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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):
December 19, 2023
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: +31-20-566-7394
(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 December 19, 2023, uniQure N.V. (the
“Company”) issued a press release announcing updates on its ongoing clinical trials of AMT-130 for the
treatment of Huntington’s disease, as described in more detail in Item 8.01 below. The
Company also announced that it will host an investor call and webcast beginning at 8:30 a.m. Eastern
Time on the same date, during which the Company will discuss these interim updates. A copy of the press release is being furnished
as Exhibit 99.1 to this Current Report on Form 8-K and is incorporated herein by reference. The virtual event can be accessed
via the Events and Presentations section of the Company’s website at
https://www.uniqure.com/investors-media/events-presentations, and will be available for replay for 90 days following the event. The
Company’s website and any information contained on the website are not incorporated into this Current Report on Form 8-K.
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.
On December 19, 2023, the Company announced updated interim data
from its ongoing clinical trials of AMT-130, a one-time administered investigational gene therapy for the treatment of Huntington’s
disease. The interim data include up to 30 months of follow-up data from the 39 patients enrolled in the Company’s ongoing
U.S. and European Phase I/II clinical trials.
In the multi-center, Phase I/II clinical trial of AMT-130 being
conducted in the U.S., 26 patients with early-manifest Huntington’s disease have been enrolled, including an initial
10-patient low-dose cohort (6 treated patients, 4 control patients) with up to 30-months of follow-up data and a subsequent
16-patient high-dose cohort (10 treated patients, 6 control patients) with up to 18 months of follow-up data. Patients in the U.S.
study were randomized to either treatment with AMT-130 or an imitation (sham) surgery. The U.S. trial consists of a blinded 12-month
core study period followed by unblinded long-term follow-up period of five years for treated patients. Four of the six control
patients in the high-dose cohort were crossed over to treatment following the initial 12-month core study period and the remaining
two patients failed to meet the trial’s inclusion criteria.
In the multi-center, open-label, Phase I/II clinical trial of AMT-130
being conducted in Europe and the United Kingdom, 13 patients with the same early-manifest criteria for Huntington’s disease as
the U.S. study have been enrolled. Six of these patients were treated with AMT-130 in the initial low-dose cohort and seven patients
were treated in the subsequent high-dose cohort.
The combined U.S. and European interim data discussed in Item 8.01
of this Current Report on Form 8-K are subject to a September 30, 2023 cut off date and do not include outcome or biomarker data from
the control patients who have crossed over to treatment with AMT-130 following the 12-month core study period.
Exploratory Interim Efficacy Data
Clinical and functional measurements for treated patients in each
dose cohort were compared to baseline measurements as well as to control patients (for which up to 12 months of data is available)
and a non-concurrent criteria-matched natural history cohort, which was developed by the Company in collaboration with the Cure
Huntington’s Disease Initiative (CHDI) using the TRACK-HD natural history study of patients with early Huntington’s
disease. The natural history cohort includes 31 patients that met the Company’s clinical trial inclusion criteria of Total
Functional Capacity, Diagnostic Classification Level and minimum striatal volumes.
| · | Updated interim clinical
data through 30 months for the low-dose cohort and 18 months for the high-dose cohort show
evidence of potential dose-dependent clinical benefit relative to the non-concurrent criteria-matched
natural history cohort. |
| · | For patients receiving
the high dose, neurological function as measured by composite Unified Huntington’s
Disease Rating Scale (cUHDRS) and each of its individual components was preserved or improved
at 18 months compared to pre-treatment baseline measurements. |
| · | For patients receiving
the low dose, neurological function as measured by Total Motor Score (TMS) and Total Functional
Capacity (TFC) was preserved at 30 months compared to pre-treatment baseline measurements. |
| · | When compared to the
expected rate of decline from the a natural history cohort, AMT-130 showed favorable trends in
cUHDRS, TFC and TMS: |
| o | cUHDRS: AMT-130
showed a favorable difference in cUHDRS of 0.39 points at 30 months and 1.24 points at 18
months for the low- and high-dose, respectively (baseline values: 14.1 in low-dose and 14.9
in high-dose). |
| o | TFC: AMT-130
showed a favorable difference in TFC of 0.95 points at 30 months in the low-dose and 0.49
points at 18 months in the high-dose (baseline values: 11.9 in low-dose and 12.2 in high-dose). |
| o | TMS: AMT-130
showed a favorable difference in TMS of 2.80 points at 30 months in the low-dose and 1.70
points in the high-dose at 18 months (baseline values: 13.3 in low-dose and 12.1 in high-dose). |
Exploratory Interim Biomarkers and Volumetric Imaging Data
| · | Neurofilament Light
Chain (NfL): Mean CSF NfL for the low-dose cohort remained below baseline through month
30 and was 6.6% below baseline. Mean CSF NfL for the high-dose cohort also further declined
and is near baseline at month 18. These data suggest a reduction in neurodegeneration when
compared to an expected increase from baseline in CSF NfL based on natural history data.
