Companion Diagnostics and Clinical Biomarkers

Clinical biomarkers and companion diagnostics deliver significant patient benefits, healthcare cost savings, and revenue opportunities while providing a foundation for precision medicine. Pharmaceutical companies are embracing biomarkers as a way to decrease drug failures in the clinic by streamlining patient selection and stratification. The need for open discussion between pharma and diagnostics partners to find common ground, address strategy and technology issues, and form sustainable partnerships is at an all-time high. Cambridge Healthtech Institute’s Companion Diagnostics and Clinical Biomarkers conference program is designed to bring together major stakeholders in the field of drug-diagnostics co-development to foster successful science and business strategies in the rapidly changing healthcare environment.

Final Agenda

Day 1 | Day 2 | Day 3 | Download Brochure

Sunday, March 1

2:00 - 5:00 pm Afternoon Short Courses*

*Separate registration required

5:30 - 8:30 Dinner Short Courses*

*Separate registration required

Monday, March 2

8:00 - 11:00 am Morning Short Courses*

*Separate registration required

10:30 Conference Program Registration Open

11:45 Organizer’s Opening Remarks

Marina Filshtinsky, MD, Executive Director, Conferences, Cambridge Healthtech Institute

11:50 Chairperson’s Remarks

Cecilia Schott, PharmD, MBA, Head, Precision Medicine Strategy, Oncology Business Unit, Novartis

11:55 From Exploratory Biomarker to Companion Diagnostics

Molinero_Luciana Luciana Molinero, PhD, Senior Scientist, Oncology Biomarker Development, Genentech

The presentation will present the opportunities and challenges in developing impactful biomarker strategies for early clinical development of new immuno-oncology drugs. We will focus on the design of fit-for-purpose entry into human clinical trials, proof or dis-proof of mechanism of action, and optimal biological dose finding.

12:25 pm NEW: Emerging Trends and Technologies in Companion Diagnostics

Green_George George Green, PhD, Executive Director, Head, Pharmacodiagnostics, Bristol-Myers Squibb

Patient selection strategies, technology, and understanding of disease are evolving. For example, tumor mutational burden and gene expression profiling have not yet met early expectations, and combination strategies and monitoring in earlier indications appear promising. Sequencing is becoming standard, and use of plasma may improve its utility. Additionally, technological advancements such as digital imaging, artificial intelligence, and single cell sequencing are approaching practical use. Here, we survey these emerging trends and perspectives in companion diagnostics.

Presentation delivered via a live, interactive video conferencing platform.

12:55 Session Break

1:05 LUNCHEON PRESENTATION I: Streamlined CDx® - A Proven Strategy to Accelerate Drug Approvals Worldwide

Gerhold_Jason Jason Gerhold, Global Director, Regulatory Affairs and Quality Assurance, Invivoscribe, Inc.

1:35 LUNCHEON PRESENTATION II: Trends in Biomarker Performance and Selection in Immunotherapy

Roberts_John John Roberts, President, Interpace Pharma Solutions

With the approvals of the immune checkpoint inhibitors, there has been an explosion in the use of PD1/PD-L1 assays as a predictive biomarker for drug response. However, real-world evidence shows that this biomarker’s ability to predict response can be limited. We will review some of the published data on the clinical performance of this and other immunotherapy biomarkers (tumor mutation burden, gene expression profiling, etc.) and what future trends are unfolding in this space.

2:05 Session Break

2:20 Chairperson’s Remarks

Scott Patterson, PhD, Vice President, Biomarker Sciences, Gilead Sciences

2:25 How Clinical Practice Is Shaping the Precision Medicine Ecosystem

Schott_Cecilia Cecilia Schott, PharmD, MBA, Head, Precision Medicine Strategy, Oncology Business Unit, Novartis

Pharma companies continue to build and invest in targeted therapy pipeline. New diagnostic platforms are being developed to identify the patient most likely to respond to a given treatment. There is an ever-growing need to understand the precision medicine landscape through the eyes of the practicing clinician. Geographical differences in the access to various testing modalities and reimbursement must be accounted for in clinical development programs and go-to-market strategies. This presentation will provide an overview of the considerations for the global development and lifecycle management of patient diagnostic and monitoring tools.

2:55 Optimization of Biospecimen Selection and Processing for Successful NGS Outcomes

Cathie G. Miller, PhD, Director, Product Marketing, Marketing, BioIVT

FFPE tissues, drug discovery workhorses, have obstacles when used in downstream applications. For 10 years, we have selected and processed specimens from BioIVT’s ASTERAND® Repository. We present case studies representing use-case scenarios: 1) Maximizing specific annotated specimens; 2) Maximizing annotated specimens across a cohort and 3) Replicating clinical validation cohort.

