• Encouraging survival; Median overall survival 13.4 months in 20 high and very high-risk r/r MDS patients treated with bexmarilimab + azacitidine; 4 patients successfully bridged to hematopoietic stem cell transplant
• 55% patients showed ≥50% reduction in bone marrow; 21% of transfusion-dependent patients became transfusion-independent
• Faron will be hosting a virtual webinar to discuss the full analysis of r/r MDS as well as new frontline HR MDS patient data on Monday, 2 June 2025. To register for the event visit: BEXMAB Phase II study results

Turku, Finland – Faron Pharmaceuticals Ltd. (AIM: FARN, First North: FARON), a clinical-stage biopharmaceutical company advancing next-generation immunotherapies, presented updated data from its ongoing BEXMAB Phase 1/2 trial at plenary oral session at the 18th International Congress on Myelodysplastic Syndromes (MDS 2025) on May 10, 2025, in Rotterdam, Netherlands.

The presentation, led by Dr. Amer Zeidan, MBBS, MHS, focused on the preliminary efficacy, safety, and immune biomarker data from 20 first patients with relapsed or refractory high-risk myelodysplastic syndromes (r/r MDS) who had failed prior hypomethylating agent (HMA) therapies. 90% of the patients were very high/high risk at baseline and 50% had tp53 mutations. These patients represent a population with extremely limited treatment options and aggressive and difficult-to-treat disease. The BEXMAB Phase 1 & 2 MDS patients with prior HMA failure experienced an estimated median overall survival (mOS) of approximately 13.4 months, compared to the 5-6 months that would typically be expected under standard of care historically.

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 HMA. By blocking Clever-1, bexmarilimab reprograms macrophages in the bone marrow, enhancing anti-tumor immunity. The combination was well tolerated, with no Grade 3–5 adverse events attributed to bexmarilimab and no treatment discontinuations due to related toxicity.

Clinically, the combination of bexmarilimab and azacitidine demonstrated encouraging efficacy in patients with r/r MDS. A reduction of 50% or more in bone marrow blast counts was observed in 55% of patients, indicating clinically significant disease control. Additionally, 21% of patients who were transfusion dependent at baseline achieved transfusion independence, reflecting a potential improvement in quality of life and a reduced need for supportive care. Importantly, four patients were able to proceed to hematopoietic stem cell transplantation, the only potentially curative treatment, suggesting that this regimen may serve as an effective bridge to transplant.

Dr. Amer Zeidan, MBBS, MHS, Professor of Medicine and Chief of Hematologic Malignancies at Yale School of Medicine and Yale Cancer Center, and the lead presenter said, “While we have a small number of patients treated on trial to date, I am encouraged by the safety and efficacy data we are observing to date with the the combination of bexmarilimab and azacitidine in patients with higher risk MDS, especially after HMA failure. If these trends continue to hold, they would provide strong rationale for pursuing a registrational approach with a randomized phase 3 trial, and could potentially offer a new therapeutic option for this difficult-to-treat patient population.”

Biomarker analysis revealed that treatment with bexmarilimab plus azacitidine was associated with a rise in immune activation markers in the bone marrow, suggesting that Clever-1 blockade enhances immune activation and engagement. Additionally, despite all patients being Clever-1 positive responding patients had higher pre-treatment Clever-1 expression on monocytes/macrophages than non-responding patients confirming the importance of Clever-1 as a target and the mode of action of bexmarilimab in the treatment of MDS patients.

Juho Jalkanen, MD, PhD, Chief Executive Officer of Faron Pharmaceuticals, said, “We are deeply encouraged by these results, which show that bexmarilimab has the potential to significantly alter the treatment landscape for patients with relapsed or refractory MDS that have no further treatment options. The combination's favorable safety profile and promising clinical activity in last line MDS strengthen our belief in the mechanism of action and therapeutic promise of Clever-1 inhibition. These results also support the company’s plans toward initiating a randomized study in frontline HR-MDS patients as suggested by the FDA earlier. New frontline data together with the fully enrolled r/r MDS Phase 2 will also be presented soon at ASCO 2025 and followed up with a webcast hosted by the company.”

Details of the presentation

• Title: Preliminary efficacy of bexmarilimab with azacitidine in relapsed or refractory MDS in BEXMAB Ph1/2 study
• Session: Plenary Session 08: Treatment High Risk
• Presenter: Dr. Amer Zeidan, MBBS, MHS
• Date & Time: May 10, 2025 | 09:50 – 11:20 CEST
• Location: Rotterdam, Netherlands
• Abstract Number: 225

Faron Pharmaceuticals remains committed to accelerating the clinical development of bexmarilimab for patients with high-risk myeloid malignancies.

Faron will be hosting a virtual webinar to discuss the full analysis of r/r MDS as well as new frontline HR MDS patient data on Monday, 2 June 2025 at 4pm EEST/9am ET.

To register for the event visit: BEXMAB Phase II study results

Amer Zeidan consulted and received honoraria from Faron. The views expressed represent his own and do not necessarily reflect those of his employer.

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About BEXMAB
The BEXMAB study is an open-label Phase I/II clinical trial investigating bexmarilimab in combination with standard of care (SoC) in the aggressive hematological malignancies of acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS). The primary objective is to determine the safety and tolerability of bexmarilimab in combination with SoC (azacitidine) treatment. Directly targeting Clever-1 could limit the replication capacity of cancer cells, increase antigen presentation, ignite an immune response, and allow current treatments to be more effective. Clever-1 is highly expressed in both AML and MDS and associated with therapy resistance, limited T cell activation and poor outcomes.

About bexmarilimab
Bexmarilimab is Faron’s wholly owned, investigational immunotherapy designed to overcome resistance to existing treatments and optimize clinical outcomes, by targeting myeloid cell function and igniting the immune system. Bexmarilimab binds to Clever-1, an immunosuppressive receptor found on macrophages leading to tumor growth and metastases (i.e. helps cancer evade the immune system). By targeting the Clever-1 receptor on macrophages, bexmarilimab alters the tumor microenvironment, reprogramming macrophages from an immunosuppressive (M2) state to an immunostimulatory (M1) one, upregulating interferon production and priming the immune system to attack tumors and sensitizing cancer cells to standard of care.

About Faron Pharmaceuticals Ltd
Faron (AIM: FARN, First North: FARON) is a global, clinical-stage biopharmaceutical company, focused on tackling cancers via novel immunotherapies. Its mission is to bring the promise of immunotherapy to a broader population by uncovering novel ways to control and harness the power of the immune system. The Company’s lead asset is bexmarilimab, a novel anti-Clever-1 humanized antibody, with the potential to remove immunosuppression of cancers through reprogramming myeloid cell function. Bexmarilimab is being investigated in Phase I/II clinical trials as a potential therapy for patients with hematological cancers in combination with other standard treatments. Further information is available at www.faron.com.