Monday, May 21
1:00-2:00 Main Conference Registration
2:00-2:10 Welcoming Remarks from Conference Director
Julia Boguslavsky, Executive Director, Conferences, Cambridge Healthtech Institute
2:10-2:15 Chairperson's Opening Remarks
2:15-2:45 Turning an Active Compound into a Personalized Medicine: Do Biomarkers Help or Hinder?
Geert Kolvenbag, M.D., Ph.D., Global Product Vice President, AstraZeneca
The co-development of drug and biomarker has several inherent risks and challenges. In addition, the expectations and demands of the oncology community have increased over the last years. These challenges will be illustrated by a case study of a very active compound destined for a fast development program as a personalized medicine, but running into scientific, diagnostic and development challenges. This experience creates questions for the development of drugs and diagnostics today and in the future.
2:45-3:15 Evaluation of Biomarker Performance in the Real World: Comparative and Cost-Effectiveness Considerations
Felix W. Frueh, Ph.D., President, Medco Research Institute, Medco Health Solutions, Inc.
The difference between the potential to achieve an outcome and actually achieving it for a biomarker-guided intervention can be measured as the performance of a biomarker under controlled (optimized) conditions and its actual performance in the real-world. Similar to post-approval safety and efficacy evaluations of drug therapies that differ from data found in pre-market assessments, the impact on clinical outcomes using biomarker-guided interventions are often not fully understood until these interventions are performed and analyzed in the heterogeneous environment of real-world clinical practice. Following the two most critical factors, clinical outcomes and cost associated with reaching these outcomes, the Medco Research Institute has been conducting a series of real-world assessments of the performance of biomarkers to determine whether or not they should be integrated into everyday clinical practice.
3:15-3:45 Biomarkers: Finding the Balance between Driving Decision Making and Value or Distraction from Developing Drugs
Duncan McHale, Ph.D., Vice President, Global Exploratory Development, UCB Pharma
The past 5 years have seen an increasing focus on the identification and qualification of biomarkers. We now have tools which can generate proteomic or nucleotide data on hundreds of thousands of markers in single experiments and whole departments whose aim is to identify and qualify biomarkers. There are now 2 ICH guidances (E15 and E16) on genomic biomarkers and new regulatory processes developed specifically for qualifying biomarkers for use in regulatory decision making. This talk provides examples of where real value has been obtained using biomarkers as well as other areas where it has not. It will ask whether we have the balance right and how much should we invest in biomarkers and when.
3:45-4:15 Predicting Benefit to Therapy: Biomarkers and Molecular Profiles in Oncology Drug Development
Nicholas C. Dracopoli, Ph.D., Head, Oncology Biomarkers, Janssen R&D
The main goals of biomarker research in the pharmaceutical industry are to increase efficiency of the drug development process by improving understanding of the mechanism of action, deeper exploration of PK-PD interactions and predicting response to novel therapies in clinical development. However, predictive biomarkers are still only included in the labels of a minority of the oncology drugs approved in the U.S. since Herceptin in 1998. The development of novel biomarkers has enormous potential to impact drug development and improve patient outcomes. However, no complex molecular or protein profiles have been approved by the FDA to drive therapeutic use of any drug. This presentation will show how the small numbers of patients enrolled in clinical trials and the lack of long-term outcome data have limited the discovery of complex molecular profiles with strong predictive values (both negative and positive), and show how simple biomarkers measuring the status of the drug target or pathway (BRAF mutation, ALK-EML4 translocation, etc.) have been much more successful in the identification of highly predictive biomarkers which have been developed as companion diagnostics to drive therapeutic use of novel oncology drugs.
4:15-5:15 Welcome Reception in the Exhibit Hall with Poster Viewing
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Tuesday, May 22
7:30-8:15 am Breakfast Presentation
Identification of Fluid Biomarkers of Treatment Response in Schizophrenia CSF and Plasma Samples
Eric Schaeffer, Ph.D., Director, Neuroscience Clinical Biomarkers, Bristol-Myers Squibb
Schizophrenia is a heterogeneous disease of complex and poorly understood etiology. Patients are often prescribed one of several approved medications based on their symptoms, but there is a high rate of switching among medications during a period of trial and error, while physicians seek to identify a drug(s) which will result in stabilization of symptoms. Given the variability in treatment response, there would be considerable value in the identification of a biomarker(s) which could provide an indication of whether a patient is responding to a particular medication early in the treatment regimen. This talk will discuss a biomarker identification and validation strategy focusing on highly multiplexed immunoassay panels offered by Myriad RBM.
