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Amicus Therapeutics Begins Phase 2 Clinical Trial of AT2220 in Pompe Disease
Date:6/3/2008

ulmonary and skeletal muscle function. Participants who complete the study may be eligible to participate in a voluntary extension study that will further evaluate the effect of AT2220 on these functional parameters.

Additional information about the Phase 2 study will be posted at ClinicalTrials.gov.

Initiation of the Phase 2 study of AT2220 follows completion of an ex vivo response study as well as multiple Phase 1 studies of AT2220 in healthy volunteers. The ex vivo response study was designed to test the effect of AT2220 on various Pompe mutations. Blood and skin samples were collected from 30 Pompe patients (26 adults, three juveniles and one infant) who had a variety of GAA mutations. Cells derived from each patient were then treated with AT2220. Of the 26 patients with available data, 24 had cells that showed a dose responsive increase in GAA activity including 22 patients who had at least one copy of the common splice site mutation IVS1-13T>G. Data from the Phase 1 studies in 72 healthy volunteers demonstrated that AT2220 was generally safe and well tolerated at all doses evaluated with no drug-related serious adverse events.

Amicus is developing AT2220 as part of a strategic collaboration with Shire Human Genetic Therapies (HGT), a business unit of Shire plc, to develop and commercialize Amicus' three lead pharmacological chaperone compounds for lysosomal storage disorders. Under the agreement, Shire received commercial rights outside of the United States. Amicus retains all U.S. rights.

About Pompe Disease

Pompe disease affects an estimated 5,000-10,000 individuals world-wide and is clinically heterogeneous in the age of onset, the extent of organ involvement, and the rate of progression. The early onset form of the disease is the most severe, progresses most rapidly, and is characterized by musculoskeletal, pulmonary, gastrointestinal, and cardiac symptoms that usually lead to death from cardio-respiratory failure between
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SOURCE Amicus Therapeutics
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