uniQure N.V. (NASDAQ: QURE), a leading gene therapy company
advancing transformative therapies for patients with severe medical
needs, announced that its partner, global biotechnology leader CSL
(ASX: CSL), has received approval from the U.S. Food and Drug
Administration (FDA) for HEMGENIX® (etranacogene
dezaparvovec-drlb), a one-time gene therapy for the treatment of
adults 18 years of age and older living with hemophilia B.
The product is approved for the treatment of
adults with hemophilia B who currently use factor IX prophylaxis
therapy or have current or historical life-threatening hemorrhage
or have repeated, serious spontaneous bleeding episodes. CSL
licensed the exclusive global rights to HEMGENIX from uniQure in
May 2021 and is now solely responsible for the further development,
registration, and commercialization of the therapy.
“Today’s landmark approval represents a major
milestone in the field of genomic medicine and ushers in a new
treatment paradigm for patients living with hemophilia B,” said
Matt Kapusta, chief executive officer of uniQure. “This is
uniQure’s second internally-developed gene therapy to achieve
approval and the world’s first gene therapy for hemophilia B, an
historic achievement based on more than a decade of research and
clinical development, as well as the tireless dedication of our
employees, clinicians, patients and their families. For nearly 25
years, uniQure has been driven by a singular mission: to transform
peoples’ lives by harnessing the power of gene therapy. This
groundbreaking approval delivers on this promise by providing
people with hemophilia B the possibility of being liberated from
burdensome infusions and an ability to unlock the potential in
their daily lives. We offer our sincere gratitude to the hemophilia
community, without whose years of dedication and invaluable support
this medical breakthrough could not have been achieved.”
Hemophilia B is a rare, lifelong bleeding
disorder caused by a single gene defect, resulting in insufficient
production of factor IX, a protein primarily produced by the liver
that helps blood clots form. Treatments for moderate to severe
hemophilia B include prophylactic infusions of factor IX
replacement therapy to temporarily replace or supplement low levels
of blood-clotting factor and, while these therapies are effective,
those with hemophilia B must adhere to strict, lifelong infusion
schedules. They may also still experience spontaneous bleeding
episodes as well as limited mobility, joint damage or severe pain
as a result of the disease. For appropriate patients, HEMGENIX
allows people living with hemophilia B to produce their own factor
IX, which can lower the risk of bleeding.
In the ongoing clinical trial, HEMGENIX reduced
the rate of annual bleeds and 94 percent of patients discontinued
factor IX prophylaxis and remained prophylaxis-free.
uniQure conducted the research and clinical
development for the product, which included three clinical trials
across 34 global sites and involving 67 adults with hemophilia B.
In May 2021, uniQure and CSL completed a licensing transaction
providing CSL Behring with exclusive rights to commercialize and
continue clinical development of HEMGENIX globally. uniQure is
responsible for the global manufacturing of the product at its
licensed Lexington, MA facility. Under the terms of the agreement,
uniQure has received payments from CSL totaling approximately $500
million and is eligible to receive up to an additional $1.5 billion
in commercial milestone payments and tiered, double-digit royalties
in a range up to a low-twenties percentage of net product sales
arising from the collaboration.
“CSL is proud to have been at the forefront of
providing life-changing medicines for rare diseases for over a
century. HEMGENIX, originally developed by our uniQure
colleagues, is just such a medicine. Today’s historic approval
builds on CSL’s promise to put patients first in all that we do to
discover, develop and deliver biotherapeutics and vaccines that
meet their needs,” said Dr. Bill Mezzanotte, head of research &
development and chief medical officer of CSL. “With HEMGENIX, CSL
now offers people living with Hemophilia B another remarkable
option for better and more durable control over their
disease.”
“We are thrilled to witness this milestone in
hemophilia B treatment,” shared Kim Phelan, chief operating officer
of the Coalition for Hemophilia B. “Over the years we have seen a
variety of advancements for the hemophilia community, but gene
therapy is the first treatment option to offer those living with
hemophilia B and caregivers the possibility of freedom from the
need for regular, ongoing infusions.”
The FDA approval is supported by results from
the pivotal HOPE-B trial, the largest gene therapy trial in
hemophilia B to date. Results from the study demonstrated that
HEMGENIX allowed patients to produce mean factor IX activity of 39
percent at six months and 36.7 percent at 24 months post infusion.
Seven to 18 months post-infusion, the mean adjusted annualized
bleeding rate (ABR) for all bleeds was reduced by 54 percent
compared to the six-month lead-in period on factor IX prophylactic
replacement therapy (4.1 to 1.9). In addition, 94 percent (51 out
of 54) of patients treated with HEMGENIX discontinued use of
prophylaxis and remained free of previous continuous routine
prophylaxis therapy. The most common side effects (incidence ≥5%)
were liver enzyme elevations, headache, elevated levels of a
certain blood enzyme, flu-like symptoms, infusion-related
reactions, fatigue, nausea and feeling unwell.
“HEMGENIX is unique in its approach to increase
mean factor IX activity and hemostatic protection in those with
hemophilia B, and today’s approval could fundamentally transform
the treatment paradigm for this life-long condition,” said Dr.
Steven Pipe, professor and the Laurence A. Boxer Research professor
of pediatrics and professor of pathology at the University of
Michigan and a lead investigator in the HOPE-B study. “As a
clinician, I look forward to being able to provide a new treatment
option that may help patients treated with HEMGENIX become free
from the regular infusion schedule that many people living with
hemophilia B rely on to protect them from the debilitating effects
of the condition.”
