AstraZeneca : Full-year and Q4 2019 results clinical trials appendix

Clinical trials appendix FY 2019 results update

Movement since Q3 2019 update

New to Phase I	New to Phase II	New to Pivotal Study	New to Registration
NME	NME	Lifecycle Management	NME
AZD0466	AZD9833	Fasenra MANDARA	selumetinib#¶SPRINT 1
BCL2/xL haematalogical and solid tumours	selective oestrogen receptor degrader oestrogen receptor	IL5R mAb eosinophilic granulomatosis with polyangiitis	MEK inhibitor paediatric neurofibromatosis type-1
	+ve breast cancer
AZD2693			Additional indication
NASH resolution nonalcoholic steatohepatitis	cotadutide		Imfinzi#+/- tremelimumab + SoC CASPIAN 1
	GLP-1 / glucagon dual agonist nonalcoholic steatohepatitis		PD-L1 mAb +/- CTLA-4 mAb + SoC 1st-lineES-SCLC
AZD4041#
orexin 1 receptor antagonist opioid use	MEDI3506		Lifecycle Management
disorder	IL33 mAb diabetic kidney disease		Farxiga3DAPA-HF1
			SGLT2 inhibitor worsening HF or CV death in patients with chronic HF
MEDI0618	MEDI5752		(HFrEF)
PAR2 antagonist mAb osteoarthritis pain	PD-1/CTLA-4 bispecific mAb solid tumours
			Lynparza#PROFOUND 1
MEDI5395	Additional indication		PARP inhibitor prostate cancer
rNDV GMCSF solid tumours	MEDI3506
	IL33 mAb atopic dermatitis		Symbicort SYGMA1
			ICS/LABA as-needed use in mild asthma
Removed from Phase I	Removed from Phase II	Removed from Phase III	Removed from Registration
NME		NME	NME
MEDI7247		Epanova STRENGTH	Enhertu(trastuzumab deruxtecan)#¶DESTINY-Breast012
ASCT2 antibody drug conjugate		omega 3 carboxylic acids CV outcomes trial in statin	HER2 targeting antibody drug conjugate HER2-Positive, Unresectable
haematological malignancies and solid tumours		treated patients at high CV risk, with persistent	and/or Metastatic Breast Cancer Subjects Previously Treated With T-
		hypertriglyceridaemia plus low HDL cholesterol	DM1
oleclumab+AZD4635#
CD73 mAb + A2aR inhibitor EGFRm NSCLC
			Lifecycle Management
			Calquence#ASCEND2
			BTK inhibitor relapsed/refractory chronic lymphocytic leukaemia
			Calquence#ELEVATE-TN2
			BTK inhibitor 1st-line chronic lymphocytic leukaemia
			Lynparza#POLO2
			PARP inhibitor pancreatic cancer

2	¶ Registrational Phase II/III study #Partnered and/or in collaboration 1	Submission Accepted 2Submission Approved 3Farxigain the US; Forxigain ROW

Q4 2019 New Molecular Entity (NME)1pipeline

Phase I		Phase II
18 New Molecular Entities		27 New Molecular Entities

Phase III

12 New Molecular Entities

Under Review

2 New Molecular Entities

AZD0466

BCL2/xL haematological and solid tumours

AZD1390 glioblastoma

AZD4573 CDK9 haematological malignancies

AZD5153

BRD4 solid tumours, haematological malignancies

AZD5991 MCL1 haematological malignancies

AZD9496

SERD ER+ breast

Calquence+ceralasertib (AZD6738) BTK+ATR haematological tumours

Calquence+danvatirsen BTK+STAT3 haematological malignancies

Imfinzi#+adavosertib#

PD-L1+Wee1 solid tumours

Imfinzi#+RT (platform) CLOVER PD-L1+RT HNSCC NSCLC SCLC

Imfinzi#+tremelimumab		adavosertib#
PD-L1+CTLA-4 solid tumours		Wee1 ovarian cancer, solid tumours
Imfinzi#+tremelimumab+chemo		AZD2811
PD-L1+CTLA-4 1L PDAC oesophageal		Aurora solid tumours, haematological
SCLC		malignancies
Imfinzi+selumetinib#		AZD4635
PD-L1+MEK solid tumours		A2aR inhibitor prostate cancer
MEDI1191		AZD9833
IL-12 mRNA solid tumours		SERD ER+ breast
MEDI2228		capivasertib#
BCMA ADC multiple myeloma		AKT breast
MEDI5083		capivasertib#
CD40 ligand fusion protein solid
	AKT prostate
tumours
MEDI5395		Enhertu#
rNDV GMCSF solid tumours		ADC colorectal cancer
oleclumab+Tagrisso		Enhertu#
CD73+EGFR EGFRm NSCLC		ADC NSCLC

Imfinzi# (platform) COAST

PD-L1+multiple novel ONC therapies

NSCLC

Imfinzi# (platform) NeoCOAST

PD-L1++multiple novel ONC therapies

NSCLC

Imfinzi#+AZD4635

PD-L1+A2aR prostate cancer

Imfinzi#+AZD5069 or Imfinzi# + danvatirsen# PD-L1+(CXCR2 or STAT3) HNSCC bladder NSCLC

Imfinzi#+Lynparza# ORION

PD-L1+PARP 1L mNSCLC

Imfinzi#+monalizumab#

PD-L1+NKG2a solid tumours

Imfinzi#+oleclumab

PD-L1+CD73 solid tumours

Imfinzi#+tremelimumab PD-L1+CTLA-4 gastric cancer

Imfinzi#+tremelimumab PD-L1+CTLA-4 biliary tract oesophageal

Imfinzi+Lynparza# BAYOU

PD-L1+PARP bladder

Lynparza#+adavosertib#

PARP+Wee1 solid tumours

Lynparza#+AZD6738 VIOLETTE PARP+ATR breast

Lynparza#+Imfinzi MEDIOLA

PARP+PD-L1 ovarian breast gastric

SCLC

MEDI5752

PD-1/CTLA-4 solid tumours

oleclumab+AZD4635 CD73+A2aR prostate cancer

oleclumab+chemo or Imfinzi#+ oleclumab+chemo CD73+chemo or PD-L1+CD73+chemo pancreatic

Tagrissocombo# TATTON EGFR+PD-L1/MEK/MET NSCLC

Tagrisso+savolitinib# SAVANNAH EGFR+MET advanced EGFRm

NSCLC

capivasertib+chemotherapy CAPItello-290 AKT+chemotherapy mTNBC 1L

Enhertu# DESTINY-Breast 02 ADC breast

Enhertu# DESTINY-Breast 03 ADC breast

Enhertu# DESTINY-Breast 04 ADC breast

Enhertu¶# DESTINY-Gastric01 ADC gastric

Imfinzi#+/-tremelimumab+chemo

POSEIDON

PD-L1+/-CTLA-4+SoC 1L NSCLC

Imfinzi#+/-tremelimumab+CRT

ADRIATIC PD-L1+/-CTLA-4+CRTLS-SCLC

Imfinzi#+tremelimumab DANUBE PD-L1+CTLA-4 1L bladder

Imfinzi#+tremelimumab HIMALAYA PD-L1+CTLA-4 1L HCC

Imfinzi#+tremelimumab KESTREL PD-L1+CTLA-4 1L HNSCC

Imfinzi#+tremelimumab+SoC NILE PD-L1+CTLA-4+SoC 1L urothelial cancer

Lynparza#+Imfinzi#+bevacizumab

DUO-OPARP+PD-L1+VEGF 1L ovarian

Imfinzi#+/-tremelimumab+SoC

CASPIAN

PD-L1+/-CTLA-4+SoC 1L ES-SCLC

selumetinib#¶ SPRINT

MEK paediatric neurofibromatosis type-1

1includes novel combinations and additional indications for assets

3	where the lead is not yet launched	Imfinzi#+MEDI0457#
PD-L1+DNA HPV vaccine HNSCC	Oncology	Precision medicine approach under investigation
# Partnered and/or in collaboration; ¶Registrational Phase II/III study

Q4 2019 New Molecular Entity (NME)1pipeline

Phase I		Phase II
16 New Molecular Entities		22 New Molecular Entities

Phase III

5 New Molecular Entities

Under Review

0 New Molecular Entities

AZD0284		AZD8233		abediterol#
RORg psoriasis / respiratory		hypercholesterolemia cardiovascular		LABA asthma / COPD
AZD4041#		AZD9977		anifrolumab#
orexin 1 receptor antagonist opinoid
	MCR cardiovascular		Type I IFN receptor SLE SC
use disorder
AZD0449		MEDI0618		anifrolumab#
	PAR2 antagonist mAb osteoarthritis
Inhaled JAK inhibitor asthma			Type I IFN receptor lupus nephritis
	pain
AZD1402#		MEDI1341#		AZD4831
inhaled IL4Ra asthma		alpha synuclein parkinson's disease		MPO HFpEF
AZD2693		MEDI1814#		AZD5718
nonalcoholic steatohepatitis		amyloidβ alzheimer's disease		FLAP coronary artery disease
AZD5634		MEDI5117# China		AZD7594
inhaled ENaC cystic fibrosis		IL6 YTE rheumatoid arthritis		Inhaled SGRM asthma / COPD
AZD6615		MEDI6570		AZD7986#
hypercholesterolemia CV disease		LOX-1 CV disease		DPP1 COPD
AZD8154		MEDI7219		AZD8601#
Inhaled PI3Kgd asthma		anti-diabetictype-2 diabetes		VEGF-A cardiovascular
				AZD8871#
				MABA COPD
				AZD9567
				SGRM RA / respiratory
				cotadutide
				GLP-1/glucagontype-2 diabetes /
				obesity / NASH

4	1includes novel combinations and additional indications for assets where the lead is not yet launched
# Partnered and/or in collaboration; ¶Registrational Phase II/III study

MEDI3506

Diabetic kidney disease

MEDI3506

IL33 AD / COPD

MEDI3902

Psl/PcrV Pseudomonas pneumonia

MEDI5884# cholesterol modulation cardiovascular

MEDI6012

LCAT cardiovascular

MEDI7352

NGF/TNF OA pain / painful diabetic neuropathy

roxadustat#

HIF-PH inhibitor chemo induced anaemia

suvratoxumab α-Toxin Staphylococcus pneumonia

tezepelumab#

TSLP atopic dermatitis

tezepelumab#

TSLP COPD

verinurad

URAT-1 chronic kidney disease

BioPharmaceuticals

anifrolumab# TULIP Type I IFN receptor SLE

nirsevimab (MEDI8897)# RSV mAb-YTE passive RSV immunisation

PT027

ICS/SABA asthma

roxadustat#

HIFPH anaemia MDS

tezepelumab#

NAVIGATOR SOURCE

TSLP severe uncontrolled asthma

Precision medicine approach under investigation

Q4 2019 Lifecycle Management (LCM)1pipeline

Phase I		Phase II
1 Project		6 Projects

Phase III

20 Projects

Under Review

1 Project

Imfinzi#+azacitidine#		Imfinzi#
PD-L1+azacitidine MDS		PD-L1 solid tumours

Imfinzi# (platform) BEGONIA

PD-L1 1L mTNBC

Imfinzi# (platform) MAGELLAN

PD-L1 1L mNSCLC

Imfinzi+FOLFOX+bevacizumab (platform) COLUMBIA1 PD-L1+chemo+VEGF+multiple novel ONC therapies 1L MSS-CRC

Lynparza# (basket) MK-7339-002 /

LYNK002

PARP HRRm cancer

Lynparza#+cediranib CONCERTO

PARP+VEGF recurrent Pt-R ovarian

Calquence#

BTK inhibitor 1st line MCL

Calquence#

BTK inhibitor r/r CLL, high risk

Calquence# + venetoclax + obinutuzumab BTK+BCL-2+anti-CD20 1st line CLL

Imfinzi# CALLA

PD-L1 adj. locally advanced cervical cancer

Imfinzi# PEARL

PD-L1 1L metastatic NSCLC

Imfinzi# post-SBRTPACIFIC-4PD-L1post-SBRT stage I/II NSCLC

Imfinzi# POTOMAC PD-L1 non muscle invasive bladder cancer

Imfinzi#+CRT PACIFIC-2

PD-L1+CRT NSCLC

Imfinzi#+CRT PACIFIC-5 (China) PD-L1+CRTlocally-advanced stage III NSCLC

Imfinzi#+CTx neoadjuvant AEGEANPD-L1+CTxlocally-advanced stage I-III NSCLC

Imfinzi#+CTx NIAGARA PD-L1+CTx muscle invasive bladder cancer

Imfinzi#+CTx TOPAZ-1PD-L1+CTx 1L biliary tract cancer

Imfinzi#+VEGF EMERALD-2

PD-L1+VEGF adjuvant HCC

Imfinzi#+VEGF+TACE EMERALD-1PD-L1+VEGF+TACE locoregional

HCC

Lynparza# OlympiA

PARP gBRCA adjuvant breast

Lynparza# SOLO-3

PARP BRCAm PSR ovarian

Lynparza+abiraterone# PROpel PARP+NHA prostate cancer

Tagrisso ADAURA

EGFR adj. EGFRm NSCLC

Tagrisso LAURA

EGFRm locally advanced unresectable

NSCLC

Tagrisso+chemo FLAURA2 EGFR+chemo 1L adv EGFRm NSCLC

Lynparza# PROfound PARP prostate cancer

5	1Includes significant LCM projects and parallel indications for assets beyond Phase III	Oncology	Precision medicine approach under investigation
# Partnered and/or in collaboration; ¶Registrational Phase II/III study

Q4 2019 Lifecycle Management (LCM)1pipeline

Phase	I	Phase II		Phase III		Under Review
0 Projects		1 Project		9 Projects		4 Projects
		Breztri		Brilinta/Brilique HESTIA		Brilinta/Brilique THEMIS
		LABA/LAMA/ICS asthma		P2Y12 paeds w/ sickle cell		P2Y12 diabetes & CAD outcomes
				Brilinta/Brilique THALES		Farxiga/Forxiga DAPA-HF
				P2Y12 stroke		SGLT2 HFrEF
				Farxiga/Forxiga Dapa-CKD		Nexium(CN only)
				SGLT2 CKD		stress ulcer prophylaxis
				Farxiga/Forxiga DELIVER		Symbicort SYGMA
				SGLT2 HFpEF		as needed in mild asthma
				Farxiga/Forxiga DETERMINE-
				Preserved
				Farxiga/Forxiga DETERMINE-
				Reduced
				Fasenra MANDARA
				IL5R EGPA
				Fasenra# OSTRO, ORCHID
				IL5R nasal polyposis
				Fasenra# RESOLUTE
				IL5R COPD

1Includes significant LCM projects and parallel indications for assets beyond Phase III

6

# Partnered and/or in collaboration; ¶Registrational Phase II/III study

BioPharmaceuticals

Precision medicine approach under investigation

Estimated key regulatory submission acceptances

NME

LCM

				PT027 asthma
				tezepelumab asthma
				NAVIGATOR
Enhertu				Enhertu		Fasenrasevere asthma (China)
gastric cancer (Japan)				DESTINY-Breast02
Imfinzi+ tremelimumab bladder		anifrolumab SLE		Imfinzi+ tremelimumab HCC		capivasertb + CTx 1L mTNBC		Imfinzi+ tremelimumab + CRT LDS-SCLC
DANUBE		TULIP		HIMALAYA		CAPItello-290		ADRIATIC
Imfinzi+ tremelimumab HNSCC		Enhertu		Imfinzi+/- tremelimumab NSCLC		Enhertu		Imfinzi+ tremelimumab+ SoC urothelial
KESTREL		DESTINY-Breast01 (EU)		POSEIDON		DESTINY-Breast03		NILE
selumetinib NF1		Imfinzi+/- tremelimumab SCLC		Lynparza+ cediranib ovarian		Enhertu		Lynparza+Imfinzi+ bevacizumab ovarian
SPRINT (EU)		CASPIAN (China)		GY004		DESTINY-Breast04		DUO-O
H1 2020		H2 2020		2021		2021+
Brilinta stroke		Lynparza ovarian		Brilintapaeds w/ sickle cell		Calquence 1L MCL		TagrissoEGFrm NSCLC
THALES		SOLO-3		HESTIA		ECHO		ADAURA
Symbicortmild astha (EU)				Fasenranasal polyposis		Calquencer/r CLL, high risk		Tagrissolocally adv. unresectable NSCLC
SYGMA				OSTRO		ELEVATE-RR		LAURA
				Farxiga CKD		Calquence+venetoclax+obinutuzumab		Tagrisso +CTx EGFRm NSCLC
				DAPA-CKD		1L CLL		FLAURA2
				FarxigaHFrEF / HFpEF		Imfinzi cervical		Bydureon Bcisetype-2 diabetes (China)
				DETERMINE		CALLA
				Imfinziadjuvant NSCLC		Imfinzi +CTx biliary tract		DuaklirGenuair COPD (China)
				BR.31		TOPAZ-1
				Imfinzineoadjuvant NSCLC		Imfinzinon muscle invasive bladder		Farxiga HFpEF
				AEGEAN		POTOMAC		DELIVER
				Imfinzi NSCLC		Imfinzi+ chemo muscle invasive bladder		Fasenra COPD
				PEARL		NIAGARA		RESOLUTE
				Imfinzi+ CRT NSCLC		Imfinzipost-SBRT NSCLC		nirsevimab passive RSV immunisation
				PACIFIC-2		PACIFIC-4
				Imfinzi + VEGF + TACE locoregional HCC		Imfinzi+ CRT NSCLC		roxadustat anemia in MDS
				EMERALD-1		PACIFIC-5 (China)
				Lynparza breast		Imfinzi+ VEGF adjuvant HCC		Fasenranasal polyposis
				OLYMPIA		EMERALD-2		ORCHID (China / Japan)
				Lynparza+ abiraterone prostate				Fasenra EGPA
				PROPEL				MANDARA

7

Note. NME section includes novel combinations and additional indications for assets where the lead is not yet launched

Oncology

BioPharmaceuticals

Designations

4

Accelerated approvals

Lynparzaovarian cancer SOLO-2 (US)

TagrissoEGFRm T790M NSCLC (US)

Imfinzibladder cancer (US)

Calquence MCL (US)

13	11
Breakthrough / PRIME1/ Sakigake2	Fast Track
TagrissoEGFRm T790M NSCLC (US)	MEDI3902 Psl-PcrV pneumo Px (US)
Lynparzaprostate cancer PROFOUND (US)	savratoxumab Staph HAP (US)
Imfinzibladder cancer 1L (US)	ImfinziNSCLC (US)
Calquence MCL (US)	nirsevimab (MEDI8897) RSV mAB (US)
Imfinzistage III NSCLC 1L PACIFIC (US)	ImfinziHNSCC HAWK (US)
TagrissoNSCLC 1L FLAURA (US)	anifrolumab SLE (US)
tezepelumab asthma (US)	Lynparzaovarian cancer SOLO-2 (US)
nirsevimab (MEDI8897) RSV mAB (US)	TagrissoEGFRm T790M NSCLC (CN)
nirsevimab (MEDI8897) RSV mAB (EU)1	FarxigaHFrEF (US)
selumetinib NFI type 1 SPRINT (US)	Farxigachronic kidney disease (US)
EnhertuDESINTY-BREAST01 (US)	cotadutide non-alcoholic steatohepatitis (US)
Calquence CLL (US)
Enhertugastric cancer (JP)2

29

Priority Review / RTOR3

TagrissoEGFRm T790M NSCLC (JP) TagrissoEGFRm T790M NSCLC (US) Imfinzibladder cancer 2L (US)

TagrissoNSCLC AURA3 (US)

Calquence MCL (US)

Lynparzabreast cancer OLYMPIAD (US) roxadustat CKD (CN)

TagrissoNSCLC FLAURA (US) Imfinzistage III NSCLC PACIFIC (EU) Imfinzistage III NSCLC PACIFIC (JP) Lynparzatablet (US)

Lynparzatablet (CN)

Lynparzabreast cancer OLYMPIAD (JP) TagrissoNSCLC 1L FLAURA (JP) LumoxitiHCL PLAIT (US)

Lynparzaovarian SOLO-1 (US)

Lynparzaovarian SOLO-1 (CN)

26

Orphan Drug

Lynparzaovarian cancer SOLO-2 (US) LumoxitiHCL PLAIT (US)

LumoxitiHCL PLAIT (EU) Crestorpaediatric (US) cediranib VEGFR tki (US) IressaEGFRm NSCLC (US)

TagrissoEGFRm T790M NSCLC (US)

AZD3241 MPO (EU)

CalquenceCLL 1L (US)

Calquence MCL (US)

Calquence WM (US)

Calquence WM (EU)

CalquenceCLL 1L (EU)

Calquence MCL (EU)

selumetinib thyroid cancer ASTRA (US) Lynparzabreast cancer OLYMPIAD (JP) Lynparzaovarian cancer SOLO-2 (JP)

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. 3REAL-TIME ONCOLOGY REVIEW (RTOR) and Project Orbis is an initiative of the FDA Oncology Centre of Excellence (OCE) providing a framework for concurrent submission and review of oncology products among international partners.

