uniQure Announces Recommendations from Data Safety Monitoring Board of Phase I/II Clinical Trial of AMT-130 for the Treatment...
September 25 2020 - 7:05AM
uniQure N.V. (NASDAQ: QURE), a leading gene therapy company
advancing transformative therapies for patients with severe medical
needs, today announced that the independent Data Safety Monitoring
Board (DSMB) overseeing the Phase I/II clinical trial of AMT-130
for the treatment of Huntington’s disease has met and reviewed
90-day safety data from the first two patients enrolled in the
trial. No significant safety concerns were noted to prevent further
dosing, and the next two patients are now cleared for enrollment in
the study. The Phase I/II study is a double-blind, randomized
clinical trial being conducted in the United States. One patient
has been treated with AMT-130, and one patient received the
imitation surgery.
“We are very pleased with the positive outcome
from this first DSMB meeting,” said Ricardo Dolmetsch, president of
research and development at uniQure. “We will now advance the study
and expect to enroll the next two patients as soon as
possible.”
About the Phase I/II Clinical Trial of
AMT-130
The 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 patients with early
manifest Huntington’s disease randomized to treatment with AMT-130
or an imitation (sham) surgery. The five-year, multi-center trial
consists of a blinded 12-month core study period followed by
unblinded long-term follow-up. 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).
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 will be able
to enroll the next two patients in the clinical trial. 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, 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 30, 2020. 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: |
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FOR MEDIA: |
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Maria E. Cantor |
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Chiara Russo |
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Tom Malone |
Direct: 339-970-7536 |
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Direct: 617-306-9137 |
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Direct: 339-970-7558 |
Mobile: 617-680-9452 |
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Mobile: 617-306-9137 |
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Mobile: 339-223-8541 |
m.cantor@uniQure.com |
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c.russo@uniQure.com |
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t.malone@uniQure.com |
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