阿斯利康 2024年年度報告-臨床研究附錄-Clinical Trials Appendix FY 2024 Results Update

ClinicalTrials

Appendix

FY2024ResultsUpdate

6February2025

191

19

Pipeline

projectsinour

developmentpipeline

newmolecularentities(NME)inourlate-stagepipeline

ataglance

130

newmolecularentities

(NME)ormajorlifecycle

management(LCM)projectsinPhaseIIandPhaseIII

31

regulatoryapprovalsinmajormarkets

sinceFY2023

2

Acrossfivefocustherapyareas:

Oncology

BioPharmaceuticals

CVRM|R&I|V&I

RareDisease

Asof6February2025.Appendix:

Glossary

.

Keyupcomingpipelinecatalysts:2025and2026

OncologyBioPharmaceuticalsRareDisease

H22025

2026

H12025

Calquence-MCL(1L)(ECHO)(EU,JP)

Calquence-CLL(1Lfixedduration)(AMPLIFY)

Imfinzi-early-stageNSCLC(perioperative)(AEGEAN)(JP)Imfinzi-limited-stageSCLC(ADRIATIC)(EU,CN)

Enhertu-HER2-lowandultralowmet.breastcancer(DESTINY-Breast06)(JP)Truqap-HR+HER2-met.breastcancer(2L)(CAPItello-291)(CN)

Datroway-HR+HER2-breastcancer(2L+)(TROPION-Breast01)(CN)Datroway-EGFRmNSCLC(laterline)(TROPION-Lung05)

Ultomiris-NMOSD(CHAMPION-NMOSD)(CN)Koselugo-adultNF1-PN(KOMET)

Imfinzi+Imjudo-NSCLC(1L)(POSEIDON)(CN)Tezspire-severeasthma(DIRECTION)(CN)

Wainua-ATTRv-PN(NEURO-TTRansform)(CN)

Tagrisso-EGFRmNSCLC(unresectable,Stg.III)(LAURA)(JP)Calquence-CLL(1L,treat-to-progression)(ELEVATE-TN)(CN)Imfinzi-early-stageNSCLC(perioperative)(AEGEAN)(EU,CN)Imfinzi-limited-stageSCLC(ADRIATIC)(JP)

Imfinzi-muscle-invasivebladdercancer(NIAGARA)

Regulatorydecision1,2

Enhertu-HER2-lowand-ultralowmet.breastcancer(DESTINY-Breast06)(EU)Datroway-HR+HER2-met.breastcancer(2L+)(TROPION-Breast01)(EU)

Wainua-ATTRv-PN(NEURO-TTRansform)(EU)Ultomiris-gMG(CHAMPION-MG)(CN)

acoramidis-ATTR-CM(ALXN2060-TAC-302)(JP)

KeyPhaseIIIdatareadouts

Enhertu-high-riskHER2+earlybreastcancer(neoadj.)

(DESTINY-Breast11

)Datroway-met.TNBC(

TROPION-Breast02

)

Breztri-severeasthma

(KALOS/LOGOS

)

eneboparatide-hypoparathyroidism

(CALYPSO

)

Imfinzi-earlyHCC

(EMERALD-2)

Imfinzi-locoregionalHCC

(EMERALD-3)

Truqap-mCRPC

(CAPItello-280)

Datroway-NSQNSCLC(1L)

(TROPION-Lung07)

Datroway+Tagrisso-EGFRm

NSCLC(2L)(TROPION-Lung15)

Datroway-PD-L1+met.TNBC(1L)

(TROPION-Breast05)

camizestrant-HR+HER2-met.breastcancer(1L)

(SERENA-4)

AZD0901-CLDN18.2+gastriccancer(2L+)

(CLARITY-Gastric01)

Saphnelo-lupusnephritis

(IRIS)

Saphnelo-systemicsclerosis(DAISY)

Wainua-ATTR-CM

(CARDIO-TTRansform)

tozorakimab-COPD

(OBERON/TITANIA)

tozorakimab-COPD

(MIRANDA)

tozorakimab-LRTD(TILIA)

efzimfotasealfa-hypophosphatasia

(MULBERRY)

Tagrisso+Orpathys-EGFRmNSCLC(

SAFFRON

)Imfinzi-resectableGC/GEJC(

MATTERHORN

)

Imfinzi-non-muscle-invasivebladdercancer(

POTOMAC

)Imfinzi-muscle-invasivebladdercancer(

VOLGA

)

Enhertu-high-riskearlyHER2+breastcancer(adjuvant)

