MJFF Strategy forAccelerating Developmentof Parkinson’s Therapies
Research RoundtableChicago, IllinoisJune 27, 2011
Today’s Agenda
MJFF OverviewDeborah W. Brooks The Michael J. Fox Foundation for Parkinson’s Research
MJFF Program HighlightsSonal S. Das, PhD The Michael J. Fox Foundation for Parkinson’s Research
PanelistsChristopher G. Goetz, MD
Rush University Medical Center
Jeffrey H. Kordower, PhDRush University Medical Center
Tanya Simuni, MD Northwestern University
Glenn T. Stebbins, PhDRush University Medical Center
Questions & Answers Session
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MJFF Overview
Deborah W. BrooksCo-Founder/Executive Vice Chairman
The Michael J. Fox Foundation for Parkinson’s Research
Parkinson’s disease: Defining the need
Parkinson’s disease (PD) is a progressive neurodegenerative disease that affects nearly one million Americans; related Parkinsonisms affect an additional half million Americans.
PD prevalence is expected to grow sizably over the next 25 years as the proportion of older Americans continues to increase.
Significant therapeutic needs exist:
– Current therapies to treat motor and non-motor symptoms are inadequate leaving substantial unmet needs for those living with PD.
– There is currently no therapy proven to modify the progression of the disease.
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MJFF was founded with clear objectives
Drive the Best Parkinson’s Research
Deliver Improved Therapies and a Cure
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2001 2002 2003 2004 2005 2006 2007 2008 2009 2010 2011
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In 2010, we received nearly 65,000 contributions and raised $57M.
Core values are efficiency and accountability: nearly 90 cents of every $1 spent goes straight to research program efforts. We work to redeploy all donations quickly. We have no endowment or excess reserves.
Since founded in 2000, MJFF has funded more than $240M in research
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Our in-house staff of 7 PhDs and 6 business strategists serve as portfolio managers, incorporating the advice and input of experts from academia and industry into their decision-making.
In 2010, we reviewed over 800 PD-specific grants and currently have roughly 250 active grants in our portfolio.
(est.)
Discovery Target Validation
Therapeutic Development
& Optimization
Pre-Clinical Testing
Clinical Testing
I II III
Regulatory Approval
Progress requires translation of discoveries through the pipeline
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PreclinicalConvert biology into therapies Partly done in academic and
biotech laboratories
ClinicalDetermine safety and efficacy
in patientsMostly done by large pharma
$680 million/year
Basic DiscoveryUnderstanding disease
mechanismsMostly done by academics
$156 million/year
Unique opportunity for MJFF’s strategic funding/leadership to bridge the gap
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Our approach has evolved as we’ve grown and learned
2000-05
2005-11
Focus on research is required to move the needle The science is ahead of the money We are not a bank, assets must be deployed quickly to the best science Our goal is to go out of business 10-year horizon ________________________________________________________________________________________________________________________
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MJFF grant-making cannot be done in a vacuum – a comprehensive view of the landscape is key
Strategy must focus on tackling roadblocks to progress in a systemic fashion, i.e. target validation, research tools, clinical trial design, recruitment
More proactive outreach and engagement with industry partners is required
Turning our knowledge out can ensure others benefit from the field knowledge we are developing
MJFF is in a unique position to bring all constituents together in the name of faster progress
Problem-solve: lead and innovate
Prioritize critical research
Facilitate handoffs and orchestrate connections Showcase top ideas to industry Tirelessly pursue smart, creative solutions to
challenges
Craft aninformed agenda
Understand needs of patients
View global field and identify most promising targets
Convene experts in non-competitive environment
Infuse capital at underfunded, high-risk stages Develop and share essential tools Pragmatically push research in a goal-directed,
milestone-driven fashion
DE-RISK PD 9
How does MJFF work to accelerate progress today?
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2011 MJFF Program HighlightsNeed for Disease-Modifying Therapies
Biomarkers and The Parkinson’s Progression Markers Initiative
Improving Symptomatic Treatments
Sonal S. Das, PhDAssociate Director, Research Programs
The Michael J. Fox Foundation for Parkinson’s Research
MJFF priority area efforts drive progress in key research topics
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Alter Disease
Validate Genetic Targets
Develop Trophic Factors
Define PD/PD Progression
Develop Biomarkers of PD Progression
Improve PD Clinical/Pathological
Understanding
Treat Symptoms & Side Effects
Dyskinesias
Non-Motor Symptoms
(Cognitive/Mood, Posture/Gait)
Altering the course of PD: Need for Disease-Modifying Therapies
Current Targets Rationale – Why prioritize?
