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

2 nd 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 3

Topics • Company Overview • Market Need • Vortex Solution • Competitive Landscape • Soft Launch – AACR • Summary 4

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) 5

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 6

Leadership team Bob Gene Steve Elodie Walther, MBA Englert Crouse, MBA Sollier-Christen, Ph.D CEO CSO & VP R&D VP Manufacturing CCO • President Chiron/Novartis • Ph.D Microfluidics • SVP FreeSlate • Abbott Laboratories Diagnostics • UCLA, Stanford • Director BioRad • Biomedtrics • CEO Gentura Dx • Invitrogen • Pelikan Technologies • Expertise in microdevices • Deputy Director Bill & Melinda • Biosource for blood sample prep. Gates Foundation 7

CTC potential $22B Clinical market by 2020 * $8B Research market by 2020 + Drug Development Cancer Diagnostic Cancer Monitoring Cancer Research Personalized Medicine CTC Analysis (Viability, Purity, CTC Enumeration (Efficiency, Cost) Automation) • Early Stage Diagnosis • Cancer Research • Patient Prognosis • Patient Prognosis • Treatment Outcome Monitoring • Personalized Therapy • Residual Disease • Drug Development & Testing • *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) 8

Vortex captures circulating tumor cells… Circulating tumor cells (CTCs) detach from a solid Challenges to CTC isolation tumor and enter the blood stream playing a crucial role in • Extremely rare : 1-100 CTCs per mL of blood cancer metastasis. • Other cells: millions of WBCs and billions of RBCs per mL of blood 9

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 10

The right tools support achieving clinical validation CTCs high in purity CTC collection supporting sensitive, unbiased by molecular accurate analysis characteristics 10-50% of CTCs are EPCAM - Clinical Success Minimal manual Unaltered by labels or intervention and reagents and easily integration with assays collected 11

Introducing VTX-1 Isolate intact, label free CTCs from whole blood in <1 hour 12

VTX-1 Technology Cell trapping enabled by inertial lift forces and microscale vortices. [1] Sollier, E. et al. Lab on a chip 14, 63–77 (2014). [2] Che, J. et al., Oncotarget (2016). 13

Technology Animation 14

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). Size Based Microfluidics Affinity Based Active Forces Filtration Imaging 15

Critical market requirements to achieve leadership 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 for 7.5 mLs of blood processed for 1 Processing time 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. 16

VTX-1 Workflow 17

Genomic Analysis Genomic analysis is expected to be the primary method of liquid biopsy characterization and cancer diagnosis. 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 18

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. 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 19

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. Vortex’s Value Vortex reproducibly enriches CTCs with high purity, capturing CTCs directly on 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 20

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. 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 21

VTX-1 Advantages Other CTC Enrichment Systems Method of CTC Non-contact, label free Affinity or size based filters separation micro-vortices Upfront sample prep None required RBC lysis, buffy coat collection, affinity incubation Sample processing time 1 – 1.5 hour 2-6 hours after pre- for 7.5mL blood processing WBCs/mL of blood 30-150 250-10,000 (purity) Integration with assays Cells are collected in Cells are difficult to retrieve containers of choice in and require cell 300 uL concentration and cell transfers 22

Broad Interest in Vortex 23

Scientists/Researchers like the approach Rapid No Pre- Processing Processing Time Scientists like Simplicity = High Purity Vortex’s Reproducible technology Easy Flexibility of Analytics the System 24

CTCs complement ctDNA analysis ctDNA Provides Tremendous Value High sensitivity for most cancers with large amounts in the blood Simplicity with direct DNA extraction from the blood However, CTCs Offer Many Advantages Allows for Provides Real Provides Protein, RNA Time Analysis Heterogeneity and Genomic of Disease of Disease Analysis Cell Mechanisms Whole Culture/CDX of Metastasis Genome Allows for Can Be Analysis is Therapy Explored Possible Testing 25

Trends are driving renewed interest in CTCs 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 26

VTX-1 Commercial Launch 1H 2017 Key Dates Soft Research Market Launch, April 2016 Global Commercial Launch, 1H 2017 Instrument price pricing study to determine pricing Cartridge price pricing study to determine pricing Target GP % Key Gener8 selected as contract manufacturer for Partnerships VTX-1 Sales Strategy Direct to user (US), academic and industry Internationally (TBD) 27