Clinical trials appendix Q1 2019 results update The following information about AstraZeneca clinical trials in Phases I-IV has been created with selected information from clinicaltrials.gov to facilitate understanding of key aspects of ongoing clinical programmes and is correct to the best of the Company’s knowledge as of 31 March 2019, unless otherwise specified.

It includes estimated timelines with regards to trial completion and first external presentations of primary data. These estimates are subject to change, as programmes recruit faster or slower than anticipated and many times are event driven.

Project postings on clinicaltrials.gov are updated on a continuous basis as projects progress. For the most up to date information on our clinical programmes please visit clinicaltrials.gov

2 List of abbreviations

ADA Anti-drug antibody ICS Inhaled corticosteroid pMDI Pressurised metered dose inhaler ADC Antibody-drug conjugate IM Intra muscular PoC Proof of concept AE Adverse event IR Immediate release PR Partial response AUC Area under curve IV Intravenous Q2W Quaque (every) two weeks BD/BID Bis in die (two times a day) LABA Long acting beta agonist Q3W Quaque (every) three weeks CE Clinically evaluable LAMA Long acting muscarinic agonist Q4W Quaque (every) four weeks CMAX Maximum concentration absorbed LCM Lifecycle management Q8W Quaque (every) eight weeks CNS Central nervous system LPCD Last patient commenced dosing QD Quaque die (one time a day) DCR Disease control rate MAD Multiple ascending dose QOD Quaque altera die (every other day) DDI Drug-drug interaction MDI Metered-dose inhaler QoL Quality of life DFS Disease free survival MTD Maximum tolerated dose SAD Single ascending dose DLT Dose-limiting toxicity NME New molecular entity SC Subcutaneous DoR Duration of response OCS Oral corticosteroid SoC Standard of care DPI Dry powder inhaler ORR Objective response rate TID Ter In die (three times a day) FDC Fixed-dose combination OS Overall survival VEGF Vascular endothelial growth factor FEV Forced-expiratory volume PARP Poly ADP ribose polymerase XR Extended release FPCD First patient commenced dosing PD Pharmacodynamics HRRm Homologous recombination repair mutation PFS Progression-free survival PK Pharmacokinetics 3 Table of contents slide

Movement since Q4 2018 update Q1 2019 NME pipeline Q1 2019 LCM pipeline

Oncology

Approved medicines and late-stage development Early-stage development

Cardiovascular, Renal & Metabolism (CVRM), Respiratory & Other medicines

Approved medicines and late-stage development Early-stage development

4 Movement since Q4 2018 update New to Phase I New to Phase II New to Pivotal Study New to Registration

Additional indication NME NME Lifecycle Management MEDI7247 trastuzumab deruxtecan¶# DESTINY-Breast01 Lynparza# + Imfinzi# + bevacizumab DUO-O Farxiga3 DECLARE-TIMI 58 [US & EU]1 ASCT2 antibody drug conjugate solid ADC HER2-positive, unresectable and/or metastatic breast cancer subjects previously treated PARP inhibitor + PD-L1 mAb + VEGF inhibitor SGLT2 inhibitor CV outcomes trial in patients tumours with trastuzumab emtansine 1st-line ovarian cancer with type-2 diabetes Additional indication Additional indication capivasertib# trastuzumab deruxtecan# DESTINY-Breast02 AKT inhibitor prostate cancer ADC HER2-positive, unresectable and/or metastatic Imfinzi# + Lynparza# ORION breast cancer pretreated with prior standard of care PD-L1 mAb + PARP inhibitor NSCLC HER2 therapies, including trastuzumab emtansine (oleclumab + chemotherapy) or (Imfinzi# + oleclumab + chemotherapy) trastuzumab deruxtecan# DESTINY-Breast03 (CD73 mAb+chemo) or (PD-L1 mAb+CD73 mAb+chemo) metastatic pancreatic cancer ADC HER2-positive, unresectable and/or metastatic ¶# breast cancer subjects previously treated with trastuzumab deruxtecan DESTINY-Gastric01 trastuzumab and taxane ADC HER2-overexpressing advanced gastric or gastroesophageal junction adenocarcinoma patients who have progressed on two prior treatment regimens trastuzumab deruxtecan# DESTINY-Breast04 # ADC HER2-low, unresectable and/or metastatic breast trastuzumab deruxtecan cancer subjects ADC HER2-expressing advanced colorectal cancer trastuzumab deruxtecan# HER2-over-expressing or -mutated, unresectable and/or metastatic NSCLC Lifecycle Management Calquence# + venetoclax + obinutuzumab Lifecycle Management BTK inhibitor + BCL-2 inhibitor + anti-CD20 mAb 1st-line Imfinzi# (platform) BEGONIA chronic lymphocytic leukaemia PD-L1 mAb breast cancer Imfinzi# CALLA Imfinzi# (platform) MEGELLAN PD-L1 mAb adjuvant locally-advanced cervical cancer PD-L1 mAb NSCLC Imfinzi# + (oleclumab or monalizumab#) COAST PD-L1 mAb + (CD73 mAb or NKG2A mAb) NSCLC Imfinzi# + (oleclumab or monalizumab# or danvatirsen#) NeoCOAST PD-L1 mAb + (CD73 mAb or NKG2A mAb or STAT3 inhibitor) NSCLC Lynparza# (basket) MK-7339-002 / LYNK002 PARP inhibitor HRRm cancer Removed from Phase I Removed from Phase II Removed from Phase III Removed from Registration

NME NME Lifecycle Management Imfinzi# + dabrafenib + trametinib AZD4547 Farxiga3 DEPICT [US & JP]2 PD-L1 mAb + BRAF inhibitor + MEK FGFR inhibitor solid tumours SGLT2 inhibitor type-1 diabetes inhibitor melanoma AZD8186 Imfinzi# + Iressa PI3k inhibitor solid tumours PD-L1 mAb + EGFR inhibitor NSCLC Imfinzi# + MEDI0680 AZD0156 PD-L1 mAb + PD-1 mAb solid tumours ATM inhibitor solid tumours prezalumab# AZD4785 B7RP1 mAb primary Sjöogren’s syndrome 5 KRAS inhibitor solid tumours ¶ Registrational Phase II/III trial # Partnered and/or in collaboration 1 Submission Accepted 2 Submission Approved 3Farxiga in the US; Forxiga in ROW Q1 2019 New Molecular Entity (NME)1 pipeline

Phase I Phase II Phase III Under Review 21 New Molecular Entities 25 New Molecular Entities 14 New Molecular Entities 0 New Molecular Entities

AZD1390 Imfinzi#+tremelimumab+chemo adavosertib#+chemotherapy Imfinzi#+tremelimumab trastuzumab deruxtecan# DESTINY-Breast02 glioblastoma PD-L1+CTLA-4 1L PDAC oesophageal SCLC Wee1+chemo ovarian cancer PD-L1+CTLA-4 gastric cancer ADC breast

AZD4573 Imfinzi+selumetinib# AZD2811# Imfinzi#+tremelimumab trastuzumab deruxtecan# DESTINY-Breast03 CDK9 haematalogical malignancies PL-L1+MEK solid tumours Aurora solid tumours PD-L1+CTLA-4 biliary tract oesophageal ADC breast

AZD5153 MEDI2228 AZD4635 Imfinzi+Lynparza# BAYOU trastuzumab deruxtecan# DESTINY-Breast04 BRD4 solid tumours BCMA ADC multiple myeloma A2aR inhibitor solid tumours PD-L1+PARP bladder ADC breast

AZD5991 MEDI3726# AZD6738 Lynparza#+adavosertib# Imfinzi#+tremelimumab DANUBE MCL1 haematalogical malignancies PSMA ADC prostate ATR solid tumours PARP+Wee1 solid tumours PD-L1+CTLA-4 1L bladder

AZD9496 MEDI5083 capivasertib# Lynparza#+AZD6738 Imfinzi#+tremelimumab HIMALAYA SERD ER+ breast CD40 ligand fusion protein solid tumours AKT breast prostate PARP+ATR gastric PD-L1+CTLA-4 1L HCC

Imfinzi#+(oleclumab or monalizumab# or AZD9833 MEDI5752 Lynparza#+AZD6738 or +adavosertib# VIOLETTE Imfinzi#+tremelimumab KESTREL danvatirsen#) NeoCOAST SERD ER+ breast PD-1/CTLA-4 solid tumours PARP+ATR or PARP+Wee1 breast PD-L1+CTLA-4 1L HNSCC PD-L1+(CD73 or NKG2A or STAT3) NSCLC

MEDI7247 Calquence+AZD6738 Imfinzi#+(oleclumab or monalizumab#) COAST Lynparza#+Imfinzi MEDIOLA Imfinzi#+tremelimumab NEPTUNE ASCT2 ADC haematological malignancies solid BTK+ATR haematalogical tumours PD-L1+(CD73 or NKG2A) NSCLC PARP+PD-L1 ovarian breast gastric SCLC PD-L1+CTLA-4 1L NSCLC tumours

Imfinzi#+AZD5069 or Imfinzi#+danvatirsen# Calquence+danvatirsen oleclumab oleclumab+chemo or Imfinzi#+oleclumab+chemo Imfinzi#+tremelimumab+CRT ADRIATIC PD-L1+(CXCR2 or STAT3) HNSCC bladder BTK+STAT3 haematalogical malignancies CD73 solid tumours CD73+chemo or PD-L1+CD73+chemo pancreatic PD-L1+CTLA-4+CRT LD-SCLC NSCLC

Imfinzi#+adavosertib# oleclumab+AZD4635 Imfinzi#+Lynparza# ORION Tagrisso combo# TATTON Imfinzi#+tremelimumab+SoC CASPIAN PD-L1+Wee1 solid tumours CD73+A2aR EGFRm NSCLC PD-L1+PARP NSCLC EGFR+PD-L1/MEK/MET NSCLC PD-L1+CTLA-4+SoC 1L SCLC

Imfinzi#+RT (platform) CLOVER oleclumab+Tagrisso Imfinzi#+MEDI0457# trastuzumab deruxtecan#¶ DESTINY-Breast01 Imfinzi#+tremelimumab+SoC NILE PD-L1+RT HNSCC NSCLC SCLC CD73+EGFR EGFRm NSCLC PD-L1+DNA HPV vaccine HNSCC ADC breast PD-L1+CTLA-4+SoC 1L urothelial cancer

Imfinzi#+tremelimumab Imfinzi#+monalizumab# trastuzumab deruxtecan#¶ DESTINY-Gastric01 Imfinzi#+tremelimumab+SoC POSEIDON PD-L1+CTLA-4 solid tumours PD-L1+NKG2a solid tumours ADC gastric PD-L1+CTLA-4+SoC 1L NSCLC

Imfinzi#+oleclumab trastuzumab deruxtecan# Lynparza#+Imfinzi#+bevacizumab DUO-O PD-L1+CD73 solid tumours ADC colorectal cancer PARP+PD-L1+VEGF 1L ovarian

trastuzumab deruxtecan# savolitinib# SAVOIR ADC NSCLC MET pRCC

selumetinib#¶ SPRINT MEK paediatric neurofibromatosis type-1 6 1 includes novel combinations and additional indications for assets where the lead is not yet launched # Partnered and/or in collaboration; ¶ Registrational Phase II/III trial Oncology Cardiovascular, Renal & Metabolism, Respiratory, Other Q1 2019 New Molecular Entity (NME)1 pipeline

Phase I Phase II Phase III Applications Under Review 13 New Molecular Entities 22 New Molecular Entities 3 New Molecular Entities 1 New Molecular Entity

AZD0284 abediterol# MEDI5884# anifrolumab# TULIP PT010 RORg psoriasis/respiratory LABA asthma/COPD cholesterol modulation cardiovascular Type I IFN receptor SLE LABA/LAMA/ICS COPD

AZD0449 anifrolumab# MEDI6012 PT027 Inhaled JAK inhibitor asthma Type I IFN receptor SLE SC LCAT cardiovascular ICS/SABA asthma

AZD1402# anifrolumab# MEDI7352 tezepelumab# NAVIGATOR SOURCE inhaled IL-4Ra asthma Type I IFN receptor lupus nephritis NGF/TNF osteoarthritis pain, painful TSLP severe uncontrolled asthma diabetic neuropathy

AZD5634 AZD1419# MEDI8852 inhaled ENaC cystic fibrosis inhaled TLR9 asthma influenza A treatment

AZD8154 AZD4831 MEDI8897# Inhaled PI3Kgd asthma MPO HFpEF passive RSV prophylaxis

AZD8233 AZD5718 PT010 hypercholesterolemia cardiovascular FLAP coronary artery disease LABA/LAMA/ICS asthma

AZD9977 AZD7594 suvratoxumab MCR cardiovascular Inhaled SGRM asthma/COPD α-Toxin Staphylococcus pneumonia

MEDI0700# AZD7986# tezepelumab# BAFF/B7RP1 SLE DPP1 COPD TSLP atopic dermatitis

MEDI1341 AZD8601# verinurad alpha synuclein parkinson's disease VEGF-A cardiovascular URAT-1 chronic kidney disease

MEDI1814# AZD8871# amyloidβ alzheimer's disease MABA COPD

MEDI3506 AZD9567 IL-33 COPD SGRM RA/respiratory

MEDI6570 cotadutide LOX-1 CV disease GLP-1/glucagon type-2 diabetes / obesity

1 includes novel combinations and additional indications for assets where the lead is not yet launched MEDI7219 MEDI3902 ¶ anti-diabetic type-2 diabetes Psl/PcrV Pseudomonas pneumonia # Partnered and/or in collaboration; Registrational Phase II/III trial

7 Oncology Cardiovascular, Renal & Metabolism, Respiratory, Other Q1 2019 Lifecycle Management (LCM)1 pipeline

Phase I Phase II Phase III Applications Under Review 1 Project 5 Projects 20 Projects 0 Projects

Imfinzi#+CTx neoadjuvant AEGEAN Imfinzi#+azacitidine# Imfinzi# Calquence# PD-L1+CTx locally-advanced stage III PD-L1+azacitidine MDS BTK inhibitor 1st line MCL PD-L1 solid tumours NSCLC

Imfinzi# (platform) BEGONIA Calquence# Imfinzi#+CTx NIAGARA PD-L1 breast cancer BTK inhibitor 1st line CLL PD-L1+CTx muscle invasive bladder cancer

Imfinzi# (platform) MAGELLAN Calquence# Imfinzi#+VEGF+TACE EMERALD-1 PD-L1 NSCLC BTK inhibitor r/r CLL, high risk PD-L1+VEGF+TACE locoregional HCC

Lynparza# (basket) MK-7339-002 / Calquence# LYNK002 Lynparza# OlympiA BTK inhibitor r/r CLL PARP HRRm cancer PARP gBRCA adjuvant breast

Lynparza#+cediranib CONCERTO Calquence#+venetoclax+obinutuzumab Lynparza# POLO PARP+VEGF recurrent Pt-R ovarian BTK+BCL-2+anti-CD20 1st line CLL PARP pancreatic cancer

Imfinzi# CALLA Lynparza# PROfound PD-L1 adj. locally advanced cervical cancer PARP prostate cancer

Imfinzi# PEARL (China) Lynparza# SOLO-3 PD-L1 1L NSCLC PARP BRCAm PSR ovarian

Imfinzi# POTOMAC PD-L1 non muscle invasive bladder Lynparza+abiraterone# PROpel cancer PARP+NHA prostate cancer

Imfinzi#+CRT PACIFIC-2 Tagrisso ADAURA PD-L1+CRT NSCLC EGFR adj. EGFRm NSCLC

Imfinzi#+CRT PACIFIC-5 (China) Tagrisso LAURA PD-L1+CRT locally-advanced stage III EGFRm locally advanced unresectable NSCLC NSCLC

1 Includes significant LCM projects and parallel indications for assets beyond Phase III 8 # Partnered and/or in collaboration; ¶ Registrational Phase II/III trial Oncology Cardiovascular, Renal & Metabolism, Respiratory, Other Q1 2019 Lifecycle Management (LCM)1 pipeline

Phase I Phase II Phase III Applications Under Review 0 Projects 0 Projects 10 Projects 4 Projects

Brilinta/Brilique HESTIA Farxiga/Forxiga DECLARE P2Y12 paeds w/ sickle cell outcomes

Brilinta/Brilique THALES Nexium (CN only) P2Y12 stroke stress ulcer prophylaxis

Brilinta/Brilique THEMIS saxagliptin+dapagliflozin metformin P2Y12 diabetes & CAD outcomes DPP4+SGLT2 type-2 diabetes

Epanova STRENGTH Symbicort SYGMA outcomes as needed in mild asthma

Farxiga/Forxiga SGLT2 HFrEF

Farxiga/Forxiga SGLT2 CKD

Farxiga/Forxiga DELIVER SGLT2 HFpEF

Fasenra# IL-5R COPD

Fasenra# OSTRO IL-5R nasal polyposis

roxadustat# HIFPH anaemia MDS

1 Includes significant LCM projects and parallel indications for assets beyond Phase III 9 # Partnered and/or in collaboration; ¶ Registrational Phase II/III trial Oncology Cardiovascular, Renal & Metabolism, Respiratory, Other Estimated key regulatory submission acceptances

roxadustat anaemia in CKD (US)

selumetinib SPRINT

Imfinzi + tremelimumab KESTREL

Imfinzi + tremelimumab NEPTUNE trastuzumab deruxtecan

Imfinzi + tremelimumab DANUBE Imfinzi + tremelimumab + CRT ADRIATIC

Imfinzi +/- tremelimumab CASPIAN Imfinzi + tremelimumab HIMALAYA PT027 asthma

Imfinzi +/- tremelimumab POSEIDON Lokelma (China) Imfinzi + tremelimumab + SoC NILE Fasenra severe asthma (China) NME trastuzumab deruxtecan DESTINY-Breast01 (US) Lumoxiti (EU) Lynparza +Imfinzi +bevacizumab DUO-O tezepelumab NAVIGATOR H2 2019 2020 2020+

Calquence CLL Imfinzi PEARL Calquence 1L MCL Brilinta HESTIA

Lynparza POLO Lynparza PAOLA-1 Calquence +venetoclax+obinutuzumab 1L CLL Farxiga DAPA-CKD

Lynparza SOLO-3 Lynparza PROFOUND Imfinzi POTOMAC Farxiga HFpEF DELIVER

LCM Brilinta THEMIS Farxiga DAPA-HF Imfinzi CALLA roxadustat anemia in MDS

Lokelma (JP) Brilinta THALES Imfinzi BR.31 ADJUVANT Duaklir Genuair (China)

Symbicort SYGMA (China) Epanova STRENGTH Imfinzi + CRT PACIFIC-2

Fasenra OSTRO Imfinzi + CRT PACIFIC-5 (China)

Xigduo (China) Imfinzi + chemo AEGEAN

Imfinzi + chemo NIAGARA

Imfinzi + VEGF + TACE EMERALD-1

Lynparza OLYMPIA

Lynparza + abiraterone PROPEL

Tagrisso LAURA

Tagrisso ADAURA

10 Oncology Cardiovascular, Renal & Metabolism, Respiratory, Other Designations 4 9 8 20 23 Accelerated approvals Breakthrough Therapy Fast Track Priority Review / PRIME Orphan Drug

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)

Tagrisso EGFRm T790M NSCLC (US) Lynparza prostate cancer PROFOUND (US) savratoxumab Staph HAP (US) Tagrisso EGFRm T790M NSCLC (US) Lumoxiti HCL PLAIT (US)

Imfinzi bladder cancer (US) Imfinzi bladder cancer 1L (US) Imfinzi NSCLC (US) Imfinzi bladder cancer 2L (US) Lumoxiti HCL PLAIT (EU)

Calquence MCL (US) Calquence MCL (US) MEDI8897 RSV mAB (US) Tagrisso NSCLC AURA3 (US) Crestor paediatric (US)

Imfinzi stage III NSCLC 1L PACIFIC (US) Imfinzi HNSCC HAWK (US) Calquence MCL (US) cediranib VEGFR tki (US)

Tagrisso NSCLC 1L FLAURA (US) anifrolumab SLE (US) Lynparza breast cancer OLYMPIAD (US) Iressa EGFRm NSCLC (US)

tezepelumab asthma (US) Lynparza ovarian cancer SOLO-2 (US) roxadustat CKD (CN) Tagrisso EGFRm T790M NSCLC (US)

MEDI8897 RSV mAB (US) Tagrisso EGFRm T790M NSCLC (CN) Tagrisso NSCLC FLAURA (US) AZD3241 MPO (EU)

selumetinib NFI type 1 SPRINT (US) Imfinzi stage III NSCLC PACIFIC (EU) Calquence CLL 1L (US)

Imfinzi stage III NSCLC PACIFIC (JP) Calquence MCL (US)

Lynparza tablet (US) Calquence WM (US)

Lynparza tablet (CN) Calquence WM (EU)

Lynparza breast cancer OLYMPIAD (JP) Calquence CLL 1L (EU)

Tagrisso NSCLC 1L FLAURA (JP) Calquence MCL (EU)

Lumoxiti HCL PLAIT (US) selumetinib thyroid cancer ASTRA (US)

Lynparza ovarian SOLO-1 (US) Lynparza breast cancer OLYMPIAD (JP)

Lynparza ovarian SOLO-1 (CN) Lynparza ovarian cancer SOLO-2 (JP)

PT010 Triple MDI COPD (CN) selumetinib NFI type 1 SPRINT (US)

MEDI8897 RSV mAB (EU) selumetinib NFI type 1 SPRINT (EU)

Tagrisso NSCLC 1L FLAURA (CN) Lynparza pancreatic cancer POLO (US)

Fasenra EGPA (US)

Fasenra HES (US) Fast Track is a process designed to facilitate the development, and expedite the review of medicines to treat serious conditions and fill an unmet medical need. Breakthrough Designation is a process designed to expedite the development and review of medicines which may demonstrate substantial improvement over available therapy. saracatinib IPF (US) Accelerated Approval, these regulations allowed medicines for serious conditions that addressed an unmet medical need to be approved based on a surrogate endpoint. Priority Review Designation is the US FDA’s goal to take action on an application within 6 months. PRIME is a scheme launched by the EMA to enhance support for the development of medicines that target an unmet medical need Orphan Drug Designation, intended for treatment, diagnosis or prevention of rare diseases/disorders that affect fewer than 200,000 patients in the US, or that affect more than 200,000 patients but are not expected to recover the costs of developing and marketing a treatment drug.

