Ultragenyx Reports Top-line Data From Gene Therapy Candidate

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Ultragenyx Pharmaceutical Inc. RARE announced positive top-line safety and efficacy data from the second dose cohort and positive longer-term data from the first dose cohort of the ongoing OTC phase I/II study of its adeno-associated virus ("AAV") gene therapy candidate - DTX301.DTX301 is being evaluated forthe treatment of ornithine transcarbamylase (OTC) deficiency.

To evaluate the therapeutic response of DTX301, the study measures the change in the rate of ureagenesis.

In cohort 1, a dose of 2.0 × 10^12 GC/kg of DTX301 was given to 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 the first patient in the cohort, who experienced ureagenesis normalization, has completed the 52-week study period and experienced an increased level of ureagenesis at 52 weeks as well as clinical stability after discontinuing alternate pathway medication.

Three patients in cohort 2 were given a single dose of DTX301 at 6.0 × 10^12 GC/kg. The first patient in Cohort 2 demonstrated normalization of ureagenesis to 104% at week 24. The patient showed normalization of ureagenesis at week 6 which then declined at Week 12 due to a tapering course of steroids. After discontinuation of steroids, the rate of ureagenesis normalized at week 20 and remained normal at week 24, at which time a second course of steroids were initiated due to mild asymptomatic ALT elevations, which were controlled.

The second and third patients in Cohort 2 did not show a clinically meaningful change in rate of ureagenesis at week 12 and did not have alanine aminotransferase (ALT) elevations nor require steroid treatment.

The share price of the company fell 6.9% which could be because the efficacy rate at the higher dose was below the market expectations.

However, share price of the company has increased 72.1% year to date against with the industry 's decline of 1.3%.

The Data Monitoring Committee (DMC) has completed its review of week 12 data from cohort 2 and recommended that Ultragenyx should proceed to the third dose cohort of the study. In cohort 3, three patients will be enrolled and each will receive a single dose of 1.0 × 10^13 GC/kg.

The company also has other gene-therapy candidates in its pipeline.

Ultragenyxis developing DTX201, its FVIII gene-therapy program, for the treatment of hemophilia A in collaboration with Bayer BAYRY . The investigational new drug (IND) application was accepted by the FDA and made active in the second quarter of 2018.

DTX401 is another AAV8 gene-therapy candidate being evaluated for the treatment of patients with glycogen storage disease type Ia (GSDIa). The FDA granted Fast Track designation to DTX401 for the treatment of GSDIa in July 2018.

A potential approval of these candidates will drive the company's revenues.

Ultragenyx Pharmaceutical Inc. Price

Ultragenyx Pharmaceutical Inc. Price | Ultragenyx Pharmaceutical Inc. Quote

Zacks Rank & Stocks to Consider

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

Some better-ranked stocks in the biotech sector are Anika Therapeutics Inc. ANIK and Ligand Pharmaceuticals Incorporated LGND . Both Anika and Ligand sport a Zacks Rank #1 (Strong Buy).You can see  the complete list of today's Zacks #1 Rank stocks here .

Anika's earnings per share estimates have increased from 81 cents to 95 cents for 2018 and from $1.43 to $1.49 for 2019 over the past 60 days. The company delivered a positive earnings surprise in two of the trailing four quarters with an average beat of 20.84%.

Ligand's earnings per share estimates have moved up from $5.64 to $6.33 for 2018 and from $5.59 to $5.74 for 2019 in the last 30 days. The company delivered a positive earnings surprise in all of the trailing four quarters with an average beat of 59.54%. Share price of the company has increased 100.1% year to date.

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The views and opinions expressed herein are the views and opinions of the author and do not necessarily reflect those of Nasdaq, Inc.

This article appears in: Investing , Business , Stocks
Referenced Symbols: BAYRY , RARE , ANIK , LGND

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