Astrazeneca Plc · LSE:AZN

Astrazeneca — Q1 2025 Earnings Presentation

143 slides from Astrazeneca's earnings presentation, Apr 29, 2025

  1. Slide 1: Clinical Trials Appendix

    Clinical Trials Appendix 1Q 2025 Results Update 29 April 2025

  2. Slide 2: Pipeline at a glance

    Pipeline 194 18 at a glance projects in our new molecular entities development pipeline (NME) in our late-stage pipeline Across five focus therapy areas: Oncology BioPharmaceuticals 132 16 CVRM | R&I | V&I new molecular entities regulatory approvals (NME) or major lifecycle in major markets manageme

  3. Slide 3: Key upcoming pipeline catalysts: 2025 and 2026

    Key upcoming pipeline catalysts: 2025 and 2026 Oncology BioPharmaceuticals Rare Disease H1 2025 H2 2025 2026 Calquence – MCL (1L) (ECHO) (EU) Calquence – MCL (1L) (ECHO) (JP) Enhertu – HER2-low and ultralow met. breast cancer (DESTINY-Breast06) (CN) Tagrisso – EGFRm NSCLC (unresectable, Stg. III) (L

  4. Slide 4: Clinical Trials Appendix: selected highlights

    Clinical Trials Appendix: selected highlights BioPharmaceuticals Oncology Rare Disease BioPharmaceuticals Oncology Rare Disease AIRSUPRA™ Inhalation Aerosol For Health Care Providers DATROWAY® (datopotamab deruxtecan-dlnk) is Now Approved Approved medicines: Koselugo® (selumetinib) | Official HCP We

  5. Slide 5: Project movements since Q4 2024 update

    Project movements since Q4 2024 update New to Phase I New to Phase II New to pivotal trial New to registration NME NME Additional indication Life-cycle management AZD2284 AZD9550 + AZD6234 baxdrostat/dapagliflozin Imfinzi + CRT PACIFIC-5 (China)# STEAP2 radioconjugate prostate cancer GLP-1R glucagon

  6. Slide 6: Project movements since Q4 2024 update

    Project movements since Q4 2024 update Removed from Phase I Removed from Phase II Removed from Phase III Approved/removed from registration NME NME Life-cycle management NME MEDI1814# MEDI0618 Truqap + docetaxel CAPItello-280 Beyonttra (acoramidis)# amyloid beta mAb Alzheimer’s disease PAR2 antagoni

  7. Slide 7: Q1 2025 Oncology new molecular entity pipeline

    Q1 2025 Oncology new molecular entity1 pipeline Phase I Phase II Phase III 23 New Molecular Entities 15 New Molecular Entities 19 New Molecular Entities Imfinzi +/- oleclumab +/- monalizumab PACIFIC-9# volrustomig + lenvatinib AZD0022 camizestrant ceralasertib camizestrant + CDK4/6i SERENA-6 PD-L1+N

  8. Slide 8: Q1 2025 Oncology lifecycle management pipeline

    Q1 2025 Oncology lifecycle management1 pipeline Phase I Phase II Phase III 1 Project 9 Projects 37 Projects AZD0486 SOUNDTRACK-E Datroway + Imfinzi + CTx AVANZAR# Datroway + Imfinzi TROPION-Breast04# Enhertu (platform) DESTINY-Breast07# Calquence + R-CHOP ESCALADE CD19/CD3 next-generation bispecific

  9. Slide 9: Q1 2025 BioPharmaceuticals new molecular entity pipeline

    Q1 2025 BioPharmaceuticals new molecular entity1 pipeline Phase I Phase II Phase III Under Review 12 New Molecular Entities 17 New Molecular Entities 7 New Molecular Entities 0 New Molecular Entities AZD0120 atuliflapon balcinrenone/dapagliflozin balcinrenone/dapagliflozin autologous anti-CD19 and a

  10. Slide 10: Q1 2025 BioPharmaceuticals life cycle management pipeline

    Q1 2025 BioPharmaceuticals life cycle management1 pipeline Phase I Phase II Phase III Under review 0 Projects 0 Projects 13 Projects 1 Project Breztri/Trixeo (PT010) KALOS LOGOS Breztri/Trixeo ATHLOS Tezspire WAYPOINT# LABA/LAMA/ICS asthma LABA/LAMA/ICS COPD cardiopulmonary exercise trial TSLP nasal

  11. Slide 11: Q1 2025 Rare Disease pipeline

    Q1 2025 Rare Disease pipeline1 Phase I Phase II Phase III Under Review 3 Projects 2 Projects 8 Projects 1 Project ALXN1920 Ultomiris Ultomiris Koselugo KOMET# kidney-targeted factor H fusion protein nephrology anti-complement C5 mAb haematopoietic stem cell transplant– anti-complement C5 mAb prolife

  12. Slide 12: Active designations in our pipeline

    Active designations in our pipeline 2 4 12 3 18 Priority Review Breakthrough / PRIME1 / Sakigake2 Fast Track Qualified infectious disease product Orphan Datroway EGFRm post-TKI NSCLC 3L+ Tezspire COPD EMBARK, JOURNEY (US) AZD0292 Psl-PcrV N3Y NCFBE (US) AZD0292 Psl-PcrV N3Y NCFBE (US) Fasenra HES NA

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

    Oncology: approved medicines and late-stage pipeline 13

  14. Slide 14: Oncology: Calquence

    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 981 • Arm 1; Calquence + venetoclax • Primary endpoint: IRC PFS (Arm 1 vs. • FPCD: Q1 2019 AMPLIFY (A

