Bexmarilimab Plus Vidaza Elicit Responses in Myelodysplastic Syndromes

Among both frontline and relapsed/refractory higher-risk myelodysplastic syndrome, treatment with bexmarilimab and Vidaza elicited high overall response rates.

Treatment with bexmarilimab and Vidaza (azacitidine) elicited high overall response rates in both frontline and relapsed/refractory higher-risk myelodysplastic syndrome, according to topline results from the BEXMAB trial, which were shared in a press release from Faron Pharmaceuticals Ltd.

These data showed that, with the high overall response rates in patients, the phase 2 BEXMAB trial met its primary end point in treatment-resistant higher-risk myelodysplastic syndrome. Moreover, the topline data confirmed earlier findings in both the frontline and relapsed/refractory patient population. The combination also remained well tolerated without any dose-limiting toxicities, according to the press release; this is important because this patient population does not currently have any effective treatment options for their disease.

“This is one of the strongest data set ever seen in an all-comer population of treatment resistant higher-risk myelodysplastic syndrome”, Dr. Juho Jalkanen, CEO of Faron Pharmaceuticals, said in the press release. “There is a significant unmet need in the treatment of higher-risk myelodysplastic syndrome, as drug development [and] macrophage re-programming has proven to be extremely challenging, with a lot of previous failures. What really makes bexmarilimab stand out in this field is its good safety profile combined with very high efficacy especially in last line higher-risk myelodysplastic syndrome. This gives us conviction that bexmarilimab is the long-awaited drug to overcome treatment resistance."

This is the first full analysis from the ongoing phase 2 clinical trial and generated an objective response rate of 63% in patients, meaning these individuals had their cancer shrink or disappear after treatment. The average length of survival is consistent with what was seen in previously reported data. 

Moreover, among patients who had not received prior treatment for high-risk myelodysplastic syndromes, there was a 76% objective response rate. Since many participants are still in the early stages of treatment, this means that these responses could continue to improve over time. The press release goes on to share that as more information becomes available, the results may change slightly.

Notably, the Company says that the full data set generated from this trial has been submitted for review to the 2025 American Society of Clinical Oncology (ASCO) Annual Meeting. Moreover, Faron Pharmaceuticals is planning to launch a phase 3 trial of bexmarilimab and Vidaza in patients with relapsed/refractory higher-risk myelodysplastic syndrome, pending feedback from the U.S Food and Drug Administration (FDA) following their End of Phase 2 meeting.

More on Myelodysplastic Syndromes and Their Treatment

Myelodysplastic syndromes are a group of rare and serious blood cancers that interfere with the body’s ability to produce healthy blood cells. Over time, these conditions can worsen and lead to reduced survival. Unfortunately, treatment options remain limited — especially for patients who are not eligible for a stem cell transplant. Without this type of transplant, long-term survival is often poor.

The emerging data from the BEXMAB clinical trial may bring a new treatment option to patients and their families, according to the press release, offering improved quality of life. Many individuals living with myelodysplastic syndromes experience debilitating symptoms that affect their ability to manage daily tasks and enjoy life. In both phase 1 and 2 trials, bexmarilimab and Vidaza was generally well tolerated, which may help improve patients’ overall well-being and day-to-day comfort.

The treatment also addresses unmet needs within the landscape. Progress in developing new myelodysplastic syndrome therapies has been slow. Many of the existing treatments work for only a limited number of patients, and resistance often develops over time. The phase 2 clinical trial data suggest that a new option may be on the way.

Bexmarilimab is a type of immunotherapy that targets a protein called CLEVER-1. By blocking this protein, the treatment helps the immune system detect and attack cancer cells more effectively, which could potentially stop tumor growth and improve patient outcomes.

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