Halo Therapeutics, LLC, a clinical-stage biopharmaceutical company developing novel therapeutics for rare fibrotic diseases, announced today that the TREAT-NMD Advisory Committee on Therapeutics (TACT) has reviewed HT-100, Halo's drug candidate for Duchenne muscular dystrophy (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. In its review of HT-100, TACT found the drug candidate to be "ready for the clinic."
"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. "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 approximately 1 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 U.S. Food and Drug Administration recently granted orphan drug designation to HT-100. The company will begin a phase 2 study in the second half of 2012.
"We submitted HT-100 for a TACT review knowing that informed, objective feedback on our pre-clinical data and development plans would be invaluable to our efforts," said Marc Blaustein, CEO of Halo Therapeutics. "We believe their recommendations for HT-100's advancement will hasten our progress toward delivering a safe and effective new therapy for DMD patients and their families."