Ultragenyx Pharmaceutical Inc.: Q4 FY2025 earnings call

What management said about Q4 FY2025, from the documents filed with the SEC on 12 February 2026: press release. Guidance figures: 7 new, 1 kept. The quarter’s numbers.

Read the filing

Key takeaways

• Full-year revenue up 20%: Total 2025 revenue was $673 million, up 20% on 2024, with product sales of $369 million and royalty revenue of $304 million. Source
• Crysvita revenue of $481 million: Full-year Crysvita revenue rose 17% to $481 million, made up of Latin America and Türkiye product sales and royalties from the U.S., Canada and Europe. Source
• 10% workforce cut announced: A 10% workforce reduction affecting about 130 employees was announced in connection with the restructuring plan. Source
• 2027 R&D to fall 38%: R&D is expected to fall about $280 million, or 38%, from 2025 levels in 2027, as multiple Phase 3 studies complete and early-stage research is reduced. Source
• UX111 received incomplete response letter: The company received an Incomplete Response Letter on the resubmitted UX111 BLA for Sanfilippo syndrome type A, the day of the release. Source
• GTX-102 Aspire data due 2H 2026: Pivotal Phase 3 Aspire data for GTX-102 in Angelman syndrome are expected in the second half of 2026. Phase 2/3 Aurora dosing began in October 2025. Source
• 2026 revenue from current products guided: 2026 total revenue from current products, excluding potential new launch revenue, is expected at $730 million to $760 million, 8% to 13% above 2025. Source
• Path to profitability in 2027 reiterated: Management said reduced and partly reinvested expenses, plus planned revenue growth from current and new launches, are designed to keep the company on its 2027 profitability path. Source

“The year ahead marks an important turning point for the company, as we approach two potential product launches and a pivotal data readout that, together, could significantly accelerate our commercial revenue trajectory,”

Emil D. Kakkis, M.D., Ph.D., chief executive officer and president of Ultragenyx

Guidance

What changed from last quarter

• New guidance: The company had said it would issue 2026 financial guidance in February 2026; the guidance is now given for total revenue, Crysvita and Dojolvi.
• New: A strategic restructuring plan was initiated to reduce and focus expenses and headcount, with a 10% workforce reduction. Source
• New: The company received an Incomplete Response Letter on its resubmitted UX111 BLA on the day of the release. Source

Results and growth drivers

• Full-year revenue up 20%: Total 2025 revenue was $673 million, up 20% on 2024, with product sales of $369 million and royalty revenue of $304 million. Source
• Fourth-quarter revenue up 25%: Fourth-quarter 2025 revenue was $207 million, up from $165 million a year earlier, with Crysvita revenue of $145 million within that total. Source
• Net loss slightly higher year on year: The 2025 net loss was $575 million, or $5.83 per share, against $569 million, or $6.29 per share, in 2024. Source

Products and segments

• Crysvita revenue of $481 million: Full-year Crysvita revenue rose 17% to $481 million, made up of Latin America and Türkiye product sales and royalties from the U.S., Canada and Europe. Source
• Latin America sales up 31%: Latin America and Türkiye product sales were $177 million for 2025, up 31%, and $40 million in the fourth quarter, up from $22 million a year earlier. Source
• Evkeeza demand building outside US: Evkeeza revenue was $59 million for 2025, up 84%, which management attributed to demand building after launches outside the United States. Source
• Dojolvi and Mepsevii grew: Dojolvi revenue was $96 million for 2025, up 9%. Mepsevii revenue was $37 million in 2025, against $30 million in 2024. Source

“We are implementing a strategic restructuring plan to reduce our operating expenses and ensure our resources are squarely aligned with our highest-impact opportunities, while leading the future of rare disease with multiple first ever treatments.”

Emil D. Kakkis, M.D., Ph.D., chief executive officer and president of Ultragenyx

Costs and restructuring

• 2025 operating expenses reached $1.2 billion: Total 2025 operating expenses were $1.2 billion, with R&D of $750 million and SG&A of $349 million; stock-based compensation was $153 million of the total. Source
• 10% workforce cut announced: A 10% workforce reduction affecting about 130 employees was announced in connection with the restructuring plan. Source
• 2026 combined costs flat to lower: Combined R&D and SG&A expenses for 2026 are expected to be flat to down low-single digits versus 2025. Source
• 2027 R&D to fall 38%: R&D is expected to fall about $280 million, or 38%, from 2025 levels in 2027, as multiple Phase 3 studies complete and early-stage research is reduced. Source
• 2027 SG&A to rise for launches: SG&A is expected to increase in 2027 to support new product launches and existing approved products, while combined R&D and SG&A is expected to fall at least 15% against 2025. Source
• About $50 million restructuring charges: Management expects about $50 million of severance, manufacturing and other non-recurring restructuring charges in 2026, included within the combined expense expectation. Source

