UroGen Pharma Ltd. (NASDAQ:URGN) Q4 2023 Earnings Conference Call March 14, 2024 10:00 AM ET Company Participants Vincent Perrone - Head of IR Elizabeth Barrett - President and CEO Mark Schoenberg - Chief Medical Officer Dong Kim - CFO Jeffrey Bova - Chief Commercial Officer Silvio Pacheco - VP of Market Access Conference Call Participants Tara Bancroft - TD Cowen Leland Gershell - Oppenheimer Paul Choi - Goldman Sachs Matt Kaplan - Ladenburg Thalmann Operator Good morning, ladies and gentlemen. Thank you f ...

PART I [Business](index=7&type=section&id=Item%201.%20Business) UroGen Pharma develops and commercializes urothelial and specialty cancer treatments using its proprietary RTGel® reverse-thermal hydrogel technology, with Jelmyto® as its primary approved product [Overview and Technology](index=7&type=section&id=Overview%20and%20Technology) The company's core is the RTGel® reverse-thermal hydrogel, a proprietary technology that is liquid at cool temperatures and becomes a gel at body temperature, allowing sustained drug release - UroGen's proprietary RTGel® is a reverse thermal hydrogel that is liquid when cool and gels at body temperature, designed to enable longer exposure of urinary tract tissue to medications[24](index=24&type=chunk)[25](index=25&type=chunk) - RTGel technology increases the dwell time of mitomycin in the upper urinary tract from approximately five minutes (standard aqueous formulation) to about **six hours**, enhancing drug-tissue coverage[29](index=29&type=chunk) [Product Pipeline](index=8&type=section&id=Product%20Pipeline) UroGen's pipeline is led by Jelmyto®, approved for low-grade UTUC, with lead candidate UGN-102 targeting low-grade intermediate-risk NMIBC, and other next-generation formulations and an immuno-oncology program in development UroGen Pharma Product Pipeline Status | Product Candidate | Indication | Development Stage | | :--- | :--- | :--- | | **Jelmyto® (UGN-101)** | Low-Grade UTUC | Approved & Commercialized | | **UGN-102** | Low-Grade Intermediate Risk NMIBC | Phase 3 (NDA Submission Planned) | | **UGN-103** | Low-Grade Intermediate Risk NMIBC | Phase 3 (Planned 2024) | | **UGN-104** | Low-Grade UTUC | Phase 3 (Planned 2024) | | **UGN-301 (Zalifrelimab)** | High-Grade NMIBC | Phase 1 | - Jelmyto® (mitomycin) for pyelocalyceal solution was approved by the FDA on April 15, 2020, for the treatment of adult patients with low-grade UTUC, holding Orphan Drug exclusivity until April 15, 2027[33](index=33&type=chunk) - The Phase 3 ENVISION trial for UGN-102 met its primary endpoint, showing a **79.2% rate of complete response (CR)** at three months, with a rolling NDA submission planned for September 2024[56](index=56&type=chunk)[58](index=58&type=chunk) - The company is developing UGN-103 and UGN-104, next-generation formulations of UGN-102 and Jelmyto, respectively, in partnership with medac, with Phase 3 studies planned for 2024[59](index=59&type=chunk)[60](index=60&type=chunk) [Strategy, Competition, and Intellectual Property](index=15&type=section&id=Strategy%2C%20Competition%2C%20and%20Intellectual%20Property) UroGen's strategy focuses on establishing Jelmyto as the standard of care, advancing UGN-102, and expanding its uro-oncology pipeline, while facing competition from surgical procedures and other pharmaceutical companies, supported by a robust intellectual property portfolio - Key growth strategies include establishing Jelmyto as the standard of care, advancing UGN-102 towards regulatory approval, expanding the uro-oncology pipeline with candidates like UGN-301, UGN-103, and UGN-104, and exploring collaborations[80](index=80&type=chunk)[81](index=81&type=chunk)[82](index=82&type=chunk) - The company holds a strong intellectual property position with **43 granted patents worldwide** and over **45 pending applications**, with U.S. patents protecting Jelmyto, UGN-102, and RTGel technology expiring between 2024 and 2037[78](index=78&type=chunk)[86](index=86&type=chunk) - Competition arises from the current standard of care (surgical tumor resection) and other pharmaceutical companies such as Janssen, Lipac, Bristol Myers Squibb, Merck, and Ferring Pharmaceuticals, developing drugs for NMIBC and other uro-oncology indications[93](index=93&type=chunk)[94](index=94&type=chunk)[96](index=96&type=chunk) [Government Regulation](index=19&type=section&id=Government%20Regulation) The company is subject to extensive FDA and global regulations covering product development, manufacturing, and marketing, utilizing the 505(b)(2) pathway for its mitomycin-based products, with ongoing post-approval oversight and compliance with healthcare laws - The FDA approval process is extensive, requiring nonclinical studies, an effective IND, and adequate and well-controlled human clinical trials (Phase 1-3) to establish safety and efficacy before an NDA submission[100](index=100&type=chunk)[101](index=101&type=chunk)[103](index=103&type=chunk) - The company utilizes the **505(b)(2) regulatory pathway**, which allows an NDA to rely on FDA's prior findings of safety and efficacy for a previously approved drug, potentially streamlining development[108](index=108&type=chunk) - Jelmyto received **Orphan Drug Designation**, providing **seven years of marketing exclusivity** in the U.S. for its approved indication, ending in April 