Vamorolone Has Efficacy for Treating Duchenne Muscular Dystrophy Without Affecting Growth

In the VISION-DMD trial (NCT02760277), children with Duchenne muscular dystrophy (DMD) treated with vamorolone (ReveraGen BioPharma, Rockville, MD), had improved motor function with no negative effects on linear growth or biomarkers of bone health. Results of this randomized, placebo-controlled, 24-week clinical trial were published in JAMA Neurology. 

Children treated with 6 mg/kg/day vamorolone vs placebo had mean time-to-stand velocity improvement of 0.05±0.01 meters/sec vs -0.01±0.01 (95% CI, 0.02-0.10; P=.002). Improvements were also observed on the 6-minute walk test and velocity to run or walk 10 meters with vamorolone vs placebo. The relative efficacy of vamorolone 6mg/kg/day was comparable to that seen with prednisone 0.75mg/kg/day across primary and secondary efficacy endpoints. 

Prednisone, considered the current standard of care, was used as an active comparator and also improved motor function vs placebo. As expected, however, growth was slowed in those treated with prednisone, resulting in -1.88±8.81 percentile drop. With 6 mg/kg/day vamorolone, in contrast, children had a growth percentile increase of +3.86 ±6.16 (P=.02). Bone turnover markers declined with prednisone but not with vamorolone. At baseline, participants had low adrenocorticotropic hormone (ACTH)-stimulated cortisol and high incidence of adrenal insufficiency, which increase in all 3 treatment groups over the 24-week trial. 

“Data from the VISION-DMD study continues to validate our ambition of developing a steroidal-like treatment where we can retain the efficacy of traditional corticosteroids and reduce some of the toxicities that all too often lead to the premature discontinuation of treatment in children with DMD,” said Eric Hoffman, PhD, professor of pharmaceutical sciences, Binghamton University-SUNY and CEO of ReveraGen. 

Vamorolone is a structurally unique dissociative steroidal anti-inflammatory drug. Participants in the study were age 4 to 7, identified as male in the study report, with genetically confirmed DMD and no prior treatment with corticosteroids. The most commonly reported adverse events versus placebo were cushingoid features, vomiting, and vitamin D deficiency. Adverse events were generally of mild-to-moderate severity.