Slide 1: Clinical trials appendix FY 2021 results update
Clinical trials appendix FY 2021 results update
Slide 2: Movement since Q3 2021 update
Movement since Q3 2021 update New to Phase I New to Phase II New to Pivotal trial New to registration NME NME NME NME AZD5055 AZD0171 + Imfinzi + CTx eplontersen# Imfinzi +/- tremelimumab + CTx POSEIDON [US, EU & JP] 1 Porcupine inhibitor idiopathic anti-LIF mAb + PD-L1 mAb + CTx ligand-conjugated a
Slide 3: Movement since Q3 2021 update
Movement since Q3 2021 update Removed from Phase I Removed from Phase II Removed from Phase III Removed from registration NME NME NME NME AZD0284 AZD2811 nanoparticle AZD2816 (next generation COVID-19 vaccine) Evusheld (AZD7442) [US EUA] 2 RORg psoriasis/respiratory Aurora B inhibitor solid tumours
Slide 4: Q4 2021 Oncology new molecular entity pipeline
Q4 2021 Oncology new molecular entity 1 pipeline Phase 1 Phase 2 Phase 3 15 New Molecular Entities 17 New Molecular Entities 15 New Molecular Entities AZD0466# adavosertib# camizestrant+CDK4/6i SERENA-6 AZD1390 AZD5305 camizestrant+palbociclib SERENA-4 BCL2/xL haematological Wee1 ovarian / uterine s
Slide 5: Q4 2021 Oncology life-cycle management pipeline
Q4 2021 Oncology life-cycle management1 pipeline Phase 1 Phase 2 Phase 3 Under review 1 Project 7 Projects 32 Projects 3 Projects Enhertu# (platform) DESTINY-Breast08 Enhertu# (platform) DESTINY-Breast07 Calquence# ECHO Calquence#+venetoclax+obinutuzumab Calquence+R-CHOP ESCALADE Enhertu# DESTINY-Br
Slide 6: Q4 2021 BioPharmaceuticals new molecular entity pipeline
Q4 2021 BioPharmaceuticals new molecular entity1 pipeline Phase 1 Phase 2 Phase 3 14 New Molecular Entities 15 New Molecular Entities 5 New Molecular Entities AZD2373 AZD2693 AZD1402# AZD4831 brazikumab¶ Podocyte health nephropathy nonalcoholic steatohepatitis inhaled IL-4Ra asthma MPO HFpEF IL-23 c
Slide 7: Q4 2021 BioPharmaceuticals life-cycle management pipeline
Q4 2021 BioPharmaceuticals life-cycle management1 pipeline Phase 1 Phase 2 Phase 3 Under review 0 Projects 5 Projects 14 Projects 1 Project Farxiga/Forxiga DAPA-MI Fasenra ARROYO Breztri Fasenra# OSTRO SGLT2 prevention of HF and CV death IL-5R chronic spontaneous urticaria LABA/LAMA/ICS asthma IL-5R
Slide 8: Q4 2021 Rare Disease pipeline
Q4 2021 Rare Disease pipeline1 Phase 1 Phase 2 Phase 3 Under review 3 Projects 3 Projects 8 Projects 2 Projects ALXN1720 ALXN2050 acoramidis (ALXN2060) Ultomiris anti-C5 bi-specific minibody generalised oral factor D inhibitor paroxysmal nocturnal oral TTR stabilizer transthyretin amyloid cardiomyop
Slide 9: Estimated key regulatory submission acceptances
Estimated key regulatory submission acceptances acoramidis (ALXN2060) ] Transthyretin Amyloid Cardiomyopathy danicopan (ALXN2040) PNH-EVH Fasenra severe asthma (China) Vaxzevria / COVID-19 Vaccine Imfinzi+ tremelimumab + CRT LDS-SCLC datopotamab deruxtecan breast cancer CAEL-101 AstraZeneca (US) ADR
Slide 10: Designations in our pipeline
Designations in our pipeline 2 16 8 12 30 Accelerated approvals Breakthrough / PRIME1 / Sakigake2 Fast Track Priority Review Orphan Andexxa Acute Major Bleed (US) danicopan (ALXN2040) PNH-EVH (US) Sapnelo SLE (US) Brilinta stroke THALES (US) ALXN1830 WAIHA (EU) Calquence MCL 1L (US) danicopan (ALXN2
Slide 11: Oncology – approved medicines and late-stage pipeline
Oncology – approved medicines and late-stage pipeline
Slide 12: Tagrisso: NSCLC trials
Approved medicines Oncology Tagrisso (highly-selective, irreversible EGFRi) Late-stage development Early development NSCLC Trial Population Patients Design Endpoints Status CVRM Phase III Adjuvant EGFRm NSCLC 682 • Arm 1: Tagrisso QD following complete tumour resection, • Primary endpoint: DFS • FPC
Slide 13: Tagrisso: NSCLC combinations
Approved medicines Oncology Tagrisso (highly-selective, irreversible EGFRi) Late-stage development Early development NSCLC, combinations Trial Population Patients Design Endpoints Status CVRM Phase III Neoadjuvant EGFRm NSCLC 351 • Arm 1: placebo plus pemetrexed/carboplatin or • Primary endpoint: mP
Slide 14: Imfinzi: early NSCLC
Approved medicines Oncology Imfinzi (PD-L1 mAb) Late-stage development Early development NSCLC, early disease Trial Population Patients Design Endpoints Status CVRM Phase III Completely resected 332 • Arm 1: Imfinzi + SoC chemo • Primary endpoint: DFS • FPCD: Q3 2020 MERMAID-1 Stage II and III NSCLC
Slide 15: Imfinzi: lung cancer, early disease
Approved medicines Imfinzi (PD-L1 mAb) Oncology Late-stage development Early development Lung cancer, early disease Trial Population Patients Design Endpoints Status CVRM Phase II NSCLC, patients who 340 Open-label, biomarker-directed, multicentre trial • Primary outcome: ORR • FPCD: Q1 2018 HUDSON
Slide 16: Imfinzi +/- treme: advanced lung cancer