3:10 Using Prototype Comp Dx IHC Assays to Guide Clinical and Business Decisions

Frank Lynch, PhD, Executive Vice President, IHC Services, Discovery Life Sciences (legacy QualTek Molecular Laboratories)

Pursuing a Drug-Diagnostics co-development approach is a monumental decision for biopharmaceutical companies. This presentation will look at considerations to strategically leverage Prototype Companion Dx IHC assays to assist in making the decision and investment to pursue a Companion Dx or not.

3:25 PANEL DISCUSSION: Novel Collaborative Business Models in Drug-Diagnostics Co-Development

Moderator: Scott Patterson, PhD, Vice President, Biomarker Sciences, Gilead Sciences

Panelists: Shirin Khambata Ford, PhD, Head, Clinical Biomarkers & Companion Diagnostics, Global Oncology R&D, Daiichi Sankyo Inc.

Marielena Mata, PhD, Director and Diagnostic Lead, Companion Diagnostics, Pfizer

Cecilia Schott, PharmD, MBA, Head, Precision Medicine Strategy, Oncology Business Unit, Novartis

4:25 Refreshment Break and Transition to Plenary Keynote

(please see Keynote pages for details)

4:35 Welcome Remarks

Cindy Crowninshield, RDN, LDN, HHC, Executive Event Director, Cambridge Healthtech Institute

4:45 PLENARY KEYNOTE INTRODUCTION

Thomas Westerling-Bui, PhD, Senior Scientist, Regional Business Development, Aiforia

5:00 PLENARY KEYNOTE PRESENTATION: High-Performance Medicine

Eric Topol, MD, Founder and Director, Scripps Research Translational Institute (SRTI); Author, Deep Medicine: How Artificial Intelligence Can Make Healthcare Human Again

6:00 Grand Opening Reception in the Exhibit Hall with Poster Viewing, Speed Networking, Book Signing, and Meetup Group

7:30 End of Day

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Tuesday, March 3

7:30 am Registration Open and Morning Coffee

8:00 Organizer’s Remarks

Marina Filshtinsky, MD, Executive Director, Conferences, Cambridge Healthtech Institute

8:05 Chairperson’s Remarks

Marielena Mata, PhD, Director and Diagnostic Lead, Companion Diagnostics, Pfizer

8:10 Companion Diagnostics in the Era of Consolidation and Globalization: Multiplexed Biomarkers across Therapeutic Areas and Around the Globe

Perez_Omar Omar Perez, PhD, Head, Precision Medicine and Diagnostics, GSK

This presentation will discuss the need of multiple CDx strategies based on local needs and local capabilities. Examples from Europe and Asia will be discussed.

8:40 Integrating China in Global Clinical Trials with a Companion Diagnostic: Challenges and Opportunities

Mata_Marielena_(Maty) Marielena Mata, PhD, Director and Diagnostic Lead, Companion Diagnostics, Pfizer

Health challenges in China offer big opportunities for pharma. While the unmet need for oncology drugs in the China market represents a large opportunity, conducting the clinical trials needed for registration presents a number of challenges, including changing regulations, restrictions for the exportation of samples, IP requirements, and availability of CROs. We will discuss these challenges and potential solutions.

9:05 The Future of Genomic Studies Must Be Globally Representative

North_Kari Kari North, PhD, Professor, Department of Epidemiology, University of North Carolina at Chapel Hill

The past decade has seen a revolution in human genetics that has empowered population-level investigations into the biology of complex traits. Here I demonstrate the value of diverse, multi-ethnic participants in large-scale genomic studies by providing an overview of strategies to improve global representation in genomics research and highlighting the successes of studies and consortia that have provided unique knowledge.

9:25 Market Access Strategies for Companion Diagnostics Outside the US

Agarwal_Arushi Arushi Agarwal, Director, Personalized Medicine, Health Advances LLC

9:40 Refreshment Break in the Exhibit Hall with Poster Viewing, Speed Networking, Book Signing, and Meetup Group

10:40 Emerging Companion Diagnostic (CDx) Regulations and Global Differences Influence Regulatory Approval Timelines

Rasmussen_Debra Debra Rasmussen, Senior Director, Global Regulatory Affairs, Janssen Diagnostics LLC

Precision medicine uses approved CDx tests to select patients to receive the right drug, at the right dose, at the right time. Addressing regulations for drug and companion diagnostics has challenges running the gamut from global clinical trial enrollment, to co-approval, to post-market commitments. This presentation focuses on differences between countries and emerging CDx regulations, especially EU IVDR, AP region, and Australia.

