Biohaven Pharmaceutical Holding Company Ltd. (NYSE:BHVN) today announced results from a focused analysis of treatment with verdiperstat in the HEALEY ALS Platform Trial in amyotrophic lateral sclerosis (ALS). The HEALEY ALS Platform Trial, the first-ever platform trial in ALS, is designed as an adaptive trial to evaluate multiple investigational treatments simultaneously, thus accelerating the development of effective and breakthrough treatments for people living with ALS.
Verdiperstat did not statistically differentiate from placebo on the prespecified primary efficacy outcome, disease progression measured by the ALS Functional Rating Scale-Revised and survival, nor the key secondary efficacy measures during the 24-week study period. Initial analysis of safety data was consistent with the overall profile of verdiperstat from prior clinical trial experience. Additional analyses are ongoing, and complete study results will be presented at an upcoming scientific meeting.
Irfan Qureshi, M.D., Senior Vice President of Neurology at Biohaven, stated, “We are incredibly grateful to our collaborators at the Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital, HEALEY ALS Platform Trial study investigators, and the ALS community – especially people with ALS and their families –– who made the trial possible. While we are disappointed that verdiperstat did not demonstrate efficacy for ALS, Biohaven remains committed to developing treatments for people who suffer from neurodegenerative diseases.”
