DART Therapeutics Inc., an innovative, new-model biotechnology firm focused on developing therapies for Duchenne muscular dystrophy (DMD), announced today that it is developing a SARM 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. DT-200 could represent a new class of therapy for DMD, a muscle-wasting disease, and offer potential benefit for multiple neuromuscular diseases where improved muscle strength and function would be beneficial. 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.
Selective androgen receptor modulators--or SARMs--promote increased muscle mass and thereby strength--through normal androgenic pathways without the negative effects of oral androgenic steroids. DT-200 is an orally available SARM that has demonstrated potential to increase muscle size and strength in preclinical studies. In DMD, SARMs could be useful in halting progressive muscle loss by increasing the size and strength of diseased muscle. However, this remains an unexplored area of DMD therapy. Although the drug effects in normal muscle cells are likely to be seen, researchers do not yet know if SARM therapy can make diseased muscle cells larger and if their growth equates to increased muscle strength. If DART demonstrates the value of SARMs for DMD, the therapy could be a valuable part of a future drug cocktail as muscle cells that remain alive through the effects of other components of the cocktail would be larger, stronger and possibly more resistant to damage. DART's research may also establish the benefit of SARMs for other neuromuscular diseases such as FSHD (Facioscapulohumeral muscular dystrophy), Spinal Muscular Atrophy, Charcot Marie Tooth and ALS (Amyotrophic lateral sclerosis).
"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."