Clinical and Translational Biomarkers in Drug Development Header

The promise of personalized medicine has been driven by the need to accurately predict patient response to therapy while ensuring drug efficacy and safety. Reducing costs and the time required for drug development is also a driving force in the use of biomarkers. Cambridge Healthtech Institute’s Seventh Annual Clinical and Translational Biomarkers in Drug Development meeting will cover novel biomarker discovery, clinical and analytical biomarker validation, and the role of biomarkers in clinical decision making.

Final Agenda

Monday, May 1

12:00 - 5:00 pm Short Course Registration and Conference Pre-Registration

1:00 - 4:00 Short Course*

SC1: Fit-for-Purpose Biomarker Assay Development and Validation

5:00 - 8:00 Dinner Workshop*

SC2: Liquid Biopsy for Immuno-Oncology and Precision Medicine

5:008:00 Dinner Short Course*

SC3: Preparing for Companion Diagnostic Device Studies and Submissions to FDA

*Separate registration required

Tuesday, May 2

7:00 am Conference Registration and Morning Coffee


Companion Diagnostic Development in Immuno-Oncology

8:00 Chairperson’s Opening Remarks

Nicholas C. Dracopoli, Ph.D., Vice President, Oncology Diagnostics, Janssen Research & Development

8:10 Immuno-Oncology Companion Diagnostics Development: A Complex Systems Approach

Lourdes Barrera, Ph.D., Global Capability Director, Diagnostics, AstraZeneca

Development of companion diagnostics for emerging immunotherapies is more complicated because they are not dependent on driver mutations in the drug target. Consequently, we need to develop new biomarker strategies for the development of immunotherapies. During this review, we will give some examples of how a complex systems approach is supporting the development of new biomarkers and potentially companion and complementary diagnostic tests.

8:35 The Evolution of Oncology Companion Diagnostics from Signal Transduction to Immuno-Oncology

Nicholas C. Dracopoli, Ph.D., Vice President, Oncology Diagnostics, Janssen Research & Development

Companion diagnostic (CDx) tests for signal transduction inhibitors measure the activation status of the drug target. CDx tests for immune modulating drugs will be much more complicated and need to measure the immune status of tumors and differentiate those with a suppressed immune response from those with no prior immune response. These new CDx tests will drive the choice of therapeutic intervention with checkpoint inhibitors or alternative approaches to prime a new immune response.

9:00 An Industry Perspective on Clinical Biomarkers and Companion Diagnostics for Checkpoint Inhibitor Therapies

Arnold B. Gelb, M.D., MS, FASCP, FCAP, Senior Director, Global Clinical Biomarkers and Companion Diagnostics, EMD Serono

Ongoing trends in clinical oncology support the value proposition of using a precision medicine approach for patient selection and enrichment strategies when developing immuno-oncology therapeutics. This presentation will review aspects pertinent to checkpoint inhibitor therapies of exploratory analyses of clinical biomarkers to identify predictive/prognostic clinical biomarkers that may lead to co-development of a companion diagnostic or a complementary diagnostic. Examples will be drawn from the current status of approved PD-L1 assays, citing the limitations thereof, and other clinical biomarkers and candidate companion or complementary diagnostics, including characterizations of the tumor microenvironment, immune cell phenotyping, T cell repertoires, IFN-gamma gene signature, neoantigen burden/mutational load, microsatellite instability status, and potentially other “hot topics” such as liquid biopsies.

9:25 Coffee Break in the Exhibit Hall with Poster Viewing


Companion Diagnostic Development in Immuno-Oncology (Cont.)

10:10 Chairperson’s Remarks

Nicholas C. Dracopoli, Ph.D., Vice President, Oncology Diagnostics, Janssen Research & Development

10:15 RNA, DNA or Protein? Or All Three? Development of Multiple Diagnostics Predicting Response to Pembrolizumab

Matt Marton, Ph.D., Director, Genomics and Companion Diagnostics, Translational Biomarkers, Merck

An effective diagnostic strategy for anti-PD1 therapy may require multiple predictive biomarkers that assess the complexity of both tumor biology and the immune system. In addition to PD-L1 protein expression, multiple biomarkers, including gene expression and mutation burden, have been proposed as predictors of response to anti-PD1 therapy. We will discuss analytical performance characteristics of potential diagnostic devices under development, including an RNA-based gene expression device being studied in multiple indications.

