uniQure N.V. (NASDAQ:QURE) on Friday reported initial six-month follow-up data from the first low-dose cohort in its ongoing Phase 1/2a trial evaluating investigational gene therapy candidate AMT-260 in patients with refractory mesial temporal lobe epilepsy (MTLE).
MTLE represents the most prevalent and treatment-resistant subtype of drug-resistant epilepsy.
uniQure Reports Early Seizure Reductions In Initial AMT-260 Epilepsy Study
The company said several patients experienced substantial reductions in disabling seizures, while the treatment continued to demonstrate a favorable safety profile.
According to data collected through May 29, 2026, three of the six patients receiving the low dose of AMT-260 achieved meaningful declines in disabling seizures during months four through six of follow-up.
Seizure reductions in those patients ranged from 79% to 100% compared with baseline levels.
The remaining three participants reported mixed outcomes during the same period, with changes in disabling seizures ranging from a 33% reduction to a 36% increase from baseline.
Safety Profile Remains Favorable
As of the presentation date, the company said no serious adverse events linked to AMT-260 or the associated surgical procedure had been reported.
All adverse events observed in the low-dose group were classified as either mild or moderate. Headache, reported in two patients, was the most frequently observed adverse event. The company also noted that no immunosuppression was required.
Walid Abi-Saab, chief medical officer at uniQure, said the results provide preliminary evidence suggesting biological activity for AMT-260, although he cautioned that the findings are based on a small patient population and require additional follow-up.
Abi-Saab added that the therapy’s tolerability profile and the responses seen so far support continued development of the candidate.
Higher-Dose Cohort Enrollment Continues
uniQure is currently enrolling patients in a second cohort evaluating a higher dose of AMT-260 at 3×1012 gc/mL. The cohort is expected to include six patients, with enrollment anticipated to conclude by mid-2026.
The company expects to provide updated data from the ongoing Phase 1/2a study during the first half of 2027.
In March, Ovid Therapeutics Inc. (NASDAQ:OVID) reported favorable topline safety and tolerability findings from a 7 mg dose cohort of OV329 for drug-resistant epilepsies.
QURE Stock Price Activity: uniQure shares were up 0.04% at $46.31 at the time of publication on Monday, according to Benzinga Pro data.
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