Web Desc
Modern Phenotypic Drug Discovery: Defining the Path Forward
Organizer(s): Jonathan A. Lee, Ellen L. Berg and Eugene C. Butcher
Date: April 02 - 06, 2016
Location: Big Sky Resort, Big Sky, MT, USA
Sponsored by Abide Therapeutics Inc., Janssen R&D: Pharmaceutical Companies of Johnson & Johnson, Merck & Co., Inc., Pfizer Inc., Takeda Pharmaceutical Company Limited and Vertex Pharmaceuticals Incorporated
Summary of Meeting:
Innovation and productivity in pharmaceutical drug discovery have been below expectations. Surprisingly, more first-in-class small molecule drugs approved by the FDA (1999-2008) were identified by functional approaches reminiscent of pre-genomics pharmacology (phenotypic drug discovery) than by contemporary molecular targeted strategies. This observation, and the unexpected difficulties of target validation, have diminished the impact of the “genomics revolution” and are arguably associated with the decline in innovation and productivity of pharmaceutical research. As a result, pharmaceutical researchers have begun to reevaluate the “Molecular Mindset” which has dominated our collective drug-hunting culture, and explore modern approaches to phenotypic screening. However, there is currently no single scientific forum where scientists from pharma, biotech, academia and instrument/service providers can meet and discuss strategies/issues related to phenotypic drug discovery (PDD). In part, this is because PDD encompasses multiple therapeutic areas and involves diverse disciplines including drug discovery, chemistry, cell biology, stem cell biology, systems biology, genomics, bioengineering and informatics. This Keystone Symposia meeting uniquely provides this interdisciplinary environment and is a particularly attractive venue given the increasing role of academia in drug discovery research. The conference will be a forum for the global PDD research community in which scientists from diverse institutions and scientific disciplines can meet to share/discuss/debate topics related to advantages/disadvantages of PDD and how PDD can complement targeted approaches. Significantly, the conference will provide a much needed forum for the growing interest in PDD, and can conceivably become the cornerstone of the global scientific movement for the reintroduction of functional biology/physiology-driven pharmaceutical research.
Scholarship Deadline: December 2 2015
Discounted Abstract Deadline: December 2 2015
Abstract Deadline: January 5 2016
Discounted Registration Deadline: February 2 2016
Keystone Symposia thanks our Sponsor(s) for generously supporting this meeting:
Abide Therapeutics Inc.Janssen R&D: Pharmaceutical Companies of Johnson & JohnsonMerck & Co., Inc.Pfizer Inc.Takeda Pharmaceutical Company LimitedVertex Pharmaceuticals Incorporated
We gratefully acknowledge additional support for this conference from:
Evercyte GmbH
We gratefully acknowledge additional in-kind support for this conference from those foregoing speaker expense reimbursements:

Emulate, Inc.

Novartis Institutes for BioMedical Research

Pfizer Inc.

Summit Therapeutics Inc

Sunovion Pharmaceuticals Inc.
We gratefully acknowledge the generous grant for this conference provided by:

National Center for Advancing Translational Sciences (NCATS)
Grant No. 1R13TR001586-01
Funding for this conference was made possible (in part) by (1R13 TR001586-01) from the National Center for Advancing Translational Sciences. The views expressed in written conference materials or publications and by speakers and moderators do not necessarily reflect the official policies of the Department of Health and Human Services; nor does mention of trade names, commercial practices, or organizations imply endorsement by the U.S. Government.
We appreciate the organizations that provide Keystone Symposia with additional support, such as marketing and advertising:

Click here to view more of these organizations
Special thanks to the following for their support of Keystone Symposia initiatives to increase participation at this meeting by scientists from underrepresented backgrounds:

Click here to view more of these organizations

Program

Saturday, April 02 | 4:00PM - 8:00PM
Arrival and Registration
Room: Lower Atrium


Saturday, April 02 | 6:00PM - 8:00PM
Welcome Mixer
Room: Jefferson
No registration fees are used to fund alcohol served at this function.

