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Ultragenyx (RARE) Q1 Loss Wider Than Expected, Revenues Rise Y/Y
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Ultragenyx Pharmaceutical Inc. (RARE - Free Report) incurred a loss of $2.03 per share in the first quarter of 2024, wider than the Zacks Consensus Estimate of a loss of $1.72 per share. The company had reported a loss of $2.33 per share in the year-ago quarter.
Ultragenyx’s total revenues amounted to $109 million in the reported quarter, up 8% year over year. The top line missed the Zacks Consensus Estimate of $114 million. The year-over-year increase failed to meet investor expectations.
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 was approved in June 2020 for all forms of long-chain fatty acid oxidation disorders.
In January 2022, Ultragenyx announced a license and collaboration agreement with Regeneron Pharmaceuticals (REGN - Free Report) . Per the deal, RARE has obtained the rights to develop, commercialize and distribute Evkeeza (evinacumab) 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 will continue to solely commercialize Evkeeza in the United States.
The company’s stock lost 5% in the after-market hours on May 2, as the investors were disappointed by the dismal first-quarter 2024 results. Year to date, shares of RARE have lost 9.7% compared with the industry’s 8.8% decline.
Image Source: Zacks Investment Research
Quarter in Detail
Crysvita’s total revenues were $83 million, up 9% year over year, driven by increased demand for approved indications. Crysvita’s net product revenues in first-quarter 2024 were primarily driven by sales of the drug in the Latin America region and Turkey, which were $36 million, representing 71% year-over-year growth. Sales of the drug in North America and the EU generated $40 and $6 million, respectively, in revenues.
Ultragenyx sold its Crysvita rights in the European territory to Royalty Pharma in December 2019. In April 2023, RARE entered into a licensing agreement with Kyowa Kirin, transitioning commercialization responsibilities for Crysvita in North America to its partner.
Mepsevii product revenues were $6.6 million in the fourth quarter, down 22% from the year-ago quarter. Dojolvi product revenues were $16.4 million, up 14% from the year-ago quarter, driven by strong new patient demand. Evkeeza recorded sales of $3 million in the reported quarter.
Operating expenses of $274 million in the reported quarter increased 8% year over year. Operating expenses for the reported quarter include research and development expenses of $178 million (up 8%), selling, general and administrative expenses of $78 million (up 2%) and cost of sales of $18 (up 43%)
Cash, cash equivalents and marketable debt securities amounted to $569 million as of Mar 31, 2024, compared with $777.1 million as of Dec 31, 2023.
2024 Guidance Reaffirmed
Ultragenyx reiterated its guidance for 2024. Revenues are expected between $500 million and $530 million. Crysvita revenues are expected in the range of $375-$400 million (including all regions where Ultragenyx will recognize revenues, comprising product sales in Latin America and Turkey, royalties in Europe, which have been ongoing, and the royalties in North America, which began in April 2023). Dojolvi revenues are projected between $75 million and $80 million.
Ultragenyx Pharmaceutical Inc. Price and EPS Surprise
Ultragenyx and its partner, Mereo BioPharma, are jointly developing UX143 (setrusumab) monoclonal antibody forpediatric and young adult patients with osteogenesis imperfecta in two late-stage studies, Orbit and Cosmic. During the reported quarter, enrollment was completed in the phase III portion of the Orbit study.
The phase III Cosmic study is evaluating the effect of setrusumab compared with intravenous bisphosphonate therapy on the annualized total fracture rate in patients aged 2 to <7 years. Enrollment in this study has also been completed.
Ultragenyx is also developing GTX-102 for treating patients with Angelman syndrome (AS) in an early to mid-stage study. Last month, RARE reported positive interim data from the expansion cohorts of the early to mid-stage study of GTX-102 gene therapy for the treatment of Angelman syndrome.
Patients treated with a set dose and regimen of GTX-102 in Expansion Cohorts A & B showed improvements across multiple functional domains including cognitive function, behavior, hyperactivity and noncompliance, sleep, receptive communication and gross motor function, at day 170.
However, three patients in the phase I/II study of GTX-102 for AS experienced serious adverse events (mild to moderate) of lower extremity weakness. The adverse events were assessed as related to the study treatment.
Ultragenyx is planning to discuss these results from the phase I/II study of GTX-102 with the FDA in mid-2024 and with other regulatory authorities in the second half of 2024 to enable the initiation of a phase III pivotal study of the candidate for Angelman syndrome around 2024-end.
Zacks Rank & Stocks to Consider
Ultragenyx currently carries a Zacks Rank #3 (Hold).
In the past 30 days, the Zacks Consensus Estimate for Ligand’s 2024 earnings per share has remained constant at $4.56. During the same time frame, the estimate for Ligand’s 2025 earnings per share has remained constant at $5.27. Year to date, shares of LGND have gained 1.3%.
Ligand beat estimates in each of the trailing four quarters, delivering an average surprise of 84.81%.
In the past 30 days, estimates for ANI Pharmaceuticals’ 2024 earnings per share have risen from $4.43 to $4.44. Meanwhile, during the same period, the estimate for ANI Pharmaceuticals’ 2025 earnings per share has remained constant at $5.04. Year to date, shares of ANIP have risen 21.7%.
ANI Pharmaceuticals beat estimates in each of the last four quarters, delivering an average surprise of 109.06%.
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Ultragenyx (RARE) Q1 Loss Wider Than Expected, Revenues Rise Y/Y
Ultragenyx Pharmaceutical Inc. (RARE - Free Report) incurred a loss of $2.03 per share in the first quarter of 2024, wider than the Zacks Consensus Estimate of a loss of $1.72 per share. The company had reported a loss of $2.33 per share in the year-ago quarter.
