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Trademark Trial and Appeal Board Electronic Filing System. http://estta.uspto.gov ESTTA Tracking number: ESTTA1088285 Filing date: 10/13/2020

IN THE UNITED STATES PATENT AND TRADEMARK OFFICE BEFORE THE TRADEMARK TRIAL AND APPEAL BOARD Proceeding 91247208 Party Plaintiff MedImmune, LLC Correspondence DAVID M KELLY Address KELLY IP LLP 1300 19TH STREET NW, SUITE 300 WASHINGTON, DC 20036 UNITED STATES Primary Email: [email protected] Secondary Email(s): [email protected], [email protected] 202-808-3570

Submission Testimony For Plaintiff Filer's Name DAVID M. KELLY Filer's email [email protected], [email protected], [email protected] Signature /David M. Kelly/ Date 10/13/2020 Attachments Group 5 Cover.pdf(16738 bytes ) Exhibit 10-1.pdf(3688207 bytes ) Exhibit 10-2.pdf(4274410 bytes ) Exhibit 10-3.pdf(3819747 bytes ) Exhibit 10-4.pdf(1951042 bytes ) Exhibit 10-5.pdf(2039739 bytes ) Exhibit 10-6.pdf(5220659 bytes ) IN THE UNITED STATES PATENT AND TRADEMARK OFFICE BEFORE THE TRADEMARK TRIAL AND APPEAL BOARD

MEDIMMUNE, LLC, Opposition No.: 91247208

Opposer,

v. Mark: Serial No.: 87953597 PRASUN J. MISHRA, PHD, Filed: June 7, 2018

Applicant.

DECLARATION OF RICHARD BUCKLEY

EXHIBIT 10

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MED001643 9/22/2020 Prasun Mishra, Founder & CEO of Agility Pharmaceuticals talks about recent advances & analytics contribution in genome editing techno…

Prasun Mishra, Founder & CEO of Agility Pharmaceuticals talks about recent advances & analytics contribution in genome editing technologies

Amit Shelke Follow Marketing Manager - UK, Europe… 22 0 0

https://www.linkedin.com/pulse/prasun-mishra-founder-ceo-agility-pharmaceuticals-talks-amit-shelke/ 1/6 MED001644 9/22/2020 Prasun Mishra, Founder & CEO of Agility Pharmaceuticals talks about recent advances & analytics contribution in genome editing techno…

Dr. Prasun Mishra - Founder and CEO of the Agility Pharmaceuticals recently spoke to MarketsandMarket’s team sharing the valuable insights into the industry with respect to emerging areas, challenges and innovations. Dr. Mishra (Ex-Genentech, Ex-NCI/NIH) has been leading Agility Pharmaceuticals, a pharmaceutical company driven by artificial intelligence (AI) to revolutionize drug discovery through BigData, Robotics and AI. He is the founding president and CEO of American Association for Precision Medicine (AAPM), one of the world’s leading professional organizations dedicated to advancing the field of precision medicine. We interviewed Dr. Mishra asking about his expert insights on the genome editing space, his presence at the 3rd Annual Genome Editing & Engineering Conference and his take on the conference program. Q: What are the current emerging areas of Genome Editing? Dr. Mishra: Recent advances in genome editing technologies have taken the scientific community by storm, working their way into virtually every corner of biological inquiry, including human health. Advances to human health by gene editing applications can be summarized in following four categories: From basic research to translational applications: Gene editing of cells, tissues, germline cells has provided a deeper understanding of human biology and disease mechanisms that has pioneered new approaches of treatment. Discovery of CRISPR-Cas9 system for genetic manipulation has been one of the most exciting developments in recent times which is already redefining our approach in identifying new drugs/targets Somatic cell genome editing to prevent/treat human diseases and disorders: Human genome editing in somatic cells, to treat/prevent a variety of genetically inherited diseases, has an immediate clinical application. This approach is already in clinical trials for several indications including to treat children with the severe combined immune deficiency or “bubble baby” disease, now in a clinic (De Ravin et al., 2016) as well as to restore hearing in mice (Pan et al., 2017, Nature Biotechnology). Recent FDA approval of tisagenlecleucel for acute lymphoblastic leukemia and axicabtagene ciloleucel for lymphoma brought gene therapy to clinic in the US Heritable genome editing to prevent/alleviate the suffering caused by genetically inherited diseases: Gene editing presents an opportunity to prevent thousands of genetically inherited diseases and can be used as a potential “surgical knife” to correct genetic mutations. For example gene editing technology was used to correct a gene mutation in human embryos https://www.linkedin.com/pulse/prasun-mishra-founder-ceo-agility-pharmaceuticals-talks-amit-shelke/ 2/6 MED001645 9/22/2020 Prasun Mishra, Founder & CEO of Agility Pharmaceuticals talks about recent advances & analytics contribution in genome editing techno… causing hypertrophic cardiomyopathy, a genetic heart disease leading to sudden cardiac arrest Sign in Join now (Ma et al., 2017, Nature) Enhancements: Gene editing can also be supplemental to human health, including enhancing human capabilities further. However, there are ethical concerns that "unregulated genetic engineering may lead to a new form of eugenics, in which people with means pay to have children with enhanced traits even as those with disabilities are devalued (Belluck, 2017, NYTimes).” (https://www.nytimes.com/2017/08/02/science/gene-editing-human-embryos.html) There are also safety concerns associated with gene editing technology raised by a recent study that attempted to restore sight in blind mice, using CRISPR-Cas9, observed “unexpected mutations" in secondary genes (Schaefer et al., 2017, Nature Methods). Hence, safety and efficacy concerns should be carefully addressed in human clinical trials employing gene editing applications. Q: For gene editing, analytics plays a vital part. How do you think analytics contributes in gene editing applications? Dr. Mishra: Data analytics tools are a key in optimizing each step of genome editing experiments regardless of the genome-editing technique utilized. Briefly, a careful monitoring and analytics of the gene editing process starting with cell culture step (accurate cell counts, viability counts & measure apoptosis), the actual genome editing step (measuring delivery of lentivirus, Cas9, guide RNA as well as confirmation of DNA cleavage and gene editing) to the cell phenotyping step (primary target and off-target phenotyping) contributes to efficient gene editing results. Moreover, several next-generation sequencing data analytics tools have made the sequence level verification of guide RNA, primary target and off-target phenotyping analytics steps more effective. Although all these steps can be analyzed separately, there are still opportunities to innovate around integrating data analytic applications for the entire gene editing process.

Q: You will be speaking at the 3rd Annual Genome Editing and Engineering Conference, could you please elaborate on few take away points from your presentation topic which will help in building the knowledge base for attendees? Dr. Mishra: I am excited to discuss innovative solutions for pharmaceutical and healthcare industry using artificial intelligence. The title of my talk is “Augmenting Human Intelligence through Artificial Intelligence.” I will focus on how we can use accelerate drug discovery process using big data analytics, robotic screens and artificial intelligence (AI). Briefly, I will discuss how big data analytics can be utilized to make complex biology simpler. Moreover, I will also emphasize the importance of automation in drug discovery process and how we can use genotypic and phenotypic screening to understand high throughput biology. I also hope to give the audience an overview of recent advances in AI field and how we can exploit it for drug discovery and precision medicine. https://www.linkedin.com/pulse/prasun-mishra-founder-ceo-agility-pharmaceuticals-talks-amit-shelke/ 3/6 MED001646 9/22/2020 Prasun Mishra, Founder & CEO of Agility Pharmaceuticals talks about recent advances & analytics contribution in genome editing techno…

Q: After seeing the Agenda for 3rd Annual Genome Editing and Engineering Conference, what are your views and how helpful do you think it is for the targeted audience? Dr. Mishra: The agenda for the Genome Editing & Engineering Conference looks very interesting and I am looking forward to meeting old friends and making new ones. This conference brings together some of the great minds in the field from both industry and academia discussing genome editing. Among many interesting talks, the speakers will also discuss CRISPR screens (to better understand biology and drug discovery applications), data analytics, gene activation, efficient animal genome engineering and improving the accuracy of gene editing methods. The audience would also gain knowledge in clinical applications of genome editing in muscular dystrophy, friedreich, microsatellite diseases as well as therapeutic genome editing of hematopoietic stem cells. Those who are interested in immunotherapy would learn about the production of allogeneic CAR-T cells and its applications in adaptive allogeneic T-cell immunotherapy.

https://www.linkedin.com/pulse/prasun-mishra-founder-ceo-agility-pharmaceuticals-talks-amit-shelke/ 4/6 MED001647 9/22/2020 Prasun Mishra, Founder & CEO of Agility Pharmaceuticals talks about recent advances & analytics contribution in genome editing techno… Dr. Prasun Mishra is one of the keynote speakers of the 3rd Annual Genome Editing & Sign in Join now Engineering Conference taking place on 08 – 09 February 2018 in San Diego USA. He will be presenting on the Day 1 of the conference on the topics - Augmenting Human Intelligence through Artificial Intelligence. Know more about his presentation visit our website - http://bit.ly/2hYF8sQ If you wish to learn a great deal from him over the insights of Drug Discovery, then register now online at http://bit.ly/2zqdXRv or email [email protected] to book your registration.

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Amit Shelke Follow Marketing Manager - UK, Europe & …

Insights on Precision Genome Editing by Dr. Prasun Mishra - Founder & CEO of Agility Pharmaceuticals

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MED001650 THE HISTORY OF PRECISION MEDICINE: THE LARGEST PRECISION MEDICINE WHERE TECHNOLOGY MEETS SCIENCE EVENT IN THE WORLD FOCUSING ON COMMERCIAL, ACTIONABLE GOALS. SCIENTIFIC BREAKTHROUGHS STATE OF HIT/MEDICAL 1871 Fredrich Meischer identiies “nuclein”, now known as DNA, in the nucleus of cells TECHNOLOGY 1910 Albrecht Kossel is awarded the Nobel Prize for his discovery of the four base pairs 100+ SPEAKERS 1950 Erwin Chargaff discovers the complimentary base pairing system 1960’s 1953 Watson and Crick determine the double-helix Computers begin to give rise to structure of DNA shared accounting systems at hospitals 1961 Codons discovered 1968 Robert Holley wins Nobel Prize for sequencing irst tRNA 1970’s 40+ PHARMA & BIOTECH COMPANIES Computers are now small enough to have departmental systems in 1977 Frederick Sanger develops irst DNA sequencing method hospitals

1980’s REGUL ATORS 1983 PCR DNA Ampliication technique 12+ Integrated inancial and clinical developed by Kary Mullis at Cetus Corporation systems begin to emerge, as 1985 Alec Jeffreys develops a method for DNA proiling well as managed care inancial and administrative systems. Early 1990 Human Genome Project is launched with goal of 15 years departmental imaging 1993 Fred Sanger opens the Sanger Center HOSPITAL SYSTEMS 1995 First bacterial genome is sequenced 1990’s 21+ 1999 Chromosome 22 irst chromosome fully sequenced, Expanded clinical departmental ensemble genome browser launched solutions and increased IDN-like integration. Emergence of integrated EHR 2000 Development of irst prenatal diagnostic offerings. 2000 Drosophila genome sequenced 6+ VENTURE CAPITAL 2001 First draft of the human genome released 2000’s 2002 Mouse is irst mammal to have genome sequenced Emergence of commercial, 2003 Human genome project completed real-time clinical decision support. Evolution of mobility. 2007 New gene sequencing technology developed, increasing capability 70-fold 5+ PAYERS 2008 Apple introduces the iPhone

2008 Barack Obama elected President of the United States 2010 PATIENT GROUPS 1,000 genomes project launched 1,000 genomes project results published 4+ 2012 ENCODE study publishes 30 papers of results of active sites in genome 2013 HIT begins to focus on data warehousing and analytics solutions President Obama launches the Precision Medicine Initiative 2015 US Supreme Court rules that naturally occurring DNA cannot be patented 2016 Launch of the World Precision Medicine Congress USA in Buy your ticket now at www.terrapinn.com/attendprecision Washington, D.C. in partnership with the FDA

MED001651 “THE MORE WE LOOKED AT IT, USING GENETIC BIOMARKERS TO DEVELOP DRUGS FOR SPECIFIC PATIENT POPULATIONS, THE MORE WE REALIZED THAT THIS WAS THE FUTURE.”

Mark Clein SOURCE: https://www.washingtonpost.com/business/economy/ CO-FOUNDER AND PRESIDENT value-added-these-health-care-entrepreneurs-try-to-keep-win-streak- PRECISION FOR MEDICINE going/2016/02/07/ebecdd8e-ca09-11e5-88ff-e2d1b4289c2f_story.html GROUP

MED001652 Robert M Michael Niven Jeffrey Karina Sicco Glenn Rahul John Maslowski Andre Robert Elizabeth Califf Linden Narain Reid Bienfait Popma Barnes Aras Vice President of Choulika Hariri Read Commissioner VP, Gene Therapy Head, Co-Founder, President Executive Director of Global Head, Genomic Scientiic Director of Gene Associate Director, Translational Chief Executive Oficer Scientiic Affairs Chairman & Chief Chairman, Founder and CSO SVP Product Development F.D.A. Genetic Medicine Institute & CEO Genome Informatics Strategy, Principal Scientist Modiied Cell Therapies Medicine Operations JUVENTAS FIBROCELL Executive Oficer CELGENE CELLULAR MEDEOR PFIZER BERG LLC REGENERON MERCK AND JANSSEN RESEARCH TAKEDA THERAPEUTICS SCIENCE INC CELLECTIS THERAPEUTICS THERAPEUTICS, INC. GENETICS CENTER CO INC & DEVELOPMENT CAMBRIDGE US

James R Eric Hans Michael D Charles Amir Jianda Richard Rao Aarash Erica Francois Prudent David Klingemann West Nicolette Handzel Yuan Buller Mulpuri Bordbar Ramos Lebel President and Chief Executive Chief Strategy Oficer & SVP CMO & CSO Ph.D., Chief Executive Chief Scientiic Oficer Statistical Science Director of Translational Immuno Vice President and Head, Chief Operating Oficer Co-Founder Senior Genetic Chief Medical Oicer Oficer Preclinical Development NANTKWEST Oficer and Director ARGOS Director Oncology Research Clinical Development PROVISTA SINOPIA Counselor ZIOPHARM CENTROSE ORGANOVO BIOTIME, INC. THERAPEUTICS ASTRAZENECA MERCK PFIZER DIAGNOSTICS BIOSCIENCES ILLUMINA ONCOLOGY, INC. ONCOLOGY

INDUSTRY LEADERS LEADING THE PRECISION MEDICINE REVOLUTION ARE SPEAKING AT WORLD PRECISION MEDICINE CONGRESS USA 2016

Steven Abdel Steven Adam Naftali Murray Brad Jonathan David Barbara Ena Matt Averbuch Halim Rosen Resnick Kaminski Brilliant Perkins Morris Pearce Conley Bromley Nelson Vice President of Translational Vice President Translational Senior Director, Diagnostic Founding Director, Center BOEHRINGER Senior Research Scientist Chief Medical Oficer Assistant Professor Director, Sanford Children's Associate Director President and Chief Scientiic Director, Genetics Clinical Development Medicine, Biomarkers and Strategy for Data Driven Discovery in INGELHEIM and Center Director HUMAN MAYO Health Research Center NATIONALCANCER Oficer GSK and Pharmacodiagnostics Diagnostics NOVARTIS Biomedicine Professor of Medicine MARSHFIELD LONGEVITY INC CLINIC SANFORD INSTITUTE BIOSTAT BRISTOL MYERS CELLDEX THE CHILDREN'S YALE SCHOOL OF CLINIC RESEARCH RESEARCH SOLUTIONS INC SQUIBB THERAPEUTICS HOSPITAL OF MEDICINE FOUNDATION PHILADELPHIA

David Jason Jennifer Craig Amit Yuong Bing Carl Gary Lavinia Andres Jeremy Brindley Moffat Hoskovec Volker Aggarwal Yue Yuan Simon Palmer Ionita Hurtado-Lorenzo Segal Senior Research Fellow Associate Professor NSGC Prenatal Expert Head of Oncology Head of Bioinformatics Head of Computational Executive Director and Global Biologist Chief Medical Oficer CEO Director of Translational Director of Clinical Genomics in Healthcare Translation UNIVERSITY OF NATIONAL SOCIETY Bioinformatics ELI LILLY AND Biology Lead of Clinical Stage Oncology NATIONAL INSTITUTE NANTHEALTH OMIXY Medicine and Molecular Diagnostics OXFORD TORONTO OF GENETIC GLAXOSMITHKLINE COMPANY BOEHRINGER Business Development OF STANDARDS & CRONH'S AND COLITIS Laboratories UNIVERSITY COUNSELORS INGELHEIM MERCK TECHNOLOGY FOUNDATION OF UNIVERSITY OF AMERICA CHICAGO MEDICINE MED001653 Steven Marcie Stephen Jan Clary Patricia Claudia Stephen Benjamin D Nathan Katherine Daryl Deitcher Glicksman Baylin Scicinski Clish Weltin Rizzini Groft Solomon Price K. Leon Kallevig CEO Chief Scientiic Deputy Director - The Sidney Sr. VP and Chief Director of the CEO Managing Director Senior Advisor to the Chief of Division of Associate Director Co-Founder and Director Chief Information MEDEOR Oficer Kimmel Comprehensive Scientiic Oficer Metabolomics Platform RARE DISEASE BRIGHAM AND Director, NCATS Medical Genomics INSTITUTE FOR of Communications Oficer THERAPEUTICS, INC. ORIG3N Cancer Center EPICENTRX BROAD UNITED WOMEN'S HOSPITAL N.I.H INOVA SYSTEMS BIOLOGY SCAD ALLIANCE RIVERWOOD JOHNS HOPKINS INSTITUTE FOUNDATION AND MASSACHUSETTS TRANSLATIONAL HEALTHCARE MEDICAL GENERAL HOSPITAL MEDICINE INSTITUTE INSTITUTIONS

Yaffa Ed Pezalla Peggy Mark David Ying Jak David Christopher Mark Fred Fred Rubinstein Vice President, National Peissig Gardner Smith Chen Knowles Ledbetter de Souza Rubin Lorey Meindl Program Director Medical Director for Center Director,Chief Chief Executive Oficer Professor of Laboratory Senior Bioinformatics Managing Director and Executive Vice President Director Founding Director Voting Member, HHS Secretary's Sales Director NATIONAL Pharmaceutical Policy Research Informatics Oficer OMNISEQ Medicine and Pathology Informatics Vice President of Medical and Chief Scientiic Oficer BROADVIEW Englander Institute Advisory Committee on Heritable PERKINELMER INSTITUTES OF and Strategy MARSHFIELD Chairman of the Technology RUTGERS CANCER and Scientiic Affairs GEISINGER VENTURES for Precision Medicine Disorders in Newborns and Children HEALTH AETNA CLINIC RESEARCH Assessment Group Center for INSTITUTE OF CUREDUCHENNE HEALTH SYSTEM WEILL CORNELL CALIFORNIA HEALTH FOUNDATION Individualized Medicine NEW JERSEY VENTURES MEDICAL COLLEGE AND HUMAN SERVICES MAYO CLINIC AGENCY

INDUSTRY LEADERS LEADING THE PRECISION MEDICINE REVOLUTION ARE SPEAKING AT WORLD PRECISION MEDICINE CONGRESS USA 2016

Omar Elizabeth David Kenna Mills Anup Sam Joshua Chris Jit Kurtis Mark Michelle Ali Ottinger Roth Shaw Kaneri Johnson Resnick Bardon Patel Bachman Clein Penny NHS Pharmacy Senior Project Director, Precision Executive Director and Portfolio Strategist Director, Health Policy and Biotechnology Managing Director Vice President US Head, Computational Co-Founder, Chief Director Computational Payer NICE Manager Medicine Program Institute For Personalized UNIVERSITY OF Interprofessional Affairs Partner MPM Oncology J.D.R.F. and Systems Biology Executive Oficer Biology and Genomics N.I.C.E. NATIONAL NIH PERELMAN SCHOOL Cancer Therapy PITTSBURGH AMERICAN COLLEGE S.V. LIFE Impact Fund JANSSEN PRECISION BIOGEN INSTITUTE FOR OF MEDICINE M.D. ANDERSON MEDICAL CENTER OF CLINICAL SCIENCES FOR MEDICINE HEALTH AND CLINICAL UNIVERSITY OF CANCER CENTER PHARMACY INC EXCELLENCE PENNSYLVANIA

Buy your ticket now at Richard Eric Mitchell Michael Peter Carolyn Sarah Julie Venners David A H Chad Hockett Lai Martin Murray Shaw Jones Nia Christensen Whiteman Clark www.terrapinn.com/ Chief Medical Oficer Senior Vice President, Head Director, Predictive Director of Government Senior Director, Medical Director of Senior Director Head of Global Patient Advocacy Global Clinical President attendprecision AFFYMETRIX of Pharmacogenomics & Medicine, Oncology Business Development Affairs Regulatory Policy Clinical Sciences for Gene Therapy Development Lead PRECISION FOR Companion Diagnostics REGENERON SOUTHERN FERRING BIOGEN ALEXION GLAXOSMITHKLINE SHIRE MEDICINE TAKEDA RESEARCH INSTITUTE PHARMACEUTICALS PHARMACEUTICALS INC.

MED001654 FEATURED KEYNOTE MAKING “TO ME, [THE POWER OF] GENETICS AND GENOMICS TO HELP US PRECISION UNDERSTAND THE MOLECULAR BASIS OF DISEASE IS CRUCIAL MEDICINE A FOR BEING ABLE TO TARGET THERAPIES BETTER THAN WE REALITY IN OUR COULD BEFORE.” LIFETIME – THE FDA’S PLANS TO “BUT IT GOES BEYOND GENETICS. IF YOU LOOK AT THE PRECISION SUPPORT THE MEDICINE INITIATIVE, THE EMERGING USE OF WEARABLE NEW PARADIGM TECHNOLOGY AND SOCIAL MEDIA ALLOWS US TO UNDERSTAND Dr. Robert Califf THINGS LIKE PATIENT PREFERENCES AND CONTINUOUSLY FDA Commissioner RECORD DATA THAT WE COULDN’T MONITOR BEFORE.”

MED001655 DAY 1 14th November, 2016 1/4 DAY 1 14th November, 2016 2/4

9:00 PLENARY KEYNOTE: Precision medicine & healthcare delivery: Making precision medicine a BIOPROCESSING AND APPLYING GENOME CLINICAL IMPACT OF NEWBORN AND reality in our lifetime through technology MANUFACTURING ENGINEERING GENOMICS PRENATAL DX • Unparalleled holistic and comprehensive approach to analyzing a patient’s genomic and proteomic make-up 12:00 Cell standardisation CRISPR gene editing Practicing better clinical Next generation • Precision treatment and clinical trials strategies to ensure technology for target trials to achieve better counselling for prenatal consistency in precision development in Oncology medicine: targeting the precision medicine • Importance of connectivity at hospitals and medical centers in pushing precision medicine forward medicine development Jason Moffat, Professor, 55% of patients with Meghan Carey, Executive Gary Palmer, Chief Medical Oficer, NantHealth and delivery University of Toronto actionable variables Director, National Society of David Brindley, Director, pointing to a speciic trial Genetic Counselors 9:20 KEYNOTE PANEL: Putting a price on precision medicine: Is the dream of the Precision Medicine CASMI (UK) instead of a drug Initiative a inancially feasible inale to the drug pricing wars? Mark Gardner, Chief Executive Oficer, OmniSeq • Is linking drug pricing to performance a realistic solution to the ballooning costs of the interim personalized/genetic medicine approach of using post-prescription pharmacogenomics to determine individual drug eficacy? 12:20 Networking Lunch • Are payers prepared for a new reimbursement infrastructure, taking genomic diagnostics into account as we drive more towards a pre-emptive model? PLENARY ROUNDTABLES • Are current drug distribution models eficient and appropriate for future precision therapies, where eficacy and dosing will be pre-determined? 2:00 Working with patient organizations to develop cohorts and stratify patients MODERATOR: Amupam B. Jena, Faculty Research Fellow, National Bureau of Economic Research Andres Hourtado-Lorenzo, Director of Translational Medicine, Crohn’s and Colitis Foundation Ed Pezalla, Vice President, National Medical Director for Pharmaceutical Policy and Strategy, Aetna 1 Jak Knowles, Managing Director, Vice President Medical and Scientiic Affairs, CureDuchenne Ventures David T. Ledbetter, Chief Scientiic Oficer, Geisinger Health System Mark Rubin, Director, Englander Institute for Precision Medicine, Weill Cornell Medical Center Barbara Conley, Associate Director, National Cancer Institute, NIH Steve Rosen, Senior Director, Diagnostic Strategy, Novartis

10:00 Networking Coffee Break and speed networking Prenatal diagnostics: capitalizing on whole genome sequencing rare disorders Fred Lorey, State Chief of Genetic Disease Screening Program (retired), Voting Member of Secretary’s Advisory Committee on 2 Heritable Disorders, State of California PERSONALIZED CELL THERAPY GENE THERAPY GENOMICS Fred Miendl, Sales Director North America, Perkin Elmer HEALTHCARE David Whiteman, Global Clinical Development Lead, Shire

11:00 Cell therapy and Gene therapy and Genomics as the 3D printing as an Genetic counselling: The expanding role in translational medicine precision medicine: an precision medicine: an enabling force in enabling technology in Erica Ramos, Director-at-Large, National Society of Genetic Counselors overview overview precision medicine non-genomic precision Jen Hoskovec, Former President, National Society of Genetic Counselors Robert Hariri, Founder Michael Linden, Head of Brad Perkins, Chief Medical medicine: what will it take 3 Katie Rock, Genetic Counselor, NIH and CEO, Celgene Cellular Gene Therapy, Pizer Oficer, Human Longevity Inc to get there? Leila Jamal, Genetic Counselor, Baylor College of Medicine Therapeutics Jonathan Morris, Assistant Toni Pollin, Primary Investigator, University of Maryland IGNITE Study Professor of Neurology, Mayo Emily Edelman, Genetic Counselor, Jackson Labs Clinic Informatics brain drain: maintaining the right talent at your irm for an evolving precision medicine industry BIOPROCESSING AND APPLYING GENOME CLINICAL IMPACT OF NEWBORN AND 4 Mark Gardner, Chief Executive Oficer, OmniSeq MANUFACTURING ENGINEERING GENOMICS PRENATAL DX Robin Toft, President and CEO, Toft Group

11:20 Methods and TALEN Gene editing Outcome prediction Rare disease diagnostics Cell therapies: how to cope with an unsuccessful clinical launch technologies for enabling “off the shelf” using a blood test – a in newborns using whole 5 analytical development: CAR-T pulmonary ibrosis case genome sequencing Kristin Comella, Chief Scientiic Oficer, US Stem Cell Inc. cell counting Andre Choulika, Chairman study Benjamin Solomon, Carl Simon, Biologist, and CEO, Cellectis Naftali Kaminsky, Professor Chief, Division of Medical From fastq to patient treatment guidance BioSystems and Biomaterials, of Medicine, The Yale School Genomics, Inova Healthcare Ying Chen, Senior Bioinformatics Specialist in Oncology and Precision Medicine, Cancer Institute of New Jersey National Institutes of of Medicine Translational Medicine Greg Volker, Head of Oncology Bioinformatics, GSK 6 Standards and Technology Institute Amit Aggarwal, Head of Bioinformatics, Eli Lilly Yong Yue, Head of Computational Biology, Boehringer Ingelheim 11:40 Sourcing and Non-viral gene delivery Personalized to Bing Yuan, Executive Director and Global Lead, Clinical Stage Oncology Business Development and Licensing, Merck qualiication for precision as an alternative in the precision – moving cell therapies precision medicine from anonymized data NGS formats: their strengths and weaknesses as clinical tools Elizabeth Read, Chief paradigm to actionable individual 7 David Smith, Professor of Laboratory Medicine and Pathology Chairman of the Technology Assessment Group Center for Scientiic Oficer, Medeor Rahul Aras, CEO, Juventas results for implementing Individualized Medicine, Mayo Clinic Therapeutics pharmacogenomics markers in the clinic Murray Brilliant, Director, Driving forward new pricing structures for precision medicine Center for Human Genetics, 8 Omar Ali, Member of Adoption & Impact Programme Reference Panel, NICE (UK) Marshield Clinic David Brindley, Director, CASMI (UK)

MED001656 DAY 1 14th November, 2016 3/4 DAY 1 14th November, 2016 4/4

ALTERNATIVE GENE COMPANION 4:50 Combination therapy: Cell therapy as one piece in the Landing among the stars: how “shooting for the COMMERCIALIZATION METABOLOMICS THERAPIES DIAGNOSTICS puzzle to achieving precision medicine moon” in translating genomic information to actionable Hans Klingemann, Chief Scientiic Oficer, NantKwest results can end up providing valuable information – 2:40 even when you miss Fund Raising to FCX-007 as a precision Driving precision Multiple in-vitro Kenna Mills Shaw, Executive Director, Personalized Medicine Partnering to Adoption gene therapy for medicine forward diagnostics for one Institute, MD Anderson Cancer Center and Commercial Recessive Dystrophic through a joint biomarker: solving the Success Epidermolysis Bullosa metabolomics, genomics problem 5:005:10 Enhancing autologous cell therapies for the precision era NCI-MATCH program: updates on the nation-wide Steven Deitcher, Co-Founder John Maslowski, SVP, and microbiome analysis Rao Mulpuri, Chief Operating Mike West, CEO, BioTime trials and planning for the future and Board Member, Medeor Scientiic Affairs, Fibrocell approach Oficer, Provista Diagnostics Barbara Conley, Associate Director, National Cancer Institute, Therapeutics Sciences Lavinia Ionita, Chief Executive NIH Oficer, Omixy 5:30 3:00 The promise and realities Putting the patient irst Using metabolomics as a Uniication of companion End of Conference and Networking Cocktails of regenerative medicines through AI analytics in irst-line phenotyping tool diagnostic development Marcie Glicksman, Chief combination therapy Clary Clish, Director of the for speedier research Scientiic Oficer, ORIG3N development Metabolomics Platform, Broad Prasun Mishra, Precision Niven Narain, CEO, Berg Institute, MIT Medicine Lead, Genetech LLC Roche

3:20 Understanding the true Novel gene expression Patient selection Using multiple sources value of cell therapy: how technologies as an approaches and of high-dimensional allogeneic therapies will alternative gene therapy actionable data using genomic data to build drive the industry forward driver in precision complex data from diagnostic algorithms in a precision medicine medicine biomarker samples Anne-Marie Martin, SVP, world Francois Lebel, Chief Glenn Barnes, Associate Head of Precision Medicine, Sicco Popma, Scientiic Medical Oficer, Ziopharm Director, Translational Novartis Director, Gene Modiied Cell Medicine Operations, Takeda Therapy Leader, Janssen Pharmaceuticals

3:40 Networking Coffee Break

NOVEL TOOLS AND TECHNOLOGIES FOR DATA COMPANION REGENERATIVE THERAPIES ANALYSIS DIAGNOSTICS

4:10 Extracellular Drug Conjugates: a small molecule/ Empowering limited data The “strategic healthcare antibody combination approach as an alternative to across communities: system”: evaluating ADC technology in immunotherapy lessons from pediatric strategies for companion James Prudent, Chief Executive Oficer, Centrose medicine diagnostics in clinical Therapeutics Adam Resnick, Head of development Precision Medicine Group, Amir Handzel, Statistical Children’s Hospital of Science Director, Philadelphia AstraZeneca

4:30 Cell and gene therapy delivery – utilizing iPSC-derived Cracking the code of PHYSICIAN-PHARMA tissues as a simple solution for precision therapies copy number variants: INTERACTION Eric David, Chief Strategy Oficer and EVP of Preclinical generating a uniform set Development, Organovo of exome data through ancestry decomposition Physician-Driven Jeffrey Reid, Executive assay design: decision Director, Head of Genome support tools informed Informatics, Regeneron by leading practitioners of genetically guided patient care in real-world settings Kristen Comella, Chief Scientiic Oficer, US Stem Cell Clinic

Register now at www.terrapinn.com/attendprecision Register now at www.terrapinn.com/attendprecision MED001657 DAY 2 15th November, 2016 1/3 DAY 2 15th November, 2016 2/3

9:00 PLENARY KEYNOTE: Precision medicine & healthcare delivery: Making precision medicine a 2:00 Novel Epigenetic therapies PANEL: Rare Diseases Contributing genetic results reality in our lifetime through technology targeting angiogenesis, Is precision medicine the end to public databases for use in • Multi-sector partnerships to foster growth and innovation modifying metastasis by of orphan drugs? How the clinical development Vanessa Rangel-Miller, VP Genetic • Building and regulating tools for detecting patients’ genomic patterns relevant to disease regulating epigenome orphan drug industry has Kurtis Bachman, Head of Computational Services, Patient Crossroads development, progression, and treatment impacted precision medicine and Systems Biology, Janssen MODERATOR: • Crowd-sourced, cloud-based informatics platform designed to advance the science and Stephen Groft, Former Director, Ofice of collaboration Rare Disease Research, NIH Dr. Robert Califf, Commissioner, US FDA PANELISTS: Yaffa Rubenstein, Rare Disease Patient PLENARY KEYNOTE: Precision approvals: How biomarkers are blurring the lines in clinical Registries Expert, NIH trials and accelerating commercial launch Ed Pezalla, Vice President, National Medical Director for Pharmaceutical Policy • Biomarker driven patient stratification is bending the curve away from traditional Phase I, II and III and Strategy, Aetna studies– leading to accelerated approvals based on fewer patients, earlier Elizabeth Ottinger, Project Manager, • Understanding the scientific, logistical and practical changes in design and execution of clinical Rare Diseases, NIH trials for precision medicine Abdel Halim, Vice President, Translational Medicine, Biomarkers & • Demonstrating value to payers and hospital systems and the healthcare system implications of Diagnostics, Celldex Therapeutics every disease becoming a rare disease Chad Clark, President, Precision for Medicine HEALTHCARE CIO-FOCUSED ROUNDTABLES 9:30 Networking Coffee Break and speed networking 2:30 Genomic proiling moving into routine clinical care PERSONALIZED CELL + GENE THERAPY GENOMICS Jeremy Segal, Director, Division of Genomic Pathology, University of Chicago HEALTHCARE David Roth, Director, Penn Center for Precision Medicine, University of Pennsylvania 1 Kenna Mills Shaw, Executive Director, Personalized Medicine Institute, MD Anderson Cancer Center Steven Averbuch, Vice President, Translational Clinical Development & Pharmacodiagnostics, Bristol-Myers Squibb GENOMIC IMPACT ON DISEASE ADVANCES IN HEALTHCARE IT Abdel Halim, Vice President, Translational Medicine, Biomarkers & Diagnostics, Celldex Therapeutics IMMUNOTHERAPY RESEARCH FOR PRECISION MEDICINE

2 Precision vs personalized: what's the difference in a clinical setting? 10:30 Engineered autologous T-Cell Routine genomic research Partnerships for co-development Peggy L. Peissig, Chief Research Informatics Oficer, Marshield Clinic Therapy during drug development of an HIT infrastructure: Charles Nicolette, CEO, Argos Karina Bienfait, Global Head, Genomic advantages over in-house 3 Integrated ambulatory and hospital EHR systems: Small health system perspective Therapeutics Strategy, Principal Scientist, Merck development Daryl Kallevig, Chief Information Oficer, Riverwood Healthcare Shivdev Rao, Director, Clinical Innovation Strategy, UPMC DRUG DEVELOPMENT FOCUSED ROUNDTABLES 11:00 Immunosequencing for a new Integrating natural histories Enabling the integration of Robust HIT infrastructure: Challenges in development class of immune diagnostics into EHRs for more accurate drug-related genetic indings into 4 Anup Kaneri, Portfolio Strategist, UPMC Enterprise Jianda Yuan, Director, Translational diagnosis clinical practice Immuno-Oncology Research, Merck David Pearce, President of Research, Peggy L. Peissig, Ph.D., MBA, Chief Payer’s perspective: realities of reimbursement and precision medicine Sanford Health Research Informatics Oficer, Marshield 5 Ed Pezalla, Vice President, National Medical Director for Pharmaceutical Policy and Strategy, Aetna Clinic Sam Johnson, Director, Health Policy and Interprofessional Affairs, American College of Clinical Pharmacy (formerly of Kaiser Permanente) 11:30 Using small molecule Cancer precision medicine: Wellness as the cornerstone to epigenetic agents to induce precision medicine: wearable 6 Cost of goods sold in Cell and Gene therapies: overcoming low ROI through rare disease designation? What we know, what we think Elizabeth Ottinger, Project Manager, Rare Diseases, NIH biomimicry for immunothery we know, and what we really devices and data mining for and trigger tumor response need to know early disease detection Stephen Baylin, Deputy Director, The Richard Buller, Vice President, Oncology Nathan Price, Professor and Associate Supply chain management for cell and gene therapies Ena Bromley, President and CSO, BioStat Solutions Sidney Kimmel Comprehensive Cancer Clinical Development, Pizer Director, Institute for Systems Biology Center, Johns Hopkins 7 Matt Nelson, Director, Genetics, GSK Michael Murray, Director, Government Business Development, Southern Research Institute Michelle Penny, Director Computational Biology and Genomics, Biogen EPIGENETICS FOR CELL CHANGING FACE OF KNOWN HEALTH DATA & ETHICS THERAPY + IMMUNOTHERAPY DISEASES Investing in precision medicine: Understanding what the capital markets are looking for in a disease-based 1:30 investment world Studying environmental Pulmonary Disease How can patient organizations Jak Knowles, Managing Director, Vice President Medical and Scientiic Affairs, CureDuchenne Ventures contributions to human disease Precision medicine outside generate valuable data – and 8 Joshua Rsenick, Partner Biotechnology, SV Life Sciences via epigenetics & genomics of oncology: developing use it to help push for precision Christina Bardon, Managing Director, MPM Oncology Impact Fund Jan Scicinski, SVP and Chief Scientiic the infrastructure for patient cures for their disease? Christopher De Souza, Director, Broadview Ventures Jit Patel, Director, JDRF Oficer, EpicentRx history-based studies Katherine Leon, CEO, SCAD Alliance Claudia Rizzini, Managing Director, Precision Medicine, Brigham Women’s 3:30 Networking Coffee Break Hospital MED001658 DAY 2 15th November, 2016 3/3

4:10 PrecisionFDA SHOWCASE

This portion of the event is for companies who have been involved with the PrecisionFDA initiative to showcase their work to an industry audience through short, 10-minute segments, and highlight the indings of the PrecisionFDA competitions. Check back soon for more information!

5:10 End of Conference

FEATURED KEYNOTE

PRECISION MEDICINE & NOVEL HOLISTIC AND COMPREHENSIVE APPROACHES HEALTHCARE DELIVERY: TO ANALYZING A PATIENT’S GENOMIC AND PROTEOMIC MAKING PRECISION MAKE-UP ARE STRESSING THE IMPORTANCE OF MEDICINE AREALITY IN CONNECTIVITY AT HOSPITALS AND MEDICAL CENTERS OUR LIFETIME THROUGH IN PUSHING PRECISION MEDICINE FORWARD TECHNOLOGY Gary Palmer CMO NANTHEALTH Register now at www.terrapinn.com/attendprecision

MED001659 WHAT IS PRECISION MEDICINE?

Richard Buller Robert Hariri Rahul Aras Vice President and Head, Founder and CEO CEO ANSWER 1: A CATALYST FOR Clinical Development CELGENE CELLULAR JUVENTAS PFIZER ONCOLOGY THERAPEUTIC THERAPEUTICS

REGENERATIVE MEDICINE 2.0 TOPIC: Cancer precision medicine - What we know, TOPIC: Cell therapy and precision medicine: an TOPIC: Non-viral gene delivery as an alternative in what we think we know, and what we really need overview the precision medicine paradigm to know • How autologous cell therapies have laid the • Utilizing endogenous stem cell repair as a therapeutic A path to commercialization for Cell and Gene Therapies • Insights into patient selection strategies groundwork for precision medicine function • What is the testing implementation on the path to • Moving forward – working towards manufacturing to • Preventing inlammation and immunogenicity in a viral approval and the highway post-approval? scale and standardization vector • Best practices when partnering with health authorities • What the coming new drug pricing infrastructure • Enabling treatment in cardiac, organ and skin tissue • What are some of the issues for resolution in the cost means for cell therapy producers damage through low-intrusion therapies COMING FROM PHARMA & BIOTECH… model?

Eric David Michael Linden Andre Choulika Karina Bienfait Mike West Jeffrey Reid SVP Research and Development Head of Gene Therapy Chairman and CEO Global Head, Genomic Strategy, CEO Executive Director, Head ORGANOVO PFIZER CELLECTIS Principal Scientist BIOTIME INC. of Genome Informatics MERCK and Board Member REGENERON LIFEMAP SOLUTIONS TOPIC: Cell and gene therapy delivery – utilizing TOPIC: Gene therapy and precision medicine: an TOPIC: TALEN Gene editing enabling “off the shelf” TOPIC: Routine genomic research during drug TOPIC: Enhancing autologous cell therapies for the TOPIC: Cracking the code of copy number variants: iPSC-derived tissues as a simple solution for overview CAR-T development precision era generating a uniform set of exome data through precision therapies • Combining genomic disease knowledge and viral • Creating allogeneic CAR T-cells derived from healthy • Informing the development strategy using genomic • Driving commercial viability of autologous cell therapy ancestry decomposition • Tech transfer in stem cells: achieving clinical-dose vectors for novel therapies donors rather than the patients themselves data acquired from clinical trials through new matrices • Growing a uniform set of exome data through scale eficiently • AAV Gene therapy for hemophilia and blood disorders • Enabling development of product candidates that • Development of set goals and distinct policy in relation • Immunotherapy and combination therapies through determining associations in genotypes and phenotypes • Helping to address the manufacturing gap using early • The issue of cost and ROI – can the precision feature additional safety and eficacy attributes, to obtaining genomic information during PhI/II/III clinical accelerated FDA tracks • 85,000 exomes from sequence and lab data to build a preventative planning medicine push overcome the pitfalls of Gene Therapy including control properties designed to prevent them trials. genomic and metabolomics infrastructure • Understanding the regulatory framework for transitional commercialization? from attacking healthy tissues • Working with patient groups and using natural history • Identifying co-normalization through k means clustering technologies • Enabling cells to tolerate standard oncology research as a catalyst for routine testing treatments, and equipping them to resist mechanisms that inhibit immune system activity

Prasun Mishra Carl Simon Amir Handzel Kurtis Bachman John Maslowski Elizabeth Read Precision Medicine Lead NATIONAL INSTITUTES Head of Statistics Head of Computational and SVP, Scientiic Affairs Chief Scientiic Oficer GENENTECH ROCHE OF STANDARDS AND ASTRAZENECA Systems Biology FIBROCELL SCIENCES MEDEOR TECHNOLOGY JANSSEN

TOPIC: Uniication of companion diagnostic TOPIC: Methods and technologies for analytical TOPIC: The “strategic healthcare system”: TOPIC: Novel Epigenetic therapies targeting TOPIC: FCX-007 as a precision gene therapy for TOPIC: Sourcing and qualiication for precision cell development for speedier research development: cell counting evaluating strategies for companion diagnostics in angiogenesis, modifying metastasis by regulating Recessive Dystrophic Epidermolysis Bullosa therapies • Identifying drugs through HTS and lead identiication • Inability to reliably characterize cells as possibly clinical development epigenome • Addressing the underlying cause of RDEB by providing • Cellular starting materials and approaches for their using sequencing technologies industry’s greatest challenge • Mass spectrometry for biomarker discovery in • Epigenetics can answer the question of stem cell functional Type VII collagen to affected areas sourcing • Bridging diagnostic and therapeutic development • Issue addressed with systematic approaches for metabolomics – driving from translational to clinical eficacy and lineage changes • Fibroblast genetically modiied to express functional • Speciic challenges in starting material qualiication for during preclinical and clinical research assessing sources of uncertainty and improving developments • Epigenetic memory and retained programming as both COL7 patient-speciic products • Biomarker driven therapeutics – implementing conidence in key measurements • Discussing the “cutoff” of companion diagnostics and a marker and tool • Dermal ibroblasts are collected from the patient, • Off-the-shelf allogeneic products (multipotent or metabolomics in the drug pipeline for disease • Applying these strategies will help to establish regulatory incentives for the combined approach • Cancer stem cell hypothesis and epigenetic autologous therapy pluripotent stem cell-derived) for patient unspeciic identiication. qualiied assays for cell characterization which help • Developing a more strategic approach to clinical trial biomarkers in oncology treatments streamline regulatory approval and enable more design using metabolomics and statistics eficient development

Hans Klingeman Lavinia Ionita Steven Deitcher Niven Narain Jan Scicinski James Prudent CSO CEO and Founder BIOTECHNOLOGY CEO SVP and Chief President and CEO NANTKWEST OMIXY INVESTOR BERG LLC Scientiic Oficer CENTROSE EPICENTRX THERAPEUTICS

TOPIC: Combination therapy: Cell therapy as one TOPIC: Driving precision medicine forward through TOPIC: Fund Raising to Partnering to Adoption and TOPIC: Putting the patient irst through AI analytics TOPIC: Restoring eficacy of immunotherapy TOPIC: Extracellular Drug Conjugates: a small piece in the puzzle to achieving precision medicine a joint metabolomics, genomics and microbiome Commercial Success in combination therapy development treatments in oncology with radical oxygen and molecule/antibody combination approach as an • NK Cells as a component in a larger precision analysis approach • Driving commercial viability of autologous cell therapy • Using deep learning AI to understand patient needs at nitrogen based epigenetic drugs alternative to ADC technology in immunotherapy medicine therapy paradigm • Cross-analyzing multi-omics testing results, to make through new matrices a genomic and phenotypical level • Acquired resistance to chemotherapies results in early • Using well-conserved sodium protein pump • Vaccine and antibody components, and how they sure that all of the pieces are being seen • Immunotherapy and combination therapies through • Developing a global architecture to connect the dots disease progression and lower survival overexpression in cancer cells to our advantage interplay with cell therapy • Showing the biochemical changes that occur in accelerated FDA tracks and give us a picture of the activity of the biological • ROS-mediated episensitizers hold the promise of not • Targeting inhibitor of pump in B-cell lymphomas • Using sequencing and proteomics to progress the organism during metabolic processes, to further systems within the patient only restoring eficacy to immunotherapy treatments • Unique approaches to using the small molecule immunotherapy and checkpoint inhibitors understand physiology • Real time analytic solutions for predicting the impact • RRx-001-mediated resensitization in the context of an approach to drive precision medicine forward • Understanding ageing physiology to extend lifespan of treatment plans at the individual level to optimize ongoing Phase 2 clinical trial in metastatic colorectal population health strategies cancer MED001660 Clary Clish Andres Stephen Baylin WHAT IS PRECISION MEDICINE? Head of Genomics Hourtado-Lorenzo Deputy Director BROAD INSTITUTE Director of Translational Medicine THE SIDNEY KIMMEL CROHN’S AND COLITIS COMPREHENSIVE FOUNDATION CANCER CENTER, JOHNS HOPKINS

TOPIC: Using metabolomics as a irst-line ROUNDTABLE DISCUSSION: Working with patient TOPIC: Using small molecule epigenetic agents to phenotyping tool organizations to develop cohorts and stratify induce biomimicry for immunotherapy and trigger ANSWER 2: PUTTING THE PATIENT FIRST • Population-based studies for metabolomics signs of patients tumor response disease • Clinical data - triggering interferon response and • Analyzing population-based cohorts for large-scale immune response to begin tumor destruction Pharma and the top provider systems in the world will studies and developing model systems • DNA demethylation agents and acetylation inhibitors • Monitoring biomarkers that can change the course of as genome-altering agents for oncology be showcasing how they are driving forward expanding individual treatments • Understanding the abnormalities of chromatin and methylation assembly that may account for the appearance of epigenetic abnormalities during tumor development, and how they mediate the transcriptional treatment methods and growing their HIT infrastructure to repression match the growing genomics data boom. Claudia Rizzini Benjamin Solomon Katherine Leon Managing Director, Chief, Division of Medical Genomics President Precision Medicine INOVA HEALTHCARE SCAD ALLIANCE BRIGHAM WOMEN’S TRANSLATIONAL HOSPITAL MEDICINE INSTITUTE COMING FROM HEALTHCARE TOPIC: Pulmonary Disease – Precision medicine TOPIC: Rare disease diagnostics in newborns using TOPIC: How can patient organizations generate outside of oncology: developing the infrastructure whole genome sequencing valuable data – and use it to help push for precision for patient history-based studies • Coupling diagnostics with genetic counseling cures for their disease? • Genomic sequencing is becoming faster, cheaper, • Tests to evaluate the health of both mother and baby • Supporting patients and caregivers while educating PROVIDERS & PATIENT more accurate, and more available in a variety of even during the irst trimester clinicians on the role that patient groups can undertake contexts. • High resolution 4-D transvaginal probe that helps • Identiication of genetic risk factors for a rare, • One major use-case for genomic sequencing is the physicians view and detect fetal abnormalities earlier seemingly random complication from disease and ability to diagnose rare diseases (which are common than ever surgery ORGANIZATIONS… in aggregate), short-circuiting long, dificult, and • Developing a registry for SCAD patients and family expensive “diagnostic odysseys.” members, including genomic data • In addition to providing answers, diagnosis through genomics may also impact medical care for patients both immediately and in the longer-term.

Jonathan Morris Adam Resnick Murray Brilliant Jeremy Segal Brad Perkins Assistant Professo Head of Precision Director Director, Division Chief Medical Oficer Patricia Weltin of Neurology Medicine Group MARSHFIELD CLINIC of Genomic Pathology HUMAN LONGEVITY INC. CEO MAYO CLINIC CHILDREN’S HOSPITAL RESEARCH FOUNDATION UNIVERSITY OF CHICAGO RARE DISEASE UNITED OF PHILADELPHIA FOUNDATION

TOPIC: 3D printing as an enabling technology in TOPIC: Empowering limited data across TOPIC: Personalized to precision – moving from ROUNDTABLE DISCUSSION: Genomic proiling moving TOPIC: Genomics as the enabling force in precision TOPIC: Putting the patient irst – the vision of non-genomic precision medicine: what will it take communities: lessons from pediatric medicine anonymized data to actionable individual results into routine clinical care. medicine – attaching meaning to the sequence Precision Medicine from the Patient Perspective to get there? • Building an infrastructure around specimens and for implementing pharmacogenomics markers in • How machine learning capabilities are transforming the • Understanding the driving forces behind rare disease • Perfecting imaging – how we can develop protocols special patient communities the clinic precision medicine landscape research as the precursor behind precision medicine for 3D printing and enhance segmentation tools • Integration between stakeholders in consumer and • Identifying “actionable genes” to reduce contra- • Predicting photograph-quality images from the • Emphasizing the role of patient groups and natural towards patient speciic tumors and wounds commercial space indicated drug assignments genome histories as a catalyst for precision therapies • How to improve software through intrinsic anatomic • Standardization of SOPs and organization of data • Placing pharmacogenomics markers within EHRs to • Attaching real meaning to the genome sequence • What is the future for rare diseases, and rare disease knowledge availability prevent interference with the clinical worklow through unique bioinformatics platforms integrating patient groups? How will they play into the new • Faster, more reliable 3D printers using better materials • Reducing the hindrance to preventative care of pharma, healthcare and reimbursement paradigm? as a catalyst for precision healthcare patients changing healthcare providers using HER interoperability

David Pearce Nathan Price Naftali Kaminski Daryl Kallevig Rasu Shretha Neal Ganguly President of Research Professor and Associate Director BOEHRINGER INGELHEIM Chief Information Oficer Chief Information Oficer Chief Information Oficer SANFORD HEALTH INSTITUTE FOR SYSTEMS PHARMACEUTICALS, INC. RIVERWOOD UPMC JFK HEALTH SYSTEM BIOLOGY Professor of Medicine HEALTHCARE YALE UNIVERSITY SCHOOL OF MEDICINE

TOPIC: Integrating natural histories into EHRs for TOPIC: Wellness as the cornerstone to precision TOPIC: Outcome prediction using a blood test – a ROUNDTABLE HOST: Integrated ambulatory and TOPIC: Partnerships for co-development of an TOPIC: Investing wisely in the right tools to entice more accurate diagnosis medicine: wearable devices and data mining for pulmonary ibrosis case study hospital EHR systems: Small health system HIT infrastructure: advantages over in-house patients to engage with healthcare providers • Implementation of newborn screening at the state level early disease detection • Solving the issue of early transplantation through perspective development to develop natural histories • Understanding the interface between genome utilization of modeling effects on referrals. • Importance of interoperability for EHR usage in sequencing and wearable devices for individual patient • Distinguishing patient drug response through diagnosis wellness genotyping and metabolic identiication • Impact of natural history screenings for Batten disease • Identiication of actionable genes through data mining • Precision medicine isn’t just genetics - Identifying through gene therapy clinical studies for early diseases innate immunity markers for biomarker development • Using the patient-physician relationship as a vehicle for and target discovery delivery of wellness information MED001661 PARTICIPATING ORGANIZATIONS REASONS TO ATTEND

1 Hear from FDA Commissioner Robert Califf in a historical keynote on Precision Medicine and the FDA’s future plans to help drive the industry.

2 Join the most comprehensive agenda on commercial and scientiic challenges in Cell and Gene Therapies, as outlined from pharmaceutical giants like Celgene and Merck, as we drive next-generation therapeutics towards the new precision paradigm.

3 See brand new prenatal and newborn diagnostics technology being implemented in healthcare clinics, such as Inova Health Systems, and how early genomic testing will change how we do medicine.

4 Learn how pharma and healthcare providers are implementing technological advances into their clinical and research worklow from the CIOs of UPMC, Riverwood Healthcare and JFK Health System.

5 Where big pharma meets big healthcare: Be a part of the largest gathering in North America of pharma and providers towards moving precision medicine forward, bridging the gap between genomic information and R&D.

6 Witness groundbreaking high-level talks on the implementation of the PMI in the private sector, from speakers like Prasun Mishra, Precision Medicine Lead at Genentech Roche and Jeff Reid, Director of Genomics, Regeneron.

7 Sit down with Jeremy Segal, Director of Genomics at University of Chicago, as he discusses moving genomic proiling into routine clinical care.

8 Rare Diseases: See how the evolving precision medicine paradigm is changing the face of the Orphan Drug industry and rare diseases, from the perspective of payers, patients and pharma.

9 Putting the patient irst: A wide array of perspectives from patient groups actively compiling natural histories of their diseases, including the Crohn’s and Collitis Foundation, and the Rare Disease United Foundation.

10 Meet, discuss research, and do business with hundreds of other industry leaders using the Jublia Networking System, where you can search not just by name and title, but by the content of their work.

The earlier you book, the more you can save. For the latest price, visit www.terrapinn.com/attendprecision Don’t forget to enter special code BD2015 to claim the Early Bird discount.

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BRING YOUR TEAM With one day packed full of great content and networking opportunities, you can’t possibly cover it all alone! Bring your team and get an extra discount Call +1 212 379 6320 for more information

MED001663 Learn more about Qwoted

Event: BioData World West 2018 Events & Awards Events Event: BioData World West 2018

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Venue Event Date Tue Mar 13 EDT - Wed Mar 14 EDT (over 2 years ago)

Location Hotel Kabuki 1625 Post St, San Francisco, CA 94115, USA

Region Americas

Details Hotel Kabuki

Hotel Kabuki, 1625 Post St, San Francisco, We pride ourselves on being the event for thought-leaders in the life science industry to not only CA 94115, USA attend and learn, but also to collaborate and to continue pushing the limits of what is possible in AI, Big Data, Genomics, and Precision Medicine. 225 high-level attendees, 102 thought-leading executives, and 14 groundbreaking solution providers joined us in San Francisco this Past April. They included such heavyweights as Atul Butte and Jeff Dean as our leading keynotes. A few of the innovators that joined us to share their expertise and meet their future collaborators included: Google Brain, NASA, Merck, The FDA, UCSF, NIH, Takeda, GlaxoSmithKline, Amgen, Shire, the U.S. Department of Veteran Affairs and What is Qwoted? , many more. Qwoted is a free expert network: we help reporters connect with experts & we help those same experts build relationships with top reporters. Speakers

Learn more 2018 Speakers Fuad Abujarad Assistant Professor of Emergency Medicine, Yale School of Medicine Anaupam Agarwal Head, Global Drug Safety and Pharmacovigilance, Zogenix

Martin Akerman CTO & Co-Founder, Envisagenics Slava Akmaev Vice President and Chief Analytics Officer, BERG Health Ron Alfa Vice President of Discovery and Product, Recursion Pharmaceuticals Brandon Allgood Chief Technology Officer, Numerate Inc Justin Aronson Founder, VariantExplorer.org Sandy Aronson Executive Director of Information Technology, Partners HealthCare Personalized Medicine Paul Avillach Associate Professor, Center For Biomedical Informatics At Countway Harvard Medical School Nazneen Aziz Executive Director, Kaiser Permanente Research Bank Munther Baara Head, New Clinical Paradigm, Pfizer Elizabeth Baca Senior Health Advisor Governor's Office of Planning and Research, State of Califonia Governor's Office of Planning and Research Vikram Bajaj Co- Founder, Google Life Sciences, Former CSO, GRAIL, Managing Director,, Foresite Capital Management

Sergio Baranzini Professor, UCSF James Barbeau Director of Laboratory Medicine, Lifespan/Brown University Afshin Beheshti Bioinformatician and Data Analyst, NASA Genlab Adam Berger Senior Staff Fellow of Personalized Medicine, Food and Drug Administration Anna Berry Scientific Director of Personalized Medicine and Medical Director of Molecular Diagnostics, Swedish Cancer Institute Jeffrey Bhasin Director of Informatics, Zymo Research Govinda Bhisetti Principal Investigator, Biogen Chris Boone Vice President, Real World Data and Analytics, Pfizer

Westyn Branch-Elliman, Instructor in Medicine, Harvard Medical School Jim Broach Director of the Penn State Institute for Personalized Medicine, Penn State College of Medicine Catherine Brownstein Scientific Director, Manton Center for Orphan Disease Research Michael Burczynski Executive Director, Head Of Integrated Clinical Biomarker Technologies, Bristol-Myers Squibb Robert Burton Co-founder and President, Center for Genomic Interpretation Noel Burtt Associate Director of Operations, Diabetes Research, Broad Institute of MIT & Harvard Atul Butte Director of Institute Computational Health Sciences and Professor, Institute of Computational Health Sciences University of California San Francisco Thomas Cahill Healthcare and Technology Portfolio, Raptor Group Tom Chittenden Vice President, Statistical Sciences and Founding Director, Advanced Artificial Intelligence Research Laboratory, WuXi NextCODE

Thomas Clozel co-founder, OWKIN

Manuel Corpas Scientific Lead, Cambridge Precision Medicine Greg Corrado Principal Scientist, Director of Augmented Intelligence Research, Google Brain Team Co-founder, Google Brain/Verily

Sylvain Costes Lead scientist for GeneLab, NASA Genlab Verner De Biasi Head Emerging Platforms, GSK Aubrey de Grey Chief Science Officer and Co Founder, SENS Foundation Arnaud Desfeux CEO, OMICtools Megan Doerr Principal Scientist, Sage BioNetworks

Yizhen Dong Principal, 11.2 Capital

Ian Dunham Scientific Director, Open Targets, European Bioinformatics Institute James Dzierzanowski Executive Director, Kaiser Permanente Olivier Elemento Director, Laboratory of Cancer Systems Biology, Englander Institute for Precision Medicine Institute for Computational Biomed, Cornell University Keith Elliston CEO, tranSMART Foundation Sunil Erraballi Information Technology & Finance, Selten Pharma Inc Preston Estep Chief Scientific Officer, VeritasGenetics Mark Fingerhuth CEO and Founder, ProteinQure Inc Kay Firth-Butterfield, Head Artificial Intelligence and Machine Learning, World Economic Forum Kristen Fortney CEO, BioAge Labs Caroline Fox Vice President and Head of Genetics and Pharmacogenomics, MSD Edward Glanville Project Director, Terrapinn USA Adam Godzik Director and Professor, Bioinformatics and Structural Biology Program, Sanford-Burnham Medical Research Institute Peter Goodhand Executive Director, Global Alliance for Genomics & Health Dennis Grishin Co-Founder, Nebula Genomics Steve Gullans Managing Director, Excel Venture Management L.L.C.

