Ultragenyx Pharmaceutical Inc. Files 8-K

TL;DR

UGNX filed an 8-K, standard SEC reporting.

AI Summary

Ultragenyx Pharmaceutical Inc. filed an 8-K on April 15, 2024, to report other events and financial statements. The filing does not contain specific details about new events or financial figures within the provided text, but it serves as a notification to the SEC.

Why It Matters

This filing indicates Ultragenyx is fulfilling its reporting obligations with the SEC, which is standard practice for publicly traded companies.

Risk Assessment

Risk Level: low — This is a routine SEC filing and does not appear to contain any new material information or significant events.

Key Players & Entities

• Ultragenyx Pharmaceutical Inc. (company) — Registrant
• April 15, 2024 (date) — Date of earliest event reported
• 60 Leveroni Court (location) — Principal Executive Offices Address
• Novato, California (location) — Principal Executive Offices City and State
• 94949 (location) — Principal Executive Offices Zip Code
• 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-20240415.htm (8-K) — 60KB
• 0000950123-24-003302.txt ( ) — 171KB
• rare-20240415.xsd (EX-101.SCH) — 23KB
• rare-20240415_htm.xml (XML) — 4KB

01 Other Events

Item 8.01 Other Events. On April 15, 2024, Ultragenyx Pharmaceutical Inc. (the "Company") announced new data from the Phase 1/2 study of GTX-102 for the treatment of Angelman syndrome. Patients in Expansion Cohorts A & B treated with a set dose and regimen of GTX-102 showed rapid and clinically meaningful improvement across multiple domains consistent with or exceeding Dose-escalation Cohorts 4-7 data at Day 170. Treatment of the Dose-escalation Cohorts 4-7 showed long-term increasing and sustained clinical benefit far exceeding Natural History data at Day 758. These data will be discussed in more detail in a corporate presentation being hosted by the Company on April 15, 2024 at 8:00 a.m. ET and will also be presented by Kemi Olugemo, M.D., FAAN at the 76th Annual American Academy of Neurology Meeting ("AAN") in Denver on Tuesday, April 16, 2024. New Expansion Cohorts A & B data include Day 170 results on 24 patients, and long-term Dose-escalation Cohorts 4–7 data include up to Day 758 results on 15 patients. Expansion Cohorts at Day 170: Cognition assessed by Bayley-4 showed rapid and clinically significant improvement compared with Natural History data. Day 170 data were consistent with the treatment benefit observed in the Dose-escalation Cohorts at a similar timepoint. Behavior assessed by the Angelman Severity Assessment (ASA) showed rapid improvement exceeding the treatment benefit observed in the Dose-escalation Cohorts at Day 170. Hyperactivity and noncompliance assessed by the Aberrant Behavior Checklist-Community (ABC-C) showed rapid and clinically significant improvement at Day 170 compared with Natural History data, providing further insight into one of the most commonly reported behavioral issues. Sleep assessed by ASA showed rapid and clinically meaningful improvement exceeding treatment benefit observed in the Dose-escalation Cohorts at Day 170. Receptive communication assessed by Bayley-4 showed rapid improvement compared with Natura

01 Financial Statements and Exhibits

Item 9.01 Financial Statements and Exhibits. (d) Exhibits Exhibit No. Description 104 The cover page from the Company's Current Report on Form 8-K dated April 15, 2024 formatted in Inline XBRL.

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: April 15, 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