uniQure (QURE) shows mixed but promising early AMT-260 MTLE results
Rhea-AI Filing Summary
uniQure N.V. reported preliminary six-month data from the first, low-dose cohort in its Phase I/IIa trial of AMT-260 for refractory mesial temporal lobe epilepsy. Three of six patients showed meaningful reductions in disabling seizures between months four and six, with declines of 79% to 100% from baseline.
The other three patients had mixed results, ranging from a 33% decrease to a 36% increase in disabling seizures over the same period. AMT-260 was generally well-tolerated, with no serious adverse events related to the therapy or surgery, only mild to moderate side effects such as headache, and no need for immunosuppression.
Positive
- None.
Negative
- None.
Insights
Early AMT-260 data show mixed efficacy but clean safety in first MTLE cohort.
uniQure disclosed initial six-month results from the first, low-dose cohort of its Phase I/IIa AMT-260 trial in refractory mesial temporal lobe epilepsy. Three of six patients achieved 79%–100% reductions in disabling seizures during months four to six, while three had more variable outcomes.
Safety appears favorable so far, with no Serious Adverse Events related to AMT-260 or the procedure, only mild to moderate adverse events and no immunosuppression. This is important given AMT-260 is a one-time intracerebral gene therapy, where tolerability can be a key hurdle.
Enrollment continues in a higher-dose cohort of up to six patients at 3x1012 gc/mL, with completion anticipated mid-2026 and updated trial results planned for the first half of 2027. Future outcomes in the higher dose and longer-term follow-up will be central to understanding AMT-260’s risk-benefit profile.
8-K Event Classification
Key Figures
Key Terms
Phase I/IIa clinical
refractory mesial temporal lobe epilepsy clinical
Serious Adverse Events clinical
microRNAs clinical
GRIK2 gene clinical
gene therapy clinical
FAQ
What preliminary AMT-260 results did uniQure (QURE) report in mesial temporal lobe epilepsy?
How many patients responded to AMT-260 in uniQure’s initial Phase I/IIa cohort?
What safety profile did AMT-260 show in uniQure’s early MTLE trial data?
What is the dosing design of uniQure’s Phase I/IIa AMT-260 trial for MTLE?
When does uniQure (QURE) expect more AMT-260 clinical data to be available?
What condition is AMT-260 targeting and how common is it in the United States?
AI-generated analysis. How Rhea-AI works. Not financial advice.
UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
WASHINGTON, D.C. 20549
FORM
CURRENT REPORT
Pursuant to Section 13 or 15(d) of the Securities Exchange Act of 1934
Date of Report (Date of earliest event reported):
(Exact Name of Registrant as Specified in Charter)
The | | | ||
(State or Other |
| (Commission |
| (IRS Employer |
| N/A | |
(Address of Principal Executive Offices) |
| (Zip Code) |
Registrant’s telephone number, including area code:
(Former Name or Former Address, if Changed Since Last Report)
Check the appropriate box below if the Form 8-K filing is intended to simultaneously satisfy the filing obligation of the registrant under any of the following provisions (see General Instruction A.2. below):
Securities registered pursuant to Section 12(b) of the Act:
Title of each class: | | Trading Symbol(s) | | Name of each exchange on which registered: |
|
|
|
Indicate by check mark whether the registrant is an emerging growth company as defined in Rule 405 of the Securities Act of 1933 (§230.405 of this chapter) or Rule 12b-2 of the Securities Exchange Act of 1934 (§240.12b-2 of this chapter).
Emerging growth company
If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act. ☐
Item 7.01 Regulation FD Disclosure.
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. A copy of the press release is being furnished as Exhibit 99.1 to this Current Report on Form 8-K and is incorporated herein by reference.
The information provided in this Item 7.01, including the accompanying Exhibit 99.1, shall be deemed “furnished” and shall not be deemed “filed” for the purposes of Section 18 of the Securities Exchange Act of 1934, as amended (the “Exchange Act”), or otherwise subject to the liability of such section, nor shall it be incorporated by reference in any filing made by the Company pursuant to the Securities Act of 1933, as amended, or the Exchange Act, regardless of the general incorporation language of such filing, except to the extent that such filing incorporates by reference any or all of such information by express reference.
Item 8.01 Other Events.
On June 19, 2026, the Company announced 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. As of the May 29, 2026 data cutoff date, three of six patients in the first, low-dose cohort (1x1012 gc/mL) achieved meaningful reductions in disabling seizures during months four through six of follow-up, ranging from a 79% to 100% decline from baseline. The remaining three patients in the low-dose cohort experienced variable changes in disabling seizures during months four through six of follow-up, ranging from a 33% decrease to a 36% increase compared to baseline. As of June 19, 2026, there have been no serious adverse events related to AMT-260 or the surgical procedure reported. All reported adverse events in the low dose cohort were classified as mild or moderate in severity, with the most common adverse event being headache (N=2). No immunosuppression was required.
Item 9.01 Financial Statements and Exhibits.
(d) Exhibits.
