Amicus Announces Positive Results From Clinical Studies Of AT2220 For Pompe Disease

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Pompe Disease

Amicus Therapeutics announced 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.

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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 ascending dose study, all possibly-related adverse events were mild in severity and resolved spontaneously.

The Company expects to initiate a Phase 2 clinical trial of AT2220 for Pompe disease in early 2008. "We are pleased to be able to advance this program forward in development and explore the potential for AT2220 as a new therapeutic option for people living with Pompe disease," said John F. Crowley, President and CEO of Amicus Therapeutics.

About Pompe Disease

Pompe disease affects an estimated 5,000-10,000 patients worldwide 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 1 and 2 years of age. The late onset form of the disease begins between childhood and adulthood and has a slower rate of progression that is characterized by musculoskeletal and pulmonary symptoms that usually lead to progressive weakness and respiratory insufficiency. The U.S. Food and Drug Administration's Office of Orphan Products Development has granted orphan drug designation for the active ingredient in AT2220 in the United States.

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