Astrazeneca Plc · LSE:AZN

Astrazeneca — Q4 2022 Earnings Presentation

140 slides from Astrazeneca's earnings presentation, February 2023

  1. Slide 1: Clinical Trials Appendix Q4 2022 Results Update

    Clinical Trials Appendix Q4 2022 Results Update

  2. Slide 2: Upcoming pipeline catalysts: 2023 and 2024

    Upcoming pipeline catalysts: 2023 and 2024 Oncology BioPharmaceuticals Rare Disease H1 2023 H2 2023 2024 Imfinzi + Imjudo – hepatocellular carcinoma (1L) (HIMALAYA) (EU) Enhertu – HER2m NSCLC (2L+) (DESTINY-Lung01) (EU, JP) Imfinzi +/- Imjudo – NSCLC (1L) (POSEIDON) (EU) Enhertu – HER2-low breast ca

  3. Slide 3: Clinical Trials Appendix: selected highlights

    Clinical Trials Appendix: selected highlights BioPharmaceuticals Oncology Rare Disease Approved medicines: key LCM Dato-DXd (TROP2 ADC) tozorakimab (IL-33) volrustomig (PD-1/CTLA-4) AZD3152 (COVID-19 LAAB) vemircopan (oral Factor D) Next-wave pipeline capivasertib (AKT) eplontersen (LICA) gefurulima

  4. Slide 4: Movements since Q3 2022 update

    Movements since Q3 2022 update New to Phase I New to Phase II New to Pivotal trial New to registration NME NME NME AZD0186# MEDI1341# AZD3152¶ GLP-1R agonism type-2 diabetes alpha synuclein mAb multiple system SARS-CoV-2 LAAB prevention of COVID-19 atrophy/Parkinson’s disease AZD6793 ceralasertib +

  5. Slide 5: Movements since Q3 2022 update

    Movements since Q3 2022 update Removed from Phase I Removed from Phase II Removed from Phase III Removed from registration NME NME Life-cycle management NME AZD8701 +/- Imfinzi # navafenterol# Fasenra HUDSON Airsupra (PT027)#2 FOXP3 +/- PD-L1 mAb solid tumours MABA chronic obstructive pulmonary IL-5

  6. Slide 6: Q4 2022 Oncology new molecular entity pipeline

    Q4 2022 Oncology new molecular entity1 pipeline Phase I Phase II Phase III Under review 8 New Molecular Entities 12 New Molecular Entities 17 New Molecular Entities 0 New Molecular Entities AZD0466# AZD0171 + Imfinzi + CTx camizestrant + CDK4/6i SERENA-6 camizestrant + palbociclib SERENA-4 BCL2/xL h

  7. Slide 7: Q4 2022 Oncology life-cycle management pipeline

    Q4 2022 Oncology life-cycle management1 pipeline Phase I Phase II Phase III 2 Projects 12 Projects 34 Projects Calquence + venetoclax + obinutuzumab Enhertu (platform) DESTINY-Breast08# Enhertu (platform) DESTINY-Breast07# Calquence + R-CHOP ESCALADE Calquence ECHO# AMPLIFY# HER2 ADC HER2-low breast

  8. Slide 8: Q4 2022 BioPharmaceuticals new molecular entity pipeline

    Q4 2022 BioPharmaceuticals new molecular entity1 pipeline Phase I Phase II Phase III Under review 17 New Molecular Entities 13 New Molecular Entities 6 New Molecular Entities 0 New Molecular Entities AZD0186 AZD0780 atuliflapon AZD3152 SUPERNOVA¶ GLP-1R agonism type-2 diabetes PCSK9 dyslipidemia FLA

  9. Slide 9: Q4 2022 BioPharmaceuticals life-cycle management pipeline

    Q4 2022 BioPharmaceuticals life-cycle management1 pipeline Phase I Phase II Phase III Under review 0 Projects 3 Projects 13 Projects 0 Projects Farxiga/Forxiga DAPA-MI Andexxa Breztri/Trixeo (PT010) KALOS LOGOS SGLT2 prevention of HF and CV death following a anti-factor Xa reversal urgent surgery LA

  10. Slide 10: Q4 2022 Rare Disease pipeline

    Q4 2022 Rare Disease pipeline1 Phase I Phase II Phase III Under review 6 New Molecular Entities 5 Projects 6 Projects 1 Project acoramidis# Ultomiris CHAMPION-NMOSD ALXN1820 danicopan oral TTR stabilizer transthyretin amyloid anti-complement C5 mAb neuromyelitis optica anti-properdin bi-specific hae

  11. Slide 11: Estimated key regulatory submission acceptances

    Estimated key regulatory submission acceptances ceralasertib + Imfinzi MONETTE Dato-DXd TROPION-Breast03 melanoma adjuvant residual disease TNBC ceralasertib + Imfinzi LATIFY Dato-DXd TROPION-Breast02 gefurulimab non-small cell lung cancer 1L TNBC generalised myasthenia gravis danicopan capivasertib

  12. Slide 12: Designations in our pipeline

    Designations in our pipeline 3 14 10 14 28 Accelerated approvals Breakthrough / PRIME1 / Sakigake2 Fast Track Priority Review Orphan Andexxa Acute Major Bleed (US) Beyfortus RSV mAb-YTE MELODY-MEDLEY (US) Beyfortus RSV mAb-YTE MELODY-MEDLEY (US) Calquence MCL 1L (US) ALXN1840 WD (US) Calquence MCL 1

  13. Slide 13: Oncology: approved medicines and late-stage pipeline

    Oncology: approved medicines and late-stage pipeline

  14. Slide 14: Tagrisso: NSCLC

    Approved medicines Oncology Tagrisso (highly-selective, irreversible EGFRi) Late-stage development Early development NSCLC Trial Population Patients Design Endpoints Status CVRM Phase III Adjuvant EGFRm NSCLC 682 • Arm 1: Tagrisso QD following complete tumour resection, • Primary endpoint: DFS • FPC

  15. Slide 15: Tagrisso: NSCLC, combinations

    Approved medicines Oncology Tagrisso (highly-selective, irreversible EGFRi) Late-stage development Early development NSCLC, combinations Trial Population Patients Design Endpoints Status CVRM Phase III Neoadjuvant EGFRm NSCLC 351 • Arm 1: placebo + pemetrexed/carboplatin or • Primary endpoint: mPR •

