Form 10-Q
UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
Washington, D.C. 20549
FORM 10-Q
(Mark One)
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þ |
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QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 |
For the quarterly period ended March 31, 2011
OR
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o |
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TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 |
For the transition period from to
Commission file number 001-33497
Amicus Therapeutics, Inc.
(Exact Name of Registrant as Specified in Its Charter)
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Delaware |
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71-0869350 |
(State or Other Jurisdiction of
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(I.R.S. Employer |
Incorporation or Organization)
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Identification Number) |
6 Cedar Brook Drive, Cranbury, NJ 08512
(Address of Principal Executive Offices and Zip Code)
Registrants Telephone Number, Including Area Code: (609) 662-2000
Indicate by check mark whether the registrant (1) has filed all reports required to be filed
by Section 13 or 15(d) of the Securities Exchange Act of 1934 during the preceding 12 months (or
for such shorter period that the registrant was required to file such reports), and (2) has been
subject to such filing requirements for the past 90 days: Yes þ No o
Indicate by check mark whether the registrant has submitted electronically and
posted on its corporate Web site, if any, every Interactive Data File required to be submitted and
posted pursuant to Rule 405 of Regulation S-T (§232.405 of this chapter) during the preceding 12
months (or for such shorter period that the registrant was required to submit and post such files).
Yes o No o
Indicate by check mark whether the registrant is a large accelerated filer, an
accelerated filer, a non-accelerated filer or a smaller-reporting company. See definition of large
accelerated filer, accelerated filer and smaller-reporting company in Rule 12b-2 of the
Exchange Act. (Check one):
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Large accelerated filer o
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Accelerated filer o
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Non-accelerated filer o
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Smaller Reporting Company þ |
Indicate by check mark whether the registrant is a shell company (as defined in Rule 12b-2 of
the Exchange Act): Yes o No þ
The number of shares outstanding of the registrants common stock, $.01 par value per
share, as of April 26, 2011 was 34,516,180 shares.
AMICUS THERAPEUTICS, INC
Form 10-Q for the Quarterly Period Ended March 31, 2011
We have filed applications to register certain trademarks in the United States and abroad,
including AMICUSTM, AMICUS THERAPEUTICSTM (and design), AMIGALTM
and PLICERATM.
- 2 -
SPECIAL NOTE REGARDING FORWARD-LOOKING STATEMENTS
This quarterly report on Form 10-Q contains forward-looking statements that involve
substantial risks and uncertainties. All statements, other than statements of historical facts,
included in this quarterly report on Form 10-Q 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 quarterly report on Form 10-Q include, among other
things, statements about:
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the progress and results of our clinical trials of our drug candidates, including
Amigal; |
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our ability to achieve development and commercialization milestone payments and sales
royalties under our collaboration with GlaxoSmithKline PLC; |
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the scope, progress, results and costs of preclinical development, laboratory testing
and clinical trials for our product candidates including those testing the use of
pharmacological chaperones co-administered with ERT and for the treatment of diseases of
neurodegeneration; |
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the costs, timing and outcome of regulatory review of our product candidates; |
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the number and development requirements of other product candidates that we pursue; |
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the costs of commercialization activities, including product marketing, sales and
distribution; |
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the emergence of competing technologies and other adverse market developments; |
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the costs of preparing, filing and prosecuting patent applications and maintaining,
enforcing and defending intellectual property related claims; |
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the extent to which we acquire or invest in businesses, products and technologies; and |
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our ability to establish collaborations and obtain milestone, royalty or other payments
from any such collaborators. |
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 Part I Item 1A Risk Factors of the
Annual Report on Form 10-K for the year ended December 31, 2010 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 quarterly report on Form 10-Q in conjunction with the documents that we
reference herein. We do not assume any obligation to update any forward-looking statements.
- 3 -
PART I. FINANCIAL INFORMATION
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Item 1. |
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Financial Statements (unaudited) |
Amicus Therapeutics, Inc.
(a development stage company)
Consolidated Balance Sheets
(Unaudited)
(in thousands, except share and per share amounts)
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December 31, |
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March 31, |
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2010 |
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2011 |
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Assets: |
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Current assets: |
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Cash and cash equivalents |
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$ |
29,572 |
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$ |
27,662 |
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Investments in marketable securities |
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77,873 |
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66,112 |
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Receivable due from GSK |
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3,969 |
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Prepaid expenses and other current assets |
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2,236 |
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2,304 |
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Total current assets |
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109,681 |
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100,047 |
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Property and equipment, less accumulated depreciation and amortization of
$8,095 and $8,533 at December 31, 2010 and March 31, 2011, respectively |
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2,604 |
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2,237 |
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Other non-current assets |
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267 |
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267 |
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Total Assets |
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$ |
112,552 |
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$ |
102,551 |
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Liabilities and Stockholders Equity |
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Current liabilities: |
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Accounts payable and accrued expenses |
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$ |
8,290 |
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$ |
7,418 |
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Current portion of capital lease obligations |
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40 |
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Current portion of deferred revenue |
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6,640 |
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7,965 |
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Current portion of secured loan |
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1,253 |
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1,253 |
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Total current liabilities |
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16,223 |
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16,636 |
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Deferred revenue, less current portion |
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25,639 |
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23,979 |
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Warrant liability |
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4,712 |
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8,144 |
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Secured loan, less current portion |
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1,044 |
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731 |
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Commitments and contingencies |
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Stockholders equity: |
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Common stock, $.01 par value, 50,000,000 shares authorized, 34,508,932
shares
issued and outstanding at December 31, 2010, 50,000,000 shares
authorized, 34,516,180 shares issued and outstanding at March
31, 2011 |
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406 |
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406 |
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Additional paid-in capital |
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290,248 |
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291,696 |
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Accumulated other comprehensive income |
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(28 |
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1 |
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Deficit accumulated during the development stage |
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(225,692 |
) |
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(239,042 |
) |
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Total stockholders equity |
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64,934 |
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53,061 |
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Total Liabilities and Stockholders Equity |
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$ |
112,552 |
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$ |
102,551 |
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See accompanying notes to consolidated financial statements
- 4 -
Amicus Therapeutics, Inc.
(a development stage company)
Consolidated Statements of Operations
(Unaudited)
(in thousands, except share and per share amounts)
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Period from |
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February 4, |
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2002 |
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Three Months |
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(inception) |
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Ended March 31, |
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to March 31, |
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2010 |
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2011 |
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2011 |
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Revenue: |
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Research revenue |
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$ |
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$ |
4,306 |
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$ |
35,414 |
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Collaboration revenue |
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1,660 |
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52,582 |
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Total revenue |
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5,966 |
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87,996 |
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Operating Expenses: |
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Research and development |
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$ |
8,889 |
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$ |
11,125 |
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$ |
225,889 |
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General and administrative |
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3,925 |
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4,402 |
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97,771 |
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Restructuring charges |
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1,522 |
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Impairment of leasehold improvements |
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1,030 |
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Depreciation and amortization |
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536 |
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438 |
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8,916 |
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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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13,350 |
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15,965 |
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335,546 |
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Loss from operations |
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(13,350 |
) |
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(9,999 |
) |
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(247,550 |
) |
Other income (expenses): |
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Interest income |
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53 |
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59 |
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13,972 |
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Interest expense |
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(83 |
) |
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(48 |
) |
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(2,233 |
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Change in fair value of warrant liability |
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204 |
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(3,432 |
) |
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(5,296 |
) |
Other income |
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70 |
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231 |
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Loss before tax benefit |
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(13,176 |
) |
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(13,350 |
) |
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(240,876 |
) |
Benefit from income taxes |
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1,834 |
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Net loss |
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(13,176 |
) |
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(13,350 |
) |
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(239,042 |
) |
Deemed dividend |
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(19,424 |
) |
Preferred stock accretion |
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(802 |
) |
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Net loss attributable to common stockholders |
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$ |
(13,176 |
) |
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$ |
(13,350 |
) |
|
$ |
(259,268 |
) |
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Net loss attributable to common stockholders per
common share basic and diluted |
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$ |
(0.54 |
) |
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$ |
(0.39 |
) |
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Weighted-average common shares outstanding
basic and diluted |
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24,289,422 |
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34,498,926 |
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See accompanying notes to consolidated financial statements
- 5 -
Amicus Therapeutics, Inc.
(a development stage company)
Consolidated Statements of Cash Flows
(Unaudited)
(in thousands)
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Period from |
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February 4, 2002 |
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Three Months |
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(inception) to |
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Ended March 31, |
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March 31, |
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2010 |
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2011 |
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2011 |
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Operating activities |
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Net loss |
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$ |
(13,176 |
) |
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$ |
(13,350 |
) |
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$ |
(239,042 |
) |
Adjustments to reconcile net loss to net cash used
in operating activities: |
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Non-cash interest expense |
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525 |
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Depreciation and amortization |
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536 |
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438 |
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8,916 |
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Amortization of non-cash compensation |
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522 |
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Stock-based compensation employees |
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1,716 |
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1,418 |
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28,477 |
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Stock-based compensation non-employees |
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853 |
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Stock-based license payments |
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1,220 |
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Change in fair value of warrant liability |
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(204 |
) |
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3,432 |
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5,296 |
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Loss on disposal of asset |
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360 |
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Impairment of leasehold improvements |
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1,030 |
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Non-cash charge for in-process research and development |
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|
418 |
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Beneficial conversion feature related to bridge financing |
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135 |
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Changes in operating assets and liabilities: |
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Prepaid expenses and other current assets |
|
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(242 |
) |
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(4,037 |
) |
|
|
(6,273 |
) |
Other non-current assets |
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218 |
|
|
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(288 |
) |
Accounts payable and accrued expenses |
|
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(2,277 |
) |
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(872 |
) |
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7,418 |
|
Deferred revenue |
|
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(335 |
) |
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31,944 |
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Net cash used in operating activities |
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(13,429 |
) |
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(13,306 |
) |
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(158,489 |
) |
Investing activities |
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Sale and redemption of marketable securities |
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19,771 |
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30,889 |
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604,503 |
|
Purchases of marketable securities |
|
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(8,799 |
) |
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(19,099 |
) |
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(670,734 |
) |
Purchases of property and equipment |
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(69 |
) |
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(71 |
) |
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(12,540 |
) |
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Net cash provided by/(used in) investing activities |
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10,903 |
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11,719 |
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(78,771 |
) |
Financing activities |
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Proceeds from the issuance of preferred stock, net of issuance costs |
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143,022 |
|
Proceeds from the issuance of common stock and warrants,
net of issuance costs |
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|
17,153 |
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|
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|
113,307 |
|
Proceeds from the issuance of convertible notes |
|
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|
|
|
|
|
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|
5,000 |
|
Payments of capital lease obligations |
|
|
(102 |
) |
|
|
(40 |
) |
|
|
(5,587 |
) |
Payments of secured loan agreement |
|
|
(313 |
) |
|
|
(313 |
) |
|
|
(1,774 |
) |
Proceeds from exercise of stock options |
|
|
11 |
|
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|
30 |
|
|
|
1,321 |
|
Proceeds from exercise of warrants (common and preferred) |
|
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|
|
|
|
|
|
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|
264 |
|
Proceeds from capital asset financing arrangement |
|
|
|
|
|
|
|
|
|
|
5,611 |
|
Proceeds from secured loan agreement |
|
|
|
|
|
|
|
|
|
|
3,758 |
|
|
|
|
|
|
|
|
|
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Net cash provided by/(used in) financing activities |
|
|
16,749 |
|
|
|
(323 |
) |
|
|
264,922 |
|
|
|
|
|
|
|
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|
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Net increase/(decrease) in cash and cash equivalents |
|
|
14,223 |
|
|
|
(1,910 |
) |
|
|
27,662 |
|
Cash and cash equivalents at beginning of period |
|
|
19,339 |
|
|
|
29,572 |
|
|
|
|
|
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|
|
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|
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Cash and cash equivalents at end of period |
|
$ |
33,562 |
|
|
$ |
27,662 |
|
|
$ |
27,662 |
|
|
|
|
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- 6 -
Amicus Therapeutics, Inc.
