uniQure Announces License Agreement with CSL Behring 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, Mass. and AMSTERDAM, June 24, 2020 (GLOBE NEWSWIRE) -- 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.