11 Oncology Cardiovascular, Renal & Metabolism, Respiratory, Other AstraZeneca

Oncology – approved medicines and late-stage pipeline Approved medicines Late-stage development Tagrisso (highly-selective, irreversible EGFRi) Early development Non-small cell lung cancer (NSCLC) Oncology

Trial Population Patients Design Endpoints Status Phase III Adjuvant EGFRm 700 • Arm 1: Tagrisso 80mg QD following complete tumour • Primary endpoint: Disease Free Survival • FPCD: Q4 2015 ADAURA resection, with or without chemotherapy (DFS) • LPCD: Q1 2019 • Arm 2: placebo • Secondary endpoints: DFS Rate, OS, • Data anticipated: 2020+

NCT02511106 OS Rate, QoL CVRM Global trial - 25 countries

Phase III Maintenance therapy in 200 • Arm 1: Tagrisso 80mg • Primary endpoint: PFS (via blinded • FPCD: Q3 2018 LAURA patients with • Arm 2: placebo independent central review (BICR)) • Data anticipated: 2020+ • Secondary endpoints: CNS PFS, OS, locally advanced, NCT03521154 unresectable EGFRm+ Stage Global trial - 11 countries DoR, ORR, DCR III whose disease has not progressed following platinum-based chemoradiation therapy

Phase II EGFRm+ / MET+, locally 172 • Single arm trial: Tagrisso + savolitinib • Primary endpoint: ORR • FPCD Q1 2019 Respiratory SAVANNAH advanced or metastatic • Secondary endpoints include PFS, DoR • Data anticipated: 2020+ NSCLC who have progressed Global trial and OS NCT03778229 following treatment with Tagrisso Phase Ib Advanced EGFRm TKI failure 308 • Arm 1: Tagrisso + Imfinzi • Safety, tolerability, pharmacokinetics • FPCD: Q3 2014 TATTON • Arm 2: Tagrisso + savolitinib and Preliminary anti-tumour activity • Data anticipated: 2020 • Arm 3: Tagrisso + selumetinib

NCT02143466 Enrolment to Imfinzi combination arms will not restart Other

Global trial Phase III Real world setting in adult 3,020 Single-arm trial - Tagrisso 80mg • Primary endpoints: OS and safety • FPCD: Q3 2015 ASTRIS patients with advanced or • Secondary endpoint: PFS • LPCD: Q4 2017 metastatic, EGFR T790M+ Global trial - 16 countries NCT02474355

Phase II EGFR TKI treatment-naïve 150 Single arm trial – Tagrisso 80 mg • Primary Endpoint: proportion of patients • FPCD: Q2 2018 ELIOS patients with locally-advanced with a given tumour genetic and or metastatic EGFRm+ Global trial - five countries proteomic marker at the point of disease NCT03239340 progression as defined by the investigator • Secondary endpoint: PFS, ORR, DoR

13 Approved medicines Late-stage development Imfinzi (PD-L1 mAb) Early development Non-small cell lung cancer (NSCLC), early use Oncology

Trial Population Patients Design Endpoints Status

Phase III Adjuvant NSCLC patients 1,360 • Arm 1: Imfinzi mg/kg IV Q4W x 12m Primary endpoint: • FPCD: Q1 2015 ADJUVANT BR.31 IB (≥4cm) – stage IIIA • Arm 2: placebo • DFS • Data anticipated: 2020+ resected NSCLC NCT02273375 (incl. EGFR/ALK positive) Global trial Secondary endpoint: • OS CVRM Partnered Phase II/III Lung Master Stage IV squamous NSCLC 140 Umbrella trial with five arms based on biomarker expression: Primary endpoints: • FPCD: Q2 2014 Protocol patients • Substudy A: Imfinzi (non-match for other biomarker driven • ORR • Data anticipated: 2020+ substudies) IVQ2W single arm Imfinzi Phase II only • PFS NCT02154490 Biomarker-targeted • Substudy B: PI3K inhibitor vs. docetaxel • OS 2L therapy • Substudy C: CDK4/6 inhibitor vs, docetaxel Partnered • Substudy D: AZD4547 (FGFR inhibitor) vs. docetaxel • Substudy E: C-MET/HGFR Inhibitor + erlotinib vs. erlotinib Phase III Unresected, locally-advanced 300 • Arm 1: Imfinzi IV Q4W + chemo/RT (radiation therapy) Primary endpoint: • FPCD: Q2 2018

NSCLC • Arm 2: placebo + chemo/RT • PFS • Data anticipated: 2020+ Respiratory PACIFIC-2 • ORR ex US global trial Secondary endpoint: NCT03519971 • OS

Phase III Imfinzi following SBRT in 630 •Arm 1: Imfinzi IV Q4W following definitive SBRT (radiation Primary endpoint: •FPCD: Q1 2019 unresected, Stage I/II NSCLC therapy) •PFS •Data anticipated: 2020+ PACIFIC-4 •Arm 2: placebo following definitive SBRT Secondary endpoint:

•OS Other NCT03833154

Phase III Unresected, locally-advanced 360 Arm 1: Imfinzi IV Q4W following chemo/RT (radiation therapy) Primary endpoint: • FPCD: Q1 2019 NSCLC Arm 2: placebo following chemo/RT • PFS • Data anticipated: 2020+ PACIFIC-5 Secondary endpoint: ex US global trial, China focus • OS NCT03706690

Phase III Neoadjuvant NSCLC patients 300 Arm 1: Imfinzi + platinum-based chemotherapy Primary endpoint: • FPCD: Q1 2019 Stage II and III resected Arm 2: placebo + platinum-based chemotherapy • Major Pathological Response (mPR) • Data anticipated: 2020 AEGEAN NSCLC Secondary endpoint (incl. EGFR/ALK positive) • Pathological complete response NCT03800134 (pCR)

14 Approved medicines Late-stage development Imfinzi (PD-L1 mAb) +/- treme (CTLA-4 mAb) Early development Lung cancer, advanced Oncology

Trial Population Patients Design Endpoints Status

Phase III Limited disease- Small cell 600 • Arm 1: Imfinzi + tremelimumab (4 doses) Primary endpoints: • FPCD: Q4 2018 ADRIATIC lung cancer (SCLC) 1L • Arm 2: Imfinzi • PFS • Data anticipated: 2020+ following platinum-based • Arm 3: placebo • OS

NCT03703297 concurrent chemoradiation CVRM therapy

Phase III NSCLC 1L 650 • Arm 1: Imfinzi Q4W Primary endpoint: • FPCD: Q1 2017 PEARL • Arm 2: chemotherapy • OS • LPCD: Q1 2019 • Data anticipated: 2020 NCT03003962 Asia trial Phase III NSCLC 1L 960 • Arm 1: Imfinzi + tremelimumab • Primary endpoint: OS • FPCD: Q4 2015 NEPTUNE • Arm 2: SoC • Secondary endpoint: PFS • LPCD: Q2 2017 • Data anticipated: H2 2019 NCT02542293

Phase III NSCLC 1L 1,000 • Arm 1: Imfinzi + CTx Primary endpoint: • FPCD: Q2 2017 Respiratory POSEIDON • Arm 2: Imfinzi + tremelimumab + chemotherapy • OS • LPCD: Q3 2018 • Arm 3: SoC • Data anticipated: H2 2019 NCT03164616

Phase III SCLC 1L 795 • Arm 1: Imfinzi + tremelimumab + EP (carboplatin or cisplatin + Primary endpoint: • FPCD: Q1 2017 CASPIAN etoposide) • OS • LPCD: Q2 2018 • Arm 2: Imfinzi + EP (carboplatin or cisplatin + etoposide) • Data anticipated: H2 2019 NCT03043872 • Arm 3: EP (carboplatin or cisplatin + etoposide) Other Phase II SCLC 80 • Arm A: Imfinzi + tremelimumab Q4W • Primary endpoint: ORR • FPCD: Q4 2016 BALTIC • Arm B: adavosertib and carboplatin BID • Data anticipated: 2020+ • Arm C: AZD6738 and Lynparza NCT02937818 Phase II NSCLC 1L 200 • Arm A1: Imfinzi Primary endpoint: • FPCD: Q1 2019 MAGELLAN • Arm A2: Imfinzi + danvatirsen • Safety & tolerability • Data anticipated: 2020+ • Arm A3: Imfinzi + oleclumab Secondary endpoint: NCT03819465 • Arm B1: Imfinzi + Investigator's choice of chemo • ORR, DoR, PFS, OS, PK, ADA • Arm B2: Imfinzi + danvatirsen + Investigator's choice of chemo • Arm B3: Imfinzi + oleclumab + Investigator's choice of chemo

15 Approved medicines Late-stage development Imfinzi (PD-L1 mAb) +/- treme (CTLA-4 mAb) Early development Other cancers, early disease Oncology

Trial Population Patients Design Endpoints Status

Phase III Non-muscle invasive bladder 975 • Arm 1: BCG (Bacillus Calmette–Guérin) (Induction + Primary endpoints: • FPCD: Q3 2018 POTOMAC cancer maintenance) • DFS • Data anticipated: 2020+ • Arm 2: Imfinzi + BCG (Induction only)

NCT03528694 • Arm 3: Imfinzi + BCG (Induction + maintenance) CVRM Phase III Muscle-invasive bladder 960 • Arm 1: Imfinzi in combination with gemcitabine + cisplatin, Coprimary endpoints: • FPCD: Q1 2019 cancer Imfinzi maintenance • pCR • Data anticipated: 2020+ NIAGARA • Arm 2: gemcitabine + cisplatin • EFS

Phase III Locoregional Hepatocellular 600 • Arm A: Transarterial Chemoembolization (TACE) in Primary endpoint • FPCD: Q1 2019 EMERALD-1 Carcinoma combination with Imfinzi PFS for Arm A vs Arm C • Data anticipated: 2020+ • Arm B: Transarterial Chemoembolization (TACE) in NCT03778957 combination with Imfinzi + Bevacizumab Secondary endpoint • Arm C: Transarterial Chemoembolization (TACE) in PFS for Arm B vs Arm C , OS combination with Placebos

Phase III Adjuvant Therapy in 888 • Arm 1: Imfinzi + bevacizumab Primary endpoint: • Initiating Respiratory EMERALD-2 Hepatocellular Carcinoma • Arm 2: Imfinzi + placebo • RFS for Arm 2 vs Arm 3 • Arm 3: placebo + placebo Secondary endpoint: • RFS Arm 1 vs Arm 3, OS, RFS at 24 mos Other

pCR = Pathologic Complete Response EFS = event free survival 16 Approved medicines Late-stage development Imfinzi (PD-L1 mAb) +/- treme (CTLA-4 mAb) Early development Other cancers, late disease Oncology

Trial Population Patients Design Endpoints Status

Phase III Cis-eligible and ineligible 1,005 • Arm 1: Imfinzi + tremelimumab Primary endpoints: • FPCD: Q4 2015 DANUBE bladder cancer 1L • Arm 2: Imfinzi • OS • LPCD: Q1 2017 • Arm 3: SoC • Data anticipated: H2 2019

NCT02516241 CVRM

Phase III Bladder cancer 1L 885 • Arm 1: Imfinzi + tremelimumab + SoC Primary endpoints: • FPCD: Q3 2018 NILE • Arm 2: Imfinzi + SoC • PFS • Data anticipated: 2020+ • Arm 3: SoC • OS NCT03682068 Phase III HNSCC 1L 823 • Arm 1: Imfinzi Primary endpoints: • FPCD: Q4 2015 KESTREL • Arm 2: Imfinzi + tremelimumab • OS • LPCD Q1 2017 • Arm 3: SoC • Data anticipated: H2 2019 NCT02551159 Phase III Unresectable Hepatocellular 1,310 • Arm 1: Imfinzi + tremelimumab (Regimen 1) Primary endpoint: • FPCD: Q4 2017

HIMALAYA Carcinoma (HCC) 1L • Arm 2: Imfinzi + tremelimumab (Regimen 2) • OS • Data anticipated: 2020+ Respiratory • Arm 3: Imfinzi Secondary endpoint: NCT03298451 • Arm 4: sorafenib • PFS, time to tumour progression (TTP), ORR

Phase II Urothelial bladder cancer 76 • Arm 1 tremelimumab (urothelial bladder cancer) Primary endpoint: • FPCD: Q4 2015 triple-negative breast cancer • Arm 2 tremelimumab (triple-negative breast cancer) • ORR • Data readout: Q4 2018 pancreatic ductal- • Arm 3 tremelimumab (pancreatic ductal-adenocarcinoma) NCT02527434 adenocarcinoma Secondary endpoints:

• Safety, DoR Other

Phase III Biliary Tract Carcinoma 474 • Treatment Arm 1 Durvalumab + Gemcitabine + Cisplatin Primary endpoint: • Initiating TOPAZ-1 (BTC) 1L • Treatment Arm 2 Placebo + Gemcitabine + Cisplatin • OS

NCT03875235 Global trial Secondary endpoint: • PFS, ORR, DoR

Phase III Locally Advanced Cervical 714 • Arm 1 Imfinzi + EBRT + brachytherapy with platinum Primary • FPCD: Q1 2019 CALLA Cancer • Arm 2 Placebo + EBRT + brachytherapy with platinum • PFS • Data anticipated: 2020+ Secondary NCT03830866 Global trial • OS, PFS, CR rate, DoR, ORR, DoCR, safety/tolerability, PRO, PK/ADA

pCR = Pathologic Complete Response EFS = event free survival 17 Approved medicines Late-stage development Imfinzi (PD-L1 mAb) +/- treme (CTLA-4 mAb) Early development Other cancers Oncology

Trial Population Patients Design Endpoints Status Phase III Advanced solid malignancies 1,200 • Arm 1: Imfinzi • Primary endpoint: Safety • FPCD: Q2 2017 STRONG • Arm 2: Imfinzi + tremelimumab • Data anticipated: 2020+

NCT03084471 CVRM

Phase I Combination in Solid tumours 80 • Arm 2 Small cell lung cancer (SCLC). Imfinzi + tremelimumab • Safety • FPCD: Q1 2016 Advanced Solid Tumours + carboplatin + etoposide • LPCD: Q1 2019 • Arm 3 TNBC (triple-negative breast cancer): Imfinzi + • Data anticipated: 2020+ NCT02658214 tremelimumab + chemo • Arm 4 TNBC: Imfinzi + tremelimumab + chemo • Arm 5 Gastric/gastro-Oesophageal junction (GEJ): Imfinzi + tremelimumab + oxaliplatin + 5-fluorouracil (5FU) + leucovorin Arm 6 PDAC (pancreatic ductal adenocarcinoma): Imfinzi + tremelimumab + chemo • Arm 7 ESSC (esophageal squamous cell carcinoma): Imfinzi + tremelimumab + chemo Respiratory Phase I Immunotherapy in Head and neck squamous- 300 • HNSCC Arm 1 • Safety • FPCD: Q2 2018 Combination With cell carcinoma (HNSCC), • NSCLC Arm 1 • Data anticipated: 2020+ Chemoradiation in Patients Non-small-cell lung cancer • NSCLC Arm 2 With Advanced Solid (NSCLC), Small-cell lung • NSCLC Arm 3 Tumours cancer (SCLC) • SCLC Arm 2 • SCLC Arm 3 CLOVER • SCLC Arm 4 Other NCT03509012

Phase II mTNBC (metastatic triple 100 • Arm 1 Imfinzi + paclitaxel Primary endpoint: • FPCD: 1Q2019 BEGONIA negative breast cancer) 1L • Arm 2 Imfinzi + paclitaxel + capivasertib • Safety and tolerability • Data anticipated: 2020+ • Arm 3 Imfinzi + paclitaxel + selumetinib NCT03742102 • Arm 4 Imfinzi + paclitaxel + danvatirsen Secondary endpoint: • Arm 5 Imfinzi + paclitaxel + oleclumab • ORR, PFS, DoR, OS, PK, ADA

Global trial

18 Approved medicines Late-stage development Lynparza (PARP inhibitor) Early development Ovarian and other cancers Oncology

Trial Population Patients Design Endpoints Status Phase III BRCAm maintenance ovarian 391 • Arm 1: Lynparza tablets 300mg BID maintenance therapy for • Primary endpoint: PFS • FPCD: Q3 2013 SOLO-1 cancer 1L two years or until disease progression • Secondary endpoint: OS • LPCD: Q1 2015 • Arm 2: placebo • Data readout: Q2 2018 • Primary endpoint met NCT01844986 Global trial CVRM

Phase III PSR gBRCAm ovarian cancer 266 • Arm 1: Lynparza 300mg BID to progression • Primary endpoint: ORR • FPCD: Q1 2015 SOLO-3 3L+ • Arm 2: physician’s choice (single-agent chemotherapy) • LPCD: Q2 2018 • Data readout: Q4 2018 NCT02282020 Global trial • Primary endpoint met

