Slide 1: Clinical Trials Appendix
Clinical Trials Appendix FY 2025 Results Update 10 February 2026
Slide 2: Pipeline at a glance
Pipeline 197 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 125 43 CVRM | R&I | V&I new molecular entities regulatory approvals (NME) or major lifecycle in major markets manageme
Slide 3: Key upcoming pipeline catalysts: 2026 and 2027
Key upcoming pipeline catalysts: 2026 and 2027 Oncology BioPharmaceuticals Rare Disease H1 2026 H2 2026 2027 Calquence – CLL (1L fixed duration) (AMPLIFY) (US) Datroway – met. TNBC not candidate for IO (TROPION-Breast02) Enhertu – previously treated HER2+ solid tumours (DESTINY-PanTumour02) (EU) Enh
Slide 4: Clinical Trials Appendix: selected highlights
Clinical Trials Appendix: selected highlights Oncology BioPharmaceuticals Oncology Rare Disease AIRSUPRA™ Inhalation Aerosol DATROWAY® For Health Care Providers Approved medicines: (datopotamab deruxtecan-dlnk) is Now Koselugo® (selumetinib) | Official HCP Website key LCM Our Antisense Medicines | I
Slide 5: Project movements since Q3 2025 update
Project movements since Q3 2025 update New to Phase I New to Phase II New to pivotal trial New to registration NME NME NME NME AZD3632 ALXN2030 CONCORD torvutatug samrotecan (AZD5335) TREVI-OC-01 anselamimab CARES MENIN inhibitor haematological malignancies siRNA targeting complement C3 antibody ant
Slide 6: Project movements since Q3 2025 update
Project movements since Q3 2025 update Removed from Phase I Removed from Phase II Removed from Phase III Approved/removed from registration NME NME NME Life-cycle management AZD0233 AZD3427 ceralasertib + Imfinzi LATIFY Enhertu + pertuzumab DESTINY-Breast09# CX3CR1 dilated cardiomyopathy relaxin mim
Slide 7: Q4 2025 Oncology new molecular entity pipeline
Q4 2025 Oncology new molecular entity 1 pipeline Phase I Phase II Phase III 23 New Molecular Entities 19 New Molecular Entities 23 New Molecular Entities surovatamig Imfinzi +/- oleclumab +/- monalizumab PACIFIC-9# surovatamig SOUNDTRACK-E cami zestrant FPI-2265# cami zestrant + palbociclib SERENA-4
Slide 8: Q4 2025 Oncology lifecycle management pipeline
Q4 2025 Oncology lifecycle management1 pipeline Phase I Phase II Phase III Under review 0 Projects 9 Projects 29 Projects 4 Projects Enhertu DESTINY-PanTumor03 (China)# Datroway + Imfinzi + CTx AVANZAR# Datroway + Imfinzi TROPION-Breast04# Datroway TROPION-Breast02# HER2 TOP1i ADC HER2 expressing so
Slide 9: Q4 2025 BioPharmaceuticals new molecular entity pipeline
Q4 2025 BioPharmaceuticals new molecular entity 1 pipeline Phase I Phase II Phase III 14 New Molecular Entities 17 New Molecular Entities 8 New Molecular Entities AZD0120 balcinrenone/dapagliflozin surovatamig atuliflapon balcinrenone/dapagliflozin CD19/BCMA CAR-T systemic lupus MR antagonist/modula
Slide 10: Q4 2025 BioPharmaceuticals life cycle management pipeline
Q4 2025 BioPharmaceuticals life cycle management1 pipeline Phase I Phase II Phase III Under review 0 Projects 0 Projects 9 Projects 2 Projects Breztri/Trixeo ATHLOS Breztri/Trixeo (PT010) KALOS LOGOS LABA/LAMA/ICS COPD cardiopulmonary exercise trial LABA/LAMA/ICS asthma Breztri/Trixeo THARROS# Fasen
Slide 11: Q4 2025 Rare Disease pipeline
Q4 2025 Rare Disease pipeline 1 Phase I Phase II Phase III Under review 4 Projects 4 Projects 7 Projects 2 Projects tarperprumig I TRANSCEND ALXN2080 cliramitug DepleTTR-CM# anselamimab CARES anti-properdin (complement factor P), dual-binding, oral factor D healthy volunteers TTR depleter transthyre
Slide 12: Active designations in our pipeline
Active designations in our pipeline 3 6 13 3 20 Priority Review Breakthrough / PRIME 1 / Sakigake 2 Fast Track Qualified infectious disease product Orphan baxdrostat HTN (US) AZD0292 Psl-PcrV N3Y NCFBE (EU) AZD0292 Psl-PcrV N3Y NCFBE (US) AZD0292 Psl-PcrV N3Y NCFBE (US) Fasenra HES NATRON (US) Datro
Slide 13: Oncology: approved medicines and late-stage pipeline
Oncology: approved medicines and late-stage pipeline 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: IRC PFS (Arm 1 vs. • FPCD: Q1 2019 AMPLIFY (ACE-CL
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 600 • 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 NSCLC 1350 • Arm 1: Datroway + Imfinzi + carboplatin • Co-primary endpoints: PFS and OS in • FPCD: Q1 2023 AVANZAR •
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 EGFRm locally advanced or metastatic 562 • Arm 1: Tagrisso + Datroway • Primary endpoint: PFS (BICR) • FPCD: Q2 2024 TRO
