Slide 1: Clinical trials appendix H1 2020 results update
Clinical trials appendix H1 2020 results update
Slide 2: Movement since Q1 2020 update
Movement since Q1 2020 update New to Phase I New to Phase II New to Pivotal trial New to registration Additional indication NME Lifecycle management brazikumab AZD1222# Enhertu#¶ DESTINY-Gastric01 [JP] 1 IL23 ulcerative colitis SARS-CoV-2 COVID vaccine HER2 targeting antibody drug conjugate HER2- ov
Slide 3: Q2 2020 new molecular entity (NME) pipeline
Q2 2020 new molecular entity (NME)1 pipeline Phase I Phase II Phase III Under Review 19 New Molecular Entities 24 New Molecular Entities 9 New Molecular Entities 0 New Molecular Entities capivasertib#+fulvestrant CAPItello-291 AZD0466 Imfinzi#+RT (platform) CLOVER adavosertib# Imfinzi#+oleclumab AKT
Slide 4: Q2 2020 new molecular entity (NME) pipeline
Q2 2020 new molecular entity (NME)1 pipeline Phase I Phase II Phase III Under Review 16 New Molecular Entities 21 New Molecular Entities 6 New Molecular Entities 0 New Molecular Entities AZD0284 AZD8154 abediterol# MEDI3506 anifrolumab# TULIP RORg psoriasis / respiratory Inhaled PI3Kgd asthma LABA a
Slide 5: Q2 2020 lifecycle management (LCM) pipeline
Q2 2020 lifecycle management (LCM)1 pipeline Phase I Phase II Phase III Under Review 1 Project 8 Projects 23 Projects 1 Project Imfinzi#+azacitidine# Calquence CALAVI-US & CALAVI-RoW Calquence# Imfinzi# post-SBRT PACIFIC-4 Imfinzi#+VEGF+TACE EMERALD-1 Enhertu¶# DESTINY-Gastric01 PD-L1+azacitidine MD
Slide 6: Q2 2020 lifecycle management (LCM) pipeline
Q2 2020 lifecycle management (LCM)1 pipeline Phase I Phase II Phase III Under Review 0 Projects 2 Projects 9 Projects 3 Projects Breztri Farxiga/Forxiga Dapa-CKD Brilinta/Brilique THALES LABA/LAMA/ICS asthma SGLT2 CKD P2Y12 stroke roxadustat# Farxiga/Forxiga DELIVER Nexium (CN only) HIF-PH inhibitor
Slide 7: Estimated key regulatory submission acceptances
Estimated key regulatory submission acceptances capivasertib + fulvestrant locally advanced or Fasenra mBC CAPItello-291 severe asthma (China) anifrolumab SLE capivasertb + CTx 1L mTNBC nirsevimab TULIP CAPItello-290 passive RSV immunisation NME tezepelumab asthma Imfinzi + tremelimumab + SoC urothe
Slide 8: Designations
Designations 5 15 12 30 29 Accelerated approvals Breakthrough / PRIME1 / Sakigake2 Fast Track Priority Review / RTOR3 Orphan Lynparza ovarian cancer SOLO-2 (US) Tagrisso EGFRm T790M NSCLC (US) MEDI3902 Psl-PcrV pneumo Px (US) Tagrisso EGFRm T790M NSCLC (JP) Lynparza ovarian cancer SOLO-2 (US) Tagris
Slide 9: Oncology - approved medicines and late-stage pipeline
Oncology - approved medicines and late-stage pipeline
Slide 10: Tagrisso (NSCLC)
Approved medicines Oncology Tagrisso (highly-selective, irreversible EGFRi) Late-stage development Early development NSCLC Trial Population Patients Design Endpoints Status CVRM Phase III Adjuvant EGFRm NSCLC 682 • Arm 1: Tagrisso QD following complete tumour resection, with • Primary endpoint: DFS
Slide 11: Tagrisso (NSCLC, combinations)
Approved medicines Oncology Tagrisso (highly-selective, irreversible EGFRi) Late-stage development Early development NSCLC, combinations Trial Population Patients Design Endpoints Status CVRM Phase III Neoadjuvant EGFRm NSCLC 351 Arm 1: placebo plus plus Pemetrexed/Carboplatin or • Primary endpoint:
Slide 12: Imfinzi (NSCLC, early disease)
Approved medicines Oncology Imfinzi (PD-L1 mAb) Late-stage development Early development NSCLC, early disease Trial Population Patients Design Endpoints Status CVRM Phase III Completely resected 332 • Arm 1: Imfinzi + SoCd chemo Primary endpoint: • Initiating MERMAID-1 Stage II and III NSCLC • Arm 2
Slide 13: Imfinzi +/- treme (lung cancer, advanced)
Approved medicines Oncology Imfinzi (PD-L1 mAb) +/- treme (CTLA-4 mAb) Late-stage development Early development Lung cancer, advanced disease Trial Population Patients Design Endpoints Status CVRM Phase III NSCLC 1L 650 • Arm 1: Imfinzi Q4W Primary endpoint: • FPCD: Q1 2017 PEARL • Arm 2: chemothera
Slide 14: Imfinzi (other cancers, early disease)
Approved medicines Oncology Imfinzi (PD-L1 mAb) Late-stage development Early development Other cancers, early disease Trial Population Patients Design Endpoints Status CVRM Phase III Non-muscle invasive bladder 975 • Arm 1: BCG (Induction + maintenance) Primary endpoints: • FPCD: Q2 2018 POTOMAC can
Slide 15: Imfinzi +/- treme (other cancers, advanced)
