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

Instructors:

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

Amy Loercher, Ph.D., Manager, Clinical Immunology, GlaxoSmithKline

This interactive session will enable attendees to work out a basic immunogenicity preclinical 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
• Preclinical 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

Amy Loercher, Ph.D., Manager, Clinical Immunology, GlaxoSmithKline

This interactive session of intermediate level will focus on the potential challenges of immunogenicity testing in preclinical 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: Immunogenicity Risk Assessment and Regulatory Strategy

Instructors:

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

Paul Chamberlain, NDA Advisory Board

The objective of this short-course is to illustrate the rationale for, and application of, the regulatory approach to identifying, evaluating and mitigating immunogenicity-related risks for different product types. The agenda is designed to encourage interaction between the presenters and the participants in sharing experience of application of a multi-disciplinary, integrated approach.

The following topics will be covered:

• Regulator’s perspective
• Overview of multi-disciplinary, integrated approach to identifying, evaluating and mitigating immunogenicity-related risks
• Examples that illustrate the “why” and “how to” for different product types / risk levels:
• Bacterial-derived proteins
• Enzyme replacement therapies
• Bone morphogenic proteins
• Allogeneic cell-based therapies
• Yeast-derived glycoproteins
• PEGylated uricase
• Novel antibody constructs

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

Instructor:

Liming Shi, MS, MA, Senior Research Scientist, Bioassay Development, Eli Lilly and 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