CSL acquires global rights to haemophilia gene therapy

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CSL Behring has acquired global rights to an adeno-associated virus (AAV) gene therapy program, AMT-061 (etranacogene dezaparvovec), for the treatment of haemophilia B from uniQure.

The therapy is currently in phase three trials. CSL said it has been 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.

In a phase two trial, all patients administered AMT-061 recorded stabilised and sustained FIX activity at functionally high levels one year after a single dose – with increases in FIX activity of up to 50 per cent of normal and a mean of 41 per cent.

"This exceeds the levels considered sufficient to eliminate or significantly reduce the risk of bleeding events," said the company in a statement.

If approved, AMT-061 would be delivered to patients as 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 the agreement, uniQure will receive an upfront cash payment of US$450 million followed by regulatory and commercial sales milestone payments and royalties. uniQure will complete the phase three trial and scale-up manufacture for early commercial supply under an agreed plan with CSL Behring.

“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.

“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.

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.”

According to CSL Behring’s executive vice president and head of Research and Development Bill Mezzanotte, “We are exceedingly encouraged by the data we’ve seen on AMT-061. Not only has the treatment option demonstrated robust clinically meaningful responses in FIX activity, but it has also exhibited excellent safety over multiple years of observation.

"Expanding our gene therapy portfolio to treat hemophilia B, a disease state well known to CSL Behring, exemplifies how we are strategically aligning our rare and serious disease focus and our targeted therapeutic area focus with our core scientific platforms to transform the lives of patients.”

The company said the acquisition will also enhance CSL Behring’s capabilities in its growing gene therapy portfolio.

It is currently developing a stem cell gene therapy (CSL200) for the treatment of sickle cell disease and has recently established an alliance with Seattle Children’s Research Institute to develop a stem cell gene therapy for primary immunodeficiency diseases.

Mr Perreault added, “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.”