Welcome to the CMC Strategy Forum

Transcription

1 Welcome to the CMC Strategy Forum 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: Amgen Inc., Biogen, Bristol- Myers Squibb Company, Celgene Corporation, Eli Lilly and Company, Genentech, a Member of the Roche Group, MedImmune, A member of the AstraZeneca Group, Merck & Co., Inc., Novo Nordisk A/S, and Pfizer Inc. We are grateful for the expert management from CASSS and the audio-visual expertise of Michael Johnstone from MJ Audio-Visual Productions. Their experience and guidance in the preparation of this Forum has been invaluable.

2 ACKNOWLEDGEMENTS CMC STRATEGY FORUM NORTH AMERICA PROGRAM COMMITTEE Siddharth Advant, Celgene Corporation Kristopher Barnthouse, Janssen Pharmaceuticals R&D LLC Barry Cherney, Amgen Inc. Fiona Cornel, Health Canada JR Dobbins, Eli Lilly and Company Michael Kennedy, CBER, FDA Taro Fujimori, AbbVie Bioresearch Center, Inc. Sarah Kennett, CDER, FDA Joseph Kutza, MedImmune, A member of the AstraZeneca Group Emanuela Lacana, CDER, FDA Kimberly May, Merck & Co., Inc. Anthony Mire-Sluis, AstraZeneca Stefanie Pluschkell, Pfizer, Inc. Nadine Ritter, Global Biotech Experts, LLC Timothy Schofield, GlaxoSmithKline Zahra Shahrokh, ZDev Consulting Jason Starkey, Pfizer, Inc. Andrew Weiskopf, Biogen Heidi Zhang, Juno Therapeutics, A Celgene Company Marcel Zocher, Bristol-Myers Squibb Company CMC STRATEGY FORUM GLOBAL STEERING COMMITTEE Siddharth Advant, Celgene Corporation, USA Daniela Cerqueria, ANVISA-Brasilian National Health Surveillance Agency, Brasil Yasuhiro Kishioka, PMDA-Pharmaceutical and Medical Devices Agency, Japan Steven Kozlowski, CDER, FDA, USA Junichi Koga, Daiichi Sankyo Co., Ltd., Japan Rohin Mhatre, Biogen, USA Anthony Mire-Sluis, AstraZeneca., USA Wassim Nashabeh, F. Hoffmann-La Roche Ltd., Switzerland (Chair) Ilona Reischl, BASG-Federal Office for Safety in Health Care, Austria Anthony Ridgway, Health Canada, Canada Nadine Ritter, Global Biotech Experts, LLC, USA Thomas Schreitmüller, F. Hoffmann-La Roche Ltd., Switzerland Mark Schenerman, CMC Biotech-MAS Consulting, USA Karin Sewerin, BioTech Development AB, Sweden

3 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 Genentech, a Member of the Roche Group STRATEGIC PLATINUM PROGRAM PARTNERS Amgen Inc. Biogen MedImmune, A member of the AstraZeneca Group STRATEGIC GOLD PROGRAM PARTNERS Eli Lilly and Company Novo Nordisk A/S Pfizer, Inc. STRATEGIC SILVER PROGRAM PARTNER Merck & Co., Inc. FORUM PROGRAM PARTNERS Bristol-Myers Squibb Company Celgene Corporation

4 The Scientific Organizing Committee gratefully acknowledges the following media for their promotional consideration of the CMC Strategy Forum North America July 2018: LEADING MEDIA PARTNERS BioProcess International International Pharmaceutical Quality MEDIA PARTNERS American Laboratory / LabCompare American Pharmaceutical Review The Analytical Scientist BioProcessing Journal Genetic Engineering & Biotechnology News The Pathologist Pharmaceutical Outsourcing Technology Networks

