Sponsor: Daiichi Sankyo (industry)
Phase: 2
Start date: Feb. 26, 2024
Planned enrollment: 740
HER3-DXd (patritumab deruxtecan; U3-1402/MK-1022) is an investigational antibody–drug conjugate being studied in HER3-expressing solid tumors, particularly EGFR-mutated non-small-cell lung cancer (NSCLC) and breast cancer. It remains investigational: a US application for accelerated approval in previously treated EGFR-mutated NSCLC was withdrawn in May 2025 after the confirmatory phase 3 trial did not significantly improve overall survival. (merck.com)
HER3-DXd combines:
After binding HER3, the conjugate is internalized and releases DXd, causing DNA damage and cell death. Preclinical evidence also suggests that the payload can induce features of immunogenic cell death. Clinical responses have occurred across different levels of HER3 expression, so HER3 immunohistochemistry has not yet been established as a definitive predictive biomarker. (pmc.ncbi.nlm.nih.gov)
EGFR-mutated NSCLC: In the single-arm phase 2 HERTHENA-Lung01 trial, 225 evaluable patients previously treated with an EGFR inhibitor and platinum chemotherapy had a confirmed objective response rate (ORR) of 29.8%, median response duration of 6.4 months, median progression-free survival (PFS) of 5.5 months, and median overall survival of 11.9 months. (pmc.ncbi.nlm.nih.gov)
In the randomized phase 3 HERTHENA-Lung02 trial after third-generation EGFR-inhibitor therapy, HER3-DXd improved median PFS modestly versus platinum-based chemotherapy—5.8 versus 5.4 months; hazard ratio 0.77. ORR was 35.2% versus 25.3%, respectively. However, overall survival was not significantly improved, which contributed to withdrawal of the US regulatory application. (ascopubs.org)
Breast cancer: A phase 1/2 study in heavily pretreated metastatic disease reported ORRs of 30.1% in HR-positive/HER2-negative breast cancer, 22.6% in triple-negative disease, and 42.9% in HER2-positive disease, although the HER2-positive cohort contained only 14 patients. (pmc.ncbi.nlm.nih.gov)
In the subsequent single-arm phase 2 ICARUS-BREAST01 trial of 99 patients with HR-positive/HER2-negative advanced breast cancer, ORR was 53.5% by investigator assessment and 55.6% by retrospective independent review. Median PFS was 9.2 months and median response duration was 9.3 months; randomized confirmation is still needed. (nature.com)
The main toxicities are gastrointestinal and hematologic, including nausea, fatigue, diarrhea, thrombocytopenia, neutropenia and anemia. Interstitial lung disease/pneumonitis is an important identified risk and has included rare fatal cases.
In HERTHENA-Lung02, grade 3 or higher treatment-emergent adverse events occurred in 73% with HER3-DXd versus 57% with chemotherapy, driven partly by grade 3 or higher thrombocytopenia (30% versus 7.9%). (ascopubs.org)
In ICARUS-BREAST01, grade 3 or higher treatment-related events occurred in 50.1% of patients. The most frequent treatment-related events were fatigue (83%), nausea (75%), diarrhea (53%) and alopecia (40%). Eight patients had independently adjudicated drug-related interstitial lung disease, predominantly grade 1 or 2. (nature.com)
Last updated: Sep 2026
Goal: Evaluate the antitumor activity and safety of patritumab deruxtecan (HER3-DXd) across previously treated, locally advanced unresectable or metastatic solid tumors, and explore whether tumor HER3 expression is associated with benefit.
Patients: Adults with ECOG performance status 0–1 and refractory disease in tumor-specific cohorts spanning melanoma, head and neck, HER2-negative gastric or gastroesophageal junction, ovarian, cervical, endometrial, urothelial, esophageal, pancreatic, prostate, nonsquamous non-small-cell lung, and HR-positive/HER2-negative breast cancers. Prior-treatment requirements vary by cohort. Pretreatment tumor tissue is required; patients with prior clinically significant interstitial lung disease or pneumonitis, prior anti-HER3 therapy, or prior exatecan-derivative antibody–drug conjugates are excluded.
