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

2ND Annual Chief Medical Officer Summit West NOVEMBER 3 - 4, 2014 MARINES’ MEMORIAL CLUB & HOTEL, SAN FRANCISCO, CA CMOW

KEYNOTE FEATURED PRESENTATION KEYNOTE Jeffrey Tobias, MD Thomas Lonngren, PhD Jakob Dupont, MD (hc), MRPharmS, FRCP OncoMed EMA

CO-CHAIR CO-CHAIR MODERATOR MODERATOR Lee Allen, MD, PhD Pamela Palmer, MD, PhD Julie Hambleton, MD Laurie Halloran, BSN, MS Spectrum AcelRx Pharmaceuticals Five Prime Therapeutics Halloran Consulting Pharmaceuticals Group

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REGISTER AT WWW.THECONFERENCEFORUM.ORG OR CALL 646-350-2580 CHIEF MEDICAL OFFICER SUMMIT WEST OVERVIEW

OVERVIEW SPEAKING FACULTY

The Conference Forum’s 2nd Annual Chief Medical Lee Allen, MD, PhD Officer Summit West for Emerging Life Science CMO Companies is delighted to present a truly distinctive and interactive event for CMOs. We are grateful to Dr. Elizabeth Joseph Arcangelo Stoner, Managing Director, MPM Capital, for suggesting Managing Partner the idea, and to our CMO advisors for their insights on InSeption Group content and agenda development.

We are dedicated to accomplishing two goals: Martin Babler 1. To bring together CMO executives to address the unique CEO challenges associated with directing and managing all R&D Principia Biopharma Inc. functions with limited resources, while raising capital, Chuck Boggs working and meeting with investors, and strategizing for Partner appropriate exits. Paladin Ventures

2. To create a network of CMOs from small to midsize life Nathaniel Brown, MD science companies to share ideas, solutions and support. CMO The Role of a CMO in Emerging Life Science Companies Presidio Pharmaceuticals We understand that the role of a Chief Medical Officer is Leon Chen, PhD dramatically different depending on the type of Venture Partner organization in which a CMO works. For a CMO in early, OrbiMed Advisors emerging and even midsize life science companies, the role can be an isolated one. In addition to the responsibility of Adam Craig, MD, PhD, MBA driving clinical development and CRO management, CMOs EVP, Development & CMO also need to meet the needs of investors and help position Sunesis Pharmaceuticals, Inc. for an appropriate exit. We are seeing many CMOs play an Brian Culley, MA, MBA increasingly larger role in raising funding for their life CEO & Director science companies. All these challenges are addressed at Mast Therapeutics, Inc. the Summit. We look forward to greeting you on November 3rd in San Jakob Dupont, MD Francisco. SVP & CMO OncoMed Sincerely, Mike Dybbs, PhD Principal New Leaf Venture Partners

Valerie Bowling Cartier Esham, PhD Executive Director EVP, Emerging Companies Biotechnology Industry Organization (BIO)

Greg Fiore, MD CMO Kristen Pisanelli SSI Strategy Conference Producer Steven Gourlay, MBBS (MD), PhD, FRACP, MBA CMO Principia Biopharma Inc.

Barry Hafkin, MD Jessica Rothenberg CMO Marketing Director Affinium Pharmaceuticals

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Laurie Halloran, BSN, MS Pamela Palmer, MD, PhD President & CEO CMO & Co-Founder Halloran Consulting Group, Inc. AcelRx Pharmaceuticals, Inc.

Julie Hambleton, MD Jay Parrish, PhD, MBA SVP & CMO Associate Director, Corporate Development Five Prime Therapeutics

Scott Harris, MD Lorraine Rusch, PhD CMO, Principal VP, Business Development Avaxia Biologics Vince & Associates

Colin Hislop, MD William Schwieterman, MD CMO & SVP Former FDA Branch Chief & CMO Anthera Pharmaceuticals Chelsea Therapeutics

Marc Hoffman, MD Oscar Segurado, MD, PhD SVP & General Manager, Development CMO Theorem Clinical Research Crescendo Bioscience/

Richard King Jaisim Shah CEO, President & Acting Chief Commercial Officer CEO AcelRx Pharmaceuticals Semnur Pharmaceuticals

