1st q 2011 v15
TRANSCRIPT
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Compugen Ltd
Establishing a New Paradigm for
Drug and Diagnostic Discovery
1st Quarter 2011 Conference Call
May 11, 2011
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Safe Harbor Statement
This presentation contains "forward-looking statements" within the
meaning of the Private Securities Litigation Reform Act of 1995. These
statements include words like "may," "expects," "believes," and
"intends," and describe opinions about future events. These forward-
looking statements involve known and unknown risks and uncertainties
that may cause the actual results, performance or achievements of
Compugen to be materially different from any future results,
performance or achievements expressed or implied by such forward-
looking statements. These risks are more fully explained under the
heading "Risk Factors" in Compugen's annual reports filed on form 20Fthat are filed with the Securities and Exchange Commission.
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Agenda
Welcome
Update Presentations:
Status of Pipeline Program
Why & HowA Quick Review Financial Status and Outlook
Questions and Answers Session
3
Compugen Participants: Martin Gerstel Chairman Anat Cohen-Dayag, PhD President & CEO Dikla Czaczkes-Axselbrad CFO
www.compugen.co.il or www.cgen.com
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Agenda
Welcome
Update Presentations:
Status of Pipeline Program
4
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Pipeline Program - Introduction
Initiated in 2010
Currently >30 product candidates in variousstages of validation
Continuing discovery runs
Market Driven Selection Pathways, protein families and targets of
high industry interest
5
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Therapeutic Fields:
Oncology
Immunology
Drug Classes:
Therapeutic Monoclonal Antibodies (mAbs)
Therapeutic Proteins
Therapeutic Peptides
Pipeline Program: Current Focus
6
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Pipeline Program - Introduction
Initiated in 2010
Currently >30 product candidates in variousstages of validation
Continuing discovery runs
Market Driven Selection Pathways, protein families and targets of
high industry interest
Moving selected candidates further indevelopment post animal disease model
Initially 12-18 months post animal diseasemodel to pre-IND stage
7
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Pipeline Program: Oncology
8
CGEN#Type of
Biologic
Therapeutic
IndicationPathway
Prediction
and
Selection
In vitro
validation
In vivo
POC/Target
Validation
(IHC)
Lead
Candidate
/mAb & in
vivo POC
Lead
Candidate/
Pre-IND
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Pipeline Program: Oncology
9
CGEN#Type of
Biologic
Therapeutic
IndicationPathway
Prediction
and
Selection
In vitro
validation
Target
Validation
(IHC)
mAb & in
vivo POC
Lead
Candidate
& Pre-IND
CGEN-928 mAb TargetMultiple
Myeloma
Uncharacterized
gene
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CGEN 928: For Multiple Myeloma
Affect on cell viability- combination with Bortezomib
Field: Oncology
Biologic Type: Novel Drug Target (and Diagnostic)
Potential use in multiple myeloma, specifically in advanced stagesand drug-resistant subtypes
Plus synergism with standard-of-care MM therapies Potentially enhanced clinical response with combination therapy
10
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Pipeline Program: Oncology
11
CGEN#Type of
Biologic
Therapeutic
IndicationPathway
Prediction
and
Selection
In vitro
validation
Target
Validation
(IHC)
mAb & in
vivo POC
Lead
Candidate
& Pre-IND
