Rafael Holdings announces topline results from pivotal Phase 3 TransportNPC study
RFL•Rafael Holdings said Trappsol Cyclo did not achieve statistical significance on the primary endpoint at week 96, while reporting a well-tolerated safety profile with no new safety signals; it remains on track to submit an NDA in Q4 2026.
1. Phase 3 results
Rafael Holdings announced topline results from the pivotal Phase 3 TransportNPC study of Trappsol Cyclo in Niemann-Pick disease type C. The treatment did not achieve statistical significance on the primary endpoint at week 96, and the company reported a well-tolerated safety profile with no new safety signals.
2. NDA timeline
Rafael said it remains on track to submit an NDA for Trappsol Cyclo in Q4 2026.
