The Next Steps For Ultragenyx' Burosumab Following Positive Phase 3 Data

Shares of Ultragenyx Pharmaceutical Inc RARE, a clinical-stage bio-pharmaceutical company, were trading higher by more than 7 percent Wednesday in reaction to an encouraging update regarding its Burosumab therapy which is used by patients with X-linked hypophosphatemia (XLH) in a Phase 3 study.

Ultragenyx said Tuesday that patients with burosumab demonstrated a statistically significant improvement in serum phosphorus levels, with 94 percent of patients achieving normal levels compared to 8 percent on placebo.

Baird Reiterates At Outperform

Michael Ulz of Baird maintained an Outperform rating on Ultragenyx's stock with a $90 price target after the company's announcement.

Ulz highlighted the fact that Ultragenyx now holds positive Phase 3 data in its hand and will meet with the U.S. Food and Drug Administration. The analyst added that the company's filing in pediatric (based on Phase 2 data) and now adults in the latest Phase 3 data remains on track for the bottom half of 2017.

Ulz believes Ultragenyx now faces a "meaningful" market opportunity of at least $1 billion in patients with XLH.

Ulz also noted burosumab remains under regulatory approval in Europe and an opinion from the regulatory authority is expected in the second half of 2017.

Meanwhile, data from the company's biopsy study (48 weeks) is expected to come by the end of 2018 and data from its Phase 3 pediatric XLH study is expected at some point in 2018 as well.

The analyst's $90 price target is based on a sum-of-the-parts net present value of various therapy products on sales in 2029.

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