Boston, MA 04/29/2014 (wallstreetpr) – Amicus Therapeutics, Inc. (NASDAQ:FOLD) announced today that the late stage trail of its fat storage disorder treating drug showed effective results.
The biopharmaceutical company, formulating treatments for orphan and rare diseases, obtained positive data for 12 and 24 months’ study of the migalastat monotherapy in patients suffering from Fabry and having amenable mutations. This was a Stage three trail for the drug.
Result Keypoints
Study 011 measured the decline in disease substrate after the treatment. The 24 month study followed a 6 month placebo therapy and patients who received the therapy reported higher decline in GL3 in contrast to placebo for the first 6 months. However, the results were less significant when observed for the study’s original preliminary endpoint. It was also noted that kidney functions in patients having amenable mutations were stable for 24 months.
The results showed that patients who received migalastat following a 6 month placebo treatment demonstrated significant decline in kidney interstitial capillary GL3 in the 12th month. Those who received mogalastat for 12 months showed durable decline. In addition, a decline of disease substrate was also noticed in an important disease biomarker, plasma lyso-Gb3, in those who shifted to migalastat from placebo. The drug was found to be overall well tolerated and safe.
Further Studies
The therapy is now being observed in two late phase studies called Study 011 and Study 012 as well as in an open label study called Study 014 for Fabry patients having amenable mutations. The company expects to receive top line data from Study 012 by the third quarter of this year. The chief aim of this study is to measure kidney function (GFR) after 18 months of therapy. The results will be measured against the enzyme replacement therapy, the usual treatment option for Fabry disease.
Amicus Therapeutics, Inc. (NASDAQ:FOLD)’ shares surged almost 33% in pre- trading hours after the announcement. The shares closed at $1.84 yesterday.
