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On Monday, Piper Sandler analyst Allison M. Bratzel revised the price target for Ultragenyx Pharmaceutical (TADAWUL:2070) Inc. (NASDAQ:RARE) to $115 from the previous $140, while maintaining an Overweight rating on the company’s shares. Currently trading at $39.34, the stock sits near its 52-week low of $37.02, with analyst targets ranging from $47 to $136. According to InvestingPro analysis, the stock appears undervalued based on its Fair Value calculation. Bratzel emphasized the company’s solid commercial business and highlighted a portfolio of high-value assets with relatively low risk at various stages of development. The analyst underscored several upcoming clinical and regulatory milestones, particularly pointing to the second interim analysis of setrusumab, intended for osteogenesis imperfecta (OI), expected around mid-year.
Bratzel also noted Ultragenyx’s significant progress in gene therapy, with a successful Biologics License Application (BLA) submission for UX111 for MPS IIIA, which could potentially lead to the company’s first gene therapy launch by the end of 2025. The optimism is fueled by the potential of the company’s diverse rare disease programs, which are anticipated to drive value in the near term.
The analyst’s comments reflect a belief that Ultragenyx is poised for growth, with several key drivers that could significantly impact the company’s valuation. The company has demonstrated strong revenue growth of 29% over the last twelve months, reaching $560 million. Despite the reduction in the price target, the Overweight rating suggests a positive outlook on the stock’s performance, with expectations of upward movement in the market. InvestingPro subscribers have access to 8 additional key insights about RARE’s financial health and market position, along with comprehensive analysis in the Pro Research Report, which transforms complex Wall Street data into actionable intelligence.
The company’s stock price will likely be influenced by the outcomes of the clinical trials and regulatory decisions mentioned by Bratzel. Investors and market watchers will be paying close attention to the results of the setrusumab interim analysis and the progress of the UX111 BLA, as these events could serve as catalysts for stock movement.
Ultragenyx Pharmaceutical Inc., which specializes in developing treatments for rare and ultra-rare diseases, has been working on a range of therapies that address unmet medical needs. With a market capitalization of $3.64 billion and a healthy current ratio of 2.37, the company maintains strong liquidity to support its operations. The company’s focus on gene therapy and its pipeline of products in various development stages contribute to its potential for future growth and value creation.
In other recent news, Ultragenyx Pharmaceutical Inc. reported fourth-quarter 2024 earnings that exceeded expectations, with total revenue reaching $164.9 million, surpassing estimates from both Goldman Sachs and Visible Alpha. Sales from key products Crysvita and Dojolvi were particularly strong, contributing $115.2 million and $31.1 million, respectively. Canaccord Genuity responded to these results by raising its price target for Ultragenyx to $136, maintaining a Buy rating, and highlighting the company’s robust growth trajectory. Meanwhile, H.C. Wainwright maintained its Buy rating with a $95 target, emphasizing the FDA’s acceptance of the Biologics License Application for UX111, a gene therapy for Sanfilippo syndrome type A, with a PDUFA date set for August 18, 2025.
Cantor Fitzgerald reiterated its Overweight rating with a $118 target, noting the progress of Ultragenyx’s DTX301 program for OTC deficiency and the potential impact of upcoming clinical readouts for setrusumab. Goldman Sachs also maintained its Buy rating with a $78 target, expressing confidence in the ongoing Phase 3 Orbit study of setrusumab for osteogenesis imperfecta. Analysts from these firms suggest that Ultragenyx’s pipeline holds significant potential, with multiple product launches anticipated in the coming years. The company has reiterated its full-year 2025 revenue guidance, projected between $640 million and $670 million, aligning closely with analysts’ estimates. These developments underscore a period of strategic growth and expansion for Ultragenyx as it continues to advance its portfolio of treatments for rare diseases.
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