Company Presentation January 2013 - Epigenomics AG

Epigenomics AG 

Corporate Presentation 

January 2013 

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 are not available 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 | January 2013

Contents 

� Epigenomics overview 

� Epi proColon 

• Unmet need & opportunity 

• Characteristics & clinical data 

• US strategy 

� Epi proLung & future product opportunities 

� Summary 

� Appendix 


Epigenomics at a Glance 

� Epigenomics is a leader in the field of DNA methylation based diagnostics for the early 

detection of cancer 

� Epigenomics has two promising commercial IVD tests with a CE mark: 

� Epigenomics’ near-term strategy is focused on obtaining FDA approval for Epi proColon® 

� Epigenomics completed its PMA filing of Epi pro Colon® to the FDA in 2012 

� A decision from the FDA is expected in the second half of 2013 

� Epigenomics is already building a customer base ahead of US approval. Currently over 

1,000 blood-based Septin9 laboratory-developed tests (LDT) are performed in the US weekly 

4 | January 2013 

� Addresses unmet need in the mass market of colorectal cancer (CRC) 

screening using the validated Septin9 biomarker 

� Is the first CE approved proprietary IVD test world wide that enables 

CRC screening based on a simple blood sample 

� Addresses unmet need in the niche market of difficult to diagnose lung 

cancer using the validated SHOX2 biomarker

Key Financials 9M 2012 

� Tightly controlled spending (incl. US commercial build-up) 

(in € thousand) 9M 2012 9M 2011 

Revenue 672 1,242 

EBIT (Operating Result) -9,430 -10,748 

(in € thousand) 09/30/2012 12/31/2011 

Liquid Assets* 6,187 13,985 

* incl. marketable securities 

� Operations in Germany and the US. Total current number of employees 35 

Full financial statements: http://www.epigenomics.com/en/news-investors/investors/financial-reports.html 


Contents 







� Summary 

� Appendix 


Colorectal Cancer (CRC) Screening Saves Lives and Money; 

However 1 in 3 Screening Eligible Adults are not Screened 

CRC Screening Saves Lives and Money… 

� CRC is the second largest cancer killer in 

the US 


140,000 new cases 

50,000 deaths 1 

� CRC is treatable if detected early enough 

� 5-year survival for diagnosed and treated 

Stage I/II CRC: 90% 

� Estimated US direct medical cost of CRC 

care in 2010 was $14 billion 

…However a Substantial Percentage of 

Citizens Remains Unscreened For CRC 

Colonoscopy 

35 million 

U.S. citizens 

are unscreened 

Stool 

based 

tests 

Key challenge: non-compliance to existing 

stool-based or invasive (colonoscopy) 

CRC screening options 

1) American Cancer Society.: Cancer Facts and Figures 2012. Atlanta, Ga: American Cancer Society, 2012. 

Graph: Centers for Disease Control. "Vital signs: Colorectal cancer screening, incidence, and mortality---United States, 2002-2010. " MMWR Morb. Mortal. Weekly Report. 2011, 60(26):884-889.

Blood-based CRC Screening: A Paradigm Shift Addressing Market Need 

When screening is easy/ 

convenient and recommended 

by their provider: 

� More patients are screened 

� More cancers are detected 

early 

� Less deaths result from cancerrelated 

disease 


Epi proColon: A Simple Blood Test for 

Early Detection of CRC 

Benefits for doctors and patients: 

� Non-invasive 

� No dietary/medication 

restrictions 

� Quick and easy, done anytime 

� Helps with screening resistant 

patients 

� Can be part of routine visits 

American Cancer Society.: Cancer Facts and Figures 2012. Atlanta, Ga: American Cancer Society, 2012. Centers for Disease Control. 

"Vital signs: Colorectal cancer screening, incidence, and mortality ---United States, 2002-2010 " MMWR Morb. Mortal. Weekly Report. 2011, 60(26):884-889.

Blood-based Screening is Widely Accepted 

62% 

Offer to participate in alternative screening 

methods to those who decline colonoscopy 


38% 

87% 

pro 

colonoscopy 

against 

colonoscopy 

4% Blood test 

9% 

Stool test 

No screening 

Berlin Adherence Study Interim 

Results: 

� Study goal: to determine the 

demographic, health psychology 

and socio-economic traits 

associated with compliance to 

CRC screening guidelines. 