BREAKTHROUGH DESIGNATION is a process designed to expedite the development and review of medicines which may demonstrate substantial improvement over available therapy. 1PRIME is a scheme launched by the EMA to enhance support for the development of medicines that target an unmet medical need. 2SAKIGAKE is aimed at early introduction of innovative medicines, medical devices, etc. that are initially developed in Japan

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.

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.

Breztri Aerosphere (PT010) COPD (CN) TagrissoNSCLC 1L FLAURA (CN)Breztri Aerosphere(PT010) (CN) Lokelmahyperkalaemia (CN) Lynparzapancreatic 1L (US)

EnhertuDESINTY-BREAST01 (US)

FarxigaHF (DAPA-HF) (US)

Imfinzi+/-treme+SOC SCLC 1L (CASPIAN) (US) Lynparzaprostate (PROfound) (US)

Lynparza+Avastin ovarian 1L (PAOLA-1) (US) selumetinib NFI type 1 SPRINT (US) CalquenceCLL (ELEVANTE-TN, ASCEND)3

selumetinib NFI type 1 SPRINT (US) selumetinib NFI type 1 SPRINT (EU) Lynparzapancreatic cancer POLO (US) FasenraEGPA (US)

FasenraHES (US) saracatinib IPF (US)

Imfinzi+/-treme+SOC SCLC 1L CASPIAN (US) FasenraEoE (US)

Imfinzi+treme HCC 1L (HIMALAYA)

8

Oncology

BioPharmaceuticals

Oncology - approved medicines and late-stage pipeline

									Approved medicines
	Tagrisso	(highly-selective, irreversible EGFRi)		Late-stage development
		Early development
NSCLC
	Trial	Population	Patients	Design	Endpoints	Status
	Phase III	Adjuvant EGFRm NSCLC	682	•	Arm 1: TagrissoQD following complete tumour resection, with	•	Primary endpoint: DFS	•	FPCD: Q4 2015
	ADAURA				or without chemo	•	Secondary endpoints: DFS Rate, OS, OS	•	LPCD: Q1 2019
	NCT02511106			•	Arm 2: placebo		Rate, QoL	•	Data anticipated: 2022
			Global trial - 25 countries
	Phase III	Maintenance therapy in	200	•	Arm 1:Tagrisso	•	Primary endpoint: PFS (BICR)	•	FPCD: Q3 2018
	LAURA	patients with		•	Arm 2: placebo	•	Secondary endpoints: CNS PFS, OS,	•	Data anticipated: 2021+
	NCT03521154	locally advanced,		Global trial - 11 countries		DoR, ORR, DCR
	unresectable EGFRm Stage
		III NSCLC whose disease has
		not progressed following
		platinum-based
		chemoradiation therapy
	Phase III	Real world setting in adult	3,020	Single-arm trial - Tagrisso	•	Primary endpoints: OS and safety	•	FPCD: Q3 2015
	ASTRIS	patients with advanced or				•	Secondary endpoint: PFS	•	LPCD: Q4 2017
		metastatic, EGFRm T790M+		Global trial - 16 countries
	NCT02474355	NSCLC
	Phase II	EGFR TKI treatment-naïve	150	Single arm trial - Tagrisso	•	Primary Endpoint: proportion of patients	•	FPCD: Q2 2018
	ELIOS	patients with locally-advanced					with a given tumour genetic and
		or metastatic EGFRm NSCLC		Global trial - five countries		proteomic marker at the point of disease
	NCT03239340						progression as defined by the
							investigator
						•	Secondary endpoint: PFS, ORR, DoR
	Phase I	Patients with EGFRm NSCLC	8	Single-arm trial - Tagrisso	•	Primary Endpoints: assessments of brain	•	FPCD: Q4 2018
	ODIN-BM	with brain metastases					standard uptake value (SUV) and
							pharmacokinetics (PK)
	NCT03463525					•	Secondary endpoints: PK

Oncology

CVRM

Respiratory

Other

10

									Approved medicines
	Tagrisso(highly-selective, irreversible EGFRi)		Late-stage development
		Early development
NSCLC, combinations
	Trial	Population	Patients	Design	Endpoints	Status
	Phase III	1st-line EGFRm NSCLC	586	Arm 1: Tagrissoplus Pemetrexed/Carboplatin or	•	Primary endpoint: PFS	•	FPCD: Q4 2019
	FLAURA2				Pemetrexed/Cisplatin	•	Secondary endpoints: OS, LOS, ORR	•	Data anticipated: 2021+
	NCT04035486			Arm 2:Tagrisso		DoR, Depth of response, PFS2. QoL, PK
			Global trial - 5+ countries
	Phase II	Advanced EGFRm NSCLC	150	Modular design platform study:	•	Primary endpoint: ORR	•	FPCD: Q3 2019
	ORCHARD	patients who have progressed		•	Module 1: Tagrisso+ savolitinib	•	Secondary endpoints: PFS, DoR, OS,	•	Data anticipated: 2021+
		on first line Tagrissotreatment		•	Module 2: Tagrisso+ gefitinib		safety and tolerability
	NCT03944772			•	Module 3: Tagrisso+ necitumumab
				•	Module 4:carboplatin + pemetrexed + Imfinzi
				•	No intervention: observational cohort - no study drug
				Global trial - 8 countries
	Phase II	EGFRm / MET+, locally	172	•	Single arm trial: Tagrisso+ savolitinib	•	Primary endpoint: ORR	•	FPCD Q1 2019
	SAVANNAH	advanced or metastatic				•	Secondary endpoints include PFS, DoR	•	Data anticipated: 2021+
		NSCLC who have progressed		Global trial		and OS
	NCT03778229	following treatment with
		Tagrisso
	Phase Ib	Advanced EGFRm NSCLC	344	•	Arm 1:Tagrisso +Imfinzi	•	Safety, tolerability, pharmacokinetics and	•	FPCD: Q3 2014
	TATTON	TKI failure		•	Arm 2: Tagrisso+ savolitinib		preliminary anti-tumour activity	•	Data anticipated: 2020
				•	Arm 3: Tagrisso+ selumetinib
	NCT02143466			Enrolment to Imfinzicombination arms will not restart
				Global trial

Oncology

CVRM

Respiratory

Other

11

								Approved medicines
Imfinzi(PD-L1 mAb)					Late-stage development
				Early development
NSCLC, early disease
	Trial	Population	Patients	Design	Endpoints	Status
	Phase III	Neoadjuvant NSCLC patients	800 / 300	•	Arm 1: Imfinzi+ platinum-based chemo	Primary endpoint:	•	FPCD: Q1 2019
		Stage II and III resected		•	Arm 2: placebo + platinum-based chemo	•	mPR	•	Data anticipated: H2 2020
	AEGEAN	NSCLC				Secondary endpoint
	NCT03800134	(incl. EGFR/ALK positive)				•	pCR
	Phase III	Adjuvant NSCLC patients	1,360	•	Arm 1: Imfinzimg/kg i.v. Q4W x 12m	Primary endpoint:	•	FPCD: Q1 2015
	ADJUVANT BR.31	Ib (≥4cm) - stage IIIa resected		•	Arm 2: placebo	•	DFS	•	Data anticipated: 2021
		NSCLC
	NCT02273375	(incl. EGFR/ALK positive)		Global trial	Secondary endpoint:
	Partnered					•	OS
	Phase III	Unresected, locally-advanced	300	•	Arm 1: Imfinzii.v. Q4W + chemo/RT	Primary endpoint:	•	FPCD: Q2 2018
		NSCLC		•	Arm 2: placebo + chemo/RT	•	PFS	•	LPCD: Q3 2019
	PACIFIC-2					•	ORR	•	Data anticipated: H2 2020
				ex US global trial	Secondary endpoint:
	NCT03519971					•	OS
	Phase III	Imfinzifollowing SBRT in	630	•	Arm 1: Imfinzii.v. Q4W following definitive SBRT	Primary endpoint:	•FPCD: Q2 2019
		unresected, Stage I/II NSCLC		•	Arm 2: placebo following definitive SBRT	•	PFS	•Data anticipated: 2021+
	PACIFIC-4					Secondary endpoint:
						•	OS
	NCT03833154
	Phase III	Unresected, locally-advanced	360	•	Arm 1: Imfinzii.v. Q4W following chemo/RT	Primary endpoint:	•	FPCD: Q1 2019
		NSCLC		•	Arm 2: placebo following chemo/RT	•	PFS	•	Data anticipated: 2021+
	PACIFIC-5					Secondary endpoint:
	NCT03706690			ex US global trial, China focus	•	OS
	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: 2021+
					substudies) i.v. Q2W single arm ImfinziPhase II only	• PFS
	NCT02154490	Biomarker-targeted		•	Substudy B: PI3K inhibitor vs. docetaxel	• OS
	Partnered	2L therapy		•	Substudy C: CDK4/6 inhibitor vs, docetaxel
			•	Substudy D: AZD4547 (FGFR inhibitor) vs. docetaxel
				•	Substudy E: C-MET/HGFR Inhibitor + erlotinib vs. erlotinib

Oncology

CVRM

Respiratory

Other

12

								Approved medicines
Imfinzi(PD-L1 mAb) +/- treme (CTLA-4 mAb)	Late-stage development
Early development
Lung cancer, advanced disease
	Trial	Population	Patients	Design	Endpoints	Status
	Phase III	NSCLC 1L	650	•	Arm 1: ImfinziQ4W	Primary endpoint:	•	FPCD: Q1 2017
	PEARL			•	Arm 2: chemotherapy	•	OS	•	LPCD: Q1 2019
	NCT03003962			Asia trial			•	Data anticipated: 2021
	Phase III	NSCLC 1L	1,000	•	Arm 1: Imfinzi+ chemo	Primary endpoint:	•	FPCD: Q2 2017
	POSEIDON			•	Arm 2: Imfinzi+ tremelimumab + chemo	•	OS	•	LPCD: Q4 2018
				•	Arm 3: SoC	•	PFS	•	Data readout: Q4 2019
	NCT03164616							•	PFS primary endpoint met
								•	OS Data anticipated: 2021
	Phase II	NSCLC 1L	200	•	Arm A1: Imfinzi	Primary endpoint:	•	FPCD: Q1 2019
	MAGELLAN			•	Arm A2: Imfinzi+ danvatirsen	•	Safety & tolerability	•	Data anticipated: H2 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
	Phase III	Limited disease- SCLC 1L	600	•	Arm 1: Imfinzi+ tremelimumab (4 doses)	Primary endpoints:	•	FPCD: Q4 2018
	ADRIATIC	following platinum-based		•	Arm 2: Imfinzi	•	PFS	•	Data anticipated: 2021
		concurrent chemoradiation		•	Arm 3: placebo	•	OS
	NCT03703297	therapy
	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 readout: Q2 2019
	NCT03043872			•	Arm 3: EP (carboplatin or cisplatin + etoposide)			•	OS Primary endpoint met for Imfinzi
									monotherapy arm
	Phase II	SCLC	80	•	Arm A: Imfinzi+ tremelimumab Q4W	•	Primary endpoint: ORR	•	FPCD: Q4 2016
	BALTIC			•	Arm B: adavosertib and carboplatin BID			•	Data anticipated: 2021
	NCT02937818			•	Arm C: AZD6738 and Lynparza

Oncology

CVRM

Respiratory

Other

13

								Approved medicines
Imfinzi(PD-L1 mAb)					Late-stage development
				Early development
Other cancers, early disease
	Trial	Population	Patients	Design	Endpoints	Status
	Phase III	Non-muscle invasive bladder	975	•	Arm 1: BCG (Induction + maintenance)	Primary endpoints:	•	FPCD: Q4 2018
	POTOMAC	cancer		•	Arm 2: Imfinzi+ BCG (Induction only)	•	DFS	•	Data anticipated: 2021+
	NCT03528694			•	Arm 3: Imfinzi+ BCG (Induction + maintenance)
	Phase III	Muscle-invasive bladder	960	•	Arm 1: Imfinziin combination with gemcitabine + cisplatin,	Coprimary endpoints:	•	FPCD: Q4 2018
	NIAGARA	cancer			Imfinzimaintenance	•	pCR	•	Data anticipated: 2021+
	NCT03732677			•	Arm 2: gemcitabine + cisplatin	•	EFS
	Phase III	Locoregional HCC	600	•	Arm A: TACE in combination with Imfinzi	Primary endpoint	•	FPCD: Q1 2019
	EMERALD-1			•	Arm B: TACE in combination with Imfinzi+ bevacizumab	PFS for Arm A vs Arm C	•	Data anticipated: 2021
	NCT03778957			•	Arm C: TACE in combination with placebo	Secondary endpoint
						PFS for Arm B vs Arm C , OS
	Phase III	Adjuvant therapy in HCC	888	•	Arm 1: Imfinzi+ bevacizumab	Primary endpoint:	•	FPCD: Q2 2019
	EMERALD-2			•	Arm 2: Imfinzi+ placebo	• RFS for Arm 2 vs Arm 3	•	Data anticipated: 2021+
	NCT03847428			• Arm 3: placebo + placebo	Secondary endpoint:
						•	RFS Arm 1 vs Arm 3, OS, RFS at 24
							mos

pCR = Pathologic Complete Response EFS = event free survival

Oncology

CVRM

Respiratory

Other

14

								Approved medicines
Imfinzi(PD-L1 mAb) +/- treme (CTLA-4 mAb)	Late-stage development
Early development
Other cancers, advanced disease
	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
	NCT02516241			•	Arm 3: SoC			•	Data anticipated: H1 2020
	Phase III	Bladder cancer 1L	885	•	Arm 1: Imfinzi+ tremelimumab + SoC	Primary endpoints:	•	FPCD: Q4 2018
	NILE			•	Arm 2: Imfinzi+ SoC	•	PFS	•	Data anticipated: 2021
	NCT03682068			•	Arm 3: SoC	•	OS
	Phase III	HNSCC 1L	823	•	Arm 1: Imfinzi	Primary endpoints:	•	FPCD: Q4 2015
	KESTREL			•	Arm 2: Imfinzi+ tremelimumab	•	OS	•	LPCD Q1 2017
	NCT02551159			•	Arm 3: SoC			•	Data anticipated: H1 2020
	Phase III	HCC 1L	1,310	•	Arm 1: Imfinzi+ tremelimumab	Primary endpoint:	•	FPCD: Q4 2017
	HIMALAYA			•	Arm 2: Imfinzi	•	OS	•	LPCD: Q4 2019
				•	Arm 3: sorafenib	Secondary endpoint:	•	Data anticipated: H2 2020
	NCT03298451					•	PFS, 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
	Phase III	BTC 1L	474	•	Treatment Arm 1 Imfinzi+ gemcitabine + cisplatin	Primary endpoint:	•	FPCD Q2 2019
	TOPAZ-1			•	Treatment Arm 2 placebo + gemcitabine + cisplatin	•	OS	•	Data anticipated: 2021+
	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: 2021+
						Secondary
	NCT03830866			Global trial	•	OS, CR rate, DoR, ORR,
							safety/tolerability

Oncology

CVRM

Respiratory

Other

15

								Approved medicines
Imfinzi(PD-L1 mAb) +/- treme (CTLA-4 mAb)	Late-stage development
Early development
Other cancers
	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: 2021+
	NCT03084471
	Phase I Combination in	Solid tumours	80	•	Arm 2 SCLC: Imfinzi+ tremelimumab + carboplatin + etoposide	•	Safety	•	FPCD: Q1 2016
	Advanced Solid Tumours			•	Arm 3 TNBC: Imfinzi+ tremelimumab + chemo			•	LPCD: Q1 2019
				•	Arm 4 TNBC: Imfinzi+ tremelimumab + chemo			•	Data anticipated: 2021+
	NCT02658214			•	Arm 5 GEJ: Imfinzi+ tremelimumab + oxaliplatin + 5-FU +
					leucovorin
				•	Arm 6 PDAC: Imfinzi+ tremelimumab + chemo
				•	Arm 7 ESSC: Imfinzi+ tremelimumab + chemo
	Phase I Immunotherapy in	HNSCC, NSCLC, SCLC	300	•	HNSCC Arm 1	•	Safety	•	FPCD: Q2 2018
	Combination With			•	NSCLC Arm 1			•	Data anticipated: 2021+
	Chemoradiation in Patients			•	NSCLC Arm 2
	With Advanced Solid			•	NSCLC Arm 3
	Tumours			•	SCLC Arm 2
				•	SCLC Arm 3
	CLOVER			•	SCLC Arm 4
	NCT03509012
	Phase II	mTNBC 1L	110	•	Arm 1 Imfinzi+ paclitaxel	Primary endpoint:	•	FPCD: Q1 2019
	BEGONIA			•	Arm 2 Imfinzi+ paclitaxel + capivasertib	•	Safety and tolerability	•	Data anticipated: H2 2020
				• Arm 4 Imfinzi+ paclitaxel + danvatirsen
	NCT03742102			•	Arm 5 Imfinzi+ paclitaxel + oleclumab	Secondary endpoint:
				Global trial	• ORR, PFS, DoR, OS, PK, ADA

Oncology

CVRM

Respiratory

Other

16

								Approved medicines
Lynparza(PARP inhibitor)			Late-stage development
		Early development
Ovarian and other cancers
	Trial	Population	Patients	Design	Endpoints	Status
	Phase III	BRCAm adjuvant breast	1,836	•	Arm 1: LynparzaBiD 12 month duration	•	Primary endpoint: invasive disease-free	•	FPCD: Q2 2014
	OlympiA	cancer		•	Arm 2: placebo 12-month duration		survival (IDFS)	•	LPCD: Q2 2019
						•	Secondary endpoint: distant disease-free	•	Data anticipated: 2021
	NCT02032823			Global trial partnership with BIG and NCI/NRG		survival and OS
	Partnered
	Phase III	gBRCAm pancreatic cancer	154	•	Arm 1: Lynparzatablets 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			Global trial			•	Primary endpoint met
	Phase III	Metastatic castration-resistant	387	•	Arm 1: LynparzaBID	•	Primary endpoint: radiologic PFS	•	FPCD: Q2 2017
	PROfound	prostate cancer		•	Arm 2: physician's choice:	•	Secondary endpoints: ORR, Time to Pain	•	LPCD: Q4 2018
		HRRm, 2L+			enzalutamide 160mg once daily or abiraterone acetate		Progression, OS	•	Data readout : Q3 2019
	NCT02987543				1,000mg once daily			•	Primary endpoint met
				Global trial

