QIAGEN Research & Development. Pipeline 2009/2010

Transcription

1 QIAGEN Research & Development Pipeline 2009/2010 QIAGEN Analyst and Investor Day 2010 Dr. Joachim Schorr Senior Vice President Global Research & Development New York, February 11th, 2010

2 Agenda QIAGEN - R&D Centers of Excellence - Development Cycles - R&D Budget Allocation New Product Launches & Regulatory Pipelines Prevention Profiling Personalized Healthcare Point of Need Regulatory Pipelines -2-

3 Global R&D Organization Personalized Healthcare CDx Program Management USA UK Germany Switzerland Basic R&D Application Development Asia MDx Assay Development MDx Prevention Systems Integration sirna Application Lab Automated System Development MDx Assay Development Pathway Analysis Systems Validation Sample Technologies 690 Scientists in Research & Development Enabling Technologies for Sample and Assay Technologies -3-

4 QIAGEN Product Development Focus & Execution PT PRC PRC PRC PRC/GRM PRC Phase 0 Phase 1 Phase 2 Phase 3 Phase 4 Launch QIAGEN s Global R&D: Running Projects: 150+ Status: 60 in phase 3 or later Development time: Approx. 12 months (LifeSciences ~ 9 months, MDx incl. approval 9-24 months) Employees: 690+ researchers allocated according priorities within portfolios Investment: Approx. 12% of revenues per year Return on investment: Approx. 5% of revenue growth from products younger 12 months IP: Expansion of IP position >2,100 patents (757 issued, 799 pending, 550+ licensed) -4-

5 R&D Budget Allocation Expenses per Technology Center MDx Sample & Assay Technology Consumables ~61% ~61% ~20% ~20% Sample & Assay Technology Instruments & SW For all customer classes ~19% ~11% ~8% Life Sciences, Pharma, Applied Testing Sample & Assay Technology Consumables Note: MDx Assay & Samples including clinical trials (US$ 18.1 M) Life Science including QIAGEN Manchester -5-

6 Agenda QIAGEN - R&D Centers of Excellence - Development Cycles - R&D Budget Allocation New Product Launches & Regulatory Pipelines Prevention Profiling Personalized Healthcare Point of Need Regulatory Pipelines -6-

7 Technology Roadmap 2010: Sample and Assay Technologies Structure Based on Identical Workflows in All Segments Prevention HBV HCV CMV EBV RSV GBS Influenza QIAsymphony Profiling Personalized Healthcare Point of Need Automated Systems Collection Stabilization & Storage Disruption Enrichment Assay Technologies Purification Amplification / Modification Sample Technologies Content Detection Bio-Informatics Integrated Microsystems -7-

8 From Components to Content Strategy for R&D Today Content engine Broad portfolio assays, incl. regulated Content engine Several novel markers in pipeline Integrated solutions Rotor-Gene Q Automation excellence QIAsymphony QIAensemble PON Devices BioRobot 8000 BioRobot MDx QIAcube QIAxcel EZ1 QIAgility PyroMark Rotor Gene Q Biology/Chemistry excellence -8-

9 The QIAGEN Content Engine MDx Roadmap Examples Near-term Examples Mid-term Examples Long-term Prevention QIAensemble Evolution care HPV (3rd world) HPV, CT/GC, Trich, GBS, Vaginosis, etc. Screening and Progression markers Screening for: cancers incl. colon, ovarian, thyroid etc. Profiling ResPlex II artus assays for QIAsymphony RGQ GastroPlex, HHV-6, Adenovirus, JC, HSV,Candida, Aspergillus Autoimmune diseases, CNS, cardiovascular diseases, women s health, etc. Personalized Healthcare KRAS, BRAF A/C-Raf, MEK-1/2, p53, EGFR, BCR-ABL, NRAS, I3-Kinase, N/H-RasAKT, BCR, PTEN et. al. CDx for cancer therapeutics Autoimmune diseases, CNS, cardiovascular diseases, others Point of Need MRSA, cdiff, VRE Pseudomonas, Acinetobacter, other bacteria Meningitis Other Blood screening (China) Blood screening emerging countries -9-

10 The 4 Ps in MDx Prevention Profiling HBV HCV CMV EBV RSV GBS Influenza QIAsymphony PHC Point of Need -10-

11 QIAensemble Ultra High to Mid Throughput Screening Platform Platform Technology Assays Samples Ultra High/High (up to 2000/shift) Throughput Instrument Floor model Configuration screening: digene HPV ehc Test genotyping: digene HPV ehc 16 18/45 Test LBC Vials Co-collected DCM Low/Medium (up to 600/shift) Throughput Instrument Benchtop Configuration screening: digene HPV ehc Test genotyping: digene HPV ehc 16 18/45 Test LBC Vials Co-collected DCM -11-