(DESTINY-Breast05

)Enhertu-HER2+met.breastcancer(1L)(

DESTINY-Breast09

)

Datroway+Imfinzi-Non-squamous/Non-squamousTROP2+NSCLC(1L)

(AVANZAR

)camizestrant-ESR1mHR+HER2-met.breastcancer(1Lswitch)

(SERENA-6

)

ceralasertib-post-IONSCLC(

LATIFY

)

Fasenra-moderatetosevereCOPD(

RESOLUTE

)Saphnelo-moderatetosevereSLE(

TULIP-SC

)

baxdrostat-uncontrolledhypertension

(BaxHTN

)Ultomiris-HSCT-TMA(

ALXN1210-TM-313/-314

)

anselamimab-ALamyloidosis(MayoStg.IIIa/b)

(CAEL101-302)/CAEL101-301)

gefurulimab-myastheniagravis

(ALXN1720-MG-301)

efzimfotasealfa-hypophosphatasia

(HICKORY/CHESTNUT)

Keyupcomingpipelinecatalystsaredefinedbyathresholdofnon-riskadjustedglobalpeakyearrevenueexpectationsasof6February2025

1Regulatorydecisionincludesprogrammesunderreviewinamajormarket

3

2Inclusiondependentonstatusofregulatorysubmissionand/orsubmissionacceptanceinregionsinwhichsubmissionacceptanceisgrantedAsof6February2025.

Appendix:

Glossary

.

ClinicalTrialsAppendix:selectedhighlights

Oncology

RareDisease

BioPharmaceuticals

Next-wavepipeline:Approvedmedicines:

registrationalstudiesongoingkeyLCM

balcinrenone/dapagliflozin(MRM/SGLT2)

baxdrostat(aldosteronesynthaseinhibitor)

baxdrostat/dapagliflozin(ASI/SGLT2)

zibotentan/dapagliflozin(ETAreceptorantagonist/SGLT2)

tozorakimab(IL-33ligandmAb)

ALXN2220(TTRdepleter)

efzimfotasealfa(enzymereplacementtherapy)

eneboparatide(PTH1agonist)

gefurulimab(C5inhibitor)

camizestrant(nextgenerationoralSERD)

saruparib(PARP1inhibitor)

rilvegostomig(PD-1/TIGITbispecific)

volrustomig(PD-1/CTLA-4bispecific)

AZD0901(CLDN18.2ADC)

AZD0486(CD19/CD3TCE)

4

Asof06February2025.Appendix:

Glossary

.

ProjectmovementssinceQ32024update

NewtoPhaseII

Newtopivotaltrial

Newtoregistration

NewtoPhaseI

NME

AZD2389

anti-fibroticmechanismmetabolic

dysfunction-associatedsteatohepatitis

Additionalindication

AZD0486-SOUNDTRACK-B

CD19/CD3next-generationbispecificT-cellengagerB-cellnon-Hodgkinlymphoma

Life-cyclemanagement

EnhertuDESTINY-PanTumor03#

HER2targetingADCHER2expressingsolidtumours

Additionalindication

rilvegostomigARTEMIDE-Lung02#

PD-1/TIGITbispecificmAbsquamousNSCLC1L

Life-cyclemanagement

Calquence+venetoclax+obinutuzumabAMPLIFY

BTKinhibitor+BCL-2inhibitor+anti-CD20mAb1st-linechroniclymphocyticleukaemia

Datroway(datopotamabderuxtecan)TROPION-Lung05#

TROP2ADCadvancedormetastaticEGFRm

NSCLCprogressedonpriorsystemictherapies,includingTKIsandplatinum-based

chemotherapy

KoselugoKOMET#

MEKinhibitorneurofibromatosistype1adult

NME

AZD7760

anti-staphaureusantibodycombinationtargetingATAndClfApreventionofstaphbloodstreaminfectionsinhaemodialysispatients

Additionalindication

AZD7003(China)

GPC3CAR-Tsquamousnon-smallcelllungcancer

Phaseprogressionsbasedonfirstsubjectinachievement#Partneredand/orincollaboration

5

Asof6February2025.Appendix:Glossary.

