elenestinib + azacitidine

**Elenestinib + azacitidine** is an investigational oral combination therapy under clinical evaluation for advanced systemic mastocytosis (AdvSM) and other KIT-altered hematologic malignancies. - **Elenestinib (BLU-263)** is a next-generation, potent, selective small molecule inhibitor of the KIT D816V mutation, which is a key driver in systemic mastocytosis. It acts as a targeted tyrosine kinase inhibitor (TKI) with limited central nervous system penetration, designed for once-daily oral administration[6][2][4]. - **Azacitidine** is a DNA methyltransferase inhibitor (hypomethylating agent) that induces cytotoxicity in abnormal hematopoietic cells and has established use in treating myelodysplastic syndromes and certain leukemias. - The combination is intended to provide disease-modifying therapy by targeting mutant mast cells and abnormal hematopoietic lineages, particularly in high-risk AdvSM and associated hematologic neoplasms[5][3][9]. - The regimen is being investigated to establish safety, efficacy, dose optimization, and recommended use in Phase 1/2 trials[5][3][7]. - Developed and sponsored by Blueprint Medicines[5][3][9].

Other names

elenestinib + azacitidine