CHIEF MEDICAL OFFICER SUMMIT ADDRESSING THE COMPLEX DUAL ROLE OF MANAGING R&D WHILE RAISING CAPITAL FOR BIOTECHS MAY 5 - 6, 2014 RITZ CARLTON, BOSTON COMMON, BOSTON, MA CO-CHAIR: CO-CHAIR: Lee Allen, MD, PhD Steven Zelenkofske, DO, FACC, FACOI, FCCP CMO SVP, Clinical and Medical Affairs & CMO Spectrum Pharmaceuticals Regado Biosciences FEATURED SPEAKER: FEATURED SPEAKER: FEATURED SPEAKER: Jakob Dupont, MD Ken Getz, MBA Pamela Palmer, MD, PhD SVP & CMO Director of Sponsored CMO & Co-Founder OncoMed Research AcelRX Pharmaceuticals, Tufts CSDD Inc. NEW FOR 2014 Keynote Case Study on the Life of a CMO How to Get Your Drug Positioned Pre and Post IPO for Reimbursement • • Best Practices to Position Your Drug for Career Development Opportunities for CMOs Regulatory Approval • • CEO Panel: What do CEOs look for in a Working through Complex Contracts CMO? • • Publication Strategy: Objectives, Organization How Investors Determine Valuation of and Process Biotechs Executive Sponsors Associate Sponsors Exhibitor Organized by TO REGISTER, VISIT WWW.THECONFERENCEFORUM.ORG OR CALL 646-350-2580 CHIEF MEDICAL OFFICER SUMMIT OVERVIEW Elizabeth Bard OVERVIEW Business Development Manager The Conference Forum’s Second Annual Chief Medical David Borrok Officer Summit for Emerging Life Science Companies is Business Development Manager delighted to present a truly distinctive and interactive event for CMOs. We are grateful to Dr. Elizabeth Stoner, Meredith Sands Managing Director, MPM Capital, for suggesting the idea Executive Director, Business Development and to our CMO advisers for their insights on content and agenda development. We are Dedicated to Accomplishing Two Goals SPEAKING FACULTY • To bring together CMO executives to address the unique challenges associated with directing and Lee Allen, MD, PhD managing all R&D functions with limited resources, CMO while raising capital, working and meeting with Spectrum Pharmaceuticals investors and strategizing for appropriate exits. Joseph Arcangelo • To create a network of CMOs from small to mid-size Managing Partner life science companies to share ideas, solutions and InSeption Group support. Jim Bolognese The World of a CMO in Emerging Life Science Senior Director, Strategic Consulting Companies Cytel, Inc. The role of a Chief Medical Officer is one that means Lou Brenner, MD many things to many people, and is radically different SVP & CMO depending on the type of organization in which a CMO Idera Pharmaceuticals works. For CMOs in early, emerging and even mid-size life science companies, the role can be an isolated one. It is Lee Brettman, MD, FACP difficult to balance the responsibility for driving clinical President development and CRO management while meeting the Greystone BioConsulting, Inc. needs of investors and positioning for an appropriate exit. Furthermore, CMOs are playing an increasingly larger role Richard Brudnick in raising funding for their life science companies. VP, Co-Head Business Development/ M&A Biogen Idec Who Should Attend The conference is designed specifically for CMOs, but Ronald Burch, MD, PhD certainly Heads of R&D, Clinical Operations/Development/ CMO Outsourcing, and Vice Presidents of Medical Affairs would Naurex also greatly benefit from attending the program. Clinical service providers and investors are encouraged to attend Keith Dionne, PhD as conversations with CMOs are very much welcomed. President & CEO Constellation Pharmaceuticals Sincerely, Valerie Bowling Jakob Dupont, MD Executive Director SVP & CMO OncoMed Kristen Pisanelli Conference Producer Cartier Esham, PhD EVP, Emerging Companies Jessica Rothenberg Biotechnology Industry Organization (BIO) Marketing Director 2 TO REGISTER, VISIT WWW.THECONFERENCEFORUM.ORG OR CALL 646-350-2580 SPEAKING FACULTY CHIEF MEDICAL OFFICER SUMMIT Parvin Fardipour, PhD Pablo Lapuerta, MD VP, Adaptive Clinical Trials EVP, Clinical Development & CMO Aptiv Solutions Lexicon Pharmaceuticals Greg Fiore, MD Thomas Lonngren, PhD (hc), MRPharmS, FRCP CMO Former Head SSI Strategy EMA Todd Foley, MBA Ivana Magovcevic-Liebisch, PhD, JD Managing Director SVP, Head of Corporate Business Development MPM Capital Teva Pharmaceuticals Shi Yin Foo, MD, PhD James Mannion, PhD CMO President, CEO & Founder Cardioxyl Pharmaceuticals Galleon Pharmaceuticals Ken Getz, MBA Richard Margolin, MD Director of Sponsored Research VP Clinical Development Tufts CSDD CereSpir, Inc. Vikas Goyal, MBA Tyler Martin, MD Senior Associate Founder & CEO SR One Great Plains Biotechnology David Grayzel, MD Ben McGraw Managing Director VP of Marketing Atlas Venture Development Corp. Comprehend Systems Laurie Halloran, BSN, MS Jesse McGreivy, MD President & CEO CMO Halloran Consulting Group Pharmacyclics Roger Hill, PhD, MBA Anthony McKinney, MBA Managing