Ultragenyx Pharmaceutical Inc. Files 8-K

TL;DR

UGNX filed an 8-K, something happened, details TBD.

AI Summary

Ultragenyx Pharmaceutical Inc. filed an 8-K on October 3, 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 filing indicates a material event has occurred for Ultragenyx Pharmaceutical Inc., requiring disclosure to investors, though the specific details are not yet elaborated.

Risk Assessment

Risk Level: medium — The filing of an 8-K often signifies important corporate developments, but the lack of specific details in this initial report creates uncertainty.

Key Players & Entities

• Ultragenyx Pharmaceutical Inc. (company) — Registrant
• October 03, 2024 (date) — Date of earliest event reported

FAQ

Key Financial Figures

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

Filing Documents

• rare-20241003.htm (8-K) — 44KB
• 0000950170-24-112402.txt ( ) — 153KB
• rare-20241003.xsd (EX-101.SCH) — 23KB
• rare-20241003_htm.xml (XML) — 4KB

01 Other Events

Item 8.01 Other Events. On October 3, 2024, Ultragenyx Pharmaceutical Inc. (the "Company") reported that the Phase 1/2/3 Cyprus2+ study of its UX701 gene therapy has demonstrated meaningful clinical activity as well as improvements in copper metabolism in Stage 1. Multiple responders have completely tapered off of standard-of-care treatment with responses seen in all three dose cohorts. The Company plans to enroll an additional cohort in Stage 1 at a moderately increased dose and with an optimized immunomodulation regimen to enhance the efficiency and efficacy of the gene therapy, with the objective of having the majority of patients come off of standard-of-care treatment before selecting a dose for the randomized placebo-controlled stage of the study. In Stage 1, 15 patients were enrolled into three sequential dosing cohorts and followed for at least 24 weeks. Six of the patients have completely tapered off of standard-of-care treatment with chelators and/or zinc therapy, and a seventh patient has begun to taper as of the data cut-off date in August. In patients who have tapered off standard-of-care, non-ceruloplasmin bound copper (NCC) has stabilized to normal, healthy levels. In some patients, there were increases in ceruloplasmin-copper activity consistent with improved ATP7b function. From a safety perspective, UX701 has been well tolerated, with no unexpected related treatment emergent adverse events and no significant immunologic safety events as of the data cut-off. The Company will be submitting a protocol amendment for the additional cohort at a moderately increased dose and with an optimized immunomodulation regimen to optimize delivery efficiency and efficacy for the AAV vector. Phase 1/2/3 Cyprus2+ Study Design This study evaluating UX701 for the potential treatment of Wilson disease is designed with three stages. During the first stage (Stage 1), the safety and efficacy of multiple dose levels of UX701 will be evaluated and a dose will be selec

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 3, 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