Oncology

CVRM

Respiratory

Other

17

								Approved medicines
Lynparza(PARP inhibitor)			Late-stage development
		Early development
Imfinzicombinations
	Trial	Population	Patients	Design	Endpoints	Status
	Phase III	Advanced ovarian cancer 1L	1,056	Non tBRCAm (tumour BRCA) patients	Primary endpoint:	•	FPCD: Q1 2019
	DuO-O			•	Arm 1: bevacizumab	•	PFS	•	Data anticipated: 2021+
				• Arm 2: bevacizumab + Imfinzi
	NCT03737643			•	Arm 3:bevacizumab + Imfinzi+ Lynparza	Secondary endpoints:
				tBRCAm patients	•	OS, PFS2
				•	bevacizumab (optional) + Imfinzi+ Lynparza
				Global trial
	Phase II	Stage IV NSCLC whose disease has	250	•	Arm 1:Imfinzi +Lynparza	Primary endpoint:	•	FPCD Q1 2019
	ORION	not progressed following SoC chemo		•	Arm 2: Imfinzi+ placebo	•	PFS	•	Data anticipated: 2021+
	NCT03775486	+ ImfinziMaintenance therapy 1L		Global trial	Secondary enpoints:
						•	OS, ORR, DoR, PFS in HRRm, PK, ADA
	Phase II	Platinum-Ineligible unresectable	154	•	Arm 1:Imfinzi +Lynparza	•	Primary endpoint: PFS	•	FPCD: Q1 2018
	BAYOU	Stage IV urothelial cancer		•	Arm 2: Imfinzi+ placebo	•	Secondary endpoints: OS, DoR, ORR,	•	Data anticipated : H1 2020
	NCT03459846			Global trial		PFS in HRRm, PFS6, PK, ADA, PRO
	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
	NCT02734004	cancer 1-3L		Global trial	•	Safety and tolerability
	SCLC 2L+
		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
	NCT02734004			•	Dose until progression	•	Safety and tolerability
			Global trial

Oncology

CVRM

Respiratory

Other

18

								Approved medicines
Lynparza(PARP inhibitor)			Late-stage development
		Early development
Other combinations
	Trial	Population	Patients	Design	Endpoints	Status
	Phase III	Advanced ovarian cancer 1L	806	•	Arm 1: Lynparzamaintenance 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 readout: Q3 2019
	NCT02477644					Secondary endpoints:	•	Primary endpoint met
	Externally sponsored			Global trial	•	OS, PFS2
	Phase III	Metastatic castration-resistant	720	•	Arm 1: Lynparza+ abiraterone	Primary Endpoint:	•	FPCD: Q4 2018
	PROpel	prostate cancer 1L		• Arm 2: placebo + abiraterone	•	rPFS	•	Data anticipated: 2021
	NCT03732820			Global trial	Secondary endpoints:
						•	TFST, TTPP, OS
	Phase II	TNBC	450	• Arm 1: AZD6738 + Lynparza	•	PFS	•	FPCD: Q2 2018
				•	Arm 2:Lynparza	•	ORR / OS	•	Data anticipated: 2021
	VIOLETTE			Trial conducted in 15 countries: North America, Europe and Asia	•	Safety and tolerability
	NCT03330847
	Phase III	Recurrent platinum sensitive	549	•	Arm 1: chemo	Primary endpoint:	•	FPCD: Q1 2016
	GY004	ovarian cancer		•	Arm 2:Lynparza	•	PFS	•	Data anticipated: H1 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: chemo	Primary endpoints:	•	FPCD: Q2 2016
	GY005	resistant/refractory ovarian		• Arm 2: cediranib + Lynparza	•	PFS, OS	•	Data anticipated: 2021+
		cancer		•	Arm 3: cediranib
	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

Oncology

CVRM

Respiratory

Other

19

								Approved medicines
Calquence(BTK inhibitor)			Late-stage development
		Early development
Blood cancers
	Trial	Population	Patients	Design	Endpoint(s)	Status
	Phase III	Previously untreated CLL	535	•	Arm A: chlorambucil + obinutuzumab	•	Primary endpoint: PFS (Arm A vs. Arm B)	•	FPCD: Q2 2015
	ACE-CL-007(ELEVATE-TN)			•	Arm B: Calquence+ obinutuzumab	•	Secondary endpoints: IRC (independent	•	Data readout: Q2 2019
				•	Arm C:Calquence		review committee) assessed ORR, OS	•	Primary endpoint met
	NCT02475681						(Arm A vs. Arm B vs. Arm C)
	Phase III	Previously untreated CLL	780	•	Arm A; Calquence+ venetoclax	•	Primary - IRC assessed PFS (arm A vs	•	FPCD: Q1 2019
		fit		•	Arm B: Calquence+ venetoclax + obinutuzumab		arm C)	•	Data anticipated: 2021+
	ACE-CL-311			•	Arm C: FCR or BR	•	Secondary - IRC assessed PFS (arm B
	NCT03836261						vs arm C); INV assessed PFS (arm A vs
							arm C; arm B vs arm C)
	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 readout: Q2 2019
	NCT02970318				choice)	•	Secondary endpoints: INV-assessed	•	Primary endpoint met
							ORR, OS, DoR, PROs
	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: 2021+
							incidence of infections, RTs (Richter's
	NCT02477696						Transformation) and atrial fibrillation, OS
	Phase III	Previously untreated MCL	546	•	Arm A: Calquence+ bendamustine + rituximab	•	Primary endpoint: PFS by Lugano	•	FPCD: Q1 2017
	ACE-LY-308			•	Arm B: bendamustine + rituximab		Classification for NHL	•	Data anticipated: 2021+
						•	Secondary endpoints: IA, PFS, ORR;
	NCT02972840						IRC-assessed ORR, DoR, time to
							response, OS
					•	ORR at 36 cycles	•	FPCD: Q1 2016
	Phase II	Relapsed/ refractory CLL,	60	Calquencemonotherapy
	ACE-CL-208	intolerant to ibrutinib						•	Data anticipated: H1 2020
	NCT02717611
	Phase II	Relapsed/refractory and	48	Calquencemonotherapy	•	ORR	•	FPCD: Q4 2014
	15-H-0016	treatment naïve/del17p		•	Arm A: lymph node biopsy			•	Data anticipated: 2021+
	NCT02337829	CLL/SLL		•	Arm B: bone marrow biopsy
	Phase I/II	CLL/SLL/Richter's	306	Calquencemonotherapy	•	Safety, PK, PD	•	FPCD: Q1 2014
	ACE-CL-001	transformation		Dose escalation and expansion			•	Data anticipated: 2021

20NCT02029443

Oncology

CVRM

Respiratory

Other

								Approved medicines
Calquence(BTK inhibitor)			Late-stage development
		Early development
Blood cancers
	Trial	Population	Patients	Design	Endpoint(s)	Status
	Phase I/II	B-cell malignancies	40	Dose escalation and expansion trial of the combination of	•	Safety	•	FPCD: Q1 2015
	ACE-LY-001			Calquenceand ACP-319 (Pi3K inhibitor)	•	ORR	•	Data anticipated: H1 2020
	NCT02328014
	Phase I/II	Haematological malignancies	161	Calquence+ pembrolizumab	•	Safety	•	FPCD: Q1 2015
	ACE-LY-005					•	Secondary endpoints: ORR, DoR, PFS,	•	Data anticipated: 2021
							OS, TTNT (time to next therapy)
	NCT02362035
	Phase I/II	Waldenstrom	106	Calquencemonotherapy	•	ORR	•	FPCD: Q3 2014
	ACE-WM-001	microglobulinaemia						•	Data readout: Q4 2019
	NCT02180724
	Phase Ib	Relapsed/refractory de novo	21	Calquencemonotherapy	•	Safety	•	FPCD: Q3 2014
	ACE-LY-002	activated B-cell DLBCL						•	Data anticipated: H2 2019
	NCT02112526
	Phase Ib	MCL	70	Calquencein combination with bendamustine and rituxumab	•	Safety	•	FPCD: Q1 2016
	ACE-LY-106			•	Arm A: treatment naive			•	Data anticipated: 2021+
				•	Arm B: relapsed/refractory
	NCT02717624			•	Arm C: treatment naïve: Calquence+ venetoclax + rituxumab
	Phase Ib	Relapsed/refractory MM	28	•	Arm A:Calquence	•	Safety	•	FPCD: Q1 2015
	ACE-MY-001			•	Arm B: Calquence+ dexamethasone			•	Data readout: Q2 2019
	NCT02211014
	Phase I	Relapsed/refractory follicular	80	•	Arm A:Calquence	•	Safety	•	FPCD: Q1 2015
	ACE-LY-003	lymphoma		•	Arm B: Calquence+ rituximab			•	Data anticipated: 2021+
	NCT02180711			•	Arm C: Calquence+ rituximab + lenolidomide
	Phase I	Relapsed/refractory CLL/ SLL	12	Calquencein combination with ACP-319	•	Safety, PK, PD	•	FPCD: Q3 2014
	ACE-CL-002			dose escalation			•	Data anticipated: H2 2020
	NCT02157324
	Phase I	CLL/SLL/PLL	69	Calquence+ obinutuzumab	•	Safety, ORR	•	FPCD: Q4 2014
	ACE-CL-003			•	Arm A: relapsed/refractory	•	Secondary endpoints: PD, PFS, TTNT,	•	Data anticipated: 2021+
	NCT02296918			•	Arm B: treatment naïve		OS
			Calquence +venetoclax + rituxumab
				•	Arm C: relapsed/refractory
21				•	Arm D: treatment naïve

Oncology

CVRM

Respiratory

Other

								Approved medicines
Calquence(BTK inhibitor)			Late-stage development
		Early development
Blood cancers
	Trial	Population	Patients	Design	Endpoint(s)	Status
	Phase I	Japanese adults with	34	•	Calquencemonotherapy	•	Safety	•	FPCD: Q2 2017
		advanced B-cell malignancies		•	Dose confirmation and expansion	•	PK	•	Data anticipated: 2021+
	NCT03198650			•	Calquence+ obinutuzumab
	Phase I/II	CLL r/r	62	•	Arm A:ceralasertib (AZD6738) monotherapy	•	Identify dose of ceralasertib and safety	FPCD: Q1 2018
	CL-110			•	Arm B: Calquence+ ceralasertib (AZD6738)		of co-administration of Calquence+	Data anticipated: H1 2020
							ceralasertib
	NCT03328273
	Phase I/II	B-cell malignancies r/r	25	•	Part 1: Calquencedaily + vistusertib daily	•	MTD and optimal dosing schedule	FPCD: Q3 2017
	LY-110			•	Part 2: Calquencedaily + vistusertib 5 days on/2 days off	•	Safety	Data anticipated: H2 2020
	NCT03205046
	Phase III	CLL TN and r/r	600	•	Arm A: treatment naïve	•	Safety	Data anticipated: 2021+
	CL-312			•	Arm B: relapsed/refractory
				•	Arm C: prior BTKi therapy
	NCT04008706			•	Arm D: concomitant vitamin K antagonists
	Phase Ib/II	Relapsed/refractory	88	•	Arm 1: Calquence+ danvatirsen	•	Primary outcome; safety & tolerability	FPCD: Q2 2018
	PRISM	aggressive NHL		•	Arm 2: Calquence+ AZD6738	•	Secondary outcomes; ORR, DOR, PFS,	Data anticipated: 2021
				•	Arm 3: Calquence+ Hu5F9G4 + Rituxan		OS
	NCT03527147			•	Arm 4: Calquence+ AZD5153
				An open-label platform study with trial centres in US and UK

Oncology

CVRM

Respiratory

Other

22

						Approved medicines
Calquence(BTK inhibitor)		Late-stage development
	Early development
Other cancers
	Trial	Population	Patients	Design	Endpoint(s)	Status
	Phase Ib/II	≥ 2L glioblastoma multiforme	52	• Arm A: Calquence200mg BID	• Safety, ORR	• FPCD: Q1 2016
	ACE-ST-209			• Arm B: Calquence400mg QD		• Data anticipated: H2 2019
	NCT02586857

Oncology

CVRM

Respiratory

Other

23

							Approved medicines
Enhertu(trastuzumab deruxtecan, HER2 ADC)	Late-stage development
Early development
Breast and gastric cancers
	Trial	Population	Patients	Design	Endpoints	Status
	Phase II	HER2-positive, unresectable and/or	230	Randomised, open label, sequential assignment	Primary endpoint ORR	•	FPCD: Q4 2017
	DESTINY-Breast01	metastatic breast cancer patients		•	Enhertu		•	LPCD: Q4 2018
		previously treated with trastuzumab				Secondary end points DoR, CBR, CBR,	•	Data readout: Q2 2019
	NCT03248492	emtansine				PFS, OS
	Partnered
	Phase III	HER2-positive, unresectable and/or	600	Randomised open label parallel assignment	Primacy endpoint PFS	•	FPCD: Q4 2018
	DESTINY-Breast02	metastatic breast cancer pretreated with		•	Enhertu		•	Data anticipated 2021
		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: Q4 2018
	DESTINY-Breast03	metastatic breast cancer patients		•	Enhertu		•	Data anticipated 2021
		previously treated with trastuzumab and		•	Ado-trastuzumab emtansine	Secondary endpoints OS, ORR, DoR, CBR
	NCT03529110	taxane
	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 patients		•	Enhertu		•	Data anticipated 2021
				•	Physicians choice of SoC chemo (choice of capecitabine,	Secondary end points OS, DoR, ORR
	NCT03734029				eribulin, gemcitabine, paclitaxel or nab-paclitaxel)
	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		•	Enhertu		•	LPCD: Q2 2019
		adenocarcinoma patients who have		•	SoC chemo	Secondary end points PFS, OS, DoR,	•	Data readout Q1 2020
	NCT03329690	progressed on two prior treatment				DCR, TTF, range of PK endpoints
	Partnered	regimens
	Phase II	HER2-positive gastric cancer that cannot	72	Open label single group assignment	Primary endpoint ORR	•	FPCD: Q3 2019
	DESTINY-Gastric02	be surgically removed or has spread		•	Enhertu	Secondary endpoints PFS, ORR, OS, DoR	•	Data anticipated: H2 2020
	NCT04014075
	Partnered

Oncology

CVRM

Respiratory

Other

24

						Approved medicines
Enhertu(trastuzumab deruxtecan, HER2 ADC)	Late-stage development
Early development
Other cancers
	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
	NCT03384940	cancer		•Enhertu	Secondary end points PFS, OS, DoR,	•	Data anticipated H2 2020
	Partnered				range of PK endpoints
	Phase II	HER2-over-expressing or mutated,	130	Non randomised parallel group assignment	Primary end point ORR	•	FPCD Q2 2018
	NCT03505710	unresectable and/or metastatic NSCLC		•Enhertu	Secondary end points DoR, PFS, OS	•	Data anticipated H2 2020
	Partnered
	Phase I	Advanced solid malignant tumours	278	Non randomised single group assignment	Primary end points number of subjects with	•	FPCD Q3 2015
	NCT02564900			•Enhertu	AEs, tumour response	•	Data read out Q3 2018
				Secondary end points PK
	Partnered

Oncology

CVRM

Respiratory

Other

25

								Approved medicines
Lumoxiti	(moxetumomab pasudotox,CD22 mAb)	Late-stage development
Early development
Blood cancer
	Trial		Population	Patients	Design	Endpoints	Status
	Phase III		Adults with relapsed or	80	• Multicentre, single-arm,open-label Phase III study	• Primary endpoint: rate of durable CR	• FPCD: Q2 2013
	PLAIT		refractory HCL		• Lumoxitii.v. at the recommended dose	(complete response): CR maintained for	• Data readout: Q3 2017
	NCT01829711					> 180 days	• Primary endpoint met
						• Secondary endpoints
	Partnered					• Efficacy: CR rate, ORR, Duration of
							CR and ORR, TTR, PFS
						•	Safety and tolerability
						•	PK and immunogenicity

Oncology

CVRM

Respiratory

Other

26

						Approved medicines
Selumetinib (MEK inhibitor)			Late-stage development
		Early development
Paediatric neurofibromatosis type 1, solid tumours
Trial	Population	Patients	Design	Endpoints	Status
Phase II	Paediatric NF1	50 (stratum 1)	• Single arm: selumetinib 25mg/m2BID with 2 strata:	•	Complete partial and complete response	•	FPCD: Q3 2015
SPRINT		25 (Stratum 2)	• 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	•	Primary endpoint met
Partnered
Phase Ib	Advanced solid tumours	80 (dose escalation	Phase Ib open-label trial of MK-8353 in combination with	•	DLTs	•	FPCD: Q1 2019
Selumetinib + MK-8353 (ERK		trial)	selumetinib in participants with advanced solid tumours	•	AEs
inhibitor)				•	Study drug discontinuations due to an
NCT03745989					AE
Partnered (Merck Lead
study)

Oncology

CVRM

Respiratory

Other

27

							Approved medicines
Savolitinib (MET inhibitor)			Late-stage development
		Early development
NSCLC and other cancers
	Trial	Population	Patients	Design	Endpoints	Status
	Phase I	Advanced NSCLC	85	• Dose escalation trial	•	Primary endpoint: safety and tolerability	•	FPCD: Q2 2013
	NCT01985555	(all comers)		Conducted in China	•	Secondary endpoint: PK profile	•	Data anticipated: H2 2020
	Partnered
	Phase II	Lung PSC and other NSCLC	65	• Single arm trial: savolitinib QD	•	Primary endpoint: ORR	•	FPCD: Q1 2017
					•	Secondary endpoint: PFS, safety	•	Data anticipated: H1 2020
	NCT02897479			Conducted in China		parameters
	Partnered

Oncology

CVRM

Respiratory

Other

28

					Approved medicines
Cediranib (VEGF receptor inhibitor)		Late-stage development
	Early development
Ovarian cancer
Trial	Population	Patients	Design	Endpoints	Status
Phase IIb	PRR ovarian cancer - heavily	62	• Cediranib 30mg + Lynparza200mg BID	Primary endpoint:	• FPCD: Q1 2017
CONCERTO	pre-treated BRCAwt			• ORR	• LPCD: Q1 2019
NCT02889900					• Data readout: Q4 2019
			Secondary endpoints:
				• PFS, DoR, DCR, QoL, OS

Oncology

CVRM

Respiratory

Other

29

								Approved medicines
Capivasertib (AKT inhibitor)			Late-stage development
		Early development
Breast cancer, prostate cancer
	Trial	Population	Patients	Design	Endpoints	Status
	Phase III	Locally advanced or	800	Double-blind randomised comparative study	•	PFS	•	FPCD Q3 2019
		metastatic TNBC		•	Arm 1: capivasertib + paclitaxel	•	OS	•	Data anticipated: 2021+
	NCT03997123			•	Arm 2: placebo + paclitaxel
	CAPItello-290
	Phase II (ESR)	Metastatic castration resistant	150	Randomised comparative	•	PFS	•	FPCD Q1 2014
		prostate cancer eligible for		•	Arm 1: docetaxel + prednisolone + capivasertib			•	Data anticipated: H1 2020
	NCT02121639	treatment with docetaxel		•	Arm 2: docetaxel + prednisolone + placebo
	PROCAID	chemotherapy

