Uniqure N.v. · NASDAQ:QURE
Uniqure — Epilepsy Pipeline Conference June 2026
6 slides from Uniqure's conference presentation, Jun 1, 2026
Slide 1: GenTLE Phase 1/2a Clinical Study of AMT-260: Preliminary Results
GenTLE Phase 1/2a Clinical Study of AMT-260: Preliminary Results of an Investigational Gene Therapy for Refractory MTLE Firas Taha, MD Medical Director, Clinical Development, uniQure biopharma B.V. AMT-260 is an investigational agent currently being studied in the treatment of mesial temporal lobe e
Slide 2: Background
Background AMT-260 • Mesial temporal lobe epilepsy (MTLE) is the most common form of focal epilepsy in adults1 hSyn1 two GRIK2 - miRNA RGB polyA • AMT-260 is a gene therapy candidate under RNAi investigation intended to reduce or eliminate GRIK2 mRNA → degradation seizures in people with refractory
Slide 3: Study Objective and Design
Study Objective and Design Objective: To evaluate the safety, tolerability, and exploratory signs of efficacy of AMT-260 in adults with unilateral refractory MTLE and a Phase 1/2a baseline mean of ≥2 focal impaired awareness seizures per month clinical trial NCT06063850 Treatment & evaluation period
Slide 4: Results: Primary Endpoints for Cohort 1
Results: Primary Endpoints for Cohort 1 Baseline Characteristics Safety Outcomes* Characteristics Cohort 1: Low • AEs were reported in 4 of 6 dose (n=6) participants; 16 were Grade 1 Age (years), median (range) 52 (36, 61) and 5 were Grade 2 severity, with Male, n (%) 4 (67) headache most common (n=
Slide 5: Results: Secondary Endpoint for Cohort 1
Results: Secondary Endpoint for Cohort 1 Median Percent Change in Disabling Seizure Frequency From Baseline (IQR) • During months 4 to 6, 3 of 6 20 participants achieved 0 reduction in disabling seizures Percent change −20 (ranging from 79-100%) −40 −60 • The remaining three participants experienced
Slide 6: Safety Profile: Summary
Summary • AEs reported in Cohort 1 were Grade 1 or 2 in Safety severity Profile: • No SAEs or DLEs in any participant to date across the study • During months 4 to 6, 3 of 6 participants achieved Initial reduction in disabling seizures (ranging from 79-100%) Clinical Data, • The remaining three part