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Ultragenyx Announces Interim Data from Early Stage Study


Ultragenyx Pharmaceutical Inc. RARE announced positive interim data from the first dose cohort of the phase I/II study on its adeno-associated virus (“AAV”) gene therapy candidate, DTX301. The study is evaluating DTX301 as a treatment for ornithine transcarbamylase (“OTC”) deficiency, the most common urea cycle disorder, which increases concentration of ammonia in blood.

However, shares of the company have declined 32.1% in the past year, underperforming the industry’s fall of 1.8% in that period.

The study evaluated a dose of 2.0 × 10^12 GC/kg of DTX301 in patients with late-onset of OTC deficiency and who are clinically stable and on a stable dose of alternate pathway medication. Data from the study showed that DTX301 normalized the rate of ureagenesis, which helps in metabolism of ammonia.

Meanwhile, the FDA has approved Ultragenyx’s first drug, MEPSEVII (vestronidase alfa), for the treatment of children and adults with mucopolysaccharidosis VII, also known as Sly syndrome in November 2017. A marketing authorization application is under review in Europe.

The company is also developing other pipeline candidates like burosumab (KRN23) in children with X-linked hypophosphatemia (“XLH”) and adult with tumor-induced osteomalacia (“TIO”), and UX007 in glucose transporter type-1 deficiency syndrome and long-chain fatty acid oxidation disorders.

Ultragenyx had announced positive data from two phase II studies on burosumab in August 2017. A biologics license application (“BLA”) filed on the basis of this data is under review in the United States seeking approval for treating XLH. Moreover, the CHMP recommended the approval of the candidate in Europe last month for the same indication.

However, the company discontinued its late stage candidate, Ace-ER, in August 2017 due to disappointing data in a phase III study, evaluating it for GNE myopathy, a progressive muscle-wasting disorder.

Investors remain focused on Ultragenyx’s progress on its pipeline and commercialization plan of MEPSEVII.

Zacks Rank & Stocks to Consider

Ultragenyx carries a Zacks Rank #3 (Hold).

Some better-ranked stocks in the pharma sector include Celldex Therapeutics, Inc. CLDX, Anthera Pharmaceuticals, Inc. ANTH and Catabasis Pharmaceuticals, Inc. CATB. All the stocks carry a Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.

Celldex’s loss estimates remained stable at 93 cents for 2017 and at 90 cents for 2018 in the last 60 days. The company delivered a positive earnings surprise in three of the trailing four quarters with an average beat of 15.36%.

Anthera’s loss estimates narrowed from $3.14 to $3.09 for 2017 and from $2.86 to $2.31 in the last 30 days. The company came up with a positive earnings surprise in three of the trailing four quarters with an average beat of 5.52%. The company’s shares have returned 5.7% so far this year.

Catabasis’s loss estimates narrowed from $1.39 to $1.37 for 2017 and from $1.09 to 89 cents for 2018 over the last 30 days. The company delivered a positive earnings surprise in all the trailing four quarters with an average beat of 9.94%.

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