Open Innovation: An Imperative for the Pharmaceutical Industry. Berkeley Innovation Forum

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Open Innovation: An Imperative for the Pharmaceutical Industry Berkeley Innovation Forum October 28-29, 2010 Minneapolis, MN Ted Torphy, Ph.D. Vice President & Head External Innovation Research Capabilities Johnson & Johnson Pharmaceutical R&D, L.L.C.

Our Industry Is Being Transformed Spiraling R&D costs coupled with decreased productivity Ever increasing regulatory requirements Reimbursement driven by medical and economic outcomes Expectation of personalized medicine Proliferation and redistribution of healthcare outcomes information 2

Productivity of the Pharmaceutical Industry Sources: FDA/CDER, PhRMA, PricewaterhouseCoopers Note: R&D spending from non-phrma companies not available Drugs Approved 50 60 R&D Expenditures Drug Approvals 50 40 40 30 30 20 20 10 10 R&D Spending (US$ Billions) 1995 2000 2005 3

Pharmaceutical R&D Is a Long Process Identify molecular target Animal Testing Demonstrate efficacy Demonstrate safety and efficacy Basic Research Drug Discovery Pre- Clinical I IIa IIb III FDA Review Synthesize drug candidate Tolerability in humans Identify appropriate dose Apply for marketing approval 0 1 2 3 4 5 6 7 Years 8 9 10 11 12 13 Developing a new drug is a lengthy process Product cycle times are extremely long The 20-year limit on patent protection means there is a limited number of years during which a marketed product can generate an attractive ROI The cost of putting a new drug on the market is $0.8 to $1.3 bn 1 1 Adams C, Brantner V (2006). "Estimating the cost of new drug development: is it really 802 million dollars?". Health Aff 25 (2): 420 8 4

Pharmaceutical R&D Is High Risk Cumulative Probability of Success through Key Stagegates 1 Discovery 2 Pre- Clinical I IIa IIb III FDA Review 0 1 2 3 4 5 6 7 8 9 11 10 12 13 Years Developing a new drug is a risky process When a new drug discovery program is initiated the overall probability of launching a new product is 1.5% 10,000 to 30,000 compounds must be synthesized for every one that reaches the market 1 CMR benchmarks used to calculate risk-adjusted values at various stages 2 Internal estimate of 30% PoS 5

R&D Risk Profile Probability of Success by Stagegate 1 1.0 0.5 0 0 35-40% of R&D investment is made in advancing products through Ph IIa Until clinical proof of concept is demonstrated, the probability of success is low Drug Discovery 2 Pre- Clinical I IIa 1 2 3 4 5 6 7 8 9 11 10 12 Years IIb III FDA Review 13 1 CMR benchmarks used to calculate risk-adjusted values at various stages 2 Internal estimate of 30% PoS 6

Pharmaceutical R&D Current State R&D productivity is decreasing and is not sufficient to drive future growth of the industry Industry consolidation is leading to fewer Large Pharma R&D organizations and the remaining R&D organizations are shedding infrastructure Increasingly, venture capital backed biotech organizations are the source of new products but the traditional venture capital model of creating, funding, and monetizing biotech companies is faltering Large Pharma wants to acquire products, not companies Building fully integrated Biotech companies is not a capital efficient way of generating new products, making product acquisition by Large Pharma prohibitively expensive There is insufficient funding for innovative early-stage product opportunities emerging from academia 7

Our Ecosystem Is Interdependent and Fragile Biotech Venture Capital Pharma CRO s Academia Image from the Huffington Post 8

Improving R&D Productivity Without a new R&D business model, our industry will not supply an adequate number of innovative new products to drive growth and increase shareholder value Pharmaceutical R&D has become far to costly and complex for any individual company to sustain its business by operating under our traditional model of vertical integration Applying the principles of Open Innovation to align the incentives of all constituents of the ecosystem is essential to creating a new, capital efficient and productive model of pharmaceutical R&D 9

The Open Innovation Mindset 1 Closed Innovation We must discover, develop, manufacture and distribute innovations ourselves in a vertically integrated model The requisite expertise in R&D must exist inside of our company If we invent and fund everything internally we will win We must control and conceal our innovation processes, technologies and tools, so that our competitors don't profit from our ideas Open Innovation Enormous value can be unlocked from external R&D and innovation networks Pharmaceutical R&D has become far too complex for us to employ all the expertise needed Creating a better business model for partnered innovation can trump internal invention We will profit from others' use of our innovations and knowledge, and we will leverage others' IP whenever it advances our own business model 1 Adapted from Open Innovation, by Henry Chesbrough Harvard Business School Press, 2006 10

