Download predictive discovery of first-in-class biologics

PREDICTIVE DISCOVERY OF FIRST-IN-CLASS BIOLOGICS
Primary focus on immuno-oncology
Corporate Overview
www.cgen.com
Ari Krashin
Chief Financial and Operating Officer
www.cgen.com
Jefferies
June 2016
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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. Forward-looking statements can be identified by the use of terminology such as “will,”
“may,” “expects,” “anticipates,” “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. Among these risks:
Compugen’s business model is substantially dependent on entering into collaboration agreements with third
parties and may not be successful in generating revenues, and the development and commercialization of
therapeutic products include many inherent risks, including failure to progress to clinical trials or, if they progress
to or enter clinical trials, failure to receive regulatory approval. These and other factors are more fully discussed
in the "Risk Factors" section of Compugen’s most recent Annual Report on Form 20-F as filed with the Securities
and Exchange Commission as well as other documents that may be subsequently filed by Compugen from time to
time with the Securities and Exchange Commission. In addition, any forward-looking statements represent
Compugen’s views only as of the date of this presentation and should not be relied upon as representing its views
as of any subsequent date. Compugen does not assume any obligation to update any forward-looking statements
unless required by law. Certain studies and data presented herein have been conducted for us by other entities as
indicated where relevant.
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Company Overview
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Leader in Predictive Drug Discovery
Mission
Discover high value novel targets and develop first-in-class
biologics for unmet medical needs
Business Model
commercialization at various stages, revenue sharing agreements
Broad Early Stage Pipeline - Next Wave of Immuno-Oncology
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Internal discoveries
High priority internal programs
Two programs partnered with Bayer HealthCare
Additional targets for antibody-drug conjugate (ADC) therapy and one
program for autoimmune diseases
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Company Fact Sheet
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Strong Intellectual Property Landscape
 Proprietary algorithms and computational biology platforms
 61 issued and allowed patents, 137 pending patent applications, in all
major countries
Experienced Multidisciplinary Teams
 ~95 employees, 70% R&D
 Algorithm development, computer sciences, biology and drug development
Financial Strength (NASDAQ: CGEN; TASE: CGEN.TA)
 Cash balance ~$81 million (March 31, 2016)
 No debt
Partners
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Bayer HealthCare, mAb drugs targeting two novel immune checkpoints
Merck-Serono, JV for biomarker discovery
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Two Centers of Excellence
Compugen Ltd.
Compugen USA, Inc.
Holon, Israel
South San Francisco, USA
Headquarters
Wholly-Owned Subsidiary
Predictive Target Discovery
& Experimental Validation
Therapeutic mAb
Research & Development
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Scientific Advisory Board
Renowned Oncologists and Immunologists
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Drew Pardoll, MD, PhD (Chairman of the SAB; Director - Compugen USA, Inc.)
