<div id="readability-page-1"><div><ul> <li>Ultragenyx Pharmaceutical recently reported two key milestones: strong Phase 3 results for setrusumab (UX143) in Osteogenesis Imperfecta and completion of its Biologics License Application to the FDA for DTX401 gene therapy in Glycogen Storage Disease Type Ia, supported by up to six years of clinical follow-up data.</li> <li>These advances highlight Ultragenyx’s push to convert late-stage rare disease programs into potential commercial therapies that could broaden its treatment portfolio and reinforce its focus on high unmet medical need.</li> <li>We’ll now examine how the BLA completion for DTX401 and positive UX143 data may reshape Ultragenyx’s investment narrative and outlook.</li> </ul><p>We've found 14 US stocks that are forecast to pay a dividend yield of over 6% next year. See the full list for free.</p><h2>Ultragenyx Pharmaceutical Investment Narrative Recap</h2><p>To own Ultragenyx, you need to believe its late stage rare disease pipeline can eventually offset persistent losses and a short cash runway. The BLA completion for DTX401 and positive UX143 Phase 3 data sharpen the near term regulatory catalysts, but they do not remove the core risk around ongoing cash burn and potential dilution.</p> <p>The most relevant recent announcement is the completed BLA submission for DTX401 in Glycogen Storage Disease Type Ia, backed by up to six years of follow up data and favorable Phase 3 GlucoGene outcomes on cornstarch dependence, glycemic control, and quality of life. This filing moves one of Ultragenyx’s key gene therapy assets closer to a potential approval decision, directly tying into the company’s reliance on regulatory success to broaden its revenue base.</p> <p>However, while these clinical and regulatory steps are encouraging, investors should still be aware of the company’s high cash burn and limited cash runway...</p> <p>Read the full narrative on Ultragenyx Pharmaceutical (it's free!)</p><p>Ultragenyx Pharmaceutical's narrative projects $1.4 billion revenue and $46.9 million earnings by 2028.</p><p>Uncover how Ultragenyx Pharmaceutical's forecasts yield a $81.85 fair value, a 247% upside to its current price.</p><h2>Exploring Other Perspectives</h2><figure><img height="476" src="https://imageproxy.pbkrs.com/https://images.simplywall.st/company/f1981b75-8949-4fa0-9111-bfb36bcac5de/chart/quote-price?x-oss-process=image/auto-orient,1/interlace,1/resize,w_1440,h_1440/quality,q_95/format,jpg" width="746" original-src="https://imageproxy.pbkrs.com/https://images.simplywall.st/company/f1981b75-8949-4fa0-9111-bfb36bcac5de/chart/quote-price"/><figcaption>RARE 1-Year Stock Price Chart</figcaption></figure> <p>Two fair value estimates from the Simply Wall St Community span roughly US$82 to US$329 per share, showing how far apart individual expectations can be. Against that wide range, Ultragenyx’s dependence on successful regulatory outcomes for assets like DTX401 and UX111 becomes a central issue for how its future performance might evolve, so it is worth reviewing several different viewpoints before forming your own stance.</p><p>Explore 2 other fair value estimates on Ultragenyx Pharmaceutical - why the stock might be worth just $81.85!</p><h2>Build Your Own Ultragenyx Pharmaceutical Narrative</h2><p>Disagree with existing narratives? Create your own in under 3 minutes - extraordinary investment returns rarely come from following the herd.</p><ul><li>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.</li><li>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.</li></ul><h2>No Opportunity In Ultragenyx Pharmaceutical?</h2><p>Every day counts. These free picks are already gaining attention. See them before the crowd does:</p><ul><li>The end of cancer? 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Simply Wall St has no position in any stocks mentioned.</em></p><div><p>Mobile Infrastructure for Defense and Disaster</p><p>The next wave in robotics isn't humanoid. Its fully autonomous towers delivering 5G, ISR, and radar in under 30 minutes, anywhere.</p><p>Get the investor briefing before the next round of contracts</p><p><span>Sponsored On Behalf of CiTech</span></p></div></div></div>

RARE

<p>Ultragenyx Pharmaceutical's stock has dropped 31% following updates on its rare disease programs. The company reported positive Phase 3 results for setrusumab in Osteogenesis Imperfecta and completed its Biologics License Application for DTX401 gene therapy in Glycogen Storage Disease Type Ia. Despite these advancements, concerns remain regarding ongoing cash burn and a short cash runway. Analysts project a fair value of $81.85 per share, indicating a potential upside, but the company's reliance on regulatory success poses risks to its future performance.</p>

Ultragenyx Pharmaceutical (RARE) Is Down 31.0% After Rare-Disease Milestone Updates - Has The Bull Case Changed?