ZEVRA THERAPEUTICS, INC. Q2 FY2025 earnings call

Vision

• Zevra aims to be a leading life sciences company serving patients with rare diseases by bringing life-changing therapeutics.

Strategy Pillars

• Commercial excellence, pipeline and innovation, talent and culture, corporate foundation.

Key Achievements

• Q2 net revenue reached $25.9 million.
• Completed the sale of PRV for $150 million, strengthening the balance sheet.
• Submitted the arimoclomol marketing authorization application (MAA) to the European Medicines Agency.
• U.S. launch of MIPLYFFA: 129 prescription forms by end of Q2, with MIPLYFFA net revenue growing 26% quarter-over-quarter from Q1 to Q2.
• In Europe, estimated 1,100 people live with NPC, and Zevra is focused on securing approval and optimizing the go-to-market strategy for MIPLYFFA.
• OLPRUVA had slow uptake, with refining marketing efforts to address challenges.
• Pipeline progress: Celiprolol Phase III DiSCOVER trial for Vascular Ehlers-Danlos syndrome (VEDs) enrolled 7 patients in Q2, with a genetic testing initiative ramping up.

In the second quarter of 2025, Zevra's net revenue totaled $25.9 million. The key product segments included: MIPLYFFA contributed $21.5 million in net revenue. OLPRUVA generated $300,000 in net revenue. Additionally, there was $2.6 million from the French Expanded Access Program (EAP) for arimoclomol, $1.2 million from royalties and other reimbursements under the AZSTARYS license, and an upfront payment of $300,000 from the out-license of dextrorphan.

Forward-Looking Statements

• MIPLYFFA revenue is expected to continue growing, building on the 26% quarter-over-quarter increase from Q1 to Q2.
• Continued focus on securing European approval for arimoclomol and executing the go-to-market strategy.
• Progress in the celiprolol Phase III trial is anticipated, with the genetic testing initiative aiming to accelerate enrollment.

Risks

• Uncertainties in regulatory approval processes for arimoclomol in Europe.
• Slow uptake of OLPRUVA and challenges in reimbursement.
• Potential variability in patient enrollment and retention rates for MIPLYFFA, especially with the early stage of the launch.

Q: About U.S. MIPLYFFA enrollments and specialties, which specialties are contributing to new patient enrollments?

A: Initially, early patients came from neurologists, pediatricians, medical geneticists, and emerging psychiatrists.

Q: How does converting EAP patients in Europe compare to the U.S. execution?

A: Zevra has an expanded access program (EAP) in Europe with 89 patients at the end of Q2, and growth in this program bodes well for conversion post-approval in Europe.

Q: Can you walk through the different patient segments for MIPLYFFA?

A: Segments include diagnosed patients not on therapy, diagnosed patients on other treatments, and undiagnosed patients. Initiatives are in place to reach each segment.

Q: What percent of patients are on paid MIPLYFFA?

A: MIPLYFFA revenue grew 26% quarter-over-quarter from Q1 to Q2, indicating conversion, but details on paid performance are tied to enrollment and reimbursement.

Q: How is OLPRUVA commercialization investment structured?

A: There is synergy with MIPLYFFA in commercial infrastructure, with overlap in efforts.

Q: What's the pace of celiprolol trial enrollment?

A: The Phase III DiSCOVER trial for celiprolol enrolled 7 patients in Q2, with a genetic testing initiative ramping up to accelerate future enrollment.

Q: How many unique MIPLYFFA prescribers are there and covered lives?

A: The number of unique prescribers has grown, and covered lives are expected to increase as the launch progresses, with coverage at 52% currently.