Akeso's Ligufalimab Shows Promising Phase II Results in Frontline AML Treatment
Key Insights
Akeso (search)'s ligufalimab (AK117) demonstrated an 80.0% objective response rate versus 66.7% in the control arm when combined with azacitidine and venetoclax in treatment-naïve AML patients.
The ligufalimab combination showed superior survival outcomes with a 9-month overall survival rate of 78.7% compared to 43.1% in the control arm.
The triplet regimen exhibited a manageable safety profile with no new safety signals, offering a potentially better-tolerated treatment option for AML patients ineligible for intensive chemotherapy.
Akeso (search) announced compelling results from its randomized, double-blind, placebo-controlled Phase II trial (AK117-206) evaluating ligufalimab (AK117) in combination with azacitidine and venetoclax for treatment-naïve acute myeloid leukemia patients ineligible for intensive chemotherapy. The data was presented at the 2026 European Hematology Association Congress.
Enhanced Efficacy Outcomes
The ligufalimab-based triplet regimen demonstrated superior efficacy compared to the control arm. The objective response rate reached 80.0% in the ligufalimab arm versus 66.7% in the control arm, with composite complete remission rates of 56.7% versus 53.3%, respectively. Among patients achieving composite complete remission, the measurable residual disease negativity rate was notably higher in the ligufalimab arm at 46.7% compared to 36.7% in the control group.
The duration of response showed substantial improvement, with median duration of composite complete remission extending to 10.4 months in the ligufalimab arm versus 6.5 months in the control arm.
Survival Benefits
At a median follow-up of 8.84 months, the survival data revealed encouraging trends favoring the ligufalimab combination. Median overall survival in the ligufalimab arm was not yet reached, compared to 8.3 months in the control arm. The 9-month overall survival rate demonstrated a striking difference at 78.7% in the ligufalimab arm versus 43.1% in the control arm, while 6-month overall survival rates were 83.3% versus 73.2%, respectively.
Safety Profile
The combination therapy exhibited a manageable safety profile with no new safety signals observed. The incidence of overall treatment-emergent adverse events and serious adverse events was comparable between treatment arms. The most common treatment-emergent adverse events were consistent with those expected in the context of AML and azacitidine plus venetoclax therapy. Anemia occurred in 46.7% of patients in the ligufalimab arm versus 50.0% in the control arm.
Drug Development and Regulatory Status
Ligufalimab is Akeso (search)'s proprietary next-generation humanized IgG4 anti-CD47 (search) monoclonal antibody. The drug has already received Orphan Drug Designation from the U.S. FDA for AML treatment. Akeso is advancing ligufalimab clinical development programs across both hematologic malignancies and solid tumors, with the distinction of being the first anti-CD47 monoclonal antibody worldwide to enter a registrational Phase III clinical trial in solid tumors.
The study targeted patients with treatment-naïve acute myeloid leukemia who are ineligible for intensive chemotherapy, representing a vulnerable patient population with significant unmet medical needs. The combination approach offers a potentially better-tolerated treatment option for these patients who cannot undergo standard intensive chemotherapy regimens.