Breakout Discussions

Join a breakout discussion group. These are informal, moderated discussions with brainstorming and interactive problem solving, allowing participants from diverse backgrounds to exchange ideas and experiences and develop future collaborations around a focused topic.

TUESDAY, JUNE 18
6:25 - 7:15 PM

3D Cellular Models

Human 3D Organ Models for Target Discovery and Drug Screening

Moderators: Hansjoerg Keller, PhD, Senior Investigator I, Musculoskeletal, Novartis Institutes for BioMedical Research
John Lowman, Partnerships and Innovation, Mimetas

• 50 Ways to leave your lover 2D cell culture, just start functional 3D cell models
• I can see clearly now; visualization and imaging challenges of 3D microtissues
• ‘It’s the economy and throughput, stupid!’ - what hinders 3D models in drug screening

Developing Organs-on-a-chip/Microphysiological Systems for Drug Discovery

Moderators: Kambez H. Benam, PhD, Assistant Professor, Division of Pulmonary Sciences and Critical Care Medicine, Departments of Medicine & Bioengineering, University of Colorado

Sina Mohammadi, PhD, Associate Principal Scientist, Merck Exploratory Science Center

• How can we scale?
• Moving towards ‘Human Body-on-a-Chip’
• Building additional organ-level micro-complexities? Hoe fare should we go and what are key determinants

Preclinical Strategies, Models & Tools in Oncology

Imaging in Drug Development

Charles Glaus, PhD, Director, Translational Research & Early Clinical, Takeda

• Emerging technologies for tissue analysis
• Advanced preclinical models for IO research
• Leveraging blood samples vs tissue biopsies
• Shortcomings of preclinical models for CAR-T drug development

External Collaboration in Preclinical and Translational Immuno-Oncology

Sybil Williams, PhD, Director, Biology Oncology Discovery, Merck

Michael Woo, Director, Head of Immuno-Oncology Search & Evaluation, External Innovation, Novartis

• Beyond checkpoint inhibitors and combinations with checkpoint inhibitors, what are future areas in IO to form external collaborations?
• Types of collaborations that pharma partners are open to
• How and when to approach a potential pharma partner
• Perspectives and insights from S&E and Biology Discovery

Chemical Biology and Target Validation

Recent Advances in Proteomics to Enable Target Discovery and Mechanism of Action Studies

Moderator: An Chi, PhD, Director, Chemical Biology Proteomics, Merck

• Recent development/trends of chemoproteomics strategies for target ID to follow up on hits from phenotypic screens
• Effective strategies to prioritize target hypothesis
• Omics use for MOA/hit prioritization in phenotypic screening campaigns today
• Best practice in assessing translatability of complex disease models

Chemical Probes as Tools for Target Validation

Moderator: Milka Kostic, PhD, Program Director, Chemical Biology, Department of Cancer Biology, Dana-Farber Cancer Institute

• What are the common strategies for validating chemical probes?
• How can we, as a community, most effectively share best practices for chemical probe use?
• What are different mechanisms for unrestricted sharing of high-quality chemical probes?
• How can we talk about the importance of chemical probes to the bottom-line?

CNS Targets and Translational Strategies

Lost in Translation? Asking the Right Questions in the Right Language

Moderator: Dario Doller, PhD., Drug Research Consulting, Alcyoneus/ScienceWorks

• CNS disease etiology understanding: Who owns it?
• What are the major disconnects between lab experimentation and clinical research?
• Are differences in physiology between preclinical species and human affecting the translational gap?
• Patient segmentation: Strategies and outcomes
• Correlation or causation? Importance of genetic links for different CNS diseases
• What are the attributes that have real impact minimizing risk during novel target selection?
• Human as a model for human – What is the future of experimental medicine in CNS drug discovery?

