Jesse’s Journey is thrilled to share the recent announcement from Santhera Pharmaceuticals, which shows a new analysis of the 2.5-year long-term extension data from ReveraGen Phase 2a study of vamorolone, which we’re currently funding.
This data demonstrates the equivalent of a delay for boys with Duchenne muscular dystrophy of about 2 years before any decline in time to stand. It also confirms the safety and tolerability of vamorolone, over the 2.5 years of the study. We hope to have the first six months of data from their VISION DMD trial by the end of this quarter – stay tuned for more details to come!
“We are very pleased with the results of this long-term follow-up which show that treatment benefits of vamorolone are sustained over the 2.5-year follow-up period. Importantly, vamorolone was well tolerated and we observed far fewer of the side effects typically seen with corticosteroids in the clinic,” said Eric Hoffman, PhD, President and CEO at ReveraGen BioPharma. “We would like to thank all patients, their caregivers and health care professionals for their much-valued participation in these studies which, we are confident, will support our approach to develop vamorolone as a novel treatment for this devastating disease.”
“These findings indicate the long-term maintenance of treatment effect and disease modifying potential with vamorolone. We are equally excited about the findings that vamorolone did not show stunting of growth typically reported for other corticosteroids, and also resulted in significantly fewer physician reported adverse events which are common reasons for corticosteroid treatment discontinuation,” said Dario Eklund, CEO of Santhera. “We are expecting the 6-month results from the pivotal Phase 2b VISIONDMD study in Q2-2021 and are confident that they will provide further evidence to establish vamorolone as an effective treatment and valuable alternative to corticosteroids for the long-term treatment for DMD.”
Vamorolone is a first-in-class drug candidate that binds to the same receptor as corticosteroids but modifies its downstream activity and as such is a dissociative partial agonist [6-8]. This mechanism has the potential to ‘dissociate’ efficacy from typical steroid safety concerns and therefore vamorolone could emerge as a promising alternative to existing corticosteroids, the current standard of care in children and adolescent patients with DMD. There is substantial unmet medical need in this patient group as highdose corticosteroids have significant systemic side effects that diminish patient quality of life. In the pivotal Phase 2b VISION-DMD trial, the last patient has completed the last visit of the 24-week, placeboand active-controlled treatment period and topline 6-month data are expected in Q2-2021, paving the way for a US NDA submission in Q1-2022. Vamorolone has been granted Orphan Drug status in the US and in Europe, and has received Fast Track and Rare Pediatric Disease designations by the US FDA and Promising Innovative Medicine (PIM) status from the UK MHRA. Vamorolone was discovered by US-based ReveraGen BioPharma, Inc. and is being developed in collaboration with Santhera, which owns worldwide rights to the drug candidate in all indications. The vamorolone development program has received funding from several international non-profit foundations and patient organizations, including Jesse’s Journey, the US National Institutes of Health, the US Department of Defense and the European Commission’s Horizon 2020 program.