Key highlights:

• Median overall survival (mOS) with bexmarilimab and azacitidine in relapsed/refractory (r/r) HR-MDS patients reached 14.5 months (increased from 13.4 months observed earlier), a significant improvement in a population with historically poor survival of only 5-6 months
• In treatment-naïve patients with HR-MDS and TP53 mutation, the combination achieved a remarkable 70% complete remission (CR) rate
• Deep responses allowed 50% of treatment-naïve TP53-mutated patients and 21% of r/r TP53-mutated patients to proceed to stem cell transplant (SCT)
• For the first time, data shows 57% of frontline patients who were transfusion-dependent at baseline achieved transfusion independence, confirming restoration of healthy bone marrow function

TURKU, FINLAND – Faron Pharmaceuticals Ltd. (AIM: FARN, First North: FARON), a clinical-stage biopharmaceutical company, today announced updated full data from its Phase 1/2 BEXMAB study, presented at the American Society of Hematology (ASH) 2025 Annual Meeting (oral and poster presentation). The data highlights significant improvement in survival outcome and new findings on hematopoietic recovery in patients with higher-risk Myelodysplastic Syndromes (HR-MDS) treated with bexmarilimab in combination with standard-of-care azacitidine.

Deep and durable response across HR-MDS subgroups

The BEXMAB study continues to show deep and durable responses across HR-MDS subgroups, particularly in patients with TP53 mutations, a genetic profile typically associated with resistance to standard therapies.

Dr. Amer Zeidan, MBBS, MHS, Professor of Internal Medicine at Yale University and chief of Hematologic Malignancies at Yale Cancer Center and Smilow Cancer Hospital said, “For patients with HR-MDS, particularly those who have exhausted standard treatments or harbor poor risk mutations like TP53, the prognosis has historically been very challenging. Observing a median overall survival of 14.5 months in the relapsed setting, together with the notably high complete remission rates in frontline TP53-mutated disease—even with the small sample size and limited follow-up—represents a highly encouraging signal of efficacy. Also, half of the frontline TP53-mutated patients were able to proceed to a stem cell transplantation, suggesting this combination could offer a vital bridge to potentially curative therapy for patients who often have limited options. We remain excited about this combination as the data continue to mature over time, and we look forward to initiating the randomized registrational phase 2/3 trial in the near future.”

In treatment-naïve HR-MDS, the bexmarilimab and azacitidine combination reported an overall response rate (ORR) of 85% and a complete remission (CR) rate of 45%. Efficacy was exceptionally strong in patients with TP53 mutations, who achieved an ORR of 80% and a CR rate of 70% (50% of this TP53 cohort proceeded to stem cell transplant). Overall, 55% of treatment-naïve patients showed complete clearance of bone marrow blasts, and 35% proceeded to stem cell transplant (SCT).

In patients with relapsed/refractory (r/r) HR-MDS, the combination achieved an ORR of 64%, with a median overall survival (mOS) of 14.5 months. When stratified by mutation status, mOS for TP53-mutated patients was 7.5 months (21% of r/r mTP53 patients proceeded to SCT), while it was not yet reached for wild-type TP53 patients.

Bexmarilimab supports recovery of the bone marrow

In treatment-naïve HR-MDS, 57% of patients who were transfusion-dependent (TD) at baseline successfully achieved transfusion independence (TI). Across the entire HR-MDS BEXMAB trial population, 23% of dependent patients converted to independence. Maintenance of TI was robust, with 65% of patients who entered the study transfusion-independent remaining so throughout treatment. Analysis of the bone marrow samples confirmed that the treatment increases erythrocyte, platelet, and leukocyte progenitors, providing biological validation for the clinical improvements in blood counts.

Dr. Petri Bono, Chief Medical Officer of Faron, added, “The new data on transfusion independence is clinically very meaningful. For MDS patients, freedom from regular transfusions is a major determinant of quality of life. By demonstrating that we can convert over half of frontline transfusion dependent patients to independence, while simultaneously driving deep molecular remissions in high-risk genetic subgroups, we are confirming that bexmarilimab acts on cancer cells and actively rehabilitates the bone marrow environment, which is a key differentiator compared to classical toxic drugs aiming to just kill cancer cells.”

The combination continues to demonstrate an excellent safety profile. There were no Grade 5 bexmarilimab-related adverse events across the HR-MDS cohort. The encouraging safety profile and clear efficacy signals support bexmarilimab’s advancement into a randomized Phase 3 registrational trial to address the urgent unmet need in patients with HR-MDS.

The details of the ASH oral and poster presentation are as follows:

Oral presentation title: Efficacy, molecular and translational analysis of TP53-mutated HR-MDS with bexmarilimab and azacitidine: Updated results from the BEXMAB Ph1/2 study

Presented by: Dr. Amer Zeidan

Room: OCCC - Valencia Room W415BC

Session Name: 637. Myelodysplastic Syndromes: Clinical and Epidemiological: Treatment Advances in Higher Risk Myelodysplastic Syndromes

Session date and time: December 6, 2025, 2:15-2:30 PM

Abstract no: 7411

Poster presentation title: Transfusion independence, hematological improvement and associated safety outcomes with bexmarilimab plus azacitidine in HR MDS: Results of the bexmab Ph1/2 study

Presented by: Dr. Mika Kontro

Room: OCCC - West Halls B3-B4

Session Name: 637. Myelodysplastic Syndromes: Clinical and Epidemiological: Poster III

Session date and time: December 8, 2025, 6:00-8:00 PM

Abstract no: 7422

For more information, please contact:

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.

Disclosure: Dr. Amer Zeidan has received consulting fees from Faron. The views expressed are his own and do not necessarily represent those of his employer.