PA is caused by a dysfunctional or missing enzyme called propionyl CoA carboxylase. Without this enzyme functioning normally, people born with this disease are unable to normally convert food into energy. This decrease of energy production – or energy deficit – hinders the ability of cells in the body to function and grow properly, and may also causes a buildup of unmetabolized fats and proteins that can become toxic at high levels.
IMB-202 is intended to directly supply the body with substrates to replenish missing components required for energy metabolism.
IMB-202 is a novel oral citrate formulation designed to enable large quantities of citrate to be delivered without the high salt load that comes from current formulations. Therapeutic quantities of citrate have been shown to correct deficits of multiple chemical intermediates that play a role in how cells generate energy.