Slide 1: Clinical Trials Appendix
Clinical Trials Appendix H1 2025 Results Update 29 July 2025
Slide 2: Pipeline at a glance
Pipeline 196 19 at a glance projects in our new molecular entities development pipeline (NME) in our late-stage pipeline Across five focus therapy areas: Oncology BioPharmaceuticals 132 24 CVRM | R&I | V&I new molecular entities regulatory approvals (NME) or major lifecycle in major markets manageme
Slide 3: Key upcoming pipeline catalysts: 2025 and 2026
Key upcoming pipeline catalysts: 2025 and 2026 Oncology BioPharmaceuticals Rare Disease H2 2025 H1 2026 H2 2026 Calquence – MCL (1L) (ECHO) (JP) Calquence – CLL (1L fixed duration) (AMPLIFY) (US) Datroway – HR + HER2- breast cancer (2L+) (TROPION-Breast01) (CN) Enhertu – HER2-low and ultralow met. b
Slide 4: Clinical Trials Appendix: selected highlights
Clinical Trials Appendix: selected highlights BioPharmaceuticals Oncology Rare Disease BioPharmaceuticals Oncology Rare Disease AIRSUPRA™ Inhalation Aerosol For Health Care Providers DATROWAY® (datopotamab deruxtecan-dlnk) is Now Approved Approved medicines: Koselugo® (selumetinib) | Official HCP We
Slide 5: Project movements since Q1 2025 update
Project movements since Q1 2025 update New to Phase I New to Phase II New to pivotal trial New to registration NME NME NME NME AZD0240 AZD0120 laroprovstat (AZD0780) AZURE camizestrant + CDK4/6i SERENA-6 KRAS G12D armoured TCR-T solid tumours autologous anti-CD19 and anti-BCMA CAR-T PCSK9 inhibitor
Slide 6: Project movements since Q1 2025 update
Project movements since Q1 2025 update Removed from Phase I Removed from Phase II Removed from Phase III Approved/removed from registration AZD5851 Additional indication GPC3 CAR-T hepatocellular carcinoma zibotentan/dapagliflozin endothelin A receptor antagonist/SGLT2 AZD6422 inhibitor liver cirrho
Slide 7: Q2 2025 Oncology new molecular entity pipeline
Q2 2025 Oncology new molecular entity1 pipeline Phase I Phase II Phase III 22 New Molecular Entities 17 New Molecular Entities 19 New Molecular Entities surovatamig (AZD0486) surovatamig (AZD0486) SOUNDTRACK-E Imfinzi +/- oleclumab +/- monalizumab camizestrant ceralasertib camizestrant + palbociclib
Slide 8: Q2 2025 Oncology lifecycle management pipeline
Q2 2025 Oncology lifecycle management1 pipeline Phase I Phase II Phase III 0 Projects 9 Projects 35 Projects Datroway + Imfinzi + CTx AVANZAR# Datroway + Imfinzi TROPION-Breast04# Enhertu (platform) DESTINY-Breast07# Calquence + R-CHOP ESCALADE TROP2 ADC + PD-L1 + CTx non-squamous or non-squamous TR
Slide 9: Q2 2025 BioPharmaceuticals new molecular entity pipeline
Q2 2025 BioPharmaceuticals new molecular entity1 pipeline Phase I Phase II Phase III 15 New Molecular Entities 15 New Molecular Entities 8 New Molecular Entities AZD0120 balcinrenone/dapagliflozin AZD0233 atuliflapon balcinrenone/dapagliflozin autologous anti-CD19 and anti-BCMA CAR-T cell MR modulat
Slide 10: Q2 2025 BioPharmaceuticals life cycle management pipeline
Q2 2025 BioPharmaceuticals life cycle management1 pipeline Phase I Phase II Phase III Under review 0 Projects 0 Projects 13 Projects 1 Project Breztri/Trixeo ATHLOS Breztri/Trixeo (PT010) KALOS LOGOS Tezspire WAYPOINT# LABA/LAMA/ICS COPD cardiopulmonary LABA/LAMA/ICS asthma TSLP nasal polyps exercis
Slide 11: Q2 2025 Rare Disease pipeline
Q2 2025 Rare Disease pipeline1 Phase 1 Phase 2 Phase 3 Reg 3 Projects 1 Project 9 Projects 1 Project ALXN1920 MEDI1341# anselamimab Koselugo KOMET# alpha synuclein mAb multiple system atrophy/Parkinson's kidney-targeted factor H fusion protein nephrology disease fibril-reactive mAb amyloid light-cha
Slide 12: Active designations in our pipeline
Active designations in our pipeline 2 6 12 3 19 Priority Review Breakthrough / PRIME1 / Sakigake2 Fast Track Qualified infectious disease product Orphan Imfinzi resectable early-stage gastric and Tezspire COPD EMBARK, JOURNEY (US) AZD0292 Psl-PcrV N3Y NCFBE (US) AZD0292 Psl-PcrV N3Y NCFBE (US) Fasen
Slide 13: Oncology: approved medicines and late-stage pipeline
Oncology: approved medicines and late-stage pipeline 13
Slide 14: Calquence
Approved medicines Late-stage development Oncology Calquence (BTK inhibitor) Early development Blood cancers Trial Population Patients Design Endpoints Status CVRM Phase III Previously untreated CLL 981 • Arm 1; Calquence + venetoclax • Primary endpoint: IRC PFS (Arm 1 vs. • FPCD: Q1 2019 AMPLIFY (A
Slide 15: Datroway: breast cancer
Approved medicines Late-stage development Oncology Datroway (datopotamab deruxtecan, TROP2 ADC) 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 O
Slide 16: Datroway: NSCLC
Approved medicines Late-stage development Oncology Datroway (datopotamab deruxtecan, TROP2 ADC) Early development NSCLC Trial Population Patients Design Endpoints Status CVRM Phase III 1L NSCLC 1280 • Arm 1: Datroway + Imfinzi + carboplatin • Co-primary endpoints: PFS and OS in • FPCD: Q1 2023 AVANZ
Slide 17: Datroway: NSCLC
Approved medicines Late-stage development Oncology Datroway (datopotamab deruxtecan, TROP2 ADC) Early development NSCLC Trial Population Patients Design Endpoints Status CVRM Phase III EGFRm locally advanced or metastatic 562 • Arm 1: Tagrisso + Datroway • Primary endpoint: PFS (BICR) • FPCD: Q2 202
