During period 1 (first 24 weeks) of the study recruited boys received either vamorolone, prednisolone or placebo. The last patient last visit has now been completed in the first treatment period of the Phase 2b study. The aim of this pivotal placebo and active controlled 24-week study is to demonstrate the efficacy and safety of vamorolone administered orally at doses of 2.0 mg/kg/day and 6.0 mg/kg/day versus prednisone 0.75 mg/kg/day and placebo. The efficacy outcome measures are motor function and strength outcomes with Time to Stand test (TTSTAND) as the primary study endpoint. Additional analyses compare safety and tolerability between the vamorolone dose groups, placebo and prednisone.
On March 3rd 2021 Santhera and ReveraGen announced the completion of the first 6-month period of this pivotal VISION-DMD trial (see Press Release). Topline 6-month data are expected in Q2-2021, paving the way for a US NDA submission in Q1-2022. Vamorolone has been granted Orphan Drug status in the US and in Europe, and has received Fast Track and Rare Pediatric Disease designations by the US FDA and Promising Innovative Medicine (PIM) status from the UK MHRA.
During treatment period 2 (week 28 to week 48) another 24 weeks of continued study will be conducted. All boys will receive vamorolone at either 2.0 or 6.0 mg/kg/day. The patients who previously received prednisone or placebo have been randomized and switched to one of the two doses of vamorolone. This second treatment period is designed to evaluate the persistence of the drug effect in the longer term. In addition to efficacy, the study aims to confirm the differentiated safety and favourable tolerability profile of vamorolone. The results will demonstrate the potential of vamorolone to offer an alternative to current standard of care.