uniQure N.V. (NASDAQ: QURE), a leading gene therapy company
advancing transformative therapies for patients with severe unmet
medical needs, today announced the publication of 26-week interim
data from the ongoing Phase IIb clinical trial of etranacogene
dezaparvovec, an investigational gene therapy for hemophilia B, in
the journal, Blood Advances.
With a combined follow-up of 78 weeks of
observation on the three adult male participants with severe
hemophilia B, the mean Factor IX (FIX) activity was 47 percent of
normal (range of 33 percent to 57 percent of normal) following a
single administration of etranacogene dezaparvovec (AMT-061).
Consistent with the observed Factor IX activity, the treatment was
associated with a complete cessation of bleeds up to the 26-week
interim endpoint, and no patient in the study required any
infusions of FIX replacement therapy during this time.
In the year prior to administration of
etranacogene dezaparvovec, all three participants received
prophylactic FIX replacement, as well as additional doses of FIX to
treat bleeding events. Medical records showed that the 3
participants experienced a total of 9 bleeds in the year prior to
treatment despite routine FIX prophylaxis.
Etranacogene dezaparvovec was generally well
tolerated, with no clinically significant elevations of liver
enzymes or inflammatory markers observed, and no use of
corticosteroids related to treatment required.
“In individuals with severe and moderately
severe hemophilia B, etranacogene dezaparvovec resulted in
clinically-relevant increases in FIX activity, cessation of bleeds
and abrogated the need for FIX replacement, despite the presence of
pre-existing anti-AAV5 neutralizing antibodies detected by the
highly sensitive luciferase assay,” 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 an investigator in the Phase IIb
clinical trial. “While these observations are limited to three
participants, the consistency of results among participants
supports the ongoing pivotal Phase III HOPE B trial which will
further assess the safety and efficacy of etranacogene
dezaparvovec. If these results are confirmed by the Phase III
trial, physicians will be provided with increased confidence that
reliable clinical outcomes can be achieved following gene
transfer.”
“This publication supports the potential of
etranacogene dezaparvovec to substantially improve the quality of
life for hemophilia B patients through a one-time administration
that results in sustained Factor IX activity and may result in a
cessation of bleeding episodes,” stated Robert Gut, M.D., Ph.D.,
chief medical officer at uniQure and co-author of the paper. “We
are pleased to have these interim safety and efficacy data of
etranacogene dezaparvovec published in a peer-reviewed journal and
look forward to sharing top-line data from the pivotal Phase III
HOPE-B study in 2020.”
The Company has completed patient enrollment in
the HOPE-B pivotal trial and expects top-line data by the end of
next year.
“Etranacogene dezaparvovec (AMT-061 Phase 2b):
Normal/Near Normal FIX Activity and Bleed Cessation in Hemophilia
B,” is available on-line in the journal, Blood Advances.
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 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 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 substantially improve the quality of
life for hemophilia B patients through a one-time administration or
to result in sustained Factor IX activity or to result in a
cessation of bleeding episodes, whether the Phase IIb or Phase III
trial data or any data will provide an increased confidence that
reliable clinical outcomes can be achieved following gene transfer,
and whether top-line data from the Phase III trial can be shared in
2020 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. CantorDirect: 339-970-7536Mobile:
617-680-9452m.cantor@uniQure.com |
Eva M. MulderDirect: +31 20 240 6103Mobile:
+31 6 52 33 15 79 e.mulder@uniQure.com |
Tom MaloneDirect: 339-970-7558Mobile:
339-223-8541t.malone@uniQure.com |
uniQure NV (NASDAQ:QURE)
Historical Stock Chart
From Mar 2024 to Apr 2024
uniQure NV (NASDAQ:QURE)
Historical Stock Chart
From Apr 2023 to Apr 2024