Slide 18: Datroway: other cancers
Approved medicines Late-stage development Oncology Datroway (datopotamab deruxtecan, TROP2 ADC) Early development Other cancers Trial Population Patients Design Endpoints Status CVRM Phase II Endometrial cancer, gastric cancer, 556 • Sub-study 1 (endometrial cancer); • Primary endpoints: ORR and saf
Slide 19: Enhertu: breast cancer
Approved medicines Late-stage development Oncology Enhertu (trastuzumab deruxtecan, HER2 ADC) Early development Breast cancer Trial Population Patients Design Endpoints Status CVRM Phase III HER2-positive, unresectable and/or 600 • Randomised, open-label, parallel assignment • Primary endpoint: PFS
Slide 20: Enhertu: breast cancer
Approved medicines Late-stage development Oncology Enhertu (trastuzumab deruxtecan, HER2 ADC) Early development Breast cancer Trial Population Patients Design Endpoints Status CVRM Phase III High-risk HER2-positive early non- 927 • Randomised, open-label, parallel assignment • Primary endpoint: pCR
Slide 21: Enhertu: gastric cancer
Approved medicines Late-stage development Oncology Enhertu (trastuzumab deruxtecan, HER2 ADC) Early development Gastric cancer Trial Population Patients Design Endpoints Status CVRM Phase III HER2-positive gastric cancer or GEJ 490 • Open-label, randomised, parallel group assignment • Primary endpoi
Slide 22: Enhertu: other cancers
Approved medicines Late-stage development Oncology Enhertu (trastuzumab deruxtecan, HER2 ADC) Early development Other cancers Trial Population Patients Design Endpoints Status CVRM Phase III HER2-mutated, unresectable, locally 450 • Randomised, parallel group assignment • Primary endpoint: PFS • FPC
Slide 23: Enhertu: other cancers
Approved medicines Late-stage development Oncology Enhertu (trastuzumab deruxtecan, HER2 ADC) Early development Other cancers Trial Population Patients Design Endpoints Status CVRM Phase III Advanced treatment-naïve HER2- 620 • Arm A: Enhertu + rilvegostomig • Primary endpoint: OS • FPCD: Q3 2024 DE
Slide 24: Imfinzi: gastrointestinal cancer
Approved medicines Late-stage development Oncology Imfinzi (PD-L1 mAb) Early development Gastrointestinal cancer Trial Population Patients Design Endpoints Status CVRM Phase III Locoregional HCC 710 • Arm 1: TACE in combination with Imfinzi • Primary endpoint: PFS (Arm 2 vs. Arm • FPCD: Q1 2019 EMER
Slide 25: Imfinzi: lung cancer
Approved medicines Late-stage development Oncology Imfinzi (PD-L1 mAb) Early development Lung cancer Trial Population Patients Design Endpoints Status CVRM Phase III Perioperative NSCLC patients, Stage II 802 • Arm 1: Imfinzi + platinum-based chemotherapy • Primary endpoints: pCR snd EFS • FPCD: Q1
Slide 26: Imfinzi: lung cancer
Approved medicines Late-stage development Oncology Imfinzi (PD-L1 mAb) Early development Lung cancer Trial Population Patients Design Endpoints Status CVRM Phase II NSCLC, patients who progressed on an 531 • Open-label, biomarker-directed, multi-centre trial • Primary endpoint: ORR • FPCD: Q1 2018 H
Slide 27: Imfinzi: other cancer
Approved medicines Late-stage development Oncology Imfinzi (PD-L1 mAb) Early development Other cancer Trial Population Patients Design Endpoints Status CVRM Phase III Non-muscle-invasive bladder cancer 1018 • Arm 1: BCG (induction + maintenance) • Primary endpoint: DFS • FPCD: Q2 2018 POTOMAC • Arm
Slide 28: Lynparza: Imfinzi combinations
Approved medicines Late-stage development Oncology Lynparza (PARP inhibitor) Early development Imfinzi combinations Trial Population Patients Design Endpoints Status CVRM Phase III 1L advanced ovarian cancer 1407 • Non-tBRCAm (tumour BRCA) patients • Primary endpoint: PFS • FPCD: Q1 2019 DUO-O • Arm
Slide 29: Lynparza: other cancer
Approved medicines Late-stage development Oncology Lynparza (PARP inhibitor) Early development Other cancer Trial Population Patients Design Endpoints Status CVRM Phase III BRCAwt advanced ovarian cancer, 1L 366 • Arm 1: Lynparza BID 24-month duration • Primary endpoints: PFS (BRCAwt HRD- • FPCD: Q3
Slide 30: Orpathys
Approved medicines Late-stage development Oncology Orpathys (savolitinib, MET inhibitor) Early development NSCLC and other cancers Trial Population Patients Design Endpoints Status CVRM Phase III Locally advanced or metastatic NSCLC 163 • Single-arm trial • Primary endpoint: ORR • FPCD: Q3 2021 NCT0
Slide 31: Tagrisso: NSCLC
Approved medicines Late-stage development Oncology Tagrisso (highly-selective, irreversible EGFR inhibitor) 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 32: Tagrisso: NSCLC combinations
Approved medicines Late-stage development Oncology Tagrisso (highly-selective, irreversible EGFR inhibitor) Early development NSCLC, combinations Trial Population Patients Design Endpoints Status CVRM Phase III Neoadjuvant EGFRm NSCLC 351 • Arm 1: placebo + pemetrexed/carboplatin or • Primary endpoi
Slide 33: Truqap
Approved medicines Late-stage development Oncology Truqap (capivasertib, AKT inhibitor) Early development Breast cancer and prostate cancer Trial Population Patients Design Endpoints Status CVRM Phase III 2L+ AI-resistant locally advanced 834 • Double-blind, randomised, comparative trial • Primary e