  16. Slide 16: Tagrisso: NSCLC, combinations

    Approved medicines Oncology Tagrisso (highly-selective, irreversible EGFRi) Late-stage development Early development NSCLC, combinations Trial Population Patients Design Endpoints Status CVRM Phase III Locally advanced or metastatic NSCLC 250 • Arm 1: Tagrisso + Orpathys • Primary endpoint: PFS • FP

  17. Slide 17: Imfinzi: Gastrointestinal cancer

    Approved medicines Oncology Imfinzi (PD-L1 mAb) Late-stage development Early development Gastrointestinal cancer Trial Population Patients Design Endpoints Status CVRM Phase III Locoregional HCC 710 • Arm 1: TACE in combination with Imfinzi • Primary endpoint: PFS for Arm 2 vs. • FPCD: Q1 2019 EMERA

  18. Slide 18: Imfinzi: Lung cancer

    Approved medicines Oncology Imfinzi (PD-L1 mAb) Late-stage development Early development Lung cancer Trial Population Patients Design Endpoints Status CVRM Phase III Neoadjuvant NSCLC patients 800 • Arm 1: Imfinzi + platinum-based chemotherapy • Primary endpoints: pCR, EFS • FPCD: Q1 2019 AEGEAN Sta

  19. Slide 19: Imfinzi: Lung cancer

    Approved medicines Oncology Imfinzi (PD-L1 mAb) Late-stage development Early development Lung cancer Trial Population Patients Design Endpoints Status CVRM Phase III NSCLC 1L 1000 • Arm 1: Imfinzi + chemotherapy • Primary endpoints: OS, PFS • FPCD: Q2 2017 POSEIDON • Arm 2: Imfinzi + Imjudo + chemot

  20. Slide 20: Imfinzi: Lung cancer

    Approved medicines Oncology Imfinzi (PD-L1 mAb) Late-stage development Early development Lung cancer Trial Population Patients Design Endpoints Status CVRM Phase II NSCLC 1L 212 • Arm A1: Imfinzi • Primary endpoints: safety and • FPCD: Q1 2019 MAGELLAN • Arm A2: Imfinzi + danvatirsen tolerability •

  21. Slide 21: Imfinzi: Other cancer

    Approved medicines Oncology Imfinzi (PD-L1 mAb) Late-stage development Early development Other cancer Trial Population Patients Design Endpoints Status CVRM Phase III Non-muscle invasive bladder cancer 1018 • Arm 1: BCG (induction + maintenance) • Primary endpoint: DFS • FPCD: Q2 2018 POTOMAC • Arm

  22. Slide 22: Lynparza: Imfinzi combinations

    Approved medicines Oncology Lynparza (PARP inhibitor) Late-stage development Early development Imfinzi combinations Trial Population Patients Design Endpoints Status CVRM Phase III Advanced ovarian cancer 1L 1256 • Non tBRCAm (tumour BRCA) patients • Primary endpoint: PFS • FPCD: Q1 2019 DUO-O • Arm

  23. Slide 23: Lynparza: Multiple cancers

    Approved medicines Oncology Lynparza (PARP inhibitor) Late-stage development Early development Multiple cancers Trial Population Patients Design Endpoints Status CVRM Phase III BRCAm adjuvant breast cancer 1836 • Arm 1: Lynparza BID 12-month duration • Primary endpoint: IDFS • FPCD: Q2 2014 OlympiA

  24. Slide 24: Lynparza: Other combinations

    Approved medicines Oncology Lynparza (PARP inhibitor) Late-stage development Early development Other combinations Trial Population Patients Design Endpoints Status CVRM Phase III Metastatic castration-resistant prostate 904 • Arm 1: Lynparza + abiraterone • Primary endpoint: rPFS • FPCD: Q4 2018 PRO

  25. Slide 25: Enhertu: Breast cancer

    Approved medicines Oncology Enhertu (trastuzumab deruxtecan, HER2 ADC) Late-stage development Early development Breast cancer Trial Population Patients Design Endpoints Status CVRM Phase III HER2-positive, unresectable and/or 600 • Randomised open-label parallel assignment • Primacy endpoint: PFS •

  26. Slide 26: Enhertu: Breast cancer

    Approved medicines Oncology Enhertu (trastuzumab deruxtecan, HER2 ADC) Late-stage development Early development Breast cancer Trial Population Patients Design Endpoints Status CVRM Phase III High-risk HER2-positive early non- 624 • Randomised open-label parallel assignment • Primary endpoint: pCR •

  27. Slide 27: Enhertu: Gastric cancer

    Approved medicines Oncology Enhertu (trastuzumab deruxtecan, HER2 ADC) Late-stage development Early development Gastric cancer Trial Population Patients Design Endpoints Status CVRM Phase III HER2-positive gastric cancer or gastro- 490 • Open-label randomised parallel group assignment • Primary endp

  28. Slide 28: Enhertu: Other cancers

    Approved medicines Oncology Enhertu (trastuzumab deruxtecan, HER2 ADC) Late-stage development Early development Other cancers Trial Population Patients Design Endpoints Status CVRM Phase III HER2-mutated, unresectable, locally 264 • Randomised parallel group assignment • Primary endpoint: PFS • FPCD

  29. Slide 29: Enhertu: Other cancers

    Approved medicines Oncology Enhertu (trastuzumab deruxtecan, HER2 ADC) Late-stage development Early development Other cancers Trial Population Patients Design Endpoints Status CVRM Phase Ib HER2-over-expressing, unresectable 136 • Non-randomised parallel group assignment • Primary endpoint: safety •

  30. Slide 30: Calquence: Blood cancers

    Approved medicines Oncology Calquence (BTK inhibitor) Late-stage development Early development Blood cancers Trial Population Patients Design Endpoints Status CVRM Phase III Previously untreated CLL 535 • Arm A: chlorambucil + obinutuzumab • Primary endpoint: PFS (Arm A vs. • FPCD: Q2 2015 ACE-CL-00

  31. Slide 31: Calquence: Blood cancers

    Approved medicines Oncology Calquence (BTK inhibitor) Late-stage development Early development Blood cancers Trial Population Patients Design Endpoints Status CVRM Phase Ib MCL 70 • Calquence in combination with bendamustine and • Primary endpoint: safety • FPCD: Q2 2016 ACE-LY-106 rituxumab • LPCD:

  32. Slide 32: Orpathys: NSCLC and other cancers

    Approved medicines Oncology Orpathys (savolitinib, MET inhibitor) Late-stage development Early development NSCLC and other cancers Trial Population Patients Design Endpoints Status CVRM Phase III Locally advanced or metastatic NSCLC 163 • Single-arm trial • Primary endpoint: ORR • FPCD: Q3 2021 NCT0

