Ultragenyx and Mereo to continue Phase 3 trials of OI treatment to completion

NOVATO, Calif. - Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) and Mereo BioPharma Group plc (NASDAQ:MREO) announced Wednesday that their Phase 3 trials evaluating UX143 (setrusumab) for osteogenesis imperfecta (OI) will continue to final analysis, expected around the end of 2025. Mereo, currently valued at approximately $450 million, has attracted significant analyst attention, with targets ranging from $6.05 to $10.06 per share, according to InvestingPro data.

The Data Monitoring Committee reviewed interim data from the Orbit study, which is testing the drug in patients aged 5 to 25 years, and determined that UX143 demonstrates an acceptable safety profile. The committee recommended continuing the study to completion rather than stopping early. While Mereo isn’t yet profitable, InvestingPro analysis shows the company maintains a strong financial position with a healthy current ratio of 8.92 and more cash than debt on its balance sheet.

The Orbit study has enrolled 159 patients across 45 sites in 11 countries, with subjects randomized to receive either setrusumab or placebo. The parallel Cosmic study, which is testing the drug in children aged 2 to under 7 years, was not analyzed at this interim timepoint but will also continue to final analysis.

"Patients will continue dosing in the ongoing Phase 3 Orbit and Cosmic clinical studies with the final analyses to be conducted after patients have been on therapy for at least 18-months," the companies stated in their press release.

Setrusumab is a fully human monoclonal antibody that inhibits sclerostin, a protein that negatively regulates bone formation. The drug is designed to increase bone formation, density and strength in OI patients, a genetic disorder that causes bone fragility and high fracture rates affecting approximately 60,000 people globally.

The Orbit study’s primary endpoint is measuring the annualized clinical fracture rate, while the Cosmic study compares setrusumab to intravenous bisphosphonates therapy.

No treatments are currently globally approved for OI, which is characterized by increased bone brittleness, inadequate bone production, and excessive bone resorption. For deeper insights into Mereo’s financial health and growth potential, including 7 additional key ProTips and comprehensive biotech sector analysis, visit InvestingPro, where you’ll find detailed research reports covering 1,400+ US stocks.

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