Epigenomics AG March 2015 www.epigenomics.com
Safe Harbor Forward Looking Statements This communication contains certain forward-looking statements, including, without limitation, statements containing the words expects, future, potential and words of similar import. Such forward-looking statements involve known and unknown risks, uncertainties and other factors, which may cause our actual results of operations, financial condition, performance, or achievements, or industry results, to be materially different from any future results, performance or achievements expressed or implied by such forward-looking statements. Such factors include, among others, the following: uncertainties related to results of our clinical trials, the uncertainty of regulatory approval and commercial uncertainty, reimbursement and drug price uncertainty, the absence of sales and marketing experience and limited manufacturing capabilities, attraction and retention of technologically skilled employees, dependence on licenses, patents and proprietary technology, dependence upon collaborators, future capital needs and the uncertainty of additional funding, risks of product liability and limitations of insurance, limitations of supplies, competition from other biopharmaceutical, chemical and pharmaceutical companies, environmental, health and safety matters, availability of licensing arrangements, currency fluctuations, adverse changes in governmental rules and fiscal policies, civil unrest, acts of God, acts of war, and other factors referenced in this communication. Given these uncertainties, prospective investors and partners are cautioned not to place undue reliance on such forward-looking statements. We disclaim any obligation to update any such forward-looking statements to reflect future events or developments. Legal Product Disclaimer Products by Epigenomics that are referred to in this presentation, especially Epi procolon, are not available and are not approved for sale in the United States. The analytical and performance characteristics of any product to be eventually sold in the U.S. based on our technology have not been established. 2 March 2015
Overview Epi procolon Unmet need & commercial opportunity for Epi procolon Commercial strategy Epi prolung and future product opportunities Financial Information and Summary Appendix 3 March 2015
DNA Methylation Diagnostic Products for Oncology Epi procolon blood-based colorectal cancer (CRC) screening 1,2 Epi prolung tissue assay for lung cancer diagnosis 1 Addressing low compliance to currently available CRC screening Aid in difficult to diagnose lung cancer Potential to be developed into blood based assay 1 CE marked and commercially available in Europe 2 under FDA review and not commercially available in the U.S. 4 March 2015
Overview Epi procolon Unmet need & commercial opportunity for Epi procolon Commercial strategy Epi prolung and future product opportunities Financial information and summary Appendix 5 March 2015
Fighting Colorectal Cancer Public Health > 136,000 new cases 50,000 deaths 1 90% 5-year survival rate in stages I and II 60% diagnosed in later stages Health Economic US$ 14 Bn CRC treatment cost p.a. Over a third in late stage disease Screening is key! Recommendation 2 : colonoscopy every 10y or annual FIT 25-30 million people not screened for CRC ACS 3 : campaign to get 80% screened by 2018 1 ACS Cancer Facts & Figures 2014, 2 recommendation of the U.S. Preventive Services Task Force (USPSTF) for people aged between 50-75 years, 3 American Cancer Society (ACS) 6 March 2015
CRC Screening with Epi procolon 7 March 2015
Blood Testing: Increased Compliance and Market Expansion Demonstrated acceptance of blood test for CRC Screening 1 Surveys in the US confirm these results 2 Patient choice of methods has shown to increase screening compliance 3 Health economic benefit demonstrated 4 Incremental U.S. market opportunity estimated in excess of US$ 1 Bn annually 5 1i Adler et al, BMC Gastroenterology 2014, 14:183 doi:10.1186/1471-230x-14-183 2. Taber et al. (2012, ASPO): Preferences for a methylated DNA blood test for colorectal cancer among a multiethnic sample of screened and unscreened adults, 3 Inadomi J et al. Adherence to colorectal cancer screening, a randomized clinical trial of competing strategies, 2012, 4 U. Ladabaum et al. 5 Company and Analyst estimates 8 March 2015
