Welcome to Bioassays 2015: Scientific Approaches & Regulatory Strategies

Transcription

1 Welcome to Bioassays 2015: Scientific Approaches & Regulatory Strategies On behalf of the Scientific Organizing Committee and CASSS, we are excited to welcome you to Bioassays 2015: Scientific Approaches & Regulatory Strategies and look forward to your participation and input March 23-24, 2015 at the Sheraton Hotel in Silver Spring, Maryland. The CASSS Bioassays meeting has established itself as a premier conference and unique opportunity for participants and opinion leaders to discuss and debate current regulatory and industry topics regarding bioassays. Bioassays are a critical component of the analytical control strategies for biologics and other complex molecules. The ability of an assay to characterize and demonstrate biological activity is essential and developing such bioassays is becoming more difficult as biologic drugs are engineered to be more complex and/or have multiple modes of action. Companies are continuously challenged with developing assays that are biologically relevant for the analysis of multiple potential mechanisms. Bioassays are also used for lot release, stability, comparability and characterization studies, which requires that the assays be robust and, in most cases, suitable for a QC lab. Bioassays 2015 is structured to encourage attendee interaction. Each session includes case study presentations followed by a panel discussion allowing for lively dialogue between attendees from academia, industry and regulatory agencies. As in previous years, we expect this format to result in additional focus on the technical and regulatory details of the topic. Regulatory participation from the US FDA, Health Canada and various European agencies has been strong each year. In addition, an exhibitor showcase and poster reception at the end of Day One will give attendees the opportunity to present additional topics and continue the day's discussion in an informal setting. We would like to thank the speakers and the panel members who are giving generously of their time and resources and to you for your attendance. We would also like to acknowledge the generosity of our program partners for the continued support of the CASSS Bioassays meeting: AbbVie, Inc.; Biogen Idec and MedImmune. 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 meeting has been invaluable. We are sure you will find Bioassays 2015 to be informative and productive, and that it will provide you with current perspectives on bioassays. Scientific Organizing Committee: Thomas Arroll, Seattle Genetics, Inc., USA Evangelos Bakopanos, Health Canada, Canada Katrin Buss, Federal Institute for Drugs and Medical Devices, BfArM, Germany Chana Fuchs, CDER, FDA, USA (Co-chair) Denise Gavin, CBER, FDA, USA (Co-chair) Hélène Gazzano-Santoro, Genentech, a Member of the Roche Group, USA Stephen Hartman, AbbVie Inc., USA Xu-Rong Jiang, MedImmune, A member of the AstraZeneca Group, USA Helena Madden, Biogen Idec, USA (Co-chair) Bruce Meiklejohn, Eli Lilly and Company, USA (Co-chair) Thomas Anders Millward, Novartis Pharma AG, Switzerland Noel Rieder, Amgen Inc., USA Sally Seaver, Seaver Associates LLC, USA

2 The Scientific Organizing Committee gratefully acknowledges the following program partners for their generous support of Bioassays 2015: SUSTAINING PLATINUM PROGRAM PARTNERS AbbVie, Inc. Biogen Idec MedImmune, A member of the AstraZeneca Group SUSTAINING SILVER PROGRAM PARTNER Pfizer, Inc. PROGRAM PARTNERS Amgen Inc. Genentech, A Member of the Roche Group

3 EXHIBITOR PROGRAM PARTNERS DiscoveRx Corporation Eurofins Lancaster Laboratories PPD Promega Corporation Stegmann Systems GmbH

4 The Scientific Organizing Committee gratefully acknowledges the following media for their promotional consideration of Bioassays 2015: MEDIA PROGRAM PARTNERS American Laboratory American Pharmaceutical Review BioProcess International BioProcessing Journal Genetic Engineering & Biotechnology News International Pharmaceutical Quality Pharmaceutical Outsourcing RSC Advances Technology Networks Limited

