Astrazeneca Plc · LSE:AZN
Astrazeneca — Q3 2024 Earnings Presentation
159 slides from Astrazeneca's earnings presentation, Nov 12, 2024
Slide 1: Clinical Trials Appendix
Clinical Trials Appendix Q3 2024 Results Update 12 November 2024
Slide 2: Pipeline at a glance
Pipeline 199 21 at a glance projects in our new molecular entities development pipeline (NME) in our late-stage pipeline Across five focus therapy areas: Oncology BioPharmaceuticals 139 25 CVRM | R&I | V&I new molecular entities regulatory approvals (NME) or major lifecycle in major markets manageme
Slide 3: Key upcoming pipeline catalysts: 2024 and 2025
Key upcoming pipeline catalysts: 2024 and 2025 Oncology BioPharmaceuticals Rare Disease H2 2024 H1 2025 H2 2025 Lynparza + Imfinzi – endometrial cancer (1L) (DUO-E) Tagrisso – EGFRm NSCLC (unresectable, Stg. III) (LAURA) (EU, JP, CN) Lynparza – prostate cancer (1L) (PROpel) (CN) Imfinzi – SCLC (limi
Slide 4: Clinical Trials Appendix: selected highlights
Clinical Trials Appendix: selected highlights BioPharmaceuticals Oncology Rare Disease Approved medicines: key LCM camizestrant (oral SERD) balcinrenone/dapagliflozin (MRM/SGLT2) registrational studies ongoing Dato-DXd (TROP2 ADC) ALXN2220 (TTR depleter) baxdrostat (aldosterone synthase inhibitor) N
Slide 5: Project movements since Q2 2024 update
Project movements since Q2 2024 update New to Phase I New to Phase II New to pivotal trial New to registration NME NME NME Life-cycle management AZD0022 AZD5004 AZD0486 SOUNDTRACK-F1 Calquence ECHO# KRas G12D inhibitor solid tumours oral GLP-1 receptor agonist chronic weight CD19-CD3 TCE follicular
Slide 6: Project movements since Q2 2024 update
Project movements since Q2 2024 update Removed from Phase I Removed from Phase II Removed from Phase III Approved/removed from registration Additional indication Life-cycle management Life-cycle management Life-cycle management sabestomig danicopan Fasenra ORCHID# Fasenra MANDARA PD-1/TIM3 bispecifi
Slide 7: Q3 2024 Oncology new molecular entity pipeline
Q3 2024 Oncology new molecular entity1 pipeline Phase I Phase II Phase III Under review 21 New Molecular Entities 15 New Molecular Entities 27 New Molecular Entities 2 New Molecular Entities AZD0120 AZD0022 AZD0171 + Imfinzi + CTx AZD0486 SOUNDTRACK-F1 AZD0901 CLARITY-Gastric01 datopotamab deruxteca
Slide 8: Q3 2024 Oncology lifecycle management pipeline
Q3 2024 Oncology lifecycle management1 pipeline Phase I Phase II Phase III 0 Projects 9 Projects 28 Projects Enhertu (platform) DESTINY-Breast07# Calquence + R-CHOP ESCALADE Calquence + venetoclax + obinutuzumab AMPLIFY# Enhertu DESTINY-Breast11# HER2 ADC HER2+ breast cancer BTK+R-CHOP 1L DLBCL BTK+
Slide 9: Q3 2024 BioPharmaceuticals new molecular entity pipeline
Q3 2024 BioPharmaceuticals new molecular entity1 pipeline Phase I Phase II Phase III 15 New Molecular Entities 20 New Molecular Entities 6 New Molecular Entities AZD0233 AZD1705 AZD0780 AZD2693 balcinrenone/dapagliflozin CX3CR1 dilated cardiomyopathy lipid lowering cardiovascular disease PCSK9 dysli
Slide 10: Q3 2024 BioPharmaceuticals life cycle management pipeline
Q3 2024 BioPharmaceuticals life cycle management1 pipeline Phase I Phase II Phase III Under review 0 Projects 1 Project 13 Projects 1 Project Tezspire COURSE# Wainua# Breztri/Trixeo (PT010) KALOS LOGOS roxadustat# TSLP chronic obstructive pulmonary disease LICA ATTR-cardiomyopathy LABA/LAMA/ICS asth
Slide 11: Q3 2024 Rare Disease pipeline
Q3 2024 Rare Disease pipeline1 Phase I Phase II Phase III Under review 4 Projects 3 Project 8 Projects 1 Project ALXN1910 vemircopan ALXN2220 DepleTTR-CM# acoramidis# next gen TNSALP ERT bone metabolism oral factor D immunoglobulin A nephropathy TTR depleter transthyretin amyloid cardiomyopathy oral
Slide 12: Designations in our pipeline
Designations in our pipeline 4 9 9 14 20 Accelerated approvals Breakthrough / PRIME1 / Sakigake2 Fast Track Priority Review Orphan Andexxa acute major bleed (US) Tezspire asthma NAVIGATOR (US) AZD3427 relaxin mimetic heart failure (US) Roxadustat chronic kidney disease (CN) Andexxa acute major bleed
Slide 13: Oncology: approved medicines and late-stage pipeline
Oncology: approved medicines and late-stage pipeline 13
Slide 14: 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 15: 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 16: 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 NSCLC, patients who progressed on an 531 • Open-label, biomarker-directed, multi-centre trial • Primary endpoint: ORR • FPCD: Q1 2018 H
Slide 17: 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 18: Lynparza: Imfinzi combinations
Approved medicines Oncology Lynparza (PARP inhibitor) Late-stage development Early development Imfinzi combinations Trial Population Patients Design Endpoints Status CVRM Phase III 1L advanced ovarian cancer 1407 • Non-tBRCAm (tumour BRCA) patients • Primary endpoint: PFS • FPCD: Q1 2019 DUO-O • Arm
Slide 19: Lynparza: Other cancer
Approved medicines Oncology Lynparza (PARP inhibitor) Late-stage development Early development Other cancer 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 • A
Slide 20: 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 21: 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- 927 • Randomised, open-label, parallel assignment • Primary endpoint: pCR
Slide 22: 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 23: 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 450 • Randomised, parallel group assignment • Primary endpoint: PFS • FPC
Slide 24: 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 to pursue 125 • Non-randomised single group assignment • Primary endpoint: ORR • FPCD:
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 Ib HER2-over-expressing, unresectable 244 • Non-randomised, parallel group assignment • Primary endpoint: safety
Slide 26: 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 27: Orpathys: NSCLC and other cancers
Approved medicines Oncology Orpathys (savolitinib, MET inhibitor) Late-stage development Early development NSCLC and other cancers Trial Population Patients Design Endpoints Status CVRM Phase III Locally advanced or metastatic NSCLC 163 • Single-arm trial • Primary endpoint: ORR • FPCD: Q3 2021 NCT0
Slide 28: Tagrisso: NSCLC
Approved medicines Oncology Tagrisso (highly-selective, irreversible EGFR inhibitor) Late-stage development Early development NSCLC Trial Population Patients Design Endpoints Status CVRM Phase III Maintenance therapy in patients with 216 • Arm 1: Tagrisso • Primary endpoint: PFS (BICR) • FPCD: Q4 20
Slide 29: Tagrisso: NSCLC, combinations
Approved medicines Oncology Tagrisso (highly-selective, irreversible EGFR inhibitor) Late-stage development Early development NSCLC, combinations Trial Population Patients Design Endpoints Status CVRM Phase III EGFR-mutated, MET-overexpressed 324 • Arm 1: Tagrisso + Orpathys • Primary enpoint: PFS •
Slide 30: Truqap: Breast and prostate cancer
Approved medicines Oncology Truqap (capivasertib, AKT inhibitor) Late-stage development Early development Breast cancer and prostate cancer Trial Population Patients Design Endpoints Status CVRM Phase III Locally advanced or metastatic TNBC 924 • Double-blind, randomised, comparative trial • Primary
Slide 31: datopotamab deruxtecan: Breast cancer
Approved medicines Oncology datopotamab deruxtecan (TROP2 ADC) Late-stage development Early development Breast cancer Trial Population Patients Design Endpoints Status CVRM Phase III Inoperable or metastatic HR+ HER2- 732 • Open-label, randomised trial • Primary endpoints: PFS (BICR) and OS • FPCD:
Slide 32: 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 33: 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 Patients with advanced or metastatic 630 • Phase III, open-label, sponsor blind, randomized study of • Dual Primary • FPCD: Q
Slide 34: 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 35: 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, 582 • Sub-study 1 (endometrial cancer); • Primary endpoints: ORR and safety • FPCD
Slide 36: datopotamab deruxtecan: Other cancers
Approved medicines Oncology datopotamab deruxtecan (TROP2 ADC) Late-stage development Early development Other cancers Trial Population Patients Design Endpoints Status CVRM Phase I/II NSCLC and TNBC and other solid 119 • Single-arm, multi-cohort trial with no blinding • Primary endpoint: ORR • FPCD:
Slide 37: AZD0486: Haematologic malignancies
Approved medicines Oncology AZD0486 (CD19/CD3 next-generation bispecific T-cell engager) Late-stage development Early development Haematologic malignancies Trial Population Patients Design Endpoints Status CVRM Phase III Previously Untreated Follicular 1005 • Multicentre, Randomised, Open-label Stud
Slide 38: AZD0901: Solid tumours
Approved medicines Oncology AZD0901 (CLDN18.2 MMAE ADC) Late-stage development Early development Solid tumours Trial Population Patients Design Endpoints Status CVRM Phase III 2L+ advanced or metastatic gastric or 589 • Multi-centre, open-label, sponsor-blinded, randomised trial • Primary endpoints:
Slide 39: camizestrant: Breast cancer
Approved medicines Oncology camizestrant (AZD9833, next-generation oral SERD) Late-stage development Early development Breast cancer Trial Population Patients Design Endpoints Status CVRM Phase III HR+ HER2-negative advanced breast 1370 • Randomised, double-blind, comparative trial • Primary endpoin
Slide 40: camizestrant: Breast cancer
Approved medicines Oncology camizestrant (AZD9833, next-generation oral SERD) Late-stage development Early development Breast cancer Trial Population Patients Design Endpoints Status CVRM Phase II HR+ advanced breast cancer 240 • Randomised, open-label, parallel-group, multi-centre trial • Primary e
Slide 41: ceralasertib: Multiple cancers
Approved medicines Oncology ceralasertib (AZD6738, ATR inhibitor) Late-stage development Early development Multiple cancers Trial Population Patients Design Endpoints Status CVRM Phase III Post-IO NSCLC 594 • Double-arm randomised: • Primary endpoint: OS • FPCD: Q4 2022 LATIFY • Arm 1: ceralasertib
Slide 42: rilvegostomig: Solid tumours
Approved medicines Oncology rilvegostomig (AZD2936, PD-1/TIGIT bispecific mAb) Late-stage development Early development Solid tumours Trial Population Patients Design Endpoints Status CVRM Phase III BTC with curative intent 750 • Randomised, Double-Blind, Placebo-Controlled, Multicenter • Primary en
Slide 43: rilvegostomig: Solid tumours
Approved medicines Oncology rilvegostomig (AZD2936, PD-1/TIGIT bispecific mAb) Late-stage development Early development Solid tumours Trial Population Patients Design Endpoints Status CVRM Phase IIb Gastric cancer 240 • Open-label gastric platform trial • Primary endpoints: safety and efficacy • FPC
Slide 44: saruparib: Solid tumours
Approved medicines Oncology saruparib (AZD5305, PARP1 inhibitor) Late-stage development Early development Solid tumours Trial Population Patients Design Endpoints Status CVRM Phase III HRRm and non-HRRm mCSPC 1800 • Randomised, placebo-controlled trial • Primary endpoint: rPFS • FPCD: Q4 2023 EvoPAR
Slide 45: saruparib: Solid tumours
Approved medicines Oncology saruparib (AZD5305, PARP1 inhibitor) Late-stage development Early development Solid tumours Trial Population Patients Design Endpoints Status CVRM Phase I Locally advanced, unresectable or 16 • Part A: to assess the effect of multiple doses of itraconazole • Primary endpo