Epi procolon : Simple and Efficient for Patients and Customers Easy for the patient - part of routine visits - no dietary restrictions Easy for the doctor - test (+): colonoscopy; test (-): get screened next year Easy for the lab - runs on existing hardware (ABI 7500 Fast Dx, Roche LC 480, others) 9 March 2015
Epi procolon : Powerful Screening Tool with Flexible Set Up Epi procolon (under US FDA review) Epi procolon 2.0 CE (CE marked, CFDA) + + + test result positive test result positive 10 ml blood sample - + + follow-up with colonoscopy follow-up with colonoscopy Epi procolon test result (3x testing 15 μl per well) - - + min. 45 μl DNA - - - negative negative 10 March 2015
Epi procolon : Validated in Clinical Trials US data generated in prospective trials for FDA submission 1 + + + US product 1,2 Sensitivity 68-73% China/EU product 3,4 Sensitivity 75-81% Non-inferiority to FIT demonstrated in headto-head study 2 (FIT sensitivity: 68%) - + + Specificity: 80-82% Specificity: 97-99% Case control studies in Europe and China using the 2 out of 3 algorithm 3,4 - - + - - - 1 Potter et al., Clinical Chemistry June 2014 clinchem.2013.221044 Validation of a Real-Time PCR-Based Qualitative Assay for the Detection of Methylated Sept9 DNA in Human Plasma, 2 Approx. 100 CRC cases from screening eligible patients and ~200 prospectively collected normal controls ; Non-inferiority (sensitivity) to OC-FIT-Chek proven in study 3 Case control study performed in Europe, 100 CRC cases, 148 normal controls, 4 Jin et al., accepted for publication 11 March 2015
Possible and Simple Performance Improvement + + + - + + positive (3% of all cases) re-test with FIT: 65% sensitivity 96% specificity overall performance! 1 - - + - - - 1 out of 3 positive results (15% of all cases) negative (82% of all cases) re-test re-test with Epi procolon: 70% sensitivity 90% specificity overall performance! 2 1 Based on post-hoc analysis of FIT/Sept9 comparison study 2 Based on post-hoc analysis of FIT/Sept9 comparison study and retesting of weak positive results with Epi procolon 12 March 2015
Regulatory Status of Epi procolon Europe: Commercially available since 2012 growing sales but at low levels since no active commercial effort underway for the time being CE marking based approval in other countries, e.g. Argentina China: Approved in China in December 2014 commercialized by BioChain U.S.A.: PMA under review by US FDA since early 2013 Response letter June 2014 FDA is looking for additional data supporting the assumption that Epi procolon will increase compliance to CRC screening recommendations in the intended use population (non-screening compliant) PMA still on file additional data to complete the PMA (no resubmission) 13 March 2015
ADMIT (Adherence to Minimally Invasive Testing) Study Study to demonstrate increased participation in CRC screening in patients being offered Epi procolon blood-based test vs. FIT stool-based test In agreement with the FDA, the study: will include patients actively managed by CRC screening programs within Kaiser Permanente and Geisinger Health Systems population which is non-compliant to CRC screening according to current screening guidelines is identified through electronic medical records 420 patients enrollment underway Primary endpoint: significant increase in compliance to CRC screening compared to current standard of care (FIT) at least 8% improvement Additional endpoint: adherence to colonoscopy upon positive test result Enrollment to be completed in Q1 2015; data expected in Q2 2015 14 March 2015
Overview Epi procolon Unmet need & commercial opportunity for Epi procolon Commercial strategy Epi prolung and future product opportunities Financial information and summary Appendix 15 March 2015
