CRANBURY, N.J., Oct. 19, 2011 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq:FOLD), a biopharmaceutical company at the forefront of developing therapies for rare diseases, today announced that patient recruitment has closed in the first Phase 3 global registration study (Study 011) of Amigal™ (migalastat HCl) for Fabry disease. Participating sites have stopped screening new patients, and Amicus and its collaborator GlaxoSmithKline (GSK) continue to anticipate full enrollment this quarter upon initial dosing of the last patient.
John F. Crowley, Chairman and Chief Executive Officer of Amicus Therapeutics stated, "I am proud to announce that we, along with our collaborator GSK, have closed recruitment for the first phase 3 study for Amigal. This is a significant accomplishment given the strict entry criteria for Study 011. I am confident in the strength of our Fabry program and impressed with the coordinated effort at Amicus and GSK as we continue to progress."
Study Design
Study 011 is a six-month, placebo-controlled Phase 3 study of migalastat HCl for Fabry disease at 37 centers worldwide to support marketing applications for the U.S. Food and Drug Administration (FDA) and other regulatory agencies. The study is expected to randomize approximately 60 male and female Fabry patients, with an equal number to receive migalastat HCl 150 mg or placebo on an every-other-day (QOD) oral dosing schedule for a six-month treatment period.
Patients enrolled in the study must meet the following entry criteria: naïve to enzyme replacement therapy (ERT) or have not received ERT for at least six months, a genetic mutation that may be addressable with migalastat HCl, and elevated urine globotriaosylceramide (GL-3) levels at least four times the upper limit of normal.
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 HCl is an investigational, oral pharmacological chaperone in Phase 3 development for the treatment of Fabry disease. On October 29, 2010, Amicus announced a definitive agreement with GlaxoSmithKline (GSK) to develop and commercialize migalastat HCl. Under the terms of the agreement, GSK received 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 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 disorders and stroke.
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 clinical development of Amicus' candidate drug products and the timing and reporting of results from 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 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, 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.
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