Santhera Pharmaceuticals and ReveraGen announce that JAMA Neurology has published positive results of the 24-week primary efficacy and safety analysis from the VISION-DMD study evaluating vamorolone, an investigational drug for the treatment Duchenne muscular dystrophy (DMD). Vamorolone met its primary endpoint by demonstrating statistically significant and clinically relevant improvement in time to stand from floor compared to placebo, the first functional milestone to deteriorate in young children with DMD. Consistent results across multiple secondary endpoints support the results of the primary endpoint.

The relative efficacy of vamorolone 6 mg/kg/day was comparable to that seen with prednisone 0.75 mg/kg/day across primary and secondary efficacy endpoints. Over the 24-week treatment period, no negative impact on biomarkers of bone health and no loss of linear growth were observed with vamorolone. Vamorolone was generally safe and well tolerated.

The most commonly reported adverse events versus placebo study were cushingoid features, vomiting and vitamin D deficiency. Adverse events were generally of mild to moderate severity.