ZEVRA THERAPEUTICS, INC. Q4 FY2024 earnings call

• MIPLYFFA launch: Early days exceeded expectations, with 109 patient enrollment forms in Q4, all active U.S. EAP participants submitted enrollment forms, and plan to expand availability outside the U.S. with a marketing authorization application in Europe in the second half of 2025.
• OLPRUVA launch: Enrollments increased to 4 in Q4 from 3 in Q3, refined strategy to target specific patient segments, added field reimbursement managers, and sponsored National Urea Cycle Disorders Foundation campaign.
• Pipeline and innovation: Celiprolol Phase 3 DiSCOVER trial enrolled 8 patients in Q4, total 27; KP1077 exploring strategic alternatives to advanced clinical development; rationalized in-house discovery efforts and outsourced activities.
• Talent and culture: Expanded executive leadership team in 2024, consolidated development and scientific functions under Chief Medical Officer.
• Corporate foundation: Refinanced debt, completed secondary offering, and entered into agreement to monetize priority review voucher for $150 million.

In the fourth quarter of 2024, net revenue was $12 million, with $10.1 million from MIPLYFFA, $0.1 million from OLPRUVA, $1.1 million from net reimbursements from the French EAP for arimoclomol, and $0.7 million from royalties and other reimbursements under the AZSTARYS license. For the full year 2024, net revenue was $23.6 million, including $10.1 million from MIPLYFFA, $0.1 million from OLPRUVA, $9.1 million from net reimbursements from the French EAP for arimoclomol, and $4.3 million from royalties and other reimbursements under the AZSTARYS license agreement.

• Plan to submit marketing authorization application in Europe for MIPLYFFA in the second half of 2025.
• Cash runway guidance excludes PRV proceeds but extends into 2029 based on current resources and operating forecast.
• Proceeds from PRV sale will provide non-dilutive capital to execute strategic priorities.

Q: Please give a sense of the last dialogue with the agency in Europe regarding MIPLYFFA filing and the plan.

A: Last interaction with EMA was about four years ago; currently consulting with corporate consultancy group to address previous comments, and planning to file in the second half of 2025 with confidence it will be accepted.

Q: How conservative are the cash runway projections and does it include European approval?

A: Cash runway projections are conservative; it does not include European approval yet but is based on current execution plans.

Q: Give quantitative color on MIPLYFFA sales during the quarter and net patient adds outlook.

A: Q4 MIPLYFFA revenue included initial patient dispenses and some refills; net patient adds expected to continue with focus on expanding beyond EAP sites to diagnosed and untreated patients.

Q: Key variables influencing MIPLYFFA patient enrollment trajectory and status of reimbursement for initial 109 patients.

A: Transition of EAP patients and deployment of sales teams are key variables; 30% of initial 90 patients had authorization for payment, and improvement expected as team continues to engage with payers.

Q: Patients submitting enrollment forms denied and rationale behind denials.

A: There have been a couple of denials, but team works with patients through appeals and medical exception pathway to help navigate and ensure no patient goes without drug.

Q: Discontinuation rates and long-term retention of current patients.

A: Too early in launch to address discontinuation rates, but history of high continuity of care seen in EAP and open-label extension studies suggests longevity if patients remain on MIPLYFFA and miglustat