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Dart Therapeutics Develops Drug Candidate for Duchenne Muscular Dystrophy


3/20/2013 9:31:26 AM

CAMBRIDGE, Mass., March 20, 2013 /PRNewswire/ -- 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."

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.

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 twenties or early thirties.

About DART Therapeutics
DART Therapeutics Inc., Cambridge, Mass., is a biotechnology firm applying a new model for drug development to rare pediatric neuromuscular diseases. In the DART model, patient foundations join with biotechnology industry veterans to impact a central problem in rare diseases: Rapid therapy development. DART is focused on Duchenne muscular dystrophy, a fatal neuromuscular disease for which there is no effective treatment. For more information, please visit www.dartrx.com.

Media Contact:
Shanti Skiffington
617.921.0808
shanti.skiffington(at)gmail.com

SOURCE DART Therapeutics Inc.



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