Oncology

CVRM

Respiratory

Other

30

Oncology - early-stage development

									Approved medicines
Imfinzi(PD-L1 mAb)					Late-stage development
				Early development
Cancer
	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: H1 2020
	NCT01693562				•	Dose exploration: cohort at 20mg Q4W		immunogenicity and antitumour activity
					Global trial - nine countries
	Phase I	Imfinzi, azacitidine	Myelodysplastic	79	Dose escalation and dose expansion trial	•	Safety and tolerability of monotherapy	•	FPCD: Q2 2014
			syndrome		•	Part 1: Imfinzi		and combination	•	Data anticipated: H2 2020
	NCT02117219				•	Part 2 Arm 1: Imfinziand tremelimumab	•	Secondary endpoints include duration of
					•	Part 2 Arm 2: Imfinzi,tremelimumab and azacitidine		response, PFS and OS, PK and
					Global trial - four countries		immunogenicity
	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
	NCT02900157				US and Japan trial centers		infusion related reaction	•	Data anticipated: H1 2020
	Phase II	Imfinzi	NSCLC	320	5 modules encompassing 13 cohorts	•	Primary outcome; ORR	•	FPCD: Q1 2018
	HUDSON	Lynparza			•	Module 1;Imfinzi andLynparza	•	Secondary outcomes; efficacy including	•	Data anticipated: 2021+
		vistusertib			•	Module 2; Imfinziand danvatirsen		OS, PFS, DCR, and safety and
	NCT03334617	ceralasertib			•	Module 3; Imfinziand ceralasertib (AZD6738)		tolerability, DoR
		(AZD6738)			•	Module 4; Imfinziand vistusertib
		danvatirsen			•	Module 5; Imfinziand oleclumab
		oleclumab			•	Module 6;Imfinzi andEnhertu
		Enhertu			•	Module 7; Imfinziand cedirinib
		cediranib			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: Imfinzi	Primary	•	FPCD: Q4 2018
	COAST		unresectable		•	Arm B: Imfinzi+ oleclumab	• OR per RECIST v1.1	•	Data anticipated: H2 2020
	NCT03822351				•	Arm C: Imfinzi+ monalizumab
	Phase II	Imfinzi	Resectable, early	160	•	Arm A: Imfinzi	Primary	•	FPCD: Q1 2019
	NeoCOAST		stage NSCLC		•	Arm B: Imfinzi+ oleclumab	•	Major pathological response rate	•	Data anticipated: H2 2020
					•	Arm C: Imfinzi+ monalizumab
	NCT03794544				•	Arm D: Imfinzi+ danvatirsen

Oncology

CVRM

Respiratory

Other

32

								Approved medicines
Imfinzi(PD-L1 mAb) +					Late-stage development
				Early development
tremelimumab (CTLA-4 mAb)
Cancer
	Trial	Population	Patients	Design	Endpoints	Status
	Phase Ib/II	Hepatocellular carcinoma	545	•	Arm A: Imfinzi+ tremelimumab	•	Primary endpoints: Safety & tolerability,	•	FPCD: Q4 2015
	STUDY 22			•	Arm B: Imfinzi2L		DLTs	•	Data anticipated: H2 2020
				•	Arm C: tremelimumab 2L	•	Secondary endpoints: ORR, DoR, OS
	NCT02519348			•	Arm D: Imfinzi + tremelimumab
				•	Arm E: Imfinziin combination with bevacizumab
	Phase Ib	NSCLC	459	•	Dose escalation: minimum 5 cohorts exploring various treme	Primary endpoints:	•	FPCD: Q4 2013
	STUDY 006	(Immunotx naïve and			Q4W and Imfinzii.v. Q4W dose combinations, higher dose	•	Safety	•	LPCD: Q4 2016
		Immunotx pretreated patient			levels and alternate Q2 schedule added with amendment	•	Optimal biologic dose for the combination	•	Data anticipated: H1 2020
	NCT02000947	cohorts)		•	Dose expansion: MTD for the combination in escalation to be	•	OR
					explored in expansion	•	Secondary endpoints include antitumour
				North American, EU and ROW trial centres		activity, PK and immunogenicity
	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 combination	•	Data anticipated: H1 2020
	NCT02261220			•	Dose exploration: 2cohorts exploring various Q4W treme and	•	Secondary endpoints include
					Imfinzidose combinations and 2 cohorts exploring various Q2W		anti-tumour activity, PK/PD and
					treme and Imfinzidose combinations		immunogenicity
				North American, EU and ROW trial centres

Oncology

CVRM

Respiratory

Other

33

							Approved medicines
Imfinzi(PD-L1 mAb) +				Late-stage development
			Early development
monalizumab (NKG2a mAb)
Cancer
	Trial	Population	Patients	Design	Endpoints	Status
	Phase I/II	Advanced solid tumours	501	Escalation phase	Primary endpoints:	•	FPCD: Q2 2016
				• monalizumab + Imfinzii.v.	•	Safety	•	Data anticipated: 2021+
	NCT02671435				•	Exploration Phase: Objective Response
				Expansion phase		per RECIST
				• monalizumab + Imfinzii.v. recommended dose	•	Secondary endpoints include tumour
				Exploration phase		response (OR, DC, DoR, PFS and OS),
				• monalizumab + Imfinzii.v. recommended dose + SoC systemic		immunogenicity, pharmacokinetics,
				therapy with or without biologic agent and monalizumab in		pharmacodynamics
				combination with a biologic agent in adult subjects with CRC
				Global trial

Oncology

CVRM

Respiratory

Other

34

	Approved medicines
Imfinzi(PD-L1 mAb) +	Late-stage development
Early development
MEDI0457 (DNA HPV Vaccine)

Head and neck squamous cell carcinoma (HNSCC)

Trial	Population	Patients	Design	Endpoints	Status
Phase Ib/IIa	HPV associated	50	Multi-centre, open label trial to evaluate the safety and efficacy of	Primary endpoints:	•	FPCD: Q3 2017
	recurrent/metastatic head and		combination treatment with MEDI0457 and Imfinzi	Safety & Tolerability, ORR	•	Data anticipated: H2 2020
NCT03162224	neck cancer			Secondary endpoints:
				PK, ADA, DCR, OS, PFS

Oncology

CVRM

Respiratory

Other

35

							Approved medicines
AZD0466 (Bcl2/xL inhibitor)			Late-stage development
		Early development
Cancer
	Trial	Population	Patients	Design	Endpoints	Status
	Phase I	Advanced hematologic	102	Monotherapy dose escalation, consisting of two arms:	•	Primary: safety	•	FPCD: Q4 2019
		malignancies or solid tumors		• Arm A: Patients with low risk for tumour lysis syndrome (solid	•	Secondary: PK, anti-tumour activity	•	Data anticipated: 2021+
	NCT04214093			tumours, lymphomas, myelomas)
				• Arm B: Patients with high risk for tumour lysis syndrome
				(relapsed/refractory haem malignancies)

Oncology

CVRM

Respiratory

Other

36

							Approved medicines
MEDI1191 (IL12 modRNA)			Late-stage development
		Early development
Cancer
	Trial	Population	Patients	Design	Endpoints	Status
	Phase I	Advanced solid tumours	87	First-time-in-human Phase I, open-label,dose-escalation and	•	Primary endpoint: safety and tolerability	•	FPCD: Q2 2019
	NCT03946800			expansion study of MEDI1191 administered intratumourally as			•	Data anticipated: 2021+
				monotherapy and in combination with Imfinzi	•	Secondary endpoints: PK,
						immunogenicity and efficacy

Oncology

CVRM

Respiratory

Other

37

					Approved medicines
AZD1390 (ATM inhibitor)		Late-stage development
	Early development
Cancer
Trial	Population	Subjects	Design	Endpoints	Status
Phase I	Recurrent glioblastoma	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: 2021
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

Oncology

CVRM

Respiratory

Other

38

								Approved medicines
Adavosertib (AZD1775, WEE-1 inhibitor)	Late-stage development
Early development
Ovarian cancer, solid tumours
	Trial	Population	Patients	Design	Endpoints	Status
	Phase II	Platinum-resistant (PR)	96	•	Arm B: paclitaxel + adavosertib	•	Primary endpoint: ORR	•	FPCD: Q1 2015
		ovarian cancer		•	Arm C: carboplatin + adavosertib			•	LPCD: Q2 2018
	D6010C00004					•	Secondary endpoints: DoR, PFS, OS,	•	Data readout: Q3 2019
	NCT02272790			Global trial		DCR, safety and tolerability
	Phase I	Advanced solid tumours	130	•	Dose escalation trial to determine MTD (adavosertib +	•	Safety and tolerability	•	FPCD: Q3 2015
					Lynparza) followed by an expansions in SCLC	•	Secondary endpoints: ORR, DCR, DoR,	•	LPCD: Q4 2018
	D6010C00005			Conducted in US, Canada		PFS	•	Data readout Q4 2019
	NCT02511795
	Phase I	Advanced solid tumours	56	•	Dose escalation trial to determine MTD (adavosertib + Imfinzi)	•	Safety and tolerability	•	FPCD: Q4 2015
								•	LPCD: Q4 2018
	D6015C00002			Conducted in US			•	Data readout Q4 2019
	NCT02617277
	Phase I	Advanced solid tumours	33	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	•	LPCD: Q4 2018
	D6014C00006				225mg bid for 2.5 days plus caffeine (200mg), omeprazole		for cocktail parent compounds	•	Data readout Q4 2019
					(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
				Conducted in US		time-matched change from baseline
	Phase I	Advanced solid tumours	48	adavosertib monotherapy once daily.	•	Safety and tolerability	•	FPCD: Q4 2017
								•	LPCD: Q1 2019
	D6014C00007			Conducted in US and Europe			•	Data readout Q4 2019
	NCT03313557

Oncology

CVRM

Respiratory

Other

39

					Approved medicines
MEDI2228 (BCMA antibody drug conjugate)	Late-stage development
Early development
Cancer
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: 2021+
NCT03489525				• Determination of MTD
				• Secondary endpoints: pPK,
				immunogenicity, ORR, DCR, DoR, PFS,
				OS

Oncology

CVRM

Respiratory

Other

40

							Approved medicines
AZD2811 (AURN)					Late-stage development
				Early development
Cancer
Trial	Population	Patients	Design	Endpoints	Status
Phase I	Solid tumours	72	•	Arm 1: AZD2811 dose escalation	•	Safety and tolerability	•	FPCD: Q4 2015
NCT02579226			•	Arm 2: AZD2811 dose expansion SCLC	•	PK and efficacy	•	Data anticipated: H2 2020
Phase I	AML/high-risk MDS	130	•	Part A: AZD2811 monotherapy / azacitidine combination /	•	Safety and tolerability	•	FPCD: Q3 2017
				venetoclax combination dose escalation cohorts	•	PK and efficacy	•	Data anticipated: 2021+
NCT03217838			• Part B: AZD2811 monotherapy / azacitidine combination /
				venetoclax combination dose expansions to further explore the
				tolerability, PK and clinical activity

Oncology

CVRM

Respiratory

Other

41

						Approved medicines
AZD4573 (CDK9 inhibitor)		Late-stage development
	Early development
Cancer
	Trial	Population	Patients	Design	Endpoints	Status
	Phase I	Relapsed/refractory	45	Dose escalation in relapsed/refractory haematological	Primary:	•	FPCD: Q4 2017
		haematologic malignancies		malignancies	• safety/PK;	•	Data anticipated: H2 2020
	NCT03263637				Secondary:
				AZD4573 will be administered in 2 parallel arms (1-6 cohorts of	• efficacy
				dose escalations) based on the haematological malignancy

Oncology

CVRM

Respiratory

Other

42

							Approved medicines
AZD4635 (A2AR inhibitor)			Late-stage development
		Early development
Cancer
Trial	Population	Patients	Design	Endpoints	Status
Phase I	Phase Ia: patients with	306	Phase Ia - solid tumours or mCPRC:	Primary outcome measure:	•	FPCD: Q2 2016
	advanced solid tumours		•	AZD4635 monotherapy	•	Safety and tolerability
NCT02740985			•	AZD4635 + Imfinzi
	Phase Ib:		•	AZD4635 + abiraterone	Secondary outcome measures:
	Post-immunotherapy NSCLC		•	AZD4635 + enzalutamide	•	Preliminary assessment of anti-tumour
	Other post-immunotherapy		•	AZD4635 + Imfinzi + oleclumab		activity
		•	AZD4635 + docetaxel.
	solid tumours
		Phase Ib: AZD4635 monotherapy or AZD4635 + Imfinzidose
	Immune checkpoint-naïve
		expansions in NSCLC, mCRPC, CRC and other post-
	mCRPC Immune checkpoint-
		immunotherapy and immune checkpoint-naïve solid tumours
	naïve CRC
	Other immune checkpoint-		Conducted at sites in the US
	naïve solid tumours
Phase I	Healthy male volunteers	21	•	Part A 2-period randomised crossover study of single doses of	Primary outcome measures:	•	FPCD: Q4 2018
				AZD4635, nanosuspension or solid oral formulation in fasted	•	Cmax and exposure (AUC) of AZD4635	•	LPCD: Q2 2019
NCT03710434				state		solid oral formulation and nano-
			•	Part B, 4-period,open-label, randomised, crossover study of		suspension
				single doses of AZD4635 in the same subjects from Part A to
				assess food effect, pH effect and formulation variants
			Both parts conducted at a site in the UK
Phase II	Prostate cancer	60	ARM 1: AZD4635 + Imfinzi	•	Primary outcome measure: Efficacy;	•	FPCD: Q3 2019
NCT04089553			ARM 2: AZD4635 + oleclumab		(ORR and PSA response)
		Conducted at sites in the US	•	Secondary outcome measure: Efficacy,
						PK, safety and tolerability
Phase I	Japanese patients with	12	AZD4635 dose escalation	Primary outcome measure:	•	FPCD: Q3 2019
NCT03980821	advanced solid malignancies		Conducted at sites in Japan	•	Safety and tolerability
		Secondary outcome measure:
					•	PK and preliminary anti-tumour activity

Oncology

CVRM

Respiratory

Other

43

						Approved medicines
AZD5069 (CXCR2 antagonist)		Late-stage development
	Early development
Cancer
	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				•	LPCD: Q3 2018
	NCT02583477			Imfinziin combination with nab-paclitaxel and gemcitabine		•	Data anticipated: H2 2020
				Imfinziin combination with AZD5069

Oncology

CVRM

Respiratory

Other

44

MEDI5083 (CD40 ligand fusion protein ) + Imfinzi(PD-L1 mAb)

Cancer

Approved medicines Late-stage development Early development

Oncology

CVRM

Trial	Population	Patients	Design	Endpoints	Status
Phase I	Advanced solid tumours	204	Dose-escalation phase	Primary endpoints:	•	FPCD: Q1 2017
			•	Part 1: MEDI5083	•	Safety	•	Data anticipated: H1 2020
NCT03089645			•	Part 2: MEDI5083 + Imfinzii.v.	•	Determination of MTD
					•	Secondary endpoints: preliminary anti-
			Dose expansion phase		tumour activity, pharmacokinetics,
			•	Part 3: MEDI5083 recommended dose + Imfinzii.v.		pharmacodynamics, and immunogenicity
			US and Australian trial centres

Respiratory

Other

45

							Approved medicines
AZD5153 (BRD4 inhibitor)			Late-stage development
		Early development
Cancer
	Trial	Population	Patients	Design	Endpoints	Status
	Phase I/Ib	Relapsed/refractory solid	60	Monotherapy dose escalation in advanced solid tumours and	•	Primary: safety	•	FPCD: Q2 2017
	NCT03205176	tumours, lymphomas		lymphomas	•	Secondary: efficacy, PK	•	Data anticipated: H2 2020
			Dose escalation of AZD5153 in combination with Lynparzain
				platinum resistant/refractory HGS patients.

Oncology

CVRM

Respiratory

Other

46

							Approved medicines
MEDI5395 (rNDV GMCSF)			Late-stage development
		Early development
Cancer
	Trial	Population	Patients	Design	Endpoints	Status
	Phase I	Select advanced solid	164	First-time-in-human Phase I, open-label,dose-escalation and	•	Primary endpoint: safety and tolerability	•	FPCD: Q4 2019
	NCT03889275	tumours		expansion study of MEDI5395 in combination with Imfinzi	•	Secondary endpoints: PK, PD,	•	Data anticipated: 2021+
						immunogenicity and efficacy

Oncology

CVRM

Respiratory

Other

47

						Approved medicines
MEDI5752 (PD-1/CTLA-4 bispecific mAb)	Late-stage development
Early development
Cancer
	Trial	Population	Patients	Design	Endpoints	Status
	Phase I	Advanced solid tumours	272	Open-label,dose-escalation and dose-expansion	Primary endpoints:	•	FPCD: Q2 2018
					• dose-escalation: safety & determination	•	Data anticipated: 2021+
	NCT03530397			Dose-escalation: MEDI5752 i.v.	of MTD
					• dose-expansion: assessment of
				Dose-expansion : 2 cohort	antitumour activity based on OR
					Secondary endpoints:
					• PK, ADA, tumoural baseline PD-L1,
					assessment of antitumour activity
					based on OR, DoR, DC, PFS, OS

Oncology

CVRM

Respiratory

Other

48

							Approved medicines
AZD5991 (MCL1 inhibitor)			Late-stage development
		Early development
Cancer
	Trial	Population	Patients	Design	Endpoints	Status
	Phase I	Relapsed/refractory	177	• Arm1: monotherapy dose escalation expansions in	•	Primary: safety	•	FPCD: Q3 2017
		haematologic malignancies		relapsed/refractory haematological malignancies	•	Secondary: efficacy, PK	•	Data anticipated: H2 2020
	NCT03218683			• Arm2: combination dose escalation (AZD5991+venetoclax) in
				relapsed/refractory AML;. Four dose escalation cohorts.
				• i.v. route of administration
				• US only

Oncology

CVRM

Respiratory

Other

49

							Approved medicines
Ceralasertib (AZD6738, ATR inhibitor)			Late-stage development
		Early development
Cancer
Trial	Population	Patients	Design	Endpoints	Status
Phase I	Solid tumours	250	•	Arm 1: ceralasertib + carboplatin	•	Safety and tolerability	•	FPCD: Q4 2014
			•	Arm 2: ceralasertib dose escalation, ceralasertib + Lynparza	•	PK and efficacy	•	Data anticipated: 2021+
NCT02264678			•	Arm 3: ceralasertib + Imfinzi
			Trial conducted in North America, Europe and South Korea
Phase I	HNSCC	44	Window of opportunity	•	Biomarker change	•	FPCD: Q4 2017
			•	Arm 1: ceralasertib			•	Data anticipated: H2 2020
NCT03022409			•	Arm 2:Lynparza
			Trial conducted in US, France, Taiwan and the UK

Oncology

CVRM

Respiratory

Other

50

							Approved medicines
Danvatirsen (AZD9150, STAT3 inhibitor)	Late-stage development
Early development
Cancer
	Trial	Population	Patients	Design	Endpoints	Status
	Phase Ib/II	HNSCC	405	Dose escalation advanced solid and blood cancers	•	Safety/efficacy trial	•	FPCD: Q3 2015
				• Arm A1: AZD9150/Imfinzi			•	LPCD: Q2 2019
	NCT02499328			• Arm A2 : AZD5069/Imfinzi			•	Data anticipated: 2021
				• Arm A4: AZD9150/Imfinzi/treme
				• 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: 2021
	NCT03421353			• Arm A2-A5 : AZD9150/Imfinzi+ SoC chemo
				Dose expansion 1L HNSCC:
				• Arm D1/D2/D3: AZD9150 i.v. vs s.c. formulations/Imfinzi
				(advanced solid tumours)

