AT2220 Co-Formulated with Amicus Proprietary Next-Generation ERT
Amicus is combining its core pharmacological chaperone technology with advanced biologics capabilities to create a next-generation Pompe ERT. The Company is designing this co-formulated chaperone-ERT product with the goal of increasing exposure and tissue uptake and reducing immunogenicity of current ERTs. The co-formulation with AT2220 may also allow the ERT to be administered through novel routes. Amicus has entered into a contract with Laureate Pharmaceuticals for the contract manufacture of this next-generation ERT. Additional details regarding this aspect of the Amicus Pompe program will also be provided during the presentation and live webcast at the JPMorgan conference.
About Amicus Therapeutics
Amicus Therapeutics (Nasdaq:FOLD) is a biopharmaceutical company at the forefront of developing therapies for rare and orphan diseases. The Company is developing orally-administered, small molecule drugs called pharmacological chaperones, a novel, first-in-class approach to treating a broad range of human genetic diseases. Amicus' late-stage programs for lysosomal storage disorders include migalastat HCl monotherapy in Phase 3 for Fabry disease; migalastat HCl co-administered with enzyme replacement therapy (ERT) in Phase 2 for Fabry disease; and AT2220 co-administered with ERT in Phase 2 for Pompe disease.About Migalastat HCl for Fabry Disease Amicus in collaboration with GlaxoSmithKline (GSK) is developing the investigational pharmacological chaperone migalastat HCl for the treatment of Fabry disease. Amicus has commercial rights to all Fabry products in the United States and GSK has commercial rights to all of these products in the rest of world. As a monotherapy, migalastat HCl is designed to bind to and stabilize, or "chaperone" alpha-galactosidase A (alpha-Gal A) enzyme in patients with genetic mutations that are amenable to this chaperone in a cell-based assay. For patients currently receiving ERT for Fabry disease, migalastat HCl in combination with ERT may improve ERT outcomes by keeping the infused alpha-Gal A enzyme in its properly folded and active form.