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Research Update Alan J. Lewis, PhD President & CEO April 6, 2009
JDRF Mission <ul><li>To find a cure for diabetes and its complications through the support of research. </li></ul>
JDRF: Constituents and Roadmap <ul><li>JDRF Acts as: </li></ul><ul><li>Catalyst / Driver </li></ul><ul><li>Funder </li></u...
We are Building a Bridge to the Cure!
Type 1 Diabetes Landscape <ul><li>Epidemic of T1D </li></ul><ul><li>Incidence increasing worldwide (3%/year) </li></ul><ul...
Current Treatment For Type 1 Diabetes Relies On Insulin Replacement <ul><li>Insulin is the only current therapeutic option...
Pancreas Derived Endocrine Cells
<ul><li>Restore, Preserve Pancreatic Beta Cell Function </li></ul><ul><ul><li>Beta Cell  Replacement </li></ul></ul><ul><u...
JDRF’s Research: Type 1 Diabetes Person/Patient Centric Orientation  Established Diabetes Recent Onset Diabetes Pre-diabet...
<ul><li>Regeneration </li></ul><ul><ul><li>Novel beta cell regeneration targets and lead small compounds identified  </li>...
We Did It! <ul><li>March 9, 2009  – President Barack Obama rescinds the directive limiting federal funding of hESC researc...
Replacement and Regeneration <ul><li>Small Molecules Biologics </li></ul>ß -cell Progenitors T1D Patient hESC Insulin Prod...
Functional Islets from hESCs Toward a renewable source of pancreatic   -cells  Kroon et al. v.26; April 2008
Adult Human Islet  vs.  hESC “Islet” Cell Grafts Human Islet Graft  Implant days : 360 hESC-Islet Graft Implant days : 377...
Delivery of Human Islets  Without Immunosuppression Immune Cells Complement 0 2 Nutrients GLUCOSE INSULIN C-Peptide Human ...
Future of… Insulin Producing Cell Transplantation: <ul><li>Islet cell transplantation still in its infancy  (600 vs. 400,0...
<ul><li>Metabolic Control </li></ul><ul><ul><li>JDRF’s CGM clinical trial results > change in reimbursement </li></ul></ul...
JDRF’s Artificial Pancreas Project <ul><li>Accelerate the availability and adoption of a first generation artificial pancr...
JDRF Artificial Pancreas Outreach
JDRF’s Role in Catalyzing Reimbursement and Adoption of CGM <ul><li>Continuous glucose monitors (CGM): new tools for contr...
JDRF Continuous Glucose Monitor Trial
<ul><li>CGM group age 25yrs and above demonstrated significant improvements across all measures of glycemic control,  with...
JDRF – Pharma Strategy <ul><li>Partnering with Pharma is critical </li></ul><ul><li>Importance of a push-pull strategy to ...
<ul><li>JDRF commitment to keeping individuals with diabetes healthy to benefit from a cure when developed </li></ul><ul><...
<ul><li>No DKA : Community outreach program to educate public on symptoms and signs of DKA to prevent mortality and prolon...
Progress Towards the Cure…One Step at a Time <ul><li>Tremendous research progress over past 20 years </li></ul><ul><li>Goo...
$ Millions Total Research Commitments New Research Commitments *projected * * * * * * $ Millions *projected Historical Con...
<ul><li>Buy a copy! </li></ul><ul><li>Proceeds to JDRF! </li></ul><ul><li>“ Thank you for all your support” </li></ul><ul>...
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Presentation from Dr. Alan Lewis

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Alan J. Lewis, PhD. PowerPoint presentation from JDRF's Tenth Annual Spring Research Briefing and Reception.

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Presentation from Dr. Alan Lewis

  1. 1. Research Update Alan J. Lewis, PhD President & CEO April 6, 2009
  2. 2. JDRF Mission <ul><li>To find a cure for diabetes and its complications through the support of research. </li></ul>
  3. 3. JDRF: Constituents and Roadmap <ul><li>JDRF Acts as: </li></ul><ul><li>Catalyst / Driver </li></ul><ul><li>Funder </li></ul><ul><li>Advocate </li></ul>T1D “Cure” Product A Academic Community Biotech Companies Pharma and Device Companies Product B Product C
  4. 4. We are Building a Bridge to the Cure!
