Small biotech advances on some big goals for Duchenne muscular dystrophy

By July 18, 2014 September 15th, 2014 News

FierceBiotech – July 3, 2014

Small biotech advances on some big goals for Duchenne muscular dystrophy

By John Carroll

Marc Blaustein never said it would be easy to develop a new drug for Duchenne muscular dystrophy. But even though he doesn’t have the biggest organization, the most money or a full set of human data on key endpoints, he has been making some steady, early progress on his quest.

This morning Blaustein, CEO of the renamed biotech Akashi Therapeutics (formerly Halo), unveiled a few new moves in that journey. The FDA awarded fast-track status for HT-100, an oral small molecule therapy that Blaustein believes could make it into one of the future cocktail therapies needed to help keep this lethal disease in check. A group of nonprofits, led by the Muscular Dystrophy Association, added some more cash to the company till, bringing the contribution of all the nonprofits up to $2.5 million–which is in addition to the money Charley’s Fund and the Nash Avery Foundation have invested as founders of the company. And he has the first preliminary snapshots on the drug’s activity on biomarkers for the disease in the first two groups totaling 12 patients in a Phase Ib/IIa study.

Two years ago, when Blaustein first talked to FierceBiotech, he was the only full-timer on the staff of Cambridge, MA-based Akashi. Now there’s a core team of 12 in the virtual organization, which includes some consultants. In addition to HT-100, which is slated to finish its Phase Ib/IIa study of 30 patients in 2015, Akashi now has another Duchenne therapy coming up behind it. And there are in-licensing talks underway as well to help build a pipeline.