US-based biotechnology firm DART Therapeutics is developing a selective androgen receptor modulator (SARM) drug candidate for Duchenne muscular dystrophy (DMD).
DMD is a pediatric rare disease that affects approximately one in 3,600 boys worldwide, and is caused by a genetic mutation that renders boys unable to make functional dystrophin, a protein critical for normal muscle function.
The drug candidate is renamed as DT-200, which is an orally available SARM, proved to be potential to increase muscle size and strength in the earlier studies.
DT-200 offers potential benefit for multiple neuromuscular diseases without negative effects of oral androgenic steroids.
Obtained from Belgium-based clinical stage biotech company Galapagos, the SARM drug candidate has showed positive results in terms of exposure and tolerability during phase one study.
DART Therapeutics CEO Gene Williams said, "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."
The company will initiate phase 2a study with normal muscle followed by abnormal muscle wasting to assess the effects of DT-200 in increasing lean body mass, muscle strength, in the next half of 2013.
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).
