Slide 1: Clinical Trials Appendix
Clinical Trials Appendix 3Q 2025 Results Update 6 November 2025
Slide 2: Pipeline at a glance
Pipeline 198 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 122 36 CVRM | R&I | V&I new molecular entities regulatory approvals (NME) or major lifecycle in major markets manageme
Slide 3: Key upcoming pipeline catalysts: 2025 and 2026
Key upcoming pipeline catalysts: 2025 and 2026 Oncology BioPharmaceuticals Rare Disease H2 2025 H1 2026 H2 2026 Imfinzi – resectable early-stage gastric and GEJ cancers (MATTERHORN) Calquence – CLL (1L fixed duration) (AMPLIFY) (US) gefurulimab – generalised myasthenia gravis (PREVAIL) Saphnelo – SL
Slide 4: Clinical Trials Appendix: selected highlights
Clinical Trials Appendix: selected highlights BioPharmaceuticals Oncology Rare Disease BioPharmaceuticals Oncology Rare Disease AIRSUPRA™ Inhalation Aerosol For Health Care Providers DATROWAY® (datopotamab deruxtecan-dlnk) is Now Approved Approved medicines: Koselugo® (selumetinib) | Official HCP We
Slide 5: Project movements since Q2 2025 update
Project movements since Q2 2025 update New to Phase I New to Phase II New to pivotal trial New to registration NME NME NME NME ALXN2350 ALXN1920 AUTUMN puxitatug samrotecan Bluestar-Endometrial01 gefurulimab PREVAIL AAV gene therapy BAG3-associated dilated kidney-targeted factor H fusion protein pri
Slide 6: Project movements since Q2 2025 update
Project movements since Q2 2025 update Removed from Phase I Removed from Phase II Removed from Phase III Approved/removed from registration NME NME Life-cycle management Life-cycle Management AZD0022 AZD2693 Fasenra RESOLUTE# Koselugo KOMET# KRASG12D inhibitor solid tumours MASH resolution metabolic
Slide 7: Q3 2025 Oncology new molecular entity pipeline
Q3 2025 Oncology new molecular entity1 pipeline Phase I Phase II Phase III 24 New Molecular Entities 18 New Molecular Entities 21 New Molecular Entities surovatamig (AZD0486) surovatamig (AZD0486) SOUNDTRACK-E Imfinzi +/- oleclumab +/- monalizumab PACIFIC-9# camizestrant ceralasertib camizestrant +
Slide 8: Q3 2025 Oncology lifecycle management pipeline
Q3 2025 Oncology lifecycle management1 pipeline Phase I Phase II Phase III Under review 0 Projects 9 Projects 31 Projects 7 Projects Datroway + Imfinzi + CTx AVANZAR# Datroway + Imfinzi TROPION-Breast04# Enhertu (platform) DESTINY-Breast07# Calquence + R-CHOP ESCALADE Enhertu DESTINY-Breast09# TROP2
Slide 9: Q3 2025 BioPharmaceuticals new molecular entity pipeline
Q3 2025 BioPharmaceuticals new molecular entity1 pipeline Phase I Phase II Phase III 14 New Molecular Entities 15 New Molecular Entities 9 New Molecular Entities AZD0120 balcinrenone/dapagliflozin balcinrenone/dapagliflozin AZD0233 atuliflapon MR antagonist/modulator + SGLT2 inhibitor autologous ant
Slide 10: Q3 2025 BioPharmaceuticals life cycle management pipeline
Q3 2025 BioPharmaceuticals life cycle management1 pipeline Phase I Phase II Phase III Under review 0 Projects 0 Projects 9 Projects 3 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: Q3 2025 Rare Disease pipeline
Q3 2025 Rare Disease pipeline1 Phase I Phase II Phase III Under review 4 Projects 2 Projects 8 Projects 1 Projects ALXN2030 anselamimab CARES gefurulimab PREVAIL ALXN1920 AUTUMN novel anti-C5, dual binding, nanobody generalised siRNA targeting complement C3 nephrology kidney-targeted factor H fusion
Slide 12: Active designations in our pipeline
Active designations in our pipeline 3 7 12 3 17 Priority Review Breakthrough / PRIME1 / Sakigake2 Fast Track Qualified infectious disease product Orphan Enhertu HER2+ breast cancer 1L AZD0292 Psl-PcrV N3Y NCFBE (EU)1 AZD0292 Psl-PcrV N3Y NCFBE (US) AZD0292 Psl-PcrV N3Y NCFBE (US) Fasenra HES NATRON
Slide 13: Oncology: approved medicines and late-stage pipeline
Oncology: approved medicines and late-stage pipeline 13
Slide 14: Oncology: Calquence
Approved medicines Late-stage development Oncology Calquence (BTK inhibitor) Early development Blood cancers Trial Population Patients Design Endpoints Status CVRM Phase III Previously untreated CLL 981 • Arm 1; Calquence + venetoclax • Primary endpoint: IRC PFS (Arm 1 vs. • FPCD: Q1 2019 AMPLIFY (A
Slide 15: Oncology: Datroway breast cancer
Approved medicines Late-stage development Oncology Datroway (datopotamab deruxtecan, TROP2 ADC) Early development Breast cancer Trial Population Patients Design Endpoints Status CVRM Phase III Inoperable or metastatic HR+ HER2- 732 • Open-label, randomised trial • Primary endpoints: PFS (BICR) and O
Slide 16: Oncology: Datroway NSCLC
Approved medicines Late-stage development Oncology Datroway (datopotamab deruxtecan, TROP2 ADC) Early development NSCLC Trial Population Patients Design Endpoints Status CVRM Phase III 1L NSCLC 1280 • Arm 1: Datroway + Imfinzi + carboplatin • Co-primary endpoints: PFS and OS in • FPCD: Q1 2023 AVANZ
Slide 17: Oncology: Datroway NSCLC
Approved medicines Late-stage development Oncology Datroway (datopotamab deruxtecan, TROP2 ADC) Early development NSCLC Trial Population Patients Design Endpoints Status CVRM Phase III EGFRm locally advanced or metastatic 562 • Arm 1: Tagrisso + Datroway • Primary endpoint: PFS (BICR) • FPCD: Q2 202
Slide 18: Oncology: Datroway other cancers
Approved medicines Late-stage development Oncology Datroway (datopotamab deruxtecan, TROP2 ADC) Early development Other cancers Trial Population Patients Design Endpoints Status CVRM Phase II Endometrial cancer, gastric cancer, 606 • Sub-study 1 (endometrial cancer); • Primary endpoints: ORR and saf
Slide 19: Oncology: Enhertu breast cancer
Approved medicines Late-stage development Oncology Enhertu (trastuzumab deruxtecan, HER2 ADC) Early development Breast cancer Trial Population Patients Design Endpoints Status CVRM Phase III HER2-positive, unresectable and/or 600 • Randomised, open-label, parallel assignment • Primary endpoint: PFS
Slide 20: Oncology: Enhertu breast cancer
Approved medicines Late-stage development Oncology Enhertu (trastuzumab deruxtecan, HER2 ADC) Early development Breast cancer Trial Population Patients Design Endpoints Status CVRM Phase III High-risk HER2-positive early non- 927 • Randomised, open-label, parallel assignment • Primary endpoint: pCR
