Slide 1: Clinical Trials Appendix
Clinical Trials Appendix H1 2026 Results Update 27 July 2026
Slide 2: Pipeline at a glance
Pipeline 183 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 116 29 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 Datroway | met. TNBC not candidate for IO | TROPION-Breast02 (EU, JP, CN) Calquence | 1L MCL | ECHO (CN) tozorakimab | COPD | OBERON/TITANIA/MIRANDA/PROSPERO Imfinzi | high-risk non-muscle
Slide 4: Clinical Trials Appendix: selected highlights
Clinical Trials Appendix: selected highlights Oncology BioPharmaceuticals Rare Disease Calquence Airsupra Koselugo Calquence Airsupra Koselugo Datroway Baxfendy (baxdrostat) Ultomiris Datroway Baxfendy (baxdrostat) Ultomiris Enhertu Breztri Enhertu Breztri Approved Etcamah (camizestrant) Fasenra med
Slide 5: Project movements since Q1 2026 update
Project movements since Q1 2026 update New to Phase I New to Phase II New to pivotal trial New to registration NME NME NME NME AZD4916 AZD8965 elecoglipron efzimfotase alfa Hickory (301), Mulberry healthy volunteers inhibition of arginase enzyme idiopathic oral GLP-1 receptor agonist weight (305), C
Slide 6: Project movements since Q1 2026 update
Project movements since Q1 2026 update Removed from Phase I Removed from Phase II Removed from Phase III Approved /removed from registration NME NME Life-cycle management NME AZD2068 AZD4604 Imfinzi + domvanalimab following cCRT Baxfendy (baxdrostat) BaxHTN EGFR/cMET actinium radioconjugate solid in
Slide 7: Q2 2026 Oncology new molecular entity pipeline
Q2 2026 Oncology new molecular entity1 pipeline Phase I Phase II Phase III 22 New Molecular Entities 17 New Molecular Entities 26 New Molecular Entities surovatamig IPH5201 + Imfinzi# Imfinzi +/- oleclumab +/- monalizumab PACIFIC-9# JAB-23E73# Etcamah (camizestrant) Etcamah (camizestrant) + palbocic
Slide 8: Q2 2026 Oncology lifecycle management pipeline
Q2 2026 Oncology lifecycle management1 pipeline Phase I Phase II Phase III 0 Projects 3 Projects 28 Projects Enhertu (platform) DESTINY-Breast07# Calquence + R-CHOP ESCALADE Datroway TROPION-Lung17 # Datroway + Imfinzi + CTx AVANZAR# HER2 TOP1i ADC HER2+ breast cancer BTKi + R-CHOP 1L DLBCL TROP2 TO
Slide 9: Q2 2026 BioPharmaceuticals new molecular entity pipeline
Q2 2026 BioPharmaceuticals new molecular entity1 pipeline Phase I Phase II Phase III Under review 12 New Molecular Entities 17 New Molecular Entities 7 New Molecular Entities 1 New Molecular Entity balcinrenone/dapagliflozin surovatamig balcinrenone/dapagliflozin opemalirsen MR antagonist/modulator
Slide 10: Q2 2026 BioPharmaceuticals life cycle management pipeline
Q2 2026 BioPharmaceuticals life cycle management1 pipeline Phase I Phase II Phase III Under review 0 Projects 0 Projects 11 Projects 0 Projects Baxfendy (baxdrostat) BaxPA aldosterone synthase inhibitor primary aldosteronism Baxfendy (baxdrostat)/dapagliflozin aldosterone synthase inhibitor and reve
Slide 11: Q2 2026 Rare Disease pipeline
Q2 2026 Rare Disease pipeline1 Phase I Phase II Phase III Under review 5 Projects 3 Projects 4 Projects 4 Projects tarperprumig I TRANSCEND ALXN2080 cliramitug DepleTTR-CM# anselamimab CARES anti-properdin (complement factor P), dual-binding, oral factor D healthy participants TTR depleter transthyr
Slide 12: Active designations in our pipeline
Active designations in our pipeline 3 5 14 3 22 Priority Review Breakthrough / PRIME 1 / Sakigake 2 Fast Track Qualified Infectious Disease product Orphan Datroway 1L TNBC TROPION-Breast02 AZD0292 Psl-PcrV N3Y NCFBE (EU) AZD0292 Psl-PcrV N3Y NCFBE (US) AZD0292 Psl-PcrV N3Y NCFBE (US) Fasenra HES NAT
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 • FPCD: Q1 2023 AV
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 Muscle-invasive bladder cancer eligible 1063 • Arm 1: Imfinzi in combination with gemcitabine + cisplatin, • Co-primary endpo
Slide 28: Lynparza (PARP inhibitor)
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: Orpathys (savolitinib, MET inhibitor)
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 30: 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 31: Truqap (capivasertib, AKT inhibitor)
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 32: zadavotide guraxetan (PSMA radioconjugate)
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 33: puxitatug samrotecan (B7-H4 ADC)
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 34: 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 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 III 1L PD-L1 TC ≥1% squamous NSCLC 1160 • Randomised, double-blind, multicentre • Primary endpoints: PFS, OS • FPCD: Q4 2
Slide 36: 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 37: 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 38: saruparib (PARP1 inhibitor)
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 39: saruparib (PARP1 inhibitor)
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 40: sonesitatug vedotin (CLDN18.2 ADC)
