With a Duchenne MD patient struggling for life, little Akashi suspends study

Nonprofit-backed Akashi Therapeutics has suspended a trial of its lead drug for Duchenne muscular dystrophy (DMD) after a patient on the highest dose experienced "life-threatening health issues." 

"We do not yet know to what extent the patient's health issues are related to HT-100 and/or to other factors," the biotech reported on Monday. But after talks with the FDA, the Cambridge, MA-based biotech says that it decided to suspend dosing of HT-100 while it investigated the incident.

Most of the attention in the DMD world has focused on BioMarin ($BMRN) and Sarepta ($SRPT), two public companies that have run into some heavy headwinds at the FDA, which has raised serious questions about the data accumulated so far on their drugs. Akashi, meanwhile, has quietly advanced HT-100 into a Phase Ib/IIa study with substantial help from Charley's Fund, the Muscular Dystrophy Association and the Nash Avery Foundation.

Just days ago the biotech reported that it had struck a $100 million collaboration with Germany's Grünenthal Group for HT-100, with the German company taking on responsibility for all post-Phase II work.

Akashi CEO Marc Blaustein

HT-100 is a small molecule designed to tamp down on inflammation and spur muscle growth, an approach that CEO Marc Blaustein has said could work as part of a future cocktail of remedies for DMD patients.

At an interim point of the study last summer, Akashi--formerly Halo--said that the 10 patients in the early study demonstrated a significant increase in muscle strength after at least 6 months of treatment at a low dose, with no serious adverse events to report. The patient now struggling for life was on a high dose of 60 μg/kg/d.

A spokesperson for the company tells FierceBiotech that there is no new information available on the patient's condition.

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