Slide 21: Oncology: Enhertu gastric cancer
Approved medicines Late-stage development Oncology Enhertu (trastuzumab deruxtecan, HER2 ADC) Early development Gastric cancer Trial Population Patients Design Endpoints Status CVRM Phase III HER2-positive gastric cancer or GEJ 490 • Open-label, randomised, parallel group assignment • Primary endpoi
Slide 22: Oncology: Enhertu other cancers
Approved medicines Late-stage development Oncology Enhertu (trastuzumab deruxtecan, HER2 ADC) Early development Other cancers Trial Population Patients Design Endpoints Status CVRM Phase III HER2-mutated, unresectable, locally 450 • Randomised, parallel group assignment • Primary endpoint: PFS • FPC
Slide 23: Oncology: Enhertu other cancers
Approved medicines Late-stage development Oncology Enhertu (trastuzumab deruxtecan, HER2 ADC) Early development Other cancers Trial Population Patients Design Endpoints Status CVRM Phase II HER2-mutated tumours 102 • Non-randomised, single group assignment • Primary endpoint: ORR • FPCD: Q1 2021 DES
Slide 24: Oncology: Enhertu other cancers
Approved medicines Late-stage development Oncology Enhertu (trastuzumab deruxtecan, HER2 ADC) Early development Other cancers Trial Population Patients Design Endpoints Status CVRM Phase III Endometrial cancer excluding sarcoma 710 • Randomised, open label, parallel assignment • Primary endpoints: D
Slide 25: Oncology: Imfinzi gastrointestinal cancer
Approved medicines Late-stage development Oncology Imfinzi (PD-L1 mAb) Early development Gastrointestinal cancer Trial Population Patients Design Endpoints Status CVRM Phase III Locoregional HCC 710 • Arm 1: TACE in combination with Imfinzi • Primary endpoint: PFS (Arm 2 vs. Arm • FPCD: Q1 2019 EMER
Slide 26: Oncology: Imfinzi lung cancer
Approved medicines Late-stage development Oncology Imfinzi (PD-L1 mAb) Early development Lung cancer Trial Population Patients Design Endpoints Status CVRM Phase III Perioperative NSCLC patients, Stage II 802 • Arm 1: Imfinzi + platinum-based chemotherapy • Primary endpoints: pCR snd EFS • FPCD: Q1
Slide 27: Oncology: Imfinzi lung cancer
Approved medicines Late-stage development Oncology Imfinzi (PD-L1 mAb) Early development Lung cancer Trial Population Patients Design Endpoints Status CVRM Phase II NSCLC, patients who progressed on an 531 • Open-label, biomarker-directed, multi-centre trial • Primary endpoint: ORR • FPCD: Q1 2018 H
Slide 28: Oncology: Imfinzi other cancer
Approved medicines Late-stage development Oncology Imfinzi (PD-L1 mAb) Early development Other cancer Trial Population Patients Design Endpoints Status CVRM Phase III Non-muscle-invasive bladder cancer 1018 • Arm 1: BCG (induction + maintenance) • Primary endpoint: DFS • FPCD: Q2 2018 POTOMAC • Arm
Slide 29: Oncology: Lynparza Imfinzi combinations
Approved medicines Late-stage development Oncology Lynparza (PARP inhibitor) Early development Imfinzi combinations Trial Population Patients Design Endpoints Status CVRM Phase III 1L advanced ovarian cancer 1407 • Non-tBRCAm (tumour BRCA) patients • Primary endpoint: PFS • FPCD: Q1 2019 DUO-O • Arm
Slide 30: Oncology: Lynparza other cancer
Approved medicines Late-stage development Oncology Lynparza (PARP inhibitor) Early development Other cancer Trial Population Patients Design Endpoints Status CVRM Phase III BRCAwt advanced ovarian cancer, 1L 366 • Arm 1: Lynparza BID 24-month duration • Primary endpoints: PFS (BRCAwt HRD- • FPCD: Q3
Slide 31: Oncology: Orpathys
Approved medicines Late-stage development Oncology Orpathys (savolitinib, MET inhibitor) Early development NSCLC and other cancers Trial Population Patients Design Endpoints Status CVRM Phase II Locally advanced or metastatic gastric 75 • Single-arm, multi-cohort, multi-centre, open-label trial • Pr
Slide 32: Oncology: Tagrisso NSCLC
Approved medicines Late-stage development Oncology Tagrisso (highly-selective, irreversible EGFR inhibitor) Early development NSCLC Trial Population Patients Design Endpoints Status CVRM Phase III Maintenance therapy in patients with 216 • Arm 1: Tagrisso • Primary endpoint: PFS (BICR) • FPCD: Q4 20
Slide 33: Oncology: Tagrisso combinations
Approved medicines Late-stage development Oncology Tagrisso (highly-selective, irreversible EGFR inhibitor) Early development NSCLC, combinations Trial Population Patients Design Endpoints Status CVRM Phase III Neoadjuvant EGFRm NSCLC 351 • Arm 1: placebo + pemetrexed/carboplatin or • Primary endpoi
Slide 34: Oncology: Tagrisso combinations
Approved medicines Late-stage development Oncology Tagrisso (highly-selective, irreversible EGFR inhibitor) Early development NSCLC, combinations Trial Population Patients Design Endpoints Status CVRM Phase II Advanced EGFRm NSCLC patients who 250 • Modular design platform trial: • Primary endpoint:
Slide 35: Oncology: Truqap
Approved medicines Late-stage development Oncology Truqap (capivasertib, AKT inhibitor) Early development Breast cancer and prostate cancer Trial Population Patients Design Endpoints Status CVRM Phase III 2L+ AI-resistant locally advanced 834 • Double-blind, randomised, comparative trial • Primary e
Slide 36: Oncology: camizestrant
Approved medicines Late-stage development Oncology camizestrant (next-generation oral SERD) Early development Breast cancer Trial Population Patients Design Endpoints Status CVRM Phase III HR+ HER2-negative advanced breast 1370 • Randomised, double-blind, comparative trial • Primary endpoint: PFS •
Slide 37: Oncology: camizestrant
Approved medicines Late-stage development Oncology camizestrant (next-generation oral SERD) Early development Breast cancer Trial Population Patients Design Endpoints Status CVRM Phase I HR+ HER2-negative advanced breast 10 • Open-label trial • Primary endpoints: safety and • FPCD: Q4 2020 NCT045414
Slide 38: Oncology: ceralasertib
Approved medicines Late-stage development Oncology ceralasertib (AZD6738, ATR inhibitor) Early development Multiple cancers Trial Population Patients Design Endpoints Status CVRM Phase III Post-IO NSCLC 594 • Double-arm randomised • Primary endpoint: OS • FPCD: Q4 2022 LATIFY • Arm 1: ceralasertib +
