Ultragenyx Q4 Loss Wider Than Expected, Revenues Increase Y/Y

Ultragenyx Pharmaceutical RARE reported fourth-quarter 2025 loss of $1.29 per share, wider than the Zacks Consensus Estimate of a loss of $1.20. The company had incurred a loss of $1.39 per share in the year-ago quarter.

Ultragenyx’s total revenues amounted to $207 million in the reported quarter, up 25% year over year, on the back of higher product sales. The top line beat the Zacks Consensus Estimate of $203 million.

The company markets four drugs, namely Crysvita, Mepsevii, Dojolvi and Evkeeza. Crysvita is approved for treating X-linked hypophosphatemia, an inherited disorder and tumor-induced osteomalacia, an ultra-rare disease. Mepsevii is approved to treat Mucopolysaccharidosis VII, also known as Sly syndrome. Dojolvi is approved for treating all forms of long-chain fatty acid oxidation disorders. Evkeeza is indicated for homozygous familial hypercholesterolemia (HoFH).

In 2022, Ultragenyx announced a license and collaboration agreement with Regeneron Pharmaceuticals REGN for Evkeeza, which is approved in multiple geographies as a first-in-class therapy for use together with diet and other low-density lipoprotein-cholesterol-lowering therapies to treat adults and adolescents aged 12 years and older with HoFH. Per the deal, RARE has obtained the rights to develop, commercialize and distribute Evkeeza outside the United States. The regions include the European Economic Area. The collaboration with Regeneron for Evkeeza gives Ultragenyx a fourth approved product that adds to the top line. However, REGN solely commercializes Evkeeza in the United States.

RARE’s Q4 Results in Detail

Crysvita’s total revenues were $145 million, up 25% year over year, driven by increased demand for approved indications. Crysvita’s net product revenues in the fourth quarter of 2025 included $97 million from North America, $40 million from Latin America and Turkey, and $8 million from Europe.

Mepsevii product revenues increased 63% year over year to $13 million in the reported quarter. Dojolvi product revenues were $32 million, up 3%, driven by new patient demand. Evkeeza recorded sales of $17 million in the fourth quarter, up 70% as Ultragenyx continues to launch the drug in its territories outside of the United States.

In the past six months, shares of Ultragenyx have lost 19% against the industry’s 22% growth.

Operating expenses of $321 million in the quarter rose 12% year over year due to increased investments in multiple late-stage pipeline programs and marketing costs for approved drugs. Operating expenses included research and development (R&D) expenses of $203 million (up 8%), selling, general and administrative (SG&A) expenses of $89 million (up 9%), and cost of sales of $29 million (up 71%).

Cash, cash equivalents and marketable debt securities amounted to $737 million as of Dec. 31, 2025, compared with $447 million as of Sept. 30, 2025.

RARE’s 2025 Results

Total revenues in 2025 came in at $673 million, up 20% from $560 million recorded in 2024. The figure missed the Zacks Consensus Estimate of $675 million.

The net loss per share in 2025 came in at $5.83, which is narrower than the loss of $6.29 reported in 2024. The reported figure was wider than the Zacks Consensus Estimate of a loss of $5.71 per share. Net cash used in operations in 2025 was $466 million.

RARE’s 2026 Financial Guidance

Ultragenyx expects total revenues in 2026, excluding potential revenues from new product launches, between $730 million and $760 million, which suggests growth of approximately 8-13% compared to 2025. Crysvita revenues in 2026 are expected to be in the range of $500-$520 million, indicating growing underlying global demand partially offset by the expected timing of ordering patterns in Brazil. On the other hand, Dojolvi revenues are expected to be between $100 million and $110 million in 2026.

In the same press release, Ultragenyx reported launching a strategic restructuring plan aimed at cutting costs, reducing headcount, and sharpening its focus on its highest-value programs, as it targets profitability in 2027. The company said the move is intended to streamline operations while supporting revenue growth from existing and upcoming product launches.

As part of the plan, Ultragenyx will reduce its workforce by 10%, affecting about 130 employees. The company expects combined R&D and SG&A expenses in 2026 to be flat to down low-single digits compared with 2025, including roughly $50 million in severance, manufacturing and other one-time restructuring costs.

Looking ahead to 2027, Ultragenyx projects R&D expenses will fall 38% from 2025 levels, or about $280 million, driven by the completion of several phase III studies and a pullback in early-stage research. While SG&A spending is expected to rise to support product launches and commercial efforts, total R&D and SG&A expenses are forecasted to decline at least 15% compared to 2025.

Ultragenyx Pharmaceutical Inc. Price, Consensus and EPS Surprise

Ultragenyx Pharmaceutical Inc. price-consensus-eps-surprise-chart | Ultragenyx Pharmaceutical Inc. Quote

 

RARE’s Key Pipeline Updates

Earlier in 2025, Ultragenyx faced a massive setback when the FDA issued a complete response letter (CRL) for its biologics license application (BLA) for UX111, which is being developed as a treatment for patients with MPS IIIA. The company resubmitted the BLA in January 2026, incorporating extended follow-up data, in line with prior FDA feedback during the last clinical review. Ultragenyx reported that it has received an Incomplete Response Letter (IRL) from the FDA on the resubmitted BLA. The letter requests additional supporting documentation tied to its prior CRL CMC responses, which the company plans to address in a subsequent resubmission.

Ultragenyx and its partner, Mereo BioPharma, are jointly developing UX143 (setrusumab) monoclonal antibody, forpediatric and young adult patients with osteogenesis imperfecta (OI) in two late-stage studies, Orbit and Cosmic. In late 2025, the companies reported that neither study achieved the primary endpoint of reduction in annualized clinical fracture rate compared to placebo (Orbit) or bisphosphonates (Cosmic).

Ultragenyx’s GTX-102, an investigational antisense oligonucleotide, is being developed in the pivotal phase III Aspire study for treating Angelman syndrome (AS) patients with a genetically confirmed diagnosis of UBE3A deletion. Top-line data is expected in the second half of 2026. Enrollment in the phase II/III Aurora study is currently ongoing to evaluate the safety and efficacy of GTX-102 for treating other AS genotypes in other patient age groups, with the first patient dosed in October 2025. This additional study aims to enable treatment for a broader range of AS patients.

During the reported quarter, Ultragenyx completed the rolling submission of a BLA for its investigational AAV8 gene therapy, DTX401, to treat glycogen storage disease type Ia. A final decision from the FDA is expected in the third quarter of 2026. The company is also evaluating UX701, an investigational AAV9 gene therapy, in a phase I/II/III Cyprus2+ study to treat Wilson disease and expects to share top-line data later in 2026.

RARE’s Zacks Rank & Stocks to Consider

Ultragenyx currently carries a Zacks Rank #3 (Hold).

Some better-ranked stocks in the biotech sector are Alkermes ALKS and Castle Biosciences CSTL, each currently sporting a Zacks Rank #1 (Strong Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.

Over the past 60 days, estimates for Alkermes’ 2026 EPS have increased from $1.54 to $1.91. ALKS shares have risen 17.8% over the past six months.

Alkermes’ earnings beat estimates in three of the trailing four quarters and missed in the remaining one, with the average earnings surprise being 4.58%.

Over the past 60 days, estimates for Castle Biosciences’ 2026 loss per share have narrowed from $1.06 to 96 cents. CSTL shares have risen 61.2% over the past six months.

Castle Biosciences’ earnings beat estimates in three of the trailing four quarters and missed in the remaining one, with the average surprise being 66.11%.

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