October 17 – 19 2017 | Boston, MA

Hilton Boston/Woburn

Day One
Wednesday, October 18, 2017

Day Two
Thursday, October 19, 2017

07.00
Registration & Breakfast

Keynote Address: “Precision Medicine” Under the Microscope: A Frank Look at the Impact of Precision Medicine on Patient Welfare

08.00
Standardizing and Validating Immunoassays to Advance the Development of IO Signatures

Synopsis

  • Investigating methods to validate antibody based immunoassays
  • Getting to the crux of standardization: Improving sensitivity and reproducibility to ensure your assay is fit for purpose
  • What constitutes a clinically validated immunoassay: Discussing criteria on assay controls, dose response and antibody specificity
  • Overcoming variability in the measurement and interpretation of early IO biomarker assays

08.30
The Pharmaceutical Perspective on the Advancement of Precision Medicine

  • Jakob Dupont Vice President & Global Head, Breast & Gynecologic Cancer Development, Genentech

Synopsis

  • Current outlook on R&D endeavours in the oncology space, are we still only capturing the lowest hanging fruit?
  • Discussing how to better invest in precision medicine and adjust business strategy to bring better medicines with better outcomes to more patients

09.00
Microfluidic Cell Tethering Enables Rapid Analysis of Drug Responses in Live Patient CTCs

  • Stuart Martin Professor , University of Maryland, speaking on behalf of ANGLE plc

Synopsis

  • Developing a novel microfluidic medical device for imaging cytoskeletal dynamics in non-adherent patient tumor cells and integrating hydrophobic lipids into an engineered surface to tether tumor cells to improve high-resolution imaging and preserves the dynamic cytoskeletal behavior of non-adherent tumor cells
  • Utilizing live-cell confocal microscopy to demonstrate that McTNs are detectable within hours of when tumor cells are recovered from breast cancer patients, either via surgery or microfluidic isolation of CTCs from patient blood samples
  • Enabling immediate testing of patient cells for responses to cytoskeletal drug treatments that could influence CTC reattachment during metastasis

09.30
Leveraging Companion Diagnostics to Drive Clinical Trial Design

  • Mark Roberts Senior Director, Diagnostics Development, Covance

Synopsis

  • Discussing how to differentiate your compound, in a market of multiple biopharma companies chasing the same biomarker
  • Understanding the role of CDx to enhance trial enrollment and execution
  • How to best prepare for critical regulatory and commercialization considerations

10.00
Keynote Panel: “Precision Medicine” Under the Microscope: A Frank Look at the Impact of Precision Medicine on Patient Welfare

  • Reena Philip Director, Division of Molecular Genetics and Pathology, OIR/CDRH , FDA
  • Khatereh Calleja Senior Vice President, Technology & Regulatory Affairs, AdvaMedDx
  • Philip Lerner Vice President & National Medical Director , Aetna

Synopsis

The keynote panel at World CDx 2017 will take a candid look at the precision medicine mantra so far. With all the investment from pharma and government alike, what  current success and tangible  real-world benefit are we seeing for patients? Is this enough? And what can we do to ensure we enter the next decade with precision medicine changing the lives of as many prospective patients as possible?

 

The following perspective will be provided:

 

  • Pharmaceutical: Discussing how to better invest in precision medicine and adjust business strategy to bring better medicines with better outcomes to more patients
  • Patient: Elevating the role of patient power and advocacy on the real-world impact of precision medicines and diagnostics
  • Payer: Getting to the crux of value/outcomes based healthcare and how we address drug and diagnostic reimbursement across the US
  • Policy: The effect of changing healthcare policy will have on future precision medicine endeavors

