Biohaven Announces Initiation Of Clinical Development For BHV-5000, A Novel Low-Trapping NMDA Antagonist

NEW HAVEN, Conn., Jan. 18, 2018 /PRNewswire/ -- Biohaven Pharmaceutical Holding Company Ltd. (Biohaven or the "Company") and its wholly owned subsidiary, Biohaven Pharmaceuticals, Inc., today announced that the U.S. Food and Drug Administration (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 www.biohavenpharma.com.

Forward-Looking Statements

This news release includes forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. These forward-looking statements involve substantial risks and uncertainties, including statements that are based on the current expectations and assumptions of the Company's management. All statements, other than statements of historical facts, included in this press release, including the Company's plans for the clinical development of BHV-5000, the expected lack of prominent psychotomimetic effects of BHV-5000 and the potential to expand clinical development of BHV-5000 into other indications, as well as the size of the potential market for BHV-5000 in Rett syndrome, are forward-looking statements. The use of certain words, including the "believe", "could", "expect" and "will" and similar expressions are intended to identify forward-looking statements. The Company may not actually achieve the plans and objectives disclosed in the forward-looking statements and you should not place undue reliance on the Company's forward-looking statements. Various important factors could cause actual results or events to differ materially from those that may be expressed or implied by our forward-looking statements, including uncertainties relating to the future clinical success of BHV-5000, and whether the results observed in previous clinical trials of BHV-5000 and lanicemine will be observed in Biohaven's future clinical trials of BHV-5000. Additional important factors to be considered in connection with forward-looking statements are described in the "Risk Factors" section of the Company's Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission on November 14, 2017. The forward-looking statements are made as of this date and the Company does not undertake any obligation to update any forward-looking statements, whether as a result of new information, future events or otherwise, except as required by law.

For further information, contact Dr. Vlad Coric, the Chief Executive

Officer at [email protected]

SOURCE Biohaven Pharmaceutical Holding Company Ltd.