Cambridge Healthtech Institute’s Sixth Annual

Biomarkers for Patient Selection

Translating Biomarkers into Companion and Complementary Diagnostics and Enabling Precision Medicine

June 12-13, 2018 | Westin Boston Waterfront | Boston, MA

Drug discovery is a long, costly process with a high attrition rate, and a personalized medicine strategy is needed to improve clinical outcomes. Identifying the subclass of patients who is likely to respond to drugs and developing biomarkers for patient stratification before recruitment for clinical trials would improve efficacy and response rates. Cambridge Healthtech Institute’s Sixth Annual Biomarkers for Patient Selection meeting will explore the integration of biomarkers and companion diagnostics into drug development, case studies of using biomarkers to guide clinical decision making, molecular profiling approaches and their impact on clinical development, as well as personalized medicine initiatives at big pharma.

Final Agenda

Tuesday, June 12

1:00 pm Conference Registration



1:55 Chairperson’s Opening Remarks

David B. Roth, MD, PhD, Simon Flexner Professor and Chair, Department of Pathology and Laboratory Medicine Director, Penn Center for Precision Medicine

2:00 Leveraging Pharmacogenetics in Drug Development

Peter M. Shaw, PhD, Head, Clinical Pharmacogenetics, Merck

This presentation will focus on routine collection of samples and generating genetic data “in life” from clinical studies to inform on drug development strategy and new targets. Case examples will be provided on improving benefit-risk considerations by identifying response variants, and understanding the contribution of ADME PGx in development programs.

2:30 Genomic and Proteomic Evaluation of Migraine

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

This talk will describe exploratory biomarker screening activities in migraine using both large-scale genomic and proteomic approaches. The challenges and methods for sampling, data generation and analysis will be reviewed and preliminary data analyses will be discussed.

3:00 Corgenix - Your Partner for Liquid Biopsy Protein Diagnostics

Dawn McHugh, Vice President, Business Development, Personalized Diagnostics, Corgenix, Inc.

Corgenix has more than 25 years developing Liquid Biopsy Protein Diagnostics. We offer our expertise and services to Pharma to support their Personalized Medicine strategies. If you are looking to convert your soluble protein biomarker to an IVD or CDx, look to Corgenix.

3:30 Refreshment Break in the Exhibit Hall. Last chance for poster viewing.

4:25 Chairperson’s Remarks

David B. Roth, MD, PhD, Simon Fexner Professor and Chair, Department of Pathology and Laboratory Medicine Director, Penn Center for Precision Medicine

4:30 Putting Precision Medicine into Clinical Practice

David B. Roth, MD, PhD, Simon Flexner Professor and Chair, Department of Pathology and Laboratory Medicine Director, Penn Center for Precision Medicine

At Penn we have focused on bringing promising new scientific developments in precision medicine to clinical practice, in oncology and other areas. The talk will cover the latest developments in several areas of our work in precision medicine.

5:00 Expanding the Reach of Precision Medicine in Non-Small Cell Lung Cancer

Bruce E. Johnson, MD, Professor of Medicine, Harvard Medical School

Fourteen years have passed since the discovery of the epidermal growth factor receptor (EGFR) mutation and its sensitivity to EGFR-tyrosine kinase inhibitors (TKIs) in approximately 15% of patients with NSCLC. The 2007 discovery of ALK rearrangements in 5% of patients with NSCLC led to both EGFR mutants and ALK rearranged NSCLC being treated with 4 different approved EGFR and ALK inhibitors. ROS1 rearrangements and BRAF V600E mutations have also been recently discovered, each representing 1% of lung cancer. Therefore, currently more than 20% of NSCLC can be treated with targeted agents.

5:30Cut-Point Signatures for Patient Selection and Precision Medicine

Viswanath Devanarayan, PhD, Global Head of Statistics & Data Sciences, Charles River Laboratories

The underlying relationship between a disease/clinical outcome and candidate predictors such as biomarkers, demographics and baseline clinical status is usually unknown and must be deduced empirically from experimental data. Such relationships enable the development of prognostic and predictive biomarker signatures for the implementation of optimal patient selection and precision medicine strategy in clinical trials. For easier implementation in practice, having such multivariate signatures in terms of cut-points on predictors is preferable. In this talk, we will review our recently published algorithms for developing such signatures. Results from simulations and case study illustration will be provided.

6:30-9:30 Dinner Short Course*

SC3: Multiplexed Analysis of Tumor Tissues Using Spatially Resolved and Quantitative Platforms

*Separate registration required

Wednesday, June 13

7:25 am Interactive Breakout Discussion Groups with Continental Breakfast

This session features discussion groups that are led by a moderator who ensures focused conversations around the key issues listed. Attendees choose to join a specific group, and the small, informal setting facilitates sharing of ideas and active networking. Details on the topics and moderators are available on the conference website.


8:25 Chairperson’s Remarks

Susanne K. Rhoades, PhD, Director, Diagnostics Development, Tailored Therapeutics, Eli Lilly and Company

8:30 Follow-on CDx Application in Clinical Practice: Is There a Gap?

