895,000 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
The exercise prices for options granted were set by our board of
directors, the members of which have extensive experience in the
life sciences industry and all but one of whom are non-employee
directors, with input from our management, based on our
determination of the fair market value of our common stock at
the time of the grants. In connection with the preparation of
the financial statements for this offering, we performed a
retrospective determination of fair value for financial
reporting purposes of our common stock underlying stock option
grants in 2005 and 2006 utilizing a combination of valuation
methods described in the AICPA Technical Practice Aid,
Valuation of Privately-Held-Company Equity Securities Issued as
Compensation (Practice Aid). Information on stock option
grants during 2005 is as follows:
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Retrospective | |
|
|
|
|
|
|
|
|
Fair Value | |
|
Intrinsic | |
|
|
Number of | |
|
Average | |
|
Estimate per | |
|
Value | |
|
|
Options | |
|
Exercise | |
|
Common | |
|
per | |
Date of 2005 Issuance |
|
Granted | |
|
Price | |
|
Share | |
|
Share | |
|
|
| |
|
| |
|
| |
|
| |
January - May
|
|
|
3,037,037 |
|
|
$ |
0.09 |
|
|
$ |
0.31 |
|
|
$ |
0.22 |
|
June - July
|
|
|
1,768,748 |
|
|
|
0.09 |
|
|
|
0.77 |
|
|
|
0.68 |
|
August - September
|
|
|
315,500 |
|
|
|
0.22 |
|
|
|
0.95 |
|
|
|
0.73 |
|
October - November
|
|
|
2,351,000 |
|
|
|
0.71 |
|
|
|
1.14 |
|
|
|
0.43 |
|
December
|
|
|
104,500 |
|
|
|
0.71 |
|
|
|
1.44 |
|
|
|
0.73 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
7,576,785 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
46
Determining the fair value of the common stock of a private
enterprise requires complex and subjective judgments. Our
retrospective estimates of enterprise value at each of the grant
dates during 2005 and 2006 used results from both the income
approach and the market approach.
Under the income approach, our enterprise value was based on the
present value of our forecasted operating results. Our revenue
forecasts were based on our estimates of expected annual growth
rates following the anticipated commercial launch of our product
candidates Amigal, AT2101 and AT2220. Estimated operating
expenses were based on our internal assumptions, including
continuing research and development activities for Amigal,
AT2101, AT2220 and other preclinical candidates, and preparation
and ongoing support for the commercialization of our lead
product candidates. The assumptions underlying the estimates are
consistent with our business plan. The risks associated with
achieving our forecasts were assessed in selecting the
appropriate discount rates, which were approximately 25% to 35%.
Under the market approach, our estimated enterprise value was
developed based on a comparison of pre-money initial public
offering, or IPO, values of recent biotechnology and emerging
pharmaceutical companies at a similar stage of development to
ours. When we achieved or exceeded a significant milestone, we
reduced the discount rate applied to determine our enterprise
value.
Once our enterprise value was established, an allocation method
was used to allocate the enterprise value to the different
classes of equity instruments. During our retrospective review,
we used the probability weighted expected returns, or
PWER, method to allocate our enterprise value to our common
stock. Under the PWER method, the value of common stock is
estimated based upon an analysis of future values for the
enterprise assuming various future outcomes. In our situation,
the future outcomes included two scenarios: (i) we become a
public company and; (ii) we remain a private company. In
general, the closer a company gets to an IPO, the higher the
probability assessment weighting is for that scenario. We used a
low probability assumption for our January 2005 grants and this
percentage increased as significant milestones were achieved and
as discussions with our investment bankers began and continued
to increase as we prepared for our IPO process. An increase in
the probability assessment for an IPO increases the value
ascribed to our common stock.
For each of the two scenarios, estimated future and present
value for the common shares were calculated using assumptions
including:
|
|
|
|
|
our expected pre-IPO valuation; |
|
|
|
a risk-adjusted discount rate associated with the IPO scenario; |
|
|
|
the liquidation preferences of our redeemable convertible
preferred stock; |
|
|
|
appropriate discount for lack of marketability assuming we
remained a private company; |
|
|
|
the expected probability of completing IPO versus remaining a
private company; and |
|
|
|
the estimated timing of a potential IPO. |
The increase in the fair value of our common stock for financial
reporting purposes during 2005 and 2006 principally reflects a
significant increase in our probability weighting for the IPO
scenario and increases resulting from achieving significant
clinical milestones.
The reassessed fair value for financial reporting purposes of
common stock underlying 3,037,037 options granted to employees
during the period from January 2005 through May 2005 was
$0.31 per share. This valuation was attributable to the
hiring of our President and Chief Executive Officer and other
members of executive management and a relatively low probability
estimate for the IPO scenario under the PWER method.
The reassessed fair value for financial reporting purposes of
common stock underlying 1,768,748 options granted to employees
during the period from June 2005 through July 2005 was
determined to be $0.77 per share based on the ongoing
clinical trial of Amigal, additional development of our
preclinical programs, and an increased probability estimate for
the IPO scenario under the PWER method.
47
The reassessed fair value for financial reporting purposes of
common stock underlying 315,500 options granted to
employees during the period from August 2005 through September
2005 was determined to be $0.95 per share. This increase in
valuation was based on the completion of Phase I clinical
trials for Amigal and completion of our series C redeemable
convertible preferred stock financing of $55 million.
The reassessed fair value for financial reporting purposes of
common stock underlying 2,351,000 options granted to employees
during the period from October 2005 through November 2005 was
determined to be $1.14 per share. This increase was
primarily based on positive developments in the capital markets
for early stage life science companies, the start of
Phase II clinical trials for Amigal, and further
preclinical development of our other programs.
The reassessed fair value for financial reporting purposes of
common stock underlying 104,500 options granted to employees in
December 2005 and 92,500 options granted to employees in the
period from January 1, 2006 to February 22, 2006 was
determined to be $1.44 per share. This increase was
primarily based on preclinical development of AT2101 and AT2220,
as well as an acceleration of our IPO planning.
The reassessed fair value for financial reporting purposes of
common stock underlying 5,802,500 options granted to employees
in the period from February 28, 2006 to March 27, 2006
was determined to be $1.84 per share. This increase was
primarily based on initial data from our Phase II studies
in Fabry disease and a further acceleration of our IPO timeline.
The intrinsic value of all outstanding vested and unvested
options based on the estimated IPO price of
$ was
$ based
on 14,573,975 options outstanding at March 31, 2006.
Results of Operations
|
|
|
Three Months Ended March 31, 2006 Compared to Three
Months Ended March 31, 2005 |
Research and Development Expense. Research and
development expense was $5.5 million for the three months
ended March 31, 2006, an increase of $3.3 million, or
150%, from $2.2 million for the three months ended
March 31, 2005. We attribute the increase primarily to a
rise in contract research and manufacturing costs of
$1.8 million due to our continued development of AT2101 and
AT2220, and increases in personnel related costs of
$1.1 million.
During the remainder of 2006, and thereafter, we expect research
and development expenses to continue to increase substantially
as our existing and future product candidates proceed through
clinical trials. The timing and amount of these expenses will
depend upon the outcome of our current clinical trials,
particularly the costs associated with our current Phase II
clinical trials of Amigal and Phase I clinical trial of
AT2101 and our planned Phase I clinical trials of AT2101
and AT2220. The timing and amount of these expenses will also
depend on the costs associated with potential future clinical
trials of our product candidates, and the related expansion of
our research and development organization, regulatory
requirements, advancement of our preclinical programs and
product candidate manufacturing costs.
General and Administrative Expense. General and
administrative expense was $2.1 million for the three
months ended March 31, 2006, an increase of
$0.9 million, or 75%, from $1.2 million in for the
three months ended March 31, 2005. The increase resulted
primarily from an increase of personnel costs of
$0.6 million attributable to increased headcount in
finance, information technology, human resources, and general
management and an increase of facility related expense of
$0.4 million related to our new facility.
During the remainder of 2006, and thereafter, we expect our
general and administrative expenses to increase substantially as
we add personnel, increase investor relations activities, obtain
insurance coverage appropriate for a public company, and become
subject to public reporting obligations.
Interest Income and Interest Expense. Interest income was
$238,000 in the three months ended March 31, 2006, compared
to $57,000 in the three months ended March 31, 2005.
Interest expense was $59,000 in the three months ended
March 31, 2006, compared to $4,000 in the three months
ended March 31, 2005. The increase in interest income
resulted from higher average cash and cash equivalents
48
balances and higher average interest rates in the 2006 period.
The increase in interest expense resulted from a increase in our
equipment financing and capital lease obligations as we
continued to expand our business.
|
|
|
Year Ended December 31, 2005 Compared to Year Ended
December 31, 2004 |
Research and Development Expense. Research and
development expense was $13.7 million in 2005, an increase
of $7.4 million, or 117%, from $6.3 million in 2004.
The increase resulted primarily from an increase in contract
research costs for Amigal, AT2101 and AT2220 of
$3.5 million during 2005, and a rise in personnel related
costs of $2.7 million.
General and Administrative Expense. General and
administrative expense was $6.9 million in 2005, an
increase of $4.8 million, or 228%, from $2.1 million
in 2004. This increase is primarily attributable to a rise in
salaries, as well as an increase in headcount in finance, human
resources, information technology and general management,
including the hiring of many of our current senior executives.
Interest Income and Interest Expense. Interest income was
$610,000 in 2005, compared to $190,000 in 2004. Interest expense
was $82,000 in 2005, compared to $550,000 in 2004. The increase
in interest income resulted from higher average cash and cash
equivalents balances and higher average interest rates in 2005.
The reduction in interest expense resulted from the conversion
of our bridge loans into series B redeemable convertible
preferred stock during 2004.
Tax Benefit. In 2005 and 2004, we recognized tax benefits
related to our sale of net operating losses in the New Jersey
Tax Transfer Program. Our tax benefit was $612,000 in 2005 and
$83,000 in 2004. We sold $6.7 million and $1.1 million
of net operating losses in 2005 and 2004, respectively.
|
|
|
Year Ended December 31, 2004 Compared to Year Ended
December 31, 2003 |
Research and Development Expense. Research and
development expense was $6.3 million in 2004, an increase
of $1.9 million, or 42%, from $4.4 million in 2003.
The increase resulted principally from an increase in contract
research costs attributable to preclinical development
activities for Amigal and AT2101 of $1.5 million, and an
increase in personnel costs of $0.2 million.
General and Administrative Expense. General and
administrative expense was $2.1 million in 2004, an
increase of $1.0 million, or 110%, from $1.0 million
in 2003. The increase resulted principally from an increase in
personnel costs of $0.1 million attributable to a rise in
salaries and increased headcount, greater legal and consulting
expense of $0.4 million, and an increase in miscellaneous
corporate expenses of $0.2 million.
Interest Income and Interest Expense. Interest income was
$190,000 in 2004, compared to $5,000 in 2003. Interest expense
was $550,000 in 2004, compared to $172,000 in 2003. The increase
in interest income resulted from higher average cash and cash
equivalents balances and higher average interest rates in 2004.
The increase in interest expense resulted from bridge loans
issued in 2003 and early 2004 which were converted to
series B redeemable convertible preferred stock during 2004.
Tax Benefit. The tax benefit related to our sale of
$1.1 million of net operating losses in the New Jersey Tax
Transfer Program was $83,000 in 2004.
49
Liquidity and Capital Resources
As a result of our significant research and development
expenditures and the lack of any approved products to generate
product sales revenue, we have not been profitable and have
generated operating losses since we were incorporated in 2002.
We have funded our operations principally with
$61.0 million of proceeds from redeemable convertible
preferred stock offerings through March 31, 2006 and
additional $27.5 million of proceeds from redeemable
convertible preferred stock offerings in April 2006. The
following table summarizes our funding sources inclusive of our
April 2006 issuance of an additional 21,825,131 shares of
series C redeemable convertible preferred stock.
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Approximate | |
Issue |
|
Year | |
|
No. Shares | |
|
Amount(1) | |
|
|
| |
|
| |
|
| |
|
|
|
|
|
|
(in millions) | |
Series A Redeemable Convertible Preferred Stock
|
|
|
2002 |
|
|
|
3,333,334 |
|
|
$ |
2.5 |
|
Series B Redeemable Convertible Preferred Stock
|
|
|
2004, 2005 |
|
|
|
36,470,591 |
|
|
|
31.0 |
|
Series C Redeemable Convertible Preferred Stock
|
|
|
2005, 2006 |
|
|
|
43,650,262 |
|
|
|
55.0 |
|
|
|
|
|
|
|
|
|
|
|
Total
|
|
|
|
|
|
|
83,454,187 |
|
|
$ |
88.5 |
|
(1) Represents gross proceeds.
As of March 31, 2006, we had cash and cash equivalents and
marketable securities of $19.6 million. An additional
$27.5 million of cash was raised in connection with our
April 2006 sale of series C redeemable convertible
preferred stock. We hold our cash and investment balances in a
variety of interest-bearing instruments, including obligations
of U.S. government agencies and money market accounts. We
invest cash in excess of our immediate requirements with regard
to liquidity and capital preservation. Wherever possible, we
seek to minimize the potential effects of concentration and
degrees of risk.
Also, we maintain cash balances with financial institutions in
excess of insured limits. We do not anticipate any losses with
respect to such cash balances.
Net cash used in operations was $2.9 million and
$6.1 million for the three months ended March 31, 2005
and 2006, respectively. The net loss for the three months ended
March 31, 2006 of $7.6 million was offset primarily by
non-cash charges for depreciation and amortization of
$0.2 million, stock-based compensation of
$0.3 million, $0.1 of non-cash compensation issued to
consultants, partially offset by changes in operating assets and
liabilities of $0.8 million. Net cash generated in
investing activities for the three months ended March 31,
2006 was $8.1 million and consisted primarily of proceeds
from the sale of marketable securities, partially offset by
$0.6 million of capital expenditures. Net cash provided by
financing activities for the three months ended March 31,
2006 was $1.9 million, consisting primarily of
$2.0 million of proceeds from our capital asset financing
arrangement, offset primarily by payments of equipment debt
financing obligations of $0.2 million.
Net cash used in operations was $9.2 million and
$18.1 million for the years ended December 31, 2004
and 2005, respectively. The net loss for 2005 of
$19.7 million was partially offset by $0.1 million of
non-cash stock issued to consultants, $0.3 million of
depreciation and amortization, and $0.4 million
amortization of non-cash compensation, and a net change in
operating assets and liabilities of $0.8 million. Net cash
used from investing activities for the year ended
December 31, 2005 was $16.9 million and consisted
primarily of $17.0 million of purchases of marketable
securities and $3.0 million of equipment purchases,
partially offset by the sale and redemption of marketable
securities for $3.1 million. Net cash from financing
activities for 2005 was $41.3 million, which consists
primarily of net proceeds from the issuance of series B
redeemable convertible preferred stock of $13.0 million and
net proceeds from the issuance of series C redeemable
convertible preferred stock of $27.3 million.
50
We expect to incur losses from operations for the foreseeable
future. We expect to incur increasing research and development
expenses, including expenses related to the hiring of personnel
and additional clinical trials. We expect that our general and
administrative expenses will also increase as we expand our
finance and administrative staff, add infrastructure, and incur
additional costs related to being a public company, including
directors and officers insurance, investor relations
programs, and increased professional fees. Our future capital
requirements will depend on a number of factors, including the
continued progress of our research and development of products,
the timing and outcome of clinical trials and regulatory
approvals, the costs involved in preparing, filing, prosecuting,
maintaining, defending, and enforcing patent claims and other
intellectual property rights, the acquisition of licenses to new
products or compounds, the status of competitive products, the
availability of financing, and our success in developing markets
for our product candidates.
We believe that the net proceeds from this offering, together
with our existing cash and cash equivalents and short-term
investments, will be sufficient to enable us to fund our
operating expenses and capital expenditure requirements at least
until .
We believe that if we sell
the shares
of our common stock in this offering at an initial public
offering price of
$ per
share ($1.00 lower than the mid-point of the price range set
forth on the cover page), the resultant reduction in proceeds we
receive from the offering would cause us to require additional
capital earlier. We have based this estimate on assumptions that
may prove to be wrong, and we could use our available capital
resources sooner than we currently expect. Because of the
numerous risks and uncertainties associated with the development
and commercialization of our product candidates, we are unable
to estimate the amounts of increased capital outlays and
operating expenditures associated with our current and
anticipated clinical trials.
Our future capital requirements will depend on many factors,
including the progress and results of our clinical trials, the
duration and cost of discovery and preclinical development and
laboratory testing and clinical trials for our product
candidates, the timing and outcome of regulatory review of our
product candidates, the number and development requirements of
other product candidates that we pursue, and the costs of
commercialization activities, including product marketing, sales
and distribution.
We do not anticipate that we will generate product revenue for
at least the next several years. In the absence of additional
funding, we expect our continuing operating losses to result in
increases in our cash used in operations over the next several
quarters and years.
To the extent our capital resources are insufficient to meet our
future capital requirements, we will need to finance our future
cash needs through public or private equity offerings, debt
financings or corporate collaboration and licensing
arrangements. We do not currently have any commitments for
future external funding. We may need to raise additional funds
more quickly if one or more of our assumptions prove to be
incorrect or if we choose to expand our product development
efforts more rapidly than we presently anticipate, and we may
decide to raise additional funds even before we need them if the
conditions for raising capital are favorable. We may seek to
sell additional equity or debt securities or obtain a bank
credit facility. The sale of additional equity or debt
securities, if convertible, could result in dilution to our
stockholders. The incurrence of indebtedness would result in
increased fixed obligations and could also result in covenants
that would restrict our operations.
Additional equity or debt financing, grants, or corporate
collaboration and licensing arrangements may not be available on
acceptable terms, if at all. If adequate funds are not
available, we may be required to delay, reduce the scope of or
eliminate our research and development programs, reduce our
planned commercialization efforts or obtain funds through
arrangements with collaborators or others that may require us to
relinquish rights to certain product candidates that we might
otherwise seek to develop or commercialize independently.
51
Contractual Obligations
The following table summarizes our significant contractual
obligations and commercial commitments at March 31, 2006
and the effects such obligations are expected to have on our
liquidity and cash flows in future periods.
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Remainder of | |
|
|
|
|
|
|
|
|
Total | |
|
2006 | |
|
2007-2009 | |
|
2010-2011 | |
|
2012 | |
|
|
| |
|
| |
|
| |
|
| |
|
| |
Operating lease obligations
|
|
$ |
8,596 |
|
|
$ |
1,019 |
|
|
$ |
4,392 |
|
|
$ |
2,940 |
|
|
$ |
245 |
|
Capital lease obligations
|
|
|
3,341 |
|
|
|
842 |
|
|
|
2,491 |
|
|
|
8 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Total fixed contractual obligations
|
|
$ |
11,937 |
|
|
$ |
1,861 |
|
|
$ |
6,883 |
|
|
$ |
2,948 |
|
|
$ |
245 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
In May 2005, we entered into a seven-year non-cancelable
operating lease agreement for office and laboratory space in
Cranbury, New Jersey. The operating lease will expire by its
terms on February 28, 2012.
In August 2002, we entered into capital lease agreements that
provide for up to $1.0 million of equipment financing
through August 2004. The facility was increased to
$3.0 million in May of 2005 and to $5.0 million in
November 2005. These financing arrangements include interest of
approximately 9-12%, and lease terms of 36 or
48 months. Eligible assets under the lease lines include
laboratory and scientific equipment, computer hardware and
software, general office equipment, furniture, and tenant
improvements. Upon termination of the lease agreements, we may
renew the lease or purchase the leased equipment for $1.00. We
also have the option to purchase the equipment at set prices
before termination of the lease. In addition, at lease
inception, we issued a warrant to the equipment financing lender
to purchase 40,000 shares of common stock. The warrant
was valued at $8,000 using a Black-Scholes option pricing model
and this value was amortized to interest.
We have entered into agreements with clinical research
organizations and other outside contractors who will be
partially responsible for conducting and monitoring our clinical
trials for Amigal and AT2101 as well as preclinical studies of
AT2220. These contractual obligations are not reflected in the
table above because we may terminate them without penalty.
Except for the capital lease agreements described above, we have
no other lines of credit or other committed sources of capital.
To the extent our capital resources are insufficient to meet
future capital requirements, we will need to raise additional
capital or incur indebtedness to fund our operations. We cannot
assure you that additional debt or equity financing will be
available on acceptable terms, if at all.
Quantitative and Qualitative Disclosures About Market Risk
The primary objective of our investment activities is to
preserve our capital to fund operations. We also seek to
maximize income from our investments without assuming
significant risk. To achieve our objectives, we maintain a
portfolio of cash equivalents and investments in a variety of
securities of high credit quality. As of March 31, 2006, we
had cash and cash equivalents and marketable securities of
$19.6 million. A portion of our investments may be subject
to interest rate risk and could fall in value if market interest
rates increase. However, because our investments are short-term
in duration, we believe that our exposure to interest rate risk
is not significant and a 1% movement in market interest rates
would not have a significant impact on the total value of our
portfolio. We actively monitor changes in interest rates.
Effects of Inflation
Inflation generally affects us by increasing our cost of labor
and clinical trial costs. We do not believe that inflation has
had a material effect on our results of operations during 2003,
2004, 2005 or for the three months ended March 31, 2006.
52
Off-Balance Sheet Arrangements
We have no off-balance
sheet arrangements as of December 31, 2004, 2005 and
March 31, 2006.
Recent Accounting Pronouncements
In February 2006, FASB, issued SFAS No. 155,
Accounting for Certain Hybrid Instruments, or
SFAS 155. SFAS 155 allows financial instruments that
have embedded derivatives to be accounted for as a whole
(eliminating the need to bifurcate the derivative from its host)
if the holder elects to account for the whole instrument on a
fair value basis. This statement is effective for all financial
instruments acquired or issued after the beginning of an
entitys first fiscal year that begins after
September 15, 2006. We believe the adoption of
SFAS 155 will not have a material impact on our financial
statements.
In May 2005, FASB issued SFAS No. 154, Accounting
Changes and Error Corrections, or SFAS 154, a
replacement of APB No. 20, Accounting Changes, and
SFAS No. 3, Reporting Accounting Changes in Interim
Financial Statements. SFAS 154 applies to all voluntary
changes in accounting principle and changes the requirements for
accounting for and reporting of a change in accounting
principle. This statement establishes that, unless
impracticable, retrospective application is the required method
for reporting a change in accounting principle in the absence of
explicit transition requirements specific to the newly adopted
accounting principle. It also requires the reporting of an error
correction which involves adjustments to previously issued
financial statements similar to those generally applicable to
reporting an accounting change retrospectively. SFAS 154 is
effective for accounting changes and corrections of errors made
in fiscal years beginning after December 15, 2005.
53
BUSINESS
Overview
We are a clinical-stage biopharmaceutical company focused on the
discovery, development and commercialization of a new class of
orally-administered, small molecule drugs, known as
pharmacological chaperones, for the treatment of a range of
human genetic diseases. Our lead product development programs
are Amigal for Fabry disease, AT2101 for Gaucher disease and
AT2220 for Pompe disease. We are currently conducting
Phase II clinical trials of Amigal and have initiated a
Phase I trial for AT2101. We expect to file an IND for
AT2220 by the end of 2006. We recently observed results from the
first four patients treated with Amigal in our Phase II
trial. We hold worldwide commercialization rights to Amigal,
AT2101 and AT2220 and we intend to establish a commercial
infrastructure and targeted sales force to market some or all of
our products.
Certain human diseases result from mutations in specific genes
that, in many cases, lead to the production of proteins with
reduced stability. Proteins with these mutations may not achieve
their correct three-dimensional shape and are generally referred
to as misfolded proteins. Misfolded proteins are often
recognized by cells as having defects and, as a result, are
eliminated prior to reaching their intended location in the
cell. The reduced or completely absent biological activity of
these proteins leads to impaired cellular function and
ultimately to disease.
Our novel approach to the treatment of human genetic diseases
consists of using a new type of drug, which we refer to as a
pharmacological chaperone, that selectively binds to the target
protein, which increases the stability of the protein and helps
it fold into the correct three-dimensional shape. This restores
appropriate trafficking of the protein, thereby increasing
protein activity, improving cellular function and reducing
stress on cells.
Current treatment for some of these genetic diseases consists of
compensating for the reduced or missing protein activity through
regular infusions with large quantities of protein. Instead of
adding protein from an external source by intravenous infusion,
which is called enzyme replacement therapy, our approach
utilizes orally-administered, small molecule pharmacological
chaperones to improve the function of the patients own
protein. Our approach to the treatment of human genetic diseases
is novel and has the potential to improve the treatment of these
diseases. In addition, we believe our technology is broadly
applicable to diseases that have been linked to misfolded
proteins, including certain types of neurological disease,
metabolic disease, cardiovascular disease and cancer.
Our Lead Programs
Our three most advanced product development programs target
lysosomal storage disorders, which are chronic genetic diseases
that frequently result in severe symptoms. Each of these
disorders results from the deficiency of a single enzyme.
|
|
|
|
|
Amigal for Fabry disease. We are developing Amigal for
the treatment of patients with Fabry disease and are currently
conducting multiple Phase II clinical studies. We expect to
complete enrollment in these studies by the end of 2006 and,
assuming positive results from these studies, to initiate a
Phase III study in 2007. |
|
|
|
|
AT2101 for Gaucher disease. We are developing AT2101 for
the treatment of Gaucher disease. We have filed an IND for
AT2101 and have initiated a Phase I study. If these studies
are successful, we plan to initiate a Phase II study in the
first half of 2007. |
|
|
|
|
AT2220 for Pompe disease. We are developing AT2220 for
the treatment of Pompe disease. We plan to file an IND for
AT2220 by the end of 2006. |
54
Our Pharmacological Chaperone Technology
In the human body, proteins are involved in almost every aspect
of cellular function. Proteins are linear strings of amino acids
that fold and twist into specific three-dimensional shapes in
order to function properly. Certain human diseases result from
mutations in specific genes that lead to the production of
misfolded proteins. The majority of genetic mutations that cause
misfolded proteins are called missense mutations. These
mutations result in the substitution of a single amino acid for
another in the protein. Because of this error, missense
mutations often result in proteins that have a reduced level of
biological activity. In addition to missense mutations, there
are also other types of genetic mutations that can result in
proteins with reduced biological activity.
Proteins generally fold in a specific region of the cell known
as the endoplasmic reticulum, or ER. The cell has quality
control mechanisms that ensure that proteins are folded into
their correct three-dimensional shape before they can move from
the ER to the appropriate destination in the cell, a process
generally referred to as protein trafficking. Misfolded proteins
are often eliminated by the quality control mechanisms after
initially being retained in the ER. In certain instances,
misfolded proteins can accumulate in the ER instead of being
eliminated.
The retention of misfolded proteins in the ER interrupts their
proper trafficking, and the resulting reduced biological
activity can lead to impaired cellular function and ultimately
to disease. In addition, the accumulation of misfolded proteins
in the ER may lead to various types of stress on cells, which
may also contribute to cellular dysfunction and disease.
At Amicus, we have developed a novel approach to address human
genetic diseases resulting from misfolded proteins. We use small
molecule drugs, which are called pharmacological chaperones, to
selectively bind to a target protein and increase its stability.
The binding of the chaperone molecule helps the protein fold
into its correct three-dimensional shape. This allows the
protein to be trafficked from the ER to the appropriate location
in the cell, thereby increasing protein activity and cellular
function and reducing stress on cells.
Pharmacological chaperones represent a new way of affecting
specific proteins, improving cellular function and treating
disease. Our proprietary approach to the discovery of
pharmacological chaperone drug candidates entails the use of
rapid molecular and cell-based screening technology combined
with our understanding of the intended biological function of
proteins implicated in disease. We use this knowledge to select
and develop compounds with optimized properties. In many cases,
we are able to start with specific molecules and classes of
compounds already known to interact with the target protein but
not used previously as therapies. This can greatly reduce the
time and cost of the early stages of drug discovery and
development.
We believe that our pharmacological chaperone technology may be
applicable to many types of diseases that involve misfolded
proteins. In particular, pharmacological chaperone therapies
could, in our view, provide a benefit in areas such as
neurological disease, metabolic disease, cardiovascular disease
and cancer, the causes of which have been linked to various
misfolded proteins. We are also exploring other applications in
which the ability of pharmacological chaperones to increase the
activity of normal proteins may provide a therapeutic benefit.
Potential Advantages of Pharmacological Chaperones for the
Treatment of Lysosomal Storage Disorders
To date, we have focused on developing pharmacological
chaperones for the treatment of lysosomal storage disorders.
Lysosomal storage disorders are a type of metabolic disorder
characterized by mutations in lysosomal enzymes, which are
specialized proteins that break down cellular substrates in a
part of the cell called the lysosome. Substrates are byproducts
of cellular metabolism.
The current therapeutic standard of care for the most common
lysosomal storage disorders is enzyme replacement therapy.
Enzyme replacement therapy involves regular infusions to
compensate for the deficient lysosomal enzyme. A therapeutic
alternative involving the use of small molecules is substrate
reduction therapy. We believe that pharmacological chaperone
therapy may have advantages relative to
55
these other therapeutic approaches for the treatment of
lysosomal storage disorders. The following table compares enzyme
replacement therapy to pharmacological chaperone therapy.
|
|
|
|
|
Product Characteristic |
|
Enzyme Replacement Therapy |
|
Pharmacological Chaperone Therapy |
|
Biodistribution
|
|
Variable tissue distribution |
|
Broad tissue distribution, including brain |
|
Treatment effect
|
|
Reduce substrate accumulation |
|
Reduce substrate accumulation |
|
|
|
|
Reduce accumulation of misfolded protein |
|
Ease of Use
|
|
Weekly or every other week intravenous infusion |
|
Oral administration |
|
Manufacturing
|
|
Recombinant protein manufacturing |
|
Chemical synthesis |
In addition, we believe our pharmacological chaperone therapies
may have advantages relative to substrate reduction therapy.
Substrate reduction therapy uses orally-administered small
molecules; however, the underlying mechanism of action is very
different than for pharmacological chaperones. Substrate
reduction therapies are designed to prevent the production of
the substrate that accumulates in disease by inhibiting an
enzyme required to make the substrate in cells. This is not the
same enzyme that is deficient in the disease. Importantly, the
enzyme that is inhibited is needed to make other molecules that
are used in many biological processes. As a result, inhibiting
this enzyme may have adverse effects on the cell that are
difficult to predict. By contrast, our pharmacological chaperone
therapies are designed to work by binding directly to the enzyme
deficient in the disease, increasing its stability and helping
it fold into its correct three-dimensional shape. This in turn
enables proper trafficking to the lysosome where the enzyme can
directly decrease substrate accumulation.
To date, one substrate reduction therapy product has received
regulatory approval in the United States and the European Union
for the treatment of one lysosomal storage disorder. Zavesca, a
substrate reduction therapy product commercialized by Actelion,
Ltd., is approved for the treatment of Gaucher disease in the
United States, the European Union and other countries.
Our Lead Product Candidates
The following table summarizes key information about our product
candidates. All of our current product candidates are
orally-administered, small molecules based on our
pharmacological chaperone technology.
|
|
|
|
|
Product Candidate Indication |
|
Stage of Development |
|
Worldwide Commercial Rights |
|
Amigal |
|
|
|
|
Fabry Disease |
|
Phase II |
|
Amicus |
|
AT2101 |
|
|
|
|
Gaucher Disease |
|
Phase I |
|
Amicus |
|
AT2220 |
|
|
|
|
Pompe Disease |
|
Preclinical |
|
Amicus |
Our most advanced product candidate, Amigal, is an
orally-administered, small molecule for the treatment of Fabry
disease. We are currently conducting Phase II clinical
studies. Administration of Amigal to the first four patients in
one of these studies, Study 201, resulted in an average five-fold
56
increase in enzyme activity in white blood cells after six weeks
of treatment. After 12 weeks of treatment, enzyme activity
levels remained elevated. The levels of
GL-3 measured in
patient plasma, urine and skin, both before treatment and after
12 weeks of treatment, were in the range of healthy
individuals. GL-3, a
complex lipid called globotriaosylceramide, is the substrate
broken down by the enzyme deficient in Fabry disease. Assuming
the successful completion of our current Phase II clinical
studies, we expect to initiate a Phase III clinical study
of Amigal in 2007. In February 2004, the FDA granted orphan drug
designation to Amigal for the treatment of Fabry disease and in
March 2006, the European Medicines Agency, or EMEA, recommended
orphan medicinal product designation for Amigal.
|
|
|
Causes of Fabry Disease and Rationale for Use of Amigal |
Fabry disease is a lysosomal storage disorder resulting from a
deficiency in a key metabolic enzyme, alpha-galactosidase A,
or α-GAL. The
deficiency of
α-GAL in Fabry patients is caused by inherited
genetic mutations. Certain of these mutations cause changes in
the amino acid sequence of
α-GAL that may result in the production
of α-GAL with
reduced stability that does not achieve its correct
three-dimensional shape. Although
misfolded α-GAL
often retains the potential for some level of biological
activity, the cells quality control mechanisms recognize
and retain misfolded
α-GAL in the ER until it is ultimately moved to
another part of the cell for degradation and elimination.
Consequently, little or no
α-GAL moves to the lysosome, where it normally
breaks down GL-3. This
leads to accumulation of
GL-3 in cells, which is
believed to contribute to most of the complications associated
with Fabry disease. In addition, accumulation of the
misfolded α-GAL
enzyme in the ER may lead to stress on cells and
inflammatory-like responses, which may contribute to cellular
dysfunction and disease. Symptoms can be severe and
debilitating, including dysfunction of major organs such as the
heart, kidneys and brain, leading to cardiac disease, renal
failure and strokes.
Amigal is designed to act as a pharmacological chaperone
for α-GAL by
selectively binding to the enzyme, which increases its stability
and helps the enzyme fold into its correct three-dimensional
shape. This stabilization of
α-GAL allows the cells quality control
mechanisms to recognize the enzyme as properly folded so that
trafficking of the enzyme to the lysosome is increased, enabling
it to carry out its intended biological function, the metabolism
of GL-3. As a result of
restoring the proper trafficking
of α-GAL from the
ER to the lysosome, Amigal reduces the accumulation of misfolded
protein in the ER, which may alleviate stress on cells and some
inflammatory-like responses that may be contributing factors in
Fabry disease.
Because Amigal works by increasing the activity of a
patients naturally
produced α-GAL,
those Fabry disease patients with a missense mutation or other
genetic mutation that results in production
of α-GAL that is
less stable but with some residual enzyme activity are the ones
most likely to respond to treatment with Amigal. We estimate
that the majority of patients with Fabry disease may respond to
pharmacological chaperone therapy. Patients with genetic
mutations leading to a partially
made α-GAL enzyme
or α-GAL enzyme
with an irreversible loss of activity are less likely to respond
to treatment with Amigal.
The clinical manifestations of Fabry disease span a broad
continuum of severity and roughly correlate with a
patients level of
residual α-GAL
activity. The majority of currently treated patients are
referred to as classic Fabry disease patients, most of whom are
males. These patients experience disease of various organs,
including the kidneys, heart and brain, with disease symptoms
first appearing in adolescence and typically progressing in
severity until death in the fourth or fifth decade of life. A
number of recent studies suggest that there are a large number
of undiagnosed male and female patients, referred to as
later-onset Fabry disease patients, with higher levels of
residual α-GAL
activity than classic Fabry disease patients. Later-onset Fabry
disease patients have a broad range of disease symptoms, such as
impaired cardiac function, stroke or renal failure, that usually
first appear in adulthood. Although Fabry disease should be
thought of as a continuum, it is useful to classify patients as
having classic or later-onset Fabry disease when discussing the
disease and its market opportunity.
57
Patients with classic Fabry disease are in most instances males.
They have little or no
detectable α-GAL
activity and are the most severely affected. These patients
first experience disease symptoms in adolescence, including pain
and tingling in the extremities, skin lesions, a decreased
ability to sweat and clouded eyes. If these patients are not
treated, their life expectancy is reduced and death usually
occurs in the fourth or fifth decade of life from renal failure,
cardiac dysfunction or stroke. Studies reported in JAMA
(January 1999) and The Metabolic and Molecular Bases of
Inherited Disease (8th edition 2001) suggest the annual
incidence of Fabry disease in newborn males is
1:40,000-1:60,000. Current estimates from the University of Iowa
and the National Kidney Foundation suggest that there are a
total of approximately 5,000 classic Fabry disease patients
worldwide.
|
|
|
Later-onset Fabry Disease |
Patients with later-onset Fabry disease can be male or female.
These patients typically first experience disease symptoms in
adulthood, and often have disease symptoms focused on a single
organ. For example, many males and females with later-onset
Fabry disease have enlargement of the left ventricle of the
heart. As the patients advance in age, the cardiac complications
of the disease progress and can lead to death. Studies reported
in Circulation, Journal of the American Heart Association
(March 2002 and August 2004), estimate that 6-12% of
patients in the 40 to 60 year age range who have an
unexplained enlargement of the left ventricle of the heart, a
condition referred to as left ventricular hypertrophy, have
Fabry disease.
A number of males and females also have later-onset Fabry
disease with disease symptoms focused on the kidney that
progress to end stage renal failure and eventually death.
Studies reported in Nephrology Dial Transplant (2003), Clinical
and Experimental Nephrology (2005) and Nephrology Clinical
Practice (2005) estimate a general range of 0.20-0.94% of the
patients on dialysis have Fabry disease.
In addition, later-onset Fabry disease may also present in the
form of strokes of unknown cause. A recent published study
reported in The Lancet (November 2005) found that
approximately 4% of 721 male and female patients in Germany
between the ages of 18 to 55 had Fabry disease with stroke of
unknown cause.
It was previously believed that it was rare for female Fabry
disease patients to develop overt clinical manifestations of
Fabry disease. However, several studies reported in the Journal
of Medical Genetics (2001), the Internal Medicine Journal (2002)
and the Journal of Inherited Metabolic Disease (2001), each of
which is summarized on the website of the Mount Sinai School of
Medicine, Department of Human Genetics, report that the majority
of female Fabry disease patients have mild symptoms and that
other female Fabry disease patients have severe symptoms,
including enlargement of the left ventricle of the heart and/or
renal failure. Fabry disease is X-linked, which means that an X
chromosome containing an
α-GAL gene mutation is inherited. Females inherit an
X chromosome from each parent and therefore can inherit a Fabry
mutation from either parent. By contrast, males inherit an X
chromosome (and potentially a Fabry mutation) only from their
mothers. For this reason, there are expected to be roughly twice
as many female Fabry disease patients as male Fabry disease
patients.
|
|
|
Fabry Disease Market Opportunity |
Fabry disease is a relatively rare disorder. The current
estimates of approximately 5,000 patients worldwide are
generally based on a small number of studies in single ethnic
populations in which people were screened for classic Fabry
disease. The results of these studies were subsequently
extrapolated to the broader world population assuming similar
prevalence rates across populations. We believe these previously
reported studies did not account for the prevalence of
later-onset Fabry disease and, as described above, a number of
recent studies suggest that the prevalence of Fabry disease
could be many times higher than previously reported.
58
We expect that as awareness of later-onset Fabry disease grows,
the number of patients diagnosed with Fabry disease will
increase. Increased awareness of all forms of Fabry disease,
particularly for specialists not accustomed to treating Fabry
disease patients, may lead to increased testing and diagnosis of
patients with the disease. We intend to develop and launch
educational and awareness campaigns targeting cardiologists,
nephrologists and neurologists regarding Fabry disease and its
under-diagnosis. Assuming we receive regulatory approval, we
expect these educational and awareness campaigns would continue
as a part of the marketing of Amigal. In order to facilitate the
proper diagnosis of Fabry disease patients seen by these
specialist physicians, we intend to provide support for testing
for the disease, which is performed using a simple blood test
for the level of
α-GAL activity.
Based on published data from the Human Gene Mutation Database
and our experience in the field, we believe the majority of the
known genetic mutations that cause Fabry disease are missense
mutations. There are few widely-occurring genetic mutations
reported for Fabry disease, suggesting that the frequency of a
specific genetic mutation reported in the Human Gene Mutation
Database reflects the frequency of that mutation in the general
Fabry patient population. In addition, data presented at the
11th International Conference on Health Problems Related to
the Chinese (2002) show that the vast majority of newly
diagnosed patients with later-onset Fabry disease also have
missense mutations. Because missense mutations often result in
less stable, misfolded
α-GAL with some residual enzyme activity, we believe
patients with these mutations are candidates for treatment with
Amigal. We also believe that other types of genetic mutations
result in misfolded
α-GAL and therefore may respond to treatment with
Amigal. Based on this, we believe that a majority of the Fabry
disease patient population may benefit from treatment with
Amigal.
