Drug intelligence / Profile preview

devimistat

Development stage
Phase 3
Lead developer
Cornerstone Pharmaceuticals
Modality
Small Molecules
Administration
Intravenous
01

Overview

Devimistat is a synthetic lipoic acid analogue and a first-in-class small molecule that targets cancer cell metabolism. It acts as an anti-mitochondrial agent by selectively inhibiting key mitochondrial enzymes—pyruvate dehydrogenase (PDH) and alpha-ketoglutarate dehydrogenase (KGDH)—which are central to the tricarboxylic acid (TCA) cycle. By blocking these enzymes, devimistat disrupts the entry of glucose and glutamine-derived carbons into the TCA cycle, collapsing mitochondrial metabolism in tumor cells and inducing cell death pathways. Devimistat is being developed primarily for hard-to-treat cancers such as metastatic pancreatic cancer and relapsed or refractory acute myeloid leukemia (AML), with ongoing clinical trials also in myelodysplastic syndromes (MDS), peripheral T-cell lymphoma, Burkitt's lymphoma, solid tumors including biliary tract cancer, and small cell lung cancer. The drug has received orphan drug designation from the FDA for several indications[1][2][3][4][5][6][7][8].

Brand names
CPI-613CPI613CPI 613
Other names
6,8-bis-benzylsulfanyl-octanoic acid6,8-bis(benzylthio)octanoic acidOctanoic acid, 6,8-bis((phenylmethyl)thio)-
02

Targets

OGDH (Dihydrolipoamide S-succinyltransferase)DLAT (Dihydrolipoamide S-acetyltransferase)α-KGDH (Alpha-ketoglutarate dehydrogenase complex)PDH (Pyruvate dehydrogenase complex)

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