Approved medicines Oncology Imfinzi (PD-L1 mAb) +/- treme (CTLA-4 mAb) Late-stage development Early development Lung cancer, advanced disease Trial Population Patients Design Endpoints Status CVRM Phase III NSCLC 1L 650 • Arm 1: Imfinzi Q4W • Primary endpoint: OS • FPCD: Q1 2017 PEARL • Arm 2: chemo
Slide 17: Imfinzi +/- treme: SCLC
Approved medicines Oncology Imfinzi (PD-L1 mAb) +/- treme (CTLA-4 mAb) Late-stage development Early development SCLC, advanced disease Trial Population Patients Design Endpoints Status CVRM Phase III Limited stage SCLC 1L 600 • Arm 1: Imfinzi + tremelimumab (4 doses) • Primary endpoints: PFS, OS • F
Slide 18: Imfinzi: other cancers
Approved medicines Oncology Imfinzi (PD-L1 mAb) Late-stage development Early development Other cancers Trial Population Patients Design Endpoints Status CVRM Phase III Non-muscle invasive bladder 1018 • Arm 1: BCG (Induction + maintenance) • Primary endpoint: DFS • FPCD: Q2 2018 POTOMAC cancer • Arm
Slide 19: Imfinzi +/- treme: other advanced cancers
Approved medicines Oncology Imfinzi (PD-L1 mAb) +/- treme (CTLA-4 mAb) Late-stage development Early development Other cancers, advanced disease Trial Population Patients Design Endpoints Status CVRM Phase III Bladder cancer 1L 1220. • Arm 1: Imfinzi + tremelimumab + SoC • Primary endpoint: OS • FPCD
Slide 20: Imfinzi +/- treme: other advanced cancers
Approved medicines Oncology Imfinzi (PD-L1 mAb) +/- treme (CTLA-4 mAb) Late-stage development Early development Other cancers, advanced disease Trial Population Patients Design Endpoints Status CVRM Phase II mTNBC 1L 220 • Arm 1 Imfinzi + paclitaxel • Primary endpoints: safety and • FPCD: Q1 2019 BE
Slide 21: 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 BRCAm adjuvant breast 1836 • Arm 1: Lynparza BID 12-month duration • Primary endpoint: invasive disease- • FPCD: Q2 2014 Ol
Slide 22: 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 Metastatic castration- 904 • Arm 1: Lynparza + abiraterone • Primary endpoint: rPFS • FPCD: Q4 2018 PROpel resistant pros
Slide 23: 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 Advanced ovarian cancer 1L 1,256 Non tBRCAm (tumour BRCA) patients • Primary endpoint: PFS • FPCD: Q1 2019 DuO-O • Arm
Slide 24: 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 • FP
Slide 25: 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 Ib/II HER2-positive metastatic breast cancer 450 Randomised open label sequential assignment • Primary endpoints:
Slide 26: 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 endpoi
Slide 27: 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 • FPCD:
Slide 28: 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 II HER2 expressing tumours 280 Non randomised single group assignment • Primary endpoint: ORR • FPCD: Q4 2020 DES
Slide 29: Calquence: blood cancers
Approved medicines Oncology Calquence (BTK inhibitor) Late-stage development Early development Blood cancers Trial Population Patients Design Endpoint(s) Status CVRM Phase III Previously untreated CLL 535 • Arm A: chlorambucil + obinutuzumab • Primary endpoint: PFS (Arm A vs. Arm • FPCD: Q2 2015 ACE
Slide 30: Calquence: blood cancers
Approved medicines Oncology Calquence (BTK inhibitor) Late-stage development Early development Blood cancers Trial Population Patients Design Endpoint(s) Status CVRM Phase I/II CLL/SLL/Richter's 306 Calquence monotherapy • Primary endpoints: safety, PK, PD • FPCD: Q1 2014 ACE-CL-001 transformation D
Slide 31: Calquence: blood and other cancers
Approved medicines Oncology Calquence (BTK inhibitor) Late-stage development Early development Blood and other cancers Trial Population Patients Design Endpoint(s) Status CVRM Phase I/II Chinese adults R/R MCL and 105 Part 1: R/R B-cell Malignancies • Primary endpoints: safety, ORR • FPCD: Q2 2020 D
Slide 32: Koselugo: paediatric NF1, solid tumours
Approved medicines Oncology Koselugo (selumetinib, MEK inhibitor) Late-stage development Early development Paediatric neurofibromatosis type 1, solid tumours Trial Population Patients Design Endpoints Status CVRM Phase II Paediatric NF1 50 (stratum 1) Single arm: Koselugo 25mg/m2 BID with 2 strata:
Slide 33: 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 II Lung PSC and other NSCLC 65 Single arm trial • Primary endpoint: ORR • FPCD: Q1 2017 NCT02897479 • Orpath
Slide 34: Capivasertib: breast and prostate cancer
Approved medicines Oncology Late-stage development Capivasertib (AKT inhibitor) Early development Breast cancer, prostate cancer Trial Population Patients Design Endpoints Status CVRM Phase III Locally advanced or 924 Double-blind randomised comparative trial • Primary endpoint: OS • FPCD: Q3 2019 C
Slide 35: Monalizumab: cancers
Approved medicines Oncology Late-stage development Monalizumab (NKG2a mAb) Early development Cancers Trial Population Patients Design Endpoints Status CVRM Phase III Recurrent or metastatic 600 • Arm A: monalizumab + cetuximab i.v. • Primary: OS • FPCD: Q4 2020 INTERLINK-1 HNSCC, 2L • Arm B: placebo
