Ultragenyx Pharmaceutical Inc. | $RARE Stock | Shares Rise Up On Positive Results from Phase 3 Study

Ultragenyx Pharmaceutical

Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE)



Ultragenyx Pharmaceutical Inc. (RARE), a clinical-stage biopharmaceutical company yesterday announced positive results from its phase 3 study of burosumab in adults with X-linked hypophosphatemia.

The study showed that burosumab showed a statistically significant improvement over the placebo, with 94% of the patients taking burosumab achieving normal phosphorus levels compared to 8% of patients taking the placebo.   


Ultragenyx Pharmaceutical Inc. CEO’s Comments

“These data demonstrate a clinical improvement in patients treated with burosumab and support the potential for treatment of adults,” said Emil D. Kakkis, M.D., Ph.D., Chief Executive Officer and President of Ultragenyx. “When combined with a favourable 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.”  Globe Newswire


RARE Technical Analysis

Ultragenyx Pharmaceutical

RARE opened trading yesterday at $60.92 which was down from the previous day’s trading close of $61.45. RARE closed trading yesterday at $60.08 and spiked up after market to $71.45, equivalent to a 19% increase from the closing price. Taking a look at the daily chart we can see that the last time RARE traded above these levels we have to go back to March 23rd when it traded at $71.99.

Taking a closer look at the daily chart we can see that before the spike up RARE had been in an overall downward trend dating back to March 15th when it traded at $88.11. RARE has a float of 38.58 million shares and traded below the normal daily trading volume on Tuesday.

For trading purposes, I would like to see RARE open trading on Wednesday above $67.75 and if it does I would be looking to take a long position at the bell. My stop loss would be $0.40 from my entry position fearing anything more than that and the stock would start to fill in the gap up.


Company Profile

Ultragenyx Pharmaceutical Inc., a clinical-stage biopharmaceutical company, focuses on the identification, acquisition, development, and commercialization of various products for the treatment of rare and ultra-rare genetic diseases in the United States. The company is developing various biologics product candidates, including KRN23, a human monoclonal antibody that in Phase III adult study to bind and reduce the biological activity of fibroblast growth factor 23 to enhance abnormally low phosphate levels in patients with X-linked hypophosphatemia, as well as for the treatment of tumor-induced osteomalacia.

Its biologics product candidates also comprise recombinant human beta -glucuronidase (rhGUS), an enzyme replacement therapy, which completed the Phase 3 clinical study for the treatment of mucopolysaccharidosis 7; and recombinant human protective protein cathepsin-A (rhPPCA), an enzyme replacement therapy, which is in preclinical development for galactosialidosis.

The company is also developing range of small-molecule product candidates, such as UX007, a substrate replacement therapy that is in Phase II study for patients with fatty acid oxidation disorders, as well as for patients with glucose transporter type-1 deficiency syndrome; and aceneuramic acid extended-release (Ace-ER), an oral formulation of sialic acid, which is in Phase III extension study to treat GNE myopathy.

It has collaboration and license agreements with Arcturus Therapeutics, Inc.; Takeda Pharmaceutical Company Limited; Kyowa Hakko Kirin Co. Ltd.; Saint Louis University; Baylor Research Institute; Nobelpharma Co., Ltd.; Alcami Corporation; HIBM Research Group; and St. Jude Children’s Research Hospital. The company was founded in 2010 and is headquartered in Novato, California.”  Yahoo Finance