Vibhor Gupta Entrepreneur In Residence, Nexgen Capital Ventures Janet Hall Director of Product and Sales, American Heart Association Jennifer Hall Chief, Institute for Precision Cardiovascular Medicine, American Heart Association Abraham Heifets Chief Executive Officer, Atomwise Inc Maria Helena van de Pol Global Area Head, Research IT, Roche Gerald Higgins Professor, University of Michigan Medical School Benson Hsu Physician, Sanford Health

Mark Hurle Director, Computational Biology, GlaxoSmithKline William (Whurley) Hurley Founder and CEO, Strangeworks Karen Hurst

Futurist & Lead Architect in Technology / R&D, Kaiser Permanente Kullo Iftikhar Professor, Mayo Clinic

Dawn Ireland President, CDH International

Olexandr Isayev Professor, University Of North Carolina Naveen Jain Founder & CEO, Viome Justin H Johnson Associate Director and Principal Translational Genomic Scientist, AstraZeneca Vipul Kashyap Chief Information Architect, Northwell Health Handol Kim Sr. Director, Quadrant Machine Learning BU, D-Wave Systems Andreas Kogelnik President and Founder, Open Medicine Institute Gautier Koscielny Scientific Leader, Glaxo Smith Kline Inc Jacqueline Law Vice President, Global Head, PHC Data Science, Personalized Healthcare, Product Development, Genentech Joseph Lehar Executive Director, Computational Biology, Merck Simon Lin Chief Research Information Officer, Nationwide Children's Hospital

Neel Madhukar Runway Startup Postdoc Forbes 30Under30 (Healthcare), Runway Startup Postdoc, Cornell University Neel S Madhukar, CEO, OneThree Biotech Lara Mangravite President, Sage BioNetworks Chris Mansi Viz.Ai Vikash Mansinghka Principal Investigator, Massachusetts Institute of Technology John Martinis Lead, Quantum Computing Group, Google Inc

Jeanette Mccarthy Adjunct Associate Professor, Duke University Sarah Meeder Research Compliance Specialist, Maimonides Medical Center Inc Helen Messier Chief Medical Officer, Viome Katie Miller Project Scientist, Nationwide Children's Hospital

David Milward CTO, Linguamatics Prasun Mishra Founder & CEO, Agility Pharmaceuticals

Pravin Mishra Founder and President, Oncotelligent, Director, Precision Genomics Core Laboratory & R&D, Intermountain Healthcare Alysson Muotri Director of Stem Cell Program, University of California Mark Musen Professor, Stanford University Philip Nelson Director, Software Engineering, Google Inc Matt Nelson Head, Genetics, GSK Kamal Obbad Project Lead, Nebula Genomics Gordon Okimoto Co-Director, Biostatistics and Informatics, University of Hawaii Cancer Center Hemai Parthasarathy Scientific Director, Thiel Foundation, Breakout Labs Benedict Paten Director of Data Management, Center for Biomolecular Science and Engineering Alexander Pickett COO, Mediqventures Amy Pienta Associate Research Scientist, ICPSR Mathew Pletcher Head of Rare Disease Discovery, Roche Linda Polfus Assistant Professor, University of Texas at Houston Ryan Poplin Machine learning technical lead, Google Inc Rudy Potenzone Vice President, tranSMART Foundation

Prad Prasoon Emerging Business & Technology Strategies Leader, American Heart Association Chris Probert

PhD Student / Startup Founder, Stanford University / Stealth genomic data startup Ronald Przygodzki Director, Genomic Medicine Implementation, U.S. Department of Veterans Affairs Armon Rahim Growth Lead, Nebula Genomics Gunaretnam Rajagopal Vice President, Computational Sciences, Janssen Research & Development

Amit Rastogi Senior Vice President for Strategy, Growth and Innovation, Inova Health System Chris Riley Research Manager- Institute for Precision Cardiovascular Medicine, American Heart Association Oliver Rocroi

Senior Director, State Government Relations, California Life Sciences Association - CLSA Adrien Rousset

Innovation Lead - Patient ID & Predictive Analytics, Amgen Kate Rubins NASA Astronaut (First person to sequence the genome in space), NASA Genlab Riccardo Sabatini Co-Founder, Chief Data Officer, Orionis Biosciences

Malaikannan Sankarasubbu VP of AI Research, Saama Nadeem Sarwar President AiM Institute, Eisai Stefan Scherer VP Head of Early Development Strategy, McKesson Specialty Health The U.S. Oncology Network Michael Sekora Reagan White House David Smith Professor of Laboratory Medicine And Pathology, Mayo Clinic Morten Sogaard Vice President and Head, Genome Sciences & Technologies, Worldwide R&D, Pfizer

Yasaman Soudagar CEO and Founder, Neuroscience Inc

Nicole St Jean, Director, Precision Medicine, AstraZeneca LP Luke Stewart Director of Product Management, Saama Andrew Stewart CEO and Founder, Autism Diagnostics Elia Stupka Senior Director, Dana Farber Cancer Institute Brandon Suh Chief Medical Officer, Lunit

Alexander Titus Biomedical Data Scientist, B.Next @ In-Q-Tel

Ali Torkamani Director of Genome Informatics, Drug Discovery, Integrative Structural & Computational Biology, The Scripps Translational Science Institute

Duygu Tosun-Turgut, Assistant Professor and Co-Director of the Center for Imaging of Neurodegenerative Diseases (CIND), San Francisco Veterans Affairs Medical Center Matthew Trunnell Vice President & CIO, Fred Hutchinson Cancer Research Center Eugene Tu Centre Director, NASA Genlab Marielle van de Pol Global Area Head, Research IT, Roche Julie Walters Founder, Raremark

Christina Waters President & Chief Executive Officer, R.A.R.E. Science

Patrick Wayte Vice President of Marketing and Health Education, American Heart Association John Weinstein Professor and Chairman for Department of Bioinformatics and Computational Biology, The University of Texas MD Anderson Cancer Center Marvin Weinstein Co-founder Quantum Insights, Quantum Insights Richard Wendell Founder & CEO, tellic Ashley Winslow Senior Director of Translational Research, University of Pennsylvania Ashkon Zariv Technical Lead, Mendel.ai Jean Claude Zenklusen, Director, The Cancer Genome Atlas, National Cancer Institute, National Institutes of Health Shanrong Zhao Director, Computational Biology and Bioinformatics,Worldwide R&D, Pfizer

Alex Zhavoronkov CEO, Insilico Medicine, Inc

Sponsors & Partners

2018 Sponsors

• Roche GOLD SPONSORS:

• Western Digital

• IBM Q

• Saama

• Viz.ai SILVER SPONSORS:

• Linguamatics

• Numerate

• Tellic

• WuXiNextCode BRONZE SPONSOR:

• OmicX SESSION FACILITATORS:

• D-Wave

• Quantum Insights ASSOCIATE SPONSOR:

• Lunit INNOVATION ZONE SPONSOR

• Insilico Medicine

• RowAnalitics

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Home Symposium Speakers Agenda Who's Who Registration Sponsors

2019 Linus-Pauling Speakers (to be announced) and our past Linus Pauling biotech pharma Symposium speakers (see jpg below)

Pre-registration is required but seminar fee is $0.

Themes: Biologics, Antibody Therapeutics and Immunotherapy What is hot in biotech pharma? Why mentors? Why science? Why open innovation and global collaboration? Themes - Career decisions: academia vs. biotech pharma for science discovery Yin & Yang: lessons learned from working in academia, biotech & pharma. If innovation is a seed, where is the soil? When will it rain?

2016 Nobel-Pauling Biologics/Antibody Symposium Agenda

www.nobel-pauling.org/BiotechAgenda.html 1/16 MED001665 9/11/2020 Nobel-Pauling Biotech Pharma Symposium - Biotech Agenda Agenda

2:00 pm Registration, networking and group photo with speakers

3:00-5:00 pm Welcome: Dr. Grace Wong, ActoKine Therapeutics (former Genentech) Legacy of Dr. Linus Pauling: Professor Scott Mohr, Professor of Chemistry at Boston University

Presentation: Biologics, Antibody Therapeutics and Immunotherapy Moderators: Dr. Ravi Kumar, CSO, Acceleron Pharma, Dr. Joseph Murphy, CRL & Dr. Jeff Boucher, UMass Medical School

Dr. Thomas Waldmann, MD, Head, Cytokine Immunology and Immunotherapy Section, National Cancer Institute (Keynote) Seminar title "IL-2 and IL-15 in the life and death of lymphocytes: Serendipity and a 60 year scientific odyssey"

Dr. Andreas Schaaf PhD, Greenovation Biotech, Germany Martine Darwish, MS, Senior Scientist, Protein Chemistry, Genentech Dr. James Ernst, PhD, Senior Scientist, gRED Research and Early Development, Genentech

Prof. Dr. Stefan Dübel PhD, Technische Universität Braunschweig, Germany (co-founder of YuMab GmbH) Seminar title: Human therapeutic antibodies and beyond + career advice for students, postdocs & scientists

Dr. Michael Blank PhD, CSO, AptaIT GmbH, Munich, Germany Shannon Chilewski, MS, Scientist, Bristol-Myers Squibb

5:00-6:00 pm Panel discussion: Why Biologocis, Antibody Therapeutics and Immunotherapy? Career decision: academia vs. industry (all speakers)

6:00-6:10 pm Dr. Grace Wong: New drug discovery: AK-2 for protection against infection by a broad spectrum of viruses Dim Sum and group photo with speakers, students, postdocs and scientists

7:00-7:40 pm Presentation: Biologics, Antibody Therapeutics and Immunotherapy Moderators: Dr. Ibraheem Badejo, Johnson & Johnson, Dr. Pallab Ghosh, Harvard Dr. Ward Yuhas, CRL & Dr. Qing Liao (Boston Paragon and Spire Metering Technology)

Dr. Andrew Bradbury, MD, PhD, Staff Scientist, Biosciences, Los Alamos National Laboratory (Keynote) Dr. Randall Brezski, PhD, Scientist, Antibody Engineering, Genentech Dr. Carolyn Cuff, PhD, Leader, Translational Research & Investigation Unit, AbbVie

7:40-9:30 pm Dr. Paul Carter, PhD, Senior Director and Staff Scientist, Antibody Engineering, Genentech (Keynote) Seminar title - Antibody Therapeutics: Past, Present and Future (+ career advice)

Jeffrey Siegel, MD, Senior Group Medical Director, Global Head, Genentech Seminar title: Biologics for Autoimmune diseases? + career advice for students, postdocs & scientists

Dr. Vibha Jawa, PhD, Principal Scientist, Clinical Immunology, Amgen Dr. Stefan Weigand PhD, Head of Large Molecule Research, Roche

Panel discussion: Why Biologocis, Antibody Therapeutics and Immunotherapy? Career decision: academia vs. industry (all speakers)

Closing remark: Dr. Grace Wong (former Genentech) ActoKine Therapeutics

9:30-10:00 pm Networking for skills & advice (sea of learning)

2015 Nobel-Pauling Biotech Pharma Christmas Symposium agenda and speakers

www.nobel-pauling.org/BiotechAgenda.html 2/16 MED001666 9/11/2020 Nobel-Pauling Biotech Pharma Symposium - Biotech Agenda

Agenda

2:00 pm Registration, networking with Dim Sum and group photo with speakers

3:30-4:30 pm Welcome: Dr. Grace Wong, ActoKine Therapeutics Legacy of Dr. Linus Pauling: Amadou Barry, South Africa, UMD

Presentation: the past, the present & the future & Why career in Science Moderators: Dr. Xin Tang, MIT & Dilip G. Gosar (former Lilly) Dr. Jiang He, MIT Dr. Christian Maass, MIT, from Germany Dr. Yogesh Dayma, MIT Dr. Ramchander Chepyala, MIT Dr. Tianmin Fu (Prof Hao Wu's lab), Harvard

4:30-5:00 pm Smart pitch Dr. Xin Tang (Harvard); Dilip Gosar (former Lilly); Dr. Rajiv Shrestha (Octet Research); Yogesh Reddy (Ascensus); Jessica Val (UMD); Alvin Lu (Harvard); Dr. Zhao Wang (MIT); Dr. Zhou Lin (MIT); Dr. Peng Du (Tufts); Weiming Sun (MIT Sloan); Dr. Jicong Cao (MIT); Dr. Jung Suh (MIT); Dr. Ahmed Fazly (MIT); Dr. Claudia Wehrspaun (MIT); Dr. Jun Yang (MIT); Dr. Ru Wang (MIT)

5:00-5:20 pm Dr. Grace: What are the most important criteria for success in scientific discovery? Getting a foot in the door & What is hot in Biotech Pharma?

5:30-6:30 pm Yin & Yang: lessons learned from working in academia, biotech & pharma. Moderators: Dr. Rajiv Shrestha (Octet Research Inc) & Yogesh Reddy, Ascensus (Investment) Dr. Yue Liu, Pfizer Dr. Robert Ng, BioMarin Pharmaceuticals Supriya Shekar, Medbiomarkers Xiaoyue Shi, Case Western Reserve University, Ohio (Why China Biotech Pharma?) www.nobel-pauling.org/BiotechAgenda.html 3/16 MED001667 9/11/2020 Nobel-Pauling Biotech Pharma Symposium - Biotech Agenda Prof DAI Huanqin is an associate professor in Prof Gil Alterovitz's lab (MIT/Harvard), as a visiting scholar from the Chinese Academy of Sciences (in Prof Lixin Zhang's lab), Beijing, China

6:30-7:00 pm Dr. Grace (Career decisions: academia vs. Industry (start up, biotech & pharma) Closing remark: Dr. Grace (founder of www.ActoKine.com; www.Nobel-Pauling.org and www.StudentVision.org)

7:00-8:00 pm Networking for skills & advice (sea of learning)

2014 Nobel-Pauling microRNA and mentor Symposium Agenda Boston (this location is shown only to confirmed attendees) 2014 Nobel Linus Pauling Mentoring & MicroRNA Symposium (March 17, 2014)

Agenda ?

3:00 pm Registration & Networking with substances

3:30 pm Welcome: Dr. Grace Wong, CEO, ActoKine Therapeutics Legacy of Dr. Linus Pauling: Marsha Paul, Linus Pauling Volunteer, volunteer for StudentVision.org Dr. Chung-Wei Lee, MIT & Dr. Weimin Guo, Harvard Bryan Germain, BiogenIdec, Career Keynote: Why career in Biotech? Dr. David Salzman, BiogenIdec, Educational Keynote: microRNA: past, present & future

4:30-5:00pm Speakers: Smart pitch from speakers from academia, biotech & pharma Dr. Chung-Wei Lee, MIT; Dr. Weimin Guo, Harvard; Dr. David Salzman, BiogenIdec; Dennis France, Advanced Cellular Dynamics Dr Grace Wong, ActoKine Therapeutics (1st seminar Title: AK-1 for cancer and AK-2 for bird flu prevention) 2nd seminar title: Advice from Nobel Laureates and experts: What are the most important criteria for success www.nobel-pauling.org/BiotechAgenda.html 4/16 MED001668 9/11/2020 Nobel-Pauling Biotech Pharma Symposium - Biotech Agenda in scientific discovery? and other speakers (students, post-docs & speakers from Academia, biotech & pharma). Prof Arthur Pardee, Harvard; Dr. James Falls, GSK; Dr. Xuemei Zhao, Merck; Dr. Rounak Nassirpour, Pfizer; Dr. Pavan Kumar, Eisai; Dr. Prasun Mishra; NIH; Prof Muller Fabbri; USC, LA: Prof Lorenzo Sempere, Van Andel Res. Inst; Prof Robert Lee, OSU, Ohio; Prof Taosheng Chen, stjude.org; Prof John Pezacki, Univ of Ottawa; Dr. Kathy Martin, KJMBiosystems; Becky Buzzeo, ThermoFisher Scientific; Dr. Jingfang Ju, Stony Brook Univ and more.

5:30 pm Speaker group photo with students & postdocs at 5:40pm and surprise pitch from Prof Pardee's four postdocs at 5:50pm Prof Charles Stiles, Harvard (surprise pitch for Prof Pardee) Keynote speakers: 6:00pm Prof Arthur Pardee, Harvard (Linus Pauling's student at Caltech) Title: microRNAs and therapy of cancer Prof Roy Glauber, Nobel Laureate, Harvard (story of Linus Pauling)

6:30-7:30 pm Speakers from Academia and biotech pharma Industry: microRNA seminars? Moderators: Dr. David Salzman, BiogenIdec & Alex Pauling, Nobel-Pauling Prof Arthur Pardee, Harvard; Dr. Bing Xia, GSK; Dr. Xuemei Zhao, Merck Dr. James Falls, GSK; Dr. Rounak Nassirpour, Pfizer; Dr. Pavan Kumar, Eisai; Dr. Prasun Mishra; NIH; Prof Muller Fabbri; USC, LA: Prof Lorenzo Sempere, Van Andel Res. Inst; Prof Robert Lee, OSU, Ohio; Prof Taosheng Chen, stjude.org; Prof John Pezacki, Univ of Ottawa and more.....

7:30 pm - 8:00 pm Biotech who's who (the joy of learning)

8pm-9pm Panel discussion: Why microRNA? Prof Arthur Pardee, Harvard; Dr. James Falls, GSK; Dr. Xuemei Zhao, Merck; Dr. Rounak Nassirpour, Pfizer; Dr. Pavan Kumar, Eisai; Dr. Prasun Mishra; NIH; Prof Muller Fabbri; USC, LA: Prof Lorenzo Sempere, Van Andel Res. Inst; Prof Robert Lee, OSU, Ohio; Prof Taosheng Chen, stjude.org; Prof John Pezacki, Univ of Ottawa; Dr. Kathy Martin, KJMBiosystems; Becky Buzzeo, ThermoFisher Scientific; Dr. Jingfang Ju, Stony Brook Univ and more....

9:00 pm Closing remark (Dr. Grace Wong, founder of Nobel-Pauling.org and StudentVision.org)

9:10-10pm Networking for skills & advice (sea of learning to be useful for the world)

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2013 Nobel-Pauling Biotech Pharma Symposium Agenda Boston (this location is shown only to confirmed attendees. Please call 617-566-0511 for the location) 2013 Christmas (11 am to 5 pm)

Agenda

11:00 am Registration & Bio who’s who (networking, 12 to 3pm)

12:00 pm Welcome: Dr. Grace Wong, ActoKine Therapeutics Legacy of Dr. Linus Pauling (Dr. Jeremy Kintigh, University of New Hampshire)

12:30 pm Keynote speakers (New drug discovery: the need for innovation & global collaboration) Dr. Michael Lam, Merck; Dr. Nanding Zhao, Eisai; Dr. Xiaoyu Tian, Bristol-Myers-Squibb; Dr. Jeff Ding, Sanofi; Dr. Bob Ward, AstraZeneca; Dr. Sherwin Shang, Abbvie; Dr. Yufei Xu, Novartis

Speakers from Academia and Industry for science innovation Moderators: Richard Shamon, Student Vision & Alex Pauling, Nobel-Pauling Sean Yang, genzyme; Yu Zhang, Pfizer; Dr. Emile Bellott, Howfond, China; Dr. CN Ramchand, CEO, Laila Pharma, India; Dr. Grace Wong, ActoKine Therapeutics; Dr. Min Wu, Harvard; Dr. Yansheng Hao, DFCI/Harvard; Dr. Haiyan Peng, BiogenIdec; Dr. Shenghong Yang, Harvard and Dr. Lichao Chen, Harvard; Dr. Leonid Gaidukov, MIT; Dr. Nikola Kojic, MIT; Prof Maolin Guo, UMass Dartmouth; Dr. Alper Kucukural, Umass Medical School; Dr. SHANKAR PRASAD DAS, Umass Medical School

2:00 pm Smart pitch – Bio Who's who (the joy of learning)

2:15 pm Short presentation: Why scientist? The past, the present & the future. Dr. Leonid Gaydukov, MIT; Dr. Shenghong Yang, Harvard; Dr. Aishuang Xiang, Novartis-MIT; Dr. Alper Kucukural, Umass Medical School; Amanuel Ghidey, University of New Hampshire; www.nobel-pauling.org/BiotechAgenda.html 6/16 MED001670 9/11/2020 Nobel-Pauling Biotech Pharma Symposium - Biotech Agenda Dr. Nikola Kojic, MIT & Dr. Zina Zhu, MIT; Dr. SHANKAR PRASAD DAS, Umass Medical School; Dr. Ahmed Bayoumi MD, Ibgen; Adil Malam, Harvard, UK

Smart pitch-Biotech who's who Dr. Ozlem YILDIRIM, Harvard; Dr. David Dai, Merck; Dr. Yuqi Qin, Dr. Haiyan Peng, BiogenIec Dr. Sha Mi, BiogenIdec; Dr. Liu shubai, Harvard and Hua Wang, Northwestern University, Chicago; Dr. Saibal Chakraborty, NIH; Dr. Ming BAI, Harvard; Dr. Helen Sadik, Johns Hopkins Medical School & Michel Lau, Johns Hopkins U; Dr. Cong Peng, Harvard; Dr. Chung-Jung Chiu, Tufts University; James Martin II, Princeton University; Dr. Shubai Liu, Harvard; Dr. Liling Zeng, BU; Shi Su in Dr. Tai Chen's lab, Boston University; Prof Smita Varia, Dr. Gunasekaran Singaravelu & Jonathon Brzezinski, Rutgers University; Dr. Minh Le, from Prof. Harvey Lodish's lab, Boston Children's Hospital; Dr. Gillian Browne, University of Vermont College of Medicine; Dr. Jiahai Shi, from Prof. Harvey Lodish's lab, Whitehead Institute for Biomedical Research; Dr. Xiaohua Huang, Harvard, Dr. Eva Maria Novoa Pardo, MIT and Dr. Yingying Huang, MGH/Harvard Dr. Nopporn Thangthaeng, USDA, Dr. Murat Keceli, MIT; Dr. Yan X Yu, Brandeis; Chip Allee, CEO CeuticalSoft

4:00 pm Networking for skills & advice (Sea of learning)

5:00 pm Closing remark (Dr. Grace Wong, ActoKine Therapeutics, former Genentech)

2012 Nobel-Pauling Biotech Pharma Dim Sum Symposium Agenda Boston; 2012 Christmas (11 am to 5 pm)

Dear Dr. Grace Wong, Many thanks for hosting an excellent Linus Pauling Biotech Symposium to mix scientists from academia with industry on Christmas holidays. It was a great opportunity for all of us to get to know each other for future collaboration.

Behzad Etemad, PhD, Postdoc, BU School of Medicine, Section of Infectious Diseases (Dec 27, 2012).

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Hi Grace: Thank you very much for inviting me to participate the symposium. You have done a wonderful job to organize the Linus Pauling Christmas Biotech Symposium. I am enjoying the meeting very much.

Wenhui Wang, MD, PhD, Professor, Department of Pharmacology, NYMC (Dec 27, 2012)

Agenda

11:00 am Registration & Biotech who’s who (networking, dim sum from 12 to 3pm)

12:00 pm Welcome: Dr. Grace Wong, ActoKine Therapeutics Legacy of Dr. Linus Pauling (Richard Shamon, Nobel-Pauling)

12:30 pm Keynote speakers (Drive the next wave to science innovation) Dr. Qiying Hu, Novartis; Dr. Lih Ling Lin, Pfizer Prof Wenhui Wang, New York Medical College; Prof Yan Zhu, Tianjin University of TCM (USA vs. China for new drug discovery)

Panel Discussion: Academia vs. Industry for science innovation Moderators: Dr. Jason Deng, MIT & Dr. Junjie Lu, Harvard Dr. Lih Ling Lin, Pfizer; Dr. Qiying Hu, Novartis; Steven Bodovitz, Aduro BioTech; Dr. Xiang Yang Yu, Ironwoodpharma; Dr. Grace Wong, ActoKine (former Genentech); Prof Wenhui Wang, New York Medical College; Prof Yan Zhu, Tianjin University of TCM; Prof Sami Noujaim, Tufts medical center; Dr. Yazdani B. Shaik Dasthagirisaheb, BU

2:00 pm Smart pitch – Biotech Who's who (the joy of learning)

2:15 pm Short presentation: Why scientist? The past, the present & the future. Dr. Jason Deng, MIT; Dr. Junjie Lu, Harvard; Dr. Izabela Durzynska, Queens University Belfast, Northern Ireland; Dr. Chuanwu Wang, MIT; Dr. Yanyan Liu, Purdue University; Dr Remi Villenave, Harvard; Dr. Behzad Etemad, BU; Dr. Ravikumar Vasudevan, University of Florida; Dr. Jiayi Zhou, BU; Dr. Weiping Wang, MIT

Smart pitch-Biotech who's who (the joy of sharing) Dr. Bin Ma, BiogenIdec; Dr. Bob Ward, AstraZeneca; Dr. XianLu Qu, Merck; Dr. Shuqi Wang, Harvard Hiromi Miura, SciVax, Japan; Dr. Elichilia Shao, East Africa; Dr. Li Xie, UMass Medical School; Dr. Changyou Zhan, Boston Children's Hospital; Dr. Aoune Barhoumi, MIT; Dr. Steve Arkinstall, EMDSerono; Dr. Mia Wang, Abbott; Julio Vito Wykrota, EincoBio, Brazil; Chip Allee, CeuticalSoft; Katarzyna Kaczmarek, BU; Dominik Conrad, Justus-Liebig University, Giessen, Germany; Anisha korde, Northeastern University; Supriya Demagu, Northeastern University Prakhar Kapoor, University of Massachusetts Lowell; Mansi Soni, University of Massachusetts Lowell

4:00 pm Networking for skills & advice (Sea of learning)

5:00 pm Closing remark (Dr. Grace Wong, ActoKine Therapeutics)

The 2011 Linus Pauling Symposium in Boston was a wonderful opportunity and experience for me to see first-hand that the spirit and camaraderie among academic and biopharmaceutical scientists in promoting science is alive and well. It was a terrific way to pay tribute to the late Dr. Linus Pauling. It was a privilege to be part of the event.

Dr. Thomas Tan, Roche NJ (Dec 26, 2011)

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2011 Nobel-Pauling Biotech Pharma Dim Sum Symposium Boston, Dec 26, 2011 (11 am to 5 pm)

Agenda

11:00 am Registration & who’s who (networking, dim sum from 11am to 2pm)

12:00 pm Welcome: Dr. Grace Wong, CEO, ActoKine Therapeutics, Boston. Legacy of Dr. Linus Pauling: Dr. Zhen Huang, Dept of Chemistry, MIT

12:30 pm Keynote speakers: Dr. Seng-Lai (Thomas) Tan, Senior Research Leader, Roche, NJ Seminar title: The Biopharma Dilemma: Unprecedented Challenges and Opportunities Dr. Zhijian Lu, Head of Biologics, China Novartis Dr. Li Xing, Senior Principal Scientist, Pfizer Dr Grace Wong, CEO, ActoKine Therapeutics 1st seminar title: AK-1 for Radioprotection & AK-2 for virus prevention 2nd seminar title: Get a foot in the door

2:00 pm Self Introduction from panel speakers – Who's who

2:30 pm Panel Discussion: Yin & Yang: lessons learned from Academia and BioPharma Moderators: Dr. Jason Xiang (Executive Director, ChemPartner) & Dr. Paul Yang, MGH (why scientist? The past, the present & the future)

Dr. Thomas Tan, Roche, Senior Research Leader, Hoffman-La Roche, NJ Dr. Zhijian Lu, Head of Biologics, China Novartis Dr. Li Xing, Senior Principal Scientist, Pfizer; Dr. Ju Huang, Postdoc, Harvard Dr. Zhen Huang, Dept of Chemistry, MIT; Dr. Andrej Jedinak, Harvard www.nobel-pauling.org/BiotechAgenda.html 9/16 MED001673 9/11/2020 Nobel-Pauling Biotech Pharma Symposium - Biotech Agenda Lan Yang, Genzyme; Dr. Bhupendra Shravage, UMass Medical School Dr. Wei Xu, CEO Broadband Photonics; Dr. Hironao Nakayama, Harvard Prof. Libin Cui, Boston U School Med; Dr. Lingge Lu, Harvard Med School Prof. Tara Devi Ashok, UMass Boston; Dr. Ravindra Prajapati, Harvard Dr. Aditya Ambade, UMass Medical School; Dr.Yaguang Si, Agios Pharma Dr. Dapeng Chen & Dr. XianLu Qu, Merck; Dr. Ravindra Prajapati, Harvard Dr. Gene Lee, EMDSerono & Dr. Steve Arkinstall, EMDSerono Dr. Jianguo Yang & Dr. Daotian Fu, Genzyme; Dr. Jiandong Geng, BU Dr. Sridaran Natesan, Scientific Site Head (R&D), Sanofi-Aventis Dr. Mia Wang, Dr. Carolyn Hsu & Dr. David He, Abbott

Dr. Victor Chu, BMS & Dr. Mak Jawadekar, former Pfizer Dr. Gunther Winkler, former BiogenIdec; Nick Leap, Genewiz Dr. B Xia & Dr. D Qin, GSK; Dr. WL Zhou & Dr. J. Cao, Novartis Keven Stevens & Mark Campbell, IDT & Dr. Jorge Andrade, BGI Dr. Xuan Liu, OriGene & Daniel Harris, UMass Boston Dr. Kevin Zhou, CEO, SSTK Biotech, Shenzhen, China Drs. Yajun Liu, CEO, Lisa Shen & George Li, Suzhou Julio Vito Wykrota, EincoBio, Brazil; Dr. Carl Edwards, ex-Amgen Hamilton Lenox, Neuland Lab, India; Dr. Mak Jawadekar, ex-Pfizer Dr. Jeff Wu, MD (BWH); Dr. Steven Levine; Dr. Lex Van der Ploeg, ex-Merck Urs Tanner, Medela, Switzerland; Chip Allee, CeuticalSoft Dr. Zhu Huaxing, CEO, Novoprotein & Patrick Burke, Director, Amarex

4:00 pm Smart pitch or tell a story: by students/postdocs/scientists

5:00 pm Closing remark (Dr. Grace Wong, CEO, ActoKine Therapeutics, USA)

Dear Grace, During the 2011 Xmas Pauling symposium, it was a great experience to see how you care for young scientists to flourish and bring out the best in them by linking the students with industry and academic personnel. It was a great honor to be part of this group. I wish you all the best in your endeavors of this kind. Thank you. "Scientific quest is unquenchable, the path most adventurous, what can be more fascinating than knowing every day a new scientific truth. Come, one and all to be initiated into scientific endeavors to be eternally in search of truth." (by Prof. Tara Devi S. Ashok, University of Massachusetts Boston).

Prof Tara Ashok, Umass Boston (Dec 26, 2011).

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Thanks to Drs Yanjun Liu, CEO, Suzhou, Lisa Shen & George Li and Novoprotein (Dr. Zhu Huaxing, CEO and Dr. Cai Lijun for sponsoring the 2011 Dec 2 Shanghai Nobel-Pauling BioPharma Symposium. Thanks for Prof Yong X. Wang and Prof Dongqing Wei for co-organizing the Linus-Pauling Biotech Pharma Symposium on Dec 2, 2011 at Shanghai Jiao Tong University (SJTU) and Huang Jinlu(黄金路 Gold Road) for volunteering to take photos at the symposium.

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Dear Grace, Thank you for taking your precious time to bring so many speakers here to speak at the Shanghai Jiao Tong University (SJTU). Honestly speaking, I learned much more knowledge not only from sciences but also from life. Thanks for teaching us to be useful for the society & the world. Thanks for inspiring us to help our boss succeed and also respect our mentors. Thanks for sharing your exciting science and practical advice. I will need to pick up a lot skills to make myself useful. The advice from the Pauling Symposium speakers has broadened my horizons. For example, how to start, develop and grow a company. I really liked the symposium so much not only because of the free pizza or Chinese food, but because the valuable advice and the opportunity to meet speakers face to face for future jobs or collaborations. I believe others also enjoyed the Pauling symposium very much. It was my pleasure to volunteer to take photos for the symposium. I look forward to taking more photos for the future Nobel-Pauling Biotech Pharma Symposium. Thank you, Gold Road

Jinlu Huang (Gold Road) 黄金路, Student at SJTU, Shanghai (Dec 2, 2011).

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2011 Nobel-Pauling Biologics Symposium at Shanghai Jiao Tong University The Nobel-Pauling Biotech Symposium series is held in honor of the Life and Work of Dr. Linus Pauling, winner of two unshared Nobel Prizes - in 1954 for Chemistry and in 1962 for Peace.

The goals of the Nobel-Pauling Biotech symposium are: To build a biotech bridge between USA, Europe and Asia for new drug discovery To encourage biotech collaboration between academia and the biotech industry To share practical advice for students, postdocs, scientists and young CEOs

The topics are: 1. Open Innovation: Academia vs. Industry for new drug discovery. 2. If Innovation is the biotech seed, where is the soil? 3. Ask the experts: how to get a foot in the door.

Date and Friday Dec 2, 2011 (2:00 - 10:00 pm) time: Fee: $0 registration at www.nobel-pauling.org Location: Shanghai Jiao Tong University, Shuhua Lecture Hall, The Biomedicine Building, 800 Dongchuan Road, Shanghai (Phone: 135-6448-3969). Sponsors: www.eincobio.com.br; www.NeulandLabs.com, www.QIAGEN.com, www.novoprotein.com Webs: www.studentvision.org, www.Pauling.us & www.nobel-pauling.org.

Agenda

2:00pm Mixing the arts with science: slide presentation through the performing arts 2 seminars by Dr. Grace Wong, CEO, ActoKine Therapeutics, Boston, USA 1st seminar: AK-1 for cancer & AK-2 for virus prevention www.nobel-pauling.org/BiotechAgenda.html 14/16 MED001678 9/11/2020 Nobel-Pauling Biotech Pharma Symposium - Biotech Agenda 2nd Seminar: Biotech: Academia vs. Industry & Get a foot in the door Smart pitch: Lin Huang, Lina Ma, Peisi He, Yukun Wang, Qiang Zhou, Yufang Wang, Huaimeng Fan, Mingzhu Zhao, Shigao Chen, Hao Dai and Xiaolei Cui

4:00 pm Who's who - Self introduction or smart pitch

5:00 pm Welcome: Dr. Grace Wong, CEO, ActoKine Therapeutics, Boston, USA

5:10 pm Introduction: Prof Yong X. Wang & Prof. Dong-Qing Wei, SJTU

5:20 pm Panel speakers – Biotech Who's who (mixing the biz with science)

6:00 pm Panel Discussion: Yin & Yang: lessons learned from working many years in BioPharma Moderators: Prof Yong X. Wang and Prof. Dong-Qing Wei, SJTU

Dr. Zhenping Zhu, Executive VP, Kadmon Corp. GM, Kadmon, Shanghai Dr. Wenzhi Tian, CEO, Huabo BioPharma, Shanghai Dr. Allan Riting Liu, VP, Wanbang Biopharmaceutical Group, China Dr. Michael Yu, CEO, Innovent Biologics, Shanghai, China Dr. Cai Lijun, VP and Dr. Zhu Huaxing, CEO, Novoprotein Dr. Zhijian Lu, Head of Biologics, China Novartis Institutes Dr. Jason Wong and Dr. Min Wu, Roche, China & Kyung-Hee Jung, Korea

Jeff Hou, Australian Institute for Bioengineering & Nanotechnology Drs. Yanjun Liu, CEO, Lisa Shen and George Li, Biotech, Suzhou Dr. Jeffrey Su, CSO, Cytovance Biologics, Oklahoma City, USA Dr. Karen Wen, CEO, Mycenax, Taiwan Dr. Joerg Lindenblatt, Sartorius, Germany Dr. Michael Lee, Biomabs Pharma, Shanghai & Akira Hosoki, BioPharma, Japan Dr. Andrew Racher, Lonza, UK & Sze Guan Chua, Lonza, Singapore

Dr. Guoqian Xu & Dr. DQ Wang, Bayer Dr. Li Feng, CEO, Beijing Mabworks Biotech Clement Leonard Trono, Tommy Tang, Huang Tao, Genor Biopharma Dr. Hongfeng Zhou, COO, YZYBio, Wuhan & Dr. Wenyong Wang, SF Dr. Bo Xu, Wuxi AppTec & Dr. James Ruan, Wuxi PharmaTech Dr. Howard Hong, Mark Hu, Craig Sng, Parker, Beijing Dr. Scott Liu, CEO, Henius Biotech & Dr. Kelvin Shao, Newsummit BioPharma Kevin Zhou, CEO, SSTK Biotech & Dr. Jian Ni, CEO, Human Antibodomics Prof Jianhua Chang, Fudan, Prof Yuhong Xu, SJTU, Prof. Yong X. Wang, SJTU & Prof. Dong-Qing Wei, SJTU

7:30 pm Presentation by speakers

9:30 pm Closing remarks (Dr. Grace, ActoKine Therapeutics, Boston)

Note: All contents Copyright 2020. All photographs belong to Nobel-Pauling. If you would like to download or use any of these photos please contact Alex Pauling alex@ Nobel-Pauling.org or call 617-566-0511. Wish to be a sponsor, please send a logo to alex@ Nobel-Pauling.org.

For past Symposia (2003 to 2010) please visit www.pauling.us and www.studentvision.org

Nobel-Pauling (coyright 2020) 12 Middlesex Rd # 411, Chestnut Hill, MA 02467, USA 1-617-566-0511 Alex Pauling: [email protected]

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www.pauling.us www.studentvision.org www.soleluna.us www.actokine.com

www.nobel-pauling.org/BiotechAgenda.html 16/16 MED001680 Register by January 15 and SAVE up to $200 Premier Sponsors

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Conference: February 3-5 | Exhibits: February 3-4 Moscone North Convention Center | San Francisco, CA

PLENARY KEYNOTES

When Drug Research is Personal John F. Crowley, Founder, Novazyme Pharmaceu cals, Inc.

Technology, Aging, and the Brain Biotechnologies GmbH ® Gary W. Small, M.D., Professor, Meso Scale Discovery David Geﬀ en School of Medicine, University of California, Los Angeles

Chips, Clones and Living

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Co - Sponsors

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Tuesday, February 2 The Open Access Publisher

8:00 AM Morning Short Course Registration and Coffee

9:00 - 12:00 PM Morning Short Courses (Courses 1-6)

10:15 - 10:30 Networking Coffee Break

1:00 - 2:00 Afternoon Short Course Registration 2:00 - 5:00 Afternoon Short Courses (Courses 7-12)

3:15 - 3:30 Networking Refreshment Break 5:00 Close of Day

Wednesday, February 3

7:00 AM Registration and Morning Coffee 8:00 - 9:40 Plenary Keynotes

9:40 - 11:00 Grand Opening Refreshment Break in the Exhibit Hall Nanomedicine 11:00 - 12:40 PM Concurrent Channels

12:40 - 1:45 Sponsored Luncheon Presentations or Lunch on Your Own

1:45 - 2:15 Dessert in the Exhibit Hall 2:15 - 4:20 Concurrent Channels 4:20 - 5:20 Reception in the Exhibit Hall (Sponsorship Available) 5:20 - 6:20 Break-out Discussions 6:20 Close of Day Sponsoring Organizations Thursday, February 4 8:25 - 10:30 AM Concurrent Channels

10:30 - 11:30 Poster Competition, Refreshment Break & Rafﬂes in the Exhibit Hall

11:30 - 12:30 PM Concurrent Channels

12:30 - 1:45 Sponsored Luncheon Presentations or Lunch on Your Own

1:45 - 2:15 Ice Cream Refreshment Break in the Exhibit Hall

2:15 - 3:05 Plenary Keynote Session

3:05 - 3:45 Refreshment Break in the Exhibit Hall CHI’s Intro-Net: 3:45 - 5:50 Concurrent Channels 5:50 Close of Day Networking at Its Best! Friday, February 5 Maximize Your Experience 8:30 - 10:20 AM Concurrent Channels onsite at the Molecular Medicine Tri-Conference! 10:20 - 11:00 Coffee Break The Intro-Net offers you the opportunity to set up 11:00 - 12:00 PM Concurrent Channels meetings with selected attendees before, during and after this conference, allowing you to connect 12:00 - 1:00 Sponsored Luncheon Presentations or Lunch on Your Own to the key people that you want to meet. This online 1:00 - 3:05 Concurrent Channels system was designed with your privacy in mind and is only available to registered session attendees of 3:05 Close of Conference this event.

2 Online: Tri-Conference.com Email: [email protected] Fax: 781-972-5425  MED001682 PRE-CONFERENCE SHORT COURSES* Tuesday, February 2 *Separate Registraon Required

MORNING COURSES: 9AM – 12PM AFTERNOON COURSES: 2PM – 5PM (SC1) APPLYING NEXT GENERATION SEQUENCING TECHNOLOGIES TO RESEARCH (SC7) BEST PRACTICES IN TRANSLATIONAL & PERSONALIZED MEDICINE Introducon to New Technologies and Applicaon in Research Real world soluons currently in place in pharma, naonal labs, academia, and industry Technologies for newest plaorms for next generaon sequencing Building collaboraons and sharing biological data between Big Pharma Strategies and tools for managing data Bridging the gap between bench and bedside Demonstraon of how tools can be applied to research Informacs soluons that link data from the clinic with cung edge research Course Moderator: Course Instructors: Stanley Gloss, Founding Partner Managing Director, BioTeam, Inc. Jeffrey S. Barre, Ph.D., FCP, Pediatrics Director, Pediatric Pharmacology Research Unit, The Course Instructors: Children’s Francisco M. De La Vega, D.Sc., Disnguished Scienfic Fellow, Computaonal Genomics Research, Hospital of Philadelphia Genecs Systems R&D, Life Technologies Lisa LaLuna, Senior Vice President, Corporate Development & Implementaon, Giles Day, Senior Director, BBC Informacs, Pfizer Biotherapeucs & Bioinnovaon Center ePharmaSoluons Ronald W. Davis, Ph.D., Professor, Biochemistry & Genecs, and Director, Stanford Genome Jeremy Packer, Head, Bioinformacs, Abbo Technology Center, Stanford University Faye D. Schilkey, Associate Director, NM Sequencing Center, Naonal Center for Genome (SC2) ONE CASE STUDY IN BREAST CANCER THREE PERSPECTIVES Resources Illustrang Current Challenges in Personalized Medicine Overview of case study Clinical perspecve (SC8) STRATEGIES FOR MOLECULAR DIAGNOSTIC COMPANIES Diagnosc and biomarker perspecve Achieving Success in Rapidly Changing Markets Payer perspecve Why and how diagnoscs markets have changed Course Instructors: Strategies for success: convenonal or new markets? Michael Liebman, Ph.D., Managing Director, Strategic Medicine, Inc. Major business model quesons including partnering Laura J. Esserman, Professor, Surgery, University of California, San Francisco Medical Center How to obtain your next round of funding Tracey Colpis, Ph.D., Manager, Abbo Molecular Course Instructors: Keith F. Batchelder, Chief Execuve Officer, Genomic Healthcare Strategies (SC3) MIGHTY MITOCHONDRIA: Their Relevance to Disease and Translaonal Peter S. Miller, Chief Operang Officer, Genomic Healthcare Strategies Medicine Mitochondria in disease and drug induced toxicity (James Dykens) (SC9) FRAGMENT-INSPIRED MEDICINAL CHEMISTRY Assessing mitochondrial funcon preclinically (Yvonne Will) Fragment-based approaches as plaorms for medicinal chemistry N N on-invasive mitochondrial assessment in the clinic (Robert Wiseman) Fragment-based methods that inspire fresh approaches to lead generaon Course Leader: Opmizaon of fragment hits Yvonne Will, Ph.D., Compound Safety Predicon- WWMC, Cell Based Assays and Mitochondrial Combining technology with fragment-based methods to advance medicinal chemistry Biology, Pfizer R&D Facing the challenge of applying fragment-based approaches when structural informaon is Course Instructors: James Dykens, Ph.D., Drug Safety R&D, Pfizer Inc not available Yvonne Will, Ph.D., Compound Safety Predicon- WWMC, Cell Based Assays and Mitochondrial Promises and pialls of surface plasmon resonance (SPR) for fragment methods Biology, Pfizer R&D Course Instructors: Robert Wiseman, Ph.D., Associate Professor, Department of Physiology, Michigan State University Michelle Arkin, Ph.D., Associate Director, Biology, Small Molecule Discovery Center, Pharmaceucal Chemistry, University of California, San Francisco (SC4) ADDRESSING SAFETY CONCERNS FOR BIOLOGICAL DRUGS Daniel A. Erlanson, Ph.D., Co-founder, Carmot Therapeucs, Inc. Overview of challenges pertaining to safety for biologics Safety assessments at pre-clinical and clinical stage (SC10) TRANSPORTER-MEDIATED DRUG-DRUG INTERACTION POTENTIAL Use of new assays, animal models and biomarkers for early predicons Strategies for in vitro Characterizaon Clinical relevance of transporter DDI’s Regulatory guidelines and their interpretaons Course Instructors: In vitro, cell based models for evaluang transporter interacons of substrates and inhibitors Hong Wang, Ph.D., DABT, Safety Assessment, Genentech Inc. Case study: minimizing p-glycoprotein interacons as a barrier to CNS penetraon Kathleen Meyer, MPH, Ph.D., DABT, Senior Director, Preclinical Safety Evaluaon, XOMA (US) LLC Course Instructors: Phil Burton, Ph.D., Chief Execuve Officer & Chief Scienfic Officer, ADMETRx, Inc. (SC5) TARGETING CANCER STEM CELLS WITH BIOLOGICS Xingrong Liu, Ph.D., Senior Scienst, DMPK, Genentech, Inc. Novel nanoparcle fusion proteins, tr1 and tr4, that achieve normal p21 delivery to p53/p21 Joseph A. Ware, Ph.D., Senior Scienst, Clinical Pharmacokinecs and Pharmacodynamics, mutated tumors (tr1) and inhibion of notch signaling (tr4) resulng in tumor eradicaon Development Sciences, Genentech, Inc. Differenaon versus self-renewal: changing cancer stem cell fate by targeng stem cell pathways Cancer stem-like cells: isolaon using biological criteria and use in drug discovery and development SC11 BASIC IMMERSION: CUTTING EDGE SCIENCE & TECHNOLOGY FOR Cd47: an adverse prognosc factor and therapeuc anbody target on human acute myeloid BIOTECH & PHARMA leukemia stem cells Gain a fundamental understanding of the science and technology driving the Biotech/ Idenficaon of stem cell markers in the normal prostate and prostate cancer Pharma industry Course Instructors: Learn basic scienfic terminology used by researchers in the life sciences Agamemnon Epenetos, Ph.D., FRCP, Chairman, Trojantec Ltd. Designed for the non-scienst working with or in the biotech/pharma industry Ausn Gurney, Ph.D., Vice President, Molecular and Cellular Biology, OncoMed Pharmaceucals, Inc. Immersion course on the biotech basics; Recombinant DNA, Proteins, Stem Cells, Biologics, Jennie P. Mather, Ph.D., Senior Vice President, Stem Cell Research, MacroGenics, Inc. Drug Discovery and Drug Development Ravi Maje M.D., Ph.D., Assistant Professor, Division of Hematology, Stanford Cancer Center, Instute Class Materials Include: The Primer: A Biotechnology Guide for Non-Sciensts for Stem Cell Biology and Regenerave Medicine Course Instructor: Kevin G. Leong, Ph.D., Scienst, Tumor Biology and Angiogenesis, Genentech, Inc. Karin Lucas, Ph.D., BioTech Primer Instructor and Scienfic Advisor (SC6) BLOOD-BRAIN BARRIER The physiological basis for the “barrier” nature of the BBB (SC12) DESIGNING RIGOROUS OMICS STUDIES FOR BIOMARKER Experimental approaches (in vitro/in vivo) that are available for screening for brain penetraon DISCOVERY AND DEVELOPMENT OF PROGNOSTIC AND Medicinal Chemistry perspecve on in vitro/in silico approaches for opmizing CNS penetraon PREDICTIVE MOLECULAR DIAGNOSTICS Mul-parameter opmizaon (MPO) for CNS penetraon Why study design is decisive for success or failure In vivo examples where all these concepts are applied together, e.g., consideraon of free Crical review of examples fracons in various compartments in relaon to in vitro pharmacology values Dos and don’ts Projecng human receptor occupancies considering species differences in affinity, A roadmap to the answers free fracon Samples: How many are enough? Exposure targeng for biomarker studies Apples and Oranges: Tackling confounding factors Course Instructors: Companion diagnoscs in clinical trials Christopher L. Shaffer, Ph.D., Associate Research Fellow, Pharmacokinecs, Dynamics & The regulatory perspecve Metabolism, Pfizer, Inc. Course Instructors: Douglas Spracklin, Ph.D., Director, Pharmacokinecs, Dynamics and Metabolism, Pfizer, Inc. Terry Speed, Ph.D., Professor, Department of Stascs, University of California, Berkeley Travis T. Wager, Ph.D. Associate Research Fellow, Neuroscience Discovery, Medicinal Chemistry, Juergen von Frese, Ph.D., Managing Director, Data Analysis Soluons, DA-Sol GmbH Pfizer, Inc. Donna Roscoe, Ph.D., Senior Reviewer, FDA/OIVD/DIHD

Online: Tri-Conference.com Email: [email protected] Fax: 781-972-5425 3  MED001683 4 DIAGNOSTICS CHANNEL pa to market. var focus ontheseelements and discuss1-2case studies ofhowwe at No- ward bringinginnova prac standing ofbothtargeted drugdiscovery anddrugcommercializa is fullyleveraged to enableinnova alized medicine.At thesame pharmaceu medicine. has exploded. Thechallenge faced byresearchers isto journey from research technology to diagnos standing ofdiseasemechanismsfor personalized medicine. Butthe adopted intheresearch labsandhelpbreakthroughs inourunder- to translate thetools for theresearch labinto solu the bridge from research tool to rou personalized medicineiscoming closerbutwhat willittake to cross and slow. Asourunderstanding ofdiseaseincreases thepromise of evant informa ly-indexed biomarker informa discussion we willintroduce BIOMARKERcenter, acomprehensive, ful- Molecular Diagnos show howitaidsthediscovery process. 7:00 AMRegistra 11:05 MolecularDiagnos Glorikian,ManagingPartner,Harry Scien 11:00 Chairperson’s Remarks has embarked onandthepartnerships andcollabora Pharma asaValue Driver for MolecularDiagnos medicine. ThePresenta 9:40 Grand OpeningRefreshment Break intheExhibitHall 8:00 PlenaryKeynote Session 12:50 LuncheonPresenta Michael C.Li diagnos Informa In recent years, thequan Thomson Reuters Healthcare andScience Colin Williams,Ph.D., Director, ProductStrategy, New technologies, suchasnext genera Mark Stevenson, President &COO, Life Technologies Corp. Personalized Medicine:ItTakesPM 12:15 aVillage seize thegrowth opportunityofpersonalized medicine. 11:40 BuildingaSuccessful Diagnos Richard Ding,CEO, bioTheranos Era ofPersonalized Medicine space. Thispresenta s been generally accepted asaninevitable trend inhealthcare. However, muchdebate is Next Wave of Personalized Medicine Personalized of Wave Next Diagnostics: Molecular Cambridge HealthtechInstitute’s Seventh Annual WEDNESDAY, FEBRUARY 3 ll ongoing related to asustainable businessmodelfor diagnos ent outcomes, itisimpera s are usingourdiscovery anddevelopment approach to work to- ce andin c companies, explore partnership modelsandpropose somebasicframework to

Online: on Trends inBiomarker Research le, GlobalHead,Diagnos cals andanessen ﬂ on thebest biomarker quicklyandreliably. Inthis KEYNOTE PRESENTATIONS Tri-Conference.com uence physician decision-making.Thekeynote will on williden cs isacri on andMorningCo ve companion andstand-alone diagnos on willfocus onthejourney Life Technologies cs, abioMerieuxCompany ty ofdata published onbiomarker research fy variousfy challenges, risks andpoten cs asaValue Driver ofPharma/ a ucs atrfrteftr f cal successfactor for thefuture of on on resource, andthrough case studies me, to progress be cs Development, Novar al aspectofthemove toward person- ve that thepharmaindustry’s under- a Advisors (See Page 26for Details) ve diagnos

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Email: c systems ischallenging cs to beputinto clinical Personalized medicinehas cs companies in this new cs companies inthisnew Sponsored by s MolecularDiagnos cs inpersonalized er healthcare and [email protected] ons personalized al returns for ons necessary cs ﬁ nd vital, rel- c tests on cs Novel Instrumenta for Molecular BiologyandMedicine Single Tube for Sensi Genotyping ofPathogens andAn and Real-Time Microarray Detec Diseases(RAP-ID): Merging Mul Vera Gor Vera consultant, Genometrica Corpora The technology pla instruments capable ofcarrying outonequalfoo and uniqueresearch capabili highly accurate genomic hybridiza- studies includingDNAsequencing, ciples, novel engineering solu croarrays (mul RAP isanovel hybrid technology combining majoradvantages ofmi- Wilhelm Pluester, Ph.D., CEO, Eppendorf ArrayTechnologies S.A. dynamic range). Target ampli proceed inasingletube(inthesamebu 4:05 Pla automated work with ven rent understanding ofthesespeci tected, and3)theselec methods used,2)speedat whichunknownposi 3:50 Real- Control ofany epidemicrelies ondetec Evanston Hospital, NorthShoreUniversityHealthSystem andUniversityofChicago Lance Peterson, R. M.D., FASCP, Director, FIDSA, Microbiology&Infec 3:20 MRSA Surveillance Programs –What ImpactsSuccess? dress various assays for detec era pathogen (infected andcolonized). For any MRSA prevalence, theop- in order to appropriately treat andisolate infected pa were available for thisunan lar approaches o The Needfor Speed Centers for DiseaseControl and Preven Applied to In 2:50 What Happened withSARS: LessonsLearnedand Joseph D. Miller, Ph.D., Chief, Laboratory PreparednessO The Novel H1N1In School ofMedicine University HealthSystem; Clinical Professor ofPathology, UniversityofChicago Pritzker and hospitals withtheneedfor highvolume tes tory perspec laboratory issues,andwillreview therecent outbreak from thelabora- Molecular Diagnos Karen L.Kaul, M.D., Ph.D., BoardofDirectors ChairofMolecularPathology, Director, 2:20 Real- 2:15 Chairperson’s Remarks 1:45 DessertintheExhibitHall on, andquan plex detec MEETING THE NEEDS OF THE COMMUNITY onal processes most in  MOLECULAR DETECTION OF PATHOGENS: ﬁ

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Collabora Advisor, Expertech Solu cess requires technology-enabled collabora bursement policiesandhealthcare ITreform. Ensuringyour lab’s suc- comes more complex. Thisisfurthercomplicated bychangingreim- and otherlabs.Thiskeynote discussesbusinessprac 9:15 Keynote Presenta performed andto builde to providers, to review payors’ reimbursement policies before tests are HIT andPM:Con for labs to o to labs for Wayne Rosenkrans, A. Jr., Ph.D., Dis Personalized Medicine Coali Innova As moleculardiagnos McKesson Corp. Advisor, Expertech Solu Strategies for Commercializa Ma Tribula Consul Moderator: IanS.Mille Patents andDiagnos Moderator: Frances Toneguzzo, Ph.D., Director, O 8:30 Keynote Presenta Adop Ensuring Responsible Tes Moderator: DavidS.Lester, Ph.D., Vice President, HumanHealthSolu 6:20 CloseofDay Moderator: Glorikian,ManagingPartner, Harry Scien 4:20 Recep Wayne Rosenkrans, A. Jr., Ph.D., Dis 8:25 AMChairperson’s Remarks Personalized MedicineCoali Innova and Licensing, Massachuse Is Economics Goingto BetheDriver ofMolecularDiagnos Execu Making MolecularDiagnos Consumer Diagnos 5:20 BREAK-OUT DISCUSSIONSintheExhibitHall Co-Moderators: Peter S.Miller, ChiefOpera THURSDAY, FEBRUARY 4 make that bill? Pushing andPulling-Whoisyour customer and just howPersonal can you Providers oftradi reaching thepublic How willthishappen? How doyou get paidfor amoleculardiagnos What are thebarriers for adop Are moleculardiagnos Type ofpatents inthediagnos Payers: USvs. interna How willvalue ofthediagnos Technology improvements andcost reduc Does implemen Other typesofintellectual property protec Consumers willremain interested andcompanies willdevelop be and proteomic analysis cheaper Strategies for e development Di Life at Ground Zero -TheFDA’s changingperspec hew B.Zubiller, VPandGeneralManager, Advanced Diagnos ff eren ve O ng Group,Inc. on; PrograminEthics andSystems Medicine,Georgetown University;Chairman, on; PrograminEthics andSystems Medicine,Georgetown University;Chairman, on? ffi ons ofLabs andMoney a cer, GenomicHealthcare Strategies ff HEALTH IT: WHY IS IT SO HARD? on Between Providers, Payors andLabs on indiagnos er decisionsupportandaccessto abroader array oftests on intheExhibitHall ng amoleculardiagnos ff ec onal care willhave to come to gripswithinformed consumers fl ons; andChiefApplica ons; andChiefApplica , Ph.D., RAC, SeniorConsultant, Medical Devices, Biologics

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Email: c, metabolic, er ways of cs ng be- fc fc fc fc [email protected] er? cs c Vance Vanier, M.D., ChiefMedical O Intel Corpora Je Mark N.Bla Brandon Savage,M.D., ChiefMedical O Panelists: 10:00 TheDevelopment ofMul and prognos 10:15 Sponsored Presenta Moderator: DavidS.Lester, Ph.D., VP, HumanHealthSolu Health IT: What WillSuccessLookLike? 11:30 ExpertPanel Exhibit Hall 12:30 PMLuncheonPresenta molecular diagnos The key to thedelivery ofpersonalized medicineisthedevelopment of Aus 10:30 Poster Compe of deliverable tests. Here we present outexperience andperspec a signatures isincreasingly ofinterest however there are many consider- 2:25 PlenaryKeynote Presenta 3:05 Refreshment Break intheExhibitHall 1:45 IceCream Refreshment Break intheExhibitHall or LunchonYour Own Jorge A. León,Ph.D.,Jorge A. President, LeomicsConsul 3:45 Chairperson’s Remarks 2:15 PlenaryKeynote Introduc inven protect theinvestment thecompany ismakingincommercializing the molecular diagnos 3:50 GenePatents, Perspec diagnos nos Gene patents are controversial, butare areality inmoleculardiag- Hospital; President Elect,Associa Hematopathology, Department ofPathology andLaboratory Medicine,Emory University Karen P. Mann,M.D., Ph.D., Associate Professor andDirector, Molecular can exclude others from prac valuable inthat they provide aperiodofexclusivity whenacompany around vate individuals/founda 4:20 GenePatents inMolecularDiagnos fi prac Prognos Frances Toneguzzo, Ph.D., Director, O or Impediments? Increasingly, gene Licensing, Massachuse or e gene fragments for accurate diagnosisofdrugresponses (toxicity and/ of situa di nance. ff ff Facilita Reducing healthcare costs Realizing solu De ons indeveloping suchsignatures from study designto development rey D. Miller erent owners, includingcompanies, academic ins ff n Tanney, Ph.D., Scien fi cs. Asalaboratorian whoco-directs anac ec ce medicineincludingthee ning thegoals on, thisfragmenta ons, thepatents covering thesegenes ortheiruseare heldby veness and/or dosing)anddiseasestra mes, choiceoflaboratories for referral tes cs laboratory, Iwilldescribehow gene patents a ng theadop , M.D., MBA, Director, Healthcare Industry Solu c andPredic on IP ISSUES ON GENE PATENTING: c tests to companion diagnos PLENARY KEYNOTE SESSION ons for reaching thegoals WHAT IS THE SOLUTION?  s GeneralHospital c diagnos cs may impedethedevelopment ofcertain tests. cs to improve pa

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5 DIAGNOSTICS CHANNEL Way to theFair? e diagnos pathways for thediagnos quate analy Safety, Food &DrugAdministra pa the diagnos IP concerns. Thistalk willdiscusspossiblesolu altera 5:20 ExpertPanel: BiosciencePatent Law including theuseofpatent poolsdriven bymedical standards. Ian S.Mille 9:05 Value BasedLaboratory Tests --What Went Wrong onthe ers eachcontrol oneorafew gene 5:50 CloseofDay all costs ofcare. ized Medicinehealthcare can improve outcomes andreduce theover- In spite oftremendous interest, expenditures, andpro-ac Group, Inc. one disease,where themul ready resulted inafailure to o gene orseveral genes involved inthedisease.Suchthickets have al- revolu by industry, academia, andgovernment, thepersonalized healthcare So called “patent thickets” have appeared whenmul PLLC Jorge Goldstein, Director, Biotechnology &Chemical Group,Sterne Kessler Goldstein Fox and use.Thistalk will focus onpoten will remind par Advances ingenomics-based discovery andtherapeu new diagnos Pharmacogenomic tests bringanewlevel ofprecision to pharmaceu- Lon Castle, M.D., SeniorDirector, Personalized Medicine, Medco HealthSolu 9:35 Medco Personalized Medicine: Advancing Healthcare the race. become more conversant withthevalue oftes apeu Director, Personalized Medicine,O 8:35 Regulatory Considera Lilly Corporate Center Brian T. Edmonds, Ph.D., Research Advisor, GlobalExternal Research &Development, 8:30 AMChairperson’s OpeningRemarks 4:50 IPFragmenta Elizabeth Mans and Personalized Medicine standard ofcare inmany therapeu have ledto greatly increased interest indevelopment ofdiagnos characteris ffi cal care, enablingtreatment that istargeted to theunique gene FRIDAY, FEBRUARY 5 How doIPfragmenta diagnos Why are somelicensingmodelsslowingdowntheadvancement ofmolecular How dothey a How dothey a users? cacy willrest. Thispresenta ents. Withthisvisioncomes therealiza U.S. UNIVERSAL HEALTH c combina ons, orhave generated test designsthat are driven primarilyby on seemsto have stalled inthearea ofnewtest development c devicesandcodevelopment willaddress proposed regulatory , Ph.D., RAC, SeniorConsultant, Medical Devices, BiologicsConsul cs? fi cs ofindividualpa cal andclinical valida c test performance uponwhichthetherapeu eld, Ph.D., SeniorGenomicsAdvisor, O c technology, willsurvey theupcoming landscape, and ff ff ect companies andacademic that discover newmarkers? ect companies that ownIPandcommercialize IVDs? cipants that intheend,itwas thetortoise that won ons that promise to deliver “personalized” therapy to

on inGene Online: on andpatent poolsa on c device,andemphasize theneedfor ade- ffi ce ofinVitroDiagnos ple patents cover eitheraltera ff Tri-Conference.com er commercial tests for allpossiblegene ons for CompanionDiagnos on of regulatory issues for companion ents. Thesetests are becoming the on. c diagnos c Diagnos c areas, asphysicians andpayers al reasons for slowuptake of BREAKING NEWS ffi ff ect theclinical labsandend ce oftheChiefScien on oftheimportance of c correla c Device Evalua ng. Medco’s Personal- cs ons to theproblem,

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Email: on and ons inone ons, Inc. st; st; ve work work ve ng c/ther- cs [email protected] ng ng c Sample-to-Result Diagnos to result. Thissessionwillprovide anintroduc ca 10:05 Innova Kevin Banks, Ph.D., HeadofMarke 20 patents with13others pending.Thecompany’s core pla Akonni Biosystems (Frederick, MD)was founded in2003andhasover es agel-drop array technology op technology andpla applica people respond to theirpersonal gene forma 11:00 Economics ofHaving Diagnos in research onapar 10:20 Co discover theirancestral roots, to for to theseservices learnabouttheirdiseaseriskorcarrier status, to and SamplePrepara 12:00 PMLuncheonPresenta does notappropriate incen markers. Inspite ofthis,thecurrent reimbursement system intheU.S. technology advancements inclinical genomics andothermolecular Prac Over 30,000individualsnowhave accessto theirpersonal gene Brian Naughton, Ph.D., Founding Scien 1:05 Consumers andTheirGenomes stakeholders (physicians, pa cen use ofdiagnos The impactofdiagnos Philip C.M.Ma,Ph.D., Director, McKinsey &Company, Inc. Sharon Terry, President MA, andCEO, Gene 2:05 Drinkingfrom theFire Hose:Are Consumers Ready? Patrick F. Terry, CEO, Technic Solu 1:35 Talk Titleto beAnnounced Challenges for Implementa 11:30 Panel Discussion:Personalized Medicineand a ers), andwillsuggest afew ways to improve themicro-economic situ- 3:05 CloseofConference 2:35 Panel DiscussionandQ&AwithAudience 1:00 Chairperson’s Remarks or LunchonYour Own VALUE OF CONSUMER BASED GENOMICS: WHAT on willdiscusstheongoing studies that are beginningto reveal how How good istheinforma How e How well can peopleunderstand theresults? How willitimpactmoleculardiagnos How willuniversal healthcare impact your business? on. ons, withanemphasisongreatly accelera IS THE CONSUMER GOING TO DO WITH IT? ves can result from theunder-lying micro-economics ofdi cal Challenges inGe on through 23andMe’s Consumers web-based services. signup ﬀ ons willbediscussed. ec ﬀ ee Break vely can they helppeoplemanage theirhealth? cs can result. Thistalk willreview howmis-alignedin- ons inMolecularDiagnos  orm. Preliminary results withanumberofclinical

cular diseasesuchasParkinson’s. Thispresenta- Fax: on Methods: Accelera cs in on delivered bythesetests? ons, LLC; Ac 781-972-5425 ves for e for ves ng andSales,Akonni Biosystems ents, payors, anddiagnos cs fl ng Reimbursement st, 23andMe on uencing care con fi on mized for developing medical appli- c Alliance nd newrela cs adop ff ng CEO, GrandTherapeu ec (Sponsorship OpportunityAvailable) c informa ve use-bothover andunder- cs Reimbursed andthe on? ng the cs on and overview ofthe on andoverview ves, or to par ng on. nues to grow with me from sample c manufactur- MED001686 Sponsored by cs, Inc. orm u ff cipate erent c in- liz-

6 DIAGNOSTICS CHANNEL Moderator: Katherine Tynan Ph.D., BusinessDevelopment &Strategic Consul Era ofEvidence BasedMedicine University Personalized Medicine:Commercial Hurdles to Adop 5:20 BREAK-OUT DISCUSSIONSintheExhibitHall 4:20 Recep Development andApplica Moderator: RayMa Pep Reac Helen Byers, Ph.D., PrincipalResearch Scien Diagnos Molecular 3:50 Presenta Fit-for-purpose assays are essen ca Mul Isotopic MassTags for theFacilitated Development of How Innova Translated for Applica of synthe standard allowingto establish TMT-SRM, amethodthat allows theuse Moderator: Kewal K.Jain,M.D., Professor, CEO, JainPharmaBiotech prietary tandem masstag (TMT)reagents to di with synthesis ofisotope-doped standard pep pep pep Moderator: Tracey Colpi Biomarkers ofE synthesize more complex standards withpost-transla (e.g. Medicine to EnhanceorReplace Conven 6:20 CloseofDay and Medical Director for Advanced Technology, ARUPLaboratories Moderator: Karl V. Voelkerding, M.D., Associate Professor ofPathology, UniversityofUtah Laboratory Next Genera The opportuni test development Thestrategic importance of“intended usestatements” inguidingproduct/ cessity for payers. and clinical validity for theFDA, clinical u The evidencerequired for eachofthestakeholders: analy Criteria Advanced 3Dco-culture approaches to model Protocols for drugscreening in3Dculture format Development of3Dformat for high-throughput assays Challenges instandardiza The idealmoleculardiagnos How willlaboratories process andinterpret thelarge amounts ofdata generated? clinical laboratory? What priori Whenandhowdowe gather prevalence data? Howdoesmechanismofac Selec What improvements would facilitate transla will beusedfor? What are the Future prospects ofdiagnos Role ofsequencing sonalized medicine enhancing orreplacing conven ons). Proteome Scienceshasdeveloped isotopic versions ofitspro- Ɵ des andproteins, butislimited byexpense anddelay connected de orprotein in any given samplematerial.. Ɵ de Biomarkers plex SRMMassSpectrometric Assays for Protein and on Monitoring (SRM)isincreasingly usedfor thequan Ɵ cs Companies,Tynan Consul on,evalua c ornatural reference standards to establish assays for any Ɵ es shouldwe bemaking? on intheExhibitHall Ɵ Ɵ Į ve Technologies Are Selected, Evaluated, and rst diagnos on SequencingintheClinical Diagnos es andchallenges withreimbursement Ɵ ƫ ngly, Ph.D., Associate Professor, Pharmacology, Wayne State on ĸ on,andtransla cacy Ʃ s, Ph.D., BusinessDevelopment, CompanionProducts,Abbo Sponsored by

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Į - to iden to Three typesofprostate cancer related gene Jianfeng Xu, M.D., Ph.D., Professor, Epidemiology, Preven vs. Non-Aggressive Prostate Cancer 9:30 MassSpecfor Prostate Biomarkers, AssessingAggressive Discovery: Applica 8:30 Personalized Oncoproteomics for CancerBiomarker Josip Blonder, M.D., Sr. Research Scien baseline PSA levels. Thesegene overall prostate cancer risk,aggressive prostate cancer risk,andhigher from genome-wide associa Metanomics Healthusesmassspectroscopy basedmetabolite pro ology ofanorganism includingdrugtreatment anddisease status. Commercializa Jack Leonard,Ph.D., Vice President ofTechnology Detec 10:00 ANovel Tool for Non-Invasive Disease We developed anovel non-invasive diagnos Discovery ofdiagnos Josip Blonder, M.D., SrResearch Scien 8:25 AMChairperson’s Remarks dogeneous andxenobio Metabolite pro Hajo Schiewe, Ph.D., SeniorManager, BusinessDevelopment, Metanomics Health Immunoassays are widely usedto measure protein biomarkers inpa- ca Iden Steven Carr, A. Ph.D., Director, Proteomics, TheBroadIns 9:00 Protein Quan 10:15 Metabolite Pro ity andclinical u u Ctrr for Cancer Genomics,Wake Forest UniversitySchoolofMedicine Spectrometry: TheWay OutofBiomarker Purgatory? of characterizing thepa personalized treatment ofcancer. Thus,proteomic approaches capable metabolome re quan targeted assay methodsemploying massspectrometry to screen and jority ofproteins. We are addressing thisseriousbarrierbydeveloping the integra Our Biomarker Signature assay hasshownanoutstanding performance for specimens are cri and progression. Theanalysis andinterpreta on thelatest developments andapplica hands-on high samplethroughput at lowcost sincefor eachtest lessthanoneminute er in can increase themechanis producibility andsensi ery intheplasmaofapa oncoproteomics isdescribedandappliedfor cancer biomarker discov- ent blood,butuseful an PERSONALIZING THERAPY: SERUM BIOMARKERS THURSDAY, FEBRUARY 4 lity. However, furtherstudies are neededto assesstheirclinical valid- ons inpre-clinical andclinical drugdevelopment, diseasediagnos Ň Ɵ uences onanorganism.

