BH-30236

BH-30236 is a novel, orally available, multi-targeted small molecule inhibitor developed by BlossomHill Therapeutics. It acts as an ATP-competitive macrocyclic inhibitor of CDC-like kinases (CLK1, CLK2, and CLK4), as well as other kinases such as PIM3, FLT3, and DYRK1/2. The drug is designed to selectively modulate aberrant alternative splicing in cancerous tissue by targeting the cell’s splicing machinery. This modulation promotes the expression of splicing isoforms that favor apoptosis and anti-proliferative pathways while disrupting cancer cells’ ability to exploit off-target resistance mechanisms. The primary indications under investigation are relapsed or refractory acute myeloid leukemia (AML) and higher-risk myelodysplastic syndrome (HR-MDS). As of early 2025, BH-30236 is in Phase 1 clinical trials for these hematologic malignancies.