  15. Slide 15: Oncology: Datroway breast cancer

    Approved medicines Oncology Datroway (dapotamab deruxtecan, TROP2 ADC) Late-stage development Early development Breast cancer Trial Population Patients Design Endpoints Status CVRM Phase III Inoperable or metastatic HR+ HER2- 732 • Open-label, randomised trial • Primary endpoints: PFS (BICR) and OS

  16. Slide 16: Oncology: Datroway NSCLC

    Approved medicines Oncology Datroway (dapotamab deruxtecan, TROP2 ADC) Late-stage development Early development NSCLC Trial Population Patients Design Endpoints Status CVRM Phase III 1L NSCLC 1280 • Arm 1: carboplatin + Datroway + Imfinzi • Co-primary endpoints: PFS and OS in • FPCD: Q1 2023 AVANZAR

  17. Slide 17: Oncology: Datroway NSCLC

    Approved medicines Oncology Datroway (dapotamab deruxtecan, TROP2 ADC) Late-stage development Early development NSCLC Trial Population Patients Design Endpoints Status CVRM Phase III EGFRm locally advanced or metastatic 562 • Arm 1: Tagrisso + Datroway • Primary endpoint: PFS (BICR) • FPCD: Q2 2024

  18. Slide 18: Oncology: Datroway other cancers

    Approved medicines Oncology Datroway (dapotamab deruxtecan, TROP2 ADC) Late-stage development Early development Other cancers Trial Population Patients Design Endpoints Status CVRM Phase II Endometrial cancer, gastric cancer, 556 • Sub-study 1 (endometrial cancer); • Primary endpoints: ORR and safet

  19. Slide 19: Oncology: 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 • Primary endpoint: PFS

  20. Slide 20: Oncology: 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- 927 • Randomised, open-label, parallel assignment • Primary endpoint: pCR

  21. Slide 21: Oncology: 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 GEJ 490 • Open-label, randomised, parallel group assignment • Primary endpoi

  22. Slide 22: Oncology: 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 450 • Randomised, parallel group assignment • Primary endpoint: PFS • FPC

  23. Slide 23: Oncology: 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 Advanced treatment-naïve HER2- 620 • Arm A: Enhertu + rilvegostomig • Primary endpoint: OS • FPCD: Q3 2024 DE

  24. Slide 24: Oncology: 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 (Arm 2 vs. Arm • FPCD: Q1 2019 EMER

  25. Slide 25: Oncology: 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 Perioperative NSCLC patients, Stage II 800 • Arm 1: Imfinzi + platinum-based chemotherapy • Primary endpoints: pCR snd EFS • FPCD: Q1

  26. Slide 26: Oncology: 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, patients who progressed on an 531 • Open-label, biomarker-directed, multi-centre trial • Primary endpoint: ORR • FPCD: Q1 2018 H

  27. Slide 27: Oncology: 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

  28. Slide 28: Oncology: 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 1L advanced ovarian cancer 1407 • Non-tBRCAm (tumour BRCA) patients • Primary endpoint: PFS • FPCD: Q1 2019 DUO-O • Arm

  29. Slide 29: Oncology: Lynparza other cancer

    Approved medicines Oncology Lynparza (PARP inhibitor) Late-stage development Early development Other cancer Trial Population Patients Design Endpoints Status CVRM Phase III BRCAwt advanced ovarian cancer, 1L 366 • Arm 1: Lynparza BID 24-month duration • Primary endpoints: PFS (BRCAwt HRD- • FPCD: Q3

  30. Slide 30: Oncology: Orpathys

    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

  31. Slide 31: Oncology: Tagrisso NSCLC

    Approved medicines Oncology Tagrisso (highly-selective, irreversible EGFR inhibitor) Late-stage development Early development NSCLC Trial Population Patients Design Endpoints Status CVRM Phase III Maintenance therapy in patients with 216 • Arm 1: Tagrisso • Primary endpoint: PFS (BICR) • FPCD: Q4 20

  32. Slide 32: Oncology: Tagrisso combinations

    Approved medicines Oncology Tagrisso (highly-selective, irreversible EGFR inhibitor) 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 endpoi

  33. Slide 33: Oncology: Truqap

    Approved medicines Oncology Truqap (capivasertib, AKT inhibitor) Late-stage development Early development Breast cancer and prostate cancer Trial Population Patients Design Endpoints Status CVRM Phase III 2L+ AI-resistant locally advanced 834 • Double-blind, randomised, comparative trial • Primary e

  34. Slide 34: Oncology: 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-negative advanced breast 1370 • Randomised, double-blind, comparative trial • Primary endpoin

  35. Slide 35: Oncology: 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-negative advanced breast 18 • Open-label trial • Primary endpoints: safety and • FPCD: Q4 2020

  36. Slide 36: Oncology: ceralasertib

    Approved medicines Oncology ceralasertib (AZD6738, ATR inhibitor) Late-stage development Early development Multiple cancers Trial Population Patients Design Endpoints Status CVRM Phase III Post-IO NSCLC 594 • Double-arm randomised • Primary endpoint: OS • FPCD: Q4 2022 LATIFY • Arm 1: ceralasertib +

  37. Slide 37: Oncology: rilvegostomig lung cancer

    Approved medicines Oncology rilvegostomig (AZD2936, PD-1/TIGIT bispecific mAb) Late-stage development Early development Lung cancer Trial Population Patients Design Endpoints Status CVRM Phase III Non-squamous NSCLC 1L patients 878 • Randomised, double-blind, multi-centre trial • Primary endpoints:

  38. Slide 38: Oncology: 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 IIb Gastric cancer 240 • Open-label gastric platform trial • Primary endpoints: safety and efficacy • FPC

