Welcome to our dedicated page for Ultragenyx Pharm news (Ticker: RARE), a resource for investors and traders seeking the latest updates and insights on Ultragenyx Pharm stock.
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) is a biopharmaceutical company focused on novel therapies for serious rare and ultra-rare genetic diseases. The RARE news feed highlights company announcements on commercial performance, clinical development, and key corporate decisions that shape its rare disease portfolio.
Investors and followers of Ultragenyx can find updates on revenue trends from its approved products Crysvita, Dojolvi, Mepsevii, and Evkeeza in select territories, as well as guidance ranges and commentary on operating expenses and cash position. Earnings-related press releases and Form 8-K summaries detail product sales, royalty revenue, and royalty financing transactions, including agreements involving future Crysvita royalties with OMERS.
The news stream also covers Ultragenyx’s late-stage pipeline and regulatory milestones. Readers can track progress of AAV gene therapy programs such as DTX401 for glycogen storage disease type Ia, UX111 for Sanfilippo syndrome type A, and UX701 for Wilson disease, along with monoclonal antibody UX143 for osteogenesis imperfecta and antisense oligonucleotide GTX-102 for Angelman syndrome. Company releases describe Phase 3 study results, longer-term follow-up data, regulatory designations, rolling BLA submissions, and responses to FDA communications such as Complete Response Letters.
In addition, the RARE news page features items on conference presentations, investor events, and equity inducement grants under Nasdaq Listing Rule 5635(c)(4). By reviewing these updates, users can follow how Ultragenyx manages its commercial portfolio, advances its clinical pipeline, and executes financing and collaboration strategies in the rare and ultra-rare disease space.
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company specializing in novel products for rare and ultrarare genetic diseases, has announced a conference call scheduled for August 1, 2024, at 5:00 p.m. ET. The call will cover the company's financial results and corporate update for the second quarter of 2024, ending June 30. Investors and interested parties can access the live webcast and replay through the company's investor relations website. The replay will remain available for three months following the call, providing extended access to the information presented.
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) will present updates on its GTX-102 Angelman syndrome program at the 2024 Angelman Syndrome Foundation (ASF) Family Conference and Research Symposium on July 24, 2024. The presentations will include:
1. A review of positive interim Phase 1/2 results previously disclosed at the American Academy of Neurology Meeting in April.
2. An update on plans to initiate a Phase 3 pivotal trial by the end of 2024.
3. Insights on patient progress, with continued development of new skills across multiple domains and no new serious adverse events reported.
Dr. Eric Crombez, Chief Medical Officer at Ultragenyx, expressed enthusiasm about working with the ASF and the broader community as they prepare for a global randomized study. The presentations will be livestreamed on the ASF website.
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company specializing in rare and ultrarare diseases, has announced the grant of 19,875 restricted stock units to 14 newly hired non-executive officers. The awards, approved by the company's compensation committee, were granted under the Ultragenyx Employment Inducement Plan on July 16, 2024. This move aligns with Nasdaq Listing Rule 5635(c)(4), serving as an inducement for new employees joining Ultragenyx.
The restricted stock units have a four-year vesting period, with 25% of the underlying shares vesting annually on the grant date anniversary. Continuous employment with Ultragenyx is required for vesting. This grant demonstrates the company's commitment to attracting and retaining talent in the competitive biopharmaceutical industry.
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) announced a successful end-of-Phase 2 meeting with the FDA for its GTX-102 Angelman syndrome program. The company achieved alignment on the Phase 3 study design, including the primary endpoint of Bayley-4 cognition and key secondary endpoint of Multi-Domain Responder Index (MDRI). The Phase 3 study is set to begin by the end of 2024, involving a global, randomized, double-blind, sham-controlled trial with approximately 120 patients with Angelman syndrome caused by full maternal UBE3A gene deletion. The study will have a 48-week primary efficacy analysis period. Ultragenyx also plans to initiate an open-label study for patients with other Angelman syndrome genotypes and age groups.
Ultragenyx Pharmaceutical announced the grant of 45,525 restricted stock units (RSUs) to 26 newly hired non-executive employees.
The awards, approved by the compensation committee, were granted under the company's Employment Inducement Plan on June 16, 2024, as per Nasdaq Listing Rule 5635(c)(4).
The RSUs will vest over four years, with 25% vesting each year on the anniversary of the grant date, contingent on the employees' continued employment.
Ultragenyx Pharmaceutical announced the pricing of its public offering of 7,435,898 shares of common stock at $39.00 per share, and pre-funded warrants for 1,538,501 shares at $38.999 each. The company expects to raise $350 million in gross proceeds before expenses and underwriting discounts. Additionally, underwriters have a 30-day option to purchase up to 1,346,153 additional shares at the public offering price. The offering is set to close around June 17, 2024, subject to customary conditions. J.P. Morgan, Goldman Sachs, BofA Securities, and TD Cowen are joint book-running managers.
Ultragenyx Pharmaceutical has announced a public offering of common stock and pre-funded warrants worth up to $350 million. An additional 30-day option allows underwriters to purchase up to $52.5 million more shares. The offering's completion is subject to market conditions. J.P. Morgan, Goldman Sachs, BofA Securities, and TD Cowen are managing the offering. A registration statement for these securities became effective on February 21, 2024. The offering will be conducted via a prospectus supplement and accompanying prospectus.
Ultragenyx announced plans to file for accelerated approval of UX111 for treating Sanfilippo Syndrome Type A (MPS IIIA) following a successful FDA meeting. The FDA agreed that cerebral spinal fluid (CSF) heparan sulfate (HS) is a valid surrogate endpoint for this submission. Ultragenyx aims to file its biologics license application (BLA) for UX111 by late 2024 or early 2025. The submission will leverage data from ongoing clinical trials, including the Transpher A study. This decision marks a significant milestone in developing treatments for neuronopathic mucopolysaccharidoses, highlighting the urgency of delivering therapies for severe neurological diseases in children.
Ultragenyx and Mereo BioPharma announced positive 14-month results from their ongoing Phase 2/3 Orbit study of setrusumab (UX143) for Osteogenesis Imperfecta (OI). The treatment showed a substantial 67% reduction in annualized fracture rate and a median annualized fracture rate of 0.00 (p=0.0014). Bone mineral density (BMD) improved by 22% on average (p<0.0001), with a mean Z-score increase of +1.25 (p<0.0001).
The study, which included 24 patients treated for at least 14 months, demonstrated sustained reductions in fractures. No treatment-related serious adverse events were reported, and adverse events were consistent with previous studies.
Ultragenyx Pharmaceutical (NASDAQ: RARE) announced its participation in the Goldman Sachs 45th Annual Global Healthcare Conference. Dr. Emil Kakkis, the CEO, will engage in a fireside chat on June 11, 2024, at 10:00 a.m. ET. The event will be live-streamed and available for later viewing on Ultragenyx's investor relations website.