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CHIEF MEDICAL OFFICER SUMMITTHE FIRST WEST COAST EVENT ADDRESSING THE COMPLEX DUAL ROLE OF

MANAGING R&D WHILE RAISING CAPITAL FOR BIOTECHS

NOVEMBER 4 - 5, 2013 MARINES’ MEMORIAL HOTEL, SAN FRANCISCO, CA

DESIGNED FOR:

✓ Chief Medical Officers, Heads of R&D/Clinical

Operations, Chief Scientific Officers

✓ Life Science Investors, Government Sources of

Capital & Passion/Patient Capital

NETWORKING ACCESS WITH:

✓ Life Science Investors

✓ Professionals who sit at the crux of drug

development, establishing proof of concept,

managing safety, planning phase 3, interacting

with regulatory bodies and driving corporate value

BEST PRACTICES ON DAY ONE:

Clinical Development/Outsourcing & Regulatory

Management in the Emerging Life Science Space

BEST PRACTICES ON DAY TWO:

Investment Strategies, Exit Planning & Trends that will

Change the Funding of Medical Research in the

Emerging Life Science Space

CO-CHAIR: REGULATORY STRATEGYLee Allen, MD, PhDCMOSpectrum Pharmaceuticals

THE CMO’S ROLE IN RAISING CAPITALSrinivas Akkaraju, MD, PhDGeneral PartnerSofinnova Ventures

OPENING ADDRESS: THE REALITY OF THE CMO ROLETyler Martin, MDFounder, CEO & CMOGreat Plains Biotechnology

CO-CHAIR: RESOURCEFUL OUTSOURCINGSheila Gujrathi, MDCMOReceptos

Organized by

Executive Sponsor Associate Sponsor

Follow us on Twitter @ConferenceForum #CMOSummit

Supporting Partners

Participating Investors

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With the huge success of Chief Medical Officer Summit East, we are thrilled to announce the launch of the Chief Medical Officer Summit for Emerging Life Science Companies on the West Coast! The Chief Medical Officer Summit West will take place in San Francisco on November 4-5, 2013 at the Marines’ Memorial Club & Hotel.

For the first time, Chief Medical Officers at biotechs on the West Coast have their own conference thanks to the idea from Dr. Elizabeth Stoner, Managing Director, MPM Capital. Valerie Bowling, Executive Director, The Conference Forum, the organizer of the event, says, “We are grateful to Dr. Stoner’s idea for the conference and her insights into the unique challenges CMOs face every day at small to mid-size biotechs.”

CMO West is dedicated to accomplishing two goals:

• To provide a forum to address the unique needs of Emerging Life Science Chief Medical Officers in de-risking drug development, selecting and managing CROs, leveraging capital to manage all R&D functions with limited resources, working more successfully with investors and strategizing for appropriate exits in the current environment.

• To create a network of Life Science CMOs to form a community that can 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 the clinical development and CRO management while meeting the needs of investors and positioning for an appropriate exit. Chief Medical Officers 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 life science investors are encouraged to attend as conversations with CMOs are very much welcomed.

Srinivas Akkaraju, MD, PhDGeneral PartnerSofinnova Ventures

Lee Allen, MD, PhDCMOSpectrum Pharmaceuticals

Zoran AntonijevicSenior Director, Strategic Consulting and Adaptive ImplementationCytel Inc.

Mohammad Azab, MD, MBACMOAstex Pharmaceuticals

Hal Barron, MDCMO & EVP, Head of Global Product DevelopmentGenentech

Jill CarrollSenior AssociateSR One

Tom Cervantez, JD, MBAPresident, Harvard AngelsManaging Director, Golden Gate Angels Group andPartner, Business Counsel Law Group

Farah Champsi, MBAManaging DirectorAlta Partners

Adam Craig, MD, PhD, MBAEVP, Development & CMOSunesis Pharmaceuticals, Inc.

Howard Dittrich, MDCMOSorbent Therapeutics

Paul Grint, MDPresidentCerexa

Sheila Gujrathi, MDCMOReceptos, Inc.

Barry Hafkin, MDCMOAffinium Pharmaceuticals

CHIEF MEDICAL OFFICER SUMMIT WEST OVERVIEW

OVERVIEW

2

SPEAKING FACULTY

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Laurie Halloran, BSN, MSPresident & CEOHalloran Consulting Group, Inc.