Approved medicines Oncology Imfinzi (PD-L1 mAb) +/- treme (CTLA-4 mAb) Late-stage development Early development Other cancers, advanced disease Trial Population Patients Design Endpoints Status CVRM Phase III Cis-eligible and ineligible 1,005 • Arm 1: Imfinzi + tremelimumab Primary endpoints: • FPCD
Slide 16: Imfinzi +/- treme (other cancers, advanced)
Approved medicines Oncology Imfinzi (PD-L1 mAb) +/- treme (CTLA-4 mAb) Late-stage development Early development Other cancers, advanced disease Trial Population Patients Design Endpoints Status CVRM Phase III Advanced solid malignancies 1,200 • Arm 1: Imfinzi • Primary endpoint: Safety • FPCD: Q2 20
Slide 17: Lynparza (other cancers)
Approved medicines Oncology Lynparza (PARP inhibitor) Late-stage development Early development Other cancers Trial Population Patients Design Endpoints Status CVRM Phase III BRCAm adjuvant breast 1,836 • Arm 1: Lynparza BiD 12 month duration • Primary endpoint: invasive disease-free • FPCD: Q2 2014
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 Advanced ovarian cancer 1L 1,256 Non tBRCAm (tumour BRCA) patients Primary endpoint: • FPCD: Q1 2019 DuO-O • Arm 1: bev
Slide 19: Lynparza (other combinations)
Approved medicines Oncology Lynparza (PARP inhibitor) Late-stage development Early development Other combinations Trial Population Patients Design Endpoints Status CVRM Phase III Advanced ovarian cancer 1L 806 • Arm 1: Lynparza maintenance therapy for two years or until Primary endpoint: • FPCD: Q2
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 II HER2-positive, unresectable and/or 230 Randomised, open label, sequential assignment Primary endpoint ORR • FP
Slide 21: 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 II HER2-overexpressing advanced gastric 220 Randomised open label parallel assignment Primary end point ORR • FP
Slide 22: Enhertu (other cancers)
Approved medicines Oncology Enhertu (trastuzumab deruxtecan, HER2 ADC) Late-stage development Early development Other cancers Trial Population Patients Design Endpoints Status CVRM Phase II HER2 expressing tumours 280 Non randomised single group assignment Primary endpoint: ORR • Initiating DPT02 •
Slide 23: Calquence (blood cancers)
Approved medicines Oncology Calquence (BTK inhibitor) Late-stage development Early development Blood cancers Trial Population Patients Design Endpoint(s) Status CVRM Phase III Previously untreated CLL 535 • Arm A: chlorambucil + obinutuzumab • Primary endpoint: PFS (Arm A vs. Arm B) • FPCD: Q2 2015
Slide 24: Calquence (blood cancers)
Approved medicines Oncology Calquence (BTK inhibitor) Late-stage development Early development Blood cancers Trial Population Patients Design Endpoint(s) Status CVRM Phase I/II B-cell malignancies 40 Dose escalation and expansion trial of the combination of • Safety • FPCD: Q1 2015 ACE-LY-001 Calque
Slide 25: Calquence (blood and other cancers)
Approved medicines Oncology Calquence (BTK inhibitor) Late-stage development Early development Blood and other cancers Trial Population Patients Design Endpoint(s) Status CVRM Phase I Japanese adults with 34 • Calquence monotherapy • Safety • FPCD: Q2 2017 advanced B-cell malignancies • Dose confirm
Slide 26: Koselugo (paediatric NF1, solid tumours)
Approved medicines Oncology Koselugo (selumetinib, MEK inhibitor) Late-stage development Early development Paediatric neurofibromatosis type 1, solid tumours Trial Population Patients Design Endpoints Status CVRM Phase II Paediatric NF1 50 (stratum 1) • Single arm: Koselugo 25mg/m2 BID with 2 strata
Slide 27: Lumoxiti (blood cancer)
Approved medicines Oncology Lumoxiti (moxetumomab pasudotox,CD22 mAb) Late-stage development Early development Blood cancer Trial Population Patients Design Endpoints Status CVRM Phase III Adults with relapsed or 80 • Multicentre, single-arm, open-label Phase III trial • Primary endpoint: rate of du
Slide 28: Savolitinib (NSCLC and other cancers)
Approved medicines Oncology Late-stage development Savolitinib (MET inhibitor) Early development NSCLC and other cancers Trial Population Patients Design Endpoints Status CVRM Phase I Advanced NSCLC 85 • Dose escalation trial • Primary endpoint: safety and tolerability • FPCD: Q2 2013 (all comers) •
Slide 29: Capivasertib (breast cancer, prostate cancer)