Lori Kunkel, MD Jeffrey Sherman, MD, FACP CMO CMO & EVP, Development, Manufacturing and Regulatory Loxo Oncology Affairs Horizon Pharma Paul Kwon, MD CMO Stephen Shrewsbury, MD Nora Therapeutics SVP, Clinical Development & CMO Aquinox Pharmaceuticals Thomas Lonngren, PhD (hc), MRPharmS, FRCP Former Head Jeffrey Spaeder, MD, MBA EMA Chief Medical & Scientific Officer Quintiles Tyler Martin, MD CEO Jeffrey Tobias, MD Adjuvance Technologies and Great Plains EVP, R&D & CMO Biotechnology Jazz Pharmaceuticals

Ben McGraw Samuel Wu, MD, PhD VP, Marketing Managing Director Comprehend Systems MedImmune Ventures

Mark Monane, MD, MS Steven Zelenkofske, DO, FACC, FACOI, FCCP CMO SVP, Clinical and Medical Affairs & CMO CardioDx, Inc. Regado Biosciences

Lisa Murtha, JD, CHC, CHRC Senior Managing Director FTI Consulting The Conference Forum is grateful to its advisers and Jonathan Norris speaking faculty for their insights on the agenda Managing Director, Healthcare Practice development and overall support. Silicon Valley Bank

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William Schwieterman, MD DAY ONE - MONDAY, NOVEMBER 3, 2014 Former FDA Branch Chief & CMO, Chelsea Therapeutics 8:00 am Jeffrey Sherman, MD, FACP Registration & Breakfast CMO & EVP, Development, Manufacturing and Regulatory Exclusive Conference Registration Host: Affairs, Horizon Pharma

10:00 am Featured Presentation: Strategies to Position your Drug for Regulatory Approval and Market Access in Europe • The EU Landscape 8:45 am • What are the regulators looking for? Co-Chairs’ Opening Remarks: Defining the Role of the CMO • What are the payers looking for? Lee Allen, MD, PhD • Strategies to successfully navigate your product into CMO, Spectrum Pharmaceuticals the European market Pamela Palmer, MD, PhD Thomas Lonngren, PhD (hc), MRPharmS, FRCP CMO & Co-Founder, AcelRx Pharmaceuticals, Inc. Former Head, EMA

10:30 am 9:00 am Networking Break Keynote: The Jazz Pharmaceutical Turn Around Case Study 11:15 am • The journey of Jazz Pharmaceuticals Clinical Development Planning Based on Business Objectives • Managing massive growth Emerging biotechs have a unique opportunity to set the • Scaling up operations “right” framework for the company. Careful thought should • Lessons learned be given when determining what areas are unique and there is no “one size fits all”. Jeffrey Tobias, MD EVP, R&D & CMO, Jazz Pharmaceuticals Key areas to consider: • Strategic development planning (When?)

9:15 am • Resourcing and Functional Area Expertise (Who?) Q&A • Operational and Systems Infrastructure (What?)

9:30 am • Program Leadership (How?) Best Practices to Position your Drug for Regulatory Moderator: Approval Laurie Halloran, BSN, MS • What is the FDA looking for? President & CEO, Halloran Consulting Group, Inc. • De-risking programs Panelists: • How to ensure a clinical program has the broadest Steven Gourlay, MBBS (MD), PhD, FRACP, MBA geographic applicability CMO, Principia Biopharma Inc. • What are all the different regulatory options for Stephen Shrewsbury, MD registration SVP, Clinical Development & CMO, Aquinox Pharmaceuticals • Breakthrough designation experience, what worked and what did not work • Best practices on how to work with the FDA

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11:45 am 12:45 pm Data Driven Approaches for the Design of a More Luncheon Efficient Clinical Study 1:10 pm In this session, increase the probability of success in your Luncheon Roundtables clinical study through understanding how to manage real world data including quantitative data from patients In this luncheon session, attendees can choose an optional themselves. topic to gain best practices and share helpful ideas on the following: Also addressed: 1. Setting up and Managing a Virtual Company • What approaches are organizations taking to reinvent • Challenges and Opportunities study design (e.g., feasibility assessment, adaptive designs, single arm trials)? • Infrastructure needs/Creating virtual platforms • Exploring new approaches to gather strategic • Publication management information informing study design • Setting up SOPs, templates Jeff Spaeder, MD, MBA • What do you really need close to you and what can Chief Medical & Scientific Officer, Quintiles you manage virtually? with • Managing without a bench Barry Hafkin, MD CMO, Affinium Pharmaceuticals • Safety issues Lori Kunkel, MD • HR issues CMO, Loxo Oncology • Ethical issues

Lorraine Rusch, PhD Joe Arcangelo VP, Business Development, Vince & Associates Managing Partner, InSeption Group

