Zevra Therapeutics, Inc. Q4 FY2025 earnings call

Welcome newly appointed CFO Justin Renz. Zebra's mission to redefine life-changing therapies for rare disease patients. MyPlypha had strong commercial performance in 2025 with 52 enrollment forms in full year and 24 in Q4. MyPipe's increasing penetration in NPC treatment. Europe EAP had 113 patients enrolled by end of 2025, submitted MAA to EMA and received 120-day list of questions. Serpol in VEDS treatment development with ongoing phase three DISCOVER study. Headquarters moved to Boston.

2025 net revenue was $106.5 million. MyPlypha contributed $87.4 million, accounting for ~82% of total revenue. Fourth quarter total net revenue was $34.1 million, with $26.4 million from MyPlypha sales. Full year operating expenses were $90.4 million, R&D expense $12.7 million, SG&A expense $77.6 million. Full year net income was $83.2 million.

Drive commercial execution for MyPlypha in US and pursue patent term extension. Continue with MAA review in EU and expand access beyond Europe. Engage with FDA regarding Solipilol development plan acceleration.

Regulatory approval uncertainties for EMA MAA and Solipilol. Variability in ordering patterns and new enrollments in global EAP. Competition risks in rare disease therapy market.

Q: 24 start forms split of new vs existing diagnosed patients and pipeline cadence.

A: Josh mentioned growth in newly diagnosed due to clinical data, disease awareness campaign and genetic testing collaborations.

Q: Time from initial suspicion to diagnosis.

A: Difficult to answer due to rare disease variability, but new collaboration with GeneDx has results in ~3 weeks.

Q: EMA response and next steps.

A: On track to submit response within 90-day clock stop, next is 150-day not 180.

Q: Persistency and data.

A: Too early for meaningful persistency data but encouraged by refill rates.

Q: EAP revenue nature and growth.

A: EAP revenue from named patient reimbursed sales, variable quarter-to-quarter with new territories contributing.

Q: US sales sequential growth and addressable market.

A: Variability expected due to ultra-rare space, addressable market between 300-350 and 900 diagnosed.

Q: EMA MAA data and Soliprolol acceleration.

A: Submitted robust data package to EMA, engaging further with FDA on Soliprolol acceleration.

Q: Annual enrollments and diagnosed patients engaged.

A: 52 in 2025, ~40-50% of diagnosed patients in US engaged.

Q: Q4 new patient starts and identification efforts.

A: Combination of full year on market, disease awareness, AI targeting model and community physician efforts.

Q: Capital allocation.

A: Evaluate quarterly, with $238.9M cash at end of 2025, focus on current execution and pipeline opportunities