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uniQure Announces Sale of Royalty Interest in HEMGENIX for Up To $400 Million ~ uniQure to receive $375 million upfront cash payment ~ ~ Under the existing agreement with CSL Behring, uniQure retains the rights to future

Key Takeaway: uniQure N.V. has announced a definitive agreement to sell a portion of its royalty rights from CSL Behring related to HEMGENIX for a total of up to $400 million. The transaction provides uniQure with an upfront payment of $375 million and allows for additional milestone payments up to $25 million based on future sales performance. This deal is expected to extend uniQure's cash runway until the second quarter of 2026 and is seen as a validation of the therapeutic's commercial potential. UniQure will continue to retain rights to significant future royalties and milestone payments under its license agreement with CSL Behring.

Market Sentiment Analysis

POSITIVE FACTORS

  • uniQure secures $375 million upfront cash, enhancing capital position.
  • Maintains future milestone payments totalings up to $1.5 billion.
  • The agreement demonstrates confidence in the potential sales of HEMGENIX.

Full Press Release Details

Announces Sale of Royalty Interest in HEMGENIX
for Up To $400 Million
~ uniQure to receive $375 million upfront cash payment ~
Under the existing agreement with CSL Behring, uniQure retains the rights to future milestones totaling up to $1.5 billion and maintains
an interest in HEMGENIX royalties ~
Capped royalty financing provides uniQure non-equity-dilutive capital to advance its broad gene therapy pipeline, including AMT-130 for
Huntington's disease ~
and Amsterdam, the Netherlands, May 15, 2023 - uniQure N.V. (NASDAQ: QURE),
a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced it has entered
into a definitive agreement to sell a portion of the royalty rights due to uniQure from CSL Behring from the net sales of HEMGENIX
(etranacogene dezaparvovec-drbl) to HealthCare Royalty (HCRx) and Sagard Healthcare for a gross purchase price of up to
$400 million in cash.
financing provides uniQure with immediate, non-equity-dilutive capital to continue to invest in our AAV gene therapy pipeline and platform
and to advance potentially breakthrough gene therapies for patients, including AMT-130 in Huntington's disease, AMT-260 for refractory
temporal lobe epilepsy, AMT-162 for SOD-1 ALS and other product candidates," stated Matt Kapusta, chief executive officer of
uniQure. "The partial monetization of this royalty stream validates the significant potential of HEMGENIX global
sales while substantially reducing commercialization risk for uniQure. We are pleased to reach this agreement with HealthCare Royalty
and Sagard Healthcare, each of whom is a recognized leader in funding innovation across life sciences, and look forward to furthering
our opportunities for growth and value creation."
of the agreement, uniQure will receive an upfront cash payment of $375 million in exchange for the lowest royalty tier on CSL Behring's
worldwide net sales of HEMGENIX up to 1.85 times the purchase price until June 30, 2032 or, if such cap is not met by
June 30, 2032, up to 2.25x the purchase price through December 31, 2038.
eligible to receive an additional $25 million milestone payment if 2024 net sales of HEMGENIX exceed a pre-specified
threshold. uniQure will retain the rights to all other royalties under its existing Commercialization and License Agreement with CSL
Behring, as well as contractual milestones totaling up to $1.5 billion, including the $100 million milestone for the first U.S. product
sale and the $75 million milestone for the first product sale in one of five major European countries if achieved prior to July 2, 2023.
The transaction is expected to close no later than 15 business days from signing.
Chairman and Chief Executive Officer of HCRx, stated, "We believe that real innovation is the lifeblood of the biotechnology industry,
and it's a rare opportunity to be part of a true first, with HEMGENIX being the first gene therapy approved for
adults with hemophilia B. We are pleased to be able to provide uniQure with the opportunity to immediately monetize their royalty asset
to allow them to continue to develop their innovative gene therapy programs."
excited to complete this transaction and support uniQure with its mission to develop one-time, transformative gene therapies for patients
in need," said David MacNaughtan, Partner and Head of Sagard Healthcare. "Sagard's investment reflects our confidence
in the commercial prospects of HEMGENIX which is marketed by CSL Behring."
multi-year development of HEMGENIX and completed an exclusive global license and commercialization agreement with CSL
Behring in May 2021. HEMGENIX is approved for the treatment of hemophilia B in the U.S., Europe and United Kingdom. CSL
Behring also is pursuing registration in additional countries.
expects the transaction will extend its cash runway into the second quarter of 2026, assuming receipt of the contractual milestone payment
for the first sale of HEMGENIX in the U.S.
LLC acted as financial advisor to uniQure in this transaction and Ropes & Gray, LLP acted as legal advisor.
HCRx was represented in the transaction
by Sidley Austin, LP.
Sagard was represented in the transaction
by Mintz, Levin, Cohn, Ferris, Glovsky and Popeo, P.C.
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
HEMGENIX is a registered
trademark of CSL Behring.
uniQure is delivering
on the promise of gene therapy - single treatments with potentially curative results. The recent approvals 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
About HealthCare Royalty
royalty acquisition company focused on commercial or near-commercial stage biopharmaceutical products. HCRx has $6.3 billion in cumulative
capital commitments with offices in Stamford (CT), San Francisco, Boston and London. For more information, visit www.healthcareroyalty.com.
HEALTHCARE ROYALTY and HCRx are registered trademarks of HealthCare Royalty Management, LLC.
About Sagard Healthcare
is Sagard's biopharmaceutical royalties investment strategy, investing in royalties and credit backed by approved and commercial
biopharmaceutical products, diagnostics and medical devices. Sagard is a multi-strategy alternative investment asset management firm
with more than $13 billion under management and professionals located in Canada, the U.S. and Europe. We invest in venture capital, private
equity, private credit, real estate and royalties. We deliver flexible capital, an entrepreneurial culture, and a global network of investors,
commercial partners, advisors, and value creation experts. Our dynamic and supportive ecosystem gives our partners the advantage they
need to learn, grow and win at every stage. Sagard also engages in private wealth management and new venture creation.
uniQure Forward-Looking Statements
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
will be a commercial success. 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 regulatory approval and commercial launch of HEMGENIX ,
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 27, 2023 and its Quarterly Report on Form 10-Q filed May 9, 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

Frequently Asked Questions

What is uniQure's recent financial agreement about?

uniQure announced a sale of royalty rights for HEMGENIX, receiving up to $400 million.

How much upfront payment will uniQure receive?

uniQure will receive a $375 million upfront cash payment.

What rights does uniQure retain from the agreement?

uniQure retains rights to future milestones totaling up to $1.5 billion.

What therapies is uniQure planning to advance?

uniQure aims to advance gene therapies for Huntington's, epilepsy, and ALS.

What is HEMGENIX approved for?

HEMGENIX is approved for treating hemophilia B in various regions.

Last updated: May 15, 2023