ProjectmovementssinceQ32024update

RemovedfromPhaseII

RemovedfromPhaseIII

Approved/removedfromregistration

RemovedfromPhaseI

NME

AZD0171+Imfinzi+CTx

anti-LIFmAb+PD-L1mAb+CTx1st-line

metastaticpancreaticductaladenocarcinoma

AZD4041#

orexin1receptorantagonistopioidusedisorder

sabestomig

PD-1/TIM3bispecificmAbsolidtumours

vemircopan

oralfactorDinhibitorimmunoglobulinAnephropathy/proliferativelupusnephritis

mitiperstat

myeloperoxidaseCOPD/heartfailurewithapreservedejectionfraction/NASH

NME

Datroway(datopotamabderuxtecan)TROPION-Breast01#

TROP2ADC2-3LHR+HER2-breastcancer

Kavigale(sipavibart)SUPERNOVA

SARS-CoV-2LAABpreventionofCOVID-19

Life-cyclemanagement

EnhertuDESTINY-Breast06#

HER2targetingADCpost-ETHER2-lowand-ultralow/HR+breastcancer2L

Imfinzi+/-Imjudo+CRTADRIATIC#

PD-L1mAb+/-CTLA-4mAb+CRT1st-linelimited-stageSCLC

NME

ALXN1910

nextgenerationTNSALPERTbonemetabolism

Phaseprogressionsbasedonfirstsubjectinachievement#Partneredand/orincollaboration

6

Asof6February2025.Appendix:Glossary.

Q42024Oncologynewmolecularentity1pipeline

PhaseI

21NewMolecularEntities

AZD0486

CD19/CD3TCEB-cellacutelymphoblasticleukaemia

AZD0120

autologousanti-CD19andanti-BCMACAR-Tcellimmunotherapymultiplemyeloma

AZD0754

STEAP2CAR-Tprostatecancer

AZD0022

KRasG12Dinhibitorsolidtumours

AZD0486

CD19/CD3TCEr/rB-cellnon-Hodgkinlymphoma

AZD0305

AZD2068

EGFRcMETradioconjugatesolidtumours

GPRC5DADCrelapsed/refractorymultiplemyeloma

AZD1390

ATMinhibitorglioblastoma

AZD5492

CD20TITANT-cellengagerhaematology

AZD3470

AZD5863

PRMT5inhibitorclassicHodgkinlymphoma,solidtumours

CLDN18.2xCD3bispecificantibody(HBM7022)solidtumours

AZD7003(China)

GPC3CAR-Thepatocellularcarcinoma/squamousnon-smallcelllungcancer

AZD9592

EGFR/cMETTOP1iADCsolidtumours

AZD5851

GPC3CAR-Thepatocellularcarcinoma

AZD6422

CLDN18.2CAR-Tsolidtumours

AZD8421

NT-112#

CDK2inhibitorsolidtumours

TGFBR2KOarmoredTCR-TtargetingKRASG12Dsolidtumour

AZD9829

NT-175#

CD123TOP1iADCAML,MDS

TGFBR2KOarmoredTCR-TtargetingTP53R175Hsolidtumours

NT-125#

autologous,fully-individualized,multi-specificTCRtherapytargetingneoantigenssolidtumours

volrustomig+lenvatinib

PD-1/CTLA-4+VEGFadvancedRCC

Phaseprogressionsbasedonfirstsubjectinachievement

7

1.Includesadditionalindicationsforassetswheretheleadisnotyetlaunched#Partneredand/orincollaboration

Asof6February2025.Appendix:Glossary.