Trophic Factors Hypothesis for use of trophic factors to treat PD remains viable and exciting Pre-clinical and early phase clinical results continue to show promise
LRRK2 Mutations in LRRK2 may be most common genetic cause of PD Protein kinase function makes LRRK2 a highly druggable target
Alpha-Synuclein Pathological presence in Lewy bodies of familial and sporadic PD Genetic association in familial cases of PD
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MJFF has committed over $100M to advance disease modifying therapies and seeks a path forward to patients.
Expert Insight, Jeffrey H. Kordower, PhD
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Disease-Modifying Therapies
Jeffrey H. Kordower, PhDDirector, Research Center for Brain Repair
The Jean-Schweppe Armour Professor of Neurological SciencesRush University Medical Center
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Obstacles include:
• Current trial paradigm requires large number of subjects
• Efficacy testing is a long process, diminishing patent value
• Reliance on clinical measures alone confound trial interpretation
• There are no biological indications of underlying disease that complement clinical changes
Markers of progression are essential to achieve near-term hopes for needed therapies
Challenges remain in testing disease-modifying therapies
Biomarkers
Therapeutic MarkersProgression MarkersDiagnostic Markers
Identify PD patients and, perhaps, even determine who is at risk for developing PD
Assist with patient selection for clinical studies…Are we studying the right population of people?
Facilitate measurement of modifications in the disease - could provide valid clinical trial endpoints
Help guide clinical trial design parameters like patient numbers, stratification, duration of treatment
Is the therapeutic reaching its target?
Is the therapeutic having its desired effect?
Without markers of progression, clinical trials to test new therapies in patients are at risk of yielding
inconclusive results
MJFF has supported almost $31M in biomarker discovery
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The Parkinson’s Progression Markers Initiative (PPMI)
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PPMI
Dataset/ sample
collection
Standardized protocols
Biomarker verification
studies
Identify progression
markers
PPMI is a ground-breaking clinical study that aims to dramatically accelerate PD drug development by identifying biomarkers of the disease. PPMI aims to recruit 400 newly diagnosed people with Parkinson’s and 200 control participants.
A pre-competitive collaboration between government and industry. A study of this size – $45M over a 5 year period – requires large scale funding: Abbott, Biogen Idec,
Covance, GE Healthcare, Genentech, Merck, Pfizer and Roche are lead industry supporters. As of Wednesday, June 22nd, 2011, 19 out of 21 PPMI sites in the US and Europe are currently
recruiting volunteers. At these sites, 140 participants have been recruited (82 PD, 58 control) with an additional 43 consents.
Expert Insight, Tanya Simuni, MD
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Insights Into PPMI and What We Hope to Learn
Tanya Simuni, MDA.C. Nielsen Research Professor of Parkinson's disease
Associate Professor of NeurologyDirector, Parkinson’s Disease and Movement Disorders Center
Northwestern University
Clear need for developing treatments for motor & non-motor symptoms
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Dyskinesia
Non-Motor Symptoms
MJFF has funded nearly $17M in dyskinesia research and $18.5M in research towards improving treatments for non-motor symptoms.
Uncontrolled body movements that result from dopamine-replacement therapy
Breakthroughs in treating dyskinesia would expand options for treating PD
Includes cognitive dysfunction, anxiety, memory loss and mood disorders
Relieving these symptoms leads to a better quality of life for those living with PD
MJFF is funding two parallel tracks in dyskinesia research: 1. Developing new therapies 2. Determining how to best assess these therapies in the
clinic (Dyskinesia Rating Scale Study)
Expert Insight: Christopher G. Goetz, MD
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Advancing Treatments for Dyskinesia
Christopher G. Goetz, MDProfessor of Neurological Sciences
Professor of PharmacologyRush University Medical Center
Co-Principal Investigator, Dyskinesia Rating Scale
Glenn T. Stebbins, PhDProfessor of Neurological Sciences
Rush University Medical CenterCo-Principal Investigator, Dyskinesia Rating Scale
Empowered coordinators
Smart matches
Active alert system
Robust content
Connecting willing volunteers with clinical trials in their area
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50% of clinical trial sites that enroll 1 or 0 subjects in their studies 85% of clinical trials finish late due to recruitment troubles Estimates hold that fewer than 1 in 10 people with Parkinson’s
participate in clinical trials
Questions & Answers Session
Deborah W. Brooks, The Michael J. Fox Foundation for Parkinson’s Research
Sonal S. Das, PhD The Michael J. Fox Foundation for Parkinson’s Research
Christopher G. Goetz, MD Rush University Medical Center
Jeffrey H. Kordower, PhD Rush University Medical Center
Tanya Simuni, MD, Northwestern University
Glenn T. Stebbins, PhD, Rush University Medical Center
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For more information, please visit:
www.michaeljfox.org
Our 2011 Research Roundtable Series is generously supported through an educational grant from Teva Neuroscience
Thank you for your participation!
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