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DS-8201a

Phase 3

HER2-Positive Primary Breast Cancer | Small molecule | Oncology |AstraZeneca PLC|Last Updated: May 15, 2026

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Trial Design

RandomizedACTIVE_CONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment1,600

FDA Designations

No designations recorded

Clinical trial landscape

DS-8201a · 3 trials · 4 indications

Phase 3 1Phase 2 1Phase 1 1
NCT04622319A Study of Trastuzumab Deruxtecan (T-DXd) Versus Trastuzumab Emtansine (T-DM1) in High-risk HER2-positive Participants With Residual Invasive Breast Cancer Following Neoadjuvant Therapy (DESTINY-Breast05)HER2-Positive Primary Breast Cancer
ACTIVE NOT_RECRUITING1,600 Analytics
PHASE3ACTIVE NOT_RECRUITING
A Study of Trastuzumab Deruxtecan (T-DXd) Versus Trastuzumab Emtansine (T-DM1) in High-risk HER2-positive Participants With Residual Invasive Breast Cancer Following Neoadjuvant Therapy (DESTINY-Breast05)
HER2-Positive Primary Breast CancerUnlock trial analytics

Study Endpoints

Primary Endpoints

Invasive Disease-free Survival (IDFS) in Participants Who Were Administered Trastuzumab Deruxtecan (T-DXd) Compared With Trastuzumab Emtansine (T-DM1) Treatment
Randomization to date of invasive local, axillary or distant recurrence, invasive contralateral breast cancer or death from any cause (whichever occurs first), up to approximately 57 months postdose
Number of Participants With Best Objective Response Based on Independent Central Review (Confirmed and Unconfirmed) Following DS-8201a Treatment in Participants With Human Epidermal Growth Factor Receptor 2 (HER2)-Expressing Advanced Colorectal Cancer
Baseline up to withdrawal of consent, progressive disease, or unacceptable toxicity (whichever occurs first), up to 18 months post-dose

Best objective response was reported based on independent central review. As per Response Evaluation Criteria In Solid Tumors (RECIST) version 1.1, CR was defined as a disappearance of all target lesions, PR was defined as at least a 30% decrease in the sum of diameters of target lesions, and stable disease (SD) was defined as neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for progressive disease (PD; at least a 20% increase in the sum of diameters of target lesions.

Number of Participants With Objective Response Rate Based on Independent Central Review (Confirmed and Unconfirmed) Following DS-8201a Treatment in Participants With Human Epidermal Growth Factor Receptor 2 (HER2)-Expressing Advanced Colorectal Cancer
Baseline up to withdrawal of consent, progressive disease, or unacceptable toxicity (whichever occurs first), up to 18 months post-dose

Objective response rate (defined as CR+PR) was reported based on independent central review. As per Response Evaluation Criteria In Solid Tumors (RECIST) version 1.1, CR was defined as a disappearance of all target lesions and PR was defined as at least a 30% decrease in the sum of diameters of target lesions.

Objective Response Rate (ORR) Following Treatment With DS-8201a in Participants With Advanced Solid Malignant Tumors (Dose Escalation and Dose Expansion Phases)
From 6 months postdose of last participant up to 3 years 5 months

Objective response rate (ORR) by independent central review was defined as the proportion of participants who achieve either complete response \[CR\] or partial response \[PR\] per Response Evaluation Criteria in Solid Tumors (RECIST) v1.1. CR was defined as a disappearance of all target lesions and PR was defined as at least a 30% decrease in the sum of diameters of target lesions. HER2-positive other solid tumors included participants with salivary/submandibular/parotid gland (8 participants), breast with HER2-mutation (2 participants), endometrial (2 participants), esophageal (2 participants), Paget's disease (2 participants), cholangiocarcinoma (1 participant), extraskeletal myxoide chondrosarcoma (1 participant), gallbladder (1 participant), pancreatic (1 participant), small intestine (1 participant), uterine cervix (1 participant) and HER2-low gastric/GEJ (1 participant who received 5.4 mg/kg) cancer.

Secondary Endpoints

Disease-free Survival (DFS) in Participants Who Were Administered Trastuzumab Deruxtecan (T-DXd) Compared With Trastuzumab Emtansine (T-DM1) Treatment
Randomization to date of the first occurrence of an IDFS event including second primary non-breast cancer event or contralateral or ipsilateral ductal carcinoma in situ (whichever occurs first), up to approximately 81 months postdose
Overall Survival (OS) in Participants Who Were Administered Trastuzumab Deruxtecan (T-DXd) Compared With Trastuzumab Emtansine (T-DM1) Treatment
Randomization to date of death from any cause, up to approximately 81 months postdose
Distant Recurrence-free Interval (DRFI) in Participants Who Were Administered Trastuzumab Deruxtecan (T-DXd) Compared With Trastuzumab Emtansine (T-DM1) Treatment
Randomization to date of distant recurrence, up to approximately 81 months postdose
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Study Design & Arms

