uniQure Announces Completion of Additional Patient Procedures Following Positive Recommendation from Data Safety Monitoring Board in Phase I/II Clinical Trial of AMT-130 for the Treatment of Huntington’s Disease
August 30 2021 - 7:05AM
uniQure Announces Completion of Additional Patient Procedures
Following Positive Recommendation from Data Safety Monitoring Board
in Phase I/II Clinical Trial of AMT-130 for the Treatment of
Huntington’s Disease
uniQure N.V. (NASDAQ: QURE), a leading gene therapy company
advancing transformative therapies for patients with severe medical
needs, today announced that two additional patient procedures have
been completed in the second, higher-dose cohort in the Phase I/II
clinical trial of AMT-130 for the treatment of Huntington’s
disease. These latest procedures follow a positive recommendation
by the independent Data Safety Monitoring Board (DSMB) after
reviewing available follow-up data from all participants in the
trial. A total of 14 blinded administration procedures have now
been completed, including four patients in the second dose cohort.
In the study to date, eight patients have been treated with
AMT-130, and six patients received imitation surgery.
In the fourth quarter of 2021, the Company
expects the DSMB to review the 1-month safety data from the two
most recently enrolled patients in the higher dose cohort, as well
as the 3-month data from the first two patients. Upon receiving a
positive recommendation from the DSMB, the Company will commence
enrollment of the final 12 patients in the second dose cohort.
“We continue to be very pleased with the
enrollment of this important study and look forward to having the
trial fully enrolled by the middle of next year,” said David
Cooper, M.D., vice president of clinical development at uniQure.
“We also look forward to sharing preliminary imaging and biomarker
data from initial patients in the U.S. clinical trial before the
end of the year and initiating a separate open-label study of
AMT-130 in Europe later this year.”
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 will explore 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, higher-dose cohort 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. Patients
will receive a single administration of AMT-130 through MRI-guided,
convection-enhanced stereotactic neurosurgical delivery directly
into the striatum (caudate and putamen). Additional details are
available on www.clinicaltrials.gov (NCT04120493).
The European, open-label Phase Ib/II study of
AMT-130 will enroll 15 patients with early manifest Huntington’s
disease across two dose cohorts. 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 central nervous system (CNS) incorporating its
proprietary miQURE™ platform.
About Huntington’s
DiseaseHuntington’s disease is a rare, inherited
neurodegenerative disorder that leads to motor symptoms including
chorea, and 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. We are leveraging our modular and
validated technology platform to rapidly advance a pipeline of
proprietary gene therapies to treat patients with hemophilia B,
Huntington's disease, Fabry disease, spinocerebellar ataxia Type 3
and other diseases. www.uniQure.com
uniQure Forward-Looking
StatementsThis press release contains forward-looking
statements. All statements other than statements of historical fact
are forward-looking statements, which are often indicated by terms
such as "anticipate," "believe," "could," "estimate," "expect,"
"goal," "intend," "look forward to", "may," "plan," "potential,"
"predict," "project," "should," "will," "would" and similar
expressions. 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.
These forward-looking statements include, but
are not limited to, whether we commence the enrollment of the final
12 patients in the second dose cohort in the fourth quarter of 2021
or ever, whether we complete enrollment of the U.S. Phase I/II
clinical study of AMT-130 by the middle of 2022 or ever, whether
we initiate dosing in our European open-label Phase Ib/II in
the second half of 2021 or ever, whether we receive a positive
recommendation from upcoming DSMB meetings, including the meeting
currently scheduled for the fourth quarter of 2021, and whether we
share initial imaging and biomarker data towards the end of the
year or ever. Our actual results could differ materially from
those anticipated in these forward-looking statements for many
reasons, including, without limitation, risks associated with the
impact of the ongoing COVID-19 pandemic on our Company and the
wider economy and health care system, our clinical development
activities, clinical results, collaboration arrangements,
regulatory oversight, manufacturing activities, product
commercialization and intellectual property claims, as well as the
risks, uncertainties and other factors described under the heading
"Risk Factors" in uniQure’s periodic securities filings, including
its Annual Report on Form 10-K filed March 2, 2020 and Quarterly
Report on Form 10-Q filed on July 26, 2021. Given these risks,
uncertainties and other factors, you should not place undue
reliance on these forward-looking statements, and we assume 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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