CGEN-928 mAb TargetMultiple
Myeloma
Uncharacterized
gene
Six
undisclosedmAb Targets Epithelial Cancers
Splice variants of
known cancer
targets or
proteins
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Compugen
mAb Targets Discovery Platform
12
MED: Disease Exp Database
Integrating >70,000Microarray experiments
Product Candidates: Novel proteins and Drug Targets
LEADS: Human Transcriptome,
Proteome & Peptidome
Specialized algorithms:
Disease specific, protein family,
pathway analysis, splice
variants, protein-proteininteractions, transcriptional
regulation and more
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Pipeline Program: Oncology
13
CGEN#Type of
Biologic
Therapeutic
IndicationPathway
Prediction
and
Selection
In vitro
validation
Target
Validation
(IHC)
mAb & in
vivo POC
Lead
Candidate
& Pre-IND
CGEN-928 mAb TargetMultiple
Myeloma
Uncharacterized
gene
Six
undisclosedmAb Targets Epithelial Cancers
Splice variants of
known cancer
targets or
proteins
CGEN-15001T mAb TargetSCLC, Cancer
Immunotherapy Novel B7-like
N M b f P t i F ili Di
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New Members of Protein Families Discovery
Platform
ExperimentalValidation
Further refinedprioritization criteria
Proteins expressed ondiseased cells
Proteins with family characteristics
All human proteins
Multiple Novel
Protein FamilyMembers
In Silico
Prediction
& Selection
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Pipeline Program: Oncology
15
CGEN#Type of
Biologic
Therapeutic
IndicationPathway
Prediction
and
Selection
In vitro
validation
Target
Validation
(IHC)
mAb & in
vivo POC
Lead
Candidate
& Pre-IND
CGEN-928 mAb TargetMultiple
Myeloma
Uncharacterized
gene
Six
undisclosedmAb Targets Epithelial Cancers
Splice variants of
known cancer
targets or
proteins
CGEN-15001T mAb TargetSCLC, Cancer
Immunotherapy Novel B7-like
CGEN-671 mAb Target Epithelial CancersSplice variant of
CD55
Two
undisclosedmAb Targets
Epithelial
Cancers
Uncharacterized
genes
Two
undisclosedmAb Targets
Lung & Ovarian
CancerNovel B7-like
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Pipeline Program: Oncology
Multiple mAb Target candidates targetingepithelial cancers
Epithelial tumors, also referred to ascarcinomas, account for approximately 85%
of all cancers including the ten most prevalent cancers in the
Western world, such as breast, colorectal, lung,
ovary and prostate
16
Large Unmet Clinical Need
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Pipeline Program: Oncology
17
CGEN#Type of
Biologic
Therapeutic
IndicationPathway
Prediction
and
Selection
In vitro
validation
Target
Validation
(IHC)
mAb & in
vivo POC
Lead
Candidate
& Pre-IND
CGEN-928 mAb TargetMultiple
Myeloma
Uncharacterized
gene
Six
undisclosedmAb Targets Epithelial Cancers
Splice variants of
known cancer
targets or
proteins
CGEN-15001T mAb TargetSCLC, Cancer
Immunotherapy Novel B7-like
CGEN-671 mAb Target Epithelial CancersSplice variant of
CD55
Two
undisclosedmAb Targets
Epithelial
Cancers
Uncharacterized
genes
Two
undisclosedmAb Targets
Lung & Ovarian
CancerNovel B7-like
CGEN#Type of
Biologic
Therapeutic
IndicationPathway
Prediction
and
Selection
In vitro
validation
In vivo
POC
Lead
CandidatePre-IND
CGEN-25017 peptide OncologyAng-2/Tie2
pathway
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Field: Oncology and Other (e.g. Inflammatory and Ocular)
Biologic Type: Peptide Therapeutic
A novel peptide antagonist of angiopoietins/Tie2 pathway
Inhibits generation of new blood vessels
Potential use in various cancers, inflammatory disorders andretinal disorders
CGEN-25017: For Angiogenesis Related Diseases
18
Tiran et al. in prep.
