Economical Approaches to Meeting Global Demand for High Value Biopharmaceuticals

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Economical Approaches to Meeting Global Demand for High Value Biopharmaceuticals Howard L. Levine and Susan Dana Jones BPI Europe 2013 Apr 17-18, 2013 Dusseldorf, Germany BioProcess Technology Consultants www.bptc.com

Current Pharmaceutical Market 1,000 800 Sales ($B) 2010 2011 Traditional 775 839 Biopharmaceutical 106 117 Total 881 956 Sales (Billions USD) 600 400 200 0 2002 2003 2004 2005 2006 2007 2008 2009 2010 2011 Traditional Pharmaceuticals Biopharmaceuticals

Top Ten Biopharmaceutical Products US Product Name Company 2011 Sales (Billions USD) Remicade J&J/Merck 8.159 Humira Abbott 7.932 Enbrel Amgen/Pfizer 7.367 Total 2011 sales of almost $60 billion Six are produced in cell culture Seven are monoclonal antibody related products CAGR for monoclonal antibody related products 2001 2010 was >40% Rituxan Roche 6.795 Avastin Roche 5.988 Herceptin Roche 5.944 Lantus Sanofi Aventis 5.453 Neulasta Amgen 3.952 Novolog Novo Nordisk 3.940 Lucentis Roche/Novartis 3.773 Rader RA. BPI 2013

Demand for Mammalian Cell Culture Capacity Total Volume (1,000s L) 3,000 2,500 2,000 1,500 1,000 500 0 2011 2012 2013 2014 2015 2016 2017 Commercial BLA/NDA Phase 3 Phase 2 Phase 1 Clinical 4,500 Well matched to supply Installed Capacity (1,000s L) 3,750 3,000 2,250 1,500 750 0 2011 2012 2013 2014 2015 2016 2017 CMO Excess Product

Global Distribution of Cell Culture Capacity 2012 2018 North America 1,821 1,963 UK/Ireland 196 241 Europe 744 954 Korea 60 270 Japan 115 115 China <1 125 India 14 35 Singapore/ Malaysia 176 264 Australia <1 3 Russia 0 7 2012 2018 Note: Perfusion capacity adjusted to equivalent fed batch capacity where appropriate South America 0 4 Volume in 000s L

Evolution of biomanufacturing facilities Increasing productivity State of the art late 1990s: 1 g/l expression; 60% overall yield Then: 100 Kg requires 17 runs at 10,000 L State of the art 2013: 5 g/l expression; 75% overall yield Now: 100 Kg requires 14 runs at 2,000 L Personalized medicine puts focus on smaller markets and niche products Improved product potency reduces capacity requirements 20,000 L Smaller markets Improved potency Manufacturing Scale 2,000 L Increased titer and yields 200 L Time

Advances in Single-use Technologies Up to 40% reduction in capital and utility Single use bioreactors available at scales up to 2,000 L Facilitates co location of manufacturing facilities with markets

Demand for Biosimilars All major markets now have biosimilar legislation in place Large players are increasingly entering this segment Biosimilar versions of first generation biopharmaceuticals already approved in many markets As of Sep 2011 Source: Decision Resources Many monoclonal antibodies will be off patent in the next few years Biosimilar monoclonal antibodies already available in India and China As of Sep 2011 Source: Decision Resources

Changing Biomanufacturing Space Requirements Upstream Upstream Processing Processing Downstream Processing Downstream Processing Relative Floor Space Traditional Facility Relative Floor Space Facility of the Future

Solutions for Future Biomanufacturing Needs Design and build standardized, pre engineered modular solutions that can be readily configured and combined to provide a facility that can be rapidly designed, built, and deployed globally Minimize cost by minimizing need for re engineering Maximize flexibility by allowing easy re configuration Improved flexibility to support a variety of biopharmaceutical products Reduce project risk through off site design, construction, and prequalification of modules, including process equipment Ca. 10% Site Specific Ca. 75% Global Standard Design Solution Overall project cost distribution Ca. 15% Customization Adapted from F. Eneqvist Achema 2012

Design of a Modular Facility 1. Each system analyzed and the process functions modularized 2. Modularized functions displayed in adjacency diagram Process modules and support modules creates a functional layout Mixing Module Separation Module Phasing Scope 3. Adjacency diagrams developed into architectural layouts Grid system to support both modular and conventional building One building module can consist of several process modules Construction can be phased in time to match increased capacity requirements Adapted from F. Eneqvist Achema 2012

