RGT-61159

RGT-61159 is a first-in-class, orally available small molecule inhibitor developed by Rgenta Therapeutics. It targets the oncogenic transcription factor MYB by selectively modulating its RNA splicing machinery. Specifically, RGT-61159 induces the inclusion of a cryptic "poison" exon into c-MYB RNA transcripts, leading to nonsense-mediated decay and robust elimination of both MYB mRNA and protein in cancer cells. This mechanism inhibits proliferation or induces cell death in cancers that overexpress MYB protein, such as adenoid cystic carcinoma (ACC), colorectal cancer (CRC), and acute myeloid leukemia (AML). The drug is currently being evaluated in Phase 1 clinical trials for ACC and CRC, with preclinical development ongoing for AML[1][2][3][4][5][6][7].