QURE

EX-99.1

2 qure-20260729xex99d1.htm

EX-99.1

Exhibit 99.1

uniQure Announces Second Quarter 2026 Financial Results and Provides Company Update

~ U.S. and U.K. regulatory submissions for AMT-130 for Huntington’s disease on track as planned for the third quarter of 2026 ~

~ Topline four-year data from the Phase I/II study of AMT-130 expected in September 2026 ~

~ Reported data from the first cohort in the Phase I/IIa trial of AMT-260 for refractory mesial temporal lobe epilepsy showed early biological signals of therapeutic activity and a favorable safety profile ~

~ Strengthened financial position with $259 million follow-on offering, extending cash runway into 2030 and funding the anticipated commercial launch of AMT-130 and continued pipeline investment ~

~ uniQure to host earnings call at 8:30 a.m. ET ~

Lexington, MA and Amsterdam, the Netherlands, July 29, 2026 — uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today reported its financial results for the second quarter of 2026 and highlighted recent progress across its business.

“This has been a defining quarter – not just for uniQure, but for the Huntington's disease community,” said Matthew Kapusta, chief executive officer at uniQure. “Following a productive Type B meeting with the FDA, we remain on track to submit our BLA for AMT-130 in the third quarter — a milestone that reflects years of rigorous science, disciplined execution, and an unwavering commitment to the patients and families living with this devastating disease. With four-year data expected in September and the U.K. regulatory activities progressing as planned, we enter the second half of 2026 with real momentum and a clear line of sight to potentially bringing this therapy to the people who need it.”

“Beyond AMT-130, we continue to execute across our broader pipeline,” Mr. Kapusta continued. “With a strong balance sheet, we believe we are well-positioned for what will be a transformative period for uniQure and the patients we serve.”

Recent Company Developments and Updates

Advancing AMT-130 for the treatment of Huntington’s disease

●In June 2026, the Company held a Type B meeting with the U.S. Food and Drug Administration (FDA). Official meeting minutes received in July 2026 confirmed that the FDA and the Company reached alignment that a Biologics License Application (BLA) submission under the accelerated approval pathway for AMT-130, based on the existing clinical data, is reasonable. In addition, the FDA seeks to align on the confirmatory study design prior to the BLA submission, including consideration of a randomized standard-of-care control design instead of a sham procedure. The FDA also stated that, in accordance with the FDA’s draft public guidance for accelerated approvals, the confirmatory study should be feasible to conduct within a reasonable timeline and be well underway, and potentially fully enrolled, at the time of accelerated approval.

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●Discussions with the FDA to align on the confirmatory study design and analysis are underway and the Company expects to submit a BLA in the third quarter of 2026. The Company plans to initiate the confirmatory study as expeditiously as possible after aligning with the FDA on the trial design.

●In September 2026, the Company plans to present data from its ongoing Phase I/II studies of AMT-130.  The update is expected to include follow-up data on all patients treated with AMT-130 in the first two cohorts, including four years of follow-up on 24 patients (12 patients at the high-dose and 12 patients at the low dose).

●In March 2026, the Company held a successful pre-submission meeting with the United Kingdom’s (U.K.) Medicines and Healthcare products Regulatory Agency (MHRA) and the regulatory submission is progressing as planned for the third quarter of 2026.

Continued clinical progress in pipeline programs

AMT-260 for the treatment of refractory mesial temporal lobe epilepsy (MTLE)

●In June 2026, the Company announced preliminary six-month follow-up data on the first, low dose cohort of six patients in the ongoing Phase I/IIa study.

oAs of the May 29, 2026 data cutoff date, three of six patients in the first, low-dose cohort 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.

oAs of the June 19, 2026 presentation date, there were 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.

●The Company expects t

EX-99.1

2 qure-20260505xex99d1.htm

EX-99.1

Exhibit 99.1

uniQure Announces First Quarter 2026 Financial Results and Provides Recent Company Updates

~ Advancing FDA interactions on AMT-130 for Huntington’s disease; Type B meeting scheduled for the second quarter of 2026 ~

~ Progressing AMT-130 toward expected UK regulatory submission; MAA on track for third quarter of 2026 following successful pre-submission meeting with UK MHRA~

~ Enrollment in AMT-260 temporal lobe epilepsy program on track; clinical update from first cohort in Phase I/IIa study to be presented at the Epilepsy Foundation Pipeline Conference ~

~ Presented updated data from AMT-191 Phase I/IIa in Fabry disease study showed sustained increases in α-Gal A Enzyme Activity and stable Lyso-Gb3 levels; subsequently all 11 dosed patients have discontinued enzyme replacement therapy ~

~ Strong balance sheet with $586.6 million in cash, cash equivalents and current investment securities as of March 31, 2026 and runway into the second half of 2029 ~

~ uniQure to host earnings call at 8:30 a.m. ET ~

Lexington, MA and Amsterdam, the Netherlands, May 5, 2026 — uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today reported its financial results for the first quarter of 2026 and highlighted recent progress across its business.

