HT-100, also known as halofuginone, is an orally available small molecule drug candidate that is being developed to lower fibrosis and facilitate healthy muscle fibre regeneration.
The company is pressing forward to start a Phase II study in 2012 to investigate HT-100 in DMD patients, said CEO Marc Blaustein.
Through the FDA's orphan designation, the sponsor may be eligible for a range of incentives including FDA grant funding for clinical study costs, tax credits related to development expenses, waiver of FDA user fees, and a seven-year period of marketing exclusivity in the US upon FDA approval.
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