Approved medicines Oncology Late-stage development Capivasertib (AKT inhibitor) Early development Breast cancer, prostate cancer Trial Population Patients Design Endpoints Status CVRM Phase III Locally advanced or 800 Double-blind randomised comparative trial • PFS • FPCD Q3 2019 CAPItello-290 metas
Slide 30: Oncology - early-stage development
Oncology - early-stage development
Slide 31: Imfinzi (cancer)
Approved medicines Oncology Imfinzi (PD-L1 mAb) Late-stage development Early development Cancer Trial Compound Population Patients Design Endpoints Status CVRM Phase I/II Imfinzi Solid tumours 1,022 • Dose escalation: 5 cohorts at Q2W and 1 cohort at Q3W • Safety • FPCD: Q3 2012 STUDY 1108 • Dose ex
Slide 32: Imfinzi (cancer)
Approved medicines Oncology Imfinzi (PD-L1 mAb) Late-stage development Early development Cancer Trial Compound Population Patients Design Endpoints Status CVRM Phase Ib/II Imfinzi 1L metastatic MSS- 112 • Part 1 S1 FOLFOX + bevacizumab + Imfinzi + oleclumab Primary • FPCD: Q3 2019 COLUMBIA 1 CRC • P
Slide 33: Imfinzi + tremelimumab
Approved medicines Oncology Imfinzi (PD-L1 mAb) + Late-stage development Early development tremelimumab (CTLA-4 mAb) Cancer CVRM Trial Population Patients Design Endpoints Status Phase Ib/II Hepatocellular carcinoma 545 • Arm A: Imfinzi + tremelimumab • Primary endpoints: Safety & tolerability, • FP
Slide 34: Imfinzi + monalizumab
Approved medicines Oncology Imfinzi (PD-L1 mAb) + Late-stage development Early development monalizumab (NKG2a mAb) Cancer CVRM Trial Population Patients Design Endpoints Status Phase I/II Advanced solid tumours 381 Escalation phase Primary endpoints: • FPCD: Q2 2016 • monalizumab + Imfinzi i.v. • Sa
Slide 35: Imfinzi + MEDI0457 (HNSCC)
Approved medicines Oncology Imfinzi (PD-L1 mAb) + Late-stage development Early development MEDI0457 (DNA HPV Vaccine) Head and neck squamous cell carcinoma (HNSCC) CVRM Trial Population Patients Design Endpoints Status Phase Ib/IIa HPV associated 50 Multi-centre, open label trial to evaluate the saf
Slide 36: AZD0466 (Bcl2/xL inhibitor)
Approved medicines Oncology Late-stage development AZD0466 (Bcl2/xL inhibitor) Early development Cancer Trial Population Patients Design Endpoints Status CVRM Phase I Advanced hematologic 102 Monotherapy dose escalation, consisting of two arms: • Primary: safety • FPCD: Q4 2019 malignancies or solid
Slide 37: MEDI1191 (IL12 modRNA)
Approved medicines Oncology Late-stage development MEDI1191 (IL12 modRNA) Early development Cancer Trial Population Patients Design Endpoints Status CVRM Phase I Advanced solid tumours 87 First-time-in-human Phase I, open-label, dose-escalation and • Primary endpoint: safety and tolerability • FPCD:
Slide 38: AZD1390 (ATM inhibitor)
Approved medicines Oncology Late-stage development AZD1390 (ATM inhibitor) Early development Cancer Trial Population Subjects Design Endpoints Status CVRM Phase I Recurrent glioblastoma 132 • Primary: investigate the safety, • FPCD Q2 2018 eligible for re-irradiation, brain • Designed to evaluate th
Slide 39: Adavosertib (WEE-1 inhibitor)
Approved medicines Oncology Late-stage development Adavosertib (AZD1775, WEE-1 inhibitor) Early development Ovarian cancer, solid tumours Trial Population Patients Design Endpoints Status CVRM Phase II Platinum-resistant (PR) 95 • Arm B: paclitaxel + adavosertib • Primary endpoint: ORR • FPCD: Q1 20
Slide 40: MEDI2228 (BCMA ADC)
Approved medicines Oncology Late-stage development MEDI2228 (BCMA antibody drug conjugate) Early development Cancer Trial Population Patients Design Endpoints Status CVRM Phase I Relapsed/refractory multiple 129 First-time-in-human Phase I, multi-centre, open-label, single-arm, Primary endpoints: •
Slide 41: AZD2811NP (AURN)
Approved medicines Oncology Late-stage development AZD2811NP (AURN) Early development Cancer Trial Population Patients Design Endpoints Status CVRM Phase I Solid tumours 72 • Arm 1: AZD2811NP dose escalation • Safety and tolerability • FPCD: Q4 2015 • Arm 2: AZD2811NP dose expansion SCLC • PK and ef
Slide 42: AZD4573 (CDK9 inhibitor)
Approved medicines Oncology Late-stage development AZD4573 (CDK9 inhibitor) Early development Cancer Trial Population Patients Design Endpoints Status CVRM Phase I Relapsed/refractory 45 Dose escalation in relapsed/refractory haematological Primary: • FPCD: Q4 2017 haematologic malignancies malignan
Slide 43: AZD4635 (A2AR inhibitor)