5 Forum Abstract Next Generation Biotechnology Product Development, Manufacturing and Control Strategies FORUM CO-CHAIRS: Sarah Kennett, Genentech, a Member of the Roche Group Anthony Mire-Sluis, AstraZeneca SCIENTIFIC ORGANIZING COMMITTEE: Siddharth Advant, Celgene Corporation Cristina Ausin, CDER, FDA Barry Cherney, Amgen Inc. Steven Falcone, Sanofi Jie He, CBER, FDA Alexey Khrenov, CBER, FDA Mike Tarlov, NIST-National Institute of Standards and Technology Kimberly Wolfram, Biogen The future of biotechnology manufacturing has to enable the drivers facing the biotechnology industry the need to decrease cost of goods, market globalization, more rapid development of pipeline products, reduced risk to patient supply, increased product quality etc. The utilization of new technologies such as single use bioreactors, alternating tangential flow during fermentation, modular and closed process equipment, process analytical technology and Analytics of the Future will allow for a more robust process, reduced waste, increased titers, fewer non-conformances, less staffing required and dramatically less risk to technology transfer to new sites. The ability to utilize single use, pre-sterilized bioreactors and raw materials in a closed process eliminates the need for cleaning and steaming in place and reduces the risk of contaminations. The ability to create modular, essentially identical, manufacturing sites across the globe means that technology transfer will require much less effort and risk. PAT and analytics of the future should allow for much more real time control of the process and less reliance on lot release assays and for driving more testing onto the manufacturing floor and reducing the costly footprint of the Quality Control Laboratories. In addition, advances in molecular modeling and cell cloning, and the ability to model processes such as column chromatography should allow for improved titers, less molecular variability and more rapid process development. However, the use of these novel technologies and the design of future biotechnology manufacturing sites will challenge regulatory paradigms and hence the need to partner with regulatory authorities as the future becomes a reality.

6 CMC Strategy Forum Program Summary Next Generation Biotechnology Product Development, Manufacturing and Control Strategies Monday, July 16, :30 17:00 Registration in the Washingtonian Ballroom Foyer 07:30 08:30 Breakfast in the Washingtonian Ballroom Foyer 08:30 08:45 CASSS Welcome and Introductory Comments in Salons D - G Nadine Ritter, Global Biotech Experts, LLC CMC Strategy Forum Welcome and Introductory Comments in Salons D - G Sarah Kennett, Genentech, a Member of the Roche Group Anthony Mire-Sluis, AstraZeneca Continuous and Advances in Manufacturing Upstream Workshop Session One in Salons D - G Session Chairs: Barry Cherney, Amgen Inc. and Siddharth Advant, Celgene Corporation 08:45 09:10 TBD Lada Laenen, Sanofi, Framingham, MA USA 09:10 09:35 Next Generations Manufacturing with a Focus on Bioreactor Perfusion Technology Frank Ritacco, Bristol-Myers Squibb Company, Hopewell, NJ USA 09:35 10:00 Raman Spectroscopy Monitoring of Product Quality in CHO Cell Culture Brian Horvath, Genentech, a Member of the Roche Group, South San Francisco, CA USA 10:00 10:25 Review of Upstream Manufacturing Platforms for Viral Vectors Christopher Murphy, Brammer Bio, Cambridge, MA USA 10:25 10:45 Networking Break in the Washingtonian Ballroom Foyer 10:45 12:00 PANEL DISCUSSION Questions and Answers Brian Horvath, Genentech, a Member of the Roche Group, South San Francisco, CA USA Nobuko Katagiri, CBER, FDA Lada Laenen, Sanofi, Framingham, MA USA Christopher Murphy, Brammer Bio, Cambridge, MA USA Frank Ritacco, Bristol-Myers Squibb Company, Hopewell, NJ USA Leslie Rivera-Rosado, CDER, FDA, Silver Spring, MD USA

7 Monday, July 16 continued 12:00 13:30 Networking Lunch in the Washingtonian Ballroom Foyer Continuous and Advances in Manufacturing Downstream Workshop Session Two in Salons D - G Session Chairs: Cristina Ausin, CDER, FDA and Steven Falcone, Sanofi 13:30 13:55 Regulatory Perspective on New Developments in Manufacture of Biological Products Scott Nichols, CDER, FDA, Silver Spring, MD USA 13:55 14:20 Multi Column Chromatography: A Major Step Towards Continuous Purification of Biopharmaceuticals Rickey Lu, MedImmune, A member of the AstraZeneca Group, Frederick, MD USA 14:20 14:45 Recent Developments in Downstream Bioprocessing: Multimodal Chromatography, Affinity Precipitation and Integrated Bioprocessing Steven Cramer, Rensselaer Polytechnic Institute, Troy, NY USA 14:45 15:10 Commercialization of an Integrated Continuous Biomanufacturing Process Franqui Jimenez, Sanofi, Framingham, MA USA 15:15 15:45 Networking Break in the Washingtonian Ballroom Foyer 15:45 17:00 PANEL DISCUSSION Questions and Answers Sarah Nilou Arden, CDER, FDA, Silver Spring, MD USA Steven Cramer, Rensselaer Polytechnic Institute, Troy, NY USA Franqui Jimenez, Sanofi, Framingham, MA USA Rickey Lu, MedImmune, A member of the AstraZeneca Group, Frederick, MD USA Scott Nichols, CDER, FDA, Silver Spring, MD USA 17:00 18:30 Networking Reception in the Washingtonian Ballroom Foyer 18:30 Adjourn Day One

