Ultragenyx Pharmaceutical(RARE) - 2025 Q2 - Quarterly Results

Financial Highlights and Corporate Outlook Ultragenyx reported $166 million in Q2 2025 revenue, reaffirmed full-year guidance, and progressed key clinical programs including upcoming Phase 3 data for UX143 and full enrollment for GTX-102 Q2 2025 Financial Metrics | Metric | Q2 2025 | | :--- | :--- | | Total Revenue | $166 million | | Crysvita® Revenue | $120 million | | Dojolvi® Revenue | $23 million | - The company reaffirmed its full-year 2025 revenue guidance, projecting total revenue between $640 million and $670 million[1] - Management emphasized continued progress towards achieving GAAP profitability in 2027 through top-line growth and fiscal discipline[2] - Key clinical updates include expected Phase 3 data for UX143 by year-end and full enrollment of the Phase 3 Aspire study for GTX-102, which received FDA Breakthrough Therapy Designation[1] Financial Performance Ultragenyx reported $166.5 million in Q2 2025 total revenues, a 13% year-over-year increase, with a reduced net loss of $115.0 million and a quarter-end cash balance of $539 million Revenues Total revenue for Q2 2025 increased 13% year-over-year to $166.5 million, primarily driven by strong performance from Crysvita, Dojolvi, and Evkeeza Q2 2025 Revenue Breakdown | Revenue Source | Q2 2025 ($M) | Q2 2024 ($M) | YoY Growth | | :--- | :--- | :--- | :--- | | Total Crysvita Revenue | 120.4 | 113.7 | 6% | | Dojolvi | 23.2 | 19.4 | 20% | | Evkeeza | 14.6 | 7.9 | 85% | | Mepsevii | 8.3 | 6.1 | 35% | | Total Revenues | 166.5 | 147.0 | 13% | Operating Expenses and Net Loss Q2 2025 operating expenses reached $274.4 million, resulting in a net loss of $115.0 million or $1.17 per share, an improvement from the prior year's net loss Q2 2025 Operating Expenses and Net Loss | Financial Metric | Q2 2025 ($M) | Q2 2024 ($M) | | :--- | :--- | :--- | | Total Operating Expenses | 274.4 | 263.4 | | Net Loss | (115.0) | (131.6) | | Net Loss Per Share | ($1.17) | ($1.52) | - Total operating expenses included $39 million in non-cash stock-based compensation[6] Cash Position As of June 30, 2025, Ultragenyx maintained a cash position of $539 million, with $108 million net cash used in operations for the quarter - Cash, cash equivalents, and marketable debt securities stood at $539 million as of June 30, 2025[8] - Net cash used in operations was $108 million for the second quarter and $275 million for the first six months of 2025[8] 2025 Financial Guidance Ultragenyx reaffirmed its full-year 2025 financial guidance, projecting 14-20% total revenue growth and maintaining its target for GAAP profitability in 2027, despite anticipated increases in net cash used in operations 2025 Full-Year Financial Guidance | Guidance Metric | 2025 Full Year Range | | :--- | :--- | | Total Revenue | $640M - $670M | | Crysvita Revenue | $460M - $480M | | Dojolvi Revenue | $90M - $100M | - Net cash used in operations is now expected to modestly increase compared to 2024 due to timing delays and changes for UX111, DTX401, and UX143 programs[9] - The company reaffirms its path to GAAP profitability in 2027 by focusing on revenue growth and prioritized spending[9] Clinical Pipeline and Regulatory Updates Ultragenyx provided significant updates on its late-stage clinical pipeline, including progress for UX143 and GTX-102, addressing the UX111 CRL, and advancing DTX401 and UX701 programs UX143 (setrusumab) for Osteogenesis Imperfecta (OI) The Phase 3 Orbit and Cosmic studies for UX143 are progressing towards final data analysis by year-end 2025, with an acceptable safety profile confirmed for the Orbit study - Final analysis for the Phase 3 Orbit and Cosmic studies is expected around the end of 2025[11] - A Data Monitoring Committee reviewed the Orbit study in July 2025, confirmed an acceptable safety profile, and recommended the study continue to its final analysis[11] GTX-102 for Angelman Syndrome GTX-102 received FDA Breakthrough Therapy Designation in June 2025, with its global Phase 3 Aspire study fully enrolled and data anticipated in the second half of 2026 - The FDA granted Breakthrough Therapy Designation for GTX-102 in June 2025 based on positive Phase 1/2 data showing consistent developmental gains[14] - The global Phase 3 Aspire study is fully enrolled with 129 patients, with primary endpoint data expected in the second half of 2026[15] UX111 for Sanfilippo Syndrome Type A (MPS IIIA) The FDA issued a Complete Response Letter (CRL) for the UX111 BLA in July 2025, requesting additional CMC information, which the company believes is addressable for resubmission - The FDA issued a Complete Response Letter (CRL) for the UX111 BLA, requesting more information on CMC procedures and validation[17] - The CRL did not note any issues with the clinical data package, and the company believes the observations are readily addressable[17][18] DTX401 for Glycogen Storage Disease Type Ia (GSDIa) Ultragenyx plans to submit a Biologics License Application (BLA) for DTX401 in Q4 2025, proactively addressing CMC observations from the UX111 review - A BLA submission for DTX401 is planned for the fourth quarter of 2025[19] - The BLA will include updates to proactively address CMC observations from the UX111 CRL[19] UX701 for Wilson Disease Enrollment for the fourth cohort of the pivotal Cyprus2+ study of UX701 is ongoing and expected to complete in the second half of 2025 - The Phase 1/2/3 Cyprus2+ study is ongoing, with enrollment in the fourth cohort expected to complete in the second half of 2025[20] Appendix: Detailed Financial Statements This appendix presents detailed unaudited financial statements for Q2 2025, including the Statement of Operations showing a $115.0 million net loss and the Balance Sheet with $539.0 million in cash and $1.31 billion in total assets Selected Statement of Operations Financial Data For Q2 2025, total revenues were $166.5 million and operating expenses $274.4 million, leading to a $115.0 million net loss Q2 2025 Selected Statement of Operations | Statement of Operations (Q2 2025, $ thousands) | Amount | | :--- | :--- | | Total revenues | 166,496 | | Total operating expenses | 274,384 | | Loss from operations | (107,888) | | Net loss | (114,951) | | Net loss per share, basic and diluted | ($1.17) | Selected Balance Sheet Financial Data As of June 30, 2025, the company reported $539.0 million in cash, $1.31 billion in total assets, and $151.3 million in total stockholders' equity June 30, 2025 Selected Balance Sheet | Balance Sheet Data (as of June 30, 2025, $ thousands) | Amount | | :--- | :--- | | Cash, cash equivalents, and marketable debt securities | 539,039 | | Working capital | 426,544 | | Total assets | 1,306,265 | | Total stockholders' equity | 151,286 |