The CSL Limited (ASX: CSL) share price will be on watch on Thursday after a promising announcement out of the biotherapeutics company.
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What did CSL announce?
Hot on the heels of its decision to acquire clinical-stage biotechnology company Vitaeris earlier this month, this morning the company announced plans to make another acquisition.
CSL has agreed to acquire the exclusive global license rights to commercialise an adenoassociated virus (AAV) gene therapy program, AMT-061 (etranacogene dezaparvovec), for the treatment of haemophilia from Nasdaq-listed gene therapy company, uniQure .
According to the release, the AMT-061 program, which is 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.
Management explained that one dose of AMT-061 has shown to increase Factor IX (FIX) plasma levels to a degree that reduces or eliminates the tendency for bleeding for many years. FIX is the blood clotting protein lacking in people with haemophilia B.
This means that should AMT-061's trials be successful, appropriate candidate haemophilia B patients will 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.
CSL's CEO and Managing Director, Paul Perreault, commented: "Our vision for haemophilia B patients is to offer transformational treatment paradigms that help free them from the lifelong burden of this disease. 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."
What is CSL paying for AMT-061?
Under the agreement with the gene therapy company, CSL will have the exclusive global right to commercialise AMT-061.
It will pay uniQure an upfront cash payment of US$450 million, followed by regulatory and commercial sales milestone payments and royalties.
In addition, uniQure will complete the Phase 3 trial and scale up manufacture for early commercial supply under an agreed plan with CSL. The transaction remains subject to customary regulatory clearances before closing.
Mr. Perreault concluded: "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 hematology."
