Zevra Therapeutics (ZVRA) Corporate presentation summary

Strategic vision and mission

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

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

Commercial and pipeline highlights

• 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, and celiprolol in Phase 3 for Vascular Ehlers-Danlos Syndrome (VEDS).

• Arimoclomol MAA under review by EMA, with global expansion plans.

Financial performance and position

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

• Net income for H1 2026 was $46.6M, with a strong cash position of $260.2M and no debt as of June 30, 2026.

• Q2 MIPLYFFA US revenue grew 23% over Q1, demonstrating strong commercial execution.