Epi proColon - Epigenomics AG

Epigenomics AG
January 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 | January 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 | January 2015
DNA Methylation Diagnostic Products for Oncology
Epi proColon®
Epi proLung®
blood-based colorectal cancer (CRC)
screening1,2
tissue assay for lung cancer
Addressing low compliance to
currently available CRC screening
Aid in difficult to diagnose lung cancer
Potential to be developed into blood based assay
diagnosis1
1CE
4 | January 2015
marked and commercially available in Europe 2under FDA review and not commercially available in the U.S.
 Overview
 Epi proColon®
• Unmet need & commercial opportunity for Epi proColon®
• Commercial strategy
 Epi proLung® and future product opportunities
 Financial information and summary
 Appendix
5 | January 2015
Colorectal Cancer in the US: A Treatable Disease
US Public Health Impact:
 > 136,000 new cases 50,000 deaths1
 90% 5-year survival rate in stages I and II
 60% diagnosed in later stages
US Health Economic Impact:
 US$ 14 Bn CRC treatment cost p.a.
 Over a third in late stage disease
Screening is key to success in fighting CRC:
 USPSTF recommendation: colonoscopy every 10y or annual FIT between age 50-75
 Currently, 25-30 million people not screened for CRC
 ACS launched campaign to get 80% of US population screened by 2018
1ACS
6 | January 2015
Cancer Facts & Figures 2014
Proposed Intended Use of Epi proColon® Test
7 | January 2015
Blood Testing: Increased Compliance and Market Expansion
 Demonstrated acceptance of blood test
for CRC Screening1
 Surveys in the US confirm these results2
 Patient choice of methods has shown
to increase screening compliance3
 Health economic benefit demonstrated4
 Incremental U.S. market opportunity
estimated in excess of US$ 1 Bn annually5
1iAdler
et al, BMC Gastroenterology 2014, 14:183 doi:10.1186/1471-230X-14-1832. 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, 4U. Ladabaum et al. 5Company and Analyst estimates
8 | January 2015
Epi proColon®: Powerful Screening Tool with Flexible Set Up
+++
- ++
10 ml blood sample
Epi proColon®
test result
(triplicate testing
of 15 μl each)
min. 45 μl DNA
- -+
- - -
Epi proColon®
Epi proColon® 2.0 CE
(under US FDA review)
(CE marked product)
test result
positive
follow-up with
colonoscopy
to confirm /
as first step
of treatment
test result
negative
re-test next year
9 | January 2015
test result
positive
follow-up with
colonoscopy
to confirm /
as first step
of treatment
test result
negative
re-test next year
Epi proColon®: Optimized for Higher Sensitivity or Specificity
 Prospective data in the US generated in connection with PMA submission to FDA
 Case-control studies (higher risk patients) in Europe and China using “2/3 algorithm”
4764
81%
75%
82%
99%
97%
EU CE study
Nov. 2011
Jin et al.
Jan.2014
7,940
3011
Sensitivity
68%
73%
Specificity
80%
Potter et al.
Dec. 2011
2503
Size (patients)
1
10 | January 2015
Epi proColon® 2.0 CE
Johnson et al.
Dec. 2012
Epi proColon®
(FIT: 68%2)
Approx. 100 CRC cases from screening eligible patients and ~200 prospectively collected normal controls 2 Non-inferiority (sensitivity) to OC-FIT-Chek™ proven in study
3 Case control study performed in Europe, 100 CRC cases, 148 normal controls, 4Jin et al., accepted for publication
Possible Performance Improvement (Next Generation Products)
+++
- ++
min. 45 μl DNA
- -+
- - -
test result
positive (3%)
follow-up with
colonoscopy
to confirm /
as first step
of treatment
re-test with FIT:
65% sensitivity
96% specificity
overall performance!1
test again
(15% of all samples)
test result
negative (82%)
re-test with EPC:
70% sensitivity
90% specificity
overall performance!2
re-test next year
1Based
2Based
11 | January 2015
on post-hoc analysis of FIT/Sept9 comparison study
on post-hoc analysis of FIT/Sept9 comparison study and retesting of „weak positive“ results with Epi proColon®
Predicted Programmatic Performance1
Initial testing
testing after
12 months
testing after
24 months
Cumulated after 24 months1
Epi proColon®
73%
+20%
+5%
98%
(55% specificity)
Epi proColon®
re-test “weak
pos.” with EPC
70%
+21%
+6%
97%
(73% specificity)
Epi proColon®
re-test “weak
pos.” with FIT
65%
+23%
+8%
96%
(89% specificity)
 Programmatic performance to be studied in post approval study to support
screening claims and guideline inclusion
 3-year cost (significantly) lower than other competing CRC screening tests
1theoretical
12 | January 2015
calculation (estimate) not validated by programmatic investigation
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 – to be commercialized by BioChain
U.S.A.:
 PMA under review by US FDA since early 2013
 Response letter
 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) – data to supplement PMA once available
13 | January 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 Clinic
• population that is non-compliant to CRC screening according to current
screening guidelines is identified through electronic medical records
• 420 patients - could be enrolled in a few months
• 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
• Data is expected in Q1/Q2 2015
• Study design agreed with FDA, first patients already enrolled
14 | January 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 | January 2015
Joint U.S.-Commercialization Agreement with Polymedco Inc.
