February 10-11, 2014 The Waldorf Astoria New York Now in its sixteenth year, the BIO CEO & Investor Conference is the largest independent investor conference focused on leading publicly-traded biotech companies, with a special focus on select established private companies. The meeting provides a neutral forum where institutional investors, industry analysts, and senior biotechnology executives have the opportunity to shape the future investment landscape of the biotechnology industry. Reasons Top 10 to attend 1 35% of the biotechs who filed an IPO in 2013 will be presenting at this year’s BIO CEO & Investor Conference. 2 Hear the Washington perspective on the Affordable Care Act and other timely policy developments affecting the industry. 3 Evaluate fresh investment opportunities including compatible, complementary and competitive companies. 4 Learn about the hottest clinical developments and industry catalysts by attending the conference’s therapeutic workshops and business roundtables. 5 Attend fireside chats with CEOs who will share their recent company successes, what keeps the C-suite up at night, and where the industry’s leading companies are headed in 2014. 6 Gain access to BIO’s 1x1 Partnering SystemTM for scouting potential investments and deal partners, optimizing your time at the event. 7 Hear presentations from more than 150 established public and private biotech companies and non-profit funding organizations, including many you won’t hear from at other investor conferences. 8 Get the pulse of the current and proposed investment trends in biotechnology. 9 Network with peers, investors, and potential partners attending the conference and our exclusive receptions. -2- Who Should Attend? Audience Profile Conference Size 1,400 One-on-One PartneringTM Meetings 1,350+ Investors Attending 700 Company Presentations 150 Market Cap >$1B+: 9% 500M-1B companies <50M 8% 300+ 24% Nearly 700 investors 250-500M 16% 50-100M 20% 100-250M 23% Rare/Genetic Therapeutic Focus Diseases: 2% Gastrointestinal Ophthalmology Disorders: 2% 2% Vaccines: 2% Diagnostics: 2% Renal: 3% 3D Metabolic: 4% Oncology 130/160/20/35 Cardiovascular 29% 4% Infectious Over 1,400 attendees Diseases: 6% CNS from 20 different countries 13% Cell Therapy/ Other Regenerative 10% Over 35% of attendees Medicine: 6% are executives Autoimmune/ Inflammation: 6% Platform/ Discovery: 9% -3- 3D 130/160/20/35 Fireside Chats Fireside Chats feature candid discussions between biopharma executives, Wall Street analysts, and other high-level industry experts. Hear about recent company successes, what keeps the C-suite up at night, and where the industry’s leading companies are headed in 2014. Leonard Bell, M.D. CEO, Alexion Pharmaceuticals Belén Garijo, M.D. President & CEO Merck Serono Richard F. Pops Chairman & CEO Alkermes plc David E.I. Pyott Chairman of the Board & CEO Allergan Christopher A. Viehbacher CEO, Sanofi Chairman, Genzyme -4- Plenary Sessions Opening Remarks • James C. Greenwood, President & CEO, Biotechnology Industry Organization Opening Plenary: A Room with a (Buy-Side) View: The Year Ahead Monday, February 10th, 12:00pm – 12:55pm Join us for a scenic view of 2014 with portfolio managers and senior buy-side analysts. This lively panel discussion will be primed with the results of this year’s Investor Perception Study, conducted by BIO’s Industry Analysis Team and International Strategy & Investment (ISI). Panelists will field a range of questions on the peaks and valleys of the 2014 landscape, from valuations and volatility to challenges and opportunities facing institutional investors today. Moderator: • Mark Schoenebaum, MD, Senior Managing Director, ISI Group Panelists: • Andrew Acker, CFA, Portfolio Manager, Janus Global Life Sciences • Oleg Nodelman, Founder & Managing Director, EcoR1 Capital • William S. Slattery, Partner, Deerfield Management Company Closing Plenary: Fundraising in the New Capital Market Environment Tuesday, February 11th, 4:00pm – 4:55pm 2013 was clearly the year of the IPO. As a result, a dynamic fundraising environment emerged last year. Many biotechs took full advantage of this market to raise capital - through follow-ons and PIPEs after their initial debut. What’s next? What challenges will await these newly public companies in 2014, or those private biotechs waiting in the wings? Hear industry experts discuss why the markets are doing well and what effect it has had on the entire biotech ecosystem. What’s their advice on how can we keep the momentum, and what do biotech CEOs need to look out for in 2014? Moderator: • Jonathan L. Kravetz, Chair of the Securities Practice Group, Mintz Levin Panelists: • John Chambers, Managing Director, Head of Healthcare Investment Banking, ROTH Capital Partners • David-Alexandre