Urothelial Cancer Monthly Rewind: Key News and Expert Insights You May Have Missed in September 2025

September brought notable progress across the bladder cancer landscape, with a new therapeutic approval, diagnostic advances, and promising early-phase data that could shape future directions in care.

Highlights included the FDA approval of gemcitabine intravesical system (Inlexzo; TAR-200), the first major therapeutic advance for BCG-unresponsive non–muscle-invasive bladder cancer (NMIBC), in over 40 years; progress toward a noninvasive genomic testing option with the PredicineCARE assay; encouraging trial results with IDE397 plus sacituzumab govitecan-hziy (Trodelvy); and the review of a nadofaragene firadenovec new drug application (NDA) in Japan. Leading experts also weighed in on emerging therapies and ongoing research, offering clinical context for a rapidly evolving field.

Check out this month-in-review recap of OncLive®’s coverage of the top news and expert insights in urothelial cancer:

FDA News

FDA Approves TAR-200 in BCG-Unresponsive NMIBC With CIS

The FDA approval of the gemcitabine intravesical system marks the first major advance in more than four decades for patients with BCG-unresponsive NMIBC, offering a nonsurgical treatment option for those who often face bladder removal.¹ The decision is based on findings from the phase 2b SunRISe-1 trial (NCT04640623), in which the monotherapy induced a complete response (CR) rate of 82% (95% CI, 72%-90%) with durable benefit at 1 year. The most common toxicities included urinary frequency (48%), urinary tract infection (44%), and dysuria (42%).

Expert Insights: Joseph Jacob, MD, MCR, of State University of New York Upstate Medical University, unpacked the significance of the regulatory approval.² “This is a new drug that's available that works well and is going to be [an] effective therapy for patients going forward. This is a drug that's going to save a lot of bladders,” he said.

FDA Approval Is Sought for PredicineCARE Assay as Companion Diagnostic in Bladder Cancer

Predicine has completed the first portion of its premarket approval application to the FDA for the PredicineCARE urine cell-free DNA next-generation sequencing assay for patients with bladder cancer, marking a key step toward advancing noninvasive precision diagnostics in this disease.³ Designed to detect a broad spectrum of genomic alterations from urine samples, the assay underscores growing momentum for liquid biopsy technologies that can guide targeted therapy selection and expand access to personalized care.

Trial Updates

IDE397 Plus Sacituzumab Govitecan Elicits Responses in MTAP-Deletion Urothelial Cancer

Early phase 1/2 trial (NCT04794699) data showed that when the MAT2A inhibitor IDE397 was paired with sacituzumab govitecan it elicited responses with manageable safety in patients with heavily pretreated, MTAP-deleted urothelial cancer.⁴ These findings suggest potential synergy between IDE397 and sacituzumab govitecan and support continued investigation of this regimen in urothelial and other MTAP-deleted solid tumors

Global Pipeline Updates

Nadofaragene Firadenovec NDA Accepted for Review in Japan for BCG-Unresponsive NMIBC

The Pharmaceuticals and Medical Devices Agency of Japan accepted for review the NDA seeking approval of nadofaragene firadenovec in patients with BCG-unresponsive NMIBC, marking a potential expansion of this gene therapy’s global reach.⁵ Supported by findings from a phase 3 trial done in Japan, which showed a 3-month CR rate of 75% and a favorable safety profile, the filing underscores continued progress toward bringing bladder-sparing, non-chemotherapy options to those with limited alternatives.

Top Expert Insights of the Month: OncLive TV Highlights

Andrea Necchi, MD, of Vita-Salute Raffaele University and IRCCS San Raffaele Hospital and Scientific Institute, reviewed the design of the phase 2 SURE-02 trial (NCT05535218) which examined the safety and efficacy of sacituzumab govitecan plus pembrolizumab (Keytruda) in patients with muscle-invasive bladder cancer (MIBC).⁶ “SURE-02 was built on the background of the studies we have performed before [in patients with] MIBC, looking at the activity of pembrolizumab monotherapy in the PURE-01 study [NCT02736266], and sacituzumab govitecan monotherapy in the SURE-01 study [NCT05226117].”

In another exclusive interview, John P. Sfakianos, MD, of Icahn School of Medicine at Mount Sinai, discussed the mechanism of action of mitomycin intravesical solution (Zusduri; formerly UGN-102) and the rationale for examining its use in patients with recurrent, low-grade, intermediate-risk NMIBC.⁷ “The biggest unmet need [has been] the [ability] to treat patients with low-grade and intermediate-risk bladder cancer. This is a disease with a high recurrence rate, and it is very cumbersome for patients because of that recurrence rate.”

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References

1. US FDA approval of INLEXZO (gemcitabine intravesical system) set to transform how certain bladder cancers are treated. News Release. Johnson & Johnson. September 9, 2025. Accessed October 8, 2025. https://www.multivu.com/johnson-and-johnson/9342851-en-johnson-and-johnson-fda-approval-inlexzo-gemcitabine-intravesical-system
2. Jacob J. Dr Jacob on the FDA approval of the gemcitabine intravesical system for NMIBC. OncLive.com. September 11, 2025. Accessed October 8, 2025. https://www.onclive.com/view/dr-jacob-on-the-fda-approval-of-the-gemcitabine-intravesical-system-for-nmibc
3. Predicine submits first module of PMA application to FDA for PredicineCARE™ as a companion diagnostic assay in bladder cancer. News release. Predicine. September 2, 2025. Accessed October 8, 2025. https://www.globenewswire.com/news-release/2025/09/02/3142717/0/en/Predicine-Submits-First-Module-of-PMA-Application-to-FDA-for-PredicineCARE-as-a-Companion-Diagnostic-Assay-in-Bladder-Cancer.html
4. IDEAYA Biosciences announces positive data from phase 1/2 combination trial of IDE397, a potential first-in-class MAT2A inhibitor, and Trodelvy in MTAP-deletion urothelial cancer. News release. IDEAYA Biosciences, Inc. September 8, 2025. Accessed October 8, 2025. https://ir.ideayabio.com/2025-09-08-IDEAYA-Biosciences-Announces-Positive-Data-From-Phase-1-2-Combination-Trial-of-IDE397,-a-potential-first-in-class-MAT2A-inhibitor,-and-Trodelvy-R-in-MTAP-Deletion-Urothelial-Cancer
5. Ferring Japan announces PMDA acceptance of NDA filing for nadofaragene firadenovec. News release. Ferring Pharmaceuticals Co., Ltd. September 11, 2025. Accessed October 8, 2025. https://www.businesswire.com/news/home/20250911332715/en/Ferring-Japan-announces-PMDA-Acceptance-of-NDA-Filing-for-nadofaragene-firadenovec
6. Necchi A. Dr Necchi on the design of the phase 2 SURE-02 study assessing pembrolizumab/sacituzumab govitecan in muscle-invasive bladder cancer. OncLive.com. September 10, 2025. Accessed October 8, 2025. https://www.onclive.com/view/dr-necchi-on-the-design-of-the-phase-2-sure-02-study-assessing-pembrolizumab-sacituzumab-govitecan-in-muscle-invasive-bladder-cancer
7. Sfakianos JP. Dr Sfakianos on addressing unmet needs with intravesical mitomycin in recurrent, low-grade, intermediate-risk NMIBC. OncLive.com. September 5, 2025. Accessed October 8, 2025. https://www.onclive.com/view/dr-sfakianos-on-addressing-unmet-needs-with-intravesical-mitomycin-in-recurrent-low-grade-intermediate-risk-nmibc