Clinical TrialSep 29, 2026, 09:11 AM
uniQure: AMT-130 Shows 80% cUHDRS Slowing at 36 Months in Huntington's Trial
AI Summary
uniQure announced additional data from its Phase I/II studies of ifezuntirgene inilparvovec (AMT-130) for Huntington's disease. The 48-month data showed continued slowing of disease progression, with TFC demonstrating 61% slowing (p=0.008), though cUHDRS did not reach statistical significance (p=0.144). The 36-month analysis, including more patients, showed substantial treatment effects with 80% slowing in cUHDRS (p=0.005) and 67% in TFC (p=0.011). The drug was generally well-tolerated, with manageable safety.
Key Highlights
- At 48 months, high-dose AMT-130 showed 44% slowing of disease progression in cUHDRS (p=0.144) and 61% slowing in TFC (p=0.008) compared to external control.
- At 36 months, high-dose AMT-130 showed 80% slowing of disease progression in cUHDRS (p=0.005) and 67% slowing in TFC (p=0.011) compared to external control.
- Post hoc analysis at 48 months using prior external control showed 54% slowing in cUHDRS (p=0.041) and 68% slowing in TFC (p=0.001).
- Mean CSF NfL was 4% above baseline at 48 months for high-dose patients.
- AMT-130 was generally well-tolerated; most common AEs were procedure-related.
- One treatment-related SAE of CNS inflammation in a Cohort 4 patient resolved.
- One suicide in a low-dose patient occurred ~5 years post-treatment, assessed as unrelated.
Price Impact
More from QURE