QURE Stock Skyrockets on Meeting Goals in Huntington's Disease Study

Core Insights - Shares of uniQure N.V. (QURE) surged 247.7% following the successful results of its pivotal study for AMT-130, a gene therapy for Huntington's disease [1][6] Study Results - The pivotal phase I/II study met its primary endpoint, showing a statistically significant 75% slowing of disease progression on the composite Unified Huntington's Disease Rating Scale (cUHDRS) at 36 months for the high dose of AMT-130 [3][6] - The study also achieved a key secondary endpoint with a 60% slowing of disease progression as measured by Total Functional Capacity (TFC) at 36 months [5][6] - Patients receiving the high dose of AMT-130 demonstrated an 88% slowing on the Symbol Digit Modalities Test, a 113% slowing on the Stroop Word Reading Test, and a 59% slowing on the Total Motor Score compared to external controls [7] Biomarker and Safety Profile - The study indicated a decrease of 8.2% in cerebrospinal neurofilament light protein, a biomarker associated with neurodegeneration in Huntington's disease [8] - AMT-130 was reported to have a manageable safety profile, with adverse events primarily mild in severity [9] Future Plans - Based on the positive data, uniQure N.V. plans to engage with the FDA to submit a biologics license application for AMT-130, targeting the first quarter of 2026 [10] Clinical Pipeline - In addition to AMT-130, uniQure N.V. has other candidates in early to mid-stage development for conditions such as refractory mesial temporal lobe epilepsy, amyotrophic lateral sclerosis, and Fabry disease [14]