Questions & discussion

Application of biomarkers to early phase oncology drug development

• Appropriate use of phase-appropriate biomarkers in early stage development can improve decision-making during cancer drug development and ultimately improve cycle times and reduce costs. When done properly, they provide the opportunity to:
  • Demonstrate a pharmacologic effect in phase I
  • Assess effects on target and or the pathway
  • Optimize dose selection
  • Decide on early termination

Christopher A. Slapak, MD, Distinguished Lilly Scholar (Executive Director), Eli Lilly and Company

Questions & discussion

KRAS: A biomarker for our time? Driving the development and validation of biomarkers to predict patient response
Short presentations, followed by panel discussion & extended audience Q&A

• Why has KRAS impacted clinical practice so quickly and how typical will it prove to be in this regard?
  • Will it present an opportunity for the future, or is it just an associated epiphenomenon which will prove to be a red herring for future researchers seeking related markers?
• How has the labelling changed and has this met the hopes and expectations of each stakeholder?
• How should we discover and develop the "next KRAS"? Assessing progress in the development of the next generation of predictive biomarkers of response
  • What lessons can be/are being taken from the original?
• How will the FDA approach the validation of future biomarkers where data is conclusive without the necessity for a randomized trial?
• What biomarker platform(s) should one invest in?
  • What type of platform is suited to a given indication/therapeutic
    approach?
    • Are there broader platforms that can cover all your needs across the portfolio, or do you need to piece together individual solutions to cover all bases: Which approach is optimal?

Panellists:
Roy Baynes, MD, PhD, Vice President, Global Clinical Development & Therapeutic Area Head Hematology/Oncology, Amgen, Inc
Dr Walter H. Koch, Vice President & Head of Research, Roche Molecular Systems, Inc
Dr Michael Shi, Senior Director, Biomarker Project Leader, Oncology Translational Medicine, Novartis Pharmaceuticals Corporation

Where are the added values to pharma of current initiatives using imaging in clinical trials?

• What are the current initiatives that are investigating imaging as a biomarker?
• What are the goals of the working groups participating on these initiatives?
• How and when might the output of these initiatives become incorporated into drug development?
  

Speakers:
Eric S. Perlman, MD, Vice President, Molecular Imaging, RadPharm, Inc
Dr Howard Higley, Director, Scientific Affairs, CCS Associates, Inc

Morning coffee in the exhibition area

Questions & discussion

Case studies
What is the latest progress with trial patient pre-selection and biomarker development for novel oncology therapeutics at various stages of development?

• Are these strategies impacting go/no go criteria through the phases in practice?
• How do you decide if your patient selection criteria are robust enough, and whether you can create a pathway to registration?

Translating preclinical biomarker-related insights into clinic candidates and trial design

Speaker to be announced

Questions & discussion

Imaging is key to success in oncology translational research and drug development

Peter Duncan, Director - Business Development, Definiens

Questions & discussion

Buffet lunch in the exhibition area

OR | Lunch briefing sponsored by
Drug development for cancers arising out of increased genetic risk
Topics to include:

• Informed consent and legal issues for clinical sites that see patients who carry increased genetic risk for cancer
• Implications for pharmaceutical industry in designing clinical trials for these patient populations
• Optimal use of specialized diagnostic procedures such as radiology and laboratory evaluation
• Current guidelines for the management of patients who carry increased genetic risk for cancer

Moderators:
Dr Gary Fishbein, Senior Medical Director, PharmaNet Development Group
Kristen Shannon, MS, Program Manager for Cancer Genetics, Massachusetts General Hospital
(For a maximum of 50 participants)
» Click here for more information

OR | Working lunch
What are the keys to accessing traditional and non-traditional early-stage R&D funding for biotech? From start-up to Proof of Concept

Moderator: Aaron Schacht, Executive Director, Global External R&D, Eli Lilly and Company
(Very interactive, informal discussion-based session for a maximum of 12 participants each)

Clinical designs in oncology: A need for innovation - theory and reality

• Review why we have to target population (size of the trial, magnitude of the effect, statistics…)
• Main issues in targeting the population (validation of the assay, statistical design…)
• Our experience in designing such trials

Dr Jean-Pierre Bizzari, Senior Vice President, Clinical Oncology/Hematology, Celgene Corporation

Phase IIb/III

• How much data do you need from an oncology Phase II to get into pivotal trials with real prospects of success?
• Is there really any value in randomized, single arm Phase II trials in oncology?
  • Do they provide any sort of worthwhile indication whether or not you should proceed to Phase III?

Dr Nancy Simonian, CMO, Millennium Pharmaceuticals, the Takeda Oncology Company

Hypothesis-driven data exploration as a platform to advance biomarker discovery programs

A significant barrier to biomarker discovery and translational research in pharma is the reality that researchers do not have easy access to data across the research and clinical divide. This is especially problematic during hypothesis generation and initial evaluation when it is necessary to explore large amounts of diverse data in rapid iterations. Topics to be discussed:

• Data exploration vs. data analysis
• Challenges and opportunities for hypothesis-driven data exploration
• Clinical data repository for research
• Deriving value from less than perfect data

Jian Wang, PhD, CEO, BioFortis

Short presentation...
Lessons learned from Independent Central Review in registrational trials

• Protocol development
• Site qualifications
• Media transfer
• Image tracking
• Clinical data
• Site vs central discordance

Michael O'Neal, MD, CMO, RadPharm, Inc

...followed by panel discussion & extended audience Q&A

• Challenges of using biomarkers (imaging and non-imaging) in registrational trials
• Incorporation of non-imaging and imaging biomarkers

Moderator's closing summary

Close of session, followed by afternoon tea in the exhibition area

Questions & discussion

What are the latest developments in the terms of a regulatory pathway to licensure for companion diagnostics? What issues still need to be addressed?

