CUREBETA

Building innovative
drug discovery alliances
Addressing
dd
g causes not
o symptoms
y po
in diabetes
Evotec AG , Analyst information on CureBeta Alliance with Janssen, Hamburg,
H
2012
Forward-looking statements
s
Information set forth in this presen
ntation contains
forward-looking statements, which invo
olve a number of
risks and uncertainties.
ncertainties The forward-lo
forward looking statements
contained herein represent the judgemen
nt of Evotec as of
the date of this report. Such forward-lo
ooking statements
are neither promises nor guarantees, bu
ut are subject to a
variety of risks and uncertainties, man
ny of which are
PAGE
1
beyond our control, and which could cause actual results
to differ materially from those contemplated in these
forward looking statements.
forward-looking
statements We expressly
e pressl disclaim any
an
obligation or undertaking to release publicly any updates
or revisions to any such statements to reflect any change in
our expectations or any change in events, conditions or
circumstances on which any such statement is based.
Agenda
• CureBeta
& Strategic
c background
• Scientific vision and disccovery background & other Cure X initiatives
• Next steps
p
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2
Cure X - First-in-cllass science
a key building bloc
ck of Action Plan 2016
Evotec´s offering for Inno
ovation Efficiency
nnovate (Cure X initiatives)
3 EVT In
• First-in
n-class discovery and product
develo
opment innovations
• Modes
st R&D in
investments
estments for high upfront,
pfront
2 EVT Integrate higherr milestone and higher royalty alliances
• Integrated drug discovery allliances on partner targets
• Best-in-class integrated drug discovery alliances (multi-target projects)
1 EVT Execute
• Risk-shared performance-base
ed alliances with research fees, milestones and
royalties
• Stand alone screening, medicinal chemistry, compound management, compound profiling,…
• Highest quality solution tools and processes
• No risk-exposure, lower margin, but long-term repeatt business
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3
Summary – “Win – Win – Win” Situation
CureBeta
terms
• Upfront
p
US $ 8 Mio, p
potential milestones about US $ 200 - 300 Mio p
per p
product
(pre-clinical, clinical, regulatory, commercial),
• Royalties
• Research payments
Win/Win/Win
alliance
• Janssen: Access to an exclusive portfolio of small molecule and biologic research programs
focused on the regeneration of inssulin-producing beta cells in people with diabetes
• Evotec: Expanding leadership in beta cell regeneration - highly systematic, unbiased and
comprehensive approach to beta cell replication
• Harvard: Accelerating developme
ent of innovative science with commercial upside
Starting point
for innovation
efficiency and
external
innovation
• Pharma industry appreciates the
e approach & seems keen to enter these type of deals
• Optimal translational strategy fo
or academia or early biotech ideas as targets get
immediately on pharma grade in
nfrastructures
• Building Cure X platforms has dual
d
upside
− Building infrastructure for EVT
T Integrate business in new fields
− Building early stage project po
ortfolios for EVT Innovate alliances
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4
Great Partners
Who is CureBeta 1)?
Harvard Stem Cell Institute /
Howard Hughes Medical
Institute 2)
Evotec 2)
• Best-in-class metabolics
discovery infrastructure
• Prof. Doug Melton – key
strategic partner for Evotec
• 15 years experience in
beta cell regeneration
1
Jansse
en
• Beta cell targets, assays,
tools
Janssen
• Perfect
and
P f t discovery
di
d development
d
l
t partne
t er
• Commited to innovation in diabetes
3
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5
1) The JDRF (Juvenile Diabetes Foundation will also supp
port CureBeta
2) Evotec / Harvard Alliance - initiated in Q1 2011
• Leader
L d iin biologics
bi l i
2
Bringing best of sc
cience and industry together
Quotes from Evotec – Jan
nssen - JDRF
"Our collaboration with Doug Melton‘s laboratory has been extremely succcessful on multiple
levels We have not only achieved our scientific goals of creating a superrior beta cell drug
levels.
discovery platform and generating a deep pipeline of novel and exciting targets,
t
but we have
also established a new model of collaboration between academia and ind
dustry that has proven
highly efficient and effective in accelerating innovative scientific developm
ment. Janssen
Pharmaceuticals perfectly complements this effort, bringing in world-lead
ding pharmaceutical
d
development
l
t expertise
ti as wellll as th
the necessary resources tto execute
t on
n our mission
i i tto
produce first-in-class therapeutics designed to restore beta cell mass and
d function."
