Biohaven (BHVN) Study Result summary

Study background and design

• Troriluzole was evaluated in pivotal phase 3 studies for spinocerebellar ataxia (SCA), a rare neurodegenerative disorder with no approved treatments, affecting about 15,000 in the U.S. and 24,000 in Europe and the U.K.

• The studies used real-world evidence with external controls from US (CRC-SCA) and Europe (EuroSCA), matched by propensity score for baseline characteristics and genotype progression rates.

• The primary outcome was change from baseline in the f-SARA scale at 3 years, with secondary endpoints at 1 and 2 years and comparisons to pooled controls.

• All endpoints and statistical plans were pre-specified and reviewed by the FDA, with Orphan Drug and Fast Track designations granted.

• Strong intellectual property protection is in place, with patent expiration anticipated in 2041.

Efficacy and safety results

• Troriluzole slowed SCA progression by 50%-70% over three years, translating to a 1.5-2.2 year delay in decline, with statistically significant benefits at all time points and across all analyses.

• Statistically significant superiority was achieved on nine consecutive pre-specified primary and secondary endpoints.

• Troriluzole reduced SCA progression by ~50% vs US controls, ~70% vs EU controls, and ~60% vs pooled controls.

• A 53% reduction in relative risk of falls was observed in the double-blind phase, and up to 60% reduction among ambulatory patients.

• Troriluzole was safe and well-tolerated, with an adverse event profile similar to placebo and no significant imbalances in serious adverse events.

Regulatory and commercial outlook

• NDA submission for troriluzole in all SCA genotypes is planned for Q4 2024, aiming for US commercialization in 2025.

• The EMA application for SCA3 remains under review, and new results will be incorporated into the ongoing European review.

• The commercial strategy targets the estimated 15,000 U.S. SCA patients, with about 6,000 currently diagnosed, and aims to accelerate diagnosis and establish troriluzole as the standard of care.

• Launch preparations include patient identification, education, access and reimbursement strategies, and a centralized support hub.

• The availability of an effective therapy is expected to increase diagnosis rates and patient engagement.