A randomized, placebo-controlled Phase II trial of ligufalimab (AK117), a CD47-targeting antibody, has reported survival data in frontline acute myeloid leukemia (AML) that could help revive interest in the CD47 field following the failure of several high-profile programs, including Gilead's magrolimab. For Hong Kong-based Akeso, Inc. (HKEX: 9926.HK), the data also represent a meaningful clinical milestone for ligufalimab, which the company is simultaneously advancing in solid tumors — making AML a potential anchor indication for a broadly deployed asset.
The AK117-206 trial is a randomized, double-blind, placebo-controlled Phase II study evaluating ligufalimab added to azacitidine and AbbVie/Genentech's Venclexta (venetoclax) versus placebo plus the same backbone in patients ineligible for intensive induction chemotherapy. At a median follow-up of approximately 10 months in the ligufalimab group, median overall survival had not been reached versus 8.3 months in the control arm, with a hazard ratio of 0.46. The 9-month OS rate was 78.7% versus 43.1%. Median event-free survival was 9.1 months versus 6.9 months, also with an HR of 0.46. The objective response rate was 80% versus 66.7%, and the proportion of patients achieving composite complete remission with MRD negativity was 46.7% versus 36.7%. The duration of composite complete remission was 10.4 months versus 5.6 months in the control arm. The primary endpoint was not disclosed in the press release. Safety was comparable between arms, with anemia rates of 46.7% versus 50%, and no new safety signals identified.
The data were presented as an oral session at the 2026 European Hematology Association Congress.
Competitive context
The frontline AML setting for patients ineligible for intensive chemotherapy is currently dominated by venetoclax-based combinations. The standard of care — venetoclax plus azacitidine — was established through the VIALE-A trial, which demonstrated a median OS of approximately 14.7 months versus 9.6 months for azacitidine alone. Ligufalimab is being added on top of this backbone, so the relevant comparison is not to azacitidine monotherapy but to the venetoclax-azacitidine doublet itself. The control arm's 8.3-month median OS in AK117-206 is lower than the VIALE-A benchmark, which may reflect differences in patient population, geography, or trial design; cross-trial comparisons are limited and should be interpreted cautiously.
The CD47 field itself carries significant reputational weight after Gilead's magrolimab was placed on clinical hold in the ENHANCE-2 Phase III AML trial due to a safety signal. Akeso has emphasized that ligufalimab's IgG4 isotype and molecular engineering are designed to reduce red blood cell binding and hemagglutination — a key differentiator from earlier CD47 antibodies. The anemia rates in AK117-206 were numerically lower in the ligufalimab arm than in controls, consistent with that framing, though the small trial size and cross-study differences preclude definitive conclusions.