Parkinson Disease, Jeffery Massey, Nonprofit

1. MJFF Strategy for Accelerating Development of Parkinson’s Therapies Research Roundtable Chicago, Illinois June 27, 2011

2. Today’s Agenda MJFF Overview Deborah W. Brooks The Michael J. Fox Foundation for Parkinson’s Research MJFF Program Highlights Sonal S. Das, PhD The Michael J. Fox Foundation for Parkinson’s Research Panelists 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 Questions & Answers Session 2

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

4. 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. 4

5. MJFF was founded with clear objectives Drive the Best Parkinson’s Research Deliver Improved Therapies and a Cure 5

10. The science is ahead of the money

11. We are not a bank, assets must be deployed quickly to the best science

13. Strategy must focus on tackling roadblocks to progress in a systemic fashion, i.e. target validation, research tools, clinical trial design, recruitment

14. More proactive outreach and engagement with industry partners is required

15. Turning our knowledge out can ensure others benefit from the field knowledge we are developing

16. MJFF is in a unique position to bring all constituents together in the name of faster progress2005-11 8 8

18. View global field and identify most promising targets

20. Develop and share essential tools

22. Showcase top ideas to industry

23. Tirelessly pursue smart, creative solutions to challengesDE-RISK PD 9

24. 10 2011 MJFF Program Highlights Need for Disease-Modifying Therapies Biomarkers and The Parkinson’s Progression Markers Initiative Improving Symptomatic Treatments Sonal S. Das, PhD Associate Director, Research Programs The Michael J. Fox Foundation for Parkinson’s Research

25. MJFF priority area efforts drive progress in key research topics 11

26. Altering the course of PD: Need for Disease-Modifying Therapies 12 MJFF has committed over $100M to advance disease modifying therapies and seeks a path forward to patients.

27. Expert Insight, Jeffrey H. Kordower, PhD 13 Disease-Modifying Therapies Jeffrey H. Kordower, PhD Director, Research Center for Brain Repair The Jean-SchweppeArmour Professor of Neurological Sciences Rush University Medical Center

29. Efficacy testing is a long process, diminishing patent value

30. Reliance on clinical measures alone confound trial interpretation

31. There are no biological indications of underlying disease that complement clinical changesMarkers of progression are essential to achieve near-term hopes for needed therapies Challenges remain in testing disease-modifying therapies

33. Assist with patient selection for clinical studies…Are we studying the right population of people?

34. Facilitate measurement of modifications in the disease - could provide valid clinical trial endpoints

35. Help guide clinical trial design parameters like patient numbers, stratification, duration of treatment

36. Is the therapeutic reaching its target?

37. 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 15

39. A pre-competitive collaboration between government and industry.

40. 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.

44. Breakthroughs in treating dyskinesia would expand options for treating PDMJFF is funding two parallel tracks in dyskinesia research: Developing new therapies Determining how to best assess these therapies in the clinic (Dyskinesia Rating Scale Study)

45. Expert Insight: Christopher G. Goetz, MD 19 Advancing Treatments for Dyskinesia Christopher G. Goetz, MD Professor of Neurological Sciences Professor of Pharmacology Rush University Medical Center Co-Principal Investigator, Dyskinesia Rating Scale Glenn T. Stebbins, PhD Professor of Neurological Sciences Rush University Medical Center Co-Principal Investigator, Dyskinesia Rating Scale

47. 85% of clinical trials finish late due to recruitment troubles

48. Estimates hold that fewer than 1 in 10 people with Parkinson’s participate in clinical trialsEmpowered coordinators Smart matches Active alert system Robust content 20

50. Sonal S. Das, PhD The Michael J. Fox Foundation for Parkinson’s Research

51. Christopher G. Goetz, MD Rush University Medical Center

52. Jeffrey H. Kordower, PhD Rush University Medical Center

53. Tanya Simuni, MD, Northwestern University

54. Glenn T. Stebbins,PhD, Rush University Medical Center21

55. 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