Slide 1: Clinical trials appendix H1 2022 results update
Clinical trials appendix H1 2022 results update
Slide 2: Movement since Q1 2022 update
Movement since Q1 2022 update New to Phase I New to Phase II New to Pivotal trial New to registration NME Additional indication Additional indication NME ALXN1910 ALXN2050 capivasertib + docetaxel CAPItello-280# PT027#1 Next Gen TNSALP ERT Bone Oral Factor D Inhibitor generalized myasthenia AKT inhi
Slide 3: Q2 2022 Oncology new molecular entity pipeline
Q2 2022 Oncology new molecular entity1 pipeline Phase 1 Phase 2 Phase 3 13 New Molecular Entities 15 New Molecular Entities 19 New Molecular Entities AZD0466# AZD0171 + Imfinzi + CTx AZD4573 camizestrant + CDK4/6i SERENA-6 AZD1390 camizestrant + palbociclib SERENA-4 BCL2/xL haematological anti-LIF+P
Slide 4: Q2 2022 Oncology life-cycle management pipeline
Q2 2022 Oncology life-cycle management1 pipeline Phase 1 Phase 2 Phase 3 Under Review 1 Project 7 Projects 30 Projects 4 Projects Calquence + venetoclax + obinutuzumab Enhertu (platform) DESTINY-Breast08# Enhertu (platform) DESTINY-Breast07# Calquence + R-CHOP ESCALADE Calquence ECHO# Enhertu DESTIN
Slide 5: Q2 2022 BioPharmaceuticals new molecular entity pipeline
Q2 2022 BioPharmaceuticals new molecular entity1 pipeline Phase 1 Phase 2 Phase 3 Under Review 15 New Molecular Entities 17 New Molecular Entities 4 New Molecular Entities 2 New Molecular Entities AZD0780 AZD2373 AZD1402# AZD4831 brazikumab INTREPID nirsevimab# PCSK9 dyslipidemia Podocyte health nep
Slide 6: Q2 2022 BioPharmaceuticals life-cycle management pipeline
Q2 2022 BioPharmaceuticals life-cycle management1 pipeline Phase 1 Phase 2 Phase 3 Under Review 0 Projects 4 Projects 17 Projects 0 Projects Farxiga/Forxiga DAPA-MI Andexxa (ALXN2070) Breztri/Trixeo (PT010) KALOS LOGOS SGLT2 prevention of HF and CV death anti-factor Xa reversal urgent surgery LABA/L
Slide 7: Q2 2022 Rare Disease pipeline
Q2 2022 Rare Disease pipeline1 Phase 1 Phase 2 Phase 3 Under Review 5 Projects 4 Projects 7 Projects 1 Project ALXN1720 ALXN2050 Ultomiris (ALXN1210) acoramidis (ALXN2060)# anti-C5 bi-specific minibody generalised myasthenia oral factor D inhibitor paroxysmal nocturnal anti-complement C5 mAb subcuta
Slide 8: Estimated key regulatory submission acceptances
Estimated key regulatory submission acceptances capivasertib + fulvestrant + palbociclib datopotamab deruxtecan CAPItello-292 HR+ breast 1L TROPION Lung08 1L metastatic NSCLC capivasertib + abiraterone CAPItello-281 Imfinzi +- oleclumab +- mona PACIFIC-9 PTEN deficient mHSPC unresectable stage III N
Slide 9: Designations in our pipeline
Designations in our pipeline 3 17 9 16 30 Accelerated approvals Breakthrough / PRIME1 / Sakigake2 Fast Track Priority Review Orphan Andexxa Acute Major Bleed (US) Calquence CLL ELEVATE-TN, ASCEND (US) CAEL-101 AL amyloidosis (US) Calquence MCL 1L (US) ALXN1840 WD (US) Calquence MCL 1L (US) Calquence
Slide 10: Oncology – approved medicines and late-stage pipeline
Oncology – approved medicines and late-stage pipeline
Slide 11: Oncology: Tagrisso NSCLC
Approved medicines Late-stage development Oncology Tagrisso (highly-selective, irreversible EGFRi) 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 12: Oncology: Tagrisso NSCLC, combinations
Approved medicines Late-stage development Oncology Tagrisso (highly-selective, irreversible EGFRi) 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 13: Oncology: Tagrisso NSCLC, combinations
Approved medicines Late-stage development Oncology Tagrisso (highly-selective, irreversible EGFRi) Early development NSCLC, combinations Trial Population Patients Design Endpoints Status CVRM Phase III Locally advanced or metastatic NSCLC 250 • Arm 1: Tagrisso + Orpathys • Primary endppoint: PFS • F
Slide 14: Oncology: Imfinzi gastrointestinal cancer
Approved medicines Late-stage development Oncology Imfinzi (PD-L1 mAb) Early development Gastrointestinal cancer Trial Population Patients Design Endpoints Status CVRM Phase III Locoregional HCC 710 • Arm 1: TACE in combination with Imfinzi • Primary endpoint: PFS for Arm 2 vs • FPCD: Q1 2019 EMERAL
Slide 15: Oncology: Imfinzi lung cancer
Approved medicines Late-stage development Oncology Imfinzi (PD-L1 mAb) Early development Lung cancer Trial Population Patients Design Endpoints Status CVRM Phase III Completely resected 86 • Arm 1: Imfinzi + SoC chemo • Primary endpoint: DFS • FPCD: Q3 2020 MERMAID-1 Stage II and III NSCLC • Arm 2:
Slide 16: Oncology: Imfinzi lung cancer
