secwatch.observer — SEC 8-K summary
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Issuer:     Ultragenyx Pharmaceutical Inc. (RARE)
CIK:        0001515673
Form:       8-K
Filed at:   2024-11-12T23:59:59+00:00
Accession:  0000950170-24-124458
Event type: other_material
Sentiment:  positive
Materiality: 0.85
Item codes: 8.01
LLM model:  deepseek-v4-flash:cloud@v2

Ultragenyx reports positive Phase 1/2 data for GTX-102 in Angelman syndrome; Phase 3 Aspire study well powered
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- Week 48 Bayley-4 Cognition GSV mean change +6.7 vs minimally important difference +5 (n=40).
- Phase 3 primary endpoint Bayley-4 Raw score mean change +10.9 from baseline.
- Study >95% powered to detect treatment effect even with up to 3x higher sham response.
- MDRI net response +2.0 (p<0.0001); 80% of patients achieved net improvement in at least one domain.
- Phase 3 Aspire will enroll ~120 patients with full maternal UBE3A deletion; 48-week primary analysis.

Source:
  EDGAR index:    https://www.sec.gov/Archives/edgar/data/1515673/000095017024124458/0000950170-24-124458-index.htm
  Primary doc:    https://www.sec.gov/Archives/edgar/data/1515673/000095017024124458/rare-20241109.htm
  HTML page:      https://secwatch.observer/filing/0000950170-24-124458

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