Slide 39: Oncology: puxitatug samrotecan
Approved medicines Late-stage development Oncology puxitatug samrotecan (AZD8205, B7H4 ADC) Early development Solid tumours Trial Population Patients Design Endpoints Status CVRM Phase III B7H4 selected 2-3L endometrial cancer 700 • Randomised, single-open label • Primary endpoints: PFS, OS • Data a
Slide 40: Oncology: rilvegostomig lung cancer
Approved medicines Late-stage development Oncology rilvegostomig (PD-1/TIGIT bispecific mAb) Early development Lung cancer Trial Population Patients Design Endpoints Status CVRM Phase III Non-squamous NSCLC 1L patients 878 • Randomised, double-blind, multi-centre trial • Primary endpoints: PFS and O
Slide 41: Oncology: rilvegostomig solid tumours
Approved medicines Late-stage development Oncology rilvegostomig (PD-1/TIGIT bispecific mAb) Early development Solid tumours Trial Population Patients Design Endpoints Status CVRM Phase III adjuvant BTC with curative intent 750 • Randomised, double-blind, placebo-controlled, multicenter • Primary en
Slide 42: Oncology: rilvegostomig solid tumours
Approved medicines Late-stage development Oncology rilvegostomig (PD-1/TIGIT bispecific mAb) Early development Solid tumours Trial Population Patients Design Endpoints Status CVRM Phase IIb Gastric cancer 240 • Open-label gastric platform trial • Primary endpoints: safety and efficacy • FPCD: Q1 202
Slide 43: Oncology: saruparib
Approved medicines Late-stage development Oncology saruparib (AZD5305, PARP1 inhibitor) Early development Solid tumours Trial Population Patients Design Endpoints Status CVRM Phase III HRRm and non-HRRm mCSPC 1800 • Randomised, placebo-controlled trial • Primary endpoint: rPFS • FPCD: Q4 2023 EvoPAR
Slide 44: Oncology: saruparib
Approved medicines Late-stage development Oncology saruparib (AZD5305, PARP1 inhibitor) Early development Solid tumours Trial Population Patients Design Endpoints Status CVRM Phase I/IIa Advanced solid tumours 702 • Modular, open-label, multi-centre dose escalation and • Primary endpoints: safety, t
Slide 45: Oncology: sonesitatug vedotin
Approved medicines Late-stage development Oncology sonesitatug vedotin (AZD0901, CLDN18.2 MMAE ADC) Early development Solid tumours Trial Population Patients Design Endpoints Status CVRM Phase III 2L+ advanced or metastatic gastric or 572 • Multi-centre, open-label, sponsor-blinded, randomised trial
Slide 46: Oncology: surovatamig
Approved medicines Late-stage development Oncology surovatamig (AZD0486, CD19/CD3 next-generation bispecific T-cell engager) Early development Haematologic malignancies Trial Population Patients Design Endpoints Status CVRM Phase III Previously untreated follicular 1015 • Multi-centre, randomised, o
Slide 47: Oncology: volrustomig
Approved medicines Late-stage development Oncology volrustomig (PD-1/CTLA-4 bispecific mAb) Early development Solid tumours Trial Population Patients Design Endpoints Status CVRM Phase III High-risk locally advanced cervical 800 • Randomised, double-blind, placebo-controlled, multi-centre • Primary
Slide 48: Oncology: volrustomig
Approved medicines Late-stage development Oncology volrustomig (PD-1/CTLA-4 bispecific mAb) Early development Solid tumours Trial Population Patients Design Endpoints Status CVRM Phase IIb NSCLC 180 • Platform, randomised, open-label, multicenter, global trial • Primary endpoints: safety, & • FPCD:
Slide 49: Oncology: early-stage development
Oncology: early-stage development 49
Slide 50: Oncology: FPI-2265
Approved medicines Late-stage development Oncology FPI-2265 (PSMA radioconjugate) Early development Prostate cancer Trial Population Patients Design Endpoints Status CVRM Phase II PSMA-positive mCRPC previously 100 • Open-label, randomised, multi-centre trial • Primary endpoints: PSA50 and safety •
Slide 51: Oncology: IPH5201
Approved medicines Late-stage development Oncology IPH5201 (CD39 mAb) Early development Solid tumours Trial Population Patients Design Endpoints Status CVRM Phase I Advanced solid tumours 57 • Open-label, dose escalation trial to determine MTD of • Primary endpoints: AE, SAE and DLT • FPCD: Q1 2020
Slide 52: Oncology: NT-112
Approved medicines Late-stage development Oncology NT-112 (KRAS G12D specific TCR) Early development Solid tumours Trial Population Patients Design Endpoints Status CVRM Phase I Unresectable, advanced and/or 24 • Open-label, single-arm, multi-centre trial with dose • Primary endpoints: incidence of
Slide 53: Oncology: NT-125
Approved medicines Late-stage development Oncology NT-125 (autologous, multi-specific neoantigen-targeting TCR-T) Early development Solid tumours Trial Population Patients Design Endpoints Status CVRM Phase I Adults with recurrent or metastatic 42 • Open-label, single-arm, single-centre trial with d
Slide 54: Oncology: NT-175
Approved medicines Late-stage development Oncology NT-175 (TP53-armored TCR) Early development Multiple cancers Trial Population Patients Design Endpoints Status CVRM Phase I Unresectable, advanced, and/or 162 • Open-label, single-arm, multi-centre trial with dose • Primary endpoint: Incidence of D
Slide 55: Oncology: tilatamig samrotecan
Approved medicines Late-stage development Oncology tilatamig samrotecan (AZD9592, EGFR-cMET TOP1i ADC) Early development Lung cancer Trial Population Patients Design Endpoints Status CVRM Phase I Advanced solid tumours including 403 • Escalation phase, open-label, multi-centre trial • Primary endpoi
Slide 56: Oncology: AZD0022
Approved medicines Late-stage development Oncology AZD0022 (KRASG12D) Early development Solid tumours Trial Population Patients Design Endpoints Status CVRM Phase I/II Metastatic or locally advanced 430 • Open-label, multi-centre trial with FIH modular protocol • Primary endpoints (Part A and B): •
Slide 57: Oncology: AZD0120
Approved medicines Late-stage development Oncology AZD0120 (GC012F, autologous anti-CD19 and anti-BCMA CAR-T) Early development Blood cancers Trial Population Patients Design Endpoints Status CVRM Phase II Relapsed/refractory multiple myeloma 20 • Open-label, multi-centre, non-randomised trial • Pri
