Organovo Investor Presentationorganovo.com/.../2015/11/Organovo-Investor-Presentation-1115.pdf ·...

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Organovo Investor Presentation November 2015

Transcript of Organovo Investor Presentationorganovo.com/.../2015/11/Organovo-Investor-Presentation-1115.pdf ·...

Organovo Investor Presentation

November 2015

This presentation contains statements about future events and expectations known as “forward-looking statements” within the meaning of Section 27A of the Securities Act of 1933, as amended (the “Securities Act”), and Section 21E of the Securities Exchange Act of 1934, as amended (the “Exchange Act”). The Company has based these forward-looking statements on its current expectations and the information currently available to it, but any forward-looking statements are subject to a number of risks and uncertainties. The factors that could cause the Company’s actual future results to differ materially from its current expectations include, but are not limited to, risks and uncertainties relating to the Company's ability to develop, market and sell products and services based on its technology; the expected benefits and efficacy of the Company's products, services and technology; the market acceptance of the Company’s products and services; the Company’s ability to generate revenue and control its operating losses; the validity of the Company’s intellectual property rights and the ability to protect those rights; and the Company's ability to implement and achieve its business, research, product development, regulatory approval, marketing and distribution plans and strategies. These and other factors are identified and described in more detail in the Company’s filings with the Securities and Exchange Commission (“the SEC”), including those factors listed under the caption "Risk Factors" in the Company’s Form 10-K for the year ended March 31, 2015, filed with the SEC on June 9, 2015, as well as other filings Organovo makes with the SEC from time to time.

Readers are cautioned not to place undue reliance on forward-looking statements, which speak only as of the date of this presentation. Except as required by applicable law, we do not intend to update any of the forward-looking statements to conform these statements to reflect actual results, later events or circumstances or to reflect the occurrence of unanticipated events.

Forward-Looking Statements

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In addition to disclosing financial results that are determined in accordance with U.S. GAAP, the Company providesnet cash utilization as a supplemental measure to help investors evaluate the Company’s fundamental operationalperformance. The Company defines net cash utilization as the change in the amount of cash and cash equivalentsat the beginning of the reporting period as compared to the end of the reporting period, excluding financing activities(which would include proceeds from the sale of common stock and the exercise of warrants and stock options). Netcash utilization is an operational measure that should be considered in addition to the Company’s results prepared inaccordance with U.S. GAAP. This operational measure should not be considered as a substitute for, or superior to,U.S. GAAP results. The Company believes net cash utilization is a relevant and useful operational measure becauseit provides information regarding its cash utilization rate. Management uses net cash utilization to manage thebusiness, including in preparing its annual operating budget, financial projections and compensation plans. TheCompany believes that net cash utilization is also useful to investors because similar measures are frequently usedby securities analysts, investors and other interested parties in their evaluation of companies in a similar stage ofproduct development and commercialization. However, there is no standardized measurement of net cash utilization,and net cash utilization as the Company presents it may not be comparable with similarly titled operational measuresused by other companies. Due to these limitations, investors should not view net cash utilization in isolation, butshould also consider other measurements, such as cash used in operating activities and revenues, to evaluate theCompany’s operating performance.

Supplemental Financial Measures

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Investment & Business Highlights

Organovo – A Bright Future

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Investment Highlights

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Technology leadership – Strong IP portfolio

Attractive and growing markets – Critical unmet needs

Growing revenue – Building product and service platform

Favorable competitive dynamics – First mover advantage

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Organovo Overview• Founded in 2007 and listed on

the NYSE MKT in 2013• Proprietary 3D bioprinting

technology invented at University of Missouri with $5 million NSF grant; continued engineering development at Organovo

• Core business focused on recreating 100% cellular native tissue architecture for in vitroand in vivo applications

• Key customers/partners include major pharma and academic institutions

2012

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• Enable better predictive, physiologically relevant in vitro modeling• Fill the gap between in vitro and in vivo preclinical research• De-risk total discovery program costs by enabling decision points further upstream• Potential to avoid ~$1 billion in development costs and timeline delays for a single drug

with better information about which drugs to advance (efficacy, toxicology)• Provide disease modeling applications across the pre-clinical drug discovery workflow

Addressing the Gaps in Pharmaceutical Discovery

Target Discovery

Target Validation

Lead Optimization

Pre-Clinical non-GLP Pre-Clinical GLP Clinical Trials

In vitro In vivo

Gap

Program Costs ->$ $$ $$ $$$ $$$$ $$$$$

Human 3D toxicity / efficacy models

Human 3D disease models

10-12 Years, ~$1.2 billion

• In 2012, there were 10 late stage clinical trial failures resulting in $7 billion-$10 billion in wasted costs

• Of 126 phase 3 clinical trial failures from 1990-2010, 73% were for either toxicity or lack of efficacy

• Of 39 drugs withdrawn from the market from 1990-2010, 26% involved hepatotoxicity and 33% were CV toxicity

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DRUGS WITHDRAWN DUE TO TOXICITY (1990-2010)

N=39

PHASE 3 FAILURES (1990-2010)

