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Zevra Therapeutics (ZVRA) investor relations material
Zevra Therapeutics Cantor Global Healthcare Conference 2025 summary
Complete event summary combining all related documents: earnings call transcript, report, and slide presentation.Cantor Global Healthcare Conference 2025 summary3 Sep, 2025
Company overview and product portfolio
Commercial stage rare disease company with two marketed products: one for Niemann Pick disease type C (NPC) and another for urea cycle disorders (UCDs).
Late-stage development program for vascular Ehlers Danlos syndrome (VEDS) and an out-licensed ADHD program generating royalties.
Strong balance sheet with approximately $217 million in cash.
NPC product launch and clinical data
NPC affects about 900 patients in the US and 1,100 in Europe, with 300–350 diagnosed in the US.
Recent mechanism of action data shows upregulation of the ClearGene network and improved lysosomal function.
Long-term data (up to five years) demonstrates halting of disease progression, supporting both physician and payer confidence.
Intellectual property and regulatory updates
Patent term extension (PTE) is under review, with potential to extend protection beyond current orphan drug exclusivity.
Public review of the PTE is expected in September, followed by a 60-day comment period and final decision.
Mephliva patent term extension timeline
Mephliva Europe commercialization approach
VEDS trial enrollment acceleration outlook
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Next Zevra Therapeutics earnings date
Q3 202511 Nov, 2025
Next Zevra Therapeutics earnings date
Q3 202511 Nov, 2025
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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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