  5. 5. Type 1 Diabetes Landscape <ul><li>Epidemic of T1D </li></ul><ul><li>Incidence increasing worldwide (3%/year) </li></ul><ul><ul><li>20-22 cases per 100,000 (US) </li></ul></ul><ul><ul><li>50-60 cases per 100,000 (Finland) </li></ul></ul><ul><ul><li>Predominantly in children under 10 years </li></ul></ul><ul><li>Unclear how many people diagnosed with T1D/year – 15,000-20,000? </li></ul><ul><li>Need for patient registry to identify, monitor patients plus allow registration for clinical trials </li></ul>
  6. 6. Current Treatment For Type 1 Diabetes Relies On Insulin Replacement <ul><li>Insulin is the only current therapeutic option </li></ul><ul><li>Can cause increase in severe hypoglycemia and weight gain </li></ul><ul><li>Glucose control NOT being met even with new insulin formulations </li></ul>1st Line 50% of patients 1 year Basal-Bolus 2nd Line 3rd Line Addition of insulin pumps and use of Symlin as co-therapy 4th Line Last Line 10% of patients N/A <ul><li>Organ Transplantation </li></ul><ul><li>Kidney </li></ul><ul><li>Kidney+Pancreas </li></ul><ul><li>Islet Cell </li></ul>40% of patients receiving pumps Source: L.E.K. Interviews and Analysis Increasing Severity
  7. 7. Pancreas Derived Endocrine Cells
  8. 8. <ul><li>Restore, Preserve Pancreatic Beta Cell Function </li></ul><ul><ul><li>Beta Cell Replacement </li></ul></ul><ul><ul><li>Beta Cell Regeneration </li></ul></ul><ul><li>Reverse, Prevent Autoimmunity and Restore Immunoregulation </li></ul><ul><li>Restore Metabolic Control </li></ul><ul><li>Arrest and Reverse Diabetic Complications </li></ul>Therapeutic Integration Requires Close Collaboration across JDRF Therapeutic Areas
  9. 9. JDRF’s Research: Type 1 Diabetes Person/Patient Centric Orientation Established Diabetes Recent Onset Diabetes Pre-diabetes Prevent Beta Cell Destruction Preserve Residual Beta Cells Restore Beta Cells, Beta Cell Function
  10. 10. <ul><li>Regeneration </li></ul><ul><ul><li>Novel beta cell regeneration targets and lead small compounds identified </li></ul></ul><ul><ul><li>Gastrin-based regenerative therapeutics in clinic </li></ul></ul><ul><ul><li>Reprogramming of non-endocrine pancreatic cells to beta cells </li></ul></ul><ul><li>Replacement </li></ul><ul><ul><li>Human embryonic stem cell differentiation to precursors that mature to become fully functional in animals </li></ul></ul><ul><ul><li>Improved results of cadaveric islet transplantation </li></ul></ul><ul><li>Autoimmunity </li></ul><ul><ul><li>Repositioning antigen non-specific immunotherapeutics </li></ul></ul><ul><ul><li>Phase II and III clinical trials of antigen-specific and antigen non-specific immunotherapeutics </li></ul></ul>Therapeutic Area Programs: FY09 Key Developments
  11. 11. We Did It! <ul><li>March 9, 2009 – President Barack Obama rescinds the directive limiting federal funding of hESC research </li></ul><ul><li>NIH still needs to develop guidelines and Congress to codify Executive Order </li></ul><ul><li>Opposition expected to limit guidelines </li></ul>
  12. 12. Replacement and Regeneration <ul><li>Small Molecules Biologics </li></ul>ß -cell Progenitors T1D Patient hESC Insulin Producing ß-cell iPS 1 2 3 4
  13. 13. Functional Islets from hESCs Toward a renewable source of pancreatic  -cells Kroon et al. v.26; April 2008
  14. 14. Adult Human Islet vs. hESC “Islet” Cell Grafts Human Islet Graft Implant days : 360 hESC-Islet Graft Implant days : 377 Glucagon Somatostatin Insulin
  15. 15. Delivery of Human Islets Without Immunosuppression Immune Cells Complement 0 2 Nutrients GLUCOSE INSULIN C-Peptide Human Islet PEG 25-50 
  16. 16. Future of… Insulin Producing Cell Transplantation: <ul><li>Islet cell transplantation still in its infancy (600 vs. 400,000 solid organ transplants) </li></ul><ul><li>Select cell population for further development </li></ul><ul><ul><li>Safe and effective </li></ul></ul><ul><li>Immunoprotection necessary </li></ul><ul><ul><li>Immunomodulators with less side effects </li></ul></ul><ul><ul><li>Immune tolerance </li></ul></ul><ul><ul><li>Encapsulation </li></ul></ul><ul><li>Transformation of treatment of T1D and T2D possible. </li></ul>
  17. 17. <ul><li>Metabolic Control </li></ul><ul><ul><li>JDRF’s CGM clinical trial results > change in reimbursement </li></ul></ul><ul><li>Complications </li></ul><ul><ul><li>Diabetic eye disease Phase III clinical trials </li></ul></ul>Therapeutic Area Programs: FY09 Key Developments
