ASCO GU 2024: Interim Analysis of a Phase I/Ib Study of Enfortumab Vedotin plus Cabozantinib in Patients with Metastatic Urothelial Carcinoma

(UroToday.com) The 2024 American Society of Clinical Oncology Genitourinary (ASCO GU) cancers symposium held in San Francisco, CA between January 25th and 27th was host to a urothelial carcinoma poster session. Dr. Jacqueline Brown presented an interim analysis of a phase I/Ib study of enfortumab vedotin plus cabozantinib in patients with metastatic urothelial carcinoma.

Despite recent major breakthroughs, metastatic urothelial carcinoma remains a relatively deadly disease with a highest reported median overall survival of 31.5 months with enfortumab vedotin + pembrolizumab. Enfortumab vedotin is an antibody-drug conjugate consisting of a fully humanized monoclonal antibody directed against the extracellular domain of Nectin-4, conjugated to a microtubule-disrupting agent, monomethyl auristatin E (MMAE) via a protease-cleavable linker. Cabozantinib is a tyrosine kinase inhibitor (TKI) that inhibits VEGF, MET, and AXL with activity in heavily-pretreated urothelial carcinoma. Preclinical data suggest that anti-angiogenic agents may increase the tumoral cell penetration of antibody-drug conjugates, potentially synergizing their therapeutic efficacy. In this report, Dr. Brown and colleagues reported the safety and preliminary efficacy data from the dose escalation cohort of an ongoing phase I/lb trial investigating cabozantinib + enfortumab vedotin for metastatic urothelial carcinoma patients.

This trial included patients with metastatic urothelial carcinoma who received or were ineligible for platinum-based chemotherapy and immune checkpoint inhibitors. The phase 1 dose escalation cohort employed a 3+3 design exploring cabozantinib 20 mg or 40 mg daily plus standard dose enfortumab vedotin (1.25 mg/kg on days 1, 8, and 15 of a 28-day cycle). The phase 1B dose expansion cohort is currently enrolling at cabozantinib 20 mg daily and will be reported in the future.

The primary endpoint of the phase I cohort was safety and tolerability to determine the recommended phase 2 dose. The key secondary endpoint was objective response rate (ORR) per RECIST v1.1. 

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To date, six patients (all male) have been enrolled. All patients had received prior chemotherapy (either gemcitabine/cisplatin or ddMVAC) and immune checkpoint inhibitors.

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With regard to safety outcomes, one serious adverse event occurred in the 20 mg cohort (dehydration). At the 40 mg dosing, three treatment-related serious adverse events occurred (neutropenia, acute kidney injury, superior vena cava syndrome). Neutropenia was the only grade 4 adverse event and drug-limiting toxicity observed. Cabozantinib 20 mg daily was selected as the recommended phase 2 dose, based on clinical judgment regarding longer-term tolerability. 

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With regards to efficacy outcomes, all six patients in the dose escalation cohort achieved a partial response as their best overall response. The median time to respond was 4.6 months.

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Presented by: Jacqueline T. Brown, MD, Assistant Professor, Department of Hematology and Medical Oncology, Winship Cancer Institute of Emory University, Atlanta, GA

Written by: Rashid Sayyid, MD, MSc – Society of Urologic Oncology (SUO) Clinical Fellow at The University of Toronto, @rksayyid on Twitter during the 2024 American Society of Clinical Oncology Genitourinary (ASCO GU) Cancers Symposium, San Francisco, CA, January 25th – January 27th, 2024