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OR-2100 is an orally bioavailable prodrug of decitabine, a hypomethylating agent. Decitabine is incorporated into DNA during the S phase of the cell cycle and inhibits DNA methyltransferase, leading to hypomethylation of DNA and reactivation of silenced genes. This mechanism is particularly relevant in hematologic malignancies such as myelodysplastic syndromes (MDS) and chronic myelomonocytic leukemia (CMML). OR-2100 was developed to provide sustained plasma concentrations via oral administration, potentially improving efficacy compared to conventional intravenous decitabine. Preclinical studies demonstrated that OR-2100 has comparable efficacy to existing hypomethylating agents in blood cancer models and a favorable safety profile in animal studies. In clinical development, it has shown expected pharmacokinetic properties in patients with relapsed or refractory higher-risk MDS and CMML[5].
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