Akashi suspends enrollment in Duchenne muscular dystrophy study

• Non-profit-backed Akashi Therapeutics suspends enrollment and dosing in its clinical trial, HALO, assessing lead product candidate and Orphan Drug- and Fast Track-tagged HT-100 in patients with Duchenne muscular dystrophy (DMD) after one patient developed serious, life-threatening health issues subsequent to receiving the highest dose in the study, 60 ug/kd/day. The company is working with the FDA to analyze the situation.
• This is the only instance of this severity observed in HT-100's clinical program. An interim safety and efficacy analysis released in June 2015 showed promising therapeutic effects with no serious drug-related adverse events. The company intends to restart the trial after the analysis is complete and the safety issues fully vetted.
• Earlier this month, the company inked a $100M global agreement with Germany's Grunenthal to develop HT-100 for DMD.
• HT-100 (delayed-release halofuginone) is an orally available small molecule designed to reduce fibrosis (scarring) and inflammation and promote healthy muscle fiber regeneration in patients with DMD.
• Previously: Akashi Therapeutics and Grunenthal to jointly develop DMD med (Jan. 8)
• Previously: Akashi Therapeutics' Duchenne MD candidate shows encouraging results in early stage study (June 18, 2015)
• DMD-related tickers: (SRPT -1.5%)(BMRN -0.8%)(PFE +0.1%)(SMMT)(MRNA +18.1%)(PTCT -0.7%)
• Update: Sadly, the patient died. The company has initiated a comprehensive investigation of all patient records with a particular focus on this patient. It will continue to work closely with the FDA and outside experts in its effort to fully understand all aspects of the situation and to insure that all study participants are safe.