Speakers: Chongwoo Yu (FDA), Yuanxin Xu (Alnylam Pharma), Jiang Wu (Shire, ), Tatiana Plavina (Biogen), John-Michael Sauer (C-Path Institute), Joe Palandra (Pfizer), Lindsay King (Pfizer), Alvydas Mikulskis (Biogen)
Organizers: Jaya Goyal (Wave Life Sciences), Karen Hongmei Cao (Shire)
Date: 2017-12-08
Time: 8:30-16:45 Eastern Time
Registration fee: (USD): Regular: $175; For Academic / Unemployed: $125
Location: Marriott Kendall Square, 50 Broadway, Cambridge MA 02142
Major Sponsor: (2)Alturas Analytics, Inc.; CMIC, Inc.
Vendor show vendors registered to date: (6)Applied BioMath; Cambridge Biomedical; Medpace; MicroConstants; QPS LLC; R&D Systems
Registration: http://www.PBSS.org
Registration deadline:2017-12-08  (it will close sooner if the seating cap is reached)

About the Topic

Biotherapeutic drugs, constitute the most rapidly growing drug class of human therapeutics and encompass numerous therapeutic modalities with new scaffolds being added over time. A thorough understanding of the structure and biology of the biotherapeutics and disease pathophysiological mechanisms is essential for defining suitable bioanalytical and biomarker strategy. Pharmacokinetic-Pharmacodynamic characterization in disease animal models, toxicology species as well as the clinical studies in healthy and patient populations relies on the quality and robustness of bioanalytical and biomarker data. In addition, recent technological advancements have led to newer assay methodologies with increased throughput and sensitivity for enhanced bioanalytical characterization. Additionally, industry practices and regulatory guidance’s have evolved and have been updated during the past decade. This workshop will focus on current practices, perspectives, industry standards, challenges, novel solutions and regulatory guidance’s for bioanalysis of biotherapeutics. In addition, speakers will discuss tactics, challenges and opportunities of translating hypotheses and biomarker assays into clinically-actionable diagnostic and prognostic tests.

Agenda

8:00-8:45 Registration and Breakfast

8:45-09:00 Welcome, Introduction and Workshop Agenda (Jaya Goyal and Karen Cao)

Morning session Biotherapeutic Bioanalysis: Current practices, Industry standards, novel solutions and regulatory guidances

Regulatory Perspective:

9:00-9:45 Regulatory Perspective on Biomarker Bioanalysis During Drug Development - Chongwoo Yu, PhD

Methodologies/Technologies/Platforms:

9:45-10:30 Antibody-free LC-MS Platforms for Quantitation of Protein Therapeutics and Biomarkers - Jiang Wu

10:30-11:15 Utilizing immunoaffinity LC-MS/MS techniques for target measurements supporting biologics development - Joe Palandra

11:15-11:30 Coffee break and Vendor Show

11:30-12:15 Preclinical Cytometry Bio measures and Biomarkers in support of Biotherapeutics Modeling and Simulation - Lindsay King

12:15-01:15 Lunch Break and Vendor Presentation

Afternoon session New therapeutic modalities and Translational Biomarkers

1:15-2:00 Bioanalysis Support of RNAi Therapeutic Development - Yuanxin Xu

2:00-04:30 PM Biomarker strategies to support therapeutic development and decision making

Translational Biomarkers

2:00-2:45 Translational Safety Biomarkers for Use in Drug Development: The Predictive Safety Testing Consortium - John Michael Sauer

2:45-3:30 Development and Implementation of Biomarker Strategy in Clinical Development of Alzheimer’s Disease Therapies - Alvydas Mikulskis

3:30-3:45 Coffee Break and Vendor show

3:45-4:30 From research biomarkers to tools for clinical decision making in Multiple Sclerosis - Tatiana Plavina

4:30-4:45 Closing remarks

Dr. Chongwoo Yu received his B.S. in Chemistry and M.S. in Physical Organic Chemistry from Hanyang University (Korea) and earned his Ph.D. in Analytical Chemistry with the focus on Drug Metabolism and Mass Spectrometry from Prof. Richard van Breemen’s group in the College of Pharmacy at the University of Illinois at Chicago (Chicago, IL). Subsequently, Dr. Yu has worked in the Department of Pharmacokinetics, Dynamics, and Metabolism (PDM) at Pfizer (Ann Arbor, MI) and the Drug Metabolism and Pharmacokinetics (DMPK) Department at Schering-Plough (currently, Merck; Kenilworth, NJ) for several years. At both organizations, Dr. Yu has been heavily involved in carrying out various types of drug metabolism, pharmacokinetics, and drug-drug interaction studies using mass spectrometry. Currently, Dr. Yu is a Senior Clinical Pharmacology Reviewer in the Office of Clinical Pharmacology (OCP) at the U.S. Food and Drug Administration (FDA). Dr. Yu’s work is focused on the evaluation of bone, reproductive, and urologic drug products. Dr. Yu served as a member of FDA guidance working group for the Bioanalytical Method Validation Guidance and he currently serves as the chair of the FDA OCP Bioanalytical Research (BAR) Scientific Interest Group (SIG).

Jiang Wu is a director of global bioanalytical and biomarker development at Shire, responsible for developing, validating, and executing PK and biomarker assays in support of nonclinical, clinical, and in vitro diagnostics programs across company’s portfolios. Prior to Shire, he worked as an associate research fellow at Pfizer-Wyeth worldwide medicinal chemistry and biotherapeutics subdivisions and a biomarker group leader at Millennium Pharmaceuticals. He has authored 60 scientific papers/book chapters/patents in the fields of biotherapeutics characterization, chemical proteomics, as well as regulated bioanalysis.