PhaseII

AZD0486SOUNDTRACK-B

CD19/CD3next-generationbispecificT-cellengagerB-cellnon-Hodgkinlymphoma

AZD0901

CLDN18.2MMAEADCsolidtumours

AZD5335

anti-FRαTOP1iADCovariancancer,lungadenocarcinoma

puxitatugsamrotecan(AZD8205)B7-H4targetingADCsolidtumours

AZD9574

PARPinhibitoradvancedsolidmalignancies

camizestrant

SERDHR+breastcancer

ceralasertib

ATRinhibitorsolidtumours

FPI-2265#

PSMAradioconjugateprostatecancer

IPH5201+Imfinzi#

CD39+PD-L1neoadjuvant/adjuvantNSCLC

rilvegostomigARTEMIDE-01#

PD-1/TIGITbispecificmAbsolidtumours

saruparib

PARP1Selsolidtumours

volrustomig

PD-1/CTLA-4solidtumours

volrustomigeVOLVE-01

PD-1/CTLA-4bispecificmAbNSCLC

volrustomigeVOLVE-02

PD-1/CTLA-4bispecificmAbcervicalcancer,headandnecksquamouscellcarcinoma

14NewMolecularEntities

PhaseIII

17NewMolecularEntities

camizestrant+CDK4/6iSERENA-6

SERD+CDK4/6i1LHR+HER2-ESR1mbreastcancer

camizestrantCAMBRIA-1

SERDHR+HER2-extendedadjuvantbreastcancer

ceralasertib+ImfinziLATIFYATRinhibitor+PDL-1NSCLC

rilvegostomigARTEMIDE-Lung02#

PD-1/TIGITbispecificmAbsquamousNSCLC1L

rilvegostomigARTEMIDE-Biliary01#

PD-1/TIGITbispecificmAbadjuvantbiliarytractcancer

saruparibEvoPAR-Prostate01

PARP1Selmetastaticcastration-sensitiveprostatecancer

volrustomigeVOLVE-Cervical

PD-1/CTLA-4bispecificmAblocallyadvancedcervicalcancer

volrustomigeVOLVE-Lung02

PD-1/CTLA-4bispecificmAb1LmetastaticNSCLC

AZD0486SOUNDTRACK-F1

CD19/CD3TCEfollicularlymphoma

AZD0901CLARITY-Gastric01

CLDN18.2MMAEADCgastric2L+

camizestrant+palbociclibSERENA-4

SERD+CDK4/6i1LHR+HER2-breastcancer

camizestrant±abemaciclibCAMBRIA-2

SERD+/-CDK4/6iER+/HER2-earlybreastcancer

Imfinzi+/-oleclumab+/-monalizumabPACIFIC-9#PD-L1+NKG2AorPD-L1+CD73unresectablestageIIINSCLC

rilvegostomig

ARTEMIDE-Lung03#

PD-1/TIGITbispecificmAbnon-squamousNSCLC1L

saruparibEvoPAR-Breast01PARP1SelBRCA/PALB2mHR+vemetastaticbreast

cancer

volrustomigeVOLVE-HNSCC

PD-1/CTLA-4bispecificmAbunresectedlocallyadvancedHNSCC

volrustomigeVOLVE-Meso

PD-1/CTLA-4bispecificmAb1Lunresectablemalignantpleuralmesothelioma

●Precisionmedicineapproachbeingexplored

Underreview

0NewMolecularEntities

Q42024Oncologylifecyclemanagement1pipeline

PhaseI

0Projects

PhaseII

Enhertu(platform)DESTINY-Breast07#

HER2targetingADCHER2+breastcancer

EnhertuDESTINY-PanTumor03#

HER2targetingADCHER2expressingsolidtumours

EnhertuDESTINY-PanTumour01#HER2ADCHER2mutanttumours

Imfinzi(platform)BEGONIAPD-L11LmetastaticTNBC

Imfinzi(platform)HUDSON

PD-L1+multiplenovelONCtherapiespostIONSCLC

Imfinzi(platform)NeoCOAST-2#

PD-L1mAb+multiplenoveloncologytherapiesNSCLC

Tagrisso+OrpathysSAVANNAH#

EGFR+METadvancedEGFRmNSCLC

TagrissoORCHARDplatformstudy#

EGFR+multiplenovelONCtherapies2LEGFRmosimertinib-resistantNSCLC

Truqap

AKTprostatecancer

9Projects

PhaseIII

38Projects

Calquence+R-CHOPESCALADEBTK+R-CHOP1LDLBCL

Datroway+ImfinziAVANZAR#

TROP2ADC+PD-L1+CTxNon-squamous/Non-squamousTROP2+NSCLC(1L)

Datroway+/-ImfinziTROPION-Breast03#

TROP2ADC+/-PD-L1adjuvantresidualdiseaseTNBC

DatrowayTROPION-Lung08#

TROP2ADC1LmetastaticNSCLC

EnhertuDESTINY-Breast11#

HER2ADCneoadjuvantHER2+breastcancer

EnhertuDESTINY-Gastric04#HER2ADCHER2+gastric2L

Imfinzi+CRTKUNLUN

PD-L1+CRTlocally-advancedESCC

Imfinzi+FLOTMATTERHORN#

PD-L1+CTxneoadjuvant/adjuvantgastriccancer

Imfinzi+Imjudo+TACE+/-lenvatinibEMERALD-3

PD-L1+CTLA4+VEGF+/-chemoembolisationlocoregionalHCC

ImfinziPOTOMAC

PD-L1non-muscleinvasivebladdercancer

LynparzaMONO-OLA1#

PARP1LBRCAwtovariancancer

TagrissoADAURA2

EGFRadjuvantEGFRmNSCLCstageIa2-Ia3followingcompletetumourresection

Datroway+rilvegostomigTROPION-Lung12#

TROP2ADC+PD-1/TIGITStageIadenocarcinomaNSCLCwhoarectDNA-positiveorhavehigh-riskpathologicalfeatures

