uniQure advances AMT-130 toward UK approval filing

uniQure N.V. announced a successful pre-submission meeting with the UK Medicines and Healthcare products Regulatory Agency for AMT-130, its investigational gene therapy for Huntington’s disease.

Rhea-AI Filing Summary

uniQure N.V. announced a successful pre-submission meeting with the UK Medicines and Healthcare products Regulatory Agency for AMT-130, its investigational gene therapy for Huntington’s disease. The company plans to submit a UK Marketing Authorization Application in the third quarter of 2026.

The planned submission will be based on three-year data from ongoing U.S. and European Phase I/II trials, where high-dose AMT-130 showed a statistically significant 75% slowing of disease progression on a composite Huntington’s scale versus a matched external control (p=.003) and was generally well-tolerated with a manageable safety profile.

uniQure has also been granted a Type B meeting with the U.S. Food and Drug Administration in the second quarter of 2026 to discuss a potential Phase III trial design and the statistical analysis plan for a four-year data readout expected in the third quarter of 2026, while pursuing additional ex-US regulatory pathways.

Insights

Biotech regulatory & clinical strategy analyst

uniQure advances AMT-130 toward UK filing using encouraging three-year data.

uniQure is moving AMT-130 for Huntington’s disease toward a UK Marketing Authorization Application after a constructive pre-submission meeting with the MHRA. The MAA is planned for Q3 2026, anchored on three-year Phase I/II data.

Those data showed a 75% slowing of disease progression at the high dose on a composite Huntington’s scale versus a propensity score-matched external control (p=.003), with a generally manageable safety profile. This suggests a potentially meaningful treatment effect, though in an early-stage setting.

A Type B meeting with the U.S. FDA in Q2 2026 will focus on possible Phase III trial design and the statistical plan for a four-year analysis expected in Q3 2026. Outcomes of these regulatory interactions and longer-term data will shape AMT-130’s path toward broader registration.

8-K Event Classification

2 items: 7.01, 9.01

2 items

Item 7.01 Regulation FD Disclosure Disclosure

Material non-public information disclosed under Regulation Fair Disclosure, often investor presentations or guidance.

Item 9.01 Financial Statements and Exhibits Exhibits

Financial statements, pro forma financial information, or exhibit attachments filed with this report.

Key Figures

Planned UK MAA timing: Q3 2026 Slowing of disease progression: 75% P-value for efficacy signal: 0.003 +3 more

6 metrics

Planned UK MAA timing Q3 2026 Target quarter for AMT-130 UK Marketing Authorization Application

Slowing of disease progression 75% High-dose AMT-130 vs external control at three years on composite Huntington’s scale

P-value for efficacy signal 0.003 Statistical significance of 75% slowing in disease progression at three years

FDA Type B meeting timing Q2 2026 Planned U.S. FDA meeting to discuss AMT-130 Phase III design and analysis plan

Four-year data analysis timing Q3 2026 Expected timing for four-year AMT-130 data analysis discussion with FDA feedback

Additional updates timing Second half of 2026 Expected timing for further updates on international regulatory pathways

Key Terms

Marketing Authorization Application, Type B meeting, composite Unified Huntington’s Disease Rating Scale, propensity score-matched external control, +2 more

6 terms

Marketing Authorization Application regulatory

"plans to submit a Marketing Authorization Application (MAA) for AMT-130"

A marketing authorization application is a formal request submitted to a government regulator asking permission to sell a prescription medicine or medical product in a country or region. Think of it like asking for a business license after showing evidence the product is safe and works; investors care because approval determines whether the product can generate sales, how soon revenue starts, and how much regulatory risk and uncertainty remains.

Type B meeting regulatory

"has been granted a Type B meeting with the U.S. Food and Drug Administration"

A Type B meeting is a formal, scheduled discussion between a drug or medical-device developer and a health regulator to resolve key mid‑ or late‑stage development issues such as clinical trial plans, interpretation of results, or steps needed for approval. Like a mid‑project review with an inspector, the meeting’s outcome can meaningfully change the timeline, cost and risk for a candidate: a clear, positive outcome lowers uncertainty for investors, while requests for more data or changes can signal delays and extra expense.

composite Unified Huntington’s Disease Rating Scale medical

"as measured by the composite Unified Huntington’s Disease Rating Scale compared to a propensity score-matched external control"

propensity score-matched external control technical

"compared to a propensity score-matched external control (p=.003)"

A propensity score-matched external control is a group of patients drawn from outside a clinical trial whose characteristics are statistically matched to trial participants using a propensity score—a single number summarizing the likelihood of being in the treated group based on observable factors. It acts like a simulated comparison arm when a randomized control is unavailable, helping companies and regulators judge a treatment’s effects; investors care because such comparisons influence regulatory review, perceived evidence strength, and clinical value.

gene therapy medical

"a leading gene therapy company advancing transformative therapies"

Gene therapy is a medical technique that involves altering or replacing faulty genes in a person's cells to treat or prevent disease. It is considered a promising area of innovation because it has the potential to provide long-term or even permanent solutions to genetic conditions. For investors, advancements in gene therapy can signal opportunities in biotech companies and emerging treatments with significant growth potential.

