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Phase I/II Study of AXL-Specific Antibody-Drug Conjugate Enapotamab Vedotin in Patients With Advanced Solid Tumors
Journal article   Open access   Peer reviewed

Phase I/II Study of AXL-Specific Antibody-Drug Conjugate Enapotamab Vedotin in Patients With Advanced Solid Tumors

Kristoffer Staal Rohrberg, Juanita S Lopez, Mohammed M Milhem, Christian U Blank, Irene Reijers, Fiona Thistlethwaite, Ruth Plummer, Sarina A Piha-Paul, Pasi A Janne, Elaine Shum, …
Cancer research communications, Vol.5(11), pp.2066-2078
11/2025
DOI: 10.1158/2767-9764.CRC-25-0359
PMCID: PMC12648153
PMID: 41166388
url
https://doi.org/10.1158/2767-9764.CRC-25-0359View
Published (Version of record) Open Access

Abstract

AXL, a receptor tyrosine kinase related to oncogenic processes, is aberrantly expressed in various cancers and associated with treatment resistance. Enapotamab vedotin (EnaV), a novel anti-AXL human IgG1 and monomethyl auristatin E antibody-drug conjugate, demonstrated antitumor activity in preclinical models including non-small cell lung cancer (NSCLC). This phase 1/2 study assessed safety and preliminary efficacy of EnaV in solid tumors. This study comprised dose-escalation and dose-expansion phases; both phases investigated EnaV once every 3 weeks (Q3W), and EnaV on days 1, 8, and 15 of a 28-day cycle (3Q4W). Primary objectives determined maximum tolerated dose (MTD) (dose escalation) and safety (dose expansion). Pharmacokinetic profile, antitumor activity, and AXL expression were also assessed. During dose escalation, 32 patients received EnaV Q3W; 15 received EnaV 3Q4W. MTD and recommended phase 2 dose were 2.2 mg/kg in Q3W and 1.0 mg/kg in 3Q4W schedules. In dose expansion, 189 patients received EnaV Q3W; 70 received EnaV 3Q4W. Common adverse events in dose expansion included fatigue, constipation, nausea, decreased appetite, and diarrhea. Overall response rates ranged from 4.5-12.5% with Q3W schedule and from 9.1-11.5% with 3Q4W dose schedule. Disease control rates for NSCLC cohorts were 40.9-50.0%. NSCLC subset analysis demonstrated correlation between radiomics signature and disease control. The relationship between clinical activity and AXL expression was not apparent. EnaV had an acceptable safety profile; however, because the evaluation of antitumor activity did not show clinically meaningful responses, clinical development of EnaV was discontinued.

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