Slide 34: camizestrant: breast cancer
Approved medicines Late-stage development Oncology camizestrant (next-generation oral SERD) 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 endpoint: PFS •
Slide 35: camizestrant: breast cancer
Approved medicines Late-stage development Oncology camizestrant (next-generation oral SERD) Early development Breast cancer Trial Population Patients Design Endpoints Status CVRM Phase I HR+ HER2-negative advanced breast 18 • Open-label trial • Primary endpoints: safety and • FPCD: Q4 2020 NCT045414
Slide 36: ceralasertib
Approved medicines Late-stage development Oncology ceralasertib (AZD6738, ATR inhibitor) 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 37: rilvegostomig: lung cancer
Approved medicines Late-stage development Oncology rilvegostomig (PD-1/TIGIT bispecific mAb) Early development Lung cancer Trial Population Patients Design Endpoints Status CVRM Phase III Non-squamous NSCLC 1L patients 878 • Randomised, double-blind, multi-centre trial • Primary endpoints: PFS and O
Slide 38: rilvegostomig: gastrointestinal cancers
Approved medicines Late-stage development Oncology rilvegostomig (PD-1/TIGIT bispecific mAb) Early development Gastrointestinal cancers Trial Population Patients Design Endpoints Status CVRM Phase III adjuvant BTC with curative intent 750 • Randomised, double-blind, placebo-controlled, multicenter •
Slide 39: rilvegostomig: gastrointestinal cancers
Approved medicines Late-stage development Oncology rilvegostomig (PD-1/TIGIT bispecific mAb) Early development Gastrointestinal cancers Trial Population Patients Design Endpoints Status CVRM Phase IIb Gastric cancer 240 • Open-label gastric platform trial • Primary endpoints: safety and efficacy • F
Slide 40: saruparib: solid tumours
Approved medicines Late-stage development Oncology saruparib (AZD5305, PARP1 inhibitor) 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 41: saruparib: solid tumours
Approved medicines Late-stage development Oncology saruparib (AZD5305, PARP1 inhibitor) Early development Solid tumours Trial Population Patients Design Endpoints Status CVRM Phase I/IIa Advanced solid tumours 804 • Modular, open-label, multi-centre dose escalation and • Primary endpoints: safety, t
Slide 42: sonesitatug vedotin: solid tumours
Approved medicines Late-stage development Oncology sonesitatug vedotin (AZD0901, CLDN18.2 MMAE ADC) Early development Solid tumours Trial Population Patients Design Endpoints Status CVRM Phase III 2L+ advanced or metastatic gastric or 572 • Multi-centre, open-label, sponsor-blinded, randomised trial
Slide 43: surovatamig: haematologic malignancies
Approved medicines Late-stage development Oncology surovatamig (AZD0486, CD19/CD3 next-generation bispecific T-cell engager) Early development Haematologic malignancies Trial Population Patients Design Endpoints Status CVRM Phase III Previously untreated follicular 1015 • Multi-centre, randomised, o
Slide 44: volrustomig: solid tumours
Approved medicines Late-stage development Oncology volrustomig (PD-1/CTLA-4 bispecific mAb) Early development Solid tumours Trial Population Patients Design Endpoints Status CVRM Phase III High-risk locally advanced cervical 800 • Randomised, double-blind, placebo-controlled, multi-centre • Primary
Slide 45: volrustomig: solid tumours
Approved medicines Late-stage development Oncology volrustomig (PD-1/CTLA-4 bispecific mAb) Early development Solid tumours Trial Population Patients Design Endpoints Status CVRM Phase IIb NSCLC 120 • Platform, randomised, open-label, multicenter, global trial • Primary endpoints: safety, & • FPCD:
Slide 46: Oncology: early-stage development
Oncology: early-stage development 46
Slide 47: FPI-2265: prostate cancer
Approved medicines Late-stage development Oncology FPI-2265 (PSMA radioconjugate) Early development Prostate cancer Trial Population Patients Design Endpoints Status CVRM Phase II PSMA-positive mCRPC previously 100 • Open-label, randomised, multi-centre trial • Primary endpoints: PSA50 and safety •
Slide 48: oleclumab: solid tumours
Approved medicines Late-stage development Oncology oleclumab (CD73 mAb) 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 49: puxitatug samrotecan: solid tumours
Approved medicines Late-stage development Oncology puxitatug samrotecan (AZD8205, B7H4 ADC) Early development Solid tumours Trial Population Patients Design Endpoints Status CVRM Phase I/II Breast cancer, ovarian cancer, 370 • Open-label dose escalation and expansion trial • Primary endpoints: AE, S
Slide 50: tilatamig samrotecan: lung cancer
Approved medicines Late-stage development Oncology tilatamig samrotecan (AZD9592, EGFR-cMET TOP1i ADC) Early development Lung cancer Trial Population Patients Design Endpoints Status CVRM Phase I Advanced solid tumours including 228 • Escalation phase, open-label, multi-centre trial • Primary endpoi
Slide 51: AZD0022: solid tumours
Approved medicines Late-stage development Oncology AZD0022 (KRASG12D) Early development Solid tumours Trial Population Patients Design Endpoints Status CVRM Phase I/II Metastatic or locally advanced 430 • Open-label, multi-centre trial with FIH modular protocol • Primary endpoints (Part A and B): •