10.45
Morning Refreshments & Networking

Enhancing the Development of ‘Omic Derived Predictive Biomarker Signatures

11.30 The Discovery and Development of a STn Antibody Biomarker Signature and Diagnostic for Ovarian Cancer

  • Discovering and developing a panel of novel, highly-selective, high-affinity monoclonal antibodies to STn using a proprietary immunization strategy and core glycan screening technologies
  • Discussing the development of a novel tissue-based companion diagnostic for Siamab’s anti-STn antibody drug conjugate to select patients that possess elevated levels of the STn carbohydrate
  • Proposing a novel exploration of correlations between an IHC signal in ovarian tumors with genetic and protein markers that offers the chance to enhance the strength of each biomarker alone

Dan Dransfield, Senior Vice President Research & Development, Siamab Therapeutics

12.00 Case Study: Exploring the Early Co-Development of Panels for Precision Medicine

  • A how to guide for success development of assay panels
  • The Randox perspective on lessons learnt and best practice for future collaborations

Claire Huguet, Head, Biomarker Services, Randox

12.30 Developing Robust RNA Sequencing Gene Expression Profiles as Predictive Signatures

  • Discussing the benefits of RNA-sequencing as a platform for robust biomarker identification
  • Harnessing RNA-Seq for targeted gene expression assays
  • Realizing the emerging role of RNA-Seq in developing predictive biomarkers signatures for IO therapy

Naveen Babbar, Executive Director, Global Program Diagnostics, Novartis

13.00 Development of a Pharmacodynamic Biomarker Assays for a First-in-Class Oral Selective ERα Covalent Antagonist (SERCA) for the Treatment of ERαWT and ERαMUT Breast Cancer

  • ERα mutations functionally confer resistance to existing classes of endocrine therapies, likely through gaining constitutive activity
  • Current ER-directed therapies are only partially effective in the ERα mutant setting, and that a significant proportion of resistant breast cancer metastases continue to remain dependent on ERα signaling for growth/survival, highlights the critical need to develop the next generation of ERα antagonists that can overcome aberrant ERα activity
  • The role and development of pharmacodynamic biomarker assays to establish SERCA activity in clinical trial samples will be discussed

Victoria Rimkunas, Senior Scientific Investigator, H3 Biomedicine

13.30 Networking Lunch

Developing Novel Biomarker Signatures for the Next Wave of Immuno-Oncology Targets

14.30 Analyzing Biomarker Identification in Immuno-Oncology

  • Discussing approaches to biomarker identification to guide the use of small molecules and biologics in the context of an integrated translational research program
  • What signatures are we looking for and where should we be looking?
  • Discussing which markers hold promise for being most clinically relevant

Jennifer Mataraza, Senior Investigator & Group Leader, Translational & Exploratory Immuno-Oncology, NIBR

15.00 From Research to the Clinical: Advancing RNA-ISH Detection in Companion Diagnostics

  • Advantages and applications of in situ RNA detection
  • Collaboration of key stakeholders for co-development of a drug and RNA-ISH diagnostic device
  • Regulatory and commercial considerations for new innovative technologies in companion diagnostics

Jessica Riley,  Senior Director, Business Development, Pharma Partnerships, Leica Biosystems

15.30 Beyond ICI: Discovery and Validation of Predictive Profiles and Platforms for T-Cell Based Therapeutics

  • Discussing novel ways of looking at TCR rearrangements and epitope identification
  • Evaluating strategies for developing predictive biomarkers for T cell therapies
  • An eye downstream: Developing IVD strategies for T cell based therapies

Sharon Moulis, Head, Companion Diagnostics, Juno Therapeutics

16.00 Afternoon Refreshments

Evolving the One Marker-One Platform Mantra: Progressing Multiplexed & Universal Signatures & Platforms

16.15 Developing Multi-Analyte Signatures for Next Generation Multiplexing Platforms

  • Overcoming inadequacies in single analyte testing for patient selection to prove the value of a multiplexed approach
  • Validating multimarker signatures on novel technology to drive multi-analyte testing: Discussing limits of detection, sensitivity, cross reactivity and sample integrity and availability
  • Contextualizing disease pathogenesis utilizing multimarker signatures to expedite the development of more robust patient selection signatures

