LEXINGTON, Mass. and AMSTERDAM, the
Netherlands, Nov. 01, 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 its company-sponsored clinical data
presentations at the 59th American Society of Hematology (ASH)
Annual Meeting, taking place in Atlanta, GA from December 9 to 12,
2017.
Poster
Presentation
Title: |
|
Predictable
Protein Expression with Enhanced Factor IX Activity Following
Administration of a Modified AAV5-hFIX Vector to Nonhuman
Primates |
Presenter: |
|
Ying Poi Liu, Ph.D., senior
scientist at uniQure |
Session
Name: |
|
801. Gene Therapy and
Transfer: Poster I |
Date: |
|
Saturday, December 9,
2017 |
Presentation Time: |
|
5:30 PM - 7:30 PM EST |
Location: |
|
Georgia World Congress Center,
Building A, Level 1, Hall A2 |
The conference abstract was made
available today: ASH abstract.
A Good Laboratory Practices (GLP),
nonclinical study of AMT-061 has been performed in non-human
primates at four different dose levels up to a dose of 9 x 1013
gc/kg. The purpose of this study was to compare AMT-061 to AMT-060
with respect to liver transduction, circulating FIX protein levels,
circulating FIX activity levels and toxicity, after a single
intravenous dose with 13- or 26-week observation periods.
Data from the study demonstrated a strong
correlation between dose and human FIX (hFIX) expression levels, as
well as biological activity of the expressed hFIX protein. At
equal doses, circulating vector DNA plasma levels, liver
distribution, liver cell transduction and hFIX protein expression
were comparable for both AMT-060 and AMT-061. Additionally,
AMT-061 demonstrated substantial increases in FIX clotting activity
compared to AMT-060, consistent with those previously reported for
FIX-Padua.
Oral
Presentation
Title: |
|
Stable Elevations in FIX Activity
and Reductions in Annualized Bleeding Rate over up to 2 Years of
Follow-up of Adults with Severe or Moderate- Severe Hemophilia B
Treated with AMT-060 (AAV5-hFIX) Gene Therapy |
Presenter: |
|
Professor Frank W.G. Leebeek, M.D. Ph.D. |
Session Name: |
|
801. Gene Therapy and Transfer: Gene Therapy for
Hemophilia and Improving Lentiviral Vectors |
Session Date: |
|
Monday, December 11, 2017 |
Presentation Time: |
|
7:15 AM |
Location: |
|
Georgia World Congress Center, Building C, Level 1,
C101 Auditorium |
The conference abstract was made
available today: ASH abstract.
Long-term clinical data from the
ongoing Phase I/II trial of AMT-060 in patients with severe
hemophilia B will be presented on up to twenty-four months of
follow-up. All ten patients in the study demonstrated
improvements in their disease state as measured by reduced FIX
replacement therapy and bleeding frequency. Across the clinical
trials' two dosing cohorts, cumulative annualized FIX consumption
decreased by 79% as of deadline for ASH abstract submission, and in
the higher-dose cohort of the study, no spontaneous bleeds were
reported in the last six months of follow-up - with a reduction in
the annualized spontaneous bleed rate of 84% compared to the
one-year period prior to gene transfer.
No patients developed inhibitors
to FIX and there were no detectable signs of sustained AAV5
capsid-specific T-cell activation. Mild, temporary elevations in
ALT were observed in three patients, none of which were associated
with changes in in FIX activity or could be referred to as
capsid-specific T-cell responses. ALT elevations have not
recurred.
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