ReveraGen BioPharma has released initial results from their clinical trial of Vamorolone, a ‘designer corticosteroid’ with the potential to cause fewer side effects and produce greater benefits than other steroids that are commonly prescribed to slow the progression of Duchenne muscular dystrophy.
The trial is involving 48 young boys with Duchenne worldwide, and thanks to early funding from Sydney-based Save Our Sons Duchenne Foundation, this included six Australians who have been given the opportunity to access this promising therapy early.
Boys in the trial took one of four different doses of the new drug Vamorolone. Preliminary results after six months of treatment show that those who took either of the two highest doses had increased muscle strength and endurance in a range of measures including timed walking tests. The magnitude of improvement is similar to the traditionally prescribed steroid prednisolone, but importantly, there have been signs of reductions in the commonly experienced side effects such as weight gain, bone fragility, adrenal suppression and insulin resistance which can lead to diabetes.
“Steroids such as prednisolone are the only widely available treatment to slow down the progression of muscle weakness in boys with Duchenne, however, the side effects have a major impact on the boys’ quality of life, often leading to the hard decision to discontinue treatment. A steroid with minimal side effects will make a huge difference to families affected by Duchenne” said Dr Kristina Elvidge, Save Our Sons Duchenne Foundation’s Research Affairs Manager.
A phase 2b trial has been launched that will enroll 120 boys with Duchenne internationally (including Australia) to further study the benefits and side effects of Vamorolone.
Vamorolone has been developed under a venture philanthropy model, with funding from more than a dozen international non-profit foundations.
Vamorolone (previously VBP15) binds to the same cellular receptors as traditional glucocorticoid drugs, but unlike these, does not enable dimerization of the drug/receptor complexes. This leads to a separation (dissociation) of anti-inflammatory benefit from safety concerns. In published Phase I studies in healthy adult volunteers, vamorolone showed reduction or loss of most side effects of glucocorticoids, as measured by blood biomarkers over a 2-week treatment period. Vamorolone has been granted Orphan Drug status by both FDA and EMA, and received Fast Track designation by the FDA.
- More information about Duchenne https://www.saveoursons.org.au/what-is-dmd/
- Read the ReveraGen press release http://www.reveragen.com/news/
- Donate to support the work of Save Our Sons Duchenne Foundation https://www.saveoursons.org.au/donations/donate-now/