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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):
October 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.) |
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
November 2, 2026, uniQure N.V. (the “Company”) issued a press release announcing the U.S. Food and Drug Administration
(“FDA”) has accepted for review the Biologics License Application (“BLA”) for ifezuntirgene inilparvovec,
an investigational gene therapy for the treatment of Huntington’s disease submitted under the Accelerated Approval pathway.
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 (the “Securities Act”), or the Exchange Act, regardless
of the general incorporation language of such filing, except to the extent that such filing incorporates by reference any or all of such
information by express reference.
Item 8.01 Other Events.
On
November 2, 2026, the Company announced the FDA has accepted for review the BLA for ifezuntirgene inilparvovec, an investigational
gene therapy for the treatment of Huntington’s disease submitted under the Accelerated Approval pathway. The application has
been granted Priority Review with a Prescription Drug User Fee Act (“PDUFA”) target action date of May 1, 2027. In
its acceptance letter, the FDA stated that they are tentatively planning to discuss this application at an Advisory Committee meeting.
The BLA is supported by the previously announced
36-month data analysis from the Phase I/II clinical study of ifezuntirgene inilparvovec, compared to a propensity score-matched external
control derived from the Enroll-HD natural history database, as previously announced on September 24, 2025.
The
Company and FDA have aligned on key elements of the confirmatory study design. The study is designed as a randomized, standard-of-care
trial enrolling approximately 200 patients in a 2:1 ratio of ifezuntirgene inilparvovec to standard of care, with Total Functional
Capacity (“TFC”) as the primary endpoint at 36 months. The Company plans to initiate patient screening in the U.S.
by the end of 2026.
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.
Forward-Looking Statements
This
Current Report on Form 8-K 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 review and approval of its biologics license application (“BLA”) for ifezuntirgene inilparvovec; expectations
regarding the adequacy of clinical data to support the marketing application and approval of ifezuntirgene inilparvovec; expectations
regarding timing of receiving potential approval of, ifezuntirgene inilparvovec; whether the FDA will hold an advisory committee to discuss
the BLA and the timing of such review and approval based on the Prescription Drug User Fee Act (“PDUFA”) target action date
of May 1, 2027 for the BLA; and its belief related to the potential for ifezuntirgene inilparvovec, if approved, to meaningfully reduce
disease progression based on the Company’s 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: the risk that the FDA ultimately concludes that the Phase I/II trial data and/or the confirmatory trial data are
not sufficient to support 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 pathways to regulatory approval; 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; and the Company’s ability to continue to build and maintain the infrastructure and personnel needed
to commercialize ifezuntirgene inilparvovec, if approved. 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.
Item 9.01 Financial Statements and Exhibits.
(d) Exhibits.
| Exhibit No. |
|
Description |
| |
|
|
| 99.1 |
|
Press Release of uniQure N.V. dated November 2, 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: November 2, 2026 |
By: |
/s/ Jeannette Potts |
| |
|
JEANNETTE POTTS |
| |
|
Chief Legal and Compliance Officer |
Exhibit 99.1
uniQure Announces
FDA Acceptance and Priority Review of the Biologics License Application for ifezuntirgene inilparvovec for the Treatment of Huntington’s
Disease
~ FDA sets PDUFA
target action date of May 1, 2027 ~
~ If approved,
ifezuntirgene inilparvovec will be the world’s first disease-modifying treatment
for Huntington’s
disease ~
Lexington, MA
and Amsterdam, the Netherlands, November 2, 2026 — uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing
transformative therapies for patients with severe medical needs, today announced the U.S. Food and Drug Administration (FDA) has accepted
for review the Biologics License Application (BLA) for ifezuntirgene inilparvovec, an investigational gene therapy for the treatment
of Huntington’s disease submitted under the Accelerated Approval pathway. The application has been granted Priority Review with
a Prescription Drug User Fee Act (PDUFA) target action date of May 1, 2027.
In its acceptance
letter, the FDA stated that they are tentatively planning to discuss this application at an Advisory Committee meeting. The Company
will be notified once the date and timelines have been finalized.
“The FDA’s
acceptance of our BLA for review marks an important milestone for the Huntington’s disease community, which has waited generations
for a treatment that changes the course of this devastating disease," stated Matt Kapusta, chief executive officer of uniQure.
"Backed by compelling data from our Phase I/II studies, we believe ifezuntirgene inilparvovec has the potential to become the first
disease modifying treatment for Huntington’s disease. With the FDA’s priority review underway, we will work in conjunction
with the Agency, clinicians, and the Huntington’s community to support the review process. The confirmatory trial remains a key
priority, with screening anticipated to begin by year end and the study is expected to be well underway by the time of potential approval.”
The BLA is supported
by the previously announced 36-month data analysis from the Phase I/II clinical study of ifezuntirgene inilparvovec, compared to a propensity
score-matched external control derived from the Enroll-HD natural history database, as previously announced on September 24, 2025.
The Company and
FDA have aligned on key elements of the confirmatory study design. The study is designed as a randomized, standard-of-care trial enrolling
approximately 200 patients in a 2:1 ratio of ifezuntirgene inilparvovec to standard of care, with Total Functional Capacity (TFC) as
the primary endpoint at 36 months. The Company plans to initiate patient screening in the U.S. by the end of 2026.
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
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).
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.1, EU2, and the UK3,
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
1
Yohrling G, et al. Neurology 2020;94(15 Suppl):954.
2
Medina A, et al. Mov Disord 2022;37(12):2327–2335
3
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," "expect," "intend," "look forward to,"
"may," "plan," "potential," “seek,” "should," "will" 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 review and approval of its biologics license application (“BLA”) for
ifezuntirgene inilparvovec; expectations regarding the adequacy of clinical
data to support the marketing application and approval of ifezuntirgene
inilparvovec; expectations regarding timing of receiving potential approval
of, ifezuntirgene inilparvovec; whether the FDA will hold an advisory committee
to discuss the BLA and the timing of such review and approval based on the Prescription Drug User Fee Act (“PDUFA”) target
action date of May 1, 2027 for the BLA; and its belief related to the potential for ifezuntirgene
inilparvovec, if approved, to meaningfully reduce disease progression based on the Company’s 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: the risk that the FDA ultimately concludes that the Phase I/II trial data and/or the confirmatory trial data are not sufficient
to support 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 pathways to regulatory approval; 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; and the Company’s ability to
continue to build and maintain the infrastructure and personnel needed to commercialize ifezuntirgene inilparvovec, if approved.
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 |
