REM-422

REM-422 is a first-in-class, potent, selective, and orally bioavailable small molecule mRNA degrader developed by Remix Therapeutics. It targets the proto-oncogene MYB (c-Myb) by facilitating the incorporation of poison exons into MYB mRNA transcripts, leading to nonsense-mediated decay (NMD) and subsequent reduction in MYB protein expression. This mechanism addresses MYB dysregulation upstream of protein expression and results in antitumor activity in models of MYB-dependent cancers. REM-422 is being investigated primarily for adenoid cystic carcinoma (ACC), acute myeloid leukemia (AML), and high-risk myelodysplastic syndromes (MDS). The drug has received orphan drug designation for ACC and AML[1][2][5][6][8].