Slide 58: Oncology: AZD0305
Approved medicines Late-stage development Oncology AZD0305 (GPRC5D ADC) Early development Blood cancers Trial Population Patients Design Endpoints Status CVRM Phase I/II R/R multiple myeloma 226 • Open-label, dose escalation and dose expansion trial • Primary endpoints: occurrence of • FPCD: Q4 2023
Slide 59: Oncology: AZD0516
Approved medicines Late-stage development Oncology AZD0516 (STEAP2 ADC) Early development Prostate cancer Trial Population Patients Design Endpoints Status CVRM Phase I/II mCRPC 177 • Open-label multi-centre, modular dose escalation and dose • Primary: safety and tolerability • Data anticipated: >20
Slide 60: Oncology: AZD0754
Approved medicines Late-stage development Oncology AZD0754 (STEAP2 dnTGFβRII-armoured CAR-T) Early development Solid tumours Trial Population Patients Design Endpoints Status CVRM Phase I/II Metastatic castration resistance 60 • Open-label, single-arm, multi-centre trial with dose • Primary endpoint
Slide 61: Oncology: AZD1390
Approved medicines Late-stage development Oncology AZD1390 (ATM inhibitor) Early development Cancer Trial Population Patients Design Endpoints Status CVRM Phase I Recurrent glioblastoma eligible for re- 180 • Open-label trial • Primary endpoints: safety, tolerability • FPCD: Q2 2018 NCT03423628 irra
Slide 62: Oncology: AZD2068
Approved medicines Late-stage development Oncology AZD2068 (FPI-2068, EGFR cMET radioconjugate) Early development Solid tumours Trial Population Patients Design Endpoints Status CVRM Phase I Advanced solid tumours 110 • Multicentre, open-label dose escalation trial • Primary endpoints: safety and •
Slide 63: Oncology: AZD2284
Approved medicines Late-stage development Oncology AZD2284 (STEAP2 radioconjugate) Early development Prostate cancer Trial Population Patients Design Endpoints Status CVRM Phase I mCRPC 134 • Part A (Imaging): • Primary endpoints: safety and • FPCD: Q1 2025 NCT06879041 • Part A (Cold Antibody Explor
Slide 64: Oncology: AZD2962
Approved medicines Late-stage development Oncology AZD2962 (IRAK4 inhibitor) Early development Blood cancers Trial Population Patients Design Endpoints Status CVRM Phase I Haematologic neoplasms 72 • Modular, open-label, mutli-centre • Primary endpoints: DLT, AEs, duration • Data anticipated: >2026
Slide 65: Oncology: AZD3470
Approved medicines Late-stage development Oncology AZD3470 (PRMT5) Early development Solid tumours and blood cancer Trial Population Patients Design Endpoints Status CVRM Phase I MTAP-deficient advanced solid tumours 234 • Open-label, multi-centre • Primary endpoints: safety and • FPCD: Q1 2024 PRIM
Slide 66: Oncology: AZD4360
Approved medicines Late-stage development Oncology AZD4360 (CLDN18.2 ADC) Early development Solid tumours Trial Population Patients Design Endpoints Status CVRM Phase I/II Histologically confirmed advanced or 117 • Open-label, multi-centre trial with FIH modular protocol • Primary endpoints: safety
Slide 67: Oncology: AZD4512
Approved medicines Late-stage development Oncology AZD4512 (CD22 ADC) Early development Blood cancers Trial Population Patients Design Endpoints Status CVRM Phase I/II Relapsed/Refractory B-cell Non-Hodgkin 91 • Modular, open-label, non-randomised, multi-centre, dose • Primary endpoints: safety meas
Slide 68: Oncology: AZD5335
Approved medicines Late-stage development Oncology AZD5335 (anti-FRα TOP1i ADC) Early development Ovarian cancer, solid tumours Trial Population Patients Design Endpoints Status CVRM Phase I/II Advanced solid tumour malignancies 506 • Module 1: AZD5335 monotherapy • Primary endpoints: safety and • F
Slide 69: Oncology: AZD5492
Approved medicines Late-stage development Oncology AZD5492 (CD20 TITAN TCE) Early development Blood cancers Trial Population Patients Design Endpoints Status CVRM Phase I CLL, MCL, LBCL, FL 176 • Module 1: AZD5492 monotherapy • Primary endpoints: safety and • FPCD: Q3 2024 TITANIUM • AZD5492 monothe
Slide 70: Oncology: AZD5863
Approved medicines Late-stage development Oncology AZD5863 (CLDN18.2 CD3 bispecific antibody) Early development Solid tumours Trial Population Patients Design Endpoints Status CVRM Phase I Advanced or metastatic solid tumours 280 • Part A: dose escalation phase to determine the safety, • Primary end
Slide 71: Oncology: AZD6621
Approved medicines Late-stage development Oncology AZD6621 (STEAP2 T-cell engager) Early development Prostate cancer Trial Population Patients Design Endpoints Status CVRM Phase I/II mCRPC 52 • Open-label, multi-centre, modular dose escalation and dose • Primary: safety, tolerability • Data anticipa
Slide 72: Oncology: AZD6750
Approved medicines Late-stage development Oncology AZD6750 (CD8 guided IL-2) Early development Solid tumours Trial Population Patients Design Endpoints Status CVRM Phase I/II Select advanced or metastatic solid 60 • Open-label, dose escalation and expansion study • Primary endpoints: Safety and effi
Slide 73: Oncology: AZD7003
Approved medicines Late-stage development Oncology AZD7003 (GPC3 CAR-T) Early development Hepatocellular carcinoma (HCC) Trial Population Patients Design Endpoints Status CVRM Phase I/II GPC3-positive advanced/recurrent HCC 121 • Open-label, single-arm, multi-centre trial with dose • Primary endpoin
Slide 74: Oncology: AZD8421
Approved medicines Late-stage development Oncology AZD8421 (CDK2 inhibitor) Early development Breast cancer Trial Population Patients Design Endpoints Status CVRM Phase I/II ER+ HER2-negative advanced breast 204 • Module 1: AZD8421 • Primary endpoints: safety and • FPCD: Q4 2023 CYCAD-1 cancer • Mod
Slide 75: Oncology: AZD9574
Approved medicines Late-stage development Oncology AZD9574 (PARP1-sel BBB inhibitor) Early development Solid tumours Trial Population Patients Design Endpoints Status CVRM Phase I/IIa Advanced solid malignancies 695 • Modular, open-label, multi-centre dose escalation and • Primary endpoints: safety
Slide 76: AZD9793 (GPC3 TITAN T-cell engager)
