Zevra Therapeutics, Inc. Q3 FY2025 earnings call

Key Points

• MIPLYFFA Performance: MIPLYFFA generated $22.4 million in Q3 net revenue. The company received 8 new prescription enrollment forms in Q3, with a total of 137 since launch. It is the first treatment approved in the U.S. for Niemann-Pick disease type C (NPC) and has strong scientific data supporting its efficacy in halting NPC progression.
• Europe Expansion: Zevra filed a Marketing Authorization Application (MAA) for MIPLYFFA in Europe, which is under review by the European Medicines Agency. The company has an Expanded Access Program in Europe and the U.K. with 92 patients enrolled, laying groundwork for potential commercial launch.
• OLPRUVA Strategy: Due to limited clinical differentiation in the UCD market, Zevra scaled back sales and marketing efforts for OLPRUVA but will maintain market access and patient support through AmplifyAssist.
• Development Pipeline: Actively recruiting patients in the DiSCOVER trial for Celiprolol as a treatment for Vascular Ehlers-Danlos Syndrome (VEDS). 44 patients enrolled out of 150 needed for full enrollment, with an interim analysis planned after 28 qualifying events.

In the third quarter of 2025, Zevra's net revenue totaled $26.1 million. MIPLYFFA contributed $22.4 million in net revenue, representing a significant portion of the total. Additionally, there were $2.4 million in net reimbursements from the French Expanded Access Program (EAP) for arimoclomol, $1.2 million from royalties under the AZSTARYS license, and $100,000 from OLPRUVA. MIPLYFFA's revenue contribution highlights its key role in the company's financial performance.

Forward-Looking Statements

• Europe Expansion: Zevra expects to continue advancing the MAA review in Europe and develop a go-to-market strategy, considering independent launch or strategic collaboration.
• Patent Term Extension: Pursuing a patent term extension for MIPLYFFA with the U.S. Patent and Trademark Office to extend coverage beyond orphan drug exclusivity.
• MIPLYFFA U.S. Performance: Committed to sustaining MIPLYFFA's strong performance in the U.S., leveraging disease awareness initiatives and clinical data to identify new patients.
• Celiprolol Development: Focus on progressing the Phase III DiSCOVER trial for Celiprolol in VEDS, with confidence in the trial's progress.

Risk Factors

• Working Capital Variability: Potential variability in working capital requirements due to timing of receivables and payables.
• Europe Launch Challenges: Uncertainties related to the outcome of the MAA review in Europe and challenges in achieving full market penetration.
• Competition: Competition in the rare disease space, which could impact MIPLYFFA's market position and commercial performance.

Q: As we think about the last 2 quarters, there's been 15 new start forms. Curious if any of these have been patients that are newly diagnosed through perhaps some of your efforts? And also, as we think about the next few quarters up ahead, how you're thinking about the potential of finding additional patients that currently aren't diagnosed today?

A: Neil McFarlane and Joshua Schafer discussed that some new enrollments are from newly diagnosed patients, driven by disease awareness initiatives, genetic testing collaborations, and analysis of electronic medical records. They mentioned that 137 prescription enrollment forms have been received in the first year, representing about 40% of the diagnosed patient population.

Q: Kristen Kluska asked about the balance sheet and how to take advantage of the robust balance sheet.

A: Neil McFarlane and LaDuane Clifton responded that the company's financial resources are sufficient to execute strategic priorities, including MIPLYFFA's U.S. launch, Europe expansion, and Celiprolol's Phase III study, with a disciplined approach to capital allocation.

Q: Samantha Corwin inquired about the average age/weight of patients on therapy and the split between Expanded Access Program and real-world patients.

A: Neil McFarlane stated that the clinical trial program was skewed towards children, but real-world experience shows a 50-50 split between children and adults. Weight was based on an average from the Expanded Access Program.

Q: Eddie Hickman asked about key drivers of coverage increase for MIPLYFFA and patient enrollment split between centers of excellence and non-centers.

A: Joshua Schafer mentioned that strong data and payer recognition of MIPLYFFA's efficacy drove coverage increase. Enrollments include both centers of excellence and community-based physicians.

Q: Sumant Kulkarni asked about MIPLYFFA's patient enrollment in the U.S. and Europe pricing, and KP1077 strategy.

A: Neil McFarlane discussed ongoing efforts to expand MIPLYFFA in Europe, with pricing to be determined, and stated that Zevra remains open to strategic options for KP1077 but provided no specific details.

Q: Jason Butler inquired about patients with enrollment forms not yet receiving reimbursed drug and time to reimbursement.

A: Joshua Schafer responded that the majority of patients eventually receive paid drug, with the time from enrollment to reimbursement shrinking due to strong data and team execution, sometimes down to 24 hours.