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BioPharm America™ 2014 Program Overview

www.ebdgroup.com/bpa/program
Sunday, September 21, 2014
6:00–8:00 pm
Level 3, Atrium
BioPharm America Welcome Reception

All BioPharm America attendees are cordially invited to join us for a welcome reception.

Boston Marriott Copley Place
110 Huntington Avenue
Boston, MA 02116 (map)

You will be able to pick up your name badge at the welcome reception.

Monday, September 22, 2014
7:30 am
Level 4, Foyer
7:30 am
Level 4, Grand Ballroom
Exhibition opens and Continental Breakfast
9:00–10:30 am
Parallel Workshops
The perfect pitch: Do you have what it takes to win over investors?

Win a one-to-one meeting with a leading life science private equity investor. Members of the audience will have 30 seconds to pitch their company, product, technology or service to a star-studded panel of judges representing at least six life science investment firms. The top picks will be selected for a second round that includes two minutes of presentation and/or Q&A time with the judges. First and second place winners will receive a full 30-minute private followup meeting with the panelist of their choice. Judges will receive a written summary of all participant pitches and their scores, should they wish to follow up on their own. Audience preparation notes:

Participants will be judged on the quality and effectiveness of the overall 30-second pitch. Remember, the primary goal of an elevator pitch is to spark interest for a followup meeting. Pre-workshop coaching is available from the moderator (contact: doug@macbiocom.com). Practice, practice, practice!

Moderator:
Douglas MacDougall – President, MacDougall Biomedical Communications

Judges:
  • Peter Barrett – Partner, Atlas Venture
  • Bernard Davitian – VP and Managing Director, Sanofi-Genzyme BioVentures, Sanofi
  • Arthur Franken – Partner, Gilde Healthcare
  • Brian Gallagher – Partner, SR One
  • Frederick Jones – Director, Broadview Ventures, Inc.
  • Nilesh Kumar – Director, MS Ventures
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1+1=3: How unconventional pharma and VC collaborations are expanding R&D budgets

In recent years, pharma company R&D budgets have been under pressure. Pharma companies are struggling to find ways to continue to advance the development of attractive drug candidates from their own pipelines while simultaneously looking outside for attractive programs they can acquire. One way pharma companies can do more within a constrained R&D budget is to partner with outside investors in innovative ways in order to develop attractive drug candidates with "other people's money." This panel of industry experts will explore some of the ways that pharma companies and VC firms are partnering to develop drugs and generate investment returns.

Moderator:
David Collier – CEO, Velocity Pharmaceutical Development

Panelists:
  • Rob Armstrong – Chief Business Officer, Gelesis, Inc.
  • David Berry – Partner, Flagship Ventures
  • David Grayzel – Partner, Atlas Venture
  • Jay Lichter – Managing Director, Avalon Ventures
  • Kareem S. Reda – Director, Business Development, Celgene
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Due diligence: How to ensure a successful process

M&A or partnering agreement, product or technology—a biotech company should be prepared to open its doors, books and personnel to a potential partner for full disclosure. Especially for large deals, due diligence is a complex commercial, legal and financial analysis that involves many within the biotech company and needs to be well orchestrated. Various companies have their own processes in place for how to conduct a thorough due diligence. This workshop will give guidance on what needs to be taken into consideration. How can a biotech company prepare for this process? Who needs to be involved? What should be laid out in your VDR? What is the expectation of timing for what needs to be accomplished? What does the pharma side expect to see and what do they not want to see? Panelists will draw from their due diligence experience and discuss what worked and what did not work so well.

Moderator:
Martina Molsbergen – CEO, C14 Consulting Group

Panelists:
  • Jan Case – Senior Director, Corporate Due Diligence, Shire
  • John Delyani – Executive Director, Business Development and Licensing, Boston Innovation Hub, Merck Research Laboratories
  • Carl Jessop – Due Diligence Director, AstraZeneca
  • Katy Moore – Senior Director, Scientific Licensing, Worldwide Business Development, GlaxoSmithKline
  • David Yeary – VP, Life Sciences, Merrill Datasite
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10:45 am–12:30 pm
Level 4, Salon G
Welcome, Keynote, and Opening Plenary Discussion
Welcome Remarks

Speakers:
  • Anna Chrisman – Group Managing Director, EBD Group
  • Robert Coughlin – President and CEO, MassBio
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Special Presentation
Announcement of the Fierce 15

The Fierce 15 is a hunt that involves the biggest ideas in biotech, the most daring characters in biopharma, and entrepreneurs doing things that haven't been done before. The result is fifteen privately held drug developers or platform biotechs with the potential to have a huge impact on the treatment of human diseases. John Carroll, Editor-in-Chief of FierceBiotech, will announce the winners live on site.