10:40 Neoantigen Biomarkers: Have We Touched the Apoapse?

Saumya Pant, Ph.D., Associate Research Director, The Biocon Bristol-Myers Squibb R&D Center


11:05 Enabling Immuno-Oncology Based Development through Image-Based Cell Sorting to Recover Pure Cell Populations from Complex Patient Tumor Tissue Specimens

Farideh Bischoff, Ph.D., Chief Clinical Development Officer, Menarini Silicon Biosystems

Ana Paula Da Silva, Ph.D., Senior Scientist, Menarini Silicon Biosystems

Tumor infiltrating lymphocytes (TILs) are biomarkers that play a critical role in cancer, including differential diagnosis, determination of prognosis, treatment response, and disease progression. However, analysis of gene expression in fresh tissue may not accurately depict the gene profile as it can change aggressively during lymphocyte isolation and RNA extraction. In this presentation, we demonstrate the use of the DEPArray™ platform to isolate pure populations of lymphocytes from fixed mouse tissue for downstream RNA analysis.

  ACD NEW11:35 Enabling Companion Diagnostic Development for Challenging Biomarkers with RNAscope: A Quantitative In Situ RNA Biomarker Platform 

Robert Monroe, M.D., Ph.D., CMO, Advanced Cell Diagnostics, Inc.

Recent advances have made RNA ISH an attractive platform for companion diagnostics. RNA ISH now has the ability to detect RNA expression in automated, chromogenic assays at the single cell level in histological sections, allowing for biomarker assessment by diagnosticians at the light microscope. This presentation will review how RNA ISH addresses various issues with IHC and other CDx platforms and how it is being used in CDx development for a variety of challenging biomarkers.

12:05 Session Break


 Quanterix12:15 pm Luncheon Presentation: Simoa for the Ultra-Sensitive Measurement of Proteins as Biomarkers of Immuno-Oncology Therapeutics

Mark Roskey, Ph.D., Vice President and General Manager, Quanterix

We will describe the use of single molecule arrays (Simoa) to measure proteins that are emerging as important biomarkers for the effectiveness of immuno-oncology therapies. Immune-targeted therapies, e.g., checkpoint inhibitors, have emerged as the next generation approaches to treating cancer.

Clinical Utility of Liquid Biopsy

1:30 Chairperson’s Remarks

George Vasmatzis, Ph.D., Assistant Professor, Laboratory Medicine and Pathology, Co-Director, Biomarker Discovery Program, Mayo Clinic

1:35 Liquid Biopsy – Next-Generation Medical Innovation

Stefan Scherer, M.D., Ph.D., Vice President, Global Head, Correlative Science, Novartis

Predictive biomarkers that can guide treatment decisions have been sought after for a long time to help identify patient sub-populations that are most likely to respond to specific cancer therapies. Cell free DNAs (cfDNAs) are short fragments of DNA present outside of cells, in the circulatory system. Specific non-invasive “liquid biopsy” can provide personalized and complementary information to help with the diagnosis, prognosis, and management of treatment in patients with cancer. In addition, it provides a dynamic management of cancer and has the potential to enable a paradigm shift in the treatment regimen and drug development.

2:00 Application of Liquid Biopsy in Characterization of Patients with Advanced Small Cell Lung Carcinoma

Sunita Badola, MS, Director, Functional Genomics, Takeda

Small cell lung cancer (SCLC) is an aggressive subset of lung cancer associated with poor survival outcome. Large needle biopsies are seldom available for characterization, especially in a refractory and relapsed SCLC (R/R SCLC) setting and there is a need for a readily available, non-invasive way to characterize this highly lethal form of cancer. We undertook a single center prospective study to evaluate feasibility and compare the profile of circulating tumor cells (CTC) and circulating tumor DNA (ctDNA). We decided to profile plasma samples from a Takeda SCLC clinical trial based on the results from these two sample formats in a pilot study. A SCLC plasma-specific next-generation DNA sequencing (NGS) panel was designed and validated before its application onto the clinical samples. Data from this pilot study and validation experiments demonstrates that it is feasible to deploy ctDNA from "liquid biopsies" for characterization of known genetic alterations in SCLC patients; such an approach may ultimately prove invaluable in identifying prognostic, predictive and resistance biomarkers in patients enrolled in prospective clinical trials.

