Development

Akeso's ligufalimab shows 80% response rate in Phase II treatment-naïve AML trial

Akeso (HKEX: 9926.HK) reported Phase II data from its ligufalimab AML trial showing an objective response rate of 80.0% versus 66.7% for placebo in...

Edited by Robert McTiernan··3 min read

Akeso (HKEX: 9926.HK) reported randomized Phase II data suggesting its anti-CD47 antibody ligufalimab may improve survival outcomes when added to azacitidine and venetoclax in frontline acute myeloid leukemia patients ineligible for intensive chemotherapy, offering one of the clearest positive signals for the CD47 class since setbacks that derailed Gilead Sciences’ magrolimab program. The data are set to be presented as an oral session at the 2026 European Hematology Association Congress 2026.

Trial specifics

The AK117-206 trial is a randomized, double-blind, placebo-controlled Phase II study evaluating ligufalimab in combination with azacitidine and venetoclax against placebo plus azacitidine and venetoclax in adults with treatment-naïve acute myeloid leukemia who are ineligible for intensive induction chemotherapy. Data were cut in November 2025 and presented as an oral session at the 2026 European Hematology Association Congress.

The 80.0% ORR in the ligufalimab arm compared to 66.7% in the control arm was accompanied by a composite complete remission rate of 56.7% versus 53.3%. Among patients achieving composite complete remission, measurable residual disease negativity was recorded in 46.7% of the ligufalimab group versus 36.7% in the control group, and median duration of composite complete remission was 10.4 months versus 6.5 months. The 9-month overall survival rate of 78.7% in the ligufalimab arm versus 43.1% in the control arm represents the most directionally striking finding in the dataset, though the follow-up remains short and the primary endpoint was not disclosed in the press release.

The safety profile was consistent with the known toxicities of azacitidine and venetoclax. Overall treatment-emergent adverse event rates and serious adverse event rates were comparable between arms. Anemia occurred in 46.7% of patients receiving ligufalimab versus 50.0% in the control arm, and Akeso said no new safety signals were identified.

Anti-CD47 therapies after magrolimab's setback

Ligufalimab is a humanized IgG4 anti-CD47 monoclonal antibody that blocks the CD47–SIRPα interaction on tumor cells, removing an inhibitory signal and enabling macrophage-mediated phagocytosis of malignant blasts. The rationale for combining it with azacitidine and venetoclax is mechanistically layered: azacitidine drives DNA hypomethylation and re-expression of silenced genes, while venetoclax restores mitochondrial apoptotic signaling through BCL-2 inhibition. Adding CD47 blockade targets a distinct immune evasion axis, in theory complementing the cytotoxic and epigenetic activity of the backbone.

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The Phase II AML data arrive in a competitive context shaped heavily by the trajectory of Gilead Sciences' magrolimab, the most advanced anti-CD47 antibody before ligufalimab. Gilead placed magrolimab on clinical hold in 2023 following a safety signal in its Phase III ENHANCE-2 trial in AML, dealing a setback to the CD47 class and raising questions about on-target hematologic toxicity. Akeso has highlighted that ligufalimab's IgG4 isotype and engineering are designed to reduce red blood cell binding and hemagglutination relative to earlier CD47 antibodies, a property the company positions as a potential tolerability advantage. The anemia rates in AK117-206 — 46.7% in the ligufalimab arm versus 50.0% in the control arm — are consistent with that framing, though cross-trial comparisons are limited by differences in patient populations, dosing, and study design.

A more recent entrant to the frontline setting is Taiho Oncology's Inqovi (decitabine/cedazuridine) in combination with venetoclax, which received US FDA approval in 2026 as the first fully oral regimen for this population. That approval raises the administrative convenience bar: future approvals will need to demonstrate either superior efficacy or a differentiated safety or convenience profile relative to a regimen that patients can take entirely at home.

Ligufalimab holds US FDA Orphan Drug Designation for AML, which provides development incentives but does not constitute a clinical endorsement. Akeso also stated that ligufalimab is the first anti-CD47 monoclonal antibody globally to enter a registrational Phase III trial in solid tumors, indicating a parallel development program outside hematology. Akeso made a presentation of Phase II data for ligufalimab in frontline AML at the EHA 2026.

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