2013 Agenda

Clinical Development/Outsourcing and Regulatory Management

• 8:00 am
  

• Registration & Breakfast

• 8:45 am
  

• Co-Chairs’ Welcome

  Greg Fiore, MD
  CMO, The Medicines Company

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

• 9:00 am
  

• Opening Address: What is a CMO? Defining the Reality of the Role

  How 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 (including how to function with restrictive capital) 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, MD
  President, CMO & Director, Dynavax Technologies

• 9:30 am
  

• Keynote: Strategic Initiatives Signaling Transformative Change in the Drug Development Landscape

  • Overview of the current global operating environment for drug development
  • Profiling strategic, transformative initiatives being implemented (including open innovation; integrated alliances with CROs and sites; risk-based monitoring; protocol design optimization)
  • Forecasting the impact of these initiatives

  Ken Getz, MBA
  Director of Sponsored Research, Tufts CSDD

• 10:20 am
  

• Networking Break

• 10:50 am
  

• Clinical Trial Design

  Part 1: 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 2: Adaptive Trial Design: Benefits & Risks

  • How to implement adaptive trial design
  • Designing clinical trials to adapt parameters along with data
  • Regulatory management
  • Barriers to adaptive trial design

  Moderator:
  Santosh Vetticaden, MD, PhD
  CMO, SVP, Mast Therapeutics, Inc.

  Panelists:
  Zoran Antonijevic
  Senior Director, Strategic Consulting and Adaptive Implementation, Cytel Inc.

  Greg Fiore, MD
  CMO, The Medicines Company

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

  Robert Pyke, MD, PhD
  CMO, S1 Biopharma

• 11:55 am
  

• Innovative Outsourcing Strategies, Models & Options with CROs

  Part I

  • How outsourcing is a decision to be made in the context of defining the way in which a company wants to programmatically differentiate itself
  • How a CRO can be much more than an execution partner, also providing insights that augment your scientific expertise
  • Assessing CRO capabilities to match your short-term and long-term needs using the metrics of: how much of a CRO’s revenue is generated by emerging biotechs, what therapeutic area expertise is of the CRO, and how biotechs fit into the growth of the CRO
  • How to maximize the potential successful collaboration with a CRO – lessons from the past

  Part II

  • 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 can you afford to outsource?
  • Managing quality expectations with vendors, maintaining accessibility to own data, avoiding fraud and misconduct, and keeping costs down
  • How are CROs thinking about longer-term partnerships and biotechs as an investment?
  • How do you negotiate unique deal structures where biotechs are entering into risk-sharing partnerships with CROs?
  • How do you develop deals with multiple programs with CROs and hold on to leverage?
  • CMO in virtual vs. non-virtual life science company
  • Pros and cons of working with small vs. large CROs
  • Finding a balance with your CRO with budget and cash flow challenges

  Moderator:
  Greg Fiore, MD
  CMO, The Medicines Company

  Panelists:
  Tyler Martin, MD
  President, CMO & Director, Dynavax Technologies

  Jeffrey Spaeder, MD
  SVP & Chief Medical and Scientific Officer, Quintiles

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

• 1:00 pm
  

• Luncheon

• 2:15 pm
  

• How 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:
  Steven Zelenkofske, DO, FACC, FACOI, FCCP
  SVP, Clinical and Medical Affairs & CMO, Regado Biosciences

  Panelists:
  Greg Dombal
  COO, Halloran Consulting Group

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

  Michael Sweeney, MD
  CMO & VP of R&D, Depomed

• 3:00 pm
  

• How to Enter Into an Effective 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
  • What makes an ideal IPO?
  • Exit opportunities

  Moderator:
  Jim Roach, MD
  CMO & SVP, Development, Momenta Pharmaceuticals

  Panelists:
  Daniel J. Curran, MD
  VP, Therapeutic Area Lead, Value Creation Alternatives, Takeda Pharmaceuticals International, Inc.

  David Grayzel, MD
  Managing Director, Atlas Venture Development Corp.

  Donna Grogan, MD
  CMO, Anexon, Inc.

• 3:45 pm
  

• Networking Break

• 4:00 pm
  

• Getting 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 pm
  

• Roundtable Session Choices

  This interactive roundtable session is designed to give all attendees an opportunity to meet one-on-one or in small group meetings with the following speakers to get questions answered to their specific needs. Attendees will have the opportunity to choose up to two roundtables. The first runs from 4:30 – 5:00 pm and the second runs from 5:00 – 5:30 pm where you will have the opportunity to switch sections.

