Ultragenyx To Cut About 20% Of Staff, Wind Down GTX-102 Studies And Drop UX701
The rare-disease drugmaker reaffirmed 2027 profitability and said the cuts should lower 2027 R&D spending by at least 50% versus 2025.
Ultragenyx Pharmaceutical, which develops treatments for serious rare and ultra-rare genetic diseases, said Tuesday it will cut about 20% of its workforce, leaving roughly 1,000 employees. It is also winding down all GTX-102 studies and terminating UX701, while reaffirming it expects full-year profitability in 2027.
Where the cuts land
The company expects the layoffs to be substantially completed by the end of 2026. It expects 2027 R&D expenses below $350 million and 2027 SG&A (selling, general and administrative) expenses below $375 million, and said the reductions should cut 2027 R&D spending by at least 50% versus 2025, according to its earnings release.
The plan also includes finishing the wind-down of all ongoing GTX-102 studies for Angelman syndrome and ending UX701 for Wilson disease, which did not achieve its study goals. Real estate footprint is being reduced too. Fourth-quarter results are expected to carry about $25 million of severance and other one-time restructuring charges.
R&D will concentrate on primary data from the ongoing DTX301 Phase 3 study, a determination on whether UX143 (setrusumab) for osteogenesis imperfecta has a viable path, and measured spending on early-stage programs. DTX301 data on its second primary endpoint are expected in the first half of 2027.
Third quarter and the cash pile
Third-quarter revenue was $173 million, against $160 million a year earlier. The net loss narrowed to $118 million, or $1.16 per share, from $180 million, or $1.81 per share. Crysvita brought in $109 million and Dojolvi $30 million; the company reaffirmed 2026 revenue guidance of $730 million to $760 million, which excludes new launches.
Cash, equivalents and marketable securities were $401 million at September 30, versus $737 million at December 31, 2025. Net cash used in operations was $43 million in the quarter.
CEO Emil D. Kakkis said that with the headcount and expense cuts, plus two launches and growth in the base business, "we expect meaningful profitability in 2027 and going forward."
Two vouchers worth $413 million
The restructuring follows FDA approval of GENGLYCOS in August and FAYUVI in September, the company's first gene therapy treatments, which completed its late-stage programs. Each earned a priority review voucher. The company agreed to sell them for a combined $413 million: $210 million for the GENGLYCOS voucher and $203 million for FAYUVI.
The GENGLYCOS sale closed October 30, with 20% of the gross proceeds owed to the NIH under a patent license. The FAYUVI sale is expected to close later this month. Net proceeds from both are expected to be about $350 million.
Management hosts a call at 2 p.m. PT on Tuesday to walk through the quarter and the restructuring.
Sources
- 8-K Filing — Ultragenyx Pharmaceutical Inc. (RARE) — SEC EDGAR
- Exhibit rare-ex99_1.htm — Ultragenyx Pharmaceutical Inc. 8-K exhibit — SEC EDGAR
