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AAN 2021 | Phase II/III trial of troriluzole in spinocerebellar ataxia

Melissa Beiner, MD, Biohaven Pharmaceuticals, Inc., New Haven, CT, discusses the findings of a Phase II/III clinical trial (NCT02960893) of troriluzole in patients with spinocerebellar ataxia. Following the demonstration of troriluzole’s ability to attenuate disease progression in a 48-week open-label study, a trial extension was launched with the aim of assessing the long-term benefit of troriluzole treatment. With 96 weeks of treatment, the mean change in total SARA score (Scale for the Assessment and Rating of Ataxia) was 0.3. Notably, scores were found to substantially worsen in patients waiting to join the study extension. The level of decline correlated with the off-drug duration. These declines improved following the re-initiation of therapy. A Phase III placebo-controlled trial (NCT03701399) to substantiate these findings is ongoing. This interview took place during the American Academy of Neurology (AAN) 2021 Annual Meeting.