  1. 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

  Jeffrey Spaeder, MD
  SVP & Chief Medical and Scientific Officer, Quintiles

   

  2. Physician Recruitment

  • Candidate Sourcing Strategies
  • How to engage the most desirable candidates (often passive) and managing the recruiting process
  • Closing the deal
  • In-house recruiters vs. external recruiters – pros and cons

  Paul Edgerton
  Managing Partner, Gold Partners

   

  3. 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

  Eric Hedrick, MD
  CMO, Epizyme

   

  4. How to Effectively Partner with Academia

  • Building a New R&D Ecosystem
  • Creating productive partnering and communication models between industry and academia and different perspectives from them
  • Bridging the cultural gap between academia and pharma
  • Accessing innovation and partnership models
  • Translational medicine: academic vs. industry perspective
  • Managing intellectual property discussions
  • Lessons learned from Pfizer CTI/Tufts Medical Center partnership

  Anthony Coyle, PhD
  VP & CSO, Centers for Therapeutic Innovation, Pfizer

  Nancy Wetherbee, MBA
  Director, Office of Research Business Development in Industry Translation, Tufts Medical Center

   

  5. 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 2013

  Todd Foley, MBA
  Managing Director, MPM Capital

  Vikas Goyal, MBA
  Senior Associate, SR One

  B. Christopher Kim, PhD
  Partner, Oxford Bioscience Partners

  Jeffrey Moore, DPhil, MBA
  VP, MP Healthcare Venture Management, Inc.

• 5:30 pm
  

• Networking Reception

2013 Agenda

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

• 8:00 am
  

• Breakfast

• 8:30 am
  

• Co-Chairs’ Welcome

  Greg Fiore, MD
  CMO, The Medicines Company

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

• 8:45 am
  

• How 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

  Moderator:
  Greg Fiore, MD
  CMO, The Medicines Company

  Panelists:
  Randall E. Kaye, MD
  Senior Vice President, Medical Affairs & CMO, Avanir Pharmaceuticals, Inc.

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

• 9:30 am
  

• What a CMO Can Do to Secure Alternative Sources of Financing: Alternative Investors’ Perspectives

  Alternative sources of capital, what alternative investors look for when funding emerging life science companies and what a CMO can do to contribute to the financing effort.

  • Angel Investors: rushing in where venture capitalists fear to tread; using angels for funding de-risking round to make you more attractive to VCs
  • Patient Advocacy and Passion Capital: foundation funding; other contributions: support and the patient perspective being brought to everything they do; how different foundations operate
  • Crowd Funding: “the best is yet to fund”: misconceptions in funding; alternative sources of funding; explaining the science and knowing the crowd
  • Government Funding: FNIH’s role in public-private partnerships; the role a CMO has in the project; direct and early access in development of drug development projects to catch attention of FDA; sharing costs across multiple identities; acting as a conduit to bring together interested parties and find a champion in industry or academia for funding
  • Government Funding: NIH’s SBIR/STTR program and true non-dilutive funding; pros and cons; practical mechanisms to secure non-dilutive funding; goals on the funding side on providing the capital; how to make non-dilutive funding successful; how to use SBIR funding to leverage other funding; when you have a government partner, what happens to the IP?; areas of disconnect as the money is being rewarded and being aware of who is doing the reviewing; “innovation conferences” and “investor forums” to decrease time between application to award

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

  Panelists:
  Carl Berke, PhD
  Partner, Partners Innovation Fund and Founding Director, Mass Medical Angels

  Jodi Black, PhD
  Deputy Director, Division of Extramural Research Activities, National Heart, Lung, and Blood Institute, NIH

  Steve Hoffmann
  Scientific Program Manager, The Biomarkers Consortium, Inflammation and Immunity Steering Committee, FNIH

  Tracey Mumford
  Senior Associate Director, Research Partnerships, The Michael J. Fox Foundation for Parkinson’s Research

  Greg Simon
  CEO, Poliwogg

• 10:15 am
  

• What 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
  • 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

  Moderator:
  Greg Fiore, MD
  CMO, The Medicines Company

  Panelists:
  David Grayzel, MD
  Managing Director, Atlas Venture Development Corp.

  Nilesh Kumar, PhD, MBA
  Director, Merck Serono Ventures

  Jeffrey Moore, DPhil, MBA
  VP, MP Healthcare Venture Management, Inc.

  Lauren Silverman, PhD
  Managing Director, Novartis Venture Funds

  Samuel Wu, MD, PhD
  Managing Director, MedImmune Ventures

• 10:50 am
  

• Morning Networking Break

• 11:15 am
  

• How to Plan for an Exit & 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

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

  Panelists:
  Kevin Bitterman, PhD
  Principal, Polaris Partners

  Chris Kiritsy, MBA
  President & CEO, Arisaph

  Mark Levin
  Partner & Co-Founder, Third Rock Ventures

• 12:00 noon
  

• Driving 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

  Greg Fiore, MD
  CMO, The Medicines Company

• 12:30 pm
  

• Advising 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 go/no-go decision?

  Carl Berke, PhD
  Partner, Partners Innovation Fund, Partners Healthcare and
  Founding Director, Mass Medical Angels

  Oscar Segurado, MD, PhD
  CMO, Crescendo Bioscience

• 1:00 pm
  

• Conference Concludes