Slide 46: volrustomig: Solid tumours
Approved medicines Oncology volrustomig (MEDI5752, PD-1/CTLA-4 bispecific mAb) Late-stage development Early development Solid tumours Trial Population Patients Design Endpoints Status CVRM Phase III High-risk locally advanced cervical 1000 • Randomised, double-blind, placebo-controlled, multi-centre
Slide 47: volrustomig: Solid tumours
Approved medicines Oncology volrustomig (MEDI5752, PD-1/CTLA-4 bispecific mAb) Late-stage development Early development Solid tumours Trial Population Patients Design Endpoints Status CVRM Phase Ib Advanced renal cell carcinoma 179 • Open-label, dose escalation and dose expansion trial • Primary end
Slide 48: Oncology: early-stage development
Oncology: early-stage development 48
Slide 49: AZD0022: Solid tumours
Approved medicines Oncology AZD0022 (KRASG12D) Late-stage development Early development Solid tumours Trial Population Patients Design Endpoints Status CVRM Phase I/II Metastatic or locally advanced 430 • Open-label, multi-centre study • Primary endpoints: safety and • FPCD: Q4 2024 ALAFOSS-01 color
Slide 50: AZD0120: Blood cancers
Approved medicines Oncology AZD0120 (GC012F, autologous anti-CD19 and anti-BCMA CAR-T) Late-stage development Early development Blood cancers Trial Population Patients Design Endpoints Status CVRM Phase I/II Relapsed/refractory multiple myeloma 68 • Open-label, single-arm, multi-centre trial • Prima
Slide 51: AZD0171: Cancer
Approved medicines Oncology AZD0171 (anti-LIF mAb) Late-stage development Early development Cancer Trial Population Patients Design Endpoints Status CVRM Phase II 1L metastatic pancreatic ductal 126 • Open-label, non-randomised trial • Primary endpoints: safety, OS at 12 • FPCD: Q1 2022 NCT04999969
Slide 52: AZD0305: Blood cancers
Approved medicines Oncology AZD0305 (GPRC5D ADC) Late-stage development Early development Blood cancers Trial Population Patients Design Endpoints Status CVRM Phase I/II R/R multiple myeloma 84 • Open-label, dose escalation and dose expansion trial • Primary endpoints: occurrence of • FPCD: Q4 2023
Slide 53: AZD0486: Haematologic malignancies
Approved medicines Oncology AZD0486 (CD19/CD3 next-generation bispecific T-cell engager) Late-stage development Early development Haematologic malignancies Trial Population Patients Design Endpoints Status CVRM Phase I/II R/R B-ALL 120 • Multi-centre, open-label, single-arm dose escalation and • Pri
Slide 54: AZD0754: Solid tumours
Approved medicines Oncology AZD0754 (STEAP2 dnTGFbetaRII armoured CAR-T) Late-stage development Early development Solid tumours Trial Population Patients Design Endpoints Status CVRM Phase I/II Metastatic castration resistance 60 • Open-label, single-arm, multi-centre trial with dose • Primary endpo
Slide 55: AZD1390: Cancer
Approved medicines Oncology AZD1390 (ATM inhibitor) Late-stage development Early development Cancer Trial Population Patients Design Endpoints Status CVRM Phase I Recurrent glioblastoma eligible for re- 165 • Open-label trial • Primary endpoints: safety, tolerability • FPCD: Q2 2018 NCT03423628 irra
Slide 56: AZD2068: Solid tumours
Approved medicines Oncology AZD2068 (FPI-2068, EGFR cMET bispecific mAb) Late-stage development Early development Solid tumours Trial Population Patients Design Endpoints Status CVRM Phase I Advanced solid tumours 110 • Multicentre, open-label dose escalation trial • Primary endpoints: safety and •
Slide 57: AZD3470: Solid tumours and blood cancer
Approved medicines Oncology AZD3470 (PRMT5) Late-stage development Early development Solid tumours and blood cancer Trial Population Patients Design Endpoints Status CVRM Phase I MTAP-deficient advanced solid tumours 210 • Arm 1: AZD3470 • Primary endpoints: safety and • FPCD: Q1 2024 PRIMROSE • Glo
Slide 58: AZD5335: Solid tumours
Approved medicines Oncology AZD5335 (anti-FRα TOP1i ADC) Late-stage development Early development Solid tumours, ovarian cancer, lung cancer Trial Population Patients Design Endpoints Status CVRM Phase I/II Advanced solid tumour malignancies 150 • Module 1: AZD5335 monotherapy • Primary endpoints: s
Slide 59: AZD5492: Blood cancers
Approved medicines Oncology AZD5492 (CD20 TITAN TCE) Late-stage development Early development Blood cancers Trial Population Patients Design Endpoints Status CVRM Phase I CLL/MCL/LBCL/FL 176 • Module 1: AZD5492 Monotherapy • -Primary: Safety and tolerability • Data anticipated: >2025 TITANIUM • AZD5
Slide 60: AZD5851: Gastrointestinal cancer
Approved medicines Oncology AZD5851 (armoured TGFbetaRIIDN GPC3 CAR-T) Late-stage development Early development Gastrointestinal cancer Trial Population Patients Design Endpoints Status CVRM Phase I/II GPC3-positive advanced/recurrent HCC 93 • Open-label, single-arm, multi-centre trial with dose • P
Slide 61: AZD5863: Solid tumours
Approved medicines Oncology AZD5863 (CLDN18.2 x CD3 bispecific antibody) Late-stage development Early development Solid tumours Trial Population Patients Design Endpoints Status CVRM Phase I Advanced or metastatic solid tumours 200 • Part A: dose escalation phase to determine the safety, • Primary e
Slide 62: AZD6422: Solid tumours
Approved medicines Oncology AZD6422 (CLDN18.2 CAR-T) Late-stage development Early development Solid tumours Trial Population Patients Design Endpoints Status CVRM Phase I Advanced or metastatic CLDN18.2- 96 • Open-label trial assessing anti-CLDN18.2 CAR-T cell therapy • Primary endpoints: incidence
Slide 63: AZD8205: Solid tumours
Approved medicines Oncology AZD8205 (B7H4 ADC) Late-stage development Early development Solid tumours Trial Population Patients Design Endpoints Status CVRM Phase I/II Breast cancer, BTC, ovarian cancer, 340 • Open-label dose escalation and expansion trial • Primary endpoints: AE, SAE, DLTs, • FPCD:
Slide 64: AZD8421: Breast cancer
Approved medicines Oncology AZD8421 (CDK2 inhibitor) Late-stage development Early development Breast cancer Trial Population Patients Design Endpoints Status CVRM Phase I/II ER+ HER2-negative advanced breast 204 • Module 1: AZD8421 • Primary endpoints: safety and • FPCD: Q4 2023 CYCAD-1 cancer • Mod
Slide 65: AZD9574: Solid tumours
Approved medicines Oncology AZD9574 (PARP1-sel BBB inhibitor) Late-stage development Early development Solid tumours Trial Population Patients Design Endpoints Status CVRM Phase I/IIa Advanced solid malignancies 490 • Modular, open-label, multi-centre dose escalation and • Primary endpoints: safety
Slide 66: AZD9592: Lung cancer
Approved medicines Oncology AZD9592 (EGFR-cMET TOP1i ADC) Late-stage development Early development Lung cancer Trial Population Patients Design Endpoints Status CVRM Phase I Advanced solid tumours including 162 • Escalation phase, open-label, multi-centre trial • Primary endpoints (escalation): safe
Slide 67: AZD9829: Blood cancers
Approved medicines Oncology AZD9829 (CD123 TOP1i ADC) Late-stage development Early development Blood cancers Trial Population Patients Design Endpoints Status CVRM Phase I/II CD123-positive haematological 60 • Open-label, multicentre trial • Primary endpoints: safety and • FPCD: Q1 2024 ADC123 malig
Slide 68: FPI-2265: Prostate cancer
Approved medicines Oncology FPI-2265 (PSMA radioconjugate) Late-stage development Early development Prostate cancer Trial Population Patients Design Endpoints Status CVRM Phase II/III PSMA-positive mCRPC previously 60 • Open-label, randomised, multi-centre trial • Primary endpoints: PSA50, safety •
Slide 69: IPH5201: Solid tumours
Approved medicines Oncology IPH5201 (CD39 mAb) Late-stage development Early development Solid tumours Trial Population Patients Design Endpoints Status CVRM Phase I Advanced solid tumours 57 • Open-label, dose escalation trial to determine MTD of • Primary endpoints: AE, SAE and DLT • FPCD: Q1 2020
Slide 70: NT-112: Solid tumours
Approved medicines Oncology NT-112 (KRAS G12D specific TCR) Late-stage development Early development Solid tumours Trial Population Patients Design Endpoints Status CVRM Phase I Unresectable, advanced and/or 24 • Open-label, single-arm, multi-centre trial with dose • Primary endpoints: incidence of
Slide 71: NT-125: Solid tumours
Approved medicines Oncology NT-125 (autologous, multi-specific neoantigen-targeting TCR-T) Late-stage development Early development Solid tumours Trial Population Patients Design Endpoints Status CVRM Phase I Adults with recurrent or metastatic 42 • Open-label, single-arm, single-centre trial with d
Slide 72: NT-175: Multiple cancers
Approved medicines Oncology NT-175 (TP53-armored TCR) Late-stage development Early development Multiple cancers Trial Population Patients Design Endpoints Status CVRM Phase I Unresectable, advanced, and/or 24 • Open-label, single-arm, multi-centre trial with dose • Primary endpoint: incidence of DL
Slide 73: oleclumab: Solid tumours
Approved medicines Oncology oleclumab (CD73 mAb) Late-stage development Early development Solid tumours Trial Population Patients Design Endpoints Status CVRM Phase Ib/II Pancreatic 1L and 2L with prior 339 • Arm A1: gemcitabine and nab paclitaxel i.v. • Primary endpoints: safety and anti- • FPCD: Q
Slide 74: sabestomig: Cancer
Approved medicines Oncology sabestomig (AZD7789, PD-1/TIM3 bispecific mAb) Late-stage development Early development Cancer Trial Population Patients Design Endpoints Status CVRM Phase I/IIa NSCLC, gastric cancer and other 192 • Open-label, non-randomised dose escalation and dose • Primary endpoints:
Slide 75: BioPharmaceuticals: approved medicines and late-stage development
BioPharmaceuticals: approved medicines and late-stage development 75
Slide 76: Andexxa: Haematology
Approved medicines Oncology Andexxa (anti-factor Xa reversal) Late-stage development Early development Haematology Trial Population Patients Design Endpoints Status CVRM Phase IV Acute intracranial 1200 • Arm 1: Andexxa • Primary endpoint: proportion of • FPCD: Q2 2019 I8-513 (post-launch) haemorrha
Slide 77: Wainua: ATTR
Approved medicines Oncology Wainua (eplontersen, ligand-conjugated antisense) Late-stage development Early development ATTR Trial Population Patients Design Endpoints Status CVRM Phase III Hereditary or wild-type 1438 • Arm 1: Wainua s.c. • Primary endpoints: composite • FPCD: Q1 2020 CARDIO-TTRansf
Slide 78: balcinrenone/dapagliflozin: Heart failure, CKD
Approved medicines Oncology balcinrenone/dapagliflozin (MR modulator + SGLT2 inhibitor) Late-stage development Early development Heart failure, CKD Trial Population Patients Design Endpoints Status CVRM Phase III Heart failure patients with 4800 • Randomised, double-blind, parallel-group, double-dum
Slide 79: baxdrostat: Hypertension
Approved medicines Oncology baxdrostat (selective aldosterone synthase inhibitor) Late-stage development Early development Hypertension Trial Population Patients Design Endpoints Status CVRM Phase III Patients with uncontrolled 720 • Arm 1: baxdrostat 1mg QD • Primary endpoint: effect of baxdrostat
Slide 80: baxdrostat: Hypertension
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 uncontrolled 175 • Arm 1: baxdrostat 2mg QD • Primary endpoints: safety and • FPCD: Q2
Slide 81: baxdrostat/dapagliflozin: CKD
Approved medicines Oncology baxdrostat/dapagliflozin Late-stage development Early development CKD Trial Population Patients Design Endpoints Status CVRM Phase III CKD and high blood pressure 2500 • Arm 1: baxdrostat/dapagliflozin QD • Primary endpoint: change from • FPCD: Q2 2024 BaxDuo-Arctic • Arm
Slide 82: zibotentan/dapagliflozin: Chronic kidney disease