Design: Multicenter, multicohort, open-label, single-arm proof-of-concept study. Efficacy is assessed separately by tumor type; there is no randomization or control arm.
Treatments: All participants receive HER3-DXd 5.6 mg/kg by intravenous infusion every 3 weeks. This investigational antibody–drug conjugate targets HER3 and delivers an exatecan-derived topoisomerase I inhibitor payload after cellular uptake. In separate studies, objective response rates were approximately 30% in previously treated EGFR-mutated NSCLC and 54% in HR-positive/HER2-negative advanced breast cancer; a randomized NSCLC trial did not demonstrate a significant overall-survival benefit. Hematologic toxicity and interstitial lung disease or pneumonitis are important risks.
Outcomes: The primary endpoint is investigator-assessed confirmed RECIST v1.1 objective response rate in all cohorts except prostate cancer, for which it is the proportion achieving a PSA decline of at least 50%. Secondary measures include adverse events, response duration, disease control and clinical benefit, progression-free and overall survival, pharmacokinetics, and the association of tumor HER3 expression with efficacy. Prostate-specific measures include radiographic progression-free survival, PSA decline of at least 30%, time to subsequent therapy, and symptomatic skeletal events. Follow-up for most outcomes extends to approximately 27 months.
Burden on patient: Moderate to high. Treatment requires travel for intravenous infusion every 3 weeks, serial disease assessments and blood tests, and a qualifying pretreatment tumor sample that may require a new biopsy. Pharmacokinetic sampling adds multiple blood draws around infusions and on days 8 and 15 of cycles 1 and 3. Monitoring for cytopenias and interstitial lung disease may add clinical assessments.
Last updated: Sep 2026
Inclusion Criteria
Participants must meet all of the following criteria to be eligible for enrollment into the study:
1. Sign and date the informed consent form prior to the start of any study-specific qualification procedures. A separate tissue screening consent will be obtained from all subjects to meet the baseline tumor tissue requirement.
2. Participants aged ≥18 years (follow local regulatory requirements if the legal age of consent for study participation is \>18 years old).
3. Has locally advanced unresectable or metastatic disease (not curable by surgery or radiation) as follows:
Cutaneous (acral and non-acral) melanoma
1. Histologically or cytologically confirmed cutaneous (acral or non-acral) melanoma
2. Disease progression while on or after having received treatment with ≥1 prior line of anti-programmed cell death protein (PD-1) or anti-programmed death-ligand 1 (PD-L1) based therapy (previous use of other immune checkpoint inhibitors \[ICIs\] \[ie, anti-CTLA4, anti- LAG-3\] is acceptable). Prior anti-PD-(L)1 therapy in the adjuvant setting is allowed if there is recurrence within 12 weeks of the last dose. If the participant had BRAFm melanoma, they must have had disease progression on BRAF/MEK inhibitor therapy as well.
Squamous cell carcinomas of the head and neck
3. Squamous cell carcinoma of the head and neck (with a primary location of oral cavity,oropharynx, larynx, hypopharynx) that is human papillomavirus (HPV) positive or negative (as determined by local standard). Excludes tumor location in the nasopharynx, nasal cavity, paranasal sinuses, and unknown primary locations.
4. Disease progression after having received treatment with ≥1 and \<3 prior lines of systemic therapy in the unresectable recurrent or metastatic setting.
Must have had disease progression on anti-PD-(L)1 (either as monotherapy or in combination with chemotherapy or other therapies). Must also have had disease progression on a platinum-based chemotherapy (PBC) regimen either in the recurrent or metastatic setting or in the locally advanced setting with curative intent.
Gastric or GEJ adenocarcinoma
5. Tumor tissue must be confirmed as negative for HER2 expression (immunohistochemistry \[IHC\] 0/1+ or IHC 2+/in situ hybridization negative) as classified by American Society of Clinical Oncology/College of American Pathologists (ASCO-CAP) guidelines and determined prior to enrollment by assessment in a local laboratory that is Clinical Laboratory Improvement Amendments certified (US sites) or accredited based on specific country regulations.