2027[33](index=33&type=chunk)[115](index=115&type=chunk) - The business is subject to numerous healthcare laws, including the federal Anti-Kickback Statute, False Claims Act, HIPAA, and the Sunshine Act, which regulate relationships with healthcare professionals and government reimbursement programs[135](index=135&type=chunk)[136](index=136&type=chunk)[140](index=140&type=chunk)[141](index=141&type=chunk) [Manufacturing, Commercial Operations, and Corporate Information](index=28&type=section&id=Manufacturing%2C%20Commercial%20Operations%2C%20and%20Corporate%20Information) UroGen relies on third-party contract manufacturers for all raw materials and finished products, has established a U.S. commercial team of approximately 80 representatives for Jelmyto, and as of January 31, 2024, had 201 employees worldwide, with principal executive offices in Princeton, NJ - The company does not own manufacturing facilities and relies on third-party contract manufacturers for all raw materials, active ingredients, and finished products for both clinical trials and commercial supply[159](index=159&type=chunk) - A customer-facing team of approximately **80 representatives**, including territory business managers, clinical nurse educators, and medical science liaisons, has been established to support the commercialization of Jelmyto in the U.S[162](index=162&type=chunk) - As of January 31, 2024, the company had **201 employees**, with **161 located in the United States** and **40 in Israel**[165](index=165&type=chunk) [Risk Factors](index=30&type=section&id=Item%201A.%20Risk%20Factors) The company faces significant risks including historical losses, financing needs, high dependency on Jelmyto and UGN-102, competition, supply chain issues, IP challenges, and regulatory and geopolitical instability - The company has a history of significant losses (**$102.2 million in 2023**) and an accumulated deficit of **$679.3 million**, raising substantial doubt about its ability to continue as a going concern without additional financing[170](index=170&type=chunk)[180](index=180&type=chunk) - High dependency on the successful commercialization of Jelmyto and the regulatory approval and success of UGN-102 are primary business risks[194](index=194&type=chunk)[215](index=215&type=chunk) - Reliance on third-party, single-source suppliers for key components like mitomycin API and RTGel hydrogel presents significant supply chain risks[257](index=257&type=chunk)[258](index=258&type=chunk) - The company faces intellectual property risks, highlighted by a Paragraph IV Certification Notice from Teva Pharmaceuticals seeking to market a generic version of Jelmyto by challenging its patents[209](index=209&type=chunk)[321](index=321&type=chunk) - Operations in Israel expose the company to political, economic, and military instability, which could disrupt research and development activities[425](index=425&type=chunk) [Unresolved Staff Comments](index=75&type=section&id=Item%201B.%20Unresolved%20Staff%20Comments) The company reports that it has no unresolved staff comments from the SEC - None[450](index=450&type=chunk) [Cybersecurity](index=75&type=section&id=Item%201C.%20Cybersecurity) The company has implemented an information security program supervised by the Chief Financial Officer and IT Department to manage cybersecurity risks, with Audit Committee oversight, utilizing third-party service providers, and maintaining an incident response policy - The company's cybersecurity risk management is supervised by the Chief Financial Officer and the IT Department, with oversight from the Audit Committee of the Board of Directors[452](index=452&type=chunk)[458](index=458&type=chunk) - The company's strategy includes using third-party service providers for security management roles to identify, assess, and manage cybersecurity threats through various methods like monitoring, threat assessments, and vulnerability scans[452](index=452&type=chunk)[455](index=455&type=chunk) - A cybersecurity incident response policy is in place, designed to escalate significant incidents to management and the Audit Committee to ensure proper mitigation and remediation[461](index=461&type=chunk) [Properties](index=76&type=section&id=Item%202.%20Properties) The company leases approximately 20,913 square feet in Princeton, NJ for its principal executive offices and an 11,495 square foot facility in Israel for research and development, which management deems adequate for current needs - The company's principal executive offices are leased in Princeton, NJ (**~20,913 sq. ft.**), and its primary R&D laboratories are in a leased facility in Israel (**~11,495 sq. ft.**)[463](index=463&type=chunk) [Legal Proceedings](index=76&type=section&id=Item%203.%20Legal%20Proceedings) The company is not currently a party to any legal proceedings that are expected to have a material adverse effect on its business - The company is not currently party to any material legal proceedings[464](index=464&type=chunk) [Mine Safety Disclosures](index=76&type=section&id=Item%204.%20Mine%20Safety%20Disclosures) This item is not applicable to the company - Not applicable[465](index=465&type=chunk) PART II [Market for Registrant's Common Equity, Related Stockholder Matters and Issuer Purchases of Equity Securities](index=77&type=section&id=Item%205.