Slide 1: Clinical Trials Appendix: H1 2023 Results Update
Clinical Trials Appendix H1 2023 Results Update
Slide 2: Key upcoming pipeline catalysts: 2023 and 2024
Key upcoming pipeline catalysts: 2023 and 2024 Oncology BioPharmaceuticals Rare Disease H2 2023 H1 2024 H2 2024 Lynparza – prostate cancer (1L) (PROpel) (JP) Enhertu – HER2+ breast cancer (3L) (DESTINY-Breast02) (US) Fasenra – asthma (MIRACLE) (CN) Enhertu – HER2m NSCLC (2L+) (DESTINY-Lung01, DESTIN
Slide 3: Clinical Trials Appendix: selected highlights
Clinical Trials Appendix: selected highlights BioPharmaceuticals Oncology Rare Disease Approved medicines: key LCM Dato-DXd (TROP2 ADC) tozorakimab (IL-33) capivasertib (AKT) AZD3152 (COVID-19 LAAB) vemircopan (oral Factor D) Next-wave pipeline camizestrant (ngSERD) eplontersen (LICA) AZD5305 (PARP-
Slide 4: Project movement since Q1 2023 update
Project movement since Q1 2023 update New to Phase I New to Phase II New to Pivotal trial New to registration NME NME NME ALXN1920 AZD2693 capivasertib + Faslodex CAPItello-291#1 kidney-targeted factor H fusion protein nephrology NASH resolution non-alcoholic steatohepatitis AKT inhibitor + Faxlodex
Slide 5: Q2 2023 Oncology new molecular entity pipeline
Q2 2023 Oncology new molecular entity1 pipeline Phase I Phase II Phase III 6 New Molecular Entities 12 New Molecular Entities 17 New Molecular Entities AZD1390 AZD0171 + Imfinzi + CTx camizestrant + CDK4/6i SERENA-6 camizestrant + palbociclib SERENA-4 ATM glioblastoma anti-LIF + PD-L1 + CTx metastat
Slide 6: Q2 2023 Oncology lifecycle management pipeline
Q2 2023 Oncology lifecycle management1 pipeline Phase I Phase II Phase III Under review 2 Projects 11 Projects 33 Projects 1 Project Calquence + venetoclax + obinutuzumab Enhertu DESTINY-Breast02# Enhertu (platform) DESTINY-Breast08# Enhertu (platform) DESTINY-Breast07# Calquence + R-CHOP ESCALADE C
Slide 7: Q2 2023 BioPharmaceuticals new molecular entity pipeline
Q2 2023 BioPharmaceuticals new molecular entity1 pipeline Phase I Phase II Phase III Under review 14 New Molecular Entities 13 New Molecular Entities 4 New Molecular Entities 1 New Molecular Entity AZD0780 atuliflapon AZD3152 SUPERNOVA¶ eplontersen# PCSK9 dyslipidemia FLAP asthma SARS-CoV-2 LAAB pre
Slide 8: Q2 2023 BioPharmaceuticals lifecycle management pipeline
Q2 2023 BioPharmaceuticals lifecycle management1 pipeline Phase I Phase II Phase III Under review 0 Projects 3 Projects 12 Projects 0 Projects Andexxa Breztri/Trixeo (PT010) KALOS LOGOS anti-factor Xa reversal urgent surgery LABA/LAMA/ICS asthma roxadustat # Farxiga DAPA-MI HIFPH anaemia chemotherap
Slide 9: Q2 2023 Rare Disease pipeline
Q2 2023 Rare Disease pipeline1 Phase I Phase II Phase III Under review 6 Projects 7 Projects 5 Projects 1 Project danicopan ALXN1850 danicopan acoramidis# factor D PNH with clinically significant extravascular next gen TNSALP ERT hypophosphatasia factor D geographic atrophy oral TTR stabilizer trans
Slide 10: Designations in our pipeline
Designations in our pipeline 3 12 8 14 27 Accelerated approvals Breakthrough / PRIME1 / Sakigake2 Fast Track Priority Review Orphan Andexxa acute major bleed (US) Beyfortus RSV mAb-YTE MELODY-MEDLEY (US) anselamibab AL amyloidosis (US) Beyfortus RSV mAb-YTE MELODY-MEDLEY (CN) Andexxa acute major ble
Slide 11: Oncology: approved medicines and late-stage pipeline
Oncology: approved medicines and late-stage pipeline
Slide 12: Oncology: Tagrisso
Approved medicines Oncology Tagrisso (highly-selective, irreversible EGFRi) Late-stage development Early development NSCLC Trial Population Patients Design Endpoints Status CVRM Phase III Maintenance therapy in patients with 200 • Arm 1: Tagrisso • Primary endpoint: PFS (BICR) • FPCD: Q4 2018 LAURA
Slide 13: Oncology: 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 + pemetrexed/carboplatin or • Primary endpoint: mPR •
Slide 14: Oncology: 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 Locally advanced or metastatic NSCLC 250 • Arm 1: Tagrisso + Orpathys • Primary endpoint: PFS • FP
Slide 15: Oncology: Imfinzi gastrointestinal cancer
Approved medicines Oncology Imfinzi (PD-L1 mAb) Late-stage development Early development Gastrointestinal cancer Trial Population Patients Design Endpoints Status CVRM Phase III Locoregional HCC 710 • Arm 1: TACE in combination with Imfinzi • Primary endpoint: PFS (Arm 2 vs. Arm • FPCD: Q1 2019 EMER
Slide 16: Oncology: Imfinzi lung cancer
Approved medicines Oncology Imfinzi (PD-L1 mAb) Late-stage development Early development Lung cancer Trial Population Patients Design Endpoints Status CVRM Phase III Neoadjuvant NSCLC patients, Stage II 800 • Arm 1: Imfinzi + platinum-based chemotherapy • Primary endpoints: pCR snd EFS • FPCD: Q1 20
Slide 17: Oncology: Imfinzi lung cancer
Approved medicines Oncology Imfinzi (PD-L1 mAb) Late-stage development Early development Lung cancer Trial Population Patients Design Endpoints Status CVRM Phase III Limited-stage SCLC 1L following 600 • Arm 1: Imfinzi + Imjudo (4 doses) • Primary endpoints: PFS and OS • FPCD: Q4 2018 ADRIATIC plati
