Biohaven Pharmaceuticals announced positive topline results from its proof of concept study of troriluzole in the treatment of obsessive-compulsive disorder (OCD). Troriluzole showed a consistent and clinically meaningful drug effect at all study timepoints in patients who had an inadequate response to existing standard of care treatments.
Troriluzole 200 mg administered once daily as adjunctive therapy in OCD patients with inadequate response to standard of care treatment showed consistent numerical improvement over placebo on the Yale-Brown Obsessive Compulsive Scale (Y-BOCS) at all study timepoints (weeks 4 to 12) but did not meet the primary outcome measure at week 12. Troriluzole treated subjects had a mean Y-BOCS improvement of -5.1 points from baseline versus -3.6 for placebo-treated at week 8, and -5.9 points versus -4.9 for placebo subjects at week 12. Troriluzole was well tolerated with a safety profile consistent with past clinical trial experience.
Based upon these results, Biohaven is planning to request an End of Phase 2 meeting with the FDA and plans to initiate an appropriately powered pivotal Phase 3 study of two doses of troriluzole versus placebo.
Troriluzole is a third-generation prodrug and new chemical entity that modulates glutamate, the most abundant excitatory neurotransmitter in the human body. The primary mode of action of troriluzole is normalization of synaptic levels of glutamate. Troriluzole increases glutamate uptake from the peri-synaptic space, by augmenting the expression and function of excitatory amino acid transporters (i.e., EAAT2 also called GLT-1) located on glial cells that play a key role in clearing glutamate from the synapse.
Obsessive-compulsive disorder is a serious psychiatric condition affecting over 2 million individuals in the U.S. and significantly impacts quality of life. Approximately 40% to 60% of OCD patients continue to experience significant residual symptoms despite approved therapies. Some refractory patients undergo psychosurgery (cingulotomy or deep brain stimulation) to alleviate their crippling symptoms.