Biohaven Ltd. (BHVN) stock soared by 13.7% on September 23 after the company announced positive top-line data from its pivotal BHV4157-206-RWE study. This study evaluated the efficacy of its pipeline candidate, troriluzole, in treating spinocerebellar ataxia (SCA), a rare and debilitating neurodegenerative disease that currently lacks FDA-approved treatments.
The study met its primary endpoint, demonstrating a significant improvement in the modified functional Scale for the Assessment and Rating of Ataxia (f-SARA) after three years of treatment with troriluzole (200 mg) in SCA patients. Notably, treatment with troriluzole resulted in statistically significant improvements in the f-SARA at years one and two as well.
Multiple analyses revealed a robust and clinically meaningful slowing of disease progression in SCA patients treated with troriluzole. This translates to a 50-70% reduction in disease progression, representing a 1.5-2.2 year delay compared to untreated patients over a three-year period. The positive results extend beyond the primary endpoint, with statistically significant superiority observed across nine pre-specified primary and secondary endpoints of the study.
This makes troriluzole the first treatment to show a delay in disease progression for SCA patients, potentially offering a significant breakthrough for those affected by this debilitating condition.
Encouraged by the positive data, Biohaven plans to submit a New Drug Application (NDA) for troriluzole in SCA to the FDA in the fourth quarter of 2024. If approved, the company aims to launch troriluzole in the United States in 2025.
Further emphasizing the potential of troriluzole, the FDA has granted both Fast-Track and Orphan Drug Designation for its treatment of SCA. The European Medicines Agency has also granted Orphan Drug Designation for the same indication. These designations signify the drug’s potential to address a serious unmet need and expedite its development and review.
Beyond SCA, troriluzole is also being evaluated in two late-stage studies for treating obsessive-compulsive disorder (OCD). Interim analysis of the second phase III OCD study is expected later in 2024, while top-line data from the first phase III OCD study is anticipated in the first half of 2025.
These promising developments highlight Biohaven’s dedication to developing innovative therapies that address critical unmet medical needs. The company’s commitment to researching and developing treatments for rare and debilitating diseases holds immense potential for improving the lives of countless individuals worldwide.