Slide 18: Oncology: Imfinzi lung cancer
Approved medicines Oncology Imfinzi (PD-L1 mAb) Late-stage development Early development Lung cancer Trial Population Patients Design Endpoints Status CVRM Phase II Early stage, resectable NSCLC (Stage II 350 • Open-label trial • Primary endpoints: pCR, safety • FPCD: Q2 2022 NeoCOAST-2 to Stage III
Slide 19: Oncology: Imfinzi other cancer
Approved medicines Oncology Imfinzi (PD-L1 mAb) Late-stage development Early development Other cancer Trial Population Patients Design Endpoints Status CVRM Phase III Non-muscle invasive bladder cancer 1018 • Arm 1: BCG (induction + maintenance) • Primary endpoint: DFS • FPCD: Q2 2018 POTOMAC • Arm
Slide 20: Oncology: Lynparza Imfinzi combinations
Approved medicines Oncology Lynparza (PARP inhibitor) Late-stage development Early development Imfinzi combinations Trial Population Patients Design Endpoints Status CVRM Phase III 1L advanced ovarian cancer 1256 • Non-tBRCAm (tumour BRCA) patients • Primary endpoint: PFS • FPCD: Q1 2019 DUO-O • Arm
Slide 21: Oncology: Lynparza multiple cancers
Approved medicines Oncology Lynparza (PARP inhibitor) Late-stage development Early development Multiple cancers Trial Population Patients Design Endpoints Status CVRM Phase III gBRCAm adjuvant breast cancer 1836 • Arm 1: Lynparza BID 12-month duration • Primary endpoint: iDFS • FPCD: Q2 2014 OlympiA
Slide 22: Oncology: Lynparza other combinations
Approved medicines Oncology Lynparza (PARP inhibitor) Late-stage development Early development Other combinations Trial Population Patients Design Endpoints Status CVRM Phase III 1L metastatic castration-resistant 904 • Arm 1: Lynparza + abiraterone • Primary endpoint: rPFS • FPCD: Q4 2018 PROpel pr
Slide 23: Oncology: Enhertu breast cancer
Approved medicines Oncology Enhertu (trastuzumab deruxtecan, HER2 ADC) Late-stage development Early development Breast cancer Trial Population Patients Design Endpoints Status CVRM Phase III HER2-positive, unresectable and/or 600 • Randomised, open-label, parallel assignment • Primacy endpoint: PFS
Slide 24: Oncology: Enhertu breast cancer
Approved medicines Oncology Enhertu (trastuzumab deruxtecan, HER2 ADC) Late-stage development Early development Breast cancer Trial Population Patients Design Endpoints Status CVRM Phase III High-risk HER2-positive early non- 624 • Randomised, open-label, parallel assignment • Primary endpoint: pCR
Slide 25: Oncology: Enhertu gastric cancer
Approved medicines Oncology Enhertu (trastuzumab deruxtecan, HER2 ADC) Late-stage development Early development Gastric cancer Trial Population Patients Design Endpoints Status CVRM Phase III HER2-positive gastric cancer or gastro- 490 • Open-label, randomised, parallel group assignment • Primary en
Slide 26: Oncology: Enhertu other cancers
Approved medicines Oncology Enhertu (trastuzumab deruxtecan, HER2 ADC) Late-stage development Early development Other cancers Trial Population Patients Design Endpoints Status CVRM Phase III HER2-mutated, unresectable, locally 264 • Randomised, parallel group assignment • Primary endpoint: PFS • FPC
Slide 27: Oncology: Enhertu other cancers
Approved medicines Oncology Enhertu (trastuzumab deruxtecan, HER2 ADC) Late-stage development Early development Other cancers Trial Population Patients Design Endpoints Status CVRM Phase Ib HER2-over-expressing, unresectable 136 • Non-randomised, parallel group assignment • Primary endpoint: safety
Slide 28: Oncology: Calquence blood cancers
Approved medicines Oncology Calquence (BTK inhibitor) Late-stage development Early development Blood cancers Trial Population Patients Design Endpoints Status CVRM Phase III Previously untreated CLL 981 • Arm 1: Calquence + venetoclax • Primary endpoint: IRC PFS (Arm 1 vs. • FPCD: Q1 2019 AMPLIFY (A
Slide 29: Oncology: Calquence blood cancers
Approved medicines Oncology Calquence (BTK inhibitor) Late-stage development Early development Blood cancers Trial Population Patients Design Endpoints Status CVRM Phase Ib MCL 61 • Calquence in combination with bendamustine and • Primary endpoint: safety • FPCD: Q2 2016 ACE-LY-106 rituxumab • LPCD:
Slide 30: Oncology: 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 III Locally advanced or metastatic NSCLC 163 • Single-arm trial • Primary endpoint: ORR • FPCD: Q3 2021 NCT0
Slide 31: Oncology: capivasertib
Approved medicines Oncology capivasertib (AKT inhibitor) Late-stage development Early development Breast cancer and prostate cancer Trial Population Patients Design Endpoints Status CVRM Phase III Locally advanced or metastatic TNBC 924 • Double-blind, randomised, comparative trial • Primary endpoin
Slide 32: Oncology: camizestrant
Approved medicines Oncology camizestrant (AZD9833, next-generation oral SERD) Late-stage development Early development Breast cancer Trial Population Patients Design Endpoints Status CVRM Phase III HR+ HER2- advanced breast cancer 1342 • Randomised, double-blind, comparative trial • Primary endpoint
Slide 33: Oncology: camizestrant