Ultragenyx’s total revenues amounted to $109 million in the reported quarter, up 8% year over year. The top line missed the Zacks Consensus Estimate of $114 million. The year-over-year increase failed to meet investor expectations.
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 was approved in June 2020 for all forms of long-chain fatty acid oxidation disorders.
In January 2022, Ultragenyx announced a license and collaboration agreement with Regeneron Pharmaceuticals (REGN - Free Report) . Per the deal, RARE has obtained the rights to develop, commercialize and distribute Evkeeza (evinacumab) 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 will continue to solely commercialize Evkeeza in the United States.
The company’s stock lost 5% in the after-market hours on May 2, as the investors were disappointed by the dismal first-quarter 2024 results. Year to date, shares of RARE have lost 9.7% compared with the industry’s 8.8% decline.
Image Source: Zacks Investment Research
Quarter in Detail
Crysvita’s total revenues were $83 million, up 9% year over year, driven by increased demand for approved indications. Crysvita’s net product revenues in first-quarter 2024 were primarily driven by sales of the drug in the Latin America region and Turkey, which were $36 million, representing 71% year-over-year growth. Sales of the drug in North America and the EU generated $40 and $6 million, respectively, in revenues.
Ultragenyx sold its Crysvita rights in the European territory to Royalty Pharma in December 2019. In April 2023, RARE entered into a licensing agreement with Kyowa Kirin, transitioning commercialization responsibilities for Crysvita in North America to its partner.
Mepsevii product revenues were $6.6 million in the fourth quarter, down 22% from the year-ago quarter. Dojolvi product revenues were $16.4 million, up 14% from the year-ago quarter, driven by strong new patient demand. Evkeeza recorded sales of $3 million in the reported quarter.
Operating expenses of $274 million in the reported quarter increased 8% year over year. Operating expenses for the reported quarter include research and development expenses of $178 million (up 8%), selling, general and administrative expenses of $78 million (up 2%) and cost of sales of $18 (up 43%)
Cash, cash equivalents and marketable debt securities amounted to $569 million as of Mar 31, 2024, compared with $777.1 million as of Dec 31, 2023.
2024 Guidance Reaffirmed
Ultragenyx reiterated its guidance for 2024. Revenues are expected between $500 million and $530 million. Crysvita revenues are expected in the range of $375-$400 million (including all regions where Ultragenyx will recognize revenues, comprising product sales in Latin America and Turkey, royalties in Europe, which have been ongoing, and the royalties in North America, which began in April 2023). Dojolvi revenues are projected between $75 million and $80 million.
Ultragenyx Pharmaceutical Inc. Price and EPS Surprise
Ultragenyx Pharmaceutical Inc. price-eps-surprise | Ultragenyx Pharmaceutical Inc. Quote
Key Pipeline Updates
Ultragenyx and its partner, Mereo BioPharma, are jointly developing UX143 (setrusumab) monoclonal antibody forpediatric and young adult patients with osteogenesis imperfecta in two late-stage studies, Orbit and Cosmic. During the reported quarter, enrollment was completed in the phase III portion of the Orbit study.
The phase III Cosmic study is evaluating the effect of setrusumab compared with intravenous bisphosphonate therapy on the annualized total fracture rate in patients aged 2 to <7 years. Enrollment in this study has also been completed.
Ultragenyx is also developing GTX-102 for treating patients with Angelman syndrome (AS) in an early to mid-stage study. Last month, RARE reported positive interim data from the expansion cohorts of the early to mid-stage study of GTX-102 gene therapy for the treatment of Angelman syndrome.
Patients treated with a set dose and regimen of GTX-102 in Expansion Cohorts A & B showed improvements across multiple functional domains including cognitive function, behavior, hyperactivity and noncompliance, sleep, receptive communication and gross motor function, at day 170.
However, three patients in the phase I/II study of GTX-102 for AS experienced serious adverse events (mild to moderate) of lower extremity weakness. The adverse events were assessed as related to the study treatment.
Ultragenyx is planning to discuss these results from the phase I/II study of GTX-102 with the FDA in mid-2024 and with other regulatory authorities in the second half of 2024 to enable the initiation of a phase III pivotal study of the candidate for Angelman syndrome around 2024-end.
Zacks Rank & Stocks to Consider
Ultragenyx currently carries a Zacks Rank #3 (Hold).
Some better-ranked stocks in the same industry are Ligand Pharmaceuticals (LGND - Free Report) and ANI Pharmaceuticals (ANIP - Free Report) , each sporting a Zacks Rank #1 (Strong Buy) at present. You can see the complete list of today’s Zacks #1 Rank stocks here.
In the past 30 days, the Zacks Consensus Estimate for Ligand’s 2024 earnings per share has remained constant at $4.56. During the same time frame, the estimate for Ligand’s 2025 earnings per share has remained constant at $5.27. Year to date, shares of LGND have gained 1.3%.
Ligand beat estimates in each of the trailing four quarters, delivering an average surprise of 84.81%.
In the past 30 days, estimates for ANI Pharmaceuticals’ 2024 earnings per share have risen from $4.43 to $4.44. Meanwhile, during the same period, the estimate for ANI Pharmaceuticals’ 2025 earnings per share has remained constant at $5.04. Year to date, shares of ANIP have risen 21.7%.
ANI Pharmaceuticals beat estimates in each of the last four quarters, delivering an average surprise of 109.06%.