Regulator's perspective
Companion drug diagnostics: Takes two to tango - regulatory perspective for personalized medicine

• FDA initiatives and activities in personalized medicine
• Regulatory framework to accommodate companion drug diagnostics
• Streamlining the co-development process
• Novel paradigms in personalized medicine

Dr Francis Kalush, Diagnostics & Personalized Medicine Network Leader, Office Center Director, CDRH/FDA

Questions & discussion

Industry perspective
Discovering and co-developing companion diagnostics in time for launch - challenges and opportunities in the current regulatory environment

• Companion diagnostic qualification process
• Challenges in discovering and developing companion diagnostics without impacting drug development timelines
• Methodological challenges in development - pragmatism vs purism
• The challenge of developing new drugs with established diagnostic tests

Dr Duncan McHale, Vice President of Translational Science, AstraZeneca

Questions & discussion

Buffet lunch in the exhibition area

OR | Lunch briefing sponsored by
Drug development for cancers arising out of increased genetic risk
Topics to include:

• Informed consent and legal issues for clinical sites that see patients who carry increased genetic risk for cancer
• Implications for pharmaceutical industry in designing clinical trials for these patient populations
• Optimal use of specialized diagnostic procedures such as radiology and laboratory evaluation
• Current guidelines for the management of patients who carry increased genetic risk for cancer

Moderators:
Dr Gary Fishbein, Senior Medical Director, PharmaNet Development Group
Kristen Shannon, MS, Program Manager for Cancer Genetics, Massachusetts General Hospital
(For a maximum of 50 participants)
» Click here for more information

OR | Working lunch
What are the keys to accessing traditional and non-traditional early-stage R&D funding for biotech? From start-up to Proof of Concept

Moderator: Aaron Schacht, Executive Director, Global External R&D, Eli Lilly and Company
(Very interactive, informal discussion-based session for a maximum of 12 participants each)

Case study
Examining the latest clinical trial design strategies for novel 'personalized' oncology therapeutics

• Accelerate Proof-of-Concept trials using biomarkers
• Increase probability of success for late phase trials by selecting the "right" patients

Dr Michael Shi, Senior Director, Biomarker Project Leader, Oncology Translational Medicine, Novartis Pharmaceuticals Corporation

Questions & discussion

Case study
A pharma-diagnostics collaboration model for drug development and the realization of personalized healthcare solutions

• How to integrate the disparate corporate and R&D models involved?
• When, in product development, should personalized medicine diagnostics be developed?

Dr Walter H. Koch, Vice President & Head of Research, Roche Molecular Systems, Inc

Questions & discussion

Roundtable debate & extended audience Q&A

• How will therapeutics with companion diagnostics be reimbursed?
  • Who will pay for them?
  • Is diagnostic reimbursement sufficient to sustain the development of the new diagnostics required for personalized medicine?
• Debating the correct apportioning of value between therapeutic and companion diagnostic components
  • What are the various stakeholders’ perspectives?
    • Where is the balancing point?
    • How does the money flow?

Moderator's closing summary

Close of session, followed by afternoon tea in the exhibition area

How can we ensure that the wealth of basic cancer biology knowledge about pathways and networks informs oncology drug discovery and development?

• Overview of state-of-the-science in cancer-relevant pathways
  • Tumor versus stromal contributions
  • Applicability of animal models
• Pathways versus organ system approaches
  • Alterations in key pathways differ in relative importance by organ system
  • Temporal and tumor/stromal interplay of pathway alterations as principal drivers of carcinogenesis
  • Methods to test multiple contributions
• Information/informatics tools to blend comparative pathway and network discoveries
  • The National Cancer Institute’s investment in the caBIG® program
  • A public-private partnership of co-clinical trials in state-of-the-art animal models
    

Dr Cheryl Marks, Associate Director, Division of Cancer Biology, & Director, Mouse Models of Human Cancers Consortium (MMHCC), National Cancer Institute

Panel discussion & extended audience Q&A

• What would be the key adaptations required to the current oncology R&D model to allow us to examine multiple pathways, and to use this as scientific hypothesis to guide the selection of therapeutic agents for combination?
• How will recently discovered phenomena such as circulating tumor cells impact R&D strategy moving forwards?
  • How will tumor microenvironment-targeting strategies develop?

Panellists:
Lee M. Nadler, MD, Dean of Clinical & Translational Research; Director, Harvard Catalyst; Virginia & D.K. Ludwig Professor of Medicine, Harvard Medical School, Senior Vice President, Experimental Medicine; Director, Center for Clinical & Translational Research; Pan Mass Challenge Senior Investigator, Dana-Farber Cancer Institute
Jian Wang, PhD, CEO, BioFortis
Dr Kevin Webster, Executive Director, Cancer BioScience, AstraZeneca

Close of day 2 followed by a cocktail reception in the exhibition area