Zevra Therapeutics (NASDAQ:ZVRA) reported fourth quarter and full-year 2025 results highlighted by growing commercial traction for its Niemann-Pick disease type C (NPC) therapy MIPLYFFA and a shift to profitability, while outlining regulatory and clinical milestones expected in 2026.
Commercial momentum for MIPLYFFA
President and CEO Neil McFarlane said 2025 was “a banner year” with net revenue of $106.5 million, driven primarily by MIPLYFFA, which delivered $87.4 million in revenue during its first full year of commercialization. In the fourth quarter, total net revenue was $34.1 million, including $26.4 million from MIPLYFFA sales.
Chief Commercial Officer Josh Shafer pointed to what he called strong adherence once patients begin therapy, adding that refill rates have been encouraging, though he said it was still too early to provide meaningful persistency data over a full 12-month period. He also noted that about half of patients treated with MIPLYFFA are adults, consistent with the company’s experience in its prior U.S. expanded access program (EAP).
Diagnosis and access initiatives
Shafer attributed increased traction among newly diagnosed patients to a “three-pronged approach” built around the company’s disease awareness campaign (“Learn NPC: Read between the signs”), an AI-driven targeting model, and collaborations with genetic testing providers.
- Disease awareness: Zevra said it is engaging stakeholders through medical congresses and online resources focused on earlier detection and treatment.
- AI targeting: The company described a proprietary predictive model using claims data and electronic health records to identify potential NPC patients who may be misdiagnosed or undiagnosed, enabling targeted outreach to physicians.
- Genetic testing partnerships: Zevra said it expanded its collaboration with GeneDx, enabling eligible patients to receive the ExomeDx test at no charge, with results expected in as little as three weeks.
On market access, Shafer said coverage reached 68% of total covered lives after the first full year in the U.S., and that for remaining patients the company has secured “high reimbursement rates” through its market access and patient services efforts.
Ex-U.S. expansion and EMA update
Zevra is expanding access to arimoclomol (MIPLYFFA) beyond the U.S. through its global EAP, which had 113 patients enrolled at the end of 2025. McFarlane said the company built a strong reputation across Europe through the EAP, and in the fourth quarter established a new distribution agreement to extend the global EAP to select territories outside of Europe, initiating shipments of named patient supply prior to year-end. He cautioned that ordering patterns and new enrollment rates may be variable in early years until the patient base stabilizes, a point reiterated during the Q&A.
Regulatory progress in Europe is also ongoing. McFarlane said Zevra submitted a Marketing Authorisation Application (MAA) to the European Medicines Agency in late July 2025, supported by data including a pivotal Phase 2/3 trial, open-label extension results, expanded access programs, and a pediatric sub-study. The company received a “120-day list of questions” from EU regulators at the end of 2025 and said it was prepared to respond during the 90-day clock stop period. In response to an analyst question, management said the next milestone would be Day 150 questions following submission of the company’s responses.
McFarlane noted higher NPC diagnosis rates in Europe than the U.S., which he attributed in part to the European approval of miglustat for NPC and the resulting physician awareness. He said the company believes it is positioned to maximize its commercial impact in Europe through partnerships or independently.
Pipeline progress: celiprolol for vEDS
Beyond NPC, Zevra discussed development of celiprolol for vascular Ehlers-Danlos syndrome (vEDS). McFarlane described vEDS as a rare inherited connective tissue disorder caused by COL3A1 mutations, noting that approximately 7,500 individuals have been diagnosed in the U.S.
The company’s Phase 3 DiSCOVER study is designed to confirm clinical benefit seen in prior studies conducted outside the U.S. Zevra enrolled eight patients in the fourth quarter, bringing total enrollment to 52 of a planned 150 patients as of year-end 2025. Management also reported one confirmed event out of the 28 events required to trigger an interim analysis.
To boost enrollment, the company said it is building a network of genetic testing centers to support diagnosis and connecting with specialists such as vascular surgeons. Zevra also recently held a Type C meeting with the FDA to discuss regulatory options to accelerate the program, with further engagement expected in the second half of the year.
Financial results and balance sheet
For the fourth quarter, Zevra reported:
- Total net revenue: $34.1 million, including $26.4 million from MIPLYFFA sales, $0.4 million from OLPRUVA, $5.6 million in net reimbursements from the global EAP, and $1.8 million from royalties and other reimbursements under the AZSTARYS license.
- Operating expenses: $23.0 million, down $1.5 million year over year. R&D expense was $2.6 million (down $5.8 million), while SG&A expense was $20.4 million (up $4.3 million), driven largely by launch investments.
- Net income: $12.2 million, or $0.20 per basic and $0.19 per diluted share, compared with a net loss of $35.7 million in the year-ago quarter.
For the full year 2025, Zevra reported net income of $83.2 million, or $1.40 per basic and $1.35 per diluted share, compared with a net loss of $105.5 million in 2024. Operating expenses totaled $90.4 million, including $12.7 million in R&D and $77.6 million in SG&A.
As of Dec. 31, 2025, Zevra had $238.9 million in cash, cash equivalents, and investments, and approximately $61.9 million in total debt. In the Q&A, management said it evaluates capital allocation quarterly and is focused on investing in U.S. commercial execution, geographic expansion efforts, and advancing celiprolol.
The company also announced leadership updates, with McFarlane welcoming newly appointed CFO Justin Renz, whose official start date was the day of the call. Renz briefly said he planned to listen, learn, and engage with stakeholders while supporting execution and long-term value creation.
About Zevra Therapeutics (NASDAQ:ZVRA)
Zevra Therapeutics, Inc discovers and develops various proprietary prodrugs to treat serious medical conditions in the United States. The company develops its products through Ligand Activated Therapy platform. Its lead product candidate is KP1077, consisting of KP1077IH, which is under Phase 2 clinical trial for the treatment of idiopathic hypersomnia, and KP1077N, which is under Phase ½ clinical trial to treat narcolepsy. The company is also developing Celiprolol, a prodrug product candidate that is under Phase 1/2 clinical trial for the treatment of vascular Ehlers Danlos syndrome.
Further Reading
- Five stocks we like better than Zevra Therapeutics
- The gold chart Wall Street is terrified of…
- What a Former CIA Agent Knows About the Coming Collapse
- Elon Musk already made me a “wealthy man”
- Buffett, Gates and Bezos Quietly Dumping Stocks—Here’s Why
- 1,500 Banks Just Handed the Fed Your Bank Account
