Epigenomics AG. May

Transcription

1 Epigenomics AG May

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 forwardlooking 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 May 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 May 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 May 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 May 2015

6 Fighting a Treatable Disease: Colorectal Cancer Public Health Impact 90% 5-year survival rate in stages I and II >136,000 new cases and 50,000 deaths 1 Health Economic Impact USD 14 Bn CRC treatment cost p.a. 2 Over a third in late stage disease Colonoscopy every 10y or annual FIT 3 Screening is key! million people not screened for CRC 1 ACS Cancer Facts & Figures 2014, 2 Centers for Disease Control. "Vital signs: Colorectal cancer screening, incidence, and mortality ---United States, " MMWR Morb. Mortal. Weekly Report. 2011, 60(26): recommendation of the U.S. Preventive Services Task Force (USPSTF) for people aged between years, 6 May 2015

7 Goal: Increase in Screening Participation to 80%+ Campaign by ACS to get 80% screened by 2018 Reaching that goal would save over 200,000 lives 1 Simple blood test can significantly contribute to this goal May 2015

8 Blood Testing Increases Compliance Demonstrated acceptance of blood test for CRC Screening 1 Of the 63% who refused colonoscopy screening, 83% opted for Septin9 blood test Surveys in the U.S. confirm these results 2 Patient choice of methods has shown to increase screening compliance 3 1 Study performed at Charité University Hospital, Berlin (Germany): 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, May 2015

9 More Options for Physicians and Patients Imaging methods colonoscopy flexible sigmoidoscopy virtual colonography ingestible devices etc. Stool sampling fecal occult blood (FOBT) immunochemical (FIT) etc. Blood based Epigenomics: methylated Septin9 9 May 2015

10 Epi procolon : Simple and Efficient for Patients and Customers Easy for the patient part of routine visits; no dietary restrictions Easy for the doctor Septin9 (+): colonoscopy Septin9 (-): return next year Easy for the lab runs on existing hardware (validated for ABI, Roche) 10 May 2015

11 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 (3x15ul) min. 45 μl DNA negative (-) negative (-) 11 May 2015

12 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 12 May 2015

13 Possible Performance Improvement (post-hoc analysis 1,2 ) positive (+) (3% of all cases) re-test with FIT: 65% sensitivity 96% specificity overall combined performance out of 3 or weak positives (15% of all cases) negative (-) (82% of all cases) re-test re-test with Epi procolon: 70% sensitivity 90% specificity overall combined 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 13 May 2015

14 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 on file additional data to complete the PMA (no resubmission) 14 May 2015

15 Regulatory Status of Epi procolon - USA ADMIT Study (Adherence to Minimally Invasive Testing) Study was conducted with Kaiser Permanente and Geisinger Health Systems 413 subjects in two study arms (Epi procolon and FIT test) Epi procolon showed nearly 100% adherence rate significantly higher than stool-based FIT (88%) Margin of at least 8.2% higher acceptance achieved, but fell just short of statistical significance With the observed FIT adherence rate, a margin of 3.2% would have been sufficient, this was met with statistical significance Results will be submitted to the FDA in the coming weeks 15 May 2015

17 Joint Commercialization Agreement with Polymedco Market leader in the CRC screening field Ideally positioned (CRC focus) to address >10M FIT tests p.a. sold >1,500 existing laboratory customers Epigenomics: Manufacturing IP Regulatory Clinical Medical networks PRODUCT Joint Efforts: Key accounts Reimbursement Strategic marketing CHANNEL Polymedco: Marketing Sales Distribution Customer support Billing Collection 17 May 2015

18 Value Chain of Septin9 based CRC Screening pays COGS 1 profit share - Guidelines - sells to laboratories US$ orders test Reference Laboratories provides result Healthcare Professionals invoices payers US$ Payors - 1 Cost of goods sold (COGS ) 2 Company estimates 18 May 2015

19 Blood Testing to drive Market Expansion Health economic benefit demonstrated 1 Septin9 based screening proven to be cost-effective Incremental U.S. market opportunity estimated in excess of USD 1 Bn annually 2 1 U. Ladabaum et al. 2 Company and Analyst estimates 19 May 2015

20 Key Elements for Successful U.S. Market Penetration Reimbursement Medical Guidelines first 6 months generate health economic data expert groups / medical societies 7-18 months ensure reimbursement selected KOL studies >18 months governmental endorsement guideline inclusion 20 May 2015

21 Strategic Collaboration with BioChain in China Stage 1: Epi procolon approved in China by CFDA 1 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 the rights for the rest of the world Enormous market opportunity CRC incidence increasing 290M people screening eligible in China 1 China Food and Drug Administration (CFDA) 21 May 2015

23 Epi prolung : Current Status in the Diagnosis of Lung Cancer Biomarker: methylated SHOX2 gene 1 Validated as reflex test for the diagnosis of lung cancer in bronchial lavage Currently marketed as CE-marked IVD test in Europe Lung cancer diagnostic work - up including measurement of SHOX2 gene methylation Prospective study demonstrated that Epi prolung testing provides additional information in cases where cytology is negative or inconclusive 2 The combination of both methods resulted in clinical sensitivity of 98% 1 Kneip et al., SHOX2 DNA Methylation Is a Biomarker for the Diagnosis of Lung Cancer in Plasma. Journal of Thoracic Oncology, Vol 6, Number 8, August Klauschen et al., Performance of Epi prolung in combination with cytology in bronchial lavage samples from clinical routine. In preparation 23 May 2015

24 Epi prolung Next Steps Development of blood-based version Applications in treatment response monitoring Study demonstrated mshox2 to be sensitive and specific for therapy monitoring and early detection of tumor response 1 More rapid and sensitive determination than CT scan Multi-centric study with larger patient population planned for verification Possible initial application in follow-up of positive results in low dose spiral CT Attractive future opportunity in screening of high risk patients 1 Fleischhacker et al. PLoS ONE 10(2): e doi: /journal.pone May 2015

25 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 25 May 2015

27 Key Financial Information (in thousand) Q Q Revenue EBIT (Operating Result) -3,164-2,000 Net loss -3,164-2,240 Cash consumption -2,288-1,475 (in thousand) Mar 31, 2015 Dec 31, 2014 Liquid Assets* 6,421 7,495 * incl. marketable securities Liquid assets to fund operations until expected US launch Up to EUR 7.9M additional cash inflow in 2015 possible from conversion of outstanding bonds (EUR 1.5M so far in 2015) Capital Increase up to EUR 5M Subscription price EUR 5.12, subscription period May 13, 15 - May 27, May 2015

28 Shareholder Structure Type of shares Security code number ISIN Stock Exchange registered shares A11QW5 DE000A11QW50 Frankfurt Stock Exchange, Prime Standard: ECX BioChain Abingworth Freefloat 9.1% 4.9% 86.0% 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 Edison Equinet First Berlin Kempen & Co Maxim 1 As of April 30, May 2015

29 Why Invest in Epigenomics... Epi procolon the world s first IVD blood test intended for CRC screening: Extensively validated Proven utility Approved in Europe and China Under FDA review Not for sale or diagnostic use in the United States of America 29 May 2015

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

32 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 32 May 2015

33 Comparison of non-invasive Screening Tests in the U.S. 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 33 May 2015

34 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 74/101 FIT pt0/tis pt1 pt2 pt3 pt4 ptx unknown TOTAL 73.3% ( %) 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/ % ( %) 34 May 2015