HONG KONG, China I September 27, 2023 I Akeso, Inc. (the Company, Akeso, 9926.HK) announced that the U.S. Food and Drug Administration (FDA) has cleared the Company’s Investigational New Drug (IND) application for AK117, a next generation CD47 monoclonal antibody in combination with azacitidine for treatment of patients with newly diagnosed higher-risk myelodysplastic syndromes (MDS). The upcoming study, known as a randomized, double-blind, global multi-center Phase II study, is set to be conducted in the United States.

Based on the favorable safety profile and remarkable efficacy of AK117 in combination with azacitidine for the treatment of newly diagnosed higher-risk MDS patients, as showcased in previous studies, and the strong demand from MDS patients worldwide, Akeso has conducted comprehensive scientific communication and discussions with the FDA. Akeso believes that this study will facilitate the global development process for AK117.

“With more time and space, we will consistently implement a comprehensive international strategy to explore and advance our innovative therapies. For the benefit of more patients, we will leverage global resources to improve the success rate of AK117.” Said Dr. Michelle Xia, the founder, Chairwoman, CEO, and President of Akeso.

Anemia is a significant symptom of MDS, and effectively managing anemia and minimizing blood transfusion are crucial aspects of disease control. In contrast to other anti-CD47 antibody drugs, which have been reported to worsen anemia in MDS patients by causing RBC hemagglutination, AK117 eliminated RBC hemagglutination and also enabled to maintain full effectiveness of CD47 blockade on tumor cells, which resulted in excellent antitumor efficacy and favorable safety profile of AK117.

The previous studies of AK117 in combination with azacitidine showed positive results in patients with newly diagnosed higher-risk MDS. AK117 reduced anemia and transfusion requirements among MDS patients, and demonstrated a favorable safety profile and remarkable efficacy. These findings position AK117 as a promising therapeutic option for MDS patients worldwide.

Notably, Akeso is consistently advancing the development of AK117 as a therapeutic agent in combination with various agents such as PD-1/CTLA-4 and PD-1/VEGF, for treatment of multiple hematologic malignancies and solid tumors.

About Ligufalimab (AK117)

AK117, independently developed by Akeso, is a next generation of humanized lgG4 anti-CD47 antibody without hemagglutination effect. AK117 can bind to CD47 expressed on tumor cells and block the interaction between CD47 and SIRPα, in order to enhance the phagocytic activity of phagocytes on tumor cells, thereby inhibiting the growth of tumors.