Approved medicines Oncology zibotentan/dapagliflozin (ETA receptor antagonist/SGLT2 inhibitor) Late-stage development Early development Chronic kidney disease Trial Population Patients Design Endpoints Status CVRM Phase III CKD and high proteinuria 1500 • Randomised, parallel, multi-centre, double-b
Slide 83: zibotentan/dapagliflozin: Liver cirrhosis
Approved medicines Oncology zibotentan/dapagliflozin (ETA receptor antagonist/SGLT2 inhibitor) Late-stage development Early development Liver cirrhosis Trial Population Patients Design Endpoints Status CVRM Phase II Part A: participants with 195 • Phase IIa/b multi-centre, randomised, double-blind,
Slide 84: Airsupra: Asthma
Approved medicines Oncology Airsupra (PT027, SABA/ICS, pMDI) Late-stage development Early development Asthma Trial Population Patients Design Endpoints Status CVRM Phase IIIb Adults and adolescents with 2517 • Randomised, double-blind, multi-centre, parallel-group, • Primary endpoint: time to first
Slide 85: Airsupra: Asthma
Approved medicines Oncology Airsupra (PT027, SABA/ICS, pMDI) Late-stage development Early development Asthma Trial Population Patients Design Endpoints Status CVRM Phase III Mild to moderate asthma 1001 • Randomised, double-blind, multi-centre and parallel-group • Dual primary endpoints: change from
Slide 86: Breztri, Trixeo: Asthma
Approved medicines Oncology Breztri, Trixeo (LAMA/LABA/ICS) Late-stage development Early development Asthma Trial Population Patients Design Endpoints Status CVRM Phase III Severe asthma 2200 • Randomised, double-blind, double-dummy, parallel group • Primary endpoint: change from • FPCD: Q1 2021 KAL
Slide 87: 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 COPD 180 • Randomised, double-blind, three-treatment, three-period, • Primary endpoint: change from • FPCD: Q4 2023 ATHLOS cross-
Slide 88: Fasenra: Nasal polyposis and eosinophilic diseases
Approved medicines Oncology Fasenra (IL-5R mAb) Late-stage development Early development Nasal polyposis and other eosinophilic diseases Trial Population Patients Design Endpoints Status CVRM Phase III Patients with severe bilateral 413 • Arm 1: Fasenra 30mg Q8W s.c. • Primary endpoint: effect of Fa
Slide 89: Fasenra: Severe 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 90: Saphnelo: Lupus
Approved medicines Oncology Saphnelo (type I interferon receptor mAb) Late-stage development Early development Lupus (SLE/LN) Trial Population Patients Design Endpoints Status CVRM Phase III Moderate to severe SLE 360 • Arm 1: Saphnelo s.c. • Primary endpoint: BICLA at Week 52 • FPCD: Q3 2021 TULIP-
Slide 91: Saphnelo: Sclerosis and other myopathies
Approved medicines Oncology Saphnelo (type I interferon receptor mAb) Late-stage development Early development Sclerosis and other myopathies Trial Population Patients Design Endpoints Status CVRM Phase III Systemic sclerosis 306 • Arm 1: Saphnelo s.c. • Primary endpoint: CRISS-25 at • FPCD: Q4 2023
Slide 92: Tezspire: CRSwNP, COPD and EoE
Approved medicines Oncology Tezspire (TSLP mAb) Late-stage development Early development CRSwNP, COPD and EoE Trial Population Patients Design Endpoints Status CVRM Phase III Severe chronic rhinosinusitis 416 • Arm 1: Tezspire s.c. • Co-primary endpoint: nasal polyp • FPCD: Q2 2021 WAYPOINT with nas
Slide 93: Tezspire: Severe, uncontrolled asthma
Approved medicines Oncology Tezspire (TSLP mAb) Late-stage development Early development Severe, uncontrolled asthma Trial Population Patients Design Endpoints Status CVRM Phase III Severe asthma; age 12 to 80 1061 • Arm 1: Tezspire s.c. • Primary endpoint: annual asthma • FPCD: Q1 2018 NAVIGATOR ye
Slide 94: HFO1234ze: pMDI
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 95: HFO1234ze: pMDI
Approved medicines Oncology HFO1234ze (next-generation propellant) Late-stage development Early development pMDI Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers 108 • Randomised, double-blind, single-dose, single-centre, • Primary endpoint: AUCinf, AUClast • FPCD: Q
Slide 96: 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 1099 • Randomised, double-blind, placebo-controlled, parallel- • Primary endpoint: annualised rate of • FP
Slide 97: tozorakimab: COPD
Approved medicines Oncology tozorakimab (IL-33 ligand mAb) Late-stage development Early development COPD Trial Population Patients Design Endpoints Status CVRM Phase III Adults with symptomatic 1240 • Randomised, double-blind, placebo-controlled, parallel • Primary endpoint: annualised rate of • FPC
Slide 98: tozorakimab: Severe viral LRTD, asthma
Approved medicines Oncology tozorakimab (IL-33 ligand mAb) Late-stage development Early development Severe viral LRTD, asthma Trial Population Patients Design Endpoints Status CVRM Phase III Adults hospitalised for viral 2870 • Randomised, double-blind, placebo-controlled, parallel • Primary endpoin
Slide 99: tozorakimab: Severe viral LRTD, asthma
Approved medicines Oncology tozorakimab (IL-33 ligand mAb) Late-stage development Early development Severe viral LRTD, 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 100: Beyfortus: Infection
Approved medicines Oncology Beyfortus (nirsevimab, RSV mAb-YTE) Late-stage development Early development Infection Trial Population Patients Design Endpoints Status CVRM Phase III Healthy infants (born 29 800 • Randomised, double-blind, placebo-controlled • Primary endpoints efficacy • FPCD: Q4 2021
Slide 101: Evusheld: COVID-19
Approved medicines Oncology Evusheld (AZD7442, tixagevimab + cilgavimab) Late-stage development Early development COVID-19 Trial Population Patients Design Endpoints Status CVRM Phase II Adults and pediatric 251 • Randomised, open-label, dose-ranging to assess safety, • Primary endpoints: safety and
