Biohaven Ltd. has announced positive topline results from a pivotal study of troriluzole in the treatment of Spinocerebellar Ataxia (SCA). The study, BHV4157-206-RWE, demonstrated the efficacy of troriluzole on the mean change from baseline in the modified functional scale for the assessment and rating of ataxia (F-SARA) after 3 years of treatment.
Key findings from the study include: Troriluzole 200 mg dosed orally, once daily, in patients with SCA met the study’s primary endpoint on the change from baseline in the F-SARA at 3 years in all study population genotypes. Troriluzole also showed statistically significant superiority after both 1 and 2 years of treatment. SCA patients treated with troriluzole showed a 50-70% slowing of disease progression, representing 1.5-2.2 years delay in disease progression over the 3-year study period. Troriluzole achieved statistically significant superiority on 9 consecutive, prespecified primary and secondary endpoints.
Dr. Susan Perlman, Director of Ataxia Clinic and Neurogenetics Clinical Trials at the David Geffen School of Medicine at UCLA, remarked that troriluzole is the very first treatment to show a delay in disease progression and can give patients additional years of independence, where they can walk without assistance, continue to work, play with their children, and participate in daily activities.
The study BHV4157-206-RWE was designed in discussion with the US Food and Drug Administration (FDA) and utilized phase 3 data and an external control of matched, untreated SCA subjects from the US Clinical Research Consortium for the Study of Cerebellar Ataxia (CRC-SCA) in accordance with the FDA’s guidance on real-world evidence (RWE) of effectiveness.
Furthermore, the study demonstrated statistically significant and sustained benefits at years 1, 2, and 3 on the F-SARA compared to a rigorously matched natural history control. The addition of European SCA data increased the external control sample size and added to the robustness of the statistically significant treatment differences at years 1, 2, and 3, favoring troriluzole.
Based on the topline data from the study, Biohaven plans to submit a new drug application (NDA) to the FDA in Q4 2024. If approved, Biohaven will be prepared to commercialize the treatment in the US in 2025.
Troriluzole is a new chemical entity that modulates glutamate, the most abundant excitatory neurotransmitter in the human body, and has the potential to be developed in a number of other diseases associated with excessive glutamate.
Biohaven is a biopharmaceutical company focused on the discovery, development, and commercialization of life-changing treatments in key therapeutic areas, including immunology, neuroscience, and oncology. Today the company's shares have moved 15.0% to a price of $46.44. For more information, read the company's full 8-K submission here.
