Biohaven Stock Spikes After Drug Candidate To Treat Rare Neurodegenerative Disease Shows Promise

Core Insights - Biohaven Ltd. announced positive topline results from pivotal Study BHV4157-206-RWE, demonstrating the efficacy of troriluzole in treating Spinocerebellar Ataxia (SCA) over three years [1][3] - The study achieved its primary endpoint, showing statistically significant improvements in the f-SARA scale at years 1 and 2, indicating a robust treatment effect [1][3] - SCA is a rare neurodegenerative disease affecting approximately 15,000 individuals in the U.S. and 24,000 in Europe and the U.K. [1] Treatment Efficacy - Data from multiple analyses indicate a clinically meaningful slowing of disease progression in SCA patients treated with troriluzole, translating to a 50-70% slower rate of decline compared to untreated patients [2] - The treatment represents a delay in disease progression of 1.5-2.2 years over the three-year study period [2] - The odds ratio for disease progression in untreated patients compared to those treated with troriluzole was 4.1, highlighting the treatment's effectiveness [3] Regulatory and Commercial Plans - Biohaven plans to submit a New Drug Application (NDA) to the FDA in Q4 2024, aiming for commercialization of SCA treatment in the U.S. by 2025, contingent on approval [3][4] - The troriluzole development program has generated the largest clinical trial dataset in SCA, with some patients followed for over five years [4] Market Reaction - Following the announcement, Biohaven's stock rose by 12%, reaching $45.20 in premarket trading [4]