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

uniQure (NASDAQ: QURE) held a Pre-Submission meeting with the UK MHRA and expects to file a Marketing Authorization Application (MAA) for AMT-130 in Huntington’s disease in Q3 2026.

Rhea-AI Summary

uniQure (NASDAQ: QURE) held a Pre-Submission meeting with the UK MHRA and expects to file a Marketing Authorization Application (MAA) for AMT-130 in Huntington’s disease in Q3 2026. The planned MAA will rely on three-year Phase I/II data showing a 75% slowing of disease progression at the high dose (p=0.003) versus a propensity score-matched external control and a generally manageable safety profile. uniQure also secured a Type B meeting with the U.S. FDA in Q2 2026 to discuss potential Phase III design and the four-year analysis expected in Q3 2026, and is pursuing additional international regulatory pathways.

Positive

• MAA submission for AMT-130 targeted in Q3 2026
• Three-year data showed 75% slowing of disease progression at high dose (p=0.003)
• Type B meeting with U.S. FDA granted in Q2 2026
• Active pursuit of additional ex-US regulatory pathways

Negative

• MAA planned on three-year Phase I/II data rather than completed Phase III evidence
• Primary comparison uses a propensity score-matched external control, which may attract regulatory scrutiny
• Key four-year analysis and Phase III design remain pending FDA feedback in Q2–Q3 2026

Argus Apr 30 session

+19.25% close to close Open Argus

Details

News Market Reaction – QURE

On Apr 30, the day this news came out, QURE closed 19.25% above the previous close.

Data tracked by StockTitan Argus for the Apr 30 session.

Key Figures

Slowing of progression: 75% P-value: p=0.003 Analysis timepoint: Three-year analysis +5 more

Slowing of progression

75%

Three-year AMT-130 high-dose data on composite UHDRS vs external control

P-value

p=0.003

Three-year AMT-130 composite UHDRS analysis

Analysis timepoint

Three-year analysis

Ongoing U.S. and European Phase I/II AMT-130 trials

Phase I/II trials

Phase I/II

Ongoing AMT-130 Huntington’s disease program in U.S. and Europe

Planned Phase III

Phase III

Potential AMT-130 trial design to be discussed with FDA Type B meeting

UK MAA timing

Q3 2026

Expected UK Marketing Authorization Application submission for AMT-130

FDA Type B timing

Q2 2026

Granted Type B meeting with U.S. FDA for AMT-130 Phase III discussion

Four-year analysis timing

Q3 2026

Planned four-year AMT-130 data analysis to support discussions

Historical Context

5 past events · Latest: Apr 28

5 events

1. Apr 28

   Earnings date notice

   24h Move

   +0.8%

   Announcement of timing for Q1 2026 financial results and conference call.

2. Mar 02

   Earnings and updates

   24h Move

   -32.8%

   2025 results with net loss and FDA recommending new sham-controlled AMT-130 study.

3. Mar 02

   Regulatory update

   24h Move

   -32.8%

   FDA said Phase I/II AMT-130 data versus external control not sufficient for filing.

4. Feb 23

   Earnings date notice

   24h Move

   +4.4%

   Scheduling of 2025 results release and related investor conference call.

5. Feb 06

   Clinical trial update

   24h Move

   +4.6%

   Updated AMT-191 Phase I/IIa data showing sustained α-Gal A increases in Fabry.

24h Move is the share-price change in the day after each event; other market factors may also have contributed.

