Erdafitinib intravesical drug-releasing system shows early activity in FGFR-altered NMIBC

An investigational intravesical drug-releasing system delivering erdafitinib directly to the bladder (Erda-iDRS) demonstrated encouraging early clinical activity and a manageable safety profile in patients with FGFR–altered non–muscle-invasive bladder cancer (NMIBC), according to final results from a first-in-human phase 1 study.¹

The results were presented by Antoni Vilaseca Cabo, MD, PhD, MSc, at the 41st Annual Congress of the European Association of Urology in London, UK.¹

“Intermediate-risk non–muscle-invasive bladder cancer is defined by recurrences, and many patients undergo repeated procedures as their tumors return,” explained Vilaseca, of Hospital Clínic de Barcelona, in a news release on the results.² “In this study, treatment with Erda-iDRS led most patients with FGFR-altered disease to achieve a complete response by the end of the second treatment cycle, and many of those responses were sustained over time. Achieving and maintaining a complete response is particularly meaningful in this setting, where recurrence is common and requires repeated surgical intervention.”

The open-label, multicenter phase 1 study evaluated the safety, pharmacokinetics, and preliminary efficacy of Erda-iDRS (formerly TAR-210) in patients with NMIBC harboring FGFR alterations identified in tumor tissue or urine cell-free DNA. Erdafitinib is an oral pan-FGFR tyrosine kinase inhibitor indicated for the treatment of locally advanced or metastatic urothelial carcinoma with susceptible FGFR3 or FGFR2 alterations after progression on prior systemic therapy. The drug-releasing system is designed to continuously deliver erdafitinib locally to the bladder over a 3-month treatment cycle. The device is inserted via a urinary placement catheter during an in-office procedure.

The study enrolled patients across 2 disease cohorts. Cohort 1 included patients with high-risk NMIBC, defined as recurrent, BCG-experienced, high-grade Ta/T1 papillary tumors only (no carcinoma in situ) following complete transurethral resection of bladder tumor (TURBT). Cohort 3 included patients with intermediate-risk NMIBC, which was defined as patients with recurrent low-grade Ta or T1 tumors with visible target lesions prior to treatment.

The primary end point was safety. Secondary end points included pharmacokinetics, recurrence-free survival (RFS) in the high-risk cohort, and complete response (CR) rate and duration of CR in the intermediate-risk cohort. Treatment was administered for up to 12 months, with responses assessed every 3 months.

As of the data cutoff on November 3, 2025, a total of 88 patients had received treatment: 26 with high-risk disease and 62 with intermediate-risk disease. The median duration of treatment was 11.9 months (range, 3 to 15) in cohort 1 and 11.8 months (range, 0.1 to 15) in cohort 3.

According to Vilaseca, treatment was well-tolerated, with mostly low-grade urinary toxicities. The most common treatment-related adverse events (TRAEs) were hematuria (32%), and dysuria (21.6%). Hematuria occurred in 38.5% of patients in cohort 1 and 29% of patients in cohort 3. Dysuria was reported in 26.9% and 19.4% of patients in each cohort, respectively. 

Grade 3 or higher TRAEs occurred in 4 patients (4.5%) across both cohorts, and 8 patients (9.1%) discontinued treatment due to adverse events. Two serious treatment-related adverse events were reported. Importantly, no hyperphosphatemia nor retinal toxicities, which are known systemic adverse effects of oral erdafitinib, were observed in the study.

Pharmacokinetic analyses demonstrated sustained drug concentrations in urine over the 3-month treatment period. According to the investigators, plasma concentrations of erdafitinib were 40 times lower than that observed with oral erdafitinib at the standard 9-mg daily dose.

Erda-iDRS also demonstrated preliminary clinical activity.

In the high-risk cohort, the therapy was evaluated in the adjuvant setting following TURBT. At 12 months, the RFS rate was 83% (95% CI, 62 to 93). The median RFS was 20 months (95% CI, 15 to 30), with a median RFS follow-up of 24 months (95% CI, 15 to 30). Approximately 31% of patients remained recurrence-free at the time of analysis.

In the intermediate-risk cohort, Erda-iDRS was used as an ablative treatment for visible tumors. The CR rate was 81% at 3 months and 89% (95% CI, 78 to 95) at 6 months. The median duration of CR was 18 months (95% CI, 14 to 25), with a median follow-up of 18 months (range, 15 to 21) among responders. At the time of data cutoff, 49% of responders remained in complete response.

According to the investigators, these phase 1 findings provide early proof-of-concept that precision-targeted therapy can be delivered locally within the bladder using a sustained drug-delivery system. Several studies are already underway within the MoonRISe clinical development program, including:

• MoonRISe-1: Phase 3 trial evaluating Erda-iDRS in intermediate-risk NMIBC in the adjuvant setting after TURBT
• MoonRISe-2: Phase 2 trial evaluating the device as an ablative therapy for intermediate-risk NMIBC
• MoonRISe-3: Phase 3 trial of adjuvant vs intravesical chemotherapy in high-risk papillary only NMIBC

These trials are expected to clarify whether targeted intravesical FGFR inhibition can improve outcomes or reduce recurrence in molecularly selected NMIBC populations.

Editor’s Note: Vilaseca reports disclosures with Johnson & Johnson/Janssen, Astellas Pharma, Recordati, Bayer, Accord Healthcare, and Ipsen.

REFERENCES

1. Vilaseca A, Li R, Meeks J, et al. Final analysis of the phase 1 first-in-human study of erda-iDRS, an erdafitinib intravesical drug-releasing system, in patients with non–muscle-invasive bladder cancer harboring select FGFR alterations. Presented at: 41st Annual Congress of the European Association of Urology. London, UK. March 13-16, 2026. Abstract LBA008

2. Johnson & Johnson highlights promising first-in-human Erda-iDRS (formerly TAR-210) results in intermediate-risk non-muscle-invasive bladder cancer. News release. Johnson & Johnson. March 13, 2026. Accessed March 13, 2026. https://www.jnj.com/media-center/press-releases/johnson-johnson-highlights-promising-first-in-human-erda-idrs-formerly-tar-210-results-in-intermediate-risk-non-muscle-invasive-bladder-cancer