Akeso's Novel Anti-CD47 Antibody AK117 Combined with Azacitidine and Venetoclax Demonstrates Significant Survival Benefits in Phase II AML Trial at EHA 2026

Akeso, Inc. (9926.HK) announced that its novel anti-CD47 antibody AK117, in combination with azacitidine and venetoclax, demonstrated significant survival benefits in a Phase II randomized, double-blind, controlled trial for acute myeloid leukemia (AML) patients ineligible for intensive chemotherapy.

The AK117-206 study data was presented as an oral presentation at the European Hematology Association (EHA) 2026 annual meeting, showing promising efficacy and safety profiles that could establish a new treatment paradigm for this difficult-to-treat patient population.

Significant Survival Benefits

At a median follow-up of 10 months for the AK117 combination arm versus 8.8 months for the control group, the treatment demonstrated substantial improvements in event-free survival (EFS). The AK117 combination achieved a median EFS of 9.1 months compared to 6.9 months with standard therapy, representing a hazard ratio of 0.46.

The EFS rates further illustrated the treatment advantage: at 6 months, 67.8% of AK117 patients remained event-free versus 55.5% in the control group. By 9 months, the gap widened considerably to 53.2% versus 14.1%, respectively.

Overall survival data proved even more compelling. The median overall survival for the AK117 combination arm had not yet been reached at the time of analysis, while the control group median stood at 8.3 months, with a hazard ratio of 0.46. The 6-month OS rate was 83.3% for AK117 versus 73.2% for control, and the 9-month OS rate reached 78.7% versus 43.1%.

Deep Tumor Response

The AK117 combination regimen induced deep tumor responses. The objective response rate reached 80.0% in the treatment arm versus 66.7% in the control group. The composite complete response rate was 56.7% versus 53.3%, respectively.

Notably, among patients achieving composite complete response, a higher proportion in the AK117 group achieved minimal residual disease negativity: 46.7% versus 36.7% in the control group. The median duration of composite complete response was substantially longer with AK117 at 10.4 months compared to 5.6 months with standard therapy.

Favorable Safety Profile

The safety profile of the AK117 combination was manageable and consistent with the known profiles of venetoclax and azacitidine. Treatment-emergent adverse events and serious adverse event rates were comparable between the two groups. No new safety signals were observed.

The most common adverse events were consistent with those expected in the AML population receiving venetoclax-based therapy. Anemia occurred in 46.7% of AK117 patients versus 50.0% in the control group.

Addressing an Unmet Need

AML remains the most common type of acute leukemia in adults, characterized by abnormal clonal proliferation of myeloid blasts in bone marrow, peripheral blood, and extramedullary tissues. For patients ineligible for intensive chemotherapy, the venetoclax-azacitidine combination represents the standard of care per National Comprehensive Cancer Network guidelines, yet overall efficacy remains suboptimal with limited options for improvement.

The encouraging efficacy and safety data from the AK117 combination provide positive signals and a solid foundation for further investigation, potentially offering a new, superior treatment option for AML patients who cannot tolerate standard induction chemotherapy.

Regulatory Recognition and Pipeline Advancement

The U.S. Food and Drug Administration has granted AK117 Orphan Drug Designation for the treatment of AML, recognizing its outstanding clinical potential. AK117 is currently being developed globally in parallel for both hematologic malignancies and solid tumors, representing the first CD47 antibody worldwide to enter registrational Phase III clinical studies in solid tumors.

Multiple Phase III trials are actively enrolling, including AK117 combined with ivonescimab versus pembrolizumab as first-line treatment for PD-L1 positive head and neck squamous cell carcinoma, and AK117 combined with ivonescimab as first-line treatment for pancreatic cancer. Phase II studies in AML and myelodysplastic syndromes continue to advance.

About AK117

AK117 is a next-generation humanized IgG4 monoclonal antibody developed by Akeso that binds to CD47 expressed on tumor cells, blocking the interaction between CD47 and its receptor SIRP alpha. This mechanism enhances phagocytic activity against tumor cells, thereby inhibiting tumor growth. Clinical studies have demonstrated excellent efficacy potential with no observed dose-limiting toxicity events and a favorable safety profile.

About Akeso, Inc.

Akeso, Inc. (9926.HK) is a leading biopharmaceutical company focused on the research, development, manufacturing, and commercialization of first-in-class or best-in-class innovative biologic drugs. Founded in 2012, the company has built a comprehensive end-to-end drug discovery and development platform.

The company has developed more than 50 innovative drug candidates for the treatment of cancer, autoimmune diseases, inflammatory conditions, and metabolic disorders. Twenty-seven drugs have entered clinical trials, including 15 bispecific or multispecific antibodies. Eight drugs are currently commercially available, and three drugs across four indications have new drug applications under regulatory review.

Akeso holds the unique distinction of being the only pharmaceutical company globally with two tumor immunotherapy bispecific antibody drugs approved, demonstrating its leadership in innovative antibody development.