  33. Slide 33: capivasertib: Breast, prostate cancer and NHL

    Approved medicines Oncology capivasertib (AKT inhibitor) Late-stage development Early development Breast cancer, prostate cancer and indolent non-hodgkin lymphoma Trial Population Patients Design Endpoints Status CVRM Phase III Locally advanced or metastatic TNBC 924 • Double-blind randomised compar

  34. Slide 34: camizestrant: Breast cancer

    Approved medicines Oncology camizestrant (AZD9833, next generation oral SERD) Late-stage development Early development Breast cancer Trial Population Patients Design Endpoints Status CVRM Phase III HR+ HER2- breast cancer 1342 • Randomised, double-blind, comparative trial • Primary endpoint: PFS • F

  35. Slide 35: camizestrant: Breast cancer

    Approved medicines Oncology camizestrant (AZD9833, next generation oral SERD) Late-stage development Early development Breast cancer Trial Population Patients Design Endpoints Status CVRM Phase I HR+ HER2- breast cancer in Chinese 30 • Dose escalation • Primary endpoints: safety and • FPCD: Q1 2021

  36. Slide 36: datopotamab deruxtecan: NSCLC

    Approved medicines Oncology datopotamab deruxtecan (TROP2 ADC) Late-stage development Early development NSCLC Trial Population Patients Design Endpoints Status CVRM Phase III Previously treated advanced or 590 • Randomised, open-label, parallel assignment • Primary endpoints: PFS, OS • FPCD: Q1 2021

  37. Slide 37: datopotamab deruxtecan: NSCLC and other cancers

    Approved medicines Oncology datopotamab deruxtecan (TROP2 ADC) Late-stage development Early development NSCLC and other cancers Trial Population Patients Design Endpoints Status CVRM Phase I Advanced or metastatic NSCLC 232 • Open-label, two-part (dose escalation, dose expansion), • Primary endpoint

  38. Slide 38: datopotamab deruxtecan: Cancer

    Approved medicines Oncology datopotamab deruxtecan (TROP2 ADC) Late-stage development Early development Cancer Trial Population Patients Design Endpoints Status CVRM Phase III Inoperable or metastatic HR+ HER2- 700 • Open-label, randomised • Primary endpoints: PFS, OS • FPCD: Q4 2021 TROPION-Breast0

  39. Slide 39: ceralasertib: Cancer

    Approved medicines Oncology ceralasertib (AZD6738, ATR inhibitor) Late-stage development Early development Cancer Trial Population Patients Design Endpoints Status CVRM Phase III Post-IO NSCLC 580 • Double-arm randomised: • Primary endpoint: OS • FPCD: Q4 2022 LATIFY • Arm 1: ceralasertib + Imfinzi

  40. Slide 40: Oncology: early-stage development

    Oncology: early-stage development

  41. Slide 41: AZD0171: Cancer

    Approved medicines Oncology AZD0171 (anti-LIF mAb) Late-stage development Early development Cancer Trial Population Patients Design Endpoints Status CVRM Phase II 1L metastatic pancreatic ductal 115 • Open-label, non-randomised trial of AZD0171 + Imfinzi + • Primary endpoints: safety, OS at 12m • FP

  42. Slide 42: AZD0466: Cancer

    Approved medicines Oncology AZD0466 (BCL2/xL inhibitor) Late-stage development Early development Cancer Trial Population Patients Design Endpoints Status CVRM Phase I/II Advanced haematologic malignancies 141 • Module 1: • Primary endpoint: safety • FPCD: Q2 2021 NCT04865419 • Part A: dose escalatio

  43. Slide 43: AZD1390: Cancer

    Approved medicines Oncology AZD1390 (ATM inhibitor) Late-stage development Early development Cancer Trial Population Patients Design Endpoints Status CVRM Phase I Recurrent glioblastoma eligible for re- 120 • Open-label trial • Primary endpoints: safety, tolerability, • FPCD: Q2 2018 NCT03423628 irr

  44. Slide 44: AZD4573: Blood cancers

    Approved medicines Oncology AZD4573 (CDK9 inhibitor) Late-stage development Early development Blood cancers Trial Population Patients Design Endpoints Status CVRM Phase II R/R peripheral T-cell lymphoma and R/R 90 • Open-label, non-randomised modular dose confirmation • Primary endpoint: efficacy •

  45. Slide 45: AZD5305: Solid tumours

    Approved medicines Oncology AZD5305 (PARP1 inhibitor) Late-stage development Early development Solid tumours Trial Population Patients Design Endpoints Status CVRM Phase I/IIa Advanced, metastatic HER2- breast 715 • Modular, open-label, multicentre dose escalation and • Primary endpoints: safety and

  46. Slide 46: AZD7789: Cancer

    Approved medicines Oncology AZD7789 (PD-1/TIM3 bispecific mAb) Late-stage development Early development Cancer Trial Population Patients Design Endpoints Status CVRM Phase I/IIa NSCLC, other tumours 81 • Open-label, non-randomised dose-escalation and dose- • Primary endpoints: AE, SAE, DLTs, ORR • F

  47. Slide 47: AZD8205: Cancer

    Approved medicines Oncology AZD8205 (B7H4 ADC) Late-stage development Early development Cancer Trial Population Patients Design Endpoints Status CVRM Phase I/II Breast cancer, biliary tract cancer, 280 • Open-label, non-randomised dose escalation and • Primary endpoints: AE, SAE, DLTs, • FPCD: Q1 20

  48. Slide 48: AZD8853: Solid tumours

    Approved medicines Oncology AZD8853 (anti-GDF15) Late-stage development Early development Solid tumours Trial Population Patients Design Endpoints Status CVRM Phase I/II Selected, advanced metastatic solid 165 • Open-label trial • Primary endpoints: safety and • FPCD: Q2 2022 NCT05397171 tumours • A

  49. Slide 49: AZD9574: Solid tumours

    Approved medicines Oncology AZD9574 (PARP1 Sel BBB inhibitor) Late-stage development Early development Solid tumours Trial Population Patients Design Endpoints Status CVRM Phase I/IIa Patients with advanced solid 255 • Modular, open-label, dose escalation trial • Primary endpoints: safety and • FPCD

  50. Slide 50: AZD9592: Lung cancer

    Approved medicines Oncology AZD9592 (EGFR-cMET TOP1i ADC) Late-stage development Early development Lung cancer Trial Population Patients Design Endpoints Status CVRM Phase I Advanced solid tumours including 108 • Escalation phase, open-label, multicentre trial • Primary endpoints (escalation): safet

