BLU-5082

BLU-5082 is a novel, brain-penetrant small molecule tyrosine kinase inhibitor (TKI) developed by Blueprint Medicines. It specifically targets the epidermal growth factor receptor (EGFR), including wild-type amplification and specific mutations such as A289T (extracellular domain) and exon 20 insertions/deletions (e.g., 767delins). The drug is being investigated for the treatment of H3K27M-mutant diffuse midline gliomas (DMGs) that harbor EGFR alterations. Preclinical studies have demonstrated nanomolar potency against human DMG cells and significant tumor regression in intracranial and flank xenograft models, highlighting its potential to cross the blood-brain barrier and address the unique biology of pediatric high-grade brain tumors.