- Biohaven Pharmaceutical ( NYSE: BHVN ) said Thursday that its candidate for rare neuromuscular disease amyotrophic lateral sclerosis (ALS), verdiperstat did not meet the main goal in a pivotal Phase 2/3 trial after 24 weeks of treatment.
- A focused analysis from the HEALEY ALS Platform Trial did not statistically differentiate verdiperstat from placebo for primary efficacy measure of disease progression, as measured by the ALS Functional Rating Scale-Revised and survival, the company said.
- The experimental therapy did not differentiate from placebo for secondary efficacy measures during the period.
- However, the initial data indicated that the safety profile of verdiperstat was in line with findings from prior studies. Further data analyses are underway, and the company expects to report complete study results at a future medical meeting.
- BHVN is currently on track to be acquired by Pfizer ( PFE ) for $11.6B after a buyout deal announced in May.
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Biohaven says ALS candidate failed in key trial