Welcome to our dedicated page for Ultragenyx Pharmaceutical news (Ticker: RARE), a resource for investors and traders seeking the latest updates and insights on Ultragenyx Pharmaceutical stock.
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.
On August 17, 2022, Ultragenyx Pharmaceutical (NASDAQ: RARE) announced the granting of stock options and restricted stock units to two new non-executive officers. A total of 19,020 stock options and 12,910 restricted stock units were awarded under the Ultragenyx Employment Inducement Plan, following Nasdaq Listing Rule 5635(c)(4). The options have a ten-year term with an exercise price of $49.31, matching the closing price on the grant date. The vesting schedule includes a four-year period with certain conditions based on continued employment.
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) announced the grant of non-qualified stock options for 27,500 shares and 14,500 restricted stock units to two newly hired non-executive officers. This award follows the Ultragenyx Employment Inducement Plan and was approved by the compensation committee on August 1, 2022. The stock options have a ten-year term with an exercise price of $50.63 per share. The restricted stock units vest over four years, promoting employee retention and aligning interests with shareholders.
Ultragenyx Pharmaceutical reported Q2 2022 total revenue of $89.3 million, with Crysvita revenue at $64.0 million. They reaffirmed 2022 revenue guidance for Crysvita between $250 million and $260 million and Dojolvi between $55 million and $65 million. The company completed a $500 million royalty interest sale for Crysvita. Operating expenses reached $230.9 million, leading to a net loss of $158.2 million. The firm continues to develop its extensive pipeline, including late-stage products for various rare diseases.
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) will host a conference call on July 28, 2022, at 5:00 PM ET to discuss its financial results and corporate updates for Q2 2022. The call will be available via live webcast on the company's website. Ultragenyx focuses on developing therapies for rare genetic diseases, boasting a portfolio of approved medicines and candidates for unmet medical needs. The company emphasizes efficient drug development to deliver timely therapies to patients.
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) announced on July 20, 2022, the grant of stock options for 10,240 shares and 5,120 restricted stock units to a newly hired non-executive officer. These awards, part of the Ultragenyx Employment Inducement Plan, were approved by the compensation committee. The stock options have a ten-year term with an exercise price of $64.75 per share. The restricted stock units and options have a vesting schedule of four years, contingent on continued employment. This reflects the company’s commitment to attracting talent within the biopharmaceutical sector.
Ultragenyx Pharmaceutical (NASDAQ: RARE) has announced interim data from its Phase 1/2 study of GTX-102 for Angelman syndrome, showing good tolerability and clinical activity across multiple domains. The UK and Canadian health authorities have approved dose escalation to higher amounts, starting at 7.5 mg and reaching up to 14 mg. Ultragenyx has also completed a $75 million acquisition of GeneTx. Early results indicate significant improvements in clinical symptoms, with no treatment-related serious adverse events noted.
Ultragenyx Pharmaceutical (NASDAQ: RARE) announced the sale of 30% of its royalty interest from Kyowa Kirin on Crysvita sales in the U.S. and Canada to OMERS for $500 million. This deal, effective from April 2023, caps total payments at 1.45 times the purchase price. Crysvita has achieved over $1.3 billion in net sales in its first four years, marking a successful launch. The proceeds from this non-dilutive financing will support ongoing commercialization and clinical development activities.
NOVATO, Calif., June 10, 2022 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical, focused on rare genetic diseases, announced that CEO Emil D. Kakkis and CFO Mardi Dier will present at the Goldman Sachs Global Healthcare Conference on June 16, 2022, at 10:00 AM PDT in Palos Verdes, CA. The live and archived webcast will be available on the company’s website for 90 days. Ultragenyx aims to develop novel therapies for rare diseases with unmet medical needs, showcasing a commitment to effective and timely drug development.
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) announced that CEO Emil D. Kakkis will present at the Jefferies Global Healthcare Conference on June 8, 2022, at 2:30 PM ET, in New York. The presentation will focus on the company's commitment to developing therapies for serious rare genetic diseases. A live and archived webcast will be available on their website for 90 days post-event. Ultragenyx aims to deliver effective treatments efficiently, addressing high unmet medical needs in rare diseases.
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) revealed promising long-term efficacy and safety data for its investigational gene therapies DTX401 and DTX301 during recent presentations. Phase 1/2 studies show DTX401 for Glycogen Storage Disease Type Ia (GSDIa) sustained responses for over 3.5 years, with 70% mean reduction in cornstarch intake. DTX301 for Ornithine Transcarbamylase (OTC) deficiency demonstrated durable metabolic control for over four years, with significant improvements in patient responses. No serious adverse events reported across both studies, highlighting the therapies’ safety profiles.
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