Slide 36: Camizestrant: breast cancer
Approved medicines Oncology Late-stage development Camizestrant (AZD9833, next generation oral SERD) Early development Breast cancer Trial Population Patients Design Endpoints Status CVRM Phase III HR+ HER2- breast cancer 1342 Randomised, double-blind, comparative trial • Primary endpoint: PFS • FPC
Slide 37: Datopotamab deruxtecan: NSCLC
Approved medicines Oncology Late-stage development Datopotamab deruxtecan (TROP2 ADC) Early development NSCLC Trial Population Patients Design Endpoints Status CVRM Phase III Previously treated advanced 590 Randomised, open label, parallel assignment • Primary endpoints: PFS, OS • FPCD: Q1 2021 TROP
Slide 38: Datopotamab deruxtecan: NSCLC and other cancers
Approved medicines Oncology Late-stage development Datopotamab deruxtecan (TROP2 ADC) Early development NSCLC and other cancers Trial Population Patients Design Endpoints Status CVRM Phase III Inoperable or metastatic HR+, 700 Open-label, randomized • Primary endpoints: PFS, OS • FPCD: Q4 2021 TROPI
Slide 39: Oncology – early-stage development
Oncology – early-stage development
Slide 40: AZD0466 (Bcl2/xL inhibitor)
Approved medicines Oncology Late-stage development AZD0466 (Bcl2/xL inhibitor) Early development Cancer Trial Population Patients Design Endpoints Status CVRM Phase I/II Advanced haematologic 160 Module 1 • Primary endpoint: safety • FPCD: Q2 2021 NCT04865419 malignancies Part A: Dose escalation • S
Slide 41: MEDI1191 (IL-12 modRNA)
Approved medicines Oncology Late-stage development MEDI1191 (IL-12 modRNA) Early development Cancer Trial Population Patients Design Endpoints Status CVRM Phase I Advanced solid tumours 87 Open-label, dose-escalation and expansion trial of MEDI1191 • Primary endpoints: safety and • FPCD: Q2 2019 NCT
Slide 42: AZD1390 (ATM inhibitor)
Approved medicines Oncology Late-stage development AZD1390 (ATM inhibitor) Early development Cancer Trial Population Subjects Design Endpoints Status CVRM Phase I Recurrent glioblastoma Up to 162 Open label trial • Primary endpoints: safety, • FPCD: Q2 2018 NCT03423628 eligible for re-irradiation, •
Slide 43: Adavosertib (WEE-1 inhibitor)
Approved medicines Oncology Late-stage development Adavosertib (WEE-1 inhibitor) Early development Ovarian cancer, uterine serous cancer, solid tumours Trial Population Patients Design Endpoints Status CVRM Phase II Platinum-resistant (PR) 95 • Arm B: paclitaxel + adavosertib • Primary endpoint: ORR
Slide 44: AZD4573 (CDK9 inhibitor)
Approved medicines Oncology Late-stage development AZD4573 (CDK9 inhibitor) Early development Blood cancers Trial Population Patients Design Endpoints Status CVRM Phase I/II R/R haematologic 78 Open label, non-randomised trial • Primary endpoint Part A: safety • FPCD: Q1 2021 NCT04630756 malignancie
Slide 45: AZD5305 (PARP1 inhibitor)
Approved medicines Oncology Late-stage development AZD5305 (PARP1 inhibitor) Early development Solid tumours Trial Population Patients Design Endpoints Status CVRM Phase I/IIa Advanced, metastatic 715 A modular, open-label, multicentre trial dose escalation • Primary endpoints: safety and • FPCD: Q4
Slide 46: MEDI5752 (PD-1/CTLA-4 bispecific mAb)
Approved medicines Oncology Late-stage development MEDI5752 (PD-1/CTLA-4 bispecific mAb) Early development Cancer Trial Population Patients Design Endpoints Status CVRM Phase I/IIa Advanced solid tumours 366 Open-label, dose-escalation and dose-expansion trial • Dose escalation primary endpoints: •
Slide 47: AZD5991 (MCL1 inhibitor)
Approved medicines Oncology Late-stage development AZD5991 (MCL1 inhibitor) Early development Blood cancers Trial Population Patients Design Endpoints Status CVRM Phase I/Ib/IIa Relapsed/refractory 144 • Arm 1: AZD5991 dose escalation and expansion in R/R • Primary endpoint: safety • FPCD: Q3 2017 N
Slide 48: Ceralasertib (AZD6738, ATR inhibitor)
Approved medicines Oncology Late-stage development Ceralasertib (AZD6738, ATR inhibitor) Early development Cancer Trial Population Patients Design Endpoints Status CVRM Phase II Solid tumours 52 • Cohort A: ceralasertib; ATM-altered AST • Cohort A primary endpoint: ORR • FPCD: Q1 2021 PLANETTE mCRPC
Slide 49: AZD7648 (DNA-PK inhibitor)
Approved medicines Oncology Late-stage development AZD7648 (selective DNA-PK inhibitor) Early development Advanced solid tumours Trial Population Patients Design Endpoints Status CVRM Phase I Advanced malignancies 234 Modular dose escalation and dose expansion trial • Primary outcomes: safety and •
Slide 50: AZD8701 (FOXP3 antisense oligonucleotide)
Approved medicines Oncology Late-stage development AZD8701 (FOXP3 antisense oligonucleotide) Early development Solid tumours Trial Population Patients Design Endpoints Status CVRM Phase I/Ib Advanced solid tumours 123 Dose escalation and dose expansion trial • Primary endpoints: safety & tolerabilit
Slide 51: MEDI9253 (rNDV-IL-12)
