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Aaron J. Kowalski Ph.D.
JDRF Chief Mission Officer
ACCELERATING PROGRESS
March 11, 2017
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OUR VISION
A W RLD WITHOUT TYPE 1 DIABETES
Accelerating Progress 2
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3
JDRF was Founded by Families Driven to Cure T1D OUR PURPOSE
Lee Ducat, Lester Salans, M.D. (NIH), Carol Lurie
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Accelerating Progress 4
THE PLAN
Doing the Greatest Good,
for the Largest Number of People,
in the Shortest Period of Time
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Accelerating Progress 5
Accelerating Progress Across the Pipeline THE PLAN
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Accelerating Progress 6
Accelerating Progress Across the Pipeline THE PLAN
TO DELIVER LIFE-CHANGING BREAKTHROUGHS
CURE Restoring Insulin Independence
PREVENT Preventing Symptomatic T1D
TREAT Improving Glucose Control
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7
WE’VE COME A LONG WAY…
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We used to measure urine glucose, there was no home blood glucose testing!
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This was my first lancet device!
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And my first test strips…
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The first artificial pancreas circa 1964…
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One of the first commercial insulin pumps, the “blue brick”
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13
BUT NOT FAR ENOUGH…
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Limited Success Achieving HbA1c Targets
14
21% 21% 17%
13%
32% 29%
0%
20%
40%
60%
80%
100%
<6 6-<13 13-<18 18-<26 26-<50 ≥50
Mean H
bA
1c
(%)
Age (years)
A1c Goal = <7.0% A1c Goal = <7.5%
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Average Current HbA1c by Age
15
7
7.5
8
8.5
9
0 5 10 15 20 25 30 35 40 45 50 55 60 65 70 75 80
Mean H
bA
1c
Age, years*
6yo
17yo
30yo
*≤2 years old and ≥80 years old are pooled
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Hypo is still common – even with incredible efforts to avoid it.
Buckingham et al. 2015.
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Continuous Therapeutic Improvements INSIDE THE PIPELINE
Targeted programs to deliver better outcomes…….
ARTIFICIAL PANCREAS COMPLICATIONS BETA CELL REPLACEMENT
GLUCOSE CONTROL (INCLUDING SMART INSULIN)
RESTORATION PREVENTION
17 The Plan for a World without T1D
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18
Moving Forward, New Therapies Must Improve Diabetes Health and Diabetes Happiness!
Diabetes Health Diabetes Happiness (QoL)
Work/Effort/Burden
ROI
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Accelerating Progress 19
Artificial Pancreas THE PLAN
Our plan is to create a future where...
Artificial Pancreas
The burden is eased all day, and you sleep safely
through the night
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Artificial Pancreas
20
TIGHTER CONTROL OF BLOOD GLUCOSE WITH REDUCED BURDEN
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We are making progress, fast….. PATHWAYS TO ARTIFICIAL PANCREAS SYSTEMS
670g Anticipated
NOW!!! Veo-worldwide
640G Australia, Denmark UK –
USA soon
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First AP-CL system on the market in 2017! PATHWAYS TO ARTIFICIAL PANCREAS SYSTEMS
BS range using open loop BS range using closed loop
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First AP-CL system on the market in 2017! PATHWAYS TO ARTIFICIAL PANCREAS SYSTEMS
BS range using open loop BS range using closed loop
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First AP-CL system on the market in 2017! PATHWAYS TO ARTIFICIAL PANCREAS SYSTEMS
BS range using open loop BS range using closed loop
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AP Systems are coming……Commercialization PATHWAYS TO ARTIFICIAL PANCREAS SYSTEMS
25 The Plan for a World without T1D
• MDT: Hybrid CL: 2017
• Animas HHM
• Bigfoot Biomedical: Hybrid CL
• Type Zero: Hybrid CL
• Boston University: Dual-hormone
• Inreda: Dual-hormone
• Tandem
• Insulet
• Roche
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26
Bigfoot Biomedical System
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Sensor Will Get Smaller and Smaller And Fingersticks Will Become Obsolete
27
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Accelerating Progress 28
Glucose Control THE PLAN
Our plan is to create a future where...
