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news.Akashi Therapeutics has formed a $100m partnership with Grünenthal Group for a global drug development program for the investigational therapeutic HT-100, an orally available small molecule drug candidate in Phase 1b/2a for the reduction of fibrosis and inflammation, and promotion of healthy muscle fiber regeneration in Duchenne muscular dystrophy (DMD).
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HT-100 has been granted Orphan Drug Designation by the U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA).
Akashi Therapeutics CEO Marc Blaustein said: "This agreement represents a critical turning point for the company and offers powerful new hope for DMD patients and their families.
"We are thrilled to find a partner who shares our commitment to this patient community, and look forward to accessing Grünenthal’s world-class scientific, clinical, regulatory and commercial capabilities to accelerate development and broad global availability of HT-100."
Under the terms of the agreement, Grünenthal will make upfront and milestone payments to Akashi. In addition, the company will assume all post-Phase 2 global development costs through commercialization of an approved product.
Grünenthal gains commercialization rights in Europe and Latin America, while Akashi retains rights for the U.S. and other markets. Akashi will receive royalties on net sales.
In total, Grünenthal plans to commit more than $100m to the partnership and will receive royalties on U.S. net sales in exchange for funding development of Akashi’s U.S. commercial infrastructure.
Grünenthal chief scientific officer Klaus-Dieter Langner said: "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.
The agreement also lays the groundwork to expand the collaboration to include Akashi’s other pipeline compounds for DMD and to explore additional indications for HT-100, which may also have activity in other fibrotic diseases such as scleroderma and idiopathic pulmonary fibrosis.
About HT-100
HT-100 (delayed-release halofuginone) is an orally available, small molecule drug candidate designed to reduce fibrosis and inflammation and promote healthy muscle fiber regeneration in DMD patients. HT-100 has been granted orphan designation for DMD in both the U.S. and E.U., and fast track designation in the U.S. A phase 1b/2a clinical program is currently underway at five hospitals in the U.S.