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RARE's Wilson Disease Candidate Betters Standard Therapy in Study
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Ultragenyx Pharmaceutical (RARE - Free Report) reported positive updates from the Stage 1 cohorts of its phase I/II/III Cyprus2+ study evaluating UX701, an investigational AAV9 gene therapy, to treat Wilson disease. Stage 1 of the study is assessing the safety and efficacy of UX701 and a dose will be selected for further evaluation in Stage 2, which is the pivotal portion of the study.
Wilson disease is a genetic disorder where the body is unable to properly eliminate excess copper, leading to its buildup in organs like the liver, brain and eyes. This can cause liver damage, neurological issues and other serious symptoms. Per Ultragenyx, more than 50,000 people suffer from this indication in commercially accessible geographies.
The Stage 1 portion of the Cyprus2+ study, which enrolled 15 patients into three sequential dosing cohorts and followed for at least 24 weeks, demonstrated meaningful clinical activity and improvements in copper metabolism. Management believes that UX701 has the potential to become a novel therapy for Wilson disease patients.
Efficacy Data From RARE’s Study of Wilson Disease Candidate
According to the data, six out of the 15 enrolled patients fully discontinued standard-of-care treatments involving chelators and/or zinc therapy, with positive responses across all three dosage groups. Furthermore, a seventh patient had started to reduce the dependency on standard-of-care treatment as of the data cut-off date in August.
Interestingly, non-ceruloplasmin-bound copper levels returned to normal in patients who stopped standard therapy. Some also showed increased ceruloplasmin-copper activity, indicating better ATP7b function.
Year to date, shares of RARE have gained 12.2% against the industry’s 1.6% decline.
Image Source: Zacks Investment Research
Ultragenyx reported that the investigational gene therapy has been overall well-tolerated in the Stage 1 cohorts of the Cyprus2+ study. As of the data cut-off date, no unexpected related treatment-emergent adverse events and no significant immunologic safety events have been observed.
Based on the encouraging update from the Wilson Disease study, Ultragenyx plans to submit a protocol amendment to add a new cohort in Stage 1, where patients will receive a slightly higher dose of UX701, along with an improved immunomodulation regimen to make the gene therapy more effective. The goal is for most patients to stop standard treatment before moving to the next phase of the study (Stage 2), which will involve a randomized placebo-controlled study.
Other Key Clinical Programs in RARE’s Pipeline
Apart from UX701, Ultragenyx has several other interesting gene therapy candidates in its pipeline. The company, in partnership with Mereo BioPharma, is developing UX143 (setrusumab) monoclonal antibody forpediatric and young adult patients with osteogenesis imperfecta in two late-stage studies, Orbit and Cosmic.
In May 2024, Ultragenyx announced that the phase III GlucoGene study (NCT05139316), 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 GlucoGene study also met its key secondary endpoints. The next step for the company involves discussing these results with regulatory authorities to support a marketing application in 2025.
Ultragenyx is also planning to seek accelerated approval for its AAV gene therapy candidate, UX111, which is being developed for the treatment of Sanfilippo syndrome type A. A biologics license application for the same will likely be filed with the FDA in late 2024 or early next year.
The company is developing GTX-102 for treating patients with Angelman syndrome (AS). It is currently gearing up to initiate a phase III AS study, named Aspire, for GTX-102 by the end of 2024. RARE also anticipates initiating 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.
Ultragenyx Pharmaceutical Inc. Price and Consensus
In the past 60 days, estimates for ANI Pharmaceuticals’ 2024 earnings per share have moved up from $4.53 to $4.81. Earnings per share estimates for 2025 have improved from $5.38 to $5.86. Year to date, shares of ANIP have gained 3%.
ANIP’s earnings beat estimates in each of the trailing four quarters, with the average surprise being 31.32%.
In the past 60 days, estimates for Krystal Biotech’s 2024 EPS have increased from $1.91 to $2.38. The consensus estimate for 2025 earnings has improved from $4.33 to $7.31. Year to date, shares of KRYS have soared 41.8%.
KRYS’ earnings beat estimates in three of the trailing four quarters while missing on the remaining occasion, the average surprise being 45.95%.
In the past 60 days, estimates for Fulcrum Therapeutics’ 2024 loss per share have narrowed from 46 cents to 28 cents. The consensus estimate for 2025 loss per share has narrowed from $1.67 to $1.14. Year to date, shares of FULC have plunged 45.7%.
FULC’s earnings beat estimates in each of the trailing four quarters, the average surprise being 393.18%.
