NEW HAVEN, Conn., Aug. 7, 2017 /PRNewswire/ -- Biohaven Pharmaceutical Holding Company Ltd. (NYSE: BHVN) announced today that it has commenced dosing of all 141 randomized patients with spinocerebellar ataxia (SCA) in its Phase 2/3 trial of trigriluzole (previously known as BHV-4157). As a result, Biohaven now expects to receive topline results in the fourth quarter of 2017, earlier than previously expected. Trigriluzole, a novel glutamate modulator, is a drug candidate being developed by Biohaven as a potential treatment for patients with SCA, a rare, debilitating, genetic neurodegenerative disorder that affects approximately 22,000 people in the United States. No medications are currently approved by the U.S. Food and Drug Administration (FDA) for the treatment of this devastating condition or any other cerebellar ataxias. Trigriluzole has received both Orphan Drug Designation and Fast Track Designation for the treatment of SCA from the FDA.
"We are pleased to reach this important milestone in our efforts to advance trigriluzole for treatment of patients with SCA, and we are likewise happy to accelerate our expectation for receiving topline data to Q4 of this year rather than Q1 of next year," said Vlad Coric, M.D., Chief Executive Officer at Biohaven. "Patients with SCA are a highly underserved population who are currently suffering without approved medications, and our goal is to continue to move as rapidly as possible to try to bring this new treatment to market. We would like to thank and acknowledge all of those who are working with us, including the patients who enrolled in this trial, their families and caregivers, the National Ataxia Foundation, and the many academic clinicians, investigators and trial sites who have helped us to advance trigriluzole."
"The rapid progress of enrollment, randomization and dosing in this study reflects the high unmet medical needs and determination of the SCA community," said Robert Berman, M.D., Chief Medical Officer at Biohaven. "Our goal is to achieve a long-awaited breakthrough in the treatment of patients with SCA and we believe that if the topline data is positive, the Phase 2/3 trial may be sufficient to support our application for regulatory approval of trigriluzole."
Trigriluzole is a novel tripeptide prodrug being developed by Biohaven and represents more than six years of chemistry research and development into over 300 drug candidates. Trigriluzole is the most advanced drug candidate in Biohaven's glutamate modulation platform.
About the Trigriluzole Phase 2/3 Trial for the treatment of SCA
Biohaven's Phase 2/3 SCA trial is a randomized, double-blind, placebo controlled, multi-center trial designed to compare the safety and efficacy of once-daily oral therapy with trigriluzole 140 mg versus placebo. The study has now fully randomized with 141 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 in patient scores on the Scale for the Assessment and Rating of Ataxia (SARA) after eight weeks of treatment. The SARA is an eight-item clinician-administered semi-qualitative performance-based assessment of cerebellar ataxia symptoms that measures impairment on a scale of zero to 40, with a higher score indicating more severe ataxia. After completion of the eight-week treatment 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. Safety and tolerability are being assessed by treatment-emergent adverse event observations, vital sign assessments and laboratory tests. More details about the study can be found at the clinicaltrials.gov website under the study identifier NCT02960893.
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, 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. Biohaven'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, including statements regarding the Company's expectations for the timing and data from its Phase 2/3 clinical trial of trigriluzole, the potential benefits of trigriluzole for patients with SCA, the size of the potential market for trigriluzole and the Company's expectations regarding trigriluzole's path to regulatory approval, are forward-looking statements. The use of certain words, including the words "believe," "expect", "may" 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 the significant risks and uncertainties regarding the future clinical success and regulatory pathway of trigriluzole . 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 June 16, 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 Vlad.Coric@biohavenpharma.com
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