Ultragenyx Pharmaceutical Inc. (RARE)
Summary
RARE Stock Gains on FDA Nod for Rare Disease Gene Therapy Genglycos
Ultragenyx stock rises as Genglycos wins FDA's accelerated approval as the first treatment targeting the underlying cause of GSDIa, boosting its rare-disease portfolio.
Ultragenyx Wins FDA Approval for GENGLYCOS, Its First Gene Therapy
Ultragenyx Pharmaceutical NASDAQ: RARE said the U.S. Food and Drug Administration has approved GENGLYCOS, also known as DTX401, for glycogen storage disease type 1A, or GSD1A. The approval marks the company's first gene therapy approval and its fifth approved medicine.
Ultragenyx: Undervalued Ahead Of Two FDA Decisions
Ultragenyx Pharmaceutical is rated a soft Buy at $26.75, with a probability-weighted fair value of ~$32 per share. Two near-term FDA decisions: DTX401 on Aug 23 and another for UX111 on Sept 19. DTX401 has a cleaner regulatory case, but UX111 has a credible approval path. Using 80% approval odds for DTX401 and 65% for UX111, our probability-weighted value is approximately $32 per share versus a current share price near $27.
Ultragenyx Pharmaceutical Inc. Investors
| Name | Quantity | Cost | Value | Profit ($) | Gain (%) |
|---|---|---|---|---|---|
 Curtis Ellergodt Rothschild Investment LLC | 1,116 | $23,513.26 | $29,607.48 | $6,094.22 | 25.92% |
| ABB Alexander Bjornager Bonde Danske Bank A/S | 100 | $2,300 | $2,653 | $353 | 15.35% |
| JD Jim Dushek HARBOUR INVESTMENTS Inc. | 30 | $854.74 | $779.05 | -$75.69 | -8.86% |
| SN Stephanie Nee HARBOR CAPITAL ADVISORS Inc. | 72,316 | $1.52M | $1.87M | $358,687.36 | 23.68% |
| BG Bart Gancher Intech Investment Management LLC | 17,316 | $502,683.5 | $464,068.8 | -$38,614.7 | -7.68% |
Ultragenyx Pharmaceutical Inc. Analysts
| Name | Quantity | Cost | Value | Profit ($) | Gain (%) |
|---|---|---|---|---|---|
 Kristen Kluska Cantor Fitzgerald | 2,907.5 | $76,351 | $77,136.03 | $785.03 | 1.03% |
 Anupam Rama JP Morgan | 1,502.14 | $38,815.38 | $39,851.86 | $1,036.48 | 2.67% |
| JH Jeffrey Hung Morgan Stanley | 3,095.81 | $79,995.84 | $81,760.46 | $1,764.62 | 2.21% |
 Yigal Nochomovitz Citigroup | 229.97 | $5,822.81 | $5,797.52 | -$25.29 | -0.43% |
Ultragenyx Pharmaceutical Inc. (RARE) FAQ
What is the stock price today?
On which exchange is it traded?
What is its stock symbol?
Does it pay dividends? What is the current yield?
What is its market cap?
Has Ultragenyx Pharmaceutical Inc. ever had a stock split?
Ultragenyx Pharmaceutical Inc. Profile
| Biotechnology Industry | Healthcare Sector | Emil D. Kakkis CEO | NASDAQ (NGS) Exchange | 90400D108 CUSIP |
| US Country | 1,371 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.