Phase I/II clinical trials medical

"three-year analysis from the ongoing U.S. and European Phase I/II clinical trials"

Phase I/II clinical trials are early-stage drug or medical-device studies that first test safety and the best dose in a small group (Phase I) and then expand to a larger group to look for early signs that the treatment works (Phase II). For investors, these combined trials are key milestones: positive results can meaningfully reduce scientific risk and boost a company’s value, while failures are common and can sharply lower expectations—think of them as the cooking test where a recipe is both adjusted and sampled before deciding to mass-produce.

FAQ

AI-generated questions and answers. How Rhea-AI works. Not financial advice.

What did uniQure (QURE) announce regarding AMT-130 and the UK MHRA?

uniQure announced it held a successful pre-submission meeting with the UK MHRA for AMT-130. The company now plans to submit a UK Marketing Authorization Application for this Huntington’s disease gene therapy candidate in the third quarter of 2026, based on three-year Phase I/II data.

When does uniQure (QURE) plan to submit the UK Marketing Authorization Application for AMT-130?

uniQure plans to submit a UK Marketing Authorization Application for AMT-130 in the third quarter of 2026. This filing will rely on a three-year analysis from ongoing U.S. and European Phase I/II trials evaluating efficacy and safety in Huntington’s disease patients receiving AMT-130.

What clinical results support uniQure’s planned AMT-130 UK filing?

At three years, high-dose AMT-130 showed a statistically significant 75% slowing of Huntington’s disease progression versus a matched external control. The effect was measured by the composite Unified Huntington’s Disease Rating Scale, with a p-value of 0.003, and treatment was generally well-tolerated with a manageable safety profile.

What interaction does uniQure (QURE) have planned with the U.S. FDA for AMT-130?

uniQure has been granted a Type B meeting with the U.S. FDA in the second quarter of 2026. The company expects to discuss key elements of a potential Phase III trial design and obtain feedback on the proposed statistical analysis plan for a four-year AMT-130 data readout.

How is uniQure (QURE) pursuing AMT-130 approvals outside the UK and U.S.?

uniQure is actively pursuing additional regulatory pathways in ex-US markets for potential registration of AMT-130. The company plans to work with regulators internationally and expects to provide further updates on these efforts in the second half of 2026 as discussions progress.

What does the 75% slowing of disease progression mean for AMT-130 in Huntington’s disease?

The 75% slowing indicates high-dose AMT-130 patients declined more slowly on a composite Huntington’s disease rating scale than a matched external control group. With a p-value of 0.003 and a generally manageable safety profile, these early results support continued regulatory engagement and potential future pivotal studies.

AI-generated analysis. How Rhea-AI works. Not financial advice.

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Accepted by SEC EDGAR 04/30/2026 - 07:07 AM EDT Learn about SEC filing dates

00-00000000001590560false00015905602026-04-302026-04-30

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UNITED STATES

SECURITIES AND EXCHANGE COMMISSION

WASHINGTON, D.C. 20549

FORM 8-K

CURRENT REPORT

Pursuant to Section 13 or 15(d) of the Securities Exchange Act of 1934

Date of Report (Date of earliest event reported): April 30, 2026

uniQure N.V.

(Exact Name of Registrant as Specified in Charter)

The Netherlands	​ ​ ​	001-36294	​ ​ ​	N/A
(State or Other Jurisdiction of Incorporation)		(Commission File Number)		(IRS Employer Identification No.)

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Paasheuvelweg 25a, 1105 BP Amsterdam, The Netherlands	​ ​ ​	N/A
(Address of Principal Executive Offices)		(Zip Code)

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Registrant’s telephone number, including area code: +31-20-240-6000

(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):

☐     Written communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425)

☐     Soliciting material pursuant to Rule 14a-12 under the Exchange Act (17 CFR 240.14a-12)

☐     Pre-commencement communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR 240.14d-2(b))

☐     Pre-commencement communications pursuant to Rule 13e-4(c) under the Exchange Act (17 CFR 240.13e-4(c))

Securities registered pursuant to Section 12(b) of the Act:

Title of each class:	​ ​ ​	Trading Symbol(s)	​ ​	Name of each exchange on which registered:
Ordinary Shares, par value €0.05 per share		QURE		The Nasdaq Stock Market LLC The Nasdaq Global Select Market

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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).

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Emerging growth company ☐

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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. ☐

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Item 7.01                        Regulation FD Disclosure.

On April 30, 2026, uniQure N.V. (the “Company”) issued a press release announcing that the Company held a pre-submission meeting with the United Kingdom’s Medicines and Healthcare products Regulatory Agency for AMT-130, the Company’s investigational gene therapy for the treatment of Huntington’s disease, and plans to submit a Marketing Authorization Application for AMT-130 in the third quarter of 2026. 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.

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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.

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Item 9.01                        Financial Statements and Exhibits.

(d)            Exhibits.

Exhibit No.	​ ​	Description
99.1		Press Release of uniQure N.V. dated April 30, 2026
104		Cover Page Interactive Data File (embedded with the Inline XBRL document).

