Form 8-K
8-K — uniQure N.V.
Accession: 0001104659-26-111654
Filed: 2026-09-29
Period: 2026-09-29
CIK: 0001590560
SIC: 2834 (PHARMACEUTICAL PREPARATIONS)
Item: Regulation FD Disclosure
Item: Financial Statements and Exhibits
Documents
8-K — tm2626514d1_8k.htm (Primary)
EX-99.1 — EXHIBIT 99.1 (tm2626514d1_ex99-1.htm)
EX-99.2 — EXHIBIT 99.2 (tm2626514d1_ex99-2.htm)
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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):
September 29, 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 7.01 Regulation FD Disclosure.
On
September 29, 2026, uniQure N.V. (the “Company”) presented an update of its ongoing clinical trials of AMT-130
(the “Presentation”) and issued a press release which summarizes the Presentation (the “Release”).
Copies of the Presentation and Release are
furnished herewith as Exhibit 99.1 and Exhibit 99.2, respectively, and are incorporated into this Item 7.01 by reference.
The information in this Item 7.01 of this
Current Report on Form 8-K, the Presentation and the Release, shall not be deemed “filed” for purposes of Section 18 of the
Securities Exchange Act of 1934, as amended, or otherwise subject to the liabilities of that section or Sections 11 and 12(a)(2) of the
Securities Act of 1933, as amended. The information contained in this Item 7.01, and in the Presentation and the Release, shall not be
incorporated by reference into any filing with the Securities and Exchange Commission made by the Company, whether made before or after
the date hereof, regardless of any general incorporation language in such filing.
Item 9.01 Financial Statements and Exhibits.
(d) Exhibits.
Exhibit No.
Description
99.1
Presentation of uniQure N.V. dated September 29, 2026
99.2
Press Release of uniQure N.V. dated September 29, 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: September 29, 2026
By:
/s/ Jeannette Potts
JEANNETTE POTTS
Chief Legal and Compliance Officer
EX-99.1 — EXHIBIT 99.1
EX-99.1
Filename: tm2626514d1_ex99-1.htm · Sequence: 2
Exhibit 99.1
Ashley –
Huntington’s Disease
Community Advocate
Phase I/II ifezuntirgene
inilparvovec (AMT-130)
Huntington’s Disease
Program Update
September 29, 2026
LEADERSHIP IN GENE THERAPY SEPTEMBER 2026 | 2
This presentation contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as
amended. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as “anticipate,” “believe,” “could,” “estimate,” “expect,”
“goal,” “intend,” “look forward to,” “may,” “plan,” “potential,” “predict,” “project,” “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 only as of the date of this presentation. Examples of these forward-looking statements include, but
are not limited to, statements concerning: the timing of our meetings and discussions with regulatory authorities; our ability to continue accumulating long-term patient data; the potential clinical and
functional effects of AMT-130; the potential for accelerated regulatory pathways, including priority review, for AMT-130; our enrollment of a fourth cohort studying AMT-130 in patients with higher
striatal volumes compared to patients in prior cohorts; the utility of NfL in CSF as an effective biomarker and indicator of clinical severity; and the potential commercialization of AMT-130 and
statements related thereto, including our potential addressable market and potential treatment centers. Because these statements are subject to risks and uncertainties, our actual results could
differ materially from those expressed in these forward-looking statements. These risks and uncertainties include, among others: risks related to our clinical trials of AMT-130, including the risk that
such trials will be unable to demonstrate data sufficient to support further clinical development and the risk that interim or topline data from the trials may not be predictive of later data readouts;
risks related to our interactions with regulatory authorities, which may affect the initiation, timing and progress of clinical trials and pathways to regulatory approval; whether the measurements that
we are evaluating continue to be viewed as robust and sensitive measurements of disease progression; whether Regenerative Medicine Advance Therapy designation, Breakthrough Therapy
designation, or any accelerated pathway, if granted, will lead to regulatory approval; our ability to conduct and fund a Phase III or confirmatory study for AMT-130 if needed; our ability to continue to
build and maintain the infrastructure and personnel needed to achieve our goals, including commercialization of AMT-130 if approved; our effectiveness in managing current and future clinical trials
and regulatory processes; our ability to demonstrate the therapeutic benefits of our gene therapy candidates in clinical trials; the continued development and acceptance of gene therapies; our
ability to obtain, maintain and protect our intellectual property; and our ability to fund our operations and to raise additional capital as needed and on acceptable terms. These and other risks and
uncertainties are described more fully under the heading “Risk Factors” in our periodic filings with the U.S. Securities and Exchange Commission (“SEC”), including in our Annual Report on Form
10-K filed with the SEC on March 2, 2026 our Quarterly Reports on Form 10-Q filed with the SEC on May 5, 2026 and July 29, 2026, and 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 and, except as required by law, we assume no obligation to update
these forward-looking statements to reflect events that occur or circumstances that exist after the date on which they were made.
The ongoing Phase I/II AMT-130 clinical trials, from which the data herein are derived, are supplemented by two additional protocols, each with a statistical plan that was submitted to the FDA. The
new protocols, among other things, provide for the pooling of data across the ongoing U.S. and EU studies, and also prespecified the comparison of AMT-130 clinical end points compared to a
propensity score-matched external control from the updated Enroll-HD natural history data set.
Certain information contained in this presentation relates to or is based on studies, publications, surveys and other data obtained from third-party sources and our own internal estimates and
research. While we believe these third-party sources to be reliable as of the date of this presentation, we have not independently verified, and makes no representation as to the adequacy,
fairness, accuracy or completeness of, any information obtained from third -party sources. Finally, while we believe our own internal research is reliable, such research has not been verified by any
independent source.