  39. Slide 39: Oncology: saruparib

    Approved medicines Oncology saruparib (AZD5305, PARP1 inhibitor) Late-stage development Early development Solid tumours Trial Population Patients Design Endpoints Status CVRM Phase III HRRm and non-HRRm mCSPC 1800 • Randomised, placebo-controlled trial • Primary endpoint: rPFS • FPCD: Q4 2023 EvoPAR

  40. Slide 40: Oncology: volrustomig solid tumours

    Approved medicines Oncology volrustomig (MEDI5752, PD-1/CTLA-4 bispecific mAb) Late-stage development Early development Solid tumours Trial Population Patients Design Endpoints Status CVRM Phase III High-risk locally advanced cervical 800 • Randomised, double-blind, placebo-controlled, multi-centre

  41. Slide 41: Oncology: volrustomig solid tumours

    Approved medicines Oncology volrustomig (MEDI5752, PD-1/CTLA-4 bispecific mAb) Late-stage development Early development Solid tumours Trial Population Patients Design Endpoints Status CVRM Phase Ib Advanced renal cell carcinoma 67 • Open-label, dose escalation and dose expansion trial • Primary endp

  42. Slide 42: Oncology: AZD0486

    Approved medicines Oncology AZD0486 (CD19/CD3 next-generation bispecific T-cell engager) Late-stage development Early development Haematologic malignancies Trial Population Patients Design Endpoints Status CVRM Phase III Previously untreated follicular 1015 • Multi-centre, randomised, open-label tri

  43. Slide 43: Oncology: AZD0901

    Approved medicines Oncology AZD0901 (CLDN18.2 MMAE ADC) Late-stage development Early development Solid tumours Trial Population Patients Design Endpoints Status CVRM Phase III 2L+ advanced or metastatic gastric or 572 • Multi-centre, open-label, sponsor-blinded, randomised trial • Primary endpoints:

  44. Slide 44: Oncology: early-stage development

    Oncology: early-stage development 44

  45. Slide 45: Oncology: FPI-2265

    Approved medicines Oncology FPI-2265 (PSMA radioconjugate) Late-stage development Early development Prostate cancer Trial Population Patients Design Endpoints Status CVRM Phase II PSMA-positive mCRPC previously 60 • Open-label, randomised, multi-centre trial • Primary endpoints: PSA50 and safety • F

  46. Slide 46: Oncology: oleclumab

    Approved medicines Oncology oleclumab (CD73 mAb) Late-stage development Early development Solid tumours 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: Q

  47. Slide 47: Oncology: puxitatug samrotecan

    Approved medicines Oncology puxitatug samrotecan (AZD8205, B7H4 ADC) Late-stage development Early development Solid tumours Trial Population Patients Design Endpoints Status CVRM Phase I/II Breast cancer, ovarian cancer, 370 • Open-label dose escalation and expansion trial • Primary endpoints: AE, S

  48. Slide 48: Oncology: AZD0022

    Approved medicines Oncology AZD0022 (KRASG12D) Late-stage development Early development Solid tumours Trial Population Patients Design Endpoints Status CVRM Phase I/II Metastatic or locally advanced 430 • Open-label, multi-centre trial with FIH modular protocol • Primary endpoints (Part A and B): •

  49. Slide 49: Oncology: AZD0120

    Approved medicines Oncology AZD0120 (GC012F, autologous anti-CD19 and anti-BCMA CAR-T) Late-stage development Early development Blood cancers Trial Population Patients Design Endpoints Status CVRM Phase I/II Relapsed/refractory multiple myeloma 68 • Open-label, single-arm, multi-centre trial • Prima

  50. Slide 50: Oncology: AZD0305

    Approved medicines Oncology AZD0305 (GPRC5D ADC) Late-stage development Early development Blood cancers Trial Population Patients Design Endpoints Status CVRM Phase I/II R/R multiple myeloma 108 • Open-label, dose escalation and dose expansion trial • Primary endpoints: occurrence of • FPCD: Q4 2023

  51. Slide 51: Oncology: AZD0754

    Approved medicines Oncology AZD0754 (STEAP2 dnTGFβRII-armoured CAR-T) Late-stage development Early development Solid tumours Trial Population Patients Design Endpoints Status CVRM Phase I/II Metastatic castration resistance 60 • Open-label, single-arm, multi-centre trial with dose • Primary endpoint

  52. Slide 52: Oncology: AZD1390

    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- 165 • Open-label trial • Primary endpoints: safety, tolerability • FPCD: Q2 2018 NCT03423628 irra

  53. Slide 53: Oncology: AZD2068

    Approved medicines Oncology AZD2068 (FPI-2068, EGFR cMET radioconjugate) Late-stage development Early development Solid tumours Trial Population Patients Design Endpoints Status CVRM Phase I Advanced solid tumours 110 • Multicentre, open-label dose escalation trial • Primary endpoints: safety and •

  54. Slide 54: Oncology: AZD2284

    Approved medicines Oncology AZD2284 (STEAP2 radioconjugate) Late-stage development Early development Prostate cancer Trial Population Patients Design Endpoints Status CVRM Phase I mCRPC 134 • Non-randomised, open-label • Primary endpoints: Safety and • FPCD: Q1 2025 NCT06879041 • Part A (Imaging): t

  55. Slide 55: Oncology: AZD3470

    Approved medicines Oncology AZD3470 (PRMT5) Late-stage development Early development Solid tumours and blood cancer Trial Population Patients Design Endpoints Status CVRM Phase I MTAP-deficient advanced solid tumours 210 • Arm 1: AZD3470 • Primary endpoints: safety and • FPCD: Q1 2024 PRIMROSE toler

