Ultragenyx PharmaceuticalNASDAQ: RARE

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.

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.

Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE ) Q2 2024 Earnings Conference Call August 1, 2024 5:00 PM ET Company Participants Joshua Higa - Vice President, Investor Relations Emil Kakkis - Chief Executive Officer & President Erik Harris - Chief Commercial Officer Howard Horn - Chief Financial Officer Eric Crombez - Chief Medical Officer Conference Call Participants Gena Wang - Barclays Salveen Richter - Goldman Sachs Dae Gon Ha - Stifel Tazeen Ahmad - Bank of America Kristen Kluska - Cantor Fitzgerald Joon Lee - Truist Maury Raycroft - Jefferies Will Soghikian - Leerink Partners Yaron Werber - TD Cowen Michael Riad - Morgan Stanley Jack Allen - Baird Lisa Walter - RBC Operator Good afternoon, and welcome to the Ultragenyx Second Quarter 2024 Financial Results Conference Call. At this time, all participants are in a listen-only mode.

Ultragenyx (RARE) reports encouraging second-quarter 2024 results, beating both earnings and revenue estimates driven by the year-over-year increase of Crysvita and Dojolvi sales.

Although the revenue and EPS for Ultragenyx (RARE) give a sense of how its business performed in the quarter ended June 2024, it might be worth considering how some key metrics compare with Wall Street estimates and the year-ago numbers.