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RARE: Upcoming Study Results Will Drive Future Reward Versus Risk Profile

Update shared on 07 Nov 2025

Fair value Decreased 2.27%
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AnalystConsensusTarget's Fair Value
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1Y
-39.2%
7D
-11.5%

Ultragenyx Pharmaceutical’s consensus analyst price target has decreased from approximately $86 to $75, as analysts cite slightly lighter total revenues, partially offset by robust product performance and reaffirmed profitability guidance.

Analyst Commentary

Analysts provided updated price targets and shared their perspectives on Ultragenyx Pharmaceutical's recent performance, balancing positive revenue highlights with areas of caution regarding growth outlook and valuation.

Bullish Takeaways
  • Bullish analysts maintain a Buy or Overweight rating, reflecting confidence in Ultragenyx's product portfolio and near- to mid-term growth prospects.
  • Revenues from Crysvita in the third quarter surpassed expectations, underpinning optimism about core product performance and execution.
  • Profitability guidance for 2025 and 2027 was reaffirmed, emphasizing management's commitment to a clear path toward achieving key financial milestones.
  • Upcoming clinical study updates, particularly for setrusumab, are viewed as significant potential catalysts. Some major institutions express a positive reward versus risk profile for the shares at current levels.
Bearish Takeaways
  • Bearish analysts highlight that overall total revenues for the quarter came in lighter than anticipated, raising some concerns about the pace of top-line growth.
  • Lowered price targets reflect adjustments for near-term revenue expectations and sector-wide peer group evaluations, which tempers valuation upside.
  • Cautious views persist around the company's pipeline execution risks. Future performance remains tied to upcoming study results and broader market conditions.
  • There is attention to the competitive biotech landscape. Some analysts moderate expectations amid industry-wide volatility and fiscal adjustments in target forecasts.

What's in the News

  • Ultragenyx reaffirmed its earnings guidance for 2025, projecting total revenue in the range of $640 million to $670 million. This represents expected growth of approximately 14-20% compared to 2024 (Key Developments).
  • The company announced that the first patient has been dosed in the global Aurora study evaluating GTX-102 (apazunersen) for Angelman syndrome. The study expands the treated patient population and includes multiple genotypes and age groups (Key Developments).
  • Enrollment for the Phase 3 Aspire study of GTX-102 in Angelman syndrome patients aged 4 to 17 was completed ahead of plan. Data from the study are expected in the second half of 2026 (Key Developments).
  • Ultragenyx reported positive longer-term results from its Phase 3 study of DTX401 gene therapy for glycogen storage disease type Ia. The results showed significant and sustained reductions in daily cornstarch intake with an acceptable safety profile (Key Developments).
  • The company has initiated a rolling submission of its Biologics License Application to the FDA for DTX401 and aims to finalize the application by the end of 2025 (Key Developments).

Valuation Changes

  • Fair Value: Decreased modestly from $86.05 to $84.10 per share.
  • Discount Rate: Increased slightly from 7.09% to 7.45%, reflecting higher perceived risk or market adjustments.
  • Revenue Growth: Projected to rise marginally, from 31.98% to 32.49%.
  • Net Profit Margin: Improved significantly from 3.34% to 9.78%, indicating expectations for stronger profitability.
  • Future P/E: Declined sharply from 243.6x to 79.3x, suggesting analyst expectations for increased earnings relative to share price.

Disclaimer

AnalystConsensusTarget is a tool utilizing a Large Language Model (LLM) that ingests data on consensus price targets, forecasted revenue and earnings figures, as well as the transcripts of earnings calls to produce qualitative analysis. The narratives produced by AnalystConsensusTarget are general in nature and are based solely on analyst data and publicly-available material published by the respective companies. These scenarios are not indicative of the company's future performance and are exploratory in nature. Simply Wall St has no position in the company(s) mentioned. Simply Wall St may provide the securities issuer or related entities with website advertising services for a fee, on an arm's length basis. These relationships have no impact on the way we conduct our business, the content we host, or how our content is served to users. The price targets and estimates used are consensus data, and do not constitute a recommendation to buy or sell any stock, and they do not take account of your objectives, or your financial situation. Note that AnalystConsensusTarget's analysis may not factor in the latest price-sensitive company announcements or qualitative material.