CD47 Monoclonal Antibody (AK117) Obtained Approval for Phase I/II Clinical Trial for Treatment of Medium- to High-Risk Myelodysplastic Syndromes (MDS)

On May 18, 2021, Akeso, Inc. (9926.HK) announced that CD47 monoclonal antibody (AK117), a novel immuno-oncology drug independently developed by Akeso, obtained approval from the National Medical Products Administration (NMPA) of the Peoples Republic of China for phase I/II clinical trials on the treatment of medium- to high-risk myelodysplastic syndromes ("MDS").


AK117 is a new-generation CD47 monoclonal antibody independently developed by Akeso that shows exceptional safety profile in previously published data. Currently, the dose escalation of 30 mg/kg once-weekly (“QW”) dosage has been completed, and subjects in the 45 mg/kg QW cohort are being administered. There has not been any incident of drug-related anemia dose-limiting toxicity (DLT) in each dose escalation subject cohort of AK117 and all the subjects in each cohort tolerated the drugs well. The CD47 receptor occupancy rate (RO) of the peripheral blood T cells has reached and maintained at 100% in the 3 mg/kg cohort.


Phase I clinical trial of dose escalation and expansion of AK117 for the treatment of patients with advanced solid tumors and lymphoma have been carried out in China and overseas in 2020. Akeso intends to publish the research results of the safety profile of AK117 for the treatment of advanced or metastatic solid tumors at the 2021 American Society of Clinical Oncology Annual Meeting (ASCO 2021).


MDS is a type of heterogeneous myeloid clonal disease originating from hematopoietic stem cells. It is characterized by ineffective hematopoiesis, refractory cytopenia and high risk of transforming into acute myeloid leukemia (AML). With the global aging population, there are huge unmet clinical needs for the treatment of MDS. According to relevant clinical trials, CD47 monoclonal antibody combination treatment of MDS has excellent potential in the efficacy and safety profile. As a new generation of CD47 monoclonal antibody, AK117 is expected to achieve better performance as compared with similar drugs.



AK117 is a novel humanized IgG4 mAb independently developed by the Company. It can bind with CD47 expressed on tumor cells to prevent the interaction between CD47 and its receptor, SIRPα, expressed on macrophages so as to enhance phagocytosis to inhibit the growth of tumor cells. According to the pre-clinical research, AK117 can maintain the activeness of antibody and prevent hemagglutination. The presence of AK117-mediated macrophages also significantly reduces phagocytosis of red blood cells. Unlike other CD47 antibodies, AK117 does not cause hemagglutination. AK117-mediated macrophages have significantly weaker phagocytosis of red blood cells in comparison to tumor cells. Compared with the obvious symptoms of anemia exhibited by other CD47 antibodies, AK117 only causes slight red blood cell changes in cynomolgus monkeys and no toxic effects on platelets have been observed.



The Company is a biopharmaceutical company dedicated to the research, development, manufacturing and commercialization of new innovative antibody drugs that are affordable to patients worldwide. Since the Company’s establishment, the Company has established an end-to-end comprehensive drug development platform (ACE Platform) and system, encompassing fully integrated drug discovery and development functions, including target validation, antibody drug discovery and development, CMC production process development, and GMP compliant scale production. The Company has also successfully developed a bi-specific antibody drug development technology (Tetrabody technology). The Company currently has a pipeline of over 20 innovative drugs for the treatment of major diseases like tumors, autoimmune diseases, inflammation and metabolism diseases, 13 of which have entered clinical stage, including two first-in-class bi-specific antibody drugs (PD-1/CTLA-4 and PD-1/VEGF). The Company’s vision is to become a global leading biopharmaceutical company through research and development of high efficacy and breakthrough new drugs that are first-in-class and best-in-class therapies.