Full Press Release Details
uniQure Announces License Agreement
to Commercialize Hemophilia B Gene Therapy
~ CSL Behring Obtains Exclusive Global
Rights to Develop and Commercialize
uniQure's Differentiated Gene Therapy
Candidate for Hemophilia B ~
~ uniQure Eligible to Receive More Than
$2 Billion, Including $450 Million in Upfront Cash, $1.6 Billion in Milestone Payments, and Double-Digit Royalties Ranging
Up to a Low-Twenties Percentage of Net Sales ~
~ Agreement Leverages CSL Behring's
Global Hematology Capabilities and Infrastructure
to Benefit Hemophilia B Patients Worldwide
~ Transaction Expected to Enable uniQure
to Strategically Expand and Accelerate Pipeline and Platform ~
~ uniQure to Host Conference Call Today,
June 24, 2020, at 5:30 p.m. EDT ~
Lexington, MA and Amsterdam, the Netherlands,
June 24, 2020 - 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 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
"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,"
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
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
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