CSL Behring Bolsters Gene Therapy Pipeline with uniQure's Hemophilia B Treatment

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CSL Behring bolstered its growing gene therapy portfolio with the acquisition of global rights to uniQure’s gene therapy program for hemophilia B that has a price tag of up to $2 billion.

CSL Behring is banking on the potential for the adeno-associated virus (AAV) gene therapy program, AMT-061 (etranacogene dezaparvovec), becoming one of the first-to-market treatments that could provide long-term benefits for the bleeding disorder following one dose. The AMT-061 program is currently in Phase III clinical studies. In previous studies, one dose of AMT-061 has shown to increase Factor IX (FIX) plasma levels – the blood clotting protein lacking in people with hemophilia B – to a degree that reduces or eliminates the tendency for bleeding for many years.

CSL Chief Executive Officer Paul Perreault said the company’s vision for hemophilia B patients is to provide them with “transformational treatment paradigms” that can change the way their disease is treated for a lifetime. 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. Perreault said CSL Behring has decades of experience working with bleeding disorders and the global community, which puts the company in a position to “maximize the potential benefit of this therapy.”

In December, uniQure said data from its Phase IIb 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% of normal and a mean of 41%. This exceeds the levels considered sufficient to eliminate or significantly reduce the risk of bleeding events.

uniQure CEO Matt Kapusta said the arrangement with CSL will deliver the gene therapy to the largest number of 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, as well as to invest further in our leading manufacturing and technology platform,” Kapusta said in a statement.

uniQure investors were not as excited about the deal as Kapusta. Share prices were down more than 16% in premarket trading.

CSL Behring’s Chief Medical Officer Bill Mezzanotte said he was excited about the data he has seen regarding AMT-061. He said it has demonstrated robust clinically meaningful responses in FIX activity and also has a strong safety profile.

“Upon approval, 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. With the license to AMT-061, we are building on our legacy of delivering lifesaving innovations in hematology where, today, we offer a market leading product for hemophilia B and we are a leader in therapies for treating hemophilia A, von Willebrand disease, thrombosis, and other life-threatening conditions,” Perrault added in a statement.

Under terms of the deal, CSL Behring will pay uniQure $450 million in upfront money, as well as up to $1.6 billion in regulatory and commercial sales milestone payments and royalties. uniQure will complete the Phase 3 trial and scale up manufacture for early commercial supply under an agreed plan with CSL Behring.

For CSL Behring, the acquisition will enhance its gene therapy portfolio. The company currently developing a stem cell gene therapy for the treatment of sickle cell disease, as well as primary immunodeficiency diseases.

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