Key Terms

marketing authorization application, maa, phase i/ii, phase iii, +4 more

8 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.

maa regulatory

"requirements to support a MAA submission for AMT-130"

MAA stands for Marketing Authorization Application, the formal request a drug developer files with regulators (commonly in the European Union) asking for permission to sell a medicine. Think of it like applying for a driver’s license for a product: approval means the company can market and earn revenue from the drug, while rejection or delays affect expected sales, timelines and the company’s valuation—so investors track MAAs as key risk/reward milestones.

phase i/ii medical

"ongoing U.S. and European Phase I/II clinical trials"

"Phase I/II" describes early stages of testing a new medicine or treatment, where researchers first evaluate its safety and then begin to see if it works. For investors, these phases are important because they indicate whether a product is progressing toward potential approval and commercialization, which can impact future value and success prospects. These stages help gauge how close a new treatment is to reaching the market.

phase iii medical

"discuss key elements of a potential Phase III trial design"

A Phase III trial is the late-stage clinical study that tests whether a medical treatment works and is safe in a large group of patients, often comparing it to standard care. Think of it as a final dress rehearsal or full-scale road test before regulators decide on approval; positive or negative results strongly influence a drug maker’s chance to sell the treatment, future revenue, and investment risk.

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.

medicines and healthcare products regulatory agency regulatory

"meeting with the United Kingdom’s (UK) Medicines and Healthcare products Regulatory Agency"

A medicines and healthcare products regulatory agency is a government body that assesses, licenses and monitors the safety, effectiveness and quality of medicines, vaccines and medical devices before and after they reach the market. For investors, its decisions act like a building inspector’s approval — they determine whether a product can be sold, the timing and scope of market access, and can therefore quickly affect a company’s revenue prospects, costs and valuation.

u.s. food and drug administration regulatory

"Type B meeting with the U.S. Food and Drug Administration in the second quarter"

The U.S. Food and Drug Administration is the federal agency that evaluates and enforces safety, effectiveness and labeling standards for medicines, medical devices, vaccines, food and related products before they reach consumers. For investors it matters because FDA approvals, warnings or recalls determine whether a product can be sold, how quickly it reaches the market and how costly compliance will be—changes that directly affect a company’s revenue, costs and stock value.

p=.003 technical

"external control (p=.003) and was generally well-tolerated"

p = .003 is a statistical shorthand meaning the observed result would be expected by random chance about 0.3% of the time if there were actually no real effect. For investors, that suggests the finding is unlikely to be a fluke and merits attention, but it does not measure how large or commercially important the effect is—think of it as a strong clue, not definitive proof.

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

See more from StockTitan in Google Search and AI answers. Adds StockTitan as a preferred source · opens Google

Add on Google Not now

~ Submission of a UK Marketing Authorization Application for AMT-130 is expected 
in the third quarter of 2026 ~

~ 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 ~

~ uniQure actively pursuing additional ex-US regulatory pathways to support potential registration of
AMT-130 in international markets ~

LEXINGTON, Mass. and AMSTERDAM, April 30, 2026 (GLOBE NEWSWIRE) -- 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.

“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.”

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.

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.

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 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.

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

FAQ

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

When will uniQure (QURE) submit the AMT-130 MAA for Huntington’s disease?

uniQure plans to submit the AMT-130 MAA in Q3 2026. According to uniQure, the filing will be based on the three-year analysis from ongoing U.S. and European Phase I/II trials.

What did the MHRA Pre-Submission meeting with uniQure (QURE) cover on April 30, 2026?

The MHRA meeting addressed the data package and manufacturing requirements for an MAA. According to uniQure, discussions focused on the three-year clinical analysis and supporting CMC elements for AMT-130.

What were the AMT-130 three-year clinical results uniQure (QURE) reported?

At year three, the high dose showed a 75% slowing of disease progression versus an external control (p=0.003). According to uniQure, the treatment was generally well-tolerated with a manageable safety profile.

What will uniQure (QURE) discuss at the Type B FDA meeting in Q2 2026?

uniQure will discuss potential Phase III trial design and the proposed statistical analysis plan for the four-year readout. According to uniQure, the meeting aims to seek FDA feedback on study design and analysis approach.

How is uniQure (QURE) approaching international regulatory strategy for AMT-130?

uniQure is actively pursuing additional ex-US regulatory pathways to support potential registration. According to uniQure, further updates on international regulatory engagement are expected in the second half of 2026.