Approved medicines Oncology camizestrant (AZD9833, next-generation oral SERD) Late-stage development Early development Breast cancer Trial Population Patients Design Endpoints Status CVRM Phase I HR+ HER2- advanced breast cancer 403 • Escalation phase: open-label multi-centre trial • Primary endpoin
Slide 34: Oncology: datopotamab deruxtecan NSCLC
Approved medicines Oncology datopotamab deruxtecan (TROP2 ADC) Late-stage development Early development NSCLC Trial Population Patients Design Endpoints Status CVRM Phase III Previously treated advanced or 590 • Randomised, open-label, parallel assignment • Primary endpoints: PFS and OS • FPCD: Q1 2
Slide 35: Oncology: datopotamab deruxtecan NSCLC
Approved medicines Oncology datopotamab deruxtecan (TROP2 ADC) Late-stage development Early development NSCLC Trial Population Patients Design Endpoints Status CVRM Phase II Advanced or metastatic NSCLC with 137 • Single-arm, open-label • Primary endpoint: ORR • FPCD: Q1 2021 TROPION-Lung05 actionab
Slide 36: Oncology: datopotamab deruxtecan other cancers
Approved medicines Oncology datopotamab deruxtecan (TROP2 ADC) Late-stage development Early development Other cancers Trial Population Patients Design Endpoints Status CVRM Phase III Inoperable or metastatic HR+ HER2- 733 • Open-label, randomised • Primary endpoints: PFS (BICR) and OS • FPCD: Q4 202
Slide 37: Oncology: datopotamab deruxtecan other cancers
Approved medicines Oncology datopotamab deruxtecan (TROP2 ADC) Late-stage development Early development Other cancers Trial Population Patients Design Endpoints Status CVRM Phase II Endometrial cancer, gastric cancer, 531 • Sub-study 1 (endometrial cancer); • Primary endpoints: ORR and safety • FPCD
Slide 38: Oncology: datopotamab deruxtecan NSCLC and other cancers
Approved medicines Oncology datopotamab deruxtecan (TROP2 ADC) Late-stage development Early development NSCLC and other cancers Trial Population Patients Design Endpoints Status CVRM Phase I/II NSCLC and TNBC and other solid 119 • Single-arm, multi-cohort study with no blinding • Primary endpoint: O
Slide 39: Oncology: ceralasertib
Approved medicines Oncology ceralasertib (AZD6738, ATR inhibitor) Late-stage development Early development Multiple cancers Trial Population Patients Design Endpoints Status CVRM Phase III Post-IO NSCLC 580 • Double-arm randomised: • Primary endpoint: OS • FPCD: Q4 2022 LATIFY • Arm 1: ceralasertib
Slide 40: Oncology: early-stage development
Oncology: early-stage development
Slide 41: Oncology: AZD0171
Approved medicines Oncology AZD0171 (anti-LIF mAb) Late-stage development Early development Cancer Trial Population Patients Design Endpoints Status CVRM Phase II 1L metastatic pancreatic ductal 115 • Open-label, non-randomised trial • Primary endpoints: safety, OS at 12 • FPCD: Q1 2022 NCT04999969
Slide 42: Oncology: AZD0466
Approved medicines Oncology AZD0466 (Bcl2/xL inhibitor) Late-stage development Early development Cancer Trial Population Patients Design Endpoints Status CVRM Phase I/II Advanced haematologic malignancies 141 • Module 1: • Primary endpoint: safety • FPCD: Q2 2021 NCT04865419 • Part A: dose escalatio
Slide 43: Oncology: AZD1390
Approved medicines Oncology AZD1390 (ATM inhibitor) Late-stage development Early development Cancer Trial Population Patients Design Endpoints Status CVRM Phase I Recurrent glioblastoma eligible for re- 120 • Open-label trial • Primary endpoints: safety, tolerability • FPCD: Q2 2018 NCT03423628 irra
Slide 44: Oncology: AZD4573
Approved medicines Oncology AZD4573 (CDK9 inhibitor) Late-stage development Early development Blood cancers Trial Population Patients Design Endpoints Status CVRM Phase II R/R Peripheral T-cell lymphoma and R/R 79 • Open label, non-randomised modular dose confirmation • Primary endpoint: efficacy •
Slide 45: Oncology: AZD5305
Approved medicines Oncology AZD5305 (PARP1 inhibitor) Late-stage development Early development Solid tumours Trial Population Patients Design Endpoints Status CVRM Phase I/IIa Advanced, metastatic HER2- breast 559 • Modular, open-label, multi-centre dose escalation and • Primary endpoints: safety an
Slide 46: Oncology: AZD5335
Approved medicines Oncology AZD5335 (anti-FRα TOP1i ADC) Late-stage development Early development Solid tumours, ovarian cancer, lung cancer Trial Population Patients Design Endpoints Status CVRM Phase I/II Advanced solid tumour malignancies 150 • Module 1: AZD5335 monotherapy • Primary endpoints: s
Slide 47: Oncology: AZD8205
Approved medicines Oncology AZD8205 (B7H4 ADC) Late-stage development Early development Solid tumours Trial Population Patients Design Endpoints Status CVRM Phase I/II Breast cancer, biliary tract cancer, 280 • Open-label, non-randomised dose-escalation, and • Primary endpoints: AE, SAE, DLTs, • FPC
Slide 48: Oncology: AZD9574
Approved medicines Oncology AZD9574 (PARP1-sel BBB inhibitor) Late-stage development Early development Solid tumours Trial Population Patients Design Endpoints Status CVRM Phase I/IIa Advanced solid malignancies 195 • Modular, open-label, multi-centre dose escalation and • Primary endpoints: safety
