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CHIEF MEDICAL OFFICER SUMMITADDRESSING THE COMPLEX DUAL ROLE OF MANAGING R&D

WHILE RAISING CAPITAL FOR BIOTECHS

MAY 5 - 6, 2014 RITZ CARLTON, BOSTON COMMON, BOSTON, MA

Keynote Case Study on the Life of a CMO

Pre and Post IPO•

Best Practices to Position Your Drug for

Regulatory Approval•

Working through Complex Contracts•

Publication Strategy: Objectives, Organization

and Process

How to Get Your Drug Positioned

for Reimbursement•

Career Development Opportunities for CMOs•

CEO Panel: What do CEOs look for in a

CMO?•

How Investors Determine Valuation of

Biotechs

FEATURED SPEAKER:Ken Getz, MBADirector of Sponsored Research Tufts CSDD

ExhibitorAssociate SponsorsExecutive Sponsors

CO-CHAIR:Steven Zelenkofske, DO, FACC, FACOI, FCCPSVP, Clinical and Medical Affairs & CMORegado Biosciences

CO-CHAIR:Lee Allen, MD, PhDCMOSpectrum Pharmaceuticals

FEATURED SPEAKER:Pamela Palmer, MD, PhDCMO & Co-FounderAcelRX Pharmaceuticals, Inc.

FEATURED SPEAKER:Jakob Dupont, MDSVP & CMOOncoMed

NEW FOR 2014

Organized by

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OVERVIEW

The Conference Forum’s Second Annual Chief Medical Officer Summit for Emerging Life Science Companies is delighted to present a truly distinctive and interactive event for CMOs. We are grateful to Dr. Elizabeth Stoner, Managing Director, MPM Capital, for suggesting the idea and to our CMO advisers for their insights on content and agenda development.

We are Dedicated to Accomplishing Two Goals

• To bring together CMO executives to address the unique challenges associated with directing and managing all R&D functions with limited resources, while raising capital, working and meeting with investors and strategizing for appropriate exits.

• To create a network of CMOs from small to mid-size life science companies to share ideas, solutions and support.

The World of a CMO in Emerging Life Science CompaniesThe role of a Chief Medical Officer is one that means many things to many people, and is radically different depending on the type of organization in which a CMO works. For CMOs in early, emerging and even mid-size life science companies, the role can be an isolated one. It is difficult to balance the responsibility for driving clinical development and CRO management while meeting the needs of investors and positioning for an appropriate exit. Furthermore, CMOs are playing an increasingly larger role in raising funding for their life science companies.

Who Should AttendThe conference is designed specifically for CMOs, but certainly Heads of R&D, Clinical Operations/Development/Outsourcing, and Vice Presidents of Medical Affairs would also greatly benefit from attending the program. Clinical service providers and investors are encouraged to attend as conversations with CMOs are very much welcomed.

Sincerely,

Valerie BowlingExecutive Director

Kristen PisanelliConference Producer

Jessica RothenbergMarketing Director

Elizabeth BardBusiness Development Manager

David BorrokBusiness Development Manager

Meredith SandsExecutive Director, Business Development

SPEAKING FACULTY

Lee Allen, MD, PhDCMOSpectrum Pharmaceuticals

Joseph ArcangeloManaging PartnerInSeption Group

Jim BologneseSenior Director, Strategic ConsultingCytel, Inc.

Lou Brenner, MDSVP & CMOIdera Pharmaceuticals

Lee Brettman, MD, FACPPresidentGreystone BioConsulting, Inc.

Richard BrudnickVP, Co-Head Business Development/ M&ABiogen Idec

Ronald Burch, MD, PhDCMONaurex

Keith Dionne, PhDPresident & CEOConstellation Pharmaceuticals

Jakob Dupont, MDSVP & CMOOncoMed

Cartier Esham, PhDEVP, Emerging CompaniesBiotechnology Industry Organization (BIO)

CHIEF MEDICAL OFFICER SUMMIT OVERVIEW

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Parvin Fardipour, PhDVP, Adaptive Clinical TrialsAptiv Solutions

Greg Fiore, MDCMOSSI Strategy

Todd Foley, MBAManaging Director MPM Capital

Shi Yin Foo, MD, PhDCMOCardioxyl Pharmaceuticals

Ken Getz, MBADirector of Sponsored ResearchTufts CSDD

Vikas Goyal, MBASenior AssociateSR One

David Grayzel, MDManaging DirectorAtlas Venture Development Corp.

