uniQure Announces Updated Clinical Data from Phase IIb Study 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 updated clinical data in patients
treated in the Company’s ongoing Phase IIb study of AMT-061, an
investigational AAV5-based gene therapy containing a
patent-protected FIX-Padua variant, for the treatment of patients
with severe and moderately severe hemophilia B. These data were
presented in an oral session at the Annual Congress of the European
Association for Haemophilia and Allied Disorders (EAHAD) and show
that therapeutic levels of Factor IX (FIX) activity continue to be
sustained in all three patients up to sixteen weeks after a single
administration of AMT-061.* AMT-061 has been granted Breakthrough
Therapy Designation by the United States Food and Drug
Administration and access to the Priority Medicine (PRIME)
regulatory initiative by the European Medicines Agency.
The Phase IIb study of AMT-061 is an open-label,
single-dose, single-arm, multi-center trial being conducted in the
United States. Three patients with severe hemophilia (endogenous
FIX activity less than one percent) were enrolled in the study and
received a single intravenous infusion of 2x1013 vc/kg. Prior to
the administration of AMT-061, all three patients showed low levels
of pre-existing antibodies to AAV5 but were not excluded from the
trial on that basis.
Updated data presented at EAHAD show that all
three patients have demonstrated increasing and sustained FIX
levels after the one-time administration of AMT-061. Mean FIX
activity for the three patients at twelve weeks increased to 38% of
normal, exceeding threshold FIX levels generally considered
sufficient to eliminate or significantly reduce the risk of
bleeding events. The first patient achieved FIX activity of 48% of
normal at sixteen weeks after administration. FIX activity in the
second patient was 25% of normal at fourteen weeks after
administration, and the third patient achieved FIX activity of 51%
of normal at twelve weeks after administration. The second and
third patients had previously screen-failed and were excluded from
another gene therapy study due to pre-existing neutralizing
antibodies to a different AAV vector. Reported FIX activity was
measured using an activated partial thromboplastin time (aPTT)
assay performed at a central laboratory.
“We are extremely pleased with these updated
data,” stated Robert Gut, M.D., Ph.D., chief medical officer of
uniQure. “The study demonstrates AMT-061 has the potential to
increase FIX activity into the normal range and continues to be
very well tolerated, with no serious adverse events reported and no
patients requiring any immunosuppression therapy. We look forward
to providing further updates on these patients later in the year at
other academic conferences.”
No patient in the study has experienced a
material loss of FIX activity, reported any bleeding events or
required any infusions of FIX replacement therapy. As previously
reported, one patient experienced slight elevations in aspartate
aminotransferase (AST), which quickly resolved without any
additional treatment or loss of FIX activity. No patient has
experienced any material elevation in alanine aminotransferase
(ALT) after the administration of AMT-061.
“Our goal with AMT-061 is to give all people
living with hemophilia B access to a one-time treatment capable of
normalizing FIX activity and eliminating the need for replacement
therapy, without the risk of immune responses that require
immunosuppression or may lead to a loss of efficacy,” stated Matt
Kapusta, chief executive officer of uniQure. “These updated data
continue to suggest that AMT-061 may be the first gene therapy able
to achieve this goal, and we remain focused on completing
enrollment in our ongoing pivotal Phase III study by the end of the
year.”
Patients in the Phase IIb study will be followed
for 52 weeks to assess FIX activity, bleeding rates and usage of
FIX replacement therapy, and will be monitored for five years to
evaluate the safety of AMT-061.
Investor Conference Call and Webcast
Information
uniQure will host a conference call today,
February 8, 2019 at 8:30 a.m. ET, to discuss these updated clinical
data from the Phase IIb study of AMT-061. To access the call by
phone, dial 1-866-966-1396 (United States) or +44 (0) 20 719 280 00
(international); the conference ID is 3588567.
The webcast may also be accessed through the
Investors section of the Company's website at www.uniQure.com.
Following the live webcast, a replay of the call will be available
for two weeks.
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 the Pivotal, Phase III HOPE-B
Study
The pivotal, Phase III HOPE-B trial is a
multinational, multi-center, open-label, single-arm study to
evaluate the safety and efficacy of AMT-061. Approximately 50 adult
hemophilia B patients 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 baseline control. After the six-month lead-in period,
patients will receive a single intravenous administration of
AMT-061. Dosing of patients in the HOPE-B pivotal trial is now
underway.
The primary endpoint of the study will be based
on the FIX activity level achieved following the administration of
AMT-061, and the secondary endpoints will measure annualized FIX
replacement therapy usage, annualized bleed rates and safety.
Patients enrolled in the HOPE-B pivotal trial
will be tested for the presence of pre-existing neutralizing
antibodies to AAV5 but will not be excluded from the trial based on
their titers. Previous studies performed by uniQure suggest that
AAV5 gene therapies may be viable treatments for at least 97% of
patients.
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, our ability to achieve the target profile for
AMT-061, our ability to provide all people living with hemophilia B
access to a one-time treatment capable of normalizing FIX activity
and eliminating the need for replacement therapy, without the risk
of immune responses that require immunosuppression or may lead to a
loss of efficacy, the ability of AMT-061 to deliver functionally
curative increases in FIX activity or to provide a favorable
immunogenicity profile or to eliminate the risk of an immune
response that may lead to a loss of efficacy or to expand patient
eligibility for treatment with gene therapy or to be the first to
market, the achievement of any of our planned near term or other
milestones, our ability to provide further clinical updates on the
Phase IIb study at medical conferences in 2019 or at any time, our
ability to complete enrollment in our pivotal Phase III trial of
AMT-061, 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: |
|
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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