Ultragenyx Pharmaceutical Inc. (RARE)
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.
Ultragenyx (RARE) Reports Q4 Loss, Beats Revenue Estimates
Ultragenyx (RARE) came out with a quarterly loss of $1.29 per share versus the Zacks Consensus Estimate of a loss of $1.2. This compares to a loss of $1.39 per share a year ago.
Ultragenyx (RARE) May Report Negative Earnings: Know the Trend Ahead of Q4 Release
Ultragenyx (RARE) doesn't possess the right combination of the two key ingredients for a likely earnings beat in its upcoming report. Get prepared with the key expectations.
Ultragenyx Pharmaceutical Resubmits UX111 BLA for Sanfilippo Syndrome
RARE resubmits FDA filing for UX111 after CRL, adding long-term neurologic and biomarker data, with a Q3 2026 decision in sight.
Ultragenyx: From Bone To Brain, Capitulation Creates A Free Option On The Neuro Pipeline
Ultragenyx is rated BUY with a $55 price target, offering 145% implied upside after a sharp post-trial selloff. RARE's commercial base, anchored by Crysvita and Dojolvi, provides a robust revenue safety net, with 2025 guidance above consensus and 20% projected annual growth. Key pipeline catalysts include GTX-102 Phase 3 data (Angelman's, H2 2025), GSDIa BLA submission, and Sanfilippo Syndrome Type A BLA resubmission, all with high success probabilities.
