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Ultragenyx (RARE) Meets Efficacy Goal in Neuro Disease Study
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Ultragenyx Pharmaceutical Inc. (RARE - Free Report) 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.
Per the data readout, patients in Expansion Cohorts A & B treated with a set dose and regimen of GTX-102 showed rapid and clinically meaningful improvement across multiple domains at day 170, which is either consistent with or exceeded the results from the Dose-escalation Cohorts 4-7 data.
Such improvements across multiple functional domains include improvements in cognitive function, behavior, hyperactivity and noncompliance, sleep, receptive communication and gross motor function.
Ultragenyx further reported that the treatment of Angelman syndrome patients in the Dose-escalation Cohorts 4-7 with GTX-102 demonstrated long-term increasing and sustained clinical benefit far exceeding the Natural History study data of the candidate at day 758.
Despite the positive results, the stock lost 8.8% in the last trading session on Apr 15. The decline in the share price was likely due to reports of three patients in the phase I/II study of GTX-102 who experienced serious adverse events (mild to moderate) of lower extremity weakness assessed as related to study treatment.
Although the adverse events in the three Angelman syndrome patients were resolved quickly without sequelae and continued treatment in the study without ongoing safety concerns, the investors remain skeptical about the safety profile of the candidate.
Year to date, shares of Ultragenyx have plunged 10.3% compared with the industry’s 7.9% decline.
Image Source: Zacks Investment Research
The company plans to report detailed results from the interim analysis of the phase I/II Angelman syndrome study at an upcoming medical conference.
The phase I/II multiple-dose and dose-escalating study is evaluating the safety and tolerability of GTX-102 administered by intrathecal injection to pediatric patients with Angelman syndrome with a genetically confirmed diagnosis of full maternal UBE3A gene deletion.
The study is further assessing clinical response as measured by a panel of efficacy assessments for the functional domains affected by Angelman syndrome. A total of 74 patients have been enrolled and treated in both the dose-escalation and expansion cohorts of the early to mid-stage study. Patients in the Cohorts 4-7 (dose-escalation) are receiving long-term maintenance dosing.
Ultragenyx stated that there are currently no approved disease-modifying treatments for Angelman syndrome, which represents a significant unmet medical need for patients suffering from the disease.
The cumulative interim analysis data from the phase I/II study of GTX-102 demonstrates the candidate's potential to address such unmet needs.
RARE is currently planning to discuss these results from the phase I/II study of GTX-102 with the FDA and other regulatory authorities to enable the timely initiation of a phase III pivotal study of the candidate for Angelman syndrome.
Ultragenyx Pharmaceutical Inc. Price and Consensus
In the past 30 days, the Zacks Consensus Estimate for ADMA Biologics’ 2024 earnings per share (EPS) has remained constant at 30 cents. During the same period, the estimate for ADMA’s 2025 EPS has remained constant at 50 cents. Year to date, shares of ADMA have soared 32.3%.
ADMA beat estimates in three of the trailing four quarters and matched in one, delivering an average earnings surprise of 85%.
In the past 30 days, the Zacks Consensus Estimate for FibroGen’s 2024 loss per share has remained constant at $1.09. During the same period, the estimate for FibroGen’s 2025 loss per share has remained constant at 6 cents. Year to date, shares of FGEN have rallied 47.8%.
FGEN beat estimates in two of the trailing four quarters, missing the mark on the other two occasions, delivering an average negative surprise of 2.26%.
In the past 30 days, the Zacks Consensus Estimate for Annovis’ 2024 loss per share has narrowed from $3.49 to $3.35. The estimate for Annovis’ 2025 loss per share is currently pegged at $2.82. Year to date, shares of ANVS have plunged 40.2%.
ANVS’ earnings beat estimates in two of the trailing four quarters and missed the mark on the other two occasions, delivering an average negative surprise of 15.70%.
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Ultragenyx (RARE) Meets Efficacy Goal in Neuro Disease Study
Ultragenyx Pharmaceutical Inc. (RARE - Free Report) 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.
