The U.S. Food and Drug Administration (FDA) has granted Fast Track designation to Akashi Therapeutics’ most advanced product candidate, HT-100 (delayed-release halofuginone), an orally available, small molecule drug candidate intended to reduce fibrosis and inflammation and promote healthy muscle regeneration in boys with DMD. Fast track designation is granted by the FDA to facilitate the development and expedite the review of new drugs that are intended to treat serious or life-threatening conditions and that demonstrate the potential to address unmet medical needs. Akashi is currently treating patients with DMD in a phase 1b/2a multi-center clinical program to evaluate HT-100 safety and tolerability and assess trends in a range of exploratory biomarkers and efficacy endpoints. There were no serious or clinically significant adverse events in the first 17 DMD patients to receive HT-100 for up to 92 days at doses ranging from 0.3 to 1.2 milligrams per day. The preliminary data on the 12 boys who received repeated doses of HT-100 indicate that the drug is having the desired effects on muscle tissue with respect to fibrosis.
