Biohaven's ALS Drug Fails To Meet Study Endpoint, Second Therapy To Fail Trials In Recent Months

Biohaven's ALS Drug Fails To Meet Study Endpoint, Second Therapy To Fail Trials In Recent Months
  • Biohaven Pharmaceutical Holding Company Ltd BHVN announced results from a focused analysis of verdiperstat in the HEALEY ALS Platform Trial in amyotrophic lateral sclerosis (ALS). 
  • Verdiperstat did not statistically differentiate from placebo on the prespecified primary efficacy outcome, disease progression measured by the ALS Functional Rating Scale-Revised, survival, or key secondary efficacy measures during the 24-week study period. 
  • Initial safety data analysis 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, Senior Vice President of Neurology at Biohaven, stated, "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."
  • In May, Biohaven announced topline Phase 3 results of troriluzole in patients with spinocerebellar ataxia (SCA). 
  • It did not reach statistical significance on the primary endpoint of the severity of symptoms in the overall SCA population as there was less than expected disease progression over the study. 
  • In May, Pharma giant Pfizer Inc PFE agreed to acquire the company for almost $11 billion.
  • Price Action: BHVN shares are down 0.26% at $151.53 on the last check Thursday.

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