Vamorolone is being developed for Duchenne muscular dystrophy. While vamorolone may show improved efficacy and safety profiles in many other disorders, we chose DMD as the initial indication due to commitment of the founders for this disease, and the effective partnerships with muscular dystrophy foundations worldwide.
Vamorolone was tested in 86 healthy adult men in 2015. This showed safety through 20 mg/kg/day, or about 30 times the prednisone dose prescribed to DMD boys. The Phase 1 study also showed loss of typical side effects of traditional corticosteroids such as prednisone and deflazacort through the study of blood biomarkers (adrenal suppression, insulin resistance, bone turnover, immune suppression).
The DMD clinical trial program was developed with guidance from the FDA (Phase 1 – Phase 2 transition meeting), EMA CHMP (two rounds of scientific guidance), and the EMA Paediatric Investigation Plan (PIP). The planned clinical trials are shown in the schematic below.