HEMGENIX is currently under assessment by other
regulatory agencies. Product information on HEMGENIX, including its
prescribing information, will be provided by CSL Behring.
About Hemophilia
B Hemophilia B is a life-threatening rare 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 treatments for moderate to
severe hemophilia B include life-long prophylactic infusions of
factor IX to temporarily replace or supplement low levels of the
blood-clotting factor.
About HEMGENIX HEMGENIX is
a gene therapy that reduces the rate of abnormal bleeding in
eligible people with hemophilia B by enabling the body to
continuously produce factor IX, the deficient protein in hemophilia
B. It uses AAV5, a non-infectious viral vector, called an
adeno-associated virus (AAV). The AAV5 vector carries the Padua
gene variant of Factor IX (FIX-Padua) to the target cells in the
liver, generating factor IX proteins that are 5x-8x more active
than normal. These genetic instructions remain in the target cells,
but generally do not become a part of a person’s own DNA. Once
delivered, the new genetic instructions allow the cellular
machinery to produce stable levels of factor IX.
About the Pivotal HOPE-B
TrialThe pivotal Phase III HOPE-B trial is an ongoing,
multinational, open-label, single-arm study to evaluate the safety
and efficacy of HEMGENIX. Fifty-four adult hemophilia B patients
classified as having moderately severe to severe hemophilia B and
requiring prophylactic factor IX replacement therapy were enrolled
in a prospective, six-month or longer 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 HEMGENIX 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
HEMGENIX 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 ABR 52 weeks after achievement of stable factor IX
expression (months 7 to 18) 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 factor IX transgene expression.
Secondary endpoints included assessment of factor IX activity.
No serious adverse reactions were reported. 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 HEMGENIX by independent molecular
tumor characterization and vector integration analysis. No
inhibitors to factor IX were reported.
Important Safety
Information (ISI)
What is HEMGENIX?HEMGENIX®,
etranacogene dezaparvovec-drlb, is a one-time gene therapy for the
treatment of adults with hemophilia B who:
- Currently use Factor IX prophylaxis
therapy, or
- Have current or historical
life-threatening bleeding, or
- Have repeated, serious spontaneous
bleeding episodes.
HEMGENIX is administered as a single intravenous
infusion and can be administered only once.
What medical testing can I expect to be
given before and after administration of HEMGENIX?To
determine your eligibility to receive HEMGENIX, you will be tested
for Factor IX inhibitors. If this test result is positive, a retest
will be performed 2 weeks later. If both tests are positive for
Factor IX inhibitors, your doctor will not administer HEMGENIX to
you. If, after administration of HEMGENIX, increased Factor IX
activity is not achieved, or bleeding is not controlled, a
post-dose test for Factor IX inhibitors will be performed.
HEMGENIX may lead to elevations of liver enzymes
in the blood; therefore, ultrasound and other testing will be
performed to check on liver health before HEMGENIX can be
administered. Following administration of HEMGENIX, your doctor
will monitor your liver enzyme levels weekly for at least 3 months.
If you have preexisting risk factors for liver cancer, regular
liver health testing will continue for 5 years post-administration.
Treatment for elevated liver enzymes could include
corticosteroids.
What were the most common side effects
of HEMGENIX in clinical trials?In clinical trials for
HEMGENIX, the most common side effects reported in more than 5% of
patients were liver enzyme elevations, headache, elevated levels of
a certain blood enzyme, flu-like symptoms, infusion-related
reactions, fatigue, nausea, and feeling unwell. These are not the
only side effects possible. Tell your healthcare provider about any
side effect you may experience.
What should I watch for during infusion
with HEMGENIX?Your doctor will monitor you for
infusion-related reactions during administration of HEMGENIX, as
well as for at least 3 hours after the infusion is complete.
Symptoms may include chest tightness, headaches, abdominal pain,
lightheadedness, flu-like symptoms, shivering, flushing, rash, and
elevated blood pressure. If an infusion-related reaction occurs,
the doctor may slow or stop the HEMGENIX infusion, resuming at a
lower infusion rate once symptoms resolve.
What should I avoid after receiving
HEMGENIX?Small amounts of HEMGENIX may be present in your
blood, semen, and other excreted/secreted materials, and it is not
known how long this continues. You should not donate blood, organs,
tissues, or cells for transplantation after receiving HEMGENIX.
Please see full prescribing
information for HEMGENIX.
You are encouraged to report negative side effects of
prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call
1-800-FDA-1088.
You can also report side effects to CSL Behring’s
Pharmacovigilance Department at 1-866-915-6958.
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
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," “establish,” "estimate,"
"expect," "goal," "intend," "look forward to", "may," "plan,"
"potential," "predict," "project," “seek,” "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,
statements about whether we are able to bring AMT-061 to people
living with hemophilia B and whether the treatment will be
transformational. 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 postponement in our clinical
trial for Huntington’s disease, the impact of financial and
geopolitical events 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: |
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FOR INVESTORS: |
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FOR MEDIA: |
Maria E. CantorDirect: 339-970-7536Mobile:
617-680-9452m.cantor@uniQure.com |
Chiara RussoDirect: 617-306-9137Mobile:
617-306-9137c.russo@uniQure.com |
Tom MaloneDirect:
339-970-7558Mobile:339-223-8541t.malone@uniQure.com |
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Photos accompanying this announcement are available
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