May 30, 2017
"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.