Trial results for the Phase 2 study (NCT04601857) investigating the combination of futibatinib and pembrolizumab in advanced or metastatic urothelial carcinoma were posted on ClinicalTrials.gov on 2025-12-31. The study, which was terminated, reported an Objective Response Rate (ORR) of 47.1% for Cohort A and 26.9% for Cohort B.
Background
Pembrolizumab is an anti-programmed cell death-1 (PD-1) antibody, while futibatinib is a Fibroblast Growth Factor Receptor (FGFR) inhibitor. This trial investigated their combination for the treatment of advanced or metastatic urothelial cancer in patients not eligible for platinum-based regimens.
Trial design
The Phase 2 study (NCT04601857) was designed to evaluate the antitumor activity and safety of the combination of futibatinib and pembrolizumab in patients with advanced or metastatic urothelial cancer who were not candidates for platinum-based treatment regimens. The trial enrolled 43 participants and was ultimately terminated. The study included two cohorts, Cohort A and Cohort B, both receiving the combination therapy.
Key results
The trial reported several key outcome measurements for both cohorts:
- Objective Response Rate (ORR): For Cohort A, the ORR was 47.1%. For Cohort B, the ORR was 26.9%.
- Disease Control Rate (DCR): Cohort A achieved a DCR of 82.4%, while Cohort B had a DCR of 53.8%.
- Duration of Response (DOR): The median DOR was 12.32 months for Cohort A and 14.46 months for Cohort B.
- Progression-free Survival (PFS): Median PFS was 8.3 months for Cohort A and 4.1 months for Cohort B.
- Overall Survival (OS): Median OS was 16.2 months for Cohort A and 18.3 months for Cohort B.
- Treatment Emergent Adverse Events (TEAEs): 17 participants in Cohort A and 26 participants in Cohort B experienced TEAEs.
What this means
The termination of this Phase 2 trial suggests that the study did not proceed as initially planned, which can occur for various reasons including safety concerns, lack of efficacy, or strategic decisions. Despite the termination, the reported data indicates that the combination of futibatinib and pembrolizumab demonstrated antitumor activity in patients with advanced or metastatic urothelial carcinoma. The observed objective response rates and disease control rates, along with median progression-free and overall survival, provide insights into the potential of this combination therapy, particularly for patients who are not candidates for platinum-based regimens. Further research or analysis would be needed to understand the specific reasons for termination and the full clinical implications of these results.
Source
The information for these trial results was obtained from ClinicalTrials.gov, a public database of clinical studies. The results for study NCT04601857, titled "Futibatinib and Pembrolizumab Combination in the Treatment of Advanced or Metastatic Urothelial Carcinoma," were posted on 2025-12-31 on clinicaltrials.gov.