Speaker:
  • John Carroll – Editor-in-Chief, FierceBiotech
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Keynote Address

Rare diseases, well done: New paths to innovation in biopharma

Speaker:
Gary NabelGary Nabel
CSO
Sanofi

Opening Plenary Discussion
Getting real: Biopharma innovation and its impact on patients

Leading biopharma executives will discuss how changes in the overall healthcare market are affecting their corporate strategies in addressing diseases. The session will cover topics such as the reimbursement landscape, patient access, and partnering deals, to understand the most significant factors affecting innovative product development and the business of fulfilling unmet medical needs.

Moderator:
Karen Bernstein – Co-Founder, Chairman, and Editor-in-Chief, BioCentury

Panelists:
  • Barry Greene – President and Chief Operating Officer, Alnylam Pharmaceuticals
  • Adam M. Koppel – Senior VP and Chief Strategy Officer, Biogen Idec
  • Gary Nabel – CSO, Sanofi
  • Richard Pops – Chairman and CEO, Alkermes plc
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12:30–1:30 pm
Level 4, Exhibit; Level 3, Gloucester
Luncheon
1:30–5:00 pm
Parallel Panel Discussions
Dealmaking and Strategy
Mayflower II: The migration of European biotechs to the US

Amid the breaking wave of US IPOs, more and more of the candidates are European. In addition to the early entrants such as UniQure from the Netherlands (IPO in February 2014), a crop of new companies is appearing from abroad on these shores.

Meanwhile, European markets seem to be (finally) opening up for biotech IPOs. Pixium Vision raised USD 46.7 million on EuroNext Paris in June 2014 and ArGEN-X has announced that it will list there soon. These IPOs are typically smaller and company valuations are lower.

What factors determine where a European company will file? Will they continue to flock to US markets? How long can this trend last? And with some of its best companies coming overseas, is Europe being relegated to a kind of "minor league" status in biotech and life sciences?

Moderator:
Steve Dickman – CEO, CBT Advisors

Panelists:
  • Philip Astley-Sparke – President US, uniQure
  • Sinclair Dunlop – Founder and Managing Partner, Epidarex Capital, LP
  • Daniel Grau – President, Heptares Therapeutics
  • Rafaèle Tordjman – Managing Partner, Sofinnova Partners
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Behind the headlines: Large series A financings deconstructed

Obtaining a large round of Series A financing can garner a promising young biotech a lot of attention and validation from the life science community with news of the deal ricocheting around inboxes, twitterfeeds and industry rags within hours. But what are the actual terms behind the headlines? How much of this capital is actually contingent? What types of milestones do entrepreneurs need to achieve? What are the risks and benefits of tranched deals? For those companies that close these deals, what is it about them that made them so appealing to investors? The product/technology? The leadership team? Please join us as we deconstruct these deals and explore how to obtain and optimize them.

Moderator:
Jeffrey L. Quillen – Partner, Foley Hoag LLP

Panelists:
  • Jim Geraghty – Entrepreneur-in-Residence, Third Rock Ventures
  • Alexandra Glucksmann – Chief Operating Officer, Editas Medicine
  • Ed Mathers – Partner, Washington DC, New Enterprise Associates
  • Pieter Muntendam – President and CEO, scPharmaceuticals
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Pharma's big bets on risky therapeutic areas

Every pharma company boasts about being innovation-driven, yet most of their R&D targets and approaches are the same. Is there an R&D "fashion victim syndrome" that makes everyone invest in the same spaces? Who is preventing true innovation? Company boards? The regulators? Ourselves? Sit down with executives from pharma to discuss why going into unpopular areas alone can actually be wildly successful.

Moderator:
Dennis J. Purcell – Founder and Senior Advisor, Aisling Capital

Panelists:
  • Anthony J. Muslin – VP, Head of Cardiovascular and Fibrosis Therapeutic Strategic Unit, Sanofi
  • Christoph Pittius – VP, Head of Transactions, Business Development, AstraZeneca
  • John M. Sorvillo – Director, Licensing, External R&D, AMGEN
  • Robert Willenbucher – Head Cell Therapy, Janssen R&D, LLC
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In the Spotlight
Challenges in orphan CNS indications

With so many obstacles in treating neurodegenerative diseases, what new strategies are being developed to advance the field? How can consortia groups and other multi-stakeholder collaborations help expedite clinical research? What are the newest developments in modalities such as gene therapy, antisense,and RNAi? This panel of R&D companies, investors, and research organizations will answer these questions by highlighting innovative research and business models that are impacting drug development.

