uniQure N.V.

uniQure N.V. is a gene therapy company focused on developing single treatments for rare and other devastating diseases with potentially curative results. The company is advancing a pipeline of gene therapies, including clinical candidates for Huntington's disease, refractory mesial temporal lobe epilepsy (MTLE), Fabry disease, and amyotrophic lateral sclerosis caused by mutations in superoxide dismutase 1 (SOD1-ALS). In September 2025, uniQure N.V. announced positive topline data from the pivotal Phase I/II study of AMT-130 for Huntington's disease. The study analyzed clinical outcomes for 29 patients treated with AMT-130 (17 high-dose; 12 low-dose). At the 36-month follow-up, 12 patients per dose group were evaluated. Outcomes for each dose group were compared to a propensity score-matched external control from the Enroll-HD natural history data set (940 for high-dose; 626 for low-dose). As of June 30, 2025, 36-month efficacy results for high-dose AMT-130 showed a statistically significant 75% slowing of disease progression as measured by composite Unified Huntington's Disease Rating Scale (cUHDRS) (p=0.003), meeting the primary endpoint. Treated patients had a mean change in cUHDRS from baseline of -0.38, compared to -1.52 for the external control. Additionally, there was a statistically significant 60% slowing of disease progression as measured by Total Function Capacity (TFC) (p=0.033), meeting a key secondary endpoint. Treated patients had a mean change in TFC from baseline of -0.36, compared to -0.88 for the external control. Favorable trends were also observed in other secondary endpoint measures of motor and cognitive function, including Symbol Digit Modalities Test (SDMT), Stroop Word Reading Test (SWRT), and Total Motor Score (TMS).