Approved medicines Late-stage development Oncology Imfinzi (PD-L1 mAb) Early development Lung cancer Trial Population Patients Design Endpoints Status CVRM Phase III Unresected, locally-advanced NSCLC 860 • Arm 1: Imfinzi + domvanalimab following chemo/RT • Primary endpoint: PFS • FPCD: Q1 2022 PACI
Slide 17: Oncology: Imfinzi lung cancer
Approved medicines Late-stage development Oncology Imfinzi (PD-L1 mAb) Early development Lung cancer Trial Population Patients Design Endpoints Status CVRM Phase II Stage III NSCLC unresectable 189 • Arm A: Imfinzi • Primary endpoint: OR per RECIST v1.1 • FPCD: Q4 2018 COAST • Arm B: Imfinzi + olecl
Slide 18: Oncology: Imfinzi other cancer
Approved medicines Late-stage development Oncology Imfinzi (PD-L1 mAb) Early development Other cancer Trial Population Patients Design Endpoints Status CVRM Phase III Non-muscle invasive bladder cancer 1018 • Arm 1: BCG (Induction + maintenance) • Primary endpoint: DFS • FPCD: Q2 2018 POTOMAC • Arm
Slide 19: Oncology: Lynparza Imfinzi combinations
Approved medicines Late-stage development Oncology Lynparza (PARP inhibitor) Early development Imfinzi combinations Trial Population Patients Design Endpoints Status CVRM Phase III Advanced ovarian cancer 1L 1256 • Non tBRCAm (tumour BRCA) patients • Primary endpoint: PFS • FPCD: Q1 2019 DUO-O • Arm
Slide 20: Oncology: Lynparza multiple cancers
Approved medicines Late-stage development Oncology Lynparza (PARP inhibitor) Early development Multiple cancers Trial Population Patients Design Endpoints Status CVRM Phase III BRCAm adjuvant breast cancer 1836 • Arm 1: Lynparza BID 12-month duration • Primary endpoint: invasive disease- • FPCD: Q2
Slide 21: Oncology: Lynparza other combinations
Approved medicines Late-stage development Oncology Lynparza (PARP inhibitor) Early development Other combinations Trial Population Patients Design Endpoints Status CVRM Phase III Metastatic castration-resistant prostate 904 • Arm 1: Lynparza + abiraterone • Primary endpoint: rPFS • FPCD: Q4 2018 PRO
Slide 22: Oncology: Enhertu breast cancer
Approved medicines Late-stage development Oncology Enhertu (trastuzumab deruxtecan, HER2 ADC) Early development Breast cancer Trial Population Patients Design Endpoints Status CVRM Phase III HER2-positive, unresectable and/or 600 • Randomised open label parallel assignment • Primacy endpoint: PFS •
Slide 23: Oncology: Enhertu breast cancer
Approved medicines Late-stage development Oncology Enhertu (trastuzumab deruxtecan, HER2 ADC) Early development Breast cancer Trial Population Patients Design Endpoints Status CVRM Phase III High-risk HER2-positive early non- 624 • Randomized open label parallel assignment • Primary endpoint: pCR •
Slide 24: Oncology: Enhertu gastric cancer
Approved medicines Late-stage development Oncology Enhertu (trastuzumab deruxtecan, HER2 ADC) Early development Gastric cancer Trial Population Patients Design Endpoints Status CVRM Phase III HER2-positive gastric cancer or gastro- 490 • Open label randomised parallel group assignment • Primary endp
Slide 25: Oncology: Enhertu other cancers
Approved medicines Late-stage development Oncology Enhertu (trastuzumab deruxtecan, HER2 ADC) Early development Other cancers Trial Population Patients Design Endpoints Status CVRM Phase III HER2 mutated, unresectable, locally 264 • Randomised parallel group assignment • Primary endpoint: PFS • FPCD
Slide 26: Oncology: Enhertu other cancers
Approved medicines Late-stage development Oncology Enhertu (trastuzumab deruxtecan, HER2 ADC) Early development Other cancers Trial Population Patients Design Endpoints Status CVRM Phase Ib HER2-over-expressing, unresectable 136 • Non randomised parallel group assignment • Primary endpoint: safety •
Slide 27: Oncology: Calquence blood cancers
Approved medicines Late-stage development Oncology Calquence (BTK inhibitor) Early development Blood cancers Trial Population Patients Design Endpoints Status CVRM Phase III Previously untreated CLL 535 • Arm A: chlorambucil + obinutuzumab • Primary endpoint: PFS (Arm A vs. Arm • FPCD: Q2 2015 ACE-C
Slide 28: Oncology: Calquence blood cancers
Approved medicines Late-stage development Oncology Calquence (BTK inhibitor) Early development Blood cancers Trial Population Patients Design Endpoints Status CVRM Phase II Relapsed/refractory and treatment 48 • Calquence monotherapy • Primary endpoint: ORR • FPCD: Q4 2014 15-H-0016 naïve/del17p CLL
Slide 29: Oncology: Calquence blood cancers