Approved medicines Oncology Late-stage development AZD4635 (A2AR inhibitor) Early development Cancer Trial Population Patients Design Endpoints Status CVRM Phase I Phase Ia: patients with 3067 Phase Ia – solid tumours or mCPRC: Primary outcome measure: • FPCD: Q2 2016 advanced solid tumours • AZD463
Slide 44: AZD5153 (BRD4 inhibitor)
Approved medicines Oncology Late-stage development AZD5153 (BRD4 inhibitor) Early development Cancer Trial Population Patients Design Endpoints Status CVRM Phase I/Ib Relapsed/refractory solid 60 Monotherapy dose escalation in advanced solid tumours and • Primary: safety • FPCD: Q2 2017 tumours, lym
Slide 45: MEDI5395 (rNDV GMCSF)
Approved medicines Oncology Late-stage development MEDI5395 (rNDV GMCSF) Early development Cancer Trial Population Patients Design Endpoints Status CVRM Phase I Select advanced solid 164 First-time-in-human Phase I, open-label, dose-escalation and • Primary endpoint: safety and tolerability • FPCD:
Slide 46: MEDI5752 (PD-1/CTLA-4 bispecific mAb)
Approved medicines Oncology Late-stage development MEDI5752 (PD-1/CTLA-4 bispecific mAb) Early development Cancer Trial Population Patients Design Endpoints Status CVRM Phase I Advanced solid tumours 272 Open-label, dose-escalation and dose-expansion: Primary endpoints: • FPCD: Q2 2018 • dose-escala
Slide 47: AZD5991 (MCL1 inhibitor)
Approved medicines Oncology Late-stage development AZD5991 (MCL1 inhibitor) Early development Cancer Trial Population Patients Design Endpoints Status CVRM Phase I/Ib/IIa Relapsed/refractory 177 • Arm1: monotherapy dose escalation & expansions in • Primary: safety • FPCD: Q3 2017 haematologic malign
Slide 48: Ceralasertib (ATR inhibitor)
Approved medicines Oncology Late-stage development Ceralasertib (AZD6738, ATR inhibitor) Early development Cancer Trial Population Patients Design Endpoints Status CVRM Phase I Solid tumours 250 • Arm 1: ceralasertib + carboplatin • Safety and tolerability • FPCD: Q4 2014 • Arm 2: ceralasertib dose
Slide 49: AZD7648 (DNA-PK inhibitor)
Approved medicines Oncology Late-stage development AZD7648 (selective DNA-PK inhibitor) Early development Advanced solid tumours Trial Population Patients Design Endpoints Status CVRM Phase I Advanced Malignancies 234 • This is a modular Phase I/IIa, open-label, multi-centre, trial of • Primary outc
Slide 50: Danvatirsen (STAT3 inhibitor)
Approved medicines Oncology Late-stage development Danvatirsen (AZD9150, STAT3 inhibitor) Early development Cancer Trial Population Patients Design Endpoints Status CVRM Phase Ib/II HNSCC 339 Dose escalation advanced solid and blood cancers • Safety/efficacy trial • FPCD: Q3 2015 • Arm A1: AZD9150/I
Slide 51: Oleclumab (CD73 mAb)
Approved medicines Oncology Late-stage development Oleclumab (MEDI9447, CD73 mAb) Early development Cancer Trial Population Patients Design Endpoints Status CVRM Phase I Advanced malignancies 348 Dose escalation phase Primary endpoints: • FPCD: Q3 2015 • oleclumab i.v. • Safety • Data anticipated: 2
Slide 52: AZD9496 (oral SERD)
Approved medicines Oncology Late-stage development AZD9496 (SERD, oral) Early development Breast cancer Trial Population Patients Design Endpoints Status CVRM Phase I ER+ breast cancer c. 50 • This is an open label randomised multicentre pre-surgical • Primary outcome measures: PD changes • FPCD: Q4
Slide 53: AZD9833 (oral SERD)
Approved medicines Oncology Late-stage development AZD9833 (SERD, oral) Early development Breast cancer Trial Population Patients Design Endpoints Status CVRM Phase I ER+ breast cancer 240 • This is a Phase I open label multicentre trial of AZD9833 • Primary outcome measures: safety and • FPCD: Q4 2
Slide 54: IPH5201 (CD39 mAb)
Approved medicines Oncology Late-stage development IPH5201 (CD39 mAb) Early development Solid Tumour Trial Population Patients Design Endpoints Status CVRM Phase I Advanced Solid tumours 204 • First time in human Phase I, open-label, dose- Primary endpoints: • FPCD: Q1 2020 escalation trial to deter
Slide 55: BioPharmaceuticals - approved medicines and late-stage pipeline
BioPharmaceuticals - approved medicines and late-stage pipeline
Slide 56: Farxiga (heart failure and CKD)
Approved medicines Oncology Farxiga (SGLT2 inhibitor) Late-stage development Early development Heart failure and chronic kidney disease Trial Population Patients Design Endpoints Status CVRM Phase III CHF patients with HFrEF 4,744 • Arm 1: Farxiga 10mg or 5 mg QD + SoC therapy • Primary endpoint: ti
