Amicus Therapeutics preclinical data

In acid alpha glucosidase (GAA) knockout mice, AT2220 co-formulated with recombinant human acid alpha glucosidase ( rhGAA) enzyme replacement therapy (ERT) given via IV bolus injection every other week for 8 weeks significantly increased rhGAA uptake by up to 2.5-fold in multiple disease-relevant tissues, including the heart, tongue, diaphragm and skin, at day 7 vs. both untreated controls and ERT alone (p<0.05 for all). Additionally, IV AT2220 co-formulated with ERT led to a significantly greater reduction in glycogen levels in the heart and tongue from baseline to day 21 vs. ERT alone (p<0.05 for all). Data were presented at the Lysosomal Disease Network WORLD Symposium in Orlando. ...