CSL Limited (ASX:CSL) has agreed to acquire exclusive global license rights to commercialise an adeno- associated virus (AAV) gene therapy program, AMT-061, for the treatment of haemophilia B from uniQure.
CSL’s CEO and Managing Director Paul Perreault, said the AMT-061 programme, currently in Phase 3 clinical trials, could be one of the first gene therapies to provide potentially long-term benefits to patients with haemophilia B.
One dose of AMT-061 has shown to increase Factor IX (FIX) plasma levels – the blood clotting protein lacking in people with haemophilia B – to a degree that reduces or eliminates the tendency for bleeding for many years.
Mr Perreault said that should AMT-061 be successful, appropriate candidate haemophilia 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 for haemophilia B patients is to offer transformational treatment paradigms that help free them from the lifelong burden of this disease,” said Mr Perreault.
“With more than three decades of providing lifesaving innovations for the global bleeding disorders community, we are well positioned to maximise the potential benefit of this therapy.”
Under the agreement with uniQure, upon closing the transaction CSL will have the exclusive global right to commercialise AMT-061.
uniQure, a leading gene therapy company, will receive an upfront cash payment of US$450 million followed by regulatory and commercial sales milestone payments and royalties.
Under the terms of the agreement, uniQure will complete the Phase 3 trial and scale up manufacture for early commercial supply under an agreed plan with CSL.
“Upon approval, we believe this next-generation therapy would be highly complementary to our existing haemophilia B product portfolio. We hope that it provides patients with an alternate best-in-class treatment option, building on our legacy of delivering lifesaving innovations in haematology,” Mr Perreault said.
“This acquisition will also enhance CSL’s capabilities in its growing gene therapy portfolio. The company 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.”