A new treatment combination—bexmarilimab plus azacitidine—has shown highly encouraging results for people with higher-risk myelodysplastic syndromes (MDS), especially those whose disease has come back or stopped responding to previous therapies. Results from the phase 2 BEXMAB trial were recently presented at the 2025 American Society of Clinical Oncology Annual Meeting.
According to the study results, patients with relapsed or refractory higher-risk MDS who received bexmarilimab plus azacitidine had an overall response rate of 63%. This is a significant improvement compared to the typical response rates of 0–15% seen with standard therapies after previous treatments have failed. For patients who had not received prior treatment, the response rate was even higher, reaching up to 72%.
Patients in the study also experienced a median overall survival of approximately 13.4 months, which is more than double the 5–6 months usually expected for this difficult-to-treat group. Many patients saw a substantial reduction in abnormal cells in the bone marrow, and over half of newly diagnosed patients had a complete clearance of these cells.
Safety and Tolerability:
The combination of bexmarilimab and azacitidine was generally well tolerated. No dose-limiting toxicities were reported, and most side effects were manageable and similar to those seen with azacitidine alone. The most common serious side effects included low blood counts and infections, which are expected in this patient population.
What Makes Bexmarilimab Unique?
Bexmarilimab is a novel antibody that targets the Clever-1 receptor, helping to reprogram immune cells in the bone marrow to better fight the cancer. This approach may help overcome treatment resistance and offers new hope for patients with few remaining options.
Next Steps:
Based on these promising results, a larger phase 3 clinical trial is being planned to further test the combination in newly diagnosed high-risk MDS. The U.S. FDA has also granted bexmarilimab orphan drug designation for MDS, recognizing its potential to address a significant unmet need.
If you or a loved one is affected by higher-risk MDS, these developments may offer new hope. Talk to your healthcare provider about ongoing clinical trials and emerging treatment options.
Reference:
Daver N, Kontro M, et al. Efficacy of macrophage checkpoint Clever-1 inhibition with bexmarilimab plus azacitidine in myelodysplastic syndrome: Results from the ph1/2 BEXMAB study. Journal of Clinical Oncology. Volume 43, Number 16_suppl. Abstract 6513.
