Cambridge Healthtech Institute’s 3rd Annual

Immuno-Oncology Biomarkers 2: Predictive Biomarkers and Companion Diagnostics

August 28-29, 2018

As pharmaceutical and biotechnology companies increase their investment in immuno-oncology programs to facilitate rapid development of novel immunotherapies, there is increasing pressure to discover and validate relevant biomarkers. Cambridge Healthtech Institute’s Third Annual Immuno-Oncology Biomarkers 2: Predictive Biomarkers and Companion Diagnostics meeting will bring together biomarkers experts from industry and academia to address rapid development of predictive and prognostic IO biomarkers, utility of these biomarkers in clinical trials, and their potential as companion diagnostics.

Final Agenda


12:00 pm Registration (Harbor Level)

Waterfront 1

1:25 Chairperson’s Opening Remarks

George Poste, DVM, PhD, Chief Scientist, Complex Adaptive Systems, Arizona State University

1:30 FEATURED PRESENTATION: Immunophenotyping to Differentiate Responder and Non-Responder Patients in Cancer Immunotherapy

Poste_GeorgeGeorge Poste, DVM, PhD, Chief Scientist, Complex Adaptive Systems, Arizona State University

The clinical benefits of immune checkpoint inhibitors in a variety of malignancies are unprecedented. Unfortunately, the level of positive therapeutic response is not consistent across different tumor classes and even in responsive tumor lineages non-responders still dominate. The need for comprehensive immunophenotyping to identify the mechanisms underlying these differential responses and better predict responder patients is an urgent clinical and economic imperative.

2:00 Molecular Mechanisms and Biomarkers Predictive of Response to Keytruda

Razvan Cristescu, PhD, Senior Principal Scientist, Merck

The talk will address molecular biomarkers of response to pembrolizumab, including the role of tumor antigenicity, as measured by mutational load (ML) and T cell inflamed microenvironment in predicting the response to pembrolizumab. Data will be presented that prospectively validates the utility of both biomarkers as tumor type agnostic and orthogonal measures of response. These findings provide a biomarker framework for development of pembrolizumab as a monotherapy and for characterizing responses to novel immunotherapy regimens.

2:30 Tissue-Based Diagnostic Tests for Immunotherapy

Rimm_DavidDavid L. Rimm, MD, PhD, Professor, Pathology, Yale

While it would be great to be able to predict response to immunotherapy without a biopsy, to date, other modalities including circulating tests and imaging approaches cannot match the sensitivity and specificity of tissue-based tests. Here we will take a closer look at the predictive value of PD-L1 by immunohistochemistry including the tumor vs. the immune cell components as well as other protein-based tests.

Advanced Cell Diagnostic - small logo3:00 Expanding CDx Strategy Options with RNAscope®, A Clinically-Validated, Quantitative in situ RNA Biomarker Platform

Bunker ChrisChris 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.

3:30 Refreshment Break in the Exhibit Hall with Poster Viewing (Commonwealth Hall)


4:20 CAR-T Therapy for B Cell Malignancies

Jennifer Brogdon, PhD, Director, Exploratory Immuno-Oncology, Novartis


Catherine Young4:55 Walking on the Moon: Reflections on the Work of the Cancer Moonshot and the Future of the Biden Cancer Initiative

Catharine Young, PhD, Senior Director, Science Policy, Biden Cancer Initiative


5:30 Welcome Reception in the Exhibit Hall with Poster Viewing (Commonwealth Hall)

5:30 Dinner Short Course Registration* (Harbor Level)

*Separate registration required.

6:30 End of Day

6:30-9:00 pm Dinner Short Course

SC1: Bioinformatics for Immuno-Oncology and Translational Research

6:30-9:00 pm Dinner Short Course

SC2: Next-Generation Immunotherapies: Part 1


7:30 am Morning Coffee (Harbor Level)

Waterfront 1

8:25 Chairperson’s Remarks

Robert Anders, MD, PhD, Associate Professor, Pathology, Johns Hopkins University

8:30 FEATURED PRESENTATION: Predictive Biomarkers in Colon Cancer and Mismatch Repair Deficient Tumors

Anders_RobertRobert Anders, MD, PhD, Associate Professor, Pathology, Johns Hopkins University

Human cancer tissue samples have been and will be an excellent platform to uncover predictive biomarkers to select a patient for immune based chemotherapy. This lecture will focus on looking back at what biomarkers lead to the success of PD-1/L1 blockade in colon cancer and mismatch repair deficient cancers. Emphasis will be on the lessons that were learned from these clinical trials and how those lessons can be applied moving forward.

9:00 The First Biomarker-Defined Tumor Indication: FDA Approval of Pembrolizumab for MSI-High Cancer

Emancipator_KennethKenneth Emancipator, MD, Executive Medical Director and Head of Companion Diagnostics, Merck & Co.

The program presents an overview of microsatellite instability (MSI) and mismatch repair defect (MMRD), and how it fits into the tumor immunogenicity-inflammation pathway. It reviews the history and clinical evidence for MSI and MMRD as a predictive biomarker for response to pembrolizumab. It discusses the unprecedented – and unorthodox – path to FDA approval of pembrolizumab. Finally, it discusses MSI and MMRD in the broader context of biomarkers in immuno-oncology.

9:30 Future Biomarker-Defined Tumor Indications: Moving Beyond Single Analyte Approaches

Morrison_CarlCarl Morrison, MD, DVM, CMO, OmniSeq

The program presents an algorithmic approach to predicting response to FDA-approved checkpoint inhibitors in multiple tumor types, including melanoma and lung cancer. From PD-L1 expression to T-cell infiltrates (hot vs cold tumors), mutational burden, mutational status, and expression of key immune-related genes we will review how these factors can be combined to improve response prediction in various scenarios and how this may impact clinical use of checkpoint inhibitors.