  56. Slide 56: Oncology: AZD5335

    Approved medicines Oncology AZD5335 (anti-FRα TOP1i ADC) Late-stage development Early development Solid tumours, ovarian cancer, lung cancer Trial Population Patients Design Endpoints Status CVRM Phase I/II Advanced solid tumour malignancies 446 • Module 1: AZD5335 monotherapy • Primary endpoints: s

  57. Slide 57: Oncology: AZD5492

    Approved medicines Oncology AZD5492 (CD20 TITAN TCE) Late-stage development Early development Blood cancers Trial Population Patients Design Endpoints Status CVRM Phase I CLL, MCL, LBCL, FL 176 • Module 1: AZD5492 monotherapy • Primary endpoints: safety and • FPCD: Q3 2024 TITANIUM • AZD5492 monothe

  58. Slide 58: Oncology: AZD5851

    Approved medicines Oncology AZD5851 (GPC3 dnTGFβRII-armoured CAR-T) Late-stage development Early development Gastrointestinal cancer Trial Population Patients Design Endpoints Status CVRM Phase I/II GPC3-positive advanced/recurrent HCC 94 • Open-label, single-arm, multi-centre trial with dose • Prim

  59. Slide 59: Oncology: AZD5863

    Approved medicines Oncology AZD5863 (CLDN18.2 CD3 bispecific antibody) Late-stage development Early development Solid tumours Trial Population Patients Design Endpoints Status CVRM Phase I Advanced or metastatic solid tumours 240 • Part A: dose escalation phase to determine the safety, • Primary end

  60. Slide 60: Oncology: AZD6422

    Approved medicines Oncology AZD6422 (CLDN18.2 CAR-T) Late-stage development Early development Solid tumours Trial Population Patients Design Endpoints Status CVRM Phase I Advanced or metastatic CLDN18.2- 96 • Open-label trial, dose escalation (Part 1) and dose • Primary endpoints: incidence of • FPC

  61. Slide 61: Oncology: AZD7003

    Approved medicines Oncology AZD7003 (GPC3 CAR-T) Late-stage development Early development Hepatocellular carcinoma (HCC) Trial Population Patients Design Endpoints Status CVRM Phase I/II GPC3-positive advanced/recurrent HCC 121 • Open-label, single-arm, multi-centre trial with dose • Primary endpoin

  62. Slide 62: Oncology: AZD8421

    Approved medicines Oncology AZD8421 (CDK2 inhibitor) Late-stage development Early development Breast cancer Trial Population Patients Design Endpoints Status CVRM Phase I/II ER+ HER2-negative advanced breast 204 • Module 1: AZD8421 • Primary endpoints: safety and • FPCD: Q4 2023 CYCAD-1 cancer • Mod

  63. Slide 63: Oncology: AZD9574

    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 Advanced solid malignancies 490 • Modular, open-label, multi-centre dose escalation and • Primary endpoints: safety

  64. Slide 64: Oncology: AZD9592

    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 228 • Escalation phase, open-label, multi-centre trial • Primary endpoints (escalation): safe

  65. Slide 65: Oncology: AZD9793

    Approved medicines Oncology AZD9793 (GPC3 TITAN T-cell engager) Late-stage development Early development Solid tumours Trial Population Patients Design Endpoints Status CVRM Phase I/II Metastatic solid tumours 304 • Open label, non-randomised, multi-centre, dose escalation • Primary endpoint: safety

  66. Slide 66: Oncology: AZD9829

    Approved medicines Oncology AZD9829 (CD123 TOP1i ADC) Late-stage development Early development Blood cancers Trial Population Patients Design Endpoints Status CVRM Phase I/II CD123-positive haematological 104 • Open-label, multi-centre trial • Primary endpoints: safety and • FPCD: Q1 2024 ADC123 mal

  67. Slide 67: Oncology: IPH5201

    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 57 • Open-label, dose escalation trial to determine MTD of • Primary endpoints: AE, SAE and DLT • FPCD: Q1 2020

  68. Slide 68: Oncology: NT-112

    Approved medicines Oncology NT-112 (KRAS G12D specific TCR) Late-stage development Early development Solid tumours Trial Population Patients Design Endpoints Status CVRM Phase I Unresectable, advanced and/or 24 • Open-label, single-arm, multi-centre trial with dose • Primary endpoints: incidence of

  69. Slide 69: Oncology: NT-125

    Approved medicines Oncology NT-125 (autologous, multi-specific neoantigen-targeting TCR-T) Late-stage development Early development Solid tumours Trial Population Patients Design Endpoints Status CVRM Phase I Adults with recurrent or metastatic 42 • Open-label, single-arm, single-centre trial with d

  70. Slide 70: Oncology: NT-175

    Approved medicines Oncology NT-175 (TP53-armored TCR) Late-stage development Early development Multiple cancers Trial Population Patients Design Endpoints Status CVRM Phase I Unresectable, advanced, and/​or 162 • Open-label, single-arm, multi-centre trial with dose • Primary endpoint: Incidence of D

  71. Slide 71: BioPharmaceuticals: approved medicines and late-stage development

    BioPharmaceuticals: approved medicines and late-stage development 71

  72. Slide 72: Wainua ATTR

    Approved medicines Oncology Wainua (eplontersen, ligand-conjugated antisense) Late-stage development Early development ATTR Trial Population Patients Design Endpoints Status CVRM Phase III Hereditary or wild-type 1438 • Arm 1: Wainua s.c. • Primary endpoints: composite • FPCD: Q1 2020 CARDIO-TTRansf

  73. Slide 73: balcinrenone/dapagliflozin heart failure, CKD

    Approved medicines Oncology balcinrenone/dapagliflozin (MR modulator + SGLT2 inhibitor) Late-stage development Early development Heart failure, CKD Trial Population Patients Design Endpoints Status CVRM Phase III Heart failure patients with 4800 • Randomised, double-blind, parallel-group, double-dum