Slide 52: AZD0120: blood cancers
Approved medicines Late-stage development Oncology AZD0120 (GC012F, autologous anti-CD19 and anti-BCMA CAR-T) 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 53: AZD0305: blood cancers
Approved medicines Late-stage development Oncology AZD0305 (GPRC5D ADC) Early development Blood cancers Trial Population Patients Design Endpoints Status CVRM Phase I/IIa R/R multiple myeloma 108 • Open-label, dose escalation and dose expansion trial • Primary endpoints: occurrence of • FPCD: Q4 202
Slide 54: AZD0754: solid tumours
Approved medicines Late-stage development Oncology AZD0754 (STEAP2 dnTGFβRII-armoured CAR-T) 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 endpoint
Slide 55: AZD1390: cancer
Approved medicines Late-stage development Oncology AZD1390 (ATM inhibitor) Early development Cancer Trial Population Patients Design Endpoints Status CVRM Phase I Recurrent glioblastoma eligible for re- 165 • Open-label trial • Primary endpoints: safety, tolerability • FPCD: Q2 2018 NCT03423628 irra
Slide 56: AZD2068: solid tumours
Approved medicines Late-stage development Oncology AZD2068 (FPI-2068, EGFR cMET radioconjugate) 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: AZD2284: prostate cancer
Approved medicines Late-stage development Oncology AZD2284 (STEAP2 radioconjugate) Early development Prostate cancer Trial Population Patients Design Endpoints Status CVRM Phase I mCRPC 134 • Part A (Imaging): • Primary endpoints: safety and • FPCD: Q1 2025 NCT06879041 • Part A (Cold Antibody Explor
Slide 58: AZD3470: solid tumours and blood cancer
Approved medicines Late-stage development Oncology AZD3470 (PRMT5) Early development Solid tumours and blood cancer Trial Population Patients Design Endpoints Status CVRM Phase I MTAP-deficient advanced solid tumours 234 • Arm 1: AZD3470 • Primary endpoints: safety and • FPCD: Q1 2024 PRIMROSE • Glo
Slide 59: AZD4360: solid tumours
Approved medicines Late-stage development Oncology AZD4360 (CLDN18.2 ADC) Early development Solid tumours Trial Population Patients Design Endpoints Status CVRM Phase I/II Histologically confirmed advanced or 117 • • Open-label, multi-centre trial with FIH modular protocol • • Primary endpoints: saf
Slide 60: AZD5335: solid tumours
Approved medicines Late-stage development Oncology AZD5335 (anti-FRα TOP1i ADC) Early development Solid tumours, ovarian cancer, lung cancer Trial Population Patients Design Endpoints Status CVRM Phase I/II Advanced solid tumour malignancies 446 • Module 1: AZD5335 monotherapy • Primary endpoints: s
Slide 61: AZD5492: blood cancers
Approved medicines Late-stage development Oncology AZD5492 (CD20 TITAN TCE) Early development Blood cancers Trial Population Patients Design Endpoints Status CVRM Phase I CLL, MCL, LBCL, FL 176 • Module 1: AZD5492 monotherapy • Primary endpoints: safety and • FPCD: Q3 2024 TITANIUM • AZD5492 monothe
Slide 62: AZD5851: gastrointestinal cancer
Approved medicines Late-stage development Oncology AZD5851 (GPC3 dnTGFβRII-armoured CAR-T) Early development Gastrointestinal cancer Trial Population Patients Design Endpoints Status CVRM Phase I/II GPC3-positive advanced/recurrent HCC 94 • Open-label, single-arm, multi-centre trial with dose • Prim
Slide 63: AZD5863: solid tumours
Approved medicines Late-stage development Oncology AZD5863 (CLDN18.2 CD3 bispecific antibody) Early development Solid tumours Trial Population Patients Design Endpoints Status CVRM Phase I Advanced or metastatic solid tumours 240 • Part A: dose escalation phase to determine the safety, • Primary end
Slide 64: AZD6422: solid tumours
Approved medicines Late-stage development Oncology AZD6422 (CLDN18.2 CAR-T) Early development Solid tumours Trial Population Patients Design Endpoints Status CVRM Phase I Advanced or metastatic CLDN18.2- 96 • Open-label trial, dose escalation (Part 1) and dose • Primary endpoints: incidence of • FPC
Slide 65: AZD7003: hepatocellular carcinoma
Approved medicines Late-stage development Oncology AZD7003 (GPC3 CAR-T) Early development Hepatocellular carcinoma (HCC) Trial Population Patients Design Endpoints Status CVRM Phase I/II GPC3-positive advanced/recurrent HCC 121 • Open-label, single-arm, multi-centre trial with dose • Primary endpoin
Slide 66: AZD8421: breast cancer
Approved medicines Late-stage development Oncology AZD8421 (CDK2 inhibitor) 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 67: AZD9574: solid tumours
Approved medicines Late-stage development Oncology AZD9574 (PARP1-sel BBB inhibitor) Early development Solid tumours Trial Population Patients Design Endpoints Status CVRM Phase I/IIa Advanced solid malignancies 535 • Modular, open-label, multi-centre dose escalation and • Primary endpoints: safety
Slide 68: AZD9793: solid tumours
Approved medicines Late-stage development Oncology AZD9793 (GPC3 TITAN T-cell engager) Early development Solid tumours Trial Population Patients Design Endpoints Status CVRM Phase I/II Metastatic solid tumours 304 • Open label, non randomised, multi-centre, dose escalation • Primary endpoint: safety
Slide 69: AZD9829: blood cancers
