CHIEF MEDICAL OFFICER SUMMIT · 11/4/2013 · CMO, Avaxia Biologics, Inc., Principal, Middleburg Consultants and Clinical Professor of Medicine, Georgetown University School of Medicine

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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

KEYNOTE: WHAT A CMO CAN LEARN FROM FOOTBALLHal Barron, MD, FACCCMO & EVP, Head of Global Product DevelopmentGenentech

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

CO-CHAIR: RESOURCEFUL OUTSOURCINGSheila Gujrathi, MDCMOReceptos

Executive Sponsor

Supporting PartnersParticipating Investors

Associate Sponsors Exhibitor

Organized by

http://WWW.THECONFERENCEFORUM.ORG


http://www.womeninbio.org/






Dear CMO Summit Attendees,

The inaugural CMO Summit for Emerging Life Science Companies was specifically tailored to meet the unique needs of the CMO. I have always felt that being a CMO in a biotech was a job no one else had in the company, and that having a community to share best practices was important. The CMO is not only responsible for delivering on the clinical trials, but also a key player in the company’s fund-raising efforts. In addition, as a CMO you must stay abreast in all the key new developments in your therapeutic areas and as well as innovative advancements in study design and regulatory science. The agenda and talented speakers that have been put forth will tackle all of those aspects of being a CMO. On behalf of the meeting organizers, speakers and sponsors who shaped the program, I want to thank all the attendees.

Best wishes,

Elizabeth Stoner, MDManaging DirectorMPM CapitalandLead Advisor to the CMO Summits

Srinivas Akkaraju, MD, PhDGeneral Partner, Sofinnova Ventures

Lee Allen, MD, PhDCMO, Spectrum Pharmaceuticals

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

Mohammad Azab, MD, MBACMO, Astex Pharmaceuticals

Solomon Babani, MBAGlobal VP, Alliance Management, Covance, Inc.

Hal Barron, MD, FACCCMO & EVP, Head of Global Product Development, Genentech

Chuck BoggsChairman, Golden Gate Angels Group and Principal, Paldin Venture Group

Jill CarrollSenior Associate, SR One

Farah Champsi, MBAManaging Director, Alta Partners

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

Howard Dittrich, MD, FACCCMO, Sorbent Therapeutics

Paul Grint, MDPresident, Cerexa

Sheila Gujrathi, MDCMO, Receptos, Inc.

Barry Hafkin, MDCMO, Affinium Pharmaceuticals

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

Julie Hambleton, MDSVP & CMO, Five Prime Therapeutics

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

Rekha Hemrajani, MBAFormer VP, Head of Licensing & M&A, Onyx Pharmaceuticals

Colin Hislop, MDCMO, Anthera Pharmaceuticals

Jan Johansson, MD, PhDSVP, Medical Affairs, Resverlogix

Jeff JonkerSVP, Corporate and Business Development, Theravance

Sam LambrosVP, Strategic Business Development, Quintiles

Jeffery Loutit, MBChBCMO, Rempex Pharmaceuticals

Jonathan MacQuitty, PhD, MBAPartner, Abingworth

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

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

Nestor Molfino, MD, MScCMO, KaloBios Pharmaceuticals, Inc.

CHIEF MEDICAL OFFICER SUMMIT WEST OVERVIEW

OVERVIEW

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SPEAKING FACULTY




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

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

Mike Royal, MD, JD, MBAChief, Clinical Affairs, AcelRx Pharmaceuticals, Inc.

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

Tracy Saxton, PhDAssociate, SV Life Sciences Advisers LLP

Oscar Segurado, MD, PhDCMO, Crescendo Bioscience

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

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

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

Frank Torti, MDPrincipal, NEA

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

Samuel Wu, MD, PhDManaging Director, MedImmune Ventures

The Conference Forum is proud to work with the following industry information providers:

Clinical Development/Outsourcing and Regulatory Management

8:00 amRegistration & Breakfast

8:45 amCo-Chairs’ Welcome



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 the trade-offs. 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

10:20 amNetworking Break

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SPEAKING FACULTY CHIEF MEDICAL OFFICER SUMMIT WEST

9:30 amKeynoteDecision-Making: What a CMO Can Learn from Football

• Differentiating smart risk from bad decisions

• Learning from failure

• The value of cognitive diversity

Hal Barron, MD, FACCCMO & EVP, Head of Global Product Development, Genentech

DAY ONE - MONDAY, NOVEMBER 4, 2013



http://www.lifescienceleader.com






http://www.Biocentury.com







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

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?

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

Panelists:Mohammad Azab, MD, MBACMO, Astex Pharmaceuticals

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?

• 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:Solomon Babani, MBAGlobal VP, Alliance Management, Covance, Inc.

