Ultragenyx (RARE) Reports Positive Phase III RhGUS Data

Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) announced positive top-line data from a pivotal phase III study on recombinant human beta-glucuronidase (rhGUS) for the treatment of mucopolysaccharidosis 7 (MPS 7), also known as Sly syndrome.

The study assessed the safety and efficacy of rhGUS in patients (n=12) aged 5–35 years.

Data demonstrated a rapid and sustained reduction of 64.8% from baseline in urinary GAG (dermatan sulfate) excretion after 24 weeks of treatment, thereby meeting the primary endpoint. It also provided evidence of clinical improvement. Treatment-emergent adverse events were generally mild to moderate in severity. Results from the study are being presented at the International Symposium on MPS and Related Diseases.

Ultragenyx intends to meet regulatory authorities in both the U.S. and the EU to discuss plans to submit regulatory filings in the first half of 2017.

We note that MPS 7 represents an underserved market with no approved therapies presently available. A potential approval and successful commercialization of rhGUS will thus be a major boost for the company.

Drugs currently approved for the treatment of other MPS disorders include Aldurazyme, Elaprase, Vimizim and Naglazyme.

Meanwhile, Ultragenyx continues to progress on its pipeline. Currently, the company is evaluating aceneuramic acid extended release (Ace-ER) in a pivotal phase III study for the treatment of patients with GNE myopathy. Results are expected in 2017.

Moreover, the company has filled a marketing application for Ace-ER in the EU, based on phase II results for the treatment of adult patients with GNE myopathy. A response from the European Medicines Agency’s (EMA) Committee for Orphan Medicinal Products for Human Use (CHMP) is expected in the second half of 2016.

We expect investor focus to remain on further pipeline updates from the company.

Currently, Ultragenyx carries a Zacks Rank #3 (Hold). Some better-ranked stocks in the health care sector include Merrimack Pharmaceuticals, Inc. (NASDAQ:MACK) , Pfizer Inc. (NYSE:PFE) and Innoviva, Inc. (NASDAQ:INVA) . Each of these stock sports a Zacks Rank #1 (Strong Buy).

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