The Future of the Biomarker Toolbox in CNS Drug Development

Moderator: Andreas Jeromin, PhD, Scientific and Medical Advisor, Quanterix

• Current technology status of multimodal biomarkers in CNS drug development
• New frontiers of blood-based biomarkers
• Regulatory and clinical considerations for the implementation of biomarkers in clinical trials
• The future of biomarkers in clinical trials based on "real-word experience" studies

Optimizing Drug Metabolism & Pharmacokinetics

Key Issues Related to Drug Transporters in a Pharma R&D Setting

Moderators: Li Di, PhD, Research Fellow, Pharmacokinetics, Dynamics and Metabolism, Pfizer, Inc.
Manthena Varma, PhD, Associate Research Fellow, PDM, Medicine Design, Worldwide R&D, Pfizer, Inc.

• How can we best generate reliable in vitro transport kinetics and inhibition data?
• Many transporters do have overlapping substrate-specificity, how can we assess and quantify an individual transporters' contribution?
• Regulation of transporters (induction, disease, epigenetics) can significantly modulate overall transport capacity. How can this be integrated into current in vitro - in vivo extrapolations?

Use of Modeling Tools and Strategies for Predicting ADME-Tox Properties

Moderator to be announced

• Machine-learning or structure-based approaches for ADME-Tox prediction and optimization?
• Should the modeling tools for toxicity predicting be specific for xenobiotics and drugs?
• Quantum-mechanics methods for drug metabolism prediction

Lead Optimization for Biologics and Combination Therapies

Moderators: Alison Betts, PhD, Associate Research Fellow, Translational Modeling & Simulation, Biomedicine Design, Pfizer Worldwide R&D
Renu Singh Dhanikula, PhD, Senior Research Investigator, Metabolism and Pharmacokinetics, Bristol-Myers Squibb

• When are relevant assays and models for PKPD testing and/or safety pharmacology studies for biologics?
• What are some emerging safety concerns for biologics and what are potential strategies to de-risk them?
• Where are there gaps in understanding preclinical to clinical safety translation for biologics?

Immuno-Oncology Biomarkers 1: Predictive Biomarkers and Companion Diagnostics

Companion Dx for Immuno-Oncology

Christine Ward, PhD, Executive Director, Late Stage Oncology Translational Medicine Program Lead, Bristol-Myers Squibb

• Translation from IO biomarker to companion Dx
• Strategies to facilitate drug/Dx approval
• What makes a good companion Dx?

Biomarkers for Combination Immunotherapy

Moderator: Jianda Yuan, MD, PhD, Senior Director, Translational Oncology, Merck

• Tumor mutational burden and gene expression profiling biomarkers for personalized cancer immunotherapy
• Biomarkers to determine the right combination regimen for each patient
• Biomarkers for patient stratification for combination immunotherapy
• Establishing the right co-development strategy

Clinical and Translational Biomarkers

The Business of Fit-for-Purpose Biomarkers

Moderator: Iman Jilani, PhD, Director, Oncology Clinical Assay Group, Early Development, Pfizer

• Sample management
• Biomarker validation
• Final sample analysis data

Challenges and Practices in Effectively Managing CRO on Clinical Biomarker Sample Analysis

Moderator: Xuemei Zhao, PhD, Senior Principal Scientist, Translational Molecular Biomarkers, Merck Research Labs

• Analytical validity: clinical biomarker assay validation to meet the intended use
• Clinical validity: using validated clinical biomarker assay to qualify exploratory biomarker in clinical study
• Implementing clinically qualified biomarker to aid decision-making in clinical study to aid patient care in the clinic
  

Digital Health: Pharmaceutical Executive Summit

Artificial Intelligence: Clinical Applications

Moderator: Ting Shih, MBA, MS, CEO & Founder, ClickMedix

• AI for healthcare - what are effective applications?
• What does it take for AI to be useful? e.g. sufficient data, well trained analysts to understand the data
• What are cost-effective ways to start learning about AI and how to use it within enterprises?

Digital Therapeutics

Moderator: Joris Van Dam, PhD, Head, Digital Therapeutics, Novartis Institutes for BioMedical Research

• In what indications do you think patients could benefit from access to a digital therapeutic? Either in lieu of or adjunct to pharmacotherapy.
• What do you see as some of the main barriers to adoption of digital therapeutics in standard of care?
• How do you think digital therapeutics could benefit from continuous improvement based on real-world performance?

