uniQure Announces Enrollment of First Two Patients in Second Cohort of Phase I/II Clinical Trial of AMT-130 for the Treatment...
June 16 2021 - 7:05AM
uniQure Announces Enrollment of First Two Patients in Second Cohort
of 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 the first two patient procedures have
been completed in the higher dose, second cohort of the Phase I/II
clinical trial of AMT-130 for the treatment of Huntington’s
disease. The initiation of patient enrollment in this 16-patient
cohort follows a meeting last month of the trial’s independent Data
Safety Monitoring Board (DSMB) that reviewed safety data for the
fully enrolled first cohort of ten patients. The Phase I/II study
is a double-blind, randomized clinical trial being conducted in the
United States. To date, seven patients now have been treated with
AMT-130 across both trial cohorts, and five patients have received
the imitation surgery.
“We are very pleased with the progress that we
continue to make in this Phase 1-2 clinical trial and that we are
now administering AMT-130 at the higher dose,” said Ricardo
Dolmetsch, Ph.D., president of research and development at uniQure.
“We look forward to ongoing patient enrollment in the second cohort
and to initiating clinical development of AMT-130 in Europe in the
second half of this year. We also remain on track to share initial
imaging and biomarker data from the U.S. clinical trial before the
end of the 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 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, whether we achieve
further patient enrollment in the second cohort, whether
we initiate dosing in our European open-label Phase Ib/II in
the second half of 2021 or ever, 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 Commercialization and License Agreement
with CSL Behring, 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 May 10, 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.
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uniQure Contacts: |
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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 |
m.cantor@uniQure.com |
c.russo@uniQure.com |
t.malone@uniQure.com |
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