uniQure QURE reported initial six-month follow-up results from the first low-dose cohort of six patients enrolled in its ongoing phase I/IIa study evaluating AMT-260, an investigational gene therapy for the treatment of refractory mesial temporal lobe epilepsy (MTLE).
The data was also presented at the Epilepsy Foundation Pipeline Conference in Leesburg, VA.
Data as of May 29, 2026, showed that three of the six patients in the first low-dose cohort (1×10¹² gc/mL) experienced substantial reductions in disabling seizures during months four through six, with declines ranging from 79% to 100% compared with baseline.
The other three patients showed mixed outcomes, with seizure frequency ranging from a 33% reduction to a 36% increase versus baseline over the same period.
As of the presentation date, no treatment-related or procedure-related serious adverse events (SAEs) had been reported.
All adverse events observed in the low-dose cohort were mild to moderate in severity, with headache being the most frequently reported event, occurring in two patients. No patient required immunosuppressive therapy.
Although patient responses were mixed, the data generated to date are encouraging as they provide preliminary evidence of biological activity of AMT-260 in MTLE.
Enrollment is currently underway in a second cohort evaluating a higher dose of AMT-260 (3×10¹² gc/mL), which is expected to include six patients, with enrollment projected to be completed by mid-2026.
uniQure plans to report updated data from the phase I/IIa study in the first half of 2027.
Temporal lobe epilepsy is a chronic neurological condition affecting an estimated 500,000 people in the United States. Approximately 300,000 patients remain inadequately controlled despite treatment with anti-seizure medications and are classified as having refractory disease. About 80% of these refractory cases are mesial temporal lobe epilepsy, which involves the medial (or internal) structures of the brain.
QURE’s Other Pipeline Candidates
uniQure’s pipeline comprises a few other candidates in development.
Shares of company have recently surged following the announcement of a significant regulatory milestone for AMT-130, the company's investigational gene therapy for Huntington's disease.
AMT-130 is based on uniQure's proprietary miQURE gene-silencing platform and is currently being evaluated in two ongoing multi-center, dose-escalation phase I/II studies. The studies are designed to assess the therapy's safety and tolerability while also exploring its potential efficacy in patients with Huntington's disease.
During a recent type B meeting, the FDA informed uniQure that the three-year data from the phase I/II study on AMT-130 study could serve as the primary basis for a biologics license application (BLA) seeking accelerated approval for the therapy for the treatment of Huntington's disease.
QURE expects to receive final minutes within 30 days of the recent type B meeting.
The FDA seeks alignment on the confirmatory study design before BLA submission, including consideration of a concurrent standard-of-care control group in place of a sham-controlled design. The agency stated that it would work expeditiously with uniQure to finalize these plans.
The company expects to complete alignment with the FDA ahead of its anticipated BLA filing in the third quarter of 2026 and is committed to initiating the confirmatory study swiftly.
The latest clinical update provided some reassurance to investors following a significant regulatory setback earlier this year. QURE shares plunged in March 2026 after the company revealed that the FDA did not agree that data from the phase I/II studies, when compared with an external control group, constituted sufficient primary evidence of effectiveness to support a marketing application for AMT-130.
QURE shares have surged 93.4% year to date against the industry’s 1.8% decline.
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Another candidate in QURE’s pipeline is AMT-191 for the treatment of Fabry disease.
Please note that the company also markets an internally developed gene therapy, in partnership with CSL Behring, for the treatment of hemophilia B in the United States and the EU under the brand name Hemgenix.
QURE’s Zacks Rank and Stocks to Consider
uniQure currently carries a Zacks Rank #3 (Hold). A couple of better-ranked biotech stocks are Liquidia Corporation LQDA and Immunocore IMCR. While LQDA sports a Zacks Rank #1 (Strong Buy), IMCR carries a Zacks Rank #2 (Buy) at present. You can see the complete list of today’s Zacks #1 Rank stocks here.
Over the past 60 days, estimates for Liquidia’s 2026 earnings per share (EPS) have increased to $2.97 from $1.50. Over the same period, EPS estimates for 2027 have risen to $4.81 from $2.91. LQDA shares have surged 106.1% year to date.
Liquidia’s earnings beat estimates in three of the trailing four quarters and missed in the remaining one, with the average surprise being 54.40%.
Over the past 30 days, EPS estimates for Immunocore have remained unchanged at 6 cents for 2026 and at 87 cents for 2027.
Immunocore’s earnings beat estimates in three of the trailing four quarters and missed in the remaining one, the average surprise being 46.66%.
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This article originally published on Zacks Investment Research (zacks.com).