Slide 1: Clinical Trials Appendix
Clinical Trials Appendix Q1 2026 Results Update 29 April 2026
Slide 2: Pipeline at a glance
Pipeline 186 21 at a glance projects in our new molecular entities development pipeline (NME) in our late-stage pipeline Across five focus therapy areas: Oncology BioPharmaceuticals 118 14 CVRM | R&I | Infectious Disease new molecular entities regulatory approvals (NME) or major lifecycle in major m
Slide 3: Key upcoming pipeline catalysts: 2026 and 2027
Key upcoming pipeline catalysts: 2026 and 2027 Oncology BioPharmaceuticals Rare Disease H1 2026 H2 2026 2027 Enhertu | neoadjuvant HER2+ Stage II or III breast cancer | DESTINY-Breast11 Calquence | CLL (1L fixed duration) | AMPLIFY (JP) Calquence | CLL (1L fixed duration) | AMPLIFY (CN) (US) Enhertu
Slide 4: Clinical Trials Appendix: selected highlights
Clinical Trials Appendix: selected highlights Oncology BioPharmaceuticals Rare Disease Oncology BioPharmaceuticals Rare Disease Calquence Farxiga Ultomiris Calquence Farxiga Ultomiris Datroway Airsupra Koselugo Datroway Airsupra Koselugo Enhertu Breztri Approved Enhertu Breztri Imfinzi/Imjudo Fasenr
Slide 5: Project movements since Q4 2025 update
Project movements since Q4 2025 update New to Phase I New to Phase II New to pivotal trial New to registration NME NME NME Life-cycle management ALXN2230 AZD4144 AZD0120 DURGA-4 Enhertu DESTINY-Breast05# healthy participants NLRP3 cardiorenal disease CD19/BCMA CAR-T relapsed/refractory HER2 TOP1i AD
Slide 6: Q1 2026 Oncology new molecular entity pipeline
Q1 2026 Oncology new molecular entity1 pipeline Phase I Phase II Phase III 23 New Molecular Entities 19 New Molecular Entities 25 New Molecular Entities surovatamig surovatamig SOUNDTRACK-E Imfinzi +/- oleclumab +/- monalizumab PACIFIC-9# camizestrant + palbociclib SERENA-4 CD19/CD3 TCE r/r B-cell n
Slide 7: Q1 2026 Oncology lifecycle management pipeline
Q1 2026 Oncology lifecycle management1 pipeline Phase I Phase II Phase III Under review 0 Projects 4 Projects 28 Projects 5 Projects Datroway TROPION-Lung17 # Datroway + Imfinzi + CTx AVANZAR# Datroway TROPION-Bre ast02# Enhertu (platform) DESTINY-Bre ast07# Calquence + R-CHOP ESCALADE TROP2 TOP1i A
Slide 8: Q1 2026 BioPharmaceuticals new molecular entity pipeline
Q1 2026 BioPharmaceuticals new molecular entity1 pipeline Phase I Phase II Phase III Under review 12 New Molecular Entities 17 New Molecular Entities 8 New Molecular Entities 1 New Molecular Entity surovatamig balcinrenone/dapagliflozin elecoglipron balcinrenone/dapagliflozin baxdrostat BaxHTN Bax24
Slide 9: Q1 2026 BioPharmaceuticals life cycle management pipeline
Q1 2026 BioPharmaceuticals life cycle management1 pipeline Phase I Phase II Phase III Under review 0 Projects 0 Projects 8 Projects 1 Project Breztri/Trixeo THARROS# Fasenra NATRON LABA/LAMA/ICS cardiopulmonary outcomes trial in IL-5R mAb hypereosinophilic syndrome COPD Saphnelo DAISY# type I IFN re
Slide 10: Q1 2026 Rare Disease pipeline
Q1 2026 Rare Disease pipeline1 Phase I Phase II Phase III Under review 5 Projects 3 Projects 6 Projects 2 Projects tarperprumig I TRANSCEND ALXN2080 anti-properdin (complement factor P), dual-binding, cliramitug DepleTTR-CM# anselamimab CARES oral factor D healthy participants TTR depleter transthyr
Slide 11: Active designations in our pipeline
Active designations in our pipeline 3 6 12 3 18 Priority Review Breakthrough / PRIME 1 / Sakigake 2 Fast Track Qualified Infectious Disease product Orphan baxdrostat HTN (US) AZD0292 Psl-PcrV N3Y NCFBE (EU) AZD0292 Psl-PcrV N3Y NCFBE (US) AZD0292 Psl-PcrV N3Y NCFBE (US) Fasenra HES NATRON (US) Datro
Slide 12: Oncology
Oncology Approved medicines and late-stage pipeline 12 12
Slide 13: Calquence (BTK inhibitor)
Approved medicines Late-stage development Calquence (BTK inhibitor) Oncology Early development Blood cancers Trial Population Patients Design Endpoints Status Phase III Previously untreated CLL 981 • Arm 1: Calquence + venetoclax • Primary endpoint: PFS (IRC) (Arm 1 vs. • FPCD: Q1 2019 AMPLIFY (ACE-
Slide 14: Datroway: breast cancer
Approved medicines Late-stage development Datroway (datopotamab deruxtecan, TROP2 ADC) Oncology Early development Breast cancer Trial Population Patients Design Endpoints Status Phase III Locally recurrent inoperable or 600 • Open-label, randomised trial • Primary endpoints: PFS (BICR) and OS • FPCD
Slide 15: Datroway: NSCLC
Approved medicines Late-stage development Datroway (datopotamab deruxtecan, TROP2 ADC) Oncology Early development NSCLC Trial Population Patients Design Endpoints Status Phase III 1L locally advanced/metastatic NSCLC 1350 • Randomised, open-label • Co-primary endpoints: PFS and OS in • FPCD: Q1 2023
Slide 16: Datroway: NSCLC
Approved medicines Late-stage development Datroway (datopotamab deruxtecan, TROP2 ADC) Oncology Early development NSCLC Trial Population Patients Design Endpoints Status Phase I Advanced or metastatic NSCLC 145 • Open-label, two-part (dose escalation and dose expansion), • Primary endpoints: DLT and
Slide 17: Datroway: other cancers
Approved medicines Late-stage development Datroway (datopotamab deruxtecan, TROP2 ADC) Oncology Early development Other cancers Trial Population Patients Design Endpoints Status Phase II Endometrial cancer, gastric cancer, 606 • Sub-study 1 (endometrial cancer); • Primary endpoints: ORR and safety •
Slide 18: Datroway: other cancers
Approved medicines Late-stage development Datroway (datopotamab deruxtecan, TROP2 ADC) Oncology Early development Other cancers Trial Population Patients Design Endpoints Status Phase I/II NSCLC and TNBC and other solid 119 • Single-arm, multi-cohort trial with no blinding • Primary endpoint: ORR •
Slide 19: Enhertu: breast cancer
Approved medicines Late-stage development Enhertu (trastuzumab deruxtecan, HER2 ADC) Oncology Early development Breast cancer Trial Population Patients Design Endpoints Status Phase III High-risk HER2-positive with residual 1600 • Randomised, open-label, parallel assignment • Primary endpoint: IDFS
