Presentation from Dr. Alan Lewis

Research Update Alan J. Lewis, PhD President & CEO April 6, 2009

JDRF Mission
To find a cure for diabetes and its complications through the support of research.

JDRF: Constituents and Roadmap
JDRF Acts as:
Catalyst / Driver
Funder
Advocate
T1D “Cure” Product A Academic Community Biotech Companies Pharma and Device Companies Product B Product C

We are Building a Bridge to the Cure!

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

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 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
Organ Transplantation
Kidney
Kidney+Pancreas
Islet Cell
40% of patients receiving pumps Source: L.E.K. Interviews and Analysis Increasing Severity

Pancreas Derived Endocrine Cells

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

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

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

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

Replacement and Regeneration
Small Molecules Biologics
ß -cell Progenitors T1D Patient hESC Insulin Producing ß-cell iPS 1 2 3 4

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 Glucagon Somatostatin Insulin

Delivery of Human Islets Without Immunosuppression Immune Cells Complement 0 2 Nutrients GLUCOSE INSULIN C-Peptide Human Islet PEG 25-50 

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.

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

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

JDRF Artificial Pancreas Outreach

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

JDRF Continuous Glucose Monitor Trial

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

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

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

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

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.

$ Millions Total Research Commitments New Research Commitments *projected * * * * * * $ Millions *projected Historical Context for Current FY09- FY11 Projections

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Proceeds to JDRF!
“ Thank you for all your support”
JDRF Family

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Presentation from Dr. Alan Lewis

by JDRF New England Chapter on Apr 16, 2009

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Presentation from Dr. Alan Lewis — Presentation Transcript