RARE · Ultragenyx Pharmaceutical Inc.

Ultragenyx reported a mixed first quarter of 2026, with total revenue declining to $136 million from $139 million in the prior year period. The revenue performance was driven by a $10 million decrease in Crysvita revenue to $93 million, attributed to expected seasonality in the U.S. and Canada and ordering patterns in Brazil. This decline was partially offset by growth in Evkeeza revenue, which increased to $18 million from $11 million due to new country launches, and a slight increase in Dojolvi revenue to $18 million. The company's profitability metrics deteriorated, with net loss widening to $185 million, or $1.84 per share, compared to a net loss of $151 million, or $1.57 per share, in the first quarter of 2025. The increased loss was driven by higher operating expenses, which rose to $305 million from $282 million. This increase included $30 million in non-cash stock-based compensation and a $30 million expense related to a previously announced restructuring. R&D expenses specifically increased to $187 million from $166 million, reflecting continued investment in the pipeline. Despite the quarterly performance, management reaffirmed its full-year 2026 financial guidance, projecting total revenue between $730 million and $760 million and expecting combined R&D and SG&A expenses to be flat to slightly down versus 2025. The company maintains its path to profitability in 2027. Significant regulatory milestones were highlighted, including the FDA acceptance of the BLA for DTX401 with a PDUFA date of August 23, 2026, and the acceptance of the resubmitted BLA for UX111 with a PDUFA date of September 19, 2026. Additionally, the Phase 3 Aspire study for GTX-102 is on track for data readout in the second half of 2026. The company ended the quarter with $534 million in cash, cash equivalents, and marketable securities, with net cash used in operations for the quarter totaling $197 million.