Ultragenyx Announces Completion of Phase 2 Study Enrollment for UX001 in Hereditary Inclusion Body Myopathy
10/30/2012 6:52:18 AM
NOVATO, Calif., Oct. 30, 2012 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc., a biotechnology company focused on developing treatments for rare and ultra-rare genetic disorders, today announced it has completed patient enrollment in a randomized, double-blind, placebo-controlled Phase 2 study of UX001 in patients with hereditary inclusion body myopathy (HIBM). The study's primary objective is to evaluate safety, dose, and improvements in sialylation biochemistry of muscle from pre-treatment to post-treatment biopsies (pharmacodynamic endpoint). Muscle strength, clinical function and patient-reported outcomes will also be evaluated, though the study is not powered for these endpoints. A total of 45 patients between 18 and 65 years of age, with a confirmed genetic mutation for HIBM, have been randomized to placebo versus two dose levels of active drug at four clinical sites in the US and Israel.