� Approx. 200 patients 

� Further surveys by Huntsman 

Cancer Center and others 

confirm these results in the US 

As presented at UEG 2012

Contents 







� Summary 

� Appendix 


Methylated Septin9 is a Suitable Marker for CRC Screening 

� Test consists of one single proprietary 

epigenetic biomarker: 

methylated Septin9 gene 

� Detection of free circulating tumor DNA in 

blood by real-time PCR test 

� Analytical sensitivity: down to 6 pg / ml 

� Strong patent position: biomarkers, 

technology, processes – Epigenomics own 

� 13 published retrospective clinical studies 

with more than 4,300 subjects 

� Two prospective clinical screening studies 

completed 


CRC Detection in Right and Left Colon (Prof. Béla Mólnar) 

� Blinded case control study, 184 study participants, 

92 CRC cases (56 left-, 36 right-sided cases) – tested with 

gFOBT* and Septin9 

� Sensitivity for detection of left- and right-sided cases was 

96% and 94% respectively 

� Septin9 was confirmed as a highly sensitive biomarker for the detection of CRC in blood 

� Septin9 methylation level shows no difference between left and right side colon cancers 


CRC 

sensitivity 

Left-sided 

cancers 

Right-sided 

cancers 

gFOBT* 68.2 83.3 50.0 70.6 

Septin9 95.6 96.4 94.4 84.8 

* guaiac Fecal Occult Blood Test 

Specificity 

Plasma Methylated Sept9 Is a Screening Marker in Both Left- and Right-Sided Colon Cancer. Comparison to FOBT and CEA Results Lead Author: 

Professor Dr. Kinga Toth from the 2nd Department of Internal Medicine of Semmelweis University in Budapest, Hungary Abstract No. u1895

Epi proColon®: One Assay – One Marker 

� Easy & fast: real-time PCR based, 

8 hours time to result 

� Compliant: cGMP manufactured, 

21 CFR Part 820 and ISO 13485 compliant; 

runs on Roche LC480 and AB7500. 

US product runs on FDA cleared 

real-time PCR device (AB7500 Fast DX) 


•Fits into Current Lab Routine 

One Assay – One Marker -> Simple Qualitative Interpretation 

� CE marked for EU market 

� Automated: multiple volume dependent 

automation solutions possible 

� Flexible: adaptable to market requirements: 

high sensitivity or high specificity 

Not for sale or diagnostic use in the United States of America

Epi proColon®: Clinical Validation Study Data 


Case control 

study (EU) 

September 2011 

Prospective 

screening study 

pivotal clinical trial for 

FDA submission 

FIT comparison 

study 

December 2012 

Samples 247 7,940* 300 

Sensitivity 95% 2 68% 1 71% 3 

Specificity 85% 2 80% 1 81% 3 

Assay / Kit 

Epi proColon® 

2.0 CE 

Epi proColon® 2.0 

Epi proColon® 2.0 

* tested samples selected from a prospectively collected cohort of 7.940 individuals at average risk for CRC. Tested samples included all CRC cases, all adenomas 

and significant number of polyps identified in this cohort in addition to a randomized selection of study individuals with no evidence of disease (NED) 

1) Prospective screening study results with Epi proColon® 2.0 final product published Dec 9, 2011 

2) Case control study results with Epi proColon® 2.0 final product published Sep 19, 2011 – also see slide 34 for set up - 3) study results published on Dec 4 th , 2012

Fecal Immunochemical Test (FIT) Comparison Study - Enrollment Metrics 

� FIT head-to-head comparison study protocol 

agreed with FDA and study started in Q2 2012 

completed Dec 04, 2012 

� FDA 510(k) cleared FIT test used is major 

commercial product in the US 

� Primary endpoint: demonstrate statistical 

non-inferiority of Epi proColon ® to FIT 

� Inclusion criteria: average risk, screening age, 

asymptomatic, no family history, no previous 

disease, screening colonoscopy 

� Blinded testing, FIT and Epi proColon®, performed 

at independent laboratory in the US 

� 70 sites throughout the US participated 

in the study 

� Testing was performed according to 

manufacturer’s IFUs (100 ng/ml cut-off for FIT) 


104 valid cancer cases included 

(screening patients, matched samples 

taken 10 days post colonoscopy) 

103 with Septin9 result 

98 with FIT result 

198 valid asymptomatic 

average risk individuals 

(matched samples taken 

pre-colonoscopy, fully prospective) 

198 with Septin9 result, 

195 with FIT result 

� 3 CRC (excluded from analysis) 

� 25 advanced adenomas (25 with Septin9 

result, 24 with FIT result) 

� 73 small polyps 

� 97 subjects with no evidence of disease 

(97 with Septin9 result, 95 with FIT result)

Comparison Study Top Line Data: Sensitivity 

FIT 

Agreement between both methods: 62.2% (52.4 - 71.2%) 

Combined sensitivity: 87.8% (79.8 - 92.9%) 



- + total 

- 12 20 32 

+ 17 49 66 

total 29 69 98 

70% sensitivity 

(60.7-78.5%) 

67% sensitivity 

(57.6-75.8%) 

Non-inferiority to FIT in respect to sensitivity demonstrated! 

