secwatch.observer — SEC 8-K summary
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Issuer:     Ultragenyx Pharmaceutical Inc. (RARE)
CIK:        0001515673
Form:       8-K
Filed at:   2024-10-03T23:59:59+00:00
Accession:  0000950170-24-112402
Event type: other_material
Sentiment:  positive
Materiality: 0.75
Item codes: 8.01
LLM model:  deepseek-v4-flash:cloud@v2

UX701 gene therapy shows clinical activity; 6 of 15 Wilson disease patients tapered off standard-of-care
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- 15 patients enrolled across 3 dose cohorts; 6 completely tapered off chelators/zinc therapy; 1 additional began tapering.
- Non-ceruloplasmin bound copper stabilized to normal levels; ceruloplasmin-copper activity increased, indicating improved ATP7b function.
- UX701 well tolerated with no unexpected treatment-emergent adverse events or significant immunologic safety events.
- Company plans to enroll a fourth cohort at moderately increased dose with optimized immunomodulation regimen to enhance efficacy.
- Dose selection for randomized placebo-controlled Stage 2 and long-term follow-up in Stage 3 are planned.

Source:
  EDGAR index:    https://www.sec.gov/Archives/edgar/data/1515673/000095017024112402/0000950170-24-112402-index.htm
  Primary doc:    https://www.sec.gov/Archives/edgar/data/1515673/000095017024112402/rare-20241003.htm
  HTML page:      https://secwatch.observer/filing/0000950170-24-112402

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