Biohaven skunked by a placebo in PhII/III flop for ataxia drug, shares slide

Biohaven $BHVN helped prove just how fast you could assemble a pipeline and a successful biotech IPO this year. And now it has shown just how risky that strategy can be.

The New Haven, CT-based company reported that its Phase II/III study of trigriluzole demonstrated not only that it didn’t work better than a placebo in treating spinocerebellar ataxia, but patients in the study also reported in larger numbers that the placebo helped them better than the drug.

The primary was a change from baseline on the score on the Scale for the Assessment and Rating of Ataxia — with the drug hitting a -0.81 point improvement compared to a -1.05 point improvement in the sugar pill arm.

Biohaven CEO Vlad Coric

The company’s stock dropped 28% on the news. Biohaven, a Yale spinout, raised $168 million from its IPO just last May.

The study is a flop, but it’s not over.

Biohaven says the long-term, open-label extension study is continuing, with a shot at detecting a drug signal down the road. But the company’s statement also leaned heavily on its lead CGRP migraine drug study now in Phase III. That drug was bought in from Bristol-Myers Squibb after they bagged the drug following a study in which it was beat out by a cheap generic.

Biohaven also assured investors that thanks to its IPO, it has $204 million in cash as a cushion for the next 12 months.

Biohaven CEO Vlad Coric had this to say today:

We are obviously disappointed that today’s topline clinical results do not support continued development of trigriluzole as a symptomatic agent for patients with SCA, a devastating neurologic disorder for which novel treatments are urgently needed. This was the largest SCA clinical trial performed to date and important knowledge has been generated — we plan to share our data with the ataxia clinical leaders and the National Ataxia Foundation to help refine clinical trials in this therapeutic area.

The best place to read Endpoints News? In your inbox.

Comprehensive daily news report for those who discover, develop, and market drugs. Join 47,200+ biopharma pros who read Endpoints News by email every day.

Free Subscription

Research Scientist - Immunology
Recursion Pharmaceuticals Salt Lake City, UT
Director of Operations
Atlas Venture Cambridge, MA

Visit Endpoints Careers ->