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Existing Products for the Treatment of Fabry Disease and
Potential Advantages of Amigal |
Prior to the availability of enzyme replacement therapy,
treatments for Fabry disease were directed at ameliorating
symptoms without treating the underlying disease. Some of these
treatments include opiates, anticonvulsants, antipsychotics and
antidepressants to control pain in the extremities and
beta-blockers, calcium channel blockers, ACE inhibitors,
angiotensin receptor antagonists and other agents to treat blood
pressure and vascular disease.
The current standard of treatment for Fabry disease is enzyme
replacement therapy. There are currently two products approved
for the treatment of Fabry disease. One of the products is
Fabrazyme, a product approved globally and commercialized by
Genzyme Corporation. Fabrazyme was approved in the United States
in 2003 and has orphan drug exclusivity in the United States
until 2010. It was approved in the European Union in 2001 and
has orphan drug exclusivity in the European Union until 2011.
The other product approved for treatment of Fabry disease is
Replagal, a product approved in the European Union and other
countries but not in the United States, commercialized by Shire
PLC. Replagal was approved in the European Union in August 2001
and has orphan drug exclusivity in the European Union until
2011. The net product sales of Fabrazyme and Replagal for 2005
were approximately $305 million and $95 million,
respectively, as publicly reported by Genzyme Corporation and
Shire PLC, respectively.
For Fabry disease patients who respond to Amigal, we believe
that the use of Amigal may have advantages relative to the use
of Fabrazyme and Replagal. Published data for patients treated
with Fabrazyme and Replagal for periods of up to five years
demonstrate that these drugs can lead to the reduction of
GL-3 in the cells that
line the blood vessels in the kidneys of Fabry disease. Because
they are large protein molecules, Fabrazyme and Replagal are
believed to have difficulty penetrating many tissues and cell
types. In particular, it is widely believed that Fabrazyme and
Replagal are unable to cross the blood-brain barrier and thus
are unlikely to address the neurological symptoms of Fabry
disease. As a small molecule therapy that has demonstrated high
oral bioavailability and good biodistribution properties in
preclinical testing, Amigal has the potential to reach cells of
all the target tissues of Fabry disease. Furthermore, treatment
with Fabrazyme and Replagal requires intravenous infusions every
other week, frequently on site at health care facilities,
presenting an inconvenience to Fabry patients. Oral treatment
with Amigal may be much more convenient for patients and may not
have the safety risks associated with
59
intravenous infusions. See Potential Advantages of
Pharmacological Chaperones in the Treatment of Lysosomal Storage
Disorders.
In February 2004, we were granted orphan drug designation by the
FDA for Amigal for treatment of Fabry disease and in March 2006
the EMEA recommended orphan medicinal product designation for
Amigal. We believe that orphan drug designation of Fabrazyme in
the United States and of Fabrazyme and Replagal in the European
Union will not prevent us from obtaining marketing approval of
Amigal in either geography. See Government
Regulation.
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Amigal Development Activities |
We have conducted multiple in vitro and in vivo preclinical
studies of Amigal. Key findings of our studies include:
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Amigal increased
α-GAL enzyme activity in cells derived from a
variety of different Fabry disease patients. Over 75
different α-GAL
missense mutations have been examined in cell culture assays
with the majority showing an increase
in α-GAL enzyme
activity after incubation with Amigal for several days. |
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Treatment of normal mice and mice that produce defective
human α-GAL
resulted in a dose-dependent increase
in α-GAL enzyme
activity in a variety of tissues including liver, heart, kidney
and spleen. The table below summarizes the results of treatment
with Amigal in mice that produce a defective
human α-GAL. |
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Note: Error bars indicate standard error of the mean. |
Amigal had an acceptable toxicity profile when tested at high
exposure levels in rats, dogs and monkeys. Amigal showed no
signs of systemic toxicity in two-week studies in rats, dogs and
monkeys, in six-month studies in rats and in nine-month studies
in monkeys when tested at levels that were well above those that
we are studying in our current Phase II clinical trials. In
the nine-month monkey study, all doses were well tolerated and
showed no signs of toxicity.
Some treatment-related impacts on reproduction and fertility
have been observed in rabbit and rat studies. At high exposure
levels that were well above those that we are studying in our
current Phase II clinical trials, maternal toxicity studies
in rabbits showed a dose-related increase in embryonic death, a
reduction in fetal weight, delayed bone development and slightly
increased incidences of other minor skeletal abnormalities.
These effects were not seen in rats. At exposure levels within
the range of those we are studying in our current Phase II
clinical trials, male rats experienced infertility, which was
completely reversible within four weeks after discontinuation of
treatment. No treatment-related changes have been detected in
the male rat reproductive organs or sperm to account for the
infertility and no mechanism of
60
action has been established to explain this effect. The
implications for humans, if any, of these treatment-related
reproductive and fertility effects in rabbit and rat studies are
unknown at this time.
We have completed three Phase I clinical studies of Amigal
in a total of 63 healthy volunteers, of which 51 were
treated with Amigal and 12 were given placebo.
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Single Dose Phase I Study. Our single dose
Phase I study was a single center, randomized, dose ranging
study in healthy volunteers. The clinical phase began in July
2004 and was completed in November 2004. The study consisted of
a total of 32 healthy volunteers divided into four groups
of eight subjects. Six subjects in each group received Amigal
and two subjects received placebo. All subjects received single
doses of 25 mg, 75 mg, 225 mg or 675 mg of
Amigal and were evaluated on Day 1 and on Day 8. The primary
objective of the study was to evaluate the safety and
pharmacokinetics of Amigal in healthy volunteers. |
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Multi-Dose Phase I Study. Our multi-dose
Phase I study was a single center, randomized, dose ranging
study in healthy volunteers. The clinical phase began in
December 2004 and was completed in January 2005. The study
consisted of a total of 16 healthy volunteers divided into
two groups of eight subjects. Six subjects in each group
received Amigal and two subjects received placebo. All subjects
in one group received 50 mg twice a day for seven days, and
all subjects in the other group received 150 mg twice a day
for seven days. Subjects were evaluated at the beginning of the
study, on Day 7 after seven days of treatment and on
Day 14 after a seven day washout period. The primary
objectives of the study were to evaluate the safety and
pharmacokinetics of Amigal in healthy volunteers and to measure
the level of α-GAL
enzyme activity in white blood cells of healthy volunteers
treated with Amigal. |
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Absorption Phase I Study. Our absorption
Phase I study was a single center, randomized, three-way
crossover, three-sequence, comparative study in healthy
volunteers. The clinical phase began in August 2005 and was
completed in September 2005. The study consisted of a total of
15 healthy volunteers divided into three groups of five
subjects. All subjects in a group received a single dose of
100 mg of Amigal as a capsule while fasting, as a capsule
after a meal or as a solution while fasting. After being
evaluated on Day 1 and on Day 8, patients in each
group crossed over to one of the other treatments. This was
repeated again after the second treatment so that each group of
patients received each of the three treatments. The primary
objective of the study was to evaluate the bioequivalency
between solution and capsule forms and the effect of food on
absorption from the capsule. |
The data from our three Phase I clinical studies in healthy
volunteers showed that Amigal was well tolerated, even at the
highest doses, without any drug related adverse effects. The
studies also demonstrate that Amigal has high oral
bioavailability, good pharmacokinetics with a half-life in
plasma of approximately three to four hours and that the drug
should be taken without food.
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In addition, the data from the multi-dose Phase I study
showed a dose-related increase in the level
of α-GAL activity
in the white blood cells of healthy volunteers. The results are
summarized below in the following graph.
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Note: Error bars indicate standard error of the mean. |
We believe these Phase I results are the first
demonstration of an increase in enzyme activity in humans
following oral administration of a pharmacological chaperone. In
normal, healthy individuals treated with Amigal for seven days
we observed a dose-related increase in enzyme activity, with the
increase maintained for at least seven days after the last dose.
We believe normal enzyme activity can be increased because some
fraction of normal protein molecules can also misfold and fail
to pass the cells quality control mechanisms. Normal
α-GAL is stabilized by binding to the pharmacological
chaperone, which results in an increase in the amount
successfully trafficked to the lysosome. We believe the
sustained elevation of enzyme activity levels following
discontinuation of treatment occurs because the enzyme is stable
for many days once it reaches the lysosome.
We are conducting open-label Phase II clinical studies in
up to 48 adult male and female patients with Fabry disease.
These studies may enroll patients that have classic Fabry
disease, as well as patients with later-onset Fabry disease,
including females and patients with cardiac symptoms.
In order to qualify for these clinical studies, patients must
have a confirmed diagnosis of Fabry disease with a documented
missense mutation in
α-GAL and a positive result in an in vitro test
of α-GAL enzyme
activity enhancement with Amigal. This in vitro test
requires a simple blood draw and consists of incubation of a
patients cells derived from white blood cells, with and
without Amigal for a period of time followed by measurement
of α-GAL enzyme
activity. For entry into the Phase II clinical studies,
patients must have a baseline
α-GAL activity level in white blood cells of at
least 3% of normal and have cells derived from white blood cells
that show a relative increase of at least 20%
in α-GAL activity
after cell culture incubation with Amigal.
We expect to complete enrollment of our current Phase II
studies by the end of 2006.
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Phase II Study 201. We are conducting a
Phase II study in which four patients are currently
enrolled. The first patient was enrolled in January 2006 and the
study is expected to complete enrollment by the end of 2006. The
study consists of treatment with Amigal for a period of twelve
weeks with a possible extension up to 48 weeks in up to
20 male Fabry disease patients that are naïve to
enzyme replacement therapy or have not had enzyme replacement
therapy for at least one month. All four patients have received
25 mg of Amigal twice a day for two weeks, followed by |
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100 mg of Amigal twice a day for two weeks, followed by
250 mg of Amigal twice a day for two weeks and followed by
25 mg of Amigal twice a day for six weeks. These patients
are currently receiving 25 mg of Amigal twice a day for the
extension phase of this study. Based on
the α-GAL activity
data observed in the first four patients after 12 weeks of
treatment with Amigal, we may amend this protocol to replace the
in vitro assay screening criteria with an in vivo drug
exposure in which patients would be given Amigal for a short
period and then tested to determine
if α-GAL activity
has increased. We believe this would allow us to enroll a larger
segment of patients with mutations likely to respond to
treatment with Amigal. We are also considering modifying the
dosing regimen for this study. |
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Phase II Study 202. We are conducting a
Phase II study in which we are seeking to enroll patients.
The study consists of treatment with Amigal for a period of
24 weeks with a possible extension to 48 weeks in up
to eight male Fabry disease patients that are naïve to
enzyme replacement therapy. All patients will receive
150 mg of Amigal every other day during the duration of the
study. |
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Phase II Study 203. We are conducting a
Phase II study in which we are seeking to enroll patients.
The study consists of treatment with Amigal for a period of
12 weeks with a possible extension to 48 weeks in up
to eight male Fabry disease patients that are naïve to
enzyme replacement therapy. All patients will receive
150 mg of Amigal every other day during the duration of the
study. |
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Phase II Study 204. We are conducting a
Phase II study in which we are seeking to enroll patients.
The study consists of treatment with Amigal for a period of 12
weeks with a possible extension to 48 weeks in up to 12
female Fabry disease patients that are naïve to enzyme
replacement therapy. Patients will receive 50 mg,
150 mg or 250 mg doses of Amigal every other day for
12 weeks. If the patient participates in the extension
phase, the dose for the extension will be determined based on
data from the first 12 weeks. |
The primary objective of the Phase II clinical studies is
to evaluate the safety and tolerability of Amigal in patients
with Fabry disease. The secondary objective is to evaluate
certain pharmacodynamic measures of treatment with Amigal. These
pharmacodynamic measures consist of the following:
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α-GAL activity in
white blood cells and skin biopsies; |
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α-GAL activity in
heart and kidney biopsies (not performed in Study 201); |
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GL-3 in plasma, urine
and skin biopsies; and |
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GL-3 in heart and
kidney biopsies (not performed in Study 201). |
An additional objective is the preliminary assessment of
Amigals impact on cardiac, renal and central nervous
system function in Fabry disease patients.
Initial
Results of Phase II Study 201
The initial data from the first four Fabry disease patients
enrolled in Study 201 showed that
the α-GAL enzyme
activity in white blood cells after six weeks of treatment was
on average more than five-fold higher than before treatment. The
four patients had three different genetic mutations and we
observed
63
an increase in the level of
α-GAL enzyme activity in all of these patients. The
graph below summarizes these data.
Note: Error bars indicate standard error of the mean.
After six weeks of treatment, in accordance with the protocol,
the dose was decreased to the same 25 mg of Amigal used in
the first two weeks of the study. Patients received this lower
dose for six weeks. An analysis of the 12 week data from
the same four patients has been completed and a summary is
provided below:
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Enzyme activity in white blood cells remained elevated at levels
approximately four-fold higher than baseline. Enzyme activity in
skin was increased in two of the four patients. Results of
α-GAL enzyme activity levels in skin of the other two
patients were inconclusive due to insufficient biopsy sample
size. |
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GL-3 levels in patient plasma, urine and skin, both before and
after 12 weeks of treatment, were in the normal range of
healthy individuals. |
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Measures of cardiac, renal and central nervous system function
before treatment were normal or near normal, and no clinically
meaningful changes were observed after 12 weeks of
treatment. |
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To enroll in this clinical study, patients were required to have
greater than or equal to 3% of
normal α-GAL
enzyme activity level in white blood cells. We believe that this
residual level of
α-GAL activity is responsible for the normal levels
of GL-3 in the plasma, urine and skin of these patients, before
treatment with Amigal and thus why levels were unchanged after
treatment. We believe the results from the 12 weeks of
treatment of the first four patients enrolled in Study 201
support the continuation of our current Phase II clinical
studies. It is generally believed that even small increases in
lysosomal enzyme activity may have clinical benefits.
Amigal was well-tolerated with no reported serious adverse
events. Adverse events were mostly mild and reported by the
investigators as unlikely to be related to Amigal. A fifth
patient with a history of hypertension discontinued study
treatment due to increased blood pressure, which was reported by
the investigator as possibly related to the study drug.
The results of our Phase II clinical studies to date do not
necessarily predict final results for our Phase II clinical
studies. The results from additional patients in our ongoing
Phase II clinical studies or additional data from these
first four patients may cause the results of Study 201 or our
other Phase II studies to differ from or be less favorable
than the initial data presented above. We cannot assure you that
our Phase II clinical studies will ultimately be successful.
Assuming successful completion of our Phase II clinical
studies, we expect to initiate a Phase III clinical study
of Amigal in Fabry patients in 2007.
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AT2101 for Gaucher Disease |
Our second most advanced clinical product candidate, AT2101, is
an orally-administered, small molecule for the treatment of
Gaucher disease. In April 2006, we filed an IND for AT2101 in
Gaucher disease, and have initiated a Phase I clinical
study. Assuming the successful completion of our Phase I
clinical studies, we expect to initiate a Phase II clinical
study of AT2101 in Gaucher patients in the first half of 2007.
In February 2006, the FDA granted orphan drug designation to
AT2101.
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Causes of Gaucher Disease and Rationale for Use of AT2101 |
Gaucher disease is a lysosomal storage disorder resulting from a
deficiency in a key enzyme,
β-glucocerebrosidase, or GCase. The deficiency of
GCase in Gaucher patients is caused by inherited genetic
mutations. Certain of these mutations cause changes in the amino
acid sequence of GCase that may result in the production of
GCase with reduced stability that does not achieve its correct
three-dimensional shape. Although misfolded GCase retains the
potential for some level of biological activity, the cells
quality control mechanisms recognize and retain misfolded GCase
in the ER until it is ultimately moved to another part of the
cell for degradation and elimination. Consequently, little or no
GCase moves to the lysosome, where it normally breaks down its
substrate, a complex lipid called glucocerebroside. This leads
to accumulation of glucocerebroside in cells, which is believed
to result in the clinical manifestations of Gaucher disease. In
addition, the accumulation of the misfolded GCase enzyme in the
ER may lead to cellular stress and inflammatory-like responses,
which may contribute to cellular dysfunction and disease.
Symptoms can be severe and debilitating, including an enlarged
liver and spleen, low levels of red blood cells and platelets,
bone pain and fractures. In addition, some patients experience
impairment of the lungs and the central nervous system.
AT2101 is designed to act as a pharmacological chaperone for
GCase by selectively binding to the enzyme, which increases the
stability of the enzyme and helps it fold into its correct
three-dimensional shape. This stabilization of GCase allows the
cells quality control mechanisms to recognize the enzyme
as properly folded so that trafficking of the enzyme to the
lysosome is increased, enabling it to carry out its intended
biological function, the metabolism of glucocerebroside. As a
result of restoring proper trafficking of GCase from the ER to
the lysosome, AT2101 reduces the accumulation of misfolded GCase
in the ER, which may alleviate cellular stress and
inflammatory-like responses that may be contributing factors in
Gaucher disease.
Because AT2101 works by increasing the activity of a
patients naturally produced GCase, those Gaucher disease
patients with a missense mutation or other genetic mutation that
results in production of GCase that is less stable but with some
residual enzyme activity are the ones most likely to respond to
treatment with AT2101. We estimate that the substantial majority
of patients with Gaucher disease may respond to pharmacological
chaperone therapy. Patients with genetic mutations leading to a
partially made GCase enzyme or GCase enzyme with an irreversible
loss of activity are less likely to respond to treatment with
AT2101.
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Gaucher Disease Background |
Gaucher disease is often described in terms of the following
three clinical subtypes:
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Type IChronic Nonneuronopathic Gaucher Disease.
Type I Gaucher disease is the most common subtype and symptoms
usually first appear in adulthood. Type I Gaucher disease is
characterized by the occurrence of an enlarged spleen and liver,
anemia, low platelet counts and fractures and bone pain.
Patients with Type I Gaucher disease do not experience the
neurological features associated with Types II and III
Gaucher disease. The clinical severity of Type I Gaucher disease
is extremely variable with some patients experiencing the full
range of symptoms, while others are asymptomatic throughout most
of their lives. |
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Type IIAcute Neuronopathic Gaucher Disease.
Type II Gaucher disease symptoms typically appear in
infancy with an average age of onset of about three months.
Type II Gaucher disease involves a rapid neurodegeneration
with extensive visceral involvement that usually results in
death before two years of age, typically due to respiratory
complications. The clinical presentation in Type II Gaucher
disease is typically more uniform than Type I Gaucher disease. |
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Type IIISubacute Neuronopathic Gaucher
Disease. Type III Gaucher disease symptoms typically
first appear in infancy or early childhood and involve some
neurological symptoms, along with visceral and bone
complications. Age of onset and disease severity can vary
widely. Disease progression in Type III Gaucher disease is
typically slower than in Type II Gaucher disease. |
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Gaucher Disease Market Opportunity |
Gaucher disease is a relatively rare disorder. According to
estimates reported by the American Society of Health-System
Pharmacists (August 2003) and the National Institute of
Neurological Disorders and Stroke (updated as of January 2006)
there are approximately 10,000 patients worldwide. By far,
Type I Gaucher disease is the most common.
Based on published data from the Human Gene Mutation Database
and our experience in the field, we believe that the majority of
the known genetic mutations that cause Gaucher disease are
missense mutations. Because missense mutations often result in
less stable, misfolded GCase, we believe patients with missense
mutations are candidates for treatment with AT2101. We also
believe that other types of genetic mutations that may result in
misfolded GCase could potentially respond to treatment with
AT2101. The majority of the Type I Gaucher patient
population in the United States, Europe and Israel have the same
missense mutation known as N370S. In preclinical tests, AT2101
has shown the ability to increase GCase activity in cells with
N370S and other mutations that cause Gaucher disease. In
addition, we believe that AT2101 may also benefit some patients
with the neurological forms of Gaucher disease (Type II and
Type III) because of the ability of the small molecule to
cross the blood-brain barrier. Based on this, we believe that a
substantial majority of the Gaucher patient population may
benefit from treatment with AT2101.
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Existing Products for the Treatment of Gaucher Disease and
Potential Advantages of AT2101 |
The current standard of treatment for Gaucher patients is enzyme
replacement therapy. There are currently two products approved
for the treatment of Gaucher disease. One of the products is
Cerezyme, a product approved globally and commercialized by
Genzyme Corporation. Cerezyme was approved in the United States
in 1994 and in the European Union in 1997 and no longer has
orphan drug exclusivity in the United States. In the United
States, Cerezyme is indicated for long-term enzyme replacement
therapy for pediatric and adult patients with a confirmed
diagnosis of Type I Gaucher disease. In the European Union, it
is indicated for long-term enzyme replacement therapy for
pediatric and adult patients with a confirmed diagnosis of Type
I Gaucher disease and for Type III Gaucher disease patients
who exhibit clinically significant non-neurological
manifestations. The other product approved for treatment of
Gaucher disease is Zavesca, a substrate reduction therapy
product approved in the United States, the European Union and
other countries and commercialized by Actelion, Ltd. Zavesca was
approved in the United States in 2003 and has orphan drug
exclusivity in the United States until 2010. It was approved in
the European Union in 2002 and has orphan drug exclusivity in
the European Union until 2012. It is indicated for adults with
mild to moderate Type I Gaucher disease for whom enzyme
replacement therapy is not an option. The net product sales of
Cerezyme and Zavesca for the year 2005 were approximately
$932 million and $11 million, respectively, as
publicly reported by Genzyme Corporation and Shire PLC,
respectively.
For Gaucher disease patients who respond to AT2101, we believe
that the use of AT2101 may have advantages relative to the use
of Cerezyme. Studies in animals show that AT2101 is distributed
throughout the body. Published data demonstrate that treatment
with Cerezyme can lead to the reduction of glucocerebroside in
multiple tissue types, especially the liver and spleen, and to
improve low levels of red blood cells and platelets. Because it
is a large protein molecule, Cerezyme is believed to have
difficulty
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penetrating some tissues and cell types. In particular, it is
widely believed that Cerezyme is unable to cross the blood-brain
barrier and thus unlikely to address the neurological symptoms
of Type II and Type III Gaucher disease. Studies in
animals show that AT2101 crosses the blood-brain barrier,
suggesting that it may provide a clinical benefit to patients
with Type II and Type III Gaucher disease. In
addition, treatment with Cerezyme requires intravenous infusions
every other week, presenting an inconvenience to Gaucher disease
patients. Oral treatment with AT2101 may be more convenient for
patients and may not have the safety risks associated with
intravenous infusions. See Potential Advantages of
Pharmacological Chaperones in the Treatment of Lysosomal Storage
Disorders.
We also believe that AT2101 may have advantages over the use of
Zavesca, a substrate reduction therapy. Zavesca is an
orally-administered small molecule; however, the underlying
mechanism of action is very different than for pharmacological
chaperones. Substrate reduction therapies are designed to
prevent the production of the substrate that accumulates in
disease by inhibiting an enzyme required to make the substrate
in cells. This is not the same enzyme that is deficient in
Gaucher disease. Importantly, the enzyme that is inhibited is
needed to make other important molecules that are used for many
types of biological processes. As a result, inhibiting this
enzyme may have adverse effects on the cell that are difficult
to predict. By contrast, AT2101 is designed to work by binding
directly to GCase, increasing its stability and helping it fold
into its correct three-dimensional shape. This in turn enables
proper trafficking to the lysosome where it can directly
decrease substrate accumulation. Several side effects were
reported by Actelion, Ltd. in clinical trials of Zavesca,
including diarrhea, which was observed in more than 85% of
patients who received the drug. Other side effects included hand
tremors and numbness and tingling in the hands, arms, legs or
feet. AT2101 is designed to work by a very different mechanism
than Zavesca, and we do not expect it to have the same
side-effect profile.
In February 2006, the FDA granted orphan drug designation to
AT2101 for the treatment of Gaucher disease. We believe that the
orphan drug designation of Zavesca in the United States and the
European Union will not prevent us from obtaining marketing
approval of AT2101 in either geography. See Government
Regulation.
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AT2101 Development Activities |
We have conducted several in vitro and in vivo preclinical
studies of AT2101. Key findings of our studies include:
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AT2101 increased GCase enzyme activity in cells derived from
Gaucher disease patients with different genetic mutations,
including cells with a genetic mutation associated with
Type II Gaucher disease. |
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In normal mice, oral administration of AT2101 resulted in a
dose-dependent increase in GCase activity in the liver, spleen,
brain and lung. The table below summarizes the results of
administration of AT2101 to normal mice for four weeks. |
Note: Error bars indicate standard error of the mean.
No mortality or morbidity was observed in the
14-day repeat dose,
oral administration studies in rats and monkeys at dose levels
up to 1,500 mg/kg of AT2101. This dose was significantly
higher than the human equivalent doses being considered for our
future clinical studies. The primary treatment-related
toxicities occurred in the stomach linings of rats and the skin
of monkeys, primarily in the eyelids. All toxicities were found
to be reversible or showed a trend toward reversibility. The
clinical implications of these preclinical observations are
unknown at this time. Chronic toxicity testing of AT2101 is
ongoing in six-month rat studies and nine-month monkey studies.
We are currently planning reproductive toxicity and
carcinogenicity studies of AT2101.
In April 2006, we filed an IND for AT2101 in Gaucher disease,
and in June 2006 initiated a Phase I clinical study. We
intend to initiate another Phase I clinical study in the
second half of 2006. The Phase Ia study is evaluating the
effects of AT2101 in subjects who have received a single dose of
AT2101. The Phase Ib study will evaluate the effects of
AT2101 in subjects who have received AT2101 for up to seven
days. Both studies will be conducted in the U.S. and will
include healthy male and female adult volunteers.
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Phase Ia Clinical Study. The Phase Ia clinical
study is a single-center, randomized, double-blind,
placebo-controlled, dose-escalation, oral-dose study in up to 40
healthy volunteers. The main objectives of the study are to
assess the safety and tolerability of a single oral dose of
AT2101 and to evaluate the pharmacokinetics of AT2101 after oral
administration. This study also allows selection of a dose level
that will be used in subsequent clinical studies. |
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Phase Ib Clinical Study. The Phase Ib clinical
study is planned to begin approximately 6 weeks after the
beginning of the Phase Ia clinical study. This study will
be a single-center, randomized, double-blind,
placebo-controlled, dose-escalation, multiple dose study to
evaluate the safety, tolerability and pharmacokinetics of
multiple doses of AT2101 in up to 16 healthy volunteers. |
Assuming successful completion of our Phase I clinical
studies, we expect to initiate a Phase II clinical study of
AT2101 in Gaucher patients in the first half of 2007.
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Our third most advanced product candidate, AT2220, is an
orally-administered small molecule for the treatment of Pompe
disease. AT2220 is currently in preclinical development and we
expect to file an IND for AT2220 in Pompe disease by the end of
2006.
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Causes of Pompe Disease and Rationale for Use of AT2220 |
Pompe disease is a neuromuscular and lysosomal storage disorder
resulting from a deficiency in a key
enzyme,
α-glucosidase, or Gaa. The deficiency of Gaa in
Pompe patients is caused by inherited genetic mutations. Certain
of these mutations cause changes in the amino acid sequence of
Gaa that may result in the production of Gaa with reduced
stability and that does not achieve its correct
three-dimensional shape. Although misfolded Gaa retains the
potential for some level of biological activity, the cells
quality control mechanisms recognize and retain misfolded Gaa in
the ER until it is ultimately moved to another part of the cell
for degradation and elimination. Consequently, little or no Gaa
moves to the lysosome, where it normally breaks down its
substrate, glycogen. This leads to accumulation of glycogen in
cells, which is believed to result in the clinical
manifestations of Pompe disease. In addition, the accumulation
and mistrafficking of Gaa may lead to stress on cells and
inflammatory-like responses, which may contribute to cellular
dysfunction and disease. Symptoms can be severe and
debilitating, including progressive muscle weakness throughout
the body, particularly the heart, skeletal muscles, liver and
nervous system.
AT2220 is designed to act as a pharmacological chaperone for Gaa
by selectively binding to the enzyme, which increases its
stability, and helps the enzyme fold into its correct
three-dimensional shape. This stabilization of Gaa allows the
cells quality control mechanisms to recognize the protein
as properly folded so that trafficking of the enzyme to the
lysosome is increased, enabling it to carry out its intended
biological function, the metabolism of glycogen. As a result of
restoring proper trafficking from the ER to the lysosome, AT2220
may reduce the accumulation of misfolded Gaa in the ER, which
may alleviate cellular stress and inflammatory-like responses
that may be contributing factors in Pompe disease.
Because AT2220 works by increasing the activity of a
patients naturally produced Gaa, those Pompe disease
patients with a missense mutation or other genetic mutation that
results in production of Gaa that is less stable but with some
residual enzyme activity are the ones most likely to respond to
treatment with AT2220. We estimate that the majority of patients
with Pompe disease may respond to pharmacological chaperone
therapy. Patients with genetic mutations leading to a partially
made Gaa enzyme or Gaa enzyme with an irreversible loss of
activity are less likely to respond to treatment with AT2220.
Pompe disease, also known as glycogen storage disease
type II or acid maltase deficiency, is a relatively rare
disorder caused by mutations in Gaa. The mutations in Gaa result
in the accumulation of lysosomal glycogen, especially in
skeletal, cardiac and smooth muscle tissues. According to
reported estimates of the Acid Maltase Deficiency Association,
the United Pompe Foundation and the Lysosomal Disease Program at
Massachusetts General Hospital, there are
5,000-10,000 patients worldwide with Pompe disease.
The onset of Pompe disease ranges from a rapidly fatal infantile
form with severe cardiac involvement to a more slowly
progressive, later-onset form primarily affecting skeletal
muscle. All forms are characterized by severe muscle weakness
that worsens over time. In the rapid onset form, patients are
usually diagnosed shortly after birth and often experience
enlargement of the heart and severe muscle weakness. In
later-onset Pompe disease, symptoms may not appear until late
childhood or adulthood and often experience progressive muscle
weakness.
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Pompe Disease Market Opportunity |
Pompe disease is a relatively rare disorder. Most reported
estimates project that there are 5,00010,000 patients
worldwide, the majority of whom have later-onset Pompe disease.
Based on published data from the Human Gene Mutation Database
and our experience in the field, we believe that the majority of
the known genetic mutations that cause Pompe disease are
missense mutations or mutations that result in measurable
residual enzyme activity. There are a few mutations reported in
Pompe disease that are more common in specific ethnic
populations, including a specific one common in Caucasians with
adult-onset disease. The majority of Pompe patients have either
juvenile or adult-onset disease, and both types of patients
generally have measurable levels of residual enzyme activity.
Because pharmacological chaperone therapy is most likely to have
a benefit in patients with some residual enzyme activity, we
believe that a majority of the Pompe patient population may
benefit from treatment with AT2220.
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Existing Products for the Treatment of Pompe Disease and
Potential Advantages of AT2220 |
The current standard of treatment for Pompe patients is enzyme
replacement therapy. There is currently one product approved for
the treatment of Pompe disease, Myozyme, approved in the United
States and the European Union and commercialized by Genzyme
Corporation. Myozyme was approved in the United States in April
2006 and has orphan drug exclusivity in the United States until
2013. It was approved in the European Union in March 2006 and
has orphan drug exclusivity in the European Union until 2016.
Although Myozyme is approved for use in all Pompe patients,
studies have only been performed in infantile-onset disease.
Myozyme has not been tested for safety and efficacy in
later-onset disease.
For Pompe disease patients who respond to AT2220, we believe
that the use of AT2220 may have advantages relative to the use
of Myozyme. Available data demonstrate that treatment with
Myozyme can improve survival in patients with the infantile form
of the disease. Because it is a large protein molecule, Myozyme
is believed to have difficulty penetrating many tissues and cell
types. As a small molecule that has demonstrated high oral
bioavailability and good biodistribution properties in
preclinical testing, AT2220 has the potential to reach all cells
of the target tissues of Pompe disease patients. Furthermore,
treatment with Myozyme requires intravenous infusions every
other week, frequently on site at health care facilities,
presenting an inconvenience to Pompe disease patients. Oral
treatment with AT2220 may be more convenient for patients and
may not have the safety risks associated with intravenous
infusions. See Potential Advantages of Pharmacological
Chaperones in the Treatment of Lysosomal Storage Disorders.
We believe that the orphan drug designation of Myozyme in the
United States and in the European Union will not prevent us from
obtaining marketing approval of AT2220 in either geography. See
Government Regulation.
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AT2220 Preclinical Development Activities |
We have conducted multiple in vitro and in vivo preclinical
studies of AT2220. Key findings of our studies include:
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AT2220 increased Gaa enzyme activity more than five-fold after
incubation with AT2220 in cells derived from Pompe disease
patients with different genetic mutations. |
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Treatment of normal mice with AT2220 resulted in an increase in
Gaa activity in the heart, brain, diaphragm and calf. The table
below summarizes the results of administration of AT2220 to
normal mice for four weeks. |
Note: Error bars indicate standard error of the mean.
AT2220 demonstrated a favorable pharmacokinetic profile when
tested in rats, including a half life of approximately five
hours and good oral bioavailability. In short-term toxicity
studies in rats (seven days, dosing up to 2,000 mg/kg) and
monkeys (five days, dosing up to 1,000 mg/kg), there were
no observed toxicities related to AT2220 except for diarrhea on
the first day at the high dose in male monkeys. The clinical
implications, if any, of this preclinical observation are
unknown at this time.
We plan to file an IND for AT2220 by the end of 2006 and intend
to develop AT2220 for all forms of Pompe disease.
Other Programs
We believe that our pharmacological chaperone technology is
applicable to the development of drugs for the treatment of a
wide range of human genetic and other diseases. We are
conducting research on the use of pharmacological chaperones for
the treatment of diseases beyond lysosomal storage disorders.
For example, we have identified a lead compound that acts as a
pharmacological chaperone for a protein implicated in one
disease of neurodegeneration. We are also actively engaged in
research on a protein target with an established link to
obesity. In addition, we are investigating targets for which the
ability of pharmacological chaperones to increase normal protein
activity may provide therapeutic benefit.
Our Strategy
Our goal is to become a leading biopharmaceutical company
focused on the discovery, development and commercialization of
pharmacological chaperone therapies for the treatment of a
wide-range of human genetic diseases. To achieve this objective,
we intend to:
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Focus our initial clinical efforts on developing
pharmacological chaperones for certain severe genetic diseases
called lysosomal storage disorders. Our most advanced
programs are for the treatment of Fabry, Gaucher and Pompe
disease. We identify the compounds for these diseases using our
proprietary approach. We believe our pharmacological chaperone
therapy may have advantages over current therapies. We have
focused initially on lysosomal storage disorders for a number of
reasons: |
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the therapeutic targets involved in these diseases are amenable
to rapid drug discovery and development using our
pharmacological chaperone technology; |
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the novel mechanisms of action of our product candidates may
allow us to better address unmet medical needs in these very
debilitating diseases; |
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the severity of these diseases may permit smaller and more
expedited clinical studies; and |
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the specialized nature of these markets allows for small,
targeted sales and marketing efforts that we can pursue
independently. |
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Rapidly advance our lead programs. We are devoting
a significant portion of our resources and business efforts to
completing the development of our most advanced product
candidates. We plan to complete enrollment in our Phase II
clinical studies of Amigal for treatment of Fabry disease and
advance this product candidate into a Phase III clinical
study in 2007. In addition, we have filed an IND to commence
clinical studies of AT2101 for the treatment of Gaucher disease
and plan to advance this compound into a Phase II study in
the first half of 2007. Also, by the end of 2006 we expect to
file an IND for AT2220 for the treatment of Pompe disease. To
accomplish these goals, we are building an appropriate medical,
clinical and regulatory operations infrastructure. In addition,
we are collaborating with physicians, patient advocacy groups,
foundations and government agencies in order to assist with the
development of our products. We plan to pursue similar
activities in future programs. |
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Leverage our proprietary approach to the discovery and
development of additional small molecules. We are
focused on the discovery and development of small molecules
designed to exert therapeutic effects by acting as
pharmacological chaperones. We have steadily advanced these
proprietary technologies and built an intellectual property
position protecting our discoveries over a number of years. Our
technologies span the disciplines of biology, chemistry and
pharmacology. We believe many diseases outside of lysosomal
storage disorders, such as neurologic diseases and other
metabolic diseases, involve misfolded proteins. We also believe
that our approach may be broadly applicable. We plan to continue
to apply our technologies to discovering and developing
treatments for genetic diseases as well as other therapeutic
areas. |
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Build a targeted sales and marketing
infrastructure. We plan to establish our own sales and
marketing capabilities in the U.S. and potentially in other
major markets. We believe that because our current clinical
pipeline is focused on relatively rare genetic disorders, we
will be able to access the market through a focused, targeted
sales force. For example, for Amigal, we believe that the
clinical geneticists who are the key specialists in treating
Fabry disease are sufficiently concentrated that we will be able
to effectively promote the product with our own targeted sales
force. |
Intellectual Property
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Patents and Trade Secrets |
Our success depends in part on our ability to maintain
proprietary protection surrounding our product candidates,
technology and know-how, to operate without infringing the
proprietary rights of others, and to prevent others from
infringing our proprietary rights. Our policy is to seek to
protect our proprietary position by filing U.S. and foreign
patent applications related to our proprietary technology,
including both new inventions and improvements of existing
technology, that are important to the development of our
business. Our patent strategy includes obtaining patent
protection, where possible, on compositions of matter, methods
of manufacture, methods of use, combination therapies, dosing
and administration regimens, formulations, therapeutic
monitoring, screening methods and assays. We also rely on trade
secrets, know-how, continuing technological innovation,
in-licensing and partnership opportunities to develop and
maintain our proprietary position. Lastly, we monitor third
parties for activities that may infringe our proprietary rights,
as well as the progression of third party patent applications
that may have the potential to create blocks to our products or
otherwise interfere with the development of our business. We are
aware, for example, of U.S. patents, and corresponding
international counterparts, owned by third parties that contain
claims related to treating protein misfolding. If any of these
patents were to be
72
asserted against us we do not believe that our proposed products
would be found to infringe any valid claim of these patents.
There is no assurance that a court would find in our favor or
that, if we choose or are required to seek a license, a license
to any of these patents would be available to us on acceptable
terms or at all.
As of the date of this prospectus, we own or license rights to a
total of 10 patents issued in the United States, 5 issued
in current member states of the European Patent Convention and
24 pending foreign applications, which are foreign counterparts
of many of our U.S. patents. We also own or license rights
to 26 pending U.S. applications, 18 of which are
provisional. Our patent portfolio includes patents and patent
applications with claims relating to methods of increasing
deficient enzyme activity to treat genetic diseases. The patent
positions for our three leading product candidates are described
below and include both patents and patent applications we own or
exclusively license:
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We have an exclusive license to five U.S. patents and a
pending U.S. application that cover use of Amigal, as well
as corresponding foreign applications. U.S. patents
relating to Amigal expire in 2018, while the foreign counterpart
patents, if granted, would expire in 2019. The patents and the
pending applications include claims covering methods of
increasing the activity of and preventing the degradation
of α-GAL, and
methods for the treatment of Fabry disease using Amigal and
other specific competitive inhibitors
of α-GAL. In
addition, we own a pending application directed to specific
treatment regimens with Amigal, which may result in a patent
that expires in 2027; pending applications directed to synthetic
steps related to the commercial process for preparing Amigal,
which may result in patents that expire in 2026; and an
application for diagnosis of Fabry patients that will respond to
treatment with Amigal, which may result in a patent that expires
in 2027. |
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We have an exclusive license to seven U.S. patents and a
pending U.S. application, and five foreign patents and one
pending foreign application, that cover AT2101 or its use. Two
of the U.S. patents relating to AT2101 compositions of
matter expire in 2015 and 2016; the five composition of matter
foreign patents and one pending foreign application expire in
2015. The other five U.S. patents and one pending
application, which claim methods of increasing the activity of
and preventing the degradation of GCase, and methods for the
treatment of Gaucher disease using AT2101 and other specific
competitive inhibitors of GCase, expire in 2018. |
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We have an exclusive license to three U.S. patents that
cover use of AT2220, as well as corresponding foreign
applications. The U.S. patents relating to AT2220 expire in
2018, while the foreign counterpart patents, if granted, would
expire in 2019. The patents and the pending applications include
claims covering methods of increasing the activity of and
preventing the degradation of Gaa, and methods for the treatment
of Pompe disease using AT2220 and other specific competitive
inhibitors of Gaa. |
Our patent estate includes patent applications relating to
several other potential product candidates. Some of these
applications are pending in the United States and foreign patent
offices, including two families of patents licensed from Mt.
Sinai School of Medicine. Others have to date only been filed as
provisional applications in the United States. We expect to file
these as non-provisional applications in United States and in
other countries at the appropriate time. One application
licensed from Mt. Sinai is only pending in the United States.
These patent applications, assuming they issue as patents, would
expire in the United States between 2023 and 2027.
