Profile: Amicus Therapeutics, Inc. (FOLD.O)

Amicus Therapeutics, Inc., incorporated on February 4, 2002, is a clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of orally administered, small molecule drugs, known as pharmacological chaperones, for the treatment of a range of human genetic diseases. The Company’s products in development are Amigal (migalastat hydrochloride) for Fabry disease, Plicera (isofagomine tartrate) for Gaucher disease and AT2220 (1-deoxynojirimycin HCl) for Pompe disease. The Company completed its Phase II clinical trials of Amigal and are conducting Phase II clinical trials of Plicera and AT2220.

Amigal for Fabry Disease

Amicus Therapeutics, Inc. is developing Amigal for the treatment of Fabry disease. In parallel with the Phase III regulatory discussions, 23 of the original 26 patients who participated in the Phase II clinical trial of Amigal continue to be treated with Amigal in the voluntary Phase II extension study to characterize the long term safety and efficacy and evaluate additional doses and dose regimens. Amigal is an orally administered, small molecule pharmacological chaperone.

Plicera for Gaucher Disease

Plicera is an orally administered, small molecule, pharmacological chaperone for the treatment of Gaucher disease. The Company completed Phase I clinical trials, which demonstrated that Plicera was safe and well tolerated in healthy subjects at all doses tested. It also conducted a four week Phase II study in Gaucher patients who switched from enzyme replacement therapy (ERT) with imiglucerase to Plicera. Results showed that Plicera was safe and tolerated at all doses and increased target enzyme activity levels in a majority of patients. In the trial, b-glucocerebrosidase (GCase) activity, as measured in white blood cells, was increased in 20 of the 26 patients with evaluable GCase data, and five of the six patients without a clear increase were either in the lowest dose cohort or the cohort dosed least frequently.

The Company is conducting a Phase II clinical trial of Plicera in Type I Gaucher patients. This study, GAU-CL-202, involves subjects with Type I Gaucher disease who are naive to ERT and substrate reduction therapy. The study is designed to evaluate the safety of Plicera and its effect on parameters that are commonly abnormal in Gaucher disease, including levels of red blood cells, platelets, liver and spleen volumes and other biomarkers related to Gaucher disease. Patients will be assigned to one of two treatment arms and will receive treatment with Plicera for approximately six months.

AT2220 for Pompe Disease

AT2220 is an orally administered small molecule pharmacological chaperone for the treatment of Pompe disease. The Company initiated a Phase II clinical trial in June 2008, in adult Pompe patients who are naive to ERT. The trial includes an 11-week treatment period with an optional extension study. The objectives of the trial include the evaluation of the safety and pharmacodynamics of multiple doses and regimens of AT2220. In addition, Amicus Therapeutics, Inc. is conducting preclinical animal studies to evaluate the effects of administering AT2220 in combination with ERT.

Other Programs

The Company’s pharmacological chaperone technology is applicable to the development of drugs for the treatment of a range of human genetic and other diseases. It is engaged in researching the use of pharmacological chaperones for the treatment of diseases other than lysosomal storage disorders, including neurological diseases, such as Parkinson’s disease. An epidemiological and biochemical link between Gaucher and Parkinson’s disease has been established. In 2008, it generated data in rodents demonstrating that brain exposure for Plicera is about 18% of plasma levels.

The Company competes with Genzyme Corporation, Shire, Actelion, Ltd. and Protalix Biotherapeutics.