Faron Pharmaceuticals Limited (AIM:FARN) has reported encouraging new results from its ongoing BEXMAB trial, showing that its experimental antibody therapy bexmarilimab continues to deliver strong responses in patients with higher-risk myelodysplastic syndromes (HR-MDS), a form of blood cancer.
Updated data presented at the European Society for Medical Oncology (ESMO) Congress in Barcelona showed that patients who received bexmarilimab in combination with azacitidine, a standard treatment for MDS, achieved an 85% overall response rate and a 45% complete remission rate.
The figures represent an improvement on earlier results and strengthen the drug’s clinical profile, particularly in newly diagnosed patients.
The company also revealed fresh biomarker findings showing a direct link between how strongly the drug binds to its target in the bone marrow and how well patients respond.
Among those with lower disease burden, defined as fewer than 5% bone marrow blasts, all patients (100%) responded to treatment.
“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,” said Dr Mika Kontro of Helsinki University Hospital, who presented the data.
“The 100% response rate in patients with low blast counts suggests that this therapy may help in a population where current investigational treatments have significant limitations.”
Bexmarilimab works by targeting a receptor called Clever-1 found on macrophages, a type of immune cell, effectively reprogramming them to attack cancer cells.
In the trial, patients were given weekly infusions of the antibody at different doses alongside azacitidine in 28-day cycles.
The updated results included 21 newly diagnosed and 32 relapsed or refractory patients whose disease had already failed to respond to standard “hypomethylating” drugs such as azacitidine alone.
In the more heavily pre-treated group, the combination achieved a 63% overall response rate and a median survival of 13.4 months.
Roughly one in four patients across the study went on to receive a stem cell transplant, the only potentially curative option for HR-MDS.
The treatment remained well tolerated, with no deaths related to bexmarilimab and fewer serious side effects than expected for combination therapy.
Dr Maija Hollmén, Faron’s chief scientific officer, said: “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.”
Following guidance from US regulators in August, Faron has begun preparing for the next phase of testing, which will optimise dosing before moving into a phase II/III registration trial that could support an accelerated approval if results hold up.
The company will discuss the latest data in more detail during a webcast for investors on Thursday.