Slide 49: Oncology: AZD9592
Approved medicines Oncology AZD9592 (EGFR-cMET TOP1i ADC) Late-stage development Early development Lung cancer Trial Population Patients Design Endpoints Status CVRM Phase I Advanced solid tumours including 108 • Escalation phase, open-label, multi-centre trial • Primary endpoints (escalation): safe
Slide 50: Oncology: IPH5201
Approved medicines Oncology IPH5201 (CD39 mAb) Late-stage development Early development Solid tumours Trial Population Patients Design Endpoints Status CVRM Phase I Advanced solid tumours 57 • Open-label, dose-escalation trial to determine MTD of • Primary endpoints: AE, SAE and DLT • FPCD: Q1 2020
Slide 51: Oncology: oleclumab
Approved medicines Oncology oleclumab (CD73 mAb) Late-stage development Early development Solid tumours Trial Population Patients Design Endpoints Status CVRM Phase Ib/II Pancreatic 1L and 2L with prior 339 • Arm A1: gemcitabine and nab paclitaxel i.v. • Primary endpoints: safety and anti- • FPCD: Q
Slide 52: Oncology: rilvegostomig
Approved medicines Oncology rilvegostomig (AZD2936, PD-1/TIGIT bispecific mAb) Late-stage development Early development Solid tumours Trial Population Patients Design Endpoints Status CVRM Phase I/II NSCLC 192 • Open-label, dose escalation and dose expansion trial • Primary endpoints (Part A): safet
Slide 53: Oncology: sabestomig
Approved medicines Oncology sabestomig (AZD7789, PD-1/TIM3 bispecific mAb) Late-stage development Early development Cancer Trial Population Patients Design Endpoints Status CVRM Phase I/IIa NSCLC, other tumours 152 • Open-label, non-randomised dose-escalation and dose- • Primary endpoints: AE, SAE,
Slide 54: Oncology: TNB-486
Approved medicines Oncology TNB-486 (CD19/CD3 next-generation bispecific T-cell engager) Late-stage development Early development Haematologic malignancies Trial Population Patients Design Endpoints Status CVRM Phase I R/R B-cell non-Hodgkin lymphoma 116 • Multi-centre, Phase I, open-label, dose-esc
Slide 55: Oncology: volrustomig
Approved medicines Oncology volrustomig (MEDI5752, PD-1/CTLA-4 bispecific mAb) Late-stage development Early development Solid tumours Trial Population Patients Design Endpoints Status CVRM Phase Ib Advanced renal cell carcinoma 70 • Open-label, dose escalation and dose expansion trial • Primary endp
Slide 56: Oncology: NT-125
Approved medicines Oncology NT-125 (autologous, multi-specific neoantigen-targeting TCR-T) Late-stage development Early development Solid tumours Trial Population Patients Design Endpoints Status CVRM Phase Ia/Ib Adults with recurrent or metastatic 42 • Open-label, single-arm, single-centre trial wi
Slide 57: BioPharmaceuticals: approved medicines and late-stage pipeline
BioPharmaceuticals: approved medicines and late-stage pipeline
Slide 58: Andexxa
Approved medicines Oncology Andexxa (anti-factor Xa reversal) Late-stage development Early development Haematology Trial Population Patients Design Endpoints Status CVRM Phase IV Acute intracranial 1200 • Arm 1: Andexxa • Primary endpoint: proportion of • FPCD: Q2 2019 I8-513 (post-launch) haemorrha
Slide 59: Farxiga
Approved medicines Oncology Farxiga (SGLT2 inhibitor) Late-stage development Early development Heart failure and chronic kidney disease Trial Population Patients Design Endpoints Status CVRM Phase III CHF patients with HFpEF 6263 • Arm 1: Farxiga 10mg QD • Primary endpoint: time to the first • FPCD:
Slide 60: Lokelma
Approved medicines Oncology Lokelma (sodium zirconium cyclosilicate) Late-stage development Early development Hyperkalaemia Trial Population Patients Design Endpoints Status CVRM Phase IIIb ESRD with hyperkalaemia and 134 • Arm 1: Lokelma 5g QD for 8 weeks on non-dialysis days with • Primary endpoin
Slide 61: roxadustat
Approved medicines Oncology roxadustat (HIF-PH inhibitor) Late-stage development Early development Anaemia Trial Population Patients Design Endpoints Status CVRM Phase III Anaemia in lower-risk MDS 184 • Open-label roxadustat lead-in • Primary endpoint: proportion of • FPCD: Q3 2017 MATTERHORN patie
Slide 62: baxdrostat
Approved medicines Oncology baxdrostat (selective aldosterone synthase inhibitor) Late-stage development Early development Hypertension Trial Population Patients Design Endpoints Status CVRM Phase II Patients with primary 18 • Arm 1: baxdrostat 2-8mg QD • Primary endpoints: safety and • FPCD: Q3 202
Slide 63: eplontersen
Approved medicines Oncology eplontersen (ligand-conjugated antisense) Late-stage development Early development ATTR Trial Population Patients Design Endpoints Status CVRM Phase III Hereditary or wild-type 1400 • Arm 1: eplontersen s.c. • Primary endpoints: composite • FPCD: Q1 2020 CARDIO-TTRansform
Slide 64: mitiperstat
Approved medicines Oncology mitiperstat (MPO inhibitor) Late-stage development Early development Cardiovascular disease Trial Population Patients Design Endpoints Status CVRM Phase IIb/III HFpEF 1485 • Randomised, double-blind • Primary endpoints: safety and efficacy • FPCD: Q3 2021 ENDEAVOR • Arm 1
