Zevra Reports Third Quarter 2025 Financial Results and Corporate Update

2025 EPS of $(0.01)

Q3 2025 net revenue of $26.1 million, driven by MIPLYFFA® net revenue of $22.4 million

Company to host conference call and webcast TODAY, November 5, 2025, at 4:30 p.m. ET

CELEBRATION, Fla., Nov. 05, 2025 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a commercial-stage company focused on providing therapies for people living with rare disease, today reported its financial results for the three and nine months ended September 30, 2025.

"Zevra is well-positioned for continued growth, driven by the strong performance of MIPLYFFA and the meaningful impact we are delivering to patients with Niemann-Pick disease type C," said Neil F. McFarlane, Zevra's President and Chief Executive Officer. "We have generated strong revenue and a solid operating runway that enables us to execute on our strategic priorities and invest in building Zevra into a leading rare disease company."

Commercial Highlights

• MIPLYFFA: Eight prescription enrollment forms during Q3 2025, bringing the total since product launch to 137. Market access has reached 66% of covered lives, which is in line with the Company's expectations one year into the launch.
• OLPRUVA: One prescription enrollment form during Q3 2025, bringing the total since product launch to 30. Market access has reached 81% of covered lives. As a data-driven organization, the Company has decided to scale back its sales and marketing efforts for OLPRUVA. While options for OLPRUVA are evaluated, Zevra will maintain product availability and support services for patients.

Pipeline and Innovation Highlights

• Arimoclomol: A Marketing Authorisation Application for the evaluation of arimoclomol for the treatment of Niemann-Pick disease type C (NPC) has been validated and is under review by the European Medicines Agency (EMA). It has been designated an Orphan Medicinal Product by the EMA. Arimoclomol is currently marketed in the U.S. under the brand name, MIPLYFFA.
• Celiprolol: Enrolled five patients in the event-driven Phase 3 DiSCOVER trial for the treatment of Vascular Ehlers-Danlos Syndrome during Q3 2025, bringing the total number of enrolled patients to 44, and there has been one confirmed event.

Publication Highlights

• Open-label extension data showing sustained long-term efficacy of MIPLYFFA for the treatment of NPC was published in the peer-reviewed journal of Molecular Genetics and Metabolism.
• MIPLYFFA data featured at the following conferences: National Niemann-Pick Disease Foundation (NNPDF) Conference, Southeastern Regional Genetics Group (SERGG), International Congress of Inborn Errors of Metabolism (ICIEM), International Niemann-Pick Disease Alliance (INPDA) Meeting, and Child Neurology Society (CNS) Conference.
  • New data from a pre-specified analysis showed that patients on concomitant miglustat treatment who were on placebo in the Phase 2/3 pivotal, double-blind study and switched to MIPLYFFA in the open-label extension phase experienced a decline in annual disease progression.
  • New data from a multi-center pediatric substudy in patients younger than two years old showed that MIPLYFFA was well tolerated in this age group with no new safety signals observed.
  • Nomination for Best Poster Award received for poster highlighting MIPLYFFA’s differentiated mechanism of action targeting the underlying pathology of NPC.

Q3 2025 Financial Highlights

• Revenue, Net: $26.1 million for Q3 2025, which includes $22.4 million of MIPLYFFA net revenue, $0.1 million of OLPRUVA net revenue, $2.4 million in net reimbursements from the French EAP for arimoclomol, and $1.2 million in royalties and other reimbursements under the AZSTARYS® license agreement. For Q3 2024, total net revenue was $3.7 million. MIPLYFFA net revenue in Q3 was impacted by the redesign of Medicare Part D rebates, resulting in a gross-to-net adjustment of $1.2 million.
• Cost of Product Revenue: $1.2 million for Q3 2025, excluding non-cash intangible asset amortization. Cost of product revenue for Q3 2024 was $2.3 million.
• Operating Expenses: $20.4 million for Q3 2025, which includes non-cash stock compensation expense of $2.8 million. Total operating expenses for Q3 2024 were $27.2 million.
  • R&D expense was $3.4 million for Q3 2025, which was a decrease of $7.5 million compared to $10.9 million for Q3 2024 due primarily to a decrease in third-party costs upon completion of the KP1077 Phase 2 trial, combined with a decrease in personnel-related costs.
  • SG&A expense was $16.9 million for Q3 2025, which was an increase of $0.7 million compared to $16.2 million for Q3 2024. Period-over-period increase was primarily related to an increase in personnel-related costs, professional fees, and other expenses associated with our commercial, medical and launch activities. 
• Net Loss: $(0.5) million, or $(0.01) per basic and diluted share for Q3 2025, compared to a net loss of $(33.2) million, or $(0.69) per basic and diluted share, in Q3 2024. Q3 2025 net loss includes non-cash warrant fair value adjustment expense of $5.5 million, non-cash stock-based compensation expense of $2.8 million, and non-cash intangible asset amortization expense of $0.3 million.
• Cash Position: Cash, cash equivalents and securities were $230.4 million as of September 30, 2025, which included $6.0 million in cash received in exchange for 1,046,890 warrants exercised at a weighted average exercise price of $5.69 per share, and $0.6 million in cash received in exchange for 86,628 options exercised at a weighted average exercise price of $6.62, during the three months ended September 30, 2025. Based on its current operating forecast, the Company believes available financial resources are sufficient to execute on its strategic priorities independent from the capital markets.
• Common and Fully Diluted Shares O/S: As of September 30, 2025, total shares of common stock outstanding were 56,217,722, and fully diluted common shares were 67,882,914, which included 7,228,555 issuable from outstanding awards under equity incentive plans, and 4,436,637 shares issuable upon exercise of warrants. 