Laurie Halloran, BSN, MSPresident & CEOHalloran Consulting Group

Roger Hill, PhD, MBAManaging PartnerWillow Tree Capital

Marc Hoffmann, MDSVP & General Manager, Biopharmaceutical DevelopmentTheorem Clinical Research

Gaytri KachrooCEO & ChairmanRapid Pharmaceuticals

Edward Kaye, MDCMO & SVPSarepta Therapeutics

Chris Kiritsy, MBAPresident & CEOArisaph

Eric Lang, MDVP, Molecule Development Group Covance, Inc.

Pablo Lapuerta, MDEVP, Clinical Development & CMOLexicon Pharmaceuticals

Thomas Lonngren, PhD (hc), MRPharmS, FRCPFormer HeadEMA

Ivana Magovcevic-Liebisch, PhD, JDSVP, Head of Corporate Business DevelopmentTeva Pharmaceuticals

James Mannion, PhDPresident, CEO & FounderGalleon Pharmaceuticals

Richard Margolin, MDVP Clinical DevelopmentCereSpir, Inc.

Tyler Martin, MDFounder & CEOGreat Plains Biotechnology

Ben McGrawVP of MarketingComprehend Systems

Jesse McGreivy, MDCMOPharmacyclics

Anthony McKinney, MBAPresident & CEO, Co-FounderNeurovance

Jeffrey Moore, DPhil, MBAVPMP Healthcare Venture Management, Inc.

Louis O’Dea, MB BCh BAO FRCP(C)SVP, CMO & Head of Regulatory AffairsModerna Therapeutics

Pamela Palmer, MD, PhDCMOAcelRx

Jim Roach, MDCMO & SVP, DevelopmentMomenta Pharmaceuticals

Noah Rosenberg, MDCMOMedimetriks

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SPEAKING FACULTY CHIEF MEDICAL OFFICER SUMMIT

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Oscar Segurado, MD, PhDCMOCrescendo Bioscience / Myriad Genetics

Lee Simon, MD, FACP, FACRFormer Division Director / Principal ConsultantFDA / SDG, LLC

Martin Tolar, MD, PhDFounder, President & CEOAlzheon

Mark Versavel, MD, PhD, MBACMOAlzheon Therapeutics

Vojo Vukovic, PhDSVP & CMOSynta Pharmaceuticals Corp.

Robert Weisskoff, PhDPartnerFidelity Biosciences

Catherine Wheeler, MDVP, Clinical DevelopmentAcetylon Pharmaceuticals

Sue YaoLicensing and Business DevelopmentPurdue Pharma

Steven Zelenkofske, DO, FACC, FACOI, FCCPCMO and SVP, Clinical & Medical AffairsRegado Biosciences

DAY ONE - MONDAY, MAY 5, 2014

8:00 amRegistration & Breakfast

8:30 amCo-Chairs’ Opening Remarks: Defining the Role of the CMO

• Differences in a CMO in large pharma, pre-IPO pharma, post IPO pharma

• Individual roles in pharma that make up a biotech

• The reality of the CMO role in an emerging biotech

• Functioning with a lot of hats

• What’s on the mind of the CMOs?

Lee Allen, MD, PhDCMO, Spectrum Pharmaceuticals

Steven Zelenkofske, DO, FACC, FACOI, FCCPCMO & SVP, Clinical and Medical Affairs, Regado Biosciences

8:45 amKeynote Case Study on the Life of a CMO Pre and Post IPO

• Insights into the experience

• Expanding the role of the CMO to meet the requirements of commercialization

• Post market expectations

• 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, MDCMO & SVP, OncoMed

9:30 amBest Practices to Position your Drug for Regulatory Approval

• What is the FDA looking for?

• De-risking programs

CHIEF MEDICAL OFFICER SUMMIT AGENDA

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• How to ensure a clinical program has the broadest geographic applicability

• What are all the different regulatory options for registration

• Breakthrough designation experience, what works and what doesn’t work

• Best practices on how to work with the FDA

Edward Kaye, MDCMO & SVP, Sarepta Therapeutics

Jesse McGreivy, MDCMO, Pharmacyclics

Lee Simon, MDFormer Division Director, FDA and Principal Consultant, SDG, LLC

10:15 amClinical Development Planning Based on Business Objectives

Emerging biotechs have a unique opportunity to set the “right” framework for the company. Careful thought should be given when determining what areas are unique and there is no “one size fits all”.

