Accelerating Development & Advancing Personalized Therapy · 2013-10-18 · Harry Glorikian, Managing director, precision for Medicine this talk will review current efforts and approaches

ADAPTcongress.comCambridge Healthtech Institute, 250 First Avenue, Suite 300, Needham, MA 02494 | www.healthtech.com

FeAtured SpeAkerS

Alan WrightChief Medical OfficerRoche Diagnostics

Carolyn ComptonProfessorArizona State University

Nicholas C. DracopoliVP, Janssen R&DJohnson & Johnson

Franklin CockerillChair, Laboratory Medicine Mayo Clinic

Accelerating Development & Advancing Personalized Therapy

Cambridge Healthtech Institute’s Fifth Annual

November 4-6, 2013 | Boston Marriott Cambridge | Cambridge, MA

The Leading Annual Meeting Dedicated to Personalized Medicine Research and Implementation

Conference Programs:

November 4-5

Executive Summit: Global Strategies for Companion Diagnostics

Clinical Biomarker Assay Development

November 5-6

Clinical and Translational Biomarkers

Dinner Courses:• executive thinktank: Strategies for Companion diagnostic development

• Fit-for-purpose Biomarker Assay development and Validation

• Next-Generation Sequencing as a Clinical test

NEW LOCATION

Register by September 27 and sAvE up to $200!

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2 | ADAPT Congress ADAPTcongress.com



Monday, November 4

7:30 am Conference Registration and Morning Coffee

8:30-10:05 Incorporating Companion Diagnostics into Drug Development Clinical Assay Development

10:05-10:45 Coffee Break

10:45-12:00 pm Global Regulatory Considerations for CDx Clinical Assay Development (continued)

12:00-1:30 Enjoy Lunch on Your Own

1:30-2:50 Making Precision Medicine More “Precise” Clinical Utility of Next-Generation Sequencing

2:50-3:50 Refreshment Break in the Exhibit Hall with Poster Viewing

3:50-5:00 Plenary Keynotes

5:00-6:00 Welcome Reception in the Exhibit Hall with Poster Viewing

6:00-9:00

Dinner Courses (Separate registration required) • SC1: Executive ThinkTank: Strategies for Companion Diagnostic Development: Addressing

Regulatory, Reimbursement, and Co-Development Challenges• SC2: Fit-for-Purpose Biomarker Assay Development and Validation

Tuesday, November 5

7:30 am Breakfast Presentation or Morning Coffee

8:30-10:05 Companion Diagnostic Assay Development

10:05-10:45 Coffee Break in the Exhibit Hall with Poster Viewing

10:45-12:00 pm Reimbursement and Economic Valuation of CDx Laboratory-Developed Tests

12:00 Close of conferences


12:00 Main Conference Registration

1:30-3:10 Implementing Personalized Medicine: Biomarkers for Patient Stratification


4:15-5:30 Implementing Personalized Medicine: Biomarkers for Patient Stratification (continued)

6:00-9:00 Dinner Courses (Separate registration required)• SC3: Next-Generation Sequencing as a Clinical Test

Wednesday, November 6

7:30 am Breakfast Presentation or Morning Coffee

8:25-10:20 Biomarker Utility in Drug Development


11:20-1:00 pm Biomarker Utility in Drug Development (continued)

1:00 Close of conference

Conference-at-a-Glance

ADAPTcongress.com ADAPT Congress | 3

Dinner Courses*

Monday, November 4, 6:00-9:00 pm

SC1: Executive ThinkTank Strategies for Companion Diagnostics Development: Addressing Regulatory, Reimbursement, and Co-Development Challenges

Panelists to be Announced

please visit www.AdAptcongress.com for more information.

SC2: Fit-for-Purpose Biomarker Assay Development and Validation

Instructor:John L. Allinson, FIBMS, Vice President, Biomarker Laboratory Services, ICON Development Solutions

this tutorial will provide recommendations on the “fit-for-purpose” best practices in the development and validation of biomarker assays for exploratory or advanced biomarker applications. Strategies for different applications at various phases of biomarker development will be described. key elements in the method of development and validation will be illustrated with examples, including reference to standard material, sample stability and collection integrity, validation and QC samples, validity of reference standards, calibration curve fitting methods, method optimization and feasibility studies. Special challenges in protein biomarker assays will be discussed, including strategies for moving from biomarker panels in the exploratory phase to the few markers chosen to support clinical trials, cross-validation of biomarker assays, etc.Outline:1. Introduction: nomenclature, types of biomarker methods/

assays, method development and validation roadmap, fundamental validity, similarity and differences from pk assays and diagnostic applications

2. pre-analytical and bioanalytical elements: target range, standards, validation and QC samples, stability, matrix effect, specificity and relative selectivity

3. Calibration curve model selection, evaluation and weighting4. Method feasibility and optimization with precision profiles5. evaluation of some pre-study validation characteristics such as

precision, bias, sensitivity and quantification limits6. use of sample controls for in-study performance monitoring

and conformance testing among laboratories7. Special considerations for multiplex assays, cross-validation of

assays, etc.8. Method comparisons

tuesday, November 5, 6:00-9:00 pm

SC3: Next-Generation Sequencing as a Clinical Test

Instructors:Nazneen Aziz, Ph.D., Director, Molecular Medicine, Transformation Program Office, College of American Pathologists

