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2023年10月2日（月）  9:00 - 12:00 pm

SC1: Development of Neutralizing Antibody Assays, Technical Considerations and Case Studies
SC1：中和抗体アッセイの開発、技術的考察とケーススタディ

The development of neutralizing antibody assays is a daunting task that is complicated by the specific nature of each biotherapeutic. Many factors must be assessed to choose the proper assay format, to develop a robust assay, and choose when to invest in the development and implementation of these assays. This short course will focus on these topics and provide examples of current industry practices and publications.
9:00 am

Development of Neutralizing Antibody Assays, Technical Considerations, and Case Studies

Lynn Kamen, PhD, Scientific Officer, Executive Director, BioAgilytix

Jim McNally, PhD, CSO, BioAgilytix

Topics to be Covered Include:

  • Current regulatory guidance
  • NAb assay strategy - Immunogenicity risk assessment
  • Special focus on assay format selection - Mechanism of action-based approach
  • Validation and implementation of NAb assays
  • Relevant case studies

INSTRUCTOR BIOGRAPHIES:

Lynn Kamen, PhD, Scientific Officer, Executive Director, BioAgilytix

Lynn Kamen is a Scientific Officer at BioAgilytix. She earned her PhD in Immunology at the University of Michigan and completed a postdoctoral fellowship in immunology at the University of California, San Francisco. Lynn has over a decade of experience working in drug development, from early target discovery through clinical development for both large and small molecules at several companies including Portola Pharmaceuticals, and Alector. More recently, Lynn was a principal scientist at Genentech where she supported the in vitro biological characterization of large molecules and lead the development of immunogenicity assays including ADA, NAb and immunogenicity risk ranking assays. She is co-lead of the AAPS NAber working group and member of the AAPS NAb drug tolerance sub-team.

Jim McNally, PhD, CSO, BioAgilytix

Dr. McNally has an extensive background in bioanalytical assay development and program leadership spanning nearly 20 years working in the pharmaceutical and biotechnology industry. Prior to joining BioAgilytix, Dr. McNally was Executive Director at CRISPR Therapeutics, where he lead a team of scientists to develop a portfolio of assays to support development of gene-based therapeutic candidates throughout their lifecycle. He has also previously held roles at Genzyme, Pfizer, EMD Serono, and Shire which have given him broad experience in the development of large molecule, gene therapy, and cell therapy biotherapeutics. Dr. McNally is a recognized thought leader in the development and application of bioanalytical methods used in regulatory submissions and is specifically skilled in progression of biotherapeutics from research through clinical development. He has a special interest in the immunogenicity of biotherapeutics and leads an industry-wide working group to address this issue. A key part of his role at BioAgilytix is advising on emerging scientific developments and providing scientific and regulatory guidance. Dr. McNally obtained his B.S. in Biology from Mississippi State University, his Ph.D. Viral Immunology from Louisiana State University School of Medicine in Shreveport, and his Post-Doc in Viral Immunology from University of Massachusetts Medical School.

2023年10月2日（月）  2:00 - 5:00 pm

SC2: Overcoming Drug and Target Interference in ADA and NAb Assays
SC2：ADAおよび中和抗体（NAb）アッセイにおける薬剤と標的干渉の克服

Soluble drug, drug target, and matrix can often interfere in the detection of anti-drug antibodies, including neutralizing Abs. Although not always straightforward, it can be addressed and mitigated in a properly designed immunoassay. This short course will give an overview of the different types of interferences, and current methodologies and approaches being utilized to resolve or reduce them.
2:00 pm

Overcoming Drug and Target Interference in ADA and NAb Assays

Lynn Kamen, PhD, Scientific Officer, Executive Director, BioAgilytix

Weifeng Xu, PhD, Director, Bioanalytical, Merck

Topics to be Covered Include:

  • Types of Interferences:         
    > Soluble Target         
    > Drug         
    > Matrix
  • Immunogenicity Assay Designs and Susceptibility to Interference
  • Competitive Ligand-Binding Immunoassays
  • Bridging Immunoassays
  • Cell-Based Immunoassays
  • Mitigation Strategies
  • Sample Pre-Treatment BEADACEBEHD
  • Heat Inactivation
  • Case Studies

INSTRUCTOR BIOGRAPHIES:

Lynn Kamen, PhD, Scientific Officer, Executive Director, BioAgilytix

Lynn Kamen is a Scientific Officer at BioAgilytix. She earned her PhD in Immunology at the University of Michigan and completed a postdoctoral fellowship in immunology at the University of California, San Francisco. Lynn has over a decade of experience working in drug development, from early target discovery through clinical development for both large and small molecules at several companies including Portola Pharmaceuticals, and Alector. More recently, Lynn was a principal scientist at Genentech where she supported the in vitro biological characterization of large molecules and lead the development of immunogenicity assays including ADA, NAb and immunogenicity risk ranking assays. She is co-lead of the AAPS NAber working group and member of the AAPS NAb drug tolerance sub-team.

