Amicus Therapeutics Records Positive Results For Migalastat Phase 3 Studies

(RTTNews) - Amicus Therapeutics (FOLD) Tuesday announced positive 12- and 24-month data from its Phase 3 study of oral small molecule chaperone migalastat HCl monotherapy in Fabry patients with amenable mutations.

The company said that subjects who switched from placebo to migalastat after the sixth month demonstrated a statistically significant reduction in kidney interstitial capillary GL-3 at month 12.

Subjects who remained on migalastat for 12 months demonstrated a durable reduction in kidney interstitial capillary GL-3 and plasma lyso-Gb3, another biomarker of disease.

Kidney function remained stable over 18 to 24 months. Of the 41 patients with GLP HEK amenable mutations who completed Study 011, 35 remained in the voluntary extension study.

Back in February 2013, the company had reported that patients on migalastat experienced greater reductions of disease substrate GL-3 compared with placebo in the initial 6-month period. However, the difference was not statistically significant under the original primary endpoint, which was for a 50 percent reduction threshold on the sixth month.

After consulting with the U.S. Food and Drug Administration, the company altered its plan to pre-specify the primary analysis at the month 12 as the mean change in GL-3.

Currently, migalastat monotherapy is being investigated in two Phase 3 registration studies (Study 011 and Study 012) and an open-label extension study (Study 041) in Fabry patients with amenable mutations. Top-line data are anticipated in the third quarter of 2014 from Study 012.

The primary analysis in Study 012 will be GFR, a clinical measure of kidney function, over 18-months of treatment with migalastat compared to enzyme replacement therapy (ERT), the current standard of care for Fabry disease.