“The Orbit study builds on the findings of the 12-month Phase 2b 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
Ultragenyx is planning additional studies for setrusumab, including an open-label, randomized Phase 3 study in patients aged 2 to <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.
Orbit Study Design
The global, seamless Phase 2/3 Orbit study will evaluate the effect of setrusumab versus placebo on fracture rate in patients aged 5 to <26 years with OI sub-types I, III, and IV. In the Phase 2 portion, 36 patients will be randomized 1:1:1 to receive setrusumab at one of two doses or placebo to determine the optimal dosing strategy for Phase 3. The Phase 3 dosing strategy will be developed based on initial Phase 2 safety, pharmacokinetic, and bone biomarker data.
The pivotal Phase 3 portion of the study will include approximately 195 participants, randomized 2:1 to receive setrusumab or placebo, with a primary endpoint of fracture rate reduction. All participants will transition to an extension period and receive open-label setrusumab after the Phase 3 primary analysis is complete, or once a participant has completed 24 months in the double-blind period, whichever is sooner.
Further details can be referenced here: https://clinicaltrials.gov/ct2/show/NCT05125809
About Osteogenesis Imperfecta
Osteogenesis Imperfecta (OI) includes a group of genetic disorders impacting bone metabolism. Approximately 85% to 90% of OI cases are caused by mutations in the COL1A1 or COL1A2 genes, leading to either reduced or abnormal collagen and changes in bone metabolism. The collagen mutations in OI can result in increased bone brittleness, which contributes to a high rate of fractures, including at atypical sites. Patients with OI also exhibit increased bone resorption (breakdown of old bone) and inadequate production of new bone, which leads to decreased bone mass, bone fragility and weakness. OI can also lead to bone deformities including abnormal spine curvature, pain, decreased mobility, and short stature. No treatments are approved for OI, which affects approximately 60,000 people in the developed world.
About Setrusumab (UX143)
Setrusumab is a fully human monoclonal antibody that inhibits sclerostin, a protein that acts on a key bone-signaling pathway that inhibits the activity of bone-forming cells. The goal of blocking inhibitory effects of sclerostin is to increase new bone formation, bone mineral density, and bone strength. Sclerostin inhibition also reduces excessive bone resorption, further enhancing its impact on bone density. In various mouse models of OI, the use of anti-sclerostin antibodies was shown to stimulate bone formation, improve bone mass and density, and reduce bone fragility as reflected in increased long bone strength and reduced the number of fractures.
Mereo BioPharma’s Phase 2b study (ASTEROID) treatment phase of the dose-finding study of setrusumab for the treatment of OI in 112 adults was concluded in 2019. The ASTEROID study demonstrated 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. Off-treatment results were followed.
Ultragenyx and
About
Ultragenyx is a biopharmaceutical company committed to bringing novel products to patients for the treatment of serious rare and ultra-rare genetic diseases. The company has built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need and clear biology for treatment, for which there are typically no approved therapies treating the underlying disease.
The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyx’s strategy is predicated upon time- and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency.
For more information on Ultragenyx, please visit the company’s website at: www.ultragenyx.com.
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Except for the historical information contained herein, the matters set forth in this press release, including statements related to Ultragenyx’s expectations and projections regarding its business plans and objectives for UX143, the therapeutic potential and clinical benefits of UX143, expectations regarding the safety and tolerability of UX143, and future clinical developments for UX143 are forward-looking statements within the meaning of the “safe harbor” provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements involve substantial risks and uncertainties that could cause our clinical development programs, collaboration with third parties, future results, performance or achievements to differ significantly from those expressed or implied by the forward-looking statements. Such risks and uncertainties include, among others, the ability of the company and
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