1:30–4:30 pm SC1: Basics of Immunogenicity Testing

Instructors:

Jim McNally, Ph.D., Associate Director,
PDM Immunogenicity Expert, EMD Serono

Jad Zoghbi, MSc, Senior Scientist,
Biomarkers and Clinical Bioanalyses, Sanofi

This interactive session will enable attendees to work out a basic immunogenicity pre-clinical and clinical testing strategy for various molecules including bi-functional and other novel scaffolds. Areas of difficulty will be discussed with specific case studies. Attendees are encouraged to contribute with their own experiences and to bring questions for discussion or submit to the meeting organizers in advance.

The following topics will be covered:

• Basic issues regarding screening, confirmatory and titer assays
• Assay methodologies and various technologies
• Current approaches to data analysis and cutpoints
• Pre-clinical and clinical considerations
• Common problems

5:30–8:30 pm Dinner SC2: Challenges of Immunogenicity Assessment

Instructors:

Jim McNally, Ph.D., Associate Director,
PDM Immunogenicity Expert, EMD Serono

Jad Zoghbi, MSc, Senior Scientist,
Biomarkers and Clinical Bioanalyses, Sanofi

This interactive session of intermediate level will focus on the potential challenges of immunogenicity testing in pre-clinical and clinical development and present case studies demonstrating how they can be handled. Attendees are encouraged to contribute with their own experiences and to bring questions for discussion or submit to the meeting organizers in advance.

The following topics will be covered:

• Challenges and approaches to resolve commonly encountered issues
• Multi-domain binding proteins
• Pre-existing ADAs
• Emerging trends in the development of neutralizing antibody assays
• Cross reactivity to endogenous proteins
• Clinical implications of ADAs

6:30–9:30 pm Dinner SC3: Advice on Putting Together an Integrated Summary of Immunogenicity - Detailed Agenda

Instructors:

João Pedras ­Vasconcelos, Ph.D., Biotech Quality and Immunogenicity Reviewer, Biotechnology Products, CDER­-FDA

Paul Chamberlain, NDA Advisory Board

Purpose

The purpose of this workshop is to share experience gained in preparing and reviewing the “Integrated Summary of Immunogenicity”, with case examples to illustrate 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.

Background

Information relevant for 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 can make 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, the recent draft revision to the main EU immunogenicity guideline has formally endorsed the concept of including a summary document in the MAA dossier, with the objective of collating the essential information required by the regulatory assessor.

Who should attend?

This workshop 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.

6:30–9:30 pm Dinner SC4: Strategic Bioassay Design and Analysis

Instructor:

Liming Shi, MS, MA, Senior Group Leader, Bioassay Development, Hospira, a Pfizer Company

This course will focus on the fundamentals of statistics and simple methodology that are routinely applied in bioassay laboratories. Covered topics will include review of statistical concepts and calculations, study design, assessing bioassay measurement quality and comparative studies.

The following topics will be covered:

• Uniqueness of bioassay, especially cell-based potency assay
• Considerations in bioassay development and validation
• Bioassay measurements and calculations
• Quality control of bioassay performance
• Comparative studies for bioassay development and transfer