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Zevra Therapeutics
Zevra Therapeutics (ZVRA) investor relations material
Zevra Therapeutics 44th Annual J.P. Morgan Healthcare Conference summary
Complete event summary combining all related documents: earnings call transcript, report, and slide presentation.44th Annual J.P. Morgan Healthcare Conference summary15 Jan, 2026
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.
MIPLYFFA: US undiagnosed patient strategy?
Celiprolol DISCOVER trial interim analysis timing?
Arimoclomol MAA: EMA approval timeline?
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Next Zevra Therapeutics earnings date
Q4 20256 Mar, 2026
Zevra Therapeutics
Next Zevra Therapeutics earnings date
Q4 20256 Mar, 2026
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Frequently asked questions
Zevra Therapeutics Inc., previously known as KemPharm Inc., is a biopharmaceutical company specializing in the development of proprietary prodrugs for the treatment of serious medical conditions, with a focus on rare diseases. The company utilizes its Ligand Activated Therapy technology to create enhanced versions of FDA-approved drugs and new compounds for potential new disease indications. Zevra's product pipeline includes KP1077, designed for the treatment of idiopathic hypersomnia (IH) and narcolepsy, and KP879 for stimulant use disorder, both in Phase II clinical trials. Additionally, Zevra has FDA-approved products like AZSTARYS for attention deficit hyperactivity disorder and APADAZ, a combination treatment containing a prodrug of hydrocodone and acetaminophen. The company is headquartered in Celebration, Florida, and its shares are listed on the NASDAQ.
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