COURSE #1
Protein Identification, Quantification, and Biomarker Analysis

Instructors: Weixun Wang, Merck; Gary Valaskovic, New Objective; Nalini Sadagopan, Agilent Technologies; Nathan Yates, University of Pittsburgh

COURSE #2
Bioanalysis of Mix Modalities via Hybrid and Orthogonal Approaches

Instructors: Suzanne Cordovado, CDC; Barry Jones, Q2 Solutions; Linlin Luo, Merck; Jim Shen, Bristol-Myers Squibb

COURSE #3
Microsampling and Patient Centric Sampling:
A Journey through What It Is and How You Can Incorporate It into Your Workflows

Instructors: Neil Spooner, Spooner Bioanalytical Solutions; Joe Siple, New Objective; Kevin Bateman, Merck; Tim Olah, Bristol-Myers Squibb; Enaksha Wickremsinhe, Eli Lilly & Co.; Melanie Anderson, Merck

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COURSE #4
Business of Science: Biomarker CRO Selection, Qualification and Oversight

Instructors: Olga Kavetskaia, Pfizer; Arkady Gusev, Novartis

Parallel Sessions

Track 1: Symposium
From Nomination to Implementation – How New Conditions are added to the National Newborn Screening Panel. The Story of Spinal Muscular Atrophy

Discussion Leaders: Suzanne Cordovado and Carla Cuthbert, Centers for Disease Control and Prevention

Charting the Path for Spinal Muscular Atrophy Newborn Screening
Carla Cuthbert, Centers for Disease Control and Prevention

Navigating the Challenges of State Implementation of Newborn Screening for Spinal Muscular Atrophy
Suzanne Cordovado, Centers for Disease Control and Prevention

Track 2: Colloquium
Powering Future Clinical Testing with Longitudinal and Predictive Markers

Discussion Leaders: Erwin Berthier, Tasso Inc.; James Rudge, Neoteryx

Longitudinal personalised clinical testing offers huge benefits in catching perturbations in biochemistry before abnormal pathology is observed allowing early intervention. In recent years, there has been a huge interest in developing highly specific tests in the omics / novel biomarkers fields to allow the dream of personalised testing to supplant standard clinical tests. This session showcases 4 leaders in the field of personalised testing and how their research and potential assay deployments will help to revolutionise future healthcare.

Delivering Mass Spectrometry-Based Multiplexed Protein Biomarkers for Improved Patient Outcomes
Steve Pennington, University College Dublin

Predictive Inflammation Biomarkers
Ben Herbert, Sangui Bio

Track 3: Colloquium
Updates on the Road to Regulation for Biomarkers

Discussion Leaders: Brad Ackermann, Eli Lilly & Co.; Robyn Rourick, Genentech

Biomarker Qualification as a regulatory process and utilization of all biomarkers in the drug development process. What is required to qualify a biomarker? How it can be used? What are current regulatory guidelines and standards for submission of biomarker data?

The Biomarker Qualification Process at the FDA
Chris Leptak, FDA

Biomarker Assay Validation for Every Conceivable Situation: One Size Fits All!
Steven Piccoli, GlaxoSmithKline

Parallel Sessions

Track 5: Symposium
Partnering with CROs for Positive and Effective Project Progression

Discussion Leader: Anahita Keyhani, Altasciences

Bioanalysis is integral to the drug development pathway. As bioanalytical outsourcing to CROs continues to increase in both volume and scope, bioanalytical support is evolving significantly beyond routine, small-molecule LC–MS. Biologics, biomarkers and new molecular modalities are a growing segment of drug-development pipelines, and bioanalytical CROs are keeping pace, transforming into centers of technological/scientific innovation and regulatory expertise. This session discusses strategies and best practices using case studies for partnering with CROs for positive and effective project progression.

