Clinical TrialJun 22, 2026, 06:56 AM
uniQure AMT-260 Phase I/IIa Trial Shows Early Efficacy Signals
AI Summary
uniQure N.V. announced preliminary six-month follow-up data from the first, low-dose cohort of its Phase I/IIa clinical trial for AMT-260, a gene therapy for refractory mesial temporal lobe epilepsy. Three of six patients achieved significant seizure reductions ranging from 79% to 100%, while the other three showed variable changes. The treatment was generally well-tolerated with no serious adverse events reported, and all adverse events were mild or moderate. The company is continuing enrollment for a higher dose cohort and expects to present updated results in the first half of 2027.
Key Highlights
- Preliminary data from Phase I/IIa trial of AMT-260 for refractory MTLE.
- 3 of 6 patients in low-dose cohort achieved 79-100% seizure reduction.
- Remaining 3 patients had variable changes (33% decrease to 36% increase).
- No serious adverse events related to AMT-260 or surgical procedure reported.
- All reported adverse events were mild or moderate; headache was most common (N=2).
- No immunosuppression was required for patients.
- Enrollment ongoing for a second, higher dose cohort (3x10^12 gc/mL).
- Updated results from the trial are expected in the first half of 2027.
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