Zevra Therapeutics (ZVRA) Corporate presentation summary

Portfolio and commercial position

• Portfolio spans U.S.-marketed MIPLYFFA for NPC and OLPRUVA for UCD, EU regulatory review of arimoclomol for NPC, and late-stage celiprolol development for VEDS.

• MIPLYFFA received FDA approval Sep 20, 2024, with orphan drug exclusivity through 2031 and patent protection through 2041; OLPRUVA received FDA approval Dec 22, 2022, with IP through 2036.

• Arimoclomol’s MAA was submitted to EMA Jul 28, 2025; the application is under review. Celiprolol’s Phase 3 trial is ongoing.

MIPLYFFA and NPC opportunity

• NPC is an ultra-rare, progressive and fatal disorder; an estimated 900 people live with NPC in the U.S., of whom 300–350 are diagnosed or treated.

• MIPLYFFA adoption reached ~50% of diagnosed patients; Q2 2026 patient enrollments totaled 184, and payor coverage reached 69% of covered lives.

• Trial and extension data presented show improvement at the first evaluation at Week 12, halted disease progression at 12 months, and a durable effect for 5+ years; ~80% of Phase 2/3 participants took miglustat.

• Growth efforts include finding undiagnosed patients through genetic testing collaborations and an AI predictive model, expanding access pathways, and building distributor networks beyond the U.S. and Europe.

Celiprolol and VEDS

• VEDS is a life-threatening connective tissue disorder with no approved treatments; ~7,500 people in the U.S. are diagnosed and living with VEDS, and median survival age is 51 years.

• Prior randomized and real-world studies reported ~5% annual major vascular event rates on celiprolol versus ~12% untreated, with improved survival observed in treated patients.

• The decentralized Phase 3 DISCOVER trial is designed to confirm prior clinical benefit; 66 of 150 patients were enrolled as of Q2 2026, with interim analysis at 28 events and final analysis at 46 events.