  74. Slide 74: baxdrostat hypertension

    Approved medicines Oncology baxdrostat (selective aldosterone synthase inhibitor) Late-stage development Early development Hypertension Trial Population Patients Design Endpoints Status CVRM Phase III Patients with uncontrolled 720 • Arm 1: baxdrostat 1mg QD • Primary endpoint: effect of baxdrostat

  75. Slide 75: baxdrostat hypertension

    Approved medicines Oncology baxdrostat (selective aldosterone synthase inhibitor) Late-stage development Early development Hypertension Trial Population Patients Design Endpoints Status CVRM Phase II Patients with uncontrolled 175 • Arm 1: baxdrostat 2mg QD • Primary endpoints: safety and • FPCD: Q2

  76. Slide 76: baxdrostat/dapagliflozin CKD

    Approved medicines Oncology baxdrostat/dapagliflozin (selective ASI/SGLT2) Late-stage development Early development CKD/Prevention of heart failure Trial Population Patients Design Endpoints Status CVRM Phase III CKD and high blood pressure 2500 • Arm 1: baxdrostat/dapagliflozin QD • Primary endpoin

  77. Slide 77: zibotentan/dapagliflozin chronic kidney disease

    Approved medicines Oncology zibotentan/dapagliflozin (ETA receptor antagonist/SGLT2 inhibitor) Late-stage development Early development Chronic kidney disease Trial Population Patients Design Endpoints Status CVRM Phase III CKD and high proteinuria 1835 • Randomised, parallel, multi-centre, double-b

  78. Slide 78: zibotentan/dapagliflozin liver cirrhosis

    Approved medicines Oncology zibotentan/dapagliflozin (ETA receptor antagonist/SGLT2 inhibitor) Late-stage development Early development Liver cirrhosis Trial Population Patients Design Endpoints Status CVRM Phase II Part A: participants with 195 • Phase IIa/b multi-centre, randomised, double-blind,

  79. Slide 79: AZD9550 + AZD6234 obesity

    Approved medicines Oncology AZD9550 + AZD6234 (GLP-1-glucagon agonist + selective amylin agonist) Late-stage development Early development Obesity Trial Population Patients Design Endpoints Status CVRM Phase IIb Adults who are living with 360 • Randomised, parallel-group, double-blind, placebo- • Pr

  80. Slide 80: Airsupra asthma

    Approved medicines Oncology Airsupra (PT027, SABA/ICS, pMDI) Late-stage development Early development Asthma Trial Population Patients Design Endpoints Status CVRM Phase IIIb Adults and adolescents with 2517 • Randomised, double-blind, multi-centre, parallel-group, • Primary endpoint: time to first

  81. Slide 81: Breztri, Trixeo asthma

    Approved medicines Oncology Breztri, Trixeo (LAMA/LABA/ICS) Late-stage development Early development Asthma Trial Population Patients Design Endpoints Status CVRM Phase III Uncontrolled asthma 2266 • Randomised, double-blind, double-dummy, parallel group • Primary endpoint: change from • FPCD: Q1 20

  82. Slide 82: Breztri, Trixeo COPD

    Approved medicines Oncology Breztri, Trixeo (LAMA/LABA/ICS) Late-stage development Early development COPD Trial Population Patients Design Endpoints Status CVRM Phase III COPD 180 • Randomised, double-blind, three-treatment, three-period, • Primary endpoint: change from • FPCD: Q4 2023 ATHLOS crosso

  83. Slide 83: Fasenra 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 r/r EGPA on 140 • Arm 1: Fasenra 30mg Q4W s.c. • Primary endpoint: proportion of • F

  84. Slide 84: Fasenra asthma and COPD

    Approved medicines Oncology Fasenra (IL-5R mAb) Late-stage development Early development Severe, uncontrolled asthma and COPD Trial Population Patients Design Endpoints Status CVRM Phase III Patients with moderate to 689 • Double-blind, placebo-controlled trial • Primary endpoint: annualised rate of

  85. Slide 85: 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 360 • Arm 1: Saphnelo s.c. • Primary endpoint: BICLA at Week 52 • FPCD: Q3 2021 TULIP-

  86. Slide 86: Saphnelo sclerosis and myopathies

    Approved medicines Oncology Saphnelo (type I interferon receptor mAb) Late-stage development Early development Sclerosis and other myopathies Trial Population Patients Design Endpoints Status CVRM Phase III Systemic sclerosis 306 • Arm 1: Saphnelo s.c. • Primary endpoint: CRISS-25 at Week • FPCD: Q4

  87. Slide 87: Tezspire CRSwNP, COPD and EoE

    Approved medicines Oncology Tezspire (TSLP mAb) Late-stage development Early development CRSwNP, COPD and EoE Trial Population Patients Design Endpoints Status CVRM Phase III Severe chronic rhinosinusitis 416 • Arm 1: Tezspire s.c. • Co-primary endpoint: nasal polyp • FPCD: Q2 2021 WAYPOINT with nas

  88. Slide 88: Tezspire severe 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

  89. Slide 89: HFO1234ze next-generation propellant

    Approved medicines Oncology HFO1234ze (next-generation propellant) Late-stage development Early development pMDI 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 • FPC

  90. Slide 90: HFO1234ze next-generation propellant

    Approved medicines Oncology HFO1234ze (next-generation propellant) Late-stage development Early development pMDI Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers 108 • Randomised, double-blind, single-dose, single-centre, • Primary endpoints: AUCinf, AUClast • FPCD:

  91. Slide 91: 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 1132 • Randomised, double-blind, placebo-controlled, parallel- • Primary endpoint: annualised rate of • FP

  92. Slide 92: tozorakimab viral LRTD, asthma

    Approved medicines Oncology tozorakimab (IL-33 ligand mAb) Late-stage development Early development Severe viral LRTD, asthma Trial Population Patients Design Endpoints Status CVRM Phase III Adults hospitalised for viral 2870 • Randomised, double-blind, placebo-controlled, parallel • Primary endpoin

  93. Slide 93: Beyfortus infection

    Approved medicines Oncology Beyfortus (nirsevimab, RSV mAb-YTE) Late-stage development Early development Infection Trial Population Patients Design Endpoints Status CVRM Phase III Healthy infants (born 29 800 • Randomised, double-blind, placebo-controlled • Primary endpoint: efficacy • FPCD: Q4 2021

  94. Slide 94: Kavigale COVID-19

    Approved medicines Oncology Kavigale (sipavibart, SARS-CoV-2 LAAB) Late-stage development Early development COVID-19 Trial Population Patients Design Endpoints Status CVRM Phase III Phase I: healthy adults; age 18 3200 • 2 parts (Phase I: sentinel safety cohort and Phase III: main • Primary endpoint

  95. Slide 95: BioPharmaceuticals: early-stage development

    BioPharmaceuticals: early-stage development 95

  96. Slide 96: opemalirsen chronic kidney disease

    Approved medicines Oncology opemalirsen (AZD2373, APOL1) Late-stage development Early development Chronic kidney disease Trial Population Patients Design Endpoints Status CVRM Phase IIb Participants diagnosed with 96 • Randomised, multi-centre, double-blind trial in US and UK • Primary endpoint: Dos

  97. Slide 97: AZD0233 dilated cardiomyopathy

    Approved medicines Oncology AZD0233 (oral CX3CR1) Late-stage development Early development Dilated cardiomyopathy Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers 96 • Randomised, SAD/MAD dose escalating trial • Primary endpoints: safety and • FPCD: Q2 2024 NCT063814

  98. Slide 98: AZD0780 dyslipidaemia

    Approved medicines Oncology AZD0780 (PCSK9 inhibitor) Late-stage development Early development Dyslipidaemia Trial Population Patients Design Endpoints Status CVRM Phase II Dyslipidaemia 428 • Randomised trial with equal distribution across five parallel • Primary endpoint: percent change in • FPCD:

  99. Slide 99: AZD0780 dyslipidaemia

    Approved medicines Oncology AZD0780 (PCSK9 inhibitor) Late-stage development Early development Dyslipidaemia Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers 183 • Randomised, placebo-controlled SAD/MAD trial • Primary endpoints: safety and • FPCD: Q2 2022 NCT0538426

  100. Slide 100: AZD1705 dyslipidaemia

    Approved medicines Oncology AZD1705 (Angptl3 inhibitor) Late-stage development Early development Dyslipidaemia Trial Population Patients Design Endpoints Status CVRM Phase I Dyslipidaemia 112 • Part A: single dose of AZD1705 with an in-clinic period of 3 • Primary endpoints: AEs and SAEs • FPCD: Q1

  101. Slide 101: AZD2389 MASH

    Approved medicines Oncology AZD2389 (anti-fibrotic mechanism) Late-stage development Early development MASH Trial Population Patients Design Endpoints Status CVRM Phase II Participants with liver fibrosis 36 • Randomised, single-blind, placebo-controlled trial • Primary endpoints: safety and • FPCD:

  102. Slide 102: AZD2693 MASH

    Approved medicines Oncology AZD2693 (PNPLA3 ASO) Late-stage development Early development MASH Trial Population Patients Design Endpoints Status CVRM Phase IIb NASH with fibrosis 180 • Randomised, double-blind, placebo-controlled, multi-centre • Primary endpoints: efficacy, safety • FPCD: Q2 2023 FO

  103. Slide 103: AZD3427 heart failure

    Approved medicines Oncology AZD3427 (relaxin) Late-stage development Early development Heart failure Trial Population Patients Design Endpoints Status CVRM Phase II HF and pulmonary 260 • Randomised, double-blind, placebo-controlled, multi-centre • Primary endpoint: change in PVR from • FPCD: Q2 202

  104. Slide 104: AZD4144 cardiorenal disease

    Approved medicines Oncology AZD4144 (inflammation modulator) Late-stage development Early development Cardiorenal disease Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers 95 • Randomised, single-blind, placebo-controlled, SAD/MAD • Primary endpoints: safety and • FPC

  105. Slide 105: AZD5004 type 2 diabetes, obesity

    Approved medicines Oncology AZD5004 (oral GLP-1 RA) Late-stage development Early development Type 2 diabetes, obesity Trial Population Patients Design Endpoints Status CVRM Phase IIb Type 2 diabetes 384 • Arm 1: AZD5004 tablet • Primary endpoint: change in HbA1c • FPCD: Q4 2024 SOLSTICE • Arm 2: AZD

  106. Slide 106: AZD5004 type 2 diabetes, obesity

    Approved medicines Oncology AZD5004 (oral GLP-1 RA) Late-stage development Early development Type 2 diabetes, obesity Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers 31 • Part A – Arm 1: AZD5004 oral tablet • Primary endpoints (Part A): Safety and • FPCD: Q3 2024 NC

  107. Slide 107: AZD5462 heart failure

    Approved medicines Oncology AZD5462 (oral relaxin) Late-stage development Early development Heart failure Trial Population Patients Design Endpoints Status CVRM Phase IIb Stable patients with chronic 360 • Two cohort, randomised, double-blind, placebo-controlled, • Primary endpoint: change in heart

