Michael J. Fox Foundation CEO Todd Sherer, PhD, presented on the recent collaboration between Vanderbilt University and the pharmaceutical giant, Bristol-Myers Squibb (BMS) in a congressional briefing to the House of Representatives. Dr. Sherer explained the Foundation’s role in the drug development process as a whole and the crucial funding that supported Vanderbilt’s early glutamate receptors work leading to this momentous partnership for PD research.

1. MJFF’s Role In
The Drug Development Process

2. About The Michael J. Fox Foundation
TO PRODUCE ONE NEW DRUG FOR PARKINSON’S DISEASE TAKES UPWARDS OF
15 YEARS AND ONE BILLION DOLLARS
• Founded in 2000 with a single urgent mission:
• Fund research that will speed a cure for Parkinson’s disease
• Streamline and orchestrate research efforts among the key
players in the drug discovery ecosystem
• Academia, industry and patients
• Funded over $300 million to PD research to-date
• Active portfolio of 450 grants in disease-modifying and symptomatic
treatments
• Supported over 350 institutions (both academic and industry) worldwide

3. The ABC’s of Drug Development
Research & Target Drug Discovery FDA
FDA
Discovery Validation & Development Phase I Phase II Phase III Approval
Approval
BASIC SCIENCE DISCOVERY PHASE
• Backbone of all drug development
• MJFF’s impact is possible because we strategically build off of the
federal government’s ongoing investment in discovery via NIH
• This phase requires over $30 billion a year through NIH alone

4. The ABC’s of Drug Development
Research & Target Drug Discovery FDA
FDA
Discovery Validation & Development Phase I Phase II Phase III Approval
Approval
TRANSLATIONAL RESEARCH PHASE
(MJFF PRIMARY AREA OF FOCUS)
• Where applied work from discovery phase are homed in and looked at
for disease-specific effects
• Few natural handoffs exist to shepherd along from academia to industry
• Due to chronic funding and expertise gap, this is where treatment
breakthroughs go to die
• Famously branded the “valley of death” by former NIH Director, Elias Zerhouni

5. The ABC’s of Drug Development
Research & Target Drug Discovery FDA
FDA
Discovery Validation & Development Phase I Phase II Phase III Approval
Approval
CLINICAL RESEARCH AND REGULATORY APPROVAL PHASE
• Largely handled by the private sector and is becoming more risk-averse
• Multiple stages in each phase could be required before a potential drug
advances along the pipeline (e.g. Phase 1a  Phase 1b)
• Next stop can be regulatory approval and practical relevance in patients’
lives
• Across all diseases, this phase costs approximately $70B a year, mostly
through private investments
• Only 1 in 12 treatments in this phase makes it to pharmacy shelves

6. The MJFF Approach

7. The MJFF Approach

8. TheThis is the first section
Impact We’re Seeing
Landmark Biomarkers Initiative First clinical trial of major genetic target
 • Modeled after ADNI  launched in 2012
• 12 industry partners and growing
Continuing investments in trophic factors
 • Latest results in patients expected in spring
2013
Pre-clinical models and reagents
 • No associated intellectual property Revitalizing the pipeline of treatments for
limitations  major side effects of current gold-standard
treatments
• 10 therapies in clinical testing
 New class of glutamate-based symptomatic
therapy approaching clinical phase

9. New Symptomatic Therapy: Jeff Conn, Vanderbilt Univ.
Research & Target Drug Discovery FDA
Discovery Validation & Development Phase I Phase II Phase III Approval
2005: MJFF $200K 2007: MJFF $4.4M 2012: Bristol-Myers Squibb Collaboration
Next Steps: Experimental drug trial
• 2005: $200K investment leads to LEAPS
• 2007: Milestone-based award of $4.4M
• 2009: Two lead compounds identified
• 2011: LEAPS supplemented for late-stage pre-clinical work/final
phases before human testing
• 2012: Vanderbilt University and BMS announce partnership

10. MJFF’s Role In
The Vanderbilt University and BMS Partnership
Identifying key components that supported Jeff’s work from
“A-P”:
• Leveraging Jeff Conn’s NIH supported glutamate receptors
discovery work
• High-risk funding in early translational stages
• Infrastructure that assesses progress and provides peer review
opportunities
• Fast and efficient turnaround
• Consistent funding to carry through final stages to partnership
with BMS

11. Key Learnings and Challenges We Still Face
• High-risk, high-reward funding in the early stages pays off
• Collaboration moves the dial
• Shared responsibility throughout all stages of the pipeline
• New kind of stakeholder can speed progress
IN OUR SHORT HISTORY, WE HAVE BUILT THE CREDIBILITY AND EXPERTISE TO MAKE
REAL INROADS IN THE BUSINESS OF SCIENCE BUT WE CAN NOT DO IT ALONE
• Evolving from “de-risking” to “co-risking”
• Funding gets more expensive as potential treatments move along to the
pipeline
• How do we attract other “BMS” style partners
• What does it take to gain access to capital that would ensure no promising
target goes unexplored for lack of funding
• Galvanizing patient engagement