VISION-DMD

Designed to ensure a timely & cost-effective drug development for Duchenne muscular dystrophy

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  • The Project
    • Summary
    • Project Objectives
    • Project Consortium
    • Contact us
  • About DMD
    • What is DMD?
    • Living with Duchenne
    • Current treatment options
    • Duchenne boy’s stories
    • DMD Community
    • Standards of Care for Duchenne muscular dystrophy
  • Study Information
    • What is a clinical study?
    • Phase 2a clinical trial information
    • Phase 2a preliminary study results
    • Phase 2b clinical trial information
    • Phase 2b Preliminary Results
    • MRI – Magnetic Resonance Imaging
  • Drug Development
    • Development of Vamorolone
    • Vamorolone development programme
  • Innovations
    • Return of patient data
    • Bone Health Outcomes
    • Biomarkers
    • Vamorolone study APP for boys
    • Patient Group Funders
    • What is Venture Philanthropy?
  • Resources
    • Relevant publications
      • VISION-DMD INFORMATION APP OVERVIEW
      • VISION-DMD Thought paper
    • VISION-DMD Workshops
      • Workshop series: Standards of Care
      • Workshop series: Biomarkers and Imaging
    • Webinars and presentations
    • Press Releases
    • VISION-DMD project Interviews
      • Collaborating for progress on rare diseases: an interview with Dr. Daria Julkowska, Scientific Coordinator of the European Joint Program on Rare Diseases.
      • Returning vamorolone study data to patient caregivers. What, when and how clinical trial data is returned to study participants. An interview with Dr. Laurie Conklin
      • An Interview with Andrea D’Alessandro – Clinical research project manager at TRiNDS
      • Interview with Jana Haberlová – Paediatric Neurologist at University Hospital Motol
      • Interview with Alex Johnson: a parent and patient foundation funder
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Development of Vamorolone

Vamorolone, (also known as VBP15) is a ‘dissociative steroid’. This means that it retains sub activities associated with efficacy (trans repression, physicochemical membrane effects, synchronisation of tissue remodelling), and dissociates these efficacy-sub activities from other sub activities more associated with harmful side effect profiles (transactivation sub activity). Vamorolone has received Orphan Drug Designation in the US and Europe and is being developed for chronic treatment of boys with Duchenne Muscular Dystrophy (DMD).

Based on the characterization and optimization of vamorolone for transactivation, transrepression and physicochemical membrane properties, ReveraGen Biopharma undertook the testing for safety and efficacy in animal models. Toxicity, pharmacokinetics, pharmacodynamics, and ADME studies (absorption, distribution, metabolism and excretion) have been conducted. Results have demonstrated that most short-term (acute) properties of vamorolone were similar to traditional glucocorticoids.

Multiple studies were conducted using the mdx mouse model of Duchenne muscular dystrophy (a mouse lacking dystrophin in muscle, similar to Duchenne muscular dystrophy patients).Vamorolone consistently showed improvements in muscle function that were similar to or superior to prednisone.  A loss of side effects was also seen, including loss of growth stunting, loss of heart fibrosis and loss of immunosuppressive effects.

Phase 1 clinical trials were completed in late 2015 in healthy adult volunteers (SAD and MAD).

–Single Ascending Dose (SAD) 0.1, 0.3, 1.0, 3.0, 8.0, 8.0 fed, 20.0 mg/kg vamorolone

  • Excellent dose proportionality, little variation between subjects, short half life (2-4 hours).

–Multiple Ascending Dose (MAD) 1.0, 3.0, 9.0, 20.0 mg/kg/day 2 weeks.

  • No drug accumulation (PK for Day 1 identical to Day 14), Vamorolone safe at highest dose tested.

For more information visit www.reveragen.com

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This project has received funding from the European Union’s Horizon 2020 research and innovation programme under grant agreement number 667078.

To find out more about the project contact us at [email protected]

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