%20Market%20for%20Registrant%27s%20Common%20Equity%2C%20Related%20Stockholder%20Matters%20and%20Issuer%20Purchases%20of%20Equity%20Securities) UroGen's ordinary shares trade on the Nasdaq Global Market under the symbol 'URGN', with 16 registered holders as of March 7, 2024, and the company has never paid dividends, intending to retain earnings for business development, also restricted by its loan agreement - The company's ordinary shares trade on the Nasdaq Global Market under the symbol **URGN**[468](index=468&type=chunk) - The company has never paid dividends and does not expect to in the foreseeable future, due to its policy of retaining earnings for growth and restrictions under its loan agreement[470](index=470&type=chunk) [Management's Discussion and Analysis of Financial Condition and Results of Operations](index=78&type=section&id=Item%207.%20Management%27s%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) UroGen's 2023 revenue increased, net loss improved, and liquidity was bolstered by financing, though current resources are projected to fund operations only until Q1 2025, raising going concern doubts [Results of Operations](index=85&type=section&id=Results%20of%20Operations) For 2023, revenue increased by 28.5% to $82.7 million, gross profit grew, operating loss narrowed to $65.5 million due to lower R&D expenses, partially offset by increased SG&A and interest expenses, resulting in a net loss of $102.2 million Results of Operations (2023 vs. 2022) | Metric | 2023 (in thousands) | 2022 (in thousands) | Change (in thousands) | | :--- | :--- | :--- | :--- | | **Revenue** | $82,713 | $64,357 | $18,356 | | Gross Profit | $73,352 | $56,703 | $16,649 | | Research and development | $45,614 | $52,906 | ($7,292) | | Selling and marketing | $54,703 | $51,920 | $2,783 | | General and administrative | $38,571 | $30,918 | $7,653 | | **Operating loss** | ($65,536) | ($79,041) | $13,505 | | **Net loss** | ($102,244) | ($109,783) | $7,539 | - Revenue increased by **$18.3 million (28.5%)** in 2023 compared to 2022, primarily due to increased sales volume of Jelmyto[529](index=529&type=chunk) - Research and development expenses decreased by **$7.3 million**, mainly due to the conclusion of the ATLAS trial and lower costs for the Phase 3 ENVISION trial for UGN-102[531](index=531&type=chunk) [Liquidity and Capital Resources](index=86&type=section&id=Liquidity%20and%20Capital%20Resources) As of December 31, 2023, the company had $141.5 million in cash and equivalents, funded by equity offerings, debt, and product sales, including a $120.0 million private placement in 2023 and an amended loan agreement with Pharmakon, though current resources are projected to fund operations only until Q1 2025, raising substantial doubt about its going concern ability - The company had **$141.5 million** in cash, cash equivalents, and marketable securities as of December 31, 2023[538](index=538&type=chunk) - In July 2023, the company raised aggregate gross proceeds of **$120.0 million** through a private placement of ordinary shares and pre-funded warrants[544](index=544&type=chunk) - The company's existing resources are estimated to be sufficient to fund operations only until the **first quarter of 2025**, which raises substantial doubt about its ability to continue as a going concern[546](index=546&type=chunk) - In March 2024, the company amended its loan agreement with Pharmakon, securing access to an additional **$25.0 million tranche** (mandatory draw by Sept 2024) and an optional **$75.0 million tranche** contingent on UGN-102 FDA approval[543](index=543&type=chunk) [Quantitative and Qualitative Disclosures about Market Risk](index=91&type=section&id=Item%207A.%20Quantitative%20and%20Qualitative%20Disclosures%20about%20Market%20Risk) The company is exposed to interest rate risk on its investment portfolio and significant foreign currency exchange risk due to New Israeli Shekel operating expenses, but does not currently use hedging instruments - The company's primary market risks are interest rate fluctuations on its marketable securities and foreign currency exchange risk[574](index=574&type=chunk)[576](index=576&type=chunk) - A significant portion of operating expenses are incurred in New Israeli Shekels (NIS), exposing the company to fluctuations between the NIS and the U.S. dollar, its reporting currency[576](index=576&type=chunk) - The company does not currently engage in currency hedging activities to reduce its foreign currency exposure[577](index=577&type=chunk) [Financial Statements and Supplementary Data](index=92&type=section&id=Item%208.%20Financial%20Statements%20and%20Supplementary%20Data) This section contains the company's audited consolidated financial statements for 2023 and 2022, including the Independent Registered Public Accounting Firm's report highlighting going concern doubt, and various financial statements with accompanying notes - The Report of Independent Registered Public Accounting Firm expresses substantial doubt about the Company's ability to continue as a going concern due to its history of losses and negative operating cash flows[583](index=583&type=chunk) Key Financial Statement Data (as of Dec 31, 2023) | Metric | Amount (in thousands) | | :--- | :--- | | **Assets** | | | Cash and cash equivalents | $95,002 | | Total Current Assets | $169,186 | | Total Assets | $178,311 | | **Liabilities & Equity** | | | Total Current Liabilities | $31,212 | | Long-term debt | $98,551 | | Total Liabilities | $243,523 | | Total Shareholders' Deficit | ($65,212) | Key Financial Statement Data (Year ended Dec 31, 2023) | Metric | Amount (in thousands) | | :--- | :--- | | Revenue | $82,713 | | Gross Profit | $73,352 | | Operating Loss | ($65,536) | | Net Loss | ($102,244) | | Net cash used in operating activities | ($76,376) | [Changes in and Disagreements with Accountants on Accounting and Financial Disclosure](index=122&type=section&id=Item%209.