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Material updates from SEC filings (8-K, 10-Q, 10-K) ranked by impact, with no firehose noise.
Departure of Directors or Certain Officers; Election of Directors; Appointment of Certain Officers; Compensatory Arrangements of Certain Officers. As reported below, at its Annual Meeting of Stockholders (the “ Annual Meeting ”) on May 14, 2026, the stockholders of Ultragenyx Pharmaceutical Inc. (“ Ultragenyx ” or the “ Company ”) approved the Third Amended and Restated 2023 Incentive Plan (the “ Third A&R 2023 Plan ”). The Third A&R 2023 Plan is described in more detail in Ultragenyx’s 2026…
Results of Operations and Financial Condition. On May 5, 2026, Ultragenyx Pharmaceutical Inc. (the “Company”) issued a press release announcing its financial results for the three months ended March 31, 2026 (the “ Press Release ”). A copy of the Press Release is furnished herewith as Exhibit 99.1 The information set forth under
Other Events On April 2, 2026, Ultragenyx Pharmaceutical Inc. (the “Company”) issued a press release announcing that the U.S. Food and Drug Administration (the “FDA”) has accepted for review the Company’s resubmitted Biologics License Application seeking accelerated approval for UX111 (rebisufligene etisparvovec) AAV9 gene therapy as a treatment for patients with Sanfilippo syndrome Type A (MPS IIIA). The FDA set a Prescription Drug User Fee Act (PDUFA) action date of September 19, 2026. Caut…
Other Events. On March 30, 2026, Ultragenyx Pharmaceutical Inc. (the “Company”) issued a press release announcing that the U.S. Food and Drug Administration (the “FDA”) has cleared the Investigational New Drug (“IND”) application for UX016, an investigational small molecule prodrug of sialic acid (“SA”) being evaluated as a substrate replacement therapy for GNE myopathy (“GNEM”). GNEM is a rare, severely debilitating, inherited neuromuscular disorder caused by mutations in the GNE gene that l…
Other Events. On March 12, 2026, Ultragenyx Pharmaceutical Inc. (the “Company”) issued a press release announcing positive results from its Phase 3 Enh3ance study of DTX301, an investigational AAV8 gene therapy for the treatment of ornithine transcarbamylase (“OTC”) deficiency. At Week 36 in the randomized, double-blind placebo-controlled period of the trial, DTX301-treated patients (n=18) demonstrated a statistically significant and clinically meaningful 18% (p=0.018) reduction in 24-hour pl…
Other Events. On February 23, 2026, Ultragenyx Pharmaceutical Inc. (the “Company”) issued a press release announcing that the U.S. Food and Drug Administration (the “FDA”) has accepted for review the Biologics License Application (the “BLA”) seeking approval of DTX401 AAV gene therapy (pariglasgene brecaparvovec) for the treatment of Glycogen Storage Disease Type Ia (GSDIa). The FDA granted the BLA Priority Review and assigned a Prescription Drug User Fee Act (PDUFA) action date of August 23,…
Costs Associated with Exit or Disposal Activities. On February 12, 2026, the Company began implementation of a strategic restructuring plan to reduce expenses (the “Restructuring”). As part of the Restructuring, the Company is implementing a 10% workforce reduction of approximately 130 employees across the Company (the “RIF”). The Company began notifying affected employees on February 12, 2026, and expects this RIF to be substantially completed in the first half of 2026. The Company estimates…
Results of Operations and Financial Condition. On February 12, 2026, Ultragenyx Pharmaceutical Inc. (the “Company”) issued a press release announcing its financial results for the three months ended December 31, 2025 and for the year ended December 31, 2025 (the “ Press Release ”). A copy of the Press Release is furnished herewith as Exhibit 99.1. The information set forth under
Other Events. On February 3, 2026, Ultragenyx Pharmaceutical Inc. (the “Company”) issued a press release announcing new long‑term data from clinical studies evaluating UX111 (rebisufligene etisparvovec), an investigational AAV9 gene therapy for Sanfilippo syndrome type A (MPS IIIA), a fatal neurodegenerative lysosomal storage disorder. The results demonstrate substantial and durable biomarker improvements and meaningful functional benefits compared with natural history, with consistent and hi…
Other Events. On January 30, 2026, Ultragenyx Pharmaceutical Inc. (the “Company”) issued a press release announcing that that it has resubmitted its Biologics License Application (“BLA”) seeking accelerated approval for UX111 (rebisufligene etisparvovec) AAV9 gene therapy as a treatment for patients with Sanfilippo syndrome type A (MPS IIIA) to the U.S. Food and Drug Administration (the “FDA” or the “Agency”). The submission contains substantial longer-term data on multiple measures of neurol…
Results of Operations and Financial Condition. On January 12, 2026, Ultragenyx Pharmaceutical Inc. (the “Company”) issued a press release (the “Press Release”) announcing preliminary unaudited fiscal year 2025 total revenue results, revenue for Crysvita® and revenue for Dojolvi®and cash and investments at 2025 fiscal year end. The Company expects to report its full year results for the 2025 fiscal year in February 2026. A copy of the Press Release is furnished herewith as Exhibit 99.1. The in…
Other Events. On December 30, 2025, Ultragenyx Pharmaceutical Inc. (the “Company”) issued a press release announcing that that it has completed the rolling submission of its Biologics License Application (“BLA”) to the U.S. Food and Drug Administration (the “FDA”) seeking approval for DTX401 AAV gene therapy (pariglasgene brecaparvovec) as a treatment for Glycogen Storage Disease Type Ia (“GSDIa”). The BLA for DTX401 is based on data from a clinical development program that includes 52 treate…
