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Ultragenyx (RARE) Q2 Loss Narrower Than Expected, Revenues Up
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Ultragenyx Pharmaceutical (RARE - Free Report) incurred a second-quarter 2024 loss of $1.52 per share, narrower than the Zacks Consensus Estimate of a loss of $1.64. The company had reported a loss of $2.25 per share in the year-ago quarter.
Ultragenyx’s total revenues amounted to $147 million in the reported quarter, up 36% year over year. The top line also beat the Zacks Consensus Estimate of $123 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 was approved in June 2020 for all forms of long-chain fatty acid oxidation disorders.
In 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 solely commercializes Evkeeza in the United States.
Year to date, shares of Ultragenyx have lost 7.7% compared with the industry’s 0.5% decline.
Image Source: Zacks Investment Research
Quarter in Detail
Crysvita’s total revenues were $114 million, up 37% year over year, driven by increased demand for approved indications. Crysvita’s net product revenues in second-quarter 2024 were primarily aided by sales of the drug in the Latin America region and Turkey, representing 140% growth year over year.
Ultragenyx sold its Crysvita rights in the European territory to Royalty Pharma in 2019. In 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 $6.1 million in the second quarter, down 27% from the year-ago quarter. Dojolvi product revenues were $19.4 million, up 17%, driven by strong new patient demand. Evkeeza recorded sales of $7.9 million in the reported quarter.
Operating expenses of $263.4 million in the quarter increased 3% year over year. Operating expenses included research and development expenses of $162 million (down 2%), selling, general and administrative expenses of $81 million (relatively flat year over year) and cost of sales of $21 (up 115%).
Cash, cash equivalents and marketable debt securities amounted to $874 million as of Jun 30, 2024, compared with $569 million as of Mar 31, 2024. The figure includes $381 million of net proceeds from the issuance of common stock and pre-funded warrants in connection with an underwritten public offering in June 2024.
2024 Guidance Updated
Ultragenyx raised its guidance for 2024 on the back of its strong financial performance in the second quarter, which was driven by increasing demand for its marketed drugs. For the full year, revenues are now expected to be in the range of $530-$550 million compared with the previous view of $500-$530 million.
Crysvita revenues are expected to be toward the upper end of 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's revenues are projected to be between $75 million and $80 million.
Ultragenyx Pharmaceutical Inc. Price and Consensus
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.
Ultragenyx is also developing GTX-102 for treating patients with Angelman syndrome (AS). Ultragenyx announced that it reached alignment with the FDA regarding the design and endpoints of the phase III AS study, named Aspire, for GTX-102 in July 2024, following the completion of a successful end-of-phase II meeting with the regulatory body. The company expects to initiate the same by the end of 2024.
RARE also discussed with the FDA its plans 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. This additional study aims to enable treatment for a broader range of AS patients.
During the reported quarter, Ultragenyx announced that the phase III GlucoGene study (NCT05139316), evaluating its investigational AAV8 gene therapy, DTX401, to treat glycogen storage disease type Ia (GSDIa) 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 further plans to discuss these encouraging results with regulatory authorities to support a marketing application in 2025.
Also during the quarter, RARE announced that it is planning to seek accelerated approval for its AAV gene therapy candidate UX111, which is being developed for the treatment of Sanfilippo syndrome type A, after a successful meeting with the FDA.
At the meeting, the company reached an agreement with the regulatory body to submit a biologics license application (BLA) seeking accelerated approval for UX111 using heparan sulfate (HS) in the cerebrospinal fluid (CSF) as a surrogate endpoint. The company will finalize the details of the above BLA and discuss the same in a pre-BLA meeting with the FDA. The BLA is likely to be filed in late 2024 or early next year.
RARE gained the FDA alignment on using CSF HS as a relevant biomarker to enable accelerated approval for UX111 in Sanfilippo syndrome.
Zacks Rank & Stocks to Consider
Ultragenyx currently carries a Zacks Rank #3 (Hold).
In the past 60 days, the Zacks Consensus Estimate for Annovis’ 2024 loss per share has remained constant at $2.46. During the same period, the consensus estimate for 2025 loss per share has narrowed from $1.95 to $1.91. Year to date, shares of ANVS have plunged 51.4%.
ANVS beat estimates in three of the trailing four quarters and missed once, delivering an average negative surprise of 1.39%.
In the past 60 days, the Zacks Consensus Estimate for Anixa Biosciences’ 2023 loss per share has narrowed from 44 cents to 43 cents. During the same time frame, the estimate for Anixa Biosciences’ 2024 loss per share has remained constant at 45 cents. Year to date, shares of ANIX have lost 12.6%.
ANIX beat estimates in three of the trailing four quarters and missed the mark once, delivering an average earnings surprise of 2.27%.