Slide 20: Enhertu: gastric cancer
Approved medicines Late-stage development Enhertu (trastuzumab deruxtecan, HER2 ADC) Oncology Early development Gastric cancer Trial Population Patients Design Endpoints Status Phase III HER2-positive gastric cancer or GEJ 490 • Open-label, randomised, parallel group assignment • Primary endpoint: O
Slide 21: Enhertu: other cancers
Approved medicines Late-stage development Enhertu (trastuzumab deruxtecan, HER2 ADC) Oncology Early development Other cancers Trial Population Patients Design Endpoints Status Phase III Advanced treatment-naïve HER2- 620 • Arm A: Enhertu + rilvegostomig • Primary endpoint: OS • FPCD: Q3 2024 DESTINY
Slide 22: Enhertu: other cancers
Approved medicines Late-stage development Enhertu (trastuzumab deruxtecan, HER2 ADC) Oncology Early development Other cancers Trial Population Patients Design Endpoints Status Phase II HER2 expressing tumours 125 • Non-randomised single group assignment • Primary endpoint: ORR • FPCD: Q3 2024 DESTIN
Slide 23: Imfinzi: gastrointestinal cancers
Approved medicines Late-stage development Imfinzi (durvalumab, PD-L1 mAb) Oncology Early development Gastrointestinal cancers Trial Population Patients Design Endpoints Status Phase III Locoregional HCC 710 • Arm 1: TACE in combination with Imfinzi • Primary endpoint: PFS (Arm 2 vs. Arm • FPCD: Q1 2
Slide 24: Imfinzi: lung cancer
Approved medicines Late-stage development Imfinzi (durvalumab, PD-L1 mAb) Oncology Early development Lung cancer Trial Population Patients Design Endpoints Status Phase III Unresected Stage I/II NSCLC 662 • Arm 1: Imfinzi i.v. Q4W with definitive SBRT • Primary endpoint: PFS • FPCD: Q2 2019 PACIFIC-
Slide 25: Imfinzi: other cancers
Approved medicines Late-stage development Imfinzi (durvalumab, PD-L1 mAb) Oncology Early development Other cancers Trial Population Patients Design Endpoints Status Phase III Muscle-invasive bladder cancer eligible 1063 • Arm 1: Imfinzi in combination with gemcitabine + cisplatin, • Co-primary endpo
Slide 26: Lynparza: ovarian, endometrial and breast cancers
Approved medicines Late-stage development Lynparza (PARP inhibitor) Oncology Early development Ovarian, endometrial and breast cancers Trial Population Patients Design Endpoints Status Phase III BRCAwt advanced ovarian cancer, 1L 366 • Global trial – 12 countries • Primary endpoints: PFS (BRCAwt HRD
Slide 27: Orpathys: gastric cancer
Approved medicines Late-stage development Orpathys (savolitinib, MET inhibitor) Oncology Early development Gastric cancer Trial Population Patients Design Endpoints Status Phase II Locally advanced or metastatic gastric 75 • Single-arm, multi-cohort, multi-centre, open-label trial • Primary endpoint
Slide 28: Tagrisso: NSCLC
Approved medicines Late-stage development Tagrisso (highly-selective, irreversible EGFR inhibitor) Oncology Early development NSCLC Trial Population Patients Design Endpoints Status Phase III Adjuvant EGFRm NSCLC Stage IA2 to IA3 380 • Arm 1: Tagrisso • Primary endpoint: DFS • FPCD: Q2 2022 ADAURA2
Slide 29: Truqap: breast and prostate cancer
Approved medicines Late-stage development Truqap (capivasertib, AKT inhibitor) Oncology Early development Breast cancer and prostate cancer Trial Population Patients Design Endpoints Status Phase III De novo PTEN deficient metastatic 1012 • Double-blind, randomised, comparative trial • Primary endpo
Slide 30: camizestrant: breast cancer
Approved medicines Late-stage development camizestrant (next-generation oral SERD) Oncology Early development Breast cancer Trial Population Patients Design Endpoints Status Phase III ER+/HER2-negative early breast cancer 4300 • Arm 1: camizestrant • Primary endpoint: IBCFS • FPCD: Q1 2023 CAMBRIA-1
Slide 31: puxitatug samrotecan: solid tumours
Approved medicines Late-stage development puxitatug samrotecan (AZD8205, B7H4 TOP1i ADC) Oncology Early development Solid tumours Trial Population Patients Design Endpoints Status Phase III 2-3L B7-H4 selected endometrial cancer 700 • Randomised, single-open label • Primary endpoints: PFS, OS • FPCD
Slide 32: rilvegostomig: gastrointestinal cancers
Approved medicines Late-stage development rilvegostomig (PD-1/TIGIT bispecific mAb) Oncology Early development Gastrointestinal cancers Trial Population Patients Design Endpoints Status Phase III Adjuvant BTC with curative intent 750 • Randomised, double-blind, placebo-controlled, multicentre • Prim
Slide 33: rilvegostomig: lung cancer
Approved medicines Late-stage development rilvegostomig (PD-1/TIGIT bispecific mAb) Oncology Early development Lung cancer Trial Population Patients Design Endpoints Status Phase III 1L PD-L1 TC ≥1% squamous NSCLC 880 • Randomised, double-blind, multicentre • Primary endpoints: PFS, OS • FPCD: Q4 20
Slide 34: rilvegostomig: lung cancer
Approved medicines Late-stage development rilvegostomig (PD-1/TIGIT bispecific mAb) Oncology Early development Lung cancer Trial Population Patients Design Endpoints Status Phase I/II Advanced or metastatic NSCLC 116 • Part A (safety run in): rilvegostomig + platinum-based CTx • Primary endpoints: s
Slide 35: rilvegostomig: solid tumours
Approved medicines Late-stage development rilvegostomig (PD-1/TIGIT bispecific mAb) Oncology Early development Solid tumours Trial Population Patients Design Endpoints Status Phase IIb HCC, BTC 294 • Open-label platform trial • Primary endpoints (HCC sub-study): • FPCD: Q2 2023 GEMINI-Hepatobiliary
Slide 36: saruparib: solid tumours
Approved medicines Late-stage development saruparib (AZD5305, PARP1 inhibitor) Oncology Early development Solid tumours Trial Population Patients Design Endpoints Status Phase III BRCA1, BRCA2, or PALB2m, HR-positive, 500 • Randomised, open-label trial • Primary endpoint: PFS (BICR) • FPCD: Q3 2024
