REC-0545

REC-0545 is a potent, selective, and orally bioavailable small molecule inhibitor of the splicing factor RNA binding motif protein 17 (RBM17), developed by Recursion Pharmaceuticals. Discovered via the Recursion OS AI-enabled phenomics platform, the drug is designed to exploit a synthetic lethal vulnerability in cancers harboring mutations or deletions in the RBM10 tumor suppressor gene. RBM10 and RBM17 are paralogous proteins involved in the regulation of alternative splicing; in the absence of functional RBM10, cells become uniquely dependent on RBM17 for the processing of essential pre-mRNAs. By inhibiting RBM17, REC-0545 triggers mis-splicing and subsequent apoptosis specifically in RBM10-deficient tumor cells, such as those found in a significant subset of non-small cell lung cancers (NSCLC). It is currently being evaluated in Phase 1 clinical trials for patients with advanced solid tumors characterized by RBM10 alterations.