Morten Sogaard, Vice President & Head, Genomic Sciences & Technologies, Pfizer

16.45 Building on the Universal Approach to Introduce the Pan-Tumor Paradigm

  • What is the pan-tumor approach and how does it differ from the universal and multiplexed approaches?
  • Discussing the “must haves” for developing and validating a pan-tumor biomarker signature and assay
  • Evaluating this new paradigm of tumor independent diagnostic tests

Speaker to be confirmed

17.15 Panel Discussion: Advancing the Development and Clinical Application of Universal and Multi-Analyte Biomarker Assays

  • Debating the clinical relevance of multiplex technology: What is the best platform and type of signature to develop?
  • Discussing the benefits and applicability of multi-analyte testing over single-marker platforms
  • Transitioning from single to multi-analyte as a paradigm: What need to do now to streamline this transition as an industry?
  • How to validate multiplexing platform and discussing the standards required to perform exploratory testing
  • How does the approval of universal companion diagnostics and multigene panels impact future biomarker discovery and development

Morten Sogaard, Vice President & Head, Genomic Sciences & Technologies, Pfizer

Additional Panellists Confirming

18.00 Chairs’ Closing Remarks

18.05 End of Day One

Clinically Validating Predictive Responder & Outcome Signatures for IO Therapies

11.30 A Review: Improving Clinical Patient Selection and Stratification for Immunotherapy

  • Getting a clean signature: How do we do robust patient selection for immunotherapies?
  • Getting granular on responder/non-responder patient populations: Promoting strategies for subpopulation identification of long-term responders to immunotherapy
  • Optimize selection of patients beyond prognostic markers to avoid excessive toxicity in IO clinical studies

Zhen Su, Vice President  & Head of Global Medical Affairs Oncology, EMD Serono

12.00 Leveraging Diagnostic Quality as a Means to Align Clinical Trial with Real World Results

  • Quality of result: Assessing assay performance and pathologist interpretation to ensure improved selection of patient responders
  • Impact: Dissecting data integrity and confidence to support submission and approval of clinical validation to ensure real world clinical results best align with clinical trial data

Rosanne Welcher, Senior Director, Regulatory, Quality & Clinical Affairs, Agilent Technologies

12.30 Advancing the Precision Medicine Approach to IO Combination Trials

  • Learning how to best derive combination signatures to improve trial strategy
  • Harnessing innovative trial design to compare effectiveness of combination versus monotherapy for biomarker signature and therapeutic validation
  • Utilizing biomarker data, in conjunction with target interaction to enhance dose selection in combination studies

David Kaufman, Executive Director, Translational Immuno-Oncology, Merck

13.00 Turning Tumor Mutations into Personalized Cancer Therapies

  • Evaluating each patient’s tumor using next-generation sequencing and to identify candidate TSNAs
  • Using proteomics and machine learning to predict which TSNAs can activate tumor-specific T cells
  • For each patient, manufacturing a TSNA-based vaccine and delivering it in combination with immune checkpoint blockade

Roman Yelensky, Executive Vice President & CTO, Gritstone Oncology

13.30 Networking Lunch

Improving the Operational Execution of Precision Medicine Clinical Trials

Innovating in Sampling and Biomarker Detection to Lay the Foundations of Success for Global Clinical Trials

  • Discussing the variability in clinical sample quality from clinical trial sites and how to best standardize sample collection and preparation across multiple geographies
  • Rolling out global training and support to best mitigate risk of quality, complex sample collection
  • Discussing the role of sample collection, both tissue and fluid, on the advancement of biomarker driven drug development
  • Discussing liquid biopsy driven trials and considerations for sample collection and downstream clinical relevance

Dmitri Mikhailov, Head, Biomarker Coordination, Novartis

15.00 Improving Patient Recruitment and Retention on Precision Medicine Clinical Trials

  • What can be done to improve clinical trial enrolment using NGS ?
  • Discussing the challenges and pitfalls of patient enrolment
  • Understanding what can be done by drug developers and testing companies do make getting patients onto trials easier
  • Improving patient advocacy and educating to promote the enrolling and retention of patients into “precision medicine” clinical trials

