First patients dosed in phase III program evaluating setrusumab for the treatment of osteogenesis imperfecta
Ultragenyx Pharmaceutical Inc. 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 III portion of the pivotal Phase II/III 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 III 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.
"I am extremely encouraged by the recent data from the Phase II portion of the Orbit study, which includes improvement in biochemical markers and bone density measures that reflect the clinical response we have observed in study participants," stated Thomas Carpenter, M.D., professor of Pediatrics (Endocrinology) and of Orthopaedics and Rehabilitation, Yale School of Medicine. "We are looking forward to evaluating the full clinical potential of setrusumab as this important Phase III program moves forward, with hopes for improving therapeutic outcomes in OI."
“Data from the Phase II portion of the Orbit study demonstrated increases in bone formation and bone mineral density, which are important markers of bone strength, as well as early indications of improved bone health from our investigators,” said Eric Crombez, M.D., chief medical officer at Ultragenyx. “Our comprehensive Phase III program is designed to study the impact of setrusumab on clinical fracture risk reduction. The two Phase III trials will evaluate patients over a broad age range, including the younger pediatric population, where the risk of fracture is higher and where we can potentially have the greatest impact on their future health.”
Ultragenyx is leading the clinical development of setrusumab as part of a collaboration and license agreement with Mereo BioPharma Group plc a clinical-stage biopharmaceutical company focused on rare diseases. The companies recently announced positive data from the dose-selection Phase II portion of the Orbit study showing that setrusumab rapidly induced bone production in OI-affected patients.
The Setrusumab Phase III Program: The global, seamless Phase II/III Orbit study is evaluating the effect of setrusumab compared to placebo on clinical fracture rate in patients aged 5 to less than 26 years. In the Phase II portion, 24 patients were randomized 1:1 to receive setrusumab at one of two doses to determine the optimal dosing strategy for Phase III. The pivotal Phase III portion of the study will include approximately 195 patients at more than 40 sites across 12 countries, randomized 2:1 to receive setrusumab or placebo, with a primary efficacy endpoint of annualized clinical fracture rate. All patients will transition to an extension period and receive open-label setrusumab after the Phase III primary analysis is complete.
The global Phase III Cosmic study is an open-label, randomized, active-controlled study in patients aged 2 to less than 5 years evaluating setrusumab compared to intravenous bisphosphonates (IV-BP) therapy on reduction in total fracture rate, including morphometric vertebral fractures. The Cosmic study will enroll approximately 65 patients at more than 20 sites across 8 countries.
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