Individual patents extend for varying periods depending on the
effective date of filing of the patent application or the date
of patent issuance, and the legal term of the patents in the
countries in which they are obtained. Generally, patents issued
in the United States are effective for:
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the longer of 17 years from the issue date or 20 years
from the earliest effective filing date, if the patent
application was filed prior to June 8, 1995; and |
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20 years from the earliest effective filing date, if the
patent application was filed on or after June 8, 1995. |
73
The term of foreign patents varies in accordance with provisions
of applicable local law, but typically is 20 years from the
earliest effective filing date.
The United States Drug Price Competition and Patent Term
Restoration Act of 1984, more commonly known as the
Hatch-Waxman Act, provides for an extension of
patent protection for drug compounds for a period of up to five
years to compensate for time spent in regulatory review. Similar
provisions are available in European countries, Japan and other
countries. However, we will not know what, if any, extensions
are available until a drug is approved. In addition, in the
U.S. we may be entitled to an additional six month period
of patent exclusivity for pediatric clinical studies.
The patent positions of companies like ours are generally
uncertain and involve complex legal, technical, scientific and
factual questions. Our ability to maintain and solidify our
proprietary position for our technology will depend on our
success in promptly filing patent applications on new
discoveries, and in obtaining effective claims and enforcing
those claims once granted. We seek to protect our proprietary
technology and processes, in part, by contracting with our
employees, collaborators, scientific advisors and our commercial
consultants to ensure that any inventions resulting from the
relationship are disclosed promptly, maintained in confidence
until a patent application is filed and preferably until
publication of the patent application, and assigned to us or
subject to a right to obtain a license. We do not know whether
any of our own patent applications or those patent applications
that are licensed to us will result in the issuance of any
patents. Our issued patents and those that may issue in the
future, or those licensed to us, may be challenged, narrowed
invalidated or circumvented or be found to be invalid or
unenforceable, which could limit our ability to stop competitors
from marketing related products and reduce the term of patent
protection that we may have for our products. Neither we nor our
licensors can be certain that we were the first to invent the
inventions claimed in our owned or licensed patents or patent
applications. In addition, our competitors may independently
develop similar technologies or duplicate any technology
developed by us and the rights granted under any issued patents
may not provide us with any meaningful competitive advantages
against these competitors. Furthermore, because of the extensive
time required for development, testing and regulatory review of
a potential product, it is possible that any related patent may
expire prior to or shortly after commencing commercialization,
thereby reducing the advantage of the patent to our business and
products.
We may rely, in some circumstances, on trade secrets to protect
our technology. However, trade secrets are difficult to protect.
We seek to protect our trade secret technology and processes, in
part, by entering into confidentiality agreements with
commercial partners, collaborators, employees, consultants,
scientific advisors and other contractors, and by contracting
with our employees and some of our commercial consultants to
ensure that any trade secrets resulting from such employment or
consulting are owned by us. We also seek to preserve the
integrity and confidentiality of our data and trade secrets by
maintaining physical security of our premises and physical and
electronic security of our information technology systems. While
we have confidence in these individuals, organizations and
systems, agreements or security measures may be breached, and we
may not have adequate remedies for any breach. In addition, our
trade secrets may otherwise become known or be discovered
independently by others. To the extent that our consultants,
contractors or collaborators use intellectual property owned by
others in their work for us, disputes may arise as to the rights
in related or resulting know-how and inventions.
We have acquired rights to develop and commercialize our product
candidates through licenses granted by various parties. The
following summarizes our material rights and obligations under
those licenses:
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Mt. Sinai School of Medicine We have acquired
exclusive worldwide patent rights to develop and commercialize
Amigal, AT2101 and AT2220 and other pharmacological chaperones
for the treatment of human diseases caused by misfolded proteins
pursuant to a license agreement with Mt. Sinai School of
Medicine of New York University. Under this agreement, to date
we have paid no upfront or annual license fees and we have no
milestone or future payments other than |
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royalties on net sales. In connection with this agreement, we
issued 1,742,000 shares of our common stock to Mt. Sinai School
of Medicine in April 2002. This agreement expires upon
expiration of the last of the licensed patent rights, which will
be in 2019 if a foreign patent is granted and 2018 otherwise. |
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University of Maryland, Baltimore County We have
acquired exclusive U.S. patent rights to develop and
commercialize AT2101 for the treatment of Gaucher disease from
the University of Maryland, Baltimore County. Under this
agreement, to date we have paid aggregate upfront and annual
license fees of $24,500. Upon the satisfaction of certain
milestones and assuming successful development of AT2101, we
could be required to make up to $175,000 in aggregate payments.
We are also required to pay royalties on net sales. This
agreement expires upon expiration of the last of the licensed
patent rights in 2015. |
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Novo Nordisk A/S We have acquired exclusive patent
rights to develop and commercialize AT2101 for all human
indications. Under this agreement, to date we have paid an
aggregate of $400,000 in license fees. Upon the satisfaction of
certain milestones and assuming successful development of
AT2101, we could be required to make up to $7,750,000 in
aggregate payments. We are also required to pay royalties on net
sales. This license will terminate in 2016. |
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Under our license agreements, if we owe royalties on net sales
for one of our products to more than one of the above licensors,
then we have the right to reduce the royalties owed to one
licensor for royalties paid to another. The amount of royalties
to be offset is generally limited in each license and can vary
under each agreement. For Amigal and AT2220, we will owe
royalties only to Mt. Sinai School of Medicine and will owe no
milestone payments. We expect to pay royalties to all three
licensors with respect to AT2101.
Our rights with respect to these agreements to develop and
commercialize Amigal, AT2101 and AT2220 may terminate, in whole
or in part, if we fail to meet certain development or
commercialization requirements or if we do not meet our
obligations to make royalty payments.
In addition to our patents and trade secrets, we have filed
applications to register certain trademarks in the U.S. and
abroad, including AMICUS, AMICUS THERAPEUTICS (and design) and
AMIGAL. At present we do not have any issued registrations for
these marks. Our ability to obtain and maintain trademark
registrations will in certain instances depend on making use of
the mark in commerce on or in connection with our products.
Manufacturing
We rely on contract manufacturers to supply the active
pharmaceutical ingredients for Amigal, AT2101 and AT2220. The
active pharmaceutical ingredients for all three products are
manufactured under current good manufacturing practices, or
cGMP, at kilogram scale initiated with commercially available
starting materials. We also rely on a separate contract
manufacturer to formulate the active pharmaceutical ingredients
into hard gelatin capsules that are also made under cGMP. The
components in the final formulation for each product are
commonly used in other encapsulated products and are well
characterized ingredients. We have implemented appropriate
controls for assuring the quality of both active pharmaceutical
ingredients and the formulated capsules. Product specifications
will be established in concurrence with regulatory bodies at the
time of product registration.
Competition
The biotechnology and pharmaceutical industries are
characterized by rapidly advancing technologies, intense
competition and a strong emphasis on proprietary products. While
we believe that our technologies, knowledge, experience and
scientific resources provide us with competitive advantages, we
face potential
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competition from many different sources, including commercial
pharmaceutical and biotechnology enterprises, academic
institutions, government agencies and private and public
research institutions. Any product candidates that we
successfully develop and commercialize will compete with
existing therapies and new therapies that may become available
in the future.
Many of our competitors may have significantly greater financial
resources and expertise in research and development,
manufacturing, preclinical testing, conducting clinical studies,
obtaining regulatory approvals and marketing approved products
than we do. These competitors also compete with us in recruiting
and retaining qualified scientific and management personnel, as
well as in acquiring technologies complementary to, or necessary
for, our programs. Smaller or early stage companies may also
prove to be significant competitors, particularly through
collaborative arrangements with large and established companies.
Our commercial opportunities could be reduced or eliminated if
our competitors develop and commercialize products that are
safer, more effective, have fewer side effects, are more
convenient or are less expensive than products that we may
develop. In addition, our ability to compete may be affected
because in some cases insurers or other third party payors seek
to encourage the use of generic products. This may have the
effect of making branded products less attractive to buyers.
Our major competitors include pharmaceutical and biotechnology
companies in the United States and abroad that have approved
therapies or therapies in development for lysosomal storage
disorders within our core programs. Other competitors are
pharmaceutical and biotechnology companies that have approved
therapies or therapies in development for genetic diseases for
which pharmacological chaperone technology may be applicable.
Additionally, we are aware of several early-stage, niche
pharmaceutical and biotechnology companies whose core business
revolves around protein misfolding; however, we are not aware
that any of these companies is currently working to develop
products that would directly compete with ours. The key
competitive factors affecting the success of our product
candidates are likely to be their efficacy, safety, convenience
and price.
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Any product candidates that we successfully develop and
commercialize will compete with existing therapies and new
therapies that may become available in the future. The following
table lists our principal competitors and publicly available
information on the status of their product offerings:
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Class of |
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Sales | |
Competitor |
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Indication |
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Product |
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Product |
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Status |
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(in millions) | |
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Genzyme Corporation
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Fabry disease |
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Fabrazyme |
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Enzyme Replacement Therapy |
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Marketed |
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$ |
305 |
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Gaucher disease |
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Cerezyme |
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Enzyme Replacement Therapy |
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Marketed |
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$ |
932 |
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Pompe disease |
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Myozyme |
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Enzyme Replacement Therapy |
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Marketed |
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N/A |
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Gaucher disease |
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Genz-112638 |
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Substrate Reduction Therapy |
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Phase I/II |
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N/A |
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Shire PLC
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Fabry disease |
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Replagal |
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Enzyme Replacement Therapy |
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Marketed |
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$95 |
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Gaucher disease |
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GA-GCB |
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Enzyme Replacement Therapy |
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Phase I/II |
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N/A |
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Actelion, Ltd.
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Gaucher disease |
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Zavesca |
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Substrate Reduction Therapy |
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Marketed |
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$11 |
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We are aware of other companies that are conducting preclinical
development activities for enzyme replacement therapies to treat
Gaucher disease and Pompe disease.
Government Regulation
Government authorities in the United States, at the federal,
state and local level, and in other countries, extensively
regulate, among other things, the research, development,
testing, approval, manufacturing, labeling, post-approval
monitoring and reporting, packaging promotion, storage
advertising, distribution, sampling, marketing, import and
export of pharmaceutical products such as those we are
developing. The process of obtaining regulatory approvals and
the subsequent substantial compliance with appropriate federal,
state, local and foreign statues and regulations require the
expenditure of substantial time and human and financial
resources.
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United States Government Regulation |
In the United States, the information that must be submitted to
the FDA in order to obtain approval to market a new drug varies
depending upon whether the drug is a new product whose safety
and efficacy have not previously been demonstrated in humans or
a drug whose active ingredients and certain other properties are
the same as those of a previously approved drug. A product whose
safety and efficacy have not previously been demonstrated in
humans has to comply with the New Drug Application, or NDA,
approval process.
In the United States, the FDA regulates drugs under the Federal
Food, Drug, and Cosmetic Act and implementing regulations.
Failure to comply with the applicable FDA requirements at any
time during the product development process, approval process or
after approval may result in administrative or judicial
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sanctions. These sanctions could include the FDAs
imposition of a clinical hold on studies, refusal to approve
pending applications, withdrawal of an approval, warning
letters, product recalls, product seizures, total or partial
suspension of production or distribution, injunctions, fines,
civil penalties or criminal prosecution. Any agency or judicial
enforcement action could have a material adverse effect on us.
The steps required before a drug may be marketed in the United
States include:
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completion of preclinical laboratory tests, animal studies and
formulation studies under the FDAs good laboratory
practices regulations; |
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development of adequate manufacturing and quality control
procedures to ensure that clinical supplies meet FDA
requirements for identity, strength, quality and purity; |
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submission to the FDA of an IND for human clinical testing,
which must become effective before human clinical studies may
begin and which must include independent Institutional Review
Board, or IRB, approval at each clinical site before the studies
may be initiated; |
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performance of adequate and well-controlled clinical studies in
accordance with Good Clinical Practices, known as GCP, to
establish the safety and efficacy of the product for each
indication; |
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submission to the FDA of an NDA; |
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satisfactory completion of an FDA Advisory Committee review, if
applicable; |
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satisfactory completion of an FDA inspection of the
manufacturing facility or facilities at which the product is
produced to assess compliance with current good manufacturing
practices, or cGMP, to assure that the facilities, methods and
controls are adequate to preserve the products identity,
strength, quality and purity; and |
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FDA review and approval of the NDA. |
Preclinical tests include laboratory evaluations of product
chemistry, toxicity and formulation, as well as animal studies.
An IND sponsor must submit the results of the preclinical tests,
together with manufacturing information and analytical data, to
the FDA as part of the IND. Some preclinical testing may
continue after the IND is submitted. The IND must become
effective before human clinical studies may begin. An IND will
automatically become effective 30 days after receipt by the
FDA, unless before that time the FDA raises concerns or
questions about issues such as the conduct of the studies as
outlined in the IND. In that case, the IND sponsor and the FDA
must resolve any outstanding FDA concerns or questions before
clinical studies can proceed. In other words, submission of an
IND may not result in the FDA allowing clinical studies to
commence.
Clinical studies involve the administration of the
investigational product to human subjects under the supervision
of qualified investigators. Clinical studies are conducted under
protocols detailing, among other things, the objectives of the
study, the parameters to be used in monitoring safety and the
effectiveness criteria to be evaluated. A protocol for each
clinical study and any subsequent protocol amendments must be
submitted to the FDA as part of the IND. In addition, an IRB at
each site at which the study is conducted must approve the
protocol and any amendments. All research subjects must provide
their informed consent in writing.
Clinical studies typically are conducted in three sequential
phases, but the phases may overlap or be combined. Phase I
studies usually involve the initial introduction of the
investigational drug into healthy volunteers to evaluate the
products safety, dosage tolerance and pharmacokinetics
and, if possible, to gain an early indication of its
effectiveness.
Phase II studies usually involve controlled studies in a
limited patient population to:
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evaluate dosage tolerance and appropriate dosage; |
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identify possible adverse effects and safety risks; and |
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provide a preliminary evaluation of the efficacy of the drug for
specific indications. |
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Phase II studies are sometimes denoted by companies as
Phase IIa or Phase IIb studies. Phase IIa studies
typically represent the first human clinical study of a drug
candidate in a smaller patient population and are designed to
provide earlier information on drug safety and efficacy.
Phase IIb studies typically involve larger numbers of
patients or longer durations of therapy and may involve
comparison with placebo, standard treatments or other active
comparators.
Phase III studies usually further evaluate clinical
efficacy and test further for safety in an expanded patient
population. Phase III studies usually involve comparison
with placebo, standard treatments or other active comparators.
These studies are intended to establish the overall risk-benefit
profile of the product and provide an adequate basis for
physician labeling. Phase III studies are usually larger,
more time consuming, more complex and more costly than
Phase I and Phase II studies. As noted above, Amigal
is currently in Phase II studies for the treatment of Fabry
disease and we have filed an IND to conduct two Phase I
studies for the treatment of Gaucher disease with AT2101.
Phase I, Phase II and Phase III testing may not
be completed successfully within any specified period, if at
all. Furthermore, the FDA or we may suspend or terminate
clinical studies at any time on various grounds, including a
finding that the subjects or patients are being exposed to an
unacceptable health risk. Similarly, an IRB can suspend or
terminate approval of research if the research is not being
conducted in accordance with the IRBs requirements or if
the research has been associated with unexpected serious harm to
patients.
Assuming successful completion of the required clinical testing,
the results of the preclinical studies and of the clinical
studies, together with other detailed information, including
information on the chemistry, manufacture and composition of the
product, are submitted to the FDA in the form of an NDA
requesting approval to market the product for one or more
indications. In most cases, the NDA must be accompanied by a
substantial user fee. The FDA will initially review the NDA for
completeness before it accepts the NDA for filing. After the NDA
submission is accepted for filing, the FDA reviews the NDA to
determine, among other things, whether a product is safe and
effective for its intended use and whether the product is being
manufactured in accordance with cGMP to assure and preserve the
products identity, strength, quality and purity.
Under the Pediatric Research Equity Act of 2003, or PREA, NDAs
or supplements to NDAs must contain data to assess the safety
and effectiveness of the drug for the claimed indications in all
relevant pediatric subpopulations and to support dosing and
administration for each pediatric subpopulation for which the
drug is safe and effective. The FDA may grant deferrals for
submission of data or full or partial waivers. Unless otherwise
required by regulation, PREA does not apply to any drug for an
indication for which orphan designation has been granted.
Before approving an NDA, the FDA will typically inspect one or
more clinical sites to assure compliance with GCP. Additionally,
the FDA will inspect the facility or the facilities at which the
product is manufactured. The FDA will not approve the product
unless cGMP compliance is satisfactory. If the FDA determines
the application, manufacturing process or manufacturing
facilities are not acceptable, it will outline the deficiencies
in the submission and often will request additional testing or
information. Notwithstanding the submission of any requested
additional information, the FDA ultimately may decide that the
application does not satisfy the regulatory criteria for
approval. The testing and approval process requires substantial
time, effort and financial resources, and may take several years
to complete. Data obtained from clinical activities are not
always conclusive and may be susceptible to varying
interpretations, which could delay, limit or prevent regulatory
approval. The FDA may not grant approval on a timely basis, or
at all. We may encounter difficulties or unanticipated costs in
our efforts to secure necessary governmental approvals, which
could delay or preclude us from marketing our products. The FDA
may limit the indications for use or place other conditions on
any approvals that could restrict the commercial application of
the products. After approval, some types of changes to the
approved product, such as adding new indications, manufacturing
changes and additional labeling claims, are subject to further
testing requirements and FDA review and approval.
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Post-Approval Requirements |
After regulatory approval of a product is obtained, we are
required to comply with a number of post-approval requirements.
For example, as a condition of approval of an NDA, the FDA may
require post-marketing testing and surveillance to monitor the
products safety or efficacy. In addition, holders of an
approved NDA are required to report certain adverse reactions
and production problems to the FDA, to provide updated safety
and efficacy information and to comply with requirements
concerning advertising and promotional labeling for their
products. Also, quality control and manufacturing procedures
must continue to conform to cGMP after approval. The FDA
periodically inspects manufacturing facilities to assess
compliance with cGMP, which imposes certain procedural,
substantive and recordkeeping requirements. Accordingly,
manufacturers must continue to expend time, money and effort in
the area of production and quality control to maintain
compliance with cGMP and other aspects of regulatory compliance.
New products that are being developed for the treatment of
serious or life-threatening diseases where the product would
provide therapeutic advantage over the existing treatment may be
considered for accelerated approval by the FDA. In these cases,
approval can be based on surrogate markers that may predict, but
do not establish, clinical benefit. Sponsors of products that
receive accelerated approval must carry out clinical trials
post-approval to verify the desired clinical benefit. If the
post-approval studies fail to demonstrate clinical benefit, the
FDA can withdraw the approval of the drug through expedited
procedures. Promotional materials for products that receive
accelerated approval must be submitted to the FDA for review
prior to use.
We rely, and expect to continue to rely, on third parties for
the production of clinical and commercial quantities of our
product candidates. Future FDA inspections may identify
compliance issues at our facilities or at the facilities of our
contract manufacturers that may disrupt production or
distribution, or require substantial resources to correct. In
addition, discovery of problems with a product or the failure to
comply with applicable requirements may result in restrictions
on a product or a manufacturer or holder of an approved NDA,
including withdrawal or recall of the product from the market or
other voluntary, FDA-initiated or judicial action that could
delay or prohibit further marketing. Newly discovered or
developed safety or effectiveness data may require changes to a
products approved labeling, including the addition of new
warnings and contraindications. Improper promotional activities
can result in significant financial penalties. Also, new
government requirements, including those resulting from new
legislation, may be established that could delay or prevent
regulatory approval of our products under development.
We have received orphan drug designation in the United States
from the FDA for Amigal for the treatment of Fabry disease and
for AT2101 for the treatment of Gaucher disease, and we
anticipate filing for orphan drug designation from the FDA for
AT2220 for the treatment of Pompe disease. The FDA may grant
orphan drug designation to drugs intended to treat a rare
disease or condition that affects fewer than 200,000
individuals in the United States, or more than 200,000
individuals in the United States but for which there is no
reasonable expectation that the cost of developing and making
available in the United States a drug for this type of disease
or condition will be recovered from sales in the United States
for that drug. Orphan drug designation must be requested before
submitting an application for marketing approval. Orphan drug
designation does not convey any advantage in, or shorten the
duration of, the regulatory review and approval process.
Orphan drug designation can provide opportunities for grant
funding towards clinical study costs, tax advantages, and
eliminates the need to submit an FDA user-fee with the NDA. In
addition, if a product which has an orphan drug designation
subsequently receives FDA marketing approval for the indication
for which it has such designation, the product is entitled to
orphan drug exclusivity, which means the FDA may not approve any
other application to market the same drug for the same
indication for a period of seven years, except in limited
circumstances, such as a showing of clinical superiority to the
product with orphan exclusivity. Competitors may receive
marketing approval of different drugs or biologics for the
indications for which the orphan product has exclusivity.
80
As described in the section of this prospectus entitled
Our Lead Product Candidates Amigal for Fabry
Disease Existing Products for the Treatment of Fabry
Disease and Potential Advantages of Amigal and Our
Lead Product Candidates AT2101 for Gaucher
Disease Existing Products for the Treatment of
Gaucher Disease and Potential Advantages of AT2101, we
believe that despite the existence of orphan drug exclusivity
for Fabrazyme for the treatment of Fabry disease and Zavesca for
the treatment of Gaucher disease these exclusivities will not
prevent us from obtaining marketing approval of Amigal and
AT2101 in the United States for the treatment of Fabry disease
and Gaucher disease, respectively, because, among other things,
Amigal and AT2101 are different molecules than Fabrazyme and
Zavesca, respectively.
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Regulation Outside the United States |
In addition to regulations in the United States, we will be
subject to a variety of regulations in other jurisdictions
governing clinical studies and commercial sales and distribution
of our products. Whether or not we obtain FDA approval for a
product, we must obtain approval of a product by the comparable
regulatory authorities of countries outside the United States
before we can commence clinical studies or marketing of the
product in those countries. The approval process varies from
country to country, and the time may be longer or shorter than
that required for FDA approval.
To obtain regulatory approval of a drug under European Union
regulatory systems, we may submit marketing authorizations
either under a centralized or decentralized procedure. The
centralized procedure, which is compulsory for medicines
produced by certain biotechnological processes and optional for
those which are highly innovative, provides for the grant of a
single marketing authorization that is valid for all European
Union member states. The decentralized procedure provides for
approval by one or more other, or concerned, member states of an
assessment of an application performed by one member state,
known as the reference member state. Under this procedure, an
applicant submits an application, or dossier, and related
materials including a draft summary of product characteristics,
and draft labeling and package leaflet, to the reference member
state and concerned member states. The reference member state
prepares a draft assessment and drafts of the related materials
within 120 days after receipt of a valid application.
Within 90 days of receiving the reference member
states assessment report, each concerned member state must
decide whether to approve the assessment report and related
materials. If a member state cannot approve the assessment
report and related materials on the grounds of potential serious
risk to the public health, the disputed points may eventually be
referred to the European Commission, whose decision is binding
on all member states.
We have obtained an orphan medicinal product designation in the
European Union from the EMEA for Amigal for the treatment of
Fabry disease and we anticipate filing for orphan medicinal
product designation from the EMEA for AT2101 for the treatment
of Gaucher disease and for AT2220 for the treatment of Pompe
disease. The EMEA grants orphan drug designation to promote the
development of products that may offer therapeutic benefits for
life-threatening or chronically debilitating conditions
affecting not more than five in 10,000 people in the European
Union. In addition, orphan drug designation can be granted if
the drug is intended for a life threatening, seriously
debilitating or serious and chronic condition in the European
Union and that without incentives it is unlikely that sales of
the drug in the European Union would be sufficient to justify
developing the drug. Orphan drug designation is only available
if there is no other satisfactory method approved in the
European Union of diagnosing, preventing or treating the
condition, or if such a method exists, the proposed orphan drug
will be of significant benefit to patients.
Orphan drug designation provides opportunities for free protocol
assistance and fee reductions for access to the centralized
regulatory procedures before and during the first year after
marketing approval, which reductions are not limited to the
first year after marketing approval for small and medium
enterprises. In addition, if a product which has an orphan drug
designation subsequently receives EMEA marketing approval for
the indication for which it has such designation, the product is
entitled to orphan drug exclusivity, which means the EMEA may
not approve any other application to market the same drug for
the same indication for a period of ten years. The exclusivity
period may be reduced to six years if the
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designation criteria are no longer met, including where it is
shown that the product is sufficiently profitable not to justify
maintenance of market exclusivity. Competitors may receive
marketing approval of different drugs or biologics for the
indications for which the orphan product has exclusivity. In
order to do so, however, they must demonstrate that the new
drugs or biologics provide a significant benefit over the
existing orphan product. This demonstration of significant
benefit may be done at the time of initial approval or in
post-approval studies, depending on the type of marketing
authorization granted.
As described in the section of this prospectus entitled
Our Lead Product Candidates Amigal for Fabry
Disease Existing Products for the Treatment of Fabry
Disease and Potential Advantages of Amigal, we believe
that the orphan designation of Fabrazyme and Replagal in the
European Union will not prevent us from obtaining marketing
approval of Amigal in the European Union for the treatment of
Fabry disease because Amigal will provide significant benefits
over Fabrazyme and Replagal. Similarly, we believe the orphan
drug designation of Zavesca in the European Union will not
prevent us from obtaining marketing approval of AT2101 in the
European Union for the treatment of Gaucher disease because
AT2101 will provide significant benefits over Zavesca.
Pharmaceutical Pricing and Reimbursement
In the United States and markets in other countries, sales of
any products for which we receive regulatory approval for
commercial sale will depend in part on the availability of
reimbursement from third party payors. Third party payors
include government health administrative authorities, managed
care providers, private health insurers and other organizations.
These third party payors are increasingly challenging the price
and examining the cost-effectiveness of medical products and
services. In addition, significant uncertainty exists as to the
reimbursement status of newly approved healthcare product
candidates. We may need to conduct expensive pharmacoeconomic
studies in order to demonstrate the cost-effectiveness of our
products. Our product candidates may not be considered
cost-effective. Adequate third party reimbursement may not be
available to enable us to maintain price levels sufficient to
realize an appropriate return on our investment in product
development.
In 2003, the United States government enacted legislation
providing a partial prescription drug benefit for Medicare
recipients, that began in 2006. Government payment for some of
the costs of prescription drugs may increase demand for any
products for which we receive marketing approval. However, to
obtain payments under this program, we would be required to sell
products to Medicare recipients through managed care
organizations and other health care delivery systems operating
pursuant to this legislation. These organizations would
negotiate prices for our products, which are likely to be lower
than we might otherwise obtain. Federal, state and local
governments in the United States continue to consider
legislation to limit the growth of healthcare costs, including
the cost of prescription drugs. Future legislation could limit
payments for pharmaceuticals such as the drug candidates that we
are developing.
The marketability of any products for which we receive
regulatory approval for commercial sale may suffer if the
government and third party payors fail to provide adequate
coverage and reimbursement. In addition, an increasing emphasis
on managed care in the United States has increased and will
continue to increase the pressure on pharmaceutical pricing.
Scientific Advisory Board
Our scientific advisory board consists of scientific and
clinical advisors who are leading experts in the fields of
lysosomal enzymes, protein folding, protein trafficking and
structure, sugar and carbohydrate chemistry, genetics,
post-transcriptional regulation, preclinical studies, drug
manufacturing and clinical studies. Our scientific advisory
board consults with us regularly on matters relating to:
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our research and development programs; |
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the design and implementation of our clinical studies; |
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market opportunities from a clinical perspective; |
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new technologies relevant to our research and development
programs; and |
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scientific and technical issues relevant to our business. |
Our current scientific advisory board members are:
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Professional Affiliation |
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Michel Bouvier, M.D., Ph.D.
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Professor and Director, Academic Research Group on Therapeutics;
Canada Research Chair in Signal Transduction and Molecular
Pharmacology; Department of Biochemistry, Faculty of Medicine,
University of Montreal |
Gregory Fricchione, M.D.
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Associate Professor of Psychiatry at the Harvard Medical School;
Associate Chief of Psychiatry and Director of the Division of
Psychiatry and Medicine and the Division of International
Psychiatry at Massachusetts General Hospital in Boston |
Bruce Ganem, Ph.D.
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Franz and Elisabeth Roessler Professor of Chemistry;
J. Thomas Clark Professor of Entrepreneurship and Personal
Enterprise, The Johnson School, Cornell University |
Arthur L. Horwich, M.D.
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Professor of Genetics and Pediatrics, Yale University;
Investigator, Howard Hughes Medical Institute |
Stuart A. Kornfeld, M.D.
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Professor, Department of Medicine, Hematology Division;
Professor, Department of Biochemistry & Molecular
Biophysics, Washington University Medical School |
Gregory A. Petsko, D.Phil., Ph.D.
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Gyula and Katica Tauber Professor, Department of Biochemistry
and Department of Chemistry and Director, Rosenstiel Basic
Medical Sciences Research Center, Brandeis University; Adjunct
Professor, Department of Neurology and Center for Neurologic
Diseases, Harvard Medical School |
Employees
As of April 30, 2006, we had 52 full-time employees,
35 of whom were primarily engaged in research and development
activities and 17 of whom provide administrative services. A
total of 24 employees have an M.D. or Ph.D. degree. None of our
employees are represented by a labor union. We have not
experienced any work stoppages and consider our employee
relations to be good.
Property
Our headquarters are located in Cranbury, New Jersey, consisting
of approximately 32,000 square feet of subleased office and
laboratory space. In May 2005, we entered into a seven-year
non-cancelable operating lease agreement for this office and
laboratory space. This operating lease will expire by its terms
on February 28, 2012.
Legal Proceedings
We are not currently a party to any material legal proceedings.
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MANAGEMENT
Our executive officers and directors and their respective ages
and positions as of June 19, 2006 are as follows:
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Age | |
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Position |
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John F. Crowley
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President and Chief Executive Officer and Director |
Matthew R. Patterson
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35 |
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Chief Business Officer |
Pedro Huertas, M.D., Ph.D.
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52 |
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Chief Strategic Officer |
David J. Lockhart, Ph.D.
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44 |
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Chief Scientific Officer |
David Palling, Ph.D.
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Senior Vice President, Drug Development |
Karin Ludwig, M.D.
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Senior Vice President, Clinical Research |
Mark Simon
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Senior Vice President, Business Development |
Gregory P. Licholai, M.D.
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41 |
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Vice President, Medical Affairs |
S. Nicole Schaeffer
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38 |
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Vice President, Human Resources and Leadership Development |
Douglas A. Branch
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Vice President, General Counsel and Secretary |
Donald J.
Hayden(3)
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50 |
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Executive Chairman |
Alexander E.
Barkas, Ph.D.(3)
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58 |
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Director |
Michael G.
Raab(2)(3)
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41 |
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Director |
James N.
Topper, M.D., Ph.D.(1)
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44 |
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Director |
Stephen
Bloch, M.D.(2)
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44 |
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Director |
Gregory M.
Weinhoff, M.D.(1)
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35 |
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Director |
P. Sherrill
Neff(1)
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54 |
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Director |
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(1) |
Member of Compensation Committee. |
(2) |
Member of Audit Committee. |
(3) |
Member of Nominating/Corporate Governance Committee. |
John F. Crowley has served as President and Chief
Executive Officer of Amicus since January 2005, and has also
served as a Director of Amicus since August 2004. He was
President and Chief Executive Officer of Orexigen Therapeutics,
Inc. from 2003 to December 2004. Mr. Crowley was President
and Chief Executive Officer of Novazyme Pharmaceuticals, Inc.,
from March 2000 until that company was acquired by Genzyme
Corporation in September 2001; thereafter he served as Senior
Vice President of Genzyme Therapeutics until December 2002.
Mr. Crowley received a B.S. degree in Foreign Service from
Georgetown Universitys School of Foreign Service, a J.D.
from the University of Notre Dame Law School, and an M.B.A. from
Harvard Business School.
Matthew R. Patterson has served as Chief Business Officer
of Amicus since December 2004. Prior to joining Amicus,
Mr. Patterson was with BioMarin Pharmaceutical Inc. from
1998 to December 2004, most recently serving as Vice President,
Commercial Planning and Government Affairs. Prior to BioMarin,
Mr. Patterson worked at Genzyme Corporation from 1993 to
1998 in Regulatory Affairs and Manufacturing. Mr. Patterson
received a B.A. in Biochemistry from Bowdoin College.
Pedro Huertas, M.D., Ph.D., is our Chief Strategic
Officer and had previously served as our Chief Development
Officer since July 2005. Prior to joining Amicus,
Dr. Huertas was Chief Medical Officer (acting) at
StemCells, Inc. from October 2003 to March 2005.
Dr. Huertas was Chief Medical Officer of Novazyme
Pharmaceuticals, Inc. from 2000 until that company was acquired
by Genzyme Corporation in September 2001; prior to joining
Novazyme he served as Director of Strategic Development and
Medical Director of Genzyme until December 2000.
Dr. Huertas received his undergraduate degree from Stanford
University, his Ph.D. from Harvard University and his M.D. from
the Division of Health Sciences and Technology, Harvard Medical
School Massachusetts Institute of Technology, and
his M.B.A. from the Sloan School at the Massachusetts Institute
of Technology.
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David J. Lockhart, Ph.D., has served as our Chief
Scientific Officer since January 2006. Prior to joining Amicus,
Dr. Lockhart served as President, Chief Scientific Officer
and co-founder of Ambit Biosciences, a biotechnology company
specializing in small molecule kinase inhibitors, from March
2001 to July 2005. Dr. Lockhart served as a consultant to
Ambit Biosciences from August 2000 to March 2001, and as a
visiting scholar at the Salk Institute for Biological Studies
from October 2000 to March 2001. Prior to that,
Dr. Lockhart served in various positions, including Vice
President of Genomics Research at Affymetrix, and was the
Director of Genomics at the Genomics Institute of the Novartis
Research Foundation from February 1999 to July 2000. He received
his Ph.D. from Stanford University and was a post-doctoral
fellow at the Whitehead Institute for Biomedical Research at the
Massachusetts Institute of Technology.
David Palling, Ph.D., has served as Senior Vice
President, Drug Development, since August, 2002. From September
1998 until August, 2002, Dr. Palling was with
Johnson & Johnson, most recently serving as Vice
President of Worldwide Assay Research and Development at Ortho
Clinical Diagnostics, a subsidiary of Johnson &
Johnson. Dr. Palling received B.Sc. and Ph.D. degrees in
Chemistry from the University of London, Kings College,
and conducted post-doctoral research in Biochemistry at Brandeis
University.
Karin Ludwig, M.D., has served as our Senior Vice
President, Clinical Research, since February 2006. From 1993
until February 2006, Dr. Ludwig served in a variety of
clinical research positions at Pharmacia Corporation and
subsequently Pfizer, Inc., after its acquisition of Pharmacia in
2003, most recently Group Leader/ Senior Director,
U.S. Medical, Endocrinology and Ophthalmology. She received
her M.D. from the University Freiburg Medical School.
Mark Simon has served as Senior Vice President, Business
Development since June 2006. Since October 2005 he has
served as an industry consultant to multiple biopharmaceutical
companies. From 2002 to 2005 he was Managing Director and Head
of Life Sciences Investment Banking for Citigroup Global
Markets. From 1989 to 2002 he served as a Senior Research
Analyst and later as Managing Director, Investment Banking for
Robertson Stephens. He received his B.A. from Columbia College
and his M.B.A. from Harvard Business School.
Gregory P. Licholai, M.D., has served as Vice President,
Medical Affairs since December 2004. From November 2002 to
December 2004, Dr. Licholai was with Domain Associates, a
venture capital firm. From September 2000 to November 2002, he
was director of Ventures and Business Associates for Medtronic
Neurological, a division of Medtronic, Inc. Dr. Licholai
received his B.A. from Boston College and completed Pre-Medical
studies at Columbia University, his M.D. from Yale Medical
School and his M.B.A. from Harvard Business School.
S. Nicole Schaeffer has served as Vice President, Human
Resources and Leadership Development since March 2005. From 2001
to 2004, she served as Senior Director, Human Resources, for
three portfolio companies of Flagship Ventures, a venture
capital firm, and in that capacity she managed human resources
for three life sciences companies. Ms. Schaeffer received
her B.A. from the University of Rochester and her M.B.A. from
Boston University.
Douglas A. Branch has served as General Counsel and
Secretary since December 2005, and as Vice President since May
2006. He is also President of Biotech Law Associates, P.C.,
a law firm, where he has practiced since April 2004. From 1996
to April 2004, he was a Director and Shareholder of Phillips
McFall McCaffrey McVay & Murrah, P.C., an Oklahoma
City law firm. He holds B.B.A. (Finance) and J.D. degrees from
the University of Oklahoma.
Donald J. Hayden, Jr. has served as our Chairman
since March 2006. From 1991 to 2005 he held several executive
positions with Bristol-Myers Squibb Company, most recently
serving as Executive Vice President and President, Americas,
Worldwide Medicines Group. Mr. Hayden holds a B.A. from
Harvard University and an M.B.A. from Indiana University.
Alexander E. Barkas, Ph.D., has served as a member of our
board of directors since 2004. Since June 1997, Dr. Barkas
has served as a Managing Member of Prospect Management
Co. II, LLC, a venture
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capital management company. Dr. Barkas also serves as the
chairman of the board of directors of Tercica, Inc. and Geron
Corporation. He holds a B.A. from Brandeis University and a
Ph.D. from New York University.
Michael G. Raab has served as a member of our board of
directors since 2004. Mr. Raab has served as a partner of
New Enterprise Associates since June 2002. From 1999 to 2002,
Mr. Raab was a Senior Vice President, Therapeutics and
General Manager,
Renagel®
at Genzyme Corporation. Mr. Raab holds a B.A. from DePauw
University.
James N. Topper, M.D., Ph.D., has served as a member
of our board of directors since 2004. Dr. Topper has been a
partner with Frazier Healthcare Ventures since August 2003,
holding the position of General Partner since 2004. Prior to
joining Frazier Healthcare, he served as Head of the
Cardiovascular Research and Development Division of Millennium
Pharmaceuticals and ran Millennium San Francisco (formerly
COR Therapeutics) from 2002 until 2003. Prior to the merger of
COR and Millennium in 2002, Dr. Topper served as the Vice
President of Biology at COR from August 1999 to
February 2002. He holds an appointment as a Clinical
Assistant Professor of Medicine at Stanford University and as a
Cardiology Consultant to the Palo Alto Veterans Administration
Hospital. Dr. Topper currently serves on the board of
La Jolla Pharmaceuticals Company. Dr. Topper holds an
M.D. and a Ph.D. in Biophysics from Stanford University School
of Medicine.
Stephen Bloch, M.D., has served as a member of our board
of directors since 2004. He has served as a Principal at Canaan
Partners since June 2002. Prior to joining Canaan,
Dr. Bloch founded and served as the Chief Executive Officer
of Radiology Management Sciences, a risk manager of diagnostic
imaging services for health plans and provider networks, from
1995 to 2002. Dr. Bloch received his M.D. from the
University of Rochester. He also received a M.A. in history of
science from Harvard University and an A.B. degree in history
from Dartmouth College.
Gregory M. Weinhoff, M.D. has served as a member of our
board of directors since our inception. Since 2001,
Dr. Weinhoff has served as a Member of Collinson
Howe & Lennox II, L.L.C., the general partner of
CHL Medical Partners II, L.P. Dr. Weinhoff served as our
founding Chief Executive Officer from inception until October
2002. From 2000 to 2001, Dr. Weinhoff was a Senior
Associate at Whitney & Co. Dr. Weinhoff holds an
A.B. degree from Harvard College, an M.D. degree from Harvard
Medical School and an M.B.A. degree from Harvard Business School.
P. Sherrill Neff has served as a member of our board of
directors since 2005. Mr. Neff is a founding partner and
has served as the managing partner of Quaker BioVentures, L.P.
since 2002. Prior to forming Quaker BioVentures, L.P., he was
President, Chief Operating Officer, and a director of Neose
Technologies, Inc. from 1994 to 2002. Mr. Neff currently
sits on the board of Resource Capital Corporation. Mr. Neff
is a graduate of Wesleyan University and the University of
Michigan Law School.