Disclaimer
Opening Remarks
Matt Kapusta
Chief Executive Officer
LEADERSHIP IN GENE THERAPY SEPTEMBER 2026 | 4
AMT-130 High-Dose Continued to Demonstrate Meaningful Slowing of
Disease Progression
Substantial
treatment effect at
36 months in
updated analysis of
all 15 high-dose
patients,
strengthening the
data at the 36-
month timepoint
48-month analysis
showed continued
evidence of
meaningful slowing
of disease
progression
Dose-dependent
response consistent
with treatment effect
Survivor bias and
missingness in the
updated external
control likely
understate disease
progression and
treatment effect at
48 months
AMT-130 continues
to be generally well-tolerated
Review of Data Supporting
the BLA and MAA
Submissions:
June 2025 Data Cut:
36 Month Analysis of AMT-130
High Dose (N=12)
Walid Abi-Saab, M.D.
Chief Medical Officer
BLA, Biologics License Application; MAA, Marketing Authorization Application
LEADERSHIP IN GENE THERAPY SEPTEMBER 2026 | 6
36-Month Statistical Analysis Plan: Basis for BLA and MAA Submissions
12 Months 24 Months 36 Months
N=12 patients with 36-months
of follow-up as of June 30, 2025
Propensity score-matched
to AMT-130 high-dose arm
High-Dose AMT-130 Arm (N=17)
ENROLL-HD A Matched External Control Arm (N=940)
Low-Dose AMT-130 Arm (N=12)
ENROLL-HD A Matched External Control Arm (N=626)
N=12 patients with 36-months
of follow-up as of June 30, 2025
Propensity score-matched
to AMT-130 low-dose arm
Change from baseline
at 36-months vs
Enroll-HD propensity score-matched external control
• Composite Unified Huntington’s Disease Rating Scale (cUHDRS) PRIMARY
ENDPOINT
• Total Functional Capacity (TFC) • Symbol Digit Modalities Test (SDMT) • Stroop Word Reading Test (SWRT) • Total Motor Score (TMS)
SECONDARY
ENDPOINTS
Phase I/II Study Design of AMT-130
NCT05243017, NCT04120493
Abbreviations: BLA, Biologics License Application; MAA, Marketing Authorization Application; HD, Huntington’s disease
LEADERSHIP IN GENE THERAPY SEPTEMBER 2026 | 7
Basis for BLA and MAA Submissions
Statistically significant 60% slowing based on
TFC at 36 months (p=0.033)
Statistically significant 75% slowing based on
cUHDRS at 36 months (p=0.003)
Abbreviations: BLA, Biologics License Application; MAA, Marketing Authorization Application; cUHDRS, composite Unified Huntington’s Disease Rating Scale; TFC, Total Functional Capacity; HD, Huntington’s disease, SE, standard error; LSM, least
squares mean; BL, baseline References: Data on file. September 2025
Participants Baseline 36 Months
AMT-130 High-Dose 17 12
Enroll-HD A 940 568
Participants Baseline 36 Months
AMT-130 High-Dose 17 12
Enroll-HD A 940 583
cUHDRS Change from Baseline at 36 Months DISEASE WORSENING
TFC Change from Baseline at 36 Months
AMT-130 High-Dose Enroll-HD A
-0.38
-1.52
-2.5
-2.0
-1.5
-1.0
-0.5
0.0
p = 0.003
-0.36
-0.88
-1.5
-1.0
-0.5
0.0
p = 0.033
Data as of June 30, 2026
Primary Analysis: Results
at 48 Months from the
Phase I/II Study
LEADERSHIP IN GENE THERAPY SEPTEMBER 2026 | 9
Enroll-HD A – Release date January 2023; Enroll-HD B – Release date September 2025 Abbreviations: HD, Huntington’s disease
NCT05243017, NCT04120493
48-Month Statistical Analysis Plan Included Updated External Control
High-Dose AMT-130 Arm (N=17)
Updated ENROLL-HD B Matched External Control Arm (N=1,337)
12 Months 24 Months 36 Months
N=12 patients with 48-months
of follow-up as of June 30, 2026
Propensity score-matched
to AMT-130 high-dose arm
Enroll-HD Matched External Control*:
Enroll-HD A: ~20,000 participants
Enroll-HD B: ~26,000 participants
48 Months
Phase I/II Study Design of AMT-130
Change from baseline
at 48-months vs
Enroll-HD B propensity
score-matched external control • Composite Unified Huntington’s Disease Rating Scale (cUHDRS) PRIMARY
ENDPOINT
• Total Functional Capacity (TFC) • Symbol Digit Modalities Test (SDMT) • Stroop Word Reading Test (SWRT) • Total Motor Score (TMS)
SECONDARY
ENDPOINTS
LEADERSHIP IN GENE THERAPY SEPTEMBER 2026 | 10
Dose Dependence Sustained Through 48 Months
Above graph represents observed data. Abbreviations: cUHDRS, composite Unified Huntington’s Disease Rating Scale; TFC, Total Functional Capacity; SE, standard error; BL, baseline
References: Data on file. September 2026.