Sunday, April 03 | 7:00AM - 8:00AM
Breakfast
Room: Gallatin


Sunday, April 03 | 8:00AM - 8:30AM
Welcoming Remarks
Room: Jefferson/Madison

Speaker 3 of 3
* Eugene C. Butcher, Stanford University, USA

Sunday, April 03 | 8:00AM - 8:30AM
Welcoming Remarks
Room: Jefferson/Madison

Speaker 2 of 3
* Ellen L. Berg, Eurofins DiscoverX, USA

Sunday, April 03 | 8:00AM - 8:30AM
Welcoming Remarks
Room: Jefferson/Madison

Speaker 1 of 3
* Jonathan A. Lee, PDD4Patients LLC, USA

Sunday, April 03 | 8:30AM - 9:30AM
Keynote Address
Room: Jefferson/Madison

Speaker 2 of 2
David C. Swinney, iRND3, Institute for Rare and Neglected Diseases, USA
The Value of Phenotypic Assays to Drug Discovery: Historical Perspective

Sunday, April 03 | 8:30AM - 9:30AM
Keynote Address
Room: Jefferson/Madison

Speaker 1 of 2
* Eugene C. Butcher, Stanford University, USA

Sunday, April 03 | 9:30AM - 9:50AM
Coffee Break
Room: Lower Atrium


Sunday, April 03 | 9:30AM - 11:00AM
Why Revisit PDD?
Room: Jefferson/Madison

Speaker 3 of 3
Bernard H. Munos, FasterCures, USA
Pharma Research Productivity: A New Perspective on Phenotypic vs. Target-Based Screening

Sunday, April 03 | 9:30AM - 11:00AM
Why Revisit PDD?
Room: Jefferson/Madison

Speaker 2 of 3
John Moffat, Genentech, Inc., USA
Phenotypic and Targeted Drug Discovery Are Complimentary Strategies

Sunday, April 03 | 9:30AM - 11:00AM
Why Revisit PDD?
Room: Jefferson/Madison

Speaker 1 of 3
* Eugene C. Butcher, Stanford University, USA

Sunday, April 03 | 11:00AM - 1:00PM
Poster Setup
Room: Gallatin


Sunday, April 03 | 11:00AM - 5:00PM
On Own for Lunch


Sunday, April 03 | 1:00PM - 10:00PM
Poster Viewing
Room: Gallatin


Sunday, April 03 | 4:30PM - 5:00PM
Coffee Available
Room: Lower Atrium


Sunday, April 03 | 5:00PM - 7:00PM
PDD: Recent Clinical Successes
Room: Jefferson/Madison

Speaker 5 of 5
Veronica Soloveva, Merck, USA
Short Talk: Identification and Characterization of the Antiviral Activity of GS-5734 using Phenotypic Approaches

Sunday, April 03 | 5:00PM - 7:00PM
PDD: Recent Clinical Successes
Room: Jefferson/Madison

Speaker 4 of 5
Jon Tinsley, Summit Therapeutics, UK
Modulators of Utrophin Production for the Treatment of Duchenne Muscular Dystrophy

Sunday, April 03 | 5:00PM - 7:00PM
PDD: Recent Clinical Successes
Room: Jefferson/Madison

Speaker 3 of 5
Rebecca M. Pruss, Windhover Biomed, France
Maintenance of Motor Function in Spinal Muscular Atrophy by Olesoxime: A Recent Clinical Success for Phenotypic Drug Discovery

Sunday, April 03 | 5:00PM - 7:00PM
PDD: Recent Clinical Successes
Room: Jefferson/Madison

Speaker 2 of 5
Nicholas A. Meanwell, Bristol-Myers Squibb, USA
Phenotypic Screening for Antiviral Agents

Sunday, April 03 | 5:00PM - 7:00PM
PDD: Recent Clinical Successes
Room: Jefferson/Madison

Speaker 1 of 5
* Ellen L. Berg, Eurofins DiscoverX, USA

Sunday, April 03 | 7:00PM - 8:00PM
Social Hour with Lite Bites
Room: Gallatin
No registration fees are used to fund alcohol served at this function.