Approved medicines Late-stage development Oncology AZD9829 (CD123 TOP1i ADC) Early development Blood cancers Trial Population Patients Design Endpoints Status CVRM Phase I/II CD123-positive haematological 104 • Open-label, multi-centre trial • Primary endpoints: safety and • FPCD: Q1 2024 ADC123 mal
Slide 70: IPH5201: solid tumours
Approved medicines Late-stage development Oncology IPH5201 (CD39 mAb) Early development Solid tumours Trial Population Patients Design Endpoints Status CVRM Phase I Advanced solid tumours 57 • Open-label, dose escalation trial to determine MTD of • Primary endpoints: AE, SAE and DLT • FPCD: Q1 2020
Slide 71: NT-112: solid tumours
Approved medicines Late-stage development Oncology NT-112 (KRAS G12D specific TCR) 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 72: NT-125: solid tumours
Approved medicines Late-stage development Oncology NT-125 (autologous, multi-specific neoantigen-targeting TCR-T) 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 73: NT-175: multiple cancers
Approved medicines Late-stage development Oncology NT-175 (TP53-armored TCR) Early development Multiple cancers Trial Population Patients Design Endpoints Status CVRM Phase I Unresectable, advanced, and/or 162 • Open-label, single-arm, multi-centre trial with dose • Primary endpoint: Incidence of D
Slide 74: BioPharmaceuticals: approved medicines and late-stage development
BioPharmaceuticals: approved medicines and late-stage development 74
Slide 75: Wainua: ATTR
Approved medicines Late-stage development Oncology Wainua (eplontersen, ligand-conjugated antisense) 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 76: balcinrenone/dapagliflozin: heart failure, CKD
Approved medicines Late-stage development Oncology balcinrenone/dapagliflozin (MR modulator + SGLT2 inhibitor) 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 77: baxdrostat: hypertension
Approved medicines Late-stage development Oncology baxdrostat (selective aldosterone synthase inhibitor) 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 78: baxdrostat: hypertension
Approved medicines Late-stage development Oncology baxdrostat (selective aldosterone synthase inhibitor) 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 79: baxdrostat/dapagliflozin: CKD
Approved medicines Late-stage development Oncology baxdrostat/dapagliflozin (selective ASI/SGLT2) Early development CKD/Prevention of heart failure Trial Population Patients Design Endpoints Status CVRM Phase III CKD and high blood pressure 2500 • Arm 1: baxdrostat/dapagliflozin QD • Primary endpoin
Slide 80: laroprovstat: dyslipidaemia
Approved medicines Late-stage development Oncology laroprovstat (AZD0780, oral PCSK9 inhibitor) Early development Dyslipidaemia Trial Population Patients Design Endpoints Status CVRM Phase III Patients with dyslipidaemia 2800 • Randomised, double-blind, placebo-controlled, parallel- • Primary endpoi
Slide 81: laroprovstat: dyslipidaemia
Approved medicines Late-stage development Oncology laroprovstat (AZD0780, oral PCSK9 inhibitor) Early development Dyslipidaemia Trial Population Patients Design Endpoints Status CVRM Phase II Dyslipidaemia 428 • Randomised trial with equal distribution across five parallel • Primary endpoint: percen
Slide 82: zibotentan/dapagliflozin: CKD
Approved medicines Late-stage development Oncology zibotentan/dapagliflozin (ETA receptor antagonist/SGLT2 inhibitor) Early development Chronic kidney disease Trial Population Patients Design Endpoints Status CVRM Phase III CKD and high proteinuria 1835 • Randomised, parallel, multi-centre, double-b
Slide 83: zibotentan/dapagliflozin: liver cirrhosis
Approved medicines Late-stage development Oncology zibotentan/dapagliflozin (ETA receptor antagonist/SGLT2 inhibitor) 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 Late-stage development Oncology Airsupra (PT027, SABA/ICS, pMDI) 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: Breztri, Trixeo: asthma
Approved medicines Late-stage development Oncology Breztri, Trixeo (LAMA/LABA/ICS) Early development Asthma Trial Population Patients Design Endpoints Status CVRM Phase III Uncontrolled asthma 2266 • Randomised, double-blind, double-dummy, parallel group • Primary endpoint: change from • FPCD: Q1 20
Slide 86: Breztri, Trixeo: COPD
Approved medicines Late-stage development Oncology Breztri, Trixeo (LAMA/LABA/ICS) 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 crosso
Slide 87: Fasenra: nasal polyposis and eosinophilic diseases
Approved medicines Late-stage development Oncology Fasenra (IL-5R mAb) Early development Nasal polyposis and other eosinophilic diseases Trial Population Patients Design Endpoints Status CVRM Phase III Patients with r/r EGPA on 140 • Arm 1: Fasenra 30mg Q4W s.c. • Primary endpoint: proportion of • F
Slide 88: Fasenra: asthma and COPD
Approved medicines Late-stage development Oncology Fasenra (IL-5R mAb) Early development Severe, uncontrolled asthma and COPD Trial Population Patients Design Endpoints Status CVRM Phase III Patients with moderate to 689 • Double-blind, placebo-controlled trial • Primary endpoint: annualised rate of
Slide 89: Saphnelo: lupus
