Epigenomics AG. March

Transcription

1 Epigenomics AG March

2 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

3 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

4 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

5 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

6 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 million people not screened for CRC ACS 3 : campaign to get 80% screened by ACS Cancer Facts & Figures 2014, 2 recommendation of the U.S. Preventive Services Task Force (USPSTF) for people aged between years, 3 American Cancer Society (ACS) 6 March 2015

7 CRC Screening with Epi procolon 7 March 2015

8 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: / x 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

9 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

10 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

11 Epi procolon : Validated in Clinical Trials US data generated in prospective trials for FDA submission 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, Potter et al., Clinical Chemistry June 2014 clinchem 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

12 Possible and Simple Performance Improvement positive (3% of all cases) re-test with FIT: 65% sensitivity 96% specificity overall performance! 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

13 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

14 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 Q March 2015

15 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

16 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

17 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

18 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

19 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

20 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

21 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): e doi: /journal.pone March 2015

22 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

23 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

24 Key Financial Information (in thousand) 9M M 2013 Revenue 1, 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

25 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 * (19.2m fully diluted) Analyst coverage Equinet First Berlin Kempen & Co Maxim * As of January 31, March 2015

26 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

27 Thank you for your attention! Contact Investor Relations Europe Antje Zeise Manager Investor Relations Epigenomics AG T U.S.A. Lauren Kwiecinski Senior Associate The Trout Group LLC T TICKER Bloomberg: ECX:GR Reuters: EXXG.DE Thomson ONE: ECX-XE ADR OTC: EPGNY INTERNET

28 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

29 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

30 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 % 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 $ e $135-$150 f 61-72% k 73.3% l % 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 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, h Colorectal Cancer Screening (PDQ ) National Cancer Institute at National Institutes of Health, newer FOBTs: nonrandomized controlled trial evidence, i Imperiale T et. al. New Engl J Med. 2014, 370( ). j Colorectal Cancer Screening (PDQ ) National Cancer Institute at National Institutes of Health,virtual colonoscopy (computer tomographic [CT] colonography), k Ganepola AP et. al. World J Gastrointest Oncolo. 2014, 6(4): l Johnson et. al. PloS One9(6): e doi: /journal.pone m IVD In Vitro Diagnostic 30 March 2015

31 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/ /1 18/ % ( %) Stage II /18 2/ / % ( %) Stage III - 0/1 0/2 14/19 1/ / % ( %) Stage IV - 2/2-0/1 6/6 1/1 3/3 12/ % ( %) Unknown* /3 10/14 13/ % ( %) TOTAL * Staging information incomplete or unavailable 2/3 8/14 10/15 28/38 9/9 4/4 13/18 74/ % ( %) FIT pt0/tis pt1 pt2 pt3 pt4 ptx unknown TOTAL Stage 0 / I 0/3 5/11 11/ /1 17/ % ( %) Stage II /18 2/ / % ( %) Stage III - 1/1 1/2 16/19 1/ / % ( %) Stage IV - 1/1-0/1 4/6 1/1 1/3 7/ % ( %) Unknown* /3 6/11 7/ % ( %) TOTAL * Staging information incomplete or unavailable 0/3 7/13 12/15 30/38 7/9 2/4 8/15 66/ % ( %) 31 March 2015