NORTH AMERICA 2020

CMC Strategies and Phase-appropriate GMP Approaches for Expedited Program Development

October 13-15, 2020 Virtual Program Agenda

Organized by

Welcome to the CMC Strategy Forum CMC Strategies and Phase-appropriate GMP Approaches for Expedited Program Development

We are pleased to welcome you to the CMC Strategy Forum. The purpose of the CMC Strategy Forum is to provide a venue for biotechnology/biological product discussion. The meetings focus on relevant CMC issues throughout the lifecycle of a product and thereby foster collaborative technical and regulatory interactions. The Forum strives to share information with the regulatory agencies to assist them in merging good scientific and regulatory practices. Outcomes of the Forum meetings are published in an appropriate peer-reviewed journal.

Each meeting will focus on a CMC related issue such as product characterization, comparability, specifications, etc. The format of each meeting will consist of case studies and presentations by industry and/or regulatory experts to introduce the topic and the key issues of concern. Workshop sessions, which consist of panel discussions and Q&A, will then be conducted to allow for additional discussion on the technical and regulatory details of the topics. It is envisioned that the final outcome of the workshop discussions will be the development of a document to be submitted to the appropriate Regulatory Agency designees for their consideration in developing and/or clarifying good regulatory practice guidelines for biotechnology derived products.

The success of the CMC Strategy Forum will depend on your active participation in discussing and raising issues pertaining to development of biologics. We encourage you to participate wholeheartedly in the workshops that have been designed to stimulate exchange of ideas and information.

We would like to thank the speakers who are giving generously of their time and resources, and to you, for your attendance. We acknowledge the generosity of our program partners: AbbVie Bioresearch Center, Inc.; Amgen Inc., AstraZeneca, Biogen, Eli Lilly and Company, F. Hoffmann-La Roche Ltd., Genentech, a Member of the Roche Group, Merck & Co., Inc., Novo Nordisk A/S and Pfizer, Inc. We are grateful for the expert management from CASSS. Their experience and guidance in the preparation of this Forum has been invaluable.

ACKNOWLEDGEMENTS

CMC STRATEGY FORUM NORTH AMERICA PROGRAM COMMITTEE Kristopher Barnthouse, Janssen Pharmaceuticals R&D LLC Lucy Chang, Merck & Co., Inc. Barry Cherney, Amgen Inc. Natalie Ciaccio, Horizon Therapeutics plc Fiona Cornel, Health Canada JR Dobbins, Eli Lilly and Company Jennifer Eck, AstraZeneca Taro Fujimori, AbbVie Bioresearch Center, Inc. Nomalie Jaya, Seattle Genetics, Inc. Carmilia Jiménez Ramirez, BioMarin Pharmaceutical Inc. Michael Kennedy, CBER, FDA Sarah Kennett, Genentech, a Member of the Roche Group Shawn Novick, BioPhia Consulting, Inc. Mary Beth Pelletier, Biogen Timothy Schofield, CMC Sciences, LLC Jennifer Sexton, Genentech, a Member of the Roche Group Zahra Shahrokh, ZDev Consulting Jason Starkey, Pfizer, Inc. Cecilia Tami, Genentech, a Member of the Roche Group Kimberly Vazquez, Immunomedics, Inc. Marc Verhagen, Sanofi Joel Welch, CDER, FDA Bernice Yeung, Biogen

CMC STRATEGY FORUM GLOBAL STEERING COMMITTEE Daniela Cerqueria, ANVISA-Brazilian National Anthony Ridgway, Health Canada, Canada Health Surveillance Agency, Brazil Ilona Reischl, BASG-Federal Office for Safety in Yasuhiro Kishioka, PMDA-Pharmaceutical and Health Care, Austria Medical Devices Agency, Japan Nadine Ritter, Global Biotech Experts, LLC, Steven Kozlowski, CDER, FDA, USA USA Junichi Koga, Daiichi Sankyo Co., Ltd., Japan Chikako Torigoe, CBER, FDA USA Rohin Mhatre, Biogen, USA Wassim Nashabeh, F. Hoffmann-La Roche Ltd., USA (Chair)

CASSS STAFF Karen A. Bertani, CMP, Director, Global Engagement and Knowledge Sharing, (CMC Forum Manager) Rose Bueno, Administrative and Accounting Specialist Alissa Doyle, Program Planning Coordinator Stephanie L. Flores, CAE, Executive Director Julie Harris, CMP, Program Manager Isolde Honoré, Exhibitor Relations and Technology Coordinator Renee Olson, Senior Program and Marketing Manager Anne Ornelas, DES, Senior Operations Manager Carolyn Slade, Program Manager Catherine Stewart, CPA, Director of Finance