cUHDRS Change from Baseline Through 48 Months
-2.5
-2
-1.5
-1
-0.5
0
0.5
1
1.5
Baseline 12 Month 24 Months 36 Months 48 Months DISEASE WORSENING
Patients Baseline 12 Months 24 Months 36 Months 48 Months
AMT-130 High-Dose 2026 17 17 15 15 12
AMT-130 Low-Dose 12 12 12 12 12
AMT-130 High-Dose AMT-130 Low-Dose
Patients Baseline 12 Months 24 Months 36 Months 48 Months
AMT-130 High-Dose 2026 12 12 12 12 12
AMT-130 Low-Dose 12 12 12 12 12
cUHDRS Change from Baseline Through 48 Months
-2.5
-2
-1.5
-1
-0.5
0
0.5
1
1.5
Baseline 12 Months 24 Months 36 Months 48 Months
All Available Patients 48-Month Completers Only (n=12)
LEADERSHIP IN GENE THERAPY SEPTEMBER 2026 | 11
AMT-130 High-Dose Continued to Demonstrate Meaningful Slowing of
Disease Progression in cUHDRS and TFC at Month 48 DISEASE WORSENING
Abbreviations: cUHDRS, composite Unified Huntington’s Disease Rating Scale; HD, Huntington’s disease, SE, standard error; LSM, least squares mean; BL, baseline
References: Data on file. September 2026; * p values are nominal
44% slowing of disease progression
at 48 months (p=0.144*)
Participants Baseline 48 months
AMT-130 High-Dose 17 12
Enroll-HD B 1337 625
-0.90
-1.61
-2.5
-2
-1.5
-1
-0.5
0
cUHDRS Change from Baseline at 48 Months
AMT-130 High-Dose
N=17
Primary Analysis (compared to Enroll-HD B)
Enroll-HD B
N=1337
-0.37
-0.94
-1.6
-1.4
-1.2
-1
-0.8
-0.6
-0.4
-0.2
0
61% slowing of disease progression
at 48 months (p=0.008*)
Participants Baseline 48 months
AMT-130 High-Dose 17 12
Enroll-HD B 1337 641
TFC Change from Baseline at 48 Months
Primary Analysis (compared to Enroll-HD B)
AMT-130 High-Dose
N=17
Enroll-HD B
N=1337
LEADERSHIP IN GENE THERAPY SEPTEMBER 2026 | 12
Participants Baseline 12 months 24 months 36 months 48 months
AMT-130 High-Dose 17 17 15 15 12
Enroll-HD B 1337 967 772 704 641 DISEASE WORSENING
Participants Baseline 12 months 24 months 36 months 48 months
AMT-130 High-Dose 17 17 15 15 12
Enroll-HD B 1337 953 760 690 625
AMT-130 High-Dose Showed Meaningful Slowing of Disease Progression
Through 48 Months
Above graph represents observed data Abbreviations: cUHDRS, composite Unified Huntington’s Disease Rating Scale; TFC, Total Functional Capacity; SE, standard error; BL, baseline; HD, Huntington’s disease
References: Data on file. September 2026
cUHDRS Change from Baseline Through 48 Months TFC Change from Baseline Through 48 Months
-2.5
-2
-1.5
-1
-0.5
0
0.5
1
1.5
Baseline 12 Months 24 Months 36 Months 48 Months
-1.4
-1.2
-1
-0.8
-0.6
-0.4
-0.2
0
0.2
0.4
0.6
Baseline 12 Months 24 Months 36 Months 48 Months
AMT-130 High-Dose Enroll-HD B
LEADERSHIP IN GENE THERAPY SEPTEMBER 2026 | 13
Abbreviations: cUHDRS, composite Unified Huntington’s Disease Rating Scale; SDMT, Symbol Digit Modalities Test; SWRT, Stroop Word Reading Test; TMS, Total Motor Score; LSM, least squares mean; BL, baseline; SE, standard error; HD, Huntington’s disease
References: Data on file. September 2026; * p-values are nominal
Other Secondary Endpoints: High-Dose AMT-130 had Favorable Trends to
External Control Across the Other Components of the cUHDRS at 48 Months DISEASE WORSENING DISEASE WORSENING DISEASE WORSENING
Slowed HD progression by 20% based on SDMT
at 48 months (p=0.731*)
Slowed HD progression by 113% based on SWRT
at 48 months (p=0.006*)
Slowed HD progression by 5.6% based on TMS
at 48 months (p=0.908*)
SDMT Change from Baseline at 48 Months SWRT Change from Baseline at 48 Months TMS Change from Baseline at 48 Months
-2.75
-3.43
-9.0
-7.0
-5.0
-3.0
-1.0
1.0
3.0
5.0
0.95
-7.53
-10.0
-8.0
-6.0
-4.0
-2.0
0.0
2.0
4.0
6.0
4.82 5.10
0.0
2.0
4.0
6.0
8.0
AMT-130 High-Dose (N=12)
Enroll-HD B (N=634)
AMT-130 High-Dose (N=12)
Enroll-HD B (N=633)
AMT-130 High-Dose (N=12)
Enroll-HD B (N=631)
A Closer Look at
Enroll-HD B
External Control
Matched to High-Dose
LEADERSHIP IN GENE THERAPY SEPTEMBER 2026 | 15
Updated Matched Natural History Control Likely Understates Disease
Progression and Treatment Effect at Month 48
Enroll-HD B: Annualized cUHDRS Change from Baseline
By Length of Follow Up
• Participants with longer follow up declined more slowly, enriching the comparator with slower progressors
• Data missingness in Enroll-HD B reached 53% at Month 48
• These effects were more evident in Enroll-HD B than in Enroll-HD A