Head-to-head comparison study results with Epi proColon® and FIT published Dec 4, 2012

Comparison Study Top Line Data: Sensitivity by pT and CRC Stage 

Epi proColon® pT0/Tis pT1 pT2 pT3 pT4 pTx unknown TOTAL 

Stage 0 / I 2/3 4/8 8/11 - - - 0/1 14/23 60.9% (36.5-75.5%) 

Stage II - - - 10/14 2/2 - - 12/16 75.0% (50,5-89.8%) 

Stage III - 0/1 - 13/18 1/1 - - 14/20 70.0% (48.1-85.5%) 

Stage IV - 2/2 - 0/1 6/6 1/1 2/2 11/12 91.7% (64.4-99.6%) 

Unknown* - 1/1 - 1/1 - 6/6 14/24 22/32 68.8% (51.4-82.0%) 

TOTAL 2/3 7/12 8/11 24/34 9/9 7/7 16/27 73/103 70.9% (61.5-78.8%) 

*…Staging information incomplete or unavailable 

FIT pT0/Tis pT1 pT2 pT3 pT4 pTx unknown TOTAL 

Stage 0 / I 0/3 3/8 10/11 - - - 1/1 14/23 60.9% (45.4-82.8%) 

Stage II - - - 10/14 2/2 - - 12/16 75.0% (50.5-89.8%) 

Stage III - 1/1 - 15/18 1/1 - - 17/20 85.0% (64.0-94.8%) 

Stage IV - 1/1 - 0/1 4/6 1/1 1/2 7/11 63.6% (35.4-84.8%) 

Unknown* - 1/1 - 1/1 - 2/6 12/20 16/28 57.1% (39.1-73.5%) 

TOTAL 0/3 6/11 10/11 26/34 7/9 3/7 14/23 66/98 67.3% (57.6-75.8%) 

*…Staging information incomplete or unavailable 



Comparison Study Top Line Data: Specificity (based on non-CRC cases) 

FIT 



- + total 

- 152 36 188 

+ 3 1 4 

total 155 37 192 

81% specificity 

(74,9-85,9%) 

98% specificity 

(94,8-99,2%) 

Agreement between both methods: 80% (73,4-84,8%) 

“Non-inferiority” to FIT (specificity) 

was statistically inconclusive 


Comparison Study Top Line Data: 

Detection of Advanced Adenoma and Small Polyps 

FIT 


•Advanced Adenoma* Small Polyps* 


- + Total 

- 21 2 23 

+ 0 1 1 

Total 21 3 24 

*…defined as high grade dysplasia or 

villous component, or >10 mm 

FIT 


- + Total 

- 62 10 72 

+ 1 0 1 

Total 63 10 73 

*…defined as

Contents 







� Summary 

� Appendix 


FDA Submission Status: Completed in 2012 


Pre-IDE meeting 

Preliminary data 

review and module 

plan determination 

� 

Module 1 

manufacturing and 

quality system 

sections 

� 

Module 2 

software validation & 

instrumentation 

Premarket 

approval 

submission 

(PMA) 

� 

Module 3 

analytical validation 

Module 4 

clinical validation 

� 

� 

� Filing completed in 2012, feedback on first modules received, 

ongoing and constructive dialogue with FDA (no major obstacles encountered so far) 

