EPI-743 is an orally absorbed small molecule that readily crosses into the central nervous system. It works by targeting an enzyme NADPH quinone oxidoreductase 1 (NQO1).
As of June 1, 2011 EPI-743 has been administered to 40 subjects diagnosed with genetically confirmed inherited respiratory chain disease of the mitochondria that were either at end-of-life or at risk for blindness.
EPI-A0001 is an orally absorbed small molecule. EPI-A0001 has completed phase 1A and phase 1B studies in healthy volunteers and phase 2 in the mitochondrial disease Friedreich's ataxia.