Updated BEXMAB Phase I/II Data presented at ESMO 2025 shows further improvement, strengthening the clinical profile of bexmarilimab in treatment-naïve HR-MDS patients

Faron Pharmaceuticals OyAnnouncement

Yesterday 07:00

Faron Pharmaceuticals Ltd | Company announcement | October 20, 2025 at
09:00:00 EEST

Updated BEXMAB Phase I/II Data presented at ESMO 2025 shows further
improvement, strengthening the clinical profile of bexmarilimab in
treatment-naïve HR-MDS patients with an 85% ORR and a 45% CR rate, bolstered
by pharmacodynamic insights

New biomarker data reveals strong correlation between target engagement and
clinical response with 100% ORR in treatment-naïve, low blast count (<5%)
patients

Key highlights:

* Bexmarilimab and azacitidine combination resulted in an 85% objective
  response rate (ORR; 17/20 evaluable patients) and a 45% complete remission
  (CR) rate (9/20) in treatment-naïve patients with higher-risk
  myelodysplastic syndrome (HR-MDS)
* 55% (11/20) of treatment-naïve patients with HR-MDS showed full clearance of
  bone marrow (BM) blasts
* Deeper BM engagement in treatment-naïve patients with <5% bone marrow blasts
  at baseline translated to 100% ORR, one of the best results ever reported in
  this patient population
* 23% of patients across the BEXMAB study were successfully bridged to a
  potentially curative stem cell transplant (SCT)
* Faron will be hosting a virtual webinar to discuss the updated BEXMAB data
  presented at ESMO 2025 on 23 October at 4pm EEST/9am ET.

Turku, Finland– Faron Pharmaceuticals Ltd. (AIM: FARN, First North: FARON), a
clinical-stage biopharmaceutical company focused on tackling cancers through
novel immunotherapies, today announced that updated data from the Phase I/II
BEXMAB study continues to show significant clinical activity for bexmarilimab.
The findings, presented in an oral session at the European Society for Medical
Oncology (ESMO) Congress 2025 by Dr. Mika Kontro from Helsinki University
Hospital, not only confirm the high response rates seen in earlier analyses
but also provide a clear pharmacodynamic rationale linking the drug’s
mechanism of action directly to patient outcomes.  

The BEXMAB study evaluates 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. The data presented at ESMO 2025
included 21 treatment-naïve (20 evaluable for efficacy) and 32
relapsed/refractory (r/r) HMA-failed patients with HR-MDS.

The updated data presented at ESMO 2025 reinforces the efficacy previously
observed for the bexmarilimab and azacitidine combination. In 20 evaluable
treatment-naïve patients, the study confirmed an 85% ORR and a 45% CR rate.
These high response rates were observed in a difficult-to-treat population,
where over 66% of patients were classified as high to very high risk at
baseline. The combination also showed robust activity in patients with
high-risk mutations like mTP53, achieving an ORR of 78%.  

In the r/r HMA-failed population (n=32), the combination achieved a 63% ORR
and a median overall survival (mOS) of 13.4 months. Notably, nearly a third of
these patients (31.3%) had received prior therapy with the Bcl-2 inhibitor
venetoclax.  

About 23% of patients across the BEXMAB study were successfully bridged to a
potentially curative stem cell transplant (SCT).

The most significant update is the new pharmacodynamic data that provided a
clear biological explanation for the strong clinical results. The analysis
showed a statistically significant correlation (p=0.0006) between deeper
engagement of the Clever-1 target in the bone marrow and a positive clinical
response. This correlation was particularly striking in the subgroup of
treatment-naïve patients with <5% bone marrow blasts at baseline (38% of the
cohort), a population for which effective, non-intensive therapies are
urgently needed. In these patients, deeper target engagement translated to a
100% ORR, supporting bexmarilimab’s unique mechanism of action as a truly
disease modifying agent, differentiating it from other investigational HR-MDS
therapies, such as Bcl-2 inhibitors. For patients with a higher blast count
(>5% at baseline), the ORR remained high at 75%.

With the result of this data, Faron is preparing for the next stage of
development. Following guidance from the FDA announced on 18 August 2025, the
Company has begun preparations for the dose-optimization stage of its Phase
II/III trial for bexmarilimab, after which the trial will transition into the
registrational stage with accelerated approval possibility. The combination
therapy continues to be well-tolerated, with a safety profile similar, or even
better to, azacitidine monotherapy. Only 36% of treatment-emergent adverse
events were considered related to bexmarilimab, with no Grade 5 events.

Dr. Mika Kontro, MD, PhD, Associate Professor at University of Helsinki and
Helsinki University Hospital Comprehensive Cancer Center, Department of
Hematology said, “The BEXMAB data are encouraging, and the new biomarker
analysis provides a clear pharmacodynamic rationale for bexmarilimab’s
clinical activity. The direct correlation between how deeply we engage the
Clever-1 target in the bone marrow and a patient’s clinical response
reinforces the drug’s mechanism. The 100% ORR in patients with low blast
counts suggest that this therapy may help in a population where current
investigational treatments, including Bcl-2 inhibitors, have significant
limitations.”

Dr. Maija Hollmén, PhD, Chief Scientific Officer of Faron Pharmaceuticals,
added, “The selection of this BEXMAB data for an oral presentation at ESMO is
a significant external validation of our science and the clinical potential of
bexmarilimab. These findings help us understand why the drug works and for
whom it works best. The clear biomarker impact in the bone marrow and unique
efficacy in patients with low blast counts highlights Bex’s ability to change
the course of the disease and provides a solid foundation for our late-stage
clinical development, bringing this promising therapy to patients who
desperately need better options.”

To register for the event visit: ESMO 2025
(https://faron.videosync.fi/esmo-2025/register). The details of the ESMO oral
presentation are as follows:

Presentation title: Macrophage reprogrammer Bexmarilimab Plus Azacitidine in
Myelodysplastic Syndrome: PK/PD and biomarker results from the Phase I/II
BEXMAB Study

Presented by: Dr. Mika Kontro

Session type and title: Mini Oral Session: Haematological Malignancies

Room: Solingen Auditorium - Hall 23

Session date & time: Oct 19, 2025 (9:31 to 9:36 am CEST)

Abstract no.: 1249MO

This announcement contains inside information for the purposes of Article 7 of
the EU Regulation 596/2014 ("MAR") and Article 7 of MAR as incorporated into
UK domestic law by virtue of the European Union (Withdrawal) Act 2018 ("UK
MAR").

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| Kare Laukkanen                      |                                      |
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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 (http://www.faron.com/).

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