Astrazeneca Plc · LSE:AZN
Astrazeneca — Q3 2026 Earnings Presentation
153 slides from Astrazeneca's earnings presentation, Oct 30, 2026
Slide 1: Clinical Trials Appendix
Clinical Trials Appendix Q3 2026 Results Update 30 October 2026
Slide 2: Pipeline at a glance
Pipeline 180 20 at a glance projects in our new molecular entities development pipeline (NME) in our late-stage pipeline Across five focus therapy areas: Oncology BioPharmaceuticals 116 45 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 H2 2026 H1 2027 H2 2027 Imfinzi | muscle invasive bladder cancer (cis-ineligible/refusal) | VOLGA Calquence | 1L MCL | ECHO (CN) Calquence | previously untreated CLL | AMPLIFY (CN) Ultomiris | IgAN | I CAN (JP) E
Slide 4: Clinical Trials Appendix: selected highlights
Clinical Trials Appendix: selected highlights Oncology BioPharmaceuticals Rare Disease Calquence Airsupra Klygefa (gefurulimab) Calquence Airsupra Klygefa (gefurulimab) Datroway Baxfendy (baxdrostat) Ultomiris Datroway Baxfendy (baxdrostat) Ultomiris Enhertu Breztri Enhertu Breztri Approved Etcamah
Slide 5: Project movements since Q2 2026 update
Project movements since Q2 2026 update New to Phase I New to pivotal trial New to registration NME NME Additional indication Lifecycle Management AZD0543 lenbelintide (AZD6234) elecoglipron/dapagliflozin FDC Imfinzi + EV +/- Imjudo VOLGA long-acting hMPV neutralising mAb selective amylin receptor ag
Slide 6: Project movements since Q2 2026 update
Project movements since Q2 2026 update Removed from Phase I Removed from Phase II Removed from Phase III Approved /removed from registration NME Additional indication NME AZD2962 volrustomig eVOLVE-Lung02 Amlypave (anselamimab) CARES IRAK4 inhibitor haematological PD-1/CTLA-4 bispecific mAb 1L fibri
Slide 7: Q3 2026 Oncology new molecular entity pipeline
Q3 2026 Oncology new molecular entity1 pipeline Phase I Phase II Phase III 19 New Molecular Entities 15 New Molecular Entities 26 New Molecular Entities Imfinzi +/- oleclumab +/- monalizumab torvutatug samrotecan +/- bevacizumab surovatamig AZD5492 IPH5201 + Imfinzi# volrustomig CANTOR PACIFIC-9# TR
Slide 8: Q3 2026 Oncology lifecycle management pipeline
Q3 2026 Oncology lifecycle management1 pipeline Phase I Phase II Phase III Under review 0 Projects 2 Projects 28 Projects 2 Projects Datroway +/-rilvegostomig TROPION- Imfinzi combinations NeoCOAST-2# Urothelial04# Datroway TROPION-Lung17# PD-L1 mAb + novel ONC therapies Calquence + R-CHOP ESCALADE
Slide 9: Q3 2026 BioPharmaceuticals new molecular entity pipeline
Q3 2026 BioPharmaceuticals new molecular entity1 pipeline Phase I Phase II Phase III 13 New Molecular Entities 15 New Molecular Entities 14 New Molecular Entities surovatamig AZD3974 AZD5148 balcinrenone/dapagliflozin elecoglipron/dapagliflozin FDC CD19/CD3 TCE B-cell driven anti-inflammatory and an
Slide 10: Q3 2026 BioPharmaceuticals lifecycle management pipeline
Q3 2026 BioPharmaceuticals lifecycle management1 pipeline Phase I Phase II Phase III Under review 0 Projects 0 Projects 11 Projects 0 Projects Baxfendy BaxPA Saphnelo JASMINE# aldosterone synthase inhibitor prim ary type I IFN receptor mA b myositis aldosteronism Baxfendy/dapagliflozin Saphnelo LAVE
Slide 11: Q3 2026 Rare Disease pipeline
Q3 2026 Rare Disease pipeline1 Phase I Phase II Phase III Under review 5 Projects 3 Projects 2 Projects 2 Projects ALXN2080 cevolisiran (ALXN2030) CONCORD cliramitug DepleTT R-CM# efzimfotas e alfa Hickory (301), Mulberry (305), Ches tnut (303) oral factor D healthy participants siRNA targe ting com
Slide 12: Active designations in our pipeline
Active designations in our pipeline 6 3 12 3 18 Priority Review Breakthrough / PRIME1 / Sakigake2 Fast Track Qualified Infectious Disease product Orphan tozorakimab COPD (US) AZD0292 Psl-PcrV N3Y NCFBE (EU) AZD0292 Psl-PcrV N3Y NCFBE (US) AZD0292 Psl-PcrV N3Y NCFBE (US) Saphnelo myositis JASMINE (US
Slide 13: Oncology
Oncology Approved medicines and late-stage pipeline 13 13