Potent reduction of neovascularization(in vivo model of retinopathy)
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Pipeline Program: Oncology
19
CGEN#Type of
Biologic
Therapeutic
IndicationPathway
Prediction
and
Selection
In vitro
validation
Target
Validation
(IHC)
mAb & in
vivo POC
Lead
Candidate
& Pre-IND
CGEN-928 mAb TargetMultiple
Myeloma
Uncharacterized
gene
Six
undisclosedmAb Targets Epithelial Cancers
Splice variants of
known cancer
targets or
proteins
CGEN-15001T mAb TargetSCLC, Cancer
Immunotherapy Novel B7-like
CGEN-671 mAb Target Epithelial CancersSplice variant of
CD55
Two
undisclosedmAb Targets
Epithelial
Cancers
Uncharacterized
genes
Two
undisclosedmAb Targets
Lung & Ovarian
CancerNovel B7-like
CGEN#Type of
Biologic
Therapeutic
IndicationPathway
Prediction
and
Selection
In vitro
validation
In vivo
POC
Lead
CandidatePre-IND
CGEN-25017 peptide OncologyAng-2/Tie2
pathway
Four
undisclosedpeptides Oncology Cancer pathways
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Pipeline Program: Oncology
20
CGEN#Type of
Biologic
Therapeutic
IndicationPathway
Prediction
and
Selection
In vitro
validation
Target
Validation
(IHC)
mAb & in
vivo POC
Lead
Candidate
& Pre-IND
CGEN-928 mAb TargetMultiple
Myeloma
Uncharacterized
gene
Six
undisclosedmAb Targets Epithelial Cancers
Splice variants of
known cancer
targets or
proteins
CGEN-15001T mAb TargetSCLC, Cancer
Immunotherapy Novel B7-like
CGEN-671 mAb Target Epithelial CancersSplice variant of
CD55
Two
undisclosedmAb Targets
Epithelial
Cancers
Uncharacterized
genes
Two
undisclosedmAb Targets
Lung & Ovarian
CancerNovel B7-like
CGEN#Type of
Biologic
Therapeutic
IndicationPathway
Prediction
and
Selection
In vitro
validation
In vivo
POC
Lead
CandidatePre-IND
CGEN-25017 peptide OncologyAng-2/Tie2
pathway
Four
undisclosedpeptides Oncology Cancer pathways
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Pipeline Program: Immunology and Other
21
CGEN#Type of
Biologic
Therapeutic
IndicationPathway
Prediction
and
Selection
In vitro
validation
In vivo
POC
Lead
CandidatePre-IND
CGEN-15001Protein
therapeutic
Multiple Sclerosis,
Rheumatoid
Arthritis
Novel B7-like
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3 times/wk
6 daily
CGEN-15001: For Multiple Sclerosis
22
Field: Immunology : Autoimmune diseases
Biologic Type: Protein Therapeutic
Potential use in various autoimmune diseases including multiple sclerosisrheumatoid arthritis, inflammatory bowel disease and type 1 diabetes
Short-term treatment resulted in long-term amelioration of
disease symptoms and complete abolishment of relapses(in vivo model of MS)
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CGEN-15001: For Rheumatoid Arthritis
Potent reduction in disease symptoms with similar efficacyas Enbrel (Etanercept) and potentially less adverse events
(in vivo model of RA)23
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Pipeline Program: Immunology and Other
24
CGEN#Type of
Biologic
Therapeutic
IndicationPathway
Prediction
and
Selection
In vitro
validation
In vivo
POC
Lead
CandidatePre-IND
CGEN-15001Protein
therapeutic
Multiple Sclerosis,
Rheumatoid
Arthritis
Novel B7-like
Eight
undisclosed
Protein
therapeutics
Autoimmune and
Inflammatory
diseases
8 Novel B7-
like
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Pipeline Program: Immunology
Compugen Announces Discovery Platform for
New Members of Protein Families withKnown Therapeutic Applications (Nov 2010)
Platform development resulted in nine predicted
novel members of B7/CD28 Protein Family
Molecules to be further researchedunder expanded arrangement with Northwestern
University
25
Significant Clinical Potential inAutoimmune Diseases and Cancer
i li l d h
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Pipeline Program: Immunology and Other
26
CGEN#Type of
Biologic
Therapeutic
IndicationPathway
Prediction
and
Selection
In vitro
validation
In vivo
POC
Lead
CandidatePre-IND
CGEN-15001Protein
therapeutic
Multiple Sclerosis,
Rheumatoid
Arthritis
Novel B7-like
Eight
undisclosed
Protein
therapeutics
Autoimmune and
Inflammatory
diseases
8 Novel B7-
like
CGEN-25007 peptide Inflammatorydiseases (IBD)
gp96 (HSP)
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DAC Blockers Discovery Platform
27
Inactive state
Active state
Blocked state
Pi li P I l d O h
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Pipeline Program: Immunology and Other
28
CGEN#Type of
Biologic
Therapeutic
IndicationPathway
Prediction
and
Selection
In vitro
validation
In vivo
POC
Lead
CandidatePre-IND
CGEN-15001Protein
therapeutic
Multiple Sclerosis,
Rheumatoid
Arthritis
Novel B7-like
Eight
undisclosed
Protein
therapeutics
Autoimmune and
Inflammatory
diseases
8 Novel B7-
like
CGEN-25007 peptide Inflammatorydiseases (IBD)
gp96 (HSP)