%20Changes%20in%20and%20Disagreements%20with%20Accountants%20on%20Accounting%20and%20Financial%20Disclosure) The company reports no changes in or disagreements with its accountants on accounting and financial disclosure - None[746](index=746&type=chunk) [Controls and Procedures](index=122&type=section&id=Item%209A.%20Controls%20and%20Procedures) Management, including the CEO and CFO, concluded that the company's disclosure controls and procedures and internal control over financial reporting were effective as of December 31, 2023, with no material changes identified - Management concluded that the company's disclosure controls and procedures were effective as of December 31, 2023[748](index=748&type=chunk) - Based on the COSO framework, management concluded that the company's internal control over financial reporting was effective as of December 31, 2023[750](index=750&type=chunk) [Other Information](index=122&type=section&id=Item%209B.%20Other%20Information) This section discloses the Chief Medical Officer's Rule 10b5-1 trading plan, the March 2024 amended loan agreement with Pharmakon providing up to $100 million in additional financing, and notice for the 2024 Annual Meeting of Shareholders - On March 13, 2024, the company entered into an amended and restated loan agreement with Pharmakon for up to **$100 million** in additional financing, structured in two tranches[753](index=753&type=chunk) - The Chief Medical Officer, Mark Schoenberg, entered into a Rule 10b5-1 trading plan on December 26, 2023, to sell up to **30,000 ordinary shares**[752](index=752&type=chunk) [Disclosure Regarding Foreign Jurisdictions that Prevent Inspections](index=123&type=section&id=Item%209C.%20Disclosure%20Regarding%20Foreign%20Jurisdictions%20that%20Prevent%20Inspections) This item is not applicable to the company - Not applicable[761](index=761&type=chunk) PART III [Directors, Executive Officers and Corporate Governance](index=124&type=section&id=Item%2010.%20Directors%2C%20Executive%20Officers%20and%20Corporate%20Governance) The information required for this item, including details on directors, executive officers, and corporate governance, will be provided in an amendment to the Form 10-K, to be filed by April 29, 2024 - Information will be provided in a future amendment to the Form 10-K[763](index=763&type=chunk) [Executive Compensation](index=124&type=section&id=Item%2011.%20Executive%20Compensation) The information required for this item regarding executive compensation will be provided in an amendment to the Form 10-K, to be filed by April 29, 2024 - Information will be provided in a future amendment to the Form 10-K[765](index=765&type=chunk) [Security Ownership of Certain Beneficial Owners and Management and Related Stockholder Matters](index=124&type=section&id=Item%2012.%20Security%20Ownership%20of%20Certain%20Beneficial%20Owners%20and%20Management%20and%20Related%20Stockholder%20Matters) The information required for this item concerning security ownership will be provided in an amendment to the Form 10-K, to be filed by April 29, 2024 - Information will be provided in a future amendment to the Form 10-K[766](index=766&type=chunk) [Certain Relationships and Related Transactions, and Director Independence](index=124&type=section&id=Item%2013.%20Certain%20Relationships%20and%20Related%20Transactions%2C%20and%20Director%20Independence) The information required for this item regarding related party transactions and director independence will be provided in an amendment to the Form 10-K, to be filed by April 29, 2024 - Information will be provided in a future amendment to the Form 10-K[767](index=767&type=chunk) [Principal Accountant Fees and Services](index=124&type=section&id=Item%2014.%20Principal%20Accountant%20Fees%20and%20Services) The information required for this item regarding principal accountant fees and services will be provided in an amendment to the Form 10-K, to be filed by April 29, 2024 - Information will be provided in a future amendment to the Form 10-K[768](index=768&type=chunk) PART IV [Exhibits, Financial Statement Schedules](index=125&type=section&id=Item%2015.%20Exhibits%2C%20Financial%20Statement%20Schedules) This section lists the financial statements, included in Item 8, and provides a comprehensive list of all exhibits filed with the Form 10-K, including corporate governance documents, material contracts, and officer certifications - This item contains a list of all exhibits filed with the annual report, including corporate governance documents, material contracts, and required certifications[772](index=772&type=chunk)[773](index=773&type=chunk) [Form 10-K Summary](index=127&type=section&id=Item%2016.%20Form%2010-K%20Summary) The company indicates that there is no Form 10-K summary - None[775](index=775&type=chunk)

