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Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), founded in 2010, is a clinical-stage biotechnology company dedicated to developing and commercializing innovative therapies for the treatment of serious rare and ultra-rare genetic diseases. The company’s core focus is on debilitating metabolic genetic diseases, for which there are high unmet medical needs, and the biology for treatment is well-understood but lacks approved therapies. Ultragenyx's management team brings extensive experience in rare disease therapeutics.
The company’s diverse product portfolio includes approved medicines like Crysvita, Dojolvi, and Mepsevii. Crysvita is designed for the treatment of X-linked hypophosphatemia (XLH), a rare genetic bone disease, and has shown significant growth with revenue reaching $328 million in 2023. Dojolvi targets long-chain fatty acid oxidation disorders and saw a 27% revenue increase in 2023. Mepsevii treats Mucopolysaccharidosis VII, another severe genetic disorder.
Recent Achievements and Financial Performance
In 2023, Ultragenyx reported total revenues of $434 million, a 20% increase from the previous year. The company has a robust pipeline with several candidates in late-stage clinical trials. For instance, the Phase 3 Orbit study for UX143 (setrusumab) in Osteogenesis Imperfecta (OI) completed enrollment and showed promising interim results with a 67% reduction in fracture rates.
Ongoing Projects
- GTX-102: An antisense oligonucleotide for Angelman syndrome showed significant improvements in clinical trials, with plans for a Phase 3 study in 2024.
- UX701: A gene therapy for Wilson disease, with data expected in mid-2024.
- DTX401: A gene therapy for Glycogen Storage Disease Type Ia (GSDIa), achieved its primary endpoint in a Phase 3 study.
- Setrusumab (UX143): Demonstrated significant fracture rate reduction and bone mineral density improvement in OI patients.
Partnerships and Collaborations
Ultragenyx collaborates with various regulatory bodies and advocacy groups to support patients and ensure high-quality clinical trials. The company’s partnership with Mereo BioPharma on setrusumab exemplifies its collaborative approach to drug development.
Corporate Responsibility
Ultragenyx's 2023 Corporate Responsibility Report highlights its commitment to innovation, patient support, diversity, and environmental sustainability. The company has been recognized with several awards, including the Top Places to Work by the Boston Globe and Healthcare’s Best Companies to Work by U.S. News & World Report.
For more information, visit www.ultragenyx.com.
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.
Ultragenyx announced positive top-line results from its Phase 3 GlucoGene study evaluating DTX401, a gene therapy for glycogen storage disease type Ia (GSDIa).
The therapy showed a statistically significant reduction in daily cornstarch intake at Week 48 compared to placebo (41.3% vs. 10.3%).
DTX401 also improved glucose control and reduced the number of daily cornstarch doses. Safety profile was consistent with previous studies, showing non-serious, manageable effects.
The results will be discussed with regulatory authorities for a marketing application in 2025.
Ultragenyx Pharmaceutical, a biopharmaceutical company focused on therapies for rare diseases, announced the inducement grant of 17,180 restricted stock units (RSUs) to ten newly hired non-executive officers. The awards, approved by the board's compensation committee under the Ultragenyx Employment Inducement Plan, were granted on May 16, 2024. These RSUs vest over four years, with 25% vesting annually, conditional on continuous employment. This grant aligns with Nasdaq Listing Rule 5635(c)(4), serving as a key employment incentive.
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) will have its chief medical officer, Eric Crombez, participate in a fireside at Bank of America’s 2024 Healthcare Conference on May 14, 2024. The company focuses on developing therapies for rare genetic diseases.
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