Drug Development

Staggered by a Duchenne patient’s death, Akashi finally gets a green light to get back into the clinic

Marc Blaustein, CEO

Akashi Therapeutics was put in limbo more than a year ago after the FDA forced the little biotech to slam the brakes on its development effort for a new drug to treat Duchenne muscular dystrophy following the death of a patient in an early-stage study. But 13 months later Akashi says it now has a green light from regulators to get back into the clinic with HT-100.

Akashi is slashing the dose to 150 µg/day after testing a range that extended from 300 µg/day to 1500 µg/day. And at the time Akashi said that the patient who died was on a high dose of the drug HT-100. The biotech is also ending the use of antiemetic therapies.

HT-100 (delayed-release halofuginone) 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. The drug is a synthetic version of an ingredient found in plants which investigators say blocks pro-inflammatory Th17 cells.

Blaustein told Xconomy that the patient’s blood pressure fell dangerously low after — not unnaturally — building up levels of halofuginone. The symptoms weren’t caught though because he was taking an anti-nausea medicine that masks symptoms, which is why they’re requiring patients to stop using those therapies.

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.

Now Akashi says it plans to get back in the clinic as soon as possible while pursuing talks with investors and possible partners. The timing on that remains up in the air.

The Duchenne MD field is dominated by seemingly unending controversy. The latest development to roil R&D came with the FDA’s controversial approval of an old generic steroid — deflazacort — as an orphan therapy for Duchenne. But after running into a buzz saw of controversy when it priced the therapy at $89,000 a year, Marathon sold it to PTC Therapeutics for $140 million up front.

PTC, meanwhile, has been grappling with the FDA’s refusal to review ataluren, which has now failed three straight trials, but remains on the market in Europe.

Sarepta won an approval for its drug eteplirsen, but now has a label that states there’s no clear evidence that it works.

Hope springs eternal, though, and Akashi plans to push ahead. The company is also working on DT-200 (selective androgen receptor modulator) and AT-300 (cation channel modulator).

“Our goal continues to be improving the lives of patients with DMD and other muscle function diseases,” sais Blaustein in a prepared statement. “We are pleased that the FDA has agreed with our conclusion that it is appropriate to resume development of HT-100 and look forward to moving ahead with the trial as quickly as possible.”


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