CRANBURY, N.J., Feb. 9, 2011 /PRNewswire/ -- Amicus Therapeutics (Nasdaq: FOLD) today announced the dosing of the first subject in a Phase 2 clinical trial designed to evaluate the co-administration of its investigational drug Amigal™ (migalastat hydrochloride) with enzyme replacement therapy (ERT) for Fabry disease. This open-label Phase 2 study is investigating drug-drug interactions between Amigal and the ERTs Fabrazyme® and Replagal®. Preclinical studies have demonstrated that coadministration of Amigal results in a prolonged circulating half-life of ERT, increased enzyme activity in cells, and greater substrate reduction in target tissues compared to that seen with ERT alone. Amigal is being developed in collaboration with GlaxoSmithKline (GSK), as part of an alliance between GSK Rare Diseases and Amicus.
John F. Crowley, Chairman and CEO of Amicus, stated "The commencement of this clinical study is an important milestone for Amicus and for pharmacological chaperone technology. Based on encouraging preclinical results we believe the co-administration of Amigal with ERT has the potential to provide significant benefit for people with Fabry disease. The advancement of this approach is a part of our vision to provide two new treatment options for Fabry patients in the future—Amigal as a monotherapy or Amigal co-administered with ERT. Finally, we also believe this study will provide us with valuable proof of concept for applying this approach to other lysosomal storage diseases and more broadly to other indications treated with therapeutic proteins."
Phase 2 Study Design
The Phase 2 trial will evaluate the safety and pharmacokinetics of Amigal and ERT when co-administered in 18 male patients with Fabry disease, ages 18-65, who have been receiving ERT for at least one month before study entry. Patients enrolled in this study need not have a genetic mutation responsive to Amigal as a monotherapy. Each patient will receive ERT alone and then ERT after administration of a single oral dose of Amigal. There will be two cohorts of nine patients treated with one of two Amigal dose levels.