Amicus gains after Amigal meets in Phase III

Amicus Therapeutics Inc. (NASDAQ:FOLD) rose $0.93 (20%) to $5.50 on Wednesday after reporting that Amigal migalastat ( AT1001) met the co-primary endpoints of comparability to enzyme replacement therapy (ERT) on two measures of kidney function in the Phase III ATTRACT (Study 012) trial to treat Fabry's disease. The measures were mean annualized estimated glomerular filtration and mean annualized measured glomerular filtration rates evaluated over the 18-month treatment period. According to Amicus, comparability was defined as either a 50% or greater overlap in the confidence intervals between the treatment groups; or mean annualized changes in the rates for patients receiving Amigal that is within a prespecified level compared with patients receiving ERT. By early next year, Amicus said it will submit an MAA to EMA for Amigal based primarily on data from Study 012. Next quarter, the company plans to meet with FDA to discuss a U.S. regulatory path for the small molecule that enhances alpha galactosidase A activity.

The Study 012 data came from 56 patients who had received standard ERT for 12 months prior to enrolling in the double-blind trial and who had genetic mutations amenable to Amigal. According to Amicus, about 30-50% of the worldwide Fabry's population has amenable mutations, which are defined as mutations leading to a 3% absolute increase in wild-type alpha galactosidase A activity and a 20% relative increase when exposed to Amigal in a cell-based in vitro assay. Amicus estimates that Fabry's affects 5,000-10,000 people worldwide. ...