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Pembrolizumab

Phase 3

Locally Advanced or Metastatic Non-Small Cell Lung Cancer | Small molecule | Oncology |AstraZeneca PLC|Last Updated: Jul 10, 2026

Target and mechanism

Molecular targetPDCD1
Target classInhibitor
ModalitySmall molecule

Success Probability

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Market & Valuation

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Trial Design

RandomizedACTIVE_CONTROLLEDDMC
Total Trials1
Total Enrollment454

FDA Designations

No designations recorded

Clinical trial landscape

Pembrolizumab · 2 trials · 4 indications

Phase 3 1Phase 2 1
NCT05048797A Study to Investigate the Efficacy and Safety of Trastuzumab Deruxtecan as the First Treatment Option for Unresectable, Locally Advanced/Metastatic Non-Small Cell Lung Cancer With HER2 MutationsLocally Advanced or Metastatic Non-Small Cell Lung Cancer
ACTIVE NOT_RECRUITING454 Analytics
PHASE3ACTIVE NOT_RECRUITING
A Study to Investigate the Efficacy and Safety of Trastuzumab Deruxtecan as the First Treatment Option for Unresectable, Locally Advanced/Metastatic Non-Small Cell Lung Cancer With HER2 Mutations
Locally Advanced or Metastatic Non-Small Cell Lung CancerUnlock trial analytics

Study Endpoints

Primary Endpoints

Progression Free Survival (PFS) by Blinded Independent Central Review (BICR)
Until progression or death, assessed up to approximately 12 months

Defined as time from randomization until progression per RECIST 1.1 as assessed by Blinded Independent Central Review (BICR), or death due to any cause.

Confirmed Intracranial Objective Response Rate (iORR) as Measured by the RANO-BM Criteria
Through study primary completion, an average of 2 years

The intracranial objective response rate (iORR) is defined as the proportion of patients achieving a complete response (CR) or partial response (PR) according to the RANO-BM (Response Assessment in Neuro-Oncology Brain Metastases) criteria, as assessed by central review. The best overall CNS (central nervous system) response is determined based on a composite assessment of radiographical CNS target and non-target lesion responses, corticosteroid use, and clinical status.

Secondary Endpoints

Overall Survival (OS)
Until death, assessed up to approximately 28 months.
Progression Free Survival (PFS) by investigator assessment
Until progression, assessed up to approximately 12 months
Objective Response Rate (ORR)
Until progression, assessed up to approximately 12 months
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Study Design & Arms

AllocationRANDOMIZED
MaskingNONE
ModelPARALLEL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Arm 1EXPERIMENTALTrastuzumab Deruxtecan (T-DXd)
Arm 2ACTIVE_COMPARATORStandard of Care Treatment (platinum, pemetrexed and pembrolizumab)
Single arm: pembrolizumab, datopotamab-deruxtecan, and carboplatinEXPERIMENTALStudy treatment: * INDUCTION: Pembrolizumab 200 mg IV Q3W; Datopotamab-Deruxtecan 6mg/kg IV Q3W; Carboplatin AUC 5 IV Q3W. * For four (4) cycles. * MAINTENANCE: Pembrolizumab 200 mg IV Q3W for 35 cycles; Datopotamab-Deruxtecan 6mg/ kg IV Q3W.\* * until disease progression or unacceptable toxicity.

Interventions

NameTypeDescription
Trastuzumab DeruxtecanDRUGTrastuzumab Deruxtecan administered by intravenous infusion
CisplatinDRUGInvestigator's choice of platinum chemotherapy (cisplatin) administered by intravenous infusion
CarboplatinDRUGInvestigator's choice of platinum chemotherapy (carboplatin) administered by intravenous infusion
PembrolizumabDRUGPembrolizumab administered by intravenous infusion
PemetrexedDRUGPemetrexed administered by intravenous infusion
Datopotamab deruxtecanDRUGDatopotamab deruxtecan (Dato-DXd) is a novel antibody-drug conjugate (ADC) composed of a humanized anti-TROP2 IgG1 monoclonal antibody linked to a topoisomerase I inhibitor payload and a cleavable linker.
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Eligibility Criteria

Age Range18 Years to 123 Years
SexALL
Healthy VolunteersNo
Study Sites130

Inclusion Criteria: * Participants at least 18 years of age * Locally advanced and unresectable NSCLC, not amenable to curative therapy, or metastatic disease * Histologically documented non-squamous NSCLC with HER2 mutation in exons 19 or 20 by tissue NGS or ctDNA * Treatment-naïve for palliative ...

Countries:United StatesAustriaBelgiumBrazilCanadaChinaDenmarkFranceGermanyHong KongIndiaItalyJapanMexicoNetherlandsPolandSouth KoreaSpainTaiwanTurkey (Türkiye)
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Recent Changes (Last 90 Days)

LOWJul 10, 2026NCT05048797primaryCompletionDate: changed
LOWJul 10, 2026NCT05048797primaryCompletionDate: changed

Frequently asked questions about Pembrolizumab

What is Pembrolizumab used for?

Pembrolizumab is being studied for use in locally advanced or metastatic non-small cell lung cancer and lung cancer, including in a Phase 3 trial for patients with HER2 mutations and a Phase 2 trial for treatment-naive brain metastases from NSCLC. It is an investigational oncology therapy.

What does Pembrolizumab target?

Pembrolizumab targets PDCD1, also known as programmed cell death protein 1, and acts as an inhibitor of this target. By inhibiting PDCD1, it is designed to modulate immune responses in the treatment of cancer.

Who is developing Pembrolizumab?

Pembrolizumab is being developed by AstraZeneca PLC, which is listed on the stock exchange under the ticker AZN. The company is conducting clinical trials to evaluate the drug's efficacy and safety in lung cancer indications.

What phase is Pembrolizumab in?

Pembrolizumab is in Phase 3 clinical development for locally advanced or metastatic non-small cell lung cancer. It is also being evaluated in a Phase 2 trial for brain metastases from NSCLC. The drug is investigational and not yet approved.

What clinical trials is Pembrolizumab in?

Pembrolizumab is being studied in two trials: NCT05048797, a Phase 3 study of trastuzumab deruxtecan as first treatment for HER2-mutant NSCLC with 454 participants, and NCT06822543, a Phase 2 study of datopotamab deruxtecan, carboplatin, and pembrolizumab for brain metastases with 46 participants.

Is Pembrolizumab the same as other drugs?

Pembrolizumab is a distinct drug candidate being developed by AstraZeneca. No alternative names for this drug have been reported in the clinical trial information available.