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 Trial Protocols: Addressing Design and Therapeutics Execution Challenges Upfront Catherine Wheeler, MD • Ensuring the right inputs are considered during the VP, Clinical Development, Acetylon Pharmaceuticals protocol design phase

11:00 am • Using simulation tools to improve trial design Networking Break • Using adaptive design to drive effective development decision-making

TO REGISTER, VISIT WWW.THECONFERENCEFORUM.ORG OR CALL 646-350-2580 5 CHIEF MEDICAL OFFICER SUMMIT AGENDA

• Deploying a risk-based monitoring to reduce cost and • How to create alignment based on key variables (data enhance data quality base quality, time to lock, enrollment, etc) • Building in flexibility • Model options and pros and cons that go with them, (i.e, hybrid mix) Parvin Fardipour, PhD VP, Adaptive Clinical Trials, Aptiv Solutions • Relationship building • Different ways to interact with CROs 12:45 pm Luncheon • Governance and steering committees • How models can be different and change? 2:00 pm Determining the Optimal Development Path: Early • Key concerns and solutions Phase Tools and Techniques Moderator: • Modern techniques/tools to help determine best Pamela Palmer, MD, PhD development path CMO & Co-Founder, AcelRx Pharmaceuticals, Inc. • Mitigating development risk using adaptive Panelists: approaches, model-building, biomarker incorporation Marc Hoffmann, MD • Determining program/portfolio valuation for more SVP & General Manager, Biopharmaceutical Development, quantitative decision-making Theorem Clinical Research

Jim Bolognese Pablo Lapuerta, MD Senior Director, Strategic Consulting, Cytel, Inc. EVP, Clinical Development & CMO, Lexicon Pharmaceuticals Kenneth Getz, MBA Director, Sponsored Research, Tufts CSDD Tyler Martin, MD CMO & Director, Dynavax Technologies 2:30 pm Mark Versavel, MD, PhD, MBA Working through Complex Contracts CMO, Alzheon Therapeutics Contracting is evolving to be even more complex and can seriously delay clinical trials. To understand what is causing 3:40 pm this and how to deal up front will reduce some of the Networking Break complexity and help with the structure and approach to DSMBs, sites, hospitals and CROs. 4:05 pm Data Communication/Publication Strategy: Objectives, More specifically: Organization and Process • How new regulations are impacting contracting • Data communication strategy and the role of the • How risk levels are impacting efficient contracting CMO Moderator: • How to communicate data Steven Zelenkofske, DO, FACC, FACOI, FCCP • Disclosure: private vs public company CMO & SVP, Clinical and Medical Affairs, Regado Biosciences • Considerations for data release and the role of the CMO interacting with Wall Street, scientists, 3:00 pm investigators, and payors Optimizing Clinical Outsourcing with Fixed Funds • Key strategic components: planning and execution, • How to build an effective outsourcing program and quality data and biostatistics, manuscript incentives to go with this? development, interactions with authors and journals • Upfront communication, what is driving the decisions/ • Many variables to think about: How to choose from priorities different types of papers, what journals to work with, etc. • Clarity of planning

6 TO REGISTER, VISIT WWW.THECONFERENCEFORUM.ORG OR CALL 646-350-2580 AGENDA CHIEF MEDICAL OFFICER SUMMIT

• Generating proof sources in support of regulatory, 5:30 pm health economics, reimbursement, guidelines and Best Practice Exchange Session commercialization efforts In this session, attendees can pick a topic to gain best • What is the most efficient organizational approach: practices and share helpful ideas on the following: internal, outsourcing or mixed model? 1. Setting up and Managing a Virtual Company Lou Brenner, MD • Challenges and Opportunities CMO & SVP, Idera Pharmaceuticals • Infrastructure needs/Creating virtual platforms Ronald Burch, MD, PhD • Publication management CMO, Naurex • Setting up SOPs, templates Vojo Vukovic, PhD SVP & CMO, Synta Pharmaceuticals Corp. • What do you really need close to you and what can you manage virtually? 4:30 pm • Managing without a bench How to Get your Product Positioned for Reimbursement • Strategies in trial design • Safety issues • Strategies around applications • HR issues • Pharmacoeconomics • Ethical issues • Value based pricing Greg Fiore, MD CMO, SSI Strategy • How to ensure a clinical program has the broadest geographic applicability 2. Creating an Effective Hiring Model, Yielding the Highest Quality Professional, Efficiently Pablo Lapuerta, MD EVP, Clinical Development & CMO, Lexicon • Outside the box strategic sourcing Pharmaceuticals • Strategic relationship building and networking Oscar Segurado, MD, PhD • “In the market” or “on the market?” CMO, Crescendo / Myriad Genetics Joseph Arcangelo 5:00 pm Managing Partner, InSeption Group Career Development Opportunities for CMOs 3. Making Medical Review Faster and Easier • View on the life cycle of a CMO • Analytical needs of small companies: Identifying the • What opportunities are there for CMOs? challenges

• Founder CMOs: how does that get translated into • How to analyze clinical, operational and safety data advancement from clinical trials visually • Venture POV • Live demonstration

Lee Brettman, MD, FACP Ben McGraw President, Greystone BioConsulting, Inc. VP of Marketing, Comprehend Systems

Greg Fiore, MD 4. Best Practices for Interacting with the Board CMO, SSI Strategy Oscar Segurado, MD, PhD Steven Zelenkofske, DO, FACC, FACOI, FCCP CMO, Crescendo / Myriad Genetics CMO & SVP, Clinical and Medical Affairs, Regado Biosciences 5. Biosimilars 101 for CMOs: Threat or Opportunity? Jim Roach, MD, FACP, FCCP CMO & SVP, Development, Momenta Pharmaceuticals

