Continues to Advance Programs and Technology for Chaperone-ERT Combination Products
Migalastat HCl Monotherapy Phase 3 Results for Fabry Disease on Track for 3Q12
Corporate Highlights and Upcoming Milestones
First Quarter 2012 Financial Highlights
Cash, cash equivalents, and marketable securities totaled
Total revenue for the three months ended
Amicus continues to expect full-year 2012 operating expenses will total between
Fabry Disease Program
Phase 3 Global Registration Studies (Study 011 and Study 012) — Migalastat HCl Monotherapy
Amicus and GSK are conducting two Phase 3 global registration studies (Study 011 and Study 012) of migalastat HCl monotherapy. Study 011 and Study 012 are investigating migalastat HCl at an oral dose of 150 mg, administered every-other-day (QOD) to Fabry patients identified as having alpha-Gal A mutations amenable to migalastat HCl as a monotherapy.
Study 011 is a six-month, placebo-controlled Phase 3 study of migalastat HCl that completed enrollment of 67 patients with Fabry disease. Results from this study are anticipated in the third quarter of 2012 to support subsequent marketing applications for the
Study 012 is a randomized, open-label, 18-month Phase 3 study investigating the safety and efficacy of migalastat HCI compared to current standard-of-care ERTs Fabrazyme (agalsidase beta) or Replagal (agalsidase alfa) for Fabry disease. A majority of patients have been enrolled in this study, which is targeting approximately 50 total patients (30 to switch to migalastat HCl and 20 to remain on ERT). Study 012 is currently underway at 25 clinical sites worldwide, including U.S. sites that are now able to enroll patients who have resumed full-dose Fabrazyme. Strong global interest in this study has resulted in the initiation of several new sites and robust patient screening and enrollment since the beginning of this year. Amicus and GSK continue to anticipate that enrollment in this study will be completed by year-end 2012.
Phase 2 and Phase 3 extension studies continue to evaluate long-term safety with migalastat HCl monotherapy in Fabry patients. As of
Phase 2 Study 013 — Drug-Drug Interaction Study of Migalastat HCl and ERT
Study 013 is an ongoing open-label Phase 2 study to investigate a single oral dose of migalastat HCl (150 mg or 450 mg) co-administered two hours prior to ERT (Fabrazyme or Replagal) in males diagnosed with Fabry disease. When co-administered with ERT, migalastat HCl is designed to bind to and stabilize the enzyme in the circulation, independent of alpha-Gal A mutation type.
Positive preliminary results from Study 013 were announced in the first quarter 2012 in patients who received migalastat HCl 150 mg co-administered with Fabrazyme (0.5 mg/kg or 1.0 mg/kg). The Company expects to provide an update from this study in the third quarter 2012 and to present final results at a Fall 2012 scientific conference. Both Amicus and GSK are committed to working together to advance this Fabry co-administration program, which has been recognized as having significant medical interest and importance. A repeat-dose global study of migalastat HCl co-administered with ERT is currently being designed as the next step in U.S. and global development. Following the withdrawal of the U.S. marketing application for Replagal, Fabrazyme remains the only ERT with conditional approval in the U.S.
Outside the Fabry collaboration with GSK, Amicus owns exclusive rights to the rest of its pipeline and applications of its platform technology. In addition to the ongoing work in Fabry, Amicus is investigating chaperone-ERT combinations as potential next-generation treatments for Pompe, Gaucher, and other undisclosed lysosomal storage diseases where the Company believes there are significant opportunities to improve treatment outcomes.
Preclinical chaperone-ERT co-administration studies in animal models of Fabry, Pompe and Gaucher have shown that a pharmacological chaperone can selectively bind to and stabilize the enzyme, prevent deactivation in the circulation, and increase uptake of active enzyme into key tissues of disease. In Fabry and Pompe animal models, chaperone-ERT co-administration has also been shown to lead to greater substrate reduction compared to ERT alone.
