- Guide to PHARMACOLOGY
Synonyms: PTC-299 | PTC299
Compound class: Synthetic organic
Comment: Emvododstat (PTC299) is an orally bioavailable inhibitor of dihydroorotate dehydrogenase (DHODH) that was developed by PTC Therapeutics  for anti-cancer potential. It inhibits de novo pyrimidine nucleotide (UMP) biosynthesis and exhibits broad activity against leukemia in vitro and in vivo.
SARS-CoV-2: Emvododstat inhibits replication of SARS-CoV-2 in vitro (EC50 2.0-31.6 nM) and other RNA viruses . It blocks production of inflammatory cytokines in infected cell cultures.
|No information available.|
|Summary of Clinical Use|
|Emvododstat (PTC299) was originally evaluated in cancer, but has been redeployed for anti-viral activity in COVID-19 patients.|
|Clinical Trial ID||Title||Type||Source||Comment||References|
|NCT04439071||A Study to Evaluate Efficacy and Safety of PTC299 in Hospitalized Participants With Coronavirus (COVID-19)||Phase 2/Phase 3 Interventional||PTC Therapeutics|
|NCT00508586||PTC299 and Hormonal Agent for Treatment of Metastatic Breast Cancer||Phase 1 Interventional||PTC Therapeutics|
|NCT03761069||Study of PTC299 in Relapsed/Refractory Acute Leukemias||Phase 1 Interventional||PTC Therapeutics|