他补充称，该公司维持其 2027 年盈利指引，并将在 2026 年初明确削减开支的计划。 Raycroft 还指出，对于刚刚在成骨不全症三期临床试验中失败的候选药物 setrusumab，希望或许并未完 全破灭。他表示："美国食品药品监督管理局(FDA)最近将'骨密度(BMD)'列为骨质疏松症的替代指标， 我们认为这可能会影响 Ultragenyx 及合作伙伴 Mereo BioPharma(MREO)与该机构的后续讨论。" 然而，花旗对 Ultragenyx 的态度则没那么乐观。该行已将该公司从其 90 天内上行催化剂观察名单中移 除。 智通财经APP获悉，由于针对罕见骨病资产的两项后期临床试验失败，Ultragenyx Pharmaceutical (RARE.US) 周一收盘下跌约 42%，但投行杰富瑞发布报告称，鉴于其管线中的其他候选药物，该公司 有望在 2026 年迎来反弹。 杰富瑞分析师 Maury Raycroft，给予 Ultragenyx "买入"评级并表示，公司明年可能会从 apazunersen (GTX-102) 的三期数据公布中获益，这是一种用于治疗天使综合征(Angelman sy ...

Core Insights - Ultragenyx Pharmaceutical Inc. has completed the rolling submission of its Biologics License Application (BLA) for DTX401, an AAV gene therapy aimed at treating Glycogen Storage Disease Type Ia (GSDIa) [1][2] - If approved, DTX401 will be the first therapy to address the underlying cause of GSDIa, which currently has no approved pharmacologic treatments [5] Company Overview - Ultragenyx is a biopharmaceutical company focused on developing novel therapies for serious rare and ultra-rare genetic diseases, with a portfolio aimed at addressing high unmet medical needs [6][7] - The company has received multiple designations from the FDA for DTX401, including Rare Pediatric Disease designation, orphan drug designation, Fast Track designation, and regenerative medicine advanced therapy (RMAT) designation [4] Clinical Development - The BLA submission for DTX401 is based on data from a clinical program involving 52 treated patients and up to six years of follow-up, demonstrating significant reductions in daily cornstarch intake and improved patient quality of life [2][3] - DTX401 has shown to improve G6Pase activity and reduce hepatic glycogen levels, which are critical for managing glucose metabolism in patients with GSDIa [4] Disease Context - GSDIa is a rare and life-threatening condition caused by mutations in the G6PC gene, leading to severe hypoglycemia and other complications [5] - The disease affects approximately 6,000 individuals in commercially accessible regions, highlighting the need for effective treatment options [5]

Key Points A Wells Fargo analyst cut his price target on Ultragenyx stock, though it still implies significant upside from yesterday's closing price. Ultragenyx reported yesterday that two Phase 3 studies failed to achieve primary endpoints for setrusumab. Despite the Wells Fargo analyst's positive outlook, Ultragenyx stock should only remain a consideration for those comfortable with more speculative investments. 10 stocks we like better than Ultragenyx Pharmaceutical › While Ultragenyx Pharmac ...

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]

U.S. stocks were mostly lower, with the Dow Jones index falling more than 0.1% on Tuesday.Shares of CapsoVision, Inc. (NASDAQ:CV) rose sharply during Tuesday's session.The company submitted its 510(k) application to the FDA for its proprietary AI-assisted module for CapsoCam Plus.CapsoVision shares jumped 8.4% to $12.55 on Tuesday.Here are some other big stocks recording gains in today’s session.AXT, Inc. (NASDAQ:AXTI) shares jumped 13.5% to $16.56. AXT recently announced it priced its $87 million public of ...

Core Viewpoint - Ultragenyx Pharmaceutical Inc. shares fell 42% following disappointing late-stage data for their rare bone disease program in collaboration with Mereo BioPharma Group plc [1] Group 1: Study Results - The Phase 3 Orbit and Cosmic studies for setrusumab (UX143) in Osteogenesis Imperfecta (OI) did not achieve statistical significance for the primary endpoints of reducing annualized clinical fracture rate compared to placebo or bisphosphonates [2] - Both studies met secondary endpoints, showing improvements in bone mineral density (BMD) against comparators, with no changes in the safety profile observed [2] Group 2: Analyst Reactions - Analysts from William Blair expressed disappointment and surprise at the results, especially given setrusumab's efficacy in the Phase 2 portion of the Orbit study and the increased enrollment of patients with severe disease subtypes in the Phase 3 study [3] - Analyst Sami Corwin is cautious about the future of setrusumab, noting several unknowns regarding the data, including potential effects in pediatric patients and the unexpectedly low annualized fracture rate in the placebo arm [4] Group 3: Market Impact and Future Outlook - Following the news, Ultragenyx is trading at a value reflective of its approved products, with William Blair maintaining an Outperform rating for the stock [4] - There is potential for upside in the next 12 months, particularly with the pivotal Angelman syndrome readout expected in Q3 2026 and the potential approval of two gene therapies eligible for priority review vouchers [5]

