CRANBURY, N.J. and ORLANDO, Fla., Feb. 15, 2013 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq:FOLD), today announced additional 6-month (Stage 1) results from the first ongoing Phase 3 global registration study ( Study 011) of investigational oral migalastat HCl monotherapy (150 mg, every-other-day) in males and females with Fabry disease who had genetic mutations identified as amenable to migalastat HCl in a cell-based assay. Stage 1 results were highlighted in an oral platform presentation 1 at the Lysosomal Disease Network WORLD Symposium ( LDN WORLD) by Dr. Fatih Ezgu, Gazi University.
Study 011 consists of a 6-month, double-blind period (Stage 1) when subjects received migalastat HCl 150 mg or placebo, a 6-month open label-follow up period (Stage 2) when all patients received migalastat HCl, and an ongoing 12-month open-label extension.
Initial top-line Stage 1 results previously reported in December, 2012 for the primary endpoint in Stage 1 did not meet statistical significance. The pre-specified primary and secondary analyses of the primary endpoint numerically favored migalastat HCl over placebo. In the primary responder analysis, 13/32 (41%) in the migalastat HCl group versus 9/32 (28%) in the placebo group demonstrated a 50% or greater reduction in kidney interstitial capillary GL-3 from baseline to month 6 (p=0.3). Taken alone the secondary analysis of the absolute percent change in kidney interstitial capillary GL-3 from baseline to month 6 showed a median reduction of 41% in the migalastat HCl group versus a median reduction of 6% in the placebo group (p=0.093).Study 011 entry criteria, particularly elevated urine GL-3, were intended to enrich for patients with higher interstitial capillary GL-3, and more measurable disease burden. Although all patients had detectable interstitial capillary GL-3 at baseline, a number of patients had low levels of GL-3 at baseline, making it difficult to detect a significant difference in responders between the 2 treatment groups. Clearance of kidney interstitial capillary GL-3, a marker of treatment effect, is being measured by histology 2 in evaluable kidney biopsies from baseline to month 6 (Stage 1) as well as baseline to month 12 (Stage 2). Stage 2 results remain blinded at this time. Dr. Ezgu said, "The 6-month data from Study 011 are encouraging, including a post-hoc subgroup analysis presented today, and we continue to evaluate patients in this ongoing study. There is still an unmet medical need for Fabry disease treatments. Migalastat HCl may potentially offer an oral treatment for Fabry patients with amenable mutations based on these results from Study 011 and earlier clinical studies, and potentially forthcoming data from ongoing studies."