12:15 pm Greg Fiore, MD Determining the Optimal Development Path: Early CMO, SSI Strategy Phase Tools and Techniques 2. Best Practices for Interacting with the Board • Modern techniques/tools to help determine best Oscar Segurado, MD, PhD development path CMO, Crescendo Bioscience / Myriad Genetics • Mitigating development risk using adaptive approaches, model-building, biomarker incorporation 3. Career Development Opportunities for CMOs • View on the life cycle of a CMO • Determining program/portfolio valuation for more quantitative decision-making • What opportunities are there for CMOs? • Finding efficient early phase data review techniques • Founder CMOs: how does that get translated into for faster medical review advancement Panelists: • Venture POV Adam Craig, MD, PhD, MBA Steven Zelenkofske, DO, FACC EVP, Development & CMO, Sunesis Pharma SVP, Clinical & Medical Affairs & CMO, Regado Scott Harris, MD Biosciences CMO, Principal, Avaxia Biologics 2:00 pm Colin Hislop, MD Working through Complex Contracts CMO and SVP, Anthera Pharmaceuticals Inc. Contracting is evolving to be even more complex and can Ben McGraw seriously delay clinical trials. To understand what is causing VP of Marketing, Comprehend Systems this and how to deal up front will reduce some of the

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complexity and help with the structure and approach to Tyler Martin, MD DSMBs, sites, hospitals and CROs. CEO, Adjuvance Technologies and Great Plains Biotechnology More specifically: Stephen Shrewsbury, MD • How new regulations are impacting contracting SVP, Clinical Development & CMO, Aquinox • How risk levels are impacting efficient contracting Pharmaceuticals F. Lisa Murtha, JD, CHC, CHRC 3:10 pm Senior Managing Director, FTI Consulting Networking Break Steven Zelenkofske, DO, FACC SVP, Clinical & Medical Affairs & CMO, Regado 3:35 pm Biosciences Data Communications/Publication Strategy: Objectives, Organization and Process 2:30 pm • Data communication strategy and the role of the Optimizing Clinical Outsourcing as an Emerging CMO Biotech with Fixed Funds • How to communicate data • How to build an effective outsourcing program and what incentives can go with this? • Disclosure: private vs public company • Upfront communication, what is driving the decisions/ • Considerations for data release and the role of the priorities? CMO interacting with Wall Street, scientists, investigators, and payors • Clarity of planning • Key strategic components: planning and execution, • How to create alignment based on key variables (data quality data and biostatistics, manuscript base quality, time to lock, enrollment, etc) development, interactions with authors and journals • Model options and pros and cons that go with them, • Many variables to think about: How to choose from (i.e, hybrid mix) different types of papers, what journals to work with, • Relationship building etc • Different ways to interact with CROs • Generating proof sources in support of regulatory, health economics, reimbursement, guidelines and • Governance and steering committees commercialization efforts • How models can be different and change? • What is the most efficient organizational approach: • Key concerns and solutions internal, outsourcing or mixed model? Moderator: Led by: Pamela Palmer, MD, PhD Lee Allen, MD, PhD CMO & Co-Founder, AcelRx Pharmaceuticals, Inc. CMO, Spectrum Pharmaceuticals Panelists: Panelists: Nathaniel Brown, MD Lori Kunkel, MD CMO, Presidio Pharmaceuticals CMO, Loxo Oncology Marc Hoffman, MD Mark Monane, MD, MS SVP & General Manager, Biopharmaceutical CMO, CardioDx, Inc. Development, Theorem Clinical Research 4:00 pm Paul Kwon, MD Best Practices in Reimbursement Planning Strategy CMO, Nora Therapeutics • Strategies in trial design • Strategies around applications

