Keywords: Halo Therapeutics, Duchenne muscular dystrophy, Halofuginone, Move into clinical trials

Halo Thera lead candidate for Duchenne ready for the clinic

Article | 20 March 2012

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Privately-held US clinical stage biopharma firm Halo Therapeutic says that an independent review of its lead drug candidate, HT-100 for the potential treatment of Duchenne muscular dystrophy (DMD), has been positive and the compound is ready to move into clinical studies.

Halo noted that the TREAT-NMD Advisory Committee on Therapeutics (TACT) has reviewed HT-100, for DMD, and believes the compound has potential based on the proposed mechanism. TREAT-NMD is a global network dedicated to expediting the delivery of promising new therapies to patients with neuromuscular disease. TACT provides independent, objective guidance on the therapeutic potential of drug candidates (novel or repurposed), which are submitted for review on a voluntary basis.

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"TACT offers the opportunity for objective review of potential new therapies to help industry, researchers and patient advocacy groups. The review provides multi-disciplinary advice on the development of the potential therapies in the context of a realistic development pathway," said Volker Straub of TREAT-NMD, adding: "TACT evaluated the proposal from Halo Therapeutics and felt it was well prepared, and that the compound has potential based on the proposed mechanism. Overall the plan to progress to a clinical trial is realistic."

DMD is a progressive and fatal neuromuscular disorder that afflicts around one in 3,500 boys worldwide. HT-100, Halo's proprietary formulation of halofuginone, is an orally available small molecule drug candidate intended to reduce fibrosis and promote healthy muscle fiber regeneration. HT-100 works by inhibiting the pathological fibrotic process in muscle and directly stimulating healthy muscle fiber regeneration. The US Food and Drug Administration recently granted orphan drug designation to HT-100. The company will begin a Phase II study in the second half of 2012.