Ultragenyx doses first patient in Phase 3 Osteogenesis Imperfecta trial

Ultragenyx (RARE) announced that the first patients have been dosed in both of its late-stage clinical trials evaluating setrusumab in pediatric and young adult patients with OI sub-types I, III and IV. The Phase 3 portion of the pivotal Phase 2/3 Orbit study is evaluating the effect of setrusumab compared to placebo on annualized clinical fracture rate in patients aged 5 to less than 26 years. The newly initiated Phase 3 Cosmic study is an active-controlled study evaluating setrusumab compared to intravenous bisphosphonate (IV-BP) therapy on annualized total fracture rate in patients aged 2 to less than 5 years. Ultragenyx is leading the clinical development of setrusumab as part of a collaboration and license agreement with Mereo BioPharma (MREO). The companies recently announced positive data from the dose-selection Phase 2 portion of the Orbit study showing that setrusumab rapidly induced bone production in OI-affected patients.