  51. Slide 51: capivasertib: indolent NHL

    Approved medicines Oncology capivasertib (AKT inhibitor) Late-stage development Early development Breast cancer, prostate cancer and indolent non-hodgkin lymphoma Trial Population Patients Design Endpoints Status CVRM Phase II Participants with R/R FL, R/R MZL and 272 • Open-label, non-randomised •

  52. Slide 52: IPH5201: Solid tumours

    Approved medicines Oncology IPH5201 (CD39 mAb) Late-stage development Early development Solid tumours Trial Population Patients Design Endpoints Status CVRM Phase I Advanced solid tumours 204 • Open-label, dose-escalation trial to determine MTD of • Primary endpoints: AE, SAE, DLT • FPCD: Q1 2020 NC

  53. Slide 53: oleclumab: Cancer

    Approved medicines Oncology oleclumab (CD73 mAb) Late-stage development Early development Cancer Trial Population Patients Design Endpoints Status CVRM Phase Ib/II Pancreatic 1L and 2L with prior 339 • Arm A1: gemcitabine and nab paclitaxel i.v. • Primary endpoints: safety and anti- • FPCD: Q2 2018

  54. Slide 54: rilvegostomig: Solid tumours

    Approved medicines Oncology rilvegostomig (AZD2936, PD-1/TIGIT Bispecific mAb) Late-stage development Early development Solid tumours Trial Population Patients Design Endpoints Status CVRM Phase I NSCLC 192 • Open-label dose-escalation and dose-expansion trial • Primary endpoints (Part A): safety, •

  55. Slide 55: TNB-486: Haematologic malignancies

    Approved medicines Oncology TNB-486 (CD19/CD3 ng bispecific T-cell engager) Late-stage development Early development Haematologic malignancies Trial Population Patients Design Endpoints Status CVRM Phase I Relapsed or refractory B-cell non- 116 • Multicentre, Phase I, open-label, dose-escalation and

  56. Slide 56: volrustomig: Cancer

    Approved medicines Oncology volrustomig (PD-1/CTLA-4 bispecific mAb) Late-stage development Early development Cancer Trial Population Patients Design Endpoints Status CVRM Phase Ib Advanced renal cell carcinoma 70 • Open-label, dose-escalation and dose-expansion trial • Primary endpoints (escalation

  57. Slide 57: BioPharmaceuticals: approved medicines and late-stage pipeline

    BioPharmaceuticals: approved medicines and late-stage pipeline

  58. Slide 58: Airsupra: Asthma

    Approved medicines Oncology Airsupra (PT027, SABA/ICS, pMDI) Late-stage development Early development Asthma Trial Population Patients Design Endpoints Status CVRM Phase III Moderate to severe asthma 3132 • Randomised, double-blind, multicentre, parallel group • Primary endpoint: time to first sever

  59. Slide 59: Andexxa: Haematology

    Approved medicines Oncology Andexxa (anti-factor Xa reversal) Late-stage development Early development Haematology Trial Population Patients Design Endpoints Status CVRM Phase IV Acute intracranial 1200 • Arm 1: Andexxa • Primary endpoint: proportion of • FPCD: Q2 2019 I8-513 (Post Launch) haemorrha

  60. Slide 60: Farxiga: Heart failure and chronic kidney disease

    Approved medicines Oncology Farxiga (SGLT2 inhibitor) Late-stage development Early development Heart failure and chronic kidney disease Trial Population Patients Design Endpoints Status CVRM Phase III CHF patients with HFpEF 6263 • Arm 1: Farxiga 10mg QD • Primary endpoint: time to the first • FPCD:

  61. Slide 61: Lokelma: Hyperkalaemia

    Approved medicines Oncology Lokelma (sodium zirconium cyclosilicate) Late-stage development Early development Hyperkalaemia Trial Population Patients Design Endpoints Status CVRM Phase IIIb Patients with ESRD with 134 • Arm 1: Lokelma 5g QD for 8 weeks on non-dialysis days; • Primary endpoint: propo

  62. Slide 62: roxadustat: Anaemia

    Approved medicines Oncology roxadustat (HIF-PH inhibitor) Late-stage development Early development Anaemia Trial Population Patients Design Endpoints Status CVRM Phase III Anaemia in lower risk MDS 204 • Efficacy and safety of FG-4592 for treatment of anemia in • Primary endpoint: proportion of • FP

  63. Slide 63: eplontersen: ATTR

    Approved medicines Oncology eplontersen (ligand-conjugated antisense) Late-stage development Early development ATTR Trial Population Patients Design Endpoints Status CVRM Phase III Patients with hereditary or 1400 • Arm 1: eplontersen s.c. • Primary endpoint: composite outcome • FPCD: Q1 2020 CARDIO

  64. Slide 64: mitiperstat: Cardiovascular disease

    Approved medicines Oncology mitiperstat (MPO inhibitor) Late-stage development Early development Cardiovascular disease Trial Population Patients Design Endpoints Status CVRM Phase IIb/III HFpEF patients 1485 • Randomised, double-blind • Primary endpoint: efficacy and safety • FPCD: Q3 2021 ENDEAVOR

  65. Slide 65: tozorakimab: Diabetic kidney disease

    Approved medicines Oncology tozorakimab (IL-33 ligand mAb) Late-stage development Early development Diabetic kidney disease Trial Population Patients Design Endpoints Status CVRM Phase II Adult patients with diabetic 581 • Arm A: tozorakimab dose 1 + Farxiga • Primary endpoint: urine albumin • FPCD:

  66. Slide 66: zibotentan: Chronic kidney disease

    Approved medicines Oncology zibotentan (endothelin receptor antagonist) Late-stage development Early development Chronic kidney disease Trial Population Patients Design Endpoints Status CVRM Phase I Healthy female volunteers of 24 • Open-label, single sequence, single centre study • Primary endpoint

  67. Slide 67: zibotentan: Liver cirrhosis

    Approved medicines Oncology zibotentan (endothelin receptor antagonist) Late-stage development Early development Liver Cirrhosis with features of portal hypertension Trial Population Patients Design Endpoints Status CVRM Phase II Part A: participants with 140 • Phase IIa/b multicentre, randomised, d

