Vamorolone trial in Duchenne muscular dystrophy shows dose-related improvement of muscle function

Objective: We carried out first-in-patient studies of vamorolone, a first-in-class dissociative steroidal anti-inflammatory drug, in Duchenne muscular dystrophy. Methods: An open-label, multiple-ascending dose study of vamorolone was conducted in 48 boys with Duchenne muscular dystrophy (4 to <7 years, steroid-naïve). Dose levels were 0.25, 0.75, 2.0 and 6.0 mg/kg/day using an oral suspension formulation (12 boys/dose level; 1/3rd to 10x glucocorticoid dose in DMD). Results: Over a 24-week treatment period, oral administration of vamorolone at all doses tested was safe and well-tolerated. The 2.0 mg/kg/day dose group met the primary efficacy outcome of improved muscle function (time to stand; 24 weeks vamorolone treatment versus natural history controls), without evidence of most adverse effects of glucocorticoids. Significant dose-responsive improvements in 10 meter run/walk, and six-minute walk test were observed for 2.0 and 6.0 mg/kg/day dose groups. The morbidity of most con...