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Elimusertib is an orally available, highly selective small molecule inhibitor of ataxia telangiectasia and Rad3-related (ATR) kinase, a serine/threonine protein kinase that plays a critical role in the DNA damage response. By selectively binding to and inhibiting ATR, elimusertib disrupts ATR-mediated signaling pathways essential for DNA repair, cell cycle progression, and cell survival in tumor cells. This inhibition leads to impaired DNA damage checkpoint activation, increased replication stress, accumulation of DNA damage (including single-stranded and double-stranded breaks), induction of apoptosis—particularly in cancer cells with high replication rates or specific defects in DNA repair pathways—and ultimately anti-tumor effects. Elimusertib is under clinical investigation for the treatment of relapsed or refractory solid tumors (including pediatric cancers such as Ewing sarcoma and alveolar rhabdomyosarcoma), advanced solid tumors (excluding prostate cancer), ovarian cancer, and breast cancer. The drug has demonstrated strong preclinical antitumor activity across various pediatric solid tumor models[1][2][5][6][7].
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