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Amicus Therapeutics Provides Updates And Final Analysis Plan For Phase 3 Fabry Monotherapy Study 012

Stocks in this article: FOLD

Last Patient Completes 18-Month Primary Treatment Period - Top-Line Data on Track to Report in 3Q14 96% of Patients with Amenable Mutations Elected to Continue in 12-Month Treatment Extension Statistical Analysis Plan Finalized

CRANBURY, N.J., June 30, 2014 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq:FOLD), a biopharmaceutical company at the forefront of therapies for rare and orphan diseases, today provided updates and detailed the statistical analysis plan for its second Phase 3 study ( Study 012) of the oral small molecule pharmacological chaperone migalastat HCl ("migalastat") monotherapy for Fabry patients with amenable mutations. The 18-month primary treatment period is now complete and top-line data from Study 012 are expected in the third quarter of 2014. If successful, Study 012 will trigger the process for European regulatory approval of migalastat as a monotherapy for Fabry patients with amenable mutations.

Study 012 is the first clinical study to compare oral migalastat to standard-of-care enzyme replacement therapies (ERTs) for Fabry disease (Fabrazyme® and Replagal®). The primary outcome measure is renal function at 18 months. This open-label registration study enrolled 60 patients (26 males and 34 females) with Fabry disease with amenable mutations who had been treated with ERT for a minimum of 12 months prior to study entry. These patients were randomized 1.5:1 to switch to migalastat (36 patients) or remain on ERT (24 patients) for the primary 18-month treatment period, after which they were eligible to receive migalastat in a 12-month extension phase. Among the 60 patients enrolled, 56 (34 in the migalastat group and 22 in the ERT group) had amenable mutations in a GLP-validated human embryonic kidney (HEK) cell-based in vitro assay ("GLP HEK amenable").

Study 012 (The ATTRACT Study, or AT1001-012) Patient Disposition Highlights

  • Following randomization, 34 of 36 patients who switched to migalastat and 18 of 24 patients who continued with ERT completed the primary 18-month treatment period.
  • Among patients completing the 18-month primary treatment period, 32 out of 34 in the migalastat group and 16 out of 18 in the ERT group had GLP HEK amenable mutations.
  • 97% of patients with GLP HEK amenable mutations in the migalastat group (31 out of 32) elected to continue to receive migalastat in the 12-month treatment extension.
  • 94% of patients with GLP HEK amenable mutations in the ERT group (15 out of 16) elected to switch from ERT to migalastat for the 12-month treatment extension.

John F. Crowley, Chairman and Chief Executive Officer of Amicus Therapeutics, Inc., stated, "We are very pleased to be advancing our Fabry monotherapy chaperone program into the final stages of drug development for Fabry patients with amenable mutations. Following on the heels of our recent positive results from our first Phase 3 registration Study 011, we are on track to report data in the third quarter of this year from Study 012, in which patients have volunteered to switch from ERT to our oral chaperone migalastat as their only therapy for Fabry disease. Pending positive data from this Study 012, we expect to submit a marketing application for migalastat monotherapy in Europe. We also plan to meet with the Food and Drug Administration in the fourth quarter of this year to discuss the data from both of our Phase 3 Fabry monotherapy studies, to determine the fastest U.S. registration pathway for migalastat. Together these studies represent the largest registration program ever conducted in Fabry disease. Hopefully, migalastat will become an important new medicine for the treatment of Fabry disease, where so much unmet need remains."

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