The double-blind, randomised, placebo-controlled trial will analyse the safety, tolerability, and exploratory clinical efficacy of vamorolone versus a placebo on motor function outcomes for 24 weeks.
It will enrol 39 male subjects aged between 18 years and less than 65 years with BMD.
Subjects will be categorised into a 2:1 ratio to receive 500mg vamorolone daily or a placebo at sites in Pittsburgh, US, and Padova, Italy.
Under the “Clinical Studies of Orphan Products Addressing Unmet Needs of Rare Diseases (R01)” grants programme, the US Food and Drug Administration (FDA) is extending a $1.2m fund for the trial.
Apart from the FDA fund, the US National Institutes of Health (NIH) unit National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS), and the Foundation to Eradicate Duchenne, are extending financial support to conduct the trial.
ReveraGen BioPharma president and CEO Eric Hoffman said: “There are currently no approved drugs for BMD in any country, and no drugs in clinical development for this indication in the US or Europe.
“Vamorolone has shown efficacy in the pivotal VISION-DMD study in Duchenne muscular dystrophy (DMD), a more severe but related disease, and, based on these findings and its mechanisms of action, this developmental compound may show a benefit in BMD.”
Deemed as a dissociative anti-inflammatory drug, vamorolone attaches to the same receptor as corticosteroids but alters its downstream activity.
This mechanism of the drug aids in ‘dissociating’ efficacy from standard steroid safety concerns and could become a potential substitute for current corticosteroids, the existing standard of care for Duchenne muscular dystrophy (DMD) in children and adolescents.
Santhera plans to conclude the rolling submission of the new drug application (NDA) to the US FDA for vamorolone in DMD in the fourth quarter of this year.
BMD is a progressive muscle wasting disease.