Encouraging Phase 1 BEXMAB Study Results in High-Risk MDS and r/r AML Patients

Recent findings from the BEXMAB clinical trial have demonstrated promising results for patients with high-risk myelodysplastic syndrome (MDS) who have previously failed hypomethylating agent (HMA) therapy. The study has observed an increased number of patients responding positively to treatment, along with a notable persistence of remission.

In the initial phase of the trial, which focused on Phase 1 high-risk HMA-failed MDS patients, 80% of them were found to be alive after a follow-up period of eight months. Although the median overall survival (mOS) has not been precisely determined yet, the survival rates observed thus far are highly encouraging compared to contemporary treatments. The trial has also enrolled three additional HMA-failed high-risk MDS patients, resulting in a total of seven out of eight showing a response to the treatment, yielding an overall response rate of 87.5%.

Faron Pharmaceuticals, the company behind the BEXMAB trial, is set to host a virtual seminar to delve deeper into these findings on the 19th of March. The BEXMAB trial, which has transitioned into Phase 2, is examining the effects of bexmarilimab in combination with standard care for aggressive hematological malignancies, including AML and MDS. The primary goal is to assess the safety and tolerability of bexmarilimab when combined with standard treatments.

Previous results from the BEXMAB trial showed a high overall response rate among the high-risk HMA-failed MDS patients, a group that currently lacks approved treatments. Most of these initial patients have been on a treatment regimen combining bexmarilimab with azacitidine for over six months, with only one patient experiencing a progression to acute myeloid leukemia (AML). The estimated mOS for these patients is expected to be significantly higher than the typical survival rates associated with the standard of care.

The addition of three new patients to the trial has further supported the high response rate observed initially, with two out of three new patients responding positively. One patient, however, had to withdraw early due to a serious adverse event unrelated to the study treatment. The responses among the eight patients enrolled include one complete response, three marrow complete remissions, one partial response, two hematological improvements, and one stable disease case that withdrew early.

Mika Kontro, a lead investigator of the BEXMAB trial, expressed optimism regarding the trial's outcomes, highlighting the potential for bexmarilimab to significantly enhance patient outcomes, especially considering the quality of life improvements observed even beyond the 12-month mark post-treatment initiation.

Dr. Markku Jalkanen, CEO of Faron, echoed this sentiment, emphasizing the remarkable nature of the data and the potential for a positive registrational trial against contemporary comparators based on survival as the final endpoint. The company is eagerly awaiting the completion of the Phase 2 part of the BEXMAB study to present the data to regulatory authorities.

For the frontline high-risk MDS patients previously reported, the mOS has not been reached. In another patient cohort with relapsed/refractory AML, the mOS is currently estimated to exceed eight months, which is a significant improvement over the historical mOS of around six months for such patients.

Bexmarilimab, the investigational immunotherapy at the heart of the BEXMAB study, is designed to overcome resistance to existing treatments by targeting myeloid cell function and stimulating the immune system. It works by binding to the Clever-1 receptor, altering the tumor microenvironment and enhancing the body's immune response to cancer cells.

Faron Pharmaceuticals is a clinical-stage biopharmaceutical company dedicated to developing novel immunotherapies to combat cancer, with bexmarilimab as its lead asset. The company aims to extend the reach of immunotherapy to a wider patient population by exploring innovative approaches to immune system manipulation.