uniQure Announces Completion of Patient Enrollment in the First Two Cohorts of its Phase I/II Clinical Trial of AMT-130 for the Treatment of Huntington’s Disease
March 21 2022 - 7:05AM
uniQure Announces Completion of Patient Enrollment in the First Two
Cohorts of its 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 completion of patient enrollment in the
first two cohorts of its randomized, double-blinded, Phase I/II
clinical trial of AMT-130 for the treatment of early stage
Huntington’s disease.
“We are very pleased to have completed the
enrollment of the first 26 patients in this ongoing clinical
trial,” stated Ricardo Dolmetsch, Ph.D., president of research and
development. “It’s a major milestone for the trial and an important
achievement for our clinical operations team. Our investigators
have done an exceptional job in enrolling this important study. We
look forward to providing a clinical update in the second quarter
of this year from the 12-month interim analysis of the 10 patients
in the first cohort, including safety, mutant HTT protein (mHTT)
and neurofilament light chain (NfL) data. We also remain on track
with the enrollment of our European open-label clinical trial of
AMT-130 and plan to begin a third patient cohort in the U.S. to
explore an improved administration procedure.”
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
includes two dose cohorts of 26 patients randomized to either
treatment with AMT-130 or to 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 includes 16 patients, of
which ten patients received treatment with AMT-130 and six patients
received imitation surgery. A third cohort exploring the use of an
alternative stereotactic navigation system to simplify placement of
infusion catheters will include up to 18 additional randomized
patients. The Phase I/II clinical trial consists of a blinded
12-month period followed by unblinded long-term follow-up for five
years. The treated patients have received a single administration
of AMT-130 using MRI-guided, convection-enhanced stereotactic
neurosurgical delivery directly into the striatum (caudate and
putamen).
The ongoing Phase Ib/II trial of AMT-130 in
Europe is an open-label study that 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 a pivotal study or into a confirmatory study
should an accelerated registration 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®
microRNA platform. miQURE is designed to degrade the messenger RNA
produced by disease-causing genes without causing toxicity and to
spread through an organ using exosomes.
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, refractory
temporal lobe epilepsy, Fabry disease, 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 re not limited to, whether we will share
mutant HTT protein (mHTT), neurofilament light chain (NfL), or
other safety and biomarker data from any patients in our AMT-130
clinical trials during the second quarter of 2022, or ever, and
whether we will initiate enrollment of a third cohort in our Phase
I/II study of AMT-130 in 2022, or ever. The Company’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 clinical development activities, regulatory
oversight, as well as the risks, uncertainties and other factors
described under the heading "Risk Factors" in the Company’s
periodic securities filings, including its Annual Report on Form
10-K filed February 25, 2022. Given these risks, uncertainties and
other factors, you should not place undue reliance on these
forward-looking statements, and the Company assumes 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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