Upgrades - Argus upgraded BHP Group (BHP) to Buy from Hold with a price target of $68, citing firming prices for iron ore, copper, and coal as the global economy improves [2] Downgrades - Jefferies downgraded Mereo BioPharma (MREO) to Hold from Buy with a price target of 50 cents, down from $7, due to Setrusumab missing primary endpoints in trials, impacting Mereo's future strategy [3] - RBC Capital downgraded DigitalBridge (DBRG) to Sector Perform from Outperform with a price target of $16, down from $23, following SoftBank's acquisition announcement at $16 per share, totaling a $4 billion enterprise value [4] Initiations - Stephens initiated coverage of Commvault (CVLT) with an Overweight rating and a $162 price target, highlighting its leadership in data protection and transition to Software-as-a-Service [5] - Stephens initiated coverage of Rubrik (RBRK) with an Overweight rating and a $105 price target, noting its strong growth outlook in data protection and expected margin expansion [5] - Stephens initiated coverage of Varonis (VRNS) with an Equal Weight rating and a $40 price target, emphasizing its growth potential in data security as a Software-as-a-Service platform [5] - Freedom Capital initiated coverage of Ero Copper (ERO) with a Buy rating and a $32 price target, pointing out its high-grade, low-cost assets in Brazil [5] - H.C. Wainwright initiated coverage of Terra Innovatum (NKLR) with a Buy rating and a $25 price target, believing its use of off-the-shelf components will lower regulatory and technological barriers [5]

Group 1 - Zijin Mining (02899) rose nearly 3% after announcing the completion of the main construction and core equipment installation for the second phase of the Xizang Julong Copper Mine, marking a key step before production [1] - Asia Pacific Satellite (01045) increased over 5.4%, with a cumulative rise of nearly 30% over the past five trading days, as it operates multiple satellites under China Satcom [1] - Zhaoyan New Drug (06127) fell over 6% following an announcement that its actual controller plans to reduce holdings by up to 14.98 million shares, representing 20.0466% of his holdings and 1.99873% of the company's total share capital [1] - Zhongwei New Materials (02579) surged nearly 7% after signing a strategic cooperation framework agreement with Xinwanda for solid-state batteries [1] Group 2 - Dongyue Group (00189) rose nearly 5% as the long-term contract prices for refrigerants were confirmed, with R32 priced at 61,200 yuan/ton (+1,000 yuan) and R410A at 55,100 yuan/ton (+1,900 yuan) [2] - MicroPort Scientific Corporation (02252) increased over 1.2%, reporting that its core products' cumulative order volume exceeded 230 units [2] - Oil stocks saw gains, with CNOOC (00883) up 4.17%, PetroChina (00857) up 2.91%, and Sinopec (00386) up 1.31%, driven by a rise in international crude oil prices due to geopolitical tensions [2] - Baidu Group (09888) rose nearly 7% after announcing a partnership with Uber and Lyft to launch a robotaxi pilot project in the UK by 2026 [2] Group 3 - NIO (09866) rose over 2.4% after announcing that it successfully delivered over 40,000 units of its new ES8 model within 100 days of the official delivery start [3] - Damai Entertainment (01060) increased nearly 5%, with the total box office for the 2025 New Year’s season reaching 5.245 billion yuan as of December 28 [3] Group 4 - Copper mining stocks collectively declined, with Hudbay Minerals (HBM.US) down 4.01%, Taseko Mines (TGB.US) down 3.1%, and Ero Copper (ERO.US) down 1.64%, as international copper futures fell over 4% [5] - Some cryptocurrency-related stocks fell, with IREN Ltd (IREN.US) down 2.21% and Circle (CRCL.US) down 0.94% [5] - NIO (NIO.US) rose 4.71% in the US market, echoing its performance in Hong Kong [6] - The Nasdaq Golden Dragon Index fell nearly 1%, with major Chinese concept stocks declining, including Alibaba (BABA.US) down 2.46% and Xpeng Motors (XPEV.US) down 1.35% [6]

Core Insights - Ultragenyx Pharmaceuticals announced disappointing Phase 3 study results for setrusumab, leading to a significant drop in stock price [1][4] - Analysts have reacted by slashing price targets for Ultragenyx stock, reflecting a negative outlook [7][9] Company Performance - Ultragenyx's shares are down 43.5%, currently priced at $19.25, with a market cap of $3.3 billion [2][5] - The Phase 3 studies, Orbit and Cosmic, failed to meet primary endpoints related to fracture rate reductions [4][6] Analyst Reactions - Cantor Fitzgerald cut its price target for Ultragenyx from $105 to $84 [9] - Barclays reduced its price target from $81 to $50 [9] - Citigroup lowered its price target from $103 to $50 [9] Market Context - The current trading range for Ultragenyx shares is between $18.41 and $20.49, with a 52-week range of $18.41 to $46.50 [5][6] - The gross margin for the company stands at 79.09% [6] Future Considerations - Investors are advised to monitor the company’s upcoming data from the Phase 3 study of GTX-102 for Angelman syndrome before making investment decisions [8]