Seth Crosby, M.D., Director, Partnerships & Alliances, Washington University School of Medicine

Next-Generation Sequencing (NGS) is used widely in clinical research for the discovery of disease-associated genes and the clinical community is beginning to embrace this technology for diagnostic testing. the rapid evolution of NGS technologies presents significant opportunities and challenges for researchers and clinicians for improving health outcomes, particularly with respect to an increased emphasis on personalized and preventive medicine. Adoption of NGS in the clinical laboratory setting requires the adoption of many processes and procedures, such as the analytic and clinical validation of the test, CLIA certification/CAp accreditation, standards for reference materials, availability for proficiency testing, and questions regarding reimbursement and informed consent. the success of NGS as a viable diagnostic modality depends on many branches of the healthcare community working together. this session will be informative and practical for the researcher and laboratorians who are considering launching NGS as a clinical test.

*Separate registration required

CORPORATE SPONSORS

Corporate Support Sponsor


InauguralNovember 4-5

MONdAy, NOvEMbEr 4


8:30-8:40 Welcome Remarks from Conference Director

Incorporating Companion Diagnostics into Drug Development

8:40-8:45 Chairperson’s Opening Remarks

8:45-9:10 Presentation to be Announced

9:10-9:35 The National Biomarker Development Alliance: An Incubator for Standards-Based Best Practices with an End-to-End Approach Carolyn Compton, M.D., Ph.D., Professor, School of Life Sciences, Arizona State University

9:35-10:05 Overview of Novel Strategies to Sponsored by Improve Drug Discovery and Development in the Era of Precision Medicine Harry Glorikian, Managing director, precision for Medicine this talk will review current efforts and approaches within industry to streamline drug development and discovery – including target selection, validation, clinical trial patient selection and monitoring.


Global Regulatory Considerations for CDx10:45-11:10 Global Regulations and Their Impact on Genetic Data/TestingAmelia Warner, Ph.D., President, Gentris Corp.Global regulations for human specimen testing, specifically genetic sample testing and data usage, are rapidly changing. Specimens collected for assay development or diagnostic development have additional requirements for consent and management. there is a growing conflict between the goals of medical research and provisions for protections for patient privacy. this talk will focus on the balance of protection of subjects while advancing the goals of biomedical research for the benefit of patients now and in the future.

11:10-11:40 Meeting the Clinical Utility Needs Sponsored by of Regulators and Payers for DiagnosticsDavid Parker, Ph.D., Vice President, Market Access Strategy, Precision for MedicineJudi Smith, Vice President, in vitro Diagnostics & Quality, Precision for MedicineIncreasingly payers and the FdA are requesting clinical utility evidence as part of their approval or acceptance processes for both Cdx and IVds. precision will explore the implications for clinical utility evidence development including the type of diagnostic test, regulatory status of the test, inclusion into the associated therapy’s label and how it will be used to guide decision making. We will take a deeper dive into considerations to for account as you plan your study – ensuring proper integration of regulatory and payer needs and ultimately, how the design will meet those needs and how it can affect the rOI on evidence development.

11:40-12:05 pm Forthcoming and Current EU Regulations Surrounding Companion DiagnosticsSylvie le Gledic, Director, Personalized Medicine, Voisin ConsultingIt is generally recognized that the current european In Vitro diagnostic (IVd) directive presents a certain number of weaknesses, and more particularly an inadequate classification system (based on positive lists) that leads to an insufficient level of scrutiny for many medium- to high-risk IVd tests. On September 26, 2012, the european Commission released the first draft of the new eu regulation for in vitro diagnostic medical devices. Companion diagnostics (Cdx) will be classified as high-risk devices and

specific requirements will be added to get these products Ce marked. this presentation will describe the anticipated additional requirements that will apply to Cdx and the practical implications for IVd and drug developers.

12:05 -1:30 Enjoy Lunch on Your Own

Making Precision Medicine More “Precise”1:30-1:35 Chairperson’s Opening Remarks

1:35-2:00 Leveraging Partnerships to Deliver Precision MedicineMorten Sogaard, Ph.D., Executive Director and Head, Biotechnology and Precision Medicine, External R&D Innovation, Worldwide R&D, PfizerHealthcare and pharmaceutical r&d today is expensive, reactive and often ineffective. We need to more precisely measure disease progression and treatment outcomes in patients to increase therapeutic index by treating only those patients likely to respond, or excluding those most likely to experience side effects. this talk will give examples of how precision medicine is applied in pfizer r&d projects leveraging diagnostics, genetics and real-world data with a particular emphasis on innovative partnerships.

