MJFF’s Purpose, Promise and Plan for speeding new Parkinson’s treatments to patients

MJFF’s Purpose, Promise and Plan for Speeding New Parkinson’s Treatments to Patients Research Roundtable New York, New York November 12, 2011

Today’s Agenda  MJFF Overview Deborah W. Brooks The Michael J. Fox Foundation for Parkinson’s Research  MJFF Research Progress & Remaining Challenges Todd Sherer, PhD The Michael J. Fox Foundation for Parkinson’s Research  Panelists Anders Björklund, MD, PhD Lund University John Dunlop, PhD Pfizer, Inc., Neuroscience Research Unit Mark Frasier, PhD The Michael J. Fox Foundation for Parkinson’s Research  Questions & Answers Session 2

MJFF Overview Deborah W. Brooks Co-Founder & Executive Vice ChairmanThe Michael J. Fox Foundation for Parkinson’s Research 3

Why we exist… Drive the Best Parkinson’s Research Deliver Improved Therapies and a Cure Our world-class team monitors developments in Parkinson’s research, identifying top priorities for the field. We work closely with the Parkinson’s community to initiate, fund, and lead high-impact projects and collaborations. 4

With over $270M funded since 2000, we are on a mission to speed a cure  In 2010, we received nearly 65,000 contributions—substantially all from individuals who have a stake in our success. And, our movement is building.  We promise impact, efficiency and accountability: over 87 cents of every $1 spent goes straight to research program efforts. We deploy donations conscientiously, with wisdom and integrity. We deliberately have no endowment or excess reserves.  Our in-house staff of 7 PhDs, 1 MD and 7 business strategists tap the advice of experts from academia and industry globally. We have an informed opinion and share it passionately. 60 new commitments 50 40 in millions 30 20 10 0 2001 2002 2003 2004 2005 2006 2007 2008 2009 2010 2011 (est.) 5

Drug development is long, costly and risky…but can be smarter Therapeutic Target Development Pre-Clinical Clinical Regulatory Discovery Testing Validation & Testing Approval Optimization I II III Basic Discovery Preclinical Clinical Understanding disease Convert biology into therapies Determine safety and efficacy mechanisms Partly done in academic and in patientsMostly done by academics biotech laboratories Mostly done by large pharma $156 million/year $680 million/year MJFF steps in to drive translation and assure that promising therapies get closer to patients 6

2011 MJFF Research Progress and Remaining ChallengesAltering Disease: Disease Modifying Therapies LRRK2: A Collaborative Example Repositioning Drugs Improving Symptomatic Treatments Targeting Serotonin Receptors Todd Sherer, PhD Chief Executive Officer The Michael J. Fox Foundation for Parkinson’s Research 7

There are numerous therapeutic needs for PD patients Treat Symptoms Alter Disease & Side Effects Validate Genetic Dyskinesias Targets Develop Non-Motor Trophic Symptoms Factors 8

Multiple approaches to altering the course of PD MJFF has committed nearly $115M to advance disease modifying therapies Cause of PD Restoration of Dopamine Biological Pathways Alpha-Synuclein: Genetic  Trophic Factors: Data  Inflammation: Inhibiting association in familial cases continues to show promise inflammation could slow the of PD; pathology evidence that increasing the levels of progression of PD trophic factors can protect LRRK2: Protein kinase brain cells in PD  Oxidative Stress: function makes LRRK2 a Decreasing oxidative stress highly druggable target can protect dopamine neurons 9

MJFF LRRK2 efforts are driving research towards the clinic MJFF focuses on four key areas within LRRK2 research, reducing research redundancies and facilitating collaborations among investigators – over $38M spent on LRRK to date Improve Develop understanding LRRK2 of LRRK 2 research tools biology Supports Study LRRK2 LRRK2 impact therapeutic clinically development 10

LRRK2 Biology Consortium – A Collaborative Example MJFF has established the LRRK2 biology consortium across 20 research labs throughout the world. The consortium is designed to promote real time data sharing, open discussion and distribution of tools among consortium members. Mechanisms • Agree to share for sharing • Prototype data and tools compound • Overlapping shared with 18 • Website – teams approaches protocols and • Novel data collaborative • Monthly calls projects and annual developed Over 20 sites summit meeting between teams worldwide Tangible interactions 11

Expert Insight: John Dunlop, PhD LRRK2 Consortium: How collaborative science is accelerating drug development John Dunlop, PhD Chief Operating Officer Neuroscience Research Unit Pfizer 12

Repositioning compounds may lead to disease altering therapies Drug repositioning aims to test therapies already clinically available for effectiveness in PD GOAL: Mitigate the time and costs involved in finding new therapies for PD Traditional drug development pipeline Phase I Phase I Preclinical Phase II/III Regulatory safety in safety in Studies efficacy Approval controls PD patients Acceleration by repositioning drugs that are deemed “safe” therapies Phase I Phase I Preclinical Phase II/III Regulatory safety in safety in Studies efficacy Approval controls PD patients 13

