2019 Archived Content

Cambridge Healthtech’s 5th Annual

Companion Diagnostics and Clinical Biomarkers in Immuno-Oncology

Predicting and Monitoring Response to Cancer Immunotherapy

August 21-22, 2019

Immunotherapy is the most effective therapeutic approach in a variety of advanced and metastatic cancers that the cancer community saw in decades. However, the success of each of the agents is highly dependent on the choice of a specific approach for a specific patient population. Therefore, the progress in adoption of cancer immunotherapy requires the development and implementation of robust clinical-grade biomarkers. Once validated the biomarkers and companion diagnostics for cancer immunotherapy undergo significant market access challenges including regulatory and reimbursement hurdles. Cambridge Healthtech Institute’s Fifth Annual Companion Diagnostics and Clinical Biomarkers in Immuno-Oncology is designed to bring together clinical immuno-oncologists, researchers from pharmaceutical and biotech companies, and members of the laboratory medicine community to discuss challenges and solutions for bringing to the market and implementing robust and cost-effective companion and complementary assays to improve patient outcomes.

Final Agenda

WEDNESDAY, AUGUST 21

10:30 am Registration

Constitution A&B
11:30 Plenary Keynote Session

11:30 Chairperson’s Remarks

Charles Mathews, Principal, ClearView Healthcare Partners

11:40 NEW: Plenary Keynote Presentation: FDA Updates: Now and Looking to the Future

Katherine Donigan, PhD, Acting Director of Personalized Medicine, Office of In Vitro Diagnostics and Radiological Health, Center for Devices and Radiological Health, U.S. Food and Drug Administration

Introduction and background of the new Office Director of OIR and updates on precision medicine and other initiatives at the FDA.

12:10-1:05 pm Plenary Keynote Discussion: Proposals and Solutions for Diagnostic Reform Including Oversight of Laboratory Developed Tests (LDTs)

Co-Organized with

Moderator:
Cynthia A. Bens, Senior Vice President, Public Policy, Personalized Medicine Coalition

How are stakeholders influencing congressional activity on the Verifying Accurate Leading-edge IVCT Development (VALID) Act?
How will the VALID Act change the current oversight landscape for diagnostics, including LDTs?
How are policymakers addressing the role of CMS and CLIA in the VALID Act?
How will increased regulatory and oversight activities at the FDA affect the diagnostics industry?
What impact will changes in diagnostics regulation and oversight have on patient care?

Panelists:

Julie Khani, MPA, President, American Clinical Laboratory Association (ACLA)

Donald E. Horton, Jr., Senior Vice President, Global Government Relations & Public Policy, Laboratory Corporation of America Holdings

Susan Van Meter, Executive Director, AdvaMedDx

Tara Burke, PhD, Senior Director, Public Policy & Advocacy, Association for Molecular Pathology (AMP)

Lasiter_Laura Laura Lasiter, PhD, Science Policy Analyst, Friends of Cancer Research

1:05 pm Luncheon Presentation (Sponsorship Opportunity Available) or Enjoy Lunch on Your Own

1:35 Ice Cream & Cookie Break in the Exhibit Hall with Poster Viewing

2:05 Chairperson’s Opening Remarks

Kenneth Emancipator, MD, DABP, Executive Medical Director, Companion Diagnostics, Translational Medicine, Merck & Co., Inc.

2:10 Biomarkers in the Context of IO Combinations

Alvarez_JD J.D. Alvarez, MD, PhD, Vice President, Oncology Diagnostics, Janssen Pharmaceutical Companies

Will current assays be combined or completely superseded? Are standard clinical biomarkers being overlooked in IO trials? What is the current utility and future potential of blood-based biomarkers? How can NGS panels data be used to improve target discovery and/or combo prioritization? These and other important issues will be addressed in this presentation. Harmonization of multiple assays for the similar biomarker(s) will be discussed as well.