  68. Slide 68: Breztri/Trixeo: Asthma

    Approved medicines Oncology Breztri/Trixeo (PT010, LAMA/LABA/ICS, pMDI) Late-stage development Early development Asthma Trial Population Patients Design Endpoints Status CVRM Phase III Severe asthma 2200 • Randomised, double-blind, double-dummy, parallel group • Primary endpoint: change from • FPCD:

  69. Slide 69: Breztri: COPD

    Approved medicines Oncology Breztri (next-generation propellant) Late-stage development Early development COPD Trial Population Patients Design Endpoints Status CVRM Phase III Moderate to very severe 542 • Randomised, double-blind, 12-week (with an extension to • Primary endpoints: number of • FPCD:

  70. Slide 70: Daliresp/Daxas: COPD

    Approved medicines Oncology Daliresp/Daxas (oral PDE4 inhibitor) Late-stage development Early development COPD Trial Population Patients Design Endpoints Status CVRM Phase IV COPD 124080 • Retrospective cohort trial comparing COPD patients aged 40 • Primary endpoint: all-cause mortality • FPCD: Q1 2

  71. Slide 71: Fasenra: Dermatology

    Approved medicines Oncology Fasenra (IL-5R mAb) Late-stage development Early development Dermatology Trial Population Patients Design Endpoints Status CVRM Phase III Patients with symptomatic 120 • Double-blind treatment period and open-label period • Primary endpoint: proportion of • FPCD: Q2 2021

  72. Slide 72: Fasenra: Gastrointestinal diseases

    Approved medicines Oncology Fasenra (IL-5R mAb) Late-stage development Early development Gastrointestinal diseases Trial Population Patients Design Endpoints Status CVRM Phase III Patients with eosinophilic 220 • Double-blind treatment period and open-label extension • Dual primary endpoints: propor

  73. Slide 73: Fasenra: Nasal polyposis and eosinophilic diseases

    Approved medicines Oncology Fasenra (IL-5R mAb) Late-stage development Early development Nasal polyposis and other eosinophilic diseases Trial Population Patients Design Endpoints Status CVRM Phase III Patients with severe bilateral 413 • Arm 1: Fasenra 30mg Q8W s.c. • Primary endpoint: effect of Fa

  74. Slide 74: Fasenra: Severe, uncontrolled asthma

    Approved medicines Oncology Fasenra (IL-5R mAb) Late-stage development Early development Severe, uncontrolled asthma Trial Population Patients Design Endpoints Status CVRM Phase IIIb Severe eosinophilic 598 • Arm 1: Fasenra 30mg Q8W s.c. • Primary endpoint: reduction of oral • FPCD: Q3 2018 PONENTE

  75. Slide 75: Fasenra: Asthma, COPD and other diseases

    Approved medicines Oncology Fasenra (IL-5R mAb) Late-stage development Early development Severe, uncontrolled asthma, COPD and other eosinophilic diseases Trial Population Patients Design Endpoints Status CVRM Phase III Patients with moderate to 642 • Double-blind, placebo-controlled • Primary endpo

  76. Slide 76: Saphnelo: Lupus

    Approved medicines Oncology Saphnelo (type I interferon receptor mAb) Late-stage development Early development Lupus (SLE / LN) Trial Population Patients Design Endpoints Status CVRM Phase III Moderate to severe SLE 630 • Arm 1: 300mg i.v. Saphnelo Q4W for 152 weeks • Primary endpoint: extension to

  77. Slide 77: Tezspire: Asthma, COPD and CRSwNP

    Approved medicines Oncology Tezspire (TSLP mAb) Late-stage development Early development Severe uncontrolled asthma, COPD and CRSwNP Trial Population Patients Design Endpoints Status CVRM Phase III Severe chronic rhinosinusitis 400 • Arm 1: Tezspire s.c. • Co-primary endpoint: nasal polyp • FPCD: Q2

  78. Slide 78: Tezspire: Severe, uncontrolled asthma

    Approved medicines Oncology Tezspire (TSLP mAb) Late-stage development Early development Severe, uncontrolled asthma Trial Population Patients Design Endpoints Status CVRM Phase III Severe asthma; age 12 to 80 1061 • Arm 1: Tezspire s.c. • Primary endpoint: annual asthma • FPCD: Q1 2018 NAVIGATOR ye

  79. Slide 79: brazikumab: Inflammatory bowel disease

    Approved medicines Oncology brazikumab (IL-23 inhibitor) Late-stage development Early development Inflammatory bowel disease (Crohn’s disease, ulcerative colitis) Trial Population Patients Design Endpoints Status CVRM Phase III Crohn’s disease 161 • Open-label, long-term extension safety study of br

  80. Slide 80: brazikumab: Inflammatory bowel disease

    Approved medicines Oncology brazikumab (IL-23 inhibitor) Late-stage development Early development Inflammatory bowel disease (Crohn’s disease, ulcerative colitis) Trial Population Patients Design Endpoints Status CVRM Phase II Ulcerative colitis 165 • A Phase II open-label, long-term extension safet

  81. Slide 81: tozorakimab: Acute respiratory failure

    Approved medicines Oncology tozorakimab (IL-33 ligand mAb) Late-stage development Early development Acute respiratory failure Trial Population Patients Design Endpoints Status CVRM Phase III Adults hospitalized for viral 2352 • Randomised, double-blind, placebo-controlled, parallel • Primary endpoin

  82. Slide 82: tozorakimab: Atopic dermatitis, asthma

    Approved medicines Oncology tozorakimab (IL-33 ligand mAb) Late-stage development Early development Atopic dermatitis, asthma Trial Population Patients Design Endpoints Status CVRM Phase II Adult subjects with atopic 148 • Randomised, double-blind, placebo-controlled trial • Primary endpoint: change

  83. Slide 83: tozorakimab: COPD

    Approved medicines Oncology tozorakimab (IL-33 ligand mAb) Late-stage development Early development COPD Trial Population Patients Design Endpoints Status CVRM Phase III Adults with symptomatic 1272 • Randomised, double-blind, placebo-controlled, parallel • Primary endpoint: annualized rate of • FPC

  84. Slide 84: Evusheld: Prevention and treatment of COVID-19

    Approved medicines Oncology Evusheld (AZD7442, tixagevimab + cilgavimab) Late-stage development Early development Prevention and treatment of COVID-19 Trial Population Patients Design Endpoints Status CVRM Phase III Adults having 5197 • Double-blinded, randomised, placebo-controlled, multi • Primary