Joe Palandra. A graduate of the University of Toronto in Biochemistry, Joe’s career in the bioanalytical industry spans over 20 years with more than 18 years in pharma. Joe’s career began in small molecule PK exposure using LC\MS which subsequently transitioned to small molecule biomarker analysis, including extensive work with endocannabinoids and leukotrienes in inflammatory diseases. Joe currently resides at Pfizer’s Andover MA campus where he has been performing targeting protein quantitation utilizing immunoanalytical tools combined with nano LC-MS/MS, with heavy emphasis on the analysis of cytokines and other low abundance proteins in serum and tissue matrices.

Lindsay King is currently an Associate Research Fellow in the BioMedicines Design department at Pfizer, in Andover, MA. He leads a Cytometry based team which brings conventional, imaging and mass cytometry together to support mechanistic modelling and simulation and early bio therapeutics portfolio progression. He has held roles as scientific leader in the GLP Biotherapeutics Bioanalytical Centre of Emphasis supporting the global Pfizer portfolio, as a leader of a non-regulated group responsible for LBA based bioanalysis of biotherapeutics, immunogenicity and biomarker/biomeasures and as a full time DMPK departmental project team representative. Dr. King is currently past chair of the Ligand Binding Assay Bioanalytical Focus Group and has been active in this group and AAPS for many years. Most recently he co-organized and co-chaired the joint FDA/AAPS Crystal City VI meeting on biomarker assay validation and the APPS workshop AAPS Workshop towards Global Harmonization of Bioanalytical Method Validation. He received his Ph.D. in Zoology from the University of Toronto and his Honours B.Sc. from the University of Western Ontario.

Yuanxin Xu joined Alnylam Pharmaceuticals (April 2015) as Sr. Director managing Bioanalytical Sciences group as part of DMPK eDEV. Her responsibilities include method development and validation for studies that are conducted at Alnylam and at CRO sites to support siRNA platform based drug development from pre-clinical to clinical stages. Multiple platforms (such as mass spec, HPLC, qPCR, and LBA) were used to evaluate PK/TK, biomarkers for PD, disease, and safety, and drug immunogenicity (ADA). Before joining Alnylam, she was with Genzyme for ~13 years including the last 3 years with Sanofi as Sr. Director at Bioanalytical Sciences. Her responsibilities included clinical assay development and validation to support drug development and NDA/BLA filing for protein therapeutics (such enzyme replacement therapies, monoclonal antibody drugs, cytokine, peptide drugs, antisense oligonucleotide, cell and gene therapies) and small molecule drugs (substrate reduction therapies and small molecule drugs for cancer). She also served as scientific liaison in Sanofi to provide scientific and technical support to CROs, especially for assay trouble shooting activities. Prior to Genzyme, she worked at BioTransplant/Immerge Biotherapeutics for organ and cell transplantation studies and induction of immune tolerance. Yuanxin received her Ph.D. from Iowa State University in Biochemistry as a China-US government CUSBEA scholar and Bachelor of Medicine from Beijing Medical University (now Peking University). She is an active member and co-lead for immunogenicity of the AAPS LBABFG steering committee and flow cytometry sub-group steering committee, participating in writing white papers, discussing technical challenges, and providing feedbacks for regulatory guidance documents.

Dr. John Michael Sauer is a toxicologist by training with over 20 years of experience in drug discovery and development. He has been responsible for leading multiple functional areas across several pharmaceutical companies. He is dedicated to bringing translational science approaches to safety assessment, as well as transforming the way we use nonclinical safety data to drive drug development. He received his Doctorate degree in Pharmacology and Toxicology from The University of Arizona. Currently, John Michael is the Program Officer of Biomarker Programs and the Executive Director of the Predictive Safety Testing Consortium at the Critical Path Institute, as well as an Adjunct Research Professor in the Department of Pharmacology at the University of Arizona, College of Medicine.

Alvydas Mikulskis is a Principal Investigator in the Biomarkers department at Biogen and has over 20 years of assay development experience. During his tenure at Biogen, Alvydas has managed bioanalytical and biomarker support for clinical development of biologics and small molecule drugs in Alzheimer’s disease (AD) and Multiple Sclerosis therapeutic areas. Alvydas has been leading the development and implementation of the fluid biomarker strategy for aducanumab, an anti-Aβ immunotherapeutic for Alzheimer’s disease currently in Ph III clinical development. Prior to joining Biogen, Alvydas worked at PerkinElmer, Inc. for 11 years on development of novel biomarker assay technologies for genomics and proteomics applications. Alvydas is technical expert in variety of established and cutting-edge immunoassay technology platforms with a track record of successfully developing and implementing bioanalytical and biomarker assays including diagnostic biomarker assays for patient stratification and enrichment in Phase III clinical trials. Alvydas is an active member of several industry committees and focus groups. He co-authored seven patents and over thirty scientific publications.

Tatiana Plavina is the Director, Translational Sciences, in the Value Based Medicine Group at Biogen Inc., Cambridge, Massachusetts. In her current role, she leads a cross-functional team focused on better understanding molecular underpinnings of disease heterogeneity, and developing biomarker tools to enhance treatment decision-making in multiple sclerosis. Tatiana has also been involved in establishing biomarker and analytical assays for many drugs in pre-clinical and clinical development at Biogen, including development of JCV antibody test to stratify PML risk. Tatiana received her M.S. degree in Physics and Chemical Biology from the Moscow Institute of Physics and Technology, and her Ph.D. degree in Chemistry and Chemical Biology from the Northeastern University. Tatiana has a number of publications in peer-reviewed journals, patents, and is a regular speaker at scientific conferences.