Glucose Control
Highs and lows are kept in check
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Insulin on demand Activity proportional to blood glucose levels
Improve Outcomes: Glucose Control: Reduce highs and lows; increase Time-in-Range (TIR);
reduce complications Quality of Life (QoL): Reduce burden, anxiety/fear, human errors
Priorities:
Support discovery and development of GRI therapies for individuals with insulin-dependent diabetes
Bring together individuals with diverse expertise required for GRI design
Partner to ensure accelerated translation into human studies
29
JDRF’S CONTINUED LEADERSHIP CRITICAL FOR DELIVERY OF GRI THERAPY
Glucose Responsive Insulin: A Closed Loop Drug
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GRI: A Design Concept
EVEN AN INSULIN THAT STOPS WORKING WHEN GLUCOSE LEVEL IS LOW IS A WIN
Increasing Glucose Level
Increasing Insulin Activity
Glucose
Vesicle
Insulin
Vesicle
Insulin inactive/bound by GR Chemistry at low BG
Insulin active/released by GR Chemistry at high BG
An insulin that stops working when glucose levels are low
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Accelerating Progress 31
Complications THE PLAN
Our plan is to create a future where...
Complications
Diabetes doesn’t bring other health problems
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Notes: * The 2011 Scottish Diabetes Survey did not have % of T1D patients that reached all three targets Source: NHS Scotland, L.E.K. interviews and analysis
Complications: Status Update RESEARCH HIGHLIGHTS
% of T1D patients reaching target level:
2010: 11%
2011: 13%
Target: ≤ 7%
A1C
Blood pressure
Cholesterol
2010: 3%
A + B + C*
2010: 39%
2011: 47%
Target: ≤ 130/80 mmHg
2010: 54%
2011: 71%
Target: ≤ 5 mmol / L
C
B
A
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PERL Study: Preventing Early Renal Loss
JDRF Research and Advocacy efforts lead to pivotal trial by NIH (SDP)
High blood uric acid levels predict progression of nephropathy in T1D
Allopurinol is a widely used generic drug that reduces uric acid levels
JDRF-funded PERL pilot clinical study initiated to see if allopurinol can reduce progression of nephropathy in T1D
SDP now funding pivotal study recruiting 450 people with T1D and nephropathy
RESEARCH HIGHLIGHTS
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Plasma kallikrein therapy enters the clinic for DME
Phase 1 study completed 2Q2016
RESEARCH HIGHLIGHTS
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Accelerating Progress 35
Prevention THE PLAN
Our plan is to create a future where...
Prevention
T1D can be prevented and will never threaten
anyone again
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The Plan for a World without T1D 36
T1D can affect family members and others WHY SHOULD I CARE ABOUT PREVENTION?
What is the risk for developing type 1 diabetes among family members compared to the rest of the population? No difference 3X greater risk 15X greater risk
….but ~85% of newly diagnosed T1D do NOT have a family history!
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37
Risk of T1D progression with autoantibodies IT IS NOT A QUESTION OF “IF” BUT “WHEN”……
37
100
80
60
40
20
0
0 5 10 15 20
Follow-up from Seroconversion (years)
Pro
bab
ility
of
pro
gres
sio
n t
o
typ
e 1
dia
bet
es (
%)
[JAMA. 2013;309(23):2473-2479]
51%
75%
George Eisenbarth: „The clock to T1D has started when islet antibodies are first detected”. Paradigm shift for staging of type 1 diabetes before clinical onset
And the Lifetime Risk approaches 100%
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T1D happens in stages and starts with 2 antibodies
38
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The Plan for a World without T1D 39
Multi-pronged strategy to eradicate T1D MULTIPLE CAUSES WITH MANY UNKNOWNS
Enteroviruses: Virus and islet cell damage in pancreas, viral antibodies and infection detected before onset of autoimmunity/disease T1D Genetics: IFIH1 gene affects response to enterovirus
Hygiene Hypothesis and Gut Microbiome: The JDRF Microbiome
Consortium created in 2011 to engage the community, create collaborations and drive the field forward Several vaccine projects, including vaccines to induce normal micro-
organisms in the GI tract
Insulin Resistance and Dysglycemia: Cause or Accelerator? Evaluate glycemic, b-cell specific and metabolic agents in preventing
progression in at-risk and preclinical stages
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The Plan for a World without T1D 40
T1D can affect family members and others WHY SHOULD I CARE ABOUT PREVENTION?
