Rafael Holdings, Inc. (RFL) Form 8-K — Sep 30, 2026
Rafael Holdings announced topline results from the pivotal Phase 3 TransportNPC study of Trappsol Cyclo in 94 patients with Niemann-Pick Disease Type C. The drug missed statistical significance on the primary endpoint but showed a directionally favorable 64% slowing of disease progression (p=0.19). A prespecified analysis in the roughly 83% of patients on background miglustat and/or leucine therapy was statistically significant, showing 71% slowing of progression (p=0.046), and separate external-control analyses suggested 85-94% mortality reductions. The safety profile was consistent with prior studies with no new safety signals.
Key figures
- Patients enrolled
- 94
- Nda submission timing
- Q4 2026
- Primary endpoint p value
- p=0.19 (not significant)
- Survival analysis hr inpdr
- HR=0.154 (85% mortality reduction, p=0.044)
- Treatment serious teae rate
- 35.9% vs 16.7% placebo
- Prespecified subgroup p value
- p=0.046 (n=78, ~83% of study population)
- Survival analysis hr expanded
- HR=0.057 (94% mortality reduction, p=0.0008)
- Primary endpoint treatment effect
- 64% slowing of disease progression (LS mean change 0.46 vs 1.28)
- Prespecified subgroup treatment effect
- 71% slowing of disease progression
AI analysis
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