On September 24, 2019 UroGen Pharma Ltd. (Nasdaq: URGN), a clinical-stage biopharmaceutical company developing treatments to address unmet needs in the field of urology, reported that updates on its advancing pipeline for urologic cancers and UGN-101 launch readiness at its Investor Day in New York on September 24, 2019 (Press release, UroGen Pharma, SEP 24, 2019, View Source [SID1234539752]).

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"At UroGen, we are 100 percent committed to overcoming obstacles that have stunted much needed innovation in uro-oncology," said Liz Barrett, President and Chief Executive Officer of UroGen. "While we prepare for a potential launch of UGN-101, we believe this is just the beginning of what is possible with our pipeline. The data from an interim analysis for UGN-102 unveiled at our Investor Day further highlight the potential of our RTGel platform to transform treatments in this space for an even larger patient population with bladder cancer. We look forward to pushing innovation beyond our foundational platform and moving UGN-201, our TLR 7/8 agonist for high-grade bladder cancer, into clinical studies in 2020."

UroGen detailed data updates for its lead investigational product candidates UGN-101 and UGN-102:

UGN-101 (mitomycin gel) for instillation for patients with low-grade upper tract urothelial cancer (LG UTUC):
Complete response (CR) rate of 59 percent observed in 71 patients with LG UTUC from Phase 3 OLYMPUS trial. Data remain consistent with previously presented data.
Durability of response determined to be 89 percent at six months and 84 percent at twelve months.
In the OLYMPUS trial, the most common treatment emergent adverse events (TEAE) included ureteral stenosis, urinary tract infection, hematuria, flank pain, dysuria, renal impairment, hydronephrosis and frequency. Most TEAEs were characterized as mild to moderate and transient.
At the time of database lock, the most common Grade 3 TEAE’s included ureteral stenosis (8.5%), hematuria, flank pain, and urinary tract infection (3% each). There was one Grade 4 TEAE of subdural hematoma (1.4%).
Rolling NDA submission is on track for Q4 2019 with potential approval and launch in 1H 2020.
If approved, UGN-101 will be the first drug for the primary chemoablative treatment of LG UTUC.
UGN-102 (mitomycin gel) for intravesical instillation for patients with intermediate risk low-grade non-muscle invasive bladder cancer (LG NMIBC):
In an interim analysis, 63% (20/32) of patients from the Phase 2b OPTIMA II trial achieved a CR.
In an interim analysis, the most common treatment emergent adverse events (TEAEs) observed were dysuria, pollakiuria, fatigue, hematuria and urinary tract infection. The majority were characterized as mild or moderate and transient.
Patients will continue to be followed with 12-month durability to be reported at a later date.
Trial enrolled patients with intermediate risk LG NMIBC, defined as those with one or two of the following criteria: multifocal disease, large tumors and rapid rates of recurrence.
Trial completed enrollment of 62 patients ahead of schedule.
The Company intends to initiate a pivotal Phase 3 trial in 2020 following discussion with the FDA.
UroGen discussed its ongoing activities to build awareness of unmet needs in UTUC, educate the market and commercialize UGN-101 following anticipated regulatory approval:

Increased scientific awareness of UGN-101 clinical data developments in urologic community.
Engagement with payers and proactive market access strategy to ensure patient access and reimbursement.
UGN-101 treatment expected to fit well into existing physician reimbursement models.
Planned convenience kit for UGN-101 will facilitate preparation and administration for practitioners.
Experienced commercial team with track record of success in uro-oncology.
Nimble salesforce with seven Regional Business Managers (RBMs) hired, and 50 sales reps to be hired by end of 2019.
The Company also provided an update on UGN-201, it’s investigational TLR 7/8 agonist for the treatment of high-grade NMIBC. UGN-201 is believed to stimulate innate and adaptive antitumor immunity. It likely works in conjunction with other potent immunoregulatory molecules. Nonclinical data shows an efficacy signal when UGN-201 is administered locally with anti-CTLA4 antibody in a murine model of high-grade bladder cancer. The Company plans to optimize combinations and advance into human studies.

About UGN-101

UGN-101 (mitomycin gel) for instillation is an investigational drug formulation of mitomycin in Phase 3 development for the treatment of low-grade upper tract urothelial cancer (LG UTUC). Utilizing the RTGel technology platform, UroGen’s proprietary sustained release, hydrogel-based formulation, UGN-101 is designed to enable longer exposure of urinary tract tissue to mitomycin, thereby enabling the treatment of tumors by non-surgical means. UGN-101 is delivered to patients using standard ureteral catheters. The Company initiated its rolling submission of the UGN-101 New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) in December 2018. The FDA previously granted Orphan Drug, Fast Track, and Breakthrough Therapy Designations to UGN-101 for the treatment of UTUC. If approved, UGN-101 would be the first drug approved for the non-surgical treatment of LG UTUC.

About UGN-102

UGN-102 (mitomycin gel) for instillation is an investigational drug formulation of mitomycin in Phase 2b development for the treatment of low-grade non-muscle invasive bladder cancer (LG NMIBC). 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 standard intravesical catheters. The Company completed enrollment in the Phase 2b OPTIMA II trial of UGN-102 for the treatment of LG NMIBC in September 2019 and intends to advance the program to a pivotal study to further investigate UGN-102 in the treatment of this condition.