Duchenne Muscular Dystrophy Drug to Be Developed by Grünenthal and Akashi Therapeutics

The Grünenthal Group, an international research and development pharmaceutical company, announced it is developing its Duchenne muscular dystrophy (DMD) medicine HT-100 in partnership with Akashi Therapeutics, a clinical stage biopharmaceutical company that focuses on the development of therapies for DMD and which until now has been funded entirely by DMD patient foundations.

HT-100 (delayed-release halofuginone), currently in clinical phase 1b/2a in five U.S. hospitals, is a small molecule drug candidate taken orally for the treatment of DMD patients primarily through its ability to reduce fibrosis and inflammation and promote muscle fiber regeneration. The medicine candidate has been granted orphan drug designation in the U.S. and the EU — meaning it has been commercially undeveloped due to its limited profitability — and fast-track designation in the U.S. — an FDA process that aims to facilitate the development and patients’ reach to novel therapies for unmet medical needs.

The partnership is key to Grünenthal’s portfolio expansion in the development of innovative therapies for rare conditions, as is the case of DMD, which is a rare but still incurable form of muscular dystrophy. Grünenthal, with headquarters in Germany, will commercialize the drug in Europe and Latin America, while Akashi Therapeutics, of Cambridge, Mass.,  will retain the rights for commercialization in the U.S. and other markets. The agreement also states that Grünenthal will assume all post-Phase 2 global development costs.

Dr. Klaus-Dieter Langner, chief scientific officer of Grünenthal, said in a press release, “We are very excited about this unique collaboration with Akashi. At Grünenthal, we are highly committed to innovation and have been focusing on bringing innovative therapies to patients with high medical need. We are very motivated to use all our strength for the development of HT-100 together with our partners from Akashi and the patient groups supporting them.”

Marc Blaustein, CEO of Akashi Therapeutics Inc. said about the new partnership: “Grünenthal brings to our collaboration outstanding scientific and intellectual capital, significant financial resources, and a powerful commitment to positively impact the treatment of DMD. Akashi was founded to bring safe and effective treatments to DMD patients, and we look forward to working with our new partners to accelerate achievement of this goal.”