6. Disease progression after having received treatment with ≥2 prior lines of therapy that include PBC with or without anti-PD-1 therapy.
Ovarian Carcinoma
7. Pathologically documented high-grade serous epithelial ovarian cancer, primary peritoneal cancer, or fallopian tube cancer.
8. Documented disease progression ≥4 weeks after the last dose of PBC and \<6 months of last dose of PBC in the advanced or metastatic setting. Prior use of folate reductase alpha targeting antibody-drug conjugate (ADC) (ie, mirvetuximab soravtansine) is allowed.
Cervical Cancer
9. Pathologically or cytologically documented recurrent or persistent squamous, adenosquamous, or adenocarcinoma of the uterine cervix.
10. Disease progression after having received ≥1 line of systemic therapy in the recurrent or metastatic setting. This may include prior anti-PD-(L)1 treatment and/or tissue factor directed ADC (tisotumab vedotin \[TV\]) per regional standard of care.
Endometrial Cancer
11. Pathologically or cytologically documented endometrial cancer (carcinoma of any histological sub-type or endometrial carcinosarcoma), irrespective of microsatellite instability (MSI) or mismatch repair (MMR) status.
12. Documented disease progression after having received ≥1 prior line of therapy (maximum of 3) PBC containing systemic treatment and an anti-PD(L)-1 therapy-containing regimen (combined or sequential) in the advanced/metastatic setting.
Bladder Cancer
13. Pathologically or cytologically documented locally advanced/unresectable or metastatic urothelial carcinoma of the bladder, renal pelvis, ureter, or urethra. Histological variants are allowed if urothelial histology is predominant. Small cell/neuroendocrine tumors are not allowed even if mixed histology.
14. Relapsed or progressed after treatment with ≥1 prior line of therapy (maximum of 3) that contains anti-PD-(L)1 therapy in the perioperative or metastatic setting. At least 1 line of therapy must also contain one of the following treatment modalities: chemotherapy or enfortumab vedotin. Prior fibroblast growth factor receptor (FGFR)-inhibitor treatment for those who are eligible are allowed.
* Required treatments can be given in combination or sequentially
* Prior cisplatin-based therapy or PD-(L)1 inhibitor therapy given for the treatment of muscle invasive urothelial carcinoma is counted as 1 line of therapy
* The same regimen administered twice in different disease settings will be counted as 1 line of prior therapy
* Participants in the second-line setting who have previously received enfortumab vedotin and pembrolizumab in combination can be enrolled.
Esophageal Carcinoma
15. Pathologically or cytologically documented esophageal squamous cell carcinoma.
16. Must have documented disease progression after having received 2 prior lines of therapy including previous PBC with or without an anti-PD-1 therapy-containing regimen (combined or sequential) in the advanced/metastatic setting.
Pancreatic Carcinoma
17. Pathologically or cytologically documented unresectable or metastatic pancreatic adenocarcinoma.
18. Relapsed or disease progression after having received 1 prior line of systemic therapy in the locally advanced/metastatic setting.
Prostate Cancer
19. Pathologically or cytologically documented unresectable locally advanced or metastatic castration-resistant prostate cancer (CRPC).
20. Adenocarcinoma of the prostate without neuroendocrine differentiation or small cell histology.
21. Surgically or medically castrated, with testosterone levels of \<50 ng/dL.
22. Documented objective progression as determined by radiographic progression for subjects with measurable disease after androgen deprivation.
23. Relapsed or disease progression after having received treatment with ≥1 of the following novel hormonal agents: abiraterone, enzalutamide, apalutamide, or darolutamide.
24. Relapsed or disease progression after having received ≥1 cytotoxic chemotherapy regimen that included a taxane.
Gastric Cancer 2L
25. Must have had gastric or GEJ adenocarcinoma confirmed as negative for HER2 expression (IHC 0/1+ or IHC 2+/in situ hybridization negative) as classified by American Society of Clinical Oncology/College of American Pathologists (ASCO-CAP) guidelines and determined prior to enrollment by assessment in a local laboratory that is Clinical Laboratory Improvement Amendments certified (US sites) or accredited based on specific country regulations.
