Ultragenyx Pharmaceutical Inc. (RARE)
Summary
Ultragenyx (RARE) Soars 5.2%: Is Further Upside Left in the Stock?
Ultragenyx (RARE) saw its shares surge in the last session with trading volume being higher than average. The latest trend in earnings estimate revisions may not translate into further price increase in the near term.
Ultragenyx: The Setrusumab Reset Creates A Cleaner Rare Disease Opportunity
Ultragenyx (RARE) is transitioning from a broad pipeline to a focused rare disease platform, entering a high-catalyst period with four approved products and multiple late-stage assets. Despite a recent Phase 3 failure and Q1 2026 net loss, RARE reaffirmed 2026 revenue guidance of $730–$760 million, supported by a scalable commercial base and cost controls. Key near-term catalysts include FDA reviews for DTX401 and UX111 (PDUFA dates in H2 2026) and pivotal Phase 3 Aspire results for GTX-102 in Angelman syndrome.
Ultragenyx Pharmaceutical Inc. (RARE) Presents at Goldman Sachs 47th Annual Global Healthcare Conference 2026 Transcript
Ultragenyx Pharmaceutical Inc. (RARE) Presents at Goldman Sachs 47th Annual Global Healthcare Conference 2026 Transcript
Ultragenyx Pharmaceutical Inc. Investors
| Name | Quantity | Cost | Value | Profit ($) | Gain (%) |
|---|---|---|---|---|---|
| CE Curtis Ellergodt Rothschild Investment LLC | 65 | $1,495 | $1,903.2 | $408.2 | 27.3% |
| ABB Alexander Bjornager Bonde Danske Bank A/S | 100 | $2,300 | $2,928 | $628 | 27.3% |
| JD Jim Dushek HARBOUR INVESTMENTS Inc. | 2 | $41.9 | $58.56 | $16.66 | 39.76% |
| SN Stephanie Nee HARBOR CAPITAL ADVISORS Inc. | 83,196 | $1.74M | $2.44M | $693,022.68 | 39.76% |
 Point72 Asset Management LP Point72 Asset Management LP | 893,295 | $18.71M | $26.16M | $7.44M | 39.76% |
Ultragenyx Pharmaceutical Inc. Analysts
| Name | Quantity | Cost | Value | Profit ($) | Gain (%) |
|---|---|---|---|---|---|
 Kristen Kluska Cantor Fitzgerald | 501.13 | $17,344.02 | $15,890.75 | -$1,453.27 | -8.38% |
| JH Jeffrey Hung Morgan Stanley | 649.52 | $17,471.87 | $20,596.26 | $3,124.39 | 17.88% |
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Ultragenyx Pharmaceutical Inc. Profile
| Biotechnology Industry | Healthcare Sector | Emil D. Kakkis CEO | NASDAQ (NGS) Exchange | 90400D108 CUSIP |
| US Country | 1,294 Employees | - Last Dividend | - Last Split | 31 Jan 2014 IPO Date |
Overview
Ultragenyx Pharmaceutical Inc. is a pioneering biopharmaceutical firm dedicated to the discovery, acquisition, development, and commercialization of innovative products for the diagnosis and treatment of rare and ultra-rare genetic disorders across North America, Latin America, Japan, Europe, and globally. Founded in 2010 and based in Novato, California, Ultragenyx operates at the forefront of medical research into genetic diseases, collaborating with esteemed institutions and companies such as Kyowa Kirin Co., Ltd., Saint Louis University, Baylor Research Institute, REGENXBIO Inc., and others to advance its mission. This fruitful collaboration ensures a robust pipeline of therapeutic candidates designed to address some of the most challenging and underserved medical needs.
Products and Services
The innovation at Ultragenyx spans several biologic products targeting a variety of rare genetic diseases, alongside a diverse pipeline of product candidates in various stages of development:
- Crysvita (burosumab) - This antibody targets fibroblast growth factor 23 for the treatment of X-linked hypophosphatemia and tumor-induced osteomalacia, offering new hope to patients suffering from these rare bone disorders.
- Mepsevii - An enzyme replacement therapy designed for children and adults with Mucopolysaccharidosis VII, Mepsevii represents a critical advancement in treating this lysosomal storage disorder.
- Dojolvi - Tailored for individuals with long-chain fatty acid oxidation disorders, Dojolvi provides a novel dietary management solution to support those affected by this metabolic condition.
- Evkeeza (evinacumab) - For patients grappling with homozygous familial hypercholesterolemia, Evkeeza offers a groundbreaking monoclonal antibody treatment aimed at reducing LDL cholesterol levels.
- DTX401 - This adeno-associated virus 8 (AAV8) gene therapy clinical candidate aims to cure glycogen storage disease type Ia, demonstrating Ultragenyx's commitment to gene therapy solutions.
- DTX301 - Another AAV8 gene therapy, designed for patients with ornithine transcarbamylase deficiency, showcases the company's innovative approach to genetic metabolic disorders.
- UX143 - A human monoclonal antibody in development for the treatment of osteogenesis imperfecta, illustrating Ultragenyx's focus on genetic disorders affecting bone density and growth.
- GTX-102 - An antisense oligonucleotide aimed at treating Angelman syndrome, this prospective therapy offers hope for a condition currently lacking specific treatments.
- UX111 - An AAV9 gene therapy product candidate for Sanfilippo syndrome type A, or MPS IIIA, represents a potential breakthrough for this rare lysosomal storage disease.
- UX701 - Focused on Wilson disease, UX701 is a gene therapy candidate that could offer a significant improvement over current treatment options for this genetic disorder affecting copper metabolism.
- UX053 - For patients with glycogen storage disease type III, UX053 represents another critical gene therapy effort by Ultragenyx to address unmet medical needs in metabolic disorders.
