- In January 2024, Bone Therapeutics initiated a Phase I/IIa clinical trial for its allogeneic cell therapy product, ALLOB, targeting patients with osteogenesis imperfecta. The trial is designed to evaluate ALLOB's efficacy in improving bone strength and reducing fracture risk in affected individuals. This trial represents a pivotal advancement in the pursuit of innovative treatments aimed at enhancing outcomes for osteogenesis imperfecta patients
- In March 2023, Mereo BioPharma Group plc announced positive topline results from its Phase 2b ASTER study evaluating setrusumab, a novel anti-sclerostin antibody, in adults with osteogenesis imperfecta. The study showed promising safety and efficacy, positioning setrusumab as a potential therapeutic breakthrough for this rare bone disorder
- In July 2023, Ultragenyx Pharmaceutical Inc. reported the treatment of the first patients in its late-stage clinical trials for setrusumab, targeting pediatric and young adult patients with osteogenesis imperfecta types I, III, and IV. The Phase 3 portion of the pivotal Phase 2/3 Orbit study is comparing setrusumab to placebo on fracture rates in patients aged 5 to under 26 years. In addition, the Phase 3 Cosmic study is underway, evaluating setrusumab versus intravenous bisphosphonate therapy in children aged 2 to under 5 years
- In October 2022, The Osteogenesis Imperfecta Federation Europe (OIFE) formally re-established itself as a new legal entity based in Belgium, following the dissolution of its previous registration in the Netherlands. This organizational restructuring is intended to streamline operations and enhance advocacy efforts, ultimately strengthening patient support and awareness initiatives throughout Europe
- In October 2021, Mereo BioPharma Group plc, in partnership with the Osteogenesis Imperfecta Federation Europe (OIFE) and the Osteogenesis Imperfecta Foundation (OIF), completed enrollment for the IMPACT Survey — the largest global data collection on osteogenesis imperfecta’s effects on patients, families, and caregivers. Gathering over 2,200 responses from approximately 65 countries in just three months, the survey’s findings are set to guide future collaborative efforts to improve diagnosis, treatment, and care, as well as accelerate new therapy availability
