Ultragenyx Pharmaceutical Inc. (RARE)
Ultragenyx Pharmaceutical: The Market Is Overlooking 2026 Catalysts
Ultragenyx Pharmaceutical remains a speculative conviction hold (3/5) as its commercial base funds a robust late-stage rare disease pipeline. Multiple pivotal catalysts in 2026–2027, including GTX-102, DTX401, and UX111, could drive significant revenue growth and shift sentiment. Conservative U.S.-only modeling suggests $1.5B–$2.4B peak annual revenue potential from late-stage programs, with Street estimates supporting multi-bagger upside.
RARE Rises as FDA Accepts Resubmitted BLA for Sanfilippo Syndrome
Ultragenyx Pharmaceutical jumps 5% as the FDA accepts UX111 BLA resubmission, setting up a September 2026 decision for a potential first treatment for MPS IIIA.
Ultragenyx Pharmaceutical Announces FDA Acceptance of UX016 IND
RARE's IND for UX016, a novel sialic acid prodrug targeting GNEM, a rare muscle disease, gets accepted by the FDA. The first-in-human study is slated for H2'26.
Ultragenyx Pharmaceutical Inc. $RARE Shares Sold by B Group Inc.
B Group Inc. reduced its stake in Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) by 24.0% in the undefined quarter, according to the company in its most recent disclosure with the Securities and Exchange Commission. The firm owned 135,000 shares of the biopharmaceutical company's stock after selling 42,600 shares during the period. Ultragenyx Pharmaceutical makes
RARE's DTX301 Meets First Key Goal in Phase III OTC Deficiency Study
Ultragenyx Pharmaceutical reports phase III success for DTX301 in OTC deficiency, lowering ammonia 18% compared to placebo and keeping levels normal through 36 weeks.
Ultragenyx's Gene Therapy Study Shows Ammonia Reduction In Rare Metabolic Disorder
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) shares are down on Thursday as the company reported data from its Phase 3 study of DTX301, a gene therapy for ornithine transcarbamylase (OTC) deficiency.
Ultragenyx's gene therapy helps control ammonia levels in late-stage study
Ultragenyx Pharmaceutical said on Thursday its experimental gene therapy helped patients in a late-stage trial to better control ammonia levels linked to a rare inherited disorder.
Ultragenyx Pharmaceutical Inc. (RARE) Presents at Barclays 28th Annual Global Healthcare Conference Transcript
Ultragenyx Pharmaceutical Inc. (RARE) Presents at Barclays 28th Annual Global Healthcare Conference Transcript
Ultragenyx Pharmaceutical Inc. (RARE) Presents at TD Cowen 46th Annual Health Care Conference Transcript
Ultragenyx Pharmaceutical Inc. (RARE) Presents at TD Cowen 46th Annual Health Care Conference Transcript
Ultragenyx Pharmaceutical Inc. (RARE) Q4 2025 Earnings Call Transcript
Ultragenyx Pharmaceutical Inc. (RARE) Q4 2025 Earnings Call Transcript
Ultragenyx Q4 Loss Wider Than Expected, Revenues Increase Y/Y
RARE posts a wider-than-expected Q4 loss even as revenues jump 25% to $207 million, beating estimates. It also announces a strategic restructuring plan targeting profitability in 2027.
Ultragenyx (RARE) Reports Q4 Earnings: What Key Metrics Have to Say
Although the revenue and EPS for Ultragenyx (RARE) give a sense of how its business performed in the quarter ended December 2025, it might be worth considering how some key metrics compare with Wall Street estimates and the year-ago numbers.
