Utilizing the SCACOMS Composite Measure to Assess Treatment Benefit of Troriluzole in Spinocerebellar Ataxia: Irfan Qureshi, MD

WATCH TIME: 4 minutes

"Together, this composite forms a sensitive measure to look at disease progression of SCA, and allows us to look at it over the duration of a clinical trial."

Spinocerebellar ataxia (SCA) is a rare inherited neurodegenerative disorder characterized by progressive ataxia affecting limb coordination, balance, and speech. Because of the wide range of symptoms of patients face, understanding true efficacy of investigational therapies has been a challenge.

At the 2024 American Academy of Neurology (AAN) Annual Meeting, held April 13-18, in Denver, Colorado, Biohaven presented several posters regarding its investigational agent troriluzole in patients with SCA and the development of the Spinocerebellar Ataxia Composite Scales (SCACOMS). Using 2 natural history dataset, SCACOMS were derived from Clinical Global Impression of Change, Friedreich’s Ataxia Rating Scale – Functional Scale (FARS-FUNC), and the Modified Functional Scale for the Assessment and Rating of Ataxia (f-SARA). A partial least squares regression model was used to objectively select the optimal combination and weighting of items for measuring progression over time.

SCACOMS was applied to BHV4157-206, a 48-week, phase 3 study (NCT03701399) assessing troriluzole (Biohaven), an investigational prodrug formulation of riluzole, in patients with SCA. A total of 96 troriluzole-treated subjects and 611 untreated natural history subjects informed the SCA genotype analysis. Using the SCACOMS, investigators observed a statistically significant treatment effect favoring troriluzole (least-square mean difference, 3.60; P = .0030) and Cohen’s d of 0.59.

Irfan Qureshi, MD, chief medical officer at Biohaven, believes this tool will allow for the development of future studies to detect small but meaningful clinical impact of disease-modifying treatments. At AAN 2024, he sat down with NeurologyLive® to share insight on the origins of SCACOMS, how it was applied to the study, and how troriluzole outperformed natural history comparisons.

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REFERENCE
1. L’Italien G, Popoff E, Rogula B, et al. Development of a novel composite measure (SCACOMS) to assess disease progression in spinocerebellar ataxia. Presented at: 2024 AAN Annual Meeting; April 13-18; Denver, CO. ABSTRACT 003476