Rafael Reports Phase 3 Trappsol Cyclo Data In NPC

(RTTNews) - Wednesday, Rafael Holdings (RFL) reported topline results from the pivotal Phase 3 TransportNPC study evaluating Trappsol Cyclo in patients with Niemann-Pick Disease Type C (NPC). Trappsol Cyclo is an investigational intravenous hydroxypropyl-ß-cyclodextrin being developed for the treatment of NPC, a rare and progressive genetic disorder. The study showed a directionally favorable treatment effect on the primary endpoint, with Trappsol Cyclo slowing disease progression by 64% at Week 96 compared with placebo, although the result did not reach statistical significance.