Round Table Discussions

Breakout Discussions in the Exhibit Hall
February 21, 2012
5:20-6:20pm

Concurrent problem solving breakout discussions, open to all attendees, speakers, sponsors, and exhibitors, provide a forum for discussing key issues and meeting potential collaborators. Plan to take part and explore these topics in-depth. Please pick a topic of your choice, find your table and join in.

Drug, Test and Lab
Moderator: Mollie Roth, J.D., COO, Diaceutics

How services are delivered
The need for real time reporting
Quality control and external quality assurance programs
Procedures for standardization
Making access to the test seamless and ensuring clear, standardized reporting

Cancer, a Molecular Pathology
Moderator: Harry Glorikian, Scientia Advisors
Has the time come where we can treat cancer for what it is – a molecular disease rather than an anatomic malady?

Enabling technologies (e.g., NGS)
Key hurdles

* Regulators – Following its recent guidance on companion diagnostics, could the FDA approve a molecular indication (e.g., EML4/ALK cancers vs. NSCLC with EML4/ALK)?
* Reimbursers – How should novel diagnostics –or off-label drug use in the interim– be reimbursed?
* Pharma – Can pan-cancer clinical trials be developed?

Impact of potential changes in cancer care

* On the FDA – How will the organization react?
* On pharma and emerging diagnostics players – How will they build a sustainable business model?

Clinical Utility: Where Regulatory and Reimbursement Meet
Moderator: Mya Thomae, CEO, Myraqa
Regulations and payer policies are in constant flux, especially with regard to lab-developed tests (LDTs). In this panel, experts from both domains will discuss a strategy that rises to both challenges by emphasizing proof of clinical value.

Outsourcing FDA: Regulated Diagnostic Product Development
Moderator: Gary Madsen, Ph.D., Biotech Resolutions, LLC

Share successes and failures when outsourcing product development (assay development, v&v, clinical trials, etc.)
What are areas of unmet need having high potential for outsourcing activities? Why?
What areas are of the most concern when outsourcing?
Are there areas that will NEVER be outsourced?
Is there a trend toward more or less outsourcing of development activities? Why?

Translating Novel Biomarker Discoveries to the Clinical Lab
Moderator: David Bourne, Senior Director, Business Development, Luminex Molecular Diagnostics

How will stakeholders deal with the next wave of diagnostic discoveries
Selecting a technology and platform, and downstream considerations
LDT or IVD kit
Outlook for LDT’s in the next 2 – 3 years

Hire Right the First Time: Interview Techniques That Work
Moderator: Tara Kochis, President, Slone Partners

Learn from recruiting experts
Must ask interview questions
How to probe and understand a candidate's motivation and why this matters
Why do people leave good jobs and how can knowing this help you retain your top talent

Maximizing Results Despite Small Sample Size
Moderator: David A. Eberhard, M.D., Ph.D., Associate Professor, Comprehensive Cancer Center and Research Associate Professor, Pathology & Lab Medicine, University of North Carolina, Chapel Hill

From Data to Diagnosis: NGS in the Clinic
Moderator: Fuad Gwadry, Bioinformatics Consultant

Adoption of WGS in the Clinic: Hurdles and Potential Solutions
Moderator: Katherine Tynan, Business Development & Strategic Consulting, Tynan Consulting LLC

Reimbursement
Regulatory
Physician education
Biological understanding
Ethics, privacy, and technology

Developments in Diagnostics Enabling Personalized Medicine
Moderator: Harald Kraushaar, Ph.D., Vice President & Head of Business Development, BioSciences, Sony DADC

Genomic testing: LDTs and IVD diagnostics
Microarrays vs. whole genome sequencing
Is sensitivity a driver for new diagnostics for PM?
Unmet needs and upcoming solutions

Integrative Genomics Approaches for Personalized Medicine
Moderator: Ilya Kupershmidt, Co-founder and Vice President of Products, NextBio

Strategic integration of NGS in translational research and the clinic
Genomic biomarkers in drug development: optimizing patient stratification in clinical trials
When to seq- a cure: clinical indications for targeted or whole genome sequencing

Cancer, a Molecular Pathology
Moderator: Harry Glorikian, Scientia Advisors
Has the time come where we can treat cancer for what it is – a molecular disease rather than an anatomic malady?

