1. © BCC Research, 49-2 Walnut Park, Wellesley, MA 02481, USA (+1) 781-489-7301 www.bccresearch.com
The ‘PredTox’ Market Opportunity
In Vitro and In Silico Methods from
Predictive Toxicology to Help Develop
Precision Medicine
Webcast 3/11/14

2. 2
Information
∗ Webcast replay recording available for a limited time: http://bit.ly/predtox-webcast
∗ Related report: In Vitro Toxicity Testing: Technologies and Global Markets (report code
PHM017E). Search report code on our website: www.bccresearch.com.
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∗ Slides and presentation by Robert Hunter.
Bob Hunter
bob@predict-medicine.com
Linkedin: Robert G Hunter
Twitter: bobghunter

3. 3
Human Genome +10 yrs: Output?
Pharma pipelines: Disastrous!
Research concerns: Not reproducible!
Healthcare IT revolution: How?
Unmet needs impacted by PredTox

4. 4
From Basic/Common approaches:
∗ Analyzed Increased Outsourcing to CRO in Preclinical
∗ Analyzed Trends with Pharma R&D Budgets
To Wider/New approaches:
∗ Extrapolated Practices from Other Industries (Cosmetics,
Chemicals)
∗ Analyzed Overlap with Key Areas (Patient Care, Social)
PredTox analysis approach – wider lens
Result: longer-range view and actionable insights

5. 5
Opportunity fits key trends & <gaps>
Genomics
Proteomics
Metabolomics
Systems biology: Targets &
Pathways
*Key: Pipelines, Precision
med, Biomarkers
Patient empowerment – history in
DTC (direct-to-consumer) advertising
Ethics impacts: stem cells, 3Rs
Social networks
Nutrition and wellness
*Key: Predictive med - Personalized,
Preventive, Participatory
Health IT- Electronic Health
Records
Diagnostics
Big data
*Key: Predictive med –
Participation by MDs and Patients
Life sciences systems Patient care
Patient as consumer
<Biomarkers>
<EHR w/ Systems
biology>

6. 6
In Vitro and In Silico Methods for
Toxicology Revenue Growth
Estimated at 15% CAGR
2012 2017
$5 Billion $10 Billion

7. 7
∗ Focuses on drug approvals
∗ Safety/tox has long been key gate for drug development
∗ Adverse reactions have become key risk for FDA in making
approvals
∗ Has history of systems approach
∗ Linked to pathology – liver, kidney systems
∗ Linked to environmental chronic exposure – population studies,
“read-across” assessments
∗ Leveraged tissue engineering – skin, lung, blood-brain-barrier
∗ Leveraged cell imaging and other physiological “read-outs”
∗ Leveraged new test systems – stem cells, primary cells vs.
immortalized cell lines
Why PredTox perspective? (1 of 2)
Continued…

8. 8
∗ Track record of innovation
∗ Global research collaborations – EU/U.S./Japan
∗ Support increased testing of environmental chemicals
∗ Reduce reliance on animal testing – future: China, Russia, Brazil
∗ Cooperation with regulatory agencies: Cosmetics in EU 1990-2013,
REACH, Tox21
Why PredTox perspective? (2 of 2)

9. 9
Brief overview of landscape
∗ Safety
∗ Cardio: Adverse events led to FDA standards in 2000 > strong commercial
uptake
∗ Next?
∗ Toxicology: By tox domains = systems
∗ Skin and Eye
∗ Acute and Cytotoxicity
∗ Chronic and Repeated Dose
∗ Carcinogenicity and Genotoxicity
∗ Endocrine System – Estrogen receptor, thyroid
∗ Developmental and Reproductive (DART)
∗ ADME (Absorption, Distribution, Metabolic, Elimination): Metabolic focus

10. 10
Key – Track record of methods rigor
∗ Inspired by incumbent in vivo approaches
∗ Compared to in vivo, found animals don’t pertain to humans
∗ Developed rigorous methods and validation
approaches
∗ Developed structured approach corresponding to human
physiology
∗ Compared in vitro to other in vitro for consistency/reproducibility
∗ Incorporate in silico approaches as well
∗ Integrate mechanistic and predictive approaches

11. 11
PredTox In Vitro and In Silico Technologies (1 of 2)
Bioinformatics ($ 0.1 B)Cellomics ($ .75 B)Foundation ($ 1.4 B) Omics ($ 1.5 B)
Tissue engineering
Stem cells
Immortalized cells
Primary cells
Microplates, Readers
Gene Expression
Classifiers (Statistical)
Structure-
Centered Tools
FLIPr
Patch clamp, APC
HCS
Label-free
Transcriptomics
Proteomics
Metabolomics
Automation
Liquid handling
Reagents
•Microarrays
-miRNA
•Next-Gen Screening
•Mass Spec
•Chromatography
•NMR
•Electrophysiology
•Ion channel
Flow cytometry
•Imaging & Analysis Functional Classifiers
(Pathways & Networks)
•Estrogen- breast cancer
•DILI
•Metabolic vs. signaling, etc.
•Multi-pathway learning
•Chemoinformatic tools
•“Pathway barcoding”
Model-based Drug
Development
•QSARs
+ Cell-based Assays
(See Figure 2)
Continued…

12. 12
Cell-based Assays
In Vitro Assays
Biochemical Assays
Secondary messenger Cell integrityFunctional mechanism
• Receptor binding
• Enzyme inhibition
• Reporter gene
(transcription- ex.
GPCRs, over 1000)
• Other
• Membrane fns (ion
channel)
• Cell proliferation
• Cell permeability
(cytotoxicity)
• Cell stability
Tissue models
• Animal
• Human
2D 3D formats>
96 > 384 > 1536 well
- $ 1.0 B kits
- $ 1.4 B reagents
Reagents
PredTox In Vitro and In Silico Technologies (2 of 2)

13. 13
Texture of Report (1 of 2)
Applications and systems context
• Historical perspective
Breakout Business Models
• Biomarkers
• Drug repositioning
• Precision medicine applications
(biosensors, organ on a chip,
systems biology)
Org/Company input, focused on:
• Adoption of in vitro and in silico
• Innovation for in vitro and in silico
Continued…
In Vitro Toxicity Testing: Technologies and Global Markets (report code PHM017E).

14. 14
Texture of Report (2 of 2)
Global drivers and trends
• Global pharma
• Cosmetics and Chemicals influence
• China
• Other BRIC
• Pharmerging
Open revenue forecast models
• Six layers of revenue models, with
individual drivers
• Annotated drivers, variables,
calculations
In Vitro Toxicity Testing: Technologies and Global Markets (report code PHM017E).

15. 15
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