2:00-2:25 From Policy to Process: FDA Expedited Programs and Precision MedicineFederico Goodsaid, Ph.D., Vice President, Strategic Regulatory Intelligence, Vertex PharmaceuticalspduFA V and FdASIA legislation in 2012 required the FdA to issue a guidance document on expedited programs. the draft for this document, issued shortly after the Final rule on Orphan drugs, discusses fast track designations, breakthrough therapy designations, accelerated approval and priority review designations. Some questions about these programs are addressed by these two documents, such as the similarities and differences between fast track and breakthrough therapy designations. Other questions about surrogates in accelerated approvals remain open. the impact of this document on the development of therapies in precision medicine will be the subject of this talk, with a focus on the impact for the development of rare disease therapies.

2:25-2:50 Use of Biomarkers towards Understanding and Implementing Personalized Medicine in the Rare Disease SettingChetan Lathia, Ph.D., Executive Director, Clinical Pharmacology, Alexion Pharmaceuticals


PLENARY KEYNoTES3:50-3:55 Chairperson’s Opening Remarks

3:55-4:20 Personalized Medicine and Shrinking Patient Populations: Rethinking the Value of Companion DiagnosticsAlan T. Wright, M.D., MPH, CMO, Roche Diagnostics Corporationdespite the critical role diagnostic tests play in personalized medicine, they are significantly undervalued, and the disparity that exists between current investment models and reimbursement policies for diagnostics is not tenable long-term. As science helps define the uniqueness of each person’s condition, the potential volume for specific tests will be less. to maintain investment from the private sector in diagnostics—which contributes significantly to medical innovation and helps demonstrate the clinical utility of FdA-approved tests—a more attractive path to market is needed.

4:20-5:00 Panel Discussion: Making Precision Medicine More “Precise”Panelists to be Announced



November 4-5


Inaugural

6:00-9:00 Dinner Courses* SC1: Executive ThinkTank: Strategies for Companion Diagnostic Development: Addressing Regulatory, Reimbursement, and Co-Development Challenges

6:00-9:00 Dinner Short Course* SC2: Fit-for-Purpose Biomarker Assay Development and Validation

See page 3 for additional details *Separate registration required

TuEsdAy, NOvEMbEr 5

7:30 am Breakfast Presentation (Sponsorship Opportunity Available) or Morning Coffee

Companion Diagnostic Assay Development8:30-8:35 Chairperson’s Opening Remarks

8:35-9:00 Assay and Platform Considerations in Companion Diagnostic DevelopmentRon Mazumder, Ph.D., MBA, Global Head, Research and Product Development, Janssen Diagnostics, Janssen Pharmaceutical Companies of Johnson & JohnsonA number of considerations dictate the choice of which diagnostic platform to use. these include time required for development, commercial access, regulatory considerations, and cost/reimbursement. In addition, flexibility in development and implementation of a validated diagnostic is required for many clinical studies. I will highlight some of these issues in this talk.

9:00-9:25 How Is Early Biomarker Assay Analytical Verification Linked into Companion Diagnostic Clinical Validation: Is It Upstream or Is It in Parallel? Shane Weber, Ph.D., Director, Diagnostics, Clinical Research and Precision Medicine, PfizerBoth the pharmaceutical and diagnostic industries are moving towards proactive precision medicine patient stratification approaches. early implementation of patient stratification biomarker strategies into precision medicine development programs are much more likely to lead to phase III registration trials that successfully validate a companion diagnostic. the biomarker assay analytical verification process must have appropriately prepared the assay in the uS for readiness as an Investigational use Only (IuO) assay under the FdA’s Investigational device exemption (Ide) process into clinical trial use.

9:25-9:50 Choosing the Right Assay for a Companion Diagnostic Development ProgramHans Christian Pedersen, MSc, Principal Scientist, Research & Development, PharmDx, Dako Many choices exist for choosing methods and platforms for companion diagnostics development as a result of the revolution in sequencing technology and the continuous development of technology for measuring dNA, rNA and protein in tissues. this session will go through the strengths and weaknesses of the various technologies, giving specific examples within immunohistochemistry (IHC) and FISH of successful Cdx assays and discussing the future development of IHC and FISH Cdx from an IVd company perspective.

9:50-10:05 A Risk Mitigation Model for CDx Sponsored by Co-Development PartnershipsKierstyn Claycomb, Senior Manager, Business Development, Companion Diagnostics, AsuragenAsuragen’s unique co-development model reduces costs, accelerates timelines and drives global commercial adoption. detailed strategic planning ensures programs align with global regulatory and healthcare authorities to enable global registration and approval. Asuragen manufactures and distributes FdA-compliant IVd products with complete sample to answer solutions.


Reimbursement and Economic Valuation of CDx

10:45-11:10 A U.S. Managed Care Organization’s Process for Developing Clinical Policies for Companion Diagnostics Robert S. McDonough, M.D., Head, Clinical Policy Research & Development, Aetnathis session will address the purposes and goals of clinical policy at a managed care organization and the criteria and sources of data used to evaluate companion diagnostics. this session will also describe the managed care organization’s clinical policy development process, the policy update process, and the process for assessing cost impact of clinical policies.

