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HJC0125 is a novel, potent, and orally active small molecule inhibitor of Signal Transducer and Activator of Transcription 3 (STAT3), developed by researchers at the University of Texas MD Anderson Cancer Center and the University of Texas Medical Branch. Designed using a fragment-based drug design strategy, HJC0125 addresses the limitations of earlier STAT3 inhibitors, such as poor solubility and bioavailability. The compound works by suppressing STAT3 promoter activity and phosphorylation (specifically at the pY705 site), while also reducing total STAT3 protein expression. In preclinical studies, HJC0125 has demonstrated the ability to induce cell growth arrest and early apoptosis in ER-negative breast cancer cell lines (e.g., MDA-MB-231) and has shown efficacy in suppressing xenograft tumor growth via both oral and intraperitoneal administration routes.
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