26. Disease progression after having received treatment with only 1 prior line of systemic anti-cancer therapy that includes 5-FU-based chemotherapy with or without an anti-PD-1 therapy. For subjects whose tumors are claudin (CLDN) 18.2 positive, treatment with 5-FU based chemotherapy with CLDN18.2 directed therapy in the first-line setting is allowed.
Non-small Cell Lung Cancer aa. Histologically or cytologically documented metastatic or locally advanced nonsquamous NSCLC not amenable to curative surgery or radiation bb. Documentation of absence of actionable driver mutation (ie, ALK rearrangement, BRAF V600E mutation, EGFR-activating mutations \[exon 19 deletion or L858R mutation\], EGFR exon 20 insertion mutation, HER2 mutation, KRAS G12C mutation, MET exon 14 skipping mutation, NTRK 1/2/3 gene fusion, RET rearrangement, or ROS1 rearrangement). New testing for these genomic alterations is not required for Screening.
cc. Relapsed or disease progression after receiving only anti-PD-(L)1 and PBC (ie, platinum doublet) administered in combination or sequentially for metastatic disease.
Breast Cancer dd. Pathologically documented breast cancer that is assessed as HER2 negative (IHC2+/ISH-, IHC1+, or IHC0 per ASCO/CAP guidelines), and HR positive (either ER and/or PgR positive \[ER or PgR ≥1%\] per ASCO/CAP guidelines). The HER2 and HR results must be from a tumor sample obtained in the metastatic setting.
ee. Participant must have received one line of chemotherapy for mBC, but not more than one line and must have a clinically or radiologically documented evidence of tumor progression on or after CDK 4/6 inhibitor combined with endocrine therapy; previous treatments with phosphoinositide 3-kinase (PI3K) inhibitors, mammalian target of rapamycin (mTOR) inhibitors, protein kinase B (PKB) inhibitors also known as AKT-inhibitors and poly ADP ribose polymerase (PARP)-inhibitors are allowed.
4. Has ≥1 measurable lesion on CT or MRI as per RECIST v1.1 by investigator assessment. Prostate cancer participants with bone only disease may be eligible.
5. Provides a pretreatment tumor tissue sample that meets 1 of the following collection requirements:
1. Tumor biopsy from ≥1 lesion not previously irradiated and performed since progression with the most recent systemic cancer therapy regimen and prior to signature of the tissue ICF (ARCHIVAL PRETREATMENT sample).
OR
2. Newly obtained pretreatment tumor biopsy from ≥1 lesion not previously irradiated and amenable to sampling, after signature of tissue ICF (FRESH PRETREATMENT sample)
6. Has Eastern Cooperative Oncology Group performance status (ECOG PS) of 0 or 1 at screening.
Exclusion Criteria
Participants who meet any of the following criteria will be disqualified from entering the study:
1. Has HER2-positive gastric cancer as classified by ASCO-CAP guidelines and determined prior to enrollment by assessment in a local laboratory that is Clinical Laboratory Improvement Amendments certified (US sites) or accredited based on specific country regulations.
2. Has nasopharyngeal cancer.
3. Has mucosal or uveal melanoma.
4. Has a history of (non-infectious) interstitial lung disease (ILD), that required corticosteroids, has current ILD/pneumonitis, or suspected ILD/pneumonitis cannot be ruled out by imaging at screening.
5. Has clinically severe respiratory compromise (based on the investigator's assessment) resulting from intercurrent pulmonary illnesses
6. Is receiving chronic systemic corticosteroids dosed at \>10 mg prednisone daily or equivalent anti-inflammatory activity or any form of immunosuppressive therapy prior to Cycle 1 Day 1.
Participants who require use of bronchodilators, inhaled or topical steroids, or local steroid injections may be included in the study.
7. Had prior treatment with an anti-HER3 antibody and/or antibody-drug conjugate (ADC) that consists of an exatecan derivative that is a topoisomerase I inhibitor (eg, trastuzumab deruxtecan).