Approved medicines Late-stage development Oncology Calquence (BTK inhibitor) Early development Blood cancers Trial Population Patients Design Endpoints Status CVRM Phase I Japanese adults with advanced B-cell 35 • Part 1: Dose confirmation in r/r B-cell Malignancies • Primary endpoint: safety • FPCD
Slide 30: Oncology: Orpathys NSCLC and other cancers
Approved medicines Late-stage development Oncology Orpathys (savolitinib, MET inhibitor) Early development NSCLC and other cancers Trial Population Patients Design Endpoints Status CVRM Phase III Locally advanced or metastatic NSCLC 163 • Single arm trial • Primary endpoint: ORR • FPCD: Q3 2021 NCT0
Slide 31: Oncology: Capivasertib breast and prostate cancer
Approved medicines Late-stage development Oncology Capivasertib (AKT inhibitor) Early development Breast cancer, prostate cancer Trial Population Patients Design Endpoints Status CVRM Phase III Locally advanced or metastatic TNBC 924 • Double-blind randomised comparative trial • Primary endpoint: OS
Slide 32: Oncology: Camizestrant breast cancer
Approved medicines Late-stage development Oncology Camizestrant (AZD9833, next generation oral SERD) Early development Breast cancer Trial Population Patients Design Endpoints Status CVRM Phase III HR+ HER2- breast cancer 1342 • Randomised, double-blind, comparative trial • Primary endpoint: PFS • F
Slide 33: Oncology: Camizestrant breast cancer
Approved medicines Late-stage development Oncology Camizestrant (AZD9833, next generation oral SERD) Early development Breast cancer Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers 32 • Randomised, open-label • Primary outcome: relative • FPCD: Q3 2020 NCT04546347 b
Slide 34: Oncology: Monalizumab cancer
Approved medicines Late-stage development Oncology Monalizumab (NKG2a mAb) Early development Cancer Trial Population Patients Design Endpoints Status CVRM Phase III Recurrent or metastatic HNSCC, 2L 600 • Arm A: monalizumab + cetuximab i.v. • Primary: OS • FPCD: Q4 2020 INTERLINK-1 • Arm B: placebo
Slide 35: Oncology: Datopotamab deruxtecan NSCLC
Approved medicines Late-stage development Oncology Datopotamab deruxtecan (TROP2 ADC) Early development NSCLC Trial Population Patients Design Endpoints Status CVRM Phase III Previously treated advanced or 590 • Randomised, open label, parallel assignment • Primary endpoints: PFS, OS • FPCD: Q1 2021
Slide 36: Oncology: Datopotamab deruxtecan other cancers
Approved medicines Late-stage development Oncology Datopotamab deruxtecan (TROP2 ADC) Early development NSCLC and other cancers Trial Population Patients Design Endpoints Status CVRM Phase III Inoperable or metastatic HR+, HER2- 700 • Open-label, randomised • Primary endpoints: PFS, OS • FPCD: Q4 20
Slide 37: Oncology – early-stage development
Oncology – early-stage development
Slide 38: Oncology: AZD0171 cancer
Approved medicines Late-stage development Oncology AZD0171 (anti-LIF mAb) Early development Cancer Trial Population Patients Design Endpoints Status CVRM Phase II 1L metastatic pancreatic ductal 115 • Open-label, non-randomised trial of AZD0171 + Imfinzi + • Primary endpoints: Safety, OS at 12m • FP
Slide 39: Oncology: AZD0466 cancer
Approved medicines Late-stage development Oncology AZD0466 (Bcl2/xL inhibitor) Early development Cancer Trial Population Patients Design Endpoints Status CVRM Phase I/II Advanced haematologic malignancies 160 • Module 1 • Primary endpoint: safety • FPCD: Q2 2021 NCT04865419 • Part A: Dose escalation
Slide 40: Oncology: AZD1390 cancer
Approved medicines Late-stage development Oncology AZD1390 (ATM inhibitor) Early development Cancer Trial Population Patients Design Endpoints Status CVRM Phase I Recurrent glioblastoma eligible for re- 120 • Open label trial • Primary endpoints: safety, tolerability, • FPCD: Q2 2018 NCT03423628 irr
Slide 41: Oncology: AZD2936 solid tumours
Approved medicines Late-stage development Oncology 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 ARTE
Slide 42: Oncology: AZD4573 blood cancers
Approved medicines Late-stage development Oncology AZD4573 (CDK9 inhibitor) Early development Blood cancers Trial Population Patients Design Endpoints Status CVRM Phase II R/R Peripheral T-cell lymphoma and R/R 90 • Open label, non-randomised modular dose confirmation • Primary endpoints: Efficacy •
Slide 43: Oncology: AZD5305 solid tumours
Approved medicines Late-stage development Oncology AZD5305 (PARP1 inhibitor) Early development Solid tumours Trial Population Patients Design Endpoints Status CVRM Phase I/IIa Advanced, metastatic HER2- breast 715 • A modular, open-label, multicentre trial dose escalation • Primary endpoints: safety
Slide 44: Oncology: AZD5991 blood cancers
