CSL Ltd Annual Report 2020

US$922 million in research and development * CSL continues to innovate, expand and diversify our R&D pipeline. We now have development products in all of our therapeutic areas – immunology, haematology, cardiovascular and metabolic, respiratory, transplant, and influenza – across all of our technology platforms – plasma fractionation, recombinant protein technology, cell and gene therapy, and vaccine development. Global collaborations for innovation access In order to further increase global collaboration and introduce external early stage projects to support growth of our research pipeline, the CSL Research External Innovation (REI) team in Europe entered into a partnership with Biopôle in Switzerland in June 2020. Biopôle is a life-sciences campus boasting a renowned community of industry and academic specialists, as well as state-of-the-art laboratories. The Biopôle community consists of leading companies, prestigious research institutes, clinical research departments and dozens of other research entities. The partnership will enhance networking with the scientific community and provide CSL with access to R&D opportunities on specific research areas organised by Biopôle and its academic partners. In June 2020, a strategic alliance was announced with the Seattle Children’s Research Institute (SCRI) – one of the top paediatric research institutions in the world – to develop stem cell gene therapies for primary immunodeficiency (PI) diseases. Initially, the alliance will focus on the development of treatment options for patients with two rare, life-threatening PI diseases: Wiskott-Aldrich syndrome and X-linked agammaglobulinemia. Expanding our gene therapy portfolio into an area of immunology well known to CSL exemplifies how we are strategically growing our capabilities in this scientific platform and collaborating with world-class institutions to access innovation with the potential to vastly improve patients’ lives. In support of the yearly seasonal influenza vaccine epidemic, Seqirus collaborates with the World Health Organization Coordinating Centre in Melbourne, Australia to prepare vaccine seeds and potency reagents that are made widely available. This is an important contribution to assist with the global effort to prepare for the forthcoming vaccination season. Strategic acquisitions to expand our therapeutic areas In June 2020, CSL entered an agreement with uniQure, a leading gene therapy company, to acquire exclusive global licence rights to commercialise an adeno-associated virus (AAV) gene therapy program, AMT-061 (etranacogene dezaparvovec; EtranaDez), for the treatment of haemophilia B. AMT-061 is currently in Phase III clinical trials and could be one of the first gene therapies to provide long-term benefits to patients with haemophilia B. A single dose of AMT-061 has been 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. If AMT-061 is successful, appropriate candidate haemophilia B patients would be able to receive a single dose to restore FIX activity to functional levels capable of eliminating the need for frequent and ongoing replacement therapies. Expanding our gene therapy portfolio to treat haemophilia B, a disease state well known to CSL, 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 acquisition of Vitaeris Inc. in June 2020 expanded our transplant therapeutic area portfolio with the addition of clazakizumab, an anti-interleukin-6 (IL-6) monoclonal antibody (mAb) currently in Phase III clinical trials for the potential treatment of chronic active antibody-mediated rejection (AMR), the leading cause of long-term rejection in kidney transplant recipients. With this acquisition, clazakizumab joins our portfolio of products in late-stage development to address significant unmet needs in the transplant community. There are currently no approved treatments for transplant recipients who develop AMR. CSL’s core therapeutic area focus also means we will choose not to develop certain internal assets that are outside these areas; instead, we will identify suitable partners and out- license assets that have promising therapeutic attributes. A recent example is our monoclonal antibody program targeting interleukin-11 receptor (IL-11R). Lassen Therapeutics acquired the IL-11R antibodies from CSL in June 2020 and will develop LASN01 in the areas of fibrosis and oncology. Accessing IL-11 research and antibodies from CSL will greatly accelerate Lassen’s efforts to bring a novel therapeutic candidate to patients. Strategic support for innovative medical research In order to accelerate the commercialisation of promising biomedical research, CSL has committed A$45 million over 10 years to the Brandon Capital–led A$230 million Biomedical Translation Fund (BTF) and the two A$200 million Medical Research Commercialisation Funds (MRCF3 and MRCF5). These funds – the largest life-science funds in Australia’s history – have continued to invest in a range of promising biomedical discoveries. Through the ongoing partnership between CSL Behring and the University City Science Center in Philadelphia, US, researchers at the University of Pittsburgh and the University of Delaware were awarded funding and support in February 2020 to accelerate their search for innovative newmedicines. The first recipients of this support from the CSL Research External Innovation initiative will investigate a potential targeted therapeutic for the treatment of pulmonary fibrosis and the use of cell-derived microparticles and vesicles for both the treatment of thrombocytopenia and as a vehicle for the delivery of gene-based therapies into blood stem cells. Following the success of the initial pilot, the CSL Science Center Research Acceleration Initiative has expanded and is accepting applications from researchers at 28 institutions across six states with awardees to receive up to US$400,000 each. Influenza remains one of our greatest global health threats. CSL is committed to collaborating with like-minded partners to advance understanding of the human response to influenza and to discover new and innovative vaccine solutions. We have continued our support of an international, non-profit venture, the Human Vaccines Project, dedicated to decoding the immune system to develop a universal influenza vaccine that affords long-lasting protection against seasonal and pandemic influenza across demographics and geography. The project unites leading academic research * Limited assurance by Ernst & Young. CSL Limited Annual Report 2020 24 Powered by Innovation 7