uniQure Presents New Clinical Data in Hemophilia B Patients Demonstrating Therapeutic Efficacy of AAV5 Gene Therapy in the Pr...
July 11 2017 - 6:30AM
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
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, 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
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