Slide 18: Datroway: NSCLC
Approved medicines Late-stage development Datroway (datopotamab deruxtecan, TROP2 ADC) Oncology Early development NSCLC Trial Population Patients Design Endpoints Status Phase I Advanced or metastatic NSCLC 155 • Open-label, two-part (dose escalation, dose expansion), • Primary endpoints: DLT and sa
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 II Endometrial cancer, gastric cancer, 606 • Sub-study 1 (endometrial cancer); • Primary endpoints: ORR and safety •
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 HER2-positive, unresectable and/or 600 • Randomised, open-label, parallel assignment • Primary endpoint: PFS • FPC
Slide 21: 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 early non- 927 • Randomised, open-label, parallel assignment • Primary endpoint: pCR • FPC
Slide 22: 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 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 III Advanced treatment-naïve HER2- 620 • Arm A: Enhertu + rilvegostomig • Primary endpoint: OS • FPCD: Q3 2024 DESTINY
Slide 24: 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-mutated, unresectable and/or 152 • Randomised, parallel group assignment • Primary endpoint: ORR • FPCD: Q1 20
Slide 25: 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 Ib HER2-over expressing, unresectable 244 • Non-randomised, parallel group assignment • Primary endpoint: safety and R
Slide 26: Imfinzi: gastrointestinal cancer
Approved medicines Late-stage development Imfinzi (PD-L1 mAb) Oncology Early development Gastrointestinal cancer 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 2019 EMERALD-1
Slide 27: Imfinzi: lung cancer
Approved medicines Late-stage development Imfinzi (PD-L1 mAb) Oncology Early development Lung cancer Trial Population Patients Design Endpoints Status Phase III Adjuvant NSCLC patients, Stage Ib 1415 • Arm 1: Imfinzi mg/kg i.v. Q4W x 12 months • Primary endpoint: DFS • FPCD: Q1 2015 ADJUVANT BR.31 (
Slide 28: Imfinzi: lung cancer
Approved medicines Late-stage development Imfinzi (PD-L1 mAb) Oncology Early development Lung cancer Trial Population Patients Design Endpoints Status Phase II NSCLC, patients who progressed on an 531 • Open-label, biomarker-directed, multi-centre trial • Primary endpoint: ORR • FPCD: Q1 2018 HUDSON
Slide 29: Imfinzi: other cancer
Approved medicines Late-stage development Imfinzi (PD-L1 mAb) Oncology Early development Other cancer Trial Population Patients Design Endpoints Status Phase III Muscle-invasive bladder cancer eligible 1063 • Arm 1: Imfinzi in combination with gemcitabine + cisplatin, • Co-primary endpoints: pCR and
Slide 30: Lynparza: Imfinzi combinations
Approved medicines Late-stage development Lynparza (PARP inhibitor) Oncology Early development Imfinzi combinations Trial Population Patients Design Endpoints Status Phase III 1L advanced and recurrent endometrial 805 • Arm 1: chemotherapy + Imfinzi placebo followed by Imfinzi • Primary endpoint: PF
Slide 31: Lynparza: other cancer
Approved medicines Late-stage development Lynparza (PARP inhibitor) Oncology Early development Other cancer Trial Population Patients Design Endpoints Status Phase III BRCAwt advanced ovarian cancer, 1L 366 • Arm 1: Lynparza BID 24-month duration • Primary endpoints: PFS (BRCAwt HRD- • FPCD: Q3 2021
Slide 32: Orpathys: gastric cancer
Approved medicines Late-stage development Orpathys (savolitinib, MET inhibitor) Oncology Early development Gastric cancer Trial Population Patients Design Endpoints Status Phase II Locally advanced or metastatic gastric 75 • Single-arm, multi-cohort, multi-centre, open-label trial • Primary endpoint
Slide 33: 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 34: Tagrisso: NSCLC combinations
Approved medicines Late-stage development Tagrisso (highly-selective, irreversible EGFR inhibitor) Oncology Early development NSCLC, combinations Trial Population Patients Design Endpoints Status Phase III Locally advanced or metastatic NSCLC 250 • Arm 1: Tagrisso + Orpathys • Primary endpoint: PFS
Slide 35: Truqap: breast and prostate cancer
Approved medicines Late-stage development Truqap (capivasertib, AKT inhibitor) Oncology Early development Breast cancer and prostate cancer Trial Population Patients Design Endpoints Status Phase III mCRPC prostate cancer 1033 • Double-blind, randomised, comparative trial • Primary endpoint: OS • FP