5 Bioassays 2015: Scientific Approaches & Regulatory Strategies Scientific Program Summary Monday, March 23, :30 17:00 Registration in the Cypress Foyer 07:30 08:30 Continental Breakfast in the Magnolia Ballroom 08:30 08:45 CASSS Welcome and Introductory Comments in the Cypress Ballroom Helena Madden, Biogen Idec, Cambridge, MA USA Bioassays 2015 Welcome and Introductory Comments in the Cypress Ballroom Helena Madden, Biogen Idec, Cambridge, MA USA Potency Tests for Cell and Gene Therapy Products: Overcoming the Challenges Workshop Session One in the Cypress Ballroom Session Chairs: Denise Gavin, CBER, FDA and Sally Seaver, Seaver Associates LLC 08:45 08:50 Introduction 08:50 09:15 Bioassays in the Lifecycle of Cell & Gene Therapy Products: A Regulatory Perspective Xiaobin (Victor) Lu, CBER, FDA, Silver Spring, MD USA 09:15 09:40 Developing a Potency Assay Matrix for a Viral Vector Gene Therapy Product Barbara Thorne, Celladon Corporation, San Diego, CA USA 09:40 10:05 Potency Assay Development for Cell Therapy Products Erik Rutjens, Novartis Pharma AG, Basel, Switzerland 10:05 10:30 Bioassay Development for Human Stem Cell-derived Retinal Pigment Epithelium: Progress and Challenges Irina Klimanskaya, Ocata Therapeutics, Inc., Marlborough, MA USA 10:30 11:00 AM Break Visit the Exhibits and Posters in the Magnolia Ballroom 11:00 12:15 PANEL DISCUSSION Questions and Answers Irina Klimanskaya, Ocata Therapeutics, Inc., USA Xiaobin (Victor) Lu, CBER, FDA, USA Anthony Ridgway, Health Canada, Canada Erik Rutjens, Novartis Pharma AG, Switzerland Barbara Thorne, Celladon Corporation, USA

6 Monday, March 23 continued 12:15 13:30 Hosted Lunch in the Magnolia Ballroom Advances in Bioassay Technologies and Platforms Workshop Session Two in the Cypress Ballroom Session Chairs: Thomas Arroll, Seattle Genetics, Inc., Stephen Hartman, AbbVie, Inc. and Xu-Rong Jiang, MedImmune, A member of the AstraZeneca Group 13:30 13:35 Introduction 13:35 14:00 Development and Validation of a To-Be-Marketed Cancer Immunotherapy Antibody Max Tejada, Genentech, a Member of the Roche Group, South San Francisco, CA USA 14:00 14:25 An ADCP Reporter Bioassay for Assessing Fc Effector Function Shihua Lin, MedImmune, A member of the AstraZeneca Group, Gaithersburg, MD USA 14:25-14:50 In-vitro Bioassay Using mrna Transcription (Dual RT-qPCR) as Assay Response Michael Sadick, Catalent Pharma Solutions, Kansas City, MO USA 14:50-15:15 Expectations and Limitations of Bioassays: An EU Regulator s View Christian Mayer, AGES Austrian Agency for Health & Food Safety, Vienna, Austria 15:15-15:45 PM Break Visit the Exhibits and Posters in the Magnolia Ballroom 15:45-17:00 PANEL DISCUSSION Questions and Answers Evangelos Bakopanos, Health Canada, Canada Shihua Lin, MedImmune, A member of the AstraZeneca Group, USA Christian Mayer, AGES Austrian Agency for Health & Food Safety, Austria Michael Sadick, Catalent Pharma Solutions, USA Jennifer Swisher, CDER, FDA, USA Max Tejada, Genentech, a Member of the Roche Group, USA 17:00 17:15 Break

7 17:15 17:30 Introduction Monday, March 23 continued Exhibitor Partner Scientific Showcase in the Cypress Ballroom Session Chairs: 17:30 17:45 17:45 18:00 18:00 18:15 18:15 18:30 18:30 18:45 Eurofins Lancaster Laboratories Promega Corporation DiscoveRx Corporation Stegmann Systems GmbH PPD 18:45 19:00 Discussion Questions and Answers 19:00 21:00 Exhibitor and Poster Reception in the Magnolia Ballroom 21:00 Adjourn Day One