Board Compensation and Election of Directors
Our board of directors is currently authorized to have, and we
currently have, eight members. In accordance with the terms of
our certificate of incorporation and bylaws that will become
effective upon the closing of this offering, our board of
directors will be divided into three classes, class I,
class II and class III, with each class serving
staggered three-year terms. Upon the closing of this offering,
the members of the classes will be divided as follows:
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the class I directors will
be ,
and ,
and their term will expire at the annual meeting of stockholders
to be held in 2007; |
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the class II directors will
be , ,
and ,
and their term will expire at the annual meeting of stockholders
to be held in 2008; and |
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|
the class III directors will
be , and and
their term will expire at the annual meeting of stockholders to
be held in 2009. |
86
Our certificate of incorporation to be effective upon the
closing of this offering provides that our directors may be
removed only for cause and by the affirmative vote of the
holders of a majority of our voting stock. Upon the expiration
of the term of a class of directors, directors in that class
will be eligible to be elected for a new three-year term at the
annual meeting of stockholders in the year in which their term
expires.
All of our current directors, except John F. Crowley and
Donald J. Hayden, are independent directors, as defined by
the applicable rules of The Nasdaq National Market. We refer to
these directors as our independent directors. Upon
the closing of this offering each of these independent directors
will serve on one or more of our audit committee, compensation
committee and nominating and corporate governance committees.
There are no family relationships among any of our directors or
executive officers.
Board Committees
Our board currently has established an audit committee, a
compensation committee and a nominating/corporate governance
committee. The composition of each committee is effective
currently but we expect will be modified prior to the closing of
this offering.
The members of our audit committee are Dr. Bloch and
Mr. Raab. Mr. Raab chairs the audit committee. Our
audit committee assists our board of directors in its oversight
of the integrity of our financial statements, our independent
registered public accounting firms qualifications and
independence and the performance of our independent registered
public accounting firm.
Upon closing of this offering, our audit committees
responsibilities will include:
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|
appointing, approving the compensation of, and assessing the
independence of our independent registered public accounting
firm; |
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|
overseeing the work of our independent registered public
accounting firm, including through the receipt and consideration
of certain reports from our independent registered public
accounting firm; |
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reviewing and discussing with management and the independent
registered public accounting firm our annual and quarterly
financial statements and related disclosures; |
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monitoring our internal control over financial reporting,
disclosure controls and procedures and code of business conduct
and ethics; |
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establishing policies regarding hiring employees from our
independent registered public accounting firm and procedures for
the receipt and retention of accounting related complaints and
concerns; |
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meeting independently with our independent registered public
accounting firm and management; and |
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preparing the audit committee report required by Securities and
Exchange Commission rules. |
All audit and non-audit services to be provided to us by our
independent registered public accounting firm must be approved
in advance by our audit committee.
We are seeking to retain a person who will qualify as our audit
committee financial expert. We believe that the composition of
our audit committee will meet the requirements for independence
under the current Nasdaq National Market and Securities and
Exchange Commission rules and regulations.
Mr. Neff and Drs. Topper and Weinhoff are the members
of our compensation committee. Mr. Neff is the chair of the
committee. Our compensation committee assists our board of
directors in the discharge of its responsibilities relating to
the compensation of our executive officers.
87
Our compensation committees responsibilities include:
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reviewing and approving, or making recommendations to our board
of directors with respect to, the compensation of our chief
executive officer and our other executive officers; |
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overseeing the evaluation of performance of our senior
executives; |
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overseeing and administering, and making recommendations to our
board of directors with respect to, our cash and equity
incentive plans; |
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calculating potential executive and succession plans; and |
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reviewing and approving non-routine employment agreements,
severance agreements and change in control agreements. |
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Nominating and Corporate Governance Committee |
Messrs. Barkas, Raab and Hayden are the members of our
nominating and corporate governance committee. Mr. Barkas
chairs the committee.
Our nominating and corporate governance committees
responsibilities include:
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recommending to our board of directors the persons to be
nominated for election as directors and to each of the board of
directors committees; |
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conducting searches for appropriate directors; |
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reviewing the size, composition and structure of our board of
directors; |
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developing and recommending to our board of directors corporate
governance principles; |
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overseeing a periodic self-evaluation of our board of directors
and any board committees; and |
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overseeing compensation and benefits for directors and board
committee members. |
Compensation Committee Interlocks and Insider
Participation
None of our executive officers serves as a member of the board
of directors or compensation committee, or other committee
serving an equivalent function, of any entity that has one or
more of its executive officers serving as a member of our board
of directors or our compensation committee. None of the members
of our compensation committee has ever been our employee.
Director Compensation
Our board of directors will consider and intends to implement a
compensation program pursuant to which we will pay each of our
non-employee directors appropriate fees, whether in the form of
cash compensation or equity or both, in support of our efforts
to attract and retain qualified board members. We reimburse each
non-employee member of our board of directors for
out-of-pocket expenses
incurred in connection with attending our board and committee
meetings.
Executive Compensation
The following persons currently constitute our five highest paid
executive officers:
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|
Name |
|
Position |
|
Base Salary | |
|
|
|
|
| |
John F. Crowley
|
|
President and Chief Executive Officer |
|
$ |
400,000 |
|
Matthew R. Patterson
|
|
Chief Business Officer |
|
$ |
280,000 |
|
David J. Lockhart, Ph.D.
|
|
Chief Scientific Officer |
|
$ |
280,000 |
|
Pedro Huertas, M.D., Ph.D.
|
|
Chief Strategic Officer |
|
$ |
281,875 |
|
David Palling, Ph.D.
|
|
Senior Vice President, Drug Development |
|
$ |
236,250 |
|
88
The following summary compensation table sets forth the
compensation paid or accrued for the year ended
December 31, 2005 to our Chief Executive Officer and the
four other highest paid executive officers serving as of
December 31, 2005 and whose total annual compensation
exceeded $100,000 for the year ended December 31, 2005. We
use the term named executive officers to refer to
these people later in this prospectus.
Summary Compensation Table
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Long-Term | |
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|
|
Compensation | |
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|
|
|
|
|
|
|
|
|
Awards | |
|
|
|
|
|
|
|
|
|
|
| |
|
|
|
|
|
|
|
|
|
|
Shares | |
|
|
|
|
|
|
|
|
|
|
Underlying | |
|
All Other | |
Name and Principal Position |
|
Year | |
|
Salary | |
|
Bonus | |
|
Options | |
|
Compensation | |
|
|
| |
|
| |
|
| |
|
| |
|
| |
John F. Crowley
|
|
|
2005 |
|
|
$ |
384,872 |
|
|
$ |
133,334 |
|
|
|
2,998,773 |
|
|
$ |
261,000(1 |
) |
|
President and Chief Executive Officer |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
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|
Matthew R. Patterson
|
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|
2005 |
|
|
|
250,962 |
|
|
|
62,500 |
|
|
|
275,000 |
|
|
|
|
|
|
Chief Business Officer |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
David Palling, Ph.D.
|
|
|
2005 |
|
|
|
225,866 |
|
|
|
56,250 |
|
|
|
225,000 |
|
|
|
|
|
|
Senior Vice President,
Drug Development |
|
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|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
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|
Pedro Huertas, M.D., Ph.D.
|
|
|
2005 |
|
|
|
121,629 |
|
|
|
59,375 |
|
|
|
894,101 |
|
|
|
16,929(2 |
) |
|
Chief Strategic Officer |
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|
|
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|
|
|
|
|
|
|
|
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|
|
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Gregory P. Licholai, M.D.
|
|
|
2005 |
|
|
|
215,827 |
|
|
|
68,000 |
|
|
|
803,417 |
|
|
|
|
|
|
Vice President, Medical
Affairs |
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|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
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|
Norman Hardman,
Ph.D.(3)
|
|
|
2005 |
|
|
|
322,355 |
|
|
|
|
|
|
|
|
|
|
|
|
|
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|
(1) |
Paid in connection with executive medical expense reimbursement. |
|
(2) |
Paid in connection with relocation expense reimbursement,
including a related tax
gross-up payment. |
|
(3) |
Dr. Hardman ceased to be our chief executive officer in
January 2005, and ceased being one of our employees in May 2005. |
Stock Option Grants
The following table contains information regarding grants of
stock options to purchase shares of our common stock made to our
named executive officers during the year ended December 31,
2005.
Amounts in the following table represent potential realizable
gains that could be obtained for the options if exercised at the
end of the option term. The 5% and 10% assumed annual rates of
compounded stock price appreciation are calculated based on the
requirements of the Securities and Exchange Commission, and do
not represent an estimate or projection of our future common
stock prices. These amounts represent certain assumed rates of
appreciation in the value of our common stock from the fair
market value on the date of grant. Actual gains, if any, on
stock option exercises depend on the future performance of the
common stock and overall stock market conditions. The amounts
reflected in the following table may not necessarily be obtained.
89
Option Grants in Last Fiscal Year
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Potential | |
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Realizable | |
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|
Value of | |
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|
Individual Grants | |
|
Assumed | |
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| |
|
Annual Rates | |
|
|
Number of | |
|
|
|
of Stock Price | |
|
|
Securities | |
|
Percent of | |
|
|
|
Appreciation for | |
|
|
Underlying | |
|
Total Options | |
|
|
|
Option Term(3) | |
|
|
Options | |
|
Granted to | |
|
Exercise | |
|
|
|
| |
|
|
Granted | |
|
Employees in | |
|
Price Per | |
|
Expiration | |
|
5% | |
|
10% | |
Name |
|
(#) | |
|
Fiscal Year(1) | |
|
Share(2) | |
|
Date | |
|
($) | |
|
($) | |
|
|
| |
|
| |
|
| |
|
| |
|
| |
|
| |
John F. Crowley
|
|
|
2,248,773 |
|
|
|
32.6 |
% |
|
$ |
.085 |
|
|
|
1/6/2015 |
|
|
|
|
|
|
|
|
|
|
|
|
750,000 |
|
|
|
10.9 |
% |
|
|
.71 |
|
|
|
10/20/2015 |
|
|
|
|
|
|
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|
Matthew R. Patterson
|
|
|
275,000 |
|
|
|
4.0 |
% |
|
|
.71 |
|
|
|
10/20/2015 |
|
|
|
|
|
|
|
|
|
David Palling, Ph.D.
|
|
|
225,000 |
|
|
|
3.3 |
% |
|
|
.71 |
|
|
|
10/20/2015 |
|
|
|
|
|
|
|
|
|
Pedro Huertas, M.D., Ph.D.
|
|
|
724,101 |
|
|
|
10.5 |
% |
|
|
.085 |
|
|
|
6/9/2015 |
|
|
|
|
|
|
|
|
|
|
|
|
20,000 |
|
|
|
0.3 |
% |
|
|
.085 |
|
|
|
6/9/2015 |
|
|
|
|
|
|
|
|
|
|
|
|
150,000 |
|
|
|
2.2 |
% |
|
|
.71 |
|
|
|
10/20/2015 |
|
|
|
|
|
|
|
|
|
Gregory P. Licholai, M.D.
|
|
|
603,417 |
|
|
|
8.7 |
% |
|
|
.085 |
|
|
|
1/3/2015 |
|
|
|
|
|
|
|
|
|
|
|
|
200,000 |
|
|
|
2.9 |
% |
|
|
.71 |
|
|
|
10/20/2015 |
|
|
|
|
|
|
|
|
|
Norman
Hardman, Ph.D.(4)
|
|
|
40,000 |
|
|
|
|
|
|
|
.085 |
|
|
|
6/9/2015 |
|
|
|
|
|
|
|
|
|
|
|
(1) |
The figures representing percentages of total options granted to
employees in the last fiscal year are based on a total of
6,904,785 option shares granted to our employees during fiscal
year 2005. |
(2) |
The exercise price of each option granted was equal to the fair
market value of our common stock as valued by our board of
directors on the date of grant. The exercise price may be paid
in cash, cash equivalents, or in shares of our common stock. |
(3) |
The dollar amounts under these columns are the result of
calculations at rates set by the Securities and Exchange
Commission and, therefore, are not intended to forecast possible
future appreciation, if any, in the price of the underlying
common stock. The potential realizable values are calculated
using the assumed initial public offering price of
$ per share
and assuming that the market price appreciates from this price
at the indicated rate for the entire term of each option and
that each option is exercised and sold on the last day of its
term at the assumed appreciated price. |
(4) |
Dr. Hardman ceased to be our chief executive officer in
January 2005, ceased being one of our employees in May 2005, and
in 2005 received an option to purchase 40,000 shares of
common stock in connection with his execution of a scientific
advisory board agreement with us after he had left our employ. |
90
Option Exercises and Year-End Option Values
The following table provides information about the number of
shares issued upon option exercises by our named executive
officers during the year ended December 31, 2005, and the
value realized by our named executive officers. The table also
provides information about the number and value of shares
underlying options held by our named executive officers at
December 31, 2005. There was no public trading market for
our common stock as of December 31, 2005. Accordingly, as
permitted by the rules of the Securities and Exchange
Commission, we have calculated the value of unexercised
in-the-money options at
fiscal year-end assuming that the fair market value of our
common stock as of December 31, 2005 was equal to the
assumed initial public offering price of
$ per
share, less the aggregate exercise price.
Aggregated Option Exercises in Last Fiscal Year and
Fiscal Year-End Option Values
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|
Number of Securities | |
|
|
|
|
|
|
|
|
Underlying Unexercised | |
|
Value of Unexercised | |
|
|
|
|
|
|
Options at | |
|
In-the-Money Options at | |
|
|
Shares | |
|
|
|
December 31, 2005 | |
|
December 31, 2005 | |
|
|
Acquired on | |
|
Value | |
|
| |
|
| |
Name |
|
Exercise (#) | |
|
Realized | |
|
Exercisable | |
|
Unexercisable | |
|
Exercisable | |
|
Unexercisable | |
|
|
| |
|
| |
|
| |
|
| |
|
| |
|
| |
John F. Crowley
|
|
|
41,224 |
|
|
|
|
|
|
|
13,736 |
|
|
|
3,108,709 |
|
|
|
|
|
|
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|
|
Matthew R. Patterson
|
|
|
|
|
|
|
|
|
|
|
181,025 |
|
|
|
818,076 |
|
|
|
|
|
|
|
|
|
David Palling, Ph.D.
|
|
|
|
|
|
|
|
|
|
|
323,409 |
|
|
|
525,008 |
|
|
|
|
|
|
|
|
|
Pedro Huertas, M.D., Ph.D.
|
|
|
20,000 |
|
|
|
|
|
|
|
|
|
|
|
874,101 |
|
|
|
|
|
|
|
|
|
Gregory P. Licholai, M.D.
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
803,417 |
|
|
|
|
|
|
|
|
|
Norman
Hardman, Ph.D.(1)
|
|
|
616,367 |
|
|
|
|
|
|
|
9,996 |
|
|
|
30,004 |
|
|
|
|
|
|
|
|
|
|
|
(1) |
Dr. Hardman ceased to be our chief executive officer in
January 2005, and ceased being one of our employees in May 2005. |
Employment Agreements
John F. Crowley. Pursuant to an amended and
restated employment agreement dated as of April 28, 2006,
we employ Mr. Crowley as our president and chief executive
officer. Under this agreement, Mr. Crowley is entitled to
an annual base salary of $400,000. Adjustments to his base
salary are in the discretion of our board of directors and we
have agreed not to reduce his base salary below $400,000. The
agreement provides that Mr. Crowley is eligible to receive
a cash bonus of up to 50% of his base salary if performance
criteria are met for the year in which the bonus is to be paid.
The agreement provides that Mr. Crowley is eligible to
participate in any executive bonus plans established by the
board from time to time. We have agreed to secure and maintain
an executive medical reimbursement contract with a named
insurance company covering Mr. Crowley, his spouse and his
dependents. We have also agreed that we shall reimburse
Mr. Crowley up to $220,000 for any medical expenses
incurred by Mr. Crowley, his spouse or his dependent
children, if the amount of those expenses are not covered by the
executive medical reimbursement contract or our medical or
health insurance policies (and such amount shall be grossed up
for any federal and state income tax incurred as a consequence
of our reimbursement of such expenses and the grossing up
thereof).
The agreement will continue for successive one-year terms until
either Mr. Crowley or we provide written notice of
termination to the other in accordance with the terms of the
agreement. Upon the termination of his employment by us other
than for cause, or if we decide not to extend
Mr. Crowleys agreement at the end of any term, or
termination by him for good reason, Mr. Crowley has the
right to receive (i) a severance payment in an amount equal
to 18 times his monthly base salary then in effect, payable in
accordance with our regular payroll practices, (ii) an
additional payment equal to 150% of the target bonus for the
year in which the termination occurs, and
(iii) continuation of benefits for a comparable period as a
result of any such termination. Further, the vesting of all
options then held by
91
Mr. Crowley shall accelerate by one year. Mr. Crowley
is not entitled to severance payments if we terminate him for
cause or if he resigns without good reason. Mr. Crowley is
bound by non-disclosure, inventions and non-competition
covenants that prohibit him from competing with us during the
term of his employment and for one year after termination of
employment.
If Mr. Crowley resigns for good reason, we or our successor
terminate him without cause, or we decide not to extend his
employment agreement at the end of any term, in each case within
3 months prior to, or 12 months following a change of
control, then Mr. Crowley has the right to receive a
severance payment in an amount equal to twice his monthly base
salary then in effect, payable over 24 months in accordance
with our regular payroll schedule, as well as an additional
payment equal to 200% of the target bonus for the year in which
the termination occurs. In addition, Mr. Crowley is
entitled to the continuation of benefits for a comparable period
as a result of any such termination. Further, the vesting of all
options then held by him shall accelerate in full, and all
repurchase rights that we may have as to any of his stock will
automatically lapse.
Other Executive Officers. We have entered into
employment agreements with the following executive officers,
which set forth the officers position, duties, base salary
and benefits, among other things: Matthew R. Patterson, David
Lockhart, Ph.D., Karin Ludwig, M.D., Mark Simon, David
Palling, Ph.D., Pedro Huertas, M.D.,
Ph.D., Gregory P. Licholai, M.D., S. Nicole Schaeffer
and Douglas Branch.
Our executive employment agreements with Drs. Lockhart,
Ludwig and Huertas and Messrs. Branch and Simon each
provide that the executive shall be eligible to receive a bonus
of up to 25% of base salary annually, if in the judgment of the
compensation committee the qualifying criteria established by
the committee for payment of a bonus have been met. Our
executive employment agreement with Dr. Palling and
Ms. Schaeffer provides that the executive shall be eligible
to receive a bonus of up to 20% of base salary annually, if in
the judgment of the compensation committee the qualifying
criteria established by the committee for payment of a bonus
have been met. Mr. Pattersons executive employment
agreement provides that he shall be eligible to receive a bonus
of up to $50,000 annually, if in the judgment of the
compensation committee the qualifying criteria established by
the committee for payment of a bonus have been met.
Dr. Licholais executive employment agreement provides
that he shall be eligible to receive a bonus of up to $43,000
annually, if in the judgment of the compensation committee the
qualifying criteria established by the committee for payment of
a bonus have been met. Our executive employment agreements with
Drs. Lockhart, Ludwig and Huertas and Mr. Patterson
provide for an initial term of two years, and will continue
thereafter for successive two-year periods until we provide the
executive with written notice of the end of the agreement in
accordance with its terms. Our executive employment agreements
with Dr. Palling, Dr. Licholai, Ms. Schaeffer and
Messrs. Branch and Simon have no term and are at
will.
If any of Drs. Lockhart, Ludwig and Huertas or
Messrs. Patterson or Simon is terminated without cause,
then we will be obligated to pay that executive six months of
base salary following that termination plus an amount equal to
any bonus paid to such executive in the previous year. In
addition, the vesting on options then held by them will
automatically accelerate by six months. If any of
Dr. Palling, Dr. Licholai, Ms. Schaeffer or
Mr. Branch is terminated without cause, we will be
obligated to pay that executive six months of base salary
following termination.
The employment agreements with Mr. Patterson,
Mr. Branch, Mr. Simon and Drs. Lockhart, Ludwig
and Huertas, as well as severance agreements with
Dr. Palling, Dr. Licholai, and Ms. Schaeffer,
provide for severance benefits for those executives in the event
we or our successor terminate such executives employment
other than for cause within six months following certain
corporate changes or if, following those changes, the executive
resigns for good reason. In either case, the executive has the
right to receive:
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a lump-sum severance payment in an amount equal to 12 times
his or her monthly base salary in effect as of the date of the
corporate change; |
92
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payment of a bonus equal to the bonus earned in the preceding
year; and |
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any outstanding unvested stock options held by the executive
will fully vest. |
Each executive is bound by non-disclosure, inventions transfer,
non-solicitation and non-competition covenants that prohibit the
executive from competing with us during the term of his or her
employment and for 12 months after termination of
employment.
Stock Option and Other Compensation Plans
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2002 Equity Incentive Plan |
Our 2002 equity incentive plan, as amended, was adopted by our
board of directors and approved by our stockholders. The plan
provides for the grant of incentive and nonstatutory stock
options to purchase shares of our common stock, and restricted
and other stock awards, in each case to our employees, directors
and consultants.
In accordance with the terms of the 2002 equity incentive plan,
our board of directors or one or more committees appointed by
the board of directors administers the plan.
Under our 2002 equity incentive plan, if a merger or other
reorganization event occurs, the board of directors may either
(i) make appropriate provision for the protection of any
outstanding options by substitution on an equitable basis of
appropriate stock of ours or securities of the merged,
consolidated or otherwise reorganized corporation which are
issuable in connection therewith, subject to certain conditions,
or (ii) provide that all unexercised options must be
exercised or they will be terminated.
As of May 3, 2006, there were options to purchase
13,552,120 shares of common stock outstanding under the
2002 equity incentive plan. After the effective date of this
offering, we will grant no further stock options or other equity
incentive awards under the 2002 equity incentive plan.
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2006 Equity Incentive Plan |
In May 2006, our board of directors and stockholders approved
our 2006 equity incentive plan, to become effective on the
closing of this offering. The 2006 equity incentive plan
provides for the grant of incentive stock options, within the
meaning of Section 422 of the Internal Revenue Code, to
employees, and non-qualified stock options and restricted and
other stock awards to our employees, directors, and consultants.
The aggregate number of shares of our common stock that may be
issued under the 2006 equity incentive plan
is .
The aggregate number of shares of common stock that may be
granted in any calendar year to any one person pursuant to the
2006 equity incentive plan may not exceed 50% of the aggregate
number shares of our common stock that may be issued pursuant to
the 2006 equity incentive plan.
The 2006 equity incentive plan will be administered by the
compensation committee of our board of directors. Subject to the
provisions of the 2006 equity incentive plan, the compensation
committee has been granted the discretion to determine when
awards are made, which directors, employees or consultants
receive awards, whether an award will be in the form of an
incentive stock option, a nonqualified stock option or stock
(with or without restrictions), the number of shares subject to
each award, and all other relevant terms of the award, including
vesting and acceleration of vesting, if any. The compensation
committee also has been granted broad discretion to construe and
interpret the 2006 equity incentive plan and adopt rules and
regulations thereunder. Generally, options granted under the
2006 equity incentive plan are expected to vest over a
four-year period from
the date of grant in the case of employees, and over a two-year
period from the date of grant for consultants.
Our board of directors may amend, modify, or terminate our 2006
equity incentive plan at any time, subject to applicable rules
and law and the rights of holders of outstanding awards. Our
2006 equity
93
incentive plan will automatically terminate in May 2016 unless
our board of directors terminates it prior to that time.
2006 Employee Stock Purchase Plan
In May 2006, our board of directors and stockholders approved
our 2006 employee stock purchase plan, to become effective upon
the closing of this offering. The 2006 employee stock purchase
plan authorizes the issuance of up to a total
of shares
of our common stock to eligible employees. The 2006 employee
stock purchase plan shall terminate in May 2011.
The 2006 employee stock purchase plan, which is intended to
qualify under Section 423 of the Internal Revenue Code,
will be implemented by a series of six-month offering periods.
New offering periods are expected to commence on January 1 or
July 1 of each year and end on the next following
June 30 or December 31, respectively. Each offering
period will generally consist of a consecutive six-month
purchase period, and at the end of each six-month period an
automatic purchase will be made for participants. The 2006
employee stock purchase plan will be administered by the board
of directors or by a committee appointed by the board. Employees
are eligible to participate if we employ them for at least
20 hours per week and more than five months per year.
Eligible employees may purchase common stock through payroll
deductions only after the effectiveness of an appropriate
registration statement, which in any event may not exceed 15% of
an employees compensation, at a price equal to the lower
of 85% of the fair market value of the common stock at the
beginning of each offering period or at the end of each purchase
period.
Under the 2006 employee stock purchase plan, no employee shall
be granted an option under the plan if immediately after the
grant the employee would own stock, including any outstanding
options to purchase stock, equaling 5% or more of the total
voting power or value of all classes of our stock. In addition,
the 2006 employee stock purchase plan provides that no employee
shall be granted an option under the 2006 employee stock
purchase plan if the option would permit the employee to
purchase stock under all of our employee stock purchase plans in
an amount that exceeds $25,000 of the fair market value of such
stock for each calendar year in which the option is outstanding
at any time, and that no employee may purchase more
than shares
of common stock under the plan in any one purchase period. The
board of directors may, at its discretion, prior to the
beginning of an offering period, subject the shares acquired (or
to be acquired) by employees for such offering period to certain
transfer restrictions.
In the event of a merger, consolidation, or other acquisition
event resulting in any change of control of us, each right to
purchase stock under the 2006 employee stock purchase plan will
be assumed, or an equivalent right will be substituted by, the
successor corporation. Any ongoing offering period, however,
will be shortened so that employees rights to purchase
stock under the 2006 employee stock purchase plan are exercised
prior to the transaction, unless the employee has withdrawn, in
the event that the successor corporation refuses to assume each
purchase right or to substitute an equivalent right. The board
of directors has the power to amend or terminate the 2006
employee stock purchase plan and to change or terminate offering
periods as long as any action does not adversely affect any
outstanding rights to purchase stock. Our board of directors may
amend or terminate the 2006 employee stock purchase plan or an
offering period even if it would adversely affect outstanding
options in order to avoid our incurring adverse accounting
charges.
401(k) plan
We have established a 401(k) plan to allow our employees to save
on a tax-favorable basis for their retirements. We have not
matched contributions made by employees pursuant to the plan.
94
Limitation of Liability and Indemnification of Officers and
Directors
Our certificate of incorporation that will be in effect upon the
closing of this offering limits the personal liability of
directors for breach of fiduciary duty to the maximum extent
permitted by the Delaware General Corporation Law. Our
certificate of incorporation provides that no director will have
personal liability to us or to our stockholders for monetary
damages for breach of fiduciary duty or other duty as a
director. However, these provisions do not eliminate or limit
the liability of any of our directors:
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for any breach of their duty of loyalty to us or our
stockholders; |
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for acts or omissions not in good faith or which involve
intentional misconduct or a knowing violation of law; |
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for voting or assenting to unlawful payments of dividends or
other distributions; or |
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for any transaction from which the director derived an improper
personal benefit. |
Any amendment to or repeal of these provisions will not
eliminate or reduce the effect of these provisions in respect of
any act or failure to act, or any cause of action, suit or claim
that would accrue or arise prior to any amendment or repeal or
adoption of an inconsistent provision. If the Delaware General
Corporation Law is amended to provide for further limitations on
the personal liability of directors of corporations, then the
personal liability of our directors will be further limited in
accordance with the Delaware General Corporation Law.
In addition, our certificate of incorporation provides that we
must indemnify our directors and officers and we must advance
expenses, including attorneys fees, to our directors and
officers in connection with legal proceedings, subject to very
limited exceptions.
We have entered into, and intend to continue to enter into,
separate indemnification agreements with each of our officers
and directors. These agreements, among other things, require us
to indemnify our officers and directors for certain expenses,
including attorneys fees, judgments, fines and settlement
amounts incurred by an officer or director in any action or
proceeding arising out of their services as one of our officers
and directors, or any of our subsidiaries or any other company
or enterprise to which the person provides services at our
request, to the fullest extent permitted by Delaware law. We
will not indemnify an officer director, however, unless he or
she acted in good faith, reasonably believed his or her conduct
was in, and not opposed, to our best interests, and, with
respect to any criminal action or proceeding, had no reason to
believe his or her conduct was unlawful.
95
CERTAIN RELATIONSHIPS AND RELATED PARTY TRANSACTIONS
Since January 1, 2003, we have engaged in the following
transactions with our directors, executive officers and holders
of more than 5% of our voting securities on an as converted to
common stock basis, and affiliates of our directors, executive
officers and holders of more than 5% of our voting securities.
The following related party transactions are in addition to the
compensation agreements and other arrangements we have made
which are described as required in Management. We
believe that all of these transactions were on terms as
favorable as could have been obtained from unrelated third
parties.
Private Placement of Securities
In May 2004 and April 2005, we issued an aggregate of
36,470,591 shares of our series B redeemable
convertible preferred stock at a price of $0.85 per share,
along with warrants entitling the holders to purchase an
aggregate of 555,003 shares of our series B redeemable
convertible preferred stock at a price of $0.85 per share
at any time before May 4, 2014, for total cash proceeds to
us of approximately $31.0 million before transaction
expenses.
In August 2005 and April 2006, we issued an aggregate of
43,650,262 shares of our series C redeemable
convertible preferred stock at a price of approximately
$1.26 per share for total cash proceeds to us of
approximately $55.0 million before transaction expenses.
The following table sets forth the number of shares of
series B redeemable convertible preferred stock and
series C redeemable convertible preferred stock sold to our
5% stockholders and directors and their affiliates in these
financings. The shares of series B redeemable convertible
preferred stock and series C redeemable convertible
preferred stock referred to in the table will convert
automatically on a one-for-one basis into shares of our common
stock upon the closing of this offering.
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Number of Shares of | |
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Number of Shares of | |
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Series B Redeemable | |
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Series C Redeemable | |
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Convertible | |
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Convertible | |
Name |
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Preferred Stock | |
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Preferred Stock | |
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| |
Entities affiliated with Prospect Venture Partners
(1)
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7,564,370 |
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7,621,664 |
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Entities affiliated with New Enterprise Associates
(2)
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7,564,369 |
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7,621,664 |
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Entities affiliated with Frazier Healthcare Ventures
(3)
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7,564,368 |
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7,621,664 |
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Entities affiliated with Canaan
Partners(4)
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7,094,582 |
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6,806,250 |
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Entities affiliated with CHL Medical
Partners(5)
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5,971,870 |
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3,968,254 |
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Entities affiliated with Quaker
Bioventures(6)
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7,936,506 |
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Total
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35,759,559 |
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41,576,002 |
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(1) |
Includes 113,467 shares of series B redeemable
convertible preferred stock (including the automatic exercise of
outstanding warrants to purchase 1,701 shares of
series B redeemable convertible preferred stock) and
114,326 shares of series C redeemable convertible
preferred stock, in each case issued to Prospect
Associates II, L.P., and 7,450,903 shares of
series B redeemable convertible preferred stock (including
the automatic exercise of outstanding warrants to
purchase 111,687 shares of series B redeemable
convertible preferred stock) and 7,507,338 shares of
series C redeemable convertible preferred stock issued to
Prospect Venture Partners II, L.P. Dr. Barkas, one of
our directors, is a Managing Member of the General Partner of
both Prospect Venture Partners II, L.P., and Prospect
Associates II, L.P. |
(2) |
Includes 20,304 shares of series B redeemable
convertible preferred stock issued to NEA Ventures 2004, Limited
Partnership (including the automatic exercise of outstanding
warrants to purchase 304 shares of series B
redeemable convertible preferred stock), and
7,544,065 shares of series B redeemable convertible
preferred stock (including the automatic exercise of outstanding
warrants to purchase 113,083 shares of series B
redeemable convertible preferred stock) and
7,621,664 shares of series C redeemable convertible
preferred stock issued to New Enterprise Associates 11,
L.P. Mr. Raab, one of our directors, is a partner of New
Enterprise Associates. |
(3) |
Includes 38,205 shares of series B redeemable
convertible preferred stock (including the automatic exercise of
outstanding warrants to purchase 573 shares of
series B redeemable convertible preferred stock) and
38,494 shares of series C redeemable convertible
preferred stock issued to Frazier Affiliates IV, L.P., and
7,526,163 shares of series B redeemable convertible
preferred stock (including the automatic exercise of outstanding
warrants to purchase 112,815 shares of series B
redeemable convertible preferred stock) and
7,583,170 shares of series C redeemable convertible
preferred stock issued to Frazier Healthcare IV, L.P.
Dr. Topper, one of our directors, holds the title of
General Partner with Frazier Healthcare Ventures. |
96
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(4) |
Includes 6,839,178 shares of series B redeemable
convertible preferred stock (including the automatic exercise of
outstanding warrants to purchase 102,518 shares of
series B redeemable convertible preferred stock) and
6,561,226 shares of series C redeemable convertible
preferred stock issued to Canaan Equity III, L.P., and
255,404 shares of series B redeemable convertible
preferred stock (including the automatic exercise of outstanding
warrants to purchase 3,828 shares of series B
redeemable convertible preferred stock) and 245,024 shares
of series C redeemable convertible preferred stock issued
to Canaan Equity III Entrepreneurs, LLC. Dr. Bloch,
one of our directors, is a Member of Canaan Equity
Partners III, LLC, the sole general partner of Canaan
Equity III, L.P. and the sole manager of Canaan Equity III
Entrepreneurs, LLC. |
(5) |
Includes 5,520,337 shares of series B redeemable
convertible preferred stock and 3,717,758 shares of
series C redeemable convertible preferred stock issued to
CHL Medical Partners II, L.P. and 451,533 shares of
series B redeemable convertible preferred stock and
250,496 shares of series C redeemable convertible
preferred stock issued to CHL Medical Partners II Side
Fund, L.P. Dr. Weinhoff, one of our directors, is a Member
of the General Partner of both CHL Medical Partners II,
L.P. and CHL Medical Partners II Side Fund, L.P. |
(6) |
Includes 5,952,380 shares of series C redeemable
convertible preferred stock issued to Quaker Bioventures, L.P.
and 1,984,126 shares of series C redeemable
convertible preferred stock issued to Garden State Life Sciences
Venture Fund, L.P. Mr. Neff, one of our directors, is a
member of the general partner of the general partner of both
Quaker Bioventures, L.P. and Garden State Life Sciences Venture
Fund, L.P. |
Bridge Financings
In April 2003, June 2003, August 2003, November 2003, February
2004 and April 2004, we issued (inclusive of certain warrants to
purchase common stock which have been exercised) convertible
promissory notes in an aggregate principal amount of
$5.5 million to certain investors.
The notes accrued interest at the prime rate plus
2%. In the event that we completed an equity financing resulting
in gross proceeds to us of at least $12.0 million, the
notes were automatically convertible into shares of the same
class of equity issued in the financing. $5,000,000 of principal
outstanding under the notes converted into shares of our
series B redeemable convertible preferred stock in
connection with our series B redeemable convertible
preferred stock financing in May 2004. The other $500,000 of
principal outstanding under the notes was repaid by the Company
in May 2004.
The following table sets forth the names of holders of more than
5% of our capital stock who participated in these bridge
financings, the principal amount of the notes held in the
aggregate by these holders, and the number of shares of our
series B redeemable convertible preferred stock issued upon
conversion of the notes.
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Shares of | |
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Series B | |
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Redeemable | |
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Convertible | |
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Aggregate Principal | |
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Preferred Stock | |
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Amount of | |
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Issued upon | |
Holders of more than 5% |
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Notes Held | |
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Conversion | |
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CHL Medical Partners II, L.P.
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$ |
5,089,438 |
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5,436,471 |
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CHL Medical Partners Side Fund II, L.P.
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$ |
410,562 |
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445,882 |
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In connection with these bridge financings, we also issued
warrants to the investors that were exercisable in the aggregate
for 999,999 shares of our common stock at an exercise price
of seven and one-half cents ($0.075) per share. The investors
exercised all of these common stock warrants in August 2005.
Certain Relationships
Pursuant to a second amended and restated investor rights
agreement among holders of our redeemable convertible preferred
stock and us, we granted registration rights to all such holders
and to the holder of a warrant to
purchase 40,000 shares of our common stock. Entities
affiliated with Prospect Venture Partners II, L.P., New
Enterprise Associates, Frazier Healthcare Ventures, Canaan
Equity, Quaker BioVentures and CHL Medical Partners, each
holders of 5% or more of our voting securities, and
97
their affiliates are parties to this investor rights agreement.
See Description of Capital Stock Registration
Rights.
Please see Management Director Compensation
for a discussion of options granted and other compensation to
our non-employee directors.
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Executive Compensation and Employment Agreements |
Please see Management Executive Compensation
and Management Stock Options for
additional information on compensation of our executive
officers. Information regarding employment agreements with our
executive officers is set forth under Management
Employment Agreements.
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Indemnification Agreements |
We have entered into indemnification agreements with each of our
officers and directors. These agreements, among other things,
require us to indemnify each officer and director to the fullest
extent permitted by Delaware law, including indemnification of
expenses such as attorneys fees, judgments, fines and
settlement amounts incurred by the officer or director in any
action or proceeding, including any action or proceeding by or
in right of us, arising out of the persons services as an
officer or director. We will not indemnify an officer or
director, however, unless he or she acted in good faith,
reasonably believed his or her conduct was in, and not opposed,
to our best interests and, with respect to any criminal action
or proceeding, had no reason to believe his or her conduct was
unlawful.
98
PRINCIPAL STOCKHOLDERS
The following table sets forth information with respect to the
beneficial ownership of our common stock, as of May 3,
2006, by:
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each of our directors; |
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each of our executive officers; |
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each person, or group of affiliated persons, who is known by us
to beneficially own more than 5% of our common stock; and |
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all of our directors and executive officers as a group. |
The column entitled Percentage of Shares Beneficially
Owned Before Offering is based on a total of
89,516,214 shares of our common stock outstanding on
May 3, 2006, assuming the automatic exercise of all
outstanding warrants to purchase 465,486 shares of
series B redeemable convertible preferred stock and the
conversion of all outstanding shares of our redeemable
convertible preferred stock into 84,009,910 shares of our
common stock upon the closing of this offering. The column
entitled Percentage of Shares Beneficially Owned
After Offering is based
on shares
of common stock to be outstanding after this offering, including
the shares
that we are selling in this offering, but not including any
shares issuable upon exercise of warrants or options outstanding
after this offering.
For purposes of the table below, we deem shares of common stock
subject to options or warrants that are currently exercisable or
exercisable within 60 days of May 3, 2006 to be
outstanding and to be beneficially owned by the person holding
the options or warrants for the purpose of computing the
percentage ownership of that person but we do not treat them as
outstanding for the purpose of computing the percentage
ownership of any other person. Except as otherwise noted, the
persons or entities in this table have sole voting and investing
power with respect to all of the shares of common stock
beneficially owned by them, subject to community property laws,
where applicable. Except as otherwise set forth below, the
street address of the beneficial owner is c/o Amicus
Therapeutics, Inc., 6 Cedar Brook Drive, Cranbury, NJ 08512.
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Percentage of Shares | |
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Beneficially Owned | |
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Number of Shares | |
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Before | |
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After | |
Name and Address of Beneficial Owner |
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Beneficially Owned | |
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Offering | |
|
Offering | |
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5% Stockholders
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Entities affiliated with Prospect Venture Partners II,
L.P.(1)
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15,186,034 |
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17.0 |
% |
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435 Tasso Street, Suite 200
Palo Alto, CA 94301 |
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Entities affiliated with New Enterprise
Associates(2)
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15,186,033 |
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17.0 |
% |
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1119 St. Paul Street
Baltimore, MD 21202 |
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Entities affiliated with Frazier Healthcare Ventures
(3)
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15,186,032 |
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17.0 |
% |
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601 Union, Two Union Square, Suite 3200
Seattle, WA 98101 |
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Entities affiliated with CHL Medical
Partners(4)
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14,273,457 |
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15.9 |
% |
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1055 Washington Boulevard, 6th Floor
Stamford, CT 06901 |
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Entities affiliated with Canaan
Partners(5)
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13,900,832 |
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15.5 |
% |
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105 Rowayton Avenue
Rowayton, CT 06853 |
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|
|
|
|
Entities affiliated with Quaker
Bioventures(6)
|
|
|
7,936,506 |
|
|
|
8.9 |
% |
|
|
|
|
|
Cira Center
2929 Arch Street
Philadelphia, PA 19104-2868 |
|
|
|
|
|
|
|
|
|
|
|
|
99
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Percentage of Shares | |
|
|
|
|
Beneficially Owned | |
|
|
|
|
| |
|
|
Number of Shares | |
|
Before | |
|
After | |
Name and Address of Beneficial Owner |
|
Beneficially Owned | |
|
Offering | |
|
Offering | |
|
|
| |
|
| |
|
| |
Executive Officers and Directors
|
|
|
|
|
|
|
|
|
|
|
|
|
John F.
Crowley(7)
|
|
|
922,302 |
|
|
|
1.0 |
% |
|
|
|
|
David Palling,
Ph.D.(8)
|
|
|
404,206 |
|
|
|
* |
|
|
|
|
|
Matthew R.
