secwatch.observer — SEC 8-K summary
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Issuer:     Ultragenyx Pharmaceutical Inc. (RARE)
CIK:        0001515673
Form:       8-K
Filed at:   2024-05-30T23:59:59+00:00
Accession:  0000950170-24-066553
Event type: other_material
Sentiment:  positive
Materiality: 0.85
Item codes: 8.01, 9.01
LLM model:  deepseek-v4-flash:cloud@v2

Ultragenyx Phase 3 DTX401 gene therapy meets primary endpoint in GSDIa
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- DTX401 reduced daily cornstarch intake by 41.3% vs 10.3% for placebo at Week 48 (p<0.0001).
- 68% of DTX401 patients achieved ≥30% reduction; 37% achieved ≥50% reduction.
- Secondary endpoint met: 1.1 fewer cornstarch doses/day vs 0.2 for placebo (p=0.0011).
- Safety profile acceptable; no dorsal root ganglion toxicity or thrombotic microangiopathy observed.
- Company plans to discuss results with regulators and submit marketing application in 2025.

Source:
  EDGAR index:    https://www.sec.gov/Archives/edgar/data/1515673/000095017024066553/0000950170-24-066553-index.htm
  Primary doc:    https://www.sec.gov/Archives/edgar/data/1515673/000095017024066553/rare-20240530.htm
  HTML page:      https://secwatch.observer/filing/0000950170-24-066553

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