Zevra Therapeutics (ZVRA) Earnings Call Presentation

Company Overview - Zevra Therapeutics is focused on bringing life-changing therapeutics to the rare disease community[9] - The company has two commercial-stage rare disease products and an advanced clinical development pipeline[8,82] - Zevra has a strong financial position with $217 million in cash, cash equivalents, and investments as of April 1, 2025, including $687 million as of March 31, 2025, plus $1483 million net proceeds from a PRV sale[8,81] Product Portfolio - MIPLYFFA (arimoclomol) is FDA-approved for Niemann-Pick Disease Type C (NPC) and launched in November 2024[20] - Approximately 900 individuals in the U S live with NPC, with 300-350 diagnosed or treated[38] - OLPRUVA (sodium phenylbutyrate) for oral suspension is FDA-approved for Urea Cycle Disorders (UCD) in patients weighing 20 kg or greater and with a body surface area (BSA) of 12 m2 or greater[20,50] - Approximately 1,100 individuals are diagnosed with UCDs in the U S [59] Pipeline Development - The company is targeting an EMA MAA submission for arimoclomol in Europe in the second half of 2025[49] - Celiprolol is in Phase 3 development for Vascular Ehlers-Danlos Syndrome (VEDS), with IP through 2038[20] - Approximately 7,500 individuals in the U S are diagnosed with VEDS[74]