Wednesday, December 6th

8:30 AM

Registration and Breakfast

9:00 AM

Welcome and Logistics

Session 1: Valuing Companion Diagnostics in the Marketplace

Session Moderator: Tom Fare

9:10 AM

The Role of Philanthropy in Accelerating Cancer Precision Medicine

Michele Cleary, Chief Executive Officer

The Mark Foundation for Cancer Research

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The Role of Philanthropy in Accelerating Cancer Precision Medicine

While precision medicine is gaining broader acceptance, critical barriers remain for delivering true precision medicine to cancer patients. Chief among these challenges are gaps in the discovery and development of optimal biomarkers for early detection, predicting response to therapeutics, forecasting resistance to treatment, and diagnosing recurrence.

Even with significant advances on these knowledge fronts, substantial obstacles remain in translating data and platforms to practical clinical applications as well as in educating practitioners on the proper use of precision tools. Unfortunately, high risk and limited prospects for financial return diminish investment in rigorous efforts to develop biomarkers and diagnostics. Such endeavors typically garner robust resourcing only when they are needed to move a blockbuster therapeutic forward commercially.

Consequently, support for translational research tends to fall “between the cracks” of the typical funding mechanisms for basic research and industrial investment. Philanthropic organizations can play an important role in accelerating advances in cancer precision medicine. Most life science philanthropists are inspired by the opportunity to address scientific knowledge gaps and fill research funding deficits. They also aspire to integrate technology innovation with basic science. Moreover, in most cases, they seek better prospects for patients as their primary return on investment.

Achieving a future of practical, actionable, and timely precision medicine for cancer patients is perfectly aligned with the mission and vision of the Mark Foundation for Cancer Research. As we build the organization, we are establishing a portfolio of funded projects that will benefit patients in the nearest term and that are centered around novel technology-driven approaches for gaining insights that have been difficult to achieve with conventional experimental strategies.

To be successful, we will identify critical unmet needs in cancer research, fund highly innovative proposals for solutions to these challenges, create a robust and dynamic network of collaborators who share our passion to deliver breakthroughs to patients, and continually invest in the next generation of revolutionary science.

9:35 AM

Keynote Presentation
Nic Dracopoli
The Evolution of Oncology Companion Diagnostics from Signal Transduction to Immuno­-oncology

Nic Dracopoli, Vice President and Head of Oncology Diagnostics

Janssen Pharmaceuticals

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The Evolution of Oncology Companion Diagnostics from Signal Transduction to Immuno­-oncology

The majority of novel oncology drugs are still approved without a companion diagnostic even though the probability of regulatory approval is much higher for drugs developed with a diagnostic biomarker. This presentation will review the development of companion diagnostics for novel immuno-oncology drugs and describe strategies to release a prior, but suppressed, immune response to a tumor, or to prime a new response in patients with no prior immune response.

10:10 AM

Factors that Affect Impact the Commercial Utilization of Companion Diagnostics

Steve Anderson, Chief Scientific Officer and Senior Vice President

Covance

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Factors that Affect Impact the Commercial Utilization of Companion Diagnostics

There are currently more than 30 companion diagnostic assays (CDx) in clinical use today, with the majority of applications in Oncology. The various CDx assays include examples of immunohistochemistry, single gene mutation assays and recent applications for next generation sequencing based assays. Oncology applications include those linked to a specific targeted therapy along with recently approved applications in the area of immuno-oncology. A number of factors may have a significant impact on the commercial introduction and clinical utilization of specific companion diagnostics. Among these factors are inclusion in clinical practice guidelines, ease of use, broad or general access to the technology and/or specific application, specimen type and requirements, and reimbursement policies. We have evaluated data on the utilization and performance for a variety of companion diagnostics and will report on the most important factors for each of the testing modalities and applications.

10:35 AM

Coffee Break

Session 2: Current issues in Regulatory Affairs

Session Moderator: Bill Pignato

10:50 AM

Regulatory Considerations for US FDA Submissions for Companion Diagnostic Tests

Jeffrey Allard, President

Lakeside Life Sciences

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Regulatory Considerations for US FDA Submissions for Companion Diagnostic Tests

This presentation will explore various aspects of regulatory submissions to the US Food and Drug Administration for Companion Diagnostic tests. We will present non­controversial information to the extent possible and will highlight the more subjective issues. Our objective will be to provide a clear and simple primer on the regulatory requirements surrounding CDx.

