Biohaven Begins Phase 3 Troriluzole Trial for Obsessive-Compulsive Disorder

Biohaven has commenced enrollment in a Phase 3 clinical trial assessing the efficacy and safety of troriluzole in patients with obsessive-compulsive disorder (OCD). Biohaven is advancing the 280 mg once daily dose of troriluzole into two double-blind, placebo-controlled Phase 3 clinical trials with identical study designs and plans to enroll approximately 600 patients in each of these adjunctive treatment trials across study sites in both the United States and Europe. A prior proof of concept study with adjunctive troriluzole in patients with OCD showed a clinically meaningful effect at all study timepoints in patients who had an inadequate response to existing standard of care treatment. Although the study sample size in Phase 2 did not demonstrate statistical significance, the data from the previous trial was instrumental in refining and powering the Phase 3 studies.

"We look forward to advancing our Phase 3 clinical trials of troriluzole 280 mg for the adjunctive treatment of OCD. If approved, this would provide hope to many who suffer with OCD and for whom standard of care medications do not provide adequate relief. No new treatments for OCD have been approved for over two decades. With its novel mechanism of action compared to currently available therapies, troriluzole would provide a significant advance in treatment for the many patients who either do not respond or continue to experience residual symptoms with standard medications," Elyse Stock, M.D., Chief Medical Officer said.

Troriluzole is a new chemical entity and third-generation glutamate modulating agent that normalizes glutamate, a key neurotransmitter implicated in obsessive-compulsive disorder. Biohaven was awarded two U.S. Patents covering troriluzole, with international patents pending, all having statutory expiration dates in 2039. The primary mechanism of action of troriluzole is enhancing synaptic glutamate cycling by augmenting the expression and function of excitatory amino acid transporters (i.e., EAAT1-2) located on glial cells that play a key role in clearing glutamate from the synapse. Glutamatergic dysfunction is implicated in the pathophysiology of a broad range of disorders including OCD, Amyotrophic Lateral Sclerosis, Spinocerebellar Ataxia, Alzheimer's Disease, depression, chronic pain, and a variety of cancers. The therapeutic potential of troriluzole is supported by clinical and translational research studies.

OCD is a serious psychiatric condition affecting over 2 million individuals in the U.S. and significantly impacts quality of life. It is a chronic and long-lasting disorder in which a person has uncontrollable, reoccurring thoughts (obsessions) and behaviors (compulsions). The current standards of care include behavioral therapy and selective serotonin reuptake inhibitors. It is estimated that 20-30% of OCD sufferers derive minimal benefit from current treatment options and continue to experience significant residual symptoms despite currently approved therapies.