11:10-11:35 Moving from Gene Testing to Genome Sequencing: Coverage and Reimbursement in a Brave New World Kathryn A. Phillips, Ph.D., Professor, Health Economics and Health Services Research; Director, UCSF Center for Translational and Policy Research on Personalized Medicine, University of California, San FranciscoNew technologies are increasing our ability to test for multiple genes simultaneously, but these technologies challenge many existing approaches to payer coverage and reimbursement. Based on our work with a wide range of private payers and thought leaders, I will discuss how healthcare decision makers will weigh the benefit-risk tradeoffs in making coverage and reimbursement decisions for new technologies such as whole-genome sequencing and what evidence will be needed to inform those decisions.

11:35-12:00 pm The Economic Value within Companion Diagnostics and Stratified Medicines RelationshipsMartina Kaufmann, Ph.D., International Projects Director, Integrated Medicines, Ltd.the development and commercialization of stratified medicines requires a whole new appreciation of the economic value of the relationship between pharmaceutical companies and medical diagnostic companies. this economic valuation also impacts other healthcare stakeholders as it influences pricing, payment and evaluation of technologies for both clinical and cost effectiveness. the clinical effectiveness of stratified medicines also impacts patients’ and clinicians’ education as it is important that these key stakeholders are able to recognize the benefits of selective treatment.

12:00 Close of Global Strategies for Companion Diagnostics Meeting



InauguralNovember 4-5

MONdAy, NOvEMbEr 4


8:30-8:40 Welcome Remarks from Conference Director

Clinical Assay Development8:40-8:45 Chairperson’s Opening Remarks

8:45-9:10 Bridging Research and “Clinical” Assays in Pharmaceutical Research & DevelopmentJohn L. Allinson, FIBMS, Vice President, Biomarker Laboratory Services, ICON Development SolutionsMany biomarker assays used in drug development are research assays (i.e., not accredited diagnostic devices). this presentation will look at the following: basic validation experiments across assays in research and diagnostics, differences and assay evolution as methods progress through different uses of results data, the requirements for accreditation of assays to be used in diagnostics, and a brief look at the development of a companion diagnostic and its implications from the laboratory perspective.

9:10-9:35 Real-Time Multiplexed Cancer Pharmacodynamics in PatientsS. Michael Rothenberg, M.D., Ph.D., Instructor, Medicine, Massachusetts General HospitalA major shortfall in patient care is the difficulty in assessing for early response to anti-cancer therapy. We have developed a methodology using multispectral imaging (MSI) immunofluorescence microscopy to quantify the effects of therapy on individual treatment biomarkers in tumor cells from patient-derived biopsies and circulating tumor cells (CtCs) in real time, while patients are undergoing therapy, weeks before radiographic response can be measured. I will discuss applying this approach to patients with diverse cancers undergoing treatment with targeted therapies.

9:35-10:05 Assay and Kit Lot Bridging Sponsored by Considerations for Single and Multiplex Biomarker Analysis in Support of Clinical StudiesAfshin Safavi, Ph.D., Senior Vice President, Bioanalytical Operations, BioAgilytix Labs Biomarker analysis has become a common practice by many pharmaceutical companies to help pk/pd modeling. the reliability of outcomes is heavily influenced by the quality of the reagents. One of the challenges that bioanalytical labs face when running biomarker studies is the control of lot-to-lot variability of critical reagents and commercial immunoassay kits. Case studies will be presented to highlight the key bioanalytical considerations involved in running successful biomarker analyses in support of clinical studies.


10:45-11:10 Development Strategies to Achieve a True Free Target AssayMartin Schwickart, Ph.D., Scientist, Clinical Pharmacology and DMPK, MedImmuneto support biologics development, free target assays are routinely used to monitor drug effect and to predict an appropriate dose. In many assays, free target is overestimated due to dilution of the sample and re-equilibration in presence of a capture reagent. We present here a structured strategy and assay development to measure true free target and compare the effect of two drugs with different affinities.

11:10-11:35 The Central Role of Human Tissue in Diagnostic Assay DevelopmentCarol Cheung, M.D., Ph.D., Department of Pathology, University Health Network, Canadareliable sources of appropriately processed and annotated human tissue are required for diagnostic assay development. As such, requests for

materials from clinical care institutions, where tissues are fixed and processed according to strict laboratory protocols, are ever increasing. understanding the regulatory requirements that institutions must abide by with respect to different classes of tissue is paramount to the responsible and safe acquisition of human tissue for development and validation of innovative new tests.

11:35-11:50 Diagnostic Classifiers for the Sponsored by Detection of Bladder CancerScott McKeown, Ph.D., Research & Development Consultant, Randox Laboratories, Ltd.patients presenting with hematuria require investigations, including cystoscopy and imaging of their upper urinary tract, to identify the source of bleeding. this is a significant health burden, which is set to increase because of our aging population. using randox Biochip Array technology (BAt), we have identified diagnostic classifiers for detecting bladder cancer.