As expected, all patients treated with AMT-130 experienced a transient increase in CSF NfL
related to the surgical procedure that peaked at approximately one month following the procedure
and declined thereafter. These transient increases were not dose-dependent. |
| · | Mutant Huntingtin
Protein (mHTT): Given AMT-130 is directly administered deep within the brain, the pharmacodynamics of mHTT in the CSF are not believed to be materially
representative of mHTT in the targeted brain regions. Mean changes in mHTT levels measured in CSF samples compared to baseline continue
to be variable and impacted by baseline levels near or below the lower limit of quantification. |
| · | Total Brain Volume:
Changes in the total brain volume of patients treated with AMT-130 were observed after the
surgical procedure and trended below natural history. The volumetric changes do not appear
to be clinically meaningful or associated with protracted increases in neurodegeneration
as measured by NfL. |
Interim Safety and Tolerability Data
AMT-130 was generally well-tolerated, with a manageable safety profile
at both the lower dose of 6x1012 vector genomes and the higher dose of 6x1013 vector genomes. The most common adverse
events in the treatment groups were related to the surgical procedure.
There were four serious adverse events (SAEs) unrelated to AMT-130
(post-operative delirium, major depression, suicidal ideation and epistaxis) in the low-dose cohort, six unrelated SAEs in the high-dose
cohort (back pain, hypothermia, post procedural hematoma, post-lumbar puncture syndrome (n=2) and pulmonary embolism), and one SAE (deep
vein thrombosis) in the control group. In addition, there were four AMT-130-related serious adverse events in the high-dose cohort (central
nervous system (CNS) inflammation (n=3), and severe headache (1) that, retrospectively, also was attributable to CNS inflammation).
Patients with symptomatic CNS inflammation improved with glucocorticoid
medication. Patients with symptomatic CNS inflammation improved with glucocorticoid medication. Additionally, six high-dose patients
have received perioperative steroids with the administration of AMT-130 to reduce the risk of inflammation.
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 potential clinical and functional effects
of AMT-130, including as an important treatment option for patients with Huntington’s disease; the Company’s plans with respect
to the clinical development of AMT-130 and regulatory pathways; the Company’s use of a natural history cohort as a basis for comparison
with respect to the efficacy of AMT-130; and the utility of mHTT and NfL in CSF as an effective biomarker for target engagement. 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 efficacy data sufficient to support further clinical development and the risk
that interim data from the trials may not be predictive of later data readouts; risks related to the Company’s financial position
and share price, including the Company’s ability to raise sufficient capital to support further development of its clinical programs,
as needed and on acceptable terms; risks related to the Company’s reliance on third parties to conduct, supervise, and monitor
its preclinical studies and clinical trials and to manufacture components of its drug product, including the clinical trials for AMT-130;
and the Company’s ability to obtain, maintain and protect its intellectual property. 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 February 27, 2023, its Quarterly Reports
on Form 10-Q filed with the SEC on May 9, 2023, August 1, 2023 and November 7, 2023, 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. |
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, thereunto
duly authorized.
