Ultragenyx Pharmaceutical Inc. Files 8-K

TL;DR

UGNX filed an 8-K on 12/19/24 for 'Other Events' - details TBD.

AI Summary

Ultragenyx Pharmaceutical Inc. filed an 8-K on December 19, 2024, to report other events. The filing does not contain specific details about the nature of these events, dollar amounts, or definitive dates beyond the filing date itself.

Why It Matters

This 8-K filing indicates that Ultragenyx Pharmaceutical Inc. has reported an event requiring immediate disclosure to the SEC, though the specifics are not detailed in the provided text.

Risk Assessment

Risk Level: low — The filing is a standard 8-K for 'Other Events' without immediate disclosed financial impact or significant operational changes.

Key Players & Entities

• Ultragenyx Pharmaceutical Inc. (company) — Registrant
• December 19, 2024 (date) — Filing Date

FAQ

Key Financial Figures

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

Filing Documents

• rare-20241219.htm (8-K) — 44KB
• 0000950170-24-138250.txt ( ) — 153KB
• rare-20241219.xsd (EX-101.SCH) — 23KB
• rare-20241219_htm.xml (XML) — 4KB

01 Other Events

Item 8.01 Other Events. On December 19, 2024, Ultragenyx Pharmaceutical Inc. (the "Company") issued a press release announcing the submission of a Biologics License Application ("BLA") to the U.S. Food and Drug Administration (the "FDA" or the "Agency") seeking accelerated approval for UX111 (ABO-102) AAV gene therapy as a treatment for patients with Sanfilippo syndrome type A (MPS IIIA). Earlier this year, the Company reached agreement with the Agency that cerebral spinal fluid ("CSF") heparan sulfate ("HS") can be used as a surrogate endpoint for accelerated approval based on the body of data presented by the Company along with a consortium of academics and other industry sponsors at a workshop hosted by the Reagan-Udall Foundation for the FDA in February 2024. The BLA submission for UX111 is supported by available data, including from the ongoing pivotal Transpher A study, demonstrating treatment with UX111 resulted in rapid and sustained decreased levels of HS in CSF in patients with Sanfilippo syndrome type A, and that sustained reduction in CSF HS exposure over time was correlated with improved long-term cognitive development compared to the decline observed during the same period of time in natural history data. The most frequently reported treatment-related adverse events to date were elevations in liver enzymes and the majority of these events were mild (Grade 1) or moderate (Grade 2) in severity and all resolved. 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 clinical benefit, tolerability and safety of

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: December 19, 2024 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