8 Tuesday, July 17, :00 17:00 Registration in the Washingtonian Ballroom Foyer 07:45 08:45 Breakfast in the Washingtonian Ballroom Foyer Modelling and Control Strategies Workshop Session Three in Salons D - G Session Chairs: Sarah Kennett, Genentech, a Member of the Roche Group and Kim Wolfram, Biogen 08:45 09:10 Leveraging an Integrated Data Platform to Advance Process Understanding and Enhance Continued Process Verification (CPV) Patrick Gammell, Amgen Inc., Thousand Oaks, CA USA 09:10 09:35 In-line Monitoring and Real-time Release Michalle Adkins, Emerson Automation Solutions, Royersford, PA USA 09:35 10:00 Advanced Process Controls and/or Process Modeling for Biopharmaceutical Manufacturing in Practice in a New Manufacturing Plant Saly Romero-Torres, Biogen, Cambridge, MA USA 10:00 10:25 DSP Meets Data Science: Applying Modeling and Machine Learning for Bioprocess Development Ferdinand Stueckler, F. Hoffmann-La Roche Ltd., Penzberg, Germany 10:25 10:45 Networking Break in the Washingtonian Ballroom Foyer 10:45 12:00 PANEL DISCUSSION Questions and Answers Michalle Adkins, Emerson Automation Solutions, Royersford, PA USA Aikaterini Alexaki, CBER, FDA Silver Spring, MD USA Katia Ethier, Health Canada, Ottawa, Canada Patrick Gammell, Amgen Inc., Thousand Oaks, CA USA Thomas O Connor, CDER, FDA, Silver Spring, MD USA Saly Romero-Torres, Biogen, Cambridge, MA USA Ferdinand Stueckler, F. Hoffmann-La Roche Ltd., Penzberg, Germany 12:00 13:30 Networking Lunch in the Washingtonian Ballroom Foyer Emerging Analytical Technologies Workshop Session Four in Salons D - G Session Chairs: Mike Tarlov, NIST-National Institute of Standards and Technology and Katia Ethier, Health Canada 13:30 13:55 Regulatory Perspective on Emerging Analytical Technologies Yan Wang, CDER, FDA, Silver Spring, MD USA 13:55 14:20 Analytics for Messenger RNA Products Charles Bowerman, Moderna Therapeutics, Cambridge, MA USA

9 Tuesday, July 17 continued 14:20 14:45 Analytical Strategies to Measure Heterogeneous Protein-Protein Interactions in Formulated Therapeutic Protein Solutions George Svitel, Merck Research Laboratories, Kenilworth, NJ USA 14:45 15:10 Next Generation Nanoscale Biosensors using Single Walled Carbon Nanotubes Corona Phase Molecular Recognition Michael Strano, Massachusetts Institute of Technology, Cambridge, MA USA 15:10 15:30 Networking Break in the Washingtonian Ballroom Foyer 15:30 16:45 PANEL DISCUSSION Questions and Answers Charles Bowerman, Moderna Therapeutics, Cambridge, MA USA Anil Choudhary, CBER, FDA, Silver Spring, MD USA Manju Joshi, CBER, FDA, Silver Spring, MD USA Michael Strano, Massachusetts Institute of Technology, Cambridge, MA USA George Svitel, Merck Research Laboratories Yan Wang, CDER, FDA, Silver Spring, MD USA 16:45 17:15 Recap of Program Summary Slide Presentation Anthony Mire-Sluis, AstraZeneca 17:15 17:30 Invitation to CMC Strategy Forum January :30 Adjournment