Zevra Therapeutics (ZVRA) Corporate presentation summary

Mission and vision

• Aims to redefine possibilities in rare disease therapeutics, focusing on patient-centric innovation and transformational impact.

• Values include patient centricity, accountability, integrity, innovation, and courage.

Financial performance and position

• Generated $106.5M net revenue in 2025, with $87.4M from MIPLYFFA and $13.0M from expanded access reimbursements.

• Ended 2025 with $238.9M in cash, cash equivalents, and investments, and $61.9M in debt.

• Achieved net income of $83.2M in 2025, or $1.40 per basic share.

Product portfolio and pipeline

• Commercial-stage products include MIPLYFFA (NPC), OLPRUVA (UCD), and AZSTARYS (ADHD), with royalties and milestones from partnered assets.

• MIPLYFFA approved by FDA in 2024, with orphan drug exclusivity through 2031; MAA under review by EMA.

• Late-stage pipeline includes arimoclomol (NPC), celiprolol (VEDS), and KP1077 (IH, narcolepsy), with ongoing Phase 3 trials.