Key areas to consider:

• Strategic development planning (When?)

• Resourcing and Functional Area Expertise (Who?)

• Operational and Systems Infrastructure (What?)

• Program Leadership (How?)

Moderator:Laurie Halloran, BSN, MSPresident & CEO, Halloran Consulting Group, Inc.

Panelists:Edward Kaye, MDCMO & SVP, Sarepta Therapeutics

Louis O’Dea, MB BCh BAO FRCP(C)SVP, CMO & Head of Regulatory Affairs, Moderna Therapeutics

Catherine Wheeler, MDVP, Clinical Development, Acetylon Pharmaceuticals

11:00 amNetworking Break

11:30 amRe-Shaping Clinical Trials to be More Cost Effective

• Prominent reasons for failure of drug development

• Early phase development signals that predict later phase success

• Leveraging knowledge on cost and PoS as the biggest driver for investment

• What strategies are companies implementing to most improve clinical trial efficiency?

• What approaches are organizations taking to reinvent study design (e.g., feasibility assessment, adaptive designs, single arm trials)?

• How should we be thinking about leveraging big data to optimize study designs?

• Exploring new approaches (e.g. point-of-care and payer practices) to gather strategic information informing study design

• The costly price of not having a plan to oversee patient safety

Moderator:Kenneth Getz, MBADirector, Sponsored Research, Tufts CSDD

Panelists:Shi Yin Foo, MD, PhDCMO, Cardioxyl Pharmaceuticals

Eric Lang, MDVP, Molecule Development Group, Covance, Inc.

Richard Margolin, MDVP Clinical Development, CereSpir, Inc.

Jim Roach, MD, FACP, FCCPCMO & SVP, Development, Momenta Pharmaceuticals

Noah Rosenberg, MDCMO, Medimetriks

12:15 pmOptimizing Trial Protocols: Addressing Design and Execution Challenges Upfront

• Ensuring the right inputs are considered during the protocol design phase

• Using simulation tools to improve trial design

• Using adaptive design to drive effective development decision-making

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• Deploying a risk-based monitoring to reduce cost and enhance data quality

• Building in flexibility

Parvin Fardipour, PhDVP, Adaptive Clinical Trials, Aptiv Solutions

12:45 pmLuncheon

2:00 pmDetermining the Optimal Development Path: Early Phase Tools and Techniques

• Modern techniques/tools to help determine best development path

• Mitigating development risk using adaptive approaches, model-building, biomarker incorporation

• Determining program/portfolio valuation for more quantitative decision-making

Jim BologneseSenior Director, Strategic Consulting, Cytel, Inc.

Kenneth Getz, MBADirector, Sponsored Research, Tufts CSDD

2:30 pmWorking through Complex Contracts

Contracting is evolving to be even more complex and can seriously delay clinical trials. To understand what is causing this and how to deal up front will reduce some of the complexity and help with the structure and approach to DSMBs, sites, hospitals and CROs.

More specifically:

• How new regulations are impacting contracting

• How risk levels are impacting efficient contracting

Moderator:Steven Zelenkofske, DO, FACC, FACOI, FCCPCMO & SVP, Clinical and Medical Affairs, Regado Biosciences

3:00 pmOptimizing Clinical Outsourcing with Fixed Funds

• How to build an effective outsourcing program and incentives to go with this?

• Upfront communication, what is driving the decisions/priorities

• Clarity of planning

• How to create alignment based on key variables (data base quality, time to lock, enrollment, etc)

• Model options and pros and cons that go with them, (i.e, hybrid mix)

• Relationship building

• Different ways to interact with CROs

• Governance and steering committees

• How models can be different and change?

• Key concerns and solutions

Moderator:Pamela Palmer, MD, PhDCMO & Co-Founder, AcelRx Pharmaceuticals, Inc.