Slide 57: Brilinta (cardiovascular risk reduction)
Approved medicines Oncology Brilinta (P2Y12 receptor antagonist) Late-stage development Early development Cardiovascular risk reduction Trial Population Patients Design Endpoints (primary) Status CVRM Phase III Patients with type-2 diabetes 19,000 • Arm 1: Brilinta 60mg BiD • Primary endpoint: compo
Slide 58: Lokelma (hyperkalaemia)
Approved medicines Oncology Lokelma (sodium zirconium cyclosilicate) Late-stage development Early development Hyperkalaemia Trial Population Patients Design Endpoints Status CVRM Phase II Patients with chronic heart 182 • Arm 1: Lokelma 5g QD for 12 weeks. Option to uptitrate to 10 • Primary endpoin
Slide 59: Roxadustat (anaemia)
Approved medicines Oncology Late-stage development Roxadustat (HIF-PH inhibitor) Early development Anaemia Trial Population Patients Design Endpoints Status CVRM Phase III Anaemia in CKD in patients 922 • Arm 1: roxadustat • Primary endpoint: Haemoglobin response • FPCD: Q4 2012 ANDES not receiving
Slide 60: Roxadustat (anaemia)
Approved medicines Oncology Late-stage development Roxadustat (HIF-PH inhibitor) Early development Anaemia Trial Population Patients Design Endpoints Status CVRM Phase III Anaemia in newly initiated 1,043 • Arm 1: roxadustat • Primary endpoint: Haemoglobin response • FPCD: Q4 2013 HIMALAYAS dialysis
Slide 61: Eklira/Tudorza (COPD)
Approved medicines Oncology Eklira/Tudorza (LAMA, DPI) Late-stage development Early development COPD Trial Population Number of patients Design Endpoints Status CVRM Phase I Healthy Chinese 18 Open-label, 2-period ascending dose incomplete block, cross-over • To investigate the PK of aclidinium • In
Slide 62: Duaklir Genuair (COPD)
Approved medicines Oncology Duaklir Genuair (LAMA/LABA, DPI) Late-stage development Early development COPD Trial Population Patients Design Endpoints Status CVRM Phase III Patients with stable COPD 1,060 • Arm 1: Duaklir Genuair 400/12 μg DPI Primary endpoints: • FPCD: Q1 2017 AVANT • Arm 2: aclidin
Slide 63: Breztri (COPD)
Approved medicines Oncology Breztri (PT010, LAMA/LABA/ICS, pMDI) Late-stage development Early development COPD Trial Population Patients Design Endpoints Status CVRM Phase III Moderate to very severe 500 Treatments (52-week treatment period) Primary endpoints: • FPCD: Q3 2015 COPD • BGF (budesonide,
Slide 64: Daliresp/Daxas (COPD)
Approved medicines Oncology Daliresp/Daxas (PDE4 inhibitor, oral) Late-stage development Early development COPD Trial Population Patients Design Endpoints Status CVRM Post Launch COPD 124,080 • This is a retrospective cohort trial comparing COPD patients • Primary endpoint: all-cause mortality (up •
Slide 65: Fasenra (severe, uncontrolled asthma)
Approved medicines Oncology Fasenra (IL5R mAb) Late-stage development Early development Severe, uncontrolled asthma Trial Population Patients Design Endpoints Status CVRM Phase III A multi-centre, open-label, 447 • Arm 1: Fasenra 30mg Q4W s.c. • Primary endpoint: safety and tolerability • FPCD: Q2 2
Slide 66: Fasenra (severe, uncontrolled asthma)
Approved medicines Oncology Fasenra (IL5R mAb) Late-stage development Early development Severe, uncontrolled asthma Trial Population Patients Design Endpoints Status CVRM Phase III Severe asthma, inadequately 2,133 • Arm 1: Fasenra 30mg Q4W s.c. • Primary endpoint: safety and tolerability • FPCD: Q4
Slide 67: Fasenra (severe, uncontrolled asthma)
Approved medicines Oncology Fasenra (IL5R mAb) Late-stage development Early development Severe, uncontrolled asthma Trial Population Patients Design Endpoints Status CVRM Phase III Severe asthma on ICS-LABA 121 Open label Fasenra 30mg Q4w • Primary endpoint: percentage of patients/ • FPCD: Q4 2016 G
Slide 68: Fasenra (nasal polyposis, eosinophilic diseases)
Approved medicines Oncology Fasenra (IL5R mAb) Late-stage development Early development Nasal polyposis and other eosinophilic diseases Trial Population Patients Design Endpoints Status CVRM Phase III Patients with severe bilateral 413 • Arm 1: Fasenra 30mg Q8W s.c. • Primary endpoint: effect of Fas
Slide 69: Fasenra (COPD)
Approved medicines Oncology Fasenra (IL5R mAb) Late-stage development Early development COPD Trial Population Patients Design Endpoints Status CVRM Phase III Patients with moderate to very 1216 • Double-blind, placebo controlled, single dose (100mg q8w) • Primary endpoint: annualized rate of • FPCD
