Ultragenyx to Cut 20 Percent of Workforce

Rare Daily Staff

Ultragenyx Pharmaceutical said it will cut approximately 20 percent of its workforce and terminate development of its experimental gene therapy for Wilson disease as it reduces research spending and shifts resources toward launching two newly approved rare disease treatments.

The company announced the restructuring alongside its third quarter financial results. It expects the cuts to leave it with approximately 1,000 employees and reduce annual research and development expenses by at least 50 percent in 2027 compared with 2025.

The changes include completing the wind-down of all ongoing studies of GTX-102 for Angelman syndrome, ending the UX701 Wilson disease program, and reducing the company’s real estate footprint. Ultragenyx expects approximately $25 million in severance and other nonrecurring restructuring charges in the fourth quarter, with the workforce reduction substantially completed by year-end.

The company reiterated its expectation of achieving full-year profitability in 2027 and remaining profitable thereafter.

“With substantial reductions in headcount and expenses, combined with two launches and continued growth in our base business, we expect meaningful profitability in 2027 and going forward,” said Emil Kakkis, Ultragenyx’s president and CEO.

Ultragenyx said additional results from the dose-finding portion of its Cyprus2+ study of UX701, including a fourth dosing cohort, did not meet the criteria needed to advance the gene therapy into pivotal phase 3 development.

Although some patients showed clinical activity, the program did not meet the requirement that a majority of patients achieve both a biochemical response and elimination of standard treatment. The company reported a favorable safety profile across all treated cohorts.

Ultragenyx said it is working with clinical sites and patient advocacy organizations to support patients through the program’s termination. It plans to present the completed study’s results at a future medical or scientific meeting.

The reductions follow FDA approvals of Ultragenyx’s first two gene therapies during the third quarter. In August, the FDA granted accelerated approval to Genglycos for adults and children ages eight and older with glycogen storage disease type Ia. In September, the agency granted full approval to Fayuvi for pediatric patients with Sanfilippo syndrome type A.

Ultragenyx described the approvals as the first for treatments targeting the two diseases. Genglycos remains subject to confirmatory study requirements associated with its accelerated approval.

The company received a rare pediatric disease priority review voucher with each approval and subsequently agreed to sell the vouchers for a combined $413 million. The $210 million Genglycos voucher sale closed in late October, while the $203 million Fayuvi voucher sale is expected to close later in November. Combined net proceeds are expected to total approximately $350 million. The company separately announced the two sale agreements in October.

Its remaining research priorities include obtaining additional phase 3 results for DTX301, a gene therapy for ornithine transcarbamylase deficiency, and determining whether there is a viable regulatory path for setrusumab, its experimental antibody for osteogenesis imperfecta.

The company also plans measured investment in early-stage programs. A phase 1/2 study of UX016 for GNE myopathy is fully funded through clinical proof of concept under a venture philanthropy agreement with a patient group, according to Ultragenyx.

Photo: Emil Kakkis, Ultragenyx’s president and CEO.