Slide 37: saruparib: solid tumours
Approved medicines Late-stage development saruparib (AZD5305, PARP1 inhibitor) Oncology Early development Solid tumours Trial Population Patients Design Endpoints Status Phase I Newly diagnosed prostate cancer 120 • Open-label, randomised, multi-centre trial • Primary endpoint: effects of • FPCD: Q3
Slide 38: sonesitatug vedotin: solid tumours
Approved medicines Late-stage development sonesitatug vedotin (AZD0901, CLDN18.2 MMAE ADC) Oncology Early development Solid tumours Trial Population Patients Design Endpoints Status Phase III 2L+ advanced or metastatic gastric or 572 • Multi-centre, open-label, sponsor-blinded, randomised trial • Pr
Slide 39: surovatamig: haematologic malignancies
Approved medicines Late-stage development surovatamig (CD19/CD3 T-cell engager) Oncology Early development Haematologic malignancies Trial Population Patients Design Endpoints Status Phase III B-cell non-Hodgkin lymphoma, diffuse 420 • Multi-centre, randomised • Primary: PFS • Data anticipated: >202
Slide 40: torvutatug samrotecan: ovarian cancer, solid tumours
Approved medicines Late-stage development torvutatug samrotecan (AZD5335, anti-FRα TOP1i ADC) Oncology Early development Ovarian cancer, solid tumours Trial Population Patients Design Endpoints Status Phase III Previously treated FRα-expressing 1100 • Randomised, open-label • Primary endpoint: PFS
Slide 41: volrustomig: solid tumours
Approved medicines Late-stage development volrustomig (PD-1/CTLA-4 bispecific mAb) Oncology Early development Solid tumours Trial Population Patients Design Endpoints Status Phase III High-risk locally advanced cervical 800 • Randomised, double-blind, placebo-controlled, multi-centre • Primary endpo
Slide 42: volrustomig: solid tumours
Approved medicines Late-stage development volrustomig (PD-1/CTLA-4 bispecific mAb) Oncology Early development Solid tumours Trial Population Patients Design Endpoints Status Phase II Advanced/metastatic solid tumours 257 • Platform, multicentre trial • Primary endpoints: ORR and safety • FPCD: Q3 20
Slide 43: AZD0120: blood cancers
Approved medicines Late-stage development AZD0120 (autologous anti-CD19 and anti-BCMA CAR-T) Oncology Early development Blood cancers Trial Population Patients Design Endpoints Status Phase III Relapsed/refractory multiple myeloma 508 • Randomised, multicentre, controlled, open-label, global • Prima
Slide 44: Oncology: Early-stage development
Oncology Early-stage development 44 44
Slide 45: IPH5201 (CD39 mAb)
Approved medicines Late-stage development IPH5201 (CD39 mAb) Oncology Early development Solid tumours Trial Population Patients Design Endpoints Status Phase I Advanced solid tumours 57 • Open-label, dose escalation trial to determine MTD of • Primary endpoints: AE, SAE and DLT • FPCD: Q1 2020 NCT04
Slide 46: NT-112 (KRAS G12D specific TCR-T)
Approved medicines Late-stage development NT-112 (KRAS G12D specific TCR-T) Oncology Early development Solid tumours Trial Population Patients Design Endpoints Status Phase I Unresectable, advanced/metastatic 24 • Open-label, single-arm, multi-centre trial with dose • Primary endpoints: incidence of
Slide 47: NT-175 (TP53 R175H armoured TCR-T)
Approved medicines Late-stage development NT-175 (TP53 R175H armoured TCR-T) Oncology Early development Solid tumours Trial Population Patients Design Endpoints Status Phase I Unresectable, advanced, and/or 45 • Open-label, single-arm, multi-centre trial with dose • Primary endpoint: Incidence of D
Slide 48: tilatamig samrotecan
Approved medicines Late-stage development tilatamig samrotecan (EGFR-cMET TOP1i ADC) Oncology Early development Lung cancer Trial Population Patients Design Endpoints Status Phase I Advanced solid tumours including 403 • Escalation phase, open-label, multi-centre trial • Primary endpoints (escalatio
Slide 49: vemzatatug vedotin
Approved medicines Late-stage development vemzatatug vedotin (AZD0305, GPRC5D MMAE ADC) Oncology Early development Blood cancers Trial Population Patients Design Endpoints Status Phase I/II R/R multiple myeloma 226 • Open-label, dose escalation and dose expansion trial • Primary endpoints: occurrenc
Slide 50: AZD0516 (STEAP2 ADC)
Approved medicines Late-stage development AZD0516 (STEAP2 ADC) Oncology Early development Prostate cancer Trial Population Patients Design Endpoints Status Phase I/II mCRPC 177 • Open-label multi-centre, modular dose escalation and dose • Primary endpoint: safety and • FPCD: Q4 2025 SEACLIFF optimis
Slide 51: AZD0754 (STEAP2 CAR-T)
Approved medicines Late-stage development AZD0754 (STEAP2 dnTGFβRII-armoured CAR-T) Oncology Early development Solid tumours Trial Population Patients Design Endpoints Status Phase I/II mCRPC with prior NHA and taxane 60 • Open-label, single-arm, multi-centre trial with dose • Primary endpoints (Pha
Slide 52: AZD2068 (EGFR cMET radioconjugate)
Approved medicines Late-stage development AZD2068 (FPI-2068, EGFR cMET radioconjugate) Oncology Early development Solid tumours Trial Population Patients Design Endpoints Status Phase I Advanced solid tumours 110 • Multicentre, open-label dose escalation trial • Primary endpoints: safety and • FPCD:
Slide 53: zadavotide guraxetan (AZD2265)
Approved medicines Late-stage development (225Ac) zadavotide guraxetan (AZD2265, PSMA radioconjugate) Oncology Early development Prostate cancer Trial Population Patients Design Endpoints Status Phase II PSMA-positive mCRPC previously 100 • Open-label, randomised, multi-centre trial • Primary endpoi
Slide 54: AZD2284 (STEAP2 radioconjugate)
Approved medicines Late-stage development AZD2284 (STEAP2 radioconjugate) Oncology Early development Prostate cancer Trial Population Patients Design Endpoints Status Phase I mCRPC 134 • Part A (imaging): • Primary endpoints: safety and • FPCD: Q1 2025 NCT06879041 • Part A (cold antibody exploration
