Exhibit 99.1
Phase 1 MAD Study Clinical Activity Significant and prolonged FXN mRNA increase in skeletal muscle Muscle FXN mRNA response correlated with dose and muscle DT-216 exposure, p < 0.05* Cohorts: 200mg and 300mg Muscle FXN mRNA (mean % change from baseline) p < 0.05** NS** 75th% FA 40 20 0 -20 -40 2 Days 7 Days Days After 3rd Weekly Dose Pooled Placebo (n=6) DT-216 200mg (n=7) DT-216 300mg (n=7) Individual FA Patients Muscle FXN mRNA (% change from baseline) 125 100 75 50 25 0 -25 -50 -75 Lower quartile in healthy Carriers Upper quartile FA patients 50th% Ca 25th% Ca 10th% Ca 90th% FA 75th% FA 50th% FA 25th% FA 2 Days After 3rd Weekly Dose Note: Based upon data transfer of August 7th 2023 * Exploratory analyses for dose-response and exposure-response were conducted using a non-parametric trend test and non-parametric correlation test, respectively. ** Exploratory analyses were conducted using a non-parametric Wilcoxon Rank-Sum model. A parametric ANCOVA model gave similar results. Bars represent standard error of the mean. NS, not significant. Percentiles and quartiles assume individual FA patient baselines in the MAD study are the median FA patient FXN mRNA value from the observational muscle biopsy study. Copyright © 2023 by Design Therapeutics, Inc. All rights reserved 1