Faron Pharmaceuticals Limited (AIM:FARN) has reported fresh data from an early-stage trial of its experimental cancer drug bexmarilimab, suggesting it may help some patients with an aggressive form of bone-marrow disorder live meaningfully longer than expected.
The results, presented at the American Society of Haematology meeting, focus on higher-risk myelodysplastic syndromes (known as HR-MDS).
This is a group of conditions in which the bone marrow struggles to produce healthy blood cells. Patients often require frequent blood transfusions and, in more advanced cases, face a risk of developing acute leukaemia.
Survival outcomes are typically poor, particularly for those who have a mutation in a gene called TP53, which makes cancers harder to treat.
Faron said the combination of bexmarilimab and azacitidine (a long-established medicine for blood disorders) has now produced a median overall survival of 14.5 months in patients whose disease has relapsed or not responded to earlier treatments.
Historically, this group has lived five to six months on average.
The company highlighted especially strong results in newly diagnosed patients carrying the TP53 mutation. In that group, 70% achieved a complete remission, meaning no detectable signs of disease in their bone marrow.
Dr Amer Zeidan of Yale University, who presented the data, said: “Observing a median overall survival of 14.5 months in the relapsed setting, together with the notably high complete remission rates in frontline TP53-mutated disease (even with the small sample size and limited follow-up) represents a highly encouraging signal of efficacy.”
He added that half of these newly diagnosed TP53-mutated patients were able to move on to a stem cell transplant, describing the combination as “a vital bridge to potentially curative therapy”.
Stem cell transplants are one of the few options that can offer a long-term solution in HR-MDS, but patients must first reach a sufficiently stable condition to undergo the procedure.
In the trial, 50% of newly diagnosed TP53-mutated patients and 21% of those with relapsed disease were able to proceed to transplant.
A notable finding in the updated read-out is the improvement in blood-cell production. More than half (57%) of newly diagnosed patients who relied on transfusions at the start of treatment became transfusion-independent.
Needing fewer transfusions can make day-to-day life considerably easier, and Faron said bone-marrow samples suggest the treatment is supporting the growth of new red cells, platelets and white cells.
Dr Petri Bono, Faron’s chief medical officer, said: “The new data on transfusion independence is clinically very meaningful. For MDS patients, freedom from regular transfusions is a major determinant of quality of life.”
Across the study, the company reported no treatment-related deaths and described the safety profile as “excellent”, an important consideration when drugs are being combined.
Faron plans to advance bexmarilimab into a larger phase III trial, the stage typically required for regulatory approval, as it seeks to offer an option for patients with limited alternatives.