Joseph Wagner, Ph.D. Chief Executive Officer OncoCyte Corporation

Transcription

1 Joseph Wagner, Ph.D. Chief Executive Officer OncoCyte Corporation

2 Forward Looking Statements The matters discussed in this presentation include forward looking statements which are subject to various risks, uncertainties and other factors that could cause actual results to differ materially from the results anticipated. Such risks and uncertainties include but are not limited to the success of BioTime and its subsidiaries in developing new stem cell products and technologies; results of clinical trials of BioTime and subsidiary products; the ability of BioTime, its subsidiaries and its licensees to obtain additional FDA and foreign regulatory approval to market BioTime and subsidiary products; competition from products manufactured and sold or being developed by other companies; the price of and demand for BioTime and subsidiary products; and the ability of BioTime and subsidiaries to raise the capital needed to finance its current and planned operations. Any statements that are not historical fact (including, but not limited to statements that contain words such as "will," "believes," "plans," "anticipates," "expects," "estimates") should also be considered to be forward-looking statements. Forward-looking statements involve risks and uncertainties, including, without limitation, risks inherent in the development and/or commercialization of potential products, uncertainty in the results of clinical trials or regulatory approvals, need and ability to obtain future capital, and maintenance of intellectual property rights. As actual results may differ materially from the results anticipated in these forwardlooking statements they should be evaluated together with the many uncertainties that affect the business of BioTime and its subsidiaries, particularly those mentioned in the cautionary statements found in BioTime's Securities and Exchange Commission filings. BioTime disclaims any intent or obligation to update these forward-looking statements. 2

3 OncoCyte Corporation Developing products combining bioinformatics and embryonic stem technology for the diagnosis and treatment of cancer Focus on Molecular Diagnostics and Therapeutics Recent $10 Million Investment (3rd round) Cancer Therapeutics - Cellular therapeutics for cancer treatment - Leverage unique biology of vascular endothelial precursor cells Molecular Diagnostics - Developing products for earlier detection and more effective treatment of numerous cancers - Microarray-based cancer diagnostic target discovery platform - Near-term revenue potential and partnering opportunities 3

4 OncoCyte Corporation- Therapeutics Focus on vascular endothelial cells engineered to deliver toxic payloads to developing blood vessels of tumors Goal: - Develop a new class of cellular therapeutics as an entry point for the delivery of regulated tumoricidal agents Current development: - Pre-clinical stage - Consistent manufacturing process yielding large numbers of vascular progenitor cells derived from multiple hes cell lines - Acquired enhanced delivery technology developed by Cell Targeting, Inc. 4

5 OncoCyte Corporation- Diagnostics Opportunity that leverages the BioTime bioinformatics program and creates a cancer marker discovery platform Established a proprietary dataset using RNA microarray technology Identified numerous genes abnormally activated in cancer or tumor cells Normal Cancers Many of these genes have not been previously associated with cancer Multiple product opportunities possible from broad discovery platform 5

6 $ Millions Proteomic Epigenomic Cellular Total , Cancer Diagnostic Markets Cancer Source: BCC diagnostic Research markets are expected to grow to over $5 billion by 2015 and will be dominated by molecular and proteomic tests SUMMARY FIGURE GLOBAL VALUE OF NEXT GENERATION CANCER DIAGNOSTICS BY ANALYSIS TYPE, ($ MILLIONS) 4,000 3,000 2,000 1, Genomic Proteomic Epigenomic Cellular Source: BCC Research 6

7 Cancer Diagnostic Markets Molecular and proteomic-based screening and companion diagnostics/prognostics will dominate the market Pre-disposition Testing: Determines risk factor in currently healthy patients Largest market, significant payer reluctance Screening: Early detection in asymptomatic patients Large market, substantial economic impact potential Companion Diagnostic/Prognostic: Test for outcomes to guide treatment path Smaller market, current area of most product development Monitoring Detects tumor progression, treatment response and recurrence Smallest market, lowest barriers to entry 7

8 Cancer Diagnostic Markets Screening diagnostics that overcome current limitations will have massive public health impact and market potential Early detection saves lives: 5-year survival highest when treated at early stages Late stage and metastatic disease patients fare poorly Large scale screening of asymptomatic patients COULD save lives and reduce overall cost burden of cancer treatment Current screening tests have limitations: Simple tests are inexpensive but can be unreliable Reliable tests tend to be invasive, expensive, unpleasant 8

9 Key Drivers: Voice of Customer Physician adoption and payer reimbursement are key barriers to entry for cancer screening diagnostics Physician Adoption: Test performance (specificity and sensitivity) Ease of interpretation Unaddressed cancers easier sell Reimbursement: Test performance (positive predictive value) Low cost Tests for currently addressed cancers must provide additional benefit(s) 9

10 Key Drivers: Test Performance Simple, low cost cancer screens that demonstrate superior performance would be used and paid for High Sensitivity: Ability to detect cancer at every stage in every patient High Specificity: Minimal false positives High Positive Predictive Value: Positive test result is accurate Solution: Detect multiple cancer-related markers 10

11 Key Drivers: Other Product Characteristics Simple, low cost cancer screens that demonstrate superior performance would be used and paid for Non-invasive: Ease of use, increased safety Blood-based ideal Ease of interpretation: Uses currently accepted methods and technologies Clear results: High risk vs. Low risk Low cost Model after current low cost screens Antibody-based protein detection, PCR-based gene expression two viable options 11

12 OncoCyte Corporation- PanC-Dx TM Evaluating and validating specific diagnostic products based on unmet medical need, large market size, low-cost and ease of use PanC-Dx TM Designed to detect pan-cancer as well as tumorspecific markers with superior sensitivity, specificity and positive predictive value Would require only a simple, easily interpreted, low-cost antibody-based blood test used as a complement to and/or replacement for current screens Positive test would identify patients at risk, initiate imaging, biopsy, pathology 12