Panelists:Marc Hoffmann, MDSVP & General Manager, Biopharmaceutical Development, Theorem Clinical Research

Pablo Lapuerta, MDEVP, Clinical Development & CMO, Lexicon Pharmaceuticals

Tyler Martin, MDCMO & Director, Dynavax Technologies

Mark Versavel, MD, PhD, MBACMO, Alzheon Therapeutics

3:40 pmNetworking Break

4:05 pmData Communication/Publication Strategy: Objectives, Organization and Process

• Data communication strategy and the role of the CMO

• How to communicate data

• Disclosure: private vs public company

• Considerations for data release and the role of the CMO interacting with Wall Street, scientists, investigators, and payors

• Key strategic components: planning and execution, quality data and biostatistics, manuscript development, interactions with authors and journals

• Many variables to think about: How to choose from different types of papers, what journals to work with, etc.

CHIEF MEDICAL OFFICER SUMMIT AGENDA

6

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• Generating proof sources in support of regulatory, health economics, reimbursement, guidelines and commercialization efforts

• What is the most efficient organizational approach: internal, outsourcing or mixed model?

Lou Brenner, MDCMO & SVP, Idera Pharmaceuticals

Ronald Burch, MD, PhDCMO, Naurex

Vojo Vukovic, PhDSVP & CMO, Synta Pharmaceuticals Corp.

4:30 pmHow to Get your Product Positioned for Reimbursement

• Strategies in trial design

• Strategies around applications

• Pharmacoeconomics

• Value based pricing

• How to ensure a clinical program has the broadest geographic applicability

Pablo Lapuerta, MDEVP, Clinical Development & CMO, Lexicon Pharmaceuticals

Oscar Segurado, MD, PhDCMO, Crescendo / Myriad Genetics

5:00 pmCareer Development Opportunities for CMOs

• View on the life cycle of a CMO

• What opportunities are there for CMOs?

• Founder CMOs: how does that get translated into advancement

• Venture POV

Lee Brettman, MD, FACPPresident, Greystone BioConsulting, Inc.

Greg Fiore, MDCMO, SSI Strategy

Steven Zelenkofske, DO, FACC, FACOI, FCCPCMO & SVP, Clinical and Medical Affairs, Regado Biosciences

5:30 pmBest Practice Exchange Session

In this session, attendees can pick a topic to gain best practices and share helpful ideas on the following:

1. Setting up and Managing a Virtual Company

• Challenges and Opportunities

• Infrastructure needs/Creating virtual platforms

• Publication management

• Setting up SOPs, templates

• What do you really need close to you and what can you manage virtually?

• Managing without a bench

• Safety issues

• HR issues

• Ethical issues

Greg Fiore, MDCMO, SSI Strategy

2. Creating an Effective Hiring Model, Yielding the Highest Quality Professional, Efficiently

• Outside the box strategic sourcing

• Strategic relationship building and networking

• “In the market” or “on the market?”

Joseph ArcangeloManaging Partner, InSeption Group

3. Making Medical Review Faster and Easier

• Analytical needs of small companies: Identifying the challenges

• How to analyze clinical, operational and safety data from clinical trials visually

• Live demonstration

Ben McGrawVP of Marketing, Comprehend Systems

4. Best Practices for Interacting with the Board

Oscar Segurado, MD, PhDCMO, Crescendo / Myriad Genetics

5. Biosimilars 101 for CMOs: Threat or Opportunity?

Jim Roach, MD, FACP, FCCPCMO & SVP, Development, Momenta Pharmaceuticals

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6:00 pmNetworking Reception

Hosted by:

DAY TWO - TUESDAY, MAY 6, 2014

8:00 amBreakfast

8:30 amCo-Chairs’ Welcome

Lee Allen, MD, PhDCMO, Spectrum Pharmaceuticals

Steven Zelenkofske, DO, FACC, FACOI, FCCPCMO & SVP, Clinical and Medical Affairs, Regado Biosciences

8:45 amFeatured Presentation: Strategies to Position your Drug for Regulatory Approval and Market Access in Europe

• The EU Landscape

• What are the regulators looking for?

• What are the payers looking for?

• Strategies to successfully navigate your product into the European market

Thomas Lonngren, PhD (hc), MRPharmS, FRCPFormer Head, EMA

9:30 amCEO Panel: What do CEOs look for in a CMO?

