Ultragenyx Files BLA To FDA For Hypophosphatemia Candidate

Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) along with Japanese partner Kyowa Hakko Kirin Co., Ltd announced filing of a biologics license application (BLA) to the FDA for its pipeline candidate burosumab (KRN23) to treat patients with X-linked hypophosphatemia (XLH). The FDA informed that a decision on acceptance of the same will be made within 60 days.

Notably, in January, European Medicines Agency (EMA) had accepted the company’s marketing application for burosumab for review. The company now expects an opinion from the EMA’s Committee for Medicinal Products for Human Use (CHMP) in the second half of this year.

Shares of the company have inched up almost 1% following the news release. However, Ultragenyx’s share price movement shows that the stock has underperformed the industry so far this year. Specifically, the stock plunged 26.8% as against the industry’s 8% gain.

The BLA submission was supported by positive data from a phase III study on adults and a phase II trial on pediatric patients for the aforementioned indication. The data was announced in April. The 24-week phase III study met the primary endpoint as the patients treated with burosumab achieved a statistically significant improvement in serum phosphorus levels compared with placebo. Further, the secondary endpoint of the study was also met as patients administered with burosumab attained a substantial improvement in stiffness as well as witnessed a strong progress in physical function and pain.

On the other hand, the 64-week data from the pediatric phase II study showed better results in serum phosphorus levels, rickets, growth rates and other functional outcomes with use of burosumab, sustained through a prolonged period of treatment.

In fact, in June 2016, the FDA had granted a therapy designation to burosumab for treatment of XLH in pediatric patients aged one year and above.

Currently, burosumab is also being evaluated in a phase II study for treating tumor-induced osteomalacia (TIO).

Importantly, Ultragenyx is making a significant progress with other pipeline candidates as well. Another candidate rhGUS (UX003) is under review in both the U.S. and the EU for treatment of mucopolysaccharidosis 7 (MPS 7). The company expects a response from the FDA by this year-end and the EU’s feedback is awaited in the first half of 2018.

In the near term, we expect investors’ focus to remain on the opinion of Committee for Medicinal Products for Human Use (CHMP) on burosumab and for regulatory responses to rhGUS.

Zacks Rank & Stocks to Consider

Ultragenyx currently carries a Zacks Rank #3 (Hold). Some better-ranked stocks in the pharma sector are Aerie Pharmaceuticals, Inc. (NASDAQ:AERI) , Aduro Biotech, Inc. (NASDAQ:ADRO) and ACADIA Pharmaceuticals Inc. (NASDAQ:ACAD) , all three carrying a Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.

Aerie’s loss per share estimates narrowed from $2.62 to $2.53 for 2017 over the last 30 days. The company delivered a positive earnings surprise in all three of the trailing four quarters with an average beat of 0.64%. Its share price soared 39.7% so far this year.

Aduro Biotech’s loss per share estimates reduced from $1.46 to $1.32 for 2017 and from $1.41 to $1.24 over the last 30 days. The company delivered positive surprises in two of the trailing four quarters with an average beat of 2.53%.

ACADIA’s loss per share estimates narrowed from $2.82 to $2.59 for 2017 and from $2.07 to $1.92 for 2018 over the last 30 days. The company came up with positive earnings surprises in two of the last four quarters with an average beat of 7.97%.

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