Faron Pharmaceuticals (FARN) ESMO 2025 Conference summary

Key clinical findings and trial data

• Bexmarilimab plus azacitidine demonstrated an 85% objective response rate and 45% complete remission rate in treatment-naive higher-risk MDS patients, with 78% response in TP53-mutated cases and 63% ORR in relapsed/refractory HMA-failed patients.

• 23% of trial participants were bridged to allogeneic transplantation, and the median overall survival for HMA-failed patients was 13.4 months.

• Higher bone marrow Clever-1 target engagement correlated with better responses, especially in patients with less than 5% blasts at baseline, who achieved a 100% response rate.

• Bexmarilimab plus azacitidine showed a favorable safety profile, with 36% of patients experiencing related adverse events, no grade 5 events, and lower rates of severe cytopenias and drug withdrawal compared to other agents.

• Case studies highlighted full disease clearance and normalization of blood values in complex, high-risk patients after therapy.

Mechanism of action and biomarker insights

• Bexmarilimab targets Clever-1, reprogramming macrophages to promote immune activation, T cell response, and impair blast cell metabolism, supporting improved hematopoiesis and faster recovery.

• Target engagement in bone marrow was significantly higher in responders, particularly in patients with low baseline blast counts, with scRNA sequencing showing increased progenitor cells and decreased immunosuppressive cells after treatment.

• Bexmarilimab plus azacitidine reactivates myeloid and T-cell immunity, with HLA-DR induction associated with response and apoptotic priming of blasts.

• The drug's unique mechanism allows efficacy in low blast count patients, a group where other agents like venetoclax have failed.

• Preclinical data in mice support improved recovery from chemotherapy when treated with bexmarilimab.

Safety profile and differentiation

• The combination therapy is well tolerated, even in frail and cytopenic patients, with a safety profile similar or better than azacitidine monotherapy.

• No grade 5 bexmarilimab-related adverse events were reported.

• Lower rates of drug withdrawal and severe cytopenias compared to other agents.

• Safety is a key differentiator, with lower rates of grade 3/4 adverse events compared to other developmental assets in MDS.