11:00 Evolving Regulatory Paradigm on Laboratory Testing in the US and Global Markets – Impact on Personalized Medicine Clinical Trials

Ramamurthy_Lakshman Lakshman Ramamurthy, PhD, Senior Director, Global Regulatory Affairs, Oncology, GSK

The advent of next-generation sequencing has been revolutionary, both for characterizing the genomic nature of cancer, and the rapid accessing of accurate patient tumor genomic information for targeted intervention. As NGS technologies gain acceptance, it has produced the need for consistency, regulatory oversight, and consideration for information privacy. The US and global markets have the challenge of continuing to evolve their oversight systems, while being careful to not stifle innovation. This presentation will compare the US with global markets, including Japan, China, Taiwan, and Brazil.

11:20 Global Commercial and Partnership Considerations for Companion Diagnostics

Ferrara_Joseph Joseph Ferrara, President & CEO, Boston Healthcare

Key commercialization considerations for drug and test innovators, including balancing test access and quality, and activating connections between oncologists, pathologists, payers, and industry stakeholders will be highlighted.

11:35 PANEL DISCUSSION: Harmonizing Technology and Strategies Implementation around the Globe: Opportunities and Challenges from Early Development to Commercialization

Topics to be Discussed:

Tips for working across different cultures to implement successful biomarker strategies
Best practices for global CDx strategies that take regional needs into consideration
What the future looks like for the harmonization of regulatory requirements

Mata_Marielena_(Maty) Moderator: Marielena Mata, PhD, Director and Diagnostic Lead, Companion Diagnostics, Pfizer

Panelists: Hisham Hamadeh, PhD, MBA, Vice President, Global Head, Data Science, GenMab

Omar Perez, PhD, Head, Precision Medicine and Diagnostics, GSK

Lakshman Ramamurthy, PhD, Senior Director, Global Regulatory Affairs, Oncology, GSK

Debra Rasmussen, Senior Director, Global Regulatory Affairs, Janssen Diagnostics LLC

Kate Sasser, PhD, Corporate Vice President, Head, Translational Research, GenMab

Joseph Ferrara, President & CEO, Boston Healthcare

Asuragen 12:20 LUNCHEON PRESENTATION I: Companion Diagnostics: Beyond Oncology to Rare and Complex Genetic Diseases

Andruss_Bernard Bernard Andruss, PhD, Senior Vice President, Operations and Regulatory Affairs Asuragen, Inc.

Recent advances offer new hope for rare and complex genetic disease treatment. Accurately diagnosing, selecting and predicting response in patients require novel technologies and information-rich diagnostic testing strategies. This talk will discuss how a biomarker-driven, diagnostics-enabled approach can facilitate the clinical, regulatory, and commercial success of drugs developed in these disease areas.

1:20 Luncheon Presentation II (Sponsorship Opportunity Available)

1:20 Refreshment Break in the Exhibit Hall with Poster Viewing, Speed Networking, Book Signing, and Meetup Group

2:00 Breakout Discussions in the Exhibit Hall (please click here for details)

3:00 Transition to Keynote Session

3:15 Organizer’s Remarks

Christina Lingham, Executive Director, Conferences and Fellow, Cambridge Healthtech Institute

3:20 Keynote Introduction

Mallory_Allison Allison Mallory, PhD, Director, R&D Molecular Biology, Stilla Technologies

3:35 What Does the New Era of Genomic Medicine Look Like? Effects on Patient Care, Therapeutics, and Diagnostics

20 years after the completion of the first draft of the Human Genome Project, there is compelling evidence of genomics delivering the rich promise of precision medicine. There have been major advances in the throughput and affordability of genome sequencing, enhanced tools for genome analysis and interpretation, new paradigms for therapeutics and strong signs of clinical benefit using genome editing. But major challenges remain. In this special plenary roundtable, three established pioneers of genomic medicine – David Haussler, Stephen Kingsmore, and Liz Worthey – offer their insights on the extraordinary advances in genomic medicine over the past 1-2 decades and share their hopes and concerns for the future of our field.

Davies_Kevin Moderator: Kevin Davies, PhD, Executive Editor, The CRISPR Journal, Mary Ann Liebert, Inc.

