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    QURE
    Earnings call· Jun 2026(Q2 FY26)

    uniQure N.V. Q2 FY26 earnings call QURE

    Jul 29, 2026 Source

    Executive summary

    uniQure Q2 FY26 — Regulatory Clarity for AMT-130 and Strong Balance Sheet

    uniQure achieved significant regulatory clarity for its lead Huntington's disease program, AMT-130, with FDA alignment for an accelerated approval pathway BLA submission in Q3 FY26. The company also reported encouraging early clinical data for its epilepsy and Fabry disease programs, while maintaining a strong financial position with funding secured into 2030. Commercial preparations for AMT-130 are intensifying, focusing on treatment center readiness, community engagement, and market access.

    Highlights

    5

    • Received FDA alignment for BLA submission of AMT-130 under accelerated approval pathway based on 3-year data, with submission on track for Q3 FY26.

    • MHRA submission for AMT-130 also on track for Q3 FY26.

    • Early data from Phase I/IIa study of AMT-260 showed meaningful seizure reductions (79%-100%) in 3 of 6 low-dose patients.

    • Early data from Phase I/II study of AMT-191 showed dose-dependent elevations of alpha-Gal A activity in all 11 patients and stable plasma lyso-Gb3 levels.

    • Strengthened balance sheet with cash, cash equivalents, and investment securities totaling $810.3 million as of June 30, 2026, extending funding into 2030.

    Concerns

    2

    • Additional dosing in AMT-191 mid- and high-dose cohorts remains paused pending FDA agreement on monitoring and management plan following Grade 3 liver enzyme elevations in 2 patients.

    • Confirmatory study design for AMT-130 still being finalized with the FDA, including sample size and recruitment strategy.

    Guidance & targets

    7
    CategoryTargetConfidence
    AMT-130 BLA Submission
    On track for Q3 FY26
    high materiality
    High
    AMT-130 MHRA Submission
    On track for Q3 FY26
    high materiality
    High
    AMT-130 4-year Data Analysis
    Expected Q3 FY26
    medium materiality
    High
    AMT-130 4-year Data Results Presentation
    September
    high materiality
    High
    AMT-260 Updated Results
    Expected in the first half of 2027
    medium materiality
    Medium
    Cash Runway
    Into 2030
    high materiality
    High
    AMT-130 EMA Engagement
    Expected in 2027
    low materiality
    Medium

    Operational metrics

    12
    Cash, cash equivalents and investment securities
    $810.3Mvs $622.5M as of Dec 31, 2025
    Q2 FY26

    Total cash, cash equivalents and investment securities as of June 30, 2026.

    R&D expense decrease driver: facility expenses
    $1.5Mdecrease YoY
    Q2 FY26

    Decrease in facility expenses contributing to overall R&D expense reduction.

    R&D expense decrease driver: employee and contractor-related expenses
    $1.3Mdecrease YoY
    Q2 FY26

    Decrease in employee and contractor-related expenses, including share-based compensation, contributing to overall R&D expense reduction.

    R&D expense decrease driver: fair value of contingent consideration
    $1.0Mdecrease YoY
    Q2 FY26

    Decrease in the fair value of contingent consideration contributing to overall R&D expense reduction.

    R&D expense increase driver: information technology costs
    $0.6Mincrease YoY
    Q2 FY26

    Increase in information technology costs partially offsetting R&D expense reduction. (Note: Transcript stated $0.5M (sic) [ $0.6 million ])

    Direct R&D expenses
    higher spendYoY
    Q2 FY26

    Direct R&D expenses increased due to higher spend on specific programs, partially offset by lower spend on AMT-130.

    SG&A expense increase driver: employee and contractor-related expenses
    $4.3Mincrease YoY
    Q2 FY26

    Increase in employee and contractor-related expenses, including share-based compensation, mainly due to higher number of employees recruited in H2 2025 to support potential commercial launches of AMT-130.

    SG&A expense increase driver: intellectual property fees
    $0.7Mincrease YoY
    Q2 FY26

    Increase in intellectual property fees contributing to SG&A expense increase.

    SG&A expense increase driver: information technology costs and other expenses
    $0.7Mincrease YoY
    Q2 FY26

    Increase in information technology costs and other expenses contributing to SG&A expense increase.

    SG&A expense decrease offset: professional fees
    $1.8Mdecrease YoY
    Q2 FY26

    Decrease in professional fees, primarily due to lower costs incurred in support of potential commercial launches of AMT-130, partially offsetting SG&A expense increase.

    Enroll-HD database participants
    26,0006,000 additional
    Q2 FY26

    Total participants in the Enroll-HD database, used as a natural history control for AMT-130 analysis, updated with approximately 6,000 additional participants.

    AMT-191 patients withdrawn from ERT
    11
    Q2 FY26

    All 11 dosed patients in the AMT-191 Phase I/II study remained withdrawn from enzyme replacement therapy (ERT).

    Industry KPIs

    3
    MetricValueDetails
    Pipeline read out calendarMultiple key milestones
    Regulatory approvals filingsMultiple submissions planned
    Collaboration milestone royalty revenue$5.8MUSD

    Risks & headwinds

    4
    Confirmatory study design and enrollment for AMT-130Near-term (prior to BLA submission) and long-term (post-approval)

    FDA requested alignment on design prior to BLA submission, including consideration of a randomized standard of care controlled study with TFC at 36 months as primary endpoint. Confirmatory study should be well underway and potentially fully enrolled at accelerated approval.