Phase III BRCAm adjuvant breast 1,800 • Arm 1: Lynparza 300mg BiD • Primary endpoint: invasive disease-free • FPCD: Q2 2014 OlympiA cancer 12 month duration survival (IDFS) • Arm 2: placebo 12-month duration • Secondary endpoint: distant disease- Respiratory NCT02032823 free survival and OS Global trial partnership with BIG and NCI/NRG Partnered Phase III BRCAm metastatic breast 302 • Arm 1: Lynparza 300mg BiD, continuous to progression • Primary endpoint: PFS • FPCD: Q2 2014 OlympiAD cancer • Arm 2: physician’s choice: • Secondary endpoint: OS • LPCD: Q4 2015 capecitabine 2500mg/m2 x 14 q 21 • Data readout: Q1 2017 NCT02000622 vinorelbine 30mg/m2 d 1, 8 q 21 • Primary endpoint met eribulin 1.4mg/m2 d 1, 8 q 21 Other to progression

Global trial

Phase III gBRCAm pancreatic cancer 154 • Arm 1: Lynparza tablets 300mg twice daily as maintenance • Primary endpoint: PFS • FPCD: Q1 2015 POLO therapy until progression • Secondary endpoint: OS • LPCD: Q1 2019 • Arm 2: placebo tablets BID • Data readout: Q1 2019 NCT02184195 • Primary endpoint met Global trial

Phase III Metastatic castration-resistant 387 • Arm 1: Lynparza 300mg BID • Primary endpoint: radiologic PFS • FPCD: Q2 2017 PROfound prostate cancer • Arm 2: physician’s choice: • Secondary endpoints: ORR, Time to • LPCD: Q4 2018 HRRm, 2L+ enzalutamide 160mg once daily Pain Progression, OS • Data anticipated : H2 2019 NCT02987543 abiraterone acetate 1,000mg once daily

Global trial

19 Approved medicines Late-stage development Lynparza (PARP inhibitor) Early development Imfinzi combinations, cancers Oncology

Trial Population Patients Design Endpoints Status Phase III Advanced ovarian cancer 1L 1,056 Non tBRCAm (tumour BRCA) patients Primary endpoint: • FPCD: Q1 2019 • Arm 1: bevacizumab • PFS • Data anticipated: 2020+ DuO-O • Arm 2: bevacizumab + Imfinzi • Arm 3: bevacizumab + Imfinzi + Lynparza NCT03737643 CVRM tBRCAm patients • bevacizumab (optional) + Imfinzi + Lynparza

Global trial

Phase II Stage IV NSCLC whose disease has 250 • Arm 1: Imfinzi + Lynparza Primary endpoint: • FPCD Q1 2019 not progressed following SoC chemo • Arm 2: Imfinzi + placebo • PFS • Data anticipated: 2020+ DuO-L + Imfinzi Maintenance therapy 1L (ORION) Global trial

NCT03775486 Respiratory Phase II Platinum-Ineligible unresectable 150 • Arm 1: Imfinzi + Lynparza • Primary endpoint: PFS • FPCD: Q1 2018 BAYOU Stage IV urothelial cancer • Arm 2: Imfinzi + placebo • Data anticipated : 2020

NCT03459846 Global trial

Phase I / II gBRCAm ovarian cancer 2L+ 148 • Arm 1: Lynparza + Imfinzi Primary endpoints: • FPCD: Q2 2016 MEDIOLA gBRCAm HER2-negative breast • Dose until progression • DCR at 12 weeks • LPCD: Q2 2017

cancer 1-3L • Safety and tolerability Other NCT02734004 Small cell lung cancer (SCLC) 2L+ Global trial Gastric cancer 2L+

Phase I / II gBRCAm ovarian cancer 2L+ 140 • Arm 1: Lynparza + Imfinzi Primary endpoints: • FPCD: Q2 2018 MEDIOLA Non-gBRCAm ovarian cancer 2L+ • Arm 2: Lynparza + Imfinzi • DCR at 12 weeks (Ovarian expansion) Non-gBRCAm ovarian cancer 2L+ • Arm 3: Lynparza + Imfinzi + bevacizumab • ORR • Dose until progression • Safety and tolerability NCT02734004 Global trial

Phase I / II HER2-negative BRCAm breast 140 • Arm 1: Lynparza + Imfinzi Primary endpoints: • Initiating MEDIOLA cancer • DCR at 12 weeks (Breast expansion) HER2-negative non-BRCA HRRm • Arm 2: Lynparza + Imfinzi • ORR breast cancer • Safety and tolerability NCT02734004 Non-HRRm triple negative breast • Arm 3: Lynparza + Imfinzi + bevacizumab cancer • Dose until progression Global trial 20 Approved medicines Late-stage development Lynparza (PARP inhibitor) Early development Combinations, cancers Oncology

Trial Population Patients Design Endpoints Status

Phase III Advanced ovarian cancer 1L 806 • Arm 1: Lynparza maintenance therapy for two years or until Primary endpoint: • FPCD: Q2 2015 PAOLA-1 maintenance disease progression • PFS • LPCD: Q2 2018 • Arm 2: Placebo for two years or until disease progression • Data anticipated: H2 2019 NCT02477644 Externally sponsored Global trial CVRM

Phase III Metastatic castration-resistant 720 • Arm 1: Lynparza + abiraterone Primary Endpoint: • FPCD: Q4 2018 prostate cancer 1L • Arm 2: placebo + abiraterone • PFS • Data anticipated: 2020+ PROPEL Global trial NCT 03732820

Phase II Triple-negative breast cancer 450 • Arm 1: AZD6738 + Lynparza • PFS • FPCD: Q2 2018 (TNBC) • Arm 2: adavosertib + Lynparza • ORR / OS • Data anticipated: 2020+ VIOLETTE • Arm 3: Lynparza • Safety and tolerability Trial conducted in 15 countries: North America, Europe and Asia

Phase III Recurrent platinum sensitive 549 • Arm 1: chemotherapy Primary endpoint: • FPCD: Q1 2016 Respiratory GY004 ovarian cancer • Arm 2: Lynparza • PFS • Data anticipated: 2020+ • Arm 3: cediranib + Lynparza NCT02446600 Secondary endpoints: Externally sponsored US/Canada/Japan sites • OS, QoL, safety

Phase II/III Recurrent platinum 680 • Arm 1: chemotherapy Primary endpoints: • FPCD: Q2 2016 GY005 resistant/refractory ovarian • Arm 2: cediranib + Lynparza • PFS, OS • Data anticipated: 2020+ cancer • Arm 3: cediranib Other NCT02502266 • Arm 4: Lynparza Secondary endpoints: Externally sponsored • ORR, QoL, safety • US/Canada sites

Phase II HRRm or HRD-positive 370 • Arm 1: Lynparza Primary endpoints: • FPCD: Q1 2019 LYNK-002 advanced cancer • ORR Trial conducted in 15 countries worldwide NCT03742895 Secondary endpoints: Partnered • DOR, OS, PFS, AE, Prog by CA-125

21 Approved medicines Late-stage development Trastuzumab deruxtecan (DS-8201, HER2 ADC) Early development Breast and gastric cancers Oncology

Trial Population Patients Design Endpoints Status

Phase II HER2-positive, unresectable and/or 230 Randomised, open label, sequential assignment Primary endpoint ORR • FPCD: Q3 2017 DESTINY-Breast01 metastatic breast cancer subjects • Trastuzumab deruxtecan • Data anticipated: H2 2019 previously treated With trastuzumab Secondary end points DoR, CBR, CBR, NCT03248492 emtansine PFS, OS Partnered CVRM

Phase III HER2-positive, unresectable and/or 600 Randomised open label parallel assignment Primacy endpoint PFS • FPC Q3 2018 DESTINY-Breast02 metastatic breast cancer pretreated with • Trastuzumab deruxtecan • Data anticipated 2020+ prior standard of care HER2 therapies, Physicians choice of Secondary endpoints OS, ORR, DoR, CBR NCT03523585 including trastuzumab emtansine • Lapatinib + capecitabine Partnered • Trastuzumab + capecitabine

Phase III HER2-positive, unresectable and/or 500 Randomised open label parallel assignment Primary endpoint PFS • FPCD Q3 2018 DESTINY-Breast03 metastatic breast cancer subjects • Trastuzumab deruxtecan • Data anticipated 2020+ previously treated with trastuzumab and • Ado-trastuzumab emtansine Secondary endpoints OS, ORR, DoR, Respiratory NCT03529110 taxane CBR, PFS Partnered

Phase III HER2-low, unresectable and/or 540 Randomised open label parallel assignment Primary end point PFS • FPCD Q4 2018 DESTINY-Breast04 metastatic breast cancer subjects • Trastuzumab deruxtecan • Data anticipated 2020+ • Physicians choice of SoC chemo (choice of capecitabine, Secondary end points OS, DoR, ORR NCT03734029 eribulin, gemcitabine, paclitaxel or nab-paclitaxel) Other Partnered

Phase II HER2-overexpressing advanced gastric 220 Randomised open label parallel assignment Primary end point ORR • FPCD Q4 2017 DESTINY-Gastric01 or gastroesophageal junction • Trastuzumab deruxtecan • Data anticipated 2020 adenocarcinoma patients who have • SoC chemo Secondary end points PFS, OS, DoR, NCT03329690 progressed on two prior treatment DCR, TTF, range of PK endpoints Partnered regimens

22 Approved medicines Late-stage development Trastuzumab deruxtecan (DS-8201, HER2 ADC) Early development Other cancers Oncology

Trial Population Patients Design Endpoints Status

Phase II HER2-expressing advanced colorectal 90 Non randomised single group assignment Primary end point ORR • FPCD Q1 2018 cancer • Trastuzumab deruxtecan • Data anticipated 2020 NCT03384940 Secondary end points PFS, OS, DoR, range of PK endpoints Partnered CVRM

Phase II HER2-over-expressing or mutated, 80 Non randomised parallel group assignment Primary end point ORR • FPCD Q2 2018 unresectable and/or metastatic NSCLC • Trastuzumab deruxtecan • Data anticipated 2020 NCT03505710 Secondary end points DoR, PFS, OS

Partnered

Phase I Advanced solid malignant tumours 278 Non randomised single group assignment Primary end points number of subjects with • FPCD Q3 2015 • Trastuzumab deruxtecan Aes, tumour response • Data read out Q2 2018 NCT02564900 Respiratory Secondary end points PK Partnered Other

23 Approved medicines Late-stage development Calquence (BTK inhibitor) Early development Blood cancers Oncology

Trial Population Patients Design Endpoint(s) Status

Phase III Previously untreated chronic 535 • Arm A: chlorambucil + obinutuzumab • Primary endpoint: PFS (Arm A vs. Arm • FPCD: Q2 2015 ACE-CL-007 (ELEVATE-TN) lymphocytic leukaemia (CLL) • Arm B: Calquence + obinutuzumab B) • Data anticipated: H2 2019 • Arm C: Calquence • Secondary endpoints: IRC (independent review committee) assessed ORR, OS NCT02475681 CVRM (Arm A vs. Arm B vs. Arm C)

Phase III Previously untreated CLL 780 • Arm A; Calquence + venetoclax (AV) • Primary - AV vs FCR/BR efficacy PFS • FPCD: Q1 2019 fit • Arm B: Calquence + venetoclax + obinutuzumab (AVG) • Secondary AVG vs FCR/BR efficacy • Data anticipated: 2020+ ACE-CL-311 • Arm C: fludarabine + cyclophosphamide + rituxumab (FCR) PFS; AV vs FCR/BR and AVG vs OR bendamustine + rituximab (BR) FCR/BR

Phase III Relapsed/refractory CLL 306 • Arm A: Calquence • Primary endpoint: IRC assessed PFS • FPCD Q3 2016 ACE-CL-309 (ASCEND) • Arm B: rituximab + idelalisib or bendamustine (investigator’s (arm A vs. Arm B) • Data anticipated: H2 2019 NCT02970318 choice) • Secondary endpoints: INV-assessed ORR, OS, DoR, patient reported outcomes (PROs) Respiratory Phase III Relapsed/refractory high risk 533 • Arm A: Calquence • Primary endpoint: PFS • FPCD: Q2 2015 ACE-CL-006 (ELEVATE-RR) CLL • Arm B: ibrutinib • Secondary endpoints: comparison of • Data anticipated: 2020+ incidence of infections, RTs (Richter’s NCT02477696 Transformation) and atrial fibrillation, OS

Phase III Previously untreated mantle 546 • Arm A: Calquence + bendamustine + rituximab • Primary endpoint: PFS by Lugano • FPCD: Q1 2017 ACE-LY-308 cell lymphoma (MCL) • Arm B: bendamustine + rituximab Classification for non-Hodgkin's • Data anticipated: 2020+ Lymphoma (NHL) NCT02972840 • Secondary endpoints: Investigator- Other assessed (IA) PFS, ORR; IRC-assessed ORR, DoR, time to response; OS Phase II Relapsed/ refractory CLL, 60 Calquence monotherapy • ORR at 36 cycles • FPCD: Q1 2016 ACE-CL-208 intolerant to ibrutinib • Data anticipated: 2020

NCT02717611 Phase II Relapsed/refractory and 48 Calquence monotherapy • ORR • FPCD: Q4 2014 15-H-0016 treatment naïve/del17p • Arm A: Lymph node biopsy • Data anticipated: 2020+ CLL/small lymphocytic • Arm B: Bone marrow biopsy NCT02337829 lymphoma (SLL) Phase I/II CLL/SLL/Richter's 286 Calquence monotherapy • Safety, PK, PD • FPCD: Q1 2014 ACE-CL-001 transformation (RT) Dose escalation and expansion • Data anticipated: 2020+

NCT02029443 24 Approved medicines Late-stage development Calquence (BTK inhibitor) Early development Blood cancers Oncology

Trial Population Patients Design Endpoint(s) Status

Phase I/II B-cell Malignancies 126 Dose escalation and expansion trial of the combination of • Safety • FPCD: Q1 2015 ACE-LY-001 Calquence and ACP-319 (Pi3K inhibitor) • ORR • Data anticipated: 2020

NCT02328014 CVRM Phase I/II Haematological Malignancies 159 Calquence + pembrolizumab • Safety • FPCD: Q1 2015 ACE-LY-005 • Secondary endpoints: ORR, DoR, PFS, • Data anticipated: 2020+ OS, TTNT (time to next therapy) NCT02362035 Phase I/II Waldenstrom 106 Calquence monotherapy • ORR • FPCD: Q3 2014 ACE-WM-001 Microglobulinaemia • Data readout: Q1 2018

NCT02180724 Phase Ib Relapsed/refractory de novo 21 Calquence monotherapy • Safety • FPCD: Q3 2014

ACE-LY-002 activated B-cell diffuse large • Data anticipated: H2 2019 Respiratory B-cell lymphoma (DLBCL) NCT02112526 Phase Ib Mantle Cell Lymphoma (MCL) 76 Calquence in combination with bendamustine and rituximab • Safety • FPCD: Q1 2016 ACE-LY-106 • Arm A: Treatment naive • Data anticipated: 2020+ • Arm B: Relapsed/refractory NCT02717624 • Arm C: Treatment naïve: Calquence+venetoclax+Rituxan Other

Phase Ib Relapsed/refractory Multiple 28 • Arm A: Calquence • Safety • FPCD: Q1 2015 ACE-MY-001 Myeloma • Arm B: Calquence + dexamethasone • Data readout: Q4 2018

NCT02211014 Phase I Relapsed/refractory Follicular 126 • Arm A: Calquence • Safety • FPCD: Q1 2015 ACE-LY-003 Lymphoma • Arm B: Calquence + rituximab • Data anticipated: 2020+ NCT02180711

Phase I Relapsed/refractory CLL/ 12 Calquence in combination with ACP-319 • Safety, PK, PD • FPCD: Q3 2014 ACE-CL-002 small lymphocytic lymphoma Dose escalation • Data anticipated: 2020 (SLL) NCT02157324 Phase I CLL/SLL/Prolymphocytic 69 Calquence + obinutuzumab • Safety, ORR • FPCD: Q4 2014 ACE-CL-003 Leukaemia (PLL) • Arm A: Relapsed/refractory • Secondary endpoints: PD, PFS, TTNT, • Data anticipated: 2020+ • Arm B: Treatment naïve OS NCT02296918 Calquence + venetoclax + rituxumab 25 • Arm C: Relapsed/refractory • Arm D: Treatment naïve Approved medicines Late-stage development Calquence (BTK inhibitor) Early development Blood cancers Oncology

Trial Population Patients Design Endpoint(s) Status

Phase I Japanese Adults with 25 • Calquence monotherapy • Safety • FPCD: Q2 2017 Advanced B-cell Malignancies • Dose confirmation and expansion • Data anticipated: 2020+

NCT03198650 CVRM

Phase I/II CLL (chronic lymphocytic 62 • Arm A: AZD6738 monotherapy • Identify dose of AZD 6738 and safety of FPCD: Q1 2018 CL-110 leukaemia) R/R • Arm B: Calquence + AZD6738 co-administration of Calquence + Data anticipated: H1 2020 AZD6738

NCT03328273

Phase I/II B-cell malignancies R/R 25 Part 1: Calquence daily + vistusertib daily • MTD and optimal dosing schedule FPCD: Q3 2017 LY-110 Part 2: Calquence daily + vistusertib 5 days on/2 days off • Safety Data anticipated: 2020

NCT03205046 Respiratory Other

26 Approved medicines Late-stage development Calquence (BTK inhibitor) Early development Other cancers Oncology

Trial Population Patients Design Endpoint(s) Status

Phase II ≥ 2L advanced or metastatic 74 • Arm A: pembrolizumab • ORR • FPCD: Q2 2015 ACE-ST-006 Head and neck squamous-cell • Arm B: Calquence + pembrolizumab • Data readout: Q2 2018 carcinoma (HNSCC)

NCT02454179 CVRM Phase II ≥ 2L advanced or metastatic 74 • Arm A: pembrolizumab • ORR • FPCD: Q2 2015 ACE-ST-007 Non-small-cell lung cancer • Arm B: Calquence + pembrolizumab • Data readout: 2017 (NSCLC) NCT02448303 Phase II Recurrent ovarian cancer 76 • Arm A: Calquence • ORR • FPCD: Q4 2015 ACE-ST-208 • Arm B: Calquence + pembrolizumab • Data readout: Q3 2018

NCT02537444 Phase II ≥ 2L advanced or metastatic 73 • Arm A: Calquence • Safety • FPCD: Q2 2015

ACE-ST-003 pancreatic cancer • Arm B: Calquence + pembrolizumab • Data readout: Q3 2017 Respiratory

NCT02362048 Phase II Platinum-resistant urothelial 75 • Arm A: pembrolizumab • ORR • FPCD: Q2 2015 ACE-ST-005 bladder cancer • Arm B: Calquence + pembrolizumab • Data readout: Q1 2018

NCT02351739 Phase Ib/II ≥ 2L glioblastoma multiforme 52 • Arm A: Calquence 200mg BID • Safety, ORR • FPCD: Q1 2016 Other ACE-ST-209 • Arm B: Calquence 400mg QD • Data anticipated: H1 2019

NCT02586857

27 Approved medicines Late-stage development Selumetinib (MEK inhibitor) Early development Paediatric neurofibromatosis type 1 Oncology