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8 DIAGNOSTICS CHANNEL 7 DIAGNOSTICS CHANNEL pa to market. var focus ontheseelements and discuss1-2case studies ofhowwe at No- for theresearch labinto solu has exploded. Thechallenge faced byresearchers isto ward bringinginnova prac standing ofbothtargeted drugdiscovery anddrugcommercializa is fullyleveraged to enableinnova alized medicine.At thesame senta and thepartnerships andcollabora pharmaceu evant informa ly-indexed biomarker informa discussion we willintroduce BIOMARKERcenter, acomprehensive, ful- show howitaidsthediscovery process. 12:50 LuncheonPresenta Molecular Diagnos 7:00 AMRegistra 11:05 MolecularDiagnos Glorikian,ManagingPartner,Harry Scien 11:00 Chairperson’s Remarks Pharma asaValue Driver for MolecularDiagnos 9:40 Grand OpeningRefreshment Break intheExhibitHall 8:00 PlenaryKeynote Session Informa In recent years, thequan Thomson Reuters Healthcare andScience Colin Williams,Ph.D., Director, ProductStrategy, Michael C.Li from research technology to diagnos ing ofdiseasemechanismsfor personalized medicine.Butthejourney adopted intheresearch labsandhelpbreakthroughs inourunderstand- research tool to rou medicine iscoming closerbutwhat willittake to cross thebridge from As ourunderstanding ofdiseaseincreases thepromise ofpersonalized 1:45 DessertintheExhibitHall diagnos New technologies, suchasnext genera Mark Stevenson, President &COO, Life Technologies Corp. Personalized Medicine:ItTakesPM 12:15 aVillage seize thegrowth opportunityofpersonalized medicine. 11:40 BuildingaSuccessful Diagnos Richard Ding,CEO, bioTheranos Era ofPersonalized Medicine space. Thispresenta s been generally accepted asaninevitable trend inhealthcare. However, muchdebate is “Under the Hood” Technologies for Molecular Diagnosti Molecular for Technologies Hood” the “Under Diagnostics Personalized Cambridge HealthtechInstitute’s Inaugural WEDNESDAY, FEBRUARY 3 ll ongoing related to asustainable businessmodelfor diagnos ent outcomes, itisimpera s are usingourdiscovery anddevelopment approach to work to- ce andin on willfocus onthejourney Life Technologies hasembarked on c companies, explore partnership modelsandpropose somebasicframework to

Online: on Trends inBiomarker Research le, GlobalHead,Diagnos cals andanessen ﬂ on thebest biomarker quicklyandreliably. Inthis KEYNOTE PRESENTATIONS Tri-Conference.com uence physician decision-making.Thekeynote will on williden cs isacri on andMorningCo ne diagnos ve companion andstand-alone diagnos cs, abioMerieuxCompany ty ofdata publishedonbiomarker research fy variousfy challenges, risks andpoten ons personalized medicine. cs asaValue Driver ofPharma/ a ucs atrfrteftr f cal successfactor for thefuture of on on resource, andthrough case studies me, to progress be cs Development, Novar al aspectofthemove toward person- ve that thepharmaindustry’s under- cs inpersonalized medicine.ThePre- a Advisors (See Page 26for Details) ve diagnos ons necessaryto translate thetools

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Email: cs to beputinto clinical Personalized medicinehas cs companies in this new cs companies inthisnew s MolecularDiagnos Sponsored by er healthcare and [email protected] al returns for cs fi nd vital, rel- c tests on cs Engineered HumanTumors correla This pla Tracey Colpi A method ofpredic peu 2:20 Rela Linda McAllister, M.D., Ph.D. 2:15 Chairperson’s Remarks pathways inDCIS. and compared. Strikingsimilari will bediscussedanddemonstrated usingdata from therapeu therapies, andsubgroups reveals arela popula We have developed atractable, and e Ray Ma Whole GenomeArray Analyses Carcinoma Modelby BothDigital GeneExpression (DGE)and 2:50 Network andPathway Analysis ofaNovel 3DBreast DNA level, andthepopula in situ genic humantumors that feature naturally occurringtumorvaria scripts suggest trunca scribed outsideofstandard (NCBI36.3)genes models.Thesetran- DCIS. DGEanalysis revealed abroad range ofproducts that are tran- lumina/Solexa) to explore thenetworks andpathways that underlie akin to that inhumantumorpopula observed microarray (A membrane (rBM).We have applied andcross-validated wholegenome that aidinselec involving EGFR inhibitors colon, andbreast inlung, cancer. Biomarkers Min Wu, Ph.D., PrincipalScien assess thee based tumormodelsystem basedonHuman-in-Mouse 3:20 Popula hibit inherent ar xenogra lenges for drugsto meet regulatory endpoints. Cancer celllinebased Human tumorpopula able to adequately capture natural varia popula results inlowresponse rates intheclinicandcreates signi where eachtumorharbors auniqueset ofgene pact prognosis andresponse to treatment. Unfortunately, thisvaria PERSONALIZING THERAPY: TISSUE BIOMARKERS ve e ve  c response inanvivo humantumorsystem. ffi ffi (DCIS)basedon3Doverlay culture inrecons cacy, whichisvisualized inthee cacy studies ofan ngly, Ph.D., Associate Professor, Pharmacology, Wayne State University

on hasbeencomprehensively characterized at theRNAand ons. To address thischallenge, we have created apopula ons between response andthegene Fax: orm enablesusto iden s have tradi s, Ph.D., Manager, Abbo ng Biomarkers to E ffi ff 781-972-5425 cacy ofclinical compounds, however, suchmodelsex- ymetrix) anddigital gene expression (DGE)analyses (Il- on Based ng subgroups ofpa facts dueto longterm invitro culture, andare un- ng response inasubgroup de onally beenthepreclinical modelofchoiceto ons andchanges inan ons exhibit signi st, Transla in vivo -cancer agents andcombina on hasbeenadapted to conduct quan : Molecular Biomarker Discovery Using onal Research, AVEO Pharmaceu ffi fy andvalidatefy biomarkers ofthera- es between thedi in vitro cacy: The E The cacy: ents ofresponse were analyzed ffi modelofductal carcinoma fi cacy curve. cacy onship between biomarkers MED001688 cant inter-tumor varia on seen in human tumor -sense driven regulatory c context ofthetumors. ons. Each tumorofthe ffi c altera fi cacy Curve cacy ned byabiomarker tuted basement ff erent cancers, ons, enabling ons that im- fi ssue trans- cals, Inc. cals, Inc. cant chal- cant cs c trials on, ta- on on on in thedevelopment ofpersonalized treatment strategies inoncology. CTCs, anddiscuss howCTC-derived biomarkers willbecome acri and protein) ofCTCs willsigni disease agent iden ta ring duringdiseaseprogression andasaresponse to therapy. Thispresen- tests, andenableserialmonitoring ofCTCs for molecularchanges occur- and Iden ated to determine ifatreatment-related reduc with metasta DNA fragments that enter theperipheral circula ing themandthendetec These includeop 12:35 LuncheonPresenta Procedures have beendeveloped to enhancethecollec Cantor,Charles R. Ph.D., CSO, Sequenom,Inc. Biomarkers prenatal diagnos nucleic acidmassspectrometry. Themethods showpromise innoninvasive 11:20 Metabolomic Analysis ofProstate CancerProgression Exhibit Hall 12:10 PMProgress inNoninvasive Detec Nanoscale protein arrays willbecome anessen Jennifer Ohayon,Ph.D., BioDiscovery ProjectLeader, NanoInk,Inc. Enhanced Sensi Enumera 10:30 Poster Compe high throughput pro in mentheUnited States anda Prostate cancer isthesecond most common cause ofcancer-related death College ofGeorgia Arun Sreekumar, Ph.D., Molecular Oncology Program,Genomics/Epigenomics, Medical ment PSA andincrease itsspeci age of65.There isanurgent needto develop biomarkers that can supple- olithography (DPN)pla suit ofpersonalized medicine.NanoInk hasdeveloped several DipPen Nan- Circula Nicholas C.Dracopoli, Ph.D., VP, Biomarkers, Centocor R&D, Inc.,Johnson& extension, organism) at aspeci lism, istheonlywindowinto thecurrent andactualstate ofthecell(orby or 109cellsinblooddrawn from somepa Comprehensive Characteriza direc the gene other omics-style sciences,where genomics isbest understood asde the singlebest windowinto thecellular state discovered to date. Like the cally alter thefuture ofhealthcare, drugdiscovery, anddrugdelivery. Itis 11:45 Circula Recently we have pro using acombina genomics andproteomics, isayoung sciencethat hasthepoten changes ingenome, proteome andmetabolome. Metabolomics, unlike term objec being characterized inthecontext ofprostate cancer progression. Ourlong pro the levels of>1000metabolites across 250biospecimens.Results ofthe In addi of sarcosine pathway were found to regulate prostate cancer aggressivity. to beassociated withadvanced prostate cancer. Importantly components prostate cancer progression. capable ofgenera surfaces. Theresultant enhancement inbiomarker sensi ve andprecise thancommonly usedalterna ve oftherapeu onal poten on willreview newapproaches for theisola fi ling study revealed elevated levels ofsarcosine orN-methyl glycine on ofthecellularac ng tumorcells(CTC) are very rare andconsist ofabout1in108 on to sarcosine we have de c poten on ofCTCs hasbeen shown to have prognos fi ve is to de ca al ofthecell;metabolomics, theomicsscienceofmetabo- c breast prostate andcolorectal cancer, andisbeingevalu- ng Tumor Cells:From Enumera on UsingNano-scale Protein Arrays c response. Comprehensive characteriza cs, tumordetec on ofGCandLC chromatography. Ourstudy quan mized methods ofrecovering smallfragments, amplify- al, transcriptomics isawindowinto thefuture (desired) vity ofBiomarker Detec ng sub-micron sized features ofbiomoleculesonsolid fi fi ca fi ling strategies hasallowed scien

led themetabolome inprostate cancer progression fi Online: ne amul on. Theoverall process isconsiderably more sensi- orms, providing adirect write spo on Refreshment Break &Ra ng andquan vity, and proteomics isawindowto thefunc- fi on I cantly addto thevalue ofCTC enumera fi Tri-Conference.com c point in fi ffl on city for prostate cancer. Theadvent of plex panelofmetabolomic markers for on andcharacteriza icts oneoutofninethoseover the fi ned addi fying sequencecharacterisfying Sponsored by me. ents withmetasta ves. on ofNucleicAcid on inCTC counts ispredic- on andcharacteriza on asaresult ofapoptosis. on onal metabolites that are al technology inthepur- on to

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Email: al to radical factor factor cal c cancer. ﬁ on of cs by cs by ning [email protected] ents ﬁ ous ous on ed 3:05 Refreshment Break intheExhibitHall most important factor intheimplementa 2:25 PlenaryKeynote Presenta 1:45 IceCream Refreshment Break intheExhibitHall The OhioState University Carlo M.Croce, M.D., Professor, Internal Medicine,CollegeofMedicine&PublicHealth, Cancer 8:35 Keynote Presenta Causes andConsequencesofmicroRNA Dysregula Genera 3:50 EnablingPersonalized Medicine:TheGrowing Role ofNext German Pihan,M.D., Dpt.ofPathology, Beth IsraelDeaconess Medical Center 3:45 Chairperson’s Remarks 2:15 PlenaryKeynote Introduc 1:05 LuncheonPresenta approach. a more complete understanding ofhumandiseasefrom asystems biology Dalia Cohen,Ph.D., CSO, Rose 8:30 AMChairperson’s OpeningRemarks Ready accessto thegenome sequenceofapa German Pihan,M.D., Department ofPathology, Beth IsraelDeaconess Medical Center our approach for diagnos 5:50 CloseofDay 4:20 Keynote Presenta ing may have thegreatest andmost immediate impact. state-of-the-art aswell astheclinical areas where massive parallel sequenc- make personalized medicinesoonareality. Here Ireview thetechnological recent development ofmassive parallel sequencingtechnologies promise to HLA Typing by HighResolu el associated withan applied thistechnology to understand viral dynamicsat thesequencelev- extreme, rapid shi Henry A. Erlich, A. Ph.D.,Henry VP, Discovery Research, HumanGene High-throughput sequencingpla Deaconess Medical Center Pathologist, Department ofPathology, Medical School,Beth BIDMCandHarvard Israel rare HIV-1 variants anddocumen Cardiomyopathy Diagnos 4:50 Next Genera diac sarcomere structure andfunc Hypertrophic cardiomyopathy isanautosomal dominant disorder ofcar- Director for Advanced Technology, ARUPLaboratories Karl V. Voelkerding, M.D., Associate Professor ofPathology, UniversityofUtah andMedical Ramy Arnaout,M.D., DPhil.,Associate Director, Clinical Microbiology, BIDMCSta 5:20 HIVDynamicsTaught by Sequencing forms, anddeepsequencingprovided adetailed viewoftherapid evolu- ing approach for theanalysis ofthismul HCM. We have currently designedandtested anext genera ons. At least 16genes withover 450muta onary impactofselec FRIDAY, FEBRUARY 5 TRANSLATING INFORMATION TO PRACTICAL USE NEXTGEN SEQUENCING AS A CLINICAL TOOL MICRORNA DIAGNOSTICS FOR CANCER: on Sequencing PLENARY KEYNOTE SESSION  s inpopula on Sequencingfor Hypertrophic

viral treatment failure. Failure was associated with Fax: on. a Genomics,Inc. c applica on on 781-972-5425 on II cs on Technology on frequencies toward speci orms provide anapproach for detec ng more fullyquasispeciesdiversity. We (Sponsorship OpportunityAvailable) on on leadingto mul on on. (See Page 26for Details) on ofpersonalized medicine.The -gene disorder andare re ons have beenimplicated in cs, Roche MolecularSystems, Inc. ent isarguably thesingle ple cardiac condi- MED001689 on in on sequenc- ﬁ c resistant ﬀ

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9 DIAGNOSTICS CHANNEL es liva, vaginal secre future for thesepla expressed miRNAs was iden sequen-omics landscape andiden solving theseissuesandchallenges. Thissessionwillprovide acomprehensive viewofthe the problem inthenear-term butover thelong-term willbringmore resources to bearin 10:05 LNA™basedUniversal RT na further technological innova enomics andpersonalized medicine. fl We performed the microRNA PCRSystem. AnewGenera pression indried,forensically relevant biological and otherarrays) have experienced atechnological revolu DNA sequencingandother“-omics”pla Hospital David Sugarbaker, M.D., Chief, Thoracic Surgery, BrighamandWomen’s Challenges 9:30 LivinginaSequen-omicsWorld: Data Integra sis inpa polymorphisms can poten quan processing advances have outpacedtheabilityto fullyintegrate theresul over thepreceding decade that shows nosignofslowing.However, data storage and microRNA pathway can in evolu Referred to asthemicromanagers ofgene expression, microRNAs are NIH Prasun J. Mishra,Ph.D., Laboratory ofCancer Biology&Gene Medicine 9:05 microRNA Polymorphisms andtheFuture ofPersonalized opportuni cogenes ortumorsuppressor genes, thesesmallRNAsprovide important control orare controlled bydysregula dence onthedysregulated expression ofmicroRNA genes, which inturn powerful tools to study thebiologyofdiseases.Detec factors targe factors microRNA loci,by epigene of mechanisms,includingdele During thepast several years ithasbecome clearthat altera Jacob UlrikFog, Ph.D., Scien haps all,humanmalignancies.Thesealtera pression ofmicroRNA genes contribute to thepathogenesis ofmost, per- Usi ngaLocked NucleicAcid(LNA™)basedmiRNAdetec A/S Joanne B.Weidhaas, Ph.D., Assistant Professor, Therapeu plasma orserum.TheuseoftheLNA™basesaddscri and sensi Since microRNAs are globalregulators, smallaberra development intheclinical anddiagnos miRNAs inclinical para for therapy. cancer risk, can actasbiomarkers ofoutcome, andmay befuture targets morphisms have turnedoutto besome ofthestrongest biomarkers of lular homeostasis andenhancecancer risk.MicroRNA binding site poly- disrupt theircoding sequencesortheirtarget bindingsites can alter cel- we have developed ahighthroughput QPCR system for detec High Throughput QPCRPla Molecular Diagnos Op and Plasma Polymorphisms asBiomarkers 11:00 TheOnco-SNPandCancerRisk:microRNA BindingSite 10:20 Co uid using50pgoftotal miRNApro RNA. ma ve approach to body mized for Development microRNA based es ofdata into biologically meaningfulandpredic on, preven onarily conserved smallnon-coding RNAs.Polymorphisms inthe ents andhasprofound implica ff vity crea es for development offuture microRNA basedtherapies. ee Break ng speci on, andcure ofhumandiseases,most notably cancer. Inaddi orms to posi ons andmenstrual blood).Apanelofninedi fi ng amore robust system for more rapid assay rst miRNAome-wideevalua fi ffi

c Assays onClinical FFPEandBloodSerum fi Online: c microRNAs. Sincemalignant cellsshowdepen- c Manager, Diagnos n-embedded on willcon fl uid iden fl ally improve diagnosis,treatment andprogno- c silencingorbydysregula uence gene regula vely a vely fi fy thekeyfy issuesthat willneedto beaddressed inthe ed that permit theiden ons, ampli Tri-Conference.com orm ff ect humanhealth. orms (e.g., mRNA, microRNA, mRNA, CGH, exon,orms (e.g., SNP, nue to drive downcosts whichwillexacerbate fi ca ssue aswell asbloodderived on ofmul on for forensic casework. c assay development. c ProductDevelopment Division,Exiqon fi ons inthe fi on ling provides apromising alter- ca ons can becaused byavariety c Radiology, Yale University ons ormuta on andare emerging as fl on ofspeci cs, Na uids (blood,semen,sa- on inthroughput andscale ve modelsto applyto risk ple protein coding on- ons suchasSNPs that Sponsored by fi

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tute, [email protected] ally on, on by highlypenetrant rare variants whichare mul gaining for theheterogeneity ofcommon disease whichislikely caused biomarkers for predic the rare variants isunprecedented, makingthemidealcandidates asnovel is discovering andusinganewstandard of“causa the metrics that de and drugdiscovery applica capable ofgenera phenotypes into genotypic di structural varia to personalize medicine.Beyond SNPs, there isasurprisingabundanceof much ofitoccurringdenovo. Recent studies have revealed “causa paving theway for anext genera GENOMETRICA -anovel, universal technology pla rare CNVassocia yan hasdeveloped novel biomarkers for detec End-Point PCRandDNASequencing propriate assay systems. ongoing clinical studies, andourprogress inthedevelopment ofap- of pa Jim Chinitz,ChiefExecu 2:05 CNVStudiesinAu ogy andmedicine. Historically, pa la together andconsidered homogeneous according to phenotype.Popu- in capillaries andoperates asamoduleofthehardware/so quan We present anovel, lowcost instrument whichperforms microbead-based Vera Gor Vera employs ultra fast, singlephoton sensi on complicated andexpensive neurological andradio-imaging Currently noFDA cleared labtests exist for TBIandthediagnosisisbased Müller,Uwe R. Ph.D., VP, ProductDevelopment, Banyan Biomarkers, Inc. 2:35 Blood-basedDiagnos 1:35 ALowCost Instrument for Microbead-Based Quan Kewal K.Jain,M.D., Professor, CEO, JainPharmaBiotech Relevance for Personalized Medicine 1:05 Introduc Kewal K.Jain,M.D., Professor, CEO, JainPharmaBiotech 1:00 Chairperson’s Remarks 3:05 CloseofConference 11:30 Role ofmicroRNA BasedPro pa Carcinoma ofunknownprimary(CUP)isaheterogenous diseasewhere a Oncology, M.D. AndersonCancer Center Gauri Varadhachary, M.D., Associate Professor ofMedicine,Department ofG.I.Medical Origin for Carcinoma ofUnknownPrimary e cancers, accurate iden important to theappropriate care ofthesepa pro in pa 12:00 PMLuncheonPresenta Lunch onYour Own ff ec on Diagnos fi ent presents withmetastases withoutaniden ling isanemerging tool to helpwithiden ve cytotoxic andtargeted therapies emerge for addi ents withCUP. ta ents within2hours ofinjury. We willpresent theresults ofour fi nkel, Ph.D., Associate Professor, Stony BrookUniversity ve end-point PCRandCEbasedDNAsequencing.Theinstrument INNOVATIVE DIAGNOSTICS FOR ents having acommon complex diseasehave beenlumped cs (“PDx”) hasledaparadigm shi cs (“PDx”) on inthegenome called CopyNumberVariants (CNVs), on to theTechnologies andtheirSigni PERSONALIZED MEDICINE ons inau  ve O ng thecommon phenotype.Itisnecessaryto dissect fi ne thelevel ofclinical relevance (i.e.oddsra

ffi Fax: ve tests andbeacons for molecularpathways. PDx fi cer, Popula ca sm andotherNeurological Disorders ons. on ofCUPsubtypeswillbecome increasingly 781-972-5425 ff sm, schizophrenia andALS.Inthesemodels, erences to understand common diseaseand cs ofBrain Injuries on ofdiagnos on Diagnos on (Sponsorship OpportunityAvailable) fi ve detec ve ling inDetermining Tissueof cs, Inc. ents. MicroRNA expression fi -genic andindependently c, personalized medicine ca on of on ofTBIintheblood fi orm for molecularbiol- where apprecia ve” biomarkers andis able primary. Asmore on of fl uorescent signals MED001690 fi ssue oforigin cance/ onal known tests. Ban- tests. ware suite suite ware ta os) of ve ve on is or ve” ve”

10 DIAGNOSTICS CHANNEL Cambridge Healthtech Institute’s Third Annual Cancer Molecular Markers

WEDNESDAY, FEBRUARY 3 3:20 Populaon Based in vivo Biomarker Discovery Using Engineered Human Tumors Min Wu, Ph.D., Principal Scienst, Translaonal Research, AVEO 7:00 AM Registraon and Morning Coffee Pharmaceucals, Inc. 8:00 Plenary Keynote Session (See Page 26 for Details) Human tumor populaons exhibit significant inter-tumor variaon, where each tumor harbors a unique set of genec alteraons that impact prognosis and response 9:40 Grand Opening Refreshment Break in the Exhibit Hall to treatment. Unfortunately, this variaon results in low response rates in the clinic KEYNOTE PRESENTATIONS and creates significant challenges for drugs to meet regulatory endpoints. Cancer cell line based xenogras have tradionally been the preclinical model of choice to assess the efficacy of clinical compounds, however, such models exhibit inherent ar- 11:00 Chairperson’s Remarks facts due to long term in vitro culture, and are unable to adequately capture natu- Michael Liebman, Ph.D., Managing Director, Strategic Medicine, Inc. ral variaon seen in human tumor populaons. To address this challenge, we have 11:10 Personalizing Medicine: It’s a System-Based Challenge created a populaon based tumor model system based on Human-in-Mouse ssue Franklyn G. Prendergast, M.D., Ph.D., Professor, Pharmacology, transgenic human tumors that feature naturally occurring tumor variaon akin to Biochemistry & Molecular Biology, Director, Center for Personalized that observed in human tumor populaons. Each tumor of the populaon has been comprehensively characterized at the RNA and DNA level, and the populaon has Medicine, Mayo Clinic been adapted to conduct quantave efficacy studies of an-cancer agents and com- 11:40 Genomic Strategies for Personalized Cancer Treatment binaons, enabling correlaons between response and the genec context of the Joseph R. Nevins, Ph.D., Barbara Levine Professor, Duke University tumors. This plaorm enables us to idenfy and validate biomarkers of therapeuc We have made use of expression profiling to develop signatures of oncogenic path- response in an in vivo human tumor system. way deregulaon that can then be used to profile the state of these pathways within populaons of tumors. In addion, the pathway signatures also link the paerns of 3:50 Sponsored Presentaon (Sponsorship Opportunity Available) pathway acvaon with therapeucs since we have shown that predicng the ac- 4:20 Recep on in the Exhibit Hall (Sponsorship Available) vaon of a pathway also predicts sensivity to drugs that target the pathway. We have extended this concept to develop more refined signatures that can dissect the 5:20 BREAK-OUT DISCUSSIONS in the Exhibit Hall complexies of many of the known signaling pathways, providing a more precise capacity to probe the acvity or deregulaon of the pathway and linking to a broader What is the Forecast for Epigenecs and microRNA? array of therapeucs. Moderator: Enal Razvi, Ph.D., System Biosciences SBI Status of the microRNA and epigenecs markets 12:10 PM Panel: Impact of Personalized Medicine on The research market for microRNA and epigenecs: growth and evoluon Oncology Drugs and Treatment Diagnoscs and therapeucs development based on microRNA and epigenec signatures Addional Panelist: Mike Boswood, President, CEO, Thomson Reuters Current challenges and opportunies in these spaces 12:40 Luncheon Presentaon (Sponsorship Opportunity Available) or Challenges to Whole Genome Sequencing Lunch on Your Own Moderator: s Ng, Ph.D., Assistant Professor, Genomic Medicine, J 1:45 Dessert in the Exhibit Hall Craig Venter Instute Challenges to whole-genome sequencing PERSONALIZING THERAPY: TISSUE BIOMARKERS Idenfying de novo and re-current mutaons in cancer Addressing tumor heterogeneity 2:15 Chairperson’s Remarks How can we move from characterizing gene variaon to ulizing the whole Tracey Colpis, Ph.D., Manager, Abbo Molecular genome 2:20 Relang Biomarkers to Efficacy: The Efficacy Curve Sequencing tumors rather than tumor cell lines DIAGNOSTICS CHANNEL Tracey Colpis, Ph.D., Manager, Abbo Molecular The Complex genomic structure of tumor cells: de novo assembly or strategy A method of predicng response in a subgroup defined by a biomarker will be dis- to detect structural variants cussed and demonstrated using data from therapeuc trials involving EGFR inhibi- Are there Cancers of Unknown Primary Tumors? tors in lung, colon, and breast cancer. Biomarkers that aid in selecng subgroups of Moderator: Dalia Cohen, Ph.D., Chief Scienfic Officer, Rosea paents of response were analyzed and compared. Striking similaries between the Genomics, Inc. different cancers, therapies, and subgroups reveals a relaonship between biomark- Debate over cancers of unknown primary tumors (CUP) ers and efficacy, which is visualized in the efficacy curve. Methods to detect CUPs 2:50 Network and Pathway Analysis of a Novel 3D Breast Consequences of detecon of primary Carcinoma Model by Both Digital Gene Expression (DGE) and Gene Signatures in Cancer Diagnoscs Whole Genome Array Analyses Co-Moderators: Gary Geiss, Ph.D., Principal Scienst, NanoString Technologies and David Kern, MBA, Director, MyRaQa Ray Mangly, Ph.D., Associate Professor, Pharmacology, Wayne State Developing a gene signature University Validaon of gene signatures We have developed a tractable, in vitro model of ductal carcinoma in situ (DCIS) Regulatory consideraons for gene signature diagnoscs based on 3D overlay culture in reconstuted basement membrane (rBM). We have applied and cross-validated whole genome microarray (Affymetrix) and digital gene Systems Chemical Biology-A New Paradigm expression (DGE) analyses (Illumina/Solexa) to explore the networks and pathways Moderator: Ally Perlina, Senior Applicaon Scienst, GeneGo Inc. that underlie DCIS. DGE analysis revealed a broad range of products that are tran- Ulizing tools for drug reposioning scribed outside of standard (NCBI 36.3) genes models. These transcripts suggest Understanding side effects truncaons and changes in an-sense driven regulatory pathways in DCIS. Understanding the mechanisms of acon for drugs Networkable compounds 6:20 Close of Day CANCER &

11 Online: Tri-Conference.com Email: [email protected] Fax: 781-972-5425  MED001691 THURSDAY, FEBRUARY 4 cancer aggressivity. In addi on to sarcosine we have deĮ ned addi onal metabolites that are being characterized in the context of prostate cancer progression. Our long term objec ve is to deĮ ne a mul plex panel of metabolomic markers for prostate PERSONALIZING THERAPY: SERUM BIOMARKERS cancer progression. 8:25 AM Chairperson’s Remarks 11:45 CirculaƟ ng Tumor Cells: From EnumeraƟ on to Josip Blonder, M.D., Sr Research ScienƟ st; Head, QuanƟ taƟ ve Proteomics, NCI Frederick Comprehensive CharacterizaƟ on Nicholas C. Dracopoli, Ph.D., VP, Biomarkers, Centocor R&D, Inc., 8:30 Personalized Oncoproteomics for Cancer Biomarker Johnson & Johnson Discovery: ApplicaƟ on to Renal Cell Carcinoma Circula ng tumor cells (CTC) are very rare and consist of about 1 in 108 or 109 cells Josip Blonder, M.D., Sr Research ScienƟ st; Head, QuanƟ taƟ ve in blood drawn from some pa ents with metasta c cancer. Enumera on of CTCs has Proteomics, NCI Frederick been shown to have prognos c value for pa ents with metasta c breast prostate and Discovery of diagnos c, therapeu c and prognos c markers is central to personal- colorectal cancer, and is being evaluated to determine if a treatment-related reduc- ized treatment of cancer. Thus, proteomic approaches capable of characterizing the on in CTC counts is predic ve of therapeu c response. Comprehensive characteriza- pa ent’s tumor phenotype using clinically relevant specimens are cri cally needed. on (DNA, RNA and protein) of CTCs will signiĮ cantly add to the value of CTC enu- A method that relies on ssue-directed oncoproteomics is described and applied for mera on tests, and enable serial monitoring of CTCs for molecular changes occurring cancer biomarker discovery in the plasma of a pa ent diagnosed with renal cell car- during disease progression and as a response to therapy. This presenta on will review cinoma. new approaches for the isola on and characteriza on of CTCs, and discuss how CTC- derived biomarkers will become a cri cal factor in the development of personalized 9:00 Protein QuanƟ Į caƟ on Through Targeted Mass treatment strategies in oncology. Spectrometry: The Way Out of Biomarker Purgatory? 12:10 PM Progress in Noninvasive Detec on of Nucleic Acid Steven A. Carr, Ph.D., Director, Proteomics, The Broad InsƟ tute of MIT Ɵ and Harvard Biomarkers Immunoassays are widely used to measure protein biomarkers in pa- Charles R. Cantor, Ph.D., CSO, Sequenom, Inc. Procedures have been developed to enhance the collec on of RNA and DNA frag- ent blood, but useful an body reagents do not exist for the vast ma- ments that enter the peripheral circula on as a result of apoptosis. These include jority of proteins. We are addressing this serious barrier by developing op mized methods of recovering small fragments, amplifying them and then detect- targeted assay methods employing mass spectrometry to screen and ing and quan fying sequence characteris cs by nucleic acid mass spectrometry. The quan fy low abundance proteins in plasma. This presenta on will fo- methods show promise in noninvasive prenatal diagnos cs, tumor detec on and cus on the latest developments and applica ons of these technologies. characteriza on, and infec ous disease agent iden Į ca on. The overall process is considerably more sensi ve and precise than commonly used alterna ves. 9:30 Mass Spec for Prostate Biomarkers, Assessing Aggressive 12:35 Luncheon PresentaƟ on (Sponsorship Opportunity Available) or vs. Non-Aggressive Prostate Cancer Jianfeng Xu, M.D., Ph.D., Professor, Wake Forest University School of Lunch on Your Own Medicine 1:45 Ice Cream Refreshment Break in the Exhibit Hall Three types of prostate cancer related gene c variants have been found from genome-wide associa on studies, including those associated with overall prostate can- PLENARY KEYNOTE SESSION cer risk, aggressive prostate cancer risk, and higher baseline PSA levels. These gene c variants may have poten al clinical u lity. However, further studies are needed to 2:15 Plenary Keynote IntroducƟ on assess their clinical validity and clinical u lity. 2:25 Plenary Keynote PresentaƟ on (See Page 26 for Details)

10:00 A Novel Tool for Non-Invasive Disease Sponsored by 3:05 Refreshment Break in the Exhibit Hall DetecƟ on NEXTGEN SEQUENCING AS A CLINICAL TOOL Jack Leonard, Ph.D., Vice President of Technology CommercializaƟ on, febit Inc. 3:45 Chairperson’s Remarks We developed a novel non-invasive diagnos c assay based on microRNAs. 3:50 Enabling Personalized Medicine: The Growing Role of Our Biomarker Signature assay has shown an outstanding performance for Next GeneraƟ on Sequencing the integra ve detec on of a broad panel of diseases and is well suited for German Pihan, M.D., Department of Pathology, Beth Israel Deaconess high sample throughput at low cost since for each test less than one min- Medical Center ute hands-on me is required. Moreover, our approach stands-out by high

Ready access to the genome sequence of a pa ent is arguably the single most impor- DIAGNOSTICS CHANNEL reproducibility and sensi vity while test-to-test varia ons are minimal. tant factor in the implementa on of personalized medicine. The recent development of massive parallel sequencing technologies promise to make personalized medicine soon 10:30 Poster CompeƟ Ɵ on Refreshment Break & Raŋ es in the a reality. Here I review the technological state-of-the-art as well as the clinical areas where massive parallel sequencing may have the greatest and most immediate impact. Exhibit Hall 11:20 Metabolomic Analysis of Prostate Cancer Progression 4:20 Keynote PresentaƟ on Arun Sreekumar, Ph.D., Molecular Oncology Program, Medical College HLA Typing by High ResoluƟ on Technology of Georgia Henry A. Erlich, Ph.D., VP, Discovery Research, Human GeneƟ cs, Roche Prostate cancer is the second most common cause of cancer-related death in men in the United States and aﬄ icts one out of nine of those over the age of 65. There is an Molecular Systems, Inc. urgent need to develop biomarkers that can supplement PSA and increase its speciĮ c- 4:50 Next GeneraƟ on Sequencing for Hypertrophic ity for prostate cancer. The advent of high throughput proĮ ling strategies has allowed Cardiomyopathy DiagnosƟ cs scien sts to look at global changes in genome, proteome and metabolome. Metabo- lomics, unlike genomics and proteomics, is a young science that has the poten al Karl V. Voelkerding, M.D., Associate Professor of Pathology, University to radically alter the future of healthcare, drug discovery, and drug delivery. It is the of Utah and Medical Director for Advanced Technology, ARUP single best window into the cellular state discovered to date. Like the other omics- Laboratories style sciences, where genomics is best understood as deĮ ning the gene c poten al, Hypertrophic cardiomyopathy is an autosomal dominant disorder of cardiac sarcom- transcriptomics is a window into the future (desired) direc on of the cellular ac vity, ere structure and func on leading to mul ple cardiac condi ons. At least 16 genes and proteomics is a window to the func onal poten al of the cell; metabolomics, the with over 450 muta ons have been implicated in HCM. We have currently designed omics science of metabolism, is the only window into the current and actual state of and tested a next genera on sequencing approach for the analysis of this mul -gene the cell (or by extension, organism) at a speciĮ c point in me. disorder and are reĮ ning our approach for diagnos c applica on. Recently we have proĮ led the metabolome in prostate cancer progression using a combina on of GC and LC chromatography. Our study quan Į ed the levels of >1000

metabolites across 250 biospecimens. Results of the proĮ ling study revealed elevated CANCER & levels of sarcosine or N-methyl glycine to be associated with advanced prostate cancer. Importantly components of sarcosine pathway were found to regulate prostate

Online: Tri-Conference.com Email: [email protected] Fax: 781-972-5425 12  MED001692 Since microRNAs are globalregulators, smallaberra Joanne B.Weidhaas, Ph.D., Assistant Professor, Yale University Polymorphisms asBiomarkers 11:00 TheOnco-SNP andCancerRisk:microRNA BindingSite 10:20 Co diseases. Detec fl conserved smallnon-coding RNAs.Polymorphisms inthemicroRNA pathway can in- or bydysregula come, andmay befuture targets for therapy. be someofthestrongest biomarkers ofcancer risk,can actasbiomarkers ofout- and enhancecancer risk.MicroRNA bindingsite polymorphismshave turnedoutto their coding sequencesortheirtarget bindingsites can alter cellularhomeostasis issues that willneedto beaddressed inthefuture for thesepla 10:05 Sponsored Presenta a which inturncontrol orare controlled bydysregula lignant cellsshowdependenceonthedysregulated expression ofmicroRNA genes, During thepast several years ithasbecome clearthat altera Medicine &PublicHealth,TheOhioState University Carlo M.Croce,M.D., Professor, Internal Medicine,Collegeof Cancer DNA sequencingandother“-omics”pla Women’s Hospital David Sugarbaker, M.D., Chief, ThoracicSurgery, Brighamand and Challenges provide acomprehensive viewofthesequen-omicslandscape andiden bring more resources to bearinsolvingtheseissuesandchallenges. Thissessionwill costs which willexacerbate theproblem inthenear-term butover thelong-term will cancer. Inaddi tuni oncogenes ortumorsuppressor genes, thesesmallRNAsprovide important oppor- nancies. Thesealtera microRNA genes contribute to thepathogenesis ofmost, perhapsall,humanmalig- Dalia Cohen,Ph.D., ChiefScien 8:30 AMChairperson’s OpeningRemarks BIDMC Sta Ramy Arnaout,M.D., DPhil.,Associate Director, ClinicalMicrobiology, 5:20 HIVDynamicsTaught by Sequencing High-throughput sequencingpla Medical School,BethIsraelDeaconessHarvard Medical Center Referred to asthemicromanagers ofgene expression, microRNAs are evolu NIH 9:30 LivinginaSequen-omicsWorld: Data Integra to apply to risk es risk to apply to SNP, andotherarrays) have experienced atechnological revolu 5:50 CloseofDay 8:35 Keynote Presenta treatment andprognosis inpa Causes andConsequencesofmicroRNA Dysregula sul storage andprocessing advances have outpacedtheabilityto fullyintegrate there- and scale over thepreceding decade that shows nosignofslowing.However, data Prasun J. Mishra,Ph.D., Laboratory ofCancerBiology&Gene Personalized Medicine 9:05 microRNA Polymorphisms andtheFuture of nology to understand viral dynamicsat thesequencelevel associated withan pharmacogenomics andpersonalized medicine. variants anddocumen treatment failure. Failure was associated withextreme, rapid shi view oftherapid evolu quencies toward speci TRANSLATING INFORMATION TO PRACTICAL USE ff uence gene regula ons, ampli FRIDAY, FEBRUARY 5 ect humanhealth. ng massive quan es for development offuture microRNA basedtherapies. MICRORNA DIAGNOSTICS FOR CANCER: ff fi ff ca ee Break Pathologist, Department ofPathology, BIDMCand on ofmicroRNA-polymorphisms can poten ons ormuta on oftranscrip on, furthertechnological innova ma on andare emerging aspowerful tools to study thebiologyof ons can becaused byavariety ofmechanisms, includingdele- fi es ofdata into biologically meaningfulandpredic c resistant forms, anddeepsequencingprovided adetailed ng more fullyquasispeciesdiversity. We appliedthistech- on, preven onary impactofselec

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ng rare HIV-1 Email: c silencing ve models models ve fy the key cs, NCI, fi onarily elds of on fre- viral [email protected] vely vely an through mul dis Tumors are madeupofaheterogeneous mixture ofcelltypesanditispossiblethat Contribu 1:35 Understanding Tumor CellHeterogeneity inNSCLC: Erica L.Jackson, Ph.D., Scien and have found that thesecellsare preferen rence. We have isolated andcharacterized CSCsfrom avariety ofmajortumortypes the decreased e One ofthemechanismsbywhichresidual diseasebecome chemo-resistant isvia Muhammad Al-Hajj,Ph.D., Director, Stem CellDiscovery Unit,GlaxoSmithKline Resistance 2:05 ABCTransporters’ Role inCancerStem Cell Drug cells from bulktumorcells. Regenera iden pies. Aspartofoure cells andtheirvalue astherapeu and metasta Jus Cancer Stem CellHypothesis 2:35 New VisionsofCancerTherapy through thePrismof casse contain acellularhierarchy ofdi heterogeneity inmost cancers. Thecancer stem cellhypothesis positsthat tumors ers inthecontext ofcancer stem cellsisparamount andtheirapplica up-regulated insomecancer stem cells.Theclinical relevance oftheABCtransport- The failure ofconven Cancer stem cellsare thought to mediate tumorini of thesepa fi the development ofmore speci be presents withmetastases withoutaniden we’ll discussthe role oftwo speci giogenic poten 1:05 ImpactofAn 1:00 Chairperson’s Remarks Underlying Pathways Essen 11:30 Role ofmicroRNA BasedPro Tim Hoey, Ph.D., VP, Cancer Biology, OncoMed Pharmaceu toxic andtargeted therapies emerge for addi 3:05 CloseofConference cancer therapies. of signalingpathways andmoleculartargets incancer stem cellsmay yieldimproved Carcinoma (CUP)isaheterogeneous ofunknownprimary diseasewhere apa Department ofG.I.MedicalOncology, M.D. AndersonCancerCenter Gauri Varadhachary, M.D., Associate Professor ofMedicine, Tissue ofOriginfor Carcinoma ofUnknownPrimary 12:00 PMLuncheonPresenta studies have demonstrated that cancer stem cellsdisplay therapeu Available) TARGETING CANCER STEM CELLS ca onally de er understanding oftherole individualtransporters play inthemechanismand n D. Lathia,Ph.D., Research Associate, Department ofStem CellBiologyand nct cellpopula on ofCUPsubtypeswillbecome increasingly important to theappropriate care fi e (ABC)proteins that are variably expressed bythetumorcellsandtend to be -DLL4 an ca ve Medicine,LernerResearch Ins orLunchonYour Own on of fi ned cellpopula ents. MicroRNA expression pro ple mechanismsincludingareduc c cancers hasmany causes butoneappears to bethestriking cellular ons to Resistance andRelapse al, andapropensity towards invasion/metastasis, theiden ffi body that blocks Notch signaling.An ssue oforigininpa ciency ofchemo-therapeu ff ons play uniqueroles intumorigenesis. We are studying func- ort to develop novel agents targe onal therapies to fundamentally alter thesurvival ofadvanced  bodies onCancerStem Cells:Discovering

Fax: ons to determine what dis fi fi ff c targets. 781-972-5425 c inhibitors withminimalo c transporters inpancrea eren st, Genentech, Inc. ents withCUP. al to CancerStem CellBiology a on tute, Cleveland ClinicFounda on andtumorpropaga fi fi able primary. Asmore e (Sponsorship Opportunity ling isanemerging tool to helpwith cs through theac onal knowncancers, accurate iden fi ally resistant to many current thera- ling inDetermining on inCSCfrequency. a -DLL4 inhibitstumorgrowth ng CSCs,we have developed on, metastasis, andrecur- nguishes chemo-resistant c andcolon cancer stem ff target e target on ofATP-binding on poten c resistance, an- MED001693 ff cals, Inc. on ec ff on requires requires on ects. Here ve cyto- ve fi ca al. As ent on -

13 DIAGNOSTICS CHANNEL Cambridge Healthtech Institute’s Seventh Annual The Senior Level Chemist Event Mastering Medicinal Chemistry WEDNESDAY, FEBRUARY 3 Tranzyme Pharma, Inc. The discovery and development path leading from a small molecule macrocyclic screening library to the novel ghrelin receptor agonist, ulimorelin (TZP-101), a Phase 7:00 AM Registraon and Morning Coffee III product for the treatment of GI hypo-molity disorders, will be presented in detail. The specific aributes of the medicinal chemistry technology (MATCH™) that enabled 8:00 Plenary Keynote Presentaons (See Page 26 for Details) the rapid progression of these efforts from hit-to-lead-to-clinic will be outlined. 9:40 Grand Opening Refreshment Break in the Exhibit Hall 3:20 Execuve Panel KEYNOTE PRESENTATIONS: RECENT APPROVALS Medicinal Chemistry Drivers: Innovaon, Technology, AND CLINICAL CANDIDATES Efficiency and Luck Moderator: Graeme Semple, Ph.D., Vice President, Discovery Chemistry, Arena 11:00 Chairperson’s Remarks Pharmaceucals, Inc. Hing Sham, Ph.D., Senior Vice President, Chemical Sciences, Elan Pharmaceucals Panelists: Michael Henning, Ph.D., Vice Director & Head, Discovery Technologies, F. Hoffmann-La 11:10 Recent Approval: Mozobil from Development to Roche Ltd Approval Kenneth A. Savin, Ph.D., Manager, Global External Research & Development, Eli Lilly & Co. Renato Skerlj, Ph.D., VP, Medicinal Chemistry, Genzyme Corporaon Drug and Gary W. Small, M.D., Parlow-Solomon Professor on Aging, Professor of Psychiatry & Biomaterial R&D Biobehavioral Sciences, Director, UCLA Center on Aging Mozobil™ (plerixafor injecon), a first in class small molecule antagonist of the chemokine receptor CXCR4, was granted markeng approval by the FDA in Decem- 3:50 Thermodynamic Sponsored by ber 2008 and indicated for the mobilizaon of hematopoiec stem cells to the blood- Contribuons of Water Molecules to Ligand-Receptor Binding stream for collecon and subsequent autologous transplantaon in paents with Christopher Higgs, Ph.D., MRSC, Senior Applicaons Scienst, Schrödinger, LLC non-Hodgkin’s lymphoma and mulple myeloma. Interpreng structure-acvity data is oen challenging even with the availability of crystal structures. The role of solvent thermodynamics in protein bind- 11:40 Discovery of a State-Dependent Cav2.2 Blocker for the ing sites is oen overlooked but can be important in explaining experimental Treatment of Chronic Pain data. Here, we present a stascal thermodynamic approach to the treatment Sco B. Hoyt, Ph.D., Research Fellow, Department of Basic Chemistry, Merck Research Laboratories of binding site water molecules and show that hydraon site displacement pat- Voltage-gated Cav2.2 calcium channels control the release of neurotransmier at terns can be used to explain SAR trends, ligand selecvity, and site-directed presynapc terminals, and thus play a crical role in pain signaling. The state-inde- mutagenesis. Applicaons of the method to the A2A adenosine receptor, PDZ pendent Cav2.2 blocker ziconode, a pepde that must be administered via intrathe- domains, a broad range of kinases, and other systems of pharmaceucal inter- cal injecon, has demonstrated clinical efficacy in the treatment of severe chronic est will be discussed. pain. State-dependent Cav2.2 blockers may likewise provide clinical pain relief without adversely affecng other nerve funcons. 4:20 Recepon in the Exhibit Hall (Sponsorship Available) 12:10 PM Discovery of Lorcaserin: A Selecve 5-HT2C Agonist 5:20 BREAK-OUT DISCUSSIONS in the Exhibit Hall for the Treatment of Obesity 6:20 Close of Day Brian Smith, Ph.D., Director, Medicinal Chemistry, Arena Pharmaceucals, Inc. Compelling evidence suggests that drugs which acvate the 5-HT2C receptor cause weight loss and thus have potenal as an-obesity agents. Because serotonin elicits THURSDAY, FEBRUARY 4 a number of biological responses through modulaon of other 5HT receptors, selecvity has been a crical challenge. This presentaon outlines events, challenges and achievements that led to the discovery and development of lorcaserin. FRAGMENTINSPIRED AND STRUCTUREGUIDED MEDICINAL CHEMISTRY 12:40 Luncheon Presentaon (Sponsorship Opportunity Available) or Lunch on Your Own 8:25 AM Chairperson’s Remarks Charles Reynolds, Ph.D., Research Fellow, Computer Aided Drug Discovery, Johnson & 1:45 Dessert in the Exhibit Hall Johnson

CHEMISTRY CHANNEL CHEMISTRY CASE STUDIES FROM CHEMISTRY TO THE CLINIC 8:30 Drug Discovery Facilitated by Fragment Screening Efforts Michael Henning, Ph.D., Vice Director & Head, Discovery Technologies, F. Hoffmann-La 2:15 Chairperson’s Remarks Roche Ltd Rapid gain in potency of compounds by structure based drug design together with the 2:20 Discovery of SCH 530348 – A Thrombin Receptor high sensivity of biophysical methods like Surface Plasmon Resonance (SPR) enable the Antagonist with Potent Anplatelet Effects use of fragment molecules to guide drug discovery efforts. The lecture will review the fragment screening e orts at Roche and analyze bene ts and challenges of the approach Samuel Chackalamannil, Ph.D., Disnguished Research Fellow, Discovery Research, ff fi from these experiences. Drug targets like β-secretase or chymase are used as case stud- Schering-Plough Research Instute ies. SCH 530348, a himbacine-based thrombin receptor antagonist (TRA), is currently un- dergoing Phase-III clinical studies for acute coronary syndrome and secondary prevenon of cardiovascular events in high risk paents. In a Phase-II clinical study in PCI 9:00 Synthesis, in vitro and in vivo Evaluaon of PI3K Inhibitors paents, SCH 530348 showed no stascally significant increase in major or minor Mahew Burger, Ph.D., Research Invesgator II, Global Discovery Chemistry, Novars bleeding when added to standard of care, and showed a non-stascally significant Instutes for Biomedical Research reducon in major adverse cardiac events, including periprocedural myocardial in- Phosphoinoside-3-Kinase (PI3K) is an important oncology target due to the deregu- farcon. laon of its signaling pathway in a wide variety of human cancers. A lead series from a combinatorial library was idenfied that potently inhibits PI3K. Using SBDD the lead 2:50 Macrocycle-based Drug Discovery: The Ulimorelin Story series was opmized to yield PI3K inhibitors with suitable PK properes to establish Helmut Thomas, Ph.D., Senior Vice President, Research & Preclinical Development, a PK/PD-efficacy relaonship in a mouse A2780 xenogra model.

14 Online: Tri-Conference.com Email: [email protected] Fax: 781-972-5425  MED001694 9:20 Design, Synthesis and Opmizaon 2-aminoquinazolines as PLENARY KEYNOTE SESSION PDK1 Inhibitors Savithri Ramurthy, Ph.D., Research Invesgator, GDC/ONC, Novars Instutes of 2:15 Plenary Keynote Introducon Biomedical Research, Emeryville, CA Herein, we describe the use of iterave structure-guided design to discover two sets of 2:25 Plenary Keynote Presentaon (See Page 26 for Details) leads from the series Quinazolines as PDK1 inhibitors. The in vitro and in vivo acvity of potent PDK1 inhibitors will be discussed along with the medicinal chemistry approaches 3:05 Refreshment Break in the Exhibit Hall ulized to opmize the chemical series for kinase selecvity, eﬄux, and hERG. TARGETS IN HOT PURSUIT I

9:40 Presentaon Sponsored by www.evotec.com 3:45 Chairperson’s Remarks Fragment Based Drug Discovery at Evotec - Applicaon to the Thomas Högberg, Ph.D., Vice President, 7TM Pharma, Hørsholm, Denmark idenﬁcaon of BACE and PDE10a inhibitors 3:50 Redesign of Arylsulfonamide Gamma Secretase James Madden, Ph.D., Principal Scienst, Evotec (UK) Limited Inhibitors to Achieve Novelty and High in vivo Acvity Evotec’s FBDD plaorm (EVOluonTM) integrates orthogonal screening technologies, Andrei Konradi, Ph.D., Senior Director, Medicinal Chemistry, Elan Pharmaceucals namely; biochemical, NMR and SPR to test fragments in a high throughput, highly The transformaon of known arylsulfonamide Gamma Secretase Inhibitors (GSIs) sensive mode. Evotec has successfully applied this technology in a number of pro- into Elan’s arylsulfonyl pyrazolopiperidine GSIs, using small molecule modeling and grams. This presentaon will describe 2 case studies where EVOluonTM has been pharmacophore hypotheses, will be described. Exploraon of analogs to maximize used to discover BACE and PDE10a inhibitors. in vitro potency, metabolic stability, pharmacokinecs, and in vivo acvity will be presented. Several novel synthec methods to prepare the target compounds will 10:00 Luncheon Presentaon Sponsored by be described.

The Role of Medicinal Chemistry in Translaonal Research 4:20 Discovery of the Nedd-8 Acvang Enzyme Inhibitor Josep Prous, Jr., Ph.D., MBA, Vice President and Chief Scienfic Officer, Thomson Reuters Healthcare & Science MLN4924 The biomedical community has embraced the translaonal research approach to find- Steve Langston, Ph.D., Senior Scienst, Millennium Pharmaceucals, the Takeda ing beer and safer medicines. However, to meet the promises of this approach, re- Oncology Company searchers need a knowledge-based methodology in which the constuent disciplines The ubiquin-proteasome system (UPS) is responsible for the regulated degradaon share data appropriately. This talk will show how medicinal chemistry provides a bridge of intracellular proteins with important roles in cellular funcon including cancer cell between early biology findings and clinical applicaon of new molecular enes. growth and survival. NEDD8-acvang enzyme (NAE) is an essenal component UPS that regulates degradaon of a subset of proteins upstream of the proteasome. The discovery of MLN4924, a first in class inhibitor of NAE, will be presented. 10:30 Poster Compeon Refreshment Break & Raffles in the Exhibit Hall 4:50 Discovery of the Hedgehog Inhibitor GDC-0449 11:30 Compung Specific Residue-ligand Interacon Energies Michael Koehler, Ph.D., Scienst, Discovery Chemistry, Genentech, Inc. using Quantum Mechanical Energy Decomposion: A Tool for 5:20 Exploraon of SAR and opmizaon of in silico derived Guiding Drug Design CRTH2 antagonists Charles Reynolds, Ph.D., Research Fellow, Computer Aided Drug Discovery, Johnson & Thomas Högberg, Ph.D., Vice President, 7TM Pharma, Hørsholm, Denmark Johnson Several chemical series of antagonists for the PGD2 receptor CRTH2 were idenfied Quantum methods are just beginning to find wider applicaon in drug discovery. We from a small focused library originang from a knowledge-based process using phys- have used pair-wise decomposion of protein-ligand interacon energies, computed icogenec relaonships and ligand informaon. The elucidaon of SAR by synthesis using the DivCon program, to analyze the interacons that drive potency in a series of smaller libraries and design of specific target structures led to idenficaon of of protein kinase B inhibitors. These computed interacon energies were used to novel drugable chemotypes. derive two heat maps: (1) an interacon energy map and (2) an SAR map. These interacon energies, and resulng maps, provide detailed informaon not otherwise available for idenfying the residues in an acve site that are most crical for ligand 5:50 Close of Day binding. FRIDAY, FEBRUARY 5 12:00 PM The Emperor’s New Crystal: Examples of X-ray Bloopers; a Cauonary Tale STRATEGIES FOR EFFECTIVE MEDICINAL Edward Kesicki, Ph.D., Director, Small Molecule Drug Discovery, Infecous Disease CHEMISTRY Research Instute I will give examples of “solved” structures in which incorrect ligands were fied to the electron density map of a kinase co-crystal, a result of a typographical error in the pa- 8:30 AM Chairperson’s Opening Remarks perwork sent to the crystallographer. In addion, I will show a class of selecve PI3 Kenneth A. Savin, Ph.D., Manager, Global External Research & Development, Eli Lilly & Co. kinase inhibitors that would have never been discovered using known X-ray crystal structures. 8:35 A Paradigm Change in the Applicaon of ADME

Resources to Early Lead Generaon Acvies CHANNEL CHEMISTRY 12:30 Luncheon Presentaon (Sponsorship Opportunity Available) or Kenneth A. Savin, Ph.D., Manager, Global External Research & Development, Eli Lilly & Co. Lunch on Your Own With the advent of new technology and processes, there are new possibilies for the applicaon of ADME resources to projects at earlier phases in a project’s life-cycle. 1:45 Ice Cream Refreshment Break in the Exhibit Hall We have been able to change the way ADME supports projects with the hope of improving our ability to come to decision points earlier in lead generaon.

9:05 Understanding Structure-Toxicity Relaonships as a Guide to Safer Drugs John C.L. Erve, Ph.D., DABT, Principal Research Scienst II, Drug Safety Metabolism, Wyeth Research Reacve metabolites are a concern due to their potenal role in drug toxicity. De- spite our understanding of bioacvaon pathways and ability to minimize reacve metabolite formaon, toxicity remains a cause of failure during drug development. This talk will review structure-toxicity relaonships so that this knowledge can beneﬁt drug discovery.

