ACT004

ACT004 is a small molecule drug developed as a derivative of ovatodiolide (OVA), a bioactive diterpenoid. It has been designed to have high metabolic stability toward cytochrome P450 enzymes, with no genotoxicity and no accumulative or delayed toxicities observed after long-term administration in animal studies. Mechanistically, ACT004 inhibits the stemness of hepatocellular carcinoma (HCC) cells by suppressing MTDH gene transcription through destabilization of SP1 protein and inhibition of the JAK/STAT3 signaling pathway. This results in decreased CD133 levels and reduced cancer stem cell properties. Preclinical studies have shown that ACT004 can inhibit tumor growth in HCC models, suggesting its potential as an anti-cancer agent targeting cancer stem cells[1]. The drug is currently in Phase 1 clinical development for immune system diseases, digestive system disorders, and respiratory diseases, with active indications including autoimmune hepatitis, nephritis, and pulmonary fibrosis[1][4].