Faron Pharmaceuticals Limited (AIM:FARN) has reported encouraging results from its phase II BEXMAB trial, with its investigational drug bexmarilimab showing a clear survival advantage for patients with high-risk myelodysplastic syndromes (MDS), a rare and aggressive group of blood disorders.
The company presented the latest findings at the European Hematology Association’s annual congress in Milan, noting that patients with relapsed or refractory MDS who received bexmarilimab in combination with azacitidine had a median overall survival of 13.4 months.
This compares with the typical five to six months seen with standard treatment.
For the subset of patients with a TP53 mutation, a known marker for particularly poor outcomes, median survival was 9.3 months.
Among those without the mutation, survival data are still maturing, with most patients still alive at the time of reporting.
In patients receiving treatment for the first time, response rates were also strong.
Of the 20 individuals in this group, 85% responded to therapy and half achieved a complete remission in the bone marrow.
Based on the safety and efficacy profile, Faron has selected a dose of 3 mg/kg for the upcoming phase III trial.
Bexmarilimab is designed to block a protein called Clever-1, which is found on the surface of certain immune cells in the bone marrow. By targeting this pathway, the drug aims to reprogramme the immune response and boost the body’s ability to fight cancer cells.
Faron said five patients in the trial had progressed to stem cell transplants, the only potential cure for MDS, underscoring the drug’s ability to extend survival long enough for patients to reach further treatment options.
The study, led by Dr Mika Kontro from the University of Helsinki, builds on preliminary findings presented at the American Society of Clinical Oncology’s meeting earlier this year and lays the groundwork for phase III development.
Dr Petri Bono, Faron's chief medical officer, 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.”