DART Therapeutics Inc., an innovative, new-model biotechnology firm focused on developing therapies for Duchenne muscular dystrophy (DMD), has initiated a phase 1b/2a study of its lead drug candidate, HT-100 (delayed-release halofuginone). The phase 1b study (with a six-month 2a extension) in patients will determine the safety and tolerability of different, increasing doses of HT-100, and explore trends in a range of efficacy endpoints. HT-100 is an orally available, small molecule drug candidate intended to reduce fibrosis and inflammation and promote healthy muscle fiber regeneration in boys with DMD. The phase 1b/2a clinical program will enroll 30 boys and young men with DMD, both ambulatory and non-ambulatory. DART’s study will include boys aged six through 20, an atypical age range that allows investigators to evaluate HT-100’s safety in a broad population as well as study its effect in different disease stages. Researchers will also evaluate a new endpoint that could make DMD studies faster, more precise, less expensive and inclusive of a larger group of boys.
