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Zanidatamab

Phase 3

Biliary Tract Cancer | Small molecule | Oncology |Jazz Pharmaceuticals plc|Last Updated: Sep 1, 2026

Target and mechanism

Molecular targetERBB2
Target classInhibitor
ModalitySmall molecule

Success Probability

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Market & Valuation

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Trial Design

RandomizedACTIVE_CONTROLLED
Total Trials1
Total Enrollment286

FDA Designations

BREAKTHROUGH_THERAPYFAST_TRACKORPHAN_DRUGPRIORITY_REVIEW

Clinical trial landscape

Zanidatamab · 7 trials · 23 indications

Phase 3 3Phase 2 3Phase 1 1
NCT06435429A Study Comparing the Efficacy and Safety of Zanidatamab to Trastuzumab, Each in Combination With Physician's Choice Chemotherapy, for the Treatment of Participants With Metastatic HER2-positive Breast CancerMetastatic HER2-positive Breast Cancer
RECRUITING550 Analytics
NCT06282575Efficacy and Safety of Zanidatamab With Standard-of-care Therapy Against Standard-of-care Therapy for Advanced HER2-positive Biliary Tract CancerBiliary Tract Cancer
RECRUITING286 Analytics
NCT05152147A Study of Zanidatamab in Combination With Chemotherapy Plus or Minus Tislelizumab in Patients With HER2-positive Advanced or Metastatic Gastric and Esophageal CancersGastric Neoplasms
ACTIVE NOT_RECRUITING920 Analytics
PHASE3RECRUITING
A Study Comparing the Efficacy and Safety of Zanidatamab to Trastuzumab, Each in Combination With Physician's Choice Chemotherapy, for the Treatment of Participants With Metastatic HER2-positive Breast Cancer
Metastatic HER2-positive Breast CancerUnlock trial analytics
PHASE3RECRUITING
Efficacy and Safety of Zanidatamab With Standard-of-care Therapy Against Standard-of-care Therapy for Advanced HER2-positive Biliary Tract Cancer
Biliary Tract CancerUnlock trial analytics
PHASE3ACTIVE NOT_RECRUITING
A Study of Zanidatamab in Combination With Chemotherapy Plus or Minus Tislelizumab in Patients With HER2-positive Advanced or Metastatic Gastric and Esophageal Cancers
Gastric NeoplasmsUnlock trial analytics

Study Endpoints

Primary Endpoints

Progression-free Survival (PFS) Per RECIST Version 1.1 As Assessed by Blinded Independent Central Review (BICR)
Until disease progression or death, up to approximately 44 months

PFS is defined as the time in months from randomization to the date of first documented disease progression (as assessed by BICR according to RECIST v1.1) or death from any cause, whichever occurs first.

Progression Free Survival (PFS) in participants with Immunohistochemistry (IHC) 3+ tumors
Up to 52 months

PFS is defined as the time from the date of randomization to the date of documented disease progression, or death from any cause.

Progression-free survival (PFS) by BICR
Up to 2.5 years

The time from randomization to the date of documented disease progression (per Response Evaluation Criteria in Solid Tumors \[RECIST\] version 1.1) as assessed by blinded independent central review (BICR) or death from any cause

Overall survival
Up to 3.5 years

The time from randomization to death due to any cause

Confirmed Objective Response Rate (ORR) by investigator
From Baseline to disease progression (up to 28 months)

Confirmed Objective Response Rate (ORR) for each cohort, based on best overall response, defined as the percentage of patients with a complete response (CR) or partial response (PR) during treatment or follow-up, assessed according to RECIST1.1, by investigator assessment and confirmed by a follow-up scan at ≥ 4 weeks from first response assessment.

Number of participants with pCR
Up to 8 months

Pathologic complete response (pCR) is defined as lacking all signs of cancer in the tissue samples removed in the breast or axilla (armpit) during surgery or biopsy after study treatment

Confirmed Objective Response Rate (cORR) per RECIST Version 1.1, as assessed by ICR
Up to 2.5 years

The Independent Central Review (ICR) assessed cORR is defined as the proportion of participants who had a best overall response of Complete Response (CR), or Partial Response (PR) based on Response Evaluation Criteria in Solid Tumors Version 1.1 (RECIST 1.1)

Incidence of dose-limiting toxicities (DLTs; Part 1)
Up to 4 weeks

Number of patients who experienced a DLT. DLTs include specifically defined adverse events (AEs) considered to be related to zanidatamab or evorpacept (ALX148), including combination of zanidatamab with evorpacept (ALX148)

