---
schema_version: "secwatch.filing_event.v1"
accession: "0001213900-26-104876"
form_type: "8-K"
ticker: "RFL"
cik: "0001713863"
company_name: "Rafael Holdings, Inc."
filed_at: "2026-09-30T11:23:27+00:00"
generated_at: "2026-10-12T13:57:22.988847+00:00"
event_type: "other_material"
sentiment: "negative"
materiality_score: 0.85
calibrated_materiality_score: 0.85
confidence: "high"
source: SEC EDGAR
---

# Rafael Holdings' Trappsol Cyclo misses Phase 3 primary endpoint; NDA still on track for Q4 2026

## Summary
- 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.

## SEC filing metadata
- accession: 0001213900-26-104876
- form_type: 8-K
- ticker: RFL
- cik: 0001713863
- company_name: Rafael Holdings, Inc.
- filed_at: 2026-09-30T11:23:27+00:00
- event_type: other_material
- sentiment: negative
- materiality_score: 0.85
- calibrated_materiality_score: 0.85
- confidence: high
- sec_items: 7.01, 2.02, 8.01, 9.01
- EDGAR index: https://www.sec.gov/Archives/edgar/data/1713863/000121390026104876/0001213900-26-104876-index.htm
- EDGAR primary document: https://www.sec.gov/Archives/edgar/data/1713863/000121390026104876/ea0307125-8k_rafael.htm

## Machine-readable alternates
- HTML: https://secwatch.observer/filing/0001213900-26-104876
- JSON: https://secwatch.observer/filing/0001213900-26-104876.json
- Plain text: https://secwatch.observer/filing/0001213900-26-104876.txt

This AI-assisted summary is a reading aid. Review the linked SEC EDGAR filing before relying on any specific claim.