CGEN-856 peptideFibrosis (Cardiac,
Renal),
Hypertension
Ang1-7/Mas
(RAS system)
CGEN-25009 peptidePulmonary
Fibrosis
LGR7
(RXFP1)
CGEN-855 peptide
Inflammatory
diseases (IBD) and
Cardiovascular
(I/R)
FPRL-1
d d l f
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GPCR Peptide Ligand Platform
Modeling the Peptidome: Endogenous Peptides
Signal
peptide
Signal
peptide
Prediction of
novel
cleavage sites
Proteome
Scoring system
ranking
possible
peptides
29
Novel GPCR Ligand PredictorIn silico
modeling and
scoring system
of GPCR ligands
Pi li P I l d Oth
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Pipeline Program: Immunology and Other
30
CGEN#Type of
Biologic
Therapeutic
IndicationPathway
Prediction
and
Selection
In vitro
validation
In vivo
POC
Lead
CandidatePre-IND
CGEN-15001Protein
therapeutic
Multiple Sclerosis,
Rheumatoid
Arthritis
Novel B7-like
Eight
undisclosed
Protein
therapeutics
Autoimmune and
Inflammatory
diseases
8 Novel B7-
like
CGEN-25007 peptide Inflammatorydiseases (IBD)
gp96 (HSP)
CGEN-856 peptideFibrosis (Cardiac,
Renal),
Hypertension
Ang1-7/Mas
(RAS system)
CGEN-25009 peptidePulmonary
Fibrosis
LGR7
(RXFP1)
CGEN-855 peptide
Inflammatory
diseases (IBD) and
Cardiovascular
(I/R)
FPRL-1
CGEN-25017 peptideOcular diseases
and Oncology
Angiopoeitin-
2/Tie2
pathway
Pi li P I l d Oth
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Pipeline Program: Immunology and Other
31
CGEN#Type of
Biologic
Therapeutic
IndicationPathway
Prediction
and
Selection
In vitro
validation
In vivo
POC
Lead
CandidatePre-IND
CGEN-15001Protein
therapeutic
Multiple Sclerosis,
Rheumatoid
Arthritis
Novel B7-like
Eight
undisclosed
Protein
therapeutics
Autoimmune and
Inflammatory
diseases
8 Novel B7-
like
CGEN-25007 peptide Inflammatorydiseases (IBD)
gp96 (HSP)
CGEN-856 peptideFibrosis (Cardiac,
Renal),
Hypertension
Ang1-7/Mas
(RAS system)
CGEN-25009 peptidePulmonary
Fibrosis
LGR7
(RXFP1)
CGEN-855 peptide
Inflammatory
diseases (IBD) and
Cardiovascular
(I/R)
FPRL-1
CGEN-25017 peptideOcular diseases
and Oncology
Angiopoeitin-
2/Tie2
pathway
Pi li P
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Pipeline Program
Initiated in 2010
Currently >30 product candidates in variousstages of validation
Continuing discovery runs
Market Driven Selection
Pathways, protein families and targets ofhigh industry interest
Moving selected candidates further indevelopment post animal disease model
Initially 12-18 months post animal diseasemodel to pre-IND stage
32
A d
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Agenda
Status of Pipeline Program
Why & HowA Quick Review Only One Key Need for the Industry
33
Ph I d t Sit ti i 2011
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Pharma Industry Situation in 2011
Still very profitable, but
Facing Blockbuster patent expirations
With limited late stage pipelines
Few clinical stage products available for licensing-in
Pricing pressures on Me-Too products with high
margins being a thing of the past
Cutting back on R&D expenses
34
O l O K N d f th I d t
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Only One Key Need for the Industry
Pharma Industry Situation in 2011
The Underlying Cause:
Long-term reliance largely on high through-put based
discovery methodologies
After finding the easy ones first it gets harder and
harder
35
The Missing Link: Systematic, constantlyimproving, discovery methodology has never
existed in the drug industry
A d
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Agenda
Status of Pipeline Program
Why & HowA Quick Review Only One Key Need for the Industry
Compugen Mission and Business Model
36
Mi i d B i M d l
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Mission:
To provide an increasing flow ofsuperior drug and
diagnostic product candidates to the industry through
leadership in science driven predictive discovery
Mission and Business Model
37
CGEN 928 f M lti l M l
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Dr. James R. Berenson (The Institute for Myeloma & Bone
Cancer Research):
These early results obtained with CGEN-928 are very encouraging,
especially given the fact that this protein is showing high levels of
expression in manymultiple myeloma tumors that are very drug
resistant and highly aggressive.The results we obtained with CGEN-928 in multiple myeloma
provide a basis for development of antibody-based therapy against
this target for MM, which shows promise both as a single therapy
as well as a combination therapywhich improves the activity of
other antimyeloma drugs. In addition, this novel target may be anew marker for this common blood-based malignancy especially for
the unmet medical need ofdetecting and treating cells that are
most aggressive and resistant to currently available drugs.
