Vamorolone (VBP15), developed by ReveraGen Biopharma, is a first-in-class dissociative steroidal drug that retains anti-inflammatory activities, but shows significant decrease in the frequencies of corticosteroid side effects.  We present the results of an open-label, multiple ascending dose (0.25, 0.75, 2.0, and 6.0 mg/kg/day) study in 48 boys with DMD, age 4 to <7 years. Participants did not have prior treatment with glucocorticoids. The initial two-week treatment period and two-week washout period was followed by a 24-week treatment study. Upon completion, participants could enroll in a 2-year long-term extension study of vamorolone treatment.

Primary outcomes were tolerability, clinical safety (change in body mass index) compared to prednisone-treated historical DMD controls and efficacy (change in time to stand) compared to untreated historical DMD controls. Secondary and exploratory outcomes included safety and efficacy pharmacodynamic biomarkers and other functional measures.

Vamorolone was well-tolerated. Pharmacodynamic biomarkers confirmed improved safety of vamorolone compared to glucocorticoids (absence of insulin resistance, reduction in adrenal suppression, beneficial changes in bone turnover) at 2 weeks of treatment.

The study showed dose-related improvements of multiple tests of strength and endurance.  Clinical efficacy was demonstrated at the 2 and 6 mg/kg/day doses compared to steroid-naïve participants in the Duchenne Natural History Study (CINRG).

The studies to date support further research testing vamorolone as a safer alternative to glucocorticoids for the treatment of many conditions where long term use leads to debilitating side effects.

Vamorolone has been developed under a venture philanthropy model, with funding from international non-profit foundations, US and EU governments.