Financial Performance - UroGen reported net product revenues of $82.7 million for 2023, representing an increase of approximately 28% compared to $64.4 million in 2022[5][11][10]. - UroGen's cash, cash equivalents, and marketable securities totaled $141.5 million as of December 31, 2023, up from $99.9 million in 2022[21]. - UroGen reported a net loss of $102.2 million for the full year 2023, compared to a net loss of $109.8 million in 2022[16]. - Selling, general and administrative expenses for 2023 were $93.3 million, compared to $82.8 million in 2022[13]. - Research and development expenses for 2023 were $45.6 million, a decrease from $52.9 million in 2022[12]. Product Development and Pipeline - The company initiated a rolling New Drug Application (NDA) for UGN-102 in January 2024, with plans to complete the submission by September 2024 and a potential FDA decision as early as Q1 2025[4][3]. - UroGen anticipates completing its NDA submission for UGN-102 in September 2024, with a potential FDA decision as early as Q1 2025[32]. - UroGen plans to initiate Phase 3 studies for UGN-103 and UGN-104 in 2024, targeting low-grade intermediate-risk non-muscle invasive bladder cancer and low-grade upper tract urothelial cancer, respectively[10]. - UroGen plans to initiate Phase 3 studies for UGN-103 and UGN-104 in 2024, expanding its product pipeline[34]. - UroGen's NDA submission for UGN-102 is contingent on positive findings from the ENVISION Phase 3 study, with data expected in Q2 2024[34]. Market Opportunity and Growth - UroGen's UGN-102 has a market opportunity exceeding $3 billion, aiming to transform treatment for low-grade, intermediate-risk, non-muscle invasive bladder cancer[34]. - The company anticipates full year 2024 net product revenues from JELMYTO to be in the range of $95 to $102 million[17]. - JELMYTO patient and physician adoption is expected to continue increasing, contributing to UroGen's growth[34]. - Patient enrollment, new patient starts, and total doses for JELMYTO grew approximately 25% year-over-year[10]. Financial Flexibility - The company signed a restructured agreement with Pharmakon Advisors, providing up to an additional $100 million credit facility[5][9]. - The company has access to an additional $75 million under an amended credit facility with Pharmakon Advisors, enhancing its financial flexibility[34]. Technology and Patient Considerations - UroGen's proprietary RTGel technology aims to improve therapeutic profiles of existing drugs, potentially making local delivery more effective[34]. - The most common side effects of JELMYTO include urinary tract infection and nausea, highlighting the need for patient monitoring[30]. - Approximately 6,000 - 7,000 new or recurrent low-grade upper tract urothelial cancer patients are diagnosed annually in the U.S.[31]. Intellectual Property - UroGen's pending U.S. patent applications could provide protection for UGN-103 and UGN-104 until December 2041, securing its competitive position[34].

Urogen Pharma Ltd (NASDAQ:URGN) Q3 2023 Earnings Conference Call November 14, 2023 10:00 AM ET Company Participants Vincent Perrone - Senior Director, IR Elizabeth Barrett - President, CEO & Director Mark Schoenberg - Chief Medical Officer Dong Kim - CFO Jeffrey Bova - Chief Commercial Officer Conference Call Participants Leland Gershell - Oppenheimer Raghuram Selvaraju - H.C. Wainwright & Co. Boris Peaker - TD Cowen Paul Choi - Goldman Sachs Group Operator Good morning, ladies and gentlemen, and thank you ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION For the quarterly period ended September 30, 2023 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from to Commission file number: 001-38079 WASHINGTON, DC 20549 FORM 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 UROGEN PHARMA LTD. N/A (Former name, former address and former fiscal year, if changed since last report) Securities re ...

UroGen Pharma Ltd. (NASDAQ:URGN) Q2 2023 Results Conference Call August 10, 2023 10:00 AM ET Company Participants Vincent Perrone - Head Investor Relations Liz Barrett - President and Chief Executive Officer Mark Schoenberg - Chief Medical Officer Jeff Bova - Chief Commercial Officer Don Kim - Chief Financial Officer Conference Call Participants Raghuram Selvaraju - H.C. Wainwright Boris Peaker - TD Cowen Leland Gershell - Oppenheimer Matt Kaplan - Ladenburg Thalmann Operator Good morning, ladies and gentle ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, DC 20549 FORM 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended June 30, 2023 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from to Commission file number: 001-38079 UROGEN PHARMA LTD. (Exact Name of Registrant as Specified in its Charter) (State or other jurisdiction of incorporation or o ...

Urogen Pharma Ltd (NASDAQ:URGN) Q1 2023 Earnings Conference Call May 11, 2023 10:00 AM ET Company Participants Vincent Perrone - Senior Director, IR Elizabeth Barrett - President, CEO & Director Mark Schoenberg - Chief Medical Officer Jeffrey Bova - Chief Commercial Officer Dong Kim - CFO Conference Call Participants Raghuram Selvaraju - H.C. Wainwright & Co. Roderick Ma - Goldman Sachs Group Matthew Kaplan - Ladenburg Thalmann & Co. Rohan Mathur - Oppenheimer Boris Peaker - TD Cowen Operator Good morning, ...

