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1 An Invitation to Innovation! Louis J DeGennaro, PhD Executive Vice President Chief Scientific Officer The Leukemia & Lymphoma Society

Leukemia Lymphoma Society: An Invitation to Innovation

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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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Page 1: Leukemia Lymphoma Society: An Invitation to Innovation

1

An Invitation to Innovation!

Louis J DeGennaro, PhDExecutive Vice PresidentChief Scientific Officer

The Leukemia & Lymphoma Society

Page 2: Leukemia Lymphoma Society: An Invitation to Innovation

Anyone can get blood cancer

In 2009:

One million North Americans

Page 3: Leukemia Lymphoma Society: An Invitation to Innovation

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

Page 4: Leukemia Lymphoma Society: An Invitation to Innovation

4

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

Page 5: Leukemia Lymphoma Society: An Invitation to Innovation

Research Value Proposition: CML & Gleevec

55%

5yr survival

Lives Saved

90%

Page 6: Leukemia Lymphoma Society: An Invitation to Innovation

The Pathway to Therapies

Research Grant

Program

TherapyAcceleration

Program

Discovery Development Commercialize

New Therapies!

7 - 10 years?

Page 7: Leukemia Lymphoma Society: An Invitation to Innovation

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

Page 8: Leukemia Lymphoma Society: An Invitation to Innovation

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

Page 9: Leukemia Lymphoma Society: An Invitation to Innovation

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

Page 10: Leukemia Lymphoma Society: An Invitation to Innovation

10

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

Page 11: Leukemia Lymphoma Society: An Invitation to Innovation

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

Page 12: Leukemia Lymphoma Society: An Invitation to Innovation

12

An Invitation to Innovation!

Louis J DeGennaro, PhDExecutive Vice PresidentChief Scientific Officer

The Leukemia & Lymphoma Society

Page 13: Leukemia Lymphoma Society: An Invitation to Innovation

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

28

Page 14: Leukemia Lymphoma Society: An Invitation to Innovation

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

Page 15: Leukemia Lymphoma Society: An Invitation to Innovation

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

Page 16: Leukemia Lymphoma Society: An Invitation to Innovation

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

Page 17: Leukemia Lymphoma Society: An Invitation to Innovation

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

Page 18: Leukemia Lymphoma Society: An Invitation to Innovation

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