uniQure Presents Clinical Case Study of First Patient Dosed with AMT-260 in Refractory Mesial Temporal Lobe Epilepsy (MTLE)
- 92% reduction in seizure frequency observed in first patient
- No serious adverse events reported through first five months
- No seizures reported during last 60 days of follow-up period
- Trial expansion to additional sites planned by end of 2025
- Data limited to only one patient so far
- Longer follow-up period needed to confirm durability of treatment
- Additional patient data required to validate efficacy
Insights
Early results from uniQure's AMT-260 gene therapy show promising 92% seizure reduction in first MTLE patient with no serious adverse events.
The data presented on uniQure's investigational gene therapy AMT-260 represents a potentially significant development in the treatment landscape for refractory mesial temporal lobe epilepsy (MTLE). The reported 92% reduction in seizure frequency in the first participant is clinically meaningful, especially considering this patient experienced no seizures during the last 60 days of the 5-month follow-up period.
Refractory MTLE is notoriously challenging to treat, with patients typically failing multiple anti-seizure medications. The standard intervention often involves resective surgery of the affected temporal lobe, which carries significant risks including memory impairment. A one-time administered gene therapy that specifically targets the underlying pathophysiology could represent a paradigm shift if these early results are maintained and replicated.
The mechanism of action is particularly interesting - AMT-260 delivers engineered microRNAs to suppress the GRIK2 gene and reduce expression of GluK2, a kainate glutamate receptor subunit implicated in seizure generation. This targeted approach addresses a specific molecular pathway believed to be instrumental in MTLE pathogenesis.
While these results are encouraging, several important caveats exist: this represents data from just one patient in a planned 12-patient study; the 5-month follow-up is relatively short for assessing durability; and as an open-label trial without a control group, placebo effects cannot be completely excluded. The company is appropriately cautious in noting that additional follow-up and more patient data are needed before drawing firm conclusions about efficacy.
~ No serious adverse events and
~ Data to be presented today at Epilepsy Therapies & Diagnostics Development Symposium (ETDD) ~
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 17th (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.”
AMT-260 is a one-time administered, in vivo gene therapy candidate intended to reduce or eliminate seizures in people with drug-resistant mesial temporal lobe epilepsy. AMT-260 is designed to locally deliver two engineered microRNAs to suppress the GRIK2 gene and the aberrant expression of GluK2, a subunit of a kainate glutamate receptor that is believed to trigger seizures in people with refractory MTLE.
GenTLE is a Phase I/IIa multi-center, open-label trial being conducted in the U.S. to evaluate the safety, tolerability and exploratory signs of efficacy of AMT-260 in individuals with refractory MTLE. The study is comprised of two dose cohorts of six patients each. The study is actively screening additional patients throughout 12 sites, with additional sites expected to be activated by the end of 2025. Additional details are available on www.clinicaltrials.gov (NCT06063850).
About Refractory Mesial Temporal Lobe Epilepsy
Temporal lobe epilepsy is a chronic neurologic disorder and is the most common form of focal epilepsy with more than 600,000 individuals suffering from the disorder in the United States. Approximately
About uniQure
uniQure is delivering on the promise of gene therapy – single treatments with potentially curative results. The approvals of uniQure’s gene therapy for hemophilia B – an historic achievement based on more than a decade of research and clinical development – represent a major milestone in the field of genomic medicine and ushers in a new treatment approach for patients living with hemophilia. uniQure is now advancing a pipeline of proprietary gene therapies for the treatment of patients with Huntington's disease, refractory temporal lobe epilepsy, ALS, Fabry disease, and other severe diseases. www.uniQure.com
uniQure Forward-Looking Statements
This press release contains forward-looking statements. Other than statements of historical fact, all statements contained in this press release are forward-looking statements, including, without limitation, statements about the design, advancement and intended effects of AMT-260, our belief that AMT-260 has the potential to be a valuable treatment alternative for people living with drug-resistant mesial temporal lobe epilepsy, our goals for site expansion and patient enrollment for AMT-260 and other statements identified by the terms “anticipate,” “believe,” “could,” “establish,” “estimate,” “expect,” “goal,” “intend,” “look forward to,” “may,” “plan,” “potential,” “predict,” “project,” “seek,” “should,” “will,” “would” and similar expressions. Forward-looking statements are not promises or guarantees of future performance, and are subject to a variety of risks and uncertainties, many of which are beyond our control. Investors are cautioned that any forward-looking statements, including updates regarding the future development of gene therapy candidates, including AMT-260 for drug-resistant mesial temporal lobe epilepsy, are not guarantees of future performance or results and involve substantial risks and uncertainties. Actual results, developments and events may differ materially from those in the forward-looking statements as a result of various risk factors including: risks associated with the clinical results and the development and timing of our clinical programs; our interactions with regulatory authorities, which may affect the initiation, timing and progress of clinical trials and pathways to approval, if any; our effectiveness in managing current and future clinical trials and regulatory processes; our ability to demonstrate the therapeutic benefits of our gene therapy candidates, including AMT-260, in clinical trials; our ability to continue to build and maintain our infrastructure and personnel needed to achieve our goals; the continued development and acceptance of gene therapies; our ability to obtain, maintain and protect our intellectual property, our ability to fund our operations and to raise additional capital as needed; and other risks as may be detailed from time to time under the heading “Risk Factors” in our periodic filings with the U.S. Securities & Exchange Commission (SEC), including our Annual Report on Form 10-K filed with the SEC on February 27, 2025, our Quarterly Report on Form 10-Q filed with the SEC on May 9, 2025, and in other filings that we make with the SEC from time to time. Given these risks, uncertainties and other factors, you should not place undue reliance on these forward-looking statements. The information contained in this press release is as of the date of this press release and we assume no obligation to update the forward-looking statements contained in this release as the result of new information or future events or developments.
uniQure Contacts:
| FOR INVESTORS: | FOR MEDIA: |
| Chiara Russo | Tom Malone |
| Direct: 617-306-9137 | Direct: 339-970-7558 |
| Mobile: 617-306-9137 | Mobile:339-223-8541 |
| c.russo@uniQure.com | t.malone@uniQure.com |
FAQ
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