ANGLE 2:25 Antibody Independent Isolation of Rare Cells: Analytical Aspects

Craig Miller, Clinical Studies Director, ANGLE plc

The requirements and challenges for analytical validation of an antibody independent rare cell isolation system will be presented.


 Ultivue2:40 High Definition Multiplexing for Biomarker Discovery 

Louis Levy, Director, Corporate and Business Development, Ultivue

Biomarker discovery in immuno-oncology requires the analysis of multiple protein markers (n>4) with their spatial relationships at an amenable throughput. The scrutiny of the tumor micro-environment demands whole-slide multiplexed images featuring immune and tumor cells. Ultivue's InSituPlex platform fulfills this need with the data reproducibility relevant to CDx.

2:55 Refreshment Break in the Exhibit Hall with Poster Viewing

3:45 Liquid Biopsies in Personalized Medicine in Cancer

Filip Janku, M.D., Ph.D., Assistant Professor, Investigational Cancer Therapeutics, The University of Texas MD Anderson Cancer Center

Assessment of genomic aberrations, which is required for precision medicine, has been challenging because of the difficulties in capturing intratumoral heterogeneity and in real-time assessment of tumors. Recent advances in technology have enabled detection and analysis of cell-free DNA in cancer patients, which provides real-time assessment of tumor evolution. The recent advances in our understanding of the clinical utility of cell-free DNA and the future directions for its use in cancer management will be discussed.

4:10 Quantification of Somatic Chromosomal Rearrangements in Circulating Cell-Free DNA from Ovarian Cancers

George Vasmatzis, Ph.D., Assistant Professor, Laboratory Medicine and Pathology; Co-Director, Biomarker Discovery Program, Mayo Clinic

Our team has developed MPseq, an accurate and inexpensive whole genome sequencing platform that has been used to detect structural variants. MPseq is a combination of a protocol and algorisms that can deliver a detailed description of all DNA rearrangements at almost nucleotide resolution, thus providing the sequence of a patient’s tumor-specific junctions. Such junctions can be subsequently detected in the plasma of these patients using quantitative PCR (qPCR).

4:35 Counterintuitive Observations Made While cfDNA-Watching

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

This presentation will cover: 1) enrichment by baits rather than amplification, 2) sometimes less (uniqueness) is actually more, 3) informatics challenges.

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

5:30 Short Course and ThinkTank Registration


6:00-9:00 pm Dinner Short Course*

SC4: Next-Generation Sequencing as a Clinical Test

6:00-9:00 pm Dinner ThinkTank*

SC5: PD-L1 Assays for Biomarkers and Companion Diagnostics

*Separate registration required

Wednesday, May 3

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


Translational Biomarkers in Drug Development

7:55 Chairperson’s Remarks

Michael E. (Ted) Burczynski , Ph.D., PPM Expert, Director, Personalized & Predictive Medicine, Analytics & Big Data, Teva Pharmaceuticals

8:00 Translational Medicine to Increase the Probability of Success of Clinical Trials

Maria Jure-Kunkel, D.V.M., Ph.D., Director, Immuno-Oncology Translational Medicine, MedImmune

To increase the probability of success of early clinical trials, we employ a translational medicine approach to first understand disease heterogeneity at the molecular level and develop hypotheses about which patients will benefit the most from therapeutics in clinical development. I will present examples from ongoing clinical development programs to illustrate how we apply this translational medicine approach to MedImmune’s pipeline.

