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CC-4066 is a small molecule dual inhibitor of Cyclophilin A and Cyclophilin D proteins, developed by Cypralis for the prevention and treatment of acute organ injury. Administered intravenously, CC-4066 targets Cyclophilin D to increase the threshold for mitochondrial permeability transition pore (mPTP) opening, a critical step in cell death pathways. By stabilizing mitochondria, the drug protects cells from both apoptotic and necrotic death associated with ischemia, reperfusion, and toxins. Clinical development has focused on acute kidney injury (AKI) following major surgery (Phase 1) and delayed graft function (DGF) in kidney transplantation (Phase 2), with additional preclinical exploration in acute pancreatitis.
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