Oncology

CVRM

Respiratory

Other

51

								Approved medicines
Oleclumab (MEDI9447, CD73 mAb)			Late-stage development
		Early development
Cancer
	Trial	Population	Patients	Design	Endpoints	Status
	Phase I	Advanced malignancies	310	Dose escalation phase	Primary endpoints:	•	FPCD: Q3 2015
				•	oleclumab i.v.	•	Safety	•	Data anticipated: 2021
	NCT02503774			•	oleclumab i.v. + Imfinzii.v.	•	Determination of MTD
						•	Secondary endpoints include preliminary
				Dose expansion phase		anti-tumour activity, PK, PD,
				•	oleclumab i.v. recommended dose + Imfinzii.v.		immunogenicity and biomarker activity
				US, South Korean and Australian trial centres
	Phase Ib/II	Pancreatic	309	•	Arm A1: gemcitabine and nab paclitaxel i.v.	Primary endpoints:	•	FPCD: Q2 2018
		1L and 2L with prior		•	Arm A2: gemcitabine and nab paclitaxel i.v. + oleclumab i.v.	•	Safety and anti-tumour activity	•	Data anticipated: 2021
	NCT03611556	gemcitabine-based		•	Arm A3: gemcitabine and nab paclitaxel i.v. + oleclumab i.v. +
		chemotherapy			Imfinzi i.v.	•	Secondary endpoints include PFS, PK,
			•	Arm B1: mFOLFOX (oxaliplatin, leucovorin, 5-FU) i.v.
					immunogenicity, safety and anti-tumour
				•	Arm B2: mFOLFOX (oxaliplatin, leucovorin, 5-FU) i.v. +
					activity
					oleclumab i.v.
				•	Arm B3: mFOLFOX (oxaliplatin, leucovorin, 5-FU) i.v. +
					oleclumab i.v. + Imfinzii.v.
				US, Norway, Spain and Australian trial centres
	Phase Ib/II	NSCLC	98	•	Arm A: oleclumab i.v. + Tagrisso	Primary endpoints:	•	FPCD: Q2 2018
						•	Safety	•	Data anticipated: 2021+
	NCT03381274			US, South Korean and Taiwan trial centres	•	ORR
						Secondary endpoints:
						• DoR, DCR, PFS, OS, PK and
							immunogenicity

Oncology

CVRM

Respiratory

Other

52

							Approved medicines
AZD9496 (SERD, oral)				Late-stage development
			Early development
Breast cancer
	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: PD changes	•	FPCD: Q4 2017
	NCT03236974			pharmacodynamics trial to compare and assess the biological		to ER expression following treatment with	•	Data readout: Q4 2019
			effects of AZD9496 and Faslodexin postmenopausal women		AZD9496 or Faslodex
				•	Secondary outcome measures:
				with ER+, HER2- primary breast cancer. Patients will receive
					pharmacodynamics changes to Ki67 and
				AZD9496 or Faslodexand will have a pre-dose and an on-
					PgR expression following treatment with
				treatment core biopsy after 5-14 days of commencing
					AZD9496 or Faslodex
				treatment.
				•	Safety, tolerability + pharmacokinetics

Oncology

CVRM

Respiratory

Other

53

							Approved medicines
AZD9833 (SERD, oral)				Late-stage development
			Early development
Breast cancer
	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 PK of AZD9833 alone and in
				of patients. The trial will determine the maximum tolerated		combination with palbociclib
				dose of AZD9833 as monotherapy and in combination with		antitumour activity
				palbociclib. In addition, randomised expansion cohort(s) at
				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

Oncology

CVRM

Respiratory

Other

54

BioPharmaceuticals - approved medicines and late-stage pipeline

									Approved medicines
Farxiga	(SGLT2 inhibitor)			Late-stage development
		Early development
Heart failure and chronic kidney disease
	Trial		Population	Patients	Design	Endpoints	Status
	Phase III		CHF patients with HFrEF	4,744	•	Arm 1: Farxiga10mg or 5 mg QD + SoC therapy	•	Primary endpoint: time to the first	•	FPCD: Q1 2017
	Dapa-HF				•	Arm 2: placebo + SoC therapy		occurrence of any of the components of	•	LPCD Q4 2018
								the composite: CV death or	•	Data readout: Q3 2019
	NCT03036124				•	Global trial - 20 countries		hospitalisation for HF or an urgent HF	•	Primary endpoint met
								visit
	Phase III		Patients With CKD	4,000	•	Arm 1: Farxiga10mg or 5 mg QD	•	Primary endpoint: time to the first	•	FPCD: Q1 2017
	Dapa-CKD				•	Arm 2: placebo		occurrence of any of the components of	•	LPCD: Q1 2019
					Global trial - 21 countries		the composite: ≥50% sustained decline	• Data anticipated: 2021
	NCT03036150					in eGFR or reaching ESRD or CV death
								or renal death
	Phase III		CHF patients with HFpEF	6,100	•	Arm 1: Farxiga10mg QD	•	Primary endpoint: time to the first	•	FPCD: Q4 2018
	DELIVER				•	Arm 2: placebo		occurrence of any of the components of	•	Data anticipated: 2021+
					•	Global trial - 21 countries		the composite: CV death or
	NCT03619213					hospitalisation for HF or an urgent HF
								visit
	Phase III		CHF patients with HFpEF	500	•	Arm 1: Farxiga10mg QD	• Dual primary endpoint: 1) change from	•	FPCD: Q2 2019
	DETERMINE-preserved				•	Arm 2: placebo		baseline in 6 min walking distance at	•	Data anticipated: H1 2020
	NCT03877224				•	Global trial - 12 countries		Week 16 2) change from baseline in
					KCCQ-TSS at Week16
	Phase III		CHF patients with HFrEF	300	•	Arm 1: Farxiga10mg QD	•	Primary endpoint: change from	•	FPCD: Q2 2019
	DETERMINE-reduced				•	Arm 2: placebo		baseline in 6 min walking distance at	•	Data anticipated: H1 2020
	NCT03877237				•	Global trial - 9 countries		Week 16

Oncology

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Respiratory

Other

56

								Approved medicines
Brilinta(P2Y12 receptor antagonist)			Late-stage development
		Early development
Cardiovascular risk reduction
	Trial	Population	Patients	Design	Endpoints (primary)	Status
	Phase III	Patients with type-2 diabetes	19,000	•	Arm 1: Brilinta60mg BiD	•	Primary endpoint: composite of CV	•	FPCD: Q1 2014
	THEMIS	and coronary artery disease		•	Arm 2: placebo BID		death, non-fatal MI and non-fatal stroke	•	LPCD: Q2 2016
		without a previous history of		on a background of acetylsalicylic acid if not contra-indicated or			•	Data readout: Q1 2019
	NCT01991795	MI or stroke		not tolerated	Secondary endpoints:	•	Primary endpoint met
				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	11,000	•	Arm 1: Brilinta90mg BiD	Primary endpoint:	•	FPCD: Q1 2018
	THALES	stroke or transient ischaemic		•	Arm 2: placebo BiD	•	Prevention of the composite of	•	LPCD: Q4 2019
		attack		on a background of acetylsalicylic acid if not contra-indicated or		subsequent stroke and death at 30 days	•	Data readout: Q1 2020
	NCT03354429			not tolerated			•	Primary endpoint met
				Global trial - 28 countries	Secondary endpoints include:
				•	Prevention of subsequent ischaemic
							stroke at 30 days
						•	Reduction of overall disability at 30 days
	Phase III	Peadiatric patients (2-18 years	182	• Arm 1: Brilinta15, 30 or 45mg (dose based on subject weight)	•	Primary endpoint: the number of vaso-	•	FPCD: Q3 2018
	HESTIA3	old) with sickle cell disease		•	Arm 2: placebo		occlusive crisis which is the composite	•	Data anticipated: 2021
	NCT03615924			Global trial - 18 countries		of painful crisis and/or acute chest pain

Oncology

CVRM

Respiratory

Other

57

								Approved medicines
Lokelma(sodium zirconium cyclosilicate)		Late-stage development
	Early development
Hyperkalaemia
	Trial	Population	Patients	Design	Endpoints	Status
	Phase II/III	Hyperkalaemia	103	•	Arm 1: Lokelma5g TID for 48 hours	•	Primary endpoint: exponential rate of	•	FPCD: Q2 2017
	Dose-response Study in			•	Arm 2: Lokelma10g TID for 48 hours		change in serum potassium	•	LPCD: Q1 2018
	Japan			•	Arm 3: placebo TID for 48 hours			•	Data readout: Q3 2018
	NCT03127644			Japan			•	Primary endpoint met
	Phase III	Hyperkalaemia	150	•	Arm 1: Open-labelLokelma10g TID for up to 72 hrs followed	•	Primary endpoint: safety and tolerability	•	FPCD: Q3 2017
					by Lokelma5g QD for 12 months. Option to uptitrate to 10		as measured by adverse events	•	LPCD: Q3 2019
	NCT03172702				and15g QD or downtitrate to 5g QOD (or 2.5g QD)		reporting, vital signs, ECGs, physical	•	Data readout: Q3 2019
	J-LTS						examinations and safety laboratory	•	Primary endpoint met
				Japan		measurements
	Phase IIIb	Patients on haemodialysis	180	•	Arm 1: Lokelma5g QD for 8 weeks on non-dialysis days.	•	Primary endpoint: proportion of patients	•	FPCD: Q4 2017
	DIALIZE	with persistent pre-dialysis			Option to uptitrate to 10 and15g QD.		who maintain a pre-dialysis serum K	•	LPCD: Q4 2018
		hyperkalaemia		•	Arm 2: placebo QD for 8 weeks on non-dialysis days		between 4.0-5.0 mmol/L on 3 out of 4	•	Data readout: Q1 2019
	NCT03303521						dialysis treatments following the long	•	Primary endpoint met
				Global trial - four countries		interdialytic interval
	Phase II	Patients with chronic heart	280	•	Arm 1: Lokelma5g QD for 12 weeks. Option to uptitrate to 10	•	Primary endpoint: difference between	•	FPCD: Q3 2018
	PRIORITIZE HF	failure and hyperkalaemia or			and 15g QD or downtitrate to 5g QOD		Lokelmaand placebo in RAAS (renin-	•	Data readout: H2 2020
		at high risk of developing		•	Arm 2: placebo QD for 12 weeks		angiotensin-aldosterone system)
	NCT03532009	hyperkalaemia		Global trial - nine countries		blockade treatment.

Oncology

CVRM

Respiratory

Other

58

								Approved medicines
Roxadustat (HIF-PH inhibitor)			Late-stage development
		Early development
Anaemia
	Trial	Population	Patients	Design	Endpoints	Status
	Phase III	Anaemia in CKD in patients	922	•	Arm 1: roxadustat	•	Primary endpoint: Haemoglobin response	•	FPCD: Q4 2012
	ANDES	not receiving dialysis		•	Arm 2: placebo			•	LPCD: Q3 2018
	NCT01750190			Global trial			•	Data readout: Q4 2018
					•	Primary endpoint met
	Partnered							Sponsored by FibroGen
	Phase III		597	•	Arm 1: roxadustat	•	Primary endpoint: Haemoglobin response	•	FPCD: Q2 2013
	ALPS			•	Arm 2: placebo			•	LPCD: Q4 2017
	NCT01887600			Global trial			•	Data readout: Q3 2018
					•	Primary endpoint met
	Partnered							Sponsored by Astellas
	Phase III		616	•	Arm 1: roxadustat	•	Primary endpoint: Haemoglobin response	•	FPCD: Q1 2014
	DOLOMITES			•	Arm 2: darbepoetin alfa			•	Data anticipated: H1 2020
	NCT02021318			Global trial			Sponsored by Astellas
	Partnered
	Phase III		2,781	•	Arm 1: roxadustat
		•	Primary efficacy endpoint: Haemoglobin	•	FPCD: Q3 2014
	OLYMPUS			•	Arm 2: placebo		response	•	LPCD: Q4 2018
	NCT02174627			Global trial			•	Data readout: Q4 2018
			•	Primary safety objective: Contribute CV	•	Primary endpoint met
							safety data to pooled safety	Sponsored by AstraZeneca
						•	analyses across the Phase III program
	Phase III	Anaemia in CKD in patients	2,133	•	Arm 1: roxadustat	•	Primary efficacy endpoint: Haemoglobin	•	FPCD: Q3 2014
	ROCKIES	receiving dialysis		•	Arm 2: epoetin alfa		response	•	LPCD: Q3 2018
	NCT02174731			Global trial			•	Data readout: Q4 2018
			•	Primary safety objective:Contribute CV	•	Primary endpoint met
							safety data to pooled safety	Sponsored by AstraZeneca
						•	analyses across the Phase III program
	Phase III		741	•	Arm 1: roxadustat	•	Primary endpoint: Haemoglobin response	•	FPCD: Q4 2014
	SIERRAS			•	Arm 2: epoetin alfa			•	LPCD: Q3 2018
	NCT02273726			Global trial			•	Data readout: Q4 2018
					•	Primary endpoint met
	Partnered							Sponsored by FibroGen
	Phase III		838	•	Arm 1: roxadustat	•	Primary endpoint: Haemoglobin response	•	FPCD: Q4 2014
	PYRENEES			•	Arm 2: epoetin alfa or darbepoetin alfa			•	LPCD: Q3 2018
	NCT02278341			Global trial			•	Data readout: Q3 2018
					•	Primary endpoint met
	Partnered							Sponsored by Astellas

Oncology

CVRM

Respiratory

Other

59

								Approved medicines
Roxadustat (HIF-PH inhibitor)			Late-stage development
		Early development
Anaemia
	Trial	Population	Patients	Design	Endpoints	Status
	Phase III	Anaemia in newly initiated	1,043	•	Arm 1: roxadustat	•	Primary endpoint: Haemoglobin response	•	FPCD: Q4 2013
	HIMALAYAS	dialysis patients		•	Arm 2: epoetin alfa			•	LPCD: Q3 2018
								•	Data readout: Q4 2018
	NCT02052310			Global trial			•	Primary endpoint met
	Partnered							Sponsored by FibroGen
	Phase III	Anaemia in lower risk MDS	184	Open label roxadustat lead-in	•	Primary endpoint: Proportion of patients	•	FPCD: Q3 2017
		patients		Arm 1: roxadustat		achieving transfusion independence	•	Data anticipated: 2021
	NCT03263091			Arm 2: placebo			Sponsored by FibroGen
	Partnered			US/global trial
	Phase II/III	Anaemia in lower risk MDS	175	Open label roxadustat lead-in	•	Primary endpoint: Haemoglobin
		patients		Arm 1: roxadustat		response	•	FPCD: Q2 2018
	NCT03303066			Arm 2: placebo			•	Data anticipated: 2021
	Partnered			China			Sponsored by FibroGen
	Phase II	Anemia in patients receiving	100	US	•	Primary endpoint: Maximum change in	•	FPCD: Q3 2019
		chemotherapy treatment for					hemoglobin within 16 weeks from	•	Data anticipated: H2 2020
	NCT04076943	non-myeloid malignancies					baseline without RBC transfusion	Sponsored by FibroGen
	Partnered

Oncology

CVRM

Respiratory

Other

60

							Approved medicines
	Eklira/Tudorza (LAMA, DPI)			Late-stage development
			Early development
COPD
	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 PK of aclidinium	•	FPCD: Q2 2018
		subjects		trial	bromide and its metabolites after single	•	Data anticipated: 2021
	NCT03276052				and multiple doses (BID) of aclidinium
				• Arm 1: aclidinium bromide 200 μg DPI	bromide 200 μg, 400 μg and 800 μg
				• Arm 2: aclidinium bromide 400 μg DPI	• To evaluate the safety, and tolerability
				• Arm 3: aclidinium bromide 800 μg DPI	of aclidinium bromide 200 μg, 400 μg and
					800 μg after single and multiple dose
				Global trial - one Country	administration (BID)

Oncology

CVRM

Respiratory

Other

61

								Approved medicines
Duaklir Genuair (LAMA/LABA, DPI)			Late-stage development
		Early development
COPD
	Trial	Population	Patients	Design	Endpoints	Status
	Phase III	Patients with stable COPD	1,060	•	Arm 1: Duaklir Genuair400/12 μg DPI	Primary endpoints:	•	FPCD: Q1 2017
	AVANT			•	Arm 2: aclidinium bromide 400 μg DPI	•	Change from baseline in one hour	•	Data anticipated: 2021
	NCT03022097			•	Arm 3: formoterol fumarate 12 μg DPI		morning post-dose dose FEV1 Duaklir
			• Arm 4: tiotropium 18 μg DPI		Genuair400/12 μg compared to
							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

Oncology

CVRM

Respiratory

Other

62

								Approved medicines
Breztri(PT010, LAMA/LABA/ICS, pMDI)	Late-stage development
Early development
COPD
	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 endpoint.	•	LPCD: Q3 2016
					MDI 320/14.4/9.6µg BID pMDI		change from baseline in BMD of the	•	Data readout: Q1 2018
	NCT02536508			•	GFF (glycopyrronium and formoterol fumarate) MDI 14.4/9.6µg		lumbar spine measured using DXA (dual	•	Primary endpoints met
					BID pMDI		energy X-ray absorptiometry) scans of
				•	BFF (budesonide and formoterol fumarate) MDI 320/9.6µg BID		L1-L4 at week 52
					pMDI	•	Ocular sub-study safety endpoint change
				Randomised, double-blind,chronic-dosing,multi-centre		from baseline in LOCS III at week 52
				Country - US
	Phase III	Moderate to very severe	8,588	Treatments (1-year treatment period)	•	Primary endpoint: rate of moderate or	•	FPCD: Q3 2015
	ETHOS	COPD		•	BGF MDI 320/14.4/9.6µg BID pMDI		severe COPD exacerbations	•	LPCD: Q3 2018
				•	BGF MDI 160/14.4/9.6µg BID pMDI			•	Data readout: Q3 2019
	NCT02465567			•	BFF MDI 320/9.6µg BID pMDI	•	Secondary endpoint: time to first	•	Primary endpoint met
				•	GFF MDI 14.4/9.6µg BID pMDI		moderate or severe COPD exacerbation
				Randomised, double-blind,multi-centre and parallel-group
				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	•	FEV1area 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-
				Randomised, double-blind,parallel-group, and chronic dosing and		dose trough FEV1over 24 weeks (BGF
				multi-centre		MDI vs. GFF MDI)
						•	TDI focal score over 24 weeks (BGF
							MDI vs. BFF MDI and BGF MDI vs. GFF
				Multi-country		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	•	Primary safety endpoint met
				•	Symbicort Turbuhaler 400/12µg BID DPI
				Randomised, double-blind,parallel-group, chronic dosing,
				multicenter
				Country: Japan

Oncology

CVRM

Respiratory

Other

63

					Approved medicines
Daliresp/Daxas	(PDE4 inhibitor, oral)		Late-stage development
	Early development
COPD
Trial	Population	Patients	Design	Endpoints	Status
Post Launch	COPD	124,080	• This is a retrospective cohort trial comparing COPD patients	• Primary endpoint: all-cause mortality (up	• Data anticipated: 2021+
PASS			aged 40 years and older with new exposure to roflumilast with	to five years)
NCT03381573			up to 5 unexposed (i.e., not roflumilast-exposed) COPD
		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
			healthcare, death, and demographics data).

