uniQure Announces First Patient Treated in Dose-Confirmation Study of AMT-061 in Patients with Hemophilia B
August 23 2018 - 7:00AM
uniQure N.V. (NASDAQ:QURE), a leading gene therapy company
advancing transformative therapies for patients with severe unmet
medical needs, today announced that it has treated the first
patient in its Phase IIb dose-confirmation study of AMT-061, an
investigational AAV5-based gene therapy incorporating the FIX-Padua
variant for the treatment of patients with severe and moderately
severe hemophilia B. AMT-061 has been granted Breakthrough
Therapy Designation by the United States Food and Drug
Administration and access to Priority Medicines (PRIME) regulatory
initiative by the European Medicines Agency.
“The initiation of the AMT-061 dose-confirmation
study is an important step toward our goal of advancing a
potentially life-changing treatment for patients with hemophilia
B,” said Robert Gut, M.D., Ph.D., chief medical officer of
uniQure. “I am extremely proud of the efforts made by the
uniQure team to initiate this study, the objective of which is to
demonstrate meaningful increases in FIX activity using the Padua
variant and confirm dosing for the HOPE-B pivotal trial initiated
this past June. We look forward to completing patient enrollment
shortly and providing top-line data before the end of the
year.”
“As a one-time administered therapy, AMT-061 has
the potential to transform the treatment paradigm for hemophilia B
patients,” said Annette von Drygalski, M.D., associate clinical
professor at the University of California San Diego and director of
its hemophilia and thrombosis treatment center. “By incorporating
both AAV5 and the FIX-Padua variant, AMT-061 has the potential to
deliver clinically relevant increases in FIX activity with low risk
of cellular immune responses, which could expand patient
eligibility for treatment with gene therapy. I greatly appreciate
the opportunity to participate in the AMT-061 clinical program and
view the initiation of the Phase IIb and Phase III studies as
important milestones in the development of this potentially
important therapy for patients with hemophilia B.”
The Phase IIb dose-confirmation study is an
open-label, single-arm, single-dose trial being conducted in the
United States. Approximately three patients are expected to receive
a single intravenous (IV) infusion of 2x1013 vc/kg and be evaluated
for a period of approximately six to eight weeks to assess Factor
IX (FIX) activity.
Phase III HOPE-B Pivotal
Trial
Patient enrollment is also underway in the
global Phase III HOPE-B clinical trial to evaluate the safety and
efficacy of AMT-061. Approximately 50 adult hemophilia B
patients classified as severe and moderately-severe will be
enrolled in a six-month observational period during which time they
will continue to use their current standard of care to establish a
baseline control. After the six-month lead-in period,
patients will go onto receive a single intravenous administration
of AMT-061. Dosing of patients in the HOPE-B pivotal trial is
expected to start early in the first quarter of
2019.
About AMT-061
AMT-061 consists of an AAV5 viral vector
carrying a gene cassette with the Padua variant of Factor IX
(FIX-Padua).FIX-Padua has been reported to provide an approximate 8
to 9-fold increase in FIX activity compared to the wild-type FIX
protein, as used in AMT-060. AAV5-based gene therapies have been
demonstrated to be safe and well-tolerated in a multitude of
clinical trials, including three uniQure trials conducted in 22
patients in hemophilia B and other indications. No patient treated
in clinical trials with the Company’s AAV5 gene therapies has
experienced any cytotoxic T-cell-mediated immune response to the
capsid.
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, the completion of
enrollment in our Phase IIb study, the release of top-line clinical
data, the ability to transform the treatment paradigm for
hemophilia B patients or to deliver clinically relevant increases
in FIX activity or to provide a low risk of cellular immune
responses or to expand patient eligibility for treatment with gene
therapy, the achievement of any of our planned near term or other
milestones, 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 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 August 8, 2018. 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: |
|
FOR
MEDIA: |
Maria E.
Cantor |
Eva M.
Mulder |
Tom
Malone |
Direct:
339-970-7536 |
Direct: +31 20 240
6103 |
Direct:
339-970-7558 |
Mobile:
617-680-9452 |
Mobile: +31 6 52 33 15
79 |
Mobile:
339-223-8541 |
m.cantor@uniQure.com |
e.mulder@uniQure.com |
t.malone@uniQure.com |
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