Daniel Simon, Vice President, Pharma Business Development, Guardant Health

15.30 Umbrella and Basket Trials for Molecular Targets in Precision Medicine

  • Utilizing basket and umbrella trials to enrich for responders to statistically power smaller, shorter and more cost-effective late stage trials
  • Harnessing these trial designs to more granularly define the best plan of action for subpopulations of patients
  • Understanding how basket and umbrella trials can be utilized to improve patient recruitment and retention on clinical studies

Jason Christiansen, Vice President, Diagnostics, Ignyta

16.00 Afternoon Refreshments

Successful Implementation of Biomarker Signatures: Clinical Trial Case Studies

16.15 Precision Medicine for Alzheimer’s Disease: Applying Genomic Stratification of Patients Based on APOE4 for Clinical Efficacy

  • By integrating information from a large dataset in patients treated with the active molecule tramiprosate, genetic markers and new insights into the pathology of Alzheimer’s disease, we have identified a group of patients that are likely to have substantial benefit from tramiprosate
  • We have created ALZ-801, an optimized prodrug of tramiprosate, that substantially improves the pharmacokinetic and tolerability profile of the active molecule
  • ALZ-801 is one of the few oral drug candidates in advanced stages of clinical testing in Alzheimer’s disease, a pill with a favorable safety profile and the potential to slow the course of the disease in all Alzheimer’s patients

Martin Tolar, Founder, President & Chief Executive Officer, Alzheon

16.45 Advancing the Precision Medicine Approach to IO Combination Trials

  • Learning how to best derive combination signatures to improve trial strategy
  • Harnessing innovative trial design to compare effectiveness of combination versus monotherapy for biomarker signature and therapeutic validation
  • Utilizing biomarker data, in conjunction with target interaction to enhance dose selection in combination studies

Jean-Marie Cuillerot, Chief Medical Officer, Agenus

17.15 Regeneron Genetics Center: From Target Discovery to Pharmacogenomics

  • Discussing the establishment and mission of the Regeneron genetics center
  • Currently performing a large number of genomic studies, including family-based mendelian studies, founder population studies, and large-scale population based studies
  • Analyzing current pharmacogenomic studies for a number of Regeneron clinical programs

Charles Paulding, Director, Pharmacogenomics, Regeneron Pharmaceuticals

17.45 Chair's Closing Remarks

17.50 Close of Day One

Overcoming Discrepancies in the Definition of “Value” Between All Industry Stakeholders

11.30 Reinventing Reimbursement Strategy to Demonstrate Value to Payers and Other Stakeholders

  • Understanding what stakeholders, including payers, providers and patients, need to see in order to reach the threshold for sufficient value demonstration
  • How do we demonstrate stand-alone value for a diagnostic test?
  • Revamping your reimbursement strategy to focus on the clinical application, validity and outcome to ensure alignment with requirements in value demonstration

Chandra Branham, Vice President, Payment & Health Care Delivery Policy, AdvaMed

12.00 A Fully Automated, Highly Accurate, Easy to Use MDx Platform: Too Much to Ask For?

  • Discussing the development of a fully automated, highly accurate MDx platform
  • Demonstrating the value of such a platform to the advancement of patient access to ensure the right treatment at the right time

 

Vishal Sikri, US General Manager, Biocartis

12.30 Evaluating the Impact of a Changing Healthcare Paradigm on Diagnostic Reimbursement

  • Discussing the reimbursement of cutting edge, complex technologies: Does the price point prevent the real world application of these tests?
  • Debating the impact of geopolitical and regulatory changes on HTA submission for diagnostic tests and precision medicines
  • How to approach reimbursement for NGS assays and understanding its impact on medical adoption

Cecilia Schott, Vice President, Precision Medicine, Global Product & Portfolio Strategy, Astrazeneca

