Ultragenyx Pharmaceutical RARE incurred a third-quarter 2024 loss of $1.40 per share, narrower than the Zacks Consensus Estimate of a loss of $1.45. The company had reported a loss of $2.23 per share in the year-ago quarter.
Ultragenyx’s total revenues amounted to $139.5 million in the reported quarter, up 42% year over year. The top line surpassed the Zacks Consensus Estimate of $134 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.
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RARE’s Q3 Results in Detail
Crysvita’s total revenues were $97.8 million, up 31% year over year, driven by increased demand for approved indications. Crysvita’s net product revenues in the third quarter of 2024 were primarily aided by the $56 million generated from sales of the drug in the Latin America region and Turkey, representing 85% growth year over year.
Mepsevii product revenues were $9.6 million in the third quarter, up 71% from the year-ago quarter. Dojolvi product revenues were $21.4 million, up 29%, driven by strong new patient demand. Evkeeza recorded sales of $10.7 million in the reported quarter.
Year to date, shares of Ultragenyx have gained 9.1% against the industry’s 3.8% decline.
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Operating expenses of $271.5 million in the quarter grew 12% year over year due to increased investments in multiple late-stage pipeline programs and costs associated with commercial product launches. Operating expenses included research and development expenses of $170.1 million (up 8%), selling, general and administrative expenses of $80.4 million (up 7%) and cost of sales of $21 (up 91%).
Cash, cash equivalents and marketable debt securities amounted to $825 million as of Sept. 30, 2024, compared with $874 million as of June 30, 2024.
RARE Maintains 2024 Guidance
Ultragenyx reiterated its previous guidance for 2024. For the full year, total revenues are expected in the range of $530-$550 million.
Crysvita revenues are expected toward the upper end of the range of $375-$400 million. Dojolvi's revenues are projected between $75 million and $80 million.
RARE’s Key Pipeline Updates
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 October, the FDA granted the Breakthrough Therapy designation to UX143 for the OI indication.
Ultragenyx is also developing GTX-102 for treating patients with Angelman syndrome (AS). In July 2024, It reached alignment with the FDA regarding the design and endpoints of the phase III AS study, named Aspire, for GTX-102. The company expects to initiate the same by the end of 2024. Ultragenyx is also developing its AAV gene therapy candidate, UX111, for the treatment of Sanfilippo syndrome type A. In October 2024, the company completed a pre-biologics license application (BLA) meeting with the FDA during which both parties aligned on the details of the impending BLA that is expected to be filed around the end of 2024.
Last quarter, Ultragenyx announced that the phase III GlucoGene study evaluating its investigational AAV8 gene therapy, DTX401, to treat glycogen storage disease type Ia patients aged eight years and older achieved its primary endpoint with statistical significance. The phase III GlucoGene study also met its key secondary endpoints. The company plans to discuss these encouraging results with regulatory authorities to support a BLA submission in mid-2025.
In early October, Ultragenyx also reported superior efficacy of UX701, an investigational AAV9 gene therapy, in the treatment of Wilson disease compared with standard therapy from the Stage 1 cohorts of its pivotal phase I/II/III Cyprus2+ study.
Ultragenyx Pharmaceutical Inc. Price and Consensus
Ultragenyx Pharmaceutical Inc. price-consensus-chart | Ultragenyx Pharmaceutical Inc. Quote
RARE’s Zacks Rank & Stocks to Consider
Ultragenyx currently carries a Zacks Rank #3 (Hold).
Some better-ranked pharma stocks are Allogene Therapeutics ALLO and Novartis NVS, 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.
Allogene Therapeutics’ loss estimates have remained constant at $1.41 per share for 2024 over the past 60 days, while that for 2025 has narrowed from $1.46 to $1.45 per share. ALLO’s shares have lost 6.9% year to date.
Allogene Therapeutics’ earnings beat estimates in three of the trailing four quarters and matched once, delivering an average surprise of 11.82%.
Novartis’ earnings estimates have risen from $7.50 to $7.56 per share for 2024 over the past 60 days, while that for 2025 has increased from $8.30 to $8.33. NVS’ shares have risen 8.3% year to date.
Novartis’ earnings beat estimates in three of the trailing four quarters and missed the same in one, delivering an average surprise of 2.22%.
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