12 QIAensemble plus Adds Module for Amplified Assays Platform Technology Assays Samples Ultra High/High (up to 5000 CT/GC/shift) Non-amplified: Throughput Instrument Floor model Configuration HPV, HPV GT Amplified: LBC Vials Co-collected DCM CT/GC Assay Ultra High/High (up to 3000 CT/GC/shift) Throughput Instrument GBS Trichomonas Vaginosis and many more Urine Vials Floor model Configuration -12-

13 CT/GC Assay Workflow (Closed Tube Format) HC thda Fluorescent Endpoint Detection QIAensemble SP+ QIAensemble Hybrid Capture Sample Preparation Amplification & Fluorescent Endpoint Detection Clinical sample ~ 20 short RNA probes Lysis & denaturation Probe hybridization & capture CT and/or NG DNA Beads with Hybrid hybrid Capture capture antibody DNA-RNA hybrid Magnetic separation of beads & wash Magnet Amplification in the presence of hybrid capture sample prep beads -13-

14 QIAGEN CT/GC Assay Project Goals Chlamydia trachomatis/neisseria gonorrhoeae (CT/GC) Assay Reaction: Isothermal Helicase-Dependent Amplification (thda) Detection: End-Point Fluorescent. Assay performance specifications Analytical sensitivity ~1 IFU of CT/GC per assay 10 copies of target per amplification Analytical specificity: No cross-reactivity with non-pathogens Input material: Urine, LBC and swabs (more to come) Assay time should allow various throughputs (design goal: 1500/shift, actual 3000/shift). Assay automation Same platform as for HPV screening Complete automation for all throughput labs De-capping capability of all sample vials -14-

15 CT/GC Assay Performance Sensitivity of 10 Copies in a Multiplex Assay Format *Research Gaithersburg End-Point Fluorescent Detection CT1/CT2/NG/IC CT/GC thda assay multiplex assay performance IC, 250 copies NG CT IC, 250 copies NG CT Real-Time Detection S/N NTC 10 copies CT/NG 100 copies CT/NG 1000 copies CT/NG Detection of Mixture of CT and NG Targets and IC in One Well -15-

16 The 4 Ps in MDx Prevention Profiling HBV HCV CMV EBV RSV GBS Influenza QIAsymphony PHC Point of Need -16-

17 Launches and Developments 2009/ 2010 QIAsymphony System Family QIAsymphony RG-Q family QIAsymphony SP QIAsymphony RG-Q QIAsymphony plus Launch in Q1/2010 artus HIV artus HBV artus HCV artus CMV artus EBV Launch in Q3/2010 artus VZV artus BKV artus HSV Currently in assay development artus HSV artus CT/GC Kras; EGFR; PIK3; MEK; BRAF; JAK2; EGFRvIII artus Influenza Planned near term (RUO, CE, 510K) artus HHV-6 artus JC (human polyomavirus) artus Adenovirus artus Aspergillus artus GBS -17-

18 The 4 Ps in MDx Prevention Profiling HBV HCV CMV EBV RSV GBS Influenza QIAsymphony PHC Point of Need -18-

19 KRAS Status Major Impact on Anti-EGFR Drug Efficacy Ras-signal transductions pathway Epidermal growth factor EGF EGFR Cell growth via EGF-Ras-Raf signal transduction Controlled normal cell growth Uncontrolled oncogenic cell growth = uncontrolled signal amplification P KRAS KRAS raf active EGFR-Inhibitors against EGF receptor block cell growth Antibodies Erbitux (Imclone), Vectibix (Amgen) About 140 EGF inhibitors in clinical trials Strategies against colon, lung, head & neck etc. MEK ERK Oncogenic Ras Mutations in 4 critical positions Found in up to 30% of all human tumors Send permanent cell growth signal Down-stream of EGF-Receptor Lead to drug resistance of EGFR-Inhibitors Cell growth Patient stratification increase efficacy of Anti-EGF drugs Erbitux and Vectibix only for KRAS Wildtype 30-40% of patients with Oncogenic KRAS Efficacy significantly increased with KRAS tests (1) Epidermal Growth Factor Receptor -19-

20 Personalized Medicine Becomes Reality Molecular Biomarker test Therapy Indication BCR-ABL Gleevec Chronic myeloid leukemia c-kit Gleevec Gastrointestinal stromal tumor (GIST) HER-2/neu receptor Herceptin Breast cancer BRCA 1, Breast/ ovarian cancer Pharmaceutical and surgical prevention options AlloMap gene profile Immunosuppressiva Monitors immune response to heart transplant Familion 5-gene profile Pharmaceutical prevention Inherited cardiac channelopathies: Prevention & drug selection p16/cdkn2a Melanoma: Preventive treatment Pharmaceutical and surgical prevention options TruGene -HIV 1 Genotyping Kit Anti-retroviral drugs Therapy selection based on resistant HIV mutations Oncotype DX 21-gene assay Cancer treatment regimens Quantifies the likelihood of breast cancer UGT1A1 Camptosar Drug side effects in colon cancer treatment Amplichip CYP2D6/CYP2C19 Drugs metabolized by P450 Treatment dose for drugs that are metabolized by P450 Estrogen receptor Tamoxifen Relevance of tamoxifen citrate in breast cancer therapy TPMT Purinethol Dose adjustment in acute lymphoblastic leukemia treatment KRAS (BRAF) Vectibix Exclusion of KRAS mutants increase efficacy Source: Personalized Medicine Coalition,

