On November 12, 2019 UroGen Pharma Ltd. (Nasdaq:URGN), a clinical-stage biopharmaceutical company developing treatments to address unmet needs in uro-oncology, reported financial results for the third quarter ended September 30, 2019 and provided an overview of the Company’s recent developments (Press release, UroGen Pharma, NOV 12, 2019, View Source [SID1234551005]).

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"On behalf of the UroGen team, we are very pleased to announce that we have completed the New Drug Application (NDA) submission for UGN-101 to the FDA. This is a very important milestone for UroGen and for patients with low-grade UTUC as UGN-101 has the potential to bring the first primary chemoablation drug to these patients. I am proud of the UroGen team and investigators who have helped to make this key milestone a reality," said Liz Barrett, President and Chief Executive Officer of UroGen. "Beyond UGN-101, we have taken important steps to build a company that can deliver long-term sustainable growth through our focus on high unmet need areas utilizing both local delivery and novel medicines. We’ve demonstrated that relentless commitment with our recently announced positive complete response data for investigational agent UGN-102 in patients with intermediate risk LG NMIBC, and our exclusive collaboration with Agenus to advance therapies for high-grade urinary tract cancers. We look forward to a strong future for UroGen."

Recent Highlights and Upcoming Milestones

UGN-101 (mitomycin gel) for instillation for patients with low-grade upper tract urothelial cancer (LG UTUC):
The Company completed its NDA submission to the FDA, and the FDA has a 60-day filing review period to determine whether the NDA submission is complete. The FDA previously granted Orphan Drug, Fast Track, and Breakthrough Therapy Designations to UGN-101 for the treatment of LG UTUC. UGN-101 is eligible for Priority Review and the Company is planning for anticipated approval and launch in 1H 2020. UGN-101 plans for launch readiness are on track for January 2020.
The NDA submission is supported by positive data from the pivotal Phase 3 OLYMPUS clinical trial.
UroGen also initiated a retreatment protocol as an extension to OLYMPUS to evaluate the efficacy and safety of retreatment with UGN-101 in patients from the trial who initially achieved a CR and were subsequently found to have a documented recurrence of LG UTUC during follow-up.
UGN-102 (mitomycin gel) in development for intravesical instillation for patients with intermediate risk low-grade non-muscle invasive bladder cancer (LG NMIBC):
UroGen recently presented interim results from the single-arm, open-label Phase 2b OPTIMA II trial of investigational UGN-102 for patients with intermediate risk LG NMIBC. LG NMIBC is defined as those with one or two of the following criteria: multifocal disease, large tumors and rapid rates of recurrence.
In an interim analysis, 63% (20/32) of patients from the Phase 2b OPTIMA II trial achieved a CR. Patients will continue to be followed with 12-month durability to be reported at a later date.
The most common treatment emergent adverse events (TEAEs) included dysuria, pollakiuria, fatigue, hematuria and urinary tract infection. The majority were characterized as mild or moderate in severity and transient.
The trial completed enrollment of 63 patients ahead of schedule at clinical sites across the U.S. and Israel. The Company intends to initiate a pivotal Phase 3 trial in 2020 following a planned discussion with the FDA in 1Q 2020.
This is a patient population whereby the current standard of care, transurethral resection of bladder tumor, or TURBT, is used repeatedly to address chronic recurrence of disease. These patients experience what can be viewed as a form of surgical failure and many undergo multiple surgical procedures during life to "manage" bladder cancer recurrences.
There are no drugs currently approved by the FDA as first-line treatment for intermediate risk LG NMIBC. UGN-102 has the potential to provide a non-surgical alternative for the treatment of chronic relapse for approximately 80,000 patients characterized as intermediate risk.
Pipeline Advancement:
UGN-201 (TLR 7/8 agonist) for patients with high-grade NMIBC:
UroGen is exploring the utility of UGN-201 in the context of novel combinatorial immunotherapy for NMIBC. Nonclinical data suggests 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.
Exclusive Worldwide License Agreement with Agenus Inc. to Advance Treatment of High-Grade Urinary Cancers
UroGen entered into an exclusive worldwide license agreement with Agenus to develop and commercialize zalifrelimab (AGEN1884, anti-CTLA-4 antibody) in combination with UGN-201 for the treatment of urinary tract cancers via intravesical delivery. Zalifrelimab (AGEN1884, anti-CTLA-4 antibody) is currently being evaluated by Agenus as a monotherapy in PD-1 refractory patients.
The initial indication for development will be high-grade NMIBC.
UroGen will be responsible for all development and commercialization activities.
Third Quarter 2019 Financial Results; 2019 Guidance

As of September 30, 2019, cash, cash equivalents and marketable securities totaled $221.7 million.
Research and development expenses for the three months ended September 30, 2019 were $9.5 million, including non-cash share-based compensation expense of $2.1 million. Research and development expenses for the nine months ended September 30, 2019 were $29.2 million, including non-cash share-based compensation expense of $6.4 million.
General and administrative expenses for the three months ended September 30, 2019 were $14.0 million, including non-cash share-based compensation expense of $5.2 million. General and administrative expenses for the nine months ended September 30, 2019 were $40.5 million, including non-cash share-based compensation expense of $15.5 million.
The Company reported a net loss of $22.3 million, or basic and diluted net loss per ordinary share of $1.06, for the three months ended September 30, 2019. The Company reported a net loss of $66.2 million, or basic and diluted net loss per ordinary share of $3.25, for the nine months ended September 30, 2019.
Including the recently announced Agenus deal, the Company is still on track to end the year with a net loss for the year in the range of $100 to $110 million, which is expected to include non-cash stock-based compensation expense in the range of $28 to $30 million, subject to market conditions.
Conference Call & Webcast Information

Members of UroGen’s management team will host a live conference call and webcast today at 8:30 am Eastern Time to review the Company’s financial results and provide a general business update.

The live webcast can be accessed by visiting the Investors section of the Company’s website at View Source Please connect at least 15 minutes prior to the live webcast to ensure adequate time for any software download that may be needed to access the webcast. Alternatively, please call (888) 771-4371 (U.S.) or (847) 585-4405 (International) to listen to the live conference call. The conference ID number for the live call will be 49159339. An archive of the webcast will be available for two weeks on the Company’s website.