Slide 70: Tezepelumab (severe asthma)
Approved medicines Oncology Late-stage development Tezepelumab (TSLP mAb) Early development Severe, uncontrolled asthma Trial Population Patients Design Endpoints Status CVRM Phase III Severe asthma 1,061 • Arm 1: tezepelumab s.c. • Primary endpoint: Annual asthma • FPCD: Q1 2018 NAVIGATOR Age 12-80
Slide 71: Tezepelumab (severe asthma)
Approved medicines Oncology Late-stage development Tezepelumab (TSLP mAb) Early development Severe, uncontrolled asthma CVRM Trial Population Patients Design Endpoints Status Phase II Severe asthma 116 • Arm 1: tezepelumab s.c. • Primary endpoint: number of airway • FPCD: Q4 2018 CASCADE Age 18-75 y
Slide 72: PT027 (asthma)
Approved medicines Oncology Late-stage development PT027 (SABA/ICS, pMDI) Early development Asthma Trial Population Patients Design Endpoints Status CVRM Phase III Moderate to severe asthma 3,100 Treatments (minimum 24-week treatment period) Primary endpoint: • FPCD: Q4 2018 MANDALA • BDA (budesonid
Slide 73: Anifrolumab (lupus)
Approved medicines Oncology Late-stage development Anifrolumab (type I interferon receptor mAb) Early development Lupus (SLE / LN) Trial Population Patients Design Endpoints Status CVRM Phase III Moderate to severe SLE 450 • Arm 1: 300mg i.v. anifrolumab Q4W for 48 weeks • Primary endpoint: response
Slide 74: Brazikumab (inflammatory bowel disease)
Approved medicines Oncology Late-stage development Brazikumab (IL-23 inhibitor) Early development Inflammatory Bowel Disease (Crohn’s Disease, Ulcerative Colitis) Trial Population Patients Design Endpoints Status CVRM Phase IIb / III Crohn’s Disease 1,140 • Arm 1: brazikumab 1440 mg IV induction + 2
Slide 75: Nirsevimab (RSV)
Approved medicines Oncology Late-stage development Nirsevimab (MEDI8897, Respiratory syncytial virus mAb-YTE ) Early development Infection Trial Population Patients Design Endpoints Status CVRM Phase IIb 29-35 WK GA (Gestational 1,453 • Randomised, double-blind, placebo-controlled trial • Safety and
Slide 76: AZD1222 (prevention of COVID-19)
Approved medicines Oncology Late-stage development AZD1222 (SARS-CoV-02) Early development Prevention of COVID-19 CVRM Trial Population Patients Design Endpoints Status Phase I/II Healthy adults 1,077 Single-blinded, randomised, controlled, multi-centre trial • Primary endpoint: efficacy and safety
Slide 77: COVID-19 trials
Approved medicines Oncology Late-stage development COVID-19 trials Early development Prevention of COVID-19 Trial Compound Population Patients Design Endpoints Status CVRM Phase IIb Calquence COVID-19 60 • Arm 1: Calquence + best supportive care • Primary outcomes: safety, subject alive • FPCD: Q2 2
Slide 78: BioPharmaceuticals - early-stage development
BioPharmaceuticals - early-stage development
Slide 79: Cotadutide (diabetes/obesity)
Approved medicines Oncology Late-stage development Cotadutide (MEDI0382, GLP-1-glucagon agonist) Early development Diabetes/obesity Trial Population Patients Design Endpoints Status CVRM Phase II Adults with type-2 65 • Arm1: cotadutide s.c. or placebo • Primary: efficacy MMT glucose AUC, body weigh
Slide 80: Cotadutide (diabetes/obesity, NASH)
Approved medicines Oncology Late-stage development Cotadutide (MEDI0382, GLP-1-glucagon agonist) Early development Diabetes/obesity, NASH Trial Population Patients Design Endpoints Status CVRM Phase II Overweight and 27 • Cotadutide or placebo s.c. • Primary: efficacy body weight loss • FPCD: Q4 201
Slide 81: Verinurad (CKD)
Approved medicines Oncology Late-stage development Verinurad (RDEA3170, URAT1 inhibitor) Early development CKD Trial Population Patients Design Endpoints Status CVRM Phase II CKD patients with 60 • Arm A: verinurad 9 mg and febuxostat 80 mg To assess the effects of intensive uric acid • FPCD: Q2 201
Slide 82: Verinurad (HFpEF)
Approved medicines Oncology Late-stage development Verinurad (RDEA3170, URAT1 inhibitor) Early development HFpEF Trial Population Patients Design Endpoints Status CVRM Phase II Patients with heart failure with 435 • Arm A: verinurad 12 mg + allopurinol 300 mg Peak V02 Change from baseline at Week •
Slide 83: AZD2373 (chronic kidney disease)
Approved medicines Oncology Late-stage development AZD2373 Early development Chronic Kidney Disease Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers 48 SAD Primary: • FPCD: Q1 2020 • Safety and tolerability NCT04269031 Dose escalation in 6 cohorts with 6 volunteers r