Approved medicines Oncology Late-stage development MEDI9253 (rNDV-IL-12) Early development Solid tumours Trial Population Patients Design Endpoints Status CVRM Phase I Advanced solid tumours 86 Open-label, dose-escalation and expansion trial • Primary endpoint: safety and • FPCD: Q4 2020 NCT04613492
Slide 52: IPH5201 (CD39 mAb)
Approved medicines Oncology Late-stage development IPH5201 (CD39 mAb) Early development Solid tumours Trial Population Patients Design Endpoints Status CVRM Phase I Advanced solid tumours 204 Open-label, dose-escalation trial to determine MTD of IPH5201 • Primary endpoints: AE, SAE, DLT • FPCD: Q1 2
Slide 53: Oleclumab (CD73 mAb)
Approved medicines Oncology Late-stage development Oleclumab (CD73 mAb) Early development Cancer Trial Population Patients Design Endpoints Status CVRM Phase Ib/II Pancreatic 339 • Arm A1: gemcitabine and nab paclitaxel i.v. • Primary endpoints: safety and anti- • FPCD: Q2 2018 NCT03611556 1L and 2L
Slide 54: AZD2936 (PD-1/TIGIT bispecific mAb)
Approved medicines Oncology Late-stage development AZD2936 (PD-1/TIGIT Bispecific mAb) Early development Solid tumours Trial Population Patients Design Endpoints Status CVRM Phase I NSCLC 147 Open-label, non-randomised dose-escalation and dose- • Part A Dose escalation primary • FPCD: Q4 2021 NCT049
Slide 55: AZD7789 (PD-1/TIM3 bispecific mAb)
Approved medicines Oncology Late-stage development AZD7789 (PD-1/TIM3 Bispecific mAb) Early development Cancer Trial Population Patients Design Endpoints Status CVRM Phase I/IIa NSCLC 81 Open-label, non-randomised dose-escalation and dose- • Primary endpoints: AE, SAE, DLTs, ORR • FPCD: Q4 2021 NCT0
Slide 56: AZD8205 (B7H4 ADC)
Approved medicines Oncology Late-stage development AZD8205 (B7H4 ADC) Early development Cancer Trial Population Patients Design Endpoints Status CVRM Phase I/II Breast Cancer, 198 Open-label, non-randomised dose-escalation and dose- • Primary endpoints: AE, SAE, DLTs, • FPCD: Q1 2022 NCT05123482 Cho
Slide 57: AZD0171 (anti-LIF mAb)
Approved medicines Oncology Late-stage development AZD0171 (anti-LIF mAb) Early development Cancer Trial Population Patients Design Endpoints Status CVRM Phase II 1L metastatic pancreatic 115 Open-label, non-randomised trial of AZD0171 + Imfinzi + • Primary endpoints: Safety, OS at 12m • FPCD: Q1 20
Slide 58: BioPharmaceuticals – approved medicines and late-stage pipeline
BioPharmaceuticals – approved medicines and late-stage pipeline
Slide 59: Farxiga: heart failure and CKD
Approved medicines Oncology Farxiga (SGLT2 inhibitor) Late-stage development Early development Heart failure and chronic kidney disease Trial Trial Population Population Patients Design Endpoints Endpoints Status CVRM Phase III Phase III Patients With with CHF patients CKD HFpEF 4,304 6100 •• Arm Ar
Slide 60: Lokelma: hyperkalaemia
Approved medicines Oncology Lokelma (sodium zirconium cyclosilicate) Late-stage development Early development Hyperkalaemia Trial Population Patients Design Endpoints Status CVRM Phase IIIb Patients with ESRD with 134 • Arm 1: Lokelma 5g QD for 8 weeks on non-dialysis days. • Primary endpoint: propo
Slide 61: Roxadustat: anaemia
Approved medicines Oncology Late-stage development Roxadustat (HIF-PH inhibitor) Early development Anaemia Trial Population Patients Design Endpoints Status CVRM Phase III Anaemia in CKD in patients 922 • Arm 1: roxadustat • Primary endpoint: haemoglobin • FPCD: Q4 2012 ANDES not receiving dialysis
Slide 62: Roxadustat: anaemia
Approved medicines Oncology Late-stage development Roxadustat (HIF-PH inhibitor) Early development Anaemia Trial Population Patients Design Endpoints Status CVRM Phase III Anaemia in CKD in patients 741 • Arm 1: roxadustat • Primary endpoint: haemoglobin • FPCD: Q4 2014 SIERRAS receiving dialysis •
Slide 63: AZD4831 (MPO inhibitor)
Approved medicines Oncology Late-stage development AZD4831 (MPO inhibitor) Early development Cardiovascular disease Trial Population Patients Design Endpoints Status CVRM Phase IIb/ Phase III HFpEF 1485 Randomised, doubl718e blind • Endpoints: Efficacy and Safety • FPCD: Q3 2021 NCT04986202 • Arm 1:
Slide 64: Eplontersen: ATTR
Approved medicines Oncology Late-stage development eplontersen (ligand-conjugated antisense) Early development ATTR Trial Population Patients Design Endpoints (primary) Status CVRM Phase III Patients with hereditary or 750 • Arm 1: eplontersen s.c. • Primary endpoint: composite • FPCD: Q1 2020 CARDI
Slide 65: Breztri, Trixeo: asthma
Approved medicines Oncology Breztri, Trixeo (PT010, LAMA/LABA/ICS, pMDI) 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 and • Primary endpoint: Change from • FP
Slide 66: Daliresp/Daxas: COPD
Approved medicines Oncology Daliresp/Daxas (PDE4 inhibitor, oral) Late-stage development Early development COPD Trial Population Patients Design Endpoints Status CVRM Post Launch COPD 124080 A retrospective cohort trial comparing COPD patients aged 40 • Primary endpoint: all-cause mortality • Data a