Moderator:
Richard Brudnick – VP and Co-Head, Business Development, Biogen Idec

Panelists:
  • Kees Been – President and CEO, Lysosomal Therapeutics, Inc.
  • Mustafa Sahin – Director, Translational Neuroscience Center, Boston Children's Hospital
  • Robert Weisskoff – Partner, Fidelity Biosciences
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The power of the patients: How patients are getting things done in the drug development process

From funding research to recruiting for clinical trials to working with the FDA and CMS on regulatory and reimbursement issues, patients have an increasingly vital role in R&D. The impatient patient is not sitting idly by waiting for much needed cures and treatments—they are active partners in the search for remedies and initiating projects like The Race to Yes social media campaign, providing millions in undiluted research capital, and collaborating with industry partners on patient-trial matching. Learn how patients are making an impact and how biopharmacos are partnering with these groups to leverage the "power of the patients" and make new treatments available as quickly as possible.

Moderator:
Karin Hehenberger – CEO and Founder, Lyfebulb; Chief Strategy Officer, IMMUNE Pharmaceuticals Ltd.

Panelists:
  • Debra Miller – Founder and CEO, CureDuchenne
  • Imran Nasrullah – Director, Innovation Sourcing, Boehringer Ingelheim
  • Michele M. Oshman – Director, Federal Alliance Development, Corporate Affairs, Eli Lilly and Company
  • Marie Schiller – Co-Founder, T1D Exchange
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Under the spotlight: The pressures of being an early stage public company

Now more than ever before, investors expect big payoffs and look for significant clinical outcomes and commercial success from their biotech investments. How do new public companies meet the demands and operate under the close scrutiny of savvy investors? Is it a race to go take advantage of the open IPO window and raise capital while it's available? What impact has going public had on the company's R&D strategy? In addition to managing their pipelines, public companies need to have sophisticated IR & PR teams to address issues around financial reporting, governance, compliance, and infrastructure. We'll examine the tradeoffs companies make when they decide to go public and hear from executives of newly minted companies to gain insights on managing the pressures of the markets, learn what it takes to keep stakeholders happy in a high risk environment, and seek guidance for private companies that are considering filing.

Moderator:
Art McGivern – Partner, Goodwin Procter

Panelists:
  • Todd Foley – Managing Director, MPM Capital
  • Christopher D. T. Guiffre – Senior VP, Chief Business Officer, Cerulean Pharma
  • Thomas E. Hughes – CEO, Zafgen, Inc.
  • Dan Paterson – Chief Business Officer, Verastem
  • James B. Weissman – Chief Business Officer, Dicerna Pharmaceuticals
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Advanced Therapies - PCT Advanced Therapies - PCT


Advanced Therapies
Clinical outlook for new immuno-oncology treatments

The clinical pipeline for new cancer immunotherapies is growing with new technologies targeting almost the entire spectrum of cancer indications. This panel will consider the clinical outlook for various cancer immunotherapies: PD1 and checkpoint inhibitors, cell-based immunotherapies and adoptive T-cell therapies, and other novel approaches to treating the disease. It will also consider the extent to which the clinical advances and breakthrough therapy opportunities are driving partnering activity.

Moderator:
Robert A. Preti – President and Chief Scientific Officer, Progenitor Cell Therapy

Panelists:
  • Philippe Lopes-Fernandes – Senior VP and Head, Global Licensing and Business Development, EMD Serono
  • Doug Plessinger – VP, Clinical and Medical Affairs, Argos Therapeutics
  • Debanjan Ray – VP, Business Development and Alliance Management, CytomX
  • Isabelle Rivière – Director, Cell Therapy and Cell Engineering Facility, Memorial Sloan Kettering Cancer Center
  • Robert Willenbucher – Head Cell Therapy, Janssen R&D, LLC
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Next generation therapies: Will they require new approaches to partnering or will traditional models prevail?

Next generation therapies involving gene therapy, gene modification technologies and cell-based therapies may not reach commercialization along traditional routes with a large pharma stepping in to acquire or license rights to clinically-advanced products. Many of the key technologies involve platforms and autologous or individualized approaches that require different skill sets than those present in more traditional product development groups. This panel will look at the types of partnerships—early and later stage—that could become more commonplace as this sector continues to evolve. It will also consider the role of stakeholders such as hospitals, patient groups, and even the large infrastructure players that will play a crucial role in the success of advanced therapies.