N=126

Lack of Efficacy,

45%Safety/Toxicity,

28%

Other, 27%

Hepatoxicity, 26%

CV Toxicity,

33%

Other, 41%

Critical Unmet Need for More Predictive Preclinical Tissue Models

References: FDA.gov; Pharmaceutical Research Manufacturers of America Profile (2011); EvaluatePharma; Parexel Sourcebook (2012); CDER Report to Nation; Tufts Center for Drug Discovery

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U.S. Transplants 1991 – 2013(1)

(1) U.S. Department Health & Human Services

Critical Unmet Need for Tissues that Replace or Repair Organ Function• Engineered tissues offer an

answer to dramatic supply issues that are a constant challenge in transplant medicine ($7 billion U.S. transplantation market)

• Tissue engineered organ and tissue replacements could address issues associated with conventional transplantation (graft vs. host disease, size mismatch)

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Favorable Markets & Competitive Dynamics

(1) Pharmaceutical Research Manufacturers of America Profile (2011)(2) Company data and projections

• $1.3 billion• 6,500 annual programs

• Gaps in 2D and animal models

• Unsatisfying Next Gen models

• Each program needs project run every 9 months

• Average contract is $150K

• $2.1 billion• 6,500 annual programs

• Gaps in 2D and animalmodels

• Pricing superior to liver tissue

• Average contract expected to be $250K

• >$50 billion in annual R&D; new drug ~$1.2 billion and 10-12 years

• Gaps in 2D and animal models

• Enable discovery in areas with roadblocks

• Royalty opportunity

• >100,000 U.S. patients on transplant waiting list

• ~30,000 transplants annually

• N/A• Partner with academia• FDA clinical trials for

marketed product

Market Size(1) CompetitionOrganovo Position &

Growth Strategy(2)

Land

M2M

Liver Toxicology

Kidney Toxicology

Efficacy Models

Therapeutic Tissues

NovoGen Bioprinting Process

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Superior predictive solution that recreates 3D native tissue architecture

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Spheroids (ex.InSphero) Scaffold + Cells

Micro-patterned cultures (ex. Hepregen)

Organovo Bioprinted Tissue

Tissue-like cellular density ✗ ✗ ✗ ✔

True 3D; >250m in x, y, and z axes ✔ ✔ ✗ ✔

Multiple tissue-specific cell types ✔ ✔ ✔ ✔

Spatially-controlled cell compartments ✗ ✗ ✔ ✔

In-vivo like tissue micro-architecture ✗ ✗ ✗ ✔

Reproducible; compatible with automated fabrication ✔ ✗ ✔ ✔

Easy-to-handle format ✗ ✔ ✔ ✔

Technology Leadership – Comparing In Vivo-Like Form & Function

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Strong IP Portfolio• Own or exclusively license 20

patents worldwide; 80 patent applications pending

• Exclusive licenses to foundational patents with early priority dates from three major U.S. universities

• Patent filings relate to bioprinting technology and its various uses in tissue creation, use in drug discovery and specific tissue constructs

• Rich portfolio of trade secrets and “know how”

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Best-In-Class Customers & Partners

Platform Technology – Growth OpportunitiesTissue

Research Programs

Preclinical Safety(Product/Service)

Efficacy Models(Partnerships)

Simple Tissues for Therapy

(Clinical)

Liver ✔ (LaunchedC4Q14) ✔ ✔

Lung ✔ ✔

Kidney ✔ (Expected launch C3Q16) ✔ ✔

Cancer ✔

Bone ✔ ✔

Blood Vessel ✔ ✔

Heart ✔ ✔ ✔

Skin ✔ ✔ ✔

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Key Focus Areas

exVive3DTM Liver Tissue - Excellent Matching of Human Liver Biology

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System Troglitazone(1) Toxic (X)(2) Methotrexate2D hepatocytes No toxicity seen No toxicity seen No fibrosis seen

Rat models No toxicity seen No toxicity seen No fibrosis seenHuman results Toxic Toxic Chronic fibrosis

exVive3D Liver Toxic Toxic Chronic fibrosis

System Liver Enzymes Longevity Cholesterol2D hepatocytes Poorly conserved 2 days No

exVive3D Liver Well conserved 42 days+ Yes

Pfizer paid $750 million in legal costs and $150 million to remove drug from market

(1) FDA Final Report (2000): Executive Summary, Liver Failure Risk with Troglitazone (Rezulin)(2) Partnership with Roche; proof of concept study with known DILI drug

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N=10 per group

* = p< 0.05** = p< 0.01

*** = p< 0.001**** = p< 0.0001

One way ANOVAGrubbs outlier analysis

exVive3D Liver Tissue – Proof of ConceptDemonstrated to reveal toxicity

• 7-day daily dosing• With known drug-induced liver

injury (DILI) drug and close analogue

• Compared biochemical markers, gene expression, cytokines, histology

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exVive3D Kidney Tissue DevelopmentPricing expected to be superior to liver tissue; no existing alternatives

• Scientific proof of concept – April 2015 ✔• Functional validation – October 2015 ✔• Beta testing – C2Q16• Initiation of contracting – C3Q16

