Ultragenyx Pharmaceutical Inc.: Q1 FY2026 earnings call

What management said about Q1 FY2026, from the documents filed with the SEC on 5 May 2026: press release. Guidance figures: 5 kept. The quarter’s numbers.

Read the filing

Key takeaways

• Total revenue slightly lower year over year: Total revenue was $136 million, compared with $139 million in the first quarter of 2025. Source
• Latin America and Türkiye product sales fell: Crysvita product sales in Latin America and Türkiye were $46 million, against $55 million a year earlier. Source
• Operating expenses rose to $305 million: Total operating expenses were $305 million, against $282 million, including $30 million of stock-based compensation and $30 million of restructuring expense. Source
• Net loss widened to $185 million: Net loss was $185 million, or $1.84 per share, against $151 million, or $1.57 per share, a year earlier. Source
• Cash fell to $534 million: Cash, cash equivalents and marketable securities were $534 million at March 31, 2026, against $737 million at December 31, 2025. Source
• DTX401 BLA accepted with Priority Review: The FDA accepted the DTX401 BLA for GSDIa in February 2026 and granted Priority Review. Source
• GTX-102 long-term extension data: As of the March 2026 cut-off, 74 patients had been treated with GTX-102, and 66 continued in the long-term extension study. Source
• DTX301 ammonia reduction versus placebo: At Week 36, DTX301 patients showed an 18% reduction in 24-hour plasma ammonia versus placebo, statistically significant at p=0.018. Source
• Profitability path reaffirmed: The company reaffirmed its 2026 financial guidance and said it remains on path to profitability in 2027. Source

“This is an important year for Ultragenyx with two BLAs under review and our Angelman Phase 3 readout coming soon.”

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

Guidance

What changed from last quarter

• Guidance kept: Full-year 2026 financial guidance reaffirmed, with no change to the revenue or expense ranges. Source
• New: DTX301 Phase 3 Week 36 ammonia results were announced in March 2026, after the prior quarter's report. Source
• New: The FDA said an Advisory Committee meeting for DTX401 is not anticipated at this time. Source
• New: The FDA accepted the resubmitted UX111 BLA for review in April 2026. Source
• New: The FDA cleared the IND application for UX016, a GNE myopathy prodrug. Source

Revenue and growth drivers

• Total revenue slightly lower year over year: Total revenue was $136 million, compared with $139 million in the first quarter of 2025. Source
• Product and royalty revenue split: Product sales were $89 million, against $91 million, and royalty revenue was $47 million, against $48 million. Source
• Crysvita down while Evkeeza grew: Crysvita revenue fell to $93 million from $103 million, while Evkeeza revenue rose to $18 million from $11 million. Source

Crysvita and regional mix

• Crysvita reflected seasonality and Brazil timing: Management tied first-quarter Crysvita revenue to seasonality in the U.S. and Canada and to ordering patterns in Brazil. Source
• Latin America and Türkiye product sales fell: Crysvita product sales in Latin America and Türkiye were $46 million, against $55 million a year earlier. Source
• Royalties from U.S., Canada and Europe: Royalty revenue was $39 million from U.S. and Canada, against $41 million, and $8 million from Europe, against $7 million. Source

“The latest long-term GTX-102 Phase 1/2 data further support the potential of the program, as it heads toward Phase 3 results later this year.”

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

Products and launches

• Evkeeza revenue rose on new launches: Evkeeza revenue was $18 million, against $11 million, driven by demand from new country launches and early access. Source
• Dojolvi revenue slightly higher: Dojolvi revenue was $18 million, compared with $17 million in the first quarter of 2025. Source
• Mepsevii revenue lower year over year: Mepsevii revenue was $7 million, against $8 million a year earlier. Source

Operating costs and restructuring

• Operating expenses rose to $305 million: Total operating expenses were $305 million, against $282 million, including $30 million of stock-based compensation and $30 million of restructuring expense. Source
• R&D spend increased year over year: Research and development expense was $187 million, against $166 million. SG&A was $88 million, against $87 million, and cost of sales was $30 million, against $29 million. Source
• Stock-based compensation fell: Non-cash stock-based compensation was $30 million, against $40 million. Restructuring expense was $30 million, with none reported a year earlier. Source

