TXR-311

TXR-311 is a preclinical, first-in-class small-molecule anticancer candidate discovered using Aria (formerly twoXAR) Pharmaceuticals’ AI-driven in silico drug discovery platform for hepatocellular carcinoma (HCC). It demonstrates potent, highly selective cytotoxicity against multiple genetically diverse HCC cell lines with nanomolar-range IC50 values that are roughly 70-fold lower than sorafenib while showing ~500-fold lower activity in primary human hepatocytes, indicating a favorable therapeutic index.[1][10][13][16] In HCC patient-derived xenograft models, TXR-311 produced tumor growth inhibition comparable to sorafenib with good tolerability, and it also shows in vitro activity at low micromolar concentrations in pancreatic ductal adenocarcinoma cells.[1][4][7][10][18][20] The compound is reported to act through a novel, undisclosed mechanism of action distinct from multikinase inhibition, and remains in preclinical optimization as a potential first-in-class therapy for liver cancer.[1][4][7][10][16][19]