JnJ’s INLEXZO™ Achieves 74% One-Year Disease-Free Survival in BCG-Unresponsive High-Risk Papillary NMIBC

Johnson & Johnson announced on 5 December 2025, compelling new data from Cohort 4 of the Phase 2b SunRISe-1 study evaluating INLEXZO™ (gemcitabine intravesical system) in patients with BCG-unresponsive, high-risk, papillary-only non-muscle invasive bladder cancer (NMIBC) achieved a 74.3% one-year disease-free survival (DFS) rate. These data were featured as a late-breaking oral presentation at the Society of Urologic Oncology (SUO) 2025 Annual Meeting.

These new papillary-only findings from Cohort 4 build on prior SunRISe-1 data from Cohort 2, which demonstrated an 83.5% complete response rate in BCG-unresponsive NMIBC with carcinoma in situ (CIS) ± papillary tumors and supported INLEXZO’s FDA approval on September 9, 2025 as detailed in our previous coverage of the approval. While Cohort 2 focused on CIS-driven disease, Cohort 4 specifically addresses papillary-only high-risk NMIBC, where recurrence and progression risks drive standard cystectomy recommendations. This complementary data further positions INLEXZO as a versatile bladder-preserving option across NMIBC subtypes.

Cohort 4 of the Phase 2b SunRISe-1 study (NCT04640623) enrolled 52 patients with papillary-only, high-risk NMIBC who did not respond to BCG therapy and were ineligible for or declined radical cystectomy. At a median follow-up of 15.9 months, the one-year DFS rate reached 74.3%, with consistent results across high-grade Ta (74.8%) and T1 (74.1%) tumors. Progression-free survival (PFS) at one year was 95.6% and overall survival (OS) was 98%.

These outcomes highlight INLEXZO’s potential to prevent recurrence and progression of high-grade papillary tumors, supporting its evaluation in the ongoing Phase 3 SunRISe-5 study (NCT06211764) against chemotherapy in BCG-treated papillary NMIBC patients. Notably, 92.3% of patients avoided radical cystectomy, with median time to cystectomy not reached.

Treatment Administration and Safety

INLEXZO delivers 225 mg of gemcitabine into the bladder via an intravesical drug-releasing system inserted outpatient without general anesthesia, remaining indwelling for three weeks per cycle. Dosing involves insertion every three weeks for six months (eight doses), then every 12 weeks for up to 18 months (six doses), or until disease recurrence, progression, or toxicity.​

The therapy proved well-tolerated, with 80.8% of patients experiencing low-grade treatment-related adverse events like mild urinary symptoms (burning, frequency, urgency). Grade 3/4 events occurred in 13.5%, mainly bladder pain; 7.7% discontinued due to side effects, and no treatment-related deaths occurred. Health status and physical functioning scores remained stable.

Dr. Siamak Daneshmand noted that bladder removal has traditionally been the primary treatment for these patients, a life-altering procedure significantly impacting quality of life, underscoring the importance of developing bladder-preserving therapies. He also emphasized that the new data showing patients remaining free of cancer recurrence at one year support hope for better, less invasive treatment options ahead.

Christopher Cutie, M.D., highlighted Johnson & Johnson’s commitment to developing innovative treatments for high-risk NMIBC patients who have limited options beyond life-altering surgery. He emphasized that patients with papillary-only disease face difficult decisions, as bladder removal has long been the standard of care, reinforcing the need for new bladder-preserving therapies.

FDA Approvals and Designations

The U.S. FDA approved INLEXZO on September 9, 2025, for BCG-unresponsive NMIBC with carcinoma in situ (CIS), with or without papillary tumors, based on SunRISe-1 data showing 82% complete response and 51% durability at one year. This first-of-its-kind system addresses unmet needs in bladder preservation post-BCG failure, earning Breakthrough Therapy, Priority Review, and Real-Time Oncology Review designations. Ongoing trials like SunRISe-3, SunRISe-4, and SunRISe-5 continue assessing its role in high-risk NMIBC and muscle-invasive bladder cancer (MIBC). Johnson & Johnson offers J&J withMe support for access, including cost aid and navigation.​