Enabling technologies (e.g., NGS)
Key hurdles
Regulators – Following its recent guidance on companion diagnostics, could the FDA approve a molecular indication (e.g., EML4/ALK cancers vs. NSCLC with EML4/ALK)?
Reimbursers – How should novel diagnostics –or off-label drug use in the interim– be reimbursed?
Pharma – Can pan-cancer clinical trials be developed?
Impact of potential changes in cancer care
On the FDA – How will the organization react?
On pharma and emerging diagnostics players – How will they build a sustainable business model?

From Data to Diagnosis: NGS in the Clinic
Moderator: Fuad Gwadry, Bioinformatics Consultant

Adoption of WGS in the Clinic: Hurdles and Potential Solutions
Moderator: Katherine Tynan, Business Development & Strategic Consulting, Tynan Consulting LLC

Reimbursement
Regulatory
Physician education
Biological understanding
Ethics, privacy, and technology

Developments in Diagnostics: Enabling Personalized Medicine
Moderator: Harald Kraushaar, Ph.D., Vice President & Head of Business Development, BioSciences, Sony DADC

Genomic testing: LDTs and IVD diagnostics
Microarrays vs. whole genome sequencing
Is sensitivity a driver for new diagnostics for PM?
Unmet needs and upcoming solutions

Innovative Techniques for Characterization of CTCs
Moderator: John F. Zhong, Ph.D., Assistant Professor, Pathology; Director, Bioinformatics, Gene Therapy Laboratories, University of Southern California School of Medicine

Which cell is a CTC
Define CTC with biomarker, DNA mutation or transcriptome
Heterogeneity of CTC population
Dynamic changes of CTC population for treatment evaluation
Challenge of CTC molecular analysis

CTCs: Value for Diagnostics, Therapeutics, and Cancer Personalized Medicine
Moderator: Enal Razvi, Ph.D., Biotechnology Analyst

CTCs in various cancer types, their origin, and role in metastasis
CTC isolation and characterization tools & technologies
CTCs in cancer personalized medicine: diagnostic and therapeutic optimization
Emerging trends and themes in the CTCs marketplace

Characterization of CTCs
Moderator: Yvon E. Cayre, M.D., D.Sci., Professor, Pierre and Marie Curie University; CSO, ScreenCell

Value of enumeration
CTCs and tumor initiating cells
CTCs and treatment monitoring

“Lost in Translation” – How to Turn Candidate Biomarkers into Validated Diagnostic Tests?
Moderator: Yuling Luo, Ph.D., Founder, President & CEO, Advanced Cell Diagnostics, Inc.

What are the current and new ways to validate biomarkers?
What are the main issues in analytical and clinical validation of biomarker assays?
What are the issues with various sample sources (e.g. blood, tissue) for biomarker analysis?
What is the impact of cellular and tissue context on biomarker analysis and clinical utility?
What is the impact of quantitation on biomarker analysis and clinical utility?
How simple and robust should a clinical diagnostic test be?

Which cell is a CTC
Define CTC with biomarker, DNA mutation or transcriptome
Heterogeneity of CTC population
Dynamic changes of CTC population for treatment evaluation
Challenge of CTC molecular analysis

CTCs: Value for Diagnostics, Therapeutics, and Cancer Personalized Medicine
Moderator: Enal Razvi, Ph.D., Biotechnology Analyst

CTCs in various cancer types, their origin, and role in metastasis
CTC isolation and characterization tools & technologies
CTCs in cancer personalized medicine: diagnostic and therapeutic optimization
Emerging trends and themes in the CTCs marketplace

Commercializing CTC technologies
Moderator: John A. Viator, Ph.D., Associate Professor, Biological Engineering and Dermatology, University of Missouri

Protecting your technology
Licensing your technology from an academic institution
Funding your venture

Characterization of CTCs
Moderator: Yvon E. Cayre, M.D., D.Sci., Professor, Pierre and Marie Curie University; CSO, ScreenCell

Value of enumeration
CTCs and tumor initiating cells
CTCs and treatment monitoring

“Lost in Translation” – How to Turn Candidate Biomarkers into Validated Diagnostic Tests?
Moderator: Yuling Luo, Ph.D., Founder, President & CEO, Advanced Cell Diagnostics, Inc.

What are the current and new ways to validate biomarkers?
What are the main issues in analytical and clinical validation of biomarker assays?
What are the issues with various sample sources (e.g. blood, tissue) for biomarker analysis?
What is the impact of cellular and tissue context on biomarker analysis and clinical utility?
What is the impact of quantitation on biomarker analysis and clinical utility?
How simple and robust should a clinical diagnostic test be?