|
UNIQURE N.V. |
|
|
|
|
|
Date: December 19, 2023 |
By: |
/s/ Jeannette
Potts |
|
|
Jeannette Potts |
|
|
Chief Legal and Compliance Officer |
Exhibit 99.1
uniQure Announces
Update on Phase I/II Clinical Trials of AMT-130
Gene Therapy
for the Treatment of Huntington’s Disease
~ Patients treated with AMT-130 continue to
show evidence of preserved neurological function with potential dose-dependent clinical benefits relative to an inclusion criteria-matched
natural history of the disease ~
~ Mean CSF NfL continue to demonstrate favorable
trends with low-dose patients below baseline at 30 months and high-dose patients near baseline at 18 months ~
~ AMT-130 continues to be generally well-tolerated
across both doses ~
~ Data support continuing clinical development
of AMT-130 and pursuing regulatory interactions to discuss potential strategies for ongoing development ~
~ Investor conference call and webcast today
at 8:30 a.m. ET ~
Lexington, MA and Amsterdam, the Netherlands,
December 19, 2023 — uniQure N.V. (NASDAQ: QURE), a leading
gene therapy company advancing transformative therapies for patients with severe medical needs, today announced updated interim data including
up to 30 months of follow-up from 39 patients enrolled in the ongoing U.S. and European Phase I/II clinical trials of AMT-130 for the
treatment of Huntington’s disease.
“The clinical assessment trends in the
ongoing studies of AMT-130 look very promising and continue to show disease stability in Huntington’s disease patients treated
with this one-time administered gene therapy, several of whom have now been followed more than two years,” stated Walid Abi-Saab,
M.D., chief medical officer of uniQure. “We are observing favorable trends in evaluation of motor skills, functional independence,
and composite rating scores as compared to a non-concurrent criteria-matched natural history cohort.”
“We also are pleased to observe further
declines in levels of NfL, a measurement of neuronal degradation and disease progression, with low-dose patients below baseline at 30
months of follow-up and high-dose patients near baseline at 18 months,” he continued. “Importantly, AMT-130 continues to be
generally well-tolerated with a manageable safety profile at both its low and high doses. We will continue to follow these patients and
look forward to initiating regulatory interactions next year.”
“The results from these Phase I/II trials
continue to be very encouraging as they show positive-trending, potentially dose-dependent signals across multiple key clinical and functional
measures, in conjunction with further declines in NfL,” stated Edward Wild, Ph.D., FRCP, professor of neurology at University College
London (UCL) Queen Square Institute of Neurology, consultant neurologist at National Hospital for Neurology & Neurosurgery, and
associate director of UCL Huntington’s Disease Centre. “While there are well-known complexities associated with analyzing
and interpreting other biomarkers in Huntington’s disease, these NfL data are consistent with the clinical data suggesting possible
disease stability and support the continued development of AMT-130. The Huntington’s disease community has endured a prolonged and
challenging wait for disease-modifying treatment options, and we enthusiastically embrace this potentially important advancement for this
devastating disease.”
Ongoing Phase I/II Trials of AMT-130 in Huntington’s
Disease
In the multi-center, Phase I/II clinical trial
of AMT-130 being conducted in the U.S., a total of 26 patients with early-manifest Huntington’s disease have been enrolled, including
an initial 10-patient low-dose cohort (6 treated, 4 control) with up to 30 months of follow-up and a subsequent 16-patient high-dose cohort
(10 treated, 6 control) with up to 18 months of follow-up. Patients were randomized to treatment with AMT-130 or an imitation (sham) surgery.
The U.S. study consists of a blinded 12-month core study period followed by unblinded long-term follow-up of five years for treated patients.
Four of the six control patients in the high-dose cohort were crossed over to treatment and the remaining two patients failed to meet
the study’s inclusion criteria.
The multi-center, open-label, Phase I/II clinical
trial of AMT-130 being conducted in Europe and the UK enrolled a total of 13 patients with the same early manifest criteria for Huntington’s
disease as the U.S. study. Six patients were treated with AMT-130 in the initial low-dose cohort and seven patients were treated in the
subsequent high-dose cohort.
The combined U.S. and European data presented
in this release are subject to a September 30, 2023 cut-off date and do not include efficacy and biomarker data from the control
patients who crossed over to treatment.
Updated Interim Data
Exploratory Efficacy Data
Clinical and functional measurements for treated
patients in each dose cohort were compared to baseline measurements, as well as to control patients (up to 12 months) and a non-concurrent
criteria-matched natural history cohort. The natural history cohort was developed by uniQure in collaboration with the Cure Huntington’s
Disease Initiative (CHDI) using the TRACK-HD natural history study of patients with early Huntington’s disease. The cohort includes
31 patients that met the uniQure clinical trial inclusion criteria of Total Functional Capacity, Diagnostic Classification Level and minimum
striatal volumes.
