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Amicus Therapeutics Presents Positive Results From Phase 1 Clinical,Studies of Plicera for Gaucher Disease

Phase 2 Clinical Trials Initiated

CRANBURY, N.J., March 21, 2007 /PRNewswire/ -- Amicus Therapeutics, a biopharmaceutical company developing small molecule, orally-administered pharmacological chaperones for the treatment of a range of human genetic diseases, announced today that it will present positive results from its recently completed Phase 1 clinical studies of Plicera(TM) (isofagomine tartrate, AT2101) for Gaucher disease at the of Medical Genetics (ACMG) Annual Meeting on March 21-25 in Nashville, TN. The Phase 1 results show that Plicera was well-tolerated and that oral administration resulted in a significant elevation of target enzyme levels in healthy volunteers. Based on these results, Amicus announced today the initiation of two Phase 2 clinical trials of Plicera for Gaucher disease.

Plicera is designed to selectively bind to and stabilize GCase, the enzyme deficient in Gaucher disease. This deficiency leads to lysosomal accumulation of glucocerebroside inside certain cells, which is believed to cause the various symptoms of Gaucher disease. Plicera facilitates proper trafficking of the enzyme to the lysosomes, the compartments in the cell where it is needed to break down glucocerebroside.

Phase 1 Plicera data being presented at ACMG

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 Plicera. In the first study, 36 subjects received a single dose of one of five dose levels of Plicera. The second study was a multiple-dose study in which 18 subjects received one of three dose levels of Plicera once daily for 7 consecutive days. In both studies, Plicera was safe and well tolerated at all doses. There were no serious adverse events and no subjects withdrew or discontinued due to an adverse event. In the multiple-dose study, a dose-depend
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