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Ultragenyx's Seizure Candidate UX007 Fails in Phase II Study
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Ultragenyx Pharmaceutical Inc. (RARE - Free Report) recently announced that its pipeline candidate UX007 (triheptanoin) failed to meet the primary endpoint in a phase II study evaluating its safety and efficacy for the treatment of patients with glucose transporter type-1 deficiency syndrome (Glut1 DS) with seizures. Shares declined in after-market trading on Mar 23.
The randomized, double-blind, placebo controlled phase II study was conducted in a total of 36 patients. The study did not show reduction of the frequency of total number of observable and absence seizures among patients treated from baseline to week eight with UX007 compared with placebo. Patients treated with UX007 demonstrated a reduction of 13.4% in overall seizure frequency versus placebo.
Moreover, 14 patients discontinued evaluation in the study – two due to adverse events at week eight, four due to tolerability-related issues, and eight due to compliance or study burden.
Though the drug did show a reduction in absence seizures captured on EEG when each seizure type was evaluated independently, it did not reduce observable seizures.
Ultragenyx’s shares have outperformed the Zacks classified Medical-Biomed/Genetics industry so far this year. Shares of the company gained 11.4% while the industry registered an increase of 5.5%.
The company said that it will evaluate plans in the seizure indication. It, however, plans to study UX007 in phase III study in Glut1 DS patients with movement disorders.
Please note that UX007 is also being evaluated in a phase II study for the treatment of patients with long-chain fatty acid oxidation disorder (LC-FAOD). In Nov 2016, the company announced positive data from the study. Results demonstrated that treatment with UX007 reduced the frequency and duration of major medical events alongside improving participants' exercise tolerance and quality of life.
We remind investors that UX007 enjoys orphan drug status in the U.S. for the treatment of FAOD and Glut1 DS. The candidate was granted orphan drug status for the treatment of Glut1 DS in the EU as well.
Meanwhile, the company continues to progress with its pipeline. Currently, the company is evaluating aceneuramic acid extended release (Ace-ER) in a pivotal phase III study for the treatment of patients with GNE myopathy. Results are expected in the second half of 2017.
Ultragenyxcurrently carries a Zacks Rank #3 (Hold). Some better-ranked stocks in the health care sector include Addus HomeCare Corporation (ADUS - Free Report) , Alliqua BioMedical, Inc. and The Advisory Board Company . Each of these stocks carries a Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.
Addus HomeCare’s earnings per share estimates increased from $1.38 to $1.41 for 2017 over the last 30 days. The company posted positive earnings surprises in two of the four trailing quarters, with an average beat of 10.10%.
Alliqua’s loss per share estimates narrowed from 23 cents to 20 cents for 2018 over the last 30 days. The company posted positive earnings surprises in two of the four trailing quarters, with an average beat of 3.56%.
The Advisory Board’s earnings per share estimates increased from $1.41 to $1.49 for 2017 over the last 30 days. The company posted positive earnings surprises in three of the four trailing quarters with an average beat of 33.41%.
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Ultragenyx's Seizure Candidate UX007 Fails in Phase II Study
Ultragenyx Pharmaceutical Inc. (RARE - Free Report) recently announced that its pipeline candidate UX007 (triheptanoin) failed to meet the primary endpoint in a phase II study evaluating its safety and efficacy for the treatment of patients with glucose transporter type-1 deficiency syndrome (Glut1 DS) with seizures. Shares declined in after-market trading on Mar 23.
The randomized, double-blind, placebo controlled phase II study was conducted in a total of 36 patients. The study did not show reduction of the frequency of total number of observable and absence seizures among patients treated from baseline to week eight with UX007 compared with placebo. Patients treated with UX007 demonstrated a reduction of 13.4% in overall seizure frequency versus placebo.
Moreover, 14 patients discontinued evaluation in the study – two due to adverse events at week eight, four due to tolerability-related issues, and eight due to compliance or study burden.
Though the drug did show a reduction in absence seizures captured on EEG when each seizure type was evaluated independently, it did not reduce observable seizures.
Ultragenyx’s shares have outperformed the Zacks classified Medical-Biomed/Genetics industry so far this year. Shares of the company gained 11.4% while the industry registered an increase of 5.5%.
The company said that it will evaluate plans in the seizure indication. It, however, plans to study UX007 in phase III study in Glut1 DS patients with movement disorders.
Please note that UX007 is also being evaluated in a phase II study for the treatment of patients with long-chain fatty acid oxidation disorder (LC-FAOD). In Nov 2016, the company announced positive data from the study. Results demonstrated that treatment with UX007 reduced the frequency and duration of major medical events alongside improving participants' exercise tolerance and quality of life.
We remind investors that UX007 enjoys orphan drug status in the U.S. for the treatment of FAOD and Glut1 DS. The candidate was granted orphan drug status for the treatment of Glut1 DS in the EU as well.
Meanwhile, the company continues to progress with its pipeline. Currently, the company is evaluating aceneuramic acid extended release (Ace-ER) in a pivotal phase III study for the treatment of patients with GNE myopathy. Results are expected in the second half of 2017.
Ultragenyx Pharmaceutical Inc. Price
Ultragenyx Pharmaceutical Inc. Price | Ultragenyx Pharmaceutical Inc. Quote
Zacks Rank & Key Picks
Ultragenyxcurrently carries a Zacks Rank #3 (Hold). Some better-ranked stocks in the health care sector include Addus HomeCare Corporation (ADUS - Free Report) , Alliqua BioMedical, Inc. and The Advisory Board Company . Each of these stocks carries a Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.
Addus HomeCare’s earnings per share estimates increased from $1.38 to $1.41 for 2017 over the last 30 days. The company posted positive earnings surprises in two of the four trailing quarters, with an average beat of 10.10%.
Alliqua’s loss per share estimates narrowed from 23 cents to 20 cents for 2018 over the last 30 days. The company posted positive earnings surprises in two of the four trailing quarters, with an average beat of 3.56%.
The Advisory Board’s earnings per share estimates increased from $1.41 to $1.49 for 2017 over the last 30 days. The company posted positive earnings surprises in three of the four trailing quarters with an average beat of 33.41%.
More Stock News: This Is Bigger than the iPhone!
It could become the mother of all technological revolutions. Apple sold a mere 1 billion iPhones in 10 years but a new breakthrough is expected to generate more than 27 billion devices in just 3 years, creating a $1.7 trillion market.
Zacks has just released a Special Report that spotlights this fast-emerging phenomenon and 6 tickers for taking advantage of it. If you don't buy now, you may kick yourself in 2020. Click here for the 6 trades >>