Open report — full analysis, no account required.
Sign up to generate reports and read filings that aren't on the open list.
Get notified when QURE files again. Create a free account and we'll email you the moment its next filing is analyzed.
Get filing alertsuniQure reports mixed Phase I/IIa results for AMT-260 epilepsy gene therapy
Filed June 22, 2026 · Period ending June 19, 2026 · ~1 min read
Key Changes
-
high
Three of six patients in the low-dose cohort achieved 79-100% seizure reductions during months 4-6, while the other three had variable outcomes ranging from a 33% decrease to a 36% increase versus baseline.
Item 8.01 — Other Events verify on EDGAR → -
high
No serious adverse events related to AMT-260 or the surgical procedure were reported; all adverse events were mild or moderate, with headache most common (2 patients), and no immunosuppression was required.
Item 8.01 — Other Events verify on EDGAR → -
medium
Enrollment is ongoing in a higher-dose cohort (3x10^12 gc/mL vs 1x10^12 gc/mL), expected to complete mid-2026, with updated trial results planned for first half 2027.
Exhibit 99.1 view on EDGAR →
Summary
uniQure disclosed preliminary Phase I/IIa data for AMT-260, an investigational gene therapy for refractory mesial temporal lobe epilepsy. In the first low-dose cohort of six patients, half achieved substantial seizure reductions (79-100% decline) during months 4-6 of follow-up, while the other half experienced variable outcomes including one patient with a 36% increase in seizures.
The safety profile was favorable, with no serious adverse events and no need for immunosuppression. The mixed efficacy results demonstrate biological activity in half the cohort but highlight significant patient-to-patient variability.
For a gene therapy in early-stage testing, the 50% response rate and clean safety profile represent a proof-of-concept, though the variability raises questions about patient selection or dosing. The company is enrolling a higher-dose cohort to explore whether increased exposure improves consistency of response. Investors should watch for the higher-dose cohort data in early 2027 to assess whether dose escalation narrows the response variability.
Section-by-Section Diff
Event · Exhibit 99.1
uniQure disclosed preliminary 6-month Phase I/IIa trial data for AMT-260 gene therapy in refractory epilepsy, showing variable efficacy and favorable safety.
Added in current filing · view on EDGAR →
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.
The company is enrolling a second cohort at a higher dose (3x1012 gc/mL vs 1x1012 gc/mL in the first cohort), with enrollment expected to complete mid-2026. Updated trial results are planned for presentation in the first half of 2027. This dose escalation is a standard Phase I/IIa approach to identify the optimal therapeutic dose while monitoring safety.
Event · Item 7.01 — Regulation FD Disclosure
uniQure announced preliminary Phase I/IIa data for AMT-260 in refractory mesial temporal lobe epilepsy.
Added in current filing · verify on EDGAR →
On June 19, 2026, uniQure N.V. (the “Company”) issued a press release announcing preliminary data on the first cohort in the Company’s Phase I/IIa clinical trial of AMT-260 for the treatment of refractory mesial temporal lobe epilepsy.
The company disclosed preliminary clinical trial results for AMT-260, a gene therapy candidate targeting refractory mesial temporal lobe epilepsy. This is the first cohort data from the Phase I/IIa trial. The 8-K does not include the actual data results, only that a press release was issued announcing them.
Thanks — your feedback helps us improve report quality.
Figures/quotes linked to EDGAR · Narrative written by AI · Jun 22, 2026 · How we verify