Clinical-stage biotechnology company Ultragenyx Pharmaceutical has reported positive interim data from Phase II study of an oral sialic acid extended-release (SA-ER) tablet, UX001, in hereditary inclusion body myopathy (HIBM).

The 24-week interim data reported dose-dependent improvement in muscle strength compared to placebo in some muscle groups, especially in the upper extremities at the 6gm dose.

Go deeper with GlobalData

Data Insights

The gold standard of business intelligence.

Find out more

The changes, which were statistically significant or trended towards significance, were more distinct in subjects with greater walking ability at baseline, while no changes were observed in other clinical endpoints at the interim assessment.

Ultragenyx chief executive officer Emil Kakkis said that early data indicates a modest dose-dependent on an improvement in muscle strength in HIBM patients treated with UX001 compared to a decline in placebo-treated patients.

"The 24-week interim data reported dose-dependent improvement in muscle strength compared to placebo in some muscle groups, especially in the upper extremities at the 6gm dose."

"We need to evaluate whether the observed treatment effect is sustained or increased over a longer 48-week period, and if higher dosing might further enhance the efficacy signal observed," Kakkis added.

According to the data, UX001 was well tolerated and 6gm dose group of the investigational agent showed a drift to improvement in creatine kinase levels compared to placebo.

How well do you really know your competitors?

Access the most comprehensive Company Profiles on the market, powered by GlobalData. Save hours of research. Gain competitive edge.

Company Profile – free sample

Thank you!

Your download email will arrive shortly

Not ready to buy yet? Download a free sample

We are confident about the unique quality of our Company Profiles. However, we want you to make the most beneficial decision for your business, so we offer a free sample that you can download by submitting the below form

By GlobalData
Visit our Privacy Policy for more information about our services, how we may use, process and share your personal data, including information of your rights in respect of your personal data and how you can unsubscribe from future marketing communications. Our services are intended for corporate subscribers and you warrant that the email address submitted is your corporate email address.

Comparing the total daily doses of 6gm or 3gm of UX001, the Phase II study’s primary objective is to evaluate safety, dose and potential pharmacodynamic effect of restoring sialylation of muscle in patients with a confirmed genetic mutation for HIBM.

Additionally, clinical measures of muscle strength, mobility, function, self-reported disability, and changes in quality of life are also being assessed.

Based on the dose-dependence observed at week 24, the company plans to treat patients in an extension study, following the 48-week analysis with an increased UX001 dosage.

The final 48-week data is anticipated by the end of 2013.