Partner President & CEO, Co-Founder Willow Tree Capital Neurovance Marc Hoffmann, MD Jeffrey Moore, DPhil, MBA SVP & General Manager, Biopharmaceutical Development VP Theorem Clinical Research MP Healthcare Venture Management, Inc. Gaytri Kachroo Louis O’Dea, MB BCh BAO FRCP(C) CEO & Chairman SVP, CMO & Head of Regulatory Affairs Rapid Pharmaceuticals Moderna Therapeutics Edward Kaye, MD Pamela Palmer, MD, PhD CMO & SVP CMO Sarepta Therapeutics AcelRx Chris Kiritsy, MBA Jim Roach, MD President & CEO CMO & SVP, Development Arisaph Momenta Pharmaceuticals Eric Lang, MD Noah Rosenberg, MD VP, Molecule Development Group CMO Covance, Inc. Medimetriks TO REGISTER, VISIT WWW.THECONFERENCEFORUM.ORG OR CALL 646-350-2580 3 CHIEF MEDICAL OFFICER SUMMIT AGENDA Oscar Segurado, MD, PhD CMO DAY ONE - MONDAY, MAY 5, 2014 Crescendo Bioscience / Myriad Genetics 8:00 am Lee Simon, MD, FACP, FACR Registration & Breakfast Former Division Director / Principal Consultant FDA / SDG, LLC 8:30 am Co-Chairs’ Opening Remarks: Defining the Role of the Martin Tolar, MD, PhD CMO Founder, President & CEO • Differences in a CMO in large pharma, pre-IPO Alzheon pharma, post IPO pharma Mark Versavel, MD, PhD, MBA • Individual roles in pharma that make up a biotech CMO • The reality of the CMO role in an emerging biotech Alzheon Therapeutics • Functioning with a lot of hats Vojo Vukovic, PhD SVP & CMO • What’s on the mind of the CMOs? Synta Pharmaceuticals Corp. Lee Allen, MD, PhD CMO, Spectrum Pharmaceuticals Robert Weisskoff, PhD Partner Steven Zelenkofske, DO, FACC, FACOI, FCCP Fidelity Biosciences CMO & SVP, Clinical and Medical Affairs, Regado Biosciences Catherine Wheeler, MD VP, Clinical Development 8:45 am Acetylon Pharmaceuticals Keynote Case Study on the Life of a CMO Pre and Post IPO Sue Yao Licensing and Business Development • Insights into the experience Purdue Pharma • Expanding the role of the CMO to meet the requirements of commercialization Steven Zelenkofske, DO, FACC, FACOI, FCCP CMO and SVP, Clinical & Medical Affairs • Post market expectations Regado Biosciences • The role of the CMO • The CMO’s contributions • Managing growth • What role does the CMO have in articulating the data of the clinical trials in the timing of exercising more shares or deal making? • Lessons learned Jakob Dupont, MD CMO & SVP, OncoMed 9:30 am Best Practices to Position your Drug for Regulatory Approval • What is the FDA looking for? • De-risking programs 4 TO REGISTER, VISIT WWW.THECONFERENCEFORUM.ORG OR CALL 646-350-2580 AGENDA CHIEF MEDICAL OFFICER SUMMIT • How to ensure a clinical program has the broadest 11:30 am geographic applicability Re-Shaping Clinical Trials to be More Cost Effective • What are all the different regulatory options for • Prominent reasons for failure of drug development registration • Early phase development signals that predict later • Breakthrough designation experience, what works phase success and what doesn’t work • Leveraging knowledge on cost and PoS as the • Best practices on how to work with the FDA biggest driver for investment Edward Kaye, MD • What strategies are companies implementing to most CMO & SVP, Sarepta Therapeutics improve clinical trial efficiency? Jesse McGreivy, MD • What approaches are organizations taking to reinvent CMO, Pharmacyclics study design (e.g., feasibility assessment, adaptive designs, single arm trials)? Lee Simon, MD Former Division Director, FDA and Principal Consultant, • How should we be thinking about leveraging big data SDG, LLC to optimize study designs? • Exploring new approaches (e.g. point-of-care and 10:15 am payer practices) to gather strategic information Clinical Development Planning Based on Business informing study design Objectives • The costly price of not having a plan to oversee Emerging biotechs have a unique opportunity to set the patient safety “right” framework for the company. Careful thought should be given when determining what areas are unique and Moderator: there is no “one size fits all”. Kenneth Getz, MBA Director, Sponsored Research, Tufts CSDD Key areas to consider: Panelists: • Strategic development planning (When?) Shi Yin Foo, MD, PhD • Resourcing and Functional Area Expertise (Who?) CMO, Cardioxyl Pharmaceuticals • Operational and Systems Infrastructure (What?) Eric Lang, MD VP, Molecule Development Group, Covance, Inc. • Program Leadership (How?) Richard Margolin, MD Moderator: VP Clinical Development, CereSpir, Inc. Laurie Halloran, BSN, MS President & CEO, Halloran Consulting Group, Inc. Jim Roach, MD, FACP, FCCP CMO & SVP, Development, Momenta Pharmaceuticals Panelists: Edward Kaye, MD Noah Rosenberg, MD CMO & SVP, Sarepta Therapeutics CMO, Medimetriks Louis O’Dea, MB BCh BAO FRCP(C) 12:15 pm SVP, CMO & Head of Regulatory Affairs, Moderna Optimizing