Slide 65: tozorakimab diabetic kidney disease
Approved medicines Oncology tozorakimab (IL-33 ligand mAb) Late-stage development Early development Diabetic kidney disease Trial Population Patients Design Endpoints Status CVRM Phase II Adult patients with diabetic 581 • Arm 1: tozorakimab dose 1 + Farxiga • Primary endpoint: change from • FPCD: Q
Slide 66: zibotentan chronic kidney disease
Approved medicines Oncology zibotentan (endothelin receptor antagonist) Late-stage development Early development Chronic kidney disease Trial Population Patients Design Endpoints Status CVRM Phase I Healthy female volunteers of 24 • Open-label, single-sequence , single-centre trial • Primary endpoin
Slide 67: zibotentan liver cirrhosis
Approved medicines Oncology zibotentan (endothelin receptor antagonist) Late-stage development Early development Liver Cirrhosis with Features of portal hypertension Trial Population Patients Design Endpoints Status CVRM Phase II Part A: participants with 140 • Phase IIa/b multi-centre, randomised,
Slide 68: Airsupra
Approved medicines Oncology Airsupra (PT027, SABA/ICS, pMDI) Late-stage development Early development Asthma Trial Population Patients Design Endpoints Status CVRM Phase III Moderate to severe asthma 3132 • Randomised, double-blind, multi-centre, parallel group • Primary endpoint: time to first seve
Slide 69: Breztri/Trixeo asthma
Approved medicines Oncology Breztri/Trixeo (LAMA/LABA/ICS) Late-stage development Early development Asthma Trial Population Patients Design Endpoints Status CVRM Phase III Severe asthma 2200 • Randomised, double-blind, double-dummy, parallel group • Primary endpoint: change from • FPCD: Q1 2021 KALO
Slide 70: Breztri/Trixeo COPD
Approved medicines Oncology Breztri/Trixeo (LAMA/LABA/ICS) Late-stage development Early development COPD Trial Population Patients Design Endpoints Status CVRM Phase III Moderate to very severe 542 • Randomised, double-blind, 12-week (with an extension to • Primary endpoints: number of • FPCD: Q3 20
Slide 71: HFO1234ze next-generation propellant
Approved medicines Oncology HFO1234ze (next-generation propellant) Late-stage development Early development pMDI Trial Population Patients Design Endpoints Status CVRM Phase III Mucociliary clearance in 30 • Randomised, double-blind, multi-site, two-way crossover • Primary endpoint: change from • FP
Slide 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, open-label trial • Primary endpoint: proportion of • FPCD: Q2 2021 FJORD (newly diagnose
Slide 73: Fasenra nasal polyposis
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 74: Fasenra asthma and COPD
Approved medicines Oncology Fasenra (IL-5R mAb) Late-stage development Early development Severe, uncontrolled asthma and COPD Trial Population Patients Design Endpoints Status CVRM Phase III Severe, uncontrolled asthma 695 • Arm 1: Fasenra 30mg Q8W s.c. • Primary endpoint: annual asthma • FPCD: Q4 2
Slide 75: Saphnelo
Approved medicines Oncology Saphnelo (type I interferon receptor mAb) Late-stage development Early development Lupus (SLE/LN) Trial Population Patients Design Endpoints Status CVRM Phase III Moderate to severe SLE 360 • Arm 1: Saphnelo s.c. • Primary endpoint: BICLA at Week 52 • FPCD: Q3 2021 TULIP-
Slide 76: Tezspire CRSwNP, COPD and EoE
Approved medicines Oncology Tezspire (TSLP mAb) Late-stage development Early development CRSwNP, COPD and EoE Trial Population Patients Design Endpoints Status CVRM Phase III Severe chronic rhinosinusitis 400 • Arm 1: Tezspire s.c. • Co-primary endpoint: nasal polyp • FPCD: Q2 2021 WAYPOINT with nas
Slide 77: Tezspire severe asthma
Approved medicines Oncology Tezspire (TSLP mAb) Late-stage development Early development Severe, uncontrolled asthma Trial Population Patients Design Endpoints Status CVRM Phase III Severe asthma; age 12 to 80 1061 • Arm 1: Tezspire s.c. • Primary endpoint: annual asthma • FPCD: Q1 2018 NAVIGATOR ye
Slide 78: brazikumab Crohn's disease
Approved medicines Oncology brazikumab (IL-23 inhibitor) Late-stage development Early development Inflammatory bowel disease (Crohn’s disease, ulcerative colitis) Trial Population Patients Design Endpoints Status CVRM Phase III Crohn’s disease 161 • Open-label, long-term extension safety trial of br
Slide 79: brazikumab ulcerative colitis
Approved medicines Oncology brazikumab (IL-23 inhibitor) Late-stage development Early development Inflammatory bowel disease (Crohn’s disease, ulcerative colitis) Trial Population Patients Design Endpoints Status CVRM Phase II Ulcerative colitis 165 • Open-label, long-term extension safety trial of
Slide 80: tozorakimab acute respiratory failure
Approved medicines Oncology tozorakimab (IL-33 ligand mAb) Late-stage development Early development Acute respiratory failure Trial Population Patients Design Endpoints Status CVRM Phase III Adults hospitalised for viral 2902 • Randomised, double-blind, placebo-controlled, parallel • Primary endpoin
Slide 81: tozorakimab atopic dermatitis, asthma