| · | Updated clinical data through 30 months for the
low-dose cohort and 18 months for the high-dose show ongoing evidence of potential dose-dependent clinical benefit relative to the non-concurrent
criteria-matched natural history. |
| · | For patients receiving the high dose, neurological
function as measured by cUHDRS and each of its individual components was preserved or improved at 18 months compared to pre-treatment
baseline measurements. |
| · | For patients receiving the low dose, neurological
function as measured by Total Motor Score (TMS) and Total Functional Capacity (TFC) was preserved at 30 months compared to pre-treatment
baseline measurements. |
| · | When compared to the expected rate of decline
from the natural history cohort, AMT-130 showed favorable trends in cUHDRS, TFC and TMS. |
| o | composite Unified Huntington’s Disease Rating Scale (cUHDRS): AMT-130 showed a favorable
difference in cUHDRS of 0.39 points at 30 months and 1.24 points at 18 months for the low- and high-dose, respectively (baseline values:
14.1 in low-dose and 14.9 in high-dose). |
| o | Total Functional Capacity (TFC): AMT-130 showed a favorable difference in TFC of 0.95 points at
30 months in the low-dose and 0.49 points at 18 months in the high-dose (baseline values: 11.9 in low-dose and 12.2 in high-dose). |
| o | Total Motor Score (TMS): AMT-130 showed a favorable difference in TMS of 2.80 points at 30 months
in the low-dose and 1.70 points in the high-dose at 18 months (baseline values: 13.3 in low-dose and 12.1 in high-dose). |
Biomarkers
and Volumetric Imaging Data
| · | Neurofilament Light Chain (NfL): Mean
CSF NfL for the low-dose cohort remained below baseline through month 30 and was 6.6% below baseline. Mean CSF NfL for the high-dose cohort
also further declined and is near baseline at month 18. These data suggest a reduction in neurodegeneration when compared to an expected
increase from baseline in CSF NfL based on natural history data. As expected, all patients treated with AMT-130 experienced a transient
increase in CSF NfL related to the surgical procedure that peaked at approximately one month following the procedure and declined thereafter.
These transient increases were not dose-dependent. |
| · | Mutant Huntingtin Protein (mHTT): Given
AMT-130 is directly administered deep within the brain, the pharmacodynamics of mHTT in the CSF are not believed to be materially representative
of mHTT in the targeted brain regions. Mean changes in mHTT levels measured in CSF samples compared
to baseline continue to be variable and impacted by baseline levels near or below the lower limit of quantification. |
| · | Total Brain Volume: Changes in the total
brain volume of patients treated with AMT-130 were observed after the surgical procedure and trended below natural history. The volumetric
changes do not appear to be clinically meaningful or associated with protracted increases in neurodegeneration as measured by NfL. |
Safety and Tolerability
AMT-130 was generally well-tolerated, with a manageable
safety profile at both the lower dose of 6x1012 vector genomes and the higher dose of 6x1013 vector genomes. The
most common adverse events in the treatment groups were related to the surgical procedure.
There were four serious adverse events (SAE) unrelated
to AMT-130 (post-operative delirium, major depression, suicidal ideation and epistaxis) in the low-dose cohort, six unrelated SAEs in
the high-dose cohort (back pain, hypothermia, post procedural hematoma, post-lumbar puncture syndrome (n=2), pulmonary embolism), and
one SAE (deep vein thrombosis) in the control group. In addition, there were four AMT-130-related serious adverse events in the high-dose
cohort (central nervous system inflammation (n=3), and severe headache (1) that, retrospectively, also was attributable to central
nervous system inflammation.
Patients with symptomatic central nervous system
inflammation improved with glucocorticoid medication. Additionally, six high-dose patients have received perioperative steroids with the
administration of AMT-130 to reduce the risk of inflammation.
Next Steps
Based on the promising
data from this interim analysis, uniQure anticipates the following next steps:
| · | uniQure began enrolling patients in a third cohort
to further investigate both doses in combination with perioperative immune suppression with a focus on evaluating near-term safety. Up
to 12 patients will be treated in this cohort, all of whom will receive AMT-130 using the current, established stereotactic neurosurgical
delivery procedure. |
| · | In the first quarter of 2024, uniQure plans to
initiate regulatory interactions to discuss the U.S. and European data and potential strategies for ongoing development of AMT-130. |
| · | In mid-2024, uniQure expects to present another
clinical update from the ongoing Phase I/II studies of AMT-130, including additional follow-up data from the treated patients in the U.S.
and European trials. |
Investor Conference Call and Webcast Information
uniQure management will host an investor conference
call and webcast today, Tuesday, December 19, 2023 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. Interested parties participating by phone will need to register using this
online form. After registering for dial-in details, all phone participants will receive an auto-generated e-mail containing
a link to the dial-in number along with a personal PIN number to use to access the event by phone. If you are joining the conference call,
please dial in 15 minutes before the start time.
