Ultragenyx Pharmaceutical Inc. (RARE)
RARE Reports New Upbeat Data From Sanfilippo Syndrome Drug Study
Ultragenyx announces new safety and efficacy data from its pivotal study of its investigational candidate, UX111, for patients with Sanfilippo syndrome type A.
Ultragenyx's Multiple Commercial Assets And Candidates Impress Analyst; Sees Solid Upside
In conjunction with the 2025 J.P. Morgan Healthcare Conference, the analyst hosted an investor lunch with Ultragenyx Pharmaceutical Inc RARE.
RARE Submits BLA to the FDA for Sanfilippo Syndrome Gene Therapy
Ultragenyx submits a BLA to the FDA seeking the accelerated approval of UX111 for patients with Sanfilippo syndrome type A in the United States.
Ultragenyx: Ready For More Growth After Q3 Earnings Beat
Ultragenyx's strong Q3 earnings report, with a 42% YoY revenue increase, has reignited my interest in initiating a starter position in RARE. The company's promising pipeline, including late-stage programs like Setrusumab and DTX401, presents multiple catalysts for future growth and higher valuation. Ultragenyx's $825M cash position and path to profitability by 2026 suggest financial stability, potentially avoiding the need for extensive dilutive financing.
Ultragenyx Q3 Loss Narrower Than Expected, Revenues Beat Estimates
RARE reports encouraging third-quarter results, beating earnings and revenue estimates. Product sales increase year over year.
Ultragenyx Pharmaceutical Inc. (RARE) Q3 2024 Earnings Call Transcript
Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE ) Q3 2024 Earnings Conference Call November 5, 2024 5:00 PM ET Company Participants Joshua Higa - VP, IR Emil Kakkis - CEO and President Erik Harris - CCO Howard Horn - CFO Eric Crombez - CMO Conference Call Participants Chris Raymond - Piper Sandler Tazeen Ahmad - Bank of America Malcolm Kuno - JPMorgan Gena Wang - Barclays Lydia Erdman - Goldman Sachs Maury Raycroft - Jefferies Mehdi Goudarzi - Truist Securities Yaron Werber - TD Cowen Yigal Nochomovitz - Citi Thomas Yip - H.C. Wainwright Jack Allen - Baird Joe Schwartz - Leerink Partners Michael Riad - Morgan Stanley Kristen Kluska - Cantor Fitzgerald Dae Gon Ha - Stifel Operator Good afternoon and welcome to the Ultragenyx Third Quarter 2024 Financial Results Conference Call.
Compared to Estimates, Ultragenyx (RARE) Q3 Earnings: A Look at Key Metrics
While the top- and bottom-line numbers for Ultragenyx (RARE) give a sense of how the business performed in the quarter ended September 2024, it could be worth looking at how some of its key metrics compare to Wall Street estimates and year-ago values.
Ultragenyx (RARE) Reports Q3 Loss, Tops Revenue Estimates
Ultragenyx (RARE) came out with a quarterly loss of $1.40 per share versus the Zacks Consensus Estimate of a loss of $1.45. This compares to loss of $2.23 per share a year ago.
RARE's Bone Disorder Drug Gets FDA's Breakthrough Therapy Tag
Ultragenyx announces that the FDA grants Breakthrough Therapy designation to its investigational osteogenesis imperfecta treatment candidate, setrusumab.
RARE's Wilson Disease Candidate Betters Standard Therapy in Study
Ultragenyx reports superior efficacy of UX701 for Wilson disease compared with standard therapy from the Stage 1 cohorts of its pivotal phase I/II/III study.
Ultragenyx's Strong Pipeline And Valuation Concerns: A Balanced Perspective
Ultragenyx develops therapies for rare genetic diseases, with a portfolio including Crysvita, Mepsevii, Dojolvi, and Evkeeza. The company's promising pipeline targets severe diseases like osteogenesis imperfecta and Angelman syndrome, and it has progressed well in clinical trials. The company's financials show a strong cash position but a high P/B and P/S ratios, as well as significant future royalty liabilities on Crysvita.
3 Genomics Stocks That May Transform Personalized Medicine
When the human genome was fully sequenced in 2003, the prospects of genetic medicine ushered in a new era of human health. Today, scientists and drug developers have a better understanding of the genetic drivers of disease than ever before.