Integrative Genomics Approaches for Personalized Medicine
Moderator: Ilya Kupershmidt, Co-founder and Vice President of Products, NextBio

Strategic integration of NGS in translational research and the clinic
Genomic biomarkers in drug development: optimizing patient stratification in clinical trials
When to seq- a cure: clinical indications for targeted or whole genome sequencing

Integration of Sequencing Technologies to the Clinic
Moderator: Stephen Kingsmore, M.B., Ch.B., BAO, D.Sc., FRCPath, Director, Center for Pediatric Genomic Medicine, Children’s Mercy Hospital

DTC Genetic Tests for Common Complex Diseases
Moderator: Nazneen Aziz, Ph.D., Director, Molecular Medicine, Transformation Program Office, College of American Pathologists

Advances in Non Invasive Genetic Testing
Moderator: Charles Cantor, Ph.D., CSO, Sequenom, Inc.

Fragment-Based Screening
Moderator: Daniel A. Erlanson, Ph.D., Carmot Therapeutics, Inc.

What screening techniques are best for finding fragments? How many orthogonal techniques are needed?
How much structural information is needed to advance fragments? Can fragments be advanced without knowing the structure of the co-complex?
What makes a good fragment – ligand efficiency, structure, novelty, chemical tractability, SAR?
At what point in a lead finding campaign should fragment-based lead discovery enter the picture – from the start of a program, or when all else fails?
Once fragments are identified, how can they be further elaborated – growing, merging, linking, library design, other?

Property-Based Design
Moderator: Darren McKerrecher, Associate Director, Medicinal Chemistry & Project Leader, Cardiovascular & Gastrointestinal Innovative Medicines Unit, AstraZeneca

What do we mean by property-based design...which properties do we most need to control?
At what stage in a drug discovery program is property-based design most effective?
Does property-based design reduce attrition, or just identify failures faster?
How do we continue to allow ourselves the benefit of serendipity within a property-based design paradigm?
What are good examples of successful property-based design?

Allosteric Modulators
Moderator to be Announced

Kinase Drug Discovery
Moderator: Jean Cui, Ph.D., Associate Research Fellow, Oncology Medicinal Chemistry, Pfizer Global R&D

Considerations for kinase selectivity:
Assays for the evaluation of kinase selectivity
Design principles for kinase selectivity
Successful examples of highly selective kinase drugs
Drug-like properties of kinase inhibitors
Non-ATP competitive kinase inhibitors

The Use of NGS in Cancer Diagnostics – Challenges in Moving from Research into the Clinic
Moderator: Jeffrey M. Otto, Ph.D., M.B.A., National Director, Catholic Health Initiative's Center for Translational Research
- Is there demonstrated clinical evidence suggesting an improvement over existing alternatives?
- Who should pay for the test? Payors? Patients? Providers?
- 85% of cancer care is delivered in community hospitals – how to drive adoption when existing molecular diagnostics are under-utilized?
- Is NGS, while a strong research tool, not a viable platform for routine molecular diagnostics?

Use of Retrospective and Prospectively collected samples for the discovery and validation of Biomarkers
Moderator: Rao Mulpuri, Ph.D. M.B.A., Director of Scientific Operations, Catholic Health Initiative's Center for Translational Research
a) Ethical, Privacy and consenting challenges
b) Challenges in using previously collected samples – how to normalize the data for sample quality
c) Clinical and genomic data integration challenges – define best practices

How do you Facilitate Excellence in Translational Research in an Academic Setting?
Moderator: Michael Kalos, Ph.D., Director, Translational and Correlative Studies Laboratory, University of Pennsylvania School of Medicine

How Can We Define the Appropriate Patient Population Early in Drug Discovery?
Moderator: Joerg Heyer, Ph.D., Director, Genetic Models, Translational Research, AVEO Pharmaceuticals

Effects of translational medicine on increasing patient population knowledge
Informatics approaches
Pharmacogenomics, clinical data, and more

Forging New Regulatory Pathways for Delivery of Personalized Medicine
Moderator: Linda Burdette, Ph.D., Director, Drug Regulatory Affairs, F. Hoffmann-La Roche, Inc.
- Brainstorm new regulatory pathways to facilitate co-development of drug/diagnostics
- Explore areas where additional FDA guidance is needed for co-development of drug/diagnostics
- Propose incentives for innovation and risk-based approaches to contemporaneous development of drug/diagnostics

