Jesse's Journey grants $1M toward the launch of a clinical trial for Duchenne muscular dystrophy in Canada
LONDON, ON, June 10, 2020 /CNW/ - Jesse's Journey, Canada's leader in Duchenne muscular dystrophy funded research, is proud to announce a $1M grant towards a clinical trial evaluating vamorolone in children and adolescents living with Duchenne, the most common fatal genetic disease diagnosed in childhood.
This announcement marks two ground-breaking achievements for the Canadian charity – the first time granting one million dollars to a single project and directly funding a clinical trial for older children and adolescents, an age range often neglected in clinical trials.
Dr. Eric Hoffman and his team at ReveraGen BioPharma have found a way to tweak the chemistry of standard corticosteroids, the current standard of care, and developed a new molecule that retains the anti-inflammatory benefits but loses much of the activity associated with side effects.
ReveraGen has successfully completed Phase I and II programs demonstrating treatment with vamorolone led to improvements of strength and mobility over six months and preserved over a year-and-a-half of treatment. Importantly, key side effects, such as stunting of growth, were not observed, and the boys grew normally. This data is supportive of vamorolone having the potential to replace corticosteroid as the standard of care treatment for children and young adults with Duchenne. Their Phase III program is currently underway
This game-changing research would not be possible without the financial investment by Jesse's Journey's.
"The VBP15-006 clinical trial funded by Jesse's Journey will provide the first vamorolone treatment data in very young treatment-naive boys with Duchenne (2 to 4 years old), as well as the first data in older boys with Duchenne already treated with corticosteroids and transitioned to vamorolone", said Dr. Hoffman.
The funding from Jesse's Journey ensures that sites across Canada will be supported to recruit boys with Duchenne into the new study.
"Families living with Duchenne across Canada and the academic medical centers have been amazing collaborators with the vamorolone development team. We are very excited to be able to continue this relationship," continued Dr. Hoffman.
"Jesse's Journey relies on the support of our donors, knowing that research is the road to hope for this devastating disease," shares Director of Research and Advocacy at Jesse's Journey, Nicola Worsfold. "Most children with Duchenne are using a wheelchair by the time they reach their early teens, and with the potential for new treatments powered by research, like this study, we hope to delay the progression of this disease."
"Our mandate as a Duchenne patient organization is to speed up the process of drug discovery and access to beneficial medications addressing all stages of the disease. The VBP15-006 clinical trial addresses the unmet need for inclusion for an expanded age range. We look forward with hope that this potential steroid alternative can increase the quality of life for children and young adults with Duchenne," concludes Nicola.
About Jesse's Journey
Jesse's Journey is Canada's leading charity in the fight to defeat Duchenne muscular dystrophy. For the past 25 years, Jesse's Journey has empowered patients, families, and caregivers living with Duchenne through education and resources, provided a collective voice to advocate for access to treatments in Canada, and has become the country's largest funder of Duchenne research investing more than $13.1M in projects around the world.
About ReveraGen BioPharma
ReveraGen was founded in 2008 to develop first-in-class dissociative steroidal drugs for Duchenne muscular dystrophy and other chronic inflammatory disorders. The development of ReveraGen's lead compound, vamorolone, has been supported through partnerships with foundations worldwide, including Muscular Dystrophy Association USA, Parent Project Muscular Dystrophy, Foundation to Eradicate Duchenne, Save Our Sons, JoiningJack, Action Duchenne, CureDuchenne, Ryan's Quest, Alex's Wish, DuchenneUK, Pietro's Fight, Michael's Cause, Duchenne Research Fund, and Jesse's Journey. ReveraGen has also received generous support from the US Department of Defense CDMRP, National Institutes of Health (NCATS, NINDS, NIAMS), and European Commission (Horizons 2020).
SOURCE Jesse's Journey
Rochelle ten Haaf, Manager, Marketing and Stakeholder Engagement, Jesse's Journey, [email protected]; Dr. Eric Hoffman, Co-founder and CEO, ReveraGen BioPharma, Email: [email protected]
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