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Rogocekib is a **first-in-class, selective, orally available small molecule inhibitor** targeting **CDC2-like kinase (CLK)**, a key dual-specificity protein kinase regulating **RNA splicing** in cell proliferation[2][3][6]. It is being developed primarily for **hematological malignancies** such as **relapsed or refractory acute myeloid leukemia (AML)** and **myelodysplastic syndromes (MDS)**, with additional exploration in other cancers[2][1][4]. It has demonstrated dose-dependent antitumor effects in preclinical models and is under active Phase 1/2 clinical evaluation in the United States and Japan[1][2][3][6][7]. Rogocekib has received **Orphan Drug Designation** from the FDA for AML[4][5][8].
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