13 - PanC-Dx TM : Breast Cancer Screen Evaluating and validating specific diagnostic products based on unmet medical need, large market size, low-cost and ease of use Current screening test: Screening mammogram every 2 yrs. in women Specificity = 85-93%, Sensitivity = 70-90% Poor performance in women < 45 (self-exam used) Market: million screening mammograms per year in US Medicare reimbursement rate = $143 Product Opportunity: Blood-based test, desired specificity & sensitivity >95% No radiation exposure or need for additional appointment Can be performed at any frequency in patients of any ag 13

14 - PanC-Dx TM : Colon Cancer Screen Evaluating and validating specific diagnostic products based on unmet medical need, large market size, low-cost and ease of use Current screening test: Colonoscopy, FOBT 33% reduction in deaths, 20% reduction in incidence Underutilized Small but real risk for severe complications Market: 6-7million screening colonoscopies per year in US Medicare reimbursement rate = ~$1000 Product Opportunity: High performance, blood-based test for early stage cancers AND adenomas (polyps) No procedure risks Low comparative cost 14

15 - PanC-Dx TM : Early Results Individual pan-cancer marker proteins are individually elevated in a subpopulation; multiplexing enhances performance 15

16 Bladder Cancer Recurrence Diagnostic Evaluating and validating specific diagnostic products based on unmet medical need, large market size, low-cost and ease of use Current test: Urine-based cytology Positive cytology requires cystoscopy for confirmation Poor performance, sensitivity <50% Market: ~400,000 patients currently monitored in US Medicare reimbursement rate = ~$60 Product Opportunity: PCR-based test using shed cells isolated from urine Superior performance Competitive cost Positive result requires cystoscopy as follow up 16

17 Diagnostic Product Development Strategy Evaluating and validating specific diagnostic products based on unmet medical need, large market size, low-cost and ease of use Focused platform Proprietary microarray dataset Pan-cancer and tumor-specific markers Targets with therapeutic potential may arise Evaluate multiple products PanC-Dx blood-based screening diagnostics for breast, colorectal cancers Other opportunities in bladder, thyroid, pancreatic cancers Strategically develop Build and validate diagnostic panels and products Aggressively build patent estate Initiate dialogue with potential partners early 17

18 PanC-Dx Cancer Diagnostic Regulatory Strategy CE Mark (EU) then CDRH/OIVD (U.S.) Near-term opportunity, low cost for approval PanC-Dx Timeline Goal is to launch PanC-Dx in 2014 Marker Validation Antibody Screening Antibody Production Final Kit Developed Patient Study Completion CE Mark Q3 Q4 Q1 Q2 Q3 Q4 Q1 Q2 Q3 Q4 Q1 Q2 Q3 Q

19 Karen B. Chapman, Ph.D. Director of Bioinformatics OncoCyte Corporation

20 PanC-Dx Cancer Diagnostic Tumor Biomarkers Diagnostic Products Discover Tumor Biomarker Discovery Screen Serum Screening Identify Identify best performing biomarkers Produce Monoclonal Antibody Production Define Define Multiplex Panel Patient study in clinical setting

21 Tumor Biomarker Discovery Platform Proprietary microarray dataset encompasses many tumor types. Microarray Analysis Normal Cells Normal Tissues Tumors Tumor Cell Lines 47,323 Probes Novel Probes RNA sequencing Sensitivity Broad Context Multiple Tumor Types Proprietary Microarray Dataset > 530 samples 21

22 Biomarker Discovery Pan-Cancer biomarker discovery enabled by broad composition of proprietary microarray dataset. Invasiveness Vascularization Normal Cancers Metastatic Immortal 22

23 Biomarker Discovery Broad context of biomarker discovery platform yields high quality targets. Proliferation Biomarker Cells Tissues Tumors Cell Lines Pan-Cancer Biomarker Cells Tissues Tumors Cell Lines 23

24 Biomarker Discovery Pan-Cancer biomarkers represent unique targets for molecular diagnostics and therapeutics. Normal Cells Normal Tissues Tumors Tumor Cell Lines 24

25 Pan-Cancer Biomarker CD31 Marker D DAPI Breast Tumor Normal Breast 25

26 Biomarker Discovery Normal Cells Normal Tissues Tumors Tumor Cell Lines Breast Tumors 26

27 Serum Screening Biomarker Discovery Serum Screening Monoclonal Antibody Production Diagnostic Product Marker A: Preliminary Serum Screening Normal Donors Breast Cancer Patients 27

28 Serum Screening via ELISA Tumor Biomarkers Diagnostic Products Detection of Biomarker F Protein in Patient Serum cutoff Normal Donors Colon Cancer Breast Cancer 28

29 Multiplex Biomarker Panel Preliminary data compilation shows discrimination between breast cancer patient serum and normal donor serum. Normal Donors Breast Cancer Patients 29

30 Breast Cancer Diagnostic Multiplex assay yields enhanced sensitivity and specificity. Highlighted markers yield 83% sensitivity, 97% specificity in preliminary 50 sample set Mammogram yields 70-90% sensitivity, 85-93% specificity Top Markers are in monoclonal antibody production Data for top markers anticipated within 2 6 months 30

31 Diagnostic Assay Development Discover Tumor Biomarker Discovery >700 biomarkers identified 20 cancer types >20 patent applications filed Screen Serum Screening >60 markers screened Identify Identify best performing biomarkers Top candidates identified for pan-cancer, breast cancer and colon cancer Produce Monoclonal Antibody Production Top 7 markers in monoclonal antibody production Define Define Multiplex Panel 31