  85. Slide 85: Evusheld: Prevention and treatment of COVID-19

    Approved medicines Oncology Evusheld (AZD7442, tixagevimab + cilgavimab) Late-stage development Early development Prevention and treatment of COVID-19 Trial Population Patients Design Endpoints Status CVRM Phase I Healthy adult; age ≥18 years 207 • Open-label, randomised three-arm, single dose trial

  86. Slide 86: Vaxzevria: Prevention of COVID-19

    Approved medicines Oncology Vaxzevria (SARS-CoV-2) Late-stage development Early development Prevention of COVID-19 Trial Population Patients Design Endpoints Status CVRM Phase IV Seronegative 360 • Open-label, non-randomised, multi-cohort, • Primary endpoints: immunogenicity • FPCD: Q1 2022 VICTORIA

  87. Slide 87: AZD3152: Prevention of COVID-19

    Approved medicines Oncology AZD3152 (SARS-CoV-2 LAAB) Late-stage development Early development Prevention of COVID-19 Trial Population Patients Design Endpoints Status CVRM Phase III Phase I: healthy adults from 3256 • 2 parts (Phase I: sentinel safety cohort, Phase: III main • Primary endpoints: to

  88. Slide 88: Beyfortus: Infection

    Approved medicines Oncology Beyfortus (RSV mAb-YTE) Late-stage development Early development Infection Trial Population Patients Design Endpoints Status CVRM Phase III Healthy infants (born 35 3000 • Randomised, double-blind, placebo-controlled • Primary endpoint: efficacy • FPCD: Q2 2021 (safety co

  89. Slide 89: BioPharmaceuticals: early-stage development

    BioPharmaceuticals: early-stage development

  90. Slide 90: atuliflapon: Cardiovascular and kidney disease

    Approved medicines Oncology atuliflapon (FLAP inhibitor) Late-stage development Early development Cardiovascular disease & chronic kidney disease Trial Population Patients Design Endpoints Status CVRM Phase IIa CAD 129 • Arm 1: atuliflapon dose A • Primary endpoint: PD effect of • FPCD: Q4 2017 NCT0

  91. Slide 91: AZD0186: Type-2 diabetes

    Approved medicines Oncology AZD0186 (oral GLP-1Ra) Late-stage development Early development Type-2 diabetes Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers 24 • Randomised, sequential assignment, sponsor-open, • Primary endpoints: safety and • FPCD: Q4 2022 NCT05694

  92. Slide 92: AZD0780: Dyslipidaemia

    Approved medicines Oncology AZD0780 (PCSK9 inhibitor) Late-stage development Early development Dyslipidaemia Trial Population Patients Design Endpoints Status CVRM Phase I Healthy adults 132 • Randomised, placebo-controlled SAD/MAD trial • Primary endpoints: safety and • FPCD: Q2 2022 NCT05384262 to

  93. Slide 93: AZD2373: Chronic kidney disease

    Approved medicines Oncology AZD2373 Late-stage development Early development Chronic kidney disease Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers 30 • SAD dose escalation in 6 cohorts with • Primary endpoints: safety and • FPCD: Q1 2020 NCT04269031 6 volunteers re

  94. Slide 94: AZD2693: NASH

    Approved medicines Oncology AZD2693 (antisense oligonucleotide) Late-stage development Early development NASH Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers 72 • SAD 6 cohorts with 6 volunteers receiving AZD2693 and 2 • Primary endpoints: safety and • FPCD: Q4 2019

  95. Slide 95: AZD3427: Heart failure

    Approved medicines Oncology AZD3427 (relaxin) Late-stage development Early development Heart failure Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers (SAD) 104 • Multicentre SAD and MAD study • Primary endpoints: safety and • FPCD: Q4 2020 NCT04630067 Heart failure (

  96. Slide 96: AZD5462: Heart failure

    Approved medicines Oncology AZD5462 (relaxin) Late-stage development Early development Heart failure Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers 0 • SAD and MAD • Primary endpoints: safety and • FPCD: Q4 2021 NCT04994106 (SAD/MAD) • Part A: SAD (8 cohorts) toler

  97. Slide 97: AZD6234: Obesity

    Approved medicines Oncology AZD6234 (long-acting amylin) Late-stage development Early development Obesity with related comorbidities Trial Population Patients Design Endpoints Status CVRM Phase I Healthy patients who are 64 • SAD trial • Primary endpoint: safety • FPCD: Q4 2022 NCT05511025 overweigh

  98. Slide 98: AZD7503: NASH

    Approved medicines Oncology AZD7503 (antisense oligonucleotide) Late-stage development Early development NASH Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers 56 • SAD 7 cohorts with 8 volunteers receiving AZD7503 and 2 • Primary endpoints: safety and • FPCD: Q4 2021

  99. Slide 99: balcinrenone/dapagliflozin: Heart failure

    Approved medicines Oncology balcinrenone/dapagliflozin (MR modulator + SGLT2i) Late-stage development Early development Heart failure Trial Population Patients Design Endpoints Status CVRM Phase IIb Heart failure with chronic 500 • Randomised, stratified according to T2DM and eGFR (≥20 to • Primary

  100. Slide 100: cotadutide: Diabetes/CKD, NASH

    Approved medicines Oncology cotadutide (GLP-1-glucagon agonist) Late-stage development Early development Diabetes/CKD, NASH Trial Population Patients Design Endpoints Status CVRM Phase II/III Patients with F2/F3 biopsy 1860 • Phase IIb/III randomised, double-blind, placebo-controlled • Primary endpo

  101. Slide 101: MEDI6570: Cardiovascular

    Approved medicines Oncology MEDI6570 Late-stage development Early development Cardiovascular Trial Population Patients Design Endpoints Status CVRM Phase IIb Post-myocardial infarction 400 • Evaluation of anti-inflammatory potential and effect on • Primary endpoints: safety and efficacy • FPCD: Q4 2

  102. Slide 102: MEDI8367: Chronic kidney disease

    Approved medicines Oncology MEDI8367 Late-stage development Early development Chronic kidney disease Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers 12 • SAD trial • Primary endpoints: safety and • FPCD: Q3 2020 NCT04365218 CKD • 6 cohorts tolerability • LPCD: Q4 20

  103. Slide 103: mitiperstat: Cardiovascular disease

    Approved medicines Oncology mitiperstat (MPO inhibitor) Late-stage development Early development Cardiovascular disease Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers 14 • Open-label • Primary endpoint: PK parameters • FPCD: Q1 2022 NCT05236543 • mitiperstat • Seco

