Ultragenyx Pharmaceutical RARE reported fourth-quarter 2024 loss of $1.39 per share, wider than the Zacks Consensus Estimate of a loss of $1.24. The company had incurred a loss of $1.52 per share in the year-ago quarter.

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Ultragenyx’s total revenues amounted to $164.9 million in the reported quarter, up 29% year over year. The top line surpassed the Zacks Consensus Estimate of $163 million on the back of higher product revenues.

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 $115.4 million, up 23% year over year, driven by increased demand for approved indications. Crysvita’s net product revenues in the fourth quarter of 2024 included $22 million generated from sales of the drug in the Latin America region and Turkey, representing 22% growth year over year.

Mepsevii product revenues were relatively flat year over year at $8 million in the reported quarter. Dojolvi product revenues were $31.1 million, up 34%, driven by strong new patient demand. Evkeeza recorded sales of $10.4 million in the fourth quarter.

In the past three months, shares of Ultragenyx have lost 2.7% against the industry’s 7.7% growth.

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Operating expenses of $287.2 million in the quarter grew 15% 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 $187.8 million (up 17%), selling, general and administrative expenses of $82.5 million (up 7%) and cost of sales of $16.9 million (up 40%).

Cash, cash equivalents and marketable debt securities amounted to $745 million as of Dec. 31, 2024, compared with $825 million as of Sept. 30, 2024.

RARE’s 2024 Results

Total revenues in 2024 came in at $560.2 million, up 29% from $434.2 million recorded in 2023. The figure beat the Zacks Consensus Estimate of $558.6 million.

The net loss per share in 2024 came in at $6.29, which is narrower than a loss of $8.25 reported in 2023. The reported figure was, however, wider than the Zacks Consensus Estimate of a loss of $6.11 per share. Net cash used in operations in 2024 was $414 million.

RARE’s 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 is expected to reduce net cash used in operations in 2025 compared with 2024.

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

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 also 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 approval for its UX111 AAV gene therapy to treat MPS IIIA patients, under the accelerated pathway. A decision is expected in the second half of 2025.

RARE’s Zacks Rank & Stocks to Consider

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

Some better-ranked stocks from the sector are BioMarin Pharmaceutical BMRN and Alnylam Pharmaceuticals ALNY, each carrying a Zacks Rank #2 (Buy) at present. You can see the complete list of today’s Zacks #1 (Strong Buy) Rank stocks here.

In the past 30 days, estimates for BioMarin Pharmaceutical’s 2024 earnings per share have increased from $3.28 to $3.29. Estimates for 2025 earnings per share have decreased from $4.02 to $4.01 during the same timeframe. In the past three months, BioMarin Pharmaceutical shares have gained 1.6%.

BMRN’s earnings beat estimates in each of the trailing four quarters, delivering an average surprise of 28.7%.

In the past 30 days, estimates for Alnylam Pharmaceuticals’ 2024 loss per share have remained constant at 39 cents. The estimate for 2025 earnings per share is currently pegged at 41 cents. In the past three months, shares of Alnylam Pharmaceuticals have gained 12.5%.

ALNY’s earnings beat estimates in three of the trailing four quarters and matched once, delivering an average surprise of 65.67%.

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