Zevra (ZVRA) Q2 FY26 earnings call: guidance and Q&A

How will Zevra leverage its strong cash position, and what are the details/implications of the EMA reexamination process with new rapporteurs?

Management reiterated its disciplined capital allocation strategy focused on MIPLYFFA growth, global expansion, celiprolol advancement, and external opportunities. For EMA, Neil McFarlane stated confidence in the comprehensive evidence and the opportunity for new expert voices to interpret the data, but could not comment on specific process details.

“our confidence is grounded in the totality of the evidence and the clear unmet patient need.”

asked by Kristen Kluska · answered by Neil McFarlane

What new information or approach will Zevra use in the EMA reexamination process compared to the initial submission?

Neil McFarlane explained that the reexamination allows Zevra to address specific grounds for refusal and bring new expert voices to interpret the robust and comprehensive evidence package, which includes long-term open-label extension data, EAP data, pediatric substudy, and a strong safety database, all building on the positive pivotal study.

“Under the reexamination process, we'll have the opportunity to focus specifically to address these specific grounds that have been cited for the refusal.”

asked by Sumant Kulkarni · answered by Neil McFarlane

Can management bracket the variability of non-French EAP numbers and provide directional color on the U.S. MIPLYFFA patient mix (new vs. previously diagnosed, adult vs. pediatric)?

Neil McFarlane stated that non-French EAP ordering patterns remain unpredictable due to timing, volume, program type, and geography, making bracketing difficult, but the French EAP is consistent at ~$2.5M net/quarter. Josh Schafer noted an equal mix of children and adults for U.S. enrollments, with a mix of newly and previously diagnosed patients from various practice settings, reinforcing confidence in the 350-900 patient TAM.

“The variability in ordering patterns that come through other named patient reimbursement programs can come in -- it's one patient at a time, and it can be one patient that orders for an annual basis, one patient that orders for quarterly or a monthly supply.”

asked by Eddie Hickman · answered by Neil McFarlane

How do current MIPLYFFA enrollment forms compare to 2025, and is there better visibility/comfort with quarterly enrollment levels? Any additional color on the celiprolol FDA meeting and confidence moving forward?

Neil McFarlane expressed confidence in the upward trend of enrollments (23 in H1 2026 vs 52 in full-year 2025) and the ability to unlock newly diagnosed patients, reinforcing the 350-900 patient TAM. For celiprolol, the FDA meeting was informative, providing opportunities to accelerate clinical development through regulatory pathways and drive enrollment, with plans to re-engage FDA in H2 FY26.

“I'm pleased with where we are through the year, but I'm even more confident about the TAM being somewhere between that 350 and 900 because we're seeing these newly diagnosed patients earlier in the launch process than we would have expected today.”

asked by Brandon Folkes · answered by Neil McFarlane

Can management provide details on patient attrition for commercial MIPLYFFA patients and compare Q2 end inventory to Q1 end?

Josh Schafer stated that MIPLYFFA refill and persistence rates are high, with most attrition due to the severity of NPC leading to patient death, which is a "super small" rate. Justin Renz clarified that channel inventory fell below target in Q1 due to shipment timing but returned to normal target levels by Q2 end.

“The patients that we do lose, unfortunately, are -- it's really a reflection more of the severity of the disease. It is a oftentimes fatal disease and patients will progress to the point of dying.”

asked by Jonathan Aschoff · answered by Joshua Schafer