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:

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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 .

Study highlights:

• Ex vivo treatment of AML and MDS bone marrow samples with bexmarilimab led to increased antigen-presenting human leukocyte antigen DR isotype (HLA-DR) expression, indicating improved antigen presentation capacity.
• Bexmarilimab, when combined with azacitidine or venetoclax, enhanced HLA-DR expression and in many samples, reduced the viability of leukemic blasts, particularly in venetoclax-resistant samples.
• The triple combination of bexmarilimab + azacitidine + venetoclax showed greater anti-leukemic effect in resistant AML cell lines than standard treatments alone.

Turku, Finland – Faron Pharmaceuticals Ltd. (AIM: FARN, First North: FARON), a clinical-stage biopharmaceutical company developing novel immunotherapies, today announced the publication of a new peer-reviewed preclinical study in Scientific Reports, validating the immune-reprogramming and anti-leukemic potential of its investigational macrophage CLEVER-1 inhibitor, bexmarilimab, in acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS).

The study, conducted in collaboration with leading Finnish and Danish academic research centers, examined AML cell lines and bone marrow samples from patients with AML (n = 34) and MDS (n = 4) and showed that CLEVER-1 is highly expressed on malignant myeloid cells, especially in AML with monocytic differentiation.

Immunotherapy treatments have revolutionized the treatment of many cancers but remain largely ineffective in AML and MDS due to immune resistance. Bexmarilimab addresses these challenges by reactivating the immune environment at its source (i.e. macrophages) and targeting CLEVER-1 directly on leukemic blasts. This approach represents a novel therapeutic avenue in myeloid malignancies where effective treatments are urgently needed.

CLEVER-1, a scavenger receptor involved in immunosuppression, was found to be highly expressed on immature myeloid cells and their monocytic derivatives, particularly in AML cases with M4-M5 FAB subtypes and in Fms Related Receptor Tyrosine Kinase 3 (FLT3) and/or Nucleophosmin 1 (NPM1) mutations.

“Our results show that CLEVER-1 is not only a macrophage checkpoint but also expressed on malignant myeloid cells and targeting it with bexmarilimab can enhance antigen presentation and sensitize these cells to standard therapies. These findings highlight a compelling opportunity to integrate bexmarilimab into standard treatment regimens for AML and MDS,” said Dr. Maija Hollmén, Chief Scientific Officer of Faron and senior author of the study, MediCity Research Laboratory and InFLAMES Flagship, University of Turku, Turku, Finland.

Using ex vivo models, the team evaluated the effects of bexmarilimab, alone and in combination with standard-of-care therapies azacitidine and venetoclax, on immune activation and cell viability. Bexmarilimab monotherapy increased the expression of antigen-presenting HLA-DR by 1.2- to 2-fold in up to 66% of patient samples, especially in those with low baseline expression and high CLEVER-1 levels, without any cytotoxic effects on the blast cells, via potentially inducing interferon gamma (IFNg). Addition of bexmarilimab helped overcome venetoclax-resistance in 33–40% of ex vivo AML samples, without adding to lymphocyte toxicity. Moreover, the triplet of bexmarilimab, azacitidine, and venetoclax showed enhanced cytotoxicity in ex vivo models of treatment-resistant AML; suggesting that bexmarilimab increases the susceptibility to venetoclax and/or azacitidine induced cell death.

“This is an important milestone in our mission to bring the benefits of macrophage checkpoint inhibition to patients with hematological cancers like AML and MDS, especially after disease relapse,” said Dr. Juho Jalkanen, CEO of Faron Pharmaceuticals. “The results strongly support the clinical rationale for combining bexmarilimab with standard therapies and also offer compelling evidence that our approach is both scientifically robust and clinically relevant, guiding the continued advancement of our BEXMAB trial.”

The ongoing BEXMAB clinical study is currently evaluating bexmarilimab in combination with azacitidine in relapsed/refractory AML and MDS patients. The trial’s most recent phase II data, to be presented at ASCO and EHA 2025, showed promising response rates in pretreated populations.

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:

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

Study highlights:

• Tissuemicroenvironments showed three distinct response patterns to bexmarilimab, driven by the level of inflammation, macrophage type, and their location within the tissue.
• Bexmarilimab stimulated response in immunologically-cold tumors and inhibited inflammation in treatment resistant tumors.
• New patent filed around gene signature validated to predict bexmarilimab sensitivity, offering a tool for patient selection.
• Cancer-free adjacent tissues also responded to bexmarilimab, primarily through B cell activation, hinting towards long lasting systemic beneficial affects against cancer.

Turku, Finland – Faron Pharmaceuticals Ltd. (AIM: FARN, First North: FARON), a clinical-stage biopharmaceutical company, today announced the publication of a new study in the peer-reviewed Journal for ImmunoTherapy of Cancer, shedding new light on how the tumor microenvironment (TME) influences the response to bexmarilimab, the company’s investigational macrophage-targeting immunotherapy.

The study, conducted by researchers at the University of Turku and Turku University Hospital, used advanced patient-derived explant culture (PDEC) models and cutting-edge transcriptomic profiling to map the immunological landscape surrounding tumors and their adjacent tissues. It identified critical molecular and cellular factors that determine how patients respond to bexmarilimab therapy.

Dr. Maija Hollmén, Chief Scientific Officer of Faron Pharmaceuticals and senior author of the study from MediCity Research Laboratory and InFLAMES Flagship, University of Turku, Finland, said, “This work gives us a mechanistic framework for understanding why some patients respond remarkably well to bexmarilimab while others do not in solid tumors. By recognizing that macrophage response is governed by the tissue’s immunological state, we open new possibilities for tailored therapy. This could help guide both clinical trial design and eventual treatment decisions, ensuring patients are matched with the right therapy to have a better chance at survival.”