Managing Bioanalytical Support Externally and Internally – Best Practices in Building Relationships through Communication
Lisa Borbridge, Allergan

A bioanalytical CRO is not just a service provider, but an extension of the internal team. As a Client, you need to build relationships, internally and externally, to ensure timelines are met. The best way to build those relationships is to communicate as much information as possible, as soon as possible. There is also the need to understand workflows, and taking the role of advocate wherever and whenever needed to keep programs moving forward.

How to Utilize the Expertise of your CRO
Joanna Gore, Appili Therapeutics

When working in a small organization, employees are frequently required to occupy multiple roles, some of which may be outside their direct area of expertise. In Clinical Research, many operational divisions assist in the planning, implementation and execution of a study. When a functional gap exists in-house, small organizations will often outsource work to a CRO to ensure successful completion of a study. Depending on the size of the organization, the services of multiple CROs may be engaged. This presentation discusses how to utilize the expertise of your CRO, and how to manage and integrate this knowledge when working with multiple CROs, to optimize the conduct and outcome of the clinical study.

Track 6: Colloquium
Biomarker Discovery and Quantitation

Discussion Leaders: Naidong Weng, Janssen Research & Development; Wenying Jian, Janssen Research & Development

Metabolic Profiling of the Cellular and Mitochondrial Dynamics by LC-MS
Yanping Lin, Janssen Research & Development

A Deconvoluted Proteomics for in Situ, Unambiguous Quantification of Cancer Markers in Patient-Derived Xenograft Models
Jun Qu, SUNY Buffalo

Anti-peptide Antibody Immunocapture-LC-MS/MS Strategy to Quantify PD-1 and PD-L1 as Clinical Biomarkers in FFPE Tissues for Immuno-Therapy Development
Naiyu Zheng, Bristol-Myers Squibb

Track 7: Colloquium
To Be Announced

Discussion Leaders: Sara Naseri, Qurasense; Søren Therkelsen, Qurasense

Social Hour & Sponsored Workshops

WORKSHOP 1
Patient Centric Sampling for Pediatric Studies–
Practical Aspects of Implementation

Sponsored by Spooner Bioanalytical Solutions

Workshop Leaders: Neil Spooner, Spooner Bioanalytical Solutions; Enaksha Wickremsinhe, Eli Lilly and Company;
Joe Siple, New Objective; Athena Zuppa, The Children's Hospital of Philadelphia

Blood sampling from neonates and children is often fraught with difficulties. The event can be stressful, painful and difficult to perform. This Workshop will concentrate on the practicalities of pediatric blood sampling, whether in the clinical setting, or in the home. The focus will be on the requirements of the patient/caregivers, carers and clinical staff. The process for blood sampling and its associated challenges will be outlined in a brief presentation by Dr. A.J. Allen (Eli Lilly and Company), who has in-depth experience of pediatric sampling, with a particular focus on pharmaceutical clinical trials. This will then be followed by an open discussion where the needs of the different parties will be explored, based upon the experience of those attending. This discussion will be used to formulate an outline for best practice for pediatric blood sampling with current technologies and will also give an insight into potential future approaches.

This Workshop will be of benefit for medical professionals, those involved in running pediatric clinical trials, bioanalysts, blood sampling and analysis technology developers and anyone who has an interest in collecting high quality blood samples from children whilst minimising the impact of this process on them.

Towards NOT Exsanguinating or Overly Traumatizing Children in Research
Albert J. Allen, Eli Lilly & Company

WORKSHOP 2
A Story of Innovation and Translation: Advances in Plasma Aß Biomarkers for Alzheimer’s Disease

