HACKENSACK, N.J., Dec. 8, 2014 /PRNewswire-USNewswire/ --Parent Project Muscular Dystrophy (PPMD), the leading advocacy organization working to end Duchenne muscular dystrophy (Duchenne) announced today that it will award Akashi Therapeutics, Inc. (Akashi) a $500,000 grant to fund clinical trials to test the safety and efficacy of an investigational new drug known as HT-100 (delayed-release halofuginone). Duchenne muscular dystrophy is the most common fatal genetic disorder diagnosed in childhood, affecting approximately one in every 3,500-5,000 live male births. HT-100 is an orally available small molecule drug candidate being developed to reduce fibrosis and inflammation and to promote healthy muscle fiber regeneration in Duchenne patients. The application of HT-100 to Duchenne and other fibrotic diseases is based on pioneering work by Dr. Mark Pines at the Volcani Institute in Israel. Akashi, led by CEO Marc Blaustein, has been granted orphan designation for Duchenne in both the U.S. and EU, as well as Fast Track designation in the U.S. A phase 1b/2a clinical program is currently underway at five hospitals across the U.S. The grant will be used to evaluate the safety, tolerability and pharmacokinetics of HT-100 in patients with Duchenne in both a phase 1b open-label, single and multiple ascending dose study … Continue reading
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