Per the data readout, patients in Expansion Cohorts A & B treated with a set dose and regimen of GTX-102 showed rapid and clinically meaningful improvement across multiple domains at day 170, which is either consistent with or exceeded the results from the Dose-escalation Cohorts 4-7 data.
Such improvements across multiple functional domains include improvements in cognitive function, behavior, hyperactivity and noncompliance, sleep, receptive communication and gross motor function.
Ultragenyx further reported that the treatment of Angelman syndrome patients in the Dose-escalation Cohorts 4-7 with GTX-102 demonstrated long-term increasing and sustained clinical benefit far exceeding the Natural History study data of the candidate at day 758.
Despite the positive results, the stock lost 8.8% in the last trading session on Apr 15. The decline in the share price was likely due to reports of three patients in the phase I/II study of GTX-102 who experienced serious adverse events (mild to moderate) of lower extremity weakness assessed as related to study treatment.
Although the adverse events in the three Angelman syndrome patients were resolved quickly without sequelae and continued treatment in the study without ongoing safety concerns, the investors remain skeptical about the safety profile of the candidate.
Year to date, shares of Ultragenyx have plunged 10.3% compared with the industry’s 7.9% decline.
Image Source: Zacks Investment Research
The company plans to report detailed results from the interim analysis of the phase I/II Angelman syndrome study at an upcoming medical conference.
The phase I/II multiple-dose and dose-escalating study is evaluating the safety and tolerability of GTX-102 administered by intrathecal injection to pediatric patients with Angelman syndrome with a genetically confirmed diagnosis of full maternal UBE3A gene deletion.
The study is further assessing clinical response as measured by a panel of efficacy assessments for the functional domains affected by Angelman syndrome. A total of 74 patients have been enrolled and treated in both the dose-escalation and expansion cohorts of the early to mid-stage study. Patients in the Cohorts 4-7 (dose-escalation) are receiving long-term maintenance dosing.
Ultragenyx stated that there are currently no approved disease-modifying treatments for Angelman syndrome, which represents a significant unmet medical need for patients suffering from the disease.
The cumulative interim analysis data from the phase I/II study of GTX-102 demonstrates the candidate's potential to address such unmet needs.
RARE is currently planning to discuss these results from the phase I/II study of GTX-102 with the FDA and other regulatory authorities to enable the timely initiation of a phase III pivotal study of the candidate for Angelman syndrome.
Ultragenyx Pharmaceutical Inc. Price and Consensus
Ultragenyx Pharmaceutical Inc. price-consensus-chart | Ultragenyx Pharmaceutical Inc. Quote
Zacks Rank & Stocks to Consider
Ultragenyx currently carries a Zacks Rank #3 (Hold).
Some better-ranked stocks from the drug/biotech industry are ADMA Biologics (ADMA - Free Report) , FibroGen (FGEN - Free Report) and Annovis Bio (ANVS - Free Report) . While ADMA sports a Zacks Rank #1 (Strong Buy), FGEN and ANVS carry a Zacks Rank #2 (Buy) each 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 ADMA Biologics’ 2024 earnings per share (EPS) has remained constant at 30 cents. During the same period, the estimate for ADMA’s 2025 EPS has remained constant at 50 cents. Year to date, shares of ADMA have soared 32.3%.
ADMA beat estimates in three of the trailing four quarters and matched in one, delivering an average earnings surprise of 85%.
In the past 30 days, the Zacks Consensus Estimate for FibroGen’s 2024 loss per share has remained constant at $1.09. During the same period, the estimate for FibroGen’s 2025 loss per share has remained constant at 6 cents. Year to date, shares of FGEN have rallied 47.8%.
FGEN beat estimates in two of the trailing four quarters, missing the mark on the other two occasions, delivering an average negative surprise of 2.26%.
In the past 30 days, the Zacks Consensus Estimate for Annovis’ 2024 loss per share has narrowed from $3.49 to $3.35. The estimate for Annovis’ 2025 loss per share is currently pegged at $2.82. Year to date, shares of ANVS have plunged 40.2%.
ANVS’ earnings beat estimates in two of the trailing four quarters and missed the mark on the other two occasions, delivering an average negative surprise of 15.70%.