Drug development

VBP15: novel steroid for Duchenne

In July 2014 we announced our partnership with the Duchenne Children’s Trust (UK), Joining Jack (UK) and the Muscular Dystrophy Association (US) to provide Phase 1 clinical trial funding for the first-in-human studies of an oral drug called VBP15, developed by ReveraGen BioPharma to treat Duchenne muscular dystrophy. The four charities together contributed $2 million under a co-ordinated venture philanthropy model, providing return on investment based on future drug sales.
VBP15 has been shown to be effective in animal models of Duchenne muscular dystrophy, and clinical trials in humans are now under way – in part, thanks to funds we and our partner charities have donated. In studies, the drug reduced inflammation and stabilised cell membranes, both of which are believed to be relevant to treating Duchenne, a disease in which muscle fibre membranes are unstable. The drug is thought to have similar properties to steroids such as prednisolone, which up until now have been the cornerstone of treatment for boys with Duchenne. The benefit of VBP15, however, is that it is thought to have significantly fewer side effects than existing steroids, which can be life changing in themselves. Side effects include diabetes, high blood pressure, osteoporosis, short stature and weight gain. Read more about Duchenne and the effects of steroid use here.

Previous projects

HT-100: Improving muscle strength

HT-100 is a new drug that has great potential to halt or even reverse fibrosis, the muscle scarring in Duchenne boys. We were part of a consortium of 12 Duchenne patient foundations that provided $1.1 million at the start of the project, conducted by Akashi Therapeutics (formerly Halo Therapeutics). Preliminary analyses of Phase 1 clinical trial results show a potential muscle strength improvement. They are now actively recruiting for Phase 2 to obtain more conclusive results. This treatment would complement other therapies, and would benefit all boys regardless of their type of mutation.

SMT C1100: Restoring muscle function

Scientists have shown that increasing the production of the protein utrophin in Duchenne patients can compensate for the missing dystrophin, and help restore healthy muscle function. We were involved in funding some of the pre-clinical development of a small molecule utrophin modulator for Duchenne, developed by Summit. This treatment would complement other therapies and would benefit all boys regardless of type of mutation. A Phase 1b clinical trial is now finishing, and preliminary results are expected this year.