Trial restart approval brings back DMD hope for Akashi
- The Food and Drug Administration has given Akashi Therapeutics the go-ahead to restart a Phase 1b/2a clinical trial of its Duchenne muscular dystrophy (DMD) drug, HT-100 (oral delayed-release halofuginone), after the company suspended dosing and recruitment of the trial over a patient with "serious health issues" and his subsequent death.
- HT-100 is in development to reduce fibrosis and inflammation in DMD; the study, HALO-DMD-04, will look at safety, tolerability, pharmacokinetics and pharmacodynamics of a lower dose without the addition of antiemetics.
- Akashi, which hopes to restart the study as soon as possible, is in discussion with investors and partners over the development and commercialization of this and other DMD drugs in its pipeline. This may affect the timeline of the clinical trial.
Akashi is now picking itself up and starting all over again with HT-100, and also aims to begin clinical trials for two other DMD drugs, DT-200 and AT-300, during 2017.
"Our goal continues to be improving the lives of patients with DMD and other muscle function diseases," said Marc Blaustein, CEO of Akashi Therapeutics. "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."
Akashi is part of a growing field of DMD drugmakers. Development of these drugs has been plagued by poor efficacy and safety issues. Yet, the genetic disease first appears in young children and therefore has spurred many parents to become advocates, who have helped pressure regulators to approve therapies that only provide minimal benefits.
While a good-sized handful of drugs for DMD are in late stages of development, pretty much all of them have been stymied on their route to market at some point or another.
In July 2016, Santhera Pharmaceuticals got a slap-down from the Food and Drug Administration for its request for accelerated approval for Raxone (idebenone), a drug to improve lung function in DMD.
Catabasis Pharmaceuticals' edasalonexent missed its primary endpoint in part B of a three-part study; interim results of part C are expected in the second quarter of 2017.
PTC Therapeutics' Translarna (ataluren) hasn't been able to make it to a U.S. submission so far, with the FDA issuing a refuse to file letter for its new drug application, and only has a conditional approval in Europe so far.
BioMarin ditched its DMD drug Kyndrisa (drisapersen) back in May 2016 when the European Medicines Agency indicated that its committee would recommend rejection.
Sarepta Therapeutics finally got conditional approval from the FDA in September 2016 for Exondys 51 (eteplirsen). Yet, the accelerated approval process will require further studies, and was perhaps unexpected after an FDA advisory committee voted against approval earlier in the year. The drug was the first DMD agent approved, and will cost around $300,000 a year, but is getting pushback or reluctant coverage from payers.
Even post-approval things remain fraught. Marathon Pharmaceuticals finally got its approval in February this year for Emflaza (deflazacort), a decades old steroid that had already been used off-label in DMD, after receiving a refusal to file letter and then filing over protest. The company faced backlash about the high price of $89,000 a year for the drug, finally caving under the pressure and selling the drug to PTC Therapeutics for $140 million.
- Akashi Therapeutics Statement
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