 Polymedco U.S. market leader in the CRC screening field >$50m in test sales
 Polymedco is ideally positioned (CRC focus) to address >1,500 existing LAB customers
Epigenomics tasks:
“Legal Manufacturer”
Joint Efforts:
Regulatory support
of product
Key account
management
Clinical studies
Reimbursement
support to customers
KOL network and
medical societies
PRODUCT
16 | January 2015
Marketing strategy
and messaging
CHANNEL
Polymedco’s tasks:
Laboratory, patient
and physician marketing
Sales, distribution
and customer support
Billing and credit collection
Value Chain of Septin9 based CRC Screening
supplies Epi proColon® in bulk ex-factory
direct cost of goods paid by Polymedco
plus 50% gross profit share
sells Epi proColon® kits to perform Septin9 test,
bears marketing and sales costs
performs Septin9 test
as ordered by HC provider
Profit share
agreement
with
Polymedco
$ 70-90*
paid by lab to
Polymedco
$ 141*
paid by CMS
or commercial
payer to lab
Healthcare provider counsels
patient about CRC screening;
orders test from the lab
* Company estimates
17 | January 2015
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
Epigenomics’ Role for Successful U.S. Market Penetration
governmental endorsement
ensure guideline inclusion
18 | January 2015
Broad Strategic Collaboration with BioChain for Chinese Market
 Stage 1: CFDA* submission for approval of Epi proColon®
 Clinical study successfully completed in April
 Approved by CFDA
 Stage 2: Exclusive license to subsequently develop and commercialize
own version of Septin9 IVD tests in the Chinese market
 Epigenomics would have rest of the world rights to this product
Enormous market opportunity –
CRC incidence increasing –
290m people screening eligible in China
* China Food and Drug Administration (CFDA)
19 | January 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 | January 2015
Epi proLung® - Market Introduction
 CE-marked
 Biomarker: methylated SHOX2 gene
 Reflex test for the diagnosis of lung cancer
 Increasing awareness through publications and
KOL driven studies
 Potential to develop into blood based assay
 Methylated SHOX2 allows rapid and sensitive
determination of tumor response and
therapy monitoring in plasma of lung
cancer patients
 Multi-centric studies with larger patient
populations planned
21 | January 2015
R&D Capabilities and Future Product Opportunities
•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, who
has option to retain Chinese market and Epigenomics gets access to results for RoW
 Broad IP protection with 70 active patent families: protection along the value chain
 Epigenetic biomarker discovery and IVD development capabilities
22 | January 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 | January 2015
Key Financial Information
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
Sep 30, 2014
Dec 31, 2013
3,887
7,957
(in € thousand)
(in € thousand)
Liquid Assets*
* incl. marketable securities
 Expected liquidity position at the end of 2014 around EUR 6-7m after
the equity investment of EUR 4.2m by BioChain in October 2014
 Liquid assets should be sufficient to fund operations until after
completion of ADMIT study and expected FDA approval decision
 Up to EUR 9.4 m cash inflow in 2015 from conversion of outstanding bonds possible
24 | January 2015
Why Invest in Epigenomics....
Epi proColon®
the world’s first IVD
blood test suitable
for CRC screening:
 extensively
validated
in clinical trials
 proven utility
in CRC early detection
 under FDA review for the U.S. market
and CFDA review in China
25 | January 2015
Not for sale or diagnostic use in the United States of America or China
Thank you for your attention!
Contact Investor Relations
Europe
U.S.A.
Antje Zeise
Manager Investor Relations
Epigenomics AG
T. +49 30 24345 386
ir@epigenomics.com
Lauren Kwiecinski
Senior Associate
The Trout Group LLC
T. +1 646 378 2934
lkwiecinski@troutgroup.com
TICKER
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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
27 | January 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
 High analytical sensitivity (6pg/ml),
specific for colorectal cancer
 Equal capability to detect left and right
sided cancerous lesions
28 | January 2015
Epi proColon®: Simple and Efficient for Patients and Customers
 Single blood tube - part of routine visits - no dietary restrictions
 Real-time PCR test, runs on existing hardware (AB 7500 Fast Dx, Roche LC 480, others)
 8 hours time to result, minimal training requirement for lab personnel
29 | January 2015
Comparison of non-invasive Screening Tests
Colonoscopy
FOBT
FIT
(multiple)
Stool DNA
(Cologuard™)
Sample
Structural
exam
Stool
Stool
Stool
Location of
Testing
Endoscopy
Center
Clinical Lab
Clinical Lab
Companyowned CLIA
Laba
Endoscopy
Center
Clinical Lab
Clinical Lab
US Pricing
(varies
globally)
$800-$3160b
(variable rates)
$4.54c
$22.22c
$599d
$550-$794b
(variable rates)
$300-400e
$135-$150f
Sensitivity
(for CRC)
95%g
70%g
(64-80%)
65.8-88.2%h
92.3%i
70-90%j
(lesion-size
dependent)
61-72%k
73.3%l
Specificity
95%g
95%g
(87-90%)
89.7-94.6%h
86.6%i
86.0%j
66-77%k
81.5%l
LDT
Under FDA
PMA Review
US:LDT /
EU:IVD
Availability
Through
specialist
IVD–FDAm
cleared
IVD–FDA
cleared
IVD–FDA
approved
CT
Blood RNA
Colonography (ColonSentry)
Virtual Imaging
Blood
Exam
Through
specialist
Septin9
(multiple)
Blood
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
a
30 | January 2015
Comparison Study Top Line Data: Cancer Detection by Stage
Epigenomics trial for PMA submission
Epi proColon®
pT0/Tis
pT1
pT2
pT3
pT4
pTx
unknown
TOTAL
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%)
2/3
8/14
10/15
28/38
9/9
4/4
13/18
74/101
73.3% (63.9-
Stage 0 / I
TOTAL
*…Staging information incomplete or unavailable
FIT
80.9%)
pT0/Tis
pT1
pT2
pT3
pT4
pTx
unknown
TOTAL
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%)
0/3
7/13
12/15
30/38
7/9
2/4
8/15
66/97
68.0% (58.2-
Stage 0 / I
TOTAL
*…Staging information incomplete or unavailable
31 | January 2015
76.5%)