Patient-funded biotech in $100 million funding deal for DMD drug

By Amrutha Penumudi

(Reuters) – Akashi Therapeutics Inc, a U.S.-based biotech funded by DMD patient foundations, said it would get up to $100 million under a partnership with Germany’s Grunenthal SA for developing a drug for the muscle-wasting disorder.

DMD, or Duchenne Muscular Dystrophy, is a genetic disorder for which there is currently no treatment and is a keenly watched area in drug development.

The progressive degenerative disorder hampers muscle movement and affects one in 3,600 newborn boys, most of whom die by the age of 30.

Akashi, which until now was running trials on funds from more than 25 DMD patient foundations, will now be able to quicken development aggressively, CEO Marc Blaustein told Reuters.

“We also now have the resources to move our other drugs to the clinic,” he added.

Grunenthal will make upfront and milestone payments to Akashi on the development of its lead DMD drug, HT-100, currently being tested in early stage studies.

BioMarin Pharmaceuticals Inc, Sarepta Therapeutics Inc and PTC Therapeutics Inc are leading the development of a drug for the disorder.

An advisory committee to the U.S. Food and Drug Administration in November expressed doubt over the effectiveness of BioMarin’s drug, while Sarepta faces a similar panel by end-January.

Akashi is taking a novel approach to treat DMD, unlike BioMarin’s and Sarepta’s drugs that look to boost dystrophin production.

DMD is caused by a gene mutation that leads to low production of dystrophin, a protein that is an important part of the muscle tissue.

Akashi’s drug candidates focus on targeting the way the disease manifests itself, for instance, reducing muscle inflammation and promoting muscle fiber growth.

Apart from HT 100, which focuses on fibrosis, inflammation and muscle regeneration, Cambridge, Massachusetts-based Akashi is also testing DT-200, which increases muscle mass.

The third drug in the company’s pipeline, AT-300, which is derived from Tarantula venom, addresses calcium imbalances in muscles.

Grunenthal is a family owned pharmaceutical company known for its pain treatments.

(Reporting by Amrutha Penumudi in Bengaluru)

• Health
• Pharmaceuticals Drug Trials
• drug development
• DMD