Slide 1: Clinical trials appendix Q2 2021 results update
Clinical trials appendix Q2 2021 results update
Slide 2: Movement since Q1 2021 update
Movement since Q1 2021 update New to Phase I New to Phase II New to Pivotal trial New to registration NME NME NME AZD1402# AZD2816¶# (next generation COVID-19 vaccine) tezepelumab# NAVIGATOR [US, EU & JP] 1 Inhaled IL4Ra asthma SARS-CoV-2 prevention of COVID-19 TSLP mAb severe, uncontrolled asthma A
Slide 3: Q2 2021 new molecular entity (NME) pipeline
Q2 2021 new molecular entity (NME)1 pipeline Phase I Phase II Phase III 16 New Molecular Entities 18 New Molecular Entities 13 New Molecular Entities adavosertib# AZD0466# Imfinzi#+RT (platform) CLOVER Imfinzi#+imaradenant#+cabazitaxel camizestrant+palbociclib SERENA-4 Imfinzi#+tremelimumab+SoC NILE
Slide 4: Q2 2021 new molecular entity (NME) pipeline
Q2 2021 new molecular entity (NME)1 pipeline Phase I Phase II Phase III Under Review 11 New Molecular Entities 22 New Molecular Entities 6 New Molecular Entities 2 New Molecular Entities cotadutide AZD2816¶# next generation COVID-19 AZD0284 anifrolumab# anifrolumab# TULIP GLP-1/glucagon T2D / obesit
Slide 5: Q2 2021 lifecycle management (LCM) pipeline
Q2 2021 lifecycle management (LCM)1 pipeline Phase I Phase II Phase III Under Review 1 Projects 9 Projects 31 Projects 0 Projects Imfinzi# CALLA Enhertu# (platform) DESTINY-Breast08 Enhertu# (platform) DESTINY-Breast07 Calquence# Imfinzi#+CTx TOPAZ-1 PD-L1 adj. locally-advanced cervical ADC breast A
Slide 6: Q2 2021 lifecycle management (LCM) pipeline
Q2 2021 lifecycle management (LCM)1 pipeline Phase I Phase II Phase III Under Review 0 Projects 3 Projects 10 Projects 1 Project Fasenra ARROYO Breztri Fasenra NATRON Fasenra# OSTRO IL-5R chronic spontaneous urticaria LABA/LAMA/ICS asthma IL-5R hypereosinophilic syndrome IL-5R nasal polyps Farxiga/F
Slide 7: Estimated key regulatory submission acceptances
Estimated key regulatory submission acceptances camizestrant + palbociclib breast cancer SERENA-4 capivasertb + abiraterone PTEN deficient Imfinzi + tremelimumab + CRT mHSPC CAPItello-281 LDS-SCLC ADRIATIC nirsevimab capivasertb + CTx 1L mTNBC Imfinzi + tremelimumab + SoC urothelial passive RSV immu
Slide 8: Designations
Designations 5 18 12 36 29 Accelerated approvals Breakthrough / PRIME1 / Sakigake2 Fast Track Priority Review Orphan ACCELERATED APPROVAL, these regulations allowed medicines for serious conditions that addressed an unmet medical need to be approved based on a surrogate endpoint. BREAKTHROUGH DESIGN
Slide 9: Oncology – approved medicines and late-stage pipeline
Oncology – approved medicines and late-stage pipeline
Slide 10: Tagrisso
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 11: Tagrisso 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 12: 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 13: Imfinzi: lung cancer
Approved medicines Oncology Imfinzi (PD-L1 mAb) 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 14: 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 15: 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 16: Imfinzi: other cancers, early disease
Approved medicines Oncology Imfinzi (PD-L1 mAb) Late-stage development Early development Other cancers, early disease 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 POTOM
Slide 17: 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 1215 • Arm 1: Imfinzi + tremelimumab + SoC • Primary endpoint: OS • FPCD:
Slide 18: 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 Advanced solid malignancies 1200 • Arm 1: Imfinzi • Primary endpoint: safety • FPCD: Q2 201
Slide 19: 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 20: 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 21: 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 22: 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 23: 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 350 Randomised open label sequential assignment • Primary endpoints:
Slide 24: 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 25: 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-over-expressing or mutated, 170 Non randomised parallel group assignment • Primary endpoint: ORR • FPCD:
Slide 26: 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 27: 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 28: 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 29: 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 III CLL TN and R/R 600 • Arm A: treatment naïve • Primary endpoint: safety • Data anticipated: 2022+ CL-312 (ASSURE) •
Slide 30: Koselugo
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 31: Orpathys
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 32: Capivasertib
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 800 Double-blind randomised comparative trial • Primary endpoint: OS • FPCD: Q3 2019 C
Slide 33: Monalizumab
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 SCCHN, 2L • Arm B: placebo
Slide 34: Camizestrant
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 35: Datopotamab deruxtecan
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 36: Datopotamab deruxtecan
Approved medicines Oncology Late-stage development Datopotamab deruxtecan (TROP2 ADC) Early development NSCLC and other cancers Trial Population Patients Design Endpoints Status CVRM Phase I Subjects with advanced solid 770 Open label, two-part (dose escalation, dose expansion), • Primary endpoint:
Slide 37: Oncology – early-stage development
Oncology – early-stage development
Slide 38: MEDI0457 + Imfinzi
Approved medicines Oncology Late-stage development MEDI0457 (DNA HPV Vaccine) Early development + Imfinzi (PD-L1 mAb) Head and neck squamous cell carcinoma (HNSCC) CVRM Trial Population Patients Design Endpoints Status Phase Ib/IIa HPV associated 50 Multicentre, open label • Primary endpoints: safet
Slide 39: AZD0466
Approved medicines Oncology Late-stage development AZD0466 (Bcl2/xL inhibitor) Early development Cancer Trial Population Patients Design Endpoints Status CVRM Phase I Advanced haematologic 9 Monotherapy dose escalation, consisting of two arms: • Primary endpoint: safety • FPCD: Q4 2019 NCT04214093 m
Slide 40: MEDI1191
Approved medicines Oncology Late-stage development MEDI1191 (IL12 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 NCT0
Slide 41: AZD1390
Approved medicines Oncology Late-stage development AZD1390 (ATM inhibitor) Early development Cancer Trial Population Subjects Design Endpoints Status CVRM Phase I Recurrent glioblastoma 186 Open label trial • Primary endpoints: safety, • FPCD: Q2 2018 NCT03423628 eligible for re-irradiation, • Arm A
Slide 42: Adavosertib
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 43: AZD2811
Approved medicines Oncology Late-stage development AZD2811 (nanoparticle, Aurora B kinase inhibitor) Early development Cancer Trial Population Patients Design Endpoints Status CVRM Phase II Extensive-stage small cell 100 Experimental: • Primary endpoints: APF12 per RECIST • FPCD: Q1 2021 NCT04745689
Slide 44: AZD4573
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: Imaradenant
Approved medicines Oncology Late-stage development Imaradenant (AZD4635, A2AR inhibitor) Early development Cancer Trial Population Patients Design Endpoints Status CVRM Phase II Prostate cancer 60 • Arm 1: imaradenant + Imfinzi • Primary outcome: efficacy (ORR and • FPCD: Q3 2019 NCT04089553 • Arm 2
Slide 46: AZD5305
Approved medicines Oncology Late-stage development AZD5305 (selective PARP1 inhibitor) Early development Solid tumours Trial Population Patients Design Endpoints Status CVRM Phase I/IIa Advanced, metastatic HER2- 612 A modular, open-label, multicentre trial dose escalation • Primary endpoints: safet
Slide 47: MEDI5395
Approved medicines Oncology Late-stage development MEDI5395 (rNDV GMCSF) Early development Cancer Trial Population Patients Design Endpoints Status CVRM Phase I Select advanced solid 188 Open-label dose escalation and dose expansion trial • Primary endpoints: safety and • FPCD: Q4 2019 NCT03889275 t
Slide 48: MEDI5752
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 261 Open-label, dose-escalation and dose-expansion trial • Dose escalation primary endpoints: •
Slide 49: AZD5991
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 50: Ceralasertib
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 51: AZD7648