Patterson(9)
|
|
|
286,627 |
|
|
|
* |
|
|
|
|
|
Gregory P. Licholai,
M.D.(10)
|
|
|
238,858 |
|
|
|
* |
|
|
|
|
|
Pedro Huertas, M.D.,
Ph.D.(11)
|
|
|
201,025 |
|
|
|
* |
|
|
|
|
|
S. Nicole
Schaeffer(12)
|
|
|
50,783 |
|
|
|
* |
|
|
|
|
|
David Lockhart, Ph.D.
|
|
|
|
|
|
|
|
|
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Karin Ludwig, M.D.
|
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|
|
Mark Simon
|
|
|
|
|
|
|
|
|
|
|
|
|
Douglas
Branch(13)
|
|
|
24,996 |
|
|
|
* |
|
|
|
|
|
Donald J.
Hayden(14)
|
|
|
52,085 |
|
|
|
* |
|
|
|
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|
Alexander E. Barkas,
Ph.D.(15)
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|
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|
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|
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Michael G.
Raab(16)
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James N. Topper, M.D.,
Ph.D.(17)
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Stephen Bloch,
M.D.(18)
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Gregory M Weinhoff,
M.D.(19)
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P. Sherrill
Neff(20)
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|
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|
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|
|
|
|
|
|
All directors and executive officers as a group (16
persons)/(21)
|
|
|
2,180,882 |
|
|
|
2.4 |
% |
|
|
|
|
|
|
|
|
* |
Represents beneficial ownership of less than one percent of our
outstanding common stock. |
|
|
|
|
(1) |
Consists of 14,958,241 shares held of record by Prospect
Venture Partners II, L.P. including 111,687 shares assuming
the exercise of outstanding warrants held by Prospect Venture
Partners II, L.P., and 227,793 shares held of record
by Prospect Associates II, L.P. including 1,701 shares
assuming the exercise of outstanding warrants held by Prospect
Associates II, L.P. Dr. Barkas, a member of our board
of directors and a Managing Member of the General Partner of
both Prospect Venture Partners II, L.P. and Prospect
Associates II, L.P., disclaims beneficial ownership of the
shares held by entities affiliated with Prospect Venture
Partners II, L.P. except, to the extent of any pecuniary
interest therein. |
|
(2) |
Consists of 15,165,729 shares held of record by New
Enterprise Associates 11, Limited Partnership including
113,083 shares assuming the exercise of outstanding
warrants held by New Enterprise Associates 11, Limited
Partnership, and 20,304 shares held of record by NEA
Ventures 2004, Limited Partnership including 304 shares
assuming the exercise of outstanding warrants held by NEA
Ventures 2004, Limited Partnership. Mr. Raab is a partner
of New Enterprise Associates but does not have voting or
dispositive power with respect to the shares held by New
Enterprise Associates 11, Limited Partnership or NEA Ventures
2004, Limited Partnership and disclaims beneficial ownership of
shares held by New Enterprise Associates 11, Limited
Partnership, except to the to the extent of his pecuniary
interest therein. Mr. Raab has no pecuniary interest in the
shares held by NEA Ventures 2004, Limited Partnership. |
|
(3) |
Consists of 15,109,333 shares held of record by Frazier
Healthcare IV, L.P. including 112,815 shares assuming the
exercise of outstanding warrants held by Frazier Healthcare IV,
L.P. and 76,699 shares held of record by Frazier Affiliates
IV, L.P. including 573 shares assuming the exercise of
outstanding warrants held by Frazier Affiliates IV, L.P.
Dr. Topper, a member of our board of directors, holds the
title of General Partner with Frazier Healthcare Ventures. In
that capacity he shares voting and investment power for the
shares held by both Frazier Healthcare IV, L.P. and Frazier
Affiliates IV, L.P. Dr. Topper disclaims beneficial
ownership of the shares held by entities affiliated with Frazier
Healthcare Ventures, except to the extent of any pecuniary
interest therein. |
|
(4) |
Consists of 13,297,885 shares held of record by CHL Medical
Partners II, L.P. and 975,572 shares held of record by
CHL Medical Partners II Side Fund, L.P. Dr. Weinhoff,
a member of our board of directors and a Member of the General
Partner of both CHL Medical Partners II, L.P. and CHL
Medical Partners II Side Fund, L.P., disclaims beneficial
ownership of the shares held by entities affiliated with CHL
Medical Partners, except to the extent of any pecuniary interest
therein. |
|
(5) |
Consists of 13,400,404 shares held of record by Canaan
Equity III, L.P. including 102,518 shares assuming the
exercise of outstanding warrants held by Canaan Equity III,
L.P. and 500,428 shares held of record by Canaan
Equity III Entrepreneurs, LLC including 3,828 shares
assuming the exercise of outstanding warrants held by Canaan
Equity III Entrepreneurs, LLC. Canaan Equity
Partners III, LLC, the sole general partner of Canaan
Equity III, L.P. and sole manager of Canaan Equity III
Entrepreneurs, LLC, has sole voting and disposition power over
these shares. The Managers of Canaan Equity Partners, III, |
100
|
|
|
|
|
LLC are John V. Balen, James C.
Furnival, Stephen L. Green, Deepak Kamra, Gregory Kopchinsly,
Seth A. Rudnick, Guy M. Russo and Eric A. Young. Dr. Bloch,
a member of our board of directors, is a member of Canaan Equity
Partners III, LLC. Dr. Bloch does not have sole or
shared voting or disposition power over these shares.
|
|
(6) |
Consists of 5,952,380 shares
held of record by Quaker Bioventures, L.P. and
1,984,126 shares held of record by Garden State Life
Sciences Venture Fund, L.P. Mr. Neff, a member of our board
of directors and a Member of the General Partner of both Quaker
Bioventures, L.P., and Garden State Life Sciences Ventured Fund,
L.P. disclaims beneficial ownership of the shares held by
entities affiliated with Quaker Bioventures, except to the
extent of any pecuniary interest therein.
|
|
(7) |
Consists of 281,078 shares
issuable upon the exercise of stock options exercisable within
60 days of May 3, 2006, and 641,224 shares held
of record. Includes 100,000 shares held of record by MPAJ,
LLC, for which Mr. Crowley has sole voting and dispositive power.
|
|
(8) |
Consists of 37,711 shares
issuable upon the exercise of stock options exercisable within
60 days of May 3, 2006, and 366,495 shares held
of record.
|
|
(9) |
Consists of 46,627 shares
issuable upon the exercise of stock options exercisable within
60 days of May 3, 2006, and 240,000 shares held
of record.
|
|
|
(10) |
Consists of 37,716 shares issuable upon the exercise of
stock options exercisable within 60 days of May 3,
2006, and 201,142 shares held of record. |
(11) |
Consists of 181,025 shares issuable upon the exercise of
stock options exercisable within 60 days of May 3,
2006, and 20,000 shares held of record. |
(12) |
Consists of 9,522 shares issuable upon the exercise of
stock options exercisable within 60 days of May 3,
2006, and 41,261 shares held of record. |
(13) |
Consists of 24,996 shares issuable upon the exercise of
stock options exercisable within 60 days of May 3,
2006. |
(14) |
Consists of 52,085 shares issuable upon the exercise of
stock options exercisable within 60 days of May 3,
2006. |
(15) |
Dr. Barkas disclaims beneficial ownership of the shares
held by affiliates of Prospect Venture Partners II, L.P. except
to the extent of his pecuniary interest therein. See
footnote 1. |
(16) |
Mr. Raab disclaims beneficial ownership of the shares held
by New Enterprise Associates, except to the extent of his
pecuniary interest therein. See footnote 2. |
(17) |
Dr. Topper disclaims beneficial ownership of the shares
held by Frazier Healthcare, except to the extent of his
pecuniary interest therein. See footnote 3. |
(18) |
Dr. Bloch does not have sole or shared voting or
dispositive power over shares owned by entities affiliated with
Canaan Partners. Dr. Bloch disclaims beneficial ownership
of such shares, except to the extent of his pecuniary interest
therein. See footnote 5. |
(19) |
Dr. Weinhoff disclaims beneficial ownership of the shares
held by CHL Medical Partners, except to the extent of his
pecuniary interest therein. See footnote 4. |
(20) |
Mr. Neff disclaims beneficial ownership of the shares held
by Quaker Bioventures, except to the extent of his pecuniary
interest therein. See footnote 6. |
(21) |
Consists of 670,760 total shares issuable upon the exercise of
stock options exercisable within 60 days of May 3,
2006, and 1,510,122 total shares held of record. |
101
DESCRIPTION OF CAPITAL STOCK
The following description of our capital stock and provisions of
our certificate of incorporation and bylaws are summaries and
are qualified by reference to the certificate of incorporation
and the bylaws that will be in effect upon the closing of this
offering. We have filed copies of forms of these documents with
the Securities and Exchange Commission as exhibits to our
Registration Statement of which this prospectus forms a part.
The description of the capital stock reflects changes to our
capital structure that will occur upon the closing of this
offering.
Upon the closing of this offering, our authorized capital stock
will consist
of shares
of common stock, par value $0.01 per share, and
5,000,000 shares of preferred stock, par value
$0.01 per share, all of which preferred stock will be
undesignated.
As of May 3, 2006, we had issued and outstanding:
|
|
|
|
|
5,507,024 shares of common stock outstanding held by 17
stockholders of record; |
|
|
|
3,333,334 shares of series A redeemable convertible
preferred stock that are convertible into 3,333,334 shares
of common stock; |
|
|
|
36,560,108 shares of series B redeemable convertible
preferred stock that are convertible into 36,560,108 shares
of common stock; and |
|
|
|
43,650,262 shares of series C redeemable convertible
preferred stock that are convertible into 43,650,262 shares
of common stock. |
As of May 3, 2006, we also had outstanding:
|
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|
|
|
|
options to purchase 13,552,120 shares of common stock at a
weighted average exercise price of $0.48 per share; |
|
|
|
|
|
warrants to purchase an aggregate of 465,486 shares of
series B redeemable convertible preferred stock at an
exercise price of $0.85 per share, which warrants are
automatically exercised upon the closing of this
offering; and |
|
|
|
|
a warrant to purchase 40,000 shares of common stock at
an exercise price of $0.75 per share. |
Upon the closing of this offering, all of the outstanding shares
of our redeemable convertible preferred stock will automatically
convert into a total of 84,009,190 shares of our common
stock, assuming the automatic exercise of all outstanding
warrants to purchase 465,486 shares of series B
redeemable convertible preferred stock.
Common Stock
Holders of our common stock are entitled to one vote for each
share held on all matters submitted to a vote of stockholders
and do not have cumulative voting rights. An election of
directors by our stockholders shall be determined by a plurality
of the votes cast by the stockholders entitled to vote on the
election. Holders of common stock are entitled to receive
proportionately any dividends as may be declared by our board of
directors, subject to any preferential dividend rights of any
outstanding preferred stock.
In the event of our liquidation or dissolution, the holders of
common stock are entitled to receive proportionately all assets
available for distribution to stockholders after the payment of
all debts and other liabilities and subject to the prior rights
of any outstanding preferred stock. Holders of common stock have
no preemptive, subscription, redemption or conversion rights.
The rights, preferences and privileges of holders of common
stock are subject to and may be adversely affected by the rights
of the holders of shares of any series of preferred stock that
we may designate and issue in the future.
Preferred Stock
Under the terms of our certificate of incorporation to be
effective at closing, our board of directors is authorized to
issue shares of preferred stock in one or more series without
stockholder approval. Our board of directors has the discretion
to determine the rights, preferences, privileges and
restrictions, including
102
voting rights, dividend rights, conversion rights, redemption
privileges and liquidation preferences, of each series of
preferred stock.
The purpose of authorizing our board of directors to issue
preferred stock and determine its rights and preferences is to
eliminate delays associated with a stockholder vote on specific
issuances. The issuance of preferred stock, while providing
flexibility in connection with possible acquisitions, future
financings and other corporate purposes, could have the effect
of making it more difficult for a third party to acquire, or
could discourage a third party from seeking to acquire, a
majority of our outstanding voting stock. Upon the closing of
this offering, there will be no shares of preferred stock
outstanding, and we have no present plans to issue any shares of
preferred stock.
Warrants
As of the closing of this offering, we have an outstanding
warrant to purchase an aggregate of 40,000 shares of common
stock at an exercise price of $0.75.
Options
As of May 3, 2006, options to purchase
13,552,120 shares of common stock at a weighted average
exercise price of $0.48 per share were outstanding.
Anti-Takeover Effects of Delaware Law and our Corporate
Charter Documents
We are subject to Section 203 of the Delaware General
Corporation Law. Subject to certain exceptions, Section 203
prevents a publicly held Delaware corporation from engaging in a
business combination with any interested
stockholder for three years following the date that the
person became an interested stockholder, unless the interested
stockholder attained such status with the approval of our board
of directors or unless the business combination is approved in a
prescribed manner. A business combination includes,
among other things, a merger or consolidation involving us,
sales of our assets, or other transactions resulting in a
financial benefit to the interested stockholder. In
general, an interested stockholder is any entity or
person beneficially owning, or in the past three years owning,
15% or more of our outstanding voting stock and any entity or
person affiliated with or controlling or controlled by such
entity or person. The restrictions contained in Section 203
are not applicable to any of our existing stockholders that will
own 15% or more of our outstanding voting stock upon the closing
of this offering. This statute could prohibit or delay the
accomplishment of mergers or other takeover or change in control
attempts with respect to us and accordingly, may discourage
attempts to acquire us.
Our certificate of incorporation and our bylaws to be effective
at closing of this offering divide our board of directors into
three classes with staggered three-year terms. In addition, our
certificate of incorporation and our bylaws to be effective upon
the closing of this offering provide that directors may be
removed only for cause and only by the affirmative vote of a
majority of the holders of our shares of capital stock present
in person or by proxy and entitled to vote. Under our
certificate of incorporation and bylaws, any vacancy on our
board of directors, including a vacancy resulting from an
enlargement of our board of directors, may be filled only by
vote of a majority of our directors then in office. Furthermore,
our bylaws provide that the authorized number of directors may
be changed only by the resolution of our board of directors. The
classification of our board of directors and the limitations on
the ability of our stockholders to remove directors, change the
authorized number of directors, and fill vacancies could make it
more difficult for a third party to acquire, or discourage a
third party from seeking to acquire, control of our company.
|
|
|
Stockholder Action; Special Meeting of Stockholders;
Advance Notice Requirements for Stockholder Proposals and
Director Nominations |
Our certificate of incorporation and our bylaws to be effective
at closing of this offering provide that any action required or
permitted to be taken by our stockholders at an annual meeting
or special meeting
103
of stockholders may only be taken if it is properly brought
before such meeting and may not be taken by written action in
lieu of a meeting. Our certificate of incorporation and our
bylaws also provide that, except as otherwise required by law,
special meetings of the stockholders can only be called by our
chairman of the board, our president, or a majority of our board
of directors. In addition, our bylaws establish an advance
notice procedure for stockholder proposals to be brought before
an annual meeting of stockholders, including proposed
nominations of candidates for election to the board of
directors. Stockholders at an annual meeting may only consider
proposals or nominations specified in the notice of meeting or
brought before the meeting by, or by a stockholder of record on
the record date for the meeting, who is entitled to vote at the
meeting and who has delivered timely written notice in proper
form to our secretary of the stockholders intention to
bring such business before the meeting. These provisions could
have the effect of delaying until the next stockholder meeting
stockholder actions that are favored by the holders of a
majority of our outstanding voting securities.
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|
|
Authorized But Unissued Shares |
The authorized but unissued shares of common stock and preferred
stock are available for future issuance without stockholder
approval, subject to any limitations imposed by the Nasdaq
National Market. These additional shares may be utilized for a
variety of corporate acquisitions and employee benefit plans.
The existence of authorized but unissued and unreserved common
stock could make more difficult or discourage an attempt to
obtain control of us by means of a proxy contest, tender offer,
merger, or otherwise.
The Delaware General Corporation Law provides generally that the
affirmative vote of a majority of the shares entitled to vote on
any matter is required to amend a corporations certificate
of incorporation or bylaws, unless a corporations
certificate of incorporation or bylaws, as the case may be,
requires a greater percentage. Our bylaws to be effective at
closing of this offering may be amended or repealed by a
majority vote of our board of directors or the affirmative vote
of the holders of at least 67% of our outstanding voting stock
to amend, repeal or adopt any provisions inconsistent with
provisions described above and contained in our by-laws. In
addition, the affirmative vote of the holders of at least 67% of
our outstanding voting stock is required to amend or repeal or
to adopt any provisions inconsistent with any of the provisions
of our certificate of incorporation described above, or to amend
certain provisions relating to indemnification.
|
|
|
Board Discretion in Considering Certain Offers |
Our certificate of incorporation to be effective at closing of
this offering empowers our board of directors, when considering
a tender offer or merger or acquisition proposal, to take into
account factors in addition to potential economic benefits to
stockholders. Such factors may include (i) comparison of
the proposed consideration to be received by stockholders in
relation to the then-current market price of our capital stock,
our estimated current value in a freely negotiated transaction,
and our estimated future value as an independent entity, and
(ii) the impact of such a transaction on our employees,
suppliers, and customers and its effect on the communities in
which we operate.
Our certificate of incorporation to be effective at closing of
this offering contains certain provisions permitted under the
Delaware General Corporation Law relating to the liability of
directors. These provisions eliminate a directors personal
liability for monetary damages resulting from a breach of
fiduciary duty, except in certain circumstances involving
certain wrongful acts, such as the breach of a directors
duty of loyalty or acts or omissions that involve intentional
misconduct or a knowing violation of law. These provisions do
not limit or eliminate our rights or the rights of any
stockholder to seek non-monetary relief, such as an injunction
or rescission, in the event of a breach of a directors
fiduciary duty. These provisions will not alter a
directors liability under federal securities laws. Our
certificate of incorporation and by-laws to be effective on
closing also contain provisions indemnifying our directors and
104
officers to the fullest extent permitted by the Delaware General
Corporation Law. We believe that these provisions will assist us
in attracting and retaining qualified individuals to serve as
directors.
Registration Rights
Upon the closing of this offering, holders of an aggregate of
84,049,190 shares of our common will have the right to
require us to register these shares under the Securities Act
under specified circumstances.
|
|
|
Demand Registration Rights |
After the closing of this offering and subject to certain
limitations, these stockholders may require on up to two
occasions, and as long as the aggregate price to the public for
the securities to be sold in each instance is $5,000,000 or
more, that we use our best efforts register all or part of their
securities for sale under the Securities Act.
|
|
|
Form S-3
Registration Rights |
If we register any of our common stock on
Form S-3, either
for our own account or for the account of other securityholders,
these stockholders holding registration rights are entitled to
notice of the registration and further entitled to include their
shares of common stock in the registration. This right is
subject to specified limitations, including but not limited to
(i) if the Company has already effected a registration
within 90 days or has effected two or more registration
statements on
Form S-3 within
the preceding 12 month period and (ii) if the
aggregate price to the public for the securities to be sold is
less than $2,500,000. Additionally, if we certify that such
registration would have a materially detrimental effect on any
material corporate event, we may delay the request for up to
three months, but not more than once in any twelve month period.
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|
|
Incidental Registration Rights |
At any time after this offering, if we register any of our
common stock, either for our own account or for the account of
other securityholders, then all holders of registrable
securities are entitled to notice of the registration and to
include their shares of common stock in the registration. In the
case of an underwritten registration, we must use our reasonable
efforts to obtain the permission of the underwriters to the
inclusion of the holders shares in the offering on the
same terms.
With specified exceptions, a holders right to include
shares in a registration is subject to the right of the
underwriters to limit the number of shares included in the
offering. All fees, costs and expenses of any registrations will
generally be paid by us.
Transfer Agent and Registrar
The transfer agent and registrar for our common stock will
be following
the closing of this offering.
Nasdaq National Market
We have applied to have our common stock approved for quotation
on The Nasdaq National Market under the symbol AMTX.
SHARES ELIGIBLE FOR FUTURE SALE
Prior to this offering, there has been no public market for our
common stock and a liquid trading market for our common stock
may not develop or be sustained after this offering. Future
sales of substantial amounts of common stock, including shares
issued upon exercise of outstanding options and warrants or in
the public market after this offering, or the anticipation of
those sales, could adversely affect market prices prevailing
from time to time and could impair our ability to raise capital
through sales of our equity securities.
105
Upon the closing of this offering, we will have
outstanding shares
of common stock, after giving effect to the issuance
of shares
of common stock in this offering and the automatic conversion of
all outstanding shares of our convertible preferred stock, into
an aggregate of 84,009,190 shares of our common stock,
assuming the automatic exercise of all outstanding warrants to
purchase 465,486 shares of series B redeemable
convertible preferred stock, and assuming no exercise of the
underwriters over-allotment option and no exercise of
options or other warrants outstanding as of May 3, 2006.
Of the shares to be outstanding immediately after the closing of
this offering,
the shares
to be sold in this offering will be freely tradable without
restriction under the Securities Act unless purchased by our
affiliates, as that term is defined in Rule 144
under the Securities Act. The remaining 89,516,214 shares
of common stock are restricted securities under
Rule 144. Substantially all of these restricted securities
will be subject to the
180-day
lock-up period
described below.
After the 180-day
lock-up period, these
restricted securities may be sold in the public market only if
registered or if they qualify for an exemption from registration
under Rules 144 or 701 under the Securities Act, which
exemptions are summarized below.
Rule 144
In general, under Rule 144, beginning 90 days after
the date of this prospectus, a person who has beneficially owned
shares of our common stock for at least one year, including the
holding period of any prior owner other than one of our
affiliates, would be entitled to sell within any three-month
period a number of shares that does not exceed the greater of:
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|
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|
|
1% of the number of shares of our common stock then outstanding,
which will equal
approximately shares
immediately after this offering, and |
|
|
|
the average weekly trading volume in our common stock on The
Nasdaq National Market during the four calendar weeks preceding
the date of filing of a Notice of Proposed Sale of Securities
Pursuant to Rule 144 with respect to the sale. |
Sales under Rule 144 are also subject to manner of sale
provisions and notice requirements, and to the availability of
current public information about us.
Upon expiration of the
180-day
lock-up period
described
below, of
shares of our common stock will be eligible for sale under
Rule 144, excluding shares eligible for resale under
Rule 144(k) as described below. We cannot estimate the
number of shares of common stock that our existing stockholders
will elect to sell under Rule 144.
Rule 144(k)
Subject to the lock-up
agreements described below, shares of our common stock eligible
for sale under Rule 144(k) may be sold immediately upon the
closing of this offering. In general, under Rule 144(k), a
person may sell shares of common stock acquired from us
immediately upon the closing of this offering, without regard to
manner of sale, the availability of public information about us
or volume limitations, if:
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|
the person is not our affiliate and has not been our affiliate
at any time during the three months preceding the sale; and |
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|
|
the person has beneficially owned the shares proposed to be sold
for at least two years, including the holding period of any
prior owner other than our affiliates. |
Upon the expiration of the
180-day
lock-up period
described below,
approximately shares
of common stock will be eligible for sale under Rule 144(k).
Rule 701
In general, under Rule 701 of the Securities Act, any of
our employees, consultants or advisors who purchased shares from
us in connection with a qualified compensatory stock plan or
other written agreement is eligible to resell those shares
90 days after the effective date of the offering in
reliance on
106
Rule 144, but without compliance with the various
restrictions, including the holding period, contained in
Rule 144. Subject to the
180-day
lock-up period
described below,
approximately shares
of our common stock will be eligible for sale in accordance with
Rule 701.
Lock-up
Agreements
We expect that the holders of substantially all of our currently
outstanding capital stock will agree that, without the prior
written consent of Morgan Stanley, they will not, during the
period ending 180 days after the date of this prospectus,
subject to exceptions specified in the
lock-up agreements,
offer, sell, contract to sell or otherwise dispose of, directly
or indirectly, or hedge our common stock or securities
convertible into or exchangeable for or exercisable for our
common stock, sell any option or contract to purchase, purchase
any option or contract to sell, grant any option, right or
warrant to purchase, lend or otherwise dispose of, directly or
indirectly, any shares of our common stock or any securities
convertible into or exercisable for our common stock. Further,
these holders have agreed that, during this period, they will
not make any demand for, or exercise any right with respect to,
the registration of our common stock.
Registration Rights
Upon the closing of this offering, the holders of an aggregate
of 84,049,190 shares of our common stock will have the
right to require us to use our best efforts register these
shares under the Securities Act under specified circumstances.
After registration pursuant to these rights, these shares will
become freely tradable without restriction under the Securities
Act. Please see Description of Capital
StockRegistration Rights for additional information
regarding these registration rights.
Stock Options
As of May 3, 2006, we had outstanding options to purchase
13,552,120 shares of common stock, of which options to
purchase 795,381 shares were vested. In connection with
this offering, we intend to file a registration statement on
Form S-8 under the
Securities Act to register all of the shares of common stock
subject to outstanding options and other awards issuable
pursuant to our 2002 equity incentive plan, our 2006 equity
incentive plan and our 2006 employee stock purchase plan. Please
see Management-Stock Option and Other Compensation
Plans for additional information regarding these plans.
Accordingly, shares of our common stock registered under the
registration statements will be available for sale in the open
market, subject to Rule 144 volume limitations applicable
to affiliates, and subject to any vesting restrictions and
lock-up agreements
applicable to these shares.
Warrants
Upon the closing of this offering, we will have an outstanding
warrant to purchase an aggregate of 40,000 shares of our
common stock at an exercise price of $0.75 per share. Any
shares purchased pursuant to the cashless exercise features of
this warrant will be freely tradeable under Rule 144(k),
subject to the 180-day
lock-up period
described above.
107
UNDERWRITERS
Under the terms and subject to the conditions contained in an
underwriting agreement dated the date of this prospectus, the
underwriters named below, for whom Morgan Stanley & Co.
Incorporated, Goldman, Sachs & Co. and Pacific Growth
Equities, LLC are acting as representatives, have severally
agreed to purchase, and we have agreed to sell to them, the
number of shares of common stock indicated in the table below:
|
|
|
|
|
|
Name |
|
Number of Shares | |
|
|
| |
Morgan Stanley & Co. Incorporated
|
|
|
|
|
Goldman, Sachs & Co.
|
|
|
|
|
Pacific Growth Equities, LLC
|
|
|
|
|
|
Total
|
|
|
|
|
The underwriters are offering the shares of common stock subject
to their acceptance of the shares from us and subject to prior
sale. The underwriting agreement provides that the obligations
of the several underwriters to pay for and accept delivery of
the shares of common stock offered by this prospectus are
subject to the approval of certain legal matters by their
counsel and to other conditions. The underwriters are obligated
to take and pay for all of the shares of common stock offered by
this prospectus if any such shares are taken. However, the
underwriters are not required to take or pay for the shares
covered by the underwriters over-allotment option
described below.
The underwriters initially propose to offer part of the shares
of common stock directly to the public at the public offering
price listed on the cover page of this prospectus, and part to
certain dealers at a price that represents a concession not in
excess of
$ a
share under the public offering price. No underwriter may allow,
and no dealer may re-allow, any concession to other underwriters
or to certain dealers. After the initial offering of the shares
of common stock, the offering price and other selling terms may
from time to time be varied by the representatives.
We have granted to the underwriters an option, exercisable for
30 days from the date of this prospectus, to purchase up to
an aggregate
of additional
shares of common stock at the public offering price, less
underwriting discounts and commissions. The underwriters may
exercise this option solely for the purpose of covering
over-allotments, if any, made in connection with the offering of
the shares of common stock offered by this prospectus. To the
extent the option is exercised, each underwriter will become
obligated, subject to certain conditions, to purchase
approximately the same percentage of the additional shares of
common stock as the number listed next to the underwriters
name in the preceding table bears to the total number of shares
of common stock listed next to the names of all underwriters in
the preceding table. If the underwriters over-allotment
option is exercised in full, the total price to the public would
be
$ ,
the total underwriters discounts and commissions would be
$ and
the total proceeds to us would be
$ .
The following table shows the per share and total underwriting
discounts and commissions that we are to pay to the underwriters
in connection with this offering. These amounts are shown
assuming both no exercise and full exercise of the
underwriters option.
|
|
|
|
|
|
|
|
|
|
|
No | |
|
Full | |
|
|
Exercise | |
|
Exercise | |
|
|
| |
|
| |
Per share
|
|
$ |
|
|
|
$ |
|
|
Total
|
|
$ |
|
|
|
$ |
|
|
In addition, we estimate that the expenses of this offering
payable by us, other than underwriting discounts and
commissions, will be approximately
$ million.
The underwriters have informed us that they do not intend sales
to discretionary accounts to exceed 5% of the total number of
shares of common stock offered by them.
108
We, all of our directors and officers and holders of
substantially all our outstanding stock have agreed that,
without the prior written consent of Morgan Stanley &
Co. Incorporated on behalf of the underwriters, we and they will
not, during the period ending 180 days after the date of
this prospectus:
|
|
|
|
|
offer, pledge, sell, contract to sell, sell any option or
contract to purchase, purchase any option or contract to sell,
grant any option, right or warrant to purchase, lend, or
otherwise transfer or dispose of, directly or indirectly, any
shares of common stock or any securities convertible into or
exercisable or exchangeable for common stock; or |
|
|
|
enter into any swap or other arrangement that transfers to
another, in whole or in part, any of the economic consequences
of ownership of the common stock. |
whether any such transaction described above is to be settled by
delivery of common stock or such other securities, in cash or
otherwise.
The 180-day restricted
period described in the preceding paragraph will be extended if:
|
|
|
|
|
during the last 17 days of the
180-day restricted
period we issue an earnings release or material news or a
material event relating to our company occurs; or |
|
|
|
prior to the expiration of the
180-day restricted
period, we announce that we will release earnings results during
the 16-day period
beginning on the last day of the
180-day period, |
in which case the restrictions described in the preceding
paragraph will continue to apply until the expiration of the
18-day period beginning
on the issuance of the earnings release or the occurrence of the
material news or material event.
These restrictions do not apply to:
|
|
|
|
|
the sale of shares to the underwriters; |
|
|
|
the issuance by us of shares of common stock upon the exercise
of an option or a warrant or the conversion of a security
outstanding on the date of this prospectus of which the
underwriters have been advised in writing; |
|
|
|
the grant of options or the issuance of shares of common stock
by us pursuant to equity incentive plans described in this
prospectus, provided that the recipient of the option or shares
agree to be subject to the restrictions described in this
paragraph; |
|
|
|
the issuance by us of shares of common stock in connection with
any strategic transactions, such as collaboration or license
agreements, provided that the recipient of the shares agrees to
be subject to the restrictions described in this paragraph; |
|
|
|
transactions by any person other than us relating to shares of
common stock or other securities acquired in open market
transactions after the completion of the offering of the shares; |
|
|
|
transfers by any person other than us of shares of common stock
or other securities as a bona fide gift or in connection with
bona fide estate planning or by intestacy; or |
|
|
|
distributions by any person other than by us of shares of common
stock or other securities to limited partners, members,
stockholders or affiliates of such person; |
provided that in the case of each of the last three
transactions, no filing under Section 16(a) of the Exchange
Act is required or is voluntarily made in connection with the
transaction, and in the case of each of the last two
transactions, each done or distribute agrees to be subject to
the restrictions on transfer described above.
In order to facilitate this offering of common stock, the
underwriters may engage in transactions that stabilize, maintain
or otherwise affect the price of the common stock. Specifically,
the underwriters may sell more shares than they are obligated to
purchase under the underwriting agreement, creating a short
position. A short sale is covered if the short position is no
greater than the number of shares available for
109
purchase by the underwriters under the over-allotment option.
The underwriters can close out a covered short sale by
exercising the over-allotment option or by purchasing shares in
the open market. In determining the source of shares to close
out a covered short sale, the underwriters will consider, among
other things, the open market price of shares compared to the
price available under the over-allotment option. The
underwriters may also sell shares in excess of the
over-allotment option, creating a naked short position. The
underwriters must close out any naked short position by
purchasing shares in the open market. A naked short position is
more likely to be created if the underwriters are concerned that
there may be downward pressure on the price of the common stock
in the open market after pricing that could adversely affect
investors who purchase in this offering. In addition, to
stabilize the price of the common stock, the underwriters may
bid for and purchase shares of common stock in the open market.
Finally, the underwriters may reclaim selling concessions
allowed to an underwriter or a dealer for distributing the
common stock in the offering, if the syndicate repurchases
previously distributed common stock to cover syndicate short
positions or to stabilize the price of the common stock. Any of
these activities may raise or maintain the market price of the
common stock above independent market levels or prevent or
retard a decline in the market price of the common stock. The
underwriters are not required to engage in these activities and
may end any of these activities at any time.
We have applied for quotation of our common stock approved for
quotation on the Nasdaq National Market under the symbol
AMTX.
We and the underwriters have agreed to indemnify each other
against certain liabilities, including liabilities under the
Securities Act.
Directed Share Program
At our request, the underwriters have reserved for sale, at the
initial public offering price, up
to shares
offered by this prospectus to directors, officers, employees and
other individuals associated with us through a directed share
program. The number of shares of our common stock available for
sale to the general public in the offering will be reduced to
the extent these persons purchase these reserved shares. Any
reserved shares not purchased by these persons will be offered
by the underwriters to the general public on the same basis as
the other shares offered by this prospectus. Recipients of
reserved shares will be required to agree with the underwriters
not to sell, transfer, assign, pledge or hypothecate these
shares for a period of 180 days after purchasing the shares.
Pricing of the Offering
Prior to this offering, there has been no public market for the
shares of common stock. The initial public offering price will
be determined by negotiations between us and the representatives
of the underwriters. Among the factors to be considered in
determining the initial public offering price will be our future
prospects and those of our industry in general; sales, earnings
and other financial operating information in recent periods; and
the price-earnings ratios, price-sales ratios and market prices
of securities and certain financial and operating information of
companies engaged in activities similar to ours. The estimated
initial public offering price range set forth on the cover page
of this preliminary prospectus is subject to change as a result
of market conditions and other factors.
A prospectus in electronic format may be made available on the
web sites maintained by one or more of the underwriters, and one
or more of the underwriters may distribute prospectuses
electronically. The underwriters may agree to allocate a number
of shares to underwriters for sale to their online brokerage
account holders. Internet distributions will be allocated by the
underwriters that make Internet distributions on the same basis
as other allocations.
110
LEGAL MATTERS
The validity of the common stock we are offering will be passed
upon by Bingham McCutchen LLP. Ropes & Gray LLP
has acted as counsel for the underwriters in connection with
certain legal matters related to this offering.
EXPERTS
Ernst & Young LLP, independent registered public
accounting firm, has audited our financial statements at
December 31, 2005 and 2004, and for each of the three years
in the period ended December 31, 2005, as set forth in
their report. We have included our financial statements in the
prospectus and elsewhere in the registration statement in
reliance on Ernst & Young LLPs report, given on
their authority as experts in accounting and auditing.
WHERE YOU CAN FIND MORE INFORMATION
We have filed with the Securities and Exchange Commission a
Registration Statement on
Form S-1 under the
Securities Act with respect to the shares of common stock we are
offering to sell. This prospectus, which constitutes part of the
Registration Statement, does not include all of the information
contained in the Registration Statement and the exhibits,
schedules and amendments to the Registration Statement. For
further information with respect to us and our common stock, we
refer you to the Registration Statement and to the exhibits and
schedules to the Registration Statement. Statements contained in
this prospectus about the contents of any contract or any other
document are not necessarily complete, and, in each instance, we
refer you to the copy of the contract or other documents filed
as an exhibit to the Registration Statement. Each of these
statements is qualified in all respects by this reference.
You may read and copy the Registration Statement of which this
prospectus is a part at the Securities and Exchange
Commissions public reference room, which is located at 100
F Street, N.E., Room 1580, Washington, D.C. 20549. You
can request copies of the Registration Statement by writing to
the Securities and Exchange Commission and paying a fee for the
copying cost. Please call the Securities and Exchange Commission
at 1-800-SEC-0330 for
more information about the operation of the Securities and
Exchange Commissions public reference room. In addition,
the Securities and Exchange Commission maintains an Internet
website, which is located at http://www.sec.gov, that contains
reports, proxy and information statements and other information
regarding issuers that file electronically with the Securities
and Exchange Commission. You may access the Registration
Statement of which this prospectus is a part at the Securities
and Exchange Commissions Internet website. Upon closing of
this offering, we will be subject to the information reporting
requirements of the Securities Exchange Act of 1934, as amended,
and we will file reports, proxy statements and other information
with the Securities and Exchange Commission.
This prospectus includes statistical data that were obtained
from industry publications. These industry publications
generally indicate that the authors of these publications have
obtained information from sources believed to be reliable but do
not guarantee the accuracy and completeness of their
information. While we believe these industry publications to be
reliable, we have not independently verified their data.
111
Amicus Therapeutics, Inc.
(a development stage company)
Financial Statements
March 31, 2006 (unaudited)
Contents
F-1
Report of Independent Registered Public Accounting Firm
Board of Directors and Stockholders
Amicus Therapeutics, Inc.
We have audited the balance sheets of Amicus Therapeutics, Inc.
(a development stage company) as of December 31, 2004 and
2005 and the related statements of operations, changes in
stockholders deficiency and cash flows for each of the
three years in the period ended December 31, 2005. These
financial statements are the responsibility of the
Companys management. Our responsibility is to express an
opinion on these financial statements based on our audits.
We conducted our audits in accordance with the standards of the
Public Company Accounting Oversight Board (United States). Those
standards require that we plan and perform the audit to obtain
reasonable assurance about whether the financial statements are
free of material misstatement. We were not engaged to perform an
audit of the Companys internal control over financial
reporting. Our audits included consideration of internal control
over financial reporting as a basis for designing audit
procedures that are appropriate in the circumstances, but not
for the purpose of expressing an opinion on the effectiveness of
the Companys internal control over financial reporting.
Accordingly, we express no such opinion. An audit also includes
examining, on a test basis, evidence supporting the amounts and
disclosures in the financial statements, assessing the
accounting principles used and significant estimates made by
management, and evaluating the overall financial statement
presentation. We believe that our audits provide a reasonable
basis for our opinion.
In our opinion, the financial statements referred to above
present fairly, in all material respects, the financial position
of Amicus Therapeutics, Inc. as of December 31, 2004 and
2005 and the results of its operations and its cash flows for
each of the three years in the period ended December 31,
2005, in conformity with U.S. generally accepted accounting
principles.
MetroPark, New Jersey
May 12, 2006
F-2
Amicus Therapeutics, Inc.
