Synribo (omacetaxine mepesuccinate)

Therefore, this platform is particularly suited for the treatment of FLT3-ITD AML while potentially applicable to other AML subtypes with high CD71 expression. By enabling specific intracellular accumulation of HHT and multitarget inhibition of FLT3 signaling pathways, this system achieves enhanced anti-AML efficacy both in vitro and in vivo, offering strong potential for future clinical translation.

The resulting nanoparticles were investigated in a refractory AML mouse model (AML1-ETO & C-KITD816V) with a high level of CXCR4 and in the t(8; 21)-positive AML cell line Kasumi-1. It was shown that E5-LNP@siAE effectively achieved RNAi of AML1-ETO and antagonism of CXCR4, thereby synergistically inducing effective multi-lineage differentiation, leading to significantly enhanced differentiation-post apoptotic responses of AML cells to homoharringtonine and remarkably prolonged survival in refractory AML mice.

During follow-up, measurable residual disease (MRD) fluctuated repeatedly despite continued morphologic remission, prompting venetoclax-based therapy, including cytarabine plus venetoclax, azacitidine plus venetoclax with homoharringtonine, and later azacitidine plus venetoclax combined with tislelizumab. At the last follow-up in December 2025, the patient remained alive. This case highlights that peri-iliac MS may mimic vascular and urologic disease and that serial MRD monitoring can be useful for guiding postremission treatment adaptation in AML with extramedullary disease.

We previously conducted a prospective clinical trial on R/R AML with chemotherapy regimen BHA (bortezomib, homoharringtonine and cytarabine), which demonstrated promising efficacy in patients with FLT3-mutated R/R AML. Notably, this degradation effect was partially reversed by chloroquine. These findings demonstrate that bortezomib and homoharringtonine have synergistic effects and lead to degradation of FLT3-ITD oncoprotein, potentially contributing to a higher complete remission rate in FLT3-ITD R/R AML.

P2, N=41, Active, not recruiting, Institute of Hematology & Blood Diseases Hospital, China | Recruiting --> Active, not recruiting | N=60 --> 41 | Trial completion date: Jun 2027 --> Jun 2028 | Trial primary completion date: Jun 2026 --> Dec 2027

We report a case of a 42-year-old patient with EVI1-positive AML harboring the MLL-AF6 fusion gene, who failed to achieve remission after undergoing standard "IA" induction therapy and was then treated with VAH (venetoclax, azacitidine, and homoharringtonine) consolidation chemotherapy. This case suggests that the combination of ATRA with the VAH regimen may demonstrate promising efficacy and an acceptable safety profile in patients with EVI1-positive AML who are refractory to conventional chemotherapy. However, further clinical studies are required to confirm its wider applicability.

P1/2, N=682, Recruiting, Ascentage Pharma Group Inc. | N=458 --> 682

In a clinical trial (ChiCTR2500097714), erlotinib (an EGFR inhibitor) combined with azacitidine plus the HAG regimen, which consists of homoharringtonine, a low dose of cytarabine, and granulocyte colony-stimulating factor priming, achieves a remission rate of 75% in relapsed/refractory AML, likely via MMRN1/EGFR axis blockade. Our findings establish MMRN1 as a dual-functional target for LSC maintenance and immune evasion and propose that disrupting MMRN1 or EGFR remodels the immunosuppressive tumor microenvironment, offering a promising strategy for AML immunotherapy.

P=N/A, N=20, The FIrst Affiliated Hospital, College of Medicine, Zhejiang University; The FIrst Affiliated Hospital, College of Medicine, Zhejiang University

P=N/A, N=60, The First Hospital of China Medical University; The First Hospital of China Medical University