38
CGEN-928 for Multiple Myeloma
CGEN 15001 f Rh t id A th iti
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Dr. Richard Williams (Imperial College London UK):
Thesepreliminary results are very impressive with CGEN-15001
showing the same level of efficacy as Enbrel. Furthermore, by testing
CGEN-15001 in established disease we are setting the bar quite high, as
not many compounds are effective in this setting. Therefore, based onthe results seen to date, this molecule definitely should be further studied
as a potential therapy for multiple autoimmune diseases.
CGEN-15001 for Rheumatoid Arthritis
CGEN 15001 for Multiple Sclerosis
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Professor Stephen Miller (Northwestern University):
The capacity of CGEN-15001 toprevent the development of
disease in this well-recognized animal model for multiple sclerosis,
and more significantlyto ameliorate its progression when
administered in the presence of pre-existing disease is quite
dramatic.Furthermore, these beneficial effects were shown to be long
lasting and persisted through the study, indicating that CGEN-
15001 may prevent disease progression as efficiently as immune
tolerance induction, a process whereby the immune system no
longer attacks the self antigens that cause the disease.These findings, together with those demonstrated in our earlier
studies, are unique among the molecules targeting the B7 family
of co-stimulatory molecules that have been published to date.
40
CGEN-15001 for Multiple Sclerosis
CGEN 25017 for Angiogenesis Related Diseases
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Professor John S. Penn (Vanderbilt University School ofMedicine):
The efficacy achieved with CGEN- 25017 is afairly rare finding in
this model. Based upon our past experience conducting efficacy
trials of this type, CGEN-25017falls within the top 10% of all testcompounds that have passed through our hands. Thus, in my
opinion, CGEN-25017warrants further development and study
as a potential therapy for angiogenesis-related diseases."
41
CGEN-25017 for Angiogenesis-Related Diseases
CGEN 25007 for IBD
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Professor Markus F. Neurath (University of Erlangen,Germany):
The results achieved with CGEN- 25007 are very impressive. In the
past, we have evaluated numerous molecules in this model but
never saw such dramatic effects.If these results continue to be confirmed in further studies, this
molecule should represent a very exciting drug candidate for this
substantial, and largely unmet medical need."
42
CGEN-25007 for IBD
Mission and Business Model
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Mission:
To provide an increasing flow ofsuperior drug and
diagnostic product candidates to the industry through
leadership in science driven predictive discovery
Business Model:To maximize the number of our product candidates in
development pipelines of, or under co-development with
drug and diagnostic companies worldwide, pursuant to
milestone/royalty, or other revenue sharing agreements
Mission and Business Model
43
Product Candidates for the Industrys Future
Agenda
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Agenda
Status of Pipeline Program
Why & HowA Quick Review Only One Key Need for the Industry
Compugen Mission and Business Model Science Driven Predictive Discovery
44
Science Driven Predictive Discovery
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Science Driven Predictive Discovery
45
Synthesis &
Experimental
Validation
In Silico
MultipleProduct
Candidates
Unmet Medical
Need of Interest
Prediction of:Possible Candidates
Selection of:
Most Probable Candidates
The New Paradigm for Drug Discovery
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The New Paradigm for Drug Discovery
46
Synthesis &
Experimental
Validation
In Silico
MultipleProduct
Candidates
Unmet Medical
Need of Interest
Easy to say, very difficult to do.Many have tried in the past and failed
Prediction of:Possible Candidates
Selection of:
Most Probable Candidates
The Required Scientific Infrastructure
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The Required Scientific Infrastructure
47
For more than a decade, Compugens research teamfocused on deeper understandings
of key biological phenomena at the molecular level
The Required Scientific Infrastructure
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The Required Scientific Infrastructure
48
48
For more than a decade, Compugens research team
focused on deeper understandings,predictive modeling and integration of
of key biological phenomena at the molecular level
Science Driven Predictive Discovery
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Science Driven Predictive Discovery
