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Ultragenyx's Gene Therapy Positive in Genetic Disorder Study

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Shares of Ultragenyx Pharmaceutical Inc. (RARE - Free Report) rallied 23.4% after it announced positive top-line safety and efficacy data from the ongoing phase I/II study of an investigational adeno-associated virus (AAV) gene therapy — DTX301. DTX301 is being evaluated for the treatment of ornithine transcarbamylase (OTC) deficiency. OTC deficiency leads to the build up of excessive levels of ammonia in the blood, resulting in acute and chronic neurological deficits and other toxicities.

However, shares of the company have gained 10.7% in the past year against the industry’s decline of 1.2%.

The company announced positive safety and efficacy data from Cohort 3 and longer-term data from Cohort 2. In Cohort 3, there were two confirmed female responders as well as a third potential male responder who required longer-term follow-up to confirm response status.  

 In Cohort 2, one female patient newly demonstrated a response starting Week 52, which was confirmed at Week 78. There were nine patients dosed in the study, of which six have demonstrated a response.

The company expects to start the fourth cohort to evaluate prophylactic steroid administration of DTX301 in the first half of 2020. The data from the fourth cohort are expected in the second half of the year. The company expects the data from the fourth cohort to be consistent with the other cohorts.

The company is also in discussions with the FDA regarding a potential phase III study design for the same.

Other than DTX301, the gene therapy pipeline includes DTX401 (for the treatment of patients with glycogen storage disease type Ia) in clinical development.

Moreover, the company has an agreement with Bayer (BAYRY - Free Report) to research, develop and commercialize AAV gene-therapy DTX 201 for the treatment of hemophilia A


Zacks Rank & Stocks to Consider

Ultragenyx is currently a Zacks Rank #3 (Hold) stock.

A couple of better-ranked stocks in the biotech sector are Alexion Pharmaceuticals Inc. (ALXN - Free Report) and Cue Biopharma Inc. (CUE - Free Report) , both sporting a Zacks Rank #1 (Strong Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.

Alexion’s’ earnings per share estimates have increased from $9.97 to $10.35 for 2019 and from $10.87 to $11.16 for 2020 in the past 90 days. The company delivered a positive earnings surprise in the trailing four quarters by 12.50%, on average.

Cue’s loss per share estimates have narrowed from $1.96 to $1.72 for 2019 and from $1.35 to $1.25 for 2020 in the past 90 days. The company delivered a positive earnings surprise in the trailing four quarters by 23.71%, on average.

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