Slide 55: AZD2962 (IRAK4 inhibitor)
Approved medicines Late-stage development AZD2962 (IRAK4 inhibitor) Oncology Early development Blood cancers Trial Population Patients Design Endpoints Status Phase I Haematologic neoplasms 72 • Modular, open-label, multicentre • Primary endpoints: DLT, AEs, duration • Data anticipated: >2027 NCT070
Slide 56: AZD3470 (PRMT5 inhibitor)
Approved medicines Late-stage development AZD3470 (PRMT5 inhibitor) Oncology Early development Solid tumours and blood cancers Trial Population Patients Design Endpoints Status Phase I/II MTAP-deficient advanced solid tumours 234 • Open-label, multicentre • Arm 1: Primary endpoints: safety and • FPC
Slide 57: AZD3632 (menin inhibitor)
Approved medicines Late-stage development AZD3632 (menin inhibitor) Oncology Early development Blood cancers Trial Population Patients Design Endpoints Status Phase I/II R/R AML, ALL and HR MDS with 84 • Module 1 (dose escalation): AZD3632 • Primary endpoints: safety and • FPCD: Q1 2026 MOMENTUM KMT
Slide 58: AZD4360 (CLDN18.2 TOP1i ADC)
Approved medicines Late-stage development AZD4360 (CLDN18.2 TOP1i ADC) Oncology Early development Solid tumours Trial Population Patients Design Endpoints Status Phase I/II Advanced or metastatic Claudin 18.2- 117 • Open-label, multicentre trial with FIH modular protocol • Primary endpoints: safety
Slide 59: AZD4512 (CD22 TOP1i ADC)
Approved medicines Late-stage development AZD4512 (CD22 TOP1i ADC) Oncology Early development Blood cancers Trial Population Patients Design Endpoints Status Phase I/II Acute Lymphoblastic Leukemia 83 • Modular phase I/II, open-label multi-centre study • Module 1: • FPCD: Q4 2025 ALLight • Module 1
Slide 60: AZD4956 (POLQ inhibitor)
Approved medicines Late-stage development AZD4956 (POLQ inhibitor) Oncology Early development Solid tumours Trial Population Patients Design Endpoints Status Phase I/II HRRm solid tumours 180 • Module 1: AZD4956 monotherapy • Primary endpoints: safety and • FPCD: Q1 2026 PARTHENON • Module 2: AZD495
Slide 61: AZD5492 (CD20 TITAN TCE)
Approved medicines Late-stage development AZD5492 (CD20 TITAN TCE) Oncology Early development Blood cancers Trial Population Patients Design Endpoints Status Phase I Relapsed/refractory B-Cell malignancies 176 • Module 1: AZD5492 monotherapy • Primary endpoints: safety and • FPCD: Q3 2024 TITANIUM t
Slide 62: AZD5863 (CLDN18.2 CD3 bispecific)
Approved medicines Late-stage development AZD5863 (CLDN18.2 CD3 bispecific antibody) Oncology Early development Solid tumours Trial Population Patients Design Endpoints Status Phase I Advanced or metastatic Claudin 18.2- 280 • Part A: AZD5863 dose escalation • Primary endpoints (Part A): safety and
Slide 63: AZD6621 (STEAP2 T-cell engager)
Approved medicines Late-stage development AZD6621 (STEAP2 T-cell engager) Oncology Early development Prostate cancer Trial Population Patients Design Endpoints Status Phase I/II mCRPC 52 • Open-label, multi-centre, modular design • Primary: safety, tolerability • FPCD: Q4 2025 ACTIVATED-4-PC • Part
Slide 64: AZD6750 (CD8 guided IL-2)
Approved medicines Late-stage development AZD6750 (CD8 guided IL-2) Oncology Early development Solid tumours Trial Population Patients Design Endpoints Status Phase I/II Select advanced or metastatic solid 60 • Open-label, dose escalation and expansion study • Primary endpoints: Safety and efficacy
Slide 65: AZD7003 (GPC3 CAR-T)
Approved medicines Late-stage development AZD7003 (GPC3 CAR-T) Oncology Early development Hepatocellular carcinoma (HCC) Trial Population Patients Design Endpoints Status Phase I/II GPC3-positive advanced/recurrent HCC 121 • Open-label, single-arm, multicentre trial • Primary endpoints (Part A): DLT
Slide 66: AZD8421 (CDK2 inhibitor)
Approved medicines Late-stage development AZD8421 (CDK2 inhibitor) Oncology Early development Breast cancer Trial Population Patients Design Endpoints Status Phase I/II ER+ HER2-negative advanced breast 204 • Module 1: AZD8421 • Primary endpoints: safety and • FPCD: Q4 2023 CYCAD-1 cancer • Module 2
Slide 67: palacaparib (AZD9574)
Approved medicines Late-stage development palacaparib (AZD9574, PARP1-sel BBB inhibitor) Oncology Early development Solid tumours Trial Population Patients Design Endpoints Status Phase I/IIa Advanced solid malignancies 695 • Modular, open-label, multi-centre dose escalation and • Primary endpoints:
Slide 68: AZD9750 (AR PROTAC)
Approved medicines Late-stage development AZD9750 (AR PROTAC) Oncology Early development Prostate cancer Trial Population Patients Design Endpoints Status Phase I/II Metastatic prostate cancer 300 • Open-label, multicenter • Primary endpoints: safety and • FPCD: Q1 2026 ANDROMEDA • Module 1, Part A
Slide 69: AZD9793 (GPC3 TITAN T-cell engager)
Approved medicines Late-stage development AZD9793 (GPC3 TITAN T-cell engager) Oncology Early development Solid tumours Trial Population Patients Design Endpoints Status Phase I/II Metastatic solid tumours 304 • Open label, non randomised, multi-centre, dose escalation • Primary endpoint: safety and
Slide 70: BioPharmaceuticals
BioPharmaceuticals Approved medicines and late-stage pipeline 70 70
Slide 71: Wainua (eplontersen)
Approved medicines Late-stage development Wainua (eplontersen, ligand-conjugated antisense) Oncology Early development ATTR Trial Population Patients Design Endpoints Status Phase III Hereditary or wild-type 1438 • Arm 1: Wainua s.c. • Primary endpoints: composite • FPCD: Q1 2020 CARDIO-TTRansform t
Slide 72: balcinrenone/dapagliflozin
Approved medicines Late-stage development balcinrenone/dapagliflozin (MR antagonist/modulator + SGLT2 inhibitor) Oncology Early development Heart failure, CKD Trial Population Patients Design Endpoints Status Phase III Heart failure patients with 4800 • Randomised, double-blind, parallel-group, doub
Slide 73: baxdrostat: hypertension
