Sponsor: Daiichi Sankyo (industry)
Phase: 3
Start date: March 27, 2025
Planned enrollment: 510
Ifinatamab deruxtecan (I-DXd), also known as MK-2400 or DS-7300, is an investigational antibody-drug conjugate (ADC) developed by Daiichi Sankyo and Merck. It targets B7-H3, a transmembrane immunoregulatory protein overexpressed in various cancers, including small cell lung cancer (SCLC). (daiichisankyo.us)
I-DXd comprises a humanized anti-B7-H3 monoclonal antibody linked to a topoisomerase I inhibitor payload. Upon binding to B7-H3-expressing tumor cells, the ADC is internalized, releasing the cytotoxic agent to induce DNA damage and cell death. (daiichisankyo.us)
In a phase 1/2 trial involving heavily pretreated patients with advanced SCLC, I-DXd demonstrated promising efficacy:
I-DXd was generally well tolerated. Common treatment-emergent adverse events (TEAEs) included nausea (59.1%), fatigue (50.0%), anemia (27.3%), vomiting (27.3%), and decreased appetite (22.7%). Grade 3 or higher TEAEs occurred in 36.4% of patients. Notably, 13.6% experienced interstitial lung disease or pneumonitis, with one grade 2 case leading to treatment discontinuation. (targetedonc.com)
Last updated: Apr 2025
Goal: To determine whether ifinatamab deruxtecan (I-DXd), a B7-H3–targeted antibody–drug conjugate, improves overall survival compared with investigator’s choice chemotherapy in previously treated unresectable or metastatic esophageal squamous cell carcinoma (ESCC). Secondary goals include assessing progression-free survival and objective response rate.
Patients: Adults (≥18 years) with histologically or cytologically confirmed unresectable locally advanced or metastatic ESCC per AJCC 8th edition, who have progressed after exactly one prior systemic regimen that included a platinum-based chemotherapy and an immune checkpoint inhibitor. Patients must have ECOG performance status 0–1, at least one measurable lesion per RECIST v1.1, and provide adequate baseline tumor tissue. Key exclusions include prior exposure to B7-H3 agents or topoisomerase I inhibitors, adenosquamous histology, lack of eligibility for all comparator chemotherapies, high-risk local invasion (e.g., aorta or airway), active or symptomatic CNS disease, recent major thromboembolic or cerebrovascular events, significant corneal disease, history or current interstitial lung disease/pneumonitis, clinically severe pulmonary compromise, and chronic systemic steroid use at ≥10 mg prednisone equivalent daily.
Design: Multicenter, randomized, open-label phase 3 trial with two parallel arms comparing I-DXd versus investigator’s choice chemotherapy. Approximately 510 participants will be randomized. Efficacy assessments will use blinded independent central review for radiographic endpoints.
Treatments: I-DXd: Ifinatamab deruxtecan 12 mg/kg IV every 3 weeks. I-DXd is an investigational antibody–drug conjugate targeting B7-H3 (CD276), a transmembrane immune checkpoint–related protein broadly expressed in solid tumors with limited normal tissue expression. The ADC consists of a humanized anti–B7-H3 IgG1 linked via a cleavable tetrapeptide linker to a topoisomerase I inhibitor payload (DXd, an exatecan derivative), delivering cytotoxic payload selectively to B7-H3–expressing tumor cells. In pretreated extensive-stage small cell lung cancer, I-DXd has shown objective response rates around 55% at 12 mg/kg with median PFS approximately 5.5 months and median OS approximately 11.8 months; interstitial lung disease/pneumonitis has been observed in roughly 9–12% in those studies. Comparator: Investigator’s choice of standard IV chemotherapy with docetaxel, paclitaxel, or irinotecan.
Outcomes: Primary endpoint is overall survival. Key secondary endpoints are progression-free survival by blinded independent central review per RECIST v1.1 and objective response rate. Additional secondary endpoints include duration of response, disease control rate, patient-reported outcomes using EORTC QLQ-C30 and OES18, safety and tolerability including TEAEs, serious TEAEs, and adverse events of special interest graded by CTCAE v5.0, immunogenicity via anti-drug antibodies, and pharmacokinetics for I-DXd, total anti–B7-H3 antibody, and released payload (MAAA-1181a). Assessments are planned up to approximately 54 months.