Other Events. On December 29, 2025, Ultragenyx Pharmaceutical Inc. (the “Company”) announced results from the Phase 3 Orbit and Cosmic studies for setrusumab (UX143) in Osteogenesis Imperfecta (“OI”). Neither study achieved statistical significance against the primary endpoints of reduction in annualized clinical fracture rate compared to placebo or bisphosphonates, respectively. Both studies achieved the secondary endpoints of improvements in bone mineral density (“BMD”) against comparators.…
Entry into a Material Definitive Agreement. On November 3, 2025, Ultragenyx Pharmaceutical Inc. (the “Company”) and its wholly owned subsidiary Rare Delaware Inc. (“Seller”) entered into a Royalty Purchase Agreement (the “Agreement”) with OCM LS23 Holdings LP, an investment vehicle of OMERS, pursuant to which OMERS paid $400 million in cash to Seller in consideration for the right (the “Purchased Interest”) to receive (i) an additional 25% of the future royalty payments due to the Company fro…
Results of Operations and Financial Condition. On November 4, 2025, Ultragenyx Pharmaceutical Inc. issued a press release announcing its financial results for the three months ended September 30, 2025 (the “ Press Release ”). A copy of the Press Release is furnished herewith as Exhibit 99.1 The information set forth under
Other Events. On October 30, 2025, Ultragenyx Pharmaceutical Inc. (the “Company”) issued a press release announcing that the first patient has been dosed in the Aurora study (NCT07157254) evaluating the efficacy and safety of GTX-102 (apazunersen), an investigational antisense oligonucleotide (“ASO”) for Angelman syndrome (“AS”). Data from the Aurora study will expand the population of Angelman patients treated to include both younger and older patients and those with other non-deletion AS ge…
Other Events. On September 8, 2025, Ultragenyx Pharmaceutical Inc. (the “Company”) issued a press release announcing positive longer-term results from its Phase 3 study of DTX401 AAV gene therapy for the treatment of glycogen storage disease type Ia (“GSDIa”). Previously reported 48 Week data show that patients treated with DTX401 (n=20) experienced statistically significant and clinically meaningful reductions in daily cornstarch intake compared to placebo, while maintaining glycemic control…
Other Events. On August 18, 2025, Ultragenyx Pharmaceutical Inc. (the “Company”) issued a press release announcing the initiation of a rolling submission of a Biologics License Application (“BLA”) to the U.S. Food and Drug Administration (“FDA”) seeking approval for DTX401 AAV gene therapy as a treatment for Glycogen Storage Disease Type Ia (“GSDIa”). The Company has submitted the non-clinical and clinical modules to the FDA and plans to complete the full BLA including submission of the chemi…
Results of Operations and Financial Condition. On August 5, 2025, Ultragenyx Pharmaceutical Inc. issued a press release announcing its financial results for the three months ended June 30, 2025 (the “ Press Release ”). A copy of the Press Release is furnished herewith as Exhibit 99.1 The information set forth under
Other Events. On July 31, 2025, Ultragenyx Pharmaceutical Inc. (the “Company”) issued a press release announcing that the Phase 3 Aspire study evaluating GTX-102 (apazunersen) as a treatment for Angelman Syndrome is fully enrolled, with approximately 129 participants ages four to 17 with a genetically confirmed diagnosis of full maternal UBE3A gene deletion. Enrollment in the global Phase 3 Aspire study (NCT06617429) began in December 2024. Participants are randomized 1:1 to receive GTX-102 b…
Other Events. On July 11, 2025, Ultragenyx Pharmaceutical Inc. (the “Company”) announced that the U.S. Food and Drug Administration (the “FDA”) has issued a Complete Response Letter (“CRL”) for its Biologics License Application (“BLA”) for UX111 (ABO-102) AAV gene therapy as a treatment for patients with Sanfilippo syndrome type A (MPS IIIA). In the CRL, the FDA requested that the Company provide additional information and improvements related to specific aspects of chemistry, manufacturing a…
Other Events. On July 9, 2025, Ultragenyx Pharmaceutical Inc. (the “Company”) announced that the randomized, placebo-controlled Phase 3 portion of the Orbit study evaluating UX143 (setrusumab) in pediatric and young adult patients with osteogenesis imperfecta (“OI”) is progressing toward a final analysis consistent with the original plan, around the end of the year. The Data Monitoring Committee (the “DMC”) met and informed the Company that UX143 demonstrates an acceptable safety profile and…
Other Events. On June 27, 2025, Ultragenyx Pharmaceutical Inc. (the “Company”) issued a press release announcing that it has received Breakthrough Therapy Designation from the U.S. Food and Drug Administration (the "FDA") for GTX-102 (apazunersen) as a treatment for Angelman syndrome. The FDA’s decision is based on preliminary clinical evidence including positive data from the Phase 1/2 study in 74 patients (4-17 years of age) with a full maternal UBE3A gene deletion, that showed participants…
Departure of Directors or Certain Officers; Election of Directors; Appointment of Certain Officers; Compensatory Arrangements of Certain Officers. As reported below, at its Annual Meeting of Stockholders (the “ Annual Meeting ”) on May 15, 2025, the stockholders of Ultragenyx Pharmaceutical Inc. (“ Ultragenyx ” or the “ Company ”) approved the Second Amended and Restated 2023 Incentive Plan (the “ Second A&R 2023 Plan ”). The Second A&R 2023 Plan is described in more detail in Ultragenyx’s 20…
Results of Operations and Financial Condition. On May 6, 2025, Ultragenyx Pharmaceutical Inc. issued a press release announcing its financial results for the three months ended March 31, 2025 (the “ Press Release ”). A copy of the Press Release is furnished herewith as Exhibit 99.1. The information set forth under
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