BBP-671

BBP-671 is an investigational oral small molecule designed to increase coenzyme A (CoA) levels by allosterically modulating pantothenate kinases, which are key enzymes in the CoA biosynthesis pathway. It was developed as a potential therapy for diseases characterized by CoA metabolism deficiencies, including propionic acidemia (PA), methylmalonic acidemia (MMA), and pantothenate kinase-associated neurodegeneration (PKAN). The drug demonstrated the ability to cross the blood-brain barrier and increase acetyl-CoA levels in clinical studies, supporting its mechanism of action. However, development for PKAN was discontinued due to challenges with dose variability and safety margins.