uniQure N.V. (NASDAQ: QURE), a leading gene therapy company
advancing transformative therapies for patients with severe medical
needs, announced the achievement of a $100 million milestone
associated with the first commercial sale of HEMGENIX® in the
United States by its partner, global biotechnology leader CSL (ASX:
CSL). HEMGENIX® (etranacogene dezaparvovec) is a one-time
administered gene therapy 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.
“The first commercial sale in the U.S. is a
major milestone for uniQure as it marks the fulfillment of our
promise to deliver genetic medicines that have the potential to
transform people’s lives,” said Matt Kapusta, chief executive
officer of uniQure. “uniQure’s successful development of HEMGENIX®
further validates our AAV platform built on the back of 25 years of
scientific leadership and innovation in the field of gene therapy.
We look forward to our continued collaboration with CSL Behring as
they work to bring this important treatment to those living with
hemophilia B.”
uniQure conducted the multi-year research and
clinical development program for HEMGENIX®, which included three
clinical trials across 34 global sites and involved 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 facility in Lexington, Massachusetts.
Under the agreement with CSL Behring, the milestone payment is due
within 30 days after achievement of the milestone.
HEMGENIX® is the first approved gene therapy for
hemophilia B in the United States, European Union (EU) and European
Economic Area (EEA), and the UK.
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® has
been shown in clinical trials to allow people living with
hemophilia B to produce their own factor IX, which can lower the
risk of bleeding.
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.
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. The recent approval of our gene
therapy for hemophilia B – an historic achievement based on more
than a decade of research and clinical development – represents a
major milestone in the field of genomic medicine and ushers in a
new treatment approach for patients living with hemophilia. We are
now leveraging our modular and validated technology platform to
advance a pipeline of proprietary gene therapies for the treatment
of patients with Huntington's disease, refractory temporal lobe
epilepsy, ALS, Fabry disease, and other severe
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 28, 2023.
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: |
|
|
|
FOR MEDIA: |
|
|
|
|
|
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 |
uniQure NV (NASDAQ:QURE)
Graphique Historique de l'Action
De Mai 2024 à Juin 2024
uniQure NV (NASDAQ:QURE)
Graphique Historique de l'Action
De Juin 2023 à Juin 2024