Approved medicines Late-stage development Oncology AZD5991 (MCL1 inhibitor) Early development Blood cancers Trial Population Patients Design Endpoints Status CVRM Phase I/Ib/IIa Relapsed/refractory haematologic 144 • Arm 1: AZD5991 dose escalation and expansion in R/R • Primary endpoint: safety • FP
Slide 45: Oncology: AZD7789 cancer
Approved medicines Late-stage development Oncology 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 NC
Slide 46: Oncology: AZD8205 cancer
Approved medicines Late-stage development Oncology AZD8205 (B7H4 ADC) Early development Cancer Trial Population Patients Design Endpoints Status CVRM Phase I/II Breast Cancer, Cholangiocarcinoma, 198 • Open-label, non-randomised dose-escalation and dose- • Primary endpoints: AE, SAE, DLTs, • FPCD: Q
Slide 47: Oncology: AZD8701 solid tumours
Approved medicines Late-stage development Oncology AZD8701 (FOXP3 antisense oligonucleotide) Early development Solid tumours Trial Population Patients Design Endpoints Status CVRM Phase I/Ib Advanced solid tumours 153 • Dose escalation and dose expansion trial • Primary endpoints: safety & • FPCD: Q
Slide 48: Oncology: AZD8853 solid tumours
Approved medicines Late-stage development Oncology AZD8853 (anti-GDF15) Early development Solid tumours Trial Population Patients Design Endpoints Status CVRM Phase I/II Advanced, unresectable or metastatic 165 • Open label trial • Primary endpoints: safety and • FPCD: Q3 2022 NCT05397171 NSCLC, Mic
Slide 49: Oncology: Ceralasertib cancer
Approved medicines Late-stage development Oncology Ceralasertib (AZD6738, ATR inhibitor) Early development Cancer Trial Population Patients Design Endpoints Status CVRM Phase II Solid tumours 61 • Cohort A: ceralasertib; ATM-altered AST • Cohort A primary endpoint: ORR • FPCD: Q1 2021 PLANETTE mCR
Slide 50: Oncology: IPH5201 solid tumours
Approved medicines Late-stage development Oncology IPH5201 (CD39 mAb) Early development Solid tumours Trial Population Patients Design Endpoints Status CVRM Phase I Advanced solid tumours 204 • Open-label, dose-escalation trial to determine MTD of • Primary endpoints: AE, SAE, DLT • FPCD: Q1 2020 NC
Slide 51: Oncology: MEDI1191 cancer
Approved medicines Late-stage development Oncology 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 • Primary endpoints: safety and • FPCD: Q2 2019 NCT0394680
Slide 52: Oncology: MEDI5752 cancer
Approved medicines Late-stage development Oncology MEDI5752 (PD-1/CTLA-4 bispecific mAb) Early development Cancer Trial Population Patients Design Endpoints Status CVRM Phase Ib Advanced renal cell carcinoma 70 • Open-label, dose-escalation and dose-expansion trial • Dose escalation primary endpoint
Slide 53: Oncology: MEDI9253 solid tumours
Approved medicines Late-stage development Oncology 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 NCT046134
Slide 54: Oncology: Oleclumab cancer
Approved medicines Late-stage development Oncology 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 Study 5 1L and 2L wit
Slide 55: BioPharmaceuticals – approved medicines and late-stage pipeline
BioPharmaceuticals – approved medicines and late-stage pipeline
Slide 56: Andexxa haematology
Approved medicines Late-stage development Oncology Andexxa (anti-factor Xa reversal) Early development Haematology Trial Population Patients Design Endpoints Status CVRM Phase IV Acute Intracranial 1200 • Arm 1: Andexxa • Primary endpoint: proportion of • FPCD: Q2 2019 I8-513 (Post Launch) Haemorrha
Slide 57: Farxiga heart failure and chronic kidney disease
Approved medicines Late-stage development Oncology Farxiga (SGLT2 inhibitor) Early development Heart failure and chronic kidney disease Trial Population Patients Design Endpoints Status CVRM Phase III CHF patients with HFpEF 6263 • Arm 1: Farxiga 10mg QD • Primary endpoint: time to the first • FPCD:
Slide 58: Lokelma hyperkalaemia
Approved medicines Late-stage development Oncology Lokelma (sodium zirconium cyclosilicate) 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 59: Roxadustat anaemia
Approved medicines Late-stage development Oncology Roxadustat (HIF-PH inhibitor) Early development Anaemia Trial Population Patients Design Endpoints Status CVRM Phase III Anaemia in lower risk MDS 204 • Open label roxadustat lead-in • Primary endpoint: proportion of • FPCD: Q3 2017 Efficacy and Saf
Slide 60: AZD4831 cardiovascular disease
Approved medicines Late-stage development Oncology AZD4831 (MPO inhibitor) Early development Cardiovascular disease Trial Population Patients Design Endpoints Status CVRM Phase IIb/III HFpEF 1485 • Randomised, double blind • Endpoints: Efficacy and Safety • FPCD: Q3 2021 ENDEAVOR • Arm 1: 2.5mg AZD4
