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Ceraxa is a fully synthetic, PEGylated nano-liposomal formulation of C6-ceramide developed by Keystone Nano, Inc. for the treatment of acute myeloid leukemia (AML). It functions by activating protein phosphatase 2A (PP2A), which leads to the dephosphorylation and inhibition of STAT3, thereby down-regulating pro-survival and pro-mitogenic signaling pathways in malignant cells. Additionally, Ceraxa has been shown to inhibit glycolysis, reduce tumor micro-vascularization, and counteract tumor-induced immunotolerance. It has been evaluated in a Phase 1 clinical trial for patients with relapsed or refractory AML to determine safety, tolerability, and pharmacokinetics.
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