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The Myelin Repair Foundation
Open Science SummitJuly 31, 2010
A New Paradigm for Medical Research
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Drug Discovery Today
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Academic Research Academic Research Drug Drug DiscoveryDiscovery
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
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Pharmaceutical R&D output flat despite increasing spend...
1. IMDs (Incrementally Modified Drugs) are new derivatives, formulations and combinations2. NME (New Molecular Entities) include both chemical and biological therapeutic agents, excludes contrast agents and diagnosticsSource: FDA, PhRMA 2005 Yearbook
…but NME approvals relatively flat…but NME approvals relatively flat
0
70
140
’91 ’92 ’93 ’94 ’95 ’96 ’97 ’98 ’99 ’00 ’01 ’02 ’03 ’04 ’05
No. of approvals
NMEs
INDs
R&D spending has grown steadily…R&D spending has grown steadily…
0
15
30
45
’91 ’92 ’93 ’94 ’95 ’96 ’97 ’98 ’99 ’00 ’01 ’02 ’03 ’04 ’05
($B)
CAGR11.3%
CAGR10.5%
Domestic R&D
R&D abroad
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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
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The System is Broken
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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.
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SponsoredResearch
BenBarres
RobertMiller
SteveMiller
BrianPopko
Mu
ltip
le
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Six Areas of Research
1. Understanding how oligodendrocytes are normally generated from neural stem cells and how MS perturbs this process
2. Understanding the underlying mechanism of myelination and how it is perturbed in MS
3. Understanding how nodes of Ranvier and paranodes are normally formed and how they are perturbed in MS
4. Understanding the immune response in MS and how inflammation affects myelin repair
5. Understanding how the Blood Brain Barrier is affected in MS and its role in the disease
6. Development of better animal models for study of MS and remyelination
MRF’s Discovery Biology Research Plan
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Crossing the Valley of Death – Accelerating the transition from promising targets to clinical candidates
0
200
400
600
800
1,000
Target ID Validation Screening Lead opt Pre-clin Phase I Phase II Phase III NDA Total
Percentage of total cost of success
$880M
19%
23%
5%
10%
7%
10%
11% 1%
Source: BCG economic model study in Biomedical Industry Advisory Group, 2005; BCG clinical development model, 2007
TargetValidation
Screen-ing
TargetID
Optimi-zation
Pre- clinical
PhaseI
PhaseII
PhaseIII
Filing NDA
14%
71% of industry costs
1.0 0.4 2.7 1.5 1.5 2.5 1.5 14.72.0 1.6Avg.
time (yr)
ARC II
15 - 30
Cost ($M)
Basic Research
MRF’s ARC Model Proposed ARC Expansion
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First Five Years – 2004 to 2009Exceeded 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)
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First Five Years – 2004 to 2009Exceeded 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.
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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
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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
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The only non-profit medical research foundation that participates in the entire continuum of drug discovery
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