Dr. Cord Dohrmann
Chief Scientific Officer of Evotec
“Beta cell survival and function play a critical role in maintaining
normall glucose
l
llevels
l and
d when
h th
they are compromised,
i d can
contribute to the onset of diabetes. This collaboration strengthens
our long term diabetes development pipeline and directly reflects
our commitment to making a difference for the millions of people
worldwide living
g with this disease.”
Peter DiBattiste, M.D., Global Therapeutic Head, Cardiovascular and
Metabolism at Janssen
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6
“We are delighted to see Evotec, Dr. Doug Melton, and
Janssen launch this collaborative partnership and
commitment to develop innovative regenerative
medicine therapies for diabetes. JDRF looks forward to
supporting the CureBeta initiative.
initiative ”
Dr. Richard Insel, Chief Scientific Officer
Juvenile Diabetes Research Foundation (JDRF)
Agenda
• CureBeta
& Strategic background
• Scientific vision and discovery background & other Cure X
initiatives
• Next steps
PAGE
7
Why is there such a dramatic unmet medical need?
Important Facts about Dia
abetes
1
Two major forms of diabetes type 1 and type 2
Insufficient beta cell function common to both
2
More than 346 million people worldwide have diabetes
183 million people (50%) with diabetes are undiagnosed
3
Diabetes caused 4.6 million deaths in 2011
Predicted to become the 7th leading cause of death in the
e world by the year 2030
4
Diabetes is a leading cause of blindness, amputation and kidney failure
Comorbidities associated with diabetes represent a tremend
dous burden to society
5
Diabetes caused at least USD 465 billion dollars in healthc
care expenditures in 2011;
11% of total healthcare expenditures in adults (20-79 years old)
6
Most people with diabetes are between 40-59 years of age
e
P ti t population
Patient
l ti iis getting
tti continuously
ti
l younger
7
Top selling drug in 2011: Lantus (Insulin analogue) – $ 4.9 billion
Followed by Januvia – $ 3.3 billion
8
U.S.
S is the single largest market in diabetes at roughly 37%
3 % off the global market
Percentage will drop to 28% by 2018, primarily as a result of
o significant growth in Asia/Pacific
9
No disease modifying therapies on the market or in late stages of clinical development
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8
1) Taken from IDF’s Diabetes Atlas, 5th edition, 2011 and from
f
the WHO
Multiple
p highly
g y inn
novative, p
potentially
y disease
modifying approac
ches
Evotec’s unique diabetes franchise
f
Targeting the root causes of diabetes
• Type 1 diabetes (Andromeda /Teva)
Metabolic alliances
− DiaP
Pep277 targeting autoimmunity, HSP60 derived peptide
− Sign
nificant milestones, product royalties
− Stattus Phase III
• Type 2 diabetes (Boehringer Ingelheim)
− Insu
ulin sensitizer targeting insulin resistance, undisclosed target identified
and validated by Evotec
− Up
U tto € 250 m milestones,
il t
significant
i ifi
t royalties
lti
− Stattus preclinical
Pain &
Inflammation
alliances
CNS
alliances
Oncology alliance
• Type 1 and 2 diabetes (MedImmune/AstraZeneca)
− Therapeutic protein targeting beta cell mass, undisclosed target identified
and validated by Evotec
− Up to
t € 230 m milestones, significant royalties
− Stattus preclinical
• CureB
Beta ( Harvard – Evotec – Janssen))
− Larg
gest collaboration in beta cell regeneration,
undisclosed targets identified and validated by Harvard / Evotec
− Stattus discovery
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9
Combining
g top
p sciience with
platforms for big unmet
u
medical need
What defines first-in-classs innovation infrastructure?
