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TTI-101 is a first-in-class, orally bioavailable, selective small molecule inhibitor of signal transducer and activator of transcription 3 (STAT3). It binds tightly to the SH2 domain of STAT3, blocking its phosphorylation at tyrosine 705 and preventing dimerization and nuclear translocation. This inhibition disrupts STAT3-mediated transcriptional activation, which plays a central role in the pathogenesis of fibrosis (notably idiopathic pulmonary fibrosis) and cancer cell survival, proliferation, immune evasion, and metastasis. TTI-101 is being developed primarily for idiopathic pulmonary fibrosis (IPF), hepatocellular carcinoma (HCC), and other solid tumors. The drug has received orphan drug designation for IPF and HCC as well as Fast Track Designation for HCC[1][2][4][5][6].
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