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) today announced the publication in the New England
Journal of Medicine (NEJM) (Vol. 388 No. 8) of results from the
pivotal HOPE-B clinical study evaluating the efficacy, durability
and safety of HEMGENIX® (etranacogene dezaparvovec-drlb). HEMGENIX®
is the first and only gene therapy approved 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.
The multi-year clinical development of HEMGENIX®
was led by uniQure and sponsorship of the clinical trials
transitioned to CSL after it licensed global rights to
commercialize the treatment. HEMGENIX® was approved in November
2022 by the U.S. Food and Drug Administration (FDA), and in
February 2023 by the European Commission (EC) for the European
Union.
Results from the HOPE-B trial, the largest gene
therapy study in hemophilia B to date, were considered by the
authors to demonstrate that HEMGENIX® is superior to routine factor
IX prophylaxis in Annualized Bleeding Rate (ABR), factor IX
activity, factor IX therapy consumption, factor IX infusion rate,
and spontaneous and joint bleeding ABR. Increased factor IX
activity was also apparent from week 3 and maintained over
18 months. There were no reported serious adverse events
related to treatment with HEMGENIX®.
Notably, the results published in NEJM show the
ABR of spontaneous bleeding episodes and all joint bleeding
episodes decreased by 71% (95% CI: 0.12, 0.71) and 78% (95% CI:
0.10, 0.46), respectively, from lead-in period to post-treatment.
Further, 96.3% of patients discontinued factor IX prophylaxis from
day 21 through month 18 post-treatment. Annualized factor IX
infusions also significantly decreased from 72.5 infusions per
participant during lead-in period (95% CI: 63.6, 82.7) to
2.5 infusions (95% CI: 0.92, 6.96)
post-treatment.
The most common adverse reactions (incidence
≥5%) were elevated ALT, headache, blood creatine kinase elevations,
flu-like symptoms, infusion-related reactions, fatigue, malaise and
elevated AST.
“The results published in NEJM add to the
established body of evidence demonstrating the long-term efficacy
and safety of HEMGENIX® and confirm that this innovative new
medicine not only restores blood clotting factor to near normal
levels and significantly reduces factor use, but also that gene
therapy may reduce the burden of care and improve quality of life
for people living with 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 lead investigator of the HOPE-B study. “HOPE-B was also the
first and only phase 3 study to demonstrate efficacy of a gene
therapy for hemophilia B in individuals with circulating
neutralizing antibodies that have the potential to interfere with
the effects of treatment. Results from HOPE-B suggest that
HEMGENIX® may be effective in a broad range of hemophilia B
patients, regardless of prior exposure to common adeno-associated
viruses.”
“We at uniQure are incredibly pleased to have
the clinical results from the global HOPE-B pivotal trial featured
in such a prominent peer-reviewed journal,” said Ricardo Dolmetsch,
Ph.D., president of research and development at uniQure. “These
results highlight the potential benefits of this novel gene therapy
approach and further reinforce that those treated with HEMGENIX® in
clinical trials have achieved durable factor IX activity levels and
remained free of prophylactic factor IX replacement for years
following a single administration. uniQure is immensely proud to
have led the multi-year clinical development program for HEMGENIX®
that included the HOPE-B pivotal trial.”
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.
HEMGENIX® is a registered trademark of CSL
Behring.
About the Pivotal HOPE-B
Trial
The 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 treatment-related 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.
Long-term 24-month data presented at the 54th
American Society of Hematology (ASH) 2022 Annual Meeting and
Exposition and at The European Association for Haemophilia and
Allied Disorders (EAHAD) 2023 Annual Meeting continue to reinforce
the potential long-lasting efficacy and safety of HEMGENIX® and the
ongoing benefit of this treatment for people living with hemophilia
B.
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.
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 HEMGENIX® may be effective for people
living with hemophilia B. 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: |
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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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