AUDIO VISUAL Michael Johnstone, MJ Audio Visual Productions The Scientific Organizing Committee gratefully acknowledges the pharmaceutical and biotechnology industry for their generous support of the CMC Strategy Forum North America series:

STRATEGIC DIAMOND PROGRAM PARTNERS F. Hoffmann-La Roche Ltd. Genentech, a Member of the Roche Group STRATEGIC PLATINUM PROGRAM PARTNERS Amgen Inc. AstraZeneca Biogen Merck & Co., Inc. STRATEGIC GOLD PROGRAM PARTNERS AbbVie Bioresearch Center, Inc. Eli Lilly and Company Novo Nordisk A/S Pfizer, Inc. The Scientific Organizing Committee gratefully acknowledges the following media for their promotional consideration of the CMC Strategy Forum North America series:

LEADING MEDIA PARTNERS BioProcess International International Pharmaceutical Quality

Forum Abstract

CMC Strategies and Phase Appropriate GMP Approaches for Expedited Program Development

FORUM CO-CHAIRS: Nina Cauchon, Amgen Inc. Carmilia Jiménez-Ramirez, BioMarin Pharmaceutical Inc. Michael Kennedy, CBER, FDA Ramesh Potla, CDER, FDA

SCIENTIFIC ORGANIZING COMMITTEE: Natalya Ananyeva, CBER, FDA Lucy Chang, Merck & Co., Inc. Bharat Dixit, ClearB Therapeutics Narasaiah Dontha, Celgene Corporation, A Bristol-Myers Squibb Company Michelle Lytle, Eli Lilly and Company Polly Mak, Genentech, a Member of the Roche Group David Robbins, AstraZeneca Karen Rowlandson, Health Canada Paula Russell, Health Canada Jason Starkey, Pfizer, Inc.

In the first two decades of the 21st century, significant advances have been made in the science of drug development resulting in an array of new modalities entering the clinic. Regulators and sponsors alike are subject to increasing pressures to expedite the availability of these drugs globally for the treatment of unmet medical needs. Beginning with the United States and the European Union, health authorities from many countries have now implemented accelerated regulatory pathways that are based on the potential for significant patient benefit. The clinical successes of these drugs have resulted in significantly reduced development timelines, thus generation of CMC information for marketing authorizations is on the critical path to approval. Additionally, companies face significant challenges when managing post-approval changes globally. Solutions are often worked out on a case-by-case basis between each sponsor and regulatory agency since each program and modality present unique challenges. However, common topics and questions do exist from a CMC perspective. In concert with CMC challenges presented when managing expedited programs, the interpretation of phase appropriate GMP guidance from health authorities remains a subject open to interpretation from industry experts. Interaction between regulatory agencies and industry representatives can assist with the development of harmonized risk-based approaches and develop tools to address these challenges, thus facilitating a common understanding of CMC challenges, and implementing strategies to ensure phase-appropriate GMP compliance.

This three-day CMC Forum will address pitfalls, risk mitigation levers, and risk-based approaches for common CMC issues, and discuss regulatory tools currently available to accelerate development in various countries. The first and second day of this forum will focus on CMC strategies for expedited program development, while the third day will focus on phase appropriate GMPs and practical considerations for expedited programs. Specific topics and questions will be discussed via the use of case studies.

CMC Strategy Forum Program Summary

CMC Strategies and Phase Appropriate GMP Approaches for Expedited Program Development

Tuesday, October 13, 2020

NOTE: All times are Eastern Daylight Time (EDT)

10:45 – 11:00 Sign-on to Virtual Platform

11:00 – 11:15 CASSS Welcome and Introductory Comments Nadine Ritter, Global Biotech Experts, LLC

11:15 – 11:30 CMC Strategy Forum Welcome and Introductory Comments Nina Cauchon, Amgen Inc. Ramesh Potla, CDER, FDA

Workshop Session One:

CMC Strategies to Expedited Program Development: Regulatory Perspectives Session Chairs: Natalya Ananyeva, CBER, FDA and David Robbins, AstraZeneca

11:30 – 11:35 Introduction

11:35 – 12:00 Expedited Development Programs: CMC Challenges and Lessons Learned Susan Kirshner, CDER, FDA, Silver Spring, MD USA

