uniQure Announces Dosing of First Patient in GenTLE Phase I/IIa Clinical Trial of AMT-260 for the Treatment of Refractory Mesial Temporal Lobe Epilepsy
21 Novembro 2024 - 9:05AM
uniQure N.V. (NASDAQ: QURE), a leading gene therapy company
advancing transformative therapies for patients with severe medical
needs, today announced that the first patient has been dosed in the
GenTLE Phase I/IIa clinical trial of AMT-260 for the treatment of
refractory mesial temporal lobe epilepsy (MTLE).
“The dosing of the first patient in our Phase
I/II trial in temporal lobe epilepsy represents an important
milestone for uniQure and our third clinical trial initiation over
the past six months,” stated Walid Abi-Saab, M.D., chief medical
officer of uniQure. “Nearly one-third of people experiencing focal
onset seizures do not respond to currently available treatments and
are left with limited therapeutic options. Our investigational
agent, AMT-260, which is a one-time administration, has the
potential to be a transformative treatment option for these
patients. We continue to actively screen patients for the trial and
look forward to providing program updates in the new year.”
AMT-260 consists of an AAV9 vector that locally
delivers two engineered miRNAs designed to reduce the expression of
GluK2 protein subunits, a subtype of glutamate receptor that is
believed to be aberrantly expressed in the hippocampus of patients
with refractory MTLE and believed to trigger their seizure
activity. In preclinical animal studies, AMT-260 reduced the number
of seizures per day in a dose-dependent manner. AMT-260 also
reduced the expression of GluK2 mRNA and protein in the hippocampus
of epileptic mice and from resected hippocampal slices from
patients with refractory MTLE.
GenTLE is a Phase I/IIa multi-center, open-label
trial being conducted in the U.S. to evaluate the safety,
tolerability and exploratory signs of efficacy of two doses of
AMT-260 in individuals with refractory MTLE. The study comprises
two dose cohorts of six patients each. The study is actively
recruiting though 10 sites with an additional two sites expected to
be activated by the end of 2024. Additional details are available
on www.clinicaltrials.gov (NCT06063850).
About Refractory Mesial Temporal Lobe
Epilepsy
Temporal lobe epilepsy is a chronic neurologic
disorder and is the most common form of focal epilepsy with more
than 600,000 individuals suffering from the disorder in the United
States. Approximately 80% of all temporal lobe epilepsy cases are
mesial, which involves the medial (or internal) structures of the
brain. The majority of MTLE cases are refractory to anti-seizure
medications, which severely limits treatment options.
About uniQure
uniQure’s mission is to reimagine the future of
medicine by delivering innovative cures that transform lives. The
recent approvals of our gene therapy for hemophilia B – a 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. We are now leveraging our modular and validated
technology and manufacturing platform to advance a pipeline of
proprietary gene therapies for the treatment of patients with
Huntington's disease, refractory mesial temporal lobe epilepsy,
amyotrophic lateral sclerosis (ALS), Fabry disease, and other
severe diseases. www.uniQure.com
uniQure Forward-Looking
Statements
This 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," “establish,” "estimate,"
"expect," "goal," "intend," "look forward to", "may," "plan,"
"potential," "predict," "project," “seek,” "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.
Examples of these forward-looking statements include, but are not
limited to, statements regarding AMT-260’s potential to be a
transformative treatment option for these patients with MTLE; the
potential efficacy profile of AMT-260 through one-time
administration with the ability to reduce the frequency of seizures
in MTLE patients; the Company’s plans to announce additional
updates on trial enrollment; the design of the AMT-260 Phase I/IIa
clinical trial and plans to activate additional study sites. The
Company’s actual results could differ materially from those
anticipated in these forward-looking statements for many reasons.
These risks and uncertainties include, without limitation, risks
associated with the clinical results and the development and timing
of the Company’s programs; the Company’s interactions with
regulatory authorities, which may affect the initiation, timing and
progress of clinical trials and pathways to approval; the Company’s
ability to continue to build and maintain the company
infrastructure and personnel needed to achieve its goals; the
Company’s effectiveness in managing current and future clinical
trials and regulatory processes; the continued development and
acceptance of gene therapies; the Company’s ability to demonstrate
the therapeutic benefits of its gene therapy candidates in clinical
trials; the Company’s ability to obtain, maintain and protect
intellectual property; and the Company’s ability to fund its
operations and to raise additional capital as needed. 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 February 28, 2024, its Quarterly Reports on Form
10-Q filed May 7, 2024, August 1,
2024 and November 5, 2024, 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 the Company
assumes no obligation to update these forward-looking statements,
even if new information becomes available in the future.
uniQure Contacts: |
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FOR INVESTORS: |
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FOR MEDIA: |
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Chiara Russo |
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Tom Malone |
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Direct: 617-306-9137 |
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Direct: 339-970-7558 |
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Mobile: 617-306-9137 |
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Mobile:339-223-8541 |
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c.russo@uniQure.com |
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t.malone@uniQure.com |
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