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Investing.com -- Ultragenyx Pharmaceutical (TADAWUL:2070) Inc. (NASDAQ:RARE) stock plummeted 33% after the company announced its Phase 3 Orbit study for UX143 (setrusumab) in osteogenesis imperfecta patients will continue to final analysis rather than stopping early for efficacy.
Mereo BioPharma Group plc (NASDAQ:MREO), Ultragenyx’s partner in developing the treatment, saw its shares sink 40% following the news. The companies revealed that the Data Monitoring Committee reviewed interim data and recommended the study continue to its planned conclusion around year-end, indicating the results did not meet the threshold for early stoppage.
"Based on the feedback we hear from investigators and families who participated in the studies, we are confident that increasing bone mass leads to stronger bone, less fractures, and improved physical abilities," said Emil D. Kakkis, M.D., Ph.D., chief executive officer and president of Ultragenyx. "While we had hoped to be able to stop the study early, we look forward to having results from both Orbit and Cosmic around the end of this year."
The Phase 3 Orbit study is evaluating UX143 in pediatric and young adult patients with osteogenesis imperfecta, a rare genetic disorder characterized by fragile bones. According to the announcement, the DMC confirmed that UX143 demonstrates an acceptable safety profile.
The companies noted that data from the companion Cosmic study, which examines the drug in younger patients, were not analyzed at this interim timepoint. Both studies will continue with final analyses planned after patients have been on therapy for at least 18 months.
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