TURKU, FINLAND – Faron Pharmaceuticals Ltd. (AIM: FARN, First North: FARON), a clinical-stage biopharmaceutical company focused on tackling cancers through novel immunological pathways, today announced the acceptance of two studies involving its lead candidate, bexmarilimab, for presentation at the 19th IUIS International Congress of Immunology in Vienna, Austria, from August 17-22, 2025.

The oral presentation will feature new translational data from the Phase 1/2 BEXMAB clinical study in myelodysplastic syndrome (MDS). The second study, accepted as a poster, details the identification of secreted Clever-1 (sClever-1) as a key driver of immune suppression and immunotherapy resistance. Together, these presentations underscore the growing body of evidence supporting bexmarilimab’s mechanism of action.  

The oral presentation, entitled ‘Dual Mechanisms of Clever-1 Inhibition with Bexmarilimab Plus Azacitidine in Myelodysplastic Syndrome: Translational Insights from the Phase 1/2 BEXMAB Study,’ will be presented by Faron’s Chief Scientific Officer, Dr. Maija Hollmén. The BEXMAB study is a Phase I/II trial investigating bexmarilimab with standard of care in patients with aggressive hematological malignancies. The study provides insights into how directly targeting the Clever-1 receptor, which is highly expressed in acute myeloid leukemia (AML) and MDS, ignite an immune response, and make current treatments more effective.  

Dr. Maija Hollmén, senior author of both studies, MediCity Research Laboratory and InFLAMES Flagship, University of Turku, Turku, Finland, said, “The BEXMAB data provides translational insights into bexmarilimab’s dual impact in a clinical setting, while our sClever-1 findings elucidate a fundamental mechanism of systemic immune suppression. Showing that bexmarilimab can block sClever-1 release suggests our drug candidate fights cancer on two fronts: reprogramming macrophages locally and preventing this inhibitory molecule’s systemic effects. We look forward to sharing these insights with the global scientific community.”  

The research accepted for a poster presentation and also published in the peer-reviewed journal Theranostics, ‘Secreted Clever-1 modulates T cell responses and impacts cancer immunotherapy efficacy,’ reveals that a soluble form of Clever-1 is significantly enriched in the blood of cancer patients. This circulating sClever-1 directly binds to and neutralizes activated T cells, impairing their ability to fight cancer. High levels of sClever-1 are associated with resistance to anti-PD-1 therapies. Importantly, the research demonstrates that bexmarilimab treatment can inhibit the release of sClever-1, highlighting a key and previously undescribed therapeutic mechanism.  

“Having two distinct and complementary datasets accepted at this prestigious congress is very encouraging and a testament to the depth of our research,” said Dr. Petri Bono, Chief Medical Officer of Faron. “High-risk MDS is associated with poor outcomes and therapy resistance. The BEXMAB study data demonstrates a dual mechanism of action in MDS, while sClever-1 research redraws a part of the cancer immunology map. These data provide a strong scientific rationale for why targeting Clever-1 is the right approach.”  

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.

TURKU, FINLAND – Faron Pharmaceuticals Ltd. (AIM: FARN, First North: FARON), a clinical-stage biopharmaceutical company focused on tackling cancers through novel immunotherapies, announced the publication of a new study in the peer-reviewed journal Theranostics. The research identified a secreted, truncated form of the Clever-1 receptor (sClever-1) as a key immunosuppressive mediator in cancer that impaired T-cell responses and was associated with resistance to anti-programmed cell death protein -1 (PD-1) immunotherapies.

The study, supported by Faron Pharmaceuticals and grants from leading international funding bodies, including Cancer Research UK (CRUK), the NIHR Birmingham Biomedical Research Centre, the Research Council of Finland, and the Sigrid Jusélius Foundation, provided significant new insights into the biology of Clever-1, the target of Faron’s wholly owned, first-in-class immunotherapy asset, bexmarilimab. Clever-1 is an immunosuppressive receptor found on macrophages leading to tumor growth and metastases (i.e. helps cancer evade the immune system). 

In this study, researchers analyzed plasma samples from breast cancer patients (n=139) and bexmarilimab-treated clinical trial participants (n=193) as well as healthy donors and discovered that a secreted form of Clever-1 or sClever-1 was significantly enriched in the plasma of cancer patients compared to healthy individuals.

“Our research unveils a previously unrecognized mechanism of immune suppression in cancer, where a secreted form of Clever-1 actively hinders T-cell responses,” said Maija Hollmén, senior author of the study, MediCity Research Laboratory and InFLAMES Flagship, University of Turku, Turku, Finland. “We have shown that sClever-1 operates independently of the cellular receptor and contributes to an immunosuppressive environment. This discovery not only deepens our understanding of cancer immunology but also strongly validates Clever-1 as a critical therapeutic target to overcome immune evasion.”

sClever-1, released by macrophages and endothelial cells, was found to bind to activated T cells, impairing their signaling and promoting the differentiation of suppressive T cells, contributing to tumor immune evasion. Moreover, high sClever-1 levels were associated with resistance to anti-PD-1 therapy.

Importantly, the study demonstrated that bexmarilimab treatment significantly reduced the release of sClever-1 in cancer patients. This reduction in circulating sClever-1 correlated with decreased engagement on T cells, providing a direct mechanistic link to the peripheral T-cell activation previously observed in bexmarilimab-treated patients.  

The findings also suggest that high levels of sClever-1 are associated with resistance to anti-PD-1 checkpoint inhibitors, identifying sClever-1 as a potential biomarker to guide immunotherapy strategies. In patient-derived tumor explants, the addition of a recombinant sClever-1 protein led to a significant increase in the secretion of TGF-β, a key immunosuppressive cytokine.  

“These unique findings further solidify our conviction in bexmarilimab’s unique mechanism of action,” said Petri Bono, Faron’s Chief Medical Officer. “The ability of bexmarilimab to reduce systemic levels of the immunosuppressive sClever-1 provides a clear rationale for its potential to overcome immunotherapy resistance. This adds to the growing body of evidence supporting bexmarilimab’s development as a potential cornerstone of immunotherapy for patients with aggressive cancers. This provides also a very intriguing basis of using soluble Clever-1 to inactivate T-cells in conditions where they are attacking our own body, i.e. in a wide range of auto-immune and inflammatory conditions”

Link to the original article.

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.

• 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.

Dr. 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.

Dr. 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:

Presentation title: Efficacy of Macrophage Checkpoint Clever-1 Inhibition with bexmarilimab plus Azacitidine in Myelodysplastic Syndrome: Results from the Ph1/2 BEXMAB Study

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.

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.

XXXX

• 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.

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.

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.

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

For more information, please contact:

IR Partners, Finland (media)
Riina Tuominen
+358 44 313 5005
riina.tuominen@irpartners.fi

Kare Laukkanen
+358 50 553 9535 / +44 7 469 766 223
kare.laukkanen@irpartners.fi

FINN Partners, US (media)
Alyssa Paldo
+1 847 791-8085
alyssa.paldo@finnpartners.com

Cairn Financial Advisers LLP, Nominated Adviser and Broker
Sandy Jamieson, Jo Turner
Phone: +44 (0) 207 213 0880

Sisu Partners Oy, Certified Adviser on Nasdaq First North
Juha Karttunen
Phone: +358 (0)40 555 4727
Jukka Järvelä
Phone: +358 (0)50 553 8990

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 .