secwatch.observer — SEC 8-K summary
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Issuer:     uniQure N.V. (QURE)
CIK:        0001590560
Form:       8-K
Filed at:   2023-06-22T23:59:59+00:00
Accession:  0001104659-23-073817
Event type: other_material
Sentiment:  positive
Materiality: 0.80
Item codes: 7.01, 8.01, 9.01
LLM model:  deepseek-v4-flash:cloud@v2

uniQure reports $100M HEMGENIX milestone; AMT-130 shows preserved function at 24 months in HD trial
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- $100M milestone achieved from CSL Behring for first US commercial sale of HEMGENIX (hemophilia B gene therapy); payment due within 30 days.
- AMT-130 well-tolerated; no treatment-emergent adverse events led to discontinuation. DSMB recommends continuing development.
- Low-dose cohort showed preserved function at 24 months; TMS improved 1.8 points vs natural history, TFC 0.8, cUHDRS 0.9.
- Mean CSF NfL 12.9% below baseline at 24 months in low-dose cohort vs predicted 22.9% increase in natural history.
- Plan to complete EU enrollment in Q3 2023, start third US cohort in H2 2023, and hold regulatory interactions by Q1 2024.

Source:
  EDGAR index:    https://www.sec.gov/Archives/edgar/data/1590560/000110465923073817/0001104659-23-073817-index.htm
  Primary doc:    https://www.sec.gov/Archives/edgar/data/1590560/000110465923073817/tm2319377d1_8k.htm
  HTML page:      https://secwatch.observer/filing/0001104659-23-073817

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