11:50 Enjoy Lunch on Your Own

Clinical Utility of Next-Generation Sequencing1:30-1:35 Chairperson’s Opening Remarks

1:35-2:00 Leaping the Valley between Research and Medicine: Notes from the First Two Years in a CAP-Certified NGS LabSeth Crosby, M.D., Director, Partnerships & Alliances, Washington University School of MedicineAre clinical standards of evidence/annotation different than those of research? How is a clinical NGS test validated? In a clinical lab, what needs to be in the analysis tool chest? What has Washington university built that allows a physician to interpret sequencing data?

2:00-2:25 College of American Pathologists’ Standards and Proficiency Testing for Next-Generation Sequencing for the Clinical Laboratory Nazneen Aziz, Ph.D., Director, Molecular Medicine, Transformation Program Office, College of American Pathologiststhe rapid and ongoing advances in the genetic test market, spurred by the opportunities of Next-Generation Sequencing (NGS), necessitate many facets of the healthcare industry to work cohesively. Adoption of NGS as a clinical test requires the adoption of many processes and procedures, such as the analytic and clinical validation of the test, CLIA certification/CAp accreditation, standards for reference materials, availability for proficiency testing, genetic counseling, and questions regarding reimbursement, informed consent and incidental findings. this talk will focus on the laboratory requirements developed at CAp for CLIA/CAp accreditation and the plans for proficiency testing for NGS.

2:25-2:50 Discovery of Predictive Biomarkers to Resolve Clinical Dilemmas Using Next-Generation Sequencing George Vasmatzis, Ph.D., Assistant Professor, Laboratory Medicine and Pathology, College of Medicine, Mayo ClinicIdentification of causal dNA rearrangements is critical to the understanding of cancer development and progression, and is important clinically for accurate prognosis and efficient treatment. Mixed grade tumors portend a significantly more aggressive phenotype than phenotypically pure highly differentiated tumors. It is critical to understand the molecular relationship of adjacent low-grade and high-grade tumor cell populations and relate these molecular abnormalities to disease progression. to decipher molecular relatedness, we used laser capture microdissection (LCM) and whole-genome amplification (WGA) to separately collect and amplify dNA from adjacent cell populations with different patterns in both prostate and lung adenocarcinomas. We then carried out massively parallel mate-pair NGS to examine the landscape of large chromosomal alterations. Our findings indicate that while patterns from the same tumor each possess unique breakpoints, they also share identical ones, pointing to a common origin. Hierarchical clustering analysis revealed that low-grade pattern has greater breakpoint similarity to its high-grade partner than to similar pattern from a different patient. Our studies provide evidence that LCM, WGA, and


November 4-5


Inaugural

NGS of adjacent tumor regions are important tools in deciphering lineage relationships and discovering chromosomal alterations associated with tumor progression.


PLENARY KEYNoTES3:50-3:55 Chairperson’s Opening Remarks

3:55-4:20 Personalized Medicine and Shrinking Patient Populations: Rethinking the Value of Companion DiagnosticsAlan T. Wright, M.D., MPH, CMO, Roche Diagnostics Corporationdespite the critical role diagnostic tests play in personalized medicine, they are significantly undervalued, and the disparity that exists between current investment models and reimbursement policies for diagnostics is not tenable long-term. As science helps define the uniqueness of each person’s condition, the potential volume for specific tests will be less. to maintain investment from the private sector in diagnostics—which contributes significantly to medical innovation and helps demonstrate the clinical utility of FdA-approved tests—a more attractive path to market is needed.

4:20-5:00 Panel Discussion: Making Precision Medicine More “Precise”Panelists to be Announced


6:00-9:00 Dinner Courses* SC1: Executive ThinkTank: Strategies for Companion Diagnostic Development: Addressing Regulatory, Reimbursement, and Co-Development Challenges

6:00-9:00 Dinner Short Course* SC2: Fit-for-Purpose Biomarker Assay Development and Validation


TuEsdAy, NOvEMbEr 5


Companion Diagnostic Assay Development8:30-8:35 Chairperson’s Opening Remarks

8:35-9:00 Assay and Platform Considerations in Companion Diagnostic DevelopmentRon Mazumder, Ph.D., MBA, Global Head, Research and Product Development, Janssen Diagnostics, Janssen Pharmaceutical Companies of Johnson & JohnsonA number of considerations dictate the choice of which diagnostic platform to use. these include time required for development, commercial access, regulatory considerations, and cost/reimbursement. In addition, flexibility in development and implementation of a validated diagnostic is required for many clinical studies. I will highlight some of these issues in this talk.

9:00-9:25 How Is Early Biomarker Assay Analytical Verification Linked into Companion Diagnostic Clinical Validation: Is It Upstream or Is It in Parallel? Shane Weber, Ph.D., Director, Diagnostics, Clinical Research and Precision Medicine, PfizerBoth the pharmaceutical and diagnostic industries are moving towards proactive precision medicine patient stratification approaches. early implementation of patient stratification biomarker strategies into precision medicine development programs are much more likely to lead to phase III registration trials that successfully validate a companion diagnostic. the biomarker assay analytical verification process must have appropriately prepared the assay in the uS for readiness as an Investigational use Only (IuO) assay under the FdA’s Investigational device exemption (Ide) process into clinical trial use.

