secwatch.observer — SEC 8-K summary
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Issuer:     Ultragenyx Pharmaceutical Inc. (RARE)
CIK:        0001515673
Form:       8-K
Filed at:   2025-07-31T23:59:59+00:00
Accession:  0000950170-25-100918
Event type: other_material
Sentiment:  neutral
Materiality: 0.65
Item codes: 8.01
LLM model:  deepseek-v4-flash:cloud@v2

Ultragenyx fully enrolls Phase 3 Aspire study (129 patients) for GTX-102 in Angelman Syndrome
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- Phase 3 Aspire study fully enrolled with ~129 patients ages 4-17 with maternal UBE3A deletion.
- Participants randomized 1:1 to GTX-102 intrathecal injection or sham comparator for 48 weeks; sham can cross over after Week 48.
- Primary endpoint: Bayley-4 cognitive raw score improvement; key secondary: MDRI across five domains.
- Study completion expected H2 2026; company plans to move to topline data and regulatory submission.

Source:
  EDGAR index:    https://www.sec.gov/Archives/edgar/data/1515673/000095017025100918/0000950170-25-100918-index.htm
  Primary doc:    https://www.sec.gov/Archives/edgar/data/1515673/000095017025100918/rare-20250731.htm
  HTML page:      https://secwatch.observer/filing/0000950170-25-100918

License: Source filings: public domain (SEC EDGAR). Summaries (headline + bullets): CC-BY-4.0; attribute https://secwatch.observer