Drug intelligence / Profile preview

RP7214 + standard of care

Development stage
Unknown
Lead developer
Rhizen Pharmaceuticals
Modality
Small Molecules
Administration
Oral (for RP7214), Route(s) Appropriate To The Specific Standard-of-care Agent(s)
01

Overview

RP7214 + standard of care is a combination therapy consisting of RP7214, a potent and selective small molecule inhibitor of human dihydroorotate dehydrogenase (DHODH), administered alongside the current standard-of-care agents for specific diseases. DHODH is a mitochondrial enzyme essential for de novo pyrimidine synthesis, which is critical for rapidly proliferating cells such as cancer cells and replicating viruses. By inhibiting DHODH, RP7214 disrupts nucleotide synthesis, thereby exerting antiviral and antileukemic effects. Preclinical studies have shown that RP7214 can reduce tumor volume in acute myeloid leukemia (AML) models both as monotherapy and in combination with standard-of-care agents such as cytarabine, venetoclax, and azacytidine. Clinical trials have evaluated this combination primarily in relapsed/refractory AML, chronic myelomonocytic leukemia (CMML), myelodysplastic syndromes (MDS), COVID-19 infection, solid tumors including breast and colorectal cancer, and dry eye disease[5][6]. The "standard of care" component varies by indication but typically includes established therapies relevant to each disease.

02

Targets

DHODH (Dihydroorotate dehydrogenase (quinone), mitochondrial)

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