Approved medicines Oncology tozorakimab (IL-33 ligand mAb) Late-stage development Early development Atopic dermatitis, asthma Trial Population Patients Design Endpoints Status CVRM Phase II Adults with uncontrolled 250 • Randomised, double-blind, placebo-controlled trial • Primary endpoint: change f
Slide 82: tozorakimab COPD
Approved medicines Oncology tozorakimab (IL-33 ligand mAb) Late-stage development Early development COPD Trial Population Patients Design Endpoints Status CVRM Phase III Adults with symptomatic 1272 • Randomised, double-blind, placebo-controlled, parallel- • Primary endpoint: annualised rate of • FP
Slide 83: Evusheld
Approved medicines Oncology Evusheld (AZD7442, tixagevimab + cilgavimab) Late-stage development Early development Prevention and treatment of COVID-19 Trial Population Patients Design Endpoints Status CVRM Phase II Adults and pediatric 251 • Randomised, open-label, dose-ranging to assess safety, • P
Slide 84: Beyfortus
Approved medicines Oncology Beyfortus (nirsevimab, RSV mAb-YTE) Late-stage development Early development Infection Trial Population Patients Design Endpoints Status CVRM Phase III Healthy infants (born 35 3000 • Randomised, double-blind, placebo-controlled • Primary endpoint: efficacy • Data readout
Slide 85: AZD3152
Approved medicines Oncology AZD3152 (SARS-CoV-2 LAAB) Late-stage development Early development Prevention of COVID-19 Trial Population Patients Design Endpoints Status CVRM Phase III Phase I: healthy adults; age 18 3200 • 2 parts (Phase I: sentinel safety cohort and Phase III: main • Primary endpoin
Slide 86: BioPharmaceuticals: early-stage development
BioPharmaceuticals: early-stage development
Slide 87: AZD0186
Approved medicines Oncology AZD0186 (oral sm GLP-1Ra) Late-stage development Early development Type 2 diabetes Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers 24 • Randomised, sequential assignment, sponsor-open, • Primary endpoints: safety and • FPCD: Q4 2022 NCT05
Slide 88: AZD0780
Approved medicines Oncology AZD0780 (PCSK9 inhibitor) Late-stage development Early development Dyslipidaemia Trial Population Patients Design Endpoints Status CVRM Phase I Healthy adults 132 • Randomised, placebo-controlled SAD/MAD trial • Primary endpoints: safety and • FPCD: Q2 2022 NCT05384262 to
Slide 89: AZD2373
Approved medicines Oncology AZD2373 Late-stage development Early development Chronic kidney disease Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers 30 • SAD dose escalation in 6 cohorts with • Primary endpoints: safety and • FPCD: Q1 2020 NCT04269031 6 volunteers re
Slide 90: AZD2693
Approved medicines Oncology AZD2693 (antisense oligonucleotide) Late-stage development Early development NASH Trial Population Patients Design Endpoints Status CVRM Phase IIb Non-cirrhotic non-alcoholic 232 • Randomised, double-blind, placebo-controlled, multi-centre • Primary endpoints: efficacy, s
Slide 91: AZD3427
Approved medicines Oncology AZD3427 (relaxin) Late-stage development Early development Heart failure Trial Population Patients Design Endpoints Status CVRM Phase II Heart failure and pulmonary 220 • Randomised, double-blind, placebo-controlled, multi- • Primary endpoint: change in PVR from • FPCD: Q
Slide 92: AZD5462
Approved medicines Oncology AZD5462 (relaxin) Late-stage development Early development Heart failure Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers 98 • Single-centre SAD and MAD • Primary endpoints: safety and • FPCD: Q4 2021 NCT04994106 (SAD/MAD) • Part A: SAD (8
Slide 93: AZD6234
Approved medicines Oncology AZD6234 (long-acting amylin) Late-stage development Early development Obesity with related comorbidities Trial Population Patients Design Endpoints Status CVRM Phase I Healthy participants who are 64 • SAD trial • Primary endpoint: safety • FPCD: Q4 2022 NCT05511025 overw
Slide 94: AZD7503
Approved medicines Oncology AZD7503 (antisense oligonucleotide) Late-stage development Early development NASH Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers 56 • SAD with 7 cohorts with 8 volunteers receiving AZD7503 • Primary endpoints: safety and • FPCD: Q4 2021
Slide 95: AZD9550
Approved medicines Oncology AZD9550 (GLP-1-glucagon agonist) Late-stage development Early development NASH Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers 64 • SAD trial • Primary endpoints: safety and • FPCD: Q2 2023 NCT05848440 tolerability • Data anticipated: H1
Slide 96: balcinrenone/dapagliflozin
Approved medicines Oncology balcinrenone/dapagliflozin (MR modulator + SGLT2 inhibitor) Late-stage development Early development Heart failure Trial Population Patients Design Endpoints Status CVRM Phase IIb Heart failure with chronic 500 • Randomised, stratified according to T2DM and eGFR (≥20 to •
Slide 97: MEDI6570
Approved medicines Oncology MEDI6570 Late-stage development Early development Cardiovascular Trial Population Patients Design Endpoints Status CVRM Phase IIb Post-myocardial infarction 400 • Evaluation of anti-inflammatory potential and effect on • Primary endpoints: safety and efficacy • FPCD: Q4 2
