Building on the very promising data from the early-stage trial, Faron Pharmaceuticals Limited (AIM:FARN, OTC:FPHAF) has launched the phase II evaluation of BEXMAB focusing on people with myelodysplastic syndromes (MDS) resistant to standard treatments.
MDS is a group of bone marrow disorders that result in an inability to produce enough healthy blood cells.
The trial will test two dosages. Guided by feedback from the US Food and Drug Administration (FDA) it will work with 3 milligrams per kilogram and 6 milligrams per kilogram of bexmarilimab.
Thirty-two patients who have not responded to hypomethylating agents (HMAs), a common MDS treatment, will be enrolled.
They will be randomly assigned to one of the two dosages. After the first 20 participants complete two treatment cycles, an interim analysis will assess the effectiveness of the dosages. Faron aims to use this data to consult with the FDA on future regulatory approval processes for bexmarilimab.
"This is a significant milestone in the development of what we hope will be the first macrophage-targeting immunotherapy for patients with aggressive myeloid leukaemias," said Dr Markku Jalkanen, Faron CEO.
"The emerging bexmarilimab data from the Phase 1 part of the study have been very encouraging, showing continued efficacy signals and long duration of responses.
"We believe that bexmarilimab has the potential to improve patient outcomes and improve quality of life for those suffering from MDS, addressing the longstanding unmet medical needs in conditions which have had no new effective treatments in decades."
Faron Pharmaceuticals has shared encouraging results from the initial phase of the BEXMAB trial with the drug showing a 50% remission rate in certain patient groups.
Specifically, 11 out of 22 patients experienced remission with the 'doublet' dose. Of these, eight patients saw a complete remission in their bone marrow.
This was sometimes accompanied by a recovery in blood cell counts. The highest response rate was 80% in patients with MDS who did not benefit from previous HMA treatments.
Bexmarilimab, combined with standard of care (SoC), has been well-received by patients. It has not caused any dose-limiting side effects. Due to these positive outcomes, Faron is planning to expand the trial. New trial sites will open in the United States and Europe to facilitate faster participant enrolment.