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Assessing Ultragenyx Pharmaceutical (RARE) Valuation After New DTX401 Filing And Setrusumab Phase 3 Results

Simply Wall St · 4d ago

Why recent trial and FDA milestones matter for Ultragenyx Pharmaceutical

Ultragenyx Pharmaceutical (RARE) has just reported two key clinical milestones and completed a Biologics License Application submission for DTX401. These developments help frame how you might think about the stock at today’s levels.

See our latest analysis for Ultragenyx Pharmaceutical.

The recent DTX401 BLA submission and positive UX143 Phase 3 data arrive after a sharp 36.48% 1 month share price return decline and a 48.0% 1 year total shareholder return decline. This suggests recent news is being weighed against ongoing risk concerns rather than driving a clear upswing in momentum.

If you are reassessing rare disease exposure after this update, it can help to widen the lens and see how other healthcare stocks compare on fundamentals and recent price performance.

With the shares down sharply over 1 month, 1 year and even 5 years, yet trading at what looks like a steep discount to analyst targets and some intrinsic value models, is this weakness offering you upside, or is the market already discounting future growth?

Most Popular Narrative Narrative: 71.8% Undervalued

With Ultragenyx Pharmaceutical last closing at $23.11 against a narrative fair value of $81.85, the current gap between price and implied worth is wide and attention grabbing.

Ultragenyx's clinical pipeline is advancing with five Phase III programs (including UX143 and GTX-102), multiple BLA submissions expected in the coming quarters, and near-term Phase III data readouts (notably for UX143 in OI by year-end and GTX-102 in Angelman syndrome in 2026) serving as upcoming value inflection points that can diversify and significantly accelerate the company's revenue base.

Read the complete narrative.

Curious how this late stage pipeline, rapid revenue build assumptions and a rich future earnings multiple all fit together? The narrative leans on aggressive growth, expanding margins and a premium P/E well above sector norms. Want to see which financial levers do the heavy lifting in that $81.85 fair value?

Result: Fair Value of $81.85 (UNDERVALUED)

Have a read of the narrative in full and understand what's behind the forecasts.

However, this upside story can crack if ongoing net losses of US$579.8 million persist, or if further regulatory setbacks, like prior FDA letters, slow key programs.

Find out about the key risks to this Ultragenyx Pharmaceutical narrative.

Build Your Own Ultragenyx Pharmaceutical Narrative

If you are not on board with this outlook or you prefer to test the assumptions yourself, you can build a fresh view in just a few minutes with Do it your way

A great starting point for your Ultragenyx Pharmaceutical research is our analysis highlighting 2 key rewards and 3 important warning signs that could impact your investment decision.

Looking for more investment ideas?

If Ultragenyx has you thinking harder about where you put your capital next, use this moment to broaden your watchlist before the next set of stories moves on.

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.