Approved medicines Late-stage development Oncology Saphnelo (type I interferon receptor mAb) Early development Lupus (SLE/LN) Trial Population Patients Design Endpoints Status CVRM Phase III Moderate to severe SLE 360 • Arm 1: Saphnelo s.c. • Primary endpoint: BICLA at Week 52 • FPCD: Q3 2021 TULIP-
Slide 90: Saphnelo: sclerosis and myopathies
Approved medicines Late-stage development Oncology Saphnelo (type I interferon receptor mAb) 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 Week • FPCD: Q4
Slide 91: Tezspire: CRSwNP, COPD and EoE
Approved medicines Late-stage development Oncology Tezspire (TSLP mAb) 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 92: Tezspire: severe asthma
Approved medicines Late-stage development Oncology Tezspire (TSLP mAb) Early development Severe, uncontrolled asthma Trial Population Patients Design Endpoints Status CVRM Phase III Severe asthma; age 12 to 80 1061 • Arm 1: Tezspire s.c. • Primary endpoint: annual asthma • FPCD: Q1 2018 NAVIGATOR ye
Slide 93: tozorakimab: COPD
Approved medicines Late-stage development Oncology tozorakimab (IL-33 ligand mAb) Early development COPD Trial Population Patients Design Endpoints Status CVRM Phase III Adults with symptomatic 1132 • Randomised, double-blind, placebo-controlled, parallel- • Primary endpoint: annualised rate of • FP
Slide 94: tozorakimab: severe viral LRTD, asthma
Approved medicines Late-stage development Oncology tozorakimab (IL-33 ligand mAb) 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 95: Next-generation propellant: pMDI
Approved medicines Late-stage development Oncology Next-generation propellant (NGP) 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 • FPCD: Q2
Slide 96: Next-generation propellant: pMDI
Approved medicines Late-stage development Oncology Next-generation propellant (NGP) Early development pMDI Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers 108 • Randomised, double-blind, single-dose, single-centre, • Primary endpoints: AUCinf, AUClast • FPCD: Q3 202
Slide 97: Beyfortus: infection
Approved medicines Late-stage development Oncology Beyfortus (nirsevimab, RSV mAb-YTE) Early development Infection Trial Population Patients Design Endpoints Status CVRM Phase III Healthy infants (born 29 800 • Randomised, double-blind, placebo-controlled • Primary endpoint: efficacy • FPCD: Q4 2021
Slide 98: Kavigale: COVID-19
Approved medicines Late-stage development Oncology Kavigale (sipavibart, SARS-CoV-2 LAAB) 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 endpoint
Slide 99: BioPharmaceuticals: early-stage development
BioPharmaceuticals: early-stage development 99
Slide 100: opemalirsen: chronic kidney disease
Approved medicines Late-stage development Oncology opemalirsen (AZD2373, APOL1) Early development Chronic kidney disease Trial Population Patients Design Endpoints Status CVRM Phase IIb Participants diagnosed with 96 • Randomised, multi-centre, double-blind trial in US and UK • Primary endpoint: Dos
Slide 101: AZD0233: dilated cardiomyopathy
Approved medicines Late-stage development Oncology AZD0233 (oral CX3CR1) Early development Dilated cardiomyopathy Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers 96 • Randomised, SAD/MAD dose escalating trial • Primary endpoints: safety and • FPCD: Q2 2024 NCT063814
Slide 102: AZD1613: ADPKD
Approved medicines Late-stage development Oncology AZD1613 (PAPPA-1 MAb) Early development ADPKD Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers 136 • Randomised, single-blind, placebo-controlled single and • Primary endpoint: safety and • FPCD: Q2 2025 NCT06995820
Slide 103: AZD1705: dyslipidaemia
Approved medicines Late-stage development Oncology AZD1705 (Angptl3 inhibitor) 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 104: AZD2389: MASH
Approved medicines Late-stage development Oncology AZD2389 (anti-fibrotic mechanism) Early development MASH Trial Population Patients Design Endpoints Status CVRM Phase II Participants with liver fibrosis 36 • Randomised, single-blind, placebo-controlled trial • Primary endpoints: safety and • FPCD:
Slide 105: AZD2693: MASH
Approved medicines Late-stage development Oncology AZD2693 (PNPLA3 ASO) Early development MASH Trial Population Patients Design Endpoints Status CVRM Phase IIb MASH with fibrosis 180 • Randomised, double-blind, placebo-controlled, multi-centre • Primary endpoints: efficacy, safety • FPCD: Q2 2023 FO
Slide 106: AZD3427: heart failure
Approved medicines Late-stage development Oncology AZD3427 (relaxin) Early development Heart failure Trial Population Patients Design Endpoints Status CVRM Phase II HF and pulmonary 260 • Randomised, double-blind, placebo-controlled, multi-centre • Primary endpoint: change in PVR from • FPCD: Q2 202
Slide 107: AZD4144: cardiorenal disease
Approved medicines Late-stage development Oncology AZD4144 (inflammation modulator) Early development Cardiorenal disease Trial Population Patients Design Endpoints Status CVRM Phase I Healthy participants 95 • Randomised, single-blind, placebo-controlled, SAD/MAD • Primary endpoints: safety and • F
Slide 108: AZD4248: CKD
Approved medicines Late-stage development Oncology AZD4248 (oral NNMT) Early development CKD Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers 164 • Randomised, single-blind, placebo-controlled • Primary endpoints: Safety and • FPCD: Q2 2025 NCT07024823 • Part A: SAD
