Welcome to our dedicated page for Ultragenyx Pharm news (Ticker: RARE), a resource for investors and traders seeking the latest updates and insights on Ultragenyx Pharm 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) has announced the grant of 23,760 restricted stock units to 10 newly hired non-executive officers. The compensation committee approved these awards under the Ultragenyx Employment Inducement Plan, with a grant date of February 16, 2025. The grants, made in accordance with Nasdaq Listing Rule 5635(c)(4), serve as employment inducements for the new hires.
The restricted stock units have a four-year vesting schedule, with 25% of the shares vesting annually on the grant date anniversary, contingent on continuous employment.
Ultragenyx Pharmaceutical (NASDAQ: RARE) announced FDA acceptance of their Biologics License Application (BLA) for UX111, an AAV gene therapy targeting Sanfilippo syndrome type A (MPS IIIA). The FDA granted Priority Review with a PDUFA date of August 18, 2025, and indicated no current plans for an advisory committee meeting.
The BLA submission is supported by data from the pivotal Transpher A and long-term follow-up studies presented at WORLDSymposium™ 2025. Results showed rapid and sustained decreases in heparan sulfate levels in cerebrospinal fluid, with statistically significant improvements in Bayley-III scores for cognition and communication compared to untreated patients. Treatment-related adverse events were primarily mild to moderate liver enzyme elevations that resolved.
Ultragenyx Pharmaceutical (NASDAQ: RARE) reported strong financial results for Q4 and full year 2024, with total revenue of $560 million, representing 29% growth compared to 2023. Key revenue drivers included Crysvita at $410 million (+25% YoY) and Dojolvi at $88 million (+25% YoY).
The company provided 2025 guidance projecting total revenue between $640-670 million, with Crysvita revenue of $460-480 million and Dojolvi revenue of $90-100 million. Despite revenue growth, Ultragenyx reported a net loss of $569 million ($6.29 per share) for 2024, though improved from $607 million loss in 2023.
The company ended 2024 with $745 million in cash and equivalents, with net cash used in operations of $414 million for the year. Multiple clinical milestones are expected in 2025, including a PDUFA decision for Sanfilippo syndrome gene therapy and Phase 3 results in osteogenesis imperfecta.
Ultragenyx Pharmaceutical (NASDAQ: RARE), a biopharmaceutical company specializing in novel products for rare and ultrarare genetic diseases, has announced a conference call scheduled for February 13, 2025, at 5:00 p.m. ET. The call will discuss the company's financial results and provide a corporate update for both the fourth quarter and full year ending December 31, 2024. 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.
Ultragenyx Pharmaceutical (NASDAQ: RARE) announced significant clinical improvements in children treated with UX111 AAV gene therapy for Sanfilippo Syndrome Type A. The modified intent-to-treat group showed a +22.7 point treatment effect in Bayley-III cognitive raw scores compared to untreated patients, with a p-value <0.0001.
The treatment demonstrated substantial reductions in heparan sulfate levels in cerebrospinal fluid, with a median reduction of 65% across all patients (N=27) and 66% in the modified intention to treat group (N=17). The mean follow-up duration was 34 months for all patients, with the longest being 77 months.
In older patients with advanced disease, 9 out of 10 children retained abilities in ambulation and self-feeding, while all 10 maintained communication skills. The therapy was generally well-tolerated, with mostly mild to moderate liver enzyme elevations that resolved. A BLA submission was filed with the FDA in December 2024, with a PDUFA decision expected in H2 2025.
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.
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