Slide 61: Eplontersen ATTR
Approved medicines Late-stage development Oncology eplontersen (ligand-conjugated antisense) Early development ATTR Trial Population Patients Design Endpoints Status CVRM Phase III Patients with hereditary or 750 • Arm 1: eplontersen s.c. • Primary endpoint: composite • FPCD: Q1 2020 CARDIO-TTRansfo
Slide 62: Tozorakimab diabetic kidney disease
Approved medicines Late-stage development Oncology 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 endpoint: Urine albumin • FPCD:
Slide 63: Breztri, Trixeo asthma
Approved medicines Late-stage development Oncology Breztri, Trixeo (PT010, LAMA/LABA/ICS, pMDI) 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
Slide 64: Daliresp/Daxas COPD
Approved medicines Late-stage development Oncology Daliresp/Daxas (PDE4 inhibitor, oral) Early development COPD Trial Population Patients Design Endpoints Status CVRM Phase IV COPD 124080 • A retrospective cohort trial comparing COPD patients aged • Primary endpoint: all-cause mortality • FPCD: Q1 2
Slide 65: Fasenra dermatology
Approved medicines Late-stage development Oncology Fasenra (IL-5R mAb) 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
Slide 66: Fasenra gastrointestinal diseases
Approved medicines Late-stage development Oncology Fasenra (IL-5R mAb) Early development Gastrointestinal diseases Trial Population Patients Design Endpoints Status CVRM Phase III Documented diagnosis of EoE 211 • Double blind treatment period and open label period(s) • Primary endpoints: histologic
Slide 67: Fasenra nasal polyposis and other eosinophilic diseases
Approved medicines Late-stage development Oncology Fasenra (IL-5R mAb) Early development Nasal polyposis and other eosinophilic diseases Trial Population Patients Design Endpoints Status CVRM Phase III Patients with severe bilateral 413 • Arm 1: Fasenra 30mg Q8W s.c. • Primary endpoint: effect of Fa
Slide 68: Fasenra severe, uncontrolled asthma
Approved medicines Late-stage development Oncology Fasenra (IL-5R mAb) Early development Severe, uncontrolled asthma Trial Population Patients Design Endpoints Status CVRM Phase IIIb Severe eosinophilic 599 • Arm 1: Fasenra 30mg Q8W s.c. • Primary endpoint: reduction of oral • FPCD: Q3 2018 PONENTE
Slide 69: Fasenra asthma, COPD and other eosinophilic diseases
Approved medicines Late-stage development Oncology Fasenra (IL-5R mAb) Early development Severe, uncontrolled asthma, COPD and other eosinophilic diseases Trial Population Patients Design Endpoints Status CVRM Phase III Patients with moderate to 642 • Double-blind, placebo controlled • Primary endpo
Slide 70: Saphnelo lupus
Approved medicines Late-stage development Oncology Saphnelo (type I interferon receptor mAb) Early development Lupus (SLE / LN) Trial Population Patients Design Endpoints Status CVRM Phase III Moderate to severe SLE 630 • Arm 1: 300mg i.v. Saphnelo Q4W for 152 weeks • Primary endpoint: extension to
Slide 71: Tezspire severe, uncontrolled asthma
Approved medicines Late-stage development Oncology Tezspire (TSLP mAb) Early development Severe, uncontrolled asthma Trial Population Patients Design Endpoints Status CVRM Phase III Severe asthma 1061 • Arm 1: Tezspire s.c. • Primary endpoint: Annual asthma • FPCD: Q1 2018 NAVIGATOR Age 12-80 years
Slide 72: Tezspire asthma, COPD and CRSwNP
Approved medicines Late-stage development Oncology Tezspire (TSLP mAb) Early development Severe, uncontrolled asthma, COPD & CRSwNP Trial Population Patients Design Endpoints Status CVRM Phase III Severe chronic rhinosinusitis 400 • Arm 1: Tezspire s.c. • Co-primary endpoint: nasal polyp • FPCD: Q2
Slide 73: Brazikumab inflammatory bowel disease
Approved medicines Late-stage development Oncology Brazikumab (IL-23 inhibitor) Early development Inflammatory bowel disease (Crohn’s disease, ulcerative colitis) Trial Population Patients Design Endpoints Status CVRM Phase III Crohn’s Disease 161 • An Open-label, Long-term Extension Safety Study of
Slide 74: Brazikumab inflammatory bowel disease
Approved medicines Late-stage development Oncology Brazikumab (IL-23 inhibitor) Early development Inflammatory bowel disease (Crohn’s disease, ulcerative colitis) Trial Population Patients Design Endpoints Status CVRM Phase II Ulcerative colitis 165 • A Phase 2 Open-label, Long-term Extension Safety
Slide 75: PT027 asthma