Julie Hambleton, MDSVP & CMOFive Prime Therapeutics

M. Scott Harris, MDCMO, Avaxia Biologics, Inc.Principal, Middleburg Consultants and Clinical Professor of Medicine, Georgetown University School of Medicine

Rekha Hemrajani, MBAVP, Head of Licensing & M&AOnyx Pharmaceuticals

Colin Hislop, MDCMOAnthera Pharmaceuticals

Jan Johansson, MD, PhDSVP, Medical AffairsResverlogix

Jeff JonkerChief Business OfficerSatori Pharmaceuticals

Lori Kunkel, MDCMOPharmacyclics

Jeffery Loutit, MBChBCMORempex Pharmaceuticals

Jonathan MacQuitty, PhD, MBAPartnerAbingworth

Kurt Marek, PhDProgram Director and SBIR CoordinatorNational Heart, Lung, and Blood Institute, NIH

Tyler Martin, MDFounder, CEO & CMOGreat Plains Biotechnology

Nestor Molfino, MDCMOKaloBios Pharmaceuticals, Inc.

Pamela Palmer, MD, PhDCMO & Co-FounderAcelRx Pharmaceuticals, Inc.

Hemai Parthasarathy, PhDScientific Director, Breakout LabsThe Thiel Foundation

Mike Royal, MD, JD, MBAChief, Clinical AffairsAcelRx Pharmaceuticals, Inc.

Chris Sasiela, PhD, RACRegulatory SpecialistNational Heart, Lung, and Blood Institute, NIH

Tracy Saxton, PhDAssociateSV Life Sciences Advisers LLP

Oscar Segurado, MD, PhDCMOCrescendo Bioscience

Jeffrey Sherman, MD, FACPCMO & EVP, Development, Manufacturing and Regulatory AffairsHorizon Pharma

Stephen Shrewsbury, MDSVP, Clinical Development & CMOAquinox Pharmaceuticals

Michael Sweeney, MDCMO & VP of R&DDepomed

Frank Torti, MDPrincipalNEA

Santosh Vetticaden, MD, PhDCMO & SVPMast Therapeutics, Inc.

Samuel Wu, MD, PhDManaging DirectorMedImmune Ventures

Steven Zelenkofske, DO, FACC, FACOI, FCCPSVP, Clinical and Medical Affairs & CMORegado Biosciences

3

SPEAKING FACULTY CHIEF MEDICAL OFFICER SUMMIT WEST

The Conference Forum is grateful to its advisers and speaking faculty for their insights on the agenda development and overall support.

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Clinical Development/Outsourcing and Regulatory Management

8:00 amRegistration & Breakfast

8:45 amCo-Chairs’ Welcome

Lee Allen, MD, PhDCMO, Spectrum Pharmaceuticals

Sheila Gujrathi, MDCMO, Receptos, Inc.

9:00 amOpening Address: What is a CMO? Defining the Reality of the RoleHow does the decision making for a CMO play out in different organizations? What people typically envision when they think of a CMO and how that stacks up against the reality of the role will be addressed. Also covered in this session are the challenges and what the trade-offs are. Other topics to address include:

• Supporting a CEO who does not have experience in the therapeutic area you are in charge of

• Functioning with restrictive capital

• What should a CMO be focused on?

• The great balancing act

Tyler Martin, MDFounder, CEO & CMO, Great Plains Biotechnology

9:30 amKeynoteHal Barron, MDCMO & EVP, Head of Global Product Development, Genentech

10:20 amNetworking Break

10:50 amClinical Trial Design Part I: Considerations and Risk Management

• Balancing trial design cost and complexity with budget and timeline

• Reducing the cost and complexity with novel clinical trials

• How much science can you afford?

• Enrichment strategies

• Optimizing the pharmacovigilance program: finding early safety signals

Mohammad Azab, MD, MBACMO, Astex Pharmaceuticals

Santosh Vetticaden, MD, PhDCMO & SVP, Mast Therapeutics, Inc.

Steven Zelenkofske, DO, FACC, FACOI, FCCPSVP, Clinical and Medical Affairs & CMO, Regado Biosciences

Part II: The Role of Adaptive Trials in Optimizing Drug Development

• Brief catch-up on adaptive trials: what adaptive trial design is, how it is useful, how it is being perceived, how to implement it and barriers to it

• Quantifying the trial design’s impact on the value of a product

• FDA’s current stance on adaptive approaches in Phases 2 and 3

• Use of simulations in determining the optimal development strategy

• What precisely is “Model-assisted Drug Development” and how is it working?

