Ultragenyx Pharmaceutical Inc. Files 8-K

TL;DR

UGNX filed an 8-K today, more details to come.

AI Summary

Ultragenyx Pharmaceutical Inc. filed an 8-K on October 7, 2024, 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 the public.

Risk Assessment

Risk Level: low — The filing is a standard disclosure and does not contain information that inherently increases risk.

Key Players & Entities

• Ultragenyx Pharmaceutical Inc. (company) — Registrant

FAQ

Key Financial Figures

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

Filing Documents

• rare-20241007.htm (8-K) — 42KB
• 0000950170-24-113085.txt ( ) — 151KB
• rare-20241007.xsd (EX-101.SCH) — 23KB
• rare-20241007_htm.xml (XML) — 4KB

01 Other Events

Item 8.01 Other Events. On October 7, 2024, Ultragenyx Pharmaceutical Inc. (the "Company") announced that it had received Breakthrough Therapy Designation from the U.S. Food and Drug Administration (the "FDA") for setrusumab (UX143) as a treatment to reduce the risk of fracture associated with osteogenesis imperfecta (OI) Type I, III, or IV in patients two years of age and older. The FDA's decision is based on preliminary clinical evidence including the positive 14-month results from the Phase 2 portion of the Orbit study, which demonstrated a rapid and clinically meaningful decrease in fracture rate in patients, and from the completed Phase 2b Asteroid study. Breakthrough Therapy Designation aims to expedite the development and review of drugs that are intended to treat serious or life-threatening diseases and whose preliminary clinical evidence indicates that the drug may demonstrate substantial improvement on one or more clinically significant endpoints over existing therapies. 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 UX143 and the corresponding impact on patients and timing for clinical development or regulatory review of UX143. Such forward-looking statements involve substantial risks and uncertainties that could cause the Company's clinical development programs, collaboration with third parties, future results, performance or achievements to differ significantly from those expressed or implied by the forward-looking statements. Such risks and uncertainties inc

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: October 7, 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