3rd Quarter 2011
Promising early results reported….. Erik Christensen MD PhD, CEO Ruben Ekbråten, Finance Director
Slide 1
3rd Quarter 2011
Highlights Promising early results for blood based diagnostic test for early Alzheimer’s disease Study results to be presented at the CTAD meeting in November Progress in discussions with pharma and imaging companies Important US breast cancer gene expression and protein patent awarded
Slide 2
DiaGenic
Business strategy
Slide 3
The goal of the company is to take a leading position in the area of blood based diagnostics within selected CNS disease areas, through its ability to deliver precise, well-documented tools for early detection. This position will be achieved through pharmaceutical partnership, sale of commercial rights and co-development agreements with large pharmaceutical companies.
Driving multiple collaborative agreements forward Pharma 3-6 Imaging 2
Pharma 7 - 8
Expressed need for noninvasive MCI biomarker
Initial talks
DiaGenic identified as technology provider
Collaborative models discussed
Pharma 1-2 Imaging 1
Semi-exclusive & R&D collab. proposed
Milestone based payments
MCI biomarker ready for research and development
DiaGenic’s current status
Multiple interactions with pharma and imaging companies advancing according to plan Collaborative partner deals yielding: ‒
R&D technology fees
‒
Exclusive and non-exclusive licenses with milestone based payments
‒
Product revenue from companion diagnostics
Ferghana Partners appointed advisors on commercial transactions ‒
Slide 4
Searches for potential licensees of DiaGenic’s technology
The value chain
DiaGenic key competencies and assets
Discovery
• Technology knowhow (WGA, PCR, RT-PCR etc) • Bioinformatics • Biomarker identification • Broad clinical network (Europe and US) • World class biobank o Sample quality o Clinical documentation • CRO competence
Development
• State of the art know-how on biological and RNA sample handling • Assay design and optimization • QA • Multiplex RNA biomarkers • Software development • Multivariate data analysis
Documentation
• QMS compliant to ISO13485 • Laboratory and operator qualification • Instrument qualification and maintenance plan • Regulatory • IVDD compliance • Technology • Product verification • Product validation • Process verification • Process validation
Distribution
• Experienced management team • Distribution network Europe • Seeks US partners
DiaGenic has the experience on taking a product from discovery to market Slide 5
DiaGenic Core Assets,
World class biobank – a key differentiator
>7000 clinical and technical donors • • • •
AD studies 2500 donors PD studies 900 donors BC studies 3500 donors Technical studies 700 donors
– Samples from clinical sites in Europe and USA – Clinical and technical samples from all relevant clinical groups and from age matched healthy controls – Comprehensive clinical info following each sample (CRF) • Strict focus on sample and data quality and on biobank QA • Source verification of medical records by in-house monitors
– Ethical approval (REK) – PAXgene blood samples, 2-4 tubes per donor • An increasing business standard
Slide 6
DiaGenic Core Assets
Alzheimer’s Disease intellectual property
DiaGenic is ahead of competition in blood based AD diagnostics – US patents effectively protects our concept/technology/tools in using gene expression in AD including MCI • Others can not launch or perform market preparation trials in the US
– EU patents protects concept/technology/tools • Exception is continued use of exploratory technologies
– DiaGenic has freedom to operate in the field • Confirmed by 3rd parties
Slide 7
DiaGenic Core Assets,
First US breast cancer patent awarded
DiaGenic’s first patent on breast cancer in the US was accepted and notice of allowance was received on the United States family 3 patent application (11/628,300). –
–
–
During the third quarter the Australian family 3 patent application (2005250219) was granted. –
The allowed claims address diagnosis and monitoring of breast cancer using both gene expression and/or protein-based technologies. A broad range of gene probes, their functional equivalents and related protein targets are important for any multimodal diagnostic approach within the breast cancer field. The family 3 patent has earlier been granted in Europe and some other countries and they are valid until 2025.
The granted claims cover the use of some important gene sequences in blood sample for detection of cancer, among others, breast cancer.
These important patents substantially strengthen the breast cancer portfolio offering to potential external partners, executed by our consultants Ferghana Partners in the US.