Approved medicines Late-stage development AZD9793 (GPC3 TITAN T-cell engager) 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 • Data an
Slide 77: Oncology: AZD9829
Approved medicines Late-stage development Oncology AZD9829 (CD123 TOP1i ADC) Early development Blood cancers Trial Population Patients Design Endpoints Status CVRM Phase I/II CD123-positive haematological 56 • Open-label, multi-centre trial • Primary endpoints: safety and • FPCD: Q1 2024 ADC123 mali
Slide 78: BioPharmaceuticals: approved medicines and late-stage development
BioPharmaceuticals: approved medicines and late-stage development 78
Slide 79: Wainua (eplontersen) ATTR
Approved medicines Late-stage development Oncology Wainua (eplontersen, ligand-conjugated antisense) Early development ATTR Trial Population Patients Design Endpoints Status CVRM Phase III Hereditary or wild-type 1438 • Arm 1: Wainua s.c. • Primary endpoints: composite • FPCD: Q1 2020 CARDIO-TTRansf
Slide 80: balcinrenone/dapagliflozin heart failure, CKD
Approved medicines Late-stage development Oncology balcinrenone/dapagliflozin (MR antagonist/modulator + SGLT2 inhibitor) Early development Heart failure, CKD Trial Population Patients Design Endpoints Status CVRM Phase III Heart failure patients with 4800 • Randomised, double-blind, parallel-group,
Slide 81: baxdrostat hypertension
Approved medicines Late-stage development Oncology baxdrostat (selective aldosterone synthase inhibitor) Early development Hypertension Trial Population Patients Design Endpoints Status CVRM Phase III Patients with uncontrolled 720 • Arm 1: baxdrostat 1mg QD • Primary endpoint: effect of baxdrostat
Slide 82: baxdrostat hypertension
Approved medicines Late-stage development Oncology baxdrostat (selective aldosterone synthase inhibitor) Early development Hypertension Trial Population Patients Design Endpoints Status CVRM Phase II Patients with uncontrolled 175 • Arm 1: baxdrostat 2mg QD • Primary endpoints: safety and • FPCD: Q2
Slide 83: baxdrostat primary aldosteronism
Approved medicines Late-stage development Oncology baxdrostat (selective aldosterone synthase inhibitor) Early development Primary aldosteronism Trial Population Patients Design Endpoints Status CVRM Phase III Primary aldosteronism 180 • Multicentre, randomised, double-blind, placebo-controlled, • P
Slide 84: baxdrostat/dapagliflozin CKD
Approved medicines Late-stage development Oncology baxdrostat/dapagliflozin (selective ASI/SGLT2) Early development CKD/Prevention of heart failure Trial Population Patients Design Endpoints Status CVRM Phase III CKD and high blood pressure 2500 • Arm 1: baxdrostat/dapagliflozin QD • Primary endpoin
Slide 85: laroprovstat dyslipidaemia
Approved medicines Late-stage development Oncology laroprovstat (AZD0780, PCSK9 inhibitor) Early development Dyslipidaemia Trial Population Patients Design Endpoints Status CVRM Phase III Patients with dyslipidaemia 2800 • Randomised, double-Blind, placebo-controlled, parallel- • Primary endpoint: •
Slide 86: laroprovstat dyslipidaemia
Approved medicines Late-stage development Oncology laroprovstat (AZD0780, PCSK9 inhibitor) Early development Dyslipidaemia Trial Population Patients Design Endpoints Status CVRM Phase II Participants with ASCVD or 172 • Multi-centre, randomised, double-blind, placebo-controlled, • Primary endpoint:
Slide 87: zibotentan/dapagliflozin chronic kidney disease
Approved medicines Late-stage development Oncology zibotentan/dapagliflozin (ETA receptor antagonist/SGLT2 inhibitor) Early development Chronic kidney disease Trial Population Patients Design Endpoints Status CVRM Phase III CKD and high proteinuria 1835 • Randomised, parallel, multi-centre, double-b
Slide 88: Airsupra asthma
Approved medicines Late-stage development Oncology Airsupra (PT027, SABA/ICS, pMDI) Early development Asthma Trial Population Patients Design Endpoints Status CVRM Phase IIIb Adults and adolescents with 2517 • Randomised, double-blind, multi-centre, parallel-group, • Primary endpoint: time to first
Slide 89: Breztri, Trixeo asthma
Approved medicines Late-stage development Oncology Breztri, Trixeo (LAMA/LABA/ICS) Early development Asthma Trial Population Patients Design Endpoints Status CVRM Phase III Uncontrolled asthma 2266 • Randomised, double-blind, double-dummy, parallel group • Primary endpoint: change from • FPCD: Q1 20
Slide 90: Breztri, Trixeo COPD
Approved medicines Late-stage development Oncology Breztri, Trixeo (LAMA/LABA/ICS) Early development COPD Trial Population Patients Design Endpoints Status CVRM Phase III COPD 180 • Randomised, double-blind, three-treatment, three-period, • Primary endpoint: change from • FPCD: Q4 2023 ATHLOS crosso
Slide 91: Fasenra nasal polyposis and eosinophilic diseases
Approved medicines Late-stage development Oncology Fasenra (IL-5R mAb) Early development Nasal polyposis and other eosinophilic diseases Trial Population Patients Design Endpoints Status CVRM Phase III Patients with r/r EGPA on 140 • Arm 1: Fasenra 30mg Q4W s.c. • Primary endpoint: proportion of • F
Slide 92: Fasenra asthma and COPD
Approved medicines Late-stage development Oncology Fasenra (IL-5R mAb) Early development Severe, uncontrolled asthma and COPD Trial Population Patients Design Endpoints Status CVRM Phase III Patients with moderate to 689 • Double-blind, placebo-controlled trial • Primary endpoint: annualised rate of
Slide 93: Saphnelo lupus
Approved medicines Late-stage development Oncology Saphnelo (type I interferon receptor mAb) Early development Lupus (SLE/LN) Trial Population Patients Design Endpoints Status CVRM Phase III Moderate to severe SLE 360 • Arm 1: Saphnelo s.c. • Primary endpoint: BICLA at Week 52 • FPCD: Q3 2021 TULIP-
Slide 94: Saphnelo sclerosis and myopathies
Approved medicines Late-stage development Oncology Saphnelo (type I interferon receptor mAb) Early development Sclerosis and other myopathies Trial Population Patients Design Endpoints Status CVRM Phase III Systemic sclerosis 306 • Arm 1: Saphnelo s.c. • Primary endpoint: CRISS-25 at Week • FPCD: Q4
Slide 95: Tezspire CRSwNP, COPD and EoE
