Ultragenyx and Mereo have recently announced new Phase 2 data from the Phase 2/3 Orbit study (NCT05125809). This global study is evaluating the safety and efficacy of a potential new therapy on clinical fracture rate in people with OI aged 5 to 25 years.
- Reduced incidence of fractures in people with OI during the study period, compared with the two previous years
- Continued, clinically meaningful increases in lumbar spine bone mineral density (BMD)
Despite these encouraging signals, it is important to remember that, at this time, the safety and efficacy of the therapy have not yet been established and the continued clinical trials will be critical in answering these questions before the potential therapy can be submitted for regulatory review and eventual approval.
What happens next?
Orbit and Cosmic studies are fully enrolled, the initial Phase 3 results from both studies are expected next year.