Joint Commercialization Agreement with Polymedco Inc. Market leader in the CRC screening field Ideally positioned (CRC focus) to address >$50m in annual sales >1,500 existing laboratory customers Epigenomics tasks: Manufacturing IP Regulatory Clinical Medical networks PRODUCT Joint Efforts: Key accounts Reimbursement Strategic marketing CHANNEL Polymedco s tasks: Marketing Sales Distribution Customer support Billing Collection 16 March 2015
Value Chain of Septin9 based CRC Screening pays COGS 1 profit share - Guidelines - sells to laboratories US$ 75-90* orders test Reference Laboratories provides result Healthcare Professionals invoices payers US$ 141* - Payors - 1 Cost of goods sold (COGS)* Company estimates 17 March 2015
Key Elements for Successful U.S. Market Penetration Reimbursement Medical Guidelines first 6 months generate health economic data convince expert groups / medical societies 7-18 months ensure reimbursement perform selected KOL studies >18 months governmental endorsement ensure guideline inclusion 18 March 2015
Strategic Collaboration with BioChain in China Stage 1: Epi procolon approved in China by CFDA* BioChain to start commercialization in 2015 Chinese guidelines for CRC screening in draft status Pricing and reimbursement discussions underway Stage 2: License to develop and commercialize Septin9 IVD tests Development underway, clinical studies started Epigenomics has rest of the world rights Enormous market opportunity CRC incidence increasing 290m people screening eligible in China * China Food and Drug Administration (CFDA) 19 March 2015
Overview Epi procolon Unmet need & commercial opportunity for Epi procolon Commercial strategy Epi prolung and future product opportunities Financial information and summary Appendix 20 March 2015
Epi prolung in the Diagnosis of Lung Cancer Currently under development into a blood-based assay Possible applications in treatment response monitoring 1 Attractive opportunity in screening of high risk patients Lung cancer diagnostic work - up including measurement of SHOX2 gene methylation Biomarker: methylated SHOX2 gene Utilization as reflex test for the diagnosis of lung cancer in bronchial lavage 1 Fleischhacker et al. PLoS ONE 10(2): e0118195. doi:10.1371/journal.pone.0118195 21 March 2015
R&D Capabilities and Future Product Opportunities Epigenomics core capabilities Biomarker discovery, confirmation and selection IVD test development, validation and regulatory >20 proprietary prognostic, predictive, response, diagnostic, and screening biomarkers in cancer indications Potential to evaluate and clinically validate in collaboration with BioChain Broad IP protection with 70 active patent families: protection along the value chain Epigenetic biomarker discovery and IVD development capabilities 22 March 2015
Overview Epi procolon Unmet need & commercial opportunity for Epi procolon Commercial strategy Epi prolung and future product opportunities Financial information and summary Appendix 23 March 2015
Key Financial Information (in thousand) 9M 2014 9M 2013 Revenue 1,095 961 EBIT (Operating Result) -5,390-5,174 Net loss -5,906-5,232 Cash consumption -5,947-5,191 (in thousand) Sep 30, 2014 Dec 31, 2013 Liquid Assets* 3,887 7,957 * incl. marketable securities Liquidity position at the end of 2014 around EUR 7.5m after the equity investment of EUR 4.2m by BioChain in October 2014 Liquid assets to fund operations until completion of ADMIT study and expected launch Up to EUR 9.4 m cash inflow in 2015 possible from conversion of outstanding bonds 24 March 2015
Shareholder Structure Type of shares Security code number ISIN registered shares A11QW5 DE000A11QW50 Abingworth BioChain Gilbert Gerber 9.6% 9.1% 3.7% Stock Exchange Frankfurt Stock Exchange, Prime Standard: ECX Freefloat 77.6% Type of program Sponsored Level 1 ADR Program Ratio Ticker symbol Total Shares Outstanding 1 ADR = 5 shares EPGNY 15.480.422* (19.2m fully diluted) Analyst coverage Equinet First Berlin Kempen & Co Maxim * As of January 31, 2015 25 March 2015
Why Invest in Epigenomics... Epi procolon the world s first IVD blood test intended for CRC screening: Extensively validated in clinical trials Proven utility in early CRC detection Not for sale or diagnostic use in the United States of America Approved in Europe and China Under FDA review for the U.S. market 26 March 2015
