Definition

An orally bioavailable, small molecule mRNA degrader of the proto-oncogene c-Myb, with potential antineoplastic activity. Upon oral administration, c-Myb mRNA degrader REM-422 promotes the inclusion of poison exon into the c-Myb pre-mRNA transcript, resulting in the degradation of c-Myb mRNA and the reduction of both c-Myb mRNA and c-Myb protein levels. This may decrease proliferation in tumor cells with c-Myb dysregulation or overexpression. c-Myb, an oncogenic transcription factor that plays a key role in the regulation of hematopoietic cell differentiation and proliferation, is dysregulated or overexpressed in a variety of tumor cell types.