Opitor-0

Opitor-0 is a small-molecule inhibitor of the mitochondrial dynamin-related protein Optic Atrophy 1 (OPA1), specifically targeting its GTPase activity. Developed through structure-activity relationship (SAR) studies of the lead compound MYLS22, Opitor-0 exhibits enhanced potency in promoting mitochondrial cristae remodeling and fragmentation, which facilitates the release of cytochrome c and triggers apoptosis. In preclinical models, Opitor-0 has demonstrated the ability to overcome resistance to Bcl-2 inhibitors, such as venetoclax, in acute myeloid leukemia (AML) and to selectively target metastatic breast cancer cells by exploiting their dependency on OPA1-mediated mitochondrial adaptations. By inhibiting OPA1, the drug disrupts mitochondrial structural integrity and metabolic flexibility, sensitizing malignant cells to programmed cell death and ferroptosis.

Other names

Opa1 inhibitor 0Opa-1 inhibitor 0Opa 1 inhibitor 0