[PART I. FINANCIAL INFORMATION](index=4&type=section&id=PART%20I.%20FINANCIAL%20INFORMATION) For the quarter ended March 31, 2023, UroGen Pharma Ltd. reported total revenues of $17.2 million, a net loss of $30.2 million, and held cash, cash equivalents, and marketable securities of $75.2 million [Item 1. Financial Statements (Unaudited)](index=4&type=section&id=Item%201.%20Financial%20Statements%20(Unaudited)) For the quarter ended March 31, 2023, UroGen Pharma Ltd. reported total revenues of $17.2 million, a net loss of $30.2 million, and held cash, cash equivalents, and marketable securities of $75.2 million - The company has experienced net losses since inception, with an accumulated deficit of **$607.3 million** as of March 31, 2023, raising substantial doubt about its ability to continue as a going concern without raising additional capital[24](index=24&type=chunk)[25](index=25&type=chunk)[26](index=26&type=chunk) [Condensed Consolidated Balance Sheets](index=4&type=section&id=Condensed%20Consolidated%20Balance%20Sheets) As of March 31, 2023, total assets were $113.0 million, down from $135.6 million at year-end 2022, primarily due to a decrease in cash and marketable securities, leading to a total shareholders' deficit of $116.6 million Condensed Consolidated Balance Sheet Data (in thousands) | Account | March 31, 2023 | December 31, 2022 | | :--- | :--- | :--- | | Cash and cash equivalents | $36,540 | $55,408 | | Total current assets | $95,973 | $128,907 | | **Total Assets** | **$112,954** | **$135,619** | | Total current liabilities | $25,234 | $23,916 | | Prepaid forward obligation | $101,912 | $98,923 | | Long-term debt | $97,941 | $97,537 | | **Total Liabilities** | **$229,508** | **$224,980** | | Accumulated deficit | ($607,317) | ($577,104) | | **Total Shareholders' Deficit** | **($116,554)** | **($89,361)** | [Condensed Consolidated Statements of Operations and Comprehensive Loss](index=5&type=section&id=Condensed%20Consolidated%20Statements%20of%20Operations%20and%20Comprehensive%20Loss) For the three months ended March 31, 2023, revenue increased to $17.2 million from $13.6 million in the prior-year period, resulting in a net loss of $30.2 million, or ($1.30) per share, compared to a net loss of $28.4 million, or ($1.25) per share, for the same period in 2022 Statement of Operations Highlights (in thousands, except per share data) | Metric | Q1 2023 | Q1 2022 | | :--- | :--- | :--- | | Revenue | $17,192 | $13,564 | | Gross Profit | $14,927 | $12,039 | | Research and development expenses | $12,498 | $12,696 | | Selling, general and administrative expenses | $24,474 | $21,300 | | Operating loss | ($22,045) | ($21,957) | | **Net Loss** | **($30,213)** | **($28,392)** | | **Net loss per ordinary share** | **($1.30)** | **($1.25)** | [Condensed Consolidated Statements of Cash Flows](index=7&type=section&id=Condensed%20Consolidated%20Statements%20of%20Cash%20Flows) In the first quarter of 2023, net cash used in operating activities was $25.8 million, net cash provided by investing activities was $6.3 million, and net cash provided by financing activities was $0.7 million, resulting in a net decrease in cash and cash equivalents of $18.9 million Cash Flow Summary (in thousands) | Activity | Q1 2023 | Q1 2022 | | :--- | :--- | :--- | | Net cash used in operating activities | ($25,807) | ($23,996) | | Net cash provided by investing activities | $6,270 | $13,596 | | Net cash provided by financing activities | $671 | $70,742 | | **Increase (Decrease) in Cash** | **($18,866)** | **$60,342** | [Management's Discussion and Analysis of Financial Condition and Results of Operations](index=25&type=section&id=Management's%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) Management discusses the company's focus on developing and commercializing treatments for urothelial cancers using its proprietary RTGel technology, with revenue from Jelmyto increasing to $17.2 million in Q1 2023, but continues to incur significant losses, raising substantial doubt about its ability to continue as a going concern without securing additional financing - The company is developing innovative solutions for urothelial cancers using its proprietary RTGel reverse-thermal hydrogel technology, including the FDA-approved Jelmyto, UGN-102, and UGN-301[111](index=111&type=chunk) - The Phase 3 ENVISION trial for UGN-102 completed its target enrollment of **220 patients** in December 2022, with an anticipated NDA submission in 2024 assuming positive findings[132](index=132&type=chunk) - The company believes its existing cash and projected revenues will only fund operations until **mid-way through the first quarter of 2024**, necessitating significant additional financing and raising substantial doubt about its ability to continue as a going concern[176](index=176&type=chunk)[177](index=177&type=chunk) [Results of Operations](index=35&type=section&id=Results%20of%20Operations) Comparing Q1 2023 to Q1 2022, revenue increased by $3.6 million due to higher Jelmyto sales volume, while operating expenses and interest expense also rose, contributing to an increased net loss Comparison of Operations for the three months ended March 31 (in thousands) | Account | 2023 | 2022 | Change | | :--- | :--- | :--- | :--- | | Revenue | $17,192 | $13,564 | $3,628 | | Cost of revenue | $2,265 | $1,525 | $740 | | Research and development | $12,498 | $12,696 | ($198) | | Selling and marketing | $14,143 | $13,351 | $792 | | General and administrative | $10,331 | $7,949 | $2,382 | | **Net loss** | **($30,213)** | **($28,392)** | **($1,821)** | [Liquidity and Capital Resources](index=36&type=section&id=Liquidity%20and%20Capital%20Resources) As of March 31, 