Troriluzole for spinocerebellar ataxia

Long-term results of the BHV4157-201 trial among patients with spinocerebellar ataxia (SCA) suggest an attenuation of disease progression in patients treated with troriluzole for up to 3 years. This analysis focused on the continued assessment of change from baseline to 96 weeks in the total score on the Scale for the Assessment and Rating of Ataxia (SARA) [1].

The phase 2/3 BHV4157-201 study (NCT02960893) comprised an 8-week randomisation phase, followed by a 48-week open-label phase. Encouraging data at 1 year were a reason to add another 48 weeks of open-label treatment. Enrolled were 136 patients with hereditary SCAs; mean age was 53 years; 53% were female; baseline SARA score was 14.4. Patients in BHV4157-201 were treated with 140 mg of troriluzole administered daily for 1 year. SARA scores after 96 weeks of open-label treatment were compared with the expected score in untreated patients, which is an increase of 1-2 points per year, based on cumulative historical data [2]. Dr Melissa W. Beiner (Biohaven Pharmaceuticals, CT, USA) presented the results.

Least squares mean change from baseline in total SARA score after 96 weeks was -0.34 (95% CI -0.94 to 0.26), a significant improvement compared with the cumulative historical data (P=0.0007). While the primary analysis suggests a therapeutic benefit from troriluzole in this patient population, these results need to be confirmed in a prospective placebo-controlled trial. Dr Beiner added that patients with a gap in troriluzole treatment (3 weeks to 12 months) had a ≥1 point worsening during off-treatment, increasing with longer duration off troriluzole. “When patients experience a gap in troriluzole dosing, the decline in total SARA scores was in accordance with the duration of the gap. When troriluzole was then resumed, patients experienced an improvement in total SARA scores similar to the 48-week open-label observation.”

1. Beiner M, et al. Analysis of 96 Week, Long-Term Open Label Extension Phase of Study 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. S3.002, AAN 2021 Virtual Congress, 17-22 April.
2. Diallo A, et al. J Neurol. 2020 Apr 7. doi: 10.1007/s00415-020-09815-2.

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Posted on 16 June 202116 June 2021