C&EN Webinar. Success in Highly Potent API Manufacturing

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C&EN Webinar Success in Highly Potent API Manufacturing Conrad Roten, Tobias Merz, Rainer Jossen/ Lonza Ltd/ 21 April 2011

Success in Highly Potent API Manufacturing Introduction- Lonza offering for highly potent APIs at Lonza Key factors for successful HPAPI manufacturing Case studies on Conclusion PAT in HPAPI projects Realization of a HPAPI in a manufacturing asset slide 2

Lonza Offers a Full Range of Manufacturing Services also for HPAPI Products Discovery Development Manufacture Distribution basic research disease discovery drug discovery drug development clinical trials production packaging marketing sales distribution R&D (multiple labs) lab supply in- market supply Kg labs (4-20L glass vessels) clinical supply launch supply Large scale HPAPI plant (10 m 3 ) Small-scale plants 8 reactors (160 to 250L) Launch plant multi-use trains (630 3 000L) slide 3

Lonza s Offering for HPAPI: 5 Different Technologies / Product Areas HPAPI capabilities available for 5 different technologies: Advanced Chemical Synthesis Antibody Drug Conjugates (ADC) Fermentation Peptides Continous Flow Technology Other specifics Specialized assets Dedicated teams slide 4

Success in Highly Potent API Manufacturing Introduction- Lonza offering for highly potent APIs at Lonza Key factors for successful HPAPI manufacturing Case studies on PAT in HPAPI projects Realization of a HPAPI in a manufacturing asset Conclusion slide 5

Highly Potent APIs Have Different Requirements Safety - EHS aspects - occupational health cgmp compliance - cross contamination Product Risk-based exposure control API OEL 10 µg/m 3 Highly Potent API (HPAPI) OEL 10 µg/m 3 steroids cytotoxics hormones highly potent compounds ß-lactams Protect employees and environment Protect patients slide 6

Key Factor Regulatory Strategy Highly Potent APIs: What Does Lonza Handle? Industry agrees - segregated equipment/plant necessary, for : Beta-Lactams Products containing live microorganism and ectoparasiticides Lonza does not currently have dedicated equipment for those categories.* Equipment requirements for Cytotoxics, Cytostatics not as clear 22% 11% Cytotoxic only Non cytotoxic only 28% Both, no segregation 39% Both, Specific units Lonza: conservative approach *figures 2009; Lonza internal data evaluation slide 7

Key Factor Toxicology and Occupational Health Expertise: OEBs Lonza Categories 1-6 and OEL (mg, μg & ng /m 3 ) 1 2 3 4 5 6 10 mg 1 mg 100 μg 10 μg 1 μg 100 ng 1 ng Manufacturing Standard trains R&D and QC Labs Standard hoods/ labs HPAPI trains Specialty hoods/ labs PCP Isolators 1 2 3 4 Safebridge Categories slide 8

Key factor - Risk Assessement and Containment strategy Collect basic information Physical properties of substances Hazardous properties of substances Processing steps and conditions / operational steps OEL-evaluation Primary Containment Exposure Potential Primary Containment Strategy Secondary Containment Displacement Potential Secondary Containment Strategy PPE is only safety net *figures 2009; Lonza internal data evaluation slide 9

Key Factor Lab and Manufacturing Assets: Setup and Containment for HPAPI Location Lonza Visp plant ist the Lonza HPAPI Centre of Excellence Centrally located in Europe Track record Since 1897 Key facts Specialist in hazardous chemistry Swiss quality and maintenance From kg to 10 s of tons Broad technology base incl. support plants and logistics Compliance track record Specialized HPAPI labs and plants Support plants View of Visp site slide 10

Key Factor People Know-how, Experience, Training, Performance Experts and experience Technical and regulatory expertise in all necessary areas Experience with HPAPI projects since years Working culture of people know where the substance is! HPAPI assets working team specialists in the labs and operations Training Verification control of system, people and process performance Passionate and loyal workforce slide 11

Analytical Labs Containment Dedicated lab teams Analytical development Handling of AHSK 4/5 compounds Containment features Access via pressure controlled locks Exhaust air via HEPA-filters Skan workstations a1-safetech weighing hood (stainless steel) Workbench Berner FlowSafe slide 12

