Ultragenyx's Fatty Acid Disorder Candidate Succeeds in Study

Ultragenyx Pharmaceutical Inc.RARE announced positive top-line data from an ongoing long-term safety and efficacy study of UX007 in patients with long-chain fatty acid oxidation disorder (LC-FAOD). Patients with this disorder are unable to properly break down fatty acids into energy, leading to serious liver, muscle and heart issues.

Shares of the company went up about 2.9% following the news. However, shares of the company have declined 10.4% in the past year compared with the industry 's decline of 22.8%.

The long-term extension study showed sustained reductions in the duration and frequency of major clinical events (MCE), after an additional 78 weeks of treatments with UX007. The study had a long-term safety profile similar to what has previously been seen with UX007 in its phase II study. Results from the phase II study enrolling 29 patients had shown that the UX007-treated patients had fewer illness-related visits to the hospital and fewer days in the hospital per year.

The current phase III study had 75 patients enrolled, out of which 24 patients were previously enrolled in the company-sponsored phase II study, 20 naïve patients who had not previously been treated with UX007 and 31 patients from expanded access or investigator-sponsored studies. The 20 patients naïve to UX007 also demonstrated meaningful reductions in major clinical events.

Ultragenyx is on track to submit the new drug application (NDA) for UX007 in mid-2019. The submission will include these data from this long-term efficacy and safety extension study in 75 patients, and the phase II study of UX007 in 29 patients. The submission will also provide a retrospective medical record review of 20 original compassionate use patients, data from 70 patients treated through expanded access, and a randomized controlled investigator-sponsored study of 32 patients showing effect of UX007 on cardiac function.

Ultragenyx has a number of products in its pipeline. The company is also looking to expand the label of Crysvita. The drug is being developed for the treatment of tumor-induced osteomalacia, (TIO). The company also has some gene therapy candidates like DTX301, which is an adeno-associated virus 8 (AAV8) being evaluated for the treatment of patients with Ornithine transcarbamylase (OTC) deficiency. DTX401 is another AAV8 gene-therapy candidate being evaluated for the treatment of patients with glycogen storage disease type Ia (GSDIa).

Ultragenyx is developing DTX201, its FVIII gene-therapy program for the treatment of hemophilia A, in collaboration with Bayer BAYRY .

A potential approval of these candidates will be a boost for the company's revenues.

Ultragenyx Pharmaceutical Inc. Price

Ultragenyx Pharmaceutical Inc. Price | Ultragenyx Pharmaceutical Inc. Quote

Zacks Rank & Stocks to Consider

Ultragenyx currently carries a Zacks Rank #3 (Hold).

Some better-ranked stocks worth considering are Celgene Corporation CELG and Axovant Sciences Ltd. AXON . Both the companies sport a Zacks Rank #1 (Strong Buy). You can see the complete list of today's Zacks #1 Rank stocks here.

Celgene's earnings per share estimates have increased from $8.78 to $8.80 for 2018 and in the past 90 days. The company delivered a positive earnings surprise in all the trailing four quarters, with average of 2.54%.

Axovant's loss per share estimates have narrowed from $1.60 to $1.25 for 2019 and from $1.35 to $1.03 for 2020 in the past 90 days.

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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.

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

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