For the Treatment of
Propionic Acidemia

IMB-202 is an oral substrate replacement therapy being studied for the treatment of propionic acidemia (PA), a rare and potentially devastating mitochondrial disease caused by an inborn error of metabolism. It is intended to deliver therapeutic quantities of a novel citrate formulation in order to replenish missing chemical intermediates necessary for normal energy metabolism.

PA can be debilitating, and may lead to damage of the heart, brain or kidneys, developmental delays, or even, tragically, death. Despite the severity of the disease, there are no approved therapies in the United States for the treatment of propionic acidemia.

IMB-202’s Mechanism of Action and Development Status

IMB-202’s Mechanism of Action

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.

IMB-202’s Development Status

In preclinical studies, IMB-202 was readily absorbed after oral administration and was found widely distributed in tissues throughout the body within one hour. After 24 hours, the majority of IMB-202 had been converted to CO2 and expelled by respiration from the body, consistent with metabolism in the tricarboxylic acid (TCA) cycle.

We intend to initiate an open-label Phase 1/2 clinical trial of IMB-202 in patients with PA in the first half of 2020.