We are delighted to announce that ReveraGen BioPharma, the company developing a steroid replacement for Duchenne muscular dystrophy, has begun recruiting patients for its Phase 2a open label study. The trial will be recruiting patients who haven’t yet started steroids, between the ages of 4 and 7, and is recruiting in the UK, United States, Canada, Israel, Sweden and Australia.
Duchenne Research Fund co-funded the successful Phase 1 study of Vamorolone (formerly called VPB15), along with Duchenne UK, Joining Jack and the MDA. We are also co-funding the leading Principal Investigator of the study in the UK, Dr Michela Guglieri.
Vamorolone is being developed as an alternative to glucocorticoid treatments (steroids), offering many of their benefits without many of the side effects.
Read more under the drug development section of our website.
In 2014, Duchenne Research Fund, along with Duchenne UK, Joining Jack, and the MDA (Muscular Dystrophy Association, USA) invested $1 million on a novel steroid alternative VBP15.
The drug is being developed by ReveraGen – a venture philanthropy drug development company.
The Phase I study was a success, and enabled ReveraGen BioPharma to raise a further $12 million from US and European government agencies, for their Phase II clinical studies. The Phase II study is being part funded by the European Commission Horizon 2020 programme.
What Is Vamorolone (VBP15)?
Vamorolone could offer a potential alternative to steroids, offering their benefits but reducing their side effects, which can be considerable.
Steroids remain the main long-term treatment for Duchenne muscular dystrophy, as they effectively reduce inflammation.
Vamorolone is an anti-inflammatory drug and it’s hoped that the drug will improve muscle strength in patients without the side-effects seen in steroid treatments, such as mood swings, stunted growth and weight gain.
Dr Michela Guglieri, whom we have co-funded in a consortium of six Duchenne charities, is the Principal Investigator of the trial at the John Walton Muscular Dystrophy Research Centre in Newcastle. Read more about our funding of her fellowship here.
For more more information about the trial please visit the Vision-DMD website.