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Ultragenyx Reports Data from Sialic Acid Deficiency Study

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Investors in the pharma/biotech sector eagerly wait for pipeline updates as they play an important role in deciding whether or not to invest in a particular company. These updates provide information on experimental drugs and at times give an insight into the commercial potential of a candidate once it is successfully developed and commercialized.

Earlier this week, Ultragenyx Pharmaceutical Inc. (RARE) announced interim data from a phase II extension study on sialic acid extended-release (SA-ER, UX001) tablets. The data was presented at the International Congress of the World Muscle Society.

Ultragenyx is evaluating SA-ER for the treatment of hereditary inclusion body myopathy (:HIBM), which is also known as GNE myopathy, in patients suffering from sialic acid deficiency.

Interim results from the extension study revealed that the 12 gram per day dose did not show superiority over the 6 gram per day dose at 24 weeks. However, the 12 gram per day dose demonstrated activity and safety in terms of a possible long-term impact on disease progression in upper extremity muscle strength as compared to the 24-week placebo group after approximately two years of treatment.

We remind investors that Ultragenyx had announced results from the phase II study on SA-ER in April this year. Results from the study demonstrated a statistically significant improvement in the upper extremity composite (UEC) of muscle strength at 48 weeks when treated with the higher dose group (6 grams/day) as compared to the lower dose group (3 grams/day).

On the back of encouraging 48-week and interim extension study data, Ultragenyx plans to initiate a pivotal study on SA-ER. Ultragenyx also intends to continue with the extension study.

According to the press release issued by Ultragenyx, there are nearly 1,200 to 2,000 HIBM patients in the developed world alone. Currently, there is no approved therapy for this disease.

We note that apart from SA-ER, Ultragenyx has another candidate in its substrate replacement therapy pipeline, triheptanoin or UX007 (treatment of long-chain fatty acid oxidation disorders and glucose transporter type-1 deficiency syndrome). The candidate is presently in phase II studies.

Ultragenyx currently carries a Zacks Rank #4 (Sell). Some better-ranked stocks in the health care sector include Emergent BioSolutions, Inc. (EBS), Halozyme Therapeutics, Inc. (HALO) and Ligand Pharmaceuticals Incorporated (LGND). All three carry a Zacks Rank #1 (Strong Buy).

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