uniQure Presents New Preclinical Data on AMT-130 at the CHDI’s 14th
Annual Huntington’s Disease Therapeutics Conference
uniQure N.V. (NASDAQ: QURE), a leading gene therapy company
advancing transformative therapies for patients with severe medical
needs, today presented new preclinical data on AMT-130, its gene
therapy candidate for the treatment of Huntington’s disease (HD) at
the 14th Annual CHDI Huntington’s disease Therapeutics Conference
in Palm Springs, California.
“Findings from our preclinical studies
illustrate the therapeutic potential of AMT-130 in restoring
function to damaged brain cells in Huntington’s disease and
providing a safe and sustained reduction of mutant huntingtin
protein with a gene therapy candidate that could be applicable to a
broad patient population,” stated Sander van Deventer, M.D., Ph.D.,
chief scientific officer at uniQure.
Five scientific abstracts submitted by uniQure
researchers were accepted for presentation at the conference.
Among these abstracts are the following poster presentations
featuring new data on AMT-130:
Magnetic resonance spectroscopy (MRS) shows
restoration of neuronal function in a HD mouse model treated with
AMT-130.
In this study, a non-invasive technique called
MRS was used to measure biomarkers for the health of brain cells in
HD mice after treatment with AMT-130 by direct injection in the
striatum. At 3 months after administration, significant levels of
vector were present in the treated areas resulting in robust
expression of microRNA and a significant knock down of the mutant
huntingtin protein (mHTT). The MRS analyses showed improvement in
brain cell function, a reversal in HD neuropathology, and a partial
reversal of volume loss in a key brain region involved in memory
called the hippocampus.
Pre-existing serum antibodies to AAV5
Neutralizing Antibodies (NABs) are not found in the cerebrospinal
fluid (CSF).
The presence of pre-existing neutralizing
antibodies (NABs) can reduce the efficacy of gene therapy. Thirty
matched serum and CSF samples from wild-type minipigs were analyzed
for NABs. Serum samples showed detectable levels of anti-AAV5 NABs
in the range of titers 2 to 256. All paired CSF samples were
negative for the presence of anti-AAV5 NABs.
For comparison, a prevalence study in 350
healthy donors showed serum anti-AAV5 NABs titers below 256 in 88%
of the subjects’ serum. The Company has previously reported data
that show anti-AAV5 NAB titers up to 340 in humans, and as high as
1,030 in primates, did not interfere with the therapeutic effect of
AAV5 gene therapies. Therefore, this study suggests the risk
of reduced efficacy due to anti-AAV5 NABs is low when AAV5 vectors
are administered into the brain or spinal fluid.
Virtual neurosurgical planning demonstrates the
safety of deep brain gene delivery in patients with early manifest
HD.
High-resolution magnetic resonance imaging (MRI)
scans from 20 patients with early manifest HD were analyzed to
simulate the neurosurgical insertion of micro-catheters by a
specialized technique called convection-enhanced delivery. The
results identify specific micro-catheter trajectories that are
projected to safely deliver gene therapy and provide enough
coverage to efficiently transduce the brain regions involved in
HD.
“uniQure’s neuroimaging translational studies in
non-human primates and Huntington’s disease patients shows the
importance of careful, individualized neurosurgical planning and
meticulous dosing parameters in delivering gene therapy products to
the brain,” added Joseph J. Higgins, M.D., F.A.A.N., vice
president, clinical development. “We are excited about
the AMT-130 clinical development program and look forward to
treating the first patient in the second half of this year and
announcing initial safety data by year-end.”
Additional data presented at the CHDI conference
include:
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“Gene Therapy for Huntington’s Disease: Silencing the Villain.”
Pavlina Konstantinova, Ph.D., vice president, new therapeutic
target discovery at uniQure, participated in the featured session
“HTT Lowering” on Tuesday, February 26 delivering an oral
presentation. |
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Transfer of therapeutic miRNAs within extracellular vesicles
secreted from Huntington’s disease iPSC-derived neurons. |
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Sustained mutant huntingtin lowering in the brain and cerebrospinal
fluid of Huntington disease minipigs mediated by AAV5-miHTT gene
therapy. |
About Huntington’s
DiseaseHuntington’s disease is a rare, inherited
neurodegenerative disorder that leads to loss of muscle
coordination, behavioral abnormalities and cognitive decline,
resulting in complete 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 HD, there are no 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 and partnered gene therapies to treat patients with
hemophilia, Huntington’s disease and other severe genetic 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, the achievement of any
of our planned near term or other milestones, our ability to become
the first AAV Gene therapy for Huntington’s Disease to begin
clinical trials, our ability to initiate our planned
dose-escalating, randomized and controlled Phase I/II clinical
trial, our ability to open several clinical sites in the United
States and begin enrolling patients in the second half of this year
or ever, the development of our gene therapy product candidates,
the ability to achieve therapeutic or curative effects in human
patients in any of our product candidates, whether our proprietary
miQURE™ gene silencing platform can be applied to any other
diseases, such as spinocerebellar ataxia type 3 (SCA3), the ability
to produce a product candidate that is safe and effective, the
ability to obtain regulatory approval for any of our product
candidates, and the risk of cessation, delay or lack of success of
any of our ongoing or planned clinical studies and/or development
of our product candidates. Our actual results could differ
materially from those anticipated in these forward-looking
statements for many reasons, including, without limitation, risks
associated with our and our collaboration activities, product
development activities, corporate reorganizations and strategic
shifts, 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 Annual Report on Form 10-K filed on March 14, 2018 and
Quarterly Report on Form 10-Q filed on November 6, 2018. 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 |
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Eva M. Mulder |
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Tom Malone |
Direct:
339-970-7536 |
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Direct: +31 20 240 6103 |
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Direct: 339-970-7558 |
Mobile:
617-680-9452 |
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Mobile: +31 6 52 33 15 79 |
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Mobile:339-223-8541 |
m.cantor@uniQure.com |
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e.mulder@uniQure.com |
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t.malone@uniQure.com |
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