Ultragenyx Pharmaceutical Inc. Files 8-K

TL;DR

UGNX filed an 8-K, but no details yet on what's up.

AI Summary

Ultragenyx Pharmaceutical Inc. filed an 8-K on September 8, 2025, to report other events. The filing does not contain specific details about the nature of these events, dollar amounts, or new agreements.

Why It Matters

This filing indicates a material event has occurred for Ultragenyx Pharmaceutical Inc. that requires disclosure to investors.

Risk Assessment

Risk Level: low — The filing is a standard 8-K for 'Other Events' and does not disclose any specific negative or positive information that would immediately impact risk.

Key Players & Entities

• Ultragenyx Pharmaceutical Inc. (company) — Registrant
• September 08, 2025 (date) — Date of Report

FAQ

Key Financial Figures

• $0.001 — nge on which registered Common Stock, $0.001 par value RARE Nasdaq Global Select

Filing Documents

• rare-20250908.htm (8-K) — 59KB
• 0000950170-25-113553.txt ( ) — 162KB
• rare-20250908.xsd (EX-101.SCH) — 23KB
• rare-20250908_htm.xml (XML) — 4KB

01

Item 8.01 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. At Week 96, even greater reductions in cornstarch were observed with maintained low levels of hypoglycemia, improved levels of euglycemia and improved fasting tolerance. The data were presented at the International Congress of Inborn Errors of Metabolism 2025 in Kyoto, Japan. Participants achieved statistically significant and clinically meaningful reductions in cornstarch while maintaining glycemic control As previously reported, the study met its primary endpoint with patients treated with DTX401 (n=20) experiencing a mean reduction in cornstarch of 41% at Week 48 compared to 10% reduction in the placebo group (n=24) (p < 0.0001). Data at Week 96 showed even greater improvements, with both the DTX401 group (n=20) and the crossover group (n=19) achieving a mean reduction in daily cornstarch intake of 61% from baseline. Participants in both groups also experienced statistically significant improvements in other cornstarch-related endpoints. At Week 96, the DTX401 group saw an increased mean reduction in nighttime cornstarch of 70% and the crossover group saw a mean reduction of 75%. Two-thirds of participants across both groups eliminated at least one nighttime cornstarch dose following treatment with DTX401. Importantly, participants maintained low levels of hypoglycemia and improved levels in euglycemic range (70-120 mg/dL) throughout the second year of the study despite substantial reductions in daily cornstarch intake. Participants dosed with DTX401 als

SIGNATURES

SIGNATURES Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned hereunto duly authorized. Ultragenyx Pharmaceutical Inc Date: September 8, 2025 By: /s/ Howard Horn Howard Horn Executive Vice President, Chief Financial Officer, Corporate Strategy

View Full Filing

View this 8-K filing on SEC EDGAR