Thank you for your attention! Contact Investor Relations Europe Antje Zeise Manager Investor Relations Epigenomics AG T. +49 30 24345 386 ir@epigenomics.com U.S.A. Lauren Kwiecinski Senior Associate The Trout Group LLC T. +1 646 378 2934 lkwiecinski@troutgroup.com TICKER Bloomberg: ECX:GR Reuters: EXXG.DE Thomson ONE: ECX-XE ADR OTC: EPGNY INTERNET www.epigenomics.com www.epiprocolon.com www.epiprolung.com www.epigenomics.com
Overview Epi procolon Unmet need & commercial opportunity for Epi procolon Commercial strategy Epi prolung and future product opportunities Financial information and summary Appendix 28 March 2015
Methylated Septin9 Blood Based Test for CRC Detection Simple blood-based tests widely seen as best way to close the screening gap Epi procolon is based on a single epigenetic biomarker: methylated Septin9 Septin9 in CRC tissue fully methylated in >95% of all cancers High analytical sensitivity (6pg/ml), specific for colorectal cancer Equal capability to detect left and right sided cancerous lesions 29 March 2015
Comparison of non-invasive Screening Tests in the US Sample Location of Testing US Pricing (varies globally) Sensitivity (for CRC) Specificity Availability Colonoscopy Structural exam Endoscopy Center $800-$3160 b (variable rates) 95% g 95% g Through specialist FOBT FIT (multiple) Stool DNA (Cologuard ) Stool Stool Stool Clinical Lab Clinical Lab Companyowned CLIA Lab a $4.54 c $22.22 c $599 d 70% g (64-80%) 95% g (87-90%) IVD FDA m cleared 65.8-88.2% h 92.3% i CT Colonography Virtual Imaging Exam Endoscopy Center $550-$794 b (variable rates) 70-90% j (lesion-size dependent) Blood RNA (ColonSentry) Blood Clinical Lab Septin9 (multiple) Blood Clinical Lab $300-400 e $135-$150 f 61-72% k 73.3% l 89.7-94.6% h 86.6% i 86.0% j 66-77% k 81.5% l IVD FDA cleared IVD FDA approved Through specialist LDT Under FDA PMA Review US:LDT / EU:IVD a CLIA Clinical Clinical Laboratory Improvement Amendments US Center for Medicare & Medicaid Services bcost to insurance, endoscopy center, no polyp removed; Healthcare Bluebook fair pricing (Consumer Reports). c Maximum Medicare reimbursement rate.d Exact Sciences, published reports. ezehr L et. al. New York approval of ColonSentry, February 2012. f Cost of CE marked EU and US LDT Septin9 tests; cost to patient may not be covered by insurance. g Zauber AG et. al. Technology assessment report, project ID CRCC0608, 2009. h Colorectal Cancer Screening (PDQ ) National Cancer Institute at National Institutes of Health, newer FOBTs: nonrandomized controlled trial evidence, 2014. i Imperiale T et. al. New Engl J Med. 2014, 370(1287-1297). j Colorectal Cancer Screening (PDQ ) National Cancer Institute at National Institutes of Health,virtual colonoscopy (computer tomographic [CT] colonography), 2014. k Ganepola AP et. al. World J Gastrointest Oncolo. 2014, 6(4):83-97. l Johnson et. al. PloS One9(6): e98238. doi:10.1371/journal.pone.0098238 m IVD In Vitro Diagnostic 30 March 2015
Comparison Study Top Line Data: Cancer Detection by Stage from head-to-head FIT/Septin9 trial Epi procolon pt0/tis pt1 pt2 pt3 pt4 ptx unknown TOTAL Stage 0 / I 2/3 6/11 10/13 - - - 0/1 18/28 64.3% (45.8-79.3%) Stage II - - - 14/18 2/2 - - 16/20 80.0% (58.4-91.9%) Stage III - 0/1 0/2 14/19 1/1 - - 15/23 65.2% (44.9-81.2%) Stage IV - 2/2-0/1 6/6 1/1 3/3 12/13 92.3% (66.7-99.6%) Unknown* - - - - - 3/3 10/14 13/17 76.4% (52.7-90.4%) TOTAL * Staging information incomplete or unavailable 2/3 8/14 10/15 28/38 9/9 4/4 13/18 74/101 73.3% (63.9-80.9%) FIT pt0/tis pt1 pt2 pt3 pt4 ptx unknown TOTAL Stage 0 / I 0/3 5/11 11/13 - - - 1/1 17/28 60.7% (42.4-76.4%) Stage II - - - 14/18 2/2 - - 16/20 80.0% (58.4-91.9%) Stage III - 1/1 1/2 16/19 1/1 - - 19/23 82.6% (62.9-93.0%) Stage IV - 1/1-0/1 4/6 1/1 1/3 7/12 58.3% (32.0-80.7%) Unknown* - - - - - 1/3 6/11 7/14 50.0% (26.8-73.2%) TOTAL * Staging information incomplete or unavailable 0/3 7/13 12/15 30/38 7/9 2/4 8/15 66/97 68.0% (58.2-76.5%) 31 March 2015