emvododstat

Emvododstat (PTC299) is a novel, orally administered small molecule developed by PTC Therapeutics. It acts as a potent inhibitor of dihydroorotate dehydrogenase (DHODH), an enzyme critical for de novo pyrimidine nucleotide synthesis. By inhibiting DHODH, emvododstat reduces the production of uridine nucleotides and subsequently inhibits the translation of vascular endothelial growth factor A (VEGFA) mRNA, leading to decreased VEGF synthesis. This dual mechanism results in antiangiogenic and antiproliferative effects in tumor cells and has shown broad activity against various solid tumors and hematologic malignancies. Additionally, emvododstat exhibits antiviral properties by blocking replication of RNA viruses such as SARS-CoV-2 and Ebola virus while also attenuating pro-inflammatory cytokine production. The drug is being investigated for cancer (including acute myeloid leukemia) and COVID-19[1][3][4][5][6][8].

Other names

1219951-09-31256565-36-2