Breaking news: Duchenne UK backed drug shows success in clinical trial Breaking News: Duchenne UK backed drug shows success in clinical trials Duchenne UK is delighted to announce that Vamorolone, a drug to treat Duchenne muscular dystrophy (DMD), has shown positive results in a Phase 2 Study. Vamorolone was developed as an alternative medicine to corticosteroids, that offers the benefits of steroids, but without some of the side effects that can have a serious impact on patients’ quality of life. The results from Santhera Pharmaceuticals and ReveraGen BioPharma showed that boys on Vamorolone did better than those taking a placebo. It also found that Vamorolone had a better safety profile than Prednisolone, one of the corticosteroids prescribed to DMD patients. Duchenne UK, Joining Jack and the Duchenne Research Fund invested £750,000 to support the Phase 1 study on Vamorolone. This funding allowed the company to raise further investment to fund the Phase 2 study. The trial, called VISION-DMD, is a Phase 2b trial looking at the efficacy, safety, pharmacokinetics (movement in the body) and pharmacodynamics (effects on the body) of Vamorolone in comparison to corticosteroids and placebo. The trial recruited patients globally, including in the UK through Duchenne UK’s DMD Hub, and Duchenne UK also co-funded Dr Michela Guglieri who is running the trial in the UK. For further information please read the press release here. Q&A What is Vamorolone? Vamorolone has been developed as an alternative treatment to steroids, which are currently the main treatment for DMD. Vamorolone was developed as a dissociative steroid. Scientists and clinicians thought it was possible to chemically separate the clinical benefits of steroids from their concerns about side effects. What was the clinical trial looking at to see if the drug had an effect. VISION-DMD is a Phase 2b trial looking at the efficacy, safety, pharmacokinetics (movement in the body) and pharmacodynamics (effects on the body) of vamorolone in comparison to corticosteroids and placebo. The trial tested how long it took for boys to stand – known as the Time to Stand test score – and this was used as the main clinical measurement to show whether or not the treatment was effective. What happens now? Vamorolone has not yet been approved for use, and will need to be approved by the regulators. It has been granted Orphan Drug status in the US and in Europe, and has Promising Innovate Medicine (PIM) status from the UK Medical and Healthcare products Regulatory Agency (MHRA). This may make it eligible for the Early Access to Medicines Scheme in the UK, which Duchenne UK successfully campaigned for. This would mean that patients in the UK could access the medicine sooner than it is formally approved for use. My son is on the trial. Why did I find out this news on social media rather than my doctor telling me? As a listed company, neither Reveragen or Santhera Therapeutics have been able to share any information ahead of time with clinicians, as they are obliged by financial law to release news of trials publicly. Duchenne UK shared the news as soon as it was made public. NOTES: In 2014 Duchenne UK invested £250,000 to help ReveraGen fund it’s Phase 1 study. As a result of this funding ReveraGen were able to raise further funds for the Phase 2 study. Duchenne UK also co-funded Dr Michela Guglieri who is running the trial in the UK.