8:25-8:30 Chairperson's Opening Remarks
8:30-9:00 Zelboraf: The Co-Development of Zelboraf and Its Companion Diagnostic
Walter H. Koch, Ph.D., Vice President and Head of Global Research, Roche Molecular Diagnostics
Cancers can be categorized based on their molecular etiology, including oncogenic driver mutations that are present. The development of targeted therapies alongside companion diagnostics that will identify patients most likely to receive benefit provides the opportunity to increase the success rate for oncology drugs and to decrease development time and associated costs. This presentation will detail the co-development of Zelboraf and the cobas 4800 BRAF V600 mutation test from the diagnostics perspective. This integrated process resulted in approval of the first personalized medicine for the treatment of metastatic melanoma within 6 years of the drug's discovery, a remarkably short time.
9:00-9:30 Crizotinib: The Xalkori, ALK CDx Partnership
Karen S. Long, Ph.D., Vice President, Medical, Regulatory and Clinical Affairs, Abbott Molecular
Pharmaceutical companies are increasingly engaging diagnostic companies to develop companion diagnostics to help select patients for their therapeutics. There are a number of factors that guide the pharmaceutical company's choice of diagnostic partner including IP considerations, diagnostic platform requirements and commercialization capabilities. However, in view of the relative size and importance of the U.S. market, the ability of the diagnostic company to navigate through the sometimes ambiguous regulatory process in the U.S. becomes a critical consideration, as this is vital to achieve product launch timing objectives and overall success of the therapeutic. There have been some recent examples of rapid co-development and approval of therapeutic/diagnostic product combinations in the U.S. including the recent Xalkori/ALK CDx approval. This session explores the factors that were most important in the successful co-development effort, approval and subsequent commercialization of the diagnostic.
9:30-10:30 Coffee Break in the Exhibit Hall with Poster Viewing
10:30-10:45 Advanced Molecular Diagnostics Based on Ultrasensitive RNA in situ Hybridization
Yuling Luo, Ph.D., Founder, President & CEO, Advanced Cell Diagnostics, Inc.
RNA biomarkers are traditionally analyzed by "grind-and-bind" assays such as RT-PCR, which loses critical cellular context for clinical interpretation. Recent advances in in situ RNA analysis capable of detecting single RNA molecules in routine clinical specimens may finally enable more advanced RNA-based diagnostics.
10:45-11:00 Lot Bridging Considerations for Single and Multiplex Immunoassay Kits in Biomarker Studies
Afshin Safavi, Ph.D., Founder & Vice President, BioAnalytical Operations, BioAgilytix Labs
Biomarker analysis has become a common practice by many pharmaceutical and biotechnology companies to help PK/PD modeling. The reliability of outcomes is heavily influenced by the quality of the kits used to support the studies. The goal of this presentation is to increase awareness of the bioanalytical considerations that are involved in bridging immunoassay assay kit lots.
11:00-12:00 Sponsored Presentations(Opportunities Available)
Contact Ilana Quigley at firstname.lastname@example.org or 781-972-5457
12:00-12:30 Utilization of One Platform from Discovery to the Clinic: A Multiplex Approach
Jeremy Bridge-Cook, Ph.D., Senior Vice President, Assay Group, Luminex Corporation
When formulating a biomarker strategy from discovery to commercialization, one ideally would utilize a flexible assay platform that could be applied to a broad array of biomarker types, could cover one or many biomarkers simultaneously, could be transitioned directly into clinical trials, regulatory submissions, and the clinic, and that is widely adopted in the diagnostics marketplace. A platform which meets all these criteria will be presented, with examples from discovery to diagnostics.
12:30-2:00 Enjoy Lunch on Your Own
2:00-2:30 Lessons Learned in the Development of Companion Diagnostics
Theo McCormick, Director, RxDx Services, Management Science Associates, Inc.
Navigating the scientific, medical, regulatory, contractual and political interactions and dynamics between healthcare professionals, the clinical laboratory, drug manufacturers, IVD diagnostic kit manufacturers and health plans are essential for optimal uptake of drug-diagnostic companion products. Those early decisions in the development cycle have complex downstream impact. This session will map out the trouble spots and potential effects of those decisions.
