Press release August 7, 2017
Biohaven Pharmaceutical Holding Company Ltd. 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. 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.