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Ultragenyx (RARE) Q2 Loss Narrower Than Expected, Revenues Up
Ultragenyx Pharmaceutical (RARE - Free Report) incurred a second-quarter 2024 loss of $1.52 per share, narrower than the Zacks Consensus Estimate of a loss of $1.64. The company had reported a loss of $2.25 per share in the year-ago quarter.
Ultragenyx’s total revenues amounted to $147 million in the reported quarter, up 36% year over year. The top line also beat the Zacks Consensus Estimate of $123 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 was approved in June 2020 for all forms of long-chain fatty acid oxidation disorders.
In 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 solely commercializes Evkeeza in the United States.
Year to date, shares of Ultragenyx have lost 7.7% compared with the industry’s 0.5% decline.
Image Source: Zacks Investment Research
Quarter in Detail
Crysvita’s total revenues were $114 million, up 37% year over year, driven by increased demand for approved indications. Crysvita’s net product revenues in second-quarter 2024 were primarily aided by sales of the drug in the Latin America region and Turkey, representing 140% growth year over year.
Ultragenyx sold its Crysvita rights in the European territory to Royalty Pharma in 2019. In 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 $6.1 million in the second quarter, down 27% from the year-ago quarter. Dojolvi product revenues were $19.4 million, up 17%, driven by strong new patient demand. Evkeeza recorded sales of $7.9 million in the reported quarter.
Operating expenses of $263.4 million in the quarter increased 3% year over year. Operating expenses included research and development expenses of $162 million (down 2%), selling, general and administrative expenses of $81 million (relatively flat year over year) and cost of sales of $21 (up 115%).
Cash, cash equivalents and marketable debt securities amounted to $874 million as of Jun 30, 2024, compared with $569 million as of Mar 31, 2024. The figure includes $381 million of net proceeds from the issuance of common stock and pre-funded warrants in connection with an underwritten public offering in June 2024.
2024 Guidance Updated
Ultragenyx raised its guidance for 2024 on the back of its strong financial performance in the second quarter, which was driven by increasing demand for its marketed drugs. For the full year, revenues are now expected to be in the range of $530-$550 million compared with the previous view of $500-$530 million.
Crysvita revenues are expected to be toward the upper end of 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's revenues are projected to be between $75 million and $80 million.
Ultragenyx Pharmaceutical Inc. Price and Consensus
Ultragenyx Pharmaceutical Inc. price-consensus-chart | 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.
Ultragenyx is also developing GTX-102 for treating patients with Angelman syndrome (AS). Ultragenyx announced that it reached alignment with the FDA regarding the design and endpoints of the phase III AS study, named Aspire, for GTX-102 in July 2024, following the completion of a successful end-of-phase II meeting with the regulatory body. The company expects to initiate the same by the end of 2024.
RARE also discussed with the FDA its plans 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. This additional study aims to enable treatment for a broader range of AS patients.
During the reported quarter, Ultragenyx announced that the phase III GlucoGene study (NCT05139316), evaluating its investigational AAV8 gene therapy, DTX401, to treat glycogen storage disease type Ia (GSDIa) 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 further plans to discuss these encouraging results with regulatory authorities to support a marketing application in 2025.
Also during the quarter, RARE announced that it is planning to seek accelerated approval for its AAV gene therapy candidate UX111, which is being developed for the treatment of Sanfilippo syndrome type A, after a successful meeting with the FDA.
At the meeting, the company reached an agreement with the regulatory body to submit a biologics license application (BLA) seeking accelerated approval for UX111 using heparan sulfate (HS) in the cerebrospinal fluid (CSF) as a surrogate endpoint. The company will finalize the details of the above BLA and discuss the same in a pre-BLA meeting with the FDA. The BLA is likely to be filed in late 2024 or early next year.
RARE gained the FDA alignment on using CSF HS as a relevant biomarker to enable accelerated approval for UX111 in Sanfilippo syndrome.
Zacks Rank & Stocks to Consider
Ultragenyx currently carries a Zacks Rank #3 (Hold).
Some better-ranked stocks in the biotech sector are Annovis Bio (ANVS - Free Report) and Anixa Biosciences (ANIX - Free Report) , both carrying a Zacks Rank #2 (Buy) at present. You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.
In the past 60 days, the Zacks Consensus Estimate for Annovis’ 2024 loss per share has remained constant at $2.46. During the same period, the consensus estimate for 2025 loss per share has narrowed from $1.95 to $1.91. Year to date, shares of ANVS have plunged 51.4%.
ANVS beat estimates in three of the trailing four quarters and missed once, delivering an average negative surprise of 1.39%.
In the past 60 days, the Zacks Consensus Estimate for Anixa Biosciences’ 2023 loss per share has narrowed from 44 cents to 43 cents. During the same time frame, the estimate for Anixa Biosciences’ 2024 loss per share has remained constant at 45 cents. Year to date, shares of ANIX have lost 12.6%.
ANIX beat estimates in three of the trailing four quarters and missed the mark once, delivering an average earnings surprise of 2.27%.