2:40 TMB as a Clinical Biomarker in Combination Trials

Raja_Rajiv Rajiv Raja, PhD, Director, Translational Medicine Oncology and Pharmacogenomics, AstraZeneca

Next Generation Sequencing and Liquid Biopsies offer exciting novel opportunities to bring innovative drugs to patients. But these opportunities do not come without challenges and will need to be resolved, particularly when looking at genomic signatures such as Tumor Mutational Burden.

3:10 PD-L1 as a Companion Diagnostic for Tumors Beyond Non-Small Cell Lung Cancer: It’s the Same Thing, Only Different

Emancipator_Ken Kenneth Emancipator, MD, DABP, Executive Medical Director, Companion Diagnostics, Translational Medicine, Merck & Co., Inc.

The PD-L1 companion diagnostic had a huge impact on the clinical development of pembrolizumab, making it the first immunotherapy approved as a first-line agent for non-small cell lung cancer. However, this is just the beginning of the story, not the end. Adapting the PD-L1 diagnostic to incorporate immune cell expression facilitated approval of pembrolizumab for several additional indications and may shed light on the mechanism of action of checkpoint inhibitors.

3:40 How Biospecimen Sourcing Can Impact Your R&D

Tumilasci_Vanessa Vanessa Tumilasci, PhD, Commercial Director, Trans-Hit Biomarkers

Biospecimen sourcing is becoming a challenge for many scientists who need to respect timelines for R&D plans as well as regulatory and ethical constraints. Are the scientists working with the samples aware of all the imperatives to obtain them; quality, respect of laws, ethics and regulations?

3:55 Sponsored Presentation (Opportunity Available)

4:10 Refreshment Break in the Exhibit Hall with Poster Viewing

4:55 Chairperson’s Remarks

Nicholas Dracopoli, PhD, CSO, DELFI Diagnostics

5:00 Immuno-Oncology Biomarkers: What’s New?

Zhang_Nancy Nancy Zhang, MD, Associate Director, Pharmacodiagnostics, Bristol-Myers Squibb

Precision medicine continues to transform treatment paradigms through development of new biomarkers and interpretation of clinical data. Testing for Immuno-Oncology biomarkers, such as PD-L1, and emerging ones, such as tumor mutational burden, may help optimize treatment decisions when assessed individually or in combination. The advancement of increasingly complex biomarkers also brings a need for advanced diagnostic tools. We will discuss biomarkers, their dynamic monitoring, and assessment in the evolving field of pharmacodiagnostics.

5:30 Companion Diagnostics in Immuno-Oncology

Pathak_Anand Anand Pathak, MD, PhD, MPH, Medical Officer, FDA

This session will focus on the implementation of immuno-oncology diagnostic tests in clinical trials, including understanding the regulatory implications of different approaches, validation of emerging pan-tumor CDx claims, and complementary diagnostics vs. companion diagnostics.

6:00 Panel Discussion: Companion Diagnostics in Immuno-Oncology

Moderator: Nicholas Dracopoli, PhD, CSO, DELFI Diagnostics

Panelists: Speakers of the Day

Topics to be discussed:

Advantages and disadvantages of complex multianalyte panels versus simple single analyte Dx tests
Issues in developing CDx tests for drugs being launched in different countries
Tumor mutation burden (TMB) and other very large panel tests
Advantages and disadvantages of plasma based ctDNA testing compared to tissue-based tests
Emergence of new CDx tests for immuno-oncology drugs

6:30 Close of Day

6:30 Dinner Short Course Registration

6:45 - 9:15 pm Recommended dinner Short Course*

SC14: Generating Evidence and Creating a Winning Dossier for Regulatory and Reimbursement Needs

Mark Hiatt, MD, Vice President, Guardant Health

John Sninsky, PhD, Consultant, Translational Sciences

Managing the clinical narrative for your diagnostic test requires careful evaluation of the evidentiary requirements of stakeholders. Using those evidentiary requirements to determine your clinical and economic study designs is the cornerstone of effective product development.

*Separate registration required.