Zoran AntonijevicSenior Director, Strategic Consulting and Adaptive Implementation, Cytel Inc.

Adam Craig, MD, PhD, MBAEVP, Development & CMO, Sunesis Pharmaceuticals, Inc.

M. Scott Harris, MDCMO, Avaxia Biologics, Inc., Principal, Middleburg Consultants and Clinical Professor of Medicine, Georgetown University School of Medicine

11:55 amInnovative Outsourcing Strategies, Models & Options with CROs

• Assessing clinical outsourcing partnering models that work best for biotech companies

• What CRO partnering models are available to biotechs and what makes the right fit?

• What are the core competencies that you should keep in-house vs. outsource? How does it change based on company size?

CHIEF MEDICAL OFFICER SUMMIT WEST AGENDA

4

DAY ONE - MONDAY, NOVEMBER 4, 2013

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• How do you ensure the team assigned stays on your program? Are there contractual ways you can lock in the original team? What do you do if there is turnover?

• How do you do the due diligence on your vendors and how are the roles and responsibilities defined in a fully outsourced program?

• What do you do to manage the scope and to mitigate risks with out of scope as quickly as possible?

• How are CROs thinking about longer-term partnerships and biotechs as an investment?

• Are you considering entering into risk-sharing partnerships with CROs? Why or why not?

• If there are risk-sharing ventures through the outsourcing of multiple programs with CROs, how do you hold on to leverage?

• Have you ever transitioned to another vendor during a program?

• The differing role of the CMO in virtual vs. fully integrated development team

• Pros and cons of working with small vs. large CROs

• How do you work with CROs so the dynamic between enrollment and cash flow challenges are fully transparent to both parties?

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

Panelists:Howard Dittrich, MDCMO, Sorbent Therapeutics

Sheila Gujrathi, MDCMO, Receptos, Inc.

Colin Hislop, MDCMO, Anthera Pharmaceuticals

Stephen Shrewsbury, MDSVP, Clinical Development & CMO, Aquinox Pharmaceuticals

1:00 pmLuncheon

2:15 pmHow to Position Your Drug For Approval and Regulatory Management

• Positioning your drug to get through regulatory processes as quickly and efficiently as possible to balance European and U.S. authorities

• Writing thoughtful protocols, investigative brochures and clinical development plans; adopting standard procedure on monitoring; writing SOPs; and dealing with regulatory agencies

• How to meet with FDA pre-IND for higher likelihood for success

• How to prepare for end of phase I and end of phase II meetings

• Hurdles standing in way of going through product approval

• How to manage regulatory requirements for Patient Reported Outcomes

• Accelerated approval and innovative ways to get to the finish line: what are relevant endpoints?

• The utility of the orphan drug process

• How to correctly use consultants

• The overlap and potential disconnect occurring between the right medical practice and clinical study

• Post-approval: CMO responsibility for promotional/educational compliance; promotional oversight and management of the medical message

• Do’s and Don’t's

Lee Allen, MD, PhDCMO, Spectrum Pharmaceuticals

Jan Johansson, MD, PhDSVP, Medical Affairs, Resverlogix

Lori Kunkel, MDCMO, Pharmacyclics

Jeffery Loutit, MBChBCMO, Rempex Pharmaceuticals

Jeffrey Sherman, MD, FACPCMO & EVP, Development, Manufacturing and Regulatory Affairs, Horizon Pharma

Michael Sweeney, MDCMO & VP of R&D, Depomed

5

AGENDA CHIEF MEDICAL OFFICER SUMMIT WEST

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3:00 pmSecuring a Development Partnership with Large Pharma

• What are the five big trends big pharma companies want to see right now (novel targets vs. differentiated therapies) and in what stages of development?

• How to position yourself with a large pharma partner

• Large pharma expectations

• Presenting clinical data

• Communication between companies

• Assessment of roles and responsibilities in a partnering arrangement

• Opportunities to pool or share resources

Lee Allen, MD, PhDCMO, Spectrum Pharmaceuticals

Paul Grint, MDPresident, Cerexa

Sheila Gujrathi, MDCMO, Receptos, Inc.

