Rafael Holdings Completes Phase 3 NPC Study With 94 Patients, NDA Submission Planned
RFL•Rafael Holdings completed its pivotal Phase 3 TransportNPC study for Trappsol Cyclo, enrolling 94 patients across 27 sites in 13 countries and 10 pediatric cases. Topline results and NDA submission are expected in the second half of 2026 following FDA pre-NDA alignment and Expanded Access Program clearance.
1. Completion of Phase 3 TransportNPC Study
Rafael Holdings has concluded the final 96-week visit of its pivotal Phase 3 TransportNPC trial of Trappsol Cyclo, studying 94 patients at 27 sites in 13 countries. A single-arm pediatric sub-study enrolled 10 newborn to three-year-old patients, with 80% showing stable or improved CGI scores and no drug-related serious adverse events.
2. Regulatory Milestones and Timeline
The company held a pre-NDA meeting with the FDA and received clearance to launch its US Expanded Access Program. Topline efficacy and safety results from the main cohort are expected in the second half of 2026, with an NDA submission slated for the same period.
3. Commercial and Strategic Outlook
NPC represents a high-unmet-need rare disease market, positioning Rafael Holdings to potentially transition into a commercial-stage biotechnology firm. Successful approval and launch of Trappsol Cyclo could unlock substantial long-term value for shareholders.
