ESG Report 2023

22 5.5 Focus on Rare Disease Under the guidance and favorable policies of the revised "Drug Registration Management Measures" and "Rare Disease Diagnosis and Treatment Guidelines", The Group actively leverages its own research system advantages, responds to national policies, thoroughly assesses the rare disease market conditions, and increases investment in the research and development of rare disease drugs. The aim is to improve the clinical treatment of rare diseases in our country. The United Laboratories is committed to enhancing the accessibility of innovative therapeutic drugs for rare disease patients, in order to benefit a greater number of patients. PNH is an acquired hemolytic disease in which a mutation in the PIG-A gene of hematopoietic stem cells leads to the loss of a group of membrane proteins anchored to the cell surface through glycosylphosphatidylinositol (GPI). This results in changes in cellular properties and sensitivity to complement, leading to intravascular hemolysis, potential bone marrow failure, and thrombosis. The incidence rate of PNH in Western countries is (1-2)/million population/year, with a standardized rate of 1.3/million population/year. In our country, the overall incidence rate is around 1/100,000, which is higher than in Europe and America. Paroxysmal Nocturnal Hemoglobinuria (PNH) At present, the treatment for PNH is to inhibit complement C3 or C5 monoclonal antibodies, which can effectively reduce their levels in PNH patients. However, these drugs have defects such as injection pain and infection risk. Our research project LB2023, an oral small molecule new drug, which is to develop a compound that inhibits the complement pathway through the CFB target to achieve the inhibitory effect on complement in PNH patients. Compared with C3 and C5 monoclonal antibodies that completely inhibit the complement pathway, this drug will be safer and more compliant with medication. In December 2023, Novartis CFB target drug LNP023 became the world's first monotherapy drug approved by the FDA for treating PNH. Therefore, our new drug targeting the same target has an opportunity to be used for the treatment of rare diseases such as PNH in the future. Environmental, Social and Governance Report 2023 The United Laboratories International Holdings Limited