Barry Hafkin, MDCMO, Affinium Pharmaceuticals

Julie Hambleton, MDSVP & CMO, Five Prime Therapeutics

3:45 pmNetworking Break

4:00 pmGetting Your Drug Reimbursed

• Reimbursement strategy planning

• What type of data package to develop to be attractive to a large pharma from a payor perspective

Speaker TBA

4:30 pmRoundtable Session Choices1. Innovative Outsourcing Strategies

• How to use outsourcing to provide access to novel planning and design capabilities

• Stratifying patient populations and engaging in novel partnerships to develop biomarkers

• Outsourcing with payers and providers

• Leveraging commercial services earlier into clinical

development and partnerships after regulatory approval

Speaker TBA

2. Acquiring Orphan Designation/Benefits and the Jounrey• Medical Subsets or “What is the disease?”

• The tangible and non-tangible benefits of orphan designation

• Where to find prevalence numbers

• FDASIA implications for rare diseases

Julie Hambleton, MDSVP & CMO, Five Prime Therapeutics

Stephen Shrewsbury, MDSVP, Clinical Development & CMO, Aquinox Pharmaceuticals

3. Raising Capital• Why venture capital? The pros/cons

• Understanding the venture model (what motivates investors) and the different types of LPs who invest in GPs

• What VCs look for in CMOs

• Defining value-creating milestones – focusing on the exit and the budget to get there and how to build your company with an exit in mind

• Winning venture capital and managing a board of venture capitalists are two very different beasts

• Communication – setting clear expectations for the Board of Directors

• Moving goalposts – how to mitigate regulatory and financing risk

• Latest technology/investment trends to note

• Outlook for fundraising in 2014

Srinivas Akkaraju, MD, PhDGeneral Partner, Sofinnova Ventures

Jill CarrollSenior Associate, SR One

Frank Torti, MDPrincipal, NEA

5:30 pmNetworking Reception

CHIEF MEDICAL OFFICER SUMMIT WEST AGENDA

6

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The Role of the CMO in Raising Capital, Meeting the Needs of Investors and Exit Planning

8:00 amBreakfast

8:30 amCo-Chairs’ Welcome

Lee Allen, MD, PhDCMO, Spectrum Pharmaceuticals

Sheila Gujrathi, MDCMO, Receptos, Inc.

8:45 amAdvising Your CEO on Scientific Advisory Board Development

• Finding experts for advisory boards who specifically can address clinical and sometimes regulatory issues

• How can you inter-face with advisors who know fields for science and medicine to design clinical trials?

• How can you minimize cost and maximize time to get a go/no-go decision?

Oscar Segurado, MD, PhDCMO, Crescendo Bioscience

9:15 amHow to Best Portray the Science in Raising Capital

• The role and expectation of the CMO in raising capital

• Knowing what investors are looking for: the technical likelihood of success, having a strong patent around the technology, the exit strategy, opportunity cost

• What's being done to mitigate risk and increase the probability of success; having a clearly differentiated product

• Communicating and portraying what is compelling about the science to non-scientists and the many types of investors and funding agencies

Mohammad Azab, MD, MBACMO, Astex Pharmaceuticals

Sheila Gujrathi, MDCMO, Receptos, Inc.

Nestor Molfino, MDCMO, KaloBios Pharmaceuticals, Inc.

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

Jeffrey Sherman, MD, FACPCMO & EVP, Development, Manufacturing and Regulatory Affairs, Horizon Pharma

10:00 amWhat a CMO Can Do to Secure Alternative Sources of Financing: Alternative Investors’ Perspectives

Part I – The National Institutes of Health: A Source for Non-Dilutive Funding and Product Development Resources

Panelists from the National Heart, Lung, and Blood Institute will describe how NIH resources and non-dilutive funding can facilitate the research, development, and clinical activities of your small biomedical business.

• NIH small business programs that can provide millions of dollars in funding and in-kind resources to advance your research and product development needs

• NIH product development, manufacturing, regulatory, and clinical trial resources

• NIH programs designed to incentivize private investors and to facilitate small business collaborations with academic institutions

• How can a strong commercialization plan complement your research plan?

