Amicus Therapeutics Initiates Phase 2 Clinical Trial Evaluating Amigal™ Co-administered with Enzyme Replacement Therapy

CRANBURY, N.J., Feb. 9, 2011 /PRNewswire/ -- Amicus Therapeutics (Nasdaq: FOLD) today announced the dosing of the first subject in a Phase 2 clinical trial designed to evaluate the co-administration of its investigational drug Amigal™ (migalastat hydrochloride) with enzyme replacement therapy (ERT) for Fabry disease.  This open-label Phase 2 study is investigating drug-drug interactions between Amigal and the ERTs Fabrazyme® and Replagal®.  Preclinical studies have demonstrated that coadministration of Amigal results in a prolonged circulating half-life of ERT, increased enzyme activity in cells, and greater substrate reduction in target tissues compared to that seen with ERT alone.  Amigal is being developed in collaboration with GlaxoSmithKline (GSK), as part of an alliance between GSK Rare Diseases and Amicus.

John F. Crowley, Chairman and CEO of Amicus, stated "The commencement of this clinical study is an important milestone for Amicus and for pharmacological chaperone technology.  Based on encouraging preclinical results we believe the co-administration of Amigal with ERT has the potential to provide significant benefit for people with Fabry disease.  The advancement of this approach is a part of our vision to provide two new treatment options for Fabry patients in the future—Amigal as a monotherapy or Amigal co-administered with ERT.  Finally, we also believe this study will provide us with valuable proof of concept for applying this approach to other lysosomal storage diseases and more broadly to other indications treated with therapeutic proteins."  

Phase 2 Study Design

The Phase 2 trial will evaluate the safety and pharmacokinetics of Amigal and ERT when co-administered in 18 male patients with Fabry disease, ages 18-65, who have been receiving ERT for at least one month before study entry.  Patients enrolled in this study need not have a genetic mutation responsive to Amigal as a monotherapy.  Each patient will receive ERT alone and then ERT after administration of a single oral dose of Amigal.  There will be two cohorts of nine patients treated with one of two Amigal dose levels.

The primary outcome measure will be a comparison of the ERT activity in plasma and safety and tolerance with and without coadministration of Amigal.  The effect of co-administration of ERT on the pharmacokinetics and safety of Amigal will also be evaluated.  A secondary outcome measure includes distribution of the ERT to skin.  Other outcome measures include evaluating the effect of co-administration of Amigal and ERT on GL-3 levels in skin and GL-3 excretion in urine.  Amicus expects results from this study to be available in the second half of 2011.

Approximately five clinical sites will participate in this trial in the United States and in Europe.

About Amigal

On October 29, 2010, Amicus announced a definitive agreement with GSK to develop and commercialize Amigal (migalastat HCl), currently in Phase 3, for the treatment of Fabry disease as a monotherapy. Under the terms of the agreement, GSK received an exclusive worldwide license to develop, manufacture and commercialize migalastat HCl. As part of the agreement, GSK and Amicus also intend to investigate Amigal as a potential treatment for Fabry disease when co-administered with ERT.  

The Phase 3 study (Study 011) of migalastat HCl is ongoing and patients are being enrolled at 36 investigational sites worldwide.  A majority of the planned 60 patients have been enrolled in the study. The Company expects to complete enrollment in the first half of 2011 and to report top line results from this study in the second half of 2011.

Amicus and GSK intend to commence an additional Phase 3 study (Study 012) in the first quarter of 2011. Study 012 will be an 18-month, randomized, open-label study comparing migalastat HCl to enzyme replacement therapy (ERT) in approximately 60 subjects. The primary outcome of efficacy will be renal function as measured by glomerular filtration rate (GFR).

About Fabry Disease

Fabry disease is an inherited lysosomal storage disorder 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.

It is currently estimated that Fabry disease affects approximately 5,000 to 10,000 people worldwide.

About Amicus Therapeutics

Amicus Therapeutics is a biopharmaceutical company focused on developing treatments 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 is in Phase 3 for the treatment of Fabry disease. 

Forward-Looking Statements

This press release contains "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 and the timing and reporting of results from preclinical studies and clinical trials evaluating Amicus' candidate drug products. 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. 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, 2009. 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.

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SOURCE Amicus Therapeutics