UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
SECURITIES AND EXCHANGE COMMISSION
Washington, D.C. 20549
FORM 8-K
CURRENT REPORT
Pursuant to Section 13 OR 15(d) of The Securities Exchange Act of 1934
Pursuant to Section 13 OR 15(d) of The Securities Exchange Act of 1934
Date of Report (Date of earliest event reported): November 1, 2011
AMICUS THERAPEUTICS, INC.
(Exact name of registrant as specified in its charter)
| Delaware | 001-33497 | 71-0869350 | ||
| (State or other jurisdiction of incorporation) |
(Commission File Number) | (IRS Employer Identification No.) |
| 6 Cedar Brook Drive, Cranbury, NJ |
08512 |
|
| (Address of principal executive offices) | (Zip Code) |
Registrant’s telephone number, including area code: (609) 662-2000
(Former name or former address, if changed since last report.)
Check the appropriate box below if the Form 8-K filing is intended to simultaneously satisfy the filing obligation of the registrant under any of the following provisions:
| o | Written communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425) | |
| o | Soliciting material pursuant to Rule 14a-12 under the Exchange Act (17 CFR 240.14a-12) | |
| o | Pre-commencement communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR 240.14d-2(b)) | |
| o | Pre-commencement communications pursuant to Rule 13e-4(c) under the Exchange Act (17 CFR 240.13e-4(c)) |
Item 2.02. Results of Operations and Financial Condition.
On November 1, 2011, Amicus Therapeutics, Inc. issued a press release announcing its financial
results for the quarter ended September 30, 2011. A copy of this press release is attached hereto
as Exhibit 99.1.
In accordance with General Instruction B.2. of Form 8-K, the information in this Current
Report on Form 8-K, including Exhibit 99.1, shall not be deemed “filed” for purposes of Section 18
of the Securities Exchange Act of 1934, as amended (the “Exchange Act”), or otherwise subject to
the liability of that section, nor shall it be deemed incorporated by reference in any filing under
the Securities Act of 1933, as amended, or the Exchange Act, except as expressly set forth by
specific reference in such a filing.
Item 9.01. Financial Statements and Exhibits.
(d) Exhibits: The Exhibit Index annexed hereto is incorporated herein by reference.
SIGNATURES
Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly
caused this report to be signed on its behalf by the undersigned hereunto duly authorized.
| Amicus Therapeutics, Inc. |
||||
| Date: November 1, 2011 | By: | /s/ Geoffrey P. Gilmore | ||
| Geoffrey P. Gilmore | ||||
| Senior Vice President and General Counsel | ||||
EXHIBIT INDEX
| Exhibit No. | Description | |||
| 99.1 | Press Release dated November 1, 2011 |
|||
Exhibit 99.1
Amicus Therapeutics Announces
Third Quarter 2011 Financial Results
Third Quarter 2011 Financial Results
Final Enrollment in Amigal Phase 3 Study on Track for 4Q11
CRANBURY, N.J., November 1, 2011 — Amicus Therapeutics (Nasdaq: FOLD), a biopharmaceutical
company at the forefront of developing therapies for rare diseases, today announced financial
results for the third quarter ended September 30, 2011. The Company also highlighted recent and
upcoming milestones surrounding Phase 3 global registration studies of Amigal™ (migalastat HCl) for
Fabry disease, and ongoing Phase 2 studies to evaluate the co-administration of pharmacological
chaperones with enzyme replacement therapy (PC-ERT) for Fabry and Pompe diseases.
