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news.US based drug maker Ultragenyx Pharmaceutical has dosed first patient in a Phase 1 trial evaluating the safety of UX001 in patients with hereditary inclusion body myopathy (HIBM).
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The study aims to investigate the pharmacokinetics (PK) and safety profile of UX001 in around 24 patients.
The study expects to test four different single-dose levels in each group of six subjects.
Following that the subjects will undergo repeat dosing at three dose levels over 7 days to determine the steady-state pharmacokinetics and safety of repeat doses of UX001.
The company expects to release the data from the Phase 1 trial in 2011.
Ultragenyx CEO Emil Kakkis said advancing UX001 to the clinic is an important milestone for both Ultragenyx and for patients suffering with HIBM.