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 (NASDAQ: RARE), a biopharmaceutical company specializing in rare and ultrarare disease therapies, has announced the grant of 7,750 restricted stock units to eight newly hired non-executive officers. The compensation committee approved these awards under the Ultragenyx Employment Inducement Plan on January 16, 2025. The grants serve as employment inducements in compliance with Nasdaq Listing Rule 5635(c)(4). The restricted stock units feature a four-year vesting schedule, with 25% of shares vesting annually on the grant date anniversary, contingent on continuous employment.
Ultragenyx Pharmaceutical (NASDAQ: RARE) reported preliminary 2024 revenue of $555-560 million, exceeding guidance, with Crysvita revenue of $405-410 million and Dojolvi revenue of $87-89 million. The company projects 2025 total revenue of $640-670 million.
Key pipeline updates include: UX143 Phase 3 Orbit study for osteogenesis imperfecta progressing to second interim analysis in mid-2025; GTX-102 Phase 3 Aspire study for Angelman syndrome expected to complete enrollment in second half of 2025; BLA submission for UX111 gene therapy for Sanfilippo syndrome with PDUFA decision expected in second half of 2025; and DTX401 BLA filing for Glycogen Storage Disease Type Ia planned for mid-2025.
The company ended 2024 with approximately $745 million in cash and investments, expecting 14-20% revenue growth in 2025 with decreased net cash usage in operations.
Ultragenyx Pharmaceutical (NASDAQ: RARE) has announced its participation in the 43rd Annual J.P. Morgan Healthcare Conference. The company's CEO and president, Emil D. Kakkis, M.D., Ph.D., will deliver a presentation on Monday, January 13, 2025, at 10:30 AM PT.
The presentation will be accessible via live webcast and can be viewed through the company's investor relations website at https://ir.ultragenyx.com/events-presentations. A replay option will remain available for 30 days following the event. Ultragenyx specializes in developing and commercializing novel therapies for serious rare and ultrarare genetic diseases.
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) announced that the European Commission has extended the approval of Evkeeza® (evinacumab) to treat children aged 6-months and older with homozygous familial hypercholesterolemia (HoFH). Evkeeza is the first medicine in the EU approved for children as young as 6-months old with this ultrarare inherited condition that causes dangerously high cholesterol levels.
The approval is based on model-based extrapolation analysis predicting similar or higher LDL-C reduction in young patients compared to adults when receiving a 15 mg/kg dose every 4 weeks. Supportive data from five patients aged 1-4 years showed clinically meaningful LDL-C reduction consistent with older patients. The safety profile is expected to be similar to that in older pediatric patients.
Evkeeza is currently reimbursed and commercially available in eight countries including the UK, U.S., Canada, and Japan, with early access schemes in 13 additional countries.
Ultragenyx Pharmaceutical (NASDAQ: RARE) announced the grant of 15,175 restricted stock units to 15 newly hired non-executive officers. The awards were approved by the company's compensation committee and granted under the Ultragenyx Employment Inducement Plan on December 16, 2024. The stock units will vest over four years, with 25% vesting annually on each grant date anniversary, contingent on continuous employment. This grant was made in accordance with Nasdaq Listing Rule 5635(c)(4) as an employment inducement.
Ultragenyx Pharmaceutical (NASDAQ: RARE) has initiated patient dosing in its pivotal Phase 3 Aspire study evaluating GTX-102 for Angelman syndrome. The global study will enroll approximately 120 children aged 4-17 with genetically confirmed maternal UBE3A gene deletion. Participants will be randomized 1:1 to receive GTX-102 or sham treatment over 48 weeks.
The treatment group will receive three monthly 8mg loading doses, followed by maintenance doses up to 14mg quarterly. The study's primary endpoint focuses on cognitive improvement measured by Bayley-4 cognitive raw score, with a key secondary endpoint using the Multi-domain Responder Index across cognition, communication, behavior, motor function, and sleep. The company plans to initiate the Aurora study in 2025 to evaluate GTX-102 in other Angelman syndrome genotypes and age groups.
Ultragenyx Pharmaceutical (NASDAQ: RARE) has submitted a Biologics License Application (BLA) to the FDA for UX111, an AAV gene therapy targeting Sanfilippo Syndrome Type A (MPS IIIA). If approved, UX111 would become the first authorized treatment in the U.S. for this condition.
The submission follows FDA's acceptance of cerebral spinal fluid (CSF) heparan sulfate (HS) as a surrogate endpoint for accelerated approval. Data from the ongoing pivotal Transpher A study showed that UX111 treatment led to rapid and sustained decreases in CSF HS levels, correlating with improved long-term cognitive development compared to natural history data. Most treatment-related adverse events were mild to moderate liver enzyme elevations that resolved.
Ultragenyx Pharmaceutical (NASDAQ: RARE) has announced its participation in three major investor conferences in December 2024. The company will attend Citi's 2024 Global Healthcare Conference in Miami, the 7th Annual Evercore ISI HealthCONx Conference in Coral Gables, and Piper Sandler's 36th Annual Healthcare Conference in New York.
Key executives including CEO Emil Kakkis, CFO Howard Horn, and CMO Eric Crombez will participate in fireside chats and host one-on-one meetings across these events. Live and archived webcasts will be available on the company's investor relations website.
Ultragenyx Pharmaceutical (NASDAQ: RARE) announced the grant of 20,820 restricted stock units to 13 newly hired non-executive officers. The compensation committee approved these awards under the Ultragenyx Employment Inducement Plan, with a grant date of November 16, 2024. The stock units will vest over four years, with 25% of shares vesting annually, contingent on continuous employment. This grant was made in accordance with Nasdaq Listing Rule 5635(c)(4) as an employment inducement.
Ultragenyx Pharmaceutical (NASDAQ: RARE) presented positive Phase 1/2 data for GTX-102, its investigational treatment for Angelman syndrome, at the FAST Global Science Summit. The data supports the upcoming Phase 3 Aspire study, showing significant improvements across multiple domains at Week 48. Patients (n=40) demonstrated a mean change of +6.7 in Bayley-4 Cognition GSV score and +10.9 in Raw score. The Phase 3 study is powered at 95% to detect treatment effects. In Expansion Cohorts A&B, 80% of patients (22 of 28) achieved clinically meaningful improvement in at least one domain, with a total net response of +2.0. The Phase 3 trial will enroll 120 patients and is set to begin by year-end.
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