Slide 1: Clinical Trials Appendix: H1 2024 Results Update
Clinical Trials Appendix H1 2024 Results Update 25 July 2024
Slide 2: Pipeline at a glance
Pipeline 189 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 131 16 CVRM | R&I | V&I new molecular entities regulatory approvals (NME) or major lifecycle in major markets manageme
Slide 3: Key upcoming pipeline catalysts: 2024 and 2025
Key upcoming pipeline catalysts: 2024 and 2025 Oncology BioPharmaceuticals Rare Disease H2 2024 H1 2025 H2 2025 Tagrisso –EGFRm NSCLC (unresectable Stg. III) (LAURA) Lynparza – gBRCA breast cancer (adjuvant) (OlympiA) (CN) Truqap – HR+/HER2-neg breast cancer (2L) (CAPItello-291) (CN) Lynparza + Imfi
Slide 4: Clinical Trials Appendix: selected highlights
Clinical Trials Appendix: selected highlights BioPharmaceuticals Oncology Rare Disease Approved medicines: key LCM camizestrant (oral SERD) balcinrenone/dapagliflozin (MRM/SGLT2) registrational studies ongoing Dato-DXd (TROP2 ADC) ALXN2220 (TTR depleter) Next-wave pipeline: baxdrostat (aldosterone s
Slide 5: Project movements since Q1 2024 update
Project movements since Q1 2024 update New to Phase I New to Phase II New to pivotal trial New to registration NME NME NME NME AZD0233 AZD4041# eneboparatide CALYPSO# parathyroid hormone sipavibart SUPERNOVA CX3CR1 dilated cardiomyopathy orexin 1 receptor antagonist opioid use disorder receptor 1 hy
Slide 6: Q2 2024 Oncology new molecular entity pipeline
Q2 2024 Oncology new molecular entity1 pipeline Phase I Phase II Phase III 17 New Molecular Entities 13 New Molecular Entities 22 New Molecular Entities AZD0120 AZD0305 AZD0171 + Imfinzi + CTx AZD0901 CLARITY-Gastric01 camizestrant + CDK4/6i SERENA-6 autologous anti-CD19 and anti-BCMA CART GPRC5D AD
Slide 7: Q2 2024 Oncology lifecycle management pipeline
Q2 2024 Oncology lifecycle management1 pipeline Phase I Phase II Phase III 1 Project 9 Projects 32 Projects Enhertu (platform) DESTINY-Breast08# Enhertu (platform) DESTINY-Breast07# Calquence + R-CHOP ESCALADE Calquence + venetoclax + obinutuzumab AMPLIFY# Calquence ECHO# HER2 ADC HER2-low breast ca
Slide 8: Q2 2024 BioPharmaceuticals new molecular entity pipeline
Q2 2024 BioPharmaceuticals new molecular entity1 pipeline Phase I Phase II Phase III Under review 17 New Molecular Entities 14 New Molecular Entities 6 New Molecular Entities 1 New Molecular Entity AZD0233 AZD0292 atuliflapon balcinrenone/dapagliflozin sipavibart SUPERNOVA pseudomonas Psl-PcrV bispe
Slide 9: Q2 2024 BioPharmaceuticals life cycle management pipeline
Q2 2024 BioPharmaceuticals life cycle management1 pipeline Phase I Phase II Phase III Under review 0 Projects 1 Project 14 Projects 2 Projects Breztri/Trixeo THARROS# Tezspire COURSE# Breztri/Trixeo (PT010) KALOS LOGOS Fasenra MANDARA LABA/LAMA/ICS cardiopulmonary outcomes trial in TSLP chronic obst
Slide 10: Q2 2024 Rare Disease pipeline
Q2 2024 Rare Disease pipeline1 Phase I Phase II Phase III Under review 4 Projects 4 Projects 8 Projects 1 Project ALXN1910 danicopan ALXN2220 DepleTTR-CM# acoramidis# next gen TNSALP ERT bone metabolism factor D geographic atrophy TTR depleter transthyretin amyloid cardiomyopathy oral TTR stabilizer
Slide 11: Designations in our pipeline
Designations in our pipeline 5 14 9 14 20 Accelerated approvals Breakthrough / PRIME1 / Sakigake2 Fast Track Priority Review Orphan Andexxa acute major bleed (US) Beyfortus RSV mAb-YTE MELODY-MEDLEY (US) AZD3427 relaxin mimetic heart failure (US) Beyfortus RSV mAb-YTE MELODY-MEDLEY (CN) Andexxa acut
Slide 12: Oncology: approved medicines and late-stage pipeline
Oncology: approved medicines and late-stage pipeline 12
Slide 13: 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. • FPCD: Q1 2019 EMERALD-
Slide 14: 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 Neoadjuvant NSCLC patients, Stage II 800 • Arm 1: Imfinzi + platinum-based chemotherapy • Primary endpoints: pCR snd EFS • FPCD: Q1 20
Slide 15: 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 529 • Open-label, biomarker-directed, multi-centre trial • Primary endpoint: ORR • FPCD: Q1 2018 H
Slide 16: 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 17: 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 18: 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 gBRCAm adjuvant breast cancer 1836 • Arm 1: Lynparza BID 12-month duration • Primary endpoint: iDFS • FPCD: Q2 2014 OlympiA • A
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 • Primacy 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- 900 • 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 gastro- 490 • Open-label, randomised, parallel group assignment • Primary en
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 Ib HER2-over-expressing, unresectable 244 • Non-randomised, parallel group assignment • Primary endpoint: safety
Slide 24: Oncology: Calquence, blood cancers
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 25: Oncology: Orpathys, NSCLC and other cancers
Approved medicines Late-stage development Oncology Orpathys (savolitinib, MET inhibitor) 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 26: 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 27: Oncology: Tagrisso, NSCLC 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 EGFR-mutated, MET-overexpressed 324 • Arm 1: Tagrisso + Orpathys • Primary enpoint: PFS •
Slide 28: Oncology: Truqap, breast and prostate cancer
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 Locally advanced or metastatic TNBC 924 • Double-blind, randomised, comparative trial • Primary
Slide 29: Oncology: datopotamab deruxtecan, breast cancer
Approved medicines Late-stage development Oncology datopotamab deruxtecan (TROP2 ADC) Early development Breast cancer Trial Population Patients Design Endpoints Status CVRM Phase III Inoperable or metastatic HR+ HER2- 733 • Open-label, randomised trial • Primary endpoints: PFS (BICR) and OS • FPCD:
Slide 30: Oncology: datopotamab deruxtecan, NSCLC
Approved medicines Late-stage development Oncology datopotamab deruxtecan (TROP2 ADC) Early development NSCLC Trial Population Patients Design Endpoints Status CVRM Phase III Previously treated advanced or 590 • Randomised, open-label, parallel assignment • Primary endpoints: PFS and OS • FPCD: Q1 2
Slide 31: Oncology: datopotamab deruxtecan, NSCLC
Approved medicines Late-stage development Oncology datopotamab deruxtecan (TROP2 ADC) Early development NSCLC Trial Population Patients Design Endpoints Status CVRM Phase II Advanced or metastatic NSCLC with 137 • Single-arm, open-label • Primary endpoint: ORR • FPCD: Q1 2021 TROPION-Lung05 actionab
Slide 32: Oncology: datopotamab deruxtecan, other cancers
Approved medicines Late-stage development Oncology datopotamab deruxtecan (TROP2 ADC) 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 safety • FPCD
Slide 33: Oncology: datopotamab deruxtecan, other cancers
Approved medicines Late-stage development Oncology datopotamab deruxtecan (TROP2 ADC) Early development Other cancers Trial Population Patients Design Endpoints Status CVRM Phase I/II NSCLC and TNBC and other solid 119 • Single-arm, multi-cohort trial with no blinding • Primary endpoint: ORR • FPCD:
Slide 34: Oncology: AZD0901, solid tumours
Approved medicines Late-stage development Oncology AZD0901 (CLDN18.2 MMAE ADC) 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 35: Oncology: camizestrant, breast cancer
Approved medicines Late-stage development Oncology camizestrant (AZD9833, 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 endpoin
Slide 36: Oncology: camizestrant, breast cancer
Approved medicines Late-stage development Oncology camizestrant (AZD9833, next-generation oral SERD) Early development Breast cancer Trial Population Patients Design Endpoints Status CVRM Phase II HR+ advanced breast cancer 240 • Randomised, open-label, parallel-group, multi-centre trial • Primary e
Slide 37: Oncology: ceralasertib, multiple cancers
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 38: 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 expressing tumours to pursue 50 • Non-randomised single group assignment • Primary endpoint: ORR • FPCD:
Slide 39: Oncology: rilvegostomig, solid tumours
Approved medicines Late-stage development Oncology rilvegostomig (AZD2936, PD-1/TIGIT bispecific mAb) Early development Solid tumours Trial Population Patients Design Endpoints Status CVRM Phase III BTC with curative intent 750 • Randomised, Double-Blind, Placebo-Controlled, Multicentre • Primary en
Slide 40: Oncology: rilvegostomig, solid tumours
Approved medicines Late-stage development Oncology rilvegostomig (AZD2936, 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 • FPC
Slide 41: Oncology: saruparib, solid tumours
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 42: Oncology: saruparib, solid tumours
Approved medicines Late-stage development Oncology saruparib (AZD5305, PARP1 inhibitor) Early development Solid tumours Trial Population Patients Design Endpoints Status CVRM Phase I Locally advanced, unresectable or 16 • Part A: to assess the effect of multiple doses of itraconazole • Primary endpo
Slide 43: Oncology: volrustomig, cancer
Approved medicines Late-stage development Oncology volrustomig (MEDI5752, PD-1/CTLA-4 bispecific mAb) Early development Cancer Trial Population Patients Design Endpoints Status CVRM Phase Ib Advanced renal cell carcinoma 179 • Open-label, dose escalation and dose expansion trial • Primary endpoints
Slide 44: Oncology: volrustomig, solid tumours
Approved medicines Late-stage development Oncology volrustomig (MEDI5752, PD-1/CTLA-4 bispecific mAb) Early development Solid tumours Trial Population Patients Design Endpoints Status CVRM Phase III High-risk locally advanced cervical 1000 • Randomised, double-blind, placebo-controlled, multi-centre
Slide 45: Oncology: early-stage development
Oncology: early-stage development 45
Slide 46: Oncology: AZD0120, blood cancers
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 I/II Relapsed/refractory multiple myeloma 68 • Open-label, single-arm, multi-centre trial • Prima
Slide 47: Oncology: AZD0171, cancer
Approved medicines Late-stage development Oncology AZD0171 (anti-LIF mAb) 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, OS at 12 • FPCD: Q1 2022 NCT04999969
Slide 48: Oncology: AZD0305, blood cancers
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 • Open-label, dose escalation and dose expansion trial • Primary endpoints: occurrence of • FPCD: Q4 2023 NCT
Slide 49: Oncology: AZD0486, haematologic malignancies
Approved medicines Late-stage development Oncology AZD0486 (CD19/CD3 next-generation bispecific T-cell engager) Early development Haematologic malignancies Trial Population Patients Design Endpoints Status CVRM Phase I/II R/R B-ALL 120 • Multi-centre, open-label, single-arm dose escalation and • Pri
Slide 50: Oncology: AZD0754, solid tumours
Approved medicines Late-stage development Oncology AZD0754 (STEAP2 dnTGFbetaRII 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 endpo
Slide 51: Oncology: AZD1390, cancer
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- 138 • Open-label trial • Primary endpoints: safety, tolerability • FPCD: Q2 2018 NCT03423628 irra
Slide 52: Oncology: AZD3470, solid tumours and blood cancer
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 210 • Arm 1: AZD3470 • Primary endpoints: safety and • FPCD: Q1 2024 PRIMROSE • Glo
Slide 53: Oncology: AZD5335, solid tumours
Approved medicines Late-stage development Oncology AZD5335 (anti-FRα TOP1i ADC) Early development Solid tumours, ovarian cancer, lung cancer Trial Population Patients Design Endpoints Status CVRM Phase I/II Advanced solid tumour malignancies 150 • Module 1: AZD5335 monotherapy • Primary endpoints: s
Slide 54: Oncology: AZD5851, gastrointestinal cancer
Approved medicines Late-stage development Oncology AZD5851 (armoured TGFbetaRIIDN GPC3 CAR-T) 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 • P
Slide 55: Oncology: AZD5863, solid tumours
Approved medicines Late-stage development Oncology AZD5863 (CLDN18.2 x CD3 bispecific antibody) 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 e
Slide 56: Oncology: AZD6422, solid tumours
Approved medicines Late-stage development Oncology AZD6422 (CLDN18.2 CAR-T) Early development Solid tumours Trial Population Patients Design Endpoints Status CVRM Phase I Advanced or metastatic CLDN18.2- 96 • Open-label trial assessing anti-CLDN18.2 CAR-T cell therapy • Primary endpoints: incidence
Slide 57: Oncology: AZD8205, solid tumours
Approved medicines Late-stage development Oncology AZD8205 (B7H4 ADC) 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: AE, SAE, DLTs, • FPCD:
Slide 58: Oncology: AZD8421, breast cancer
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 59: Oncology: AZD9574, solid tumours
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 490 • Modular, open-label, multi-centre dose escalation and • Primary endpoints: safety
Slide 60: Oncology: AZD9592, lung cancer
Approved medicines Late-stage development Oncology AZD9592 (EGFR-cMET TOP1i ADC) Early development Lung cancer Trial Population Patients Design Endpoints Status CVRM Phase I Advanced solid tumours including 108 • Escalation phase, open-label, multi-centre trial • Primary endpoints (escalation): safe
Slide 61: Oncology: AZD9829, blood cancers
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 60 • Open-label, multicentre trial • Primary endpoints: safety and • FPCD: Q1 2024 NCT06179511
Slide 62: Oncology: FPI-2265, prostate cancer
Approved medicines Late-stage development Oncology FPI-2265 (PSMA radioconjugate) Early development Prostate cancer Trial Population Patients Design Endpoints Status CVRM Phase II/III PSMA-positive mCRPC previously 60 • Open-label, randomised, multi-centre trial • Primary endpoints: PSA50, safety •
Slide 63: Oncology: IPH5201, solid tumours
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 64: Oncology: NT-112, solid tumours
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 65: Oncology: NT-125, solid tumours
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 66: Oncology: NT-175, multiple cancers
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 24 • Open-label, single-arm, multi-centre trial with dose • Primary endpoint: incidence of DL
Slide 67: Oncology: oleclumab, solid tumours
Approved medicines Late-stage development Oncology oleclumab (CD73 mAb) 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 68: Oncology: sabestomig, cancer
Approved medicines Late-stage development Oncology sabestomig (AZD7789, PD-1/TIM3 bispecific mAb) 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 69: BioPharmaceuticals: approved medicines and late-stage development
BioPharmaceuticals: approved medicines and late-stage development 69
Slide 70: Andexxa, haematology
Approved medicines Late-stage development Oncology Andexxa (anti-factor Xa reversal) Early development Haematology Trial Population Patients Design Endpoints Status CVRM Phase IV Acute intracranial 1200 • Arm 1: Andexxa • Primary endpoint: proportion of • FPCD: Q2 2019 I8-513 (post-launch) haemorrha
Slide 71: roxadustat, anaemia
Approved medicines Late-stage development Oncology roxadustat (HIF-PH inhibitor) Early development Anaemia Trial Population Patients Design Endpoints Status CVRM Phase II/III Anaemia in lower-risk MDS 43 • Open-label roxadustat lead-in • Primary endpoint: haemoglobin • FPCD: Q2 2018 NCT03303066 pati
Slide 72: Wainua, 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 73: balcinrenone/dapagliflozin, heart failure, CKD
Approved medicines Late-stage development Oncology balcinrenone/dapagliflozin (MR 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, double-dum
Slide 74: 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 75: 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 primary 18 • Arm 1: baxdrostat 2-8mg QD • Primary endpoints: safety and • FPCD: Q3 202
Slide 76: baxdrostat/dapagliflozin, CKD
Approved medicines Late-stage development Oncology baxdrostat/dapagliflozin 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 2024 BaxDuo-Arctic • Arm
Slide 77: 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 1500 • Randomised, parallel, multi-centre, double-b
Slide 78: zibotentan/dapagliflozin, liver cirrhosis
Approved medicines Late-stage development Oncology zibotentan/dapagliflozin (ETA receptor antagonist/SGLT2 inhibitor) 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 79: 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 2518 • Randomised, double-blind, multi-centre, parallel-group, • Primary endpoint: time to first
Slide 80: Airsupra, asthma
Approved medicines Late-stage development Oncology Airsupra (PT027, SABA/ICS, pMDI) Early development Asthma Trial Population Patients Design Endpoints Status CVRM Phase III Mild to moderate asthma 1001 • Randomised, double-blind, multi-centre and parallel-group • Dual primary endpoints: change from
Slide 81: 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 Severe asthma 2200 • Randomised, double-blind, double-dummy, parallel group • Primary endpoint: change from • FPCD: Q1 2021 KAL
Slide 82: 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 cross-
Slide 83: 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 severe bilateral 413 • Arm 1: Fasenra 30mg Q8W s.c. • Primary endpoint: effect of Fa
Slide 84: 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 Severe, uncontrolled asthma 695 • Arm 1: Fasenra 30mg Q8W s.c. • Primary endpoint: annual asthma • FPCD: Q4 2
Slide 85: 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 86: Saphnelo, sclerosis and other 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 87: 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 88: Tezspire, severe uncontrolled 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 89: HFO1234ze, pMDI
Approved medicines Late-stage development Oncology HFO1234ze (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 • FP
Slide 90: HFO1234ze, pMDI
Approved medicines Late-stage development Oncology HFO1234ze (next-generation propellant) Early development pMDI Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers 108 • Randomised, double-blind, single-dose, single-centre, • Primary endpoint: AUCinf, AUClast • FPCD: Q
Slide 91: 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 1060 • Randomised, double-blind, placebo-controlled, parallel- • Primary endpoint: annualised rate of • FP
Slide 92: 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 1240 • Randomised, double-blind, placebo-controlled, parallel • Primary endpoint: annualised rate of • FPC
Slide 93: tozorakimab, severe 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 2902 • Randomised, double-blind, placebo-controlled, parallel • Primary endpoin
Slide 94: 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 35 3012 • Randomised, double-blind, placebo-controlled • Primary endpoint: efficacy • Data readout
Slide 95: Evusheld, COVID-19
Approved medicines Late-stage development Oncology Evusheld (AZD7442, tixagevimab + cilgavimab) 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 96: sipavibart, COVID-19
Approved medicines Late-stage development Oncology sipavibart (AZD3152, 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 endpoints
Slide 97: BioPharmaceuticals: early-stage development
BioPharmaceuticals: early-stage development 97
Slide 98: AZD0233, dilated cardiomyopathy
Approved medicines Late-stage development Oncology AZD0233 Early development Dilated Cardiomyopathy Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers 96 • Randomised, SAD/MAD dose escalating trial • Primary endpoint: safety and • FPCD: Q2 2024 NCT06381466 Tolerability
Slide 99: AZD0780, dyslipidaemia
Approved medicines Late-stage development Oncology AZD0780 (PCSK9 inhibitor) Early development Dyslipidaemia Trial Population Patients Design Endpoints Status CVRM Phase II Dyslipidaemia 428 • Randomised trial with equal distribution across 5 parallel • Primary endpoint: percent change in • FPCD: Q1
Slide 100: 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 101: AZD2373, chronic kidney disease
Approved medicines Late-stage development Oncology AZD2373 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 volunteers re
Slide 102: AZD2389, MASH
Approved medicines Late-stage development Oncology AZD2389 (anti-fibrotic mechanism) Early development MASH Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers 104 • Randomised, placebo-controlled SAD/MAD trial • Primary endpoints: safety and • FPCD: Q4 2023 NCT06138795
Slide 103: AZD2693, MASH
Approved medicines Late-stage development Oncology AZD2693 (antisense oligonucleotide) 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 • F
Slide 104: AZD3427, heart failure
Approved medicines Late-stage development Oncology AZD3427 (relaxin) Early development Heart failure Trial Population Patients Design Endpoints Status CVRM Phase II Heart failure and pulmonary 220 • Randomised, double-blind, placebo-controlled, multi-centre • Primary endpoint: change in PVR from • F
Slide 105: 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 volunteers 96 • Randomised, single-blind, placebo-controlled, single • Primary endpoints: safety and • FPCD
Slide 106: AZD5462, heart failure
Approved medicines Late-stage development Oncology AZD5462 (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 • FPC
Slide 107: AZD6234, obesity
Approved medicines Late-stage development Oncology AZD6234 (long-acting amylin) Early development Obesity with related comorbidities Trial Population Patients Design Endpoints Status CVRM Phase I Healthy participants who are 64 • SAD trial • Primary endpoint: safety • FPCD: Q4 2022 NCT05511025 overw
Slide 108: AZD7503, MASH
Approved medicines Late-stage development Oncology AZD7503 (antisense oligonucleotide) Early development MASH Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers 56 • SAD, 7 cohorts • Primary endpoints: safety and • FPCD: Q4 2021 NCT05143905 • Arm 1: AZD7503 s.c. tolera
Slide 109: 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 endpoint: safety and • FPCD: Q2 2023 NCT05848440 tolerability • LPCD: Q4 2023 • Data r
Slide 110: MEDI6570, cardiovascular disease
Approved medicines Late-stage development Oncology MEDI6570 Early development Cardiovascular disease Trial Population Patients Design Endpoints Status CVRM Phase IIb Post-myocardial infarction 400 • Evaluation of anti-inflammatory potential and effect on • Primary endpoints: safety and efficacy • FP
Slide 111: mitiperstat, cardiovascular disease
Approved medicines Late-stage development Oncology mitiperstat (MPO inhibitor) Early development Cardiovascular disease Trial Population Patients Design Endpoints Status CVRM Phase IIb HFpEF 711 • Randomised, double-blind • Primary endpoints: safety and efficacy • FPCD: Q3 2021 ENDEAVOR • Arm 1: 2.5
Slide 112: mitiperstat, MASH
Approved medicines Late-stage development Oncology mitiperstat (MPO inhibitor) Early development MASH Trial Population Patients Design Endpoints Status CVRM Phase II NASH 90 • Randomised, placebo-controlled, double-blind • Primary endpoints: safety, tolerability • FPCD: Q1 2023 COSMOS • Arm 1: 5mg m
Slide 113: 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 114: 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 24 • Randomised, single-blind, placebo-controlled trial • Primary endpoints: AEs and • Data anti
Slide 115: AZD1163, rheumatoid arthritis
Approved medicines Late-stage development Oncology AZD1163 (bispecific antibody) Early development Rheumatoid arthritis Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers 64 • Randomised, double-blind, placebo-controlled SAD/MAD • Primary endpoint: number of • FPCD: Q4
Slide 116: 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 • Multicentre, randomised, placebo-controlled, double-blind, • Primary endpoint: time to first
Slide 117: AZD6793, inflammatory diseases
Approved medicines Late-stage development Oncology AZD6793 (IRAK4) 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 118: 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 64 • Randomised, double-blind, placebo-controlled SAD trial • Primary endpoint: incidence of AEs • FPCD: Q4 2023
Slide 119: 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 I Global, Japanese and Chinese 144 • SAD repeating dose trial • Primary endpoints: safety and • FPCD: Q3 2022 NCT05452304 healthy
Slide 120: AZD8630, asthma
Approved medicines Late-stage development Oncology AZD8630 (inhaled TSLP) Early development Asthma Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers and 232 • SAD and MAD trial • Primary endpoints: safety and • FPCD: Q1 2022 NCT05110976 patients with asthma tolerabili
Slide 121: mitiperstat, COPD
Approved medicines Late-stage development Oncology mitiperstat (MPO inhibitor) Early development COPD Trial Population Patients Design Endpoints Status CVRM Phase II Moderate to severe COPD; 406 • Randomised, double-blind trial • Primary endpoint: time to first COPD • FPCD: Q1 2023 CRESCENDO age 40
Slide 122: AZD4041, opioid use disorder
Approved medicines Late-stage development Oncology AZD4041 (orexin 1 receptor antagonist) 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. AZD4041 and • Primary endpoints (Part
Slide 123: MEDI0618, osteoarthritis pain, migraine prevention
Approved medicines Late-stage development Oncology MEDI0618 (PAR2 antagonist mAb) Early development Osteoarthritis pain, migraine prevention Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers 48 • Randomised, double-blind, placebo-controlled MAD trial • Primary endpoin
Slide 124: 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 138 • Randomised, double-blind, placebo-controlled trial • Primary endpoints: efficac
Slide 125: MEDI1341, Parkinson's disease
Approved medicines Late-stage development Oncology MEDI1341 (alpha-synuclein mAb) Early development Parkinson’s disease Trial Population Patients Design Endpoints Status CVRM Phase I Parkinson's disease 25 • MAD trial • Primary endpoints: safety and • FPCD: Q3 2020 NCT04449484 • Arm 1: MEDI1341 i.v.
Slide 126: MEDI7352, osteoarthritis pain
Approved medicines Late-stage development Oncology MEDI7352 (NGF TNF bispecific mAb) 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 127: 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 128: AZD5148, infection
Approved medicines Late-stage development Oncology AZD5148 (Clostridium difficile mAb) Early development Infection Trial Population Patients Design Endpoints Status CVRM Phase I Healthy adult volunteers 84 • Randomised, double-blind, placebo-controlled, dose • Primary endpoint: safety • FPCD: Q2 202
Slide 129: Rare Disease: approved medicines and late-stage development
Rare Disease: approved medicines and late-stage development 129
Slide 130: Koselugo, neurofibromatosis type 1, solid tumours
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 who have 146 • Multi-centre, international trial with a par
Slide 131: Ultomiris, haematology, nephrology
Approved medicines Late-stage development Oncology Ultomiris (anti-C5 mAb) Early development Haematology, nephrology Trial Population Patients Design Endpoints Status CVRM Phase III Thrombotic microangiopathy-associated 106 • Arm 1: Ultomiris Q8W • Primary endpoint: TMA response • FPCD: Q4 2020 ALXN
Slide 132: 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 spectrum disorder 58 • Arm 1: Ultomiris Q8W • Primary endpoint: time to first • FPCD: Q4 2019 ALXN1210-NMO-307
Slide 133: Voydeya, haematology
Approved medicines Late-stage development Oncology Voydeya (factor D inhibitor) Early development Haematology Trial Population Patients Design Endpoints Status CVRM Phase III PNH with clinically significant EVH 86 • Arm 1: Voydeya + C5 Inhibitor • Primary endpoint: change from baseline • FPCD: Q1 20
Slide 134: acoramidis, ATTR-CM
Approved medicines Late-stage development Oncology acoramidis (ALXN2060) 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 baseline • FPCD: Q4 2020 ALXN2060-TAC-302
Slide 135: ALXN2220, amyloidosis
Approved medicines Late-stage development Oncology ALXN2220 (NI006, TTR depleter) 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 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 IIIa) 267 • Arm 1: anselamimab combined with SoC for PCD • Primary endpoint: tim
Slide 137: 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 138: eneboparatide, hypoparathyroidism
Approved medicines Late-stage development Oncology eneboparatide (PTH 1 inhibitor) Early development Hypoparathyroidism Trial Population Patients Design Endpoints Status CVRM Phase III Chronic hypoparathyroidism (cHP) 165 • Arm 1: 20mcg eneboparatide administered once daily via s.c. • Primary endpoi
Slide 139: gefurulimab, neurology, nephrology
Approved medicines Late-stage development Oncology gefurulimab (ALXN1720, anti-C5 humanised bispecific heavy-chain antibody) Early development Neurology, nephrology Trial Population Patients Design Endpoints Status CVRM Phase III Generalised myasthenia gravis 254 • Arm 1: weight-based maintenance tr
Slide 140: Rare Disease: early-stage development
Rare Disease: early-stage development 140
Slide 141: ALXN1910, bone metabolism
Approved medicines Late-stage development Oncology ALXN1910 (next-generation TNSALP ERT) Early development Bone metabolism Trial Population Patients Design Endpoints Status CVRM Phase I Healthy adults 48 • Randomised, placebo-controlled SAD • Primary endpoint: safety • FPCD: Q2 2022 ALXN1910-HV-101
Slide 142: 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 I Healthy adults 48 • Randomised, double-blind, placebo-controlled, SAD • Primary endpoint: safety and • FPC
Slide 143: ALXN2030, nephrology
Approved medicines Late-stage development Oncology ALXN2030 (siRNA targeting complement C3) Early development Nephrology Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers 48 • Randomised, placebo-controlled SAD • Primary endpoint: safety • FPCD: Q4 2022 ALXN2030-HV-10
Slide 144: ALXN2080, complement-mediated disease
Approved medicines Late-stage development Oncology ALXN2080 (factor D inhibitor) 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: danicopan, ophthalmology
Approved medicines Late-stage development Oncology danicopan (factor D inhibitor) Early development Ophthalmology Trial Population Patients Design Endpoints Status CVRM Phase II Geographic atrophy 365 • Arms 1-3: danicopan dosed at 100mg-400mg QD • Primary endpoint: mean rate of change • FPCD: Q3 20
Slide 146: vemircopan, haematology, nephrology, neurology
Approved medicines Late-stage development Oncology vemircopan (ALXN2050, factor D inhibitor) Early development Haematology, nephrology, neurology Trial Population Patients Design Endpoints Status CVRM Phase II Generalised myasthenia gravis 70 • Arm 1: vemircopan 180mg • Primary endpoint: MG-ADL tota
Slide 147: Glossary – 1 of 5
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 Vasculitis A
Slide 148: Glossary – 2 of 5
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 exacerbations dnTGFb D
Slide 149: Glossary – 3 of 5
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-acting beta
Slide 150: Glossary – 4 of 5
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 Monomethyl aurist
Slide 151: Glossary – 5 of 5
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 receptor