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Investing.com -- uniQure N.V. (NASDAQ:QURE) stock surged 150% Wednesday after the gene therapy company announced positive topline results from its pivotal Phase I/II study of AMT-130 in patients with Huntington’s disease, showing significant disease slowing compared to controls.
The study met its primary endpoint with high-dose AMT-130 demonstrating a statistically significant 75% disease slowing at 36 months as measured by the composite Unified Huntington’s Disease Rating Scale (cUHDRS) compared to a propensity score-matched external control. The treatment also achieved a key secondary endpoint with a statistically significant 60% slowing of disease progression measured by Total Functional Capacity.
High-dose AMT-130 showed favorable trends across additional clinical measures, including an 88% slowing in Symbol Digit Modalities Test and a 113% slowing in Stroop Word Reading Test. Mean cerebrospinal fluid neurofilament light protein levels, a biomarker of neurodegeneration, were 8.2% below baseline at 36 months.
The company reported that AMT-130 continued to be generally well-tolerated with a manageable safety profile. No new drug-related serious adverse events have been observed since December 2022, with the most common adverse events related to the administration procedure.
"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," stated Sarah Tabrizi, professor of clinical neurology and director of the University College London Huntington’s Disease Center.
uniQure plans to submit a Biologics License Application (BLA) in the first quarter of 2026, with an anticipated U.S. launch later that year, pending approval. The treatment has already received Breakthrough Therapy designation and Regenerative Medicine Advanced Therapy designation from the FDA.
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