On June 13, 2024, UroGen Pharma Ltd. announced positive secondary endpoint duration of response (?DOR?) data from the Phase 3 ENVISION trial investigating UGN-102 for intravesical solution in patients with low-grade intermediate risk non-muscle invasive bladder cancer (?LG-IR-NMIBC?). In the ENVISION trial, the 12-month DOR data by Kaplan-Meier (?KM?) estimate for patients who achieved a complete response (?CR?) at three months after the first instillation of UGN-102 was 82.3% (95% CI, 75.9%, 87.1%). This is in line with the 69.9% (51.8%, 82.3%) nine-month DOR data by KM estimate for patients who achieved a CR at three months after the first instillation of UGN-102 observed in the Company?s Phase 2b OPTIMA II trial and the 79.6% (69.3%, 86.8%) 12-month DOR data by KM estimate for patients who achieved a CR at three months after the first instillation of UGN-102 alone observed in the Company?s Phase 2 ATLAS trial.

The ENVISION trial met its primary endpoint with patients having a 79.6% (73.9%, 84.5%) CR rate at three months after the first instillation of UGN-102. This is in line with the CR rates at three months after the first instillation of UGN-102 of 65.1% (52.0%, 76.7%) and 64.8% (56.3%, 72.6%) observed in the OPTIMA II trial and ATLAS trial, respectively. Among the patients in the ENVISION trial who achieved a CR at three months, 76.4% (69.8%, 82.3%) maintained a CR at 12 months.

Among the 240 patients enrolled in the ENVISION trial, 60.8% (54.3%, 67.0%) were still in response at 12 months. In the ENVISION trial, DOR KM estimates at 15 (n=43) and 18 (n=9) months were both 80.9% (95% CI, 73.9%, 86.2%). Although median DOR was not estimable due to the number of patients remaining in CR, the predicted median DOR is 40 months based on the Weibull Predicted Curve.

The ENVISION trial demonstrated a similar safety profile to that observed in the OPTIMA II and ATLAS trials, with treatment-emergent adverse events typically mild-to-moderate in severity. In January 2024, the Company initiated the submission of a rolling New Drug Application (?NDA?) to the U.S. Food and Drug Administration (?FDA?) for UGN-102 as a treatment for LG-IR-NMIBC. The latest DOR data are expected to support the UGN-102 NDA, which the Company plans to complete in the third quarter of 2024, with a potential FDA acceptance in the fourth quarter of 2024 and potential FDA approval in the first quarter of 2025 (assuming priority review) or the second quarter of 2025 (assuming standard review).

If approved, UGN-102 may become the first FDA-approved medicine for LG-IR-NMIBC. The Company estimates that the annual addressable U.S. patient population for LG-IR-NMIBC is approximately 82,000, of which approximately 23,000 are estimated to be newly diagnosed and 59,000 are estimated to be recurrent patients. The Company estimates that the total addressable market opportunity for UGN-102 in LG-IR-NMIBC is potentially over $5.0 billion, assuming an expected pricing range of $16,000 to $19,000 per dose.

UGN-102, if approved, may be an alternative to the current standard of care for LG-IR-NMIBC, trans-urethral resection of bladder tumor (?TURBT?). The Company estimates that approximately 68% of LG-IR-NMIBC patients have two or more recurrences, with approximately 23% having five or more recurrences. Repeated TURBT procedures to treat these recurrences can impact patients?

physical health and quality of life. The Company estimates that around 35% of patients will experience an adverse event within 90 days of undergoing a TURBT, and patients who have had two to four procedures have an estimated 14% greater risk of death than patients who have only had one procedure.