CSL Behring announced that it has agreed to acquire exclusive global license rights to commercialize an adeno-associated virus (AAV) gene therapy program, AMT 061 (etranacogene dezaparvovec), for the treatment of hemophilia B from uniQure , a leading gene therapy company. The AMT-061 program, currently in Phase III clinical trials, could be one of the first gene therapies to provide potentially long-term benefits to patients with hemophilia B.
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. 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.
Under the agreement with uniQure, upon closing the transaction CSL Behring will have the exclusive global right to commercialize AMT 061. uniQure 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 III trial and scale up manufacture for early commercial supply under an agreed plan with CSL Behring. The transaction is subject to customary regulatory clearances before closing.