Devimistat is a first-in-class agent that targets enzymes in the mitochondria of cancer cells that affect cancer cell energy metabolism, resulting in tumor cell apoptosis and necrosis. Based on the performance of the drug in multiple disease states, the FDA has granted it orphan drug designation in pancreatic cancer, acute myeloid leukemia, myelodysplastic syndrome, peripheral T-cell lymphoma, and Burkitt’s lymphoma. The drug also has orphan drug designation from the European Medicines Agency (EMA). Read more . . .
A pivotal multicenter, open-label, randomized phase III trial (AVENGER 500, NCT03504423) has been initiated to evaluate the efficacy and safety of the novel mitochondrial inhibitor, devimistat (CPI-613) in combination with a modified FOLFIRNOX regimen for the first-line treatment of patients with metastatic pancreatic cancer, according to a press release from Rafael Pharmaceuticals.1