• Hiring: Identifying the appropriate CMO

• Board interaction

• Development path

• What makes a CEO cringe?

• How the CMO can help position the company

Moderator:Pamela Palmer, MD, PhDCMO & Co-Founder, AcelRx Pharmaceuticals, Inc.

Panelists:Keith Dionne, PhDPresident & CEO, Constellation Pharmaceuticals

Gaytri KachrooCEO & Chairman, Rapid Pharmaceucticals

Chris Kiritsy, MBAPresident & CEO, Arisaph

James Mannion, PhDPresident, CEO & Founder, Galleon Pharmaceuticals

Anthony McKinney, MBAPresident & CEO, Co-Founder, Neurovance

Martin Tolar, MD, PhDFounder, President & CEO, Alzheon

10:15 amNetworking Break

10:45 amHow Investors Determine Valuation of Biotechs

• Evolution on what investors are looking for from an end game

• Good ideas funded and great ideas not funded

• How venture looks at management

• POV on exits

Moderator:Steven Zelenkofske, DO, FACC, FACOI, FCCPCMO and SVP, Clinical & Medical Affairs, Regado Biosciences

Panelists:Todd Foley, MBAManaging Director, MPM Capital

Vikas Goyal, MBASenior Associate, SR One

David Grayzel, MDManaging Director, Atlas Venture Development Corp.

Roger Hill, PhD, MBAManaging Partner, Willow Tree Capital

Jeffrey MooreVP, MP Healthcare Venture Management

Robert Weisskoff, PhDPartner, Fidelity Biosciences

CHIEF MEDICAL OFFICER SUMMIT AGENDA

8

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11:30 amPharma Business Development POV on Partnering

• Structuring deals

• Early phase investment vs late phase investment

• Strategy

• Selecting the right partner

Moderator:Lee Allen, MD, PhDCMO, Spectrum Pharmaceuticals

Panelists:Richard BrudnickVP, Co-Head Business Development/ M&A, Biogen Idec

Ivana Magovcevic-Liebisch, PhD, JDSVP, Head of Corporate Business Development, Teva Pharmaceuticals

Sue YaoLicensing and Business Development, Purdue Pharma

12:15 pmHow BIO is Representing Emerging Biotechs

• What is BIO doing to support emerging biotechs?

• An overview on policy changes and market trends

• The relationship between BIO and the FDA

• BIO’s list of priorities for emerging biotechs

Cartier Esham, PhDEVP, Emerging Companies, Biotechnology Industry Organization (BIO)

12:45 pmConference Concludes

VENUE

The Ritz-Carlton, Boston Common10 Avery StreetBoston, MA 02111617-574-7100www.ritzcarlton.com

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.

Conveniently located on Boston Common between the Boston Theater District and the Financial District, The Ritz-Carlton, Boston Common is front and center to all of the excitement and activity of downtown Boston. The hotel is a sanctuary of modern urban luxury in the heart of one of the nation’s most historic cities.

REGISTRATION

Early-Bird - before April 11, 2014 $1,295

Standard - after April 11, 2014 $1,495

The conference fee includes the program, morning coffee/tea, luncheon, reception and conference documentation.

Discount ProgramsGroup Registration – Receive a 10% discount off each registration when two or more colleagues from the same company register online.

Large group discounts available.

Please contact Service@theconferenceforum.org or call 646-350-2580 for any questions on discount rates.

Payment PolicyPayment must be received in full by the event date. All discounts will be applied to the Conference-Only fee (excluding add-ons)‚ cannot be combined with any other offer‚ and must be paid in full at the time of conference.

Substitution and Cancellation PolicyIf you are unable to attend and would like to register a substitute, please email Service@theconferenceforum.org. Please ask your substitute to provide identification on-site.

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 administration fee of $295, or a pass to another event valid for two years from the date of issue.

Less than ten business days prior: A pass to a future event for either yourself or a colleague from the same company valid for two years from the date of issue.

To cancel and receive a refund or pass, please email Service@theconferenceforum.org or call 646-350-2580.

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VENUE / REGISTRATION CHIEF MEDICAL OFFICER SUMMIT

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EXECUTIVE SPONSORS

ASSOCIATE SPONSORS

EXHIBITOR

MEDIA PARTNERS

CHIEF MEDICAL OFFICER SUMMIT SPONSORS

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