RapidFire High-throughput MS technology. Enhancing Drug Discovery Turning Mass Specs into Plate Readers



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RapidFire High-throughput MS technology Enhancing Drug Discovery Turning Mass Specs into Plate Readers 1

Drug Discovery with RapidFire Target Selection High-throughput Target Screening (HTS) Lead Optimization Preclinical Testing Clinical Trials Genomics Proteomics Metabolomics Lipidomics Animal models Knockouts Label-free HTS using native substrate Secondary screen Selectivity screen Med Chem / SAR ADMET PK/PD Toxicology PK/PD TDM DDI Met Stab Permeability Protein binding Identify the best hits: Eliminate HTS compromises Select the best leads: Data-driven lead selection Eliminate compromises when designing experiments 2

RapidFire Mass Spectrometry Fast autosampler & SPE system Replaces LC in LC/MS Integrated, automated, micro-scale solid-phase extraction Integrates with standard ESI MS instruments cycle time: 5 10s/sample Compatible with biological matrices Microsomal preparations Cell culture supernatants Tissue extracts Plasma

SPE-MS/MS Analysis Mass spec Aqueous wash Organic reverse elution waste

RapidFire Data 96-well plate analyzed in <12 minutes IS data Sample data 5

RapidFire Cycle Sip/Equilibrate Sip Elute Load Standard 96 or 384-well plates Sample interface (sipper) Washed with aqueous and organic solvents Optical sip sensor o wells missed (even if half full) 6

RapidFire Cycle Load/Wash Sip Elute Load 10-µL injection loop onto 4-µL RapidFire cartridge o offline sample preparation SPE purification by proprietary cartridge (variety of packing beds) of quenched assays Cartridge changer (6 slots) Automatic or programmed switch 24-hour unattended runs Facilitates assay development Packing Material C4, C8, C18 Phenyl, Cyano HILIC Client specified 1.4 cm 7

RapidFire Cycle Elute/Clean Sip Elute Load Compatible with any ESI or APCI flow injection analysis MS MS at 5-10 seconds/sample Micro-scale fluidic path Baseline resolution PEEK minimizes carryover 8

RapidFire - Automation & Efficiency Acquisition Software Features Sequence-based protocol injections from plates in any well order Multiple MS methods per plate Enter all pre-developed parameters at once for full stack of plates (7000 samples/run) Barcode scanner 24-hour unattended operation Active monitoring Error flag upon suspension 9

RapidFire Integrator - Data Analysis & Output Proprietary, intuitive data analysis software Outputs results as text files compatible with standard databases 10

RapidFire Integrator Software Internal Standard (d4-1 OH-midazolam) 1 2 3 4 5 6 7 8 9 10 11 12 A 13515322 14269894 14332623 14471745 15116982 15301635 14690330 14579803 14465625 15011230 15237832 15546360 B 17434008 16604680 16948062 16097807 16634851 16445362 16467090 16236566 15934448 16043856 16149485 16732055 C 18620363 17813741 17284582 16651378 16716736 17219301 17699784 17024495 16106934 16662407 17022034 17631943 D 18853237 18639570 17545882 16707887 16859350 17082515 17135769 16917660 16866547 16762991 16662805 16932106 E 19095034 19155820 18632830 17190636 16915409 17698485 18031132 17904120 17498786 16646769 17301091 18276269 F 19501938 18858411 17530737 17476044 17285001 17765209 18191777 17539311 18035244 17721958 17420392 18124271 G 19617689 19641455 20695544 18748491 17433257 17236209 18041601 20432135 18674406 18662003 17706549 18696053 H 19645157 19177960 20062090 19103985 18990975 17804109 18038389 19058414 19193675 19711093 18461084 17856780 Product (1 OH-midazolam) 1 2 3 4 5 6 7 8 9 10 11 12 A 81217 100602 88629 94434 113207 115549 83772 87924 97878 244550 235432 262948 B 152871 118289 139825 182411 176312 168628 104282 104738 108970 661471 664829 633719 C 246216 215908 197602 291126 302567 285434 149992 150230 142836 1236854 1168309 1328238 D 544895 555992 471635 426800 398887 403895 227402 206803 200213 1849887 1893075 2030754 E 1541495 1436062 1357041 507776 540945 564612 453009 332389 361539 2609866 2769033 3056845 F 2261622 2187441 1995732 715854 714291 764022 583844 527665 515286 3324764 3182154 3653594 G 2834427 2233412 2213744 932589 818246 821289 694814 514781 630866 4214068 3234244 3427977 H 2323314 2461589 2282049 910427 836034 920960 666413 632365 550910 3351768 3794636 3518838 Turning a mass spectrometer into a plate reader

