PPI-1040

PPI-1040 is an orally bioavailable, synthetic vinyl-ether plasmalogen precursor in development by Med-Life Discoveries for the treatment of Rhizomelic Chondrodysplasia Punctata (RCDP). RCDP is an ultra-rare genetic disorder characterized by severe plasmalogen deficiency due to mutations in peroxisomal genes. Unlike other precursors that require metabolic conversion by peroxisomal enzymes, PPI-1040 contains a fully intact and stabilized vinyl-ether bond, allowing it to directly replace or augment deficient plasmalogens without requiring metabolic conversion. In preclinical models, PPI-1040 has been shown to normalize circulating plasmalogen levels and improve hyperactive behaviors.

Other names

1-(((Z)-hexadec-1-en-1-yl)oxy)-3-((2-oxido-1,3,2-oxazaphospholidin-2-yl)oxy)propan-2-yl (4Z,7Z,10Z,13Z,16Z,19Z)-docosa-4,7,10,13,16,19-hexaenoatesynthetic plasmalogen replacementsynthetic vinyl-ether plasmalogen precursorvinyl-ether plasmalogen precursor