Working to beat cancer Capital Markets Day June 14, 2016 2 A next - - PowerPoint PPT Presentation

2nd Annual Capital Markets Day Gene Walther

CEO, Vortex BioSciences, Inc.

Working to beat cancer

Capital Markets Day

June 14, 2016

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A next generation CTC enrichment technology

Topics

• Company Overview
• Market Need
• Vortex Solution
• Competitive Landscape
• Soft Launch – AACR
• Summary

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Executive Summary

• Research tools and diagnostics company developing a novel liquid

biopsy technology that could revolutionize cancer research, drug development, diagnosis, monitoring and treatment

• $22B liquid biopsy diagnostic market opportunity*
• >$8B cancer research market opportunity**
• Patented microfluidic technology yields circulating tumor cells (CTCs)
• Vortex outperforms competitors in key fields – purity (specificity) and

collection efficiency (sensitivity) based on published data

• Significant growth opportunities as the product pipeline expands from

research to also providing clinical information from CTCs

• *J.P. Morgan Liquid Biopsy Report – May 27, 2015
• **US NIH spending c$8.2bn on cancer research (source: NIH, 2016), the UK £500m (source: Cancer Research UK)

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The Company

• Formed in 2012 based on research done by PECASE (Presidential

Early Career Award for Science and Engineering) winner Dino Di Carlo at UCLA

• Late stage development phase
• Exciting R&D presents near-term growth opportunities in the rapidly

growing liquid biopsy market

• Building a pipeline of partners and potential customers to support

Research Use Only sales and clinical research

• Experienced leadership team in place to guide development and

delivery of first commercial product in early 2017

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Leadership team

Elodie Sollier-Christen, Ph.D

CSO & VP R&D

• President Chiron/Novartis

Diagnostics

• CEO Gentura Dx
• Deputy Director Bill & Melinda

Gates Foundation

• Ph.D Microfluidics
• UCLA, Stanford
• Expertise in microdevices

for blood sample prep.

Gene Walther, MBA

CEO

Steve Crouse, MBA

CCO

• SVP FreeSlate
• Director BioRad
• Invitrogen
• Biosource

Bob Englert

VP Manufacturing

• Abbott Laboratories
• Biomedtrics
• Pelikan Technologies

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CTC Analysis (Viability, Purity, Automation)

• Cancer Research
• Patient Prognosis
• Personalized Therapy
• Drug Development & Testing

CTC Enumeration (Efficiency, Cost)

• Early Stage Diagnosis
• Patient Prognosis
• Treatment Outcome Monitoring
• Residual Disease
• *JP Morgan liquid biopsy report – May 27, 2015
• +US NIH spending c$8.2bn on cancer research (source: NIH, 2016), the UK £500m (source: Cancer Research UK)

CTC potential

$22B Clinical market by 2020*

Cancer Research Personalized Medicine Drug Development Cancer Diagnostic Cancer Monitoring

$8B Research market by 2020+

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Circulating tumor cells (CTCs) detach from a solid tumor and enter the blood stream playing a crucial role in cancer metastasis. Challenges to CTC isolation

• Extremely rare: 1-100 CTCs per mL of blood
• Other cells: millions of WBCs and billions of RBCs

per mL of blood

Vortex captures circulating tumor cells…

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The promise has not been realized yet

• CTC enrichment technologies available today are

limited by

– Complex sample processing – Long processing time – Reliance on cell surface proteins for isolation – Low sample purity – Dilute output volumes that require additional cell concentration steps. – Poor integration with downstream analytics

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The right tools support achieving clinical validation

CTCs high in purity supporting sensitive, accurate analysis Unaltered by labels or reagents and easily collected CTC collection unbiased by molecular characteristics

Clinical Success

Minimal manual intervention and integration with assays

10-50% of CTCs are EPCAM -

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Introducing VTX-1

Isolate intact, label free CTCs from whole blood in <1 hour

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[1] Sollier, E. et al. Lab on a chip 14, 63–77 (2014). [2] Che, J. et al., Oncotarget (2016).

Cell trapping enabled by inertial lift forces and microscale vortices.

VTX-1 Technology

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Technology Animation

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The CTC market is crowded

• To our knowledge, as for today: 34 CTC companies.
• Principle: affinity-based (17), Imaging (7), filtration (5),

active forces (1), size based microfluidics (4).

Affinity Based Imaging Filtration Active Forces Size Based Microfluidics

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Critical market requirements to achieve leadership

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Category Requirement Cell recovery >60% Cell recovery variability Standard deviation of <10% Cell purity performance The average WBCs/mL will be <80 WBCs/mL Processing time Processing time for 7.5 mLs of blood processed for 1 cycle will be <1.5 hours. For 3 cycles it will be < 3 hours. Sample volume 7.5 mLs of whole blood with one consumable.

VTX-1 Workflow

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Genomic Analysis

Genomic analysis is expected to be the primary method of liquid biopsy characterization and cancer diagnosis.

