ATX-968

ATX-968 is a potent, selective, and orally available small molecule inhibitor of the ATP-dependent RNA helicase A (DHX9), also known as DExH-box helicase 9. DHX9 plays a critical role in the regulation of transcription, translation, and maintenance of genome stability by unwinding double-stranded DNA/RNA and secondary structures such as R-loops and G-quadruplexes. Elevated expression of DHX9 is observed in multiple cancer types, particularly those with microsatellite instability-high (MSI-H) or deficient mismatch repair (dMMR). In preclinical studies, inhibition of DHX9 by ATX-968 leads to replication stress, DNA damage, cell cycle arrest, apoptosis in MSI-H/dMMR cancer cells, and robust tumor regression in xenograft models. The compound demonstrates high selectivity for DHX9 over related helicases and kinases. Its primary development focus is on cancers characterized by genomic instability such as colorectal cancer (especially MSI-H/dMMR subtypes) and small cell lung cancer[1][3][4][5][7].

Other names

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