Ultragenyx to Host Conference Call for Fourth Quarter and Full-Year 2025 Financial Results and Corporate Update NOVATO, Calif., Feb. 05, 2026 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultra-rare genetic diseases, today announced that it will host a conference call at 5:00 p.m. ET on Thursday, February 12, 2026, to discuss its financial results and corporate update for the quarter and the year ending December 31, 2025. The live and replayed webcast o...
Ultragenyx Announces Positive Longer-Term Data Demonstrating Treatment with UX111 Gene Therapy Results in Sustained, Significant Reductions in CSF-HS and Continued Meaningful Improvements in Clinical Function Across Multiple Developmental Domains in Children with Sanfilippo Syndrome (MPS IIIA) Data represents up to 8.5 years of follow-up and are consistent across age, dose, and genotype BLA resubmitted to U.S. FDA in January 2026; Company expects up to six-month review period per FDA guidelines NOVATO, Calif., Feb. 03, 2026 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. today annou...
Ultragenyx Resubmits Biologics License Application for UX111 AAV Gene Therapy to Treat Sanfilippo Syndrome Type A (MPS IIIA) to U.S. FDA Company expects up to six-month review period per FDA guidelines New longer-term clinical data demonstrating durable positive brain biochemical and clinical effect for as long as 8.5 years to be presented at WORLDSymposium™ 2026 NOVATO, Calif., Jan. 30, 2026 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced that it has resubmitted its Biologics License Application (BLA) seeking accelerated approval for UX111 (rebisuflig...
Ultragenyx Reports Inducement Grant Under Nasdaq Listing Rule 5635(c)(4) NOVATO, Calif., Jan. 23, 2026 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for rare and ultra-rare diseases, today reported the grant of 16,355 restricted stock units of the company’s common stock to nine newly hired non-executive officers of the company. The awards were approved by the compensation committee of the company’s board of directors and granted under the Ultragenyx Employment Inducement Pl...
Ultragenyx Provides Financial and Business Updates at J.P. Morgan Annual Healthcare Conference Preliminary 2025 total revenue of $672 million to $674 million, exceeding top end of guidance Preliminary cash and investments of approximately $735 million as of December 31, 2025 Anticipated 2026 catalysts include two potential approvals and pivotal Phase 3 data from the GTX-102 Phase 3 Aspire study for Angelman syndrome NOVATO, Calif., Jan. 12, 2026 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization ...
Ultragenyx to Present at the 44th Annual J.P. Morgan Healthcare Conference NOVATO, Calif., Jan. 07, 2026 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for serious rare and ultra-rare genetic diseases, today announced that Emil D. Kakkis, M.D., Ph.D., the company's chief executive officer and president, will present at the 44th Annual J.P. Morgan Healthcare Conference on Monday, January 12, 2026, at 10:30 AM PT. The live and archived webcast of the presentation will be acce...
Ultragenyx Completes Rolling Submission of Biologics License Application (BLA) to U.S. FDA for DTX401 AAV Gene Therapy for Glycogen Storage Disease Type Ia (GSDIa) If approved, DTX401 will be the first therapy approved to treat the underlying cause of GSDIa NOVATO, Calif., Dec. 30, 2025 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced that it has completed the rolling submission of its Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA), seeking approval for DTX401 AAV gene therapy (pariglasgene brecaparvovec) as a treatmen...
Ultragenyx Announces Phase 3 Orbit and Cosmic Results for Setrusumab (UX143) in Osteogenesis Imperfecta Neither study achieved its primary endpoint of reduction in annualized clinical fracture rate compared to placebo (Orbit) or bisphosphonates (Cosmic) Both studies achieved the secondary endpoint of improvements in bone mineral density with strong statistical significance The Company will implement significant expense reductions NOVATO, Calif., Dec. 29, 2025 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced results from the Phase 3 Orbit and Cosmic stud...
Ultragenyx Reports Inducement Grant Under Nasdaq Listing Rule 5635(c)(4) NOVATO, Calif., Dec. 19, 2025 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for rare and ultra-rare diseases, today reported the grant of 13,144 restricted stock units of the company’s common stock to nine newly hired non-executive officers of the company. The awards were approved by the compensation committee of the company’s board of directors and granted under the Ultragenyx Employment Inducement Pl...
Ultragenyx to Participate in Investor Conferences in December NOVATO, Calif., Nov. 24, 2025 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for serious rare and ultra-rare genetic diseases, today announced participation in two upcoming investor conferences. Citi’s 2025 Global Healthcare Conference (Miami, FL) Tuesday, December 2, 2025, Eric Crombez, M.D., Chief Medical Officer will participate in a fireside chat. 8th Annual Evercore ISI Healthcare Conference (Coral Gables,...
Ultragenyx Reports Inducement Grant Under Nasdaq Listing Rule 5635(c)(4) NOVATO, Calif., Nov. 21, 2025 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for rare and ultra-rare diseases, today reported the grant of 18,180 restricted stock units of the company’s common stock to 12 newly hired non-executive officers of the company. The awards were approved by the compensation committee of the company’s board of directors and granted under the Ultragenyx Employment Inducement Plan...
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