Slide 14: 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 15: 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 644 • Open-label, randomised trial • Primary endpoints: PFS (BICR) and OS • FPCD
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 III 1L locally advanced/metastatic NSCLC 1350 • Randomised, open-label • Primary endpoints: PFS and OS in NSQ • FPCD: Q1 202
Slide 17: Datroway: NSCLC
Approved medicines Late-stage development Datroway (datopotamab deruxtecan, TROP2 ADC) Oncology Early development NSCLC Trial Population Patients Design Endpoints Status Phase III TROP2 QCS NMR+ non-squamous NSCLC 400 • Randomized, double blind, placebo-controlled • PFS by BICR and OS • FPCD: Q4 202
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 III High-risk muscle Invasive urothelial 915 • Global, randomised, open label trial • Primary endpoint: DFS (INV) •
Slide 19: 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 20: 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 21: 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 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 III Advanced treatment-naïve HER2- 620 • Arm A: Enhertu + rilvegostomig • Primary endpoint: OS • FPCD: Q3 2024 DESTINY
Slide 23: 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 127 • Non-randomised single group assignment • Primary endpoint: ORR • FPCD: Q3 2024 DESTIN
Slide 24: Etcamah: Breast cancer
Approved medicines Late-stage development Etcamah (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: Etcamah • Primary endpoint: IBCFS • FPCD: Q1 2023 CAMBR
Slide 25: 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 26: 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 27: 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 1L bladder cancer 1246 • Arm 1: Imfinzi + Imjudo + SoC • Primary endpoint: OS • FPCD: Q4 2018 NILE • Arm 2: Imfinzi + SoC • L
Slide 28: 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 29: 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 30: Truqap: Breast cancer 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 31: zadavotide guraxetan: Prostate cancer
Approved medicines Late-stage development (225Ac) zadavotide guraxetan (AZD2265, PSMA radioconjugate) Oncology Early development Prostate cancer Trial Population Patients Design Endpoints Status Phase III PSMA-positive mCRPC post-ARPI, - 670 • Multicentre, randomised controlled trial • Primary endpo
Slide 32: puxitatug samrotecan: Solid tumours
Approved medicines Late-stage development puxitatug samrotecan (AZD8205, B7-H4 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 • FPC
Slide 33: 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 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 III 1L PD-L1 TC ≥1% squamous NSCLC 1160 • Randomised, double-blind, multicentre • Primary endpoints: PFS, OS • FPCD: Q4 2
Slide 35: 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 36: 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 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 III BRCA1, BRCA2, or PALB2m, HR-positive, 788 • Randomised, open-label trial • Primary endpoint: PFS (BICR) • FPCD: Q3 2024
Slide 38: 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/IIa Metastatic prostate cancer 175 • Multi-arm, open-label, non-randomised, multi-centre trial • Primary endpoints: safety
Slide 39: 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 594 • Multi-centre, open-label, sponsor-blinded, randomised trial • Pr
Slide 40: 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 41: 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 I R/R B-cell non-Hodgkin lymphoma 317 • Multi-centre, open-label, dose escalation and dose • Primary endpoints:
Slide 42: torvutatug samrotecan: Ovarian cancer, solid tumours
Approved medicines Late-stage development torvutatug samrotecan (torvu-sam, 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 endp
Slide 43: 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 44: 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 Metastatic colorectal cancer 80 • Platform, randomised, open-label, multicentre, global trial • Primary endpoints: PF