Professor of Oncology, Medicine, Pathology, and Molecular Biology and Genetics and
Director of the Bloomberg~Kimmel Institute for Cancer Immunotherapy at the Sidney
Kimmel Cancer Center at Johns Hopkins University of Medicine
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Charles G. Drake, MD, PhD
Professor of Oncology, Immunology and Urology at Johns Hopkins School of Medicine,
the Sidney Kimmel Comprehensive Cancer Center
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Iain B. McInnes, MD, PhD
Director of the Institute of Infection, Immunity, and Inflammation at the University of
Glasgow
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Antoni Ribas, MD, PhD
Professor at the Department of Medicine, Hematology/Oncology, University of
California, Los Angeles
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Howard R. Soule, PhD
Executive Vice President and Chief Science Officer, Prostate Cancer Foundation
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Pipeline Program Focus Areas
Multiple Novel Target Candidates for First-in-Class Opportunities
Oncology
Novel
immune checkpoint
target candidates
Novel
Antibody Drug Conjugate
target candidates
Immunology
One program for autoimmune diseases based on a novel immune checkpoint target
Diverse portfolio based on Compugen’s predictive discovery
capabilities
B7/CD28 Immune Checkpoints for Cancer
Immunotherapy
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Harnessing the immune system to
destroy cancer cells
Breakthrough approach to cancer
therapy
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Impressive, long-lasting clinical
benefits
Still, the majority of cancer patients
do not respond
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New checkpoints, drug combinations and
drug modalities required
Checkpoint-based drugs expected to become
the largest drug class in history
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Targeting of Immune Checkpoints Today
Crowded Competition on Same Targets
PI
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PIII
PIII
PII
PI
PC
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M
PC
PC
PC
M
PII
PC
PI
PC
PI
PI
M
PI
PI
PII
PC
PI
PC
PI
PC
PC
PI
PC
PC
PC
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PC
PC
PC
From the Genome to the Clinic
An Integrated Approach
PREDICTIVE
PREDICTIVE
DISCOVERY
DISCOVERY
PIPELINE
PROGRAM
STRATEGIC
PARTNERSHIPS
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Pharma’s Observational Path to Novel Targets
COLLECTION OF
KNOWN
GENES / PROTEINS
DISEASE
SAMPLES
EXPERIMENTAL
CHARACTERIZATION
DATA ANALYSIS
NOVEL
TARGETS
FIRST-IN-CLASS
Known proteins/genes, disease-specific, assay systems limitation
No learning
Immune Checkpoint Platform
Genomic Detection Leading to Diverse Set of Novel Candidates
Diversified portfolio of immuno-oncology targets
CGEN candidate
Shown for illustrative purpose only
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Applying Our Predictive Approach
First Focused Discovery Program: Novel Immune Checkpoint Target
CGEN-15001T
CGEN-15052
CGEN-15022
CGEN-15092
CGEN-15049
CGEN - 15029
Undisclosed Targets
Partial data sources shown for illustrative purposes
B7 refers to B7/CD28
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From the Genome to the Clinic
An Integrated Approach
PREDICTIVE
DISCOVERY
PIPELINE
PIPELINE
PROGRAM
PROGRAM
STRATEGIC
PARTNERSHIPS
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Pipeline Program
Novel Immune Checkpoint Target Candidates
CGEN-15029
Lead drug program
Shown for illustrative purpose only
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CGEN-15029 mRNA Is Expressed in Many Cancers
Expression in multiple cancer indications
Elevated expression
in kidney cancers
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CGEN-15029 Expression Pattern Associated With Tumor
Infiltrating Immune Cells (TILs)
RNA correlation with CD8 T cells and PD-1
(Kidney Clear Cell Cancer)
CD8 vs 15029
Confirmed TIL protein expression
(Melanoma)
PD-1 vs 15029
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RNA Expression of CGEN-15029 Highly Correlated With
PD-1, TIM-3 and TIGIT in Solid Tumors
All tumors (TCGA)
Melanoma (TCGA)
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CGEN-15029 Inhibits T Cell Cytotoxic Activity
Inhibition of Cytotoxic
T Cell Activation
Cohen and Safion, Bar Ilan Univ.
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CGEN-15029: Binding Partner Identified
Binding of hCGEN-15139-Fc to HEK293
cells over expressing hCGEN-15029
Binding of ligand (CGEN-15139) to receptor (CGEN-15029) was confirmed by SPR, ELISA and FACS
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CGEN-15029
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Highest Priority mAb Program
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High-affinity blocking antibodies have been identified
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Ongoing functional screening of blocking mAbs to select clinical
lead candidate
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IND anticipated in 2017
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Pipeline Program
Novel Myeloid Expressed Immune Checkpoint Target Candidates
CGEN-15029
CGEN candidate
Shown for illustrative purposes only
Immune checkpoints have relevance for other multiple mechanism
not only on T-cells
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CGEN Myeloid Candidates
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Myeloid cells are important components of the tumor
microenvironment
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Very few therapeutic targets
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Promising emerging area of tumor biology
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Included in CGEN’s high priority target programs
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May provide new treatment options for patients refractory to
existing immune checkpoint inhibitors (mono and combination
therapy)
Ongoing verification of MoA and direct T cell inhibition
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CGEN Myeloid Candidate
CD68 Expression
Induction in Cancer Correlated with Myeloid Specific Markers
CGEN Candidate Expression
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Cell Surface Tumor Antigen Targets
Second Focused Discovery Program: Antibody Drug Conjugate – ADC Targets
The goal: identify membrane proteins over-expressed on cancer but with
little or no expression on normal tissues
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Example: Expression Profile Analysis
High Cancer Expression, Low Toxicity, Low Sink Potential
Predictive discovery driven analysis
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CGEN-15027: A Novel ADC Target
High Expression in Ovarian and Lung Cancers
Normal Ovary
Normal Lung
Ovarian Cancer
(Endometroid adenocar.)