(a development stage company)
Consolidated Statements of Cash Flows (continued)
(Unaudited)
(in thousands)
|
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Period from |
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|
February 4, 2002 |
|
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|
Three Months |
|
|
(inception) to |
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|
Ended March 31, |
|
|
March 31, |
|
|
|
2010 |
|
|
2011 |
|
|
2011 |
|
Supplemental disclosures of cash flow information |
|
|
|
|
|
|
|
|
|
|
|
|
Cash paid during the period for interest |
|
$ |
83 |
|
|
$ |
48 |
|
|
$ |
1,932 |
|
Non-cash activities |
|
|
|
|
|
|
|
|
|
|
|
|
Conversion of notes payable to preferred stock |
|
$ |
|
|
|
$ |
|
|
|
$ |
5,000 |
|
Conversion of preferred stock to common stock |
|
$ |
|
|
|
$ |
|
|
|
$ |
148,951 |
|
Accretion of redeemable convertible preferred stock |
|
$ |
|
|
|
$ |
|
|
|
$ |
802 |
|
Beneficial conversion feature related to the issuance
of Series C redeemable convertible preferred stock |
|
$ |
|
|
|
$ |
|
|
|
$ |
19,424 |
|
See accompanying notes to consolidated financial statements
- 7 -
Note 1. Description of Business and Significant Accounting Policies
Corporate Information, Status of Operations and Management Plans
Amicus Therapeutics, Inc. (the Company) was incorporated on February 4, 2002 in Delaware and
is a biopharmaceutical company focused on the discovery, development and commercialization of
orally-administered, small molecule drugs known as pharmacological chaperones for the treatment of
rare diseases. Pharmacological chaperones are a novel, first-in-class approach to treating a broad
range of diseases including lysosomal storage disorders and diseases of neurodegeneration. 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.
In October 2010, the Company entered into the License and Collaboration Agreement with Glaxo
Group Limited, an affiliate of GlaxoSmithKline PLC (GSK), to develop and commercialize Amigal.
Under the terms of the License and Collaboration Agreement, GSK received an exclusive worldwide
license to develop, manufacture and commercialize Amigal. In consideration of the license grant,
the Company received an upfront, license payment of $30 million and a premium related to the equity
portion of the transaction of $3.2 million from GSK and is eligible to receive further payments of
approximately $170 million upon the successful achievement of development and commercialization
milestones, as well as tiered double-digit royalties on global sales of Amigal. GSK and the Company
will jointly fund development costs in accordance with an agreed upon development plan. For further
information, see Note 8. Collaborative Agreements.
The Company had an accumulated deficit of approximately $239.0 million at March 31, 2011 and
anticipates incurring losses through the year 2011 and beyond. The Company has not yet generated
commercial sales revenue and has been able to fund its operating losses to date through the sale of
its redeemable convertible preferred stock, issuance of convertible notes, net proceeds from our
initial public offering (IPO) and subsequent stock offerings, payments from partners during the
terms of the collaboration agreements and other financing arrangements. In March 2010, the Company
sold 4.95 million shares of its common stock and warrants to purchase 1.85 million shares of common
stock in a registered direct offering to a select group of institutional investors for net proceeds
of $17.1 million. In October 2010, the Company sold 6.87 million shares of its common stock as part
of the License and Collaboration Agreement with GSK for proceeds of $31 million. The Company
believes that its existing cash and cash equivalents and short-term investments will be sufficient
to cover its cash flow requirements for 2011.
Basis of Presentation
The Company has prepared the accompanying unaudited consolidated financial statements in
accordance with accounting principles generally accepted in the United States of America (U.S.
GAAP) for interim financial information and with the instructions to Form 10-Q and Article 10-01 of
Regulations S-X. Accordingly, they do not include all of the information and disclosures required
by generally accepted accounting principles for complete financial statements. In the opinion of
management, the accompanying unaudited financial statements reflect all adjustments, which include
only normal recurring adjustments, necessary to present fairly the Companys interim financial
information.
The accompanying unaudited consolidated financial statements and related notes should be read
in conjunction with the Companys financial statements and related notes as contained in the
Companys Annual Report on Form 10-K for the year ended December 31, 2010. For a complete
description of the Companys accounting policies, please refer to the Annual Report on Form 10-K
for the fiscal year ended December 31, 2010.
Revenue Recognition
The Company recognizes revenue when amounts are realized or realizable and earned. Revenue is
considered realizable and earned when the following criteria are met: (1) persuasive evidence of an
arrangement exists; (2) delivery has occurred or services have been rendered; (3) the price is
fixed or determinable; and (4) collection of the amounts due are reasonably assured.
- 8 -
In multiple element arrangements, revenue is allocated to each separate unit of accounting and
each deliverable in an arrangement is evaluated to determine whether it represents separate units
of accounting. A deliverable constitutes a separate unit of accounting when it has standalone value
and there is no general right of return for the delivered elements. In instances when the
aforementioned criteria are not met, the deliverable is combined with the undelivered elements and
the allocation of the arrangement consideration and revenue recognition is determined for the
combined unit as a single unit of accounting. Allocation of the consideration is determined at
arrangement inception on the basis of each units relative selling price. In instances where there
is determined to be a single unit of accounting, the total consideration is applied as revenue for
the single unit of accounting and is recognized over the period of inception through the date where
the last deliverable within the single unit of accounting is expected to be delivered.
The Companys current revenue recognition policies provide that, when a collaboration
arrangement contains multiple deliverables, such as license and research and development services,
the Company allocates revenue to each separate unit of accounting based on a selling price
hierarchy. The selling price hierarchy for a deliverable is based on (i) its vendor specific
objective evidence (VSOE) if available, (ii) third party evidence (TPE) if VSOE is not available,
or (iii) estimated selling price (BESP) if neither VSOE nor TPE is available. The Company would
establish the VSOE of selling price using the price charged for a deliverable when sold separately.
The TPE of selling price would be established by evaluating largely similar and interchangeable
competitor products or services in standalone sales to similarly situated customers. The best
estimate of selling price would be established considering internal factors such as an internal
pricing analysis or an income approach using a discounted cash flow model.
The revenue associated with reimbursements for research and development costs under
collaboration agreements is included in Research Revenue and the costs associated with these
reimbursable amounts are included in research and development expenses. The Company records these
reimbursements as revenue and not as a reduction of research and development expenses as the
Company has not commenced its planned principal operations (i.e., selling commercial products) and
is a development stage enterprise, therefore development activities are part of its ongoing central
operations.
The Companys collaboration agreement with GSK provides for, and any future collaborative
agreements the Company may enter into also may provide for, contingent milestone payments. In order
to determine the revenue recognition for these contingent milestones, the Company evaluates the
contingent milestones using the criteria as provided by the Financial Accounting Standards Boards
(FASB) guidance on the milestone method of revenue recognition at the inception of a collaboration
agreement. The criteria requires that (i) the Company determines if the milestone is commensurate
with either its performance to achieve the milestone or the enhancement of value resulting from the
Companys activities to achieve the milestone, (ii) the milestone be related to past performance,
and (iii) the milestone be reasonable relative to all deliverable and payment terms of the
collaboration arrangement. If these criteria are met then the contingent milestones can be
considered as substantive milestones and will be recognized as revenue in the period that the
milestone is achieved.
Fair Value Measurements
The Company records certain asset and liability balances under the fair value measurements as
defined by the FASB guidance. Current FASB fair value guidance emphasizes that fair value is a
market-based measurement, not an entity-specific measurement. Therefore, a fair value measurement
should be determined based on the assumptions that market participants would use in pricing the
asset or liability. As a basis for considering market participant assumptions in fair value
measurements, current FASB guidance establishes a fair value hierarchy that distinguishes between
market participant assumptions based on market data obtained from sources independent of the
reporting entity (observable inputs that are classified within Levels 1 and 2 of the hierarchy) and
the reporting entitys own assumptions that market participants assumptions would use in pricing
assets or liabilities (unobservable inputs classified within Level 3 of the hierarchy).
- 9 -
Level 1 inputs utilize quoted prices (unadjusted) in active markets for identical assets or
liabilities that the Company has the ability to access at measurement date. Level 2 inputs are
inputs other than quoted prices included in Level 1 that are observable for the asset or liability,
either directly or indirectly. Level 2 inputs may include quoted prices for similar assets and
liabilities in active markets, as well as inputs that are observable for the asset or
liability (other than quoted prices), such as interest rates, foreign exchange rates, and
yield curves that are observable at commonly quoted intervals. Level 3 inputs are unobservable
inputs for the asset or liability, which are typically based on an entitys own assumptions, as
there is little, if any, related market activity. In instances where the determination of the fair
value measurement is based on inputs from different levels of the fair value hierarchy, the level
in the fair value hierarchy within which the entire fair value measurement falls is based on the
lowest level input that is significant to the fair value measurement in its entirety. The Companys
assessment of the significance of a particular input to the fair value measurement in its entirety
requires judgment, and considers factors specific to the asset or liability.
New Accounting Standards
In April 2010, the FASB issued guidance on revenue recognition related to the milestone
method of revenue recognition. This guidance provides criteria on defining a substantive
milestone and determining when it may be appropriate to apply the milestone method of revenue
recognition for research or development transactions. Early adoption is permitted
retrospectively from the beginning of an entitys fiscal year. The Company early adopted this
guidance on the milestone method of revenue recognition and retrospectively applied this
guidance to the beginning of 2010. This method was first applied in conjunction with the
License and Collaboration Agreement with GSK during the fourth quarter of 2010; there have been
no milestones recognized in the year of adoption. This guidance did not have a material impact
on the timing or pattern of revenue recognition relative to the agreement nor is expected to in
future periods.
Subsequent Events
On April 18, 2011, John F. Crowley resigned as Chairman and Chief Executive Officer of
the Company and was appointed by the Board of Directors of the Company (the Board) as
Executive Chairman. Mr. Crowley will serve as Executive Chairman until September 30, 2011,
subject to an extension of up to three months upon the mutual agreement of Mr. Crowley and the
Company. In his role as Executive Chairman, Mr. Crowley will perform all the duties of
Chairman of the Board and such other executive officer duties as the Board may assign him from
time to time at its sole discretion, and will provide at least 20 hours of service per week to
the Company.
