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.
Study 012 Statistical Analysis Plan:Taking into account scientific advice from European regulatory authorities, the pre-specified co-primary outcome measures of efficacy in Study 012 are the descriptive assessments of comparability of the mean annualized change in measured (iohexol) glomerular filtration rate (mGFR) and estimated GFR (eGFR) for migalastat and ERT. Both mGFR and eGFR are considered important measures of renal function. Success on mGFR and eGFR will be measured in two ways:
- A 50% overlap in the confidence intervals between the migalastat and ERT treatment groups; and
- Whether the mean annualized changes for patients receiving migalastat are within 2.2 mL/min/1.73 m2/yr of patients receiving ERT.
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