Ultragenyx Pharmaceuticals (RARE) Ticks Upward on Phase 3 Trial Data

After the market close yesterday, Ultragenyx Pharmaceuticals Inc (NASDAQ:RARE) announced encouraging trial results from a Phase 3 study evaluating its treatment for X-linked hypophosphatemia (XLH) – a product called burosumab . At the time of writing in mid-afternoon trading, the company’s stock is up $1.60 and currently at $61.68.

According to Ultragenyx, patients who were given burosumab saw their phosphorus levels improve considerably. In fact, 94 percent of the patients being observed reached normal phosphorus levels at week 25 of the program – compared to just eight percent of the patients in the placebo group.

The study participants treated with burosumab also saw improvement in stiffness, as well as enhanced physical function and pain levels. Any adverse events observed in the first 24 weeks of the Phase 3 program were consistent with what had been seen in earlier research.

“These data demonstrate a clinical improvement in patients treated with burosumab and support the potential for treatment of adults,” said Ultragenyx’s President and Chief Executive Officer, Emil D. Kakkis, M.D., Ph.D. “When combined with a favorable safety profile and a strong serum phosphorus response, we believe these clinical data should support regulatory submissions in adults with XLH, and we look forward to discussing our filing plans with the U.S. FDA.”

Following the company’s announcement, Canaccord Genuity analyst Arlinda Lee restated a Buy rating on Ultragenyx’s common stock – reiterating a $100.00 price target. The upside indicated is about 62 percent.

Similarly, SunTrust Robinson Humphrey told investors this morning that it was reaffirming its buy rating and lifting its price target from $100.00 to $105.00. The firm said that the company’s programs have a high chance of success, and that the remainder of the year should have significant data catalysts.

Also this morning, Cowen & Company announced that it was reiterating its Outperform rating on Ultragenyx – saying that forecasts for burosumab’s Phase 2 pediatric results and Phase 3 adult data will support a broad label. The firm also projects $2.0 billion in global sales.

Not everyone was feeling as positive today, however. HC Wainwright’s Carol An Werther announced that she was downgrading Ultragenyx this morning from Buy to Neutral, and slashing her price target from $88.00 to $72.00. Werther told investors that the company’s shares are expected to stay range bound for the near-term, and that risk is elevated due to growing scrutiny on drug pricing and the extra data required for orphan disease programs.

“Therefore we prefer to stay on the sidelines until there is better clarity on the three leading programs KRN23, rhGUS, and Ace-ER,” the analyst added.

Wedbush also downgraded the company’s stock this morning from Outperform to Neutral – reducing its price target from $80.00 to $75.00. The firm stated that burosumab demonstrated a “lack of clear benefit in physical function and pain,” and that it has more gradual launch forecasts for the product.

The author of this article holds no position in any of the stocks mentioned above.

Tim is a contributing analyst for EconomicCalendar.com and specializes in equities trading and public offerings. He is a graduate of UCLA and began his career doing capital markets research for a top investment banking software provider.