Stock Report: Amicus Therapeutics Inc. (FOLD) Report Updated: Jun 17, 2013 | Print This Page

Amicus Therapeutics, Inc., a biopharmaceutical company, focuses on the discovery, development, and commercialization of orally-administered, small molecule drugs for the treatment of lysosomal storage disorders and diseases of neurodegeneration. Its drugs are known as pharmacological chaperones, which selectively bind to the target protein, enhance the stability of the protein, help it fold into the three-dimensional shape, as well as allow proper trafficking of the protein, thereby increasing protein activity, enhance cellular function, and reduce cell stress. The company focuses on the development of pharmacological chaperone monotherapy programs and pharmacological chaperones in combination with enzyme replacement therapy (ERT). Its pharmacological chaperone monotherapy programs for genetic diseases include migalastat HCl, which is in Phase III clinical trial for the treatment of Fabry disease; and AT3375, which is in preclinical stage for the treatment of Parkinson’s disease. The company’s pharmacological chaperone-ERT combination programs comprise Migalastat HCl co-administered with ERT for patients with Fabry disease receiving ERT treatment with any genetic mutation; AT2220 (duvoglustat HCl) co-administered with ERT for Pompe disease; AT3375 and afegostat tartrate co-administered with ERT for Gaucher disease; and pharmacological chaperone programs for other lysosomal storage diseases. Amicus Therapeutics, Inc. has a license and collaboration agreement with Glaxo Group Limited to develop and commercialize migalastat HCl. The company was founded in 2002 and is based in Cranbury, New Jersey.