Amicus Therapeutics (FOLD) Reports Encouraging Data from AT2220 Phase 2
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Amicus Therapeutics (Nasdaq: FOLD), today announced positive preliminary results from an ongoing Phase 2 open-label drug-drug interaction study (Study 010) to evaluate the safety and pharmacokinetic (PK) effects of the pharmacological chaperone AT2220 (duvoglustat HCl) co-administered with enzyme replacement therapy (ERT) for Pompe disease.
AT2220 is an investigational oral pharmacological chaperone being developed exclusively by Amicus. Study 010 is investigating four ascending doses of AT2220 co-administered with the ERT alglucosidase alfa (Myozyme®/Lumizyme®), the first and only treatments approved for Pompe disease. In addition to safety and plasma PK effects, enzyme uptake into muscle tissue is being measured with and without AT2220.
Previous preclinical studies using acid alpha-glucosidase (GAA) knock-out mouse models of Pompe disease demonstrated that AT2220 co-administered with ERT increased the ERT uptake in key tissues of disease, including skeletal muscle and heart. This increased ERT uptake into muscle following AT2220-ERT co-administration corresponded in preclinical studies with greater reductions in muscle glycogen compared to ERT alone. Glycogen is the substrate that accumulates in the lysosomes of muscles in patients with Pompe disease.
Data from Study 010 are currently available for all patients enrolled in Cohorts 1 (n=4) and 2 (n=6), representing the two lowest dose groups of AT2220. Each patient received one infusion of ERT alone, and then a single oral dose of AT2220 just prior to the next ERT infusion. Muscle biopsies were performed seven days following each infusion in all four patients in Cohort 1. Among the six patients in Cohort 2, three had muscle biopsies three days after each infusion and three had muscle biopsies seven days after each infusion.
AT2220 is an investigational oral pharmacological chaperone being developed exclusively by Amicus. Study 010 is investigating four ascending doses of AT2220 co-administered with the ERT alglucosidase alfa (Myozyme®/Lumizyme®), the first and only treatments approved for Pompe disease. In addition to safety and plasma PK effects, enzyme uptake into muscle tissue is being measured with and without AT2220.
Previous preclinical studies using acid alpha-glucosidase (GAA) knock-out mouse models of Pompe disease demonstrated that AT2220 co-administered with ERT increased the ERT uptake in key tissues of disease, including skeletal muscle and heart. This increased ERT uptake into muscle following AT2220-ERT co-administration corresponded in preclinical studies with greater reductions in muscle glycogen compared to ERT alone. Glycogen is the substrate that accumulates in the lysosomes of muscles in patients with Pompe disease.
Data from Study 010 are currently available for all patients enrolled in Cohorts 1 (n=4) and 2 (n=6), representing the two lowest dose groups of AT2220. Each patient received one infusion of ERT alone, and then a single oral dose of AT2220 just prior to the next ERT infusion. Muscle biopsies were performed seven days following each infusion in all four patients in Cohort 1. Among the six patients in Cohort 2, three had muscle biopsies three days after each infusion and three had muscle biopsies seven days after each infusion.
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