Amicus Thera leaps on positive migalastat results

US biotech firm Amicus Therapeutics (Nasdaq: FOLD) has released positive 12- and 24-month data from its first Phase III study (Study 011) of the oral small-molecule chaperone migalastat HCl monotherapy in Fabry patients with amenable mutations, with the news pushing the firm’s shares 33% higher in premarket trading.

As previously reported, patients on migalastat experienced greater reductions in GL-3 compared to placebo during the initial six-month period; however, this difference was not statistically significant under the original study primary endpoint (responder analysis with a 50% reduction threshold at month six).

Following a Type C Meeting with the US Food and Drug Administration in the second quarter of 2013, and based on feedback from the agency at that meeting, Amicus revised the Statistical Analysis Plan to pre-specify the primary analysis at month 12 as the mean change in GL-3 in patients with amenable mutations in a GLP-validated human embryonic kidney (HEK) cell-based in vitro assay.