9:25-9:50 Choosing the Right Assay for a Companion Diagnostic Development ProgramHans Christian Pedersen, MSc, Principal Scientist, Research & Development, PharmDx, Dako Many choices exist for choosing methods and platforms for companion diagnostics (Cdx) development as a result of the revolution in sequencing technology and the continuous development of technology for measuring dNA, rNA and protein in tissues. this session will go through the strengths and weaknesses of the various technologies, giving specific examples within immunohistochemistry (IHC) and FISH of successful Cdx assays and discussing the future development of IHC and FISH Cdx from an IVd company perspective.


Laboratory-Developed Tests10:45-11:10 Regulatory Issues Facing Laboratory-Developed TestsAndrea Ferreira-Gonzalez, Ph.D., Professor and Chair, Division of Molecular Diagnostics; Director, Molecular Diagnostics Laboratory, Department of Pathology, Virginia Commonwealth University

11:10-11:35 The Value of LDTs as Companion Diagnostics: Utility and Compliance RequirementsFranklin Cockerill, M.D., Chair, Laboratory Medicine and Pathology, Mayo ClinicLaboratory-developed tests (Ldts) provide important value for patient care outside of FdA-approved kits. Ldts can be developed quickly to address acute needs for therapeutic decisions, including de novo discoveries of drug-target interactions. Ldts can provide important updating and improvements for current companion diagnostic IVd assays, and serve as pre-market diagnostic tests for clinical drug trials. Ldts also fill an important niche in cases of low burden of disease (“orphan” diseases) where commercialization by IVd manufacturers is not economical. the focus of this talk will not only include the utility of Ldts in patient care, but describe the regulatory requirements for CLIA, FdA and other agencies, including New York State.

11:35 Close of Clinical Biomarker Assay Development Meeting



November 5-6

TuEsdAy, NOvEMbEr 5

12:00 pm Main Conference Registration

Implementing Personalized Medicine: Biomarkers for Patient Stratification

1:30-1:35 Chairperson’s Opening Remarks

1:35-2:00 Patient Selection Biomarker for Notch Inhibitors in CancerTheresa Zhang, Ph.D., Associate Director, Exploratory and Translational Sciences, MerckNotch can play both oncogenic and tumor suppressive roles in human cancer. therefore, it is critical to identify biomarkers that can predict response to Notch inhibition (e.g., GSI [gamma-secretase inhibitors]) and develop clinical assays that can select appropriate patients into clinical trials. this talk will focus on efforts to discover and validate predictive biomarkers for GSI and to develop potential clinical assays for the resulting biomarkers.

2:00-2:25 Broad-Based Cancer Genotyping in Routine Clinical PracticeDarrell R. Borger, Ph.D., Co-Director, Translational Research Laboratory; Director, Biomarker Laboratory, Massachusetts General Hospital Cancer CenterMultiplexed tumor genotyping has been offered as a physician-ordered clinical test at a major uS cancer center, with over 5,000 patients tested across cancer disease types. Advantages of a broad-based profiling approach will be highlighted, indicating how this has revealed new molecular signatures and has been used to foster a genotype-directed approach to clinical trial design. this has provided the foundation for expanding into next-generation sequencing approaches.

2:25-2:40 Sponsored Presentation Sponsored by

Michael Cummens, M.D., CMO, Remedy Informatics, Inc.

2:40-3:05 Talk Title to be AnnouncedKimberly Walter, Ph.D., Senior Research Assistant, Genentech


4:15-4:40 Stratifying Patients to Molecular Clinical Trials in the Era of Precision MedicineDavid A. Fenstermacher, Ph.D., Chief Research Information Officer, Virginia Commonwealth UniversityA prospective longitudinal study, total Cancer Care, currently underway at the Moffitt Cancer Center, has been used to create a comprehensive data warehouse that is able to integrate clinical and molecular data that is being used for in silico clinical trial design in solid tumor malignancies to match patients with novel therapeutics. the studies presented will provide insights to the challenges to use point-of-care clinical data and research-grade molecular data to aid in the design of biomarker-based clinical trials.

4:40-5:05 Implementation of Personalized Cancer TherapyApostolia-Maria Tsimberidou, M.D., Ph.D., Associate Professor, Investigational Cancer Therapeutics, University of Texas MD Anderson Cancer CenterImplementation of personalized medicine requires the consistent and successful identification of genetic alterations in patients’ tumors and effective inhibition of the function of the altered genes. use of molecular profiling and targeted therapy in our phase I program has been associated with improved rates of response, time to treatment failure and survival compared to the standard approach in patients with advanced cancer. Standardization of molecular profiling, novel drug discovery, carefully designed prospective clinical trials and development of “N of 1” shared databases will expedite the implementation of personalized medicine.

