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Leukemia Lymphoma Society: An Invitation to Innovation - as presented at the Partnering for Cures meeting, December 2, 2009, New York
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1
An Invitation to Innovation!
Louis J DeGennaro, PhDExecutive Vice PresidentChief Scientific Officer
The Leukemia & Lymphoma Society
Anyone can get blood cancer
In 2009:
One million North Americans
3
The Leukemia & Lymphoma Society
• Mission: Cure leukemia, lymphoma and myeloma while improving the quality of life for patients and their families.
• Advocacy-Patient Services-Research
• LLS is largest private funder of blood cancer research. FY2009 Research spend = $72 million ($250 million committed to 400 active projects)
• Our Vision: blood cancer is eradicated
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Goal & Strategy
Goal: significantly improve the standard of care
Multi-pronged strategy:• Raise & deploy capital
– Fund discovery research in academia– Advance development-stage projects to Phase I– Alliances with pharma & biotech companies
• Partner our pipeline– Alliances with pharma & biotech– Share risk and cost with partners
Research Value Proposition: CML & Gleevec
55%
5yr survival
Lives Saved
90%
The Pathway to Therapies
Research Grant
Program
TherapyAcceleration
Program
Discovery Development Commercialize
New Therapies!
7 - 10 years?
Grant Portfolio Overview by Disease
$21.2 MM (32.4)%
$4.3 MM (6.6)%
$8.5 MM (13.0%)$3.6 MM (5.4%)
$28 MM (42.7%)Leukemia
Lymphoma
Myeloma
Basic Biology Gen IM/SCT
$68 MM; 400 active projects
CPX Project - AML
• Test a panel of 5000 FDA approved, generic drugs
• CPX– kills AML cells in patient samples
• Toe Nail Fungus Cream
Our Challenge: • Create an oral formulation – done• Regulatory approval for clinical trial – opened in October• Accelerate it – 9 months from concept to trial
LLS Therapeutic Pipeline
Discovery Preclinical Phase I Phase II Phase III Patients
Lymphoma - PTLD
Acute Leukemia - AML
Lymphoma – CLL, FL
Lymphoma - CLL
Acute Leukemia
Lymphoma
AML
Multiple Trials
Novel Drug Delivery
Multiple Project Analysis
Repurposed drug novel indication
Antisense Inhibitor
Small Molecule
Engineered Immunotherapy
Vaccine
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Budget
• FY09 Audited Financials– Revenue: $288 million– Expenses: $280 million– 76% ($213 million) deployed to mission
– We are not a bank: we raise and deploy these dollars each year
Why donate to, or partner with, LLS?
• Proven research strategy– 400 cutting-edge research projects – A 50-project drug development pipeline
• Industry-quality approach– Competitive intelligence– Project management expertise – Smart, non-dilutive capital– Access to Key Opinion Leaders
• A sense of urgency
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An Invitation to Innovation!
Louis J DeGennaro, PhDExecutive Vice PresidentChief Scientific Officer
The Leukemia & Lymphoma Society
Understanding the unmet needsNew cases per year
Deaths per year
5 year overall survival rate
Drugs in development
MM 20,500 10,600 35% 56
NHL 63,000 19,000 69% 104
HL 8,500 1,300 86% 27
CML 5,000 500 89% 42
CLL 15,500 4500 79% 55
AML 12,800 9,000 60% Children
15% Adults
70
ALL 5,800 1,400 90% Children~50% Adults
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Significant Market Opportunity in Blood Cancers
Indication 2005 2009 est. 2012 est.
CLL 1.661 3.674 5.585
NHL 2.695 3.937 5.538
AML .152 .262 .941
Multiple Myeloma
.965 2.943 3.814
CML 1.661 3.674 5.585
ALL, MDS, Hodgkin’s
.493 .995 1.265
Total 7.626 15.485 22.728
Global Sales by Indication
(US$ Billions)
Gleevec Sprycel Rituxan Velcade
2.6 .102 2.852 .265
US only
3.2 7 4.646 WW sales
1.203WW sales
Current Reported Sales Followed by Estimates for 2010
(US$ Billions)
Per Espicom Business Intelligence
In The Historical Record
• 1995: Stem Cell IP > licensed to Athersys
• 1996: “Hedgehog IP > partnered with Genentech, compounds in trials
• 1997: Uses of Inorganic Arsenicals > marketed as Trisenox by Cephalon
• 1998: Second generation Proteosome Inhibitor > licensed to Proteolix , compound in Phase II, myeloma
• 1998: Vertebrate Apoptosis Gene > led to formation of Idun, acquired by Pfizer
Portfolio by Target, Mechanism & Modality
0
10
20
30
40
50
60
70
80
Tyrosi
ne Kin
ase
Kinas
e (N
ot Tyr
osine)
Met
hyl/A
cety
l
Anti-se
nse
Diagnost
ic/B
iom
arke
r
Transc
riptio
n
Cell C
ycle
Cell D
ivis
ion
Apoptosi
s
Smal
l Mole
cule
Antibody
Vacci
ne
Imm
unother
apy
Transp
lant
Stem
Cel
l
Cell ProcessesTargeted Therapy Treatment Modality
Portfolio Disease Detail
$5
$10
$15
$20
$25
$30
$35
$40
HL
NHL
CLL, SLL
Gen
eral
LY
AML
CML
ALLO
M
Gen
eral
L
Mye
lom
a
Gen
IM/S
CT
Basic
Biolo
gy
Dol
lars
Fu
nd
ed in
Mil
lion
s
$40
$35
$25
$15
$20
$5
$10
$0
$30
44.9% 34.2% 15% 5.9%% $
Disease
Development Project Portfolio
21
212 23
Scale
Up
IND
PrepTox
Form
Dev
PK &
ADME
Med
Chem
IND
Sub
In vivo
POP
21
Clinical
Trials
10
1
62 Pipeline projects28 on Watch List 26 Grants in 2009 with potential to move into development
~350 DiscoveryProjects
TH
ER
AP
IES