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    ZVRA
    Earnings call· Jun 2026(Q2 FY26)

    ZEVRA THERAPEUTICS Q2 FY26 earnings call ZVRA

    Aug 5, 2026 Source

    Executive summary

    Zevra Q2 FY26 — Strong MIPLYFFA Growth and Pipeline Advancement Amidst EMA Reexamination

    Zevra Therapeutics delivered strong Q2 FY26 results, driven by robust U.S. commercial performance of MIPLYFFA and continued pipeline progress for celiprolol. The company is actively pursuing a reexamination process with the CHMP for arimoclomol following a negative opinion, while maintaining patient access through its Expanded Access Program. Management emphasizes disciplined capital allocation to support growth and pipeline advancement.

    Highlights

    5

    • Total net revenue increased by 53% year-over-year to $39.7 million in Q2 FY26.

    • MIPLYFFA net U.S. revenue grew 40% year-over-year and 23% quarter-over-quarter to $30.2 million.

    • Strengthened MIPLYFFA intellectual property with an Orange Book listing through 2041.

    • Ended the quarter with a robust cash, cash equivalents, and investments balance of $260.2 million, an increase of $23.4 million QoQ.

    • Celiprolol Phase III DISCOVER trial enrolled 66 patients, with 3 confirmed events towards interim analysis.

    Concerns

    2

    • CHMP issued a negative opinion regarding the arimoclomol MAA, leading to a reexamination process expected to take 120 days.

    • Global EAP reimbursements for arimoclomol, excluding the French program, remain highly variable and unpredictable.

    Guidance & targets

    2
    CategoryTargetConfidence
    arimoclomol MAA Reexamination Opinion
    Expected in Q4
    high materiality
    Medium
    French EAP Net Reimbursements
    Approximately $2.5 million net per quarter
    medium materiality
    High

    Segment performance

    1
    SegmentRevenueYoYQoQMargin
    MIPLYFFA (U.S.)
    Reflects continued momentum in reaching patients and establishing MIPLYFFA as a foundational treatment for NPC. Enrollments include newly and previously diagnosed patients across centers of excellence and community practices.
    Prescription enrollment forms (total): 184Prescription enrollment forms (Q2): 14Prescription enrollment forms (H1 2026): 23Payer coverage: 69% of covered livesPatient mix (adult vs pediatric): 50-50
    		$30.2 million40%23%—

    Operational metrics

    20
    Total Net Revenue Growth
    53%YoY
    Q2 2026

    Driven by MIPLYFFA net U.S. revenue.

    Operating Expenses
    $21.0 milliondecreased $3.2 million YoY
    Q2 2026

    Decrease primarily due to professional fees and third-party spending, partially offset by increased personnel costs.

    R&D Expense
    $4.5 millionincreased $1.1 million YoY
    Q2 2026

    Primarily due to ongoing arimoclomol efforts.

    SG&A Expense
    $16.6 milliondecreased $4.2 million YoY
    Q2 2026

    Primarily due to a decrease in professional fees and third-party spending, partially offset by an increase in personnel-related costs.

    Income Tax Provision
    $4.0 million
    Q2 2026
    Adjusted Net Income
    $15.2 million
    Q2 2026

    This figure provides a more comparable view of period performance.

    Adjusted Net Loss
    $2.4 million
    Q2 2025

    This figure provides a more comparable view of prior period performance.

    Cash, Cash Equivalents & Investments
    $260.2 millionincreased $23.4 million compared to March 31, 2026
    as of June 30, 2026

    Provides flexibility to invest in commercial execution, pipeline, and growth opportunities.

    Global EAP Net Reimbursements
    $9.0 million
    Q2 2026

    Includes reimbursements from the global Expanded Access Program for arimoclomol.

    French EAP Net Reimbursements
    $2.5 millionconsistent
    per quarter

    This program has been ongoing for a multitude of years and remains very consistent.

    Commave Royalty Revenue
    $0.3 million
    Q2 2026

    Past due royalty revenue.

    MIPLYFFA Prescription Enrollment Forms
    184
    as of June 30, 2026

    Total forms received since launch.

    MIPLYFFA Prescription Enrollment Forms
    14
    Q2 2026

    Forms received in the second quarter, including patients from Puerto Rico.

    MIPLYFFA Prescription Enrollment Forms
    23
    H1 2026

    Total forms received in the first six months of the year.