-0.91
-0.82
-0.73
-0.41
-1
-0.8
-0.6
-0.4
-0.2
0
Participants with
last data at Y1
Participants with
last data at Y2
Participants with
last data at Y3
Participants with
last data at Y4 or
more Annualized cUHDRS Change from Baseline
cUHDRS Trajectories over Time Change from baseline in cUHDRS
-2.5
-2
-1.5
-1
-0.5
0
0.5
1
1.5
Baseline Year 1 Year 2 Year 3 Year 4
Enroll-HD B Enroll-HD A
At 48 months,
Enroll-HD B decline
was 20% slower than
Enroll-HD A
-1.65
-2.05
Above analyses are post Abbreviations: cUHDRS, composite Unified Huntington’s Disease Rating Scale; HD, Huntington’s disease
Post Hoc Analysis
using Enroll-HD A at
48 Months from the
Phase I/II Study
Data as of June 30, 2026
LEADERSHIP IN GENE THERAPY SEPTEMBER 2026 | 17
-0.90
-1.94
-2.5
-2
-1.5
-1
-0.5
0
AMT-130 High-Dose Continued to Demonstrate Meaningful Slowing of
Disease Progression in cUHDRS at Month 48 DISEASE WORSENING
Abbreviations: cUHDRS, composite Unified Huntington’s Disease Rating Scale; HD, Huntington’s disease, SE, standard error; LSM, least squares mean; BL, baseline
References: Data on file. September 2026; * p values are nominal
54% slowing of disease progression
at 48 months (p=0.041*)
Participants Baseline 48 months
AMT-130 High-Dose 17 12
Enroll-HD A 619 349
cUHDRS Change from Baseline at 48 Months
AMT-130 High-Dose
N=17
Post Hoc Analysis (compared to Enroll-HD A)
Enroll-HD A
N=619
-0.39
-1.24
-1.6
-1.4
-1.2
-1
-0.8
-0.6
-0.4
-0.2
0
68% slowing of disease progression
at 48 months (p<0.001*)
Participants Baseline 48 months
AMT-130 High-Dose 17 12
Enroll-HD A 619 364
TFC Change from Baseline at 48 Months
Post Hoc Analysis (compared to Enroll-HD A)
AMT-130 High-Dose
N=17
Enroll-HD A
N=619
LEADERSHIP IN GENE THERAPY SEPTEMBER 2026 | 18 DISEASE WORSENING
Above graph represents observed data
Abbreviations: cUHDRS, composite Unified Huntington’s Disease Rating Scale; TFC, Total Functional Capacity; SE, standard error; BL, baseline References: Data on file. September 2026
cUHDRS Change from Baseline Through 48 Months TFC Change from Baseline Through 48 Months
-2.5
-2
-1.5
-1
-0.5
0
0.5
1
1.5
Baseline 12 Months 24 Months 36 Months 48 Months
-1.4
-1.2
-1
-0.8
-0.6
-0.4
-0.2
0
0.2
0.4
0.6
Baseline 12 Months 24 Months 36 Months 48 Months
AMT-130 High-Dose Enroll-HD B Enroll-HD A
AMT-130 High-Dose Showed Meaningful Slowing of Disease Progression
Through 48 Months
Participants Baseline 12 months 24 months 36 months 48 months
AMT-130 High-Dose 17 17 15 15 12
Enroll-HD B 1337 967 772 704 641
Enroll-HD A 619 492 401 360 364
Participants Baseline 12 months 24 months 36 months 48 months
AMT-130 High-Dose 17 17 15 15 12
Enroll-HD B 1337 953 760 690 625
Enroll-HD A 619 484 398 355 349
Results at 36 Months
from the Phase I/II
Study
Data as of June 30, 2026
LEADERSHIP IN GENE THERAPY SEPTEMBER 2026 | 20
-1.4
-1.2
-1
-0.8
-0.6
-0.4
-0.2
0
0.2
0.4
0.6
Baseline 12 Months 24 Months 36 Months
-2.5
-2
-1.5
-1
-0.5
0
0.5
1
1.5
Baseline 12 Months 24 Months 36 Months
Participants Baseline 12 months 24 months 36 months
AMT-130 High-Dose 2025 17 17 15 12
AMT-130 High-Dose Showed Meaningful Slowing of Disease Progression
Through 36 Months
Above graph represents observed data
Abbreviations: cUHDRS, composite Unified Huntington’s Disease Rating Scale; TFC, Total Functional Capacity; SE, standard error; BL, baseline References: Data on file. September 2026 DISEASE WORSENING
Participants Baseline 12 months 24 months 36 months
AMT-130 High-Dose 2025 17 17 15 12
cUHDRS Change from Baseline Through 36 Months TFC Change from Baseline Through 36 Months
AMT-130 High-Dose 2025 AMT-130 High-Dose 2025
LEADERSHIP IN GENE THERAPY SEPTEMBER 2026 | 21
-1.4
-1.2
-1
-0.8
-0.6
-0.4
-0.2
0
0.2
0.4
0.6
Baseline 12 Months 24 Months 36 Months
-2.5
-2
-1.5
-1
-0.5
0
0.5
1
1.5
Baseline 12 Months 24 Months 36 Months
Enroll-HD B
Participants Baseline 12 months 24 months 36 months
AMT-130 High-Dose 2026 17 17 15 15
AMT-130 High-Dose 2025 17 17 15 12
Enroll-HD B 1337 967 772 704
AMT-130 High-Dose Showed Meaningful Slowing of Disease Progression
with Three Additional Subjects Through 36 Months
Above graph represents observed data Abbreviations: cUHDRS, composite Unified Huntington’s Disease Rating Scale; TFC, Total Functional Capacity; SE, standard error; BL, baseline; HD, Huntington’s disease References: Data on file. September 2026 DISEASE WORSENING
