Date: 2013-03-20
Type of information: Initiation of preclinical development
phase:
Announcement: initiation of development
Company: DART Therapeutics (USA)
Product: DT-200
Action
mechanism: DT-200 is a SARM drug candidate obtained from Belgium-based Galapagos NV. In early studies, the drug candidate demonstrated significant potential to increase muscle size and strength. 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.
Disease: Duchenne muscular dystrophy
Therapeutic area: Genetic diseases - Neuromuscular diseases - Rare diseases
Country:
Trial details:
Latest
news: * On March 20, 2013, DART Therapeutics, a new-model biotechnology firm focused on developing therapies for Duchenne muscular dystrophy (DMD), has announced 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.
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. 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.