Kingsmore_Stephen Panelists: Stephen Kingsmore, MD, DSc, President/CEO, Rady Children’s Institute for Genomic Medicine

Haussler_David David Haussler, PhD, Investigator, Howard Hughes Medical Institute; Distinguished Professor, Biomolecular Engineering, University of California, Santa Cruz; Scientific Director, UC Santa Cruz Genomics Institute; Scientific Co-Director, California Institute for Quantitative Biosciences (QB3)

Worthey_Liz Elizabeth Worthey, PhD, Director, Genomic Medicine, University of Alabama, Birmingham School of Medicine

4:50 Spring Fling Celebration in the Exhibit Hall with Poster Viewing, Speed Networking, Book Signing, and Meetup Group

6:00 End of Day

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6:30 - 9:30 Dinner Short Courses*

*Separate registration required

Wednesday, March 4

6:45 am Registration Open

7:00 BREAKFAST PANEL DISCUSSION: The Time is NOW: Creating Meaningful Change for Women in the Workplace (Sponsorship Opportunity Available)

(please see Women in Science page for details)

Toft_Robin Moderator: Robin Toft, Author of WE CAN, The Executive Woman’s Guide to Career Advancement; Founder and Chairman, Toft Group Executive Search

Samuels_Camille Panelists: Camille Samuels, MBA, Partner, Venrock

Hastings_Paul Paul Hastings, President and CEO, Nkarta Therapeutics, Inc

Wright_Terry Teresa L. Wright, MD, Staff Physician, Medicine, San Francisco Veterans Administration

8:00 Organizer’s Remarks

Marina Filshtinsky, MD, Executive Director, Conferences, Cambridge Healthtech Institute

8:05 Chairperson’s Remarks

Khosrow R. Shotorbani, President, Executive Director, Project Santa Fe Foundation

8:10 Paradigms for MolDX Coverage

bienwillner Gabriel Bien-Willner, MD, PhD, Medical Director, Moldx, Palmetto GBA

This talk will address the LCD process and how this process is implemented for molecular diagnostic testing by MolDX and its partner MACs. This presentation will review the information that is required for technical assessment and how this relates to a new LCD request or to coverage under an existing LCD. We will review MolDX’s approach to evidence evaluation, including the ACCE model process and chains of evidence.

8:25 Humana’s Perspective on Personalized Medicine

Kristine Bordenave, MD, FACP, Former Corporate Medical Director, Humana

This presentation will explore the impact of coverage of genetic testing on precision medicine, and payment considerations based on these relationships.

8:40 Navigating the Reimbursement Landscape in the Era of Precision Medicine

Doshi_Shivang Shivang Doshi, Director, Boston Healthcare Associates, Inc.

The increasing complexity of cancer care and accelerated approvals of targeted and immuno-oncology therapies has shifted CDx testing from a one test-one drug approach to a next-generation sequencing (NGS)-based multi-gene approach. Despite recent improvements, coverage and reimbursement remains fragmented and challenging for NGS testing.

8:55 PANEL DISCUSSION: Coverage and Reimbursement of Advanced Diagnostics

As healthcare is transitioning from “sick” care to “well” care and shifting from fee-for-service to value-based models, it’s essential that we develop evidence to demonstrate the new value of the laboratory for optimized reimbursement.

Topics to be discussed:

Understand how to define and measure value using appropriate KPIs to help influence reimbursement policy.
Learn ways to survive financially as payment models shift and reimbursement is driven by outcomes and risk with potential upside depending upon the reimbursement model.
Explore key partnership opportunities that are aligned with the changes in healthcare to help shape value.

Shotorbani_Khosrow Moderator: Khosrow R. Shotorbani, President, Executive Director, Project Santa Fe Foundation

bienwillner Panelists: Gabriel Bien-Willner, MD, PhD, Medical Director, Moldx, Palmetto GBA

Bordenave_Kristine Kristine Bordenave, MD, FACP, Former Corporate Medical Director, Humana

Castle_Lon Lon Castle, CMO, Molecular Genetics and Personalized Medicine, eviCore Healthcare

Doshi_Shivang Shivang Doshi, Director, Boston Healthcare Associates, Inc.

9:40 Refreshment Break in the Exhibit Hall with Poster Viewing, Speed Networking, Book Signing, and Meetup Group

10:40 The Wide World of Liquid Biopsy-Based Biomarker for Cancer Immunotherapy

Sam Hanash, MD, PhD, Director, McCombs Institute for Cancer Detection and Treatment, University of Texas MD Anderson Cancer Center

Multiple circulating biomarkers that have the potential to serve as predictive prognostic biomarkers to guide cancer immunotherapy are currently being pursued. These include tumor-derived microvesicles, soluble immune proteins, and metabolites. The current opportunities and challenges will be presented.