  104. Slide 104: mitiperstat: NASH

    Approved medicines Oncology mitiperstat (MPO inhibitor) Late-stage development Early development NASH Trial Population Patients Design Endpoints Status CVRM Phase II NASH patients 90 • Randomised, placebo-controlled, double-blind • Primary endpoint: safety, tolerability • FPCD: Q4 2022 COSMOS • Arm

  105. Slide 105: zibotentan: Chronic kidney disease

    Approved medicines Oncology zibotentan (endothelin receptor antagonist) Late-stage development Early development Chronic kidney disease Trial Population Patients Design Endpoints Status CVRM Phase IIb CKD 495 • Arm 1: zibotentan dose A + Farxiga 10mg QD (n=166) • Primary endpoint: change in log- • F

  106. Slide 106: atuliflapon: Asthma

    Approved medicines Oncology atuliflapon (FLAP inhibitor) Late-stage development Early development Asthma Trial Population Patients Design Endpoints Status CVRM Phase II Patients with moderate-to- 1928 • Randomised, placebo-controlled, double-blind, multicentre, • Primary endpoint: time to first • FP

  107. Slide 107: AZD1402: Asthma

    Approved medicines Oncology AZD1402 (IL-4 receptor alpha antagonist) Late-stage development Early development Asthma Trial Population Patients Design Endpoints Status CVRM Phase IIa Patients with asthma on 225 • Randomised, placebo-controlled, double-blinded, • Primary endpoints (Part 1): safety and

  108. Slide 108: AZD4604: Asthma

    Approved medicines Oncology AZD4604 (inhaled JAK-1 inhibitor) Late-stage development Early development Asthma Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers and 137 • SAD/MAD/POM trial • Primary endpoints: safety and • FPCD: Q4 2021 NCT04769869 patients with mild a

  109. Slide 109: AZD5055: Idiopathic pulmonary fibrosis

    Approved medicines Oncology AZD5055 (oral porcupine inhibitor) Late-stage development Early development Idiopathic pulmonary fibrosis (IPF) and other ILDs with progressive fibrosis Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers 90 • SAD/MAD trial • Primary endpoint

  110. Slide 110: AZD6793: Inflammatory diseases

    Approved medicines Oncology AZD6793 (IRAK4) Late-stage development Early development Inflammatory diseases Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers 133 • Single blind, randomised, placebo-controlled study to • Primary endpoints: safety and • FPCD: Q4 2022 NCT

  111. Slide 111: AZD7798: Crohn's disease

    Approved medicines Oncology AZD7798 (humanized monoclonal antibody) Late-stage development Early development Crohn's disease Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers 64 • SAD • Primary endpoints: safety and • FPCD: Q3 2022 NCT05452304 • Arm1: AZD7798 tolerabi

  112. Slide 112: AZD8630: Asthma

    Approved medicines Oncology AZD8630 (inhaled TSLP) Late-stage development Early development Asthma Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers and 232 • SAD and MAD trial • Primary endpoints: safety and • FPCD: Q1 2022 NCT05110976 patients with asthma tolerabili

  113. Slide 113: mitiperstat: COPD

    Approved medicines Oncology mitiperstat (MPO inhibitor) Late-stage development Early development COPD Trial Population Patients Design Endpoints Status CVRM Phase IIa Moderate to severe COP; age 288 • Randomised, double-blind study • Primary endpoint: time to first COPD • FPCD: Q1 2023 CRESCENDO 40

  114. Slide 114: AZD4041: Opioid use disorder

    Approved medicines Oncology AZD4041 (orexin 1 receptor antagonist) Late-stage development Early development Opioid use disorder Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers 48 • Randomised, double-blind, SAD trial • Primary endpoints: safety and • FPCD: Q4 2019 N

  115. Slide 115: MEDI0618: Osteoarthritis pain

    Approved medicines Oncology MEDI0618 (PAR2 antagonist mAb) Late-stage development Early development Osteoarthritis pain Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers 64 • SAD trial • Primary endpoints: safety and • FPCD: Q4 2019 NCT04198558 • Arm 1: MEDI0618 i.v.

  116. Slide 116: MEDI1341: Multiple system atrophy

    Approved medicines Oncology MEDI1341 (alpha-synuclein mAb) Late-stage development Early development Multiple system atrophy Trial Population Patients Design Endpoints Status CVRM Phase II Patients with diagnosis of 138 • Randomised, double-blind placebo-controlled study • Primary endpoint: efficacy,

  117. Slide 117: MEDI1341: Parkinson's disease

    Approved medicines Oncology MEDI1341 (alpha-synuclein mAb) Late-stage development Early development Parkinson’s disease Trial Population Patients Design Endpoints Status CVRM Phase I Parkinson’s disease 25 • MAD trial • Primary endpoints: safety and • FPCD: Q3 2020 NCT04449484 • Arm 1: MEDI1341 i.v.

  118. Slide 118: MEDI7352: Osteoarthritis pain

    Approved medicines Oncology MEDI7352 (NGF TNF bispecific mAb) Late-stage development Early development Osteoarthritis pain Trial Population Patients Design Endpoints Status CVRM Phase IIb Painful osteoarthritis of the 300 • MAD trial • Primary endpoint: dose response • FPCD: Q1 2021 NCT04675034 knee

  119. Slide 119: Rare Disease: approved medicines and late-stage pipeline

    Rare Disease: approved medicines and late-stage pipeline

  120. Slide 120: Koselugo: Neurofibromatosis Type 1

    Approved medicines Oncology Koselugo (selumetinib, MEK inhibitor) Late-stage development Early development Neurofibromatosis Type 1 (NF1) Trial Population Patients Design Endpoints Status CVRM Phase III Adult age ≥18 years with NF1 who have 146 • Multicentre, international study with a parallel, ran

  121. Slide 121: Koselugo: Paediatric NF1, solid tumours

    Approved medicines Oncology Koselugo (selumetinib, MEK inhibitor) Late-stage development Early development Paediatric neurofibromatosis type 1, solid tumours Trial Population Patients Design Endpoints Status CVRM Phase II Paediatric NF1 75 • Single-arm: Koselugo 25mg/m2 BID with 2 strata • Primary e

  122. Slide 122: Ultomiris: Haematology & nephrology

    Approved medicines Oncology Ultomiris (anti-C5 mAb) Late-stage development Early development Haematology & nephrology Trial Population Patients Design Endpoints Status CVRM Phase III PNH and aHUS 136 • Ultomiris s.c. • Primary endpoint: Day 71 serum • FPCD: Q1 2019 ALXN1210-PNH-303 Ultomiris Ctrough