Participants Baseline 12 months 24 months 36 months
AMT-130 High-Dose 2026 17 17 15 15
AMT-130 High-Dose 2025 17 17 15 12
Enroll-HD B 1337 953 760 690 Mean Change from BL cUHDRS (±SE) Mean Change from BL TFC (±SE)
cUHDRS Change from Baseline Through 36 Months TFC Change from Baseline Through 36 Months
AMT-130 High-Dose 2026
AMT-130 High-Dose 2025
Enroll-HD B
AMT-130 High-Dose 2026
AMT-130 High-Dose 2025
LEADERSHIP IN GENE THERAPY SEPTEMBER 2026 | 22
Abbreviations: cUHDRS, composite Unified Huntington’s Disease Rating Scale; HD, Huntington’s disease, TFC, Total Functional Capacity SE, standard error;; LSM, least squares mean; BL, baseline; HD, Huntington’s disease
References: Data on file. September 2026 * p values are nominal
AMT-130 high-dose significantly reduced
HD progression by 80% based on
cUHDRS at 36 months
cUHDRS Change from Baseline at 36 Months TFC Change from Baseline at 36 Months DISEASE WORSENING
p = 0.005*
AMT-130 High-Dose Showed Meaningful Slowing of Disease Progression
on cUHDRS and TFC at 36 Months
-0.27
-0.82
-2.5
-2.0
-1.5
-1.0
-0.5
0.0
p = 0.011*
AMT-130 high-dose significantly reduced
HD progression by 67% based on TFC at
36 months
-0.28
-1.39
-2.5
-2.0
-1.5
-1.0
-0.5
0.0
AMT-130 High-Dose (N=17)
Enroll-HD B (N=1337)
AMT-130 High-Dose (N=17)
Enroll-HD B (N=1337)
LEADERSHIP IN GENE THERAPY SEPTEMBER 2026 | 23
Abbreviations: cUHDRS, composite Unified Huntington’s Disease Rating Scale; SDMT, Symbol Digit Modalities Test; SWRT, Stroop Word Reading Test; TMS, Total Motor Score; LSM, least squares mean; BL, baseline; SE, standard error; HD, Huntington’s disease
References: Data on file. September 2026; * p-values are nominal
Other Secondary Endpoints: High-Dose AMT-130 was Favorable to External
Control Across the Other Components of the cUHDRS at 36 Months DISEASE WORSENING DISEASE WORSENING DISEASE WORSENING
Slowed HD progression by 124% based on SDMT
at 36 months (p=0.040*)
Slowed HD progression by 123% based on SWRT
at 36 months (p=0.047*)
Slowed HD progression by 59% based on TMS
at 36 months (p=0.114*)
SDMT Change from Baseline at 36 Months SWRT Change from Baseline at 36 Months TMS Change from Baseline at 36 Months
0.67
-2.78
-9.0
-7.0
-5.0
-3.0
-1.0
1.0
3.0
5.0
1.32
-5.75
-9.0
-7.0
-5.0
-3.0
-1.0
1.0
3.0
5.0
1.93
4.77
-9.0
-7.0
-5.0
-3.0
-1.0
1.0
3.0
5.0
AMT-130 High-Dose (N=17)
Enroll-HD B (N=1337)
AMT-130 High-Dose (N=17)
Enroll-HD B (N=1337)
AMT-130 High-Dose (N=17)
Enroll-HD B (N=1337)
Neurofilament
Light Chain (NfL)
LEADERSHIP IN GENE THERAPY SEPTEMBER 2026 | 25
-100
0
100
200
300
400
500
0 12 24 36 48 Percentage change in CSF NfL (SE)
Months
CSF NfL Percentage Change from Baseline (0-48 Months)
Patients Base 1M 3M 6M 9M 12M 15M 18M 24M 30M 36M 48M
High-Dose 17 17 16 16 15 14 14 15 15 14 14 11
Low-Dose 12 12 11 12 12 12 12 12 12 12 12 11 REDUCTION IN NEURODEGENERATION
Abbreviations: CSF, cerebrospinal fluid; NfL, neurofilament light chain References: Data on file. September 2026
References: 1) Rodrigues et al. Sci Transl Med 2021, Dr. Ed Wild, personal communication
CSF NfL Near Baseline at 48 Months
• CSF NfL levels maintained near baseline starting from
Month 18 and up to 4 years
• Mean CSF NfL was 4% above baseline for the high-dose
and 8% below baseline for the low-dose at Month 48
• Early manifest HD patients, when untreated, show a 10-
15% increase per year1
Safety
LEADERSHIP IN GENE THERAPY SEPTEMBER 2026 | 27
AMT-130 Was Generally Well-Tolerated
• Most common adverse events in the treatment groups related to
the administration procedure, which all resolved
• Since September 2025: - No new treatment-related serious adverse events (SAE)
reported in Cohorts 1 and 2
- One treatment-related SAE of CNS inflammation in a Cohort 4
patient, fully resolved following a short course of corticosteroids
- One suicide in low-dose, approximately five years after
treatment which was assessed as unrelated to treatment
AMT-130
was generally
well-tolerated
Abbreviations: CNS, central nervous system Data on file. September 2026
References: Kachian, et al (2021)
LEADERSHIP IN GENE THERAPY SEPTEMBER 2026 | 28
All AMT-130