Slide 67: Fasenra: severe, uncontrolled asthma
Approved medicines Oncology Fasenra (IL-5R mAb) Late-stage development Early development Severe, uncontrolled asthma Trial Population Patients Design Endpoints Status CVRM Phase III A multicentre, open-label, 447 • Arm 1: Fasenra 30mg Q4W s.c. • Primary endpoint: safety and • FPCD: Q2 2016 MELTEMI s
Slide 68: Fasenra: severe, uncontrolled asthma
Approved medicines Oncology Fasenra (IL-5R mAb) Late-stage development Early development Severe, uncontrolled asthma Trial Population Patients Design Endpoints Status CVRM Phase III Severe asthma, inadequately 2133 Randomised double-blind safety extension trial • Primary endpoint: safety and • FPCD:
Slide 69: Fasenra: asthma, COPD and eosinophilic diseases
Approved medicines Oncology Fasenra (IL-5R mAb) Late-stage development Early development Severe, uncontrolled asthma, COPD and other eosinophilic diseases Trial Population Patients Design Endpoints Status CVRM Phase III Severe asthma on ICS/LABA 121 Open label • Primary endpoint: percentage of • FPC
Slide 70: 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 71: Fasenra: gastrointestinal diseases
Approved medicines Oncology Fasenra (IL-5R mAb) Late-stage development Early development Gastrointestinal diseases Trial Population Patients Design Endpoints Status CVRM Phase III Documented diagnosis of EoE 170 Double blind treatment period and open label period(s) • Primary endpoints: histologic r
Slide 72: Fasenra: dermatology
Approved medicines Oncology Fasenra (IL-5R mAb) Late-stage development Early development Dermatology Trial Population Patients Design Endpoints Status CVRM Phase III Patients with symptomatic 120 Double blind treatment period and open label period • Primary endpoint: Proportion of • FPCD: Q2 2021 FJ
Slide 73: 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 1061 • Arm 1: Tezspire s.c. • Primary endpoint: Annual asthma • FPCD: Q1 2018 NAVIGATOR Age 12-80 years
Slide 74: Tezspire: asthma, COPD and CRSwNP
Approved medicines Oncology Tezspire (TSLP mAb) Late-stage development Early development Severe, uncontrolled asthma, COPD & CRSwNP CVRM Trial Population Patients Design Endpoints Status Phase III Severe chronic rhinosinusitis 400 • Arm 1: Tezspire s.c. • Co-primary endpoint: nasal polyp score • FPC
Slide 75: PT027 (SABA/ICS): asthma
Approved medicines Oncology Late-stage development PT027 (SABA/ICS, pMDI) Early development Asthma Trial Population Patients Design Endpoints Status CVRM Phase III Moderate to severe asthma 3123 Randomised, double-blind, multicentre, parallel group • Primary endpoint: Time to first severe • FPCD: Q4
Slide 76: 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 450 • Arm 1: 300mg i.v. anifrolumab Q4W for 48 weeks • Primary endpoint: SRI4 at wee
Slide 77: Brazikumab: inflammatory bowel disease
Approved medicines Oncology Late-stage development Brazikumab (IL-23 inhibitor) Early development Inflammatory bowel disease (Crohn’s disease, ulcerative colitis) Trial Population Patients Design Endpoints Status Phase IIb / III Crohn’s disease 928 Stage 1 • Stage 1 primary endpoint: percentage of p
Slide 78: Tozorakimab: COPD
Approved medicines Oncology Late-stage development Tozorakimab (IL-33 ligand mAb) Early development COPD Trial Population Patients Design Endpoints Status CVRM Phase II Adult subjects with COPD and 144 Randomised, double-blind, placebo-controlled, parallel group, • Primary endpoint: change from • FP
Slide 79: Tozorakimab: atopic dermatitis, asthma
Approved medicines Oncology Late-stage development Tozorakimab (IL-33 ligand mAb) Early development Atopic dermatitis, asthma Trial Population Patients Design Endpoints Status CVRM Phase II Adult subjects with atopic 152 Randomised, double-blind, placebo-controlled trial • Primary endpoint: change f
Slide 80: Nirsevimab: infection
Approved medicines Oncology Late-stage development Nirsevimab (Respiratory syncytial virus mAb-YTE) Early development Infection Trial Population Patients Design Endpoints Status CVRM Phase III Healthy infants (born 35 3000 (total) Randomised, Double-blind, placebo-controlled • Primary endpoints: eff
Slide 81: AZD1222/AZD2816: prevention of COVID-19
Approved medicines Oncology Late-stage development AZD1222/AZD2816 (SARS-CoV-2) Early development Prevention of COVID-19 Trial Population Patients Design Endpoints Status CVRM Phase II/III Main efficacy trial: healthy 10812 Single-blinded, randomised, controlled, multicentre trial with • Primary end
Slide 82: AZD1222/AZD2816: prevention of COVID-19
Approved medicines Oncology Late-stage development AZD1222/AZD2816 (SARS-CoV-2) Early development Prevention of COVID-19 Trial Population Patients Design Endpoints Status CVRM Phase I/II Healthy adults 256 Double-blinded, randomised, placebo-controlled multicentre • Primary endpoints: safety, tolera