Slide 109: AZD4954: dyslipidaemia
Approved medicines Late-stage development Oncology AZD4954 (Lp(a) inhibitor) Early development Dyslipidaemia Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers 120 • Randomised, placebo-controlled SAD/MAD trial • Primary endpoints: safety and • FPCD: Q2 2025 NCT0698042
Slide 110: AZD5004: type 2 diabetes, obesity
Approved medicines Late-stage development Oncology AZD5004 (oral GLP-1 RA) Early development Type 2 diabetes, obesity Trial Population Patients Design Endpoints Status CVRM Phase IIb Type 2 diabetes 406 • Arm 1: AZD5004 tablet • Primary endpoint: change in HbA1c • FPCD: Q4 2024 SOLSTICE • Arm 2: AZD
Slide 111: AZD5004: type 2 diabetes, obesity
Approved medicines Late-stage development Oncology AZD5004 (oral GLP-1 RA) Early development Type 2 diabetes, obesity Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers 31 • Part A – Arm 1: AZD5004 oral tablet • Primary endpoints (Part A): Safety and • FPCD: Q3 2024 NC
Slide 112: AZD5004: type 2 diabetes, obesity
Approved medicines Late-stage development Oncology AZD5004 (oral GLP-1 RA) Early development Type 2 diabetes, obesity Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers 16 • Open-label, randomized, 4-period, 4-treatment, single-dose • Primary endpoint: PK profile (rela
Slide 113: AZD5462: heart failure
Approved medicines Late-stage development Oncology AZD5462 (oral relaxin) 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
Slide 114: AZD6234: obesity
Approved medicines Late-stage development Oncology AZD6234 (selective amylin agonist) 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 endp
Slide 115: AZD9550: MASH
Approved medicines Late-stage development Oncology AZD9550 (GLP-1-glucagon agonist) Early development MASH Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers 64 • SAD trial • Primary endpoints: safety and • FPCD: Q2 2023 NCT05848440 tolerability • LPCD: Q4 2023 • Data
Slide 116: AZD9550+AZD6234: obesity
Approved medicines Late-stage development Oncology AZD9550+AZD6234 (GLP-1-glucagon agonist + selective amylin agonist) Early development Obesity Trial Population Patients Design Endpoints Status CVRM Phase IIb Adults who are living with 360 • Randomised, parallel-group, double-blind, placebo- • Prim
Slide 117: atuliflapon: asthma
Approved medicines Late-stage development Oncology atuliflapon (FLAP inhibitor) 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 118: AZD0120: SLE
Approved medicines Late-stage development Oncology AZD0120 (GC012F, autologous anti-CD19 and anti-BCMA CAR-T) Early development SLE Trial Population Patients Design Endpoints Status CVRM Phase Ib/II Refractory systemic lupus 24 • Single-arm, open-label, multi-center trial • Primary endpoints (Phase
Slide 119: AZD1163: rheumatoid arthritis
Approved medicines Late-stage development Oncology AZD1163 (anti-PAD2/4 bispecific antibody) Early development Rheumatoid arthritis Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers 99 • Randomised, double-blind, placebo-controlled SAD/MAD • Primary endpoint: incidenc
Slide 120: AZD4604: asthma
Approved medicines Late-stage development Oncology AZD4604 (inhaled JAK-1 inhibitor) Early development Asthma Trial Population Patients Design Endpoints Status CVRM Phase IIa Moderate-to-severe asthma 320 • Multi-centre, randomised, placebo-controlled, double- • Primary endpoint: time to first • FPC
Slide 121: AZD5492: SLE
Approved medicines Late-stage development Oncology AZD5492 (CD20 TITAN TCE) Early development SLE Trial Population Patients Design Endpoints Status CVRM Phase I Systemic lupus 50 • Open-label, multii-centre • Primary endpoints: safety and • Data anticipated: >2026 TITAN erythematosus (SLE) or • Part
Slide 122: AZD6793: inflammatory diseases
Approved medicines Late-stage development Oncology AZD6793 (IRAK4) Early development Inflammatory diseases Trial Population Patients Design Endpoints Status CVRM Phase II moderate to very severe 1160 • Randomised, double-blind, placebo controlled 4 arm study • Primary endpoint: annualised rate of •
Slide 123: AZD6912: rheumatoid arthritis
Approved medicines Late-stage development Oncology AZD6912 (siRNA) 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 124: AZD7798: Crohn's disease
Approved medicines Late-stage development Oncology AZD7798 (humanised mAb) 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 125: AZD8630: asthma
Approved medicines Late-stage development Oncology AZD8630 (inhaled TSLP) 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 126: AZD8965: IPF
Approved medicines Late-stage development Oncology AZD8965 (arginase enzyme inhibitor) Early development IPF Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers 163 • Randomised, single-blind, SAD/MAD, placebo-controlled, • Primary endpoints (Part 1, 2, 3): safety • FPC
Slide 127: mRNA VLP vaccine: COVID-19
Approved medicines Late-stage development Oncology mRNA VLP vaccine 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 128: AZD0292: bronchiectasis
Approved medicines Late-stage development Oncology AZD0292 (Psl-PcrV N3Y-bispecific mAb) 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 129: AZD5148: Clostridium difficile