THURSDAY, AUGUST 22

7:15 am Registration

Independence B-E and Foyer
7:30 Problem Solving Breakout Discussions with Continental Breakfast

Clinical Biomarkers in the Era of Combination Cancer Therapy

Moderator: Jamie Shaw, Principal Scientist, Clinical Biomarkers and Diagnostics, EMD Serono, Inc.

How can clinical sample testing be prioritized to be most informative when tissue is limited?
What are the challenges of assessing pretreatment markers vs. resistance markers acquired upon progression?
Which design parameters are essential for robust evaluation of multiple markers within a clinical study?
What strategic considerations need to be taken when there are multiple CDx assays commercially available?

Next Generation Oncology Biomarkers: Value Demonstration and Role of Real-World Data

Moderators: Shuvayu S. Sen, PhD, Executive Director, CORE Oncology, Merck & Co., Inc.

Kaushal Desai, PhD, Director, Informatics & Outcomes Research, Merck & Co., Inc.

Robert Metcalf, CEO, Concert Genetics

What are the critical questions drug developers face in showing the value of such markers for approval and utilization?
What are the methodological and data challenges do we face in conducting value assessment of biomarkers and relevant drugs?
What are the perspectives of diagnostic labs and data integrators in terms of linking data of different part of the health care system including biomarkers?

8:25 Chairperson’s Remarks

Mitch Raponi, PhD, Vice President, Biomarkers & Translational Research, BeiGene

8:30 Silver Linings: Embracing the Positive and Negative in Predictive Biomarker Development

Shaw_Jamie Jamie Shaw, Principal Scientist, Clinical Biomarkers and Diagnostics at EMD Serono, Inc.

Despite development of countless drugs targeting the PI3K/Akt/mTOR pathway in cancer, no biomarkers have been validated to identify patients likely to benefit from these therapies. This presentation will review findings from the PhI trial of M2698, a p70S6K/AKT inhibitor, which suggest that tumor resistance markers could identify a patient population benefitting from treatment and illustrates how implementing a combination of positive and negative selection markers may increase robustness and predictivity.

9:00 PARPi Development in Prostate Cancer: Limitations and Opportunities for Companion Diagnostic Development

Raponi_Mitch Mitch Raponi, PhD, Vice President, Biomarkers & Translational Research, BeiGene

PARP inhibitors have demonstrated clinical utility in treating metastatic prostate cancer due to enrichment of DNA repair deficiencies in a subset of these tumors. NGS testing of tumor tissue to identify patients with BRCA defects or other homologous recombination repair deficiencies has limited utility due to limited amount of tumor isolated from bone biopsies. Opportunities of developing liquid biopsy-based companion diagnostics for this disease will be discussed.

9:30 Streamlined CDx™ - A Proven Strategy to Accelerate Drug Approvals

Vishnevetsky_Michael Michael Vishnevetsky, PhD, Head of Global Business Development, Invivoscribe

Companion Diagnostics have revolutionized precision medicine as they play a pivotal role in defining the efficacy of targeted therapies. Invivoscribe’s Streamlined CDx™ program has been shown to collapse development timelines, improve and accelerate selection of patient cohorts, leading to earlier submissions and accelerated FDA, EMA and PMDA approvals of new targeted therapies. Streamlined CDx™ partnership model has proven successful in approval of the first ever AML companion diagnostic – The LeukoStrat® CDx FLT3 Mutation Assay.

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

11:00 Chairperson’s Remarks

Iulian Pruteanu-Malinici, Investigator III, Lab Head, Immuno-Oncology, Novartis

11:05 Personalizing Immunotherapy for Each Cancer Patient

Yelensky_Roman Roman Yelensky, MD, Executive Vice President and CTO, Sequencing and Bioinformatics, Gritstone Oncology

Gritstone Oncology is a cancer immunotherapy company working to help patients with the most difficult-to-treat tumors. Gritstone’s personalized immunotherapy process leverages our EDGE AI platform to predict neoantigens that will be presented on a patient’s tumor, allowing us to create a patient-specific heterologous prime-boost immunotherapy that is designed to elicit a potent anti-tumor T cell response.