Slide 83: Evusheld: prevention and treatment of COVID-19
Approved medicines Oncology Evusheld (AZD7442, tixagevimab + cilgavimab) Late-stage development Early development Prevention and treatment of COVID-19 CVRM Trial Population Patients/Subjects Design Endpoints Status Phase III Adults having 5197 Double-blinded, randomised, placebo controlled, multi ce
Slide 84: Evusheld: prevention and treatment of COVID-19
Approved medicines Oncology Evusheld (AZD7442, tixagevimab + cilgavimab) Late-stage development Early development Prevention and treatment of COVID-19 CVRM Trial Population Patients/Subjects Design Endpoints Status Phase I Healthy adults 60 Double-blinded, randomised, placebo controlled, single • Pr
Slide 85: BioPharmaceuticals – early-stage development
BioPharmaceuticals – early-stage development
Slide 86: Cotadutide: diabetes/CKD, NASH
Approved medicines Oncology Late-stage development Cotadutide (GLP-1-glucagon agonist) Early development Diabetes/CKD, NASH Trial Population Patients Design Endpoints Status CVRM Phase II Adults with type-2 44 • Part A: cotadutide or placebo s.c. • Primary endpoint: change in hepatic glycogen concen
Slide 87: AZD2373: chronic kidney disease
Approved medicines Oncology Late-stage development 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 rec
Slide 88: AZD2693: NASH
Approved medicines Oncology Late-stage development AZD2693 (antisense oligonucleotide) Early development NASH Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers 62 SAD. 6 cohorts with 6 volunteers receiving AZD2693 and 2 • Primary endpoints: safety and • FPCD: Q4 2019
Slide 89: AZD7503: NASH
Approved medicines Oncology Late-stage development AZD7503 (antisense oligonucleotide) Early development NASH Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers 64 SAD. 6 cohorts with 6 volunteers receiving AZD7503 and 2 • Primary endpoints: safety and • FPCD: Q4 2021
Slide 90: AZD3427 (relaxin): heart failure
Approved medicines Oncology Late-stage development AZD3427 (relaxin) Early development Heart failure CVRM Trial Population Patients Design Endpoints Status Phase I SAD – Healthy volunteers 104 Multicentre single and multiple ascending dose study • Primary endpoints: safety and • FPCD: Q4 2020 NCT046
Slide 91: AZD3366: cardiovascular disease
Approved medicines Oncology Late-stage development AZD3366 Early development Cardiovascular disease Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers 103 SAD trial • Primary endpoints: safety and • FPCD: Q4 2020 NCT04588727 Part A tolerability • Data anticipated: H1 2
Slide 92: AZD5718 (FLAP inhibitor)
Approved medicines Oncology Late-stage development AZD5718 (FLAP inhibitor) Early development Cardiovascular disease & Chronic Kidney Disease Trial Population Patients Design Endpoints Status CVRM Phase IIb Proteinuric CKD 632 Randomised, double-blind, placebo-controlled, multicentre, • Primary endp
Slide 93: AZD8233 (PCSK9 inhibitor): dyslipidaemia
Approved medicines Oncology Late-stage development AZD8233 (PCSK9 inhibitor, subcutaneous) Early development Dyslipidaemia Trial Population Patients Design Endpoints Status CVRM Phase II Dyslipidaemia 376 • Arm 1: AZD8233 dose • Primary endpoint: safety • FPCD: Q3 2021 SOLANO • Arm 2: placebo • LPCD
Slide 94: MEDI8367: chronic kidney disease
Approved medicines Oncology Late-stage development MEDI8367 Early development Chronic kidney disease Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers 70 SAD trial • Primary endpoints: safety and • FPCD: Q3 2020 NCT04365218 CKD 6 cohorts tolerability • LPCD: Q4 2020 •
Slide 95: AZD8601 (VEGF-A modified RNA)
Approved medicines Oncology Late-stage development AZD8601 (VEGF-A modified RNA) Early development Cardiovascular disease Trial Population Patients Design Endpoints Status CVRM Phase IIa HF Up to 33 • Arm 1: AZD8601 Dose A • Primary endpoints: safety and • FPCD: Q1 2018 NCTT03370887 • Arm 2: AZD8601
Slide 96: AZD9977 (MR modulator): heart failure
Approved medicines Oncology Late-stage development AZD9977 (MR modulator) Early development Heart failure Trial Population Patients Design Endpoints Status CVRM Phase IIb Heart failure with chronic 540 Randomised, stratified according to T2DM and eGFR (≥20 to • Primary endpoint: percent change • FPC
Slide 97: AZD9977 (MR modulator): heart failure
Approved medicines Oncology Late-stage development AZD9977 (MR modulator) Early development Heart failure Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers 45 JSMAD trial • Primary endpoints: safety and • FPCD: Q1 2019 NCT03801967 Single and multiple-ascending dose ad
Slide 98: Zibotentan: chronic kidney disease
Approved medicines Oncology Late-stage development Zibotentan (endothelin receptor antagonist) Early development Chronic kidney disease Trial Population Patients Design Endpoints Status CVRM Phase IIb CKD 660 Part A: 132 participants equally randomised across 4 arms: • Primary endpoint: change in lo
