Amicus Therapeutics, Inc. (FOLD) announced additional positive data from a phase III study (Study 011) on the oral small molecule chaperone migalastat HCl, which is being developed as a monotherapy for treating Fabry disease patients with amenable mutations.
Amicus presented the results at the American Society of Human Genetics (ASHG) Annual Meeting. The results also included data from an open-label extension study (Study 041) on migalastat.
Additional data from Study 011 across all subgroups revealed that patients, when treated with migalastat, demonstrated continued stability in kidney function for an average period of 32 months. Additionally, patients with amenable mutations showed a reduction in disease substrate (globotriaosylceramide, or GL-3) when treated for six months with migalastat.
We remind investors that Amicus had reported positive data from two phase III studies on migalastat in April and August this year. Study 011 evaluated migalastat as a monotherapy, while Study 012 compared migalastat to enzyme replacement therapies (ERT) for Fabry disease.
Amicus will be meeting with regulatory agencies in the fourth quarter to discuss migalastat.
We note that prescribed treatments for Fabry disease include Sanofi’s (SNY) Fabrazyme among others.
We are pleased with the positive data on migalastat considering that it is one of the key candidates at Amicus. Meanwhile, the company is currently evaluating AT2220 in combination with ERT for Pompe disease.
Amicus currently carries a Zacks Rank #4 (Sell). Some better-ranked stocks in the health care sector include Emergent BioSolutions, Inc. (EBS) and Halozyme Therapeutics, Inc. (HALO). Both hold a Zacks Rank #1 (Strong Buy).
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