Biohaven (BHVN) R&D Day 2025 Presentation summary

Pipeline and innovation overview

• Advancing a diversified portfolio across rare disease, oncology, renal, obesity, cardiovascular, immunology, inflammation, and neuroscience, with multiple assets in clinical and preclinical stages.

• Proprietary platforms include MODE™ and TRAP™ degraders, next-gen antibody drug conjugates (ADCs), and novel small molecules targeting key disease pathways.

• Integrated discovery engine supports rapid development and value creation, with a focus on pioneering therapies for rare diseases and advancing cancer treatments.

Troriluzole (VYGLXIA®) for spinocerebellar ataxia (SCA)

• Troriluzole NDA under FDA priority review for SCA, with PDUFA date in 2H 2025; commercial launch preparations underway.

• Demonstrated 50–70% slowing of disease progression over 3 years in real-world evidence studies, with robust and durable treatment benefit across SCA genotypes.

• Efficacy confirmed by multiple studies, with significant reduction in falls and favorable safety profile; studied in over 2,000 subjects.

• VYGLXIA® would be the first and only approved treatment for SCA, targeting a US prevalence of ~15,000 patients.

• Commercial strategy includes patient identification, genetic testing programs, and partnerships with advocacy groups and centers of excellence.

Kv7 platform: BHV-7000 for epilepsy and major depressive disorder (MDD)

• BHV-7000 is a next-generation Kv7 activator with a wide therapeutic index, minimal neurological side effects, and strong anti-seizure efficacy.

• Phase 1 and ongoing Phase 2/3 studies show favorable safety and tolerability, with no serious or dose-limiting toxicities.

• Pivotal topline results for MDD expected 2H 2025; focal epilepsy study topline results expected 1H 2026.

• Kv7 activation offers a novel mechanism for treating both epilepsy and depression, with potential to address unmet needs in efficacy and tolerability.