12:00 – 12:25 Regulatory Considerations for Expedited Vaccine Development and Review in Response to Novel Emerging Disease Robin Levis, CBER, FDA, Silver Spring, MD USA

12:25 – 12:50 Pandemic Preparedness: Regulatory Agility in the Era of COVID-19 Jason Fernandes, Health Canada, Ottawa, ON Canada

12:50 – 13:15 Regulatory Approaches for Accelerated Development in Europe Mats Welin, Medical Products Agency, Uppsala, Sweden

13:15 – 13:25 Break

13:25 – 14:30 PANEL DISCUSSION – Questions and Answers Jason Fernandes, Health Canada Veronika Jekerle, European Medicines Agency (EMA) Susan Kirshner, CDER, FDA Robin Levis, CBER, FDA Mikhail Ovanesov, CBER, FDA Kimberly Schultz, CBER, FDA Mats Welin, Medical Products Agency

14:30 – 15:15 Networking Reception via Remo

15:15 Adjourn Day One Wednesday, October 14, 2020

NOTE: All times are Eastern Daylight Time (EDT)

10:45 – 11:00 Sign-on to Virtual Platform

Workshop Session Two:

Industry Perspectives and Challenges for Expedited Programs Session Chairs: Michelle Lytle, Eli Lilly and Company and Jason Starkey, Pfizer, Inc.

11:00 – 11:05 Introduction

11:05 – 11:30 Commercial Process Control Strategy Considerations for Cell Therapy Products Saravanamoorthy Rajendran, Celgene Corporation, A Bristol-Myers Squibb Company, Summit, NJ USA

11:30 – 11:55 Specific Regulatory Strategies for Process Validation for Biologics Diane Wilkinson, AstraZeneca, Macclesfield, United Kingdom

11:55 – 12:20 Taking Stability Off of the Critical Path of Product Development Andrew Lennard, Amgen Limited, Uxbridge, United Kingdom

12:20 – 12:45 Accelerated CMC Development and Considerations for the Global Post- approval Landscape Janet Tien, Genentech, a Member of the Roche Group, South San Francisco, CA USA

12:45 – 12:55 Break

12:55 – 14:00 PANEL DISCUSSION – Questions and Answers Joslyn Brunelle, Amgen Inc. Richard Keane, Biogen Idec Limited Andrew Lennard, Amgen Limited Saravanamoorthy Rajendran, Celgene Corporation, A Bristol-Myers Squibb Company Janet Tien, Genentech, a Member of the Roche Group Diane Wilkinson, AstraZeneca

14:00 Adjourn Day Two

Thursday, October 15, 2020

NOTE: All times are Eastern Daylight Time (EDT)

10:45 – 11:00 Sign-on to Virtual Platform

Workshop Session Three:

Phase Appropriate Development from a GMP Perspective Session Chairs: Lucy Chang, Merck & Co., Inc., Bharat Dixit, ClearB Therapeutics, Polly Mak, Genentech, a Member of the Roche Group and Paula Russell, Health Canada

11:00 – 11:05 Introduction

11:05 – 11:30 Phase Appropriate Regulatory Expectations for Microbial Control and Sterility Assurance for Expedited Programs Patricia Hughes, CDER, FDA, Silver Spring, MD USA

11:30 – 11:55 Validation and Implementation of an Automated Colony Counter for Product Testing in Biopharmaceutical Manufacturing Sven Deutschmann, Roche Diagnostics GmbH, Penzberg, Germany

11:55 – 12:20 Approaches and Challenges for Implementation of Phase-appropriate GMP Continuum Todd Mabe, Merck & Co., Inc., Wayland, MA USA

12:20 – 12:45 Phase Appropriate GMP Expectations for Analytical Methods and Process Validation from a Regulatory Perspective Megan Powdrill, Health Canada, Ottawa, ON Canada

12:45 – 12:55 Break

12:55 – 14:00 PANEL DISCUSSION – Questions and Answers Sven Deutschmann, Roche Diagnostics GmbH Patricia Hughes, CDER, FDA Kathy Lee, Eli Lilly and Company Todd Mabe, Merck & Co., Inc. Megan Powdrill, Health Canada Balaji Viswanathan, Pfizer, Inc.

14:00 – 14:15 Closing Remarks and Invitation to WCBP 2021 Special Edition Michael Kennedy, CBER, FDA Carmilia Jiménez-Ramirez, BioMarin Pharmaceutical Inc.

14:15 Adjournment