BDTX-GBM-001

BDTX-GBM-001 is a brain-penetrant small molecule inhibitor of the Epidermal Growth Factor Receptor (EGFR) specifically designed to target oncogenic mutations in the extracellular domain (ECD) found in glioblastoma (GBM). Developed by Black Diamond Therapeutics, the drug selectively inhibits 'Locked-dimer' (LoDi)-EGFR mutants, which are constitutively active and ligand-independent. BDTX-GBM-001 demonstrates high potency against the five major LoDi-EGFR mutants while maintaining selectivity over wild-type EGFR, potentially minimizing systemic toxicities. Preclinical data in intracranial patient-derived xenograft models showed significant tumor growth reduction and improved survival, supporting its development as a targeted therapy for GBM patients harboring EGFR ECD mutations.