Biohaven (BHVN) Corporate Presentation summary

Pipeline and innovation highlights

• Advancing a diversified portfolio across neuroscience, immunology, inflammation, oncology, renal, obesity, and rare diseases, with multiple assets in late-stage development and pivotal trials planned or ongoing in 2025-2026.

• Novel degrader platform (MODE™ and TRAP™) validated in clinic, showing rapid, deep, and selective removal of disease-causing proteins in over 160 individuals dosed, with promising safety and efficacy.

• Kv7 activator (Opakalim/BHV-7000) in late-stage trials for epilepsy and major depressive disorder, demonstrating strong tolerability and efficacy in both preclinical and clinical studies.

• TRPM3 antagonist (BHV-2100) shows proof-of-concept for pain, with favorable safety and pharmacokinetics, advancing to later-stage trials.

• Oncology pipeline features next-generation ADCs, including Trop2 and FGFR3 ADCs, with early clinical data showing tumor reduction and favorable safety profiles.

Key clinical programs and data

• Vyglxia® (troriluzole) for spinocerebellar ataxia (SCA) under FDA priority review, with 8 years of data showing 50-70% slowing of disease progression and significant reduction in fall risk.

• Taldefgrobep alfa (BHV-2000) for obesity and spinal muscular atrophy (SMA) demonstrates improved body composition, muscle mass, and metabolic health, with robust safety and efficacy in over 700 participants.

• IgG and Gd-IgA1 degraders (BHV-1300, BHV-1400) show rapid, tunable, and selective reduction of pathogenic antibodies, with pivotal trials planned for Graves' disease, myasthenia gravis, and IgA nephropathy.

• BHV-8000, a brain-penetrant TYK2/JAK1 inhibitor, is in Phase 2/3 for Parkinson's disease and Alzheimer's, with strong preclinical and clinical evidence for targeting neuroinflammation.

• Oncology assets (BHV-1510, BHV-1530) demonstrate early efficacy in difficult-to-treat tumors, with combination strategies showing synergy and favorable safety.

Financial and strategic outlook

• Cash position of ~$408M as of June 30, 2025, with additional royalty streams from Pfizer on rimegepant and zavegepant sales, and potential for further debt drawdown upon regulatory approvals.

• Multiple key milestones anticipated in 2025-2026, including pivotal trial readouts, regulatory submissions, and potential product launches in SCA, epilepsy, depression, obesity, and oncology.

• Commercial strategy for Vyglxia® in SCA leverages centralized care, patient advocacy, and targeted outreach to drive early diagnosis and adoption.

• Discovery engine continues to deliver new INDs and candidates, ensuring pipeline sustainability and expansion into additional indications.