Rafael Holdings completes Phase 3 TransportNPC™ trial for Niemann-Pick Disease Type C

Rafael Holdings (NYSE:RFL) announced that the last patient has completed the final 96-week study visit in its pivotal Phase 3 TransportNPC™ trial evaluating Trappsol® Cyclo™ for the treatment of Niemann-Pick Disease Type C (NPC).

The global study included 94 patients across 27 sites in 13 countries, making it the most comprehensive controlled pivotal trial to date in NPC in terms of patient numbers, geographic footprint, duration, and clinical outcomes.

In addition, a single-arm pediatric sub-study enrolled 10 patients from birth to age three under the Pediatric Investigational Plan (PIP).

Data from this sub-study, presented at WORLDSymposium 2026, showed that 80% of patients’ Clinical Global Impression (CGI) scores either improved or remained stable, with no serious adverse events attributed to the study drug.

The TransportNPC™ study positions Rafael Holdings to potentially deliver a life-changing therapy for NPC, a rare and devastating lysosomal storage disorder.

Topline results from the main study cohort are expected later this year.