Ultragenyx PharmaceuticalNASDAQ: RARE

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 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.

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) 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.

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.

NOVATO, Calif., Oct. 03, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today reported that the Phase 1/2/3 Cyprus 2+ study of its UX701 gene therapy has demonstrated meaningful clinical activity as well as improvements in copper metabolism in Stage 1. Multiple responders have completely tapered off of standard-of-care treatment with responses seen in all three dose cohorts. The company plans to enroll an additional cohort in Stage 1 at a moderately increased dose and with an optimized immunomodulation regimen to enhance the efficiency and efficacy of the gene therapy, with the objective of having the majority of patients come off of standard-of-care treatment before selecting a dose for the randomized placebo-controlled stage of the study.

NOVATO, Calif., Sept. 26, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced that it will present seven abstracts related to its ongoing late-stage program evaluating setrusumab (UX143) and osteogenesis imperfecta (OI), including a late-breaker oral presentation of the 14-month data from the Phase 2/3 Orbit study, at the American Society for Bone and Mineral Research (ASBMR) 2024 Annual Meeting. The meeting is being held September 27-30, 2024, in Toronto, Canada.

THOUSAND OAKS, Calif. , Sept. 23, 2024 /PRNewswire/ -- Amgen (NASDAQ:AMGN) will host a webcasted call for the investment community at 1:30 p.m.

Morgan Stanley 22 nd Annual Global Healthcare Conference on September 4 Cantor Global Healthcare Conference on September 17 Bank of America Global Healthcare Conference on September 18 NOVATO, Calif., Aug. 29, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for serious rare and ultrarare genetic diseases, today announced participation in three upcoming investor conferences.