Trial Population Patients Design Endpoints Status

Phase II Paediatric neurofibromatosis 50 (stratum 1) • Single arm: selumetinib 25mg/m2 BID with 2 strata: • Complete partial and complete response • FPCD: Q3 2015 SPRINT type 1 (NF1) • Stratum 1: PN related morbidity present at enrolment rate measured by volumetric MRI; • LPCD: Q4 2016 • Stratum 2: No PN related morbidity present at enrolment • Duration of response and functional • Data readout: Q1 2019 NCT01362803

outcomes/QoL CVRM

Partnered

Phase Ib Advanced solid tumours 80 (dose escalation Phase Ib open-label trial of MK-8353 in combination with • Dose-limiting toxicities (DLTs) • FPCD: Q1 2019 Selumetinib + MK-8353 (ERK trial) selumetinib in participants with advanced solid tumours • Adverse Events (AEs) inhibitor) • Study drug discontinuations due to an AE NCT03745989

Partnered (Merck Lead study) Respiratory Other

28 Approved medicines Late-stage development Savolitinib (MET inhibitor) Early development Papillary renal cell, NSCLC and other cancers Oncology

Trial Population Patients Design Endpoints Status

Phase III MET-driven, papillary renal 180 • Arm 1: savolitinib 600mg QD • Primary endpoint: PFS • FPCD: Q4 2017 cell cancer • Arm 2: sunitinib 50mg QD (4 weeks on / 2 weeks off) • Secondary endpoints include ORR, DoR • Data anticipated: 2020 NCT03091192 and OS Global trial Partnered CVRM

Phase I Advanced cancer ~70 • Dose escalation trial • Safety and tolerability • FPCD: Q2 2013 (all comers) • Data anticipated: 2020+ NCT01985555 Conducted in China

Partnered

Phase I NSCLC 64 • Dose escalation trial • Safety and tolerability • FPCD: Q2 2015

• Data readout: Q4 2018 Respiratory NCT02374645 Conducted in China

Phase II Lung Pulmonary Sarcomatoid 92 • Single arm trial: savolitinib 600mg QD • ORR • FPCD: Q1 2017 Carcinoma (PSC) and other • Data anticipated: 2020+ NCT02897479 NSCLC Conducted in China Other

Partnered

29 Approved medicines Late-stage development Cediranib (VEGF receptor inhibitor) Early development Ovarian cancer Oncology

Trial Population Patients Design Endpoints Status

Phase IIb Recurrent platinum resistant 62 • Cediranib 30mg + Lynparza 200mg bd • ORR DoR, DCR, QoL. OS; Safety • FPCD: Q1 2017 CONCERTO (PRR) ovarian cancer - heavily • LPCD: Q1 2019 pre-treated BRCAwt NCT02889900 CVRM Respiratory Other

30 AstraZeneca

Oncology – early-stage development Approved medicines Late-stage development AZD1390 (ATM inhibitor, blood brain barrier) Early development Cancer Oncology

Trial Population Subjects Design Endpoints Status Phase I Healthy volunteers 8 • Positron-Emission Tomography (PET) trial • Brain distribution of AZD1390 to assess • FPCD: Q4 2017 if [11C]AZD1390 crosses the blood brain • Data anticipated: H2 2019 NCT03215381 • [11C]AZD1390 microdose administered by IV bolus barrier in healthy volunteers

Trial conducted in a single centre in Sweden CVRM

Phase I Recurrent Glioblastoma c. 132 • Primary: Investigate the safety, • FPCD Q2 2018 eligible for re-irradiation, brain • Designed to evaluate the safety, tolerability and PK of tolerability, and MTD of AZD1390 • Data anticipated: 2020+ NCT03423628 metastases and AZD1390 in combination with radiation therapy in patients with administered in combination with radiation leptomeningeal disease, GBM and brain metastases from solid tumours therapy in brain malignancies newly-diagnosed glioblastoma patients • Dose and schedule of AZD1390 administration will be adjusted during assessment of safety and tolerability during this Phase I trial

Conducted across seven sites in USA and UK Respiratory Other

32 Approved medicines Late-stage development Adavosertib (AZD1775, WEE-1 inhibitor) Early development Ovarian cancer, triple-negative breast cancer, small cell lung cancer (SCLC) Oncology

Trial Population Patients Design Endpoints Status Phase II Platinum-resistant (PR) 97 • Arm B: paclitaxel + adavosertib • Primary endpoint: ORR • FPCD: Q1 2015 ovarian cancer • Arm C: carboplatin + adavosertib • LPCD: Q2 2018 NCT02272790 • Secondary endpoints: DoR, PFS, OS, Global trial Disease Control Rate, safety and tolerability CVRM

Phase I Advanced solid tumours 102 • Dose escalation trial to determine MTD (adavosertib + • Safety and tolerability • FPCD: Q3 2015 Lynparza) followed by an expansions in SCLC • Secondary endpoints: Overall response NCT02511795 rate, Disease Control Rate, Duration of Conducted in US, Canada Response, PFS Phase I Advanced solid tumours 55 • Dose escalation trial to determine MTD (adavosertib + Imfinzi) • Safety and tolerability • FPCD: Q4 2015

NCT02617277 Conducted in US

Phase I Advanced solid tumours 30 Part A: caffeine (200mg), omeprazole (20mg) and midazolam • Primary endpoints: • FPCD: Q4 2017

(1mL of 2mg/mL syrup) followed 7-14 days later by adavosertib • Part A: Plasma AUC, AUC0-t and CMAX Respiratory D6014C00006 225mg bid for 2.5 days plus caffeine (200mg), omeprazole for cocktail parent compounds (20mg) and midazolam (1mL of 2mg/mL syrup) on day 3. (midazolam, omeprazole and caffeine) NCT03333824 Part B: 7-14 days after end of Part A, adavosertib 225mg BID for • Part B: dECG (Differentiated ECG) 2.5 days. intervals (QTcF) for absolute values and time-matched change from baseline Conducted in US Phase I Advanced solid tumours 54 adavosertib monotherapy once daily. • Safety and tolerability • FPCD: Q4 2017 Other D6014C00007 Conducted in US and Europe

NCT03313557

33 Approved medicines Late-stage development Capivasertib (AZD5363, AKT inhibitor) Early development Cancer Oncology

Trial Population Patients Design Endpoints Status Phase I Breast and gynaecological 12-24 per arm (Parts E AZD5363 400mg BD 4 days on 3 days off combined with 500mg • Safety and tolerability • Data anticipated: H2 2019 cancers with PIK pathway & F) fulvestrant [initially 12 patients per arm with option to expand to • ORR NCT01226316 mutation 24 patients in one or more arms] • Clinical Benefit Rate at 24 weeks • Part E arm 1: ER+ Breast with AKT-1 mutation (prior Faslodex (CBR24) resistance) [Parts E & F only] CVRM • Part E arm 2: ER+ Breast with AKT-1 mutation (first exposure to Faslodex) • Part F arm 1: ER+ Breast with PTEN mutation (prior Faslodex resistance) • Part F arm 2: ER+ Breast with PTEN mutation (first exposure to Faslodex) Phase II (ESR) Advanced / metastatic triple 140 Randomised comparative • Progression Free Survival • Data readout: Q2 2018 negative breast cancer ARM1: Paclitaxel + capivasertib • Overall survival • Final OS data awaited NCT02423603 receiving 1L chemotherapy ARM 2: Paclitaxel + placebo with paclitaxel Overall population and in sub-group with

PAKT tumours harbouring PIK3CA/AKT1/PTEN Respiratory alterations

Phase II (ESR) Post menopausal women with 140 Randomised comparative • Progression Free Survival • Data anticipated: H1 2019 advanced ER+/Her2- breast ARM 1: Faslodex + capivasertib • Overall survival NCT01992952 cancer previously treated with ARM 2: Faslodex + placebo aromatase inhibition Overall population and sub-group with FAKTION activation of the tumour PI3K/Akt/PTEN Other pathway (eg. PIK3CA/AKT1/PTEN alterations)

Phase II (ESR) Metastatic castration resistant 150 Randomised comparative • Progression Free Survival • Data anticipated: 2020 prostate cancer eligible for ARM 1: Docetaxel + prednisolone + capivasertib NCT02121639 treatment with docetaxel ARM 2: Docetaxel + prednisolone + placebo chemotherapy PROCAID

34 Approved medicines Late-stage development Imfinzi (PD-L1 mAb) Early development Cancer Oncology

Trial Compound Population Patients Design Endpoints Status 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 expansion: 16 tumour type cohorts at the Q2W MTD • Optimal biologic dose • LPCD: Q4 2016 defined during dose escalation • Secondary endpoints include PK, • Data anticipated: H2 2019 NCT01693562 • Dose exploration: cohort at 20mg Q4W immunogenicity and antitumour activity CVRM

Global trial - nine countries

Phase I Imfinzi, azacitidine Myelodysplastic 79 Dose escalation and dose expansion trial • Safety and tolerability of monotherapy • FPCD: Q2 2014 (Vidaza) syndrome • Part 1: Imfinzi and combination • Data anticipated: 2020 NCT02117219 • Part 2 Arm 1: Imfinzi and tremelimumab • Secondary endpoints include duration of • Part 2 Arm 2: Imfinzi, tremelimumab and azacitidine response, PFS and OS, PK and immunogenicity

Global trial - four countries Respiratory Phase I MEDI9090 Solid tumours 42 Multi-centre, open-label, single-arm trial for adult subjects • Safety, PK, number of subjects reporting • FPCD: Q3 2016 infusion related reaction • Data anticipated: H2 2019 NCT02900157 US and Japan trial centers

Phase II Imfinzi Non-small-cell lung 260 5 modules encompassing 13 cohorts • Primary outcome; ORR • FPCD: Q1 2018 Lynparza cancer (NSCLC) Module 1; Imfinzi and Lynparza • Secondary outcomes; efficacy including • Data anticipated: 2020+ Other HUDSON Vistusertib Module 2; Imfinzi and danvatirsen OS, PFS, DCR, and safety and AZD6738 Module 3; Imfinzi and AZD6738 tolerability NCT03334617 Danvatirsen Module 4; Imfinzi and vistusertib Oleclumab Module 5; Imfinzi and oleclumab

Open-label, biomarker-directed, multi-centre Phase II umbrella trial in patients with NSCLC, who progressed on an anti-PD-1/PD- L1 containing therapy Phase II Imfinzi Stage III NSCLC 300 Arm A: Control (Imfinzi) Primary • FPCD: Q4 2018 COAST Unresectable Arm B: Imfinzi + oleclumab • OR per RECIST v1.1 • Data anticipated: 2020+ Arm C: Imfinzi + monalizumab NCT03822351

Phase II Imfinzi Resectable, Eaarly 160 Arm A: Control (Imfinzi) Primary • FPCD: Q1 2019 NeoCOAST Stage NSCLC Arm B: Imfinzi + oleclumab • Major pathological response rate • Data anticipated: 2020+ Arm C: Imfinzi + monalizumab 35NCT03794544 Arm D: Imfinzi + danvatirsen Approved medicines Late-stage development Imfinzi (PD-L1 mAb) + Early development tremelimumab (CTLA-4 mAb) Oncology Cancer

Trial Population Patients Design Endpoints Status CVRM Phase Ib/II Gastric/gastro-Oesophageal 114 • Arm A: Imfinzi + tremelimumab 2L • Primary endpoints: Safety & tolerability, • FPCD: Q2 2015 STUDY 21 junction (GEJ) • Arm B: Imfinzi 2L ORR, PFS • Data anticipated: H2 2019 adenocarcinoma • Arm C: tremelimumab 2L • Secondary endpoints: DCR, OS, DoR, NCT02340975 • Arm D: Imfinzi + tremelimumab 3L PD-L1 Expression • Arm E: Imfinzi + tremelimumab 2L & 3L

US and ROW trial centres

Phase Ib/II Hepatocellular Carcinoma 545 • Arm A: Imfinzi + tremelimumab • Primary endpoints: Safety & tolerability, • FPCD: Q4 2015 STUDY 22 • Arm B: Imfinzi 2L ORR, PFS • Data anticipated: 2020 • Arm C: tremelimumab 2L • Secondary endpoints: DCR, OS, DoR, NCT02519348 • Arm D: Imfinzi + tremelimumab PD-L1 Expression • Arm E: Imfinzi in combination with bevacizumab Respiratory

Phase Ib Non-small-cell lung cancer 459 • Dose Escalation: minimum 5 cohorts exploring various treme Primary endpoints: • FPCD: Q4 2013 STUDY 006 (NSCLC) Q4W and Imfinzi IV Q4W dose combinations, higher dose • Safety • LPCD: Q4 2016 (Immunotx naïve and levels and alternate Q2 schedule added with amendment • Optimal biologic dose for the • Data anticipated: H1 2019 NCT02000947 Immunotx pretreated patient • Dose Expansion: MTD for the combination in escalation to be combination cohorts) explored in expansion • OR • Secondary endpoints include antitumour North American, EU and ROW trial centres activity, PK and immunogenicity Other Phase I Solid tumours (Basket trial) 380 • Dose Expansion: MTD for the combination in escalation to be Primary endpoints: • FPCD: Q4 2014 STUDY 10 explored in expansion cohorts specific for each of 7 tumour • Safety • LPCD: Q2 2017 types • Optimal biologic dose for the • Data anticipated: H2 2019 NCT02261220 • Dose Exploration: 2 cohorts exploring various Q4W treme and combination Imfinzi dose combinations and 2 cohorts exploring various • Secondary endpoints include Q2W treme and Imfinzi dose combinations anti-tumour activity, PK/PD and North American, EU and ROW trial centres immunogenicity

Phase Ib Diffuse Large B cell 32 • Arm A: Imfinzi • Primary endpoint: Safety & tolerability • FPCD: Q3 2016 STUDY 23 Lymphoma • Arm B: Imfinzi + tremelimumab • Secondary endpoints: OR, DC, DoR, • Data readout: Q2 2019 • Arm C: Imfinzi + AZD9150 PFS, OS, PK/PD, immunogenicity and NCT02549651 US and European trial centres biomarkers

36 Approved medicines Late-stage development Imfinzi (PD-L1 mAb) + Early development monalizumab (NKG2a mAb) Oncology Cancer

Trial Population Patients Design Endpoints Status CVRM Phase I/II Advanced solid tumours 501 Escalation phase Primary endpoints: • FPCD: Q2 2016 • monalizumab + Imfinzi IV • Safety • Data anticipated: 2020+ NCT02671435 • Optimal biologic dose for the Expansion phase combination • monalizumab + Imfinzi IV recommended dose • Secondary endpoints include tumour Exploration phase response (CR, PR, SD, PD), Objective • monalizumab + Imfinzi IV recommended dose + SoC systemic response rate, disease control rate, therapy with or without biologic agent in adult subjects with progression-free survival, CRC (Colorectal cancer) and monalizumab in combination with immunogenicity, pharmacokinetics, biologic agent in adult subjects with colorectal cancer pharmacodynamics Respiratory Global trial Other

37 Approved medicines Late-stage development Imfinzi (PD-L1 mAb) + Early development MEDI0457 (DNA HPV Vaccine) Oncology Squamous cell carcinoma of the Head and Neck (SCCHN)

Trial Population Patients Design Endpoints Status CVRM Phase Ib/IIa Human papillomavirus (HPV) 50 Multi-centre, open label trial to evaluate the safety and efficacy of Primary endpoints: • FPCD: Q3 2017 Associated combination treatment with MEDI0457 and Imfinzi Safety & Tolerability, ORR • Data anticipated: 2020 NCT03162224 Recurrent/Metastatic Head and Neck Cancer Secondary endpoints: PK, ADA, DCR, OS, PFS Respiratory Other

38 Approved medicines Late-stage development Oleclumab (MEDI9447, CD73 mAb) Early development Cancer Oncology

Trial Population Patients Design Endpoints Status Phase I Advanced malignancies 310 Dose escalation phase Primary endpoints: • FPCD: Q3 2015 • oleclumab IV • Safety • Data anticipated: 2020 NCT02503774 • oleclumab IV + Imfinzi IV • Determination of MTD CVRM • Secondary endpoints include preliminary Dose expansion phase anti-tumour activity, pharmacokinetics, • oleclumab IV recommended dose + Imfinzi IV pharmacodynamics, and immunogenicity US, South Korean and Australian trial centres

Phase Ib/II Pancreatic 204 • Arm A1: Gemcitabine and nab Paclitaxel IV Primary endpoints: • FPCD: Q2 2018 1L and 2L with prior • Arm A2: Gemcitabine and nab Paclitaxel IV + oleclumab IV • Safety and anti-tumour activity • Data anticipated: 2020+ NCT03611556 gemcitabine-based • Arm A3: Gemcitabine and nab Paclitaxel IV + oleclumab IV + Imfinzi IV • Secondary endpoints include chemotherapy • Arm B1: mFOLFOX (oxaliplatin, leucovorin, 5-FU) IV pharmacokinetics, pharmacodynamics, • Arm B2: mFOLFOX (oxaliplatin, leucovorin, 5-FU) IV + immunogenicity, and safety oleclumab IV Respiratory • Arm B3: mFOLFOX (oxaliplatin, leucovorin, 5-FU) IV + oleclumab IV + Imfinzi IV

US, Norway, Spain and Australian trial centres Phase I/II Non-small-cell lung cancer 98 • Arm A: oleclumab IV + Tagrisso Primary endpoints: • FPCD: Q2 2018 (NSCLC) • Arm B: oleclumab IV + AZD4635 • Safety • Data anticipated: 2020+ NCT03381274 • ORR

• PoC for future registrational studies Secondary endpoints: Other • DoR, DCR, PFS, OS, PK and US, South Korean and Taiwan trial centres immunogenicity

39 Approved medicines Late-stage development AZD2811 (AURN) Early development Cancer Oncology

Trial Population Patients Design Endpoints Status

Phase I Solid tumours 72 • Arm 1: AZD2811 dose escalation • Safety and tolerability • FPCD: Q4 2015 • Arm 2: AZD2811 dose expansion • Pharmacokinetics and efficacy • Data anticipated: H2 2019 NCT02579226 CVRM

Phase I Acute Myeloid 130 • Part A: AZD2811 single agent dose escalation cohorts • Safety and tolerability • FPCD: Q3 2017 Leukaemia/High-Risk • Part B: AZD2811 single agent and azacytidine combination • Pharmacokinetics and efficacy • Data anticipated: 2020+ NCT03217838 Myelodysplastic Syndrome dose expansions to further explore the tolerability, PK and clinical activity. Respiratory Other

40 Approved medicines Late-stage development AZD4573 (CDK9 inhibitor) Early development Cancer Oncology

Trial Population Patients Design Endpoints Status Phase I Relapsed/refractory 42 Dose escalation in relapsed/refractory haematological Primary: • FPCD: Q4 2017 haematologic malignancies malignancies • safety/PK; • Data anticipated: H2 2019 NCT03263637 AZD4573 will be administered 2 parallel arms of (1-6 cohorts of Secondary: dose escalations) based on the haematological malignancy • efficacy CVRM Respiratory Other

41 Approved medicines Late-stage development AZD4635 (A2AR inhibitor) Early development Cancer Oncology

Trial Population Patients Design Endpoints Status Phase I Phase Ia: patients with 104-118 • Phase Ia: dose escalation to determine the MTD of AZD4635 Primary outcome measure: • FPCD: Q2 2016 advanced solid tumours given as monotherapy and in combination with Imfinzi in • Safety and tolerability • Data anticipated: 2020 NCT02740985 patients with solid malignancies. Also, to determine the MTD of AZD4635 in combination with abiraterone or with enzalutamide

Secondary outcome measures: CVRM in patients with metastatic castrate-resistant prostate • PK of AZD4635 as monotherapy and carcinoma (mCRPC). When the combination MTD is combination with Imfinzi abiraterone and determined, additional patients with advanced solid enzalutamide. malignancies and mCRPC will be enrolled to a dose expansion • Preliminary assessment of anti-tumour Phase Ib: 190 cohort to explore further the safety, tolerability, PK, and activity Post-immunotherapy NSCLC biological activity. Other post-immunotherapy • Phase Ia: will also enroll patients to a capsule formulation of solid tumours AZD4635 to determine the PK of the capsule formulation and the MTD in combination with Imfinzi plus oleclumab or with Immune checkpoint-naïve docetaxel. metastatic castrate-resistant • Phase Ib will consist of additional expansions in NSCLC, prostate carcinoma (mCRPC) mCRPC, CRC and other post-immunotherapy and immune

Immune checkpoint-naïve checkpoint-naïve solid tumours at the combination and/or Respiratory colorectal carcinoma (CRC) monotherapy MTD or maximum feasible dose Other immune checkpoint- naïve solid tumours Both parts conducted at sites in the US

Phase I Healthy male volunteers 20 • Part A is a 2-period randomised crossover study of single Primary outcome measures: • FPCD: Q4 2018 doses of AZD4635. Subjects will be randomised to receive • Maximum observed plasma • Data anticipated: H1 2019 NCT03710434 50mg AZD4635 nano-suspension (reference) or 50mg concentration (Cmax) of AZD4635 solid AZD4635 solid oral formulation, in the fasted state. oral formulation and nano-suspension Other • Part B is a 4-period, open-label, randomised, crossover study • Exposure to AZD4635 solid oral of single doses of AZD4635 in the same subjects from Part A. formulation and nano-suspension The treatments selected for Part B will depend on the outcome of interim analyses of AZD4635 exposure.

