Zevra Therapeutics (ZVRA) 44th Annual J.P. Morgan Healthcare Conference summary

Strategic vision and values

• Mission to redefine rare disease therapy and become a leading rare disease therapeutics company, driven by patient centricity, accountability, and innovation.

• Focus on disciplined investment, leveraging a strong balance sheet and experienced team to drive sustainable growth.

• Emphasis on not overextending resources, instead targeting high-value opportunities and building a culture dedicated to patient impact.

• Values include integrity, innovation, and courage in delivering on commitments.

Financial performance and resources

• Generated $72.3 million in net revenue in the first nine months of 2025, with $26.1 million in Q3.

• Ended Q3 2025 with $230.4 million in cash, cash equivalents, and investments, and $61.3 million in debt.

• Monetized a Pediatric Priority Review Voucher for $150 million, adding non-dilutive capital.

• Maintains multiple revenue streams, including product sales, expanded access programs, and royalties.

Product portfolio and pipeline

• Miplyffa, FDA-approved in September 2024 for Niemann-Pick disease type C (NPC), holds orphan drug exclusivity through 2031, with potential patent extension under review.

• OLPRUVA for urea cycle disorders (IP through 2036) and AZSTARYS for ADHD (royalties/milestones through 2037) complement the portfolio.

• Celiprolol in Phase III for vascular Ehlers-Danlos syndrome (vEDS), with IP through 2038 and ongoing pivotal DISCOVER trial.

• KP1077 in late-stage development for idiopathic hypersomnia and narcolepsy.