OKN-007

OKN-007 is an investigational small molecule drug developed as a novel anti-cancer agent for the treatment of high-grade gliomas, particularly glioblastoma (GBM) and diffuse intrinsic pontine glioma (DIPG). Chemically described as a disulfonyl derivative of α-phenyl-tert-butyl nitrone (PBN), it acts primarily by eliminating reactive oxygen species and modulating tumor microenvironment pathways. Preclinical studies have shown that OKN-007 reduces tumor cell proliferation and angiogenesis while increasing apoptosis in various animal models of adult and pediatric high-grade gliomas. Mechanistically, it downregulates genes associated with the extracellular matrix via inhibition of the transforming growth factor β1 (TGF-β1) pathway. It also temporarily opens the blood-brain barrier to enhance drug delivery to tumors and alters gene expression in tumor cells. Clinical trials have demonstrated a favorable safety profile with minimal side effects when administered intravenously or orally in patients with recurrent or newly diagnosed high-grade gliomas[2][3][5][6][9].

Brand names

Cerovive

Other names

disodium 4-[(tert-butyl-imino) methyl] benzene-1,3-disulfonate N-oxide2,4-disulfophenyl-N-tert-butyl nitrone