  123. Slide 123: Ultomiris: Neurology

    Approved medicines Oncology Ultomiris (anti-C5 mAb) Late-stage development Early development Neurology Trial Population Patients Design Endpoints Status CVRM Phase III NMOSD 58 • Arm 1: Ultomiris Q8W • Primary endpoint: time to first • FPCD: Q4 2019 ALXN1210-NMO-307 adjudicated on-trial relapse • LP

  124. Slide 124: acoramidis: ATTR-CM

    Approved medicines Oncology acoramidis (ALXN2060) Late-stage development Early development ATTR-CM Trial Population Patients Design Endpoints Status CVRM Phase III ATTR-CM 22 • Arm 1: 800mg acoramidis (ALXN2060) administered twice • Primary endpoint: change from baseline • FPCD: Q4 2020 ALXN2060-TAC

  125. Slide 125: ALXN1840: Wilson disease

    Approved medicines Oncology ALXN1840 (bis-choline tetrathiomolybdate) Late-stage development Early development Wilson disease Trial Population Patients Design Endpoints Status CVRM Phase III Wilson disease 215 • Arm 1: ALXN1840 administered orally for 48 weeks at doses • Primary endpoint: daily mean

  126. Slide 126: anselamimab: AL amyloidosis

    Approved medicines Oncology anselamimab (CAEL-101, fibril-reactive mAb) Late-stage development Early development AL amyloidosis Trial Population Patients Design Endpoints Status CVRM Phase III Mayo Stage IIIa amyloidosis 267 • Arm 1: anselamimab combined with SoC for PCD • Primary endpoint: time fro

  127. Slide 127: danicopan: Haematology & ophthalmology

    Approved medicines Oncology danicopan (ALXN2040, oral factor D inhibitor) Late-stage development Early development Haematology & ophthalmology Trial Population Patients Design Endpoints Status CVRM Phase III PNH with clinically meaningful EVH 84 • Arm 1: danicopan + C5 inhibitor • Primary endpoint:

  128. Slide 128: gefurulimab: Neurology and nephrology

    Approved medicines Oncology gefurulimab (ALXN1720, anti-C5 bi-specific minibody) Late-stage development Early development Neurology and nephrology Trial Population Patients Design Endpoints Status CVRM Phase III Generalized myasthenia gravis 200 • Arm 1: weight-based maintenance treatment with • Pri

  129. Slide 129: Rare Disease: early-stage development

    Rare Disease: early-stage development

  130. Slide 130: ALXN1820: Haematology

    Approved medicines Oncology ALXN1820 (anti-properdin) Late-stage development Early development Haematology Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers 60 • Arm 1: ALXN1820 administered s.c. or i.v., multiple ascending • Primary endpoint: incidence of TEAEs • FPC

  131. Slide 131: ALXN1850: Hypophosphatasia

    Approved medicines Oncology ALXN1850 (next generation asfotase alfa) Late-stage development Early development Hypophosphatasia Trial Population Patients Design Endpoints Status CVRM Phase I Hypophosphatasia 15 • Arm 1: ALXN1850, 3 cohorts at low, medium and high dosages • Primary endpoint: incidence

  132. Slide 132: ALXN1910: Bone metabolism

    Approved medicines Oncology ALXN1910 (next generation TNSALP ERT) Late-stage development Early development Bone metabolism Trial Population Patients Design Endpoints Status CVRM Phase I Healthy adults 48 • Randomised, placebo-controlled SAD • Primary endpoint: safety • FPCD: Q2 2022 ALXN1910-HV-101

  133. Slide 133: ALXN2030: Nephrology

    Approved medicines Oncology ALXN2030 (siRNA targeting complement C3) Late-stage development Early development Nephrology Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers 48 • Randomised, placebo-controlled SAD • Primary endpoint: safety • FPCD: Q4 2022 ALXN2030-HV-10

  134. Slide 134: ALXN2080: Complement-mediated disease

    Approved medicines Oncology ALXN2080 (oral factor D inhibitor) Late-stage development Early development Complement-mediated disease Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers 100 • SAD/MAD trial • Primary endpoints: safety and • FPCD: Q3 2022 ALXN2080-HV-101 to

  135. Slide 135: vemircopan: Haematology, nephrology, neurology

    Approved medicines Oncology vemircopan (ALXN2050, oral factor D inhibitor) Late-stage development Early development Haematology, nephrology, neurology Trial Population Patients Design Endpoints Status CVRM Phase II Paroxysmal nocturnal hemoglobinuria 28 • Arm 1: vemircopan monotherapy with groups in

  136. Slide 136: Glossary

    Previous Next Glossary 14C Carbon 14 AUCinf Area under plasma concentration time curve from zero to infinity BTK Bruton's tyrosine kinase Area under plasma concentration curve from zero to the last 1L, 2L, 3L 1st-, 2nd- or 3rd-line AUClast BVAS Birmingham Vasculitis Activity Score quantifiable conce

  137. Slide 137: Glossary

    Previous Next Glossary CRC Colorectal cancer DSQ Dysphagia Symptom Questionnaire ET Endocrine therapy CrCl Creatinine clearance DXA Dual energy X-ray absorptiometry EVH Extravascular haemolysis CRR Complete response rate EBRT External beam radiation therapy FAF Fundus autofluorescence CRR Complete r

  138. Slide 138: Glossary

    Previous Next Glossary hADME Human mass balance ICS Inhaled corticosteroid m Mutation HCC Hepatocellular carcinoma ICU Intensive care unit mAb Monoclonal antibody HD High dose IDFS Invasive disease-free survival MABA Muscarinic antagonist-beta2 agonist HDL-C High-density lipoprotein cholesterol IgAN

  139. Slide 139: Glossary

    Previous Next Glossary NCFB Non-cystic fibrosis bronchiectasis PD Pharmacodynamics PSMA Prostate-specific membrane antigen NCI National Cancer Institute PD-1 Programmed cell death protein-1 PSR Platinum-sensitive relapsed NCPV Noncalcified plaque volume PDAC Pancreatic ductal adenocarcinoma PTCL Per

  140. Slide 140: Glossary

    Previous Next Glossary SAE Serious adverse event TIGIT T-cell immunoreceptor with Ig and ITIM domains SBRT Stereotactic body radiation therapy TIM3 T-cell immunoglobulin and mucin domain 3 SCCHN Squamous-cell carcinoma of the head and neck TJC Tender joint count SCLC Small cell lung cancer TKI Tyros

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