Zevra Therapeutics Q3 Earnings Call Highlights

Key Points

• Revenue rose 45% year over year to $37.8 million, driven by MIPLYFFA U.S. sales of $34 million, up 52%. Zevra reported $19.2 million in net income, including a $5.5 million non-cash warrant valuation credit.
• MIPLYFFA enrollment forms reached 197 cumulatively, with 13 added in the quarter; payer coverage stood at 69%. Management cited steady new-patient identification and increased disease-awareness activity as signs the addressable NPC population may exceed earlier estimates.
• Zevra expects to seek FDA approval in the first quarter of 2027 to extend MIPLYFFA use to infants under 24 months, and expects a European regulatory opinion on arimoclomol in the fourth quarter. The company ended September with $270.9 million in cash and investments.

Zevra Therapeutics NASDAQ: ZVRA reported third-quarter 2026 net revenue of $37.8 million, up 45% from $26.1 million a year earlier, driven primarily by U.S. sales of MIPLYFFA for Niemann-Pick disease type C, or NPC.

Net U.S. product revenue from MIPLYFFA reached $34 million in the quarter, rising 12.6% sequentially and 52% from the prior-year period, according to Chief Financial Officer Justin Renz. The company also reported $3.6 million in net reimbursements from its global expanded-access program and $0.2 million from OLPRUVA.

Zevra recorded net income of $19.2 million, or $0.26 per diluted share, compared with a net loss of $0.5 million, or $0.01 per share, in the third quarter of 2025. The results included a $5.5 million favorable non-cash mark-to-market credit related to outstanding common-stock warrants.

MIPLYFFA launch and patient identification

President and Chief Executive Officer Neil McFarlane said MIPLYFFA, the first approved therapy for NPC, received 13 prescription enrollment forms during the third quarter, bringing cumulative enrollment forms since launch to 197. The company estimates that about 900 people in the United States may be living with NPC, although only 300 to 350 had historically been diagnosed based on a 2021 study.

Management said it is increasingly seeing newly diagnosed patients and believes the addressable diagnosed population may be larger than its earlier estimates. Chief Commercial Officer Josh Schafer said third-quarter prescriptions included a mix of adult and pediatric patients, as well as a meaningful proportion of newly diagnosed patients.

“We’re seeing a pretty stable number of new patients coming in every quarter,” Schafer said. He added that analysis of medical claims data, along with increased disease awareness, suggests more patients may have NPC than previously estimated.

The company said it has modestly expanded its customer-facing commercial team and broadened outreach to community practices beyond centers of excellence. Schafer said the flexible commercial model is intended to allow Zevra to scale its field organization as it gains more information about the market.

Zevra reported that payer coverage for MIPLYFFA is currently 69%, while most remaining patients receive coverage through medical-exception pathways. The company said it continues to see high patient retention and refill rates.

Its disease-awareness efforts included the Learn NPC, Read Between the Signs campaign and collaborations with genetic-testing providers. Visits to the company’s disease-awareness website increased 123% sequentially, to nearly 56,000 in the third quarter from 25,000 in the second quarter, aided by the launch of an NPC Knowledge Hub for patients and caregivers.

Potential pediatric label expansion

Zevra is pursuing a potential supplemental new drug application to expand the MIPLYFFA label to include infants younger than 24 months. McFarlane said the company held a Type B meeting with the FDA after publishing data from an open-label pediatric substudy of arimoclomol used with miglustat in infants ages six months to less than 24 months.

The substudy evaluated safety and tolerability for up to 36 months and found arimoclomol was well tolerated, with no new safety signals observed, the company said. Based on FDA feedback, Zevra expects to file the sNDA in the first quarter of 2027.

Management said the younger-than-24-month population represents a relatively small number of additional patients—estimated at roughly 5% of the total addressable market—but viewed the potential expansion as meaningful for broadening access.

The company also highlighted MIPLYFFA’s intellectual-property position. McFarlane said patents listed in the FDA’s Orange Book extend to 2041, while U.S. orphan-drug exclusivity for NPC extends through 2031. Zevra is also pursuing a potential patent-term extension and now expects a decision from the U.S. Patent and Trademark Office in 2027.

International access and European review

Zevra’s global expanded-access program for arimoclomol enrolled 134 patients through compassionate-use and named-patient reimbursement programs. The company said it added an additional territory during the third quarter and continues to assess further opportunities to expand access internationally.

In Europe, the re-examination of Zevra’s marketing authorization application for arimoclomol is underway at the Committee for Medicinal Products for Human Use. A new rapporteur and co-rapporteur have been appointed, and an ad hoc expert group is expected to review the data. Zevra expects the CHMP’s opinion in the fourth quarter.

Celiprolol program and financial position

Zevra is also developing celiprolol for vascular Ehlers-Danlos syndrome, or vEDS. The Phase III DiSCOVER trial enrolled six patients during the quarter, reaching 72 total participants. The study’s interim analysis is triggered at 28 events; four confirmed events had occurred through the end of the third quarter.

The company held a second Type C meeting with the FDA regarding a possible submission strategy incorporating retrospective real-world data sets. Zevra is developing a statistical analysis plan and intends to seek a follow-up discussion with the agency in the first half of 2027, pending formal meeting minutes.

Operating expenses were $24.1 million in the third quarter, up $3.7 million from a year earlier. Research and development expense increased to $4.9 million, reflecting investments in arimoclomol and celiprolol, while selling, general and administrative expense rose to $19.1 million due to commercial and infrastructure costs.

As of Sept. 30, Zevra had $270.9 million in cash equivalents and investments, up approximately $10.7 million from June 30. Renz said the company does not anticipate meaningful increases in marketing spending next year following its modest commercial expansion.

About Zevra Therapeutics (NASDAQ:ZVRA)

Zevra Therapeutics, Inc is a biopharmaceutical company focused on developing and commercializing treatments for rare diseases and other conditions with significant unmet medical needs. The company combines traditional drug development with data science and patient-focused research to advance therapies for diseases that may have limited treatment options.

Zevra's lead commercial product is MIPLYFFA (arimoclomol), which is approved in the United States for the treatment of neurological manifestations of Niemann-Pick disease type C in adults and children aged 2 years and older, in combination with miglustat.

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