CERC-801
Targeting the Glycosylation Defects Associated with Phosphoglucomutase 1 (PGM1) Deficiency
CERC-801 is an ultra-pure oral formulation of D-galactose currently in development for the treatment of phosphoglucomutase 1 (PGM1) deficiency, also known as PGM1-CDG.
About PGM1 Deficiency
Currently there is no approved product for the treatment of PGM1-CDG.
PGM1 Deficiency is caused by loss of function mutations in the PGM1 gene encoding an enzyme responsible for the interconversion of glucose-6-phosphate to glucose-1-phosphate. Glucose-1-phosphate can be utilized to supply UDP-galactose, a substrate that donates galactose subunits for glycoprotein synthesis.
We believe that substrate replacement with CERC-801 in patients with PGM1 deficiency should increase available UDP-galactose pools and repair galactose-deficient glycoproteins.
Mechanism of Action
CERC-801 is an ultra-pure formulation of D-galactose that serves as a substrate replacement therapy. Oral administration of D-galactose replenishes critical metabolic intermediates, reduced or absent due to a genetic mutation, to support glycoprotein synthesis, maintenance, and function.