uniQure N.V. (NASDAQ:QURE) stock surged 150% Wednesday following the announcement of encouraging topline results from its pivotal Phase I/II study of AMT-130 in patients with Huntington’s disease, showing substantial slowing of disease progression compared to controls.
The trial successfully hit its primary endpoint, with high-dose AMT-130 demonstrating a 75% reduction in disease progression at 36 months as measured by the composite Unified Huntington’s Disease Rating Scale (cUHDRS) against a propensity score-matched external control. A key secondary endpoint was also achieved, showing a 60% slowing of disease progression on Total Functional Capacity.
Additional clinical measures reflected positive trends, including an 88% reduction in decline on the Symbol Digit Modalities Test and a 113% reduction in the Stroop Word Reading Test. Mean cerebrospinal fluid neurofilament light protein levels, a biomarker for neurodegeneration, were 8.2% below baseline at 36 months.
The therapy was generally well-tolerated, with a manageable safety profile and no new serious drug-related adverse events since December 2022. Most adverse events were related to the administration procedure.
Sarah Tabrizi, professor of clinical neurology and director of the University College London Huntington’s Disease Center, commented: “I am thrilled that this pivotal study of AMT-130 showed statistically significant effects on both cUHDRS and TFC at 36 months, supported by mean CSF NfL remaining below baseline.”
uniQure plans to file a Biologics License Application (BLA) in the first quarter of 2026, with a U.S. launch expected later that year, subject to FDA approval. The treatment has already been granted Breakthrough Therapy designation and Regenerative Medicine Advanced Therapy designation by the FDA.
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