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].

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