Ultragenyx Pharmaceutical Inc. RARE announced positive data from a phase II study on UX007 (triheptanoin) for the treatment of patients with long-chain fatty acid oxidation disorder (LC-FAOD). The data was collected at the end of the 78-week treatment period.
The single-arm, open-label phase II study evaluated patients (n=29) with moderate-to-severe FAOD across three symptoms groups – musculoskeletal, liver/hypoglycemia and cardiac symptoms.
Results demonstrated that treatment with UX007 reduced the frequency and duration of major medical events while improving participants' exercise tolerance and quality of life. Data revealed a 48.1% reduction in the mean annualized rate of major medical events (events that led to hospitalization, emergency room visit or an emergency intervention at home).
However, five patients discontinued evaluation in the study, one due to diarrhea at week 1 which was cured after treatment was stopped and four for reasons not related to treatment with the candidate.
The company disclosed that it is developing the design and endpoints of a phase III study before meeting with regulators, with plans to initiate the study in 2017.
Note that UX007 is also being evaluated in a phase II study for the treatment of patients with glucose transporter type-1 deficiency syndrome (Glut1 DS). Results from the study are expected in the first quarter of 2017. Moreover, a phase III study on the candidate for Glut1 DS is expected to be initiated by the year end.
Note that UX007 enjoys orphan drug status in the U.S. for the treatment of FAOD and Glut1 DS. In the EU as well, the candidate was granted orphan drug status for the treatment of Glut1 DS.
A look at Ultragenyx’s year-to-date share price movement shows that the stock has tanked 30.2%, compared to the 23.2% decline for the Zacks categorized Medical/Biomedical Genetics industry.
Zacks Rank & Key Picks
Ultragenyx currently carries a Zacks Rank #3 (Hold). Some better-ranked stocks in the health care sector include Cambrex Corporation CBM, Heska Corporation HSKA and Vanda Pharmaceuticals, Inc. VNDA. Each of these stocks sports a Zacks Rank #1 (Strong Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.
Cambrex’s earnings estimates increased from $2.46 to $2.55 for 2016 and from $2.91 to $3.06 for 2017 over the last 60 days. The company posted a positive earnings surprise in three of the four trailing quarters with an average beat of 19.78%. Its share price has increased 6.4% year to date.
Heska’s earnings estimates have increased from $1.13 to $1.35 for 2016 and from $1.38 to $1.53 for 2017 over the last 60 days. The company posted a positive earnings surprise in all of the four trailing quarters with an average beat of 301.64%. Its share price has surged 70.9% year to date.
Vanda’s loss estimates narrowed from 68 cents to 56 cents for 2016, while its earnings estimates have increased from 16 cents to 17 cents for 2017 over the last 60 days. The company posted a positive earnings surprise in three of the four trailing quarters with an average positive surprise of 56.65%. Its share price has surged 76.7% year to date.
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