AllocationRANDOMIZED
MaskingNONE
ModelPARALLEL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Trastuzumab deruxtecan (T-DXd)EXPERIMENTALParticipants who will be randomized to receive trastuzumab deruxtecan (T-DXd) at a starting dose of 5.4 mg/kg.
Trastuzumab ematansine (T-DM1)ACTIVE_COMPARATORParticipants who will be randomized to receive trastuzumab ematansine (T-DM1) at a starting dose of 3.6 mg/kg.
DS-8201a Cohort AEXPERIMENTALCohort A is comprised of participants with HER2-positive (IHC 3+ or IHC 2+/ISH +) who will receive DS-8201a once every 3 weeks
DS-8201a Cohort BEXPERIMENTALCohort B is comprised of participants with HER2 IHC 2+/ISH - who will receive DS-8201a once every 3 weeks
DS-8201a Cohort CEXPERIMENTALCohort C is comprised of participants with HER2 IHC 1+ who will receive DS-8201a once every 3 weeks
Part 1 Dose escalationEXPERIMENTALPart 1 is a dose escalation to identify the Maximum Tolerated dose (MTD) or the recommended phase 2 dose of DS-8201a guided by the modified continuous reassessment method using a Bayesian logistic regression model following escalation with overdose control principal.
Part 2 Dose expansionEXPERIMENTALPart 2 is a dose expansion to examine the safety and efficacy of DS-8201a and it is consist of multiple cohorts: in subjects with trastuzumab emtansine (T-DM1)-treated HER2 overexpressing breast cancer (Part 2a); trastuzumab-treated HER2 overexpressing gastric or gastroesophageal junction adenocarcinoma (Part 2b); HER2 low expressing breast cancer (Part 2c), HER2 expressing other solid malignant tumor (Part 2d); HER2 expressing breast cancer (Japan only; Part 2e)

Interventions

NameTypeDescription
DS-8201aDRUGAdministered initially as an intravenous (IV) infusion at a dose of 5.4 mg/kg on Day 1 of each 21-day cycle
T-DM1DRUGAdministered initially as an intravenous (IV) infusion at a dose of 3.6 mg/kg on Day 1 of each 21-day cycle
DS-8201a (DP1)DRUGDS-8201a to be administered via intravenous (IV) dose. DS-8201a (DP1) was used for the Dose Escalation phase and for Dose expansion Parts 2a, 2b, 2c, and 2d.
DS-8201a (DP2)DRUGDS-8201a is to be administered via intravenous (IV) dose. DS-8201a (DP2) was used only used for Dose Expansion Part 2e.
DS-8201a (DP)DRUGDS-8201a (DP) is to be administered via intravenous (IV) dose.
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Eligibility Criteria

Age Range18 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites482

Key Inclusion Criteria: * Adults ≥18 years old (local regulatory requirements will apply if the legal age of consent for study participation is \>18 years old). * Pathologically documented HER2-positive breast cancer (BC): * HER2-positive expression defined as an immunohistochemistry (IHC) score...

Countries:United StatesArgentinaAustraliaBelgiumBrazilCanadaChileChinaCzechiaDenmarkFranceGermanyGreeceHong KongIrelandIsraelItalyJapanMexicoNetherlandsPeruPolandPortugalRomaniaRussiaSingaporeSouth KoreaSpainTaiwanTurkey (Türkiye)United Kingdom
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Frequently asked questions about DS-8201a

What is DS-8201a used for?

DS-8201a is an investigational oncology drug being studied for colorectal neoplasms, advanced colorectal cancer, advanced solid tumors, and HER2-positive primary breast cancer. It is a small molecule in clinical development for these cancer indications.

Who makes DS-8201a?

DS-8201a is being developed by AstraZeneca PLC, which trades under the ticker AZN. The company is conducting clinical trials to evaluate the drug's safety and efficacy in various cancer types.

What phase is DS-8201a in?

DS-8201a is in Phase 1 clinical development. It is an investigational drug, meaning it has not been approved by regulatory authorities and is still undergoing clinical trials to assess its safety and effectiveness.

What clinical trials is DS-8201a in?

DS-8201a has been studied in several clinical trials, including NCT02564900 for advanced solid tumors, NCT03384940 for HER2-expressing colorectal cancer, NCT04622319 for HER2-positive breast cancer, and NCT04744831 for advanced colorectal cancer.

Is DS-8201a the same as trastuzumab deruxtecan?

DS-8201a is also known as trastuzumab deruxtecan (T-DXd). Clinical trials such as NCT04622319 and NCT04744831 refer to the drug by this alternative name, confirming that DS-8201a and trastuzumab deruxtecan are the same compound.

What is the enrollment for DS-8201a clinical trials?

DS-8201a clinical trials have enrolled a total of 1,600 participants across multiple studies. The largest trial, NCT04622319, has an enrollment of 1,600 participants, while other trials have smaller cohorts ranging from 86 to 292 participants.