  18. 18. JDRF’s Artificial Pancreas Project <ul><li>Accelerate the availability and adoption of a first generation artificial pancreas </li></ul><ul><li>Ensure devices from multiple companies are approved and reimbursed, encouraging investment in next generation technologies </li></ul>
  19. 19. JDRF Artificial Pancreas Outreach
  20. 20. JDRF’s Role in Catalyzing Reimbursement and Adoption of CGM <ul><li>Continuous glucose monitors (CGM): new tools for control (Continuous readings, Trends, Alarms) </li></ul><ul><li>Three models approved by FDA, but health plan coverage limited </li></ul><ul><li>JDRF funded independent, multicenter clinical trial to provide data to support reimbursement and clinician adoption – accelerate path to next generation of devices </li></ul>
  21. 21. JDRF Continuous Glucose Monitor Trial
  22. 22. <ul><li>CGM group age 25yrs and above demonstrated significant improvements across all measures of glycemic control, without increasing hypoglycemia </li></ul><ul><li>Limited benefits were observed in patients ages 15-24yrs – likely due to limited CGM use in some patients </li></ul><ul><li>CGM group age 8-14 yrs had significant improvements across many measures of glycemic control </li></ul><ul><li>Twice as many reduced A1c by 10%, which reduces risk of long-term microvascular complications by about 40% </li></ul><ul><li>Increased time wearing CGM was associated with better outcomes in all three age groups </li></ul>JDRF CGM Trial: Key Findings
  23. 23. JDRF – Pharma Strategy <ul><li>Partnering with Pharma is critical </li></ul><ul><li>Importance of a push-pull strategy to advance and bundle IP and of Pharma commitment </li></ul><ul><li>Fostering a Pharma commitment to cell-based therapy, closed loop, diabetes vaccine development, combination therapies, repositioning drugs for T1D </li></ul><ul><li>Educating/informing Pharma (Overlap of T1D/T2D, IP, market opportunities, etc.) </li></ul><ul><li>JDRF-Pharma Partnerships </li></ul><ul><ul><li>GNF </li></ul></ul><ul><ul><li>J&J, Pfizer, Merck, GSK, BMS, Lilly, NovoNordisk, Sanofi, Vaccine Industry, Genzyme, etc. </li></ul></ul><ul><ul><li>Various structures, experiments, non-exclusivity </li></ul></ul><ul><li>Multi-Pharma alliance in precompetitive space </li></ul><ul><ul><li>Biomarkers, imaging, animal models, regulatory </li></ul></ul>
  24. 24. <ul><li>JDRF commitment to keeping individuals with diabetes healthy to benefit from a cure when developed </li></ul><ul><li>Keeping our stakeholders engaged </li></ul><ul><li>Short-term clinically impactful deliverables </li></ul><ul><li>Created Department of Medical Affairs </li></ul>JDRF as the Patient Advocate for a Cure
  25. 25. <ul><li>No DKA : Community outreach program to educate public on symptoms and signs of DKA to prevent mortality and prolonged hospitalization at diagnosis of T1D </li></ul><ul><li>Prevention of Diabetic Eye Disease : Improved implementation of standard of care </li></ul><ul><li>Development of uniform standard of care for islet cell transplantation </li></ul><ul><li>Pregnancy and Diabetes : Increased public awareness of need for early first trimester medical care </li></ul>Proposed Short-term Deliverables Related to Potential Immediate T1D Clinical Impact
  26. 26. Progress Towards the Cure…One Step at a Time <ul><li>Tremendous research progress over past 20 years </li></ul><ul><li>Good at predicting disease </li></ul><ul><li>Several agents under clinical evaluation (anti-CD3, anti-CD20 and GAD65 vaccine) </li></ul><ul><li>Combinations of therapies likely to be next step (more efficacious and synergistic) </li></ul><ul><li>Need to determine efficacy then take them to prevention studies </li></ul><ul><li>Excellent progress to control blood sugar </li></ul><ul><ul><li>Artificial pancreas driving this ahead. </li></ul></ul>
  27. 27. $ Millions Total Research Commitments New Research Commitments *projected * * * * * * $ Millions *projected Historical Context for Current FY09- FY11 Projections
  28. 28. <ul><li>Buy a copy! </li></ul><ul><li>Proceeds to JDRF! </li></ul><ul><li>“ Thank you for all your support” </li></ul><ul><li>JDRF Family </li></ul>

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