Slide 36: camizestrant: breast cancer
Approved medicines Late-stage development camizestrant (next-generation oral SERD) Oncology Early development Breast cancer Trial Population Patients Design Endpoints Status Phase III HR+ HER2-negative advanced breast 1370 • Randomised, double-blind, comparative trial • Primary endpoint: PFS • FPCD:
Slide 37: camizestrant: breast cancer
Approved medicines Late-stage development camizestrant (next-generation oral SERD) Oncology Early development Breast cancer Trial Population Patients Design Endpoints Status Phase I HR+ HER2-negative advanced breast 396 • Escalation phase: open-label multi-centre trial • Primary endpoints: safety an
Slide 38: ceralasertib: multiple cancers
Approved medicines Late-stage development ceralasertib (AZD6738, ATR inhibitor) Oncology Early development Multiple cancers Trial Population Patients Design Endpoints Status Phase III Post-IO NSCLC 594 • Double-arm randomised • Primary endpoint: OS • FPCD: Q4 2022 LATIFY • Arm 1: ceralasertib + Imfi
Slide 39: puxitatug samrotecan: solid tumours
Approved medicines Late-stage development puxitatug samrotecan (AZD8205, B7H4 ADC) Oncology Early development Solid tumours Trial Population Patients Design Endpoints Status Phase III B7-H4 selected 2-3L endometrial cancer 700 • Randomised, single-open label • Primary endpoints: PFS, OS • Data antic
Slide 40: 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 1L Advanced BTC 1100 • Randomised, open label, Global, Multicenter • Primary endpoint: OS • FPCD: Q4 202
Slide 41: 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 squamous NSCLC 1L patients whose 880 • Randomised, double-blind, multicenter, • Primary endpoints: PFS, OS • FPCD: Q4
Slide 42: 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 NSCLC 210 • Open-label, dose escalation and dose expansion trial • Primary endpoints (Part A): safety, • FPCD: Q4 20
Slide 43: 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 III adjuvant BTC with curative intent 750 • Randomised, double-blind, placebo-controlled, multicenter • Primary endpoin
Slide 44: 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 Gastric cancer 360 • Open-label gastric platform trial • Primary endpoints: safety and efficacy • FPCD: Q1 2023 GEM
Slide 45: saruparib: solid tumours
Approved medicines Late-stage development saruparib (AZD5305, PARP1 inhibitor) Oncology Early development Solid tumours Trial Population Patients Design Endpoints Status Phase III BRCA1, BRCA2, or PALB2m, HR-positive, 500 • Randomised, open-label trial • Primary endpoint: PFS (BICR) • FPCD: Q3 2024
Slide 46: 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/II Saruparib mono as neoadj. treatment in 30 • A Master Protocol Phase I/II Study to Investigate Biomarker • • FPCD: Q4 20
Slide 47: sonesitatug vedotin: solid tumours
Approved medicines Late-stage development sonesitatug vedotin (AZD0901, CLDN18.2 MMAE ADC) Oncology Early development Solid tumours Trial Population Patients Design Endpoints Status Phase III 2L+ advanced or metastatic gastric or 572 • Multi-centre, open-label, sponsor-blinded, randomised trial • Pr
Slide 48: surovatamig: haematologic malignancies
Approved medicines Late-stage development surovatamig (AZD0486, 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 anticipa
Slide 49: 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 platinum- 1100 • Randomised, open-label • Primary endpo
Slide 50: 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 51: 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 Colorectal Cancer (mCRC) 120 • Platform, randomised, open-label, multicenter, global trial • Primary endpoints: PFS,
Slide 52: 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 I Advanced solid tumours 400 • Open-label, dose escalation and dose expansion trial • Primary endpoints (escalation): sa
Slide 53: Oncology: early-stage development
Oncology: early-stage development 53
Slide 54: FPI-2265: prostate cancer
Approved medicines Late-stage development FPI-2265 (PSMA radioconjugate) Oncology Early development Prostate cancer Trial Population Patients Design Endpoints Status Phase II PSMA-positive mCRPC previously 100 • Open-label, randomised, multi-centre trial • Primary endpoints: PSA50 and safety • FPCD:
Slide 55: IPH5201: solid tumours
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 56: NT-112: solid tumours
