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Kenneth Getz, MBADirector, Sponsored Research ProgramsTufts CSDD

DRUG DEVELOPMENT KEYNOTE

Organized by

3RD Annual

Chief Medical Officer Summit EastMAY 4 - 5, 2015 BATTERY WHARF HOTEL, BOSTON MACM

OEDriving and maintaining the appropriate balance between science, medicine & finance

Lee Allen, MD, PhDCMOSpectrum Pharmaceuticals

Executive Sponsor Supporting Sponsor

CHAIRCEO POV on CMOs

PHYSICIAN CEO KEYNOTE

Leslie Williams, MBACEOImmusanT

David Schenkein, MDCEOAgios

The Annual Event for Physicians in Life Sciences to:

✓ Address the unique challenges associated with directing and managing all R&D functions, while raising capital, and strategizing for appropriate exits.

✓ Create a network of CMOs from emerging life science companies to share ideas, solutions and support.

CSOs WelcomeToo!

Associate Sponsors

Follow us on Twitter @ConferenceForum #CMOE2015

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OVERVIEWThe Conference Forum’s Third 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 Role of a CMO in Emerging Life Science CompaniesThe role of a Chief Medical Officer is one that means many things to many people, and indeed 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, but with all the responsibility for driving clinical development and CRO management while meeting the needs of investors and positioning for an appropriate exit. 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, CSOs, 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,

Jennifer MoranConference Producer

Valerie BowlingExecutive Director

Jessica RothenbergMarketing Director

Elizabeth BardBusiness Development Manager

David BorrokBusiness Development Manager

Meredith SandsExecutive Director, Business Development

SPEAKING FACULTYLee Allen, MD, PhDCMOSpectrum Pharmaceuticals

Solomon BabaniGlobal Vice President, Alliance ManagementCovance Inc.

John Balser, PhDPresident, Founder and Chief BiostatisticianVeristat

Jonathan Behr, PhDEIR and Market Sector Leader, InnovationPartners Healthcare

Lew Bender, MBAFounder and CEOIntensity Therapeutics

David Berry, MD, PhDPartnerFlagship Ventures

Lou Brenner, MDSVP and CMOIdera Pharmaceuticals

David Clark, MDCMOWilson Therapeutics

Mark Corrigan, MDChairman of the BoardEpirus Biopharmaceuticals

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

Greg Fiore, MDCMOSSI Strategy

Barbara Fox, PhDCEOAvaxia Biologics

CHIEF MEDICAL OFFICER SUMMIT EAST OVERVIEW

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John Friend, MDSVP, R&DHelsinn Therapeutics

Kenneth Getz, MBADirector of Sponsored ResearchTufts CSDD

Vikas Goyal, MBAPrincipalSR One

Laurie Halloran, BSN, MSPresident & CEOHalloran Consulting Group

M. Scott Harris, MDCMOAvaxia Biologics, Inc.

Seth Hetherington, MDCMOGenocea Biosciences

John KnappManaging DirectorFTI Consulting

Rolf Linke, MDCMOSFJ Pharmaceuticals Group

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

Raj Malik, MDCMOG1 Therapeutics

Jeffrey Moore, DPhil, MBAPresidentMP Healthcare Venture Management (MPH)

Richard Moscicki, MDDeputy Center Director for Science OperationsFDA, CDER

Scott Orchard, JDSenior CounselJohnson & Johnson Innovation

Stephanie Plent, MBBSEVP, Chief Value OfficerThe Medicines Company

Horacio Plotkin, MDCMORetrophin

Al O. PacinoPresidentHealthCare Point

Jane Quigley, RN, BSN, OCNVP, Medical InformaticsPRA Health Sciences

Jim Roach, MDCMO and SVP, DevelopmentMomenta Pharmaceuticals

Oliver Rosen, MDCMODeciphera Pharmaceuticals

Mike Royal, MD, JD, MBAEVP, Clinical & Regulatory AffairsSorento Therapeutics

Lorraine Rusch, PhDVP, Business DevelopmentVInce & Associates

David Schenkein, MDCEOAgios

Oscar Segurado, MD, PhDDirectorMedicAffairs

Jeffrey Sherman, MD, FACPCMO and EVP, Research & DevelopmentHorizon Pharma

Joseph Stauffer, DO, MBACMOCara Therapeutics

Lynn SuttonVP, Clinical OperationsInSeption Group, LLC

Mary Szela, MBACEOMelinta Therapeutics

Samuel Wertheimer, PhDManaging DirectorPoliwogg

Leslie Williams, MBACEOImmusanT

Steven Zelenkofske, DO, FACC, FACOI, FCCPVP, Cardiovascular Medical Affairs Therapeutic Area HeadAstraZeneca