Upon Mr. Crowleys resignation as Chairman and Chief Executive Officer, the Board
appointed Matthew R. Patterson as Acting Chief Executive Officer and Principal Executive
Officer of the Company, in addition to his duties as President of the Company. The Board
intends to shortly initiate a search for a permanent Chief Executive Officer and will include
Mr. Patterson as a candidate for the position.
As previously disclosed, in connection with Mr. Crowleys transition to his new role as
Executive Chairman and Mr. Pattersons appointment as Acting Chief Executive Officer, the
Company entered into a new employment agreement with Mr. Crowley and an amended severance
agreement with Mr. Patterson. Mr. Crowleys employment agreement included terms that modified
his outstanding stock option grants. As a result of this modification, the Company expects to
record additional compensation expense of approximately $0.7 million in the second quarter of
2011. The Company also granted Mr. Patterson 50,000 shares of restricted stock.
Note 2. Cash, Cash Equivalents and Available-for-Sale Investments
As of March 31, 2011, the Company held $27.7 million in cash and cash equivalents and
$66.1 million of available-for-sale investment securities which are reported at fair value on the
Companys balance sheet. Unrealized holding gains and losses are reported within accumulated other
comprehensive income/(loss) as a separate component of stockholders equity. 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. To date, only
temporary impairment adjustments have been recorded.
- 10 -
Consistent with the Companys investment policy, the Company does not use derivative financial
instruments in its investment portfolio. The Company regularly invests excess operating cash in
deposits with major financial
institutions, money market funds, notes issued by the U.S. government, as well as fixed income
investments and U.S. bond funds both of which can be readily purchased and sold using established
markets. The Company believes that the market risk arising from its holdings of these financial
instruments is mitigated as many of these securities are either government backed or of the highest
credit rating.
The Companys investment portfolio has not been materially adversely impacted by the recent
disruption in credit markets.
Cash and available for sale securities consisted of the following as of December 31, 2010
and March 31, 2011 (in thousands):
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
As of December 31, 2010 |
|
|
|
|
|
|
|
Unrealized |
|
|
Unrealized |
|
|
Fair |
|
|
|
Cost |
|
|
Gain |
|
|
Loss |
|
|
Value |
|
Cash balances |
|
$ |
29,572 |
|
|
$ |
|
|
|
$ |
|
|
|
$ |
29,572 |
|
U.S. government agency securities |
|
|
12,000 |
|
|
|
|
|
|
|
(9 |
) |
|
|
11,991 |
|
Corporate debt securities |
|
|
42,075 |
|
|
|
2 |
|
|
|
(33 |
) |
|
|
42,044 |
|
Commercial paper |
|
|
23,476 |
|
|
|
12 |
|
|
|
|
|
|
|
23,488 |
|
Certificate of deposit |
|
|
350 |
|
|
|
|
|
|
|
|
|
|
|
350 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
$ |
107,473 |
|
|
$ |
14 |
|
|
$ |
(42 |
) |
|
$ |
107,445 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Included in cash and cash equivalents |
|
$ |
29,572 |
|
|
$ |
|
|
|
$ |
|
|
|
$ |
29,572 |
|
Included in marketable securities |
|
|
77,901 |
|
|
|
14 |
|
|
|
(42 |
) |
|
|
77,873 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Total cash and available for sale securities |
|
$ |
107,473 |
|
|
$ |
14 |
|
|
$ |
(42 |
) |
|
$ |
107,445 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
As of March 31, 2011 |
|
|
|
|
|
|
|
Unrealized |
|
|
Unrealized |
|
|
Fair |
|
|
|
Cost |
|
|
Gain |
|
|
Loss |
|
|
Value |
|
Cash balances |
|
$ |
27,662 |
|
|
$ |
|
|
|
$ |
|
|
|
$ |
27,662 |
|
U.S. government agency securities |
|
|
3,000 |
|
|
|
|
|
|
|
(1 |
) |
|
|
2,999 |
|
Corporate debt securities |
|
|
43,531 |
|
|
|
5 |
|
|
|
(17 |
) |
|
|
43,519 |
|
Commercial paper |
|
|
19,230 |
|
|
|
14 |
|
|
|
|
|
|
|
19,244 |
|
Certificate of deposit |
|
|
350 |
|
|
|
|
|
|
|
|
|
|
|
350 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
$ |
93,773 |
|
|
$ |
19 |
|
|
$ |
(18 |
) |
|
$ |
93,774 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Included in cash and cash equivalents |
|
$ |
27,662 |
|
|
$ |
|
|
|
$ |
|
|
|
$ |
27,662 |
|
Included in marketable securities |
|
|
66,111 |
|
|
|
19 |
|
|
|
(18 |
) |
|
|
66,112 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Total cash and available for sale securities |
|
$ |
93,773 |
|
|
$ |
19 |
|
|
$ |
(18 |
) |
|
$ |
93,774 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Unrealized gains and losses are reported as a component of accumulated other
comprehensive income/(loss) in stockholders equity. For the year ended December 31, 2010,
unrealized holding gains included in accumulated other comprehensive income was less than $0.1
million. For the three months ended March 31, 2011, unrealized holding gains included in
accumulated other comprehensive income were de minimis.
For the year ended December 31, 2010 and the three months ended March 31, 2011, there
were no realized gains or losses. The cost of securities sold is based on the specific
identification method.
- 11 -
Unrealized loss positions in the available for sale securities as of December 31, 2010 and
March 31, 2011 reflect temporary impairments that have not been recognized and have been in a loss
position for less than twelve months. The fair value of these available for sale securities in
unrealized loss positions was $46.1 million and $31.2 million as of December 31, 2010 and March 31,
2011, respectively.
Note 3. Basic and Diluted Net Loss Attributable to Common Stockholders per Common Share
The Company calculates net loss per share as a measurement of the Companys performance while
giving effect to all dilutive potential common shares that were outstanding during the reporting
period. The Company has a net loss for all periods presented; accordingly, the inclusion of common
stock options would be anti-dilutive. Therefore, the weighted average shares used to calculate both
basic and diluted earnings per share are the same.
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:
|
|
|
|
|
|
|
|
|
|
|
Three Months Ended |
|
|
|
March 31, |
|
(In thousands, except per share amounts) |
|
2010 |
|
|
2011 |
|
Statement of Operations |
|
|
|
|
|
|
|
|
Net loss attributable to common stockholders |
|
$ |
(13,176 |
) |
|
$ |
(13,350 |
) |
Net loss attributable to common stockholders per
common share basic and diluted |
|
$ |
(0.54 |
) |
|
$ |
(0.39 |
) |
Dilutive common stock equivalents would include the dilutive effect of common stock options
and warrants for common stock equivalents. Potentially dilutive common stock equivalents totaled
approximately 6.6 million and 7.7 million for the three months ended March 31, 2010 and 2011,
respectively. Potentially dilutive common stock equivalents were excluded from the diluted earnings
per share denominator for all periods because of their anti-dilutive effect.
Note 4. Comprehensive Loss
The components of comprehensive loss are as follows (in thousands):
|
|
|
|
|
|
|
|
|
|
|
Three Months Ended |
|
|
|
March 31, |
|
|
|
2010 |
|
|
2011 |
|
|
|
|
|
|
|
|
|
Net loss |
|
$ |
(13,176 |
) |
|
$ |
(13,350 |
) |
Change in unrealized net (loss)/gain on marketable
securities |
|
|
(41 |
) |
|
|
29 |
|
|
|
|
|
|
|
|
Comprehensive loss |
|
$ |
(13,217 |
) |
|
$ |
(13,321 |
) |
|
|
|
|
|
|
|
Accumulated other comprehensive loss equals the unrealized net gains and losses on marketable
securities which are the only components of other comprehensive loss included in the Companys
financial statements.
Note 5. Stockholders Equity
Common Stock and Warrants
As of March 31, 2011, the Company was authorized to issue 50,000,000 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.
- 12 -
In October 2010 in connection with the License and Collaboration Agreement, GSK purchased
approximately 6.9 million shares of the Companys common stock at $4.56 per share. The total value
of this equity investment was approximately $31 million and represents a 19.9% ownership position
in the Company.
In March 2010, the Company sold 4.9 million shares of its common stock and warrants to
purchase 1.9 million shares of common stock in a registered direct offering to a selected group of
institutional investors through a Registration Statement on Form S-3 that was declared effective by
the SEC on May 27, 2009. The shares of
common stock and warrants were sold in units consisting of one share of common stock and one
warrant to purchase 0.375 shares of common stock at a price of $3.74 per unit. The warrants have a
term of four years and are exercisable any time on or after the six month anniversary of the date
they were issued, at an exercise price of $4.43 per share. The aggregate offering proceeds were
$18.5 million. Leerink Swann LLC served as sole placement agent for the offering. The Company
intends to use the net proceeds from the sale of the common stock and warrants for general
corporate purposes and to further to advance the development of the Companys lead product
candidate, Amigal, and the completion of certain activities required for the submission of a
license application globally.
Stock Option Plans
During the three months ended March 31, 2011, the Company recorded compensation expense of
approximately $1.4 million. The stock-based compensation expense had no impact on the Companys
cash flows from operations and financing activities. As of March 31, 2011, the total unrecognized
compensation cost related to non-vested stock options granted was $8.9 million and is expected to
be recognized over a weighted average period of 2.5 years.
The fair value of the options granted is estimated on the date of grant using a
Black-Scholes-Merton option pricing model with the following weighted-average assumptions:
|
|
|
|
|
|
|
|
|
|
|
Three Months Ended |
|
|
|
March 31, |
|
|
|
2010 |
|
|
2011 |
|
Expected stock price volatility |
|
|
80.5 |
% |
|
|
78.8 |
% |
Risk free interest rate |
|
|
2.7 |
% |
|
|
2.3 |
% |
Expected life of options (years) |
|
|
6.25 |
|
|
|
6.25 |
|
Expected annual dividend per
share |
|
$ |
0.00 |
|
|
$ |
0.00 |
|
A summary of option activities related to the Companys stock options for the three months
ended March 31, 2011 is as follows:
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Weighted |
|
|
|
|
|
|
|
|
|
|
Weighted |
|
|
Average |
|
|
|
|
|
|
Number |
|
|
Average |
|
|
Remaining |
|
|
Aggregate |
|
|
|
of |
|
|
Exercise |
|
|
Contractual |
|
|
Intrinsic |
|
|
|
Shares |
|
|
Price |
|
|
Life |
|
|
Value |
|
|
|
(in thousands) |
|
|
|
|
|
|
|
|
(in millions) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Balance at December 31, 2010 |
|
|
5,104.1 |
|
|
$ |
7.27 |
|
|
|
|
|
|
|
|
|
Options granted |
|
|
938.8 |
|
|
$ |
5.88 |
|
|
|
|
|
|
|
|
|
Options exercised |
|
|
(7.2 |
) |
|
$ |
4.16 |
|
|
|
|
|
|
|
|
|
Options forfeited |
|
|
(141.2 |
) |
|
$ |
5.74 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Balance at March 31, 2011 |
|
|
5,894.5 |
|
|
$ |
7.09 |
|
|
7.7 years |
|
$ |
9.3 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Vested and unvested expected to vest, March 31, 2011 |
|
|
5,584.9 |
|
|
$ |
7.19 |
|
|
7.6 years |
|
$ |
8.6 |
|
Exercisable at March 31, 2011 |
|
|
3,007.4 |
|
|
$ |
8.58 |
|
|
6.4 years |
|
$ |
3.1 |
|
- 13 -
Note 6. Short-Term Borrowings and Long-Term Debt
In May 2009, the Company entered into a loan and security agreement with Silicon
Valley Bank that provides for up to $4 million of equipment financing through October 2012.