6:00-9:00 Dinner Course*SC3: Next-Generation Sequencing as a Clinical Test


WEdNEsdAy, NOvEMbEr 6


Biomarker Utility in Drug Development8:25-8:30 Chairperson’s Opening RemarksRakesh Dixit, Ph.D., DABT, Vice President, R&D and Global Head, Biologics Safety Assessment, MedImmune

8:30-8:55 Big Data and Small Clinical Trials: Translating Biological Data into Useful BiomarkersNicholas C. Dracopoli, Ph.D., Vice President, Janssen R&D, Johnson & Johnsondeveloping useful clinical biomarkers has proved difficult. thousands of biomarkers have been identified, but few have sufficient clinical utility to justify widespread implementation. this presentation will describe the minimal criteria needed to develop predictive markers including discussion of samples, variables, endpoints and algorithms. the development of two different predictive markers will be described to demonstrate the different steps and potential errors in developing robust predictive markers. A comparison will be made between developing single analyte markers and complex molecular profiles. Finally, a summary of the role of biomarkers in oncology drug development will be made showing the recent history of companion diagnostic development in this field.

8:55-9:20 Impact of Protein Biomarkers in a Clinical StudyYoshiya Oda, Ph.D., President, Biomarkers and Personalized Medicine Core Function Unit, EisaiAppropriate biomarkers play important roles in drug development to confirm target engagement, to determine right dosage, to select right patients and to monitor drug efficacy. One of the bottlenecks in biomarker research is to obtain a sufficient quantity and quality of clinical samples. Access to tumor tissues is not always straightforward. Biofluid would be ideal as clinical samples and protein biomarkers are probably major molecules in biofluid samples. examples using clinical samples will be shown.

9:20-9:50 Circulating Tumor Cells: From Sponsored by Enumeration to Comprehensive CharacterizationMark Connelly, Ph.D., Scientific Director, Janssen R&D

9:50-10:05 Identifying the Enemy: Sponsored by.

Decoding the Malignant MessageKalle Ojala, Ph.D., Chief Product Officer, MediSapiens, Ltd.using NGS and oncogenomics Big data to identify clinically relevant oncogenic biomarkers.


Fifth Annual



November 5-6

11:20-11:45 RNA Sequencing to Help Decision Making in Drug Development: An Example in Hepatocellular CarcinomaOscar Puig, Ph.D., Biomarker Experimental Medicine Leader, RocheHepatocellular carcinoma (HCC) is a devastating disease with 5-year survival rates of about 12%. Sorafenib is the only approved systemic treatment for advanced HCC; thus, new therapies are a critical medical need. Biomarkers that can select patients benefiting from specific therapies is an active field of research. We will share our experience using rNAseq to profile liver tumor biopsies in search of predictive markets of response and efficacy.

11:45-12:10 pm Stromics: A Renewed Focus on Host BiomarkersRichard Levenson, M.D., Professor and Vice Chair for Strategic Technologies, Department of Pathology & Laboratory Medicine, University of California, Davis Medical Center

12:10-12:35 Using Clinical Trial Data as a Tool in Evaluating New Safety BiomarkersStephen T. Furlong, Ph.D., Safety Science Lead, AstraZenecathe qualification process for new safety biomarkers is a fundamentally new approach, and an important aspect of qualifying new biomarkers is an understanding of how they compare to the assays they will replace. In this talk we will describe our process for using retrospective data across large sets of clinical trials to review laboratory safety data in healthy volunteers and patient populations. this analysis provides the basis for a comparison of classical safety assays and new safety biomarkers, and helps provide an understanding of how to use these new biomarkers.

12:35-1:00 Pharmacodynamics and Safety Biomarkers of Mavrilimumab Activity in a Phase 2a study in RA PatientsDominic Sinibaldi, Ph.D., Scientific Manager, R&D IS Clinical Informatics, MedImmuneGM-CSF plays a central role in the pathogenesis of rheumatoid arthritis (rA) through effects on macrophages and neutrophils. Mavrilimumab (CAM-3001) is a human monoclonal antibody targeting the alpha subunit of GM-CSF receptor. Marvrilimumab was recently evaluated in rA subjects in a phase 2a study (eArtH). Biomarker (BM) assessments were performed to elucidate mechanistic and safety aspects of mavrilimumab. results suggest that suppressing macrophage activity by targeting GM-CSF receptor alpha may be a novel approach for the treatment of rA.

1:00 Close of Clinical and Translational Biomarkers Meeting

Clinical and Translational BiomarkersFifth Annual

Lead Sponsoring Publications

Sponsoring Publications

Lead Media Partners

Media Partners


John L. Allinson, FIBMS, Vice President, Biomarker Laboratory Services, ICON Development Solutions

Nazneen Aziz, Ph.D., Director, Molecular Medicine, Transformation Program Office, College of American Pathologists

Darrell R. Borger, Ph.D., Co-Director, Translational Research Laboratory; Director, Biomarker Laboratory, Massachusetts General Hospital Cancer Center

Carol Cheung, M.D., Ph.D., Department of Pathology, University Health Network, Canada

Kierstyn Claycomb, Senior Manager, Business Development, Companion Diagnostics, Asuragen

Franklin Cockerill, M.D., Chair, Laboratory Medicine and Pathology, Mayo Clinic

Carolyn Compton, M.D., Ph.D., Professor, School of Life Sciences, Arizona State University

Seth Crosby, M.D., Director, Partnerships & Alliances, Washington University School of Medicine

Michael Cummens, M.D., CMO, Remedy Informatics, Inc.