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SPEAKING FACULTY CHIEF MEDICAL OFFICER SUMMIT EAST

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DAY ONE - MONDAY, MAY 4, 20158:00 amRegistration & Coffee

8:45 amChair’s Opening RemarksLee Allen, MD, PhDCMO, Spectrum Pharmaceuticals

9:00 amKeynote: Characterizing the High Capitalized Cost of Drug Development and the Critical Drivers of R&D InefficiencyIn this annual keynote address Ken Getz, Director of Sponsored Programs at Tufts CSDD addresses:

• A review of the Tufts CSDD’s new R&D cost study results

• Root causes of inefficiencies, low success rates and high costs

• Major strategies and operating models transforming the R&D paradigm

• The value proposition, impact and challenges of these strategies and operating models

Kenneth Getz, MBADirector of Sponsored Programs, Tufts CSDD

9:45 amHow the Availability of Capital Impacts the Role of the CMOIn this session, the panelists cover:

• Optimal use of the proceeds

• Determining a development path, new molecules vs. new studies on the same molecules

• Appropriate stewardship of the capital

• Balancing short-term and long-term objectives; particularly when they may conflict

• Concerns and/or benefits of early financing without having reached clinical goals

• Examining the new threshold in moving development forward

• How is the progress in the industry translating into new drug discovery?

• Maintaining the same rigor for investment

Moderated by:Lou Brenner, MDSVP & CMO, Idera Pharmaceuticals

Panelists:Raj Malik, MDCMO, G1 Therapeutics

Jim Roach, MDCMO and SVP, Development, Momenta Pharmaceuticals

Oliver Rosen, MDCMO, Deciphera Pharmaceuticals

10:30 amNetworking Break

11:00 amBuilding Successful Trials in a Small CompanyIn this session, the panelists examine the positives and negatives of implementing design strategies and best practices in deploying them. More specifically:

• Adaptive design (Not just dose change)

• Regulatory POV on changes to trial design

• Navigating regulatory landmines

• Trial design strategies

• Determining endpoints

Moderator:Joseph Stauffer, DO, MBACMO, Cara Therapeutics

Panelists:John Balser, PhDPresident, Founder and Chief Biostatistician, Veristat

John Friend, MDSVP, R&D, Helsinn Therapeutics

Mike Royal, MD, JD, MBAEVP, Clinical & Regulatory Affairs, Sorrento Therapeutics

Lorraine Rusch, PhDVP, Business Development, Vince & Associates

Jeffrey Sherman, MD, FACPCMO & EVP, R&D, Horizon Pharma

11:45 amOptimizing Clinical Outsourcing for Small PharmaIn this session, the panelists cover:

• Current thinking on what is the best R&D operating model

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• Current thinking on risk sharing models

• How to engage in a deep dialogue to create strategy with partners and make sure goals are aligned to deliver for patients

• How to build a truly collaborative partnership in the confines of today’s market place that allows for execution of strategic plans beyond the bottom line

• What is important to the stakeholders (CEO, Wall Street, Customers, etc)

• Working collaboratively to constant regulatory changes

Moderated by:Lee Allen, MD, PhDCMO, Spectrum Pharmaceuticals

Panelists:Solomon BabaniGlobal Vice President, Alliance Management, Covance Inc.

Jeffrey Sherman, MD, FACPCMO & EVP, R&D, Horizon Pharma

Lynn SuttonVP, Clinical Operations, InSeption Group, LLC

12:15 pmKeynote: The Journey of a Physician CEOIn this session, gain an insight into Dr. David Schenkein’s journey from physician to CEO of a biopharmaceutical company leading the way in the field of cancer metabolism. More specifically:

• The journey

• Prioritizing resources

• Successes and failures

• Overcoming hurdles

• Strategic partnerships

• Business plan

David Schenkein, MDCEO, Agios

1:00 pmLuncheon

2:00 pmCapturing the Value of InnovationJane Quigley, RN, BSN, OCNVP, Medical Informatics, PRA Health Sciences

2:45 pmThe Cultural Shifts During the Growth of a Biotech: CMO as a Change AgentIn this session the panelists review the lifecycle of a biotech, from start-up through to commercialization and what these transitions mean for a CMO. More specifically:

• Navigating the growing pains:

• The “new departments on the block”

• New processes when going public

• Document management

• Increase in external communications

• The good, the bad and the ugly of social media

• Maintaining communications and that “small group” feel internally

• Strategies to alleviate growing pains

Moderated by:Seth Hetherington, MDCMO, Genocea Biosciences

Panelists:Lee Allen, MD, PhDCMO, Spectrum Pharmaceuticals

Lou Brenner, MDSVP and CMO, Idera Pharmaceuticals

M. Scott Harris, MDCMO, Avaxia Biologics, Inc.

Steven Zelenkofske, DO, FACC, FACOI, FCCPVP, Cardiovascular Medical Affairs Therapeutic Area Head, AstraZeneca

3:15 pmExploring Alternate Regulatory Pathways: an FDA & EMA UpdateThis session provides the latest trends and case examples on:

• Breakthrough designation

• Fast Track

• Priority Review

• Orphan Designations

• Accelerated Approval

• EMA’s pilot project on adaptive pathways

• Other initiatives to eary access to medicines

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

Richard Moscicki, MDDeputy Center Director for Science Operations, FDA

3:45 pmNetworking Break

4:00 pmWhy CMOs Have to be Prepared for Regulatory InspectionsThere are never clinical development programs without issues, which must either be prevented or fixed before regulatory inspections occur at the time of marketing submission. Preventing them ultimately will save both time and money, but in the incredibly busy time of late stage clinical trials, there is often little attention paid to what might not happen for a year or more. This session will address options to build in the problem solving and remediation through prospective activities.

• Infrastructure and resources to manage and prepare for regulatory inspections

• Offensive vs defensive program management and documentation

• What should you do if you’re already there to close the gaps

Laurie Halloran, BSN, MSPresident and CEO, Halloran Consulting Group

4:30 pmBiosimilars 101 for CMOs: Brief Overview, Hot Topics, and Implications for Your BusinessSince the Biologics Price Competition and Innovation Act (BPCIA) and the 351(k) biosimilar pathway was signed into law in 2010, the field of biosimilars has been exploding. In this session which can have broad implications for many companies, Dr. Jim Roach, SVP, Development and CMO, Momenta addresses the following:

• What is a biosimilar and what does the 351(k) pathway allow for?

• What is the commercial opportunity?

• What are the unique regulatory and development opportunities and challenges?

• Should “innovator” companies be supportive or afraid of biosimilar competition?

• Who are the stakeholders and what are they advocating for?

Jim Roach, MDCMO and SVP, Development, Momenta Pharmaceuticals

5:00 pmThe Shift in Focus of Orphan DrugsOrphan drugs have similar considerations which also apply to other therapeutic areas. Approvals for orphan drugs went up last year and there is no slow down in drug designation requests, which are on pace to break last year’s record This session addresses:

• Commercial and humanitarian

• Sustainability

• What are the internal pressures

• Value proposition

• Out-of-the-box thinking for approval process

• Challenges to get ODD

• Unmet medical need

• More favorable regulatory climate

• Pricing flexibility

• External pressures; patient advocacy groups & clinical KOL networks

Moderated by:Lee Allen, MD, PhDCMO, Spectrum Pharmaceuticals

Panelists:David Clark, MDCMO, Wilson Therapeutics

Horacio Plotkin, MDCMO, Retrophin

Jeffrey Sherman, MD, FACPCMO and EVP, R&D, Horizon Pharma, Inc.

5:30 pmNetworking ReceptionSponsored by:

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DAY TWO - TUESDAY, MAY 5, 20158:15 amCoffee

8:45 amChair’s Opening RemarksLee Allen, MD, PhDCMO, Spectrum Pharmaceuticals

9:00 amCEO Panel: Striking a Balance with your CEOThis panel explores the balanced working relationship necessary for product and company success. More specifically:

• How to engage with your CEO

• What does a CEO look for in a CMO?