Incidence of AEs (Part 1)
Up to 7 months

Number of patients who experienced AEs, serious adverse events (SAEs), or adverse events of special interest (AESIs)

Incidence of clinical laboratory abnormalities (Part 1)
Up to 7 months

Number of patients who experienced a Grade 3 or higher post-baseline laboratory abnormality, including either hematology or chemistry. Grades are defined using National Cancer Institute's Common Terminology Criteria for Adverse Events (NCI-CTCAE), version 5.0

Confirmed objective response rate (ORR)(Part 2)
Up to 2 years

Number of patients who achieved a confirmed best overall response (BOR) of either complete response (CR) or partial response (PR) during treatment per Response Evaluation Criteria in Solid Tumors (RECIST) version 1.1

Secondary Endpoints

Overall Survival (OS)
Until death, up to approximately 80 months
Confirmed Objective Response Rate (ORR) Per RECIST Version 1.1, As Assessed by BICR
Until disease progression or death, up to approximately 44 months
Duration of Response (DOR) Per RECIST Version 1.1, As Assessed by BICR
Until disease progression or death, up to approximately 44 months
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Study Design & Arms

AllocationRANDOMIZED
MaskingNONE
ModelPARALLEL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Zanidatamab plus physician's choice of chemotherapyEXPERIMENTALParticipants with HER2-positive metastatic breast cancer (mBC) who have progressed on, or are intolerant to, previous T-DXd treatment will be randomized to receive intravenous infusion of zanidatamab plus physician's choice of chemotherapy (eribulin, or vinorelbine, or gemcitabine, or capecitabine).
Trastuzumab plus physician's choice of chemotherapyACTIVE_COMPARATORParticipants with HER2-positive metastatic breast cancer (mBC) who have progressed on, or are intolerant to, previous T-DXd treatment will be randomized to receive intravenous infusion of trastuzumab plus physician's choice of chemotherapy (eribulin, or gemcitabine, or vinorelbine, or capecitabine).
Zanidatamab with Standard-of-care Therapy ArmEXPERIMENTALZanidatamab plus standard of care treatment of CisGem with or without a PD-1/L1 inhibitor. PD-1/L1 inhibitor will be physician's choice of either Durvalumab or Pembrolizumab, where approved under local regulations.
Standard-of-care Therapy ArmACTIVE_COMPARATORStandard of care treatment of CisGem with or without a PD-1/L1 inhibitor. PD-1/L1 inhibitor will be physician's choice of either Durvalumab or Pembrolizumab, where approved under local regulations.
Arm AACTIVE_COMPARATORTrastuzumab (Herceptin®) plus physician's choice of capecitabine plus oxaliplatin (CAPOX) or 5-fluorouracil (5-FU) plus cisplatin (FP)
Arm BEXPERIMENTALZanidatamab plus physician's choice of CAPOX or FP
Arm CEXPERIMENTALZanidatamab and tislelizumab plus physician's choice of CAPOX or FP
zanidatamabEXPERIMENTALSingle arm study where the experimental regimen used for all patients will be zanidatamab, administered intravenously every 3 weeks : * Patients \<70 kg: 1800 mg IV Q3W on Day 1 of each 21-day cycle * Patients ≥70 kg: 2400 mg IV Q3W on Day 1 of each 21-day cycle.
Zanidatamab with paclitaxelACTIVE_COMPARATORZanidatamab in combination with chemotherapy paclitaxel
Zanidatamab with docetaxel and carboplatinACTIVE_COMPARATORZanidatamab in combination with chemotherapy docetaxel and carboplatin
Trastuzumab and pertuzumab with docetaxel and carboplatinACTIVE_COMPARATORTrastuzumab and pertuzumab in combination with chemotherapy docetaxel and carboplatin
Zanidatamab treatment armEXPERIMENTALEligible participants receiving zanidatamab treatment
Zanidatamab plus evorpacept (ALX148)EXPERIMENTAL -

Interventions

NameTypeDescription
ZanidatamabDRUGAdministered by intravenous infusion
TrastuzumabDRUGAdministered by intravenous infusion
EribulinDRUGAdministered by intravenous infusion
VinorelbineDRUGAdministered by intravenous infusion
GemcitabineDRUGAdministered by intravenous infusion
CapecitabineDRUGGiven orally
CisplatinDRUGAdministered intravenously (IV)
PembrolizumabDRUGAdministered intravenously (IV)
DurvalumabDRUGAdministered intravenously (IV)
TislelizumabDRUGAdministered IV
OxaliplatinDRUGAdministered IV
5-FluorouracilDRUGAdministered IV
PaclitaxelDRUGAdministered intravenously (IV)
DocetaxelDRUGAdministered intravenously (IV)
CarboplatinDRUGAdministered intravenously (IV)
PertuzumabDRUGAdministered intravenously (IV)
EvorpaceptDRUGAdministered IV
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Eligibility Criteria