Development Pipeline Progress and Upcoming Milestones
| • | Patient recruitment closed in first Phase 3 study of migalastat HCl (Study 011) — final
enrollment on track for 4Q11 |
| • | First patient dosed in second Phase 3 study of migalastat HCl (Study 012) |
| • | Seventeen subjects treated with migalastat HCl for up to five years in Phase 2 and Phase 2
extension studies |
| • | Preliminary Phase 2 migalastat HCl-ERT co-administration study (Study 013) results
anticipated in 4Q11 |
| • | Patient recruitment underway for Phase 2 study (Study 010) of AT2220 (duvoglustat HCI)
co-administered with ERT for Pompe disease — dosing of first patient anticipated in 4Q11 |
| • | Results from preclinical proof-of-concept study of AT3375 for Parkinson’s disease expected
in 4Q11 |
John F. Crowley, Chairman and Chief Executive Officer of Amicus Therapeutics said, “With the close
of recruitment in Study 011 and patient dosing underway in Study 012, we continue toward the
objective of global registration of migalastat HCl for Fabry disease. These achievements reflect
our dedication to the Fabry program and the strength of our collaboration with GSK. Additionally,
the co-administration of chaperones with ERT has the potential to be an important expansion of our
technology that may provide additional treatment options for lysosomal storage disorders. Based on
our current cash position and migalastat HCl cost-sharing under the GSK agreement, we are
well-capitalized to continue advancing our pipeline as we head toward major catalysts this quarter
and into next year.”
Third Quarter 2011 Financial Summary
As of September 30, 2011, cash, cash equivalents, and marketable securities totaled $69.5 million,
compared to $83.0 million at June 30, 2011.
Total revenue was $5.8 million for the third quarter 2011, compared to no revenue in the prior year
period. Total revenue consists of collaboration and research revenues recognized under the
Company’s collaboration with Glaxo Group Limited (GSK). In October 2010, Amicus and GSK entered
into an agreement pursuant to which GSK received an exclusive worldwide license to develop,
manufacture and commercialize migalastat HCl for the treatment of Fabry disease.
Research revenue for the third quarter 2011 was $4.1 million, and reflects payments received from
GSK for shared development costs. Amicus and GSK are equally sharing development costs for
migalastat HCl in 2011, and GSK will be responsible for 75% of these costs in 2012 and beyond.
Collaboration revenue for the third quarter 2011 was $1.66 million, and reflects the recognized
portion of the $33.2 million upfront payment received from GSK upon signing the agreement.
Total operating expenses in the third quarter 2011 were $18.9 million, compared to $13.3 million in
the prior year period. The increase was primarily attributed to higher expenses for research and
development, one-time stock-based compensation and severance.
Net loss attributable to common stockholders for the three months ended September 30, 2011 was $9.8
million, or $0.28 per share, compared to a net loss of $15.4 million, or $0.56 per share, for the
same period in 2010. Weighted-average common shares outstanding were 35.0 million and 27.6 million
for the three months ended September 30, 2011 and September 30, 2010, respectively.
2011 Financial Guidance
Amicus continues to expect to spend a total of $50 million to $55 million on 2011 operating
expenses, net of cost sharing and milestones related to the GSK collaboration.
The Company continues to expect that the current cash position, including anticipated collaboration
payments from GSK, will be sufficient to fund the Company’s operations and capital expenditure
requirements through at least the end of 2012.
Pipeline Highlights
Migalastat HCl for Fabry Disease: Phase 3 Global Registration Program
Amicus
and its collaborator GSK are conducting two Phase 3 registration
studies (Study 011 and
Study 012) to support the global approval of migalastat HCl for the treatment of Fabry disease.
Both studies are evaluating Fabry patients with genetic mutations that may be amenable to
migalastat HCl monotherapy.
Study 011 is a six-month, randomized, double-blind, placebo-controlled study to support marketing
applications to the U.S. Food and Drug Administration (FDA) and other regulatory agencies. Patient
recruitment has closed at 37 centers worldwide and final enrollment is expected by year-end.
“Based on the number of patients currently in screening and randomized in Study 011, we are on
track to exceed our target enrollment of 60 patients,” added Mr. Crowley. “There has been positive
momentum in this study, particularly over the past few months, and we believe that each and every
patient plays a very important role in building our data set for migalastat HCl.”
