CERC-913 is a nucleotide prodrug being developed for an ultra-rare mitochondrial DNA depletion syndrome (MDS) known as Deoxyguanosine Kinase (DGUOK) deficiency.
CERC-913 incorporates ProTide prodrug chemistry to overcome the key limitations of direct substrate replacement. This approach has been successfully utilized in several approved drugs, including Sovaldi (Sofosbuvir).
ProTide nucleotides have been shown to significantly increase plasma stability and cell permeability relative to the parent compound and have also demonstrated the ability to bypass nucleoside kinases to circumvent the rate limiting step in nucleic acid synthesis.
CERC-913 has demonstrated proof-of-concept in patient-derived & animal-based disease models.
CERC-913 is a ProTide prodrug of deoxyguanosine monophosphate (dGMP) designed to act as a substrate replacement therapy. Oral administration of CERC-913 is expected to increase intracellular availability of dGMP for mitochondrial DNA (mtDNA) synthesis, particularly in the liver.