Sunday, April 03 | 7:30PM - 10:00PM
Poster Session 1
Room: Gallatin


Monday, April 04 | 7:00AM - 8:00AM
Breakfast
Room: Gallatin


Monday, April 04 | 8:00AM - 11:00AM
Challenges to Modern PDD/Lessons Learned
Room: Jefferson/Madison

Speaker 1 of 6
* Jonathan A. Lee, PDD4Patients LLC, USA

Monday, April 04 | 8:00AM - 11:00AM
Challenges to Modern PDD/Lessons Learned
Room: Jefferson/Madison

Speaker 2 of 6
Neil O. Carragher, University of Edinburgh, UK
Accelerating PDD through Integrating MOA Profiling with Agile Chemical Design: Shifting the Bottlenecks from Target Deconvolution to Disease Relevance

Monday, April 04 | 8:00AM - 11:00AM
Challenges to Modern PDD/Lessons Learned
Room: Jefferson/Madison

Speaker 3 of 6
Ellen L. Berg, Eurofins DiscoverX, USA
Phenotypic Chemical Biology for Predicting Efficacy and Safety

Monday, April 04 | 8:00AM - 11:00AM
Challenges to Modern PDD/Lessons Learned
Room: Jefferson/Madison

Speaker 4 of 6
Fabien Vincent, Pfizer Inc., USA
Lessons Learned in Phenotypic Screening

Monday, April 04 | 8:00AM - 11:00AM
Challenges to Modern PDD/Lessons Learned
Room: Jefferson/Madison

Speaker 5 of 6
Michael R. Jackson, SBP Medical Discovery Institute, USA
Phenotypic Screening of Patient Derived Cells: Balancing Throughput with Relevance

Monday, April 04 | 8:00AM - 11:00AM
Challenges to Modern PDD/Lessons Learned
Room: Jefferson/Madison

Speaker 6 of 6
Bridget Wagner, Broad Institute, USA
Short Talk: Small-Molecule Stimulation of Human Beta-Cell Proliferation

Monday, April 04 | 9:20AM - 9:40AM
Coffee Break
Room: Lower Atrium


Monday, April 04 | 11:00AM - 12:00PM
Panel 1: PDD Trends and Roadblocks
Room: Jefferson/Madison

Speaker 1 of 7
* David C. Swinney, iRND3, Institute for Rare and Neglected Diseases, USA

Monday, April 04 | 11:00AM - 12:00PM
Panel 1: PDD Trends and Roadblocks
Room: Jefferson/Madison

Speaker 2 of 7
Ellen L. Berg, Eurofins DiscoverX, USA

Monday, April 04 | 11:00AM - 12:00PM
Panel 1: PDD Trends and Roadblocks
Room: Jefferson/Madison

Speaker 3 of 7
Jonathan A. Lee, PDD4Patients LLC, USA

Monday, April 04 | 11:00AM - 12:00PM
Panel 1: PDD Trends and Roadblocks
Room: Jefferson/Madison

Speaker 4 of 7
Bernard H. Munos, FasterCures, USA

Monday, April 04 | 11:00AM - 12:00PM
Panel 1: PDD Trends and Roadblocks
Room: Jefferson/Madison

Speaker 5 of 7
Fabien Vincent, Pfizer Inc., USA

Monday, April 04 | 11:00AM - 12:00PM
Panel 1: PDD Trends and Roadblocks
Room: Jefferson/Madison

Speaker 6 of 7
Neil O. Carragher, University of Edinburgh, UK

Monday, April 04 | 11:00AM - 12:00PM
Panel 1: PDD Trends and Roadblocks
Room: Jefferson/Madison

Speaker 7 of 7
Michael R. Jackson, SBP Medical Discovery Institute, USA

Monday, April 04 | 12:00PM - 1:00PM
Poster Setup
Room: Gallatin


Monday, April 04 | 12:00PM - 2:30PM
On Own for Lunch


Monday, April 04 | 1:00PM - 10:00PM
Poster Viewing
Room: Gallatin


Monday, April 04 | 4:30PM - 5:00PM
Coffee Available
Room: Lower Atrium


Monday, April 04 | 5:00PM - 7:00PM
New Biological Models
Room: Jefferson/Madison

Speaker 1 of 5
Eugen Dhimolea, Dana Farber Cancer Institute, Harvard Medical School, USA
Engineering 3-D in vitro Tumor Models of Antineoplastic Drug Resistance

