4'-phosphopantetheine

4'-PPT (4'-phosphopantetheine) is an investigational small molecule being developed as a disease-modifying therapy for pantothenate kinase-associated neurodegeneration (PKAN). PKAN is a rare, autosomal recessive neurodegenerative disorder caused by mutations in the PANK2 gene, which encodes the enzyme pantothenate kinase 2. This enzyme is responsible for the first step in coenzyme A (CoA) biosynthesis. 4'-PPT is a downstream metabolic intermediate that bypasses the blocked PANK2 step, effectively restoring CoA levels and ameliorating the biochemical defects associated with the disease, such as iron accumulation and dopamine dysregulation. The drug is currently being evaluated in an investigator-initiated Phase II clinical trial led by the University Medical Center Groningen.

Other names

4'-phosphopantetheinephosphopantetheine