During the third quarter 2011, the first patient was dosed in Study 012, a randomized, open-label,
18-month Phase 3 study to compare the safety and efficacy of migalastat HCl and ERT in
approximately 50 male or female patients (30 to switch from ERT to migalastat HCl and 20 to remain
on ERT).
Pharmacological Chaperone-ERT (PC-ERT) Co-Administration: Phase 2 Studies in Fabry and Pompe
Two open-label Phase 2 studies are underway in Fabry and Pompe patients to evaluate safety,
pharmacokinetic (PK) and pharmacodynamic (PD) parameters for PC-ERT co-administration versus ERT
alone. Patients in each study will receive a regular ERT infusion and on a subsequent occasion will
receive an orally administered pharmacological chaperone just prior to the next scheduled ERT
infusion.
Amicus and GSK are investigating migalastat HCl co-administered with ERT as part of the global
collaboration for Fabry disease. An ongoing Phase 2 study (Study
013) will evaluate two different
doses of migalastat HCl (150 mg and 450 mg) co-administered with ERT in males with Fabry disease.
Study 013 will measure ERT plasma PK and enzyme activity in skin biopsies, with and without an oral
dose of migalastat HCl. Preliminary results from this study are anticipated in the fourth quarter
2011.
Patient recruitment is also underway for a Phase 2 study
(Study 010) to evaluate four different
doses of a second pharmacological chaperone owned exclusively by Amicus, AT2220, co-administered
with ERT in
approximately 16 Pompe patients. The study will measure ERT plasma PK and enzyme activity in muscle
biopsies, with and without an oral dose of AT2220. Dosing of the first patient in Study 010 is
anticipated in the fourth quarter of 2011.
Preclinical Pharmacological Chaperones
Amicus is conducting preclinical studies of AT3375 for the treatment of Parkinson’s disease. AT3375
is a pharmacological chaperone targeted at glucocerobrosidase (GCase), the enzyme deficient in
Gaucher disease. Mutations in the GBA1 gene that encodes for the GCase enzyme are the most common
genetic risk factor known for Parkinson’s disease.
Posters at Human Genetic Meeting (ICHG/ASHG 2011)
Migalastat HCl for Fabry disease and AT2220 for Pompe disease were featured in posters at the 12th
International Congress of Human Genetics (ICHG) and the 61st
ASHG Annual Meeting (ICHG/ASHG 2011),
held October 11-15, 2011 in Montreal, Canada.
Phase 2 Study in Female Fabry Patients
Oral migalastat HCL (AT1001/GR181314A) as an investigational therapy evaluated in females with
Fabry disease. P. Fernhoff, R. Giugliani, K. Nicholls, et al.
One of
four open-label Phase 2 studies (Study 204) evaluated the safety and tolerability of oral
migalastat HCl (50, 150, or 250 mg once every other day, or QOD) in a total of nine symptomatic
female Fabry patients. All nine females completed the initial 12-week treatment and a 36-week
treatment extension period with migalastat HCl. No treatment-related serious adverse events (SAEs)
and no treatment-limiting toxicities were identified. No patients interrupted, reduced, or
discontinued study drug dosing due to an AE. All treatment-related AEs were mild or moderate in
severity, and only two were reported as likely or related to study drug (atrioventricular block,
resolved without any intervention while patient continued migalastat HCl treatment, and abdominal
discomfort).
After 48 weeks of treatment, females treated with 150 mg or 250 mg migalastat HCl, who were
retrospectively identified as having amenable GLA mutations, showed the earliest and most
significant declines in urine globotriaosylceramide (GL-3), and also demonstrated decreases in GL-3
inclusions in interstitial capillary cells. In contrast, none of the patients with non-amenable GLA
mutations showed a consistent reduction in urine GL-3 at any dose in this study. Kidney and urine
GL-3 are biomarkers of Fabry disease.