Datroway+TagrissoTROPION-Lung15#

TROP2ADC+EGFRinhibitor2LadvancedormetastaticEGFRmNSCLC

Datroway+ImfinziTROPION-Breast04#

TROP2ADC+PD-L1perioperativetriplenegativeorHR-low/HER2-negativebreastcancer

Datroway+pembrolizumabTROPION-Lung07#TROP2ADC1LNSCLCPD-L1<50%non-squamous

Enhertu+rilvegostomigDESTINY-BTC01

HER2targetingADC+PD-1/TIGITbispecificmAb1LHER2+biliarytractcancer

EnhertuDESTINY-Breast09#

HER2ADCHER2+breastcancer1L

Imfinzi+CRTPACIFIC-5(China)#

PD-L1+CRTlocally-advancedstageIIINSCLC

Imfinzi+EV+/-ImjudoVOLGA

PD-L1+nectin-4targetingADC+/-CTLA-4MIBC

Imfinzi+VEGF+TACEEMERALD-1#PD-L1+VEGF+TACElocoregionalHCC

Imfinzipost-SBRTPACIFIC-4#

PD-L1mAbpost-SBRTstageI/IINSCLC

Orpathys+ImfinziSAMETA#

MET+PD-L11Lpapillaryrenalcellcarcinoma

Tagrisso+/-CTxneoadjuvantNeoADAURA

EGFR+/-CTxstageII/IIIresectableEGFRmNSCLC

Datroway+rilvegostomigTROPION-Lung10#

TROP2ADC+PD-1/TIGITlocallyadvancedormetastaticnon-squamousNSCLCwithhighPD-L1expression(TC≥50%)andwithoutactionablegenomicalterations

DatrowayTROPION-Breast02#

TROP2ADC1Ltriplenegativebreastcancer

Datroway+ImfinziTROPION-Breast05#

TROP2ADC+PD-L11Ltriplenegativebreastcancer

Datroway+TagrissoTROPION-Lung14#

TROP2ADC+EGFRinhibitor1LEGFRmNSCLC

EnhertuDESTINY-Breast05#

HER2ADCHER2+post-neoadjuvanthigh-riskbreastcancer

EnhertuDESTINY-Lung04#HER2ADCHER2mNSCLC1L

Imfinzi+domvanalimab(AB154)PACIFIC-8#PD-L1+TIGIT+CTxunresectablestageIIINSCLC

Imfinzi+Imjudo+SoCNILE

PD-L1+CTLA-4+SoC1Lurothelialcancer

Imfinzi+VEGFEMERALD-2#PD-L1+VEGFadjuvantHCC

Lynparza+Imfinzi+bevacizumabDUO-O#PARP+PD-L1+VEGF1Lovariancancer

Tagrisso+OrpathysSAFFRON#

EGFR+METadvancedEGFRmnon-smallcelllungcancer

Truqap+abirateroneCAPItello-281

AKT+abirateronePTENdeficientmHSPC

Underreview

3Projects

Calquence+venetoclax+obinutuzumabAMPLIFYBTK+BCL-2+anti-CD201LCLL

DatrowayTROPION-Lung05#TROP2ADCadvancedor

metastaticEGFRmNSCLCprogressedonpriorsystemictherapies,includingTKIsandplatinum-basedchemotherapy

Imfinzi+CTxNIAGARA

PD-L1+CTxmuscleinvasivebladdercancer

Truqap+docetaxelCAPItello-280

AKT+docetaxelmCRPCprostatecancer

Phaseprogressionsbasedonfirstsubjectinachievement

8

1.IncludessignificantlifecyclemanagementprojectsandparallelindicationsforassetsbeyondPhaseIII#Partneredand/orincollaboration

Asof6February2025.Appendix:Glossary.

