Slide 1: Clinical Trials Appendix
Clinical Trials Appendix FY 2024 Results Update 6 February 2025
Slide 2: Pipeline at a glance
Pipeline 191 19 at a glance projects in our new molecular entities development pipeline (NME) in our late-stage pipeline Across five focus therapy areas: Oncology BioPharmaceuticals 130 31 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 H1 2025 H2 2025 2026 Tagrisso – EGFRm NSCLC (unresectable, Stg. III) (LAURA) (JP) Calquence – MCL (1L) (ECHO) (EU, JP) Imfinzi + Imjudo – NSCLC (1L) (POSEIDON) (CN) Calquence – CLL (1L, treat-to-progression) (ELE
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 Q3 2024 update
Project movements since Q3 2024 update New to Phase I New to Phase II New to pivotal trial New to registration NME NME Additional indication Life-cycle management AZD7760 AZD2389 rilvegostomig ARTEMIDE-Lung02# Calquence + venetoclax + obinutuzumab anti-staph aureus antibody combination anti-fibrotic
Slide 6: Project movements since Q3 2024 update
Project movements since Q3 2024 update Removed from Phase I Removed from Phase II Removed from Phase III Approved/removed from registration NME NME NME ALXN1910 AZD0171 + Imfinzi + CTx Datroway (datopotamab deruxtecan) next generation TNSALP ERT bone metabolism anti-LIF mAb + PD-L1 mAb + CTx 1st-lin
Slide 7: Q4 2024 Oncology new molecular entity pipeline
Q4 2024 Oncology new molecular entity1 pipeline Phase I Phase II Phase III Under review 21 New Molecular Entities 14 New Molecular Entities 17 New Molecular Entities 0 New Molecular Entities AZD0486 AZD0486 SOUNDTRACK-B AZD0022 AZD0486 SOUNDTRACK-F1 camizestrant + CDK4/6i SERENA-6 CD19/CD3 TCE B-cel
Slide 8: Q4 2024 Oncology lifecycle management pipeline
Q4 2024 Oncology lifecycle management1 pipeline Phase I Phase II Phase III Under review 0 Projects 9 Projects 38 Projects 3 Projects Datroway + rilvegostomig TROPION-Lung10# Enhertu (platform) DESTINY-Breast07# Calquence + R-CHOP ESCALADE Datroway + rilvegostomig TROPION-Lung12 # Calquence + venetoc
Slide 9: Q4 2024 BioPharmaceuticals new molecular entity pipeline
Q4 2024 BioPharmaceuticals new molecular entity1 pipeline Phase I Phase II Phase III Under review 15 New Molecular Entities 17 New Molecular Entities 6 New Molecular Entities 0 New Molecular Entities AZD0120 balcinrenone/dapagliflozin AZD0233 atuliflapon AZD0780 autologous anti-CD19 and anti-BCMA CA
Slide 10: Q4 2024 BioPharmaceuticals life cycle management pipeline
Q4 2024 BioPharmaceuticals life cycle management1 pipeline Phase I Phase II Phase III Under review 0 Projects 1 Project 13 Projects 0 Projects Tezspire COURSE# Breztri/Trixeo (PT010) KALOS LOGOS Breztri/Trixeo ATHLOS TSLP chronic obstructive pulmonary disease LABA/LAMA/ICS asthma LABA/LAMA/ICS COPD
Slide 11: Q4 2024 Rare Disease pipeline
Q4 2024 Rare Disease pipeline1 Phase I Phase II Phase III Under review 3 Projects 2 Projects 8 Projects 2 Projects MEDI1341# ALXN1920 ALXN2220 DepleTTR-CM# acoramidis# alpha synuclein mAb multiple system atrophy/Parkinson's kidney-targeted factor H fusion protein nephrology TTR depleter transthyreti
Slide 12: Designations in our pipeline
Designations in our pipeline 4 10 14 13 24 Accelerated approvals Breakthrough / PRIME1 / Sakigake2 Fast Track Priority Review Orphan Andexxa acute major bleed (US) Tezspire asthma NAVIGATOR (US) AZD0292 Psl-PcrV N3Y NCFBE (US) Tezspire asthma NAVIGATOR (US) Andexxa acute major bleed (JP) Kavigale SA
Slide 13: Oncology: approved medicines and late-stage pipeline
Oncology: approved medicines and late-stage pipeline 13
Slide 14: Oncology: Imfinzi gastrointestinal cancer
Approved medicines Oncology Imfinzi (PD-L1 mAb) Late-stage development 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. • FPCD: Q1 2019 EMERALD-
Slide 15: Oncology: Imfinzi lung cancer
Approved medicines Oncology Imfinzi (PD-L1 mAb) Late-stage development Early development Lung cancer Trial Population Patients Design Endpoints Status CVRM Phase III Perioperative NSCLC patients, Stage II 800 • Arm 1: Imfinzi + platinum-based chemotherapy • Primary endpoints: pCR snd EFS • FPCD: Q1
Slide 16: Oncology: Imfinzi lung cancer
Approved medicines Oncology Imfinzi (PD-L1 mAb) Late-stage development 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 17: Oncology: Imfinzi other cancer
Approved medicines Oncology Imfinzi (PD-L1 mAb) Late-stage development 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 18: Oncology: Lynparza Imfinzi combinations
Approved medicines Oncology Lynparza (PARP inhibitor) Late-stage development 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 19: Oncology: Lynparza other cancer
Approved medicines Oncology Lynparza (PARP inhibitor) Late-stage development 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 20: Oncology: Enhertu breast cancer
Approved medicines Oncology Enhertu (trastuzumab deruxtecan, HER2 ADC) Late-stage development 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 21: Oncology: Enhertu breast cancer
Approved medicines Oncology Enhertu (trastuzumab deruxtecan, HER2 ADC) Late-stage development 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 22: Oncology: Enhertu gastric cancer
Approved medicines Oncology Enhertu (trastuzumab deruxtecan, HER2 ADC) Late-stage development 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 23: Oncology: Enhertu other cancers
Approved medicines Oncology Enhertu (trastuzumab deruxtecan, HER2 ADC) Late-stage development 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 24: Oncology: Enhertu other cancers
Approved medicines Oncology Enhertu (trastuzumab deruxtecan, HER2 ADC) Late-stage development Early development Other cancers Trial Population Patients Design Endpoints Status CVRM Phase Ib HER2-overexpressing, unresectable 244 • Non-randomised, parallel group assignment • Primary endpoint: safety a
Slide 25: Oncology: Calquence blood cancers
Approved medicines Oncology Calquence (BTK inhibitor) Late-stage development 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 26: Oncology: Orpathys NSCLC and other cancers
Approved medicines Oncology Orpathys (savolitinib, MET inhibitor) Late-stage development Early development NSCLC and other cancers Trial Population Patients Design Endpoints Status CVRM Phase III Locally advanced or metastatic NSCLC 163 • Single-arm trial • Primary endpoint: ORR • FPCD: Q3 2021 NCT0
Slide 27: Oncology: Tagrisso NSCLC
Approved medicines Oncology Tagrisso (highly-selective, irreversible EGFR inhibitor) Late-stage development 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 28: Oncology: Tagrisso NSCLC combinations
Approved medicines Oncology Tagrisso (highly-selective, irreversible EGFR inhibitor) Late-stage development 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 29: Oncology: Tagrisso NSCLC combinations
Approved medicines Oncology Tagrisso (highly-selective, irreversible EGFR inhibitor) Late-stage development Early development NSCLC, combinations Trial Population Patients Design Endpoints Status CVRM Phase II EGFRm/MET+, locally advanced or 360 • Protocol v1-6: single-arm, open-label trial • Primar
Slide 30: Oncology: Truqap breast and prostate cancer
Approved medicines Oncology Truqap (capivasertib, AKT inhibitor) Late-stage development 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 31: Oncology: Datroway breast cancer
Approved medicines Oncology Datroway (datopotamab deruxtecan, TROP2 ADC) Late-stage development 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 32: Oncology: Datroway NSCLC
Approved medicines Oncology Datroway (datopotamab deruxtecan, TROP2 ADC) Late-stage development Early development NSCLC Trial Population Patients Design Endpoints Status CVRM Phase III 1L NSCLC 1350 • Arm 1: carboplatin + Datroway + Imfinzi • Co-primary endpoints: PFS and OS in • FPCD: Q1 2023 AVANZ
Slide 33: Oncology: Datroway NSCLC
Approved medicines Oncology Datroway (datopotamab deruxtecan, TROP2 ADC) Late-stage development 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 34: Oncology: Datroway other cancers
Approved medicines Oncology Datroway (datopotamab deruxtecan, TROP2 ADC) Late-stage development Early development Other cancers Trial Population Patients Design Endpoints Status CVRM Phase II Endometrial cancer, gastric cancer, 556 • Sub-study 1 (endometrial cancer); • Primary endpoints: ORR and saf
Slide 35: Oncology: AZD0486 haematologic malignancies
Approved medicines Oncology AZD0486 (CD19/CD3 next-generation bispecific T-cell engager) Late-stage development Early development Haematologic malignancies Trial Population Patients Design Endpoints Status CVRM Phase III Previously untreated follicular 1005 • Multi-centre, randomised, open-label tri
Slide 36: Oncology: AZD0901 solid tumours
Approved medicines Oncology AZD0901 (CLDN18.2 MMAE ADC) Late-stage development Early development Solid tumours Trial Population Patients Design Endpoints Status CVRM Phase III 2L+ advanced or metastatic gastric or 589 • Multi-centre, open-label, sponsor-blinded, randomised trial • Primary endpoints:
Slide 37: Oncology: camizestrant breast cancer
Approved medicines Oncology camizestrant (AZD9833, next-generation oral SERD) Late-stage development 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 endpoin
Slide 38: Oncology: camizestrant breast cancer
Approved medicines Oncology camizestrant (AZD9833, next-generation oral SERD) Late-stage development Early development Breast cancer Trial Population Patients Design Endpoints Status CVRM Phase I HR+ HER2-negative advanced breast 18 • Open-label trial • Primary endpoints: safety and • FPCD: Q4 2020
Slide 39: Oncology: ceralasertib multiple cancers
Approved medicines Oncology ceralasertib (AZD6738, ATR inhibitor) Late-stage development 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 40: Oncology: rilvegostomig lung cancer
Approved medicines Oncology rilvegostomig (AZD2936, PD-1/TIGIT bispecific mAb) Late-stage development 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:
Slide 41: Oncology: rilvegostomig solid tumours
Approved medicines Oncology rilvegostomig (AZD2936, PD-1/TIGIT bispecific mAb) Late-stage development 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 • FPC
Slide 42: Oncology: saruparib solid tumours
Approved medicines Oncology saruparib (AZD5305, PARP1 inhibitor) Late-stage development 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 43: Oncology: volrustomig solid tumours
Approved medicines Oncology volrustomig (MEDI5752, PD-1/CTLA-4 bispecific mAb) Late-stage development Early development Solid tumours Trial Population Patients Design Endpoints Status CVRM Phase Ib Advanced renal cell carcinoma 67 • Open-label, dose escalation and dose expansion trial • Primary endp
Slide 44: Oncology: volrustomig solid tumours
Approved medicines Oncology volrustomig (MEDI5752, PD-1/CTLA-4 bispecific mAb) Late-stage development 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
Slide 45: Oncology: early-stage development
Oncology: early-stage development 45
Slide 46: Oncology: AZD0022 solid tumours
Approved medicines Oncology AZD0022 (KRASG12D) Late-stage development 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 47: Oncology: AZD0120 blood cancers
Approved medicines Oncology AZD0120 (GC012F, autologous anti-CD19 and anti-BCMA CAR-T) Late-stage development Early development Blood cancers Trial Population Patients Design Endpoints Status CVRM Phase I/II Relapsed/refractory multiple myeloma 68 • Open-label, single-arm, multi-centre trial • Prima
Slide 48: Oncology: AZD0171 cancer
Approved medicines Oncology AZD0171 (anti-LIF mAb) Late-stage development Early development Cancer Trial Population Patients Design Endpoints Status CVRM Phase II 1L metastatic pancreatic ductal 126 • Open-label, non-randomised trial • Primary endpoints: safety and OS at • FPCD: Q1 2022 NCT04999969
Slide 49: Oncology: AZD0305 blood cancers
Approved medicines Oncology AZD0305 (GPRC5D ADC) Late-stage development Early development Blood cancers Trial Population Patients Design Endpoints Status CVRM Phase I/II r/r multiple myeloma 84 • Open-label, dose escalation and dose expansion trial • Primary endpoints: occurrence of • FPCD: Q4 2023
Slide 50: Oncology: AZD0754 solid tumours
Approved medicines Oncology AZD0754 (STEAP2 dnTGFβRII-armoured CAR-T) Late-stage development 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 51: Oncology: AZD1390 cancer
Approved medicines Oncology AZD1390 (ATM inhibitor) Late-stage development Early development Cancer Trial Population Patients Design Endpoints Status CVRM Phase I Recurrent glioblastoma eligible for re- 165 • Open-label trial • Primary endpoints: safety, tolerability • FPCD: Q2 2018 NCT03423628 irra
Slide 52: Oncology: AZD2068 solid tumours
Approved medicines Oncology AZD2068 (FPI-2068, EGFR cMET radioconjugate) Late-stage development 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 53: Oncology: AZD3470 solid tumours and blood cancer
Approved medicines Oncology AZD3470 (PRMT5) Late-stage development Early development Solid tumours and blood cancer Trial Population Patients Design Endpoints Status CVRM Phase I MTAP-deficient advanced solid tumours 210 • Arm 1: AZD3470 • Primary endpoints: safety and • FPCD: Q1 2024 PRIMROSE • Glo
Slide 54: Oncology: AZD5335 solid tumours
Approved medicines Oncology AZD5335 (anti-FRα TOP1i ADC) Late-stage development Early development Solid tumours, ovarian cancer, lung cancer Trial Population Patients Design Endpoints Status CVRM Phase I/II Advanced solid tumour malignancies 446 • Module 1: AZD5335 monotherapy • Primary endpoints: s
Slide 55: Oncology: AZD5492 blood cancers
Approved medicines Oncology AZD5492 (CD20 TITAN TCE) Late-stage development 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 56: Oncology: AZD5851 gastrointestinal cancer
Approved medicines Oncology AZD5851 (GPC3 dnTGFβRII-armoured CAR-T) Late-stage development Early development Gastrointestinal cancer Trial Population Patients Design Endpoints Status CVRM Phase I/II GPC3-positive advanced/recurrent HCC 93 • Open-label, single-arm, multi-centre trial with dose • Prim
Slide 57: Oncology: AZD5863 solid tumours
Approved medicines Oncology AZD5863 (CLDN18.2 CD3 bispecific antibody) Late-stage development Early development Solid tumours Trial Population Patients Design Endpoints Status CVRM Phase I Advanced or metastatic solid tumours 200 • Part A: dose escalation phase to determine the safety, • Primary end
Slide 58: Oncology: AZD6422 solid tumours
Approved medicines Oncology AZD6422 (CLDN18.2 CAR-T) Late-stage development Early development Solid tumours Trial Population Patients Design Endpoints Status CVRM Phase I Advanced or metastatic CLDN18.2- 96 • Open-label trial, dose escalation (Part 1) and dose • Primary endpoints: incidence of • FPC
Slide 59: Oncology: AZD7003 hepatocellular carcinoma
Approved medicines Oncology AZD7003 (GPC3 CAR-T) Late-stage development 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 60: Oncology: puxitatug samrotecan solid tumours
Approved medicines Oncology puxitatug samrotecan (AZD8205, B7H4 ADC) Late-stage development Early development Solid tumours Trial Population Patients Design Endpoints Status CVRM Phase I/II Breast cancer, BTC, ovarian cancer, 340 • Open-label dose escalation and expansion trial • Primary endpoints:
Slide 61: Oncology: AZD8421 breast cancer
Approved medicines Oncology AZD8421 (CDK2 inhibitor) Late-stage development 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 62: Oncology: AZD9574 solid tumours
Approved medicines Oncology AZD9574 (PARP1-sel BBB inhibitor) Late-stage development Early development Solid tumours Trial Population Patients Design Endpoints Status CVRM Phase I/IIa Advanced solid malignancies 490 • Modular, open-label, multi-centre dose escalation and • Primary endpoints: safety
Slide 63: Oncology: AZD9592 lung cancer
Approved medicines Oncology AZD9592 (EGFR-cMET TOP1i ADC) Late-stage development Early development Lung cancer Trial Population Patients Design Endpoints Status CVRM Phase I Advanced solid tumours including 162 • Escalation phase, open-label, multi-centre trial • Primary endpoints (escalation): safe
Slide 64: Oncology: AZD9829 blood cancers
Approved medicines Oncology AZD9829 (CD123 TOP1i ADC) Late-stage development Early development Blood cancers Trial Population Patients Design Endpoints Status CVRM Phase I/II CD123-positive haematological 104 • Open-label, multi-centre trial • Primary endpoints: safety and • FPCD: Q1 2024 ADC123 mal
Slide 65: Oncology: FPI-2265 prostate cancer
Approved medicines Oncology FPI-2265 (PSMA radioconjugate) Late-stage development Early development Prostate cancer Trial Population Patients Design Endpoints Status CVRM Phase II PSMA-positive mCRPC previously 60 • Open-label, randomised, multi-centre trial • Primary endpoints: PSA50 and safety • F
Slide 66: Oncology: IPH5201 solid tumours
Approved medicines Oncology IPH5201 (CD39 mAb) Late-stage development 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 67: Oncology: NT-112 solid tumours
Approved medicines Oncology NT-112 (KRAS G12D specific TCR) Late-stage development 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 68: Oncology: NT-125 solid tumours
Approved medicines Oncology NT-125 (autologous, multi-specific neoantigen-targeting TCR-T) Late-stage development 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 69: Oncology: NT-175 multiple cancers
Approved medicines Oncology NT-175 (TP53-armored TCR) Late-stage development Early development Multiple cancers Trial Population Patients Design Endpoints Status CVRM Phase I Unresectable, advanced, and/or 24 • Open-label, single-arm, multi-centre trial with dose • Primary endpoint: incidence of DL
Slide 70: Oncology: oleclumab solid tumours
Approved medicines Oncology oleclumab (CD73 mAb) Late-stage development Early development Solid tumours Trial Population Patients Design Endpoints Status CVRM Phase Ib/II Pancreatic 1L and 2L with prior 339 • Arm A1: gemcitabine and nab paclitaxel i.v. • Primary endpoints: safety and anti- • FPCD: Q
Slide 71: Oncology: sabestomig cancer
Approved medicines Oncology sabestomig (AZD7789, PD-1/TIM3 bispecific mAb) Late-stage development Early development Cancer Trial Population Patients Design Endpoints Status CVRM Phase I/IIa NSCLC, gastric cancer and other 192 • Open-label, non-randomised dose escalation and dose • Primary endpoints:
Slide 72: BioPharmaceuticals: approved medicines and late-stage development
BioPharmaceuticals: approved medicines and late-stage development 72
Slide 73: Wainua ATTR
Approved medicines Oncology Wainua (eplontersen, ligand-conjugated antisense) Late-stage development 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 74: balcinrenone/dapagliflozin heart failure, CKD
Approved medicines Oncology balcinrenone/dapagliflozin (MR modulator + SGLT2 inhibitor) Late-stage development Early development Heart failure, CKD Trial Population Patients Design Endpoints Status CVRM Phase III Heart failure patients with 4800 • Randomised, double-blind, parallel-group, double-dum
Slide 75: baxdrostat hypertension
Approved medicines Oncology baxdrostat (selective aldosterone synthase inhibitor) Late-stage development 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 76: baxdrostat hypertension
Approved medicines Oncology baxdrostat (selective aldosterone synthase inhibitor) Late-stage development 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 77: baxdrostat/dapagliflozin CKD
Approved medicines Oncology baxdrostat/dapagliflozin (selective ASI/SGLT2) Late-stage development Early development CKD Trial Population Patients Design Endpoints Status CVRM Phase III CKD and high blood pressure 2500 • Arm 1: baxdrostat/dapagliflozin QD • Primary endpoint: change from • FPCD: Q2 20
Slide 78: zibotentan/dapagliflozin chronic kidney disease
Approved medicines Oncology zibotentan/dapagliflozin (ETA receptor antagonist/SGLT2 inhibitor) Late-stage development 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 79: zibotentan/dapagliflozin liver cirrhosis
Approved medicines Oncology zibotentan/dapagliflozin (ETA receptor antagonist/SGLT2 inhibitor) Late-stage development Early development Liver cirrhosis Trial Population Patients Design Endpoints Status CVRM Phase II Part A: participants with 195 • Phase IIa/b multi-centre, randomised, double-blind,
Slide 80: Airsupra asthma
Approved medicines Oncology Airsupra (PT027, SABA/ICS, pMDI) Late-stage development 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 81: Breztri, Trixeo asthma
Approved medicines Oncology Breztri, Trixeo (LAMA/LABA/ICS) Late-stage development 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 82: Breztri, Trixeo COPD
Approved medicines Oncology Breztri, Trixeo (LAMA/LABA/ICS) Late-stage development 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 83: Fasenra nasal polyposis and eosinophilic diseases
Approved medicines Oncology Fasenra (IL-5R mAb) Late-stage development Early development Nasal polyposis and other eosinophilic diseases Trial Population Patients Design Endpoints Status CVRM Phase III Patients with severe bilateral 413 • Arm 1: Fasenra 30mg Q8W s.c. • Primary endpoint: effect of Fa
Slide 84: Fasenra severe asthma and COPD
Approved medicines Oncology Fasenra (IL-5R mAb) Late-stage development 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 85: Saphnelo lupus
Approved medicines Oncology Saphnelo (type I interferon receptor mAb) Late-stage development 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 86: Saphnelo sclerosis and other myopathies
Approved medicines Oncology Saphnelo (type I interferon receptor mAb) Late-stage development 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 • FPCD: Q4 2023
Slide 87: Tezspire CRSwNP, COPD and EoE
Approved medicines Oncology Tezspire (TSLP mAb) Late-stage development 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 88: Tezspire severe, uncontrolled asthma
Approved medicines Oncology Tezspire (TSLP mAb) Late-stage development 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 89: HFO1234ze next-generation propellant
Approved medicines Oncology HFO1234ze (next-generation propellant) Late-stage development 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 • FP
Slide 90: HFO1234ze next-generation propellant
Approved medicines Oncology HFO1234ze (next-generation propellant) Late-stage development Early development pMDI Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers 108 • Randomised, double-blind, single-dose, single-centre, • Primary endpoints: AUCinf, AUClast • FPCD:
Slide 91: tozorakimab COPD
Approved medicines Oncology tozorakimab (IL-33 ligand mAb) Late-stage development Early development COPD Trial Population Patients Design Endpoints Status CVRM Phase III Adults with symptomatic 1099 • Randomised, double-blind, placebo-controlled, parallel- • Primary endpoint: annualised rate of • FP
Slide 92: tozorakimab severe viral LRTD, asthma
Approved medicines Oncology tozorakimab (IL-33 ligand mAb) Late-stage development 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 93: Beyfortus infection
Approved medicines Oncology Beyfortus (nirsevimab, RSV mAb-YTE) Late-stage development 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 94: Evusheld COVID-19
Approved medicines Oncology Evusheld (AZD7442, tixagevimab + cilgavimab) Late-stage development Early development COVID-19 Trial Population Patients Design Endpoints Status CVRM Phase II Adults and pediatric 251 • Randomised, open-label, dose-ranging to assess safety, • Primary endpoints: safety and
Slide 95: Kavigale COVID-19
Approved medicines Oncology Kavigale (sipavibart, SARS-CoV-2 LAAB) Late-stage development 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 96: BioPharmaceuticals: early-stage development
BioPharmaceuticals: early-stage development 96
Slide 97: AZD0233 dilated cardiomyopathy
Approved medicines Oncology AZD0233 (oral CX3CR1) Late-stage development 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 98: AZD0780 dyslipidaemia
Approved medicines Oncology AZD0780 (PCSK9 inhibitor) Late-stage development Early development Dyslipidaemia Trial Population Patients Design Endpoints Status CVRM Phase II Dyslipidaemia 428 • Randomised trial with equal distribution across five parallel • Primary endpoint: percent change in • FPCD:
Slide 99: AZD0780 dyslipidaemia
Approved medicines Oncology AZD0780 (PCSK9 inhibitor) Late-stage development Early development Dyslipidaemia Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers 183 • Randomised, placebo-controlled SAD/MAD trial • Primary endpoints: safety and • FPCD: Q2 2022 NCT0538426
Slide 100: AZD1705 dyslipidaemia
Approved medicines Oncology AZD1705 (Angptl3 inhibitor) Late-stage development 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 101: AZD2373 chronic kidney disease
Approved medicines Oncology AZD2373 (APOL1) Late-stage development Early development Chronic kidney disease Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers 30 • SAD dose escalation in 6 cohorts with • Primary endpoints: safety and • FPCD: Q1 2020 NCT04269031 6 volun
Slide 102: AZD2389 MASH
Approved medicines Oncology AZD2389 (anti-fibrotic mechanism) Late-stage development Early development MASH Trial Population Patients Design Endpoints Status CVRM Phase II Participants with liver fibrosis 36 • Randomised, single-blind, placebo-controlled trial • Primary endpoints: safety and • FPCD:
Slide 103: AZD2693 MASH
Approved medicines Oncology AZD2693 (PNPLA3 ASO) Late-stage development Early development MASH Trial Population Patients Design Endpoints Status CVRM Phase IIb NASH with fibrosis 180 • Randomised, double-blind, placebo-controlled, multi-centre • Primary endpoints: efficacy, safety • FPCD: Q2 2023 FO
Slide 104: AZD3427 heart failure
Approved medicines Oncology AZD3427 (relaxin) Late-stage development Early development Heart failure Trial Population Patients Design Endpoints Status CVRM Phase II HF and pulmonary 220 • Randomised, double-blind, placebo-controlled, multi-centre • Primary endpoint: change in PVR from • FPCD: Q2 202
Slide 105: AZD4144 cardiorenal disease
Approved medicines Oncology AZD4144 (inflammation modulator) Late-stage development Early development Cardiorenal disease Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers 95 • Randomised, single-blind, placebo-controlled, SAD/MAD • Primary endpoints: safety and • FPC
Slide 106: AZD5004 type 2 diabetes, obesity
Approved medicines Oncology AZD5004 (oral GLP-1 RA) Late-stage development Early development Type 2 diabetes, obesity Trial Population Patients Design Endpoints Status CVRM Phase IIb Type 2 diabetes 384 • Arm 1: AZD5004 tablet • Primary endpoint: change in HbA1c • FPCD: Q4 2024 SOLSTICE • Arm 2: AZD
Slide 107: AZD5004 type 2 diabetes, obesity
Approved medicines Oncology AZD5004 (oral GLP-1 RA) Late-stage development Early development Type 2 diabetes, obesity Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers 31 • Part A – Arm 1: AZD5004 oral tablet • Primary endpoints (Part A): safety and • FPCD: Q3 2024 NC
Slide 108: AZD5462 heart failure
Approved medicines Oncology AZD5462 (oral relaxin) Late-stage development 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 109: AZD6234 obesity with related co-morbidities
Approved medicines Oncology AZD6234 (long-acting amylin) Late-stage development 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 endpoints:
Slide 110: AZD9550 MASH
Approved medicines Oncology AZD9550 (GLP-1-glucagon agonist) Late-stage development 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 111: mitiperstat cardiovascular disease, MASH
Approved medicines Oncology mitiperstat (MPO inhibitor) Late-stage development Early development Cardiovascular disease, MASH Trial Population Patients Design Endpoints Status CVRM Phase IIb HFpEF 711 • Randomised, double-blind trial • Primary endpoints: safety and efficacy • FPCD: Q3 2021 ENDEAVOR
Slide 112: atuliflapon asthma
Approved medicines Oncology atuliflapon (FLAP inhibitor) Late-stage development 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 113: AZD0120 lupus (SLE)
Approved medicines Oncology AZD0120 (GC012F, autologous anti-CD19 and anti-BCMA CAR-T) Late-stage development Early development Lupus (SLE) Trial Population Patients Design Endpoints Status CVRM Phase I/II Refractory systemic lupus 21 • Single-arm, open label, multi-centre trial • Primary endpoint (
Slide 114: AZD1163 rheumatoid arthritis
Approved medicines Oncology AZD1163 (bispecific antibody) Late-stage development Early development Rheumatoid arthritis Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers 108 • Randomised, double-blind, placebo-controlled SAD/MAD • Primary endpoint: number of • FPCD: Q
Slide 115: AZD4604 asthma
Approved medicines Oncology AZD4604 (inhaled JAK-1 inhibitor) Late-stage development 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 116: AZD6793 inflammatory diseases
Approved medicines Oncology AZD6793 (IRAK4) Late-stage development Early development Inflammatory diseases Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers 133 • Single-blind, randomised, placebo-controlled trial • Primary endpoints: safety and • FPCD: Q4 2022 NCT056
Slide 117: AZD6912 rheumatoid arthritis
Approved medicines Oncology AZD6912 (siRNA) Late-stage development Early development Rheumatoid arthritis Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers 64 • Randomised, double-blind, placebo-controlled SAD trial • Primary endpoint: incidence of AEs • FPCD: Q4 2023
Slide 118: AZD7798 Crohn's disease
Approved medicines Oncology AZD7798 (humanised mAb) Late-stage development 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 119: AZD8630 asthma
Approved medicines Oncology AZD8630 (inhaled TSLP) Late-stage development 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 120: AZD8965 IPF
Approved medicines Oncology AZD8965 (arginase enzyme inhibitor) Late-stage development Early development IPF Trial Population Patients Design Endpoints Status CVRM Phase I Healthy participants 163 • Randomised, single-blind, SAD/MAD, placebo-controlled, • Primary endpoints (Part 1, 2, 3): safety • F
Slide 121: mitiperstat COPD
Approved medicines Oncology mitiperstat (MPO inhibitor) Late-stage development Early development COPD Trial Population Patients Design Endpoints Status CVRM Phase II Moderate to severe COPD; 381 • Randomised, double-blind trial • Primary endpoint: time to first COPD • FPCD: Q1 2023 CRESCENDO age 40
Slide 122: AZD4041 opioid use disorder
Approved medicines Oncology AZD4041 (orexin 1 receptor antagonist) Late-stage development Early development Opioid use disorder Trial Population Patients Design Endpoints Status CVRM Phase II Healthy volunteers and 100 • Part 1: open label, fixed sequence trial of AZD4041 and • Primary endpoints (Pa
Slide 123: MEDI0618 migraine prevention
Approved medicines Oncology MEDI0618 (PAR2 antagonist mAb) Late-stage development Early development Migraine prevention Trial Population Patients Design Endpoints Status CVRM Phase II Cohort of participants failed 408 • Arm 1: CGRP-N MEDI0618 Dose A • Primary endpoint: change in number • FPCD: Q4 20
Slide 124: MEDI0618 osteoarthritis pain, migraine prevention
Approved medicines Oncology MEDI0618 (PAR2 antagonist mAb) Late-stage development Early development Osteoarthritis pain, migraine prevention Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers 112 • Randomised, double-blind, placebo-controlled MAD trial • Primary endpoi
Slide 125: MEDI7352 osteoarthritis pain
Approved medicines Oncology MEDI7352 (NGF TNF bispecific mAb) Late-stage development Early development Osteoarthritis pain Trial Population Patients Design Endpoints Status CVRM Phase IIb Painful osteoarthritis of the 350 • MAD trial • Primary endpoint: dose response • FPCD: Q1 2021 NCT04675034 knee
Slide 126: AZD0292 bronchiectasis
Approved medicines Oncology AZD0292 (Psl-PcrV N3Y-bispecific mAb) Late-stage development 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 127: AZD5148 Clostridium difficile
Approved medicines Oncology AZD5148 (anti-TcdB mAb) Late-stage development 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 128: AZD7760 prevention of Staph aureus infection
Approved medicines Oncology AZD7760 (mAb combination targeting S aureus virulence factors) Late-stage development 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 129: mRNA VLP vaccine COVID-19
Approved medicines Oncology mRNA VLP vaccine Late-stage development 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 130: Rare Disease: approved medicines and late-stage development
Rare Disease: approved medicines and late-stage development 130
Slide 131: Koselugo neurofibromatosis type 1, solid tumours
Approved medicines Oncology Koselugo (selumetinib, MEK inhibitor) Late-stage development 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 132: Ultomiris haematology, nephrology
Approved medicines Oncology Ultomiris (anti-C5 mAb) Late-stage development Early development Haematology, nephrology Trial Population Patients Design Endpoints Status CVRM Phase III Thrombotic microangiopathy- 106 • Arm 1: Ultomiris Q8W • Primary endpoint: TMA response • FPCD: Q4 2020 ALXN1210-TM-31
Slide 133: Ultomiris neurology
Approved medicines Oncology Ultomiris (anti-C5 mAb) Late-stage development 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 134: acoramidis ATTR-CM
Approved medicines Oncology acoramidis (ALXN2060, TTR stabiliser) Late-stage development Early development ATTR-CM Trial Population Patients Design Endpoints Status CVRM Phase III ATTR-CM 22 • Arm 1: 800mg acoramidis administered twice daily • Primary endpoint: change from • FPCD: Q4 2020 ALXN2060-T
Slide 135: ALXN2220 amyloidosis
Approved medicines Oncology ALXN2220 (NI006, TTR depleter) Late-stage development Early development Amyloidosis Trial Population Patients Design Endpoints Status CVRM Phase III ATTR-CM 1000 • Arm 1: ALXN2220 via i.v. infusion Q4W for at least 24 months • Primary endpoints: all-cause mortality • FPCD
Slide 136: anselamimab AL amyloidosis
Approved medicines Oncology anselamimab (CAEL-101, fibril-reactive mAb) Late-stage development Early development AL amyloidosis Trial Population Patients Design Endpoints Status CVRM Phase III AL amyloidosis 267 • Arm 1: anselamimab combined with SoC for PCD • Primary endpoint: A hierarchical • FPCD
Slide 137: efzimfotase alfa hypophosphatasia
Approved medicines Oncology efzimfotase alfa (ALXN1850, next-generation asfotase alfa) Late-stage development Early development Hypophosphatasia Trial Population Patients Design Endpoints Status CVRM Phase I Hypophosphatasia 15 • Arm 1: ALXN1850, 3 cohorts at low, medium and high • Primary endpoint:
Slide 138: eneboparatide hypoparathyroidism
Approved medicines Oncology eneboparatide (parathyroid hormone receptor 1 agonist) Late-stage development 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 139: gefurulimab neurology, nephrology
Approved medicines Oncology gefurulimab (ALXN1720, anti-C5 humanised bispecific heavy-chain antibody) Late-stage development Early development Neurology, nephrology Trial Population Patients Design Endpoints Status CVRM Phase III Generalised myasthenia 254 • Arm 1: weight-based maintenance treatment
Slide 140: Rare Disease: early-stage development
Rare Disease: early-stage development 140
Slide 141: ALXN1910 bone metabolism
Approved medicines Oncology ALXN1910 (next-generation TNSALP ERT) Late-stage development Early development Bone metabolism Trial Population Patients Design Endpoints Status CVRM Phase I Healthy adults 48 • Randomised, placebo-controlled SAD trial • Primary endpoint: safety • FPCD: Q2 2022 ALXN1910-H
Slide 142: ALXN1920 nephrology
Approved medicines Oncology ALXN1920 (kidney-targeted factor H fusion protein) Late-stage development Early development Nephrology Trial Population Patients Design Endpoints Status CVRM Phase I Healthy adults 48 • Randomised, double-blind, placebo-controlled, SAD trial • Primary endpoints: safety an
Slide 143: ALXN2030 nephrology
Approved medicines Oncology ALXN2030 (siRNA targeting complement C3) Late-stage development Early development Nephrology 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 144: ALXN2080 complement-mediated disease
Approved medicines Oncology ALXN2080 (factor D inhibitor) Late-stage development Early development Complement-mediated disease Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers 90 • SAD/MAD trial • Primary endpoints: safety and • FPCD: Q3 2022 ALXN2080-HV-101 tolerabi
Slide 145: MEDI1341 multiple system atrophy
Approved medicines Oncology MEDI1341 (alpha-synuclein mAb) Late-stage development Early development Multiple system atrophy Trial Population Patients Design Endpoints Status CVRM Phase II Patients with diagnosis of 138 • Randomised, double-blind, placebo-controlled trial • Primary endpoints: efficac
Slide 146: vemircopan haematology, nephrology, neurology
Approved medicines Oncology vemircopan (ALXN2050, factor D inhibitor) Late-stage development Early development Haematology, nephrology, neurology Trial Population Patients Design Endpoints Status CVRM Phase II Lupus nephritis or 126 • Arm 1 – LN cohort: vemircopan 180mg • Primary endpoint (both coho
Slide 147: 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 148: 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 149: 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 150: 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 151: 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