Focus
Integrate
Engineer
Screen
disease modifying
mechanisms
emerging insights in
disease biology
models with higher
predictability
systematically,
unbiased &
comprehensive
• Clear benefit to patients • Align with key opinion
leaders in academia
• Obvious differentiation
to existing therapies
• Tractable in drug
discovery process
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10
• Relevance to human
disease
• Establish new
collaboration models
• Proximity to disease
specific mechanism
• Acquire innovative
tools and assets
• Combine HC readouts
g p
with throughput
• Leverage disease
models for targets
and compounds
• Avoid biased
approaches
• Comprehensive
p
data
sets improve decision
making
Systematic,
y
unbias
sed and comprehensive
p
mechanism-focuse
ed infrastructures
The CureBeta “ “Autobah
hn”
High
R
Resolution
l ti
Expression
Profiling
• Transcriptome / proteome analysis
• Profiling of specific cells and tissues
− Disease relevant mechanisms
ssion
− Longitudinal analysis of disease progres
• Mechanisms focused cellular assay
− Primary cells / tissues
Mechanism
− Immortalized cells
Focused
• High content screening of
Chemical
− Annotated libraries
Genomics
− Diverse libraries
− Natural products
High
Content
Functional
Genetics
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11
•
•
•
•
Sophisticated high content cellular asssay
Loss of function
Gain of function
High content readouts
First in class
Candidate
Leads /
Targets
Pipeline of
selected
Targets and
Leads
advance into
Drug
Discovery
Process
CureBeta - – Esta
ablishing infrastructure standards
Targets, assays, models, an
nalysis…
1
Unique primary islets assays suitable for
HTS/HCS
• Highly reliable source of mammalian islets
with minimal variability compared to alternative
islet sources
• HCS capabilities and expertise based on
the Evotec Opera systems – leading HCS
system worldwide
• Industry leading quantitative chemoproteomics
capabilities
Highly comprehensive, fully automated,
quantitative analysis of pancreatic tissue
• Most comprehensive and robust analysis of
pancreas and islets currently available
• Includes overall pancreas morphology
morphology, islet
morphology, islet composition, beta cell
number, beta cell replication, beta cell
size/morphology
Targets stimulation of beta cell replication with unparalleled efficacy / selectivity
• Synthetic peptides
• Secreted factors
• Small molecule
3
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12
2
Islets Degeneration
in Type 2 diabetes
Pancreas of a male ZDF rat
Red: BrdU; green: Insulin; blue: DAPI
21 megapixel image composite taken at
10x magnification
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13
Key mechanisms regulating
r
beta cell mass
Beta cell replication and in
nsulin resistance
Increased beta cell mass in obese humans
Mouse islets transplanted into insulin resistant mice
+50%
Butler et al.,
Diabetes 2003
Human islets transplanted under ob/ob kidney capsule
Tyrberg et al.,
DIABETES, 2001
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14
Flier et al.,
PNAS 98, 2001
Increased beta cell mass in iLIRKO mice
Escribano et al.,
Diabetes 2009
Building a Cure X pipeline
High unmet medical need and paucity of disease modifying targets…
CureBeta
• Started collaboration with Harvard University in March 2011
• Partnered with Janssen in July 2012
CureNephron
• Started collaboration with Harvard University in February 2012
• Highly productive in gene
erating candidate targets & compounds
• Marketing efforts will start in September 2012
CureNeuron
• Concept phase for intern
nal projects
• Exploring potential collab
boration opportunities
CureHeart
• Concept phase for intern
nal projects
• Exploring potential collab
boration opportunities
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15
Agenda
• CureBeta
& Strategicc background
• Scientific vision and disccovery background & other Cure X initiatives
• Next steps
p
PAGE
16
CUREBETA - disru
uptive innovation in diabetes
BETALunch in Harvard: The
T root cause – Not the symptoms
BETALunch, Scientific Workshop, Fall 2012 1)
Workshop / Scientific Seminar on
• Disease modifying drugs in diabetes
• Beta
B t cellll mass and
d ffunction
ti are pivotal
i t l ffor th
the
development of disease modifying drugs
• Systematic, unbiased and comprehensive apprroach to
b t cellll regeneration
beta
ti
• Unique tools and models for the target/drug discovery
and rapid development
• Combining leading expertise in academic beta cell
c
biology, biotech discovery and pharma develop
pment
Key
y Contributions
Doug Melton, others
Prof Doug Melton
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17
1) By invitation only
Strong news flow to come
Outlook and next steps forr 2012 ff
Key milestones for 2012
1 EVT Execute
• Double digit revenue gro
owth 2012 – 2016
• Expansion success of exxisting alliances
• Significant long-term dea
als with major pharma
2 EVT Integrate
• At least
l
t 2 significant
i ifi
t new
w integrated
i t
t d technology/disease
t h l
/di
alliances
lli
• Deliver significant and accelerated
a
preclinical/clinical milestones
• Show operational synerg
gies of recent acquisitions
3 EVT Innovate
• At least 1 strategic deal for early assets
• Even more innovation upsides (e
(e.g.
g Cure X,
X …))
• Phase III data in DiaPep
p277 and Phase IIb start within product
development partnership
ps
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18
Building innovative
drug discovery alliances
Your contact:
Dr Werner Lanthaler
Chief Executive Officer
+49.(0).40.560 81-242
+49.(0).40.560 81-333 Fax
[email protected]