Monday, April 04 | 5:00PM - 7:00PM
New Biological Models
Room: Jefferson/Madison

Speaker 2 of 5
Salman Khetani, University of Illinois at Chicago, USA
Microengineered Human Liver Models for Drug Screening and Disease Modeling

Monday, April 04 | 5:00PM - 7:00PM
New Biological Models
Room: Jefferson/Madison

Speaker 3 of 5
Sandy Hinckley, Sanford Burnham Prebys Medical Discovery Institute, USA
Short Talk: Phenotypic Screening of Neuronal Networks using High-throughput Multielectrode Arrays

Monday, April 04 | 5:00PM - 7:00PM
New Biological Models
Room: Jefferson/Madison

Speaker 4 of 5
Deborah Greene Nguyen, Organovo, USA
Short Talk: Bioprinted Three-dimensional (3D) Human Tissues for Toxicology and Disease Modeling

Monday, April 04 | 5:00PM - 7:00PM
New Biological Models
Room: Jefferson/Madison

Speaker 5 of 5
David Gerber, Galenea Corp., USA
Short Talk: Targeting Synaptic Function for CNS Drug Discovery

Monday, April 04 | 7:00PM - 8:00PM
Social Hour with Lite Bites
Room: Gallatin
No registration fees are used to fund alcohol served at this function.

Monday, April 04 | 7:30PM - 10:00PM
Poster Session 2
Room: Gallatin


Tuesday, April 05 | 7:00AM - 8:00AM
Breakfast
Room: Gallatin


Tuesday, April 05 | 8:00AM - 11:00AM
Translational PDD: Physiologically Relevant Models
Room: Jefferson/Madison

Speaker 1 of 7
* Fabien Vincent, Pfizer Inc., USA

Tuesday, April 05 | 8:00AM - 11:00AM
Translational PDD: Physiologically Relevant Models
Room: Jefferson/Madison

Speaker 2 of 7
Andrew Reaume, Melior Discovery, Inc., USA
Case Studies and Overall Learnings from 10 Years of Phenotypic Screening with a Broad-Based Panel of In Vivo Models

Tuesday, April 05 | 8:00AM - 11:00AM
Translational PDD: Physiologically Relevant Models
Room: Jefferson/Madison

Speaker 3 of 7
Mark Mercola, Stanford University, USA
Functional and Chemical Genomics to Decipher Heart Failure Mechanisms

Tuesday, April 05 | 8:00AM - 11:00AM
Translational PDD: Physiologically Relevant Models
Room: Jefferson/Madison

Speaker 4 of 7
Tom Large, Blue Oak Pharmaceuticals, USA
Discovery of Novel Psychiatric Drugs using a Target-agnostic, Systems Neurobiology Strategy

Tuesday, April 05 | 8:00AM - 11:00AM
Translational PDD: Physiologically Relevant Models
Room: Jefferson/Madison

Speaker 5 of 7
Jason Ptacek, Ionpath, Inc, USA
Short Talk: Phenotypic Human Primary Cell Based Tumor Microenvironment Models for Evaluation of Drug Combinations for Immune Oncology

Tuesday, April 05 | 8:00AM - 11:00AM
Translational PDD: Physiologically Relevant Models
Room: Jefferson/Madison

Speaker 6 of 7
David A. Mann, Cellular Dynamics International Inc., USA
Short Talk: Relevant Phenotypic Drug Discovery: Innate, Induced and Engineered Human iPSC Models

Tuesday, April 05 | 8:00AM - 11:00AM
Translational PDD: Physiologically Relevant Models
Room: Jefferson/Madison

Speaker 7 of 7
Denise V. Barrault, University of Edinburgh, UK
Short Talk

Tuesday, April 05 | 9:20AM - 9:40AM
Coffee Break
Room: Lower Atrium


Tuesday, April 05 | 11:00AM - 11:30AM
NIH Common Fund Program
Room: Jefferson/Madison

Speaker 1 of 1
Aaron Pawlyk, Eunice Kennedy Shriver National Institute of Child Health and Human Development, USA
In Search of Phenotypes to Illuminate the Druggable Genome

