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.