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 52: AZD8701
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 53: MEDI9253
Approved medicines Oncology Late-stage development MEDI9253 (rNDV-IL12) 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 54: Oleclumab
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 55: IPH5201
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 56: BioPharmaceuticals – approved medicines and late-stage pipeline
BioPharmaceuticals – approved medicines and late-stage pipeline
Slide 57: Farxiga
Approved medicines Oncology Farxiga (SGLT2 inhibitor) Late-stage development Early development Heart failure and chronic kidney disease Trial Population Patients Design Design Endpoints Endpoints Status Status CVRM Phase III Phase III Patients Patients With with CKD CKD 4,304 4304 •• Arm Arm1: 1: Fa
Slide 58: Brilinta
Approved medicines Oncology Brilinta (P2Y12 receptor antagonist) Late-stage development Early development Cardiovascular risk reduction Trial Population Patients Design Endpoints (primary) Status CVRM Phase III Patients with acute ischaemic 11000 • Arm 1: Brilinta 90mg BID • Primary endpoint: preven
Slide 59: Lokelma
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 60: Roxadustat
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 61: Roxadustat
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 62: Eklira/Tudorza
Approved medicines Oncology Eklira/Tudorza (LAMA, DPI) Late-stage development Early development COPD Trial Population Number of patients Design Endpoints Status CVRM Phase I Healthy Chinese 20 Open-label trial in healthy Chinese male and female participants. • Primary endpoints: safety and • Initiat
Slide 63: Duaklir Genuair
Approved medicines Oncology Duaklir Genuair (LAMA/LABA, DPI) Late-stage development Early development COPD Trial Population Patients Design Endpoints Status CVRM Phase III Patients with stable COPD 1060 • Arm 1: Duaklir Genuair 400/12 μg DPI • Primary endpoints: • FPCD: Q1 2017 AVANT • Arm 2: aclidi
Slide 64: Breztri, Trixeo
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 2800 Randomised, double-blind, double dummy, parallel group and • Primary endpoint: Change from • FP
Slide 65: Daliresp/Daxas
Approved medicines Oncology Daliresp/Daxas (PDE4 inhibitor, oral) Late-stage development Early development COPD Trial Population Patients Design Endpoints Status CVRM Post Launch COPD 124,080 A retrospective cohort trial comparing COPD patients aged 40 • Primary endpoint: all-cause mortality • Data
Slide 66: Fasenra: severe, uncontrolled asthma
Approved medicines Oncology Fasenra (IL5R 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 sa
Slide 67: Fasenra: severe, uncontrolled asthma
Approved medicines Oncology Fasenra (IL5R 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 68: Fasenra: asthma, COPD and eosinophilic diseases
Approved medicines Oncology Fasenra (IL5R 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 • FPCD
Slide 69: Fasenra: nasal polyposis
Approved medicines Oncology Fasenra (IL5R 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 Fas
Slide 70: Fasenra: gastrointestinal diseases
Approved medicines Oncology Fasenra (IL5R mAb) Late-stage development Early development Gastrointestinal diseases Trial Population Patients Design Endpoints Status Documented diagnosis of EoE 170 Double blind treatment period and open label period(s) • Primary endpoints: Histologic • FPCD: Q4 2020 C
Slide 71: Fasenra: dermatology
Approved medicines Oncology Fasenra (IL5R 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 FJO
Slide 72: Tezepelumab: severe asthma
Approved medicines Oncology Late-stage development Tezepelumab (TSLP mAb) Early development Severe, uncontrolled asthma Trial Population Patients Design Endpoints Status CVRM Phase III Severe asthma 1,061 • Arm 1: tezepelumab s.c. • Primary endpoint: Annual asthma • FPCD: Q1 2018 NAVIGATOR Age 12-80
Slide 73: Tezepelumab: asthma, COPD and CRSwNP
Approved medicines Oncology Late-stage development Tezepelumab (TSLP mAb) Early development Severe, uncontrolled asthma, COPD & CRSwNP CVRM Trial Population Patients Design Endpoints Status Phase III Severe chronic rhinosinusitis 400 • Arm 1: tezepelumab s.c. • Co-primary endpoint: Nasal Polyp • FPC
