Ultragenyx and Mereo advance UX143 osteogenesis imperfecta study to final analysis

Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) and Mereo BioPharma Group plc (NASDAQ: MREO) announced that their Phase 3 Orbit study evaluating UX143 (setrusumab) in pediatric and young adult patients with osteogenesis imperfecta will continue to final analysis, expected around the end of the year.

The Data Monitoring Committee reviewed the study and determined that UX143 demonstrates an acceptable safety profile, recommending the companies continue the study to its planned conclusion. The committee did not recommend early termination based on efficacy.

The randomized, placebo-controlled Phase 3 portion of the Orbit study enrolled 159 patients at 45 sites across 11 countries. Subjects were randomized 2:1 to receive setrusumab or placebo, with the primary endpoint measuring annualized clinical fracture rate. The threshold for statistical significance in the final analysis is p<0.04.

Data from the companion Cosmic study, which enrolled 69 patients aged 2 to under 7 years at 21 sites across 7 countries, were not analyzed at this interim timepoint. The Cosmic study compares setrusumab to intravenous bisphosphonates therapy and requires p<0.05 for statistical significance.

Both studies will conduct final analyses after patients complete at least 18 months of therapy. All patients will transition to receive open-label setrusumab after the Phase 3 primary analysis.

Setrusumab is a monoclonal antibody that inhibits sclerostin, a protein that regulates bone formation. The drug has received orphan designation from the European Commission and FDA, along with breakthrough therapy designation from the FDA for osteogenesis imperfecta.

Osteogenesis imperfecta affects approximately 60,000 people in commercially accessible regions and has no globally approved treatments. The condition causes bone brittleness and frequent fractures due to genetic variants affecting collagen production.

The information is based on a press release statement from the companies.

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