Clinical Innovation for Combination Immunotherapy

Optimizing for Value in the Age of Precision Medicine: Complications and Opportunities

Moderator: Nikolas T. Weissmueller, PhD, Associate Director, Strategic Options & Assessment, Business Insights & Analytics, R&D, Bristol-Myers Squibb

• Value-based IO development
• Probability of success assessment
• Use of computational methods to inform trial design choices

Benefit-Risk Evaluation of Immunotherapies Today: A Discussion of EMA/FDA Guidance for Industry

Moderator: Céline Adessi, PhD, Senior Group Director, Product Development, Safety Science Oncology, Licensing and Early Development, F. Hoffmann-La Roche

• Modernizing the reporting, collection, assessment of adverse events
• Patient reporting outcome
• Benefit-risk balance evaluation

Bispecific Antibodies for Immunotherapy

Moderator: Raphael Clynes, MD, PhD, Vice President, Translational Biology, Xencor

• T cell engaging bispecific antibodies
• Bispecifics in combination immunotherapy
• Engineering bispecific and multi-specific antibodies

THURSDAY, JUNE 20
7:15 - 8:10 AM

iPS Cells for Disease Modeling and Drug Discovery

iPSC-Based Disease Modeling

Moderator: Qin Wang, PhD, Scientist, Drug Safety Research and Evaluation, Takeda

• Utilizing patient-derived iPSCs to study disease pathogenesis and develop therapeutics in relevant genetic and cellular context
• Can human iPSCs systems replace animal models? How close are we (in lab and regulatory agencies)?
• What are the current challenges or weaknesses for adopting iPSCs-derived in vitro systems in discovery and toxicology?

Using iPSC for Drug Safety Screening

Moderators: Gary Gintant, PhD, Senior Research Fellow, AbbVie
Paul W. Burridge, PhD, Assistant Professor, Department of Pharmacology, Center for Pharmacogenomics, Northwestern University Feinberg School of Medicine

• What are concerns for using human induced pluripotent stem cell (iPSC)-derived cells to assess drug safety screening?
• How to consider standardizing assays and comparing results? Assay reproducibility?
• Generation of patient derived hiPSC-derived cardiomyocytes: present and future roles.
• The role of newer co-cultures and 3D structures in present and future efficacy and safety studies.
• How to best adapt hiPSCs for high-throughput phenotypic screening.

Patient-Derived Tumor Organoids

Deploying Bioengineered 3D Tumor Models/Organoids into Clinical Practice And The Pharmaceutical Pipeline

Moderator: Aaron Goldman, PhD , Director, Drug Resistance Group, Brigham and Women's Hospital, Faculty and Instructor in Medicine, Harvard Medical School, Breast Cancer Alliance Early Career Investigator, Harvard-MIT Health Sciences and Technology

• Where is the clinical enterprise and/or pharma in terms of seeing value in these model systems?
• How do we convince clinicians and more traditional scientists that these new technologies are important and effective?
• Complexity (more cell types or multiple organoids per model system) versus scale-up/high throughput

Developing Patient-Derived Tumoroids

Moderator: Alejandro Amador, PhD, GSK, Scientific Leader, Platform Biology Automation, GSK

• Evaluating tumor organoid protocols
• Characterizing growth characteristics
• Improving establishing success rates

Target Identification & Phenotypic Screening

Exploiting CRISPR for In vivo Screening: What You Need to Know Before and After

Moderators: Danilo Maddalo, PhD, Lab Head, ONC Pharmacology, Novartis Institutes for BioMedical Research, Novartis Pharma AG

• Overview of current strategies for in vivo screening for target identification
• Optimization and validation of current models
• Understanding inherent limitations and need for using complementary techniques
• Analyzing and interpreting the data

Exploring Artificial Intelligence/Machine Learning for Improving Drug Discovery

Moderators: Arvind Rao, PhD, Associate Professor, Department of Computational Medicine and Bioinformatics, University of Michigan
Deepak K. Rajpal, Senior Scientific Director, Computational Biology, GSK R&D
Johannes M. Freudenberg, Senior Scientific Investigator, Computational Biology, GSK R&D