Oncology

CVRM

Respiratory

Other

64

								Approved medicines
Fasenra(IL5R mAb)					Late-stage development
				Early development
Severe, uncontrolled asthma
	Trial	Population	Patients	Design	Endpoints	Status
	Phase III	A multi-centre,open-label,	770	•	Arm 1: Fasenra30mg Q4W s.c.	•	Primary endpoint: safety and tolerability	•	FPCD: Q2 2016
	MELTEMI	safety extension trial with		•	Arm 2: Fasenra30mg Q8W s.c.			•	LPCD: Q3 2019
		Fasenrafor asthmatic adults						•	Data anticipated: H2 2020
	NCT02808819	on ICS plus LABA2 Agonist		Global trial - 15 countries
		Age 18-75 years
	Phase IIIb	Severe eosinophilic	600	Arm 1: Fasenra30mg Q8W s.c.	•	Primary endpoint: reduction of oral	•	FPCD: Q3 2018
	PONENTE	asthmatics receiving HD ICS					corticosteroid dose	•	LPCD: Q3 2019
		+ LABA and chronic OCS with		38-week trial			•	Data anticipated: H2 2020
	NCT03557307	or without additional asthma		Global trial - 16 countries
		controller(s).
		Age 18 Years and older
	D3250C00036 China	Severe, uncontrolled asthma,	666	•	Arm 1: Fasenra30mg Q8W s.c.	•	Primary endpoint: annual asthma	•	FPCD: Q4 2017
	ICS/LABA Trial (MIRACLE)	despite background controller		•	Arm 2: placebo s.c.		exacerbation rate	•	Data readout: 2021+
		medication, MD & HD ICS +				•	Secondary endpoints: assess pulmonary
	NCT03186209	LABA ± chronic OCS		56-week trial		function, asthma symptoms, other
		Age 12-75 years		Global trial - 4 countries		asthma control metrics

Oncology

CVRM

Respiratory

Other

65

								Approved medicines
Fasenra(IL5R mAb)					Late-stage development
				Early development
Severe, uncontrolled asthma
	Trial	Population	Patients	Design	Endpoints	Status
	Phase III	Severe asthma, inadequately	2,550	•	Arm 1: Fasenra30mg Q4W s.c.	•	Primary endpoint: safety and tolerability	•	FPCD: Q4 2014
	BORA	controlled despite background		•	Arm 2: Fasenra30mg Q8W s.c.*			•	Data readout: Q3 2018
		controller medication, MD &						•	Primary endpoint met
	NCT02258542	HD ICS + LABA ± chronic		•	placebo administered at select interim visits to
		OCS			maintain blind between treatment arms
		Age 12-75 years
				56-week (adults)
				108-week (adolescents)
				Global trial - 24 countries
	Phase III	Severe asthma, inadequately	120	•	Arm 1: Fasenra30mg Q4W s.c.	•	Primary endpoint: functionality, reliability,	•	FPCD: Q2 2015
	GREGALE	controlled despite background					and performance of a pre-filled syringe	•	Data readout: Q2 2016
		controller medication, MD &		28-week (adults)		with Fasenraadministered at home	•	Primary endpoint met
	NCT02417961	HD ICS + LABA ± chronic		Global trial - two countries
		OCS
		Age 18-75 years
	Phase lll	A double-blind, randomised,	38	•	Arm 1 : Fasenra30mg Q4W s.c.	•	Primary endpoint: safety and tolerability	•	FPCD Q4 2016
	ARIA	parallel group, placebo-		•	Arm 2: placebo s.c.			•	Data anticipated: H1 2020
		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
		Age 18-65 years
	Phase lll	A multi-centre, randomised,	100	•	Arm 1: Fasenra30mg Q4W s.c. with one dose of seasonal	Primary endpoints:	•	FPCD: Q3 2016
	ALIZE	double-blind, parallel group,			influenza virus vaccine IM at week eight	•	Post-dosestrain-specific HAI) antibody	•	Data readout: Q3 2017
		placebo-controlled, Phase IIIb		•	Arm 2: placebo Q4W s.c. with one dose of seasonal influenza		GMFRs	•	Primary endpoint met
	NCT02814643	trial to evaluate the potential			virus vaccine intra muscular at week	•	Post-dosestrain-specific serum HAI
		effect of Fasenraon the					antibody GMTs
		humoral immune response to		12-week trial	•	Proportion of patients who experience a
		the seasonal influenza					strain-specificpost-dose antibody
		vaccination in adolescent and					response with antibody response defined
		young adult patients with					as a ≥4-fold rise in HAI antibody titer
		severe asthma
		Ages 12-21 years

Oncology

CVRM

Respiratory

Other

66

								Approved medicines
Fasenra(IL5R mAb)					Late-stage development
				Early development
Severe, uncontrolled asthma
	Trial	Population	Patients	Design	Endpoints	Status
	Phase III	Severe asthma on ICS-LABA	120	Open label Fasenra30mg Q4w	•	Primary endpoint: percentage of patients/	•	FPCD: Q4 2016
	GRECO	Age 18-75 years					caregivers who successfully self	•	Data readout: Q4 2017
				28-week trial		administer at home	•	Primary endpoint met
	NCT02918071			Global trial - two countries
	Phase lllb	A multi-centre, randomised,	800	•	Arm 1: Fasenra30mg Q8W s.c.	•	Primary endpoint: rate of asthma	•	FPCD: Q3 2017
	ANDHI	double-blind, parallel group,		•	Arm 2: placebo s.c.		exacerbations	•	LPCD: Q1 2019
		placebo controlled, Phase IIIb				•	Secondary outcome measures: Saint	•	Data readout: Q4 2019
	NCT03170271	trial to evaluate the safety and		24-week trial		George Respiratory Questionnaire	•	Primary endpoint met
		efficacy of Fasenra30 mg s.c.		Global trial - 15 countries		(SGRQ)
		in patients with severe asthma
		uncontrolled on SoC
		treatment.
		Age 18-75
	Phase I	Healthy volunteers	162	Open label trial to compare 30 mg FasenraPK 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

Oncology

CVRM

Respiratory

Other

67

								Approved medicines
Fasenra(IL5R mAb)					Late-stage development
				Early development
Nasal polyposis, other
	Trial	Population	Patients	Design	Endpoints	Status
	Phase III	Patients with severe bilateral	400	•	Arm 1: Fasenra30mg Q8W s.c.	•	Primary endpoint: effect of Fasenraon	•	FPCD: Q1 2018
	OSTRO	nasal polyposis who are still		•	Arm 2: placebo s.c.		nasal polyp burden and on patient	•	LPCD: Q2 2019
		symptomatic despite standard					reported nasal blockage	•	Data anticipated: H2 2020
	NCT03401229	of care therapy		56-week trial
		Age 18-75 years		Global trial- 8 countries
	Phase III	Patients with eosinophilic	148	Arm 1: Fasenra30mg Q8W s.c.	•	Primary endpoint: Change in endoscopic	•	FPCD: Q4 2019
	ORCHID	chronic rhinosinusitis with		Arm 2: placebo Q8W s.c.		total nasal polyp score and Change in	•	Data anticipated: 2021+
		severe nasal polyposis					mean nasal blockage score
	NCT04157335	Age 18-75 years		56-week trial
				Asian countries (4 countries)
	Phase III	Patients with relapsing or	140	•	Arm 1: Fasenra30mg Q4W s.c.	•	Primary endpoint: Proportion of patients	•	FPCD: Q4 2019
	MANDARA	refractory EGPA on		•	Arm 2: mepolizumab 300mg Q4W s.c.		achieving remission (BVAS=0 and OCS	•	Data anticipated: 2021+
		corticosteroid therapy with or					dose ≤ 4mg/day) at both weeks 36 and
	NCT04157348	without stable		52-week trial with a minimum 1 year open label extension		48.
		immunosuppressive therapy		Global trial- 9 countries
		Age 18 years and older
	Phase III	Patients with HES (history of	120	•	Arm 1: Fasenra30mg Q4W s.c.	•	Primary endpoint: Time to first HES	•	FPCD: Q4 2019
	NATRON	persistent eosinophilia >1500		•	Arm 2: placebo Q4W s.c.		worsening/flare.	•	Data anticipated: 2021+
		cells/μL with evidence of
	NCT04191304	end organ manifestations		24-week trial with a minimum 1 year open label extension
		attributable to eosinophilia)		Global trial- 9-12 countries
		and signs or symptoms of HES
		worsening/flare at Visit 1
		Age 12 years and older
	Phase III	Documented diagnosis of EoE	170	•	Arm 1: Fasenra30mg Q4W s.c.	•	Primary endpoints:	•	Initiating
	Age 12 to 65 years		•	Arm 2: placebo Q4W s.c.	Histologic response at week 24	•	Data anticipated: 2021+
	MESSINA
			24-week double blind treatment period and open label period(s)	Change from baseline in DSQ score at
						week 24

Oncology

CVRM

Respiratory

Other

68

								Approved medicines
Fasenra	(IL5R mAb)				Late-stage development
			Early development
COPD
	Trial		Population	Patients	Design	Endpoints	Status
	Phase III		Patients with moderate to very	1216	•	Double-blind, placebo controlled, single dose (100mg q8w)	• Primary endpoint: annualized rate of	•	FPCD Q4 2019
	RESOLUTE		severe COPD with a history of		•	56-week treatment	moderate or severe exacerbations over	•	Data anticipated: 2021+
			frequent exacerbations on a		•	Global trial	56 weeks
	NCT04053634		background triple therapy
			(ICS/LABA/LAMA)
			Age 40-85 years

Oncology

CVRM

Respiratory

Other

69

									Approved medicines
	PT027 (SABA/ICS, pMDI)				Late-stage development
				Early development
Asthma
	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: 2021
	NCT03769090			•	BDA MDI 160/180 μg prn	Secondary endpoints:
			•	AS (albuterol sulphate) MDI 180 μg prn
				•	Severe exacerbation rate (annualised)
	Managed by Avillion
			Randomised, double-blind,multi-centre, parallel group	•	Total corticosteroid exposure over the
				Multi-country		treatment period
				•	Asthma Control Questionnaire -5 change
							from baseline and responder analysis at
							Week 24
						•	Asthma quality of life questionnaire for 12
							years and older/peadiatric asthma quality
							of life questionnaire change from baseline
							and responder analysis at week 24
	Phase III	Mild to moderate asthma	600	Treatments (12 week treatment period)	Dual primary endpoints:	•	FPCD: Q2 2019
	DENALI			• BDA MDI 80/180 μg QID	• Change from baseline in FEV1 AUC0-6	• Data anticipated: H2 2020
				• 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
				Multi-country
	Phase III	Asthma with exercise induced	60	Treatments (single dose)	Primary endpoint:	•	FPCD Q1 2019
	TYREE	bronchoconstriction		• BDA MDI 160/180 μg	•	The maximum percentage fall from	• Data anticipated: H2 2020
				• placebo MDI QID		post-dose,pre-exercise baseline in
							forced expiratory volume in 1 second
	Managed by Avillion			Randomised, double-blind,multi-centre crossover		(FEV1) observed up to 60 minutes post-
						exercise challenge
				Country: US

Oncology

CVRM

Respiratory

Other

70

									Approved medicines
	Tezepelumab (TSLP mAb)				Late-stage development
				Early development
Severe, uncontrolled asthma
	Trial	Population	Patients	Design	Endpoints	Status
	Phase III	Severe asthma	1,061	•	Arm 1: tezepelumab s.c.	•	Primary endpoint: Annual asthma	•	FPCD: Q1 2018
	NAVIGATOR	Age 12-80 years		•	Arm 2: placebo s.c.		exacerbation rate	•	LPCD: Q3 2019
						•	Secondary endpoints: Change from	•	Data anticipated: H2 2020
	NCT03347279			52 week trial		baseline in pre-BD FEV1, asthma related
							QoL (AQLQ(S)+12), asthma control
	Partnered			Global trial - 18 countries		(ACQ-6)
	Phase III	Severe asthma	150	•	Arm 1: tezepelumab s.c.	•	Primary endpoint: Reduction from	•	FPCD: Q2 2018
	SOURCE	Age 18-80 years		•	Arm 2: placebo s.c.		baseline in daily OCS dose while not	•	LPCD: Q4 2019
							losing asthma control	•	Data anticipated: H2 2020
	NCT03406078			48 week trial	•	Secondary endpoint: Annual asthma
							exacerbation rate
	Partnered			Global trial - seven countries
	Phase III	Severe asthma	~975	•	Arm 1: tezepelumab s.c.	•	Primary endpoint: Exposure adjusted	•	FPCD: Q1 2019
	DESTINATION	Age 12-80 years		•	Arm 2: placebo s.c.		rates of AEs/SAEs Secondary endpoints:	•	Data anticipated: 2021+
							Annual asthma exacerbation rate
	NCT03706079			Extension Study to NAVIGATOR and SOURCE. 52 week trial
	Partnered			(subjects from NAVIGATOR); 56 week trial (subjects from
			SOURCE)
				Global trial - ~ 20 countries
	Phase III	Severe asthma	216	• Arm 1: tezepelumab s.c. via autoinjector (AI)	Primary endpoint: Proportion of health care	•	FPCD: Q2 2019
	PATH-HOME	Age 12-80 years		• Arm 2: tezepelumab s.c. via accessorized pre-filled syringe	professionals and	•	LPCD: Q3 2019
	NCT03968978				(APFS)	subjects /caregivers who successfully	•	Data anticipated: H2 2020
			24 week trial	administrated tezepelumab in clinic and
	Partnered			at home with an APFS or an AI,
			Global trial - 4 countries
			respectively

Oncology

CVRM

Respiratory

Other

71

Tezepelumab (TSLP mAb)

Severe, uncontrolled asthma

Approved medicines Late-stagedevelopmentEarly development

Oncology

Trial	Population	Patients	Design	Endpoints	Status
Phase II	Severe asthma	116	•	Arm 1: tezepelumab s.c.	•	Primary endpoint: number of airway	•	FPCD: Q4 2018
CASCADE	Age 18-75 years		•	Arm 2: placebo s.c.		submucosal inflammatory cells/mm2 of	•	LPCD: Q4 2019
NCT03688074			28 week trial		bronchoscopic biopsies
Partnered			Global trial - five countries
Phase III	Severe asthma	396	•	Arm 1: tezepelumab s.c.	•	Primary endpoint: Annual asthma	•	FPCD: Q3 2019
DIRECTION	Age 18-80 years		•	Arm 2: placebo s.c.		exacerbation rate
					•	Secondary endpoints: Change from
NCT03927157			52 week trial		baseline in pre-BD FEV1, asthma related
						QoL (AQLQ(S)+12), asthma control
Partnered			Regional Asia study - three countries		(ACQ-6)
Phase III	Severe asthma	66	Arm 1: tezepelumab s.c.	•	Primary endpoint: Number of subjects	•	FPCD: Q2 2019
NOZOMI	12-80 years		52 week trial		with adverse events
NCT04048343			Local study - Japan
Partnered

CVRM

Respiratory

Other

72

								Approved medicines
	Tezepelumab (TSLP mAb)			Late-stage development
			Early development
Atopic dermatitis, COPD
	Trial	Population	Patients	Design	Endpoints	Status
	Phase IIb	Patients with chronic atopic	300	A dose-ranging,double-blind,placebo-controlled study to evaluate	The effect of tezepelumab compared with	•	FPCD: Q1 2019
	NCT03809663	dermatitis		the safety and efficacy of tezepelumab alone or combined with	placebo, assessed using the IGA and EASI	•	Data anticipated: 2021
			topical corticosteroids in moderate-to-severe atopic dermatitis
	Partnered			•	Arm 1: tezepelumab HD, s.c. Q2W
				•	Arm 2: tezepelumab MD, s.c. Q4W
				•	Arm 3: tezepelumab LD, s.c. Q2W
				•	Arm 4: placebo, s.c. Q2W or Q4W
	Phase IIa	Moderate to very severe	282	•	Arm 1: tezepelumab s.c.	• Primary endpoint: Rate of moderate or	•	FPCD Q3 2019
	COURSE	COPD		•	Arm 2: placebo s.c.	severe COPD exacerbations	•	Data anticipated: 2021+
	NCT04039113	Age 40-80		52 week trial
	Partnered			Global trial - 10 countries

Oncology

CVRM

Respiratory

Other

73

								Approved medicines
Anifrolumab (type I interferon receptor mAb)	Late-stage development
Early development
Lupus (SLE / LN)
	Trial	Population	Patients	Design	Endpoints	Status
	Phase III	Moderate to severe SLE	450	•	Arm 1: 300mg i.v. anifrolumab Q4W for 48 weeks	•	Primary endpoint: response in SLE	•	FPCD: Q4 2015
	TULIP SLE 1			•	Arm 2: 150mg i.v. anifrolumab Q4W for 48 weeks		responder index at week 52	•	LPCD: Q4 2017
				•	Arm 3: placebo i.v. Q4W for 48 weeks			•	Data readout: Q3 2018
	NCT02446912							•	Primary endpoint not met
	Phase III	Moderate to severe SLE	360	•	Arm 1: 300mg i.v. anifrolumab Q4W for 48 weeks	•	Primary endpoint: response in SLE	•	FPCD: Q4 2015
	TULIP SLE 2			•	Arm 2: placebo i.v. Q4W for 48 weeks		responder index at week 52	•	LPCD: Q4 2017
							BICLA at week 52	•	Data readout: Q3 2019
	NCT02446899							•	Primary endpoint met
	Phase III	Moderate to severe SLE	630	•	Arm 1: 300mg i.v. anifrolumab Q4W for 152 weeks	•	Primary endpoint: extension to evaluate	•	FPCD: Q2 2016
	TULIP LTE			•	Arm 2: placebo i.v. Q4W for 152 weeks		long-term safety and tolerability	•	LPCD: Q4 2018
	NCT02794285							•	Data anticipated: 2021+
	Phase ll	Moderate to severe SLE	307	•	Arm 1: 300mg i.v. anifrolumab Q4W for 48 weeks	•	Primary endpoint: response in SLE	•	FPCD: Q1 2012
		patients		•	Arm 2: 1000mg i.v. anifrolumab Q4W for 48 weeks		responder index at 6 months	•	LPCD: Q1 2015
	NCT01438489			•	Arm 3: placebo i.v. Q4W for 48 weeks			•	Data readout: Q3 2014
	Phase II	Moderate to severe SLE	218	•	Arm 1: anifrolumab, i.v. Q4W for 104 weeks	•	Primary endpoint: open-label extension	•	FPCD: Q1 2013
		patients					to evaluate long-term safety and	•	Data readout: Q4 2018
	NCT01753193						tolerability
	Phase II​	Moderate to severe SLE	32​	• Arm 1: 150mg s.c. every other week​	• PK/PD, safety, tolerability, primary	• FPCD: Q1 2017​
		patients​		•	Arm 2: 300mg s.c. every other week​		analysis at week 12, secondary analysis	•	LPCD: Q4 2017
	NCT02962960​			• Arm 3: placebo s.c. every other week​​		at week 52​	• Data readout: Q1 2018​
	Phase II	Active Proliferative LN	150	•	Arm 1: 900 mg i.v. Q4W for 12 weeks then 300mg i.v.	•	Response in proteinuria at week 52	•	FPCD: Q4 2015
	TULIP-LN1				anifrolumab Q4W for 36 weeks			•	LPCD: Q4 2018
				•	Arm 2: 300 mg i.v. anifrolumab Q4W for 48 weeks			•	Data anticipated: 2021
	NCT02547922			•	Arm 3: placebo i.v. Q4W for 48 weeks