“In addition to supporting our Phase 3 Fabry registration program, Study 204 demonstrates our
ongoing commitment to gathering more data in female Fabry patients, who constitute a significant
fraction of the overall Fabry population,” said Pol F. Boudes, M.D., Chief Medical Officer of
Amicus.
Different doses and regimens of migalastat HCl were previously evaluated in four Phase 2 studies
that support the design of ongoing Phase 3 registration studies of migalastat HCl in male and
female Fabry patients that have been enrolled on the basis of specific genetic mutation types.
Preclinical PC-ERT Co-administration Results in Fabry and Pompe
Two studies investigated the effects of PC-ERT co-administration in vitro and in vivo in models of
Fabry and Pompe. Results from both studies indicate that PC-ERT co-administration increases ERT
stability, enzyme activity, and substrate reduction in disease-relevant tissues compared to ERT
alone.
The pharmacological chaperone AT1001 increases the tissue uptake of agalsidase alfa resulting in
greater substrate reduction in a mouse model of Fabry disease. L. Pellegrino, J. Feng, M.
Frascella, et al.
The pharmacological chaperone AT2220 increases the muscle uptake of recombinant human acid
a-glucosidase resulting in greater glycogen reduction in a mouse model of Pompe disease. J. Feng,
R. Soska, L. Pellegrino, et al.
“Our preclinical PC-ERT co-administration work formed the basis for our ongoing Phase 2 studies in
Fabry and Pompe diseases, which we hope will provide initial proof-of-concept for this approach in
humans and guide us in the design of later-stage studies,” stated David J. Lockhart, Ph.D., Chief
Scientific Officer of Amicus.
Conference Call and Webcast
Amicus Therapeutics will host a conference call and webcast today, November 1, 2011, at 5:00 P.M.
ET to review financial results and provide a corporate update. Interested participants and
investors may access the conference call at 5 p.m. ET by dialing 877-303-5859 (U.S./Canada) or
678-224-7784 (international).
An audio webcast can also be accessed via the Investors section of the Amicus Therapeutics
corporate web site at http://www.amicustherapeutics.com, and will be archived for 30 days. Web
participants are encouraged to go to the Web site 15 minutes prior to the start of the call to
register, download and install any necessary software.
A telephonic replay of the call will be available for seven days beginning at 8 p.m. ET today.
Access numbers for this replay are 855-859-2056 (U.S./Canada) and 404-537-3406
(international); participant code 22660442.
About Amicus Therapeutics
Amicus Therapeutics (Nasdaq: FOLD) is a biopharmaceutical company at the forefront of developing
therapies for rare diseases. The Company is developing orally-administered, small molecule drugs
called pharmacological chaperones, a novel, first-in-class approach to treating a broad range of
diseases including lysosomal storage disorders and diseases of neurodegeneration. Amicus’ lead
program Amigal™ (migalastat HCl) is in Phase 3 for the treatment of Fabry disease.
About Migalastat HCl
Migalastat HCI is an investigational, orally-administered pharmacological chaperone in Phase 3
development for the treatment of Fabry disease being developed in collaboration with
GlaxoSmithKline (GSK). Under the terms of the collaboration, GSK has an exclusive worldwide license
to develop, manufacture and commercialize migalastat HCl. Amicus and GSK are conducting two Phase 3
global registration studies (Study 011 and Study 012) of migalastat HCl monotherapy, along with a
Phase 2 study (Study 013) evaluating migalastat HCl-ERT co-administration for the treatment of
Fabry disease.
About Pharmacological Chaperone-ERT (PC-ERT) Co-Administration
The broader use of pharmacological chaperones co-administered with ERT may represent an important
extension of the Company’s chaperone technology platform. Phase 2 co-administration studies of
migalastat HCl-ERT for Fabry disease and AT2220 (duvoglostat HCl)-ERT for Pompe disease are
currently underway.