Lung Cancer
(Adenocarcinoma.)
Ovarian Cancer
(Mucinous cystadenocar.)
Lung Cancer
(Adenocarcinoma)
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From the Genome to the Clinic
An Integrated Approach
PREDICTIVE
DISCOVERY
PIPELINE
PROGRAM
STRATEGIC
STRATEGIC
PARTNERSHIP
PARTNERSHIPS
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Targets for First-in-Class Opportunities
BayerNovel
Partnership
Strategic
Collaboration
Cancer Immunotherapies
The
Next WavetoinDevelop
Immuno-Oncology
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Two novel immune checkpoint CGEN-15001T and CGEN-15022
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Joint preclinical research program
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Bayer assumes full control over subsequent development and commercialization
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Terms include:
 $10M upfront payment
 Preclinical milestone payments of up to $30M
 15001T - To date achieved three milestones totaling $15M
 15022 - To date achieved first milestones $0.4M
 Eligible to receive over $500M in potential milestone payments
 Eligible to receive mid-to-high single digit royalties on global net sales
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Targets for First-in-Class
Opportunities
BayerNovel
Partnership:
Current Status
Next Targets
for Anti-Tumor
Immunity
The Next
Wave in Immuno-Oncology
• CGEN-15001T and CGEN-15022: Two novel immune checkpoints
• Each presents a distinctive differentiation profile compared to known
checkpoints
• Targets are expressed on numerous types of solid and hematological cancer
as well as on immune cells residing in both tumor and normal tissues
• CGEN-15001T antibody program:
• Recently transferred to the full control of Bayer (achievement 3rd milestone)
• From computer prediction to functional activity in preclinical models
• CGEN-15022 antibody program:
• Novel mechanism of action has been revealed
• Further characterization of its role in anti-cancer immune responses
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Collaboration with Johns Hopkins School of Medicine
Multi-Year Immuno-Oncology Collaboration
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Focused on the Company’s novel immune checkpoint target
candidates
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Led by Johns Hopkins Professors Drew Pardoll and Charles Drake
Supports and accelerates translation to the clinic
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Target characterization and differentiation
Evaluation of therapeutic potential as monotherapy and combination
therapy
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From the Genome to the Clinic
Drivers and Anticipated Milestones
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hPOC in clinical trials: Partnered and un-partnered programs
 First IND anticipated in 2017 for CGEN-15029
 Continuing progress on Bayer’s programs
 Multiple programs in various stages of target validation or antibody
discovery
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Product-oriented collaborations
 Potential collaborations at various development stages
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Development milestones in collaborations
 Potential milestones under Bayer collaboration
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Financial Status
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Strong Financial position
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Gross Cash Expenditures*
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Cash balance ~$81 million (March 31, 2016)
No Debt
Forecast 2016 ~$31-$33 million
Market Capitalization
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~$350 million (May 2016)
NASDAQ (CGEN); Indices – NBI (Nasdaq Biotech Index)
TASE (CGEN.TA); Indices- TA-75, TA-Biomed, TA
BlueTech, TA Tech-Elite
* Does not include any anticipated cash receipts
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Thank You!
Ari Krashin
Chief Financial and Operating Officer
www.cgen.com
June 2016
www.cgen.com