Slide 45: 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 46: Oncology: Early-stage development
Oncology Early-stage development 46 46
Slide 47: 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 48: JAB-23E73 (pan-KRAS inhibitor)
Approved medicines Late-stage development JAB-23E73 (pan-KRAS inhibitor) Oncology Early development Solid tumours Trial Population Patients Design Endpoints Status Phase Ib/III 1L metastatic PDAC harbouring KRAS 80 • JAB-23E73 + nab-paclitaxel + gemcitabine • Primary endpoints (Phase Ib): DLT, • FPC
Slide 49: 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 108 • Open-label, adult, single-arm, multi-centre trial with dose • Primary endpoints: inci
Slide 50: 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 51: 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 52: vemzatatug vedotin (AZD0305)
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 53: 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 54: 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 55: 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 56: 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 7 • Modular, open-label, multicentre • Primary endpoints: DLT, AEs, duration • FPCD: Q4 2025 NCT07064122 • AZD
Slide 57: AZD3470 (PRMT5 inhibitor)
Approved medicines Late-stage development AZD3470 (PRMT5 inhibitor) Oncology Early development Solid tumours and blood cancer Trial Population Patients Design Endpoints Status Phase I/II R/R haematologic malignancies 110 • Modular Phase I/II open-label dose escalation and • Primary endpoints: safety
Slide 58: 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 59: AZD4045 (BCMA UEC)
Approved medicines Late-stage development AZD4045 (allogeneic BCMA-targeting Universal Effector Cell (UEC)) Oncology Early development Haematology Trial Population Patients Design Endpoints Status Phase I/II Relapsed/refractory multiple myeloma 101 • Modular, open-label, multicentre trial • Primary:
Slide 60: 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 61: 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 62: 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 63: 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/II Advanced or metastatic Claudin 18.2- 392 • Part A: AZD5863 dose escalation • Primary endpoints (Part A): safety a
Slide 64: 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 65: 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 120 • Open-label, dose escalation and expansion study • Primary endpoints: Safety and efficacy
Slide 66: 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 GPC3-positive advanced/recurrent HCC 121 • Open-label, single-arm, multicentre trial • Primary endpoints (Part A): DLT, A
Slide 67: AZD8359 (STEAP2 T-cell engager)
Approved medicines Late-stage development AZD8359 (STEAP2 T-cell engager) Oncology Early development Prostate cancer Trial Population Patients Design Endpoints Status Phase I/II Prostate cancer 42 • Open-label, multi-centre, modular design • Primary: safety, tolerability • FPCD: Q2 2026 CRIUS-1 • Pa
Slide 68: 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 69: 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 70: 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 71: BioPharmaceuticals
BioPharmaceuticals Approved medicines and late-stage pipeline 71 71
Slide 72: Baxfendy: Hypertension
Approved medicines Late-stage development Baxfendy (baxdrostat, selective aldosterone synthase inhibitor) Oncology Early development Hypertension Trial Population Patients Design Endpoints Status Phase III Patients with resistant 218 • Arm 1: Baxfendy 2mg QD • Primary endpoint: effect of Baxfendy •
Slide 73: Baxfendy: Primary aldosteronism
Approved medicines Late-stage development Baxfendy (baxdrostat, selective aldosterone synthase inhibitor) Oncology Early development Primary aldosteronism Trial Population Patients Design Endpoints Status Phase III Primary aldosteronism 250 • Multicentre, randomised, double-blind, placebo-controlled
