DART Therapeutics Develops Drug Candidate For Duchenne Muscular Dystrophy
"We know that making DMD a chronic, manageable disease will require a cocktail of therapies," said Dr. Diana Escolar, Chief Medical Officer of DART Therapeutics. "Phase one studies in adult volunteers show that DT-200 is safe and well-tolerated. Through our work with this SARM, we intend to explore the value of increasing muscle mass and potentially strength in diseased muscle, which could lead to development of a therapy that addresses a key unmet need in the future DMD cocktail."
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 pediatric DMD or other neuromuscular disorders will follow if these studies are positive.
"DART was created in part to explore the unanswered questions in DMD," said Gene Williams, CEO of DART. "Our model gives us a unique opportunity to ask the difficult questions and commit research dollars to areas that may have been neglected due to time and cost constraints normally associated with traditional biotech firms. We are optimistic that SARMs will play a role in the eventual DMD cocktail and may yield benefits for several other rare diseases, which we hope will benefit children beyond the DMD community."
Galapagos completed a phase one study of its SARM drug candidate in healthy volunteers, which showed positive results in terms of exposure and tolerability. Several Proof of Concept studies in MDX mice, the most accepted model for understanding muscle pathology in DMD, demonstrated short- and long-term ability to prevent fatigability after sustained exercise. DART Founders Charley's Fund and the Nash Avery Foundation funded a portion of the early studies.
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