Akashi Therapeutics announced they are suspending both dosing and patient enrollment in the HALO trial – a study investigating their experimental therapy, HT-100, in patients with Duchenne muscular dystrophy.
The trial’s suspension came after a patient enrolled in the highest dose-group of the study began experiencing life-threatening adverse effects. At this time, Akashi does not know if the negative effects are related to HT-100 treatment and is currently investigating the situation together with the FDA.
HT-100 is an anti-inflammatory and anti-fibrotic agent developed to promote the regeneration of healthy muscle fibers in muscular dystrophy patients.
In a press release, the company reports that no other patients participating in the study have experienced health issues this severe. Patients in the lower dose groups have been treated for between 11 and 19 months with the drug — totaling more than 20 patient-years of data — suggesting good safety and no serious adverse events. The patient experiencing life-threatening health issues had been on the higher dose of HT-100 for about two weeks.
A report released in June 2015 showed that safety and efficacy of HT-100 in boys and men looked promising, with data supporting increased muscle strength and no serious adverse effects related to the treatment.
The decision to suspend the trial was taken together with the FDA and will allow time for a thorough investigation of the circumstances that led to the condition of the patient. Akashi reports that the HT-100 trial will be restarted once the analysis is complete, and both Akashi and the FDA have approved of potential measures that might be necessary to address the situation.
“As we are at the very beginning of the investigation, we cannot provide a projected timeline,” Akashi wrote in the press release. “As always, our first priority is patient safety.”
Other preclinical and clinical trials at earlier stages involving the compounds DT-200 and AT-300 will not be affected.