• Managing the board

• Boundaries and scope in decision-making

• How best to utilize consultants and manage them

Moderated by:Lee Allen, MD, PhDCMO, Spectrum Pharmaceuticals

Jim Roach, MDSVP, Development and CMO, Momenta Pharmaceuticals

Panelists:Lew BenderFounder and CEO, Intensity Therapeutics

Mark Corrigan, MDChairman of the Board, Epirus Biopharmaceuticals

Barbara Fox, MDCEO, Avaxia

Mary Szela, MBACEO, Melinta Therapeutics

Leslie Williams, MBACEO, ImmusanT

9:45 amHow Investors Assess ManagementIn this session, we begin with a presentation on a perspective on the method of evaluating a disease space, which includes a comprehensive understanding of 1) the science, 2) the competitive landscape and 3) the management teams for each company. How does this get mapped out? Following will be more discussion on:

• Understanding the context of value proposition

• Strategic fit

• Processes used to evaluate management and an understanding of competitive strategy

• How best to convey your plans

• How best to communicate with investors (before investment) and boards (afterwards) – via the CEO or directly?

• Importance of clinical development plan expectations

Moderated by:Peter Kolchinsky, PhDManaging Director, RA Capital Management, LLC

Panelists:Jonathan Behr, PhDEIR and Market Sector Leader, Innovation, Partners Healthcare

David Berry, MD, PhDPartner, Flagship Ventures

Vikas Goyal, MBAPrincipal, SR One

Jeffrey Moore, DPhil, MBAPresident, MPH Venture Management

10:30 amNetworking Break

11:00 amAlternative Investment and Partnering ModelsRolf Linke, MDCMO, SFJ Pharmaceuticals Group

Samuel Wertheimer, PhDManaging Director, Poliwogg

11:30 amLarge Pharma Business Development POV on Partnering with BiotechsIn this session we address:

• Early engagement with large pharma

• Identify the key value drivers

• What do large pharma look for from the CMO

• What makes it worthwhile for large pharma

• Best practices and changes during a transition period

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

Daniel Curran, MD, MBAVP, Value Creation Therapeutic Areas, Takeda Pharmaceutical Company (invited)

John KnappManaging Director, FTI Consulting

Scott Orchard, JDSenior Counsel, Johnson & Johnson Innovation

12:30 pmExecuting a Reimbursement StrategyIn this session, we address:

• Leveraging a payor advisory board

• Conducting health economic studies in parallel with regulatory clinical trials

• Planning a reimbursement strategy in early stages of clinical development

Stephanie Plent, MBBSEVP, Chief Value Officer, The Medicines Company

Oscar Segurado, MDDirector, MedicAffairs

1:00 pmQ&A with BIOCartier Esham, EVP, Emerging Companies at BIO speaks with our Chair, Lee Allen on what the organization are doing to represent the industry in Washington. We explore how BIO are tackling tax reform and 21st century cures in support of emerging biotechs.

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

with

Lee Allen, MD, PhDCMO, Spectrum Pharmaceuticals

1:30 pmWhat Does a Responsible Exit Look Like?Companies with ‘hot’ assets may be courted by multiple potential acquirers during the process of exit. With well-placed effort, company leaders can ensure best likelihood of benefit for their patients by following a few simple steps. This session will touch on the following, using case study methodology:

• Selecting the best partners during development

• Performing appropriate diligence on potential acquirers

• Ensuring that pre-acquisition commitments are fulfilled post-acquisition to investigators, health authorities, patient advocacy groups and patients

• Positioning the acquiring entity for success

Lee Allen, MD, PhDCMO, Spectrum Pharmaceuticals

Greg Fiore, MDCMO, SSI Strategy

2:30 pmConference Concludes

VENUEBattery Wharf HotelThree Battery WharfBoston, MA 02109617-994-9000

www.batterywharfhotelboston.com

To make a reservation, please call 866-235-7555 and ask for the “Chief Medical Officer Summit” group rate or visit https://aws.passkey.com/event/13814455/owner/81422/home.

The centerpiece of Boston’s Battery Wharf renaissance, The Battery Wharf Hotel, Boston Waterfront merges the North End’s trademark charm with a contemporary appreciation for history and sense of place. Featuring 150 stylishly designed guest rooms and suites with extraordinary waterfront views, this luxurious boutique hotel on the pedestrian-friendly HarborWalk boasts two bars and restaurants, 6,000 square feet of meeting space, an award-winning Spa and Fitness Center and easy access to Boston’s bustling Financial District, Freedom Trail, Faneuil Hall Marketplace and TD Garden, New England’s premier sports and entertainment arena.

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REGISTRATIONEarly-Bird - before March 28, 2015 $1,395

Standard EarlyBird - before April 17, 2015 $1,495

Standard - after April 17, 2015 $1,595

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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REGISTRATION / SPONSORS & PARTNERS CHIEF MEDICAL OFFICER SUMMIT EAST

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