49
49
UnmetMedical Need
of Interest
MultipleProduct
Candidates
Prediction of:
Possible Candidates
Selection of:
Most Probable Candidates
Synthesis &Experimental
Validation
The Proof is In the Pudding
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The Proof is In the Pudding
0
5
10
15
20
25
30
CumulativeProductCandidates
Product Candidates
demonstrating positivedisease animal model*
Increasing discovery rate over time
50
* or equivalent experimental validation
50
Agenda
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Agenda
Status of Pipeline Program
Why & How A Quick Review Only One Key Need for the Industry
Compugen Mission and Business Model
Science Driven Predictive Discovery
The Obvious Advantages
51
Advantages of Compugen Approach
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Largely random Hypothesis Driven andSystematic
Advantages of Compugen Approach
ExperimentalHit or Miss
The CompugenApproach
52
Advantages of Compugen Approach
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Largely random
Learn very little from
failures
Hypothesis Driven andSystematic
Gain knowledge from
successes and failures
Advantages of Compugen Approach
ExperimentalHit or Miss
The CompugenApproach
53
Advantages of Compugen Approach
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Largely random
Learn very little from
failures
More difficult overtime
Innovative, HypothesisDriven and Systematic
Gain knowledge from
successes and failures
Easier with each discoveryround
Advantages of Compugen Approach
ExperimentalHit or Miss
The CompugenApproach
54
Systematic and Continuously Improving
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Systematic and Continuously Improving
New
Product
Candidates
Model and
Algorithm
Improvement
55
Synthesis &Experimental
Validation
Data
Prediction of:
Possible CandidatesSelection of:
Most Probable Candidates
UnmetMedical Need
of interest
Agenda
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Agenda
Status of Pipeline Program
Why & HowA Quick Review Only One Key Need for the Industry
Compugen Mission and Business Model
Science Driven Predictive Discovery
A New Paradigm for Drug Discovery
56
A New Paradigm in Drug Discovery
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A New Paradigm in Drug Discovery
Systematic, constantly improving
Provides flexibility in selecting unmet medical
needs and targets
Deals with biological complexity
Allows powerful Integration of different discoveryapproaches and platforms
Results in novel product candidates
First-in-Class or Best-in-Class
57
Potentially better drugs, higher success rate, strong IPCombined with a low-risk, high reward, business model
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Financial Status and Outlook
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Financial Status and Outlook
Projected 2011 Cash Expenditures = ~$10 million*
* Net Burn for year would be reduced by any revenues received
59
Extremely Cost Efficient
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Extremely Cost Efficient
New Platforms:
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Projected 2011 Cash Expenditures = ~$10 million*
Available Resources at 03/31/11: >$30 million**
Market Cap: ~$160 million
* Net Burn for year would be reduced by any revenues received
** Includes ~$25.5 million in cash and ~$5.5 million in EVGN (Evogene Ltd) shares
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Financial Status and Outlook
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Projected 2011 Cash Expenditures = ~$10 million
Available Resources at 03/31/11: >$30 million
Market Cap: ~$160 million
Outlook:
Expenditures: Largely dependent on Pipeline Programproducts and arrangements
Revenues: Potential sources of short term revenues in
the amounts required for cash breakeven include
upfront fees from licenses, research revenues, andpayments related to strategic-type arrangements.
Possible timing for such revenues based on current
expectations and discussions is later this year or first half
2012
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Agenda
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g
Welcome Update Presentations:
Status of Pipeline Program
Why & HowA Quick Review
Financial Status and Outlook
Questions and Answers Session
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Compugen Participants: Martin Gerstel Chairman Anat Cohen-Dayag, PhD President & CEO Dikla Czaczkes Axselbrad CFO
[email protected] or www.cgen.com
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Compugen Ltd
Establishing a New Paradigm for
Drug and Diagnostic Discovery
1st Quarter 2011 Conference CallMay 11, 2011