The FDA had ordered a hold on the development of delayed-release halofuginone (HT-100) after a clinical-trial participant began experiencing life-threatening adverse effects two weeks after beginning the therapy.
HT-100 is an oral anti-inflammatory, anti-fibrotic agent designed to promote healthy muscle fiber regeneration in DMD patients.
“We are pleased to learn that the FDA is allowing Akashi to resume the clinical development of HT-100,” Pat Furlong, founding president and chief executive officer of Parent Project Muscular Dystrophy (PPMD), said in a press release. “Preventing fibrosis is an important target and an essential piece of a combination of therapies that will be required to end Duchenne.”
The trial was suspended when the patient, who was in the highest-dose group of the study, began experiencing the serious adverse effects. It wasn’t clear whether the patient’s problems were related to HT-100, however, so Akashi and the FDA agreed to investigate the situation further.
No other patients had experienced issues that severe. And patients in the lower-dose groups had received treatment for 11 to 19 months by then, suggesting good safety and no serious adverse events.
Although the final status of the patient has not been disclosed, the FDA decided to allow Akashi to continue developing HT-100.
Akashi plans to start a new trial, HALO-DMD-4, to evaluate the safety, tolerability, and properties of HT-100.
Previous studies showed that the lowest doses tested — as low as 300 µg/day — were as effective as the highest doses — up to ,1500 µg/day. So the new trial will start with a lower dose of 150 µg/day. In addition, patients will not be given anti-emetics to prevent vomiting and nausea.
The company said it plans to start the new trial as soon as possible.
“Our goal continues to be improving the lives of patients with DMD and other muscle function diseases,” said Marc Blaustein, chief executive officer 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.”
HT-100 has been granted orphan drug designation in the United States and European Union and fast track designation in the United States.
Parent Project Muscular Dystrophy will host a webinar with Akashi Therapeutics on Monday, March 27, to discuss HT-100. It will start at 1:30 p.m. U.S. Eastern Time.