Effect of AT2220 co-administration on rhGAA-mediated glycogen reduction (% change from Baseline) in <i>GAA</i> KO mice.

Twelve-week old male GAA KO mice were administered vehicle (water) or AT2220 via oral gavage once every other week for 8 weeks. Thirty minutes after each AT2220 oral administration, rhGAA (20 mg/kg) was administered via bolus tail vein injection. Mice were euthanized 21 days after the last (i.e., 4th) rhGAA administration, and tissue glycogen levels were measured as described in ‘Materials and Methods’. Baseline glycogen levels in untreated GAA KO mice were 417±32, 340±24, 405±27, 353±21, 446±18, and 389±10 µg/mg protein in heart, diaphragm, quadriceps, gastrocnemius, triceps, and tongue, respectively, and in wild-type C57BL/6 mice were 23±2, 50±6, 40±5, 45±2, 35±7, and 32±2 µg/mg protein, respectively (mean±SEM of 7 mice). The data shown represent the percent glycogen change from baseline in each tissue as normalized between wild-type (0%) and GAA KO (100%) levels. Each value represents the mean±SEM of 12 mice. Statistically significant reductions were seen in glycogen levels compared to baseline (*p<0.05, t-test) and compared to rhGAA administration alone (#p<0.05, t-test).