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    Does uniQure (QURE) FDA Feedback on AMT-130 Reframe Its Risk, Funding and Strategy Profile?

    Simply Wall St·12/18/2025 01:34:42
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    • In early December 2025, uniQure reported that final FDA meeting minutes indicated Phase I/II data for its Huntington's gene therapy AMT-130 are currently unlikely to provide the primary evidence needed for a Biologics License Application, prompting the company to seek a follow-up meeting in early 2026.
    • This feedback raises questions about trial design, endpoint robustness, and the potential need for additional studies, sharpening focus on uniQure’s regulatory pathway and funding capacity for AMT-130.
    • We’ll now examine how the FDA’s view that current AMT-130 data are insufficient reshapes uniQure’s investment narrative and risk profile.

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    uniQure Investment Narrative Recap

    To own uniQure today, you have to believe AMT-130 can still become a viable Huntington’s treatment despite the FDA now viewing current Phase I/II data as insufficient for a BLA. This news directly affects the key near term catalyst, shifting focus from a potential 2026 filing to whether uniQure can agree on an acceptable path with regulators before cash needs or program delays become the dominant risk.

    The most relevant prior announcement is the September 24, 2025 topline readout, where high dose AMT-130 showed a slowing of disease progression versus external controls. That signal had underpinned expectations for an accelerated U.S. pathway, so the FDA’s more cautious stance now forces investors to reassess how much weight to place on those data as a value driver and how much additional clinical or analytical work might sit between here and any commercial opportunity.

    Yet beneath the promise of gene therapy progress, investors should be aware of the company’s reliance on successful AMT-130 regulatory outcomes and...

    Read the full narrative on uniQure (it's free!)

    uniQure’s narrative projects $306.4 million revenue and $32.3 million earnings by 2028. This requires 147.5% yearly revenue growth and a $249.9 million earnings increase from $-217.6 million today.

    Uncover how uniQure's forecasts yield a $55.44 fair value, a 141% upside to its current price.

    Exploring Other Perspectives

    QURE 1-Year Stock Price Chart
    QURE 1-Year Stock Price Chart

    Six members of the Simply Wall St Community have fair value estimates for uniQure ranging from US$13.55 to US$344.18, showing how far apart views can be. You can set those against the current concern that the FDA may require more robust AMT-130 evidence before any BLA, which could have wide implications for timing and funding of the whole story.

    Explore 6 other fair value estimates on uniQure - why the stock might be a potential multi-bagger!

    Build Your Own uniQure Narrative

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    This article by Simply Wall St is general in nature. We provide commentary based on historical data and analyst forecasts only using an unbiased methodology and our articles are not intended to be financial advice. It does not constitute a recommendation to buy or sell any stock, and does not take account of your objectives, or your financial situation. We aim to bring you long-term focused analysis driven by fundamental data. Note that our analysis may not factor in the latest price-sensitive company announcements or qualitative material. Simply Wall St has no position in any stocks mentioned.