Zevra Therapeutics (ZVRA) Corporate presentation summary

Mission and vision

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

• Seeks to make a transformational impact through insights from patients and a culture of integrity and courage.

Portfolio and pipeline

• Offers commercial-stage rare disease products and late-stage clinical assets, including MIPLYFFA for NPC and OLPRUVA for UCD.

• Arimoclomol (MIPLYFFA) approved by FDA in 2024 for NPC, with EU MAA under review and IP protection through 2031.

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

Financial performance and position

• Generated $106.5M net revenue in 2025 and exited Q1 2026 with $236.8M in cash, cash equivalents, and investments.

• Q1 2026 net income was $37.9M, with no debt and strong operational discipline.

• Revenue sources include product sales, expanded access reimbursements, and licensing/royalty income.