We will begin with a discussion of the philosophical approach to working with FDA and will cover topics such as:

  • What does FDA want?
  • Are there formulas for CDx test submissions that we can follow?
  • When and how to ask FDA for guidance?
  • What is the Pre­submission process and does it work?

We will describe the Companion Dx Regulatory Process in detail including:

  • Class I/II/III: Where do CDx fall?
  • How does a PMA differ from a Class II 510(k) or De Novo submission?
  • Major Requirements for a CDx submission
  • What is the Office of Combination Products and what is their role?

Many recent publications have addressed the issue of Companion and Complementary testing. We will demystify these terms and explain:

  • How are these terms distinguished from each other?
  • How do the regulatory classification and requirements differ?
  • Advantages and risks

The regulatory literature is replete with discussions, warnings and predictions on the issue of Laboratory Developed Testing. We will discuss the legality, the ethics and the regulations around this approach with emphasis on:

  • How is an LDT distinguished from an FDA­regulated IVD?
  • What are the risks of offering a test as an LDT?
  • Can a Companion Dx test be offered commercially as an LDT?
  • Current and future status of FDA regulation of LDT testing

11:15 AM

PANEL: Regulatory Considerations for US FDA Submissions for Companion Diagnostic Tests

Chair: Jeff Allard; Panelists: Debra Rasmussen, Julie Engel, Bill Pignato, Anand Pathak, MD, PhD, MPH (FDA, CDRH/OIR),

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PANEL: Regulatory Considerations for US FDA Submissions for Companion Diagnostic Tests

11:55 AM

Networking Lunch; Visit Sponsors; View Posters

1:05 PM

Effective Strategies to Ensure Successful Drug/Diagnostic Co­Development

William Pignato, Founder and Principal

W.J. Pignato & Associates

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Effective Strategies to Ensure Successful Drug/Diagnostic Co­Development

The successful integration of a companion diagnostic strategy into a therapeutics’ clinical development plan are dependent on a number of key factors to ensure concurrent review and approval of the drug and diagnostic. While some issues may be viewed as self­evident, this talk will discuss effective strategies and requirements that companies must consider in order to lead to a successful co­development outcome. The importance of the right technology and partner selection, relationship management and the incorporation of global regulatory affairs considerations will be reviewed.

1:30 PM

Precision Medicine – where do we go from here?

Julie Engel, Director of Precision Medicine and Diagnostics Regulatory Affairs

Celgene

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Precision Medicine – where do we go from here?

At Celgene, we are investigating biomarkers for cancer, solid and hematologic tumors, as well as for chronic and debilitating diseases with the aim toward developing more effective therapies for patients worldwide. The recent approval of IDIFA for the treatment of patients with AML, with a corresponding companion diagnostic, demonstrates Celgene’s commitment to this targeted therapy approach. In this presentation, I will discuss the changing regulatory landscape in the US and how this impacts our approach to drug and diagnostic codevelopment. I will highlight what we have learned from Blue Print, as well as from other recent FDA approvals. I will also touch on changes to the regulatory climate world wide, e.g., the impact of IVDR in EU, China,and the rest of the world and their impact on codevelopment. Where do we go from here?

  • Overview of the changing regulatory landscape in the US and World Wide
    • US – LDT Discussion Paper
    • Short Discussion on the Impact of IVDR/EU Guidance on CDx
    • Short Discussion on CDx/Dx Regulatory Changes ROW
  • Companion vs Complementary Diagnostics
    • What have we learned from PD-L1/Blue Print?
  • Where do we go from here?
    • Single Site CDx Approvals
    • Pan tumor markers
    • NGS and genetic approaches
    • Bioinformatics/Big Data
    • Impact of Clinical Trial Designs Etc.

Session 3: Enabling and Emerging CDx Technology Platforms

Session Moderator: Oscar Puig

1:55 PM

The First Biomarker-Defined Tumor Indication: FDA Approval of Pembrolizumab For Msi-high cancer

Ken Emancipator, Executive Medical Director and Head of Companion Diagnostics

Merck & Co.

