PRELIMINARY PROGRAM Bay Area Discussion Group Co-chairs: Min Chen, Ultragenyx Pharmaceutical Vinaya Kapoor, Janssen, Pharmaceutical Companies of Johnson & Johnson South San Francisco Conference Center South San Francisco, CA June 9, 2016 Table of Contents Conference Program Partners…………………………………………………………….…….3 Acknowledgements……………………………………………………………………………..4 General Information…………………………………………………………………………….5 Scientific Program Summary……………………………………………………………...……6 Program and Speaker Abstracts………………………………………………………………...7 Roundtable Discussion Point Sheets……………………………………………………………8 Note Paper……………………………………………………………………………………… The Organizing Committee gratefully acknowledges the Conference Program Partners for their generous support of this Bay Area Discussion Group workshop Program Partners Agilent Technologies Boehringer Ingelheim Genentech, a Member of the Roche Group Acknowledgements Special Thanks to all of the Program Committee Members who helped develop this Bay Area Discussion Group Workshop Program Committee Kris Antonsen, BioMarin Pharmaceutical, Inc. Dawn Benson, Coherus Min Chen, Ultragenyx Pharmaceutical Judy Chou, Medivation Kathy Francissen, Genentech, a Member of the Roche Group Malou Gemeniano, Boehringer Ingelheim Vinaya Kapoor, Janssen, Pharmaceutical Companies of Johnson & Johnson David Passmore, Bristol-Meyers Squibb Company Aran Paulus, Thermo Fisher Scientific Joanne Severs, Bayer Lance Wong, Strand Bio DBA Christopher Yu, Genentech, a Member of the Roche Group Eike Zimmermann, Boehringer Ingelheim Roundtable Facilitators Dawn Benson, Coherus Adam Catherman, Genentech, a Member of the Roche Group Susan Chen, Boehringer Ingelheim Natalie Ciaccio, BioMarin Pharmaceutical, Inc. Guifeng Jiang, Boehringer Ingelheim Julian Kissmann, Boehringer Ingelheim Srikanth Kotapati, Bristol-Myers Squibb Weijun Li, Bayer Ying Liu, Janssen, Pharmaceutical Companies of Johnson & Johnson Rob McCombie, Genentech, a Member of the Roche Group Yanli Mi, Gilead Steven Rabin, Janssen, Pharmaceutical Companies of Johnson & Johnson Nicole Samuels, Bayer Jennifer Thompson, Agilent Technologies Audio Visual: Michael Johnstone, MJ Audio-Visual Productions CASSS Staff Stephanie L. Flores, CAE, Executive Director Anna Lingel, Registration Manager Linda Mansouria, CMP, CMM, Conference Manager General Information Name Badges Please wear your name badge throughout the day. Registration The registration desk will be open from 8:00 a.m. to 3:30 p.m. We will accept walk-in registrations. Registration is located in the Baden A & B Foyer at the South San Francisco Conference Center. Roundtable Session There are 7 roundtable topics for you to determine which table topic you would like to be part of the discussion. The 7 table topics are: Table Topic 1: Don’t Get in a Bind Over Specifications Table Topic 2: Leveraging Platform Information Appropriately Table Topic 3: S-T-A-B-I-L-I-T-Y – You Ain’t Got No Alibi: Stability Strategies for Phase I and Beyond Table Topic 4: Structural Characterization Table Topic 5: Global Filing Challenges Table Topic 6: To Infinity and Beyond – Analytical Method Development Table Topic 7: Working with Contract Manufacturers Tables are set with 10 seats to a table. Table topics are on a first come, first serve basis. These roundtables will include two facilitators, whose role is to help assist the discussion and ensure a lively exchange, and a scribe, whose role is to make general, anonymous notes about the discussion so others can have a change to view the discussion even if they could not participate. The discussion notes will be shared with all attendees during the summary period of the program. Wi-Fi Access The South San Francisco Conference Center has complimentary Wi-Fi Access. Log into SSFCC- FreeWiFi network. Scientific Program Summary THURSDAY, JUNE 9, 2016 08:00 – 15:30 Registration in the Baden Room Foyer 08:00 – 09:00 Continental Breakfast in Salons B-D 09:00 – 09:30 CASSS Welcome and Introductory Comments Kathleen Francissen, Genentech, a Member of the Roche Group 09:30 – 10:15 Building A Quality Dossier Howard Anderson, CDER, FDA 10:15 – 11:00 Let’s Start at the Very Beginning, a Very Good Place to Start! Building and Maintaining a Quality CMC Dossier Michelle Frazier, AbbVie, Inc. 11:00 – 11:30 AM Break in Salons B - D 11:30– 12:15 Panel Discussion Facilitated by: Vinaya Kapoor, Janssen, Pharmaceutical Companies of Johnson & Johnson Panel Members: Howard Anderson, CDER, FDA Michael Boyne, BioTech Logic Michelle Frazier, AbbVie, Inc. Malou Gemeniano, Boehringer Ingelheim Lisa Regan, Bayer 12:15 – 13:15 Networking Lunch in Salons B-D 13:15 – 14:00 Roundtable Discussions in Baden Room 14:00 – 14:15 PM Break and Roundtable Summaries Created in the Baden Room 14:15 – 15:15 Summary of Roundtable