13.00 NGS Panels: From Patient Selection to CDx

  • Harnessing NGS to facilitate biomarker discovery in early phase clinical trials and subsequent development of CDx assays with potential utility across multiple drug targets
  • Case studies: Exploring the main considerations for development and analytical validation of such panels and
  • Identifying important technical and regulatory challenges in CDx development

Katarina Wikstrom, Director of US Operations, Almac Diagnostics

13.30 Networking Lunch

The Regulatory & Policy Landscape of Diagnostic Enabled Therapeutics & IVDs

14.30 FDA Presentation: The Inside Track on IVD Regulation

  • Understand how the FDA are viewing the shift beyond the one marker-one platform mantra
  • An update on complementary and companion diagnostic regulation
  • Views on the regulation of NGS based, universal and pan-tumour diagnostic platforms

Reena Philip,  Director, Division of Molecular Genetics & Pathology, OIR/CDRH, FDA

15.00 NGS Solutions Across the Oncology Drug Development Continuum: From Biomarker Discovery to IVD

  • Applications of ctDNA analysis in pharmaceutical research and clinical development
  • Challenges in development of IVDs based on liquid biopsy
  • How can the decentralized assay model be applied to liquid biopsy approaches

John Simmons, Director, Translational Science & Diagnostics, PGD

15.30 Understanding the Impact of Recent NGS Regulatory Success on Future R&D and Approval Efforts

  • Discussing the IDE and PMA process for NGS based platforms: Lessons for streamlining future approval
  • Debating the impact of NGS platform approval on the future development of multigene assays
  • What can we learn and apply from tissue based NGS platforms to advance the approval of liquid based multigene assays

Hakan Sakul, Vice President & Head of Diagnostics, Pfizer

16.00 Afternoon Refreshments

Streamlining Diagnostic Co-Development from Clinical Assay to Commercial IVD

16.15 Which Comes First, Diagnostic or the Drug?

  • Discussing a novel paradigm in diagnostic development paired with drug development
  • Evaluating a “diagnostic first” process for matching drug development to Dx design
  • Debating the impact of such a re-think in drug-Dx co-development on commercial success

Jonathan Pan, Vice President & Head of Biomarker Strategy & Development, Aevi Genomic Medicine

16.45 Streamlining the Transit from RUO to IVD to Expedite Development

  • How and when to transition your assay from RUO to IVD to maximize chances on timeline delivery for the approval process
  • Discussing best practice in validating fit for purpose assay and when an assay is validated enough to trigger IVD development
  • Maximizing the affinity of antibodies for RUO assay to streamline transition to IVD

Moderated by Nick Ibery, Partner, Healthcare & Life Sciences

17.15 Dx Strategy Considerations for Commercial Targeted Therapeutic Success

  • Discussing the commercial impact of complementary vs companion diagnostics and the implications for market penetration and physician adoption of a targeted therapeutic
  • How do you handle a complementary diagnostic commercially, when it’s not in the Rx label and it’s not required for Rx treatment, to ensure your drug is adopted?
  • To CDx or not to CDx: Discussing the impact of the current LDT climate on the adoption and integration of IVDs into healthcare
  • Discussing the commerciality of multigene panels: How do we harness these platforms in the real world to get drugs adopted?
  • Does the price of multigene NGS panels rule out their commercial viability? Do we need a multi-panel approach or is the refinement and utility of a few selective markers the best approach?
  • What are the advantages of developing a multi-analyte CDx and how can they improve market share?
  • Discussing the merits and shortfalls of centralized and de-centralized models for testing

 

Hakan Sakul, Vice President & Head of Diagnostics, Pfizer

Jeffrey Emch, Biomarker & Companion Diagnostic Lead, Merck

Rosanne Welcher, Senior Director, Regulatory, Quality & Clinical Affairs, Agilent Technologies

Gina Wallar,  Division Vice President, Pharma Services, National Sales, NeoGenomics Laboratories

Eric Faulkner, Vice President, Precision & Transformative Technology Solutions, Evidera

18.00 Chairs' Closing Remarks

18.05 End of Day One