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Vortex’s Value

Vortex rapidly enriches CTCs with high efficiency and purity, capturing intact CTCs for immediate downstream genomic analysis.

• Higher DNA/RNA yields can be extracted from intact cells vs fixed cells

increasing sensitivity

• High CTC purity enables sensitive and specific assays
• Enrichment in <1 hour ensures RNA expression has not changed

Cell Culture/CDX/Live Cell Assays

Cell culture and CDX models allow scientists and clinicians to explore potential treatments before administering them to the patient.

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Vortex’s Value

Vortex enriches intact CTCs with high efficiency, enabling immediate cell culturing or CDX creation.

• The fast, gentle process means cells are viable and unharmed.
• Cells can be captured directly into a petri dish for culturing
• Vortex can easily handle small sample volumes allowing for mouse blood

processing for monitoring CDX and PDX models

Enumeration/IHC/FISH

CTC enumeration has been shown to have both prognostic and diagnostic potential. IHC and FISH align well with some existing companion diagnostics and will continue to be a valuable tool.

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Vortex’s Value

Vortex reproducibly enriches CTCs with high purity, capturing CTCs directly

• n a slide chamber for downstream processing.
• The elegant process provides the reproducibility needed for clinical

decisions

• The rapid, gentle process results in unchanged protein expression
• A 300 µL capture volume makes FISH or IHC protocols simple

Protein Analysis

Protein expression levels can be used as a stratifying assay for many potential treatments. While genomic analysis is favored, actual protein expression will be most appropriate for some drug targets.

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Vortex’s Value

Vortex rapidly enriches CTCs with high efficiency and high purity, capturing CTCs directly into microwells for downstream protein analysis.

• The fast, gentle process means protein expression levels are unchanged
• The high purity of the CTCs improves the accuracy and sensitivity of

protein assays

VTX-1 Advantages

Other CTC Enrichment Systems Method of CTC separation Non-contact, label free micro-vortices Affinity or size based filters Upfront sample prep None required RBC lysis, buffy coat collection, affinity incubation Sample processing time for 7.5mL blood 1 – 1.5 hour 2-6 hours after pre- processing WBCs/mL of blood (purity) 30-150 250-10,000 Integration with assays Cells are collected in containers of choice in 300 uL Cells are difficult to retrieve and require cell concentration and cell transfers

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Broad Interest in Vortex

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Scientists/Researchers like the approach

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Scientists like Vortex’s technology

No Pre- Processing Rapid Processing Time High Purity

Simplicity = Reproducible

Flexibility of the System Easy Analytics

CTCs complement ctDNA analysis

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High sensitivity for most cancers with large amounts in the blood Simplicity with direct DNA extraction from the blood ctDNA Provides Tremendous Value However, CTCs Offer Many Advantages Allows for Protein, RNA and Genomic Analysis Whole Genome Analysis is Possible Provides Heterogeneity

• f Disease

Cell Culture/CDX Allows for Therapy Testing Mechanisms

• f Metastasis

Can Be Explored Provides Real Time Analysis

• f Disease

Trends are driving renewed interest in CTCs

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Mutations found in the primary and secondary tumors sometimes show up in only CTC or ctDNA Stopping metastasis (and understanding mechanisms of metastasis) vs killing the tumor is becoming a key area for future therapeutics Interest in heterogeneity of disease and how cancer evolves Interest in RNA expression as an indicator of disease Interest in CDX and PDX models of disease Interest in immune checkpoints and ongoing identification of neoantigens for immunotherapy

VTX-1 Commercial Launch 1H 2017

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Key Dates Soft Research Market Launch, April 2016 Global Commercial Launch, 1H 2017 Instrument price pricing study to determine pricing Cartridge price Target GP % pricing study to determine pricing Key Partnerships Gener8 selected as contract manufacturer for VTX-1 Sales Strategy Direct to user (US), academic and industry Internationally (TBD)

EPFL SRA

CTC Enrichment Platforms VTX-n

2016 2017 2018 2019 2020 2020+ VTX-1

Capture of intact CTCs Flexible CTC Collection

VTX-2

Capture of intact CTCs Label-free Enumeration

VTX-3

Capture of intact CTCs Label-free Enumeration Single Cell Release & Encapsulation OR Electroporation System Integration RUO Commercial Product SRA - EPFL RUO Commercial Product Vortex Development

SRA - UCLA Vortex HQ

Vortex Development SRA - UCLA

Product

Clinical Trial

Commercial Product

Product Timeline

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Summary

• Vortex Biosciences is well positioned to capture a

significant share of the evolving liquid biopsy research market

• Strong interest from a broad selection of customers
• CTCs are complementary with ctDNA and have large

potential in the diagnostic market

• Investing in next generation technologies to enter the

clinical applications market within the next 2 – 3 years

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The leader in CTC enrichment technologies, a critical component of Moonshot 2020

Questions?

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