Abstract Details

Title Results from the Long-Term Open Label Extension Phase Analyses of BHV4157-201: A Phase IIb/III, Randomized, Double-blind, Placebo-controlled Trial of the Safety and Efficacy of Troriluzole in Adult Subjects with Spinocerebellar Ataxia

Topic Movement Disorders

Presentation(s) P1 - Poster Session 1 (5:30 PM-6:30 PM)

Poster/Presentation
Number 10-001

Objective Provide a preliminary assessment of the long term benefit of treatment with troriluzole (also known as BHV-4157) among patients with Spinocerebellar Ataxias (SCA).

Background Hereditary Spinocerebellar Ataxias (SCA) are progressive neurodegenerative disorders that are characterized clinically by progressive ataxia and are attributed to various autosomal dominant genetic mutations. Currently, there are no FDA-approved medications for this debilitating disorder and treatment remains supportive.

Design/Methods The study was comprised of two periods: an 8-week randomization phase, followed by a 96-week open-label extension period. Subjects were male and female outpatients between the ages of 18-75 years, inclusive, with a known or suspected diagnosis of the following hereditary ataxias: SCA1, SCA2, SCA3, SCA6, SCA7, SCA8 and SCA10. The current analysis focused on the assessment of change from baseline to a modified version of the Scale for the Assessment and Rating of Ataxia (SARA). These were deemed to be more sensitive to both disease progression and treatment effect than the total SARA (axial + appendicular items). The analyses describe the extension phase study results for the modified SARA at weeks 8 & 24.

Results In the ongoing, 96-week extension phase of BHV4157-201, a majority (99%) of the randomization phase patients participated (N=131). Among all patients, change from randomization baseline on the modified SARA (mean change±SE) was -0.40±0.13 at week 8, and -0.30±0.13 at week 24. These changes contrast to an increase in modified SARA of +0.39±0.11 from a natural history cohort at 24 weeks of follow up.

Conclusions The observed changes provide consistent sustained improvements in modified SARA scores in the overall population. Contrasting to the natural history cohort, these observed changes with treatment suggest a potential therapeutic benefit of troriluzole.

Authors/Disclosures
Melissa Beiner PRESENTER	Melissa Beiner has received personal compensation for serving as an employee of Biohaven Pharmaceuticals. Melissa Beiner has stock in Biohaven Pharmaceuticals.
Victoria Wirtz	Victoria Wirtz has received personal compensation for serving as an employee of Biohaven Pharmaceuticals, Inc. Victoria Wirtz has stock in Biohaven.
Vladimir Coric	Vladimir Coric has received personal compensation for serving as an employee of Biohaven. Vladimir Coric has received personal compensation in the range of $1,000,000+ for serving as an officer or member of the Board of Directors for Bioahven. Vladimir Coric has stock in Biohaven. Vladimir Coric has received intellectual property interests from a discovery or technology relating to health care.
Robert Berman	Robert Berman has received personal compensation for serving as an employee of Biohaven. Robert Berman has received personal compensation in the range of $100,000-$499,999 for serving as an officer or member of the Board of Directors for Biohaven. Robert Berman has received stock or an ownership interest from Biohaven.
Gilbert J. L'Italien	Gilbert J. L'Italien has received personal compensation for serving as an employee of Biohaven Pharmaceuticals. Gilbert J. L'Italien has stock in biohaven pharmaceuticals.

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