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Amicus Therapeutics And GlaxoSmithKline Announce Top Line 6-Month Primary Treatment Period Results From First Phase 3 Fabry Monotherapy Study

Stocks in this article: FOLD

CRANBURY, N.J. and LONDON, Dec. 19, 2012 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq:FOLD) and GlaxoSmithKline plc (GSK) today announced the 6-month primary treatment period results from the first Phase 3 global registration study ( Study 011) of investigational oral migalastat HCl monotherapy in males and females with Fabry disease who had genetic mutations identified as amenable to migalastat HCl in a cell-based assay. Study 011 randomized a total of 67 patients to receive oral migalastat HCl 150 mg or placebo on an every-other-day (QOD) dosing schedule during a 6-month, double-blind primary treatment period.

Reduction of GL-3 Substrate in Kidney Interstitial Capillaries:

Globotriaosylceramide (GL-3) is the lipid substrate that accumulates in tissues of patients with Fabry disease, most notably in the kidney. GL-3 clearance from the kidney interstitial capillaries has been used as a marker of treatment effect in Fabry disease. The pre-designated primary endpoint of Study 011 was a responder analysis evaluating the number of patients who demonstrated a 50% or greater reduction in kidney interstitial capillary GL-3 after 6 months of treatment with migalastat HCl compared to placebo. During a 6-month open label follow-up period all patients received migalastat HCl. The FDA has also indicated that it will consider the 12-month efficacy and safety data from Study 011. The paired kidney biopsies from baseline and month 6 were assessed by histological scoring using the published, quantitative Barisoni Lipid Inclusion Scoring System with Virtual Microscopy (BLISS-VM). 1 This methodology will also be utilized for the evaluation of the kidney biopsies at month 12. Amicus and GSK remain blinded to the 12-month data.

In Study 011 patients with evaluable baseline biopsies, 13/32 (41%) in the migalastat HCl treatment group demonstrated a 50% or greater reduction in kidney interstitial capillary GL-3 after 6 months of study treatment versus 9/32 (28%) in the placebo group. This difference did not achieve statistical significance (p=0.3) according to the pre-specified primary endpoint analysis.

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