10:00 Coffee Break in the Exhibit Hall with Poster Viewing (Commonwealth Hall)

10:45 The Return of Precision Medicine in Immuno-Oncology

Su_ZhenZhen Su, MD, MBA, Senior Vice President & CMO, NA, EMD Serono, Inc.

With IO therapies rapidly evolving, the question still remains, which patients will respond? IO has changed the treatment landscape significantly; however, the respective biomarkers and diagnostics do not yet provide a clear direction for clinicians to identify the responsive patient sub-populations. From tumor associated PD-L1 expression, to T-cell infiltrates and mutational burden, we will review the current and future trends as well as discuss the challenges for IO biomarker biology.

11:15 Tumor Mutational Burden Testing for Patient Selection: Implementation of TMB Testing in the Real World

Green_GeorgeGeorge A. Green, IV, PhD, Head, Pharmacodiagnostic Center of Excellence, Bristol-Myers Squibb

Tumor mutational burden (TMB), the frequency of somatic mutations in the tumor, is an emerging biomarker that predicts responses to immunotherapy. Tumors with high TMB may express novel protein sequences leading to the formation of neoantigens, and are believed to make the tumor more visible to the immune system. This may induce tumor-specific T cell responses that can be enhanced by checkpoint inhibitors. TMB is measured using next-generation sequencing, enabling assessment of hundreds of pre-specified genes. Discussion will provide insight into the rationale behind using TMB and practical considerations for its implementation.

11:45 The Cancer Genome’s Influence on Immunotherapy

Riaz_NadeemNadeem Riaz, MD, Associate Director, Immunogenomics and Precision Oncology Platform, Radiation Oncology, Memorial Sloan Kettering Cancer Center

Will review data showing the importance of tumor mutation burden in predicting outcomes to checkpoint blockade therapy. Will subsequently discuss emerging genetic features associated with response to therapy including microsatellite instability, HLA genotype, tumor clonality, and neo-antigen modeling amongst others.

12:15 pm Luncheon Presentation (Sponsorship Opportunity Available) or Enjoy Lunch on Your Own

12:45 Session Break

Waterfront 1

1:40 Chairperson’s Remarks

Emmett Schmidt, PhD, Distinguished Scientist & Executive Director, Merck Research Labs

1:45 Emerging Biomarker Strategies in the Era of PD-1 Checkpoint Combination Therapies

Schmidt_EmmettEmmett Schmidt, PhD, Distinguished Scientist & Executive Director, Merck Research Labs

PD-1 checkpoint inhibition demonstrates unusually broad monotherapy activity across tumor types. Addressing the unmet medical need of patients not responding to checkpoint monotherapy can be achieved by finding highly effective combination therapies, by finding predictive biomarkers or a combination of both strategies. Biomarker strategies will need to evolve to accommodate the emerging landscape of broadly active PD-1 combination therapies.

2:15 Taking Immune-Oncology beyond Checkpoint Blockade

Pelletier_MarcMarc Pelletier, MSc, Investigator III, Translational Immune Oncology, Novartis Institutes for Biomedical Research

Cancer treatment has been revolutionized by the success of checkpoint blockade in the clinic. In order to expand the pool of responsive patients, clinical trials are focused on combining checkpoint blockade with other immune modulating mechanisms. Critical to this success is the elucidation of biomarkers that presage response or highlight potential therapeutic combinations. TGFβ antagonism offers potential combination benefit and biomarker assessment for clinical development of this target will be discussed.

2:45 Application of Next Generation Sequencing to Biomarkers in Immunoncology

Brohawn_ZachZach Brohawn, Associate Director, Research & Development, MedImmune

Advances in the field of next generation sequencing have expanded the applications across multiple therapeutic areas, including a rapid expansion of applications in the field of immunoncology. Deployment of these applications with respect to clinical trials and drug development will be discussed as they relate to biomarker discovery and potential companion diagnostics with examples of practical implementation.

3:15 Refreshment Break in the Exhibit Hall with Poster Viewing (Commonwealth Hall)

4:00 Biomarkers to Assess Efficacy and Toxicity of Immune Checkpoint Immunotherapies in Patients with Melanoma

Philip Friedlander, MD, PhD, Assistant Professor, Medicine, Hematology & Medical Oncology; Director, Melanoma Medical Oncology Program, Icahn School of Medicine at Mount Sinai

The use of anti-CTLA-4 and anti-PD-1 inhibitors to treat stage IV melanoma confers survival benefit. Limitations include the development of response in a subset of patients and the development of immune mediated toxicity. Identifying biomarkers’ predicting response or toxicity can optimize treatment choice and minimize toxicity risk. Biomarkers may reflect tumor or blood based characteristics. The identification and validation of whole-blood RNA transcript based gene signatures is a potential strategy.

 4:30 Prognostic and Predictive Markers for Immunotherapy and Combination Therapy

Mahoney_KathleenKathleen M. Mahoney, MD, PhD, Clinical Instructor, Beth Israel Deaconess Medical Center; Research Fellow, Dana-Farber Cancer Institute

5:30 Dinner Short Course Registration* (Harbor Level)

*Separate registration required.

5:00 Close of Immuno-Oncology Biomarkers 2: Predictive Biomarkers and Companion Diagnostics

6:00-9:00 pm Dinner Short Course

SC3: Next-Generation Immunotherapies: Part 2

preliminary Agendas Available