uniQure N.V. (NASDAQ: QURE), a leading gene therapy company
advancing transformative therapies for patients with severe medical
needs, today announced updated clinical data on the three patients
treated in uniQure’s ongoing Phase IIb study of etranacogene
dezaparvovec, 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. In addition,
uniQure presented up to 4 years of follow-up data on the 10
patients in the Phase I/II trial of AMT-060, its first-generation
gene therapy for the treatment of hemophilia B. These clinical data
are being presented this weekend in poster presentations at the
61st Annual Meeting of the American Society of Hematology (ASH),
taking place in Orlando, Florida.
At Least One Year of Stable, Therapeutic
Levels of FIX Activity in Patients Treated with Etranacogene
Dezaparvovec
The Phase IIb study of etranacogene dezaparvovec
is an open-label, single-dose, single-arm, multi-center trial being
conducted in the United States. Three patients with severe
hemophilia (endogenous Factor IX (FIX) activity less than or equal
to one percent) were enrolled in the study and received a single
intravenous infusion of 2x1013 gc/kg. Prior to the administration
of etranacogene dezaparvovec, all three patients showed low levels
of pre-existing neutralizing antibodies to AAV5 but were not
excluded from the trial on that basis. The patients in the Phase
IIb study were 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 etranacogene
dezaparvovec.
Featured in a poster presentation at ASH, the 52
weeks of follow-up data show that all three patients have
stabilized and sustained FIX activity at therapeutic levels after
the one-time administration of etranacogene dezaparvovec. Mean FIX
activity for the three patients at 52 weeks after administration
was 41% of normal, with the first patient achieving FIX activity of
50% of normal, the second patient achieving FIX activity of 31% of
normal and the third patient achieving FIX activity of 41% of
normal. 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.
At one year after dosing, no patient in the
study has reported any bleeding events. All patients have remained
free of prophylaxis after receiving etranacogene dezaparvovec. One
patient, who had earlier in the study undergone hip surgery due to
a pre-existing condition, experienced back pain and treated himself
with a single infusion of factor replacement, which was later
determined by the patient and the investigator to be unrelated to a
bleed. No other FIX infusions were reported by any patient.
“These updated data show that a single
administration of etranacogene dezaparvovec has been well-tolerated
now out 52 weeks and has increased FIX activity into the
therapeutic range for people living with hemophilia B,” stated
Steven Pipe, M.D., professor of pediatrics and pathology and
pediatric medical director of the hemophilia and coagulation
disorders program at the University of Michigan and a principal
investigator in the HOPE-B clinical trial. “These data show a full
year of meaningful clinical benefit for all three patients in the
study, including durable levels of FIX activity with no bleeds, no
requirement for infusions of FIX replacement therapy outside of
surgery, and no need for immunosuppression.”
“We are very pleased with these latest results,
and continue to believe that etranacogene dezaparvovec has the
potential to be the first- and best-in-class gene therapy for
patients with hemophilia B,” stated Robert Gut, M.D., Ph.D., chief
medical officer of uniQure. “We remain focused on dosing all
patients in our ongoing, fully-enrolled HOPE-B pivotal trial, and
expect to announce top-line data on our primary endpoint of Factor
IX activity by the end of 2020.”
Stable FIX Expression and Durable
Reductions in Bleeding and FIX Consumption for up to 4 Years
Following AMT-060 Gene Therapy
In the ongoing Phase I/II study of AMT-060, all
10 patients continue to show long-term clinical benefit, including
sustained increases in FIX activity, reduced usage of FIX
replacement therapy, and decreased bleeding frequency. At up to 4
years of follow-up, AMT-060 continues to be well-tolerated, with no
new serious adverse events and no development of inhibitors since
the last reported data.
All five patients in the second dose cohort of
2x1013 gc/kg (the dose being studied in the ongoing Phase III
HOPE-B study of etranacogene dezaparvovec) continue to be free of
routine FIX replacement therapy at 3.5 years after treatment. Based
on the six months of data collected during the fourth year of
follow-up, the mean annualized bleeding rate was zero compared to
an average of 4 bleeds during the year prior to treatment,
representing a 100% reduction. During this same period, the usage
of FIX replacement therapy also declined 100% compared to the year
prior to treatment. Mean FIX activity over 3.5 years was 7.5%.
AMT-060 is uniQure’s first-generation gene
therapy, consisting of an AAV5 vector carrying a gene cassette with
the wild-type FIX gene. uniQure expects that data from this Phase
I/II trial of AMT-060 will be part of the regulatory submission for
marketing approval of etranacogene dezaparvovec.
“The Phase I/II study of AMT-060 continues to
demonstrate notable long-term tolerability,” stated Professor
Wolfgang Miesbach, M.D., Ph.D., of the University Hospital
Frankfurt in Germany. “We have now demonstrated evidence of durable
clinical benefits, including sustained FIX activity, improved
disease phenotype and substantial reductions in spontaneous bleeds
for up to 4 years following administration. These data have the
potential to be very meaningful for hemophilia B patients.”
About Etranacogene
Dezaparvovec
Etranacogene dezaparvovec 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.
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 uniQure’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 viable treatments 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, open-label, single-arm study to evaluate the safety
and efficacy of etranacogene dezaparvovec. Sixty-two adult
hemophilia B patients classified as severe or moderately severe
were enrolled in a six-month observational period, during which
time they continued to use their current standard of care to
establish a baseline control. After the six-month lead-in period,
patients 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.
Investor/Analyst Breakfast and Webcast
Information
uniQure will host an investor breakfast and
webcast on Monday, December 9, 2019 at 7:00 a.m. EST featuring
Dr. Steven Pipe, principal investigator of the HOPE-B clinical
trial, to review the updated data on etranacogene dezaparvovec. To
access the call by phone, dial 1-866-966-1396 (United States) or
+44 20 719 280 00 (international); the conference ID is 41 64
967.
The webcast may also be accessed through the
Investors section of the uniQure’s website at
http://uniqure.com/investors-newsroom/overview.php. Following the
live webcast, a replay of the call will be available for two
weeks.
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 B, hemophilia A,
Huntington's disease, Fabry disease, spinocerebellar ataxia Type 3
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, whether etranacogene
dezaparvovec will be the first-in-class or best-in-class gene
therapy for patients with hemophilia B, whether uniQure will dose
all patients in its ongoing HOPE-B pivotal trial, whether uniQure
will announce top-line data on the primary endpoint of Factor IX
activity in its HOPE-B pivotal trial by the end of 2020 or ever,
and whether any clinical data associated with etranacogene
dezaparvovec or AMT-060 will be meaningful for hemophilia B
patients. 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 October 28, 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 Direct:
339-970-7536Mobile: 617-680-9452m.cantor@uniQure.com |
Eva M. Mulder Direct: +31 20 240 6103
Mobile: +31 6 52 33 15 79e.mulder@uniQure.com |
Tom MaloneDirect: 339-970-7558Mobile:
339-223-8541t.malone@uniQure.com |
____________1 Epidemiological data indicate that factor activity
above 12% of normal is associated with substantial reduction or
elimination of spontaneous bleeds and factor usage. Den Uijl IE et
al Haemophilia 2011; 17(6):849-53
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