Ultragenyx Cuts Workforce 20%, Targets 2027 Profitability
Ultragenyx plans to reduce its workforce by about 20% and lower operating expenses as it targets full-year GAAP profitability in 2027. The company also reported a third-quarter loss of $1.16 per share and revenue of $173 million, both below estimates.
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE ) shares were trading lower in Wednesday’s premarket session after the company missed third-quarter estimates and announced substantial expense reductions, including layoffs, as it aims to reach profitability in 2027. Strategic Workforce Reduction and Expense Targets The rare disease-focused company said Tuesday that it is shifting its focus to commercial execution and a more targeted R&D strategy following the completion of several late-stage development programs, including its FDA approvals for glycogen storage disease type Ia (GSDIa) and Sanfilippo syndrome type A.
To support the transition, the company plans to reduce its workforce by approximately 20% to around 1,000 employees and lower operating expenses. It expects 2027 R&D expenses to fall below $350 million and SG&A expenses to fall below $375 million. Read Also: Ultragenyx Wins First Sanfilippo Approval As Analyst Keeps Ratings Modest Commercial Launch Priorities and Focused R&D Strategy Ultragenyx will prioritize the commercial launches of Genglycos and Fayuvi, while continuing to grow revenue from existing products.
R&D spending will focus on generating primary data from the responder endpoint in the ongoing Phase 3 DTX301 study for ornithine transcarbamylase deficiency, assessing a potential development path for UX143 (setrusumab) for osteogenesis imperfecta, and advancing select early-stage programs toward future IND filings. The restructuring is expected to result in approximately $25 million in severance and other one-time charges in the fourth quarter of 2026, with workforce reductions largely completed by year-end. Ultragenyx continues to expect full-year GAAP profitability in 2027 and beyond.
Discontinuation of Wilson Disease and Angelman Syndrome Trials Ultragenyx will discontinue UX701 (rivunatpagene miziparvovec), its AAV9 gene therapy candidate for Wilson disease, after additional data from the dose-finding stage of the Phase 1/2 Cyprus2+ study failed to support advancement into a pivotal Phase 3 trial. Although the fourth dosing cohort and other study data showed clinical activity in some patients, the results did not meet the company’s criteria for a majority of patients to achieve a biochemical response and discontinue standard-of-care treatment. UX701 demonstrated a favorable safety profile across all treated cohorts.
The company will wind down all ongoing studies for GTX-102 for Angelman syndrome. In September, the Phase 3 data showed that the trial missed its primary endpoint, which measured the change from baseline in the Bayley-4 cognitive raw score. Q3 Financial Results, Priority Review Voucher Sales and Guidance Ultragenyx on Tuesday reported a third-quarter loss of $1.16 per share, missing the consensus loss of $1.13. Sales were $173 million, missing the Wall Street estimate of $183.12 million.
In October, the company entered into two definitive agreements to sell the priority review vouchers (PRVs) received upon the approval of Genglycos and Fayuvi for a combined price of $413 million. Ultragenyx reaffirmed its fiscal 2026 sales guidance of $730 million to $760 million, versus the $762.26 million consensus. The guidance excludes revenue from the launches of Fayuvi and Genglycos. Ultragenyx also reaffirmed full-year guidance for Crysvita revenue of $500 million to $520 million and Dojolvi revenue of $100 million to $110 million.
RARE Stock Price Activity: Ultragenyx Pharmaceutical shares are trading lower by 3.67% at $16.02 during premarket trading on Wednesday, according to Pro data. Photo: Shutterstock Read Also: Lockheed Martin, RTX, Northrop Stocks Fall After Democratic Midterm Sweep Raises Questions Over Trump's Defense Spending Plan, While Ukraine Bonds Rally