Burden on patient: Moderate to high. Patients will receive IV therapy every 3 weeks, similar to standard second-line regimens, but the investigational arm includes intensive pharmacokinetic sampling in early cycles (multiple blood draws across hours to days in cycle 1, and repeated sampling in cycles 2–5 and every other cycle thereafter), which increases visit time and needle sticks. Baseline tumor tissue is required, potentially necessitating a new biopsy if archival material is insufficient. Regular imaging per RECIST with blinded central review is expected, comparable to standard-of-care frequency, but the long follow-up period (up to 54 months) adds ongoing visits. Safety monitoring is rigorous, with particular attention to pulmonary symptoms due to ILD/pneumonitis risk and ocular assessments for corneal disease, which may require additional evaluations and prompt intervention. Travel burden will depend on site proximity but is typical for a phase 3 IV regimen with added PK and safety surveillance early on.
Inclusion Criteria:
Participants must meet all of the following criteria to be eligible for randomization into the study:
1. Participants aged ≥18 years (follow local regulatory requirements if the legal age of consent for study participation is \>18 years old).
2. Has histologically or cytologically documented unresectable locally advanced or metastatic ESCC according to American Joint Committee on Cancer 8th edition staging system on ESCC.
3. Has disease progression post a platinum-based chemotherapy and an ICI treatment per global or local guidelines, with a maximum of 1 prior line of systemic therapy for unresectable advanced or metastatic ESCC.
4. The participant must provide adequate baseline tumor samples with sufficient quantity and quality of tumor tissue content as defined in the Laboratory Manual.
5. Has at least 1 measurable lesion on computed tomography (CT)/magnetic resonance imaging (MRI) according to RECIST v1.1 as assessed by the investigator. Measurable lesions should not be from a previously irradiated site. If the lesion at a previously irradiated site is the only selectable target lesion, a radiological assessment showing significant progression of the irradiated lesion should be provided by the investigator.
6. Has an ECOG PS of 0 or 1 within 7 days prior to Cycle 1 Day 1.
Exclusion Criteria:
Participants who meet any of the following criteria will be disqualified from entering the study:
1. Has received prior treatment with orlotamab, enoblituzumab, or other B7-H3 targeted agents, including I-DXd.
2. Has received any topoisomerase inhibitor.
3. Has histologically or cytologically confirmed adenosquamous carcinoma subtype.
4. Is ineligible to all the chemotherapies in the comparator arm due to prior progression or intolerance.
5. Has tumor invasion into organs located adjacent to the esophageal disease site (eg, aorta or respiratory tract) at an increased risk of bleeding or fistula as assessed by the investigator.
6. Clinically active brain metastases, spinal cord compression, or leptomeningeal carcinomatosis, defined as untreated or symptomatic, or requiring therapy with steroids or anticonvulsants to control associated symptoms. Subjects with clinically inactive or treated brain metastases who are asymptomatic (ie, without neurologic signs or symptoms and not requiring treatment with corticosteroids or anticonvulsants) may be included in the study. Subjects must have a stable neurologic status and discontinue corticosteroid usage for at least 2 weeks prior to Screening.
7. Has any of the following within the past 6 months: cerebrovascular accident, transient ischemic attack, other arterial thromboembolic event, or pulmonary embolism.
8. Has a clinically significant corneal disease.
9. Has a history of (non-infectious) interstitial lung disease (ILD)/pneumonitis that required corticosteroids, current ILD/pneumonitis, or suspected ILD/pneumonitis that cannot be ruled out by imaging at Screening.
10. Has clinically severe pulmonary compromise resulting from intercurrent pulmonary illnesses, including, but not limited to, any underlying pulmonary disorder (ie, pulmonary emboli within 3 months of the study randomization, severe asthma, chronic obstructive pulmonary disease \[COPD\], restrictive lung disease, pleural effusion, etc), and potential pulmonary involvement caused by any autoimmune, connective tissue, or inflammatory disorders (eg, rheumatoid arthritis, Sjögren's syndrome, sarcoidosis, etc), prior pneumonectomy, or requirement for supplemental oxygen.
11. Is on chronic steroid treatment (dose of 10 mg daily or more prednisone equivalent), except for low-dose inhaled steroids (for asthma/COPD), topical steroids (for mild skin conditions), or intra-articular steroid injections.
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