uniQure Presents Clinical Case Study of First Patient Dosed with AMT-260 in Refractory Mesial Temporal Lobe Epilepsy (MTLE)

~ No serious adverse events and 92% reduction in seizure frequency observed in the first trial participant through first five months of follow up ~

~ Data to be presented today at Epilepsy Therapies & Diagnostics Development Symposium (ETDD) ~

Excerpt from the Press Release:

LEXINGTON, Mass. and AMSTERDAM, May 29, 2025 (GLOBE NEWSWIRE) — uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced the presentation of a clinical case study of the first participant dosed in the first cohort of its ongoing Phase I/IIa trial of an investigational gene therapy candidate, AMT-260, in patients with refractory MTLE. The data are being presented today, May 29, 2025 by Firas Taha, M.D., Medical Director, Clinical Development at uniQure, at the Epilepsy Therapies & Diagnostics Development Symposium in Leesburg, Virginia.

The first participant in the GenTLE clinical trial has been followed for five months post-administration of AMT-260. Prior to treatment, the participant experienced an average of seven seizures a month, including five seizures in the 30-day period immediately prior to dosing. Since receiving AMT-260, the participant has reported two seizures during the five-month follow-up period, with no seizures reported during the last 60 days as of April 17^th (cutoff date), the last date data were obtained on this participant for presentation in the ETDD symposium case study and no reported serious adverse events as of the cutoff date.

“These early results from the first trial participant are very encouraging and support our belief that AMT-260 has the potential to be a valuable treatment alternative for people living with drug-resistant mesial temporal lobe epilepsy,” stated Walid Abi-Saab, M.D., chief medical officer of uniQure. “While additional follow-up on this first trial participant and data from additional participants in the trial are needed, the reduction in seizure frequency and tolerability in this first trial participant as of the cutoff date offer a compelling early signal of the potential impact of AMT-260 that warrants continued investigation. There remains a high unmet need for safer, more effective treatment options for people with drug-resistant MTLE and we’re eager to continue evaluating AMT-260 in additional trial participants.”

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