Both parts conducted at a site in the UK

42 Approved medicines Late-stage development AZD5069 (CXCR2 antagonist) Early development Cancer Oncology

Trial Population Patients Design Endpoints Status Phase Ib/II Metastatic pancreatic ductal 16 Dose escalation and expansion arms: • Safety/efficacy trial • FPCD: Q1 2016 carcinoma • Data anticipated: H2 2019 NCT02583477 Imfinzi in combination with nab-paclitaxel and gemcitabine Imfinzi in combination with AZD5069 CVRM Respiratory Other

43 Approved medicines Late-stage development AZD5153 (BRD4 inhibitor) Early development Cancer Oncology

Trial Population Patients Design Endpoints Status Phase I/Ib Relapsed/refractory solid 54 Dose Escalation advanced solid and lymphomas • Primary: safety • FPCD: Q2 2017 tumours, lymphomas Six dose escalation cohorts of AZD5153 • Secondary: efficacy • Data anticipated: H2 2019 NCT03205176 Dose and schedule from dose escalation will be applied in dose expansion Phase in platinum-resistant or platinum-refractory high CVRM grade serous (HGS) ovarian cancer Respiratory Other

44 Approved medicines Late-stage development AZD5991 (MCL1 inhibitor) Early development Cancer Oncology

Trial Population Patients Design Endpoints Status Phase I Relapsed/refractory 48 Dose escalation in relapsed/refractory haematological • Primary: safety • FPCD: Q3 2017 haematologic malignancies malignancies • Secondary: efficacy • Data anticipated: H2 2019 NCT03218683 Five dose escalation cohorts of AZD5991 CVRM Respiratory Other

45 Approved medicines Late-stage development AZD6738 (ATR inhibitor) Early development Cancer Oncology

Trial Population Patients Design Endpoints Status Phase I Solid tumours 160 • Arm 1: AZD6738 + carboplatin • Safety and tolerability • FPCD: Q4 2014 • Arm 2: AZD6738 dose escalation, AZD6738 + Lynparza • PK and efficacy • Data anticipated: H2 2019 NCT02264678 • Arm 3: AZD6738 + Imfinzi

Trial conducted in North America, Europe and South Korea CVRM

Phase I SCCHN 40 Window of opportunity • Biomarker change • FPCD: Q4 2017 • Arm 1: AZD6738 • Data anticipated: 2020 NCT03022409 • Arm 2: Lynparza

• Trial conducted in US, France, Taiwan and the UK Respiratory Other

46 Approved medicines Late-stage development AZD9150 (STAT3 inhibitor) Early development Cancer Oncology

Trial Population Patients Design Endpoints Status Phase Ib/II Head and neck squamous-cell 405 Dose escalation advanced solid and blood cancers • Safety/efficacy trial • FPCD: Q3 2015 carcinoma (HNSCC) • Arm A1: AZD9150/Imfinzi • Data anticipated: H2 2020 NCT02499328 • Arm A2 : AZD5069/Imfinzi • Arm A4: AZD9150/Imfinzi/treme CVRM • Arm A5: AZD5069/Imfinzi/treme Dose expansion 2L HNSCC: • Arm B1: AZD9150 • Arm B2: AZD5069 • Arm B3: AZD9150/Imfinzi • Arm B4: AZD5069/Imfinzi • Arm B5: AZD9150 mono • Arm B6: AZD5069 mono • Arm B7: AZD9150/Imfinzi (1L HNSCC) • Arm B8: AZD9150 Q2W/Imfinzi (1L HNSCC) Phase Ib/II NSCLC, advanced solid 213 Dose escalation advanced solid and blood cancers • Safety/efficacy trial • FPCD: Q1 2018 tumours • Arm A1: AZD9150 alternate week/Imfinzi • Data anticipated: 2020 Respiratory NCT03421353 • Arm A2-A5 : AZD9150/Imfinzi + SoC chemotherapy Dose Expansion 1L HNSCC: • Arm D1/D2/D3: AZD9150 IV vs SC formulations/Imfinzi (advanced solid tumours) Other

47 Approved medicines Late-stage development AZD9496 (selective estrogen receptor degrader) Early development Breast cancer Oncology

Trial Population Patients Design Endpoints Status

Phase I ER+ Breast Cancer c. 50 • This is an open label randomised multicentre pre-surgical • Primary Outcome Measures: • FPCD: Q4 2017 pharmacodynamics trial to compare and assess the Pharmacodynamics changes to • Data anticipated: H2 2019 NCT03236974 biological effects of AZD9496 and Faslodex in estrogen receptor (ER) expression

postmenopausal women with oestrogen receptor positive following treatment with AZD9496 or CVRM Faslodex (ER+), human epidermal growth factor receptor 2 negative • Secondary Outcome Measures: (HER2-) primary breast cancer. Patients will receive Pharmacodynamics changes to Ki67 AZD9496 or Faslodex and will have a pre-dose and an on- and progesterone receptor (PgR) treatment core biopsy after 5-14 days of commencing expression following treatment with treatment. AZD9496 or Faslodex Phase I ER+ Breast Cancer c. 50 • This is a Phase I open label multicentre trial of AZD9496 • Primary Outcome Measures: Safety and • FPCD: Q4 2014 administered orally in patients with advanced ER+ HER2 tolerability • LPCD: Q2 2016 NCT02248090 negative breast cancer. The trial design allows an escalation • Secondary Outcome Measures: Single • Data readout: Q2 2017 of dose with intensive safety monitoring to ensure the safety and multiple dose pharmacokinetics of of patients. The trial will determine the maximum tolerated AZD9496

dose. In addition, expansion cohort(s) at potential 4β-hydroxycholesterol concentration in Respiratory therapeutic dose(s) in patients with or without ESR1 blood mutations will be enrolled to further determine the safety, • Anti-tumour activity tolerability, pharmacokinetics and biological activity of AZD9496 Phase I Healthy subjects 14 • This is a Phase I open label single centre trial to assess the • Primary Outcome Measures: • FPCD: Q2 2016 pharmacokinetics and safety of different forms and Pharmacokinetics for AZD9496 and its • LPCD: Q3 2016 NCT02780713 formulations of AZD9496 in healthy subjects metabolites • Data readout: Q2 2017

• Secondary Outcome Measures: Safety Other and tolerability

48 Approved medicines Late-stage development AZD9833 (selective estrogen receptor degrader) Early development Breast cancer Oncology

Trial Population Patients Design Endpoints Status Phase I ER+ breast cancer 240 • This is a Phase I open label multicentre trial of AZD9833 • Primary outcome measures: safety and • FPCD: Q4 2018 administered orally in patients with advanced ER+ HER2 tolerability NCT03616587 negative breast cancer. The trial design allows an escalation • Secondary outcome measures: multiple of dose with intensive safety monitoring to ensure the safety

dose pharmacokinetics of AZD9833 CVRM of patients. The trial will determine the maximum tolerated alone and in combination with dose of AZD9833 as monotherapy and in combination with palbociclib. palbociclib. In addition, randomised expansion cohort(s) at Antitumour activity potential therapeutic dose(s) in patients will be enrolled to further determine the safety, tolerability, pharmacokinetics and biological activity of AZD9833 alone and in combination with palbociclib. Respiratory Other

49 Approved medicines Late-stage development MEDI2228 (BCMA antibody drug conjugate) Early development Cancer Oncology

Trial Population Patients Design Endpoints Status Phase I Relapsed/Refractory Multiple 129 First-time-in-human Phase I, multi-centre, open-label, single-arm, Primary endpoints: • FPCD: Q2 2018 Myeloma dose-escalation, and dose-expansion trial for adult subjects • Safety • Data anticipated: 2020+ NCT03489525 • Determination of MTD

• Secondary endpoints: pharmacokinetics, CVRM immunogenicity, ORR, DoR, PFS, OS Respiratory Other

50 Approved medicines Late-stage development MEDI3726 (PSMA antibody drug conjugate) Early development Cancer Oncology

Trial Population Patients Design Endpoints Status Phase I/Ib Subjects with metastatic 224 Open-label, Dose-escalation and Dose-expansion Primary endpoint: • FPCD: Q1 2017 castration resistant prostate • Three arm trial • Safety • Data anticipated: 2020+ NCT02991911 cancer • Post-chemo Secondary endpoints • Pre-chemo • RECIST response • MEDI3726+Enzalutamide • PSA50 response CVRM • CTC response • Pharmacokinetics, and immunogenicity Respiratory Other

51 Approved medicines Late-stage development MEDI5083 (CD40 Ligand fusion protein ) Early development + Imfinzi (PD-L1 mAb) Oncology Cancer

Trial Population Patients Design Endpoints Status CVRM Phase I Advanced solid tumours 204 Dose-escalation phase Primary endpoints: • FPCD: Q1 2017 • Part 1: MEDI5083 • Safety • Data anticipated: 2020+ NCT03089645 • Part 2: MEDI5083 + Imfinzi IV • Determination of MTD

• Secondary endpoints: preliminary anti- Dose expansion phase tumour activity, pharmacokinetics, • Part 3: MEDI5083 recommended dose + Imfinzi IV pharmacodynamics, and immunogenicity US and Australian trial centres Respiratory Other

52 Approved medicines Late-stage development MEDI5752 (PD-1/CTLA-4 bispecific mAb) Early development Cancer Oncology

Trial Population Patients Design Endpoints Status Phase I Advanced solid tumour 263 Open-label, Dose-escalation and Dose-expansion Primary endpoints: • FPCD: Q2 2018 • Dose-escalation: Safety & • Data anticipated: 2020+ NCT03530397 Dose-escalation: MEDI5752 IV determination of MTD • Dose-expansion: Assessment of Dose-expansion : 2 cohorts with 2 arms each antitumour activity based on OR CVRM

Secondary endpoints: • PK, ADA, tumoural baseline PD-L1, Assessment of antitumour activity based on OR, DoR, DC, PFS, OS, Respiratory Other

53 Approved medicines Late-stage development MEDI7247 (PBD ADC mAb) Early development Cancer Oncology

Trial Population Patients Design Endpoints Status Phase I Relapsed/Refractory 408 First-time-in-human Phase I, multi-centre, open-label, single-arm, • Primary endpoint: safety • FPCD: Q2 2017 NCT03106428 Haematological Malignancies dose-escalation, and dose-expansion trial for adult subjects • Data anticipated: 2020+ • Secondary endpoints: Pharmacokinetics, immunogenicity and antitumour activity CVRM

Phase I/Ib Advanced or metastatic solid 336 Open-label, dose-escalation and dose-expansion. • Primary endpoint: safety • FPCD: Q4 2018 tumours • Data anticipated: 2020+ NCT03811652 • Secondary endpoints: Pharmacokinetics, immunogenicity and antitumour activity Respiratory Other

54 AstraZeneca

CVRM, Respiratory and Other medicines – approved medicines and late-stage pipeline Approved medicines Late-stage development Farxiga (SGLT2 inhibitor) Early development Diabetes Oncology

Trial Population Patients Design Endpoints Status Phase III/IV Type-2 diabetes 17,190 • Arm 1: Farxiga 10mg QD + SoC therapy QD • Primary endpoints: Superiority for major • FPCD: Q2 2013 DECLARE with high risk for CV event • Arm 2: Placebo + SoC therapy for type-2 Diabetes adverse cardiac events (MACE) (CV • LPCD: Q2 2015 death, non-fatal MI (myocardial • Data Readout: Q3 2018 NCT01730534 Global trial – 33 countries infarction) or non-fatal stroke). • Met primary safety endpoint and one of Superiority for the composite endpoint of two primary efficacy endpoints (hHF or CVRM CV death or hospitalisation for heart CV death) failure.

Phase III Patients with type-2 diabetes 302 • Arm 1: Farxiga 10mg QD for 24 weeks • Primary endpoint: Change from baseline • FPCD: Q2 2015 DERIVE and moderate renal • Arm 2: Placebo 10mg QD for 24 weeks in HbA1c at week 24 • LPCD: Q2 2017 impairment • Data readout: Q1 2018 NCT02413398 Global trial – eight countries • Primary endpoint met

Phase III Type-1 diabetes 833 • Arm 1: Farxiga 5mg QD 52 weeks + insulin • Primary endpoint: : Adjusted Mean • FPCD: Q4 2014 DEPICT 1 • Arm 2: Farxiga 10mg QD 52 weeks + insulin Change From Baseline in HbA1c at • LPCD Q2 2016

• Arm 3: Placebo QD 52 weeks + insulin week 24 • Data readout: Q1 2017 Respiratory NCT02268214 • Primary endpoint met Global trial – 17 countries Partnered Phase III Type-1 diabetes 813 • Arm 1: Farxiga 5mg QD 52 weeks + insulin • Primary endpoint: Adjusted Mean • FPCD: Q3 2015 DEPICT 2 • Arm 2: Farxiga 10mg QD 52 weeks + insulin Change From Baseline in Haemoglobin • LPCD: Q1 2017 • Arm 3: Placebo QD 52 weeks + insulin A1C (HbA1c) at week 24 • Data readout: Q4 2017 NCT02460978 • Primary endpoint met

Global trial – 14 countries Other Partnered

56 Approved medicines Late-stage development Farxiga (SGLT2 inhibitor) Early development Diabetes / cardiovascular risk reduction Oncology

Trial Population Patients Design Endpoints Status

Phase III Chronic Heart Failure (CHF) 4,744 • Arm 1: Farxiga 10mg or 5 mg QD + standard of care therapy • Primary endpoint: Time to the first • FPCD: Q1 2017 Dapa-HF patients with reduced ejection • Arm 2: Placebo + standard of care therapy occurrence of any of the components of • LPCD Q3 2018 fraction (HFrEF) the composite: CV death or • Data anticipated: H2 2019 • Global trial - 20 countries

NCT03036124 hospitalisation for heart failure (HF) or CVRM an urgent HF visit

Phase III Patients With Chronic Kidney 4,000 • Arm 1: Farxiga 10mg or 5 mg QD • Primary endpoint: Time to the first • FPCD: Q1 2017 Dapa-CKD Disease (CKD) • Arm 2: Placebo occurrence of any of the components of • LPCD: Q1 2019 the composite: ≥50% sustained decline • Data anticipated: 2020+ NCT03036150 Global trial - 20 countries in estimated glomerular filtration rate (eGFR) or reaching end stage renal disease (ESRD) or CV death or renal death Phase III Chronic Heart Failure (CHF) 4,700 • Arm 1: Farxiga 10mg QD • Primary endpoint: Time to the first • FPCD: Q3 2018

DELIVER patients with preserved • Arm 2: Placebo occurrence of any of the components of • Data anticipated: 2020+ Respiratory ejection fraction (HFpEF) the composite: CV death or NCT03619213 • Global trial - 21 countries hospitalisation for heart failure (HF) or an urgent HF visit Other

57 Approved medicines Late-stage development Brilinta (ADP receptor antagonist) Early development Cardiovascular risk reduction Oncology

Trial Population Patients Design Endpoints (primary) Status

Phase III Patients with type-2 diabetes 19,000 • Arm 1: Brilinta 60mg BiD • Primary endpoint: Composite of • FPCD: Q1 2014 THEMIS and coronary artery disease • Arm 2: Placebo BID cardiovascular (CV) death, non-fatal MI • LPCD: Q2 2016 without a previous history of on a background of acetylsalicylic acid if not contra-indicated or and non-fatal stroke • Data readout: Q1 2019 NCT01991795 myocardial infarction (MI) or not tolerated • Primary endpoint met CVRM stroke Secondary endpoints: Global trial – 42 countries • Prevention of CV death • Prevention of MI • Prevention of ischaemic stroke • Prevention of all-cause death Phase III Patients with acute ischaemic 13,000 • Arm 1: Brilinta 90mg BiD Primary endpoint: • FPCD: Q1 2018 THALES stroke or transient ischaemic • Arm 2: placebo BiD • Prevention of the composite of • Data anticipated: 2020 attack on a background of acetylsalicylic acid if not contra-indicated or subsequent stroke and death at 30 days NCT03354429 not tolerated Secondary endpoints include: Global trial – 28 countries • Prevention of subsequent ischaemic

stroke at 30 days Respiratory • Reduction of overall disability at 30 days

Phase III Pediatric patients (2-18 years 182 • Arm 1: Brilinta 15, 30 or 45mg (dose based on subject • Primary endpoint: The number of • FPCD: Q3 2018 HESTIA3 old) with sickle cell disease weight) vaso-occlusive crisis which is the • Data anticipated: 2020+ • Arm 2: Placebo composite of painful crisis and/or NCT03615924 acute chest pain Global trial – 18 countries Other

58 Approved medicines Late-stage development Epanova (omega-3 carboxylic acids) Early development Hypertriglyceridaemia Oncology

Trial Population Patients Design Endpoints Status Phase III Patients with 13,000 • Arm 1: Epanova 4g QD + statin • Primary endpoint: Composite of Major • FPCD: Q4 2014 STRENGTH (CVOT) hypertriglyceridaemia and • Arm 2: Placebo (corn oil) + statin Adverse Cardiac Events (MACE) • LPCD: Q2 2017 high cardiovascular disease • Data anticipated: 2020 NCT02104817 risk Global trial – 22 countries CVRM

Phase III Japanese patients with 375 • Epanova 2g and 4g vs. Placebo (after meal) daily for 52 weeks Primary endpoints: • FPCD: Q2 2015 hypertriglyceridaemia • Safety in Japanese patients • LPCD: Q1 2016 NCT02463071 Global trial – one country • percentage change in triglycerides • Data readout: Q2 2017

Phase III Severe hypertriglyceridaemia 162 • Arm 1: Epanova 2g QD • Primary endpoint: Change in serum • FPCD: Q4 2013 EVOLVE II • Arm 2: Placebo (olive oil) triglycerides over 12 weeks • LPCD: Q4 2014 • Data readout: Q4 2015 NCT02009865 Global trial – seven countries • Primary endpoint met Respiratory

Phase I Healthy Chinese subjects 14 Open-label trial to evaluate the pharmacokinetics of single and • Primary endpoints: Plasma • FPCD: Q2 2018 China PK multiple doses of Epanova 4 g/day in Chinese healthy subjects concentrations versus time profile of • LPCD: Q2 2018 EPA and DHA to assess PK parameters • Data readout: Q4 2018 NCT03574142 Local trial – China Other