Slide 74: Wainua: ATTR
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 75: balcinrenone/dapagliflozin: Heart failure, CKD
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 Patients with CKD Stage 3b 2800 • Randomised, double-blind, double-dummy, paralle
Slide 76: balcinrenone/dapagliflozin: Heart failure, CKD
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 IIb CKD 300 • Multicentre, randomised, double-blind, dose-finding, • Primary endpoint
Slide 77: baxdrostat/dapagliflozin: CKD
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 78: elecoglipron: CKD
Approved medicines Late-stage development elecoglipron (oral GLP-1 RA) Oncology Early development CKD Trial Population Patients Design Endpoints Status Phase III Chronic kidney disease 7000 • Randomised, double-blind, parallel-group, placebo- • Primary endpoint: Time to the first • FPCD: Q3 2026 Ele
Slide 79: elecoglipron: HFpEF
Approved medicines Late-stage development elecoglipron (oral GLP-1 RA) Oncology Early development HFpEF Trial Population Patients Design Endpoints Status Phase III HFpEF and HFmrEF 6950 • Randomised, double-blind, parallel-group, placebo- • Primary endpoint: Time to first event • FPCD: Q3 2026 ELEVA
Slide 80: elecoglipron: Type 2 diabetes
Approved medicines Late-stage development elecoglipron (oral GLP-1 RA) Oncology Early development Type 2 diabetes Trial Population Patients Design Endpoints Status Phase III Type 2 diabetes 800 • Randomised, double-blind, placebo-controlled • Primary endpoint: change in HbA1c • FPCD: Q3 2026 Elumina
Slide 81: elecoglipron: Weight management
Approved medicines Late-stage development elecoglipron (oral GLP-1 RA) Oncology Early development Weight management Trial Population Patients Design Endpoints Status Phase III Obesity or overweight with 4500 • Randomised, parallel-group, double-blind trial • Primary endpoint: Percent change • FPCD:
Slide 82: elecoglipron: Weight management
Approved medicines Late-stage development elecoglipron (oral GLP-1 RA) Oncology Early development Weight management Trial Population Patients Design Endpoints Status Phase I Healthy volunteers or 35 • SAD: 3 cohorts to receive elecoglipron or placebo tablet • Primary endpoints: safety and • FPCD: Q4
Slide 83: elecoglipron: Weight management
Approved medicines Late-stage development elecoglipron (oral GLP-1 RA) Oncology Early development Weight management Trial Population Patients Design Endpoints Status Phase I Healthy volunteers 65 • Open-label, randomised, 2 part, 3-period, single-dose trial • Part A and B: • FPCD: Q2 2026 NCT0745582
Slide 84: laroprovstat: Dyslipidaemia
Approved medicines Late-stage development laroprovstat (AZD0780, PCSK9 inhibitor) Oncology Early development Dyslipidaemia Trial Population Patients Design Endpoints Status Phase III Heterozygous familial 473 • Randomised, double-blind, placebo-controlled, parallel- • Primary endpoint: Relative chan
Slide 85: laroprovstat: Dyslipidaemia
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 86: laroprovstat: Dyslipidaemia
Approved medicines Late-stage development laroprovstat (AZD0780, PCSK9 inhibitor) Oncology Early development Dyslipidaemia Trial Population Patients Design Endpoints Status Phase I Healthy volunteers 59 • Open-label, fixed sequence trial to assess the PK of • Primary endpoint: PK parameters • FPCD:
Slide 87: laroprovstat/ezetimibe FDC
Approved medicines Late-stage development laroprovstat/ezetimibe FDC (PCSK9 inhibitor + NPC1L1 inhibitor) Oncology Early development Dyslipidaemia Trial Population Patients Design Endpoints Status Phase I Healthy adults 18 • Open-label, cross-over trial • Primary endpoint: relative • FPCD: Q3 2026 N
Slide 88: laroprovstat/rosuvastatin FDC
Approved medicines Late-stage development laroprovstat/rosuvastatin FDC (PCSK9 inhibitor + HMG-CoA reductase inhibitor) Oncology Early development Dyslipidaemia Trial Population Patients Design Endpoints Status Phase III Treatment naive patients 600 • Randomised, double-blind, factorial design trial
