FOLD - Amicus falls after announcing late-stage data for AT-GAA trial in Pompe disease

Amicus Therapeutics (FOLD) has lost 21.3% in the post market after the company announced the topline results of its Phase 3 PROPEL pivotal trial for AT-GAA in the treatment of late-onset Pompe disease (“LOPD”).The 52-week, double-blind randomized global study evaluates the efficacy, safety, and tolerability of the two-component experimental therapy, AT-GAA (cipaglucosidase alfa and miglustat) compared to the current standard of care, alglucosidase alfa.Randomized in a 2:1 basis with either AT-GAA or alglucosidase alfa, the trial involved 123 adult Pompe patients who still had the ability to walk and to breathe without mechanical ventilation.77% were being treated with alglucosidase alfa (n=95) immediately prior to enrollment and 23% had never been treated with any enzyme replacement therapy such as  alglucosidase alfa (n=28).The primary endpoint of the study was the mean change in 6-minute walk distance vs. baseline measurements at 52 weeks and the first key secondary endpoint was the mean change in percent-predicted

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Amicus falls after announcing late-stage data for AT-GAA trial in Pompe disease