2013 | November 3-5 Grand Hyatt Hotel New York City P4C Connect helps you find ADVANCE YOUR nontraditional allies—like leaders from nonprofit foundations, academic institutions, and R&D OBJECTIVES philanthropic organizations— who can help you advance THROUGH your R&D goals. REVIEW PROFILES OF OTHER P4C PARTICIPANTS. P4C CONNECT! SCHEDULE ONE-ON-ONE AND/OR GROUP MEETINGS. BUILD YOUR CUSTOMIZED P4C SCHEDULE—FROM PANELS TO PRESENTATIONS TO PARTNERING MEETINGS—AND SYNC IT WITH YOUR CALENDAR OF CHOICE. It also allows you to request free, private, 25-minute advice sessions with expert consultants on: STRATEGIC PLANNING COLLABORATION STRUCTURING ATTRACTING CAPITAL CLINICAL RESEARCH, REGULATORY, & REIMBURSEMENT STRATEGY Get the mobile P4C Connect app: P4C Connect is accessible DATA NETWORKING via your iPhone, iPad, or Android device. You can download it from the iTunes App Store, Google Play Store, or scan the All partnering meetings are held on QR codes here. the Conference Level. iPHONE iPAD ANDROID TABLE OF CONTENTS WELCOME LETTERS ..........................................................................2 HOTEL MAP .........................................................................................4 PROGRAM & ACTIVITIES ................................................................... 5 THERAPEUTIC AFFINITY & RESEARCH BUILDING BLOCKS ROUNDTABLES ................................................................. 33 PANELISTS ....................................................................................... 37 EXPERT CONSULTANTS ................................................................ 123 SPONSORS ......................................................................................141 TEAM ..............................................................................................163 ACKNOWLEDGMENTS .................................................................... 167 WELCOME TO THE FIFTH PARTNERING FOR CURES CONFERENCE! Partnering for Cures is a platform for big ideas, transformative programs, and tangible results brought forth by some of the most disruptive innovators. Each year, a kinetic energy permeates this meeting as participants are bound by a shared sense of urgency and a drive toward solutions. We measure the impact of this conference by the progress each of you can make—whether it’s finding vital new partners or garnering the latest intel on innovative programs or leaving with new vantage points to approach a problem. We’ve designed the program to do just that: • 16 dynamic panel discussions will tackle issues that are key to advancing medical progress. From big science to big data to big systems, the issues at hand are immense. With more than 80 panelists focused on collaborative solutions that are forward thinking and pragmatic, we know that you will leave with great optimism and practical strategies. • 30 innovators will be presenting their cross-sector programs—chosen after the most competitive application cycle to date. From novel financing models to adaptive clinical trial designs to data sharing initiatives, these programs provide all of us with a first-person account of the innovation that is underway. Take the time to review these programs—their transformative nature is sure to have ripple effects for years to come. • P4C Connect, our easy-to-use partnering system, is yours to utilize and leverage. You can schedule one-on-one meetings to advance your own research and development goals. This system allows participants—from innovators to investors, scientists to philanthropists, federal officials to advocates, to find each other and get things done. Through this system, you can also request private pro-bono consulting sessions with dozens of leading experts ready to give advice on topics including attracting capital, collaboration structuring, and regulatory and reimbursement strategy. • Time Equals Lives Talks—the latest addition to the program will feature remarkable speakers with incredible stories to tell about what drives them to continue to pave the path toward solutions— sometimes against many odds. Each 10-minute story will bring to life the compelling personal factors that come to play in the search for cures. • Numerous opportunities for productive collisions. Starting with breakfast roundtables focused on therapeutic affinity areas or research tools, networking opportunities throughout, and a late night gathering that will entertain and inspire you. • The P4C Lounge—be sure to stop by and check out the photo booth, add your thoughts to the “big picture,” and take advantage of areas for networking! We so appreciate your carving out time to join us. We know you will find these two days a productive and meaningful use of your time. Enjoy the meeting! Sincerely, Margaret