Weifeng Xu, PhD, Director, Bioanalytical, Merck

Weifeng has been in the field of immunogenicity for biologics for about 10 years. He had developed cell-based neutralization Ab assays for multiple key product at BMS including Opdivo and Yervoy. He is an active member in AAPS NAb work group as well as EBA NAb team; he is also co-leading the NAb assay drug tolerance subteam at AAPS. After join Merck at the end of 2018, Weifeng is now leading Cell Assay group within PPDM Regulated Immunogenicity to develop neutralizing assays for both biologics and vaccines.

2023年10月2日（月）  5:30 - 8:30 pm

SC3: Validation of ADA Assays and Cut Point Calculations - IN-PERSON ONLY
SC3：ADAアッセイとカットポイント測定の検証

This short course will focus on the validation of ADA assays and cut-point evaluations. We will provide an in-depth overview of the basic considerations around ADA assay validation, with significant focus on the process of evaluating different types of cut-points, and the translation of the cut-point established during validation to the real-world implementation during a preclinical or clinical study.
5:30 pm

Validation of ADA Assays and Cut Point Calculations

Kavitha Akula, PhD, Principal Scientist, Bristol Myers Squibb Co.

Ang Liu, Director, Clinical Bioanalysis, Daiichi Sankyo

Topics to Be Covered Include: 

  • Tiered testing strategy: Basic issues regarding screening, confirmatory, and titer assays
  • ADA assay validation strategies: Experimental design to execute a validation
  • Step-wise process for calculating different types of cut-points
  • Practical challenges for the in-study implementation of cut-points
  • Case studies related to the implementation of validation and study-specific cut-points

INSTRUCTOR BIOGRAPHIES:

Kavitha Akula, PhD, Principal Scientist, Bristol Myers Squibb Co.

Dr. Kavitha Akula is currently a Principal Scientist at Bristol-Myers Squibb (BMS) in the Non-clinical Disposition and Bioanalysis Group. Kavitha joined BMS in 2019 as Research Investigator-II with about five years of experience in regulated bioanalysis from different contract research organizations (CRO). She received her PhD from Temple University, Philadelphia in Organic Chemistry in 2017. Kavitha has extensive experience in regulate bioanalysis of large molecule drugs and new treatment modalities in support of PK and Immunogenicity. She recently took co-lead position in the Early Career Bioanalytical Scientists (ECBS) sub-team in AAPS.

Ang Liu, Director, Clinical Bioanalysis, Daiichi Sankyo

Dr. Ang Liu is currently a Director of Clinical Bioanalysis in the department of Quantitative Clinical Pharmacology at Daiichi Sankyo. Prior to joining Daiichi Sankyo in July, 2021, Ang was a Senior Principal Scientist in the Department of Bioanalytical Sciences at Bristol-Myers Squibb. Ang received her Ph.D. from University of Illinois at Chicago in 2009 and started her industry career since then. Ang has extensive experience in regulated bioanalysis of large-molecule and small-molecule drugs, and new treatment modalities in support of PK and immunogenicity using LC-MS based and LBA based technologies. Ang has had increasing responsibilities throughout her career at Tandem Labs (now LabCorp), Bristol-Myers Squibb, and Daiichi Sankyo. Ang has authored or co-authored a book chapter and over 15 scientific publications in peer-reviewed journals. She also serves as a reviewer of a number of journals. Ang has served on the organizing committee of Applied Pharmaceutical Analysis (APA) Conference since 2016 and she is the Presiding Chair of APA 2022.

SC4: Recent Advances with Cell and Gene Therapy - IN-PERSON ONLY
SC4：細胞・遺伝子治療での最近の進展 - 対面式のみ

Topics to be covered in this course include: Immunogenicity assessment of cell therapies, examining recent developments with CAR T cells & edited stem cell, immunogenicity assessment of gene therapies, recent data on pre-existing reactivity for AAV, advances with redosing, application of current guidance to novel modalities, and what is your product, the vector, the expressed product?
5:30 pm

Recent Advances with Cell and Gene Therapy

Jim McNally, PhD, CSO, BioAgilytix

Topics to be Covered Include: 

  • Immunogenicity assessment of cell therapies 
  • Examining recent developments with CAR T cells and edited stem cells
  • Immunogenicity assessment of gene therapies
  • Recent data on pre-existing reactivity for AAV
  • Advances with redosing 
  • Application of current guidance to novel modalities
  • What is your product, the vector, the expressed product?

INSTRUCTOR BIOGRAPHIES:

Jim McNally, PhD, CSO, BioAgilytix

Dr. McNally has an extensive background in bioanalytical assay development and program leadership spanning nearly 20 years working in the pharmaceutical and biotechnology industry. Prior to joining BioAgilytix, Dr. McNally was Executive Director at CRISPR Therapeutics, where he lead a team of scientists to develop a portfolio of assays to support development of gene-based therapeutic candidates throughout their lifecycle. He has also previously held roles at Genzyme, Pfizer, EMD Serono, and Shire which have given him broad experience in the development of large molecule, gene therapy, and cell therapy biotherapeutics. Dr. McNally is a recognized thought leader in the development and application of bioanalytical methods used in regulatory submissions and is specifically skilled in progression of biotherapeutics from research through clinical development. He has a special interest in the immunogenicity of biotherapeutics and leads an industry-wide working group to address this issue. A key part of his role at BioAgilytix is advising on emerging scientific developments and providing scientific and regulatory guidance. Dr. McNally obtained his B.S. in Biology from Mississippi State University, his Ph.D. Viral Immunology from Louisiana State University School of Medicine in Shreveport, and his Post-Doc in Viral Immunology from University of Massachusetts Medical School.

