Amicus Therapeutics, a biopharmaceutical company at the forefront of developing therapies for rare and orphan diseases, announced that it has now achieved target enrollment and has 56 patients in its second Phase 3 study (Study 012) of migalastat HCl monotherapy for Fabry disease. Screening is now closed at participating sites, and final enrollment is anticipated by year-end 2012. Amicus in collaboration with GlaxoSmithKline (GSK) is developing the investigational pharmacological chaperone migalastat HCl for the treatment of Fabry disease.
Study 012 (The ATTRACT, or FAB-AT1001-012 Study) Highlights:
· First clinical study to compare oral migalastat HCl to standard-of-care ERTs (Fabrazyme and Replagal)
· Enrolled males and females with Fabry disease, who had genetic mutations amenable to migalastat HCl as a monotherapy, and were on ERT for a minimum of 12 months
· Primary outcome measure is renal function assessed by Glomerular Filtration Rate (GFR) at 18 months
John F. Crowley, Chairman and Chief Executive Officer of Amicus, stated, “We are very pleased to have met the enrollment objectives for this important Phase 3 Fabry monotherapy study ahead of the year-end target. The willingness of these Fabry patients to switch from an approved ERT to migalastat HCl to participate in this study highlights the unmet medical needs that persist in this community. We look forward to evaluating the effects of migalastat HCl, as well as currently used ERTs, on renal function over the course of 18 months of treatment in this study.”
Amicus and GSK are co-developing all formulations of migalastat HCl under a global Fabry collaboration. Migalastat HCl monotherapy is in Phase 3 development (Study 011 and Study 012) for Fabry patients with genetic mutations that are amenable to this chaperone monotherapy, as determined by a cell-based assay. Study 011 is a placebo-controlled study intended primarily to support U.S. registration, and Study 012 compares migalastat HCl to ERT to primarily support global registration. Migalastat HCl coadministered with ERT is in Phase 2 (Study 013) and migalastat HCl co-formulated with JCR Pharmaceutical Co. Ltd's proprietary ERT (JR-051, recombinant human alpha-Gal A enzyme) is in preclinical development. Amicus has commercial rights to all Fabry products in the United States and GSK has commercial rights to all of these products in the rest of world.
Date: October 22, 2012
Source: Amicus Therapeutics