Biohaven to begin clinical development of BHV-5000 to treat Rett syndrome

Clinical Trials

Biohaven Pharmaceutical Holding Company Ltd. and its wholly owned subsidiary, Biohaven Pharmaceuticals, Inc., today announced that the U.S. FDA has notified the Company that it may proceed with its clinical investigation of BHV-5000. 

Enrollment of subjects has started for the IND opening study, which will test single and multiple doses of a solid-dose formulation of BHV-5000 in healthy volunteers.  BHV-5000 is being developed as a potential treatment for symptoms associated with Rett syndrome, including breathing irregularities.  Biohaven also plans to explore development of BHV-5000 in other potential future indications including neuropathic pain and treatment-resistant depression.

BHV-5000 is a low trapping, potent N-methyl-D-aspartate (NMDA) receptor antagonist licensed to Biohaven from AstraZeneca.  BHV-5000 was previously advanced into a Phase 1 clinical trial, as a drug in solution formulation, and the active metabolite of BHV-5000, lanicemine, was previously advanced through Phase 2 trials.  Unlike other potent NMDA receptor targeting compounds, BHV-5000 is a NMDA antagonist that has not been associated with the prominent psychotomimetic effects. In addition, BHV-5000 is orally bioavailable.

“BHV-5000 is a novel glutamate receptor antagonist that we are planning to evaluate for efficacy in neurologic and neuropsychiatric indications, including Rett syndrome,” said Vlad Coric, M.D., CEO of Biohaven. “Currently there are no approved drugs for this severe neurodevelopmental disorder. Advancement of the clinical investigation of BHV-5000 supports Biohaven’s global development strategy of providing improved therapies for patients suffering from some of the most disabling neurologic disorders.”

“This IND allows Biohaven to study the first solid-dose formulation of BHV-5000 in healthy volunteers,” said Robert Berman, M.D., Chief Medical Officer of Biohaven.  “Based on protocol feedback from the FDA, Biohaven will initially assess BHV-5000 in single and multiple doses to characterize its pharmacokinetic and safety profile.  Subsequently, Biohaven plans to advance BHV-5000 into clinical trials in patient populations. We are eager to advance BHV-5000 in clinical testing to understand if preclinical effects translate into therapeutic benefits for patients.”

About Biohaven
Biohaven is a clinical-stage biopharmaceutical company with a portfolio of innovative, late-stage product candidates targeting neurological diseases, including rare disorders. Biohaven has combined internal development and research with intellectual property licensed from companies and institutions including Bristol-Myers Squibb Company, AstraZeneca AB, Yale University, Catalent, Rutgers, ALS Biopharma LLC and Massachusetts General Hospital. Currently, Biohaven’s lead development programs include multiple compounds across its CGRP receptor antagonist and glutamate modulation platforms. The Company’s common shares are listed on the New York Stock Exchange and traded under the ticker symbol BHVN. More information about Biohaven is available at

For further information,
Contact Dr. Vlad Coric, the Chief Executive
Officer at This email address is being protected from spambots. You need JavaScript enabled to view it.

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