Core Viewpoint - The Portnoy Law Firm has initiated an investigation into Ultragenyx Pharmaceutical, Inc. for possible securities fraud following the failure of its clinical trials, which has led to a significant drop in the company's stock price [1][3]. Group 1: Company Performance - On December 29, 2025, Ultragenyx announced that its Phase III Orbit and Cosmic studies for setrusumab (UX143) did not meet their primary endpoint of reducing annualized clinical fracture rates compared to placebo and biophosphonates [3]. - Although the studies showed improvements in secondary endpoints related to bone material density, these did not correlate with a statistically significant reduction in fractures [3]. - Following the announcement, Ultragenyx's stock price fell by $14.87 (43.49%) to open at $19.32 per share, marking a new 52-week low, significantly below the previous low of $25.81 [3]. Group 2: Legal Actions - The Portnoy Law Firm is representing investors in pursuing claims related to corporate wrongdoing, and has a history of recovering over $5.5 billion for aggrieved investors [3]. - Investors are encouraged to contact the firm for a complimentary case evaluation and to discuss their legal rights regarding potential claims [2].

Core Insights - Shares of Ultragenyx Pharmaceutical (RARE) and Mereo BioPharma (MREO) fell significantly by 42.3% and 87.6% respectively after disappointing results from phase III studies evaluating setrusumab for osteogenesis imperfecta treatment [1][2] Study Results - The Orbit and Cosmic studies did not achieve statistical significance for their primary endpoints related to annualized clinical fracture rate compared to placebo or bisphosphonates [2][7] - Both studies showed statistically significant improvements in bone mineral density (BMD) as a secondary endpoint, with no changes in safety profiles [2][9] - In the Orbit study, participants had meaningful BMD improvements, but these did not lead to a reduction in fracture rates [9] - The Cosmic study indicated that while BMD improvements were observed in a younger patient population, the reduction in fracture rates compared to bisphosphonates was not statistically significant [10] Company Actions - Ultragenyx is reviewing its operations and will implement significant cost-reduction measures following the study results [3][7] - The company is conducting additional analyses on the data from both studies to explore other bone health and clinical endpoints beyond fractures [10] Stock Performance - Over the past six months, Ultragenyx shares have declined by 45.7%, while Mereo BioPharma shares have dropped by 89.7%, contrasting with a 25.9% increase in the industry [4]

Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) shares plunged 42% on Monday after the company and partner Mereo BioPharma Group plc (NASDAQ:MREO) disclosed disappointing late-stage data for their rare bone disease program.Mereo BioPharma Group plc and Ultragenyx revealed results from the Phase 3 Orbit and Cosmic studies for setrusumab (UX143) in Osteogenesis Imperfecta (OI).The companies said that neither study achieved statistical significance against the primary endpoints of reduction in annualized clinical ...

Major U.S. indices closed lower on Monday, with the Dow Jones Industrial Average slipping 0.5% to 48,461.93. The S&P 500 declined 0.35% to 6,905.74, while the Nasdaq fell 0.5% to 23,474.34.These are the top stocks that gained the attention of retail traders and investors throughout the day:Brand Engagement Network Inc. (NASDAQ:BNAI)Brand Engagement Network stock surged by 108.75%, closing at $2.50. It reached an intraday high of $2.91 and a low of $1.77, with a 52-week range between $11.80 and $1.18. In the ...

A pharmaceutical company's success with a Phase 2 study doesn't guarantee positive results in Phase 3.Heading into 2026 on an inauspicious note, Ultragenyx Pharmaceuticals (RARE 43.70%), a pharmaceutical company specializing in treatments for rare and ultra-rare genetic diseases, announced disappointing results from its Phase 3 studies for setrusumab (UX143). Add in a flurry of analysts slashing their price targets on Ultragenyx stock, and it's clear why investors are clicking the sell button today.As of 2: ...

Shares of Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) fell sharply on Monday.Mereo BioPharma and its partner Ultragenyx revealed results from the Phase 3 Orbit and Cosmic studies for setrusumab (UX143) in Osteogenesis Imperfecta (OI). Osteogenesis Imperfecta (OI) includes a group of genetic disorders impacting bone metabolism. The collagen mutations in OI can result in increased bone brittleness, which contributes to a high rate of fractures.Ultragenyx Pharmaceutical shares dipped 42% to $19.84 on Monday.H ...

Core Insights - Mereo BioPharma announced that neither the Phase 3 ORBIT nor COSMIC studies for setrusumab (UX143) achieved their primary endpoint of reducing annualized clinical fracture rates compared to placebo or bisphosphonates, respectively. However, both studies successfully met their secondary endpoints, showing significant improvements in bone mineral density (BMD) [1][2][3]. Study Results - In the ORBIT study, participants showed statistically significant improvements in BMD compared to placebo, consistent with Phase 2 study results, but did not see a corresponding reduction in fracture rates [3]. - The pediatric COSMIC study revealed that patients had a higher baseline fracture rate compared to those in ORBIT. Although setrusumab-treated patients showed meaningful BMD improvements and a reduction in fracture rates compared to bisphosphonate-treated patients, this reduction did not reach statistical significance [4]. Financial and Strategic Considerations - Mereo's cash balance was reported at $48.7 million at the end of Q3 2025. The company plans to manage cash resources carefully, implementing immediate reductions in pre-commercial and manufacturing activities while continuing to analyze the setrusumab data for future steps [5]. - The company is also advancing discussions for partnerships related to alvelestat, another of its drug candidates [2][9]. Product Development Context - Setrusumab is being developed for pediatric and young adult patients with Osteogenesis Imperfecta (OI) across various sub-types, with the ORBIT and COSMIC studies being pivotal in this development [6][8]. - The ORBIT study involved 159 patients aged 5 to 25 years, while the COSMIC study enrolled 69 patients aged 2 to under 7 years, both randomized to receive either setrusumab or a comparator [7][8]. Company Overview - Mereo BioPharma focuses on developing innovative therapeutics for rare diseases, with setrusumab being one of its key candidates for OI. The company has retained commercial rights in the EU and UK and has partnered with Ultragenyx Pharmaceutical for the development of setrusumab [12].

Neither study achieved its primary endpoint of reduction in annualized clinical fracture rate compared to placebo (Orbit) or bisphosphonates (Cosmic) Both studies achieved the secondary endpoint of improvements in bone mineral density with strong statistical significance The Company will implement significant expense reductions NOVATO, Calif., Dec. 29, 2025 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced results from the Phase 3 Orbit and Cosmic studies for setrusumab (UX14 ...