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Amicus Therapeutics Presents Data from Clinical Ex Vivo Response Study and Phase 1 Studies of AT2220
Date:3/13/2008

Data Suggest Majority of Pompe Patients May Be Amenable to Chaperone

Therapy

CRANBURY, N.J., March 13 /PRNewswire/ -- Amicus Therapeutics, a biopharmaceutical company developing small-molecule, orally administered pharmacological chaperones for the treatment of human genetic diseases, announced today that the Company will present positive results from an ex vivo response study and three Phase 1 studies of AT2220 (1-deoxynojirimycin HCl), Amicus' compound in development for the treatment of Pompe disease, at the American College of Medical Genetics (ACMG) Annual Meeting from March 12-16 in Phoenix, AZ. The results of the ex vivo response study along with the previously reported results of the Phase 1 studies support moving into Phase 2 trials in the first half of 2008.

Ex Vivo Response Study data

Interim data will be presented from an ex vivo response study designed to test the effect of AT2220 on various Pompe mutations. Blood and skin samples were collected from 30 Pompe patients (26 adults, 3 juveniles and 1 infant) with a variety of different mutations in acid alpha-glucosidase (GAA), the target enzyme in Pompe disease. Cells derived from these samples were then tested to determine whether treatment with AT2220 caused an increase in the level of GAA. Of the 26 patients with available data, 24 had cells that showed a dose responsive increase in GAA levels, including 22 patients who had at least 1 copy of the common splice site mutation IVS1-13T>G. It has been reported that more than 80% of Caucasian adult Pompe patients have at least 1 copy of this common splicing mutation.

Phase 1 AT2220 data

A total of 72 healthy volunteers were treated in three double-blind, placebo-controlled, dose escalation Phase 1 studies designed to evaluate the safety, tolerability and pharmacokinetics of AT2220. Across all three studies, AT2220 was shown to be generally safe and well tolerated at all doses. The
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SOURCE Amicus Therapeutics
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