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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.

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	​ ​ ​	UNIQURE N.V.
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Date: April 30, 2026	​	By:	/s/ Jeannette Potts
	​		JEANNETTE POTTS
	​		Chief Legal and Compliance Officer

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Exhibit 99.1

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uniQure Announces Successful UK MHRA Pre-Submission Meeting for AMT-130 in Huntington’s Disease

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~ Submission of a UK Marketing Authorization Application for AMT-130 is expected

in the third quarter of 2026 ~

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~ Type B Meeting with U.S. FDA granted in second quarter; expect to discuss potential Phase III design

and analysis plan for AMT-130 four-year data ~

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~ uniQure actively pursuing additional ex-US regulatory pathways to support potential registration of

AMT-130 in international markets ~

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Lexington, MA and Amsterdam, the Netherlands, April 30, 2026 — uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced the Company held a Pre-Submission Meeting with the United Kingdom’s (UK) Medicines and Healthcare products Regulatory Agency (MHRA) and plans to submit a Marketing Authorization Application (MAA) for AMT-130 for the treatment of Huntington’s disease in the third quarter of 2026.

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“We are encouraged by the constructive feedback from the MHRA as we continue to work towards a regulatory submission for AMT-130 in Huntington’s disease, which we now expect to submit in the third quarter of this year,” said Matt Kapusta, chief executive officer of uniQure. “This is an important milestone for the Huntington’s disease community and we remain committed to working closely with regulators globally, with the goal of bringing this potentially transformative therapy to patients in the UK and internationally.”

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During the meeting, the Company and MHRA discussed the data package and manufacturing requirements to support a MAA submission for AMT-130.  Following these discussions, the Company expects to submit a MAA based on the three-year analysis from the ongoing U.S. and European Phase I/II clinical trials. At year three, these data showed a statistically significant 75% slowing of disease progression at the high dose as measured by the composite Unified Huntington’s Disease Rating Scale compared to a propensity score-matched external control (p=.003) and was generally well-tolerated, with a manageable safety profile.

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In addition to the continued engagement with the MHRA, the Company has been granted a Type B meeting with the U.S. Food and Drug Administration in the second quarter of 2026. The Company expects to discuss key elements of a potential Phase III trial design and to receive feedback on the proposed statistical analysis plan for the four-year analysis expected in the third quarter of 2026. The Company is also actively pursuing additional regulatory pathways in international markets for potential registration of AMT-130 and expects to provide further updates in the second half of 2026.

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About uniQure

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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

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uniQure Forward-Looking Statements

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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 and the negatives of those terms. Forward-looking statements are based on management's beliefs and assumptions and on information available to management as of the date of this press release. Examples of these forward-looking statements include, but are not limited to, statements concerning: plans to submit a Marketing Authorization Application in the third quarter of 2026 for AMT-130 based on the three-year analysis from the ongoing Phase I/II clinical trials; plans to meet with the U.S. Food and Drug Administration (FDA) in the second quarter of 2026 to discuss a potential Phase III trial design for AMT-130 and a statistical analysis plan for a four-year analysis of AMT-130 expected in the third quarter of 2026; plans to work with regulators globally to potentially bring AMT-130 to patients; plans to provide future updates regarding the Company’s interactions with regulatory authorities; and the potential benefits of AMT-130. 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 related to the Company’s Phase I/ll clinical trials of AMT-130, including the risk that such trials will be unable to demonstrate data sufficient to support further clinical development or regulatory approval; the risk that more patient data become available that results in a different interpretation then the one derived from topline data; risks related to the Company’s interactions with regulatory authorities, which may affect the initiation, timing and progress of clinical trials and pathways to regulatory approval; the risk that the Company will be unable to align with the FDA, MHRA, or other regulatory authorities on an approval pathway for its gene therapy candidates, including AMT-130; whether the measurements that the Company is evaluating are viewed as robust and sensitive measurements of disease progression; whether RMAT designation, Breakthrough Therapy designation, or any accelerated pathway, if granted, will lead to regulatory approval; the Company’s ability to conduct and fund a Phase III or confirmatory study for AMT-130; the Company’s ability to continue to build and maintain the infrastructure and personnel needed to achieve its goals; the Company’s effectiveness in managing current and future clinical trials and regulatory processes; the Company’s ability to demonstrate the therapeutic benefits of its gene therapy candidates in clinical trials; the continued development and acceptance of gene therapies; the Company’s ability to obtain, maintain and protect its intellectual property; and the Company’s ability to fund its operations and to raise additional capital as needed and on acceptable terms. 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 its Annual Reports on Form 10-K, its 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, except as required by law, the Company assumes no obligation to update these forward-looking statements, even if new information becomes available in the future.

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uniQure Contacts:

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FOR INVESTORS:	FOR MEDIA:
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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

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Filing Exhibits & Attachments

4 documents

Press Releases

• EX-99.1 EX-99.1 19.6 KB

Other Documents

• EX-101 EX-101.SCH 2.6 KB
• EX-101 EX-101.LAB 14.9 KB
• EX-101 EX-101.PRE 9.7 KB