Ultragenyx Pharmaceutical (RARE) is back in focus after the FDA accepted its resubmitted biologics license application for UX111, a gene therapy for Sanfilippo syndrome Type A, with an accelerated approval decision expected later this year.
See our latest analysis for Ultragenyx Pharmaceutical.
The FDA milestones around UX111 and UX016 have arrived against a mixed backdrop, with a sharp 18.5% 7 day share price return contrasting with a 1 year total shareholder return of 28.84% and a 5 year total shareholder return of 77.25%. This suggests near term momentum following a long stretch of weaker results.
If this kind of biotech volatility has your attention, it could be a good moment to broaden your watchlist with other healthcare names working at the intersection of medicine and machine learning through our screener for 37 healthcare AI stocks
With Ultragenyx shares still trading well below many analysts’ targets despite the recent UX111 and UX016 progress, the key question is whether this reflects an overlooked entry point or a market that already prices in future growth.
Most Popular Narrative: 56.4% Undervalued
Analysts following Ultragenyx see a fair value of $52.45 per share, compared with the last close at $22.87, and anchor that gap to an aggressive earnings turnaround story.
Analysts expect earnings to reach $69.1 million (and earnings per share of $0.59) by about March 2029, up from -$575.0 million today. However, there is a considerable amount of disagreement amongst the analysts with the most bullish expecting $289.8 million in earnings, and the most bearish expecting $-226.7 million.
Curious how a loss making rare disease specialist gets to that earnings profile and still lands on a very high implied future P/E? The narrative leans on brisk revenue expansion, a shift in profit margins, and a valuation multiple more often seen in faster growing sectors. Want to see exactly which assumptions carry the most weight in that $52.45 figure?
Result: Fair Value of $52.45 (UNDERVALUED)
Have a read of the narrative in full and understand what's behind the forecasts.
However, the bullish story also leans on successful UX111 and DTX401 approvals and tighter cost control, and setbacks on either front could quickly weaken that thesis.
Find out about the key risks to this Ultragenyx Pharmaceutical narrative.
Next Steps
With a mix of optimism around UX111 and UX016 and clear concerns in the background, it helps to review the details for yourself and move quickly while opinions are still forming. Start with a closer look at the 2 key rewards and 2 important warning signs.
Looking for more investment ideas?
If Ultragenyx has you thinking more broadly about your portfolio, now is the time to widen your search before other investors crowd into the same opportunities.
- Target potential mispricings by checking companies that screen well on quality and valuation through the 62 high quality undervalued stocks.
- Strengthen your income stream by focusing on companies with reliable payouts using the 13 dividend fortresses.
- Prioritize capital preservation by zeroing in on businesses that clear high bars for financial resilience in the 68 resilient stocks with low risk scores.
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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