Slide 102: sipavibart: COVID-19
Approved medicines Oncology sipavibart (AZD3152, SARS-CoV-2 LAAB) Late-stage development Early development COVID-19 Trial Population Patients Design Endpoints Status CVRM Phase III Phase I: healthy adults; age 18 3200 • 2 parts (Phase I: sentinel safety cohort and Phase III: main • Primary endpoints
Slide 103: BioPharmaceuticals: early-stage development
BioPharmaceuticals: early-stage development 103
Slide 104: AZD0233: Dilated cardiomyopathy
Approved medicines Oncology AZD0233 (oral CX3CR1) Late-stage development Early development Dilated cardiomyopathy Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers 96 • Randomised, SAD/MAD dose escalating trial • Primary endpoint: safety and • FPCD: Q2 2024 NCT0638146
Slide 105: AZD0780: Dyslipidaemia
Approved medicines Oncology AZD0780 (PCSK9 inhibitor) Late-stage development Early development Dyslipidaemia Trial Population Patients Design Endpoints Status CVRM Phase II Dyslipidaemia 428 • Randomised trial with equal distribution across five parallel • Primary endpoint: percent change in • FPCD:
Slide 106: AZD1705: Dyslipidaemia
Approved medicines Oncology AZD1705 (Angptl3 inhibitor) Late-stage development Early development Dyslipidaemia Trial Population Patients Design Endpoints Status CVRM Phase I Dyslipidaemia 112 • Part A: single dose of AZD1705 with an in-clinic period of 3 • Primary endpoints: AEs and SAEs • FPCD: Q1
Slide 107: AZD2373: Chronic kidney disease
Approved medicines Oncology AZD2373 (PNPLA3 ASO) 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
Slide 108: AZD2389: MASH
Approved medicines Oncology AZD2389 (anti-fibrotic mechanism) Late-stage development Early development MASH Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers 104 • Randomised, placebo-controlled SAD/MAD trial • Primary endpoints: safety and • FPCD: Q4 2023 NCT06138795
Slide 109: AZD2693: MASH
Approved medicines Oncology AZD2693 (antisense oligonucleotide) Late-stage development Early development MASH Trial Population Patients Design Endpoints Status CVRM Phase IIb NASH with fibrosis 180 • Randomised, double-blind, placebo-controlled, multi-centre • Primary endpoints: efficacy, safety • F
Slide 110: AZD3427: Heart failure
Approved medicines Oncology AZD3427 (relaxin) Late-stage development Early development Heart failure Trial Population Patients Design Endpoints Status CVRM Phase II Heart failure and pulmonary 220 • Randomised, double-blind, placebo-controlled, multi-centre • Primary endpoint: change in PVR from • F
Slide 111: AZD4144: Cardiorenal disease
Approved medicines Oncology AZD4144 (inflammation modulator) Late-stage development Early development Cardiorenal disease Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers 96 • Randomised, single-blind, placebo-controlled, SAD/MAD • Primary endpoints: safety and • FPC
Slide 112: AZD5004: Type 2 diabetes, obesity
Approved medicines Oncology AZD5004 (oral GLP-1 RA) Late-stage development Early development Type 2 diabetes, obesity Trial Population Patients Design Endpoints Status CVRM Phase IIb Type 2 diabetes 384 • Arm 1: AZD5004 tablet • Primary endpoint: change in HbA1c • FPCD: Q4 2024 SOLSTICE • Arm 2: AZD
Slide 113: AZD5462: Heart failure
Approved medicines Oncology AZD5462 (relaxin) Late-stage development Early development Heart failure Trial Population Patients Design Endpoints Status CVRM Phase IIb Stable patients with chronic 360 • Two cohort, randomised, double-blind, placebo-controlled, • Primary endpoint: change in heart • FPC
Slide 114: AZD6234: Obesity with related co-morbidities
Approved medicines Oncology AZD6234 (long-acting amylin) Late-stage development Early development Obesity with related co-morbidities Trial Population Patients Design Endpoints Status CVRM Phase II Participants living with 231 • Randomised, double-blind, placebo-controlled trial • Primary endpoints:
Slide 115: AZD9550: MASH
Approved medicines Oncology AZD9550 (GLP-1-glucagon agonist) Late-stage development Early development MASH Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers 64 • SAD trial • Primary endpoint: safety and • FPCD: Q2 2023 NCT05848440 tolerability • LPCD: Q4 2023 • Data r
Slide 116: mitiperstat: Cardiovascular disease
Approved medicines Oncology mitiperstat (MPO inhibitor) Late-stage development Early development Cardiovascular disease Trial Population Patients Design Endpoints Status CVRM Phase IIb HFpEF 711 • Randomised, double-blind • Primary endpoints: safety and efficacy • FPCD: Q3 2021 ENDEAVOR • Arm 1: 2.5
Slide 117: mitiperstat: MASH
Approved medicines Oncology mitiperstat (MPO inhibitor) Late-stage development Early development MASH Trial Population Patients Design Endpoints Status CVRM Phase II NASH 90 • Randomised, placebo-controlled, double-blind • Primary endpoints: safety, tolerability • FPCD: Q1 2023 COSMOS • Arm 1: 5mg m
Slide 118: atuliflapon: Asthma
Approved medicines Oncology atuliflapon (FLAP inhibitor) Late-stage development Early development Asthma Trial Population Patients Design Endpoints Status CVRM Phase IIa Patients with moderate-to- 666 • Randomised, placebo-controlled, double-blind, multi-centre • Primary endpoint: time to first • FP
Slide 119: AZD0120: Lupus (SLE)
Approved medicines Oncology AZD0120 (GC012F, autologous anti-CD19 and anti-BCMA CAR-T) Late-stage development Early development Lupus (SLE) Trial Population Patients Design Endpoints Status CVRM Phase I/II Refractory Systemic Lupus 21 • Single-arm, open label, multicentre • Phase I primary endpoint:
Slide 120: AZD0292: Bronchiectasis
Approved medicines Oncology AZD0292 (Psl-PcrV N3Y-bispecific mAb) Late-stage development Early development Bronchiectasis Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers 32 • Randomised, single-blind, placebo-controlled trial • Primary endpoints: AEs and • FPCD: Q2
