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. Per RARE, the pivotal phase III sham-controlled study will evaluate the safety and primary efficacy of GTX102 in approximately 120 Angelman syndrome patients, ...

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 purcha ...

and, when available, its final prospectus supplement, and the documents incorporated by reference therein, including its Annual Report on Form 10-K filed with the Securities and Exchange Commission on February 21, 2024, and its subsequent periodic reports filed with the Securities and Exchange Commission. This press release shall not constitute an offer to sell or the solicitation of an offer to buy, nor shall there be any sale of these securities in any state or jurisdiction in which such offer, solicitati ...

For Ultragenyx, the consensus EPS estimate for the quarter has remained unchanged over the last 30 days. And a stock's price usually doesn't keep moving higher in the absence of any trend in earnings estimate revisions. So, make sure to keep an eye on RARE going forward to see if this recent jump can turn into more strength down the road. 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 stud ...

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 ...

A registration statement relating to these securities has been filed with the Securities and Exchange Commission and became automatically effective on February 21, 2024. This offering is being made solely by means of a prospectus supplement and accompanying prospectus. When available, copies of the preliminary prospectus supplement and the accompanying prospectus related to the offering may be obtained from J.P. Morgan Securities LLC, c/o Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, Ne ...

Agreement reached with FDA that cerebral spinal fluid (CSF) heparan sulfate (HS) can be used as a reasonable surrogate endpoint for accelerated approval For more information on Ultragenyx, please visit the company's website at: www.ultragenyx.com. Ultragenyx Forward-Looking Statements and Use of Digital Media Except for the historical information contained herein, the matters set forth in this press release, including statements related to Ultragenyx's expectations and projections regarding its future opera ...

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. 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). OI is a group of genetic disorders that impact bone metabolism. It is ch ...

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. This isn't the only program to drive the stock price higher, either. Another promising program would be the advancement of setrusumab for the treatment of pediatric and young adults of osteogenesis imperfecta [OI] subtypes I, ...

NOVATO, Calif., June 04, 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 Emil Kakkis, M.D., Ph.D., the company's chief executive officer, will participate in a fireside at the Goldman Sachs 45th Annual Global Healthcare Conference on Tuesday, June 11, 2024, at 10:00 a.m. ET. The live and archived webcast of the panel will ...