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VISION-DMD Working Group Meeting

Athens, 6 and 8 December 2019 – The Vision-DMD Working Group Meeting organized by the World Duchenne Organization, preceded the Duchenne Patient Academy


All clinical trial countries were represented during the Working Group Meeting: Australia, Belgium, Canada, Czech Republic, Greece, Israel, the Netherlands, Spain, Sweden, the United States and the United Kingdom.


The Vision-DMD Working Group Meeting was held on the 6th of December, and brought together 25 representatives of 11 clinical trial countries. The topic of this meeting was to provide an update on the phase 2b study of vamorolone by Dr. Eric Hoffman and Laurie Conklin from ReveraGen. Next, Nefeli Panousis, CRO Advisor of MDA Hellas explained the study design, and addressed questions on concerns the participants were asking regarding the enrollment criteria and study features. On Sunday December 8, Laurie Conklin, MD, medical and regulatory director at ReveraGen BioPharma presented the programme to return trial data to participating families during Duchenne Patient Academy, holding 80 patient advocates from 37 countries over the world. Participants agreed to collaborate further regarding the patient enrollment before the end of January to complete the study requirements.

Scientific update

Eric Hoffman, scientific rationale and data from vamorolone trials

Robust evidence of vamorolone efficacy and improved safety over corticosteroids in 18-month open-label trial series (VBP15-002, -003, -LTE)

All efficacy outcomes show dose-dependent improvements over 6 months (Hoffman et al. 2019)

  • All efficacy outcomes show improvement over 18 months treatment (2.0, 6.0 mg/kg/day)
  • Efficacy similar to corticosteroids
  • Dose ranging and biomarkers argue against placebo effect
  • Objective safety outcomes show vamorolone improvements over corticosteroids.
  • Stunting of growth, osteocalcin biomarkers
  • Cushingoid appearance
  • Weight gain
  • No SAEs attributable to the drug, ~70 patient years of high dose drug exposure in DMD

Duchenne boys eligible to participate in the phase 2b study need to be between 4 and 7 years old when the study starts. The representatives from study countries expressed their concerns on boys needing to be steroid negative. A possibility for boys being off steroids for a wash-out period is currently being discussed.

Too often, Duchenne boys participating in a study will receive either the drug, or a placebo. In the case of vamorolone, every participant will receive the study drug for the last 6 months of the project. The first 6 months, participants will either receive vamorolone (50%), prednisone (25%) or a placebo (25%). All boys will receive a high dose vamorolone, 6.0 mg/kg.

Venture philanthropy

Laurie Conklin, MD, medical and regulatory director at ReveraGen BioPharma presented the programme to return trial data to participating families. Funded by the US National Institutes of Health under a Bioethics Administrative supplemental grant, a plan was developed by ReveraGen to return clinical trial results to families (including their child’s individual data) at the close of the trials. Approved under a central IRB, informed consent is obtained from the parents by Suzanne Gaglianone, ReveraGen’s travel coordinator.  To learn more about perceptions, priorities, and reactions to the process, an electronic questionnaire will be administered to the parents pre and post data-return, and to the child’s study physician post data-return.

The business model for development of vamorolone is through venture philanthropy. ReveraGen Biopharma Inc, the drug developer, has had no venture capital investors to date. Instead the company has accessed grant funding and partnered with stakeholder foundations finance to carry out the complex process of developing vamorolone for the treatment of DMD. Venture philanthropy (VP) aims to invest in organisations or activities by providing both financial and non-financial support in order to increase the social impact.


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www.clinicaltrials.gov  NCT03439670