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Myelin repair open science summit 07.31.10 v2

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  • Title slide present while callers are logging/calling in.
  • Scott
  • Scott
  • Russell: Discusses 2-3 particularly interesting discoveries. Scott: Thank you Russell. Other comments?? I am pleased to introduce Dr. Jay Tung, the MRF Vice President for Drug Discovery…
  • Scott
  • Transcript

    • 1. The Myelin Repair Foundation Open Science Summit July 31, 2010 A New Paradigm for Medical Research
    • 2. Drug Discovery Today
    • 3. Academic Research  Drug Discovery
      • Proposal, peer review and grant system results in:
      • Incremental experiments
      • Competition and lack of coordination between scientific disciplines
      • Inefficient communication process
      • Scientists do not specialize in diseases
      • Chaotic progress - no disease based research roadmap
      • Intellectual Property is not protected
    • 4. Pharmaceutical R&D output flat despite increasing spend... 1. IMDs (Incrementally Modified Drugs) are new derivatives, formulations and combinations 2. NME (New Molecular Entities) include both chemical and biological therapeutic agents, excludes contrast agents and diagnostics Source: FDA, PhRMA 2005 Yearbook … but NME approvals relatively flat No. of approvals NMEs INDs R&D spending has grown steadily… ($B) CAGR 11.3% CAGR 10.5% Domestic R&D R&D abroad
    • 5. Many Players but historically no overall coordinator/manager
      • National Institutes of Health
      • Legacy non-profit disease research organizations
      • Academic scientists
      • Contract research organizations
      • Biotech and pharmaceutical industry
      • Food and Drug Administration
      • Medical service providers
      • Insurance companies
      • Patients and their families
    • 6. The System is Broken
    • 7. MRF’s Mission
      • To accelerate the discovery of myelin repair treatments to improve the lives of people suffering from Multiple Sclerosis (MS), and
      • To establish the MRF Accelerated Research Collaboration™ (ARC™) model as a new paradigm for medical research.
    • 8. 08/01/10 Sponsored Research Ben Barres Robert Miller Steve Miller Brian Popko Multiple
    • 9. 08/01/10
      • Six Areas of Research
      • Understanding how oligodendrocytes are normally generated from neural stem cells and how MS perturbs this process
      • Understanding the underlying mechanism of myelination and how it is perturbed in MS
      • Understanding how nodes of Ranvier and paranodes are normally formed and how they are perturbed in MS
      • Understanding the immune response in MS and how inflammation affects myelin repair
      • Understanding how the Blood Brain Barrier is affected in MS and its role in the disease
      • Development of better animal models for study of MS and remyelination
      MRF’s Discovery Biology Research Plan
    • 10. Crossing the Valley of Death – Accelerating the transition from promising targets to clinical candidates Percentage of total cost of success $880M 19% 23% 5% 10% 7% 10% 11% 1% Source: BCG economic model study in Biomedical Industry Advisory Group, 2005; BCG clinical development model, 2007 Target Validation Screen- ing Target ID Optimi-zation Pre- clinical Phase I Phase II Phase III Filing NDA 14% 71% of industry costs 1.0 0.4 2.7 1.5 1.5 2.5 1.5 14.7 2.0 1.6 Avg. time (yr) ARC II 15 - 30 Cost ($M) Basic Research MRF’s ARC Model Proposed ARC Expansion
    • 11. First Five Years – 2004 to 2009 Exceeded milestones and goals
      • Established Center for Myelin Repair Discovery Biology with replicable infrastructure.
      • Engaged leading myelin repair scientists in a managed, patient-outcome-driven research plan.
      • Discovered 144 putative targets.
      • Developed 24 new research tools with application to all neurological disease research.
      • (continued)
    • 12. First Five Years – 2004 to 2009 Exceeded milestones and goals
      • Awarded first patent in 2008; 18 more patent applications have been submitted or are in preparation.
      • Developed “virtual biotech” infrastructure for evaluating targets.
      • Established myelin repair as a viable alternative approach to treating multiple sclerosis
      • Established first-of-a-kind partnership with an pharmaceutical company to advance one or more therapeutic targets and/or models .
      • Raised $33M but accomplished the above for $21M.
    • 13. MRF’s Second Five Years 2009 - 2014
      • Identify more high-quality potential therapeutic targets
      • Validate therapeutic targets to meet industry standards
      • Build industry partnerships leading to clinical trials
      • Identify new approaches to measuring clinical outcomes and to speed clinical trials
    • 14. 08/01/10
    • 15. Strategies for Translating MRF Discoveries
      • Support existing pharma company pipelines
        • Offer access to MRF Target Validation toolkit
        • Provide data for “repurposing” existing clinical candidates
      • Contracting
        • Promote research collaborations with corporate entities
        • Fund Target Validation/Drug Discovery studies at CROs
      • Partnering
        • Offer exclusive licensing options to MRF targets/tools
        • Create Joint IP with biotech/pharma partners
      • Spinoffs
        • Create new corporate entities to develop innovative MRF programs
    • 16. The only non-profit medical research foundation that participates in the entire continuum of drug discovery

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