TissueResearch Programs

Preclinical Safety (Product/Service)

Efficacy Models(Partnerships)

Simple Tissues for Therapy

(Clinical)

Liver ✔ (LaunchedC4Q14) ✔ ✔

Lung ✔ ✔

Kidney ✔ (Expected launch C3Q16) ✔ ✔

Cancer ✔

Bone ✔ ✔

Blood Vessel ✔ ✔

Heart ✔ ✔ ✔

Skin ✔ ✔ ✔

Platform Technology – Growth Opportunities

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Key Focus Areas

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(1) U.S. Department of Health & Human Services(2) Dr. Francis Collins, Director of the National Institutes of Health

Efficacy Models – HighlightsRoyalty and licensing opportunities for successful drugs

• A new drug costs ~$1.2 billion and takes 10-12 years to develop(1)

• Significantly impact cost, predictability and speed of drug discovery

• High failure rates in clinical trials due to failure to predict efficacy

• Many areas where animal models are known to be poor predictors

– “The use of animal models for therapeutic development and target validation is time consuming, costly and may not accurately predict efficacy in humans.”(2)

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Efficacy Models – L’Oreal

• ~$30 billion annual sales; $1.1 billion R&D budget in 2014

• Second-stage contract; first contract in 2013 to prototype 3D skin models

• Multiple potential revenue sources –commercial supplier for bioprinted skin tissue, co-marketing skin model, royalty opportunity on cosmetic products tested in 3D skin model

• Toxicology testing with skin tissues

3D skin model partnership with world leader in cosmetics

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Efficacy Models – Cancer Opportunity

• Premier academic and industry partnerships

• Breast cancer initial model already established – bioprinted with defined multi-cellular composition and architecture

• Clear differential outcomes vs. 2D cultures

• Multiple solid tumor types under investigation

• Active business development pipeline in oncology

Move upstream in discovery efforts with strong royalty opportunity

• Recent positive data from The Hamner Institutes showing robust liver fibrosis model validation

• Non-alcoholic steatohepatitis (NASH/fatty liver disease) is a significant area of current research in pharma drug discovery

• Cholesterol is not seen in other models and one can potentially modify its expression with high specificity

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Efficacy Models – Liver TissueexVive3D Liver Tissue can also be leveraged for drug discovery efforts

Healthy ExVive 3D Liver Tissue

Fibrotic ExVive 3D Liver Tissue After TGF-B Exposure

TissueResearch Programs

Preclinical Safety(Product/Service)

Efficacy Models(Partnerships)

Simple Tissues for Therapy

(Clinical)

Liver ✔ (LaunchedC4Q14) ✔ ✔

Lung ✔ ✔

Kidney ✔ (Expected launch C3Q16) ✔ ✔

Cancer ✔

Bone ✔ ✔

Blood Vessel ✔ ✔

Heart ✔ ✔ ✔

Skin ✔ ✔ ✔

Platform Technology – Growth Opportunities

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Key Focus Areas

• Creating tissue replacement products for surgical implantation

• Critical unmet need with significant challenges

• Multiple tissue types (ex. liver patch) being evaluated – solid early results in animal models

• Early feedback from key constituents suggests focus on areas of high unmet clinical need where a tissue is the best hope for treatment

• Aiming for IND submission in at least one tissue in 3-4 years

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Simple Tissue for TherapyPotential to revolutionize therapeutic applications – “Tissue-On-Demand”

Financial Overview

Key Financial Metrics F1H16 (9/30/15) ($M)

Product & Service Revenue $0.4

Total Revenue $0.6

Cash & Cash Equivalents $76.9

Key Financial Metrics & FY16 Outlook

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FY16 Outlook(1)

Total Revenue Recognized From Previously Reported Contract Bookings(2)

$1.2 million - $1.4 million

Net Cash Utilization $32 million - $36 million

(1) Guidance speaks only as of the date it was originally provided (November 9, 2015). Inclusion of guidance herein should not be interpreted as a re-affirmation by Organovo of its guidance. Organovo undertakes no obligation to update its guidance after the date it was originally provided.

(2) In fiscal 2016, the Company expects to recognize between $1.2 million and $1.4 million of its approximately $2 million in contract bookings for its exVive3D Liver Tissue previously reported for the period from April 1, 2014 to June 9, 2015.

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Revenue Recognition Summary

Customer engagement and work planning (4-6 months)

Completion of work (4-5 months for single stage contract)

Revenue recognition (8-11 months after initial customer contact)

Accelerating Breakthrough Research…

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Technology leadership – Strong IP portfolio

Attractive and growing markets – Critical unmet needs

Growing revenue – Building product and service platform

Favorable competitive dynamics – First mover advantage

Organovo Investor Presentation

November 2015

Appendix

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• The automated fabrication of a tissue through the spatially-controlled deposition of cells and/or cell-containing materials in defined, user-controlled geometric patterns, wherein the resulting multi-cellular tissue is viable, three-dimensional and mimics key aspects of native tissue architecture and/or function

• Our finished tissues are scaffold-free, comprised only of relevant tissue cell types and the extracellular matrix the cells produce

What Is Bioprinting?