(Cohorts 1, 2, 3 and 4)
(n=51)
High-dose AMT-130
(Cohort 4)
(n= 6)
Dose-Blinded AMT-130
(Cohort 3)
(n=12)
High-dose AMT-130
(Cohorts 2)
(n=20*)
Low-dose AMT-130
(Cohorts 1)
(n=13*)
Sham Surgery
(n=10)
N (%) N (%) N (%) N (%) N (%) N (%)
Any TEAEs 10 100.0 12 92.3 20 100.0 12 100 6 100 50 98
Any SAEs 1 10.0 4 30.8 10 50.0 4 33.3 2 33 20 39.2
Any SAEs (peri-operative) 1 10.0 2 15.4 6 30.0 0 0.0 1 16.7 9 17.6
Any Drug-Related TEAE 0 0.0 0 0.0 6 30.0 3 25.0 2 33.3 11 21.5
Any Drug-Related SAE 0 0.0 0 0.0 4 20.0 0 0.0 1 16.7 5 9.80
Most Common TEAEs (≥30% in at least one group)
Headache 3 30.0 2 15.4 9 45.0 6 50.0 4 66.7 21 41.2
Procedural headache 5 50.0 4 30.8 10 50.0 2 16.7 0 0.0 16 31.4
Procedural pain 6 60.0 2 15.4 7 35.0 3 25.0 1 16.7 13 25.5
Post lumbar puncture syndrome 6 60.0 2 15.4 6 30.0 2 16.7 0 0.0 10 19.6
Procedural complication 4 40.0 3 23.1 4 20.0 0 0.0 0 0.0 7 13.7
Anxiety 0 0.0 0 0.0 4 20.0 4 33.3 3 50.0 11 21.6
Constipation 0 0.0 0 0.0 2 10.0 6 50.0 1 16.7 9 17.6
Insomnia 0 0.0 1 7.7 1 5.0 6 50.0 2 33.3 10 19.6
Back pain 1 10.0 0 0.0 0 0.0 5 41.7 0 0.0 5 9.8
Procedural nausea 0 0.0 3 23.1 3 15.0 1 8.3 2 33.3 9 17.6
Paraesthesia 1 10.0 0 0.0 1 5.0 1 8.3 2 33.3 4 7.8
Sleep disorder 0 0.0 0 0.0 0 0.0 4 33.3 0 0.0 4 7.8
Sensory loss 0 0.0 0 0.0 0 0.0 1 8.3 2 33.3 3 5.9
AE, adverse event; N, number of patients; TEAE, treatment-emergent adverse event; SAE, serious adverse event. TEAEs are defined as AEs on or after Day 0.
Perioperative AEs had onset Day 0 to 13. Safety data as of June 30, 2026; * 1 low dose and 3 high dose cross-over patients included
AMT-130 Was Generally Well-Tolerated
Summary
LEADERSHIP IN GENE THERAPY SEPTEMBER 2026 | 30
AMT-130 High-Dose Continued to Demonstrate Meaningful Slowing of
Disease Progression
Substantial
treatment effect at
36 months in
updated analysis of
all 15 high-dose
patients,
strengthening the
data at the 36-
month timepoint
48-month analysis
showed continued
evidence of
meaningful slowing
of disease
progression
Dose-dependent
response consistent
with treatment effect
Survivor bias and
missingness in the
updated external
control likely
understate disease
progression and
treatment effect at
48 months
AMT-130 continues
to be generally well-tolerated
Clinician’s
Perspective
Victor Sung, M.D.
University of Alabama at Birmingham (UAB)
Research Analyst
Questions
Ashley –
Huntington’s Disease
Community Advocate
Closing
Remarks
Matt Kapusta
Chief Executive Officer
EX-99.2 — EXHIBIT 99.2
EX-99.2
Filename: tm2626514d1_ex99-2.htm · Sequence: 3
Exhibit 99.2
uniQure Announces
Additional Data from Ongoing Phase I/II Studies of ifezuntirgene inilparvovec (AMT-130) in Huntington’s Disease Showing Continued
Slowing of Disease Progression
~ In 12 high-dose
patients at 48 months, both cUHDRS and TFC continued to demonstrate meaningful slowing of disease progression and clear dose-dependent
response; the primary endpoint of cUHDRS showed 44% slowing of disease progression (non-significant p=0.144) and TFC showed a 61% slowing
of disease progression (nominal p=0.008) ~
~ Updated data
reflecting all 15 high-dose patients showed substantial treatment effect on both cUHDRS and TFC at 36 months, the timepoint that is the
regulatory anchor for the submitted BLA and the confirmatory study; the new analysis demonstrated 80% slowing of disease progression
based on cUHDRS (nominal p=0.005) and 67% based on TFC (nominal p=0.011) ~
~
Treatment effect at 48 months likely understated by substantial missing data and survivor bias in updated external control; post hoc
analysis using prior external control showed slowing of disease of 54% on cUHDRS (nominal p=0.041) and 68% on TFC (nominal p=0.001) at
48 months ~
~ These positive
data are meaningful for Huntington's disease patients who currently have no approved disease-modifying treatment options ~
~ Investor conference
call and webcast today at 8:30 a.m. ET ~
Lexington, MA
and Amsterdam, the Netherlands, September 29, 2026 — uniQure N.V. (NASDAQ: QURE), a leading
gene therapy company advancing transformative therapies for patients with severe medical needs, today announced additional data from
the ongoing Phase I/II studies of ifezuntirgene inilparvovec for the treatment of Huntington’s disease.
