Amicus Therapeutics Presents Updated Patient Screening Profiles From Phase 3 Fabry Study

CRANBURY, N.J., Sept. 6, 2012 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq:FOLD), a biopharmaceutical company at the forefront of therapies for rare and orphan diseases, today announced updated details on patients screened for one of the global Phase 3 registration studies ( Study 011) to investigate the pharmacological chaperone migalastat HCl for Fabry disease. These results were presented in a poster 1 at the Society for the Study of Inborn Errors of Metabolism (SSIEM) Annual Symposium 2012 ( SSIEM 2012).

Migalastat HCl is in Phase 3 development for Fabry disease in patients with genetic mutations that are amenable to this chaperone as a monotherapy. Study 011 - also referred to as FACETS - is one of two ongoing Phase 3 studies of migalastat HCl monotherapy being conducted by Amicus and GlaxoSmithKline (GSK). Updated screening profiles at SSIEM included the following:
  • A total of 180 Fabry patients (60 males and 120 females) were screened for Study 011. Prior to screening sites could have used genotype information when available to enrich for Fabry patients with amenable mutations who were more likely to be interested in participating.
  • Approximately 86% (154/180) of patients screened had missense mutations (compared to a current estimate in the Fabry population of approximately 60%)
  • Approximately 88% (136/154) of those patients, or 76% of patients screened, had alpha-galactosidase A mutations amenable to migalastat HCl monotherapy, and were potentially eligible for enrollment.
  • Approximately 50% (67/136) of those patients, or 37% of all patients screened, enrolled in Study 011 upon meeting all entry criteria, including: 1) naïve to ERT or had not received ERT for at least 6 months prior to study entry; 2) genetic mutations amenable to chaperone monotherapy and; 3) for study purposes, urine globotriaosylceramide (GL-3) levels at least four-times the upper limit of normal at baseline.

Pol F. Boudes, MD, Chief Medical Officer of Amicus Therapeutics stated, "Not all Fabry patients have been genotyped and new patients continue to be diagnosed. During screening for Study 011, we discovered 15 unique missense mutations that contribute to the growing database of new mutations within the broader Fabry population. We continue to believe the randomized population in Study 011 is fairly representative of a broad range of Fabry patients by gender, disease burden, and genotype."

Study 011 is a randomized study with a 6-month, double-blind, placebo-controlled primary treatment period and a 6-month, open-label follow-up period in which all subjects receive treatment with migalastat HCl. The primary endpoint in Study 011 is a change in interstitial capillary GL-3 as measured in kidney biopsy at 6 months versus baseline.  The 6-month primary treatment period in Study 011 was completed in the second quarter 2012 and the 6-month follow-up period is expected to complete in the fourth quarter 2012. Amicus and GSK expect to unblind and analyze data from the primary 6-month treatment arm during the fourth quarter 2012. Both companies remain blinded to all results at this time.

John F. Crowley, Chairman and Chief Executive Officer of Amicus stated, "Screening results from Study 011 suggest that migalastat HCl monotherapy may address a substantial portion of the Fabry population on the basis of genotype. In addition, these screening results may provide the Fabry patient and physician community with valuable information on a wide variety of different mutations, including newly discovered mutations."

1.     Migalastat HCl, an Investigational Pharmacological Chaperone Therapy: Screening Results from FACETS, a Phase 3 Study in Male and Female Fabry Patients. – Nicholls, K., Castelli J., Winkler R., Sitaraman, S., Benjamin E., Wu, X., Duke C., Boudes, P., on Behalf of FACETS Study AT1001-011 Principal Investigators.

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 human genetic diseases. Amicus' late-stage programs for lysosomal storage disorders include migalastat HCl monotherapy in Phase 3 for Fabry disease; migalastat HCl co-administered with enzyme replacement therapy (ERT) in Phase 2 for Fabry disease; and AT2220 co-administered with ERT in Phase 2 for Pompe 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). Over 600 mutations in the alpha-Gal A gene are associated with Fabry disease. 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, 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.


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