Online: Tri-Conference.com Email: [email protected] Fax: 781-972-5425 15  MED001695 9:35 Biotransformaon to Enable Chemistry SAR 2:05 From a Concept towards a First-In-Class Drug for a Douglas Spracklin, Ph.D., Director, Pharmacokinecs, Dynamics and Metabolism, Pfizer, Inc. Human Amyloid Disease Biotransformaon science has evolved well beyond tradional struc- Jeffery W. Kelly, Ph.D., Chair, Molecular and Experimental Medicine, Lita Annenberg tural elucidaon of metabolites. Contemporary biotransformaon Hazen Professor of Chemistry, The Skaggs Instute, The Scripps Research Instute data is especially well suited to aid chemistry SAR development, i.e., The seminar will cover the twenty-one year adventure from our inial idenfying metabolic hot spots, non-obvious metabolic pathways, po- demonstraon that rate-liming transthyren tetramer dissociaon tenal reacve metabolites, etc. Knowledge around these aributes and monomer misfolding was sufficient for transthyren amyloido- can be extremely helpful in priorizing chemical series and selecng genesis linked to neurodegeneraon, to the recent clinical trial re- individual molecules for development. sults of FoldRx demonstrang that a transthyren kinec stabilizer halts neurodegeneraon in familial amyloid polyneuropathy. This is 10:05 Sponsored Presentaon (Sponsorship Opportunity Available) the first pharmacologic evidence supporng the validity of the amyloid hypothesis. 10:20 Coffee Break IMAGING AS AN EXCITING TOOL IN DRUG 2:35 DNA-Programmed Chemistry Approach to Macrocyclic DISCOVERY Lead Compounds Nick Terre, Ph.D., CSO, Ensemble Discovery Corp. Chair: Michael A. Letavic, Research Fellow, Neuroscience, Johnson & Johnson Pharmaceucal R&D DNA-programmed chemistry is an integrated plaorm for the synthesis and screening of macrocycles that interact with protein-protein 11:00 Imaging Drug Acon in the Human Brain drug discovery targets such as the oncology target, BCL-XL. We have Joanna Fowler, Ph.D., Senior Chemist, Brookhaven Naonal Laboratory also discovered a series of macrocycles that compevely antagonize Radiotracers and drug molecules labeled with short-lived positron the interacon of TNFα with TNF receptors in both biochemical and eming isotopes such as carbon-11 (t1/2: 20.4 min), fluorine-18 cell-based assays, and that also have an-inflammatory acvity in (t1/2: 110 min) or nitrogen-13 (t1/2: 10 min) are unique scienfic vivo. tools for measuring biochemical transformaons and drug pharmacokinecs and pharmacodynamics in the living human and animal 3:05 Close of Conference body.

11:30 Image Analysis Consideraons for Preclinical, in vivo Medical Imaging Ma Silva, Head, Imaging Sciences, Millennium, The Takeda Oncology Company With the expanding role of preclinical and translaonal imaging in drug research, it is necessary to consider not only study design and imaging modality but also visualizaon and image quanﬁcaon. This presentaon will review the role of imaging technologies and show examples of experiments and image analysis procedures, including kinec analysis of dynamic contrast-enhanced MRI and bone topology analysis from 3D CT data.

12:00 PM Luncheon Presentaon (Sponsorship Opportunity Available) or Lunch on Your Own TARGETS IN HOT PURSUIT II

1:00 Chairperson’s Remarks Nick Terre, Ph.D., Chief Scienﬁc Oﬃcer, Ensemble Discovery Corp. 1:05 Modulators and Consequences of Hsp90 Regulaon by Small Molecules Brian S. J. Blagg, Ph.D., Associate Professor of Medicinal Chemistry, The University of Kansas; Winner of the 2009 David W. Robertson Award in Medicinal Chemistry PRIME POSTER POSITION The 90 kDa heat shock proteins (Hsp90) are molecular chaperones re- • Your Poster will be available to over 3,000 delegates quired for the refolding of denatured proteins and the maturaon of • You’ll automatically be entered into our Poster Competition, where two nascent polypepdes into their biologically acve, three-dimensional winners will receive $500 structures. In fact, numerous proteins represented in all six hallmarks • $50 off your registration fee CHANNEL CHEMISTRY of cancer are dependent upon Hsp90 for conformaonal maturaon. • Your Poster Abstract will be published on the conference proceedings link Innovave approaches toward C-terminal inhibion of Hsp90 will be • Your research will be seen by leaders from pharmaceutical, biotech, discussed. academic and government institute • Posters will be on display for two full days 1:35 Design and Synthesis of RDEA119, a Potent and Orally Bioavailable MEK Inhibitor Jean-Michel Vernier, Ph.D., VP, Chemistry Discovery, Ardea Biosciences Reserve Your Space By January 13! This presentaon will discuss the design, synthesis and structural-acvity relaonship that led to the discovery of RDEA119, a novel highly potent and selecve MEK inhibitor currently in Phase I clinical trial. RDEA119 is being developed under a global license agreement with Bayer HealthCare.

Online: Tri-Conference.com Email: [email protected] Fax: 781-972-5425 16  MED001696 Cambridge Healthtech Institute’s Second Annual Adopting R&D Informatics Systems Data Management, Integration & Knowledge Management

WEDNESDAY, FEBRUARY 3 1:10pm Luncheon Presentaon II Sponsored by Empowering Sciensts with Hypothesis-Driven Data Exploraon 7:00 AM Registraon and Morning Coffee Jian Wang, Ph.D., CEO, BioFors Inc. 8:00 Plenary Keynote Session (See Page 26 for Details) A significant boleneck on producvity in translaonal research is the inability for sciensts to directly interrogate data by themselves. We 9:40 Grand Opening Refreshment Break in the Exhibit Hall present a novel soluon & case study to demonstrate how, with the EXECUTIVE STRATEGIESINTEGRATED R&D right tools, sciensts can be more self-sufficient, efficient and produc- INFORMATICS ve, while enabling informacs specialists to focus more on higher value contribuons instead of mundane ad hoc data manipulaons. 11:00 Chairperson’s Remarks David M. Sedlock, Ph.D., Senior Director, Research & Development Systems, Millennium, 1:45 Dessert in the Exhibit Hall The Takeda Oncology Company 2:15 Chairperson’s Remarks 11:10 Enterprise Scienfic Workflow Environment Drives David M. Sedlock, Ph.D., Senior Director, Research & Development Systems, Millennium, Innovaon The Takeda Oncology Company Daniel J. Chin, Ph.D., Senior Principal Research Scienst, Roche Palo Alto 2:20 Integrated Informacs Systems for R&D Most informacs investments increase the efficiency of drug discovery. Vaibhav A. Narayan, Ph.D., Senior Director, Integrave Neurosciences & Biomarkers, The introducon of an enterprise-wide scienfic workflow plaorm en- Johnson & Johnson Pharmaceuical Research & Development ables research informacs organizaons to shi their efforts towards 2:50 Execuve Panel with Q&A scienfic innovaon. Researchers apply scienfic workflows for in silico experimentaon and exploraon, leading to scienfic hypotheses and Are We Integrang the Right Data: Extending Beyond discoveries. Enterprise environments enable researchers to share and Laboratory Data to Decisions Impacng Project Success evolve their scienfic workflows, further increasing research produc- Moderator: David M. Sedlock, Ph.D., Senior Director, Research & Development Systems, vity. Examples of scienfic workflows and the seng required to run Millennium, The Takeda Oncology Company scienfic workflow plaorms effecvely in pharmaceucal research Data Aggregaon vs. Data Integraon will be discussed. Data Management vs. Knowledge Management Integrang Data Management Systems across Mulple Sites Effecve Data Integraon in Translaonal Medicine Research 11:40 The Pistoia Alliance, Inc.–A Construct for Role of Open Source Technology in Systems Design Precompeve Collaboraon Is There a ‘Cloud’ in Your Future? Chris Waller, Ph.D., Senior Director, Precompeve Collaboraons, Research, Barriers Sharing Research and Development Data Development & Medical Informacs, Worldwide Technology, Pfizer, Inc. Process Management vs. Technology Consideraons when Deploying New Systems The Pistoia Alliance has been established to provide the foundaon of Panelists: data standards, ontologies and associated web-services to enable the Susie Stephens, Director, Biomedical Informacs, Pharmaceucal Research & Pharmaceucal discovery workflow through common business terms, Development, Johnson & Johnson relaonships and processes. Current progress, learnings and how com- Daniel J. Chin, Ph.D., Senior Principal Research Scienst, Roche Palo Alto Chris Waller, Ph.D., Senior Director, Precompeve Collaboraons, Research, panies, academics and others can parcipate in this approach will be Development & Medical Informacs, Worldwide Technology, Pfizer, Inc. described. Giles M. Day, Senior Director, BBC Informacs, Pfizer, Inc.

12:10 PM Recent Strategies with Cloud, Wikis, Ontologies 3:50 Presentaon Sponsored by and Open Source Data Standards Workflow based Enterprise Informacs Giles M. Day, Senior Director, BBC Informacs, Pfizer, Inc. Frank Brown, Ph.D., Vice President & Chief Scienfic Officer, Accelrys Accelrys is producing a new generaon of Enterprise Informacs sys- 12:40 Luncheon Presentaon I Sponsored by *By aending Microso’s Luncheon presentaon you are opng to receive further tems for chemical, biological and image data registraon and mining. communicaons from Microso. The new generaon features workflow driven applicaon logic and Personalized Medicine: The Missing Pieces business rules, clients that leverage the latest collaborave environ- Jim Karkanias, Senior Director of Applied Research & Technology, Microso Health ments such as Microso SharePoint, and novel storage techniques to Soluons Group

INFORMATICS CHANNEL INFORMATICS handle the complexity and diversity of today’s data types. Discoveries to make personalized medicine a reality depend on leveraging the “open universe” of life sciences data. To assist invesgators 4:05 Sponsored Presentaon (Sponsorship Opportunity Available) with ad hoc quesons, hypothesis generaon, and validaon, invesgators must describe and verify systems about the life science uni- 4:20 Recepon in the Exhibit Hall (Sponsorship Available) verse. This session will introduce a strongly typed repository of linked 5:20 BREAK-OUT DISCUSSIONS in the Exhibit Hall data that makes it possible to conceive and deliver game-changing therapies. 6:20 Close of Day

17 Online: Tri-Conference.com Email: [email protected] Fax: 781-972-5425  MED001697 THURSDAY, FEBRUARY 4 12:00 PM Applicaon of Translaonal Informacs in Tailored Therapeucs Susie Stephens, Director, Biomedical Informacs, Pharmaceucal Research & DATA INFORMATION AND KNOWLEDGE Development, Johnson & Johnson MANAGEMENT 12:30 Managing Research Porolios – Sponsored by 8:25 AM Chairperson’s Remarks Can IT help you? Thomas P. Hill, Principal, The Leverage Innovaon Group; former Director, Learning and Arvindh Balakrishnan, Vice President Life Sciences, Oracle Joe Duncan, Chief Execuve Officer, Teranode Knowledge Management, Genentech Research departments work on hundreds of thousands of molecules, 8:30 SparkLab 360 - The Complete System Sponsored by markers and targets. How can we simplify informaon gathering across for Managing your Lab Research your scienfic community in order make consistent porolio manage- Roi Paz, Chief Execuve Officer, SparkLix Bio-IT ment decisions? This talk will focus on using Oracle technologies like Electronic lab notebooks (ELNs) and laboratory informaon manage- semanc web; and how porolios can be reflected in best in class port- ment systems (LIMS) are essenal tools in lab management. SparkLix folio management tools. – an Innovaon Award recipient from the Associaon for Laboratory Automaon – developed SparkLab 360, which integrates features from 1:00 Luncheon Presentaon Sponsored by ELNs and LIMS in one user-friendly system. This presentaon focuses on how SparkLab enables design, planning, execuon and analysis of SOA-based IT Framework for Life Science Research the enre research process. David A. Medina, Worldwide Life Science and Pharma Segment Execuve, Hewle-Packard Company This presentaon will present a collaborave plaorm for bioinformat- 9:00 Knowledge for Strategic Advantage: Accelerang the ics used in bioresearch based on a scalable, standards-based, easy- R&D Cycle to-deploy, SOA-based architecture. This plaorm will facilitate the Thomas P. Hill, Principal, The Leverage Innovaon Group; former Director, Learning and integra on of intra-organiza onal research e orts and enable inter-o Knowledge Management, Genentech ff This presentaon focuses on key elements of knowledge leveraged for rganizaonal R&D collaboraon. The plaorm will also enable pharma strategic advantage in the Life Sciences industry, the key challenge of R&D organizaons to effecvely access disparate data sources and fa- how to accelerate the R&D process by using collaborave informacs cilitate the cross-analysis of genomic, proteomic and clinical data. technologies and an examinaon of specific scienfic business soluon implementaons for results. In addion, the key features of a robust 1:45 Ice Cream Refreshment Break collaborave scienfic business soluon will be idenfied. in the Exhibit Hall PLENARY KEYNOTE SESSION 9:30 ASAP-Emphasizing Muldimensional Drug Discovery W. Patrick Walters, Ph.D., Senior Research Fellow & Group Head, Computaonal Drug 2:15 Plenary Keynote Introducon Discovery Technologies, Vertex Pharmaceucals, Inc. ASAP is new soware plaorm designed to help drug discovery teams 2:25 Plenary Keynote Presentaon (See Page 26 for Details) make beer decisions. ASAP provides an intuive overview of the data 3:05 Refreshment Break in the Exhibit Hall that also allows sciensts to easily “drill down” and examine the details of parcular experiments. A combinaon of “filters” and heat maps INTEGRATIVE DATA MANAGEMENT THROUGH allows teams to focus on aspects of the data while remaining aware of CLOUD, WIKIS, ONTOLOGIES & SEMANTIC WEB the “big picture”. CONT.

10:00 RISe Architecture – Architectural Sponsored by 3:45 Chairperson’s Remarks Aspects of an Integrated Research Informacs Plaorm Susie Stephens, Director, Biomedical Informacs, Pharmaceucal Research & Ajay Shah, Ph.D., MBA, PMP, Director of Research Informacs, Elan Development, Johnson & Johnson Pharmaceucals Inc. 3:50 Data Integraon–What’s in it for Me? Elan and Infosys are building a research data integraon plaorm called RISe Randal Chen, Ph.D., Director, Research Informacs, Amgen, South San Francisco (Research Informacs System at élan). RISe enables registraon of biological enes, their inventory, associated workflows, and integraon with chemical data 4:20 Semanc Web and Cloud Compung for Integrave Data ulizing a workflow driven, mul-ered, SOA based architecture built on the Management and Analysis Infrastructure Microso.NET plaorm. To maximize extensibility in a research environment, Jonas S. Almeida, Ph.D., Abell-Hanger Disnguished Professor, Bioinformacs and the database combines Enty-Aribute-Value design for flexible definion of Computaonal Biology, University of Texas, M.D. Anderson Cancer Center enes, efficiency-priorized OLTP schema for inventory management, and a The systems nature of biological processes and the scalability of cloud com- planned ETL interface to a semanc database. pung created an irresisble trend towards distribuon of both the data 10:30 Poster Compeon Refreshment Break & Raffles in the and the ecosystem of applicaons that analyze them. Accordingly, at M.D. Anderson Cancer Center we are exploring the use of semanc web to render Exhibit Hall distributed infrastructure manageable and its contents safely discoverable. 11:30 Agile Soware Development: Meeng the Rapidly An open-source prototype was developed, see s3db.org. CHANNEL INFORMATICS Changing Needs in Drug Discovery Man-Ling Lee, Ph.D., Senior Program Analyst, Discovery Chemistry, Small Molecule Drug 4:50 Collaborave Drug Discovery Humanitarian and Discovery, Genentech, Inc. Commercial Researcher Network Case Studies To support drug discovery project teams meeng their melines, the Com- Barry A. Bunin, Ph.D., Chief Execuve Officer & President, Collaborave Drug Discovery pChem/ChemInformacs Group at Genentech has established an agile ap- (CDD), Inc. proach to sasfy the changing needs. The basis are two flexible soware Collaborave Drug Discovery (CDD) has created a community based plaorms, AEREA (Aestel Scienfic Informaon) and Pipeline Pilot (Accel- plaorm that combines tradional drug discovery informacs with rys). The pres entaon will discuss the implementaon and impact of two Web2.0 features to provide the best of both worlds. Recent efforts applicaons: one for lead selecon and one for DMPK data analysis. to selecvely arrest TB in the dormant phase working with leading researchers and mining SAR data will be presented. A global community INTEGRATIVE DATA MANAGEMENT THROUGH of leading TB researchers supported by leading foundaons will be re- CLOUD, WIKIS, ONTOLOGIES & SEMANTIC WEB viewed. Advances from communies working together on commercial

Online: Tri-Conference.com Email: [email protected] Fax: 781-972-5425 18  MED001698 drug discovery bringing together industry, foundaons, and academia to taking the types of data SMRT technologies will generate to get at will also be emphasized. predicve models of disease that can be used to drive the idenficaon and validaon of drug targets and biomarkers. 5:05 Panel: Drug Discovery Collaboraons in 2010 Moderator: Barry A. Bunin, Ph.D., Chief Execuve Officer & President, Collaborave 10:05 Sponsored Presentaon (Sponsorship Opportunity Available) Drug Discovery (CDD), Inc. Biopharmaceucal – CRO collaboraons 10:20 Coffee Break Virtual Pharmaceucal collaboraons PPP (academic-industry-foundaon) collaboraons 11:00 Profiling Paents to Drive Biomarker Development N. R. Nirmala, Ph.D., Director, Biomarker Analysis and Informacs Unit, Translaonal Panelists: Sciences, Novars Instutes of Biomedical Research Vaibhav A. Narayan, Ph.D., Senior Director, Integrave Neurosciences & Biomarkers, Johnson & Johnson Pharmaceuical Research & Development Gene expression profiling is one of the key ways in which a genome- Uli Schmitz, Ph.D., Director, Structural Chemistry, Gilead wide view of a paent’s response to drug treatment can be obtained. Adam Renslo, Ph.D., Associate Director, Chemistry, Adjunct Assistant Professor, Such a molecular level view can provide strategies for customized Pharmaceucal Chemistry therapies in many contexts. In this talk, the oppportunies and chal- 5:50 Close of Day lenges that this technology presents will be discussed with a couple of case studies. Extension of this approach to other technologies will FRIDAY, FEBRUARY 5 also be presented in the context of biomarker development. 11:30 Panel: Informacs at R&D Interphases TRANSLATIONAL INFORMATICS Moderator: Pearl S. Huang, Ph.D., Integrator, Oncology Franchise, Research & Early HOW FAR HAVE WE COME? Development, Merck & Co. Linking clinical outcome with molecular data: filling the gaps 8:30 AM Chairperson’s Opening Remarks Capturing uniform clinical language for outcomes Pearl S. Huang, Ph.D., Integrator, Oncology Franchise, Research & Early Development, Compable and user- friendly data systems—can one size fit all? Merck & Co. Disease cohorts-how many, how big, what is acceptable quality 8:35 Implemenng a Translaonal Biomarker Strategy to 12:00 PM Luncheon Presentaon (Sponsorship Opportunity Reduce Arion in Drug Development Available) or Lunch on Your Own Irina Antonijevic, M.D., Ph.D., Director, Translaonal Research, Biological Research, INTEGRATED GENOMIC, BIOLOGY, AND IMAGE DATA Lundbeck Research, Inc. USA Early efforts towards the discovery of molecular biomarkers for CNS 1:00 Chairperson’s Remarks disorders are encouraging. However, confirmaon, and ulmately Ajay Shah, Ph.D., MBA, PMP, Director, Research Informacs, Elan Pharmaceucals, Inc. validaon of such biomarkers is dependent on state-of-the-art bioinformacs analyses as well as assay development. These prerequisites 1:05 Image Analysis Consideraons for Pre-clinical, in vivo will ensure idenficaon of biomarkers that are reproducible and Medical Imaging hence of clinical relevance. Matt Silva, Head, Imaging Sciences, Millennium, The Takeda Oncology Company With the expanding role of preclinical and translaonal imaging in drug re- 9:05 High Content Mining of Disease Biomarkers search, it is necessary to consider not only study design and imaging mo- Jake Chen, Ph.D., Assistant Professor, Informacs & Computer Science, Indiana dality but also visualizaon and image quanficaon. This presentaon will University School of Informacs; Director, Indiana Center for Systems Biology and review the role of imaging technologies and show examples of experiments Personalized Medicine, Indiana University-Purdue University Indianapolis; Founder, and image analysis procedures, including kinec analysis of dynamic con- MedeoLinx, Inc. trast-enhanced MRI and bone topology analysis from 3D CT data. To facilitate the interpretaon of raw Omics data into detailed disease-specific knowledge of candidate biomarkers, we developed a 1:35 RISe Prowler: A Semanc Web Approach to Integrang “high-content biomarker mining” soware system. The system can External and In-house Biology and Chemistry Informaon help manage and correlate molecular funcons, molecular connec- Ajay Shah, Ph.D., MBA, PMP, Director, Research Informacs, Elan Pharmaceucals, Inc. vity, biological pathways, and literature informaon. Its applicaon into the current biomarker development process will help improve 2:05 Development of a Registraon System for Biologics in a the success rate and quality of candidate biomarkers. Collaborave Special Interest Group Jeremy Packer, Ph.D., Head, Bioinformacs, Abbo 9:35 Single Molecule Real Time Biology: New technologies 2:35 Development of Combinaon Therapies for Mulple Enabling a More Complete Characterizaon of Disease Sclerosis Using Systems Level Informacs Biology Frederic S. Young, Ph.D., Chief Scienst, Vicus Therapeucs Eric Schadt, Ph.D., Chief Scienfic Officer, Pacific Biosciences We start with a mullevel systems physiology model that combines While there has been an explosion of technologies that enable more metabolomic analysis with integrated physiological analysis. The comprehensive characterizaons of complex biological processes like model is used to define a set of systems informac features of on- common human diseases, we are sll unable to glimpse a large enough togeny, phylogeny, homeostasis, and repair that disnguishes the CHANNEL INFORMATICS fracon of the biology of these systems to build models that are pre- disease state from homeostasis. We describe our use of this systems dicve enough to achieve clinical ulity. However, with a new wave of informac signature as an algorithm for the development of combi- technologies on the horizon, providing for the capability to examine naon therapies for mulple sclerosis. the acvity of single molecules real me, we will soon be capable of generang the right scale and diversity of data (DNA sequence, RNA 3:05 Close of Conference sequence, real me monitoring of mRNA translaon, full characterizaons of base modificaons in genomes and transcriptomes) at low cost to dramacally enhance the construcon of models for common human diseases that achieve clinical ulity. I will cover the single molecule real me (SMRT) technologies from Pacific Biosciences and how these technologies will revoluonize our ability to characterize living systems, and then present a number of integrave biology approaches

Online: Tri-Conference.com Email: [email protected] Fax: 781-972-5425 19  MED001699 Cambridge Healthtech Institute’s Sixth Annual Cancer Proﬁling and Pathways: Next Sequence in the War against Cancer

WEDNESDAY, FEBRUARY 3 ized with barcode sequences corresponding to each shRNA in the pool. Here, we present a novel pooled shRNA screening approach for iden- 7:00 AM Registraon and Morning Coffee fying regulators of endogenous gene expression. Epithelial cell adhesion molecule (EpCAM), a cell surface receptor that is highly expressed 8:00 Plenary Keynote Session (See Page 26 for Details) 9:40 Grand Opening Refreshment Break in the Exhibit Hall in a variety of tumorigenic cells, promotes cell proliferaon and tumor KEYNOTE PRESENTATIONS formaon via transcriponal acvaon of mitogenic genes. Thus, Ep- CAM represents a target for the development of new cancer therapeu- 11:00 Chairperson’s Remarks cs. We performed a whole-genome pooled shRNA screen to idenfy Michael Liebman, Ph.D., Managing Director, Strategic Medicine, Inc. novel regulators of EpCAM expression. OVCAR8 cells were transduced 11:10 Personalizing Medicine: It’s a System-Based Challenge with the Thermo Fisher Scienfic Decode™ RNAi Viral shRNAmir Pools. Franklyn G. Prendergast, M.D., Ph.D., Professor, Pharmacology, Following puromycin selecon, we used magnec-acvated cell sort- Biochemistry & Molecular Biology, Director, Center for Personalized ing (MACS) to separate cells on the basis of EpCAM protein expression. Medicine, Mayo Clinic Genomic DNA was isolated from EpCAM+ and EpCAM- cells and the 11:40 Genomic Strategies for Personalized Cancer Treatment shRNAs enriched or depleted within each populaon were idenfied Joseph R. Nevins, Ph.D., Barbara Levine Professor, Breast Cancer using custom microarrays. The genes targeted by shRNAs enriched in Genomics, Center for Applied Genomics & Technology, Duke University Perhaps the major challenge in developing more effecve therapeuc strategies for the EpCAM- cells were idenfied as candidate regulators of EpCAM expres- treatment of most major cancers is confronng the heterogeneity of the disease, rec- sion. This work demonstrates that pooled shRNA libraries may be used ognizing that most cancers are not one disease but mulple disorders with disnct un- in a variety of novel screening strategies, including the idenficaon of derlying mechanisms. We have made use of expression profiling to develop signatures novel regulators of tumor-associated genes. of oncogenic pathway deregulaon that can then be used to profile the state of these pathways within populaons of tumors. In addion, the pathway signatures also link the paerns of pathway acvaon with therapeucs since we have shown that predicng 1:45 Dessert in the Exhibit Hall the acvaon of a pathway also predicts sensivity to drugs that target the pathway. 1:45 Dessert in the Exhibit Hall We have extended this concept to develop more refined signatures that can dissect the WORKING BACKWARDS IN CANCER: complexies of many of the known signaling pathways, providing a more precise capacity to probe the acvity or deregulaon of the pathway and linking to a broader array FROM THE CLINIC TO DISCOVERY of therapeucs. We suggest that this approach can provide a framework for an overall strategy towards the development of personalized treatment opons for the individual 2:15 Chairperson’s Remarks paent, including strategies for personalized combinaon therapy. Michael Liebman, Ph.D., Managing Director, Strategic Medicine, Inc. 2:20 Using Drug-Induced Feedback Loops to Idenfy Indicaons 12:10 PM Panel: Impact of Personalized Medicine on Oncology Drugs and Combinaon Partners and Treatment Donald Bergstrom, Director, Experimental Medicine Oncology, Merck Addional Panelist: Mike Boswood, President, CEO, Thomson Reuters James W. Waers, Associate Director, Molecular Profiling Oncology, How could informaon about differences of individuals become a way to im- Merck prove drug discovery rather than reduce ROI? Treatment with molecular targeted agents can result in compensatory feedback regula- How can you change it to bring in new knowledge? on as cells respond to inhibion of signaling pathways. We will present clinical evi- How do you change percepon of culture? dence that treatment with a small molecule inhibitor of gamma secretase results in Is it purely technology that is needed to solve the problem? pathway modulaon and compensatory feedback, and describe pre-clinical experiments designed to leverage this concept for drug response predicon. 12:40 Luncheon Presentaon I Sponsored by Digital, Mulplexed Measurements of up to 550 mRNAs in 2:50 Genomic Soluons to Diagnosc and Prognosc Clinical

INFORMATICS CHANNEL INFORMATICS Clinically Relevant Sample Types Using the nCounter™ Analysis Predicons in Head and Neck Cancer System Geoffrey Childs, Ph.D., Professor of Pathology, Albert Einstein College of Gary Geiss, Ph.D., Principal Scienst, NanoString Technologies, Inc. Medicine Richard V. Smith, M.D., FACS, Professor, Vice-Chair, Department of The nCounter Analysis System ulizes color-coded molecular barcodes to digitally count Otorhinolaryngology-Head and Neck Surgery, Montefiore Medical nucleic acid molecules. The system can mulplex 550 targets without enzymac ma- Center and Albert Einstein College of Medicine nipulaon. A variety of input samples have been tested, including FFPE and crude cell The strategy our group employs is to ulize the data obtained from high throughput as- lysates. The technology has been applied to mul-gene expression cancer signatures says including gene expression measurements of mRNA and miRNA, global methylaon and mRNA fusion-transcripts. Products to measure miRNAs and dsDNA are under de- paerns of DNA and global proteomics to develop prognosc and diagnosc signatures velopment. to predict outcome, local regional recurrence presence/absence of lymph node metastasis at inial diagnosis and to predict opmal treatment opons. 1:10-1:40 A Novel Genome-Wide Screening Sponsored by 3:20 Moving Research Closer to the Bedside, in vitro and in vivo Applicaon Using Pooled Viral miRNA-adapted Analyses with Primary Tumors shRNA (shRNAmir) Libraries Fred Poordad, M.D., Chief of Hepatology, Liver Disease and Transplant Kae Jansen Spayd, Ph.D., Research Scienst, Thermo Fisher Scienfic Center, Cedars-Sinai Medical Center, Xin Wei Wang, Ph.D., Senior Pooled shRNA libraries are powerful genec discovery tools that al- Invesgator Head, Liver Carcinogenesis Secon, Laboratory of Human low for high-throughput screening of the enre genome in a cost-ef- Carcinogenesis, Naonal Cancer Instute, NIH, and Michael R. Briggs, fecve and less labor-intensive manner. Unlike arrayed shRNA library Ph.D., Senior Director, Biology, Vertex Pharmaceucals, Inc. approaches requiring many mul-well plates, screens using lenviral The incidence of Primary Liver Cancer is increasing in the west and constutes a tremen- shRNA pools can be performed in a single ssue culture plate. Clonal dous burden on world health as the third leading cause of cancer deaths worldwide. The 5 year survival rate is a dismal 11 %, due in large part to late diagnosis and limited populaons of cells expressing an individual shRNA become enriched treatment opons. The eology of this devastang disease as well as current and pro- or depleted in this mixed populaon in response to a selecve pres- posed new therapies wil be discussed. Steps to beer diagnose and strafy paents for targeted therapy will be considered as a new and excing phase of cancer research. CANCER & sure. Genes required for cell enrichment or cell depleon can then be deconvoluted by next-generaon sequencing or microarrays hybrid- Finally, a move toward more relevant research will be presented as an hypothesis that

20 Online: Tri-Conference.com Email: [email protected] Fax: 781-972-5425  MED001700 will be tested in the coming years as more new and current therapies are compared and deregulaon will help developing reﬁned therapeuc strategies to treat these complex contrasted to current best pracce. diseases.

4:05 How do you go from Pathways to Sponsored by 9:30 Expression Based Paent Straficaon for Cancer Clinical Outcomes? Prognoscs Aris Persidis, Ph.D., President, Biovista, Inc. Peter J. van der Spek, Ph.D., Department of Bioinformacs, Erasmus MC - In drug discovery and development what what really counts is the Medical Faculty clinical outcome, the Benefit/Risk of the drug within the context of its pathway or Systems biology approaches in life sciences and health open new perspecves for mechanism of acon (MoA). Biovista screens the MoA of any drug or target against paent straficaon. Microarray and next-generaon sequencing techniques provide the MoA of 8,000 indicaons and 12,000 adverse events (AEs). This is simultaneous, vast volumes of data and detailed informaon about natural variants vs. mutaons in unbiased indicaon discovery and AE profiling, and it is unique. It helps to bridge the the underlying molecular eology of the disease. Knowledge bases allow sciensts to gap from molecular pathway to clinical outcome in a single step. Case studies in cancer place their research results in perspecve. and other diseases will be described. 10:00 Funconal Analysis of Omics Data in Cancer Sponsored by 4:35 Recepon in the Exhibit Hall (Sponsorship Available) Yuri Nikolsky, Ph.D., CEO, GeneGo, Inc. 5:20 BREAK-OUT DISCUSSIONS in the Exhibit Hall High-throughput assays are indispensable in studies of complex human diseases. Numerous methods have What is the Forecast for Epigenecs and microRNA? been developed for Omics data analysis. I will describe GeneGo techniques of path- Moderator: Enal Razvi, Ph.D., System Biosciences SBI way, network, and interactome analysis, and summarize recent results of our collab- Status of the microRNA and epigenecs markets orave studies on breast, colorectal, pancreac, and glial cancers. I will also describe funconal analysis of predicve gene signatures developed for FDA’s MAQCII project. The research market for microRNA and epigenecs: growth and evoluon Diagnoscs and therapeucs development based on microRNA and epigenec 10:15 Cellular Target Profiling and Sponsored by Biotechnologies GmbH signatures Current challenges and opportunies in these spaces Quantave Phosphoproteomics Reveal Insight Challenges to Whole Genome Sequencing into a Drug’s Efficiency and Cellular Mode of Acon Moderator: s Ng, Ph.D., Assistant Professor, Genomic Medicine, J Craig Jua Fritz, Ph.D., Head of Technology, Kinaxo Biotechnologies Venter Instute System-wide approaches integrang drug target idenficaon and global phosphoproteomics depict a compound’s cellular mode of acon and its impact on signal trans- Challenges to whole-genome sequencing ducon. KINAXO’s chemical proteomics and global quantave phosphoproteomics Idenfying de novo and re-current mutaons in cancer plaorm revealed Sorafenib’s target profile and allowed quanficaon of phosphoryla- Addressing tumor heterogeneity on paerns in relaon to drug administraon, thereby facilitang monitoring of the How can we move from characterizing gene variaon to ulizing the whole ge- integraon of signaling and poinng at addional therapeuc applicaons. nome Sequencing tumors rather than tumor cell lines 10:30 Poster Compeon Refreshment Break & Raffles in the The Complex genomic structure of tumor cells: de novo assembly or strategy to detect structural variants Exhibit Hall Are there Cancers of Unknown Primary Tumors? 11:30 microRNA Expression Profiling for the Idenficaon of Moderator: Dalia Cohen, Ph.D., Chief Scienfic Officer, Rosea Forensically Relevant Biological Fluids Genomics, Inc. Jack Ballantyne, Ph.D., Professor, Department of Chemistry, Associate Debate over cancers of unknown primary tumors (CUP) Director for Research, Naonal Center for Forensic Science, University Methods to detect CUPs of Central Florida Consequences of detecon of primary We performed the first miRNAome-wide evaluaon of specific miRNA expression in dried, forensically relevant biological fluids (blood, semen, saliva, vaginal secreons Gene Signatures in Cancer Diagnoscs and menstrual blood). A panel of nine differenally expressed miRNAs was idenfied Co-Moderators: Gary Geiss, Ph.D., Principal Scienst, NanoString that permit the idenficaon of the body fluid using 50pg of total RNA. miRNA profil- Technologies and David Kern, MBA, Director, MyRaQa ing provides a promising alternave approach to body fluid idenficaon for forensic Developing a gene signature casework. Validaon of gene signatures Regulatory consideraons for gene signature diagnoscs 12:00 pm Gene Expression Signatures of Pathway Acvity as Systems Chemical Biology-A New Paradigm Biomarkers in Oncology: RAS Pathway Signature Moderator: Ally Perlina, Senior Applicaon Scienst, GeneGo Inc. Andrey P. Loboda, Ph.D., Research Fellow, Oncology Molecular Profiling, Merck Ulizing tools for drug reposioning Research Laboratories Understanding side effects

Understanding the mechanisms of acon for drugs CHANNEL INFORMATICS Networkable compounds 12:30 Luncheon Presentaon I Sponsored by 6:20 Close of Day Overview of Metabolomics John Ryals, Ph.D., Chief Execuve Officer, Metabolon, Inc. Metabolomics is the global profiling of biochemicals and metabolites in THURSDAY, FEBRUARY 4 biological samples and provides a snapshot of the metabolic state of a biological system. As such, it can rapidly characterize and idenfy metabolic changes REAL EXAMPLES OF INTEGRATING PATHWAY DATA caused by drugs, disease, diet or environment effects. This talk provides an overview of metabolomics and the technology requirements for profiling 8:25 AM Chairperson’s Remarks hundreds of biochemicals. The technology plaorm deployed at Metabolon involves the separaon of analytes on three independent analycal plaorms Megan Laurance, Ph.D., Senior Scienst, Ingenuity Systems, Inc. (GC-MS, LC-MS/MS(+), LC-MS/MS(-)). Proprietary soware processes the mass 8:30 Keynote Presentaon spectral data and retenon mes by matching the run data to a database of Kenneth H. Buetow, Ph.D., Associate Director, Bioinformacs and biochemical standards. This “chemo-centric” approach results in the posive Informaon Technology, Naonal Cancer Instute idenficaon of hundreds of biochemicals in a single sample. Through stascal analysis, the significant changes are idenfied and mapped onto biochemical 9:00 Cooperave and Complementary Genec Selecon in pathways. Because biochemicals are closely related to biological phenotype, Brain Tumors idenficaon of affected pathways not only provides insight into the biological Markus Bredel, M.D., Ph.D., Director, Northwestern Brain Tumor mechanism but uncovers biomarkers useful in diagnosing and monitoring. Instute Research Program, Assistant Professor, Department of Neurological Surgery, Robert H. Lurie Comprehensive Cancer Center, Northwestern University Feinberg School of Medicine, and Lurie Center for Cancer Genecs Research and Center for Genec Medicine Brain tumors are a disease of the genome. These tumors show recurrent paerns of genec aberraons. Dissecng which genec events funcon cooperavely to deregu- CANCER & late principal signaling pathways in brain tumors and which are complementary to such

Online: Tri-Conference.com Email: [email protected] Fax: 781-972-5425 21  MED001701 1:00 Luncheon Presentaon II Sponsored by FRIDAY, FEBRUARY 5 Regulatory Network Analysis of Cancer Gene Expression Profiles for Target and Biomarker Discovery Using the ExPlain™ MICRORNA DIAGNOSTICS FOR CANCER: Analysis System TRANSLATING INFORMATION TO PRACTICAL Raymond DiDonato, Ph.D., Product Manager, BIOBASE Corporaon USE High-throughput gene expression analysis techniques generate large 8:30 AM Chairperson’s Opening Remarks amounts of data, which pose a parcular challenge of transforming Dalia Cohen, Ph.D., Chief Scienfic Officer, Rosea Genomics, Inc. expression data into meaningful hypotheses for target discovery and candidate biomarker idenficaon. Tradional approaches for interpret- 8:35 Keynote Presentaon: Causes and Consequences of ing expression data rely on mapping differenally expressed genes to microRNA Dysregulaon in Cancer canonical pathways, biological processes, or disease states. However, Carlo M. Croce, M.D., Professor, Internal Medicine, College of Medicine understanding the transcriponal regulators and upstream signaling & Public Health, The Ohio State University During the past several years it has become clear that altera ons in the expression events that lead to differenal gene expression can help beer idenfy of microRNA genes contribute to the pathogenesis of most, perhaps all, human ma- the molecular mechanisms that influence changes in gene expression lignancies. These alteraons can be caused by a variety of mechanisms, including profiles, facilitang discovery of targets which themselves are not differ- deleons, amplificaons or mutaons involving microRNA loci, by epigenec silenc- enally expressed, but which are key to underlying disease mechanisms. ing or by dysregulaon of transcripon factors targeng specific microRNAs. Since In this session we present a case study in which ExPlain™, a tool that malignant cells show dependence on the dysregulated expression of microRNA genes, employs the BIOBASE Knowledge Library™ for promoter and regulatory which in turn control or are controlled by dysregulaon of mulple protein coding oncogenes or tumor suppressor genes, these small RNAs provide important opportu- network analysis, was used to uncover target and biomarker candidates nies for development of future microRNA based therapies. from a cancer gene expression experiment, by idenfying upstream signaling molecules likely involved in the underlying pathways leading to 9:05 microRNA Polymorphisms and the Future of the disease state. Personalized Medicine Prasun J. Mishra, Ph.D., Laboratory of Cancer Biology & Genecs, 1:45 Ice Cream Refreshment Break in the Exhibit Hall Naonal Cancer Instute, NIH PLENARY KEYNOTE SESSION Referred to as the micromanagers of gene expression, microRNAs are evoluonarily conserved small non-coding RNAs. Polymorphisms in the microRNA pathway can in- 2:15 Plenary Keynote Introducon fluence gene regulaon and are emerging as powerful tools to study the biology of 2:25 Plenary Keynote Presentaon (See Page 26 for Details) diseases. Detecon of microRNA-polymorphisms can potenally improve diagnosis, 3:05 Refreshment Break in the Exhibit Hall treatment and prognosis in paents and has profound implicaons in the fields of pharmacogenomics and personalized medicine. SYSTEMS BASED APPROACHES TO CANCER SEQUENCING: PUTTING TOGETHER A NETWORK OF 9:35 Living in a Sequen-omics World: Data Integraon Issues CHANGES and Challenges 3:45 Chairperson’s Remarks Gavin Gordon, Ph.D. Co-Director, Thoracic Surgery Oncology Lab, Brigham & Womens Hospital Robert L. Strausberg, Ph.D., Deputy Director, J. Craig Venter Instute DNA sequencing and other “-omics” plaorms (e.g., mRNA, microRNA, CGH, exon, 3:50 Whole Genome Sequencing in Cancer SNP, and other arrays) have experienced a technological revoluon in throughput and Gad Getz, Ph.D., Head, Cancer Genome Analysis, The Broad Instute scale over the preceding decade that shows no sign of slowing. However, data storage and processing advances have outpaced the ability to fully integrate the resulng 4:20 Systemac Discovery of Cancer Gene Fusions using massive quanes of data into biologically meaningful and predicve models to apply Paired End Transcriptome Sequencing to risk esmaon, prevenon, and cure of human diseases, most notably cancer. In Chandan Kumar, Ph.D., Michigan Center for Translaonal Pathology, addion, further technological innovaon will connue to drive down costs which University of Michigan will exacerbate the problem in the near-term but over the long-term will bring more resources to bear in solving these issues and challenges. This session will provide a Gene fusions represent common genec aberraons in cancers that can serve as comprehensive view of the sequen-omics landscape and idenfy the key issues that specific biomarkers and therapeuc targets. The recent discovery of recurrent gene will need to be addressed in the future for these plaorms to posively affect human fusions in prostate and lung cancers portends similar aberraons in other common health. carcinoma. We employ paired end transcriptome sequencing and customized bioin- formac pipelines to characterize gene fusions and chimeric transcripts in cancer. 10:05 Sponsored Presentaon (Sponsorship Opportunity Available) 4:50 Mapping Cancer Genomics Data to Pathways 10:20 Coffee Break David Haussler, Ph.D., Professor & Director, Biomolecular Science & 11:00 The Onco-SNP and Cancer Risk: microRNA Binding Site CHANNEL INFORMATICS Engineering, University of California, Santa Cruz Polymorphisms as Biomarkers It is essenal, but challenging to interpret cancer genomics data in terms of biological Joanne B. Weidhaas, Ph.D., Assistant Professor, Therapeuc Radiology, meaningful perturbaons of molecular pathways within tumor cells. I will discuss a new Cancer Genomics Browser, on the web at genome-cancer.ucsc.edu, that accom- Yale University Since microRNAs are global regulators, small aberraons such as SNPs that disrupt plishes this through large-scale data analysis and probabilisc modeling. This method- their coding sequences or their target binding sites can alter cellular homeostasis and ology is currently being used in several large-scale cancer studies, including the ISPY enhance cancer risk. MicroRNA binding site polymorphisms have turned out to be breast cancer trial, the TCGA project and by one of the SU2C Dream Teams. some of the strongest biomarkers of cancer risk, can act as biomarkers of outcome, 5:20 Analyzing Coding Variants and may be future targets for therapy. Pauline Ng, Ph.D., Senior Scienst, Human Genomic Medicine, J. Craig 11:30 Role of microRNA Based Profiling in Determining Tissue Venter Instute Whole genome and whole exome sequencing are idenfying a large number of coding of Origin for Carcinoma of Unknown Primary variants. Some of these coding variants may have funconal consequences that lead Gauri Varadhachary, M.D., Associate Professor of Medicine, to disease. I will discuss the behavior of coding variants and the webtools we have Department of G.I. Medical Oncology, M.D. Anderson Cancer Center made to analyze them. Carcinoma of unknown primary (CUP) is a heterogeneous disease where a paent presents with metastases without an idenfiable primary. As more effecve cytotoxic 5:50 Close of Day and targeted therapies emerge for addional known cancers, accurate idenficaon of CUP subtypes will become increasingly important to the appropriate care of these paents. MicroRNA expression profiling is an emerging tool to help with idenficaon of ssue of origin in paents with CUP.

12:00 PM Luncheon Presentaon (Sponsorship Opportunity Available) or Lunch on Your Own CANCER &

Online: Tri-Conference.com Email: [email protected] Fax: 781-972-5425 22  MED001702 TARGETING CANCER STEM CELLS 1:00 Chairperson’s Remarks Tim Hoey, Ph.D., VP, Cancer Biology, OncoMed Pharmaceucals, Inc. 1:05 Impact of Anbodies on Cancer Stem Cells: Discovering Underlying Pathways Essenal to Cancer Stem Cell Biology Tim Hoey, Ph.D., VP, Cancer Biology, OncoMed Pharmaceucals, Inc. Cancer stem cells are thought to mediate tumor iniaon, metastasis, and recurrence. We have isolated and characterized CSCs from a variety of major tumor types and have found that these cells are preferenally resistant to many current therapies. As part of our effort to develop novel agents targeng CSCs, we have developed an an- DLL4 anbody that blocks Notch signaling. An-DLL4 inhibits tumor growth through mulple mechanisms including a reducon in CSC frequency. 1:35 Understanding Tumor Cell Heterogeneity in NSCLC: Contribuons to Resistance and Relapse Erica L. Jackson, Ph.D., Scienst, Department of Tumor Biology and Angiogenesis, Genentech, Inc. Tumors are made up of a heterogeneous mixture of cell types and it is possible that disnct cell populaons play unique roles in tumorigenesis. We are studying funconally defined cell populaons to determine what disnguishes chemo-resistant cells from bulk tumor cells. 2:05 New Visions of Cancer Therapy through the Prism of the Cancer Stem Cell Hypothesis Jusn D. Lathia, Ph.D., Research Associate, Department of Stem Cell Biology and Regenerave Medicine, Lerner Research Instute, Cleveland Clinic Foundaon The failure of convenonal therapies to fundamentally alter the survival of advanced and metastac cancers has many causes but one appears to be the striking cellular heterogeneity in most cancers. The cancer stem cell hypothesis posits that tumors contain a cellular hierarchy of differenaon and tumor propagaon potenal. As studies have demonstrated that cancer stem cells display therapeuc resistance, angiogenic potenal, and a propensity towards invasion/metastasis, the idenficaon of signaling pathways and molecular targets in cancer stem cells may yield improved cancer therapies.

2:35 Close of Conference INFORMATICS CHANNEL INFORMATICS CANCER &

Online: Tri-Conference.com Email: [email protected] Fax: 781-972-5425 23  MED001703 Cambridge Healthtech Institute’s Fifth Annual Stem Cells Shaping the Future of Regenerative Medicine

WEDNESDAY, FEBRUARY 3 3:50 Presentaon Sponsored by 4:20 Recepon in the Exhibit Hall (Sponsorship Available) 7:00 AM Registraon and Morning Coffee 5:20 BREAK-OUT DISCUSSIONS in the Exhibit Hall 8:00 Plenary Keynote Session (See Page 26 for Details) Forecasng Cell Therapy Sales - How and Why 9:40 Grand Opening Refreshment Break in the Exhibit Hall Host: Dawn Driscoll, MBA, Ph.D., Principal, DCi Biotech Investors, Manufacturing, HR, and Management all want to know, “How much of ADVANCING REGENERATIVE MEDICINE this cell therapy are you really going to sell, and when?” This interacve discussion will address: Addressing the numerous topics central to the theme of regenerave medicine, this meeng assembles prominent researchers, clinicians, businessmen, and regulators Basics of forecasng for both allogeneic and autologous cell therapies, in or- who not only are at the cung edge of their respecve fields, but also represent wide der to support decision making for clinical development, investor presenta- areas of experse. The cross-ferlizaon of the informaon presented in the area of ons, and manufacturing capacity; stem cell research engineers brainstorming and provides a forum for discussion to Determining realisc paent numbers, the impact of reimbursement on enable the rapidly expanding therapeuc potenal of regenerave medicine. sales, the impact of projected sales on GMP manufacturing capacity; Staffing needs to support a given forecast. 11:00 Chairperson’s Remarks Dawn Driscoll, MBA, Ph.D., Principal, DCi 6:20 Close of Day 11:10 Keynote Presentaon THURSDAY, FEBRUARY 4 Stem Cells: Moving from Discovery Towards the Clinic Alan Trounson, Ph.D., President, California Instute of Regenerave Medicine ENABLING TECHNOLOGIES FOR REGENERATIVE 11:55 Featured Presentaon MEDICINE Diabetes Under Control Andrew Rakeman, Ph.D., Scienfic Program Manager, Regeneraon, Juvenile Diabetes 8:25 Chairperson’s Remarks Research Foundaon Marc Unger, Ph.D., CSO, Fluidigm Diabetes treatments necessitate the replacement or regeneraon of pancreac beta cells to improve glucose control and avoid serious side effects. Re- 8:30 Human Embryonic Stem Cells (hESCs) for Tissue placement of beta cells has aracted considerable aenon with the use of Regeneraon: How to Get the Cells We Need cadaveric islets, pig islets and a variety of adult stem cells. It appears that Harold S. Bernstein, M.D., Ph.D., Professor of Pediatrics, Eli and Edythe Broad Center the best source to date is cells obtained from human embryonic stem cells of Regeneraon Medicine & StemCell Research, University of California, San Francisco and there is hope that iPS cells may one day also be an appropriate source. Cardiovascular Research Instute More recently, interest has focused on regenerang the pancreas as a result of access Cell therapies derived from hESCs have shown promise in animal models of human to human progenitors as well as a beer understanding of cell proliferaon and neo- disease. However in some cases, such as in aempts to augment myocardial ssue, genesis will accelerate the development of beta cell regenerave medicines. fully differenated hESC-derived cells may be beyond the ability to fully incorporate into and improve the funcon of exisng ssue. To address this, we have focused 12:40 PM Luncheon Presentaon (Sponsorship Opportunity on idenfying subpopulaons of hESCs that preferenally differenate into specific embryonic germ layers, developing chemical and miRNA-based enrichment strate- Available) or Lunch on Your Own gies for directed differenaon of hESCs, and creang reporter hESC lines and non- 1:45 Dessert in the Exhibit Hall integrang molecular beacons that facilitate the selecon of precursors commied to specific lineages. CONSIDERATIONS FOR ADVANCING REGENERATIVE MEDICINE INTO THE CLINIC 9:00 Engineering the Morphogenesis of Pluripotent Stem Cells Todd McDevi, Ph.D., Assistant Professor, Wallace H. Coulter Department of Biomedical 2:15 Chairperson’s Remarks Engineering, Georgia Instute of Technology and Emory Dawn Driscoll, MBA, Ph.D., Principal, DCi Biotech 9:30 Improved Culture Condions for the Growth and BIOLOGICS CHANNEL 2:20 hESC-Derived Oligodendrocyte Progenitor Cells- Recovery of Cryopreserved Human Pluripotent Stem Cells GRNOPC1 for Acute Spinal Cord Injury Angie Rizzino, Ph.D., Professor, Eppley Instute for Research in Cancer and Allied Edward Wirth III, M.D., Ph.D., Medical Director, Geron Corporaon Diseases, University of Nebraska Medical Center Poor recovery of cryopreserved hES cells and iPS cells is a significant impediment to 2:50 From Tissue Engineering to Regenerave Medicine: An progress with pluripotent stem cells. To address this problem, we have determined Evoluon in Understanding that Y-27632, a specific inhibitor of Rho kinase (ROCK) acvity, significantly enhances recovery of hES cells from cryopreserved stocks when cultured with or without a Damien Bates, M.D., Ph.D., Chief Medical Officer, Organogenesis, Inc. growth inacvated feeder layer. Remarkably, hES cells that had formed relavely 3:20 MSC-Derived SB623 Cells for Stable Stroke few colonies even seven days aer thawing exhibited rapid growth upon addion of Casey Case, Ph.D., Vice President of Research, SanBio, Inc. Y-27632. Addionally, we determined that Y-27632 significantly improves the recov- SB623 cells are derived from bone marrow stromal cells (MSCs). They have ery of cryopreserved human iPS cells and their growth upon subculture. shown great potenal in models of CNS regeneraon. The cells are used al- logeneically and implanted directly at the site of injury. Our first clinical applicaon will be in stable ischemic stroke paents. Models of efficacy and safety will be discussed as will issues pertaining to manufacturing and clinical plans.

24 Online: Tri-Conference.com Email: [email protected] Fax: 781-972-5425  MED001704 tent ES cell-derived cardiac myocytes. We use high-throughput gene inacvaon 10:00 Presentaon Sponsored by methods, including siRNA and gene trapping in ES cells, and then analyze ES cell- Programmable, Fully Automated Microfluidic Stem Cell derived cardiomyocytes. Our inial signaling studies focused on mouse ES cells, and ES cell-derived mice. We are using human iPS cells for similar signaling studies and to Culture System produce models of human cardiac disease including Long QT syndrome. Marc A. Unger, Ph.D., CSO, Fluidigm Corporaon Cell reprogramming techniques require treang cells with mulple factors, either 4:50 Using iPS Cells to Model Neurological Diseases for conversion of differenated cells into induced pluripotent stem (IPS) cells or for Clive N. Svendsen, Ph.D., Professor, Anatomy & Neurology, University of Wisconsin conversion of pluripotent cells into a desired type of differenated cells. Fluidigm is 5:20 Hoseok Song, Ph.D., Professor of Biology, University of developing a versale, automated cell culture system which can culture cells, carry California, San Diego out mul-factor dosing experiments, and image the cells in both fluorescence and incident light modes in any desired me sequence. The results of in-chip cell culture 5:50 Close of Day and mul-factor dosing experiments will be described and applicaons discussed. FRIDAY, FEBRUARY 5 10:30 Poster Compeon Refreshment Break & Raffles in the Exhibit Hall IPS CELLS FOR REGENERATIVE HEALING 11:30 Integrated Chemical Genomics Reveals Modifiers of 8:30 AM Chairperson’s Opening Remarks Cell Fate in Pluripotent Stem Cells Timothy J. Kamp, M.D., Ph.D., Professor of Medicine & Physiology; Co-Director Stem Cell April Pyle, Ph.D., Assistant Professor, Microbiology, Immunology & Molecular Genecs, and Regenerave Medicine Center, University of Wisconsin Eli and Edythe Broad Center of Regenerave Medicine & Stem Cell Research; Jonsson 8:35 microRNA-Target Gene Networks as Fundamental Comprehensive Cancer Center, University of California, Los Angeles Factors in the Next Generaon Regenerave Strategies While hESCs can be maintained in vitro, cells grown in connuous culture have been Preethi H. Gunaratne, Ph.D., Assistant Professor, Department of Biology & Biochemistry, shown to develop cytogenec and genec aberraons associated with cancer in University of Houston vivo. Addionally, hESCs exhibit poor survival as single cells following dissociaon, MicroRNAs are small non-coding RNAs that integrate mulple genes within and which limits the ability to perform genec manipulaon and homogenous differen- across biological pathways. LIN28/let-7; c-MYC-E2F/miR-17-92 and Oct4/Sox2/miR- aon of hESCs. In order to idenfy pathways involved in regulang self-renewal and 302-cyclin D1 networks have been ghtly linked to embryonic (ES) and more recently survival without instability, we have developed a cell-based high content screening to iPS cells. We have also uncovered addional miRNAs regulated by Ronin, a non- (HCS) assay using small molecules. This method provided a comprehensive approach canonical pluripotency factor that target genes regulang cytoskeletal remodeling for studying hESC fate in vitro and idenfied a number of novel regulators of hESC and epigenec silencing. Discussed is the potenal role of these key miRNAs in the growth. next generaon regenerave strategies. 12:00 PM Biocompable Graed Carbon Nanotubes as 9:05iPS Cells Offer New Alternave and Early Treatment in Scaffolds for Preferenal hESC Differenaon Genec Diseases Jennifer Lu, Ph.D., Professor, School of Engineering, University of California, Merced Yuet Wai Kan, MB, BS, D.Sc., Professor, Department of Medicine, University of California, Presented is our research on using biocompable graed carbon nanotubes as scaf- San Francisco folds for preferenal neuron cells differenaon from hESCs. It has been found that Two alternaves are currently available to parents if the prenatal diagnosis of a seri- carbon nanotube-based scaffolds promote the growth factor adsorpon, leading to ous genec disease is made: to terminate the pregnancy, or to connue it and take more selecve differenaon. It has been observed that surface properes such as care of a seriously ill child. Generaon of iPS cells from the amnioc fluid or CVS cells hydrophilicity and charge can play important roles in direcng hESC differenaon. used for the diagnosis, correcon of the mutaon, and differenaon of the cells Novel responsive scaffolds have been synthesized and the potenal use of such dy- into specific ssues may in the future offer a new alternave. In addion, it will allow namic scaffolds for cell growth, differenaon and proliferaon will be discussed. early treatment of the genec disease, an important consideraon in diseases where

® organ damage begins early in life. 12:30 Luncheon Presentaon Sponsored by Mulplex Biomarker Assays for Translaonal Research Meso Scale Discovery 9:35 Pluripotent Stem Cells Derived from Adult Human Testes Marn Dym, Ph.D., Professor, Biochemistry & Molecular & Cellular Biology, Georgetown Robert Umek, Ph.D., Director of Research, Meso Scale Discovery University Medical Center Male germline stem cells obtained from adult human testes can be reprogrammed 1:45 Ice Cream Refreshment Break in the Exhibit Hall spontaneously to generate pluripotent stem cells. The producon of these “non- canonical” iPS cells is spontaneous, and do not require the addion of exogenous PLENARY KEYNOTE SESSION genes, some of which may be cancer causing. Our results suggest that human sper- 2:15 Plenary Keynote Introducon matogonial stem cells have great potenal for cell-based, autologous organ regeneraon therapy for various diseases and it is thus possible that in the near future men 2:25 Plenary Keynote Presentaon (See Page 26 for Details) could be cured of disease with a biopsy of their own testes. 3:05 Refreshment Break in the Exhibit Hall IPS CELLS: FROM SCREENING TO THERAPY 10:05 Poster Presentaon: Differenaon of Human Embryonic and Human Induced Pluripotent Stem Cells Along Induced pluripotent stem cells (iPS) cells, the most recent advancement in stem cell the Oc Lineage research, even further widen and generate applicaons for stem cell research. iPS Kinuko Masaki, Stanford University cells exhibit great promise in drug discovery and screening as well as in regenerave 10:20 Coffee Break medicine. iPS Cells: From Screening to Therapies not only explores current methods BIOLOGICS CHANNEL of generang, maintaining, and ulizing iPS cells but will address the shi in using IPS CELLS FOR REGENERATIVE HEALING CONT’D them to contribute to Shaping the Future of Regenerave Medicine. 11:00 Directed Differenaon of Human iPS Cells Generates 3:45 Chairperson’s Remarks Acve Motor Neurons Bruce Conklin, M.D., Senior Invesgator, Gladstone Instute of Cardiovascular Disease, William Lowry, Ph.D., Assistant Professor, Department of Molecular, Cell and Professor of Medicine, Division of Medical Genecs, University of California, San Developmental Biology, University of California, Los Angeles Francisco A study of gene expression profiles of mouse and human ESCs and iPSCs suggests IPS CELLS FOR DISEASE MODELS that, while iPSCs are quite similar to their embryonic counterparts, a recurrent gene expression signature appears in iPSCs regardless of their origin or the method by 3:50 Featured Speaker which they were generated. Shown is how both hESCs and hiPSCs can differenate to form fully funconal differenated progeny. We are now seng out to understand Potenal Promise of iPS Cells for Understanding Disease whether the differenated progeny of hESCs and hiPSCs share commonalies or dif- Progression ferences as was observed in their undifferenated parent cells in an aempt to make Sheng Ding, Ph.D., Assistant Professor, Chemistry, Scripps Research Instute predicons about whether these two types of pluripotent cells have similar poten- 4:20 iPS Cells for Cardiovascular Models and Diagnoscs als in regenerave medicine. Bruce Conklin, M.D., Senior Invesgator, Gladstone Instute of Cardiovascular Disease, Professor of Medicine, Division of Medical Genecs, University of California, San Francisco Our funconal genomic experiments focus on GPCR signaling pathways in pluripo-

Online: Tri-Conference.com Email: [email protected] Fax: 781-972-5425 25  MED001705 11:30 Funconal Cardiomyocytes Derived from Human Induced Pluripotent Stem Cells Timothy J. Kamp, M.D., Ph.D., Professor of Medicine & Physiology; Co-Director Stem Cell and Regenerave Medicine Center, University of Wisconsin Human iPS cells hold great promise for cardiovascular research and therapeuc applicaons, but the ability of human iPS cells to form funconal cardiomyocytes requires careful analysis and opmizaon. We provide electrophysiological, pharmacological, and biochemical evidence that iPS cells can diﬀerenate into the three major types of funconal cardiomyocytes which can be used in a variety of applicaons. 12:00 PM Lunch on Your Own IPS CELLS FOR DRUG SCREENING

1:00 Chairperson’s Remarks 1:05 Featured Speaker Stem Cells and Drug Discovery: The Beginning of a New Era? Lee Rubin, Ph.D., Director, Translaonal Medicine, Harvard Stem Cell Instute 1:35 CATALYST: The Industrializaon of Sponsored by Advanced iPSC Technology for Drug Discovery Dan Shoemaker, Chief Technology Officer, Fate Therapeucs CATALYST is designed to accelerate the innovaon of induced pluripotent stem cell (iPSC) technology in collaboraon with industry to support launching a fully-enabled plaorm in this new paradigm of drug discovery and development. CATALYST is exploring the creaon of iPSC-derived, disease-specific model systems that improve the recapitulaon of human physiology and more effecvely predict clinical response. CATALYST is commied to developing an iPS cell technology plaorm to accelerate candidate idenficaon and lead validaon for drug discovery and development for pharmaceucal members. This session will discuss:

The crical elements of industrializaon, including cell sourcing, reprogramming, differenaon and commercial supply Approaches for creang non-genecally modified iPS cells and mature phenotypes Quantave methods to analyze cell states for high-quality differenaon and disease modeling Uses of iPSC technology in drug discovery 1:50 Sponsored Presentaon Sponsored by HOTEL & Targeng Muscular Dystrophy: How do we Mimic the In Vivo System? TRAVEL INFORMATION Lorena Griparic, Ph.D., Research Scienst, DV Biologics Conference Venue Muscular dystrophy (MD) is a well characterized neuromuscular disorder. Here we The Moscone North Convenon Center show that using cells isolated from different ssues of MD paents and their pedi- 747 Howard Street gree is an effecve tool for understanding how to treat the disease. Our MD pedigree San Francisco, CA 94103 system is the first commercially available tool allowing the study of this disease and www.moscone.com the producon of iPS cells. Host Hotel Marrio San Francisco Hotel 2:05 iPSC-enabled Drug Discovery: A Paradigm Sponsored by 55 Fourth Street San Francisco, CA 94103 Shi to Increase POS in the Clinic Discounted Group Rate: $199* s/d Berta Strulovici, Chief Technology Officer, iPierian *Room Rate includes Unl recently, human disease specific pluripotent stem cells could be made only complimentary internet by tedious genec modificaon of exisng hES cells or by generang such cells access in your guestroom from embryos with diagnosed monogenic diseases. Recent advances using in- Discounted Room Rate Cut Off Date: duced pluripotent stem cells (iPSCs) have enabled the producon of unlimited January 6, 2010 numbers of cells with a very specific genec background that can be used as (T) 415-896-1600 models for drug discovery. Coupled with the ability of these cells to be differ- (F) 415-486-8101 enated to virtually “any type of cell in the body”, the iPSC technology has the ability to revoluonize the way drug discovery is done today. In my presentaon, Minutes from the Moscone Convenon Center, discover the beauful San Francis- I will describe the use of human iPSC-based assays for drug discovery in our co Marrio rising 39 stories high into the city skyline. Just south of Market Street, therapeuc areas of focus. the hotel is steps away from the city’s top aracons, including the Yerba Buena Gardens, world-class shopping on Union Square, and AT&T Park, home of the San Francisco Giants. Enjoy magnificent views of downtown San Francisco. 2:20 iPS Cells Panel of Experts BIOLOGICS CHANNEL iPS cells have invigorated and united the stem cell research community and strides Please visit the conference website to make your hotel reservaon on-line. connue in efficient re-programming. This is evident through funding and companies You may also call the hotel directly to make your reservaon by asking for the invesng their future through this revoluonary technology. Hear the experts in the Cambridge Healthtech and/or Molecular Medicine Tri-Conference group rate. iPS Cells field as they present their latest technology followed by an interacve panel Reservaons made aer the cut-off date or aer the group room block has been discussion. filled (whichever comes first) will be accepted on a space and-rate availability basis. Rooms are limited, so please book early. 3:05 Close of Conference For additional Travel Information, visit the hotel and travel page of the conference website tri-conference.com.

