uniQure N.V. (NASDAQ: QURE), a leading gene therapy company
advancing transformative therapies for patients with severe medical
needs, today announced that the U.S. Food and Drug Administration
(FDA) has accepted for priority review the Biologics License
Application (BLA) submitted by CSL Behring for etranacogene
dezaparvovec, an investigational gene therapy for the treatment of
adults with hemophilia B.
CSL Behring is uniQure’s global
commercialization partner for etranacogene dezaparvovec and is
solely responsible for all regulatory activities, including filings
and agency interactions, associated with etranacogene
dezaparvovec.
uniQure led the multi-year clinical development
of etranacogene dezaparvovec, including the pivotal Phase III
HOPE-B clinical trial, and the company will be responsible for the
global commercial supply of etranacogene dezaparvovec.
"Etranacogene dezaparvovec has the potential to
be the first gene therapy approved in hemophilia B and the
acceptance of the BLA marks an important milestone in uniQure’s
mission of delivering the promise of gene therapy to people living
with rare diseases,” stated Matt Kapusta, chief executive officer
of uniQure. “uniQure has been a leader in gene therapy for nearly
25 years and, if approved, etranacogene dezaparvovec would
represent our second gene therapy to complete its journey to
patients. We are proud of this accomplishment, which is a
culmination of significant contributions from all our employees,
clinical investigators and the hemophilia community. We are pleased
with the promising results generated from our HOPE-B pivotal study,
the largest gene therapy trial in hemophilia B to date and,
assuming FDA approval, we look forward to partnering with our
colleagues at CSL Behring to bring this life-changing treatment
option to people with hemophilia B.”
Etranacogene dezaparvovec was specifically
designed to make near-normal blood-clotting ability possible by
addressing the underlying cause of hemophilia B: a faulty gene that
causes a deficiency in clotting Factor IX (FIX). Etranacogene
dezaparvovec has been shown in clinical trials to significantly
reduce the rate of annual bleeds in trial participants after a
single one-time infusion and, if approved, would be the first ever
gene therapy treatment option for the hemophilia B community.
The BLA is supported by results from the pivotal
HOPE-B trial, which demonstrated in trial participants a reduction
in adjusted annualized bleeding rate (ABR) of 64% and superiority
to prophylaxis treatment at 18 months post-treatment compared to a
6-month run in period (p=0.0002).
Priority review of a BLA is reserved for
medicines that, if approved, would be potentially significant
improvements in the safety or effectiveness of the treatment,
diagnosis, or prevention of serious conditions when compared to
standard applications. Upon acceptance for priority review, the FDA
goal is to take action on the BLA in 6 months as compared to 10
months for standard review. Previously, the European Medicines
Agency (EMA) accepted the Marketing Authorization Application
(MAA) for etranacogene dezaparvovec under its accelerated
assessment procedure.
About Hemophilia BHemophilia B
is a life-threatening degenerative disease. People with the
condition are particularly vulnerable to bleeds in their joints,
muscles, and internal organs, leading to pain, swelling, and joint
damage. Current treatment includes life-long prophylactic infusions
of FIX to temporarily replace or supplement low levels of the
blood-clotting factor.
About Gene Therapy in Hemophilia
BGene therapy has the potential to enable more normal
clotting in patients with hemophilia B. Gene therapy achieves this
with modified non-infectious viruses that serve as “vectors” that
can enter certain cells. Vectors carry genetic instructions to
specific cells. Once delivered, the new genetic instructions allow
the cellular machinery to produce their own stable levels of FIX. A
certain type of vector, called an adeno-associated virus, or AAV,
dissolves after delivering its genetic instructions. These genetic
instructions remain in the target cells, but never actually become
a part of a person’s own DNA.
About Etranacogene
DezaparvovecEtranacogene dezaparvovec (also known as
CSL222, previously known as AMT-061) uses a specific type of AAV,
called AAV5, as its vector. The AAV5 vector carries the Padua gene
variant of FIX (FIX-Padua), which generates FIX proteins that are
5x-8x more active than normal. Preclinical and clinical data show
that AAV5-based gene therapies may be clinically effective in a
large percentage of hemophilia B patients with pre-existing
antibodies to AAV vectors, thereby potentially increasing patient
eligibility for treatment.
About the Pivotal HOPE-B
TrialThe pivotal Phase III HOPE-B trial is a
multinational, open-label, single-arm study to evaluate the safety
and efficacy of etranacogene dezaparvovec. Fifty-four adult
hemophilia B patients requiring prophylactic FIX replacement
therapy were enrolled in a prospective, six-month observational
period during which time they continued to use their current
standard of care therapy to establish a baseline Annual Bleeding
Rate (ABR). After the six-month lead-in period, patients received a
single intravenous administration of etranacogene dezaparvovec at
the 2x10^13 gc/kg dose. Patients were not excluded from the trial
based on pre-existing neutralizing antibodies (NAbs) to AAV5. A
total of 54 patients received a single dose of etranacogene
dezaparvovec in the pivotal trial, with 53 patients completing at
least 18 months of follow-up. The primary endpoint in the pivotal
HOPE-B study was 52-week ABR after achievement of stable FIX
expression compared with the six-month lead-in period. For this
endpoint, ABR was measured from month seven to month 18 after
infusion, ensuring the observation period represented a
steady-state FIX transgene expression. Secondary endpoints included
assessment of FIX activity and statistical superiority of ABR after
dosing.
Results from the pivotal HOPE-B study
demonstrated that etranacogene dezaparvovec produced mean FIX
activity of 39.0 IU/dL at six months and 36.9 IU/dL at 18 months
post infusion. After the six-month lead-in period post-infusion,
the adjusted annualized bleeding rate (ABR) for all bleeds was
reduced by 64 percent (4.19 to 1.51 p=0.0002) and all FIX-treated
bleeds was reduced by 77 percent (3.65 to 0.83; p<0.0001) over
months seven to 18. In addition, 98 percent of subjects treated
with a full dose of etranacogene dezaparvovec discontinued use of
prophylaxis, with an overall 97 percent reduction in mean
unadjusted annualized FIX consumption of 257338.8
IU/yr/participant to 8486.6 IU/yr/participant (from lead-in
period to months 13-18).
Etranacogene dezaparvovec was generally
well-tolerated with the majority of adverse events (80.4 percent)
considered mild. One death resulting from urosepsis and cardiogenic
shock in a 77-year-old patient at 65 weeks following dosing was
considered unrelated to treatment by investigators and the company
sponsor. A serious adverse event of hepatocellular carcinoma was
determined to be unrelated to treatment with etranacogene
dezaparvovec by independent molecular tumor characterization and
vector integration analysis. No inhibitors to FIX were
reported.
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, refractory temporal lobe epilepsy, Fabry
disease, 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, the potential timelines
for and outcome of BLA review. The Company’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 the
Company’s periodic securities filings, including its Annual Report
on Form 10-K filed February 25, 2022. Given these risks,
uncertainties and other factors, you should not place undue
reliance on these forward-looking statements, and the Company
assumes no obligation to update these forward-looking statements,
even if new information becomes available in the future.
uniQure Contacts:
FOR INVESTORS: |
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FOR MEDIA: |
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Maria E. Cantor |
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Chiara Russo |
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Tom Malone |
Direct: 339-970-7536 |
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Direct: 617-306-9137 |
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Direct: 339-970-7558 |
Mobile: 617-680-9452 |
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Mobile: 617-306-9137 |
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Mobile:339-223-8541 |
m.cantor@uniQure.com |
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c.russo@uniQure.com |
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t.malone@uniQure.com |
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