• In silico modeling of complex cellular phenotypes and disease models: paradigms to bridge the computational-experimental gap
• Deconvolution of preclinical data and translating into clinical space: integrative modeling, generalizability, transportability and data harmonization
• Understanding limitations and where the opportunities lie: the power of scale and the complexity of medicine

Image-Based Approaches to Single-Cell 'Omics

Moderator: Jeffrey Moffitt, PhD, Assistant Professor, Program in Cellular and Molecular Medicine, Boston Children's Hospital, Department of Microbiology, Harvard Medical School

• State-of-the-art methods for massively multiplexed protein and RNA imaging
• Construction of spatially resolved cellular atlases and their potential in basic and clinical science
• Image-based approaches to high-throughput screening of pooled libraries of genetic perturbations

Blood-Brain Barrier

Project Human BBB Penetration from Preclinical Studies

Moderator: Guangqing Xiao, PhD, Associate Director, DMPK, Takeda

• In vitro and in vivo models to assess brain penetration
• Study design

Image-Guided Opening of the Blood Brain Barrier and Intra-Arterial Drug Delivery

Moderator: Piotr Walczak, MD, PhD, Associate Professor, Radiology, Johns Hopkins University

• Techniques for blood brain barrier opening (osmotic vs. FUS)
• Advantages of Intraarterial route for targeting the brain
• Need for image-guidance of BBB manipulation and drug targeting

Brain Regional Drug Disposition in CNS Diseases and Drug Development

Moderator: Irena Loryan, PhD, Researcher, Department of Pharmaceutical Biosciences, Translational PKPD, Uppsala University

• Brain regional drug disposition
• State of regional blood-brain barrier transport in Alzheimer’s disease
• How to address brain regional drug disposition in CNS drug development?

Predicting Drug Toxicity

Gaps in Translating Preclinical Findings to the Clinic

Moderators: William Proctor, PhD, DABT, Associate Director/Senior Scientist, Investigative Toxicology, Department of Safety Assessment, Genentech Inc.
Terry Van Vleet, PhD, DABT, Head of Molecular and Computational Toxicology, Department of Preclinical Safety, AbbVie

• Model/assay sensitivity – what level (or magnitude) of change can we detect?
• Do we have appropriate study design, data analysis, statistical analysis, and statistical power?
• How predictive are our preclinical findings/models to the clinic?
• What is it we are missing? Are there other useful models? Are their gaps in our translation knowledge?

Using iPSC for Drug Safety Screening

Moderators: Gary Gintant, PhD, Senior Research Fellow, AbbVie
Paul W. Burridge, PhD, Assistant Professor, Department of Pharmacology, Center for Pharmacogenomics, Northwestern University Feinberg School of Medicine

• What are concerns for using human induced pluripotent stem cell (iPSC)-derived cells to assess drug safety screening?
• How to consider standardizing assays and comparing results? Assay reproducibility?
• Generation of patient derived hiPSC-derived cardiomyocytes: present and future roles.
• The role of newer co-cultures and 3D structures in present and future efficacy and safety studies.
• How to best adapt hiPSCs for high-throughput phenotypic screening.

Use of 3D and Microphysiological Systems for Drug Safety Assessments

Moderators: Bo Yeon Lee, PhD, Scientific Program Manager, Office of the Director, Tissue Chip for Drug Screening Program, National Center for Advancing Translational Sciences, National Institutes of Health
Sophie Lelievre, DVM, PhD, LLM, Professor, Cancer Pharmacology, Purdue University College of Veterinary Medicine

• Update on benchmarking studies covering emerging cell models and 3D platforms
• How to best integrate on-a-chip devices with other toxicity studies
• Speed, accuracy and reliability of tissue-on-a-chip systems
• How to utilize these systems for predicting toxicity, efficacy and designing clinical trials

Immuno-Oncology Biomarkers 2: Immune Profiling and Immune Monitoring

Exosomes as IO Biomarkers

Moderator: Samir Hanash, MD, PhD, Director, McCombs Institute for Cancer Early Detection and Treatment, MD Anderson Cancer Center