    Mitigation: Working collaboratively with FDA to finalize design; committed to initiating global confirmatory trial as soon as possible; preparatory activities for global study underway; confident in ability to recruit and complete study in a timely manner by prioritizing US recruitment and then countries where drug is not commercially available.

    Dosing pause in AMT-191 Fabry disease programOngoing

    Additional dosing in mid- and high-dose cohorts remains paused following Grade 3 liver enzyme elevations reported in 2 patients from the mid-dose cohort.

    Mitigation: Pending agreement with the FDA on a monitoring and management plan; LFT elevations have resolved following immunosuppression.

    Potential for AdCom for AMT-130 BLADuring BLA review process

    Likely to have an AdCom meeting for the BLA submission, as it would be the first disease-modifying treatment for Huntington's disease.

    Mitigation: Company welcomes and is preparing for an AdCom; confident in the data and constructive interactions with the FDA.

    Dropout rate in long-term confirmatory studiesThroughout the confirmatory study duration

    Any long-term study will always have a risk of a dropout rate.

    Mitigation: Will be using statistical techniques and in agreement with the agency about how to deal with dropouts; patients randomized to standard of care will be eligible for AMT-130 after 3 years; patients with Huntington's disease are dedicated to being part of studies.

    What to watch in Q3 FY26

    5

    AMT-130 BLA Submission

    Q3 FY26
    CurrentOn track for Q3 FY26
    TargetSubmission completed

    Why it matters

    Successful submission is a critical step towards potential accelerated approval for Huntington's disease.

    We are on track to submit the BLA this quarter, and Walid will provide additional details later in the call.

    Q&A highlights

    6

    How quickly can the team operationalize the confirmatory study, given the FDA's requirement for it to be nearly fully enrolled at accelerated approval, and what is the likely patient enrollment number?

    Management believes the FDA's intent is for timely completion post-approval, not full enrollment at approval. They are confident in demonstrating this and have started preparatory activities for a global study. Recruitment will prioritize countries where the drug is not yet available. Sample size is still under discussion with the FDA, so it's premature to disclose.

    “So the idea is that most of the recruitment post approval will occur in countries before the drug becomes available in those countries such that we can complete the study on time.”

    asked by Debjit Chattopadhyay · answered by Walid Abi-Saab

    3 min read5 chapters

    Detailed Narrative

    01

    AMT-130 Regulatory Pathway in US and UK

    uniQure received FDA alignment in June 2026 that a BLA submission for AMT-130 under the accelerated approval pathway, based on 3-year data, is reasonable. The FDA requested alignment on the confirmatory study design prior to BLA submission, recommending a randomized standard of care controlled study with total functional capacity (TFC) at 36 months as the primary endpoint, and stated the study should be well underway or fully enrolled at accelerated approval. The company is on track for BLA submission in Q3 FY26 and MHRA submission in the UK in the same quarter.

    02

    AMT-130 4-Year Data Analysis and Confirmatory Study

    The AMT-130 clinical team is on track for 4-year data analysis from the Phase I/II studies, with a June 30 cutoff date, and expects to present results in September. The update will include safety and tolerability data through 4 years, and top-line data from 12 high and 12 low-dose patients at 4 years, plus an additional 3 high-dose patients with 3 years of follow-up (total 15 patients). Clinical data will include cUHDRS and TFC compared to a propensity score-matched natural history control from the Enroll-HD database, which has been updated with approximately 6,000 additional participants for a total of 26,000. CSF NfL change from baseline at 4 years will also be provided. The company is confident in operationalizing the global confirmatory study, prioritizing US recruitment initially and then focusing on countries where AMT-130 is not yet commercially available.

    03

    Progress in Broader Pipeline Programs

    Early data from the Phase I/IIa study of AMT-260 in refractory mesial temporal lobe epilepsy showed meaningful reductions in disabling seizures (79%-100%) in 3 of 6 low-dose patients during months 4-6, with a manageable safety profile. Enrollment in the second higher-dose cohort is expected to complete imminently, with updated results anticipated in H1 2027. For AMT-191 in Fabry disease, updated preliminary data showed dose-dependent elevations of alpha-Gal A activity in all 11 patients and stable plasma lyso-Gb3 levels, with all 11 patients remaining withdrawn from ERT. However, additional dosing in mid- and high-dose cohorts is paused pending FDA agreement on a monitoring plan following Grade 3 liver enzyme elevations in 2 patients, which have since resolved.

    04

    Commercial Readiness for AMT-130

    uniQure's commercial team is intensifying preparations for the potential launch of AMT-130, focusing on three key priorities: treatment center readiness, community engagement and education, and market access readiness. Deep engagement with Huntington's disease centers of excellence in the US and UK is ongoing to map institutional workflows and ensure readiness. Payer engagement is advancing, supported by health economics and outcomes research. Potential approval in the US or UK could also enable named patient and early access programs in other geographies like the Middle East, Latin America, and Central/Eastern Europe.

    05

    Financial Performance and Outlook

    Revenue for Q2 FY26 was $5.8 million, up from $5.3 million in Q2 FY25, driven by an increase in license revenue. R&D expenses decreased by $1.4 million to $34 million, primarily due to lower facility and employee-related expenses, partially offset by increased direct R&D spend on AMT-260, AMT-162, and AMT-191. SG&A expenses increased by $3.9 million to $17.4 million, mainly due to higher employee-related expenses for commercial launch support. Cash, cash equivalents, and investment securities totaled $810.3 million as of June 30, 2026, compared to $622.5 million at year-end 2025, providing funding into 2030.

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