DART Therapeutics Inc. is a company focused on developing therapies for Duchenne muscular dystrophy (DMD). The company is currently developing a drug candidate obtained from Belgium-based Galapagos NV. In early studies, the drug candidate, renamed DT-200, demonstrated significant potential to increase muscle size and strength in DMD patients. The drug candidate, a selective androgen receptor modulator (SARM), promotes increased muscle mass and thereby strength through normal androgenic pathways without the negative effects of oral androgenic steroids. According to the company, DT-200 could represent a new class of therapy for the muscle-wasting disease, and offer potential benefits for multiple neuromuscular diseases. In the second half of 2013, DART will initiate a phase 2a study in adults with normal muscle followed by a study in abnormal muscle wasting to assess the effects of DT-200 in increasing lean body mass, muscle strength, and motor function. This study will provide proof of concept that either normal muscle, abnormal muscle, or both can respond to SARMs, and that the effect in increasing muscle mass will be clinically beneficial. Further studies in paediatric DMD or other neuromuscular disorders will follow if these studies are positive.