Approved medicines Late-stage development NT-112 (KRAS G12D specific TCR) Oncology Early development Solid tumours Trial Population Patients Design Endpoints Status Phase I Unresectable, advanced and/or 24 • Open-label, single-arm, multi-centre trial with dose • Primary endpoints: incidence of DLTs,
Slide 57: NT-125: solid tumours
Approved medicines Late-stage development NT-125 (autologous, multi-specific neoantigen-targeting TCR-T) Oncology Early development Solid tumours Trial Population Patients Design Endpoints Status Phase I Adults with recurrent or metastatic 42 • Open-label, single-arm, single-centre trial with dose •
Slide 58: NT-175: multiple cancers
Approved medicines Late-stage development NT-175 (TP53-armored TCR) Oncology Early development Multiple cancers Trial Population Patients Design Endpoints Status Phase I Unresectable, advanced, and/or 162 • Open-label, single-arm, multi-centre trial with dose • Primary endpoint: Incidence of DLTs,
Slide 59: tilatamig samrotecan: lung cancer
Approved medicines Late-stage development tilatamig samrotecan (AZD9592, 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 (
Slide 60: AZD0120: blood cancers
Approved medicines Late-stage development AZD0120 (GC012F, autologous anti-CD19 and anti-BCMA CAR-T) Oncology Early development Blood cancers Trial Population Patients Design Endpoints Status Phase II Relapsed/refractory multiple myeloma 20 • Open-label, multi-centre, non-randomised trial • Primary
Slide 61: AZD0305: blood cancers
Approved medicines Late-stage development AZD0305 (GPRC5D 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: occurrence of • FPCD: Q4 2023 NCT0
Slide 62: AZD0516: prostate cancer
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: safety and tolerability • FPCD: Q4 2025 SEACLIFF opt
Slide 63: AZD0754: solid tumours
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 Metastatic castration resistance 60 • Open-label, single-arm, multi-centre trial with dose • Primary endpoints (Ph
Slide 64: AZD2068: solid tumours
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 65: AZD2284: prostate cancer
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 66: AZD2962: blood cancers
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, mutli-centre • Primary endpoints: DLT, AEs, duration • Data anticipated: >2027 NCT07
Slide 67: AZD3470: solid tumours and blood cancer
Approved medicines Late-stage development AZD3470 (PRMT5) Oncology Early development Solid tumours and blood cancer Trial Population Patients Design Endpoints Status Phase I/II MTAP-deficient advanced solid tumours 234 • Open-label, multi-centre • Arm 1: Primary endpoints: safety and • FPCD: Q1 2024
Slide 68: AZD3632: blood cancers
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 is a dose escalation of AZD3632 monotherapy. • Primary endpoints: safety and • FPCD: Q1
Slide 69: AZD4360: solid tumours
Approved medicines Late-stage development AZD4360 (CLDN18.2 ADC) Oncology Early development Solid tumours Trial Population Patients Design Endpoints Status Phase I/II Histologically confirmed advanced or 117 • Open-label, multi-centre trial with FIH modular protocol • Primary endpoints: safety • FPC
Slide 70: AZD4512: blood cancers
Approved medicines Late-stage development AZD4512 (CD22 ADC) Oncology Early development Blood cancers Trial Population Patients Design Endpoints Status Phase I/II Acute Lymphoblastic Leukemia (ALL) 83 • Modular phase I/II, open-label multi-centre study • Module 1: • FPCD: Q4 2025 ALLight • Module 1:
Slide 71: AZD5492: blood cancers
Approved medicines Late-stage development AZD5492 (CD20 TITAN TCE) Oncology Early development Blood cancers Trial Population Patients Design Endpoints Status Phase I CLL, MCL, LBCL, FL 176 • Module 1: AZD5492 monotherapy • Primary endpoints: safety and • FPCD: Q3 2024 TITANIUM • AZD5492 monotherapy
Slide 72: AZD5863: solid tumours
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 solid tumours 280 • Part A: dose escalation phase to determine the safety, • Primary endpoint
Slide 73: AZD6621: prostate cancer
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 dose escalation and dose • Primary: safety, tolerability • FPCD: Q4 2025 ACTI
Slide 74: AZD6750: solid tumours
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 75: AZD7003: hepatocellular carcinoma