“Four years
after a single administration, ifezuntirgene inilparvovec continues to show meaningful slowing of disease progression, further strengthening
our conviction in its benefit for people living with Huntington’s disease," stated Walid Abi-Saab,
M.D., chief medical officer of uniQure. "At 48 months, Total Functional Capacity (TFC), the primary measure of our confirmatory
study, demonstrated consistent slowing of functional decline, with the absolute treatment benefit maintained in Month 48. Furthermore,
the observed differences between the high and low doses on both composite Unified Huntington’s Disease Rating Scale (cUHDRS) and
TFC are consistent with a dose-dependent treatment effect. The updated 36-month analysis, which now includes three additional high-dose
patients, showed a substantial effect on cUHDRS and TFC, further reinforcing the data included in our license applications. We believe
these data are clinically meaningful for Huntington’s disease patients, and we look forward to presenting them at a future scientific
meeting.”
Topline Clinical
Data at 36 months and 48 months1
Today's announcement
comprises results from the ongoing Phase I/II studies at two timepoints, with a data cutoff of June 30, 2026. Twenty-nine patients
have been treated with ifezuntirgene inilparvovec (n=17 high dose; n=12 low dose) across the studies’ first two cohorts. The new
36-month analysis now includes 15 high-dose and 12 low-dose patients, with three additional high-dose patients having reached that timepoint
since the September 2025 analysis. The 48-month analysis includes 12 patients at each dose.
Outcomes for the
36-month and 48-month analyses were compared to propensity score-matched external controls (n=1,337 for high dose) from an updated ENROLL-HD
natural history dataset with a September 2025 cutoff. As is common with longitudinal natural history studies, missingness of data
increased with duration of follow-up, reaching 53% in the updated ENROLL-HD matched controls at 48 months. The Company’s analysis
of the updated control showed that patients discontinuing follow-up were progressing materially faster than those remaining in the control
group. The Company believes these factors likely understated disease progression in the control and the resulting treatment effect of
ifezuntirgene inilparvovec at 48 months.
At the June 2026
Type B meeting, the FDA communicated that the 36-month data from 12 high-dose patients would be acceptable as the primary basis for the
BLA submission under the accelerated approval pathway, and the submission was made accordingly. The BLA submission of ifezuntirgene inilparvovec
predated the topline results announced today and these results were not part of the submission.
High-Dose Results
at 36 Months (n=15)
· cUHDRS
showed 80% slowing of disease progression compared to the updated external control (nominal
p=0.005). Treated patients had a mean change in cUHDRS from baseline of -0.28 compared to
a change of -1.39 for the external control, a favorable treatment difference of 1.12 compared
to baseline.
· TFC
showed 67% slowing of disease progression compared to the updated external control (nominal
p=0.011). Treated patients had a mean change in TFC from baseline of -0.27 compared to a
change of -0.82 for patients in the propensity score-matched external control, a favorable
treatment difference of 0.55 compared to baseline.
· Mean cerebrospinal neurofilament light protein (CSF NfL) was 6% below baseline
(n=14).
High-Dose Results
at 48 Months (n=12)
· cUHDRS
showed a 44% slowing of disease progression compared to the updated external control and
did not reach statistical significance (p=0.144). Treated patients had a mean change in cUHDRS
from baseline of -0.90 compared to a change of -1.61 for the external control, a favorable
treatment difference of 0.71 compared to baseline.
· TFC
showed 61% slowing of disease progression compared to the updated external control (nominal
p=0.008). Treated patients had a mean change in TFC from baseline of -0.37 compared to a
change of -0.94 for the external control, a favorable treatment difference of 0.57 compared
to baseline.
1
cUHDRS is the pre-specified primary endpoint for the 48 months analysis; all other p-values are nominal.
The 36-month data included 15 high-dose patients; the 48-month analysis included 12 high-dose patients. Percentage slowing was sensitive
to the magnitude of decline in the external control.
· In
a post-hoc sensitivity analysis using the prior ENROLL-HD external control, the 48-month
analysis showed 53.5% slowing based on cUHDRS (nominal p=0.041) and 68.3% based on TFC (nominal
p=0.001).
· Mean
cerebrospinal neurofilament light protein (CSF NfL) was 4% above baseline (n=11).
Dose Comparison at 48 Months (high
dose n=12, low dose n=12)
· The
observed differences between the high and low doses are consistent with a dose-dependent
treatment effect. At 48 months, mean change from baseline in cUHDRS was –0.91 in high-dose
patients compared with −1.94 in low-dose patients, a difference of 1.03 in favor of
the high dose. Mean change from baseline in TFC was −0.30 in high-dose patients compared
with −0.70 in low-dose patients, a difference of 0.40 in favor of the high dose.
"Huntington’s
disease does not slow on its own; the biology is one of inevitable progressive decline," stated Victor Sung, M.D., professor
of neurology at the University of Alabama at Birmingham (UAB), director of the UAB Huntington’s Disease Clinic.
“What I find particularly notable in the expanded data is the consistently meaningful treatment effect at 36 months, and the apparent
stability of the functional capacity benefit through Month 48. We see this even as the rate of decline in the updated comparator dataset
slowed, an anticipated shift which appears to reflect some attrition in the longitudinal external control data, and which does not reflect
the typical clinical presentation of accelerating decline over time. Total Functional Capacity tracks things that matter the most to
patients and families – ability to work, perform household chores and handle daily self-care activities. Seeing that treatment
difference maintained at four years is meaningful for people living with this relentlessly progressive degenerative disease.”
