uniQure Announces First Patient Treated in HOPE-B Pivotal Trial of AMT-061 in Patients with Hemophilia B
February 04 2019 - 7:00AM
uniQure Announces First Patient Treated in HOPE-B Pivotal Trial of
AMT-061 in Patients with Hemophilia B
uniQure N.V. (NASDAQ: QURE), a leading gene therapy company
advancing transformative therapies for patients with severe unmet
medical needs, today announced that it treated the first patient in
its HOPE-B pivotal trial of AMT-061, an investigational AAV5-based
gene therapy incorporating the patent-protected 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.
“We are very pleased to have successfully
administered AMT-061 to the first patient enrolled in the HOPE-B
pivotal trial and mark this as a milestone for the field in
advancing a potential one-time 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 advance this study, and we plan to complete full
patient enrollment in this study before the end of this
year.”
“Our mission in hemophilia B has always been to
be first to market with a best-in-class gene therapy,” said Matt
Kapusta, chief executive officer. “With the initiation of the
dosing phase of our pivotal study for AMT-061 and the data
generated to date in our Phase IIb study, we believe that we are on
track toward our goal of developing the first gene therapy that
provides durable, functionally-curative benefits to nearly all
patients with hemophilia B, without the complications associated
with capsid-related immune responses or the need for
immunosuppression therapy.”
The global HOPE-B Phase III clinical trial will
evaluate the efficacy and safety of AMT-061. Approximately 50 adult
patients with hemophilia B classified as severe or 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 prospective comparator. After the six-month
lead-in period, patients will receive a single intravenous
administration of AMT-061. The primary outcome measure is the
assessment of Factor IX activity 26 weeks after AMT-061 dosing.
Secondary outcome measures include annualized bleeding rate (ABR)
and usage of Factor IX replacement therapy over a 52-week
time frame, as well as other efficacy and safety aspects.
Post-treatment, patients will be followed for 5 years.
About AMT-061
AMT-061 consists of an AAV5 viral vector
carrying a gene cassette with the patent-protected Padua variant of
Factor IX (FIX-Padua). uniQure holds multiple issued patents in the
United States and Canada broadly covering methods of treating
bleeding disorders, including hemophilia B, using AAV gene therapy
with the FIX-Padua variant. Additional patents are pending in the
European Union.
AAV5-based gene therapies have been demonstrated
to be safe and well-tolerated in a multitude of clinical trials,
including four uniQure trials conducted in 25 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.
Additionally, preclinical and clinical data show that AAV5-based
gene therapies may be clinically effective in patients with
pre-existing antibodies to AAV5, thereby potentially increasing
patient eligibility for treatment compared to other gene therapy
product candidates.
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 other severe genetic 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 our
Phase IIb study, the completion of full patient enrollment for the
HOPE-B Phase III pivotal trial of AMT-061 before the end of this
year, our ability to be first to market, our ability to develop a
best-in-class gene therapy, our ability to develop a gene therapy
that provides durable, functionally-curative benefits to nearly all
patients with hemophilia B, without the complications associated
with capsid-related immune responses or the need for
immunosuppression, the ability of AMT-061 to be a successful gene
therapy treatment or to deliver sustained increases in FIX activity
or to provide a favorable immunogenicity profile or to eliminate
the risk of an immune response, the determination that any of
AMT-060, AMT-061 and the AAV5 capsid used in those product
candidates are safe or effective or will be determined by any
regulatory body to be safe and effective, the risk of cessation,
delay or lack of success of any of our ongoing or planned clinical
studies such as the dosing of patients in the HOPE-B pivotal trial
in the first quarter of 2019 or at any time, and/or the development
and regulatory approval of our product candidates in the United
States or in Europe. 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, clinical
results, 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 6, 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: |
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FOR MEDIA: |
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Maria E. Cantor |
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Eva M. Mulder |
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Tom Malone |
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Direct: 339-970-7536 |
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Direct: +31 20 240 6103 |
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Direct: 339-970-7558 |
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Mobile: 617-680-9452 |
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Mobile: +31 6 52 33 15 79 |
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Mobile: 339-223-8541 |
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m.cantor@uniQure.com |
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e.mulder@uniQure.com |
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t.malone@uniQure.com |
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