Truqap+Faslodex+palbociclibCAPItello-292

AKT+fulvestrant+CDK4/61Ltripletinearlyrelapse/ETresistantlocallyadvancedormBC

·Precisionmedicineapproachbeingexplored

Q42024BioPharmaceuticalsnewmolecularentity1pipeline

PhaseI

15NewMolecularEntities

AZD0120

autologousanti-CD19andanti-BCMACAR-Tcellimmunotherapysystemiclupuserythematosus

AZD0233

CX3CR1dilatedcardiomyopathy

AZD0292

AZD1163

bispecificantibodyrheumatoidarthritis

pseudomonasPsl-PcrVbispecificmAbnon-CFbronchiectasis

AZD2373

podocytehealthnephropathy

AZD1705

lipidloweringcardiovasculardisease

AZD5148

anti-clostridioidesdifficileTcdBmAbreductionofC.diffrecurrence

AZD6912

siRNArheumatoidarthritis

AZD4144

inflammationmodulatorcardiorenaldisease

AZD6793

IRAK4inhibitorinflammatorydiseases

AZD8965

AZD7760

inhibitionofarginaseenzymeidiopathicpulmonaryfibrosis

MEDI1814#

amyloidbetamAbAlzheimer’sdisease

mAbcombinationtargetingSaureusvirulencefactorspreventionofStaphaureusinfection

AZD9550

GLP-1Rglucagondualagonistnon-alcoholicsteatohepatitis

mRNAVLPvaccine

mRNA-VLPvaccinepreventionofCOVID-19

PhaseII

17NewMolecularEntities

atuliflaponFLAPasthma

AZD0780

PCSK9dyslipidemia

AZD2693

AZD2389

anti-fibroticmechanismmetabolic

dysfunction-associatedsteatohepatitis

NASHresolutionnon-alcoholicsteatohepatitis

AZD3427

relaxinmimeticheartfailure

AZD4604

inhaledJAK1inhibitorasthma

AZD5004

oralGLP-1receptoragonistT2D/chronicweightmanagement

AZD6234

peptidechronicweightmanagementinoverweightorobesity

AZD5462#

RXFP1agonistheartfailure

AZD7798

humanisedmonoclonalantibody

targetsT-cellssubsetCrohn'sdisease

AZD8630#

inhaledTSLPFAbasthma

balcinrenone/dapagliflozin

MRmodulator+SGLT2inhibitorCKD

IVX-A12

virus-likeparticle(VLP)vaccineRSVandhumanmetapneumovirus(hMPV)

MEDI0618

PAR2antagonistmAbmigraine

MEDI7352

NGF/TNFOApain/PDN

tozorakimabFRONTIER3IL-33asthma

zibotentan/dapagliflozinendothelinAreceptor

antagonist/SGLT2ilivercirrhosis

PhaseIII

6NewMolecularEntities

balcinrenone/dapagliflozin

MRmodulator+SGLT2inhibitorheartfailurewithCKD

baxdrostatBaxHTN

aldosteronesynthaseinhibitorhypertension

baxdrostat/dapagliflozin

aldosteronesynthaseinhibitorandreversibleinhibitorofSGLT2CKD

tozorakimabOBERONTITANIA

PROSPEROMIRANDAIL-33COPD

tozorakimabTILIA

IL-33severevirallowerrespiratorytractdisease

zibotentan/dapagliflozin

endothelinAreceptorantagonist/SGLT2iCKDwithhighproteinuria

Underreview

0NewMolecularEntities

Phaseprogressionsbasedonfirstsubjectinachievement

9

1.Includesadditionalindicationsforassetswheretheleadisnotyetlaunched#Partneredand/orincollaboration

Asof6February2025.Appendix:Glossary.

·Precisionmedicineapproachbeingexplored

Q42024BioPharmaceuticalslifecyclemanagement1pipeline

PhaseI

0Projects

PhaseII

1Project

TezspireCOURSE#

TSLPchronicobstructivepulmonarydisease

PhaseIII

13Projects

Breztri/TrixeoATHLOS

LABA/LAMA/ICSCOPDcardiopulmonaryexercisetrial

FasenraRESOLUTE#

IL-5Rchronicobstructivepulmonarydisease

SaphneloDAISY#

typeIIFNreceptorsystemicsclerosis

SaphneloJASMINE#

typeIIFNreceptormAbmyositis

SaphneloTULIP-SC#

typeIIFNreceptorsystemiclupuserythematosus(subcutaneous)

TezspireCROSSING#

TSLPeosinophilicoesophagitis

Breztri/TrixeoTHARROS#

LABA/LAMA/ICScardiopulmonaryoutcomestrialinCOPD

FasenraNATRON

IL-5Rhypereosinophilicsyndrome

SaphneloIRIS#

typeIIFNreceptormAblupusnephritis

SaphneloLAVENDER#

typeIIFNreceptormAbcutaneouslupuserythematosus

TezspireWAYPOINT#TSLPnasalpolyps

Wainua#

LICAATTR-cardiomyopathy

Breztri/Trixeo(PT010)KALOSLOGOSLABA/LAMA/ICSasthma

Underreview

0Projects

Phaseprogressionsbasedonfirstsubjectinachievement

10

1.IncludessignificantlifecyclemanagementprojectsandparallelindicationsforassetsbeyondPhaseIII#Partneredand/orincollaboration

Asof6February2025.Appendix:Glossary.

