uniQure, CSL Behring Collaborate on Hemophilia B Gene Therapy in Over $1.6-Billion Deal

June 29, 2020
BioPharm International Editors

The companies are entering into a license agreement to provide CSL Behring with exclusive global rights to etranacogene dezaparvovec, uniQure’s investigational gene therapy for patients with hemophilia B.

uniQure and CSL Behring announced on June 24, 2020 that they are entering into a license agreement to provide CSL Behring with exclusive global rights to etranacogene dezaparvovec, uniQure’s investigational gene therapy for patients with hemophilia B, in a deal worth potentially over $1.6 billion.

The agreement includes a $450-million upfront cash payment to uniQure, which will also be  qualified to receive up to $1.6 billion in payments based on regulatory and commercial milestones, according to a uniQure press release. uniQure will also obtain tiered double-digit royalties of up to a low-twenties percentage of net product sales from the partnership.

“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,” said Matt Kapusta, CEO of uniQure, in the press release. “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.” 

“Our vision with hemophilia B patients is to offer transformational treatment paradigms that help free them from the lifelong burden of this disease,” added CSL’s CEO and managing director, Paul Perreault, in the press release. “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.”

Source: uniQure