8 Tuesday, March 24, :00 17:00 Registration in the Cypress Foyer 07:30 08:30 Continental Breakfast in the Magnolia Ballroom Bioassays to Support Commercialization of Drug Products Workshop Session Three in the Cypress Ballroom Session Chairs: Hélène Gazzano-Santoro, Genentech, a Member of the Roche Group, Bruce Meiklejohn, Eli Lilly and Company and Thomas Millward, Novartis Pharma AG 08:30 08:35 Introduction 08:35 09:00 Health Canada s Lot Release Program & Potency Testing of Biotherapeutic Products Evangelos Bakopanos, Health Canada, Ottawa, ON Canada 09:00 09:25 Lifecycle of an Enzyme Activity Assay: Development and Validation to Postmarketing Commitments Loc Vo, BioMarin Pharmaceutical Inc., Novato, CA USA 09:25 09:50 Bioassay Strategies for Assessment of Co-stimulation Inhibitors in Immunooncology: Considerations from Development to Commercialization Jeffrey Glenn, Bristol-Myers Squibb Company, Princeton, NJ USA 09:50 10:15 Bioassay Evolution and Lessons Learned Thomas Luntz, Catalent Pharma Solutions, Morrisville, NC USA 10:15-10:45 AM Break Visit the Exhibits and Posters in the Magnolia Ballroom 10:45 12:00 PANEL DISCUSSION Questions and Answers Evangelos Bakopanos, Health Canada, Canada Jeffrey Glenn, Bristol-Myers Squibb Company, USA Thomas Lunz, Catalent Pharma Solutions, USA Emily Shacter, ThinkFDA, USA Craig Thelwell, National Institute for Biological Standards and Control (NIBSC), United Kingdom Loc Vo, BioMarin Pharmaceutical Inc., USA 12:00 13:15 Hosted Lunch in the Magnolia Ballroom

9 Tuesday, March 24 continued Bioassay Challenges during Product Globalization Workshop Session Four in the Cypress Ballroom Session Chairs: Katrin Buss, Federal Institute for Drugs and Medical Devices, BfArM, Helena Madden, Biogen Idec and Noel Rieder, Amgen Inc. 13:15 13:20 Introduction 13:20 13:45 Global Company, Global Products, Local Laboratories Camille Dycke, F. Hoffmann-La Roche Ltd., Basel, Switzerland 13:45 14:10 Collaborative Study for Proposed 1 st International Standard for PEGylated G-CSF (PEG-G-CSF) Meenu Wadhwa, National Institute for Biological Standards and Control (NIBSC), South Mimms, United Kingdom 14:10 14:35 Bioassay Strategy to Support the Transfer of a Monoclonal Antibody for Manufacturing and Filing in China Justin Jia, WuXi AppTec, Shanghai, China 14:35 15:00 Strategies to Assist Global Regulatory Acceptance of Assurance of Potency Anthony Mire-Sluis, Amgen Inc., Thousand Oaks, CA USA 15:00 15:30 PM Break Visit the Exhibits and Posters in the Magnolia Ballroom 15:30 16:45 PANEL DISCUSSION Questions and Answers Katrin Buss, Federal Institute for Drugs and Medical Devices, BfArM, Germany Camille Dycke, F. Hoffmann-La Roche Ltd., Switzerland Chana Fuchs, CDER, FDA, USA Justin Jia, WuXi AppTec, China Anthony Mire-Sluis, Amgen Inc., USA Meenu Wadhwa, National Institute for Biological Standards and Control (NIBSC), United Kingdom 16:45 17:00 Bioassays Workshop Recap Closing Remarks and Invitation to Bioassays 2016 Bruce Meiklejohn, Eli Lilly and Company, Indianapolis, IN USA 17:00 Adjournment

