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Ultragenyx's (RARE) XLH Drug Positive in Phase II Study
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Ultragenyx Pharmaceutical Inc. (RARE - Free Report) and its Japanese partner Kyowa Kirin International plc recently announced positive 64-week data from a pediatric phase II study, evaluating its investigational candidate burosumab (KRN23). It is being developed for the treatment of X-linked hypophosphatemia (XLH) in children aged five to twelve years of age.
The multicentre, open-label, dose-finding phase II study (n=52) showed improvement in serum phosphorus levels, rickets, growth rates and other functional outcomes with use of KRN23, sustained through 64 weeks of treatment.
Additionally, a separate phase II study (n=13) evaluating KRN23 patients aged one to five years demonstrated that KRN23 increased serum phosphorus levels in the low normal range.
Ultragenyx’s share price movement shows that the stock has significantly underperformed the Zacks classified Medical-Biomedical/Genetics industry so far this year. Specifically, the stock lost 16.4% during this period compared with the industry’s increase of 2.8%.
Currently, KRN23 is being evaluated in a phase III study in adults with XLH. Data from the study are expected to be out in the first half of 2017. A biologics license application (BLA) for KRN23 for the above indication is expected to be filed in the U.S. in the second half of 2017.
In Jan 2017, European Medicines Agency (EMA) accepted the company’s Marketing Authorization Application (MAA) for KRN23 for the treatment of XLH in adults for review. The company expects an opinion from the EMA’s Committee for Medicinal Products for Human Use (CHMP) in the second half of this year.
We remind investors that in Jun 2016, the FDA had granted breakthrough therapy designation to KRN23 for the treatment of XLH in pediatric patients aged one year and above.
KRN23 is also being developed in a phase II study for the treatment of tumor-induced osteomalacia (TIO).
We note that Ultragenyx is making impressive progress with its pipeline candidates. The company expects to submit regulatory filings for rhGUS (UX003) for the treatment of mucopolysaccharidosis 7 (MPS 7) in both the U.S. and the EU in the first half of 2017.
Going forward, we expect investor focus to remain on the CHMP’s opinion on KRN23 and the company’s regulatory filing for rhGUS.
Ultragenyx currently carries a Zacks Rank #3 (Hold). Some better-ranked stocks in the health care sector are Heska Corporation , Galena Biopharma, Inc. and Infinity Pharmaceuticals, Inc. . Each of these stock sports a Zacks Rank #1 (Strong Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.
Heska’s earnings per share estimates increased from $1.53 to $1.65 for 2017 and from $1.90 to $2.01 for 2018, over the last 60 days. The company posted positive surprises in three of the four trailing quarters with an average beat of 291.54%.
Galena’s loss per share estimates narrowed from $2.03 to 58 cents 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 53.83%.
Infinity Pharma’s loss per share estimates narrowed from $1.43 to $1.03 for 2017 and from $1.75 to $1.52 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 36.64%.
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Ultragenyx's (RARE) XLH Drug Positive in Phase II Study
Ultragenyx Pharmaceutical Inc. (RARE - Free Report) and its Japanese partner Kyowa Kirin International plc recently announced positive 64-week data from a pediatric phase II study, evaluating its investigational candidate burosumab (KRN23). It is being developed for the treatment of X-linked hypophosphatemia (XLH) in children aged five to twelve years of age.
The multicentre, open-label, dose-finding phase II study (n=52) showed improvement in serum phosphorus levels, rickets, growth rates and other functional outcomes with use of KRN23, sustained through 64 weeks of treatment.
Additionally, a separate phase II study (n=13) evaluating KRN23 patients aged one to five years demonstrated that KRN23 increased serum phosphorus levels in the low normal range.
Ultragenyx’s share price movement shows that the stock has significantly underperformed the Zacks classified Medical-Biomedical/Genetics industry so far this year. Specifically, the stock lost 16.4% during this period compared with the industry’s increase of 2.8%.
Currently, KRN23 is being evaluated in a phase III study in adults with XLH. Data from the study are expected to be out in the first half of 2017. A biologics license application (BLA) for KRN23 for the above indication is expected to be filed in the U.S. in the second half of 2017.
In Jan 2017, European Medicines Agency (EMA) accepted the company’s Marketing Authorization Application (MAA) for KRN23 for the treatment of XLH in adults for review. The company expects an opinion from the EMA’s Committee for Medicinal Products for Human Use (CHMP) in the second half of this year.
We remind investors that in Jun 2016, the FDA had granted breakthrough therapy designation to KRN23 for the treatment of XLH in pediatric patients aged one year and above.
KRN23 is also being developed in a phase II study for the treatment of tumor-induced osteomalacia (TIO).
We note that Ultragenyx is making impressive progress with its pipeline candidates. The company expects to submit regulatory filings for rhGUS (UX003) for the treatment of mucopolysaccharidosis 7 (MPS 7) in both the U.S. and the EU in the first half of 2017.
Going forward, we expect investor focus to remain on the CHMP’s opinion on KRN23 and the company’s regulatory filing for rhGUS.
Ultragenyx Pharmaceutical Inc. Price
Ultragenyx Pharmaceutical Inc. Price | Ultragenyx Pharmaceutical Inc. Quote
Zacks Rank & Key Picks
Ultragenyx currently carries a Zacks Rank #3 (Hold). Some better-ranked stocks in the health care sector are Heska Corporation , Galena Biopharma, Inc. and Infinity Pharmaceuticals, Inc. . Each of these stock sports a Zacks Rank #1 (Strong Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.
Heska’s earnings per share estimates increased from $1.53 to $1.65 for 2017 and from $1.90 to $2.01 for 2018, over the last 60 days. The company posted positive surprises in three of the four trailing quarters with an average beat of 291.54%.
Galena’s loss per share estimates narrowed from $2.03 to 58 cents 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 53.83%.
Infinity Pharma’s loss per share estimates narrowed from $1.43 to $1.03 for 2017 and from $1.75 to $1.52 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 36.64%.
Zacks' Hidden Trades
While we share many recommendations and ideas with the public, certain moves are hidden from everyone but selected members of our portfolio services. Would you like to peek behind the curtain today and view them?
Starting now, for the next month, I invite you to follow all Zacks' private buys and sells in real time from value to momentum...from stocks under $10 to ETF to option movers...from insider trades to companies that are about to report positive earnings surprises (we've called them with 80%+ accuracy). You can even look inside portfolios so exclusive that they are normally closed to new investors. Click here for Zacks' secret trade>>