21 Covering the Entire Pharma Development Process From Discovery through Development to Companion Diagnostics 5 Years 1.5 Years 6 Years 2 Years 2 Years 10 Years + Discovery Preclinical Clinical Trials FDA Launch Approval Marker Marker Marker Marker Marker Marker Marker Marker Marker Marker Marker Marker Marker Approval Diagnostic Explore pathways and molecules Validate Biomarkers - Possible Companion Dx? File for IVD Clearance Use IVD for More data Routine Use -21-

22 Technology Toolbox and Assays Roadmap Personalized Healthcare Biomarker First Indication (Cancer) Partnered CE FDA KRAS Colon Lung Yes BRAF Colon Melanoma No 2010/ EGFR Lung Yes H EGFR viii Glioblastoma Yes H PCR PI3K Breast At proposal NRAS Melanoma In discussion ABL Chronic myelogenous leukemia (CML) In discussion

23 The 4 Ps in MDx Prevention Profiling HBV HCV CMV EBV RSV GBS Influenza QIAsymphony PHC Point of Need -23-

24 Sigma 42 Project Core Technologies: Helicase Dependent Amplification (HDA) thda mimics nature s method of replicating DNA by using helicase (± ssbp) to denature the DNA at a constant temperature of 65 C Like in PCR two sequence specific primers are flanking the DNA fragment enabling the amplification by using an enzymatic mixture QIAGEN is adding proprietary hot start function to HDA reaction to improve enzyme function -24-

25 Sigma 42 Project Portable Device for CT/GC Detection. BMBF funded Project. 09/08 applied. 02/09 approved. 06/09 launched CT/GC 50µl Process Load chip into the processing device -25-

26 Fluorescence Detection Technology by ESE Today s Standard in Point of Need Target: Method: Amplification specific for Chlamydia trachomatis detecting a portion of the major outer membrane protein (omp) Genomic DNA from Chlamydia trachomatis was used as template in a thda reaction and fluorescence was measured in real-time mode on the ESE Tube Scanner from samples containing the equivalent of 100 copies of the omp gene and negative control samples (NTC). thda Reaction on a ESE Tube Scanner -26-

27 Agenda QIAGEN - R&D Centers of Excellence - Development Cycles - R&D Budget Allocation New Product Launches & Regulatory Pipelines Prevention Profiling Personalized Healthcare Point of Need Regulatory Pipelines -27-

28 Regulatory Approval Projects USA PROJECT QUARTER YEAR Q1 Q Q3 Q4 Q1 Q Q3 Q4 Q1 Q Q3 Q4 Q1 Q Q3 Q4 artus CMV RG 6000 artus EBV RG 6000 NextGen HPV Screening TheraScreen KRAS AXpH QIAsymphony for HC 2.0 Resplex II artus Influenza cador BVDV (USDA) TheraScreen EGFR Running / planned project Start clinical trial 510K Submission PMA Submission Estimated Approval/Launch (typically 15 months after submission for PMA, 6 months for 510K) -28-

29 Selected Regulatory Approval Projects Europe PROJECT QUARTER YEAR Q1 Q Q3 Q4 Q1 Q Q3 Q4 Q1 Q Q3 Q4 Q1 Q Q3 Q4 artus HSV 1,2 artus HHV-6 NextGen HPV Screening TheraScreen KRAS RGQ TheraScreen EGFR RGQ AXpH QIAsymphony for HC 2 Resplex II Plus PyroMark Cancer Panel (12 markers) artus VZV and artus BKV Running / planned project Estimated CE Mark -29-

30 Regulatory Approval Projects China / Asia PROJECT QUARTER YEAR Q1 Q Q3 Q4 Q1 Q Q3 Q4 Q1 Q Q3 Q4 Q1 Q Q3 Q4 Digene HC2 HPV care HPV NextGen HPV Screening TheraScreen KRAS RGQ HBV v2 Blood Screening UU Test CT and NG TheraScreen EGFR RGQ Running / planned project Start clinical trial Submission Flag indicates Estimated Approval/Launch -30-

31 Regulatory Project Pipeline ( CE, FDA, SFDA) In Preparation Prevention Profiling Personalized Healthcare Point of Need CT/GC artus HHV-6 EGFR viii Tuberculosis Tricomonas artus JC (human polyomavirus) PI3K Influenza Vaginosis artus adenovirus NRAS MRSA GBS artus aspergillus ABL Clostridium difficile (CD MEK BRAF BCR-ABL -31-

32 . Thank you! -32-