AVTX-802

AVTX-802

AVTX-802

AVTX-802 is a therapeutic dose of D-mannose currently in development for the treatment of mannose phosphate isomerase (MPI) deficiency, also known as MPI-CDG.

Targeting Glycosylation Defects in MPI 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.

MPI deficiency is caused by a loss-of-function variant in the MPI gene encoding an enzyme responsible for the conversion of fructose-6-phosphate to mannose-6-phosphate, eventually providing guanosine diphosphate (GDP)-mannose as a donor substrate for glycoprotein synthesis. Currently, there is no approved product for the treatment of MPI-CDG.

Mannose Metabolism in Mammalian Cells

AVTX-802 is an oral formulation of D-mannose that replenishes critical metabolic intermediates, reduced or absent due to a genetic variant, to support glycoprotein synthesis, maintenance, and function. Therapy with AVTX-802 in patients with MPI deficiency should facilitate the synthesis of GDP-mannose via compensatory pathways, thereby restoring the ability to produce mannose-derived glycans.

Attributes of AVTX-802

AVTX-802
  • 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