Slide 98: MEDI8367
Approved medicines Oncology MEDI8367 Late-stage development Early development Chronic kidney disease Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers 12 • SAD trial • Primary endpoints: safety and • FPCD: Q3 2020 NCT04365218 CKD • 6 cohorts tolerability • LPCD: Q4 20
Slide 99: mitiperstat cardiovascular disease
Approved medicines Oncology mitiperstat (MPO inhibitor) Late-stage development Early development Cardiovascular disease Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers 14 • Open-label • Primary endpoints: PK parameters • FPCD: Q1 2022 NCT05236543 • mitiperstat vs. m
Slide 100: mitiperstat NASH
Approved medicines Oncology mitiperstat (MPO inhibitor) Late-stage development Early development NASH Trial Population Patients Design Endpoints Status CVRM Phase II NASH 90 • Randomised, placebo-controlled, double-blind • Primary endpoints: safety, tolerability • FPCD: Q1 2023 COSMOS • Arm 1: 5mg m
Slide 101: zibotentan chronic kidney disease
Approved medicines Oncology zibotentan (endothelin receptor antagonist) Late-stage development Early development Chronic kidney disease Trial Population Patients Design Endpoints Status CVRM Phase IIb CKD 495 • Arm 1: zibotentan dose A + Farxiga 10mg QD • Primary endpoint: change in log- • FPCD: Q2
Slide 102: atuliflapon
Approved medicines Oncology atuliflapon (FLAP inhibitor) Late-stage development Early development Asthma Trial Population Patients Design Endpoints Status CVRM Phase II Patients with moderate-to- 1102 • Randomised, placebo-controlled, double-blind, multi- • Primary endpoint: time to first • FPCD: Q2
Slide 103: AZD4604
Approved medicines Oncology AZD4604 (inhaled JAK-1 inhibitor) Late-stage development Early development Asthma Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers and 137 • SAD/MAD/POM trial • Primary endpoints: safety and • FPCD: Q4 2021 NCT04769869 patients with mild a
Slide 104: AZD5055
Approved medicines Oncology AZD5055 (oral porcupine inhibitor) Late-stage development Early development Idiopathic pulmonary fibrosis (IPF) and other ILDs with progressive fibrosis Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers 90 • SAD/MAD trial • Primary endpoint
Slide 105: AZD6793
Approved medicines Oncology AZD6793 (IRAK4) Late-stage development Early development Inflammatory diseases Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers 133 • Single-blind, randomised, placebo-controlled trial to • Primary endpoints: safety and • FPCD: Q4 2022 NCT
Slide 106: AZD7798
Approved medicines Oncology AZD7798 (humanised mAb) Late-stage development Early development Crohn's disease Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers 64 • SAD • Primary endpoints: safety and • FPCD: Q3 2022 NCT05452304 • Arm1: AZD7798 tolerability • Data anti
Slide 107: AZD8630
Approved medicines Oncology AZD8630 (inhaled TSLP) Late-stage development Early development Asthma Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers and 232 • SAD and MAD trial • Primary endpoints: safety and • FPCD: Q1 2022 NCT05110976 patients with asthma tolerabili
Slide 108: elarekibep
Approved medicines Oncology elarekibep (AZD1402, IL-4 receptor alpha antagonist) Late-stage development Early development Asthma Trial Population Patients Design Endpoints Status CVRM Phase IIa Patients with asthma on 225 • Randomised, placebo-controlled, double-blinded, multi- • Primary endpoints (
Slide 109: mitiperstat COPD
Approved medicines Oncology mitiperstat (MPO inhibitor) Late-stage development Early development COPD Trial Population Patients Design Endpoints Status CVRM Phase II Moderate to severe COPD; 288 • Randomised, double-blind trial • Primary endpoint: time to first COPD • FPCD: Q1 2023 CRESCENDO age 40
Slide 110: AZD4041
Approved medicines Oncology AZD4041 (orexin 1 receptor antagonist) Late-stage development Early development Opioid use disorder Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers 36 • Randomised, double-blind MAD trial • Primary endpoints: safety and • FPCD: Q1 2022 NC
Slide 111: MEDI0618
Approved medicines Oncology MEDI0618 (PAR2 antagonist mAb) Late-stage development Early development Osteoarthritis pain, migraine prevention Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers 64 • SAD trial • Primary endpoints: safety and • FPCD: Q4 2019 NCT04198558 •
Slide 112: MEDI1341 multiple system atrophy
Approved medicines Oncology MEDI1341 (alpha-synuclein mAb) Late-stage development Early development Multiple system atrophy Trial Population Patients Design Endpoints Status CVRM Phase II Patients with diagnosis of 138 • Randomised, double-blind, placebo-controlled trial • Primary endpoints: efficac
Slide 113: MEDI1341 Parkinson's disease
Approved medicines Oncology MEDI1341 (alpha-synuclein mAb) Late-stage development Early development Parkinson’s disease Trial Population Patients Design Endpoints Status CVRM Phase I Parkinson's disease 25 • MAD trial • Primary endpoints: safety and • FPCD: Q3 2020 NCT04449484 • Arm 1: MEDI1341 i.v.