(a development stage company)
Balance Sheets
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
March 31, | |
|
|
December 31, | |
|
| |
|
|
| |
|
|
|
Pro Forma | |
|
|
2004 | |
|
2005 | |
|
2006 | |
|
(Note 1) | |
|
|
| |
|
| |
|
| |
|
| |
|
|
|
|
|
|
(unaudited) | |
|
(unaudited) | |
Assets |
Current assets:
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Cash and cash equivalents
|
|
$ |
257,036 |
|
|
$ |
6,449,151 |
|
|
$ |
10,298,878 |
|
|
$ |
38,270,631 |
|
|
Investment in marketable securities
|
|
|
4,078,925 |
|
|
|
17,969,096 |
|
|
|
9,253,492 |
|
|
|
9,253,492 |
|
|
Prepaid expenses and other current assets
|
|
|
155,383 |
|
|
|
441,081 |
|
|
|
278,538 |
|
|
|
278,538 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Total current assets
|
|
|
4,491,344 |
|
|
|
24,859,328 |
|
|
|
19,830,908 |
|
|
|
47,802,661 |
|
Property and equipment, net
|
|
|
541,277 |
|
|
|
3,278,887 |
|
|
|
3,696,596 |
|
|
|
3,696,596 |
|
Other non-current assets
|
|
|
40,537 |
|
|
|
531,739 |
|
|
|
467,338 |
|
|
|
467,338 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
$ |
5,073,158 |
|
|
$ |
28,669,954 |
|
|
$ |
23,994,842 |
|
|
$ |
51,966,595 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Liabilities, redeemable convertible preferred stock and
stockholders equity (deficiency) |
Current liabilities:
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Accounts payable
|
|
|
589,919 |
|
|
|
906,226 |
|
|
|
532,866 |
|
|
|
532,866 |
|
|
Accrued expenses and other current liabilities
|
|
|
157,820 |
|
|
|
1,407,025 |
|
|
|
2,439,732 |
|
|
|
2,439,732 |
|
|
Current portion of capital lease obligations
|
|
|
174,545 |
|
|
|
279,265 |
|
|
|
852,527 |
|
|
|
852,527 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Total current liabilities
|
|
|
922,284 |
|
|
|
2,592,516 |
|
|
|
3,825,125 |
|
|
|
3,825,125 |
|
Capital lease obligations, less current portion
|
|
|
|
|
|
|
734,370 |
|
|
|
1,993,960 |
|
|
|
1,993,960 |
|
Commitments and contingencies
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Series A redeemable convertible preferred stock,
$.01 par value; 3,333,334 shares authorized, issued
and outstanding at December 31, 2004, 2005 and
March 31, 2006 (unaudited) (aggregate liquidation
preference $2,500,000 at December 31, 2004, 2005, and
March 31, 2006 (unaudited)), zero pro-forma shares
outstanding (unaudited)
|
|
|
2,449,321 |
|
|
|
2,466,214 |
|
|
|
2,470,437 |
|
|
|
|
|
Series B redeemable convertible preferred stock,
$.01 par value; 37,025,594 shares authorized and
21,176,472, 36,470,591, and 36,470,591 shares issued and
outstanding at December 31, 2004, 2005, and March 31,
2006 (unaudited), respectively (aggregate liquidation preference
$18,000,000, $31,000,000, $31,000,000 at December 31, 2004,
2005, and March 31, 2006 (unaudited) respectively),
zero pro-forma shares outstanding (unaudited)
|
|
|
17,564,636 |
|
|
|
30,668,842 |
|
|
|
30,696,342 |
|
|
|
|
|
Series C redeemable convertible preferred stock,
$.01 par value; 43,650,262 shares authorized and
21,825,131 shares issued and outstanding at
December 31, 2005 and March 31, 2006 (unaudited)
(aggregate liquidation preference $27,499,665 at
December 31, 2005 and March 31, 2006 (unaudited)),
zero pro-forma shares outstanding (unaudited)
|
|
|
|
|
|
|
27,333,758 |
|
|
|
27,342,646 |
|
|
|
|
|
Stockholders equity (deficiency):
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Common stock, $.01 par value; 115,000,000 shares
authorized and 2,306,541, 4,035,231, 4,635,231, and
66,264,287 shares issued and outstanding at
December 31, 2004, 2005, March 31, 2006 (unaudited),
and March 31, 2006 Pro Forma (unaudited), respectively
|
|
|
23,065 |
|
|
|
40,352 |
|
|
|
46,352 |
|
|
|
886,444 |
|
|
Additional paid-in capital
|
|
|
1,604,349 |
|
|
|
4,436,942 |
|
|
|
2,296,784 |
|
|
|
89,937,870 |
|
|
Accumulated other comprehensive loss
|
|
|
(9,083 |
) |
|
|
(16,139 |
) |
|
|
(5,320 |
) |
|
|
(5,320 |
) |
|
Deferred compensation
|
|
|
(133,174 |
) |
|
|
(2,546,846 |
) |
|
|
|
|
|
|
|
|
|
Deficit accumulated during the development stage
|
|
|
(17,348,240 |
) |
|
|
(37,040,055 |
) |
|
|
(44,671,484 |
) |
|
|
(44,671,484 |
) |
|
|
|
|
|
|
|
|
|
|
|
|
|
Total stockholders equity (deficiency)
|
|
|
(15,863,083 |
) |
|
|
(35,125,746 |
) |
|
|
(42,333,668 |
) |
|
|
46,147,510 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
$ |
5,073,158 |
|
|
$ |
28,669,954 |
|
|
$ |
23,994,842 |
|
|
$ |
51,966,595 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
See accompanying notes.
F-3
Amicus Therapeutics, Inc.
(a development stage company)
Statements of Operations
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Period | |
|
|
|
|
|
|
|
|
|
|
|
|
from | |
|
|
|
|
|
|
|
|
|
|
February 4, | |
|
|
|
|
Three Months Ended | |
|
2002 | |
|
|
Years Ended December 31, | |
|
March 31, | |
|
(inception) to | |
|
|
| |
|
| |
|
March 31, | |
|
|
2003 | |
|
2004 | |
|
2005 | |
|
2005 | |
|
2006 | |
|
2006 | |
|
|
| |
|
| |
|
| |
|
| |
|
| |
|
| |
|
|
|
|
|
|
|
|
|
|
(unaudited) | |
|
(unaudited) | |
|
|
|
|
|
|
|
|
(unaudited) | |
|
(Restated) | |
|
(Restated) | |
Revenue
|
|
$ |
|
|
|
$ |
|
|
|
$ |
|
|
|
$ |
|
|
|
$ |
|
|
|
$ |
|
|
Operating Expenses:
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Research and development
|
|
|
4,433,059 |
|
|
|
6,300,885 |
|
|
|
13,651,640 |
|
|
|
2,238,366 |
|
|
|
5,545,735 |
|
|
|
30,719,421 |
|
|
General and administrative
|
|
|
1,005,416 |
|
|
|
2,081,203 |
|
|
|
6,876,883 |
|
|
|
1,177,779 |
|
|
|
2,065,467 |
|
|
|
12,580,823 |
|
|
Impairment of leasehold improvements
|
|
|
1,029,696 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
1,029,696 |
|
|
Depreciation and amortization
|
|
|
131,931 |
|
|
|
145,961 |
|
|
|
302,832 |
|
|
|
46,712 |
|
|
|
199,224 |
|
|
|
804,088 |
|
|
In-process research and development
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
418,080 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Total operating expenses
|
|
|
6,600,102 |
|
|
|
8,528,049 |
|
|
|
20,831,355 |
|
|
|
3,462,857 |
|
|
|
7,810,426 |
|
|
|
45,552,108 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Loss from operations
|
|
|
(6,600,102 |
) |
|
|
(8,528,049 |
) |
|
|
(20,831,355 |
) |
|
|
(3,462,857 |
) |
|
|
(7,810,426 |
) |
|
|
(45,552,108 |
) |
Other income (expenses):
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Interest income
|
|
|
4,878 |
|
|
|
189,847 |
|
|
|
609,519 |
|
|
|
56,976 |
|
|
|
237,909 |
|
|
|
1,054,767 |
|
|
Interest expense
|
|
|
(172,472 |
) |
|
|
(550,004 |
) |
|
|
(81,776 |
) |
|
|
(4,069 |
) |
|
|
(58,912 |
) |
|
|
(868,955 |
) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Loss before tax benefit
|
|
|
(6,767,696 |
) |
|
|
(8,888,206 |
) |
|
|
(20,303,612 |
) |
|
|
(3,409,950 |
) |
|
|
(7,631,429 |
) |
|
|
(45,366,296 |
) |
Income tax benefit
|
|
|
|
|
|
|
83,015 |
|
|
|
611,797 |
|
|
|
|
|
|
|
|
|
|
|
694,812 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Net loss
|
|
|
(6,767,696 |
) |
|
|
(8,805,191 |
) |
|
|
(19,691,815 |
) |
|
|
(3,409,950 |
) |
|
|
(7,631,429 |
) |
|
|
(44,671,484 |
) |
|
Preferred stock accretion
|
|
|
(16,893 |
) |
|
|
(125,733 |
) |
|
|
(138,742 |
) |
|
|
(31,723 |
) |
|
|
(40,611 |
) |
|
|
(332,699 |
) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Net loss attributable to common stockholders
|
|
$ |
(6,784,589 |
) |
|
$ |
(8,930,924 |
) |
|
$ |
(19,830,557 |
) |
|
$ |
(3,441,673 |
) |
|
$ |
(7,672,040 |
) |
|
$ |
(45,004,183 |
) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Net loss attributable to common stockholders per common
share basic and diluted
|
|
$ |
(2.94 |
) |
|
$ |
(3.87 |
) |
|
$ |
(6.45 |
) |
|
$ |
(1.49 |
) |
|
$ |
(1.81 |
) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Weighted-average common shares outstanding basic and
diluted
|
|
|
2,306,541 |
|
|
|
2,306,541 |
|
|
|
3,076,649 |
|
|
|
2,314,804 |
|
|
|
4,228,564 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Unaudited pro forma net loss
|
|
|
|
|
|
|
|
|
|
$ |
(19,691,815 |
) |
|
|
|
|
|
$ |
(7,631,429 |
) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Unaudited basic and diluted pro forma net loss per share
|
|
|
|
|
|
|
|
|
|
$ |
(0.23 |
) |
|
|
|
|
|
$ |
(0.09 |
) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Unaudited basic and diluted pro forma weighted-average shares
outstanding
|
|
|
|
|
|
|
|
|
|
|
87,085,839 |
|
|
|
|
|
|
|
88,237,754 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
See accompanying notes.
F-4
Amicus Therapeutics, Inc.
(a development stage company)
Statements of Changes in Stockholders Deficiency
Period from February 4, 2002 (inception) to
December 31, 2002,
the three year period ended December 31, 2005,
and the three months ended March 31, 2006 (unaudited)
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Accumulated | |
|
|
|
|
|
|
|
|
Common Stock | |
|
Additional | |
|
Other | |
|
|
|
Deficit Accumulated | |
|
Total | |
|
|
| |
|
Paid-In | |
|
Comprehensive | |
|
Deferred | |
|
During the Development | |
|
Stockholders | |
|
|
Shares | |
|
Amount | |
|
Capital | |
|
Loss | |
|
Compensation | |
|
Stage | |
|
Deficiency | |
|
|
| |
|
| |
|
| |
|
| |
|
| |
|
| |
|
| |
Balance at February 4, 2002 (inception)
|
|
|
|
|
|
$ |
|
|
|
$ |
|
|
|
$ |
|
|
|
$ |
|
|
|
$ |
|
|
|
$ |
|
|
|
Issuance of common stock to a consultant
|
|
|
562,041 |
|
|
|
5,620 |
|
|
|
78,243 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
83,863 |
|
|
Stock issued for in-process research and development
|
|
|
1,742,000 |
|
|
|
17,420 |
|
|
|
400,660 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
418,080 |
|
|
Deferred compensation
|
|
|
|
|
|
|
|
|
|
|
208,866 |
|
|
|
|
|
|
|
(208,866 |
) |
|
|
|
|
|
|
|
|
|
Amortization of deferred compensation
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
27,348 |
|
|
|
|
|
|
|
27,348 |
|
|
Issuance of warrants with financing arrangement
|
|
|
|
|
|
|
|
|
|
|
8,000 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
8,000 |
|
|
Accretion of series A redeemable convertible preferred stock
|
|
|
|
|
|
|
|
|
|
|
(10,720 |
) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
(10,720 |
) |
|
Net loss
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
(1,775,353 |
) |
|
|
(1,775,353 |
) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Balance at December 31, 2002
|
|
|
2,304,041 |
|
|
|
23,040 |
|
|
|
685,049 |
|
|
|
|
|
|
|
(181,518 |
) |
|
|
(1,775,353 |
) |
|
|
(1,248,782 |
) |
|
Stock issued from exercise of options
|
|
|
2,500 |
|
|
|
25 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
25 |
|
|
Deferred compensation
|
|
|
|
|
|
|
|
|
|
|
14,138 |
|
|
|
|
|
|
|
(14,138 |
) |
|
|
|
|
|
|
|
|
|
Amortization of deferred compensation
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
70,340 |
|
|
|
|
|
|
|
70,340 |
|
|
Issuance of stock warrants with convertible notes
|
|
|
|
|
|
|
|
|
|
|
210,000 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
210,000 |
|
|
Issuance of stock options to consultants
|
|
|
|
|
|
|
|
|
|
|
4,434 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
4,434 |
|
|
Accretion of series A redeemable convertible preferred stock
|
|
|
|
|
|
|
|
|
|
|
(16,893 |
) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
(16,893 |
) |
|
Beneficial conversion feature related to bridge financing
|
|
|
|
|
|
|
|
|
|
|
40,500 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
40,500 |
|
|
Net loss
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
(6,767,696 |
) |
|
|
(6,767,696 |
) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Balance at December 31, 2003
|
|
|
2,306,541 |
|
|
|
23,065 |
|
|
|
937,228 |
|
|
|
|
|
|
|
(125,316 |
) |
|
|
(8,543,049 |
) |
|
|
(7,708,072 |
) |
|
Deferred compensation
|
|
|
|
|
|
|
|
|
|
|
67,700 |
|
|
|
|
|
|
|
(67,700 |
) |
|
|
|
|
|
|
|
|
|
Amortization of deferred compensation
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
59,842 |
|
|
|
|
|
|
|
59,842 |
|
|
Issuance of stock options to consultants
|
|
|
|
|
|
|
|
|
|
|
16,118 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
16,118 |
|
|
Accretion of series A redeemable convertible preferred stock
|
|
|
|
|
|
|
|
|
|
|
(16,893 |
) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
(16,893 |
) |
|
Accretion of series B redeemable convertible preferred stock
|
|
|
|
|
|
|
|
|
|
|
(108,840 |
) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
(108,840 |
) |
|
Issuance of warrants with series B redeemable convertible
preferred stock
|
|
|
|
|
|
|
|
|
|
|
421,802 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
421,802 |
|
|
Interest waived on converted convertible notes
|
|
|
|
|
|
|
|
|
|
|
192,734 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
192,734 |
|
|
Beneficial conversion feature related to bridge financing
|
|
|
|
|
|
|
|
|
|
|
94,500 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
94,500 |
|
|
Comprehensive loss:
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Unrealized holding loss on available-for-sale securities
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
(9,083 |
) |
|
|
|
|
|
|
|
|
|
|
(9,083 |
) |
|
|
Net loss
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
(8,805,191 |
) |
|
|
(8,805,191 |
) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Net total comprehensive loss
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
(8,814,274 |
) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Balance at December 31, 2004
|
|
|
2,306,541 |
|
|
$ |
23,065 |
|
|
$ |
1,604,349 |
|
|
$ |
(9,083 |
) |
|
$ |
(133,174 |
) |
|
$ |
(17,348,240 |
) |
|
$ |
(15,863,083 |
) |
F-5
Amicus Therapeutics, Inc.
(a development stage company)
Statements of Changes in Stockholders
Deficiency(Continued)
Period from February 4, 2002 (inception) to
December 31, 2002,
the Three Year Period Ended December 31, 2005,
and the Three Months Ended March 31, 2006 (unaudited)
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Accumulated | |
|
|
|
|
|
|
|
|
Common Stock | |
|
Additional | |
|
Other | |
|
|
|
Deficit Accumulated | |
|
Total | |
|
|
| |
|
Paid-In | |
|
Comprehensive | |
|
Deferred | |
|
During the Development | |
|
Stockholders | |
|
|
Shares | |
|
Amount | |
|
Capital | |
|
Loss | |
|
Compensation | |
|
Stage | |
|
Deficiency | |
|
|
| |
|
| |
|
| |
|
| |
|
| |
|
| |
|
| |
Balance at December 31, 2004 (carried forward)
|
|
|
2,306,541 |
|
|
$ |
23,065 |
|
|
$ |
1,604,349 |
|
|
$ |
(9,083 |
) |
|
$ |
(133,174 |
) |
|
$ |
(17,348,240 |
) |
|
$ |
(15,863,083 |
) |
|
Stocks issued from exercise of stock options
|
|
|
728,691 |
|
|
|
7,287 |
|
|
|
16,641 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
23,928 |
|
|
Stocks issued from exercise of warrants
|
|
|
999,999 |
|
|
|
10,000 |
|
|
|
65,000 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
75,000 |
|
|
Deferred compensation
|
|
|
|
|
|
|
|
|
|
|
2,778,223 |
|
|
|
|
|
|
|
(2,778,223 |
) |
|
|
|
|
|
|
|
|
|
Amortization of deferred compensation
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
364,551 |
|
|
|
|
|
|
|
364,551 |
|
|
Non-cash charge for stock options to consultants
|
|
|
|
|
|
|
|
|
|
|
111,471 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
111,471 |
|
|
Accretion of series A redeemable convertible preferred stock
|
|
|
|
|
|
|
|
|
|
|
(16,893 |
) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
(16,893 |
) |
|
Accretion of series B redeemable convertible preferred stock
|
|
|
|
|
|
|
|
|
|
|
(109,999 |
) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
(109,999 |
) |
|
Accretion of series C redeemable convertible preferred stock
|
|
|
|
|
|
|
|
|
|
|
(11,850 |
) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
(11,850 |
) |
|
Comprehensive loss:
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Unrealized holding loss on available-for-sale securities
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
(7,056 |
) |
|
|
|
|
|
|
|
|
|
|
(7,056 |
) |
|
|
Net loss
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
(19,691,815 |
) |
|
|
(19,691,815 |
) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Net total comprehensive loss
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
(19,698,871 |
) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Balance at December 31, 2005
|
|
|
4,035,231 |
|
|
|
40,352 |
|
|
|
4,436,942 |
|
|
|
(16,139 |
) |
|
|
(2,546,846 |
) |
|
|
(37,040,055 |
) |
|
|
(35,125,746 |
) |
|
Stocks issued from exercise of options
|
|
|
600,000 |
|
|
|
6,000 |
|
|
|
45,000 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
51,000 |
|
|
Reversal of deferred compensation upon adoption of
FAS 123(R)
|
|
|
|
|
|
|
|
|
|
|
(2,546,846 |
) |
|
|
|
|
|
|
2,546,846 |
|
|
|
|
|
|
|
|
|
|
Stock-based compensation
|
|
|
|
|
|
|
|
|
|
|
315,671 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
331,791 |
|
|
Issuance of stock options to consultants
|
|
|
|
|
|
|
|
|
|
|
86,628 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
70,508 |
|
|
Accretion of series A redeemable convertible preferred stock
|
|
|
|
|
|
|
|
|
|
|
(4,223 |
) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
(4,223 |
) |
|
Accretion of series B redeemable convertible preferred stock
|
|
|
|
|
|
|
|
|
|
|
(27,500 |
) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
(27,500 |
) |
|
Accretion of series C redeemable convertible preferred stock
|
|
|
|
|
|
|
|
|
|
|
(8,888 |
) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
(8,888 |
) |
|
Comprehensive loss:
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Unrealized gain on available-for-sale securities
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
10,819 |
|
|
|
|
|
|
|
|
|
|
|
10,819 |
|
|
|
Net loss
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
(7,631,429 |
) |
|
|
(7,631,429 |
) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Net total comprehensive loss
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
(7,620,610 |
) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Balance at March 31, 2006 (unaudited)
|
|
|
4,635,231 |
|
|
$ |
46,352 |
|
|
$ |
2,296,784 |
|
|
$ |
(5,320 |
) |
|
$ |
|
|
|
$ |
(44,671,484 |
) |
|
$ |
(42,333,668 |
) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
See accompanying notes.
F-6
Amicus Therapeutics, Inc.
(a development stage company)
Statements of Cash Flows
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Period | |
|
|
|
|
|
|
|
|
|
|
|
|
from | |
|
|
|
|
|
|
|
|
|
|
February 4, | |
|
|
|
|
Three Months Ended | |
|
2002 | |
|
|
Years Ended December 31, | |
|
March 31, | |
|
(inception) to | |
|
|
| |
|
| |
|
March 31, | |
|
|
2003 | |
|
2004 | |
|
2005 | |
|
2005 | |
|
2006 | |
|
2006 | |
|
|
| |
|
| |
|
| |
|
| |
|
| |
|
| |
|
|
|
|
|
|
|
|
(unaudited) | |
|
(unaudited) | |
|
(unaudited) | |
Operating activities
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Net loss
|
|
$ |
(6,767,696 |
) |
|
$ |
(8,805,191 |
) |
|
$ |
(19,691,815 |
) |
|
$ |
(3,409,950 |
) |
|
$ |
(7,631,429 |
) |
|
$ |
(44,671,484 |
) |
Adjustments to reconcile net loss to net cash used in operating
activities:
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Non-cash interest expense
|
|
|
86,666 |
|
|
|
435,934 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
525,267 |
|
|
Depreciation and amortization
|
|
|
131,931 |
|
|
|
143,293 |
|
|
|
302,832 |
|
|
|
46,712 |
|
|
|
199,224 |
|
|
|
801,420 |
|
|
Amortization of non-cash compensation
|
|
|
70,340 |
|
|
|
59,842 |
|
|
|
364,551 |
|
|
|
91,138 |
|
|
|
|
|
|
|
522,081 |
|
|
Stock-based compensation
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
315,671 |
|
|
|
315,671 |
|
|
Non-cash charge for stock issued to consultants
|
|
|
4,434 |
|
|
|
16,118 |
|
|
|
111,471 |
|
|
|
27,868 |
|
|
|
86,628 |
|
|
|
302,514 |
|
|
Impairment of leasehold improvements
|
|
|
1,029,696 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
1,029,696 |
|
|
Non-cash charge for in process research and development
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
418,080 |
|
|
Beneficial conversion feature related to bridge financing
|
|
|
40,500 |
|
|
|
94,500 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
135,000 |
|
Changes in operating assets and liabilities:
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Prepaid expenses and other current assets
|
|
|
1,217 |
|
|
|
(147,664 |
) |
|
|
(285,698 |
) |
|
|
(17,637 |
) |
|
|
162,543 |
|
|
|
(278,538 |
) |
|
|
Other non-current assets
|
|
|
|
|
|
|
(19,936 |
) |
|
|
(491,202 |
) |
|
|
(28,813 |
) |
|
|
64,401 |
|
|
|
(488,505 |
) |
|
|
Accounts payable and accrued expenses
|
|
|
1,526,439 |
|
|
|
(1,008,299 |
) |
|
|
1,565,512 |
|
|
|
422,700 |
|
|
|
659,347 |
|
|
|
2,972,598 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Net cash used in operating activities
|
|
|
(3,876,473 |
) |
|
|
(9,231,403 |
) |
|
|
(18,124,349 |
) |
|
|
(2,867,982 |
) |
|
|
(6,143,615 |
) |
|
|
(38,416,200 |
) |
Investing activities
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Sale and redemption of marketable securities
|
|
|
|
|
|
|
2,162,275 |
|
|
|
3,092,620 |
|
|
|
|
|
|
|
8,726,423 |
|
|
|
13,981,318 |
|
Purchases of marketable securities
|
|
|
(4,956 |
) |
|
|
(6,362,527 |
) |
|
|
(16,989,847 |
) |
|
|
(10,065,364 |
) |
|
|
|
|
|
|
(23,357,330 |
) |
Purchases of property and equipment
|
|
|
(1,088,009 |
) |
|
|
(227,317 |
) |
|
|
(3,040,442 |
) |
|
|
(71,230 |
) |
|
|
(616,933 |
) |
|
|
(5,527,712 |
) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Net cash (used in) provided by investing activities
|
|
|
(1,092,965 |
) |
|
|
(4,427,569 |
) |
|
|
(16,937,669 |
) |
|
|
(10,136,594 |
) |
|
|
8,109,490 |
|
|
|
(14,903,724 |
) |
Financing activities
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Proceeds from issuance of preferred stock, net of issuance costs
|
|
|
|
|
|
|
12,877,598 |
|
|
|
40,316,115 |
|
|
|
13,000,000 |
|
|
|
|
|
|
|
55,598,528 |
|
Proceeds from issuance of convertible notes
|
|
|
3,800,000 |
|
|
|
1,200,000 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
5,000,000 |
|
Payments of capital lease obligations
|
|
|
(152,303 |
) |
|
|
(171,914 |
) |
|
|
(272,697 |
) |
|
|
|
|
|
|
(175,114 |
) |
|
|
(772,028 |
) |
Proceeds from exercise of stock options
|
|
|
25 |
|
|
|
|
|
|
|
23,928 |
|
|
|
832 |
|
|
|
51,000 |
|
|
|
74,953 |
|
Proceeds from exercise of warrants
|
|
|
|
|
|
|
|
|
|
|
75,000 |
|
|
|
|
|
|
|
|
|
|
|
75,000 |
|
Proceeds from capital asset financing arrangement
|
|
|
|
|
|
|
|
|
|
|
1,111,787 |
|
|
|
(50,748 |
) |
|
|
2,007,966 |
|
|
|
3,642,349 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Net cash provided by financing activities
|
|
|
3,647,722 |
|
|
|
13,905,684 |
|
|
|
41,254,133 |
|
|
|
12,950,084 |
|
|
|
1,883,852 |
|
|
|
63,618,802 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Net (decrease) increase in cash and cash equivalents
|
|
|
(1,321,716 |
) |
|
|
246,712 |
|
|
|
6,192,115 |
|
|
|
(54,492 |
) |
|
|
3,849,727 |
|
|
|
10,298,878 |
|
Cash and cash equivalents at beginning of year/ period
|
|
|
1,332,040 |
|
|
|
10,324 |
|
|
|
257,036 |
|
|
|
257,036 |
|
|
|
6,449,151 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Cash and cash equivalents at end of year/ period
|
|
$ |
10,324 |
|
|
$ |
257,036 |
|
|
$ |
6,449,151 |
|
|
$ |
202,544 |
|
|
$ |
10,298,878 |
|
|
$ |
10,298,878 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Supplemental disclosures of cash flow information
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Cash paid during the period for interest
|
|
$ |
45,306 |
|
|
$ |
19,570 |
|
|
$ |
481,577 |
|
|
$ |
3,935 |
|
|
$ |
34,453 |
|
|
$ |
549,577 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Non-cash activities
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Warrant issued with convertible notes
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
8,000 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Warrant issued with Series B redeemable convertible
preferred stock
|
|
|
|
|
|
|
1,802 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
49,950 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Conversion of notes payable to Series B redeemable
convertible preferred stock
|
|
|
|
|
|
|
5,000,000 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
5,000,000 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Accretion of redeemable convertible preferred stock
|
|
$ |
16,893 |
|
|
$ |
125,733 |
|
|
$ |
138,742 |
|
|
$ |
31,723 |
|
|
$ |
40,611 |
|
|
$ |
332,699 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
See accompanying notes.
F-7
Amicus Therapeutics, Inc.
(a development stage company)
Notes To Financial Statements
|
|
1. |
Description of Business |
|
|
|
Corporate Information, Status of Operations, and
Management Plans |
Amicus Therapeutics, Inc. (the Company) was
incorporated on February 4, 2002 in Delaware for the
purpose of creating a premier drug development company at the
forefront of therapy for human genetic diseases initially based
on intellectual property in-licensed from Mount Sinai School of
Medicine. The Companys activities since inception have
consisted principally of raising capital, establishing
facilities, and performing research and development.
Accordingly, the Company is considered to be in the development
stage.
The Company has an accumulated deficit of $44.7 million at
March 31, 2006 and anticipates incurring losses through the
year 2006. The Company has not yet generated revenues and has
been able to fund its operating losses to date through the sale
of its redeemable convertible preferred stock, issuance of
convertible notes, and other financing arrangements. The
Companys management intends to raise additional funds
through the issuance of equity securities. If adequate funds are
not available, the Company may have to substantially reduce or
eliminate expenditures for the development of its products or
cease operations.
In April 2006, the Company received cash amounting to
approximately $27.5 million from the issuance of its second
tranche series C redeemable convertible preferred stock.
Management believes that the Companys current cash
position and the additional funds received in April 2006 are
sufficient to cover its cash flow requirements for 2006.
|
|
2. |
Summary of Significant Accounting Policies |
|
|
|
Unaudited Interim Financial Statements |
The financial statements as of March 31, 2006 and for the
three months ended March 31, 2005 and 2006 have been
prepared by the Company without an audit. All disclosures as of
March 31, 2006 and for the three months ended
March 31, 2005 and 2006, presented in the notes to the
financial statements are unaudited. In the opinion of
management, all adjustments (which include only normal recurring
adjustments) considered necessary to present fairly the
financial condition as of March 31, 2006 and results of
operations and cash flows for the three months ended
March 31, 2005 and 2006, have been made. The results of
operations for the three months ended March 31, 2006 are
not necessarily indicative of the results that may be expected
for the full year ended December 31, 2006.
|
|
|
Unaudited Pro Forma Information |
The unaudited pro forma balance sheet data as of March 31,
2006 gives effect to the Companys issuance on
April 17, 2006 of 21,825,131 shares of series C
redeemable convertible preferred stock, the automatic or
voluntary exercise of warrants outstanding as of March 31,
2006 to purchase 555,003 shares of series B redeemable
convertible preferred stock, and the automatic conversion of all
outstanding shares of the Companys series A, B, and C
redeemable convertible preferred stock into an aggregate of
84,009,190 shares of common stock upon completion of the
Companys initial public offering.
Pro forma net loss per share is computed using the
weighted-average number of common shares outstanding, including
the pro forma effects of the items in the foregoing paragraph
effective upon the assumed closing of the Companys
proposed initial public offering, as if they had occurred at the
beginning of the period.
F-8
Amicus Therapeutics, Inc.
(a development stage company)
Notes To Financial Statements (Continued)
The preparation of financial statements in conformity with
U.S. generally accepted accounting principles requires
management to make estimates and assumptions that affect the
reported amounts of assets and liabilities, the disclosure of
contingent assets and liabilities at the date of the financial
statements, and the reported amounts of revenues and expenses
during the reporting periods. Actual results could differ from
those estimates.
The Company considers all highly liquid investments purchased
with a maturity of three months or less at the date of
acquisition, to be cash equivalents.
|
|
|
Investment in Marketable Securities |
Marketable securities consist of fixed income investments with a
maturity of greater than three months and other highly liquid
investments that can be readily purchased or sold using
established markets. In accordance with Financial Accounting
Standards Board (FASB) Statement of Financial
Accounting Standards (SFAS) No. 115,
Accounting for Certain Investments in Debt and Equity
Securities, these investments are classified as
available-for-sale and are reported at fair value on the
Companys balance sheet. Unrealized holding gains and
losses are reported within accumulated other comprehensive
income as a separate component of stockholders deficiency.
If a decline in the fair value of a marketable security below
the Companys cost basis is determined to be other than
temporary, such marketable security is written down to its
estimated fair value as a new cost basis and the amount of the
write-down is included in earnings as an impairment charge. No
other than temporary impairment charges have been recorded in
any of the years presented herein.
Property and equipment are stated at cost, less accumulated
depreciation and amortization. Depreciation is calculated over
the estimated useful lives of the respective assets, which range
from three to six years, or the lesser of the related initial
term of the lease or useful life for leasehold improvements.
Assets under capital leases are amortized over the terms of the
related leases or their estimated useful lives, whichever is
shorter.
The initial cost of property and equipment consists of its
purchase price and any directly attributable costs of bringing
the asset to its working condition and location for its intended
use. Expenditures incurred after the fixed assets have been put
into operation, such as repairs and maintenance, are charged to
income in the period in which the costs are incurred. Major
replacements, improvements and additions are capitalized in
accordance with Company policy.
Costs directly attributable to the Companys offering of
its equity securities have been deferred and capitalized as part
of other non-current assets. These costs will be charged against
the proceeds of the offering once completed. The amount deferred
as of March 31, 2006 was not significant.
|
|
|
Impairment of Long-Lived Assets |
The Company performs a review of long-lived assets for
impairment when events or changes in circumstances indicate the
carrying value of such assets may not be recoverable. If an
indication of impairment is present, the Company compares the
estimated undiscounted future cash flows to be
F-9
Amicus Therapeutics, Inc.
(a development stage company)
Notes To Financial Statements (Continued)
generated by the asset to its carrying amount. If the
undiscounted future cash flows are less than the carrying amount
of the asset, the Company records an impairment loss equal to
the excess of the assets carrying amount over its fair
value. The fair value is determined based on valuation
techniques such as a comparison to fair values of similar assets
or using a discounted cash flow analysis. The Company reported
an impairment charge of $1,029,696 during 2003 related to
impaired capitalized leasehold improvements. There were no other
impairment charges recognized during the years ended
December 31, 2004 and 2005, or the three months ended
March 31, 2005 and 2006.
|
|
|
Redeemable Convertible Preferred Stock |
The carrying value of redeemable convertible preferred stock is
increased by periodic accretions so that the carrying amount
will equal the redemption amount at the earliest redemption
date. These increases are reflected through charges to
additional paid-in capital since the Company does not have
retained earnings.
|
|
|
Research and Development Costs |
Research and development costs are expensed as incurred.
Research and development expense consists primarily of costs
related to personnel, including salaries and other
personnel-related expenses, consulting fees and the cost of
facilities and support services used in drug development. Assets
acquired that are used for research and development and have no
future alternative use are expensed as in-process research and
development.
|
|
|
Concentration of Credit Risk |
The Companys financial instruments that are exposed to
concentration of credit risk consist primarily of cash and cash
equivalents and marketable securities. The Company maintains its
cash and cash equivalents in bank accounts, which, at times,
exceed federally insured limits. The Company invests its
marketable securities in high-quality commercial financial
instruments. The Company has not recognized any losses from
credit risks on such accounts during any of the periods
presented. The Company believes it is not exposed to significant
credit risk on cash and cash equivalents or its marketable
securities.
|
|
|
Fair Value of Financial Instruments |
SFAS No. 107, Disclosures about Fair Value of
Financial Instruments, requires disclosures of fair value
information about financial instruments, whether or not
recognized in the balance sheet, for which it is practicable to
estimate that value. Due to the short-term nature, the carrying
amounts reported in the financial statements approximate the
fair value for cash and cash equivalents, accounts payable and
accrued expenses. The estimated fair value of the Companys
redeemable convertible preferred stock at March 31, 2006 is
approximately $77.7 million based on the August 2005
series C redeemable convertible preferred stock price of
$1.26 per share. The redeemable convertible preferred stock
will be converted into common stock of the Company upon
consummation of a qualified initial public offering.
The Company accounts for income taxes under the liability
method. Under this method deferred income tax liabilities and
assets are determined based on the difference between the
financial statement carrying amounts and tax basis of assets and
liabilities and for operating losses and tax credit
carryforwards, using enacted tax rates in effect in the years in
which the differences are expected to reverse. A valuation
allowance is recorded if it is more likely than not
that a portion or all of a deferred tax asset will not be
realized.
F-10
Amicus Therapeutics, Inc.
(a development stage company)
Notes To Financial Statements (Continued)
SFAS No. 130, Reporting Comprehensive Income,
requires components of other comprehensive loss, including
unrealized gains and losses on available-for-sale securities, to
be included as part of total comprehensive loss. The components
of comprehensive loss are included in the statements of changes
in stockholders deficiency.
At December 31, 2005 and March 31, 2006, the Company
has one stock-based employee compensation plan, which is
described more fully in Note 7. Prior to December 31,
2005, the Company accounted for those plans under the
recognition and measurement provisions of Accounting Principles
Board Opinion (APB) No. 25, Accounting for
Stock Issued to Employees, and related Interpretations, as
permitted by FASB Statement No. 123 (SFAS
No. 123), Accounting for Stock-Based
Compensation. Stock-based employee compensation cost was
recognized in the Statements of Operations for the years ended
December 31, 2003, 2004, and 2005 and for the three month
period ended March 31, 2005 to the extent the options
granted under the plan had an exercise price that was less than
the market value of the underlying common stock on the date of
grant. Effective January 1, 2006, the company adopted the
fair value recognition provisions of FASB Statement
No. 123(R), Share-Based Payment (SFAS
No. 123(R)), using the prospective transition method.
Under that transition method, compensation cost is recognized
for all share-based payments granted subsequent to
January 1, 2006, based on the grant-date fair value
estimated in accordance with the provisions of SFAS
No. 123(R). Results for prior periods have not been
restated. As a result of adopting SFAS No. 123(R) on
January 1, 2006, the Companys net loss is larger than
had it continued to account for share-based compensation under
Opinion 25.
Prior to the adoption of SFAS No. 123(R), the Company
presented its unamortized portion of deferred compensation cost
for non-vested stock options in the statement of changes in
stockholders deficiency with a corresponding credit to
additional paid in capital. Upon the adoption of SFAS
No. 123(R), these amounts were offset against each other.
Under SFAS No. 123(R), an equity instrument is not
considered to be issued until the instrument vests. As a result,
compensation cost is recognized over the requisite service
period with an offsetting credit to additional paid in capital,
and the deferred compensation balance of $2.5 million at
January 1, 2006 was net against additional paid in capital
during the first quarter of 2006.
F-11
Amicus Therapeutics, Inc.
(a development stage company)
Notes To Financial Statements (Continued)
The following table illustrates the effect on net loss and net
loss per share if the company had applied the minimum value
recognition provisions of SFAS No. 123 to options granted
under the companys stock option plans in all periods
presented prior to adoption of SFAS No. 123(R). For
purposes of this pro forma disclosure, the value of the options
is estimated using a minimum value option-pricing formula and
amortized to expense over the options vesting periods.
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Three Months | |
|
|
Years Ended December 31, | |
|
Ended | |
|
|
| |
|
March 31, | |
|
|
2003 | |
|
2004 | |
|
2005 | |
|
2005 | |
|
|
| |
|
| |
|
| |
|
| |
Numerator
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Net loss attributable to common stockholders, as reported
|
|
$ |
(6,784,589 |
) |
|
$ |
(8,930,924 |
) |
|
$ |
(19,830,557 |
) |
|
$ |
(3,441,673 |
) |
Add: Non-cash employee compensation
|
|
|
70,340 |
|
|
|
59,842 |
|
|
|
364,551 |
|
|
|
91,138 |
|
Less: Total stock-based employee compensation expense determined
under the minimum value method for all awards
|
|
|
(76,207 |
) |
|
|
(74,499 |
) |
|
|
(437,296 |
) |
|
|
(109,324 |
) |
|
|
|
|
|
|
|
|
|
|
|
|
|
Pro forma net loss attributable to
common stockholders
|
|
$ |
(6,790,456 |
) |
|
$ |
(8,945,581 |
) |
|
$ |
(19,903,302 |
) |
|
$ |
(3,459,859 |
) |
|
|
|
|
|
|
|
|
|
|
|
|
|
Net loss attributable to common stockholders per common share:
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Basic and fully diluted:
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
As reported
|
|
$ |
(2.94 |
) |
|
$ |
(3.87 |
) |
|
$ |
(6.45 |
) |
|
$ |
(1.49 |
) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Pro forma
|
|
$ |
(2.94 |
) |
|
$ |
(3.88 |
) |
|
$ |
(6.47 |
) |
|
$ |
(1.49 |
) |
|
|
|
|
|
|
|
|
|
|
|
|
|
Pro forma information regarding net loss is required by SFAS
No. 123 and has been determined as if the Company has been
accounting for its stock options awards under the minimum value
option pricing method as of that statement. The value of these
options was estimated at the date of grant using a minimum value
method with the following weighted-average assumptions:
Employee Stock Options
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Years Ended December 31, | |
|
Three Months | |
|
|
| |
|
Ended | |
|
|
2003 | |
|
2004 | |
|
2005 | |
|
March 31, 2005 | |
|
|
| |
|
| |
|
| |
|
| |
Expected term (in years)
|
|
|
6.5 |
|
|
|
6.5 |
|
|
|
6.0 |
|
|
|
6.0 |
|
Risk-free interest rate
|
|
|
4.26 |
% |
|
|
3.92 |
% |
|
|
4.15 |
% |
|
|
4.15 |
% |
Dividend yield
|
|
|
0.00 |
% |
|
|
0.00 |
% |
|
|
0.00 |
% |
|
|
0.00 |
% |
Upon adoption of SFAS No. 123(R), the Company selected the
Black-Scholes option pricing model as the most appropriate model
for determining the estimated fair value for stock-based awards.
The fair value is then amortized on a straight-line basis over
the requisite service periods of the awards, which is generally
the vesting period. Use of a valuation model requires management
to make certain assumptions with respect to selected model
inputs. Expected volatility was calculated based on a blended
weighted average of historical information of the Companys
stock and the weighted average of historical information of
similar public entities for which historical information was
available. The Company will continue to use a weighted average
approach using its own historical volatility and other similar
public entity volatility information until historical volatility
of the Company is relevant to measure expected
F-12
Amicus Therapeutics, Inc.
(a development stage company)
Notes To Financial Statements (Continued)
volatility for future option grants. The average expected life
was determined according to the SEC shortcut approach as
described in Staff Accounting Bulletin (SAB)
No. 107, Disclosure about Fair Value of Financial
Instruments, which is the mid-point between the vesting date
and the end of the contractual term. The risk-free interest rate
is based on U.S. Treasury zero-coupon issues with a
remaining term equal to the expected life assumed at the date of
grant. Forfeitures are estimated based on voluntary termination
behavior, as well as a historical analysis of actual option
forfeitures. The weighted-average assumptions used in the
Black-Scholes option pricing model are as follows:
|
|
|
|
|
|
|
Three Months Ended | |
|
|
March 31, 2006 | |
|
|
| |
Expected stock price volatility
|
|
|
72.70 |
% |
Risk free interest rate
|
|
|
4.59 |
% |
Expected life of options (years)
|
|
|
6.25 |
|
Expected annual dividend per share
|
|
$ |
0.00 |
|
The weighted-average grant date fair value for options granted
during the three months ended March 31, 2006 was
approximately $1.52 per share.