Approved medicines Late-stage development baxdrostat (selective aldosterone synthase inhibitor) Oncology Early development Hypertension Trial Population Patients Design Endpoints Status Phase III Patients with resistant 218 • Arm 1: baxdrostat 2mg QD • Primary endpoint: effect of baxdrostat • FPCD:
Slide 74: baxdrostat: hypertension, primary aldosteronism
Approved medicines Late-stage development baxdrostat (selective aldosterone synthase inhibitor) Oncology Early development Hypertension, primary aldosteronism Trial Population Patients Design Endpoints Status Phase II Patients with uncontrolled 45 • Arm 1: baxdrostat 2mg QD • Primary endpoint: indiv
Slide 75: baxdrostat/dapagliflozin
Approved medicines Late-stage development baxdrostat/dapagliflozin (selective ASI/SGLT2) Oncology Early development CKD/Prevention of heart failure Trial Population Patients Design Endpoints Status Phase III CKD and high blood pressure 2500 • Arm 1: baxdrostat/dapagliflozin QD • Primary endpoint: ch
Slide 76: laroprovstat (AZD0780)
Approved medicines Late-stage development laroprovstat (AZD0780, PCSK9 inhibitor) Oncology Early development Dyslipidaemia Trial Population Patients Design Endpoints Status Phase III Heterozygos familial 405 • Randomised, double-blind, placebo-controlled, parallel- • Primary endpoint: Relative chang
Slide 77: laroprovstat (AZD0780)
Approved medicines Late-stage development laroprovstat (AZD0780, PCSK9 inhibitor) Oncology Early development Dyslipidaemia Trial Population Patients Design Endpoints Status Phase II Dyslipidaemia 428 • Randomised trial with equal distribution across five parallel • Primary endpoint: percent change i
Slide 78: laroprovstat (AZD0780)
Approved medicines Late-stage development laroprovstat (AZD0780, PCSK9 inhibitor) Oncology Early development Dyslipidaemia Trial Population Patients Design Endpoints Status Phase I Healthy volunteers 40 • Phase I, randomised, single-blind, placebo-controlled study • Primary endpoint: PK measures • F
Slide 79: laroprovstat + rosuvastatin
Approved medicines Late-stage development laroprovstat + rosuvastatin (PCSK9 inhibitor + HMG-CoA reductase inhibitor) Oncology Early development Dyslipidaemia Trial Population Patients Design Endpoints Status Phase I Healthy adults 44 • Randomised, open-label, 3 or 4-period, single-dose, two- • Prim
Slide 80: zibotentan/dapagliflozin
Approved medicines Late-stage development zibotentan/dapagliflozin (ETA receptor antagonist/SGLT2 inhibitor) Oncology Early development Chronic kidney disease Trial Population Patients Design Endpoints Status Phase III CKD and high proteinuria 1835 • Randomised, parallel, multi-centre, double-blind
Slide 81: Airsupra (PT027)
Approved medicines Late-stage development Airsupra (PT027, SABA/ICS, pMDI) Oncology Early development Asthma Trial Population Patients Design Endpoints Status Phase IIIb Adolescents with asthma 440 • Randomised, double-blind, multi-centre, parallel-group • Primary endpoint: severe asthma • FPCD: Q2
Slide 82: Breztri, Trixeo: asthma
Approved medicines Late-stage development Breztri, Trixeo (LAMA/LABA/ICS) Oncology Early development Asthma Trial Population Patients Design Endpoints Status Phase III Uncontrolled asthma 2266 • Randomised, double-blind, double-dummy, parallel group • Primary endpoint: change from • FPCD: Q1 2021 KA
Slide 83: Breztri, Trixeo: COPD
Approved medicines Late-stage development Breztri, Trixeo (LAMA/LABA/ICS) Oncology Early development COPD Trial Population Patients Design Endpoints Status Phase III COPD 180 • Randomised, double-blind, three-treatment, three-period, • Primary endpoint: change from • FPCD: Q4 2023 ATHLOS crossover t
Slide 84: Fasenra (IL-5R mAb)
Approved medicines Late-stage development Fasenra (IL-5R mAb) Oncology Early development Other eosinophilic diseases Trial Population Patients Design Endpoints Status Phase III Patients with r/r EGPA on 140 • Arm 1: Fasenra 30mg Q4W s.c. • Primary endpoint: proportion of • FPCD: Q4 2019 MANDARA cort
Slide 85: Saphnelo (type I interferon receptor mAb)
Approved medicines Late-stage development Saphnelo (type I interferon receptor mAb) Oncology Early development Lupus (SLE/LN), sclerosis and other myopathies Trial Population Patients Design Endpoints Status Phase III Moderate to severe SLE 276 • Randomised, double-blind, placebo-controlled • Primar
Slide 86: Tezspire (TSLP mAb)
Approved medicines Late-stage development Tezspire (TSLP mAb) Oncology Early development CRSwNP, COPD, EoE and severe, uncontrolled asthma Trial Population Patients Design Endpoints Status Phase III Adult and paediatric aged 12 360 • Arm 1: Tezspire s.c. low dose • Co-primary endpoints: histologic •
Slide 87: tozorakimab: COPD
Approved medicines Late-stage development tozorakimab (IL-33 ligand mAb) Oncology Early development COPD Trial Population Patients Design Endpoints Status Phase III Adults with symptomatic 1132 • Randomised, double-blind, placebo-controlled, parallel- • Primary endpoint: annualised rate of • FPCD: Q
Slide 88: tozorakimab: COPD
Approved medicines Late-stage development tozorakimab (IL-33 ligand mAb) Oncology Early development COPD Trial Population Patients Design Endpoints Status Phase III Adults with symptomatic 1454 • Randomised, double-blind, placebo-controlled, parallel • Primary endpoint: annualised rate of • FPCD: Q4
Slide 89: tozorakimab: severe viral LRTD, asthma
Approved medicines Late-stage development tozorakimab (IL-33 ligand mAb) Oncology Early development Severe viral LRTD, asthma Trial Population Patients Design Endpoints Status Phase III Adults hospitalised for viral 2870 • Randomised, double-blind, placebo-controlled, parallel • Primary endpoint: pr
Slide 90: Next-generation propellant pMDI
Approved medicines Late-stage development Next-generation propellant Oncology Early development pMDI Trial Population Patients Design Endpoints Status Phase III Mucociliary clearance in 30 • Randomised, double-blind, multi-site, two-way crossover • Primary endpoint: change from • FPCD: Q2 2023 NCT05
