TLDR
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QURE fell 3.88% as uniQure reported early AMT-260 epilepsy data.
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Three low-dose patients saw disabling seizures fall between 79% and 100%.
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Other patients showed mixed results, ranging from a decline to an increase.
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uniQure reported no serious AMT-260-related adverse events so far.
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Higher-dose enrollment continues, with updated results expected in 2027.
uniQure N.V. reported early seizure-reduction data from its AMT-260 epilepsy trial, while QURE stock fell 3.88% to $46.29. The stock dropped sharply after the market open, then steadied near $46.50 through most of the session. The update showed early biological activity, but patient responses varied across the first low-dose cohort.
AMT-260 Data Shows Early Seizure Reductions
uniQure released initial six-month follow-up data from the first cohort in its Phase I/IIa GenTLE study. The trial evaluates AMT-260 in patients with refractory mesial temporal lobe epilepsy. The company presented the data on June 19, 2026, at the Epilepsy Foundation Pipeline Conference.
The first low-dose cohort included six patients who received AMT-260 at 1×1012 gc/mL. Three patients recorded meaningful reductions in disabling seizures during months four through six. Their seizure reductions ranged from 79% to 100% compared with baseline levels.
However, the other three patients showed mixed seizure outcomes during the same follow-up period. Their results ranged from a 33% decrease to a 36% increase from baseline. Therefore, the first cohort showed early signals, but it also showed variable responses.
Trial Safety Profile Supports Continued Evaluation
uniQure reported no serious adverse events linked to AMT-260 or the surgical procedure. The company said all reported adverse events in the low-dose cohort remained mild or moderate. Headache was the most common adverse event, and it affected two patients.
The company also reported that patients did not require immunosuppression during the low-dose cohort evaluation. That detail matters because some gene therapy approaches require immune management during treatment. As a result, the early safety profile supported continued testing in the program.
AMT-260 uses a one-time, locally delivered intracerebral infusion for refractory MTLE. The therapy aims to deliver two engineered microRNAs that suppress the GRIK2 gene. The approach targets abnormal GluK2 expression, which researchers link to seizure activity in refractory MTLE.
uniQure Continues Higher-Dose Enrollment
Enrollment continues in the second cohort, which will test a higher AMT-260 dose of 3×1012 gc/mL. uniQure expects that cohort to include six patients, matching the first cohort size. The company anticipates completing enrollment around mid-2026.
The GenTLE trial remains a multi-center, open-label Phase I/IIa study in the United States. It includes an initial 12-month evaluation period, followed by four years of long-term follow-up. The study measures safety, tolerability, and exploratory signs of efficacy.
Refractory mesial temporal lobe epilepsy represents a major unmet medical need in the United States. Temporal lobe epilepsy affects about 500,000 people nationwide, and many patients do not respond well to medication. uniQure expects updated AMT-260 trial results in the first half of 2027.
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