Ultragenyx Pharmaceutical Inc. Files 8-K

TL;DR

UGNX filed an 8-K, likely containing important corporate updates.

AI Summary

Ultragenyx Pharmaceutical Inc. filed an 8-K on May 30, 2024, to report other events and financial statements. The filing does not detail specific transactions or financial results but serves as a notification of these items being filed.

Why It Matters

This filing indicates that Ultragenyx has submitted important corporate and financial information to the SEC, which may include updates on business operations or financial performance.

Risk Assessment

Risk Level: low — The filing is a standard procedural document and does not disclose new material risks or events.

Key Players & Entities

• Ultragenyx Pharmaceutical Inc. (company) — Registrant
• May 30, 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-20240530.htm (8-K) — 49KB
• 0000950170-24-066553.txt ( ) — 159KB
• rare-20240530.xsd (EX-101.SCH) — 23KB
• rare-20240530_htm.xml (XML) — 4KB

01 Other Events

Item 8.01 Other Events. On May 30, 2024, Ultragenyx Pharmaceutical Inc. (the "Company") announced positive topline results from its Phase 3 GlucoGene study (NCT05139316) evaluating DTX401, an investigational gene therapy for the treatment of patients aged eight years and older with Glycogen Storage Disease Type Ia ("GSDIa"). The study achieved its primary endpoint, demonstrating that treatment with DTX401 resulted in a statistically significant and clinically meaningful reduction in daily cornstarch intake compared with placebo at Week 48. The mean percent reduction was 41.3% in the DTX401 group (n=20) compared with 10.3% in the placebo group (n=24) at Week 48 (p<0.0001). Across patients treated with DTX401, the mean reduction in cornstarch continued to decline over the 48-week period. In the treatment group, all patients achieved a reduction in cornstarch, with 68% achieving 30% reduction and 37% achieving 50% reduction compared to the placebo group, which achieved the same reductions in 13% and 4% of patients, respectively, at Week 48. The study also successfully met key secondary endpoints of reduction in the number of cornstarch doses per day and maintenance of glucose control at Week 48. Treatment with DTX401 resulted in a mean reduction of 1.1 cornstarch doses per day in the DTX401 treatment group compared with a mean reduction of 0.2 in the placebo group (p=0.0011). Patients in the DTX401 group also showed significant improvement in both frequency and quantity of nighttime cornstarch dosing compared with the placebo group. This blinded study established non-inferiority (p<0.0001) of glucose control between the study groups while the treatment group significantly reduced daily cornstarch intake. The Patient Global Impression of Change ("PGIC") at Week 48 showed a median score of 2.0 (moderately improved) for the DTX401 treatment group and 1.0 (minimally improved) for the placebo group (p=0.132). Moderately or higher improved PGIC scores correlated with

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 May 30, 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: May 30, 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