Approved medicines Late-stage development Oncology Tezspire (TSLP mAb) Early development CRSwNP, COPD and EoE Trial Population Patients Design Endpoints Status CVRM Phase III Severe chronic rhinosinusitis 416 • Arm 1: Tezspire s.c. • Co-primary endpoint: nasal polyp • FPCD: Q2 2021 WAYPOINT with nas
Slide 96: Tezspire severe asthma
Approved medicines Late-stage development Oncology Tezspire (TSLP mAb) Early development Severe, uncontrolled asthma Trial Population Patients Design Endpoints Status CVRM Phase III Severe asthma; age 12 to 80 1061 • Arm 1: Tezspire s.c. • Primary endpoint: annual asthma • FPCD: Q1 2018 NAVIGATOR ye
Slide 97: tozorakimab COPD
Approved medicines Late-stage development Oncology tozorakimab (IL-33 ligand mAb) Early development COPD Trial Population Patients Design Endpoints Status CVRM Phase III Adults with symptomatic 1132 • Randomised, double-blind, placebo-controlled, parallel- • Primary endpoint: annualised rate of • FP
Slide 98: tozorakimab viral LRTD, asthma
Approved medicines Late-stage development Oncology tozorakimab (IL-33 ligand mAb) Early development Severe viral LRTD, asthma Trial Population Patients Design Endpoints Status CVRM Phase III Adults hospitalised for viral 2870 • Randomised, double-blind, placebo-controlled, parallel • Primary endpoin
Slide 99: Next-generation propellant pMDI
Approved medicines Late-stage development Oncology Next-generation propellant Early development pMDI Trial Population Patients Design Endpoints Status CVRM Phase III Mucociliary clearance in 30 • Randomised, double-blind, multi-site, two-way crossover • Primary endpoint: change from • FPCD: Q2 2023
Slide 100: Next-generation propellant pMDI
Approved medicines Late-stage development Oncology Next-generation propellant Early development pMDI Trial Population Patients Design Endpoints Status CVRM Phase III Moderate to very severe 542 • Randomised, double-blind, 12-week (with an extension to • Primary endpoints: number of • FPCD: Q3 2022 N
Slide 101: Beyfortus infection
Approved medicines Late-stage development Oncology Beyfortus (nirsevimab, RSV mAb-YTE) Early development Infection Trial Population Patients Design Endpoints Status CVRM Phase III Healthy infants (born 29 800 • Randomised, double-blind, placebo-controlled • Primary endpoint: efficacy • FPCD: Q4 2021
Slide 102: Kavigale COVID-19
Approved medicines Late-stage development Oncology Kavigale (sipavibart, SARS-CoV-2 LAAB) Early development COVID-19 Trial Population Patients Design Endpoints Status CVRM Phase III Phase I: healthy adults; age 18 3200 • 2 parts (Phase I: sentinel safety cohort and Phase III: main • Primary endpoint
Slide 103: BioPharmaceuticals: early-stage development
BioPharmaceuticals: early-stage development 103
Slide 104: opemalirsen chronic kidney disease
Approved medicines Late-stage development Oncology opemalirsen (AZD2373, APOL1) Early development Chronic kidney disease Trial Population Patients Design Endpoints Status CVRM Phase IIb Participants diagnosed with 96 • Randomised, multi-centre, double-blind trial in US and UK • Primary endpoint: Dos
Slide 105: AZD0233 dilated cardiomyopathy
Approved medicines Late-stage development Oncology AZD0233 (oral CX3CR1) Early development Dilated cardiomyopathy Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers 96 • Randomised, SAD/MAD dose escalating trial • Primary endpoints: safety and • FPCD: Q2 2024 NCT063814
Slide 106: AZD1613 ADPKD
Approved medicines Late-stage development Oncology AZD1613 (inflammation and metabolism modulation) Early development ADPKD Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers 136 • Randomised, single-blind, placebo-controlled single and • Primary endpoint: safety and •
Slide 107: AZD1705 dyslipidaemia
Approved medicines Late-stage development Oncology AZD1705 (Angptl3 inhibitor) Early development Dyslipidaemia Trial Population Patients Design Endpoints Status CVRM Phase I Dyslipidaemia 112 • Part A: single dose of AZD1705 with an in-clinic period of 3 • Primary endpoints: AEs and SAEs • FPCD: Q1
Slide 108: AZD2389 MASH
Approved medicines Late-stage development Oncology AZD2389 (anti-fibrotic mechanism) Early development MASH Trial Population Patients Design Endpoints Status CVRM Phase II Participants with liver fibrosis 40 • Randomised, single-blind, placebo-controlled trial • Primary endpoints: safety and • FPCD:
Slide 109: AZD2693 MASH
Approved medicines Late-stage development Oncology AZD2693 (PNPLA3 ASO) Early development MASH Trial Population Patients Design Endpoints Status CVRM Phase IIb MASH with fibrosis 180 • Randomised, double-blind, placebo-controlled, multi-centre • Primary endpoints: efficacy, safety • FPCD: Q2 2023 FO
Slide 110: AZD3427 heart failure
Approved medicines Late-stage development Oncology AZD3427 (relaxin) Early development Heart failure Trial Population Patients Design Endpoints Status CVRM Phase II HF and pulmonary 260 • Randomised, double-blind, placebo-controlled, multi-centre • Primary endpoint: change in PVR from • FPCD: Q2 202
Slide 111: AZD4144 cardiorenal disease
Approved medicines Late-stage development Oncology AZD4144 (inflammation modulator) Early development Cardiorenal disease Trial Population Patients Design Endpoints Status CVRM Phase I Healthy participants 95 • Randomised, single-blind, placebo-controlled, SAD/MAD • Primary endpoints: safety and • F
Slide 112: AZD4248 CKD
Approved medicines Late-stage development Oncology AZD4248 (cellular health) Early development CKD Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers 164 • Randomised, single-blind, placebo-controlled • Primary endpoints: Safety and • FPCD: Q3 2025 NCT07024823 • Part A
Slide 113: AZD4954 dyslipidaemia
Approved medicines Late-stage development Oncology AZD4954 (Lp(a) inhibitor) Early development Dyslipidaemia Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers 120 • Randomised, placebo-controlled SAD/MAD trial • Primary endpoints: safety and • FPCD: Q2 2025 NCT0698042
Slide 114: AZD5004 type 2 diabetes, obesity
Approved medicines Late-stage development Oncology AZD5004 (oral GLP-1 RA) Early development Type 2 diabetes, obesity Trial Population Patients Design Endpoints Status CVRM Phase IIb Type 2 diabetes 406 • Arm 1: AZD5004 tablet • Primary endpoint: change in HbA1c • FPCD: Q4 2024 SOLSTICE • Arm 2: AZD
Slide 115: AZD5004 type 2 diabetes, obesity
Approved medicines Late-stage development Oncology AZD5004 (oral GLP-1 RA) Early development Type 2 diabetes, obesity Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers or 35 • SAD: 3 cohorts to receive AZD5004 or placebo tablet • Primary endpoints: safety and • FPCD:
Slide 116: AZD5462 heart failure
Approved medicines Late-stage development Oncology AZD5462 (oral relaxin) Early development Heart failure Trial Population Patients Design Endpoints Status CVRM Phase IIb Stable patients with chronic 360 • Two cohort, randomised, double-blind, placebo-controlled, • Primary endpoint: change in heart
Slide 117: AZD6234 obesity
Approved medicines Late-stage development Oncology AZD6234 (selective amylin agonist) Early development Obesity with related co-morbidities Trial Population Patients Design Endpoints Status CVRM Phase II Participants living with 231 • Randomised, double-blind, placebo-controlled trial • Primary endp
Slide 118: AZD6234 obesity
Approved medicines Late-stage development Oncology AZD6234 (selective amylin agonist) Early development Obesity with related co-morbidities Trial Population Patients Design Endpoints Status CVRM Phase I Healthy participants who are 64 • SAD trial • Primary endpoint: safety • FPCD: Q4 2022 NCT0551102
Slide 119: AZD9550 MASH
Approved medicines Late-stage development Oncology AZD9550 (GLP-1-glucagon agonist) Early development MASH Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers 64 • SAD trial • Primary endpoints: safety and • FPCD: Q2 2023 NCT05848440 tolerability • LPCD: Q4 2023 • Data
Slide 120: AZD9550+AZD6234 obesity
Approved medicines Late-stage development Oncology AZD9550+AZD6234 (GLP-1-glucagon agonist + selective amylin agonist) Early development Obesity Trial Population Patients Design Endpoints Status CVRM Phase IIb Adults who are living with 360 • Randomised, parallel-group, double-blind, placebo- • Prim
Slide 121: atuliflapon asthma
Approved medicines Late-stage development Oncology atuliflapon (FLAP inhibitor) Early development Asthma Trial Population Patients Design Endpoints Status CVRM Phase IIa Patients with moderate-to- 666 • Randomised, placebo-controlled, double-blind, multi-centre • Primary endpoint: time to first • FP
Slide 122: AZD0120 SLE
Approved medicines Late-stage development Oncology AZD0120 (GC012F, autologous anti-CD19 and anti-BCMA CAR-T) Early development SLE Trial Population Patients Design Endpoints Status CVRM Phase Ib/II Refractory systemic lupus 24 • Single-arm, open-label, multi-center trial • Primary endpoints (Phase
Slide 123: AZD1163 rheumatoid arthritis
Approved medicines Late-stage development Oncology AZD1163 (anti-PAD2/4 bispecific antibody) Early development Rheumatoid arthritis Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers 107 • Randomised, double-blind, placebo-controlled SAD/MAD • Primary endpoint: inciden
Slide 124: AZD4604 asthma
Approved medicines Late-stage development Oncology AZD4604 (inhaled JAK-1 inhibitor) Early development Asthma Trial Population Patients Design Endpoints Status CVRM Phase IIa Moderate-to-severe asthma 320 • Multi-centre, randomised, placebo-controlled, double- • Primary endpoint: time to first • FPC
Slide 125: AZD5492 SLE
Approved medicines Late-stage development Oncology AZD5492 (CD20 TITAN TCE) Early development SLE Trial Population Patients Design Endpoints Status CVRM Phase I Systemic lupus 70 • Open-label, multii-centre • Primary endpoints: safety and • Data anticipated: >2026 TITAN erythematosus (SLE) or • Part
Slide 126: AZD6793 COPD
Approved medicines Late-stage development Oncology AZD6793 (IRAK4) Early development COPD Trial Population Patients Design Endpoints Status CVRM Phase II moderate to very severe 1160 • Randomised, double-blind, placebo controlled 4 arm study • Primary endpoint: annualised rate of • FPCD: Q3 2025 PRE
Slide 127: AZD6912 rheumatoid arthritis
Approved medicines Late-stage development Oncology AZD6912 (siRNA) Early development Rheumatoid arthritis Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers 40 • Randomised, double-blind, placebo-controlled SAD trial • Primary endpoint: incidence of AEs • FPCD: Q4 2023
Slide 128: AZD7798 Crohn's disease
Approved medicines Late-stage development Oncology AZD7798 (humanised mAb) Early development Crohn's disease Trial Population Patients Design Endpoints Status CVRM Phase IIa Moderate to severe Crohn’s 192 • Randomised, double-blind, placebo-controlled trial • Primary endpoint: Crohn's Disease • FPCD
Slide 129: AZD8630 asthma
Approved medicines Late-stage development Oncology AZD8630 (inhaled TSLP) Early development Asthma Trial Population Patients Design Endpoints Status CVRM Phase II Adults with uncontrolled 516 • Randomised, placebo-controlled, double-blind, dose range- • Primary endpoint: time to first • FPCD: Q3 202
Slide 130: AZD8965 IPF
Approved medicines Late-stage development Oncology AZD8965 (arginase enzyme inhibitor) Early development IPF Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers 163 • Randomised, single-blind, SAD/MAD, placebo-controlled, • Primary endpoints (Part 1, 2, 3): safety • FPC
Slide 131: mRNA VLP vaccine COVID-19
Approved medicines Late-stage development Oncology mRNA VLP vaccine Early development COVID-19 Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers ≥18+ with 240 • Arm 1: dose 1 via i.m. injection AZD9838 in 18-64-year-olds • Primary endpoints: safety as measured • FPCD:
Slide 132: AZD0292 bronchiectasis
Approved medicines Late-stage development Oncology AZD0292 (Psl-PcrV N3Y-bispecific mAb) Early development Bronchiectasis Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers 32 • Randomised, single-blind, placebo-controlled trial • Primary endpoints: AEs and • FPCD: Q2
Slide 133: AZD5148 Clostridium difficile
Approved medicines Late-stage development Oncology AZD5148 (anti-TcdB mAb) Early development Clostridium difficile Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers 84 • Randomised, double-blind, placebo-controlled, dose • Primary endpoint: safety • FPCD: Q2 2024 NCT0
Slide 134: AZD7760 prevention of Staph aureus infection
Approved medicines Late-stage development Oncology AZD7760 (mAb combination targeting S aureus virulence factors) Early development Prevention of Staph aureus infection Trial Population Patients Design Endpoints Status CVRM Phase I/IIa Phase I: healthy volunteers 231 • Phase I: randomised, double-bl
Slide 135: Rare Disease: approved medicines and late-stage development
Rare Disease: approved medicines and late-stage development 135
Slide 136: Beyonttra ATTR-CM
Approved medicines Late-stage development Oncology Beyonttra (acoramidis, ALXN2060) Early development ATTR-CM Trial Population Patients Design Endpoints Status CVRM Phase III ATTR-CM 22 • Arm 1: 800mg Beyonttra administered twice daily • Primary endpoint: change from • FPCD: Q4 2020 ALXN2060-TAC-302
Slide 137: Koselugo neurofibromatosis type 1
Approved medicines Late-stage development Oncology Koselugo (selumetinib, MEK inhibitor) Early development Neurofibromatosis type 1, solid tumours Trial Population Patients Design Endpoints Status CVRM Phase III Adult age ≥18 years with NF1 145 • Multi-centre, international trial with a parallel, ra
Slide 138: Ultomiris haematology, nephrology, transplant
Approved medicines Late-stage development Oncology Ultomiris (anti-C5 mAb) Early development Haematology, nephrology, transplant Trial Population Patients Design Endpoints Status CVRM Phase III Thrombotic microangiopathy- 146 • Arm 1: Ultomiris Q8W • Primary endpoint: event free survival • FPCD: Q4
Slide 139: Ultomiris neurology
Approved medicines Late-stage development Oncology Ultomiris (anti-C5 mAb) Early development Neurology Trial Population Patients Design Endpoints Status CVRM Phase III Neuromyelitis optica 58 • Arm 1: Ultomiris Q8W • Primary endpoint: time to first • FPCD: Q4 2019 ALXN1210-NMO-307 spectrum disorder
Slide 140: anselamimab AL amyloidosis
Approved medicines Late-stage development Oncology anselamimab (CAEL-101, fibril-reactive mAb) Early development AL amyloidosis Trial Population Patients Design Endpoints Status CVRM Phase III AL amyloidosis (Mayo Stage 267 • Arm 1: anselamimab combined with SoC for PCD • Primary endpoint: a hierarc
Slide 141: cliramitug amyloidosis
Approved medicines Late-stage development Oncology cliramitug (ALXN2220, TTR depleter) Early development Amyloidosis Trial Population Patients Design Endpoints Status CVRM Phase III ATTR-CM (wild-type and 1180 • Arm 1: cliramitug via i.v. infusion Q4W for at least 24 months • Primary endpoint: compo
Slide 142: efzimfotase alfa hypophosphatasia
Approved medicines Late-stage development Oncology efzimfotase alfa (ALXN1850, next-generation asfotase alfa) Early development Hypophosphatasia Trial Population Patients Design Endpoints Status CVRM Phase III Hypophosphatasia 114 • Arm 1: placebo on Day 1 followed by Q2W via s.c. injection • Primar
Slide 143: eneboparatide hypoparathyroidism
Approved medicines Late-stage development Oncology eneboparatide (parathyroid hormone receptor 1 agonist) Early development Hypoparathyroidism Trial Population Patients Design Endpoints Status CVRM Phase III Chronic hypoparathyroidism 165 • Arm 1: 20mcg eneboparatide administered once daily via s.c.
Slide 144: gefurulimab neurology, nephrology
Approved medicines Late-stage development Oncology gefurulimab (ALXN1720, anti-C5 dual-binding nanobody) Early development Neurology, nephrology Trial Population Patients Design Endpoints Status CVRM Phase III Generalised myasthenia 254 • Arm 1: weight-based maintenance treatment with • Primary endp
Slide 145: Rare Disease: early-stage development
Rare Disease: early-stage development 145
Slide 146: ALXN1920 nephrology
Approved medicines Late-stage development Oncology ALXN1920 (kidney-targeted factor H fusion protein) Early development Nephrology Trial Population Patients Design Endpoints Status CVRM Phase II Primary membranous 30 • Randomised, double-blind, placebo-controlled • Primary Endpoint: Change From • Da
Slide 147: ALXN2030 transplant
Approved medicines Late-stage development Oncology ALXN2030 (siRNA targeting complement C3) Early development Transplant Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers 48 • Randomised, placebo-controlled SAD trial • Primary endpoint: safety • FPCD: Q4 2022 ALXN2030
Slide 148: ALXN2350 BAG3-associated DCM
Approved medicines Late-stage development Oncology ALXN2350 (AAV gene therapy) Early development BAG3-associated dilated cardiomyopathy (DCM) Trial Population Patients Design Endpoints Status CVRM Phase I/II BAG3 mutation associated 18 • Open-label, dose finding and dose expansion study • Primary en
Slide 149: ALXN2420 acromegaly
Approved medicines Late-stage development Oncology ALXN2420 (GH receptor antagonist) Early development Acromegaly Trial Population Patients Design Endpoints Status CVRM Phase IIb Acromegaly 60 • A Phase 2, randomised, double-blinded, placebo-controlled, • Primary endpoint: Percentage change • Data a
Slide 150: AZD0120 AL amyloidosis
Approved medicines Late-stage development Oncology AZD0120 (GC012F, autologous anti-CD19 and anti-BCMA CAR-T) Early development AL amyloidosis Trial Population Patients Design Endpoints Status CVRM Phase I/II Relapsed or Refractory AL 91 • Open-label, multicentre, non-randomised trial • Primary endp
Slide 151: MEDI1341 multiple system atrophy
Approved medicines Late-stage development Oncology MEDI1341 (alpha-synuclein mAb) Early development Multiple system atrophy Trial Population Patients Design Endpoints Status CVRM Phase II Patients with diagnosis of 158 • Randomised, double-blind, placebo-controlled trial • Primary endpoints: efficac
Slide 152: 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 153: 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 154: 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 155: 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 156: 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