Chaperone-ERT Combination for Pompe Disease
Amicus is investigating four ascending doses of AT2220 co-administered with the ERT alglucosidase alfa (Myozyme or Lumizyme) in a Phase 2 open-label study (Study 010) for Pompe disease. Approximately 22 patients will receive one infusion of ERT alone, and a single oral dose of AT2220 prior to the next ERT infusion. In addition to safety and pharmacokinetic effects, Study 010 will measure uptake of active enzyme in muscle tissue with and without the chaperone, three or seven days following each infusion. Enrollment has been completed for the first two dose groups, and preliminary results from Study 010 continue to be anticipated in the second quarter of 2012.
In parallel with Study 010, Amicus is evaluating ERT-related immunogenicity in Pompe disease. Immune responses occur in a majority of Pompe patients receiving alglucosidase alfa infusions1 which have the potential to limit treatment outcomes with ERT. Preclinical results to date suggest that AT2220 when co-administered with Myozyme may mitigate immunogenicity induced by this ERT by stabilizing the enzyme in its properly folded and active form.
As part of a grant from the
Chaperone-ERT Combinations for Gaucher Disease
Amicus is also investigating chaperone-ERT combinations as potential next-generation treatments for Gaucher and other undisclosed lysosomal storage diseases where there are significant opportunities to improve treatment outcomes. In Gaucher disease, Amicus is continuing preclinical studies to evaluate two pharmacological chaperones, AT2101 (afegostat tartrate) and AT3375, in combination with ERT (beta-glucosidase). Both of these chaperones target the enzyme deficient in Gaucher disease.
Chaperone for Parkinson's Disease in Gaucher Carriers
Gaucher disease is caused by inherited genetic mutations in the GBA gene, and mutations in this gene that encodes for the GCase enzyme are the most common genetic risk factor for Parkinson's. By targeting GCase in the brain, AT3375 could potentially treat Gaucher, Parkinson's disease in Gaucher carriers, and possibly the general Parkinson's population. By year-end 2012, Amicus expects to complete preclinical and IND-enabling studies of AT3375, which are supported in part by a grant from the
Conference Call and Webcast
An audio webcast can also be accessed via the Investors section of the
A telephonic replay of the call will be available for seven days beginning at
About Fabry Disease
Fabry disease is an inherited lysosomal storage disease 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 disorders and stroke.
About Pompe Disease
Pompe disease is a lysosomal storage disease characterized by progressive skeletal muscle weakness and respiratory insufficiency. It is caused by a deficiency in lysosomal alpha-glucosidase (GAA) activity, which leads to accumulation of glycogen in tissues affected by the disease (primarily muscle). Pompe disease affects an estimated 5,000 to 10,000 individuals worldwide and is clinically heterogeneous in the age of onset, the extent of organ involvement, and the rate of progression.
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, 2011. 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.
|(a development stage company)|
|Consolidated Statements of Operations|
|(In thousands, except share and per share amounts)|
|Research and development||11,125||14,004||279,624|
|General and administrative||4,402||4,095||117,344|
|Impairment of leasehold improvements||--||--||1,030|
|Depreciation and amortization||438||420||10,483|
|In-process research and development||--||--||418|
|Total operating expenses||15,965||18,519||410,421|
|Loss from operations||(9,999)||(10,745)||(299,183)|
|Other income (expenses):|
|Change in fair value of warrant liability||(3,432)||(2,376)||(1,476)|
|Loss before tax benefit||(13,350)||(13,137)||(288,704)|
|Benefit from income taxes||--||--||5,463|
|Preferred stock accretion||--||--||(802)|
|Net loss attributable to common stockholders||$ (13,350)||$ (13,137)||$ (303,467)|
|Net loss attributable to common stockholders per common share — basic and diluted||$ (0.39)||$ (0.35)|
|Weighted-average common shares outstanding — basic and diluted||34,498,926||37,887,520|
Sara Pellegrinospellegrino@amicustherapeutics.com (609) 662-5044
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