CSL Limited Annual Report 2022/23 12 CSL’s Product Portfolio and Therapeutic Areas Therapeutic Areas Platform Plasma Protein Technology Immunology Haematology Respiratory Cardiovascular and metabolic Vaccines Transplant Recombinant Technology Cell and Gene Therapy Cell-based Adjuvant mRNA Egg-based CSL Behring CSL Behring discovers, develops and delivers the broadest range of products in the industry for treating rare and serious diseases such as haemophilia, von Willebrand disease (vWD), primary and secondary immune deficiencies (PID/SID), chronic inflammatory demyelinating polyneuropathy (CIDP), hereditary angioedema (HAE) and inherited respiratory disease. CSL Behring’s products are also used in cardiac surgery, for burns treatment and for urgent warfarin reversal. We strive to meet patients’ needs using the latest recombinant and plasma-derived technologies as well as gene therapy approaches. Immunology CSL’s world leading immunoglobulin franchise is the cornerstone of the immunology therapeutic area and is focused on developing and delivering trusted products and technologies to serve patients with a range of serious immunologic and neurologic diseases, including primary and secondary immunodeficiencies (PID/ SID), chronic inflammatory demyelinating polyneuropathy (CIDP) and hereditary angioedema (HAE). Key CSL Behring products on the market include PRIVIGEN®, HIZENTRA®, BERINERT®, HAEGARDA® and a range of hyperimmunes. Building on CSL’s long history of providing patients with immunoglobulin products, it continues to optimise the patient experience by developing more convenient and flexible ways to dose and administer existing immunoglobulin products. Key recombinant assets are also progressing in early development to treat underserved immune-mediated diseases. CSL continues to build on its strong 40-year legacy in HAE, working to expand on current medicines to provide optimal treatments for the full range of HAE patients. Garadacimab, CSL’s first-in-class monoclonal antibody targeting activated Factor XII (FXIIa), is being developed as a prospective long-term prophylactic treatment for patients with HAE. Haematology CSL remains focused on easing the burden of disease and improving the lives of patients with rare bleeding disorders. Major advances have been made in haemophilia A and B in recent years with the launch of novel recombinant coagulation factor medicines and through the acquisition of exclusive global licence rights to commercialise HEMGENIX® (etranacogene dezaparvovec), an AAV5 (adeno-associated virus) gene therapy for the treatment of haemophilia B, which has been approved in the United States, Europe and the UK. Other key CSL Behring products on the market include IDELVION®, AFSTYLA®, HUMATE P®/HAEMATE®, BERIPLEX®/KCENTRA®, RIASTAP®/HEMOCOMPLETTAN®, VONCENTO®/BIOSTATE® and albumin. Additionally, exciting R&D efforts are underway to explore new indications in benign haematology as well as novel therapeutics in haemostasis and thrombosis. This includes initiating an important global Phase III study to evaluate the early administration of KCENTRA® (4-factor prothrombin complex concentrate) on survival in trauma patients suffering life-threatening bleeding, and a Phase II study under a licensing agreement with Translational Sciences using CSL301 (α2 anti-plasmin), a first-in-class, chimeric monoclonal antibody as thrombolytic treatment in adults with acute sub-massive pulmonary embolism. Respiratory Respiratory diseases impose an enormous burden on patients and society and are a leading cause of death and disability worldwide. In addition to CSL’s existing product, ZEMAIRA®/RESPREEZA® for patients with alpha-1 antitrypsin deficiency, CSL is investigating potential new clinical treatments for respiratory diseases using novel recombinant monoclonal antibodies and plasma-derived therapies to address this need. Trabikibart, an anti-beta common monoclonal antibody, is being investigated for the treatment of severe uncontrolled asthma and severe chronic obstructive pulmonary disease (COPD). In idiopathic pulmonary fibrosis (IPF), a severe debilitating disease, a clinical development program has started with garadacimab, the first of CSL’s compounds being explored in this disease area. CSL787, a plasma-derived, inhaled immunoglobulin is being investigated for patients with bronchiectasis. 2 Our Company
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