Types of Modules Indoor modules for placement in existing building Indoor modules placed on concrete slab Pipe racks above or below modules Piers to create space for drains, pipe racks etc. HVAC placed on top of or beside modules Modular facility as a free standing building with its own façade and roofing Built on concrete slab foundation with piers to create space for drain/waste pipes etc. Modular components can placed on top of or next to an existing building Process skids and other pre assemblies Adapted from F. Eneqvist Achema 2012

Facility of the Future Standardized design based on standard platform process for monoclonal antibody production Readily adaptable to alternative processes and product types GMP manufacturing areas built using modular construction Based on maximum use of disposable technology, including disposable bags for buffer, media, and product storage and single use bioreactors Overall facility design includes Administration Process development laboratories Bulk manufacturing facility Aseptic fill/finish facility QC laboratories Warehouse Process and facility design developed in collaboration with KeyPlants AB

Biomanufacturing Facility Design Basis Overall design based on standard monoclonal antibody platform process Standard design incorporates single use bioreactors Economic model shows lower costs Operating Costs Stainless Steel Facility Single-Use Facility Annual Batch Annual Batch Quantity of Product Produced 105 Kg 7 Kg 105 Kg 7 Kg Materials $607,428 $40,495 $590,334 $39,356 Consumables $3,611,689 $240,779 $4,268,890 $284,593 Labor (Direct/Indirect) $17,133,065 $1,142,204 $12,011,414 $800,761 Maintenance/Utilities/Waste $2,268,686 $151,246 $1,454,109 $96,941 Total $23,620,868 $1,574,725 $18,324,748 $1,221,650 Cost Per Gram Monoclonal Antibody $225/gram $175/gram

Standardized Modular Drug Substance Manufacturing Suite Air Lock Air Lock Locker Final Purification and bulk filling Cell Culture and Purification Locker Media and Buffer Preparation Dispensing Raw Material storage Consumables storage Equipment Storage Washing Seed Lab Air Lock

Bulk Manufacturing Area GMP production area divided into six functional modules: Material Staging and Dispensing Media and Buffer Preparation and Storage Cell Culture and Purification Inoculum preparation Bioreactor harvest Pre virus removal purification operations Final Purification Post virus removal purification operations Formulation and bulk filling Support Areas Air Locks and Corridors Raw Material Media and Buffer

Segregation of Operations Minimizes Risk Multiple air handling zones cover different process areas Segregated pre and post virus removal steps Maximize use of closed processes and unit operations where possible Grade D Open processing areas separated to avoid cross contamination Grade C Use of laminar flow bio safety cabinets where appropriate

Flexible Design to Adjust Area Segregation Standard design open ballroom configuration Airlocks and Corridors Final Purif. Cell Culture and Purification Material Staging and Dispensing Media and Buffer Preparation/Hold Support areas Airlocks and Corridors Inoculum

Flexible Design to Adjust Area Segregation Standard design open ballroom configuration Add segregation between cell culture and initial purification Airlocks and Corridors Final Purif. Media and Buffer Preparation/Hold Initial A/L Cell Purification Culture and Purification Cell Culture and Harvest Material Staging and Dispensing Support areas Airlocks and Corridors Inoculum

Flexible Design to Adjust Area Segregation Standard design open ballroom configuration Add segregation between cell culture and initial purification Add column cleaning and packing areas for reusable columns Airlocks and Corridors Final Purif. Media and Buffer Preparation/Hold Initial A/L Cell Purification Culture and Purification Cell Culture and Harvest Material Staging and Dispensing Support areas Airlocks and Corridors Inoculum

Readily Scalable Design for Future Capacity Optimized adjacencies facilitate easy expansion

Fully Integrated Facility Easy expansion and connections according to needs Add on for utilities Connection to administration areas Connection to development and QC laboratories Connection to warehouse Add on separate module for aseptic fill/finish, packaging, and labeling

Accelerated Construction Timeline Standardized design and modular construction allow predictable construction schedule with Enhanced cash flow, releasing funds for new investments Higher Net Present Value for faster recovery of capital expenditures

Modular BioSolutions TM Bulk Biomanufacturing Facility Predictable 12 month construction schedule Flexible design GMP Compliant High quality Expandable and movable Single use bioreactors Standard design 1 x 2,000 L SUB Scalable up to 2 x 2000 liter SUBs Adaptable to single use equipment for downstream processing

Acknowledgments BioProcess Technology Consultants Susan Dana Jones Rick Stock Tom Ransohoff KeyPlants Jan Lilja Åsa Gaasvik Fredrik Eneqvist Hans Hummel Pär Almhem

Thank You! Howard L. Levine, Ph.D. hlevine@bptc.com +1.781.281.2703 BioProcess Technology Consultants, Inc. 12 Gill Street, Suite 5450 Woburn, MA 01801