Zevra Therapeutics — Third Quarter 2026 Earnings Summary

Financial Performance:

• Total net revenue reached $37.8 million for Q3 2026, a 45% increase from $26.1 million in Q3 2025.
• Revenue composition included $34.0 million from MIPLYFFA, $0.2 million from OLPRUVA, and $3.6 million in net reimbursements from the global Expanded Access Program (EAP).
• Net income was $19.2 million ($0.31 basic, $0.26 diluted per share) for Q3 2026, compared to a net loss of $0.5 million ($(0.01) per share) in Q3 2025.
• Operating expenses were $24.1 million for Q3 2026, up from $20.4 million in Q3 2025, driven by a $1.5 million increase in R&D to $4.9 million and a $2.2 million increase in SG&A to $19.1 million.
• Cost of product revenue was $1.8 million for Q3 2026, excluding non-cash intangible asset amortization, compared to $1.2 million in Q3 2025.
• The company recognized $5.1 million in non-cash income from fair value adjustments related to warrant and contingent value rights (CVR) liability in Q3 2026, versus a $5.5 million non-cash expense in Q3 2025.
• Cash, cash equivalents, and investments totaled $270.9 million as of September 30, 2026.
• Long-term debt was eliminated, dropping from $61.9 million as of December 31, 2025, to zero as of September 30, 2026.

Guidance and Future Outlook:

• The Company anticipates filing a supplemental New Drug Application (sNDA) for MIPLYFFA in infants less than 24 months of age in Q1 2027.
• Management stated that available financial resources are sufficient to execute on strategic priorities based on the current operating forecast.
• The Company is awaiting the outcome of a requested re-examination of its Marketing Authorisation Application for arimoclomol in the EU.
• Zevra recently engaged with the FDA for a Type C meeting to discuss a potential submission strategy for celiprolol.

Business Segments and Product Lines:

• MIPLYFFA (arimoclomol) received 13 prescription enrollment forms for Niemann-Pick disease type C (NPC) in Q3 2026, bringing the total to 197 since launch.
• The global Expanded Access Program enrolled 134 patients as of September 30, 2026.
• The Phase 3 DiSCOVER trial for celiprolol (Vascular Ehlers-Danlos Syndrome) enrolled six patients in Q3 2026, bringing the total enrolled to 72 with four confirmed events.
• OLPRUVA contributed $0.2 million in net revenue for the quarter.

Market and Competitive Landscape:

• MIPLYFFA has received Orphan Medicinal Product designation by the European Medicines Agency (EMA) for the treatment of NPC.
• Celiprolol holds Orphan Drug and Breakthrough Therapy designations from the U.S. FDA.
• The Company is pursuing European access for arimoclomol and expanding U.S. commercialization for NPC.

Risks and Challenges:

• MIPLYFFA carries risks of hypersensitivity reactions (urticaria, angioedema), embryofetal toxicity, and increased serum creatinine without affecting glomerular function.
• OLPRUVA carries risks of neurotoxicity, hypokalemia, and edema.
• Forward-looking statements regarding EU approval, sNDA filing, and financial resource sufficiency are subject to uncertainties and risks detailed in the company's Form 10-K.

Management Commentary and Tone:

• CEO Neil F. McFarlane stated the company made continued progress across the business, building on MIPLYFFA's commercial performance and advancing the pipeline.
• Management emphasized a focus on reaching more patients with Niemann-Pick disease type C, advancing celiprolol for vascular Ehlers-Danlos syndrome, and pursuing European access to arimoclomol.
• The tone reflected confidence in the strong financial position enabling the advancement of strategic priorities.

Other Key Points:

• The Company hosted a conference call and webcast on November 3, 2026, to discuss results.
• Total shares of common stock outstanding were 59,439,959 as of September 30, 2026.
• Fully diluted common shares were 68,968,909, including 6,999,571 shares from equity incentive plans and 2,529,379 shares from warrant exercises.
• The company recognized a $43.3 million gain on the sale of future royalties, intellectual property, and other assets in the nine months ended September 30, 2026.
• A $58.7 million impairment of intangible assets was recorded in the nine months ended September 30, 2025.