Zevra Therapeutics (ZVRA) Corporate presentation summary

Mission and vision

• Focused on redefining possibilities for rare disease therapies, aiming for transformational impact through patient insights and innovation.

• Core values include patient centricity, accountability, integrity, innovation, and courage.

Portfolio and pipeline

• Commercial-stage rare disease products with late-stage clinical assets and multiple revenue sources.

• MIPLYFFA (arimoclomol) approved for Niemann-Pick Disease Type C (NPC) in the US, with orphan drug exclusivity through 2031 and patent protection through 2041.

• OLPRUVA approved for Urea Cycle Disorders, with IP through 2036.

• Celiprolol in Phase 3 for Vascular Ehlers-Danlos Syndrome (VEDS), with ongoing pivotal trial and regulatory engagement.

Financial performance and position

• H1 2026 net revenue of $75.9M, with $54.9M from MIPLYFFA and $19.1M from expanded access reimbursements.

• Q2 2026 ended with $260.2M in cash, cash equivalents, and investments, and no debt.

• H1 2026 net income of $46.6M, or $0.76 per basic share.