uniQure reports $100M HEMGENIX milestone; AMT-130 shows preserved function at 24 months in HD trial

uniQure N.V.

$100M milestone achieved from CSL Behring for first US commercial sale of HEMGENIX (hemophilia B gene therapy); payment due within 30 days.
AMT-130 well-tolerated; no treatment-emergent adverse events led to discontinuation. DSMB recommends continuing development.
Low-dose cohort showed preserved function at 24 months; TMS improved 1.8 points vs natural history, TFC 0.8, cUHDRS 0.9.
Mean CSF NfL 12.9% below baseline at 24 months in low-dose cohort vs predicted 22.9% increase in natural history.
Plan to complete EU enrollment in Q3 2023, start third US cohort in H2 2023, and hold regulatory interactions by Q1 2024.

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