During the three months ended March 31, 2006, the Company
recorded compensation expense of approximately $152,000
($0.04 per basic and diluted share) related to the
expensing of the Companys options under SFAS
No. 123(R) during the quarter. The compensation expense had
no impact on the Companys cash flows from operations and
financing activities. The total compensation cost related to
non-vested stock option awards not yet recognized as of
March 31, 2006 was approximately $7.1 million. This
expense will be recorded on a straight basis over approximately
4 years.
|
|
|
Beneficial Conversion Feature |
When the Company issues debt or equity which is convertible into
common stock at a discount from the common stock fair value at
the date the debt or equity is issued, a beneficial conversion
feature for the difference between the closing price and the
conversion price multiplied by the number of shares issuable
upon conversion is recognized. The beneficial conversion feature
is presented as a discount to the related debt or a deemed
dividend to the related equity, with an offsetting amount
increasing additional paid in capital. The Company recorded a
beneficial conversion charge for its bridge loan financing of
$135,000 which was initially recorded as debt discount and
amortized to interest expense through May 2004. The Company
expects to record a beneficial conversion charge (deemed
dividend) during the second quarter of 2006 of approximately
$19.4 million related to the issuance of certain shares of
series C redeemable convertible preferred stock. The
estimated fair value of the common stock was approximately
$2.15 per share at the measurement date based on estimates
of the projected initial public offering price of the
Companys common stock which is planned in mid-2006.
The accompanying financial statements for the quarter ended
March 31, 2006 were restated as the Company determined that
its deemed dividend related to the Series C redeemable
convertible preferred stock should have been recorded as a
second quarter event instead of a first quarter event. The
deemed dividend was initially recorded in the financial
statements as of the measurement date instead of the date the
shares were issued, which occurred in April 2006. The change had
no impact on net loss for the three months ended March 31,
2006 and decreased net loss attributable to common shareholders
for the three months ended March 31, 2006 by
$19.4 million; resulting in an increase to basic and
diluted earnings per share attributable to common stockholders
of $4.60, from ($6.41) to ($1.81). This change was confined to
the first quarter of 2006 only and had no impact on the related
balance sheet accounts and statement of
F-13
Amicus Therapeutics, Inc.
(a development stage company)
Notes To Financial Statements (Continued)
cash flows. The statement of changes in stockholders deficiency
were revised to remove the beneficial conversion charges,
however, the net impact was zero.
The effects of the restatement on the financial statement items
were as follows:
|
|
|
|
|
|
|
|
|
|
|
Previously Reported | |
|
|
|
|
Three Months | |
|
As Restated Three | |
|
|
Ended March 31, | |
|
Months Ended | |
|
|
2006 | |
|
March 31, 2006 | |
Statement of Operations |
|
| |
|
| |
Net loss attributable to common stockholders
|
|
$ |
(27,096,407 |
) |
|
$ |
(7,672,040 |
) |
Net loss attributable to common stockholders per common share
basic and diluted
|
|
$ |
(6.41 |
) |
|
$ |
(1.81 |
) |
|
|
|
Basic and Diluted Net Loss Attributable to Common
Stockholders per Common Share |
The Company calculates net loss per share in accordance with
SFAS No. 128, Earnings Per Share. The Company
has determined that its series A, B, and C redeemable
convertible preferred stock represent participating securities
in accordance with Emerging Issue Task Force
(EITF) 03-6
Participating Securities and the TwoClass Method under
FASB Statement No. 128. However, since the Company
operates at a loss, and losses are not allocated to the
redeemable convertible preferred stock, the two class method
does not affect the Companys calculation of earnings per
share. The Company has a net loss for all periods presented;
accordingly, the inclusion of common stock options and warrants
would be anti-dilutive. Therefore, the weighted average shares
used to calculate both basic and diluted earnings per share are
the same.
F-14
Amicus Therapeutics, Inc.
(a development stage company)
Notes To Financial Statements (Continued)
The following table provides a reconciliation of the numerator
and denominator used in computing basic and diluted net loss
attributable to common stockholders per common share and pro
forma net loss attributable to common stockholders per common
share:
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Three Months Ended | |
|
|
Years Ended December 31, | |
|
March 31, | |
|
|
| |
|
| |
|
|
2003 | |
|
2004 | |
|
2005 | |
|
2005 | |
|
2006 | |
|
|
| |
|
| |
|
| |
|
| |
|
| |
|
|
|
|
|
|
|
|
(unaudited) | |
|
(unaudited) | |
|
|
|
|
|
|
|
|
|
|
(Restated) | |
Historical
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Numerator:
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Net loss
|
|
$ |
(6,767,696 |
) |
|
$ |
(8,805,191 |
) |
|
$ |
(19,691,815 |
) |
|
$ |
(3,409,950 |
) |
|
$ |
(7,631,429 |
) |
|
Accretion of series B redeemable convertible preferred stock
|
|
|
(16,893 |
) |
|
|
(125,733 |
) |
|
|
(138,742 |
) |
|
|
(31,723 |
) |
|
|
(40,611 |
) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Net loss attributable to common stockholders
|
|
$ |
(6,784,589 |
) |
|
$ |
(8,930,924 |
) |
|
$ |
(19,830,557 |
) |
|
$ |
(3,441,673 |
) |
|
$ |
(7,672,040 |
) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Denominator:
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Weighted average common shares outstandingbasic and diluted
|
|
|
2,306,541 |
|
|
|
2,306,541 |
|
|
|
3,076,649 |
|
|
|
2,314,804 |
|
|
|
4,228,564 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Unaudited Pro forma
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Numerator:
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Net loss
|
|
|
|
|
|
|
|
|
|
$ |
(19,691,815 |
) |
|
|
|
|
|
$ |
(7,631,429 |
) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Denominator:
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Pro forma weighted average common shares outstandingbasic
and diluted
|
|
|
|
|
|
|
|
|
|
|
87,085,839 |
|
|
|
|
|
|
|
88,237,754 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Dilutive common stock equivalents would include the dilutive
effect of convertible securities, common stock options and
warrants for common stock equivalents. Potentially dilutive
common stock equivalents totaled approximately 5,496,110,
28,749,798 and 70,948,031 for the years ended December 31,
2003, 2004 and 2005, respectively and 47,059,954 and 76,243,031
for the three months ended March 31, 2005 and 2006,
respectively. Potentially dilutive common stock equivalents were
excluded from the diluted earnings per share denominator for all
periods because of their anti-dilutive effect.
Recent Accounting
Pronouncements
In February 2006, FASB issued SFAS No. 155, Accounting
for Certain Hybrid Instruments. SFAS 155 allows
financial instruments that have embedded derivatives to be
accounted for as a whole (eliminating the need to bifurcate the
derivative from its host) if the holder elects to account for
the whole instrument on a fair value basis. This statement is
effective for all financial instruments acquired or issued after
the beginning of an entitys first fiscal year that begins
after September 15, 2006. The Company believes the adoption
of SFAS No. 155 will not have a material impact on its
financial statements.
In May 2005, FASB issued SFAS No. 154, Accounting
Changes and Error Corrections (SFAS
No. 154), a replacement of APB No. 20,
Accounting Changes and SFAS No. 3, Reporting
Accounting Changes in Interim Financial Statements. SFAS
No. 154 applies to all voluntary changes in accounting
principle and changes the requirements for accounting for and
reporting of a change in accounting
F-15
Amicus Therapeutics, Inc.
(a development stage company)
Notes To Financial Statements (Continued)
principle. This statement establishes that, unless
impracticable, retrospective application is the required method
for reporting a change in accounting principle in the absence of
explicit transition requirements specific to the newly adopted
accounting principle. It also requires the reporting of an error
correction which involves adjustments to previously issued
financial statements similar to those generally applicable to
reporting an accounting change retrospectively.
SFAS No. 154 is effective for accounting changes and
corrections of errors made in fiscal years beginning after
December 15, 2005.
Segment Information
The Company currently operates in one business segment focusing
on the development and commercialization of small molecule,
orally administered therapies to treat a range of human genetic
diseases. The Company is not organized by market and is managed
and operated as one business. A single management team reports
to the chief operating decision maker who comprehensively
manages the entire business. The Company does not operate any
separate lines of business or separate business entities with
respect to its products. Accordingly, the Company does not
accumulate discrete financial information with respect to
separate service lines and does not have separately reportable
segments as defined by SFAS No. 131, Disclosure
About Segments of an Enterprise and Related Information.
|
|
3. |
Investments in Marketable Securities |
The following is a summary of available for sale securities held
by the Company:
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Gross Unrealized |
|
Gross Unrealized | |
|
Fair | |
|
|
Cost | |
|
Gains |
|
Losses | |
|
Value | |
|
|
| |
|
|
|
| |
|
| |
March 31, 2006 (unaudited)
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Corporate Bonds
|
|
$ |
9,258,812 |
|
|
$ |
|
|
|
$ |
(5,320 |
) |
|
$ |
9,253,492 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
December 31, 2005
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Corporate Bonds
|
|
$ |
17,985,235 |
|
|
$ |
|
|
|
$ |
(16,139 |
) |
|
$ |
17,969,096 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
December 31, 2004
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Corporate Bonds
|
|
$ |
4,088,008 |
|
|
$ |
|
|
|
$ |
(9,083 |
) |
|
$ |
4,078,925 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
The Companys available for sale investments have the
following maturities at:
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
December 31, | |
|
|
|
|
| |
|
March 31, | |
|
|
2004 | |
|
2005 | |
|
2006 | |
|
|
| |
|
| |
|
| |
|
|
|
|
|
|
(unaudited) | |
Due in one year or less
|
|
|
$4,078,925 |
|
|
|
$17,969,096 |
|
|
$ |
9,253,492 |
|
Unrealized gains and losses are reported as a component of
accumulated other comprehensive loss in stockholders
deficiency. For the years ended December 31, 2003, 2004,
2005, and for the three months ended March 31, 2005 and
2006, realized losses were $0, $704, $1,228, $1,228, and $0,
respectively. The cost of securities sold is based on specific
identification method.
Unrealized loss positions for which other than temporary
impairments have not been recognized at December 31, 2004,
2005, and March 31, 2006 are summarized as follows:
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
December 31, | |
|
|
|
|
| |
|
March 31, | |
|
|
2004 | |
|
2005 | |
|
2006 | |
|
|
| |
|
| |
|
| |
|
|
|
|
|
|
(unaudited) | |
Less than 12 months
|
|
|
$(9,083) |
|
|
|
$(16,139) |
|
|
$ |
(5,320 |
) |
|
|
|
|
|
|
|
|
|
|
F-16
Amicus Therapeutics, Inc.
(a development stage company)
Notes To Financial Statements (Continued)
Unrealized losses in the Companys portfolio relate to
fixed income debt securities. For these securities, the
unrealized losses are due to increases in interest rates and not
changes in credit risk. The Company has concluded that the
unrealized losses in its marketable securities are not
other-than-temporary as the Company has the ability to hold the
securities to maturity or a planned forecasted recovery.
|
|
4. |
Property and Equipment |
Property and equipment consist of the following:
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
December 31, | |
|
|
|
|
| |
|
March 31, | |
|
|
2004 | |
|
2005 | |
|
2006 | |
|
|
| |
|
| |
|
| |
|
|
|
|
|
|
(unaudited) | |
Computer equipment
|
|
$ |
105,637 |
|
|
$ |
284,913 |
|
|
$ |
307,609 |
|
Computer software
|
|
|
|
|
|
|
15,921 |
|
|
|
80,143 |
|
Research equipment
|
|
|
737,672 |
|
|
|
1,790,873 |
|
|
|
1,929,599 |
|
Furniture and fixtures
|
|
|
|
|
|
|
251,703 |
|
|
|
291,514 |
|
Leasehold improvements
|
|
|
|
|
|
|
109,345 |
|
|
|
1,891,819 |
|
Construction in progress
|
|
|
|
|
|
|
1,430,996 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
843,309 |
|
|
|
3,883,751 |
|
|
|
4,500,684 |
|
Less accumulated depreciation and amortization
|
|
|
(302,032 |
) |
|
|
(604,864 |
) |
|
|
(804,088 |
) |
|
|
|
|
|
|
|
|
|
|
|
|
$ |
541,277 |
|
|
$ |
3,278,887 |
|
|
$ |
3,696,596 |
|
|
|
|
|
|
|
|
|
|
|
In 2003, the Company capitalized costs related to an additional
facility that it had leased in Cranbury, New Jersey. However,
because the Company was not able to raise the necessary capital
it required to continue the construction of the leasehold
improvements in a timely manner, it decided to cease activities
related to the construction. As a result, the Company expensed
all capitalized leasehold improvements amounting to $1,029,696
in 2003.
Included in property and equipment is costs capitalized pursuant
to a capital lease obligation of $0, $1,146,007, and $3,247,858
at December 31, 2004, 2005, and March 31, 2006.
Depreciation and amortization expense was $0, $0 and $137,504
for the years ended December 31, 2003, 2004, and 2005,
respectively; and $0 and $152,097 for the three months ended
March 31, 2005 and 2006, respectively.
Accrued expenses consist of the following:
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
December 31, | |
|
|
|
|
| |
|
March 31, | |
|
|
2004 | |
|
2005 | |
|
2006 | |
|
|
| |
|
| |
|
| |
|
|
|
|
|
|
(unaudited) | |
Accrued construction costs
|
|
$ |
|
|
|
$ |
592,594 |
|
|
$ |
|
|
Accrued professional fees
|
|
|
85,930 |
|
|
|
312,244 |
|
|
|
57,593 |
|
Accrued contract manufacturing & contract research costs
|
|
|
|
|
|
|
53,163 |
|
|
|
1,796,713 |
|
Accrued compensation and benefits
|
|
|
|
|
|
|
14,719 |
|
|
|
230,185 |
|
Accrued facility costs
|
|
|
|
|
|
|
182,303 |
|
|
|
196,600 |
|
Accrued other
|
|
|
71,890 |
|
|
|
252,002 |
|
|
|
158,641 |
|
|
|
|
|
|
|
|
|
|
|
|
|
$ |
157,820 |
|
|
$ |
1,407,025 |
|
|
$ |
2,439,732 |
|
|
|
|
|
|
|
|
|
|
|
F-17
Amicus Therapeutics, Inc.
(a development stage company)
Notes To Financial Statements (Continued)
Redeemable Convertible
Preferred Stock
At March 31, 2006 the Company is authorized to issue
3,333,334 shares of series A redeemable convertible
preferred stock (Series A),
37,025,594 shares of series B redeemable convertible
preferred stock (Series B) and
43,650,262 shares of series C redeemable convertible
preferred stock (Series C). At
December 31, 2005 and March 31, 2006, the Company had
outstanding 3,333,334 shares, 36,470,591 shares, and
21,825,131 shares of Series A, B, and C, respectively.
Voting
Series A, Series B and Series C stockholders are
entitled to vote on substantially all matters based on the
number of votes equal to the number of shares of common stock
into which each share of preferred stock is convertible.
Dividends
Dividends are payable when, as and if declared by the board of
directors and are non-cumulative. Series A, Series B
and Series C stockholders shall be entitled to receive
dividends at the same rate as dividends paid with respect to the
common stock. Such preferred dividends will be determined by the
number of shares of common stock into which each share of
redeemable convertible preferred stock is convertible.
Conversion
Series A, Series B and Series C stockholders are
entitled, at any time, to cause their shares to be converted
into fully-paid and non-assessable shares of common stock on a
one-for-one basis. However, if there is a stock
dividend, stock split or a capital reorganization of the common
stock before conversion of preferred stock, the conversion
factor will be adjusted in accordance with the Companys
amended and restated certificate of incorporation. Additionally,
the Series A, Series B and Series C will convert
automatically immediately upon the closing of a firmly
underwritten public offering pursuant to an effective
registration statement under the Securities Act of 1933, as
amended, covering the offer and sale of common stock for the
account of the Company, which results in aggregate net proceeds
to the Company of at least $40,000,000 and a per share price of
at least $2.52 and the common stock is listed on a
U.S. national securities exchange or admitted for quotation
on the NASDAQ National Market.
Liquidation
In the event of any liquidation, dissolution or winding up of
the Company (including a merger or sale of all or substantially
all of the assets of the Company), either voluntary or
involuntary, the Series A, Series B and Series C
holders are entitled to receive, in preference to common stock,
an amount equal to $0.75 per share, $0.85 per share
and $1.26 per share, respectively, adjusted for any
combinations, splits, and other recapitalizations plus all
declared but unpaid dividends. For any remaining assets, the
Series A, Series B and Series C shareholders
shall participate with the holders of common stock on an
as-converted basis.
Redemption Rights
The holders of the redeemable convertible preferred stock are
entitled to require the Company to redeem all shares of the
redeemable convertible preferred stock at any time after the
fifth anniversary of the Series C original issue date. The
redeemable convertible preferred stock may be redeemed at an
F-18
Amicus Therapeutics, Inc.
(a development stage company)
Notes To Financial Statements (Continued)
amount equal to the liquidation preference upon receipt by the
Company of a request from the holders of at least 60% of the
then outstanding shares of Series C that the redeemable
convertible preferred stock be redeemed.
As of December 31, 2004, 2005, and March 31, 2006,
Series A, Series B and Series C are recorded at
its stated values (estimated fair value of $0.75 per share,
$0.85 per share and $1.26, respectively, less issuance
costs, plus accrued but unpaid dividends, if any, and accretion
adjustments).
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Series A | |
|
Series B | |
|
Series C | |
|
|
| |
|
| |
|
| |
|
|
Shares | |
|
Amount | |
|
Shares | |
|
Amount | |
|
Shares | |
|
Amount | |
|
|
| |
|
| |
|
| |
|
| |
|
| |
|
| |
Balance at February 4, 2002 (inception)
|
|
|
|
|
|
$ |
|
|
|
|
|
|
|
$ |
|
|
|
|
|
|
|
$ |
|
|
|
Issuance of Series A at $0.75 per share
|
|
|
3,333,334 |
|
|
|
2,500,000 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Issuance costs
|
|
|
|
|
|
|
(95,185 |
) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Accretion to redemption value
|
|
|
|
|
|
|
10,720 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Balance at December 31, 2002
|
|
|
3,333,334 |
|
|
|
2,415,535 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Accretion to redemption value
|
|
|
|
|
|
|
16,893 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Balance at December 31, 2003
|
|
|
3,333,334 |
|
|
|
2,432,428 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Issuance of Series B at $0.85 per share
|
|
|
|
|
|
|
|
|
|
|
21,176,472 |
|
|
|
18,000,000 |
|
|
|
|
|
|
|
|
|
|
Issuance cost
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
(122,402 |
) |
|
|
|
|
|
|
|
|
|
Issuance of warrants with Series B
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
(421,802 |
) |
|
|
|
|
|
|
|
|
|
Accretion to redemption value
|
|
|
|
|
|
|
16,893 |
|
|
|
|
|
|
|
108,840 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Balance at December 31, 2004
|
|
|
3,333,334 |
|
|
|
2,449,321 |
|
|
|
21,176,472 |
|
|
|
17,564,636 |
|
|
|
|
|
|
|
|
|
|
Issuance of Series B at $0.85 per share
|
|
|
|
|
|
|
|
|
|
|
15,294,119 |
|
|
|
13,000,000 |
|
|
|
|
|
|
|
|
|
|
Issuance cost
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
(5,793 |
) |
|
|
|
|
|
|
|
|
|
Issuance of Series C at $1.26 per share
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
21,825,131 |
|
|
$ |
27,499,665 |
|
|
Issuance cost
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
(177,757 |
) |
|
Accretion to redemption value
|
|
|
|
|
|
|
16,893 |
|
|
|
|
|
|
|
109,999 |
|
|
|
|
|
|
|
11,850 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Balance at December 31, 2005
|
|
|
3,333,334 |
|
|
|
2,466,214 |
|
|
|
36,470,591 |
|
|
|
30,668,842 |
|
|
|
21,825,131 |
|
|
|
27,333,758 |
|
|
|
Accretion to redemption value
|
|
|
|
|
|
|
4,223 |
|
|
|
|
|
|
|
27,500 |
|
|
|
|
|
|
|
8,888 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Balance at March 31, 2006 (unaudited)
|
|
|
3,333,334 |
|
|
$ |
2,470,437 |
|
|
|
36,470,591 |
|
|
$ |
30,696,342 |
|
|
|
21,825,131 |
|
|
$ |
27,342,646 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
On April 15, 2002, the Company issued 666,668 shares
of Series A. On July 15, 2002, the Company issued
2,666,666 shares of Series A. Subsequently, on May 3,
2004, the Company amended its certificate of incorporation to
create the Series B and to set forth the rights and
preferences of such stock. On May 4, 2004, the Company
issued 21,176,472 shares of its Series B at
$0.85 per share and 555,003 warrants with a gross proceeds
(exclusive of proceeds from the potential future exercise of the
warrants)
F-19
Amicus Therapeutics, Inc.
(a development stage company)
Notes To Financial Statements (Continued)
amounting to approximately $18 million. The values of the
warrants were classified as a cost of issuance and are being
accreted through the earliest redemption date. In April of 2005,
the Company issued 15,294,119 shares of its Series B
and in August of 2005, 21,825,131 shares of Series C.
|
|
|
Bridge Loans for Series B redeemable convertible
preferred stock |
During 2003 and 2004, prior to the closing of the issuance of
the Series B, the Company issued a series of notes and
warrants in connection with short-term loans (Bridge
Loans) to help fund the Companys operations prior to
the closing of the Series B shares. The principal owed on
all of these notes issued in 2003 and in the first quarter 2004
totaled $5.5 million. $5.0 million of principal
outstanding under the Bridge Loans was converted into 5,882,353
Series B shares and $500,000 of principal outstanding under
the Bridge Loans was repaid, in each case in May 2004 at the
closing of the Series B financing. Approximately $193,000
in interest payable at such closing was waived by the holders.
The interest was recorded and charged to expense and credited to
additional paid-in
capital during 2004. The interest owed on such notes was waived
by the holders thereof and recorded as additional
paid-in capital.
In addition, the Company issued warrants for 999,999 shares
of common stock in connection with some of the Bridge Loans.
These warrants were valued using a Black-Scholes option pricing
model, amortized over the term of the notes, and charged to
interest expense. The total interest charge related to these
warrants was $210,000. In addition, the Company recognized a
beneficial conversion charge of $135,000 related to the
conversion feature in the Bridge Loans.
|
|
|
Common Stock and Stock Options |
As of March 31, 2006 the Company was authorized to issue
115,000,000 shares of common stock.
Dividends on common stock will be paid when, and if declared by
the board of directors. Each holder of common stock is entitled
to vote on all matters and is entitled to one vote for each
share held. The Company will, at all times, reserve and keep
available out of its authorized but unissued shares of common
stock sufficient shares to affect the conversion of the shares
of the redeemable convertible preferred stock and the exercise
of outstanding warrants and stock options.
|
|
|
Restricted Common Stock Issuances |
In connection with the formation of the Company, the Company
issued 1,742,000 shares of common stock to the Mount Sinai
School of Medicine of New York University in exchange for
exclusive license rights for certain intellectual property. The
value of the shares was accounted for as in-process research and
development (see Note 11).
In connection with a 2002 consulting arrangement, the Company
issued 562,041 shares of common stock in return for
services. The shares are fully vested and the Company recorded
$83,863 as compensation expense in the financial statements
during the year ended 2002.
During 2002, the Company issued 40,000 common stock warrants to
a vendor as part of a capital lease agreement. These warrants
were outstanding at December 31, 2003, 2004 and 2005. The
warrants have an exercise price of $0.75 per share
(adjusted for stock splits, stock dividends, etc.). The value of
the warrants was calculated using the
Black-Scholes option
pricing model and was capitalized as debt issuance cost and
amortized to interest expense over the term of the obligation.
The value of the warrants and total charge to interest expense
was not material for each of the years presented.
F-20
Amicus Therapeutics, Inc.
(a development stage company)
Notes To Financial Statements (Continued)
In 2003, the Company issued 999,999 common stock warrants
to certain investors in connection with its Bridge Loans. The
warrants had an exercise price of $0.075 per share
(adjusted for stock splits, stock dividends, etc.). The value of
the warrants of $210,000 was calculated using the Black-Scholes
option pricing model and was accounted for as debt discount and
amortized to interest expense over the term of the loans. These
same warrant shares were exercised in 2005. The total charge to
interest expense was $84,000 and $126,000 for the years ended
December 31, 2003 and 2004, respectively.
In 2004, the Company issued warrants to purchase
555,003 Series B shares to certain investors as part
of the Series B financing. These warrants were outstanding
at March 31, 2006. The warrants have an exercise price of
$0.85 per share (adjusted for stock splits, stock
dividends, etc.). The value of the warrants of $422,000 was
calculated using the
Black-Scholes option
pricing model and was capitalized as issuance cost and is being
accreted through the earliest redemption date. The total
accretion amount related to the warrants was $84,360, for each
of the years ended December 31, 2004, 2005, and $21,090 for
the three months ended March 31, 2006, respectively.
In April 2002, the Companys board of directors and
shareholders approved the Companys 2002 Stock Option Plan
(the 2002 Plan). The 2002 Plan provides for the
granting of options to purchase common stock in the Company to
employees, advisors and consultants at a price to be determined
by the Companys board of directors. The 2002 Plan is
intended to encourage ownership of stock by employees and
consultants of the Company and to provide additional incentives
for them to promote the success of the Companys business.
The Options may be incentive stock options
(ISOs) or non-statutory stock options
(NSOs). Under the provisions of the 2002 Plan,
no option will have a term in excess of 10 years.
The board of directors, or its committee, is responsible for
determining the individuals to be granted options, the number of
options each individual will receive, the option price per
share, and the exercise period of each option. Options granted
pursuant to the 2002 Plan generally vest over a four year term.
As of March 31, 2006, the Company reserved up to
17,500,000 shares for issuance under the 2002 Plan.
In establishing its estimates of fair value of its common stock,
the Company considered the guidance set forth in the AICPA
Practice Aid, Valuation of Privately-Held-Company Equity
Securities Issued as Compensation, and performed a
retrospective determination of the fair value of its common
stock utilizing a combination of valuation methods.
In December 2004, the FASB issued SFAS No. 123(R), which
requires compensation costs related to share-based transactions,
including employee share options, to be recognized in the
financial statements based on fair value. SFAS No. 123(R)
revises SFAS No. 123, as amended, Accounting for
Stock-Based Compensation, and supersedes APB Opinion
No. 25, Accounting for Stock Issued to Employees.
On January 1, 2006, the Company adopted SFAS
No. 123(R) using the prospective method. Under SFAS
No. 123(R), the Company has elected to recognize the
compensation cost of all share-based awards on a straight-line
basis over the vesting period of the award. Benefits of tax
deductions in excess of recognized compensation expense will be
reported as a financing cash flow, rather than an operating cash
flow as prescribed under the prior accounting rules.
Prior to January 1, 2006, the Company applied APB
No. 25 to account for its stock-based compensation plans.
Under APB No. 25 the Company used the minimum value method
of calculating unrecognized compensation expense for disclosure
purposes in the Financial Statements.
F-21
Amicus Therapeutics, Inc.
(a development stage company)
Notes To Financial Statements (Continued)
The following table summarizes information about stock options
outstanding:
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Options Outstanding | |
|
|
|
|
| |
|
|
|
|
|
|
Weighted- | |
|
|
|
|
|
|
Average | |
|
|
|
|
|
|
Grant Date | |
|
|
Shares | |
|
|
|
Option Price | |
|
Weighted- | |
|
Fair Value | |
|
|
Available | |
|
Number | |
|
Per Share | |
|
Average | |
|
or Calculated | |
|
|
For Grant | |
|
of Shares | |
|
Range | |
|
Exercise Price | |
|
Value | |
|
|
| |
|
| |
|
| |
|
| |
|
| |
Balance at February 4, 2002 (inception)
|
|
|
|
|
|
|
|
|
|
$ |
|
|
|
$ |
|
|
|
$ |
|
|
|
Shares authorized
|
|
|
1,212,611 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Options granted
|
|
|
(961,111 |
) |
|
|
961,111 |
|
|
|
0.01 0.08 |
|
|
|
0.02 |
|
|
|
0.23 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Balance at December 31, 2002
|
|
|
251,500 |
|
|
|
961,111 |
|
|
|
0.01 0.08 |
|
|
|
0.02 |
|
|
|
|
|
|
Shares authorized
|
|
|
100,000 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Options exercised
|
|
|
|
|
|
|
(2,500 |
) |
|
|
0.01 |
|
|
|
0.01 |
|
|
|
|
|
|
Options granted
|
|
|
(164,166 |
) |
|
|
164,166 |
|
|
|
0.08 |
|
|
|
0.08 |
|
|
|
0.23 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Balance at December 31, 2003
|
|
|
187,334 |
|
|
|
1,122,777 |
|
|
|
0.01 0.08 |
|
|
|
0.02 |
|
|
|
|
|
|
Shares authorized
|
|
|
6,401,492 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Options granted
|
|
|
(2,083,882 |
) |
|
|
2,083,882 |
|
|
|
0.08 0.09 |
|
|
|
0.08 |
|
|
|
0.12 |
|
|
Options forfeited
|
|
|
6,666 |
|
|
|
(6,666 |
) |
|
|
0.08 |
|
|
|
0.08 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Balance at December 31, 2004
|
|
|
4,511,610 |
|
|
|
3,199,993 |
|
|
|
0.01 0.09 |
|
|
|
0.06 |
|
|
|
|
|
|
Shares authorized
|
|
|
4,125,000 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Options granted
|
|
|
(7,576,785 |
) |
|
|
7,576,785 |
|
|
|
0.09 0.71 |
|
|
|
0.29 |
|
|
|
0.72 |
|
|
Options exercised
|
|
|
|
|
|
|
(728,691 |
) |
|
|
0.01 0.09 |
|
|
|
0.03 |
|
|
|
|
|
|
Options forfeited
|
|
|
769,112 |
|
|
|
(769,112 |
) |
|
|
0.01 0.09 |
|
|
|
0.06 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Balance at December 31, 2005
|
|
|
1,828,937 |
|
|
|
9,278,975 |
|
|
|
0.01 0.09 |
|
|
|
0.28 |
|
|
|
|
|
|
Shares authorized
|
|
|
5,660,897 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Options granted
|
|
|
|
|
|
|
5,895,000 |
|
|
|
0.71 |
|
|
|
0.71 |
|
|
|
1.84 |
|
|
Options exercised
|
|
|
|
|
|
|
(600,000 |
) |
|
|
0.09 |
|
|
|
0.09 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Balance at March 31, 2006 (unaudited)
|
|
|
7,489,834 |
|
|
|
14,573,975 |
|
|
$ |
0.01 $0.71 |
|
|
$ |
0.43 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Exercisable at December 31, 2003
|
|
|
|
|
|
|
116,731 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Exercisable at December 31, 2004
|
|
|
|
|
|
|
221,315 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Exercisable at December 31, 2005
|
|
|
|
|
|
|
930,397 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Vested and Exercisable at March 31, 2006
|
|
|
|
|
|
|
1,213,538 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
As of March 31, 2006, there was approximately
$9.8 million of total unrecognized compensation cost
related to non-vested share-based compensation arrangements
granted under the Plan. Of this amount approximately
$2.7 million is related to stock option grants issued prior
to January 1, 2006 and approximately $7.1 million is
related to 2006 grants. That cost is expected to be recognized
over a weighted-average period of 3.5 years and
3.92 years for 2005 and 2006, respectively.
As of March 31, 2006, outstanding options had aggregate
intrinsic value amounting to $8.6 million. Exercisable
options had aggregate intrinsic value amounting to $121,370. The
Company estimates that 13,845,276 of the options outstanding at
March 31, 2006 will vest over the remaining vesting periods
for such options.
F-22
Amicus Therapeutics, Inc.
(a development stage company)
Notes To Financial Statements (Continued)
Following is a summary of the status of stock options
outstanding at March 31, 2006:
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Outstanding Options | |
|
Exercisable Options | |
|
|
| |
|
| |
|
|
|
|
Weighted Average | |
|
Weighted Average | |
|
|
|
Weighted Average | |
|
|
|
|
Remaining | |
|
Exercise | |
|
|
|
Exercise | |
Exercise Price per Range |
|
Number | |
|
Contractual Life | |
|
Price | |
|
Number | |
|
Price | |
|
|
| |
|
| |
|
| |
|
| |
|
| |
$0.01
|
|
|
160,000 |
|
|
|
6.44 |
|
|
$ |
0.01 |
|
|
|
132,292 |
|
|
$ |
0.01 |
|
$0.075 - $0.085
|
|
|
6,012,475 |
|
|
|
8.85 |
|
|
|
0.08 |
|
|
|
1,081,246 |
|
|
|
0.08 |
|
$0.71
|
|
|
8,401,500 |
|
|
|
9.81 |
|
|
$ |
0.71 |
|
|
|
|
|
|
$ |
0.71 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
14,573,975 |
|
|
|
|
|
|
|
|
|
|
|
1,213,538 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
As of December 31, 2005 and March 31, 2006, the
average remaining contractual life of outstanding options was
approximately 9.0 and 9.4 years, respectively. The total
intrinsic value of options exercised during the years ended
December 31, 2003, 2004, and 2005, was $575, $0, and
$140,235, respectively, and $1,832 and $135,000 for the three
months ended March 31, 2005 and 2006. For the three months
ended March 31, 2006, the fair value of the options
granted, based upon the Black-Scholes calculation ranged from
$1.14 to $1.52 per share.
Options may be exercised in whole or in part for 100% of the
shares subject to vesting at any time after the date of grant.
Options generally vest 25% on the first year anniversary date of
grant plus an additional 1/48 for each month thereafter.
The Company performed a retrospective determination of the fair
value of the Companys common stock and granted stock
options with exercise prices as follows:
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Retrospective | |
|
|
|
|
|
|
|
|
Determination of | |
|
|
|
|
|
|
|
|
Fair Value of | |
|
|
|
|
Number of | |
|
Weighted Average | |
|
Underlying | |
|
|
2005 Grant Date |
|
Options Granted | |
|
Exercise Price | |
|
Stock | |
|
Intrinsic Value | |
|
|
| |
|
| |
|
| |
|
| |
January - May
|
|
|
3,037,037 |
|
|
$ |
0.09 |
|
|
$ |
0.31 |
|
|
$ |
0.22 |
|
June - July
|
|
|
1,768,748 |
|
|
|
0.09 |
|
|
|
0.77 |
|
|
|
0.68 |
|
August - September
|
|
|
315,500 |
|
|
|
0.22 |
|
|
|
0.95 |
|
|
|
0.73 |
|
October - November
|
|
|
2,351,000 |
|
|
|
0.71 |
|
|
|
1.14 |
|
|
|
0.43 |
|
December
|
|
|
104,500 |
|
|
$ |
0.71 |
|
|
$ |
1.44 |
|
|
$ |
0.73 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
7,576,785 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
The intrinsic value of options granted prior to 2005 is not
significant.
The Company recorded approximately $2.8 million in gross
deferred compensation expense and recognized compensation
expense of approximately $364,551 during the year ended
December 31, 2005 in connection with these stock grants
which is net of approximately $47,000 related to employee
terminations during the year ended December 31, 2005.
F-23
Amicus Therapeutics, Inc.
(a development stage company)
Notes To Financial Statements (Continued)
During the three months ended March 31, 2006 the Company
granted stock options with exercise prices as follows:
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Number of | |
|
|
|
Fair Value of | |
|
|
Grant Date |
|
Options Granted | |
|
Exercise Price | |
|
Underlying Stock | |
|
Intrinsic Value | |
|
|
| |
|
| |
|
| |
|
| |
January 2, 2006
|
|
|
17,000 |
|
|
$ |
0.71 |
|
|
$ |
1.44 |
|
|
$ |
0.73 |
|
January 12, 2006
|
|
|
5,000 |
|
|
|
0.71 |
|
|
|
1.44 |
|
|
|
0.73 |
|
February 6, 2006
|
|
|
5,000 |
|
|
|
0.71 |
|
|
|
1.44 |
|
|
|
0.73 |
|
February 9, 2006
|
|
|
23,000 |
|
|
|
0.71 |
|
|
|
1.44 |
|
|
|
0.73 |
|
February 13, 2006
|
|
|
7,500 |
|
|
|
0.71 |
|
|
|
1.44 |
|
|
|
0.73 |
|
February 22, 2006
|
|
|
35,000 |
|
|
|
0.71 |
|
|
|
1.44 |
|
|
|
0.73 |
|
February 28, 2006
|
|
|
5,752,500 |
|
|
|
0.71 |
|
|
|
1.84 |
|
|
|
1.13 |
|
March 27, 2006
|
|
|
50,000 |
|
|
$ |
0.71 |
|
|
$ |
1.84 |
|
|
$ |
1.13 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
5,895,000 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Compensation expense of $70,340, $59,842, $364,551, and $91,138
and $315,671 was recognized for the years ended 2003, 2004 and
2005 and for the three months ended March 31, 2005 and
2006, respectively.
The Company has a 401(k) plan (the Plan) covering
all eligible employees. The Plan allows for a discretionary
employer match. Through March 31, 2006 the Company has not
made any match on employee contributions.
On May 12, 2005, the Company entered into a Sublease
Agreement for its Corporate Office in Cranbury, NJ. The sublease
term will expire on February 28, 2012 or on such earlier
date upon mutual agreement of both parties. At March 31,
2006, aggregate annual future minimum lease payments under this
lease are as follows:
|
|
|
|
|
|
Years ending December 31:
|
|
|
|
|
|
2006
|
|
$ |
1,018,859 |
|
|
2007
|
|
|
1,451,463 |
|
|
2008
|
|
|
1,470,060 |
|
|
2009
|
|
|
1,470,060 |
|
|
2010
|
|
|
1,470,060 |
|
|
2011 and thereafter
|
|
|
1,715,070 |
|
|
|
|
|
|
|
$ |
8,595,572 |
|
|
|
|
|
Rent expense for the years ended December 31, 2003, 2004,
2005 and for the three months ended March 31, 2005 and 2006
were $110,000, $152,668, $971,687, $48,385, and $353,377,
respectively.
In August 2002, the Company entered into capital lease
agreements that provides for up to $1 million of equipment
financing through August 2004. The facility was increased to
$3 million in May of 2005 and
F-24
Amicus Therapeutics, Inc.
(a development stage company)
Notes To Financial Statements (Continued)
to $5 million in November 2005. These financing
arrangements include interest of approximately 9-12%, and lease
terms of 36 or 48 months. Eligible assets under the lease
lines include laboratory and scientific equipment, computer
hardware and software, general office equipment, furniture, and
tenant improvements.
At December 31, 2005 and March 31, 2006, the total
amount available to the Company under these agreements is
$4.0 million and $2.2 million, respectively.
The remaining future minimum payments due in 2006 for all
non-cancelable capital leases as of March 31, 2006 are as
follows:
|
|
|
|
|
|
Years ending December 31:
|
|
|
|
|
|
2006
|
|
$ |
842,013 |
|
|
2007
|
|
|
1,122,684 |
|
|
2008
|
|
|
1,056,522 |
|
|
2009
|
|
|
311,274 |
|
|
2010
|
|
|
8,317 |
|
|
|
|
|
|
|
|
3,340,810 |
|
|
Less payments for interest
|
|
|
(494,323 |
) |
|
|
|
|
|
Total principal obligation
|
|
|
2,846,487 |
|
|
Less short-term portion
|
|
|
(852,527 |
) |
|
|
|
|
|
Long-term portion
|
|
$ |
1,993,960 |
|
|
|
|
|
The capital lease obligation is secured by the related assets
financed by the leases.
F-25
Amicus Therapeutics, Inc.