Age Range18 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites166

Participants are eligible to be included in the study only if all of the following criteria apply: 1. Is 18 years of age or of the legal adult age per local standard at the time of signing the informed consent. 2. Has histologically confirmed HER2-positive breast cancer according to ASCO-CAP Guidel...

Countries:United StatesAustraliaAustriaBelgiumBrazilCanadaFranceGermanyGreeceItalyJapanPolandSouth KoreaSpainUnited KingdomArgentinaChileChinaCzechiaFinlandIndiaIsraelPortugalPuerto RicoRomaniaSerbiaSwedenTaiwanTurkey (Türkiye)EstoniaGeorgiaGuatemalaIrelandMalaysiaMexicoNetherlandsSingaporeSouth AfricaThailandUkraine
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Recent Changes (Last 90 Days)

LOWSep 1, 2026NCT05152147primaryCompletionDate: changed
LOWSep 1, 2026NCT05152147primaryCompletionDate: changed
LOWAug 25, 2026NCT07102381lastUpdatePostDate: changed
LOWAug 25, 2026NCT07102381lastUpdatePostDate: changed
MEDIUMAug 1, 2026NCT05027139TRIAL_REMOVED: changed
MEDIUMAug 1, 2026NCT05027139TRIAL_REMOVED: changed
MEDIUMAug 1, 2026NCT05027139TRIAL_REMOVED: changed
LOWJul 17, 2026NCT05152147lastUpdatePostDate: changed
LOWJul 17, 2026NCT05152147lastUpdatePostDate: changed
LOWJul 15, 2026NCT07102381lastUpdatePostDate: changed
LOWJul 15, 2026NCT07102381lastUpdatePostDate: changed
LOWJul 14, 2026NCT06282575lastUpdatePostDate: changed
LOWJul 14, 2026NCT06282575lastUpdatePostDate: changed
LOWJul 10, 2026NCT06695845lastUpdatePostDate: changed
LOWJul 10, 2026NCT06695845lastUpdatePostDate: changed
LOWJul 2, 2026NCT07102381lastUpdatePostDate: changed
LOWJul 2, 2026NCT07102381lastUpdatePostDate: changed
LOWJul 2, 2026NCT07102381lastUpdatePostDate: changed

Frequently asked questions about Zanidatamab

What is Zanidatamab used for?

Zanidatamab is an investigational oncology therapy being studied for HER2-expressing cancers, including gastric neoplasms, HER2-positive breast cancer, metastatic HER2-positive breast cancer, and non-small cell lung cancer. It is currently in Phase 3 clinical development and has not been approved by the FDA.

What does Zanidatamab target?

Zanidatamab targets ERBB2, also known as HER2, and acts as an inhibitor of this receptor. By inhibiting ERBB2, the drug is designed to interfere with signaling pathways that drive tumor growth in HER2-expressing cancers.

Who makes Zanidatamab?

Zanidatamab is being developed by Jazz Pharmaceuticals plc, a biopharmaceutical company traded on the NASDAQ under the ticker JAZZ. The company is conducting clinical trials to evaluate the drug's safety and efficacy in multiple HER2-positive cancer indications.

What phase is Zanidatamab in?

Zanidatamab is in Phase 3 clinical development for gastric and esophageal cancers and metastatic HER2-positive breast cancer. It has received FDA designations including Breakthrough Therapy, Fast Track, Orphan Drug, and Priority Review, but it remains investigational and is not yet FDA approved.

What clinical trials is Zanidatamab in?

Zanidatamab is being studied in several trials, including NCT05152147, a Phase 3 study in HER2-positive gastric and esophageal cancers; NCT06435429, a Phase 3 study in metastatic HER2-positive breast cancer; and NCT06695845, a Phase 2 study in HER2-expressing tumors. These trials are actively recruiting or ongoing.

Is Zanidatamab the same as other HER2-targeting drugs?

Zanidatamab is a distinct investigational agent that targets ERBB2 (HER2) and is being compared against trastuzumab in a Phase 3 breast cancer trial. No alternative names for Zanidatamab have been disclosed, and it is not the same as other approved HER2 inhibitors.