TO REGISTER, VISIT WWW.THECONFERENCEFORUM.ORG OR CALL 646-350-2580 7 CHIEF MEDICAL OFFICER SUMMIT AGENDA

6:00 pm Panelists: Networking Reception Keith Dionne, PhD President & CEO, Constellation Pharmaceuticals Hosted by: Gaytri Kachroo CEO & Chairman, Rapid Pharmaceucticals Chris Kiritsy, MBA President & CEO, Arisaph

James Mannion, PhD President, CEO & Founder, Galleon Pharmaceuticals Anthony McKinney, MBA DAY TWO - TUESDAY, MAY 6, 2014 President & CEO, Co-Founder, Neurovance Martin Tolar, MD, PhD 8:00 am Founder, President & CEO, Alzheon Breakfast 10:15 am 8:30 am Networking Break Co-Chairs’ Welcome Lee Allen, MD, PhD 10:45 am CMO, Spectrum Pharmaceuticals How Investors Determine Valuation of Biotechs Steven Zelenkofske, DO, FACC, FACOI, FCCP • Evolution on what investors are looking for from an CMO & SVP, Clinical and Medical Affairs, Regado end game Biosciences • Good ideas funded and great ideas not funded

8:45 am • How venture looks at management Featured Presentation: Strategies to Position your Drug • POV on exits for Regulatory Approval and Market Access in Europe • The EU Landscape Moderator: Steven Zelenkofske, DO, FACC, FACOI, FCCP • What are the regulators looking for? CMO and SVP, Clinical & Medical Affairs, Regado • What are the payers looking for? Biosciences • Strategies to successfully navigate your product into Panelists: the European market Todd Foley, MBA Managing Director, MPM Capital Thomas Lonngren, PhD (hc), MRPharmS, FRCP Former Head, EMA Vikas Goyal, MBA Senior Associate, SR One 9:30 am David Grayzel, MD CEO Panel: What do CEOs look for in a CMO? Managing Director, Atlas Venture Development Corp. • Hiring: Identifying the appropriate CMO Roger Hill, PhD, MBA • Board interaction Managing Partner, Willow Tree Capital • Development path Jeffrey Moore VP, MP Healthcare Venture Management • What makes a CEO cringe? Robert Weisskoff, PhD • How the CMO can help position the company Partner, Fidelity Biosciences Moderator: Pamela Palmer, MD, PhD CMO & Co-Founder, AcelRx Pharmaceuticals, Inc.

8 TO REGISTER, VISIT WWW.THECONFERENCEFORUM.ORG OR CALL 646-350-2580 VENUE / REGISTRATION CHIEF MEDICAL OFFICER SUMMIT

11:30 am Conveniently located on Boston Common between the Pharma Business Development POV on Partnering Boston Theater District and the Financial District, The Ritz- • Structuring deals Carlton, Boston Common is front and center to all of the excitement and activity of downtown Boston. The hotel is a • Early phase investment vs late phase investment sanctuary of modern urban luxury in the heart of one of the • Strategy nation’s most historic cities. • Selecting the right partner Moderator: Lee Allen, MD, PhD REGISTRATION CMO, Spectrum Pharmaceuticals Early-Bird - before April 11, 2014 $1,295 Panelists: Standard - after April 11, 2014 $1,495 Richard Brudnick VP, Co-Head Business Development/ M&A, Biogen Idec The conference fee includes the program, morning coffee/ tea, luncheon, reception and conference documentation. Ivana Magovcevic-Liebisch, PhD, JD SVP, Head of Corporate Business Development, Teva Discount Programs Pharmaceuticals Group Registration – Receive a 10% discount off each registration when two or more colleagues from the same Sue Yao company register online. Licensing and Business Development, Purdue Pharma Large group discounts available. 12:15 pm Please contact [email protected] or call How BIO is Representing Emerging Biotechs 646-350-2580 for any questions on discount rates. • What is BIO doing to support emerging biotechs? Payment Policy • An overview on policy changes and market trends Payment must be received in full by the event date. All • The relationship between BIO and the FDA discounts will be applied to the Conference-Only fee (excluding add-ons)‚ cannot be combined with any other • BIO’s list of priorities for emerging biotechs offer‚ and must be paid in full at the time of conference.

Cartier Esham, PhD Substitution and Cancellation Policy EVP, Emerging Companies, Biotechnology Industry If you are unable to attend and would like to register a Organization (BIO) substitute, please email [email protected]. Please ask your substitute to provide identification on-site. 12:45 pm Conference Concludes If you need to cancel your registration, please note the following policies based on the start date of the event:

Ten or more business days prior: A full refund less the VENUE administration fee of $295, or a pass to another event valid for two years from the date of issue. The Ritz-Carlton, Boston Less than ten business days prior: A pass to a future Common event for either yourself or a colleague from the same 10 Avery Street company valid for two years from the date of issue. Boston, MA 02111 617-574-7100 To cancel and receive a refund or pass, please email www.ritzcarlton.com [email protected] or call 646-350-2580. For Reservations call 800-241-3333 or 617-574-7100 and ask for The Chief Medical Officer Summit group rate or click https://www.ritzcarlton.com/en/Properties/ BostonCommon/Reservations/Default.htm#top and enter the Group Code: CFMCFMA.

TO REGISTER, VISIT WWW.THECONFERENCEFORUM.ORG OR CALL 646-350-2580 9 CHIEF MEDICAL OFFICER SUMMIT SPONSORS

EXECUTIVE SPONSORS MEDIA PARTNERS

ASSOCIATE SPONSORS

EXHIBITOR

10 TO REGISTER, VISIT WWW.THECONFERENCEFORUM.ORG OR CALL 646-350-2580