RP7214 + azacitidine

**RP7214 + azacitidine** is an investigational combination therapy currently in clinical trials for hematological malignancies such as acute myeloid leukemia (AML), myelodysplastic syndromes (MDS), and chronic myelomonocytic leukemia (CMML)[1][7]. RP7214 is a novel, potent small molecule inhibitor of dihydroorotate dehydrogenase (DHODH), a rate-limiting enzyme in pyrimidine biosynthesis that is overexpressed in certain cancers and impedes tumor cell proliferation via cell cycle arrest[3][5]. Azacitidine is a pyrimidine nucleoside analogue and DNA demethylation agent that exerts antineoplastic effects by inhibiting DNA methyltransferase and incorporating into RNA and DNA to impair cancer cell proliferation[2]. Preclinical studies demonstrate that RP7214 potentiates the activity of azacitidine, showing enhanced growth inhibition, increased differentiation (CD11b+ expression), and antitumor effects relative to single agents[3][5]. The combination is being explored in a multi-center, open-label, dose-escalation and expansion Phase I/Ib trial for patients with relapsed/refractory or newly diagnosed AML, intermediate/high-risk MDS, and CMML, especially those ineligible for intensive induction chemotherapy[1][7].

Other names

5-azacytidineRP7214 + azacitidine