Biohaven Reports Positive Results in Key Troriluzole Study for SCA

Biohaven Ltd. recently revealed promising topline results from its pivotal Study BHV4157-206-RWE (NCT06529146). The study demonstrated the efficacy of troriluzole in treating spinocerebellar ataxia (SCA), a rare and progressively debilitating neurodegenerative disease. The study, which spanned three years, successfully met its primary endpoint by showing significant improvements in the f-SARA (functional Scale for the Assessment and Rating of Ataxia) scores at years one and two, with continued benefits observed into the third year.

SCA affects around 15,000 individuals in the United States and 24,000 in Europe and the United Kingdom. Despite the severe impact of this disease, there are currently no FDA-approved treatments. The data from multiple analyses within this study illustrated a clinically meaningful slowdown in disease progression for SCA patients treated with troriluzole. These benefits translated into a 50-70% slower rate of decline compared to untreated individuals, equating to a delay of 1.5-2.2 years in disease progression over the three-year period.

Additionally, a responder sensitivity analysis revealed that patients who experienced a worsening of 2 points or more on the f-SARA after three years had an odds ratio of 4.1 for those in the untreated control group compared to those treated with troriluzole, emphasizing the drug's effectiveness.

Dr. Susan Perlman from UCLA highlighted the significance of these findings, noting that troriluzole could potentially provide SCA patients with additional years of independence, allowing them to maintain a higher quality of life.

The design of Study BHV4157-206-RWE was developed in collaboration with the FDA to evaluate the long-term effectiveness of troriluzole in SCA patients. The study utilized both Phase 3 data and an external control group of untreated SCA patients sourced from the US Clinical Research Consortium for SCA. All study endpoints were pre-specified, and the protocol and statistical analysis plan received FDA approval before the topline data analysis commenced. The study included 63 subjects who completed three years of treatment, with rigorous matching to the external control using Propensity Score Matching (PSM).

The primary goal was to assess the treatment effects of troriluzole over three years. The results showed statistically significant and sustained benefits in f-SARA scores for the treated group compared to the natural history control. Additional analyses included another independent natural history control group from the European SCA study (EUROSCA). The pooled analyses, incorporating both CRC-SCA and EUROSCA data, reinforced the significant treatment differences favoring troriluzole at all time points.

Dr. Jeremy Schmahmann from Harvard Medical School underscored the impact of these findings, reflecting on how they could change the lives of patients and families who have suffered from SCA for generations. The data suggest that troriluzole could be an effective daily treatment option, offering a meaningful intervention in slowing the disease's progression.

SCA, characterized by progressive loss of motor control and cerebellar atrophy, leads to severe morbidity, including speech and swallowing difficulties, gait impairment, and premature death. Symptoms typically manifest in early adulthood and worsen over time. Given the lack of current FDA-approved treatments, this breakthrough represents a significant milestone.

Dr. Vlad Coric, CEO of Biohaven, emphasized the importance of collaboration in advancing treatments for rare diseases. The successful results from Study BHV4157-206-RWE, conducted with FDA guidance, pave the way for a New Drug Application (NDA) submission to the FDA in the fourth quarter of 2024. If approved, troriluzole could be commercialized in the US by 2025, offering hope to SCA patients.

Andrew Rosen, CEO of the National Ataxia Foundation, praised Biohaven's efforts and commitment to developing therapies for ataxia through collaborative research initiatives. The positive topline results of this study mark a culmination of years of dedication towards finding a treatment for SCA.

Based on these findings, Biohaven intends to move forward with the NDA submission, aiming for priority FDA review given its Fast-Track and Orphan Drug Designations. The company is poised to bring troriluzole to the market and make a significant impact on the lives of SCA patients.