Verification of Containment by Occupational Health Monitoring Where: labs, production, QC Procedure design / review / act Surrogate monitoring (naproxen, lactose) or actual compound Air monitoring Personnel Equipment Korridor Materiallock 5 Background Wipe tests Equipment Environment 6 8 12 7 Büro 21 sq m 6 13 2 4 8 Personnellock 9 7 Decontaminationlock 10 1 3 16 3 1 2 15 Technical Büro Installations 31 sq m Wipe Samples Air Samples Man-Monitoring slide 13

Results Surrogate Monitoring QC Lab Weighing 20 mg of surrogate (naproxen) Target: Air concentration < 40 ng/m 3 Sampling results in ng/m 3 1st Run 2nd Run 3rd Run Safetech weighing hood Operator < 7.4 < 6.1 < 14 Front, left < 7.4 < 6.1 < 15 Inside 42 103 685 Berner FlowSafe Operator < 12 < 8.4 < 8.7 Front, center < 12 < 8.5 < 8.7 Inside < 12 < 8.5 - slide 14

HAPI R&D Lab 6 Lab Suites 1 kg lab, 20 Liter Scale Floor and pressure zoning plan of HAPI lab E38 Above: Safetech hoods Below: Hallway HAPI lab E38 slide 15

LCRDH HAPI Lab E38 Labs Containment, Characteristics Containment adaptable to process, combination of Glove boxes Safetech hoods Laminar flow standard lab work benches flexible, staggered approach slide 16

Results Air-monitoring Isolator vs Safetech Sampling results in ng/m 3 Safety hood Isolator Operator <2.36 <2.51 Front outside <2.72 <2.62 Inside 7012 Under door of lock <2.60 Background lab <0.536 <1.70 Personnel lock <0.516 <1.74 Above: Isolator Below: Safetech hood slide 17

Difference Between Different Operators Sampling results in ng/m 3 Operator < 6.24 < 6.60 Front outside < 6.19 < 6.60 Inside 14392 23583 Work opening 62 38 Background lab < 4.97 < 2.58 Air sampling setup Bench chemist operating in safetech hood slide 18

Manufacturing Assets Small Molecules HAPI lab 1080 / 20 lt SSP plant / 160-2500 lt Launch plant / 630-2500 lt HAPI plant / 10 m3 slide 19

Key Factor Development Process Tailored to Meet Customer and Product Needs Familiarization Development Qualification Validation / Production Team Work Expertise Innovation and Creativity Voice of Customer Customer Expectations Target Product Profile Critical Quality Attributes Risk Assessment Design Space Control Strategy Continuous Improvement Pre-Clinical Phase Phase I Phase II Phase III Launch/ Production slide 20

Success in Highly Potent API Manufacturing Introduction- Lonza offering for highly potent APIs at Lonza Key factors for successful HPAPI manufacturing Case studies on PAT in HPAPI projects Realization of a HPAPI in a manufacturing asset Conclusion slide 21

Analytical PAT Solutions for HAPI Manufacturing T.Merz / Lonza Ltd/ 21 April 2011

Process understanding Strategy Phase 1 Phase 2 Feasibility study Know how collection Scale-up Process monitoring Analytical afford Phase 3 Process control slide 23

PAT Implementation Identification of the key process parameters Identification of the most appropriate PAT tools Lab or production scale (analyzer/ sensor/ probe/ software ) Automation, sampling, data storage Method development in R&D phase Process data collection Statistical evaluation Definition of the design space Support process development and validation Method transfer in production Statistical evaluation slide 24

Why PAT Tools in HAPI Manufacturing No sampling necessary Non-destructive analysis Real-time information In-situ information Chemical and physical information More information (conc, polymorph) no contamination no waste no sample alterations less experimentation Preferred spectroscopic techniques slide 25

PAT Toolbox Process Analyzers UV/VIS NIR MIR Raman FBRM Selectivity + ++ +++ +++ - Sensitivity +++ ++ +++ + +++ Sampling +++ +++ + + +++ Aqueous solution +++ + + +++ +++ Applicability +++ ++ + + + Process capable Light guide/ glas +++ +++ + +++ ++ +++ +++ + +++ ++ Signal Absorption Absorption Absorption Intensity Chord length Samples Techniques solid, liquid, gaseous Transmission Reflexion ATR solid, liquid, Transmission Reflexion ATR solid, liquid, gaseous ATR Transmission solid, liquid, gaseous Reflexion liquid Reflexion rel. costs 1 3-5 6-10 8-12 8-10 slide 26