Approved medicines Late-stage development Oncology PT027 (SABA/ICS, pMDI) Early development Asthma Trial Population Patients Design Endpoints Status CVRM Phase III Moderate to severe asthma 3132 • Randomised, double-blind, multicentre, parallel group • Primary endpoint: Time to first severe • FPCD:
Slide 76: Tozorakimab atopic dermatitis, asthma
Approved medicines Late-stage development Oncology 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
Slide 77: Tozorakimab COPD
Approved medicines Late-stage development Oncology Tozorakimab (IL-33 ligand mAb) Early development COPD Trial Population Patients Design Endpoints Status CVRM Phase III Adults with symptomatic 1272 • Randomised, double-blind, placebo-controlled, parallel • Primary endpoint: annualized rate of • FPC
Slide 78: Evusheld COVID-19 prevention and treatment
Approved medicines Late-stage development Oncology Evusheld (AZD7442, tixagevimab + cilgavimab) Early development Prevention and treatment of COVID-19 Trial Population Patients Design Endpoints Status CVRM Phase III Adults having 5197 • Double-blinded, randomised, placebo controlled, multi • Primary
Slide 79: Evusheld COVID-19 prevention and treatment
Approved medicines Late-stage development Oncology Evusheld (AZD7442, tixagevimab + cilgavimab) Early development Prevention and treatment of COVID-19 Trial Population Patients Design Endpoints Status CVRM Phase I Healthy adults 207 • Open-label, randomised three-arm, single dose trial • Key Endpoin
Slide 80: Vaxzevria prevention of COVID-19
Approved medicines Late-stage development Oncology Vaxzevria (SARS-CoV-2) Early development Prevention of COVID-19 Trial Population Patients Design Endpoints Status CVRM Phase IV Seronegative 360 • Open-label, non-randomized, multi-cohort, • Immunogenicity and safety • FPCD: Q1 2022 VICTORIA immunoc
Slide 81: Vaxzevria prevention of COVID-19
Approved medicines Late-stage development Oncology Vaxzevria (SARS-CoV-2) Early development Prevention of COVID-19 Trial Population Patients Design Endpoints Status CVRM Phase II Healthy children and 261 • Single-blinded, randomized multicentre trial • Primary endpoints: safety, tolerability, • FPCD
Slide 82: Vaxzevria prevention of COVID-19
Approved medicines Late-stage development Oncology Vaxzevria (SARS-CoV-2) Early development Prevention of COVID-19 Trial Population Patients Design Endpoints Status CVRM Phase I/II Healthy adults 400 • Double-blinded, randomised, placebo-controlled multicentre • Primary endpoints: safety, tolerabili
Slide 83: Evusheld COVID-19 prevention and treatment
Approved medicines Late-stage development Oncology Evusheld (AZD7442, tixagevimab + cilgavimab) Early development Prevention and treatment of COVID-19 Trial Population Patients Design Endpoints Status CVRM Phase II Adults and pediatric 200 • Randomized, open-label, dose-ranging to assess safety, • P
Slide 84: Nirsevimab respiratory syncytial virus infection
Approved medicines Late-stage development Oncology Nirsevimab (Respiratory syncytial virus mAb-YTE) Early development Infection Trial Population Patients Design Endpoints Status CVRM Phase III Healthy infants (born 35 3000 • Randomised, Double-blind, placebo-controlled • Primary endpoints: efficacy
Slide 85: BioPharmaceuticals – early-stage development
BioPharmaceuticals – early-stage development
Slide 86: AZD0780 dyslipidaemia
Approved medicines Late-stage development Oncology AZD0780 (PCSK9 inhibitor) Early development Dyslipidaemia Trial Population Patients Design Endpoints Status CVRM Phase I Healthy adults 132 • Randomised, placebo controled SAD/MAD trial • Primary endpoint: safety and • FPCD: Q3 2022 NCT05384262 tole
Slide 87: AZD2373 chronic kidney disease
Approved medicines Late-stage development Oncology AZD2373 Early development Chronic kidney disease Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers 30 • SAD. Dose escalation in 6 cohorts with • Primary endpoints: safety and • FPCD: Q1 2020 NCT04269031 6 volunteers r
Slide 88: AZD2693 NASH
Approved medicines Late-stage development Oncology 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 201
Slide 89: AZD3366 cardiovascular disease
Approved medicines Late-stage development Oncology 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 readout: Q3 2
Slide 90: AZD3427 heart failure
Approved medicines Late-stage development Oncology 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 NCT0
Slide 91: AZD4831 cardiovascular disease
Approved medicines Late-stage development Oncology AZD4831 (MPO inhibitor) Early development Cardiovascular disease Trial Population Patients Design Endpoints Status CVRM Phase I Healthy Patients 14 • Open label • Primary endpoints: PK parameters • FPCD: Q1 2022 NCT05236543 • AZD4831 • Secondary end
