uniQure Announces Completion of Enrollment in First Cohort of Phase I/II Clinical Trial of AMT-130 for the Treatment of Hunti...
April 05 2021 - 7:05AM
uniQure N.V. (NASDAQ: QURE), a leading gene therapy company
advancing transformative therapies for patients with severe medical
needs, today announced the completion of patient enrollment in the
first dose cohort of a randomized, double-blinded, Phase I/II
clinical trial of AMT-130 for the treatment of early stage
Huntington’s disease. The Company also announced plans to begin an
open-label clinical trial of AMT-130 in Europe later this year.
“This is an important milestone in our ongoing
clinical development of AMT-130,” stated David Cooper, M.D., vice
president, clinical development at uniQure. “Nine U.S. study sites
are now active to support enrollment in the next cohort, which is
expected to start after the Data Safety Monitoring Board’s review
in the middle of the year. Completing enrollment of the first,
10-patient cohort ahead of schedule highlights the high level of
interest among the Huntington’s disease patient and clinical
community, and the collaboration between our participating HD
Centers of Excellence and the expert neurosurgical sites performing
the MRI-guided procedures. We also look forward to initiating a new
clinical study of AMT-130 in Europe later this year. It is
estimated that there could be as many as 75,000 Europeans affected
by Huntington’s disease.”
The ongoing Phase I/II clinical trial of AMT-130
is a randomized, sham controlled, double-blinded study to explore
the safety, tolerability, and proof of concept of AMT-130 in
patients with early manifest Huntington’s disease. The study, which
includes two dose cohorts, will randomize a total of 26 patients to
either treatment with AMT-130 or an imitation surgical procedure.
The first dose cohort includes 10 patients, of which six patients
received treatment with AMT-130 and four patients received
imitation surgery. The second dose cohort is planned to include 16
patients, of which 10 patients will receive treatment with AMT-130
and six patients will receive imitation surgery. The trial consists
of a blinded 12-month study period followed by unblinded long-term
follow-up for 5 years after administration of AMT-130. Patients
receive a single administration of AMT-130 through MRI-guided,
convection-enhanced stereotactic neurosurgical delivery directly
into the striatum (caudate and putamen).
The planned Phase Ib/II study of AMT-130 will be
conducted in Europe and is expected to begin enrolling patients in
the second half of 2021. This open-label study 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 comprises a recombinant AAV5 vector
carrying a DNA cassette encoding a microRNA that lowers Huntingtin
protein in Huntington’s disease patients. AMT-130 is uniQure’s
first clinical program incorporating its proprietary miQURE™
platform. miQURE is designed to degrade disease-causing genes
without off-target toxicity and induce silencing of the entire
target organ through secondary exosome-mediated delivery.
About Huntington’s Disease
Huntington’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
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," "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, the enrollment of
patients in, or Data Safety Monitoring Board review of, our Phase
I/II gene therapy clinical trial of AMT-130 in Huntington’s
disease, including whether we will be able to fully enroll the
second dose cohort as currently planned, and whether we will
initiate our P1b/II clinical study of AMT-130 in Europe later this
year or ever. uniQure’s 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 Commercialization and
License Agreement with CSL Behring, the regulatory approval of that
transaction, our clinical development activities, clinical results,
collaboration arrangements, regulatory oversight, 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 1, 2021. Given these
risks, uncertainties and other factors, you should not place undue
reliance on these forward-looking statements, and uniQure assumes
no obligation to update these forward-looking statements, even if
new information becomes available in the future.
uniQure Contacts:
FOR
INVESTORS: |
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FOR
MEDIA: |
|
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|
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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