CoA-Z

**CoA-Z (4'-phosphopantetheine)** is a vitamin B5 intermediate designed to treat Pantothenate Kinase-Associated Neurodegeneration (PKAN), a rare neurodegenerative disorder caused by mutations in the PANK2 gene that block pantothenate kinase activity, impairing coenzyme A biosynthesis. It bypasses this metabolic defect by providing a downstream precursor in the vitamin B5 pathway, restoring CoA production and normalizing disease biomarkers in blood in a dose-dependent manner, as demonstrated in preclinical models and a phase 1/2 clinical trial. Developed by the NBIAcure team led by Drs. Susan Hayflick and Penny Hogarth at Oregon Health & Science University (OHSU), CoA-Z showed excellent safety and tolerability across doses in a double-blind, placebo-controlled study, with plans for FDA submission in 2024.[1][2][3]

Other names

4'-phosphopantetheinephosphopantetheinepantetheine 4'-phosphatepantotheine-4'-phosphoric acidPSH-4'-P