uniQure N.V. (NASDAQ: QURE), a leading gene therapy company
advancing transformative therapies for patients with severe medical
needs, today announced that uniQure and CSL Behring have entered
into a licensing agreement providing CSL Behring with exclusive
global rights to etranacogene dezaparvovec, uniQure’s
investigational gene therapy for patients with hemophilia B.
Etranacogene dezaparvovec consists of an AAV5 viral vector carrying
a gene cassette with the patent-protected Padua variant of Factor
IX (FIX-Padua). Under the terms of the agreement, uniQure will
receive a $450 million upfront cash payment and be eligible to
receive up to $1.6 billion in payments based on regulatory and
commercial milestones. uniQure will also be eligible to receive
tiered double-digit royalties in a range of up to a low-twenties
percentage of net product sales arising from the collaboration.
The collaboration leverages CSL Behring’s strong
global reach and commercial infrastructure in hematology to
accelerate access of etranacogene dezaparvovec to hemophilia B
patients around the world.
“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 globally 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, and to
invest further in our leading gene therapy manufacturing and
technology platform to support pipeline growth,” he added.
As a CSL Limited (ASX:CSL;USOTC:CSLLY) company,
CSL Behring is a global biotherapeutics leader delivering
lifesaving medicines to patients with rare and serious diseases. A
global leader in treating bleeding disorders, CSL Behring has been
delivering innovations for the hemophilia patient community for
more than 30 years. The company reported more than $1 billion in
sales of hemophilia-related medicines in 2019.
“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. Upon approval, we believe 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.”
Under the terms of the agreement, uniQure will
be responsible for the completion of the HOPE-B pivotal study,
manufacturing process validation, and the manufacturing supply of
etranacogene dezaparvovec until such time that these capabilities
are transferred to CSL Behring. Clinical development and regulatory
activities performed by uniQure under the agreement will be
reimbursed by CSL Behring. CSL Behring will be responsible for
regulatory submissions and commercialization of etranacogene
dezaparvovec.
The closing of the transaction is contingent on
completion of review under antitrust laws in the United States,
Australia and the United Kingdom.
Accelerate Build-out of Innovative Gene Therapy
Pipeline and Platform
uniQure expects that the agreement will provide
additional capital to significantly accelerate and expand its
pipeline of innovative gene therapies, including advancing the
Phase I/II study of AMT-130 in Huntington’s disease, initiating
IND-enabling studies of AMT-150 in spinocerebellar ataxia type 3,
selecting a lead candidate in Fabry disease and progressing other
current and new candidates for central nervous system disorders and
rare liver-directed diseases. Regarding AMT-130, uniQure recently
announced the successful completion of the first two patient
procedures in the Phase I/II study and anticipates announcing early
safety data in the second half of 2020 and initial efficacy data in
2021.
uniQure plans to continue to leverage its
leading gene therapy platform, including the Company’s deep
expertise with AAV5, to develop potentially best-in-class gene
therapies. AAV5-based gene therapies have been demonstrated to be
safe and well tolerated in a multitude of clinical trials,
including uniQure trials conducted in hemophilia B and other
indications. No patient treated in clinical trials with uniQure’s
AAV5 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 viable
treatments in patients with pre-existing antibodies to AAV5,
thereby potentially increasing patient eligibility for
treatment. uniQure also may seek to in-license or acquire
additional product candidates that align with its research and
development strategy.
In addition, uniQure plans to further strengthen
its proprietary gene therapy platform by expanding its
manufacturing capacity to support a broad pipeline, including
product candidates for diseases with larger prevalence, as well as
investing further in new technologies to improve the efficacy,
safety and applicability of its gene therapies to patients.
As part of uniQure’s effort to focus on those
gene therapy programs that have the greatest potential to improve
patients’ lives and generate long-term value for shareholders,
uniQure plans to de-prioritize its research program of AMT-180 for
patients with hemophilia A.
Moelis & Company acted as a financial
advisor to uniQure in this transaction.
Conference Call Today at 5:30 p.m.
EDT
uniQure will host a conference call today, June
24, 2020, at 5:30 p.m. Eastern Daylight Time. The conference call
may be accessed by dialing (877) 870-9135 for domestic callers and
+44 020 719 283 38 for international callers. The passcode for the
call is 9499239. Please specify to the operator that you would like
to join the "uniQure Conference Call." The conference call will be
webcast live under the investor relations section of uniQure’s
website at www.uniQure.com and will be archived there following the
call for 90 days.
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, Fabry disease,
spinocerebellar ataxia Type 3 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, whether the parties
will successfully complete the review under applicable antitrust
laws or otherwise close the transaction, whether uniQure will
receive the upfront cash payment or any of the financial benefits
of the agreement; whether the collaboration will benefit Hemophilia
B patients worldwide, whether the parties to the agreement will
establish a new standard of care for patients with hemophilia B,
whether uniQure will be able to accelerate or expand its pipeline
of innovative gene therapies or its technology platform, including
advancing the Phase I/II study of AMT-130 in Huntington’s disease,
initiating IND-enabling studies of AMT-150 in spinocerebellar
ataxia type 3, selecting a lead product candidate for Fabry
disease, or progressing current or additional candidates for
central nervous system disorders and other genetic diseases,
whether uniQure will announce early safety data from its Phase I/II
study of AMT-130 in the second half of 2020 and initial efficacy
data in 2021 or ever, whether uniQure will develop best-in-class
gene therapies, whether uniQure will in-license or acquire
additional product candidates, whether uniQure will expand its
manufacturing capacity to support a broad pipeline, such as product
candidates for diseases with larger prevalence, and whether uniQure
will obtain enabling technologies that improve the efficacy or
safety of its gene therapies. uniQure’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 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
uniQure’s Annual Report on Form 10-Q filed on April 29, 2020. Given
these risks, uncertainties and other factors, you should not place
undue reliance on these forward-looking statements, and uniQure
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 Direct: 339-970-7536
Mobile: 617-680-9452m.cantor@uniQure.com |
Chiara Russo Direct: 617-306-9137
Mobile: 617-306-9137 c.russo@uniQure.com |
Tom
MaloneDirect: 339-970-7558Mobile: 339-223-8541
t.malone@uniQure.com |
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