Therapeutic Areas and Product Portfolio Therapeutic Areas Immunology CSL’s world leading immunoglobulin and plasma protein products are the cornerstone of the immunology therapeutic area, which 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). Building on CSL’s long history of providing patients with immunoglobulin products, CSL continues to optimise the patient experience by developing more convenient and flexible ways to dose and administer existing immunoglobulin products, including offering the only approved 20% subcutaneous immunoglobulin therapy for CIDP (HIZENTRA®) and the only pre-filled syringe options for patient self-administration. 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, expanding on current medicines to provide optimal treatments for the full range of patients with HAE, including expanding beyond plasma products in this area – such as advancing garadacimab, CSL’s first-in-class monoclonal antibody targeting activated Factor XII (FXIIa) as a prospective long‑term prophylactic treatment for patients for HAE. Haematology CSL remains focused on easing the burden of disease and improving the lives of patients with rare bleeding disorders. Significant progress has been achieved in recent years in the treatment of haemophilia A and B through the introduction of innovative recombinant coagulation factor medicines and HEMGENIX® (etranacogene dezaparvovec), an AAV5 (adenoassociated virus) gene therapy for the treatment of haemophilia B, which has been approved in the United States, Europe, United Kingdom, Switzerland and Australia. Moreover, exciting R&D efforts are underway to explore potential new indications in benign haematology and innovative therapies in haemostasis and thrombosis. This includes initiating a pivotal 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 (anti- 2 anti‑plasmin), a first-in-class, chimeric monoclonal antibody as a 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. A nebulised delivery of immunoglobulin is an example of the intersection between immunology and respiratory disease. Limited Annual Report 2023/24 20
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