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(Sharecast News) - Faron Pharmaceuticals announced on Monday that, following guidance from the US Food and Drug Administration (FDA), the initial indication for phase two advancement in the study investigating bexmarilimab in combination with standard of care, would focus on hypomethylating agents (HMAs)-refractory or relapsed myelodysplastic syndromes (MDS).
The AIM-traded firm said the BEXMAB study is a multicentre trial in Finland and the US, aiming to assess the safety and efficacy of bexmarilimab, a novel anti-Clever-1 humanised antibody, when used in conjunction with standard of care for patients with aggressive myeloid leukaemias.
It said the phase two segment of the study would enrol 32 patients with HMA-failed MDS, who would be randomly assigned to receive either three or six milligrams per kilogram doses of bexmarilimab, with the possibility of proceeding to a phase two and three extension.
The data from the first 20 patients would be assessed to determine the optimal dosing level before discussing a potential registrational study plan with the FDA.
Data from the phase one portion of the study indicated that three milligrams per kilogram dosing achieve optimal target engagement, with the highest immune activation observed in both the three and six-milligram cohorts.
Notably, both dose levels demonstrated safety and tolerability thus far.
The company said the selection of the three and six-milligram doses aligned with the FDA's 'Project Optimus' initiative, which was looking to redefine dose optimisation and selection in oncology drug development.
Faron Pharmaceuticals recently reported encouraging data from the phase one part of the trial, where bexmarilimab yielded a 50% remission rate in doublet dose groups, with eight out of 11 patients achieving complete remission in the bone marrow.
Among patients who had previously failed HMA therapy, the overall response rate was 80%.
Importantly, the bexmarilimab and standard of care combination maintained a favourable safety profile across all dose levels, with no dose-limiting toxicities reported.
Faron anticipated expanding trial sites in the US and Europe to accommodate the accelerated recruitment pace.
"This is a significant milestone in the development of what we hope will be the first macrophage-targeting immunotherapy for patients with aggressive myeloid leukemias," said chief executive officer Dr Markku Jalkanen.
"The emerging bexmarilimab data from the phase one 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."
At 1035 GMT, shares in Faron Pharmaceuticals were up 6.08% at 270.5p.
Reporting by Josh White for Sharecast.com.