Pipeline and regulatory updates

• UX111 received incomplete response letter: The company received an Incomplete Response Letter on the resubmitted UX111 BLA for Sanfilippo syndrome type A, the day of the release. Source
• Letter seeks CMC documentation: The letter asks for additional supporting documentation on CMC responses; the company said it will provide this in a resubmission. Source
• DTX401 action date Q3 2026: The DTX401 gene therapy for glycogen storage disease type Ia had its rolling BLA completed in December 2025, with an anticipated PDUFA date in the third quarter of 2026. Source
• GTX-102 Aspire data due 2H 2026: Pivotal Phase 3 Aspire data for GTX-102 in Angelman syndrome are expected in the second half of 2026. Phase 2/3 Aurora dosing began in October 2025. Source
• UX701 data now expected in 2026: Enrollment is complete for the fourth cohort of the dose-finding stage of UX701 for Wilson disease. The January release had said data in the first half of 2026. Source

Cash and balance sheet

• Cash and securities of $737 million: Cash, cash equivalents and marketable securities were $737 million at December 31, 2025, against $745 million a year earlier. Source
• Operating cash use of $466 million: Net cash used in operations was $100 million in the fourth quarter and $466 million for the full year 2025. Source
• Stockholders' deficit of $80 million: Total stockholders' equity was a deficit of $80 million at December 31, 2025, against equity of $255 million a year earlier. Source

Revenue expectations for 2026

• 2026 revenue from current products guided: 2026 total revenue from current products, excluding potential new launch revenue, is expected at $730 million to $760 million, 8% to 13% above 2025. Source
• Crysvita 2026 range with Brazil timing: Crysvita is expected at $500 million to $520 million in 2026; underlying global demand growth is partly offset by expected Brazil ordering timing. Source
• Dojolvi 2026 range set: Dojolvi revenue is expected at $100 million to $110 million in 2026, against $96 million in 2025. Source

Strategy and profitability path

• Path to profitability in 2027 reiterated: Management said reduced and partly reinvested expenses, plus planned revenue growth from current and new launches, are designed to keep the company on its 2027 profitability path. Source
• Two potential approvals and Phase 3 data: Management cited two potential approvals and pivotal Phase 3 Aspire data for GTX-102 as 2026 catalysts. Source
• Restructuring focused on largest drivers: The restructuring plan reduces headcount and expenses and concentrates resources on the company's largest value drivers. Source

Coming up, as management said on 12 February 2026

• DTX401 action date third quarter 2026: The anticipated PDUFA action date for DTX401 is in the third quarter of 2026. Source
• Aspire Phase 3 data second half 2026: Pivotal Phase 3 Aspire data for GTX-102 are expected in the second half of 2026. Source
• UX111 resubmission to follow letter: The company will provide additional CMC documentation in a UX111 resubmission following the Incomplete Response Letter. Source
• UX701 data expected in 2026: Data from the dose-finding stage of the UX701 Cyprus2+ study are expected in 2026. Source

Figures management reported

Risks management discussed

• Regulatory: The UX111 BLA received an Incomplete Response Letter asking for more CMC documentation, which must be provided before review proceeds. Source
• Demand: Expected timing of Brazil ordering patterns is a factor offsetting underlying global Crysvita demand growth in the 2026 range. Source

Earlier plans management reported on

• Ahead: Preliminary 2025 revenue was estimated to exceed the top end of the guidance range.
• On track: The January release said resubmission was on track for early 2026; the February release reports the BLA was resubmitted in January 2026.
• Not stated: Management reiterated the path to profitability in 2027 alongside the restructuring plan. Source

Other quarters

• Q3 FY2026 earnings call
• Q2 FY2026 earnings call
• Q1 FY2026 earnings call
• Q3 FY2025 results

Summarised with AI from the documents the company filed with the SEC. Every point is checked against the filing before it is shown: management’s own words word for word, figures against the line or table row they come from. A point that fails the check is left out. Source opens the filing at that line.