2023, the company had $75.2 million in cash, cash equivalents, and marketable securities, but with an accumulated deficit of $607.3 million and anticipated continued losses, management concluded there is substantial doubt about the company's ability to continue as a going concern without securing additional financing - The company has an at-the-market (ATM) sales agreement with Cowen with approximately **$83.4 million** remaining capacity as of March 31, 2023[172](index=172&type=chunk) - The company entered into a loan agreement with Pharmakon for a senior secured term loan of up to **$100 million**, fully funded as of December 2022, requiring interest-only payments for the first 48 months[174](index=174&type=chunk)[81](index=81&type=chunk) - Net cash used in operating activities increased to **$25.8 million** in Q1 2023 from **$24.0 million** in Q1 2022, primarily due to financing costs and timing of accruals, partially offset by increased Jelmyto sales[190](index=190&type=chunk) [Quantitative and Qualitative Disclosures About Market Risk](index=40&type=section&id=Quantitative%20and%20Qualitative%20Disclosures%20About%20Market%20Risk) The company is exposed to market risks, including interest rate fluctuations on its investment portfolio, inflation affecting labor and clinical trial costs, and foreign currency exchange risk, particularly with New Israeli Shekels (NIS) operating expenses - The company's primary market risks are interest rate fluctuations on its **$75.2 million** in cash and marketable securities, and foreign currency exchange risk[193](index=193&type=chunk) - A significant portion of operating expenses are in New Israeli Shekels (NIS), creating exposure to adverse movements in the NIS/USD exchange rate, though a **10% change** in Q1 2023 would not have had a material effect on operating expenses[196](index=196&type=chunk) [Controls and Procedures](index=42&type=section&id=Controls%20and%20Procedures) Management, including the CEO and CFO, evaluated the company's disclosure controls and procedures as of March 31, 2023, concluding they were effective at a reasonable assurance level, with no material changes in internal control over financial reporting during the quarter - The principal executive officer and principal financial officer concluded that as of March 31, 2023, the company's disclosure controls and procedures were effective at a reasonable assurance level[199](index=199&type=chunk) - No changes in internal control over financial reporting occurred during the quarter ended March 31, 2023, that have materially affected, or are reasonably likely to materially affect, internal controls[200](index=200&type=chunk) [PART II. OTHER INFORMATION](index=43&type=section&id=PART%20II.%20OTHER%20INFORMATION) [Item 1. Legal Proceedings](index=43&type=section&id=Item%201.%20Legal%20Proceedings) The company is not currently a party to any legal proceedings that management believes are likely to have a material adverse effect on its business - UroGen is not currently a party to any legal proceedings that, in the opinion of management, are likely to have a material adverse effect on the business[202](index=202&type=chunk) [Item 1A. Risk Factors](index=43&type=page&id=Item%201A.%20Risk%20Factors) The company outlines numerous risks to its business, including substantial doubt about its ability to continue as a going concern due to the need for significant additional financing, high dependence on Jelmyto's commercial success, and risks in clinical development, manufacturing, competition, intellectual property, regulatory landscapes, and operations in Israel - **Financial Risk:** The company will require additional financing to fund operations and achieve its goals, as existing resources are only expected to be sufficient until **mid-way through Q1 2024**, raising substantial doubt about its ability to continue as a going concern[204](index=204&type=chunk)[213](index=213&type=chunk)[214](index=214&type=chunk) - **Commercial Risk:** The company is highly dependent on the successful commercialization of Jelmyto and faces significant competition, with market opportunities potentially smaller than anticipated and achieving broad physician adoption as a key challenge[204](index=204&type=chunk)[229](index=229&type=chunk)[244](index=244&type=chunk) - **Development and Regulatory Risk:** The company is dependent on the success of its pipeline, particularly UGN-102, as clinical drug development is a lengthy, expensive, and uncertain process, and earlier trial results may not predict future success[204](index=204&type=chunk)[252](index=252&type=chunk)[273](index=273&type=chunk) - **Operational and Supply Chain Risk:** The company relies on third-party, single-source suppliers for key raw materials and components, increasing the risk of supply disruptions that could impair development and commercialization[206](index=206&type=chunk)[297](index=297&type=chunk) [Item 2. Unregistered Sales of Equity Securities and Use of Proceeds](index=118&type=section&id=Item%202.%20Unregistered%20Sales%20of%20Equity%20Securities%20and%20Use%20of%20Proceeds) There were no unregistered sales of equity securities during the period - None[503](index=503&type=chunk) [Item 3. Defaults Upon Senior Securities](index=118&type=section&id=Item%203.%20Defaults%20Upon%20Senior%20Securities) There were no defaults upon senior securities during the period - None[503](index=503&type=chunk) [Item 4. Mine Safety Disclosures](index=118&type=section&id=Item%204.%20Mine%20Safety%20Disclosures) This item is not applicable to the company - None[504](index=504&type=chunk) [Item 5. Other Information](index=118&type=section&id=Item%205.%20Other%20Information) There was no other information to report for the period - None[505](index=505&type=chunk) [Item 6. Exhibits](index=119&type=section&id=Item%206.%20Exhibits) This section lists the exhibits filed with the quarterly report, including the Articles of Association, certifications by the CEO and CFO pursuant to the Sarbanes-Oxley Act, and Inline XBRL data files