Slide 99: Zibotentan: liver cirrhosis
Approved medicines Oncology Late-stage development Zibotentan (endothelin receptor antagonist) Early development Liver Cirrhosis with Features of portal hypertension Trial Population Patients Design Endpoints Status CVRM Phase 1 Cohort 1: Moderate hepatic 12+12 Single dose, single centre, Open label
Slide 100: AZD5462 (relaxin): heart failure
Approved medicines Oncology Late-stage development AZD5462 (relaxin) Early development Heart failure CVRM Trial Population Patients Design Endpoints Status Phase I SAD – Healthy volunteers Single centre single and multiple ascending dose study • Primary endpoints: safety and • FPCD: Q4 2021 NCT04994
Slide 101: MEDI6570: cardiovascular
Approved medicines Oncology Late-stage development MEDI6570 Early development Cardiovascular Trial Population Patients Design Endpoints Status CVRM Phase IIb Post MI 400 Evaluation of anti-inflammatory potential • Primary endpoints: efficacy and safety • FPCD: Q4 2020 NCT04610892 and effect on surro
Slide 102: Tozorakimab: diabetic kidney disease
Approved medicines Oncology Late-stage development Tozorakimab (IL-33 ligand mAb) Early development Diabetic kidney disease Trial Population Patients Design Endpoints Status CVRM Phase II Adult patients with diabetic 565 • Arm A: tozorakimab dose 1 + Farxiga • Primary endpoints: safety and efficacy
Slide 103: AZD4604 (inhaled JAK-1 inhibitor)
Approved medicines Oncology Late-stage development AZD4604 (inhaled JAK-1 inhibitor) Early development Asthma Trial Population Patients Design Endpoints Status CVRM Phase I Healthy subjects and patients 137 SAD/MAD/POM trial • Primary endpoints: safety and • FPCD: Q4 2021 NCT04769869 with mild asthm
Slide 104: AZD1402 (IL-4 receptor alpha antagonist)
Approved medicines Oncology Late-stage development AZD1402 (IL-4 receptor alpha antagonist) Early development Asthma Trial Population Patients Design Endpoints Status CVRM Phase IIa Patients with asthma on 345 Randomised, placebo-controlled, double-blinded, multicentre, • Part 1 primary endpoints: s
Slide 105: AZD5055 (oral porcupine inhibitor)
Approved medicines Oncology Late-stage development AZD5055 (oral porcupine inhibitor) Early development Idiopathic pulmonary fibrosis (IPF) and other ILDs with progressive fibrosis CVRM Trial Population Patients Design Endpoints Status Phase I Healthy subjects 104 SAD/MAD trial • Primary endpoints:
Slide 106: AZD8630 (inhaled TSLP)
Approved medicines Oncology Late-stage development AZD8630 (inhaled TSLP) Early development Asthma Trial Population Patients Design Endpoints Status CVRM Phase I Healthy subjects and patients 232 SAD & MAD trial • Primary endpoints: safety and • FPCD: Q4 2021 NCT05110976 with asthma tolerability • D
Slide 107: MEDI0618 (PAR2 antagonist mAb)
Approved medicines Oncology Late-stage development MEDI0618 (PAR2 antagonist mAb) Early development Osteoarthritis pain Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers 64 SAD trial • Primary endpoints: safety and • FPCD: Q4 2019 NCT04198558 • Arm 1: MEDI0618 i.v. to
Slide 108: MEDI1341 (alpha-synuclein mAb)
Approved medicines Oncology Late-stage development MEDI1341 (alpha-synuclein mAb) Early development Parkinson’s disease Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers 48 SAD trial • Primary endpoints: safety and • FPCD: Q4 2017 NCT03272165 • Arm 1: MEDI1341 i.v. to
Slide 109: AZD4041 (orexin receptor antagonist)
Approved medicines Oncology Late-stage development AZD4041 (orexin 1 receptor antagonist) Early development Opioid use disorder Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers 48 Randomised, double blind, SAD trial • Primary endpoints: safety and • FPCD: Q4 2019 NCT
Slide 110: MEDI7352 (NGF TNF bispecific mAb)
Approved medicines Oncology Late-stage development MEDI7352 (NGF TNF bispecific mAb) Early development Osteoarthritis pain Trial Population Patients Design Endpoints Status CVRM Phase IIb Painful osteoarthritis of the knee 300 MAD trial • Primary endpoint: dose response • FPCD: Q1 2021 NCT04675034 •
Slide 111: MEDI3902 (Anti-Pseudomonas A mAb)
Approved medicines Oncology Late-stage development MEDI3902 (Anti-Pseudomonas A mAb) Early development Infections Trial Population Patients Design Endpoints Status CVRM Phase II Intubated ICU 195 SAD trial • Primary endpoints: safety and efficacy • FPCD: Q2 2016 NCT02696902 • Arm 1: MEDI3902 i.v. •
Slide 112: Rare Disease – approved medicines and late-stage pipeline
Rare Disease – approved medicines and late-stage pipeline
Slide 113: Soliris: neurology
Approved medicines Oncology Soliris (anti-complement C5 mAb) Late-stage development Early development Neurology Trial Population Patients Design Endpoints Status CVRM Phase III Guillain Barré syndrome 57 • Arm 1: Soliris i.v. once weekly for 4 weeks • Primary endpoint: Time to first reaching • FPCD:
Slide 114: Ultomiris: haematology & nephrology