  108. Slide 108: AZD6234 obesity

    Approved medicines Oncology AZD6234 (selective amylin agonist) Late-stage development Early development Obesity with related co-morbidities Trial Population Patients Design Endpoints Status CVRM Phase II Participants living with 231 • Randomised, double-blind, placebo-controlled trial • Primary endp

  109. Slide 109: AZD9550 MASH

    Approved medicines Oncology AZD9550 (GLP-1-glucagon agonist) Late-stage development Early development MASH Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers 64 • SAD trial • Primary endpoints: safety and • FPCD: Q2 2023 NCT05848440 tolerability • LPCD: Q4 2023 • Data

  110. Slide 110: atuliflapon asthma

    Approved medicines Oncology atuliflapon (FLAP inhibitor) Late-stage development Early development Asthma Trial Population Patients Design Endpoints Status CVRM Phase IIa Patients with moderate-to- 666 • Randomised, placebo-controlled, double-blind, multi-centre • Primary endpoint: time to first • FP

  111. Slide 111: AZD0120 lupus (SLE)

    Approved medicines Oncology AZD0120 (GC012F, autologous anti-CD19 and anti-BCMA CAR-T) Late-stage development Early development Lupus (SLE) Trial Population Patients Design Endpoints Status CVRM Phase I/II Refractory systemic lupus 21 • Single-arm, open label, multi-centre trial • Primary endpoint (

  112. Slide 112: AZD1163 rheumatoid arthritis

    Approved medicines Oncology AZD1163 (anti-PAD2/4 bispecific antibody) Late-stage development Early development Rheumatoid arthritis Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers 107 • Randomised, double-blind, placebo-controlled SAD/MAD • Primary endpoint: Inciden

  113. Slide 113: AZD4604 asthma

    Approved medicines Oncology AZD4604 (inhaled JAK-1 inhibitor) Late-stage development Early development Asthma Trial Population Patients Design Endpoints Status CVRM Phase IIa Moderate-to-severe asthma 320 • Multi-centre, randomised, placebo-controlled, double- • Primary endpoint: time to first • FPC

  114. Slide 114: 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 trial • Primary endpoints: safety and • FPCD: Q4 2022 NCT056

  115. Slide 115: AZD6912 rheumatoid arthritis

    Approved medicines Oncology AZD6912 (siRNA) Late-stage development Early development Rheumatoid arthritis Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers 64 • Randomised, double-blind, placebo-controlled SAD trial • Primary endpoint: incidence of AEs • FPCD: Q4 2023

  116. Slide 116: AZD7798 Crohn's disease

    Approved medicines Oncology AZD7798 (humanised mAb) Late-stage development Early development Crohn's disease Trial Population Patients Design Endpoints Status CVRM Phase IIa Moderate to severe Crohn’s 192 • Randomised, double-blind, placebo-controlled trial • Primary endpoint: Crohn's Disease • FPCD

  117. Slide 117: AZD8630 asthma

    Approved medicines Oncology AZD8630 (inhaled TSLP) Late-stage development Early development Asthma Trial Population Patients Design Endpoints Status CVRM Phase II Adults with uncontrolled 516 • Randomised, placebo-controlled, double-blind, dose range- • Primary endpoint: time to first • FPCD: Q3 202

  118. Slide 118: AZD8965 IPF

    Approved medicines Oncology AZD8965 (arginase enzyme inhibitor) Late-stage development Early development IPF Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers 163 • Randomised, single-blind, SAD/MAD, placebo-controlled, • Primary endpoints (Part 1, 2, 3): safety • FPC

  119. Slide 119: MEDI0618 migraine prevention

    Approved medicines Oncology MEDI0618 (PAR2 antagonist mAb) Late-stage development Early development Migraine prevention Trial Population Patients Design Endpoints Status CVRM Phase II Cohort of participants failed 408 • Arm 1: CGRP-N MEDI0618 Dose A • Primary endpoint: change in number • FPCD: Q4 20

  120. Slide 120: mRNA VLP vaccine COVID-19

    Approved medicines Oncology mRNA VLP vaccine Late-stage development Early development COVID-19 Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers ≥18+ with 240 • Arm 1: dose 1 via i.m. injection AZD9838 in 18-64-year-olds • Primary endpoints: safety as measured • FPCD:

  121. Slide 121: AZD0292 bronchiectasis

    Approved medicines Oncology AZD0292 (Psl-PcrV N3Y-bispecific mAb) Late-stage development Early development Bronchiectasis Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers 32 • Randomised, single-blind, placebo-controlled trial • Primary endpoints: AEs and • FPCD: Q2

  122. Slide 122: AZD5148 Clostridium difficile

    Approved medicines Oncology AZD5148 (anti-TcdB mAb) Late-stage development Early development Clostridium difficile Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers 84 • Randomised, double-blind, placebo-controlled, dose • Primary endpoint: safety • FPCD: Q2 2024 NCT0

  123. Slide 123: AZD7760 prevention of Staph aureus infection

    Approved medicines Oncology AZD7760 (mAb combination targeting S aureus virulence factors) Late-stage development Early development Prevention of Staph aureus infection Trial Population Patients Design Endpoints Status CVRM Phase I/IIa Phase I: healthy volunteers 231 • Phase I: randomised, double-bl

  124. Slide 124: Rare Disease: approved medicines and late-stage development

    Rare Disease: approved medicines and late-stage development 124

  125. Slide 125: Beyonttra ATTR-CM

    Approved medicines Oncology Beyonttra (acoramidis, ALXN2060) Late-stage development Early development ATTR-CM Trial Population Patients Design Endpoints Status CVRM Phase III ATTR-CM 22 • Arm 1: 800mg Beyonttra administered twice daily • Primary endpoint: change from • FPCD: Q4 2020 ALXN2060-TAC-302

