Ultragenyx Pharmaceutical Inc. — Third Quarter 2026 Earnings Summary

Financial Performance:

• Total revenue for the three months ended September 30, 2026, was $173 million, compared to $160 million in the same period of 2025.
• Net loss for the quarter was $118 million ($1.16 per share), a reduction from $180 million ($1.81 per share) in the prior year period.
• Net loss for the nine months ended September 30, 2026, was $395 million ($3.89 per share), compared to $446 million ($4.55 per share) in 2025.
• Total operating expenses for the quarter were $283 million, down from $331 million in the prior year period, including $34 million in non-cash stock-based compensation.
• Cash, cash equivalents, and marketable securities totaled $401 million as of September 30, 2026, down from $737 million as of December 31, 2025.
• Net cash used in operations for the quarter was $43 million.
• Cost of sales was $32 million for the quarter, up from $28 million in the prior year period.
• Research and development expenses were $154 million for the quarter, down from $216 million in the prior year period.
• Selling, general and administrative expenses were $97 million for the quarter, up from $87 million in the prior year period.

Guidance and Future Outlook:

• Reaffirmed full-year 2026 total revenue guidance of $730 million to $760 million, excluding revenue from new product launches.
• Reaffirmed full-year 2026 CRYSVITA revenue guidance of $500 million to $520 million.
• Reaffirmed full-year 2026 DOJOLVI revenue guidance of $100 million to $110 million.
• Expects to reduce 2027 R&D expenses to less than $350 million and 2027 SG&A expenses to less than $375 million.
• Anticipates achieving full-year GAAP profitability in 2027 and going forward.
• Fourth quarter 2026 financial results are expected to include approximately $25 million of severance and other non-recurring restructuring charges.
• The reduction in force is expected to be substantially completed by the end of 2026.

Business Segments and Product Lines:

• CRYSVITA revenue was $109 million for the quarter, consistent with expected ordering patterns.
• DOJOLVI revenue was $30 million for the quarter.
• EVKEEZA revenue was $24 million for the quarter, driven by increased demand from new country launches and early access.
• MEPSEVII revenue was $10 million for the quarter.
• FDA granted accelerated approval for GENGLYCOS (GSDIa) in August 2026 and standard full approval for FAYUVI (Sanfilippo syndrome type A) in September 2026.
• GENGLYCOS and FAYUVI BLAs were approved in the third quarter, with launches progressing well.
• The GTX-102 program for Angelman syndrome is being wound down.
• The UX701 program for Wilson disease is being terminated after not achieving study goals.
• The UX016 program for GNE myopathy is fully funded through an external venture philanthropy agreement, with dosing to be initiated soon.
• The DTX301 Phase 3 study for OTC deficiency continues, with data expected in the first half of 2027.
• The company is engaging regulatory agencies regarding UX143 for osteogenesis imperfecta to determine a potential path forward.

Market and Competitive Landscape:

• The company is transitioning to a focus on commercial execution and a smaller, more targeted R&D footprint following the completion of late-stage development programs.
• Demand for GENGLYCOS and FAYUVI is growing.
• The company relies on Kyowa Kirin for the commercialization of CRYSVITA in certain major markets, including the U.S. and Canada, and for commercial supply in those markets.

Risks and Challenges:

• Risks include the uncertainty of clinical drug development, regulatory approval timelines, and the potential for the FDA to withdraw accelerated approval of GENGLYCOS if confirmatory studies do not verify clinical benefit.
• Risks related to product liability, manufacturing, and market acceptance are noted.
• Risks associated with the headcount and expense reductions include disruption to operations and the ability to retain key employees.
• Risks include the potential for PRV sales not to close on the expected timeline or at all.
• The company faces risks related to insurance coverage and reimbursement.

Management Commentary and Tone:

• CEO Emil D. Kakkis stated that the company received two BLA approvals within one month and that launches are progressing well with growing demand.
• Management expressed confidence that substantial reductions in headcount and expenses, combined with new launches and base business growth, will drive meaningful profitability in 2027.
• The tone indicates a strategic shift toward commercial execution and a more targeted R&D footprint.

Other Key Points:

• The company entered into two definitive agreements in October 2026 to sell Priority Review Vouchers (PRVs) received from GENGLYCOS and FAYUVI approvals for a combined price of $413 million.
• The sale of the GENGLYCOS PRV closed in October 2026 for $210 million, and the FAYUVI PRV sale is expected to close later in the month for $203 million.
• Net proceeds from both PRV sales are expected to be approximately $350 million.
• The company is reducing headcount by approximately 20%, resulting in a revised workforce of approximately 1,000 employees.
• The company is reducing its real estate footprint as part of the expense reduction strategy.
• The company will host a conference call on November 3, 2026, to discuss the results.