Aushon Biosystems8:25 Use of Ultra-Sensitive Multiplex Protein Arrays in Immuno-Oncology Biomarker Development  
Andrew Nixon, Ph.D., MBA, Associate Professor of Medicine and Director of the Duke Phase I Biomarker Laboratory, Molecular Reference Laboratory, Duke University
This study helps us to understand the application of ultra-sensitive protein multiplex array in biomarker development, describe the levels of several key immunologic cytokines across a variety of trials and patients and benchmark to more traditional methodologies, and understand the use of these data to develop novel pharmacodynamics, prognostic and predictive biomarkers.

8:55 From Preclinical Model Mechanisms to Clinical Hypotheses Testing in Huntington’s Disease

Michael E. (Ted) Burczynski , Ph.D., PPM Expert, Director, Personalized & Predictive Medicine, Analytics & Big Data, Teva Pharmaceuticals

The present talk will discuss recent laboratory investigations into, and subsequent modeling of, disease mechanisms in an animal model of Huntington’s disease. The talk will highlight insights gained into the mechanism of action of therapeutic candidate(s), as well as potential patient stratification approaches to be tested in future clinical studies which were informed by a systems-level analysis of the animal disease model and subsequent pathway and informatics approaches to identify relevant human markers to evaluate.

9:20 Improved Monitoring of Tumor Growth with a Novel Serum Proliferation Biomarker

Martin Shaw, Business Development Manager, AroCell AB

The AroCell TK210 ELISA is a novel, sensitive and specific assay for serum Thymidine Kinase 1, a well-known proliferation biomarker. It is the first CE-marked TK1 ELISA. Data will be presented on the value of the AroCell TK210 ELISA in the study of a range of hematological and solid tumors.

Second Genome9:35 The Gut Microbiome as a Biomarker of Disease and Treatment Response 

Take Ogawa, Director, Second Genome, Inc.

Microbiomes are complex communities of microbes that interact with each other and their surrounding environment (e.g. tumors, gut or skin). Understanding which microbes alleviate or exacerbate health outcomes is leading to novel insights for deepening our understanding of disease, uncovering new therapeutic approaches and uncovering predictive biomarkers.

9:50 Coffee Break in the Exhibit Hall with Poster Viewing

10:45 Can We Predict Nephrotoxicity before It Occurs: The Promise of Metabolomic Biomarkers

Lawrence J. Lesko, Ph.D., Clinical Professor, Center for Pharmacometrics and Systems Pharmacology, University of Florida, Lake Nona

Safety, not efficacy, is the single most important reason for project closure in new drug development. Over 50% of failures in early phase development and 30% in mid- to late-phase development are due to unacceptable adverse drug events. Unanticipated renal toxicity accounts for 10% of these adverse events. Metabolomic biomarkers can be used as early reporters of drug-induced acute kidney injury thereby improving upon the use of traditional markers of renal function.

11:10 Identification and Translation of Pharmacodynamic Biomarkers from Bench to Bedside

Tammie Yeh, Ph.D., Associate Director, iMED Oncology Translational Sciences, AstraZeneca

Providing evidence in the clinic that the compound being tested is modulating its target and having the expected effects can be extremely informative during drug development. This information can help with building confidence in the compound, determining optimal dosing/scheduling, or understanding negative efficacy data; ultimately, these data can be useful when programs need to be prioritized. Two examples of pharmacodynamic (PD) biomarkers will be presented. The first is on the identification and validation of a robust PD biomarker for BET/BRD4 inhibitors using preclinical studies. The second is on the generation and interpretation of PD data from paired tumor biopsies from a Phase I “anti PD-L1 + kinase inhibitor” combination trial.

11:35 Enabling Clinical Development of Therapeutics with Greater Confidence: The Use of Pharmacodynamic Biomarkers in Early Stage Clinical Studies to Demonstrate Target Engagement

Mark Matijevic, Associate Director and Head, Translational Biomedicine Lab, Eisai AiM Institute

Perkin Elmer logo small 12:00 pm Advanced and Agile Visual Analytics for Biomarker Discovery 

Eduardo Gonzalez, Ph.D., Product Manager, PerkinElmer Informatics

- Using the cloud for translational data storage and advanced analytics
- Signals for Translational: a platform for Translational medicine
- Extending Spotfire visual analytics with flexible Apps

 12:30 Close of Conference

Health and Safety

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