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Ultragenyx (RARE) Begins Angelman Syndrome Study in Canada

Ultragenyx (RARE) and GeneTx Biotherapeutics dose the first patient in a phase I/II study on GTX-102 for treating patients with Angelman syndrome in Canada.

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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 they have dosed the first patient in a phase I/II study in Canada, evaluating GTX-102 for the treatment of Angelman syndrome, a rare neurogenetic disorder. Three other patients have been enrolled and scheduled for dosing.

- Zacks

The open-label, multiple-dose study is investigating the safety, tolerability and efficacy of GTX-102 in pediatric patients with Angelman syndrome having genetically confirmed diagnosis of full maternal UBE3A gene deletion.

Shares of Ultragenyx have plunged 38.3 % so far this year compared with the industry’s decrease of 11.9%.

Zacks Investment ResearchImage Source: Zacks Investment Research

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.

Per the approved protocol in the United Kingdom and Canada, 12 patients will be enrolled in the study and split into two cohorts according to their age. Patients aged between four to less than eight years will be enrolled in the cohort 4 and receive GTX-102 (3.3 mg). Patients aged between eight to less than 18 years will be enrolled in the cohort 5 and receive GTX-102 (5 mg). A preliminary update on the same is expected by the end of 2021.

We remind investors that in October 2020, Ultragenyx announced interim data from the phase I/II study on GTX-102 for treating Angelman syndrome. Back then, the companies decided to cease enrollment and dosing in the study after all patients experienced a serious adverse event of lower extremity weakness related to local inflammation, following treatment with the highest doses of GTX-102.

Last month, the FDA removed the clinical hold from Ultragenyx and GeneTx’s phase I/II study evaluating GTX-102 for the treatment of Angelman syndrome. The companies can now begin dosing naïve patients in the same for treating pediatric patients with Angelman syndrome.

Zacks Rank & Stocks to Consider

Ultragenyx currently carries a Zacks Rank #4 (Sell).

Some better-ranked stocks in the biotech sector include vTv Therapeutics Inc. VTVT, Gritstone bio, Inc. GRTS and Evelo Biosciences, Inc. EVLO, 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.

vTv Therapeutics’ loss per share estimates have narrowed 8% for 2021 and 2.8% for 2022 over the past 60 days.

Gritstone bio’s loss per share estimates have narrowed 26.1% for 2021 and 31.8% for 2022, over the past 60 days. The stock has skyrocketed 142.7% year to date.

Evelo Biosciences’ loss per share estimates have narrowed 0.4% for 2021 and 3.5% for 2022, over the past 60 days.



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