AVTX-801

AVTX-801

AVTX-801

AVTX-801 is a therapeutic dose of D-galactose currently in development for the treatment of phosphoglucomutase 1 (PGM1) deficiency, also known as PGM1-CDG.

Targeting Glycosylation Defects in PGM1 Deficiency

CDG represent a large group of rare inherited diseases affecting glycosylation, the process of adding sugar-building blocks (glycans) to proteins or lipids. CDG are typically present from infancy and can affect any part of the body, with symptoms that range from mild to potentially life-threatening. Individual CDG are caused by mutations in a specific gene.

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 uridine diphosphate (UDP)-galactose, a substrate that donates galactose subunits for glycoprotein synthesis. Currently, there is no approved product for the treatment of PGM1-CDG.

Role of PGM1 in Sugar Metabolism

AVTX-801 is an oral formulation of D-galactose that replenishes critical metabolic intermediates to support glycoprotein synthesis, maintenance, and function.

Attributes of AVTX-801

AVTX-801
  • Orphan Drug designation and Rare Pediatric Disease designation; eligible for Priority Review Voucher upon approval
  • Established proof-of-concept in published literature
  • Potential for insurer reimbursement