FDA permits Akashi Therapeutics to resume clinical trials of experimental DMD drug HT-100

Akashi Therapeutics on Wednesday announced that it received approval from the FDA to restart clinical studies of the experimental Duchenne muscular dystrophy (DMD) treatment HT-100. Development of the drug, a delayed-release formulation of halofuginone, was paused last year following the death of a patient in a clinical trial. Akashi CEO Marc Blaustein remarked "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 indicated that it plans to initiate a new study, dubbed HALO-DMD-04 at a new lower dose and eliminate the use of antiemetic therapies in patients with DMD.  Akashi noted that last year, a patient who had received a high dose of HT-100 for two weeks developed "serious, life threatening issues," while the patient's death was disclosed the following month. In its analysis, the drugmaker found that treatment with HT-100 in combination with repeated doses of the anti-nausea therapy ondansetron resulted in high levels of halofuginone through the body and dangerously low blood pressure.  

Blaustein remarked that generally, a "multi-day early-warning" appears before any serious health problems related to HT-100 occur, but these signals were masked by ondansetron. The CEO said that patients will not be permitted to take anti-nausea drugs during the HALO-DMD-04 study. 

HT-100, which Akashi explained is an oral small molecule designed to reduce fibrosis and inflammation and promote healthy muscle fibre regeneration in patients with DMD, has been awarded fast track designation by the FDA for the treatment of DMD, as well as orphan drug status in the US and EU. 

The drugmaker noted that it has held talks with potential investors and development partners regarding the clinical development and commercialisation of HT-100, in addition to the experimental DMD drugs DT-200 [https://www.firstwordpharma.com/node/1066363?tsid=33] and AT-300.