Borrowings under the loan agreement are collateralized by equipment purchased with the
proceeds of the loan and bear interest at a fixed rate of approximately 9%. The loan agreement
contains customary terms and conditions, including a financial covenant whereby the Company
must maintain a minimum amount of liquidity measured at the end of each month equal to the
greater of (i) $30 million of unrestricted cash, cash equivalents, and marketable securities,
or (ii) six months of trailing cash burn net of outstanding borrowings under the loan
agreement. The Company has at all times been in compliance with this covenant during the term
of the agreement.
At March 31, 2011, the current and long-term amounts due under the loan agreement were $1.3
million and $0.7 million, respectively. The carrying amount of the Companys borrowings
approximates fair value at March 31, 2011.
Note 7. Assets and Liabilities Measured at Fair Value
The Companys financial assets and liabilities are measured at fair value and classified
within the fair value hierarchy which is defined as follows:
|
|
|
Level 1 Quoted prices in active markets for identical assets or liabilities that the
Company has the ability to access at the measurement date. |
|
|
|
Level 2 Inputs other than quoted prices in active markets that are observable for
the asset or liability, either directly or indirectly. |
|
|
|
Level 3 Inputs that are unobservable for the asset or liability. |
Cash, Money Market Funds and Marketable Securities
The Company classifies its cash and money market funds within the fair value hierarchy as
Level 1 as these assets are valued using quoted prices in active market for identical assets at the
measurement date. The Company considers its investments in marketable securities as available for
sale and classifies these assets within the fair value hierarchy as Level 2 primarily utilizing
broker quotes in a non-active market for valuation of these securities. No changes in valuation
techniques or inputs occurred during the three months ended March 31, 2011. No transfers of assets
between Level 1 and Level 2 of the fair value measurement hierarchy occurred during the three
months ended March 31, 2011.
Secured Debt
As disclosed in Note 6, the Company has a loan and security agreement with Silicon Valley
Bank. The carrying amount of the Companys borrowings approximates fair value at March 31, 2011.
The Companys secured debt is classified as Level 2 and the fair value is estimated using quoted
prices for similar liabilities in active markets, as well as inputs that are observable for the
liability (other than quoted prices), such as interest rates that are observable at commonly quoted
intervals.
- 14 -
Warrants
The Company allocated $3.3 million of proceeds from its March 2010 registered direct offering
to warrants issued in connection with the offering that was classified as a liability. The
valuation of the warrants is determined using the Black-Scholes model. This model uses inputs such
as the underlying price of the shares issued when the warrant is exercised, volatility, risk free
interest rate and expected life of the instrument. The Company has determined that the warrant
liability should be classified within Level 3 of the fair value hierarchy by evaluating each input
for the Black Scholes model against the fair value hierarchy criteria and using the lowest level of
input as the basis for the fair value classification. There are six inputs: closing price of Amicus
stock on the day of evaluation; the exercise price of the warrants; the remaining term of the
warrants; the volatility of Amicus stock over that term; annual rate of dividends; and the
riskless rate of return. Of those inputs, the exercise price of the warrants and the remaining term
are readily observable in the warrant agreements. The annual rate of dividends is based on the
Companys historical practice of not granting dividends. The closing price of Amicus stock would
fall under Level 1 of the fair value hierarchy as it is a quoted price in an active market. The
riskless rate of return is a Level 2, while the historical volatility is a Level 3 input in
accordance with the fair value accounting guidance. Since the lowest level input is a Level 3, the
Company determined the warrant liability is most appropriately classified within Level 3 of the
fair value hierarchy. This liability is subject to fair value mark-to-market adjustment each
period. As a result, for the three month period ended March 31, 2011, the Company recognized the
change in the fair value of the warrant liability as a non-operating expense of $3.4 million. The
resulting fair value of the warrant liability at March 31, 2011, was $8.1 million.
A summary of the fair value of the Companys assets and liabilities aggregated by the level in
the fair value hierarchy within which those measurements fall as of March 31, 2011, are identified
in the following table (in thousands):
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Level 1 |
|
|
Level 2 |
|
|
Total |
|
Assets: |
|
|
|
|
|
|
|
|
|
|
|
|
Cash/Money market funds |
|
$ |
27,662 |
|
|
$ |
|
|
|
$ |
27,662 |
|
U.S. government agency securities |
|
|
|
|
|
|
2,999 |
|
|
|
2,999 |
|
Corporate debt securities |
|
|
|
|
|
|
43,519 |
|
|
|
43,519 |
|
Commercial paper |
|
|
|
|
|
|
19,244 |
|
|
|
19,244 |
|
Certificate of deposit |
|
|
|
|
|
|
350 |
|
|
|
350 |
|
|
|
|
|
|
|
|
|
|
|
|
|
$ |
27,662 |
|
|
$ |
66,112 |
|
|
$ |
93,774 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Level 1 |
|
|
Level 2 |
|
|
Level 3 |
|
|
Total |
|
Liabilities: |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Secured debt |
|
$ |
|
|
|
$ |
1,984 |
|
|
$ |
|
|
|
$ |
1,984 |
|
Warrants liability |
|
|
|
|
|
|
|
|
|
|
8,143 |
|
|
|
8,143 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
$ |
|
|
|
$ |
1,984 |
|
|
$ |
8,143 |
|
|
$ |
10,127 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
The following table summarizes the changes in Level 3 liability for the three months
ended March 31, 2011 (in thousands):
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Balance as of |
|
|
|
|
|
|
Balance as of |
|
|
|
December 31, |
|
|
Increase/(decrease) |
|
|
March 31, |
|
|
|
2010 |
|
|
in fair value |
|
|
2011 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Warrants liability |
|
$ |
4,712 |
|
|
$ |
3,432 |
|
|
$ |
8,144 |
|
|
|
|
|
|
|
|
|
|
|
- 15 -
Note 8. Collaborative Agreements
GSK
On October 28, 2010, the Company entered into the License and Collaboration Agreement with
Glaxo Group Limited, an affiliate of GSK, to develop and commercialize Amigal. Under the terms of
the License and Collaboration Agreement, GSK received an exclusive worldwide license to develop,
manufacture and commercialize Amigal. In consideration of the license grant, the Company received
an upfront, license payment of $30 million from GSK and is eligible to receive further payments of
approximately $170 million upon the successful achievement of development and commercialization
milestones, as well as tiered double-digit royalties on global sales of Amigal. GSK and the Company
will jointly fund development costs in accordance with an agreed upon development plan. This plan
provides that the Company will fund 50% of the development costs for 2011 and 25% of the
development costs in 2012 and beyond. The Companys development costs are subject to annual and
aggregate caps. Additionally, GSK purchased approximately 6.9 million shares of the Companys
common stock at $4.56 per share. The total value of this equity investment to the Company was
approximately $31 million and represents a 19.9% ownership position in the Company. Under the terms
of the collaboration agreement, while the Company will collaborate with GSK, GSK will have
decision-making authority over clinical, regulatory and commercial matters. Additionally, GSK will
have primary responsibility for interactions with regulatory agencies and prosecuting applications
for marketing and reimbursement approvals worldwide.
In accordance with the revenue recognition guidance related to multiple-element arrangements,
the Company identified all of the deliverables at the inception of the agreement. The significant
deliverables were determined to be the worldwide licensing rights to Amigal, the technology and
know how transfer of Amigal development to date, the delivery of the Companys common stock and
the research services to continue and complete the development of Amigal. The Company determined
that the worldwide licensing rights, the technology and know how transfer together with the
research services represent one unit of accounting as none of these three deliverables on its own
has standalone value separate from the other. The Company also determined that the delivery of the
Companys common stock does have standalone value separate from the worldwide licensing rights, the
technology and know how transfer and the research services. As a result, the Companys common
stock is considered a separate unit of accounting and was accounted for as an issuance of common
stock. However, as the Companys common stock was sold at a premium to the market closing price,
the premium amount paid over the market closing price was considered as additional consideration
paid to the Company for the collaboration agreement and was included as consideration for the
single unit of accounting identified above.
The total arrangement consideration which was allocated to the single unit of accounting
identified above was $33.2 million which consists of the upfront license payment of $30 million and
the premium over the closing market price of the common stock transaction of $3.2 million. The
Company will recognize this consideration as Collaboration Revenue on a straight-line basis over
the development period of 5.2 years as included in the detailed development plan that was included
in the collaboration agreement. The Company determined that the overall level of activity over the
development period approximates a straight-line approach. At March 31, 2011, the Company recognized
approximately $1.7 million of the total arrangement consideration as Collaboration Revenue.
The revenue associated with reimbursements for research and development costs under
collaboration agreements is included in Research Revenue and the costs associated with these
reimbursable amounts are included in research and development expenses. The Company records
these reimbursements as revenue and not as a reduction of research and development expenses as
the Company has not commenced its planned principal operations (i.e., selling commercial
products) and is a development stage enterprise, therefore development activities are part of
its ongoing central operations. During the three months ended March 31, 2011, the Company
recorded $4.3 million in Research Revenue. As of March 31, 2011, the Company recorded $4.0
million of current receivable due from GSK related to reimbursed research and development
costs.
- 16 -
The Company evaluated the contingent milestones included in the collaboration agreement at the
inception of the collaboration agreement and determined that the contingent milestones are
substantive milestones and will be recognized as revenue in the period that the milestone is
achieved. The Company determined that the research based
milestones are commensurate with the enhanced value of each delivered item as a result of the
Companys specific performance to achieve the milestones. There is considerable effort underway to
meet the specified milestones and complete the development of Amigal. Additionally, there is
considerable time and effort involved in evaluating the data from the clinical trials that are
planned and underway and if acceptable, in preparing the documentation required for filing for
approval with the applicable regulatory authorities. The research based milestones would relate to
past performances when achieved and are reasonable relative to the other payment terms within the
collaboration agreement, including the $30 million upfront payment and the cost sharing
arrangement.
Note 9. Restructuring Charges
In October 2009, the Company announced a work-force reduction of approximately 20 percent, or
26 employees, as a part of a corporate restructuring, with reductions occurring across all levels
and organizations within the Company. This measure was intended to reduce costs and to align the
Companys resources with its key strategic priorities. The Company recorded restructuring charges
of $0.9 million during the fourth quarter of 2009 for employment termination costs payable in cash
in connection with the workforce reduction. There were no restructuring charges related to
employment termination costs unpaid at March 31, 2011. There were no additional restructuring costs
incurred through March 31, 2011.
