Rafael Holdings says Trappsol Cyclo Phase 3 study in Niemann-Pick Type C results
Rafael Holdings reported topline results from a pivotal Phase 3 Transportnpc study of Trappsol Cyclo in Niemann-Pick disease Type C, saying it was well-tolerated but did not achieve statistical significance on the primary endpoint at Week 96. The company said it is on track to submit an NDA in Q4 2026.