2:30-3:00 Strategies for Companion Diagnostic Development and Commercialization: Perspectives from a Global IVD Company
John F. Beeler, Ph.D., Director, Theranostics and Business Development, bioMerieux
The drug development process is witnessing a paradigm shift in which new therapies need to be tailored to well-defined patient subgroups via a companion diagnostic assay. With several theranostics partnerships in place at bioMerieux, the speaker will address the challenges of co-development programs and discuss strategies to achieve successful commercialization of these IVD products.
3:00-3:30 The Drug/Diagnostic Development Continuum: Does the Ideal Co-Development Scenario Really Exist?
Rosanne Welcher, Ph.D., M.B.A., Vice President, pharmDx Research and Development, Dako North America
The ideal scenario for co-development of a diagnostic and targeted therapeutic is for both parties to engage at an early stage of drug development. The accepted framework for the development cycle is to align IUO assays to phases of the drug clinical program. Despite best efforts, there are numerous examples of diagnostic assays utilized in pivotal clinical trials that have not yet been fully validated or validated on the target indication. What are the strategies employed to "cut in" or post-validate a diagnostic? This presentation will focus on various scenarios that deviate from the ideal, focusing on the risks and benefits to pharma and the diagnostic partner.
3:30-4:00 Combined CLIA and IVD Strategy Accelerates Timing and Mitigates Risk for Companion Diagnostics
Andrew Grupe, Ph.D., Senior Director, Pharmacogenomics, Celera/Quest Diagnostics
Personalized medicines are heralded as the future of drug development. Bringing a targeted medicine to market requires the concerted efforts of a drug developer and a diagnostics partner. There is no one-size-fits-all approach for these partnerships, and often a tailored relationship is forged between the two partners either before or during a pivotal Phase III trial. Late stage partnerships increase the drug candidate's risk profile when it is deemed to need a predictive biomarker for a pivotal Phase III trial. This presentation will provide specific examples that illustrate the benefits of working with a diagnostics organization with both experienced IVD manufacturing and an extensive CLIA laboratory infrastructure. The tangible benefits that mitigate the drug development risk and may be attractive to both partners if the relationship starts before Phase II will be described.
4:00-5:00 Refreshment Break in the Exhibit Hall with Poster Viewing
5:00-5:30 Pathologists, Participatory Medicine and the Third Wave of Medical Genomics
Mark S. Boguski, M.D., Ph.D., Associate Professor, Center for Biomedical Informatics, Harvard Medical School; Founder, Genome Health Solutions, Inc.
The first two waves of medical genomics focused on therapeutics and pre-symptomatic testing for disease risk assessment. These waves were largely within the purview of the pharmaceutical industry and primary care and public health communities, respectively. The Third Wave focuses on post-symptomatic, precision diagnostics for individualized and optimized disease management. The Third Wave is rising around laboratory physicians and empowered patients working together in a cooperative model of healthcare and outcomes research. Genomics for precision medicine and social networking tools for health communication are key technology enablers of the Third Wave.
5:30-6:00 Use of Next-Generation Sequencing in Clinical Development: The Future Is Here
Premal Shah, Ph.D., Director, Business Development, Genomic Health, Inc.
The use of next-generation sequencing promises to change the drug development paradigm—particularly in oncology. As prices for sequencing drop faster than a Moore's law effect, the real challenge will be to process all the data, ask the right questions, and share information throughout the organization. Just last year it seemed the use of NGS in the clinic seemed 3-5 years away. Not so. The power of NGS combined with falling prices provides an opportunity for biopharma companies to quickly move into this space, gaining a competitive advantage and most importantly, developing targeted therapeutics that will help patients.
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Wednesday, May 23
7:30-8:15 am Breakfast Presentation (Opportunity Available)
Contact Ilana Quigley at email@example.com or 781-972-5457
8:25-8:30 Chairperson's Opening Remarks
8:30-9:00 Clinical Microfluidics: Bioengineering and Clinical Applications of the Circulating Tumor Cell Chip (CTC-Chip)
Shannon Stott, Ph.D., Research Fellow, Surgery, Massachusetts General Hospital, Harvard Medical School
This presentation will describe the engineering design and clinical validation of a high-throughput microfluidic mixing device, the "CTC-chip," that allows the isolation and characterization of CTCs from the peripheral blood of cancer patients. The chip design was centered on the concept of passive mixing of blood through the generation of microvortices, ultimately improving the capture of rare cells by dramatically increasing the number of interactions between the target CTCs and the antibody-coated substrate. Multiparameter characterization was conducted on CTCs to help better understand their origin and metastatic potential.
