KING OF PRUSSIA, Pa.,
June 24, 2020 /PRNewswire/
-- Global biotherapeutics leader CSL Behring announced today
that it has agreed to acquire exclusive global license rights to
commercialize an adeno-associated virus (AAV) gene therapy
program, AMT-061 (etranacogene dezaparvovec), for the treatment of
hemophilia B from uniQure (NASDAQ: QURE), a leading gene therapy
company. The AMT-061 program, currently in Phase 3 clinical trials,
could be one of the first gene therapies to provide potentially
long-term benefits to patients with hemophilia B.
One dose of AMT-061 has shown to increase Factor IX (FIX) plasma
levels – the blood clotting protein lacking in people with
hemophilia B – to a degree that reduces or eliminates the tendency
for bleeding for many years. Should AMT-061 be successful,
appropriate candidate hemophilia B patients would be able to have a
one-time treatment to restore FIX activity to functional levels
capable of eliminating the need for frequent and ongoing
replacement therapies.
"Our vision with hemophilia B patients is to offer
transformational treatment paradigms that help free them from the
lifelong burden of this disease," said CSL's CEO and Managing
Director Paul Perreault. "With more
than three decades of providing lifesaving innovations for the
global bleeding disorders community, we are well positioned to
maximize the potential benefit of this therapy."
Under the agreement with uniQure, upon closing the transaction
CSL Behring will have the exclusive global right to commercialize
AMT-061. uniQure will receive an upfront cash payment of
US$450 million followed by regulatory
and commercial sales milestone payments and royalties. Under the
terms of the agreement, uniQure will complete the Phase 3 trial and
scale up manufacture for early commercial supply under an agreed
plan with CSL Behring. The transaction is subject to
customary regulatory clearances before closing.
"We are thrilled to enter into this commercialization and
license agreement with CSL Behring, an ideal commercial partner
with global reach and decades of expertise in hemophilia," stated
Matt Kapusta, chief executive
officer of uniQure. "We believe that through this arrangement, we
are ideally positioned to deliver our innovative gene therapy to
the largest number of hemophilia B patients as quickly as possible.
The transaction represents a major milestone in the development of
etranacogene dezaparvovec and, when closed, we expect that it will
provide uniQure with significant financial resources to advance and
expand our pipeline of gene therapy candidates, anchored by AMT-130
in Huntington's disease, as well as to invest further in our
leading manufacturing and technology platform."
In December 2019, uniQure
announced that data from its Phase 2b
dose-confirmation study of AMT-061 showed that all patients
stabilized and sustained FIX activity at functionally high levels
one year after a single dose – with increases in FIX activity of up
to 50% of normal and a mean of 41%. This exceeds the levels
considered sufficient to eliminate or significantly reduce the risk
of bleeding events.
According to CSL Behring's Executive Vice President, Head of
Research and Development, and Chief Medical Officer Bill
Mezzanotte, "We are exceedingly encouraged by the data we've seen
on AMT-061. Not only has the treatment option demonstrated robust
clinically meaningful responses in FIX activity, but it has also
exhibited excellent safety over multiple years of observation.
Expanding our gene therapy portfolio to treat hemophilia B, a
disease state well known to CSL Behring, exemplifies how we are
strategically aligning our rare and serious disease focus and our
targeted therapeutic area focus with our core scientific platforms
to transform the lives of patients."
This acquisition will also enhance CSL Behring's capabilities in
its growing gene therapy portfolio. The company is currently
developing a stem cell gene therapy (CSL200) for the treatment of
sickle cell disease and has recently established an alliance with
Seattle Children's Research Institute to develop a stem cell gene
therapy for primary immunodeficiency diseases -- another rare
disease area where CSL Behring has leading capabilities.
Perreault added, "Upon approval, this next-generation therapy
will be highly complementary to our existing best-in-class
hemophilia B product portfolio with an alternate best-in-class
treatment option. With the license to AMT-061, we are building on
our legacy of delivering lifesaving innovations in hematology
where, today, we offer a market leading product for hemophilia B
and we are a leader in therapies for treating hemophilia A, von
Willebrand disease, thrombosis, and other
life-threatening conditions."
CSL Behring has put patients first by addressing the world's
most serious, complicated and rare diseases for over 100 years. The
company is now bringing that same commitment to gene therapy; its
mission is to address unmet patient needs and enable patients to
get the very most out of life.
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 uniQure'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 CSL Behring
CSL Behring is a global biotherapeutics leader driven by
its promise to save lives. Focused on serving patients' needs by
using the latest technologies, we develop and deliver innovative
therapies that are used to treat coagulation disorders, primary
immune deficiencies, hereditary angioedema, respiratory disease,
and neurological disorders. The company's products are also used in
cardiac surgery, burn treatment and to prevent hemolytic disease of
the newborn.
CSL Behring operates one of the world's largest plasma
collection networks, CSL Plasma. The parent company, CSL Limited
(ASX:CSL;USOTC:CSLLY), headquartered in Melbourne, Australia, employs more than 26,000
people, and delivers its life-saving therapies to people in
more than 100 countries. For inspiring stories about the promise of
biotechnology, visit Vita CSLBehring.com/vita and follow us on
Twitter.com/CSLBehring.
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SOURCE CSL Behring