UniQure Reports 75% Progression Slow in Huntington's Treatment Trial

uniQure has announced positive topline results from its pivotal Phase 1/2 trial of AMT-130, a gene therapy for Huntington's disease, demonstrating a statistically significant 75% slowing of disease progression after 36 months compared to external controls. The trial also met key secondary endpoints, including a 60% slowing of disease progression as measured by Total Functional Capacity, with favorable trends in motor and cognitive function tests, and an 8.2% reduction in a neurodegeneration biomarker. The company plans to submit a Biologics License Application to the FDA in the first quarter of 2026, aiming for a potential U.S. launch later that year, with AMT-130 having received Breakthrough Therapy and Regenerative Medicine Advanced Therapy designations. Following these results, uniQure's stock surged significantly, hitting a 52-week high and increasing over 150% in trading, reflecting strong investor optimism. Despite this clinical success, uniQure faces financial challenges, including negative margins, declining revenue, and a distress-level Altman Z-Score, though it has secured a $175 million non-dilutive senior secured term loan to improve financial flexibility for the potential commercial launch of AMT-130. Overall, the trial results mark a major advancement for Huntington's disease treatment and represent a promising, though financially complex, milestone for uniQure.