Approved medicines Oncology Ultomiris (anti-complement C5 mAb) Late-stage development Early development Haematology & nephrology Trial Population Patients Design Endpoints Status CVRM Phase III PNH and aHUS 136 • Ultomiris s.c. • Primary endpoint: Day 71 serum • FPCD: Q1 2019 ALXN-1210-PNH-303 Ultom
Slide 115: Ultomiris: neurology
Approved medicines Oncology Ultomiris (anti-complement C5 mAb) Late-stage development Early development Neurology Trial Population Patients Design Endpoints Status CVRM Phase II/III Amyotrophic lateral sclerosis 382 • Arm 1: Ultomiris • Primary endpoint: change from baseline • Trial halted post futi
Slide 116: ALXN1840: Wilson disease
Approved medicines Oncology Late-stage development ALXN1840 (bis-choline tetrathiomolybdate) Early development Wilson disease Trial Population Patients Design Endpoints Status CVRM Phase III Wilson Disease 215 • Arm 1: ALXN1840 was administered orally for 48 weeks at • Primary endpoint: daily mean A
Slide 117: Andexxa: haematology
Approved medicines Oncology Andexxa (anti-factor Xa reversal) Late-stage development Early development Haematology Trial Population Patients Design Endpoints Status CVRM Post Launch Acute Intracranial 1200 • Arm 1: Andexxa • Primary endpoint: proportion of • FPCD: Q2 2019 Phase IV Haemorrhage • Arm
Slide 118: CAEL-101: AL amyloidosis
Approved medicines Oncology Late-stage development CAEL-101 (fibril-reactive mAb) Early development AL amyloidosis Trial Population Patients Design Endpoints Status CVRM Phase III Mayo stage IIIa 267 • Arm 1: CAEL-101 combined with SoC for plasma cell dyscrasia • Primary: time from first dose of tri
Slide 119: acoramidis (ALXN2060): ATTR-CM
Approved medicines Oncology Late-stage development acoramidis (ALXN2060) Early development ATTR-CM Trial Population Patients Design Endpoints Status CVRM Phase III ATTR-CM 22 • Arm 1: 800 mg acoramidis (ALXN2060) administered twice • Primary endpoint: Change from • FPCD: Q4 2020 ALXN2060-TAC-302 dai
Slide 120: danicopan (ALXN2040): haematology & ophthalmology
Approved medicines Oncology Late-stage development danicopan (ALXN2040 Factor D inhibitor) Early development Haematology & ophthalmology Trial Population Patients Design Endpoints Status CVRM Phase III PNH with clinically relevant 84 • Arm 1: danicopan (ALXN2040) + C5 Inhibitor • Primary endpoint: c
Slide 121: Rare Disease – early-stage development
Rare Disease – early-stage development
Slide 122: ALXN1720 (anti-C5 bi-specific minibody)
Approved medicines Oncology Late-stage development ALXN1720 (anti-C5 bi-specific minibody) Early development Neurology Trial Population Patients Design Endpoints Status CVRM Phase I Healthy Volunteers 96 • Single & Multiple Ascending Dosing of ALXN1720 via s.c. and i.v. • Primary: Incidence of TEAEs
Slide 123: ALXN2050 (factor D inhibitor)
Approved medicines Oncology Late-stage development ALXN2050 (factor D inhibitor) Early development Haematology, nephrology, neurology Trial Population Patients Design Endpoints Status CVRM Phase II PNH 26 • Arm 1: ALXN2050 Monotherapy, with groups including • Primary endpoint: Change in • FPCD: Q4 2
Slide 124: ALXN1820 (anti-properdin)
Approved medicines Oncology Late-stage development ALXN1820 (anti-properdin) Early development Haematology Trial Population Patients Design Endpoints Status CVRM Phase I Healthy Volunteers 80 • Arm 1: ALXN1820 administered s.c. or i.v., multiple ascending • Primary: Participants with TEAEs • FPCD: Q
Slide 125: ALXN1850 (next generation asfotase alfa)
Approved medicines Oncology Late-stage development ALXN1850 (next generation asfotase alfa) Early development Hypophosphatasia Trial Population Patients Design Endpoints Status CVRM Phase 1 HPP 15 • Arm 1: ALXN1850, 3 cohorts at low, medium, and high • Primary: Incidence of TEAEs and TESAEs • FPCD:
Slide 126: List of abbreviations
List of abbreviations 14C Radioactive isotope of carbon, Carbon 14 BTK Bruton's tyrosine kinase DRFI Disease recurrence-free interval 1L, 2L, 3L 1st, 2nd or 3rd line CA-125 Cancer antigen 125 DXA Dual energy X-ray absorptiometry 5-FU 5-fluorouracil CAD Coronary artery disease EBRT External beam radi
Slide 127: List of abbreviations
List of abbreviations HFrEF Heart failure with reduced ejection fraction mCRPC Metastatic castrate-resistant prostate cancer PFS Progression free survival HGFR Met/hepatocyte growth factor receptor MD Medium dose PgR Progesterone receptor HGSC High grade serous carcinoma MDI Metered-dose inhaler PI3
Slide 128: List of abbreviations
List of abbreviations s.c. Subcutaneous SCCHN Squamous-cell carcinoma of the head and neck SCLC Small cell lung cancer SD Stable disease SERD Selective oestrogen receptor degrader SGLT2 Sodium-glucose transport protein 2 SGRM Selective glucocorticoid receptor modulator SGRQ Saint George respiratory
Slide 129: Clinical trials appendix Q1 2021 results update
Clinical trials appendix Q1 2021 results update