Ultragenyx Pharmaceutical RARE reported first-quarter 2025 loss of $1.57 per share, wider than the Zacks Consensus Estimate of a loss of $1.54. The company had incurred a loss of $2.03 per share in the year-ago quarter.
Ultragenyx’s total revenues amounted to $139.3 million in the reported quarter, up 28% year over year, on the back of higher product sales. The top line, however, missed the Zacks Consensus Estimate of $142 million. (See the Zacks Earnings Calendar to stay ahead of market-making news.)
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 Q1 Results in Detail
Crysvita’s total revenues were $102.9 million, up 25% year over year, driven by increased demand for approved indications. Crysvita’s net product revenues in the first quarter of 2024 included $55 million generated from sales of the drug in the Latin America region and Turkey, which represents 52% growth year over year.
Mepsevii product revenues increased 27% year over year to $8.4 million in the reported quarter. Dojolvi product revenues were $17 million, up 4%, driven by new patient demand. Evkeeza recorded sales of $11 million in the first quarter, up significantly as Ultragenyx continues to launch the drug in its territories outside of the United States.
Year to date, shares of RARE have lost 16.7% compared with the industry’s 8% decline.
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Operating expenses of $282.2 million in the quarter grew 3% 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 $165.8 million (down 7%), selling, general and administrative expenses of $87.8 million (up 12%) and cost of sales of $28.7 million (up 63%).
Cash, cash equivalents and marketable debt securities amounted to $563 million as of March 31, 2025, compared with $745 million as of Dec. 31, 2024.
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.
RARE’s Key Pipeline Updates
RARE 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. An update from the studies is expected in mid-2025.
The company is evaluating UX701, an investigational AAV9 gene therapy, in a phase I/II/III Cyprus2+ study to treat Wilson disease. Ultragenyx is also planning to submit a biologics license application (BLA) for its investigational AAV8 gene therapy, DTX401, to treat glycogen storage disease type Ia in mid-2025.
In December 2024, Ultragenyx initiated dosing in the pivotal phase III Aspire study evaluating the efficacy and safety of GTX-102, its investigational antisense oligonucleotide, for Angelman syndrome (AS). The company is also on track to initiate 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 in 2025. This additional study aims to enable treatment for a broader range of AS patients.
Ultragenyx is also developing its AAV gene therapy candidate, UX111, for the treatment of Sanfilippo syndrome type A. In December 2024, the company submitted a BLA to the FDA seeking accelerated approval for its UX111 AAV gene therapy to treat MPS IIIA patients. In February 2025, the FDA accepted the UX111 regulatory filing for review under the Priority Review pathway. A decision is expected on Aug. 18, 2025, that would be followed by a potential launch in the second half of 2025.
Ultragenyx Pharmaceutical Inc. Price, Consensus and EPS Surprise
Ultragenyx Pharmaceutical Inc. Price, Consensus and EPS Surprise
Ultragenyx Pharmaceutical Inc. price-consensus-eps-surprise-chart | Ultragenyx Pharmaceutical Inc. Quote
RARE’s Zacks Rank & Stocks to Consider
Ultragenyx currently carries a Zacks Rank #3 (Hold).
Some better-ranked stocks in the biotech sector are Bayer BAYRY and ADMA Biologics Inc. ADMA, each carrying a Zacks Rank #2 (Buy) at present. You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.
In the past 60 days, estimates for Bayer’s earnings per share have increased from $1.17 to $1.23 for 2025. During the same time, earnings per share have increased from $1.27 to $1.31 for 2026. Year to date, shares of Bayer have gained 39.3%.
BAYRY’s earnings matched estimates in two of the trailing three quarters while missing the same on the remaining occasion, the average negative surprise being 19.61%.
In the past 60 days, estimates for ADMA Biologics’ earnings per share have increased from 70 cents to 71 cents for 2025. During the same time, earnings per share estimates for 2026 have remained constant at 93 cents. Year to date, shares of ADMA have rallied 34.8%.
ADMA’s earnings beat estimates in three of the trailing four quarters while missing the same on the remaining occasion, the average surprise being 32.80%.
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