Moderator:
Brock Reeve – Executive Director, Harvard Stem Cell Institute

Panelists:
  • Jennifer Elisseeff – Professor, Jules Stein Chair in Ophthalmology, Director, Translational Tissue Engineering Center, Wilmer Eye Institute and Departments of Biomedical Engineering, Orthopedic Surgery, Chemical and Biological Engineering, and Materials Science and Engineering, Johns Hopkins
  • Eric Halioua – Co-Founder and CEO, Promethera Biosciences
  • Frederick Jones – Director, Broadview Ventures, Inc.
  • Stephen Yoder – Global Lead, CV/Metabolic, Search and Evaluation, Bristol-Myers Squibb
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Developing a sustainable commercial model: Can we devise a pricing system for advanced therapies with multiple winners?

Despite the interest and enthusiasm around the therapeutic potential for these products in a variety of indications, the pricing question remains unanswered. This session will include stakeholders currently thinking through these issues and working to develop successful commercialization models.

Moderator:
Michael J. Werner – Executive Director, Alliance for Regenerative Medicine; Partner, Holland & Knight

Panelists:
  • Philip Astley-Sparke – President US, uniQure; Venture Partner, Forbion Capital Partners
  • Jeff Goater – VP, Business Development, Voyager Therapeutics
  • Brian Rosen – Chief Policy, Advocacy and Patient Access Officer, The Leukemia & Lymphoma Society
  • Jayson Slotnik – Partner, Health Policy Strategies Inc.
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1:00–6:00 pm
One-to-one Meetings ►
5:15–6:15 pm
Plenary Discussion
State of the Industry: A focus on the financial landscape in the life sciences

Moderator:
Bruce Booth – Partner, Atlas Venture

Speakers:
  • Robert Gould – CEO, Epizyme
  • Jeff Jonas – CEO, Sage Therapeutics
  • Liam Ratcliffe – Managing Director, New Leaf Venture Partners
  • Kevin Starr – Partner, Third Rock Ventures
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7:00–9:30 pm
Evening Networking Reception with Buffet-style Dinner
Skywalk Observatory

Skywalk Observatory
800 Boylston Street
Boston, MA 02199 | (map)

Network. Dine. Enjoy an amazing view of Boston

The Skywalk Observatory is a short walk through the Prudential Center, attached to the Marriott Boston Copley Place. BioPharm America staff will be posted along the way to guide you to the event.

Tuesday, September 23, 2014
7:30 am
Level 4, Foyer
7:30 am
Level 4, Grand Ballroom
Exhibition opens and Continental Breakfast
9:00 am–12:10 pm
Pharma and Large Biotech Presentations

Moderator:
Ed Saltzman – President, Defined Health

9:10 am – Merck
9:30 am – AstraZeneca
9:50 am – Bayer HealthCare
10:10 am – Bristol-Myers Squibb
10:30 am – Johnson & Johnson Innovation
10:50 am – EMD Serono
11:10 am – Novo Nordisk
11:30 am – Sanofi
11:50 am – Vertex Pharmaceuticals

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8:00 am–6:00 pm
One-to-one Meetings ►
10:00–11:00 am
Workshop
Challenges in developing differentiated treatments for autoimmune diseases

Humira is currently the top selling pharmaceutical in the world and represents just one of eight approved targeted therapies for RA, a market with an estimated prevalence of less than USD 5 million in the seven major pharmaceutical markets. Rheumatoid arthritis, as well as many of the other indications in which anti-TNFs are entrenched, has all the hallmarks of a mature market (including biosimilars on the horizon), and many view the market as overcrowded. However, significant unmet medical needs remain in RA, where approximately one-third of patients are not achieving a 20% improvement in symptoms on biologics, and needs are even greater in other indications such as IBD. Within this setting, developers of early stage programs are struggling to differentiate their programs and to choose the appropriate indication and path forward for clinical development. Our panel will discuss the challenges associated with differentiating products at early stages of development in autoimmune disease, and potential strategies for choosing lead indications and demonstrating proof-of-relevance to potential partners and/or investors, as well as patients and payers.

