-- Strong Dose-Dependent
Transduction in All Animals Tested Regardless of the Level of
Anti-AAV5 Antibodies at Pre-Administration --
-- Data Suggests AAV5 Gene
Therapies May be Viable Treatments for Nearly All Patients
--
-- Preclinical Data Presented Today at American Society of Gene
& Cell Therapy (ASGCT) Annual Meeting in Washington, D.C.
--
-- Investor Webcast Friday, May 12,
2017 at 7:00 a.m. ET --
LEXINGTON, Mass. and AMSTERDAM,
the Netherlands, May 10, 2017 (GLOBE NEWSWIRE) -- 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. EDT
Location: 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 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 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.