Slide 8
Providing CNS biomarkers for clinical trials and prescription drug use Creating one–to–one relationships
Slide 9
Utility of blood based gene expression applications
A multitude of opportunities Application
Biomarkers
• Reduced size of clinical trials • Reduced cost of clinical trials
Early onset
• Selection tool which potentially can open up the market for expensive PIB PET imaging
PET enabler
• Classification of patients into different stages of the disease (e.g. drug efficacy measurement)
Staging
Prospective tool to differentiate expected speed of progression
• Classification of patients into different treatment schemes (e.g. dosage optimisation)
Progression
Select patients for therapy – Companion Diagnostics
• New therapies and expanded target populations
Companion Dx
Early disease and prodromal stages detection
• Speed up diagnosis and reduce cost of diagnosis • Enable earlier intervention
Prodromal
Patient selection tool for clinical trials Selection tool for expensive imaging procedures
Monitor disease progression
Slide 10
Utility
Development of blood based biomarkers for early stages of Alzheimer’s disease
Promising early results reported The objective was to identify gene expression patterns in blood from patients who progress from MCI to Alzheimer’s disease and on further disease progression Compared longitudinal changes in subjects with Alzheimer’s disease using; DiaGenic’s extended gene set from whole genome studies DiaGenic’s blood samples from our own clinical studies on MCI patients with validated expert consensus diagnosis Validated diagnostic technology platforms Next generation FDA compliant instrumentation (ABI ViiA7)
Proof of concept established Successfully delivered on time DiaGenic’s bioinformatic findings in the study verified Study results to be reported at the CTAD congress November 3-5th 2011
Slide 11
Validating the prototype biomarker for MCI converters
Taking MCI blood based biomarkers to FDA and beyond
Calibration
• Build solid calibration model from a larger data set • Larger cohort (n>100): • 50 MCI converters, including the 30 converters from pilot
Validation & Verification
• Independent validation cohort: • Aiming for 100 MCI converters • Additional DiaGenic samples from our ongoing clinical studies
Technical verification to prove robustness (like our earlier CE studies) (Samples: from DiaGenic biobank Controls+AD+MCI)
Submission
Validated biomarker qualified by FDA for investigational use
• Application in Phase III designs: Qualification of biomarker by FDA to support use of the biomarker during drug development (e.g. patient enrichment tools) Ready to use in 2 years - depending on sample availability - depending on regulatory process
PMA for IVD purposes
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Selection tools for Prodromal AD clinical trials
Example: 24-Month Clinical Trial Screening (up to 4 weeks)
Double-Blind treatment period (104 weeks)
Selection based upon: • Cognitive testing • MRI-brain
Subject continues in Extension
Compound X low dose QD (n=500) Compound X high dose QD (n=500)
and • MCItect
Placebo (n=500)
Standard of Care Interim analysis occurs when approximately 50% of the planned subjects in each treatment group have either completed or discontinued before 52w of treatment.
Visit 1 MRI Up to 4 w before Baseline
Week 0
Visit 2 Visit 3 Baseline Randomization PET CSF ADAS-cog ADCS-ADL
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Week 52
Visit 4 PK/PD CSF
Visit 5 PK/PD
Visit 6 PK/PD
Week 104
Visit 7 PK/PD
Visit 8 PK/PD
Visit 9 PK/PD End of treatment MRI PET CSF ADAS-cog ADCS-ADL
Selection tools for Prodromal AD clinical trials
Enrichment of MCI converters to AD Reducing sample size in MCI clinical trials
- Prodromal AD population, disease modification claim, primary outcome conversion to AD supported cognitive and functional testing (CDR). - The duration of prodromal AD trials is 2 years with annual conversion rate to AD of 12-14% - The theoretical example below is based on a Phase III trial scenario with 125 MCI converters per arm required to demonstrate a statistical difference between active treatment.
