UroGen Pharma(US:URGN)2023-03-24 12:17Patient 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].