Slide 91: Next-generation propellant pMDI
Approved medicines Late-stage development Next-generation propellant Oncology Early development pMDI Trial Population Patients Design Endpoints Status Phase I Healthy volunteers 108 • Randomised, double-blind, single-dose, single-centre, • Primary endpoints: AUCinf, AUClast • FPCD: Q3 2022 NCT054771
Slide 92: BioPharmaceuticals: Early-stage development
BioPharmaceuticals Early-stage development 92 92
Slide 93: elecoglipron (AZD5004)
Approved medicines Late-stage development elecoglipron (AZD5004, oral GLP-1 RA) Oncology Early development Type 2 diabetes, obesity Trial Population Patients Design Endpoints Status Phase IIb Type 2 diabetes 406 • Arm 1: AZD5004 tablet • Primary endpoint: change in HbA1c • FPCD: Q4 2024 SOLSTICE • A
Slide 94: elecoglipron (AZD5004)
Approved medicines Late-stage development elecoglipron (AZD5004, oral GLP-1 RA) Oncology Early development Type 2 diabetes, obesity Trial Population Patients Design Endpoints Status Phase I Healthy volunteers or 16 • Multi-centre, single-dose, non-randomised, open-label, • Primary endpoint: PK param
Slide 95: elecoglipron (AZD5004)
Approved medicines Late-stage development elecoglipron (AZD5004, oral GLP-1 RA) Oncology Early development Type 2 diabetes, obesity Trial Population Patients Design Endpoints Status Phase I Healthy volunteers 32 • Open-label, fixed-sequence, two-part, single-centre trial • Part A and B: • FPCD: Q2 2
Slide 96: opemalirsen (AZD2373)
Approved medicines Late-stage development opemalirsen (AZD2373, APOL1) Oncology Early development Chronic kidney disease Trial Population Patients Design Endpoints Status Phase IIb Participants diagnosed with 96 • Randomised, multi-centre, double-blind trial in US and UK • Primary endpoint: Dose-res
Slide 97: AZD1043 (mAb)
Approved medicines Late-stage development AZD1043 (mAb) Oncology Early development Weight management Trial Population Patients Design Endpoints Status Phase I Healthy participants living 104 • Randomised, single-blind, placebo-controlled trial • Primary endpoints: Adverse events • Initiating D9740C0
Slide 98: AZD1613 (PAPPA-1 mAb)
Approved medicines Late-stage development AZD1613 (PAPPA-1 mAb) Oncology Early development ADPKD Trial Population Patients Design Endpoints Status Phase I Healthy volunteers 136 • Randomised, single-blind, placebo-controlled single and • Primary endpoint: safety and • FPCD: Q2 2025 NCT06995820 multi
Slide 99: AZD1705 (Angptl3 inhibitor)
Approved medicines Late-stage development AZD1705 (Angptl3 inhibitor) Oncology Early development Dyslipidaemia Trial Population Patients Design Endpoints Status Phase I Dyslipidaemia 112 • Part A: single dose of AZD1705 with an in-clinic period of 3 • Primary endpoints: AEs and SAEs • FPCD: Q1 2024
Slide 100: AZD2389 (anti-fibrotic mechanism)
Approved medicines Late-stage development AZD2389 (anti-fibrotic mechanism) Oncology Early development MASH Trial Population Patients Design Endpoints Status Phase II Participants with liver fibrosis 40 • Randomised, single-blind, placebo-controlled trial • Primary endpoints: safety and • FPCD: Q4 2
Slide 101: AZD3974
Approved medicines Late-stage development AZD3974 (anti-inflammatory and anti-fibrotic mechanism) Oncology Early development Cirrhosis Trial Population Patients Design Endpoints Status Phase I Healthy volunteers 176 • Single-blind, placebo-controlled, randomised • Primary endpoints: Safety/tolerabil
Slide 102: AZD4063 (PLN siRNA)
Approved medicines Late-stage development AZD4063 (PLN siRNA) Oncology Early development Dilated cardiomyopathy Trial Population Patients Design Endpoints Status Phase I R14del dilated 31 • Unblinded, SAD/MAD with 3 cohorts • Primary endpoints: Safety and • FPCD: Q4 2025 PULSE cardiomyopathy tolerab
Slide 103: AZD4144 (NLRP3)
Approved medicines Late-stage development AZD4144 (NLRP3) Oncology Early development Cardiorenal disease Trial Population Patients Design Endpoints Status Phase IIa Participants with Sepsis- 124 • Phase IIa, randomised, double-blind, placebo-controlled • Primary endpoint: AUC 24-hour CrCl • FPCD: Q1
Slide 104: AZD4144 (NLRP3)
Approved medicines Late-stage development AZD4144 (NLRP3) Oncology Early development Cardiorenal disease Trial Population Patients Design Endpoints Status Phase I Healthy participants with 28 • Placebo-controlled, parallel group study • Primary endpoints: safety, tolerability • FPCD: Q2 2025 NCT0694
Slide 105: AZD4248 (NNMT)
Approved medicines Late-stage development AZD4248 (NNMT) Oncology Early development CKD Trial Population Patients Design Endpoints Status Phase I Healthy volunteers 164 • Randomised, single-blind, placebo-controlled • Primary endpoints: Safety and • FPCD: Q3 2025 NCT07024823 • Part A: SAD in healthy
Slide 106: AZD4954 (Lp(a) inhibitor)
Approved medicines Late-stage development AZD4954 (Lp(a) inhibitor) Oncology Early development Dyslipidaemia Trial Population Patients Design Endpoints Status Phase I Healthy volunteers 120 • Randomised, placebo-controlled SAD/MAD trial • Primary endpoints: safety and • FPCD: Q2 2025 NCT06980428 tol
Slide 107: AZD5462 (oral relaxin)
Approved medicines Late-stage development AZD5462 (oral relaxin) Oncology Early development Heart failure Trial Population Patients Design Endpoints Status Phase IIb Stable patients with chronic 375 • Two cohort, randomised, double-blind, placebo-controlled, • Primary endpoint: change in heart • FPC
Slide 108: AZD6234 (amylin receptor agonist)
Approved medicines Late-stage development AZD6234 (selective amylin receptor agonist) Oncology Early development Obesity with related co-morbidities Trial Population Patients Design Endpoints Status Phase II Participants living with 231 • Randomised, double-blind, placebo-controlled trial • Primary
