Results

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.


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