·Precisionmedicineapproachbeingexplored

Q42024RareDiseasepipeline1

PhaseI

3Projects

ALXN1920

kidney-targetedfactorHfusionproteinnephrology

ALXN2030

siRNAtargetingcomplementC3nephrology

ALXN2080

oralfactorDhealthyvolunteers

PhaseII

2Projects

MEDI1341#

alphasynucleinmAbmultiplesystematrophy/Parkinson'sdisease

Ultomiris

anti-complementC5mAb

lupusnephritis

proliferative

PhaseIII

8Projects

ALXN2220DepleTTR-CM#

cardiomyopathy

TTRdepletertransthyretinamyloid

anselamimab

fibril-reactivemAbamyloidlight-chainamyloidosis

efzimfotasealfa

nextgenerationTNSALPERThypophosphatasia

eneboparatideCALYPSO

parathyroidhormonereceptor1hypoparathyroidism

gefurulimabPREVAIL

humanisedbispecificVHHantibodygeneralisedmyastheniagravis

Ultomiris

anti-complementC5mAbhaematopoieticstemcelltransplant-associatedthromboticmicroangiopathy

UltomirisARTEMIS

anti-complementC5mAbcardiacsurgery-associatedacutekidneyinjury

UltomirisICAN

anti-complementC5mAbimmunoglobulinAnephropathy

Underreview

2Projects

acoramidis#

oralTTRstabilisertransthyretinamyloid

cardiomyopathy

KoselugoKOMET#

MEKinhibitorneurofibromatosistype1adult

Phaseprogressionsbasedonfirstsubjectinachievement

11

1.Includesnewmolecularentitiesandsignificantlifecyclemanagementprojects#Partneredand/orincollaboration

Asof6February2025.Appendix:Glossary.

·Precisionmedicineapproachbeingexplored

Designationsinourpipeline

14

FastTrack

4

Acceleratedapprovals

10

Andexxaacutemajorbleed(US)

KavigaleSARS-CoV-2LAABpreventionofCOVID-19(EU)

Calquencer/rMCLACE-LY-004(US)

EnhertuHER2overexptumors(DESTINY-PanTumor02)(US)

TezspireasthmaNAVIGATOR(US)

TezspireCOPDCOURSE(US)

tozorakimabsevereviralLRTDTILIA(CN)

Calquencer/rMCLACE-LY-004(US)

CalquenceCLL(1L)ELEVATE-TN(US)

Datrowaypost-TKINSCLC3L+TROPION-Lung05(US)

EnhertuHER2-overexpressingtumoursDESTINY-PanTumor02(US)

Enhertupost-ETHER2lowand-ultralowHR+breast1LDESTINY-Breast06(US)

Imfinzi+/-Imjudo+CRTLS-SCLC(1L)ADRIATIC(US)

TagrissostageIIIEGFRmNSCLCLAURA(US)

Breakthrough/PRIME1/Sakigake2

AZD0292Psl-PcrVN3YNCFBE(US)

AZD3427relaxinmimeticheartfailure(US)

AZD7760StaphaureusmAbs-Hemodialysis(US)

balci/dapaHFwithCKD(US)

SaphneloSLE(US)

tozorakimabCOPD(US)

tozorakimabsevereviralLRTD(US)

WainuaATTR-Cardiomyopathy(US)

camizestrant1LHR+HER2-ESR1mbreastcancerSERENA-6(US)

Orpathys+TagrissoNSCLCSAVANNAH/SAFFRON(US)

Truqap+fulvHR+breast(2L+)CAPItello-291(US)

ALXN2220DepleTTR-CM(US)

anselamimabALamyloidosisCAEL101-301/2(US)

eneboparatideHypoPT(US)

ACCELERATEDAPPROVAL,theseregulationsallowedmedicinesforseriousconditionsthataddressedanunmetmedicalneedtobeapprovedbasedonasurrogateendpoint

BREAKTHROUGHDESIGNATIONisaprocessdesignedtoexpeditethedevelopmentandreviewofmedicineswhichmaydemonstratesubstantialimprovementoveravailabletherapy.1PRIMEisaschemelaunchedbytheEMAtoenhancesupportforthedevelopmentofmedicinesthattargetanunmetmedicalneed.