Slide 114: MEDI7352
Approved medicines Oncology MEDI7352 (NGF TNF bispecific mAb) Late-stage development Early development Osteoarthritis pain Trial Population Patients Design Endpoints Status CVRM Phase IIb Painful osteoarthritis of the 350 • MAD trial • Primary endpoint: dose response • FPCD: Q1 2021 NCT04675034 knee
Slide 115: Rare Disease: approved medicines and late-stage pipeline
Rare Disease: approved medicines and late-stage pipeline
Slide 116: Koselugo
Approved medicines Oncology Koselugo (selumetinib, MEK inhibitor) Late-stage development Early development Neurofibromatosis type 1, solid tumours Trial Population Patients Design Endpoints Status CVRM Phase III Adult age ≥18 years with NF1 who have 146 • Multi-centre, international trial with a par
Slide 117: Ultomiris haematology, nephrology
Approved medicines Oncology Ultomiris (anti-C5 mAb) Late-stage development Early development Haematology, nephrology Trial Population Patients Design Endpoints Status CVRM Phase III Thrombotic microangiopathy-associated 184 • Arm 1: Ultomiris Q8W • Primary endpoint: TMA response • FPCD: Q4 2020 ALXN
Slide 118: Ultomiris neurology
Approved medicines Oncology Ultomiris (anti-C5 mAb) Late-stage development Early development Neurology Trial Population Patients Design Endpoints Status CVRM Phase III Neuromyelitis optica spectrum disorder 58 • Arm 1: Ultomiris Q8W • Primary endpoint: time to first • FPCD: Q4 2019 ALXN1210-NMO-307
Slide 119: acoramidis
Approved medicines Oncology acoramidis (ALXN2060) Late-stage development Early development ATTR-CM Trial Population Patients Design Endpoints Status CVRM Phase III ATTR-CM 22 • Arm 1: 800mg acoramidis administered twice daily • Primary endpoint: change from baseline • FPCD: Q4 2020 ALXN2060-TAC-302
Slide 120: anselamimab
Approved medicines Oncology anselamimab (CAEL-101, fibril-reactive mAb) Late-stage development Early development AL amyloidosis Trial Population Patients Design Endpoints Status CVRM Phase III Mayo Stage IIIa amyloidosis 267 • Arm 1: anselamimab combined with SoC for PCD • Primary endpoint: time fro
Slide 121: danicopan
Approved medicines Oncology danicopan (ALXN2040, factor D inhibitor) Late-stage development Early development Haematology, ophthalmology Trial Population Patients Design Endpoints Status CVRM Phase III PNH with clinically significant EVH 84 • Arm 1: danicopan + C5 Inhibitor • Primary endpoint: chang
Slide 122: gefurulimab
Approved medicines Oncology gefurulimab (ALXN1720, anti-C5 bispecific mini-body) Late-stage development Early development Neurology, nephrology Trial Population Patients Design Endpoints Status CVRM Phase III Generalised myasthenia gravis 200 • Arm 1: weight-based maintenance treatment with • Primar
Slide 123: Rare Disease: early-stage development
Rare Disease: early-stage development
Slide 124: ALXN1850
Approved medicines Oncology ALXN1850 (next-generation asfotase alfa) Late-stage development Early development Hypophosphatasia Trial Population Patients Design Endpoints Status CVRM Phase I Hypophosphatasia 15 • Arm 1: ALXN1850, 3 cohorts at low, medium and high dosages • Primary endpoint: incidence
Slide 125: ALXN1910
Approved medicines Oncology ALXN1910 (next-generation TNSALP ERT) Late-stage development Early development Bone metabolism Trial Population Patients Design Endpoints Status CVRM Phase I Healthy adults 48 • Randomised, placebo-controlled SAD • Primary endpoint: safety • FPCD: Q2 2022 ALXN1910-HV-101
Slide 126: ALXN1920
Approved medicines Oncology ALXN1920 (kidney-targeted factor H fusion protein) Late-stage development Early development Nephrology Trial Population Patients Design Endpoints Status CVRM Phase I Healthy adults 48 • Randomised, double-blind, placebo-controlled, SAD • Primary endpoint: safety and • FPC
Slide 127: ALXN2030
Approved medicines Oncology ALXN2030 (siRNA targeting complement C3) Late-stage development Early development Nephrology Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers 48 • Randomised, placebo-controlled SAD • Primary endpoint: safety • FPCD: Q4 2022 ALXN2030-HV-10
Slide 128: ALXN2080
Approved medicines Oncology ALXN2080 (oral factor D inhibitor) Late-stage development Early development Complement-mediated disease Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers 100 • SAD/MAD trial • Primary endpoints: safety and • FPCD: Q3 2022 ALXN2080-HV-101 to
Slide 129: tarperprumig
Approved medicines Oncology tarperprumig (ALXN1820, anti-properdin) Late-stage development Early development Haematology Trial Population Patients Design Endpoints Status CVRM Phase IIa SCD 30 • Randomised, open-label • Primary endpoints: TEAEs and SAEs • FPCD: Q2 2023 PHOENIX (ALXN1820-SCD- • Arm 1
Slide 130: vemircopan
Approved medicines Oncology vemircopan (ALXN2050, factor D inhibitor) Late-stage development Early development Haematology, nephrology, neurology Trial Population Patients Design Endpoints Status CVRM Phase II PNH 28 • Arm 1: vemircopan monotherapy with groups including • Primary endpoint: change in
Slide 131: 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 132: 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 133: 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