Amicus Announces Positive Phase I Results for Pompe Disease Drug
Posted on: Wednesday, 24 October 2007, 15:00 CDT
Amicus Therapeutics has announced positive results from two recently completed double-blind, placebo-controlled, dose escalation, Phase I clinical studies of 1-deoxynojirimycin HCl for Pompe disease.
The phase I results show that 1-deoxynojirimycin was well-tolerated in healthy volunteers with good oral bioavailability and pharmacokinetic parameters. 1-deoxynojirimycin HCl (AT2220) is designed to selectively bind to and stabilize acid a-glucosidase (GAA), the enzyme deficient in Pompe disease. The deficiency leads to lysosomal accumulation of glycogen inside muscle cells, which is believed to cause the various symptoms of Pompe disease.
The 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 600mg AT2220 or placebo. In a multiple ascending dose study, 24 individuals received oral doses of 50, 150, or 450mg/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.
John Crowley, president and CEO of Amicus, said: "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."
Source: Datamonitor
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