Ultragenyx Pharmaceutical Inc.: Q2 FY2026 earnings call

What management said about Q2 FY2026, from the documents filed with the SEC on 4 August 2026: press release. Guidance figures: 10 kept. The quarter’s numbers.

Read the filing

Key takeaways

• Record quarterly revenue reported: Total revenue was $214 million, up from $167 million in the second quarter of 2025. Management described it as the highest quarterly revenue in company history. Source
• Net loss narrowed year on year: Net loss was $92 million, or $0.90 per share, against $115 million, or $1.17 per share, in the second quarter of 2025. Source
• Cash balance declined this year: Cash, cash equivalents and marketable securities were $436 million at June 30, 2026, down from $534 million at March 31 and $737 million at December 31, 2025. Source
• Two gene therapies under FDA review: The FDA accepted DTX401 for review in February 2026 and the resubmitted UX111 BLA in April 2026. Both are seeking approval for rare diseases. Source
• Angelman Phase 3 readout pending: Management listed a pivotal Phase 3 GTX-102 data readout in Angelman syndrome among second-half 2026 catalysts, alongside two FDA action dates. Source
• Positioned for growth after launches: Management said the company is preparing for two gene therapy launches and expects these to support growth in the years ahead. Source

“In the second quarter we generated the highest quarterly revenue in the history of the company, supporting our full-year revenue guidance, and keeping us on track toward profitability in 2027,”

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

Guidance

What changed from last quarter

• Guidance kept: 2026 financial guidance was reaffirmed, including total revenue and combined R&D and SG&A expense ranges. The company stated it remains on path to profitability in 2027. Source
• Guidance kept: The company reaffirmed its 2026 and 2027 guidance for combined R&D and SG&A operating expenses. Source
• New: Dojolvi was listed on Japan's National Health Insurance drug price list and launched in Japan in May 2026, after conditional approval on March 23, 2026. Source

Results and growth drivers

• Record quarterly revenue reported: Total revenue was $214 million, up from $167 million in the second quarter of 2025. Management described it as the highest quarterly revenue in company history. Source
• Net loss narrowed year on year: Net loss was $92 million, or $0.90 per share, against $115 million, or $1.17 per share, in the second quarter of 2025. Source
• Six-month revenue higher: Six-month total revenue was $350 million, against $306 million a year earlier. Six-month net loss was $277 million, the same per-share loss as the prior year period. Source

Products and regions

• Crysvita revenue split across lines: Crysvita revenue includes Latin America and Türkiye product sales, U.S. and Canada royalties, and Europe royalties. Management tied the quarter to seasonality and ordering patterns. Source
• Evkeeza growth from new countries: Evkeeza revenue rose on demand from new country launches and early access, according to management. Source
• Mepsevii revenue up slightly: Mepsevii revenue rose from the second quarter of 2025, and six-month revenue was level with the prior year. Source

“We are ready to launch two gene therapy products and are preparing for a pivotal Phase 3 GTX-102 data readout in Angelman syndrome.”

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

Costs and operating expenses

• Operating expenses and stock compensation: Total operating expenses were $289 million, against $275 million a year earlier. Non-cash stock-based compensation was $34 million, against $39 million. Source
• Research and development close to prior year: Research and development expense was close to the level of the second quarter of 2025, and SG&A was nearly unchanged. Source
• Restructuring expense not repeated: The company recorded $30 million of restructuring expense in the first quarter of 2026 and none in the second quarter. Source
• Royalty financing interest expense rose: Non-cash interest expense on liabilities for sales of future royalties was $22 million in the quarter, against $14 million a year earlier. Source

Cash, balance sheet and shares

• Cash balance declined this year: Cash, cash equivalents and marketable securities were $436 million at June 30, 2026, down from $534 million at March 31 and $737 million at December 31, 2025. Source
• Operating cash use lower than first quarter: Net cash used in operations was $97 million in the quarter. The first quarter included annual bonus payments and $38 million tied to UX143 manufacturing. Source
• Stockholders' deficit and working capital: Working capital was $255 million at June 30, 2026, and the total stockholders' deficit was $291 million, against $80 million at December 31, 2025. Source
• Weighted share count increased: Shares used in computing net loss per share were 101.9 million in the quarter, against 98.5 million a year earlier. Source

Pipeline and regulatory progress

• Two gene therapies under FDA review: The FDA accepted DTX401 for review in February 2026 and the resubmitted UX111 BLA in April 2026. Both are seeking approval for rare diseases. Source
• Angelman Phase 3 readout pending: Management listed a pivotal Phase 3 GTX-102 data readout in Angelman syndrome among second-half 2026 catalysts, alongside two FDA action dates. Source
• Wilson disease dosing stage progressing: Enrollment is complete for the fourth cohort in the dose-finding stage of the Cyprus2+ study for UX701. Source
• OTC study continuing toward second endpoint: The DTX301 Phase 3 study continues with patients in treatment and cross-over groups through 64 weeks of follow-up. Source
• UX016 IND cleared: The FDA cleared the investigational new drug application for UX016, a substrate replacement therapy for GNE myopathy. Source

Strategy and path to profitability

• Positioned for growth after launches: Management said the company is preparing for two gene therapy launches and expects these to support growth in the years ahead. Source

Coming up, as management said on 4 August 2026

• DTX401 FDA action date: The FDA action date for DTX401 in glycogen storage disease type Ia is August 23, 2026. Source
• UX111 FDA action date: The FDA action date for UX111 in Sanfilippo syndrome type A is September 19, 2026. Source
• Aspire Phase 3 data readout: Data from the Aspire study of GTX-102 in Angelman syndrome are expected in a September or October timeframe. Source
• UX701 dose-finding data: Data from the dose-finding stage of the Cyprus2+ study are expected in the fourth quarter of 2026. Source
• DTX301 second endpoint data: Data from the second primary endpoint of the DTX301 study are expected in the first half of 2027. Source
• Aurora enrollment completion: Enrollment in the open-label Aurora study is expected to complete in the second half of 2026. Source

Figures management reported

Risks management discussed

• Demand: Management attributed the quarter's Crysvita revenue to seasonality in the U.S. and Canada and to ordering patterns in Latin America. Source

Earlier plans management reported on

• On track: Management said the company remains on track toward profitability in 2027. Source
• Not stated: Management said the company is ready to launch two gene therapy products, subject to FDA action on the two applications. Source

Other quarters

• Q3 FY2026 earnings call
• Q1 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.