Oncology

CVRM

Respiratory

Other

74

BioPharmaceuticals - early-stage development

									Approved medicines
Cotadutide (MEDI0382, GLP-1-glucagon agonist)		Late-stage development
	Early development
Diabetes/obesity
	Trial	Population	Patients	Design	Endpoints	Status
	Phase II	Adults with type-2	65	•	Arm1: cotadutide s.c. or placebo	•	Primary: efficacy MMT glucose AUC, body weight loss	•	FPCD: Q3 2017
		diabetes		•	Arm2: cotadutide s.c. or placebo	•	Secondary: efficacy HbA1c, fasting plasma glucose	•	LPCD: Q4 2017
	NCT03244800			•	Germany	•	Secondary: safety profile in terms of adverse events, heart rate, blood	•	Data readout: Q1 2018
						pressure, vital signs, ECG, lab variables
	Phase II	Overweight and	834	•	Arm1: cotadutide low dose s.c. + metformin	•	Primary: efficacy HbA1c, body weight loss	•	FPCD: Q3 2017
		Obese subjects with		•	Arm2: cotadutide mid dose s.c. + metformin	•	Secondary: percentage of subjects achieving weight loss of ≥5% and	•	LPCD: Q1 2018
	NCT03235050	type-2 diabetes		• Arm3: cotadutide high dose s.c. + metformin		≥10%	• Data readout Q3 2019
		•	Arm4: placebo s.c. + metformin	•	Secondary: proportion of subjects rescued or discontinued for lack of
				•	Arm5: liraglutide s.c. + metformin		glycaemic control
				US, Canada, Bulgaria, Czech Rep, Germany,	•	Secondary: PK and immunogenicity
				Mexico, Russia, Slovakia
	Phase II	Overweight/obese	49	•	Arm1: cotadutide + dapagliflozin	•	Primary: efficacy MMT glucose AUC	•	FPCD: Q3 2018
	NCT03444584	subjects with type-2		•	Arm2: placebo + dapagliflozin	•	Secondary: safety	•	LPCD: Q4 2018
		diabetes		•	Germany, Hungary	•	Secondary: PK	•	Data readout: Q1 2019
						•	Secondary: immunogenicity
	Phase II	Adults with type-2	41	•	Cotadutide or placebo s.c.	•	Primary: efficacy MMT glucose AUC	•	FPCD Q2 2018
	NCT03550378	diabetes and renal		•	Germany, UK	•	Secondary: safety	•	LPCD; Q4 2018
		impairment				•	Secondary: tolerability	•	Data readout: Q1 2019
					•	Secondary: PK
						•	Secondary: immunogenicity
	Phase II	Adults with type-2	44	•	Part A: cotadutide or placebo s.c.	•	Primary: change in hepatic glycogen concentration postprandially,	•	FPCD: Q2 2018
	NCT03555994	diabetes		•	Part B: cotadutide s.c. or placebo s.c. or		adjusted by liver volume	•	Part A LPCD: Q4 2018
					liraglutide s.c.	•	Secondary: safety	•	Data readout: Q1 2019
				•	Sweden	•	Secondary: tolerability
						•	Secondary: immunogenicity

Oncology

CVRM

Respiratory

Other

76

									Approved medicines
Cotadutide (MEDI0382, GLP-1-glucagon agonist)		Late-stage development
	Early development
Diabetes/obesity, NASH
	Trial	Population	Patients	Design	Endpoints	Status
	Phase II	Overweight and	27	•	Cotadutide or placebo s.c.	•	Primary: efficacy body weight loss	•	FPCD: Q4 2018
	NCT03596177	obese subjects with		•	UK	•	Secondary: change in total energy intake	•	LPCD: Q4 2019
		type-2 diabetes				•	Secondary: change in total energy expenditure, active energy
							expenditure, resting energy expenditure
						•	Secondary: safety
	Phase I	Non-diabetic obese	51	•	Cotadutide or placebo s.c. with 7 week, 10	•	Primary: safety, tolerability	•	FPCD: Q3 2018
	NCT03625778	subjects			week or 16 week titration period	•	Secondary: PK	•	LPCD: Q2 2019
				•	US	•	Secondary: immunogenicity
	Phase II	Overweight and	20	•	Cotadutide or placebo s.c.	•	Primary: safety, tolerability	•	FPCD: Q1 2019
	NCT03745937	obese subjects with		•	Germany	•	Secondary: PK	•	LPCD: Q2 2019
		type-2 diabetes				•	Secondary: immunogenicity	•	Data readout: Q3 2019
						•	Secondary: glucose control
	Phase II	Japanese preobese	61	•	MAD s.c. 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	•	Data readout: Q2 2019
						•	Secondary: PK, immunogenicity
	Phase II	Obese subjects with	72	•	Arm1: cotadutide high dose s.c.	•	Primary: safety and tolerability	•	FPCD: Q4 2019
	NCT04019561	non-alcoholic fatty		•	Arm2: placebo high dose s.c.	•	Secondary: change in hepatic fat fraction,
		liver disease		•	Arm3: cotadutide low dose s.c.	•	Secondary: change in liver fat volume
		(NAFLD)/non-		•	Arm4: placebo low dose s.c.	•	Secondary: change in visceral adipose tissue
		alcoholic		•	US
		steatohepatitis
		(NASH)
	Phase I	Healthy adult subjects	36			•	Primary: to evaluate exposure following a single s.c of cotadutide at	•	FPCD: Q4 2019
	NCT04091373						each of 3 different sites of injection
						•	Secondary: immunogenicity
						•	Secondary: safety and tolerability

Oncology

CVRM

Respiratory

Other

77

							Approved medicines
Verinurad (RDEA3170, URAT1 inhibitor)	Late-stage development
Early development
CKD
	Trial	Population	Patients	Design	Endpoints	Status
	Phase II	CKD patients with	60	•	Arm A: verinurad 9 mg and febuxostat 80 mg	To assess the effects of intensive uric acid	•	FPCD: Q2 2017
		hyperuricaemia,		•	Arm B: placebo	lowering therapy with RDEA3170 and	•	LPCD: Q3 2018
	NCT03118739	albuminuria, and Type 2		The trial is a multi-centre trial conducted in the US	febuxostat on UACR	•	Data readout: Q4 2018
		diabetes
	Phase II	Asymptomatic hyperuricaemic	36	• Arm A: 9 mg verinurad + 80 mg febuxostat + 10 mg	Primary: Peak uric acid excretion during	•	FPCD: Q4 2017
		subjects (sUA (serum uric acid			dapagliflozin	the first 8 hours) on Day 7 of treatment	•	LPCD: Q3 2018
	NCT03316131	levels) > 6.0 mg/dL)		•	Arm B: 9 mg verinurad + 80 mg febuxostat + placebo	Secondary: serum uric acid levels after 7	•	Data readout: Q4 2019
			The trial is a two-centre trial conducted in the US	days of treatment.
	Phase II	Healthy volunteers of Asian	23	•	Arm A: verinurad 24 mg + allopurinol 300 mg	Safety analyses (AEs, ECG abnormalities,	•	FPCD: Q1 2019
		descent		•	Arm B; verinurad 12 mg + allopurinol 300 mg	vital sign abnormalities, laboratory	•	LPCD: Q2 2019
				•	Arm C: placebo	abnormalities)	•	Data readout: Q3 2019
				This trial is a single centre study conducted in the US	PK outcomes (AUC, Cmax, tmax)

Oncology

CVRM

Respiratory

Other

78

						Approved medicines
AZD2693 (resolution of NASH)		Late-stage development
	Early development
NASH
	Trial	Population	Patients	Design	Endpoints	Status
	Phase I	Healthy subjects	48	SAD	Primary:	•	FPCD: Q4 2019
	NCT04142424			6 cohorts with 6 subjects receiving AZD2693 and 2 subjects	• Safety and tolerability	•	Data anticipated: H2 2020
				receiving placebo in each cohort	Secondary;
				Route of administration: subcutaneous injections	• PK
				Trial conducted in the US.

Oncology

CVRM

Respiratory

Other

79

						Approved medicines
MEDI3506 (IL33 ligand mAb)		Late-stage development
	Early development
Diabetic Kidney Disease (DKD)
	Trial	Population	Patients	Design	Endpoints	Status
	Phase II	Adult subjects with diabetic	168	Randomized, double-blind,placebo-controlled, multicenter study	• Efficacy and safety	• FPCD: Q4 2019
		kidney disease		to evaluate the efficacy, safety, PK, and immunogenicity of
	NCT04170543			MEDI3506 in adult subjects with diabetic kidney disease

Oncology

CVRM

Respiratory

Other

80

							Approved medicines
AZD4831 (MPO inhibitor)			Late-stage development
		Early development
Cardiovascular disease
	Trial	Population	Patients	Design	Endpoints	Status
	Phase I	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
	NCT02712372			up to 10 cohort may be used			•	Data readout Q2 2017
	Phase I	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
	NCT03136991			may be included			•	Data readout: Q1 2018
	Phase IIa	HFpEF	96	Arm 1: AZD4831	•	Primary endpoint: The change from	•	FPCD: Q4 2018
				Arm 2: placebo		baseline in MPO activity in % after
	NCT03756285					AZD4831 treatment
				Global trial - five countries

Oncology

CVRM

Respiratory

Other

81

								Approved medicines
AZD5718 (FLAP inhibitor)			Late-stage development
		Early development
Cardiovascular disease
	Trial	Population	Patients	Design	Endpoints	Status
	Phase IIa	CAD	138	•	Arm 1: AZD5718 Dose A	•	Primary endpoint: PD effect of AZD5718	•	FPCD: Q4 2017
	NCT03317002			•	Arm 2: AZD5718 Dose B		by assessment of u-LTE4
			•	Arm 3: placebo
				Global trial - three countries in Europe
	Phase I	Healthy subjects	6	hADME trial (one trial site in UK)	•	Mass balance, with routes and rates of	•	FPCD: Q2 2019
	NCT03948451			•	Oral administration		elimination of [14C]AZD5718.	•	LPCD: Q2 2019
			Open-label study to characterize the absorption, distribution,	•	Metabolite profiling and structural
				metabolism and excretion following a single oral dose of		identification
				[14C]AZD5718 in healthy male volunteers	•	PK and total radioactivity
	Phase I	Healthy subjects	14	BA trial (one trial site in UK)	•	To evaluate the pharmacokinetics and	•	FPCD: Q4 2019
	NCT04087187			An open-label, randomized, 3-period,3-treatment, crossover		exposure of 3 different doses	•	LPCD: Q4 2019
			study to assess the drug absorption into the blood after		of AZD5718
				administration of 3 doses of AZD5718	•	Safety and tolerability

Oncology

CVRM

Respiratory

Other

82

						Approved medicines
AZD6615 (anti-hypercholesterolemia)		Late-stage development
	Early development
Hypercholesterolemia
	Trial	Population	Patients	Design	Endpoints	Status
	Phase I	Healthy subjects	40	SAD	Primary:	• FPCD: Q3 2019
	NCT04055168			3 cohorts of non-Asian subjects (Part 1) and 2 cohorts of	• Safety and tolerability
				Japanese subjects (Part 2). 6 subjects receiving AZD6615 and	Secondary;
				2 subjects receiving placebo in each cohort.	• PK and PD parameters
				Trial conducted in the US.

Oncology

CVRM

Respiratory

Other

83

						Approved medicines
MEDI7219 (anti-diabetic)		Late-stage development
	Early development
Diabetes
	Trial	Population	Patients	Design	Endpoints	Status
	Phase I	Healthy Volunteers	130	• 5 part trial	• Safety and tolerability	• FPCD: Q1 2018
				• Part A : SAD	• Pharmacokinetics	• Data anticipated: H1 2020
	NCT03362593			• Part B, C & E : open label, single dose studies
				• Part D : MAD

Oncology

CVRM

Respiratory

Other

84

						Approved medicines
AZD8233 (anti-hypercholesterolemia)		Late-stage development
	Early development
Hypercholesterolemia
	Trial	Population	Patients	Design	Endpoints	Status
	Phase I	Healthy subjects	56	SAD	Primary:	• FPCD: Q3 2018
	NCT03593785			7 cohorts with 6 subjects receiving AZD8233 and 2 subjects	• Safety and tolerability	• LPCD: Q3 2019
				receiving placebo in each cohort	Secondary;
				Trial conducted in the US.	• PK and PD parameters

Oncology

CVRM

Respiratory

Other

85

								Approved medicines
AZD8601 (VEGF-A modified RNA)			Late-stage development
		Early development
Cardiovascular disease
	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
	Phase IIa	HF	Up to 33	Phase IIa trial (two trial sites in Finland)	•	Safety and tolerability	•	FPCD: Q1 2018
	NCTT03370887			•	Arm 1: AZD8601 Dose A
			•	Arm 2: AZD 8601 Dose B
				•	Arm 3: placebo

Oncology

CVRM

Respiratory

Other

86

							Approved medicines
AZD9977					Late-stage development
				Early development
Heart failure with preserved ejection fraction
	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;
				Trial conducted in the UK.	•	PK parameters
	Phase I	Healthy subjects	12	Bioavailability trial	Primary:	•	FPCD: Q2 2018
					•	relative bioavailability vs. oral suspension	•	LPCD: Q2 2018
	NCT03450759			Investigation of four different oral formulations of AZD9977 and		(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
	NCT03682497			To compare the effect of AZD9977 with spironolactone on serum	•	serum potassium	•	LPCD Q1 2019
				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			•	Data readout: Q3 2019
				AZD9977	Secondary;
				Trial conducted in the US	•	Safety and tolerability
	Phase I	Healthy subjects	45	JSMAD	Primary:	•	FPCD: Q1 2019
					•	Safety and tolerability	•	LPCD: Q2 2019
	NCT03801967			Single and multiple-ascending dose administration in Japanese			•	Data readout: Q3 2019
				healthy volunteers.	Secondary;
				Trial conducted in the UK	•	PK parameters
	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)	•	Data readout: Q3 2019
				influence of food.	•	PK parameters
				Trial conducted in the UK

Oncology

CVRM

Respiratory

Other

87

									Approved medicines
Biologics							Late-stage development
						Early development
Cardiovascular & metabolic diseases
	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
	EudraCT 2017-	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: 2021+
		inclusive,		(loading dose) prior to pPCI followed by a	compared to placebo
	004521-32		presenting with
			second inpatient dose	Secondary endpoints:
			acute STEMI
				of 150 mg or placebo on Day 3 by i.v.	• 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 day	• Incidence of TEAEs and treatment-emergent SAEs.
					3 and outpatient maintenance doses of 100	• LCAT mass and ADAs
					mg or placebo on days 10, 17, 24,
					and 31 by i.v. push.
	Phase IIa	MEDI5884	Adults with stable	133	• MEDI5884 (5 dose cohorts) vs. placebo in	•	Safety profile in terms of AEs, vital signs, ECG, lab variables	•	FPCD Q4 2017
		cholesterol	CHD			stable CHD patients	•	Changes in HDL-C over time	•	Data readout: Q4 2018
	NCT03351738	modulation					•	PK, immunogenicity, and Apolipoprotein B
	Phase I	MEDI6570	Atherosclerotic	88	•	SAD followed by multi ascending dose	•	Primary endpoints: Safety and tolerability	•	FPCD: Q4 2018
			cardiovascular			with 3 monthly doses in T2DM subjects			•	Data anticipated: 2021
	NCT03654313		disease

Oncology

CVRM

Respiratory

Other

88

							Approved medicines
	AZD0449 (inhaled JAK-1 inhibitor)			Late-stage development
			Early development
Asthma
	Trial	Population	Patients	Design	Endpoints	Status
	Phase I	Healthy subjects and patients	156	SAD/MAD/Bridge trial (UK)	Primary endpoint:	• FPCD: Q4 2018
		with mild asthma		Part 1 SAD	•	Safety and tolerability
	NCT03766399			• Dose escalation in 6 cohorts with 6 subjects receiving
				AZD0449 and 2 subjects receiving placebo in each cohort	Secondary endpoint:
				• i.v. cohort with 8 subjects	•	PK parameters
				Part 2 MAD:	•	FENO
				• 3 cohorts of (6, 6, 18) subjects receiving three different doses
				of AZD0449 and (3,3, 12) subjects receiving placebo in each
				cohort
				Part 3 bridge
				• 18 subjects will receive AZD0449 and 6 subjects receiving
				placebo
				Trial conducted in the UK

Oncology

CVRM

Respiratory

Other

89

							Approved medicines
AZD1402 (IL4 receptor antagonist)			Late-stage development
		Early development
Asthma
	Trial	Population	Patients	Design	Endpoints	Status
	Phase Ib	Patients with mild asthma	75	PoM.	Primary endpoint:	• FPCD: Q3 2018
				A dose-escalating, single blind trial to assess the	•	Safety and tolerability
	NCT03574805			safety, tolerability, and pharmacokinetics of multiple
				doses of PRS-060 administered by oral Inhalation In	Secondary endpoint:
	Partnered			subjects with mild asthma	•	PK parameters
					•	Potential immunogenicity
				Australia	•	Change in FENO

Oncology

CVRM

Respiratory

Other

90

							Approved medicines
MEDI3506 (IL33 ligand mAb)			Late-stage development
		Early development
COPD, atopic dermatitis
	Trial	Population	Patients	Design	Endpoints	Status
	Phase I (Combined SAD /	SAD: healthy subjects with	SAD: 56	SAD:	•	Safety and tolerability	•	FPCD: Q2 2017
	MAD)	mild atopy		• 7 sequential placebo-controlled single dose cohorts by either			•	LPCD: Q2 2019
				SC or IV route (active N=6 / placebo N = 2 within each			•	Data anticipated: H1 2020
	NCT03096795			cohort)
		J-SD: healthy Japanese	J-SD: 8
		subjects		J-SD:
				• single placebo-controlled single dose cohort by IV route
				(active N=6 / placebo N = 2 within cohort)
		MAD: GOLD I-II COPD	MAD: 24
				MAD:
				• 3 sequential placebo-controlled multiple dosing cohorts by SC
				route (active N=6 / placebo N = 2 within each cohort)
				Conducted in UK
	Phase II	Adult subjects with atopic	108	Randomised, blinded, placebo-controlled trial to determine the	•	Efficacy and safety	•	FPCD: Q4 2019
		dermatitis		efficacy and safety of different strengths of MEDI3506 by SC
	NCT04212169			route
				Conducted in US, Australia, Germany & Poland

Oncology

CVRM

Respiratory

Other

91

							Approved medicines
AZD7594 (SGRM, inhaled)			Late-stage development
		Early development
Asthma
	Trial	Population	Patients	Design	Endpoints	Status
	Phase I	Adolescent asthma patients	24	An open-label,multi-centre, Phase I study to assess the PK, PD	Primary endpoint:	•	FPCD: Q3 2019
				and safety of 2-eeek treatment with inhaled AZD7594 in	•	PK, safety and tolerability following 2	•	Data readout: H1 2020
	NCT03976869			adolescents (12 to 17 Years) with asthma		weeks treatment with AZD7594
					Secondary endpoints
					•	Changes from baseline in lung function,
						asthma control and plasma cortisol on
						day 15

