Dive Brief:
- Biohaven Pharmaceutical's BHV-0223, a sublingual formulation of the amyotrophic lateral sclerosis (ALS) drug riluzole, reduced anxiety compared with placebo in an anxiety-provoking speech task.
- The reduction of anxiety was significant, with a p-value of 0.056 relative to the protocol specified p-value of 0.10. Based on these findings, which "suggest the therapeutic potential of glutamate modulation in the treatment of anxiety disorder," Biohaven plans to expand its glutamate modulating program to include the treatment of generalized anxiety disorder.
- Biohaven's target indication for BHV-0223 is ALS, and the company plans to submit a New Drug Application (NDA) for the Food and Drug Administration in the third quarter of this year.
Dive Insight:
Following a stock value slide of nearly 30% on weak migraine data for rimegepant, the market is showing support for Biohaven once more.
Positive data for BHV-0223 in anxiety helped boost shares around 6% by market's close Thursday.
There are major unmet needs in anxiety, with current options — including antidepressants, benzodiazepines and beta-adrenergic blockers — having side effects such as sedation, cognitive impairment and potential for addiction.
Biohaven CEO Vlad Coric believes that glutamate modulating agents will combine anxiolytic properties and a favorable safety profile to provide "a novel treatment option." This supports the company pushing forward its glutamate platform should its migraine drug not live up to earlier expectations
Besides BHV-0223, Biohaven's glutamate platform includes trigriluzole, a prodrug of riluzole in a pivotal Phase 2/3 trial for obsessive-compulsive disorder (OCD), with enrollment due to complete this year.
A long-term extension study of trigriluzole in spinocerebellar ataxia will report topline results later this year and the company plans to start a second trial by the end of 2018 as well. In July 2018, a 48-week Phase 2/3 trial of trigriluzole began in mild-to-moderate Alzheimer's disease.
Rimegepant is Biohaven's lead drug, and despite its wobble, the company plans to submit an NDA in 2019 for acute treatment of migraine. Initial data from a long-term safety study is expected in the fourth quarter this year, along with topline results from a sublingual oral dissolving tablet formulation.
Biohaven also has a follow-up drug, BHV-3500, in preclinical trials for the acute treatment and prevention of migraine, with a Phase 1 trial planned before the end of the year.
The company's development programs are funded until the fourth quarter of 2019 by a $150 million royalty funding and stock purchase with Royalty Pharma. Biohaven, perhaps recognizing its migraine approach may not have the strength it hoped, has restructured a license agreement with Bristol-Myers Squibb to reduce royalties payable for its CGRP receptor antagonist platform, which includes rimegepant and BHV-3500.
Paid for by a private placement with institutional investors, Biohaven has made an upfront payment of $50 million to Bristol-Myers in return for a low single-digit reduction on the rimegepant royalties and a mid-single-digit reduction on the BHV-3500 royalties.
Further results from the BHV-0223 study will be presented at scientific meetings over the next year.