Rising Drug Costs & Lack of New Treatments As the U.S. deals with spiraling drug costs and an urgent need for new treatments, the Myelin Repair Foundation has pioneered a holistic approach to multiple sclerosis drug development that bridges the gap between the promising discoveries made in academic laboratories and big pharmaceutical drug research and development. The approach shows early promise in cutting development costs and shaving years off of the time it takes to develop a drug from basic science to patient therapy. The Myelin Repair Foundation’s approach will not only benefit the millions living with MS but also provides a new source of high-quality drug targets for the dwindling biopharma pipelines. Investing in Innovation The Ewing Marion Kauffman Foundation, Pioneer Portfolio of the Robert Wood Johnson Foundation, and the Scott Cook & Signe Ostby Foundation are investing in MRF’s unique approach, its potential to accelerate the drug development process. Online Webinar: March 25, 2010 All three organizations held a webinar on March 25, 2010 session to discuss why they support the MRF model and why attention should be paid to this new research paradigm. Contact info@myelinrepair.org with questions or media requests.

1. Developing Cures with
Less Time & Capital:
A New Research Model to
Accelerate Drug Development
March 25, 2010

2. Margaret Anderson
Executive Director, FasterCures
Catalyzing efforts across sectors to
improve the effectiveness and efficiency
of the medical research enterprise
Committed to analyzing barriers to
progress and overcoming them through
action

3. Agenda
Overview of Research Development Process
Panel Discussion
Open Questions

4. Participants
• Scott Johnson, President and Founder, Myelin Repair
Foundation
• Lesa Mitchell, Vice President, Advancing Innovation,
Kauffman Foundation
• Nancy Barrand, Special Adviser for Program
Development, Robert Wood Johnson Foundation
• Scott Cook, Co-founder of the Scott Cook & Signe Ostby
Foundation and co-founder of Intuit Inc.
• Host: Margaret Anderson, Executive Director of
FasterCures

5. Scott Johnson
Founder and President, Myelin Repair
Foundation
Waiting for a cure for MS since 1976
Leading the way toward faster
development of new treatments and cures
for ALL diseases

6. Crisis in Health Care
Effective new treatments for difficult diseases are
far too slow in reaching patients who can’t afford
to wait.
The economic impact in the U.S. of these untreated
diseases is estimated at $1.3 trillion annually.
Shrinking pharma pipeline threatens industry.
Shrinking venture capital funding for biotech
startups.

7. Increasing R&D Spend, but Flat Results
R&D spending has grown steadily… …but NME approvals relatively flat
No. of approvals
($B) 140
45
CAGR
11.3%
30
70
INDs
R&D abroad CAGR
10.5%
15
Domestic R&D
NMEs
0 0
’91 ’92 ’93 ’94 ’95 ’96 ’97 ’98 ’99 ’00 ’01 ’02 ’03 ’04 ’05 ’91 ’92 ’93 ’94 ’95 ’96 ’97 ’98 ’99 ’00 ’01 ’02 ’03 ’04 ’05
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

8. Evolution of the Medical Research Enterprise
Paradigm
1940 2010
shift

9. Holistic Approach Manages to Milestones:
Accelerating Basic Science

10. Holistic Approach Manages to Milestones:
Building a Network of CROs

11. Holistic Approach Manages to Milestones:
Building Partnerships with Biopharma

12. Holistic Approach Manages to Milestones
MRF ARC Model

13. Shifting the Paradigm:
Rapid Results at Far Lower Cost
Published > 75 peer-reviewed scientific articles.
Established first partnership with pharmaceutical
company to review and validate academic discoveries.
• Demonstrated that myelin can be repaired in animal models of
MS.
• Identified 144 discoveries that have the potential to repair
myelin; prioritized top 20 discoveries to develop further.
• Developed 24 new research tools.
• Created significant intellectual property; 20 patentable
inventions

14. Panelists
• Scott Johnson, President and Founder, Myelin
Repair Foundation
• Lesa Mitchell, Vice President, Advancing
Innovation, Kauffman Foundation
• Nancy Barrand, Special Adviser for Program
Development, Robert Wood Johnson Foundation
• Scott Cook, Co-founder of the Scott Cook & Signe
Ostby Foundation and co-founder of Intuit Inc.

15. Nancy Barrand
Special Adviser for Program
Development, Robert Wood Johnson
Foundation
Works to ensure health coverage for all
Americans, improve the social factors that
shape health for vulnerable populations,
and support innovative ideas that can
transform the future of health and health
care
Wants to make the research enterprise
work faster and more openly to accelerate
treatment discoveries and cures

16. Lesa Mitchell
Vice President of Advancing Innovation,
Ewing Marion Kauffman Foundation
20 years experience in biotech and
pharmaceutical entrepreneurship
Committed to improving pathways that
shepherd research from universities and
labs through commercialization

17. Scott Cook
Founder & Chairman of the Executive
Committee - Intuit
Board Member – Procter & Gamble,
eBay, Intuit
Catalyst of paradigm change in health
care, education, and Internet access for
the developing world

18. Panelists
• Scott Johnson, President and Founder, Myelin
Repair Foundation
• Lesa Mitchell, Vice President, Advancing
Innovation, Kauffman Foundation
• Nancy Barrand, Special Adviser for Program
Development, Robert Wood Johnson Foundation
• Scott Cook, Co-founder of the Scott Cook & Signe
Ostby Foundation and co-founder of Intuit Inc.
• Host: Margaret Anderson, Executive Director of
FasterCures

19. For More Information
Carol Menaker
carol@myelinrepair.org
408-871-2410
Myelin Repair Foundation
http://myelinrepair.org
http://wherearethecures.org
Kelly Rohrs
kelly.rohrs@edelman.com
212-819-4852
Edelman