CSL Ltd Annual Report 2021

7 Powered by Innovation CSL Limited Annual Report 2020/21 26 Strategic acquisitions to expand our therapeutic areas EtranaDez – a novel therapy for a rare disease CSL’s focus remains on extending and improving the lives of patients with rare and serious diseases. Our R&D and in-market product portfolios have advanced and changed considerably over the past few years and look very different from the last decade. We continue to search for new and exciting opportunities that allow us to address previously unmet patient needs and improve the quality of patients’ lives. Through the years, we have kept our promise to patients with haemophilia B to be leaders of innovation to ease the burden of treatment and ultimately help patients realise a life full of potential. Haemophilia B is a rare life-threatening degenerative disease that results from the congenital absence or deficiency of normally functioning blood clotting factor IX protein, which prevents excessive bleeding. The deficiency of factor IX activity leaves people with haemophilia B particularly vulnerable to bleeds in their muscles, internal organs and joints, leading to pain, swelling and joint damage. Current treatment includes life-long prophylactic infusions of factor IX to temporarily replace or supplement low levels of the blood-clotting factor and missing an infusion may increase their likelihood of a life-threatening bleed or even premature death. For decades, CSL Behring’s plasma-derived clotting factor products have offered haemophilia patients effective therapy to achieve haemostasis. The launch of IDELVION ® in 2016 advanced the company’s commitment to the haemophilia B community by the generation of a bioengineered extended-activity factor IX product for prophylactic infusion that achieves a zero median annualised spontaneous and joint bleeding rate. The initiation of prophylactic factor replacement from early childhood to prevent musculoskeletal bleeding has been shown to be essential to maintaining normal joint function into adulthood. Today, IDELVION ® is the number one, globally prescribed factor IX product for the prophylactic treatment of haemophilia B. ‘Haemophilia B patients live with the knowledge that they are at constant risk of bleeds, and that every bleed can mean that tissue or joints are irreparably damaged. Imagine what it might mean to be freed from that fear, secure in the knowledge that your self-generated factor IX levels will be high enough to protect you today, tomorrow and every day, ideally for years to come. This is the essence of great science bringing hope to patients.’ Dr WilliamMezzanotte Executive Vice President, Head of Research & Development & Chief Medical Officer In May 2021, CSL closed its Commercialization and License Agreement with uniQure, a leading gene therapy company, for EtranaDez (etranacogene dezaparvovec; formerly AMT-061). EtranaDez is an adeno-associated virus vector serotype 5-based (AAV5) gene therapy for adult patients with haemophilia B. The vector is engineered to direct the recipient’s liver cells to produce and release a variant of naturally occurring factor IX, designated FIX-Padua, into the bloodstream. EtranaDez is currently in Phase III clinical trials and has been shown to result in functional levels of factor IX. If approved, EtranaDez has the potential to be the first-ever gene therapy for haemophilia and will deliver on CSL’s ongoing promise to improving the lives of those living with haemophilia B. Expanding CSL’s expertise in gene therapy demonstrates the company’s commitment to innovation, expanding beyond the traditional plasma-derived and recombinant protein therapies consistent with our long-term strategy. The parallel development of both ex vivo and in vivo approaches to the correction of inherited disease provides our R&D clinicians, scientists and researchers an extensive toolkit to be able to match the optimal technology to specific disease challenges. EtranaDez has the potential to be life-changing, offering people with haemophilia B years of functional factor IX levels generated by their own bodies. The acquisition and clinical development of EtranaDez builds on our commitment to complement the company’s existing factor IX assets and provides an exciting opportunity to profoundly transform the treatment of haemophilia B.