Drug intelligence / Profile preview

RP7214

Development stage
Phase 2
Lead developer
Rhizen Pharmaceuticals
Modality
Small Molecules
Administration
Oral
01

Overview

RP7214 is an orally bioavailable small molecule inhibitor of dihydroorotate dehydrogenase (DHODH), a mitochondrial enzyme essential for de novo pyrimidine synthesis. By inhibiting DHODH, RP7214 disrupts nucleotide biosynthesis required for the proliferation of rapidly dividing cells and viral replication. This mechanism confers potential antineoplastic, anti-inflammatory, and antiviral activities. Preclinical studies have demonstrated its efficacy in models of acute myeloid leukemia (AML) and SARS-CoV-2 infection. In clinical trials, it has shown favorable safety and pharmacokinetic profiles in healthy volunteers. The drug is being developed primarily for indications including AML, chronic myelomonocytic leukemia (CMML), myelodysplastic syndromes (MDS), and COVID-19 infections[1][2][3][4][6][7].

Other names
DHODH inhibitor RP7214
02

Targets

DHODH (Dihydroorotate dehydrogenase (quinone), mitochondrial)

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