In December 2009, the Company initiated and completed a facilities consolidation effort,
closing one of its subleased locations in Cranbury, NJ. The Company recorded a charge of $0.7
million during the fourth quarter of 2009 for minimum lease payments of $0.5 million and the
write-down of fixed assets in the facility.
The following table summarizes the restructuring charges and utilization for the three months
ended March 31, 2011 (in thousands):
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Balance |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Balance |
|
|
|
as of |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
as of |
|
|
|
December 31, |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
March 31, |
|
|
|
2010 |
|
|
Charges |
|
|
Cash Payments |
|
|
Adjustments |
|
|
2011 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Facilities consolidation |
|
$ |
268 |
|
|
|
|
|
|
$ |
(57 |
) |
|
|
|
|
|
$ |
211 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
- 17 -
|
|
|
ITEM 2. |
|
MANAGEMENTS DISCUSSION AND ANALYSIS OF FINANCIAL CONDITION AND RESULTS OF
OPERATIONS |
Overview
Amicus Therapeutics, Inc. (Amicus) is a biopharmaceutical company focused on the discovery,
development and commercialization of orally-administered, small molecule drugs known as
pharmacological chaperones for the treatment of rare diseases. Pharmacological chaperones are a
novel, first-in-class approach to treating a broad range of diseases including lysosomal storage
disorders and diseases of neurodegeneration. Our current areas of focus include the following:
|
|
|
the Phase 3 development of our lead product candidate, Amigal for Fabry disease; |
|
|
|
the preclinical and clinical development of pharmacological chaperones co-administered
with enzyme replacement therapy; and |
|
|
|
the preclinical evaluation of the use of pharmacological chaperones for
neurodegenerative diseases. |
Our novel approach to the treatment of human genetic diseases consists of using
pharmacological chaperones that selectively bind to the target protein; increasing the
stability of the protein and helping it fold into the correct three-dimensional shape. This
allows proper trafficking of the protein within the cell, thereby increasing protein activity,
improving cellular function and potentially reducing cell stress. We have also demonstrated in
preclinical studies that pharmacological chaperones can further stabilize normal, or
wild-type proteins. This stabilization could lead to a higher percentage of the target
proteins folding correctly and more stably, which can increase cellular levels of that target
protein and improve cellular function, making chaperones potentially applicable to a wide
range of diseases.
Our lead product candidate, Amigal (migalastat hydrochloride) for Fabry disease, is in Phase 3
development. We are developing and commercializing Amigal with an affiliate of GlaxoSmithKline PLC
(GSK) pursuant to a License and Collaboration Agreement entered into in October 2010. Our
partnership with GSK allows us to utilize GSKs significant expertise in clinical, regulatory,
commercial and manufacturing matters in the development in Amigal. In addition, the cost-sharing
arrangements and potential milestone and royalty payments under the License and Collaboration
Agreement provide us with financial strength and allow us to continue the development of Amigal
while also advancing our other programs. We also believe this collaboration is important in
validating our status as a leader in the development of treatments for rare diseases given the
increasing focus placed on the rare disease field.
Our Phase 3 clinical development program for the use of Amigal as monotherapy in Fabry disease
includes two clinical trials: Study 011 and Study 012. We have enrolled a majority of the planned
60 patients for Study 011, and have commenced an additional Phase 3 study (Study 012). We plan to
use the data from Study 011 to support the filing of a New Drug Application, or NDA, for marketing
approval in the United States and the data from Study 012 to support the filing of an application
for marketing authorization in Europe.
While our initial clinical efforts have focused on the use of pharmacological chaperones to
treat lysosomal storage diseases, we believe that our technology may be applicable to the treatment
of certain diseases of neurodegeneration. Our lead preclinical program in this area is focused on
Parkinsons disease, where we expect to complete late-stage preclinical proof of concept studies,
including IND-enabling activities, for our pharmacological chaperone molecule AT3375 during 2011.
Our second preclinical program in this area is focused on Alzheimers disease. Our preclinical work
in both Parkinsons and Alzheimers disease is presently focused on genetically-defined
subpopulations of Parkinsons and Alzheimers patients and leverages our expertise and knowledge in
the rare disease field.
We have generated significant losses to date and expect to continue to generate losses as we
continue the clinical development of our drug candidates, including Amigal, and conduct preclinical
studies on other programs. These activities are budgeted to expand over time and will require
further resources if we are to be successful. From our inception in February 2002 through March 31,
2011, we have accumulated a deficit of $239.0 million. As we
have not yet generated commercial sales revenue from any of our product candidates, our losses
will continue and are likely to be substantial over at least the next couple of years.
- 18 -
In June 2007, we completed our initial public offering (IPO) of 5,000,000 shares of common
stock at a public offering price of $15.00 per share. Net cash proceeds from the initial public
offering were approximately $68.1 million. In March 2010, we sold 4.95 million shares of our common
stock and warrants to purchase 1.85 million shares of common stock in a registered direct offering
to a select group of institutional investors. The shares of common stock and warrants were sold in
units consisting of one share of common stock and one warrant to purchase 0.375 shares of common
stock at a price of $3.74 per unit. The warrants have a term of four years and are exercisable any
time on or after the six month anniversary of the date they were issued, at an exercise price of
$4.43 per share. The net proceeds of the offering were $17.1 million.
As previously disclosed, on April 18, 2011, John F. Crowley resigned as Chairman and Chief
Executive Officer of the Company and was appointed by the Board of Directors of the Company (the
Board) as Executive Chairman, He will serve as Executive Chairman until September 30, 2011,
subject to an extension of up to three months if he and the Company agree. Our Board has appointed
Matthew R. Patterson as Acting Chief Executive Officer and Principal Executive Officer of the
Company, in addition to his duties as President of the Company. Our Board intends to shortly
initiate a search for a permanent Chief Executive Officer, and will consider Mr. Patterson as a
candidate for the position.
Collaboration with GSK
On October 28, 2010, the Company entered into the License and Collaboration Agreement with
Glaxo Group Limited, an affiliate of GSK, to develop and commercialize Amigal. Under the terms of
the License and Collaboration Agreement, GSK received an exclusive worldwide license to develop,
manufacture and commercialize Amigal. In consideration of the license grant, the Company received
an upfront, license payment of $30 million from GSK and is eligible to receive further payments of
approximately $170 million upon the successful achievement of development and commercialization
milestones, as well as tiered double-digit royalties on global sales of Amigal. GSK and the Company
will jointly fund development costs in accordance with an agreed upon development plan. This plan
provides that the Company will fund 50% of the development costs for 2011 and 25% of the
development costs in 2012 and beyond. The Companys development costs are subject to annual and
aggregate caps. Additionally, GSK purchased approximately 6.9 million shares of the Companys
common stock at a price of $4.56 per share. The total value of this equity investment to the
Company is approximately $31 million and represents a 19.9% ownership position in the Company.
Under the terms of the collaboration agreement, while we will collaborate with GSK, GSK will have
decision-making authority over clinical, regulatory and commercial matters related to Amigal.
Additionally, GSK will have primary responsibility for interactions with regulatory agencies and
prosecuting applications for marketing and reimbursement approvals worldwide.
Financial Operations Overview
Revenue
In November 2010, GSK paid us an initial, non-refundable license fee of $30 million and a
premium of $3.2 million related to GSKs purchase of an equity investment in Amicus. The total
upfront consideration received of $33.2 million will be recognized as Collaboration Revenue on a
straight-line basis over the development period of the collaboration agreement which is
approximately 5.2 years. At March 31, 2011, we recognized approximately $1.7 million of the total
upfront consideration as Collaboration Revenue. For the three months ended March 31, 2011, we
recognized $4.3 million of Research Revenue for reimbursed research and development costs. We have
not generated any commercial sales revenue since our inception.
- 19 -
Research and Development Expenses
We expect to continue to incur substantial research and development expenses as we continue to
develop our product candidates and explore new uses for our pharmacological chaperone technology.
However, we will share
future research and development costs related to Amigal with GSK in accordance with the
License and Collaboration Agreement. Research and development expense consists of:
|
|
|
internal costs associated with our research and clinical development activities; |
|
|
|
payments we make to third party contract research organizations, contract manufacturers,
investigative sites, and consultants; |
|
|
|
technology license costs; |
|
|
|
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, facility maintenance, 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. We 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,
2011, we have incurred research and development expense in the aggregate of $225.9 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).
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Period from |
|
|
|
|
|
|
|
|
|
|
|
February 4, 2002 |
|
|
|
Three Months Ended |
|
|
(inception) to |
|
|
|
March 31, |
|
|
March 31, |
|
Projects |
|
2010 |
|
|
2011 |
|
|
2011 |
|
Third party direct project expenses |
|
|
|
|
|
|
|
|
|
|
|
|
Amigal (Fabry Disease Phase 3) |
|
$ |
2,563 |
|
|
$ |
4,062 |
|
|
$ |
50,092 |
|
Plicera (Gaucher Disease Phase 2*) |
|
|
253 |
|
|
|
(200 |
) |
|
|
26,027 |
|
AT2220 (Pompe Disease Phase 1) |
|
|
176 |
|
|
|
4 |
|
|
|
13,138 |
|
Neurodegenerative Diseases
(Preclinical) |
|
|
349 |
|
|
|
451 |
|
|
|
6,850 |
|
|
|
|
|
|
|
|
|
|
|
Total third party direct project expenses |
|
|
3,341 |
|
|
|
4,317 |
|
|
|
96,107 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Other project costs (1) |
|
|
|
|
|
|
|
|
|
|
|
|
Personnel costs |
|
|
4,181 |
|
|
|
4,923 |
|
|
|
79,360 |
|
Other costs (2) |
|
|
1,367 |
|
|
|
1,885 |
|
|
|
50,422 |
|
|
|
|
|
|
|
|
|
|
|
Total other project costs |
|
|
5,548 |
|
|
|
6,808 |
|
|
|
129,782 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Total research and development costs |
|
$ |
8,889 |
|
|
$ |
11,125 |
|
|
$ |
225,889 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
(1) |
|
Other project costs are leveraged across multiple projects. |
|
(2) |
|
Other costs include facility, supply, overhead, and licensing costs that support multiple
clinical and preclinical projects. |
|
* |
|
We do not plan to advance Plicera into Phase 3 development at this time. |
- 20 -
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 our product candidates. As a result, we
are not able to reasonably estimate the period, if any, in which material net cash inflows may
commence from our product candidates, including Amigal or any of our other
preclinical product candidates. This uncertainty is due to the numerous risks and
uncertainties associated with the conduct, duration and cost of clinical trials, which vary
significantly over the life of a project as a result of evolving events 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; |
|
|
|
the results of our clinical trials; and |
|
|
|
any mandate by the U.S. Food and Drug Administration (FDA) or other regulatory authority
to conduct clinical trials beyond those currently anticipated. |
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. In addition, GSK has considerable influence over and decision-making authority
related to our Amigal program. 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, regulatory approval and commercialization 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, or if we experience significant delays
in enrollment in any of 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, legal, information technology and human resource functions. Other general and
administrative expense includes facility-related costs not otherwise included in research and
development expense, promotional expenses, costs associated with industry and trade shows, and
professional fees for legal services, including patent-related expense and accounting services.