Slide 84: AZD2693 (NASH)
Approved medicines Oncology Late-stage development AZD2693 (resolution of NASH) Early development NASH Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers 48 SAD Primary: • FPCD: Q4 2019 • Safety and tolerability • Data anticipated: H2 2020 NCT04142424 6 cohorts with 6
Slide 85: MEDI3506 (diabetic kidney disease)
Approved medicines Oncology Late-stage development MEDI3506 (IL33 ligand mAb) Early development Diabetic Kidney Disease (DKD) Trial Population Patients Design Endpoints Status CVRM Phase II Adult patients with diabetic 168 Randomized, double-blind, placebo-controlled, multicenter trial to • Efficacy
Slide 86: AZD4831 (cardiovascular disease)
Approved medicines Oncology Late-stage development AZD4831 (MPO inhibitor) Early development Cardiovascular disease Trial Population Patients Design Endpoints Status CVRM Phase I Healthy patients c. 96 SAD trial (one trial site in Germany) • Safety and tolerability • FPCD: Q3 2016 • Planned to inves
Slide 87: AZD5718 (cardiovascular disease)
Approved medicines Oncology Late-stage development AZD5718 (FLAP inhibitor) Early development Cardiovascular disease Trial Population Patients Design Endpoints Status CVRM Phase IIa CAD 129 • Arm 1: AZD5718 Dose A • Primary endpoint: PD effect of AZD5718 • FPCD: Q4 2017 • Arm 2: AZD5718 Dose B by as
Slide 88: AZD6615 (hypercholesterolemia)
Approved medicines Oncology Late-stage development AZD6615 (anti-hypercholesterolemia) Early development Hypercholesterolemia Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers 40 SAD Primary: • FPCD: Q3 2019 • Safety and tolerability NCT04055168 3 cohorts of non-Asian
Slide 89: AZD8233 (hypercholesterolemia)
Approved medicines Oncology Late-stage development AZD8233 (anti-hypercholesterolemia) Early development Hypercholesterolemia Trial Population Patients Design Endpoints Status CVRM Phase I Healthy subjects 72 SAD Primary: • FPCD: Q3 2018 • Safety and tolerability NCT03593785 7 cohorts with 6 subject
Slide 90: AZD8601 (cardiovascular disease)
Approved medicines Oncology Late-stage development AZD8601 (VEGF-A modified RNA) Early development Cardiovascular disease Trial Population Patients Design Endpoints Status CVRM Phase I Type 2 diabetic patients c. 60 SAD trial (one trial site in Germany) • Safety and tolerability • FPCD: Q1 2017 • Pl
Slide 91: AZD9977 (heart failure)
Approved medicines Oncology Late-stage development AZD9977 Early development Heart failure Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers 27 MAD Primary: • FPCD: Q1 2018 • Safety and tolerability • LPCD: Q2 2018 NCT03435276 Dose escalation in 3 cohorts with 6 subje
Slide 92: Biologics (cardiovascular & metabolic diseases)
Approved medicines Oncology Late-stage development Biologics Early development Cardiovascular & metabolic diseases Trial Compound Population Patients Design Endpoints Status CVRM Phase IIb MEDI6012 Subjects 30-80 540 • Cohort A: 2-dose regimen Primary endpoints: Infarct size as a percentage of left
Slide 93: AZD0449 (asthma)
Approved medicines Oncology Late-stage development AZD0449 (inhaled JAK-1 inhibitor) Early development Asthma Trial Population Patients Design Endpoints Status CVRM Phase I Healthy subjects and patients 156 SAD/MAD/Bridge trial (UK) Primary endpoint: • FPCD: Q4 2018 with mild asthma Part 1 SAD • Saf
Slide 94: AZD1402 (asthma)
Approved medicines Oncology Late-stage development AZD1402 (IL4 receptor antagonist) Early development Asthma Trial Population Patients Design Endpoints Status CVRM Phase Ib Patients with mild asthma 75 PoM. Primary endpoint: • FPCD: Q3 2018 A dose-escalating, single blind trial to assess the • Safe
Slide 95: MEDI3506 (COPD, atopic dermatitis)
Approved medicines Oncology Late-stage development MEDI3506 (IL33 ligand mAb) Early development COPD, atopic dermatitis Trial Population Patients Design Endpoints Status CVRM Phase I (Combined SAD / SAD: healthy subjects with SAD: 56 SAD: • Safety and tolerability • FPCD: Q2 2017 MAD) mild atopy • 7
Slide 96: Velsecorat (asthma)
Approved medicines Oncology Late-stage development Velsecorat (AZD7594, SGRM, inhaled) Early development Asthma Trial Population Patients Design Endpoints Status CVRM Phase I Adolescent asthma patients 24 An open-label, multi-centre, Phase I trial to assess the PK, PD Primary endpoint: • FPCD: Q3 20
