uniQure Announces Latest Positive Recommendation from Data Safety Monitoring Board in Phase I/II Clinical Trial of AMT-130 for the Treatment of Huntington’s Disease
November 02 2021 - 7:05AM
uniQure Announces Latest 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 the positive recommendation by the
independent Data Safety Monitoring Board (DSMB) following a review
of safety data from the four patient procedures in the higher-dose
cohort of the Phase I/II clinical trial of AMT-130 for the
treatment of Huntington’s disease. With the positive
recommendation, the final 12 patients in this second cohort are now
cleared for enrollment.
A total of 14 blinded administration procedures
have been completed as of August 2021. In the study to date, eight
patients have been treated with AMT-130, and six patients have
received imitation surgery. Full enrollment of the second,
higher-dose cohort is expected to be completed in mid-2022.
“The AMT-130 program continues to advance at a
steady pace, and with this positive recommendation from the DSMB,
we are eager to enroll the final 12 patients in the higher-dose
cohort,” said David Cooper, M.D., vice president of clinical
development at uniQure. “We also look forward to expanding our
efforts with the initiation of a separate open-label study of
AMT-130 in Europe and to sharing preliminary imaging and biomarker
data from the initial four patients in 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 is exploring the safety,
tolerability, and efficacy signals in a planned 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;
patients will be 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. A total of 16 patients in the clinical
trial 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 trial of AMT-130 by the middle of 2022 or ever, whether
we initiate dosing in our European open-label Phase Ib/II
clinical trial in the second half of 2021 or ever, whether we are
able to enroll the currently planned number of patients in the U.S.
Phase I/II or the European open-label Phase Ib/II clinical trials,
, 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 October 25, 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: |
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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 |
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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