Ultragenyx Revenue Reaches $173 Million as Company Plans to Cut R&D Spending in 2027
Key Facts
Ultragenyx reported revenue of about $173 million in the third quarter ended September 30, 2026, compared with $160 million in the same period a year earlier, according to results announced by the company on November 3. Alongside the results, the company reaffirmed a restructuring plan targeting research and development expenses of less than $350 million in 2027 and selling, general and administrative expenses of less than $375 million. It said it expects to reduce its workforce by about 20%, to approximately 1,000 employees. The company reaffirmed its expectation of achieving annual profitability under GAAP in 2027 and beyond. These targets put spending cuts at the center of the company’s plan to reach profitability as it continues to report quarterly losses.
Quarterly revenue was below the level in the second quarter of 2026, when the company reported $214 million and maintained its full-year revenue guidance at $730 to $760 million. In August, the company described the second quarter as the strongest in its history up to that point, and its chief executive said the results kept it on track for profitability in 2027. In the September quarter, its products generated revenue of $109 million for CRYSVITA, $30 million for DOJOLVI, $24 million for EVKEEZA and $10 million for MEPSEVII. These figures show the distribution of revenue among the four products listed by the company in its results. Analysts’ estimates before the announcement had pointed to revenue of $182.19 million and a loss per share of $1.10. The actual net loss was $118 million, or $1.16 per share, compared with a loss of $180 million, or $1.81 per share, a year earlier.
Research and development expenses fell to $154 million in the third quarter from $216 million in the same period of 2025, while selling, general and administrative expenses rose to $97 million from $87 million. The new targets follow the company’s statement in August that combined expenses for the two categories in 2027 would fall by at least 15% compared with 2025; the current plan sets a ceiling for each category and adds an expected workforce reduction. The measures include ending studies of GTX-102 for Angelman syndrome and discontinuing the UX701 program for Wilson disease after it failed to meet the study’s objectives. The company said on September 2 that the Phase 3 Aspire study of GTX-102 failed to meet its primary and key secondary endpoints. Its shares then fell more than 40% in premarket trading on September 3, according to Reuters.
The plan also relies on cash resources and sales of priority review vouchers. Cash, cash equivalents and marketable securities totaled $401 million on September 30, while the company used $43 million in cash in operations during the three months ended on that date. In October, it entered into two agreements to sell priority review vouchers for a total of $413 million, with net proceeds expected to be about $350 million after both transactions close. The company said the sale of the GENGLYCOS voucher closed in the week before the results announcement, while the FAYUVI voucher sale was expected to close later in November. GENGLYCOS received accelerated approval in August 2026, while FAYUVI received full approval in September. Fourth-quarter results are expected to include about $25 million in one-time severance and restructuring costs, and the workforce reduction is expected to be largely completed by the end of 2026.
On development, the company expects data on the primary response endpoint from the Phase 3 DTX301 study in the first half of 2027. The timing comes as the company reallocates spending among its research and development programs and moves to focus more on products that have received recent regulatory approvals. The 2027 annual profitability target remains the framework the company has tied to expense cuts and workforce adjustments. In trading on November 2, RARE shares closed at $16.3, according to EL7 data. The results announcement provides the latest reading on the company’s revenue, losses and cash flow, alongside the measures it said it will implement in the coming period.