Examine the treatment benefits of troriluzole over 3 years in patients with spinocerebellar ataxia (SCA) with a time to progression to wheelchair analysis in troriluzole-treated subjects vs untreated external controls.

SCA is an autosomal-dominant neurodegenerative disease predominantly characterized by atrophy of the cerebellum, brainstem, and spinal cord. Clinically, SCA is characterized by progressive ataxia, severe disability, and premature death.

Data on troriluzole treatment was obtained from BHV4157-206 (NCT03701399), a 48-week double blinded study with a long-term open-label extension and compared to a combined cohort from the US SCA natural history study (CRC-SCA) and the European natural history study (EUROSCA). Data were censored at 3 years. Participants who were ambulatory based on the f-SARA gait item (≤3) at the start of follow-up were included. Time to gait=4 (wheelchair use) was analyzed using Cox proportional hazards model adjusting for baseline gait item, sex, race, age group (<40 years; ≥40 years), and time since onset of symptoms (<8 years; ≥8 years).

Comparison of 197 troriluzole-treated subjects and 703 CRC-SCA/EEUROSCA participants showed a delay in time to progression to wheelchair in the troriluzole-treated SCA patients as compared to untreated patients. The risk of progression to wheelchair was more than 4 times greater in untreated compared to troriluzole-treated patients; adjusted hazard ratio = 4.4, 95% CI (1.90, 10.14); p=0.0005.

This analysis demonstrates that troriluzole treatment affords SCA patients a longer period of time to remain ambulatory and highlights troriluzole’s role in slowing disease progression. Further, this reflects a clinically relevant benefit, allowing patients to maintain functionality and relative independence for a longer period of time.