uniQure Announces Achievement of Planned Enrollment in HOPE-B Pivotal Trial of AMT-061 (Etranacogene Dezaparvovec) in Patient...
September 03 2019 - 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 the planned enrollment of 56
patients has been achieved in the HOPE-B pivotal trial of
etranacogene dezaparvovec (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. Due to the high level of interest in the study from
both patients and study investigators, uniQure expects to
over-enroll up to six additional patients before the end of
September. Etranacogene dezaparvovec 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 extremely pleased to reach this
important milestone in our ongoing development of etranacogene
dezaparvovec, which we believe has the potential to be the first
and best-in-class gene therapy for patients with hemophilia B,”
stated Matt Kapusta, chief executive officer of uniQure. “We
appreciate the tremendous support from the hemophilia patient
community in achieving this important goal ahead of schedule and
look forward to sharing top-line data from the Phase III trial,
which we expect to do next year.”
The pivotal Phase III HOPE-B study builds on the
success of the interim 36-week results of the Company’s ongoing
Phase IIb study of etranacogene dezaparvovec, which demonstrated
that a single administration of the investigational gene therapy
resulted in sustained increases in Factor IX (FIX) levels up to 54%
of normal, and a mean FIX level of 45% of normal. During that time,
no patient reported any bleeding events or required any infusion of
FIX replacement therapy for bleeds or experienced any material loss
of FIX activity. Additionally, an ongoing Phase I/II study of
AMT-060, the Company’s first-generation gene therapy for the
treatment of hemophilia B, demonstrated that all 10 patients
continue to show sustained and stable increases in FIX activity and
long-term clinical benefits at up to 3.5 years of observation.
“We are very excited and proud to have achieved
the targeted patient enrollment in the HOPE-B study in just a
little more than one year from study initiation,” added Robert Gut,
M.D., Ph.D., chief medical officer at uniQure. “This
multi-center, multinational trial involves 39 clinical sites across
9 countries, and highlights the outstanding effort and passion of
our clinical operations, clinical development, medical affairs and
project management teams. We would like to thank all study
participants, advisors, primary investigators and the whole study
staff in the United States and Europe for their great contribution
and support.”
About the Pivotal Phase III HOPE-B
Study
The pivotal Phase III HOPE-B trial is a
multinational, open-label, single-arm study to evaluate the safety
and efficacy of etranacogene dezaparvovec. Adult hemophilia B
patients classified as severe or moderately severe are 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 etranacogene
dezaparvovec at the 2x1013 gc/kg dose. Dosing of patients in the
HOPE-B pivotal trial was initiated in January 2019.
The study’s primary endpoint is the assessment
of Factor IX activity 26 weeks after dosing. Secondary
endpoints 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.
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-based gene therapies may be viable treatments for at least 97%
of patients.
About Etranacogene Dezaparvovec
(AMT-061)
Etranacogene dezaparvovec, also known as
AMT-061, consists of an AAV5 viral vector carrying a gene cassette
with the patent-protected Padua variant of Factor IX (FIX-Padua).
AAV5-based gene therapies have been demonstrated to be safe and
well tolerated in many 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-based 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 gene therapies to treat
patients with hemophilia B, hemophilia A, Huntington's disease,
Fabry disease, spinocerebellar ataxia Type 3 and other
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 ability of
etranacogene dezaparvovec to be a first-in-class or best-in-class
gene therapy for patients with hemophilia B, the ability to add up
to six additional patients before the end of September 2019, and
whether top-line data from the Phase III trial can be shared next
year or ever. 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 July 29, 2019.
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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