Amicus Therapeutics, Inc.
1 Cedar Brook Drive
Amicus Therapeutics is a biopharmaceutical company at the forefront of developing therapies for rare and orphan diseases. The Company is developing novel, first-in-class, small molecules called pharmacological chaperones, to treat human genetic diseases, including lysosomal storage diseases. The Company’s lead pharmacological chaperone migalastat HCl is in Phase 3 as a monotherapy for Fabry disease. Amicus and its partner GlaxoSmithKline Plc (GSK) are conducting Phase 3 registration studies of migalastat HCl for the treatment of Fabry disease. A Phase 3 study intended to support approval of migalastat HCl in the United States is fully enrolled and results are anticipated in the third quarter of 2012. Additionally, Amicus and GSK are currently conducting a separate Phase 3 study intended to further support global registration of migalastat HCl.
Amicus continues to advance its program evaluating the use of pharmacological chaperones in combination with enzyme replacement therapy (ERT) as an expansion of the chaperone technology platform. Two chaperone-ERT combinations, migalastat HCl co-administered with ERT for Fabry disease and AT2220 (duvoglustat HCl) co-administered with ERT for Pompe disease, are currently in Phase 2 development. The Company is also investigating chaperone-ERT combinations for Gaucher disease in preclinical studies.
Amicus also has a preclinical pipeline of pharmacological chaperones for diseases of neurodegeneration, including AT3375 for Parkinson’s disease. The Company has broad, issued intellectual property around the methods, use, and composition of pharmacological chaperones to treat genetic diseases and diseases of neurodegeneration. In addition to the currently issued patents, Amicus has several patent applications pending.