Ultragenyx Pharmaceutical (RARE) announced that it has reached alignment with the FDA regarding the phase III study design and endpoints for GTX-102, an antisense oligonucleotide for Angelman syndrome, following the completion of a successful end-of-phase II meeting with the regulatory body. The company expects to initiate the same by the end of 2024.In the meeting, Ultragenyx mainly discussed the positive interim data from the phase I/II study, announced in April 2024, with the FDA. The study evaluated the ...

NOVATO, Calif., June 13, 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 the pricing of its underwritten public offering of 7,435,898 shares of its common stock at a price to the public of $39.00 per share. In addition, in lieu of issuing common stock to certain investors, the company is offering pre-funded warrants to purchas ...

NOVATO, Calif., June 13, 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 the pricing of its underwritten public offering of 7,435,898 shares of its common stock at a price to the public of $39.00 per share. In addition, in lieu of issuing common stock to certain investors, the company is offering pre-funded warrants to purchas ...

Ultragenyx (RARE) shares soared 7.8% in the last trading session to close at $44.64. The move was backed by solid volume with far more shares changing hands than in a normal session. This compares to the stock's 1.1% gain over the past four weeks. The stock rallied after the company, along with its partner Mereo BioPharma, announced new positive 14- month data from the phase II portion of the phase II/III Orbit study evaluating setrusumab (UX143) in patients with osteogenesis imperfecta (OI). The data showe ...

NOVATO, Calif., June 12, 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 that it has commenced an underwritten public offering of up to $350,000,000 of shares of its common stock and, in lieu of issuing common stock to certain investors, pre- funded warrants to purchase shares of its common stock. In addition, the company is e ...

NOVATO, Calif., June 12, 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 that it has commenced an underwritten public offering of up to $350,000,000 of shares of its common stock and, in lieu of issuing common stock to certain investors, pre- funded warrants to purchase shares of its common stock. In addition, the company is e ...

Agreement reached with FDA that cerebral spinal fluid (CSF) heparan sulfate (HS) can be used as a reasonable surrogate endpoint for accelerated approval Data from existing clinical studies appear sufficient for a biologics license application (BLA) submission NOVATO, Calif., June 12, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced that the company held a successful meeting with the U.S. Food and Drug Administration (FDA or the Agency) during which the company reached a ...

Ultragenyx Pharmaceutical Inc. (RARE) , along with partner Mereo BioPharma (MREO), announced new positive 14-month data from the phase II portion of the phase II/III Orbit study evaluating setrusumab (UX143) in patients with osteogenesis imperfecta (OI). The data showed that the large reduction in fracture rates previously reported in patients treated for a minimum of six months was sustained in patients treated for at least 14 months. OI is a group of genetic disorders that impact bone metabolism. It is ch ...

Ariawan Armoko/iStock via Getty Images Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) is a good long-term biotech to own in my opinion. That's because it has already been able to receive regulatory approvals for several of its drugs in its pipeline. Matter of fact, the drugs being sold by the company are Crysvita and Dojolvi. In Q1 of 2024, it generated $109 million in total revenue, which was an 8% growth year-over-year. As such, if sales continue like this, it will continue to build shareholder value. If th ...

NOVATO, Calif., May 17, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for rare and ultrarare diseases, today reported the grant of 17,180 restricted stock units of the company's common stock to ten newly hired non-executive officers of the company. The awards were approved by the compensation committee of the company's board of directors and granted under the Ultragenyx Employment Inducem ...