secwatch.observer — SEC 8-K summary
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Issuer:     Rafael Holdings, Inc. (RFL)
CIK:        0001713863
Form:       8-K
Filed at:   2026-09-30T11:23:27+00:00
Accession:  0001213900-26-104876
Event type: other_material
Sentiment:  negative
Materiality: 0.85
Item codes: 7.01, 2.02, 8.01, 9.01
LLM model:  deepseek-v4.1-flash:cloud@v2

Rafael Holdings' Trappsol Cyclo misses Phase 3 primary endpoint; NDA still on track for Q4 2026
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- Phase 3 TransportNPC (CTD-TCNPC-301) in 94 NPC patients: Trappsol Cyclo slowed progression 64% on 4DNPCSS (LS mean 0.46 vs 1.28; difference -0.81; p=0.19), missing statistical significance.
- Prespecified analysis of 78 patients on background miglustat and/or leucine (~83% of study) hit significance: 71% slowing (0.46 vs 1.57; difference -1.11; 95% CI -2.19 to -0.02; p=0.046).
- Safety: TEAEs 93.8% Trappsol vs 100% placebo; serious TEAEs 35.9% vs 16.7%, but treatment-related serious TEAEs similar at 3.1% vs 3.3%; one severe bilateral deafness case.
- Overall survival: infantile-onset patients (n=41) showed 85% mortality risk reduction vs 93 matched INPDR controls (HR=0.154, p=0.044); 94% reduction vs expanded controls (HR=0.057, p=0.0008).
- NDA submission remains on track for Q4 2026 following a pre-NDA FDA meeting; full results to be presented at an upcoming medical meeting.

Source:
  EDGAR index:    https://www.sec.gov/Archives/edgar/data/1713863/000121390026104876/0001213900-26-104876-index.htm
  Primary doc:    https://www.sec.gov/Archives/edgar/data/1713863/000121390026104876/ea0307125-8k_rafael.htm
  HTML page:      https://secwatch.observer/filing/0001213900-26-104876

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