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A Tech Transfer Case Study From a CDMO Sigma S. Mostafa Director, Process Development KBI Biopharma 10-03-2015 Overview • Introduction – CDMO – KBI • Tech Transfer in a CDMO – Unique constrains – KBI’s work paradigm • Case Study What is a CDMO? • Contract Development & Manufacturing Organization • Responsible for clinical trial material (CTM) development and manufacturing – Process development & manufacturing of Bulk Drug Substance (BDS) – Formulation and manufacturing of dosage forms, i.e. Drug Product (DP) – Analytical support for BDS and DP • A large portion of CTM development & manufacturing expenditure is outsourced to CDMOs 3 | Confidential DURHAM SITE a customer and science-focused contract development & manufacturing organization KBI Locations KBI Durham • Analytical and Formulation Dev. • Cell Line Development • Cell Culture cGMP Manufacturing • Microbial cGMP Manufacturing KBI Boulder • Microbial Process Development • Microbial cGMP Manufacturing • Analytical Development KBI RTP • Cell Culture Process Development • Downstream Process Development • Microbial Process Development • Analytical Development 5 | Confidential About KBI – Our Services Cell line Development 6 | Process Development Analytical Method Development Preformulation and Formulation Development Confidential API Manufacturing -Microbial -Mammalian Release testing and stability studies Types of Projects in Process Development Process Transfer Full Process Development Material Supply Process Characterization Process Optimization Tech Transfer Aspects in a CDMO • Large number of tech transfers per year – KBI has >10 tech transfers per year from PD to manufacturing • Each cell type, molecule, and process are different; opportunities to leverage platform is limited • Timeline for tech transfer is short and overlaps with development work – PD scale-up run and first manufacturing run are 1 -2 months apart – Short timeframe necessitates staggered tech transfer approach Process Development Work Flow Process Development Tech Transfer to Manufacturing - Kick-off meeting Scale-up Run - Shake flask study - Facility Fit - ambr, 3L, & 15L bioreactor study - Risk Assessment - Harvest study - PFD - BOM - Process Description - 200L Disposable Reactor - MCB vial - Representative seed train - Final Process Tools of Tech Transfer Development Report, Demo Report, Process Description Process Flow Diagrams Raw Data Bill of Material Process Control Trends Risk Assessment Technology Transfer – Key Contributors • • People: experienced & dedicated Process: well characterized for scale up & Mfg Facility cGMP compliant Facility fit Program management: System for information transfer 11 | 30+ with advanced scientific and technical degrees in Proc Dev 30+ manufacturing staff with significant operations experience with small and large biotech/biopharma Confidential KBI’s Business Process for Tech Transfer PD Develop upstream and downstream Perform Confirmation and Demo runs Prepare process overview, facility fit, process description, solution and buffer recipes, sampling plan w/AD Review batch records, person in plant for key steps for 1st run AFS Method Development Method Qualifications Formulation Development Sampling and Testing plans, Specifications IP and release Stability MFG Facility Fit Draft batch records and solution records Finalize BOM, order raw materials, ensure solutions and buffers specifications Execute Eng and GMP runs, close deviations Campaign Summary QA Client 12 | Review and approve MBRs, solutions records, sampling plans Review process overview, facility fit and process descriptions Confidential Deviation closure and Batch release Approve process descriptions, BRs, BOM, solutions records, person in plant as decided Single use disposable technologies Media and Feed preparation utilizing disposable mixing, filtration and storage systems Aseptic connection MCB or WCB vial Disposable shake flasks or disposable spinner flasks Hold vessel (bag) Disposable expansion reactor Disposable fluid path purification system Disposable mixing tank Disposable seed bioreactor 0,2 µm filter Hold vessel (bag) Disposable production bioreactor Disposable fluid path purification system Disposable fluid path centrifuge Disposable mixing tank 0,2 µm filter Hold vessel (bag) Hold vessels (Bags) 0,2 µm filter Disposable depth filtration system Virus filter Hold vessel (bag) Retentate BPC Permeate BPC BPC BPC PD Hold vessel (bag) Disposable fluid path UF/DF system 0,2 µm filter Hold vessel (bag) 0,2 µm filter Sterile bulk fill and sampling bags Buffer preparation utilizing disposable mixing, filtration and storage systems Shukla, A., Mostafa, S., Wilson, M., Lange, D. Vertical Integration of Disposables in Biopharmaceutical Drug Substance Manufacturing, Bioprocess International, 10(6), 34-47, 2012. KBI’s Cell Culture Platform Process Shake Flasks ambrTM Bioreactors Medium and Supplement Screening Process Parameter Screening 200L Bioreactor 3L Bioreactors 15L Bioreactors Process Optimization and Robustness Demonstration Run 14 | 3L Bioreactors Confidential Mixing Characteristics in Bioreactors Data from mixing studies used to set agitation rate, aeration strategy, process control strategy Comparability Across Scale – 3L, 15L, 200L, and 2000L Viable Cell Density • VCD data matches across scale Gottschalk, U., Shukla, A. Single-use disposable technologies for biopharmaceutical manufacturing, Trends in Biotechnology, 31(3), 147-154, 2013. Shukla, A., Mostafa, S., Wilson, M., Lange, D. Vertical Integration of Disposables in Biopharmaceutical Drug Substance Manufacturing, Bioprocess International, 10(6), 3447, 2012. Comparability Across Scale – 3L, 15L, 200L, and 2000L