Core Viewpoint - Akeso Inc. has received Orphan Drug Designation from the U.S. FDA for its monoclonal antibody ligufalimab (AK117) aimed at treating acute myeloid leukemia (AML) [1][2]. Company Overview - Akeso is a biopharmaceutical company focused on developing innovative biological medicines, with a robust pipeline of over 50 assets in various disease areas, including cancer and autoimmune diseases [15]. - The company utilizes an integrated R&D innovation system and has developed a comprehensive drug development platform [15]. Drug Development and Clinical Trials - Ligufalimab is currently in international clinical development for both hematologic malignancies and solid tumors, with completed patient enrollment in a Phase II study for higher-risk myelodysplastic syndromes [3][4]. - It is the first CD47 monoclonal antibody to enter Phase III trials for solid tumors, with ongoing studies for head and neck squamous cell carcinoma and pancreatic cancer [4]. Clinical Need and Market Opportunity - AML is the most common type of acute leukemia in adults, with limited treatment options for patients ineligible for intensive chemotherapy [5]. - Current treatments, such as venetoclax combined with azacitidine, show a high relapse rate, indicating a significant unmet clinical need [6]. Mechanism of Action - Ligufalimab targets CD47 on tumor cells, enhancing macrophage-mediated phagocytosis and inhibiting tumor growth, with improved safety and efficacy compared to other CD47-targeting agents [7]. Efficacy and Safety Profile - Clinical trials indicate that ligufalimab combined with azacitidine has a favorable safety profile, achieving a complete remission rate of 50% and a composite complete remission rate of 55% [9]. - A Phase II study is underway to further assess the safety and efficacy of ligufalimab in combination with venetoclax and azacitidine for AML patients [10].

Business Highlights - Akeso's commercial sales revenue in 2025H1 reached RMB 1.402 billion, a 49% year-on-year increase[16, 24] - The company has 13 Phase III/registrational trials of ivonescimab covering lung cancer and expanding to cold tumors[13] - The company has 10 phase III clinical studies of cadonilimab across lung cancer, gastric cancer, liver cancer and cervical cancer[13] - Penpulimab receives FDA approval in the United States[14] Pipeline Development - Three blockbuster new drug candidates entered clinical stage[19] - Six Phase III clinical trials reached the primary endpoint[20] - Ivonescimab's first global Phase III trial received positive results[84] - Manfidokimab (IL-4Rα) Reached Endpoints in the Registrational Phase III Trial for Atopic Dermatitis[116] Financial Performance - 1H 2025 Revenue reached RMB 1.412 billion, a 33.7% growth from 1H in 2024[16] - The company has cash and short-term financial assets of approximately RMB 7.14 billion[16] - Sales and Marketing expense as a percentage of sales decreased from 55% in 2024H1 to 48% in 2025 H1[136, 140]