CRANBURY, N.J., Feb. 13, 2012 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq:FOLD), a biopharmaceutical company at the forefront of therapies for rare and orphan diseases, recently presented updated information on the number and type of patients screened for and enrolled in the global Phase 3 registration study (Study 011) of the investigational pharmacological chaperone migalastat HCl, being studied as a potential monotherapy for Fabry disease.
At the 8th Annual Lysosomal Disease Network WORLD Symposium (LDN WORLD), Dr. Daniel Bichet, Universite de Montreal, gave an oral presentation titled, "Fabry Disease Mutations Addressable With Migalastat HCl, an Investigational Chaperone Therapy. Screening Results from FACETS, a Phase 3 Study in Male and Female Patients." Key findings presented by Dr. Bichet include:
- 140 Fabry patients (46 males and 94 females) with mutations in Alpha-galactosidase A (alpha-Gal A) had been screened for Study 011 as of July 2011
- Approximately 85% (119/140) of the patients screened carried missense mutations
- Approximately 82% (97/119) of those patients met one of the key enrollment criteria of having amenable mutations and were potentially eligible for the study.
Dr. Bichet stated, "A majority of the first 140 subjects screened in Study 011 had missense mutations, and most of these mutations were also amenable to migalastat HCl as a monotherapy. Given the entry criteria for Study 011, we generally screened patients that we believed were likely to have missense mutations. The clinical significance of having these mutations is unknown and can only be established in studies such as Study 011. We continued to gather data among patients screened through the close of enrollment, and look forward to sharing additional findings at a future scientific congress."
Update on Patients Enrolled in Study 011
Amicus and its collaborator GlaxoSmithKline (GSK) have enrolled 67 (24 males and 43 females) out of 180 total subjects screened in Study 011. The study completed enrollment in December 2011. All patients enrolled in the study have been diagnosed with Fabry disease, and were naïve to enzyme replacement therapy (ERT) or had not received ERT for at least six months prior to study entry.
In addition, each patient enrolled in Study 011 met further entry criteria, which included having alpha-Gal A mutations amenable to migalastat HCl monotherapy in vitro. For study purposes, all patients also needed to have urine globotriaosylceramide (GL-3) at least four times the upper limit of normal. A number of patients screened had amenable mutations but did not have sufficiently elevated urine GL-3 at baseline to qualify for enrollment into Study 011.
Elevated levels of GL-3 in urine have been shown to correlate with GL-3 levels in kidney tissue, including the interstitial capillaries. The primary efficacy endpoint for Study 011 is a change in interstitial capillary globotriaosylceramide (GL-3) as measured in kidney biopsies. Patients in Study 011 with a reduction of GL-3 deposits per capillary of at least 50% at six months will be considered responders.
John F. Crowley, Chairman and Chief Executive Officer of Amicus stated, "We are pleased that the 67 Fabry patients enrolled in Study 011 include a range of patients by gender, disease burden and genotype who are living with Fabry disease today. Study 011 is the first randomized, controlled Fabry study to ever include a large number of female patients, who represent more than half of all patients in Fabry disease registries. We look forward to seeing these study results in the third quarter of this year."
About Amicus Therapeutics
Amicus Therapeutics (Nasdaq:FOLD) is a biopharmaceutical company at the forefront of developing therapies for rare and orphan 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 migalastat HCl is in Phase 3 for the treatment of Fabry disease.
About Migalastat HCl
Migalastat HCI is an investigational oral pharmacological chaperone 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 co-administered with enzyme replacement therapy (ERT) for the treatment of Fabry disease.
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.
Forward-Looking Statements
This press release contains "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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