Ultragenyx Q3 Earnings and Sales Miss Estimates, Increase Y/Y

Ultragenyx Pharmaceutical RARE reported third-quarter 2025 loss of $1.81 per share, wider than the Zacks Consensus Estimate of a loss of $1.23. The company had incurred a loss of $1.40 per share in the year-ago quarter.

Ultragenyx’s total revenues amounted to $159.9 million in the reported quarter, up 15% year over year, on the back of higher product sales. The top line, however, missed the Zacks Consensus Estimate of $168 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 Q3 Results in Detail

Crysvita’s total revenues were $111.9 million, up 14% year over year, driven by increased demand for approved indications. Crysvita’s net product revenues in the third quarter of 2025 included $57 million from North America, $47 million from Latin America and Turkey, and $8 million from Europe.

Mepsevii product revenues declined 27% year over year to $7 million in the reported quarter. Dojolvi product revenues were $24.3 million, up 14%, driven by new patient demand. Evkeeza recorded sales of $16.7 million in the third quarter, up 57% as Ultragenyx continues to launch the drug in its territories outside of the United States.

Year to date, shares of Ultragenyx have lost 22.9% against the industry’s 10.2% growth.

Operating expenses of $330.8 million in the quarter grew 22% 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 expenses of $216.2 million (up 27%), selling, general and administrative expenses of $86.6 million (up 8%), and cost of sales of $28 million (up 33%).

Cash, cash equivalents and marketable debt securities amounted to $447 million as of Sept. 30, 2025, compared with $539 million as of June 30, 2025.

RARE Reaffirms 2025 Financial Guidance

Ultragenyx expects total revenues in 2025 between $640 million and $670 million, representing growth of approximately 14-20% compared to 2024. Crysvita revenues are expected in the range of $460-$480 million (up 12-17% year over year), while Dojolvi revenues are expected between $90 million and $100 million (up 2-14% year over year) in 2025.

The company will maintain its focus on expense management while strategically investing in upcoming commercial launches and advancing multiple phase III programs, which are expected to reduce net cash used in operations in 2025 compared with 2024, reaffirming its path to full-year GAAP profitability in 2027.

RARE’s Key Pipeline Updates

Earlier this year, 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. In the CRL, the FDA requested more information and enhancements concerning certain chemistry, manufacturing and controls elements and findings from recent manufacturing facility inspections. Per Ultragenyx, the issues raised are facility- and process-related, not tied to product quality, and can be addressed quickly. It plans to resubmit the BLA early in 2026, triggering a review period of up to six months per FDA regulations.

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 July, the companies reported that the Orbit and Cosmic studies are on track for a final analysis around the end of 2025.

Ultragenyx Pharmaceutical Inc. Price, Consensus and EPS Surprise

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

The FDA recently granted the Breakthrough Therapy designation to Ultragenyx’s GTX-102, its investigational antisense oligonucleotide, for treating Angelman syndrome (AS). Last year, RARE initiated dosing in the pivotal phase III Aspire study evaluating the efficacy and safety of GTX-102 for AS. Ultragenyx completed enrollment in the Aspire study during the reported quarter, with data expected in the second half of 2026. The company has also begun dosing patients in an open-label clinical study, named Aurora, to evaluate the safety and efficacy of GTX-102 for treating other AS genotypes in other patient age groups. This additional study aims to enable treatment for a broader range of AS patients.

Ultragenyx has begun the rolling submission of a BLA for its investigational AAV8 gene therapy, DTX401, in August 2025 to treat glycogen storage disease type Ia and is expected to be completed in the fourth quarter of 2025. 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 in the first half of 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 Beam Therapeutics BEAM and Amicus Therapeutics FOLD. While FOLD currently sports a Zacks Rank #1 (Strong Buy), BEAM carries a Zacks Rank #2 (Buy) at present. You can see the complete list of today’s Zacks #1 Rank stocks here.

In the past 60 days, estimates for Amicus Therapeutics’ earnings per share have remained constant at 31 cents for 2025. During the same time, earnings per share estimates for 2026 have increased from 69 cents to 70 cents. Year to date, shares of FOLD have lost 4.5%.

Amicus Therapeutics’ earnings beat estimates in one of the trailing four quarters, missing the mark thrice, with the average negative surprise being 24.38%.

In the past 60 days, estimates for Beam Therapeutics' loss per share have narrowed from $4.36 to $4.23 for 2025. During the same time, loss per share estimates for 2026 have narrowed from $4.41 to $4.21. Year to date, shares of BEAM have lost 8.3%.

Beam Therapeutics’ earnings beat estimates in two of the trailing four quarters while missing the same on the remaining two occasions, the average negative surprise being 2.62%.

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