Tumor-associated macrophages, or TAMs, are specific immune cells found within and around solid tumors. Depending on their type, they can either help the body fight cancer (immunostimulatory) or protect the tumor (immunosuppressive) and help it grow. Hence, it is important to study them in the tumor microenvironment (TME or the area around the tumor where these cells are present). By understanding how TAMs behave in different tumors, researchers can find better ways to treat cancer with medicines that reprogram these cells to attack the tumor instead of helping it. Bexmarilimab is a first-in-class humanized anti–Clever-1 antibody designed to reprogram TAMs from an immunosuppressive to an immunostimulatory phenotype. By inhibiting the scavenger receptor Clever-1, bexmarilimab enhances antigen presentation and promotes anti-tumor immunity. This study addresses the heterogeneity of TAMs by showing how their type, origin, and the tumor environment influence response to therapy.

Researchers analyzed samples of tumor and adjacent cancer-free tissue from patients with breast cancer who underwent mastectomy (PDEC model). They also applied a combination of single-cell RNA sequencing, spatial transcriptomics, and cytokine profiling to identify the genes associated with bexmarilimab’s response.

Bexmarilimab stimulated response in immunologically “cold” tumors (low inflammation) and reduced inflammation in TMEs with strong IFN signaling and advanced TAM activity. The analysis validated five genes (including CXCL9, FCGR1A, GBP5, SLAMF7, and SERPING1) that accurately predicted sensitivity to bexmarilimab. This allows for a potential biomarker-based strategy to enrich patient populations in clinical trials in solid tumors and optimize therapeutic outcomes. A new patent application has been filed for using this method for patient selection.

Dr. Petri Bono, Chief Medical Officer, Faron Pharmaceuticals, said, “As the immunotherapy landscape evolves, understanding the TME’s influence on treatment response is becoming increasingly critical. These findings lay a scientific foundation for advancing macrophage-targeting therapies beyond trial-and-error toward a more predictive treatment. It can potentially impact the design of future trials for bexmarilimab in solid tumors, including potential companion diagnostics based on the identified gene signature.”

Moreover, despite that macrophage are known to be crucial for innate immune responses cancer-free adjacent tissues consistently showed B cell activation regardless of the corresponding tumor’s response, hinting at systemic immune benefits as a part of adaptive immunity, i.e. the memory side of the immune system. These findings underscore the context-dependent nature of macrophage reprogramming and the need for precise patient selection in clinical development.

The study also highlighted that bexmarilimab may complement existing immune checkpoint therapies. Bexmarilimab targeted tumor environments that are immunologically opposite to those responsive to anti-PD-(L)1 therapy. While each associated differently with baseline IFN signaling, both triggered IFN responses when effective.

For the latest findings in Finnish, see the University of Turku press release.

Bexmarilimab is currently under investigation for both hematological cancers and advanced solid tumors. The ongoing BEXMAB clinical study is evaluating bexmarilimab in combination with azacitidine in relapsed/refractory AML and MDS patients. The trial’s most recent phase II data, to be presented at ASCO and EHA 2025, showed promising response rates in pretreated populations.

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:

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 developing novel immunotherapies, today announced that data from its ongoing BEXMAB study evaluating bexmarilimab in patients with high-risk myelodysplastic syndromes (HR-MDS) have been accepted for an oral presentation at the 30th European Hematology Association’s (EHA 2025) Congress, taking place in Milan from June 12–15, 2025.

The oral presentation will feature detailed efficacy and safety results from the study of bexmarilimab in combination with standard-of-care (azacitidine) in frontline with HR-MDS and those with refractory or relapsed HR-MDS who have failed hypomethylating agent (HMA) therapies (r/r MDS). This follows the recent positive findings of the Phase II data from the BEXMAB study, and reaffirms bexmarilimab’s mechanism of action and its potential in improving outcomes for patients with MDS.

Dr. Juho Jalkanen, CEO of Faron Pharmaceuticals, said: “We are thrilled to see bexmarilimab’s data receive acceptance for oral presentation at EHA, following similar recognition at MDS and ASCO. This continued momentum reinforces our belief that bexmarilimab holds real promise as a much-needed therapeutic option for patients with higher-risk MDS, a rare and challenging condition with few effective treatments. We remain deeply committed to advancing its development and bringing meaningful innovation to those who need it most.”

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

Further details will be shared closer to the conference dates.

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

• 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.

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), a clinical-stage biopharmaceutical company developing novel immunotherapies, announces that new Phase II data from its ongoing BEXMAB study evaluating bexmarilimab in high-risk myelodysplastic syndromes (HR-MDS) will be presented 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 abstract, selected for oral communication, highlights the efficacy and tolerability of bexmarilimab, Faron’s novel humanized anti-Clever-1 antibody, with no dose-limiting toxicities during dose escalation, when used in combination with standard-of-care, azacitidine, treatment in patients with both treatment naïve (frontline) and relapsed/refractory (r/r) HR MDS patients.

“We are thrilled to see bexmarilimab’s potential continuing to translate into clinical benefit for patients with this challenging hematologic malignancy,” said Dr. Petri Bono, Chief Medical Officer of Faron Pharmaceuticals. “The Phase II results build on our understanding of Clever-1 biology and bexmarilimab’s mode of action, and we believe this novel immunotherapy could offer a meaningful new treatment pathway for patients. The acceptance of our results for an oral presentation also represents an important company milestone, as it reflects the scientific interest we have generated, as recognized by the largest global oncology community — ASCO “

Faron’s presence at ASCO 2025 reinforces the Company’s commitment to advancing science-driven novel immunotherapy solutions and to addressing unmet medical needs in oncology.

The details of the oral presentation are as follows:

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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.