First patient dosed in pivotal phase II/III Orbit clinical study of setrusumab for the treatment of osteogenesis imperfecta.
Ultragenyx Pharmaceutical Inc. announced the first patient has been dosed in a clinical study for the treatment of Osteogenesis Imperfecta (OI) in patients ages 5 to less than 26 years of age.
The pivotal Orbit study is a seamless Phase II/III study of setrusumab (UX143), an investigational anti-sclerostin fully human monoclonal antibody in development for the treatment of OI in children and adults with OI sub-types I, III and IV.
Ultragenyx is leading the clinical development of setrusumab as part of a collaboration and license agreement with Mereo BioPharma Group plc (MREO), a clinical-stage biopharmaceutical company focused on oncology and rare diseases.
“The Orbit study builds on the findings of the 12-month Phase IIb ASTEROID study, which demonstrated that treatment with setrusumab resulted in a clear, dose-dependent and statistically significant effect on bone formation and bone density at multiple anatomical sites among adult participants with OI,” said Camille Bedrosian, M.D., chief medical officer at Ultragenyx. “The Orbit Phase II/III study is designed to study fracture risk reduction in a younger population, where the risk of fracture is higher and where we can potentially have the greatest impact on their future health.”
Ultragenyx is planning additional studies for setrusumab, including an open-label, randomized Phase III study in patients aged 2 to less than 5 years evaluating setrusumab versus bisphosphonates for the treatment of OI over a period of 12 to 24 months that is anticipated to start in the second half of 2022. The company also plans to continue to study setrusumab in adult patients over the age of 25 with OI.
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