UroGen Pharma(URGN) - 2024 Q4 - Annual Report

Market Potential and Product Development - UGN-102, if approved, could address a treatable population of approximately 82,000 low-grade intermediate risk NMIBC patients in the U.S., with a total addressable market exceeding $5.0 billion [506]. - The company has initiated the Phase 3 ENVISION trial to evaluate UGN-102 as primary chemoablative therapy for low-grade intermediate risk NMIBC [524]. - The FDA accepted the NDA for UGN-102, with a PDUFA goal date of June 13, 2025, potentially making it the first FDA-approved treatment for low-grade intermediate-risk NMIBC [530]. - The company entered into a licensing agreement with medac for UGN-103 and UGN-104, aiming for an NDA submission for UGN-103 in 2026 and a commercial launch in 2027 [531]. Clinical Trial Results - The complete response rate for Jelmyto in the OLYMPUS trial was 58%, with a durability of response estimated at 81.8% at 12 months [513]. - In the OPTIMA II trial, 65% of patients achieved a complete response three months after treatment with UGN-102, with a 72.5% probability of durable response at nine months [522]. - In the Phase 3 ATLAS trial, UGN-102 reduced the risk of recurrence, progression, or death by 55% compared to TURBT alone, with a 64.8% complete response (CR) rate at three months for UGN-102 patients [525]. - The ENVISION trial demonstrated a 79.6% CR rate at three months for patients treated with UGN-102, with 60.8% of all enrolled patients in CR at 12 months [526][527]. - Updated 18-month duration of response (DOR) data from the ENVISION trial showed an 80.6% DOR for patients who achieved CR at three months [528]. - The company completed a Phase 3b study indicating that UGN-102 can be administered at home, with 75% of patients achieving CR three months after treatment [529]. Financial Performance - Revenue from Jelmyto sales increased to$ 90.4 million in 2024 from $82.7 million in 2023 [539]. - The consolidated net revenue for the year ended December 31, 2024, was$ 90.4 million, an increase from $82.7 million in 2023, representing a growth of approximately 9.3% [628]. - Gross profit for 2024 was$ 81.5 million, up from $73.4 million in 2023, reflecting an increase of$ 8.2 million [562]. - The net loss for the year ended December 31, 2024, was $126.9 million, compared to a net loss of$ 102.2 million in 2023, an increase of $24.6 million [562]. - Selling, general and administrative expenses rose significantly to$ 121.2 million in 2024, up from $93.3 million in 2023, marking an increase of about 30% [636]. Research and Development Expenses - Research and development expenses increased from$ 45.6 million in 2023 to $57.1 million in 2024, primarily due to clinical development costs [543]. - Research and development expenses rose to$ 57.1 million in 2024 from $45.6 million in 2023, an increase of$ 11.5 million primarily due to higher manufacturing costs and regulatory expenses [565]. - Research and development expenses are expensed as incurred, primarily consisting of salaries, share-based compensation, and costs related to clinical trials and professional services [685]. Cash Flow and Financing - The company expects to finance cash needs through equity or debt financings and collaboration arrangements until substantial product revenue is generated [585]. - As of December 31, 2024, the company had $241.7 million in cash and cash equivalents and marketable securities [572]. - Net cash used in operating activities increased to$ 96.8 million in 2024 from $76.4 million in 2023, primarily due to higher operating expenses related to UGN-102 [588]. - Net cash provided by financing activities increased to$ 194.6 million in 2024 from $116.9 million in 2023, driven by proceeds from the issuance of ordinary shares and debt related to the Pharmakon loan [590]. Inventory and Asset Management - As of December 31, 2024, total inventories increased to$ 9,446,000 from $7,341,000 as of December 31, 2023, representing a year-over-year growth of approximately 28.6% [699]. - The Company commenced capitalization of inventory costs for Jelmyto upon receipt of FDA approval, with inventory valued at the lower of cost or net realizable value [671]. - The company’s inventory assessment includes a review for recoverability each reporting period to determine any necessary write-downs due to excess or obsolete inventories [672]. Regulatory and Compliance - The company has adopted new accounting standards updates, including ASU 2023-07 for segment reporting disclosures, effective for fiscal years beginning after December 15, 2023 [693]. - The Company is party to a loan agreement with Pharmakon Advisors, recognizing interest expense in current earnings and capitalizing financing expenses as a direct offset to long-term debt [676]. Shareholder Information - The company reported a weighted average number of shares outstanding of 42,876,737 for the year ended December 31, 2024, compared to 28,834,303 in 2023, an increase of approximately 48.7% [636]. - The net loss attributable to equity holders of the Company for the year ended December 31, 2024, was$ 126.874 million, compared to a net loss of $102.244 million for 2023, resulting in a loss per ordinary share of$ (2.96) for 2024 and $(3.55) for 2023 [692].