Expert Insight, Mark Frasier, PhD Repositioning Pioglitazone: From Diabetes to PD Mark Frasier, PhD Director, Research Programs The Michael J. Fox Foundation for Parkinson’s Research 14

Repurposed drug made ready for significant NIH support Pioglitazone as a disease-modifying therapy Therapeutic Target Development Pre-Clinical Clinical Regulatory Discovery Testing Validation & Testing Approval Optimization I II III MJFF brokers introduction to clinicians 2004 Grant: Pre-clinical testing 2007 Grant: Dosing and Bioavailability Available compound ID 2010: NIH funded clinical trial 2010: MJFF supports biomarker add on 15

Multiple promising trials and approaches are taking place Novel Drug Targets  Hypothesis for use of trophic factors to treat PD remains viable and exciting Trophic Factors/Ceregene  Pre-clinical and early phase clinical results continue to show promise  Attempts to remove alpha-synuclein protein aggregates AFFITOPE PD01  First time a vaccine approach has been tested in the clinic for PD Repositioned Compounds  Calcium channel blocker for hypertension Isradipine  Found to be neuroprotective in pre-clinical models of PD  Increasing urate levels could both lower the risk Inosine of getting PD and slow the progression of the disease  Smoking linked to decreased risk of PD Nicotine  First test as a disease-modifying therapy in PD 16

Clear need to develop treatments for motor & non-motor symptoms MJFF has funded over $40M in research towards developing treatments for both treating dyskinesia and non-motor symptoms.  Includes cognitive dysfunction, anxiety, memory Non-Motor loss and mood disorders Symptoms  Relieving these symptoms would lead to a better quality of life for those living with PD  Uncontrolled body movements that result from dopamine-replacement therapy Dyskinesia  Breakthroughs in treating dyskinesia would expand options for treating PD 17

Funding two parallel tracks for improving symptomatic treatments MJFF continues to invest in developing new therapies as well as determining how to best assess these therapies in the clinic Efforts include: Rationale – Why prioritize?  Shown to reduce levodopa induced dyskinesia in pre-clinical studies mGluR5  Addex Pharmaceuticals and Novartis are conducting trials to test mGluR5 antagonists in PD patients  New methods of delivering levodopa that will L-Dopa Delivery result in constant blood levels compared to “peaks and valleys” currently experienced  Non-dopamine gene therapy strategy in development Neurologix  Designed to normalize brain physiology and reduce the symptoms of PD  Repurposing droxidopa (orthostatic Droxidopa hypotension) in an effort to see if it can abate gait, sleep and cognitive disorders in PD  Targeting serotonin receptors could be key in Serotonin Receptors reducing dyskinesia 18

Expert Insight: Anders Björklund, MD, PhD Advancing Treatments for Dyskinesia – Targeting Serotonin Receptors Anders Björklund, MD, PhD Professor, Department of Neurobiology Wallenbery Neuroscience Center Lund University 19

Development of a serotonin agonist as a treatment for dyskinesia Therapeutic Target Development Pre-Clinical Clinical Regulatory Discovery Testing Validation & Testing Approval Optimization I II III 2005 Grant: Initial pre-clinical testing 2008 Grant: Preclinical development Available compound ID 2009 Grant: Clinical Trial funded Industry partner - Psychogenics 20

Progress is being made in all areas of drug development Therapeutic Target Development Pre-Clinical Clinical Regulatory Discovery Testing Validation & Testing Approval Optimization I II III  MJFF has researched between 75-80% of targets being actively investigated across the PD pipeline and has validated at least 6 novel targets  There are currently 139 drugs in the discovery phase for PD and 110 drugs being tested in the clinic  Growing interest with the pharmaceutical industry in PD drug development 21

Questions & Answers Session  Anders Björklund, MD, PhD, Lund University  Deborah W. Brooks, The Michael J. Fox Foundation  John Dunlop, PhD, Pfizer, Inc., Neuroscience Research Unit  Mark Frasier, PhD, The Michael J. Fox Foundation  Irene Hegemen Richard, MD, University of Rochester  Peter Reinhart, PhD, Proteostasis  Todd Sherer, PhD, The Michael J. Fox Foundation  Andrew Singleton, PhD, National Institute on Aging/NIH 22

Thank you for your participation! For more information, please visit: www.michaeljfox.org Our 2011 Research Roundtable Series is generously supported through an educational grant from Teva Neuroscience 23

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MJFF’s Purpose, Promise and Plan for speeding new Parkinson’s treatments to patients

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