Inborn Errors of Metabolism
Diseases that arise from inborn errors of metabolism are characterized by deficiencies in metabolic enzymes that alter the production of substrates that are critical for proper functioning of the tricarboxylic acid, or TCA, cycle, a central pathway used to extract energy stored in carbohydrates, fats, and proteins into forms that can be used by cells.
Left untreated, inborn errors of metabolism can lead to significant morbidity, progressive neurologic injury, or death. Despite the potential of these diseases to severely shorten a patient’s life, there are very few treatment options.
Mitochondrial substrate replacement is the most promising therapy for the treatment of patients with certain inborn errors of metabolism. IMB-203 is intended to directly supply the body with depleted substrates by the oral or gastro-intestinal tube route, thereby bypassing the need for the internally generated substrates via the affected metabolic pathways.
IMB-203 is designed to allow high levels of succinate to be delivered to cells throughout the body. The ability to deliver succinate to cells should allow cells to continue using the TCA cycle for metabolism, by replenishing the energetic deficits caused by enzyme deficiencies in certain inborn errors of metabolism.