A little more than a week after Cambridge, MA-based Akashi Therapeutics suspended its study of a new drug for Duchenne muscular dystrophy (DMD) after a patient was sent to the hospital, the biotech reports that the trial subject has died.
"It is with great sorrow that we share that the brave young man who was experiencing serious, life-threatening health issues has passed away," the company said in a statement to FierceBiotech. "We offer our deepest condolences to his family and loved ones. Akashi has initiated a comprehensive investigation, beginning with detailed data reviews and in vitro studies, to evaluate the extent to which the patient's health issues are related to HT-100 and/or to other factors. We will share updates as we reach conclusions in the investigation."
The company did not respond to a query from FierceBiotech about the age of the patient who died. DMD age groups for these studies typically skew toward boys and adolescents. But the company acknowledged that the FDA has stepped in.
"Akashi voluntarily suspended the highest dose cohort immediately upon learning of the situation," the company stated. "The FDA then asked the company to suspend all cohorts of the trial to allow us time to better understand the circumstances that led to this patient's experiences. We continue discussions with the FDA and will provide additional information once the investigation is complete."
With some crucial support from nonprofits like Charley's Fund, the Muscular Dystrophy Association and the Nash Avery Foundation, little Akashi was able to take its therapy into a Phase Ib/IIa study while striking a $100 million collaboration with Germany's Grünenthal Group.
The lion's share of attention in DMD has focused on two controversial late-stage therapies from BioMarin ($BMRN) and Sarepta Therapeutics ($SRPT). BioMarin's candidate, acquired in its acquisition of Prosensa, was recently rejected by the FDA, while Sarepta's treatment was treated to a scathing review from regulators in the lead-up to an upcoming panel review.
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 who has now died was on a high dose of 60 μg/kg/d.