Gros, MD, Executive Vice President, Chief Strategy Officer, Sanofi Speakers added daily. -5- Therapeutic Workshops Delivering the [RNAi] Goods Monday, February 10th, 10:30am - 11:25am Investing in the RNA space? What delivery technology will win the race to turn these novel molecules into ap- proved therapies? Is there a “winner take all” in this space or does each type of nucleic acid technology require a unique platform for delivery? This panel will discuss ongoing innovation in the nanoparticle, liposomal, nucleic acid, and conjugate-based approaches to delivering one of the most promising modalities in biotech. Panelists: • Douglas Fambrough, PhD, CEO, Dicerna Pharmaceuticals • Bruce Given, MD, Chief Operating Officer, Arrowhead Research Corporation • John Maraganore, PhD, Chief Executive Officer, Alnylam Pharmaceuticals • Kleanthis G. Xanthopoulos, PhD, President & CEO, Regulus Therapeutics Balancing the Risk-Reward in Rare Disease Drug Development Monday, February 10th, 2:00pm – 2:55pm There were certainly positive developments in the rare diseases space last year – as we saw with the success of recent IPOs and new deals. These possibilities of large returns often outweigh the inherent risk involved, and while the path to approval is not always as straightforward as it seems, success is there to be had. Challenges also exist throughout development however, from validating endpoints and reimbursement costs, to the difficulties of trial recruitment. How do you navigate this dynamic environment? CEOs of companies paving the way will be on-hand to discuss the advantages and challenges involved in bringing rare disease treatments to market, and industry experts will dissect some of the myths that have revolved around the space. Panelists: • Timothy R. Coté, MD, MPH, Principal & CEO, Coté Orphan Consulting • Hans GCP Schikan, PharmD, Chief Executive Officer, Prosensa Keeping an “I” on Oncology: The ASCO Immunotherapy Preview Tuesday, February 11th, 1:30pm – 2:25pm Immuno-oncology has been earning the spotlight over the past year, having been named as Science’s Breakthrough of the Year, and this year’s ASCO will be no exception. Expert panelists will discuss the latest developments in the immuno-oncology space. What will be the talk of ASCO? What does it take to get breakthrough status? Why has this field taken so long to come to fruition? Are immunotherapies the answer to managing cancer over a lifetime? Panelists: • Annalisa Jenkins, MBBS, MRCP, Executive VP & Head of Global Research and Development, Merck KGaA • Michael G. King, Jr., Senior Analyst, Biotechnology, JMP Securities • Anthony S. Marucci, Founder, President, CEO & Director, Celldex Therapeutics • Jon Wigginton, MD, Senior Vice President, Clinical Research, MacroGenics -6- Business Roundtables Solution Development: Aiming the Whole Pipeline at a Single Goal Monday, February 10th, 9:00am - 9:55am One model for maximizing biotech value creation is ‘Solution Development’, which calls for a company to develop multiple agents for a single overarching indication, either to identify the best one of many or to develop combinations. The panel will contrast this Solution Development model against the traditional model of developing a single drug for a single indication or multiple drugs but for disparate indications. How will using a Solution Development model impact clinical/regulatory strategy, competitive positioning, financing strategy, and M&A game theory? Moderator: • Peter Kolchinsky, PhD, Managing Director, RA Capital Management, LLC Panelists: • Christopher Anzalone, PhD, President & CEO, Arrowhead Research Corporation • Stanley C. Erck, President & CEO, Novavax • Milind S. Deshpande, PhD, President & CEO, Achillion Pharmaceuticals • Kurt Graves, Chairman, President & CSO, Intarcia Therapeutics The Reimbursement Landscape Under ACA Tuesday, February 11th, 12:00pm – 12:55pm The Affordable Care Act brings a great promise to the biotech industry and our nation as a whole. Now, a couple months in, what do we know? What pressure will ACA put on the forces that impact product reimbursement, and how will those forces respond? What can the biomedical ecosystem do to protect the industry’s pipelines? How did – and have - companies prepared? Come hear a discussion among industry representatives on this new environment under ACA, and get their take on where the law will lead one of the most important elements of biotech development. Panelists: • J.D. Kleinke, Healthcare Business Strategist • Laurel Todd, Managing Director, Reimbursement & Health Policy, BIO Let’s Make a Deal – Strategic Licensing and M&A Trends Tuesday, February 11th, 9:00am - 9:55am This panel will evaluate licensing and M&A trends of recent deals as the parties involved aim to get the biggest bang