Net loss and share count

• Net loss widened to $185 million: Net loss was $185 million, or $1.84 per share, against $151 million, or $1.57 per share, a year earlier. Source
• Royalty-sale interest expense rose: Non-cash interest expense on liabilities for sales of future royalties was $21 million, against $14 million. Other income was $6 million, against $7 million. Source
• Weighted share count higher: Shares used in computing net loss per share were 100.6, against 96.3 a year earlier. Source

Cash and balance sheet

• Cash fell to $534 million: Cash, cash equivalents and marketable securities were $534 million at March 31, 2026, against $737 million at December 31, 2025. Source
• Operating cash outflow was $197 million: Net cash used in operations was $197 million, including annual bonus payments and $38 million for UX143 manufacturing activities. Source
• Working capital and equity deficit changed: Working capital was $332 million, against $567 million. Total assets were $1,296 million, against $1,532 million. Stockholders' deficit was $236 million, against $80 million. Source

Regulatory reviews and filings

• DTX401 BLA accepted with Priority Review: The FDA accepted the DTX401 BLA for GSDIa in February 2026 and granted Priority Review. Source
• No advisory committee expected for DTX401: The FDA said an Advisory Committee meeting for DTX401 is not anticipated at this time. Source
• UX111 resubmission accepted for review: The FDA accepted the resubmitted UX111 BLA in April 2026, seeking accelerated approval for Sanfilippo syndrome type A. Source
• UX111 longer-term data included in filing: The resubmitted BLA included follow-up of up to eight years, presented in February 2026 at the WORLD Symposium, with a durable treatment effect across evaluations. Source

Clinical programs

• GTX-102 long-term extension data: As of the March 2026 cut-off, 74 patients had been treated with GTX-102, and 66 continued in the long-term extension study. Source
• GTX-102 safety profile consistent: Management reported a consistent safety profile over multiple years of treatment, with no new cases of transient lower extremity weakness. Source
• Aspire Phase 3 enrolled 129 patients: The Phase 3 Aspire study in patients with a full maternal UBE3A deletion enrolled 129 patients, randomized 1:1 to GTX-102 or sham. Source
• DTX301 ammonia reduction versus placebo: At Week 36, DTX301 patients showed an 18% reduction in 24-hour plasma ammonia versus placebo, statistically significant at p=0.018. Source
• UX701 fourth cohort enrolled: Enrollment is complete for the fourth cohort of the dose-finding stage of the Cyprus2+ study for UX701 in Wilson disease. Source

Strategy and profitability path

• Profitability path reaffirmed: The company reaffirmed its 2026 financial guidance and said it remains on path to profitability in 2027. Source
• Two gene therapy approvals anticipated: Management said it is preparing for two potential gene therapy approvals and launches in two urgent diseases without approved therapies. Source
• Two BLAs and Angelman readout this year: The CEO described 2026 as an important year with two BLAs under review and the Angelman Phase 3 readout approaching. Source

Coming up, as management said on 5 May 2026

• DTX401 FDA action date: The FDA action date for DTX401 is August 23, 2026. Source
• UX111 FDA action date: The FDA action date for UX111 is September 19, 2026. Source
• Aspire Phase 3 data timing: Data from the Aspire Phase 3 study are expected in the second half of 2026. Source
• Aurora enrollment completion: Enrollment in the Phase 2/3 Aurora study is expected to complete in the second half of 2026. Source
• UX701 dose-finding data: Data from the dose-finding stage of UX701 are expected in 2026. Source
• DTX301 second endpoint data: Data on the second primary endpoint for DTX301, covering treatment burden, are expected in the first half of 2027. Source
• UX016 Phase 1/2 study start: An externally funded Phase 1/2 study of UX016 is expected to begin in the second half of 2026. Source
• GTX-102 updated data presentation: Updated GTX-102 efficacy and safety data are planned for presentation at a future scientific meeting, with no date given. Source

Figures management reported

Risks management discussed

• Demand: Crysvita revenue reflects seasonality in the U.S. and Canada and ordering patterns in Brazil, which move quarter to quarter. Source

Earlier plans management reported on

• Not stated: The company reaffirmed its full year 2026 and 2027 guidance for combined R&D and SG&A operating expenses. Source
• On track: The company said it remains on path to profitability in 2027. Source

Other quarters

• Q3 FY2026 earnings call
• Q2 FY2026 earnings call
• Q4 FY2025 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.