Slide 97: AZD7986 (COPD)
Approved medicines Oncology Late-stage development AZD7986 (DPP1) Early development COPD Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers 15 This is a phase I, non-randomised, fixed sequence, 3-period, • Safety and tolerability • FPCD: Q1 2016 drug-drug interaction t
Slide 98: AZD8154 (asthma)
Approved medicines Oncology Late-stage development AZD8154 (PI3Kδ inhibitor) Early development Asthma Trial Population Patients Design Endpoints Status CVRM Phase I Healthy subjects 78 SAD/MAD Primary endpoint: • FPCD: Q3 2018 A Phase I trial to assess the safety, tolerability and PK of • Safety an
Slide 99: AZD8871 (respiratory)
Approved medicines Oncology Late-stage development AZD8871 (MABA, inhaled) Early development Respiratory Trial Population Patients Design Endpoints Status CVRM Phase IIa Patients with COPD 73 Randomised, double-blind, placebo and active-controlled Primary endpoint: • FPCD: Q4 2018 crossover trial. E
Slide 100: AZD9567 (respiratory)
Approved medicines Oncology Late-stage development AZD9567 (SGRM, oral) Early development Respiratory Trial Population Patients Design Endpoints Status CVRM Phase I Healthy subjects 71 MAD trial with a total of 6 dose levels of AZD9567: 10 mg, 20mg, Primary endpoint: • FPCD: Q2 2016 40mg, 80mg and 1
Slide 101: AZD0284 (plaque psoriasis)
Approved medicines Oncology Late-stage development AZD0284 (RORγ inverse agonist) Early development Plaque psoriasis vulgaris Trial Population Patients Design Endpoints Status CVRM Phase I Healthy subjects 80 Part 1 (SAD) • Safety and tolerability and PK following • FPCD: Q3 2016 • Seven different d
Slide 102: MEDI0618 (osteoarthritis pain)
Approved medicines Oncology Late-stage development MEDI0618 (PAR2 antagonist mAb) Early development Osteoarthritis pain Trial Population Patients Design Endpoints Status CVRM Phase I Painful osteoarthritis of the knee 64 (healthy • SAD • Safety, tolerability and PK • FPCD: Q4 2019 volunteers) • Up t
Slide 103: MEDI1341 (Parkinson's disease)
Approved medicines Oncology Late-stage development MEDI1341 (alpha-synuclein mAb) Early development Parkinson’s Disease Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers 48 • SAD • Safety, tolerability, PK, PD • FPCD: Q4 2017 • Up to 6 i.v. cohorts are planned vs. pla
Slide 104: AZD4041 (opioid use disorder)
Approved medicines Oncology Late-stage development AZD4041 (orexin 1 receptor antagonist) Early development Opioid use disorder Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers 48 healthy • Randomised, double blind, single ascending dose • Safety, tolerability, PK, P
Slide 105: AZD5634 (cystic fibrosis)
Approved medicines Oncology Late-stage development AZD5634 (ENaC, inhaled) Early development Cystic Fibrosis Trial Population Patients Design Endpoints Status CVRM Phase I Healthy volunteers 56 A randomised, single-blind, placebo-controlled trial to assess the • Safety and tolerability • FPCD: Q1 20
Slide 106: MEDI7352 (osteoarthritis pain)
Approved medicines Oncology Late-stage development MEDI7352 (NGF TNF bispecific mAb) Early development Osteoarthritis pain Trial Population Patients Design Endpoints Status CVRM Phase I Painful osteoarthritis of the knee 160 • SAD & MAD • Safety, tolerability, PK, PD • FPCD: Q1 2016 • Up to 12 i.v.
Slide 107: Other biologics (infections)
Approved medicines Oncology Late-stage development Other biologics Early development Infections Trial Compound Population Patients Design Endpoints Status CVRM Phase II Anti-Staph AT Intubated ICU 213 • Placebo-controlled, single-dose, dose-ranging • Efficacy and safety • FPCD: Q4 2014 (suvratoxumab
Slide 108: List of abbreviations
List of abbreviations 14C Radioactive isotope of carbon, Carbon 14 CHF Chronic heart failure FLAP 5-lipoxygenase-activating protein 1L, 2L, 3L 1st, 2nd or 3rd line CKD Chronic kidney disease FPDC First patient commenced dosing 5-FU 5-fluorouracil CLL Chronic lymphocytic leukaemia FPG Fasting plasma
Slide 109: List of abbreviations
List of abbreviations LABA Long acting beta agonist PASI Psoriasis area severity index SAE Serious adverse event LAMA Long acting muscarinic agonist PBD Pyrrolobenzodiazepine SBRT Stereotactic body radiation therapy LCAT Lecithin-cholesterol acyltransferase pCR Pathological complete response s.c. Su
Slide 110: Clinical trials appendix H1 2020 results update
Clinical trials appendix H1 2020 results update