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Key Takeaways RARE and MREO fell after delaying new data from the phase III UX143 Orbit study to year-end. The Orbit study continues as planned, with final data expected by the end of 2025. Interim safety review found UX143 acceptable, allowing studies to proceed without changes.Ultragenyx Pharmaceutical (RARE) and partner, Mereo BioPharma (MREO) , have reported an update on the progress of the mid-to-late-stage Orbit study, which is evaluating UX143 (setrusumab) for the treatment of osteogenesis imperfec ...

Data from Orbit and Cosmic studies expected around the end of the yearNOVATO, Calif., July 09, 2025 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) and Mereo BioPharma Group plc (NASDAQ: MREO), today announced that the randomized, placebo-controlled Phase 3 portion of the Orbit study evaluating UX143 (setrusumab) in pediatric and young adult patients with osteogenesis imperfecta (OI) is progressing toward a final analysis consistent with the original plan, around the end of the year. The D ...

Key Takeaways The FDA granted Breakthrough Therapy designation to Ultragenyx's GTX-102 for Angelman syndrome. GTX-102 showed rapid, lasting improvements in AS patients during a phase I/II study over up to 3 years. RARE is enrolling for a phase III Aspire study and planning a broader phase II/III Aurora study for GTX-102.Ultragenyx Pharmaceuticals (RARE) announced that the FDA has granted the Breakthrough Therapy designation to its investigational antisense oligonucleotide therapy, GTX-102 (apazunersen), t ...

Progress continues in Phase 3 Orbit study of setrusumab in osteogenesis imperfecta (OI) Cash of $62.5 million as of March 31, 2025, expected to fund operations into 2027 LONDON, May 13, 2025 (GLOBE NEWSWIRE) -- Mereo BioPharma Group plc (NASDAQ: MREO) (“Mereo” or the “Company”), a clinical-stage biopharmaceutical company focused on rare diseases, today announced its financial results for the first quarter ended March 31, 2025, and provided recent corporate highlights. “As we close out the first quarter of 2 ...

Corporate Presentation May 2025 Confidential and Proprietary Forward looking statements Cautionary note regarding forward-looking statements: This presentation contains forward-looking statements, including, but not limited to, statements regarding our expectations, estimates, assumptions, and projections regarding our future operating results and financial performance, including our expectations for profitability in 2027, anticipated cost or expense management, plans with respect to commercializing our pro ...