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.
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) has reported promising results from the Phase 1/2/3 Cyprus2+ study of its UX701 gene therapy for Wilson Disease. The study has shown meaningful clinical activity and improvements in copper metabolism in Stage 1. Notably, six patients have completely tapered off standard-of-care treatment, with responses observed across all three dose cohorts.
The company plans to enroll an additional cohort in Stage 1 with a moderately increased dose and an optimized immunomodulation regimen to enhance the therapy's efficiency and efficacy. The goal is to have the majority of patients discontinue standard-of-care treatment before selecting a dose for the randomized placebo-controlled stage.
UX701 has been well-tolerated, with no unexpected related treatment emergent adverse events or significant immunologic safety issues reported. The study's design includes three stages, with the primary efficacy endpoints being change in 24-hour urinary copper concentration and percent reduction in standard-of-care medication by Week 52 of Stage 2.
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) announced it will present seven abstracts related to its setrusumab (UX143) program and osteogenesis imperfecta (OI) at the American Society for Bone and Mineral Research (ASBMR) 2024 Annual Meeting. The presentations include a late-breaker oral presentation of 14-month data from the Phase 2/3 Orbit study, which demonstrated a rapid and clinically meaningful increase in bone mineral density and a corresponding decrease in annualized fracture rate.
The abstracts cover various aspects of OI, including:
- Integrated bone biomarker analyses
- Burden of illness in Ontario, Canada
- Manifestations and comorbid conditions among OI patients
- Fracture rates in OI patients
- Population pharmacokinetics analyses
- Healthcare resource use and costs for OI patients
The ASBMR 2024 Annual Meeting will be held from September 27-30, 2024, in Toronto, Canada.
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company specializing in rare and ultrarare diseases, has announced the grant of 62,555 restricted stock units to 16 newly hired non-executive officers. The grants were approved by the company's compensation committee and issued under the Ultragenyx Employment Inducement Plan on September 16, 2024. This action complies 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 schedule, with 25% of the shares vesting annually, contingent on continuous employment.
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on developing therapies for rare genetic diseases, has announced its participation in three upcoming investor conferences in September 2024:
- Morgan Stanley 22nd Annual Global Healthcare Conference on September 4
- Cantor Global Healthcare Conference on September 17
- Bank of America Global Healthcare Conference on September 18
Key executives, including CEO Emil Kakkis, CFO Howard Horn, and CMO Eric Crombez, will participate in fireside chats and host one-on-one meetings. Live and archived webcasts of the fireside chats will be available on the company's investor relations website. Ultragenyx is committed to developing novel products for serious rare and ultrarare genetic diseases, with a diverse portfolio of approved therapies and product candidates.
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company specializing in rare and ultrarare disease therapies, has granted 37,975 restricted stock units to 12 newly hired non-executive officers. The awards, approved by the company's compensation committee, were issued under the Ultragenyx Employment Inducement Plan on August 16, 2024. This grant aligns with Nasdaq Listing Rule 5635(c)(4) and serves as an employment inducement. The restricted stock units have a four-year vesting schedule, with 25% of the underlying shares vesting annually, contingent on continuous employment with Ultragenyx.
Amlogenyx Inc., a subsidiary of Ultragenyx Pharmaceutical (NASDAQ: RARE), has successfully closed a $14 million seed funding round led by GordonMD® Global Investments LP. The company is developing novel gene therapies for Alzheimer's disease and other amyloid diseases. Amlogenyx's first product candidate is a protease developed in collaboration with St. Jude Children's Research Hospital, which has shown promising results in breaking down Aβ42, a protein believed to be responsible for Alzheimer's effects.
Dr. Craig Gordon, Founder and CIO of GordonMD®, expressed excitement about the collaboration and the potential of Amlogenyx's Alzheimer's disease therapeutic. The funding round also included participation from Ultragenyx and associated investors, demonstrating strong support for Amlogenyx's innovative approach to treating amyloid diseases.
Ultragenyx reported second quarter 2024 financial results with total revenue of $147 million, up 36% YoY. Key revenue drivers were Crysvita ($114 million, +37%) and Dojolvi ($19 million, +17%). The company raised its 2024 revenue guidance to $530-$550 million from $500-$530 million.
Ultragenyx also disclosed significant clinical advancements including positive data from Phase 1/2 study in Angelman syndrome, Phase 2/3 study in osteogenesis imperfecta, and Phase 3 study in GSDIa. Phase 3 studies for Angelman syndrome and UX111 for Sanfilippo syndrome type A are on track to start by end of 2024.
The company reported a net loss of $132 million, down from $160 million YoY. Operating expenses were $263 million, including $39 million in non-cash stock-based compensation. Cash reserves stood at $874 million as of June 30, 2024.
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.
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