Monosaccharide substrate replacement therapy for an ultra-rare congenital disorder of glycosylation


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.

of CERC-801

  • High purity and quality
  • Ease of use and administration
  • Documented efficacy and safety in published literature
  • Orphan Drug and Rare Pediatric Disease designation
  • Potential for insurer reimbursement

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.