Approved medicines Late-stage development AZD7003 (GPC3 CAR-T) Oncology Early development Hepatocellular carcinoma (HCC) Trial Population Patients Design Endpoints Status Phase I/II GPC3-positive advanced/recurrent HCC 121 • Open-label, single-arm, multi-centre trial with dose • Primary endpoints (P
Slide 76: AZD8421: breast cancer
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 77: AZD9574: solid tumours
Approved medicines Late-stage development 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: safety and •
Slide 78: AZD9750: prostate cancer
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 • Part A: monothera
Slide 79: AZD9793: solid tumours
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 80: BioPharmaceuticals: approved medicines and late-stage development
BioPharmaceuticals: approved medicines and late-stage development 80
Slide 81: 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 82: 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 Heart failure patients with 4800 • Randomised, double-blind, parallel-group, doub
Slide 83: baxdrostat: hypertension
Approved medicines Late-stage development baxdrostat (selective aldosterone synthase inhibitor) Oncology Early development Hypertension Trial Population Patients Design Endpoints Status Phase III Patients with resistant 218 • Arm 1: baxdrostat 2mg QD • Primary endpoint: effect of baxdrostat • FPCD:
Slide 84: baxdrostat: hypertension
Approved medicines Late-stage development baxdrostat (selective aldosterone synthase inhibitor) Oncology Early development Hypertension Trial Population Patients Design Endpoints Status Phase II Patients with uncontrolled 45 • Arm 1: baxdrostat 2mg QD • Primary endpoint: individual cortisol • FPCD:
Slide 85: baxdrostat: primary aldosteronism
Approved medicines Late-stage development baxdrostat (selective aldosterone synthase inhibitor) Oncology Early development Primary aldosteronism Trial Population Patients Design Endpoints Status Phase III Primary aldosteronism 180 • Multicentre, randomised, double-blind, placebo-controlled, • Primar
Slide 86: 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 87: laroprovstat: dyslipidaemia
Approved medicines Late-stage development laroprovstat (AZD0780, PCSK9 inhibitor) Oncology Early development Dyslipidaemia Trial Population Patients Design Endpoints Status Phase III Heterozygos familial 405 • Randomised, double-blind, placebo-controlled, parallel- • Primary endpoint: Relative chang
Slide 88: 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 89: 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 90: 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-center, parallel-group • Primary endpoint: severe asthma • FPCD: Q2
Slide 91: 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 92: 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 93: 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 94: 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 • Arm 1: 300mg Saphnelo i.v. Q4W • Primary endpoint: BICLA at Week 52 • FPCD: Q4 2021 A
Slide 95: 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 306 • Arm 1: Saphnelo s.c. • Primary endpoint: CRISS-25 at Week • FPCD: Q4 2023
Slide 96: 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 97: 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 • Arm 1: Tezspire s.c. • Primary endpoint: annual asthma • FPCD: Q3 2019 DIRECTION years •
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 1132 • Randomised, double-blind, placebo-controlled, parallel- • Primary endpoint: annualised rate of • FPCD: Q
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 2870 • Randomised, double-blind, placebo-controlled, parallel • Primary endpoint: pr
Slide 101: Next-generation propellant pMDI
Approved medicines Late-stage development Next-generation propellant Oncology Early development pMDI Trial Population Patients Design Endpoints Status Phase III Mucociliary clearance in 30 • Randomised, double-blind, multi-site, two-way crossover • Primary endpoint: change from • FPCD: Q2 2023 NCT05
Slide 102: 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: Q4 2022 NCT055694
Slide 103: Beyfortus: infection
Approved medicines Late-stage development Beyfortus (nirsevimab, RSV mAb-YTE) Oncology Early development Infection Trial Population Patients Design Endpoints Status Phase III Healthy infants (born 29 800 • Randomised, double-blind, placebo-controlled • Primary endpoint: efficacy • FPCD: Q4 2021 CHIM
