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Highlights
- 80% overall response rate (16 out of 20) in refractory/relapsed MDS patient population.
- 70% of patients achieved complete/partial response, with four progressing to bone marrow transplant.
- Webinar scheduled for today to discuss promising BEXMAB results.
Faron Pharmaceuticals Ltd. (AIM:FARN, First North: FARON), a clinical-stage biopharmaceutical company focused on developing novel immunotherapies for cancer, has announced the full analysis of its positive Phase 2 interim readout for BEXMAB, presented at the 66th American Society of Hematology (ASH) Annual Meeting and Exposition. The data reveals significant and promising results in the treatment of refractory or relapsed myelodysplastic syndromes (r/r MDS).
Breakthrough Clinical Results
The study, which evaluates the safety and efficacy of bexmarilimab – a novel anti-Clever-1 humanized antibody – in combination with standard-of-care azacitidine, showed an impressive 80% overall response rate (ORR) in the r/r MDS patient population. This marks a critical achievement, especially in patients who have previously failed treatment with hypomethylating agents (HMAs). Notably, 70% of patients (14 out of 20) achieved deep and durable responses, including complete responses (CR), marrow complete remissions (mCR), or partial responses (PR). Four patients have already progressed to receive bone marrow transplants, reflecting the therapy's potential to provide meaningful clinical benefits.
Promising Overall Survival and Tolerability
The estimated median overall survival (mOS) for this patient cohort is approximately 13.4 months, which is a significant improvement over typical outcomes in this difficult-to-treat population. The combination of bexmarilimab and azacitidine remains well-tolerated by patients, further emphasizing the potential of this treatment strategy to improve outcomes without excessive toxicity.
The findings are underscored by compelling mechanistic insights. Bexmarilimab's action targets the Clever-1 protein, which is implicated in suppressing the immune response in cancer. The study demonstrated robust Clever-1 target engagement and expression in the bone marrow, leading to increased antigen presentation and the presence of CD8 T cells and natural killer (NK) cells. These results support the mechanism of action of bexmarilimab, highlighting its ability to modulate the immune system effectively and address the underlying causes of MDS.
Industry Reactions and Future Directions
Dr. Juho Jalkanen, CEO of Faron Pharmaceuticals, expressed optimism regarding the results, stating, "The BEXMAB results continue to improve over time showing a remarkable 80% ORR in r/r MDS patients. The combination is well-tolerated and generates strong and durable reductions in cancer blasts. This solidifies bexmarilimab's leading position in myeloid cell reprogramming and MDS treatment. We are well-positioned to advance to the full Phase 2 efficacy readout and engage in further regulatory interactions as we refine the pathway toward BLA filing."
Dr. Mika Kontro, Associate Professor at the Helsinki University Hospital Comprehensive Cancer Center and Principal Investigator of the BEXMAB trial, also commented on the significance of the data, noting, "Addressing MDS remains a considerable challenge due to limited efficacy of current treatments, particularly for TP53-mutated and HMA-failed MDS. The data presented at ASH are highly promising and demonstrate meaningful improvements in patient outcomes."
The BEXMAB study, which spans multiple centers in Finland, the UK, and the US, is ongoing and continues to evaluate bexmarilimab's impact in patients with aggressive myeloid leukemias. With this positive data, Faron is moving forward with plans for a full Phase 2 efficacy readout and engaging in further development discussions to pursue regulatory filings and commercial opportunities.
