Faron Pharmaceuticals Ltd. provided first data from patients treated during the Phase 2 part of the ongoing BEXMAB trial in myelodysplastic syndrome (MDS) patients that have failed a hypomethylating agent (HMA), also known as relapsed/refractory MDS (r/r MDS). There are limited viable treatment options for r/r MDS and the mOS for these patients is only 5.6 months historically. The BEXMAB Phase 1 results have already indicated a high overall response rate (ORR) of 87.5% (7/8) amongst HMA-failed MDS patients treated with a combination of bexmarilimab + azacitidine.

There are now a total of 14 HMA-failed MDS patients treated in both Phase 1 & 2 with this novel combination. The treatment has been well tolerated, without any dose-limiting toxicity. The ORR in this otherwise untreatable population is 79% (11/14).

The current true remission rate is 64% (9/14). Similar size patient cohorts treated with existing alternatives have reported 0-20% ORR, without deep and durable remissions. The best responses for these 14 patients are as follows: 1 complete response (CR), 7 marrow complete remissions (mCR), 1 partial response (PR), 2 hematological improvements, 2 stable diseases (SD) and 1 progressive disease (PD).

Two patients have moved on to receive bone marrow transplantation for a possibility of curative treatment. This is seldom seen in this population because patients usually cannot be brought to remission. For Phase 1 patients with adequate follow-up available the estimated mOS is currently 13.4 months, but still subject to change.