Rakesh Dixit, Ph.D., DABT, Vice President, R&D and Global Head, Biologics Safety Assessment, MedImmune

Brian Dougherty, Ph.D., Translational Genomics Lead, AstraZeneca R&D

Nicholas C. Dracopoli, Ph.D., Vice President, Janssen R&D, Johnson & Johnson

Andrea Ferreira-Gonzalez, Ph.D., Professor and Chair, Division of Molecular Diagnostics; Director, Molecular Diagnostics Laboratory, Department of Pathology, Virginia Commonwealth University

David A. Fenstermacher, Ph.D., Chief Research Information Officer, Virginia Commonwealth University

Stephen T. Furlong, Ph.D., Safety Science Lead, AstraZeneca

Sylvie le Gledic, Director, Personalized Medicine, Voisin Consulting

Harry Glorikian, Managing Director, Precision for Medicine

Federico Goodsaid, Ph.D., Vice President, Strategic Regulatory Intelligence, Vertex Pharmaceuticals

Martina Kaufmann, Ph.D., International Projects Director, Integrated Medicines, Ltd.

Chetan Lathia, Ph.D., Executive Director, Clinical Pharmacology, Alexion Pharmaceuticals

Richard Levenson, M.D., Professor and Vice Chair for Strategic Technologies, Department of Pathology & Laboratory Medicine, University of California, Davis Medical Center

Ron Mazumder, Ph.D., MBA, Global Head, Research and Product Development, Janssen Diagnostics, Janssen Pharmaceutical Companies of Johnson & Johnson

Robert S. McDonough, M.D., Head, Clinical Policy Research & Development, Aetna

Scott McKeown, Ph.D., Research & Development Consultant, Randox Laboratories, Ltd.

Yoshiya Oda, Ph.D., President, Biomarkers and Personalized Medicine Core Function Unit, Eisai

Kalle Ojala, Ph.D., Chief Product Officer, MediSapiens, Ltd.

David Parker, Ph.D., Vice President, Market Access Strategy, Precision for Medicine

Hans Christian Pedersen, MSc, Principal Scientist, Research & Development, PharmDx, Dako

Kathryn A. Phillips, Ph.D., Professor, Health Economics and Health Services Research; Director, UCSF Center for Translational and Policy Research on Personalized Medicine, University of California, San Francisco

Oscar Puig, Ph.D., Biomarker Experimental Medicine Leader, Roche

S. Michael Rothenberg, M.D., Ph.D., Instructor, Medicine, Massachusetts General Hospital

Afshin Safavi, Ph.D., Senior Vice President, Bioanalytical Operations, BioAgilytix Labs

Judi Smith, Vice President, in vitro Diagnostics & Quality, Precision for Medicine

Martin Schwickart, Ph.D., Scientist, Clinical Pharmacology and DMPK, MedImmune

Dominic Sinibaldi, Ph.D., Scientific Manager, R&D IS Clinical Informatics, MedImmune

Denis Smirnov, Ph.D., Associate Scientific Director, Oncology Biomarkers, Janssen Pharmaceuticals & Janssen Diagnostics

Morten Sogaard, Ph.D., Executive Director and Head, Biotechnology and Precision Medicine, External R&D Innovation, Worldwide R&D, Pfizer

Apostolia-Maria Tsimberidou, M.D., Ph.D., Associate Professor, Investigational Cancer Therapeutics, University of Texas MD Anderson Cancer Center

George Vasmatzis, Ph.D., Assistant Professor, Laboratory Medicine and Pathology, College of Medicine, Mayo Clinic

Kimberly Walter, Ph.D., Senior Research Assistant, Genentech

Amelia Warner, Ph.D., President, Gentris Corp.

Alan T. Wright, M.D., MPH, CMO, Roche Diagnostics Corporation

Theresa Zhang, Ph.D., Associate Director, Exploratory and Translational Sciences, Merck

Distinguished Faculty for ADAPT Congress 2013

Maximize your experience on-site at ADAPT Congress 2013!

The Intro-Net offers you the opportunity to set up meetings with selected attendees before, during and after this conference, allowing you to connect to the key people you want to meet. This online system was designed with your privacy in mind and is available only to registered session attendees of this event. Registered conference attendees will receive more information on accessing the Intro-Net in the weeks leading up to the event!


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Sponsorship & Exhibit

OPPORTUNITIESHotel & Travel

INFORMATIONCONFERENCE VENUE & HOTEL: Boston Marriott Cambridge2 Cambridge Center, 50 BroadwayCambridge, MA 02142tel: 617-494-6600

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discounted room rate Cut-off date: October 7, 2013

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Documents

Accelerating Development & Advancing Personalized Therapy · 2013-10-18 · Harry Glorikian, Managing director, precision for Medicine this talk will review current efforts and approaches