CSL Limited Annual Report 2021/22 30 CSL’s therapeutic areas Therapeutic Areas Platform Plasma Fractionation Immunology Haematology Respiratory Cardiovascular and Metabolic Influenza Vaccines (Seasonal, Pandemic) Transplant Recombinant Technology Cell and Gene Therapy Cell-based Adjuvanted Egg-based Immunology Our efforts in this area focus on providing trusted products and technologies to serve patients with a range of serious immunologic and neurologic diseases, including primary and secondary immunodeficiencies (PID and SID) and chronic inflammatory demyelinating polyneuropathy (CIDP). We are optimising patient experience and convenience through more flexible ways to dose and administer our existing immunoglobulin products. We are also progressing key recombinant assets in early development such as our anti-G-CSFR monoclonal antibody, CSL324, in certain neutrophilic dermatoses. We continue to build on our strong 40-year legacy in hereditary angioedema (HAE) as we look to expand on our current medicines to provide optimal treatments for the full range of HAE patients, including our recombinant monoclonal antibody garadacimab, which is currently in Phase III development. Haematology CSL remains focused on easing the burden of disease and improving the lives of patients with rare bleeding disorders. We have made major advances in haemophilia A and B in recent years with the launch of our novel recombinant coagulation factor medicines and through the acquisition of exclusive global licence rights to commercialise etranacogene dezaparvovec, an AAV5 (adeno-associated virus) gene therapy for the treatment of haemophilia B, which is currently under regulatory review. Additionally, we are undertaking exciting research and development efforts to explore new indications in haematology as well as novel therapeutics in haemostasis and thrombosis. This includes planning for 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. Cardiovascular and Metabolic The cardiovascular and metabolic therapeutic area is focused on improving and extending the lives of patients with cardiovascular disease (CVD) and diabetes. CSL112, apolipoprotein A-I (human), is being developed to reduce the risk of recurrent cardiovascular events during the 90-day high-risk period following a heart attack, the period when the majority of first-year recurrent cardiovascular events occur. If successful, CSL112 will be the first therapy to demonstrate cardiovascular risk reduction through the novel apoA-I mechanism and will transform how acute myocardial infarction patients at high-risk of recurrent cardiovascular events are treated. Beyond CVD, type 2 diabetes is one of the fastest growing chronic diseases. CSL346, our innovative anti-VEGF-B monoclonal antibody therapy is being studied to augment the current standard of care to decrease the progression of diabetic kidney disease, a frequent and serious long-term diabetic complication. Respiratory In addition to our existing product ZEMAIRA®/RESPREEZA® for patients with alpha 1 antitrypsin deficiency, CSL is investigating new clinical treatments for respiratory diseases using novel recombinant monoclonal antibodies and plasma-derived therapies to address this need. Trabikibart CSL311, our 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, we have started a clinical development program with garadacimab, the first of our compounds being explored in this disease area. CSL787, our plasma-derived, inhaled immunoglobulin is being investigated for patients with bronchiectasis and severe COPD patients. Transplant In kidney transplant recipients, antibody-mediated rejection (AMR) is a leading cause of allograft loss, and there is significant unmet need for effective treatments. Clazakizumab, our anti-interleukin-6 (IL-6) monoclonal antibody, is currently being investigated in a Phase III clinical trial (IMAGINE) for the potential treatment of chronic active antibody-mediated rejection. In haematopoietic stem cell transplantation, acute graft-versus-host disease (GvHD) is a life-threatening type of rejection where the donor cells attack the recipient; it is a leading cause of mortality and morbidity following transplant. There is a significant unmet need for more effective, less toxic therapies for GvHD. We are investigating alpha 1 antitrypsin (AAT, ZEMAIRA®) for the prevention and treatment of acute GvHD in two Phase III studies. Influenza Vaccines With a focus on influenza, developing new and better vaccines across all age groups in expanded markets is a strategic priority for CSL Seqirus, including further advancing our cell-based manufacturing technology and our MF59® adjuvant and developing our self-amplifying messenger RNA (sa-mRNA) technology, to enhance the immune response of those particularly vulnerable to influenza such as children and older adults. We are also investigating a quadrivalent adjuvanted cell culture influenza vaccine (aQIVc) which combines FLUCELVAX® antigen with MF59® adjuvant, an additive that acts to strengthen the immune response to vaccination. 6 Powered by Innovation
RkJQdWJsaXNoZXIy MjE2NDg3