“During the first quarter of 2026, we remained focused on advancing AMT-130 to patients globally as rapidly as possible while executing across our broader pipeline,” said Matthew Kapusta, chief executive officer at uniQure. “We believe our data continue to support the potential for AMT-130 to fundamentally change the treatment landscape for Huntington’s disease, and we look forward to continued engagement with the FDA. In parallel, following a constructive interaction with the MHRA, we are preparing to submit an MAA in the third quarter and evaluating additional international opportunities.”

“We expect to deliver key clinical updates throughout 2026, including data from our AMT-260 program in refractory mesial temporal lobe epilepsy later in the second quarter and four-year AMT-130 data analysis in the third quarter,” Mr. Kapusta continued. “With these important milestones ahead, we remain committed to advancing our programs with urgency while maintaining disciplined capital allocation to drive long-term shareholder value.”

Recent Company Developments and Updates

Advancing AMT-130 for the treatment of Huntington’s disease

●The Company held a Type A meeting with the U.S. Food and Drug Administration (FDA) in January 2026 to discuss the regulatory path forward following an October 2025 pre-Biologics License Application (BLA) meeting. Following receipt of final meeting minutes from the Type A meeting, the Company announced that the FDA stated it cannot agree that data from the Phase

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I/II studies, compared to an external control, are sufficient to provide the primary evidence of effectiveness required to support a marketing application for AMT-130.

●The Company has been granted a Type B meeting with the FDA and plans to discuss key elements of a potential new clinical trial design and to solicit feedback on the proposed statistical analysis plan for the four-year data expected in the third quarter of 2026.

●Following a constructive pre-submission meeting with the United Kingdom’s (UK) Medicines and Healthcare products Regulatory Agency (MHRA), the Company expects to submit a Marketing Authorization Application (MAA) for AMT-130 based on three-year data in the third quarter of 2026.

Continued clinical progress in pipeline programs

AMT-260 for the treatment of refractory mesial temporal lobe epilepsy (MTLE)

●In 2025, the Company completed enrollment in the first dose cohort of six patients in the Phase I/IIa study. Enrollment is ongoing in a second dose cohort, expected to include an additional six patients. Enrollment of the second cohort is expected to be completed in mid-2026.

●The Company expects to provide data from the first cohort of six patients in the Phase I/IIa study with up to six months of follow-up at the Epilepsy Foundation Pipeline Conference, June 18 -19, 2026.

AMT-191 for the treatment of Fabry disease

●In February 2026, the Company presented updated safety and exploratory efficacy data from the Phase I/II study of AMT-191 in Fabry disease (data cutoff as of January 8, 2026):

oDose-dependent elevations were observed across 11 patients in three dose levels with α-Gal A activity ranging from 0.34- to 82.2-fold above mean normal range1 at the lowest dose, 1.6- to 312.5-fold at the mid dose, and 27.7- to 223.7-fold at the highest dose. These increases were durable across follow-up periods ranging from four months to more than one year.

oPlasma lyso-Gb3 levels were stable post-dose across all cohorts, regardless of enzyme replacement therapy (ERT) status.

●As of February 18, 2026, all 11 dosed patients were withdr

uniQure N.V._March 2, 2026 000159056000-0000000false00015905602026-03-022026-03-02 ​ ​

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): March 2, 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.)

​

Paasheuvelweg 25a, 1105 BP Amsterdam, The Netherlands ​ N/A

(Address of Principal Executive Offices) (Zip Code)

​ 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

​ 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 2.02 Results of Operations and Financial Condition.

​ On March 2, 2026, uniQure N.V. (the “Company”) issued a press release announcing its financial results for the quarter and full year ended December 31, 2025 and providing a corporate update. A copy of the press release is furnished as Exhibit 99.1 to this Current Report on Form 8-K and is incorporated herein by reference. ​ The information furnished pursuant to this Item 2.02, including Exhibit 99.1, shall not be deemed “filed” for purposes of Section 18 of the Securities Exchange Act of 1934, as amended (the “Exchange Act”), or otherwise subject to the liabilities under that section, and shall not be deemed to be incorporated by reference into any filing of the Company under the Securities Act of 1933, as amended (the “Securities Act”), or the Exchange Act, except as shall be expressly set forth by specific reference in such filing. ​ Item 7.01 Regulation FD Disclosure. ​ On March 2, 2026, the Company issued a press release announcing a regulatory update on AMT-130, the Company’s investigational gene therapy for Huntington’s disease. A copy of the press release is being furnished as Exhibit 99.2 to this Current Report on Form 8-K and is incorporated herein by reference. ​ The information furnished pursuant to this Item 7.01, including Exhibit 99.2, shall not be deemed “filed” for purposes of Section 18 of the Exchange Act or otherwise subject to the liabilities under that section, and shall not be deemed to be incorporated by reference into any filing of the Company under the Securities Act or the Exchange Act, except as shall be expressly set forth by specific reference in such filing. ​ ​ ​ Item 9.01 Financial Statements and Exhibits. ​ (d) Exhibits. ​

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Exhibit No. ​ ​ ​ Description

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99.1 ​ Press Release of uniQure N.V. regarding its financial results dated March 2, 2026

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99.2 ​ Press Release of uniQure N.V. regarding a regulatory update dated March 2, 2026

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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. ​

UNIQURE N.V.

Date: March 2, 2026 By: /s/ JEANNETTE POTTS

Jeannette Potts

Chief Legal and Compliance Officer

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