About Fabry Disease
Fabry disease is an inherited lysosomal storage disorder that is currently estimated to affect
approximately 5,000 to 10,000 people worldwide. Fabry Disease is caused by deficiency of an enzyme
called alpha-galactosidase A (alpha-Gal A). The role of alpha-Gal A within the body is to break
down a complex lipid called globotriaosylceramide (GL-3). Reduced or absent levels of alpha-Gal A
activity leads to the accumulation of GL-3 in the affected tissues, including the central nervous
system, heart, kidneys, and skin. This accumulation of GL-3 is believed to cause the various
symptoms of Fabry disease, including pain, kidney failure, and increased risk of heart attack and
stroke.
About Pompe Disease
Pompe disease is an inherited lysosomal storage disorder that affects an estimated 5,000 to 10,000
individuals worldwide and is caused by deficiency in an enzyme called alpha-glucosidase (GAA).
Pompe disease is clinically heterogeneous in the age of onset, extent of organ involvement, and
rate of progression. The early onset form is most severe, progresses most rapidly, and is
characterized by musculoskeletal, pulmonary, gastrointestinal, and cardiac symptoms that usually
lead to death between one and two years of age. A high majority of patients develop the late onset
form of Pompe disease between childhood and adulthood, which has a slower rate of progression and
usually leads to progressive muscle weakness and respiratory insufficiency.
Forward-Looking Statements
This press release contains, and the accompanying conference call will contain, “forward-looking
statements” within the meaning of the Private Securities Litigation Reform Act of 1995 relating to
preclinical and clinical development of Amicus’ candidate drug products, the timing and reporting
of results from preclinical studies and clinical trials evaluating Amicus’ candidate drug products,
and the projected cash position for the Company, including achievement of development and
commercialization milestone payments and sales royalties under our collaboration with
GlaxoSmithKline. Words such as, but not limited to, “look forward to,” “believe,” “expect,”
“anticipate,” “estimate,” “intend,” “plan,” “targets,” “likely,” “will,” “would,” “should” and
“could,” and similar expressions or words identify forward-looking statements. Such forward-looking
statements are based upon current expectations that involve risks, changes in circumstances,
assumptions and uncertainties. The inclusion of forward-looking statements should not be regarded
as a representation by Amicus that any of its plans will be achieved. Any or all of the
forward-looking statements in this press release may turn out to be wrong. They can be affected by
inaccurate assumptions Amicus might make or by known or unknown risks and uncertainties. For
example, with respect to statements regarding the goals, progress, timing and outcomes of
discussions with regulatory authorities and the potential goals, progress, timing and results of
preclinical studies and clinical trials, actual results may differ materially from those set forth
in this release due to the risks and uncertainties inherent in the business of Amicus, including,
without limitation: the potential that results of clinical or pre-clinical studies indicate that
the product candidates are unsafe or ineffective; the potential that it may be difficult to enroll
patients in our clinical trials; the potential that regulatory authorities may not grant or may
delay approval for our product candidates; the potential that preclinical and clinical studies
could be delayed because we identify serious side effects or other safety issues; the potential
that we will need additional funding to complete all of our studies and, our dependence on third
parties in the conduct of our clinical studies. Further, the results of earlier preclinical studies
and/or clinical trials may not be predictive of future results. With respect to statements
regarding projections of the Company’s cash position, actual results may differ based on market
factors and the Company’s ability to execute its operational and budget plans, including
achievement of development and commercialization milestone payments and sales royalties under our
collaboration with GlaxoSmithKline. In addition, all forward looking statements are subject to
other risks detailed in our Annual Report on Form 10-K for the year ended December 31, 2010. You
are cautioned not to place undue reliance on these forward-looking statements, which speak only as
of the date hereof. All forward-looking statements are qualified in their entirety by this
cautionary statement, and Amicus undertakes no obligation to revise or update this news release to
reflect events or circumstances after the date hereof. This caution is made under the safe harbor
provisions of Section 21E of the Private Securities Litigation Reform Act of 1995.