Anderson Michael L. Klowden Executive Director CEO FasterCures Milken Institute T HE C ITY OF N EW Y ORK O FFICE OF THE M AYOR N EW Y ORK, NY 10007 November 3, 2013 Dear Friends: It is a pleasure to welcome everyone to New York City as the Milken Institute’s FasterCures center convenes the fifth annual Partnering for Cures meeting. New York really is the perfect place to host this event. Our city has a wealth of individual and institutional medical talent. We are also increasingly becoming a capital of the commercial life sciences, thanks in part to the ready access to venture capital that entrepreneurs have here. Earlier this fall, we celebrated the opening of the New York Genome Center, to which our Administration contributed $5 million in financing. These kinds of targeted investments are key to bringing new products and procedures to the market, and together – during this, the era of healthcare reform and innovation – we can expect Partnering for Cures to further advance the efforts of medical researchers from across the metropolitan area and beyond. On behalf of our great city, I offer my best wishes for a productive meeting and continued success in your vitally important work. Sincerely, Michael R. Bloomberg Mayor FINDING YOUR WAY AROUND Elevators Registration Empire V Empire Ballroom Empire I Breakout Breakout Ballroom Level Majestic Partnering Shubert Help Desk Elevators Booth Partnering Booths 3 Imperial 2 Morosco 1 Wintergarden Plymouth Palace Broadway-Julliard-Uris Royale Alvin-Carnegie Networking Lounge Conference Level 1. Ambassador 2. Orpheum 3.Gershwin Elevators Regency Room Park Ave. Entrance Park Chrysler Avenue Room Mezzanine Level Elevators Front Desk Concierge 4 Lobby Level 42nd St. entrance PROGRAM & ACTIVITIES PROGRAM & ACTIVITIES & PROGRAM NOVEMBER 3, 2013 SUNDAY ACTIVITIES & PROGRAM 6:00 PM - 8:00 PM EMPIRE BALLROOM FOYER Welcome Reception & Registration PARTNERING FOR CURES CURES NOVEMBER 4, 2013 MONDAY 7:00 AM - 7:00 PM EMPIRE BALLROOM FOYER Registration 7:00 AM - 8:00 AM EMPIRE BALLROOM Therapeutic Affinity & Research Building Blocks Breakfast Roundtables (For a list of roundtables and hosts, go to page 35) 8:00 AM - 9:00 AM EMPIRE BALLROOM Opening Plenary Big Science in the 21st Century SPEAKERS: The BRAIN (Brain Research through Advancing Innovative Francis Collins, Director, Neurotechnologies) effort initiated by the Obama Administration National Institutes of Health has captured imaginations as a national commitment to unlock Michel Goldman, Executive Director, the secrets of the human brain and find new ways to fight and treat Innovative Medicines Initiative neurological disease. Described by some as the next “big leap” since Richard Pops, Chairman and CEO, the Human Genome Project, completed more than a decade ago, Alkermes how does or should a “big science” project like this look different in 2013? Is this the right place to focus our scientific energies? What can Arati Prabhakar, Director, Defense be done to more quickly spin basic discoveries from such efforts out Advanced Research Projects Agency into therapies? Who should decide what the “grand challenges” Marc Tessier-Lavigne, President, to pursue are? And how does the rest of the scientific community The Rockefeller University get integrated with efforts like this – including citizen scientists? MODERATOR: Margaret Anderson, Executive Director, FasterCures 7 MONDAY 9:15 AM - 12:00 PM BOOTH/IMPERIAL/MOROSCO Partnering and Expert Consultations PROGRAM & ACTIVITIES & PROGRAM 9:15 AM - 10:10 AM ALVIN The Other Translation Gap: SPEAKERS: Turning Outputs into Outcomes Peter Margolis, Professor of Pediatrics Beyond the “first translation gap,” between basic scientific and Director of Research, James M. discoveries and research in human subjects, is “T2,” the gap Anderson Center for Health Systems Excellence, Cincinnati Children’s between approval of a treatment and its adoption into Hospital Medical Center widespread use to improve patient outcomes, which can take PARTNERING FOR CURES BREAKOUT PANEL as long as the R&D process. Where are we seeing examples Joe V. Selby, Executive Director, Patient- of rapid learning systems that are working to speed the Centered Outcomes Research Institute adoption of evidence-based care practices and improve Mark Wagar, President, health? What’s needed to build and sustain these efforts? Heritage Medical Systems John Walsh, President and CEO, Alpha-1 Foundation MODERATOR: Cecilia Arradaza, Director, Communications & Policy, FasterCures 9:15 AM - 10:10 AM EMPIRE BALLROOM I The Art and Science of Multi-Stakeholder SPEAKERS: Collaboration Walter Capone, Chief Operating Officer, So many challenges in medical research require solutions Multiple Myeloma Research Foundation that engage multiple stakeholders,