59 Approved medicines Late-stage development Lokelma (sodium zirconium cyclosilicate) Early development Hyperkalaemia Oncology

Trial Population Patients Design Endpoints Status Phase III Hyperkalaemia 248 Open-label Lokelma 10g TID for 48 hours followed by: • Primary endpoint: Maintenance of • FPCD: Q1 2017 • Arm 1: Lokelma 5g QD for 28 days normokalaemia • LPCD: Q1 2018 Harmonize Global • Arm 2: Lokelma 10g QD for 28 days • Data readout: Q4 2018

NCT02875834 • Arm 3: Placebo QD for 28 days • Primary endpoint met CVRM

Global trial – four countries Phase II/III Hyperkalaemia 103 Arm 1: Lokelma 5g TID for 48 hours • Primary endpoint: Exponential rate of • FPCD: Q2 2017 Arm 2: Lokelma 10g TID for 48 hours change in serum potassium • LPCD: Q1 2018 NCT03127644 Arm 3: Placebo TID for 48 hours • Data readout: Q3 2018 Japan • Primary endpoint met Phase III Hyperkalaemia 150 Arm 1: Open-label Lokelma 10g TID for up to 72 hrs followed by • Primary endpoint: Safety and tolerability • FPCD: Q3 2017 Lokelma 5g QD for 12 months. Option to uptitrate to 10 and15g as measured by adverse events NCT03172702 QD or downtitrate to 5g QOD (or 2.5g QD) reporting, vital signs, ECGs, physical examinations and safety laboratory

Japan measurements Respiratory Phase I Healthy Subjects 22 Arm 1: Open-label Lokelma 5g QD for 4 days • Primary endpoint: Mean change from • FPCD: Q4 2017 Arm 2: Open-label Lokelma 10g QD for 4 days baseline to Lokelma treatment period in • LPCD: Q4 2017 NCT03283267 urine potassium excretion • Data readout: Q1 2018 China Phase IIIb Patients on haemodialysis 180 Arm 1: Lokelma 5g QD for 8 weeks on non-dialysis days. Option • Primary endpoint: Proportion of patients • FPCD: Q4 2017 with persistent pre-dialysis to uptitrate to 10 and15g QD. who maintain a pre-dialysis serum K • LPCD: Q4 2018

DIALIZE hyperkalaemia Arm 2: Placebo QD for 8 weeks on non-dialysis days between 4.0-5.0 mmol/L on 3 out of 4 • Primary endpoint met Other NCT03303521 dialysis treatments following the long Global trial – four countries interdialytic interval Phase II Hyperkalaemia 132 Arm 1: Lokelma 10g TID for 24 hours on top off SoC (insulin and • Primary endpoint: Mean absolute • FPCD: Q1 2018 glucose) change in S-K from baseline until 4h • LPCD: Q4 2018 ENERGIZE Arm 2: Placebo TID for 24 hours on top off SoC (insulin and after start of dosing NCT03337477 glucose)

Global trial – four countries Phase II Patients with chronic heart 280 Arm 1: Lokelma 5g QD for 12 weeks. Option to uptitrate to 10 and • Primary endpoint: Difference between • FPCD: Q3 2018 failure and hyperkalaemia or 15g QD or downtitrate to 5g QOD Lokelma and placebo in RAAS (renin– PRIORITIZE HF at high risk of developing Arm 2: Placebo QD for 12 weeks angiotensin–aldosterone system) NCT03532009 hyperkalaemia blockade treatment. Global trial – six countries

60 Approved medicines Late-stage development Roxadustat (China: 爱瑞卓) (HIF-PHI inhibitor) Early development Anaemia Oncology

Trial Population Patients Design Endpoints Status

Phase III ANDES Anaemia in CKD (Chronic 922 • Arm 1: roxadustat • Primary endpoint: Haemoglobin • FPCD: Q4 2012 Kidney Disease) patients not • Arm 2: placebo response • Data readout: Q4 2018 NCT01750190 receiving dialysis • Primary endpoint met Partnered Global trial Sponsored by FibroGen CVRM

Phase III ALPS 597 • Arm 1: roxadustat • Primary endpoint: Haemoglobin • FPCD: Q2 2013 • Arm 2: Placebo response • Data readout: Q3 2018 NCT01887600 • Primary endpoint met Partnered Global trial Sponsored by Astellas

Phase III DOLOMITES 616 • Arm 1: roxadustat • Primary endpoint: Haemoglobin • FPCD: Q1 2014 • Arm 2: darbepoetin alfa response • Data anticipated: H1 2019 NCT02021318 Partnered Global trial Sponsored by Astellas Respiratory Phase III OLYMPUS 2,781 • Arm 1: roxadustat • Primary efficacy endpoint: Haemoglobin • FPCD: Q3 2014 • Arm 2: Placebo response • Data readout: Q4 2018 NCT02174627 • Primary endpoint met Global trial • Primary safety objective: Contribute CV safety data to pooled safety Sponsored by AstraZeneca • analyses across the Phase III program

Phase III ROCKIES 2,133 • Arm 1: roxadustat

Anaemia in CKD in patients • Primary efficacy endpoint: Haemoglobin • FPCD: Q3 2014 Other receiving dialysis • Arm 2: epoetin alfa response • Data readout: Q4 2018 NCT02174731 • Primary endpoint met Global trial • Primary safety objective:Contribute CV safety data to pooled safety Sponsored by AstraZeneca • analyses across the Phase III program

Phase III SIERRAS 820 • Arm 1: roxadustat • Primary endpoint: Haemoglobin • FPCD: Q4 2014 • Arm 2: epoetin alfa response • Data readout: Q4 2018 NCT02273726 • Primary endpoint met Partnered Global trial Sponsored by FibroGen

Phase III PYRENEES 838 • Arm 1: roxadustat • Primary endpoint: Haemoglobin • FPCD: Q4 2014 • Arm 2: erythropoiesis stimulating agent response • Data anticipated: H2 2018 NCT02278341 • Arm 3: darbepoetin alfa Partnered Sponsored by Astellas Global trial HIF-PHI = Hypoxia-Inducible Factor Prolyl Hydroxylase Inhibitor 61 Approved medicines Late-stage development Roxadustat (China: 爱瑞卓) (HIF-PHI inhibitor) Early development Anaemia Oncology

Trial Population Patients Design Endpoints Status Phase III HIMALAYAS Anaemia in newly initiated 900 • Arm 1: roxadustat • Primary endpoint: Haemoglobin • FPCD: Q4 2013 dialysis patients • Arm 2: epoetin alfa response • Data readout: Q4 2018 NCT02052310 • Primary endpoint met Partnered Global trial Sponsored by FibroGen CVRM Phase III Anaemia in CKD (Chronic 154 • Arm 1: roxadustat • Primary endpoint: Haemoglobin • FPCD: Q4 2015 Kidney Disease) patients not • Arm 2: placebo response • LPCD: Q4 2016 NCT02652819 receiving dialysis • Data readout: Q2 2017 Partnered China trial • Primary endpoint met

Sponsored by FibroGen Phase III Anaemia in CKD patients 305 • Arm 1: roxadustat • Primary endpoint: Haemoglobin • FPCD: Q4 2015 receiving dialysis • Arm 2: epoetin alfa response • LPCD: Q2 2016 NCT02652806 • Data readout: Q2 2017

Partnered China trial • Primary endpoint met Respiratory

Sponsored by FibroGen

Phase III Anaemia in lower risk 184 Open label roxadustat lead-in • Primary endpoint: Proportion of patients FPCD: Q3 2017 Myelodysplastic Syndrome Arm 1: roxadustat achieving transfusion independence NCT03263091 (MDS) patients Arm 2: placebo Sponsored by FibroGen Partnered US/global trial Other Phase II/III Anaemia in lower risk MDS 175 Open label roxadustat lead-in • Primary endpoint: Haemoglobin patients Arm 1: roxadustat response Sponsored by FibroGen NCT03303066 Arm 2: placebo Partnered China HIF-PHI = Hypoxia-Inducible Factor Prolyl Hydroxylase Inhibitor

62 Approved medicines Late-stage development Eklira/Tudorza (LAMA, DPI) Early development Chronic obstructive pulmonary disease (COPD) Oncology

Trial Population Number of patients Design Endpoints Status

Phase I Healthy Chinese 18 Open-label, 2-period ascending dose incomplete block, cross-over • To investigate the pharmacokinetics • FPCD: Q2 2018 subjects trial (PK) of aclidinium bromide and its • Data anticipated: H2 2019 NCT03276052 metabolites after single and multiple • Arm 1: Aclidinium bromide 200 μg DPI doses (twice-daily [BID]) of aclidinium • Arm 2: Aclidinium bromide 400 μg DPI bromide 200 μg, 400 μg and 800 μg CVRM • Arm 3: Aclidinium bromide 800 μg DPI • To evaluate the safety, and tolerability of aclidinium bromide 200 μg, 400 μg and 800 μg after single and multiple dose Global trial – one Country administration (twice-daily [BID]) Respiratory Other

63 Approved medicines Late-stage development Duaklir Genuair (LAMA/LABA, DPI) Early development Chronic obstructive pulmonary disease (COPD) Oncology

Trial Population Patients Design Endpoints Status

Phase III Patients with stable COPD 1,060 • Arm 1: Duaklir Genuair 400/12 μg DPI Primary endpoints: • FPCD: Q1 2017 AVANT • Arm 2: aclidinium bromide 400 μg DPI • Change from baseline in one hour • Data anticipated: 2020 • Arm 3: formoterol fumarate 12 μg DPI morning post-dose dose FEV1 Duaklir NCT03022097 • Arm 4: tiotropium 18 μg DPI Genuair 400/12 μg compared to CVRM Aclidinium bromide at Week 24 • Change from baseline in morning pre- Global trial – five countries dose (trough) FEV1 of Duaklir Genuair 400/12 μg compared to Formoterol fumarate at Week 24 • Change from baseline in trough FEV1 of Aclidinium bromide 400 µg compared to placebo at Week 24 Respiratory Other

64 Approved medicines Late-stage development Bevespi Aerosphere (LAMA/LABA, pMDI) Early development Chronic obstructive pulmonary disease (COPD) Oncology

Trial Population Patients Design (G = glycopyrronium, F = formoterol fumarate) Endpoints Status

Phase IIIb Moderate to very severe 1,000 Treatments (24-week treatment period) Co-primary endpoints: • FPCD: Q2 2017 AERISTO COPD • GFF MDI (Bevespi Aerosphere) 14.4/9.6μg BID pMDI • Change from baseline in morning pre- • LPCD: Q4 2017 • Umeclidinium/vilanterol DPI 62.5/25μg QD dose trough FEV1 over 24 weeks • Data readout: Q3 2018 • Peak change from baseline in FEV1

NCT03162055 CVRM Randomised, double-blind, double-dummy, multi-centre, parallel within two hours post-dosing over 24 group weeks

US, Canada, Bulgaria, France, Hungary, Russia, Ukraine Respiratory Other

65 Approved medicines Late-stage development Daliresp/Daxas (PDE4 inhibitor, oral) Early development Chronic obstructive pulmonary disease (COPD) Oncology

Trial Population Patients Design Endpoints Status

Phase IV COPD 2,354 • 52W, randomised, DB with Daliresp 500µg OD vs. placebo, in • Primary endpoint: Rate of moderate or • FPCD: Q4 2011 RESPOND COPD on top of ICS/LABA severe COPD exacerbations per subject • LPCD: Q1 2016 per year • Data readout: Q4 2016

NCT01443845 CVRM

Phase IV COPD 1,323 • 12W, randomised, DB to evaluate tolerability and PK of • Primary endpoint: Percentage of • FPCD: Q2 2014 OPTIMIZE Daliresp 500μg OD with an up-titration regimen during the first participants prematurely discontinuing • LPCD: Q3 2015 4Ws, including an open label down-titration evaluating trial treatment for any reason during the • Data readout: Q4 2016 NCT02165826 tolerability and PK of 250µg Daliresp OD in patients not main period tolerating 500μg OD Phase IIIb COPD 158 • 16W, randomised, placebo-controlled, DB, parallel-group trial • Primary endpoint: Number of • FPCD: Q1 2012 ROBERT to assess the anti-inflammatory effects of Daliresp in COPD inflammatory cells CD8+ in bronchial • LPCD: Q1 2016 biopsy tissue specimen (sub-mucosa) • Data readout: Q4 2016 NCT01509677 measured at randomisation and at the

end of the intervention period Respiratory Post Launch COPD 124,080 • This is a retrospective cohort trial comparing COPD patients • Primary endpoint: All-cause mortality (up • Data anticipated: 2020+ PASS aged 40 years and older with new exposure to roflumilast with to five years) up to 5 unexposed (i.e., not roflumilast-exposed) COPD NCT03381573 controls matched by propensity score (PS), age, sex, and year of cohort entry. The trial is using electronic healthcare databases in the US (Military Health System database), Germany (German Pharmacoepidemiological Research Database), and Sweden (national databases including Other healthcare, death, and demographics data).

66 Approved medicines Late-stage development Fasenra (IL-5R mAb) Early development Severe, uncontrolled asthma Oncology

Trial Population Patients Design Endpoints Status

Phase III A multi-centre, open-label, 770 • Arm 1: Fasenra 30mg Q4W SC • Primary endpoint: Safety and tolerability • FPCD: Q2 2016 MELTEMI safety extension trial with • Arm 2: Fasenra 30mg Q8W SC • Data anticipated: 2020 Fasenra for asthmatic adults on ICS plus LABA2 Agonist NCT02808819 Global trial - 15 countries CVRM Age 18-75 years

Phase IIIb Severe eosinophilic 600 Arm 1: Fasenra 30mg Q8W SC • Primary endpoint: Reduction of oral • FPCD: Q3 2018 PONENTE asthmatics receiving HD (high corticosteroid dose • Data anticipated: 2020 dose) ICS + LABA and chronic 38-week trial NCT03557307 OCS with or without additional Global trial – 16 countries asthma controller(s). Age: 18 Years and older D3250C00036 China Severe, uncontrolled asthma, 666 • Arm 1: Fasenra 30mg Q8W SC • Primary endpoint: Annual asthma • FPCD: Q3 2017 ICS/LABA Trial (MIRACLE) despite background controller • Arm 2: Placebo SC exacerbation rate • Data readout: 2020+ medication, medium dose • Secondary endpoints: Assess

NCT03186209 (MD) & high dose (HD) ICS + 56-week trial pulmonary function, asthma symptoms, Respiratory LABA ± chronic OCS Global trial – 4 countries other asthma control metrics Age 12-75 years Other

67 Approved medicines Late-stage development Fasenra (IL-5R mAb) Early development Severe, uncontrolled asthma Oncology

Trial Population Patients Design Endpoints Status

Phase III Severe asthma, inadequately 2,550 • Arm 1: Fasenra 30mg Q4W SC • Primary endpoint: Safety and tolerability • FPCD: Q4 2014 BORA controlled despite background • Arm 2: Fasenra 30mg Q8W SC* • Data readout: Q3 2018 controller medication, MD • Primary endpoint met (medium dose) & HD (high NCT02258542 • Placebo administered at select interim visits to CVRM dose) ICS + LABA ± chronic maintain blind between treatment arms OCS Age 12-75 years 56-week (adults) 108-week (adolescents) Global trial – 24 countries

Phase III Severe asthma, inadequately 120 • Arm 1: Fasenra 30mg Q4W SC • Primary endpoint: Functionality, • FPCD: Q2 2015 GREGALE controlled despite background reliability, and performance of a pre-filled • Data readout: Q2 2016 controller medication, MD & 28-week (adults) syringe with Fasenra administered at • Primary endpoint met NCT02417961 HD ICS + LABA ± chronic Global trial – two countries home OCS Age 18-75 years Respiratory

Phase lll A double-blind, randomised, 38 • Arm 1 : Fasenra 30mg Q4W SC • Primary endpoint: Safety and tolerability • FPCD Q4 2016 ARIA parallel group, placebo- • Arm 2: placebo SC • Data anticipated: H2 2019 controlled multi-centre trial to • Primary endpoint: the effect of Fasenra NCT02821416 evaluate the effect of Fasenra 37-week trial on allergen induced eosinophil changes on allergen-induced in sputum and allergen-induced late inflammation in Mild, atopic asthmatic response asthmatic Other Age 18-65 years

Phase lll A multi-centre, randomised, 100 • Arm1 Fasenra 30mg Q4W SC with one dose of seasonal Primary endpoints: • FPCD: Q3 2016 ALIZE double-blind, parallel group, influenza virus vaccine Intramuscular (IM) at week eight • Post-dose strain-specific • Data readout: Q3 2017 placebo-controlled, Phase IIIb • Arm1 Placebo Q4W SC with one dose of seasonal influenza haemagglutination-inhibition (HAI) • Primary endpoint met NCT02814643 trial to evaluate the potential virus vaccine intra muscular at week antibody geometric mean fold rises effect of Fasenra on the (GMFRs) humoral immune response to 12-week trial • Post-dose strain-specific serum HAI the seasonal influenza antibody geometric mean titers (GMTs) vaccination in adolescent and • Proportion of patients who experience a young adult patients with strain-specific post-dose antibody severe asthma response with antibody response Ages 12-21 years defined as a ≥4-fold rise in HAI antibody titer

68 Approved medicines Late-stage development Fasenra (IL-5R mAb) Early development Severe, uncontrolled asthma Oncology

Trial Population Patients Design Endpoints Status

Phase III Severe uncontrolled asthma 230 • Arm 1: Fasenra 30mg Q4W SC • Primary endpoint: Onset and • FPCD: Q4 2016 SOLANA despite background controller • Arm 2: Placebo SC maintenance of effect on lung function • Data anticipated: Q3 2018 medication HD ICS + LABA • Primary endpoint not met

NCT02869438 with or without controllers 16-week trial CVRM Age 18-75 years Global trial – six countries

Phase III Severe asthma on ICS-LABA 120 Open label Fasenra 30mg Q4w • Primary endpoint: percentage of • FPCD: Q4 2016 GRECO Age 18-75 years patients/ caregivers who successfully • Data readout: Q4 2017 28-week trial self administer at home • Primary endpoint met NCT02918071 Global trial - two countries Respiratory

Phase lllb A multi-centre, randomised, 800 • Arm 1: Fasenra 30mg Q8W SC • Primary endpoint: rate of asthma • FPCD: Q3 2017 ANDHI double-blind, parallel group, • Arm 2: placebo SC exacerbations • Data anticipated: 2020 placebo controlled, Phase IIIb • Secondary outcome measures: Saint NCT03170271 trial to evaluate the safety and 24-week trial George Respiratory Questionnaire efficacy of Fasenra 30 mg sc Global trial – 15 countries (SGRQ) in patients with severe asthma uncontrolled on SoC Other treatment. Age 18-75

Phase I Healthy Volunteer 162 Open label trial to compare 30 mg Fasenra PK administered by • Primary endpoint: PK comparability • FPCD: Q1 2017 AMES Age 18-55 years APFS or AI device • Data readout: Q3 2017

NCT02968914 8-week trial Global trial – two countries

69 Approved medicines Late-stage development Fasenra (IL-5R mAb) Early development Nasal polyps Oncology

Trial Population Patients Design Endpoints Status Phase III Patients with severe bilateral 400 • Arm 1: Fasenra 30mg Q8W SC • Primary endpoint: Effect of Fasenra on • FPCD: Q1 2018 OSTRO nasal polyps who are still • Arm 2: Placebo SC nasal polyp burden and on patient • Data anticipated: 2020 symptomatic despite standard reported nasal blockage of care therapy

NCT03401229 56-week trial CVRM Global trial- 8 countries Age 18-75 years Respiratory Other