CONTACTS:
Investors/Media:
Sara Pellegrino
spellegrino@amicustherapeutics.com
(609) 662-5044
Sara Pellegrino
spellegrino@amicustherapeutics.com
(609) 662-5044
FOLD—G
Table 1
Amicus Therapeutics, Inc.
(a development stage company)
Consolidated Statements of Operations
(Unaudited)
(In thousands, except share and per share amounts)
(a development stage company)
Consolidated Statements of Operations
(Unaudited)
(In thousands, except share and per share amounts)
| Period from | ||||||||||||||||||||
| February 4, | ||||||||||||||||||||
| 2002 | ||||||||||||||||||||
| (inception) | ||||||||||||||||||||
| Three Months | Nine Months | to | ||||||||||||||||||
| Ended Sept. 30, | Ended Sept. 30, | Sept. 30, | ||||||||||||||||||
| 2010 | 2011 | 2010 | 2011 | 2011 | ||||||||||||||||
Revenue: |
||||||||||||||||||||
Research revenue |
$ | — | $ | 4,138 | $ | — | $ | 10,824 | $ | 41,932 | ||||||||||
Collaboration revenue |
— | 1,660 | — | 4,980 | 55,902 | |||||||||||||||
Total revenue |
— | 5,798 | — | 15,804 | 97,834 | |||||||||||||||
Operating Expenses: |
||||||||||||||||||||
Research and development |
8,862 | 13,711 | 25,888 | 36,455 | 251,219 | |||||||||||||||
General and administrative |
3,892 | 4,841 | 11,837 | 15,963 | 109,332 | |||||||||||||||
Restructuring charges |
— | — | — | — | 1,522 | |||||||||||||||
Impairment of leasehold
improvements |
— | — | — | — | 1,030 | |||||||||||||||
Depreciation and amortization |
511 | 380 | 1,577 | 1,243 | 9,721 | |||||||||||||||
In-process research and
development |
— | — | — | — | 418 | |||||||||||||||
Total operating expenses |
13,265 | 18,932 | 39,302 | 53,661 | 373,242 | |||||||||||||||
Loss from operations |
(13,265 | ) | (13,134 | ) | (39,302 | ) | (37,857 | ) | (275,408 | ) | ||||||||||
Other income (expenses): |
||||||||||||||||||||
Interest income |
33 | 31 | 121 | 136 | 14,049 | |||||||||||||||
Interest expense |
(66 | ) | (32 | ) | (203 | ) | (121 | ) | (2,306 | ) | ||||||||||
Change in fair value of
warrant liability |
(2,059 | ) | 3,376 | (464 | ) | 2,022 | 158 | |||||||||||||
Other income |
— | — | — | 70 | 231 | |||||||||||||||
Loss before tax benefit |
(15,357 | ) | (9,759 | ) | (39,848 | ) | (35,750 | ) | (263,276 | ) | ||||||||||
Benefit from income taxes |
— | — | — | — | 1,834 | |||||||||||||||
Net Loss |
(15,357 | ) | (9,759 | ) | (39,848 | ) | (35,750 | ) | (261,442 | ) | ||||||||||
Deemed dividend |
— | — | — | — | (19,424 | ) | ||||||||||||||
Preferred stock accretion |
— | — | — | — | (802 | ) | ||||||||||||||
Net Loss attributable to common
stockholders |
$ | (15,357 | ) | $ | (9,759 | ) | $ | (39,848 | ) | $ | (35,750 | ) | $ | (281,668 | ) | |||||
Net Loss attributable to common
stockholders per
common share — basic and
diluted |
$ | (0.56 | ) | $ | (0.28 | ) | $ | (1.50 | ) | $ | (1.03 | ) | ||||||||
Weighted-average common shares
outstanding — basic and diluted |
27,625,137 | 34,979,702 | 26,516,688 | 34,544,768 | ||||||||||||||||
Source: FOLD -G