• Exosomes as a window on tumor molecular profile
• The role of exosomes in tumor monitoring
• The role of exosomes in the cyber war between the tumor and the immune system

Genomic Immuno-Oncology Biomarkers

Moderator: Jennifer J.D. Morrissette, Scientific Director, Clinical Cancer Cytogenetics, Clinical Director, Center for Personalized Diagnostics, University of Pennsylvania

• What is necessary for calling TMB in the laboratory: What should be the inclusion criteria for variants in the TMB calculation and should there be a cut-off based on tumor type or an overall number of mutations per megabase?
• Is matched germline testing necessary for TMB prediction? Does it vary depending on tumor type?
• What other factors may be associated with immunotherapy treatment response in addition to TMB?

Digital Biomarkers: Biosensors, Wearables, and mHealth

mHealth: Implementation and Regulatory Strategies

Moderators: Yu-Feng (Yvonne) Chan, MD, PhD, Associate Professor, Genetics & Genomics Sciences, Icahn School of Medicine; Director, Digital Health & Personalized Medicine, Icahn Institute for Genomics and Multiscale Biology at Mt. Sinai
Michael J. Benecky, PhD, Senior Director, Global Regulatory Affairs, Precision and Digital Medicine, R&D Chief Regulatory Office, GlaxoSmithKline

• FDA Software Pre-Certification Program
• FDA Draft Guidance on Prescription Drug User Software
• Digital Health Regulation in the EU

Biosensors and Wearables as Digital Biomarkers

Moderator: Gergely Vértes, MBA, Solution Accelerator Lead, Wearable for Epilepsy, UCB Pharma

• Patient reported outcome enhanced with digital reported outcome
• Regulatory acceptance and inclusion into clinical trials
• New development paradigm (so far Drug vs. Placebo; future state of art vs. new compounds)
• 35 anti-epileptic on the market. Who will create the baseline with digital reported outcomes

Real-World Evidence

Moderator: Wasim Malik, PhD, Professor, Harvard Medical School; Co-Founder & Managing Partner, Iaso Ventures

• Platform approach to real-world evidence
• Technology-enabled approaches to clinical trials
• Standardization of digital biomarkers to facilitate data sharing

Digital Biomarkers as Clinical Endpoints

Moderator: Jeffrey Kaye, MD, Layton Professor, Neurology & Biomedical Engineering, Director, ORCATECH - Oregon Center for Aging & Technology, Oregon Health & Science University

• Scientific, operational, and financial challenges in implementing digital biomarkers in clinical trials
• Reducing time needed to obtain early meaningful signals of clinical efficacy and safety
• Evaluating wearable and mobile assessments; regulatory considerations

Small Molecules for Immuno-Oncology Therapeutics

Why is STING so IntereSTING as an IO Target?

Moderators:

Jeremy Presland, PhD, Principal Scientist, Discovery Pharmacology, Merck Research Labs Boston

Gottfried Schroeder, PhD, Senior Scientist, Department of Pharmacology, Merck Research Labs Boston

• Distinct structural features of STING to design modulators against
• Biologics v. small molecule approaches for modulating STING
• MOA and physiological considerations, possible side effects, etc.

Targeting the Tumor Microenvironment

Moderator: Earp, PhD, Director, University of North Carolina Lineberger Cancer Center and Co-Founder, Meryx Pharmaceuticals

• Types of Tumor-associated Macrophages (TAMs) in the TME
• Promising intracellular kinase targets in TAMs
• Other TAM targets

Targeting the IDO/TDO - Kynurenine - Arylhydrocarbon Receptor Pathway and Beyond

Moderators: Claus Kremoser, PhD, CEO, Phenex Pharmaceuticals

Sheena Pinto, PhD, Senior Scientist, Drug Discovery, Phenex Pharmaceuticals

• What are the pros and cons of IDO / TDO single / dual inhibitors as opposed to AHR antagonists?
• Which tumor autonomous effects and which effects on the immune system can you expect?
• For which cancers might such IDO / TDO / AHR inhibitors be applicable?