uniQure N.V. (NASDAQ:QURE) on Wednesday released topline data from the pivotal Phase 1/2 study of AMT-130 for Huntington’s disease.

Huntington’s disease is an inherited condition in which nerve cells in the brain break down over time. The disease affects a person’s movements, thinking ability and mental health.

The study met its prespecified primary endpoint, with high-dose AMT-130 demonstrating a statistically significant slowing of disease progression as measured by the composite Unified Huntington’s Disease Rating Scale (cUHDRS) at 36 months compared to a propensity score-matched external control.

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Data

Topline 36-month efficacy results for patients receiving high-dose AMT-130 (data cutoff as of June 30, 2025):

A statistically significant 75% slowing of disease progression as measured by cUHDRS (p=0.003), which met the primary endpoint of the study.

The mean change in cUHDRS from baseline ...