Slide 104: Kavigale: COVID-19
Approved medicines Late-stage development Kavigale (sipavibart, SARS-CoV-2 LAAB) Oncology Early development COVID-19 Trial Population Patients Design Endpoints Status Phase III Phase I: healthy adults; age 18 3200 • 2 parts (Phase I: sentinel safety cohort and Phase III: main • Primary endpoints (Ph
Slide 105: BioPharmaceuticals: early-stage development
5 BioPharmaceuticals: early-stage development 105
Slide 106: elecoglipron: type 2 diabetes, obesity
Approved medicines Late-stage development elecoglipron (AZD5004, oral GLP-1 RA) Oncology Early development Type 2 diabetes, obesity Trial Population Patients Design Endpoints Status Phase IIb Type 2 diabetes 406 • Arm 1: AZD5004 tablet • Primary endpoint: change in HbA1c • FPCD: Q4 2024 SOLSTICE • A
Slide 107: elecoglipron: type 2 diabetes, obesity
Approved medicines Late-stage development elecoglipron (AZD5004, oral GLP-1 RA) Oncology Early development Type 2 diabetes, obesity Trial Population Patients Design Endpoints Status Phase I Healthy volunteers or 16 • Multi-centre, single-dose, non-randomised, open-label, • Primary endpoint: PK param
Slide 108: opemalirsen: chronic kidney disease
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 109: AZD0233: dilated cardiomyopathy
Approved medicines Late-stage development AZD0233 (oral CX3CR1) Oncology Early development Dilated cardiomyopathy Trial Population Patients Design Endpoints Status Phase I Healthy volunteers 96 • Randomised, SAD/MAD dose escalating trial • Primary endpoints: safety and • FPCD: Q2 2024 NCT06381466 to
Slide 110: AZD1613: ADPKD
Approved medicines Late-stage development AZD1613 (PAPPA-1 mAb) Oncology Early development ADPKD Trial Population Patients Design Endpoints Status Phase I Healthy volunteers 136 • Randomised, single-blind, placebo-controlled single and • Primary endpoint: safety and • FPCD: Q2 2025 NCT06995820 multi
Slide 111: AZD1705: dyslipidaemia
Approved medicines Late-stage development AZD1705 (Angptl3 inhibitor) Oncology Early development Dyslipidaemia Trial Population Patients Design Endpoints Status Phase I Dyslipidaemia 112 • Part A: single dose of AZD1705 with an in-clinic period of 3 • Primary endpoints: AEs and SAEs • FPCD: Q1 2024
Slide 112: AZD2389: MASH
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 113: AZD3427: heart failure
Approved medicines Late-stage development AZD3427 (relaxin) Oncology Early development Heart failure Trial Population Patients Design Endpoints Status Phase II HF and pulmonary 260 • Randomised, double-blind, placebo-controlled, multi-centre • Primary endpoint: change in PVR from • FPCD: Q2 2023 Re-
Slide 114: 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 115: AZD4063: dilated cardiomyopathy
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 116: AZD4144: cardiorenal disease
Approved medicines Late-stage development AZD4144 (NLRP3) Oncology Early development Cardiorenal disease Trial Population Patients Design Endpoints Status Phase I Healthy participants 95 • Randomised, single-blind, placebo-controlled, SAD/MAD • Primary endpoints: safety and • FPCD: Q4 2023 NCT061227
Slide 117: AZD4248: CKD
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 118: AZD4954: dyslipidaemia
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 119: AZD5462: heart failure
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 120: AZD6234: obesity with related co-morbidities
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 121: AZD6234: obesity with related co-morbidities
Approved medicines Late-stage development AZD6234 (selective amylin receptor agonist) Oncology Early development Obesity with related co-morbidities Trial Population Patients Design Endpoints Status Phase I Participants include those 48 • Phase I multicentre, single-dose, non-randomised, open- • com
Slide 122: AZD9550: MASH, obesity
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 123: 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 124: atuliflapon: asthma
Approved medicines Late-stage development atuliflapon (FLAP inhibitor) Oncology Early development Asthma Trial Population Patients Design Endpoints Status Phase IIa Patients with moderate-to- 666 • Randomised, placebo-controlled, double-blind, multi-centre • Primary endpoint: time to first • FPCD: Q
Slide 125: surovatamig: RA, SLE