70 Approved medicines Late-stage development PT010 (LAMA/LABA/ICS, pMDI) Early development Chronic obstructive pulmonary disease (COPD) Oncology

Trial Population Patients Design Endpoints Status Phase III Moderate to very severe 500 Treatments (52-week Treatment Period) Primary endpoints: • FPCD: Q3 2015 COPD • BGF (Budesonide, Glycopyrronium, and Formoterol Fumarate) • Bone Mineral Density sub-study • LPCD: Q3 2016 MDI 320/14.4/9.6µg BID pMDI Endpoint. Change from baseline in BMD • Data readout: Q1 2018 NCT02536508 • GFF (Glycopyrronium and Formoterol Fumarate) MDI of the lumbar spine measured using 14.4/9.6µg BID pMDI DXA (dual energy X-ray absorptiometry) CVRM • BFF (Budesonide and Formoterol Fumarate) MDI 320/9.6µg scans of L1-L4 at week 52 BID pMDI • Ocular Sub-study Safety Endpoint Randomised, double-blind, chronic-dosing, multi-centre Change from baseline in LOCS III at week 52. Country – US

Phase III Moderate to very severe 8,000 Treatments (1-year Treatment Period) • Primary endpoint: Rate of moderate or • FPCD: Q3 2015 ETHOS COPD (possible increase by • BGF MDI 320/14.4/9.6µg BID pMDI severe COPD exacerbations • LPCD: Q3 2018 4,000 after blinded • BGF MDI 160/14.4/9.6µg BID pMDI • Data anticipated: H2 2019 NCT02465567 sample size re- • BFF MDI 320/9.6µg BID pMDI • Secondary endpoint: Time to first assessment) • GFF MDI 14.4/9.6µg BID pMDI moderate or severe COPD exacerbation

Randomised, double-blind, multi-centre and parallel-group Respiratory

Multi-country

Phase III Moderate to very severe 1,800 Treatments (24-week Treatment Period) Primary Endpoints: • FPCD: Q3 2015 KRONOS COPD • BGF MDI 320/14.4/9.6µg BID pMDI • FEV1 area under curve from 0 to 4 hours • LPCD: Q2 2017 • GFF MDI 14.4/9.6µg BID pMDI (AUC0-4) over 24 weeks (BGF MDI vs. • Data readout: Q1 2018 NCT02497001 • BFF MDI 320/9.6µg BID pMDI BFF MDI and BGF MDI vs. Symbicort • 8/9 Primary endpoints met • Symbicort Turbuhaler 400/12µg BID DPI Turbuhaler)

• Change from baseline in morning pre- Other Randomised, double-blind, parallel-group, and chronic dosing and dose trough FEV1 over 24 weeks (BGF multi-centre MDI vs. GFF MDI) • Transition dyspnoea index (TDI) focal score over 24 weeks (BGF MDI vs. BFF Multi-country MDI and BGF MDI vs. GFF MDI)

Phase III Moderate to very severe 324 Treatments (28-week Treatment Period) Primary outcome measures: • FPCD Q3 2016 COPD • BGF MDI 320/14.4/9.6µg BID pMDI • Long-term safety and tolerability (52 • LPCD Q4 2017 NCT03262012 • GFF MDI 14.4/9.6µg BID pMDI weeks): adverse events, 12-lead ECG, • Data readout: Q3 2018 • BFF MDI 320/9.6µg BID pMDI laboratory tests, vital signs • Symbicort Turbuhaler 400/12µg BID DPI

Randomised, double-blind, parallel-group, chronic dosing, multicenter

Country: Japan

71 Approved medicines Late-stage development PT027 (ICS/SABA, pMDI) Early development Asthma Oncology

Trial Population Patients Design Endpoints Status

Phase III Moderate to severe asthma 3,100 Treatments (minimum 24-week treatment period) Primary endpoint: • FPCD: Q4 2018 MANDALA • BDA (budesonide albuterol) MDI 80/180 μg prn • Time to first severe asthma exacerbation • Data anticipated: 2020+ • BDA MDI 160/180 μg prn NCT03769090 • AS (albuterol sulphate) MDI 180 μg prn Secondary endpoints: CVRM • Severe exacerbation rate (annualised) Managed by Avillion Randomised, double-blind, multi-centre, parallel group • Total corticosteroid exposure over the treatment period Multi-country • Asthma Control Questionnaire -5 change from baseline and responder analysis at Week 24 • Asthma Quality of Life Questionnaire for 12 years and older/Pediatric Asthma Quality of Life Questionnaire change from baseline and responder analysis at

Week 24 Respiratory Phase III Mild to moderate asthma 600 Treatments (12 week treatment period) Dual primary endpoints: Initiating DENALI • BDA MDI 80/180 μg QID • Change from baseline in FEV1 AUC0-6 • BDA MDI 160/180 μg QID hours over 12 weeks • BD MDI 160 μg QID • Change from baseline in trough FEV1 at Managed by Avillion • AS MDI 180 μg QID Week 12 • Placebo MDI QID

Randomised, double-blind, multi-centre and parallel-group Other

Multi-country

72 Approved medicines Late-stage development Tezepelumab (TSLP mAb) Early development Severe, uncontrolled asthma Oncology

Trial Population Patients Design Endpoints Status

Phase III Severe asthma 1,060 • Arm 1: tezepelumab SC • Primary endpoint: Annual asthma • FPCD: Q1 2018 NAVIGATOR Age 12-80 years • Arm 2: placebo SC exacerbation rate • Data anticipated: 2020 • Secondary endpoints: Change from

NCT03347279 52 week trial baseline in pre-BD FEV1, asthma related CVRM QoL (AQLQ(S)+12), asthma control Partnered Global trial – 18 countries (ACQ-6)

Phase III Severe asthma 152 • Arm 1: tezepelumab SC • Primary endpoint: Reduction from • FPCD: Q2 2018 SOURCE Age 18-80 years • Arm 2: placebo SC baseline in daily OCS dose while not losing asthma control NCT03406078 48 week trial • Secondary endpoint: Annual asthma exacerbation rate Partnered Global trial – seven countries Respiratory

Phase III Severe asthma ~975 • Arm 1: tezepelumab SC • Primary endpoint: Exposure adjusted • FPCD: Q1 2019 DESTINATION Age 12-80 years • Arm 2: placebo SC rates of AEs/SAEs Secondary endpoints: Annual asthma exacerbation rate NCT03706079 Extension Study to NAVIGATOR and SOURCE. 52 week trial (subjects from NAVIGATOR); 56 week trial (subjects from Partnered SOURCE) Other Global trial – ~ 20 countries

73 Approved medicines Late-stage development Anifrolumab (type I IFN receptor mAb) Early development Systemic lupus erythematosus (SLE) / Lupus nephritis (LN) Oncology

Trial Population Patients Design Endpoints Status

Phase III Moderate to severe SLE 450 • Arm 1: 300mg IV anifrolumab Q4W for 48 weeks • Primary endpoint: Response in SLE • FPCD: Q3 2015 TULIP SLE 1 • Arm 2: 150mg IV anifrolumab Q4W for 48 weeks responder index at week 52 • Data readout: Q3 2018 NCT02446912 • Arm 3: placebo IV Q4W for 48 weeks • Primary endpoint not met

Phase III Moderate to severe SLE 360 • Arm 1: 300mg IV anifrolumab Q4W for 48 weeks • Primary endpoint: Response in SLE • FPCD: Q3 2015 CVRM TULIP SLE 2 • Arm 2: placebo IV Q4W for 48 weeks responder index at week 52 • Data anticipated: H2 2019 NCT02446899

Phase III Moderate to severe SLE 630 • Arm 1: 300mg IV anifrolumab Q4W for 152 weeks • Primary endpoint: Extension to evaluate • FPCD: Q2 2016 TULIP LTE • Arm 2: placebo IV Q4W for 152 weeks long-term safety and tolerability • Data anticipated: 2020+ NCT02794285 Phase ll Moderate to severe SLE 307 • Arm 1: 300mg IV anifrolumab Q4W for 48 weeks • Primary endpoint: Response in SLE • FPCD: Q1 2012 patients • Arm 2: 1000mg IV anifrolumab Q4W for 48 weeks responder index at 6 months • LPCD: Q1 2015 NCT01438489 • Arm 3: placebo IV Q4W for 48 weeks • Data readout: Q3 2014 Phase II Moderate to severe SLE 218 • Arm 1: anifrolumab, IV Q4W for 104 weeks • Primary endpoint: Open-label extension • FPCD: Q1 2013 Respiratory patients to evaluate long-term safety and • Data readout: Q4 2018 NCT01753193 tolerability

Phase II Moderate to severe SLE 32 • Arm 1: 150mg SC every other week • PK/PD, Safety, tolerability, Primary • FPCD: Q1 2017 patients • Arm 2: 300mg SC every other week analysis at week 12, Secondary analysis • Data readout: Q1 2018 NCT02962960 • Arm 3: placebo SC every other week at week 52

Phase II Active Proliferative LN (TULIP- 150 • Arm 1: 900 mg IV Q4W for 12 weeks then 300mg IV • Response in proteinuria at week 52 • FPCD: Q4 2015 Other LN1) anifrolumab Q4W for 36 weeks • Data anticipated: 2020 NCT02547922 • Arm 2: 300 mg IV anifrolumab Q4W for 48 weeks • Arm 3: placebo IV Q4W for 48 weeks

74 AstraZeneca

CVRM, Respiratory and Other medicines – early-stage development Approved medicines Late-stage development Verinurad (RDEA3170, URAT1 inhibitor) Early development Chronic kidney disease Oncology

Trial Population Patients Design Endpoints Status

Phase II CKD (Chronic Kidney 60 • Arm A: verinurad 9 mg and febuxostat 80 mg To assess the effects of intensive uric acid • FPCD: Q2 2017 Disease) patients with • Arm B: Placebo lowering therapy with RDEA3170 and • LPCD: Q3 2018 NCT03118739 hyperuricaemia, The trial is a multi-centre trial conducted in the US febuxostat on UACR (urine albumin • Data readout: Q4 2018

albuminuria, and Type 2 creatinine ratio) CVRM diabetes

Phase II Asymptomatic hyperuricemic 36 • Arm A: 9 mg verinurad + 80 mg febuxostat + 10 mg dapagliflozin Primary: Peak uric acid excretion during • FPCD: Q4 2017 subjects (sUA (serum uric acid • Arm B: 9 mg verinurad + 80 mg febuxostat + placebo the first 8 hours) on Day 7 of treatment • LPCD: Q3 2018 • Data readout: Q4 2018 levels) > 6.0 mg/dL) The trial is a two-centre trial conducted in the US Secondary: serum uric acid levels after 7 NCT03316131 days of treatment. Respiratory Other

76 Approved medicines Late-stage development AZD4831 (MPO inhibitor) Early development Cardiovascular disease Oncology

Trial Population Patients Design Endpoints Status AZD4831 (MPO) Healthy subjects c. 96 SAD trial (one trial site in Germany) • Safety and tolerability • FPCD: Q3 2016 • Planned to investigate 6 different dose levels vs. placebo but • PK parameters • LPCD: Q4 2016 Phase I up to 10 cohort may be used • Data readout Q2 2017

NCT02712372 CVRM

AZD4831 (MPO) Healthy subjects c. 40 MAD (one trial site in USA) • Safety and tolerability • FPCD: Q2 2017 • The planned number of cohorts is four but up to five cohorts • PK parameters • LPCD: Q4 2017 Phase I may be included • Data readout: Q1 2018

NCT03136991

AZD4831 (MPO) HFpEF 96 Arm 1: AZD4831 • Primary Endpoint: The change from • FPCD: Q4 2018 Arm 2: Placebo baseline in MPO activity in % after Phase IIa AZD4831 treatment Global trial – five countries

NCT03756285 Respiratory Other

77 Approved medicines Late-stage development AZD5718 (FLAP inhibitor) Early development Cardiovascular disease Oncology

Trial Population Patients Design Endpoints Status AZD5718 (FLAP) Healthy subjects 96 SMAD trial (one trial site in UK) • Safety and tolerability • FPCD: Q1 2016 SAD • PK parameters, bioavailability • LPCD: Q3 2016 Phase I • Oral administration • Data readout: Q4 2016 MAD NCT02632526 CVRM

AZD5718 (FLAP) Healthy subjects 12 DDI/BA trial (one trial site in UK) • PK and bioavailability • FPCD: Q2 2016 • To further assess the safety of single • LPCD: Q1 2017 Phase I A randomised, 5-period, 5-treatment, single-dose, crossover trial doses of AZD5718 in healthy subjects • Data readout Q2 2017 to estimate the effect of AZD5718 on the PK of rosuvastatin, and NCT02963116 to assess the relative BA of different formulations of AZD5718 as well as the food effect of AZD5718 AZD5718 (FLAP) Coronary Artery Disease 138 Phase IIA trial • Primary endpoint: PD effect of AZD5718 • FPCD: Q4 2017 (CAD) • Arm 1: AZD5718 Dose A by assessment of u-LTE4 Phase IIa • Arm 2: AZD 5718 Dose B • Arm 3: Placebo Respiratory NCT03317002 Global trial – three countries in Europe AZD5718 (FLAP) Healthy subjects 48 Combined SAD and MAD trial in Japanese subjects (one trial site • Safety and tolerability • FPCD: Q1 2018 in USA) • PK and PD parameters • LPCD: Q2 2018 Phase I • Data readout: Q4 2018

NCT03400488 Other AZD5718 (FLAP) Healthy subjects 14 BA trial (one trial site in UK) • PK and bioavailability • FPCD: Q1 2018 A randomised, 6-period, 6-treatment, single-dose, open-label, • Safety and tolerability • LPCD: Q2 2018 Phase I crossover trial to assess the relative bioavailability of different • Data readout: Q3 2018 formulations of AZD5718 and the food effect NCT03420092

78 Approved medicines Late-stage development AZD8233 (anti-hypercholesterolemia) Early development Hypercholesterolemia Oncology

Trial Population Patients Design Endpoints Status Phase I Healthy subjects 40 SAD Primary: • FPCD: Q3 2018 • Safety and tolerability NCT03593785 Dose escalation in 5 cohorts with 6 subjects receiving AZD8233 and 2 subjects receiving placebo in each cohort Secondary; • PK and PD parameters CVRM Trial conducted in the US. Respiratory Other

79 Approved medicines Late-stage development AZD8601 (VEGF-A modified RNA) Early development Cardiovascular disease Oncology

Trial Population Patients Design Endpoints Status Phase I Type 2 diabetic patients c. 60 SAD trial (one trial site in Germany) • Safety and tolerability • FPCD: Q1 2017 • Planned to investigate 3 different dose levels vs. placebo but • LPCD: Q3 2017 NCT02935712 up to 5 cohort may be used • Data readout: Q1 2018 CVRM

Phase IIa Heart Failure Up to 33 Phase IIa trial (two trial sites in Finland) • Safety and tolerability • FPCD: Q1 2018 • Arm 1: AZD8601 Dose A NCTT03370887 • Arm 2: AZD 8601 Dose B • Arm 3: Placebo Respiratory Other

80 Approved medicines Late-stage development AZD9977 Early development Heart failure with preserved ejection fraction Oncology

Trial Population Patients Design Endpoints Status Phase I Healthy subjects 27 MAD Primary: • FPCD: Q1 2018 • Safety and tolerability • LPCD: Q2 2018 NCT03435276 Dose escalation in 3 cohorts with 6 subjects receiving AZD9977 • Data readout: Q3 2018 and 3 subjects receiving placebo in each cohort Secondary; • PK parameters CVRM Trial conducted in the UK. Phase I Healthy subjects 12 Bioavailability trial Primary: • FPCD: Q2 2018 • relative bioavailability vs. oral • LPCD: Q2 2018 NCT03450759 Investigation of four different oral formulations of AZD9977 and suspension (reference) • Data readout: Q3 2018 influence of food. • PK parameters

Trial conducted in the UK. Phase I HFpEF 60 Proof of differentiation Primary: • FPCD Q4 2018 • serum potassium • LPCD Q1 2019

NCT03682497 To compare the effect of AZD9977 with spironolactone on serum Respiratory potassium

Phase I Healthy subjects 14 DDI Primary: • FPCD: Q1 2019 • PK parameters • LPCD: Q1 2019 NCT03843060 To assess the effect of itraconazole on the pharmacokinetics of AZD9977 Secondary; • Safety and tolerability Trial conducted in the US Other Phase I Healthy subjects 45 JSMAD Primary: • FPCD: Q1 2019 • Safety and tolerability NCT03801967 Single and multiple-ascending dose administration in Japanese healthy volunteers. Secondary; • PK parameters Trial conducted in the UK Phase I Healthy subjects 12 Bioavailability trial Primary: • FPCD: Q1 2019 • relative bioavailability vs. capsule • LPCD: Q2 2019 NCT03804645 Investigation of four different oral formulations of AZD9977 and formulation (reference) influence of food. • PK parameters

Trial conducted in the UK

81 Approved medicines Late-stage development Cotadutide (MEDI0382, GLP-1-glucagon agonist) Early development Diabetes/obesity Oncology

Trial Population Patients Design Endpoints Status

Phase I Healthy adult subjects 64 • SAD SC administration • Primary: Safety profile in terms of adverse events (AE), vital signs, • FPCD: Q1 2015 • Germany ECG, telemetry, lab variables, nausea, immunogenicity and physical • LPCD: Q4 2015 NCT02394314 examination • Data readout: Q4 2015 CVRM

Phase II Adults with type-2 113 • MAD SC administration • Safety profile in terms of adverse events (AE), vital signs, ECG, • FPCD: Q1 2016 diabetes telemetry, lab variables, nausea, immunogenicity and physical • LPCD: Q1 2017 NCT02548585 • Germany examination • Data readout: Q1 2017 Completed • Efficacy: MMT glucose AUC, HbA1c, fructosamine and body weight loss

Phase II Adults with type-2 65 • Arm1: MEDI0382 SC or placebo • Efficacy: MMT glucose AUC, body weight loss, HbA1c, fasting plasma • FPCD: Q3 2017 diabetes • Arm2: MEDI0382 SC or placebo glucose • LPCD: Q4 2017 • Germany NCT03244800 • Safety profile in terms of adverse events (AE), heart rate, blood • Data readout: Q1 2018

pressure, vital signs, ECG, lab variables Respiratory

Phase II Overweight and 834 • Arm1: MEDI0382 low dose SC + metformin • Efficacy; HbA1c, body weight loss • FPCD: Q3 2017 Obese subjects with • Arm2: MEDI0382 mid dose SC + metformin • Percentage of subjects achieving weight loss of ≥5% and ≥10% • LPCD: Q1 2018 NCT03235050 type-2 diabetes • Arm3: MEDI0382 high dose SC + metformin • Proportion of subjects rescued or discontinued for lack of glycaemic • Data anticipated H2 2019 • Arm4: placebo SC + metformin control • Arm5: liraglutide SC + metformin • PK and immunogenicity

US, Canada, Bulgaria, Czech Rep, Germany, Other Mexico, Russia, Slovakia

Phase I Adults with renal 37 • ARM1: Subjects with CrCl <20ml/min • PK, safety, tolerability and immunogenicity • FPCD: Q3 2017 impairment MEDI0382 SC • LPCD: Q1 2018 NCT03235375 • ARM2: Subjects with CrCl 20-30ml/min MEDI0382 SC • ARM3: Subjects with CrCl >90ml/min MEDI0382 SC

82 Approved medicines Late-stage development Cotadutide (MEDI0382, GLP-1-glucagon agonist) Early development Diabetes/obesity Oncology