Sponsored by Shimadzu Scientific Instruments

Accurate biomarker information is necessary to facilitate clinical trials of disease-modifying therapies for Alzheimer's disease, which are expected to be most efficacious at the earliest and mildest stages of the disease. Currently, only positron-emission tomography (PET) imaging and the measurement of amyloid-ß in cerebrospinal fluid are validated methods for identifying amyloid-ß deposition in the brain. Minimally invasive, cost-effective blood-based biomarkers are needed. Recently, Koichi Tanaka et al. (Nature. 2018 Feb 8;554(7691):249-254. doi: 10.1038/nature25456. Epub 2018 Jan 31) demonstrated the ability of amyloid-ß precursor protein (APP)669-711/amyloid-ß (Aß)1-42 and Aß1-40/Aß1-42 ratios to predict individual brain amyloid-ß-positive or -negative status. These biomarkers showed high performance when predicting brain amyloid-ß burden. The composite biomarker showed very high areas under the receiver operating characteristic curves (AUCs) in both data sets (discovery, 96.7%, n = 121 and validation, 94.1%, n = 111) with an accuracy approximately equal to 90% when using PIB-PET as a standard of truth. These test biomarkers were correlated with amyloid-ß-PET burden and levels of Aß1-42 in cerebrospinal fluid. These results demonstrate great potential utility of these research use only plasma biomarkers for predicting brain amyloid-ß burden at an individual level. This talk will review these innovative biomarkers as well as describe efforts to bring reliable and accurate testing services online in North America.

Sponsor Hospitality & Evening Workshops

Advancing Therapeutic Protein Characterization with New Orbitrap-based Mass Spectrometers

Sponsored by Thermo Fisher Scientific

Workshop Leader: Reiko Kiyonami, Thermo Fisher Scientific

Comprehensive characterization of therapeutic proteins remains challenging due to their complex and heterogeneous structure. Two new introduced Orbitrap-based mass spectrometers (Thermo Scientific™ Orbitrap Eclipse™ Tribrid™ Mass Spectrometer and Thermo Scientific™ Orbitrap Exploris™ 480 mass spectrometer) enable rapid structure characterization of therapeutic proteins through native and denatured MS intact analysis, subunit and top/middle-down analysis and peptide mapping. In this talk, we will provide a general introduction about these two new mass spectrometers and how their unique features, such as Proton Transfer Charge Deduction (PTCR) and High Mass Range MSn (HMRn) can help to facilitate fusion protein characterization and protein-drug characterization. Several analysis examples to demonstrate the improved sensitivity and robustness of the new mass spectrometers for accurate and reproducible routine native LC-MS screening of large sample set will also be introduced.

Parallel Sessions

Track 1: Symposium
Enabling Mass Spectrometry-based Assays via Innovations in Sample Processing

Discussion Leader: Brian Musselman, IonSense, Inc.

Bead-Assisted Mass Spectrometry (BAMS™): Higher Order Immunoassay Multiplexing for Targeted Quantification and Biomarker Discovery
Vladislav Bergo, Jeffrey Silva, Sergey Mamaev, Camilla Worsfold, and W. Adam Hill of Adeptrix

Molecular Imaging in Preclinical Drug Discovery
B. Healey, Revolution Biosciences

Submicroliter Sampling for Ambient MS - Reducing Sample size for Improved Analyte Detection
B. Musselman, F. Li, and P. Liang of IonSense, Inc.

TRACK 2: Colloquium
Patient Centric Sampling Interest Group–
Achievements and Next Steps

Discussion Leaders: Erwin Berthier, Tasso; Søren Therkelsen, Qurasense; Joe Siple, New Objective, Neil Spooner, Spooner Bioanalytical Solutions

The Patient Centric Sampling Interest Group (PCSIG) is a not-for-profit, free membership, community led organisation which has the aim of enabling cross organisational cooperation around the development and implementation of novel technologies and approaches for the collection of high quality blood samples from human subjects for the quantitative determination of drug, metabolite and biomarker concentrations. The group was founded at a CPSA USA meeting in 2017 and is composed of >90 members from over 50 organisations, with backgrounds including technology companies, instrument and consumable vendors, pharmaceutical companies, contract research organisations, publishers, central laboratories, academics and independent consultants.

