Clinical TrialJun 19, 2026, 07:06 AM
uniQure AMT-260 Phase I/IIa shows early efficacy in MTLE
AI Summary
uniQure N.V. announced initial six-month follow-up data from its Phase I/IIa trial of AMT-260 for refractory mesial temporal lobe epilepsy (MTLE). Three of six patients in the low-dose cohort achieved significant reductions in disabling seizures, ranging from 79% to 100% decline from baseline. The investigational gene therapy was generally well-tolerated with no serious adverse events reported, and enrollment is continuing for a higher dose cohort, with updated results anticipated in the first half of 2027.
Key Highlights
- uniQure announced initial six-month follow-up data from its Phase I/IIa trial of AMT-260 for refractory MTLE.
- 3 of 6 patients in the low-dose cohort achieved 79% to 100% reduction in disabling seizures.
- No Serious Adverse Events (SAEs) related to AMT-260 or the surgical procedure were reported.
- All reported adverse events in the low-dose cohort were mild or moderate, with headache (N=2) being most common.
- Enrollment is ongoing in a second, higher dose cohort (3x10^12 gc/mL) of six patients.
- uniQure expects to present updated results from the Phase I/IIa clinical trial in the first half of 2027.
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