VB-1197

VB-1197 is an investigational small-molecule therapy emerging from the CoMET modular platform, originally developed by Comet Therapeutics and now advanced by VectivBio/Ironwood Pharmaceuticals for severe inherited metabolic diseases characterized by impaired coenzyme A–dependent energy metabolism, particularly methylmalonic acidemia and propionic acidemia.[1][3][5][6][8][9] It is designed as a novel small-molecule modulator of cellular metabolism to correct core biochemical dysfunction in organic acidemias and related acidaemias, but its precise molecular target and mechanism of action have not yet been disclosed publicly.[1][3][6] VB-1197 remains in preclinical development and IND-enabling studies, with plans for first-in-human evaluation in pediatric and potentially broader patient populations affected by these ultra-rare metabolic disorders.[1][3][5][6][8][9][10][11]