Ultragenyx (RARE) Is Up 5.2% After DTX401 Phase 3 Gene Therapy Results Show Lasting Patient Benefit – Has the Bull Case Changed?

• On September 8, 2025, Ultragenyx Pharmaceutical announced positive longer-term Phase 3 results for its DTX401 gene therapy, showing significant and sustained reductions in daily and nighttime cornstarch intake for patients with glycogen storage disease type Ia, alongside maintained glycemic control and an acceptable safety profile.
• These findings, presented at a major international congress, highlight meaningful improvements in patients’ quality of life and may set a new standard for gene therapy in rare metabolic diseases.
• We’ll explore how these robust and durable clinical results for DTX401 could influence Ultragenyx’s pipeline-driven investment narrative.

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Ultragenyx Pharmaceutical Investment Narrative Recap

To be a shareholder in Ultragenyx Pharmaceutical, you need confidence in the company’s ability to advance key late-stage gene therapy programs and turn promising clinical results into regulatory and commercial success. The latest compelling Phase 3 data for DTX401 strengthens the near-term catalyst of regulatory approval for GSDIa, but the company’s persistent cash burn and unprofitability remain serious concerns, especially if revenue acceleration does not materialize as anticipated.

Of the recent announcements, the August 18 initiation of a rolling FDA Biologics License Application for DTX401 is most relevant, as it signals regulatory momentum directly tied to the new clinical results. The BLA progress is closely watched, since successful approval could help mitigate liquidity worries while a delay or further regulatory hurdles for DTX401 would amplify execution risk and financial pressure in the coming quarters.

Yet, despite clinical momentum, investors should be aware that ongoing cash burn and profitability risks remain front of mind for...

Read the full narrative on Ultragenyx Pharmaceutical (it's free!)

Ultragenyx Pharmaceutical is forecast to generate $1.4 billion in revenue and $46.9 million in earnings by 2028. This outlook assumes a 32.0% annual revenue growth rate and a $579.8 million increase in earnings from the current earnings of -$532.9 million.

Uncover how Ultragenyx Pharmaceutical's forecasts yield a $86.05 fair value, a 173% upside to its current price.

Exploring Other Perspectives

The Simply Wall St Community provided just 2 fair value estimates for Ultragenyx, ranging widely from US$86.05 to US$375.18 per share. As upbeat regulatory updates focus attention on new therapies, persistent cash burn and lack of profitability highlight key uncertainties that the market continues to weigh.

Explore 2 other fair value estimates on Ultragenyx Pharmaceutical - why the stock might be worth just $86.05!

Build Your Own Ultragenyx Pharmaceutical Narrative

Disagree with existing narratives? Create your own in under 3 minutes - extraordinary investment returns rarely come from following the herd.

• A great starting point for your Ultragenyx Pharmaceutical research is our analysis highlighting 2 key rewards that could impact your investment decision.
• Our free Ultragenyx Pharmaceutical research report provides a comprehensive fundamental analysis summarized in a single visual - the Snowflake - making it easy to evaluate Ultragenyx Pharmaceutical's overall financial health at a glance.

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This article by Simply Wall St is general in nature. We provide commentary based on historical data and analyst forecasts only using an unbiased methodology and our articles are not intended to be financial advice. It does not constitute a recommendation to buy or sell any stock, and does not take account of your objectives, or your financial situation. We aim to bring you long-term focused analysis driven by fundamental data. Note that our analysis may not factor in the latest price-sensitive company announcements or qualitative material. Simply Wall St has no position in any stocks mentioned.

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