Reserve your hotel and save $75 oﬀ your conference registraon.* *You must book your reservaon under the Molecular Medicine Tri-Conference room block for a minimum of three nights at the Marrio San Francisco Hotel.

Online: Tri-Conference.com Email: [email protected] Fax: 781-972-5425 26  MED001706 Cambridge Healthtech Institute’s Second Annual RNA Interference: From Tools to Therapies WEDNESDAY, FEBRUARY 3 EXPLORING HIGH CONTENT RNAI SCREENS 2:15 Chairperson’s Remarks 7:00 AM Registraon and Morning Coffee Paul Kassner, Ph.D., Scienfic Director, Lead Discovery, Amgen, Inc. 8:00 Plenary Keynote Session (See Page 26 for Details) 2:20 Use of Fluorescence Microscopy to Track Protein Localizaon in siRNA Screening 9:40 Grand Opening Refreshment Break in the Exhibit Hall Paul Kassner, Ph.D., Scienfic Director, Lead Discovery, Amgen, Inc. THE POWER AND POTENTIAL OF 2:50 Comparave Analysis of RNAi Screening Performance WHOLE GENOME RNAi SCREENS Across Mulple Kinase-Focused Libraries: How Good is a Good Kinase Targeng Sequence? 11:00 Chairperson’s Remarks Hakim Djaballah, Ph.D., Director, HTS Core Facility, Memorial Sloan Keering Cancer Christophe J. Echeverri, Ph.D., CEO and CSO, Cenix BioScience GmbH Center 11:10 Genome-wide RNAi Screen in Drosophila Cells Idenfies We have assembled and obtained several kinase focused libraries for use G Protein-coupled Receptor Kinase 2 as an Evoluonarily in the comparave analysis of RNAi knockdown performance. We have Conserved Regulator of the NFКB Signaling performed a systemac RNAi screening of the Kinome represented in sev- Mika Rämet, M.D., Ph.D., Professor of Experimental Pediatric Immunology and eral siRNA and shRNA libraries for gene inacvaon that modulate apop- Infecous Diseases, University of Tampere, Finland toc events in an isogenic pair of cell lines, namely HeLa/B5 and HeLa/ We have carried out an RNA interference-based genome-wide in vitro N10. HeLa B5 is stably transfected and over expresses Bcl-XL, a member of reporter assay screen in Drosophila for components of NF-КB pathways. the an-apoptoc Bcl-2 family, whereas the HeLa N10 contains an empty We analyzed 16, 025 dsRNA-treatments and idenfied ten novel NF-КB expression vector as a control. We have employed a high content assay regulators. Of these, Gprk2 was shown to be evoluonary conserved measuring real me inducon of apoptosis in live cells together with an regulator of NF-КB signalling. siRNA-silencing of human ortholog GRK5 end point measure of nuclear count and morphological changes post fixa- in HeLa cells impaired NF-КB reporter acvity. Morpholino-silencing of on and staining. We will present and discuss our findings. zebrafish GRK5 homolog in fish embryos caused impaired IL-1β and TNF-α expression aer E. coli infecon. Gprk2/GRK5 was idenfied as 3:20 Speaker to be Announced an evoluonarily conserved modulator of NF-КB signaling. 3:50 Sponsored Presentaon (Sponsorship Opportunity Available) 11:40 Alexander Bishop, Ph.D., Associate Professor, UT San 4:20 Recepon in the Exhibit Hall (Sponsorship Available) Antonio 5:20 BREAK-OUT DISCUSSIONS in the Exhibit Hall 12:10 PM Generaon And Integraon of HTRNAi Screening Data Design of HT-RNAi Screens for Target Idenficaon and Validaon Pedro Aza-Blanc, Ph.D., Director, Funconal Genomics Resources, The Burnham Instute Moderators: Paul Kassner, Ph.D., Scienfic Director, Lead Discovery, Amgen Inc. for Medical Research Christopher Miller, Ph.D., Group Director, Applied Genomics, Bristol-Myers Squibb Co. Topics for discussion: 12:40 Luncheon Presentaon I Sponsored by Choice of screening format, libraries and reagents Using siRNA to Invesgate Non-Coding RNA Design of posive and negave controls (ncRNA) Funcon in Control of Mitosis and Addressing the issue of off-target effects Apoptosis in Cells Stascal approaches for priorizing hits Susan Magdaleno, Ph.D., Senior Manager, Scienst, RNAi Technologies Research & Perspecves on siRNA Delivery Systems Development, Applied Biosystems Moderators: Ian MacLachlan, Ph.D., Chief Scienfic Officer, Tekmira Pharmaceucals Inc. Long non-coding RNAs (ncRNA) are crical to biology and disease. Life Antonin de Fougerolles, Ph.D., Vice President, Research, Alnylam Pharmaceucals Inc. Technologies has now developed a suite of integrated tools and work- Topics for discussion: flows to discover, validate, and knock-down ncRNA which will acceler- Key aributes necessary for delivery Pharmaceucal aspects of siRNA formulaons BIOLOGICS CHANNEL ate understanding of the funcon of ncRNA in the cell. We will describe the special requirements for using siRNAs to knock down ncRNA and Formulaon-specific safety quesons will highlight the applicaon of siRNAs to invesgate ncRNA funcon in Immune acvaon and RNAi delivery Progress in Developing RNAi Therapeucs regulang mitosis and apoptosis in normal and cancer cells. Moderator: Bob Brown, Ph.D., Senior Vice President, Research, Dicerna Pharmaceucals Corp. Topics for discussion: 1:10 Luncheon Presentaon II (Sponsorship Opportunity Available) Transioning from the lab to the clinic 1:45 Dessert in the Exhibit Hall Challenges in the clinic RNAi therapies for acute versus chronic condions Lessons learnt from gene therapy and ansense 6:20 Close of Day

27 Online: Tri-Conference.com Email: [email protected] Fax: 781-972-5425  MED001707 THURSDAY, FEBRUARY 4 PLENARY KEYNOTE SESSION

SCREENING AND VALIDATING DRUG TARGETS 2:15 Plenary Keynote Introducon USING RNAi SCREENS 2:25 Plenary Keynote Presentaon (See Page 26 for Details) 3:05 Refreshment Break in the Exhibit Hall 8:25 AM Chairperson’s Remarks Christopher Miller, Ph.D., Group Director, Applied Genomics, Bristol-Myers Squibb Co. CLINICAL CHALLENGES WITH RNAi THERAPEUTICS 8:30 Cancer Target Idenficaon and Validaon by siRNA Library Screening 3:45 Chairperson’s Remarks Xiaoyu Lin, Ph.D., Associate Research Invesgator, siRNA Therapeucs, Abbo Laboratories Crisna Rondinone, Ph.D., Director Research, Metabolic Diseases, Hoffmann La Roche Inc. We have been performing large-scale siRNA library screens to idenfy novel cancer targets. One of the crical aspects of screen data analysis 3:50 LNA Anmirs – Pioneering microRNA Therapeucs is to discard false posive hits due to siRNA off-target effect. We will Henrik Orum, M.Sc., Ph.D., VP and CSO, Santaris Pharma discuss several different approaches to confirm on-target effect of siR- Short, single stranded LNA oligonucleodes delivered systemically as na- NA library hits. Using several targets idenfied in the library screen as ked molecules are able to potently and safely inhibit therapeucally arac- examples, we will update on the progress of how RNAi-based technolo- ve miRNAs in a range of ssues in experimental animals. The presenta- gies have helped target discovery and validaon in the oncology area. on will provide an update on the unique features of LNA oligonucleodes in miRNA therapeucs with parcular emphasis on the pre-clinical and 9:00 Hing Cancer Where it Hurts Most: Large Scale RNAi clinical development of SPC3649, an LNA AnmiR targeng miRNA-122. Screens for Cancer Cell Vulnerability Roderick L. Beijersbergen, Ph.D., Group Leader, Division of Molecular Carcinogenesis, The 4:20 Pre-clinical and Clinical Development of Atu027 (siRNA- Netherlands Cancer Instute lipoplex/AtuPLEX) for Oncology Large scale RNAi screens for cancer cell vulnerability RNA interference based Ansgar Santel, Ph.D., Senior Scienst, Silence Therapeucs plc technologies allow for the interrogaon of the role and phenotype of all Atu027 refers to a liposomally formulated siRNA targeng PKN3 expres- individual genes in the human genome. Using these techniques we aim to sion in the vascular endothelium. Pre-clinical studies in rodents and idenfy those genes that upon funconal inacvaon have a causal effect non-human primates demonstrated that intravenous administraon is on tumor cell behavior and survival represenng novel drug targets. well tolerated and gives rise to RNAi-mediated suppression of PKN3 gene expression. Various proof-of-concept experiments on mouse tu- 9:30 Leveraging RNAi and Chemogenomic Screens for Target mor xenogras suggest profound inhibion of tumor progression and Idenficaon and Validaon parcularly of metastasis, which laid the foundaon for therapeuc ap- Christopher Miller, Ph.D., Group Director, Applied Genomics, Bristol-Myers Squibb Co. plicaon in oncology. Atu027 is currently tested in a Phase-I clinical trial Libraries of RNAi reagents are being widely used for screening cellular on subjects with advanced solid cancers. Pre-clinical data emphasizing assays for target idenficaon. We are using RNAi libraries in combinaon pharmacological acvity in mouse models and an update on the cur- with libraries of small molecule tool compounds. Using both types of rent Phase-I study will be discussed. libraries adds confidence to hits idenfied from these screens and provides genec and chemical tools for hit follow up. Examples will be presented to 4:50 Talk Title to be Announced illustrate this approach in target idenficaon and validaon. 5:20 Talk Title to be Announced Bob D. Brown, Ph.D., Senior Vice President, Research, Dicerna Pharmaceucals Corp. 10:00 Sponsored Presentaon (Sponsorship Opportunity Available) 5:50 Close of Day 10:30 Poster Compeon Refreshment Break & Raffles in the Exhibit Hall FRIDAY, FEBRUARY 5 11:30 Panel: Do’s and Don’ts in RNAi Screening Panelists: Christophe J. Echeverri, Ph.D., CEO and CSO, Cenix BioScience GmbH NOVEL FORMULATIONS FOR RNAi DELIVERY Christopher Miller, Ph.D., Group Director, Applied Genomics, Bristol-Myers Squibb Co. Hakim Djaballah, Ph.D., Director, HTS Core Facility, Memorial Sloan Keering Cancer Center 8:30 AM Chairperson’s Opening Remarks Paul Kassner, Ph.D., Scienfic Director, Lead Discovery, Amgen, Inc. Bob D. Brown, Ph.D., Senior Vice President, Research, Dicerna Pharmaceucals, Corp.

12:30 PM Luncheon Presentaon Sponsored by 8:35 Therapeuc siRNA Delivery: Tackling the 800 RNAi and KinaseSwitch Technology Plaorms Pound Gorilla BIOLOGICS CHANNEL Chrisne L. Olsson, Ph.D., Taconic Steven F. Dowdy, Ph.D., Invesgator, Howard Hughes Medical Instute; Professor, Novel in vivo technology plaorms have recently been developed that Department of Cellular & Molecular Medicine, University of California, San Diego School will enable invesgators to gain greater insights into drug and target- of Medicine related disease mechanisms. TaconicArtemis RNAi and KinaseSwitch To date, siRNA delivery remains the rate-liming step for RNAi thera- mouse models are the newest commercially available addions to peucs development. We developed a Pepde Transducon Domain- these technologies. Inducible/reversible RNAi technology enables gene dsRNA Binding Domain (PTD-DRBD) fusion protein siRNA delivery ap- knockdown in all ssues of the body and can be induced and reversed, proach. PTD-DRBD delivered siRNAs induced RNAi responses in the providing an opmal surrogate for therapeuc drug acon. More re- enre populaon of all cell types assayed (primary and tumorigenic) cently, KinaseSwitch technology, an invaluable tool during key stages of in a non-cytotoxic fashion. PTD-DRBD combinatorial in vivo delivery drug development, allows invesgators to idenfy biological roles of a of EGFR and Akt2 siRNAs induced a synthec lethal response that sig- specific kinase and possible side effects that result from its inhibion. nificantly increased survival of intracerebral glioblastoma pre-clinical models. These observaons demonstrate the ability of PTD-DRBD to 1:45 Ice Cream Refreshment Break in the Exhibit Hall efficiently delivery siRNAs in vivo.

Online: Tri-Conference.com Email: [email protected] Fax: 781-972-5425 28  MED001708 9:05 Inducon of Therapeuc Gene Silencing in Leukocyte- 2:05 Development of Novel Therapeuc RNAi Compounds Implicated Diseases by Targeted and Stabilized Nanoparcles and Effecve in vivo Delivery Approaches Dan Peer, Ph.D., Head, Laboratory of Nanomedicine, Department of Cell Research and Joanne Kamens, Ph.D., Senior Director of Research Collaboraon Management, RXi Immunology and the Center for Nanoscience and Nanotechnology, Tel Aviv University Pharmaceucals Leukocytes are among the most difficult cells to transduce with RNAi. RNA interference (RNAi) offers a novel approach to the drug develop- We developed a strategy that can target different subsets of leuko- ment process, because RNAi compounds can potenally be designed cytes and selecvely silence genes in vivo using targeted, stabilized to target any one of the genes in the human genome. Other potenal nanoparcles (tsNPs). These carriers do not induce lymphocyte ac- advantages of RNAi therapeucs include, rapid development of lead vaon, interferon responses or release liver enzymes and are fully compounds, high selecvity for the target gene, high potency (low degradable. Three pre-clinical examples inflammatory bowel disease dose) and low toxicity due to natural mechanism of acon. We will (IBD), blood cancer and viral infecon will be discussed. We will show introduce unique single-oligo (rxRNAsolo) and short-duplex (rxRNAnano) that tsNPs can be used for in vivo validaon of new drug targets, for RNAi compounds, as well as novel in vivo delivery approaches, includ- prevenon of viral infecon and for inducing therapeuc gene silencing self-delivering rxRNA molecules (sd-rxRNA) for local and systemic ing in a preclinical seng. delivery, and targeted delivery to phagocyc immune cells using.

9:35 Characterizaon of Immune Responses to tkRNAi Therapeucs 2:35 Development of RNA Interference Therapeucs Johannes Fruehauf, M.D., Ph.D., VP, Research, Cequent Pharmaceucals, Inc. Antonin De Fougerolles, VP, Research, Alnylam Pharmaceucals Transkingdom RNA interference (tkRNAi) describes a novel method for Novel therapies based on short interfering RNA (siRNA) duplexes have tremendous delivery of therapeuc RNA interference into gastrointesnal ssues us- potenal to treat diseases by silencing the expression of otherwise non-druggable proteins. Development of therapeucs using siRNA has advanced rapidly, with mulple ing engineered bacteria which produce and deliver mediators of RNAi. different clinical trials ongoing and several more poised to enter the clinic in the Clinical trials are about to begin for the prevenon of colon Polyposis, coming years. Although challenges remain, delivery represents the main hurdle for and for the treatment of Inflammatory Bowel Disease (IBD). Here we faster and broader development of siRNA therapeucs. A summary of the advances demonstrate recent results from large screening efforts characterizing made around siRNA delivery will be presented. the effects of tkRNAi on cytokine profiles and innate immunity. 3:05 Close of Conference

10:05 Sponsored Presentaon (Sponsorship Opportunity Available) 10:20 Coﬀee Break 11:00 Panel: Do We Understand the Challenges We Face With RNAi Therapeucs? Panelists: Bob D. Brown, Ph.D., Senior Vice President, Research, Dicerna Pharmaceucals Corp. Ian MacLachlan, Ph.D., CSO, Tekmira Pharmaceucals John Rossi, Ph.D., Professor and Chair, Molecular Biology, Beckman Research Instute of the City of Hope Steven Highlander, Ph.D., Partner, Intellectual Property, Fulbright & Jaworski, L.L.P.

12:00 PM Luncheon Presentaon (Sponsorship Opportunity Available) or Lunch on Your Own NOVEL APPROACHES FOR TARGETED DELIVERY 1:00 Chairperson’s Remarks John Rossi, Ph.D., Professor and Chair, Molecular Biology, Beckman Research Instute of the City of Hope

vivo. The results obtained demonstrate that Dicer substrate siRNAs can BIOLOGICS CHANNEL be delivered in a cell type speciﬁc manner with an aptamer, and can be generally delivered with dendrimers to eﬀecvely inhibit HIV replicaon in a humanized mouse model.

1:35 Targeted RNA-based Cancer Therapies Paloma H. Giangrande, Ph.D., Assistant Professor, Department of Internal Medicine, University of Iowa A major hurdle for the clinical translaon of siRNAs into effecve therapies is delivery. We describe an RNA aptamer-based approach for the targeted delivery of siRNAs to prostate cancer (PC) cells. The aptamer- siRNA reagent (chimera) is effecve when administered systemically and is suitable for efficient chemical synthesis. When administered systemically to mice bearing PSMA-posive tumors, the RNA chimera triggered tumor regression without affecng normal ssues. This work is the first descripon of in vivo efficacy following systemic administraon of an aptamer-siRNA chimera and thus represents a milestone for this plaorm technology.

Online: Tri-Conference.com Email: [email protected] Fax: 781-972-5425 29  MED001709 Cambridge Healthtech Institute’s Inaugural Cancer Biologics

WEDNESDAY, FEBRUARY 3 ity aﬀorded by these molecules can potenally be leveraged for the development of new immunodrug conjugates.

7:00 AM Registraon and Morning Coffee 2:50 Selecve Penetraon and Targeng of Tumors Tapas K. Das Gupta, M.D., Ph.D., D.Sc., Professor and Head, Surgical Oncology, 8:00 Plenary Keynote Session (See Page 26 for Details) University of Illinois Chicago; Co-founder, CDG Therapeucs, Inc. 9:40 Grand Opening Refreshment Break in the Exhibit Hall CDG Therapeucs has developed a cell penetrang pepde (28aa) DELIVERY OF CANCER BIOLOGICS from azurin, a redox protein secreted by Pseudomonas aeruginosa. PENETRATION AND DISTRIBUTION p28 preferenally enters cancer cells, localizes in the nucleus and sta- bilizes p53 inducing cell cycle arrest and apoptosis in a series of solid tumors. p28 is stable, nontoxic and currently in a Phase I clinical trial. 11:00 Introducon and Welcome Gregory P. Adams, Ph.D., Co-Leader, Molecular and Translaonal Medicine Program, Fox Chase Cancer Center 3:20 A Systems Biology Approach to Engineering Therapeuc Anbodies: Development of an ErbB2/ErbB3 Bispecific 11:10 Tumor Penetraon of Therapeuc Anbodies -The Anbody Impact of Size and Exposure Time on Distribuon Alexandra Huhalov, Ph.D., Principal Scienst, Merrimack Pharmaceucals David Blakey, Ph.D., Chief Scienst, Cancer and Infecon Research Area, AstraZeneca Using quantave biological datasets of cell signaling we have gener- The ability of intact anbodies and fragments to access tumor cells ated computaonal models of the ErbB signaling network and iden- distant from the tumor blood supply is an important therapeuc con- fied ErbB3 as a promising target. The applicaon of these models to sideraon for anbody based oncology drugs. Pre-clinical and clinical guide the design of MM-111, a bispecific anbody-based therapeuc data will be reviewed regarding the impact of size and exposure me targeng the ErbB2/ErbB3 heterodimer, and its antumor acvity will on anbody distribuon within tumors. be discussed.

11:40 An-tumor Efficacy Maximizaon through Blocking 3:50 Large Volume Subcutaneous Delivery: Challenges and Mulple Targets of Angiogenesis Opportunies Dana Hu-Lowe, Ph.D., Group Leader, Associate Research Fellow, Cancer Biology, Pfizer, Robin Hwang, Ph.D., Execuve Director, Halozyme Inc., PGRD-La Jolla There are many monoclonal anbodies (mAbs) in development for cancer thera- Vascular normalizaon and adapvety potenally contribute to resis- peucs. Generally, mAbs require a higher dosage than the typical protein thera- tance to an-VEGF/VEGFR therapies in the clinic. Other targets, in- peucs. It has been shown clinically that the “standard” subcutaneous injecon cluding the Acvin receptor Like Kinase 1 (ALK-1), also play a role in can go up to 1.5 mL, beyond which skin distoron and pain can occur. As a re- promong tumor angiogenesis. A fully human mAb against ALK-1 was sult, most biotech companies spend much effort in concentrang MAbs to ≈100

BIOLOGICS CHANNEL generated. The differenal and complimentary outcome of an-ALK-1 mg/mL and then trying to stabilize these formulaons to avoid aggregates and and an-VEGF will be discussed. parculates. Halozyme’s Enhanze™ Technology permits the large volume subcutaneous (SC) dosing, with corresponding lower protein concentraon which was not previously feasible. Bypassing high-concentraon formulaon challenges 12:10 PM Opmizing Targeng of An-tumor Anbodies has the potenal to accelerate the meline to bring a product to the clinic, Gregory P. Adams, Ph.D., Co-Leader, Molecular and Translaonal Medicine Program, Fox enables an IV-SC switch, in some cases improves bioavailability, and improve Chase Cancer Center paent convenience and compliance. In this talk, large volume SC delivery and We have found that an-HER2 scFv molecules penetrate and localize in device opons will be presented. a solid tumors less efficiently with increasing affinity. We will describe studies performed with an-HER2 human IgGs molecules that dem- 4:20 Recepon in the Exhibit Hall (Sponsorship Available) onstrate that affinity also impacts the targeng of intact anbodies. 5:20 BREAK-OUT DISCUSSIONS in the Exhibit Hall Studies examining the roles of affinity on in vitro ADCC and internalizaon into tumor cells will also be discussed. 6:20 Close of Day

12:40 Luncheon Presentaons (Sponsorship Opportunity Available) THURSDAY, FEBRUARY 4 or Lunch on Your Own 1:45 Dessert in the Exhibit Hall ENGINEERING FOR DELIVERY SELECTIVE TARGETING OF TUMORS 8:25 AM Chairperson’s Remarks 2:15 Chairperson’s Remarks Tugrul Kararli, Ph.D., President & Founder, Pharmacircle Mahew K. Robinson, Ph.D., Assistant Professor, Molecular and Translaonal Medicine 8:30 Tumor Targeng Theory-Kinec & Diffusive Processes Program, Fox Chase Cancer Center that Determine Anbody Macro & Microdistribuon 2:20 Bispecific Anbodies: An Approach to Enhance Targeng K. Dane Wirup, Ph.D., C.P. Dubbs Professor, Chemical Engineering & Biological Selecvity and Efficacy Engineering, Associate Director, Koch Instute for Integrave Cancer Research, Mahew K. Robinson, Ph.D., Assistant Professor, Molecular and Translaonal Medicine Massachuses Instute of Technology CANCER & Program, Fox Chase Cancer Center A diverse array of tumor targeng agents ranging in size from pepdes to Work will be presented on our efforts to develop and opmize the nanoparcles is currently under development for applicaons in cancer targeng selecvity of bispecific anbodies that co-target two disnct imaging and therapy. However, it remains largely unclear how size differ- tumor associated angens. We hypothesize that the targeng selecv- ences among these molecules influence their targeng properes. Here

30 Online: Tri-Conference.com Email: [email protected] Fax: 781-972-5425  MED001710 we develop a simple, mechanisc model that can be used to understand PLENARY KEYNOTE SESSION and predict the complex interplay between molecular size, affinity, and tumor uptake. 2:15 Plenary Keynote Introducon 9:00 Nanoparcle Agents for Tumor Targeng and 2:25 Plenary Kenynote Presentaon (See Page 26 for Details) Penetraon 3:05 Refreshment Break in the Exhibit Hall Shuming Nie, Ph.D., Wallace H. Coulter Disnguished Faculty Chair in Biomedical Engineering, Director of Emory-Georgia Tech Cancer Nanotechnology Center, Professor NOVEL MODES OF ACTION FOR CANCER of BME. Chemistry, Materials Science and Engineering, and Hematology and Oncology, BIOTHERAPEUTICS Emory University and Georgia Instute of Technology BI AND TRISPECIFIC ANTIBODIES Nanoparcles have funconal and structural properes not available from discrete molecules or bulk materials. When conjugated with 3:45 Chairperson’s Remarks monoclonal anbodies, pepdes or small molecules, nanoparcles can Stefan Dübel, Ph.D., Director, Biotechnology, Technische Universität Braunschweig, be used to target malignant tumors with high specificity and affinity. Germany We developed a new class of biocompable and nontoxic nanopar- 3:50 Mode of BiTE Anbody Acon in Cancer Therapy in vivo cles for tumor targeng and detecon based on self-assembled Patrick Baeuerle, Ph.D., CSO and Senior Vice President, Research & Development, nanostructures and pegylated colloidal gold. Micromet AG BiTE (bispecific T cell engager) anbody blinatumumab targets CD19 on 9:30 Delivery of Anbodies – Market Analysis & Overview B cell malignancies and has provided clinical proof of concept in phases Tugrul Kararli, Ph.D., President & Founder, Pharmacircle 1 and 2. We will discuss the mode of BiTE anbody acon in inducing 10:00 Sponsored Presentaon (Sponsorship Opportunity Available) highly efficient cancer cells lysis, and provide background on how BiTE anbodies are produced, are administered in the clinic, and have been 10:30 Poster Compeon Refreshment Break & Raffles in the pre-clinically developed. Exhibit Hall DELIVERY OF ANTIBODIES 4:20 Catumaxomab (Removab), the First EC-approved Trifunconal Bispecific Anbody: The Road from Pre-clinical 11:30 Engineered Anbodies for Molecular Imaging of Cancer Development to Approval and Beyond Anna M. Wu, Ph.D., Professor, David Geffen School of Medicine at UCLA Diane Seimetz, Ph.D., M.D.R.A., CSO and Execuve Vice President, Fresenius Biotech Cancer-targeng anbodies have been opmized for in vivo imaging by GmbH conversion into fragments such as diabodies, minibodies, and scFv-Fc. Catumaxomab, a targeted therapy for intraperitoneal treatment of ma- Recombinant fragments recognizing a variety of cell-surface markers lignant ascites, targets the epithelial cell-adhesion molecule (EpCAM) have been labeled with positron-eming radionuclides (I-124, Cu-64, and CD3 evoking T-cell cytotoxicity on EpCAM-expressing tumor cells. F-18) for positron-emission tomography (PET) detecon of tumors in The Fc-region of catumaxomab provides a third funconal binding site, preclinical models. ImmunoPET represents a broad plaorm for con- which binds and acvates Fcγ-receptor-posive accessory cells. The ducng “immunohistochemistry in vivo” to address biological quesons development raonale, pre-clinical and clinical data, approval process in living organisms, including target expression, target coverage, and and preparaons for further clinical development will be presented. response to therapy. 4:50 Bispecific EGFR-IGF1R Program 12:00 Advanced Polymer Conjugate Technology for Eric Furfine, Ph.D., Senior Vice President, Research and Pre-clinical Development, Adnexus Therapeucs, a Bristol-Myers Squibb R&D Company BIOLOGICS CHANNEL Op miza on of Cancer Therapeu cs Adnecns offer several potenal advantages compared to tradional Chrisne Loehrlein, Ph.D., A.D., New Products and Technology Strategy Research, Nektar Therapeucs targeted biologics, including speed of discovery, efficient manufactur- Conjugaon of a therapeuc agent to polyethylene glycol and other ing, and the ability to create mul-funconal targeted products. We polymers is a general strategy that can be used to opmize pharma- are currently advancing products combining two Adnecns to enable cological parameters of that drug, with the ability to affect both its ef- modulaon of two disnct targets. We will present methods to engi- ficacy and side effect profile. Nektar’s Advanced Polymer Conjugate neer and opmize mul-specific Adnecns, and pre-clinical data on a Technology plaorm can be used to enable a wide range of molecules, bispecific Adnecn to EGFR and IGF1R. including proteins, pepdes, small molecule oral and parenteral drugs, and anbody fragments. Nektar is currently using this approach to 5:20 Mul-specific Anbody by Design develop advanced oncolycs with sustained exposure to tumor cells, Changshou Gao, Ph.D., Principal Scienst, Anbody Discovery and Protein Engineering, MedImmune LLC and exploring opportunies to extend this technology to other cancer We’ll discuss efforts to engineer and opmize mulspecific anbody therapeucs. formats to address the challenges pertaining to bispecific and mulspecific molecules, and provide data of our mulspecific constructs with 12:30 Luncheon Presentaon (Sponsorship Opportunity Available) or excellent expression level, great biophysical stability, good in vitro and in Lunch on Your Own vivo acvies, and potenal manufacturing feasibility. Our mulspecific 1:45 Ice Cream Refreshment Break in the Exhibit Hall constructs retain similar in vivo half-life and effector funcons to their parental anbodies.

5:50 Close of Day CANCER &

Online: Tri-Conference.com Email: [email protected] Fax: 781-972-5425 31  MED001711 FRIDAY, FEBRUARY 5 John F. McDonald, Professor and Director, Integrated Cancer Research Center, School of Biology, Georgia Instute of Technology Targeted cancer therapy by RNA interference (RNAi) is a promising ADVANCES IN ANTIBODYDRUG CONJUGATES approach to silence genes in vivo. Delivery is a major hurdle for the development of RNAi therapeucs. We report on the successful use of 8:30 AM Chairperson’s Opening Remarks hydrogel nanoparcles (nanogels) funconalized with pepdes that Hans-Peter Gerber, Ph.D., Senior Director, Tumor Therapies, Wyeth Oncology Discovery specially target the EphA2 receptor to deliver small interfering RNAs 8:35 A Novel Minor Groove Binding Alkylang Agent for (siRNAs) targeng EGFR to increase sensivity to Taxane therapy. Anbody Targeted Chemotherapy of CD70 Expressing 12:00 PM Luncheon Presentaon (Sponsorship Opportunity Avail- Cancers able) or Lunch on Your Own Nils Lonberg, Ph.D., Senior Vice President and Scienfic Director, Medarex, Inc. An anbody drug conjugate comprising a CD70 targeng monoclonal anbody and a novel alkylang agent is now in Phase I clinical devel- EFFECTORENHANCED BIOTHERAPEUTICS opment for kidney cancer and lymphoma. The mechanism of acon of this novel therapeuc, acvated through a mulstep mechanism 1:00 Chairperson’s Remarks including esterase mediated removal of a blocking group and pro- John F. McDonald, Professor and Director, Integrated Cancer Research Center, School of Biology, Georgia Instute of Technology tease mediated release of the cytotoxic drug, will be discussed. 1:05 Human RNase Fusion Proteins for Tumor Therapy 9:05 Pre-clinical and Clinical Development of Calicheamicin Stefan Dübel, Ph.D., Director, Biotechnology, Technische Universität Braunschweig, Derivaves Conjugated to Monoclonal Anbodies Germany Hans-Peter Gerber, Ph.D., Senior Director, Tumor Therapies, Wyeth Oncology Discovery RNases are non-toxic while in circulaon but highly effecve in cell Gemtuzumab ozogamicin (Mylotarg), a semi-synthec derivave of killing aer targeted internalizaon. An enrely human immunoen- calicheamicin linked to a humanized an-CD33 monoclonal anbody, zyme against CD30+ lymphomas was constructed from a human is approved for the treatment of AML. CMC-544 (inotuzumab ozo- scFv-Fc anbody fragment and a human RNase. It did not affect the gamicin), an an-CD22 immuno-conjugate of calicheamicin, is cur- human embryonlal kidney used for its producon but strongly inhib- rently being evaluated in B-cell non-Hodgkin’s lymphoma (B-NHL) ited proliferaon of CD30+ lymphoma cells. paents. I will describe the mechanism of acon and the pharmacology of calicheamicin conjugates and provide an overview of clinical 1:35 Glycoengineering for the Enhancement of Anbody trials. Acvity Dennis Benjamin, Ph.D., Senior Director, Anbody Technologies, Seale Genecs, Inc. 9:35 Anbody-Maytansinoid Conjugates: Demonstrang 2:05 Anbody Fc Engineering to Enhance Cytotoxicity, Benefit in the Treatment of Solid and Liquid Tumors Pharmacokinecs, and Pharmacodynamics John M. Lambert, Ph.D., Execuve Vice President and CSO, ImmunoGen, Inc. John R. Desjarlais, Ph.D., VP, Research, Xencor, Inc. Several new highly potent cell-killing agents such as derivaves of We have engineered the anbody Fc domain to enhance its affinity for the an-mitoc microtubule agent, maytansine, are currently being Fc receptors, leading to a set of variants that confer high ADCC acv- ulized in ADCs to achieve effecve, well tolerated ancancer drugs. ity onto anbodies targeng a wide range of tumor targets. These vari- Several AMCs show encouraging efficacy in clinical trials, including ants also enhance an-tumor acvity in mouse models and cynomolgus T-DM1, currently being developed by Genentech using ImmunoGen’s monkeys. A phase I trial is underway to determine their effects in hu- maytansinoid technology. New payloads for ADCs are realizing the mans. promise of anbody-mediated delivery in cancer. BIOLOGICS CHANNEL 2:35 ArzerraTM (ofatumumab), a Novel Human Therapeuc 10:05 Broad Applicaon of Scaffold Anbodies for Targeted CD20-Anbody: Mechanisms of Acon and Efficacy in B-CLL Tumor Therapy Frank Beurskens Ph.D., Senior Scienst, Strategic Research, Genmab, B.V. Gary Woodnu, Ph.D., Vice President,Biology, CovX Research, A Pfizer We developed a unique human monoclonal CD20 anbody, ofatu- organizaon mumab, that targets a disnct epitope encompassing both the small The progression of novel cancer therapeucs that have the potenal of and large extracellular loops of the CD20 molecule and displays an truly impacng disease requires the idenficaon of targets that affect tu- excep onal e cacy in inducing complement dependent cell lysis mor growth combined with a modality capable of rapid exploitaon of those ffi targets either as monotherapy or in combinaon. We will describe how the (CDC). Novel insights into the mechanisms of tumor cell killing by ofa- use of bioconjugaon to a proprietary scaffold anbody allows us to develop tumumab and its efficacy in clinical trials in B-CLL will be discussed. these therapeucs rapidly and effecvely. 3:05 Close of Conference 10:20 Coffee Break EMERGING NEW TECHNOLOGIES

11:00 Bi-specific, High Affinity T Cell Receptor Fusions as An-Cancer Therapeucs Rebecca Ashfield, D.Phil., Senior Research Project Manager, Immunocore Ltd Bi-specific TCRs, consisng of high affinity T cell receptors fused to an an-CD3 scFv, are being developed for the treatment of several tu- Receive mor types. The presentaon will cover engineering of these reagents, ALUMNI DISCOUNT demonstraon of efficacy including animal models, and a discussion Cambridge Healthtech Institute (CHI) appreciates your past 20% Off Your participation at the Molecular Medicine Tri-Conference. Through loyalty of the planned first-in-man clinical trial including toxicity tesng, a like yours, CHI has been able to build this event into a must attend for Registration! challenge since the molecules are enrely human specific. senior level decision-makers. As a result of the great loyalty you have shown us, we are pleased to extend to you the exclusive opportunity to save an additional 20% off the registration rate. Just check off the CANCER & box marked Alumni Discount on the registration form to receive the 11:30 Chemosensizaon of Cancer Cells by siRNA Using Targeted discount! Please note: Our records must indicate you were an attendee Nanogel Delivery of the Tri-Conference event in the past in order to qualify.

Online: Tri-Conference.com Email: [email protected] Fax: 781-972-5425 32  MED001712 Cambridge Healthtech Institute’s Inaugural Delivery of Biologics

WEDNESDAY, FEBRUARY 3 2:20 Bispecific Anbodies: An Approach to Enhance Targeng Selecvity and Efficacy Mahew K. Robinson, Ph.D., Assistant Professor, Molecular and Translaonal Medicine 7:00 AM Registraon and Morning Coffee Program, Fox Chase Cancer Center Work will be presented on our efforts to develop and opmize the 8:00 Plenary Keynote Session (See Page 26 for Details) targeng selecvity of bispecific anbodies that co-target two disnct 9:40 Grand Opening Refreshment Break in the Exhibit Hall tumor associated angens. We hypothesize that the targeng selecv- PENETRATION AND DISTRIBUTION ity afforded by these molecules can potenally be leveraged for the development of new immunodrug conjugates. 11:00 Introducon and Welcome Gregory P. Adams, Ph.D., Co-Leader, Molecular and Translaonal Medicine Program, Fox 2:50 Selecve Penetraon and Targeng of Tumors Chase Cancer Center Tapas K. Das Gupta, M.D., Ph.D., D.Sc., Professor and Head, Surgical Oncology, University of Illinois Chicago; Co-founder, CDG Therapeucs, Inc. 11:10 Tumor Penetraon of Therapeuc Anbodies -The CDG Therapeucs has developed a cell penetrang pepde (28aa) Impact of Size and Exposure Time on Distribuon from azurin, a redox protein secreted by Pseudomonas aeruginosa. David Blakey, Ph.D., Chief Scienst, Cancer and Infecon Research Area, AstraZeneca p28 preferenally enters cancer cells, localizes in the nucleus and sta- The ability of intact anbodies and fragments to access tumor cells bilizes p53 inducing cell cycle arrest and apoptosis in a series of solid distant from the tumor blood supply is an important therapeuc con- tumors. p28 is stable, nontoxic and currently in a Phase I clinical trial. sideraon for anbody based oncology drugs. Preclinical and clinical data will be reviewed regarding the impact of size and exposure me 3:20 A Systems Biology Approach to Engineering Therapeuc on anbody distribuon within tumors. Anbodies: Development of an ErbB2/ErbB3 Bispecific Anbody 11:40 An-tumor Efficacy Maximizaon through Blocking Alexandra Huhalov, Ph.D., Principal Scienst, Merrimack Pharmaceucals Mulple Targets of Angiogenesis Using quantave biological datasets of cell signaling we have gener- Dana Hu-Lowe, Ph.D., Group Leader, Associate Research Fellow, Cancer Biology, Pfizer, ated computaonal models of the ErbB signaling network and iden- Inc., PGRD-La Jolla fied ErbB3 as a promising target. The applicaon of these models to Vascular normalizaon and adapvety potenally contribute to resis- guide the design of MM-111, a bispecific anbody-based therapeuc tance to an-VEGF/VEGFR therapies in the clinic. Other targets, in- targeng the ErbB2/ErbB3 heterodimer, and its antumor acvity will cluding the Acvin receptor Like Kinase 1 (ALK-1), also play a role in be discussed. promong tumor angiogenesis. A fully human mAb against ALK-1 was generated. The differenal and complimentary outcome of an-ALK-1 3:50 Sponsored Presentaon (Sponsorship Opportunity Available) and an-VEGF will be discussed. 4:20 Recepon in the Exhibit Hall (Sponsorship Available) 12:10 PM Opmizing Targeng of An-tumor Anbodies 5:20 BREAK-OUT DISCUSSIONS in the Exhibit Hall Gregory P. Adams, Ph.D., Co-Leader, Molecular and Translaonal Medicine Program, Fox Chase Cancer Center 6:20 Close of Day We have found that an-HER2 scFv molecules penetrate and localize in a solid tumors less efficiently with increasing affinity. We will describe THURSDAY, FEBRUARY 4 studies performed with an-HER2 human IgGs molecules that demonstrate that affinity also impacts the targeng of intact anbodies. Studies examining the roles of affinity on in vitro ADCC and internaliza- ENGINEERING FOR DELIVERY on into tumor cells will also be discussed. 8:25 AM Chairperson’s Remarks 12:40 Luncheon Presentaon (Sponsorship Opportunity Available) or Tugrul Kararli, Ph.D., President & Founder, Pharmacircle BIOLOGICS CHANNEL Lunch on Your Own 8:30 Tumor Targeng Theory-Kinec & Diffusive Processes 1:45 Dessert in the Exhibit Hall that Determine Anbody Macro & Microdistribuon K. Dane Wirup, Ph.D., C.P. Dubbs Professor, Chemical Engineering & Biological SELECTIVE TARGETING OF TUMORS Engineering, Associate Director, Koch Instute for Integrave Cancer Research, Massachuses Instute of Technology 2:15 Chairperson’s Remarks A diverse array of tumor targeng agents ranging in size from pepdes Mahew K. Robinson, Ph.D., Assistant Professor, Molecular and Translaonal Medicine to nanoparcles is currently under development for applicaons in Program, Fox Chase Cancer Center cancer imaging and therapy. However, it remains largely unclear how size differences among these molecules influence their targeng properes. Here we develop a simple, mechanisc model that can be used to understand and predict the complex interplay between molecular size, affinity, and tumor uptake.

9:00 Nanoparcle Agents for Tumor Targeng and Penetraon

33 Online: Tri-Conference.com Email: [email protected] Fax: 781-972-5425  MED001713 Shuming Nie, Ph.D., Wallace H. Coulter Disnguished Faculty Chair in Biomedical naked molecules are able to potently and safely inhibit therapeucally Engineering, Director of Emory-Georgia Tech Cancer Nanotechnology Center, Professor aracve miRNAs in a range of ssues in experimental animals. The of BME. Chemistry, Materials Science and Engineering, and Hematology and Oncology, presentaon will provide an update on the unique features of LNA oli- Emory University and Georgia Instute of Technology gonucleodes in miRNA therapeucs with parcular emphasis on the Nanoparcles have funconal and structural properes not available pre-clinical and clinical development of SPC3649, an LNA AnmiR tar- from discrete molecules or bulk materials. When conjugated with geng miRNA-122. monoclonal anbodies, pepdes or small molecules, nanoparcles can be used to target malignant tumors with high specificity and affinity. 4:20 Pre-clinical and Clinical Development of Atu027 (siRNA- We developed a new class of biocompable and nontoxic nanoparcles for in vivo tumor targeng and detecon based on self-assembled lipoplex/AtuPLEX) for Oncology nanostructures and pegylated colloidal gold. Klaus Giese, Ph.D., CSO, Silence Therapeucs plc Atu027 refers to a liposomally formulated siRNA targeng PKN3 expression in the vascular endothelium. Pre-clinical studies in rodents and 9:30 Delivery of Anbodies – Market Analysis & Overview non-human primates demonstrated that intravenous administraon is Tugrul Kararli, Ph.D., President & Founder, Pharmacircle well tolerated and gives rise to RNAi-mediated suppression of PKN3 10:00 Sponsored Presentaon (Sponsorship Opportunity Available) gene expression. Various proof-of-concept experiments on mouse tu- 10:30 Poster Compeon Refreshment Break & Raffles in the mor xenogras suggest profound inhibion of tumor progression and parcularly of metastasis, which laid the foundaon for therapeuc ap- Exhibit Hall plicaon in oncology. Atu027 is currently tested in a Phase-I clinical trial DELIVERY OF ANTIBODIES on subjects with advanced solid cancers. Pre-clinical data emphasizing pharmacological acvity in mouse models and an update on the cur- 11:30 Engineered Anbodies for Molecular Imaging of Cancer rent Phase-I study will be discussed. Anna M. Wu, Ph.D., Professor, David Geffen School of Medicine at UCLA Cancer-targeng anbodies have been opmized for in vivo imaging by 4:50 Talk Title to be Announced conversion into fragments such as diabodies, minibodies, and scFv-Fc. Ian MacLachlan, Ph.D., CSO, Tekmira Pharmaceucals Recombinant fragments recognizing a variety of cell-surface markers 5:20 Talk Title to be Announced have been labeled with positron-eming radionuclides (I-124, Cu-64, Bob D. Brown, Ph.D., Senior Vice President, Research, Dicerna Pharmaceucals Corp. F-18) for positron-emission tomography (PET) detecon of tumors in preclinical models. ImmunoPET represents a broad plaorm for con- 5:50 Close of Day ducng “immunohistochemistry in vivo” to address biological quesons in living organisms, including target expression, target coverage, and FRIDAY, FEBRUARY 5 response to therapy.

12:00 Advanced Polymer Conjugate Technology for NOVEL FORMULATIONS FOR RNAI DELIVERY Opmizaon of Cancer Therapeucs 8:30 AM Chairperson’s Opening Remarks Chrisne Loehrlein, Ph.D., A.D., New Products and Technology Strategy Research, Nektar C. Sashchandran, Ph.D., Chief Technology Officer, Research Technology Center, Pfizer, Therapeucs Inc. Conjugaon of a therapeuc agent to polyethylene glycol and other polymers is a general strategy that can be used to opmize pharma- 8:35 Therapeuc siRNA Delivery: Tackling the 800 cological parameters of that drug, with the ability to affect both its ef- Pound Gorilla ficacy and side effect profile. Nektar’s Advanced Polymer Conjugate Steven F. Dowdy, Ph.D., Invesgator, Howard Hughes Medical Instute; Professor, Technology plaorm can be used to enable a wide range of molecules, Department of Cellular & Molecular Medicine, University of California, San Diego School including proteins, pepdes, small molecule oral and parenteral drugs, of Medicine and anbody fragments. Nektar is currently using this approach to To date, siRNA delivery remains the rate-liming step for RNAi thera- develop advanced oncolycs with sustained exposure to tumor cells, peucs development. We developed a Pepde Transducon Domain- and exploring opportunies to extend this technology to other cancer dsRNA Binding Domain (PTD-DRBD) fusion protein siRNA delivery ap- therapeucs. proach. PTD-DRBD delivered siRNAs induced RNAi responses in the enre populaon of all cell types assayed (primary and tumorigenic) 12:30 Luncheon Presentaon (Sponsorship Opportunity Available) or in a non-cytotoxic fashion. PTD-DRBD combinatorial delivery of EGFR Lunch on Your Own and Akt2 siRNAs induced a synthec lethal response that significantly increased survival of intracerebral glioblastoma pre-clinical models. 1:45 Ice Cream Refreshment Break in the Exhibit Hall These observaons demonstrate the ability of PTD-DRBD to efficiently

PLENARY KEYNOTE SESSION delivery siRNAs. BIOLOGICS CHANNEL

2:15 Plenary Keynote Introducon 9:05 Inducon of Therapeuc Gene Silencing in Leukocyte- Implicated Diseases by Targeted and Stabilized Nanoparcles 2:25 Plenary Kenynote Presentaon (See Page 26 for Details) Dan Peer, Ph.D., Head, Laboratory of Nanomedicine, Department of Cell Research and Immunology and the Center for Nanoscience and Nanotechnology, Tel Aviv University 3:05 Refreshment Break in the Exhibit Hall Leukocytes are among the most difficult cells to transduce with RNAi. CLINICAL CHALLENGES WITH RNAI We developed a strategy that can target different subsets of leukocytes THERAPEUTICS and selecvely silence genes in vivo using targeted, stabilized nanoparcles (tsNPs). These carriers do not induce lymphocyte acvaon, in- 3:45 Chairperson’s Remarks terferon responses or release liver enzymes and are fully degradable. Bob D. Brown, Ph.D., Senior Vice President, Research, Dicerna Pharmaceucals Corp. Three preclinical examples inflammatory bowel disease (IBD), blood cancer and viral infecon will be discussed. We will show that tsNPs 3:50 LNA Anmirs – Pioneering microRNA Therapeucs can be used for in vivo validaon of new drug targets, for prevenon of Henrik Orum, M.Sc., Ph.D., VP and CSO, Santaris Pharma Short, single stranded LNA oligonucleodes delivered systemically as viral infecon and for inducing therapeuc gene silencing in a preclinical seng.

Online: Tri-Conference.com Email: [email protected] Fax: 781-972-5425 34  MED001714 9:35 Characterizaon of Immune Responses to tkRNAi Therapeucs Johannes Fruehauf, M.D., Ph.D., VP, Research, Cequent Pharmaceucals, Inc. Transkingdom RNA interference (tkRNAi) describes a novel method for delivery of therapeuc RNA interference into gastrointesnal ssues using engineered bacteria which produce and deliver mediators of RNAi. Clinical trials are about to begin for the prevenon of colon Polypo- sis, and for the treatment of Inflammatory Bowel Disease (IBD). Here we demonstrate recent results from large screening efforts characterizing the effects of tkRNAi on cytokine profiles and innate immunity.

10:05 Sponsored Presentaon (Sponsorship Opportunity Available) 10:20 Coﬀee Break 11:00 Panel: Do We Understand the Challenges We Face With RNAi Therapeucs? Panelists: Bob D. Brown, Ph.D., Senior Vice President, Research, Dicerna Pharmaceucals Corp. Dmitry Samarsky, Ph.D., VP, Technology Development, RXi Pharmaceucals Ian MacLachlan, Ph.D., CSO, Tekmira Pharmaceucals John Rossi, Ph.D., Professor and Chair, Molecular Biology, Beckman Research Instute of the City of Hope Steven Highlander, Ph.D., Partner, Intellectual Property, Fulbright & Jaworski, L.L.P.

12:00 PM Luncheon Presentaon (Sponsorship Opportunity Available) or Lunch on Your Own NOVEL APPROACHES FOR TARGETED DELIVERY

1:00 Chairperson’s Remarks John Rossi, Ph.D., Professor and Chair, Molecular Biology, Beckman Research Instute of the City of Hope

1:05 In vivo delivery of Dicer substrate RNAs for treatment of HIV infecon John Rossi, Ph.D., Professor and Chair, Molecular Biology, Beckman Research Instute of the City of Hope The applicaon of RNAi for treatment of HIV infecon has many advantages over convenonal drugs. The inhibitors can be rapidly changed according to the viruses ability to mutate. This presentaon will discuss the use of aptamers and dendrimers to deliver Dicer substrate RNAs in vivo. The results obtained demonstrate that Dicer substrate siRNAs can be delivered in a cell type speciﬁc manner with an aptamer, and can be generally delivered with dendrimers to eﬀecvely inhibit HIV replicaon in a humanized mouse model.

1:35 Targeted RNA-based Cancer Therapies Paloma H. Giangrande, Ph.D., Assistant Professor, Department of Internal Medicine, University of Iowa A major hurdle for the clinical translaon of siRNAs into effecve therapies is delivery. We describe an RNA aptamer-based approach for the targeted delivery of siRNAs to prostate cancer (PC) cells. The aptamer-siRNA reagent (chimera) is effecve when administered systemically and is suitable for efficient chemical synthesis. When administered systemically to mice bearing PSMA-posive tumors, the RNA chimera triggered tumor regression without affecng normal ssues. This work is the first descripon of in vivo efficacy following systemic administraon of an aptamer-siRNA chimera and thus represents a milestone for this plaorm technology.

2:05 Development of Novel Therapeuc RNAi Compounds and Eﬀecve in vivo Delivery Approaches Dmitry Samarsky, Ph.D., VP, Technology Development, RXi Pharmaceucals RNA interference (RNAi) oﬀers a novel approach to the drug development process, because RNAi compounds can potenally be designed to target any one of the genes in the human genome. Other potenal advantages of RNAi therapeucs include, rapid development of lead compounds, high selecvity for the target gene, high potency (low dose) and low toxicity due to natural mechanism of acon. We will introduce unique single-oligo (rxRNAsolo) and short-duplex (rxRNAnano) RNAi compounds, as well as novel in vivo delivery approaches, including self-delivering rxRNA molecules (sd-rxRNA) for local and systemic delivery, and targeted delivery to phagocyc immune cells using.

2:35 Talk Title to be Announced C. Sashchandran, Ph.D., Chief Technology Oﬃcer, Research Technology Center, Pﬁzer, Inc. 3:05 Close of Conference BIOLOGICS CHANNEL

Online: Tri-Conference.com Email: [email protected] Fax: 781-972-5425 35  MED001715 Cambridge Healthtech Institute’s Sixth Annual Translational Medicine Completing the Circle: Bedside Back to Bench

WEDNESDAY, FEBRUARY 3 Translaonal Medicine, Wyeth Research Labs Lise Kjems, M.D., Ph.D., Execuve Director, Global Program DiagnoscDirector, Molecular Diagnoscs Novars Francesco Marincola, M.D., Chief, Infecous Disease and 7:00 AM Registraon and Morning Coffee Immunogenecs Secon, NIH; Editor-in-Chief, Journal of Translaonal MedicineWilliam B. Maes, PhD, DABT, PharmPoint Consulng, Former Execuve Director, Predicve 8:00 Plenary Keynote Session (See Page 26 for Details) Safety Tesng Consorum Yali Fu, Ph.D., Program Director, Grants and Contracts Operaons Branch, 9:40 Grand Opening Refreshment Break in the Exhibit Hall Developmental Therapeucs Program, Division of Cancer Treatment and Diagnosis, TRENDS IN TRANSLATIONAL MEDICINE Naonal Cancer Instute Hans Winkler, Ph.D., Senior Director, Global Head Oncology Biomarkers, Johnson & Johnson 11:00 Chairperson’s Remarks Chrisna M. Coughlin, M.D., Ph.D.; Medical Director, Oncology; Clinical Research and 12:40 Luncheon Presentaon I Sponsored by Development; Pfizer Oncology ZDSD Rat: A Model for Diabetes, Metabolic Syndrome, and 11:10 Translaonal Approach to Studying Stroke Obesity without Lepn or Lepn Receptor Mutaons Giora Z. Feuerstein, M.D., Assistant VP, Discovery Translaonal Medicine, Wyeth Richard G. Peterson, Ph.D.; Execuve VP Research & Development, PreClinOmics, Inc. Research Labs The ZDSD rat is a model for obesity, insulin resistance, metabolic syndrome To improve success of clinical trials and speed drug development, de- and diabetes with a normal lepn axis. partments of Translaonal Medicine in pharma have formed to figure The disease condions expressed in the ZDSD more closely resemble the hu- out the which molecular, biochemical and physiological biomarkers man situaon when compared to other animal models. can best substute for the absence of cinical outcome studies. My The obesity and diabetes in the ZDSD rat can be modulated with diet. presentaon will illustrate how we’ve applied a translaonal approach The obesity and diabetes in the ZDSD rat can be treated with standard pharmaceucals. to develop beer therapies for stroke. Speci cally we’ve focused on fi The ZDSD rat expresses the bone, renal and other complicaons seen in diabetes. reducing arion rate of compounds/biologicals by opmizing 1. Tar- get Validaon; 2. Compound -Target interacon; 3. innovave Pharma- 1:10 Luncheon Presentaon II (Sponsorship Opportunity Available) cokinec-Pharmacodynamic and proof of Mechanism of Acon (MoA); 1:45 Dessert in the Exhibit Hall 4. disease biomarkers; 5. Paent selecons for clinical trials based on evidence for likelihood to respond to treatment. WORKING BACKWARDS IN CANCER: FROM THE CLINIC TO DISCOVERY 11:40 Contribuon of Translaonal Approaches to Recent Advances in Immuno-therapeucs, Immuno-rejecon and Beyond 2:15 Chairperson’s Remarks Francesco Marincola, M.D., Chief, Infecous Disease and Immunogenecs Secon, NIH; Editor Michael Liebman, Ph.D., Managing Director, Strategic Medicine, Inc. in Chief, Journal of Translaonal Medicine 2:20 Using Drug-Induced Feedback Loops to Idenfy The complexity underlying a pathological process does not necessarily require complex soluons. The biology determining allogra or can- Indicaons and Combinaon Partners Donald Bergstrom, Ph.D., Director, Experimental Medicine Oncology, Merck cer rejecon, autoimmunity or ssue damage during pathogen infec- James W. Waers, Ph.D., Associate Director, Molecular Profiling Oncology, Merck ons is complex; however, common paerns are emerging that lead Treatment with molecular targeted agents can result in compensatory to a common final outcome: ssue destrucon with resoluon of the feedback regulaon as cells respond to inhibion of signaling path- pathogenic process (cancer, infecon) or ssue damage and organ fail- ways. We will present clinical evidence that treatment with a small ure (allogra rejecon, autoimmunity). Human observaons based on molecule inhibitor of gamma secretase results in pathway modulaon transcriponal profiling converge into an “immunological constant of and compensatory feedback, and describe pre-clinical experiments de- rejecon” that signals such occurrences. This constant includes the co- signed to leverage this concept for drug response predicon. ordinate acvaon of interferon smulated genes (ISGs) and immune effector funcons (IEFs). Understanding this nal effector pathway may fi 3:00 Moving Research Closer to the Bedside, in vitro and in vivo suggest novel strategies for the inducon or inhibion of ssue-specif- EXECUTIVE CHANNEL Analyses with Primary Tumors ic destrucon with therapeuc intent in cancer and other immune pa- Fred Poordad, M.D., Chief of Hepatology, Liver Disease and Transplant Center, thologies. This presentaon will discuss how vaccines may play a role Cedars-Sinai Medical Center, Xin Wei Wang, Ph.D., Senior Invesgator Head, Liver in ssue-specific destrucon and use this as a model to demonstrate Carcinogenesis Secon, Laboratory of Human Carcinogenesis, Naonal Cancer Instute, how to understand the dynamics of therapeucs by studying target NIH, and Michael R. Briggs, Ph.D., Senior Director, Biology, Vertex Pharmaceucals, Inc. ssues in real me . The incidence of Primary Liver Cancer is increasing in the west and constutes a tremendous burden on world health as the third lead- 12:10 PM Panel: Praccal Translaonal Medicine ing cause of cancer deaths worldwide. The 5 year survival rate is a Moderator: Vivek Kadambi, Ph.D., Senior Director, Millenium Pharmaceucals dismal 11 %, due in large part to late diagnosis and limited treatment When to use biomarkers for go/no-go decisions on proceeding with develop- opons. The eology of this devastang disease as well as current and ment of a clinical compound proposed new therapies wil be discussed. Steps to beer diagnose How has translaonal medicine changed over the past 5 years? and strafy paents for targeted therapy will be considered as a new Fostering partnerships between industry, academics and government grant- ing instuons and excing phase of cancer research. Finally, a move toward more How are pharmaco-dynamic and predicve markers being used right now in relevant research will be presented as an hypothesis that will be tested clinical development? in the coming years as more new and current therapies are compared Panelists: Giora Z. Feuerstein, M.D., Assistant Vice President and Head, Discovery and contrasted to current best pracce.

