Biohaven Ltd. (BHVN) has unveiled promising news for patients battling Spinocerebellar Ataxia (SCA), a rare neurodegenerative disease affecting approximately 15,000 people in the United States and 24,000 in Europe and the United Kingdom. The company released topline results from its pivotal Study BHV4157-206-RWE, showcasing the effectiveness of its drug candidate, troriluzole, in slowing disease progression.
The study achieved its primary endpoint, demonstrating statistically significant improvements in the f-SARA (Functional Scale for Assessment of Rare Ataxia) score after three years of treatment. Notably, these improvements were observed at both year one and year two, indicating a sustained benefit over time.
Currently, there are no FDA-approved treatments for SCA. However, Biohaven’s research offers a glimmer of hope for patients. The data from multiple analyses highlight a robust and clinically significant slowing of disease progression in those treated with troriluzole.
Compared to untreated patients, the rate of decline was reduced by a remarkable 50-70%, translating into a 1.5-2.2 years delay in disease progression over the three-year study period. The odds ratio for disease progression was 4.1 times higher for the untreated control group compared to those receiving troriluzole.
The study’s primary objective was to evaluate the long-term efficacy of troriluzole. The results consistently demonstrated statistically significant and sustained benefits in f-SARA scores at year one, year two, and year three for patients receiving troriluzole treatment, when compared to a rigorously matched natural history control group.
Based on these encouraging findings, Biohaven is poised to submit a New Drug Application (NDA) to the FDA in the fourth quarter of 2024. The company’s troriluzole development program has produced the largest clinical trial dataset for SCA and now has a follow-up in some patients who have been treated with troriluzole for over five years.
If approved, Biohaven aims to commercialize troriluzole for the treatment of SCA in the United States in 2025. The company is optimistic about the drug’s potential for priority review, which could expedite its availability to patients.
The positive news sent Biohaven’s stock soaring by 12% in premarket trading, reaching $45.20 at the last check on Monday.
