Vamorolone Safety, Pharmacokinetics, and Exploratory Efficacy in Duchenne Muscular Dystrophy

Figures

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  Figure 1. Study Design for the 2–<4-Year-Old CS-Naive DMD Cohort in VBP15-006, Including EAP Canada The 6 mg/kg/d group started after completion of the 2 mg/kg/d group. Both studies were registered on ClinicalTrials.gov: VBP15-006 open-label study (NCT05185622), and EAP Canada study (NCT03863119). CS = corticosteroid; DMD = Duchenne muscular dystrophy; EAP = expanded access protocol.

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  Figure 2. Growth Trajectories From Baseline of VBP15-006 to Last Available Observation in EAP Canada (A) Height z-scores over time from VBP15-006 baseline to last available data in EAP Canada (N = 19). (B) Individual height trajectories with CDC growth charts (source: Centers for Disease Control and Prevention)21 (left) or DMD-specific growth curves22 (right) as a reference22; 1 value was removed for a patient with erroneously recorded data. (C) BMI z-scores over time from VBP15-006 baseline to last available data in EAP Canada (N = 19). (D) Individual BMI trajectories with CDC growth charts (source: Centers for Disease Control and Prevention)21 (left) or DMD-specific growth curves22 (right) as a reference22; 1 value was removed for a patient with erroneously recorded data. BMI = body mass index; CBL = change from baseline; CDC = Centers for Disease Control and Prevention; DMD = Duchenne muscular dystrophy; EAP = expanded access protocol; Q = quartile.

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  Figure 3. Morning Serum Cortisol Levels Measured in VBP15-006 After 12 Weeks' Vamorolone Treatment (A) Morning serum cortisol at baseline and week 12, and (B) individual patient morning serum cortisol levels between baseline and week 12. *Two patients in the 6 mg/kg/d group did not have baseline cortisol values available. Q = quartile.

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  Figure 4. Pharmacokinetics of Vamorolone at Day 1 and Week 2 in VBP15-006 Individual plasma concentration measurements at day 1 and week 2 for both dose groups. (A) 2 mg/kg/d. (B) 6 mg/kg/d. Data are shown on a semi-log scale (natural log); each y-axis tick mark represents a 2.7-fold change in concentration. At day 1, n = 10 for both dose groups; at week 2, n = 9 for both dose groups (exception is predose measurement in 6 mg/kg/d group where n = 10).

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  Figure 5. Change in Bayley-III Gross Motor Scaled Score From Baseline to Week 12 in VBP15-006 Individual scores between baseline and week 12 for (A) 2 mg/kg/d (n = 9) and (B) 6 mg/kg/d (n = 10) vamorolone groups. In the 2 mg/kg/d group, 1 patient is not shown due to missing data, and 2 patients have overlapping data. CBL = change from baseline; Q = quartile.