-- Findings Further Support Expanding the Eligibility of AAV5 Gene
Therapies to Nearly All Patients with Hemophilia B --
LEXINGTON, Mass. and AMSTERDAM,
the Netherlands, July 11, 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 clinical data demonstrating that the presence
of pre-existing anti-AAV5 neutralizing antibodies (NABs) does not
predict the potential efficacy of AAV5-mediated gene transfer in
patients with hemophilia B. Clinically meaningful factor IX (FIX)
activity levels from the ongoing Phase I-II trial of AMT-060 were
observed at NAB titers up to 1:341, determined as corresponding up
to the 90th percentile
of a healthy control population. NABs were quantified in the blood
sera of these patients using a highly sensitive assay. These
clinical data were presented today in a poster presentation at the
26th Biennial
Congress of the International Society on Thrombosis and
Hemostasis (ISTH), taking place this week in Berlin,
Germany.
The presence of pre-existing NABs
to adeno-associated virus (AAV) vectors has long posed a critical
challenge for the clinical application of gene therapies, as
patients who currently screen positive for NABs are generally
excluded from treatment. Researchers from uniQure recently
presented data in non-human primates suggesting that AAV5 could
successfully mediate gene transfer in the presence of NABs at
levels as high as 1:1031.
In a poster presentation at the
ISTH meeting, a re-analysis was described of pre-gene transfer
screening samples from the 10 patients who have been treated in the
ongoing Phase I/II trial of AMT-060 for hemophilia B. The patients
had tested negative for pre-existing anti-AAV5 NAbs using a green
fluorescent protein-based (GFP) assay before receiving treatment.
These samples were later re-assessed using a highly sensitive
luciferase-based (LUC) NAB assay. Anti-AAV5 NABs were
detected retrospectively in three patients who had been treated
with the low dose (5x1012 gc/kg)
of AMT-060. However, all three patients presented increases in FIX
expression and, especially, the patient with the highest NAB level
(titer 1:341) had the highest FIX-activity (steady-state FIX 6.8%
of normal; latest FIX measurement 10.7% of normal) among all five
patients treated in the low-dose cohort. None of the three patients
who tested positive for NAB titers, experienced over time
elevations in liver enzymes post gene transfer, FIX activity loss,
or clinically relevant T-cell responses to the capsid.
"These clinical data show that
hemophilia B patients presenting with neutralizing antibodies may
be considered eligible for AAV5-mediated gene transfer," stated
Matthew Kapusta, chief executive officer at uniQure. "This
development potentially expands the applicability of AAV5 gene
therapies to nearly all hemophilia B patients. We believe these
factors contribute to making AAV5 a potential best-in-class vector
for delivering gene therapies more effectively and safely to a
greater portion of patients in need of treatment."
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, statements regarding
the development of our gene therapy product candidates, including
the future development of AMT-060, the success of our
collaborations 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 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