Complete and Durable Responses Observed in OPTIMA II Phase 2b Final Results for UGN-102 in Patients with Low-Grade Intermediate Risk Non-Muscle Invasive Bladder Cancer
- 65% Complete Response Rate at Three Months
Duration of Response Estimated to be 72.5% at Nine Months (12-Months from Initiation of Therapy) by
Kaplan-Meier Method; Median Duration of Response was Not Reached
- Treatment with UGN-102 was Generally Well Tolerated, with Mostly Mild to Moderate Adverse Events Reported
- Phase 3 Pivotal Trial Evaluating UGN-102 Versus Current Standard of Care Expected to Start by Year-End
Treatment with UGN-102 was generally well tolerated and the safety profile was consistent with previously reported results. In the OPTIMA II trial, the majority of the most common adverse events (≥ 10%) were reported as mild to moderate in severity and include dysuria, urinary frequency, hematuria, urinary urgency, urinary tract infection and fatigue. No treatment related serious adverse events were reported.
“The current approach to treating patients diagnosed with low-grade intermediate risk non-muscle invasive bladder cancer is surgery. In most cases, the cancer comes back and repetitive surgical intervention is required. This puts a tremendous burden on patients and their families and can even be life-threatening,” said
Patients with LG IR-NMIBC are chronically relapsing and currently, their only treatment option is repeated transurethral resection of bladder tumors (TURBT). Some patients require multiple TURBT surgeries per year, which can lead to increased morbidity and risks associated with repetitive anesthesia. It is estimated that 80,000 people in the
“We are extremely encouraged by the OPTIMA II data and believe UGN-102 has the potential to provide a safe, durable, outpatient treatment alternative for low-grade intermediate risk non-muscle invasive bladder cancer,” said Dr.
The Company anticipates submitting the data for presentation at an upcoming medical meeting as well as potential publication in a peer-reviewed journal.
About the Phase 2b OPTIMA II Trial
OPTIMA II (OPTimized Instillation of Mitomycin for Bladder Cancer Treatment) is an open-label, single-arm, multi-center Phase 2b clinical trial of investigational agent UGN-102 (mitomycin) for intravesical solution to evaluate its safety and efficacy in patients with low-grade non-muscle invasive bladder cancer (LG NMIBC) at intermediate risk of recurrence. Intermediate risk is defined as one or two of the following: multiple tumors, solitary tumor >3 cm, or recurrence (≥ 1 occurrence of LG NMIBC within one year of the current diagnosis). Patients were to receive six weekly intravesical instillations of 75 mg UGN-102 in an office setting. The chemoablative effect of UGN-102 was assessed three months after initiation of study treatment with complete response (CR) defined as a negative endoscopic examination, negative cytology, and when indicated, a negative for-cause biopsy. Patients achieving CR were followed quarterly to 12 months after initiation of study treatment to evaluate safety, efficacy, and durability.
UGN-102 (mitomycin) for intravesical solution is an investigational drug formulation of mitomycin in Phase 2b development for the treatment of low-grade intermediate risk non-muscle invasive bladder cancer. Utilizing the RTGel Technology Platform, UroGen’s proprietary sustained release, hydrogel-based formulation, UGN-102 is designed to enable longer exposure of bladder tissue to mitomycin, thereby enabling the treatment of tumors by non-surgical means. UGN-102 is delivered to patients using a standard urinary catheter. The Company reported topline interim results from the Phase 2b OPTIMA II trial in
UroGen is a biopharmaceutical company dedicated to building novel solutions that treat specialty cancers and urologic diseases because patients deserve better options. UroGen has developed RTGel™ reverse-thermal hydrogel, a proprietary sustained release, hydrogel-based platform technology that has the potential to improve therapeutic profiles of existing drugs. UroGen’s sustained release technology is designed to enable longer exposure of the urinary tract tissue to medications, making local therapy a potentially more effective treatment option. UroGen’s approved product, Jelmyto (mitomycin) for pyelocalyceal solution, and investigational treatment UGN-102 (mitomycin) for intravesical solution, are designed to ablate tumors by non-surgical means and to treat several forms of non-muscle invasive urothelial cancer, including low-grade upper tract urothelial cancer and low-grade non-muscle invasive bladder cancer, respectively. UroGen is headquartered in
This press release contains forward-looking statements as that term is defined in the Private Securities Litigation Reform Act of 1995, including, without limitation: the potential of UroGen’s proprietary RTGel technology platform to improve therapeutic profiles of existing drugs; the potential of UGN-102 for treatment of LG IR-NMIBC; the expected initiation of the Phase 3 trial of UGN-102 in LG IR-NMIBC by the end of 2020; and the submission of UGN-102 data for presentation at an upcoming medical meeting as well as potential publication in a peer-reviewed journal. These statements are subject to a number of risks, uncertainties and assumptions, including, but not limited to: the timing and success of clinical trials and potential safety and other complications thereof; the ability to obtain regulatory approval within the timeframe expected, or at all; the ability to maintain regulatory approval; complications associated with commercialization activities, including complications resulting from the ongoing COVID-19 pandemic; the labeling for any approved product; the scope, progress and expansion of developing and commercializing UroGen’s product candidates; the size and growth of the market(s) therefor and the rate and degree of market acceptance thereof vis-à-vis alternative therapies; UroGen’s ability to attract or retain key management, members of the board of directors and personnel; and any negative effects on UroGen’s business, commercialization and product development plans caused by or associated with COVID-19. In light of these risks and uncertainties, and other risks and uncertainties that are described in the Risk Factors section of UroGen’s Form 10-Q filed with the