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RARE Q1 Loss Wider Than Expected, Sales Down Y/Y on Seasonal Effect
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Key Takeaways
Ultragenyx Q1 revenues fell 2% as Crysvita sales declined due to seasonal and ordering effects.
RARE saw Evkeeza sales jump 64% on new country launches and early access demand.
Ultragenyx expects FDA decisions on UX111 and DTX401 later in 2026 amid other pipeline progress.
Ultragenyx Pharmaceutical (RARE - Free Report) reported first-quarter 2026 loss of $1.84 per share, wider than the Zacks Consensus Estimate of a loss of $1.55. The company had incurred a loss of $1.57 per share in the year-ago quarter.
Ultragenyx’s total revenues amounted to $136 million in the reported quarter, which declined 2% year over year due to lower product sales. The top line missed the Zacks Consensus Estimate of $161 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 - Free Report) 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 $93 million, down 10% year over year, due to expected seasonality in the U.S. and Canada and ordering patterns in Brazil. Crysvita’s net product revenues in the first quarter of 2026 included $39 million from North America, $46 million from Latin America and Turkey, and $8 million from Europe.
Mepsevii product revenues decreased 13% year over year to $7 million in the reported quarter. Dojolvi product revenues were $18 million, up 6%, driven by new patient demand. Evkeeza recorded sales of $18 million in the first quarter, up 64%, driven by increased demand from new country launches and early access.
Year to date, shares of Ultragenyx have gained 8.7% against the industry’s 2.4% decline.
Image Source: Zacks Investment Research
Operating expenses of $305 million in the quarter rose 8% 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 $187 million (up 13%), selling, general and administrative (SG&A) expenses of $88 million (up 1%) and cost of sales of $30 million (up 3%).
Cash, cash equivalents and marketable securities amounted to $534 million as of March 31, 2026, compared with $737 million as of Dec. 31, 2025.
RARE Reiterates 2026 Financial Guidance
Ultragenyx continues to expect 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.
RARE’s Key Pipeline Updates
In April 2026, the FDA accepted the resubmitted biologics license application (BLA) seeking accelerated approval of UX111 for the treatment of MPS IIIA. The application included extensive long-term data with follow-up of up to eight years. The data showed sustained clinical benefits compared with the decline seen in natural history studies, along with durable treatment effects across multiple clinical measures and biomarkers, while maintaining an acceptable safety profile. A final decision from the regulatory body is expected on Sept. 19, 2026.
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.
The FDA has also accepted for review Ultragenyx’s BLA for its investigational AAV8 gene therapy, DTX401, to treat glycogen storage disease type Ia. A final decision from the FDA is expected on Aug. 23, 2026. The regulatory body also recently informed RARE that an Advisory Committee meeting is not anticipated at this time. Ultragenyx 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.
Ultragenyx Pharmaceutical Inc. Price, Consensus and EPS Surprise
Over the past 60 days, estimates for Catalyst Pharmaceuticals’ 2026 EPS have declined from $2.82 to $2.79. CPRX shares have gained 32.3% year to date.
Catalyst Pharmaceuticals’ earnings beat estimates in each of the trailing four quarters, with the average surprise being 35.19%.
Over the past 60 days, estimates for Inovio Pharmaceuticals’ 2026 loss per share have narrowed from $1.26 to $1.06. INO shares have plunged 34.5% year to date.
Inovio Pharmaceuticals’ earnings beat estimates in each of the trailing four quarters, with the average surprise being 57.94%.
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RARE Q1 Loss Wider Than Expected, Sales Down Y/Y on Seasonal Effect
Key Takeaways
Ultragenyx Pharmaceutical (RARE - Free Report) reported first-quarter 2026 loss of $1.84 per share, wider than the Zacks Consensus Estimate of a loss of $1.55. The company had incurred a loss of $1.57 per share in the year-ago quarter.
Ultragenyx’s total revenues amounted to $136 million in the reported quarter, which declined 2% year over year due to lower product sales. The top line missed the Zacks Consensus Estimate of $161 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 - Free Report) 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 $93 million, down 10% year over year, due to expected seasonality in the U.S. and Canada and ordering patterns in Brazil. Crysvita’s net product revenues in the first quarter of 2026 included $39 million from North America, $46 million from Latin America and Turkey, and $8 million from Europe.
Mepsevii product revenues decreased 13% year over year to $7 million in the reported quarter. Dojolvi product revenues were $18 million, up 6%, driven by new patient demand. Evkeeza recorded sales of $18 million in the first quarter, up 64%, driven by increased demand from new country launches and early access.
Year to date, shares of Ultragenyx have gained 8.7% against the industry’s 2.4% decline.
Image Source: Zacks Investment Research
Operating expenses of $305 million in the quarter rose 8% 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 $187 million (up 13%), selling, general and administrative (SG&A) expenses of $88 million (up 1%) and cost of sales of $30 million (up 3%).
Cash, cash equivalents and marketable securities amounted to $534 million as of March 31, 2026, compared with $737 million as of Dec. 31, 2025.
RARE Reiterates 2026 Financial Guidance
Ultragenyx continues to expect 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.
RARE’s Key Pipeline Updates
In April 2026, the FDA accepted the resubmitted biologics license application (BLA) seeking accelerated approval of UX111 for the treatment of MPS IIIA. The application included extensive long-term data with follow-up of up to eight years. The data showed sustained clinical benefits compared with the decline seen in natural history studies, along with durable treatment effects across multiple clinical measures and biomarkers, while maintaining an acceptable safety profile. A final decision from the regulatory body is expected on Sept. 19, 2026.
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.
The FDA has also accepted for review Ultragenyx’s BLA for its investigational AAV8 gene therapy, DTX401, to treat glycogen storage disease type Ia. A final decision from the FDA is expected on Aug. 23, 2026. The regulatory body also recently informed RARE that an Advisory Committee meeting is not anticipated at this time. Ultragenyx 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.
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 Catalyst Pharmaceuticals (CPRX - Free Report) and Inovio Pharmaceuticals (INO - Free Report) , 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.
Over the past 60 days, estimates for Catalyst Pharmaceuticals’ 2026 EPS have declined from $2.82 to $2.79. CPRX shares have gained 32.3% year to date.
Catalyst Pharmaceuticals’ earnings beat estimates in each of the trailing four quarters, with the average surprise being 35.19%.
Over the past 60 days, estimates for Inovio Pharmaceuticals’ 2026 loss per share have narrowed from $1.26 to $1.06. INO shares have plunged 34.5% year to date.
Inovio Pharmaceuticals’ earnings beat estimates in each of the trailing four quarters, with the average surprise being 57.94%.