FLAG-003

FLAG-003 is a multi-acting, water-soluble small molecule therapeutic developed by FLAG Therapeutics for the treatment of gliomas, particularly diffuse intrinsic pontine glioma (DIPG) and glioblastoma multiforme (GBM). It operates through two primary mechanisms of action: inhibition of angiogenesis and tubulin inhibition. Specifically, it targets three cell surface receptors essential for tumor vascularization—epidermal growth factor receptor (EGFR), vascular endothelial growth factor receptor 2 (VEGF-R2), and platelet-derived growth factor receptor beta (PDGFR-β)—thereby disrupting the tumor’s blood supply. Concurrently, it inhibits tubulin production required for cellular division in cancer cells. Preclinical studies have shown that FLAG-003 can cross the blood-brain barrier and evade P-glycoprotein efflux pumps. The drug has demonstrated potent anti-tumor activity in DIPG and GBM cell lines with greater efficacy than ONC201 in preclinical models. It has received Orphan Drug Designation from both the FDA and EMA for glioma as well as Rare Pediatric Disease designation from the FDA[1][2][3][4][5][6].

Other names

FLAG-003FLAG003FLAG 003