From our inception in February 2002 through March 31, 2011, we spent $97.8 million on general and
administrative expense.
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 and our
equipment financing agreement.
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 there were no significant changes during the quarter ended March 31, 2011 to the items
that we disclosed as our significant accounting policies and estimates described in Note 2 to the
Companys financial statements as contained in the Companys Annual Report on Form 10-K for the
year ended December 31, 2010, 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.
- 21 -
Revenue Recognition
We recognize revenue when amounts are realized or realizable and earned. Revenue is considered
realizable and earned when the following criteria are met: (1) persuasive evidence of an
arrangement exists; (2) delivery has occurred or services have been rendered; (3) the price is
fixed or determinable; and (4) collection of the amounts due are reasonably assured.
In multiple element arrangements, revenue is allocated to each separate unit of accounting and
each deliverable in an arrangement is evaluated to determine whether it represents separate units
of accounting. A deliverable constitutes a separate unit of accounting when it has standalone value
and there is no general right of return for the delivered elements. In instances when the
aforementioned criteria are not met, the deliverable is combined with the undelivered elements and
the allocation of the arrangement consideration and revenue recognition is determined for the
combined unit as a single unit of accounting. Allocation of the consideration is determined at
arrangement inception on the basis of each units relative selling price. In instances where there
is determined to be a single unit of accounting, the total consideration is applied as revenue for
the single unit of accounting and is recognized over the period of inception through the date where
the last deliverable within the single unit of accounting is expected to be delivered.
Our current revenue recognition policies, which were applied in fiscal 2010, provide that,
when a collaboration arrangement contains multiple deliverables, such as license and research and
development services, we allocate revenue to each separate unit of accounting based on a selling
price hierarchy. The selling price hierarchy for a deliverable is based on (i) its vendor specific
objective evidence (VSOE) if available, (ii) third party evidence (TPE) if VSOE is not available,
or (iii) estimated selling price (BESP) if neither VSOE nor TPE is available. We would establish
the VSOE of selling price using the price charged for a deliverable when sold separately. The TPE
of selling price would be established by evaluating largely similar and interchangeable competitor
products or services in standalone sales to similarly situated customers. The best estimate of
selling price would be established considering internal factors such as an internal pricing
analysis or an income approach using a discounted cash flow model.
The revenue associated with reimbursements for research and development costs under
collaboration agreements is included in Research Revenue and the costs associated with these
reimbursable amounts are included in research and development expenses. We record these
reimbursements as revenue and not as a reduction of research and development expenses as we have
not commenced our planned principal operations (i.e., selling commercial products) and we are a
development stage enterprise, therefore development activities are part of our ongoing central
operations.
Our collaboration agreement with GSK provides for, and any future collaboration agreements we
may enter into also may provide for contingent milestone payments. In order to determine the
revenue recognition for these contingent milestones, we evaluate the contingent milestones using
the criteria as provided by the FASB guidance on the milestone method of revenue recognition at the
inception of a collaboration agreement. The criteria requires that (i) we determine if the
milestone is commensurate with either our performance to achieve the milestone or the enhancement
of value resulting from our activities to achieve the milestone, (ii) the milestone be related to
past performance, and (iii) the milestone be reasonable relative to all deliverable and payment
terms of the collaboration arrangement. If these criteria are met then the contingent milestones
can be considered as substantive milestones and will be recognized as revenue in the period that
the milestone is achieved.
- 22 -
Accrued Expenses
When we are required to estimate accrued expenses because we have not yet been invoiced
or otherwise notified of actual cost, we identify services that have been performed on our behalf
and estimate the level of service performed and the associated cost incurred. 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 owed 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. |
Stock-Based Compensation
We adopted the fair value method of measuring stock-based compensation, which requires a
public entity to measure the cost of employee services received in exchange for an award of equity
instruments based upon the grant-date fair value of the award. We chose the straight-line
attribution method for allocating compensation costs and recognized the fair value of each stock
option on a straight-line basis over the vesting period of the related awards.
We use the Black-Scholes option pricing model when estimating the value for stock-based
awards. 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 using 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:
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|
|
|
|
|
|
|
|
Three Months Ended |
|
|
|
March 31, |
|
|
|
2010 |
|
|
2011 |
|
Expected stock price volatility |
|
|
80.5 |
% |
|
|
78.8 |
% |
Risk free interest rate |
|
|
2.7 |
% |
|
|
2.3 |
% |
Expected life of options (years) |
|
|
6.25 |
|
|
|
6.25 |
|
Expected annual dividend per
share |
|
$ |
0.00 |
|
|
$ |
0.00 |
|
- 23 -
Warrants
The warrants issued in connection with the March 2010 registered direct offering are
classified as a liability. The fair value of the warrants liability is evaluated at each balance
sheet date using the Black-Scholes valuation model. This model uses inputs such as the underlying
price of the shares issued when the warrant is exercised, volatility, risk free interest rate and
expected life of the instrument. Any changes in the fair value of the warrants liability is
recognized in the consolidated statement of operations. The weighted average assumptions used in
the Black-Scholes valuation model for the warrants March 31, 2011 and 2010 are as follows:
|
|
|
|
|
|
|
|
|
|
|
March 31, 2010 |
|
|
March 31, 2011 |
|
Expected stock price volatility |
|
|
80.9 |
% |
|
|
77.9 |
% |
Risk free interest rate |
|
|
1.99 |
% |
|
|
1.24 |
% |
Expected life of warrants (years) |
|
|
3.92 |
|
|
|
2.92 |
|
Expected annual dividend per
share |
|
$ |
0.00 |
|
|
$ |
0.00 |
|
Basic and Diluted Net Loss Attributable to Common Stockholders per Common Share
We calculated net loss per share as a measurement of the Companys performance while
giving effect to all dilutive potential common shares that were outstanding during the reporting
period. We had 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.
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:
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|
|
|
|
|
|
|
|
|
Three Months Ended |
|
|
|
March 31, |
|
(In thousands, except per share amount) |
|
2010 |
|
|
2011 |
|
Historical |
|
|
|
|
|
|
|
|
Numerator: |
|
|
|
|
|
|
|
|
Net loss attributable to common
stockholders |
|
$ |
(13,176 |
) |
|
$ |
(13,350 |
) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Denominator: |
|
|
|
|
|
|
|
|
Weighted average common shares
outstanding basic and diluted |
|
|
24,289,422 |
|
|
|
34,498,926 |
|
|
|
|
|
|
|
|
Dilutive common stock equivalents would include the dilutive effect of common stock options
and warrants for common stock equivalents. Potentially dilutive common stock equivalents totaled
approximately 6.6 million and 7.7 million for the three months ended March 31, 2010 and 2011,
respectively. Potentially dilutive common stock equivalents were excluded from the diluted earnings
per share denominator for all periods because of their anti-dilutive effect.
- 24 -
Results of Operations
Three Months Ended March 31, 2011 Compared to Three Months Ended March 31, 2010
Revenue. In November 2010, GSK paid us an initial, non-refundable license fee of $30 million
and a premium of $3.2 million related to GSKs purchase of an equity investment in Amicus. The
total upfront consideration received of $33.2 million will be recognized as Collaboration Revenue
on a straight-line basis over the development period of the collaboration agreement which is
approximately 5.2 years. For the three months ended March 31, 2011, we recognized $1.7 million of
the total upfront consideration as Collaboration Revenue. For the three months ended March 31,
2011, we recognized $4.3 million of Research Revenue for reimbursed research and development costs.
We have not generated any commercial sales revenue since our inception.
Research and Development Expense. Research and development expense was $11.1 million for the
three months ended March 31, 2011, representing an increase of $2.2 million or 25% from $8.9
million for the three months ended March 31, 2010. The variance was primarily attributable to
higher personnel costs, an increase in consulting costs and an increase in contract research and
manufacturing costs due to the increased activity within the Fabry program.
General and Administrative Expense. General and administrative expense was $4.4 million for
the three months ended March 31, 2011, representing a increase of $0.5 million or 13% from $3.9
million for the three months ended March 31, 2010. The variance was primarily due to higher
personnel costs, and an increase in third party legal and consulting fees.
Interest Income and Interest Expense. Interest income was $0.1 million for the three months
ended March 31, 2011, and 2010. Interest expense was approximately $0.05 million for the three
months ended March 31, 2011 compare to $0.1 for the three months ended March 31, 2010. The decrease
was due to less outstanding debt during the period on the secured loan.
Change in Fair Value of Warrant Liability. In connection with the sale of our common stock and
warrants from the registered direct offering in March 2010, we recorded the warrants as a liability
at their fair value using a Black-Scholes model and remeasure the fair value at each reporting date
until exercised or expired. Changes in the fair value of the warrants are reported in the
statements of operations as non-operating income or expense. For the three months ended March 31,
2011, we reported a expense of $3.4 million related to the increase in fair value of these warrants
from issuance dates. The market price for our common stock has been and may continue to be
volatile. Consequently, future fluctuations in the price of our common stock may cause significant
increases or decreases in the fair value of these warrants.
Other Income/Expense. Other income increased due to funds received from the U.S. Treasury
Department in February 2011 of $0.07 million for the Qualified Therapeutic Discovery Projects tax
credit and grant program.
- 25 -
Liquidity and Capital Resources
Source of Liquidity
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 $148.7 million of proceeds from redeemable convertible preferred stock
offerings, $75.0 million of gross proceeds from our IPO in June 2007, $18.5 million of gross
proceeds from our Registered Direct Offering in March 2010, $80.0 million from the
non-refundable license fees from the collaboration agreements and $31.0 million from GSKs
investment in the Company at the time the collaboration was formed. In the future, we expect
to fund our operations, in part, through the receipt of cost-sharing and milestone payments
from GSK. The following table summarizes our significant funding sources as of March 31, 2011:
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|
|
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|
|
|
|
|
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|
Approximate |
|
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|
|
|
|
|
|
|
|
Amount(1) |
|
Funding |
|
Year |
|
|
No. Shares |
|
|
(in thousands) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Series A Redeemable
Convertible Preferred Stock |
|
|
2002 |
|
|
|
444,443 |
|
|
$ |
2,500 |
|
Series B Redeemable
Convertible Preferred Stock |
|
|
2004, 2005, 2006, 2007 |
|
|
|
4,917,853 |
|
|
|
31,189 |
|
Series C Redeemable Convertible Preferred Stock |
|
|
2005, 2006 |
|
|
|
5,820,020 |
|
|
|
54,999 |
|
Series D Redeemable Convertible Preferred Stock |
|
|
2006, 2007 |
|
|
|
4,930,405 |
|
|
|
60,000 |
|
Common Stock |
|
|
2007 |
|
|
|
5,000,000 |
|
|
|
75,000 |
|
Upfront License Fee from Shire |
|
|
2007 |
|
|
|
|
|
|
|
50,000 |
|
Registered Direct Offering |
|
|
2010 |
|
|
|
4,946,525 |
|
|
|
18,500 |
|
Upfront License Fee from GSK |
|
|
2010 |
|
|
|
|
|
|
|
30,000 |
|
Common Stock GSK |
|
|
2010 |
|
|
|
6,866,245 |
|
|
|
31,285 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
32,925,491 |
|
|
$ |
353,473 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
(1) |
|
Represents gross proceeds |
In addition, in conjunction with the GSK collaboration agreement, we received
reimbursement of research and development expenditures from the date of the agreement (October 28,
2010) through March 31, 2011 of $4.3 million. We also received $31.1 million in reimbursement of
research and development expenditures from the Shire collaboration from the date of the agreement
(November 7, 2007) through year-end 2009.