Slide 89: lenbelintide: Obesity
Approved medicines Late-stage development lenbelintide (AZD6234, selective amylin receptor agonist) Oncology Early development Obesity with related co-morbidities Trial Population Patients Design Endpoints Status Phase III Adults with obesity or 2500 • Randomised, parallel-group, double-blind trial
Slide 90: zibotentan/dapagliflozin: Chronic kidney disease
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 91: AZD4144 (NLRP3)
Approved medicines Late-stage development AZD4144 (NLRP3) Oncology Early development Cardiorenal disease Trial Population Patients Design Endpoints Status Phase I Partipants with hepatic 30 • Phase I, open-label, parallel group study • Primary endpoint: PK • FPCD: Q3 2026 NCT07783308 impairment and
Slide 92: Airsupra: Asthma
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 93: 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 94: 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 95: Fasenra: Other eosinophilic diseases
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 HES (history of 134 • Randomised, double-blind, placebo controlled • Primary endpoint: time to first HES • FP
Slide 96: Saphnelo: Lupus (SLE/LN)
Approved medicines Late-stage development Saphnelo (type I interferon receptor mAb) Oncology Early development Lupus (SLE/LN) Trial Population Patients Design Endpoints Status Phase III Moderate to severe SLE 276 • Randomised, double-blind, placebo-controlled • Primary endpoint: BICLA at Week 52 • F
Slide 97: Saphnelo: Sclerosis and other myopathies
Approved medicines Late-stage development Saphnelo (type I interferon receptor mAb) Oncology Early development Sclerosis and other myopathies Trial Population Patients Design Endpoints Status Phase III Systemic sclerosis 314 • Randomised, double-blind, placebo-controlled • Primary endpoint: CRISS-25
Slide 98: Tezspire: CRSwNP, COPD and EoE
Approved medicines Late-stage development Tezspire (TSLP mAb) Oncology Early development CRSwNP, COPD and EoE 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 • FPCD: Q1 2023 CROSSING years
Slide 99: Next-generation propellant pMDI
Approved medicines Late-stage development Next-generation propellant Oncology Early development pMDI Trial Population Patients Design Endpoints Status Phase III Moderate to very severe 542 • Randomised, double-blind, 12-week (with an extension to • Primary endpoints: number of • FPCD: Q3 2022 NCT055
Slide 100: 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 105 • Randomised, double-blind, single-dose, single-centre, 3-way • Primary endpoints: AUClast and Cmax • FPCD: Q1 2025 NC
Slide 101: 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 102: tozorakimab: COPD
Approved medicines Late-stage development tozorakimab (IL-33 ligand mAb) Oncology Early development COPD Trial Population Patients Design Endpoints Status Phase III Subjects who completed 1713 • Randomised, double-blind, placebo-controlled, parallel- • Primary endpoint: annualised rate of • FPCD: Q1
Slide 103: 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 3527 • Randomised, double-blind, placebo-controlled, parallel • Primary endpoint: pr
Slide 104: BioPharmaceuticals: Early-stage development
BioPharmaceuticals Early-stage development 104 104
Slide 105: lenbelintide: Obesity with related co-morbidities
Approved medicines Late-stage development lenbelintide (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 tr
Slide 106: opemalirsen (AZD2373, APOL1)
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 107: surovatamig: Antibody-mediated kidney disease
Approved medicines Late-stage development surovatamig (CD19/CD3 T-cell engager) Oncology Early development Antibody-mediated kidney disease Trial Population Patients Design Endpoints Status Phase II Primary membranous 25 • Part A: Multiple ascending with sentinel dosing • Primary endpoint: safety an
Slide 108: AZD1043 (ActRII mAb)
