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EF-009 is a small molecule drug developed for the treatment of pancreatic cancer, particularly targeting non-resectable and borderline resectable cases. It is delivered locally as an implantable wafer (Cerebraca Wafer) via endoscopic surgery, allowing slow release of the active ingredient directly into the tumor over approximately one month. The active pharmaceutical ingredient, EF-API-001, is a synthetic small molecule derived from natural product research. EF-009 acts primarily as a DNA (cytosine-5)-methyltransferase 1 (DNMT1) inhibitor and also targets AMP activated protein kinase (AMPK), contributing to epigenetic regulation. Its mechanism includes inhibiting tumor growth, stemness, chemo drug resistance, and stimulating immune responses by inhibiting PD-L1 expression on cancer cells—thereby enhancing T cell-mediated anti-tumor immunity. Preclinical studies have shown significant tumor shrinkage with manageable toxicity and prolonged survival in animal models. The drug has completed preclinical development and entered Phase 2 clinical trials for pancreatic carcinoma[1][2][3][4][5].
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