AMT-130: An Update

Annnnd we're off with uniQure N.V.

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REITERATE LONG
Ticker: QURE
Duration: +12 months
Target: +$55

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Consider this: your professor agrees to a test where you dictate all the terms. You supply the questions, you provide the answers you already know, and then you ask for a grade. That scenario mirrors the alignment call.

uniQure hosted a regulatory update call announcing significant progress for their Huntington's disease gene therapy candidate, AMT-130. Company executives reported alignment with the U.S. Food and Drug Administration (FDA) on crucial aspects of the Biologics License Application (BLA) for an accelerated approval pathway. This alignment, achieved after two Type B meetings, covers both the Chemistry, Manufacturing, and Controls (CMC) requirements and the primary statistical analysis plan. This news has bolstered uniQure's confidence in their development program and their ability to submit a BLA in the first quarter of 2026.

Regarding CMC, the FDA agreed that uniQure can leverage its experience and prior knowledge from its approved gene therapy, HEMGENIX, to validate the AMT-130 manufacturing process. This will be supplemented by additional full-scale AMT-130 GMP batches and a single process performance qualification (PPQ) batch. This accord is viewed as a strong endorsement of uniQure's manufacturing capabilities and is expected to streamline the process towards BLA submission. The FDA also agreed with uniQure's proposed drug product release testing plan, including the potency assay, pending final qualification.

For the statistical analysis, the FDA concurred that the primary efficacy analysis will focus on the 3-year change in the composite Unified Huntington's Disease Rating Scale (cUHDRS) in high-dose AMT-130 patients (again, uniQure ASKED for this). This will be compared against a propensity score-adjusted external control group derived from the Enroll-HD natural history database. The FDA re-confirmed cUHDRS as an acceptable intermediate clinical endpoint and noted that reductions in cerebrospinal fluid (CSF) neurofilament light chain (NFL) levels could serve as supportive evidence of therapeutic benefit. The use of Enroll-HD, a large, contemporaneous database, was specifically agreed upon.

The company outlined its next steps, including submitting an updated statistical analysis plan later in the current quarter, presenting top-line 3-year Phase I/II data in the third quarter of 2025, holding a pre-BLA meeting with the FDA in the fourth quarter of 2025, and ultimately submitting the BLA with a request for priority review in the first quarter of 2026. The team expressed gratitude towards the FDA for their constructive engagement and the Huntington's disease community for their support, highlighting the significant unmet medical need AMT-130 aims to address.

Throughout the call, uniQure emphasized the robustness of their approach. They believe the Enroll-HD database provides a strong comparator, and their propensity score methodology (weighted for primary analysis, matched for sensitivity) is well-established. By the way, THEY wanted this database to be used. The company also noted that prior, smaller data sets at the 2-year mark showed promising results, including statistically significant slowing of disease progression. Regarding 3 year data, here’s a quote from Dr. Abi-Saab (their CSO): “And with the robustness of the plan that we have, and I expect that the 3 years, this will continue to remain steady and actually become even more salient when we compare to natural history”. They are also advancing commercial readiness in a phased manner for a potential launch in 2026, focusing on medical education, market access, and treatment center identification.

Why AMT-130 is Positioned for Approval Shortly