UroGen Pharma Ahead of Schedule to Complete UGN-102 NDA Submission and Reports 2024 Second Quarter Financial Results and Business Highlights

• Potential for an FDA decision as early as the first quarter of 2025, assuming priority review
• UGN-102 Phase 3 ENVISION trial demonstrated an unprecedented 82.3% Duration of Response at 12 Months by Kaplan-Meier analysis in LG-IR-NMIBC patients who achieved a complete response at three months
• Ended Q2 2024 with $241.3 million in cash, cash equivalents and marketable securities
• Reported JELMYTO^® Q2 2024 net product revenues of $21.8 million
• Conference call and webcast to be held today at 10:00 AM ET

“Our immediate priority is completing the submission of a New Drug Application in the very near term for UGN-102, which we believe has the potential to be a practice-changing therapy for the treatment of low-grade intermediate-risk non-muscle invasive bladder cancer,” said Liz Barrett, President and Chief Executive Officer of UroGen. “The compelling body of clinical data, including the ENVISION trial, which demonstrated an unprecedented 82.3% 12-month duration of response by Kaplan-Meier analysis in patients who had previously achieved a complete response at three months, reinforces the opportunity for UGN-102 to be the first FDA-approved medicine for the treatment of low-grade intermediate-risk non-muscle invasive bladder cancer.”

• In June 2024, UroGen reported positive 12-month duration of response (DOR) data from the Phase 3 ENVISION pivotal trial evaluating UGN-102 (mitomycin) for intravesical solution in patients with low-grade intermediate-risk non-muscle invasive bladder cancer (LG-IR-NMIBC). The 12-month DOR was 82.3% (95% CI, 75.9%, 87.1%) by Kaplan-Meier estimate in patients who had achieved complete response (CR) at three months from the first instillation of investigational drug UGN-102. The ENVISION trial previously met its primary endpoint by demonstrating that patients treated with UGN-102 had a 79.6% (95% CI, 73.9%, 84.5%) CR rate at three months following the first instillation of UGN-102. UGN-102 was well tolerated, with a safety profile that was consistent with previous clinical trials.

• The ENVISION 12-month DOR data were presented in a virtual event “New Horizons in Bladder Cancer” hosted by UroGen on June 13. This event included presentations by company management and several key opinion leaders with expertise in urology. There was also a panel discussion on the treatment of LG-IR-NMIBC and insights from a patient from the ENVISION trial. A replay of the event can be accessed here.

• The latest DOR data is expected to support a New Drug Application (NDA) for UGN-102 as a treatment for LG-IR-NMIBC, which the Company plans to complete in the very near term. There is potential for an FDA decision as early as the first quarter of 2025, assuming the FDA grants priority review. UroGen initiated submission of the rolling NDA for UGN-102 in January 2024.

• Generated net product revenue of $21.8 million in the second quarter of 2024, compared to $21.1 million in the second quarter of 2023.

• JELMYTO was featured in three presentations at the AUA 2024 Annual Meeting. Independent long-term, real-world analyses explored use of the product in broad patient types, and with different methods of administration. The results showed that JELMYTO treatment appears to demonstrate favorable recurrence-free survival rates for patients with LG-UTUC who respond to initial induction. There does not appear to be a recurrence difference according to the intent of JELMYTO induction, original tumor size, multifocality or tumor location.

• UroGen is developing UGN-103 and UGN-104, next-generation novel mitomycin-based formulations for UGN-102 and JELMYTO, respectively. These candidates combine UroGen’s RTGel® technology with a novel mitomycin formulation licensed from medac GmbH in an agreement signed in January 2024. The development programs potentially offer both manufacturing efficiencies and additional intellectual property protection for the Company’s low-grade urothelial cancer franchise.

• In April 2024, the U.S. FDA accepted the Company’s Investigational New Drug (IND) application for UGN-103. If approved, UGN-103 is expected to provide several advantages related to production, cost, supply, and product convenience.

• UroGen has initiated the Phase 3 study and has onboarded three clinical sites to explore the safety and efficacy of UGN-103 in LG-IR-NMIBC. UroGen plans to initiate a Phase 3 study of UGN-104 in LG-UTUC early next year.

• In June 2024, UroGen appointed David Lin as Chief Commercial Officer and member of the Executive Leadership Team. Mr. Lin is spearheading UroGen’s commercial strategy and will be leveraging his extensive experience to prepare for the potential launch of UGN-102, if approved.

• In June 2024, the Company completed an underwritten public offering of 5,000,000 ordinary shares at a price to the public of $17.50 per ordinary share, and, to certain investors in lieu of issuing ordinary shares, pre-funded warrants to purchase 1,142,857 ordinary shares at a purchase price of $17.499 per pre-funded warrant, which equals the public offering price per ordinary share less the $0.001 per share exercise price for each pre-funded warrant. Gross proceeds to UroGen from the offering, before deducting underwriting discounts and commissions and estimated offering expenses, were approximately $107.5 million.

• In July 2024 the underwriters exercised their option to purchase the full 921,428 additional shares. This yielded further gross proceeds to the Company of $16.1 million, before deducting underwriting discounts and commissions and estimated offering expenses.