Inducement Awards

The Company granted options to purchase an aggregate of 93,000 shares of the Company’s common stock (the “Inducement Awards”) to 8 new employees pursuant to the Company’s 2023 Employment Inducement Award Plan (as amended and/or restated, the “Inducement Award Plan”). Each Inducement Award vests over four years, with 25% vesting on the first anniversary of the employee’s start date, and the remainder vesting in three equal annual installments thereafter (subject to each such employee’s continued employment on each vesting date).

Each Inducement Award was approved by the Compensation Committee of the Board of Directors and granted as an inducement material to the individual entering into employment with Zevra, in accordance with Nasdaq Listing Rule 5635(c)(4). The Inducement Award Plan is used exclusively for the grant of equity awards to individuals who were not previously employees of Zevra, or following a bona fide period of non-employment, as an inducement material to such individuals entering into employment with Zevra.

Conference Call Information

Zevra will host a conference call and audio webcast TODAY at 4:30 p.m. ET to discuss its corporate update and financial results for the third quarter 2025. 

A link to the audio webcast is accessible on the “Events & Presentations” page in the Investor Relations section of the Zevra's website at investors.zevra.com. A replay of the webcast will be available for 90 days beginning at approximately 5:30 p.m. ET on November 5, 2025. 

Additionally, interested participants and investors may access the conference call by dialing either:

• (800) 579-2543 (United States)
• +1 (785) 424-1789 (International)
• Conference ID: ZVRAQ325

About MIPLYFFA® (arimoclomol)

MIPLYFFA (arimoclomol) is Zevra’s approved therapy for the treatment of Niemann-Pick disease type C (NPC). Approved by the U.S. Food and Drug Administration on Sep. 20, 2024, MIPLYFFA (arimoclomol) increases the activation of the transcription factors EB (TFEB) and E3 (TFE3) resulting in the upregulation of coordinated lysosomal expression and regulation (CLEAR) genes. MIPLYFFA has also been shown to reduce unesterified cholesterol in the lysosomes of human NPC fibroblasts. The clinical significance of these findings is not fully understood. In the pivotal phase 3 trial, MIPLYFFA halted disease progression compared to placebo over the one-year duration of the trial when measured by the only validated disease progression measurement tool, the NPC Clinical Severity Scale. MIPLYFFA has also received Orphan Medicinal Product designation by the European Medicines Agency (EMA) for the treatment of NPC. The extensive data generated for MIPLYFFA has shown long-term, meaningful clinical outcomes with 5 and in some patients 7 years of patient experience across more than 270 NPC patients worldwide through a Phase 2/3 clinical trial, Open-Label Extension (OLE) study, Expanded Access Programs (EAP), and a pediatric sub-study, which is the most expansive clinical development program in NPC to date. Zevra has submitted a Marketing Authorization Application to the European Medicines Agency for the evaluation of arimoclomol for the treatment of Niemann-Pick disease type C.

INDICATIONS AND USAGE

MIPLYFFA is indicated for use in combination with miglustat for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adult and pediatric patients 2 years of age and older.

IMPORTANT SAFETY INFORMATION

Hypersensitivity Reactions:

Hypersensitivity reactions such as urticaria and angioedema have been reported in patients treated with MIPLYFFA during Trial 1: two patients reported both urticaria and angioedema (6%) and one patient (3%) experienced urticaria alone within the first two months of treatment. Discontinue MIPLYFFA in patients who develop severe hypersensitivity reactions. If a mild or moderate hypersensitivity reaction occurs, stop MIPLYFFA and treat promptly. Monitor the patient until signs and symptoms resolve.

Embryofetal Toxicity:

MIPLYFFA may cause embryofetal harm when administered during pregnancy based on findings from animal reproduction studies. Advise pregnant females of the potential risk to the fetus and consider pregnancy planning and prevention for females of reproductive potential.

Increased Creatinine without Affecting Glomerular Function:

Across clinical trials of MIPLYFFA, mean increases in serum creatinine of 10% to 20% compared to baseline were reported. These increases occurred mostly in the first month of MIPLYFFA treatment and were not associated with changes in glomerular function.

During MIPLYFFA treatment, use alternative measures that are not based on creatinine to assess renal function. Increases in creatinine reversed upon MIPLYFFA discontinuation.

The most common adverse reactions in Trial 1 (≥15%) in MIPLYFFA-treated patients who also received miglustat were upper respiratory tract infection, diarrhea, and decreased weight.

Three (6%) of the MIPLYFFA-treated patients had the following adverse reactions that led to withdrawal from Trial 1: increased serum creatinine (one patient), and progressive urticaria and angioedema (two patients). Serious adverse reactions reported in MIPLYFFA-treated patients were hypersensitivity reactions including urticaria and angioedema.

To report SUSPECTED ADVERSE REACTIONS, contact Zevra Therapeutics, Inc. at toll-free phone 1-844-600-2237 or FDA at 1 800-FDA-1088 or www.fda.gov/medwatch.Drug Interaction(s):

Arimoclomol is an inhibitor of the organic cationic transporter 2 (OCT2) transporter and may increase the exposure of drugs that are OCT2 substrates. When MIPLYFFA is used concomitantly with OCT2 substrates, monitor for adverse reactions and reduce the dosage of the OCT2 substrate.

Use in Females and Males of Reproductive Potential:

Based on animal findings, MIPLYFFA may impair fertility and may increase post-implantation loss and reduce maternal, placental, and fetal weights.

Renal Impairment:

The recommended dosage of MIPLYFFA, in combination with miglustat, in patients with an eGFR ≥15 mL/minute to