The U.S. Food and Drug Administration has authorized the initiation of a Phase 2/3 trial to investigate PTC299, a dihydroorotate dehydrogenase (DHODH) inhibitor, as a potential treatment for COVID-19. PTC299 is an oral investigational drug with a novel dual-mechanism of action that has the potential to address the two critical elements of COVID-19:
- High viral replication
- Uncontrolled inflammatory response that ensues after infection
“We are excited about the potential of PTC299 to be part of the solution to this unprecedented global public health crisis and have made it a high priority within our organization.” – CEO, Stuart Peltz, Ph.D.
PTC299 is also being developed for treatment in oncological indications and has been extensively studied in nine clinical trials in over 300 individuals, including both oncology patients and healthy volunteers. In addition, PTC299 is currently being studied in a clinical trial in acute myeloid leukemia (AML) patients. In a PTC299 clinical study, results showed that PTC299 inhibited DHODH, confirming the mechanism of action in patients.