Cecilia Schott, PharmD, MBA, Former Vice President, Precision Medicine, AstraZeneca Oncology Business Unit

9:00 From Clinical Trial Assays to IVD Companion Diagnostics: Lessons Learned from Bridging Studies

Christopher Major, PhD, Scientific Director, Diagnostic Development, Janssen

Commercial-ready companion diagnostic assays are not available to initiate potential registrational therapeutic trials. As such, it is often necessary to initiate registrational therapeutic trials using a prototype Clinical Trial Assay (CTA), followed by a migration to commercial-ready companion diagnostic assay, via a bridging study. This session will present Janssen’s recent experience in bridging from CTA’s to companion diagnostics for global submission.

Advanced Cell Diagnostic - small logo9:30 Expanding CDx Strategy Options with RNAscope®, a Clinically-Validated, Quantitative in situ RNA Biomarker Platform

Christopher Bunker, PhD, Vice President, Business Development, Advanced Cell Diagnostics

RNAscope is a clinically validated in situ hybridization assay that enables multiplexed tissue expression analysis in routine clinical biopsy tissues. RNAscope is the most sensitive and specific platform for tissue-based expression analysis. It is enabling faster, robust and wide dynamic range tissue-based biomarker expression analysis for accurate correlation to treatment outcome and selection of responsive patient for clinical trial enrollment and CDx.

10:00 Networking Coffee Break

10:30 Qualification of a Biomarker for Patient Selection – Opportunity and Challenges

Abdel B. Halim, PharmD, PhD, DABCC, FAACC, Vice President, Translational Medicine, Biomarkers & Diagnostics, Celldex Therapeutics

Incredibly high failure rate in the pharmaceutical industry has been positioning biomarkers and personalized medicine in the frontline as optimistic rescuers. Successful development and implementation of biomarkers and companion diagnostic strategies will likely mark the difference between winners and losers in this crowded space. To achieve this ambitious goal, some prerequisites should be fulfilled, principally, embracing an effective biomarker strategy as early as possible during the drug development phase and implementation of the right processes.

11:00 Improving Outcomes in Auto-Immune Disease: Progress toward Prediction and Prognosis

Mark E. Curran, PhD, Vice President and Head of Companion Diagnostics, Janssen Immunology

Auto-immune disease including rheumatoid arthritis and inflammatory bowel disease dramatically impacts quality of life for patients. Despite advances in treatment there remains a significant unmet clinical need for new therapies, companion diagnostics and integrated treatment solutions. Our team is focused on transforming treatment of these diseases by applying systems pharmacology, precision medicine and digital health to create new treatment paradigms. Progress toward these objectives will be discussed.

11:30 Selecting Patients Using Investigational Companion/Complementary Diagnostics in Clinical Trials

Susanne K. Rhoades, PhD, Director, Diagnostics Development, Tailored Therapeutics, Eli Lilly and Company

Incorporating an investigational in vitro diagnostic that has potential to be a companion or complementary diagnostic into a clinical trial for a therapeutic product adds complexities and unique aspects that must be carefully considered. These considerations and impact on study initiation activities and clinical trial implementation will be discussed.

12:00 pm Luncheon Presentation (Sponsorship Opportunity Available) or Enjoy Lunch on Your Own



1:25 Chairperson’s Remarks

Jennifer J.D. Morrissette, PhD, Scientific Director, Clinical Cancer Cytogenetics; Clinical Director, Center for Personalized Diagnostics, University of Pennsylvania

1:30 Somatic Mutation Testing: Beyond NGS

Gregory Tsongalis, PhD, Professor, Pathology & Laboratory Medicine, Dartmouth College

Precision medicine has impacted cancer patient management by allowing for more tailored therapeutic strategies to be selected based on the tumor cell genotype. More often, laboratories use next-generation sequencing (NGS) to obtain a molecular profile for use in this therapeutic selection process. Here we will discuss the limitations of NGS and alternative technical strategies to obtaining actionable mutation data.

2:00 Association of Cytogenetic Risk Categories with Functional Categories of Mutations in AML

Jennifer J.D. Morrissette, PhD, Scientific Director, Clinical Cancer Cytogenetics; Clinical Director, Center for Personalized Diagnostics, University of Pennsylvania

We have studied the diagnostic specimens of over 350 patients with acute myeloid leukemia (AML) that have had routine cytogenetic studies and were sequenced using the hematological-NGS panel. Analysis of the data shows that there are different mutational profiles based on the functional genetic categories in patients with different cytogenetic profiles. The strategies proposed in this talk will inform the audience about the functional categories of mutations associated with different cytogenetic abnormalities at diagnosis in AML.

2:30 Genome-Wide Somatic Copy Number Alteration Assessment in Diagnosing and Treating Cancer

Joel Lefferts, PhD, Assistant Professor of Pathology; Assistant Director, Clinical Genomics and Advanced Technology (CGAT), Department of Pathology and Laboratory Medicine, Geisel School of Medicine at Dartmouth, Dartmouth-Hitchcock Medical Center

Gene amplifications and other copy number alterations (CNAs) are well-established biomarkers in oncology that can be invaluable in arriving at a definitive diagnosis as well as in determining appropriate drug targets for cancer patients. In the clinical setting testing for CNAs is most common for specific targets in specific tumor types but the use of comprehensive, genome-wide CNA detection by a variety of techniques is growing. This CNA data is used in differentiating related tumor types at diagnosis and can also provide information regarding dysregulated pathways that may predict response to targeted therapies that are currently available as well as those in development or in clinical trials.

3:00 Close of Conference

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