Discussions by Table Facilitators 15:15 – 15:30 Closing Remarks Lance Wong, Strand Bio DBA Program Abstract Building a “Quality” Dossier from Phase 1 and Beyond Development of a “Quality” Dossier containing the appropriate level of Chemistry, Manufacturing and Control information is an integral element of global regulatory submissions to support clinical development programs. First and foremost, regulations and guidance must be considered as the dossier is being prepared. Additionally, the phase of development, molecule complexity, and available molecule knowledge are key elements to consider when developing the dossier content. This meeting will provide an interactive forum for individuals from academia, industry and the FDA to come together to discuss the challenges and best practices for preparing a phase appropriate “Quality” Dossier. Key discussion topics will include, establishing specifications, analytical method development, structural characterization, stability approaches, and leveraging platform information when preparing dossiers to support global clinical programs. The meeting format is designed to provide attendees the opportunity to hear current perspectives from the FDA and Industry and to network through participation in facilitated small group discussions. Speaker Abstracts An FDA Reviewer’s View on Building a Quality Dossier Howard Anderson, CDER/OPQ/OBP, FDA The number of FDA approved original Biological License Applications (BLA) for monoclonal antibody and therapeutic protein products is growing at an accelerated rate. In 2015, CDER approved 45 novel new drug therapies. Twelve of which are BLA products. Product quality submissions to regulatory authorities are a critical component for successful drug development. This presentation will provide a FDA product reviewer’s perspective on building a quality dossier from the pIND meeting through filling BLA supplements for product life cycle management. Focused quality regulatory submissions make FDA sponsor meeting more productive, facilitate application review, and best utilize FDA resources for application decisions. In contrast a deficient submission may delay the product development program. The goal for all stake holders is to facilitate the rapid clinical development and approval of high quality biopharmaceuticals for patients. Case studies will be provided for biopharmaceutical monoclonal antibody and therapeutic protein products regulated by the Office of Biotechnology Products (OBP) at CDER. The case studies will illustrate lessons learned and best practices for providing adequate information to the FDA at the various stages of product development. The examples involve analytical method development, product characterization, justifying specifications, and stability commitments. Let’s Start at the Very Beginning, a Very Good Place to Start! Building and Maintaining a Quality CMC Dossier Michelle Frazier, AbbVie Inc. High quality submissions are essential for the success of any development program from pre-first in human (FIH) through post-licensure, whether they are meeting packages or Module 3 quality sections in support of a clinical trial application or a marketing application. In contrast, submissions that are not focused and do not contain the appropriate level of detail or data can have a negative outcome for a given clinical program. This presentation will outline Industry best practices and lessons learned in building quality regulatory submissions throughout the product lifecycle. Roundtable Discussion Points TABLE 1 TOPIC: Don’t Get in a Bind over Specifications – Setting Appropriate Specifications FACILITATORS: Ying Liu, Janssen, Pharmaceutical Companies of Johnson & Johnson Yanli Mi, Gilead SCRIBE: Kris Antonsen, BioMarin Pharmaceutical, Inc. SCOPE: Setting realistic specifications for analytical analyses is an exercise that almost every company conducts routinely. However, the strategies and practices could be diverse. We will discuss strategies for setting specifications and what to consider when setting specifications. DISCUSSION POINTS: 1. Are quantitative specifications required or is report results sufficient? a. When is “Report Results” acceptable for early-phase products? What analyses (appearance, ID, assay, impurities/degradation products, counterion potency, etc.)? b. Should “Report Results” be used alone or should a “Target” be listed as well? c. DS versus DP? Does this matter? 2. What is the focus for setting specifications for early-phase products? Patient safety, efficacy, etc? a. Typically, early-phase products focus on patient safety first and foremost. Is this the correct approach? What else should be focused on for early-phase products? b. What other attributes with regards to specifications should be focused on