Vamorolone Matches Deflazacort Cortisol Suppression Yet Minimizes Immunosuppression in Phase 1
CPRX•Catalyst’s Phase 1 trial in 60 healthy adults showed vamorolone at clinical doses (300 mg single dose or 9–27 mg/kg daily) achieves comparable cortisol suppression to deflazacort but with minimal immunosuppressive effects. Immunosuppression was only observed at supratherapeutic 40 mg/kg/day doses, supporting vamorolone’s safety profile for chronic inflammatory rare diseases.
1. Study Design and Objectives
The Phase 1 trial enrolled 60 healthy adult volunteers in two parts: Part A assessed equipotency between single-dose vamorolone (300 mg) and deflazacort (0.9 mg/kg), while Part B evaluated ascending daily doses of vamorolone (9, 27, 40 mg/kg) over seven days to gauge immunosuppressive potential and anti-inflammatory activity.
2. Part A Equipotency Results
In a randomized crossover of 24 subjects, vamorolone and deflazacort showed similar on-target glucocorticoid effects, including cortisol suppression, leukocyte redistribution and functional immune biomarker changes, with both drugs exhibiting a 2–4 hour onset. Vamorolone demonstrated less pronounced immunosuppressive biomarker suppression compared with deflazacort at clinical doses.
3. Part B Dose-Dependent Effects
Among 36 volunteers receiving 9, 27 or 40 mg/kg of vamorolone daily, clinically relevant immunosuppressive effects emerged only at the highest 40 mg/kg dose and persisted post-treatment. No significant immunosuppression occurred at 9 or 27 mg/kg, indicating a favorable safety margin for clinically approved vamorolone dosing.