36 Online: Tri-Conference.com Email: [email protected] Fax: 781-972-5425  MED001716 4:05 How do you go from Pathways to Sponsored by 9:00 Animal Imaging for Translaonal Approaches to CNS Clinical Outcomes? Drug Discovery Aris Persidis, Ph.D., President, Biovista, Inc. Rudy Schreiber, Ph.D., Senior Director, Pharmacology, Discovery and Early Clinical Research In drug discovery and development what what really counts is the In this presentaon, I demonstrate how Sepracor’s translaonal medi- clinical outcome, the Benefit/Risk of the drug within the context cine approach for CNS drug discovery using emerging imaging methods of its pathway or mechanism of acon (MoA). Biovista screens the and collaborave approaches such as cooperave technology develop- MoA of any drug or target against the MoA of 8,000 indicaons and ment within a pain consorum. I’ll present animal SPECT data for our 12,000 adverse events (AEs). This is simultaneous, unbiased indicaon inhibitors of monoamine neurotransmier transporters (serotonin, no- discovery and AE profiling, and it is unique. It helps to bridge the gap radrenaline and dopamine) in rodents and non human primates (with from molecular pathway to clinical outcome in a single step. Case one example of human PET): I will include also brain microdialysis data studies in cancer and other diseases will be described. in rodents where we measured all 3 neurotransmiers, and share human data generated in a fMRI pain imaging consorum. 4:35 Recepon in the Exhibit Hall (Sponsorship Available)l 5:20 BREAK-OUT DISCUSSIONS in the Exhibit Hall 9:30 Image Analysis Consideraons for Pre-clinical, in vivo 1. Novel Imaging Biomarkers in Drug Development Medical Imaging Moderator: Jingsong Wang, M.D., Director of Immunology, Discovery Medicine & Clinical Mahew Silva, Ph.D., Head, Imaging Sciences, Millennium, The Takeda Oncology Pharmacology, Bristol-Myers Squibb, Co. Company The disnct advantage and unique challenges in applying imaging biomarkers in 10:00 Standardized Soluons for Non- Sponsored by drug development The most promising novel imaging biomarkers for drug development, and Invasive Imaging of Cell Trafficking which therapeuc areas will benefit the most from using imaging biomarker Eric T. Ahrens, Ph.D., Founder and Chief Scienfic The role of pharmaceucal company, CRO, academia and regulatory agency in Officer, Celsense, Inc. the discovery, development and qualificaon of imaging biomarkers A liming factor in the development of new therapies is an inability to non-invasively assay cell trafficking in vivo. In this talk Dr. Eric Ahrens 2. Biomarkers in Translaonal Medicine will describe a unique non-invasive imaging plaorm for visualizing and Co-Moderators: quanfying cells in vivo using magnec resonance techniques. Appli- Chrisna M. Coughlin, M.D., Ph.D.; Medical Director, Oncology; Clinical Research and Development; Pfizer Oncology caons include visualizaon and quanficaon of transplanted cells in Hans Winkler, Ph.D., Senior Director, Global Head Oncology Biomarkers, Johnson & immunotherapy and regenerave medicine and monitoring of inflam- Johnson matory processes. Which type of biomarkers will speed drug development the most? Predicve marker clinical validaon: retrospecve vs. prospecve trial re- 10:30 Poster Compeon Refreshment Break & Raffles in the quirements Exhibit Hall Do we sll need phamaco dynamic analysis in Phase II? CHALLENGES IN TRANSLATIONAL BIOMARKER 3. New Funding Opportunies for Biotechs Moderator: DEVELOPMENT Yali Fu, Ph.D., Program Director, Grants and Contracts Operaons Branch, Developmental Therapeucs Program, Division of Cancer Treatment and Diagnosis, 11:30 Biomarkers for Proof of Concept and Paent Selecon Naonal Cancer Instute Hans Winkler, Ph.D., Senior Director, Global Head Oncology Biomarkers, Johnson & Understand NIH Enhanced Peer Review Johnson NCI’s new iniaves on SBIR funding The talk will comprise a discussion about the value of pharmacody- Phase II Bridge awards to help biotechs further develop their technologies namic markers for proof of principle and decision making in PhaseI/II. 4. New Animal Models in Translaonal Medicine Furthermore, the necessity and ulity of predicve markers that can be Ideal characteriscs for new animal models applied for paent selecon will be discussed. Advantages of new animal models Designing studies and using new animal models in discovery research 12:00 PM Paent Selecon Biomarker Approaches in Co-Moderators: Oncology Richard G. Peterson, Ph.D., Professor Emeritus, Indiana University School of Medicine Daniel S. Johnston, Ph.D., Principal Research Scienst II, Pfizer Research and and EVP, Research and Development PreClinOmics, Inc. Development Troy A. Gobbe, MS, Director, Sales & Markeng for PreClinOmics,Inc. This presentaon will focus on the current strategies to idenfy paent 6:20 Close of Day selecon biomakers in oncology. Both clinical and preclinical approaches will be discussed. THURSDAY, FEBRUARY 4

12:30 Luncheon Presentaon (Sponsorship Opportunity Available) or EXECUTIVE CHANNEL IMAGING: CLINICAL TO PRECLINICAL Lunch on Your Own 8:25 AM Chairperson’s Remarks 1:45 Ice Cream Refreshment Break in the Exhibit Hall Jingsong Wang, M.D., Director, Immunology, Discovery Medicine & Clinical PLENARY KEYNOTE SESSION Pharmacology, Bristol-Myers Squibb, Co. 8:30 Assessing Mechanism of Acon of Ancancer Agents 2:15 Plenary Keynote Introducon using Funconal Imaging in Oncology Drug Development 2:25 Plenary Keynote Presentaon (See Page 26 for Details) Dana Hu-Lowe, Ph.D., Group Leader, Associate Research Fellow, Cancer Biology, Pfizer, Inc., PGRD-La Jolla 3:05 Refreshment Break in the Exhibit Hall Mulple imaging modalies have helped us to gain a deeper understanding of the mechanism of acon of drug candidates beyond convenonal REDESIGNED ANIMAL MODELS pharmacological end points used in nonclinical and clinical sengs. More importantly, funconal imaging modalies are providing in-depth 3:45 Chairperson’s Remarks informaon on the modes of acon of various an-angiogenesis agents. Alain Stricker-Krongrad, Ph.D., Senior Scienfic Adviser, Global Business Development, Charles River These learnings are vital for improving efficiency in drug development.

Online: Tri-Conference.com Email: [email protected] Fax: 781-972-5425 37  MED001717 3:50 An Animal Model of Parkinson’s Disease Psychosis: daon of such biomarkers is dependent on state-of-the-art bioinfor- Assessing Potenal Therapeuc Efficacy of 5-HT2A Inverse macs analyses as well as assay development. These prerequisites will Agonists ensure idenficaon of biomarkers that are reproducible and hence of Krista McFarland, Ph.D., Team Leader, In Vivo Pharmacology, ACADIA Pharmaceucals, clinical relevance. Inc. Available anpsychoc drugs do not provide an ideal treatment for 9:05 High Content Mining of Disease Biomarkers psychosis in Parkinson’s disease (PDP) because their blockade of dop- Jake Chen, Ph.D., Assistant Professor, Informacs & Computer Science, Indiana amine receptors counteracts the dopamine replacement therapy used University School of Informacs; Director, Indiana Center for Systems Biology and to alleviate the motor symptoms of PD. Development of alternate Personalized Medicine, Indiana University - Purdue University Indianapolis; Founder, pharmacotherapies is limited by the lack of an animal model. Recent MedeoLinx, Inc. efforts to develop such a model and assess the potenal therapeuc To facilitate the interpretaon of raw Omics data into detailed disease- efficacy of 5-HT2A inverse agonists for the treatment of PDP will be specific knowledge of candidate biomarkers, we developed a “high- discussed. content biomarker mining” soware system. The system can help manage and correlate molecular funcons, molecular connecvity, 4:20 Engineered Human-In-Mouse Tumors for Populaon biological pathways, and literature informaon. Its applicaon into the current biomarker development process will help improve the success Based in vivo Biomarker Discovery rate and quality of candidate biomarkers. Min Wu, Ph.D., Principal Scienst, Translaonal Research, AVEO Pharmaceucals, Inc. I will present a NEW populaon-based tumor model system using Hu- man-in-Mouse ssue transgenic human tumors that feature naturally 9:35 Single Molecule Real Time Biology: New technologies occurring tumor variaon akin to that observed in human tumor popu- Enabling a More Complete Characterizaon of Disease laons. The goal is to idenfy and validate biomarkers that ulmately Biology predict responsive versus non-responsive paent populaons to guide Eric Schadt, Ph.D., Chief Scienfic Officer, Pacific Biosciences clinical development. While there has been an explosion of technologies that enable more comprehensive characterizaons of complex biological processes like 4:50 Appropriate Animals Models for Safety Assessment of common human diseases, we are sll unable to glimpse a large enough Biologics fracon of the biology of these systems to build models that are pre- Timothy MacLachlan, Ph.D., Associate Director of Nonclinical Safety Assessment, dicve enough to achieve clinical ulity. However, with a new wave of Genzyme Corporaon technologies on the horizon, providing for the capability to examine The proper safety assessment of biopharmaceucals has been an the acvity of single molecules real me, we will soon be capable of evolving process. While the paradigm set for small molecules was generang the right scale and diversity of data (DNA sequence, RNA applied early, and some aspects have remained the same, other ar- sequence, real me monitoring of mRNA translaon, full character- eas have required modificaon. The specificity of some biologics like izaons of base modificaons in genomes and transcriptomes) at low monoclonal anbodies has necessitated study in higher order species. cost to dramacally enhance the construcon of models for common However, alternaves such as mice transgenic for the human target human diseases that achieve clinical ulity. I will cover the single mol- have proved useful, and at mes, more accurate in risk assessment. ecule real me (SMRT) technologies from Pacific Biosciences and how Examples of these approaches will be discussed. these technologies will revoluonize our ability to characterize living systems, and then present a number of integrave biology approaches 5:20 Comparave Oncology Drug Development to taking the types of data SMRT technologies will generate to get at Melissa C. Paoloni, DVM, DACVIM, Director, Comparave Oncology Program, Naonal predicve models of disease that can be used to drive the idenfica- Instutes of Health, Naonal Cancer Instute on and validaon of drug targets and biomarkers. Comparave oncology is a model system to evaluate novel drugs, devices, biologics and imaging strategies in pet dogs with cancer to help 10:05 Automang Biomarker Discovery Sponsored by inform their development for human cancer paents. Although much and Qualificaon; Capturing Hypothesis, of this effort is preclinical, it also applies to agents that are already Analysis and IP “first in man.” The goal behind it is to garner informaon to help make Jonathan Sheldon, Ph.D., Director of Translaonal Medicine, IDBS informed go and no-go decisions to drive human oncology clinical trial Long lists of un-annotated proteins and genes are not a sufficient end point design by answering quesons about PK, PD, schedule, regime, dose, for ‘omics analysis, they need to be annotated with data from many public and toxicity and clinical outcome (to just name a few). It has been well rec- proprietary sources. IDBS provide soluons to not only automate the discovery ognized and ulized by many within the pharmaceucal industry. It and subsequent annotaon of biomarker results, but to capture each step of also has the ability to pilot personalized medicine approaches-a key to the experimental set up, data capture, and analysis in a compliant manner. the future of oncology drug development. 10:20 Coffee Break 5:50 Close of Day TRANSLATIONAL INFORMATICS HOW FAR EXECUTIVE CHANNEL HAVE WE COME? CONTINUED FRIDAY, FEBRUARY 5 11:00 Profiling Paents to Drive Biomarker Development TRANSLATIONAL INFORMATICS N. R. Nirmala, Ph.D.,Director, Biomarker Analysis and Informacs Unit, Translaonal HOW FAR HAVE WE COME? Sciences, Novars Instutes of Biomedical Research Gene expression profiling is one of the key ways in which a genome- 8:30 AM Chairperson’s Opening Remarks wide view of a paent’s response to drug treatment can be obtained. Pearl S. Huang, Ph.D., Integrator, Oncology Franchise, Research & Early Development, Such a molecular level view can provide strategies for customized ther- Merck & Co. apies in many contexts. In this talk, the oppportunies and challenges that this technology presents will be discussed with a couple of case 8:35 Implemenng a Translaonal Biomarker Strategy to studies. Extension of this approach to other technologies will also be Reduce Arion in Drug Development presented in the context of biomarker development. Irina Antonijevic, M.D. Ph.D., Director, Translaonal Research, Biological Research, Lundbeck Research, Inc. USA Early efforts towards the discovery of molecular biomarkers for CNS disorders are encouraging. However, confirmaon, and ulmately vali-

Online: Tri-Conference.com Email: [email protected] Fax: 781-972-5425 38  MED001718 11:30 Panel: Informacs at R&D Interphases 1:35 Fostering Collaboraons between Biotech and Moderator: Pearl S. Huang, Ph.D., Integrator, Oncology Franchise, Research & Early Academics to Speed Translaonal Medicine Development, Merck & Co. Thomas Ichim, Ph.D., CEO, MediStem Labs, Inc. Linking clinical outcome with molecular data: filling the gaps Capturing uniform clinical language for outcomes 2:05 Commercial Collaboraons and other Approaches Compable and user- friendly data systems—can one size fit all? to Direct Academic Cancer Research towards Clinical Disease cohorts-how many, how big, what is acceptable quality Outcomes 12:00 PM Luncheon Presentaon (Sponsorship Opportunity Clive Stanway, Ph.D., CSO, Cancer Research Technology Ltd., Wolfson Instute for Biomedical Research Available) or Lunch on Your Own Cancer Research Technology (CRT) is the development and commer- CASE STUDIES ON SUCCESSFUL cializaon arm of Cancer Research UK (CR-UK) which has an annual ORGANIZATIONAL COLLABORATIONS AND science spend in excess of $500M.CRT works with CR-UK through SYSTEM APPROACHES mulple tracks to drive translaonal research including dedicated industry experienced, peer-reviewed funding for managed research 1:00 Chairperson’s Remarks in the PI’s laboratory or in collaboraon with focused drug discovery William B. Maes, Ph.D., DABT; Former Execuve Director, Predicve Safety Tesng research groups around the UK. Specific examples and outcomes of Consorum, Crical Path Instute this strategy will be presented with some discussion of CRT’s flexible 1:05 Taking personalized medicine to the next level- The and creave approach to partnerships. crical Interface between Translaonal Medicine and Molecular Diagnoscs 2:35 Development of Combinaon Therapies for Mulple Lise Kjems, M.D. Ph.D., Execuve Director, Global Program DiagnoscDirector, Sclerosis Using Systems Level Informacs Molecular Diagnoscs Novars Frederic S. Young, Ph.D., Chief Scienst, Vicus Therapeucs Translaonal Medicine has reformed our drug development para- We start with a mullevel systems physiology model that combines digm. Early clinical profiling of New Molecular Enes can enable metabolomic analysis with integrated physiological analysis. The model idenficaon of subsets of paents with a unique risk/benefit pro- is used to define a set of systems informac features of ontogeny, phy- file. In this talk, the opportunies and challenges related to this logeny, homeostasis, and repair that disnguishes the disease state from idenficaon will be discussed. The role of Molecular Diagnoscs is homeostasis. We describe our use of this systems informac signature as crical in developing and integrang novel paent treatment algo- an algorithm for the development of combinaon therapies for mulple rithms sclerosis.

3:05 Close of Conference EXECUTIVE CHANNEL

Online: Tri-Conference.com Email: [email protected] Fax: 781-972-5425 39  MED001719 PLENARY KEYNOTE PRESENTATIONS

WEDNESDAY, FEBRUARY 3

8:108:10 - 8:55 am When Drug Research is Personal

John F. Crowley, Founder, Novazyme Pharmaceucals, Inc. This BioTech Primer short course is geared for the non-scienst. It highlights the basic science of recombinant DNA used in drug discovery and the basic science of how biologics are produced in drug development. Parcipants will walk away with a clear understanding of the difference between a biologic and the more tradi- Mr. Crowley’s emoon-packed presentaon will focus on his personal struggle to find onal small molecule drugs historically made by the pharmaceucal industry. a cure for Pompe disease, a rare and fatal illness that is caused by a defecve or missing enzyme. Pompe disease affects fewer than 10,000 people world-wide, including Mr. Basic Immersion Topics Include: Crowley’s two small children. Learning Objecves: Industry Overview Industry Sectors Mr. Crowley, a Harvard educated businessman, created and built a pharmaceucal Gain a fundamental Drug Discovery: company devoted expressly to finding a cure for the disease. He will detail his journey understanding of the The Science: Finding a Target through the labyrinth of scien c and business fronts, which lead up to a rst-round science and technology DNA Designing a Targeng fi fi driving the Biotech/ clinical trial. Genec Variaon Strategy Pharma industry Proteins Assay Development Genec Basis of Learn basic scienfic Pharmacogenomics 8:55 – 9:40 am Technology, Aging, and the Brain terminology used by Disease researchers in the life Drug Development: Gary W. Small, M.D., Parlow-Solomon Professor on Aging, sciences The Technology: Preclinical Trials Professor of Psychiatry & Biobehavioral Sciences, Director, Restricon Enzymes Clinical Trials Recombinant DNA Small and Large UCLA Center on Aging, Director, Memory & Aging Recombinant Proteins Molecule Generics Research Center, Director, Geriatric Psychiatry Division, Plasmids Biologics Deliverables: Semel Instute for Neuroscience & Human Behavior, A colored handout of all presented slides. David Geffen School of Medicine at UCLA New neuroimaging and other technologies are teaching us about how the brain ages A 64-page booklet:The Primer: A Biotechnology Guide for Non-Sciensts and what we can do about it. Although memory declines as we age, medical and non- pharmacological strategies may protect brain health and improve memory performance. At the same me, innovaon in digital technology is not only changing the way we live and communicate, it appears to be altering how our brains funcon. As a consequence of this high-tech smulaon, we are witnessing the beginning of a new form of the generaon gap – a brain gap dividing younger digital naves, immersed in the technol- Empower yourself ogy early in life, from older digital immigrants, who adapt to the new technology more and your company by learning the reluctantly. This lecture will describe this current pivotal point in brain evoluon and science and technology driving the how we can harness the new technology and lifestyle choices to improve memory and biotech and pharmaceucal industries brain funcon so we can live beer and longer. Who Will Benefit? Professionals from all sectors of the biotech, pharma and life sciences industries, including: sales, markeng, HR, legal, THURSDAY, FEBRUARY 4 manufacturing, business development, finance, management, government relaons, IT, safety, tech transfer Policy makers, lobbyists, aorneys 2:25 – 3:05 pm Chips, Clones and Living Beyond 100 Venture capitalists, angel investors, banks, analysts, Paul J.H. Schoemaker, Ph.D., M.B.A., Chairman and Chief financial managers Execuve Officer, Decision Strategies Internaonal, Inc.; Insurance brokers, real estate professionals Research Director, Mack Center for Technological Consultants, public relaons specialists, journalists Innovaon, The Wharton School; Adjunct Bioscience associaon staff, economic development execuves Professor of Markeng, The Wharton School Adjunct University administrators, research instute support staff Professor, WhartonSchool of Business Course Instructor Karin Lucas, Ph.D., Karin Lucas, Ph.D., BioTech Primer Instructor and Scienfic Advisor As informaon technologies and life sciences connue to converge, new business op- Biotech Primer Instructor Karin Lucas, Ph.D., has been teaching with BioTech Primer, Inc. for the past five years. As a scienst at Biogen Idec she develops protein pharmaceucals for the treatment of portunies and challenges will arise for the field of diagnoscs and beyond. This keynote and Scienfic Advisor cancer and mulple sclerosis. Previously, Dr. Lucas was a scienst and project director reviews the deeper forces shaping the future of the biosciences, from social and eco- at Cardinal Health where she worked on the development of over 25 products with mulple pharmaceucal and biotechnology companies. In addion to her laboratory nomic to technological and polical, including the stresses they will introduce for exisng role, she is also trained as a Lean Six Sigma greenbelt. Dr. Lucas is an acve community volunteer and has served as the PR chair and later Vice President of the San Diego business models and healthcare. Not only will bio-convergence introduce new products, chapter of AWIS (Associaon forWomen in Science). In 1998, Dr. Lucas was honored as services and competors, it may create enrely new industries on a scale larger than the the Cal Poly Physical Chemistry Student of the Year and in 2003 she was selected for the AWIS San Diego Rookie of the Year Award. Dr. Lucas received her B.S. in biochemistry computer revoluon has to date. Several broad scenarios will be painted for the state of from California Polytechnic State University, San Luis Obispo and went on to complete the biosciences in 2025 and the forces that might take us there, summarizing a mul- her Ph.D. in biochemistry at University of California San Diego. year strategy study conducted and supervised by the speaker at the Wharton school.

40 Online: Tri-Conference.com Email: [email protected] Fax: 781-972-5425  MED001720 EXHIBIT AND SPONSORSHIP INFORMATION NEW SPONSORSHIP OPPORTUNITIES Other Promotional Opportunities 2010! Sponsorships allow you to achieve your • Conference Tote Bags FOR objectives before, during and long after • Tote Bag Inserts the event. Any sponsorship can be cus- • Badge Lanyards New Product tomized to meet your company’s needs • Refreshment Breaks Showcase Pavilion and budget. Signing on earlier will allow • Program Guide Event you to maximize exposure to hard-to- • Program Guide Sponsorship For the first me ever, the Molecular reach decision-makers. CHI events • Poster Abstract Book Medicine Tri-Conference will feature a New bring you the highly qualified, targeted • Poster Award Sponsorship Product Showcase Pavilion. The New Product audience you are trying to reach. • Corporate Branding Packages Showcase Pavilion is the place for exhibitors to introduce and promote their new product OPPORTUNITIES INCLUDE: to conference aendees. CHI will promote the New Product Showcase Pavilion in our Podium Presentations pre-show promoons, on our website, as well Present your solution for 15 or 30 min- as on-site. utes in the session room during lunch or as part of the main conference program. For more informaon, and to discuss your sponsorship needs, please contact: Invitation-Only Networking Functions Carol Dinerstein Target specific delegates for an invi- Director, Exhibit & Sponsorship Sales tation-only dinner or hospitality suite. Exhibiting 781-972-5471 You will select attendees directly from Exhibiting allows your company to [email protected] the event pre-registration list. differentiate your products, services or technology from competitors, and Jon Stroup demonstrate your commitment to this Manager, Business Development science. It is also a perfect platform to 781-972-5483 launch a new product, collect feedback, [email protected] network, and generate new leads. Reserve your exhibit space by September 15th, SAVE $300, and select a prime location!

Exclusive CocktailCo Receptions (Program-specific) CHI will invite all delegates from a specific conference program to your private reception at the host hotel. Cocktails and hors d’oeuvres will be served in a setting conducive to networking. These receptions are available on a first-come, first serve basis. Sponsorship packages include multiple branding benefits for your company, additional conference passes, plus a premium exhibit location in the exhibit hall SPONSORS & EXHIBITORS 2010 as of 11/06/09

Accelrys Docro KINAXO Biotechnologies GmbH SCHOTT North America Inc Advanced Chemistry Development, Inc. (ACD Labs) DV Biologics Linguamatics Scottish Development Intl. Adis(a Wolters Kluwer Business) DxS Marken Sequenom Almac Diagnostics Elsevier Medical Device Consultants, Inc. Sigma Aldrich Ambry Genetics Enamine Ltd. Mercodia Simulations Plus, Inc. Analytical Biological Services Epicentre Biotechnologies Metanomics Health Specs Analyticon Discovery Eppendorf Array Technologies Microsoft System Biosciences (SBI) Applied Biosystems Esoterix Clinical Trials Services Miltenyi Biotec Taconic Aragen Bioscience Inc Evotec NanoInk Teledyne Archimedes Inc Exiqon Nanostring Thermo Scientiﬁc Asuragen, Inc. Fate Therapeutics Nanosyn Thomson Reuters Biobase Febit Novartis Molecular Diagnostics Transgenomic, Inc. BioFocus DPI Limited Fluidigm Onyx Scientiﬁc Trans-Hit Biomarkers Inc. Bright Star Research Future Science Group Pall Life Sciences TriLink BioTechnologies CDD (Collaborative Drug Discovery) GeneGo Pharmacore VisualSonics ChemBridge Genometrica Ltd. Phylogeny WiCell Research Institute Chemical Computing Group High Throughput Genomics (HTG) PreClinOmics Wolters Kluwer Health Clinical Reference Laboratory Ingenuity Systems Prestwick Chemical Inc. World Courier, Inc. Collaborative Drug Discovery Integrated DNA Technologies ProteoGenex Zeptosens Compendia Bioscience InterMed Discovery GmbH Quosa Cureline, Inc. Kalexsyn Rules Based Medicine

Online: Tri-Conference.com Email: [email protected] Fax: 781-972-5425 41  MED001721 SHAPING FUTURE MEDICINE Conference: February 3-5 | Exhibits: February 3-4 Moscone North Convention Center | San Francisco, CA

HOW TO REGISTER: Online: Tri-Conference.com Email: [email protected] Phone: 781-972-5400  Fax: 781-972-5425

REGISTRATION INFORMATION 1037 F Mr. Ms. Mrs. Dr. Prof. Name Job Title Div./Dept. Company Address City/State/Postal Code Country Telephone How would you prefer to receive notices from CHI? Email: Yes No Fax: Yes ฀No Email* Fax *Email is not a mandatory field. However, by excluding your email you will not receive notification about online access to pre-conference presenter materials, conference updates, networking opportunities and requested eNewsletters. Academic, Government, PRE-CONFERENCE EVENT PRICING (FEBRUARY 2) Commercial Hospital-affiliated ฀Choose 1 Short Course $695 $345 ฀Choose 2 Short Courses $995 $595

MORNING SHORT COURSES: AFTERNOON SHORT COURSES:

฀SC1 Applying Next Generation Sequencing Technologies To Research ฀SC7 Best Practices In Translational & Personalized Medicine ฀SC2 One Case Study In Breast Cancer-Three Perspectives ฀SC8 Strategies for Molecular Diagnostic Companies

฀SC3 Mighty Mitochondria: Their Relevance to Disease and Translational Medicine ฀SC9 Fragment - Inspired Medicinal Chemistry Please refer to the Registration Code below: ฀SC4 Addressing Safety Concerns For Biological Drugs ฀SC10 Transporter-Mediated Drug-Drug Interaction Potential ฀SC5 Targeting Cancer Stem Cells With Biologics ฀SC11 Basic Immersion: Cutting Edge Science & Technology for Biotech & Pharma ฀SC6 Blood-Brain Barrier ฀SC12 Designing Rigorous Omics Studies

PROGRAM PRICING (FEBRUARY 3-5) Access to 200+ Presentations Covering 11 programs, CHI’s Intro-Net, Scientiﬁc Posters and more!

Advance Registration until January 15, 2010 ฀$1795 ฀$1045 Registration after January 15, 2010 ฀$1995 ฀$1095 Yes! I would like to receive a FREE PROGRAM SELECTION: (REQUIRED) Please indicate the ONE program you are most likely to attend. eNewsletter subscription to: www.chimediagroup.com DIAGNOSTICS CHANNEL CHEMISTRY CHANNEL ฀Molecular Diagnostics ฀Cancer Molecular Markers ฀Mastering Medicinal Chemistry ฀Personalized Diagnostics The latest industry news, commentaryand highlights from BioIT World INFORMATICS CHANNEL BIOLOGICS CHANNEL Innovative management in clinical trials ฀Adopting R&D Informatics Systems ฀Stem Cells ฀Cancer Biologics Pharma Services News ฀ ฀Cancer Profiling and Pathways ฀RNA Interference ฀Delivery of Biologics Service solutions for discovery, pre-clinical and clinical trials CANCER CHANNEL EXECUTIVE CHANNEL Additional Registration Details Each registration includes all conference sessions, posters and exhib- ฀Cancer Biologics ฀Cancer Profiling and Pathways ฀Translational Medicine its, food functions, and a copy of the conference proceedings link. ฀Cancer Molecular Markers ฀REGISTER 3 - 4th IS FREE DISCOUNTS* Individuals must register for the same conference or conference ฀Poster ($50 off) ฀Alumni (20% off) (10% off) ฀Hotel ($75 off) see pg 18 for details combination and submit completed registration form together for Hotel Confirmation number is ______discount to apply. Please reproduce this registration form as needed. Group Discounts *Alumni and Bay Bio Discount cannot be combined. Discounts not applicable on Pre-Conference Events, (Conference registrations only) Special rates are available for multiple attendees from the same I cannot attend but would like to purchase the Molecular Medicine Tri-Conference CD for $750 (plus shipping). organization. Contact David Cunningham at 781-972-5472 to Massachusetts delivery will include sales tax discuss your options and take advantage of the savings.

PAYMENT INFORMATION Handicapped Equal Access In accordance with the ADA, Cambridge Healthtech Institute is Enclosed is a check or money order payable to Cambridge Healthtech Institute, drawn on a U.S. bank, in U.S. currency. pleased to arrange special accommodations for attendees with Invoice me, but reserve my space with credit card information listed below. special needs. All requests for such assistance must be submitted in writing to CHI at least 30 days prior to the start of the meeting. Invoices unpaid two weeks prior to conference will be billed to credit card at full registration rate. Invoices must be paid in full and checks received by the deadline date to retain registration discount. If you plan to register on site, please check with CHI beforehand for space availability. Substitution/Cancellation Policy In the event that you need to cancel a registration, you may: Please charge: AMEX (15 digits) Visa (13-16 digits) MasterCard (16 digits) t Transfer your registration to a colleague within your organization. Card # Expiration Date t Credit your registration to another Cambridge Healthtech Cardholder Institute program. t Request a refund minus a $100 processing fee per conference. Signature t Request a refund minus the cost ($750) of ordering a copy of the CD. Cardholder’s Address (if different from above) NOTE: Cancellations will only be accepted up to two weeks prior to City/State/Postal Code the conference. Program and speakers are subject to change. Country CHI Insight Pharma Reports A series of diverse reports designed to keep life science profes- Present a Poster and Save $50! January 8, 2010 sionals informed of the salient trends in pharmaceutical technology, Poster abstracts submitted and approved between December 24, 2009 and January business, clinical development, and therapeutic disease markets. Cambridge Healthtech Institute encourages attendees to gain further exposure by 8, 2010 will not be included in the Program Guide, but instead will be included in the For a detailed list of reports, visit InsightPharmaReports.com, or presenting their work in the poster sessions. Program Addendum and Conference Proceedings Link. contact Rose LaRaia, SMBSBJB!IFBMUIUFDIDPm, 781-972-5444. Special poster deadlines apply. To secure a poster board and inclusion in speciﬁc All poster abstracts are due no later than January 8, 2010. Register online, or Barnett Educational Services conference materials, your abstract must be submitted, approved and your by phone, fax or mail. Indicate that you would like to present a poster and you will Barnett is a recognized leader in clinical education, training, and registration paid in full by the following deadlines: receive abstract submission instructions via email. reference guides for life science professionals involved in the drug December 23, 2009 ฀Yes, I am interested in presenting a poster at: development process. For more information, Poster abstracts submitted and approved by December 23, 2009 will be included in Molecular Medicine Tri-Conference visit www.barnettinternational.com. the Program Guide and Conference Proceedings Link. Video and or audio recording of any kind is prohibited onsite at all CHI events. MED001722 2016 Annual Meeting American College of Clinical Pharmacology Clinical Pharmacology: Discovery and Application in the Era of Precision Medicine

September 25 – 27, 2016 Bethesda N Marriott Hotel & Conference Ctr, Bethesda, MD

Co-chairs: Vikram Arya, PhD, Honghui Zhou, PhD and Manish Gupta, PhD

FINAL PROGRAM

MED001723 Join Us for the 2017 ACCP Annual Meeting!

ACCP is a proud provider of Continuing Medical Education (CME) & Continuing Pharmacy Education (CPE)

FUTURE MEETINGS: 2018 Annual Meeting September 23 – 25, 2018 Bethesda N Marriott Hotel & Conference Ctr Bethesda, MD 2

MED001724 Did You Know?

Use the ACCP Mobile App to find all the • Open access options available for authors who wish to make their article free for all to access online meeting information you’re looking for! • Immediate international exposure with PubMed/MEDLINE®, Web of • Session schedules organized by each day and track, with search Science: Science Citation Index Expanded (SCIE), Scopus, Chemical functionality Abstracts, Embase and Google Scholar indexation • Session details including times, locations, session description, Faculty profiles and presentations Attending the Meeting as a Student or Trainee? • Presentations from all sessions available real time and archival viewing ACCP has planned a series of events specifically to benefit Students & (.pdf format) during and after the event Trainees! See page 43 for details. • Access attendee and Faculty lists, with search functionality • Establish contact and network with fellow attendees Interested in joining ACCP? • Chat/instant message with fellow attendees Stop by the ACCP Registration Desk for complete information or to • View profiles of Faculty, ACCP Staff, Exhibitors & Sponsors with contact complete a profile and pay 2017 Dues entitling you to ACCP Member information Benefits. • Customize profile and business card on mobile device and/or import LinkedIn profile Take Time to Visit Our Exhibitors! Exhibitor support is critical to the success of the ACCP Annual Meeting. ACCP continues to expand its Continuing We encourage you to visit our Exhibitors in Ballroom Salon E-H Education Program! during breakfast, breaks or the evening receptions to learn about new An accredited provider of Continuing Medical Education (CME) and technologies and service offerings. These exceptional Exhibitors are Continuing Pharmacy Education (CPE), ACCP continues to expand its the leaders in their fields and are anxious to share with you the latest Continuing Education Program. In 2016, ACCP provided Journal CE information on how they can help you meet your goals! Please take articles each month and continued the ACCP Virtual Journal Club and a moment to thank them for their support. All attendees are invited to Fundamentals Tutorials webinars, in participate in the Exhibit Hall Contest to win one of two $50 gift cards by addition to launching the Therapeutic getting your game card stamped by all the Exhibitors. Game cards are Dilemmas webinars. Each of the provided in attendee tote bags. live webinars is then made available On Demand. See page 13 for more “Like” ACCP and add to “My Page’s Favorites” on information. Facebook or join ACCP’s Group for Publish Your Manuscript in The Journal of regular updates. Clinical Pharmacology ACCP Registration Desk Hours / Grand Foyer • The average time from submission to first decision is 14 days • For manuscripts that are revised, the average time from the first Friday, September 23rd 4:00 – 7:00 pm Grand Foyer submission to final decision is 25 days Saturday, September 24th 7:00 am – 5:30 pm Grand Foyer • Downloads from JCP have increased by approximately 150% since 2013 Sunday, September 25th 6:30 am – 7:00 pm Grand Foyer • With the JCP App you can read the Journal anytime on your smart Monday, September 26th 7:00 am – 7:00 pm Grand Foyer phone or tablet. Download this from the Apple App store. Tuesday, September 27th 7:00 am – 5:30 pm Grand Foyer Clinical Pharmacology in Drug Development is now indexed by MEDLINE® and SCIE Lost & Found Why publish with CPDD? Any found items should be given to ACCP Staff at the Registration Desk • Well respected: Official Journal of the ACCP in the Grand Foyer. Persons wishing to retrieve a lost item should also • A renowned international Editorial Board contact ACCP Staff at the ACCP Registration Desk. • Growing international readership: full text article downloads increased by >38% in 2015! • Quick and easy online submission and review process • Rapid publication: articles are available online weeks ahead of issue publication

3 MED001725 Table of Contents

Invitation to 2017 ACCP Annual Meeting ...... 2

American College of Did You Know? ...... 3

Clinical Pharmacology Letter of Welcome from President 2016 Program Committee & Program Co-chairs ...... 5

Program at a Glance ...... 6 Co-chairs: Vikram Arya, PhD Keynote Speaker ...... 8 Manish Gupta, PhD 2016 ACCP Recognition Award Winners ...... 9 Honghui Zhou, PhD Educational Accreditation ...... 12 Members: Nageshwar R. Budha, PhD Educational Activities ...... 13 Lawrence J. Cohen, PharmD Faculty Disclosure Information ...... 14 Amelia N. Deitchman, PharmD Nilima A. Kshirsagar, MBBS, MD, PhD Pre-meeting Workshops ...... 16 Nitin Mehrotra, PhD Symposia ...... 20 Robert J. Noveck, MD, PhD, CPI Stephan Schmidt, PhD Faculty ...... 37 Jayabharathi Vaidyanathan, PhD Why Join ACCP? ...... 42 John van den Anker, MD, PhD Students & Trainees ...... 43

Sponsors ...... 44

Exhibitors ...... 45

Poster Sessions ...... 50

ACCP Oficers, Regents, Vision & Mission ...... 58

New Members: August 1, 2015 – July 31, 2016 ...... 59

MED001726 Letter of Welcome from President & Program Co-chairs

Welcome to the 2016 ACCP Annual Meeting! Clinical Pharmacology: Discovery and Application in the Era of Precision Medicine

Dear Colleague:

It is our pleasure to welcome you to the 2016 Annual Meeting of the Poster Sessions held on Sunday and Monday evening will focus on new American College of Clinical Pharmacology (#2016ACCP), Clinical indings and preliminary data presented by a wide spectrum of attendees. Pharmacology: Discovery and Application in the Era of Precision Socialize and network at the catered receptions during the Poster Sessions, Medicine. The 2016 Annual Meeting Program Committee, co-chaired at twice-daily tea/coffee breaks and at the Lunch & Awards Sessions on by Drs. Vikram Arya, Honghui Zhou and Manish Gupta, has developed Monday and Tuesday. At the lunch session on Tuesday, Kathy Hudson, a diverse and exceptional educational program to meet the needs of PhD, Deputy Director for Science, Outreach and Policy at the National Inst a broad spectrum of healthcare professionals and scientists with an of Health, will present the Keynote address. interest in clinical pharmacology applications from research and drug development to patient care. ACCP is pleased to host a global panel of Experience for yourself how ACCP makes a difference by providing speakers from academia, industry, regulatory and clinical entities that healthcare professionals and scientists with a forum to exchange knowledge will present programs organized into topic tracks, allowing each attendee and ideas that promote and expand the value of clinical pharmacology in to uniquely tailor content selection based on individual interests. Major healthcare and drug development. clusters of topic areas include oncology drug development, including immuno-oncology, biologics and biosimilars, pediatric drug development Please note that there is complimentary Internet access provided to all and precision medicine. The exciting mix of sessions includes the use of Annual Meeting attendees in guest rooms for the duration of the meeting. All novel and innovative clinical pharmacology tools and principles to improve of the educational sessions, social events and networking will be held at the drug development and therapeutics for effective treatment of disease states hotel, facilitating the ease with which meeting attendees can participate in such as HIV/AIDS, Hepatitis C and cancer; orphan drug development; the events. With the hotel’s convenient location to the White Flint Metro Station, application of the animal rule; the management of opioid dependence; the there is easy access to downtown Bethesda, Rockville, Washington, use of Big Data, physiologically-based PK/PD modeling and novel trial DC and northern Virginia attractions, making it easy to enjoy all that the designs for pediatric drug development programs; improvements in clinical Washington metro area has to offer. pharmacology labeling and streamlining of clinical pharmacology activities in early development. ACCP remains an accredited provider of Continuing Medical Education (CME) and Continuing Pharmacy Education (CPE) credits for our New this year is a mixture of several shorter Symposia combined with our educational events, provided to meeting attendees at no additional cost. traditional four-hour format. For the irst time, a select group of cutting-edge poster presentations will be hosted in an intimate setting that encourages We welcome you to an outstanding 2016 ACCP Annual Meeting and look discussion in a relaxed atmosphere. Of special note are Student & Trainee- forward to feedback about your participation! focused programs that provide exposure to cutting-edge science and career development.

Bernd Meibohm, PhD Vikram Arya, PhD Honghui Zhou, PhD Manish Gupta, PhD ACCP President Program Co-chair Program Co-chair Program Co-chair 5 MED001727 Program at a Glance

The 2016 ACCP Annual Meeting is supported in part by an Educational Grant from Pizer Inc

Welcome and Opening Remarks by President FRIDAY, SEPTEMBER 23, 2016 7:45 – 8:00 am l Ballroom Salon A-C Symposium 1 l 8:00 am – 12:00 pm ACCP Registration Desk Open l 4:00 – 7:00 pm Grand Foyer Clinical Pharmacology as a Cornerstone for Development of Products Under the Animal Rule: Determining an Effective Dose ACCP Executive Committee Meeting l 6:00 – 9:00 pm in Humans Middlebrook CO-CHAIRS: Nitin Mehrotra, PhD and Kimberly L. Bergman, PharmD Ballroom Salon A-C SATURDAY, SEPTEMBER 24, 2016 Symposium 2 l 8:00 – 9:30 am A 360⁰ View of Immunogenicity: Qualitative & Quantitative ACCP Registration Desk Open l 7:00 am – 5:30 pm Assessments to Understand Its Implications on Grand Foyer Pharmacokinetics, Safety & Eficacy CO-CHAIRS: Chaitali Passey, PhD and Sumit Rawal, PhD Continental Breakfast l 7:30 – 8:30 am Ballroom Salon D Glen Echo Foyer Symposium 3 l 10:00 am – 12:00 pm ACCP Board of Regents Meeting l 8:00 am – 1:00 pm Glen Echo Helping Advance the Immuno-Oncology Revolution: Trends in Translational Immuno-Oncology Pre-meeting Workshop 1 l 8:00 am – 12:00 pm CO-CHAIRS: Sree Kasichayanula, PhD and Yu-Nien (Tom) Sun, PhD Translational Pharmacokinetics/Pharmacodynamics in Ballroom Salon D Biotherapeutic Minimum Anticipated Biological Effect Level Honors & Awards Committee Meeting l 12:00 – 1:30 pm Dose Selection & Novel Protein Scaffolds Forest Glen CO-CHAIRS: Honghui Zhou, PhD and Rong Shi, PhD Ballroom Salon H 2016 – 2017 Program Committee Meeting 12:00 – 1:30 pm l Glen Echo Pre-meeting Workshop 2 l 8:00 am – 12:00 pm Combating HIV/AIDS: Treatment, Pharmacogenetics & Pre- Membership Committee Meeting l 12:00 – 1:30 pm exposure Prophylaxis Middlebrook CO-CHAIRS: Sam Harirforoosh, PharmD, PhD and Ganesh Cherala, PhD Public Policy Committee Meeting l 12:00 – 1:30 pm Ballroom Salon G Timberlawn Pre-meeting Workshop 3 l 1:30 – 5:30 pm Symposium 4 l 1:30 – 5:30 pm Improving Therapeutics to Better Care for Older Adults & Clinical Development of Biologics: Current Strategy, Challenges the Young & Future Considerations CO-CHAIRS: S.W. Johnny Lau, PhD and Thomas Eissing, PhD CO-CHAIRS: Gaurav Bajaj, PhD and Ping Zhao, PhD Ballroom Salon H Ballroom Salon A-C Pre-meeting Workshop 4 l 1:30 – 5:00 pm Symposium 5 l 1:30 – 3:00 pm The Other Bound of the Therapeutic Window: Exposure-Safety Addressing Opioid Dependence: Now is the Time Analysis to Inform Dosing Decisions in Oncology CO-CHAIRS: Lorraine M. Rusch, PhD and Michael J. Fossler, Jr, PharmD, CO-CHAIRS: Justin C. Earp, PhD and Anshu Marathe, PhD PhD Ballroom Salon G Ballroom Salon D ACCP Finance Committee Meeting l 3:00 – 5:00 pm Student Panel Discussion & Career Guidance Middlebrook 2:00 – 3:30 pm l White Flint Amphitheater Regents & Awards Reception (invitation only) Student Podium Presentations l 3:30 – 4:30 pm 5:30 – 6:30 pm l Brookside Foyer White Flint Amphitheater Regents & Awards Dinner (invitation only) Symposium 6 l 3:30 – 5:30 pm 6:30 – 8:30 pm l Brookside A&B Treatment of Hepatitis C with Direct-acting Antiviral Drugs: Opportunities & Challenges SUNDAY, SEPTEMBER 25, 2016 CO-CHAIRS: Vikram Arya, PhD and Shirley Seo, PhD Ballroom Salon D ACCP Registration Desk Open l 6:30 am – 7:00 pm Student Networking Reception l 4:30 – 5:30 pm Grand Foyer Brookside Foyer Continental Breakfast l 7:00 – 8:00 am Opening Reception & Poster Session 1 & Exhibits Grand Foyer 5:30 – 7:30 pm l Ballroom Salon E-H Student Poster Tour l 5:45 – 6:30 pm Meet at ACCP Registration Desk at 5:30 pm 6

MED001728 Program at a Glance

Editorial Board Dinner (invitation only) l 7:30 – 9:30 pm MONDAY, SEPTEMBER 26, 2016 Brookside A&B

ACCP Registration Desk Open l 7:00 am – 7:00 pm Grand Foyer TUESDAY, SEPTEMBER 27, 2016 Continental Breakfast l 7:00 – 8:00 am ACCP Registration Desk Open l 7:00 am – 5:30 pm Ballroom Salon E-H Grand Foyer Exhibit Hall Open l 7:00 – 10:00 am Continental Breakfast l 7:00 – 8:00 am Ballroom Salon E-H Ballroom Salon E-H Annual Business Meeting l 7:15 – 8:00 am Student Event: Special Access to the Experts Ballroom Salon A-C 7:00 – 8:00 am l Ballroom Salon E-H Symposium 7 l 8:00 am – 12:00 pm Exhibit Hall Open l 7:00 – 10:00 am Establishing Biosimilarity: The European Perception, Experience Ballroom Salon E-H & Future Trends Education Committee Meeting 7:00 – 8:00 am CO-CHAIRS: Hildegard Sourgens, MD, PhD and Hartmut Derendorf, PhD l Great Falls Ballroom Salon A-C Publications Committee Meeting 7:00 – 8:00 am Symposium 8 8:00 – 9:30 am l l Middlebrook Informing Pediatric Development Programs: Leveraging Big Data CO-CHAIRS: Jeffrey Barrett, PhD and Lily (Yeruk) Mulugeta, PharmD Symposium 13 l 8:00 am – 12:00 pm Ballroom Salon D Orphan Drug Development in Adults & Pediatrics: Industry, Academia & Regulatory Perspectives Symposium 9 10:00 am – 12:00 pm l CO-CHAIRS: Vijay Ivaturi, PhD and Venkatesh Atul Bhattaram, PhD Little Children, Big Challenges: The Problems for Neonatal Drug Ballroom Salon A-C Trials & the Way Forward CO-CHAIRS: Jian Wang, PhD and John van den Anker, MD, PhD Symposium 14 l 8:00 – 9:30 am Ballroom Salon D Clinical Applications of Physiologically-based Pharmacokinetics/ Pharmacodynamics for Pediatrics: Academic, Industry & Lunch Buffet l 11:45 am – 1:45 pm l Grand Foyer A-D Regulatory Perspectives CO-CHAIRS: Jennifer Sheng, PhD, PharmD and Diansong Zhou, PhD Lunch & Awards Session l 12:10 – 1:20 pm Ballroom Salon D Ballroom Salon A-D Symposium 15 l 10:00 – 11:45 am • Distinguished Investigator Award Combination Therapy in Oncology: Challenges & Strategies in • Honorary Fellowship Award Clinical Pharmacology • Nathaniel T. Kwit Memorial Distinguished Service Award CO-CHAIRS: Yilong Zhang, PhD and Satyendra Suryawanshi, PhD • McKeen Cattell Memorial Award Ballroom Salon D • Abstract Awards • Member-Get-a-Member Awards Lunch Buffet l 11:45 am – 1:45 pm l Grand Foyer A-D

Symposium 10 1:30 – 5:30 pm Lunch & Awards Session l 12:10 – 1:20 pm l Ballroom Salon A-D Streamlining Clinical Pharmacology Strategies During Early Development: Assessment of Drug-Drug Interactions, Food • Tanabe Young Investigator Award Effect & QTc • Bristol-Myers Squibb Mentorship in Clinical Pharmacology Award CO-CHAIRS: Suraj G. Bhansali, MS, PhD and Xiao Hu, PhD • Keynote Presentation: “The Precision Medicine Initiative” Kathy L. Ballroom Salon A-C Hudson, PhD, Deputy Director for Science, Outreach and Policy at the National Inst of Health Symposium 11 1:30 – 3:00 pm l Cutting-edge Abstract Presentations CO-CHAIRS: Lawrence J. Cohen, PharmD, Walter K. Kraft, MD and Amalia Symposium 16 l 1:30 – 5:30 pm M. Issa, PhD Clinical Pharmacology Strategies in Precision Medicine-based Ballroom Salon D Drug Development & Preventive Medicine CO-CHAIRS: Priyanka Jadhav, PhD, Jinshan Shen, PhD and Manoj P. Exhibit Hall Open 3:00 – 7:30 pm Ballroom Salon E-H l l Jadhav, PhD Symposium 12 l 3:30 – 5:30 pm Ballroom Salon A-C Rethinking Clinical Pharmacology-related Labeling for Improved Symposium 17 l 1:30 – 5:30 pm Utility & Comprehension Reproducible Visualization & Data Analysis With R CO-CHAIRS: Joseph A. Grillo, PharmD and Julie Bullock, PharmD Ballroom Salon D CHAIR: Devin Pastoor, MTOX Ballroom Salon D Evening Reception & Poster Session 2 & Exhibits 5:30 – 7:30 pm l Ballroom Salon E-H 7 MED001729 Keynote Speaker

Tuesday, September 27, 2016 | 12:50 – 1:20 pm | Ballroom Salon A – D

Kathy L. Hudson, PhD Deputy Director for Science, Outreach and Policy at the National Inst of Health “The Precision Medicine Initiative” Kathy L. Hudson, PhD, is the Deputy Director for Science, Outreach and Policy at the National Inst of Health (NIH). Dr. Hudson leads the science policy, legislation, communication and outreach efforts of the NIH and serves as a senior advisor to the NIH Director. She is responsible for creating major new strategic and scientiic initiatives for NIH and is currently leading the planning and creation of the President’s Precision Medicine Initiative Cohort Program. Dr. Hudson was a key architect of the National Ctr for Advancing Translational Sciences and the NIH BRAIN Initiative. She directs the agency’s efforts to advance biomedical research through policy development, public and stakeholder communication & education and innovative projects & partnerships. Dr. Hudson’s professional experience includes serving as the NIH Chief of Staff; Acting Deputy Director of the National Ctr for Advancing Translational Sciences, NIH; the Assistant Director of the National Human Genome Research Inst, NIH; and the founder and Director of the Genetics and Public Policy Ctr at Johns Hopkins Univ. Also at Hopkins, Dr. Hudson was an Associate Professor in the Berman Inst of Bioethics, Inst of Genetic Medicine and Dept of Pediatrics. Dr. Hudson holds a PhD in Molecular Biology from the Univ of California at Berkeley, an MS in Microbiology from the Univ of Chicago and a BA in Biology from Carleton Coll.

Abstract Awards Program

Student & Trainee Abstract Award Winners 2016 Student & Trainee Abstract Award Winners Student & Trainee Abstract Awards are given for the best Student Abstract Award Winners will present their posters at both Poster Sessions abstracts submitted by Students & Trainees for presentation at • Sumit Basu, PhD (Poster #089) Univ of Florida, Orlando, FL each year’s Annual Meeting. • Kristina M. Brooks, PharmD (Poster #041) National Inst of Health, Wayne A. Colburn Memorial Award Bethesda, MD The Wayne A. Colburn Memorial Award honors the memory of • Amelia N. Deitchman, PharmD (Poster #013) Univ of Florida, the late Wayne A. Colburn, former ACCP President, and will be Orlando, FL given for the best paper among the Student & Trainee Award Winners, as judged by the Program Committee during the Poster • Asma El-Zailik, BS (Poster #014) Univ of Houston, Houston, TX Sessions at the Annual Meeting. The winner will be announced • Edwin Lam, PharmD (Poster #061) Long Island Univ, Brooklyn, NY during the Monday luncheon and the author will give a short talk • Naveen Mangal, BS, MS (Poster #088) Univ of Florida, Orlando, outlining the indings of the study in Symposium 11. FL New Member Abstract Award • Mahua Sarkar, MS (Poster #001) Univ of Houston, Houston, TX The New Member Abstract Award is given for the best abstract • Tanaya Vaidya, MS (Poster #040) Univ of Florida, Orlando, FL submitted by a New Member of ACCP for presentation at the Annual Meeting. Abstracts submitted by New Members will • Jessica Wojciechowski (Poster #076) Univ of South Australia, be judged during the Poster Sessions. The winner will be Adalaide, AU announced during the Monday luncheon and the author will give a short talk outlining the indings of the study in Symposium 11.

MED001730 2016 ACCP Recognition Award Winners

Distinguished Investigator Award Monday, September 26, 2016 | 12:15 – 12:35 pm | Ballroom Salon A – D “From Methotrexate to Targeted Therapies for Cancer: Individualizing the Approach” Bruce A. Chabner, MD – Professor of Medicine, Harvard Medical School; Emeritus Director of Clinical Research, Massachusetts General Hosp Cancer Ctr; Co-leader, Translational Pharmacology & Early Therapeutic Trials Program at the Dana Farber/ Harvard Cancer Ctr The Distinguished Investigator Award is given annually and is intended to recognize superior scientiic expertise and accomplishments by a senior investigator, usually involving a distinct area of research in basic or clinical pharmacology, for which the individual is internationally known. The candidate need not be a Member or Fellow of ACCP. Dr. Chabner has performed seminal and extensive work in the ield of cancer drug discovery and development. His lifelong contributions to the ield of clinical pharmacology and oncology make him a worthy recipient of the 2016 ACCP Distinguished Investigator Award.

Honorary Fellowship Award Monday, September 26, 2016 | 12:35 – 12:55 pm | Ballroom Salon A – D “Optimal Design in Pharmacometrics and Dose of Favipiravir for Ebola” France Mentré, PhD, MD – Director of Research, Vice Director, Graduate School of Public Health, Univ of Paris & Head, Biostatistics Dept, Bichat Hosp The Honorary Fellowship Award is given annually to a Non-member of ACCP and is meant to recognize primary activities within the immediate domain of clinical pharmacology. The award recognizes overall contributions to the ield, rather than any particular scientiic work, by a senior investigator or authority having a national or international reputation in the scientiic, public service, legislative, governmental or other area of endeavor impacting the ield. Dr. Mentré holds leadership positions in several scientiic organizations which support clinical pharmacology research and is the current Chair of the Executive Committee of the World Conference on Pharmacometrics and an Associate Editor of the Journal of Pharmacometrics and Systems Pharmacology. She has made substantial contributions to the ield of clinical pharmacology through research and training of basic and clinical pharmacologists, as well as through the development of tools to facilitate research in clinical pharmacology, making her a itting recipient of the 2016 ACCP Honorary Fellowship Award.

2016 Vera Donnenberg, PhD • Claude Abdallah, MD, MSc Pharm • April Barbour, PhD Honors & Steven J. Crosby, MA, BSP, RPh • Navin Goyal, PhD • Howard Greenberg, MD, MSE, MBA Awards Manoj P. Jadhav, PhD • Jatinder Mukker, PhD • Eric Olson, PhD Committee Laurent Vernillet, PharmD, PhD • Peter Wiernik, MD

9 MED001731 2016 ACCP Recognition Award Winners

Nathaniel T. Kwit Memorial Distinguished Service Award Monday, September 26, 2016 | 12:55 – 1:15 pm | Ballroom Salon A – D Margaret Hamburg, MD – Foreign Secretary for the National Academy of Medicine; previously Commissioner, US Food & Drug Administration The Nathaniel T. Kwit Memorial Distinguished Service Award is given in memory of the late Nathaniel T. Kwit, MD, FCP, a founding Fellow of ACCP, who served as a Regent for 5 years and as Treasurer for 20 years. The primary intent of this award is to recognize accomplishments of a general nature which beneit the ield of clinical pharmacology. These may be in the area of teaching, administration, service with ACCP or long-term and wide-ranging scientiic studies having practical importance and other service-related functions. It is differentiated from the Distinguished Investigator Award in that it is not intended to recognize any distinct area of scientiic investigation, but rather an overall contribution to the ield. The candidate need not be an ACCP Member or Fellow. While Commissioner of the US Food & Drug Administration, Dr. Hamburg supported regulatory initiatives for personalized drug therapy. Her extensive contributions, directly and indirectly relevant to clinical pharmacology, make her an outstanding recipient of the 2016 Nathaniel T. Kwit Memorial Distinguished Service Award.

McKeen Cattell Memorial Award Monday, September 26, 2016 | 1:15 – 1:20 pm | Ballroom Salon A – D Daniel A. Spyker, PhD, MD – Consulting Senior Director, Drug Safety & Pharmacovigilance, Alexza Pharmaceuticals Inc; Adjunct Professor, Dept of Internal Medicine, Uniformed Services Univ of Health Sciences; Adjunct Assistant Professor of Emergency Medicine, Oregon Health & Science Univ The McKeen Cattell Memorial Award is made in memory of the late McKeen Cattell, MD, PhD, FCP, the irst editor of The Journal of Clinical Pharmacology (JCP) and co-founder of ACCP. This award is made annually, recognizing an outstanding research paper published in the JCP during the preceding year. The award is typically presented to the irst author of the paper. This year’s award-winning journal article is: “Multiple-dose Pharmacokinetics of Inhaled Loxapine in Subjects on Chronic, Stable Antipsychotic Regimens” Authors: Daniel A. Spyker, PhD, MD, Robert A. Riesenberg, MD and James V. Cassella, PhD. Published in The Journal of Clinical Pharmacology Volume 55, Issue 9, pages 985–994, September 2015.

MED001732 2016 ACCP Recognition Award Winners

Tanabe Young Investigator Award Tuesday, September 27, 2016 | 12:15 – 12:30 pm | Ballroom Salon A – D “How Quantitative & Systems Pharmacology Can Bring Value to R&D and, Ultimately, to the Patient” Stephan Schmidt, PhD – Assistant Professor, Univ of Florida, Ctr for Pharmacometrics & Systems Pharmacology; Chair of the Int’l Pharmaceutical Federation’s Special Interest Group on Pharmacometrics & Systems Pharmacology; Incoming Chair of the ASCPT’s Special Interest Group on Systems Pharmacology The Tanabe Young Investigator Award recognizes the signiicant contributions of an investigator who has made unusual strides in research related to clinical pharmacology and whose career shows promise of outstanding achievements at a relatively early stage, typically 10 – 12 years post-research degree. The candidate need not be a Member or Fellow of ACCP. Dr. Schmidt’s research focuses on the application of quantitative systems pharmacology to address clinically-relevant questions in the areas of antimicrobial chemotherapy, pediatrics, diabetes, cardiovascular safety and post-menopausal osteoporosis. He is a bright young investigator in drug modeling and clinical pharmacology and has an extraordinary track record of achievement since joining the faculty at the Univ of Florida in 2012, making him a deserving recipient of the 2016 Tanabe Young Investigator Award.

Bristol-Myers Squibb Mentorship in Clinical Pharmacology Award Tuesday, September 27, 2016 | 12:30 – 12:50 pm | Ballroom Salon A – D “From Books to Grooks” Richard Brundage, PharmD, PhD – Professor of Experimental & Clinical Pharmacology, Univ of Minnesota, Coll of Pharmacy The Bristol-Myers Squibb Mentorship in Clinical Pharmacology Award is given annually to an awardee who demonstrates exemplary promotion of clinical pharmacology, with emphasis on training/guidance of junior scientists and/or colleagues. Dr. Brundage is widely recognized for exceptional mentoring abilities and unfailing dedication to students and trainees. He is an extraordinary teacher and an enthusiastic mentor who is passionate about his work. His academic accomplishments and mentorship of the current and future generation of clinical pharmacologists and pharmacists make Dr. Brundage a well-deserving recipient of the 2016 Bristol-Myers Squibb Mentorship in Clinical Pharmacology Award.

11 MED001733 Educational Accreditation

Accreditation Statements The American College of Clinical Pharmacology is accredited by the Accreditation Council for Pharmacy Education (ACPE) as a provider of continuing pharmacy education. The ACPE universal program numbers assigned and hours of credit are noted within each segment of the program for a maximum of 28 Contact Hours. All CPE activities are application-based.

The American College of Clinical Pharmacology is accredited by the Accreditation Council for Continuing Medical Education (ACCME) to provide continuing medical education for physicians.

Designation Statement The American College of Clinical Pharmacology designates this live educational activity for a maximum of 28 AMA PRA Category 1 Credits™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.

Symposium 7: Establishing Biosimilarity: The European Perception, Experience & Future Trends has been planned and implemented in accordance with the accreditation requirements and policies of the Accreditation Council for Continuing Medical Education (ACCME) through the joint providership of the American College of Clinical Pharmacology and the European Federation for Exploratory Medicines Development. The American College of Clinical Pharmacology is accredited by the ACCME to provide continuing medical education for physicians.

Continuing Education Process for 2016 What is CPE Monitor? Attendees interested in earning continuing education credit should CPE Monitor is a national, collaborative effort by speciically request that when they register for the 2016 Annual Meeting. ACPE and the National Association of Boards Attendees who indicated they want to obtain continuing education credit of Pharmacy (NABP) to provide an electronic will be provided with access to post-event tests related to the courses they system for pharmacists/pharmacy technicians attend. Completion of the post-event tests is required to earn the credit and to track their completed Continuing Pharmacy to print continuing education credit certiicates. Post-event tests require a Education (CPE) credits. It also offers state boards 75% passing score. of pharmacy the opportunity to electronically authenticate the CPE units completed by their licensees, rather than requiring pharmacists/pharmacy Attendees seeking CPE credit should, if they have not already done so, technicians to submit proof of completion statements upon request or for provide ACCP with their NABP Proile Number and the month and date random audits. of their birthday via email at [email protected]. The proile number and birthday information is used when ACCP sends CPE credit information to the National Association of Boards of Pharmacy (NABP) using CPE Monitor. Pharmacists/pharmacy technicians are asked to obtain their NABP e-Proile ID by contacting the National Association of Boards of Pharmacy or by contacting NABP Customer Service at 847-391-4406.

Please note: If pharmacists/pharmacy technicians fail to set up their NABP e-Proile Identiication Number, ACCP will not be able to provide the ACPE/NABP with the information which will allow pharmacists/pharmacy technicians to track completed continuing pharmacy education credit(s). ACCP cannot be responsible for individuals who have not taken the necessary steps to obtain their NABP e-Proile Identiication Number and who have not provided this to ACCP prior to CPE post-event testing. For more information, or for answers to Frequently Asked Questions regarding CPE Monitor, please visit Accreditation Council for Pharmacy Education.

MED001734 Educational Activities

Spend an hour each month with ACCP to stay in the forefront of your ield!

ACCP offers several ways to spend that hour each month! Stay current and earn continuing education credit!

• The Journal of Clinical Pharmacology CE Program: Each month an outstanding, relevant article is selected to offer CE credits; available On Demand; • ACCP Virtual Journal Club: Offered at least eight times per year, this is an opportunity to interact with the author of a selected article in a webinar format, including an engaging Q&A session; available as a live webinar, then On Demand; • ACCP Fundamentals Tutorials: Designed to provide a “not too technical” overview of clinical pharmacology, this series is available On Demand; • Therapeutic Dilemmas: This webinar series will continue in the fall of 2016 and is available as a live webinar, then On Demand; • On Demand Library: The ACCP CE Catalog now contains over 20 sessions that you can view On Demand – any time, any place!

