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Ultragenyx (RARE) Reports FDA Removal of Clinical Hold on Study

Ultragenyx (RARE) and GeneTx Biotherapeutics to start dosing in the phase I/II study on GTX-102 for treating pediatric patients with Angelman syndrome...

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This story originally appeared on Zacks

Ultragenyx Pharmaceutical Inc. RARE, along with partner GeneTx Biotherapeutics LLC, a privately-held biotechnology company, announced that the FDA has removed the clinical hold from their phase I/II study evaluating GTX-102 for the treatment of Angelman Syndrome, a rare neurogenetic disorder.

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The companies can now begin dosing naïve patients in the above-mentioned study for treating pediatric patients with Angelman syndrome. The open-label, multiple-dose study will evaluate the safety, tolerability, and plasma and cerebrospinal fluid concentrations of GTX-102 in the given patient population.

Per the company, under the amended study protocol in the United States, eight patients aged between four to less than eight years who have not received previous treatment with GTX-102 will be enrolled in the study into two groups – an active group and an age-matched comparator group.

Patients in the active group will receive four monthly doses of GTX-102 (2 mg), while patients in the comparator group will have limited assessments at baseline and day 128.

Shares of Ultragenyx declined 35.9% so far this year compared with the industry’s decrease of 1.3%.

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We remind investors that last October, Ultragenyx announced interim data from the phase I/II study on GTX-102 for treating Angelman syndrome. Data from the same showed that all the patients who were treated with GTX-102 had experienced substantial improvements in at least two disease domains, including communication, behavior, sleep, gross motor function and fine motor function, as measured by the Clinical Global Impression of Improvement Scale for Angelman Syndrome at day 128.

However, all patients experienced a serious adverse event of lower extremity weakness related to local inflammation following treatment with the highest doses of GTX-102. Back then, the companies decided to cease enrollment and dosing in the study.

Earlier this year, Health Canada cleared a protocol amendment and the Medicines and Healthcare Products Regulatory Agency in the United Kingdom approved a Clinical Trial Application to initiate the phase I/II study on GTX-102 in the two countries, respectively. Early clinical data from the same is expected before the end of 2021.

Zacks Rank & Stocks to Consider

Ultragenyx currently carries a Zacks Rank #3 (Hold). Better-ranked stocks in the biotech sector include Spero Therapeutics, Inc. SPRO, Corvus Pharmaceuticals, Inc. CRVS and Vertex Pharmaceuticals Incorporated VRTX, all carrying a Zacks Rank #2 (Buy) at present. You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.

Spero Therapeutics’ loss per share estimates have narrowed 8.2% for 2021 and 10.6% for 2022 over the past 60 days.The stock has inched up 0.4% year to date.

Corvus Pharmaceuticals’ loss per share estimates have narrowed 13.9% for 2021 and 7.2% for 2022, over the past 60 days. The stock has skyrocketed 115.5% year to date.

Vertex’s earnings estimates have been revised 10.2% upward for 2021 and 7.3% upward for 2022 over the past 60 days.



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