Publication of full Phase I BEXMAB study data in Lancet Haematology

Phase I study shows promising clinical activity and tolerability of bexmarilimab plus standard-of-care in high-risk and HMA-refractory MDS patients

Key findings:

Bexmarilimab in combination with azacitidine showed ORR of 100% and 89% in treatment-naïve and HMA-failed HR-MDS patients, respectively

Majority of treatment-emergent adverse events (TEAE) were mild to moderate, and only 6% were related to bexmarilimab

Treatment was well-tolerated, with no dose-limiting toxicities

Estimated median overall survival of 13.4 months in patients with HMA-failed MDS and 8.1 months in patients with r/r AML

Two HMA-failed MDS patients received a hematopoietic stem cell transplantation (HSCT) after treatment

Bone marrow immune biomarkers increased after treatment by nearly 3-fold versus baseline. An increase in biomarkers (HLA-DR molecules) seen in 67% of patients with HMA-failed MDS reinforces bexmarilimab’s mode of action.

TURKU, FINLAND – Faron Pharmaceuticals Ltd. (AIM: FARN, First North: FARON), a clinical-stage biopharmaceutical company focused on tackling cancers via novel immunotherapies, today announced the publication of the results of its Phase I BEXMAB study in the prestigious Lancet Haematology which reinforced the safety and efficacy of the treatment in patients with high-risk myelodysplastic syndrome (HR-MDS) and relapsed/refractory (r/r) acute myeloid leukemia (AML).

“Treating patients with high-risk MDS who don’t respond to current therapies remains a serious challenge. That’s why the promising results seen with bexmarilimab that show strong response rates and manageable side effects genuinereal hope for patients in urgent need of better treatment options,” says the lead investigator Dr. Mika Kontro, Associate Professor atUniversity of Helsinki and Helsinki University Hospital Comprehensive Cancer Center, Department of Hematology.

A total of 33 patients across six centres in the US and Finland were treated with increasing doses of bexmarilimab (1, 3, and 6 mg/kg once a week in a 28-day cycle) in combination with azacitidine (as per label). Out of these, five were patients with HR-MDS, nine were hypomethylating agents (HMA)-failed MDS patients, and 19 were relapsed/refractory AM (r/r AML) patients (unresponsive to earlier treatments). The combination therapy was effective with objective responses observed in 45% of patients across all the studied indications.

The majority of treatment-emergent adverse events (TEAE) were mild to moderate, and only 6% of them were related to bexmarilimab. The treatment was well-tolerated, and no dose-limiting toxicities were observed.

Among the patients studied in BEXMAB, the estimated median overall survival (mOS) was 8.1 months in patients with r/r AML, and 13.4 months in patients with HMA-failed MDS. Generally, in these relapsed or refractory patients’ remissions are rare and median overall survival is only 5-6 months. The mOS of 13.4 months for patients with HMA-failed MDS is notable and this indication was advanced into Phase 2. Moreover, two HMA-failed MDS patients received a hematopoietic stem cell transplantation (HSCT).

“These Phase I results with bexmarilimab are promising as patients lived longer than typically expected, and some even improved enough to receive a stem cell transplant. It offers new hope for those with high-risk MDS,” adds Senior Investigator Dr. Naval Daver, MD, Professor in the Department of Leukemia at The University of Texas MD Anderson Cancer Center.

Also, a reduction in bone marrow blast cells (immature cells) was seen in 64% of all patients across all doses of bexmarilimab.

The study also evaluated the immune response in the bone marrow and found that some biomarkers (human leukocyte antigen-DR isotype or HLA-DR molecules and CD4+ T cells) involved in the immune response increased by nearly three-fold versus baseline after treatment with bexmarilimab and azacitidine. Interestingly, an increase in HLA-DR molecules was seen even in 67% of patients with HMA-failed MDS indicating increased antigen presentation.

“The encouraging immune response observed even in patients unresponsive to standard treatments reinforces our belief in the unique mode of action of bexmarilimab. It gives us a strong reason and responsibility to stay committed to its continued development and exploration in future studies of both MDS and AML,” says Juho Jalkanen, Chief Executive Officer, Faron Pharmaceuticals.

The article is freely accessible until 17 July 2025 via this link.

About bexmarilimab