Couldn’t attend all the sessions or have a colleague that couldn’t attend? Coming in October – the following sessions from the 2016 ACCP Annual Meeting will be available On Demand:

Attendees of the 2016 Annual Meeting can access these events at no charge. • Symposium 1: Clinical Pharmacology as a Cornerstone for Development of Products Under the Animal Rule: Determining an Effective Dose in Humans • Symposium 2: A 360 View of Immunogenicity: Qualitative & Quantitative ̊ Assessments to Understand Its Implications on Pharmacokinetics, Safety & Eficacy To view the entire • Symposium 3: Helping Advance the Immuno-Oncology Revolution: Trends in ACCP CE Catalog, visit Translational Immuno-Oncology https://ce.accp1.org/ • Symposium 4: Clinical Development of Biologics: Current Strategy, Challenges & Future Considerations • Symposium 5: Addressing Opioid Dependence: Now is the Time • Symposium 6: Treatment of Hepatitis C with Direct-acting Antiviral Drugs: Opportunities & Challenges

13 MED001735 Faculty Disclosure Information

The following Faculty participants have indicated they have a disclosure related to the content of their presentation. Gaurav Bajaj: employee (salary) – Bristol-Myers Squibb Co George R. Gunn, III: employee (salary/stock/stock options) – Janssen Jeffrey Barrett: employee (salary) – Sanofi Research & Development LLC; patent licensing (payment) – Advaxis Inc Jonathan Benjamin: employee (salary/stock) – Amgen Inc Manish Gupta: employee (salary/stock) – Bristol-Myers Squibb Co Suraj G. Bhansali: employee (salary/stock) – Novartis Pharmaceuticals R. Donald Harvey: clinical trial conduct (research funding to Emory) Corp – Amgen Inc, ArQule Inc, AstraZeneca plc, Aveo Pharmaceuticals Inc, Bristol-Myers Squibb Co, Celgene Corp, Cleave Biosciences, Calithera Indranil Bhattacharya: employee (salary/ownership interest) – Pfizer Inc; Biosciences Inc, Eli Lilly & Co, Merck & Co, Novartis, Pfizer Inc, Sanofi, former employee (ownership interest) – GlaxoSmithKline plc Takeda Pharmaceuticals USA Inc; advisory board (honorarium) – Bristol- *Satjit Brar: employee (salary/stock options) – Pfizer Inc Myers Squibb Co, Takeda Pharmaceuticals USA Inc Julie Bullock: employee (salary) – d3 Medicine LLC Tycho Heimbach: employee (salary/stock options) – Novartis *Ganesh Cherala: employee (salary) – Novo Nordisk Inc Pharmaceuticals Corp *Andrew T. Chow: employee (salary/stock) – Amgen Inc Bart Hendriks: employee (salary/stock options) – Merrimack Pharmaceuticals Inc *James Cloyd III: licensing agreement (royalties) – Univ of Minnesota, Ligand Pharmaceuticals Inc; licensing agreement (future royalties) – Univ *Craig W. Hendrix: consulting (fees) – Oak Crest Inst of Science, Univ of of Minnesota, Allaysis LLC; advisory board and licensing agreement California, Los Angeles, Univ of Washington; principal investigator (pending (honorarium/royalty agreement) – Lundbeck; consulting (fees) – Upsher- research contract) – ViiV Healthcare, GlaxoSmithKline plc through Johns Smith Laboratories Inc, Xeris Pharmaceuticals Inc, Neurelis Inc, UCB Inc, Hopkins Eisai Co Ltd Xiao Hu: employee (salary/bonus/stock) – Biogen Inc Dora W. Cohen: employee (salary) – Amgen Inc Kaori Ito: employee (salary/stock) – Pfizer Inc *Lawrence J. Cohen: consultant and committee member (consulting fee) – Manoj P. Jadhav: employee (salary) – CRC Pharma LLC PharMerica Priyanka Jadhav: employee (salary) – CRC Pharma LLC *Alfred Corey: former employee (salary) – PAREXEL Intl; former employee Xiling Jiang: employee (salary/stock) – Johnson & Johnson (stock) – GlaxoSmithKline plc Trevor N. Johnson: employee (salary) – Simcyp Ltd (part of Certara) *Gabriele Dallmann: consulting (fees) – various pharma companies *Mats O. Karlsson: consulting (fees) – Boehringer Ingelheim GmbH, Pfizer John D. Davis: employee (salary/stock options) – Regeneron Inc; (stock) – Pharmetheus AB, Wellhagen & Karlsson AB Pharmaceuticals Inc Sree Kasichayanula: employee (salary/stock) – Amgen Inc *Paul Declerck: speaking and teaching, non-product related (honoraria) – Celltrion Inc, Hospira, Novo Nordisk Inc, Pfizer Inc, Roche *Parag Kumar: collaboration (product) – Matinas BioPharma Holdings Inc *Hartmut Derendorf: consulting (honorarium) – PK PDyne Inc *J. Steven Leeder: consulting (fees go to employer – US Food & Drug Administration) – Neurocrine Biosciences Inc Thomas Eissing: employee (salary/stock) – Bayer Technology Svcs Co/ Bayer AG Lawrence J. Lesko: consulting (fees) – Amgen Inc, Biogen Inc, Relypsa Inc, Merrimack Pharmaceuticals Inc Raffaella Faggioni: employee (salary/stock) – MedImmune LLC/ AstraZeneca plc Chunze Li: employee (salary/stock) – Genentech Inc Michael J. Fossler, Jr: employee (salary/stock) – Trevena Inc Donald E. Mager: Principal Investigator (potential research grant for systems modeling in immuno-oncology) – Bristol-Myers Squibb Co Leonid Gibiansky: consulting (fees) – multiple pharmaceutical companies; published papers in collaboration with Genentech Inc, Bristol-Myers Squibb Christina L. Mayer: employee (salary/stock) – Janssen Research & Co and Roche during 2015 Development LLC *Varun Goel: employee (salary/stock) – Novartis Inst for Biomedical Bernd Meibohm: consulting (fees) – Alexion Pharmaceuticals Inc, Research; spouse: employee (salary/stock) – Alnylam Pharmaceuticals, AstraZeneca plc, Boehringer Ingelheim GmbH, Biogen Inc, F Hoffmann-La Vertex Pharmaceuticals Inc Roche AG, Merck KGaA, Novartis, Sanofi, Teva Pharmaceutical Industries Ltd, Tonix Pharmaceuticals Holding Corp Adam Golden: consulting (fees) – Magellan Health Inc Prasun Mishra: employee (salary/stock) – Genentech Inc – a subsidy of Roche group; former employee (salary/stock) – Gilead Sciences Inc 14

MED001736 Faculty Disclosure Information

Diane R. Mould: consulting (salary) – Projections Research Inc The following Faculty have indicated they have Cara Nelson: employee (salary) – Gilead Sciences Inc no disclosures related to their presentation: Chaitali Passey: employee (salary) – Bristol-Myers Squibb Co; former Darrell R. Abernethy Joseph A. Grillo Michael Pacanowski employee (salary) – Merck & Co *Bilal S. AbuAsal *Sam Harirforoosh Devin Pastoor *Ronald J. Portman: employee (salary/stock) – Novartis Pharmaceuticals *Hae-Young Ahn *Amalia M. Issa *Andrea M. Powell Corp *Shashi Amur *Vijay Ivaturi Mattia Prosperi Sumit Rawal: employee (salary) – Regeneron Pharmaceuticals Inc *Vikram Arya Brian Jacobs *Atiqur Rahman *Zachary Rome: employee (salary) – Patagonia Pharmaceuticals LLC *Gerri Baer *Devanand Jillapalli Amy Rosenberg Karen Rowland Yeo: employee (salary) – Simcyp Ltd (Certara); spouse: *Kimberly L. Bergman *Shyam Kottilil *Anindya Roy employee (salary) – Pfizer Inc *Venkatesh Atul Bhattaram *Walter K. Kraft *Shirley Seo Lorraine M. Rusch: employee (salary) Celerion Inc; spouse: former Eric Brodsky S.W. Johnny Lau Patricia W. Slattum employee (stock) – Cara Therapeutics Inc, Acorda Therapeutics Inc *Gilbert J. Burckart Jinhee Lee *John van den Anker *Huub Schellekens: member ad board (honorarium) – Merck Serono & Leonard Campanello Jiang Liu *Jian Wang Merck & Co; consulting (fees) – Eagle Pharmaceuticals Inc Ruth S. Day Qi Liu Yow-Ming C. Wang Jan-Frederik Schlender: employee (salary) – Bayer Technology Svcs GmbH *Gustavo F. Doncel *Lian Ma *Lynne Yao Edward M. Sellers: consulting (fees) – DL Global Partners Inc Sir Gordon W. Duff *Anshu Marathe Anne Zajicek Jinshan Shen: employee (salary/stock options) – Radius Health Inc; former *Justin C. Earp *Susan McCune Hong Zhao employee (salary/stock options/restricted stock) – Vertex Pharmaceuticals Inc Jeffry Florian *Nitin Mehrotra Ping Zhao Jennifer Sheng: employee (salary) – Bristol-Myers Squibb Co Christine Garnett *Jonathan P. Moorman Jogarao V. Gobburu Lily (Yeruk) Mulugeta Rong Shi: employee (salary/stock) – Bristol-Myers Squibb Co Vikram Sinha: employee (salary) – Merck & Co *This disclosure list includes all 2016 Annual Meeting Faculty. Continuing education credit is offered for ten of the 17 available Workshops and *Konstantine W. Skordos: employee (salary/stock) – Novartis Inst for Symposia. The Faculty participating in Workshops and Symposia offering Biomedical Research; former employee (salary/stock) – Biogen Inc CE credit are noted with an asterisk. *P. Brian Smith: consulting (fees) – Abbvie Inc *Hildegard Sourgens: consulting, medical writing (honoraria) – Bayer AG, Formycon AG, mibe GmbH Arzneimittel, Max Zeller Söhne AG The following activity planners have indicated Sven Stegemann: employee part-time (salary) – Capsugel they have disclosures: Nageshwar Budha: employee (salary) – Genentech Inc; former employee *Mark Sulkowski: consultant/advisory board (honorarium) – AbbVie Inc, (stock) – Hoffmann-La Roche Bristol-Myers Squibb Co, Cocrystal Pharma Inc, Gilead Sciences Inc, Merck & Co, Janssen Pharmaceuticals Inc, Trek Therapeutics PBC Lawrence J. Cohen: consultant and committee member (consulting fee) – PharMerica Yu-Nien (Tom) Sun: employee (salary/stock) – Johnson & Johnson Manish Gupta: employee (salary/stock) – Bristol-Myers Squibb Co Satyendra Suryawanshi: employee (salary/stock) – Bristol-Myers Squibb Co Honghui Zhou: employee (salary/stock/stock options) – Johnson & Johnson Zheng Yang: employee (salary/stock/stock options) – Bristol-Myers Squibb Co Yilong Zhang: employee (salary/stock) – Amgen Inc The following planners have indicated they have Diansong Zhou: employee (salary) – AstraZeneca plc no disclosures: Honghui Zhou: employee (salary/stock/stock options) – Johnson & Johnson Vikram Arya Robert Noveck Amelia N. Deitchman Stephan Schmidt Nilima A. Kshirsagar Jayabharathi Vaidyanathan Nitin Mehrotra John van den Anker

15 MED001737 Pre-meeting Workshops

SATURDAY, SEPTEMBER 24, 2016 l Pre-meeting Workshop 1 l 8:00 am – 12:00 pm

BALLROOM SALON H Bispeciic Antibodies: What Can We Learn from Translational Pharmacokinetics/ Mechanistic PK/PD Modeling? Pharmacodynamics in Biotherapeutic Bernd Meibohm, PhD, Professor & Associate Dean, Univ of Tennessee Health Science Ctr, Coll of Pharmacy Minimum Anticipated Biological Effect Level Dose Selection & Novel Protein 8:40 – 9:10 am Experience in Oncology Clinical Pharmacology Scaffolds & Oversight on ADC: Challenges & Opportunities for Translational PK/PD in ADC Development DISCOVERY TRACK Chunze Li, PhD, Senior Scientist, Genentech Inc

CO-CHAIRS: 9:10 – 9:40 am Honghui Zhou, PhD, Senior Director & Janssen Fellow, Janssen Research & Development LLC Perspectives on Clinical Development of Abbreviated Antibody Constructs Rong Shi, PhD, Clinical Pharmacology Lead, Bristol-Myers Squibb Co Indranil Bhattacharya, PhD, Director, Pizer Inc TARGET AUDIENCE: The target audience includes preclinical and translational pharmacokinetic 9:40 – 10:00 am / Break and pharmacodynamic (PK/PD) scientists, drug development scientists, clinical pharmacologists and those working in clinical and regulatory settings. 10:00 – 10:30 am

GOALS AND OBJECTIVES: Intensive Review of “Expert Group on Phase 1 Clinical Trials: Final Report” Following completion of this activity, the learner will be able to: Sir Gordon W. Duff, Professor & Chair of the Biotechnology & 1. Discuss the advantages of novel scaffolds (eg, modiied ADCC/CDC Biological Sciences Research Council, St Hilda’s Coll, Univ of

response, tissue penetration, speciic targeting) & potential challenges Oxford (eg, PK, immunogenicity) relative to traditional monoclonal antibodies; 2. Discuss approaches to address these challenges and to maximize the advantages of using translational and PK/PD tools; 10:30 – 10:50 am 3. Have a holistic understanding of the available translational and PK/PD To MABEL or Not to MABEL: A Biomarker & tools to face the challenges of drug development with novel scaffolds; Model-based Approach to Dose Selection for 4. Provide an overview and share knowledge on lessons learned about First-in-Human Studies of Biologics the TeGenero anti-CD28 antagonist TNG1412 cytokine storm Raffaella Faggioni, PhD, Senior Director, Clinical Pharmacology incidence; & DMPK, MedImmune LLC/AstraZeneca plc 5. Provide a comprehensive understanding of the concept of the Minimum Anticipated Biological Effect Level (MABEL) by Sir Gordon Duff; 10:50 – 11:10 am 6. Demonstrate examples of MABEL calculations in biologics programs; PK/PD Integration of Nonclinical Data for the 7. Discuss the challenges and methodologies in calculating MABEL for Determination of MABEL & First-in-Human First-in-Human starting dose; Starting Dose: Case Studies with Biologics in 8. Discuss under what circumstances the MABEL approach for First-in- Immunoscience Human starting dose should be used. Zheng Yang, PhD, Director, Bristol-Myers Squibb Co

8:00 – 8:10 am 11:10 – 11:30 am Introduction Regulatory Perspectives in Developing Honghui Zhou, PhD, Senior Director & Janssen Fellow, Janssen Biotherapeutics with Novel Protein Scaffolds Research & Development LLC and Rong Shi, PhD, Clinical Yow-Ming C. Wang, PhD, Clinical Pharmacology (Biologics) Pharmacology Lead, Bristol-Myers Squibb Co Team Leader, US Food & Drug Administration

8:10 – 8:40 am 11:30 am – 12:00 pm 16 Translational Considerations in Developing Panel Discussion

MED001738 Pre-meeting Workshops

SATURDAY, SEPTEMBER 24, 2016 l Pre-meeting Workshop 2 l 8:00 am – 12:00 pm

BALLROOM SALON G

Combating HIV/AIDS: Treatment, 8:35 – 9:00 am Pharmacogenetics & Pre-exposure Pharmacogenetics of HIV Drugs Sam Harirforoosh, PharmD, PhD, Associate Professor, Dept of Prophylaxis Pharmaceutical Sciences, East Tennessee State Univ, Gatton Coll of Pharmacy APPLICATION TRACK Offers both CME and CPE Credit 9:00 – 9:30 am UAN #0238-0000-16-002-L02-P Biomarkers in HIV & HCV Drug Development ACPE – 3.5 CONTACT HOURS/APPLICATION-BASED Shashi Amur, PhD, Scientiic Lead, Biomarker Qualiication Program, US Food & Drug Administration CO-CHAIRS: Sam Harirforoosh, PharmD, PhD, Associate Professor, Dept of Pharmaceutical Sciences, East Tennessee State Univ, Gatton Coll of 9:30 – 10:00 am / Break Pharmacy Ganesh Cherala, PhD, Research Scientist, Research Technologies, Novo 10:00 – 10:30 am Nordisk Inc HIV Pre-exposure Prophylaxis Drug Development: A Clinical Pharmacologist’s Inside TARGET AUDIENCE: View A better understanding of critical contributors of successful Craig W. Hendrix, MD, Wellcome Professor & Director, Johns pharmacotherapy is an important step in delivering optimal healthcare. Hopkins Univ School of Medicine This Workshop will distill information, both evidence-based and theoretical, to the target audience of clinicians, pharmacists and scientists in practice, as well as in clinical research and drug development environments. 10:30 – 11:00 am Development of Multipurpose Technologies for GOALS AND OBJECTIVES: the Prevention of HIV & Unintended Pregnancies: Following completion of this activity, the learner will be able to: Can We Kill Two Birds with One Stone? 1. Describe HIV pharmacotherapy and analyze the potential of pre- Gustavo F. Doncel, MD, PhD, Scientiic Director, CONRAD & exposure prophylaxis (PrEP) in combating the HIV epidemic globally; Professor of Obstetrics & Gynecology, Eastern Virginia Medical 2. Describe the inluence of pharmacogenetics herein and the utility of School pharmacogenetic biomarkers; 3. Demonstrate the utility of multi-purpose technologies to improve 11:00 – 11:30 am reproductive and sexual health; Update on Drug Interactions in HIV 4. Provide an update on drug-drug, drug-food and drug-herb interactions Parag Kumar, PharmD, Director, Clinical Pharmacokinetics in HIV pharmacotherapy. Research Lab, National Inst of Health

8:00 – 8:05 am 11:30 am – 12:00 pm Introduction Panel Discussion Ganesh Cherala, PhD, Research Scientist, Research Technologies, Novo Nordisk Inc

8:05 – 8:35 am Challenges of HIV Infection in 2016 Jonathan P. Moorman, MD, PhD, Professor, East Tennessee State Univ, Quillen Coll of Medicine

17 MED001739 Pre-meeting Workshops

SATURDAY, SEPTEMBER 24, 2016 l Pre-meeting Workshop 3 l 1:30 – 5:30 pm

BALLROOM SALON H

Improving Therapeutics to Better 2:35 – 3:00 pm Regulatory & Clinical Pharmacology Care for Older Adults & the Young Considerations for Developing Drug Products for DISCOVERY & APPLICATION TRACKS Pediatric Patients Gilbert J. Burckart, PharmD, Associate Director for Pediatrics, CO-CHAIRS: US Food & Drug Administration S.W. Johnny Lau, PhD, Senior Clinical Pharmacologist, US Food & Drug Administration 3:00 – 3:30 pm / Break Thomas Eissing, PhD, Head of Systems Pharmacology CV, Bayer Technology Svcs GmbH 3:30 – 4:00 pm Pharmaceutical Drug Product Design in the TARGET AUDIENCE: Context of Effectiveness & Safety & Their The target audience includes clinical pharmacologists, pharmacometricians, Importance in Achieving Therapeutic Outcomes systems pharmacologists, pharmaceutical scientists and clinicians, as well Sven Stegemann, PhD, Professor, Graz Univ of Technology as fellows and students from industry, academia and regulatory institutions. 4:00 – 4:30 pm GOALS AND OBJECTIVES: Applying Pharmacokinetic & Pharmacodynamic Following completion of this activity, the learner will be able to: Principles to Improve Care for Older Adults 1. Understand the issues of developing pharmacotherapy for older adults and the young; Patricia W. Slattum, PharmD, PhD, Professor of Pharmacotherapy & Outcomes Science, Virginia Commonwealth Univ 2. Understand the regulatory aspects of developing pharmacotherapy for older adults and the young; 3. Learn from the regulatory aspect of developing drug products for the 4:30 – 5:00 pm young and apply that to the older adult population; Pharmacometric Approaches to Better Care for 4. Develop patient-centric or age-appropriate pharmaceutical products; Older Adults & the Young 5. Apply pharmacokinetics and pharmacodynamics, as well as Thomas Eissing, PhD, Head of Systems Pharmacology CV, pharmacometrics and systems pharmacology, to better care for older Bayer Technology Svcs GmbH and Jan-Frederik Schlender, MSc, adults and the young. Pharmacist, Scientist Systems Pharmacology, Bayer Technology Svcs GmbH

1:30 – 1:40 pm 5:00 – 5:30 pm Introduction Panel Discussion S.W. Johnny Lau, PhD, Senior Clinical Pharmacologist, US Food & Drug Administration

1:40 – 2:10 pm Medication Issues & Potential Solutions for Frail Older Adults Adam Golden, MD, MBA, Professor, Internal Medicine, Univ of Central Florida, Coll of Medicine, Associate Chief of Staff, Geriatrics & Extended Care, Orlando Veterans Affairs Medical Ctr

2:10 – 2:35 pm Are There Unique Issues for the Development of Drug Products for the Older Adult? Darrell R. Abernethy, MD, PhD, Associate Director for Drug Safety, Ofice of Clinical Pharmacology, US Food & Drug Administration

MED001740 Pre-meeting Workshops

SATURDAY, SEPTEMBER 24, 2016 l Pre-meeting Workshop 4 l 1:30 – 5:00 pm

BALLROOM SALON G

The Other Bound of the Therapeutic 2:10 – 2:40 pm Window: Exposure-Safety Analysis to Strategies for Exposure-Safety Modeling & Simulation in Oncology with a Focus on Trial Inform Dosing Decisions in Oncology Execution Aspects Mats O. Karlsson, PhD, Professor, Dept of Pharmaceutical DISCOVERY TRACK Biosciences, Uppsala Univ Offers both CME and CPE Credit UAN #0238-0000-16-003-L05-P 2:40 – 3:00 pm ACPE – 3 CONTACT HOURS/APPLICATION-BASED Q&A

CO-CHAIRS: 3:00 – 3:30 pm / Break Justin C. Earp, PhD, Pharmacometrics Reviewer, US Food & Drug Administration 3:30 – 4:00 pm Anshu Marathe, PhD, Team Leader, Div of Clinical Pharmacology II, US Food & Drug Administration Exposure-Safety Analysis for Oncology Drugs: An Industry Perspective TARGET AUDIENCE: Varun Goel, PhD, Fellow, Clinical Pharmacology, Novartis Inst for Biomedical Research The target audience includes drug development scientists from the pharmaceutical industry working in the area of oncology, academic organizations, scientists from cancer hospitals involved in drug 4:00 – 4:30 pm development of oncology agents and regulatory scientists working in Balancing Exposure-Safety & Eficacy Analysis the area of oncology. Although the focus of the activity is in the oncology for Deriving Dosing in Oncology: Case Examples therapeutic area, the principles discussed in this topic can be applied to Satjit Brar, PharmD, PhD, Associate Director, Clinical other therapeutic areas as well. Pharmacology, Pizer Inc

GOALS AND OBJECTIVES: 4:30 – 5:00 pm Following completion of this activity, the learner will be able to: 1. Understand the uniqueness of characterizing exposure-response for Panel Discussion safety in oncology drug development programs; (including Konstantine W. Skordos, PhD [Novartis Inst for 2. Review current practices and identify the methodological challenges Biomedical Research] and Atiqur Rahman, PhD [US Food & Drug involved in conducting exposure-safety analyses for oncology agents; Administration]) 3. Highlight case studies demonstrating the common methodologies/ challenges in conducting oncology exposure-safety analyses; 4. Analyze and compare possible approaches for adequate exposure- safety analyses that can contribute to informed dosing decisions.

1:30 – 1:40 pm Introduction Justin C. Earp, PhD, Pharmacometrics Reviewer, US Food & Drug Administration

1:40 – 2:10 pm Exposure-Safety Analyses to Drive Decision Making in Oncology Anshu Marathe, PhD, Team Leader, Div of Clinical Pharmacology II, US Food & Drug Administration

19 MED001741 Symposia

SUNDAY, SEPTEMBER 25, 2016 l Symposium 1 l 8:00 am – 12:00 pm

BALLROOM SALON A–C

Clinical Pharmacology as a 8:15 – 8:40 am The Animal Rule: Approval of New Drugs & Cornerstone for Development of Biological Products When Human Eficacy Products Under the Animal Rule: Studies Are Not Ethical or Feasible Andrea M. Powell, PhD, Pharmacologist, Counter-terrorism & Determining an Effective Dose in Emergency Coordination, US Food & Drug Administration Humans 8:40 – 9:05 am The Animal Rule: The Role of Clinical DISCOVERY TRACK Pharmacology in Determining an Effective Dose Offers both CME and CPE Credit in Humans UAN #0238-0000-16-004-L01-P Kimberly L. Bergman, PharmD, Lead Pharmacologist, US Food & ACPE – 3.5 CONTACT HOURS/APPLICATION-BASED Drug Administration

CO-CHAIRS: 9:05 – 9:30 am Nitin Mehrotra, PhD, Team Leader, Div of Pharmacometrics, US Food & Perspective on the Clinical Pharmacology Drug Administration Approach for Rational Choice of Drug & Dose Kimberly L. Bergman, PharmD, Lead Pharmacologist, US Food & Drug in Product Development Under the Animal Rule: Administration Example for Treating Patients Acutely Exposed to Myelosuppressive Doses of Radiation TARGET AUDIENCE: Andrew T. Chow, PhD, Executive Director, Amgen Inc The target audience includes clinical pharmacologists, pharmacometricians and translational medicine scientists from the pharmaceutical industry, 9:30 – 10:00 am / Break academia and regulatory agencies who have an interest in applying and/ or currently apply the principles of clinical pharmacology modeling and 10:00 – 10:25 am simulation in drug development of products under the Animal Rule. The Use of Modeling & Simulation in the Raxibacumab Development Program GOALS AND OBJECTIVES: Alfred Corey, BS, Consultant, AC Pharmaco LLC Following completion of this activity, the learner will be able to: 1. Understand the drug development and approval process of products 10:25 – 10:50 am under the Animal Rule; 2. Analyze and understand the role of clinical pharmacology modeling Application of Quantitative Clinical Pharmacology and simulation in dose selection in humans for development of products in Dose Selection for Products Developed Under under the Animal Rule; the Animal Rule: Case Studies 3. Highlight the case studies where clinical pharmacology modeling and Lian Ma, PhD, Pharmacometrics Reviewer, US Food & Drug simulation played a signiicant role in drug development or regulatory Administration decisions. 10:50 am – 12:00 pm 8:00 – 8:15 am Panel Discussion Introduction: Setting the Stage Nitin Mehrotra, PhD, Team Leader, Div of Pharmacometrics, US Food & Drug Administration

MED001742 Symposia

SUNDAY, SEPTEMBER 25, 2016 l Symposium 2 l 8:00 – 9:30 am

BALLROOM SALON D

A 360⁰ View of Immunogenicity: 8:05 – 8:30 am Qualitative & Quantitative A Qualitative Look at Immunogenicity Data During Drug Development Assessments to Understand Its George R. Gunn III, PhD, Associate Scientiic Director, Janssen Research & Development LLC Implications on Pharmacokinetics, 8:30 – 8:55 am Safety & Eficacy Quantitative Approaches to Assess DISCOVERY TRACK Immunogenicity During Drug Development of Biologics CO-CHAIRS: Leonid Gibiansky, PhD, President, QuantPharm LLC Chaitali Passey, PhD, Senior Research Investigator, Bristol-Myers Squibb Co 8:55 – 9:20 am Sumit Rawal, PhD, Scientist, Regeneron Pharmaceuticals Inc Risk Assessment & Mitigation Strategies for Immunogenicity of Therapeutic Proteins TARGET AUDIENCE: Amy Rosenberg, MD, Supervisory Medical Oficer, Div of The target audience includes clinical pharmacologists, clinicians, Therapeutic Proteins, US Food & Drug Administration pharmacometricians, regulators and bioanalytical scientists. 9:20 – 9:30 am GOALS AND OBJECTIVES: Q&A Following completion of this activity, the learner will be able to: 1. Understand the best practices for reporting and visualization of immunogenicity data; 2. Demonstrate and compare quantitative approaches to assess the impact of immunogenicity on pharmacokinetics; 3. Understand the implications of immunogenicity on safety and eficacy of therapeutic protein products in a clinical setting.

8:00 – 8:05 am Session Overview Chaitali Passey, PhD, Senior Research Investigator, Bristol-Myers Squibb Co

21 MED001743 Symposia

SUNDAY, SEPTEMBER 25, 2016 l Symposium 3 l 10:00 am – 12:00 pm

BALLROOM SALON D

Helping Advance the Immuno- 10:10 – 10:30 am Bench to Bedside: Recent Examples in Oncology Revolution: Trends in Translating Medicine in Cancer Drug Translational Immuno-Oncology Development Jonathan Benjamin, MD, Medical Director, Amgen Inc DISCOVERY & APPLICATION TRACKS 10:30 – 10:50 am CO-CHAIRS: Understanding the Utility of Imaging in Targeted Sree Kasichayanula, PhD, Principal Scientist, Amgen Inc Drug Delivery: Opportunities & Challenges in Yu-Nien (Tom) Sun, PhD, Senior Director, Janssen Research & Immuno-Oncology Development LLC Bart Hendriks, PhD, Senior Director of Nanoimaging, Merrimack Pharmaceuticals Inc TARGET AUDIENCE: The target audience includes physicians, pharmacists, researchers 10:50 – 11:10 am and regulators who are seeking to understand translational research in Preclinical Models for Deining Eficacy of oncology immunotherapy, along with pharmacokinetics/pharmacodynamics Immunotherapy Combinations: Mapping the Road (PK/PD) and systems modeling and the future of drug development to for Acceleration to Clinic treat patients with cancer. The Symposium attendees will be able to learn Christina L. Mayer, PharmD, Senior Scientist, Biologics Clinical and appreciate the utility of novel technologies, such as imaging, and Pharmacology, Janssen Research & Development LLC their roles, along with PK/PD, in targeted therapy in oncology. Recent translational advances that helped accelerate combination immunotherapy development, along with future outlook in this disease area, will also be 11:10 – 11:30 am covered. Advances & the Future Role of Immuno-Oncology Systems Models GOALS AND OBJECTIVES: Donald E. Mager, PharmD, PhD, Associate Professor, Univ at Following completion of this activity, the learner will be able to: Buffalo, State Univ of New York 1. Understand recent advances in translational drug development in cancer immunotherapy; 11:30 am – 12:00 pm 2. Appreciate the utility of imaging in oncology translational models; Panel Discussion 3. Conceptualize the differences in combination immunotherapy and the utility of translational models in acceleration of combination oncology development; 4. Compare the role of traditional PK/PD models and recent advances in systems modeling.

10:00 – 10:10 am Introduction Yu-Nien (Tom) Sun, PhD, Senior Director, Janssen Research & Development LLC

MED001744 Symposia

SUNDAY, SEPTEMBER 25, 2016 l Symposium 4 l 1:30 – 5:30 pm

BALLROOM SALON A–C

Clinical Development of Biologics: 1:40 – 2:10 pm Current Strategy, Challenges & Clinical Pharmacology Considerations for Biologics: Important Concepts Future Considerations Diane R. Mould, PhD, President, Projections Research Inc

DISCOVERY TRACK 2:10 – 2:35 pm Communicating Concepts Correctly CO-CHAIRS: John D. Davis, BPharm, PhD, Senior Director, Regeneron Gaurav Bajaj, PhD, Senior Research Investigator, Bristol-Myers Squibb Co Pharmaceuticals Inc Ping Zhao, PhD, Lead, PBPK Program, Div of Pharmacometrics, US Food & Drug Administration 2:35 – 3:00 pm

TARGET AUDIENCE: How the Development of Combination Therapy in Biologics Can Be Different Than Monotherapy The session will cover the challenges in clinical pharmacology associated with development of early clinical candidates during Phase 1/2 stages. The Manish Gupta, PhD, Director, Clinical Pharmacology & target audience includes clinical pharmacologists and pharmacometricians Pharmacometrics, Bristol-Myers Squibb Co from the pharmaceutical and biotech industries and academia, clinicians and regulatory scientists, scientists working on early drug development 3:00 – 3:30 pm / Break and graduate students/trainees in pharmaceutical sciences and clinical pharmacology. 3:30 – 4:00 pm Application of Physiologically-based GOALS AND OBJECTIVES: Pharmacokinetics in Biologics in Drug Following completion of this activity, the learner will be able to: Development With a Case Example to Predict 1. Understand challenges in clinical development of monoclonal Disease-mediated Therapeutic Protein Interaction antibodies (mAbs); Xiling Jiang, PhD, Senior Scientist, Janssen Research & 2. Discuss dose-optimization strategies of mAbs for pivotal trials and Development LLC the possible strategies for mAbs that are approved and are being used in combination with another biologic; 4:00 – 4:30 pm 3. Discuss challenges related to characterization of non-linear Application of Physiologically-based pharmacokinetics of mAbs and the implication on clinical Pharmacokinetic Models to Predict Drug development; Interactions for Antibody-Drug Conjugates 4. Analyze and predict immunogenicity of mAbs and the impact on Chunze Li, PhD, Senior Scientist, Genentech Inc clinical eficacy and safety; 5. Understand the current status, limitation, challenges and future 4:30 – 5:00 pm directions of using physiologically-based pharmacokinetic and pharmacodynamic (PBPK/PD) models in drug development for Regulatory Considerations in Biologics biologics; Development 6. Apply PBPK models to predict drug interaction potential for antibody- Hong Zhao, PhD, Master Reviewer of Clinical Pharmacology/ drug conjugates. Team Leader, US Food & Drug Administration

5:00 – 5:30 pm 1:30 – 1:40 pm Panel Discussion Introduction Gaurav Bajaj, PhD, Senior Research Investigator, Bristol-Myers Squibb Co

23 MED001745 Symposia

SUNDAY, SEPTEMBER 25, 2016 l Symposium 5 l 1:30 – 3:00 pm

BALLROOM SALON D

Addressing Opioid Dependence: 1:35 – 1:55 pm Addiction: An Imprecise Problem in a World of Now is the Time Precision Medicine APPLICATION TRACK Edward M. Sellers, MD, PhD, Professor Emeritus, Pharmaceuticals, Medicine & Psychiatry, Univ of Toronto CO-CHAIRS: Lorraine M. Rusch, PhD, Vice President, Commercial Development, 1:55 – 2:20 pm Celerion Inc HHS: Policies to Address Opioid-drug Related Michael J. Fossler, Jr, PharmD, PhD, Vice President, Quantitative Overdose, Death & Dependence Sciences, Trevena Inc Jinhee Lee, PharmD, Senior Pharmacy Advisor, Substance Abuse & Mental Health Svcs Administration TARGET AUDIENCE: The target audience includes clinical pharmacologists involved in basic and 2:20 – 2:40 pm applied clinical research focused on analgesia management, pharmacists Gloucester Police Department Angel Initiative & involved in illing and reporting opioid prescriptions, medical directors, chief the Police Assisted Addiction Recovery Initiative medical oficers of organizations developing new chemical entities for pain (PAARI) management, physicians (both those practicing in the pain management Leonard Campanello, MS, Chief of Police, City of Gloucester, MA area and those not as familiar) and health economics professionals. Police Dept MAFE

GOALS AND OBJECTIVES: 2:40 – 3:00 pm Following completion of this activity, the learner will be able to: 1. Understand and delineate common challenges that persons suffering Panel Discussion with substance use disorder face while attempting to secure treatment options such as suboxone and/or methadone and counseling programs; 2. Demonstrate a basic understanding of the principles of the neurobiology involved in addictions, treatment options available and basic medical practice towards the management of addiction; 3. Consider the alternative of decriminalizing those who voluntarily commit to substance treatment through novel community policing programs that secure immediate aid for addicts in need; 4. Understand and utilize new programs (training, grants, increased buprenorphine prescribing) proposed by the US Health & Human Services (HHS) and budgeted for 2016 ($133M in new funding).

1:30 – 1:35 pm Introduction Michael J. Fossler, Jr, PharmD, PhD, Vice President, Quantitative Sciences, Trevena Inc

MED001746 Symposia

SUNDAY, SEPTEMBER 25, 2016 l Symposium 6 l 3:30 – 5:30 pm

BALLROOM SALON D

Treatment of Hepatitis C with Direct- 3:35 – 4:00 pm acting Antiviral Drugs: Opportunities Challenges Associated with the Use of Direct- acting Antiviral Drugs in Speciic Populations & Challenges Mark Sulkowski, MD, Professor of Medicine, Johns Hopkins Univ School of Medicine APPLICATION TRACK Offers both CME and CPE Credit 4:00 – 4:25 pm UAN #0238-0000-16-005-L01-P Ultra-short Duration Therapy: Reality or Myth? ACPE – 2 CONTACT HOURS/APPLICATION-BASED Shyam Kottilil, MD, PhD, Professor of Medicine, Inst of Human Virology, Univ of Maryland This Symposium is supported in part by an Educational Grant from Merck Sharp & Dohme Corp (A subsidiary of Merck & Co Inc) 4:25 – 4:50 pm Knowledge Gaps in the Development of CO-CHAIRS: Direct-acting Antiviral Drugs: How Clinical Vikram Arya, PhD, Silver Spring, MD Pharmacology Has Contributed to Closing Them Shirley Seo, PhD, Team Leader, Ofice of Clinical Pharmacology, US Food ShirleySeo, PhD, Team Leader, Ofice of Clinical Pharmacology, & Drug Administration US Food & Drug Administration

TARGET AUDIENCE: 4:50 – 5:30 pm The target audience includes physicians, clinical pharmacologists, Panel Discussion pharmacists and academic research scientists.

GOALS AND OBJECTIVES: The goal of this course is to provide participants with an insight into the various strategies for treatment of Hepatitis C and to discuss the various challenges of treating HCV-infected patients in the era of all oral direct- acting antiviral (DAA) therapies. Following completion of this activity, the learner will be able to: 1. Understand recent advances in the treatment of Hepatitis C with DAA drugs and identify the various knowledge gaps; 2. Demonstrate knowledge of the role of clinical pharmacology in optimizing the dose and treatment duration of DAAs for various genotypes; 3. Understand the various dosing recommendations of DAAs in some speciic populations (for example HIV/HCV co-infected and transplant patients).

3:30 – 3:35 pm Introduction Vikram Arya, PhD, Silver Spring, MD

25 MED001747 Symposia

MONDAY, SEPTEMBER 26, 2016 l Symposium 7 l 8:00 am – 12:00 pm

BALLROOM SALON A–C

Establishing Biosimilarity: 8:00 – 8:20 am The European Perception, Introduction/EMA & FDA Guidance Review Hildegard Sourgens, MD, PhD, President Elect, European Experience & Future Trends Federation for Exploratory Medicines Development

DISCOVERY & APPLICATION TRACKS 8:20 – 9:00 am Offers both CME and CPE Credit How Similar is Similar? The European Biosimilar UAN #0238-9999-16-006-L01-P Quality Experience ACPE – 3.5 CONTACT HOURS/APPLICATION-BASED Paul Declerck, PhD, Professor, Dean of the Faculty of Pharmaceutical Sciences, Univ of Leuven This activity has been planned and implemented in accordance with the accreditation requirements and policies of the Accreditation Council for 9:00 – 9:40 am Continuing Medical Education (ACCME) through the joint providership Clinical Strategies for Biosimilar Development: A of the American College of Clinical Pharmacology and the European European Perspective Federation for Exploratory Medicines Development. The American College Gabriele Dallmann, PhD, Co-founder, Biopharma Excellence GbR of Clinical Pharmacology is accredited by the ACCME to provide continuing medical education for physicians. 9:40 – 10:10 am / Break

CO-CHAIRS: 10:10 – 10:50 am Hildegard Sourgens, MD, PhD, President Elect, European Federation for Exploratory Medicines Development The European Experience With Safety Testing of Biologicals and Biosimilars Hartmut Derendorf, PhD, Distinguished Professor & Chair, V. Ravi Chandran Professor in Pharmaceutical Sciences, Univ of Florida Huub Schellekens, MD, PhD, Chair, Professor in Pharmaceutical Biotechnology, Utrecht Univ TARGET AUDIENCE: The target audience includes healthcare professionals who are involved 10:50 – 11:30 am in the research and development of biopharmaceuticals/biosimilars, Current Status & Future Trends in Biologics & regulatory affairs (competent authorities; pharmaceutical industry), Biosimilar Development & Approval in the US pharmacovigilance and/or biotech start-ups. Hae-Young Ahn, PhD, RAC, Deputy Director, Div of Clinical Pharmacology III, US Food & Drug Administration GOALS AND OBJECTIVES: The goal is for participants to learn from the European Medicines Agency’s 11:30 am – 12:00 pm (EMA) 15-year experience in biosimilars and assess if similar concepts can Panel Discussion be adopted in the US. Following completion of this activity, the learner will be able to: 1. Demonstrate the primary contribution of analytical comparability and its meaning for clinical development; 2. Analyze the European experience with respect to clinical and nonclinical development programs, approval success, post-marketing performance and the failures of European biosimilar programs (as far as these can be made public); 3. Develop the impact of pharmacokinetics/pharmacodynamics to detect differences between a reference medicinal product and a biosimilar; 4. Demonstrate the safety of biologicals and biosimilars based on the European experience; 5. Demonstrate the discriminative power of analytics and pharmacokinetic and/or pharmacodynamic proiles vs Phase 3 trials.

MED001748 Symposia

MONDAY, SEPTEMBER 26, 2016 l Symposium 8 l 8:00 – 9:30 am

BALLROOM SALON D

Informing Pediatric Development 8:15 – 8:30 am Programs: Leveraging Big Data The Value of Historical Electronic Health Records Data to Guide Relevant Clinical Questions DISCOVERY TRACK Around Pediatric Standard of Care: A Perspective from Cerner CO-CHAIRS: Brian Jacobs, MD, Vice President, Chief Medical Information Jeffrey Barrett, PhD, Vice President, Translational Informatics, Sanoi Oficer & Chief Information Oficer, Children’s National Health System Lily (Yeruk) Mulugeta, PharmD, Scientiic Lead for Pediatrics, Div of Pharmacometrics, US Food & Drug Administration 8:30 – 8:45 am TARGET AUDIENCE: Combining Bedside & Clinical Research Data The target audience includes drug development scientists in both to Inform Disease Progression and Outcomes/ academia and industry, regulators, clinicians, clinical pharmacologists and Biomarker Selection statisticians. Diane R. Mould, PhD, President, Projections Research Inc

GOALS AND OBJECTIVES: 8:45 – 9:00 am One of the more compelling challenges in pediatric drug development, as well as the consideration on expanded indications in children for existing Deriving Insight & Value from Electronic Health approved agents, is understanding the pediatric disease progression. Records: Opportunities and Challenges of Data sources that include large and unstructured formats, ie, Big Data, Neonatal Clinical Research in the Big Data Era are available, but their role in pediatric drug development is only at the P. Brian Smith, MD, MHS, MPH, Professor of Pediatrics, Duke genesis stage. Sources of Big Data include the electronic medical record Univ Medical Ctr (EMR) and large multi-institution administrative databases which consist of information on demographics, laboratory indings, microbiology data, 9:00 – 9:15 am medical order, procedures, surgery and clinical outcomes. The session will Using Existing Data Sources for Advancing explore potential frameworks for how existing data can be used in pediatric Clinical Trials: A Regulatory Perspective drug development to optimize protocol design and enhance patient Jeffry Florian, PhD, Team Leader, Div of Pharmacometrics, US recruitment. The session will highlight case studies and discuss unique Food & Drug Administration data sources that can be leveraged. Following completion of this activity, the learner will be able to: 9:15 – 9:30 am 1. Review varying types of data that can be leveraged to support pediatric trial design; Panel Discussion 2. Present examples on how eficiency of pediatric trials can be improved (including Brian Jacobs, MD [Children’s National Health System], using existing data; P. Brian Smith, MD, MHS, MPH [Duke Univ Medical Ctr], Anne 3. Discuss the limitations and generalizability of data from EMR as it Zajicek, MD, PharmD [National Inst of Health], Lynne Yao, MD applies to pediatric drug development. [US Food & Drug Administration], Vikram Sinha, PhD [Merck Research Laboratories] and Diane R. Mould, PhD [Projections Research Inc]) 8:00 – 8:15 am Introduction to Big Data & Its Relevance for Pediatric Drug Development Jeffrey Barrett, PhD, Vice President, Translational Informatics, Sanoi

27 MED001749 Symposia

MONDAY, SEPTEMBER 26, 2016 l Symposium 9 l 10:00 am – 12:00 pm

BALLROOM SALON D

Little Children, Big Challenges: The 10:05 – 10:20 am Ontogeny of Drug-metabolizing Enzymes & Drug Problems for Neonatal Drug Trials & Transporters: What is Known & Unknown the Way Forward J. Steven Leeder, PharmD, PhD, Director, Div of Clinical Pharmacology, Toxicology & Therapeutic Innovation, Children’s APPLICATION TRACK Mercy Hosp Offers both CME and CPE Credit 10:20 – 10:35 am UAN #0238-0000-16-007-L01-P Considerations on Clinical Outcome Measures & ACPE – 2 CONTACT HOURS/APPLICATION-BASED Biomarkers: Pediatric Trials Network Experience CO-CHAIRS: P. Brian Smith, MD, MHS, MPH, Professor of Pediatrics, Duke Univ Medical Ctr Jian Wang, PhD, Senior Clinical Pharmacologist, US Food & Drug Administration 10:35 – 10:50 am John van den Anker, MD, PhD, Chief, Div of Clinical Pharmacology, Children’s National Health System Innovative Trial Designs for Neonatal Studies Lynne Yao, MD, Director, Div of Pediatric & Maternal Health, US TARGET AUDIENCE: Food & Drug Administration The target audience includes clinical pharmacologists from both pharmaceutical & biotechnology companies and regulatory agencies, 10:50 – 11:05 am pharmacometricians, clinical researchers and drug development scientists Extrapolation in Neonates: What is the Role of who have an interest in applying and/or currently apply principles Clinical Pharmacology? of pediatric clinical pharmacology to innovate and accelerate drug Ronald J. Portman, MD, Executive Director, Pediatric Therapeutic development for neonatal patients. Area, Novartis Pharmaceuticals Corp

GOALS AND OBJECTIVES: 11:05 – 11:15 am Following completion of this activity, the learner will be able to: 1. Review the challenges and opportunities in neonatal drug development Neonatal Safety Studies from a preclinical and clinical pharmacology perspective; Gilbert J. Burckart, PharmD, Associate Director for Pediatrics, US 2. Demonstrate how understanding ontogeny and clinical data together Food & Drug Administration inform neonatal dose selection; 3. Discuss approaches that can be used to improve eficiency and 11:15 – 11:30 am feasibility of neonatal trials; International Neonatal Consortium Update 4. Evaluate the use of optimal clinical trial design in neonatal patients; Susan McCune, MD, Deputy Director, Ofice of Translational 5. Discuss safety evaluation in neonates; Sciences, US Food & Drug Administration 6. Update participants about the FDA pediatric guidances and the international neonatal consortium. 11:30 am – 12:00 pm Panel Discussion 10:00 – 10:05 am (including Gerri Baer, MD, Medical Oficer/Neonatologist, US Overview: Challenges & Opportunities Food & Drug Administration) John van den Anker, MD, PhD, Chief, Div of Clinical Pharmacology, Children’s National Health System

MED001750 Symposia

MONDAY, SEPTEMBER 26, 2016 l Symposium 10 l 1:30 – 5:30 pm

BALLROOM SALON A–C

Streamlining Clinical Pharmacology 2:10 – 2:35 pm Strategies During Early Development: Early Assessment of Drug-Drug Interaction Potential in Combination Clinical Trials Assessment of Drug-Drug Konstantine W. Skordos, PhD, Director, Clinical Pharmacology, Translational Clinical Oncology, Novartis Pharmaceuticals Corp Interactions, Food Effect & QTc 2:35 – 3:00 pm DISCOVERY TRACK Food Effects in Early Cancer Drug Development: CO-CHAIRS: More Than Meets the Eye Suraj G. Bhansali, MS, PhD, Manager, Oncology Clinical Pharmacology, Lawrence J. Lesko, PhD, Clinical Professor & Director, Ctr for Novartis Pharmaceuticals Corp Pharmacometrics & Systems Pharmacology, Univ of Florida Xiao Hu, PhD, Senior Pharmacometrician, Biogen Inc 3:00 – 3:30 pm / Break

TARGET AUDIENCE: 3:30 – 4:00 pm The target audience includes clinical pharmacologists, pharmacokineticists and clinicians. The international scientiic community in academia, the It’s Time to Revise ICH E14 Guidance: The pharmaceutical & biotechnology industries or regulatory authorities History, Opportunities, Challenges & Directions associated with clinical drug development will also be particularly of QTc Analysis interested in this topic based on the common challenges faced during Christine Garnett, PharmD, Clinical Analyst, US Food & Drug development of oncology compounds. Administration

GOALS AND OBJECTIVES: 4:00 – 4:30 pm Following completion of this activity, the learner will be able to: Key Considerations in Study Design & Analysis 1. Understand challenges speciic to oncology clinical drug development; Methods of New Drugs’ QT Assessment Using a 2. Evaluate the timing of clinical pharmacology studies for oncology Phase 1 Study compounds in reference to the normal clinical development plan and Jiang Liu, PhD, Scientiic Lead for QT, US Food & Drug discuss beneits of doing studies at an early stage; Administration 3. Discuss strategies for early assessment of food effect, QTc and drug- drug interactions (DDI); 4:30 – 5:00 pm 4. Discuss considerations to streamline proarrhythmic risk assessment during early clinical development. Using Concentration-QTc Analysis to Obtain a Waiver for TQT Study Cara Nelson, PhD, Clinical Pharmacologist II, Gilead Sciences 1:30 – 1:40 pm Inc Introduction Suraj G. Bhansali, MS, PhD, Manager, Oncology Clinical 5:00 – 5:30 pm Pharmacology, Novartis Pharmaceuticals Corp and Xiao Hu, PhD, Panel Discussion Senior Pharmacometrician, Biogen Inc

1:40 – 2:10 pm An Overview of Early Clinical Pharmacology Studies: Assessment of Drug-Drug Interactions, Food Effect & QTc in an Oncology Setting Suraj G. Bhansali, MS, PhD, Manager, Oncology Clinical Pharmacology, Novartis Pharmaceuticals Corp

29 MED001751 Symposia

MONDAY, SEPTEMBER 26, 2016 l Symposium 11 l 1:30 – 3:00 pm

BALLROOM SALON D

Cutting-edge Abstract Presentations 1:40 – 1:49 pm

DISCOVERY & APPLICATION TRACKS Abstract #1 Wayne A. Colburn Memorial Award (5 minute presentation; Offers both CME and CPE Credit 3 minutes of questions) UAN #0238-0000-16-008-L01-P ACPE – 1.5 CONTACT HOURS/APPLICATION-BASED 1:50 – 1:59 pm Abstract #2 CO-CHAIRS: Lawrence J. Cohen, PharmD, Professor & Coordinator of New Member Abstract Award (5 minute presentation; 3 minutes Interprofessional Education, Univ of North Texas System Coll of Pharmacy of questions) Walter K. Kraft, MD, Professor, Thomas Jefferson Univ 2:00 – 2:09 pm Amalia M. Issa, PhD, Professor & Chair, Personalized Medicine & Targeted Therapeutics, Univ of the Sciences Abstract #3 (5 minute presentation; 3 minutes of questions) TARGET AUDIENCE: The target audience includes physicians, pharmacists and clinical 2:10 – 2:19 pm pharmacologists involved in basic and applied clinical research who are Abstract #4 interested in state-of-the-art investigations. (5 minute presentation; 3 minutes of questions)

GOALS AND OBJECTIVES: 2:20 – 2:29 pm Following completion of this activity, the learner will be able to: Abstract #5 1. Describe at least two of the abstracts from a curated list of the top abstracts submitted for the 2016 ACCP Annual Meeting; (5 minute presentation; 3 minutes of questions) 2. Identify a novel area or focus of clinical pharmacology research; 2:30 – 2:39 pm 3. Interact with the authors of multiple cutting-edge abstracts from a variety of disciplines. Abstract #6 (5 minute presentation; 3 minutes of questions) 1:30 – 1:40 pm 2:40 – 2:49 pm Introduction & Abstract Award Winner Announcement Abstract #7 Lawrence J. Cohen, PharmD, Professor & Coordinator of (5 minute presentation; 3 minutes of questions) Interprofessional Education, Univ of North Texas System Coll of Pharmacy, Walter K. Kraft, MD, Professor, Thomas Jefferson 2:50 – 2:59 pm Univ and Amalia M. Issa, PhD, Professor & Chair, Personalized Abstract #8 Medicine & Targeted Therapeutics, Univ of the Sciences (5 minute presentation; 3 minutes of questions)

3:00 pm Wrap Up

MED001752 Symposia

MONDAY, SEPTEMBER 26, 2016 l Symposium 12 l 3:30 – 5:30 pm

BALLROOM SALON D

Rethinking Clinical Pharmacology- 3:40 – 4:00 pm Developing Clinical Pharmacology Labeling for related Labeling for Improved Utility & Improved Utility & Comprehension: Industry Comprehension Perspective Dora W. Cohen, BA, MA, Executive Director, Global Labeling, DISCOVERY & APPLICATION TRACKS Amgen Inc Offers both CME and CPE Credit UAN #0238-0000-16-009-L03-P 4:00 – 4:20 pm ACPE – 2 CONTACT HOURS/APPLICATION-BASED Strategies for Enhancing Quality, Utility & Clarity in Clinical Pharmacology Labeling: A Regulatory CO-CHAIRS: Perspective Joseph A. Grillo, PharmD, Associate Director for Labeling & Health Joseph A. Grillo, PharmD, Associate Director for Labeling & Communications, US Food & Drug Administration Health Communications, US Food & Drug Administration Julie Bullock, PharmD, Senior Director, d3 Medicine LLC 4:20 – 4:40 pm TARGET AUDIENCE: Utility & Comprehension of Clinical The target audience includes scientists in the pharmaceutical industry, Pharmacology Labeling: A Healthcare Provider healthcare providers and regulatory professionals. Perspective Patricia W. Slattum, PharmD, PhD, Professor of Pharmacotherapy GOALS AND OBJECTIVES: & Outcomes Science, Virginia Commonwealth Univ Following completion of this activity, the learner will be able to: 1. Present stakeholder experiences (eg, industry, academia/clinical 4:40 – 5:00 pm practice, FDA, cognitive scientist) regarding clinical pharmacology- Enhanced Displays of Clinical Pharmacology related information in labeling; Information: Effects on Attention, Comprehension 2. Explore different labeling formats (eg, tables, igures, structured text) & Memory to further enhance the presentation of clinical pharmacology information Ruth S. Day, PhD, Director, Medical Cognition Laboratory, in labeling; Psychology & Neuroscience, Duke Univ 3. Provide an overview of key principles included in the FDA Clinical Pharmacology Section Labeling guidance (under revision). 5:00 – 5:30 pm Panel Discussion 3:30 – 3:40 pm (including Eric Brodsky, MD [US Food & Drug Administration]) Introduction Julie Bullock, PharmD, Senior Director, d3 Medicine LLC

31 MED001753 Symposia

TUESDAY, SEPTEMBER 27, 2016 l Symposium 13 l 8:00 am – 12:00 pm

BALLROOM SALON A–C

Orphan Drug Development in Adults 8:15 – 8:40 am FDA Flexibility in Facilitating Drug Development & Pediatrics: Industry, Academia & in Rare Diseases Regulatory Perspectives Devanand Jillapalli, MD, Medical Oficer, Ofice of Orphan Drug Products Development, US Food & Drug Administration DISCOVERY TRACK Offers both CME and CPE Credit 8:40 – 9:10 am UAN #0238-0000-16-010-L01-P Can Universities Make a Difference in the ACPE – 3.5 CONTACT HOURS/APPLICATION-BASED Development of Orphan Drugs? James Cloyd III, PharmD, Professor, Neurology & Experimental This Symposium is supported in part by an Educational Grant from Amicus & Clinical Pharmacology, Univ of Minnesota, Coll of Pharmacy Therapeutics 9:10 – 9:35 am CO-CHAIRS: Drug Development in Rare Neurological Vijay Ivaturi, PhD, Assistant Professor, Univ of Maryland Baltimore Diseases: Clinical Pharmacology Perspective Venkatesh Atul Bhattaram, PhD, Senior Staff Fellow, Ofice of Clinical Bilal S. AbuAsal, PhD, Clinical Pharmacologist, US Food & Drug Pharmacology, US Food & Drug Administration Administration

TARGET AUDIENCE: 9:35 – 10:00 am / Break The target audience includes researchers, clinicians, entrepreneurs and academic technology transfer staff interested in orphan drug development. 10:00 – 10:30 am Bootstrapping Orphan Drug Development GOALS AND OBJECTIVES: Zachary Rome, BS, MST, Co-founder & Executive Vice Following completion of this activity, the learner will be able to: President, Patagonia Pharmaceuticals LLC 1. Describe state-of-the-art research on orphan drug development; 2. Understand the regulatory pathways available for orphan drug 10:30 – 11:00 am development; Statistical Issues In Rare Disease Clinical Trial 3. Facilitate greater research collaboration and creation of learning Design communities across disciplines, sectors and initiatives; Anindya Roy, PhD, Professor, Univ of Maryland Baltimore County 4. Discuss entrepreneurial opportunities and mechanisms to innovate and excel in orphan drug development. 11:00 am – 12:00 pm Panel Discussion 8:00 – 8:15 am Introduction Vijay Ivaturi, PhD, Assistant Professor, Univ of Maryland Baltimore and Venkatesh Atul Bhattaram, PhD, Senior Staff Fellow, Ofice of Clinical Pharmacology, US Food & Drug Administration

MED001754 Symposia

TUESDAY, SEPTEMBER 27, 2016 l Symposium 14 l 8:00 – 9:30 am

BALLROOM SALON D

Clinical Applications of 8:10 – 8:30 am Physiologically-based Overview of Development of Integrated Pediatric Physiologically-based Pharmacokinetic/ Pharmacokinetics/ Pharmacodynamic Models Pharmacodynamics for Pediatrics: Trevor N. Johnson, PhD, Principal Scientist, Simcyp Ltd 8:30 – 8:50 am Academic, Industry & Regulatory Pediatric PBPK Strategies & Tools During Drug Perspectives Development Tycho Heimbach, PhD, Director, Drug Metabolism & DISCOVERY TRACK Pharmacokinetics, Novartis Pharmaceuticals Corp

CO-CHAIRS: 8:50 – 9:10 am Jennifer Sheng, PhD, PharmD, Associate Director, Clinical Pharmacology Pediatric PBPK Modeling: Regulatory Experience & Pharmacometrics, Bristol-Myers Squibb Co & Perspective Diansong Zhou, PhD, Director, Quantitative Clinical Pharmacology, Ping Zhao, PhD, Lead, PBPK Program, Div of Pharmacometrics, AstraZeneca plc US Food & Drug Administration

TARGET AUDIENCE: 9:10 – 9:30 am The target audience includes clinical pharmacologists in the pharmaceutical industry and reviewers at the US Food & Drug Panel Discussion Administration.

GOALS AND OBJECTIVES: Following completion of this activity, the learner will be able to: 1. Describe the opportunities and challenges in pediatric physiologically- based pharmacokinetic and pharmacodynamic (PBPK/PD) modeling; 2. Illustrate how pediatric PBPK modeling can be used to answer clinical development questions with case examples.

