Ultragenyx's Q2 Earnings Beat Estimates, Revenues Jump Y/Y

Management stated that second-quarter 2026 revenues were the highest quarterly revenues ever reported by the company.

Ultragenyx 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.

Year to date, shares of Ultragenyx have gained 12.2% compared with the industry’s 1.6% rise.

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RARE’s Q2 Results in Detail

Crysvita’s total revenues were $156 million, up 28.9% year over year. Management noted that Crysvita sales were consistent with expected seasonality in the United States and Canada and ordering patterns in Latin America. Crysvita’s net product revenues in the second quarter of 2026 included $94 million from North America, $54 million from Latin America and Turkey, and $8 million from Europe.

Mepsevii product revenues increased 11.1% year over year to $10 million in the reported quarter. Dojolvi product revenues were $27 million, up 17.4%, driven by strong demand. Evkeeza recorded sales of $21 million in the second quarter, up 50%, driven by increased demand from new country launches and early access.

Operating expenses of $289 million in the quarter rose 5.1% 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 $167 million (up 1.2%), selling, general and administrative (SG&A) expenses of $88 million (up 1.1%) and cost of sales of $34 million (up 47.8%).

Cash, cash equivalents and marketable securities amounted to $436 million as of June 30, 2026, compared with $534 million as of March 31, 2026.

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.

Crysvita revenues in 2026 are expected to be in the range of $500-$520 million, reflecting growing underlying global demand. Meanwhile, 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.

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 regulatory agency is expected on Aug. 23, 2026.

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 in the fourth quarter of 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 are expected in September or October 2026. Meanwhile, 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. This additional study aims to enable treatment for a broader range of AS patients. The study is expected to complete enrollment in the second half of 2026.

RARE’s Zacks Rank & Other Stocks to Consider

Ultragenyx currently carries a Zacks Rank #2 (Buy).

Some other top-ranked stocks in the biotech sector are Repligen RGEN and Liquidia Corporation LQDA, 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 Repligen’s 2026 earnings per share have risen from $1.99 to $2.06, while estimates for 2027 have increased from $2.57 to $2.62 during the same time. RGEN shares have declined 8.6% year to date.

Repligen’s earnings beat estimates in each of the trailing four quarters, with the average surprise being 16.80%.

Over the past 60 days, estimates for Liquidia’s 2026 earnings per share have risen from $2.97 to $3.02, while estimates for 2027 have increased from $4.81 to $5.31 during the same time. LQDA shares have surged 156.3% year to date.

Liquidia’s earnings beat estimates in three of the trailing four quarters, while missing the same on the remaining occasion, with the average surprise being 54.40%.

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