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 corporate foundation

• Achieved net revenue of $106.5M 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.

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

• Demonstrated financial discipline and operational flexibility.

Product portfolio and pipeline

• Commercial-stage rare disease products include MIPLYFFA (arimoclomol) for NPC and OLPRUVA for UCD.

• MIPLYFFA received FDA approval in September 2024 and is under EMA review; IP protection through 2031.

• Celiprolol in Phase 3 for VEDS, with orphan drug and breakthrough therapy designations.

• Multiple revenue sources and late-stage clinical assets support future growth.