Faron Pharmaceuticals has received Orphan Drug Designation (ODD) from the U.S. Food and Drug Administration (FDA) for its drug bexmarilimab, which is being developed for the treatment of acute myeloid leukemia (AML). ODD is granted to support the development of medicines for rare disorders that affect fewer than 200,000 people in the U.S. The designation provides Faron with benefits such as market exclusivity upon regulatory approval, exemption from FDA application fees, and tax credits for qualified clinical trials. The company believes that bexmarilimab, when combined with standard of care, will lead to better patient outcomes and improved quality of life.

Bexmarilimab is currently in Phase I/II of the BEXMAB study, which is investigating the drug in combination with standard of care for the treatment of relapsed/refractory AML and myelodysplastic syndromes (MDS). Positive data from the Phase I portion of the trial was reported last month, with objective responses observed in patients receiving bexmarilimab in combination with azacitidine. The completion of dose escalation, readout of enrichment cohorts, and initiation of the Phase II portion of the trial are expected in Q4 2023.

Bexmarilimab is an investigational immunotherapy designed to overcome resistance to existing treatments and optimize clinical outcomes in AML. It targets the Clever-1 receptor on macrophages, altering the tumor microenvironment and priming the immune system to attack tumors. By targeting Clever-1, bexmarilimab aims to increase antigen presentation, ignite an immune response, and sensitize cancer cells to standard of care.

Faron Pharmaceuticals is a clinical-stage biopharmaceutical company focused on developing novel immunotherapies to tackle cancers. Its lead asset is bexmarilimab, which is being investigated in Phase I/II clinical trials for hematological cancers in combination with other standard treatments.