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Akashi Therapeutics Presents Positive Clinical Data On HT-100 In Patients With Duchenne Muscular Dystrophy


7/3/2014 6:06:50 AM

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CAMBRIDGE, Mass.--(BUSINESS WIRE)--Akashi Therapeutics, Inc., a clinical stage biopharmaceutical company developing treatments for Duchenne muscular dystrophy, today announced the presentation of preliminary positive clinical data for its most advanced drug candidate, HT-100 (delayed-release halofuginone), an orally available, small molecule developed to reduce fibrosis and inflammation and promote healthy muscle regeneration in boys with Duchenne muscular dystrophy (DMD). Preliminary clinical data for HT-100 on the first DMD patient cohorts suggests promising signs of biological activity and a favorable safety profile to date in this ongoing phase 1b/2a multi-center clinical program to evaluate the safety and tolerability of increasing doses of HT-100 and assess trends in a range of exploratory biomarkers and efficacy endpoints. The preliminary Phase 1b/2a data was presented at the New Directions in Biology and Disease of Skeletal Muscle 6th Biennial Conference held in Chicago June 29 through July 2, 2014.

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