From the Abstract13 Oct 2025 08:00
Results
Combination treatment induced immune activation in the BM, with a mean increase of 87% in monocyte HLA-DR expression, reflecting antigen presentation capacity and 25% increase of BM CD8+ T cells, already after cycle 1. Single-cell RNA sequencing of bone marrow samples from responding patients revealed extensive remodelling of the myeloid compartment, including a reduction in immature monocytes and GMP-like cells. Gene-set enrichment analysis indicated suppression of oxidative phosphorylation in blasts, suggesting a direct metabolic effect. These findings support a dual mechanism of action: immune-mediated activation and direct modulation of blast metabolism. In addition to these translational results, full pharmacokinetic (PK) and pharmacodynamic (PD, including correlation with clinical response) data will be presented for the first time.
Conclusions
Together, the clinical and biological findings highlight bexmarilimab’s potential as a novel therapeutic strategy for HR MDS and support its advancement into phase 3.