Biohaven Pharma Reports Positive Topline Results in Pivotal Study of Troriluzole for Spinocerebellar Ataxia

Biohaven Ltd. (BHVN) has announced promising topline results from its pivotal Study BHV4157-206-RWE, which highlights the efficacy of troriluzole in reducing symptoms of spinocerebellar ataxia (SCA) after three years of treatment. The study met its primary endpoint, demonstrating statistically significant improvements on the functional Scale for the Assessment and Rating of Ataxia (f-SARA) at both one and two years.

SCA is a rare and progressively debilitating neurodegenerative disease that impacts approximately 15,000 individuals in the United States and around 24,000 in Europe and the United Kingdom. Currently, there are no FDA-approved treatments for this condition.

The data indicate a meaningful and clinically relevant slowing of disease progression in SCA patients treated with troriluzole, translating to a 50-70% reduction in the rate of decline compared to those who are untreated. This represents a delay of 1.5 to 2.2 years in disease progression over the three-year period. Furthermore, in a responder analysis, a worsening of two points or more on the f-SARA at the three-year mark showed an odds ratio of 4.1 for untreated subjects compared to those receiving troriluzole.

Dr. Susan Perlman, Director of the Ataxia Clinic at the David Geffen School of Medicine, emphasized the significance of these findings: “Troriluzole is the first treatment that has shown a delay in disease progression, ultimately allowing patients to maintain a greater level of independence and quality of life.”

The study was conducted with guidance from the FDA to evaluate the effectiveness of troriluzole after a three-year treatment period. It utilized Phase 3 data alongside an external control group of matched, untreated SCA patients, aligning with FDA recommendations for real-world evidence. All endpoints were predefined, and the study protocol was thoroughly reviewed by the FDA prior to the results being analyzed. The final analysis doubled the previous data from the initial trial and involved 63 participants completing three years of treatment, matched with external controls using rigorous statistical methods.

In addition, the study incorporated another independent natural history control group from a European SCA study to further bolster the findings. Results from this group confirmed the primary efficacy analysis, showing consistent and statistically significant results at all time points.

Dr. Jeremy Schmahmann from Harvard Medical School remarked on the implications of the topline results: “The stabilization of SCA symptoms observed at three years, along with the reported reductions in falls, underscores the therapeutic potential of troriluzole.”

Spinocerebellar ataxia, a group of dominantly inherited disorders, is characterized by the gradual loss of voluntary motor control and atrophy of the cerebellum. Symptoms can occur from childhood to late adulthood, but typically begin in early adulthood, leading to severe morbidity and eventual decline. There are currently no FDA-approved treatments or cures for SCA.

Vlad Coric, CEO of Biohaven, stated, “The journey to develop new therapies for rare conditions requires extensive collaboration across various stakeholders. Our team is committed to following the scientific approach, and we are thrilled to present these positive results from Study BHV4157-206-RWE.”

Andrew Rosen, CEO of the National Ataxia Foundation, expressed the significance of these findings for patients and families affected by SCA: “These topline results represent the culmination of years of dedication to finding a treatment that can slow the progression of this devastating disorder.”

Based on the topline data and previous efficacy findings, Biohaven plans to submit a New Drug Application to the FDA in the fourth quarter of 2024. The development program for troriluzole has generated one of the largest clinical trial datasets in SCA, with patient follow-up extending beyond five years. Biohaven has previously received Fast-Track and Orphan Drug designations for this treatment in SCA, positioning them for a potentially expedited review process.

Biohaven is set to hold a live conference call to discuss these results and outline next steps for advancing this promising treatment for patients with SCA.