UGN-102 Trials Show No Impact on Function, Symptoms, or QoL in LG-IR-NMIBC Patients
UroGen Pharma Ltd. (Nasdaq: URGN), a biotechnology company focused on developing and commercializing innovative treatments for urothelial and specialty cancers, announced new data supporting its investigational intravesical therapy UGN-102 (mitomycin) at the American Urological Association (AUA) 2025 Annual Meeting, held in Las Vegas, Nevada. Presented as a Moderated Poster (MP15), the data highlight robust and durable complete response (CR) rates in patients with low-grade intermediate-risk non-muscle invasive bladder cancer (LG-IR-NMIBC) treated with UGN-102 — importantly, without compromising patients’ quality of life.
Patient-reported outcomes (PROs) have become increasingly important in modern drug development, particularly in cancers like NMIBC, where the disease and its treatments can significantly affect urinary function and overall quality of life. Recognizing this, UroGen incorporated extensive PRO evaluations into its late-phase clinical trials to better understand the real-world patient experience with UGN-102.
“Patient-reported quality of life outcomes are critical in evaluating the usefulness of investigational drugs, particularly when delivered directly into the bladder, which frequently causes urinary symptoms,” said Charles Peyton, M.D., Assistant Professor of Urology at the University of Alabama’s Heersink School of Medicine, and an investigator on the study. “The aggregated quality of life data across three rigorous clinical trials suggest that UGN-102 is highly tolerable, without significantly impacting symptom burden, patient function, or overall quality of life compared to baseline.”
Dr. Peyton emphasized that these findings position UGN-102 as a promising alternative to traditional surgical approaches, offering patients a less invasive, potentially more tolerable option for managing LG-IR-NMIBC.
Data Across Three Major Clinical Trials
The patient-reported outcomes analysis pooled results from three late-stage clinical trials: OPTIMA II, ATLAS, and ENVISION. Across these studies, patients with LG-IR-NMIBC were treated with UGN-102 and followed closely to evaluate both cancer-related outcomes and self-reported experiences of symptoms and health status over time.
Remarkably, a high proportion of participants—over 91% in ATLAS and over 94% in ENVISION—completed detailed quality of life questionnaires at multiple timepoints: baseline, three months after starting treatment, and at 12 months or at the end of the study period. Baseline results indicated that most patients had high functional status and low symptom burdens prior to treatment initiation.
Crucially, the studies found that UGN-102 did not cause sustained declines in patient functioning or significant increases in symptom burden. No measured domains — such as urinary symptoms, body image concerns, or emotional distress — showed a worsening that exceeded clinically meaningful thresholds either at three months or at the 12-month follow-up. These findings suggest that UGN-102 preserves quality of life while effectively treating the disease.
Mark Schoenberg, M.D., Chief Medical Officer at UroGen Pharma, underscored the significance of these findings. “The patient-reported outcomes data showed that UGN-102 did not adversely impact quality of life while achieving high response rates in LG-IR-NMIBC patients,” he said. “Hearing directly from patients provides us with invaluable insight and reaffirms the importance of pursuing treatments that not only control cancer but also minimize the burden of living with it.”
Dr. Schoenberg added that the PRO findings are aligned with UroGen’s broader mission to develop therapies that genuinely improve patients’ lives, moving beyond traditional measures of success like tumor recurrence rates to consider holistic patient well-being.
Study Design and Methods
Across the three studies, the design involved a standardized six-week induction course of UGN-102 delivered intravesically (directly into the bladder). Patients were evaluated for complete response — defined as the absence of visible tumor or evidence of disease — at three months post-treatment. Those achieving CR were followed for varying durations, ranging from nine months to as long as 63 months in some cases.
In the OPTIMA II and ENVISION studies, all participants received UGN-102. In contrast, the ATLAS trial randomized patients to receive either UGN-102 or undergo traditional transurethral resection of bladder tumor (TURBT), the current standard of care. However, for the purposes of this PRO analysis, only data from UGN-102-treated patients were included.
Patient-reported symptoms and overall health status were assessed using the European Organisation for Research and Treatment of Cancer’s Non-Muscle Invasive Bladder Cancer Questionnaire (EORTC-QLQ-NMIBC24). This validated instrument measures key domains relevant to NMIBC patients, including urinary symptoms, body image, sexual health, and concerns about treatment side effects.
Scores on the questionnaire were transformed to a 0-100 scale, where higher scores indicate greater symptom burden or more severe issues. Descriptive statistics summarized baseline scores and changes over time, with positive changes interpreted as worsening symptoms.
However, across all domains and individual items, UGN-102 treatment did not lead to clinically meaningful deteriorations in scores, confirming that the treatment’s benefit does not come at the expense of daily functioning or well-being.
Why Patient-Reported Outcomes Matter
The field of oncology has increasingly recognized that survival and recurrence rates, while critical, are not the only important endpoints. How patients feel and function during and after treatment is equally vital, particularly for diseases like NMIBC, where patients may live for many years following diagnosis and treatment.
Traditional interventions, such as TURBT, can be associated with substantial symptom burdens, including urinary frequency, urgency, incontinence, and sexual dysfunction. Moreover, the need for repeated surgical interventions can impact emotional health, body image, and overall quality of life.
By demonstrating that UGN-102 is not only effective in achieving durable complete responses but also maintains high quality of life, UroGen strengthens the case for its use as a less invasive, patient-friendly alternative to surgery.
With regulatory filings anticipated based on the ENVISION and ATLAS study results, UroGen Pharma is preparing to position UGN-102 as a potential new standard of care for LG-IR-NMIBC. If approved, UGN-102 could provide a meaningful non-surgical treatment alternative for thousands of patients diagnosed each year with this form of bladder cancer.
“These findings represent an important milestone in our journey to offer patients innovative, non-invasive options that allow them to live healthier, fuller lives,” said Dr. Schoenberg. “We remain deeply committed to patient-centered innovation and will continue advancing solutions that prioritize both efficacy and the human experience of cancer treatment.”
As UroGen moves forward with regulatory discussions, the strong patient-reported outcomes data from OPTIMA II, ATLAS, and ENVISION will be a key component in demonstrating the overall value of UGN-102 — not just in treating cancer, but in helping patients maintain the lives they cherish.
