Realizing the Promise of Predictive Drug Discovery

Transcription

1 Realizing the Promise of Predictive Drug Discovery Dikla Czaczkes Axselbrad, CFO TASE & NASDAQ 5 th Annual Investor Conference, New York May 17, 2012

2 Safe Harbor Statement This presentation contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of These statements include words like "may," "expects," "believes," anticipates 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 on form 20-F that are filed with the Securities and Exchange Commission. Compugen is presenting this information as of the date of the presentation and expressly disclaims any duty to update the information contained in this presentation. This presentation contains information from third-party sources, including data from studies conducted by others and market data and industry forecasts obtained from industry publications. Although Compugen believes that such information is reliable, we have not independently verified any of this information and we do not guarantee the accuracy or completeness of this information. 2

3 Mission, Area of Focus and Business Model Mission Utilize Compugen s leadership in science driven predictive discovery to provide pharma with superior product candidates on a continuing and systematic basis Area of focus Protein and antibody therapeutics in the fields of oncology and immunology Business Model Maximize the number of Compugen-discovered product candidates in the development pipelines of leading pharmaceutical and biotech companies worldwide, pursuant to milestone/royalty or other revenue sharing arrangements 3

4 The Promise of Predictive Drug Discovery Discovery by Observation Discovery by Prediction & Selection History of Drug Discovery Relying upon observations of experimental results Easy discoveries made first, then gets more and more difficult Compugen Utilizing in silico models based on proprietary understandings of key biological phenomena to predict novel molecules and their functions in disease modulation Continuous improvement of in silico models with each discovery attempt whether successful or not 4

5 Utilizing the Integrated Infrastructure Protein databases Sequences EST s LEADS Next Generation Sequence Data MED Expression data Drug Response RefSeq and mrnas Drug data Non human databases Protein interaction data GeneDex Engine & Unified Resource DISCOVERY PROGRAM Gene mutation data Gene ontology Pathway data Transcription Factors Specialized Algorithms and Queries Disease specific, protein family, pathway analysis, protein prediction and annotation, splice variants, protein-protein interactions, drug response and more Novel Therapeutic Proteins & Drug Targets 5

6 Compugen s Predictive Discovery: Key Advantages Superior product candidates First-in class, novel pathways Strong and broad IP for discoveries Speed of discovery process New candidate predictions in months Broad applicability Various therapeutic and diagnostic applications High entry barrier for technology Years to establish competitive capabilities 6

7 Compugen s Focus Protein therapeutics and mab therapeutics Higher development success rate and reduced toxicity compared with small molecule drugs Global market is substantial, with highest growth rates in drug industry Oncology and Immunology Unmet medical needs of high interest to pharma Compugen s predictive discovery is well suited to disease complexity Commercial opportunities for early stage discoveries Early stage compounds dominated cancer and autoimmune & inflammatory licensing* In 2011, almost 50% of therapeutic alliances with over $10M upfront, were signed around preclinical stage assets** 7

8 Compugen s Pipeline Program Initiated in 2010 Oncology and immunology focus Based on pathways, protein families and targets of high industry interest ~30 early stage product candidates Continuing discovery runs Moving selected candidates further in development from POC in animal disease model to pre-ind stage 8

9 Pipeline Program Highlights: Protein Therapeutics Product Candidate In Silico Prediction & Selection In Vitro Validation In Vivo Proof of Concept Lead Candidate Pre-IND CGEN CGEN CGEN CGEN CGEN Protein Therapeutic (novel B7-like) Autoimmune diseases (*) (**) Inflammatory diseases Protein Therapeutic (novel B7-like) Autoimmune diseases (*) (**) Inflammatory diseases Protein Therapeutic (novel B7-like) Autoimmune diseases (*) Inflammatory diseases Protein Therapeutic (novel B7-like) Autoimmune diseases (*) Inflammatory diseases Protein Therapeutic (novel B7-like) Autoimmune diseases (**) Inflammatory diseases Four Undisclosed Protein Therapeutics (immunomodulatory) Autoimmune diseases Inflammatory diseases 9

10 Example: Applying our Capabilities Discovery of Novel Protein Family Members Prediction of all human proteins Prediction of proteins with family characteristics Prediction of proteins expressed on diseased cells In Silico Prediction and Selection Further refined prioritization criteria Discovery of multiple novel protein family members, e.g., CGEN-15001, for autoimmune diseases Experimental validation 10

