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Zevra Therapeutics (ZVRA) investor relations material
Zevra Therapeutics Guggenheim Securities 2nd Annual Healthcare Innovation Conference summary
Complete event summary combining all related documents: earnings call transcript, report, and slide presentation.Guggenheim Securities 2nd Annual Healthcare Innovation Conference summary12 Nov, 2025
Company overview and portfolio
Commercial-stage rare disease company with two marketed products for urea cycle disorders and Niemann-Pick disease type C (NPC).
Late-stage development program in vascular Ehlers-Danlos Syndrome (phase III, enrolling patients).
Holds royalties and milestones from an ADHD program commercialized by another party.
Maintains a robust balance sheet with approximately $230 million in capital.
Niemann-Pick disease type C (NPC) launch and market dynamics
U.S. NPC product approved about a year ago; 137 prescription enrollment forms received, covering ~40% of diagnosed patients.
Patient identification efforts include disease state awareness and genetic testing, expanding beyond centers of excellence to community physicians.
Early adopters include both pediatric and adult patients, with a 50-50 split mirroring the expanded access program (EAP).
Increasing diagnosis of new patients through education and genetic counseling, expanding the addressable market beyond initial estimates.
High adherence and persistency rates observed, similar to EAP experience.
Commercial and payer feedback
Market research indicates improvements in gait and swallowing, with physicians preferring the agent based on long-term data.
Five-year open-label extension (OLE) data and additional pediatric data strengthen the product’s value proposition.
Payers are generally supportive, with strong data facilitating reimbursement and multi-modality treatment approaches.
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Next Zevra Therapeutics earnings date
Q4 20256 Mar, 2026
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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