Zevra Therapeutics, Inc. Q1 FY2026 earnings call

• Building a durable rare disease company with disciplined execution, financial strength, and commitment to patients. - Advancing MyPlypha as a foundational treatment for NPC in the US, with 170 prescription enrollment forms since launch, 9 in Q1, and added to NPC clinical practice guidelines. - Advancing Soliprolol through a Phase III study for VEDS, with 62 patients enrolled in the DISCOVER trial, 10 in Q1. - Optimizing portfolio by divesting non-core assets, selling FDX portfolio for $50 million and resolving legal dispute. - Balance sheet is strong with $236.8 million cash and no debt. - Operating expenses in Q1 2026 were $25.2 million, R&D expense $4.4 million, SG&A expense $20.8 million. - Relocated corporate headquarters to Boston and strengthened leadership.

In the first quarter of 2026, total net revenue was $36.2 million, a 78% increase over Q1 2025. Net revenue is composed of $24.6 million from MyPlypha net sales in the US, $0.3 million from OPRUVA, $10.2 million in net reimbursements from the global EAP for Aramacomal, and $1.1 million in royalty revenue. The sale of the FDX portfolio to ComAv Therapeutics for $50 million impacted financials, with a one-time gain of approximately $43.3 million and a one-time expense of approximately $10 million associated with early debt extinguishment.

Q: Now that MyPlypha has been added to the NPC clinical practice guidelines, what does this mean for physician adoption?

A: The guidelines reinforce the NPC severity scale, genetic testing as a key endpoint, and the importance of early detection and combination therapy. It helps build confidence and consistency for HCPs.

Q: TAP revenue growth was robust. Is France driving that growth or other countries? And what are quantitative milestones around AI-driven patient identification and genetic testing efforts?

A: 122 patients enrolled in Q1 2026 in multiple territories, with variability in ordering pattern. Internally monitor number of newly diagnosed patients, new prescribers, etc.

Q: Miplypha is at almost 50% share of diagnosed and treated patients in the US. What about competitor share and concurrent use?

A: Clinicians use combination therapy, guidelines reinforce combination therapy consideration. Coverage exists for patients, including through medical exception pathways.

Q: On Celeprolol, what options for moving faster to bring to VEDS patients?

A: Had Type C meeting in Q1, preparing for follow-up meeting in second half of 2026, focusing on boosting enrollment and accelerating clinical development.

Q: Update on marking authorization application questions and CHMP opinion timeline?

A: Responded to 120-day list of questions within clock stop period, engagement is standard, looking forward to next engagement with European regulators.

Q: Profile of newly diagnosed patients and inventory reversal in 2Q?

A: Newly diagnosed patients have heterogeneous symptoms, inventory expected to fall back within targeted range by end of second quarter.

Q: Time from enrollment form to getting product and difference in net revenue per patient?

A: Time varied due to payer type and reauthorization. 69% covered lives allows formulary access, no preferred position, and net price variability due to no current contracting.