Slide 121: AZD1163: Rheumatoid arthritis
Approved medicines Oncology AZD1163 (bispecific antibody) Late-stage development Early development Rheumatoid arthritis Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers 108 • Randomised, double-blind, placebo-controlled SAD/MAD • Primary endpoint: number of • FPCD: Q
Slide 122: AZD4604: Asthma
Approved medicines Oncology AZD4604 (inhaled JAK-1 inhibitor) Late-stage development Early development Asthma Trial Population Patients Design Endpoints Status CVRM Phase IIa Moderate-to-severe asthma 320 • Multicentre, randomised, placebo-controlled, double-blind, • Primary endpoint: time to first
Slide 123: AZD6793: Inflammatory diseases
Approved medicines Oncology AZD6793 (IRAK4) Late-stage development Early development Inflammatory diseases Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers 133 • Single-blind, randomised, placebo-controlled trial • Primary endpoints: safety and • FPCD: Q4 2022 NCT056
Slide 124: AZD6912: Rheumatoid arthritis
Approved medicines Oncology AZD6912 (siRNA) Late-stage development Early development Rheumatoid arthritis Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers 64 • Randomised, double-blind, placebo-controlled SAD trial • Primary endpoint: incidence of AEs • FPCD: Q4 2023
Slide 125: AZD7798: Crohn's disease
Approved medicines Oncology AZD7798 (humanised mAb) Late-stage development Early development Crohn's disease Trial Population Patients Design Endpoints Status CVRM Phase IIa Moderate to severe Crohn’s 192 • Randomised, double-blind, placebo-controlled trial • Primary endpoint: Crohn's Disease • FPCD
Slide 126: AZD8630: Asthma
Approved medicines Oncology AZD8630 (inhaled TSLP) Late-stage development Early development Asthma Trial Population Patients Design Endpoints Status CVRM Phase II Adults with uncontrolled 516 • Randomised, placebo-controlled, double-blind, dose range- • Primary endpoint: time to first • FPCD: Q3 202
Slide 127: AZD8965: IPF
Approved medicines Oncology AZD8965 (arginase enzyme inhibitor) Late-stage development Early development IPF Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers 163 • SAD/MAD placebo-controlled • Primary endpoint: safety and • Data anticipated: H2 2025 NCT06502379 toler
Slide 128: 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; 381 • Randomised, double-blind trial • Primary endpoint: time to first COPD • FPCD: Q1 2023 CRESCENDO age 40
Slide 129: AZD4041: Opioid use disorder
Approved medicines Oncology AZD4041 (orexin 1 receptor antagonist) Late-stage development Early development Opioid use disorder Trial Population Patients Design Endpoints Status CVRM Phase II Healthy volunteers and 100 • Part 1: open label, fixed sequence trial of AZD4041 and • Primary endpoints (Pa
Slide 130: MEDI0618: Migraine prevention
Approved medicines Oncology MEDI0618 (PAR2 antagonist mAb) Late-stage development Early development Migraine prevention Trial Population Patients Design Endpoints Status CVRM Phase II Cohort of participants failed > 408 • Arm 1: CGRP-N MEDI0618 Dose A • Primary endpoint: change in number • FPCD: Q4
Slide 131: MEDI0618: Osteoarthritis pain, migraine prevention
Approved medicines Oncology MEDI0618 (PAR2 antagonist mAb) Late-stage development Early development Osteoarthritis pain, migraine prevention Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers 112 • Randomised, double-blind, placebo-controlled MAD trial • Primary endpoi
Slide 132: 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 133: 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 134: MEDI7352: Osteoarthritis pain
Approved medicines Oncology MEDI7352 (NGF TNF bispecific mAb) Late-stage development Early development Osteoarthritis pain Trial Population Patients Design Endpoints Status CVRM Phase IIb Painful osteoarthritis of the 350 • MAD trial • Primary endpoint: dose response • FPCD: Q1 2021 NCT04675034 knee
Slide 135: mRNA VLP vaccine: COVID-19
Approved medicines Oncology mRNA VLP vaccine Late-stage development Early development COVID-19 Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers ≥18+ with 240 • Arm 1: dose 1 via i.m. injection AZD9838 in 18-64-year-olds • Primary endpoints: safety as measured • FPCD:
Slide 136: AZD5148: Clostridium difficile
Approved medicines Oncology AZD5148 (anti-TcdB mAb) Late-stage development Early development Clostridium difficile Trial Population Patients Design Endpoints Status CVRM V&I Healthy volunteers 84 • Primary endpoint: safety • FPCD: Q2 2024 Phase I • Randomised, double-blind, placebo-controlled, dose
Slide 137: Rare Disease: approved medicines and late-stage development
Rare Disease: approved medicines and late-stage development 137
Slide 138: Koselugo: Neurofibromatosis type 1, solid tumours
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 145 • Multi-centre, international trial with a par
Slide 139: Ultomiris: Haematology, nephrology
Approved medicines Oncology Ultomiris (anti-C5 mAb) Late-stage development Early development Haematology, nephrology Trial Population Patients Design Endpoints Status CVRM Phase III Thrombotic microangiopathy-associated 106 • Arm 1: Ultomiris Q8W • Primary endpoint: TMA response • FPCD: Q4 2020 ALXN
Slide 140: 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 141: Voydeya: Haematology
Approved medicines Oncology Voydeya (factor D inhibitor) Late-stage development Early development Haematology Trial Population Patients Design Endpoints Status CVRM Phase III PNH with clinically significant EVH 86 • Arm 1: Voydeya + C5 Inhibitor • Primary endpoint: change from baseline • FPCD: Q1 20
Slide 142: acoramidis: ATTR-CM
