Aims: Standard of care for cisplatin-ineligible patients with muscle-invasive bladder cancer (MIBC) is radical cystectomy + pelvic lymph node dissection (RC+PLND), but patients experience high rates of disease recurrence and poor overall survival (OS). The PD-1 inhibitor pembrolizumab has shown promising antitumor activity in MIBC as monotherapy in the neoadjuvant setting and in combination with the nectin 4–directed antibody-drug conjugate enfortumab vedotin (EV) in metastatic urothelial carcinoma. KEYNOTE‑905/EV‑303, a randomized phase 3 study (NCT03924895), will investigate the efficacy and safety of perioperative pembrolizumab monotherapy with RC+PLND versus perioperative EV+pembrolizumab with RC+PLND versus RC+PLND alone for cisplatin-ineligible patients with MIBC.
Methods: Cisplatin-ineligible adults with confirmed treatment-naive MIBC (T2-T4aN0M0 or T1-T4aN1M0), ECOG PS score 0-2, and predominant (≥50%) urothelial histology are eligible to enroll. Patients will be stratified by PD-L1 status (combined positive score [CPS] ≥10 vs <10), region (United States vs Europe vs most of the world), and disease stage (T2N0 vs T3/T4N0 vs T1-T4aN1). Approximately 836 patients will be randomly assigned 1:1:1 to receive neoadjuvant pembrolizumab (3 cycles) before RC+PLND and adjuvant pembrolizumab (14 cycles) in arm A, RC+PLND alone followed by observation in arm B, or neoadjuvant EV+pembrolizumab (3 cycles) before RC+PLND and adjuvant EV+pembrolizumab (6 cycles) and adjuvant pembrolizumab (8 cycles) in arm C. Pembrolizumab 200 mg IV Q3W will be administered in both the neoadjuvant and the adjuvant phases of arm A and the adjuvant phase of arm C. In arm C, pembrolizumab 200 mg IV Q3W will be administered on day 1 of each cycle, and EV 1.25 mg/kg will be administered on days 1 and 8 of each cycle in both phases. Coprimary end points are event-free survival and pathologic complete response in all patients and in patients with PD-L1 CPS ≥10. Secondary end points are OS, disease-free survival, pathologic downstaging, and safety.