Cambridge company testing muscular dystrophy drug

Cambridge’s DART Therapeutics Inc., a company focused on developing therapies for Duchenne muscular dystrophy (DMD), is 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. Galapagos has provided the rights for its SARM drug candidate in DMD to the patient foundations Charley’s Fund and the Nash Avery Foundation, who co-founded DART Therapeutics.

Terms of the rights transfer from Charley’s Fund and Nash Avery to DART were not disclosed.

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“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.

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.

DMD is a pediatric rare disease that affects approximately 1 in 3,600 boys worldwide. It is caused by a genetic mutation that renders boys unable to make functional dystrophin, a protein critical for normal muscle function. Young men with the disease show progressive signs of physical impairment as early as age three, lose the ability to walk in their teens, and die of cardiac or respiratory failure in their late 20s or early 30s.