(a development stage company)
Notes To Financial Statements (Continued)
Deferred income taxes reflect the net effect of temporary
difference between the carrying amounts of assets and
liabilities for financial reporting purposes and the amounts
used for income tax purposes. The significant components of the
deferred tax assets and liabilities are as follows:
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
December 31, | |
|
|
|
March 31, | |
|
|
2003 | |
|
2004 | |
|
2005 | |
|
2006 | |
|
|
| |
|
| |
|
| |
|
| |
|
|
|
|
|
|
|
|
(unaudited) | |
Current deferred tax asset
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Non-cash stock issue to consultants
|
|
$ |
|
|
|
$ |
|
|
|
$ |
63,747 |
|
|
$ |
91,951 |
|
|
Others
|
|
|
|
|
|
|
|
|
|
|
32,983 |
|
|
|
27,663 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
96,730 |
|
|
|
119,614 |
|
Non-current deferred tax assets
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Amortization
|
|
|
236,957 |
|
|
|
198,941 |
|
|
|
132,097 |
|
|
|
129,246 |
|
|
Research tax credit
|
|
|
62,513 |
|
|
|
730,903 |
|
|
|
1,344,230 |
|
|
|
1,572,505 |
|
|
Net operating loss carryforwards
|
|
|
3,143,749 |
|
|
|
6,387,827 |
|
|
|
14,463,790 |
|
|
|
17,374,472 |
|
|
Others
|
|
|
42,411 |
|
|
|
75,165 |
|
|
|
28,829 |
|
|
|
22,029 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Total deferred tax asset
|
|
|
3,485,630 |
|
|
|
7,392,836 |
|
|
|
16,065,676 |
|
|
|
19,217,866 |
|
Non-current deferred tax liability:
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Depreciation
|
|
|
(27,896 |
) |
|
|
(29,865 |
) |
|
|
(57,027 |
) |
|
|
(66,169 |
) |
|
|
|
|
|
|
|
|
|
|
|
|
|
Total net deferred tax asset
|
|
|
3,457,734 |
|
|
|
7,362,971 |
|
|
|
16,008,649 |
|
|
|
19,151,697 |
|
Less valuation allowance
|
|
|
(3,457,734 |
) |
|
|
(7,362,971 |
) |
|
|
(16,008,649 |
) |
|
|
(19,151,697 |
) |
|
|
|
|
|
|
|
|
|
|
|
|
|
Net deferred tax asset
|
|
$ |
|
|
|
$ |
|
|
|
$ |
|
|
|
$ |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
The Company records a valuation allowance for temporary
differences for which it is more likely than not that the
Company will not receive future tax benefits. At
December 31, 2003, 2004, 2005, and at March 31, 2006,
the Company recorded valuation allowances of $3,457,734,
$7,362,971, $16,008,649, and $19,151,697, respectively,
representing a change in the valuation allowance of $3,905,237
and $8,645,678 for the two previous fiscal year-ends and
$3,143,048 for the three months ended March 31, 2006, due
to the uncertainty regarding the realization of such deferred
tax assets, to offset the benefits of net operating losses
generated during those years.
Utilization of the net operating loss carryforwards and credits
may be subject to a substantial annual limitation due to the
ownership change limitations provided by the Internal Revenue
Code of 1986, as amended and similar state provisions. The
annual limitation may result in the expiration of net operating
losses and credits before utilization. The company has not
performed an analysis to determine if there has been a
change in ownership as defined by the Tax Reform Act
of 1986.
The Company recognized a tax benefit of approximately $83,000
and $612,000 in connection with the sale of New Jersey state net
operating loss during the years ended December 31, 2004 and
2005.
F-26
Amicus Therapeutics, Inc.
(a development stage company)
Notes To Financial Statements (Continued)
A reconciliation of the statutory tax rates and the effective
tax rates for the three years ended December 31, 2003,
2004, 2005, and for the three months ended March 31, 2005
and 2006 are as follows:
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Year Ended | |
|
Three Months Ended | |
|
|
December 31, | |
|
March 31, | |
|
|
| |
|
| |
|
|
2003 | |
|
2004 | |
|
2005 | |
|
2005 | |
|
2006 | |
|
|
| |
|
| |
|
| |
|
| |
|
| |
|
|
|
|
|
|
|
|
(unaudited) | |
|
(unaudited) | |
Statutory rate
|
|
|
(34 |
)% |
|
|
(34 |
)% |
|
|
(34 |
)% |
|
|
(34 |
)% |
|
|
(34 |
)% |
State taxes, net of federal benefit
|
|
|
(6 |
) |
|
|
(6 |
) |
|
|
(6 |
) |
|
|
(6 |
) |
|
|
(6 |
) |
Permanent adjustments
|
|
|
|
|
|
|
|
|
|
|
1 |
|
|
|
|
|
|
|
|
|
Non deductible interest
|
|
|
|
|
|
|
1 |
|
|
|
|
|
|
|
|
|
|
|
|
|
R&D Credit
|
|
|
|
|
|
|
(5 |
) |
|
|
(3 |
) |
|
|
(3 |
) |
|
|
(3 |
) |
Other
|
|
|
|
|
|
|
(2 |
) |
|
|
(1 |
) |
|
|
(4 |
) |
|
|
2 |
|
Benefit sale of NOL
|
|
|
|
|
|
|
1 |
|
|
|
(3 |
) |
|
|
|
|
|
|
|
|
Valuation allowance
|
|
|
40 |
|
|
|
44 |
|
|
|
43 |
|
|
|
47 |
|
|
|
41 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Net
|
|
|
|
% |
|
|
(1 |
)% |
|
|
(3 |
)% |
|
|
|
% |
|
|
|
% |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Income tax expense (benefit) consisted of the following
components:
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Three Months Ended |
|
|
Year Ended December 31, | |
|
March 31, |
|
|
| |
|
|
|
|
2003 |
|
2004 | |
|
2005 | |
|
2005 |
|
2006 |
|
|
|
|
| |
|
| |
|
|
|
|
|
|
|
|
|
|
|
|
(unaudited) |
|
(unaudited) |
Current payable (benefit):
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Federal
|
|
$ |
|
|
|
$ |
|
|
|
$ |
|
|
|
$ |
|
|
|
$ |
|
|
|
State
|
|
|
|
|
|
|
(83,015 |
) |
|
|
(611,797 |
) |
|
|
|
|
|
|
|
|
Deferred:
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Federal
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
State
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Income tax benefit
|
|
$ |
|
|
|
$ |
(83,015 |
) |
|
$ |
(611,797 |
) |
|
$ |
|
|
|
$ |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
The Company acquired rights to develop and commercialize its
product candidates through licenses granted by various parties.
The following summarizes the Companys material rights and
obligations under those licenses:
Mt. Sinai School of Medicine The Company acquired
exclusive worldwide patent rights to develop and commercialize
Amigal, AT2101 and AT2220 and other pharmacological chaperones
for the treatment of diseases caused by misfolded proteins
pursuant to a license agreement with Mt. Sinai School of
Medicine of New York University. Under this agreement, the
Company has no milestone or future payments other than royalties
on net sales. This agreement expires upon expiration of the last
of the licensed patent rights, which will be in 2019 if a
foreign patent is granted and 2018 otherwise.
University of Maryland, Baltimore County The
Company acquired exclusive U.S. patent rights to develop and
commercialize AT2101 for the treatment of Gaucher disease from
the University of Maryland, Baltimore County. Under this
agreement, the Company paid upfront and annual license fees of
$24,500, which were expensed as research and development
expense. Upon the satisfaction of certain milestones and
assuming successful development of AT2101, the Company could be
required to make up
F-27
Amicus Therapeutics, Inc.
(a development stage company)
Notes To Financial Statements (Continued)
to $175,000 in aggregate payments. The Company is also required
to pay royalties on net sales. In connection with this
agreement, the Company issued 1,742,000 shares of common
stock to Mt. Sinai School of Medicine in April 2002. This
agreement expires upon expiration of the last of the licensed
patent rights in 2015.
Novo Nordisk A/S The Company acquired exclusive
patent rights to develop and commercialize AT2101 for all human
indications. Under this agreement, to date the Company paid
$400,000 in license fees which were expensed as research and
development expense. Upon the satisfaction of certain milestones
and assuming successful development of AT2101, the Company could
be required to make up to $4,750,000 in aggregate payments. The
Company is also required to pay royalties on net sales. This
license will terminate in 2016.
Under our license agreements, if the Company owes royalties on
net sales for one of its products to more than one of the above
licensors, then we have the right to reduce the royalties owed
to one licensor for royalties paid to another. The amount of
royalties to be offset is generally limited in each license and
can vary under each agreement. For Amigal and AT2220, the
Company will owe royalties only to Mt. Sinai School of
Medicine and will owe no milestone payments. The Company expects
to pay royalties to all three licensors with respect to AT2101.
The Companys rights with respect to these agreements to
develop and commercialize Amigal, AT2101 and AT2220 may
terminate, in whole or in part, if the Company fails to meet
certain development or commercialization requirements or if the
Company does not meet its obligations to make royalty payments.
|
|
12 |
In-Process Research and Development |
During 2002, the Company acquired certain development rights to
intellectual property in the form of patent rights owned by
Mount Sinai School of Medicine of New York University in
exchange for 1,742,000 shares of common stock. The patent
rights cover compounds that improve protein folding and protein
stability.
The patent rights were reviewed to determine the stage of their
development, the achievement of technological feasibility, and
the technical milestones needed before commercialization is
possible. It was determined, as of the acquisition date, that
each patent had significant technical risk associated with
achieving the technological feasibility needed for FDA approval
and each patent has significant milestones to reach before
commercialization is reasonably certain. It was also determined
that all of the patents had no alternative future uses if they
were not successful. Accordingly, the license was classified as
in-process research and development and expensed immediately as
of the acquisition date and included in research and development
expense. The Company valued the acquired patents using fair
value techniques, as a quoted market price was not available.
The estimated fair value of the transfer at the date of the
transaction was approximately $418,000.
F-28
Amicus Therapeutics, Inc.
(a development stage company)
Notes To Financial Statements (Continued)
|
|
13. |
Selected Quarterly Financial Data (Unaudited) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Quarter Ended | |
|
|
| |
|
|
March 31 | |
|
June 30 | |
|
September 30 | |
|
December 31 | |
|
|
| |
|
| |
|
| |
|
| |
2004
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Net loss
|
|
$ |
(3,339,570 |
) |
|
$ |
(1,657,368 |
) |
|
$ |
(1,047,599 |
) |
|
$ |
(2,760,654 |
) |
Net loss attributable to common stockholders
|
|
$ |
(3,343,793 |
) |
|
$ |
(1,697,871 |
) |
|
$ |
(1,088,102 |
) |
|
$ |
(2,801,158 |
) |
Basic and diluted net loss per common share(1)
|
|
$ |
(1.45 |
) |
|
$ |
(0.74 |
) |
|
$ |
(0.47 |
) |
|
$ |
(1.21 |
) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Quarter Ended | |
|
|
| |
|
|
March 31 | |
|
June 30 | |
|
September 30 | |
|
December 31 | |
|
|
| |
|
| |
|
| |
|
| |
2005
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Net loss
|
|
$ |
(3,409,950 |
) |
|
$ |
(5,348,166 |
) |
|
$ |
(5,215,161 |
) |
|
$ |
(5,718,538 |
) |
Net loss attributable to common stockholders
|
|
$ |
(3,441,673 |
) |
|
$ |
(5,379,889 |
) |
|
$ |
(5,252,809 |
) |
|
$ |
(5,756,186 |
) |
Basic and diluted net loss per common share(1)
|
|
$ |
(1.49 |
) |
|
$ |
(2.13 |
) |
|
$ |
(1.54 |
) |
|
$ |
(1.43 |
) |
|
|
(1) |
Per common share amounts for the quarters and full years have
been calculated separately. Accordingly, quarterly amounts do
not add to the annual amounts because of differences on the
weighted-average common
shares outstanding during each period principally due to the
effect of the Companys issuing shares of its common stock
during the year. |
|
|
14. |
Subsequent Event (Unaudited) |
In April 2006, the Company received approximately
$27.5 million from the issuance of 21,825,131 shares
of series C redeemable convertible preferred stock at
$1.26 per share. The Company expects to record a beneficial
conversion charge (deemed dividend) during the second quarter of
2006 of approximately $19.4 million related to the issuance
of the Series C redeemable convertible preferred stock.
F-29
PART II
INFORMATION NOT REQUIRED IN PROSPECTUS
|
|
Item 13. |
Other Expenses of Issuance and Distribution. |
The following table indicates the expenses to be incurred in
connection with the offering described in this Registration
Statement, other than underwriting discounts and commissions,
all of which will be paid by the Registrant. All of the amounts
are estimated except the Securities and Exchange Commission
registration fee and the National Association of Securities
Dealers, Inc. filing fee.
|
|
|
|
|
|
Securities and Exchange Commission registration fee
|
|
$ |
9,229 |
|
|
|
|
|
National Association of Securities Dealers, Inc. filing fee
|
|
$ |
9,125 |
|
|
|
|
|
Nasdaq National Market listing fee
|
|
$ |
5,000 |
|
Accounting fees and expenses
|
|
|
* |
|
Legal fees and expenses
|
|
|
* |
|
Blue Sky fees and expenses
|
|
|
* |
|
Transfer Agents expenses
|
|
|
* |
|
Printing and engraving fees
|
|
|
* |
|
Miscellaneous
|
|
|
* |
|
|
Total expenses
|
|
$ |
* |
|
|
|
* |
To be filed by amendment. |
|
|
Item 14. |
Indemnification of Directors and Officers. |
Section 102 of the Delaware General Corporation Law permits
a corporation to eliminate the personal liability of directors
of a corporation to the corporation or its stockholders for
monetary damages for a breach of fiduciary duty as a director,
except where the director breached his or her duty of loyalty,
failed to act in good faith, engaged in intentional misconduct
or knowingly violated a law, authorized the payment of a
dividend or approved a stock repurchase in violation of Delaware
corporate law or obtained an improper personal benefit. The
Registrants restated certificate of incorporation to be
effective upon closing of this offering provides that no
director of the Registrant shall be personally liable to it or
its stockholders for monetary damages for any breach of
fiduciary duty as director, notwithstanding any provision of law
imposing such liability, except to the extent that the Delaware
General Corporation Law prohibits the elimination or limitation
of liability of directors for breaches of fiduciary duty.
Section 145 of the Delaware General Corporation Law
provides that a corporation has the power to indemnify any
person who was or is a party or is threatened to be made a party
to any threatened, pending or completed action, suit or
proceeding, whether civil, criminal, administrative or
investigative, by reason of the fact that the person is or was a
director, officer, employee or agent of the corporation, or is
or was serving at the request of the corporation as a director,
officer, employee or agent of another corporation, partnership,
joint venture, trust or other enterprise, against expenses
(including attorneys fees), judgments, fines and amounts
paid in settlements actually and reasonably incurred by the
person in connection with an action, suit or proceeding to which
he or she is or is threatened to be made a party by reason of
such position, if such person acted in good faith and in a
manner he or she reasonably believed to be in or not opposed to
the best interests of the corporation, and, in any criminal
action or proceeding, had no reasonable cause to believe his or
her conduct was unlawful, except that, in the case of actions
brought by or in the right of the corporation, no
indemnification shall be made with respect to any claim, issue
or matter as to which such person shall have been adjudged to be
liable to the corporation unless and only to the extent that the
Delaware Court of Chancery or other adjudicating court
determines that, despite the adjudication of liability but in
view of all of the circumstances of the case, such person is
fairly and reasonably entitled to indemnity for such expenses
which the Delaware Court of Chancery or such other court shall
deem proper.
II-1
The Registrants restated certificate of incorporation,
which is to be effective upon the closing of this offering,
provides that the Registrant will, to the fullest extent
permitted by Section 145 of the Delaware General
Corporation Law and the Registrants by-laws (each as
amended from time to time), indemnify each person who was or is
a party or is threatened to be made a party to any threatened,
pending or completed action, suit or proceeding, whether civil,
criminal, administrative or investigative, by reason of the fact
that he or she is or was, or has agreed to become, a director or
officer of the Registrant, or is or was serving, or has agreed
to serve, at the request of the Registrant, as a director,
officer, partner, or trustee of, or in a similar capacity with,
another corporation, partnership, joint venture, trust or other
enterprise, including any employee benefit plan (all such
persons being referred to hereafter as an
Indemnitee), or by reason of any action alleged to
have been taken or omitted in such capacity, against all
expenses (including attorneys fees), judgments, fines and
amounts paid in settlement actually and reasonably incurred by,
or on behalf of, the Indemnitee in connection with such action,
suit or proceeding and any appeal therefrom. Such
indemnification may include payment by the Registrant of
expenses in defending an action or proceeding in advance of the
final disposition of such action or proceeding upon receipt of
an undertaking by the Indemnitee (such undertaking acceptable by
the Registrant without reference to the financial ability of the
Indemnitee) to repay such payment if it is ultimately determined
that the Indemnitee is not entitled to indemnification under the
Registrants restated certificate of incorporation;
however, the Registrant will not indemnify any person seeking
indemnification in connection with a proceeding (or part
thereof) initiated by such person, unless such initiation was
approved by the Registrants board of directors. Also, the
indemnification rights provided in the Registrants
restated certificate of incorporation (i) are not exclusive
of any other rights to which those indemnified may be entitled
under any law, agreement or vote of stockholders or
disinterested directors or otherwise, and (ii) will inure
to the benefit of the heirs, executors and administrators of
such persons. The Registrant may, to the extent authorized from
time to time by its board of directors, grant indemnification
rights to other employees of the Registrant or other persons
serving the Registrant and such rights may be equivalent to, or
greater or less than, those set forth in the Registrants
restated certificate of incorporation.
The Registrant has entered into indemnification agreements with
each of its directors. These agreements, among other things,
require the Registrant to indemnify each director to the fullest
extent permitted by Delaware law, including indemnification of
expenses such as attorneys fees, judgments, fines and
settlement amounts incurred by the director in any action or
proceeding, including any action or proceeding by or in right of
the Registrant, arising out of the persons services as a
director.
The Registrant maintains a general liability insurance policy
that covers certain liabilities of the Registrants
directors and officers arising out of claims based on acts or
omissions in their capacities as directors or officers.
In any underwriting agreement that the Registrant enters into in
connection with the sale of common stock being registered
hereby, the underwriters will agree to indemnify, under certain
conditions, the Registrant, its directors, its officers and
persons who control the Registrant within the meaning of the
Securities Act, against certain liabilities.
|
|
Item 15. |
Recent Sales of Unregistered Securities. |
Set forth below is information regarding shares of common stock
and preferred stock issued, and options and warrants granted, by
the Registrant within the past three years that were not
registered under the Securities Act. Also included is the
consideration, if any, received by the Registrant for such
shares, options and warrants and information relating to the
section of the Securities Act, or rule of the Securities and
Exchange Commission, under which exemption from registration was
claimed.
|
|
|
(a) Issuances of Securities |
|
|
|
1. On June 20, 2003, the
Registrant issued a promissory note in the amount of $936,875 to
CHL Medical Partners, II, L.P. The Registrant also issued a
promissory note in the amount of |
II-2
|
|
|
$63,125 to CHL Medical Partners Side Fund II, L.P. The
principal outstanding under the notes was converted into shares
of series B redeemable convertible preferred stock in May
2004. |
|
|
2. On August 25, 2003, the
Registrant issued a promissory note in the amount of $936,875,
together with a warrant to purchase 312,291 shares of
common stock at an exercise price of $0.075 per share, to
CHL Medical Partners II, L.P. The Registrant also issued a
promissory note in the amount of $63,125, together with a
warrant to purchase 21,042 shares of common stock at
an exercise price of $0.075 per share, to CHL Medical
Partners Side Fund II, L.P. The principal outstanding under
the notes was converted into shares of series B redeemable
convertible preferred stock in May 2004. CHL Medical
Partners II, L.P. and CHL Medical Partners Side
Fund II, L.P. exercised their warrants in August 2005. |
|
|
3. On November 26, 2003, the
Registrant issued a promissory note in the amount of $936,875,
together with a warrant to purchase 312,291 shares of
common stock at an exercise price of $0.075 per share, to
CHL Medical Partners II, L.P. The Registrant also issued a
promissory note in the amount of $63,125, together with a
warrant to purchase 21,042 shares of common stock at
an exercise price of $0.075 per share, to CHL Medical
Partners Side Fund II, L.P. CHL Medical Partners II,
L.P. and CHL Medical Partners Side Fund II, L.P. exercised
their warrants in August 2005. |
|
|
4. On February 5, 2004, the
Registrant issued a promissory note in the amount of $1,873,750,
together with a warrant to purchase 312,291 shares of
common stock at an exercise price of $0.075 per share, to
CHL Medical Partners II, L.P. The promissory note issued on
February 5, 2004 amended and restated in its entirety the
promissory note issued to CHL Medical Partners II, L.P. on
November 26, 2003. The Registrant also issued a promissory
note in the amount of $126,250, together with a warrant to
purchase 21,042 shares of common stock at an exercise
price of $0.075 per share, to CHL Medical Partners Side
Fund II, L.P. The promissory note issued on
February 5, 2004 amended and restated in its entirety the
promissory note issued to CHL Medical Partners Side
Fund II, L.P. on November 26, 2003. CHL Medical
Partners II, L.P. and CHL Medical Partners Side
Fund II, L.P. exercised their warrants in August 2005. |
|
|
5. On April 19, 2004, the
Registrant issued a promissory note in the amount of $2,342,188
to CHL Medical Partners II, L.P. This promissory note
amended and restated in its entirety the promissory note issued
to CHL Medical Partners II, L.P. on February 5, 2004. The
Registrant also issued a promissory note in the amount of
$157,812 to CHL Medical Partners Side Fund II, L.P. This
promissory note amended and restated in its entirety the
promissory note issued to CHL Medical Partners Side
Fund II, L.P. on February 5, 2004. The principal
outstanding under the notes was converted into shares of
Series B convertible preferred stock in May 2004. |
|
|
6. On May 4, 2004 and
March 24, 2005, the Registrant issued an aggregate of
36,470,591 shares of our series B redeemable
convertible preferred stock at a price of $0.85 per share,
together with warrants to purchase an aggregate of
555,003 shares of series B redeemable convertible
preferred stock at an exercise price of $0.85 per share, to
institutional investors for aggregate cash proceeds of
approximately $31 million. |
|
|
7. On August 17, 2005 and
April 17, 2006, the Registrant issued an aggregate of
43,650,262 shares of our series C redeemable
convertible preferred stock at a price of $1.26 per share
to institutional investors for aggregate cash proceeds of
approximately $55 million. |
|
|
8. On August 23, 2005, the
Registrant issued, pursuant to the exercise of common stock
purchase warrants, (i) 936,873 shares of our common
stock at a purchase price of $0.075 per share to CHL
Medical Partners II, L.P., and (ii) 63,126 shares
of our common stock at a purchase price of $0.075 per share
to CHL Medical Partners II Side Fund, L.P., for aggregate
cash proceeds of approximately $75,000. |
No underwriters were involved in the foregoing sales of
securities. The securities described in this
section (a) of Item 15 were issued to a
combination of foreign and U.S. investors in reliance upon
the
II-3
exemption from the registration requirements of the Securities
Act, as set forth in Section 4(2) under the Securities Act
and Rule 506 of Regulation D promulgated thereunder,
relative to sales by an issuer not involving any public
offering, to the extent an exemption from such registration was
required. All purchasers of shares of convertible preferred
stock described above represented to the Registrant in
connection with their purchase that they were accredited
investors and were acquiring the shares for investment and not
distribution, that they could bear the risks of the investment
and could hold the securities for an indefinite period of time.
The purchasers received written disclosures that the securities
had not been registered under the Securities Act and that any
resale must be made pursuant to a registration statement or an
available exemption from such registration.
Since inception, the Registrant has granted options to certain
employees, consultants and others to purchase an aggregate of
16,030,166 shares of common stock as of May 3, 2006.
As of May 3, 2006, options to purchase
2,202,984 shares of common stock had been exercised,
options to purchase 1,060,840 shares of common stock had
been forfeited, and options to purchase 13,552,120 shares
of common stock remained outstanding at a weighted average
exercise price of $0.48 per share.
The issuance of stock options and the common stock issuable upon
the exercise of such options as described in this
section (b) of Item 15 were issued pursuant to
written compensatory plans or arrangements with the
Registrants employees, directors and consultants, in
reliance on the exemption provided by Rule 701 promulgated
under the Securities Act. All recipients either received
adequate information about the Registrant or had access, through
employment or other relationships, to such information.
All of the foregoing securities are deemed restricted securities
for purposes of the Securities Act. All certificates
representing the issued shares of common stock described in this
Item 15 included appropriate legends setting forth that the
securities had not been registered and the applicable
restrictions on transfer.
|
|
Item 16. |
Exhibits and Financial Statement Schedules. |
|
|
|
|
|
Exhibit |
|
|
Number |
|
Description of Exhibit |
|
|
|
|
1 |
.1** |
|
Form of Underwriting Agreement |
|
3 |
.1** |
|
Certificate of Incorporation of the Registrant, as amended |
|
3 |
.2* |
|
Form of Restated Certificate of Incorporation of the Registrant
to be effective upon completion of this offering |
|
3 |
.3* |
|
By-laws of the Registrant |
|
3 |
.4* |
|
Form of Restated By-laws of the Registrant to be effective upon
completion of this offering |
|
4 |
.1** |
|
Specimen Stock Certificate evidencing shares of common stock |
|
4 |
.2* |
|
Second Amended and Restated Investor Rights Agreement, dated as
of August 17, 2005, as amended |
|
4 |
.3* |
|
Warrant to purchase shares of common stock, dated
August 28, 2002 |
|
5 |
.1** |
|
Opinion of Bingham McCutchen LLP |
|
10 |
.1* |
|
2002 Equity Incentive Plan, as amended |
|
10 |
.2* |
|
2006 Equity Incentive Plan |
|
10 |
.3* |
|
2006 Employee Stock Purchase Plan |
|
10 |
.4*+ |
|
License Agreement, dated as of April 15, 2002, by and
between the Registrant and Mount Sinai School of Medicine of New
York University |
|
10 |
.5*+ |
|
License Agreement, dated as of June 26, 2003, by and
between the Registrant and University of Maryland, Baltimore
County |
|
10 |
.6*+ |
|
Exclusive License Agreement, dated as of June 8, 2005, by
and between the Registrant and Novo Nordisk, A/ S |
II-4
|
|
|
|
|
Exhibit |
|
|
Number |
|
Description of Exhibit |
|
|
|
|
10 |
.7* |
|
Sublease Agreement, dated as of May 12, 2005, by and
between the Registrant and Purdue Pharma, L.P. |
|
10 |
.8* |
|
Amended and Restated Employment Agreement, dated as of
April 28, 2006, by and between the Registrant and John F.
Crowley |
|
10 |
.9* |
|
Letter Agreement, dated as of November 9, 2004, by and
between the Registrant and Matthew R. Patterson |
|
10 |
.10* |
|
Letter Agreement, dated as of June 3, 2005, by and between
the Registrant and Pedro Huertas, M.D. |
|
10 |
.11* |
|
Letter Agreement, dated as of December 19, 2005, by and
between the Registrant and David Lockhart, Ph.D. |
|
10 |
.12* |
|
Letter Agreement, dated as of February 2, 2006, by and
between the Registrant and Karin Ludwig, M.D. |
|
10 |
.13* |
|
Change in Control Agreement, dated as of March 6, 2006, by
and between the Registrant and David Palling, Ph.D. |
|
10 |
.14* |
|
Change in Control Agreement, dated as of March 6, 2006, by
and between the Registrant and S. Nicole Schaeffer |
|
10 |
.15* |
|
Change in Control Agreement, dated as of March 6, 2006, by
and between the Registrant and Gregory P. Licholai, M.D. |
|
10 |
.16* |
|
Consulting Agreement, dated as of February 28, 2006, by and
between the Registrant and Donald J. Hayden, Jr. |
|
10 |
.17* |
|
Letter Agreement, dated as of May 12, 2006, by and between
the Registrant and Douglas A. Branch |
|
10 |
.18* |
|
Form of Director and Officer Indemnification Agreement |
|
10 |
.19 |
|
Letter Agreement, dated as of May 12, 2006, by and between
the Registrant and Mark Simon. |
|
23 |
.1 |
|
Consent of Ernst & Young LLP |
|
23 |
.2** |
|
Consent of Bingham McCutchen LLP (included in Exhibit 5.1) |
|
24 |
.1* |
|
Powers of Attorney |
|
|
|
* |
Previously filed. |
|
|
|
** |
To be filed by amendment. |
|
|
|
+ |
Portions of this exhibit have been omitted pursuant to a
confidential treatment request. This information has been filed
or will be filed separately with the Securities and Exchange
Commission. |
|
Financial Statement Schedules
All schedules have been omitted because they are not required or
are not applicable or the required information is shown in the
financial statements or notes thereto.
(a) The undersigned Registrant hereby undertakes to provide
to the underwriters at the closing specified in the Underwriting
Agreement, certificates in such denominations and registered in
such names as required by the underwriters to permit prompt
delivery to each purchaser.
(b) Insofar as indemnification for liabilities arising
under the Securities Act may be permitted to directors, officers
and controlling persons of the Registrant pursuant to the
provisions described under Item 14 above, or otherwise, the
Registrant has been advised that in the opinion of the
Securities and Exchange Commission such indemnification is
against public policy as expressed in the Securities Act and is,
therefore, unenforceable. In the event that a claim for
indemnification against such liabilities (other than the payment
by the Registrant of expenses incurred or paid by a director,
officer or controlling person of the Registrant in the
successful defense of any action, suit or proceeding) is
asserted by such director, officer or controlling person in
connection with the securities being registered, the Registrant
will, unless in the opinion of its counsel the matter has been
settled by controlling precedent, submit to a court of
appropriate jurisdiction the question whether such
indemnification by it is against public policy as expressed in
the Securities Act and will be governed by the final
adjudication of such issue.
II-5
(c) The undersigned Registrant hereby undertakes that:
|
|
|
(1) For purposes of determining any liability under the
Securities Act, the information omitted from the form of
prospectus filed as part of this Registration Statement in
reliance upon Rule 430A and contained in a form of
prospectus filed by the Registrant pursuant to
Rule 424(b)(1) or (4) or 497(h) under the Securities
Act shall be deemed to be part of this Registration Statement as
of the time it was declared effective. |
|
|
(2) For the purpose of determining any liability under the
Securities Act, each post-effective amendment that contains a
form of prospectus shall be deemed to be a new Registration
Statement relating to the securities offered therein, and the
offering of such securities at that time shall be deemed to be
the initial bona fide offering thereof. |
II-6
SIGNATURES
Pursuant to the requirements of the Securities Act, the
Registrant certifies that it has duly caused this Registration
Statement to be signed on its behalf by the undersigned,
thereunto duly authorized, in the city of Cranbury, New Jersey,
on the 19th day of June, 2006.
|
|
|
AMICUS THERAPEUTICS, INC. |
|
|
|
|
By: |
/s/ Douglas A. Branch
|
|
|
|
|
|
|
Douglas A. Branch |
|
|
|
Vice President Secretary and General Counsel |
|
Pursuant to the requirements of the Securities Act, this
Registration Statement has been signed by the following persons
in the capacities and on the dates indicated.
|
|
|
|
|
|
|
Signature |
|
Title |
|
Date |
|
|
|
|
|
|
* |
|
President, Chief Executive Officer and Director (principal
executive officer) |
|
June 19, 2006 |
|
/s/ John M.
McAdam |
|
Director, Finance and Accounting, and Corporate Controller
(principal financial and accounting officer) |
|
June 19, 2006 |
|
* |
|
Chairman of the Board |
|
June 19, 2006 |
|
* |
|
Director |
|
June 19, 2006 |
|
* |
|
Director |
|
June 19, 2006 |
|
* |
|
Director |
|
June 19, 2006 |
|
* |
|
Director |
|
June 19, 2006 |
|
* |
|
Director |
|
June 19, 2006 |
|
* |
|
Director |
|
June 19, 2006 |
|
*By: |
|
/s/ Douglas A. Branch
Douglas
A. Branch
Attorney-in-Fact |
|
|
|
|
II-7
EXHIBIT INDEX
|
|
|
|
|
Exhibit |
|
|
Number |
|
Description of Exhibit |
|
|
|
|
1 |
.1** |
|
Form of Underwriting Agreement |
|
3 |
.1** |
|
Certificate of Incorporation of the Registrant, as amended |
|
3 |
.2* |
|
Form of Restated Certificate of Incorporation of the Registrant
to be effective upon completion of this offering |
|
3 |
.3* |
|
By-laws of the Registrant |
|
3 |
.4* |
|
Form of Restated By-laws of the Registrant to be effective upon
completion of this offering |
|
4 |
.1** |
|
Specimen Stock Certificate evidencing shares of common stock |
|
4 |
.2* |
|
Second Amended and Restated Investor Rights Agreement, dated as
of August 17, 2005, as amended |
|
4 |
.3* |
|
Warrant to purchase shares of common stock, dated
August 28, 2002 |
|
5 |
.1** |
|
Opinion of Bingham McCutchen LLP |
|
10 |
.1* |
|
2002 Equity Incentive Plan, as amended |
|
10 |
.2* |
|
2006 Equity Incentive Plan |
|
10 |
.3* |
|
2006 Employee Stock Purchase Plan |
|
10 |
.4*+ |
|
License Agreement, dated as of April 15, 2002, by and
between the Registrant and Mount Sinai School of Medicine of New
York University |
|
10 |
.5*+ |
|
License Agreement, dated as of June 26, 2003, by and
between the Registrant and University of Maryland, Baltimore
County |
|
10 |
.6*+ |
|
Exclusive License Agreement, dated as of June 8, 2005, by
and between the Registrant and Novo Nordisk, A/ S |
|
10 |
.7* |
|
Sublease Agreement, dated as of May 12, 2005, by and
between the Registrant and Purdue Pharma, L.P. |
|
10 |
.8* |
|
Amended and Restated Employment Agreement, dated as of
April 28, 2006, by and between the Registrant and John F.
Crowley |
|
10 |
.9* |
|
Letter Agreement, dated as of November 9, 2004, by and
between the Registrant and Matthew R. Patterson |
|
10 |
.10* |
|
Letter Agreement, dated as of June 3, 2005, by and between
the Registrant and Pedro Huertas, M.D. |
|
10 |
.11* |
|
Letter Agreement, dated as of December 19, 2005, by and
between the Registrant and David Lockhart, Ph.D. |
|
10 |
.12* |
|
Letter Agreement, dated as of February 2, 2006, by and
between the Registrant and Karin Ludwig, M.D. |
|
10 |
.13* |
|
Change in Control Agreement, dated as of March 6, 2006, by
and between the Registrant and David Palling, Ph.D. |
|
10 |
.14* |
|
Change in Control Agreement, dated as of March 6, 2006, by
and between the Registrant and S. Nicole Schaeffer |
|
10 |
.15* |
|
Change in Control Agreement, dated as of March 6, 2006, by
and between the Registrant and Gregory P. Licholai, M.D. |
|
10 |
.16* |
|
Consulting Agreement, dated as of February 28, 2006, by and
between the Registrant and Donald J. Hayden, Jr. |
|
10 |
.17* |
|
Letter Agreement, dated as of May 12, 2006, by and between
the Registrant and Douglas A. Branch |
|
10 |
.18* |
|
Form of Director and Officer Indemnification Agreement |
|
10 |
.19 |
|
Letter Agreement, dated as of May 12, 2006, by and between
the Registrant and Mark Simon. |
|
23 |
.1 |
|
Consent of Ernst & Young LLP |
|
23 |
.2** |
|
Consent of Bingham McCutchen LLP (included in Exhibit 5.1) |
|
24 |
.1* |
|
Powers of Attorney |
|
|
|
* |
Previously filed. |
|
|
|
** |
To be filed by amendment. |
|
|
|
+ |
Portions of this exhibit have been omitted pursuant to a
confidential treatment request. This information has been filed
or will be filed separately with the Securities and Exchange
Commission. |
|
Exhibit 10.19
[AMICUS LOGO]
AMICUS
Therapeutics--------------------------------------------------------------------
May 12, 2006
Mr. Mark Simon
37 Kennedy Lane
Morristown NJ. 07960
Dear Mark:
On behalf of Amicus Therapeutics, Inc. (the "Company"), I am pleased to
confirm our offer to you for the position of Senior Vice President Business
Development reporting to me. Your start date will be mutually agreed upon but no
later then June 12, 2006. With this offer you agree to devote no less then 30
hrs per week of your time and professional efforts to Amicus Therapeutics.
Prior to the commencement of your employment you will be required to
execute the Company's Confidentiality, Disclosure and Non-Competition Agreement.
A copy of this agreement is attached. In addition, as a condition of employment
Amicus requires a pre-employment drug screening.
In consideration for all your services to be rendered to the Company
your annual base salary will be $100,000, to be paid bi-weekly in accordance
with the Company's payroll practices. Upon the completion of mutually agreed
upon individual goals and objectives as well as the achievement of specific
Company goals, you will be eligible to receive a year end bonus target of 25% of
your base salary, prorated for your date of hire, minus customary deductions.
Upon approval by the Board of Directors, you will receive an incentive
stock option to purchase 375,000 shares of the Company's common stock, par value
$.01 per share (the "Common Stock") pursuant to a stock option agreement in form
and substance acceptable to the Company. The options will become exercisable
over a four-year period as follows: 25% on the first anniversary of the date of
grant, and the remaining 75% in equal monthly increments thereafter. The
exercise price of the options will be the fair market value of the Company's
common stock on the date of grant. Shares issuable upon exercise of each option
will be subject to certain transfer restrictions including the right of first
refusal. Additionally, exercise of the options will be governed in accordance
with the provisions of the Company's stock option plan.
You will be eligible to participate in the Company's health benefits
program and are eligible to participate in the Company's 401(k) as well as any
other employee benefit plan(s) that are generally made available by the Company
to its employees from time to time when and as the Company may make them
available.
- --------------------------------------------------------------------------------
6 Cedar Brook Drive Cranbury, NJ 08512 T: 609-662-2000 F: 609-662-2001
www.amicustherapeutics.com
Mr. Mark Simon
May 12, 2006
Page #2 of 3
You will be eligible for paid Company holidays as outlined in our
Holiday Policy and you will be eligible for twenty (20) days paid vacation,
three weeks during the year and one between Christmas and New Years. Vacation
accrues on a monthly basis. Because the Company expects to regularly review its
benefit programs to keep them up to date and competitive, these programs are
subject to periodic adjustments so that certain features may be added, modified
or deleted over time.
If you are terminated without Cause, you will be eligible for a
continuation of six (6) months salary, an additional six (6) months of option
vesting, plus payment of a bonus payment equal to the bonus earned in the
preceding year. "Cause" means for any of the following reasons: (i) willful or
deliberate misconduct by you that materially damages the company; (ii)
misappropriation of company assets; (iii) conviction of or a plea of guilty or
"no contest" to, a felony; or (iv) any willful disobedience of the lawful and
unambiguous instructions of the CEO of the company; provided that the CEO has
given you written notice of such disobedience or neglect and you have failed to
cure such disobedience or neglect within a period reasonable under the
circumstances.
If there is a Change in Control Event and you resign for Good Reason or
are terminated without Cause within six months of such Change in Control Event,
then (i) you will be entitled to receive a continuation of twelve (12) months
salary, plus payment of a bonus payment equal to the bonus earned in the
preceding year and (ii) all unvested stock options will have their remaining
vesting schedule accelerated so that all stock options are fully vested.
"Change in Control Event" means any of the following: (i) any person or
entity (except for a current stockholder) becomes the beneficial owner of
greater than 50% of the then outstanding voting power of the company; (ii) a
merger or consolidation with another entity where the voting securities of the
company outstanding immediately before the transaction constitute less than a
majority of the voting power of the voting securities of the company or the
surviving entity outstanding immediately after the transaction, or (iii) the
sales or disposition of all or substantially all of the company's assets. "Good
Reason" means (i) a change in your position with the company or its successor
that materially reduces your title, duties or level of responsibility; or (ii)
the relocation of the company or its successor greater than 50 miles away from
the then current location of the company's principal offices.
Your right to receive accelerated vesting and severance payments
pursuant to the preceding three paragraphs shall be subject to the condition
that you execute a full release and waiver of all claims against the company and
related parties, in a form acceptable to the company.
There is a two (2) year term on this agreement that will automatically
renew unless either party provides a thirty (30) day notice of termination.
Mr. Mark Simon
May 12, 2006
Page #3 of 3
In accordance with the Immigration and Naturalization Control Act, all
new employees must provide documentation that they have the legal right to work
in the United States. A copy of Form I-9 and a list of the acceptable documents
confirming your right to work in the United States are also attached for your
convenience.
To indicate your acceptance of our offer, please sign one copy of this
letter in the space indicated below and return it to the attention of Nicole
Schaeffer, Vice President of Human Resources & Leadership Development by May 19,
2006. Acceptance of this offer constitutes your agreement with all of the above
terms and conditions of employment with Amicus Therapeutics, Inc., and
constitutes agreement to conform to Amicus Therapeutics, Inc. rules and
procedures. By signing below, you agree that no other promises, express or
implied, have been made to you either verbally or in writing and that no further
modifications to these terms and conditions will be effective except by a
written agreement signed by the Chief Executive Officer of the Company and you.
The formality of this letter not withstanding, I extend my personal
best wishes and sincere pleasure that you are joining our team. I look forward
to working with you.
Sincerely,
John F. Crowley
President & CEO
I accept the offer of employment under the terms and conditions stated above. No
other promises, express or implied, have been made to me either verbally or in
writing.
By: Date:
------------------------------------------- -------------------------
Mark Simon
EX-23.1
Exhibit 23.1
Consent of Independent Registered Public Accounting Firm
We consent to the reference to our firm under the caption
Experts and to the use of our report dated
May 12, 2006, in Amendment No. 1 to the Registration Statement
(Form S-1
No. 333-134191) and related Prospectus of Amicus
Therapeutics, Inc. for the registration of shares of its
common stock.
MetroPark, New Jersey
June 19, 2006