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Amicus Therapeutics Announces Positive 12- And 24-Month Data From Phase 3 Fabry Monotherapy Study 011

Migalastat Demonstrated Statistically Significant (p=0.013) and Durable Substrate Reductions on 12-Month Pre-Specified Primary Analysis in Fabry Patients with Amenable Mutations

Statistically Significant (p<0.0001) Reduction Also Seen in Important Fabry Disease Biomarker, Plasma Lyso-Gb3

Kidney Function Remained Stable Up to 24 Months in Fabry Patients with Amenable Mutations

85% of Patients with Amenable Mutations Completing Month 24 Remain in Ongoing Voluntary Extension Study (Study 041)

Conference Call and Webcast Today at 8:00 a.m. ET

CRANBURY, N.J., April 29, 2014 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq:FOLD), a biopharmaceutical company at the forefront of therapies for rare and orphan diseases, today announced positive 12- and 24-month data from its first Phase 3 study ( Study 011) of the oral small molecule chaperone migalastat HCl ("migalastat") monotherapy in Fabry patients with amenable mutations. Detailed results are available in a slide presentation that will be shared by the Amicus management team on a conference call today at 8:00 a.m. ET. Please visit

Study 011 was designed to measure the reduction of disease substrate (Globotriaosylceramide, or GL-3) following treatment with migalastat. The 24-month study began with a 6-month double-blind, placebo-controlled treatment period, after which all patients were treated with migalastat for a 6-month open-label follow-up period and a subsequent 12-month open-label extension phase. The study also measured clinical outcomes, including renal function, as secondary endpoints.

As previously reported, patients on migalastat experienced greater reductions in GL-3 as compared to placebo during the initial 6-month period; however, this difference was not statistically significant under the original study primary endpoint (responder analysis with a 50% reduction threshold at month 6). Following a Type C Meeting with the U.S. Food and Drug Administration (FDA) in the second quarter of 2013, and based on feedback from the agency at that meeting, Amicus revised the Statistical Analysis Plan to pre-specify the primary analysis at month 12 as the mean change in GL-3 in patients with amenable mutations in a GLP-validated human embryonic kidney (HEK) cell-based in vitro assay ("GLP HEK amenable").

Summary of Study 011 12- and 24-Month Data in GLP HEK Amenable Patients
  • Subjects who switched from placebo to migalastat after month 6 demonstrated a statistically significant reduction in kidney interstitial capillary GL-3 at month 12 (p=0.013).
  • Subjects who remained on migalastat for 12 months demonstrated a durable reduction in kidney interstitial capillary GL-3.
  • Reduction in disease substrate was also observed in plasma lyso-Gb3, another important biomarker of disease, in subjects who switched from placebo to migalastat (p<0.0001). Subjects who remained on migalastat demonstrated a durable reduction in lyso-Gb3.
  • Kidney function (estimated glomerular filtration rate (eGFR), iohexol mGFR) remained stable over 18-24 months
  • Migalastat was generally safe and well-tolerated.
  • Of 41 subjects with GLP HEK amenable mutations who completed Study 011, 35 (85%) remain in the voluntary extension study ( Study 041).

John F. Crowley, Chairman and Chief Executive Officer of Amicus Therapeutics, Inc., stated, "Today is a great day for Amicus and the Fabry community. We are pleased to report that the 12 and 24 month results from Study 011 have met our pre-defined criteria for success in terms of substrate reduction at 12 months, as well as clinical measures of kidney function maintained out to 24 months. We believe these data provide important validation that a small-molecule chaperone can restore the function of a patient's own enzyme in patients with amenable mutations, and that our pharmacogenomic assays can identify these patients. Together these results demonstrate the power of personalized medicine in rare diseases and offer the prospect of a new treatment option that differs from traditional enzyme replacement therapy. Pending positive data from our second Phase 3 study we expect to meet with regulatory authorities to discuss these data and determine the fastest registration pathway for migalastat."

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