Cohort
Eligible for inclusion
MCI biomarker
Final inclusion
Estimated Conversion rate per arm
MCI
522
No
522
125 converters
MCItect enriched
685
Yes
255
125 converters
Enrichment of target population using MCItect – attracts pharma interest! Saving operational costs by 30%-40% by reducing study size Increasing trial success rate Improved ratio responders vs. non responders in treatment arms Homogenous study cohort Reduce attrition and non-compliance Slide 14
Companion diagnostic value proposition DiaGenic to develop key solutions for Amyloid PET producers
Characteristics
High end imaging diagnostics (PET)
Slide 15
Challenges
PET imaging diagnostics are the most accurate diagnostic tool for Alzheimer Disease
High cost per patient
Expensive equipment and procedures
Lack of objective selection criteria for reimbursements
Capacity constraints – limited no of scanners available due to cost
Value proposition
Blood-based diagnostics as a tool for preselecting patients for PET Increases hit-rates
Reduces capacity constraints Validates reimbursement
Dialogues with producers of amyloid PET tracers on R&D collaborations since last report have advanced – Collaborative study protocols developed, clinical site reviewed and funding scenarios pursued – Collaboration with PET producer will ensure traceability to AD amyloid diagnostics – PET producers see a potential use of blood based biomarkers together with high cost PET imaging as part of a multimodal approach of future AD management
3rd quarter 2011 financials
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Finance,
Income Income (thousand NOK)
• Operating revenue in Q3: recognition of milestone revenue from R&D collaboration with pharma and ADtect sales
2 800
2 400
2 000
1 600
1 200
800
400
Operating revenue Grants
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Q3 10
Q4 10
Q1 11
Q2 11
Q3 11
8
1 078
1 608
1 151
276
946
1 338
990
926
1 100
• Research grants in Q3 totalled NOK 1.1 million
Finance,
Profit & Loss Other Operating Cost
P&L 3Q
(thousand NOK)
(thousand NOK)
3Q ‘11
3Q ‘10
12 000
Revenue
276
8
10 000
COGS
133
178
8 000
9,611
9,445
6 000
(9,468)
(9,615)
4 000
Net finance
493
15
2 000
Net income
(8,975)
(9,600)
Other Operating Cost Operating loss
Salaries and related Depreciation and Write downs Other operating cost
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3Q 11 9,611
3Q 10 9,445
Q3 10
Q4 10
Q1 11
Q2 11
Q3 11
5 618
5 944
6 177
4 787
5 808
250
279
238
239
261
3 577
4 019
4 005
3 844
3 542
Finance,
Cash position Cash and Cash equivalents (million NOK)
• Long term financing secured in Q4 2010
120,0
• Cash balance end of September 2011: NOK 66 million
100,0 80,0
• Allotment of options in the quarter
60,0
40,0
Allotment of 420,000 options 4 years life and can be exercised after 3 years Strike NOK 6 per share
20,0 Cash
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Q3 '10 10,1
Q4 '10 98,8
Q1 '11 86,7
Q2 '11 76,1
Q3 '11 66,3
• No significant changes in cost level expected for Q4
Outlook & Summary
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3rd Quarter 2011
Outlook
Execute on the companion diagnostics initiative towards pharma and diagnostic companies Presentation of study results at the 4th Conference Clinical Trial on Alzheimer’s Disease in November 2011
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DiaGenic
Your preferred partner for gene expression profiling in blood Core competence and assets:
Delivers unique biomarkers in a well defined market characterized by a large unmet medical need World’s first company with – – –
Strong IP protection within blood based AD diagnosing and monitoring. –
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Broad claims protects against infringement.
Competence and experience in all aspects of product development from discovery to regulatory – – – –
Approved blood based test in AD diagnosis Prodromal AD proof of concept demonstrated Progression markers identified
Strong knowhow on technologies and platforms Strong competence in bioinformatics R&D collaboration with reputable university hospitals in US and Europe World Class Biobank
Good track record on receiving public grants Overall aim is to provide Companion Diagnostics tools for pharma and imaging companies
DiaGenic ASA Grenseveien 92, N-0663 Oslo, Norway Tel +47 23 24 89 50 Mail:
[email protected] www.diagenic.com
For more information, see www.diagenic.com
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Disclaimer This presentation includes forward-looking statements regarding DiaGenic ASA, including projections and expectations, which involve risk and uncertainty. Such statements are included without any guarantees to their future realization. Although DiaGenic
believes that the expectations regarding the Company
reflected in such forward-looking statements are based on reasonable assumptions, no assurance can be given that such projections will be fulfilled. Any such forward-looking statement must be considered along with knowledge that actual events or results may vary materially from such predictions due to, among other things, political, economic, financial or legal changes in the markets in which DiaGenic does business, and competitive developments or risks inherent to the Company’s business plans. Many of these factors are beyond DiaGenic’s ability to control or predict. Given these uncertainties, readers are cautioned not to place undue reliance on any forward-looking statements. The Company does not intend, and does not assume any obligation, to update the forward-looking statements included in this
presentation as of any date subsequent to the date hereof.
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