Trappsol Cyclo - (TransportNPC)

June 10, 2026: Rafael Holdings, Inc. (NYSE: RFL) reported that the last patient completed the final 96-week visit in its pivotal Phase 3 TransportNPC™ trial evaluating Trappsol® Cyclo™ for Niemann-Pick Disease Type C (NPC). The randomized, controlled study enrolled 94 patients across 27 sites in 13 countries; an additional 10 patients enrolled in a single-arm pediatric sub-study (newborn to 3 years) under an adopted Pediatric Investigational Plan (PIP). The company expects topline data from the main cohort in the second half of calendar 2026.

The company said it presented WORLDSymposium 2026 data from the 0–3-year-old sub-study: 80% of patients’ CGI scores improved or remained stable, and no serious adverse events were considered related to Trappsol® Cyclo™.

Regulatory: Rafael said it has completed a pre-NDA meeting with FDA and expects to submit an NDA in the second half of 2026. The company also said FDA acknowledged that its U.S. Expanded Access Program (EAP) can proceed.