Interactive Brokers shares jump as it secures spot in S&P 500
On Wednesday, H.C. Wainwright reaffirmed its Buy rating and $95.00 price target for Ultragenyx Pharmaceutical (TADAWUL:2070) Inc. (NASDAQ:RARE) shares, significantly above the current trading price of $43.62. According to InvestingPro data, analysts maintain a strong bullish consensus on the stock, with price targets ranging from $47 to $140. The firm’s analyst highlighted the recent development where the U.S. Food and Drug Administration (FDA) accepted the Biologics License Application (BLA) of UX111 (ABO-102), Ultragenyx’s gene therapy candidate for Sanfilippo syndrome type A (MPS IIIA), for Priority Review. The FDA has set a Prescription Drug User Fee Act (PDUFA) action date for August 18, 2025, and has indicated that it does not anticipate convening an advisory committee meeting for the application.
Sanfilippo syndrome type A is a rare, life-threatening lysosomal storage disorder that primarily affects the brain, leading to rapid neurodegeneration starting in early childhood. Currently, there is no approved treatment for this condition, which has an estimated patient population of 3,000 to 5,000 individuals in commercially accessible regions and a median life expectancy of 15 years. For Ultragenyx, which has shown impressive revenue growth of 29% over the last twelve months and maintains a market capitalization of $4 billion, this represents a significant market opportunity. The BLA for UX111 is backed by data from the ongoing pivotal Transpher A and long-term follow-up studies, which have demonstrated that treatment with UX111 results in a rapid and sustained reduction of heparan sulfate levels in the cerebrospinal fluid of patients with MPS IIIA, irrespective of their age or disease progression stage at the time of treatment.
Ultragenyx’s management has expressed readiness to launch UX111 upon potential FDA approval by the PDUFA date. This event is viewed as a critical milestone, being one of three anticipated near-term approvals that are part of the company’s strategy to achieve full-year GAAP profitability by 2027. While InvestingPro data shows the company is not currently profitable, with a moderate debt level, analysts anticipate improving financial metrics in the coming years. The analyst’s commentary underscores the importance of this development for Ultragenyx and its commitment to addressing the unmet medical needs of patients with MPS IIIA. For detailed analysis and additional insights, including 6 more key ProTips, check out the comprehensive Pro Research Report available on InvestingPro.
In other recent news, Ultragenyx Pharmaceutical Inc. reported fourth-quarter 2024 earnings that exceeded expectations, driven by strong sales from its Crysvita, Dojolvi, and Evkeeza product lines. Analysts from Canaccord Genuity responded by raising their price target for the company to $136, maintaining a Buy rating. Ultragenyx’s total revenue for the quarter was $164.9 million, surpassing estimates from Goldman Sachs and Visible Alpha. The company has reiterated its full-year 2025 revenue guidance, projected to be between $640 million and $670 million.
Truist Securities echoed positive sentiments, maintaining a Buy rating and a $140 price target, while highlighting Ultragenyx’s path to GAAP profitability by 2027. Meanwhile, Cantor Fitzgerald reaffirmed its Overweight rating and $118 price target, noting the progress in the DTX301 program for OTC deficiency. The firm’s analysts suggested that upcoming clinical readouts for setrusumab could significantly impact the stock.
Goldman Sachs also maintained a Buy rating with a $78 target, emphasizing Ultragenyx’s promising pipeline, including the UX111 gene therapy for Sanfilippo syndrome. Recent data from Ultragenyx showed significant cognitive improvements in patients treated with UX111, with a Biologics License Application decision expected in the second half of 2025.
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