Approved medicines Late-stage development surovatamig (AZD0486, CD19/CD3 T-cell engager) Oncology Early development RA, SLE Trial Population Patients Design Endpoints Status Phase I Adult Participants With 48 • Open-label Multicenter study • Primary endpoints: safety and • FPCD: Q1 2026 NCT07201558
Slide 126: AZD0120: autoimmune
Approved medicines Late-stage development AZD0120 (GC012F, autologous anti-CD19 and anti-BCMA CAR-T) Oncology Early development autoimmune Trial Population Patients Design Endpoints Status Phase Ib Adult participants with 27 • Open-label, multi-center, parallel-assignment, multi-cohort • Primary: In
Slide 127: AZD0120: neurology
Approved medicines Late-stage development AZD0120 (GC012F, autologous anti-CD19 and anti-BCMA CAR-T) Oncology Early development Neurology Trial Population Patients Design Endpoints Status Phase Ib Adults with refractory active 24 • Open-label, multi-center, parallel-assignment, randomized • Primary:
Slide 128: AZD0120: SLE
Approved medicines Late-stage development AZD0120 (GC012F, autologous anti-CD19 and anti-BCMA CAR-T) Oncology Early development SLE Trial Population Patients Design Endpoints Status Phase Ib/II Refractory systemic lupus 24 • Single-arm, open-label, multi-center trial • Primary endpoints (Phase I): s
Slide 129: AZD1163: rheumatoid arthritis
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 • A 24-week multicentre, double-blind, 4-arm, randomised • Primary: Change
Slide 130: AZD4604: asthma
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 131: AZD5492: SLE
Approved medicines Late-stage development AZD5492 (CD20 TITAN TCE) Oncology Early development SLE Trial Population Patients Design Endpoints Status Phase I Systemic lupus 70 • Open-label, multii-centre • Primary endpoints: safety and • Data anticipated: 2027 TITAN erythematosus (SLE) or • Part 1: Si
Slide 132: AZD6793: COPD
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 133: AZD6912: rheumatoid arthritis
Approved medicines Late-stage development AZD6912 (siRNA) Oncology Early development Rheumatoid arthritis Trial Population Patients Design Endpoints Status Phase I Healthy volunteers 40 • Randomised, double-blind, placebo-controlled SAD trial • Primary endpoint: incidence of AEs • FPCD: Q4 2023 NCT0
Slide 134: AZD7798: Crohn's disease
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 107 • Randomised, double-blind, placebo-controlled trial • Primary endpoint: Crohn's Disease • FPCD: Q4
Slide 135: AZD8630: asthma
Approved medicines Late-stage development AZD8630 (inhaled TSLP) Oncology Early development Asthma Trial Population Patients Design Endpoints Status Phase II Adults with uncontrolled 516 • Randomised, placebo-controlled, double-blind, dose range- • Primary endpoint: time to first • FPCD: Q3 2024 LEV
Slide 136: AZD8965: IPF
Approved medicines Late-stage development AZD8965 (arginase enzyme inhibitor) Oncology Early development IPF Trial Population Patients Design Endpoints Status Phase I Healthy volunteers 163 • Randomised, single-blind, SAD/MAD, placebo-controlled, • Primary endpoints (Part 1, 2, 3): safety • FPCD: Q3
Slide 137: mRNA VLP vaccine: COVID-19
Approved medicines Late-stage development mRNA VLP vaccine Oncology Early development COVID-19 Trial Population Patients Design Endpoints Status Phase I Healthy volunteers ≥18+ with 240 • Arm 1: dose 1 via i.m. injection AZD9838 in 18-64-year-olds • Primary endpoints: safety as measured • FPCD: Q4 2
Slide 138: 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 patients ≥ 12 435 • Randomized, double-blind, placebo-controlled, parallel, • Primary: efficacy • FPCD:
Slide 139: AZD5148: Clostridium difficile
Approved medicines Late-stage development AZD5148 (anti-TcdB mAb) Oncology Early development Clostridium difficile Trial Population Patients Design Endpoints Status Phase IIb ≥ 18 years, with a qualifying C. 230 • Randomized, Double-blind, Placebo-controlled • Primary endpoint: efficacy • FPCD: Q4 2
Slide 140: 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 141: Rare Disease: approved medicines and late-stage development
1 Rare Disease: approved medicines and late-stage development 141
Slide 142: Beyonttra: ATTR-CM
Approved medicines Late-stage development Beyonttra (acoramidis, ALXN2060) Oncology Early development ATTR-CM Trial Population Patients Design Endpoints Status Phase III ATTR-CM 22 • Arm 1: 800mg Beyonttra administered twice daily • Primary endpoint: change from • FPCD: Q4 2020 ALXN2060-TAC-302 • Ja