• Eligibility requirements and tips to strengthen your small business applications

Kurt Marek, PhDProgram Director and SBIR Coordinator, National Heart, Lung, and Blood Institute, NIH

Chris Sasiela, PhD, RACRegulatory Specialist, National Heart, Lung, and Blood Institute, NIH

Part II – Breakout Labs & Angel Investment

Breakout Labs, a new model of philanthropic support for early-stage companies, is a revolving-fund within the Thiel Foundation that provides milestone-driven funding for scientific and technical de-risking.

• After receiving Breakout Labs funding, high-risk companies pursue multiple strategies to finance their next stages: non-dilutive government grants, seed rounds, and even revenues

7

AGENDA CHIEF MEDICAL OFFICER SUMMIT WEST

DAY TWO - TUESDAY, NOVEMBER 5, 2013

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• Breakout Labs provides more than just funding: they help build relationships with potential strategic partners and follow-on investors

• Angel Investors: rushing in where venture capitalists fear to tread; using angels for funding de-risking round to make you more attractive to VCs

Tom Cervantez, JD, MBAPresident, Harvard Angels, Managing Director, Golden Gate Angels Group and Partner, Business Counsel Law Group

Hemai Parthasarathy, PhDScientific Director, Breakout Labs, The Thiel Foundation

10:40 amNetworking Break

11:00 amWhat a CMO Can Do to Secure Traditional Sources of Financing: VC and Corporate Venture Investors’ Perspectives

What venture and corporate venture investors look for when funding the emerging life sciences and what a CMO can do that will contribute to the financing effort when thinking about trial design, choice of indications, and even the supporting science.

• Understanding the business of raising capital, where the target profile needs to be in order to be attractive to pharma, what the product needs to look like and what makes it acquirable as a CMO

• Aside from world-class science, world-class scientists, and unmet needs, what the difference is between companies funded and companies unfunded

• Getting involved as early as possible and building a relationship with the board by being at board meetings, presenting the plan and updating them on what you’re thinking about clinical development as a CMO

• How CMOs can be more involved and build relationships with VCs

• Being involved in presentations and making sure they include real plans for clinical development and budget

• How a CMO can contribute to the company and not just the financing process

Farah Champsi, MBAManaging Director, Alta Partners

Jonathan MacQuitty, PhD, MBAPartner, Abingworth

Tracy Saxton, PhDAssociate, SV Life Sciences

Samuel Wu, MD, PhDManaging Director, MedImmune Ventures

11:45 amPlanning for the Future: Exits & Sustainability Strategies

• How do you position for an appropriate exit?

• How do you develop a clinical plan to match your exit strategy?

• Creating fluid strategies and building a compelling database

• Building a data package for an acquirer

• How to position for exits in both public and private companies

• How the exit environment has changed

• What are the contingency plans in place?

• How to prepare from the beginning

• Planning for sustainability: looking at all the options

Rekha Hemrajani, MBAVP, Head of Licensing & M&A, Onyx Pharmaceuticals

Jeff JonkerFormer Chief Business Officer, Satori Pharmaceuticals

12:30 pmDriving Investor Interest with External Communications

• Managing external communications

• Balancing the business need of maintaining a funding stream and/or stock prices with maintaining scientific integrity and not over-reporting data

• When and how should you get data out there? Press releases to general public, academic meetings, industry sponsored meetings, peer-reviewed publications

• Early vs. later disclosure and nature of disclosure

• How to handle when data is not worthy of a press release or the strength of statement does not support data to drive investor interest

• Managing investors

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

1:00 pmConference Concludes

CHIEF MEDICAL OFFICER SUMMIT WEST VENUE / REGISTRATION

8

Marines’ Memorial Club & Hotel609 Sutter Street San Francisco, CA 94102www.marineclub.com

For reservations, call 800-562-7463 or 415-673-6672 and ask for the CMO Summit group rate. You may also email your reservations to reservations@marineclub.com.

Marines’ Memorial Club’s 1920s Beaux-Arts building is located in the Union Square neighborhood and combines a blend of classic with modern. Ideal for leisure and business travel with sophisticated accommodations and beautiful meeting space.

Early-Bird - before September 6, 2013 $1,295

Standard - after September 6, 2013 $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 order.

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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SPONSORS CHIEF MEDICAL OFFICER SUMMIT WEST

EXECUTIVE SPONSOR

ASSOCIATE SPONSOR

SUPPORTING PARTNERS

PARTICIPATING INVESTORS

MEDIA PARTNERS

VENUE

REGISTRATION