Approved medicines Oncology acoramidis (ALXN2060) Late-stage development Early development ATTR-CM Trial Population Patients Design Endpoints Status CVRM Phase III ATTR-CM 22 • Arm 1: 800mg acoramidis administered twice daily • Primary endpoint: change from baseline • FPCD: Q4 2020 ALXN2060-TAC-302
Slide 143: ALXN2220: Amyloidosis
Approved medicines Oncology ALXN2220 (NI006, TTR depleter) Late-stage development Early development Amyloidosis Trial Population Patients Design Endpoints Status CVRM Phase III ATTR-CM 1000 • Arm 1: ALXN2220 via i.v. infusion Q4W for at least 24 months • Primary endpoints: all-cause mortality • FPCD
Slide 144: anselamimab: AL amyloidosis
Approved medicines Oncology anselamimab (CAEL-101, fibril-reactive mAb) Late-stage development Early development AL amyloidosis Trial Population Patients Design Endpoints Status CVRM Phase III AL amyloidosis (Mayo Stage IIIa) 267 • Arm 1: anselamimab combined with SoC for PCD • Primary endpoint: tim
Slide 145: efzimfotase alfa: Hypophosphatasia
Approved medicines Oncology efzimfotase alfa (ALXN1850, next-generation asfotase alfa) Late-stage development Early development Hypophosphatasia Trial Population Patients Design Endpoints Status CVRM Phase III Hypophosphatasia 114 • Arm 1: placebo on Day 1 followed by Q2W via s.c. injection • Primar
Slide 146: eneboparatide: Hypoparathyroidism
Approved medicines Oncology eneboparatide (parathyroid hormone receptor 1) Late-stage development Early development Hypoparathyroidism Trial Population Patients Design Endpoints Status CVRM Phase III Chronic hypoparathyroidism 165 • Arm 1: 20mcg eneboparatide administered once daily via s.c. • Prima
Slide 147: gefurulimab: Neurology, nephrology
Approved medicines Oncology gefurulimab (ALXN1720, anti-C5 humanised bispecific heavy-chain antibody) Late-stage development Early development Neurology, nephrology Trial Population Patients Design Endpoints Status CVRM Phase III Generalised myasthenia gravis 254 • Arm 1: weight-based maintenance tr
Slide 148: Rare Disease: early-stage development
Rare Disease: early-stage development 148
Slide 149: ALXN1910: Bone metabolism
Approved medicines Oncology ALXN1910 (next-generation TNSALP ERT) Late-stage development Early development Bone metabolism Trial Population Patients Design Endpoints Status CVRM Phase I Healthy adults 48 • Randomised, placebo-controlled SAD • Primary endpoint: safety • FPCD: Q2 2022 ALXN1910-HV-101
Slide 150: ALXN1920: Nephrology
Approved medicines Oncology ALXN1920 (kidney-targeted factor H fusion protein) Late-stage development Early development Nephrology Trial Population Patients Design Endpoints Status CVRM Phase I Healthy adults 48 • Randomised, double-blind, placebo-controlled, SAD • Primary endpoint: safety and • FPC
Slide 151: ALXN2030: Nephrology
Approved medicines Oncology ALXN2030 (siRNA targeting complement C3) Late-stage development Early development Nephrology Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers 48 • Randomised, placebo-controlled SAD • Primary endpoint: safety • FPCD: Q4 2022 ALXN2030-HV-10
Slide 152: ALXN2080: Complement-mediated disease
Approved medicines Oncology ALXN2080 (factor D inhibitor) Late-stage development Early development Complement-mediated disease Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers 90 • SAD/MAD trial • Primary endpoints: safety and • FPCD: Q3 2022 ALXN2080-HV-101 tolerabi
Slide 153: danicopan: Ophthalmology
Approved medicines Oncology danicopan (factor D inhibitor) Late-stage development Early development Ophthalmology Trial Population Patients Design Endpoints Status CVRM Phase II Geographic atrophy 365 • Arms 1-3: danicopan dosed at 100mg-400mg QD • Primary endpoint: mean rate of change • FPCD: Q3 20
Slide 154: vemircopan: Haematology, nephrology, neurology
Approved medicines Oncology vemircopan (ALXN2050, factor D inhibitor) Late-stage development Early development Haematology, nephrology, neurology Trial Population Patients Design Endpoints Status CVRM Phase II Lupus nephritis or immunoglobulin A 126 • Arm 1 – LN cohort: vemircopan 180mg • Primary en
Slide 155: Glossary – 1 of 5
NEXT Glossary – 1 of 5 14C Carbon 14 ASO Antisense oligonucleotide BTK Bruton's tyrosine kinase 1L, 2L, 3L 1st-, 2nd- or 3rd-line ATM Ataxia telangiectasia mutated kinase BTKi Bruton's tyrosine kinase 5-FU 5-fluorouracil ATR Ataxia telangiectasia and Rad3-related protein BVAS Birmingham Vasculitis A
Slide 156: Glossary – 2 of 5
BACK NEXT Glossary – 2 of 5 CNS Central nervous system DNA Deoxyribonucleic acid ETA Endothelin A CNS-PFS Central nervous system progression-free survial dNCC Directly measured non-ceruloplasmin-bound copper ETA RA Endothelin receptor A antagonist CompEx Composite endpoint for exacerbations dnTGFb D
Slide 157: Glossary – 3 of 5
BACK NEXT Glossary – 3 of 5 GPRC5D G protein-coupled receptor, class C, group 5, member D HSD17B13 Hydroxysteroid 17-beta dehydrogenase 13 LA amylin Long-acting amylin GU Genitourinary HVPG Hepatic venous pressure gradient LAAB Long-acting antibody GYN Gynaecologic i Inhibitor LABA Long-acting beta
Slide 158: Glossary – 4 of 5
BACK NEXT Glossary – 4 of 5 MI Myocardial infarction NME New molecular entity PFS Progression-free survival mL Millilitre NMOSD Neuromyelitis optica spectrum disorder PFS2 Time to second disease progression or death MM Multiple myeloma NP Nasal polyps PgR Progesterone receptor MMAE Monomethyl aurist
Slide 159: Glossary – 5 of 5
BACK Glossary – 5 of 5 QD Once daily SGLT2 Sodium-glucose transport protein 2 Tmax Time to reach maximum observed plasma concentration QID Four times per day SGLT2i Sodium/glucose cotransporter 2 inhibitor TNBC Triple negative breast cancer QOD Every other day SGRM Selective glucocorticoid receptor