11:15 NEW: Blood-Based Biomarkers in Immuno-Oncology

Baden_Jonathan Jonathan Baden, MS, Director, Pharmacodiagnostics, Bristol-Myers Squibb

Blood is accessible with minimally invasive and cost-effective methods, so it has always been considered an attractive source of biomarkers. With rapid technological advancements circulating tumor DNA (ctDNA), in particular, has become an invaluable diagnostic material with multiple potential applications across the disease continuum. Here, we review the recent findings on ctDNA to aid in patient selection and disease monitoring from an immuno-oncology perspective, and we discuss potential future directions.

Presentation delivered via a live, interactive video conferencing platform.

11:50 NEW: Characterizing the Bone Marrow Immune Contexture of Acute Myeloid Leukemia Using MultiOmyx^TM

Au_Qingyan-(Sandy) Qingyan (Sandy) Au, PhD, Principal Scientist, Pharma Services, NeoGenomics Laboratories, Inc.

Bone marrow (BM) constitutes the home niche for leukemia cells in AML. Emerging data indicates BM’s crucial role in cancer development. MultiOmyx IF assay was used to characterize the BM immune contexture of AML. Direct assessment of immune phenotypes by MultiOmyx enables understanding of the immune landscape in AML.

12:15 pm NEW: Combining Cell Harvesting and Imaging Technologies for CTC Liquid Biopsy Sample-to-answer Analysis

Anne-Sophie Pailhes-Jimenez, R&D Group Leader, Cell Biology, Imaging, ANGLE Ltd

Alan Schwebel, President, CEO, BioView Ltd

Circulating Tumor Cells provide access to protein and genetic information on patient cancer through a simple blood draw. Here we share the combination of ANGLE’s Parsortix® system for CTC harvesting with BioView’s automated CTC cell imaging and analysis scanner to deliver robust workflow to support the drive towards precision medicine.

12:40 Session Break

12:50 PRECISION HEALTH LUNCHEON PRESENTATION: Validation of A Next Generation Sequencing Gene Panel for Detection of Variants in Plasma Total Nucleic Acid

Tan_Xin-Xing Xin-Xing Tan, PhD, Principal Scientist, Molecular, NeoGenomics Laboratories, Inc.

Liquid biopsy next generation sequencing (NGS) gene panel assays provide a powerful non-invasive tool to detect tumor-derived variants for clinic diagnostics in a massively parallel manner. We present here a NGS assay designed specifically for liquid biopsy clinical applications, and its analytical and clinical validation to assess accuracy, specificity, sensitivity, repeatability, and reproducibility, etc.

1:20 Refreshment Break in the Exhibit Hall with Last Chance Poster Viewing, Speed Networking, Book Signing, and Meetup Group

2:00 Chairperson’s Remarks

Geraldine A. Van der Auwera, PhD, Director of Outreach and Communications, Data Sciences Platform, Broad Institute

2:05 NEW: Fake It ‘til You Make It (Reproducible): Synthetic Data Resources for Genomics

VanderAuwera_Geraldine Geraldine A. Van der Auwera, PhD, Director of Outreach and Communications, Data Sciences Platform, Broad Institute

The computational reproducibility of published biomedical research is limited by data access restrictions, affecting not just researchers who wish to reuse published analysis code, but also tool developers and educators who lack suitable example data for testing and training. We present: 1) a prototype pipeline that wraps established open-source data simulation tools to generate publicly shareable synthetic sequence data at any scale; and 2) a plan to develop community resources.

2:35 Drug Targets with Genomic Support: A Genomics-based Strategic Framework for Improving Target Discovery and Accelerating Drug Development

Justin Wade Davis, PhD, ACOS Research Fellow, Director, Computational Genomics, Genomics Research Center (GRC), AbbVie

Despite strong vetting for disease activity, only 10% of candidate new molecular entities in Ph1 clinical trials are approved. Analyzing historical data, Nelson et al. 2015 concluded pipeline drug targets with human genetic evidence of disease association are twice as likely to lead to approved drugs. We extend this using updated data, test prospectively whether genetic evidence predicts future successes, and introduce statistical models adjusting for target and indication-level properties.

3:05 Close of Conference

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March 11-12, 2025

March 12-13, 2025