Exhibit No. | | Description |
99.1 |
| Press Release of uniQure N.V. dated June 19, 2026 |
104 |
| Cover Page Interactive Data File (embedded with the Inline XBRL document). |
SIGNATURES
Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned hereunto duly authorized.
| | UNIQURE N.V. | |
|
| ||
|
|
| |
Date: June 22, 2026 | By: | /s/ Jeannette Potts | |
|
| Jeannette Potts | |
|
| Chief Legal and Compliance Officer | |
Exhibit 99.1
uniQure Announces Preliminary Data on the First Cohort in the Phase I/IIa Clinical Trial of AMT-260 in Refractory Mesial Temporal Lobe Epilepsy
~ AMT-260 was generally well-tolerated with no serious adverse events observed to date, with early biological signals of potential therapeutic activity ~
Lexington, MA and Amsterdam, the Netherlands, June 19, 2026 — uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced initial six-month follow-up data on the first, low dose cohort of six patients in its ongoing Phase I/IIa trial of an investigational gene therapy candidate, AMT-260, for the treatment of refractory mesial temporal lobe epilepsy (MTLE). The data will be presented today, Friday, June 19, 2026, at the Epilepsy Foundation Pipeline Conference in Leesburg, VA.
As of the May 29, 2026 data cutoff date, three of six patients in the first, low-dose cohort (1x1012 gc/mL) achieved meaningful reductions in disabling seizures during months four through six of follow-up, ranging from a 79% to 100% decline from baseline. The remaining three patients in the low-dose cohort experienced variable changes in disabling seizures during months four through six of follow-up, ranging from a 33% decrease to a 36% increase compared to baseline.
As of the date of the presentation, there have been no Serious Adverse Events (SAEs) related to AMT-260 or the surgical procedure reported. All reported adverse events in the low dose cohort were classified as mild or moderate in severity, with the most common adverse event being headache (N=2). No immunosuppression was required.
“While patient responses have varied, we believe the data generated to date provide preliminary evidence of biological activity of AMT-260,” said Walid Abi-Saab, M.D., chief medical officer of uniQure. “Although these findings are based on a limited number of patients and require longer follow-up, the favorable tolerability profile and observed responses support continued evaluation of AMT-260.”
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.
About the Phase I/IIa Clinical Program of AMT-260
GenTLE is a Phase I/IIa multi-center, open-label trial being conducted in the United States currently consisting of two dosing cohorts of up to six refractory MTLE patients each receiving a locally delivered, one-time intracerebral infusion of AMT-260. The study consists of an initial 12-month evaluation period followed by long-term follow-up for four years. The trial will evaluate the safety, tolerability and exploratory signs of efficacy of AMT-260 in individuals with refractory MTLE. Additional details are available on www.clinicaltrials.gov (NCT06063850).
About AMT-260
AMT-260 is a one-time administered, in vivo gene therapy candidate intended to reduce or eliminate seizures in people with refractory 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.
About Refractory Mesial Temporal Lobe Epilepsy
Temporal lobe epilepsy is a chronic neurologic disorder and affects approximately 500,000 people in the United States, of which approximately 300,000 are inadequately treated through anti-seizure medications and are considered refractory. Approximately 80% of United States refractory temporal lobe epilepsy cases are mesial, which involves the medial (or internal) structures of the brain.
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 – a 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, Fabry disease, and other severe diseases. www.uniQure.com
uniQure Forward-Looking Statements
This press release contains forward-looking statements. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as “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 based on management's beliefs and assumptions and on information available to management only as of the date of this press release. Examples of these forward-looking statements include, but are not limited to, statements concerning: the advancement and intended effects of AMT-260, plans to complete enrollment in the second cohort of the Phase I/IIa clinical trial in mid-2026, and plans to present updated results from the Phase I/IIa clinical trial in the first half of 2027. The Company’s actual results could differ materially from those anticipated in these forward-looking statements for many reasons. These risks and uncertainties include, among others: risks associated with the clinical results and the development and timing of the Company’s clinical programs; the risk that more patient data become available that results in different findings than that presented in preliminary or interim data; the Company’s interactions with regulatory authorities, which may affect the initiation, timing and progress of clinical trials and pathways and timing for regulatory approval; the Company’s ability to continue to build and maintain the company infrastructure and personnel needed to achieve its goals; the Company’s effectiveness in managing current and future clinical trials and regulatory processes; the continued development and acceptance of gene therapies; the Company’s ability to demonstrate the therapeutic benefits of its gene therapy candidates, including AMT-260, in clinical trials; the Company’s ability to obtain, maintain and protect intellectual property; and the Company’s ability to fund its operations and to raise additional capital as needed. These risks and uncertainties are more fully described under the heading "Risk Factors" in the Company’s periodic filings with the U.S. Securities & Exchange Commission (“SEC”), including the Company’s Annual Reports on Form 10-K and Quarterly Reports on Form 10-Q, and in other filings that the Company makes 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, and the Company assumes no obligation to update these forward-looking statements, even if new information becomes available in the future.
uniQure Contacts:
FOR INVESTORS: | FOR MEDIA: |
| |
Chiara Russo | Tom Malone |
Direct: 781-491-4371 | Direct: 339-970-7558 |
Mobile: 617-306-9137 | Mobile:339-223-8541 |
c.russo@uniQure.com | t.malone@uniQure.com |