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 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 III B-cell non-Hodgkin lymphoma, diffuse 420 • Multi-centre, randomised • Primary: PFS • Data anticipated: >202
Slide 42: 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/II Mature B-cell malignancies (chronic 180 • Multi-centre, open-label trial • Primary endpoints: DLT and safe
Slide 43: torvutatug samrotecan (anti-FRα ADC)
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 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 III High-risk locally advanced cervical 800 • Randomised, double-blind, placebo-controlled, multi-centre • Primary endpo
Slide 45: 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 46: AZD0120 (CAR-T): 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 47: Oncology: Early-stage development
Oncology Early-stage development 47 47
Slide 48: 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 49: 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 50: NT-112 (KRAS G12D 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 51: NT-175 (TP53 R175H 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 52: 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 53: tilatamig samrotecan (EGFR-cMET ADC)
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 54: 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 55: 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 56: 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 57: 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 58: 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 59: 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 60: 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 61: 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 62: AZD4360 (CLDN18.2 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 63: AZD4512 (CD22 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 64: 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 65: 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 66: 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 67: 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 68: 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 69: 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 70: 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 71: 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 72: 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 73: 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 74: BioPharmaceuticals
BioPharmaceuticals Approved medicines and late-stage pipeline 74 74
Slide 75: 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 76: 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 77: Wainua (eplontersen): 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 78: 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 79: 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 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 • Data anticipated: >20
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 Q
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 33 • Multi-centre, single-dose, non-randomised, open-label, • Primary endpoint: PK parameters • FPCD: Q1
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 32 • Open-label, fixed-sequence, two-part, single-centre trial • Part A and B: • FPCD: Q2 2026 NCT07444424
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 78 • Open-label, fixed sequence trial to assess the PK of • Primary endpoint: PK parameters • FPCD:
Slide 87: 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 88: 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 89: 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 90: 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 91: 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 r/r EGPA on 140 • Arm 1: Fasenra 30mg Q4W s.c. • Primary endpoint: proportion of • FPCD: Q4 2019 MANDARA cort
Slide 92: Saphnelo: lupus
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 93: Saphnelo: sclerosis and 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 94: 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 95: Tezspire: severe uncontrolled asthma
Approved medicines Late-stage development Tezspire (TSLP mAb) Oncology Early development Severe, uncontrolled asthma Trial Population Patients Design Endpoints Status Phase III Severe asthma; age 18 to 80 405 • Randomised, double-blind, placebo controlled • Primary endpoint: annual asthma • FPCD: Q3
Slide 96: 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 97: 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 98: 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 99: 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 100: 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 101: BioPharmaceuticals: Early-stage development
BioPharmaceuticals Early-stage development 101 101