About the Phase I/II Clinical Program of AMT-130
The U.S. Phase I/II clinical trial of
AMT-130 for the treatment of Huntington’s disease is exploring the safety, tolerability, and efficacy signals in 26 total patients
with early manifest Huntington’s disease split into a 10-patient low-dose cohort followed by a 16-patient high-dose cohort; patients
are randomized to treatment with AMT-130 or an imitation (sham) surgery. The multi-center trial consists of a blinded 12-month core study
period followed by unblinded long-term follow-up for five years. A total of 16 patients in the clinical trial were randomized to treatment
and received a single administration of AMT-130 through MRI-guided, convection-enhanced stereotactic neurosurgical delivery directly into
the striatum (caudate and putamen). An additional four control patients in the high-dose cohort crossed over to treatment. Additional
details are available on www.clinicaltrials.gov (NCT04120493).
The European, open-label Phase Ib/II study of
AMT-130 enrolled 13 patients with early manifest Huntington’s disease across two dose cohorts; a low-dose cohort of six patients
and a high-dose cohort of seven patients. Together with the U.S. study, the European study is intended to establish safety,
proof of concept, and the optimal dose of AMT-130 to take forward into Phase III development or into a confirmatory study should an accelerated
registration pathway be feasible.
AMT-130 is uniQure’s first clinical program
focusing on the CNS incorporating its proprietary miQURE® platform.
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. Despite the clear
etiology of Huntington’s disease, there are no currently 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 recent 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 usher in a new treatment approach for patients living with hemophilia. uniQure is now leveraging its modular and
validated technology platform to advance a pipeline
of proprietary gene therapies for the treatment of patients with Huntington's disease, refractory temporal lobe epilepsy, ALS, Fabry disease,
and other severe diseases. www.uniQure.com
uniQure Forward-Looking Statements
This press release 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," “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 press release. Examples of these forward-looking statements include, but are not limited to, statements concerning:
the potential clinical and functional effects of AMT-130, including as an important treatment option for patients with Huntington’s
disease; the company’s plans to continue clinical development of AMT-130 and to initiate interactions with regulatory authorities;
the potential for accelerated regulatory pathways; the company’s use of a natural history cohort as a basis for comparison with
respect to the efficacy of AMT-130; the company’s enrollment plans with respect to the third cohort studying AMT-130 in combination
with perioperative immune suppression; the utility of mHTT or NfL in CSF as an effective biomarker; and the company’s plans for
further clinical updates. uniQure’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 efficacy data sufficient to support further clinical
development and the risk that interim data from the trials may not be predictive of later data readouts; risks related to the company’s
financial position and stock price, including the company’s ability to raise sufficient capital to support further development of
the company’s clinical programs, as needed and on acceptable terms; risks related to the company’s reliance on third parties
to conduct, supervise, and monitor its preclinical studies and clinical trials and to manufacture components of its drug product, including
the clinical trials for AMT-130; and the company’s ability to obtain, maintain and protect its intellectual property. These risks
and uncertainties are more fully described under the heading "Risk Factors" in uniQure’s periodic filings with the
U.S. Securities & Exchange Commission (SEC), including its Annual Report on Form 10-K filed with the SEC on February 27,
2023, its Quarterly Reports on Form 10-Q filed with the SEC on May 9, 2023, August 1, 2023 and November 7, 2023, 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, uniQure assumes no obligation to update these
forward-looking statements, even if new information becomes available in the future.
uniQure Contacts:
FOR INVESTORS: | | FOR
MEDIA: |
| | |
Maria E. Cantor | Chiara Russo | Tom Malone |
Direct: 339-970-7536 | Direct:
617-306-9137 | Direct:
339-970-7558 |
Mobile: 617-680-9452 | Mobile: 617-306-9137 | Mobile:339-223-8541
|
m.cantor@uniQure.com | c.russo@uniQure.com | t.malone@uniQure.com |
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