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
Stafford O'Kelly, M.B.A., President, 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 iquigley@healthtech.com 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.
• • •
Wednesday, May 23
7:30-8:15 am Breakfast Presentation (Opportunity Available)
Contact Ilana Quigley at iquigley@healthtech.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 Sponsored Presentation
Speaker to be Announced
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 Presentation Advanced Single Molecular Detection: Accelerating Biomarker Development through
Ultrasensitive Immunoassay Technology
Lynn Zieske, Ph.D., Vice President, Commercial Solutions,
Singulex, Inc.
Biomarker verification and validation programs are in need of
sensitive detection technologies to provide precise biomarker measurements in
clinically relevant samples. To address this critical need, the patented
Erenna® Immunoassay system from Singulex offers sub-picogram per mL resolution
at an improvement of 1-3 fold over standard ELISAs. Here we present case
studies demonstrating how the use of the Erenna Immunoassay System has provided
critical insights toward improving the clinical utility of biomarkers.
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 Utilization of Biomarkers in Development of
Treatments for Heterogeneous Immunologic Diseases
Carrie Brodmerkel, Ph.D., Director, Immunology Biomarkers,
Centocor Research & Development
Ulcerative colitis (UC) and Crohn's disease (CD) are
heterogeneous diseases that impact the gastrointestinal system. While anti-TNF
therapy is effective in both diseases, there remains a significant population
that does not derive benefit. Understanding the molecular signature of response
to anti-TNF treatment can identify the core pathway modulation required to
achieve clinical response while understanding the non-response signature can
aid in identifying novel pathways which may be driving disease in anti-TNF
non-responders. Here we present molecular cross-comparison of UC and CD as well
as the effects of biologic therapies on the dysregulated pathways. These
results offer insight into the patient subpopulations most likely to benefit
from therapy.
3:00 Close of Conference