Slide 92: AZD5462 heart failure
Approved medicines Late-stage development Oncology AZD5462 (relaxin) Early development Heart failure Trial Population Patients Design Endpoints Status CVRM Phase I SAD – Healthy volunteers 0 • Single centre single and multiple ascending dose study • Primary endpoints: safety and • FPCD: Q4 2021 NCT0
Slide 93: AZD5718 cardiovascular and chronic kidney disease
Approved medicines Late-stage development Oncology AZD5718 (FLAP inhibitor) Early development Cardiovascular disease & Chronic Kidney Disease Trial Population Patients Design Endpoints Status CVRM Phase IIb Proteinuric CKD 692 • Randomised, double-blind, placebo-controlled, multicentre, • Primary en
Slide 94: AZD7503 NASH
Approved medicines Late-stage development Oncology 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 202
Slide 95: AZD8233 dyslipidaemia
Approved medicines Late-stage development Oncology AZD8233 (PCSK9 inhibitor, subcutaneous) Early development Dyslipidaemia Trial Population Patients Design Endpoints Status CVRM Phase II Dyslipidaemia 376 • Arm 1: AZD8233 dose • Primary endpoints: safety and efficacy • FPCD: Q3 2021 SOLANO • Arm 2:
Slide 96: AZD9977 heart failure
Approved medicines Late-stage development Oncology AZD9977 (MR modulator) 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 • Primary endpoint: percent change • F
Slide 97: Cotadutide diabetes/CKD, NASH
Approved medicines Late-stage development Oncology Cotadutide (GLP-1-glucagon agonist) Early development Diabetes/CKD, NASH Trial Population Patients Design Endpoints Status CVRM Phase II Obese patients with non- 74 • Arm 1: cotadutide high dose s.c. • Primary endpoints: safety and • FPCD: Q4 2019 N
Slide 98: MEDI6570 cardiovascular
Approved medicines Late-stage development Oncology MEDI6570 Early development Cardiovascular Trial Population Patients Design Endpoints Status CVRM Phase IIb Post MI 400 • Evaluation of anti-inflammatory potential and effect on • Primary endpoints: efficacy and safety • FPCD: Q4 2020 NCT04610892 sur
Slide 99: MEDI8367 chronic kidney disease
Approved medicines Late-stage development Oncology 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 20
Slide 100: Zibotentan chronic kidney disease
Approved medicines Late-stage development Oncology Zibotentan (endothelin receptor antagonist) Early development Chronic kidney disease Trial Population Patients Design Endpoints Status CVRM Phase IIb CKD 495 • • Primary endpoint: • FPCD: Q2 2021 ZENITH-CKD • Arm 1: zibotentan dose A + Farxiga 10 mg
Slide 101: Zibotentan liver cirrhosis
Approved medicines Late-stage development Oncology Zibotentan (endothelin receptor antagonist) Early development Liver Cirrhosis with Features of portal hypertension Trial Population Patients Design Endpoints Status CVRM Phase I Cohort 1: Moderate hepatic 24 • Single dose, single centre, Open label,
Slide 102: AZD1402 asthma
Approved medicines Late-stage development Oncology 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, • Part 1 primary endpoints: safety and •
Slide 103: AZD4604 asthma
Approved medicines Late-stage development Oncology 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 ast
Slide 104: AZD5055 idiopathic pulmonary fibrosis
Approved medicines Late-stage development Oncology AZD5055 (oral porcupine inhibitor) Early development Idiopathic pulmonary fibrosis (IPF) and other ILDs with progressive fibrosis Trial Population Patients Design Endpoints Status CVRM Phase I Healthy subjects 90 • SAD/MAD trial • Primary endpoints:
Slide 105: AZD5718 asthma
Approved medicines Late-stage development Oncology AZD5718 (FLAP inhibitor) Early development Asthma Trial Population Patients Design Endpoints Status CVRM Phase II Patients with moderate-to- 1928 • Randomised, placebo-controlled, double-blind, multicentre, • Primary endpoint; Time to first • FPCD:
Slide 106: AZD8630 asthma
Approved medicines Late-stage development Oncology 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: Q1 2022 NCT05110976 with asthma tolerability •
Slide 107: AZD4041 opioid use disorder
Approved medicines Late-stage development Oncology AZD4041 (orexin 1 receptor antagonist) Early development Opioid use disorder Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers 48 • Randomised, double blind, SAD trial • Primary endpoints: safety and • FPCD: Q4 2019 N
Slide 108: MEDI0618 osteoarthritis pain
Approved medicines Late-stage development Oncology 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.
