Faron Pharmaceuticals Presents Phase II Data from BEXMAB Study at ASCO 2025

TURKU, FI / ACCESS Newswire / June 2, 2025 / Faron Pharmaceuticals (HEL:FARON)(LSE:FARN) - Favorable response rates and survival data in high/very high-risk frontline and r/r MDS support advancement to Phase III trial in frontline MDS, per previous FDA guidance

• Median overall survival of 13.4 months in 32 r/r MDS patients treated with bexmarilimab + azacitidine

• ORR of 63% and 72% observed in patients with r/r MDS (n=32) and frontline MDS patients (n=18), respectively 56% composite CR (cCR) rate in frontline MDS per the new IWG 2023 criteria

• 72% of frontline or treatment-naïve MDS patients showed >50% reduction and 56% showed 100% reduction of bone marrow blasts with the combination

• will be hosting a virtual webinar to discuss the full analysis of r/r MDS as well as frontline HR MDS patient data on Today, June 2 at 4pm EEST/9am ET. 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 phase II data from its ongoing BEXMAB study evaluating bexmarilimab in high-risk myelodysplastic syndromes (HR-MDS) as a part of a Rapid Oral Abstract Session at the 2025 American Society of Clinical Oncology (ASCO) Annual Meeting, taking place from 30 May to 3 June 2025, in Chicago, Illinois, USA.

The oral presentation was led by Dr. Naval Daver, MD, Professor of Medicine and Director of Leukemia Research Alliance Program, Department of Leukemia, MD Anderson Cancer Center . It highlighted the efficacy and tolerability of bexmarilimab , Faron's novel humanized anti-Clever-1 antibody, when used in combination with standard-of-care, azacitidine, treatment in patients with both treatment naïve (frontline) (n=21) and relapsed/refractory (r/r) MDS (n=32). More than 66% of the former and 80% of latter group were very high/high risk at baseline. The patients who have failed treatment with hypomethylating agents (HMA) or r/r MDS have very limited treatment choices and difficult-to-treat disease. The phase II BEXMAB data demonstrated an estimated median overall survival (mOS) of approximately 13.4 months in patients with r/r MDS, compared to the 5-6 months that would typically be expected under standard of care.

Dr. Naval Daver, MD, said, "This data with bexmarilimab plus azacitidine is highly encouraging, especially in the r/r MDS setting where treatment options are limited and outcomes remain poor. Seeing promising survival outcomes and overall response rates in patients after HMA failure highlights the potential of this approach. The observed immune activation in the bone marrow further reinforces the mode of action of bexmarilimab and offers hope for bridging patients towards a bone marrow transplant, which could lead to a possible cure."

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.

Results support path to Phase III trial

The combination of bexmarilimab and azacitidine demonstrated significant efficacy in patients with r/r MDS with a high objective response rate (ORR) of 63%. For the past 20 years re-challenging with an HMA after HMA failure would result in an ORR of 0-15% per the IWG 2006 criteria. Similarly, an ORR of 72% was observed in patients with treatment naïve HR-MDS per the International Working Group (IWG) 2006 criteria. As per the new IWG 2023 criteria, the composite CR (cCR) rate in frontline HR-MDS was 56%, while historically up to 20-25% can be expected with azacitidine alone.

This supports moving on to a confirmatory registrational frontline HR-MDS trial comparing bexmarilimab + azacitidine against placebo + azacitidine according to previously received guidance from the FDA. This and the proposed primary endpoint cCR which is strongly linked to survival will be discussed in an upcoming end-of-phase II meeting with the Agency.

Data reaffirms efficacy and immune activation with CLEVER-1 inhibition

44% of r/r HR MDS and 72% of the frontline MDS patients treated with bexmarilimab and azacitidine showed >50% reduction of bone marrow blasts. Additionally, 56% of the frontline patients showed 100% reduction of bone marrow blasts. The combination also increased the markers of immune activation and hematological improvement in several cell lines in the bone marrow, such red blood cells, white blood cells and platelets. The treatment was also well-tolerated in this otherwise frail population with severe anemia and infections. Based on the safety and efficacy data of the trial, the recommended phase III bexmarilimab dose is 3 mg/kg.

Dr. Mika Kontro, Associate Professor at University of Helsinki and Helsinki University Hospital Comprehensive Cancer Center, Department of Hematology said, "For patients living with high-risk MDS, especially those who have exhausted current treatments, any therapy that offers patients more time and improves hematological parameters is a welcomed development. The continued progress of therapies like bexmarilimab brings much-needed optimism to patients and families navigating this difficult disease."

Juho Jalkanen, MD, PhD, Chief Executive Officer of Faron Pharmaceuticals, said, " These amazing results reinforce our confidence in bexmarilimab's potential to transform care for patients with high-risk MDS. This data not only strengthens the rationale for advancing to a randomized Phase III trial in frontline HR-MDS, in line with FDA guidance, but also marks a significant step forward in our clinical development strategy towards approval."

Details of the presentation

• Presentation title: Efficacy of macrophage checkpoint Clever-1 inhibition with bexmarilimab plus azacitidine in myelodysplastic syndrome: Results from the ph1/2 BEXMAB study.

• Session type and title: Rapid Oral Abstract - Hematologic Malignancies - Leukemia, Myelodysplastic Syndromes, and Allotransplant

• Session date: 30 May 2025

• Time: 1:00 PM - 2:30 PM CDT

• Abstract no: 6513

• Presenter: Dr. Naval Daver, MD | Department of Leukemia, The University of Texas MD Anderson Cancer Center

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

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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 .

SOURCE: Faron Pharmaceuticals