11 CGEN a Novel Immunomodulatory B7-like Therapeutic Protein CGEN is a first-in-class therapeutic agent targeting a negative costimulatory pathway with a potential for better safety and efficacy compared to standard-of-care drugs of autoimmune diseases Long term amelioration of disease symptoms following short administration in EAE model, indicating potential for induction of immune tolerance (model of multiple sclerosis) An immunomodulator restoring the immune Th1/Th2 balance Potent reduction in clinical signs in CIA model (model of rheumatoid arthritis) Th1 Th17 Th2 Th2 11

12 Pipeline Program Highlights: Monoclonal Antibody Therapeutics Product Candidate In Silico Prediction & Selection Target Characterization Target Validation (IHC) mab and In Vivo POC Lead Candidate & Pre-IND CGEN-928 mab Target (uncharacterized gene) Multiple myeloma CGEN-15001T CGEN CGEN Six Undisclosed mab Target (novel B7-like) Solid & hematological cancers Immunotherapy mab Targets Epithelial cancers immunotherapy mab Targets Epithelial cancers immunotherapy mab Targets Epithelial cancers Immunotherapy 12

13 2012: Incorporate Development Capabilities Therapeutic proteins Step-wise internal growth initiated in late 2010 mab therapeutics: wholly-owned Compugen Inc South San Francisco in Q Develop monoclonal antibodies against targets selected from Compugen s Pipeline Program Headed by a team with extensive expertise A world leading expert as the Executive Chairperson: Dr. Mary Haak-Frendscho, former CEO of Takeda SF The combination of predictive novel target discovery and therapeutic mab development expertise positions Compugen to become a potential world leader in mab therapeutics 13

14 Mission, Area of Focus and Business Model Mission Utilize Compugen s leadership in science driven predictive discovery to provide pharma with superior product candidates on a continuing and systematic basis Area of focus Protein and antibody therapeutics in the fields of oncology and immunology Business Model Maximize the number of Compugen-discovered product candidates in the development pipelines of leading pharmaceutical and biotech companies worldwide, pursuant to milestone/royalty or other revenue sharing arrangements 14

15 Pathways for Commercialization Establishing the Required Infrastructure Scientists, Science, Predictive Models Research & Discovery Collaborations Validation - Initial Predictive Models Technology Driven discoveries 3 rd Party Funded Move to Market Driven Discovery Unmet Needs Pipeline Program Collaborations & Licenses

16 Platform Validation Stage Discoveries in 2011 Arrangements are being made to advance certain of our platform validation stage discoveries without the need for further Compugen financial resources, but with sharing in any future revenues BiolineRx for several peptide drug candidates DiscoveRx for ~900 peptides designed to potentially serve as GPCR modulating molecules Pulmonary Fibrosis Foundation for pulmonary fibrosis therapeutic peptide 16

17 Financial Status Available Resources as of 03/31/12: ~$36 million* Anticipated Cash Uses for the remainder of 2012: $13 million Market Cap: ~$130 million NASDAQ (CGEN) TASE (CGEN.TA) * Includes $25 million 3/31/12 ending cash, adjusted for remaining $6 million from Baize II Agreement signed in Q4/2011 and ~$5 million market value of Evogene Ltd (TASE: EVGN.TA) shares at the end of Q

18 In Closing - A Quick Overview World leader in the paradigm shift from drug discovery by observation to drug discovery by prediction: q Novel discoveries offering competitive landscape q Increased success rates with significantly decreased costs and time 1997 and beyond - Establishing proprietary and continuously improving science based & broadly applicable predictive discovery infrastructure Scientific understandings, predictive technology, expert scientists 2010 and beyond - Focus on mab and protein therapeutics in immunology and oncology Initiated Pipeline Program with ~30 product candidates 2011 and beyond - As Pipeline product candidates advance, a series of agreements for advancing non-focus area discoveries made during platform validation 2012 and beyond - Realizing the promise of predictive discovery Anticipated collaborations, added development capabilities, new platforms, new product candidates, advanced pipeline candidates 18

19 Thank You Realizing the Promise of Predictive Drug Discovery