Approved medicines Late-stage development AZD1043 (ActRII 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 • Data antici
Slide 109: 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 Autosomal dominant 40 • Randomised, single-blind, placebo-controlled study with • Primary endpoint: Safety and • FPCD: Q1 2026 PIONEER-PKD polycy
Slide 110: 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 111: AZD3974: cirrhosis
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 112: 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 113: AZD4144 (NLRP3)
Approved medicines Late-stage development AZD4144 (NLRP3) Oncology Early development Cardiorenal disease Trial Population Patients Design Endpoints Status Phase IIa Sepsis-associated acute 124 • Phase IIa, randomised, double-blind, placebo-controlled • Primary endpoint: AUC 24-hour CrCl • FPCD: Q1 2
Slide 114: AZD4144 (NLRP3)
Approved medicines Late-stage development AZD4144 (NLRP3) Oncology Early development Cardiorenal disease Trial Population Patients Design Endpoints Status Phase I Healthy participants 12 • Open-label, 2-period, 2-sequence cross over trial • Primary endpoints: PK and safety • FPCD: Q2 2025 NCT0692585
Slide 115: 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 116: 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 117: 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 118: 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 119: AZD9550+AZD6234: Obesity
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 120: sunakiment (AZD8630, inhaled TSLP)
Approved medicines Late-stage development sunakiment (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:
Slide 121: 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 122: 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/II Refractory systemic lupus 24 • Single-arm, open-label, multi-centre trial • P
Slide 123: 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 124: AZD4916: Respiratory
Approved medicines Late-stage development AZD4916 Oncology Early development Respiratory Trial Population Patients Design Endpoints Status Phase I Healthy volunteers 178 • Randomised, single-blind, placebo controlled SAD/MAD • Primary endpoints: Safety and • FPCD: Q3 2026 NCT06951880 trial tolerabil
Slide 125: 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: H1 2027 TITAN erythematosus, idiopathic • Part
Slide 126: AZD6793 (IRAK4): COPD
Approved medicines Late-stage development AZD6793 (IRAK4) Oncology Early development COPD Trial Population Patients Design Endpoints Status Phase II Moderate to very severe 987 • Randomised, double-blind, placebo controlled 3 arm 24 • Primary endpoint: annualised rate of • FPCD: Q3 2025 PRESTO COPD
Slide 127: 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 128: AZD8965 (arginase enzyme inhibitor)
Approved medicines Late-stage development AZD8965 (arginase enzyme inhibitor) Oncology Early development IPF Trial Population Patients Design Endpoints Status Phase IIb Participants with IPF 359 • Randomised, double-blind, placebo-controlled, parallel- • Primary endpoint: Absolute change • FPCD: Q3
Slide 129: AZD0292: Bronchiectasis
Approved medicines Late-stage development AZD0292 (Psl-PcrV N3Y-bispecific mAb) Oncology Early development Bronchiectasis Trial Population Patients Design Endpoints Status Phase IIb Bronchiectasis and chronic 84 • Randomized, double-blind, placebo-controlled, parallel trial • Primary: Annualised rat
Slide 130: AZD0543: Prevention of hMPV respiratory illness
Approved medicines Late-stage development AZD0543 (hMPV long-acting neutralising antibody) Oncology Early development Prevention of hMPV respiratory illness Trial Population Patients Design Endpoints Status Phase I Healthy volunteers 144 • Randomised, double-blind, placebo-controlled trial • Primary
Slide 131: 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 132: AZD7760: Prevention of Staph aureus infection
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 133: Rare Disease
