Biohaven Reports Positive Early Clinical Trial Results for Anxiety Drug

Biohaven Pharma, headquartered in New Haven, Conn., announced positive data from a proof-of-concept trial for its BHV-0223 in social anxiety and public speaking anxiety disorders.

The trial was conducted at Yale University School Of Medicine. It looked at 21 patients who had been diagnosed with social anxiety disorder. They participated in an anxiety-provoking speech task in a double-blind, crossover trial. In the trial, BHV-0223 reduced anxiety by 8.3 points compared to placebo based on the 100-point Visual Analogue Scale (VAS). On a likelihood-based analysis, it had a 14.4-point advantage compared to placebo. The drug was safe and well-tolerated.

This shows promise, although the p-value of 0.056 isn’t statistically significant by standard measures, but the company is indicating it was positive “relative to the protocol specified level of p = 0.10.” It’s a very small study, so the company plans to expand into a larger group.

“The findings from this anxiety-provoking challenge study in patients with social anxiety disorder demonstrated anti-anxiety effects of BHV-0223 in a public speaking scenario and suggest the therapeutic potential of glutamate modulation in the treatment of anxiety disorders,” said Michael Bloch, principal investigator and an associate professor at Yale University School of Medicine, in a statement.

BHV-0223 is a sublingual—administered under the tongue—formulation of riluzole. It uses the Zydis orally dissolving tablet technology. Biohaven has exclusive intellectual property rights related to the use of riluzole for specific neuropsychiatric disorders licensed from Yale University. It also has worldwide exclusive rights to the use of Zydis in riluzole products from Catalent.

Another of Biohaven’s compounds using its glutamate modulating platform is BHV-4157, or troriluzole. Troriluzole is presently being studied in Phase II/III clinical trials in spinocerebellar ataxia (SCA), obsessive-compulsive disorder (OCD) and Alzheimer’s disease (AD). Its BHV-5000, an NMDA antagonist, which was in-licensed from AstraZeneca, is being studied in development programs for Rett syndrome, treatment-resistant depression and chronic pain disorders.

The company plans to file a marketing application with the U.S. Food and Drug Administration (FDA) this quarter for BHV-0223 for the treatment of ALS.

On August 1, the company announced it had enrolled its first patient in a Phase II/III clinical trial of trigriluzole in mild-to-moderate Alzheimer’s disease. It is being conducted in collaboration with the Alzheimer’s Disease Cooperative Study (ADCS) throughout the U.S.”

“We are excited that the first patients have been enrolled into this innovative clinical trial that we are conducting with Biohaven,” said Howard Feldman, director of the ADCS and professor of Neurosciences at University of California San Diego School of Medicine, in a statement. “The preclinical evidence for the active metabolite of trigriluzole to modulate glutamate and confer neuroprotective effects in patients with AD is compelling, and the new formulation of trigriluzole should improve its pharmaceutical properties with potential for efficacy in AD.”

This social anxiety disorder in the current clinical trial qualifies as a DSM-5 condition. As it expands its program, it will include generalized anxiety disorder, as well. Vlad Coric, Biohaven’s chief executive officer, stated, “Generalized anxiety disorder (GAD) affects approximately 6.8 million adults, or 3 percent, of the U.S. population. Although a common disorder, current medication treatments are not fully effective in half of patients. GAD is characterized by excessive anxiety, uncontrollable worry, restlessness, fatigue, difficulty concentrating, irritability, muscle tension and sleep disturbances. Current medication treatments, including antidepressants, benzodiazepines and beta-adrenergic blockers, can have side effects such as sedation and often are of limited effectiveness.”