Moderator:
Jeffrey M. Bockman – VP, Defined Health

Panelists:
  • Michael Elliott – VP, Immunology Scientific Innovation, Johnson & Johnson
  • Anand Gautam – Director, Biopharm Innovation Sourcing, Novo Nordisk
  • Michael Gilman – CEO, Padlock Therapeutics
  • Debanjan Ray – VP, Business Development and Alliance Management, CytomX
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11:15 am–12:15 pm
Workshop
Early stage financing: The new roadmap to funding R&D

The investor landscape has changed and now more than ever before there are new opportunities to attract capital. This session will identify new categories of investors, share practical and tactical methods to raise capital, provide insight into the regional, national, and global investment arenas, and highlight the latest best practices being deployed by fundraising executives.

Moderator:
Dennis Ford – Founder and CEO, Life Science Nation

Panelists:
  • Rick Berenson – CEO, Thermalin Diabetes; Executive Committee Member, Mass Medical Angels
  • Meredith Fisher – Director and Partner Investments, Private Investment Office
  • David Sandak – VP, Strategy and Research, Accelerate Brain Cancer Cure
  • Dave Taylor – Founder and Managing Director, Mankind Venture Group (MvG)
  • David Verrill – Founder and Managing Director, Hub Angels Investment Group LLC
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12:30–1:30 pm
Level 4, Exhibit; Level 3, Gloucester
Luncheon
1:30–4:30 pm
Company Presentations ►
4:45–5:45 pm
Keynote Panel
The nearest exit may be behind you: The story of CoStim Pharmaceuticals

On February 17, 2014, CoStim Pharmaceuticals, a little startup just about a year removed from its first big funding round and focused on the hot field of immune checkpoint biology, was acquired by Novartis. In this session, the key players—co-founders, investors and Novartis—will share the company's story, from vision and formation through exit and beyond.

Moderator:
Michal Preminger – Executive Director, Office of Technology Development, Harvard University

Panelists:
  • Philip Gotwals – Executive Director, Oncology Research Collaborations, Novartis Institutes for Biomedical Research
  • Roger Kitterman – Managing Partner, Partners HealthCare Innovation Fund
  • Robert Millman – Managing Director, MPM; Co-Founder and President, CoStim Pharmaceuticals
  • Arlene H. Sharpe – Co-Founder, CoStim Pharmaceuticals; George Fabyan Professor of Comparative Pathology, Harvard Medical School
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5:45–7:00 pm
Level 4, Exhibit
Harvard Innovation Exhibit and Reception

Hosted by:

Harvard University Harvard University
Wednesday, September 24, 2014
7:30 am
Level 4, Foyer
7:30 am
Level 4, Grand Ballroom
Exhibition opens and Continental Breakfast
8:30 am–12:30 pm
Company Presentations ►
8:00 am–3:00 pm
One-to-one Meetings ►
9:15–10:15 am
Panel Discussion
All innovation roads lead to Massachusetts: Pharma's approach to the Boston biotech cluster

Undoubtedly, Boston is one of the most fertile biotech breeding grounds in the world. Top research institutes, early stage VC money, the who-is-who in serial entrepreneurs and a solid representation of mature biotechs, all within a few square miles, create an unmatched environment for innovation to thrive. No wonder most pharma companies are building a strong presence to be close to the action. During this session the leaders of several Boston R&D centers will discuss their approach to accessing innovation.

Moderator:
Johannes Fruehauf – Founder and President, LabCentral; CEO, Cambridge BioLabs

Panelists:
  • Kumar Srinivasan – VP, Scientific Partnering and Alliances, AstraZeneca
  • Benjamin Thorner – Associate VP, Head of Business Development and Licensing, Boston Innovation Hub, Merck Research Laboratories
  • Robert Urban – Head, Johnson & Johnson Innovation LLC, Boston
  • Leigh Zawel – Site Head, Boston Center for Therapeutic Innovation (CTI), Pfizer BioTherapeutics R&D
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10:30 am–12:30 pm
Academic Innovators™ Showcase

Join us for our Academic Innovators™ presentation track as we work with industry leaders to shine a spotlight on the many scientific advances with strong translational potential being made in the academic world.

Moderators:
Shakti Narayan – Senior Director, Transactions, Johnson & Johnson Innovation
Jeffrey S. Nye – VP, Neuroscience, Innovation and Scientific Partnership Strategy, Johnson & Johnson Innovation

Organizations:
  • Boston Children's Hospital
  • Broad Institute
  • Cincinnati Children's Hospital Medical Center
  • Duke University
  • Harvard University OTD
  • Hospital for Sick Children
  • New York University Medical Center
  • University of Coimbra
  • University of Texas at Austin
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12:30–1:30 pm
Level 4, Exhibit
Luncheon
3:00–4:00 pm
Level 4, Exhibit
Closing Reception
Arrow Up


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