Patient Population and Treatment Efficacy - The annual treatable patient population for low-grade UTUC in the United States is estimated to be approximately 6,000 to 7,000, while the annual treatable population for low-grade intermediate risk NMIBC is around 80,000[490]. - Jelmyto achieved a complete response (CR) rate of 58% (41 out of 71 patients) in the intent-to-treat population during the Phase 3 OLYMPUS trial, with a durability of response estimated at 81.8% at 12 months[496]. - The median duration of response for Jelmyto was reported to be 28.9 months based on long-term follow-up data from the OLYMPUS trial[497]. - UGN-102 demonstrated a complete response rate of 65% (41 out of 63 patients) in the Phase 2b OPTIMA II trial, with a durable response probability of 72.5% at nine months after CR[503]. - The Phase 3 ENVISION trial for UGN-102 has completed enrollment of 220 patients across 90 sites, with an NDA submission anticipated in 2024 assuming positive findings[508]. Financial Performance - Revenue for the year ended December 31, 2022, was $64.4 million, an increase of $16.4 million from $48.0 million in 2021, reflecting higher sales volume of the product Jelmyto[520][541]. - The company's consolidated net revenue for the year ended December 31, 2022, was $64.4 million, with gross revenue from product sales representing a majority[598]. - Total revenue for 2022 was $64.36 million, a 34% increase from $48.04 million in 2021[606]. - Gross profit for 2022 reached $56.70 million, compared to $42.89 million in 2021, reflecting a gross margin improvement[606]. - The net loss for the year ended December 31, 2022, was $109.8 million, a slight improvement from a net loss of $110.8 million in 2021[540]. - The operating loss narrowed to $79.0 million in 2022 from $92.3 million in 2021, indicating improved operational efficiency[540]. - The accumulated deficit as of December 31, 2022, was $577.1 million, with expectations of continued losses for the foreseeable future[554]. Expenses and Cost Management - Cost of revenue increased to $7.7 million in 2022 from $5.2 million in 2021, resulting in a gross margin of approximately 87.5% for 2022[522][542]. - Research and development expenses rose to $52.9 million in 2022, up from $47.6 million in 2021, indicating continued investment in product development[540]. - Selling and marketing expenses increased to $51.9 million in 2022 compared to $47.6 million in 2021, reflecting efforts to promote Jelmyto[540]. - General and administrative expenses decreased to $30.9 million in 2022 from $39.9 million in 2021, showing cost management in administrative functions[540]. - Interest expense on long-term debt was $8.4 million in 2022, compared to zero in 2021, related to the Pharmakon loan initiated in March 2022[547]. Cash Flow and Financing - As of December 31, 2022, the company had $100.0 million in cash and cash equivalents and marketable securities, with cash in excess of immediate requirements invested primarily in U.S. dollars[549]. - Net cash used in operating activities was $87.6 million in 2022, compared to $84.9 million in 2021, reflecting an increase of $2.7 million due to financing costs and timing of accruals[559]. - Net cash provided by financing activities was $97.1 million in 2022, up from $72.3 million in 2021, primarily due to net proceeds from the Pharmakon loan[561]. - The company anticipates needing significant additional financing within the next twelve months to continue operations, as existing resources are projected to be insufficient beyond mid-way through the first quarter of 2024[555]. Inventory and Assets - As of December 31, 2022, total inventories increased to $6.695 million from $5.852 million in 2021, representing a growth of approximately 14.4%[662]. - Total assets increased to $135.62 million in 2022, up from $119.75 million in 2021, indicating growth in the company's asset base[604]. - Cash and cash equivalents at the end of 2022 were $55.41 million, an increase from $44.36 million at the end of 2021[604]. Market and Regulatory Environment - Jelmyto's new product exclusivity lasts until April 15, 2023, and orphan drug exclusivity extends to April 15, 2027, with key patents expiring in January 2031[493]. - All Medicare patients are covered for Jelmyto, with over 150 million lives covered by commercial plans[499]. - The company has initiated a registry to capture data and evaluate real-world outcomes for patients treated with Jelmyto[500]. Debt and Obligations - The Company entered into a loan agreement with Pharmakon for a senior secured term loan of up to $100 million, funded in two tranches: $75 million in March 2022 and $25 million in December 2022[685]. - The loan requires interest-only payments for the first 48 months, followed by principal and interest payments, with an interest rate of 3-month LIBOR plus 8.25%[685]. - The carrying value of the Pharmakon loan as of December 31, 2022, was $97.537 million after accounting for capitalized costs and discounts[687]. Tax and Regulatory Compliance - The company provides for income taxes based on pretax income and applicable tax rates in various jurisdictions, including Israel and the U.S.[581]. - The Company has incurred tax losses totaling approximately $419.1 million as of December 31, 2022, which can be carried forward indefinitely[538].