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

HONG KONG, Aug. 6, 2024 /PRNewswire/ -- Akeso has announced the completion of the first patient enrollment in the US for the phase II clinical trial of its innovative CD47 monoclonal antibody, ligufalimab (AK117), in combination with azacitidine for patients with newly diagnosed higher-risk myelodysplastic syndrome (HR-MDS).

Preliminary studies show that combining AK117 with azacitidine for treating MDS is safe and significantly effective. In response to the urgent need for new therapies among global MDS patients and the evolving market landscape, Akeso has launched an international multicenter Phase II clinical trial. This initiative aims to expedite AK117's global approval and commercialization process.

CD47-targeted drug development for treating MDS shows promising potential. AK117, a next-generation humanized IgG4 anti-CD47 antibody, effectively blocks the CD47-SIRPα interaction to enhance phagocytic activity against tumor cells by phagocytes.

Recent data presented at the 65th American Society of Hematology (ASH) Annual Meeting demonstrated that AK117 combined with azacitidine significantly reduces anemia and transfusion requirements in MDS patients, with favorable safety and notable efficacy. This positions AK117 as a promising treatment option for MDS patients worldwide.

In the United States alone, approximately 40,000 new cases of MDS are diagnosed annually. High-risk MDS patients typically start with azacitidine as standard therapy, but only 20% to 30% achieve complete remission, underscoring significant unmet clinical needs in global MDS treatment.

In addition to ongoing clinical trials for MDS globally, a Phase II study is underway to evaluate AK117 in combination with venetoclax and AZA as frontline therapy for AML patients who are not eligible for intensive chemotherapy.

Akeso is also actively progressing the global market development of AK117 for solid tumors. Multiple clinical trials exploring AK117's efficacy with other agents, such as PD-1/VEGF bispecific antibodies and PD-1/CTLA-4 bispecific antibodies, are enrolling participants efficiently.

About Ligufalimab (AK117)
AK117, independently developed by Akeso, is a next-generation 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, to enhance the phagocytic activity of phagocytes on tumor cells, thereby inhibiting the growth of tumors.

Currently, several phase II clinical trials are underway to investigate the potential of AK117 in combination with azacitidine for hematological tumors, as well as AK117 alone or in combination with ivonescimab and cadonilimab for various solid tumors. Preliminary studies have shown promising efficacy and safety profiles, with no observed dose-limiting toxicity events. Additionally, international multicenter clinical studies evaluating AK117 for treating MDS and AML are enrolling patients.

About Akeso
Akeso (HKEX: 9926.HK) is a leading biopharmaceutical company committed to the research, development, manufacturing and commercialization of the world's first or best-in-class innovative biological medicines. Founded in 2012, the company has created a unique integrated R&D innovation system with the comprehensive end-to-end drug development platform (ACE Platform) and bi-specific antibody drug development technology (Tetrabody) as the core, a GMP-compliant manufacturing system and a commercialization system with an advanced operation mode, and has gradually developed into a globally competitive biopharmaceutical company focused on innovative solutions.

With fully integrated multi-functional platform, Akeso is internally working on a robust pipeline of over 50 innovative assets in the fields of cancer, autoimmune disease, inflammation, metabolic disease and other major diseases, with 19 drug candidates in the clinical stage, including 8 multispecific antibodies. Akeso has successfully promoted the commercialization of three innovative biological drugs, and marketing applications of multiple indications are submitted for 4 new drugs. 安尼可®, approved for marketing in August 2021, is currently the only differentiated PD-1 monoclonal antibody that applies the IgG1 subtype with modified Fc-nulldomain. 开坦尼® (PD-1/CTLA-4 bi-specific antibody, Cadonilimab injection) was granted marketing approval in June 2022, making it the world's first bi-specific antibody drug for tumor immunotherapy and the first bi-specific antibody new drug in China.In May 2024, 依达方® (PD-1/VEGF bi-specific antibody, Ivonescimab injection), the first-in-class PD-1/VEGF bi-specific antibody independently developed by the Company, was granted marketing approval for the treatment of epidermal growth factor receptor ("EGFR") mutated locally advanced or metastatic non-squamous non-small cell lung cancer ("nsq-NSCLC"), making it the world's first approved PD-1/VEGF bi-specific antibody. The drug had been granted three Breakthrough Therapy Designations for the treatment of lung cancer by the Center for Drug Evaluation (CDE). In December 2022, a license agreement with total potential deal value of USD5 billion, plus a low double-digit royalty of product net sales in the authorized countries of the new drug, 依达方®, set a new record in overseas licensing for the transaction amount of a single innovative drug in China.

Through efficient and breakthrough R&D innovation, Akeso always integrates superior global resources, develops the first-in-class and best-in-class new drugs, provides affordable therapeutic antibodies for patients worldwide, and continuously creates more commercial and social values to become a global leading biopharmaceutical enterprise.