uniQure announces 36-month Phase 1/2 data for AMT-130: 75% slowing of Huntington's disease progression in high-dose cohort

uniQure/AMT-130

uniQure announced positive 36-month data from its Phase 1/2 study of AMT-130 for Huntington's disease. Twelve patients in the high-dose cohort showed statistically significant 75% slowing of disease progression measured by the composite Unified Huntington's Disease Rating Scale (cUHDRS), with a 60% slowing measured by total functional capacity. The therapy was generally well-tolerated with a manageable safety profile.

• 36-month efficacy data shows 75% slowing of Huntington's disease progression in 12 high-dose patients.
• Total functional capacity declined 60% less than historical controls, indicating functional preservation.
• Study enrolled 29 total patients: 17 received high-dose AMT-130, 12 received low-dose.
• No serious adverse events related to treatment; side effects were generally mild.
• Supports progression to regulatory submissions planned for Q3 2026.