The Grünenthal Group will partner with Akashi Therapeutics to codevelop its Phase Ib/IIa candidate HT-100 (delayed-release halofuginone) for Duchenne muscular dystrophy (DMD), the companies said today. The collaboration could generate more than $100 million for Akashi, the companies added.

HT-100 is an oral small molecule drug candidate designed to reduce fibrosis and inflammation and to promote healthy muscle fiber regeneration in DMD patients. It has won orphan drug designations in both the U.S. and the EU, as well as the FDA’s fast track designation in the U.S. The drug is currently in clinical Phase Ib/IIa at five hospitals across the U.S.

Grünenthal agreed to oversee commercialization of HT-100 in Europe and Latin America, while Akashi will retain rights for the U.S. and all other markets. The partnership could be expanded in the future, the companies said. 

In return, Grünenthal agreed to make upfront and milestone payments to Akashi. Grünenthal said it plans to commit more than $100 million to the partnership, and also agreed to assume all post-Phase II global development costs through commercialization of an approved product.

Akashi will receive royalties on net sales—except for royalties on U.S. sales that Grünenthal will receive in exchange for funding development of Akashi's U.S. commercial infrastructure.

Akashi is a biopharma founded by patient organizations Charley's Fund and Nash Avery Foundation, working with biopharma industry veterans, to develop treatments for DMD. The company is developing a pipeline of therapies aimed at transforming DMD into a chronic but manageable condition.

“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 CEO Marc Blaustein said in a statement.

Grünenthal said the collaboration was a key milestone in its strategy, first articulated in 2014, of broadening its focus from pain and inflammation to niche indications with high unmet medical need.

Grünenthal CSO Klaus-Dieter Langner, Ph.D., added that, “as a mid-cap pharmaceutical company, we will be in a position to give the development program of HT-100 high priority in order to hopefully improve the devastating situation of DMD patients.”

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