8:00 – 8:10 am Introduction Jennifer Sheng, PhD, PharmD, Associate Director, Clinical Pharmacology & Pharmacometrics, Bristol-Myers Squibb Co

33 MED001755 Symposia

TUESDAY, SEPTEMBER 27, 2016 l Symposium 15 l 10:00 – 11:45 am

BALLROOM SALON D

Combination Therapy in Oncology: 10:10 – 10:30 am Challenges & Strategies in Clinical Clinical Perspectives in Combination Requirements & Challenges in Small- & Large- Pharmacology molecule Oncology Drug Development R. Donald Harvey, PharmD, Associate Professor & Director, APPLICATION TRACK Phase I Clinical Trials Program, Winship Cancer Inst, Emory Univ

CO-CHAIRS: 10:30 – 10:50 am Yilong Zhang, PhD, Principal Scientist, Amgen Inc Application of Physiologically-based Satyendra Suryawanshi, PhD, Associate Director, Bristol-Myers Squibb Pharmacokinetic Modeling to Facilitate Dose Co Optimization in Combination Therapy Karen Rowland Yeo, PhD, Senior Scientiic Advisor, Simcyp Ltd TARGET AUDIENCE: The target audience includes clinical pharmacologists, physicians and 10:50 – 11:10 am healthcare professionals, oncology researchers in both industry and academics and graduate students in pharmaceutical sciences and clinical Clinical Pharmacology Considerations in pharmacology. Oncology Combination Studies Sree Kasichayanula, PhD, Principal Scientist, Amgen Inc GOALS AND OBJECTIVES: Following completion of this activity, the learner will be able to: 11:10 – 11:30 am 1. Understand the combination requirements and challenges in small- and Utility of PBPK in the Oncology Drug large-molecule oncology drug development; Development Experience of Using Quantitative 2. Utilize clinical pharmacology strategies to optimize dose selection in Data Generated from Clinical Pharmacology oncology combination trials; Programs in Regulatory Decision Making & 3. Apply mechanistic models including physiologically-based Product Labels pharmacokinetics (PBPK) to address complex clinical pharmacology Ping Zhao, PhD, Lead, PBPK Program, Div of Pharmacometrics, issues; US Food & Drug Administration 4. Gain regulatory insight on current status of supporting regulatory decisions using PBPK results, including the use of PBPK data in 11:30 – 11:45 am product labels. Panel Discussion

10:00 – 10:10 am Introduction Yilong Zhang, PhD, Principal Scientist, Amgen Inc

MED001756 Symposia

TUESDAY, SEPTEMBER 27, 2016 l Symposium 16 l 1:30 – 5:30 pm

BALLROOM SALON A–C

Clinical Pharmacology Strategies 1:40 – 2:05 pm in Precision Medicine-based Drug Why Clinical Pharmacology is Positioned Well to Excel in Precision Medicine Development & Preventive Medicine Jogarao V. Gobburu, PhD, MBA, Professor, Univ of Maryland

DISCOVERY & APPLICATION TRACKS 2:05 – 2:30 pm

CO-CHAIRS: Clinical Pharmacology of Precision Medicine Treating Cancer Patients Priyanka Jadhav, PhD, Translational Clinical Pharmacologist, CRC Pharma LLC Qi Liu, PhD, Clinical Pharmacology Team Leader, US Food & Drug Administration Jinshan Shen, PhD, Senior Director, Drug Metabolism & Pharmacokinetics, Radius Health Inc 2:30 – 3:00 pm Manoj P. Jadhav, PhD, Translational Clinical Pharmacologist, CRC Pharma LLC Genetics & Precision Medicine: The Way Forward! TARGET AUDIENCE: Prasun Mishra, MSc, PhD, Scientist/Principal Investigator, The target audience includes healthcare professionals, including Genentech Inc physicians, clinical pharmacologists and basic scientists who are involved in drug development and research in industry and academics. The course 3:00 – 3:30 pm / Break is also applicable to students pursuing their MD, PhD or PharmD. 3:30 – 3:55 pm GOALS AND OBJECTIVES: Multi-domain Inference in Healthcare Following completion of this activity, the learner will be able to: Mattia Prosperi, PhD, Associate Professor, Univ of Florida 1. Understand recent advances in research and development in the area of precision medicine; 3:55 – 4:20 pm 2. Implement recent advances related to research and treatment using a personalized medicine approach, as appropriate; Clinical Pharmacology in Cardiovascular Precision Medicine 3. Understand and discuss the role of genetics, pharmacogenomics, Big Data and regulations in the implementation of this concept of precision Manoj P. Jadhav, PhD, Translational Clinical Pharmacologist, medicine. CRC Pharma LLC

4:20 – 4:45 pm 1:30 – 1:40 pm Pharmacogenomics in Drug Development of Introduction Precision Medicine – An FDA Perspective Jinshan Shen, PhD, Senior Director, Drug Metabolism & Michael Pacanowski, PharmD, MPH, Associate Director for Pharmacokinetics, Radius Health Inc Genomics & Targeted Therapy, US Food & Drug Administration

4:45 – 5:30 pm Panel Discussion

35 MED001757 Symposia

TUESDAY, SEPTEMBER 27, 2016 l Symposium 17 l 1:30 – 5:30 pm

BALLROOM SALON D

Reproducible Visualization & Data 1:45 – 2:00 pm Analysis With R Introduction to a Reproducible R Worklow with Rstudio, Rmarkdown DISCOVERY TRACK Devin Pastoor, MTOX, Software Engineer & PhD Candidate, Ctr for Translational Medicine, Schools of Pharmacy & Medicine, Offers both CME and CPE Credit Univ of Maryland Baltimore UAN #0238-0000-16-018-L-01-P ACPE – 3.5 CONTACT HOURS/APPLICATION-BASED 2:00 – 2:20 pm

CHAIR: Introduction to ggplot2 for Data Visualization Devin Pastoor, MTOX, Software Engineer & PhD Candidate, Ctr for Kaori Ito, PhD, Director, Pizer Inc Translational Medicine, Schools of Pharmacy & Medicine, Univ of Maryland Baltimore 2:20 – 2:45 pm Hands-on Activity TARGET AUDIENCE: Kaori Ito, PhD, Director, Pizer Inc The target audience includes persons that use R for data management, statistical analysis or visualization. Attendees should have basic R 2:45 – 3:00 pm exposure (read in a dataset, basic data management), with varied examples, material and solutions catered to beginner, intermediate and Solutions Demonstration advanced users. Kaori Ito, PhD, Director, Pizer Inc

GOALS AND OBJECTIVES: 3:00 – 3:30 pm / Break Following completion of this activity, the learner will be able to: 3:30 – 4:00 pm 1. Use best practices in quickly managing, analyzing and visualizing data in a reproducible fashion; Introduction to Data Management with dplyr 2. Use hands-on examples to introduce and explore a data management Devin Pastoor, MTOX, Software Engineer & PhD Candidate, Ctr pipeline that leverages best-in-class R packages for easy-to-use, for Translational Medicine, Schools of Pharmacy & Medicine, powerful worklows. Univ of Maryland Baltimore

4:00 – 4:15 pm 1:30 – 1:45 pm Additional dplyr & Introduction to tidyr Introduction Vijay Ivaturi, PhD, Assistant Professor, Univ of Maryland Devin Pastoor, MTOX, Software Engineer & PhD Candidate, Ctr Baltimore for Translational Medicine, Schools of Pharmacy & Medicine, Univ of Maryland Baltimore 4:15 – 5:00 pm Hands-on Activity Using dplyr, tidyr & ggplot2 Vijay Ivaturi, PhD, Assistant Professor, Univ of Maryland Baltimore

5:00 – 5:30 pm Wrap Up, Q&A, Additional Resources Devin Pastoor, MTOX, Software Engineer & PhD Candidate, Ctr for Translational Medicine, Schools of Pharmacy & Medicine, Univ of Maryland Baltimore

MED001758 Faculty

Last Name First Name Activity Afiliation

Associate Director for Drug Safety, Ofice of Clinical Pharmacology, US Food & Drug Abernethy Darrell R. Pre-meeting Workshop 3 Administration AbuAsal Bilal S. Symposium 13 Clinical Pharmacologist, US Food & Drug Administration Ahn Hae-Young Symposium 7 Deputy Director, Div of Clinical Pharmacology lll, US Food & Drug Administration Amur Shashi Pre-meeting Workshop 2 Scientific Lead, Biomarker Qualification Program, US Food & Drug Administration Arya Vikram Symposium 6 Silver Spring, MD Baer Gerri Symposium 9 Medical Officer/Neonatologist, US Food & Drug Administration Bajaj Gaurav Symposium 4 Senior Research Investigator, Bristol-Myers Squibb Co Barrett Jeffrey Symposium 8 Vice President, Translational Informatics, Sanofi Benjamin Jonathan Symposium 3 Medical Director, Amgen Inc

Bergman Kimberly L. Symposium 1 Lead Pharmacologist, US Food & Drug Administration Bhansali Suraj G. Symposium 10 Manager, Oncology Clinical Pharmacology, Novartis Pharmaceuticals Corp Bhattacharya Indranil Pre-meeting Workshop 1 Director, Pfizer Inc Bhattaram Venkatesh Atul Symposium 13 Senior Staff Fellow, Office of Clinical Pharmacology, US Food & Drug Administration Brar Satjit Pre-meeting Workshop 4 Associate Director, Clinical Pharmacology, Pfizer Inc Associate Director, Labeling Development Team, Office of New Drugs, US Food & Drug Brodsky Eric Symposium 12 Administration Bullock Julie Symposium 12 Senior Director, d3 Medicine LLC Pre-meeting Workshop 3 Burckart Gilbert J. Associate Director for Pediatrics, US Food & Drug Administration & Symposium 9 Campanello Leonard Symposium 5 Chief of Police, City of Gloucester, MA Police Dept MAFE Cherala Ganesh Pre-meeting Workshop 2 Research Scientist, Research Technologies, Novo Nordisk Inc Chow Andrew T. Symposium 1 Executive Director, Amgen Inc Professor, Neurology & Experimental & Clinical Pharmacology, Univ of Minnesota, Coll of Cloyd III James Symposium 13 Pharmacy Cohen Dora W. Symposium 12 Executive Director, Global Labeling, Amgen Inc Professor & Coordinator of Interprofessional Education, Univ of North Texas System Coll of Cohen Lawrence J. Symposium 11 Pharmacy Corey Alfred Symposium 1 Consultant, AC Pharmaco LLC Dallmann Gabriele Symposium 7 Co-founder, Biopharma Excellence GbR

Davis John D. Symposium 4 Senior Director, Regeneron Pharmaceuticals Inc Day Ruth S. Symposium 12 Director, Medical Cognition Laboratory, Psychology & Neuroscience, Duke Univ

37 MED001759 SymposiaFaculty

Last Name First Name Activity Afiliation

Declerck Paul Symposium 7 Professor, Dean of the Faculty of Pharmaceutical Sciences, Univ of Leuven Distinguished Professor & Chair, V. Ravi Chandran Professor in Pharmaceutical Sciences, Derendorf Hartmut Symposium 7 Univ of Florida Scientific Director, CONRAD & Professor of Obstetrics & Gynecology, Eastern Virginia Doncel Gustavo F. Pre-meeting Workshop 2 Medical School Professor & Chair of the Biotechnology & Biological Sciences Research Council, St Hilda’s Duff Sir Gordon W. Pre-meeting Workshop 1 Coll, Univ of Oxford Earp Justin C. Pre-meeting Workshop 4 Pharmacometrics Reviewer, US Food & Drug Administration

Eissing Thomas Pre-meeting Workshop 3 Head of Systems Pharmacology CV, Bayer Technology Svcs GmbH

Faggioni Raffaella Pre-meeting Workshop 1 Senior Director, Clinical Pharmacology & DMPK, MedImmune LLC/AstraZeneca plc

Florian Jeffry Symposium 8 Team Leader, Div of Pharmacometrics, US Food & Drug Administration

Fossler, Jr Michael J. Symposium 5 Vice President, Quantitative Sciences, Trevena Inc Garnett Christine Symposium 10 Clinical Analyst, US Food & Drug Administration

Gibiansky Leonid Symposium 2 President, QuantPharm LLC

Gobburu Jogarao V. Symposium 16 Professor, Univ of Maryland

Goel Varun Pre-meeting Workshop 4 Fellow, Clinical Pharmacology, Novartis Inst for Biomedical Research Professor, Internal Medicine, Univ of Central Florida, Coll of Medicine, Associate Chief of Golden Adam Pre-meeting Workshop 3 Staff, Geriatrics and Extended Care, Orlando Veterans Affairs Medical Ctr Grillo Joseph A. Symposium 12 Associate Director for Labeling & Health Communications, US Food & Drug Administration

Gunn, III George R. Symposium 2 Associate Scientific Director, Janssen Research & Development LLC

Gupta Manish Symposium 4 Director, Clinical Pharmacology & Pharmacometrics, Bristol-Myers Squibb Co Associate Professor, Dept of Pharmaceutical Sciences, East Tennessee State Univ, Gatton Harirforoosh Sam Pre-meeting Workshop 2 Coll of Pharmacy Associate Professor & Director, Phase I Clinical Trials Program, Winship Cancer Inst, Harvey R. Donald Symposium 15 Emory Univ Heimbach Tycho Symposium 14 Director, Drug Metabolism & Pharmacokinetics, Novartis Pharmaceuticals Corp

Hendriks Bart Symposium 3 Senior Director of Nanoimaging, Merrimack Pharmaceuticals Inc

Hendrix Craig W. Pre-meeting Workshop 2 Wellcome Professor & Director, Johns Hopkins Univ School of Medicine

Hu Xiao Symposium 10 Senior Pharmacometrician, Biogen Inc Issa Amalia M. Symposium 11 Professor & Chair, Personalized Medicine & Targeted Therapeutics, Univ of the Sciences

Ito Kaori Symposium 17 Director, Pizer Inc

MED001760 Faculty

Last Name First Name Activity Afiliation

Ivaturi Vijay Symposia 13 & 17 Assistant Professor, Univ of Maryland Baltimore Vice President, Chief Medical Information Officer & Chief Information Officer, Children’s Jacobs Brian Symposium 8 National Health System Jadhav Manoj P. Symposium 16 Translational Clinical Pharmacologist, CRC Pharma LLC Jadhav Priyanka Symposium 16 Translational Clinical Pharmacologist, CRC Pharma LLC Jiang Xiling Symposium 4 Senior Scientist, Janssen Research & Development LLC Medical Officer, Office of Orphan Drug Products Development, US Food & Drug Jillapalli Devanand Symposium 13 Administration Johnson Trevor N. Symposium 14 Principal Scientist, Simcyp Ltd Karlsson Mats O. Pre-meeting Workshop 4 Professor, Dept of Pharmaceutical Biosciences, Uppsala Univ Kasichayanula Sree Symposia 3 & 15 Principal Scientist, Amgen Inc Kottilil Shyam Symposium 6 Professor of Medicine, Inst of Human Virology, Univ of Maryland Kraft Walter K. Symposium 11 Professor, Thomas Jefferson Univ Kumar Parag Pre-meeting Workshop 2 Director, Clinical Pharmacokinetics Research Lab, National Inst of Health Lau S.W. Johnny Pre-meeting Workshop 3 Senior Clinical Pharmacologist, US Food & Drug Administration

Lee Jinhee Symposium 5 Senior Pharmacy Advisor, Substance Abuse & Mental Health Svcs Administration Director, Div of Clinical Pharmacology, Toxicology & Therapeutic Innovation, Children’s Leeder J. Steven Symposium 9 Mercy Hosp Clinical Professor & Director, Ctr for Pharmacometrics & Systems Pharmacology, Univ of Lesko Lawrence J. Symposium 10 Florida Pre-meeting Workshop 1 Li Chunze Senior Scientist, Genentech Inc & Symposium 4 Liu Jiang Symposium 10 Scientific Lead for QT, US Food & Drug Administration Liu Qi Symposium 16 Clinical Pharmacology Team Leader, US Food & Drug Administration Ma Lian Symposium 1 Pharmacometrics Reviewer; US Food & Drug Administration Mager Donald E. Symposium 3 Associate Professor, Univ at Buffalo, State Univ of New York Marathe Anshu Pre-meeting Workshop 4 Team Leader, Div of Clinical Pharmacology II, US Food & Drug Administration Mayer Christina L. Symposium 3 Senior Scientist, Biologics Clinical Pharmacology, Janssen Research & Development LLC

McCune Susan Symposium 9 Deputy Director, Ofice of Translational Sciences, US Food & Drug Administration Mehrotra Nitin Symposium 1 Team Leader, Div of Pharmacometrics, US Food & Drug Administration Meibohm Bernd Pre-meeting Workshop 1 Professor & Associate Dean, Univ of Tennessee Health Science Ctr, Coll of Pharmacy

39 MED001761 SymposiaFaculty

Last Name First Name Activity Afiliation

Mishra Prasun Symposium 16 Scientist/Principal Investigator, Genentech Inc

Moorman Jonathan P. Pre-meeting Workshop 2 Professor, East Tennessee State Univ, Quillen Coll of Medicine Mould Diane R. Symposia 4 & 8 President, Projections Research Inc

Mulugeta Lily (Yeruk) Symposium 8 Scientific Lead for Pediatrics, Div of Pharmacometrics, US Food & Drug Administration

Nelson Cara Symposium 10 Clinical Pharmacologist II, Gilead Sciences Inc

Pacanowski Michael Symposium 16 Associate Director for Genomics & Targeted Therapy, US Food & Drug Administration

Passey Chaitali Symposium 2 Senior Research Investigator, Bristol-Myers Squibb Co Software Engineer & PhD Candidate, Ctr for Translational Medicine, Schools of Pharmacy Pastoor Devin Symposium 17 & Medicine, Univ of Maryland Baltimore Portman Ronald J. Symposium 9 Executive Director, Pediatric Therapeutic Area, Novartis Pharmaceuticals Corp Pharmacologist, Counter-terrorism & Emergency Coordination, US Food & Drug Powell Andrea M. Symposium 1 Administration Prosperi Mattia Symposium 16 Associate Professor, Univ of Florida

Rahman Atiqur Pre-meeting Workshop 4 Director, Div of Clinical Pharmacology V, US Food & Drug Administration

Rawal Sumit Symposium 2 Scientist, Regeneron Pharmaceuticals Inc

Rome Zachary Symposium 13 Co-founder & Executive Vice President, Patagonia Pharmaceuticals LLC

Rosenberg Amy Symposium 2 Supervisory Medical Officer, Div of Therapeutic Proteins, US Food & Drug Administration Rowland Yeo Karen Symposium 15 Senior Scientific Advisor, Simcyp Ltd

Roy Anindya Symposium 13 Professor, Univ of Maryland Baltimore County

Rusch Lorraine M. Symposium 5 Vice President, Commercial Development, Celerion Inc

Schellekens Huub Symposium 7 Chair, Professor in Pharmaceutical Biotechnology, Utrecht Univ

Schlender Jan-Frederik Pre-meeting Workshop 3 Pharmacist, Scientist Systems Pharmacology, Bayer Technology Svcs GmbH

Sellers Edward M. Symposium 5 Professor Emeritus, Pharmaceuticals, Medicine & Psychiatry, Univ of Toronto

Seo Shirley Symposium 6 Team Leader, Ofice of Clinical Pharmacology, US Food & Drug Administration Shen Jinshan Symposium 16 Senior Director, Drug Metabolism & Pharmacokinetics, Radius Health Inc

Sheng Jennifer Symposium 14 Associate Director, Clinical Pharmacology & Pharmacometrics, Bristol-Myers Squibb Co

MED001762 Faculty

Last Name First Name Activity Afiliation

Shi Rong Pre-meeting Workshop 1 Clinical Pharmacology Lead, Bristol-Myers Squibb Co Sinha Vikram Symposium 8 Associate Vice President, Head Quantitative Pharmacology, Merck Research Laboratories Pre-meeting Workshop 4 Director, Clinical Pharmacology, Translational Clinical Oncology, Novartis Pharmaceuticals Skordos Konstantine W. & Symposium 10 Corp Pre-meeting Workshop 3 Slattum Patricia W. Professor of Pharmacotherapy & Outcomes Science, Virginia Commonwealth Univ & Symposium 12 Smith P. Brian Symposia 8 & 9 Professor of Pediatrics, Duke Univ Medical Ctr Sourgens Hildegard Symposium 7 President Elect, European Federation for Exploratory Medicines Development Stegemann Sven Pre-meeting Workshop 3 Professor, Graz Univ of Technology Sulkowski Mark Symposium 6 Professor of Medicine, Johns Hopkins Univ School of Medicine Sun Yu-Nien (Tom) Symposium 3 Senior Director, Janssen Research & Development LLC Suryawanshi Satyendra Symposium 15 Associate Director, Bristol-Myers Squibb Co van den Anker John Symposium 9 Chief, Div of Clinical Pharmacology, Children’s National Health System

Wang Jian Symposium 9 Senior Clinical Pharmacologist, US Food & Drug Administration Wang Yow-Ming C. Pre-meeting Workshop 1 Clinical Pharmacology (Biologics) Team Leader, US Food & Drug Administration Yang Zheng Pre-meeting Workshop 1 Director, Bristol-Myers Squibb Co Yao Lynne Symposia 8 & 9 Director, Div of Pediatric & Maternal Health, US Food & Drug Administration Zajicek Anne Symposium 8 Branch Chief, National Inst of Health Zhang Yilong Symposium 15 Principal Scientist, Amgen Inc Zhao Hong Symposium 4 Master Reviewer of Clinical Pharmacology/Team Leader, US Food & Drug Administration Zhao Ping Symposia 4, 14 & 15 Lead, PBPK Program, Div of Pharmacometrics, US Food & Drug Administration Zhou Diansong Symposium 14 Director, Quantitative Clinical Pharmacology, AstraZeneca plc Zhou Honghui Pre-meeting Workshop 1 Senior Director & Janssen Fellow, Janssen Research & Development LLC

41 MED001763 Why Join ACCP?

The American College of Clinical Pharmacology (ACCP) is a non-proit membership association with a 45+ year history of providing exceptional interprofessional, accredited Continuing Education programs, publications, networking and other career-enhancing opportunities to a wide spectrum of healthcare professionals using clinical pharmacology in disciplines from research to patient care. Membership includes MDs, PharmDs, PhDs, post- doctoral candidates, students and others from academia, industry, regulatory and clinical entities who are seeking to advance their career through the Member Beneits offered by ACCP.

Why Should You Join the American College of Clinical • Opportunity to develop educational activities that make a Pharmacology? difference by submitting proposals for ACCP educational events and getting involved in the clinical pharmacology community. Your membership in ACCP now gets you more and is your way to stay at the top of your professional game! • Access to the ACCP Job Center to view jobs and post your resume. • Conidently achieve a high level of professional performance • Receipt of information from the clinical pharmacology by staying on the cutting edge of clinical pharmacology community for Members who opt in to receive the daily news developments; format, routine recall/drug safety notices from FDA Medwatch, • Build professional relationships that last a lifetime; FDA Bursts or AAMC notiications. • Be part of a vibrant professional community with similar • Receipt of routine updates from ACCP about developments in goals and objectives; the ield of clinical pharmacology and future ACCP events. • Shape the future of clinical pharmacology. ACCP membership runs on a calendar year, January to December. Dues renewal notiications are sent in September for the coming year. Persons ACCP Member Beneits get you there! joining between May 1st and July 31st pay a reduced half-year fee for the current • Free access to the latest scientiic research. Members have calendar year. Please note that the half-year option is only available the irst year free online access to ACCP’s high-quality publications, The of ACCP membership. All future payments must be full-year payments. Persons Journal of Clinical Pharmacology, published for over 50 years, joining for the irst time as of August 1st pay for the coming full calendar year and Clinical Pharmacology in Drug Development, introduced in dues and receive August – December of the current year at no cost. 2012. eTOC notiications are sent for both journals, and the JCP eTOC highlights journal articles eligible for Continuing Education BEFORE YOU APPLY FOR MEMBERSHIP, PLEASE NOTE IF ANY OF THE credit and Editor’s Choice articles. Archives of The Journal of FOLLOWING PERTAIN TO YOU AND CONTACT [email protected] FOR Clinical Pharmacology dating back to 1961 and Clinical EXISTING LOGIN CREDENTIALS: Pharmacology in Drug Development since 2012 are available • Been a Member of ACCP in the past; to Members. • Have attended an ACCP Annual Meeting; • Free CME and CPE credits on selected articles in The Journal • Presented a poster at an ACCP Annual Meeting; of Clinical Pharmacology. • Participated as Faculty at an ACCP Annual Meeting. • Free online educational activities Our program of online How to Join ACCP educational events provides you with 24/7 access and includes ACCP has several categories of membership, please join using the membership the ACCP Fundamentals Tutorials series, the ACCP Virtual Journal category that is most appropriate for you. Club and the ACCP Therapeutic Dilemmas series (New in 2016!), all available live, then on demand. Please note: A membership application is not considered complete until all required documents have been submitted and acknowledged by the ACCP • Discounted registration for the ACCP Annual Meeting, your Executive Ofice and dues have been paid. All applications must be submitted in source for current, interprofessional ACCME & ACPE-accredited full 30 days before the Board of Regents Meetings, the dates of which are noted Continuing Education programs in a live format. below: • Free access to Annual Meeting recorded events for Annual • February 12, 2017 Meeting attendees and discounted access for other Members. • May 7, 2017 • Networking opportunities and, for Students & Trainees, access • September 16, 2017 to Mentors. Persons interested in becoming a Fellow should join as a Member and • Opportunity to enhance your leadership skills by volunteering notify [email protected] about their interest in becoming a Fellow. for one of ACCP’s many committees or by Mentoring Students & Trainees.

MED001764 Students & Trainees

Annual Meeting Events for Students & Trainees Special Access to the Experts th Student & Trainee membership and participation in ACCP’s Annual On Tuesday, September 27 , from 7:00 – 8:00 am in Ballroom Salon Meeting are strongly encouraged and are beneicial on several E – H, schools represented by groups of six or more Students & Trainees levels: will be provided with a higher level of access to ACCP leadership. Select • Mentoring and expert guidance ACCP leaders will have a sit-down roundtable session with those Students & Trainees to discuss opportunities for further involvement in ACCP during • Student & Trainee-speciic events at the Annual Meeting their training and how to subsequently grow in the organization throughout • Substantially discounted registration fees for educational their careers. programs • ACCP Student Abstract Awards Program CV Reviews! All Students & Trainees were encouraged to provide their CV for review Student & Trainee-speciic Events and suggestions by ACCP Mentors. If you submitted a resume and did Panel Discussion, Podium Presentations, Student Networking not make arrangements in advance, but wish to meet with the Mentor who Reception and Poster Tours reviewed your CV, please stop by the ACCP Registration Desk by the end On Sunday, September 25th, the following events will be hosted: of the day on Sunday, September 25th, to set up an appointment. • Panel Discussion on Career Guidance (2:00 – 3:30 pm, White Flint Amphitheater) – A select group of ACCP Mentors whose careers Join, Get Involved and Enjoy the Beneits of ACCP have spanned various settings and disciplines within the ield of Membership! clinical pharmacology will share their experiences and answer your Visit us at questions in a relaxed, intimate atmosphere. If you are considering a career that includes any combination of academia, industry, The Student, Trainee & Young Professional Committee, co-chaired regulatory or clinical roles, don’t miss this opportunity to hear what by Daniel Gonzalez, PharmD, PhD and Amelia N. Deitchman, PharmD, the experts have to say about how their own career paths is critical in providing guidance regarding Student, Trainee & Young progressed and what guidance they can provide to ensure your Professional needs and ensuring that those needs are consistently met by personal success! ACCP. The committee is comprised of Student Members, Members and • Podium Presentations (3:30 – 4:30 pm, White Flint Amphitheater) Fellows and it focuses on activities at the Annual Meeting and provides – Immediately following the Panel Discussion, a select number of guidance on programs, new and old, required to effectively support Student Abstract Award winners will present their research in a Students, Trainees & Young Professionals. Have a great idea? Please Podium Presentation to an audience of Annual Meeting attendees. share it with us at [email protected]. Support your colleagues by being part of this important event. • Student Networking Reception (4:30 – 5:30 pm, Brookside Foyer) – After the Podium Presentations, join us for the Student Networking Reception where you can interact on a more personal level with Panel Discussion speakers and other ACCP Mentors to ask the burning questions that will help you make decisions about your future. • Poster Tours (Meet at ACCP Registration Desk at 5:30 pm for a tour from 5:45 – 6:30 pm) – Small groups of Students & Trainees will be hosted by an ACCP Fellow or senior Member to tour the poster area and discuss preselected posters that provide exceptional educational content or presentation.

Amelia N. Deitchman, PharmD Daniel Gonzalez, PharmD, PhD

43 MED001765 Sponsors

ACCP gratefully acknowledges Sponsors of the 2016 Annual Meeting:

GOLD LEVEL SPONSORS

SILVER LEVEL SPONSOR

BRONZE LEVEL SPONSORS

MED001766 Exhibitors

ACCP gratefully acknowledges Exhibitors of the 2016 Annual Meeting:

Altasciences Clinical Research encompasses Algorithme Pharma in Montreal, QC and Vince & Associates Clinical Research in Overland Park, KS, as well as Algorithme Pharma USA in Fargo, ND, thereby making it one of the largest early-phase clinical CROs in North America. With over 25 years of industry experience, Altasciences provides early-phase clinical development services to an international customer base of biopharmaceutical and generic companies. Altasciences’ full-service solutions offering in this critical stage of drug development includes medical writing, biostatistics, data management and bioanalysis. www.altasciences.com

Celerion leverages over 40 years of experience, 600 beds and locations in North America, Europe and Asia to conduct and analyze First-in-Human, clinical Proof-of-Concept and dose response in patients, cardiovascular safety assessments, ADME and NDA-enabling clinical pharmacology studies. Celerion provides expertise on clinical data analysis, as well as small and large molecule bioanalytical assay services. www.celerion.com

Clinical Pharmacology of Miami Inc is a private pharmaceutical research organization dedicated to the conduct of clinical trials (Phase I-IV) in the South Florida area. Kenneth C. Lasseter, MD, Stacy C. Dilzer, RN, BSN and E. Cooper Shamblen are the principals who make up our experienced management team. We have the experience, expertise and facility to conduct safe, well-controlled clinical research with new and existing drugs. Our research facility is state-of-the-art and fully equipped with 120 beds. Our local subject population includes healthy males and females, Hepatically impaired, Renal insuficiency, Hypertensive, Geriatric, Diabetic and Obese volunteers. www.clinpharmmiami.com

Covance – As one of the world’s largest and most comprehensive drug development service companies, we have helped pharmaceutical and biotech companies develop one-third of all prescription drugs in the marketplace today. Because of our broad experience and specialized expertise, we’re in a unique position to supply insights that go above and beyond testing. Together with our clients, we create solutions that transform potential into reality. www.covance.com

45 MED001767 Exhibitors

ERT is a leading provider of high-quality patient safety & eficacy endpoint data collection solutions for use in clinical trial development. ERT delivers a combination of technology, services and consulting that increase the accuracy and reliability of the patient data and improve the eficiency of the clinical development process throughout the product lifecycle. www.ert.com

The Medpace Clinical Pharmacology Unit (MCPU) is an early-phase clinical pharmacology unit, conducting studies in normal healthy volunteers, special populations and patient populations over a spectrum of diseases. Medpace CPU is owned by Medpace Inc. MCPU features a 96-bed, state- of-the-art facility housed on the Medpace clinical research campus, which is centrally located in Cincinnati, Ohio. www.medpace.com

For 125 years, Merck has been a global healthcare leader working to help the world be well. Merck is known as MSD outside the United States and Canada. Through our prescription medicines, vaccines, biologic therapies and animal health products, we work with customers and operate in more than 140 countries to deliver innovative health solutions. We also demonstrate our commitment to increasing access to healthcare through far-reaching policies, programs and partnerships. For more information, visit www.merck.com and connect with us on Twitter, Facebook, YouTube and LinkedIn. www.merck.com

MED001768 Exhibitors

NOCCR and VRG are privately-owned, multispecialty clinical research groups which have conducted over 2,000 clinical trials in the last 30 years. With combined space exceeding 24,500 sq ft, full-time MDs, Nurse Practitioners, Nurse/Coordinators, EMTs, nursing assistants and separate regulatory, data and recruiting departments, we have earned a reputation for excellence and consistently exceeding enrollment goals. NOCCR-Knoxville is primarily a Phase I Unit with up to 50 beds. It is particularly well suited for conducting First-in-Human trials as it is situated within the Univ of Tennessee Medical Ctr, with a code team and 24-hour critical care coverage. This Unit is widely known for its ability to conduct procedurally-dificult trials and to recruit special populations, including volunteers with renal and hepatic insuficiency, elderly, postmenopausal, heart failure, hypertension and normal healthy volunteers. VRG and NOCCR-New Orleans are primarily focused on conducting later phase studies in a broad array of therapeutic areas. www.noccr.com

PRA Health Sciences’ early-phase professionals live and breathe clinical pharmacology. As the most comprehensive high-end Phase I CRO in the world, PRA Early Development Svcs provide a unique scientiic environment required for complex compound development in both healthy volunteers and special patient populations. Committed to the highest standards of clinical excellence and scientiic expertise, we operate state-of-the-art facilities in The Netherlands and North America, as well as an innovative patient pharmacology model in Central and Eastern Europe. Our fully-harmonized, GLP-compliant laboratories are located close to our clinical units, enabling us to quickly analyze time- critical samples. www.prahs.com

Richmond Pharmacology – Under the same senior management for over a decade, Richmond Pharmacology offers the continuity of a highly-specialized medical team that has worked closely with its UK NHS partners to ensure a safe environment for our patients and volunteers. Our wide-ranging experience is complemented by our specialist focus on Adaptive Phase 1, TQT, Japanese and Patient Studies. www.richmondpharmacology.com

47 MED001769 Exhibitors

Simulations Plus – Cognigen and Simulations Plus provide modeling & simulation software and consulting services from discovery through clinical development. Our GastroPlus™ platform is the leading PBPK modeling solution for prediction of absorption/DDI/population outcomes in humans and animals. This is complemented by our pharmacometric modeling & simulation services and clinical pharmacology support. www.cognigencorp.com

TNO has over 3,000 professionals who put their knowledge and experience to work in creating smart solutions for complex issues. These innovations help to sustainably strengthen industrial competitiveness and social wellbeing. On the topic of Healthy Living, we initiate technological and societal innovation for healthy living and a dynamic society. We are partnered by some 3,000 companies and organizations, including SMEs, in The Netherlands and around the world. www.tno.nl/en/

Worldwide Clinical Trials provides full-service drug development services to the pharmaceutical industry. We can support you in your pre-registration through peri-approval studies, but also specialize in bioanalytical and early- phase development. This powerful combination of services ensures that you are working with a global CRO that can understand and meet the needs of your entire drug development program. www.wwctrials.com

MED001770 Exhibitors

IGNITING INDUSTRY CHANGE. It takes bold moves, unique perspectives and unmatched scientiic expertise to reshape an industry. Learn more about how Covance is delivering clinical pharmacology solutions to ignite industry change.

You have a number of ways to connect with us at ACCP: ▶ Bullet style ▶ Visit Booth #303 ▶ Attend Poster Session 2, Posters 64 and 94, featuring: Angela Hassman Clinical Study Operations

TO LEARN MORE CALL The Americas +1.888.COVANCE | Europe/Africa +00.800.2682.2682 Asia Paciﬁc +800.6568.3000 | Or go to covance.com

Covance Inc., headquartered in Princeton, NJ, USA is the drug development business of Laboratory Corporation of America Holdings (LabCorp). COVANCE is a registered trademark and the marketing name for Covance Inc. and its subsidiaries around the world. © Copyright 2016. Covance Inc.

Two peer-reviewed periodicals Visit www.thieme-connect.com/products for complete online access

Pharmacopsychiatry Drug Research

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49 MED001771 Poster Session 1

Sunday, September 25, 2016 / 5:30 – 7:30 pm, Ballroom Salon E – H Absorption, Distribution, Metabolism & Elimination

Poster Type Title Authors

S, SA, Impact of Coadministered Minocycline on Plasma Pharmacokinetics and Mahua Sarkar, Raymond Grill, Robert Grossman, 001 P Central Nervous System Distribution of Riluzole Diana Chow John P. Sabo, Fabian Mueller, Arvid Jungnik, Effect of BI 187004, a Selective 11β-HSD1 Inhibitor, on Cytochrome P450 002 Habib Esmaeili, Ralf Thiedmann, Jing Wu, and P-glycoprotein Substrates in Healthy Subjects Lois Rowland, Susanna Freude, Laurent Vernillet Biliary Excretions of CZ48 and Its Active Moiety, Camptothecin, After 003 S, NM Yu Jin Kim, Yifan Tu, Daping Zhang, Diana Chow Intravenous Administration of CZ48 in Rats Absolute Oral Bioavailability of Selexipag, a Novel Oral Prostacyclin IP Noémie Hurst, Priska Kaufmann, Muriel Richard, 004 Receptor Agonist, in Healthy Subjects Béatrice Astruc, Jasper Dingemanse A Change in Posture Significantly Affects Plasma Concentrations of Mattheus (Thijs) P. van Iersel, Maria Velinova, 005 Immunoglobulin G, Such as Monoclonal Antibodies Ruud Lutgerink Does Gastric Bypass Surgery Affect Bioavailability of Orally-administered Jihye Ahn, Bilal AbuAsal, Neha Pandit, 008 S, NM Darunavir? A Physiologically-based Pharmacokinetic Modeling Approach Mathangi Gopalakrishnan

Clinical Pharmacokinetics & Pharmacodynamics (cont on next page) Poster Type Title Authors

Stabilization of Pitavastatin and Its Lactone Metabolite in Clinical Kristina M. Brooks, Raul Alfaro, David Ng, Jomy 009 S, NM Pharmacokinetic Study Samples George, Tara Kuhn, Leslie G. Biesecker, Parag Kumar Miriam D. Carrasco-Portugal, Maria G. Lozoya-Moreno, Population Pharmacokinetics of Lopinavir/Ritonavir in Mexican Patients 010 Gustavo Reyes-Terán, Lina M. Barranco-Garduño, Infected with HIV Francisco J. Flores-Murrieta Pharmacokinetics and Safety of NSI-189 in Healthy Subjects: First-in-Human Ronald Christopher, Lev Gertsik, Molly Sherman, 011 Single, Ascending Dose and Food Effect Study Karl Johe, Larry Ereshefsky The Relative Bioavailability of Rilpivirine Following Administration of the Joseph M. Custodio, Steve West, Heena Patel, New Tenofovir Alafenamide Single-tablet Regimen Rilpivirine/Emtricitabine/ 012 NM Kah Hiing J. Ling, Huyen Cao, Erin Quirk, Tenofovir Alafenamide vs Rilpivirine/Emtricitabine/Tenofovir Disoproxil Brian P. Kearney Fumarate

LEGEND: E = Encore Presentation NM = New Member (Dues paid by April, 2016) Please visit www.ACCP1.org for updates on the Abstract P = Podium Presentation S = Student Abstract Submission process and deadlines for the 2017 Annual Meeting SA = Student Award Winner

Vikram Arya, PhD • Gaurav Bajaj, PhD • Nageshwar R. Budha, PhD • Steven J. Crosby, MA, BSP, RPh 2016 Amelia N. Deitchman, PharmD • Vineet Goti, BPharm • Manish Gupta, PhD • Elisha R. Injeti, PhD, MPharm Abstract Amalia M. Issa, PhD, MPH • Otito Frances Iwuchukwu, RPh, PhD • Nitin Mehrotra, PhD Reviewers Robert J. Noveck, MD, PhD, CPI • Charles Oo, PharmD, PhD • Stephan Schmidt, PhD Catherine M.T. Sherwin, PhD, MS • Honghui Zhou, PhD

MED001772 Poster Session 1

Sunday, September 25, 2016 / 5:30 – 7:30 pm, Ballroom Salon E – H

Clinical Pharmacokinetics & Pharmacodynamics (cont on next page) Poster Type Title Authors

Potential Implications of Atypical, Nonlinear Plasma Protein Binding on Amelia N. Deitchman, Ravi S. Singh, Johannes Kast, 013 S, SA Tigecycline Clinical Pharmacokinetics Uwe Liebchen, Hartmut Derendorf S, SA, Impact of Gastric Bypass Surgery on the Pharmacokinetics and Asma El-Zailik, Lily K. Cheung, Vadim Sherman, 014 P Pharmacodynamics of Simvastatin, Atorvastatin and Rosuvastatin Diana Chow Pharmacokinetics of Oral Tizanidine, a CYP1A2 Substrate, in Mexicans: Francisco J. Flores-Murrieta, Miriam D. Carrasco- 015 Evidence for Interethnic Variability Portugal Population Pharmacokinetic Analysis of Mycophenolate Mofetil in Rodrigo Gonzalez, Alan Quiroz-Moguel, Pilar Garcia, 016 S Pre-transplant and Post-transplant Pediatric Patients Mara Medeiros, Gilberto Castaneda, Hartmut Derendorf Effect of Intrinsic Factors on the Exposure of Faldaprevir in HCV-infected Fenglei Huang, Sebastian Haertter, 017 Patients: Pooled Analysis of Data from Three Faldaprevir Phase 3 Studies Anne-Marie Quinson Assessment of the Effect of Faldaprevir on the QT Interval in Healthy John P. Sabo, Michael Koenen-Bergmann, Anna Unseld, 018 Subjects Armin Schultz, Fenglei Huang, Anne-Marie Quinson Klaus Peter Kammerer, Peter Ruus, Simone Graeber, Effect of Food on the Pharmacokinetics of a 150 mg Oral Dose of BI 019 David Joseph, Verena Endriss, Alison Mackie, 1060469, a Novel Oral CRTH2 Antagonist, in Healthy Male Volunteers Chester Wood The Pharmacokinetics, Pharmacodynamics, Safety, Tolerability and Food Jahnavi Kharidia, Mattheus (Thijs) P. van Iersel, Jan 020 Effect of Duvelisib, an Oral, Dual PI3K-δ and PI3K-γ Inhibitor, in Healthy Hartstra, Kerstin Allen, Charlotte McKee, Joi Dunbar Human Subjects Safety and Pharmacokinetics of BI 1060469, a Novel Oral CRTH2 Peter Ruus, Irene Vroegrijk, David Joseph, 021 Antagonist, in Fed Healthy Females Verena Endriss, Alison Mackie, Chester Wood Pooja Manchandani, Yanina Dubrovskaya, Song Gao, 022 S, NM Comparative Pharmacokinetic Proiling of Various Polymyxin B Components Vincent H. Tam Pharmacokinetics and Safety of TAK-648 Following Single and Repeat Michael D. Mayer, Juliana Oliveira, Stephanie Moran, 023 Dosing in Healthy and Type 2 Diabetes Mellitus Subjects Sai Nudurupati, Rui Sun Shailly Mehrotra, Mathangi Gopalakrishnan, Population Pharmacokinetics of Veliparib With and Without Topotecan Plus 024 S, NM Jogarao V. Gobburu, Jacqueline M. Greer, Ivana Gojo, Carboplatin in Patients With Hematological Malignancies Judy Karp, Keith Pratz, Michelle A. Rudek Bonnie Shaddinger, Malcolm Young, Julia Billiard, Effect of Albiglutide on Cholecystokinin-induced Gallbladder Emptying in 026 David A. Collins, Nandana Prabhu, Azra Hussaini, Healthy Subjects Antonio Nino Jie Shao, Mong-jen Chen, Philip J. Kuehl, 027 NM Pharmacokinetic and Pharmacodynamic Modeling of Peptide YY in Mice 3-36 David Vodak, Guenther Hochhaus Bioequivalence Evaluation of Three Olanzapine-containing Tablet Lei Sun, David McDonnell, Wenlei Liu, Denise Carter, 028 Formulations in Healthy Volunteers Adam Simmons, Lisa L. von Moltke Plasma Pharmacokinetic Proile of Trabedersen – TGF-β2-speciic 029 Wen Wang, Larn Hwang Antisense Oligonucleotide in Cancer Patients Safety and Pharmacokinetics of Multiple Rising, Oral Doses of BI 1060469, Peter Ruus, Irene Vroegrijk, David Joseph, 030 a Novel CRTH2 Antagonist, in Fasted Healthy Males and Males with Mild Verena Endriss, Alison Mackie, Chester Wood Asthma

LEGEND: E = Encore Presentation NM = New Member (Dues paid by April, 2016) P = Podium Presentation S = Student Abstract SA = Student Award Winner 51 MED001773 Poster Session 1

Sunday, September 25, 2016 / 5:30 – 7:30 pm, Ballroom Salon E – H

Clinical Pharmacokinetics & Pharmacodynamics Poster Type Title Authors

Jing Wu, Valerie Nock, Arvid Jungnik, Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Single Corinna Schoelch, Michael Wolff, Susanna Freude, and Multiple, Oral Doses of BI 187004, a Selective 11β-hydroxysteroid 031 Cornelia Schepers, John Yu, Tim Heise, Dehydrogenase1 Inhibitor, in Healthy Subjects and Patients With Type 2 Leona Plum-Moerschel, Fenglei Huang, Diabetes Mellitus Laurent Vernillent Population Pharmacokinetics of Canakinumab in Children Younger Than Lucy Xu, Bruno Bieth, Martin Fink, James Kalabus, 032 Two Years Old With Cryopyrin-associated Periodic Syndrome Haiying Sun

Clinical Pharmacology Education

Poster Type Title Authors

033 Some Thoughts About the Mean Concentration vs Time Plot Michael J. Fossler, Jr

Clinical Trials & Human Pharmacology Poster Type Title Authors

Fausto Zaruma-Torres, Ismael Lares-Asseff, FPGS rs1544105 and rs4451422 are Involved With High Levels of Juan L. Chávez-Pacheco, Aarón Reyes-Espinoza, 034 S Plasmatic Concentration of Methotrexate in Mexican Children With Acute Ossyneidee Gutiérrez-Alvarez, Verónica Loera- Lymphoblastic Leukemia Castañeda, María C. Arias-Peláez Cardiac Safety and Pharmacokinetics of Pacritinib: A Phase 1, Randomized, Suliman Al-Fayoumi, Sherri Amberg, Huafeng Zhou, 035 Active- and Placebo-controlled, Three-way Crossover Study Lindsey Millard, Jack W. Singer, James P. Dean Pharmacokinetics, Pharmacodynamics and Tolerability of Multiple-dose 036 Administration of Cenerimod, a Selective Sphingosine-1-phosphate Subtype Pierre-Eric Juif, Jasper Dingemanse 1 Receptor Modulator Pharmacokinetics, Pharmacodynamics, Tolerability and Food Effect of 037 Single-dose Administration of Cenerimod, a Selective Sphingosine-1- Pierre-Eric Juif, Jasper Dingemanse phosphate Subtype 1 Receptor Modulator, in the First-in-Human Study Profiling Adverse Events and Laboratory Measurements in Healthy Hong Li, Jack Clifton II, Mukul Minocha, David Carter, 038 Volunteers Who Received Placebo in AbbVie Phase 1 Trials Ahmed A. Othman, Yi-Lin Chiu Lihua Wu, Jian Liu, Jianzhong Shentu, You Zhai, A Phase 1 Dose-escalation Study of the Safety and Pharmacokinetics of 039 Guolan Wu, Xingjiang Hu, Yunliang Zheng, Meihua Lin, Felbinac Trometamol in Healthy Chinese Volunteers Duo Lv, Juan Xu, Huili Zhou, Meixiang Zhu, Minglan Wu

LEGEND: E = Encore Presentation NM = New Member (Dues paid by April, 2016) P = Podium Presentation S = Student Abstract SA = Student Award Winner

MED001774 Poster Session 1

Sunday, September 25, 2016 / 5:30 – 7:30 pm, Ballroom Salon E – H Oncology

Poster Type Title Authors

Development and Evaluation of Tri-functional Lipid Nanoparticles for 040 S, SA Tanaya R. Vaidya, Sihem Bihorel Treatment of HER2-positive Breast Cancer Refractory to HER2 Therapy Kristina M. Brooks, Paul Jarosinski, Elizabeth Kang, S, SA, Failure of Intravenous Busulfan Test Dose Pharmacokinetics to Predict 041 Jomy George, Nirali N. Shah, John B. Le Gall, NM, P Once-daily Dosing in Pediatric Transplant Recipients Suk See De Ravin, Thomas Hughes, Parag Kumar Echocardiographic Evaluation of Cardiotoxic Drugs: Is Ejection Fraction Gerald H. Sokol, Loretta S. Loftus, Jorge Ayub, 042 Adequate? Louis Cantilena Preclinical Evaluations of Two Vascular Endothelial Growth Factor Inhibitors Maher Chaar, Ferraro Mattew, Erica Johnson, 043 S in Combination With Rapamycin for the Treatment of Hepatocellular Maxime Le Merdy, Ashley Brown, Sihem Bihorel Carcinoma Model-based Evaluations of the Exposure, Efficacy and Safety of a Xiaochen Zhao, Satyendra Suryawanshi, Yan Feng, 044 NM Nivolumab Flat-dosing Regimen in Patients With Melanoma or Non-small Xiaoning Wang, Brent McHenry, Ian M. Waxman, Anand Cell Lung Cancer Achanta, Akintunde Bello, Amit Roy, Shruti Agrawal

Translational Medicine, Including Biomarkers and/or Imaging Poster Type Title Authors

Tal Burt, David MacLeod, Kihak Lee, Thomas Hawk, Intra-target Microdosing, A Novel Drug Development Approach: Proof-of- Timothy Turkington, Antoinette Santoro, 045 NM Concept in Humans Daniel K. DeMasi, Mark Feinglos, Robert Noveck, Malcolm Rowland

Chronic Pain Management

Poster Type Title Authors

Randomized, Double-blind, Placebo- and Comparator-controlled Human 057 Abuse Liability Study of an Experimental, Triple Monoamine Reuptake Lynn R. Webster, Michael Smith Inhibitor in Recreational Drug Abusers

LEGEND: E = Encore Presentation NM = New Member (Dues paid by April, 2016) P = Podium Presentation S = Student Abstract SA = Student Award Winner

53 MED001775 Poster Session 2

Monday, September 26, 2016 / 5:30 – 7:30 pm, Ballroom Salon E – H Applications of Modeling & Simulation

Poster Type Title Authors

Application of a Physiologically-based Pharmacokinetic Model for the Rui Chen, Amin Rostami-Hodjegan, Haotian Wang, 046 Evaluation of Single-point Plasma Phenotyping Method of CYP2D6 David Berk, Jun Shi, Pei Hu Comparative Application of Published Top-down and Bottom-up 047 Pharmacokinetic Models of Efavirenz With Respect to Observed Ethiopian Abiy Habtewold, Andrew Castleman, Joel S. Owen Clinical Data Development of a Physiologically-based Pharmacokinetic Model for Viera Lukacova, Petra Goelzer, Micaela Reddy, 048 Description of Valganciclovir and Ganciclovir Pharmacokinetics Gerard Greig, Bruno Reigner, Neil Parrott Simulating Altered Omeprazole Kinetics in Elderly Individuals Using Jan-Frederik Schlender, Niels Lautenbach, 050 Physiologically-based Pharmacokinetic Population Modeling Tobias Kanacher, Michael Block, Thomas Eissing Physiologically-based Pharmacokinetic Modeling to Predict the Human 051 Pharmacokinetics of Olanzapine and Samidorphan When Administered in Lei Sun, Karen Rowland Yeo Combination as ALKS 3831 Nonlinear Mixed-effects Pharmacokinetic-Pharmacogenetic Model of William R. Wolowich, Leah Bensimon, Robert Greif, 052 Intravenously-administered Delta-9-tetrahydrocannibinol in Healthy Maren Kleine-Bruggeney, Hans Sachs, Volunteers Werner Bernhard, Lorenz Theiler Population Pharmacokinetics of Unbound Mycophenolic Acid in Pediatric and 053 S Daping Zhang, Diana Chow, Jamie L. Renbarger Adolescent Patients Post-hematopoietic Stem Cell Transplantation

Biosimilars

Poster Type Title Authors

Comparison of Metabolic and Mitogenic Response In Vitro of the Rapid- Juergen Dedio, Birgit Niederhaus, Marcus Korn, 054 E acting Insulin Lispro Product SAR342434 and US- and EU-approved Surya Prakash, Norbert Tennagels Humalog® Ernesto Luna, Pankaj Agrawal, Riyaz Mehta, Use of an In Vitro Model of Human Immunity to Evaluate the Innate Immune 055 E Maria E. Boone, Charlotte Vernhes, Colombe Denys, Profile of Originator and Biological Copies of Insulin Glargine Bhaswati Mukherjee, Norbert Tennagels, Donald Drake

Decision Making in Research & Development Poster Type Title Authors

Use of an Obese Population in Phase 1 to Evaluate the Pharmacology of Carla Chieffo, Jeff Botbyl, Kim Perry, Thomas M. Blok, 058 Oral CXA-10, an Endogenous, Nitro-fatty Acid Signaling Agent Diane K. Jorkasky Placebo as Gold Standard in Randomized, Controlled Trials Based on 059 Charles Oo Literature Search: Potential Confounders and Ethical Concerns

LEGEND: E = Encore Presentation NM = New Member (Dues paid by April, 2016) P = Podium Presentation S = Student Abstract SA = Student Award Winner

MED001776 Poster Session 2

Monday, September 26, 2016 / 5:30 – 7:30 pm, Ballroom Salon E – H Drug Interactions Poster Type Title Authors Effects of Multiple-dose Administration of Itraconazole on the Single- Carol Cronenberger, Anna Plotka, Kelly Ryan, 060 E dose Pharmacokinetics of Conjugated Estrogens/Bazedoxifene in Joanne Salageanu, William McKeand Postmenopausal Women Communication of Drug Interactions with Gastric Acid-reducing Agents: A 061 S, SA Edwin Lam, Sue Chih Lee Survey of Clinical Practice Guidelines The Effect of Milk Thistle (Silybum marianum) and its Main Flavonolignans Ahmed A. Albassam, John S. Markowitz, 062 NM on CYP2C8 Enzyme Activity in Human Liver Microsomes Reginald F. Frye Levo-tetrahydropalmatine Does Not Affect the Pharmacokinetics of Shamia Faison, William D. Hedrich, Robert Gharavi, 063 S Concomitantly-administered Cocaine in Rats Hongbing Wang, Hazem Hassan Effect of Steady-state Esuberaprost on the Safety, Pharmacokinetics and Gina Patel, Hugh Coleman, Xin Chen, Marni Peterson, 064 E Pharmacodynamics of Warfarin in Healthy Adult Subjects Kirby von Kessler, Jordan Shin, Prakash Sista

Experimental Pharmacology in In Vitro & In Vivo Studies Poster Type Title Authors

Defining Endothelin-B Receptor-mediated Neurogenesis in Mice Tolerant to Shruti Gulati, Shantel Jones, Seema Briyal, Anil Gulati, 065 Opioids Shaifali Bhalla Shruti Gulati, Seema Briyal, Mary Leonard, Muhammad Prenatal Exposure to Ethanol and Oxycodone Affects Neurogenesis and 066 Ansari, Muralidhara Devarapalli, Lorene Schweig, Impairs Development of the Neonatal Rat Brain Bhagya Puppala, Anil Gulati Extended Therapeutic Window for Neuroprotection by IRL-1620 in the 067 Ahmed Maki, Seema Briyal, Anil Gulati Treatment of Cerebral Ischemia Effect of Maternal Cannabinoid Abuse on CB1, ETB, VEGF and NGF Kevin Cooper, Mary Leonard, Seema Briyal, Aarti 068 Expression in the Postnatal Rat Brain Amlani, Preetha Prazad, Ramona Donovan, Anil Gulati Effect of Tidal Volume on Cardiovascular and Blood Gas Parameters in a Gwendolyn Pais, Zhong Zhang, Suresh Havalad, 069 NM Lipopolysaccharide Infusion Model of Septic Shock in the Rat Anil Gulati Dimitri Grigoriadis, Evan Smith, Ajay Madan, 070 In Vitro Pharmacologic Characteristics of Valbenazine and its Metabolites Bill Aurora, Haig Bozigian

Mechanism of Action Poster Type Title Authors

Abhigyan Ravula, Adriaan Bruijnzeel, Barry Setlow, 071 S, NM Development of Dependence After Exposure to Cannabis Smoke in Rats Marcelo Febo, Darin Jagnarine, Hartmut Derendorf

Model-based Drug Development Poster Type Title Authors

Population Pharmacokinetics and Exposure-Response Modeling Analyses of Yan Xu, Omoniyi Adedokun, Daphne Chan, Chuanpu 072 NM Golimumab in Pediatric Patients With Moderate-to-Severe Ulcerative Colitis Hu, Zhenhua Xu, Richard Strauss, Honghui Zhou

LEGEND: P = Podium Presentation E = Encore Presentation S = Student Abstract NM = New Member (Dues paid by April, 2016) SA = Student Award Winner 55 MED001777 Poster Session 2

Monday, September 26, 2016 / 5:30 – 7:30 pm, Ballroom Salon E – H Pharmacogenomics

Poster Type Title Authors

Analysis of the CYP2D6*10 Allele in Relation to Dextromethorphan 074 Rui Chen, Xin Zheng, Jun Shi, Pei Hu O-demethylation Capacity in a Chinese Population

Pharmacometrics

Poster Type Title Authors

Jessica Wojciechowski, Julie Desrochers, S, SA, To Antidote or Not? Web-based Antidote Recommendation Tool for Acute 076 Wendy Klein-Schwartz, Suzanne Doyon, P Acetaminophen Overdose Jogarao V. Gobburu, Mathangi Gopalakrishnan Population Pharmacokinetic and Pharmacodynamic Modeling of Toshimi Kimura, Hiroshi Kawamoto, Yutaka Fukaya, 077 Glucalpidase and High-dose Methotrexate Using Modified Michaelis-Menten Shinobu Kashiwase, Mariko Takahashi Elimination Model Michael Neely, Xiaowei Fu, Ashley Margol, 078 Voriconazole Dosing in Children Under Two Years Teresa Rushing, Siyu Liu, Stan Louie Eun B. Lee, Nikki Daskalakis, Christine Xu, Disease-Drug Interaction of Sarilumab and Simvastatin in Patients With 079 E Anne Paccaly, Barry Miller, Roy Fleischmann, Rheumatoid Arthritis Inga Bodrug, Alan Kivitz Model-based Meta-analysis of Progression-free Survival Time in Non- Mengyao Li, Ahmed Hamed Salem, Shekman Wong, 080 Hodgkin’s Lymphoma Patients Kevin Freise

Regulatory Issues Poster Type Title Authors

Alfred H. Balch, Tian Yu, Joseph E. Rower, Joseph R. 081 Is Generic Tacrolimus Bioequivalent In Patients? Sherbotie, E.K. Korgenski, Catherine M.T. Sherwin Impact of Variability on Therapeutic Success for Drugs with Narrow Elyes Dahmane, Mathangi Gopalakrishnan, Liang 082 NM Therapeutic Index Zhao, Lanyan Fang, Jogarao V. Gobburu, Vijay Ivaturi A Signal-to-Noise Ratio Classification System of Drugs to Investigate Generic Eliford N. Kitabi, Vijay Ivaturi, Lanyan Fang, Liang Zhao, 083 NM Drug Ineffectiveness Claims Jogarao V. Gobburu, Mathangi Gopalakrishnan Jörg Täubel, Dilshat Djumanov, Sara Fernandes, 084 Evaluation of QT Variation Over 24 h: Correlation With Food Intake Georg Ferber

085 Stability of the Effect of a Standardized Meal on QTc Jörg Täubel, Sara Fernandes, Georg Ferber

LEGEND: E = Encore Presentation NM = New Member (Dues paid by April, 2016) P = Podium Presentation S = Student Abstract SA = Student Award Winner

MED001778 Poster Session 2

Monday, September 26, 2016 / 5:30 – 7:30 pm, Ballroom Salon E – H

Risk Management, Legal Issues Poster Type Title Authors

Risk-based Monitoring: A Global Study Focusing on Perception and Merits 086 Prajna Kumar, Manoj P. Jadhav Among Clinical Investigational Sites

Safety & Eficacy Poster Type Title Authors

087 QT Correction For Heart Rate: Use of the Absolute Correction Factor Charles Oo, Thomas Chou, Michael J. Fossler, Jr

Special Populations, Including Women, Children, the Elderly & Obese Patients Poster Type Title Authors

S, SA, Dose Optimization of Dichloroacetate Using a Semi-mechanistic Population Naveen Mangal, Albert Shroads, Taimour Langaee, 088 P, NM Pharmacokinetic Model Peter Stacpoole, Stephan Schmidt

Development and Qualification of a Pediatric Population-based Sumit Basu, Christoph Niederalt, Haitao Yang, 089 S, SA Pharmacokinetic Model for Biologics in PK-Sim® Yi Ting Lien, Thomas Eissing, Stephan Schmidt

Vancomycin-associated Nephrotoxicity Among Pediatric Patients With Rose Caston, Jonathan E. Constance, Alfred H. Balch, 090 S Hematologic Malignancy E.K. Korgenski, Catherine M.T. Sherwin Pharmacokinetics of Clofarabine in Pediatric Subjects Prior to Allogenic 091 S Aksana K. Jones, Vijay Ivaturi, Janel Long-Boyle Hematopoietic Cell Transplantation Prediction of Valganciclovir and Ganciclovir Pharmacokinetics in Different Viera Lukacova, Petra Goelzer, Micaela Reddy, 092 Pediatric Groups Using a Physiologically-based Pharmacokinetic Model Gerard Greig, Bruno Reigner, Neil Parrott Further Validation of a Novel Descriptor of Renal Drug Elimination in 093 Virginia Ramos-Martín, Walter Yamada, Michael Neely Neonates Using Teicoplanin Gina Patel, Thomas Marbury, Kenneth Lasseter, A Comparison of Safety and Pharmacokinetics of Esuberaprost (BPS-314d- 094 E Jolene K. Berg, Xin Chen, Stephanie Peychal, MR) in Subjects with Normal, Mild and Moderate Hepatic Impairment Kirby von Kessler, Jordan Shin, Prakash Sista Aksana Jones, Kevin Freise, Suresh Agarwal, Venetoclax Pharmacokinetics in Hematological Malignancies Subjects with 095 Maria Verdugo, Rod Humerickhouse, Shekman Wong, Renal and Hepatic Impairment Ahmed Hamed Salem

LEGEND: E = Encore Presentation NM = New Member (Dues paid by April, 2016) P = Podium Presentation S = Student Abstract SA = Student Award Winner

57 MED001779 VISION & MISSION To improve health by optimizing therapeutics; Provide innovative leadership and interdisciplinary education that will enable the generation, integration and translation of scientiic knowledge to optimize research, development and utilization of medication for the beneit of all.

ACCP OFFICERS ACCP REGENTS

PRESIDENT Jeffrey S. Barrett, PhD, FCP Bernd Meibohm, PhD, FCP Sanoi Univ of Tennessee Lawrence J. Cohen, PharmD, BCPP, FASHP, FCCP, FCP PRESIDENT ELECT Univ of North Texas System Coll of Pharmacy John van den Anker, MD, PhD, FCP Vera Donnenberg, PhD, FCP Children’s National Health System & Univ Children’s Hosp, Basel Univ of Pittsburgh School of Medicine SECRETARY Megan A. Gibbs, BSc Pharm, PhD, FCP Vikram Arya, PhD, FCP Amgen Inc

TREASURER Guenther Hochhaus, PhD, FCP Michael W. Jann, PharmD, FCP Univ of Florida Univ of North Texas System Nancy A. Lass, MD, FAAP, FCP, LLD Coll of Pharmacy NL Specialty Consulting Inc IMMEDIATE PAST PRESIDENT Donald E. Mager, PharmD, PhD, FCP Lisa L. von Moltke, MD, FCP Univ of Buffalo SUNY Alkermes Inc Diane R. Mould, PhD, FCP Projections Research Inc Anne N. Nafziger, MD, PhD Bertino Consulting Michael N. Neely, MD, MSc, FCP Univ of Southern California Keck School of Medicine Peter H. Wiernik, MD, FCP Cancer Research Fdtn Honghui Zhou, PhD, FCP Johnson & Johnson 58

MED001780 New Members: August 1, 2015 – July 31, 2016

Oluseyi Adeniyi, PharmD, PhD** William Hedrich, BS** Sarayu Pai, MS** Jihye Ahn, MS** Bassant Ibrahim, MD Gwendolyn Pais, PhD Ahmed A. Albassam, PhD Vijay Ivaturi, MS, PhD Chaitali Passey, PhD Celina Alvarez, BS Sibo Jaing, BS, MS** Devin Pastoor, MTOX** Daren Austin, PhD Kathleen Job, PhD** Antonia Periclou, PhD Janelle Baker, MD** Shamir Kalaria, PharmD** Haili Ping, PhD Howard Ball, PhD Vipada Khaowroongrueng** Abhigyan Ravula, PhD** Cindy Bednasz, PharmD** Yu Jin Kim, MPharm** Micaela Reddy, PhD S. Eralp Bellibas, MD, PhD, FAHA Eliford N. Kitabi, PhD** Victoria Richards, PhD Manju Bhaskar, PhD** Aravinda Kumar, MBBS, MD Keith Schmidt, PharmD** Sihem Bihorel, MS, PharmD, PhD Parag Kumar, PharmD Renato Scialis, PhD Edward Brennan, MD Marc Lefebvre, PhD Jie Shao** Kristina M. Brooks, PharmD** Chunze Li, PhD Jie Shen, PhD Tal Burt, MD Tao Liu, BSc** Surjit Singh, MBBS, MD, DM Hongliang Cai, PhD Kristi Lorelli, BA, MA** Harpreet Smith, RN, MSN, CCRC Tatiana Claro da Silva, PhD Stan Louie, PharmD Fabricio Souza, MD Sarah Cook, PhD** Hong Lu, PhD Daniel Spyker, PhD, MD Joseph M. Custodio, PhD Jia Lu, PhD Ajit Suri, PhD Elyes Dahmane, PhD** Pooja Manchandani, MS** Sekihiro Tamaki, MSc Shailendra Dandge, MBBS, MD Naveen Mangal, BS, MS** Stacey Tannenbaum, PhD Durdant Dave, MS, MBBS Francisco Jorge Batel Marques, PhD, PharmD Michiel van Agtmael, MD Oscar Della Pasqua, PhD Kiran Marthak, MD* Hechuan Wang, MS** Clapton Dias, PhD Anne Mattson, PharmD Yan Xu, MD, PhD Elena Enioutina, MD, PhD Shailly Mehrotra, BPharm** Ryoichi Yano, PhD Galen Eversole, MD Prithpal Singh Metreja, MBBS, MD Zhiling Yu, PhD** Abiy Habtewold Eyakem, PhD** Lindsey Millard, PhD Shadia Zaman, PhD** Nashid Farhan** Tsitsi Grace Monera-Penduka, MSc Nicole Zane, PharmD, PhD** Andrea Flynn, MD** Jason Moore, PharmD** Yilong Zhang, PhD Jomy George, PharmD, BCPS (AQ-ID) Yeruk Mulugeta, PhD Xiaochen (Molly) Zhao, PhD Roberto Gomeni, PhD, HDR James J. O’Donnell, PhD Nancy Zheng, PhD Dionna Greene, MD Akinyemi Oni-Orison, PharmD, PhD** Hazem Hassan, PhD, RPh, RCDS Ying Ou, PhD

* Fellow ** Student Member

59 MED001781 American College of Clinical Pharmacology 21750 Red Rum Dr, Suite 137, Ashburn, VA 20147

mailing address: PO Box 1758, Ashburn, VA 20146-1758

phone: 571.291.3493

website: www.ACCP1.org

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