To Randomize or Not to Randomize in Oncology Phase 2 Clinical TrialsModerator: Ionel Mitrica, Ph.D., Director, Clinical Development, Oncology, GlaxoSmithKline
- Review experience with/bias toward Phase 2 single-arm trials (SATs) vs randomized controlled trials (RCTs) in roundtable participants’ institutions
- Review some of the pros and cons for SATs vs RCTs, and when one or the other may make more sense
- Discuss the role of RCTs in early oncology drug development

Genomic Based Approach to Cancer Clinical TrialsModerator: Apostolia-Maria Tsimberidou, M.D., Ph.D., Associate Professor, Department of Investigational Cancer Therapeutics, University of Texas, MD Anderson Cancer Center
- Optimization of molecular profiling: challenges
- Development of novel targeted agents
- Collaboration and exchange of information regarding molecular profiling and treatment selection

Network Pharmacology for Cancer Drug Discovery: Are We There Yet?
Moderator: Asfar S. Azmi, Ph.D., Research Associate, Pathology, Karmanos Cancer Institute, Wayne State University

It’s Never Too Early to De-Risk Your Lead Compounds!
Moderator:Glenn Short, Ph.D., Director, Discovery Sciences, Zalicus, Inc.
·Cell-based approaches to de-risking potential lead compounds in discovery phase
·Utility of cell-based disease models
·Assay strategies for indication selection, target/mechanism i.d. and triage for potential lead molecules

Standardizing External Data
Moderator: Sandor Szalma, Head, External Innovation, Research & Development IT, J&J Pharmaceutical Research & Development, LLC

What is external data? Who needs it anyway?
Standards – pre-competitive collaboration or competitive advantage?
Who should drive standards development?
Best practices for standards development? Large groups or small groups?
Adopting standards – what if the “standard” does not cover my use case?
The devil is in the detail – or is it?

Translational Informatics
Moderator: Shoibal Datta, Ph.D., Associate Director, R&D Information Technology, Therapeutic Areas & Translational Medicine, Biogen Idec, Inc.

Informatics for Biologics
Moderator to be Announced

Cancer, a Molecular Pathology
Moderator: Harry Glorikian, Scientia Advisors
Has the time come where we can treat cancer for what it is – a molecular disease rather than an anatomic malady?

Enabling technologies (e.g., NGS)
Key hurdles
Regulators – Following its recent guidance on companion diagnostics, could the FDA approve a molecular indication (e.g., EML4/ALK cancers vs. NSCLC with EML4/ALK)?
Reimbursers – How should novel diagnostics –or off-label drug use in the interim– be reimbursed?
Pharma – Can pan-cancer clinical trials be developed?
Impact of potential changes in cancer care
On the FDA – How will the organization react?
On pharma and emerging diagnostics players – How will they build a sustainable business model?

From Data to Diagnosis: NGS in the Clinic
Moderator: Fuad Gwadry, Bioinformatics Consultant

Adoption of WGS in the Clinic: Hurdles and Potential Solutions
Moderator: Katherine Tynan, Business Development & Strategic Consulting, Tynan Consulting LLC

Reimbursement
Regulatory
Physician education
Biological understanding
Ethics, privacy, and technology

Developments in Diagnostics: Enabling Personalized Medicine
Moderator: Harald Kraushaar, Ph.D., Vice President & Head of Business Development, BioSciences, Sony DADC

Genomic testing: LDTs and IVD diagnostics
Microarrays vs. whole genome sequencing
Is sensitivity a driver for new diagnostics for PM?
Unmet needs and upcoming solutions

Which cell is a CTC
Define CTC with biomarker, DNA mutation or transcriptome
Heterogeneity of CTC population
Dynamic changes of CTC population for treatment evaluation
Challenge of CTC molecular analysis

CTCs: Value for Diagnostics, Therapeutics, and Cancer Personalized Medicine
Moderator: Enal Razvi, Ph.D., Biotechnology Analyst

CTCs in various cancer types, their origin, and role in metastasis
CTC isolation and characterization tools & technologies
CTCs in cancer personalized medicine: diagnostic and therapeutic optimization
Emerging trends and themes in the CTCs marketplace

Characterization of CTCs
Moderator: Yvon E. Cayre, M.D., D.Sci., Professor, Pierre and Marie Curie University; CSO, ScreenCell

Value of enumeration
CTCs and tumor initiating cells
CTCs and treatment monitoring

“Lost in Translation” – How to Turn Candidate Biomarkers into Validated Diagnostic Tests?
Moderator: Yuling Luo, Ph.D., Founder, President & CEO, Advanced Cell Diagnostics, Inc.

