Amicus Therapeutics (FOLD) Updates on Study 011 in Fabry Disease

Amicus Therapeutics (Nasdaq: FOLD) 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 poster1 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.

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.

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