German pharmaceutical firm Grünenthal has entered a joint global drug development programme to develop US-based Akashi Therapeutics’ orally available small molecule drug candidate, HT-100, to treat patients with Duchenne muscular dystrophy (DMD).
Currently, HT-100 (delayed-release halofuginone) is in clinical Phase Ib/IIa at five hospitals across the US. The drug is designed to reduce fibrosis and inflammation, as well promoting healthy muscle fibre regeneration in these patients.
The deal will see Grünenthal become responsible for commercialisation of the product in Europe and Latin America, while Akashi will retain rights for the US and all other markets.
As part of the deal, Grünenthal will make combined upfront and milestone payments of around $100m to Akashi, which will also receive royalties on net sales.
In addition, the company will assume all post-Phase II global development costs through commercialisation of an approved product.
The German firm is also eligible for receiving royalties on US net sales in exchange for funding development of Akashi’s US commercial infrastructure.
Grünenthal chief scientific officer Dr 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 drug has secured orphan designation in both the US and the EU, in addition to fast-track designation in the US.
In future, the alliance could be expanded for improvements to treat boys and young men affected by DMD.
In 2014, the German firm unveiled its strategy to strengthen its focus from pain and inflammation to niche indications with high unmet medical need.
Akashi Therapeutics CEO Marc Blaustein said: "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."