2SAKIGAKEisaimedatearlyintroductionofinnovativemedicines,medicaldevices,etc.thatareinitiallydevelopedinJapan

FASTTRACKisaprocessdesignedtofacilitatethedevelopment,andexpeditethereviewofmedicinestotreatseriousconditionsandfillanunmetmedicalneedPRIORITYREVIEWDESIGNATIONistheUSFDA’sgoaltotakeactiononanapplicationwithin6months

ORPHANDRUGDESIGNATION,intendedfortreatment,diagnosisorpreventionofrarediseases/disordersthataffectfewerthan200,000patientsintheUS,orthataffectmorethan200,000patientsbutarenotexpectedtorecoverthecostsofdevelopingandmarketingatreatmentdrug

QUALIFIEDINFECTIOUSDISEASEPRODUCTdesignationconfersparticularadvantages,includingpriorityreviewbytheUSFoodandDrugAdministration(FDA)andfast-trackdesignation,whichcanacceleratedevelopmentofaproduct,aswellasanadditionalfiveyears'marketexclusivityifaproductislicensed.

13

PriorityReview

TezspireasthmaNAVIGATOR(US)

CalquenceMCL(1L)ECHO(US)

DatrowayEGFRmpost-TKINSCLC3L+TROPION-Lung05(US)

EnhertuHER2overexpressingtumorsDESTINY-PanTumor02(US)

Enhertupost-ETHER2low/ultralowHR+breast1LDESTINY-Breast06(US)

Imfinzi+CTxMIBCNIAGARA(US)

Imfinzi+ImjudoHCC(1L)HIMALAYA(US)

Imfinzi+ImjudoLS-SCLCADRIATIC(US)

Lynparza+abirateroneall-comersmCRPC(1L)PROpel(US)

TagrissostageIIIEGFRmNSCLCLAURA(US)

TagrissostageIIIEGFRmNSCLCLAURA(CN)

Truqap+fulvHR+breast(2L+)CAPItello-291(US)

ALNX2220ATTR-CM(JP)

3

Qualifiedinfectiousdiseaseproduct

AZD0292Psl-PcrVN3YNCFBE(US)

AZD5148C.difficilemAb-PreventionofRecurrence(US)

AZD7760preventionofStaphaureusinfection(US)

24

Orphan

Andexxaacutemajorbleed(JP)

FasenraEGPAMANDARA(US)

FasenraHESNATRON(US)

SaphnelomyositisJASMINE(US)

Saphnelosystemicsclerosis(US)

TezspireEoECROSSING(US)

Wainuatransthyretin-mediatedamyloidosis(US)

CalquenceCLL(1L)ELEVATE-TN(US)

CalquenceCLL(1L)ELEVATE-TN(EU)

Calquencer/rMCLACE-LY-004(US)

Imfinzi+ImjudoLS-SCLCADRIATIC(JP)

Imfinzi+/-ImjudoHCC(1L)HIMALAYA(EU)

Imfinzi+/-ImjudoHCC(1L)HIMALAYA(US)

ALXN2220ATTR-CMDepleTTR-CM(US)

ALXN2220ATTR-CMDepleTTR-CM(EU)

ALXN2220ATTR-CMDepleTTR-CM(JP)

anselamimabALamyloidosisCAEL101-301/2(US)

anselamimabALamyloidosisCAEL101-301/2(EU)

gefurulimabmyastheniagravisPREVAIL(US)

KoselugoNF1adult1LKOMET(US)

KoselugoNF1adult1LKOMET(EU)

KoselugoNF1adult1LKOMET(JP)

KoselugoNF1adult1LKOMET(CN)

UltomirisHSCT-TMAALXN1210-TM-313(US)

12

NOTE:excludesdesignationsforprojectswhichhavelaunchedinallapplicablemajormarketsAsof6February2025.

Appendix:Glossary.

Oncology

BioPharmaceuticals

RareDisease

Oncology:

approvedmedicinesandlate-stage

pipeline

Oncology

Approvedmedicines

Late-stagedevelopmentEarlydevelopment

Imfinzi(PD-L1mAb)

V&IOtherR&ICVRM

Gastrointestinalcancer

Trial

Population

Patients

Design

Endpoints

Status

PhaseIII

EMERALD-1

NCT03778957

LocoregionalHCC

710

?Arm1:TACEincombinationwithImfinzi

?Arm2:TACEincombinationwithImfinzi+bevacizumab

?Arm3:TACEincombinationwithplacebo

?Primaryendpoint:PFS(Arm2vs.Arm3)

?Secondaryendpoints:PFS(Arm1vs.Arm3)andOS

?FPCD:Q12019

?LPCD:Q32021

?Datareadout:Q42023

?Primaryendpointmet

PhaseIII

HCC(adjuvant)

908

?Arm1:Imfinzi+bevacizumab

?Primaryendpoint:RFS(Arm1vs.

?FPCD:Q22019

EMERALD-2

?Arm2:Imfinzi+