secwatch.observer — SEC 8-K summary
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Issuer:     ROCKET PHARMACEUTICALS, INC. (RCKT)
CIK:        0001281895
Form:       8-K
Filed at:   2024-11-18T23:59:59+00:00
Accession:  0001140361-24-047166
Event type: other_material
Sentiment:  positive
Materiality: 0.85
Item codes: 7.01, 8.01, 9.01
LLM model:  deepseek-v4-flash:cloud@v2

Rocket Pharma reports positive long-term Phase 1 Danon Disease data; PKD program enrollment delayed to 2025
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- All evaluable Danon disease patients showed cardiac LAMP2 protein expression at 12 months, sustained up to 60 months, and ≥10% LV mass index reduction sustained up to 54 months.
- Median reduction in LV mass index 24%; cardiac biomarkers cTnI down 84%, NTproBNP down 57%; NYHA class improved II to I; KCCQ score improved 27 points.
- RP-A501 generally well tolerated with manageable AEs; one patient with baseline LVEF<40% required heart transplant 5 months post-treatment.
- Phase 2 pivotal trial for Pyruvate Kinase Deficiency (RP-L301) design agreed with FDA; enrollment not started, anticipates resuming in 2025.

Source:
  EDGAR index:    https://www.sec.gov/Archives/edgar/data/1281895/000114036124047166/0001140361-24-047166-index.htm
  Primary doc:    https://www.sec.gov/Archives/edgar/data/1281895/000114036124047166/ef20038805_8k.htm
  HTML page:      https://secwatch.observer/filing/0001140361-24-047166

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