secwatch.observer — SEC 8-K summary
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Issuer:     Ultragenyx Pharmaceutical Inc. (RARE)
CIK:        0001515673
Form:       8-K
Filed at:   2024-12-19T23:59:59+00:00
Accession:  0000950170-24-138467
Event type: other_material
Sentiment:  neutral
Materiality: 0.75
Item codes: 8.01
LLM model:  deepseek-v4-flash:cloud@v2

First patient dosed in Ultragenyx Phase 3 Aspire study of GTX-102 for Angelman syndrome
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- Global Phase 3 Aspire study will enroll ~120 children ages 4-17 with full maternal UBE3A deletion.
- Patients randomized 1:1 to GTX-102 intrathecal injections or sham comparator for 48-week primary analysis.
- Primary endpoint: cognition improvement (Bayley-4 cognitive raw score); key secondary: Multi-domain Responder Index.
- Active group receives three monthly 8 mg loading doses then quarterly maintenance up to 14 mg; sham crossover after Week 48.

Source:
  EDGAR index:    https://www.sec.gov/Archives/edgar/data/1515673/000095017024138467/0000950170-24-138467-index.htm
  Primary doc:    https://www.sec.gov/Archives/edgar/data/1515673/000095017024138467/rare-20241219.htm
  HTML page:      https://secwatch.observer/filing/0000950170-24-138467

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