About AT2220 for Pompe Disease
AT2220 is an investigational, orally-administered pharmacological chaperone owned exclusively by Amicus. The Company has completed a Phase 2 study ( Study 010) of AT2220 (duvoglustat HCl) co-administered with the ERT alglucosidase alfa (Myozyme/Lumizyme) in individuals with Pompe disease. Published preclinical data 1 suggest that AT2220 in combination with this ERT may improve rhGAA enzyme activity, reduce glycogen accumulation, and potentially mitigate ERT-related immunogenicity in patients with Pompe disease.
Pompe disease is a lysosomal storage disease characterized by progressive skeletal muscle weakness and respiratory insufficiency. It is caused by a deficiency in GAA activity, which leads to accumulation of glycogen in tissues affected by the disease (primarily muscle). Pompe disease affects an estimated 5,000 to 10,000 individuals worldwide and is clinically heterogeneous in the age of onset, the extent of organ involvement, and the rate of progression. Myozyme and Lumizyme (alglucosidase alfa, or recombinant human GAA enzyme, rhGAA) are the first and only approved treatments for Pompe disease. The clinical benefit of Myozyme and Lumizyme may be limited by low stability of the recombinant enzyme at neutral pH and body temperature, modest tissue uptake, and immune responses that affect tolerability and efficacy. Immune responses in the form of antibodies to rhGAA occur in a majority of Pompe patients receiving Myozyme/Lumizyme infusions 3 and may limit treatment outcomes with ERT.
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1. Khanna R, et al., The Pharmacological Chaperone AT2220 Increases Recombinant Human Acid α-Glucosidase Uptake and Glycogen Reduction in a Mouse Model of Pompe Disease., PLoS ONE (2012) 7(7): e40776. doi:10.1371/journal.pone.0040776.2. Banati M, et al., Enzyme Replacement Therapy Induces T-cell Responses in Late-Onset Pompe Disease., Muscle Nerve. 2011 Nov;44(5):720-6. 3. Lacana E, et al., The Role of Immune Tolerance Induction in Restoration of the Efficacy of ERT in Pompe Disease. , Am J Med Genet C Semin Med Genet. 2012 160C:30-39