Rafael Holdings says Phase 3 TransportNPC trial of Trappsol Cyclo misses primary endpoint in Niemann-Pick type C
RFL•Rafael Holdings said its Phase 3 TransportNPC trial of Trappsol Cyclo missed its primary endpoint, though results favored the drug over placebo on disease progression. A prespecified subgroup showed a statistically significant benefit, and the company maintained its plan to file a U.S. NDA in the fourth quarter of 2026.
1. Trial results
Rafael Holdings reported topline Phase 3 results for Trappsol Cyclo in Niemann-Pick disease type C. The trial missed its primary endpoint, although results favored Trappsol Cyclo over placebo on disease progression. A prespecified subgroup of patients taking background miglustat and/or leucine showed a statistically significant benefit. Safety was broadly consistent with prior studies, with no new safety signals reported; the company said it plans to file a U.S. NDA in the fourth quarter of 2026.