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The First Biomarker-Defined Tumor Indication: FDA Approval of Pembrolizumab For Msi-high cancer

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

2:20 PM

Multidrug analyses in cancer patients – a functional approach to precision oncology

Rich Klinghoffer, Chief Scientific Officer

Presage Biosciences

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Multidrug analyses in cancer patients – a functional approach to precision oncology

The vision of a precision medicine-guided approach to novel cancer drug development is challenged by high intra-tumor heterogeneity, interpatient diversity, and complex interactions between tumor cells and the surrounding microenvironment. Most ex vivo platforms do not accurately capture this complexity, hampering predictions of clinical drug efficacy. The era of genomics, highlighted by remarkable advances in high-throughput next generation sequencing technology, has sparked hope that this issue can be solved through a precision medicine approach to cancer treatment: matching the right drugs to the right patients. However, despite early successes, genome-based precision medicine has been limited by an incomplete understanding of the relationship between cancer mutations and drug responsiveness. To overcome these issues, we developed CIVO (Comparative In Vivo Oncology), an arrayed microinjection technology that tests tumor responsiveness to microdoses of multiple drugs simultaneously and directly in a patient’s tumor. CIVO consists of a multidrug microinjection device, fluorescent tracking microspheres which accurately denote the position of each drug injection site, and an automated image analysis package capable of quantifying both tumor cell and microenvironmental biomarkers of response to local drug exposure. To date, CIVO has been employed in 1000s of mice and over 70 canine patients, and it is currently being tested in the human clinic. Here we will present some of the first data from our clinical study now underway at the Fred Hutchinson Cancer Research Center and University of Washington School of Medicine in patients with soft tissue sarcoma. The ability to evaluate and cross- compare multiple drugs and drug combinations simultaneously in living tumors across a diverse immune competent patient population provides an innovative and exciting functional approach to complement current precision oncology strategies.

2:45 PM

Overcoming Challenges of Clinical Biomarker Development

Xuemei Zhao, Senior Principal Scientist

Merck & Co.

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Overcoming Challenges of Clinical Biomarker Development

The utility of various types of clinical biomarkers is to aid decision-making in drug development. After a pre-clinical candidate is approved for development in clinical studies, biomarker activities are transitioned from discovery to clinical biomarker development and implementation. Clinical biomarker development includes clinical biomarker assay development, fit-for-purpose assay validation, and clinical biomarker qualification in clinical studies. After a clinical biomarker is qualified, it will then be implemented in clinical studies to aid decision-making. Overcoming challenges of clinical biomarker development such as preanalytics, assay selectivity, and matrix effect will be discussed.

 

3:10 PM

Break and Visit Sponsors

3:25 PM

The End of the Beginning: Advancing the Application of Next Generation Sequencing for Companion Diagnostics in Oncology

Brian Kelly, Global Director, Diagnostic Partnering

ThermoFisher Scientific

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The End of the Beginning: Advancing the Application of Next Generation Sequencing for Companion Diagnostics in Oncology

The approval of the first Next Generations Sequencing-based companion diagnostics has taken us from a concept to reality. While we appreciate the initial accomplishments, we recognize the remaining challenges that now must be faced to take full advantage of the opportunity for changing the paradigm in diagnostics. This talk will explore several key questions including:

  • How can we leverage initial applications in targeted therapies to expand to address combination therapies, Immuno-oncology and beyond?
  • What are potential opportunities for change in regulation and commercialization that will shape this market?
  • Is there continued opportunity for pre-competitive collaboration between Pharma or Diagnostics organizations that can push us further faster?

3:50 PM

Companion Diagnostics Development on the nCounter® Analysis Platform

James Storhoff, Director, Diagnostic Product Development

NanoString Technologies

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Companion Diagnostics Development on the nCounter® Analysis Platform

TBA

4:15 PM

Genetic diagnosis of cardiac arrhythmias and cardiomyopathies

Oscar Puig, Chief Scientific Officer

Phosphorus

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Genetic diagnosis of cardiac arrhythmias and cardiomyopathies

Cardiac arrhythmias are the leading cause of sudden cardiac death in people under the age of 35. Arrhythmia disorders are often inherited, and genetic testing has become a routine diagnostic tool for cardiology clinics. Without genetic testing, this process may take several weeks and sometimes, if symptoms are not clearly manifested, may result in misdiagnosis. We developed a genetic diagnostic panel consisting of 137 genes, including exons, promoters, 5’-UTRs, 3’-UTRs, selected introns, coding sequences, and specific mutations designed to include genes directly linked with cardiovascular genetic disorders like cardiomyopathies, arrhythmias, and specific lipid disorders. This diagnostic assay, performed in a CAP/CLIA laboratory, was validated using clinical samples with known mutations, and it shows excellent analytical and clinical accuracy. Phosphorus cardio genetic test is currently commercialized as a Laboratory Developed Test, and it is being successfully used by physicians for the diagnosis of cardiac arrhythmias, cardiomyopathies and dyslipidemias.

4:45 PM

Closing Comments

Tom Fare, Director of Strategic Alliances

PlanetConnect

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Closing Comments