First US Patient Enrolled in Phase 2 Therapy of Akeso's Ligufalimab with Azacitidine for Myelodysplastic Syndrome

HONG KONG, Aug. 6, 2024 — Akeso has achieved a significant milestone with the enrollment of the first patient in the United States for its Phase II clinical trial of ligufalimab (AK117), a novel CD47 monoclonal antibody. This trial, which pairs AK117 with azacitidine, targets patients newly diagnosed with higher-risk myelodysplastic syndrome (HR-MDS).

Initial research suggests that the combination of AK117 and azacitidine is both safe and highly effective in treating MDS. This has driven Akeso to initiate a global, multicenter Phase II trial, aiming to accelerate the worldwide approval and market introduction of AK117.

The development of CD47-targeted therapies holds substantial promise for treating MDS. AK117, a next-generation humanized IgG4 anti-CD47 antibody, disrupts the CD47-SIRPα interaction, thereby enhancing the ability of phagocytes to attack tumor cells.

At the 65th American Society of Hematology (ASH) Annual Meeting, recent data highlighted that AK117 combined with azacitidine significantly alleviates anemia and reduces the need for transfusions in MDS patients. This combination has shown favorable safety and efficacy, positioning AK117 as a potential global treatment option for MDS.

In the U.S. alone, around 40,000 new MDS cases are diagnosed each year. Standard treatment for high-risk MDS patients often begins with azacitidine, but only 20% to 30% achieve complete remission. This underscores a significant unmet need in MDS therapy globally.

Beyond MDS, Akeso is conducting a Phase II study to evaluate AK117 in combination with venetoclax and azacitidine as a frontline treatment for AML patients ineligible for intensive chemotherapy.

Akeso is also making strides in developing AK117 for solid tumors. Several clinical trials are assessing the efficacy of AK117 when used alongside other agents, such as PD-1/VEGF and PD-1/CTLA-4 bispecific antibodies, with participant enrollment progressing efficiently.

About Ligufalimab (AK117)

Ligufalimab (AK117) is Akeso's independently developed next-generation humanized IgG4 anti-CD47 antibody that does not induce hemagglutination. It binds to CD47 on tumor cells and blocks its interaction with SIRPα, enabling phagocytes to target and eliminate tumor cells more effectively, thus inhibiting tumor growth.

Multiple Phase II trials are currently exploring AK117's potential, both alone and in combination with other therapies, for treating hematological and solid tumors. Preliminary findings indicate promising efficacy and safety profiles, with no dose-limiting toxicities observed. Furthermore, international multicenter clinical studies are ongoing for MDS and AML treatment.

About Akeso

Founded in 2012, Akeso is a leading biopharmaceutical company dedicated to the research, development, manufacturing, and commercialization of pioneering biological medicines. The company has developed a unique integrated R&D innovation system, known as the ACE Platform, along with its proprietary bi-specific antibody drug development technology, Tetrabody. Akeso also features a GMP-compliant manufacturing system and a cutting-edge commercialization approach.

Akeso's robust pipeline includes over 50 innovative assets targeting cancer, autoimmune diseases, inflammation, metabolic disorders, and more, with 19 candidates currently in clinical stages. This includes eight multispecific antibodies. The company has brought three innovative biological drugs to market and has submitted marketing applications for multiple indications for four new drugs.

Akeso integrates superior global resources to develop groundbreaking drugs, providing affordable therapeutic options for patients worldwide. Through its commitment to innovation and social value, Akeso aims to be a global leader in the biopharmaceutical industry.

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