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• Pharmacoeconomics • What role does the CMO have in business development opportunities • Value based pricing • What are the personal implications of being a CMO in • How to ensure a clinical program has the broadest a publicly traded company geographic applicability • Lessons learned Oscar Segurado, MD, PhD CMO, Crescendo Bioscience / Myriad Genetics Jakob Dupont, MD CMO & SVP, OncoMed 4:30 pm Networking Reception 9:30 am Graciously sponsored by: CEO Panel: What do CEOs look for in a CMO? • Hiring: Identifying the appropriate CMO • Transitioning from pharma to biotech • How much time has a CMO spent in biotech? and • The range of a CMO • Therapeutic area expert, how well versed in biotech? • What if your company has multiple therapeutics areas? • CMAs – Chief Medical Advisors as an alternative DAY TWO - TUESDAY, NOVEMBER 4, 2014 model? • Interacting with VCs 8:00 am Breakfast • Board interaction • How the CMO can help position the company 8:30 am Co-Chairs’ Welcome • The importance of connecting the CMO to the team/ community Lee Allen, MD, PhD CMO, Spectrum Pharmaceuticals • Development path Pamela Palmer, MD, PhD Moderator: CMO & Co-Founder, AcelRX Pharmaceuticals, Inc. Pamela Palmer, MD, PhD CMO & Co-Founder, AcelRx Pharmaceuticals, Inc. 8:45 am Panelists: Keynote Case Study on the Life of a CMO Pre- and Martin Babler Post-IPO CEO, Principia Biopharma Inc. • Expanding the role of the CMO to meet the Brian Culley, MA, MBA requirements of a public company CEO & Director, Mast Therapeutics • Insights into the experience Richard King • Expanding the role of the CMO to meet the CEO, President & Acting Chief Commercial Officer, AcelRx requirements of commercialization Pharmaceuticals • The role of the CMO Tyler Martin, MD CEO, Adjuvance Technologies and Great Plains • Managing growth Biotechnology • What role does the CMO have in articulating the data Jaisim Shah of the clinical trials CEO, Semnur Pharmaceuticals

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10:15 am 12:15 pm Networking Break How BIO is Representing Emerging Biotechs • What is BIO doing to support emerging biotechs? 10:45 am How Investors Determine Valuation of Biotechs • An overview on policy changes and market trends • Evolution on what investors are looking for from an • The relationship between BIO and the FDA end game • BIO’s list of priorities for emerging biotechs • Good ideas funded and great ideas not funded Cartier Esham, PhD • How venture looks at management EVP, Emerging Companies, Biotechnology Industry Organization (BIO) • POV on exits

Moderator: 12:45 pm Julie Hambleton, MD Luncheon SVP & CMO, Five Prime Therapeutics

Panelists: 1:45 pm Chuck Boggs Afternoon Workshop on Alternative Methods to Partner, Paladin Ventures Designing Clinical Trials Leon Chen, PhD The purpose of the workshop is to identify and explore Venture Partner, OrbiMed Advisors different methods to designing clinical trials. What are the different options and which are best to see how the Mike Dybbs, PhD drug is behaving to make earlier and more accurate Principal, New Leaf Venture Partners decisions? Jonathan Norris Agenda Managing Director, Healthcare Practice, Silicon Valley Bank • Overview of Model Based Designs and Medical necessity 11:30 am • Issues and Challenges from Medical and Pharma Business Development POV on Partnering Operational considerations • Structuring deals • Software tools to elucidate Decision Rules and • Early phase investment vs late phase investment provide intuitive details for Medical perspectives • Strategy • Drafting the SAP for the protocol • Selecting the right partner • Operational tools needed – IVRS/EDC, etc.

• What is gained? What is lost? This workshop is inclusive as part of the CMOW Summit, but it is also offered as a stand-alone program. Moderator: It is open to Directors and Managers of clinical trials Lee Allen, MD, PhD and statisticians. CMO, Spectrum Pharmaceuticals Speakers TBA Panelists: Jay Parrish, PhD, MBA Associate Director, Corporate Development, Gilead 4:45 pm Sciences Program Concludes Samuel Wu, MD, PhD Managing Director, Medimmune Ventures

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HOTEL INFORMATION REGISTRATION & PRICING

Marines’ Memorial Club 1st Early Bird - before September 12, 2014 $1,295 & Hotel 2nd Early Bird - before October 10, 2014 $1,395 609 Sutter Street Standard - after October 10, 2014 $1,495 San Francisco, CA 94102 The conference fee includes the program, morning coffee/ www.marineclub.com tea, luncheon, reception and conference documentation.

For reservations, call 800-562-7463 or 415-673-6672 and Discount Programs ask for the CMO Summit group rate. You may also email Group Registration – Receive a 10% discount off each your reservations to [email protected]. registration when two or more colleagues from the same Marines’ Memorial Club’s 1920s Beaux-Arts building is company register online. located in the Union Square neighborhood and combines a blend of classic with modern. Ideal for leisure and business Large group discounts available. travel with sophisticated accommodations and beautiful meeting space. Please contact [email protected] or call 646-350-2580 for any questions on discount rates.

Payment Policy Payment 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 Policy If you are unable to attend and would like to register a substitute, please email [email protected]. 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 [email protected] or call 646-350-2580.

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