Ultragenyx Pharm Stock Price, News & Analysis

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.

Ultragenyx Pharmaceutical Inc. reported first quarter total revenue of $109 million with Crysvita® revenue at $83 million and Dojolvi® revenue at $16 million. The company reaffirmed its 2024 total revenue guidance of $500 million to $530 million, with Crysvita revenue between $375 million to $400 million and Dojolvi revenue at $75 million to $80 million. Positive Phase 1/2 data from the GTX-102 Angelman syndrome study was presented, showing significant improvement. The company continues to see revenue growth and has completed enrollment for its Phase 3 program in osteogenesis imperfecta.

Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) released its 2023 Corporate Responsibility Report, emphasizing its commitment to improving equity and access to innovation in rare diseases. The report showcases efforts in drug development, patient support, employee diversity, community impact, environmental sustainability, and governance. Ultragenyx received multiple awards for its corporate responsibility initiatives, including high rankings in the biotech industry and recognition as a top workplace. The company's dedication to rare disease medicine and community welfare is evident in its comprehensive report.