11:35 Correlative and Release Test Assays for Evaluating Gene Edited Human Cell Products

Lacey_Simon Simon Lacey, PhD, Director, Translational and Correlative Studies Laboratory, Center for Cellular Immunotherapies, University of Pennsylvania

Gene editing techniques such as CRISPR/Cas9 hold potential for augmentation of CAR-T and TCR engineered cell products to be used in human clinical trials. The FDA requires additional evaluations of such gene edited and engineered cells prior to, and post infusion. This presentation will discuss the development and validation of correlative and release test assays to evaluate efficiency of transduction, gene editing and other aspects of engineered and edited cells.

12:05 pm Modeling Flow High-Dimensional Data and Biomarker Identification in a CART/Multiple Myeloma Study

Pruteanu-Malinici_Iulian Iulian Pruteanu-Malinici, Investigator III, Lab Head, Immuno-Oncology, Novartis

Here we present a Flow Cytometry based framework that, when combined with state-of-the-art bioinformatics, enables for the discovery of biomarkers that predict clinical response of CART therapeutic products.

12:35 Sponsored Presentation (Opportunity Available)

1:05 Luncheon Presentation (Sponsorship Opportunity Available) or Enjoy Lunch on Your Own

1:35 Dessert Break in the Exhibit Hall with Poster Viewing

2:15 Chairperson’s Remarks

Kathryn Lang, MBBS, MRCP, Vice President, Outcomes and Evidence, Guardant Health

2:20 TLR Agonist NKTR-262 Immunotherapy Combination with Bempegaldesleukin (NKTR-214) Harnessing Innate and Adaptive Immune System for the Treatment of Solid Tumors

Kivimae_Saul Saul Kivimäe, PhD, Head of Pharmacology, Nektar Therapeutics

NKTR-262 is a novel TLR agonist therapeutic designed to deliver intratumoral TLR7/8 engagement and is currently evaluated in Phase 1 dose escalation study with bempegaldesleukin, a CD122-preferential IL-2 pathway agonist. NKTR-262 combination treatment with bempegaldesleukin is designed to provide a synergistic effect of localized intratumoral innate immune stimulation with systemic sustained T cell activation for comprehensive anti-tumor immune activation mimicking a natural immune response.

2:50 Integrating Clinical Research and Care in a Perpetual Trial to Continuously Learn From All Patients, on All Treatments, All the Time

Newton_Mika Mika Newton, BSc, CEO, xCures

The explosion of molecular subtypes and oncology drugs means R&D is about to hit a wall. There are not enough patients to explore all the potential options. A Perpetual Trial provides patients and their physicians with individualized treatment options. RWD is generated longitudinally to learn what works in which patients, and coordinate treatment prioritizing the most promising therapies. This RWE is used to accelerate regulatory approvals, label expansions, and reimbursement.

3:20 How Can Real-World Data Enhance Clinical Evidence Generation?

Mccusker_Margaret Margaret McCusker, MD, Senior Medical Director, Flatiron Health

As the cancer treatment landscape becomes increasingly complex, biomarkers are playing a more prominent role in drug development and clinical decision support. In this setting, research-quality clinical evidence derived from cancer patients’ electronic health records and paired genomic data are a valuable asset. This session will highlight examples of how clinico-genomic data can provide critical insights into the impact of biomarkers on real-world oncology practice and accelerate clinical research.

3:50 Establishing the Next Generation of Clinico-Genomic Data in Oncology and Healthcare

Lang_Kathryn Kathryn Lang, MBBS, MRCP, Vice President, Outcomes and Evidence, Guardant Health

The emergence of real-world clinico-genomic data promises great value for all aspects of the healthcare system. How do organizations and individuals combine efforts to support all aspects of care and treatment?

4:20 End of Summit