Rafael Holdings Announces Topline Results from the Pivotal Phase 3 TransportNPC™ Study of Trappsol® Cyclo™ in Niemann-Pick Disease Type C (NPC)
Rafael Holdings' pivotal Phase 3 TransportNPC study of Trappsol Cyclo in Niemann-Pick Disease Type C missed statistical significance on the primary endpoint, showing a 64% slowing of disease progression (p=0.19) at Week 96. A prespecified analysis in the ~83% of patients on background miglustat and/or leucine therapy showed a statistically significant 71% slowing of progression (p=0.046), and separate survival analyses vs. external controls suggested 85-94% mortality risk reduction. The safety profile was well tolerated with no new safety signals, though serious TEAEs were more frequent on drug (35.9% vs 16.7%) and one severe treatment-related bilateral deafness occurred.
Key figures
- Drug Name
- Trappsol® Cyclo™ (intravenous hydroxypropyl-β-cyclodextrin)
- Phase Of Trial
- Phase 3
- Nda submission
- Q4 2026
- Subgroup pvalue
- 0.046
- Patients enrolled
- 94
- Subgroup patients
- 78
- Primary endpoint pvalue
- 0.19
- Overall survival hr inpdr
- HR=0.154; 95% CI: 0.025-0.950; p=0.044
- Overall survival hr expanded
- HR=0.057; 95% CI: 0.011-0.306; p=0.0008
- Subgroup disease progression slowing
- 71%
- Primary endpoint disease progression slowing
- 64%
AI analysis
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