9:00-9:30 CellSearch®: Prelude to an Information Rich FutureMark C. Connelly, Ph.D., Scientific Director, Cellular Research, Janssen Oncology Biomarkers & Site Director, Veridex• Technology development for rare cell capture and isolation
• Prognostic applications for drug and diagnostic development
• Clinical relevance in breast, prostate and colorectal cancer
9:30-10:00 CTCs: Beyond the Enumeration of EpCam Positive Cells
Marielena Mata, Ph.D., Principal Research Scientist, Oncology Biomarkers, CNTUS, Janssen Research & Development
10:00-11:00 Coffee Break in the Exhibit Hall with Poster Viewing
11:00-11:30 Personalization of Ischemic Disease: Brain and Heart
Jennifer E. Van Eyk, Ph.D., Professor, Medicine, Biomedical Engineering, and Biological Chemistry; Director, Biomarker Development Group, Johns Hopkins University School of Medicine
There is a need to develop circulating biomarkers for assessing an individual's physiological and pathological status. Our initial focus was on the assessment of brain and myocardial ischemia and injury in adult and pediatric clinical settings. This required development of proteomic-based strategies for the robust identification of circulating biomarkers and the large-scale accurate quantification of proteins and/or their disease-induced modified forms using ELISA or targeted multiplex mass spectrometry assays to tease out the individualized response to injury in a large number of clinical settings. This comprehensive approach personalizes the application of biomarkers, enhancing their usability in specific clinical situations.
11:30-12:00 Presentation to be Announced
12:00-1:30 Luncheon on Your Own
1:30-2:00 Discovery of Pharmacogenomic Biomarkers in Cardiovascular and CNS Disorders through Expression Genomics
Wolfgang Sadee, Dr.rer.nat., Professor and Chair, Pharmacology; Director, Program in Pharmacogenomics, The Ohio State University
Genetic biomarkers have potential clinical utility for predicting disease risk and outcomes, including response to therapy. Pharmacogenomic biomarkers are rapidly gaining acceptance in drug discovery, development and therapy; however, a substantial portion of the responsible genetic factors is still uncertain ("missing heritability"). Available evidence suggests that genetic variants affecting expression regulation and RNA processing/translation may account for a large portion of genetically-determined phenotypic variability. We have developed comprehensive assays to detect regulatory variants, recently including the use of second-generation sequencing. Results have revealed the existence of frequent genetic variants with strong influence on clinical phenotypes, including drug response, even in important pharmaco-genes that had been under intense study for some time (e.g., CYP3A4, DRD2, DAT, HTR2A, CETP, ACE, NAT1). Several of these variants have promise as biomarkers for drug therapy, possibly as companion diagnostics in specific cases.
2:00-2:30 Improving Efficacy and Reliability for Personalized Medicine: The Role of Companion Diagnostics in Drug Development
Amelia Warner, Ph.D., Director, Clinical Pharmacogenomics, Merck
Identification of subpopulations of patients who either have subclinical response or toxicity to a drug in clinical development is a challenge. Historically, identification of patients who have less than optimal response to drugs has occurred late in large global clinical trial review. However, the pharmaceutical industry is working to optimize preclinical models, known genetic variation across populations, and earlier inclusion of surrogate biomarkers in early development programs to allow for early identification of populations that will benefit from alternate dosing or alternate therapies. Adapting development strategies to include companion diagnostics remains a challenge, but pharmaceutical companies are developing clear working models to enable development of personalized therapies.
2:30-3:00 Applying Pharmacogenomics Effectively in Psychiatry and How to Overcome the Hurdles
Nadine Cohen, Ph.D., Senior Director, Head of Pharmacogenomics and Biomarker Execution Leader, Neuroscience Biomarkers, Janssen Pharmaceuticals Companies of Johnson & Johnson
An overview of pharmacogenomics applications at Janssen Neurosciences in Psychiatry will be discussed. Learn about using genomics to redefine mental disorders, managing complexity in psychiatric diseases with heterogeneity in disease biology and treatment response, and identifying genetic markers of clinical utility associated with treatment response for psychiatric drugs.
3:00 Close of Conference