~ Updated, Long-term Results from the Phase I/II Dose-Escalating
Study of AMT-060 to be Presented on Monday, July 10, 2017 ~
LEXINGTON, Mass. and AMSTERDAM,
the Netherlands, June 23, 2017 (GLOBE NEWSWIRE) -- 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-up
Oral Session Title: Gene Therapy for
Hemophilia: Clinical
Date and Time: Monday, July 10,
2017, 6:00 p.m. CET / 12:00 p.m. EDT
Location: Hall B, CityCube
Berlin, Messedamm 26, 14055, Berlin
Updated 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 transfer
Poster Session Date and
Time: Tuesday, July 11, 2017, 12:00 pm CET / 6:00
a.m. EDT
Location: 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