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RARE's Wilson Disease Candidate Betters Standard Therapy in Study
Ultragenyx Pharmaceutical (RARE - Free Report) reported positive updates from the Stage 1 cohorts of its phase I/II/III Cyprus2+ study evaluating UX701, an investigational AAV9 gene therapy, to treat Wilson disease. Stage 1 of the study is assessing the safety and efficacy of UX701 and a dose will be selected for further evaluation in Stage 2, which is the pivotal portion of the study.
Wilson disease is a genetic disorder where the body is unable to properly eliminate excess copper, leading to its buildup in organs like the liver, brain and eyes. This can cause liver damage, neurological issues and other serious symptoms. Per Ultragenyx, more than 50,000 people suffer from this indication in commercially accessible geographies.
The Stage 1 portion of the Cyprus2+ study, which enrolled 15 patients into three sequential dosing cohorts and followed for at least 24 weeks, demonstrated meaningful clinical activity and improvements in copper metabolism. Management believes that UX701 has the potential to become a novel therapy for Wilson disease patients.
Efficacy Data From RARE’s Study of Wilson Disease Candidate
According to the data, six out of the 15 enrolled patients fully discontinued standard-of-care treatments involving chelators and/or zinc therapy, with positive responses across all three dosage groups. Furthermore, a seventh patient had started to reduce the dependency on standard-of-care treatment as of the data cut-off date in August.
Interestingly, non-ceruloplasmin-bound copper levels returned to normal in patients who stopped standard therapy. Some also showed increased ceruloplasmin-copper activity, indicating better ATP7b function.
Year to date, shares of RARE have gained 12.2% against the industry’s 1.6% decline.
Image Source: Zacks Investment Research
Ultragenyx reported that the investigational gene therapy has been overall well-tolerated in the Stage 1 cohorts of the Cyprus2+ study. As of the data cut-off date, no unexpected related treatment-emergent adverse events and no significant immunologic safety events have been observed.
Based on the encouraging update from the Wilson Disease study, Ultragenyx plans to submit a protocol amendment to add a new cohort in Stage 1, where patients will receive a slightly higher dose of UX701, along with an improved immunomodulation regimen to make the gene therapy more effective. The goal is for most patients to stop standard treatment before moving to the next phase of the study (Stage 2), which will involve a randomized placebo-controlled study.
Other Key Clinical Programs in RARE’s Pipeline
Apart from UX701, Ultragenyx has several other interesting gene therapy candidates in its pipeline. The company, in partnership with Mereo BioPharma, is developing UX143 (setrusumab) monoclonal antibody forpediatric and young adult patients with osteogenesis imperfecta in two late-stage studies, Orbit and Cosmic.
In May 2024, Ultragenyx announced that the phase III GlucoGene study (NCT05139316), 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 GlucoGene study also met its key secondary endpoints. The next step for the company involves discussing these results with regulatory authorities to support a marketing application in 2025.
Ultragenyx is also planning to seek accelerated approval for its AAV gene therapy candidate, UX111, which is being developed for the treatment of Sanfilippo syndrome type A. A biologics license application for the same will likely be filed with the FDA in late 2024 or early next year.
The company is developing GTX-102 for treating patients with Angelman syndrome (AS). It is currently gearing up to initiate a phase III AS study, named Aspire, for GTX-102 by the end of 2024. RARE also anticipates initiating 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.
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 stocks in the biotech sector are ANI Pharmaceuticals (ANIP - Free Report) , Krystal Biotech, Inc. (KRYS - Free Report) and Fulcrum Therapeutics (FULC - 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 60 days, estimates for ANI Pharmaceuticals’ 2024 earnings per share have moved up from $4.53 to $4.81. Earnings per share estimates for 2025 have improved from $5.38 to $5.86. Year to date, shares of ANIP have gained 3%.
ANIP’s earnings beat estimates in each of the trailing four quarters, with the average surprise being 31.32%.
In the past 60 days, estimates for Krystal Biotech’s 2024 EPS have increased from $1.91 to $2.38. The consensus estimate for 2025 earnings has improved from $4.33 to $7.31. Year to date, shares of KRYS have soared 41.8%.
KRYS’ earnings beat estimates in three of the trailing four quarters while missing on the remaining occasion, the average surprise being 45.95%.
In the past 60 days, estimates for Fulcrum Therapeutics’ 2024 loss per share have narrowed from 46 cents to 28 cents. The consensus estimate for 2025 loss per share has narrowed from $1.67 to $1.14. Year to date, shares of FULC have plunged 45.7%.
FULC’s earnings beat estimates in each of the trailing four quarters, the average surprise being 393.18%.