2023年10月4日（水）  5:30 - 8:30 pm

SC5: Advice on Putting Together an Integrated Summary of Immunogenicity - IN-PERSON ONLY
SC5：免疫原性の統合サマリーをまとめる際の提言- 対面式のみ

The purpose of this workshop is to share experience gained in preparing and reviewing the “Integrated Summary of Immunogenicity (ISI)” for submission in regulatory filings. We will overview examples of the multi-disciplinary information that is most useful for the regulator assessing the scale of risk of undesirable immunogenicity for overall clinical benefit vs. risk. We will also examine the sponsor team's role, the general format of an ISI, and provide examples of how to anticipate and address potential issues (and how to avoid introducing any new ones!) by generating a well-thought-out and constructed integrated summary.
5:30 pm

Advice on Putting Together an Integrated Summary of Immunogenicity

Bonnie Rup, PhD, Biotechnology Consultant, Bonnie Rup Consulting

The purpose of this workshop is to share experience gained in preparing and reviewing the “Integrated Summary of Immunogenicity (ISI)” for submission in regulatory filings. We will overview examples of the multi-disciplinary information that is most useful for the regulator assessing the scale of risk of undesirable immunogenicity for overall clinical benefit vs. risk. We will also examine the sponsor team's role, the general format of an ISI, and provide examples of how to anticipate and address potential issues (and how to avoid introducing any new ones!) by generating a well-thought-out and constructed integrated summary.

Background

Information relevant to the assessment of the impact of undesirable immunogenicity of therapeutic proteins on overall clinical benefit vs. risk balance is distributed across many different sections of the regulatory dossier. This has made it difficult for regulatory reviewers to locate the requisite data. Moreover, essential background information to describe the intrinsic immunogenic potential of the molecule, and how extrinsic factors (product quality, patient variables, dose regimen, etc.) might interact to influence clinical manifestations, is often missing. Although there might be valid reasons for applying a particular strategy for evaluating immunogenicity, the sponsor’s rationale is often not clearly explained. For this reason, recently issued FDA and EMA guidance has recommended submitting integrated summary documents in regulatory submissions with the objective of collating the essential information required by the regulatory assessor.

Who Should Attend?

This short course is relevant to anyone who is involved in generating and compiling the input data for immunogenicity--related sections of regulatory dossiers, including CMC, bioanalytical, non--clinical, clinical, and regulatory specialists.

Topics to be Covered Include:

Part 1 - Defining the Gap:

  • Priorities for the regulator
  • Common gaps in dossiers 
  • Examples of agency questions triggered by missing information 
  • The regulator’s recommendations

Part 2 - Addressing the Gap:

  • Suggested structure of the Integrated Summary of Immunogenicity 
  • Relationship to other parts of the dossier What, where, and how? 
  • Examples to illustrate how to present relevant information, including: Intrinsic immunogenicity and systems biology 
  • Linkage to product quality control strategy 
  • Rationale for extent of evaluation 
  • Correlation of bioanalytical and clinical signals

The Role of the Sponsor Team:

  • Role of the Sponsor cross-functional project team from early Immunogenicity Risk Assessment through Integrated Summary of Immunogenicity 
  • Identifying relevant information for your project's Integrated Summary 
  • Determining the Integrated Summary structure, level of detail and analysis, and links to other parts of the dossier 
  • Using a well-constructed Integrated Summary to reduce potential concerns

Part 3 - Interactive Discussion: Using the Integrated Summary of Immunogenicity to Minimize Regulatory Questions at the Marketing Authorization Stage

  • Discussion topics to be driven by participant’s questions and case examples

INSTRUCTOR BIOGRAPHIES:

Bonnie Rup, PhD, Biotechnology Consultant, Bonnie Rup Consulting

Bonita Rup is a biopharmaceutical development consultant, providing expert advice on bioanalysis, immunogenicity risk assessment, and related regulatory strategy aspects of biopharmaceutical development. Previously she was Research Fellow and lead for the Immunogenicity Discipline at Pfizer, Assistant Vice President of Protein Bioanalytics in Wyeth, and held various positions directing development and application of immuno-ligand binding assay technologies for PK, immunogenicity and protein impurity analysis, and other aspects of biopharmaceutical development. During her career, she has been involved in multiple regulatory filings during preclinical, clinical development and marketing approval of biopharmaceutical products. She has been a member of AAPS, EIP, European IMI ABIRISK consortium, and Biosafe; with these organizations, she has been a co-author for multiple publications related to monitoring immunogenicity and bioanalysis of therapeutic proteins. Bonnie received her B.S. from University of Massachusetts, Amherst, Ph.D. from University of Texas, Austin, and conducted postdoctoral research at Duke University and University of Rochester, NY.

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