8. Has history of other active malignancy within 3 years prior to Cycle 1 Day 1, except the following:
1. Adequately treated nonmelanoma skin cancer
2. Adequately treated intraepithelial carcinoma of the cervix
3. Any other curatively treated in situ disease
9. Has any evidence of severe or uncontrolled diseases (eg, active bleeding diatheses, active serious infection) psychiatric illness/social situations, geographical factors, substance abuse, or other factors that, in the investigator's opinion, make it high risk for the subject to participate in the study or that would jeopardize compliance with the protocol
10. Has previously received topoisomerase-1 inhibitors (e.g., irinotecan) treatment in the advanced or metastatic disease setting.
Camperdown, 2050, Australia
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Chermside, 4032, Australia
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Hobart, 7000, Australia
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Hyde Park, 4812, Australia
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Clayton, 3168, Australia
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Brussels, Belgium
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Leuven, 3000, Belgium
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Edegem, 2650, Belgium
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Jette, Belgium
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Ghent, 9000, Belgium
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Vancouver, V5Z4E6, Canada
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Toronto, M5G2M9, Canada
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Toronto, M4N 3M5, Canada
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Edmonton, Alberta, T6G 1Z2, Canada
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Dijon, 21079, France
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Lyon, 69008, France
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Bordeaux, 33000, France
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Nantes, 44093, France
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Lille, 59000, France
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Marseille, 13005, France
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Toulouse, 31100, France
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Villejuif, 94805, France
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Kecskemét, 6000, Hungary
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Napoli, 80131, Italy
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Verona, 37134, Italy
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Genova, 16132, Italy
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Nagoya, 464-8681, Japan
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Tokyo, 135-8550, Japan
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Osakasayama-shi, 589-8511, Japan
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Matsuyama, 791-0245, Japan
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Tokyo, 104-0045, Japan
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Kashiwa-shi, 277-8577, Japan
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Hidaka, 350-1298, Japan
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Nagaizumi-cho, 411-8777, Japan
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Yokohama, 232-0024, Japan
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Amsterdam, 1081 HV, Netherlands
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Leiden, 2333 ZG, Netherlands
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Maastricht, 6229 HX, Netherlands
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Nijmegen, 6525 GA, Netherlands
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Groningen, 9713 GZ, Netherlands
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Lørenskog, 1478, Norway
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Lørenskog, 1478, Norway
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Oslo, 0379, Norway
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Seoul, 05505, South Korea
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Seongnam, 13496, South Korea
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Seoul, 06351, South Korea
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Seongnam, 13620, South Korea
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Seoul, 03080, South Korea
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Seoul, 03722, South Korea
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Málaga, 29010, Spain
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Barcelona, 08036, Spain
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Valencia, 46010, Spain
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Madrid, 28009, Spain
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Barcelona, 08035, Spain
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Madrid, 28041, Spain
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Madrid, 28034, Spain
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Seville, 41009, Spain
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Barcelona, 08041, Spain
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Taoyuan District, 333, Taiwan
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Kaohsiung City, 833, Taiwan
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Tainan, 704, Taiwan
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Taipei, 100225, Taiwan
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Taipei, 11217, Taiwan
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London, EC1A 7BE, United Kingdom
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Nottingham, NG5 1PB, United Kingdom
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London, NW3 2QG, United Kingdom
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Coventry, CV2 2DX, United Kingdom
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Duarte, California, 91010, United States
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New Haven, Connecticut, 06510, United States
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Kissimmee, Florida, 34747, United States
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Chicago, Illinois, 60612, United States
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Baltimore, Maryland, 21205, United States
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Saint Paul, Minnesota, 55101, United States
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Saint Louis Park, Minnesota, 55426, United States
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St Louis, Missouri, 63110, United States
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New York, New York, 10065, United States
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Buffalo, New York, 14203, United States
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Nashville, Tennessee, 37203, United States
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Houston, Texas, 77030, United States
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Seattle, Washington, 98109, United States
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