uniQure Releases Initial Data on AMT-260

uniQure announced initial six-month follow-up data on the first, low dose cohort of six patients in its ongoing Phase I/IIa trial of an investigational gene therapy candidate, AMT-260, for the treatment of refractory mesial temporal lobe epilepsy. The data will be presented Friday, June 19, at the Epilepsy Foundation Pipeline Conference in Leesburg, VA. As of the May 29 data cutoff date, three of six patients in the first, low-dose cohort achieved meaningful reductions in disabling seizures during months four through six of follow-up, ranging from a 79% to 100% decline from baseline. The remaining three patients in the low-dose cohort experienced variable changes in disabling seizures during months four through six of follow-up, ranging from a 33% decrease to a 36% increase compared to baseline. As of the date of the presentation, there have been no Serious Adverse Events related to AMT-260 or the surgical procedure reported. All reported adverse events in the low dose cohort were classified as mild or moderate in severity, with the most common adverse event being headache. No immunosuppression was required. Enrollment is ongoing in a second, higher dose cohort (3x1012 gc/mL), expected to consist of six patients with enrollment anticipated to be completed mid-2026. uniQure expects to present updated results from the Phase I/IIa clinical trial in the first half of 2027. "While patient responses have varied, we believe the data generated to date provide preliminary evidence of biological activity of AMT-260," said Walid Abi-Saab, M.D., chief medical officer of uniQure. "Although these findings are based on a limited number of patients and require longer follow-up, the favorable tolerability profile and observed responses support continued evaluation of AMT-260."