TUESDAY, NOVEMBER 6 

8:00-8:20 am Morning Coffee

8:20-8:30 Chairperson’s Remarks


NOVEL INDICATIONS DEVELOPMENT- NEW STRATEGIES FOR OLD DRUGS 

8:30-9:00 Efficient Strategies to Identify & Validate Novel Indications for Patient-Ready Molecules

Speaker to be Announced

Presentation will discuss the strategies to successfully implement drug repositioning/repurposing within your current development portfolio. The discussion will focus on assets that are “patient-ready” and do not require additional discovery-phase evaluation.

9:00-9:30 Systematic Drug Repositioning: Can Computational Methods Work

Pankaj Agarwal, Ph.D., Director, Computational Biology, GlaxoSmithKline

Drug repositioning offers the potential to create value for patients by accelerating time to proof of concept studies. We will discuss two recent methods that can systematically identify disease indications for drugs. The first one uses genetics associations (from Genome Wide Association Studies – GWAS) to find new indications for drugs some of which are being validated experimentally. The second one frames the side effects of drugs as pharmacological effects, relies on the observation that many drugs that treat a disease have similar side effects, and then predicts other drugs with similar side effects as potential treatments for the disease.

9:30-10:00 Looking Forward: Impact of the NCATS Drug Repositioning & Repurposing Initiative on PoC Studies

Christine Colvis, Ph.D., Director, Therapeutics Discovery Program, National Center for Advancing Translational Sciences, NIH

The mission of the National Center for Advancing Translational Sciences is to catalyze the generation of innovative strategies, methods and technologies that enhance the development, testing and implementation of diagnostics and therapeutics.  Programs and concepts to advance translational science will be presented.

10:00-10:40 Coffee Break in the Exhibit Hall


NOVEL ALTERNATE ENDPOINTS FOR CLINICAL PoC 

10:40-11:10 Sponsored Presentation (Opportunity Available) 

11:10-11:40 CASE STUDY: Intratumoral Heterogeneity: Impact on Personalized Medicine and Biomarker Development

Khurum Khan, Ph.D., MBBS, Specialist Registrar and Clinical Research Fellow, Drug Development Unit, Royal Marsden NHS Foundation Trust, London, United Kingdom

Most advanced solid tumors remain incurable, with resistance to chemotherapeutics and targeted therapies a common cause of poor clinical outcome. Intratumor heterogeneity may contribute to this failure by initiating phenotypic diversity enabling drug resistance to emerge and by introducing tumor sampling bias. Envisaging tumor growth as a Darwinian tree with the trunk representing ubiquitous mutations and the branches representing heterogeneous mutations may help in drug discovery and the development of predictive biomarkers of drug response.

11:40-12:10pm Composite Endpoints in Clinical Trials – Continuing Controversies and Recent Developments

Cono Ariti, Ph.D., Lecturer, London School of Hygiene and Tropical Medicine

Composite endpoints are a common feature of many clinical trials. However, questions remain over their appropriate design, analysis, interpretation and reporting. This talk will examine the use and abuse of composite endpoints in recently reported clinical trials. I will summarize contemporary practice and introduce a number of new approaches to dealing with the analysis of composite endpoints that attempt to better reflect trial outcomes that are capturing the attention of trial investigators, sponsors, statisticians and regulators will be described.