Vertical Integration of Large Pharma Is it Time to Disintegrate? Vertical Integration A characteristic of industries in which firms attempt to own and control all aspects of making, selling, and delivering the product or service that constitutes their core business Examples: Ma Bell, Oil, Steel, Large Pharma Vertical Disintegration A characteristic of industries in which there is an advantage for companies to access from independent suppliers some or all of the materials, intellectual capital, and/or human resources that are essential to delivering the finished product or service that constitutes their core business Examples: Semiconductors, Automobiles, Electronics, Movies 11

Vertical Integration of Large Pharma Is it Time to Disintegrate? Core Business Delivering Cost Effective Outcomes Core Functions Research & Early Development Late Development Supply Chain Stakeholder Relations Marketing approval Pricing Reimbursement Policy Sales & Marketing Administration (Legal, Finance, etc.) Support Functions Human Resources Procurement Information Technology Infrastructure (e.g., Engineering, Maintenance, etc.) 12

Research & Early Development Disintegration as an Opportunity Research and Early Development Late Development Basic Research Drug Discovery Pre- Clinical I IIa IIb III FDA Review PoS Inflection Point 0 1 2 3 4 5 6 7 Years 8 9 10 11 12 13 Large pharma no longer leads in the area of research and early development Innovation in this stage of the pharmaceutical R&D cycle is spread among thousands of Biotechs and academic laboratories Elements of the research and early development process are becoming commodities 13

J&J s External Innovation Initiative By working with experts at other companies, universities, and research institutes, we tap a wider range of expertise, capabilities, and resources. 1 Aggressively pursue licensing activities at all stages of drug development Initiate extensive in-depth collaborations with academia, Biotech companies and CRO s Create new business models for research & early development: the Open Platform 1 Paul Stoffels, Chairman of Pharmaceutical R&D, Johnson & Johnson, Boston Globe, February 2, 2009 14

Open Platform A New Model for R&D Networked Partnering In partnership with external innovators and investors, build and manage an external portfolio of early-stage product opportunities Employ the principles of Open Innovation to identify and advance early-stage innovative product opportunities Link these opportunities with external management expertise and capital Apply internal expertise, capabilities and alliance networks to facilitate the success of our partners Institute innovative financial risk-sharing strategies with external investors to support the development of products Retain options to acquire these opportunities under financial terms that are attractive to both Pharma and its partners 15

The Open Platform Concept Basic Research Drug Discovery Pre- Clinical I IIa IIb III FDA Review Academia Pharma Provides: Funding Guidance Expertise Commercialization path Venture Backed External R&D Entity Venture Capital Provides: Capital Management R&D expertise Pharma Provides: Projects R&D expertise R&D infrastructure CRO networks Pharma Pharma Provides: Licensing partner Late development Worldwide commercialization 16

Open Platform A New Model for R&D Example of Networked Partnering VCs provide capital, funding syndicate and management team VCs Acme DevCo Pharma Pharma provides projects, guidance, expertise and CRO network Acme Management Team CROs CROs provide virtual, flexible R&D organization Project A Project B Project C Project D Project E Project F Project G Project Project Managers As Needed Total of 10-20 Projects Entering at 2-3/Year Management team runs multiple projects to exit or termination NuCo or Licensing NuCo or Licensing Exit at end of PhIIa / PoC NuCo or Licensing Exit to Pharma partner, other Pharma or form NuCo

Benefits Stakeholders Large Pharma Expand pipeline with an external portfolio of product opportunities Leverage existing infrastructure and expertise Minimize fixed costs and infrastructure while maximizing flexibility Venture Capital Opportunity to invest in market scarcity Assets monetized at an early stage of the R&D process to yield attractive returns with a mid-term horizon Capital investment reduced by accessing Pharma infrastructure and expertise Academia Progress opportunities that would otherwise stagnate Access to external funding vehicles Attractive financial incentives for innovators and universities Contract Research Organizations Expand business Opportunity to risk share

Key Learnings Open Innovation must be driven from the top Do not underestimate the organization s capacity to mount an immune response The fear that competitors will profit from Open Innovation can outweigh the promise it holds for your organization Run pilot programs outside of the traditional organizational structure On occasion, ships must be burned to break through entrenched behaviors business practices Do not assume that all external constituents in your industry share your vision 19