Risk of T1D in relatives of individuals with T1D: Identical Twin: 30-70% Multiple Affected First Degree Relatives: 20-50% Sibling: 8% (but if HLA risk genes identical:30-70%) Offspring:
Father: 5% Mother: 3%
If no Family Hx - General Population: 0.4% (but if HLA risk genes: 4%)
JDRF supporting activities toward regulatory acceptance and cost-effective screening tools for global implementation
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Accelerating Progress 41
Beta Cell Replacement THE PLAN
Our plan is to create a future where...
Beta Cell Replacement
You can skip counting carbs for months at a time
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Replacement program priorities SOLUTION/S FOR PUTTING THE BETA CELLS BACK IN THE BODY
Macro-encapsulation
Micro & nano encapsulation
Scaffold/Hydrogels Engineered cell source: with genetic modification Local immune modulators
Non-T1D T1D
b cell sources from stem cells and pigs are available but need optimization Encapsulation Systems that provide complete immunoisolation Retrievable scaffold (open concept)
Reliant on cell engineering or other forms of immune tolerance to confer immune protection
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Accelerating Progress 43
Restoration THE PLAN
Our plan is to create a future where...
Restoration
Producing your own insulin doesn’t require
a second thought
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Generating Improved b/Islet Cells through Genetic Modifications INNOVATIVE SOLUTION FOR PUTTING BETA CELLS BACK IN THE BODY
Recent advances in gene editing technologies open up the intriguing possibility of generating modified cell sources that have improved survival, function and can protect themselves from the immune system without encapsulation or immunosuppressive drugs, leading to new program focus
7 new projects to use this approach to both human and porcine islet sources
Genes to confer immune protection or increase b cell function
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Dead β cell
No insulin
JDRF Changes Dogma in β Cell Health and Survival
• Stress feedback loop • b cell demise • Accelerates autoimmunity
Relieve b cell stress -> restore b cell function and slow/stop autoimmune process
Healthy β cell
Glucose
Normal, processed insulin
Stressed β cell
Inactive, misfolded insulin
Glucose
45
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Dead β cell
No insulin
Multiple clinical studies in b cell survival
Healthy β cell
Glucose
Normal, processed insulin
Stressed β cell
Inactive, misfolded insulin
Glucose
Verapamil DFMO
TUDCA
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Continuous Therapeutic Improvements INSIDE THE PIPELINE
Right Individual Right Stage Right Therapy Right Dose
ARTIFICIAL PANCREAS COMPLICATIONS BETA CELL REPLACEMENT
GLUCOSE CONTROL (INCLUDING SMART INSULIN)
RESTORATION PREVENTION
47 The Plan for a World without T1D
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Accelerating Progress 48
Raising Awareness. Raising Funds. YOUR SUPPORT
AN OPPORTUNITY TO TURN A FAVORITE SPORT, HOBBY OR LIFE EVENT INTO A JDRF FUNDRAISER
BIKE RIDERS REACH PERSONAL TRAINING GOALS AS THEY RAISE FUNDS FOR CRITICAL RESEARCH
AN IN-SCHOOL PROGRAM GEARED TOWARD AWARENESS, EDUCATION AND FUNDRAISING FOR T1D RESEARCH
THE FUNDS RAISED BY JDRF ONE WALK® – OUR FLAGSHIP FUNDRAISING EVENT AND THE LARGEST T1D EVENT IN THE WORLD – HELP US TURN TYPE ONE INTO TYPE NONE.
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Accelerating Progress 49
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#T1DNoLimits!
#RunningwithT1D
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jdrf.org
Accelerating Progress 51
Here’s Where You Can Find Us CONTACT
o: 212-479-7512
Twitter:
@aaronjkowalski
Aaron J. Kowlaski, Ph.D.
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THANK YOU March 11, 2017