New Haven, Connecticut (NYSE: BHVN) May 30, 2017 – Biohaven Pharmaceutical Holding Company Ltd. (Biohaven) announced today that the company has completed enrollment in its clinical study of trigriluzole (BHV-4157) in patients with spinocerebellar ataxia (SCA). Trigriluzole, a novel drug candidate being developed by Biohaven, has received both Orphan Drug Designation and Fast Track Designation from the U.S. Food and Drug Administration (FDA) as a potential treatment for SCA. SCA is a rare, debilitating neurodegenerative disorder that is estimated to affect approximately 22,000 people in the United States. No medications are currently approved for this often severe condition.
“The completion of enrollment in this trial represents an important milestone for our glutamate modulating program in neurologic illnesses. We would like to thank the patients with SCA who are participating in this trial, the National Ataxia Foundation, and the clinical research sites for helping us to meet our enrollment target in such a timely fashion,” said Vlad Coric, M.D., Chief Executive Officer at Biohaven. “We are acutely aware of the high unmet medical need in SCA, and if the results of the trial are positive, we expect to submit a new drug application to the FDA in early 2018.”
About the Trigriluzole Trial in SCA
Biohaven’s SCA trial is a randomized, double-blind, placebo controlled, multi-center study designed to compare the safety and efficacy of once-daily oral therapy with trigriluzole 140 mg versus placebo. The study has now fully enrolled with approximately 180 adult SCA patients and is being conducted at 18 centers in the United States.
The primary efficacy endpoint of the trial is the change from baseline on the Scale for the Assessment and Rating of Ataxia (SARA) after eight weeks of treatment with trigriluzole. The SARA is an eight item clinician-administered instrument that measures severity of ataxia symptoms. After completion of the initial efficacy phase, subjects may be followed for 48 weeks on an open-label basis, during which time the subjects are eligible to continue to receive daily treatment with trigriluzole. Secondary efficacy endpoints include an 8-meter timed walk test and Sheehan Disability Scale, as well as the Patient and Clinician Global Impression of Change scales. Safety and tolerability are being assessed by treatment-emergent adverse event observations, vital sign assessments and laboratory tests.
“The completion of recruitment in our SCA trial reflects the culmination of our efforts with the leading academic and clinical sites in the United States that treat patients with SCA. Their high levels of expertise and engagement were essential in helping us fully enroll the trial so successfully,” said Robert Berman, M.D., Chief Medical Officer at Biohaven. “Our clinical operations team and trial sites are now focused on completing the eight-week treatment period and ultimately preparing for top-line data.”
Trigriluzole is a novel tripeptide prodrug being developed by Biohaven and represents more than six years of chemistry development and research into over 300 drug candidates. The company previously announced that trigriluzole has received the FDA’s Orphan Drug and Fast Track Designations for the treatment of patients with SCA. Trigriluzole is the most advanced drug candidate from Biohaven’s glutamate modulator platform.
About Orphan Drug Designation
The FDA, through its Office of Orphan Products Development (OOPD), grants orphan status to drugs and biologic products that are intended for the treatment, diagnosis, or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States. Orphan drug designation provides a drug developer with certain benefits and incentives, including a potential period of marketing exclusivity if regulatory approval is ultimately received for the designated indication.
About Fast Track Designation
As one of its Expedited Programs for Serious Conditions, the FDA grants Fast Track Designation to “facilitate the development and expedite the review of drugs to treat serious conditions and fill an unmet medical need” in order to make important new drugs available to patients earlier.
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, ALS Biopharma LLC and Massachusetts General Hospital. Currently, Biohaven’s lead development programs include multiple compounds across its CGRP receptor antagonist and glutamate modulator 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.
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 regarding the potential results of the ongoing clinical trial of trigriluzole, the submission of a new drug application to the FDA for trigriluzole and the potential timing thereof and the potential effects of Fast Track Designation and Orphan Drug Designation, as well as the Company’s plans and objectives, expectations and assumptions of management are forward-looking statements. The use of certain words, including the words “estimate,” “intend,” “expect,” “believe,” “anticipate,” “will, “potential,” “plan,” “could,” “may” and similar expressions are intended to identify forward-looking statements. The Company may not actually achieve the plans, intentions or expectations 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 the fact that: completion of enrollment in the SCA trial of trigriluzole does not guarantee retention of the enrolled population to the efficacy assessment point or a successful outcome of the trial; submission of an NDA does not guarantee that the FDA will accept the regulatory package for review or that it would find the primary outcome measure used in this trial, the SARA, acceptable for approval; and the receipt of Fast Track Designation does not guarantee that trigriluzole will experience a faster development process. Additional important factors to be considered in connection with forward-looking statements are described in the “Risk Factors” section of the Company’s final prospectus dated May 3, 2017, filed with the Securities and Exchange Commission on May 5, 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.
Dr. Vlad Coric
Chief Executive Officer
Biohaven Pharmaceutical Holding Company Ltd.
Phone: (203) 404-0410