Tuesday, April 05 | 11:30AM - 1:00PM
Poster Setup
Room: Gallatin


Tuesday, April 05 | 11:30AM - 5:00PM
On Own for Lunch


Tuesday, April 05 | 1:00PM - 10:00PM
Poster Viewing
Room: Gallatin


Tuesday, April 05 | 4:30PM - 5:00PM
Coffee Available
Room: Lower Atrium


Tuesday, April 05 | 5:00PM - 7:00PM
Biologically Relevant Chemical Diversity
Room: Jefferson/Madison

Speaker 1 of 4
* Paul Clemons, Broad Institute of Harvard and MIT, USA
Chemical Diversity Expressed through Small-Molecule Performance: Diverse Readouts, Phenotypes and Genetic Determinants

Tuesday, April 05 | 5:00PM - 7:00PM
Biologically Relevant Chemical Diversity
Room: Jefferson/Madison

Speaker 2 of 4
Herbert Waldmann, Max Planck Institute for Molecular Physiology, Germany
Natural Product Inspired Compound Collections: Evolutionary Principle, Chemical Synthesis, Phenotypic Screening and Target Identification

Tuesday, April 05 | 5:00PM - 7:00PM
Biologically Relevant Chemical Diversity
Room: Jefferson/Madison

Speaker 3 of 4
Nicole E. Bodycombe, Pfizer, USA
Compound Subsets in Phenotypic Screening

Tuesday, April 05 | 5:00PM - 7:00PM
Biologically Relevant Chemical Diversity
Room: Jefferson/Madison

Speaker 4 of 4
Andrea Galmozzi, The Scripps Research Institute, USA
Short Talk: Phenotypic Screening of Fully Functionalized Fragment-Based Chemical Probes Reveals PGRMC2 As A New Regulator of Adipocyte Physiology of Therapeutic Potential for Obesity-Linked Diabetes

Tuesday, April 05 | 7:00PM - 8:00PM
Social Hour with Lite Bites
Room: Gallatin
No registration fees are used to fund alcohol served at this function.

Tuesday, April 05 | 7:30PM - 10:00PM
Poster Session 3
Room: Gallatin


Wednesday, April 06 | 7:00AM - 8:00AM
Breakfast
Room: Gallatin


Wednesday, April 06 | 8:00AM - 9:00AM
Keynote Address
Room: Jefferson/Madison

Speaker 1 of 2
* John Moffat, Genentech, Inc., USA

Wednesday, April 06 | 8:00AM - 9:00AM
Keynote Address
Room: Jefferson/Madison

Speaker 2 of 2
Enrique Saez, The Scripps Research Institute, USA
Chemical Proteomic Strategies to Advance Phenotypic Screening

Wednesday, April 06 | 9:00AM - 11:30AM
Target/MOA Identification/Deconvolution
Room: Jefferson/Madison

Speaker 1 of 6
* John Moffat, Genentech, Inc., USA

Wednesday, April 06 | 9:00AM - 11:30AM
Target/MOA Identification/Deconvolution
Room: Jefferson/Madison

Speaker 2 of 6
Monica Schenone, Pfizer Inc., USA
Target Identification and Mechanism of Action in Drug Discovery

Wednesday, April 06 | 9:00AM - 11:30AM
Target/MOA Identification/Deconvolution
Room: Jefferson/Madison

Speaker 3 of 6
Jeremy Jenkins, Novartis Institutes for BioMedical Research, USA
Systems Approaches to Elucidating Compound-Effect Pathways

Wednesday, April 06 | 9:00AM - 11:30AM
Target/MOA Identification/Deconvolution
Room: Jefferson/Madison

Speaker 4 of 6
Andreas Bender, University of Cambridge, UK
Towards Understanding Phenotypic Readouts - Utilizing Multiple Types of Chemical and Biological Data to Rationalize Compound Action

Wednesday, April 06 | 9:00AM - 11:30AM
Target/MOA Identification/Deconvolution
Room: Jefferson/Madison

Speaker 5 of 6
Vladimir Chupakhin, Janssen Pharmaceuticals, Belgium
Short Talk: The New Approaches for a Computational Target Deconvolution of the Phenotypic Hits

Wednesday, April 06 | 9:00AM - 11:30AM
Target/MOA Identification/Deconvolution
Room: Jefferson/Madison