Howard Dittrich, MD, FACCCMO, Sorbent Therapeutics


Colin Hislop, MDCMO, Anthera Pharmaceuticals

Sam LambrosVP, Strategic Business Development, Quintiles


1:00 pmLuncheon

CHIEF MEDICAL OFFICER SUMMIT WEST AGENDA

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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

Moderator:Lee Allen, MD, PhDCMO, Spectrum Pharmaceuticals

Panelists:Jan Johansson, MD, PhDSVP, Medical Affairs, Resverlogix

Jeffery Loutit, MBChBCMO, Rempex Pharmaceuticals


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

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 get big pharma to look at you

• What small companies are looking for in big pharma

• How to position yourself with a large pharma partner

• Large pharma expectations

• Presenting clinical data

• Communication between companies: ongoing joint development committees vs. joint commercialization

• Assessment of roles and responsibilities in a partnering arrangement and what you should watch out for

• Opportunities to pool or share resources

Moderator:Sheila Gujrathi, MDCMO, Receptos, Inc.

Panelists:Lee Allen, MD, PhDCMO, Spectrum Pharmaceuticals

Paul Grint, MDPresident, Cerexa

Barry Hafkin, MDCMO, Affinium Pharmaceuticals


3:45 pmNetworking Break

4:00 pmRoundtable Session Choices1. Innovative Outsourcing Strategies

• Evolution of the CRO Industry and Traditional Outsourcing Strategies

• Outsourcing Models in Action: An overview/case studies of innovative strategic partnership solutions that helped reduce fixed costs and improve R&D efficiencies, including:

- Full Molecule Development (to IND/CTA, First in Human, or POC)

- Clinical Partnership

- Sole Sourcing

- Flexible Sourcing

- Single Compound Phase I-III Strategic Alliance

- Asset Transfer

- Dedicated Capacity

• Small Pharma/Biotech & CRO Partnering Models of the Future

5

AGENDA CHIEF MEDICAL OFFICER SUMMIT WEST




Solomon Babani, MBAGlobal VP, Alliance Management, Covance, Inc.

2. Acquiring Orphan Designation/Benefits and the Journey• 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



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

Jill CarrollSenior Associate, SR One

Frank Torti, MDPrincipal, NEA

5:00 pmNetworking Reception hosted by

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

8:00 amBreakfast

8:30 amCo-Chairs’ Welcome



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, and 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


Panelists:Mohammad Azab, MD, MBACMO, Astex Pharmaceuticals

Nestor Molfino, MD, MScCMO, KaloBios Pharmaceuticals, Inc.


CHIEF MEDICAL OFFICER SUMMIT WEST AGENDA

6

DAY TWO - TUESDAY, NOVEMBER 5, 2013





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

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

- 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

Moderator:Lee Allen, MD, PhDCMO, Spectrum Pharmaceuticals

Panelists:Chuck BoggsChairman, Golden Gate Angels Group and Principal, Paldin Venture Group

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

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

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

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 your thoughts 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


Panelists:Srinivas Akkaraju, MD, PhDGeneral Partner, Sofinnova Ventures

Farah Champsi, MBAManaging Director, Alta Partners

Jonathan MacQuitty, PhD, MBAPartner, Abingworth

7

AGENDA CHIEF MEDICAL OFFICER SUMMIT WEST




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, MBAFormer VP, Head of Licensing & M&A, Onyx Pharmaceuticals

Jeff JonkerSVP, Corporate and Business Development, Theravance

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


Mike Royal, MD, JD, MBAChief, Clinical Affairs, AcelRx Pharmaceuticals, Inc.

1:00 pmConference Concludes

Our mission at Cytel is to improve success rates in the development of drugs, biologics and medical devices. We do this by improving clinical trials through innovative application of

statistical science, operations research, and new computational methods We are leaders in adaptive trial design and implementation. All 25 leading biopharma companies rely on our technology in their clinical studies. For more information, visit www.cytel.com.

Covance, with headquarters in Princeton, New Jersey, is one of the world’s largest and most comprehensive drug development

services companies with annual revenues greater than $2.2 billion, global operations in 60 countries, and more than 12,000 employees worldwide. Through its lead optimization, nonclinical, clinical and commercialization services, Covance has helped pharmaceutical and biotech companies develop one-third of all prescription medicines in the market today. Covance generates more safety and efficacy data for the evaluation of new prescription drugs than any other entity. The firm recognizes the importance of every data point, in every study, and of getting things right every day. Covance is proud and honored to have worked on 82 percent of the drugs approved by the U.S. Food & Drug Administration in 2012. For more information, visit www.covance.com.

Future State has over 30 years of helping people remain productive and thrive inside a constant state of

change. We design change strategy where every tactic is informed by our deep understanding of human behavior, complex business problems and optimized organizational design to arrive at leadership alignment and an engaged workforce. For more information, visit www.futurestate.com.

Halloran Consulting Group is a boutique management consulting

firm that specializes in navigating and accelerating life science product development. As an objective advisor and trusted partner to our clients, we work with industry organizations of all types and sizes in all stages of development, including pharma, biotech, medical devices and service providers. We identify developmental challenges and devise and implement the best-fit, practical, long term solutions effectively and efficiently. For more information, please visit our website at www.hallorancg.com.

CHIEF MEDICAL OFFICER SUMMIT WEST SPONSORS

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EXECUTIVE SPONSOR

ASSOCIATE SPONSORS



Documents

CHIEF MEDICAL OFFICER SUMMIT · 11/4/2013 · CMO, Avaxia Biologics, Inc., Principal, Middleburg Consultants and Clinical Professor of Medicine, Georgetown University School of Medicine