This session will give attendees an introduction to the PCSIG, an update on current activities and will discuss potential future collaborative efforts for the group, including an independent clinical trial into the use of these novel technologies.

Track 3: Colloquium
Self-Innovation Strategies

Discussion Leaders: Lucinda Hittle, Merck; Liz Pierson, Merck

Social Hour & Sponsored Workshops

WORKSHOP 1
The Future of LC-MS: High Performance Redefined

Sponsored by New Objective

High Performance: Simple, Easy, Scalable
Emily Ehrenfeld, New Objective

High Performance: Integrated Solutions
Neil Spooner, Spooner Bioanalytical Solutions

High Performance: Education and Specialized Training
Jeremiah Tipton, BioTech & OMICSx Solutions, LLC

High Performance Redefined
Dan Spellman, Merck

WORKSHOP 2
Mitra^® VAMS: From Sample Collection to Bioanalysis, What are the Remaining Challenges?

Sponsored by Altasciences

Workshop Leaders: Jeff Plomley and Anahita Keyhani, Altasciences

The increased adoption of Mitra^® VAMS technology for supporting low-volume blood collection in clinical studies may be attributed to the elimination of the HCT effect biasing sampling volume, coupled with the ease of device implementation. However, additional downstream bioanalytical challenges remain, such as age-related extractability and recovery bias linked to blood HCT. Solutions to these bioanalytical challenges shall be addressed by Jeff Plomley in his presentation detailing the application of Impact-Assisted Extraction to Mitra® microsamples as a novel, and potentially universal, sample preparation approach. In addition, Altasciences will share their recent results evaluating the Tasso On-Demand "anywhere, anytime" pain-free blood sampling device, supporting the industry drive towards patient centricity.

Following the presentation, an open discussion shall follow where participants can share their experiences and challenges incorporating Mitra® technology into their microsampling programs, from sample collection to bioanalysis. By leveraging the combined expertise of all workshop members, the remaining "pain-points" associated with Mitra® technology can be identified and potential solutions derived to facilitate continued implementation of the technique.

The Workshop is expected to benefit key stakeholders involved with Mitra^® VAMS technology, including technology developers.

WORKSHOP 3
Mass Spectrometry Trends in Clinical Research

Sponsored by Thermo Fisher Scientific

Zhaoxiang (Sean) Wu, Thermo Fisher Scientific

Over the last decade, liquid chromatography coupled to mass spectrometry (LC-MS) has gained widespread popularity and transitioned from research applications to more routine clinical environment. While triple quadrupole mass spectrometers combined with liquid chromatography (LC-MS/MS) are used extensively for targeted, selective and sensitive quantitation. However, there is a growing demand for one single MS platform that can efficiently perform a multitude of assays – from routine quantitation of analytes to screening, confirmation, and characterization of unknown analytes (ranging from small to large sizes). As a result we observe increasing demands for ready-to-use complete kits for small molecules running on high resolution instruments.

LC-MS based assays for clinical research and forensic toxicology demand high degree of efficiency – from identification and confirmation of analytes with high resolution accurate mass spectrometry to sensitive, robust, reliable quantitation with triple quadrupole mass spectrometry. In this presentation, we shall offer a comprehensive overview of different LC-MS platforms and applications that can be leveraged for clinical research applications regardless of matrix complexity, type of molecules, and user expertise.

Parallel Sessions

TRACK 5: Colloquium
Biomarkers and Business of Science:
Fueling the Disruptive Innovation

Discussion Leaders: Arkady Gusev, Novartis; Olga Kavetskaia, Pfizer

Biomarkers and Business of Science: Fuelling the Disruptive Innovation” This session continues to build on 2018’s CPSA session theme on “The Business of Science”. This year we focus on leveraging CRO experiences for biomarker analysis in clinical studies including using cutting edge technologies, operationalizing of complex clinical biomarker strategies and CRO-Pharma partnership models.