10 Potency Tests for Cell and Gene Therapy Products: Overcoming the Challenges Session Abstract Session Chairs: Denise Gavin, CBER, FDA and Sally Seaver, Seaver Associates LLC Cell and Gene therapies (CGT) are an evolving class of promising biological products. However, despite over two decades of investigation there are still few cell and gene therapies (CGT) that are commercially available or in late stage clinical development. Successful development of any biological therapeutic requires full product characterization to ensure safety and efficacy. Animal or cell based bioassays are a critical part of this product characterization. Usually several bioassay candidates are developed as potential potency tests, and the most robust, quantitative and QC friendly method that can be validated is selected to become the potency test for lot release and monitoring lot stability. The other bioassays are reserved for product characterization and comparability assessment after process changes including scale-up. To date few CGT have had robust and meaningful potency tests. Some of the challenges faced by developers of potency tests for CGT products include (1) extremely short product shelf life (hours to days), (2) the need to express a GT vector in one cell type and to quantitate its biological activity in a second cell type, (3) developing bioassays for all of the active ingredients, especially for multi-gene or multi-cell products, (4) developing bioassays that measure relevant biological activities (5) defining and banking a reference standard for these products, esp. if the product is autologous, and (6) determining which bioassays need to be executed for routine lot release and stability assessments. In addition, implementing a potency assay early in CGT product development is critical for understanding the product attributes related to biological activity and clinical efficacy. This session will provide an overview of regulatory expectations for measuring potency for CGT products as well as several case studies describing how these challenges were overcome to develop potency tests for lot release and stability and comparability assessments. NOTES:

11 Advances in Bioassay Technologies and Platforms Session Abstract Session Chairs: Thomas Arroll, Seattle Genetics, Inc., Stephen Hartman, AbbVie, Inc. and Xu-Rong Jiang, MedImmune Bioassays are the only test methods that quantify a drug s biological activity and thus play a crucial role in the characterization of biologic therapies. The interplay of living cells, biologically active reagents, various quantifiable readouts, multi-step procedures, specific instrumentation, and complex data analysis makes the development of a robust, accurate, precise bioassay extremely challenging. Products with multiple MoAs pose an even greater challenge. The increasing complexity of large molecule modalities (such as ADCs, bispecifics/bifunctionals, and immunomodulators) and their multiple biological activities also greatly increases the complexity of bioassay development. Many new technologies, approaches, and platforms can facilitate bioassay development, as well as provide additional information or performance enhancements, but they may also introduce new challenges. This session will discuss some recent advances in technologies and platforms that have improved bioassay method performance, facilitated/accelerated development and implementation, and revealed new/additional information about the product. We will also address the challenges faced when introducing a new technology/approach and consider the risks and benefits to making these changes. We will learn about bioassay approaches used for cancer immunotherapy products and assessing effector functions, as well as learn about alternative bioassay readouts. We will also discuss regulatory agency experiences, opinions, and expectations regarding the use of new bioassay method technologies. NOTES:

12 Bioassays to Support Commercialization of Drug Products Session Abstract Session Chairs: Hélène Gazzano-Santoro, Genentech, a Member of the Roche Group, Bruce Meiklejohn, Eli Lilly and Company and Thomas Millward, Novartis Pharma AG As a product progresses through clinical development towards commercialization, the requirements and expectations of the bioassays used to release and characterize the product evolve. By the time the product is approaching registration, the bioassay needs to be fully validated, and specifications need to be defined and justified. But in addition, the relationship between the bioassay and the clinical mode of action needs to be clearly established and reagents that are critical for the bioassay need to be under control and their supply guaranteed. For some products more than one bioassay may be needed for commercial release, and these bioassay(s) may need to be transferred to one or more QC testing labs. This session will discuss these and other aspects relating to bioassays during product commercialization. NOTES:

13 Bioassay Challenges during Product Globalization Session Abstract Session Chairs: Katrin Buss, Federal Institute for Drugs and Medical Devices, BfArM, Helena Madden, Biogen Idec and Noel Rieder, Amgen Inc. Global drug registration enables the biopharmaceutical industry to enter markets all over the world and to provide life-changing medicines to patients everywhere. This requires a strategic approach to analytics from early drug development throughout post-approval lifecycle management. Analytical control strategies are influenced by country and region specific expectations and testing requirements that result in added complexity. Scientists and regulators must work together to understand regional requirements and best approaches to ensure successful licensure and assurance of product quality. Considerations for globalization of bioassays include: instrument and software selection, critical reagent availability, method robustness and reproducibility, and the requirement for registration and import testing. These challenges are more daunting when considered in the face of likely language, time and cultural differences. This session will explore and contrast requirements from several areas of the world. We will also discuss strategies for selection, development, transfer, performance and maintenance of a bioassay to support successful biopharmaceutical globalization. NOTES: