Slide 1: Clinical Trials Appendix
Clinical Trials Appendix FY and Q4 2023 Results Update 8th February 2024
Slide 2: Pipeline at a glance
Pipeline 178 17 at a glance projects in our new molecular entities development pipeline (NMEs) in our late-stage pipeline Across five focus therapy areas: Oncology BioPharmaceuticals 123 24 CVRM | R&I | V&I new molecular entities or regulatory approvals major lifecycle management in major markets pr
Slide 3: Key upcoming pipeline catalysts: 2024 and 2025
Key upcoming pipeline catalysts: 2024 and 2025 Oncology BioPharmaceuticals Rare Disease H1 2024 H2 2024 2025 Tagrisso – EGFRm NSCLC (1L) (FLAURA2) Lynparza + Imfinzi – endometrial cancer (1L) (DUO-E) Lynparza – gBRCA breast cancer (adjuvant) (OlympiA) (CN) Imfinzi – NSCLC (neoadjuvant) (AEGEAN) Enhe
Slide 4: Approved medicines: Next-wave pipeline
Clinical Trials Appendix: selected highlights BioPharmaceuticals Oncology Rare Disease Approved medicines: key LCM Dato-DXd (TROP2 ADC) tozorakimab (IL-33 ligand mAb) camizestrant (oral SERD) vemircopan (factor D inhibitor) Next-wave pipeline sipavibart (AZD3152, COVID-19 LAAB) saruparib (PARP1 inhi
Slide 5: Project movements since Q3 2023 update
Project movements since Q3 2023 update New to Phase I New to Phase II New to pivotal trial New to registration NME NME NME Life-cycle management AZD0305 (GPRC5D ADC) AZD0780 (PCSK9 inhibitor) ALXN2220# (transthyretin (TTR) depleter) Enhertu (HER2-targeting ADC) relapsed/refractory multiple myeloma d
Slide 6: Project movements since Q3 2023 update
Project movements since Q3 2023 update Removed from Phase I Removed from Phase II Removed from Phase III Approved/removed from registration Additional indication Life-cycle management NME tozorakimab (IL-33 ligand mAb) Imfinzi (PD-L1 mAb + CRT) Truqap (capivasertib, AKT inhibitor) + Faslodex diabeti
Slide 7: Q4 2023 Oncology new molecular entity pipeline
Q4 2023 Oncology new molecular entity1 pipeline Phase I Phase II Phase III 15 New Molecular Entities 10 New Molecular Entities 21 New Molecular Entities camizestrant + CDK4/6i (oral SERD + CDK4/6i) AZD0305 (GPRC5D ADC) AZD0486 (CD19-CD3 TCE) AZD0171 + Imfinzi + CTx (anti-LIF + PD-L1 + CTx) camizestr
Slide 8: Q4 2023 Oncology lifecycle management pipeline
Q4 2023 Oncology lifecycle management1 pipeline Phase I Phase II Phase III Under review 1 Project 9 Projects 33 Projects 4 Projects Enhertu (HER2 ADC) Enhertu (HER2 ADC) Enhertu (HER2 ADC) Calquence + R-CHOP (BTK inhibitor + R-CHOP) Calquence + venetoclax + obinutuzumab (BTK Calquence (BTK inhibitor
Slide 9: Q4 2023 BioPharmaceuticals new molecular entity pipeline
Q4 2023 BioPharmaceuticals new molecular entity1 pipeline Phase I Phase II Phase III Under review 17 New Molecular Entities 13 New Molecular Entities 5 New Molecular Entities 0 New Molecular Entities AZD1163 (bispecific antibody) AZD2373 atuliflapon (FLAP inhibitor) baxdrostat (aldosterone synthase
Slide 10: Q4 2023 BioPharmaceuticals lifecycle management pipeline
Q4 2023 BioPharmaceuticals new molecular entity1 pipeline Phase I Phase II Phase III Under review 0 Projects 2 Projects 12 Projects 2 Projects Andexxa (anti-factor Xa reversal) Breztri/Trixeo (LABA/LAMA/ICS) Fasenra (IL-5R mAb) urgent surgery KALOS LOGOS asthma MANDARA eosinophilic granulomatosis wi
Slide 11: Q4 2023 Rare Disease pipeline
Q4 2023 Rare Disease pipeline1 Phase I Phase II Phase III Under review 4 Projects 4 Projects 7 Projects 0 Projects ALXN1910 (next-generation TNSALP ERT) vemircopan (factor D inhibitor) acoramidis# (oral TTR stabiliser) bone metabolism generalised myasthenia gravis transthyretin amyloid cardiomyopath
Slide 12: Designations in our pipeline
Designations in our pipeline 3 11 9 12 20 Accelerated approvals Breakthrough / PRIME1 / Sakigake2 Fast Track Priority Review Orphan Andexxa acute major bleed (US) Beyfortus RSV mAb-YTE MELODY-MEDLEY (US) anselamibab AL amyloidosis (US) Beyfortus RSV mAb-YTE MELODY-MEDLEY (CN) ALXN2220 ATTR-CM (US) B
Slide 13: Oncology: approved medicines and late-stage pipeline
Oncology: approved medicines and late-stage pipeline 13
Slide 14: 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 200 • Arm 1: Tagrisso • Primary endpoint: PFS (BICR) • FPCD: Q4 20
Slide 15: 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 16: 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 Locally advanced or metastatic NSCLC 250 • Arm 1: Tagrisso + Orpathys • Primary endpoint:
Slide 17: 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. Arm • FPCD: Q1 2019 EMER
Slide 18: 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 Neoadjuvant NSCLC patients, Stage II 800 • Arm 1: Imfinzi + platinum-based chemotherapy • Primary endpoints: pCR snd EFS • FPCD: Q1 20
Slide 19: 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 Patients with locally advanced (Stage 999 • Arm 1: Imfinzi + oleclumab • Primary endoint: PFS • FPCD: Q2 2022 PACIFIC-9 III), unresect
Slide 20: 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 21: 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 22: Oncology: Lynparza multiple cancers
Approved medicines Oncology Lynparza (PARP inhibitor) Late-stage development Early development Multiple cancers 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
Slide 23: Oncology: Lynparza other combinations
Approved medicines Oncology Lynparza (PARP inhibitor) Late-stage development Early development Other combinations Trial Population Patients Design Endpoints Status CVRM Phase III 1L metastatic castration-resistant 906 • Arm 1: Lynparza + abiraterone • Primary endpoint: rPFS • FPCD: Q4 2018 PROpel pr
Slide 24: 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 • Primacy endpoint: PFS
Slide 25: 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- 900 • Randomised, open-label, parallel assignment • Primary endpoint: pCR
Slide 26: 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 gastro- 490 • Open-label, randomised, parallel group assignment • Primary en
Slide 27: 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 264 • Randomised, parallel group assignment • Primary endpoint: PFS • FPC
Slide 28: 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-expressing locally 115 • Non-randomised, parallel group assignment • Primary endpoints: DLT and ORR • FPC
Slide 29: 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 30: 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 31: 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 Locally advanced or metastatic TNBC 924 • Double-blind, randomised, comparative trial • Primary
Slide 32: Oncology: datopotamab deruxtecan breast cancer
Approved medicines Oncology 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- 733 • Open-label, randomised trial • Primary endpoints: PFS (BICR) and OS • FPCD:
Slide 33: Oncology: datopotamab deruxtecan NSCLC
Approved medicines Oncology datopotamab deruxtecan (TROP2 ADC) Late-stage development 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 34: Oncology: datopotamab deruxtecan NSCLC
Approved medicines Oncology datopotamab deruxtecan (TROP2 ADC) Late-stage development Early development NSCLC Trial Population Patients Design Endpoints Status CVRM Phase I Advanced or metastatic NSCLC 145 • Open-label, two-part (dose escalation and dose expansion), • Primary endpoints: DLT and safe
Slide 35: Oncology: datopotamab deruxtecan other cancers
Approved medicines Oncology datopotamab deruxtecan (TROP2 ADC) Late-stage development Early development Other cancers Trial Population Patients Design Endpoints Status CVRM Phase II Endometrial cancer, gastric cancer, 531 • Sub-study 1 (endometrial cancer); • Primary endpoints: ORR and safety • FPCD
Slide 36: Oncology: datopotamab deruxtecan other cancers
Approved medicines Oncology datopotamab deruxtecan (TROP2 ADC) Late-stage development 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 study with no blinding • Primary endpoint: ORR • FPCD:
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 1342 • 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 II HR+ advanced breast cancer 240 • Randomised, open-label, parallel-group, multi-centre trial • Primary e
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 580 • Double-arm randomised: • Primary endpoint: OS • FPCD: Q4 2022 LATIFY • Arm 1: ceralasertib
Slide 40: 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 III BTC with curative intent 750 • Randomized, Double-Blind, Placebo-Controlled, Multicenter • Primary en
Slide 41: 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 Nnn-HRRm mCSPC 1800 • Randomised, placebo-controlled trial • Primary endpoint: rPFS • FPCD: Q4 2023 EvoPAR
Slide 42: 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 1000 • Randomised, double-blind, placebo-controlled, multi-centre
Slide 43: Oncology: early-stage development
Oncology: early-stage development 43
Slide 44: 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 115 • Open-label, non-randomised trial • Primary endpoints: safety, OS at 12 • FPCD: Q1 2022 NCT04999969
Slide 45: 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 • Open-label, dose escalation and dose expansion trial • Primary endpoints: occurrence of • FPCD: Q4 2023 NCT
Slide 46: Oncology: AZD0486 haematologic malignancies
Approved medicines Oncology AZD0486 (TNB-486, CD19/CD3 next-generation bispecific T-cell engager) Late-stage development 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
Slide 47: 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 II Locally advanced unresectable or 123 • Open-label, multi-centre trial of AZD0901 administered via • Primary endpoints: AEs, S
Slide 48: 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- 120 • Open-label trial • Primary endpoints: safety, tolerability • FPCD: Q2 2018 NCT03423628 irra
Slide 49: 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 • Initiating PRIMROSE • Global
Slide 50: 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 150 • Module 1: AZD5335 monotherapy • Primary endpoints: s
Slide 51: Oncology: AZD5851 gastrointestinal cancer
Approved medicines Oncology AZD5851 (armoured TGFbetaRIIDN GPC3 CAR-T) Late-stage development Early development Gastrointestinal cancer Trial Population Patients Design Endpoints Status CVRM Phase I/II GPC3-positive advanced/recurrent HCC • Open-label, single-arm, multi-centre trial with dose • Prim
Slide 52: Oncology: AZD5863 solid tumours
Approved medicines Oncology AZD5863 (CLDN18.2 x 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 e
Slide 53: 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 assessing anti-CLDN18.2 CAR-T cell therapy • Primary endpoints: incidence
Slide 54: Oncology: AZD8205 solid tumours
Approved medicines Oncology 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, 280 • Open-label, non-randomised dose escalation, and • Primary endpoints: AE, SAE, DLTs, • FPCD
Slide 55: 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 • Arm 1: AZD8421 • Primary endpoints: safety and • FPCD: Q4 2023 NCT06188520 cancer • Ar
Slide 56: 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 57: 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 108 • Escalation phase, open-label, multi-centre trial • Primary endpoints (escalation): safe
Slide 58: 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 60 • Open-label, multicentre trial • Primary endpoints: safety and • Initiating NCT06179511 mal
Slide 59: 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 60: 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 61: 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 62: 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 63: 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 study • Primary endpoints: safety and efficacy • FPC
Slide 64: 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 65: 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 I/IIa Advanced solid tumours 804 • Modular, open-label, multi-centre dose escalation and • Primary endpoints: safety an
Slide 66: Oncology: volrustomig cancer
Approved medicines Oncology volrustomig (MEDI5752, PD-1/CTLA-4 bispecific mAb) Late-stage development 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 67: BioPharmaceuticals: approved medicines and late-stage development
BioPharmaceuticals: approved medicines and late-stage development 67
Slide 68: Andexxa haematology
Approved medicines Oncology Andexxa (anti-factor Xa reversal) Late-stage development 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 69: Farxiga heart failure and chronic kidney disease
Approved medicines Oncology Farxiga (SGLT2 inhibitor) Late-stage development Early development Heart failure and chronic kidney disease Trial Population Patients Design Endpoints Status CVRM Phase III Myocardial infarction 6400 • Arm 1: Farxiga 10mg QD • Primary endpoint: time to the first • FPCD: Q
Slide 70: Lokelma hyperkalaemia
Approved medicines Oncology Lokelma (sodium zirconium cyclosilicate) Late-stage development Early development Hyperkalaemia Trial Population Patients Design Endpoints Status CVRM Phase III Recurrent hyperkalaemia on 2800 • Arm 1: Lokelma 5g to 15g QD for 4 weeks on non-dialysis • Primary endpoint: t
Slide 71: roxadustat anaemia
Approved medicines Oncology roxadustat (HIF-PH inhibitor) Late-stage development Early development Anaemia Trial Population Patients Design Endpoints Status CVRM Phase III Anaemia in lower-risk MDS 184 • Open-label roxadustat lead-in • Primary endpoint: proportion of • FPCD: Q3 2017 MATTERHORN patie
Slide 72: 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 73: 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 74: tozorakimab diabetic kidney disease
Approved medicines Oncology tozorakimab (IL-33 ligand mAb) Late-stage development Early development Diabetic kidney disease Trial Population Patients Design Endpoints Status CVRM Phase II Adult patients with diabetic 581 • Arm 1: tozorakimab dose 1 + Farxiga • Primary endpoint: change from • FPCD: Q
Slide 75: 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 1500 • Randomised, parallel, multi-centre, double-b
Slide 76: 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 77: 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 1910 • Randomised, double-blind, multi-centre, parallel-group, • Primary: time to first severe as
Slide 78: 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 Severe asthma 2200 • Randomised, double-blind, double-dummy, parallel group • Primary endpoint: change from • FPCD: Q1 2021 KAL
Slide 79: 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 Moderate to very severe 542 • Randomised, double-blind, 12-week (with an extension to • Primary endpoints: number of • FPCD: Q3 2
Slide 80: 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 81: Fasenra 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 Severe, uncontrolled asthma 695 • Arm 1: Fasenra 30mg Q8W s.c. • Primary endpoint: annual asthma • FPCD: Q4 2
Slide 82: 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 83: Saphnelo sclerosis
Approved medicines Oncology Saphnelo (type I interferon receptor mAb) Late-stage development Early development Sclerosis 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 2023 DAISY • Arm 2:
Slide 84: 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 85: 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 86: 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 87: 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 • Randomised, double-blind, single-dose, cross-over trial to • Primary endpoint: AUClast and Cmax • FPCD:
Slide 88: tozorakimab asthma
Approved medicines Oncology tozorakimab (IL-33 ligand mAb) Late-stage development Early development Asthma Trial Population Patients Design Endpoints Status CVRM Phase II Adults with uncontrolled 250 • Randomised, double-blind, placebo-controlled trial • Primary endpoint: change from • FPCD: Q4 2020
Slide 89: 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 1060 • Randomised, double-blind, placebo-controlled, parallel- • Primary endpoint: annualised rate of • FP
Slide 90: 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 1240 • Randomised, double-blind, placebo-controlled, parallel • Primary endpoint: annualised rate of • FPC
Slide 91: tozorakimab severe viral LRTD
Approved medicines Oncology tozorakimab (IL-33 ligand mAb) Late-stage development Early development Severe viral LRTD Trial Population Patients Design Endpoints Status CVRM Phase III Adults hospitalised for viral 2902 • Randomised, double-blind, placebo-controlled, parallel • Primary endpoint: progr
Slide 92: 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 35 3012 • Randomised, double-blind, placebo-controlled • Primary endpoint: efficacy • Data readout
Slide 93: 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 94: sipavibart COVID-19
Approved medicines Oncology sipavibart (AZD3152, 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 endpoints
Slide 95: BioPharmaceuticals: early-stage development
BioPharmaceuticals: early-stage development 95
Slide 96: AZD0780 dyslipidaemia
Approved medicines Oncology AZD0780 (PCSK9 inhibitor) Late-stage development Early development Dyslipidaemia Trial Population Patients Design Endpoints Status CVRM Phase II Dyslipidemia 175 • Randomised trial with equal distribution across 5 parallel • Primary endpoint: percent change in • FPCD: Q1
Slide 97: AZD2373 chronic kidney disease
Approved medicines Oncology AZD2373 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 volunteers re
Slide 98: AZD2389 MASH
Approved medicines Oncology AZD2389 (anti-fibrotic mechanism) Late-stage development 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 99: AZD2693 MASH
Approved medicines Oncology AZD2693 (antisense oligonucleotide) 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 • F
Slide 100: AZD3427 heart failure
Approved medicines Oncology AZD3427 (relaxin) Late-stage development 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 101: 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 104 • Randomised, single-blind, placebo-controlled, single • Primary endpoints: safety and • FPC
Slide 102: AZD5462 heart failure
Approved medicines Oncology AZD5462 (relaxin) Late-stage development Early development Heart failure Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers 98 • Single-centre SAD and MAD • Primary endpoints: safety and • FPCD: Q4 2021 NCT04994106 • Part A: SAD (8 cohorts)
Slide 103: AZD6234 obesity
Approved medicines Oncology AZD6234 (long-acting amylin) Late-stage development 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 104: AZD7503 MASH
Approved medicines Oncology AZD7503 (antisense oligonucleotide) Late-stage development 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 105: 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 endpoint: safety and • FPCD: Q2 2023 NCT05848440 tolerability • Data anticipated: H1 2
Slide 106: balcinrenone/dapagliflozin heart failure
Approved medicines Oncology balcinrenone/dapagliflozin (MR modulator + SGLT2 inhibitor) Late-stage development Early development Heart failure Trial Population Patients Design Endpoints Status CVRM Phase IIb Heart failure with chronic 500 • Randomised, stratified according to T2DM and eGFR (≥20 to •
Slide 107: MEDI6570 cardiovascular disease
Approved medicines Oncology MEDI6570 Late-stage development 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 108: mitiperstat cardiovascular disease
Approved medicines Oncology mitiperstat (MPO inhibitor) Late-stage development 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 109: mitiperstat MASH
Approved medicines Oncology mitiperstat (MPO inhibitor) Late-stage development 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 110: atuliflapon asthma
Approved medicines Oncology atuliflapon (FLAP inhibitor) Late-stage development Early development Asthma Trial Population Patients Design Endpoints Status CVRM Phase II Patients with moderate-to- 1102 • Randomised, placebo-controlled, double-blind, multi- • Primary endpoint: time to first • FPCD: Q2
Slide 111: 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 64 • Randomised, double-blind, placebo-controlled SAD/MAD • Primary endpoint: number of • FPCD: Q4
Slide 112: 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 • Multicentre, randomised, placebo-controlled, double-blind, • Primary endpoint: time to first
Slide 113: AZD5055 IPF and other ILDs
Approved medicines Oncology AZD5055 (oral porcupine inhibitor) Late-stage development Early development IPF and other ILDs with progressive fibrosis Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers 90 • SAD/MAD trial • Primary endpoints: safety and • FPCD: Q4 2021 NC
Slide 114: 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 study to • Primary endpoints: safety and • FPCD: Q4 2022 NCT
Slide 115: 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 116: 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 I Healthy volunteers 144 • SAD • Primary endpoints: safety and • FPCD: Q3 2022 NCT05452304 • Arm1: AZD7798 tolerability • Data rea
Slide 117: AZD8630 asthma
Approved medicines Oncology AZD8630 (inhaled TSLP) Late-stage development 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 118: 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; 406 • Randomised, double-blind trial • Primary endpoint: time to first COPD • FPCD: Q1 2023 CRESCENDO age 40
Slide 119: 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 I Healthy recreational opioid 36 • Randomised, double-blind, placebo-controlled, fixed • Primary endpoint: chan
Slide 120: 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 48 • Randomised, double-blind, placebo-controlled MAD trial • Primary endpoin
Slide 121: 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 122: MEDI1341 Parkinson's disease
Approved medicines Oncology MEDI1341 (alpha-synuclein mAb) Late-stage development 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 123: 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 124: Rare Disease: approved medicines and late-stage development
Rare Disease: approved medicines and late-stage development 124
Slide 125: Koselugo neurofibromatosis type 1
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 who have 146 • Multi-centre, international trial with a par
Slide 126: 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-associated 106 • Arm 1: Ultomiris Q8W • Primary endpoint: TMA response • FPCD: Q4 2020 ALXN
Slide 127: 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 spectrum disorder 58 • Arm 1: Ultomiris Q8W • Primary endpoint: time to first • FPCD: Q4 2019 ALXN1210-NMO-307
Slide 128: Voydeya haematology, ophthalmology
Approved medicines Oncology Voydeya (danicopan, factor D inhibitor) Late-stage development Early development Haematology, ophthalmology Trial Population Patients Design Endpoints Status CVRM Phase III PNH with clinically significant EVH 86 • Arm 1: Voydeya + C5 Inhibitor • Primary endpoint: change f
Slide 129: acoramidis ATTR-CM
Approved medicines Oncology acoramidis (ALXN2060) 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 baseline • FPCD: Q4 2020 ALXN2060-TAC-302
Slide 130: 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 131: 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 (Mayo Stage IIIa) 267 • Arm 1: anselamimab combined with SoC for PCD • Primary endpoint: tim
Slide 132: 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 III Hypophosphatasia 114 • Arm 1: placebo on Day 1 followed by Q2W via s.c. injection • Primar
Slide 133: 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 gravis 254 • Arm 1: weight-based maintenance tr
Slide 134: Rare Disease: early-stage development
Rare Disease: early-stage development 134
Slide 135: 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 • Primary endpoint: safety • FPCD: Q2 2022 ALXN1910-HV-101
Slide 136: 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 • Primary endpoint: safety and • FPC
Slide 137: 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 Generalised myasthenia gravis 70 • Arm 1: vemircopan 180mg • Primary endpoint: MG-ADL tota
Slide 138: 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 139: 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 • Primary endpoint: safety • FPCD: Q4 2022 ALXN2030-HV-10
Slide 140: tarperprumig haematology
Approved medicines Oncology tarperprumig (ALXN1820, anti-properdin) Late-stage development Early development Haematology Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers 60 • Arm 1: tarperprumig administered s.c. or i.v., multiple • Primary endpoint: participants wit
Slide 141: Glossary – 1 of 4
NEXT Glossary – 1 of 4 14C Carbon 14 BA Bioavailability CD39 Cluster of differentiation 39 1L, 2L, 3L 1st-, 2nd- or 3rd-line BAFF B-cell activating factor CD73 Cluster of differentiation 73 5-FU 5-fluorouracil B-ALL B cell acute lymphoblastic leukaemia CD8 Cluster of differentiation 8 6MWT 6-minute
Slide 142: Glossary – 2 of 4
BACK NEXT Glossary – 2 of 4 CTx Chemotherapy ESAI Eczema Area and Severity Index H1 H1-antihistamine CV Cardiovascular ESCC Esophageal squamous cell carcinoma hADME Human mass balance CVOT Cardiovascular outcomes trial ESKD Early-stage kidney disease HCC Hepatocellular carcinoma CXCR2 C-X-C Motif ch
Slide 143: Glossary – 3 of 4
BACK NEXT Glossary – 3 of 4 IDFS Invasive disease-free survival MCC Mucociliary clearance NMOSD Neuromyelitis optica spectrum disorder IgAN Immunoglobulin A nephropathy MCL Mantle cell lymphona NRG National Clinical Trials Network in Oncology IHF Impaired hepatic function mCRPC Metastatic castrate-r
Slide 144: Glossary – 4 of 4
BACK Glossary – 4 of 4 post-BD Post-bronchodilator s.c. Subcutaneous TKI Tyrosine kinase Inhibitor pPCI Primary percutaneous coronary intervention SABA Short-acting beta2-agonist TLR Toll-like receptor 9 PR Partial response SAD Single ascending dose TMA Thrombotic microangiopathy pre-BD Pre-bronchod