Slide 74: PT027
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 3100 Randomised, double-blind, multicentre, parallel group • Primary endpoint: Time to first severe • FPCD: Q4
Slide 75: Anifrolumab: lupus
Approved medicines Oncology Late-stage development Anifrolumab (type I interferon receptor mAb) 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: response
Slide 76: Anifrolumab: lupus
Approved medicines Oncology Late-stage development Anifrolumab (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: 120 mg SC QW, 52 wk in aPFS • Primary endpoint: reduction in overall
Slide 77: Brazikumab
Approved medicines Oncology Late-stage development Brazikumab (IL23 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 endpoints: percentage of p
Slide 78: Nirsevimab
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: ef
Slide 79: AZD1222/AZD2816 (SARS-CoV-2)
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 80: AZD1222/AZD2816 (SARS-CoV-2)
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 AZD2816 Healthy adults 2590 Partially double-blinded, randomized, active-controlled trial in • Primary endpoint
Slide 81: AZD7442
Approved medicines Oncology Late-stage development AZD7442 (LAAB combination of AZD8895 & AZD1061) Early development Prevention and treatment of COVID-19 Trial Population Patients/Subjects Design Endpoints Status CVRM Phase III Adults having 5,000 Double-blinded, randomised, placebo controlled, mult
Slide 82: Other COVID-19 trials
Approved medicines Oncology Late-stage development Other COVID-19 trials Early development Treatment of COVID-19 Trial Population Patients/Subjects Design Endpoints Status CVRM Phase III COVID-19 1250 • Current SoC or current SoC + Farxiga • Primary outcome measures: time to • FPCD: Q2 2020 DARE-19
Slide 83: BioPharmaceuticals – early-stage development
BioPharmaceuticals – early-stage development
Slide 84: Cotadutide
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 85: Verinurad
Approved medicines Oncology Late-stage development Verinurad (URAT1 inhibitor) Early development CKD, HFpEF Trial Population Patients Design Endpoints Status CVRM Phase II Patients with: 861 • Arm A: verinurad 12 mg + allopurinol 300 mg • Primary endpoints: ratio of urinary • FPCD: Q3 2019 SAPPHIRE
Slide 86: Verinurad
Approved medicines Oncology Late-stage development Verinurad (URAT1 inhibitor) Early development CKD, HFpEF Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers 14 Single-centre, randomised, open-label, 3-period, fixed • Primary endpoint: PK • FPCD: Q3 2020 NCT04532918 s
Slide 87: AZD2373
Approved medicines Oncology Late-stage development AZD2373 Early development Chronic kidney disease Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers 48 SAD. Dose escalation in 6 cohorts with 6 volunteers receiving • Primary endpoints: safety and • FPCD: Q1 2020 NCT04
Slide 88: AZD2693
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: AZD3427
Approved medicines Oncology Late-stage development AZD3427 (relaxin) Early development Heart failure Trial Population Patients Design Endpoints Status CVRM Phase I SAD – Healthy volunteers 104 Multicentre single and multiple ascending dose study • Primary endpoints: safety and • FPCD: Q4 2020 NCT046
Slide 90: AZD3366
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 7 cohorts with 6 volunte
Slide 91: MEDI3506: diabetic kidney disease
Approved medicines Oncology Late-stage development MEDI3506 (IL33 ligand mAb) Early development Diabetic kidney disease Trial Population Patients Design Endpoints Status CVRM Phase II Adult patients with diabetic 565 • Arm A: MEDI3506 dose 1 + Farxiga • Primary endpoints: safety and efficacy • FPCD:
Slide 92: AZD4831
Approved medicines Oncology Late-stage development AZD4831 (MPO inhibitor) Early development Cardiovascular disease Trial Population Patients Design Endpoints Status CVRM Phase IIa HFpEF 96 • Arm 1: AZD4831 • Primary endpoint: The change from • FPCD: Q4 2018 NCT03756285 • Arm 2: placebo baseline in
Slide 93: AZD5718
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 94: AZD8233