Slide 109: MEDI1341 Parkinson's disease
Approved medicines Late-stage development Oncology MEDI1341 (alpha-synuclein mAb) Early development Parkinson’s disease Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers 48 • SAD trial • Primary endpoints: safety and • FPCD: Q4 2017 NCT03272165 • Arm 1: MEDI1341 i.v.
Slide 110: MEDI7352 osteoarthritis pain
Approved medicines Late-stage development Oncology MEDI7352 (NGF TNF bispecific mAb) Early development Osteoarthritis pain Trial Population Patients Design Endpoints Status CVRM Phase IIb Painful osteoarthritis of the 300 • MAD trial • Primary endpoint: dose response • FPCD: Q1 2021 NCT04675034 knee
Slide 111: Rare Disease – approved medicines and late-stage pipeline
Rare Disease – approved medicines and late-stage pipeline
Slide 112: Koselugo neurofibromatosis type 1
Approved medicines Late-stage development Oncology Koselugo (selumetinib, MEK inhibitor) Early development Neurofibromatosis Type 1 (NF1) Trial Population Patients Design Endpoints Status CVRM Phase III Adult age ≥18 years with NF1 who have 146 • Multicentre, international study with a parallel, ran
Slide 113: Koselugo paediatric NF1, solid tumours
Approved medicines Late-stage development Oncology Koselugo (selumetinib, MEK inhibitor) Early development Paediatric neurofibromatosis type 1, solid tumours Trial Population Patients Design Endpoints Status CVRM Phase II Paediatric NF1 75 • Single arm: Koselugo 25mg/m2 BID with 2 strata: • Primary
Slide 114: Soliris neurology
Approved medicines Late-stage development Oncology Soliris (anti-complement C5 mAb) 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 115: Ultomiris haematology and nephrology
Approved medicines Late-stage development Oncology Ultomiris (anti-complement C5 mAb) 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 116: Ultomiris neurology
Approved medicines Late-stage development Oncology Ultomiris (anti-complement C5 mAb) Early development Neurology Trial Population Patients Design Endpoints Status CVRM Phase III Neuromyelitis optica spectrum disorder 58 • Arm 1: Ultomiris Q8W • Primary endpoint: time to first • FPCD: Q4 2019 ALXN-1
Slide 117: Acoramidis ATTR-CM
Approved medicines Late-stage development Oncology acoramidis (ALXN2060) Early development ATTR-CM Trial Population Patients Design Endpoints Status CVRM Phase III ATTR-CM 22 • Arm 1: 800 mg acoramidis (ALXN2060) administered twice • Primary endpoint: Change from • FPCD: Q4 2020 ALXN2060-TAC-302 dai
Slide 118: ALXN1840 Wilson disease
Approved medicines Late-stage development Oncology 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 119: ALXN2050 haematology, nephrology, neurology
Approved medicines Late-stage development Oncology ALXN2050 (factor D inhibitor) Early development Haematology, nephrology, neurology Trial Population Patients Design Endpoints Status CVRM Phase II Generalized myasthenia gravis (gMG) 70 • Arm 1: ALXN2050 180mg • Primary endpoint: Myasthenia Gravis •
Slide 120: CAEL-101 AL amyloidosis
Approved medicines Late-stage development Oncology CAEL-101 (fibril-reactive mAb) Early development AL amyloidosis Trial Population Patients Design Endpoints Status CVRM Phase III Mayo stage IIIa amyloidosis 267 • Arm 1: CAEL-101 combined with SoC for plasma cell dyscrasia • Primary: time from first
Slide 121: Danicopan haematology and ophthalmology
Approved medicines Late-stage development Oncology 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 122: Rare Disease – early-stage development
Rare Disease – early-stage development
Slide 123: ALXN1720 neurology
Approved medicines Late-stage development Oncology 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 • Primary: Incidence of TEAEs and
Slide 124: ALXN1820 haematology
Approved medicines Late-stage development Oncology 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 hypophosphatasia
Approved medicines Late-stage development Oncology ALXN1850 (next generation asfotase alfa) Early development Hypophosphatasia Trial Population Patients Design Endpoints Status CVRM Phase I HPP 15 • Arm 1: ALXN1850, 3 cohorts at low, medium, and high • Primary: Incidence of TEAEs and TESAEs • FPCD:
Slide 126: ALXN1910 bone metabolism
Approved medicines Late-stage development Oncology ALXN1910 (Next Gen TNSALP ERT) Early development Bone metabolism Trial Population Patients Design Endpoints Status CVRM Phase I Healthy adults 48 • Randomised, placebo controlled SAD • Primary endpoint:: safety • FPCD: Q2 2022 ALXN1910-HV-101 • Data
Slide 127: ALXN2050 haematology, nephrology, neurology
Approved medicines Late-stage development Oncology 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 128: 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 129: 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 130: List of abbreviations
List of abbreviations SBRT Stereotactic body radiation therapy 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