Faron Pharmaceuticals presents updated Phase II data from BEXMAB Study at EHA 2025

Strong efficacy and survival outcomes with bexmarilimab in high-risk MDS support Phase III advancement

Median overall survival (mOS) of 13.4 months in r/r HR-MDS patients (n=32) treated with bexmarilimab + azacitidine; mOS of 9.3 months in TP53 mutated patients (n=13)

ORR of 85% and cCR of 50% in patients with frontline MDS (n=20)

Deep bone marrow responses demonstrated at all dose levels in frontline MDS and based on the safety and efficacy data of the trial, the recommended phase III bexmarilimab dose is 3 mg/kg

Turku, Finland – Faron Pharmaceuticals Ltd. (AIM: FARN, First North: FARON), a clinical-stage biopharmaceutical company advancing next-generation immunotherapies, presents detailed phase II data from its ongoing BEXMAB study evaluating bexmarilimab in high-risk myelodysplastic syndromes (HR-MDS) as an oral presentation at the 30th European Hematology Association’s (EHA) Congress, taking place in Milan from 12–15 June 2025.

The presentation will be led by Dr. Mika Kontro, Associate Professor at University of Helsinki and Helsinki University Hospital Comprehensive Cancer Center, Department of Hematology, and will contain detailed results from the study of bexmarilimab, Faron’s investigational anti-Clever-1 antibody, in combination with azacitidine for patients with relapsed or refractory (r/r) and frontline or treatment-naïve HR-MDS. These results expand upon those presented at ASCO 2025 and reaffirm the efficacy and safety of bexmarilimab in this difficult-to-treat population.

Strong efficacy data in both r/r HR-MDS and frontline MDS, support advancement into phase III

The BEXMAB study evaluated bexmarilimab (1, 3, or 6 mg/kg weekly in 28-day cycles), a first-in-class monoclonal antibody targeting the Clever-1 receptor, in combination with azacitidine, a standard-of-care hypomethylating agent (HMA). By blocking Clever-1, bexmarilimab reprograms macrophages in the bone marrow, enhancing anti-tumor immunity.

With 80% of patients with r/r HR-MDS (n=32) falling in the very high/high risk category at baseline, the phase II BEXMAB data demonstrated an estimated median overall survival (mOS) of approximately 13.4 months, significantly surpassing historical outcomes of 5-6 months expected under standard of care. A mOS of 9.3 months was observed in patients with the aggressive mTP53 mutation (n=13). Five patients in the r/r HR-MDS group proceeded to stem cell transplant (SCT), the only curative option. Of the non-mTP53 mutated patients mOS has not yet been reached and 15 out of 19 patients are still alive.

• Mika Kontro, MD, PhD, also the principal investigator of the BEXMAB study, said, “The consistent and durable responses observed with bexmarilimab are notable in this patient population. The survival data with bexmarilimab are encouraging for r/r HR-MDS patients, who have limited treatment options after failing HMA therapy.”

In an updated analysis compared to the ASCO data, patients with frontline MDS (n=20; 45% with the TP53 mutation), the combination of bexmarilimab with azacitidine demonstrated an ORR of 85% and a cCR of 50%. In those with TP53, the ORR and cCR were 78% and 44%, respectively. In this group too, 7(35%) patients proceeded to SCT (n=5) or are in planning for transplant (n=2). Though bone marrow responses were observed across all dose levels in these patients, the 3 mg/kg appeared to be more favourable at this early stage, indicating the use of this dose for further trials.

• Petri Bono, Chief Medical Officer of Faron, said, “Achieving this milestone underscores our commitment to addressing the urgent needs of MDS patients. These results support ongoing positive and pivotal discussions with regulatory authorities. We are dedicated to advancing bexmarilimab through clinical development into phase III, with the goal of offering new hope to patients suffering from HR-MDS.”

Favourable immune activation with 3 mg/kg bexmarilimab dose

According to new data to be presented at EHA, bexmarilimab showed CLEVER-1 target engagement in both blood and bone marrow across all doses with no indication of accumulation with repeated dosing. Favourable activation of both the adaptive and innate immune system indicators was seen in r/r HR-MDS and frontline MDS with 3 mg/kg bexmarilimab and azacitidine. The combination was well-tolerated in all patients with HR-MDS.

The details of the oral presentation are as follows:

Session: Oral presentation

Presenter: Dr. Mika Kontro, MD, PhD

Date and Time: 15 June 2025 11:00 – 12:15 CEST

Location: Milan, Italy

Abstract no: S178

Faron Pharmaceuticals remains committed to accelerating the clinical development of bexmarilimab for patients with high-risk myeloid malignancies and anticipates initiating preparations for a Phase III registrational trial in the second half of 2025, following discussions with the FDA.

About bexmarilimab

XXXX