uniQure Presents HOPE-B Clinical Data Demonstrating Clinical Benefit in Hemophilia B Patients with Pre-existing Antibodies to...
May 13 2021 - 7:00AM
uniQure Presents HOPE-B Clinical Data Demonstrating Clinical
Benefit in Hemophilia B Patients with Pre-existing Antibodies to
AAV5 Vector
uniQure N.V. (NASDAQ: QURE), a leading gene therapy company
advancing transformative therapies for patients with severe medical
needs, yesterday presented 26-week clinical data from the pivotal,
Phase III HOPE-B gene therapy trial of etranacogene dezaparvovec
showing clinical benefit in hemophilia B patients with pre-existing
neutralizing antibodies (NAbs) to AAV5, and no relationship between
AAV5 NAbs and the tolerability of the therapy.
The data were featured on Wednesday, May 12,
2021 at the American Society of Gene and Cell Therapy (ASGCT)
Annual Meeting in an oral presentation by Michael Recht, M.D.,
Ph.D., M.B.A., professor of pediatrics, division of hematology and
oncology at the Oregon Health & Science University School of
Medicine. These data were previously presented at a medical meeting
late last year.
“The ASGCT presentation highlights these initial
HOPE-B data that demonstrate the successful treatment with an AAV5
gene therapy of patients with pre-existing NAbs,” stated Ricardo
Dolmetsch, Ph.D., president of Research and Development at uniQure.
“Patients in the trial who may not have been eligible for other
gene therapies because of pre-existing neutralizing antibodies have
achieved similar results with etranacogene dezaparvovec compared to
those who did not have pre-existing NAbs. We believe this
distinguishes etranacogene dezaparvovec as the only hemophilia gene
therapy shown in a clinical trial to have the potential to treat
nearly all patients, regardless of NAb levels in the generally
prevalent range.”
Patients in the Phase III HOPE-B clinical study
were initially enrolled into a prospective, observational lead-in
period of at least six months during which bleeding events and FIX
replacement therapy usage were monitored. All patients required
prophylactic routine FIX replacement prior to entering the clinical
trial, and patients were not excluded from the trial based on
pre-existing NAbs to AAV5.
Fifty-four participants were dosed and completed
26 weeks of follow-up, 31 of whom (57.4%) had no pre-existing NAbs
to AAV5. Of the 23 participants (42.6%) with pre-existing NAbs, the
median titer was 56.9, with a distribution representative of the
general population.
No clinically significant correlation of
pre-existing NAbs with FIX activity was observed up to a titer of
678, a range expected to include more than 95 percent of the
general population. Mean FIX activity at 26 weeks was 32.7 percent
in participants with NAbs versus 41.3 percent in those without. A
single participant with a NAb titer of 3,212 did not respond and
remained on prophylaxis; it is expected that less than 1 percent of
the general population have a pre-existing NAb titer of more than
3,000. All other participants, both with and without NAbs,
discontinued prophylaxis and remained prophylaxis-free at 26
weeks.
The data presentation from ASGCT can be found on
the Investors & Newsroom section of uniQure’s corporate
website.
About Etranacogene Dezaparvovec
Etranacogene dezaparvovec (AMT-061) is an investigational
adeno-associated virus five (AAV5)-based gene therapy for the
treatment of patients with severe and moderately severe hemophilia
B. Etranacogene dezaparvovec consists of an AAV5 viral vector
carrying a gene cassette with the patent-protected Padua variant of
Factor IX (FIX-Padua). uniQure and CSL Behring have entered into a
commercialization and license agreement providing CSL Behring
exclusive global commercialization rights to etranacogene
dezaparvovec. The collaboration combines uniQure’s differentiated
gene therapy candidate in hemophilia B and CSL Behring’s strong
global reach and commercial infrastructure in hematology in an
effort to accelerate access of etranacogene dezaparvovec to
hemophilia B patients around the world.
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,
Huntington's disease, Fabry disease, spinocerebellar ataxia Type 3
and other 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, whether etranacogene dezaparvovec
or AAV5-based gene therapies generally can provide clinical benefit
to patients with pre-existing neutralizing antibodies.
uniQure’s actual results could differ materially from those
anticipated in these forward-looking statements for many reasons,
including, without limitation, risks associated with the impact of
the ongoing COVID-19 pandemic on our Company and the wider economy
and health care system, our Commercialization and License Agreement
with CSL Behring, our clinical development activities, clinical
results, collaboration arrangements, 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
May 10, 2021. 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 |
Chiara Russo |
Tom Malone |
Direct:
339-970-7536 |
Direct:
617-306-9137 |
Direct:
339-970-7558 |
Mobile:
617-680-9452 |
Mobile:
617-306-9137 |
Mobile:
339-223-8541 |
m.cantor@uniQure.com |
c.russo@uniQure.com |
t.malone@uniQure.com |
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