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Amicus Therapeutics Announces Positive Results From Two Phase 1 Clinical Studies of AT2220 for Pompe Disease
Date:10/23/2007

CRANBURY, N.J., Oct. 23 /PRNewswire-FirstCall/ -- Amicus Therapeutics, a biopharmaceutical company developing small molecule, orally-administered pharmacological chaperones for the treatment of a range of human genetic diseases, announced today positive results from two recently completed Phase 1 clinical studies of AT2220 (1-deoxynojirimycin HCl) for Pompe disease. The Phase 1 results show that AT2220 was well-tolerated in healthy volunteers with good oral bioavailability and pharmacokinetic parameters. These results will be presented at the American Society of Human Genetics (ASHG) Annual Meeting on October 23-27 in San Diego, CA.

AT2220 is designed to selectively bind to and stabilize acid a-glucosidase (GAA), the enzyme deficient in Pompe disease. This deficiency leads to lysosomal accumulation of glycogen inside muscle cells, which is believed to cause the various symptoms of Pompe disease. AT2220 facilitates proper trafficking of the enzyme to the lysosomes, the compartments in the cell where it is needed to break down glycogen. AT2220 has been shown to increase GAA levels in cell lines derived from Pompe patients, in transfected cells expressing mutant forms of GAA, and in healthy mice and monkeys.

Two double-blind, placebo-controlled, dose escalation Phase 1 studies in healthy volunteers were completed. These studies were designed to evaluate the safety, tolerability and pharmacokinetics of AT2220. In a single ascending dose study, 32 individuals received oral doses of 50, 150, 300, or 600 mg AT2220 or placebo. In a multiple ascending dose study, 24 individuals received oral doses of 50, 150, or 450 mg/day AT2220 or placebo for 7 days. In both studies, AT2220 was generally safe and well-tolerated at all doses and was orally bioavailable with a plasma half-life of 4 to 5 hours. There were no drug-related serious adverse events and no adverse events were considered to be definitely or probably related to study treatment. In the multiple asce
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