uniQure Announces Clinical Data Presentations at the Upcoming International Society on Thrombosis and Hemostasis (ISTH) Congr...
June 23 2017 - 7:00AM
uniQure N.V. (NASDAQ:QURE), a leading gene therapy company
advancing transformative therapies for patients with severe medical
needs, today announced that two abstracts have been accepted for
presentation at the International Society on Thrombosis and
Hemostasis (ISTH) Congress held in Berlin, Germany, July 8 - 13,
2017.
“The data to be featured in an oral presentation
at ISTH now demonstrate long-term safety, efficacy and the
potential for broad application of AAV5 in hemophilia B,” stated
Matthew Kapusta, chief executive officer of uniQure. “We also
look forward to the presentation of clinical data demonstrating
successful and effective transduction in patients with pre-existing
neutralizing antibodies to AAV5, further supporting our belief that
our AAV5-based gene therapies may provide clinical benefits to
nearly all patients suffering from hemophilia B and other severe
diseases.”
Specific details on uniQure’s presentations at
ISTH include:
- Title: Updated Results from a Dose-escalating
Study in Adults with Severe or Moderate-Severe Hemophilia B Treated
with AMT-060 (AAV5-hFIX) Gene Therapy: Up to 1.5 Years
Follow-upOral Session Title: Gene Therapy for
Hemophilia: ClinicalDate and Time: Monday, July
10, 2017, 6:00 p.m. CET / 12:00 p.m. EDTLocation:
Hall B, CityCube Berlin, Messedamm 26, 14055, BerlinUpdated data on
AMT-060 will be presented by study investigator Professor Frank W.
Miesbach, M.D. Ph.D. of the Universitäts Klinikum Frankfurt,
Germany. AMT-060 is an AAV5-FIX gene therapy developed by uniQure
using its proprietary insect-cell based technology platform. The
updated data will include up to 1.5 years of follow-up on the first
patient cohort receiving 5x1012 gc/kg and up to a year of follow-up
on the second patient cohort receiving a higher dose of 2x1013
gc/kg.
- Title: Pre-existing anti-AAV5 neutralizing
antibodies measured using a highly sensitive assay in sera of
Hemophilia B patients in a Phase I/II clinical trial of AMT-060 do
not predict efficacy of AAV5-mediated liver-directed gene
transferPoster Session Date and Time: Tuesday,
July 11, 2017, 12:00 pm CET / 6:00 a.m.
EDTLocation: Exhibition Hall 4.2, CityCube,
Messedamm 26, 14055, Berlin
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
liver/metabolic, central nervous system 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.
Our actual results could differ materially from those anticipated
in these forward-looking statements for many reasons, including,
without limitation, risks associated with 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 with the Securities
and Exchange Commission 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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