uniQure Announces Positive Recommendation to Advance Phase I/II Clinical Trial of AMT-130 for the Treatment of Huntington’s...
May 27 2021 - 7:05AM
uniQure Announces Positive Recommendation to Advance 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 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
safety data for the fully enrolled first cohort of ten patients.
This data set included nine-month safety data from the first two
enrolled patients, six-month safety data from the next two enrolled
patients, and 30-day safety data on remaining six patients in the
trial. The DSMB recommended continued dosing in the study, and
uniQure will now begin to enroll patients in the higher-dose cohort
of the trial. The Phase I/II study is a double-blind, randomized
clinical trial being conducted in the United States. To date, six
patients have been treated with AMT-130, and four patients received
the imitation surgery.
“We are very pleased with the progress of the
study and with the recommendations of the DSMB. We look forward to
proceeding with dose-escalation in the second cohort of patients,”
said Ricardo Dolmetsch, Ph.D., president of research and
development at uniQure. “We remain highly focused on progressing
this therapy given the significant unmet need in the Huntington’s
disease community and are committed to initiating clinical
development of AMT-130 in Europe in the second half of this year
and to sharing 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
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 begin dosing
the second cohort of the U.S. Phase I/II clinical study of AMT-130,
whether we initiate dosing in our European open-label Phase
Ib/II in the second half of 2021 or ever, 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.
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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