Slide 109: AZD6234 (amylin receptor agonist)
Approved medicines Late-stage development AZD6234 (selective amylin receptor agonist) Oncology Early development Obesity with related co-morbidities Trial Population Patients Design Endpoints Status Phase I Participants include those 48 • Phase I multicentre, single-dose, non-randomised, open- • com
Slide 110: AZD9550 (GLP-1-glucagon receptor agonist)
Approved medicines Late-stage development AZD9550 (GLP-1-glucagon receptor agonist) Oncology Early development MASH, Obesity Trial Population Patients Design Endpoints Status Phase I/II Overweight and obese 118 • Randomised, single-blind, placebo-controlled, MAD trial • Primary endpoints: safety, to
Slide 111: AZD9550+AZD6234
Approved medicines Late-stage development AZD9550+AZD6234 (GLP-1-glucagon receptor agonist + selective amylin receptor agonist) Oncology Early development Obesity Trial Population Patients Design Endpoints Status Phase IIb Adults who are living with 360 • Randomised, parallel-group, double-blind, pl
Slide 112: atuliflapon (FLAP inhibitor)
Approved medicines Late-stage development atuliflapon (FLAP inhibitor) Oncology Early development Asthma Trial Population Patients Design Endpoints Status Phase IIa Patients with moderate-to- 666 • Randomised, placebo-controlled, double-blind, multi-centre • Primary endpoint: time to first • FPCD: Q
Slide 113: surovatamig: RA, SLE
Approved medicines Late-stage development surovatamig (CD19/CD3 T-cell engager) Oncology Early development RA, SLE Trial Population Patients Design Endpoints Status Phase I Adult Participants With 48 • Open-label, multicentre • Primary endpoints: safety and • FPCD: Q1 2026 NCT07201558 Rheumatoid Art
Slide 114: AZD0120: autoimmune conditions, neurology and SLE
Approved medicines Late-stage development AZD0120 (autologous anti-CD19 and anti-BCMA CAR-T) Oncology Early development Autoimmune conditions, neurology and SLE Trial Population Patients Design Endpoints Status Phase Ib Adult participants with 27 • Open-label multi-cohort • Primary: Incidence and se
Slide 115: AZD1163 (anti-PAD2/4 bispecific antibody)
Approved medicines Late-stage development AZD1163 (anti-PAD2/4 bispecific antibody) Oncology Early development Rheumatoid arthritis Trial Population Patients Design Endpoints Status Phase IIb Moderate-to-severely active 320 • Double-blind, 4-arm study • Primary endponit: Change from • FPCD: Q1 2026
Slide 116: AZD4604 (inhaled JAK-1 inhibitor)
Approved medicines Late-stage development AZD4604 (inhaled JAK-1 inhibitor) Oncology Early development Asthma Trial Population Patients Design Endpoints Status Phase IIa Moderate-to-severe asthma 320 • Multi-centre, randomised, placebo-controlled, double- • Primary endpoint: time to first • FPCD: Q4
Slide 117: AZD5492 (CD20 TITAN TCE): SLE
Approved medicines Late-stage development AZD5492 (CD20 TITAN TCE) Oncology Early development SLE Trial Population Patients Design Endpoints Status Phase I Systemic lupus 70 • Open-label, multii-centre • Primary endpoints: safety and • Data anticipated: 2027 TITAN erythematosus (SLE) or • Part 1: Si
Slide 118: AZD6793 (IRAK4)
Approved medicines Late-stage development AZD6793 (IRAK4) Oncology Early development COPD Trial Population Patients Design Endpoints Status Phase II Moderate to very severe 1160 • Randomised, double-blind, placebo controlled 4 arm study • Primary endpoint: annualised rate of • FPCD: Q3 2025 PRESTO C
Slide 119: AZD6912 (siRNA)
Approved medicines Late-stage development AZD6912 (siRNA) Oncology Early development Rheumatoid arthritis Trial Population Patients Design Endpoints Status Phase I Healthy volunteers 40 • Randomised, double-blind, placebo-controlled SAD trial • Primary endpoint: incidence of AEs • FPCD: Q4 2023 NCT0
Slide 120: AZD7798 (humanised mAb)
Approved medicines Late-stage development AZD7798 (humanised mAb) Oncology Early development Crohn's disease Trial Population Patients Design Endpoints Status Phase IIa Moderate to severe Crohn’s 120 • Randomised, double-blind, placebo-controlled trial • Primary endpoint: Crohn's Disease • FPCD: Q4
Slide 121: AZD8630 (inhaled TSLP)
Approved medicines Late-stage development AZD8630 (inhaled TSLP) Oncology Early development Asthma Trial Population Patients Design Endpoints Status Phase II Adults with uncontrolled 516 • Randomised, placebo-controlled, double-blind, dose range- • Primary endpoint: time to first • FPCD: Q3 2024 LEV
Slide 122: AZD8965 (arginase enzyme inhibitor)
Approved medicines Late-stage development AZD8965 (arginase enzyme inhibitor) Oncology Early development IPF Trial Population Patients Design Endpoints Status Phase I Healthy volunteers 163 • Randomised, single-blind, SAD/MAD, placebo-controlled, • Primary endpoints (Part 1, 2, 3): safety • FPCD: Q3
Slide 123: AZD0292 (Psl-PcrV N3Y-bispecific mAb)
Approved medicines Late-stage development AZD0292 (Psl-PcrV N3Y-bispecific mAb) Oncology Early development Bronchiectasis Trial Population Patients Design Endpoints Status Phase IIb Bronchiectasis patients ≥ 12 435 • Randomised, double-blind, placebo-controlled, parallel, • Primary: efficacy • FPCD:
Slide 124: AZD5148 (anti-TcdB mAb)
Approved medicines Late-stage development AZD5148 (anti-TcdB mAb) Oncology Early development Clostridium difficile Trial Population Patients Design Endpoints Status Phase IIb Adults with qualifying C. 230 • Randomised, double-blind, placebo-controlled • Primary endpoint: efficacy • FPCD: Q4 2025 PRI
Slide 125: AZD7760 (mAb combination)
Approved medicines Late-stage development AZD7760 (mAb combination targeting S aureus virulence factors) Oncology Early development Prevention of Staph aureus infection Trial Population Patients Design Endpoints Status Phase I/IIa Phase I: healthy volunteers 231 • Phase I: randomised, double-blind,
Slide 126: Rare Disease