Approved medicines Oncology Late-stage development AZD8233 (PCSK9 inhibitor, subcutaneous) Early development Dyslipidaemia Trial Population Patients Design Endpoints Status CVRM Phase II Dyslipidaemia 108 • Arm 1: High AZD8233 dose • Primary endpoint: efficacy • FPCD: Q4 2020 NCT04641299 • Arm 2: Me
Slide 95: MEDI8367
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 • Arm 1: MEDI8367
Slide 96: AZD8601
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: AZD 860
Slide 97: AZD9977
Approved medicines Oncology Late-stage development AZD9977 (MCR 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 • FP
Slide 98: AZD9977
Approved medicines Oncology Late-stage development AZD9977 (MCR 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 a
Slide 99: Zibotentan
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 100: MEDI5884
Approved medicines Oncology Late-stage development MEDI5884 (cholesterol modulation) Early development Cardiovascular disease Trial Population Patients Design Endpoints Status CVRM Phase IIa Adults with stable 133 5 dose cohorts • Primary endpoints: safety profile in terms of AEs, vital signs, • FPC
Slide 101: MEDI6570
Approved medicines Oncology Late-stage development MEDI6570 Early development Cardiovascular & metabolic diseases Trial Population Patients Design Endpoints Status CVRM Phase IIb Post MI 792 Evaluation of anti-inflammatory potential • Primary endpoints: efficacy and safety • FPCD: Q4 2020 NCT0461089
Slide 102: AZD0449
Approved medicines Oncology Late-stage development AZD0449 (inhaled JAK-1 inhibitor) Early development Asthma Trial Population Patients Design Endpoints Status CVRM Phase I Healthy subjects and patients 130 SAD/MAD/Bridge trial • Primary endpoints: safety and • FPCD: Q4 2018 NCT03766399 with mild as
Slide 103: AZD1402
Approved medicines Oncology Late-stage development AZD1402 (IL4 receptor alpha antagonist) Early development Asthma Trial Population Patients Design Endpoints Status CVRM Phase IIa Patients with asthma on 405 Randomised, placebo-controlled, double-blinded, multicentre, • Part 1 primary endpoints: sa
Slide 104: MEDI3506: COPD, atopic dermatitis, asthma
Approved medicines Oncology Late-stage development MEDI3506 (IL33 ligand mAb) Early development COPD, atopic dermatitis, asthma Trial Population Patients Design Endpoints Status CVRM Phase II Adult subjects with atopic 152 Randomised, blinded, placebo-controlled trial • Primary endpoint: change from
Slide 105: AZD9567
Approved medicines Oncology Late-stage development AZD9567 (SGRM, oral) Early development Immunology Trial Population Patients Design Endpoints Status CVRM Phase II Patients with type 2 diabetes 42 • Arm 1: 72mg AZD9567 + 40mg prednisolone • Primary endpoint: change in glucose • FPCD: Q4 2020 NCT045
Slide 106: MEDI0618
Approved medicines Oncology Late-stage development MEDI0618 (PAR2 antagonist mAb) Early development Osteoarthritis pain Trial Population Patients Design Endpoints Status CVRM Phase I Painful osteoarthritis of the knee 64 (healthy SAD trial • Primary endpoints: safety and • FPCD: Q4 2019 NCT02508155
Slide 107: MEDI1341
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 108: AZD4041
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 109: MEDI7352
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 110: MEDI3902
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 111: List of abbreviations
List of abbreviations 14C Radioactive isotope of carbon, Carbon 14 CHF Chronic heart failure FGFR Fibroblast growth factor receptor 1L, 2L, 3L 1st, 2nd or 3rd line CKD Chronic kidney disease FLAP 5-lipoxygenase-activating protein 5-FU 5-fluorouracil CLL Chronic lymphocytic leukaemia FPDC First patie
Slide 112: List of abbreviations
List of abbreviations LABA Long -acting beta agonist PARP Poly ADP ribose polymerase SAD Single ascending dose LAMA Long-acting muscarinic agonist PASI Psoriasis area severity index SAE Serious adverse event LCAT Lecithin-cholesterol acyltransferase PBD Pyrrolobenzodiazepine SBRT Stereotactic body r
Slide 113: Clinical trials appendix Q1 2021 results update
Clinical trials appendix Q1 2021 results update