As of March 31, 2011, we had cash, cash equivalents and marketable securities of $93.8
million. We invest cash in excess of our immediate requirements with regard to liquidity and
capital preservation in a variety of interest-bearing instruments, including obligations of U.S.
government agencies and money market accounts. Wherever possible, we seek to minimize the potential
effects of concentration and degrees of risk. Although 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 Operating Activities
Net cash used in operations for the three months ended March 31, 2010 was $13.4 million
due to the net loss for the three months ended March 31, 2010 of $13.2 million and the change in
other operating assets and liabilities of $2.3 million which primarily consisted of the change in
accounts payable and accrued expenses of $2.2 million.
Net cash used in operations for the three months ended March 31, 2011 was $13.3 million due to
the net loss for the three months ended March 31, 2011 of $13.4 million. There were also increases
in receivables of $4.0 million related to amounts due from GSK from the collaboration agreement and
other operating assets and liabilities of $1.2 million.
- 26 -
Net Cash Provided By Investing Activities
Net cash provided by investing activities for the three months ended March 31, 2010 was $10.9
million. Net cash provided by investing activities reflects $19.8 million for the sale and
redemption of marketable securities, partially offset by $8.8 million for the purchase of
marketable securities and $0.1 million for the acquisition of property and equipment.
Net cash provided by investing activities for the three months ended March 31, 2011 was $11.7
million. Net cash provided by investing activities reflects $30.9 million for the sale and
redemption of marketable securities partially offset by $19.1 million for the purchase of
marketable securities and $0.1 million for the acquisition of property and equipment.
Net Cash Provided by/(Used in) Financing Activities
Net cash provided by financing activities for the three months ended March 31, 2010 was
$16.7 million, consisting of $17.1 million from the issuance of common stock primarily offset by
the payments of our secured loan agreement and capital lease obligations of $0.3 million and $0.1
million, respectively.
Net cash used in financing activities for the three months ended March 31, 2011 was $0.3
million, consisting primarily of payments of our secured loan agreement and capital lease
obligations.
Funding Requirements
We expect to incur losses from operations for the foreseeable future primarily due to research
and development expenses, including expenses related to conducting clinical trials. Our future
capital requirements will depend on a number of factors, including:
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|
|
the progress and results of our clinical trials of our drug candidates, including
Amigal; |
|
|
|
our ability to achieve development and commercialization milestone payments and sales
royalties under our collaboration with GSK; |
|
|
|
the scope, progress, results and costs of preclinical development, laboratory
testing and clinical trials for our product candidates including those testing the use
of pharmacological chaperones co-administered with ERT and for the treatment of
diseases of neurodegeneration; |
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|
|
the costs, timing and outcome of regulatory review of our product candidates; |
|
|
|
the number and development requirements of other product candidates that we pursue; |
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|
|
the costs of commercialization activities, including product marketing, sales and
distribution; |
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|
|
the emergence of competing technologies and other adverse market developments; |
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|
|
the costs of preparing, filing and prosecuting patent applications and maintaining,
enforcing and defending intellectual property related claims; |
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|
the extent to which we acquire or invest in businesses, products and technologies;
and |
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|
our ability to establish collaborations and obtain milestone, royalty or other payments
from any such collaborators. |
We do not anticipate that we will generate revenue from commercial sales until at least 2013,
if at all. 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.
However, we believe that our existing cash and cash equivalents and short-term investments,
including anticipated payments from GSK in connection with the collaboration, is expected to be
sufficient to fund our operating expenses and capital expenditure requirements through the
anticipated commercial launch of Amigal in the United States.
- 27 -
Financial Uncertainties Related to Potential Future Payments
Milestone Payments
We have acquired rights to develop and commercialize our product candidates through licenses
granted by various parties. While our license agreements for Amigal and AT2220 do not contain
milestone payment obligations, two of these agreements related to Plicera do require us to make
such payments if certain specified pre-commercialization events occur. Upon the satisfaction of
certain milestones and assuming successful development of Plicera, we may be obligated, under the
agreements that we have in place, to make future milestone payments aggregating up to approximately
$7.9 million. However, such potential milestone payments are subject to many uncertain variables
that would cause such payments, if any, to vary in size.
Royalties
Under our license agreements, if we owe royalties on net sales for one of our products to more
than one licensor, 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 (MSSM). We would expect to pay royalties to all three licensors with
respect to Plicera should we advance Plicera to commercialization. To date, we have not made any
royalty payments on sales of our products and believe we are at least a couple years away from
selling any products that would require us to make any such royalty payments.
In accordance with our license agreement with MSSM, we paid $3 million of the $30 million
upfront payment received from GSK to MSSM in the fourth quarter of 2010. We will also be obligated
to pay MSSM royalties on worldwide net sales of Amigal.
Whether we will be obligated to make milestone or royalty payments in the future is
subject to the success of our product development efforts and, accordingly, is inherently
uncertain.
|
|
|
ITEM 3. |
|
Quantitative and Qualitative Disclosures about Market Risk |
Market risk is the risk of change in fair value of a financial instrument due to changes
in interest rates, equity prices, creditworthiness, financing, exchange rates or other factors. Our
primary market risk exposure relates to changes in interest rates in our cash, cash equivalents and
marketable securities. We place our investments in high-quality financial instruments, primarily
money market funds, corporate debt securities, asset backed securities and U.S. government agency
notes with maturities of less than one year, which we believe are subject to limited interest rate
and credit risk. The securities in our investment portfolio are not leveraged, are classified as
available-for-sale and, due to the short-term nature, are subject to minimal interest rate risk. We
currently do not hedge interest rate exposure and consistent with our investment policy, we do not
use derivative financial instruments in our investment portfolio. At March 31, 2011, we held $93.8
million in cash, cash equivalents and available for sale securities and due to the short-term
maturities of our investments, we do not believe that a 10% change in average interest rates would
have a significant impact on our interest income. Our outstanding debt has a fixed interest rate
and therefore, we have no exposure to interest rate fluctuations.
We have operated primarily in the U.S., although we do conduct some clinical activities
outside the U.S. While most expenses are paid in U.S. dollars, there are minimal payments made in
local foreign currency. If exchange rates undergo a change of 10%, we do not believe that it would
have a material impact on our results of operations or cash flows.
- 28 -
|
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ITEM 4. |
|
CONTROLS AND PROCEDURES |
As of the end of the period covered by this Quarterly Report on Form 10-Q, an evaluation of
the effectiveness of our disclosure controls and procedures (pursuant to Rule 13a-15(e) and
15d-15(e) under the Securities Exchange Act of 1934, as amended (the Exchange Act)) was carried out
under the supervision of our Principal Executive Officer and Principal Financial Officer, with the
participation of our management. Based on that evaluation, the Principal Executive Officer and the
Principal Financial Officer concluded that, as of the end of such period, our disclosure controls
and procedures are effective in recording, processing, summarizing and reporting, on a timely
basis, information required to be disclosed by us in the reports that we file or submit under the
Exchange Act and are effective in ensuring that information required to be disclosed by us in the
reports that we file or submit under the Exchange Act is accumulated and communicated to our
management, including our Principal Executive Officer and Principal Financial Officer, as
appropriate to allow timely decisions regarding required disclosure.
During the fiscal quarter covered by this report, there has been no change in our internal
control over financial reporting (as defined in Rules 13a-15(f) and 15d-15(f) under the Exchange
Act) that occurred during the fiscal quarter that has materially affected, or is reasonably likely
to materially affect, our internal control over financial reporting.
- 29 -
PART II. OTHER INFORMATION
|
|
|
ITEM 1. |
|
LEGAL PROCEEDINGS |
We are not a party to any material legal proceedings.
There have been no material changes with respect to the Risk Factors disclosed in our Annual
Report on Form 10-K for the year ended December 31, 2010.
|
|
|
ITEM 2. |
|
UNREGISTERED SALES OF EQUITY SECURITIES AND USE OF PROCEEDS |
Recent Sales of Unregistered Securities
None.
Use of Proceeds
Initial Public Offering
Our initial public offering of common stock was effected through a Registration Statement on
Form S-1 (File No. 333-141700) that was declared effective by the Securities and Exchange
Commission (SEC) on May 30, 2007. We registered an aggregate of 5,750,000 shares of our common
stock. On June 5, 2007, at the closing of the offering, 5,000,000 shares of common stock were sold
on our behalf at an initial public offering price of $15.00 per share, for aggregate offering
proceeds of $75.0 million. The initial public offering was underwritten and managed by Morgan
Stanley, Merrill Lynch & Co., JPMorgan, Lazard Capital Markets and Pacific Growth Equities, LLC.
Following the sale of the 5,000,000 shares, the public offering terminated.
After deducting expenses of approximately $6.9 million, we received net offering proceeds of
approximately $68.1 million from our initial public offering. As of March 31, 2011, approximately
$18.4 million of the net proceeds from our initial public offering were maintained in money market
funds and in investment-grade, interest bearing instruments, pending their use. We have used the
remaining proceeds of approximately $49.7 million for clinical development of our projects,
research and development activities relating to additional preclinical projects and to fund working
capital and other general corporate purposes.
March 2010 Registered Direct Offering
In March 2010, we sold 4,946,524 million shares of our common stock and warrants to purchase
1,854,946 million shares of common stock in a registered direct offering to a select group of
institutional investors through a Registration Statement on Form S-3 (File No. 333-158405) that was
declared effective by the SEC on May 27, 2009. The shares of common stock and warrants were sold in
units consisting of one share of common stock and one warrant to purchase 0.375 shares of common
stock at a price of $3.74 per unit. The warrants have a term of four years and are exercisable any
time on or after the six month anniversary of the date they were issued, at an exercise price of
$4.43 per share. The aggregate offering proceeds were $18.5 million. Leerink Swann LLC served as
sole placement agent for the offering. Following the sale of the common stock and warrants, the
public offering terminated.
We paid Leerink Swann a placement agency fee equal to 5.7% of the aggregate offering proceeds,
approximately $1.05 million. The net proceeds of the offering were approximately $17.1 million
after deducting the placement agency fee and all other estimated offering expenses. No offering
expenses were paid directly or indirectly to any of our directors or officers (or their associates)
or persons owning ten percent or more of any class of our equity securities or to any other
affiliates.
- 30 -
As of March 31, 2011, we had invested the $17.1 million in net proceeds from our registered
direct offering in money market funds and in investment-grade, interest bearing instruments,
pending their use. Through March 31,
2011, we have not used the net proceeds from this offering. We intend to use the proceeds from
this offering to further advance the development of our lead product candidate, Amigal, and the
completion of certain activities required for the submission of a license application globally, as
well as for general corporate matters.