    MIPLYFFA Prescription Enrollment Forms
    52
    FY 2025

    Total forms generated for the full year 2025.

    arimoclomol Expanded Access Program Patients
    13210 added in Q2
    as of June 30, 2026

    Comprised of compassionate use and reimbursed named patient programs.

    Niemann-Pick Disease Type C (NPC) Estimated Prevalence
    900
    current

    Supports confidence in the estimated total addressable market.

    MIPLYFFA Payer Coverage
    69%consistent with expectations
    Q2 2026

    Maintained strong access for patients.

    MIPLYFFA Patient Mix
    50-50
    current

    Reflects a diversity of instances and age groups, reinforcing the heterogeneity of the disease.

    Channel Inventory
    returned to target levelscompared to Q1 when it fell below the low end of our range
    Q2 2026

    Normalization expected going forward.

    Industry KPIs

    1
    MetricValueDetails
    Business development capacity deal appetiterobust cash position of $260.2 millionUSD

    Risks & headwinds

    2
    Negative CHMP opinion for arimoclomol MAAQ4 (expected reexamination opinion)

    negative opinion

    Mitigation: advancing the reexamination process, which will provide the opportunity to address the specific grounds cited... and have our comprehensive evidence package reviewed by a new rapporteur and co-rapporteur.

    Variability in global EAP reimbursements (excluding French EAP)first few years until the patient base has stabilized

    highly variable and unpredictable

    Mitigation: evaluating opportunities to expand access to MIPLYFFA through the global EAP in response to strong interest from additional countries.

    What to watch in Q3 FY26

    5

    CHMP opinion on arimoclomol MAA reexamination

    Q4
    CurrentNegative opinion issued, reexamination process initiated
    TargetPositive opinion or further clarity on path forward

    Why it matters

    Regulatory approval in Europe would significantly expand market access for arimoclomol and validate the comprehensive evidence package.

    This process typically takes 120 days from the submission of a reexamination to the opinion from the CHMP, which is expected in Q4.

    Q&A highlights

    5

    How will Zevra leverage its strong cash position, and what are the details/implications of the EMA reexamination process with new rapporteurs?

    Management reiterated its disciplined capital allocation strategy focused on MIPLYFFA growth, global expansion, celiprolol advancement, and external opportunities. For EMA, Neil McFarlane stated confidence in the comprehensive evidence and the opportunity for new expert voices to interpret the data, but could not comment on specific process details.

    “our confidence is grounded in the totality of the evidence and the clear unmet patient need.”

    asked by Kristen Kluska · answered by Neil McFarlane

    1 min read5 chapters

    Detailed Narrative

    01

    MIPLYFFA Commercial Strategy

    Zevra's commercial strategy for MIPLYFFA focuses on accelerating diagnosis and treatment, driving demand, and facilitating access. Initiatives include the Learn NPC disease awareness campaign, genetic testing collaborations, an AI-driven targeting model, the Expert Connect program for healthcare professionals, and the NPC Knowledge Hub for patients and caregivers. Payer coverage remained at 69% of covered lives, with ongoing engagement with payers and participation in medical congresses.

    02

    Arimoclomol Regulatory Process

    Following a negative opinion from the CHMP for the arimoclomol MAA, Zevra is advancing a reexamination process. This process allows for a review by new rapporteurs and co-rapporteurs, focusing on specific grounds cited for the refusal. The company maintains confidence in its comprehensive evidence package, including long-term open-label extension data, EAP findings, and pediatric substudy results. Access for eligible patients continues through the global Expanded Access Program.

    03

    Celiprolol Clinical Development

    The Phase III DISCOVER trial for celiprolol in Vascular Ehlers-Danlos Syndrome (VEDS) is ongoing, with 66 patients enrolled and 3 confirmed events towards the 28 required for interim analysis. Zevra is implementing strategies to accelerate enrollment, including expanding genetic testing centers and re-engaging with the FDA in H2 FY26 to explore clinical development options.

    04

    Intellectual Property

    Zevra strengthened MIPLYFFA's U.S. intellectual property with an Orange Book listing providing protection through 2041. The company is awaiting determination for a separately requested patent term extension, which would fall within this coverage period, aiming to maximize the long-term commercial opportunity.

    05

    Capital Allocation Philosophy

    Zevra maintains a robust cash position of over $260 million, providing flexibility to invest in commercial execution, pipeline advancement, and future growth opportunities. The company emphasizes a disciplined capital allocation strategy, focusing on value-creating opportunities and is not currently pursuing share buybacks.

    AI-generated summary of the company’s earnings call. Not investment advice.