Slide 102: 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 103: 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 I Healthy adults 44 • Randomised, open-label, 3 or 4-period, single-dose, two- • Pr
Slide 104: 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 105: 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 106: 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 107: 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 108: 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 109: 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 110: 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 111: 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 112: 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 113: 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 114: 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 115: 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 116: 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 117: 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 Overweight or obese 142 • Randomised, single-blind, placebo-controlled trial with • Primary e
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
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 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)
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: 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 125: AZD4916
Approved medicines Late-stage development AZD4916 Oncology Early development Respiratory Trial Population Patients Design Endpoints Status Phase I Healthy volunteers 160 • Randomised, single-blind, placebo controlled SAD/MAD • Primary endpoints: Safety and • Data anticipated: H2 2027 NCT06951880 tri
Slide 126: 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 (SLE) or • Part 1:
Slide 127: 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 128: 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 129: 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 130: AZD0292 (Psl-PcrV 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 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 (S aureus 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 133: Rare Disease
Rare Disease Approved medicines and late-stage pipeline 133 133
Slide 134: Koselugo (selumetinib, MEK inhibitor)
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 135: 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 136: 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 137: 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 138: cliramitug (TTR depleter)
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 139: 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 140: 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 141: 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 142: Rare Disease: Early-stage development
Rare Disease Early-stage development 142 142
Slide 143: 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 144: 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 145: ALXN2030 (siRNA targeting 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 146: 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 147: 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 148: 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 149: 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 150: Glossary – 1 of 5
NEXT NEXT Glossary – 1 of 5 14C Carbon 14 ASO Antisense oligonucleotide BTKi Bruton's tyrosine kinase inhibitor 1L, 2L, 3L 1st-, 2nd- or 3rd-line ATM Ataxia telangiectasia mutated kinase BVAS Birmingham Vasculitis Activity Score 5-FU 5-fluorouracil ATR Ataxia telangiectasia and Rad3-related protein
Slide 151: Glossary – 2 of 5
BACK NEXT BACK NEXT Glossary – 2 of 5 CNS-PFS Central nervous system progression-free survival DoCR Durability of complete response FCR Fludarabine, cyclophosphamide and rituximab CompEx Composite endpoint for exacerbations DoR Duration of response FDC Fixed-dose combination COPD Chronic obstructive
Slide 152: Glossary – 3 of 5
BACK NEXT BACK NEXT Glossary – 3 of 5 HbA1c Glycated haemoglobin IA Investigator-assessed LCM Lifecycle management HCC Hepatocellular carcinoma IBD Inflammatory bowel disease LDH Lactate dehydrogenase HD High dose ICR Independent central review LDL-C Low-density lipoprotein cholesterol HDL-C High-de
Slide 153: Glossary – 4 of 5
BACK NEXT BACK NEXT Glossary – 4 of 5 MoA Mechanism of action NSCLC Non-small cell lung cancer PIK3CA Phosphatidylinositol-4,5-biphosphate 3-kinase catalytic subunit mPFS Median progression-free survival NSQ Non-squamous PK Pharmacokinetic MPO Myeloperoxidase NST Neoadjuvant systemic treatment PK/PD
Slide 154: Glossary – 5 of 5
BACK BACK Glossary – 5 of 5 QT Duration of ventricular electrical systole sK Serum potassium TNSALP Tissue-nonspecific alkaline phosphatase QTcF Corrected QT interval by Fredericia SLE Systemic lupus erythematosus TOP1i Topoisomerase 1 inhibitor r/r Relapsed/refractory SLL Small lymphocytic lymphoma