Ultragenyx (RARE) Posts Update From AS Study of GTX-102
Ultragenyx Pharmaceutical Inc. RARE completed enrollment in the new expansion cohorts in its early to mid-stage study of GTX-102 for the treatment of pediatric patients with Angelman syndrome (AS). The company reported that it has enrolled 53 patients in the dose-expansion cohorts (Cohorts A-E) for a total of 74 patients enrolled globally in the phase I/II AS study.
GTX-102 is Ultragenyx’s investigational antisense oligonucleotide, which is designed to target and inhibit the expression of UBE3A antisense transcript.
Having completed enrollment, RARE claims to remain on track to report results from the phase I/II study of GTX-102 for AS in the first half of 2024 from at least 20 expansion cohort patients on therapy for at least six months.
Management expects the cumulative safety and efficacy data from new expansion cohorts to verify the GTX-102 dose range, treatment regimen and endpoint selection, supporting the initiation of the company’s phase III program for AS.
Over the past year, shares of Ultragenyx have gained 1.5% against the industry’s 9.7% decline.
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The phase I/II dose-escalating study is evaluating the safety and tolerability of GTX-102 administered by intrathecal injection to pediatric patients with AS with a genetically confirmed diagnosis of full maternal UBE3A gene deletion. The study is also evaluating the clinical response in patients treated with GTX-102 as measured by a panel of efficacy assessments for the functional domains impacted in AS.
In October 2023, Ultragenyx reported positive interim data from the extension cohorts (Cohorts 4-7) of the ongoing phase I/II study of GTX-102 for AS.
The candidate also continues to demonstrate a manageable safety profile with no additional treatment-related adverse events reported in the study. Per the data readout, improvements were observed across multiple clinical domains compared with natural history data, where available and clinical changes were associated with quantitative changes in EEG. Furthermore, long-term data demonstrated that patients who stopped and restarted treatment with GTX-102 reacquired previously gained developmental skills when they were re-dosed with the current regimen.
GTX-102 currently enjoys the FDA’s Orphan Drug, Rare Pediatric Disease and Fast Track designation in the United States for the AS indication.
AS is a rare, neurogenetic disorder caused by loss-of-function of the maternally inherited allele of the UBE3A gene. Per Ultragenyx, AS is estimated to affect one in 12,000 to one in 20,000 people globally. There are no currently approved therapies for AS.
Patients suffering from AS have developmental delay, balance issues, motor impairment and debilitating seizures. Some of these patients are also unable to walk and most do not speak. This takes a serious toll on the quality of life of patients suffering from AS and represents a significant unmet medical need.
Ultragenyx Pharmaceutical Inc. Price and Consensus
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Zacks Rank and Other Stocks to Consider
Ultragenyx currently carries a Zacks Rank #2 (Buy).
Some other top-ranked drug/biotech stocks worth mentioning are Puma Biotechnology, Inc. PBYI, ADMA Biologics ADMA and Acadia Pharmaceuticals ACAD. While PBYI sports a Zacks Rank #1 (Strong Buy), ADMA and ACAD carry a Zacks Rank #2 each at present. You can see the complete list of today’s Zacks #1 Rank stocks here.
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ADMA beat estimates in three of the trailing four quarters and matched in one, delivering an average earnings surprise of 63.57%.
In the past 30 days, the Zacks Consensus Estimate for Acadia’s 2023 loss per share has remained constant at 33 cents. During the same time frame, the consensus estimate for Acadia’s 2024 EPS is pegged at $1.04. Over the past year, shares of ACAD have gained 71.7%.
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