uniQure announces positive interim Phase I/II data for AMT-130: 80% slowing of Huntington's progression at high dose

uniQure N.V.

High-dose AMT-130 showed 80% slowing of cUHDRS decline vs. external control at 24 months (p=0.007).
CSF neurofilament light chain reduced 11% from baseline at 24 months in treated patients (p=0.02).
AMT-130 received first-ever RMAT designation for Huntington's disease from FDA.
Company expects to meet with FDA in H2 2024 to discuss expedited development and accelerated approval.
No new AMT-130-related serious adverse events; safety profile manageable at both doses.

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