uniQure AMT-130 Phase I/II meets primary endpoint: 75% HD progression slowing at 36 months

uniQure N.V.

High-dose AMT-130 showed 75% slowing on cUHDRS (p=0.003) vs external control; key secondary TFC endpoint also met (60% slowing, p=0.033).
Mean CSF NfL reduced 8.2% from baseline at 36 months, supporting potential disease modification.
No new drug-related serious adverse events since Dec 2022; manageable safety profile.
Company plans pre-BLA meeting with FDA later 2025, BLA submission targeted for Q1 2026.

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