Perspectives of Innovative Small Companies on the Industry s Prospects for 2012 and Beyond

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Perspectives of Innovative Small Companies on the Industry s Prospects for 2012 and Beyond ACI Forum on Structuring, Negotiating and Managing Life Sciences Collaborations and Acquisitions February 28, 2012 2012 Foley Hoag LLP. All Rights Reserved.

Panelists Wendy Tsai, Founder and VP Alliances, Cellona Therapeutics Julia C. Owens, Senior V.P. Corporate Development and Strategy, Lycera Gregory Miller, Head, Business & Corporate Development, CoNCERT Pharmaceuticals Hemmie Chang, Chair, Licensing & Strategic Alliances Group, Partner, Foley Hoag LLP 2012 Foley Hoag LLP. All Rights Reserved. Perspectives of Innovative Small Companies 2

Wendy Tsai Cellona Therapeutics Mission: Develop targeted therapeutics to improve survival paradigms in cancer patients. Strengths: Management team has an extensive and successful track record in the critical elements of Cellona s business model. Pioneering science and robust patents. Milestone-driven corporate planning with non-dilutive seed capital from government QTDP grant; foundation and angels. Partnering Strategy: Academic collaboration 2012 Foley Hoag LLP. All Rights Reserved. Perspectives of Innovative Small Companies 3

Julia Owens - Lycera Mission: Discovery and development of first-in-class oral medications to treat patients with serious immune diseases for which existing therapies are inadequate. Strengths: Experienced and accomplished leadership and scientific teams with core capabilities in discovery research, medicinal chemistry and early development. $36 million Series A financing. Significant research collaboration with Merck. Partnering Strategy: Big Pharma 2012 Foley Hoag LLP. All Rights Reserved. Perspectives of Innovative Small Companies 4

Gregory Miller CoNCERT Pharmaceuticals Mission: Applying DCE Platform (deuterated chemical entity platform) to create novel, differentiated drugs. Strengths: Successful clinical demonstration achieved. Rapid progression from initial synthesis to clinic. Broad patent estate and intellectual property position. Strong financial position over $110 million raised. Partnering Strategy: Pharma and Government, with parallel development of unpartnered programs 2012 Foley Hoag LLP. All Rights Reserved. Perspectives of Innovative Small Companies 5

Licensing Trends: Recent Deal Activity Licensing Deals With >$10M Upfront Payment Source: EBI Transaction Tracker, Accessed November 11, 2010 and October 30, 2011 2012 Foley Hoag LLP. All Rights Reserved. Perspectives of Innovative Small Companies 6

Challenging Environment for Early Stage Funding Life sciences funding by stage 2009-2011 Why did your company delay R&D 2011? Source: PricewaterhouseCoopers, Zigzagging upward, February 2012 Source: California Biomedical Industry 2012 Report 2012 Foley Hoag LLP. All Rights Reserved. Perspectives of Innovative Small Companies 7

Expanding Base of Early Stage Investors 2012 Foley Hoag LLP. All Rights Reserved. Perspectives of Innovative Small Companies 8

Financing Trends: Positioning for Success DE-RISKED ASSETS VC INCUBATED CAPITAL EFFICIENT, VIRTUAL MODEL SYNDICATED RISK PHARMA PARTNERSHIP EARLY STAGE DEAL ROBUST R&D PIPELINE 2012 Foley Hoag LLP. All Rights Reserved. Perspectives of Innovative Small Companies 9 3979044v2

Licensing Deal Term Trends: Up-fronts Millions ($) Source: Elsevier s Strategic Transactions. Data set includes 299 alliances by big pharma, mid-sized European companies, top Japanese, and big biotech for which upfronts were disclosed. 2010 Phase II average skewed by mammoth Abbott/Reata deal; if excluded average drops to $38.3mm. 2012 Foley Hoag LLP. All Rights Reserved. Perspectives of Innovative Small Companies 10

Evaluating Potential Partnerships Some overlapping priorities: Exclusivity Upfront and nearterm milestones Retain profit share option Partner with resources, marketing expertise Shared vision Scientific alignment Innovation Diverse partners Complementary program to internal R&D Ability to make rapid progress SMALL INNOVATOR POTENTIAL PARTNER 2012 Foley Hoag LLP. All Rights Reserved. Perspectives of Innovative Small Companies 11

Process of Seeking a Collaboration???? Royalties Upfront payments Milestone payments Profit share option Timing Ownership of IP Control 2012 Foley Hoag LLP. All Rights Reserved. Perspectives of Innovative Small Companies 12

Going From Early Stage to the Next Stage Develop technology Raise $110M $1B strategic alliance 2012 Foley Hoag LLP. All Rights Reserved. Perspectives of Innovative Small Companies 13

Developing Diverse Pipeline in Parallel Research Development Development Programs CTP-499: Diabetic Nephropathy (anti-inflammatory/anti-fibrotic/anti-oxidant) CTP-298: HIV (protease inhibitor) Select Research Programs C-21191: Spasticity, Pain C-10003: Pain, PBA C-10068: Epilepsy, Depression C-21359: Hematological Cancers Research Preclinical Phase I Phase 2 C-10291: Narcolepsy, Fibromyalgia D-Praziquantel: Schistosomiasis 2012 Foley Hoag LLP. All Rights Reserved. Perspectives of Innovative Small Companies 14

Thinking About Exit: What s Pharma Buying? Source: Elsevier s Strategic Transactions 2012 Foley Hoag LLP. All Rights Reserved. Perspectives of Innovative Small Companies 15

VCs: In which industries do you expect to see investment froth in 2012? Biopharma Source: National Venture Capital Association and DowJones Venture Source, Venture View 2012: Venture Capital Industry Predictions, December 2011 2012 Foley Hoag LLP. All Rights Reserved. Perspectives of Innovative Small Companies 16

Final thoughts from our panel Wendy Tsai, Founder and VP Alliances, Cellona Therapeutics Julia C. Owens, Senior V.P. Corporate Development and Strategy, Lycera Gregory Miller, Head, Business & Corporate Development, CoNCERT Pharmaceuticals Hemmie Chang, Chair, Licensing & Strategic Alliances Group, Partner, Foley Hoag LLP 2012 Foley Hoag LLP. All Rights Reserved. Perspectives of Innovative Small Companies 17