uniQure Announces Presentation of Non-Clinical Data of AMT-061 at the 59th American Society of Hematology (ASH) Annual Meeting
December 09 2017 - 9:00AM
YASTEST
Non-human primate study of AMT-061
utilizing FIX-Padua shows 6.5-fold increase in FIX clotting
activity
compared to AMT-060
Data supports nonclinical
comparability plan agreed upon with FDA
LEXINGTON, Mass. and AMSTERDAM, the
Netherlands, Dec. 09, 2017 (GLOBE NEWSWIRE) -- uniQure
N.V.(NASDAQ:QURE), a leading gene therapy company advancing
transformative therapies for patients with severe unmet medical
needs, today announced new nonclinical data on AMT-061, its
investigational gene therapy for patients with hemophilia B.
AMT-061 combines an AAV5 vector with the FIX-Padua variant. The
data will be presented today in a poster session at the 59th
American Society of Hematology (ASH) Annual Meeting
taking place in Atlanta, GA.
The data presented from the study in non-human
primates demonstrated dose-dependent expression of human Factor IX
protein (hFIX) and FIX clotting activity. Animals receiving the
same dose of AMT-060 and AMT-061 showed comparable levels of hFIX
protein, but the animals receiving AMT-061 demonstrated
approximately 6.5-fold higher FIX activity compared to those
receiving AMT-060.
"These data establish that AMT-061 is
well-tolerated and provides a substantial increase in FIX activity
compared to AMT-060, consistent with the gain-of-function reported
in previous preclinical studies of FIX-Padua," stated Sander van
Deventer, M.D., Ph.D., chief scientific officer of uniQure. "At a
dose of 2x1013 gc/kg, the same dose used in the second patient
cohort of our ongoing Phase I/II clinical trial of AMT-060, AMT-061
has the potential to achieve mean FIX activity in humans of
approximately 30 to 50 percent of normal."
Key Data Findings Presented at ASH
The goal of this non-human primate study was to
evaluate the efficacy and safety of introducing the FIX-Padua
variant into the AMT-060 cassette. AMT-061 leverages the same AAV5
capsid and insect cell-based manufacturing platform as
AMT-060. The study also compared AMT-061 to AMT-060 with
respect to liver transduction, circulating FIX protein levels,
circulating FIX activity levels and toxicity.
- Cynomolgus monkeys in 6 groups
(n = 3 per group) received a one-time
intravenous administration of either AMT-060 (5x1012 gc/kg),
AMT-061 in a range of four doses (5x1011 to 9x1013 gc/kg)
or the vehicle control.
- Animals receiving AMT-061 showed
clear, dose-dependent increases in FIX protein and an approximately
6.5-fold increase in FIX clotting activity compared to
AMT-060.
- At a dose of 5x1012 gc/kg,
AMT-061 FIX activity was at average of 58.9% of normal in week 4 to
week 13 post administration, whereas AMT-060 FIX activity was 9.1%
of normal during this same time period. AMT-060 and AMT-061 showed
similar circulating vector DNA plasma levels, liver distribution,
liver cell transduction and transgene expression.
- Safety measures, including various
markers of coagulation and fibrinolytic activation did not
significantly differ between the groups, suggesting that there is
no increased risk of thrombosis in animals expressing the Padua-FIX
variant.
- No toxicological findings or target
organ defects were detected after either AMT-060 or AMT-061
administration.
"The data confirms that the biological activity of
AMT-060 and AMT-061 are highly comparable, but the FIX clotting
activity of AMT-061 is substantially higher than AMT-060," added
van Deventer. "We believe the enhanced potency of AMT-061 has
the potential to provide substantial benefit to patients, and we
look forward to advancing this promising gene therapy candidate
into a pivotal study in 2018."
About
Hemophilia B
Hemophilia B is a serious and
rare inherited disease in males characterized by insufficient blood
clotting. The condition can lead to repeated and sometimes
life-threatening episodes of external and internal bleeding
following accidental trauma or medical interventions. The
episodes can cause long-term damage, for example to the joints, and
can be fatal if they occur in the brain. The deficient blood
clotting results from the lack of functional human Factor IX, or
hFIX. Treatment of hemophilia B today consists of prophylactic or
on-demand protein replacement therapy, in which frequent
intravenous administrations of plasma-derived or recombinant hFIX
are required to stop or prevent bleeding. Hemophilia B occurs in
approximately 1 out of 30,000 live births.
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, 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, and the scope of protection
provided by our patent portfolio. 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 collaborators' clinical development
activities, collaboration arrangements, 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 Quarterly Report on Form 10-Q filed on
November 1, 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
For Investors:
Maria E.
Cantor
Direct:
339-970-7536
Mobile: 617-680-9452
m.cantor@uniQure.com
Eva M.
Mulder
Direct: +31 20 240 6103
Mobile: +31 6 52 33 15 79
e.mulder@uniQure.com
For Media:
Tom
Malone
Direct: 339-970-7558
Mobile: 339-223-8541
t.malone@uniQure.com
This
announcement is distributed by Nasdaq Corporate Solutions on behalf
of Nasdaq Corporate Solutions clients.
The issuer of this announcement warrants that they are solely
responsible for the content, accuracy and originality of the
information contained therein.
Source: uniQure N.V. via Globenewswire
uniQure NV (NASDAQ:QURE)
Historical Stock Chart
From Apr 2024 to May 2024
uniQure NV (NASDAQ:QURE)
Historical Stock Chart
From May 2023 to May 2024