Rare Disease Approved medicines and late-stage pipeline 126 126
Slide 127: Koselugo (selumetinib)
Approved medicines Late-stage development Koselugo (selumetinib, MEK inhibitor) Oncology Early development Neurofibromatosis type 1, solid tumours Trial Population Patients Design Endpoints Status Phase III Adults with NF1 who have 145 • Multi-centre, international trial with a parallel, randomised,
Slide 128: Ultomiris: haematology, nephrology, transplant
Approved medicines Late-stage development Ultomiris (anti-C5 mAb) Oncology Early development Haematology, nephrology, transplant Trial Population Patients Design Endpoints Status Phase III CSA-AKI 736 • Randomised, double-blind, placebo-controlled, muticentre • Primary endpoint: to assess the • FPCD
Slide 129: Ultomiris: nephropathy, neurology
Approved medicines Late-stage development Ultomiris (anti-C5 mAb) Oncology Early development Nephropathy, neurology Trial Population Patients Design Endpoints Status Phase II Proliferative lupus nephritis or 120 • Arm 1: LN cohort, Ultomiris • Primary endpoint: percentage change • FPCD: Q1 2021 SANC
Slide 130: anselamimab (CAEL-101)
Approved medicines Late-stage development anselamimab (CAEL-101, fibril-reactive mAb) Oncology Early development AL amyloidosis Trial Population Patients Design Endpoints Status Phase III AL amyloidosis (Mayo Stage 124 • Arm 1: anselamimab combined with SoC for PCD • Primary endpoint: a hierarchical
Slide 131: cliramitug (ALXN2220)
Approved medicines Late-stage development cliramitug (ALXN2220, TTR depleter) Oncology Early development Amyloidosis Trial Population Patients Design Endpoints Status Phase III ATTR-CM (wild-type and 1180 • Arm 1: cliramitug via i.v. infusion Q4W for at least 30 months • Primary endpoint: composite
Slide 132: efzimfotase alfa (ALXN1850)
Approved medicines Late-stage development efzimfotase alfa (ALXN1850, next-generation asfotase alfa) Oncology Early development Hypophosphatasia Trial Population Patients Design Endpoints Status Phase III Hypophosphatasia 40 • Arm 1: bodyweight-dependent doses of either 20mg, 35mg • Primary endpoint
Slide 133: eneboparatide
Approved medicines Late-stage development eneboparatide (parathyroid hormone receptor 1 agonist) Oncology Early development Hypoparathyroidism Trial Population Patients Design Endpoints Status Phase III Chronic hypoparathyroidism 165 • Arm 1: 20mcg eneboparatide administered once daily via s.c. • Pr
Slide 134: gefurulimab (ALXN1720)
Approved medicines Late-stage development gefurulimab (ALXN1720, anti-C5 dual-binding nanobody) Oncology Early development Neurology Trial Population Patients Design Endpoints Status Phase III Generalised myasthenia 260 • Arm 1: weight-based maintenance treatment with • Primary endpoint: change from
Slide 135: Rare Disease: Early-stage development
Rare Disease Early-stage development 135 135
Slide 136: tarperprumig (ALXN1820)
Approved medicines Late-stage development tarperprumig (ALXN1820, anti-properdin) Oncology Early development Anti-neutrophil cytoplasmic antibody (ANCA) associated vasculitis Trial Population Patients Design Endpoints Status Phase II Newly diagnosed or relapsing 75 • Randomised, double-blind, placeb
Slide 137: ALXN1920 (factor H fusion protein)
Approved medicines Late-stage development ALXN1920 (kidney-targeted factor H fusion protein) Oncology Early development Nephrology Trial Population Patients Design Endpoints Status Phase II Primary membranous 30 • Randomised, double-blind, placebo-controlled • Primary Endpoint: Change From • FPCD: Q
Slide 138: ALXN2030 (siRNA targeting complement C3)
Approved medicines Late-stage development ALXN2030 (siRNA targeting complement C3) Oncology Early development Transplant Trial Population Patients Design Endpoints Status Phase II Kidney transplant recipients 45 • Randomised, controlled, double-blind • Primary endpoint: Biopsy-proven • FPCD: Q4 2025
Slide 139: ALXN2230
Approved medicines Late-stage development ALXN2230 Oncology Early development Neurology and haematology Trial Population Patients Design Endpoints Status Phase I Healthy participants 40 • Randomised, double-blind, placebo controlled trial • Primary endpoint: Safety and • FPCD: Q1 2026 NCT07352423 •
Slide 140: ALXN2350 (AAV gene therapy)
Approved medicines Late-stage development ALXN2350 (AAV gene therapy) Oncology Early development BAG3-associated dilated cardiomyopathy (DCM) Trial Population Patients Design Endpoints Status Phase I/II BAG3 mutation associated 18 • Open-label, dose finding and dose expansion study • Primary endpoin
Slide 141: ALXN2420 (GH receptor antagonist)
Approved medicines Late-stage development ALXN2420 (GH receptor antagonist) Oncology Early development Acromegaly Trial Population Patients Design Endpoints Status Phase IIb Acromegaly 60 • Randomised, double-blinded, placebo-controlled, dose • Primary endpoint: Percentage change • FPCD: Q4 2025 AST
Slide 142: AZD0120: AL amyloidosis
Approved medicines Late-stage development AZD0120 (autologous anti-CD19 and anti-BCMA CAR-T) Oncology Early development AL amyloidosis Trial Population Patients Design Endpoints Status Phase I/II Relapsed or Refractory AL 91 • Open-label, multicentre, non-randomised trial • Primary endpoint: % of pt
Slide 143: AZD1390 (ATM inhibitor)
Approved medicines Late-stage development AZD1390 (ATM inhibitor) Oncology Early development Solid tumours Trial Population Patients Design Endpoints Status Phase I Recurrent glioblastoma 180 • Open-label trial • Primary endpoints: safety, tolerability • FPCD: Q2 2018 NCT03423628 eligible for re-irr
Slide 144: 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
Slide 145: 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
Slide 146: 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
Slide 147: 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
Slide 148: 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