� Panel meeting expected to be convened by FDA upon review of submitted data

Building a Customer Base Ahead of US Approval 

Increased Adoption of Septin9 

� Available coast-to-coast in the US 

� Continuous growth of Septin9 testing through 

LDT partners: Quest, ARUP, Companion Dx 

(recently launched) and Gamma Dynacare 

� >1,000 tests per week in North America 

� LDT partners likely to become future 

Epigenomics customers 

� CPT code 81401 announced and payment 

being established 

� Payer dialogue with private insurers & CMS ongoing 


Expected US Product Launch of Epi proColon® in 2013 

Pre-launch 

Private payer & CMS 

dialogue for reimbursement 

Enlist thought leaders & 

MAB to guide launch 

Leverage LDT partner test 

marketing 

Build proprietary core 

commercial team 


Pre- and post- 

launch 

Create awareness through 

publications, medical 

conferences & symposia 

Educate clinical labs, 

primary care and GI 

Target advocacy groups to 

drive public awareness 

Perform health 

economic studies 

FDA approval 

Post-launch 

Guideline inclusion 

Adherence & compliance 

studies 

Expand proprietary 

commercial team 

Direct marketing efforts

Contents 







� Summary 

� Appendix 


Epi proLung® - Market Introduction 

� Marketed in Europe since mid-2010 

� Reflex test for the diagnosis of lung cancer 

� Biomarker: methylated SHOX2 gene 

� Increasing awareness and interest in expert 

community 

� Market introduction through academic studies 


CE validation 

study 2010 

Study by Charité Berlin 2012 - 228 samples analyzed 

Epi proLung® 

performance 

Cytology 

Epi proLung® combined 

with cytology 

Sensitivity 81% 77% 77% 98% 

Specificity 95% 97% 95% 92%

R&D Capabilities and Future Product Opportunities 

Biomarker discovery, 

confirmation and selection 

� Epigenomics’ core capabilities and proprietary technology enabling epigenetic 

biomarker discovery and IVD development 

� Epigenomics’ DNA methylation biomarkers have broad IP protection: 70 active patent 

families and licenses provide extensive protection for markers and methods 

� Epigenomics has identified and validated over 20 proprietary prognostic, predictive, 

response, diagnostic, and screening biomarkers in colorectal cancer, lung cancer, 

prostate cancer, and bladder cancer 

� Internal development is currently focused on Epi proColon®, Epigenomics actively 

pursues license opportunities for several biomarkers 


•core capabilities 

IVD test development, 

validation and regulatory 

Commercialization

Contents 







� Summary 

� Appendix 


•ACHIEVEMENTS 

•UPCOMING MILESTONES 

Recent Achievements & Upcoming Milestones: 2011 - 2013 

Signed Septin9 option agreement 

with QIAGEN N.V. 

Restructured operations: 

focus on the US 


EU launch of Epi proColon® 2.0 CE 

Completed FIT comparison study 

Filed 4 modules of PMA to FDA 

Supervisory and Management 

Board changes implemented 

2011 2012 2013 

Securing of additional 

financial resources 

Commercial 

Development 

Corporate 

Receive decision from FDA 

on Epi proColon PMA 

Develop automation solutions 

Launch Epi proColon® in the US 

Drive sales and adoption in Europe 

Seek additional partnerships

Why Invest in Epigenomics.... 


� Epi proColon®: the world’s first IVD test for early 

detection of colorectal cancer in blood 

� Methylated Septin9: the world’s most thoroughly 

validated biomarker for blood based CRC detection 

- Prospectively validated 

- Proven utility in CRC early detection 

- Globally available as IVD (EU) or LDT (US) 

- Under FDA review as IVD for the US market 

� IVD test for lung cancer diagnosis in the EU market 

� Attractive pipeline of epigenetic biomarkers for 

numerous applications 

� Strong partnerships: IVD companies and laboratories

Thank You for Your Attention! 

Contact Investor Relations 

Europe U.S.A. 

Antje Zeise 

Manager Investor Relations 

Epigenomics AG 

T +49 30 24345 0 

ir@epigenomics.com 

www.epigenomics.com 

Christine Yang 

Vice President 

The Trout Group LLC 

T +1 646 378 2929 

cyang@troutgroup.com 

TICKER 

Bloomberg: ECX:GR 

Reuters: EXXG.DE 

Thomson ONE: ECX-XE 

INTERNET 

www.epigenomics.com 

www.epiprocolon.com 

www.epiprolung.com

Contents 







� Summary 

� Appendix 


CRC Tumorgenesis and Septin9 


Septin9 Tests: Continious Improvement as Assay Technology Evolved 

Sensitivity / Specificity 

100% 

90% 

80% 

70% 

60% 

50% 

40% 

30% 

20% 

10% 

0% 


1 2 3 4 5 6 7 8 9 10 11 12 13 

Technology Development 

1 st Gen. Tests Epi proColon® 

Case Control Studies 

5.000 

4.500 

4.000 

3.500 

3.000 

2.500 

2.000 

1.500 

1.000 

500 

0 

Subjects tested (cumulative)

Epi proColon® 2.0: Powerful Screening Tool with Flexible Set Up 


Method Specificity Sensitivity Positive 

Predictive Value 3) 

Epi proColon® 2.0 CE* 

(marketed in Europe) 1) 99.3% 80.6% 45.7% 

OC-Sensa Micro qFIT1x2 2) 93.7% 69.2% 7.5% 

1) Tetzner et al. UEGW 2011, 2 Allison et al. 1996 NEJM, 2) Park et al. 2010. Am. J. Gastro. 

3) Orange figure: CRC with positive test result, grey figure: healthy with positive test result assuming a prevalence for CRC of 0.7%.

Company Presentation January 2013 - Epigenomics AG

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