Ifezuntirgene inilparvovec
continues to be generally well-tolerated, with a manageable safety profile at both doses. The most common adverse events in the treatment
groups were related to the administration procedure, and all have resolved. As previously disclosed, five high dose participants (17%)
experienced a treatment-related serious adverse event (SAE) related to central nervous system inflammation, all of which fully resolved.
Since the September 2025
data readout, one suicide in a low-dose patient occurred, approximately five years after receiving treatment. This was assessed as unrelated
to treatment by the study investigator. Suicidal ideation and completed suicide occur at substantially elevated rates in Huntington’s
disease relative to the general population, and suicide is among the leading causes of death in those with the disease.
Investor
Conference Call and Webcast Information
uniQure management
will host an investor conference call and webcast today, Tuesday, September 29 at 8:30 a.m. ET. The event will be
webcast under the Events & Presentations section of uniQure’s website at https://www.uniqure.com/investors-media/events-presentations,
and following the event a replay will be archived for 90 days. Analysts wishing to participate in the question and answer session should
access the live call by dialing (646) 307-1963 or toll-free (800) 715-9871 and entering conference ID 5075555. If you are joining the
conference call, please join 15 minutes before the start time.
About
Ifezuntirgene Inilparvovec (AMT-130)
Ifezuntirgene inilparvovec
is a novel gene therapy candidate for the treatment of Huntington’s disease, which utilizes a proprietary, gene-silencing miQURE®
platform and incorporates a miRNA, specifically designed to silence the huntingtin gene and the potentially highly toxic exon 1 protein
fragment. Treated patients receive a single administration through targeted, MRI-guided, convection-enhanced stereotactic neurosurgical
delivery directly into the striatum (caudate and putamen). Ifezuntirgene inilparvovec is the first investigational therapy for Huntington’s
disease to have received Breakthrough Therapy and Regenerative Medicine Advanced Therapy (RMAT) designations from the FDA. Ifezuntirgene
inilparvovec also holds Fast Track designation from the FDA.
About the Phase
I/II Clinical Program of Ifezuntirgene Inilparvovec
uniQure is conducting
two multi-center Phase I/II clinical studies evaluating the safety, tolerability, and efficacy of ifezuntirgene inilparvovec for the
treatment of Huntington’s disease.
In total, the Phase
I/II clinical studies have dosed 51 patients with ifezuntirgene inilparvovec. The U.S. randomized study enrolled 26 patients who received
either a single administration of ifezuntirgene inilparvovec (n=6 low dose; n=10 high dose) or a sham procedure (n=10); four control
patients subsequently crossed over to treatment after approximately 12 months. The European open-label study enrolled 13 patients (n=6
low dose; n=7 high dose). A third cohort of 12 patients explored both doses in combination with immunosuppression, and a fourth cohort
of six U.S. patients is evaluating the high dose in patients with lower striatal volumes compared to those enrolled in previous cohorts.
Additional details
are available on www.clinicaltrials.gov (NCT05243017, NCT04120493)
About Huntington’s Disease
Huntington’s
disease is a rare, inherited neurodegenerative disorder that leads to motor symptoms including chorea, behavioral abnormalities and cognitive
decline resulting in progressive physical and mental deterioration. The disease is an autosomal dominant condition with a disease-causing
CAG repeat expansion in the first exon of the huntingtin gene that leads to the production and aggregation of abnormal protein in the
brain. Approximately 75,000 people have Huntington’s disease in the U.S.2, EU3, and the UK4,
with hundreds of thousands of others at risk of inheriting the disease. Despite the clear etiology of Huntington’s disease, there
are currently no approved therapies to delay the onset or to slow the disease’s progression.
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, Fabry disease, and other severe diseases. www.uniQure.com
2
Yohrling G, et al. Neurology 2020;94(15 Suppl):954.
3
Medina A, et al. Mov Disord 2022;37(12):2327–2335
4
Furby H, et al. Eur J Neurol 2022;29(8):2249–2257.
uniQure Forward-Looking
Statements
This press release
contains forward-looking statements within the meaning of Section 27A of the Securities Act and Section 21E of the Exchange
Act. 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
only as of the date of this report. Examples of these forward-looking statements include, but are not limited to, statements concerning:
the Company’s beliefs related to the factors that likely understate disease progression in the updated ENROLL-HD controls and the
Company’s belief that ifezuntirgene inilparvovec meaningfully reduces disease progression based on its evaluation and view of certain
measurements of disease progression. 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/II clinical
trials of ifezuntirgene inilparvovec, including the risk that such trials will be unable to continue to demonstrate data sufficient to
support further clinical development or regulatory approval; the risk that the FDA ultimately concludes that the Phase I/II trial data
are not sufficient to support a BLA or accelerated approval; the risk that more patient data become available that results in a different
interpretation than the one derived from the year three and four data analyses, respectively; 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;
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 any required confirmatory study for ifezuntirgene inilparvovec; the Company’s ability to successfully
complete any required confirmatory study for ifezuntirgene inilparvovec; the risk that accelerated approval, if granted, may be subject
to post-approval requirements that are difficult or costly to satisfy; the Company’s ability to continue to build and maintain
the infrastructure and personnel needed to commercialize ifezuntirgene inilparvovec, if approved; 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 Report on Form 10-K filed with the SEC on March 2, 2026, its Quarterly Report on Forms 10-Q filed with the SEC on
May 5, 2026 and July 29, 2026, respectively, 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
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Sep. 29, 2026
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