Slide 143: Koselugo: neurofibromatosis type 1, solid tumours
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 Adult age ≥18 years with NF1 145 • Multi-centre, international trial with a parallel, randomi
Slide 144: Ultomiris: haematology, nephrology, transplant
Approved medicines Late-stage development Ultomiris (anti-C5 mAb) Oncology Early development Haematology, nephrology, transplant Trial Population Patients Design Endpoints Status Phase III CSA-AKI 736 • Randomised, double-blind, placebo-controlled, muticentre • Primary endpoint: to assess the • FPCD
Slide 145: Ultomiris: neurology
Approved medicines Late-stage development Ultomiris (anti-C5 mAb) Oncology Early development Neurology Trial Population Patients Design Endpoints Status Phase II/III Neuromyelitis optica 12 • Arm 1: Ultomiris Q8W • Primary endpoint: change from • FPCD: Q3 2022 ALXN1210-NMO-317 spectrum disorder base
Slide 146: anselamimab: AL amyloidosis
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 147: cliramitug: amyloidosis
Approved medicines Late-stage development cliramitug (ALXN2220, TTR depleter) Oncology Early development Amyloidosis Trial Population Patients Design Endpoints Status Phase III ATTR-CM (wild-type and 1180 • Arm 1: cliramitug via i.v. infusion Q4W for at least 24 months • Primary endpoint: composite
Slide 148: 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 149: 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 150: gefurulimab: neurology, nephrology
Approved medicines Late-stage development gefurulimab (ALXN1720, anti-C5 dual-binding nanobody) Oncology Early development Neurology, nephrology Trial Population Patients Design Endpoints Status Phase III Generalised myasthenia 260 • Arm 1: weight-based maintenance treatment with • Primary endpoint:
Slide 151: Rare Disease: early-stage development
1 Rare Disease: early-stage development 151
Slide 152: 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 153: 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 154: ALXN2030: transplant
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 155: ALXN2350: BAG3-associated dilated cardiomyopathy
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 156: ALXN2420: acromegaly
Approved medicines Late-stage development ALXN2420 (GH receptor antagonist) Oncology Early development Acromegaly Trial Population Patients Design Endpoints Status Phase IIb Acromegaly 60 • A Phase 2, randomised, double-blinded, placebo-controlled, • Primary endpoint: Percentage change • Data antici
Slide 157: AZD0120: AL amyloidosis
Approved medicines Late-stage development AZD0120 (GC012F, 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:
Slide 158: AZD1390: solid tumours
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 159: Glossary – 1 of 5
NEXT NEXT Glossary – 1 of 5 14C Carbon 14 ASO Antisense oligonucleotide BTK Bruton's tyrosine kinase 1L, 2L, 3L 1st-, 2nd- or 3rd-line ATM Ataxia telangiectasia mutated kinase BTKi Bruton's tyrosine kinase 5-FU 5-fluorouracil ATR Ataxia telangiectasia and Rad3-related protein BVAS Birmingham Vasculi
Slide 160: Glossary – 2 of 5
BACK NEXT BACK NEXT Glossary – 2 of 5 CNS Central nervous system DNA Deoxyribonucleic acid ETA Endothelin A CNS-PFS Central nervous system progression-free survial dNCC Directly measured non-ceruloplasmin-bound copper ETA RA Endothelin receptor A antagonist CompEx Composite endpoint for exacerbation
Slide 161: Glossary – 3 of 5
BACK NEXT BACK NEXT Glossary – 3 of 5 GPRC5D G protein-coupled receptor, class C, group 5, member D HSD17B13 Hydroxysteroid 17-beta dehydrogenase 13 LA amylin Long-acting amylin GU Genitourinary HVPG Hepatic venous pressure gradient LAAB Long-acting antibody GYN Gynaecologic i Inhibitor LABA Long-ac
Slide 162: Glossary – 4 of 5
BACK NEXT BACK NEXT Glossary – 4 of 5 MI Myocardial infarction NME New molecular entity PFS Progression-free survival mL Millilitre NMOSD Neuromyelitis optica spectrum disorder PFS2 Time to second disease progression or death MM Multiple myeloma NP Nasal polyps PgR Progesterone receptor MMAE Monomet
Slide 163: Glossary – 5 of 5
BACK BACK Glossary – 5 of 5 QD Once daily SGLT2 Sodium-glucose transport protein 2 Tmax Time to reach maximum observed plasma concentration QID Four times per day SGLT2i Sodium/glucose cotransporter 2 inhibitor TNBC Triple negative breast cancer QOD Every other day SGRM Selective glucocorticoid rece