presentation from dr. alan lewis
DESCRIPTION
Alan J. Lewis, PhD. PowerPoint presentation from JDRF's Tenth Annual Spring Research Briefing and Reception.TRANSCRIPT
Research Update
Alan J. Lewis, PhDPresident & CEO
April 6, 2009
2
JDRF Mission
To find a cure for diabetes and its complications through the support of research.
3
JDRF: Constituents and Roadmap
JDRF Acts as: Catalyst / Driver Funder Advocate
T1D “Cure”
Product A
Academic Community
Biotech Companies
Pharma and Device
CompaniesProduct B
Product C
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We are Building a Bridge to the Cure!
5
Type 1 Diabetes Landscape
Epidemic of T1D Incidence increasing worldwide (3%/year)
– 20-22 cases per 100,000 (US)– 50-60 cases per 100,000 (Finland)– Predominantly in children under 10 years
Unclear how many people diagnosed with T1D/year – 15,000-20,000?
Need for patient registry to identify, monitor patients plus allow registration for clinical trials
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Current Treatment For Type 1 Diabetes Relies On Insulin Replacement
Insulin is the only current therapeutic option Can cause increase in severe hypoglycemia and weight gain Glucose control NOT being met even with new insulin formulations
1st Line50% of patients
1 year
Basal-Bolus
2nd Line 3rd Line
Addition of insulin pumps and use of Symlin as co-therapy
4th LineLast Line
10% of patients
N/A
Organ Transplantation
• Kidney• Kidney+Pancreas• Islet Cell
40% of patients receiving pumps
Source: L.E.K. Interviews and Analysis
Increasing Severity
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Pancreas Derived Endocrine Cells
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Restore, Preserve Pancreatic Beta Cell Function
– Beta Cell Replacement– Beta Cell Regeneration
Reverse, Prevent Autoimmunity and Restore Immunoregulation
Restore Metabolic Control
Arrest and Reverse Diabetic Complications
Therapeutic Integration Requires Close Collaboration across JDRF Therapeutic Areas
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JDRF’s Research: Type 1 Diabetes Person/Patient Centric Orientation
Established Diabetes
Recent Onset
DiabetesPre-diabetes
Prevent Beta Cell
Destruction
Preserve Residual
Beta Cells
Restore Beta Cells, Beta Cell Function
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Regeneration– Novel beta cell regeneration targets and lead small
compounds identified – Gastrin-based regenerative therapeutics in clinic– Reprogramming of non-endocrine pancreatic cells to beta
cells
Replacement– Human embryonic stem cell differentiation to precursors that
mature to become fully functional in animals – Improved results of cadaveric islet transplantation
Autoimmunity– Repositioning antigen non-specific immunotherapeutics– Phase II and III clinical trials of antigen-specific and antigen
non-specific immunotherapeutics
Therapeutic Area Programs: FY09 Key Developments
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We Did It!
March 9, 2009 – President Barack Obama rescinds the directive limiting federal funding of hESC research
NIH still needs to develop guidelines and Congress to codify Executive Order
Opposition expected to limit guidelines
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Replacement and Regeneration
Small Molecules Biologics
hESCß-cell
ProgenitorsInsulin
Producing ß-cell
iPS
T1D Patient
1
2
3 4
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Functional Islets from hESCsToward a renewable source of
pancreatic -cells
Kroon et al. v.26; April 2008
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Adult Human Islet vs. hESC “Islet” Cell Grafts
Human Islet Graft Implant days : 360
hESC-Islet Graft Implant days : 377
Glucagon Somatostatin Insulin
15Immune Cells
Complement
02
Nutrients
GLUCOSE INSULINC-Peptide
HumanIslet
PEG
25-50
Delivery of Human Islets Without Immunosuppression
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Future of…Insulin Producing Cell Transplantation:
Islet cell transplantation still in its infancy (600 vs. 400,000 solid organ transplants)
Select cell population for further development– Safe and effective
Immunoprotection necessary– Immunomodulators with less side effects– Immune tolerance– Encapsulation
Transformation of treatment of T1D and T2D possible.
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Metabolic Control– JDRF’s CGM clinical trial results > change in
reimbursement
Complications– Diabetic eye disease Phase III clinical trials
Therapeutic Area Programs:FY09 Key Developments
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JDRF’s Artificial Pancreas Project
Accelerate the availability and adoption of a first generation artificial pancreas
Ensure devices from multiple companies are approved and reimbursed, encouraging investment in next generation technologies
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JDRF Artificial Pancreas Outreach
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JDRF’s Role in Catalyzing Reimbursement and Adoption of CGM
Continuous glucose monitors (CGM): new tools for control (Continuous readings, Trends, Alarms)
Three models approved by FDA, but health plan coverage limited
JDRF funded independent, multicenter clinical trial to provide data to support reimbursement and clinician adoption – accelerate path to next generation of devices
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JDRF Continuous Glucose Monitor Trial
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CGM group age 25yrs and above demonstrated significant improvements across all measures of glycemic control, without increasing hypoglycemia
Limited benefits were observed in patients ages 15-24yrs – likely due to limited CGM use in some patients
CGM group age 8-14 yrs had significant improvements across many measures of glycemic control
Twice as many reduced A1c by 10%, which reduces risk of long-term microvascular complications by about 40%
Increased time wearing CGM was associated with better outcomes in all three age groups
JDRF CGM Trial: Key Findings
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JDRF – Pharma Strategy
Partnering with Pharma is critical Importance of a push-pull strategy to advance and bundle IP
and of Pharma commitment Fostering a Pharma commitment to cell-based therapy, closed
loop, diabetes vaccine development, combination therapies, repositioning drugs for T1D
Educating/informing Pharma (Overlap of T1D/T2D, IP, market opportunities, etc.)
JDRF-Pharma Partnerships– GNF– J&J, Pfizer, Merck, GSK, BMS, Lilly, NovoNordisk, Sanofi,
Vaccine Industry, Genzyme, etc.– Various structures, experiments, non-exclusivity
Multi-Pharma alliance in precompetitive space– Biomarkers, imaging, animal models, regulatory
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JDRF commitment to keeping individuals with diabetes healthy to benefit from a cure when developed
Keeping our stakeholders engaged Short-term clinically impactful deliverables Created Department of Medical Affairs
JDRF as the Patient Advocate for a Cure
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No DKA: Community outreach program to educate public on symptoms and signs of DKA to prevent mortality and prolonged hospitalization at diagnosis of T1D
Prevention of Diabetic Eye Disease: Improved implementation of standard of care
Development of uniform standard of care for islet cell transplantation
Pregnancy and Diabetes: Increased public awareness of need for early first trimester medical care
Proposed Short-term Deliverables Related to Potential Immediate T1D Clinical Impact
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Progress Towards the Cure…One Step at a Time Tremendous research progress over past 20 years Good at predicting disease Several agents under clinical evaluation (anti-CD3,
anti-CD20 and GAD65 vaccine) Combinations of therapies likely to be next step (more
efficacious and synergistic) Need to determine efficacy then take them to
prevention studies Excellent progress to control blood sugar
– Artificial pancreas driving this ahead.
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22 20
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88
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60
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100
'02 '03 '04 '05 '06 '07 '08 '09 '10 '11
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0
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150
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'98 '99'00 '01 '02 '03'04 '05 '06 '07'08 '09 '10 '11
$ M
illio
ns
Total Research Commitments
New Research Commitments
*projected
*
*
*
*
*
*
$ M
illio
ns
*projected
Historical Context for Current FY09- FY11 Projections
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