The foregoing represents our best estimate of our use of proceeds for the period indicated.
Issuer Purchases of Equity Securities
There were no purchases of our common stock for the three months ended March 31, 2011.
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|
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ITEM 3. |
|
DEFAULTS UPON SENIOR SECURITIES |
None.
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|
|
ITEM 5. |
|
OTHER INFORMATION |
None.
- 31 -
|
|
|
|
Exhibit |
|
|
Number |
|
Description |
|
|
|
|
3.1 |
(1) |
|
Restated Certificate of Incorporation |
|
|
|
|
3.2 |
(2) |
|
Amended and Restated By-laws |
|
|
|
|
31.1
|
* |
|
Certification of Principal Executive Officer pursuant to
Rules 13a-14 and 15d-14 promulgated pursuant to the
Securities Exchange Act of 1934, as amended |
|
|
|
|
31.2 |
* |
|
Certification of Principal Financial Officer pursuant to
Rules 13a-14 and 15d-14 promulgated pursuant to the
Securities Exchange Act of 1934, as amended |
|
|
|
|
32.1 |
* |
|
Certification of Principal Executive Officer and Principal
Financial Officer pursuant to Section 906 of the
Sarbanes-Oxley Act of 2002 |
|
|
|
(1) |
|
Incorporated by reference to Exhibit 3.2 to our Registration Statement on Form S-1 |
|
(2) |
|
Incorporated by reference to Exhibit 3.4 to our Registration Statement on Form S-1 |
|
* |
|
These certifications are being furnished solely to accompany this quarterly report pursuant to 18
U.S.C. Section 1350, and are not being filed for purposes of Section 18 of the Securities Exchange
Act of 1934 and are not to be incorporated by reference into any filing of Amicus Therapeutics,
Inc., whether made before or after the date hereof, regardless of any general incorporation
language in such filing. |
- 32 -
SIGNATURES
Pursuant to the requirements of the Securities and Exchange Act of 1934, the registrant has
duly caused this report to be signed on its behalf by the undersigned thereunto duly authorized.
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|
|
|
|
|
AMICUS THERAPEUTICS, INC.
|
|
Date: May 4, 2011 |
By: |
/s/ MATTHEW R. PATTERSON
|
|
|
|
Matthew R. Patterson |
|
|
|
President, and Acting Chief Executive Officer
(Principal Executive Officer) |
|
|
Date: May 4, 2011 |
By: |
/s/ DAPHNE QUIMI
|
|
|
|
Daphne Quimi |
|
|
|
Corporate Controller
(Principal Financial and Accounting Officer) |
|
- 33 -
INDEX TO EXHIBITS
|
|
|
|
Exhibit |
|
|
Number |
|
Description |
|
|
|
|
3.1 |
(1) |
|
Restated Certificate of Incorporation |
|
|
|
|
3.2 |
(2) |
|
Amended and Restated By-laws |
|
|
|
|
31.1
|
* |
|
Certification of Principal Executive Officer pursuant to
Rules 13a-14 and 15d-14 promulgated pursuant to the
Securities Exchange Act of 1934, as amended |
|
|
|
|
31.2 |
* |
|
Certification of Principal Financial Officer pursuant to
Rules 13a-14 and 15d-14 promulgated pursuant to the
Securities Exchange Act of 1934, as amended |
|
|
|
|
32.1 |
* |
|
Certification of Principal Executive Officer and Principal
Financial Officer pursuant to Section 906 of the
Sarbanes-Oxley Act of 2002 |
|
|
|
(1) |
|
Incorporated by reference to Exhibit 3.2 to our Registration Statement on Form S-1 |
|
(2) |
|
Incorporated by reference to Exhibit 3.4 to our Registration Statement on Form S-1 |
|
* |
|
These certifications are being furnished solely to accompany this quarterly
report pursuant to 18 U.S.C. Section 1350, and are not being filed for purposes
of Section 18 of the Securities Exchange Act of 1934 and are not to be
incorporated by reference into any filing of Amicus Therapeutics, Inc., whether
made before or after the date hereof, regardless of any general incorporation
language in such filing. |
- 34 -
Exhibit 31.1
Exhibit 31.1
CERTIFICATIONS PURSUANT TO SECTION 302 OF
THE SARBANES-OXLEY ACT OF 2002
CERTIFICATION BY PRINCIPAL EXECUTIVE OFFICER
I, Matthew R. Patterson, certify that:
1. I have reviewed this quarterly report on Form 10-Q of Amicus Therapeutics, Inc.;
2. Based on my knowledge, this report does not contain any untrue statement of a material fact
or omit to state a material fact necessary to make the statements made, in light of the
circumstances under which such statements were made, not misleading with respect to the period
covered by this report;
3. Based on my knowledge, the financial statements, and other financial information included
in this report, fairly present in all material respects the financial condition, results of
operations and cash flows of the registrant as of, and for, the periods presented in this report;
4. The registrants other certifying officer and I are responsible for establishing and
maintaining disclosure controls and procedures (as defined in Exchange Act Rules 13a-15(e) and
15d-15(e)) and internal control over financial reporting (as defined in Exchange Act Rules
13a-15(f) and 15d-15(f)) for the registrant and have:
(a) designed such disclosure controls and procedures, or caused such disclosure controls
and procedures to be designed under our supervision, to ensure that material information
relating to the registrant, including its consolidated subsidiaries, is made known to us by
others within those entities, particularly during the period in which this report is being
prepared;
(b) designed such internal control over financial reporting, or caused such internal
control over financial reporting to be designed under our supervision, to provide reasonable
assurance regarding the reliability of financial reporting and preparation of financial
statements for external purposes in accordance with generally accepted accounting principles;
(c) evaluated the effectiveness of the registrants disclosure controls and procedures and
presented in this report our conclusions about the effectiveness of the disclosure controls and
procedures, as of the end of the period covered by this report based on such evaluation; and
(d) disclosed in this report any change in the registrants internal control over
financial reporting that occurred during the registrants most recent fiscal quarter (the
registrants fourth fiscal quarter in the case of an annual report) that has materially
affected, or is reasonably likely to materially affect, the registrants internal control over
financial reporting; and
5. The registrants other certifying officer and I have disclosed, based on our most recent
evaluation of internal control over financial reporting, to the registrants auditors and the audit
committee of the registrants board of directors (or persons performing the equivalent functions):
(a) all significant deficiencies and material weaknesses in the design or operation of
internal control over financial reporting which are reasonably likely to adversely affect the
registrants ability to record, process, summarize and report financial information; and
(b) any fraud, whether or not material, that involves management or other employees who
have a significant role in the registrants internal control over financial reporting.
|
|
|
|
|
Date: May 4, 2011 |
/s/ Matthew R. Patterson
|
|
|
Matthew R. Patterson |
|
|
President and Acting Chief Executive Officer |
|
Exhibit 31.2
Exhibit 31.2
CERTIFICATIONS PURSUANT TO SECTION 302 OF
THE SARBANES-OXLEY ACT OF 2002
CERTIFICATION BY PRINCIPAL FINANCIAL OFFICER
I, Daphne Quimi, certify that:
1. I have reviewed this quarterly report on Form 10-Q of Amicus Therapeutics, Inc.;
2. Based on my knowledge, this report does not contain any untrue statement of a material fact
or omit to state a material fact necessary to make the statements made, in light of the
circumstances under which such statements were made, not misleading with respect to the period
covered by this report;
3. Based on my knowledge, the financial statements, and other financial information included
in this report, fairly present in all material respects the financial condition, results of
operations and cash flows of the registrant as of, and for, the periods presented in this report;
4. The registrants other certifying officer and I are responsible for establishing and
maintaining disclosure controls and procedures (as defined in Exchange Act Rules 13a-15(e) and
15d-15(e)) and internal control over financial reporting (as defined in Exchange Act Rules
13a-15(f) and 15d-15(f)) for the registrant and have:
(a) designed such disclosure controls and procedures, or caused such disclosure controls
and procedures to be designed under our supervision, to ensure that material information
relating to the registrant, including its consolidated subsidiaries, is made known to us by
others within those entities, particularly during the period in which this report is being
prepared;
(b) designed such internal control over financial reporting, or caused such internal
control over financial reporting to be designed under our supervision, to provide reasonable
assurance regarding the reliability of financial reporting and preparation of financial
statements for external purposes in accordance with generally accepted accounting principles;
(c) evaluated the effectiveness of the registrants disclosure controls and procedures and
presented in this report our conclusions about the effectiveness of the disclosure controls and
procedures, as of the end of the period covered by this report based on such evaluation; and
(d) disclosed in this report any change in the registrants internal control over
financial reporting that occurred during the registrants most recent fiscal quarter (the
registrants fourth fiscal quarter in the case of an annual report) that has materially
affected, or is reasonably likely to materially affect, the registrants internal control over
financial reporting; and
5. The registrants other certifying officer and I have disclosed, based on our most recent
evaluation of internal control over financial reporting, to the registrants auditors and the audit
committee of the registrants board of directors (or persons performing the equivalent functions):
(a) all significant deficiencies and material weaknesses in the design or operation of
internal control over financial reporting which are reasonably likely to adversely affect the
registrants ability to record, process, summarize and report financial information; and
(b) any fraud, whether or not material, that involves management or other employees who have a
significant role in the registrants internal control over financial reporting.
|
|
|
|
|
Date: May 4, 2011 |
/s/ Daphne Quimi
|
|
|
Daphne Quimi |
|
|
Corporate Controller |
|
Exhibit 32.1
Exhibit 32.1
CERTIFICATION OF PRINCIPAL EXECUTIVE OFFICER AND
PRINCIPAL FINANCIAL OFFICER PURSUANT TO
18 U.S.C. SECTION 1350, AS ADOPTED PURSUANT TO
SECTION 906 OF THE SARBANES-OXLEY ACT OF 2002
Each of the undersigned hereby certifies, pursuant to 18 U.S.C. Section 1350, as adopted
pursuant to Section 906 of the Sarbanes-Oxley Act of 2002, in his capacity as an officer of Amicus
Therapeutics, Inc. (the Company), that, to his knowledge, the Quarterly Report of the Company on
Form 10-Q for the period ended March 31, 2011, fully complies with the requirements of Section
13(a) or 15(d) of the Securities Exchange Act of 1934 (15 U.S.C. 78m or 78o(d)) and that the
information contained in such report fairly presents, in all material respects, the financial
condition and results of operations of the Company. This written statement is being furnished to
the Securities and Exchange Commission as an exhibit to such Form 10-Q. A signed original of this
statement has been provided to the Company and will be retained by the Company and furnished to the
Securities and Exchange Commission or its staff upon request.
|
|
|
|
|
Date: May 4, 2011 |
By: |
/s/ Matthew R. Patterson
|
|
|
|
Matthew R. Patterson |
|
|
|
President and Acting Chief Executive Officer |
|
|
|
|
Date: May 4, 2011 |
By: |
/s/ Daphne Quimi
|
|
|
|
Daphne Quimi |
|
|
|
Corporate Controller |
|
|