uniQure Announces Positive Topline Results from Pivotal Phase I/II Study of AMT-130 in Patients with Huntington’s Disease

Core Insights - uniQure N.V. announced positive topline data from the pivotal Phase I/II study of AMT-130 for Huntington's disease, demonstrating statistically significant slowing of disease progression [1][2][3] Study Results - The pivotal study met its primary endpoint, showing a 75% slowing of disease progression at 36 months as measured by the composite Unified Huntington's Disease Rating Scale (cUHDRS) compared to a matched external control [3][4] - High-dose AMT-130 also achieved a statistically significant 60% slowing of disease progression as measured by Total Functional Capacity (TFC), meeting a key secondary endpoint [4][6] - Favorable trends were observed in additional clinical measures, including an 88% slowing of disease progression as measured by the Symbol Digit Modalities Test (SDMT) [4][6] Safety Profile - AMT-130 was generally well-tolerated with a manageable safety profile, and no new drug-related serious adverse events were reported since December 2022 [8][9] - The most common adverse events were related to the administration procedure, all of which resolved [8] Regulatory Plans - uniQure plans to submit a Biologics License Application (BLA) in the first quarter of 2026, with a potential U.S. launch later that year pending approval [1][9] - AMT-130 has received Breakthrough Therapy designation and Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA [9] Clinical Program Overview - The Phase I/II clinical program includes two multi-center, dose-escalating studies to explore the safety, tolerability, and exploratory efficacy signals of AMT-130 for Huntington's disease [11] - Data from the studies will be compared to a propensity score-matched external control derived from the Enroll-HD natural history dataset [11] Background on Huntington's Disease - Huntington's disease is a rare, inherited neurodegenerative disorder affecting approximately 75,000 people in the U.S., EU, and the UK, with no approved therapies currently available to slow its progression [15]