  126. Slide 126: Koselugo neurofibromatosis type 1

    Approved medicines Oncology Koselugo (selumetinib, MEK inhibitor) Late-stage development Early development Neurofibromatosis type 1, solid tumours Trial Population Patients Design Endpoints Status CVRM Phase III Adult age ≥18 years with NF1 145 • Multi-centre, international trial with a parallel, ra

  127. Slide 127: 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 Thrombotic microangiopathy- 106 • Arm 1: Ultomiris Q8W • Primary endpoint: event-free survival • FPCD: Q4 2020 ALXN121

  128. Slide 128: Ultomiris neurology

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

  129. Slide 129: 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 AL amyloidosis (Mayo Stage 124 • Arm 1: anselamimab combined with SoC for PCD • Primary endpoint: a hierarc

  130. Slide 130: efzimfotase alfa hypophosphatasia

    Approved medicines Oncology efzimfotase alfa (ALXN1850, next-generation asfotase alfa) Late-stage development Early development Hypophosphatasia Trial Population Patients Design Endpoints Status CVRM Phase III Hypophosphatasia 114 • Arm 1: placebo on Day 1 followed by Q2W via s.c. injection • Primar

  131. Slide 131: eneboparatide hypoparathyroidism

    Approved medicines Oncology eneboparatide (parathyroid hormone receptor 1 agonist) Late-stage development Early development Hypoparathyroidism Trial Population Patients Design Endpoints Status CVRM Phase III Chronic hypoparathyroidism 165 • Arm 1: 20mcg eneboparatide administered once daily via s.c.

  132. Slide 132: gefurulimab neurology, nephrology

    Approved medicines Oncology gefurulimab (ALXN1720, anti-C5 dual-binding nanobody) Late-stage development Early development Neurology, nephrology Trial Population Patients Design Endpoints Status CVRM Phase III Generalised myasthenia 254 • Arm 1: weight-based maintenance treatment with • Primary endp

  133. Slide 133: ALXN2220 amyloidosis

    Approved medicines Oncology ALXN2220 (NI006, TTR depleter) Late-stage development Early development Amyloidosis Trial Population Patients Design Endpoints Status CVRM Phase III ATTR-CM 1000 • Arm 1: ALXN2220 via i.v. infusion Q4W for at least 24 months • Primary endpoints: all-cause mortality • FPCD

  134. Slide 134: Rare Disease: early-stage development

    Rare Disease: early-stage development 134

  135. Slide 135: ALXN1920 nephrology

    Approved medicines Oncology ALXN1920 (kidney-targeted factor H fusion protein) Late-stage development Early development Nephrology Trial Population Patients Design Endpoints Status CVRM Phase I Healthy adults 48 • Randomised, double-blind, placebo-controlled SAD trial • Primary endpoints: safety and

  136. Slide 136: 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 trial • Primary endpoint: safety • FPCD: Q4 2022 ALXN2030

  137. Slide 137: ALXN2080 complement-mediated disease

    Approved medicines Oncology ALXN2080 (factor D inhibitor) Late-stage development Early development Complement-mediated disease Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers 90 • SAD/MAD trial • Primary endpoints: safety and • FPCD: Q3 2022 ALXN2080-HV-101 tolerabi

  138. Slide 138: 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 trial • Primary endpoints: efficac

  139. Slide 139: Glossary – 1 of 5

    NEXT NEXT Glossary – 1 of 5 14C Carbon 14 ASO Antisense oligonucleotide BTK Bruton's tyrosine kinase 1L, 2L, 3L 1st-, 2nd- or 3rd-line ATM Ataxia telangiectasia mutated kinase BTKi Bruton's tyrosine kinase 5-FU 5-fluorouracil ATR Ataxia telangiectasia and Rad3-related protein BVAS Birmingham Vasculi

  140. Slide 140: Glossary – 2 of 5

    BACK NEXT BACK NEXT Glossary – 2 of 5 CNS Central nervous system DNA Deoxyribonucleic acid ETA Endothelin A CNS-PFS Central nervous system progression-free survial dNCC Directly measured non-ceruloplasmin-bound copper ETA RA Endothelin receptor A antagonist CompEx Composite endpoint for exacerbation

  141. Slide 141: Glossary – 3 of 5

    BACK NEXT BACK NEXT Glossary – 3 of 5 GPRC5D G protein-coupled receptor, class C, group 5, member D HSD17B13 Hydroxysteroid 17-beta dehydrogenase 13 LA amylin Long-acting amylin GU Genitourinary HVPG Hepatic venous pressure gradient LAAB Long-acting antibody GYN Gynaecologic i Inhibitor LABA Long-ac

  142. Slide 142: Glossary – 4 of 5

    BACK NEXT BACK NEXT Glossary – 4 of 5 MI Myocardial infarction NME New molecular entity PFS Progression-free survival mL Millilitre NMOSD Neuromyelitis optica spectrum disorder PFS2 Time to second disease progression or death MM Multiple myeloma NP Nasal polyps PgR Progesterone receptor MMAE Monomet

  143. Slide 143: Glossary – 5 of 5

    BACK BACK Glossary – 5 of 5 QD Once daily SGLT2 Sodium-glucose transport protein 2 Tmax Time to reach maximum observed plasma concentration QID Four times per day SGLT2i Sodium/glucose cotransporter 2 inhibitor TNBC Triple negative breast cancer QOD Every other day SGRM Selective glucocorticoid rece

AstraZeneca — Q1 2025 Earnings Presentation on 6ix