uniQure Presents New Data Demonstrating Successful and Effective Transduction of AAV5 Vector in the Presence of Pre-Existing ...
May 10 2017 - 7:00AM
-- Strong Dose-Dependent Transduction in All
Animals Tested Regardless of the Level of Anti-AAV5 Antibodies at
Pre-Administration --
uniQure N.V. (NASDAQ:QURE), a leading gene therapy company
advancing transformative therapies for patients with severe medical
needs, today presented new preclinical data demonstrating
successful and effective transduction of AAV5 in non-human primates
with pre-existing anti-AAV5 neutralizing antibodies (NABs). At all
observed levels, pre-existing neutralizing antibodies for AAV5 did
not have a negative impact on the transduction effectiveness of the
AAV5 vector. This suggests a much broader potential population of
eligible patients than previously expected for AAV5-based gene
therapies, including AMT-060, uniQure’s investigational gene
therapy in patients with severe hemophilia B.
The data were presented today at the American
Society of Gene & Cell Therapy (ASGCT) Annual Meeting in
Washington, D.C., the preeminent gene and cell therapy conference
in the world.
In ongoing gene therapy clinical trials using
adeno-associated virus (AAV) vectors, patients who present levels
of anti-AAV NABs are excluded from treatment due to concern that
the efficacy of AAV vectors may be negatively influenced by their
presence. In this study, uniQure researchers sought to assess the
impact of pre-existing anti-AAV5 NAB levels on the liver
transduction efficacy of an AAV5-based vector.
Preclinical Data Findings
Fourteen non-human primates with pre-existing
anti-AAV5 NABs at titers ranging from 1:57 to 1:1031, tested using
a highly-sensitive, luciferase-based assay, received intravenous
administration of an AAV5 vector (AAV5-hFIX). Within each dose
group, successful and comparable transduction was achieved,
independent of the level of pre-existing anti-AAV5 NABs. The data
show that anti-AAV5 NAB titers of up to at least 1:1031 have no
impact on the transduction of the liver by AAV5 in non-human
primates.
Low levels of NABs have been reported to block
liver transduction for other vector serotypes. This finding was not
seen in the present study with AAV5 in non-human primates, where
transduction was not impacted by moderate levels of NABs. Titers of
NABs against AAV5 in the general population are generally
characterized as low, suggesting that a greater portion of patients
can be effectively treated with AAV5 gene therapy, compared to
other AAV-based gene therapies.
“This important data suggests that patients with
pre-existing anti-AAV5 NABs may be able to be successfully treated
with AAV5 gene therapies, such as our product candidates AMT-060 in
hemophilia B and AMT-130 in Huntington’s disease,” stated Matthew
Kapusta, chief executive officer at uniQure. “This development has
the potential to significantly expand the applicability of AAV5
gene therapies to nearly all patients, regardless of pre-existing
antibodies. In addition, AAV5 also appears to have a more favorable
immunogenicity profile, with no immune responses detected across
two clinical studies involving intravenous administration to 18
patients. We believe these factors make AAV5 a highly
differentiated, best-in-class vector with the potential to more
effectively and safely deliver gene therapies to a greater portion
of patients in need of treatment.”
Investor/Analyst Breakfast and Webcast
on Friday, May 12, 2017
An investor and analyst breakfast meeting will
be held on Friday, May 12 at 7:00 a.m. Senior members of
uniQure's research and development team will discuss the results of
this study along with the four additional abstracts that are being
presented at ASGCT. The meeting will be webcast live along
with slides and can be accessed by visiting the investor relations
section of the Company's website at www.uniQure.com.
Date and Time: Friday,
May 12 at 7:00 a.m. EDTLocation: Omni Shoreham
Hotel, The Congressional Room, 2500 Calvert Street NW, Washington,
D.C. The Omni Shoreham hotel is located directly across from the
conference venue.
To request attendance at the meeting, please
RSVP to Investors@uniQure.com as space is limited.
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 cardiovascular 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, the development of our
gene therapy 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 corporate reorganizations and strategic shifts,
collaboration arrangements, our and our collaborators’ clinical
development activities, 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 2016 Annual Report on Form 10-K filed
on March 15, 2017. 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:
Maria E. Cantor
Direct: 339-970-7536
Mobile: 617-680-9452
m.cantor@uniQure.com
Tom Malone
Direct: 339-970-7558
Mobile: 339-223-8541
t.malone@uniQure.com
Eva M. Mulder
Direct: +31 20 240 6103
Mobile: +31 6 52 33 15 79
e.mulder@uniqure.com
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