PAT Tools at Lonza Important for HPAPI Process Development Spectroscopy Raman UV/VIS NIR Solid phase Lasentec Turbidity probes XRD Gas phase Chemscan Hydrogenation units Online MS Calorimetry LabMax, Flexylab, Mettler RC, CRC CRC equipment, in collaboration with Prof. Dr. K. Hungerbühler ETH Zürich http://www.sust-chem.ethz.ch/research/groups/reactioncalorimetry slide 27

FBRM + PVM 1 2 3 4 5 6 FBRM-probe PVM-probe 3 5 6 slide 28

Raman Spectroscopic Tools Quantification of a HAPI through glass vials Calibration Prediction slide 29

Raman Spectroscopic Tools Crystallization: trend lines of the concentrations (solid and liquid with semi-quantitative modeling) slide 30

Raman Spectroscopic Tools Trending of precipitation in production Multi curve resolution with 3 components Educts in Ethanol / Water Precipitant seeding Product in Ethanol / Water slide 31

Raman Spectroscopic Tools Polymorph investigation with multi-curve resolution (MCR) Original Calculated slide 32

Raman Spectroscopic Tools Prediction in production Seeding form I Seeding with form II 6:20 h slide 33

Implementation of a HPAPI Product in Production R. Jossen / Lonza Ltd/ 21 April 2011

HPAPI Assets and Resources Launch Plant Train 102 - OEL 0.1-1 µg/m3 Consists of Reactor: chemical reactions Reactor: mainly for work up Design for volume flexibility Isolation equipment (filter dryer) No open solid handling, repackaging in glove box All 4 operation floors with enclosed working areas and air locks Operators in PEDI suits Several buffer tanks Train 102 Unloading product slide 35

HPAPI Assets Launch Plant Train 102 OEL 0.1 µg/m3 Project Implementation Task: Production of peptide 100 ng/m3 OEL ph-sonde Preparation: Risk assessement focus solid or open handling! Detailed SOPs for focus activities Establishment of the production concept Accident containment plan Defined interaction with disposal plants, logistics, analytics, production, QA Cleaning concept (risk assessment) slide 36

HPAPI Assets Launch Plant Train 102 OEL 0.1 µg/m3 Project Implementation Material and personnel flow: Definition and risk assessment Venting: CIP, filter Venting line setup for cleaning in place w/o dead volume, safety filters to prevent contamination of vent lines slide 37

HPAPI Assets Launch Plant Train 102 OEL 0.1 µg/m3 Project Implementation Completion of setup additional risk assessment and test Detailed risk assessment at the plant side Simulation run utilizing lactose checking for leckage Basic spec of test: same conc. as API, process flow same as for API, representative sampling positions (24) Sampling position Result Split butterfly valve 4.St. adding raw materials 17µg Split butterfly valve 4.St. connector 1212 µg Flange 1 transfer 102.4.R1/3.R1 0.07 µg Transfer to Nutro 102 0.01 µg Split butterfly valve inside containment clean room; procedure for decontamination established and tested slide 38

HPAPI Assets Launch Plant Train 102 OEL 0.1 µg/m3 Project Implementation Production and cleaning: Verification performance by occupational health Air monitoring and swabbing while working Results officially published for operators each day Overview results supporting working concept Dry coupling drum above spec; expected Decontamination inside clean room Measure working proven by verification samples Sampling position Manifold RW 102.3.R1 Next to nutro 102 Clean room 4.St. Utility room 1.St. Result < 2.12 ng/m3 <1.45 ng/m3 <1.77 ng/m3 <1.41 ng/m3 Surface drum <0.025ng/cm 2 Floor next to nutro 102 <0.025ng/cm 2 Air lock 1.St. <0.025ng/cm 2 Clean room 1.St. <0.025ng/cm 2 Dry coupling drum (Vaterstück) Dry coupling drum (Mutterstück) 534 ng 45000ng slide 39

And There is Disposable Containment Need for a Centrifuge in a HPAPI Process Problem: Filtration high LOD, potential wrong polymorph; OEL 1 µg/m3 Target: Use of a centrifuge not typical HPAPI equipment Solution: Custom made disposable containment Preparation: Layout designed by operators, risk assessment, interaction with supplier Performance verification: Occupational health monitoring inside containment All results below LOD of 0.015 µg/m3 Next step: Optimization of layout based on campaign experience slide 40

Summary Success in HPAPI Manufacturing PEOPLE slide 41

Thank You