RARE Daily

UniQure Grants CSL Behring Global Rights to Hemophilia B Gene Therapy Deal Worth up to $2B

June 25, 2020

In a licensing agreement potentially worth up to more than $2 billion, Uniqure has granted CSL Behring exclusive global rights to commercialize its adeno-associated virus (AAV) gene therapy program for the treatment of hemophilia B.

For CSL, already a leading producer of plasma-based therapies for the treatment of blood disorders, the deal marks a major move into gene therapy. The company is currently developing a stem cell gene therapy for the treatment of sickle cell disease, and recently partnered with the Seattle Children’s Research Institute to develop a stem cell gene therapy for primary immunodeficiency diseases.  

At the same time that the deal was announced, UniQure said it would be putting its experimental gene therapy for the treatment of hemophilia A on hold, and would put its resources into advancing its gene therapy program in Huntington’s disease, as well as moving forward experimental candidates for the treatment of rare central nervous system disorders and rare liver-directed diseases.

The program, AMT-061 (etranacogene dezaparvovec) is currently in a phase 3 clinical trials, and could be one of the first gene therapies to provide potentially long-term benefits to patients with hemophilia B.

Hemophilia B is a rare bleeding disorder caused by mutations in the F9 gene resulting in insufficient levels of the blood clotting protein factor IX. Symptoms can range from mild to severe depending on the level of factor IX in the blood, and can range from prolonged bleeding after injury to frequent spontaneous bleeds into the muscles and joints and organs in severe cases that cause pain and other problems. Bleeding in the brain can lead to death if untreated.

One dose of AMT-061 has been shown to increase factor IX (FIX) plasma levels to a degree that reduces or eliminates the tendency for bleeding for many years. Data from the phase 2b dose-confirmation study of AMT-061 showed that all patients stabilized and sustained FIX activity at functionally high levels one year after a single dose – with increases in FIX activity of up to 50 percent of normal and a mean of 41percent. This exceeds the levels considered sufficient to eliminate or significantly reduce the risk of bleeding events. Should AMT-061 be successful, appropriate candidate hemophilia B patients would be able to have a one-time treatment to restore FIX activity to functional levels capable of eliminating the need for frequent and ongoing replacement therapies.

“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.”

Under their agreement, UniQure will receive an upfront cash payment of $450 million followed by regulatory and commercial sales milestone payments and royalties valued at up to $1.6 billion. UniQure will complete the phase 3 trial and scale up manufacture for early commercial supply under an agreed plan with CSL Behring.

“We believe that through this arrangement, we are ideally positioned to deliver our innovative gene therapy to the largest number of hemophilia B patients as quickly as possible,” said Matt Kapusta, CEO of UniQure.

Photo: Matt Kapusta, CEO of UniQure



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