Amicus Therapeutics to initiate Phase 1 study of AT2220

Amicus Therapeutics (Nasdaq: FOLD) today announced it plans to initiate a Phase 1 study of AT2220 (1-deoxynojirimycin HCl), its investigational drug in development for the treatment of Pompe Disease. The primary objective of this study is to evaluate the pharmacokinetics of AT2220 in muscle tissue in healthy adult subjects. The U.S. Food and Drug Administration (FDA) has agreed to Amicus' proposal for the Phase 1 study and subsequently converted the clinical hold of AT2220 to a partial hold to allow the conduct of this study.

In June 2008, the Company announced the commencement of a Phase 2 clinical trial of AT2220 in adults with Pompe disease based on data from both preclinical and Phase 1 studies. In February 2009, the Company announced it had suspended enrollment after two patients enrolled in the trial experienced serious adverse events that were probably related to treatment with AT2220. The AT2220 Investigational New Drug application (IND) was subsequently placed on clinical hold by the FDA.

The Company has completed a thorough evaluation of all data from these two subjects and has completed additional preclinical studies of AT2220. Based on these data, the Company proposed the planned Phase 1 study to FDA in order to further evaluate the pharmacokinetics of AT2220 in muscle, the key target tissue in Pompe disease. This study will be an open label, single dose study and will commence in the fourth quarter of this year. The Company expects data from this trial to be available in the first quarter of 2010. Based on the results of this study, the Company will determine the appropriate next steps for the program working in close collaboration with the FDA.

One of the recently completed preclinical studies evaluated the effects of various doses and regimens of AT2220 on glycogen reduction in an appropriate transgenic mouse model of Pompe disease. Preliminary results of this study demonstrate consistent glycogen reduction across a range of different tissues, including heart, diaphragm and multiple skeletal muscles. Glycogen is the substrate that accumulates in the cells of patients with Pompe disease, which is believed to result in the clinical symptoms of the disease. The Company expects to present the results of this and other preclinical studies at appropriate scientific conferences in 2010.

Additionally, Amicus continues to be encouraged by the results of preclinical studies designed to evaluate the use of AT2220 in combination with enzyme replacement therapy (ERT). The Company expects to report additional data from these studies at scientific conferences in 2010.