Speaker 6 of 6
Susanne Swalley, Biogen, USA
Short Talk: Phenotypic Drug Discovery in SMA: Parallel Efforts in Preclinical Development and Target Identification

Wednesday, April 06 | 9:40AM - 10:00AM
Coffee Break
Room: Lower Atrium


Wednesday, April 06 | 11:30AM - 12:30PM
Panel 2: Identification of Molecular Targets and Biological
'Mechanisms of Action': How Much is Enough?
Room: Jefferson/Madison

Speaker 1 of 6
* Eugene C. Butcher, Stanford University, USA

Wednesday, April 06 | 11:30AM - 12:30PM
Panel 2: Identification of Molecular Targets and Biological
'Mechanisms of Action': How Much is Enough?
Room: Jefferson/Madison

Speaker 2 of 6
Susanne Swalley, Biogen, USA

Wednesday, April 06 | 11:30AM - 12:30PM
Panel 2: Identification of Molecular Targets and Biological
'Mechanisms of Action': How Much is Enough?
Room: Jefferson/Madison

Speaker 3 of 6
Tom Large, Blue Oak Pharmaceuticals, USA

Wednesday, April 06 | 11:30AM - 12:30PM
Panel 2: Identification of Molecular Targets and Biological
'Mechanisms of Action': How Much is Enough?
Room: Jefferson/Madison

Speaker 4 of 6
Marco Prunotto, F. Hoffmann-La Roche Ltd., Switzerland

Wednesday, April 06 | 11:30AM - 12:30PM
Panel 2: Identification of Molecular Targets and Biological
'Mechanisms of Action': How Much is Enough?
Room: Jefferson/Madison

Speaker 5 of 6
Nicholas A. Meanwell, Bristol-Myers Squibb, USA

Wednesday, April 06 | 11:30AM - 12:30PM
Panel 2: Identification of Molecular Targets and Biological
'Mechanisms of Action': How Much is Enough?
Room: Jefferson/Madison

Speaker 6 of 6
Rebecca M. Pruss, Windhover Biomed, France

Wednesday, April 06 | 12:30PM - 5:00PM
On Own for Lunch


Wednesday, April 06 | 4:30PM - 5:00PM
Coffee Available
Room: Lower Atrium


Wednesday, April 06 | 5:00PM - 7:00PM
Network Biology: New Technologies and Informatics
Room: Jefferson/Madison

Speaker 1 of 4
Andrea Califano, Columbia University, USA
Systematic Elucidation of Druggable Dependencies in Human Malignancies: A New Take on Precision Medicine

Wednesday, April 06 | 5:00PM - 7:00PM
Network Biology: New Technologies and Informatics
Room: Jefferson/Madison

Speaker 2 of 4
Sean Bendall, Stanford University, USA
Revealing Hidden Behavior One Cell at a Time

Wednesday, April 06 | 5:00PM - 7:00PM
Network Biology: New Technologies and Informatics
Room: Jefferson/Madison

Speaker 3 of 4
Anne E. Carpenter, Broad Institute of Harvard and MIT, USA
Increasing the Content of High-Content Phenotypic Assays: Targeting Diseases and Characterizing Compounds via Cell Painting and Image-Based Profiling

Wednesday, April 06 | 5:00PM - 7:00PM
Network Biology: New Technologies and Informatics
Room: Jefferson/Madison

Speaker 4 of 4
* Marco Prunotto, F. Hoffmann-La Roche Ltd., Switzerland
Short Talk: Application of Molecular Phenotype to Phenotypic Drug Discovery: Results of a PoC Study

Wednesday, April 06 | 7:00PM - 8:00PM
Social Hour with Lite Bites
Room: Gallatin
No registration fees are used to fund alcohol served at this function.

Wednesday, April 06 | 8:00PM - 11:00PM
Entertainment
Room: Gallatin
Entertainment is not subsidized by conference registration fees nor any U.S. federal government grants. Funding for this expense is provided by other revenue sources.

Wednesday, April 06 | 8:00PM - 11:00PM
Cash Bar
Room: Gallatin


Thursday, April 07 | 10:25AM - 10:25AM
Departure


*Session Chair.