Our Partnership

In 2014 Duchenne UK, along with The Duchenne Research Fund, invested $1million on a novel steroid alternative VBP15. Muscular Dystrophy America also invested in this project.

The drug is being developed by ReveraGen Biopharma – a venture philanthropy drug development company.

The Phase I study was a success, and enabled ReveraGen Biopharma to raise a further $12million from US and European government agencies, for their Phase II clinical studies. 

As of September 2016 ReveraGen Biopharma has begun recruiting patients for its Phase 2a open label study of Vamorolone. The trial will be recruiting patients who haven’t yet started steroids, between the ages of 4-7, and is recruiting in the UK, United States, Canada, Israel, Sweden and Australia.

For more information please visit: http://vision-dmd.info/2a-trial-information/

The Phase 2a study completed recruitment in November 2017. The purpose of this study was to test how safe and tolerated vamorolone was in boys with Duchenne, and how the acts drug in the body.

This study is an open label, multiple ascending dose study of vamorolone on 48 steroid naïve boys aged from 4 years old to less than 7 years old. Boys were enrolled in four dose cohorts sequentially – 0.25 mg/kg, 0.75 mg/kg, 2.0 mg/kg and 6.0 mg/kg vamorolone, dosing once a day for 2 weeks followed by 2 weeks of follow up. The primary outcome of this trial is acute safety, tolerability and pharmacokinetics of vamorolone administration. The Phase 2a study database lock was achieved in February 2018. Results of this study will be published imminently.

The Phase 2a extension study is ongoing with the participants from the 2a study, at the same dose level for 24 weeks. The primary outcome of the 2a extension trial is long term safety, tolerability, efficacy as measured by the time to stand test and safety as measured by body mass index. Database lock for the 2a extension study is expected in June 2018. Following this study patients have the option of entering a long term extension study.
Twelve trial sites in seven countries are participating in the 2a studies including the USA, Canada, the UK, Israel, Sweden, and Australia.

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 – also known as VBP15 - is an anti-inflammatory drug.

It is expected that the new treatment 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, who we have funded via our Clinical Trial Capacity programme, is the Principal Investigator of the trial at the John Walton Muscular Dystrophy Research Centre in Newcastle.

What Is Happening About Clinical Trials?

Vamorolone has already been successfully tested on healthy volunteers in a Phase I Study.

As of September 2016 ReveraGen Biopharma has begun recruiting patients from UK, United States, Canada, Israel, Sweden and Australia for its Phase 2a open label study. Please see the information above and visit: http://vision-dmd.info/2a-trial-information/

The first Phase 2b study site is expected to open for recruitment in April 2018 in the USA. Phase 2b trial sites are expected in the USA, Canada, Belgium, Czech Republic, Germany, Italy, the Netherlands, Sweden, Israel, the UK and Australia

For more information, visit:
https://vision-dmd.info/2b-trial-information/

Find out more about clinical trials here