Efficiently Quantifying Peripheral Blood-Based Proteins for Clinical Trials: Recent Case Studies of Pharmacodynamics, Prognostic, and Predictive Biomarkers of Drug Response
Dominic Eisinger, Myriad RBM

The Nuts and Bolts of Making Complex Biomarker Strategies Work in Clinical Trials
Chad J Briscoe, PRA Health Sciences

Can a CRO Select its Pharma Clients?
Roger Hayes, ICON

Track 6: Colloquium
Women in Science and Technology

Discussion Leaders: Kayte Parlevliet, Trajan Scientific & Medical and Robyn Rourick, Genentech; Suzanne Cordovado, Centers for Disease Control and Prevention; Amanda Berg, New Objective; Alla Kloss, Sanofi; Lucinda Hittle, Merck; Emily Ehrenfeld, New Objective

This facilitator led workshop will focus on discussing the role of women in science and technology and how we all as individuals can contribute to promoting and progressing this important cause in our own careers and lives.

Track 7: Colloquium
Frontiers of Innovation, Technology and Solutions

Discussion Leaders: Alla Kloss, Sanofi and Ganesh Moorthy, Children's Hospital of Philadelphia

Challenges to Personalized Antimicrobial Dosing in Pediatrics: How Microsampling Can Help Us Move from Therapeutic Drug Monitoring to Management
Kevin J. Downes, The Children’s Hospital of Philadelphia

Real Time Therapeutic Drug Monitoring: Is This a Reality or Just a Dream?
Ganesh S. Moorthy, The Children's Hospital of Philadelphia

The Surprising Value of Real World Data
Arthur H. Combs, MC 10, Inc.

Parallel Sessions

TRACK 1: Symposium
The DAVINCI Project:
Development of Next Generation Analytical Technologies

Discussion Leader: Eugene Ciccimaro, Agilent Technologies

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This workshop style session guided by an expert panel will walk through the stages of vendor-industry collaborations. Our goal is to begin to answer the question: how can our community work together better to more quickly and successfully nurture collaborations leading to new analytical solutions to address today's challenges?

• Vendors should consider publishing in per-reviewed journals rather than application notes. Bioanalysis will support an all vendor author list.
• Collaborations should begin with clear and transparent criteria of success from both sides.
• Three-way CDA/NDAs are difficult, but the norm for CRO-Client-Industry relations. Is there a simplifier path?
• MS data sharing is practically difficult. Cloud resources often blocked by industry. Perhaps designating data sharing mechanism early is critical.
• Communication is key. Damage is done in the dark. Share news of progress and failed attempts.

Panelists and Presenters:
Carl Myerholtz, Agilent Technologies
Harsha Gunawardena, Janssen Research & Development
Jay Johnson, Pfizer
Jonathan Josephs, Sanofi
Jim Murphy, Waters Corporation
Tim Olah, Bristol-Myers Squibb
Scott Peterman, Thermo Fisher Scientific
Kayte Parlevliet, Trajan Scientific and Medical
Dan Spellman, Merck
Patty Sun, SCIEX

Parallel Sessions

TRACK 4: Symposium
Presentations from the Edge: Young Scientist Session

Discussion Leaders: Naidong Weng, Janssen R&D; Chuping Luo, Advanced Materials Technology

High Resolution Mass Spectrometry and Isotope Tracing to Probe Lipid Metabolism
Erik Allman, Penn State University

Sensitive Antibody Free Strategies for Targeted Protein Quantification in Biomatrices
Bo An, GlaxoSmithKline

Development of A Streamlined Workflow for Quantitative Analysis of Antisense Oligonucleotide (ASO) Therapeutics
Fengjian Shi, Biogen

CLOSING REMARKS & ADJOURN

Timothy Olah, Bristol-Myers Squibb
Neil Spooner, Spooner Bioanalytical Solutions
Mike Lee, Milestone Development Services