Oncology

CVRM

Respiratory

Other

92

							Approved medicines
AZD7986 (DPP1)				Late-stage development
			Early development
COPD
	Trial	Population	Patients	Design	Endpoints	Status
	Phase I	Healthy volunteers	15	This is a phase I, non-randomised, fixed sequence, 3-period,	•	Safety and tolerability	•	FPCD: Q1 2016
	NCT02653872			drug-drug interaction study to assess the PK of AZD7986 in	•	PK/PD and DDI	•	Data readout: Q2 2016
			healthy subjects when administered alone and in combination
				with multiple doses of verapamil and itraconazole or diltiazem
				• Arm 1: AZD7986 (alone) treatment period 1
				• Arm 2: verapamil (with AZD7986) treatment period 2
				• Arm 3: itraconazole (with AZD7986) treatment Period 3
				• Arm 4: diltiazem (with AZD7986) treatment period 3
	Phase I	Healthy volunteers	89	A phase I, randomised, single-blind,placebo-controlled,2-part	•	Safety and tolerability	•	FPCD: Q4 2014
	NCT02303574			study to assess the safety, tolerability, PK and food effect of	•	PK/PD	•	Data readout: Q3 2016
			single and multiple oral doses of AZD7986 in healthy volunteers.	•	Bioavailability
				• Arm 1: AZD7986, single and multiple oral doses
				• Arm 2: placebo, single and multiple doses

Oncology

CVRM

Respiratory

Other

93

						Approved medicines
AZD8154 (PI3Kγδinhibitor)		Late-stage development
	Early development
Asthma
	Trial	Population	Patients	Design	Endpoints	Status
	Phase I	Healthy subjects	78	SAD/MAD	Primary endpoint:	•	FPCD: Q3 2018
				A Phase I trial to assess the safety, tolerability and PK of	• Safety and tolerability	•	LPCD: Q3 2019
	NCT03436316			AZD8154 following single dose administration and multiple dose		•	Data readout: Q4 2019
				administration in healthy subjects	Secondary endpoint:
					• PK parameters

Oncology

CVRM

Respiratory

Other

94

							Approved medicines
AZD8871 (MABA, inhaled)			Late-stage development
		Early development
Respiratory
	Trial	Population	Patients	Design	Endpoints	Status
	Phase IIa	Patients with COPD	73	Randomised, double-blind, placebo and active-controlled	Primary endpoint:	•	FPCD: Q4 2018
	NCT03645434			crossover trial. Eligible patients will be randomised in 1:1:1:1:1:1	•	Change from baseline in trough FEV1on	•	LPCD: Q2 2019
			ratio to 1 of 6 treatment sequences and will receive 1 of the		day 15	•	Data anticipated: Q3 2019
				Secondary endpoints:
				following 3 treatments sequence in the form of dry powder
				•	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

Oncology

CVRM

Respiratory

Other

95

							Approved medicines
AZD9567 (SGRM, oral)			Late-stage development
		Early development
Respiratory
	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
						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
	Phase IIa	Patients with active RA	40	A randomised, double-blind, parallel trial to assess the efficacy,	Primary endpoint:	•	FPCD: Q1 2018
	NCT03368235			safety and tolerability of AZD9567 compared to prednisolone 20	To assess the efficacy of AZD9567, 40 mg,
				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 s.c./i.v.
					biological DMARDs (Disease-modifying
					antirheumatic drugs)
					Secondary endpoints:
					• 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

Oncology

CVRM

Respiratory

Other

96

							Approved medicines
AZD0284 (RORγinverse agonist)			Late-stage development
		Early development
Plaque psoriasis vulgaris
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
						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	•	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 single centre, open-label,non-randomised, single dose trial	•	Determination of absolute bioavailability	•	FPCD: Q1 2017
			performed in 6 healthy male subjects aged 18 to 65 years,		of AZD0284	•	LPCD: Q1 2017
NCT03029741			inclusive. The trial will assess the absolute bioavailability of a	•	Safety and tolerability of AZD0284
			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 plaque	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 Ib study, designed to evaluate the		weeks treatment, in gene expression	•	LPCD: Q2 2018
NCT03310320		9 dosed	pharmacodynamic effects, clinical efficacy and safety of		level of IL-17A and CCL20 relative to
			AZD0284 compared with placebo as measured by the relative		placebo
			change from baseline in Psoriasis Area Severity Index (PASI	•	Change (percent improvement) in PASI	•	The trial was temporarily suspended ~5
			score), other disease assessments of involved body surface area		compared to placebo		months due to preclinical findings.
			(BSA), static physicians global assessment score (sPGA), pruritis	•	Safety and tolerability and PK following 4
			and biomarkers associated with the mechanism of disease and		weeks oral administration with single	•	However, whilst the intention was to re-
			AZD0284		ascending dose		open the DERMIS study, in the
								meantime, and for portfolio and
								prioritisation reasons, a decision was
								taken in Q3 2018 to end the study.

Oncology

CVRM

Respiratory

Other

97

						Approved medicines
MEDI0618 (PAR2 antagonist mAb)	Late-stage development
Early development
Osteoarthritis pain
	Trial	Population	Patients	Design	Endpoints	Status
	Phase I	Painful osteoarthritis of the knee	64 (healthy	• SAD	• Safety, tolerability and PK	• FPCD: Q4 2019
			volunteers)	• Up to 8	i.v. cohorts are planned vs. placebo
	NCT02508155			• 1 s.c. cohortis planned vs. placebo
				Europe only

Oncology

CVRM

Respiratory

Other

98

						Approved medicines
MEDI1341 (alpha-synuclein mAb)		Late-stage development
	Early development
Parkinson's Disease
	Trial	Population	Patients	Design	Endpoints	Status
	Phase I	Healthy volunteers	48	• SAD	• Safety, tolerability, PK, PD	• FPCD: Q4 2017
	NCT03272165			• Up to 6 i.v. cohorts are planned vs. placebo		• Data anticipated: H2 2020
			US only

Oncology

CVRM

Respiratory

Other

99

						Approved medicines
AZD4041 (orexin 1 receptor antagonist)		Late-stage development
	Early development
Opioid use disorder
	Trial	Population	Patients	Design	Endpoints	Status
	Phase I	Healthy volunteers	48 healthy	• Randomised, double blind, single ascending dose	• Safety, tolerability, PK, PD	• FPCD: Q4 2019
	NCT04076540		volunteers	• Up to 6 cohorts are planned vs. placebo		• Data anticipated: H2 2020
			Single centre in US only
	Partnered with Eolas
	Therapeutics Inc
	and NIH.

Oncology

CVRM

Respiratory

Other

100

							Approved medicines
AZD5634 (ENaC, inhaled)			Late-stage development
		Early development
Cystic Fibrosis
	Trial	Population	Patients	Design	Endpoints	Status
	Phase I	Healthy volunteers	56	A randomised, single-blind,placebo-controlled trial to assess the	•	Safety and tolerability	•	FPCD: Q1 2016
	NCT02679729			safety, tolerability and pharmacokinetics of AZD5634 following	•	PK/PD	•	Data readout: Q4 2016
			single-ascending inhaled doses (Part A) and after single inhaled
				and intravenous doses (Part B) in healthy subjects
				• Arm 1: AZD5634 following inhaled administration of SAD
				(Part A) and following administration of single inhaled and i.v.
				doses (Part B)
				• Arm 2: placebo
	Phase Ib	Patients with cystic fibrosis	9	A randomised blinded placebo-controlled,cross-over trial to	•	Safety and tolerability	•	FPCD: Q2 2017
	NCT02679729			assess the effect of AZD5634 on mucociliary clearance as well as	•	PK/PD	•	Data readout: Q2 2018
			safety, tolerability, and PK parameters following single inhaled
				dose administration to patients with cystic fibrosis
				• Arm 1: subjects were administered single dose of placebo in
				period 1 and AZD5634 in period 2
				• Arm 2: subjects were administered single dose of AZD5634 in
				period 1 and placebo in period 2

Oncology

CVRM

Respiratory

Other

101

									Approved medicines
MEDI7352 (NGF TNF bispecific mAb)				Late-stage development
			Early development
Osteoarthritis pain
	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 12 i.v. cohorts are planned vs. placebo			•	Data anticipated: H1 2020
	NCT02508155			•	1 s.c. cohorts are planned vs. placebo
				Europe only
	Phase II	Painful diabetic neuropathy	271	•	Multiple dose study	•	Dose response, safety, tolerability, PK,	•	FPCD Q4 2018
	NCT03755934			• Up to 4 i.v. cohorts are planned vs. placebo		PD	•	Data anticipated: 2021
				Europe only

Oncology

CVRM

Respiratory

Other

102

									Approved medicines
Other biologics						Late-stage development
					Early development
Infections
	Trial	Compound	Population	Patients	Design	Endpoints	Status
	Phase II	Anti-Staph AT	Intubated ICU	213	•	Placebo-controlled,single-dose,dose-ranging	•	Efficacy and safety	•	FPCD: Q4 2014
		(suvratoxumab,			•	Route of administration: intravenous			•	Data readout: Q4 2018
	EudraCT 2014-	MEDI4893)
	001097-34
	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: intramuscular			•	Data readout: Q4 2018
	NCT02878330	mAb-YTE	infants
		nirsevimab
		(MEDI8897)
	Phase II	Anti-Pseudomonas	Intubated ICU	195	•	Placebo-controlled,single-dose,dose-ranging	•	Efficacy and safety	•	FPCD: Q2 2016
	NCT02696902	A mAb (MEDI3902)			•	Route of administration: intravenous			•	Data anticipated: H2 2020

Oncology

CVRM

Respiratory

Other

103

List of abbreviations

14C	Radioactive isotope of carbon, Carbon 14	CHF	Chronic heart failure	FLAP	5-lipoxygenase-activating protein
1L, 2L, 3L	1st, 2nd or 3rd line	CKD	Chronic kidney disease	FPDC	First patient commenced dosing
5-FU	5-fluorouracil	CLL	Chronic lymphocytic leukaemia	FPG	Fasting plasma glucose
A2AR	Adenosine A2A receptor	CMAX	Maximum observed plasma concentration	GA	Gestational age
ACQ	Asthma control questionnaire	C-MET	Tyrosine-protein kinase Met	GBM	Glioblastoma
ACR	American college of rheumatology response scoring system	CNS	Central nervous system	gBRCAm or
ADA	Anti-drug antibodies	COPD	Chronic obstructive pulmonary disease	tBRCAm	Germline or tumour BRCA mutation somatic
ADC	Antibody-drug conjugate	CR	Complete response	GEJ	Gastric/gastro-oesophageal junction
ADP	Adenosine diphosphate	CRC	Colorectal cancer	GFF	Glycopyrronium and formoterol fumarate
AE	Adverse Event	CrCl	Creatinine clearance	GLP-1	Glucagon-likepeptide-1
AI	Auto-injector	CRR	Complete response rate	GMFRs	Geometric mean fold rises
AKT	Protein kinase B	CTC	Circulating tumour cell	GMTs	Geometric mean titers
ALK	Anaplastic large-cell lymphoma kinase	CTLA-4	Cytotoxic T-lymphocyte-associated antigen 4	HAI	Haemagglutination-inhibition
APFS	Accessorised pre-filled syringe	CV	Cardiovascular	HbA1c	Hemoglobin A1c
AQLQ	Asthma quality of life questionnaire	CVOT	Cardiovascular outcomes trial	HCC	Hepatocellular carcinoma
AS	Albuterol sulphate	CVRM	Cardiovascular renal and metabolism	HD	High dose
ATM	Ataxia-telangiectasia mutated kinase	CXCR2	C-X-C Motif chemokine receptor 2	HDL-C	High-density lipoprotein cholesterol
ATR	Ataxia telangiectasia and rad3-related protein	DB	Double blind	HER2	Human epidermal growth factor receptor 2
AUC	Area under curve	DC	Disease control	HF	Heart failure
B7RP	B7-relatedprotein-1	DCR	Disease control rate	HFpEF	Heart failure with preserved ejection fraction
BA	Bioavailability	DDI	Drug-drug Interaction	HFrEF	Heart failure with reduced ejection fraction
BAFF	B-cell activating factor	dECG	Differentiated electrocardiogram	HGFR	Met/hepatocyte growth factor receptor
BCG	Bacillus Calmette-Guérin	DFS	Disease free survival	HGSC	High grade serous carcinoma
BCMA	B-cell maturation antigen	DLBCL	Diffuse large B-cell lymphoma	hHF	Hospitalisation for heart failure
BDA	Budesonide albuterol	DLT	Dose-limiting toxicity	HIF-PHI	Hypoxia inducible factor - prolyl hydroxylase inhibitor
BFF	Budesonide and formoterol fumarate	DMARDs	Disease-modifying antirheumatic drugs	HNSCC	Head and neck squamous-cell carcinoma
BGF	Budesonide, glycopyrronium and formoterol fumarate	DNA	Deoxyribonucleic acid	HPV	Human papillomavirus
BICR	Blinded independent central review	DoCR	Durability of complete response	HRD	Homologous recombination deficiency
BID	Bis in die (twice per day)	DoR	Duration of response	HRRm	Homologous recombination repair mutation
BIG	Big ten cancer research consortium	DPI	Dry powder inhaler	i	inhibitor
BMD	Bone mineral density	DXA	Dual energy X-ray absorptiometry	IA	Investigator-assessed
BMI	Body mass index	EBRT	External beam radiation therapy	ICS	Inhaled corticosteroid
BRCAwt	Breast cancer wild-type gene	ECG	Electrocardiogram	ICU	Intensive care unit
BRD4	Bromodomain-containing protein 4	EFS	Event-free survival	IDFS	Invasive disease-free survival
BTC	Biliary tract carcinoma	eGFR	Estimated glomerular filtration rate	IL	Interleukin
BTK	Bruton's tyrosine kinase	EGFR	Epidermal growth factor receptor	i.m.	Intramuscular
CA-125	Cancer antigen 125	ER	Oestrogen receptor	IRC	Independent review committee
CAD	Coronary artery disease	ERK	Extracellular signal-regulated kinase	ISS	Investigator-sponsored studies
CBR	Clinical benefit rate	ESR	Externally sponsored study	i.v.	Intraveneous
CCL20	Chemokine (C-C motif) ligand 20	ESR1	Oestrogen receptor 1	J-SD	Japanese single dose
CD	Cluster of differentiation	ESSC	Esophageal squamous cell carcinoma	Ki67	Protein that is encoded by the MKI67 gene in human
CDK	Cyclin-dependent kinase	FDC	Fixed-dose combination
CE	Clinically evaluable	FeNO	Fractional nitric oxide concentration in exhaled breath
CHD	Coronary heart disease	FEV	Forced-expiratory volume
Chemo	Chemotherapy	FGFR	Fibroblast growth factor receptor

104

List of abbreviations

LABA	Long acting beta agonist	PASI	Psoriasis area severity index	SAE	Serious adverse event
LAMA	Long acting muscarinic agonist	PBD	Pyrrolobenzodiazepine	SBRT	Stereotactic body radiation therapy
LCAT	Lecithin-cholesterol acyltransferase	pCR	Pathological complete response	s.c.	Subcutaneous
LCM	Lifecycle management	PD	Pharmacodynamics	SCLC	Small cell lung cancer
LN	Lupus nephritis	PD-1	Programmed cell death protein 1	SD	Stable disease
LOCS III	Lens opacities classification system III	PDAC	Pancreatic ductal adenocarcinoma	SGLT2	Sodium-glucose transport protein 2
LPCD	Last patient commenced dosing	PDE4	Phosphodiesterase type 4	SGRM	Selective glucocorticoid receptor modulator
LV	Left ventricle	PD-L1	Programmed death-ligand 1	SGRQ	Saint George respiratory questionnaire
m	Mutation	PET	Positron-emission tomography	SJC	Swollen joint count
mAb	Monoclonal antibody	PFS	Progression free survival	SLE	Systemic lupus erythematosus
MABA	Muscarinic antagonist-beta2 agonist	PgR	Progesterone receptor	SLL	Small lymphocytic lymphoma
MACE	Major adverse cardiac events	PI3K	Phosphoinositide 3-kinase	SMAD	Single and multiple ascending dose trial
MAD	Multiple ascending dose	PIK3CA	Phosphatidylinositol 3 kinase catalytic alpha gene	SoC	Standard of care
MCC	Mucociliary clearance	PK	Pharmacokinetics	sPGA	Static physicians global assessment score
MCL	Mantle cell lymphoma	PLL	Prolymphocytic leukaemia	STAT3	Signal transducer and activator of transcription 3
MCL1	Myeloid leukemia cell differentiation protein 1	pMDI	Pressurised metered dose inhaler	sUA	Serum uric acid
mCRPC	Metastatic castrate-resistant prostate carcinoma	PN	Plexiform neurofibromas	T2DM	Type 2 Diabetes Mellitus
MD	Medium dose	POC	Proof of concept	T790M	Threonine 790 substitution with methionine
MDI	Metered-dose inhaler	POM	Proof of mechanism	TACE	Transarterial Chemoembolization
MDS	Myelodysplastic syndrome	pPCI	Primary percutaneous coronary intervention	TEAEs	Treatment-emergent adverse events
MEK	Mitogen-activated protein kinase	PR	Partial response	TID	Ter in die (three times a day)
MET	Tyrosine-protein kinase Met	pre-BD	Pre-bronchodilator	TJC	Tender joint count
MI	Myocardial infarction	PRO	Patient reported outcome	TKI	Tyrosine kinase Inhibitor
MMT	Mixed meal test	PRR	Recurrent platinum resistant	TLR	Toll-like receptor 9
MPO	Myeloperoxidase	PS	Propensity score	TNBC	Triple negative breast cancer
mPR	Major pathological response	PSA	Prostate-specific antigen	TNF	Tumour necrosis factor
MRI	Magnetic resonance imaging	PSC	Pulmonary sarcomatoid carcinoma	TSLP	Thymic stromal lymphopoietin
MTD	Maximum tolerated dose	PSMA	Prostate-specific membrane antigen	TTF	Time to treatment failure
NaC	Sodium channel	PTEN	Phosphatase and tensin homolog gene	TTNT	Time to next therapy
NCI	National cancer institute (US)	Q2,3,4,8W	Quaque (every) two, three... weeks	TTP	Time to tumour progression
NCPV	Noncalcified plaque volume	QD	Quaque in die (once a day)	UACR	Urine albumin creatinine ratio
NF1	Neurofibromatosis type 1	QID	Quarter in die (four times a day)	UMEC	Umeclidinium
NGF	Nerve growth factor	QOD	Quaque altera die (every other day)	URAT1	Uric Acid Transporter 1
NHL	Non-Hodgkin's lymphoma	QoL	Quality of Life	VEGF	Vascular endothelial growth factor
NIH	National Institute of Health (US)	QTcF	Corrected QT interval by Fredericia	YTE	Triple-amino-acid (M252Y/S254T/T256E [YTE]) substitution
NKG2a	Natural killer cell C-type lectin receptor G2A	RA	Rheumatoid Arthritis
NME	New molecular entity	RAAS	Renin-angiotensin-aldosterone system
NRG	National clinical trials network in oncology (US)	RECIST	Response evaluation criteria in solid tumours
NSCLC	Non-small cell lung cancer	RFS	Relapse-free survival
OCS	Oral corticosteroid	rhLCAT	Recombinant human Lecithin-cholesterol acyltransferase
OD	Once daily	RORγ	Related orphan receptor gamma
OGTT	Oral glucose tolerance test	r/r	Relapsed/refractory
ORR	Objective response rate	RT	Radiation therapy
OS	Overall survival	SABA	Short-actingbeta2-agonist
PARP	Poly ADP ribose polymerase	SAD	Single ascending dose

105

Clinical trials appendix FY 2019 results update