Approved medicines Late-stage development Oncology AZD5148 (anti-TcdB mAb) Early development Clostridium difficile Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers 84 • Randomised, double-blind, placebo-controlled, dose • Primary endpoint: safety • FPCD: Q2 2024 NCT0
Slide 130: AZD7760: prevention of Staph aureus infection
Approved medicines Late-stage development Oncology AZD7760 (mAb combination targeting S aureus virulence factors) Early development Prevention of Staph aureus infection Trial Population Patients Design Endpoints Status CVRM Phase I/IIa Phase I: healthy volunteers 231 • Phase I: randomised, double-bl
Slide 131: Rare Disease: approved medicines and late-stage development
Rare Disease: approved medicines and late-stage development 131
Slide 132: Beyonttra: ATTR-CM
Approved medicines Late-stage development Oncology Beyonttra (acoramidis, ALXN2060) Early development ATTR-CM Trial Population Patients Design Endpoints Status CVRM Phase III ATTR-CM 22 • Arm 1: 800mg Beyonttra administered twice daily • Primary endpoint: change from • FPCD: Q4 2020 ALXN2060-TAC-302
Slide 133: Koselugo: neurofibromatosis type 1
Approved medicines Late-stage development Oncology Koselugo (selumetinib, MEK inhibitor) Early development Neurofibromatosis type 1, solid tumours Trial Population Patients Design Endpoints Status CVRM Phase III Adult age ≥18 years with NF1 145 • Multi-centre, international trial with a parallel, ra
Slide 134: Ultomiris: haematology, nephrology, transplant
Approved medicines Late-stage development Oncology Ultomiris (anti-C5 mAb) Early development Haematology, nephrology, transplant Trial Population Patients Design Endpoints Status CVRM Phase III Thrombotic microangiopathy- 146 • Arm 1: Ultomiris Q8W • Primary endpoint: event free survival • FPCD: Q4
Slide 135: Ultomiris: neurology
Approved medicines Late-stage development Oncology Ultomiris (anti-C5 mAb) Early development Neurology Trial Population Patients Design Endpoints Status CVRM Phase III Neuromyelitis optica 58 • Arm 1: Ultomiris Q8W • Primary endpoint: time to first • FPCD: Q4 2019 NMO-307 spectrum disorder adjudicat
Slide 136: anselamimab: AL amyloidosis
Approved medicines Late-stage development Oncology anselamimab (CAEL-101, fibril-reactive mAb) Early development AL amyloidosis Trial Population Patients Design Endpoints Status CVRM Phase III AL amyloidosis (Mayo Stage 267 • Arm 1: anselamimab combined with SoC for PCD • Primary endpoint: a hierarc
Slide 137: efzimfotase alfa: hypophosphatasia
Approved medicines Late-stage development Oncology efzimfotase alfa (ALXN1850, next-generation asfotase alfa) 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 138: eneboparatide: hypoparathyroidism
Approved medicines Late-stage development Oncology eneboparatide (parathyroid hormone receptor 1 agonist) 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.
Slide 139: gefurulimab: neurology, nephrology
Approved medicines Late-stage development Oncology gefurulimab (ALXN1720, anti-C5 dual-binding nanobody) Early development Neurology, nephrology Trial Population Patients Design Endpoints Status CVRM Phase III Generalised myasthenia 254 • Arm 1: weight-based maintenance treatment with • Primary endp
Slide 140: cliramitug: amyloidosis
Approved medicines Late-stage development Oncology cliramitug (ALXN2220, TTR depleter) Early development Amyloidosis Trial Population Patients Design Endpoints Status CVRM Phase III ATTR-CM (wild-type and 1180 • Arm 1: cliramitug via i.v. infusion Q4W for at least 24 months • Primary endpoint: compo
Slide 141: Rare Disease: early-stage development
Rare Disease: early-stage development 141
Slide 142: ALXN1920: nephrology
Approved medicines Late-stage development Oncology ALXN1920 (kidney-targeted factor H fusion protein) Early development Nephrology Trial Population Patients Design Endpoints Status CVRM Phase I Healthy adults 48 • Randomised, double-blind, placebo-controlled SAD trial • Primary endpoints: safety and
Slide 143: ALXN2030: transplant
Approved medicines Late-stage development Oncology ALXN2030 (siRNA targeting complement C3) Early development Transplant Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers 48 • Randomised, placebo-controlled SAD trial • Primary endpoint: safety • FPCD: Q4 2022 ALXN2030
Slide 144: MEDI1341: multiple system atrophy
Approved medicines Late-stage development Oncology MEDI1341 (alpha-synuclein mAb) Early development Multiple system atrophy Trial Population Patients Design Endpoints Status CVRM Phase II Patients with diagnosis of 158 • Randomised, double-blind, placebo-controlled trial • Primary endpoints: efficac
Slide 145: Glossary – 1 of 5
NEXT 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 Vasculi
Slide 146: Glossary – 2 of 5
BACK NEXT 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 exacerbation
Slide 147: Glossary – 3 of 5
BACK NEXT 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-ac
Slide 148: Glossary – 4 of 5
BACK NEXT 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 Monomet
Slide 149: Glossary – 5 of 5
BACK 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 rece