AVTX-803 (elfucose)

AVTX-803 is a therapeutic dose of L-fucose currently in development for the treatment of leukocyte adhesion deficiency type II (LAD II), also known as SLC35C1-CDG.

Targeting Glycosylation Defects in LAD II

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.

LAD II is caused by loss of function mutations in the SLC35C1 gene encoding a GDP-fucose transporter found in the Golgi apparatus, where it acts as the donor substrate for protein fucosylation. Currently, there is no approved product for the treatment of LAD II.

Role of GDP-Fucose

AVTX-803 is an oral formulation of L-fucose that replenishes critical metabolic intermediates, reduced or absent due to a genetic variant, to support glycoprotein synthesis, maintenance, and function.

Attributes of AVTX-803

  • Orphan Drug designation, Fast Track Designation (FTD) and Rare Pediatric Disease designation
  • Established proof-of-concept in published literature
  • Potential for insurer reimbursement