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

Rafael Holdings, Inc.

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.

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