uniQure (NASDAQ: QURE) highlights AMT-130 75% slowing at 36 months; FDA follow-up set

uniQure (NASDAQ: QURE) reported Q3 2025 results and program updates on Nov 10, 2025. Key clinical highlights include AMT-130 Phase I/II topline 36-month data showing a 75% slowing in disease progression on the cUHDRS (p=0.003) and 60% slowing on TFC (p=0.033) versus an external control; safety remained manageable through 36 months. The company said preliminary FDA pre-BLA feedback introduced uncertainty about using Phase I/II versus external control as primary evidence and plans urgent follow-up.

Other updates: AMT-260 and AMT-191 have additional data expected H1 2026; AMT-162 enrollment paused after a dose-limiting toxicity. Financially, uniQure raised ~$323.7M net proceeds and held $694.2M in cash and investments as of Sept 30, 2025.

https://www.stocktitan.net/news/QURE/uni-qure-announces-third-quarter-2025-financial-results-and-provides-5s4fxvl5fi2r.html