ZEVRA THERAPEUTICS, INC. Q1 FY2025 earnings call

• Commercial Excellence: MIPLYFFA launch exceeded expectations with over one-third of US NPC patients enrolled, and OLPRUVA's marketing strategy refined to target adult and OTC deficient populations. - Pipeline and Innovation: Phase 3 Discover trial of Celiprolol for VEDS progressing with 32 patients enrolled, assessing strategic alternatives for KP1077, out-licensing IP related to a pre-clinical pro-drug, and withdrawing NDA for a product. - Talent and Culture: Recognized on Fast Company's top 10 most innovative companies list. - Corporate Foundation: Monetization of pediatric rare disease PRV added $148.3 million non-dilutive capital, enhancing financial flexibility.

In the first quarter of 2025, Zevra reported net revenue of $20.4 million. MIPLYFFA contributed $17.1 million to the revenue, OLPRUVA contributed $0.1 million, net reimbursements from the French EAP for arimoclomol were $2.3 million, and royalties and other reimbursements under the AZSTARYS license totaled $0.9 million. MIPLYFFA's strong performance was a key driver, with over 122 prescription enrollment forms received since launch, and OLPRUVA saw 5 prescription enrollment forms in the first quarter of 2025.

Management highlighted ongoing execution of strategic pillars including commercial launches of MIPLYFFA and OLPRUVA, and support for Phase 3 trial of Celiprolol. No specific quantitative guidance provided beyond general progress expectations.

No detailed discussion of specific risks during the call, but it was noted that forward-looking statements are subject to risks and uncertainties outlined in SEC filings.

Q: Relative to the 122 patient enrollment forms, could you give details on the number of patients actually on MIPLYFFA right now and spread of reimbursed patients?

A: Josh Schafer mentioned they don't report on how many are actively on drug, but majority are on drug with some in benefits investigation process.

Q: Any sense of the top reason for denial of reimbursement for MIPLYFFA?

A: Josh Schafer stated denials are typical for rare disease products, but MIPLYFFA's differentiated clinical profile (halting disease progression, long-term data) helps address reimbursement challenges.

Q: How much visibility into whether patients are coming from undiagnosed vs diagnosed bucket for MIPLYFFA?

A: Josh Schafer mentioned NPC prevalence and 3 distinct cohorts, with initiatives to reach undiagnosed and diagnosed patients.

Q: Cadence of new patients starts for MIPLYFFA throughout the year?

A: Neil McFarlane stated they won't provide guidance but will be disciplined in metrics, with strong performance based on data.

Q: On Celiprolol trial, timeline for enrollment completion?

A: Neil McFarlane said they inherited the trial, are working on driving enrollment through targeting COL3A1 patient centers and genetic testing, but couldn't provide exact timeline.