Trial Population Patients Design Endpoints Status

Phase I Healthy adult subjects 22 • Open label, one sequence, cross-over • Effect of MEDI0382 on PK & PD of warfarin & esmolol • FPCD: Q4 2017 NCT03347968 MEDI0382 with warfarin and esmolol • Safety profile • LPCD: Q1 2018 US • Immunogenicity CVRM

Phase I Healthy adult subjects 24 • Open label, cross-over, two period • PK • FPCD: Q4 2017 NCT03341013 • Single dose MEDI0382 formulation 2 SC • Safety profile • LPCD: Q4 2017 • Single dose MEDI0382 formulation 3 SC • Immunogenicity • Data readout: Q2 2018 US

Phase I Overweight/obese 32 • Arm1: Single low dose of MEDI0382 or • Safety profile • FPCD: Q1 2018 NCT03385369 subjects of Japanese placebo (Japanese) • Tolerability • LPCD: Q2 2018 or Chinese descent • Arm2: Single intermediate-low dose of • PK MEDI0382 or placebo (Japanese) • Immunogenicity • Arm3: Single intermediate-high dose of

MEDI0382 or placebo (Japanese) Respiratory • Arm4: Single high dose of MEDI0382 or placebo (Japanese) • Arm5: Single intermediate-low dose of MEDI0382 or placebo US

Phase II Overweight/obese 46 • Arm1: MEDI0382 + dapagliflozin • Efficacy: MMT glucose AUC • FPCD: Q3 2018 NCT03444584 subjects with type-2 • Arm2: placebo + Dapagliflozin • Safety • LPCD: Q4 2018

diabetes • PK • Data readout: Q1 2019 Other • Immunogenicity

Phase II Adults with type-2 40 • MEDI0382 or placebo SC • Efficacy: MMT glucose AUC • FPCD Q2 2018 NCT03550378 diabetes and renal • Safety • LPCD; Q4 2018 impairment Germany, UK • Tolerability • Data readout: Q1 2019 • PK • Immunogenicity Phase II Adults with type-2 40 • Part A: MEDI0382 or placebo SC • Change in hepatic glycogen concentration postprandially, adjusted by • FPCD: Q2 2018 NCT03555994 diabetes • Part B: MEDI0382 SC or placebo SC or liver volume • Part A LPCD: Q4 2018 liraglutide SC • Safety • Data readout: Q1 2019 • Tolerability • Immunogenicity

83 Approved medicines Late-stage development Cotadutide (MEDI0382, GLP-1-glucagon agonist) Early development Diabetes/obesity Oncology

Trial Population Patients Design Endpoints Status

Phase II Overweight and 26 • MEDI0382 or placebo SC • Primary: Efficacy body weight loss • FPCD: Q4 2018 NCT03596177 obese subjects with • UK • Secondary: change in total energy intake type-2 diabetes • Secondary: change in total energy expenditure, active energy

expenditure, resting energy expenditure CVRM • Secondary: safety Phase I Non-diabetic obese 51 • MEDI0382 or placebo SC with 7 week, 10 • Primary: safety, tolerability • FPCD: Q3 2018 NCT03625778 subjects week or 16 week titration period • Secondary: PK • Secondary: Immunogenicity • US

Phase II Overweight and 20 • MEDI0382 or placebo SC • Primary: Safety, tolerability • FPCD: Q1 2019 NCT03745937 obese subjects with • Germany • Secondary: PK type-2 diabetes • Secondary: Immunogenicity • Secondary: Glucose control Respiratory Phase II Japanese preobese 61 • MAD SC administration • Primary: Safety, glucose AUC, body weight • FPCD: Q3 2018 NCT03645421 or obese subjects • Japan • Secondary: HbA1c, FPG, fructosamine • LPCD: Q3 2018 with type-2 diabetes • Secondary: glucose control • Secondary: PK, immunogenicity Other

84 Approved medicines Late-stage development MEDI7219 (anti-diabetic) Early development Diabetes Oncology

Trial Population Patients Design Endpoints Status

Phase I Healthy Volunteers 104 • 4 part trial • Safety and tolerability • FPCD: Q1 2018 • Part A : SAD • Pharmacokinetics • Data anticipated: H2 2019 NCT03362593 • Part B & C : open label, single dose studies

• Part D : MAD CVRM Respiratory Other

85 Approved medicines Late-stage development Biologics Early development Cardiovascular & metabolic diseases Oncology

Trial Compound Population Patients Design Endpoints Status

Phase IIb MEDI6012 Subjects 30-80 414 • Cohort A: 2-Dose Regimen Primary endpoints: Infarct size as a percentage of left ventricle • FPCD: Q2 18 rhLCAT years of age 300 mg of MEDI6012 or placebo on Day 1 (LV) mass at 10-12 weeks post-MI (myocardial infarction) • Data anticipated: 2020+ EudraCT 2017- inclusive, (loading dose) prior to pPCI followed by a compared to placebo. 004521-32 presenting with second inpatient dose Secondary endpoints: CVRM acute STEMI of 150 mg or placebo on Day 3 by i IV • Ejection Fraction at 10-12 weeks post-MI compared to placebo. push. • Change in NCPV in the coronary arteries from at10-12 • Cohort B: 6-Dose Regimen weeks post-MI compared with placebo. 300 mg of MEDI6012 or placebo on Day 1 • Myocardial mass and LV volumes at end-systole and end- prior to pPCI followed by a second diastole. inpatient dose of 150 mg or placebo on • Incidence of treatment-emergent adverse events (TEAEs) and Day 3 and outpatient maintenance doses treatment-emergent serious adverse events of 100 mg or placebo on Days 10, 17, 24, (SAEs). and 31 by IV push. • Lecithin-cholesterol acyltransferase (LCAT) mass and ADAs.

Phase IIa MEDI5884 Adults With Stable 133 • MEDI5884 (5 dose cohorts) vs. placebo • Safety profile in terms of adverse events (AE), vital signs, • FPCD Q4 2017 Cholesterol Coronary Heart in stable CHD patients ECG, lab variables • Data readout: Q4 2018 Respiratory NCT03351738 modulation Disease (CHD) • Changes in HDL-C over time • PK, immunogenicity, and Apolipoprotein B

Phase I MEDI6570 Atherosclerotic 72 • Single Ascending Dose followed by Multi • Primary Endpoints: Safety and tolerability • FPCD: Q4 2018 cardiovascular Ascending dose with 3 monthly doses in • Data anticipated: 2020 NCT03654313 disease T2DM subjects Other

86 Approved medicines Late-stage development AZD1402 (IL4 receptor antagonist) Early development Asthma Oncology

Trial Population Patients Design Endpoints Status Phase I Healthy subjects Inhaled: 56 SAD. Primary endpoint: • FPCD: Q4 2017 A dose escalating single blind trial to assess the safety, • Safety and tolerability • LPCD: Q3 2018 NCT03384290 IV: 16 tolerability and pharmacokinetics of single dose of PRS-060 • Data readout: Q1 2019 administered by oral Inhalation or IV Infusion in healthy subjects Secondary endpoint: Partnered • PK parameters CVRM • ARM 1-7 (Inhaled (nebulizer) PRS-060 and matched placebo • ARM8-9 (IV) PRS-060 and matched placebo

Australia Phase Ib Patients with mild asthma 70 PoM. Primary endpoint: • FPCD: Q3 2018 A dose-escalating, single blind trial to assess the • Safety and tolerability • LPCD Q2 2019 NCT03574805 safety, tolerability, and pharmacokinetics of multiple • Data anticipated: H2 2019 doses of PRS-060 administered by oral Inhalation In Secondary endpoint: Partnered subjects with mild asthma • PK parameters

• Potential immunogenicity Respiratory • ARM 1-4 (ARM 5 optional) (inhaled nebulizer) and matched • Change in fractional nitric oxide placebo concentration in exhaled breath (FeNO) Australia Other

87 Approved medicines Late-stage development AZD1419 (TLR9 agonist) Early development Asthma Oncology

Trial Population Patients Design Endpoints Status

Phase IIa Adults with eosinophilic, 81 • Arm 1: AZD1419, once-weekly adaptive dosing (4mg, 1mg, • Time to loss of asthma control • FPCD: Q4 2016 INCONTRO moderate to severe asthma on 8mg) • LPCD: Q4 2017 ICS + LABA background • Arm 2: placebo • Data readout: Q4 2018 NCT02898662 treatment Inhaled (nebulised) administration CVRM Trial conducted in EU Respiratory Other

88 Approved medicines Late-stage development AZD8154 (PI3Kgδ inhibitor) Early development Asthma Oncology

Trial Population Patients Design Endpoints Status Phase I Healthy subjects 54 SAD Primary endpoint: • FPCD: Q3 2018 A Phase 1 trial to assess the safety, tolerability and • Safety and tolerability NCT03436316 pharmacokinetics of AZD8154 following single dose administration in healthy subjects Secondary endpoint: • PK parameters CVRM Respiratory Other

89 Approved medicines Late-stage development AZD8871 (MABA, inhaled) Early development Respiratory Oncology

Trial Population Patients Design Endpoints Status

Phase IIa Patients with chronic 72 Randomised, double-blind, placebo and active-controlled Primary endpoint: • FPCD: Q4 2018 obstructive pulmonary disease crossover trial. Eligible patients will be randomized in 1:1:1:1:1:1 • Change from baseline in trough FEV1 on • Data anticipated: H2 2019 NCT03645434 (COPD) ratio to 1 of 6 treatment sequences and will receive 1 of the day 15

following 3 treatments sequence in the form of dry powder Secondary endpoints: CVRM • To characterize the pharmacokinetics of inhalation: AZD8871 following multiple inhaled • AZD8871 600 µg once daily doses • Anoro® Ellipta® (55 µg umeclidinium [UMEC]/ 22 µg • To assess safety and tolerability of vilanterol [VI]) once daily AZD8871 • Placebo Respiratory Other

90 Approved medicines Late-stage development AZD9567 (SGRM, oral) Early development Respiratory Oncology

Trial Population Patients Design Endpoints Status

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 125 mg as well as with 3 dose levels of • To assess the safety and tolerability of • Data readout: Q2 2018 NCT02760316 prednisolone: 5 mg, 20 mg and 40 mg AZD9567 following multiple oral

ascending doses in subjects with BMI CVRM between 28 and 38 kg/m2 and with a positive glucose tolerance test (7,8 to 11,0 mmol/L) Secondary endpoints: • To characterise the pharmacokinetics of AZD9567 following multiple oral administration of ascending doses • To characterise the pharmacodynamics of AZD9567 assessed as effect on glucose homeostasis through OGTT (oral glucose tolerance test) in comparison with prednisolone Respiratory Phase IIa Patients with active 40 A Phase II, randomised, double-blind, parallel trial to assess the Primary endpoint: • FPCD: Q1 2018 NCT03368235 Rheumatoid Arthritis (RA) efficacy, safety and tolerability of AZD9567 compared to To assess the efficacy of AZD9567, 40 mg, prednisolone 20 mg in patients with active rheumatoid arthritis compared to prednisolone 20 mg in patients with active RA in spite of stable treatment with conventional and/or sc/i.v. biological DMARDs (Disease-modifying antirheumatic drugs)

Secondary endpoints: Other • To further assess the efficacy of AZD9567, 40 mg, compared to prednisolone 20 mg in patients with active rheumatoid arthritis in spite of stable treatment with conventional and/or s.c./i.v. biological DMARDs • (e g SJC 66/TJC68, ACR response criteria) • To evaluate the pharmacokinetic profile of AZD9567

91 Approved medicines Late-stage development MEDI3506 (IL-33 mAb) ligand Early development Chronic obstructive pulmonary disease (COPD) Oncology

Trial Population Patients Design Endpoints Status

Phase I (Combined SDE / SDE: Healthy subjects with SDE: 56 SDE: • Safety and tolerability • FPCD: Q2 2017 MDE) mild atopy • 7 sequential placebo-controlled single dose cohorts (active • LPCD: H1 2019 N=6 / placebo N = 2 within each cohort) • Data anticipated: H2 2019

NCT03096795 • Dose levels: 1mg SC, 3 mg SC, 10 mg SC, 30 mg SC, 100 CVRM mg SC, 300 mg SC and 300 mg IV

J-SD: Healthy Japanese J-SD: 8 J-SD subjects • A single placebo-controlled single dose cohort (active N=6 / placebo N = 2 within cohort) • Dose level: 300 mg IV

MDE: COPD MAD: 24 MDE: • 3 sequential placebo-controlled multiple dosing cohorts

(active N=6 / placebo N = 2 within each cohort) Respiratory • Dose levels: 30 mg SC, 100 mg SC and 300 mg SC Other

92 Approved medicines Late-stage development AZD0284 (RORγ inverse agonist) Early development Plaque psoriasis vulgaris Oncology

Trial Population Patients Design Endpoints Status Phase I Healthy subjects 80 Part 1 (SAD) • Safety and tolerability and PK following • FPCD: Q3 2016 • Seven different dose levels investigated vs. placebo oral administration with single ascending • LPCD: Q2 2017 NCT02976831 • Oral administration dose • Preliminary assessment of the effect of food on the single dose PK parameters CVRM of AZD0284

Part 2 (MAD) • Safety and tolerability & PK in healthy • FPCD: Q1 2017 • Three different dose levels investigated vs. placebo in healthy subjects following administration of • LPCD: Q1 2017 subjects multiple ascending oral doses • Oral administration • Proof of Mechanism (PoM) confirmed by demonstrating that oral dosing of AZD0284 reduces IL-17 secretion by ex vivo stimulated whole blood T cells

Phase I Healthy subjects 6 A Phase I, single centre, open-label, non-randomised, single • Determination of absolute bioavailability • FPCD: Q1 2017

dose trial performed in 6 healthy male subjects aged 18 to 65 of AZD0284 • LPCD: Q1 2017 Respiratory NCT03029741 years, inclusive. The trial will assess the absolute bioavailability of • Safety and tolerability of AZD0284 a single oral dose of AZD0284 and the pharmacokinetics (PK) of a single intravenous (IV) microdose of [14C]AZD0284 in healthy male and female subjects. Oral AZD0284 and [14C] AZD0284 intravenous solution are referred to as the investigational products in this trial Phase Ib Moderate to severe plaques 25 Planned A randomised, double-blind, placebo-controlled, multi-centre, • Reduction from baseline to the end of 4 • FPCD:Q4 2017

psoriasis 5 Completed parallel group Phase 1b study, designed to evaluate the weeks treatment, in gene expression • LPCD: Q2 2018 Other NCT03310320 9 Dosed pharmacodynamic effects, clinical efficacy and safety of AZD0284 level of IL-17A and CCL20 relative to compared with placebo as measured by the relative change from placebo baseline in Psoriasis Area Severity Index (PASI score), other • Change (percent improvement) in • The trial was temporarily suspended ~5 disease assessments of involved body surface area (BSA), static Psoriasis Area and Severity Index months due to preclinical findings. physicians global assessment score (sPGA), pruritis and score(PASI) compared to placebo biomarkers associated with the mechanism of disease and • Safety and tolerability and PK following • However, whilst the intention was to re- AZD0284 4 weeks oral administration with single open the DERMIS study, in the ascending dose meantime, and for portfolio and prioritization reasons, a decision was taken in Q3 2018 to end the study.

93 Approved medicines Late-stage development AZD5634 (epithelial NaC inhibitor) Early development Cystic fibrosis Oncology

Trial Population Patients Design Endpoints Status Phase I Healthy subjects Part A: 57 SAD Primary endpoint: • FPCD: Q1 2016 Part B: 6 A Phase I, randomised, single-blind, placebo-controlled trial to • Safety and tolerability • LPCD: Q3 2016 NCT02679729 assess the safety, tolerability and pharmacokinetics of AZD5634 • Data readout: Q2 2017 following single-ascending inhaled doses (Part A) and after single Secondary endpoint: inhaled and intravenous doses (Part B) in healthy subjects • PK parameters CVRM

Phase Ib Patients with cystic fibrosis 10 PoM Primary endpoint: • FPCD: Q2 2017 A Phase Ib, randomised, blinded, placebo-controlled cross-over • Mucociliary clearance (MCC) • LPCD Q1 2018 NCT02950805 trial to assess the effect of AZD5634 on mucociliary clearance as • Data readout: Q2 2018 well as safety, tolerability and pharmacokinetic parameters Secondary endpoint: following single inhaled dose administration to patients with cystic • PK parameters fibrosis • Safety and tolerability Respiratory Other

94 Approved medicines Late-stage development Other biologics Early development Infections Oncology

Trial Compound Population Patients Design Endpoints Status Phase II Anti-Staph AT Intubated Intensive 213 • Placebo-controlled, single-dose, dose-ranging • Efficacy and safety • FPCD: Q4 2014 (suvratoxumab, Care Unit (ICU) • Route of administration: intravenous • Data readout: Q4 2018 EudraCT 2014- MEDI4893) 001097-34 CVRM Phase IIb Anti-Respiratory 29-35 WK GA 1,453 • Randomised, double-blind, placebo-controlled trial • Safety and efficacy • FPCD: Q4 2016 Syncytial Virus (Gestational age) • Route of administration: IM • Data readout: Q4 2018 NCT02878330 mAb-YTE infants (MEDI8897) Phase II Anti-Pseudomonas Intubated ICU 195 • Placebo-controlled, single-dose, dose-ranging • Efficacy and safety • FPCD: Q2 2016 A mAb (MEDI3902) • Route of administration: intravenous • Data anticipated: 2020 NCT02696902 Respiratory Other

95 Approved medicines Late-stage development MEDI0700 - AMG 570 (Anti-B7RP-1 mAb/BAFF) Early development Systemic lupus erythematosus (SLE) Oncology

Trial Population Patients Design Endpoints Status

Phase Ia Healthy volunteers 56 Single Ascending Dose • Safety and tolerability • FPCD: Q1 2016 • Arm 1: MEDI0700 administered as single SC dose • PK/PD • Data readout: Q3 2018 NCT02618967

• Arm 2: Dose levels of Placebo administered as single SC dose CVRM Partnered Respiratory Other

96 Approved medicines Late-stage development MEDI1341 (alpha-synuclein mAb) Early development Parkinson’s Disease Oncology

Trial Population Patients Design Endpoints Status Phase I Healthy volunteers 40 • SAD • Safety, tolerability, PK, PD • FPCD: Q4 2017 • Up to 5 IV cohorts are planned vs placebo • Data anticipated: H2 2019 NCT03272165 US only CVRM Respiratory Other

97 Approved medicines Late-stage development MEDI1814 (amyloid beta mAb) Early development Alzheimer’s disease Oncology

Trial Population Patients Design Endpoints Status Phase I Alzheimer’s disease & healthy 121 • SAD & MAD • Safety, tolerability • FPCD: Q2 2014 elderly • Up to 10 IV cohorts are planned vs. placebo • LPCD: Q2 2016 NCT02036645 • 2 SC cohorts are planned vs. placebo • Data readout: Q4 2016

US only CVRM Respiratory Other

98 Approved medicines Late-stage development MEDI7352 (NGF TNF bispecific mAb) Early development Osteoarthritis pain Oncology

Trial Population Patients Design Endpoints Status Phase I Painful osteoarthritis of the knee 160 • SAD & MAD • Safety, tolerability, PK, PD • FPCD: Q1 2016 • Up to 10 IV cohorts are planned vs. placebo • Data anticipated: H2 2019 NCT02508155 • 2 SC cohorts are planned vs. placebo

Europe only CVRM

Phase II Painful diabetic neuropathy 271 • Multiple dose study • Dose response, safety, tolerability, PK, • FPCD Q4 2018 NCT03755934 • Up to 4 IV cohorts are planned vs.placebo PD • Data anticipated: 2020+

Europe and Russia only Respiratory Other

99 Clinical trials appendix Q1 2019 results update