Ultragenyx Pharmaceutical Inc., a biotechnology company focused on developing treatments for rare and ultra-rare genetic disorders, today announced that the Phase 1 study of UX001 for hereditary inclusion body myopathy (HIBM) has been completed. UX001 is an extended release formulation of sialic acid intended as a substrate replacement therapy for HIBM, a severe, neuromuscular disease caused by sialic acid deficiency. UX001 is the first program from the company's pipeline to enter the clinic since its founding in 2010. The FDA Office of Orphan Products Development has granted orphan drug designation for UX001 for the treatment of HIBM.
"Ultragenyx continues to make excellent progress in advancing and expanding our clinical development pipeline with the completion of our first clinical study," said Emil D. Kakkis, MD, PhD, Chief Executive Officer of Ultragenyx. "We deeply appreciate the participation and enthusiastic support of the HIBM patient community for our Phase 1 study. We anticipate releasing the results from this study within the next couple of months, and will look forward to presenting the data at a scientific meeting in the fall. We plan to initiate a Phase 2 trial of SA-ER in patients with HIBM in the second quarter of this year."
The Phase 1 clinical study evaluated the pharmacokinetics (PK) and safety of UX001 in 28 HIBM patients. The study tested five different single-dose levels in each group of six subjects. Subjects then underwent repeat dosing at four dose levels over 7 days to establish the steady-state pharmacokinetics and safety of repeat doses of UX001. Ultragenyx anticipates data from the Phase 1 study in April 2012.
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