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Public-Private Partnership Model:
  The Parkinson’s Progression
        Markers Initiative




  •Sohini Chowdhury, Vice President, Research
                Partnerships

                 Sept 16, 2011
Parkinson’s is a global disease and prevalence is growing


     Parkinson’s disease (PD) is a progressive neurodegenerative disease
      that affects nearly six million worldwide.


     PD prevalence is expected to grow sizably over the next 25 years as the
      proportion of older citizens continues to increase globally.


     Current therapies address mild/moderate motor symptoms


     There is currently no therapy proven to modify – slow or halt – the
      progression of the disease.




                                                               2
Drug development is a lengthy and costly process




    Takes ~15 years for drug therapy to go through R&D and get to
    market




                                                  ~8 of those years are spent in clinical
                                                  testing




                                                                       3
Clinical testing of PD therapies is challenging




     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



     Increases risk for pharma, the main sponsors of therapeutic
                                trials
The Parkinson’s Progression Markers Initiative (PPMI)


     PPMI is a ground-breaking clinical study that aims to accelerate PD drug
         development by identifying biomarkers of Parkinson’s Disease
Critical to PPMI’s success is recruiting a cohort of 600




    21 sites in Europe and US are recruiting subjects; soon to be expanded to
                                    Australia




                                                            6
Various groups provide input/management of study facets




                                             7
Industry is a valuable partner
A strong emphasis on standardization and sharing SOPs




                                            9
A strong emphasis on recruitment




                                   PPMI Enrollment Status
                                   As of Sept 2011




                                                     10
Data made available and shared in real-time via study website




                                               11

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Sohini Chowdhury, 2011

  • 1. Public-Private Partnership Model: The Parkinson’s Progression Markers Initiative •Sohini Chowdhury, Vice President, Research Partnerships Sept 16, 2011
  • 2. Parkinson’s is a global disease and prevalence is growing  Parkinson’s disease (PD) is a progressive neurodegenerative disease that affects nearly six million worldwide.  PD prevalence is expected to grow sizably over the next 25 years as the proportion of older citizens continues to increase globally.  Current therapies address mild/moderate motor symptoms  There is currently no therapy proven to modify – slow or halt – the progression of the disease. 2
  • 3. Drug development is a lengthy and costly process Takes ~15 years for drug therapy to go through R&D and get to market ~8 of those years are spent in clinical testing 3
  • 4. Clinical testing of PD therapies is challenging  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 Increases risk for pharma, the main sponsors of therapeutic trials
  • 5. The Parkinson’s Progression Markers Initiative (PPMI) PPMI is a ground-breaking clinical study that aims to accelerate PD drug development by identifying biomarkers of Parkinson’s Disease
  • 6. Critical to PPMI’s success is recruiting a cohort of 600 21 sites in Europe and US are recruiting subjects; soon to be expanded to Australia 6
  • 7. Various groups provide input/management of study facets 7
  • 8. Industry is a valuable partner
  • 9. A strong emphasis on standardization and sharing SOPs 9
  • 10. A strong emphasis on recruitment PPMI Enrollment Status As of Sept 2011 10
  • 11. Data made available and shared in real-time via study website 11