RapidFire applications for in vitro ADME (absorption-distribution-metabolism-excretion) 12

RapidFire for in vitro ADME assays Strong results correlation with traditional technologies CYP inhibition (drug-drug interaction) Reversible and Time-dependent Metabolic stability HLM or S9 Permeability cell lines (Caco-2) and/or PAMPA P-glycoprotein inhibition Plasma protein binding HSA, brain, microsomal binding 13

IC 50 (um) by Traditional RF-MS Summary of Analytical Methods Comparison 1000 R 2 = 0.986 Direct CYP Inhibition: Comparable results obtained >20-fold faster analysis 8 different isoforms/probe pairs IC50 (um) by RapidFire @ BioTrove 100 10 1 0.1 0.01 CYP1A2 CYP2B6 CYP2C8 CYP2C9 CYP2C19 CYP2D6 CYP3A4 Caco-2 Permeability: Comparable results obtained nearly 10x faster with RF-MS LC-MS RF-MS Analysis Time 20 hours 2.5 hours 0.001 0.001 0.01 0.1 1 10 100 1000 IC IC 50 (um) 50 (um) by by Traditional LCMS LC-MS @BD Gentest Linearity R > 0.99 R > 0.98 LLOQ 5 nm 5 nm Carry-over < 0.3% < 0.3% Generic method applicability Generic LC method Generic SPE method 14

RapidFire 360 High Resolution System Eliminates bottlenecks in high-throughput MS The most efficient analysis available o MRM method development Fully integrated sample preparation Walk-away automation

RF360 Removes the MRM Bottleneck Traditional MS/MS RF360 Run Assay Run Assay 4-10 min/ analyte 1-3/sample (15-45 min/ analyte) QqQ method development Data Acquisition Generic Methods & Full MS Scan Data Acquisition Data Analysis 6-10 sec/sample (1-2 min/analyte) Data Analysis

RF 360 Data Microsomal Stability Assay with Midazolam TIC Parent (Midazolam) Metabolite (Hydroxymidazolam) 0 min 5 min 10 min 20 min 30 min 60 min Extraction and peak integration of analytes from TIC is fully automated Internal Standard (Bupivicaine) 17

Metabolite Analysis Data 18

High-throughput TOF Data Analysis monoxidation dioxidation demethylation deethylation reduction desatuation glucuronadation sulphation acetylation Import mass table or formulas Select metabolites of interest Obtain fully-integrated peak areas 19

Prod/IS, AUC Metabolite Analysis Results - Midazolam Midazolam 0.9 0.8 0.7 0.6 0.5 0.4 0.3 0.2 0.1 0 0 20 40 60 Time, min Midazolam Oxidation Cl F Cl F HO

Prod/IS, AUC Prod/IS, AUC Metabolite Analysis Results - Chloropromazine 1.2 1 0.8 0.6 0.4 0.2 0 Chloropromazine 0 10 20 30 40 50 60 0.05 0.04 0.03 0.02 0.01 0 Chloropromazine Metabolites 0 20 40 60 oxidation demethylation 2 demethylations Time, min Time, min H H 2 Cl Cl Cl S S S Cl OH S

Reducing Bottlenecks in ADME Sample Analysis using Solid Phase Extraction with a Quadrupole Time-of-Flight Mass Spectrometer Traditional LC-MS Workflow 38.5 hr 0.5 hr 12 hr 24 hr 2 hr Samples Received Batch Creation Analyte Tuning RapidFire 300 MS QQQ Work Flow Samples Received 16.5 hr 0.5 hr 12 hr 2 hr 2 hr Sample Analysis Data Delivery Data Analysis Data Delivery Adapted from oral presentation by Panos Hatsis, ovartis, 58 th ASMS Conference on Mass Spectrometry and Allied Topics, May 23-27, 2010 RapidFire 360 TOF Work Flow Samples Received 4.5 hr Data Delivery

Resolves Bottlenecks Improved Data Quality: Throughput allows for Multiple concentrations: run full IC 50 s Multiple time points Avoids surrogate substrates, indirect and coupled assays Maximizes Productivity: Fastest data turnaround 10x data generated per FTE compared to LC-MS Rapid assay development in HTS faster time to answer Strong Correlation with Traditional Technologies: LC-MS/MS: CYP450 inhibition, metabolic stability, etc Optical-probe or radioactivity-based detectors

Advantages of Lower Operational Costs: Solvents: 6 s @3.0 ml/min = 0.25 ml (~2 /sample) Cartridge: $200/cartridge or ~ 3000 samples/cartridge (~7 /sample) Minimal Reagent & Disposal Costs in HTS: Only native substrate & enzyme are required o antibodies, luminescent or radioactive reagents, kits Fits Existing Workflows Designed to operate similar to a plate reader