Rare Disease Approved medicines and late-stage pipeline 133 133
Slide 134: 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 Delayed graft function in high 450 • Arm1: Placebo • Primary endpoint: time to freedom • FPCD: Q2 2025 AWAKE ri
Slide 135: Ultomiris: Neurology
Approved medicines Late-stage development Ultomiris (anti-C5 mAb) Oncology Early development Neurology Trial Population Patients Design Endpoints Status Phase II/III Paediatric patients (6 to < 18 12 • Single Arm • PK, PD, Safety, Efficacy at Week 18 • FPCD: Q3 2023 ALXN1210-MG-319 years of age) wit
Slide 136: Amlypave: AL amyloidosis
Approved medicines Late-stage development Amlypave (fibril-reactive mAb) Oncology Early development AL amyloidosis Trial Population Patients Design Endpoints Status Phase III AL amyloidosis (Mayo Stage 124 • Arm 1: Amlypave combined with SoC for PCD • Primary endpoint: a hierarchical • FPCD: Q1 2021
Slide 137: cliramitug: Amyloidosis
Approved medicines Late-stage development cliramitug (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 all-cause
Slide 138: efzimfotase alfa: Hypophosphatasia
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 139: eneboparatide: Hypoparathyroidism
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 140: Klygefa: Neurology
Approved medicines Late-stage development Klygefa (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 Klygefa on • Primary endpoint: change from •
Slide 141: Rare Disease: Early-stage development
Rare Disease Early-stage development 141 141
Slide 142: tarperprumig: ANCA associated vasculitis
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 143: ALXN1920: Nephrology
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 144: cevolisiran: Transplant
Approved medicines Late-stage development cevolisiran (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 •
Slide 145: ALXN2230: Neurology and haematology
Approved medicines Late-stage development ALXN2230 Oncology Early development Neurology and haematology Trial Population Patients Design Endpoints Status Phase I Healthy participants 48 • Randomised, double-blind, placebo controlled trial • Primary endpoint: Safety and • FPCD: Q2 2026 NCT07352423 •
Slide 146: 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 147: 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 148: 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 149: Glossary – 1 of 5
NEXT NEXT Glossary – 1 of 5 14C Carbon 14 AS Albuterol sulfate BR Bendamustine and rituximab 1L, 2L, 3L 1st-, 2nd- or 3rd-line ASCO American Society of Clinical Oncology BRCA BReast CAncer gene 5-FU 5-fluorouracil ASCVD Atherosclerotic cardiovascular disease BRCAm BReast CAncer gene-mutated 6MWT 6-m
Slide 150: Glossary – 2 of 5
BACK NEXT BACK NEXT Glossary – 2 of 5 CLDN18.2 Claudin 18.2 DDFS Distant disease-free survival ESCC Esophageal squamous cell carcinoma CLL Chronic lymphocytic leukaemia DDI Drug-drug Interaction ESKD End-stage kidney disease CM Cardiomyopathy DDR DNA damage response ESR1 Estrogen receptor 1 cm Centi
Slide 151: Glossary – 3 of 5
BACK NEXT BACK NEXT Glossary – 3 of 5 GN Glomerulonephritis HSCT-TMA hematopoietic stem cell transplantation-associated thrombotic LA amylin Long-acting amylin GPC3 Glypican-3 microangiopathy LAAB Long-acting antibody GPC3-positive Glypican 3-positive HSD17B13 Hydroxysteroid 17-beta dehydrogenase 13
Slide 152: Glossary – 4 of 5
BACK NEXT BACK NEXT Glossary – 4 of 5 mL Millilitre NMR+ Normalised membrane ratio-positive PFS6 Progression-free survival at 6 months MM Multiple myeloma NNMT Nicotinamide N-methyltransferase PgR Progesterone receptor MMAE Monomethyl auristatin E NP Nasal polyps PI3K Phosphoinositide 3 kinase MMT M
Slide 153: Glossary – 5 of 5
BACK BACK Glossary – 5 of 5 QCS Quantitative continuous scoring SD Stable disease TID Three times per day QD Once daily SERD Selective estrogen receptor degrader TIGIT T-cell immunoreceptor with Ig and ITIM domains QID Four times per day SES-CD Simple Endoscopic Score for Crohn’s Disease TIM-3 T-cel