Ultragenyx Pharmaceutical Inc. Files 8-K

TL;DR

UGNX filed an 8-K, something happened but details are scarce.

AI Summary

Ultragenyx Pharmaceutical Inc. filed an 8-K on August 18, 2025, to report other events. The filing does not contain specific details about the nature of these events, dollar amounts, or other parties involved.

Why It Matters

This filing indicates a material event has occurred for Ultragenyx Pharmaceutical Inc. that requires disclosure to the public, though the specifics are not yet detailed.

Risk Assessment

Risk Level: low — The filing is a standard disclosure form without immediate negative or positive financial implications detailed within the provided text.

Key Players & Entities

• Ultragenyx Pharmaceutical Inc. (company) — Registrant
• August 18, 2025 (date) — Date of Report
• 415-483-8800 (phone_number) — Registrant's Telephone Number

FAQ

Key Financial Figures

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

Filing Documents

• rare-20250818.htm (8-K) — 50KB
• 0000950170-25-109570.txt ( ) — 150KB
• rare-20250818.xsd (EX-101.SCH) — 23KB
• rare-20250818_htm.xml (XML) — 4KB

01

Item 8.01 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 chemistry, manufacturing and controls ("CMC") module in the fourth quarter of 2025. The BLA for DTX401 includes the previously disclosed 96-week data from the randomized, placebo-controlled Phase 3 study that demonstrated patients had even greater reductions in total daily cornstarch at their last visit compared to baseline in both the ongoing DTX401 group (-60%) and the Crossover Placebo to DTX401 group (-64%) when compared to the 48-week data. It will also include updates to proactively respond to related FDA observations identified in the UX111 complete response letter in the CMC section and at the Company's gene therapy manufacturing facilities. Cautionary Note Regarding Forward-Looking Statements This Current Report on Form 8-K contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. These statements may be identified by the use of words such as, but not limited to, "anticipates," "continue," "will," or other similar terms or expressions that concern the Company's expectations, plans and intentions. Forward-looking statements include, without limitation, statements regarding the future operating results and financial performance, business plans and objectives for DTX401, expectations regarding the tolerability and safety of DTX401, expectations regarding the adequacy of clinical data to support the marketing application and approval of DTX401, the Company's intent to file, and potential timing and success

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: August 18, 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