What are the current and new ways to validate biomarkers?
What are the main issues in analytical and clinical validation of biomarker assays?
What are the issues with various sample sources (e.g. blood, tissue) for biomarker analysis?
What is the impact of cellular and tissue context on biomarker analysis and clinical utility?
What is the impact of quantitation on biomarker analysis and clinical utility?
How simple and robust should a clinical diagnostic test be?

Which cell is a CTC
Define CTC with biomarker, DNA mutation or transcriptome
Heterogeneity of CTC population
Dynamic changes of CTC population for treatment evaluation
Challenge of CTC molecular analysis

CTCs: Value for Diagnostics, Therapeutics, and Cancer Personalized Medicine
Moderator: Enal Razvi, Ph.D., Biotechnology Analyst

CTCs in various cancer types, their origin, and role in metastasis
CTC isolation and characterization tools & technologies
CTCs in cancer personalized medicine: diagnostic and therapeutic optimization
Emerging trends and themes in the CTCs marketplace

Commercializing CTC technologies
Moderator: John A. Viator, Ph.D., Associate Professor, Biological Engineering and Dermatology, University of Missouri

Protecting your technology
Licensing your technology from an academic institution
Funding your venture

Characterization of CTCs
Moderator: Yvon E. Cayre, M.D., D.Sci., Professor, Pierre and Marie Curie University; CSO, ScreenCell

Value of enumeration
CTCs and tumor initiating cells
CTCs and treatment monitoring

“Lost in Translation” – How to Turn Candidate Biomarkers into Validated Diagnostic Tests?
Moderator: Yuling Luo, Ph.D., Founder, President & CEO, Advanced Cell Diagnostics, Inc.

What are the current and new ways to validate biomarkers?
What are the main issues in analytical and clinical validation of biomarker assays?
What are the issues with various sample sources (e.g. blood, tissue) for biomarker analysis?
What is the impact of cellular and tissue context on biomarker analysis and clinical utility?
What is the impact of quantitation on biomarker analysis and clinical utility?
How simple and robust should a clinical diagnostic test be?

Developing Antibody Drug Conjugates
Moderator: Puja Sapra, Ph.D., Director, Bioconjugates, Oncology Research Unit, Pfizer Biotherapeutics

Novel approaches to find targets
Novel payloads beyond tubulin inhibitors and DNA binders
Site specific conjugation approaches: Hope or hype
Precision medicine for ADCs: biomarker assays (IHCs, imaging, CTCs etc)
Investment in translational biology: from preclinical to clinic
Beyond whole antibody-drug conjugates: smaller fragments, other approaches

Technological Challenges with Antibody Drug Conjugates
Moderator: Ho Sung Cho, Ph.D., Chief Technical Officer, Ambrx

Determining the best combination of warhead and target
Selection of right linker technology for the product
Ensuring uptake, internalization and activation of the product
Ensuring specificity to tumor cells and not normal ones
In vitro studies and in animal models

Selection of good predictive biomarkers
Challenges with optimizing performance
Challenges with toxicity

Challenges with characterization (heterogeneous) and comparability

Measures to Improve ADC Linkers
Moderator: Bob Lutz, Ph.D., VP, Translational R&D, ImmunoGen, Inc.

Development of payload and linker technology
Is linker stability the property of importance?
Impact of linker on the safety profile of the ADC

Challenges of Targeting Cancer Stem Cells
Moderator: Norman J. Maitland, Ph.D., Chief Scientific Officer, Procure Therapeutics Ltd

How cancer stem cells differ from normal stem cells and other cancer cells
Approaches to the identification and evaluation of new cancer stem cell targets
Payloads that overcome the enhanced resistance of cancer stem cells
Issues to do with selection of animal model
Concerns to do with delivery and clearance
Potential safety issues
Strategy for entering clinical trials
Demonstration clinical benefit

Finding the right targets
Moderator: Patrick A. Baeuerle, Ph.D., CSO, Senior Vice President, R&D, Micromet, Inc.

Targets for dual ligand or receptor blockers
How to select target combinations
Targets for T- or NK cell-engaging antibodies
How to achieve a therapeutic window

Pre-clinical Assessment for Prediction of Clinical Success for Cancer Biologics
Moderator: Gary Starling, Ph.D., Director, GPRD Discovery, Oncology Biologics, Abbott Biotherapeutics Corp.