Titer • Titer data matches across scale Scale-up studies Cell Growth • Titers Product Quality Attributes Comparison across scales for the production of a recombinant glycoprotein in a recombinant CHO cell line – The process decisions and results from ambrTM were reproducible to other scales Rameez, S., Mostafa, S., Miller, C., Shukla, A. High-throughput miniaturized bioreactors for cell culture process development – reproducibility, scalability and control, Biotechnology Progress, 30(3), 718-727, 2014. Case Study – Project Start-up • Expedited Process Development for a novel mAb • Data from client on shake flask batch study • CHO DG44 cell line from client (prepared by a third party) • Client expressed need for 200L scale material delivery before process development started Case Study – Project Scope of Work • Contract Signed in early September • Face to Face Kick-off meeting in mid September • Tech Transfer into KBI – A shake flask study and a • • • • Material Supply Run (200 L Scale) Oct 2013 Process Optimization (Ambr Study) Nov 2013 Process Optimization (3 L bioreactor-scale) Dec 2013 Process Confirmation (15 L bioreactor-scale) Jan 2014 Demo Run (200 L-scale) Feb 2014 Material Supply Run (15 L Scale) Tech Transfer • 3 x 3L bioreactor study completed by mid October A 200L Disposable Bioreactor Supply Run completed by mid October Studies at ambr, 3L and 15L bioreactor scaled conducted November through January PD Demonstration Run at 200L Disposable Bioreactor done in Feb Vial thaw for cGMP run in KBI manufacturing facility in March Start of project to manufacturing vial thaw in 7 months for a full development mAb project. Sep 2013 Tech Transfer to KBI (Shake flask and 3L bioreactor scale) cGMP Run (2000 L-scale) Mar 2014 Case Study – Tech Transfer into KBI • Client transferred shake flask batch process • In Tech Transfer Run, client’s process was carried out in shake flask and 3L reactors • In parallel one feed was tested in preparation for 200L material supply run • Cell growth improved in the fed-batch culture Case Study – Tech Transfer into KBI • At 3L bioreactor scale productivity with the KBI fed-batch process was 2.2x higher than the original batch process Case Study – 200L Material Supply Run • An initial 200L Material Supply Run was carried out following the 3L Tech Transfer Run • This 200L run was done prior to initiation of Process development • Initial cell growth and peak cell density in the 200L run was slightly higher than the 3L scale • Viability drop was faster in the 200L scale Compared to the 3L fed-batch culture Case Study – 200L Material Supply Run • Titer in the 200L scale did not match the 3L fed-batch data • A significant amount of base addition occurred in the run • Maintaining pH within deadband (0.05 pH) Was difficult Case Study – 200L Material Supply Run • Glucose uptake was somewhat higher in the 200L scale compared to the 3L fed-batch culture • Final lactate level was around 10 g/L in the 200L scale (compared to 7 g/L in 3L fed-batch reactor) Case Study – Shake Flask & ambr Studies re CS1 - Control (pH 7.0) CS2 - Control (pH 7.0) Cell Boost 6 - pH 6.90 Feed C - pH 6.90 ambr Lactate Comparison 2.00 Lactate (g/L) 0.47 Titer Harvest Titer (Day 12 or Day 14) 2.50 Shake flasks 0.56 0.38 1.50 1.00 0.29 0.50 0.00 0.2 4 Feed A+B Feed (A+B) Feed C Cell Boost 6 5 6 7 8 9 10 11 12 Time (Days) • Project required expedited process development A:Feed • Shake flask study focused on feed and supplement evaluation • ambr study focused on pH set-point and feed impact on lactate & titer 13 14 Case Study – 3L Bioreactor Study • • • • A fractional factorial DOE was carried out in the 3L scale Multiple feeds, temperature scheme, and pH set point were tried pH dead band was expanded Feeds with lower lactate level and higher productivity were identified cGMP Manufacturing (2000 L-scale) Comparison w/ Small-scale 16 120.0 100.0 12 Cell Viability (%) VCD (1 x 10^6 cells/mL) 14 10 8 6 2000 L GMP Run #1 2000 L GMP Run #2 200 L-scale PD Demo Run 3 L-scale Final Process 4 2 80.0 60.0 40.0 2000 L GMP Run #1 2000 L GMP Run #2 200 L-scale PD Demo Run 3 L-scale Final Process 20.0 0 0.0 0 2 4 6 8 Time (Days) • 10 12 14 16 0 2 4 6 8 10 12 14 Time (Days) Cell growth and viability compared well among 3L, 200L, and 2000L scales Vesper - Confidential 16 cGMP Manufacturing (2000 L-scale) Comparison w/ Small-scale • Lactate profile was much improved compared to the 200L material supply run • Maximum lactate level in the 2000L was 4 g/L, less than half of the level observed in the material supply run Vesper - Confidential cGMP Manufacturing (2000 L-scale) Comparison w/ Small-scale • Titer was comparable across 3L, 200L, and 2000L scales • 4.5X increase in titer compared to the 200L material supply run. Vesper - Confidential Conclusions • Understanding of cell line characteristics and process parameter impact on cell line is imperative for successful tech transfer • Use of high throughput systems such as ambr micro-bioreactors provide significant advantage during expedited process development • For an expedited manufacturing plan, a phased approach to tech transfer is needed; identifying and ordering the long lead items and determining facility fit are often the most rate limiting activities • Testing of scalability early in process development allows identification of cell line specific scalability challenges; therefore, using material supply runs as scale-up tests is advisable. Acknowledgements Process Development Analytical - - Niket Bubna Lynwel Cunanan Brian Baker Ronnie Nichols Michael Pollock James Smedley Executive Management Tech Transfer - - Sam Pallerla - Manufacturing - Les Smith Michael Huerta Joaquin Lopez Abhinav Shukla Prathima Acharya Joe McMahon