Day One

September 26th, 2012

08.20 Registration, Coffee and Networking

08.50 Chairman’s Opening Remarks

Brandon Jeffy, Principal Scientist, Exploratory Toxicology, Celgene

In Vitro Models: Enhancing Their Predictive Capacity And In Vivo Relevance

09.00 An Integrative Approach to Preclinical Data for Improved In Vitro – In Vivo Clinical Translation

• Expectations of preclinical data and evolving technologies

• Can integrative approaches improve preclinical-clinical translation?

Gary Gintant, Senior Group Leader, Abbott

09.30 Translating Preclinical Cardiovascular Biomarker Identification to Improved Clinical Safety

• Cardiac safety implications of ion channel inhibition

• Recent developments in drug induced vascular injury

Peter Hoffmann, Executive Director, Preclinical Safety, Novartis

10.00 Strategic Use of Translational In Vitro Models in Non-Clinical Toxicology

• Characterization of conserved in vitro – in vivo mechanisms in toxicological models

• Appropriate uses and limitations of in vitro models to predict human toxicities

Brandon Jeffy, Principal Scientist, Exploratory Toxicology, Celgene

10.30 Immune Function Biomarkers as Signposts of Therapeutic Efficacy

• Enhancing cell-based biomarkers to assess pharmacological efficacy
• Implementing early trial efficacy assessments to direct clinical strategy

Julie Wilkinson, Director of Pharma Services, Chief Information Officer, Immunosite

10.45 Morning Refreshments and Speed Networking

12.15 Designing an In Vitro Panel of Assays to Predict In Vivo Toxicity

• Additional value is gained by the inclusion of selected in vitro assays that reflect broad mechanisms of toxicity, including mitochondrial function, apoptosis, and cytotoxicity

Karen Leach, Associate Research Fellow, Pfizer

12.45 How Predictive Can We Be?

• Which of the new breed of in vitro technologies hold the most promise?

• Will animal models ever be fully replaced?

• Future directions for in vitro models in drug development

Gary Gintant, Senior Group Leader, Abbott

Peter Hoffmann, Executive Director, Preclinical Safety, Novartis

Joe Senn, Lead - Issue Management, Drug Safety Evaluation, Millennium

1.15 Lunch

Unlocking The Power Of Stem Cell Models To Facilitate Rapid Drug Development

2.15 Stem Cell Based Safety Assessment Strategies in Drug Development

• How stem cells are helping to establish reliable human cellular models with improved reproducibility and predictability

• High throughput screening, human disease models and safety and toxicology testing

Hirdesh Uppal, Scientist, Genentech

2.45 Implementing iPS Cell Based Screens in Early Drug Development

• Implementing a human iPSC derived cardiomyocyte assay for assessing risk of drug induced arrhythmia

• The hPST assay: A high-throughput human PSC based assay for assessing teratogenic risk

Eric Chiao, Head, Pluripotent Stem Cell Lab, Non-Clincal Safety, Roche

Effective Validation Of New In Vitro Technologies

3.15 Validating and Qualifying Biomarkers: An FDA Success Story

• What impact are regulatory processes having on collaborative efforts to develop new biomarkers?

• Effective pathways for acceptance of biomarkers

Federico Goodsaid, VP, Strategic Regulatory Intelligence, Vertex Pharmaceuticals

3.45 Afternoon Refreshment Break

4.15 Novel In Vitro Models in Toxicology: The Long Way From An Exploratory to a Trusted Tool

• Different approaches & examples for complex in vitro models i.e. 3D models for organ toxicity

• Outlook: What is needed for exploratory tools to become trusted tools?

Adrian Roth, Section Head, Mechanistic Safety, Non-Clinical Safety, Roche

4.45 Jumping Through the Hoops: The Pathway to Technological Validation

• An in vitro mechanistic study indicated that leflunomideinduced liver injury may involve toxic reactive metabolites

• Clinical data showed that leflunomide-induced liver injury likely associates with CYP polymorphism or coadministration of CYP-inhibitory drugs

Xi Yang, Toxicologist, FDA

5.15 Overview of the In Vitro Assay Models of the US Tox21 Program

• qHTS-based compound testing to streamline safety profiling

• The Tox21 initiative’s validation of novel screening assays

Menghang Xia, Group Leader, National Center for Advancing Translational Sciences

5.45 Chairman’s Closing remarks