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Ultragenyx Pharmaceutical Inc. RARE announced that it has dosed the first patient in a phase I/II study evaluating its investigational messenger RNA (mRNA) therapy, UX053, for the treatment of Glycogen Storage Disease Type III (GSDIII), a rare disease affecting more than 10,000 patients across the world.
The two-part phase I/II study will investigate the safety, tolerability and efficacy of UX053 in patients aged 18 years and above with GSDIII.
Per the company, part 1 of the above-mentioned study is open label and will enroll up to 10 patients who will get a single ascending dose of intravenous UX053. While part 2 is double-blind and will evaluate five repeat doses of UX053 at escalating dose levels in up to 16 patients across four cohorts.
The primary endpoints of the study are to check the treatment-emergent adverse events (TEAEs), serious TEAEs and related TEAEs in both parts of the study, while the secondary endpoints include pharmacokinetic parameters.
Shares of Ultragenyx have plunged 45% so far this year compared with the industry’s decrease of 16.1%.
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We note that Ultragenyx’s most advanced investigational mRNA therapy is UX053, which is being developed for treating GSDIII. In pre-clinical studies, UX053 has demonstrated reductions in glycogen content in the liver in multiple models of the disease.
The FDA and the European Commission have granted Orphan Drug designation to UX053 for the treatment of GSDIII.
Apart from mRNA candidates, Ultragenyx also has some promising gene therapy candidates in its pipeline.
DTX301, which is an adeno-associated virus 8 (AAV8) gene therapy product candidate, is being evaluated for the treatment of patients with Ornithine transcarbamylase deficiency. DTX401 is also an AAV8 gene therapy candidate currently being evaluated for the treatment of patients with glycogen storage disease type Ia (GSDIa).
In October 2021, Ultragenyx initiated a seamless phase I/II/III study on UX701, an AAV type 9 gene therapy product candidate being developed for the treatment of Wilson disease — a rare metabolic disease.
The successful development and commercialization for any of these candidates will be a boost for the company’s growth prospects.
Zacks Rank & Stocks to Consider
Ultragenyx currently carries a Zacks Rank #3 (Hold). Better-ranked stocks in the biotech sector include Sarepta Therapeutics, Inc. SRPT, Editas Medicine, Inc. EDIT and vTv Therapeutics Inc. VTVT, 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.
Sarepta Therapeutics’ loss per share estimates have narrowed 31.3% for 2021 and 26% for 2022, over the past 60 days.
Earnings of Sarepta Therapeutics have surpassed estimates in two of the trailing four quarters, and missed the same on the other two occasions.
Editas Medicine’s loss per share estimates have narrowed 11.2% for 2021 and 4.6% for 2022, over the past 60 days.
Editas Medicine’s earnings have surpassed estimates in two of the trailing four quarters and missed the same on the other two occasions.
vTv Therapeutics’ loss per share estimates have narrowed 21.7% for 2021 and 2.9% for 2022, over the past 60 days.
vTv Therapeutics’ earnings have surpassed estimates in three of the trailing four quarters and missed the same on the other occasion.
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